Posts

Posted on

Lilly’s Jaypirca (pirtobrutinib) significantly reduced the risk of disease progression or death by 45% when added to a venetoclax time-limited regimen in people with previously treated CLL/SLL

PR Newswire

BRUIN CLL-322 is the first Phase 3 study to demonstrate superiority over a venetoclax-containing control arm in CLL, and, with
the majority of
patients previously treated with a covalent BTK inhibitor, reflects current practice patterns

These data will be highlighted in a late-breaking oral presentation at the 2026 European Hematology Association (EHA) Annual Meeting

INDIANAPOLIS, June 14, 2026 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) today announced results from the Phase 3 BRUIN CLL-322 clinical trial of Jaypirca (pirtobrutinib), a non-covalent Bruton tyrosine kinase (BTK) inhibitor, plus venetoclax and rituximab versus venetoclax and rituximab in patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL). The study met its primary endpoint of independent review committee (IRC)-assessed progression-free survival (PFS), demonstrating that the addition of pirtobrutinib to a two-year venetoclax plus rituximab regimen reduced the risk of disease progression or death by 45% (HR=0.55 [95% CI, 0.40-0.75]; p=0.0001).

These data will be highlighted in a late-breaking oral presentation at the 2026 European Hematology Association (EHA) Annual Meeting taking place in Stockholm, Sweden, as well as featured in the meeting’s press program.

“These results from BRUIN CLL-322 show that the addition of pirtobrutinib as part of a time-limited regimen further enhanced an already effective treatment and extended the duration of remission for patients with previously treated CLL. Importantly, the study provides the first robust evidence for such an approach in patients who received a prior BTK inhibitor,” said Matthew S. Davids, M.D., M.M.Sc., Chief of the Division of Lymphoma at Dana-Farber Cancer Institute, who is the lead author on the study. “Time-limited regimens are an important option in CLL care and provide patients with meaningful treatment-free intervals. In the context of the modern CLL treatment landscape, where many patients may only receive two lines of therapy, these results speak to the potential benefits that improving second-line therapy can have. Our study has the potential to establish a new standard of care in this population.”

BRUIN CLL-322 enrolled 639 relapsed or refractory patients, with 79.8% having prior covalent BTK inhibitor exposure, who were randomized 1:1 to receive pirtobrutinib plus venetoclax and rituximab (PVR, n=321) or venetoclax and rituximab alone (VR, n=318). Patients in the PVR arm received three cycles of pirtobrutinib and the first three cycles of rituximab before venetoclax was introduced. The efficacy results are based on a Feb. 2, 2026 data cutoff. At a median follow-up of 27.3 months, the primary endpoint of IRC-assessed PFS was significantly improved with the addition of pirtobrutinib to VR compared to VR alone (HR=0.55 [95% CI, 0.40-0.75]; p=0.0001). Median PFS in the PVR arm was not reached (95% CI, 43.3-NE), versus 39.7 months (95% CI, 35.9-NE) in the VR arm. The PFS results were consistent across prespecified subgroups, including patients with prior covalent BTK inhibitor exposure (PVR: not reached [95% CI, 41.5-NE] versus VR: 36.2 months [95% CI, 33.2-NE]), those who discontinued prior covalent BTK inhibitor due to progressive disease (PVR: 43.3 months [95% CI, 39.2-NE] versus VR: 33.2 months [95% CI, 28.3-37.5]), as well as those with high-risk features such as unmutated IGHV, TP53 mutation and/or 17p deletion, and/or complex karyotype. In an exploratory analysis of second-line patients whose disease progressed after a first-line covalent BTK inhibitor, the median PFS was not reached (95% CI, 30.1-NE) in the PVR arm and was 28.3 months (95% CI, 20.5-NE) in the VR arm (HR=0.32 [95% CI, 0.14-0.73]), with 24-month PFS rates of 88% (95% CI, 75.7-94.6) and 52% (95% CI, 34.7-66.2), respectively, and consistent benefit was observed regardless of the specific prior covalent BTK inhibitor received.

Overall survival (OS), a key secondary endpoint, was not yet mature at this analysis (HR=0.89 [95% CI, 0.57-1.40]), and final testing of OS superiority is planned at a future date. An additional secondary endpoint, time to next treatment (TTNT), consistently favored the pirtobrutinib combination regimen (HR=0.50 [95% CI, 0.35-0.70]; nominal p<0.0001).

The overall safety profile of this regimen in BRUIN CLL-322 was consistent with the known safety profile of each medicine, with little additive toxicity observed with the addition of pirtobrutinib to venetoclax and rituximab. Rates of Grade ≥3 adverse events (AEs) were similar with PVR compared to VR (78.8% versus 73.0%, respectively). Low rates of any grade atrial fibrillation/flutter (3.5% versus 2.6%, respectively), hypertension (12.0% versus 7.4%, respectively), and hemorrhage (14.2% versus 10.6%, respectively) were seen with PVR versus VR. Grade ≥3 clinical AEs of interest included neutropenia (50.3% versus 43.7%, respectively) and tumor lysis syndrome (0.9% versus 3.9%, respectively) in the PVR and VR arms. Discontinuation rates due to treatment-related AEs were similar across the PVR and VR study arms (5.4% versus 5.1%, respectively). The addition of pirtobrutinib to VR also allowed for downgrading of tumor lysis risk, with 78% of high-risk patients downgraded to medium (n=20) or low risk (n=18), and 61% of medium-risk patients downgraded to low risk.

“These remarkable findings support the potential addition of two years of Jaypirca to a time-limited venetoclax-based regimen in relapsed or refractory CLL,” said Jacob Van Naarden, executive vice president and president of Lilly Oncology. “BRUIN CLL-322 enrolled a mostly covalent BTK inhibitor-pretreated population, ensuring that these results have applicability to the modern CLL treatment landscape where covalent BTK inhibitor use is now common. Additionally, these data further strengthen the unique body of evidence for Jaypirca across the CLL continuum, from monotherapy to combination therapy and across multiple settings where CLL patients need effective treatment.”

Lilly plans to submit results from the BRUIN CLL-322 study to global regulatory authorities with the goal of further expanding Jaypirca’s label.

Lilly is studying Jaypirca in CLL/SLL in multiple Phase 3 studies. Details on the trials can be found by visiting clinicaltrials.gov.

About BRUIN CLL-322

BRUIN CLL-322 is a global, randomized, open-label, Phase 3 study comparing time-limited pirtobrutinib plus venetoclax and rituximab versus venetoclax and rituximab in previously treated CLL/SLL patients. The trial enrolled 639 patients, who were randomized 1:1 to receive pirtobrutinib (200 mg, once daily) plus venetoclax and rituximab per their labeled doses or venetoclax and rituximab alone. The primary endpoint is PFS as assessed by blinded IRC. Secondary endpoints include PFS as assessed by investigator, OS, TTNT, event-free survival, overall response rate, time to worsening of CLL/SLL-related symptoms, time to worsening of physical functioning, safety and tolerability.

About Jaypirca (pirtobrutinib) 
Jaypirca (pirtobrutinib) (pronounced jay-pihr-kaa) is a highly selective (300 times more selective for BTK versus 98% of other kinases tested in preclinical studies), non-covalent inhibitor of the enzyme BTK.1 BTK is a validated molecular target found across numerous B-cell leukemias and lymphomas including mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL).2,3 Jaypirca is a U.S. FDA-approved oral prescription medicine, 100 mg or 50 mg tablets taken as a once-daily 200 mg dose with or without food until disease progression or unacceptable toxicity.

INDICATIONS FOR JAYPIRCA (pirtobrutinib)
Jaypirca is indicated for the treatment of

  • Adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have previously been treated with a covalent BTK inhibitor.
  • Adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

IMP
ORTANT SAFETY INFORMATION FOR JAYPIRCA (pirtobrutinib)

Infections: Fatal and serious infections (including bacterial, viral, fungal) and opportunistic infections occurred in Jaypirca-treated patients. Across clinical trials, Grade ≥3 infections occurred (25%), most commonly pneumonia (20%); fatal infections (5%), sepsis (6%), and febrile neutropenia (3.8%) occurred. In patients with CLL/SLL, Grade ≥3 infections occurred (32%), with fatal infections occurring in 8%. Opportunistic infections included Pneumocystis jirovecii pneumonia and fungal infection. Consider prophylaxis, including vaccinations and antimicrobial prophylaxis, in patients at increased risk for infection, including opportunistic infections. Monitor for signs and symptoms, evaluate, and treat. Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca.

Hemorrhage: Fatal and serious hemorrhage has occurred with Jaypirca. Across clinical trials, major hemorrhage (Grade ≥3 bleeding or any central nervous system bleeding) occurred (2.6%), including gastrointestinal hemorrhage; fatal hemorrhage occurred (0.3%). Bleeding of any grade, excluding bruising and petechiae, occurred (16%). Major hemorrhage occurred when taking Jaypirca with (2.0%) and without (0.6%) antithrombotic agents. Consider risks/benefits of co-administering antithrombotic agents with Jaypirca. Monitor for signs of bleeding. Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca. Consider withholding Jaypirca 3-7 days pre- and post-surgery based on surgery type and bleeding risk.

Cytopenias: Jaypirca can cause cytopenias, including neutropenia, thrombocytopenia, and anemia. Across clinical trials, Grade 3 or 4 cytopenias, including decreased neutrophils (27%), decreased platelets (13%), and decreased hemoglobin (11%), developed. Grade 4 decreased neutrophils (15%) and Grade 4 decreased platelets (6%) developed. Monitor complete blood counts regularly. Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca.

Cardiac Arrhythmias: Cardiac arrhythmias occurred in patients taking Jaypirca. Across clinical trials, atrial fibrillation or flutter were reported in 3.4% of Jaypirca treated patients, with Grade 3 or 4 atrial fibrillation or flutter in 1.6%. Other serious cardiac arrhythmias such as supraventricular tachycardia and cardiac arrest occurred (0.4%). Cardiac risk factors such as hypertension or previous arrhythmias may increase risk. Monitor and manage signs and symptoms of arrhythmias (e.g., palpitations, dizziness, syncope, dyspnea). Based on severity, reduce dose, temporarily withhold, or permanently discontinue Jaypirca.

Second Primary Malignancies: Across clinical trials, second primary malignancies, including non-skin carcinomas, developed in 9% of Jaypirca-treated patients, most frequently non-melanoma skin cancer (4.4%). Other second primary malignancies included solid tumors (including genitourinary and breast cancers) and melanoma. Advise patients to use sun protection and monitor for development of second primary malignancies.

Hepatotoxicity, Including Drug-Induced Liver Injury (DILI): Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of DILI, has occurred in patients treated with BTK inhibitors, including Jaypirca. Evaluate bilirubin and transaminases at baseline and throughout Jaypirca treatment. For patients who develop abnormal liver tests after Jaypirca, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold Jaypirca. If DILI is confirmed, discontinue Jaypirca.

Embryo-Fetal Toxicity: Jaypirca can cause fetal harm. Administration of pirtobrutinib to pregnant rats caused embryo-fetal toxicity, including embryo-fetal mortality and malformations at maternal exposures (AUC) approximately 3-times the recommended 200 mg/day dose. Advise pregnant women of fetal risk and females of reproductive potential to use effective contraception during treatment and for one week after last dose.

Adverse Reactions (ARs) in Patients Who Received Jaypirca

The most common (≥30%) ARs in the pooled safety population of patients with hematologic malignancies (n=704) were decreased neutrophil count (54%), decreased hemoglobin (43%), decreased leukocytes (32%), fatigue (31%), decreased platelets (31%), decreased lymphocyte count (31%), calcium decreased (30%).

Mantle Cell Lymphoma

Serious ARs occurred in 38% of patients, with pneumonia (14%), COVID-19 (4.7%), musculoskeletal pain (3.9%), hemorrhage (2.3%), pleural effusion (2.3%), and sepsis (2.3%) occurring in ≥2% of patients. Fatal ARs within 28 days of last dose occurred in 7% of patients, most commonly due to infections (4.7%), including COVID-19 (3.1% of all patients).

Dose Modifications and Discontinuations Due to ARs: Dose reductions in 4.7%, treatment interruption in 32%, and permanent discontinuation of Jaypirca in 9% of patients. Permanent discontinuation in >1% of patients included pneumonia.

Most common ARs (≥15%) and Select Laboratory Abnormalities (≥10%) (all Grades %; Grade 3-4 %): hemoglobin decreased (42; 9), platelet count decreased (39; 14), neutrophil count decreased (36; 16), lymphocyte count decreased (32; 15), creatinine increased (30; 1.6), fatigue (29; 1.6), musculoskeletal pain (27; 3.9), calcium decreased (19; 1.6), diarrhea (19; -), edema (18; 0.8), dyspnea (17; 2.3), AST increased (17; 1.6), pneumonia (16; 14), bruising (16; -), potassium decreased (13; 1.6), sodium decreased (13; -), lipase increased (12; 4.4), ALT increased (11; 1.6), potassium increased (11; 0.8), alkaline phosphatase increased (11; -). Grade 4 laboratory abnormalities in >5% of patients included neutrophils decreased (10), platelets decreased (7), lymphocytes decreased (6).

Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma from Single-Arm and Randomized Controlled Clinical Trials

Serious ARs occurred in 47-56% of patients across clinical trials. Serious ARs in ≥5% of patients in the single-arm trial were pneumonia (18%), COVID-19 (9%), sepsis (7%), febrile neutropenia (7%). Serious ARs in ≥3% of patients in the randomized controlled trial were pneumonia (21%), COVID-19 (5%), sepsis (3.4%). Fatal ARs within 28-30 days of last Jaypirca dose occurred in 8-11% of patients, most commonly due to infections (7-10%), including sepsis (5%), COVID-19 (2.7-5%), and pneumonia (3.4%).

Dose Modifications and Discontinuations Due to ARs: Dose reductions in 3.6-10%, treatment interruption in 42-51%, and permanent discontinuation of Jaypirca in 9-17% of patients. Permanent discontinuation in >1% of patients included second primary malignancy, pneumonia, COVID-19, neutropenia, sepsis, anemia, and cardiac arrhythmias.

Most common ARs and Select Laboratory Abnormalities (≥20%) (all Grades %, Grade 3-4 %)–in a randomized controlled trial: neutrophil count decreased (54; 26), hemoglobin decreased (45; 10), platelet count decreased (37; 17), pneumonia (28; 16), ALT increased (25; 1.8), creatinine increased (25; -), calcium decreased (23; 0.9), sodium decreased (22; 0.9), bilirubin increased (21; 0.9), upper respiratory tract infections (21; 0.9); in a single-arm trial: neutrophil count decreased (63; 45), hemoglobin decreased (48; 19), calcium decreased (40; 2.8), fatigue (36; 2.7), bruising (36; -), cough (33; -), musculoskeletal pain (32; 0.9), platelet count decreased (30; 15), sodium decreased (30; -), COVID-19 (28; 7), pneumonia (27; 16), diarrhea (26; -), abdominal pain (25; 2.7), lymphocyte count decreased (23; 8), ALT increased (23; 2.8), AST increased (23; 1.9), creatinine increased (23; -), dyspnea (22; 2.7), hemorrhage (22; 2.7), lipase increased (21; 7), alkaline phosphatase increased (21; -), edema (21; -), nausea (21; -), pyrexia (20; 2.7), headache (20; 0.9). Grade 4 laboratory abnormalities in >5% of patients included neutrophils decreased (23).

Drug Interactions

Strong CYP3A Inhibitors: Concomitant use increased pirtobrutinib systemic exposure, which may increase risk of Jaypirca ARs. Avoid using strong CYP3A inhibitors with Jaypirca. If concomitant use is unavoidable, reduce Jaypirca dose according to approved labeling.

Strong or Moderate CYP3A Inducers: Concomitant use decreased pirtobrutinib systemic exposure, which may reduce Jaypirca efficacy. Avoid using Jaypirca with strong or moderate CYP3A inducers. If concomitant use with moderate CYP3A inducers is unavoidable, increase Jaypirca dose according to approved labeling.

Sensitive CYP2C8, CYP2C19, CYP3A, P-gp, or BCRP Substrates: Use with Jaypirca increased their plasma concentrations, which may increase risk of ARs related to these substrates for drugs sensitive to minimal concentration changes. Follow recommendations for these sensitive substrates in their approved labeling.

Use in Specific Populations

Pregnancy and Lactation: Due to potential for Jaypirca to cause fetal harm, verify pregnancy status in females of reproductive potential prior to starting Jaypirca. Presence of pirtobrutinib in human milk is unknown. Advise women to use effective contraception and to not breastfeed while taking Jaypirca and for one week after last dose.

Geriatric Use: In the pooled safety population of patients with hematologic malignancies, patients aged ≥65 years experienced higher rates of Grade ≥3 ARs and serious ARs compared to patients <65 years of age.

Renal Impairment: Because severe renal impairment increases pirtobrutinib exposure, reduce Jaypirca dose in these patients according to approved labeling.

PT HCP ISI MCL_CLL Q42025

Please see Prescribing Information and Patient Information for Jaypirca.

About Lilly                                        
Lilly is a medicine company turning science into healing to make life better for people around the world. We’ve been pioneering life-changing discoveries for 150 years, and today our medicines help tens of millions of people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world’s most significant health challenges: redefining diabetes care; treating obesity and curtailing its most devastating long-term effects; advancing the fight against Alzheimer’s disease; providing solutions to some of the most debilitating immune system disorders; and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we’re motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visit Lilly.com and Lilly.com/news, or follow us on Facebook, Instagram, and LinkedIn. P-LLY

© Lilly USA, LLC 2026. ALL RIGHTS RESERVED.

Trademarks and Trade Names
All trademarks or trade names referred to in this press release are the property of the company, or, to the extent trademarks or trade names belonging to other companies are referenced in this press release, the property of their respective owners. Solely for convenience, the trademarks and trade names in this press release are referred to without the ® and ™ symbols, but such references should not be construed as any indicator that the company or, to the extent applicable, their respective owners will not assert, to the fullest extent under applicable law, the company’s or their rights thereto. We do not intend the use or display of other companies’ trademarks and trade names to imply a relationship with, or endorsement or sponsorship of us by, any other companies.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about Jaypirca (pirtobrutinib), as a potential treatment for adults with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and the timeline for future regulatory submissions, presentations, and other milestones relating to Jaypirca and its clinical trials, and reflects Lilly’s current beliefs and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of drug research, development, and commercialization. Among other things, there is no guarantee that planned or ongoing studies will be completed as planned, that future study results will be consistent with study results to date, or that Jaypirca will receive additional regulatory approvals. For further discussion of these and other risks and uncertainties that could cause actual results to differ from Lilly’s expectations, see Lilly’s Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.

Endnotes & References

  1. Mato AR, Shah NN, Jurczak W, et al. Pirtobrutinib in relapsed or refractory B-cell malignancies (BRUIN): a phase 1/2 study. Lancet. 2021;397(10277):892-901. doi:10.1016/S0140-6736(21)00224-5
  2. Hanel W, Epperla N. Emerging therapies in mantle cell lymphoma. J Hematol Oncol. 2020;13(1):79. Published 2020 Jun 17. doi:10.1186/s13045-020-00914-1
  3. Gu D, Tang H, Wu J, Li J, Miao Y. Targeting Bruton tyrosine kinase using non-covalent inhibitors in B cell malignancies. J Hematol Oncol. 2021;14(1):40. Published 2021 Mar 6. doi:10.1186/s13045-021-01049-7


Refer to:

Kyle Owens; [email protected] (Media)

Michael Czapar; [email protected] (Investors)

Eli Lilly and Company logo. (PRNewsFoto, Eli Lilly and Company)

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/lillys-jaypirca-pirtobrutinib-significantly-reduced-the-risk-of-disease-progression-or-death-by-45-when-added-to-a-venetoclax-time-limited-regimen-in-people-with-previously-treated-cllsll-302799348.html

SOURCE Eli Lilly and Company

Posted on

Legend Biotech Establishes Clinical Proof-of-Concept for LB2501, a Potential First-in-Class In Vivo CD19/CD20 Dual-Targeting CAR-T, in Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma

  •  Achieved 100% ORR and 83.3% CR rate at dose level 2 following a single infusion in patients with relapsed/refractory B-NHL in an ongoing Phase 1 study
  • Single infusion of LB2501 generated dose-dependent in vivo CAR-T
    expansion without lymphodepletion
  • No dose-limiting toxicities, serious adverse events, ICANS, or deaths were reported; infusion-related reactions and CRS were Grade 1–2, and none required glucocorticoids for CRS management
  • Additional translational data showed rapid vector clearance, polyclonal vector integration, and no evidence of non-specific transduction
  • Proof-of-concept progress demonstrates leadership in next-generation cell therapies, with results presented in a late-breaking session at EHA 2026

BRIDGEWATER, N.J., June 14, 2026 (GLOBE NEWSWIRE) — Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, today announced first clinical proof-of-concept data for LB2501, its investigational in vivo CD19/CD20 dual-targeting CAR-T cell therapy, in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (R/R B-NHL). The results are being presented today in a late-breaking session at the European Hematology Association (EHA) 2026 Congress (Abstract #LB5006).

In the ongoing Phase 1 study, a single infusion of LB2501 generated dose-dependent in vivo CAR-T expansion without lymphodepletion. At the higher dose level (DL2), LB2501 achieved a 100% objective response rate (ORR) (6/6) and an 83.3% complete response rate (CR) (5/6), with all responses ongoing at the time of data cutoff. LB2501 also showed a favorable safety profile, with no dose-limiting toxicities (DLTs), serious adverse events (SAEs), immune effector cell-associated neurotoxicity syndrome (ICANS), or deaths reported.

“In vivo CAR-T represents a compelling frontier in cell therapy, enabling the generation of CAR-T cells directly within the patient, with the potential to simplify treatment and expand access over time,” said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. “LB2501 is our step toward realizing that vision and reflects further progress toward our goal of leading the future of cell therapy. Backed by the commercial and scientific foundation we have built with CARVYKTI, we are well-positioned to advance this next generation of CAR-T delivery. These early data, with deep responses from a single infusion across patients, give us confidence in the path ahead.”

LB2501 Demonstrates

In Vivo

CAR-T Generation and Early Clinical Activity

In an ongoing Phase 1 study, 12 patients with R/R B-NHL received LB2501 across two dose levels, DL1 (n=6) and DL2 (n=6). Patients had received a median of three prior lines of therapy, and 58.3% were refractory to their most recent treatment. The open-label, multi-center, dose-escalation study is evaluating safety, recommended Phase 2 dose, pharmacokinetics, and preliminary efficacy in adults with R/R B-NHL. The study was conducted without lymphodepletion.

At DL2, LB2501 achieved a 100% ORR (6/6) and an 83.3% CR rate (5/6), with responses observed across patients with diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and follicular lymphoma (FL). Across both dose levels, the ORR was 50.0% (6/12), and the CR rate was 41.7% (5/12). At the time of data cutoff, all responses at DL2 were ongoing.

LB2501 showed a favorable safety profile. No DLTs, SAEs, ICANS, or deaths were reported. Infusion-related reactions (IRR) and cytokine release syndrome (CRS) were the most common adverse events of special interest and were all Grade 1–2. Infusion-related reactions occurred in 75.0% (9/12) of patients overall, with a median onset of 1.4 hours after infusion and a median recovery time of 18.6 hours. CRS occurred in 66.7% (8/12) of patients overall, with a median onset at Day 11 and a median duration of 4.5 days. IRR and CRS were all Grade 1–2, no patients required glucocorticoids for CRS management. Four patients received tocilizumab.

Pharmacokinetic analyses showed dose-dependent in vivo CAR-T expansion in 100% (6/6) of patients at DL2 and 83% (5/6) of patients at DL1. CAR-T cells remained detectable in peripheral blood for up to 116 days. Viral copy number in peripheral blood peaked immediately after infusion and decreased to undetectable concentrations within 24 hours.

Additional translational analyses further characterized the in vivo profile of LB2501. No evidence of non-specific transduction was detected in NK cells or other non-T/B/NK lymphocyte populations. Vector integrations were highly polyclonal and diverse. These findings support proof-of-concept for in vivo T-cell engineering, with polyclonal vector integration and rapid vector clearance.

“These early clinical findings are encouraging in a heavily pretreated relapsed or refractory B-cell non-Hodgkin lymphoma population,” said Lei Fan, M.D., Ph.D., Professor, Doctoral Supervisor, and Administrative Director, Hematology Department, Jiangsu Province Hospital, Nanjing, China. “The responses observed at the higher dose level achieved a 100% objective response rate, together with a favorable safety profile and the absence of lymphodepletion, support further investigation of LB2501 as a novel in vivo CAR-T approach. The additional pharmacokinetic and translational findings presented at EHA further support the feasibility of generating CAR-T cells directly within the patient.” ‡

ABOUT LB2501

LB2501 is an investigational, potential first-in-class CD19/CD20 dual-targeting in vivo CAR-T therapy designed to generate CAR-T cells directly within the patient following a single intravenous infusion. It is being evaluated in an ongoing Phase 1, open-label study (NCT07002112) in patients with relapsed/refractory B-cell malignanciesi to assess safety, tolerability, and preliminary efficacy.[i]

ABOUT B-CELL NON-HODGKIN LYMPHOMA

Non-Hodgkin lymphoma (NHL) is a group of cancers that originate in lymphocytes, a type of white blood cell that plays a key role in the body’s immune system.ii B-cell lymphomas account for approximately 85% of NHL cases and arise from abnormal growth of B lymphocytes (B cells), which are responsible for producing antibodies. These malignancies include a range of subtypes that vary in aggressiveness, from slow-growing to highly aggressive disease.iii

While treatment advances have improved outcomes for some patients, those with relapsed or refractory B-cell NHL, particularly after multiple lines of therapy, often face limited options.

ABOUT LEGEND BIOTECH

With over 3,000 employees, Legend Biotech is the largest standalone cell therapy company and a pioneer in treatments that change cancer care forever. Legend Biotech is at the forefront of the CAR-T cell therapy revolution with CARVYKTI®, a one-time treatment for relapsed or refractory multiple myeloma, which it develops and markets with collaborator Johnson & Johnson. Centered in the United States, Legend Biotech is building an end-to-end cell therapy company by expanding its leadership to maximize CARVYKTI’s patient access and therapeutic potential. From this platform, Legend Biotech plans to drive future innovation across its pipeline of cutting-edge cell therapy modalities.

Learn more at https://legendbiotech.com and follow us on X, Instagram, and LinkedIn.

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to Legend Biotech’s strategies and objectives, the Phase 1 clinical trial of LB2501, and the potential benefits of LB2501, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, and LB2501’s potential to be first-in-class. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Legend Biotech’s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products; unexpected clinical trial results, including as a result of additional analysis of existing clinical data or unexpected new clinical data; unexpected regulatory actions or delays, including requests for additional safety and/or efficacy data or analysis of data, or government regulation generally; unexpected delays as a result of actions undertaken, or failures to act, by Legend Biotech’s third-party partners; uncertainties arising from challenges to Legend Biotech’s patent or other proprietary intellectual property protection, including the uncertainties involved in the U.S. litigation process; government, industry, and general product pricing and other political pressures; as well as the other factors discussed in the “Risk Factors” section of Legend Biotech’s Annual Report on Form 20-F filed with the Securities and Exchange Commission on March 10, 2026. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in this press release as anticipated, believed, estimated, or expected. Any forward-looking statements contained in this press release speak only as of the date of this press release. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.

‡ Lei Fan, M.D., Ph.D., Professor, Doctoral Supervisor, and Administrative Director, Hematology Department, Jiangsu Province Hospital, Nanjing, China, has provided consulting and advisory services to Legend Biotech; he has not been paid for any media work.

INVESTOR CONTACT:

Jessie Yeung
Tel: (732) 956-8271
[email protected]

PRESS CONTACT:

Kim Fox
Tel: (848) 388-8445
[email protected]

i ClinicalTrials.Gov. The CD19/ CD20 Dual-Target in Vivo CAR-T Lentiviral Product in the Treatment of Relapsed/ Refractory B-cell Malignancies. https://clinicaltrials.gov/study/NCT07002112. Accessed May 2026
ii American Cancer Society. “What Is Non-Hodgkin Lymphoma?”. Available at: https://www.cancer.org/cancer/types/non-hodgkin-lymphoma/about/what-is-non-hodgkin-lymphoma.html.Accessed May 2026.
iii American Cancer Society. “Types of B-cell Lymphoma.” Available at: https://www.cancer.org/cancer/types/non-hodgkin-lymphoma/about/b-cell-lymphoma.html.Accessed May 2026.



Posted on

Babcock & Wilcox Deadline: BW Investors Have Opportunity to Lead Babcock & Wilcox Enterprises, Inc. Securities Fraud Lawsuit

PR Newswire

NEW YORK, June 13, 2026 /PRNewswire/ —

Rosen Law Firm Logo

Why: Rosen Law Firm, a global investor rights law firm, reminds purchasers of securities of Babcock & Wilcox Enterprises, Inc. (“B&W”) (NYSE: BW) between November 5, 2025 and March 11, 2026, inclusive (the “Class Period”), of the important June 15, 2026 lead plaintiff deadline.

So what: If you purchased B&W securities during the Class Period you may be entitled to compensation without payment of any out of pocket fees or costs through a contingency fee arrangement.

What to do next: To join the B&W class action, go to https://rosenlegal.com/submit-form/?case_id=60146 or call Phillip Kim, Esq. toll-free at 866-767-3653 or email [email protected] for information on the class action. A class action lawsuit has already been filed. If you wish to serve as lead plaintiff, you must move the Court no later than June 15, 2026. A lead plaintiff is a representative party acting on behalf of other class members in directing the litigation.

Why Rosen Law: We encourage investors to select qualified counsel with a track record of success in leadership roles. Often, firms issuing notices do not have comparable experience, resources, or any meaningful peer recognition. Many of these firms do not actually handle securities class actions, but are merely middlemen that refer clients or partner with law firms that actually litigate the cases. Be wise in selecting counsel. The Rosen Law Firm represents investors throughout the globe, concentrating its practice in securities class actions and shareholder derivative litigation. Rosen Law Firm has achieved, at that time, the largest ever securities class action settlement against a Chinese Company. Rosen Law Firm was Ranked No. 1 by ISS Securities Class Action Services for number of securities class action settlements in 2017. The firm has been ranked in the top 4 each year since 2013 and has recovered hundreds of millions of dollars for investors. In 2019 alone the firm secured over $438 million for investors. In 2020, founding partner Laurence Rosen was named by law360 as a Titan of Plaintiffs’ Bar. Many of the firm’s attorneys have been recognized by Lawdragon and Super Lawyers.

Details of the case: According to the lawsuit, throughout the Class Period, defendants made false and/or misleading statements and/or failed to disclose that: (1) B&W’s largest shareholder, BRC Group Holdings, Inc. (“BRC”), stood on both sides of the Power Generation Contract and had close ties to B&W’s counterparty; (2) Applied Digital did not need the products and services that B&W would purportedly supply pursuant to the Power Generation LNTP and Contract; (3) the foregoing, at the very least, would raise questions about the parties’ actual intent behind entering into the Power Generation LNTP and Contract, including whether B&W is likely to recognize revenues from these agreements; (4) accordingly, the business and financial prospects of B&W were overstated; and (5) as a result, defendants’ public statements were materially false and misleading at all relevant times. When the true details entered the market, the lawsuit claims that investors suffered damages. 

To join the B&W class action, go to https://rosenlegal.com/submit-form/?case_id=60146 or call Phillip Kim, Esq. toll-free at 866-767-3653 or email [email protected] for information on the class action.

No Class Has Been Certified. Until a class is certified, you are not represented by counsel unless you retain one. You may select counsel of your choice. You may also remain an absent class member and do nothing at this point. An investor’s ability to share in any potential future recovery is not dependent upon serving as lead plaintiff.

Follow us for updates on LinkedIn: https://www.linkedin.com/company/the-rosen-law-firm, on Twitter: https://twitter.com/rosen_firm or on Facebook: https://www.facebook.com/rosenlawfirm/.

Attorney Advertising. Prior results do not guarantee a similar outcome.

Contact Information:

     Laurence Rosen, Esq.
     Phillip Kim, Esq.
     The Rosen Law Firm, P.A.
     275 Madison Avenue, 40th Floor
     New York, NY 10016
     Tel: (212) 686-1060
     Toll Free: (866) 767-3653
     Fax: (212) 202-3827
     [email protected]
     www.rosenlegal.com

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/babcock–wilcox-deadline-bw-investors-have-opportunity-to-lead-babcock–wilcox-enterprises-inc-securities-fraud-lawsuit-302799353.html

SOURCE THE ROSEN LAW FIRM, P. A.

Posted on

Phase 3 Data Show TransCon® PTH Replicated Systemic Actions of Endogenous PTH Through Week 182 in Adults with Hypoparathyroidism

  • Multi-organ system and quality-of-life benefits sustained through three and a half years 
  • 86% response rate for the multi-component endpoint
  • 89% of patients completed the three-and-a-half-year trial

COPENHAGEN, Denmark, June 13, 2026 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND) today announced Week 182 data from its completed Phase 3 PaTHway Trial showing that long-term treatment with TransCon PTH (palopegteriparatide) demonstrated sustained efficacy and safety in adults with hypoparathyroidism. Over the three-and-a-half-year duration of the trial, TransCon PTH replicated the systemic actions of endogenous PTH, with a balanced, beneficial impact on the main target organ systems – CNS, kidney, small intestine, and bone – as demonstrated by improved quality of life and normalized and stable urine calcium, serum calcium, serum phosphate, and bone mineral density. These benefits were sustained while enabling independence from conventional therapy with active vitamin D and calcium.

“With its unique ability to replicate the systemic actions of endogenous parathyroid hormone, TransCon PTH has successfully addressed the physical and psychological burdens of hypoparathyroidism for the majority of treated patients,” said Aliya Khan, M.D., Clinical Professor of Medicine, Division of Endocrinology & Geriatrics, and Director of the Calcium Disorders Clinic at McMaster University in Canada. “These Phase 3 data reinforce the rapid and sustained benefits seen throughout clinical trials of TransCon PTH, including compelling improvements in quality of life reported by patients previously limited by the fatigue, cognitive challenges, and reduced physical functioning and well-being that are the hallmarks of this disease.”

Highlights of Week 182 Results from the Phase 3 PaTHway Trial

  • 86% of patients were responders for the multi-component endpoint of (1) serum calcium in the normal range, (2) taking no active vitamin D, and (3) taking ≤600 mg/day of calcium.
    • 89% of patients had normal albumin-adjusted serum calcium levels and a mean value of 8.8 mg/dL.
    • 100% of patients achieved independence from active vitamin D, defined as not taking calcitriol or alfacalcidol.
    • 96% of patients achieved independence from therapeutic doses of calcium, defined as taking <600 mg/day of calcium.
  • Significant improvements in kidney function were maintained, with mean (SE) eGFR of 80.2 (1.8) mL/min/1.73 m2 at Week 182, reflecting a mean (SE) increase of 11.0 (1.4) mL/min/1.73 m2 from baseline. Among patients randomized to TransCon PTH, eGFR increased from baseline through Week 38 and stabilized thereafter. After initiation of open-label treatment at Week 26, patients who had been receiving placebo in the double-blind period experienced a similar increase in eGFR. Following these eGFR increases, mean eGFR values were maintained through Week 182, in contrast to the expected typical age-related decline in eGFR in adults.i
  • Mean 24-hour urine calcium decreased substantially, normalized within 26 weeks, and remained normal through Week 182.
  • As measured by Hypoparathyroidism Patient Experience Scales (HPES), patients reported improvements in symptoms and health-related quality of life across all domains. Hypoparathyroidism-related physical and cognitive symptoms and impacts on physical functioning and daily life improved rapidly with TransCon PTH treatment and were maintained through Week 182.
  • As measured by SF-36, all subscale scores and component summary scores demonstrated rapid and clinically meaningful improvements with TransCon PTH treatment which were sustained through Week 182.
  • Mean BMD Z-scores (matched for age and sex) corrected from high baseline levels through Week 26 and remained above 0 through Week 182.
  • In the trial, TransCon PTH treatment was generally well-tolerated, with no new safety signals identified. Treatment-emergent adverse events (AEs) were mostly mild or moderate, and no discontinuations were related to study drug.
  • Over three and a half years of treatment, no patients developed anti-PTH antibodies.

“Regardless of disease origin, TransCon PTH has normalized key biochemistries and skeletal health while significantly improving kidney function and quality of life beginning at the earliest timepoints and continuing through multiple years of treatment,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “We remain committed to continuing our work to advance treatment options for patients around the world living with this often-debilitating chronic disease.”

The PaTHway Trial of 82 adults with hypoparathyroidism (85% post-surgical, 15% non-surgical) included a 26-week randomized, double-blind, placebo-controlled period followed by a 156-week open-label extension (OLE) period, and measured a wide array of clinical, biochemical, and quality of life endpoints, consistent with the breadth of negative long-term impacts experienced by patients with hypoparathyroidism. Seventy-three of the original 82 patients enrolled (89%) completed the three-and-a-half-year trial. Endpoints included independence from conventional therapy (defined as <600 mg/day of calcium and no active vitamin D) and maintenance of normocalcemia (8.3 to 10.6 mg/dL). Renal function was assessed by estimated glomerular filtration rate (eGFR). Bone mineral density (BMD) measured by DXA scan was assessed at baseline and regular intervals through Week 182. Hypoparathyroidism-related symptoms and functional impacts were measured using the HPES. Health-related quality of life was measured using the 36-Item Short Form Survey (SF-36 version 2). Safety assessments included treatment-emergent AEs and 24-hour urine calcium excretion.

TransCon PTH is a prodrug of PTH (1-34), administered once daily, designed to provide stable levels of active PTH within the physiological range for 24 hours/day, approved as YORVIPATH® in the United States, European Union, European Economic Area, and certain other jurisdictions as a treatment for adults with hypoparathyroidism.

A slide presentation with these data will be made available on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com.

About Hypoparathyroidism

Hypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune, idiopathic, and genetic causes, including ADH1.

About Ascendis Pharma A/S

Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) TransCon PTH’s ability to replicate endogenous parathyroid hormone and address the physical and psychological burdens of hypoparathyroidism, (ii) Ascendis’ commitment to continuing its work to advance treatment options for patients around the world with hypoparathyroidism, (iii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (iv) Ascendis’ use of TransCon to create new and potentially best-in-class therapies to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 11, 2026, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.

Ascendis, Ascendis Pharma, the Ascendis Pharma logo, TransCon, and YORVIPATH

®

are trademarks owned by the Ascendis Pharma group. © June 2026 Ascendis Pharma A/S.


Investor Contact:
 
Media Contact:
Chad Fugere Melinda Baker
Ascendis Pharma Ascendis Pharma
+1 (650) 519-7494 +1 (650) 709-8875

iGuppy M et al. BMJ Open. 2024;14(11):e089783. doi:10.1136/bmjopen-2024-089783



Posted on

Genmab Announces Epcoritamab Monotherapy and Epcoritamab-Based Combination Regimens Demonstrate High Response Rates in Elderly Patients with Newly Diagnosed Diffuse Large B-Cell Lymphoma (DLBCL)

Genmab Announces Epcoritamab Monotherapy and Epcoritamab-Based Combination Regimens Demonstrate High Response Rates in Elderly Patients with Newly Diagnosed Diffuse Large B-Cell Lymphoma (DLBCL)

  • Results from the Phase 2 EPCORE® DLBCL-3 trial show fixed-duration epcoritamab monotherapy demonstrated early responses in elderly patients with newly diagnosed diffuse large B-cell lymphoma (DLBCL) ineligible for anthracycline-based chemotherapy
  • Results from the Phase 1b/2 EPCORE® NHL-2 trial show fixed-duration epcoritamab plus standard of care R-mini-CHOP demonstrated sustained minimal residual disease (MRD) negativity and durable remissions in elderly patients with newly diagnosed DLBCL ineligible for full dose R-CHOP
  • Data were presented at the 2026 European Hematology Association (EHA) Congress

COPENHAGEN, Denmark–(BUSINESS WIRE)–Genmab A/S (Nasdaq: GMAB) today announced new data from two studies evaluating epcoritamab, a T-cell engaging antibody administered subcutaneously, in the first-line (1L) treatment of patients with diffuse large B-cell lymphoma (DLBCL) who may have limited treatment options due to advanced age or multiple health conditions. Results from the Phase 2 EPCORE® DLBCL-3 study showed an overall response rate (ORR) of 67% and a complete response (CR) rate of 58% with epcoritamab monotherapy in elderly patients with newly diagnosed DLBCL. In the Phase 1b/2 EPCORE NHL-2 study, epcoritamab plus rituximab plus dose-attenuated cyclophosphamide, doxorubicin, vincristine, and prednisone (R-mini-CHOP) demonstrated an ORR of 93% and a CR rate of 86% in elderly patients with newly diagnosed DLBCL.

The results from both studies were presented in two poster presentations (abstracts PS2082 and PF1007) at the European Hematology Association (EHA) 2026 Congress held in Stockholm, Sweden, June 11-14. Additionally, the full EPCORE DLBCL-3 results have been simultaneously published in The Lancet Haematology.

EPCORE DLBCL-3 Results

The Phase 2 EPCORE DLBCL-3 study (abstract PS2082) evaluated the efficacy and safety of fixed-duration epcoritamab monotherapy in newly diagnosed CD20+ large B-cell lymphoma (LBCL) patients ineligible for anthracycline-based chemotherapy due to age (≥80 years) or comorbidities (≥75 years with comorbidities). Among 66 enrolled patients, the median age was 82.5 years, and all had comorbid conditions (94% with ≥3 comorbidities). With a median follow-up of 21.9 months, epcoritamab monotherapy demonstrated responses in this population with high unmet medical need.

An ORR of 67% and a CR rate of 58% were observed in evaluable patients (n=66). Median time to response was 1.5 months, and median time to CR was 2.2 months. Notably, 11 of 17 patients with a partial response or stable disease at first assessment subsequently achieved a CR.

“For newly diagnosed elderly patients with diffuse large B-cell lymphoma and comorbidities, who are often excluded from standard curative chemotherapy and ineligible for doxorubicin, finding more options is paramount,” said Umberto Vitolo, M.D. Candiolo Cancer Institute, FPO-IRCCS, Candiolo (Turin), Italy. “The EPCORE DLBCL-3 study showed that epcoritamab monotherapy offers robust data. Importantly, its safety profile, including cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome, was consistent with expected rates in this fragile population with a high unmet medical need for new therapeutic options.”

Responses were durable, with median duration of response (DOR) and duration of complete response (DOCR) not reached. At 12 months, an estimated 67% of responses and 73% of CRs remained ongoing. Median progression-free survival (PFS) was 13.0 months, while median overall survival (OS) was not reached; an estimated 43% of patients remained progression-free and 62% were alive at 18 months. High rates of minimal residual disease (MRD) negativity were observed, with 92% of evaluable responders achieving MRD negativity, typically by Cycle 3 Day 1 and sustained through Cycle 12 Day 1 in most patients.

The safety profile was consistent with expected rates in this elderly population. Cytokine release syndrome (CRS) occurred in 71% of patients, most commonly during Cycle 1, and immune effector cell-associated neurotoxicity syndrome (ICANS) occurred in 18%. Infections of any grade occurred in 68% of patients (26% Grade ≥3), and neutropenia was reported in 16%, with no febrile neutropenia or clinical tumor lysis syndrome observed. Eight Grade 5 TEAEs occurred.

EPCORE NHL-2, Arm 8 Results

Arm 8 of the Phase 1b/2 EPCORE NHL-2 study (abstract PF1007) evaluated epcoritamab plus R-mini-CHOP in 28 newly diagnosed CD20+ DLBCL patients ineligible for full-dose R-CHOP due to age (≥75 years) or comorbidities (≥65 years with comorbidities). With more than two years of follow-up, fixed-duration epcoritamab plus R-mini-CHOP demonstrated high response rates, sustained MRD negativity and durable remissions.

An ORR of 93% and a CR rate of 86% were observed. Median DOR, DOCR, PFS, and OS were not reached. At two years, estimated DOR and DOCR rates were 79%, while estimated PFS and OS rates were 76% and 82%, respectively.

“The EPCORE NHL-2 Arm 8 results are very encouraging, showing that combining epcoritamab with R-mini-CHOP led to high overall response rates and complete response rates, rapid and sustained minimal residual disease negativity, and durable remissions in this population,” said David Belada, M.D., Department of Internal Medicine—Haematology, Charles University, Hospital and Faculty of Medicine, Hradec Králové, Czech Republic. “These outcomes, alongside a consistent safety profile, potentially support the integration of epcoritamab with standard of care for these vulnerable patients, and highlight its broad utility in combinations across a range of disease settings and patient populations.”

Rapid and sustained MRD negativity was observed, with 95% of evaluable patients achieving MRD negativity, including high rates in high-risk subgroups. Outcomes compared favorably with historical results for R-mini-CHOP alone.

The safety profile was consistent with prior reports and the known safety profiles of epcoritamab and R-mini-CHOP. The most common Grade ≥3 treatment-emergent adverse events (TEAEs) were neutropenia (54%), serious infections (33%) and anemia (14%). Most Grade ≥3 serious infections occurred during the first six cycles of treatment with R-mini-CHOP coadministration. TEAEs led to epcoritamab discontinuation in three patients (11%).

“Genmab is committed to evaluating epcoritamab as a potential treatment option in earlier lines of therapy for patients who traditionally struggle with aggressive treatment,” said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. “The robust data observed in both the monotherapy and combination approaches reinforce our vision of making epcoritamab a foundational therapy across the spectrum of B-cell malignancies. These Phase 2 results support our ongoing commitment to addressing the significant unmet medical needs of elderly and comorbid patients, as we seek to identify effective, less intensive and tolerable options.”

About Diffuse Large B-Cell Lymphoma

Diffuse large B-cell lymphoma (DLBCL) DLBCL is the most common type of non-Hodgkin lymphoma (NHL) worldwide, accounting for approximately 25-30 percent of all NHL cases.i,ii DLBCL can arise in lymph nodes as well as in organs outside of the lymphatic system, occurs more commonly in the elderly and is slightly more prevalent in men.iii,iv DLBCL is a fast-growing type of NHL, a cancer that develops in the lymphatic system and affects B-cell lymphocytes, a type of white blood cell. For many people living with DLBCL, their cancer either relapses, which means it may return after treatment, or becomes refractory, meaning it does not respond to treatment. Although new therapies have become available, treatment management can remain a challenge.iv,v

About the EPCORE® DLBCL-3 Trial

EPCORE DLBCL-3 (NCT05660967) is an open-label, randomized, global, Phase 2 trial to evaluate the efficacy and safety of epcoritamab as monotherapy or in combination with lenalidomide as first-line therapy for anthracycline-ineligible subjects with diffuse large B-cell lymphoma (DLBCL). This is a 2-stage trial. In Stage 1, eligible patients were randomized to either epcoritamab monotherapy or epcoritamab plus lenalidomide. In Stage 2, additional patients were enrolled to the epcoritamab monotherapy arm. Each treatment cycle is 28 days. Patients will receive a maximum of 12 cycles (up to 1 year) of treatment. The primary objective is to evaluate the clinical efficacy of epcoritamab monotherapy or epcoritamab and lenalidomide. The primary endpoint is to achieve a complete response rate determined by Lugano criteria. Additional secondary endpoints include overall response rate, duration of response, duration of complete response, rate of minimal residual disease negativity, progression-free survival and overall survival.

More information on this trial can be found at www.clinicaltrials.gov/.

About the EPCORE® NHL-2 Trial

EPCORE NHL-2 (NCT04663347) is a Phase 1b/2 open-label interventional trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics/biomarkers, immunogenicity, and preliminary efficacy of epcoritamab as a monotherapy and in combination with other standard of care agents in patients with B-cell non-Hodgkin lymphoma (B-NHL). The trial consists of two parts: Part 1 (Dose Escalation) and Part 2 (Dose Expansion). The primary objective of Part 1 is safety, and the primary goal of Part 2 is preliminary efficacy. The primary efficacy endpoint is overall response rate (ORR) based on best overall response per Lugano criteria. MRD negativity was assessed as a secondary endpoint.

More information on this trial can be found at www.clinicaltrials.gov.

About Epcoritamab

Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.vi

Epcoritamab (approved under the brand name EPKINLY® in the U.S. and Japan, and TEPKINLY® in the EU) has received regulatory approval in certain lymphoma indications in more than 65 territories. Where approved, epcoritamab is a readily accessible therapy. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational relapsed or refractory (R/R) follicular lymphoma (FL) indication and additional approvals for the R/R diffuse large B-cell lymphoma (DLBCL) indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes several Phase 3, open-label, randomized trials, including a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with lenalidomide compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658), and a trial evaluating epcoritamab in combination with lenalidomide and rituximab (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

What is EPKINLY?

EPKINLY is a prescription medicine used to treat adults with:

  • certain types of diffuse large B-cell lymphoma (DLBCL) or high-grade B-cell lymphoma that has come back (relapsed) or that did not respond (refractory) after 2 or more treatments.

  • follicular lymphoma (FL) that has come back or that did not respond to previous treatment, together with lenalidomide and rituximab

  • follicular lymphoma (FL) that has come back or that did not respond after 2 or more treatments.

EPKINLY for the treatment of DLBCL is approved based on patient response data. Studies are ongoing to confirm the clinical benefit of EPKINLY.

It is not known if EPKINLY is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Important Warnings—EPKINLY can cause serious side effects, including:

  • Cytokine release syndrome (CRS), which is common during treatment with EPKINLY and can be serious or lead to death. To help reduce your risk of CRS, you will receive EPKINLY on a step-up dosing schedule (when you receive 2 or 3 smaller step-up doses of EPKINLY before your first full dose during your first cycle of treatment), and you may also receive other medicines before and for 3 days after receiving EPKINLY. If your dose of EPKINLY is delayed for any reason, you may need to repeat the step-up dosing schedule.
  • Neurologic problems that can be serious, and can be life-threatening, and lead to death. Neurologic problems may happen days or weeks after you receive EPKINLY.

People with DLBCL or high-grade B-cell lymphoma may be hospitalized after receiving their first full dose of EPKINLY on Day 15 of Cycle 1 due to the risk of CRS and neurologic problems.

People with FL may be hospitalized after receiving their first full dose of EPKINLY on Day 22 of Cycle 1 due to the risk of CRS and neurologic problems.

Tell your healthcare provider or get medical help right away if you develop a fever of 100.4°F (38°C) or higher; dizziness or lightheadedness; trouble breathing; chills; fast heartbeat; feeling anxious; headache; confusion; shaking (tremors); problems with balance and movement, such as trouble walking; trouble speaking or writing; confusion and disorientation; drowsiness, tiredness or lack of energy; muscle weakness; seizures; or memory loss. These may be symptoms of CRS or neurologic problems. If you have any symptoms that impair consciousness, do not drive or use heavy machinery or do other dangerous activities until your symptoms go away.

EPKINLY can cause other serious side effects, including:

  • Infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment and treat you as needed if you develop an infection. You should receive medicines from your healthcare provider before you start treatment to help prevent infection. Tell your healthcare provider right away if you develop any symptoms of infection during treatment, including fever of 100.4°F (38°C) or higher, cough, chest pain, tiredness, shortness of breath, painful rash, sore throat, pain during urination, feeling weak or generally unwell, or confusion.
  • Low blood cell counts, which can be serious or severe. Your healthcare provider will check your blood cell counts during treatment. EPKINLY may cause low blood cell counts, including low white blood cells (neutropenia and lymphopenia), which can increase your risk for infection; low red blood cells (anemia), which can cause tiredness and shortness of breath; and low platelets (thrombocytopenia), which can cause bruising or bleeding problems.

Your healthcare provider will monitor you for symptoms of CRS, neurologic problems, infections, and low blood cell counts during treatment with EPKINLY. Your healthcare provider may temporarily stop or completely stop treatment with EPKINLY if you develop certain side effects.

Before you receive EPKINLY, tell your healthcare provider about all your medical conditions, including if you have an infection, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. If you receive EPKINLY while pregnant, it may harm your unborn baby. If you are a female who can become pregnant, your healthcare provider should do a pregnancy test before you start treatment with EPKINLY and you should use effective birth control (contraception) during treatment and for 4 months after your last dose of EPKINLY. Tell your healthcare provider if you become pregnant or think that you may be pregnant during treatment with EPKINLY. Do not breastfeed during treatment with EPKINLY and for 4 months after your last dose of EPKINLY.

The most common side effects of EPKINLY when used alone in DLBCL or high-grade B-cell lymphoma or FL include CRS, injection site reactions, tiredness, muscle and bone pain, fever, diarrhea, COVID-19, rash, and stomach-area (abdominal) pain. The most common severe abnormal laboratory test results with EPKINLY when used alone include decreased white blood cells, decreased red blood cells, and decreased platelets.

The most common side effects of EPKINLY when used together with lenalidomide and rituximab in FL include rash, upper respiratory tract infections, tiredness, injection site reactions, constipation, diarrhea, CRS, pneumonia, COVID-19, and fever. The most common severe abnormal laboratory test results with EPKINLY when used together with lenalidomide and rituximab include decreased white blood cells and decreased platelets.

These are not all of the possible side effects of EPKINLY. Call your doctor for medical advice about side effects.

You are encouraged to report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch or to Genmab US, Inc. at 1-855-4GENMAB (1-855-443-6622).

Please see Medication Guide, including Important Warnings.

About Genmab

Genmab is an international biotechnology company dedicated to improving the lives of people with cancer and other serious diseases through innovative antibody medicines. For over 25 years, its passionate, innovative and collaborative team has advanced a broad range of antibody-based therapeutic formats, including bispecific antibodies, antibody–drug conjugates (ADCs), immune-modulating antibodies and other next-generation modalities. Genmab’s science powers eight approved antibody medicines, and the company is advancing a strong late-stage clinical pipeline, including wholly owned programs, with the goal of delivering transformative medicines to patients.

Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit Genmab.com and follow us on LinkedIn and X.

This Media Release contains forward looking statements. The words “believe,” “expect,” “anticipate,” “intend” and “plan” and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with preclinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmab’s most recent financial reports, which are available on www.genmab.comand the risk factors included in Genmab’s most recent Annual Report on Form 20-F and other filingswith the U.S. Securities and Exchange Commission (SEC), which are available at www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Media Release nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab®; the Y-shaped Genmab logo®; Genmab in combination with the Y-shaped Genmab logo®; HuMax®; DuoBody®; HexaBody®; DuoHexaBody®, HexElect® and KYSO®. EPCORE®, EPKINLY®, TEPKINLY® and their designs are trademarks of AbbVie Biotechnology Ltd.

____________________

i Lymphoma Research Foundation. Diffuse Large B-Cell Lymphoma. Accessed February 2026. https://lymphoma.org/understanding-lymphoma/aboutlymphoma/nhl/dlbcl/

ii Padala, et al. Diffuse Large B-Cell Lymphoma. StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan. 2023 Apr 24.

iii Sehn, et al. Diffuse Large B-Cell Lymphoma. N Engl J Med. 2021;384:842-858. doi: 10.1056/NEJMra2027612.

iv Kanas, et al. Epidemiology of Diffuse Large B-Cell Lymphoma (DLBCL) and Follicular Lymphoma (FL) in the United States and Western Europe: Population-Level Projections for 2020-2025. Leuk Lymphoma. 2022;63(1):54-63. doi: 10.1080/10428194.2021.1975188.

v Crump, et al. Outcomes in Refractory Diffuse Large B-Cell Lymphoma: Results From the International SCHOLAR-1 Study. Blood. 2017;130(16):1800-1808. doi: 10.1182/blood-2017-03-769620.

vi Engelberts PJ, Hiemstra IH, de Jong B, et al. DuoBody-CD3xCD20 induces potent T-cell-mediated killing of malignant B cells in preclinical models and provides opportunities for subcutaneous dosing. EBioMedicine. 2020;52:102625. DOI: 10.1016/j.ebiom.2019.102625.

 

David Freundel, Senior Director, Global Communications & Corporate Affairs

T: +1 609 613 0504; E: [email protected]

Andrew Carlsen, Vice President, Head of Investor Relations

T: +45 3377 9558; E: [email protected]

KEYWORDS: Europe Sweden Denmark

INDUSTRY KEYWORDS: Cardiology Seniors Biotechnology Pharmaceutical Oncology Health Consumer Clinical Trials

MEDIA:

Logo
Logo
Posted on

Verra Mobility Corporation (VRRM) Securities Fraud Class Action Lawsuit Filed; August 4, 2026, Lead Plaintiff Deadline

PR Newswire

Did you buy
VRRM common stock
between February 24, 2026 and May 26, 2026?

Affected VRRM Investor Summary

  • Who: Verra Mobilty Corporation (NASDAQ: VRRM)
  • What: Securities fraud class action lawsuit filed
  • Class Period: February 24, 2026 through May 26, 2026
  • Deadline to Seek Lead Plaintiff Status: August 4, 2026
  • Key Lawsuit Allegations: Material misstatements and/or omissions concerning the company’s continued growth in its Commercial Services business and contract with Avis Budget Group.
  • Investor Action: Contact Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) for recovery options

RADNOR, Pa., June 13, 2026 /PRNewswire/ — Kessler Topaz Meltzer & Check, LLP (www.ktmc.com), a nationally recognized securities litigation law firm, informs investors that a securities fraud class action lawsuit has been filed against Verra Mobility Corporation (Verra) (NASDAQ: VRRM) on behalf of those who purchased or acquired Verra common stock between February 24, 2026 and May 26, 2026, inclusive. The lawsuit is filed in the United States District Court for the District of Arizona and is captioned Otucu v. Verra Mobility Corporation, Case No.2:26-cv-03973 (D. Ariz.). Investors have until August 4, 2026, to file for lead plaintiff status.

KTMC Icon


CONTACT KTMC TO DISCUSS YOUR LEGAL RIGHTS:

If you purchased or acquired Verra common stock and have lost money on your investment, you are encouraged to contact KTMC attorney Jonathan Naji, Esq. at:

Phone: (484) 270-1453
Email: [email protected]
Website: https://www.ktmc.com/vrrm-verra-mobility-corporation-class-action-lawsuit?utm_source=PR_Newswire&utm_medium=pressrelease&utm_campaign=vrrm&mktm=PR

There is no cost or obligation to speak with an attorney.


VERRA MOBILITY CORPORATION


CLASS ACTION LAWSUIT – COMPLAINT ALLEGATION SUMMARY:

The complaint alleges that, throughout the Class Period, Defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the company’s business, operations, and prospects. Specifically, Defendants failed to disclose to investors that: (1) Verra’s optimistic plan for continued growth in its Commercial Services business was dependent on its relationship with Avis, and in particular obtaining a contract extension with Avis Budget Group; (2) Verra minimized concerns that major rent-a-car customers could replace Verra with in-house solutions or outsourced alternatives, making Verra’s 2026 full year guidance increasingly unlikely to be met; and (3) as a result, Defendants’ positive statements about the company’s business, operations, and prospects were materially misleading and/or lacked a reasonable basis at all relevant times.

Why did Verra’s Stock Drop?
On May 26, 2026, Verra disclosed that the company had received a termination notice from Avis Budget Group regarding its contract, which becomes effective in September 2026. Verra further disclosed that it “expects the termination to reduce Commercial Services’ 2026 annualized revenue by approximately $135 million to $145 million and 2026 annualized segment profit by approximately $120 million to $125 million, before taking into account expected cost reduction initiatives.” Verra accordingly lowered its full year 2026 financial outlook. On this news, Verra’s stock price fell $9.23 per share, or 70.6%, to close at $3.85 per share on May 27, 2026.

On June 1, 2026, Verra announced that its President and Chief Executive Officer had been terminated as “the Board determined that a change in leadership [was] needed[.]”


WHAT VRRM INVESTORS CAN DO NOW:

  1. File to be lead plaintiff by August 4, 2026.
  2. Contact KTMC for a free case evaluation. All representation is on a contingency fee basis, there is no cost to you.
  3. Retain counsel of choice or take no action.


THE LEAD PLAINTIFF PROCESS FOR VERRA MOBILITY CORPORATION INVESTORS:

Verra investors may, no later than August 4, 2026, seek to be appointed as a lead plaintiff representative of the class through Kessler Topaz Meltzer & Check, LLP or other counsel, or may choose to do nothing and remain an absent class member. A lead plaintiff is a representative party who acts on behalf of all class members in directing the litigation. The lead plaintiff is usually the investor or small group of investors who have the largest financial interest and who are also adequate and typical of the proposed class of investors. The lead plaintiff selects counsel to represent the lead plaintiff and the class and these attorneys, if approved by the court, are lead or class counsel. Your ability to share in any recovery is not affected by the decision of whether or not to serve as a lead plaintiff.

Kessler Topaz Meltzer & Check, LLP encourages Verra investors to contact the firm for more information.


ABOUT KESSLER TOPAZ MELTZER & CHECK, LLP (KTMC):


Kessler Topaz Meltzer & Check, LLP (KTMC) is a leading U.S. plaintiff-side law firm focused on securities-fraud class actions and global investor protection. The firm represents individual investors as well as institutions, such as major pension funds, asset managers, and international investors. KTMC has led some of the largest recoveries in securities litigation and has been recognized by peers and the legal media with numerous accolades, including The National Law Journal’s Plaintiff’s Hot List and Trailblazers in Plaintiffs’ Law, BTI Consulting Group’s Honor Roll of Most Feared Law Firms, The Legal Intelligencer’s Class Action Firm of the Year, Lawdragon’s Leading Plaintiff Financial Lawyers, and Law360’s Titans of the Plaintiffs Bar. The firm operates globally with offices in Pennsylvania and California. KTMC has recovered over $25 billion for our clients and the classes they represent. For more information about Kessler Topaz Meltzer & Check, LLP, please visit www.ktmc.com. The complaint in this matter was not filed by KTMC.

CONTACT:
Jonathan Naji, Esq.
(484) 270-1453
280 King of Prussia Road
Radnor, PA 19087
[email protected]

May be considered attorney advertising in certain jurisdictions. Past results do not guarantee future outcomes.

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/verra-mobility-corporation-vrrm-securities-fraud-class-action-lawsuit-filed-august-4-2026-lead-plaintiff-deadline-302799274.html

SOURCE Kessler Topaz Meltzer & Check, LLP

Posted on

Lilly to present initial clinical data for first-in-class type II JAK2 inhibitor in patients with previously treated myelofibrosis at the 2026 EHA Annual Meeting

PR Newswire

INDIANAPOLIS, June 13, 2026 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) today announced new data from the Phase 1 AJX-101 study showing that its investigational type II JAK2 inhibitor (AJ1-11095) demonstrated an encouraging safety profile and promising clinical activity in patients with myelofibrosis who have been failed by a type I JAK2 inhibitor. This first-in-class type II JAK2 inhibitor was designed to selectively bind the type II conformation of the JAK2 kinase in order to potentially provide greater efficacy than existing therapies and a novel treatment option for patients who become resistant to type I JAK2 inhibitors. Lilly recently added this program to its pipeline following the completion of the acquisition of Ajax Therapeutics, Inc.

These data will be highlighted in an oral presentation at the 2026 European Hematology Association (EHA) Annual Meeting taking place in Stockholm, Sweden (Abstract number: S218) and featured in the meeting’s press program.

“Patients with myelofibrosis who have been previously treated with an existing type I JAK2 inhibitor face very limited treatment options, highlighting an urgent need for new therapies,” said John Mascarenhas, MD, professor of medicine, Icahn School of Medicine at Mount Sinai and principal investigator of the AJX-101 study. “These early clinical findings suggest that selective targeting of the type II conformation of JAK2 may provide a differentiated approach. With an encouraging safety profile, meaningful spleen size reduction, symptom improvement, and decrease in underlying mutant disease burden, these data, while early, point to the potential to meaningfully impact treatment options for people with certain myeloproliferative neoplasms.”

AJX-101 is the first clinical trial to evaluate a type II selective JAK2 inhibitor in patients with myelofibrosis. The trial enrolled 23 patients across five dose levels (25, 50, 75, 100, and 125 mg once daily) in its dose escalation phase. Patients had received a median of two prior therapies, and all had previously received a type I JAK2 inhibitor. The trial enrolled patients across all major myelofibrosis subtypes and driver mutations.

AJ1-11095 demonstrated responses across the standard efficacy endpoints of spleen volume reduction and symptom improvement.1 The SVR35 rate, a reduction in spleen volume of at least 35%, was observed as best response in 70% of patients. The TSS50 rate, indicating at least a 50% improvement in symptom burden, was also seen in 70% of patients at week 12. In addition, reductions in driver mutation variant allele frequency (VAF) were observed in 21 out of 23 patients. Among the 17 patients who reached week 24 of treatment, 59% saw a reduction of 20% or greater and 35% saw a reduction of 50% or greater, including JAK2, MPL, and CALR type 1 and type 2 mutations. VAF reductions are uncommonly observed with existing type I JAK2 inhibitors.2

The overall safety profile for the medicine was generally manageable. No dose-limiting toxicities were observed, and most patients enrolled in the dose escalation phase remain on study (78%).3 The most common treatment-emergent adverse events across all dose levels included anemia, dysgeusia, decreased platelet count, and increased alanine aminotransferase.

“The depth of response seen across spleen, symptoms, and VAF from these early phase results is in excess of what has been seen historically in this disease setting,” said Jacob Van Naarden, executive vice president and president of Lilly Oncology. “These data provide clear proof of concept for what this selective type II JAK2 inhibitor could mean for patients with myelofibrosis and shed light on the conviction we brought to the acquisition of Ajax. With this program now officially part of Lilly’s pipeline, we are committed to rapidly advancing it through clinical development and further exploring its potential to meaningfully improve outcomes for people with myeloproliferative neoplasms across a range of disease settings.”

AJ1-11095 is currently being evaluated in an expansion cohort in second-line myelofibrosis, with plans to investigate in patients with high-risk polycythemia vera and those with myelofibrosis who have not yet received a JAK2 inhibitor. Details on the AJX-101 trial can be found by visiting clinicaltrials.gov.

About AJ1
-11095

AJ1-11095 is an investigational, oral, first-in-class type II JAK2 inhibitor. AJ1-11095 is designed to bind JAK2 in its inactive conformation — an approach intended to more completely suppress the aberrant signaling that drives myelofibrosis, in contrast to currently approved JAK2 inhibitors that bind JAK2 in its active state. AJ1-11095 demonstrated superior activity compared to ruxolitinib in preclinical models of myelofibrosis. AJ1-11095 is currently being studied in AJX-101, a global, open-label, multicenter, Phase 1 study in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis who have previously been treated with a type I JAK2 inhibitor, NCT06343805.

About Lilly

Lilly is a medicine company turning science into healing to make life better for people around the world. We’ve been pioneering life-changing discoveries for 150 years, and today our medicines help tens of millions of people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world’s most significant health challenges: redefining diabetes care; treating obesity and curtailing its most devastating long-term effects; advancing the fight against Alzheimer’s disease; providing solutions to some of the most debilitating immune system disorders; and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we’re motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visit Lilly.com and Lilly.com/news, or follow us on Facebook, Instagram, and LinkedIn. P-LLY

© Lilly USA, LLC 2026. ALL RIGHTS RESERVED.

Trademarks and Trade Names

All trademarks or trade names referred to in this press release are the property of the company, or, to the extent trademarks or trade names belonging to other companies are referenced in this press release, the property of their respective owners. Solely for convenience, the trademarks and trade names in this press release are referred to without the ® and ™ symbols, but such references should not be construed as any indicator that the company or, to the extent applicable, their respective owners will not assert, to the fullest extent under applicable law, the company’s or their rights thereto. We do not intend the use or display of other companies’ trademarks and trade names to imply a relationship with, or endorsement or sponsorship of us by, any other companies.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about AJ1-11095 as a potential treatment for adults with myelofibrosis and other myeloproliferative neoplasms, and the timeline for future studies, regulatory submissions, presentations, and other milestones relating to AJ1-11095 and the AJX-101 clinical program, and reflects Lilly’s current beliefs and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of drug research, development, and commercialization. Among other things, there is no guarantee that planned or ongoing studies will be completed as planned or that future study results will be consistent with study results to date. For further discussion of these and other risks and uncertainties that could cause actual results to differ from Lilly’s expectations, see Lilly’s Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.


Refer to:

Megan Talon; [email protected] (Media)

Michael Czapar; [email protected] (Investors)



_________________________________
1
 May 28, 2026 data cutoff
2 Meyer SC et al Cancer Cell 2015: 28:P15-28
3 May 12, 2026 data cutoff

Eli Lilly and Company logo. (PRNewsFoto, Eli Lilly and Company)

 

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/lilly-to-present-initial-clinical-data-for-first-in-class-type-ii-jak2-inhibitor-in-patients-with-previously-treated-myelofibrosis-at-the-2026-eha-annual-meeting-302799309.html

SOURCE Eli Lilly and Company

Posted on

Incyte Announces New Positive Data at EHA 2026 Showed INCA033989 Achieved Rapid, Robust and Sustained Clinical and Molecular Responses and Was Well Tolerated in Patients with Myelofibrosis and Essential Thrombocythemia

Incyte Announces New Positive Data at EHA 2026 Showed INCA033989 Achieved Rapid, Robust and Sustained Clinical and Molecular Responses and Was Well Tolerated in Patients with Myelofibrosis and Essential Thrombocythemia

  • In myelofibrosis (MF), INCA033989 delivered rapid and durable clinical benefits including meaningful spleen volume reductions, symptom improvement and anemia responses, both as a monotherapy and in combination with ruxolitinib
  • In essential thrombocythemia (ET), 87% of patients achieved a hematologic response, including 70% complete responses; responses were rapid (median ~2 weeks to a durable complete hematologic response) and durable (median response duration of 23 weeks)
  • Across MF and ET, INCA033989 consistently reduced mutant CALR (mutCALR) variant allele frequency (VAF) in most evaluable patients, with reductions correlating with clinical responses and supporting its potential for disease modification
  • First-in-class mutCALR-targeted antibody shows potential to modify disease biology in both MF and ET
  • INCA033989 demonstrated a favorable and manageable safety profile with no dose-limiting toxicities, with most patients with MF and ET continuing treatment

WILMINGTON, Del.–(BUSINESS WIRE)–
Incyte (Nasdaq:INCY) today announced updated clinical data from two Phase 1 studies evaluating the safety, tolerability and efficacy of INCA033989, a first-in-class mutant calreticulin (mutCALR)-targeted monoclonal antibody, in patients with mutCALR-expressing myeloproliferative neoplasms (MPNs). INCA033989 demonstrated rapid, clinically meaningful responses and consistent molecular activity across both myelofibrosis (MF) and essential thrombocythemia (ET), with convergent evidence supporting the potential for disease modification.

These findings are being presented in oral and poster presentations at the European Hematology Association (EHA) 2026 Congress in Stockholm, Sweden (Session: Myeloproliferative neoplasms – Clinical, Presentation numbers: S216, PS1983, PF884).

“The data presented at EHA 2026 demonstrate clinically meaningful and consistent responses with INCA033989 across both myelofibrosis and essential thrombocythemia,” said Pablo J. Cagnoni, M.D., President of Incyte and Global Head of Research and Development. “What distinguishes INCA033989 is its potential to deliver disease control while targeting the biology that drives it. We remain on track to initiate our pivotal ET study by mid-2026 and are actively engaging regulators on a pivotal MF program.”

Results in Patients with Myelofibrosis (MF)

The safety, tolerability, and efficacy of INCA033989 in Type 1 and non-Type 1 patients with MF harboring a CALR mutation is being evaluated in two ongoing Phase 1 studies. Results demonstrate that INCA033989 delivers broad, clinically meaningful improvements across spleen volume, symptom burden and anemia in patients with MF. As a monotherapy and in combination with ruxolitinib, INCA033989 had a manageable safety profile and the majority of patients remained on treatment – no dose-limiting toxicities were observed, and a maximum tolerated dose was not reached.

Monotherapy: INCA033989 was evaluated as monotherapy in patients who were resistant, refractory or intolerant to JAK inhibitor treatment after >12 weeks (JAK R/R/I), or ineligible to JAK inhibitor therapy. The dose escalation cohort evaluated INCA033989 from 24-3500 mg, and the dose expansion cohort evaluated 250 mg and 2000 mg.

INCA033989 monotherapy demonstrated durable clinical benefit, with clinically meaningful improvements across spleen volume, symptoms and anemia across both JAK R/R/I and JAK ineligible patients.

  • Spleen Volume Reduction (SVR): Rapid and robust spleen volume reductions were observed in patients, with 55% (38/69) and 39% (27/69) of patients achieving the best SVR25 and SVR35 reduction, respectively. At Week 24, 27% (17/62) patients achieved SVR35, including 47% (8/17) JAK ineligible and 20% (9/45) JAK R/R/I. Robust responses were observed in JAK ineligible patients regardless of mutation type (60% [6/10] Type-1 vs. 29% [2/7] non-Type 1). In JAK R/R/I patients, clinically meaningful reductions were observed in 31% (8/26) of Type-1 patients across all evaluated doses at Week 24, and 33% (1/3) of non-Type-1 patients evaluated at 2500 mg, the highest evaluated dose.
  • Symptom Improvement: Improvements in symptoms were also observed in the majority of patients, with 53% of patients achieving at least a 50% best TSS reduction (TSS50). At Week 24, 32% of patients achieved TSS50, including 29% and 33% of JAK ineligible and JAK R/R/I patients, respectively.
  • Anemia: Rapid and durable anemia improvements were observed in most patients, with anemia response occurring in 60% of evaluable anemic patients, and 52% of patients achieved a major anemia response. Improvements in anemia were observed across patients regardless of prior JAK exposure, including 63% of JAK R/R/I patients and 55% of JAK ineligible patients.
  • Molecular: Consistent reductions in variant allele frequency (VAF) were observed across most patients, regardless of prior JAK exposure and mutation type, with 89% of patients achieving a reduction in whole blood mutCALR VAF (Type 1: 90%, Non-Type 1: 88%), and 81% of patients achieving a ≥25% reduction in mutCALR peripheral blood mononuclear cells (PBMC) from baseline (Type 1: 62%, Non-Type 1: 38%).

INCA033989 was generally well-tolerated, with 84% (70/83) of patients remaining on therapy at the time of the data cut off. Treatment emergent adverse events (TEAEs) occurred in 92% (76) of patients, with 27% (22) of patients experiencing Grade ≥3 TEAEs, the most frequent of which were cytopenias. No dose-limiting toxicities were observed, and discontinuations due to TEAEs were limited (n=2).

Combination therapy: INCA033989 (dose range: 70 to 2,500 mg) was evaluated in combination with ruxolitinib in patients with MF who experience a suboptimal response to ruxolitinib monotherapy. INCA033989 demonstrated additive, multi-domain clinical activity in patients when administered in combination with ruxolitinib.

  • SVR: At Week 24, 55% (11/20) and 30% (6/20) of patients achieved SVR25 and SVR35, respectively.
  • Symptom Improvement: 31% (5/16) of patients achieved TSS50 at Week 24.
  • Anemia: Anemia response occurred in 35% (6/17) of evaluable anemic patients.

INCA033989 in combination with ruxolitinib was generally well-tolerated, with 76% (16) of patients remaining on treatment at the time of the data cut off.In the combination arm (n=21), all patients experienced TEAEs. Grade ≥3 TEAEs were reported in 67% (14) of patients, most commonly anemia (33%).

Translational data

  • Clinical response occurred regardless of mutational complexity with SVR, anemia and molecular responses observed in patients with and without high molecular risk (HMR) mutations.

  • 93% of patients with HMR had a reduction in whole blood mutCALR VAF, as did 88% of those without HMR mutations.

  • A reduction in mutCALR-positive hematopoietic stem and progenitor cells (HSPCs) was also seen, indicating activity at the level of disease-initiating cells.

“Patients with CALR-mutated MF have distinct disease biology and often respond poorly to available therapies, underscoring the need for treatments targeting the underlying driver of disease,” said Claire Harrison, M.D., Professor of MPNs and Deputy Chief Medical Officer, Guy’s and St. Thomas’ NHS Foundation Trust. “What stands out in these data is that INCA033989 produced rapid and robust spleen, symptom and anemia responses, alongside reductions in mutCALR allele burden regardless of HMR mutations, pointing to activity at the level of the disease-initiating clone.”

Results in Patients with Essential Thrombocythemia

Inpatients with ET, INCA033989 demonstrated rapid, deep and durable hematologic and molecular responses across both Type 1 and non-Type 1 CALR patients, supporting potential for disease modification in a population resistant or intolerant to prior cytoreductive therapy.

Hematologic Response:

  • Across doses, 70% (80/114) of patients achieved a complete hematologic response (CHR, platelet count ≤400 × 109/L and leukocytes <10 × 109/L) and 87% achieved complete or partial hematologic response (CHR/PHR, platelet count ≤600 × 109/L and leukocytes <10 × 109/L).

  • 81% of patients with Type 1 mutCALR achieved a durable (>12 weeks) CHR at 750 mg and above; and 50% of patients with non–Type 1 mutCALR achieved a durable CHR/PHR at 2500 mg. The median time to onset of durable CHR was 2.1 weeks.

Molecular Response and Disease Biology:

  • ≥25% reduction in mutCALR VAF correlated with durable CHR (nominal P<0.0001, n=103).

  • Of the patients who achieved a CHR and had ≥1 post-baseline VAF assessment, 73% achieved ≥25% reduction in VAF.

  • Durable molecular response was observed in both Type 1 and non–Type 1 mutCALR.

  • A reduction in mutCALR megakaryocytes was seen in both Type 1 and non-Type 1 patients treated with INCA033989

INCA033989 was well tolerated with 95% of patients remaining on treatment. The median duration of INCA033989 exposure was 8.1 months (range from 0.59 to 27.0 months). A low incidence of Grade ≥3 adverse events was observed (19%); the most common were neutropenia (4.4%) and lipase increase (3.5%). Grade ≥3 cytopenia TEAEs occurred in 6% (7/114) of patients; no Grade ≥3 thrombocytopenia TEAEs were observed.

“In patients with ET who were resistant to or intolerant of prior cytoreductive therapy, INCA033989 resulted in rapid and durable normalization of platelet counts with accompanying molecular responses, with the majority of patients achieving a CHR,” said John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mt. Sinai and Director, Center of Excellence for Blood Cancers and Myeloid Disorders, The Tisch Cancer Institute. “As there are currently no mutation-specific treatments available for patients with ET, this approach is critically important for this high-risk patient population. These results provide a strong foundation for advancing INCA033989 into a registrational Phase 3 study.”

In November of 2025, INCA033989 was granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with ET harboring a Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy. A Phase 3 study of INCA033989 in mutCALR positive patients with ET who are resistant or intolerant to at least one prior cytoreductive therapy (EXCALIBUR-ET2, NCT07623200) is being initiated in mid-2026.

More information regarding the EHA 2026 Congress can be found on the EHA website: https://ehaweb.org/connect-network/eha2026-congress.

About Myeloproliferative Neoplasms (MPNs) and Mutations in Calreticulin (mutCALR)

Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),1 a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.2,3 Among two types of MPNs, essential thrombocythemia (ET) and myelofibrosis (MF), mutCALR drives 25-35% of all cases.4 In MF, it is estimated that 70-83% of CALR mutations in the U.S. are identified as Type 1, with 15-30% identified as non-Type 1.4,5 There are currently no targeted therapies for CALR mutations.

Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy. INCA033989 received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with ET harboring a Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy. A Phase 3 study of INCA033989 in patients with ET with a Type 1 or non-Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy is being initiated (EXCALIBUR-ET2, NCT07623200).

About the INCA33989-101 & INCA33989-102 Trials

The clinical trial program for INCA033989 includes two multicenter, open-label Phase 1 studies, INCA33989-101 (NCT05936359) and INCA33989-102 (NCT06034002). The studies are evaluating the safety, tolerability and efficacy of INCA033989 in ~455 adult (≥18 years old) patients with mutCALR-expressing myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET) and myelofibrosis (MF).

The primary endpoint of the studies is measured by the number of participants with dose limiting toxicities (DLTs), treatment-emergent adverse events (TEAEs) and the number of participants with TEAEs leading to dose modification or discontinuation. Secondary endpoints include response rates, mean change of ET total symptom score, percentage of MF patients achieving spleen volume reduction, MF patient anemia response, mean change in disease-related allele burden and various pharmacokinetics measures.

For more information on the studies, please visit: https://clinicaltrials.gov/study/NCT05936359 and https://clinicaltrials.gov/study/NCT06034002.

About Incyte®

Incyte is redefining what’s possible in biopharmaceutical innovation. Through deep scientific expertise and a relentless focus on patients, we have built an established portfolio of first-in-class medicines and an extensive portfolio of next-generation medicines across our key franchises: Hematology, Oncology and Inflammation & Autoimmunity.

To learn more, visit Incyte.com and Investor.Incyte.com. Follow us on social media: LinkedIn, X and Instagram.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the presentation of data for INCA033989; the potential for disease modification and the potential to benefit patients offered by INCA033989; expectations regarding ongoing and future clinical trials, including the timing of such trials; and Incyte’s aspirations and goals as set forth under the heading “About Incyte.”

Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including the sufficiency of clinical trial data to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; the efficacy or safety of Incyte’s products; Incyte’s ability to achieve commercial success for its products, once approved; Incyte’s ability to obtain and maintain protection of intellectual property for its products and technology; Incyte’s reliance on third parties and partners; the acceptance of Incyte’s products in the marketplace; market competition, sales, marketing, manufacturing and distribution requirements; greater than expected expenses, including expenses relating to litigation or strategic activities; and those risks and uncertainties discussed in greater detail in Incyte’s reports filed with the U.S. Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2025, and its quarterly report on Form 10-Q for the quarter ended March 31, 2026. Incyte disclaims any intent or obligation to update these forward-looking statements.

1 Raghavan, M., Wijeyesakere S.J., Peters L.R., Del Cid N. (2013) Calreticulin in the immune system: ins and outs. Trends in Immunology, 34(1):13-21. Link to source (https://www.cell.com/trends/immunology/abstract/S1471-4906(12)00131-7?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1471490612001317%3Fshowall%3Dtrue)

2 Nangalia J. Massie C.E., Baxter E.J., Nice F.L., et al. (2013) Somatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2. New England Journal of Medicine, 369(25):2391-2405. Link to source (https://www.nejm.org/doi/10.1056/NEJMoa1312542?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov)

3 Klampfl T., Gisslinger, H., Harutyunyan A.S., et al. (2013) Somatic mutations of calreticulin in myeloproliferative neoplasms. New England Journal of Medicine, 369(25):2379-2390. Link to source (https://www.nejm.org/doi/10.1056/NEJMoa1311347?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200www.ncbi.nlm.nih.gov)

4 Salzman G. and Mullally A. (2026) Novel strategies targeting mutant calreticulin in essential thrombocythemia and myelofibrosis. Blood, 147(12):1267-1277. Link to source (https://doi.org/10.1182/blood.2025028642)

5 Guglielmelli, P., Maccari, C., Sordi, B. et al. Phenotypic correlations of CALR mutation variant allele frequency in patients with myelofibrosis. Blood Cancer J. 13, 21 (2023). Link to source (https://doi.org/10.1038/s41408-023-00786-x)

Contacts:

Media

[email protected]

Investors

[email protected]

KEYWORDS: Delaware Europe Sweden United States North America

INDUSTRY KEYWORDS: Biotechnology Pharmaceutical Health Clinical Trials

MEDIA:

Logo
Logo
Posted on

PicS N.V. (PICS) Class Action Lawsuit: Investors Face August 4, 2026, Deadline

PR Newswire

Did you buy
PICS Class A common stock on or around January 30, 202
6?

Affected PICS Investor Summary

  • Who: PicS N.V. (NASDAQ: PICS)
  • What: Securities fraud class action lawsuit filed
  • Class Period: pursuant and/or traceable to PicS’s initial public offering (IPO) on or about January 30, 2026
  • Deadline to Seek Lead Plaintiff Status: August 4, 2026
  • Key Lawsuit Allegations: Material misstatements and/or omissions concerning the company’s credit models and user data.
  • Investor Action: Contact Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) for recovery options

RADNOR, Pa., June 13, 2026 /PRNewswire/ — Kessler Topaz Meltzer & Check, LLP (www.ktmc.com), a nationally recognized securities litigation law firm, informs investors that a securities fraud class action lawsuit has been filed against PicS N.V. (PicS) (NASDAQ: PICS) on behalf of those who purchased or acquired PicS Class A common stock pursuant and/or traceable to PicS’s January 30, 2026 IPO. The lawsuit is filed in the United States District Court for the Southern District of New York and is captioned FirstFire Global Opportunities Fund, LLC v. PicS N.V., Case No. 1:26-cv-04793 (S.D.N.Y).  Investors have until August 4, 2026, to file for lead plaintiff status. 

KTMC Icon


CONTACT KTMC TO DISCUSS YOUR LEGAL RIGHTS:


If you purchased or acquired PicS Class A common stock and have lost money on your investment, you are encouraged to contact KTMC attorney Jonathan Naji, Esq. at:

Phone: (484) 270-1453
Email: [email protected]
Website: https://www.ktmc.com/pics-pics-nv-class-action-lawsuit?utm_source=PR_Newswire&utm_medium=pressrelease&utm_campaign=pics&mktm=PR  

There is no cost or obligation to speak with an attorney.


PICS N.V.


CLASS ACTION LAWSUIT – COMPLAINT ALLEGATION SUMMARY:

The complaint alleges that PicS’s IPO documents contained materially false and/or misleading statements, as well as failed to disclose material adverse facts about the company’s business, operations, and prospects. Specifically, Defendants failed to disclose to investors that: (1) PicS had conducted an evaluation of its credit evaluation procedures in December 2025 and determined that such procedures were deficient and in need of enhancement; (2) as a result of the new procedures PicS had implemented in December 2025, PicS had reclassified approximately R$590 million of exposures previously classified as Stage 2 to Stage 3, leading to an incremental Expected Credit Loss (ECL) charge of R$88 million in the three months ended December 31, 2025; (3) PicS had experienced a heightened, but unreported, Stage 3 formation rate of more than 7% in the fourth quarter of 2025 that deviated substantially from the historical results and trends provided in the IPO documents; (4) the IPO documents had materially overstated the quality and ability of PicS’s credit models and user data to inform the company’s underwriting practices and to allow PicS to timely and effectively monitor, assess, and identify adverse credit events, credit risks, and credit deterioration across its portfolio; (5) PicS suffered from degradations in customer credit quality and heightened risks of default and loan impairment as a result of its entrance into materially riskier business lines leading up to the IPO, resulting in undisclosed adverse financial and operational trends such as heightened incidents of default, which predated the IPO and were internally projected by PicS to continue to worsen following the IPO; and (6) as a result, Defendants’ positive statements about the company’s business, operations, and prospects were materially misleading and/or lacked a reasonable basis at all relevant times.

At the time of filing of the complaint, PicS’s stock price had fallen to a low of less than $9 per share, representing a more than 50% decline from the $19 per share IPO price.


WHAT PICS INVESTORS CAN DO NOW:

  1. File to be lead plaintiff by August 4, 2026.
  2. Contact KTMC for a free case evaluation. All representation is on a contingency fee basis, there is no cost to you.
  3. Retain counsel of choice or take no action.


THE LEAD PLAINTIFF PROCESS FOR PICS N.V. INVESTORS:

PicS investors may, no later than August 4, 2026, seek to be appointed as a lead plaintiff representative of the class through Kessler Topaz Meltzer & Check, LLP or other counsel, or may choose to do nothing and remain an absent class member. A lead plaintiff is a representative party who acts on behalf of all class members in directing the litigation.  The lead plaintiff is usually the investor or small group of investors who have the largest financial interest and who are also adequate and typical of the proposed class of investors. The lead plaintiff selects counsel to represent the lead plaintiff and the class and these attorneys, if approved by the court, are lead or class counsel. Your ability to share in any recovery is not affected by the decision of whether or not to serve as a lead plaintiff.

Kessler Topaz Meltzer & Check, LLP encourages PicS investors to contact the firm for more information.


ABOUT KESSLER TOPAZ MELTZER & CHECK, LLP (KTMC):
    
Kessler Topaz Meltzer & Check, LLP (KTMC) is a leading U.S. plaintiff-side law firm focused on securities-fraud class actions and global investor protection. The firm represents individual investors as well as institutions, such as major pension funds, asset managers, and international investors. KTMC has led some of the largest recoveries in securities litigation and has been recognized by peers and the legal media with numerous accolades, including The National Law Journal’s Plaintiff’s Hot List and Trailblazers in Plaintiffs’ Law, BTI Consulting Group’s Honor Roll of Most Feared Law Firms, The Legal Intelligencer’s Class Action Firm of the Year, Lawdragon’s Leading Plaintiff Financial Lawyers, and Law360’s Titans of the Plaintiffs Bar.  The firm operates globally with offices in Pennsylvania and California.  KTMC has recovered over $25 billion for our clients and the classes they represent.  For more information about Kessler Topaz Meltzer & Check, LLP, please visit www.ktmc.com.  The complaint in this matter was not filed by KTMC.

CONTACT:
Jonathan Naji, Esq.
(484) 270-1453
280 King of Prussia Road
Radnor, PA 19087
[email protected]

May be considered attorney advertising in certain jurisdictions.  Past results do not guarantee future outcomes.

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/pics-nv-pics-class-action-lawsuit-investors-face-august-4-2026-deadline-302799265.html

SOURCE Kessler Topaz Meltzer & Check, LLP

Posted on

Agios Showcases RISE UP Phase 3 Results at EHA 2026 Plenary Session Reinforcing Strong Anti-Hemolytic Profile of Mitapivat in Sickle Cell Disease

  • Mitapivat demonstrated statistically significant improvement in hemoglobin response compared with placebo, with rapid onset and durable effects
  • New analyses showed patients in mitapivat arm had clinically meaningful reduction in transfusion burden compared with placebo
  • Patients in mitapivat arm who achieved hemoglobin response had clinically meaningful benefits across measures of sickle cell pain crises, fatigue, and other patient-reported outcomes
  • Mitapivat was well-tolerated, with a safety profile consistent with previous trials of mitapivat in sickle cell disease
  • Company to host investor conference call and webcast today at 9:00 a.m. ET (3:00 p.m. CEST)

CAMBRIDGE, Mass., June 13, 2026 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today presented detailed results from the 52-week double-blind period of the global RISE UP Phase 3 trial of mitapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years or older with sickle cell disease. These efficacy and safety results, which include new transfusion burden and hemoglobin responder analyses reinforcing the strong anti-hemolytic profile of mitapivat, were presented during the distinguished Plenary Abstracts Session at the 31st European Hematology Association (EHA) Congress (EHA 2026) in Stockholm, Sweden.

In November 2025, topline results from RISE UP demonstrated a significant improvement in the trial’s primary endpoint of hemoglobin response with mitapivat compared with placebo. The trial also met two key secondary endpoints, showing rapid and durable improvements in hemoglobin concentration and indirect bilirubin, a marker of hemolysis (red blood cell destruction). Although mitapivat showed a reduction in the annualized rate of sickle cell pain crises (SCPCs) compared with placebo, this primary endpoint did not reach statistical significance, and there was no overall difference between mitapivat and placebo for the key secondary endpoint measuring patient-reported fatigue. However, patients in the mitapivat arm who achieved a hemoglobin response experienced clinically meaningful reductions in the annualized rate of SCPCs and related hospitalizations, as well as improvements in fatigue.

New RISE UP analyses, not previously disclosed by the company, further highlight the potential for mitapivat to offer clinical benefits for patients with sickle cell disease, as evidenced by a clinically meaningful reduction in transfusion burden and, for hemoglobin responders in the mitapivat arm, improvements observed across additional measures of pain and physical function.

“Patients living with sickle cell disease are in critical need of new treatments that can effectively manage the debilitating impact of their condition,” said Biree Andemariam, M.D., Professor of Medicine and American Red Cross Endowed Chair in Transfusion Medicine, University of Connecticut Health, and a RISE UP trial investigator. “The RISE UP Phase 3 data presented today showcase the strong anti-hemolytic profile of mitapivat, with rapid and durable improvements in both hemoglobin and indirect bilirubin as well as a meaningful reduction in transfusion burden. Importantly, this anti-hemolytic effect is translating to clear clinical benefits, including improvements for hemoglobin responders across measures of sickle cell pain crises, pain, sleep, and physical function compared with non-responders. Together, these data reinforce the potential for mitapivat to improve the relentless physical toll that comes with living with sickle cell disease.”

New RISE UP Phase 3 Trial Results at EHA 2026

Reduction in Transfusion Burden

New analyses from RISE UP show that mitapivat was associated with a clinically meaningful reduction in transfusion burden compared with placebo. Patients in the mitapivat arm had a 41.1% relative reduction in the proportion of patients requiring blood transfusions compared with placebo (23.9% with mitapivat vs. 40.6% with placebo), as well as a 55.9% relative reduction in average red blood cell units transfused per patient compared with placebo (0.70 units with mitapivat vs. 1.59 with placebo). These benefits were observed regardless of whether patients were also taking hydroxyurea. A reduction in transfusion burden in sickle cell disease can reflect decreased dependence on supportive care.

Hemoglobin Responders Post-Hoc Analysis

As previously reported, 40.6% of patients in the mitapivat arm achieved the primary endpoint of hemoglobin response (≥1.0 g/dL increase from baseline in average hemoglobin from Week 24 through Week 52) compared with 2.9% in the placebo arm, a statistically significant improvement (2-sided p<0.0001). Among these hemoglobin responders, the mean change from baseline in average hemoglobin concentration from Week 24 through Week 52 was 1.6 g/dL.

A post-hoc analysis showed patients in the mitapivat arm who achieved a hemoglobin response also experienced clinically meaningful reductions in pain crises and related hospitalizations, including a 26% reduction in the annualized rate of SCPCs (2.20 for responders vs. 2.98 for non-responders) and 34% fewer related hospitalizations (1.16 for responders vs. 1.76 for non-responders). These patients also had improvements in healthcare utilization, with a 53% reduction in the annualized rate of emergency room visits for SCPCs (1.11 for responders vs. 2.33 for non-responders) and a 37% decrease in the annualized rate of hospitalization days for SCPCs (7.83 for responders vs. 12.34 for non-responders).

Hemoglobin responders in the mitapivat arm also reported greater improvements in patient-reported fatigue scores than non-responders (-5.19 for responders vs. -2.55 for non-responders), as measured by change from baseline in average Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 13a Short Form scores from Week 24 through Week 52. The magnitude of this improvement in hemoglobin responders exceeded the predefined 4.1-point threshold required to be considered clinically meaningful.

In the mitapivat arm, improvements across several additional patient-reported outcomes, including measures of pain, sleep, and physical function, were observed for hemoglobin responders compared with non-responders:

  • PROMIS Pain Intensity 1a: The mean change from baseline was -1.63 points for hemoglobin responders and -0.59 for non-responders, with a least squares mean (LSM) difference of -1.04 (95% confidence interval [CI]: -1.66 to -0.42), favoring hemoglobin responders. The LSM difference is used throughout to represent the model-adjusted difference between hemoglobin responders and non-responders.
  • Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) Pain Impact: The mean change from baseline was 4.09 points for hemoglobin responders and 0.85 for non-responders, with an LSM difference of 3.24 (95% CI: 1.18 to 5.30), favoring hemoglobin responders.
  • PROMIS Physical Functioning 8a: The mean change from baseline was 5.30 points for hemoglobin responders and 1.79 for non-responders, with an LSM difference of 3.51 (95% CI: 0.62 to 6.39), favoring hemoglobin responders.
  • ASCQ-Me Sleep Impact: The mean change from baseline was 2.39 points for hemoglobin responders and -0.48 for non-responders, with an LSM difference of 2.87 (95% CI: 0.22 to 5.53), favoring hemoglobin responders.
  • EuroQol-5 Dimension Visual Analog Scale (EQ-5D VAS): The mean change from baseline was 3.27 points for hemoglobin responders and -6.77 for non-responders, with an LSM difference of 10.04 (95% CI: 2.41 to 17.66), favoring hemoglobin responders.

“Having the opportunity to present these comprehensive results during the EHA 2026 Plenary Session highlights the strength of the RISE UP Phase 3 data – the first pivotal trial to validate pyruvate kinase activation as a new treatment approach in sickle cell disease,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “Building on over a decade of clinical experience with mitapivat across several hemolytic anemias, these results reinforce both its consistent benefits and its well-established safety profile, which is supported by over 1,300 patient-years of data. Taken together, mitapivat represents a differentiated anti-hemolytic approach that can provide meaningful clinical benefits for patients with sickle cell disease – an underserved population in desperate need of innovative therapies.”

Safety Profile

Mitapivat was well-tolerated, with a safety profile consistent with previous trials of mitapivat in sickle cell disease. The percentage of patients with any reported treatment-emergent adverse events was similar between the mitapivat and placebo arms (97.1% vs. 98.6%, respectively). No treatment-related deaths occurred during the trial.

EHA 2026 Investor Event

Agios will host a conference call and live webcast during EHA 2026 today, June 13, 2026, at 9:00 a.m. ET (3:00 p.m. CEST). The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

About Sickle Cell Disease

Sickle cell disease is a rare, inherited blood disorder caused by the production of abnormal hemoglobin that disrupts the ability of red blood cells to carry oxygen throughout the body. As a result, red blood cells become rigid and sickle-shaped, causing deformation of red blood cell membranes and the premature death of the cells. These effects lead to chronic hemolytic anemia, vaso-occlusion, and a cascade of severe and life-threatening complications, including long-term damage to the lungs, kidneys, and cardiovascular system. Due to its physical toll, sickle cell disease imposes a profound burden on patients and their families, marked by increased healthcare needs and early mortality.

About Mitapivat in Sickle Cell Disease

Mitapivat, an oral pyruvate kinase (PK) activator, is designed to enhance the process by which red blood cells produce energy. This approach has the potential to improve red blood cell health by increasing ATP levels to support increased energy demands and lowering levels of a molecule called 2,3-diphosphoglycerate (2,3-DPG). In sickle cell disease, increased stress on red blood cells results in elevated levels of 2,3-DPG, which raises the likelihood that red blood cells develop the abnormal “sickle” shape that triggers vaso-occlusive crises. In May 2026, Agios announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of mitapivat in sickle cell disease.

About the RISE UP Phase 3 Trial

The global RISE UP Phase 3 trial (NCT05031780) is evaluating the efficacy and safety of mitapivat in patients with sickle cell disease aged 16 years or older, representative of the global population. The trial included a 52-week, double-blind, randomized, placebo-controlled period, in which 207 participants were randomized 2:1 to receive oral mitapivat (100 mg) twice daily (n=138) or matched-placebo (n=69).

To comprehensively evaluate objective measures of hemolysis alongside other clinically relevant outcomes in sickle cell disease, the double-blind period of RISE UP included two primary endpoints – hemoglobin response and annualized rate of sickle cell pain crises – as well as five key secondary endpoints:

  • Average change from baseline in hemoglobin concentration from Week 24 through Week 52
  • Average change from baseline in indirect bilirubin from Week 24 through Week 52
  • Average change from baseline in Patient Reported Outcome Measurement Information System Fatigue 13a (PROMIS Fatigue) Short Form scores from Week 24 through Week 52
  • Annualized frequency of hospitalizations for sickle cell pain crises
  • Average change from baseline in percent reticulocyte levels from Week 24 through Week 52

Of the 176 participants who completed the double-blind period of the trial, nearly all (n=174/176) opted to transition into a 216-week open-label extension (OLE) period, during which all participants receive mitapivat.

About Agios: Fueled by Connections to Transform Rare Diseases™

At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X.

Available Information about Agios

To achieve broad dissemination, Agios may disclose information to the public through a variety of disclosure channels including press releases, SEC filings, and public conference calls and webcasts. Some of the information distributed through these disclosure channels may be considered material information. Investors and others should note that Agios plans to use its website (www.agios.com) as a distribution channel to announce and give notice of Agios’ upcoming events and presentations (including, but not limited to, presentations at medical or healthcare conferences). Such information, which may be deemed material, will be available on the Investors section of the company’s website under the “Events & Presentations” tab. In addition, you may sign up to automatically receive email alerts about Agios’ upcoming events and presentations (“Calendar Alerts”) by visiting the “Email Alerts” option under the “IR Resources” tab of the Investors section of the company’s website and submitting your email address.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of mitapivat and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios’ ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236 or cevidoplenib, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption “Risk Factors” included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:
Investor Contact
Morgan Sanford, Vice President, Investor Relations
Agios Pharmaceuticals
[email protected]

Media Contact

Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
[email protected]