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Data show investigational atumelnant drove sustained androgen reductions while enabling lowering of glucocorticoid supplementation to physiologically normal levels in adults with classic CAH

New results from the Phase 1b/2a ACTH-dependent Cushing’s syndrome trial also presented, showing atumelnant rapidly lowered early morning cortisol and normalized urinary free cortisol levels even at lower dose

SAN DIEGO, June 14, 2026 (GLOBE NEWSWIRE) — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) presented data today from the open-label, Phase 2 congenital adrenal hyperplasia (CAH) adult study of investigational atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate being developed for the treatment of classic CAH and ACTH-dependent Cushing’s syndrome. The findings were included in an oral presentation titled “Once Daily Atumelnant (CRN04894) Enables Lowering of Glucocorticoid Doses with Sustained Androgen Reduction in Adults with Congenital Adrenal Hyperplasia” at Endocrine Society’s Annual Meeting, ENDO 2026.

“Atumelnant is designed to block the effect of excess ACTH, the fundamental driver of symptoms and complications of CAH and ADCS,” said Dr. Alan Krasner, M.D., Chief Endocrinologist, Crinetics. “Based on promising results from phase 2 clinical trials presented today, we are advancing atumelnant into late phase clinical development. The data suggest atumelnant could represent a uniquely effective and simple to use oral therapy for many patients who need new options.”

“It’s exciting to see that glucocorticoid dose reduction did not impact the atumelnant-induced decline in androstenedione in adults with classic CAH who participated in this Phase 2 trial,” said Dr. Umasuthan Srirangalingam, Consultant Physician in Endocrinology and Diabetes at University College London Hospitals NHS Foundation Trust and TouCAHn Investigator. “We are looking forward to learning more about the full potential of atumelnant in the treatment of CAH from adult and pediatric Phase 3 trials that are already underway.”

At ENDO 2026, findings from Cohort 4 of the Phase 2 CAH trial were presented for the first time, including the percent change from baseline in morning serum A4, 11-OHA4, and 11-KT with GC reduction. Participants in Cohort 4 received dosing of 80 mg once daily in the morning. Beginning at week 2 of treatment, each participant’s previous GC dose was reduced stepwise by 5-10 mg HC equivalents, independent of A4 measurement, to target <11 mg/m²/day HC equivalents.

Phase 2 CAH Cohort 4 Results

• At week 12, the mean percentage change from baseline in A4 morning serum levels in Cohort 4 was -67%.
• Seven out of eight participants (88%) who completed 12 weeks of treatment achieved a physiologic daily dose of GC.
• Reductions in pre-GC serum 11-OHA4 and 11-KT were rapid and sustained, with mean change from baseline of -64% and -56% at week 12, respectively.
• Morning dosing of atumelnant resulted in similar androgen reductions as seen in previous cohorts with evening administration.

Atumelnant was generally well tolerated with no treatment-related severe or serious adverse events to date, irrespective of disease severity or dose level.

Initial findings from the adult Phase 2 trial in CAH, including A4 reduction levels compared to baseline for cohorts 1-3, in which participants did not change previous GC doses, were presented at ENDO 2025.

Topline results from Cohort 4 were announced in January 2026.

Previously Reported A4 Reductions for Cohorts 1-3 (no GC reduction)

New Phase 1b/2a ADCS Trial Results

Data presented at ENDO 2026 include findings from a cohort dosed with atumelnant 40 mg once daily (n=6). Findings include:

• Atumelnant rapidly lowered early morning serum cortisol in all participants.
• Atumelnant also rapidly lowered UFC. At the end of the 10-day dosing period, UFC remained ≤ upper limit of normal (ULN) in 3/6 participants.
• Most AEs were mild to moderate and consistent with symptoms of adrenal insufficiency. Most improved with initiation of GC replacement.

Atumelnant ENDO 2026 presentations can be found at: https://crinetics.com/news-events/endo-2026/

About Atumelnant

Atumelnant, Crinetics’ second investigational compound, is the first once-daily, oral adrenocorticotropic hormone (ACTH) receptor antagonist that acts selectively at the melanocortin type 2 receptor (MC2R) on the adrenal gland. Diseases associated with excess ACTH can have significant impact on physical and mental health. Atumelnant has exhibited strong binding affinity for MC2R in preclinical models and has demonstrated suppression of adrenally derived glucocorticoids and androgens that are under the control of ACTH. Data from a 12-week Phase 2 study demonstrated compelling treatment benefits of atumelnant, evidenced by the rapid, substantial and sustained statistically significant reductions in key CAH disease related biomarkers, including androstenedione and 17-hydroxyprogesterone, in a diverse population. Atumelnant is in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, with the Phase 3 CALM-CAH trial and a Phase 1/2b trial in ADCS currently enrolling patients.

About the Phase 2 TouCAHn Trial (CAH)

The TouCAHn trial is an open-label, global, Phase 2 study designed to evaluate the efficacy, safety, and pharmacokinetics of atumelnant when administered for 12 weeks in people with classic CAH (21-hydroxylase deficiency). A total of 38 participants were enrolled, with a median A4 of 980.8 (range=116-2755) ng/dL were enrolled in four cohorts: (40 mg, n=11; 80 mg, n=11; 120 mg, n=6; 80 mg morning dosing with GC reduction, n=10).

Primary endpoints included change from baseline in morning serum androstenedione (A4) levels and incidence of treatment-emergent adverse events. Percent change-from-baseline in GC daily dose was an exploratory endpoint for Cohort 4.

About the Phase 1b/2a Study in ACTH-dependent Cushing’s Syndrome

The Phase 1b/2a, is the first-in-disease, open-label, multiple-ascending dose exploratory study to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamic biomarker responses associated with atumelnant over a 10-day inpatient treatment period in participants with ACTH-dependent Cushing’s syndrome.

The study is being conducted in collaboration with the National Institutes of Health and led by Dr. Lynnette Nieman. Participants received oral atumelnant once daily for 10 days, followed by monitoring during four wash-out days.

About Crinetics Pharmaceuticals 

Crinetics Pharmaceuticals is a global pharmaceutical company committed to transforming the treatment of endocrine diseases and endocrine-related tumors through science rooted in patient needs. Crinetics is focused on discovering, developing, and commercializing novel therapies, with a core expertise in targeting G-protein coupled receptors (GPCRs) with small molecules that have specifically tailored pharmacology and properties.

Crinetics’ first commercial product, PALSONIFY™ (paltusotine), is the first once-daily, oral treatment approved by the U.S. FDA and EMA for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. Paltusotine is also in clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics’ deep pipeline of 10+ disclosed programs includes late-stage investigational candidate atumelnant, which is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, and CRN09682, a nonpeptide drug conjugate candidate that is being developed to treat somatostatin receptor 2 (SST2) expressing neuroendocrine tumors and other SST2 expressing solid tumors. Additional discovery programs are focused on a variety of endocrine targets such as thyroid stimulating hormone (TSH), parathyroid hormone (PTH), somatostatin receptor 3 (SST3), growth hormone (GH), glucagon-like peptide-1 (GLP-1), and glucose-dependent insulinotropic polypeptide (GIP), as well as GPCR-targeted oncology indications.

Forward-Looking Statements 

This press release contains 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. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the plans and timelines for the clinical development of atumelnant for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome and paltusotine for the treatment of carcinoid syndrome; or the potential for our development candidates to transition to clinical development. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, we may not be able to obtain, maintain and enforce our patents and other intellectual property rights, and it may be prohibitively difficult or costly to protect such rights; geopolitical events may disrupt Crinetics’ business and that of the third parties on which it depends, including delaying or otherwise disrupting clinical studies and preclinical studies, manufacturing and supply chain, or impairing employee productivity; unexpected adverse side effects, complications and/or drug interactions or inadequate efficacy of the Company’s product candidates that may limit their development, regulatory approval and/or commercialization; the Company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; regulatory developments or political changes, including policies related to pricing and pharmaceutical drug reimbursement, in the United States and foreign countries; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development or be approved for marketing; Crinetics may use its capital resources sooner than expected or our cash burn rate may accelerate; any future impacts to our business resulting from geopolitical developments outside our control; and the other risks and uncertainties described in the Company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2025. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
 

Media: 

Natalie Badillo 
Head of Corporate Communications 
[email protected] 
(858) 345-6075 

Investors: 

Gyathri Diwakar 
Head of Investor Relations 
[email protected] 
(858) 345-6340 

Data from up to two years of treatment in PATHFNDR-1 and PATHFNDR-2 open-label extension studies show PALSONIFY maintained lower IGF-1 levels and stable symptoms

Treatment also resulted in stable or reduced pituitary tumor volumes for up to 48 weeks

Additional ENDO highlights include long-term ACROBAT data demonstrating PALSONIFY and cabergoline combination improves biochemical control of acromegaly

SAN DIEGO, June 14, 2026 (GLOBE NEWSWIRE) — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced new long-term data from its clinical development program evaluating novel PALSONIFY^TM (paltusotine) in acromegaly during an oral presentation at the Endocrine Society’s Annual Meeting, ENDO 2026. Notably, pooled data from the open-label extension (OLE) trials of PATHFNDR-1 and PATHFNDR-2 show that after two years of treatment, PALSONIFY was effective and well-tolerated in patients who were switched from standard-of-care monthly injectable somatostatin receptor ligands (SRLs) and those who were medically untreated, respectively, when oral, once-daily PALSONIFY was initiated.

“To assess acromegaly disease control while on medication, endocrinologists carefully monitor control of IGF-1 levels, control of acromegaly symptoms, and stabilization of pituitary tumors,” said Dr. Alan Krasner, M.D., Chief Endocrinologist, Crinetics. “At this year’s ENDO meeting, long-term safety and efficacy data from the PATHFNDR OLE trials will be presented. These studies indicate that Palsonify is well tolerated and maintains control of all three aspects of disease control with long-term follow-up. Since its launch late last year, we are learning that Palsonify is already making a meaningful difference in the lives of many people with acromegaly, and we hope these data will be helpful for patients and for their health care providers.”

Pooled OLE Efficacy and Safety Results

PATHFNDR-1 Data

The PATHFNDR-1 Phase 3 trial enrolled adults with acromegaly who were biochemically controlled on monthly injectable SRLs. Following a 36-week randomized, placebo-controlled period, 53 of 57 participants (93%) entered the ongoing single-arm open-label extension (OLE) trial.

Baseline mean IGF-1 levels for OLE participants (n=53) was 0.91x Upper Limit of Normal (ULN). These levels remained stable at both 48 weeks (n=50) and 96 weeks (n=47) of the study: 0.82x ULN and 0.81, respectively. Symptoms associated with acromegaly, as measured by the Acromegaly Symptom Diary (ASD), remained stable from baseline at assessed timepoints. Additionally, pituitary tumor volumes were reported as stable in all patients at week 48, relative to OLE baseline.

PATHFNDR-2 Data

The PATHFNDR-2 trial evaluated once-daily oral PALSONIFY in adults with biochemically uncontrolled acromegaly (baseline IGF-1 > 1.3 × ULN). After a 24-week randomized controlled (RC period, 103 of 106 completers (97.2%) entered the ongoing OLE, along with 11 additional patients who were eligible for the RC phase but enrolled directly into the OLE.

Baseline mean IGF-1 levels for OLE participants (n=114) was 1.64×ULN. These levels decreased from baseline at both 48 weeks (n=98) and 72 weeks (n=78) of the study: 1.06×ULN and 0.96×ULN, respectively.

Relative to OLE baseline, pituitary tumor volume was reduced by >20% in 7 of 83 PATHFNDR-2 patients with available MRI scans at OLE Week 24. Tumor volume was reported as stable in the other 76 participants.

In both OLEs, median ASD scores were stable at the timepoints assessed. Symptoms associated with acromegaly, as measured by the Acromegaly Symptom Diary (ASD), remained stable from baseline at assessed timepoints.

No new safety signals were found. In the pooled OLE population (n=167), the most common adverse events (incidence>10%) were diarrhea (15.6%), arthralgia (11.4%), headache (11.4%), and urinary tract infection (10.2%). Four patients (2.4%) discontinued from an OLE due to adverse events as of this analysis.

These results were included in an oral presentation at ENDO 2026 titled “Efficacy and Safety of Once-Daily Oral Paltusotine in Patients with Acromegaly: Up to 2 Years in the PATHFNDR-1 and PATHFNDR-2 Open-Label Extension Studies.”

Additionally, an analysis was presented at ENDO 2026 that evaluated the safety and efficacy of PALSONIFY in combination with oral cabergoline in patients with acromegaly who have been followed for up to four years in ACROBAT Advance, an ongoing, single-arm, open-label extension phase 2 study. IGF-I levels on paltusotine monotherapy were similar to parent study baseline values (on injected SRL), but for those in whom IGF-1 had not yet normalized, it further improved when oral cabergoline was added. Combination therapy was well tolerated.

Crinetics’ ENDO 2026 presentations can be found at: https://crinetics.com/news-events/endo-2026/

PALSONIFY™ (paltusotine) INDICATION:

PALSONIFY is a somatostatin receptor agonist indicated for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS:

• Cholelithiasis and Its Complications: Cholelithiasis, including related complications such as acute cholecystitis and pancreatitis, have been reported. Monitor patients periodically. Discontinue PALSONIFY if complications of cholelithiasis occur and treat appropriately.
• Hyperglycemia and Hypoglycemia: Hyperglycemia, diabetes mellitus, or hypoglycemia, may occur. Monitor blood glucose levels when PALSONIFY treatment is initiated or when dosage is altered. Adjust antidiabetic treatment accordingly.
• Cardiovascular Abnormalities: Cardiac conduction abnormalities and other ECG changes such as PR interval prolongation, bradycardia, sinus arrest, and atrioventricular block may occur in patients with acromegaly and were reported in PALSONIFY clinical trials. Dosage adjustments of concomitant drugs that have bradycardic effects may be necessary.
• Thyroid Function Abnormalities: Somatostatin analogs may suppress the secretion of thyroid-stimulating hormone, which may result in hypothyroidism. Periodic assessment of thyroid function is recommended.
• Steatorrhea and Malabsorption of Dietary Fats: Somatostatin analog treatment may result in malabsorption of dietary fats and subsequent symptoms of steatorrhea, loose stools, abdominal bloating, and weight loss. If new or worsening symptoms are reported with PALSONIFY, evaluate patients for potential pancreatic exocrine insufficiency and manage accordingly.
• Vitamin B12 Deficiency: Vitamin B12 deficiency may occur. Monitor vitamin B12 levels, if clinically indicated.

ADVERSE REACTIONS:

Most common adverse reactions (>5%) are diarrhea, abdominal pain, nausea, decreased appetite, sinus bradycardia, hyperglycemia, palpitations, and gastroenteritis.

DRUG INTERACTIONS:

• Strong or Moderate CYP3A4 Inducers: may decrease PALSONIFY exposure. May require an increased dosage of PALSONIFY.
• Proton Pump Inhibitors: may decrease PALSONIFY exposure. May require an increased dosage of PALSONIFY. Avoid concomitant use of proton pump inhibitors in patients who are already on PALSONIFY 60 mg.
• Cyclosporine: may decrease cyclosporine exposure. May require cyclosporine dosage adjustment when used with PALSONIFY; follow therapeutic monitoring recommendations.

Please report adverse events to Crinetics Pharmaceuticals at 1-833-CRN-INFO (1-833-276-4636) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.ww.fda.gov/medwatch.

Please see 

Full Prescribing Information

 including 

Patient Information

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About
PALSONIFY™ (Paltusotine) 

PALSONIFY, a selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist, is the first and only once-daily, oral therapy approved for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. In Phase 3 studies, once-daily, oral PALSONIFY maintained IGF-1 levels and symptom control in patients with acromegaly who were switched from monthly injectable medications (PATHFNDR-1) and rapidly decreased IGF-1 levels and symptom burden in medically untreated acromegaly patients (PATHFNDR-2). IGF-1 is the primary biomarker endocrinologists use to manage acromegaly patients. Paltusotine is also in Phase 3 clinical development for carcinoid syndrome associated with neuroendocrine tumors (CAREFNDR). Results from a Phase 2 study in carcinoid syndrome demonstrated rapid and sustained reductions in flushing episodes and bowel movement frequency, which are the most common symptoms of carcinoid syndrome.

PALSONIFY is approved in the U.S. for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. It is also approved for use in the EU for the medical treatment of adult patients with acromegaly.

About Crinetics Pharmaceuticals 
Crinetics Pharmaceuticals is a global pharmaceutical company committed to transforming the treatment of endocrine diseases and endocrine-related tumors through science rooted in patient needs. Crinetics is focused on discovering, developing, and commercializing novel therapies, with a core expertise in targeting G-protein coupled receptors (GPCRs) with small molecules that have specifically tailored pharmacology and properties.

Crinetics’ first commercial product, PALSONIFY™ (paltusotine), is the first once-daily, oral treatment approved by the U.S. FDA and EMA for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. Paltusotine is also in clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics’ deep pipeline of 10+ disclosed programs includes late-stage investigational candidate atumelnant, which is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, and CRN09682, a nonpeptide drug conjugate candidate that is being developed to treat somatostatin receptor 2 (SST2) expressing neuroendocrine tumors and other SST2 expressing solid tumors. Additional discovery programs are focused on a variety of endocrine targets such as thyroid stimulating hormone (TSH), parathyroid hormone (PTH), somatostatin receptor 3 (SST3), growth hormone (GH), glucagon-like peptide-1 (GLP-1), and glucose-dependent insulinotropic polypeptide (GIP), as well as GPCR-targeted oncology indications.

Forward-Looking Statements 
This press release contains 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. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the plans and timelines for the clinical development of atumelnant for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome and PALSONIFY for carcinoid syndrome, including the therapeutic potential and clinical benefits or safety profile thereof; and the therapeutic potential for our development candidates. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, initial or topline data that we report may change following completion or a more comprehensive review of the data related to the clinical studies and such data may not accurately reflect the complete results of a clinical study, and the FDA and other regulatory authorities may not agree with our interpretation of such results; geopolitical events may disrupt Crinetics’ business and that of the third parties on which it depends, including delaying or otherwise disrupting its clinical studies and preclinical studies, manufacturing and supply chain, or impairing employee productivity; the success of Crinetics’ clinical studies and nonclinical studies; regulatory developments in the United States and foreign countries; clinical studies and preclinical studies may not proceed at the time or in the manner expected, or at all; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development or be approved for marketing; and the other risks and uncertainties described in the Company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2025 and quarterly report on Form 10-Q for the quarter ended March 31, 2026. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contact:  

Media:  
Natalie Badillo  
Head of Corporate Communications  
[email protected]  
(858) 345-6075   

Investors:  
Gayathri Diwakar  
Head of Investor Relations  
[email protected]  
(858) 345-6340  

• Treatment with barzolvolimab resulted in rapid, significant, and durable improvements in angioedema in patients with moderate to severe CSU
• Seven months after the completion of dosing (Week 76), up to 64% of patients treated with barzolvolimab who had angioedema at baseline remained angioedema-free
• Barzolvolimab has potential to shift treatment goals from symptom control to disease modification
• Results continue to support ongoing Phase 3 trials of barzolvolimab in CSU; topline data expected in Q4 2026

HAMPTON, N.J., June 14, 2026 (GLOBE NEWSWIRE) — Celldex (NASDAQ:CLDX) announced today the presentation of long-term results from the Phase 2 study of barzolvolimab in a flash talk session at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting in Istanbul, Türkiye. The data presented demonstrated that barzolvolimab treatment results in rapid, significant, and durable improvements in angioedema in patients with chronic spontaneous urticaria (CSU) refractory to antihistamines. These results were sustained off-treatment, seven months after completion of barzolvolimab dosing (Week 76). The data continue to demonstrate barzolvolimab’s potential to shift the goal of CSU treatment from symptom control to disease modification and further support the ongoing Phase 3 trials of barzolvolimab in CSU.

“Angioedema plays a major role in the physical and mental health of the majority of people living with CSU, causing extremely painful swelling and disfigurement that dramatically impacts quality of life,” said Diane C. Young, MD, Senior Vice President and Chief Medical Officer of Celldex Therapeutics. “Barzolvolimab has consistently shown profound, lasting results including high rates of complete response, defined as complete absence of itch and hives, and dramatic improvements in quality of life and angioedema control through 52 weeks of therapy and now seven months after the last dose, demonstrating its potential for disease modification and the ability to change how CSU patients live their lives.”

Angioedema occurs in 55% of people with CSU¹ and patients report a mean of 7.7 angioedema episodes annually.² Patients with CSU-related angioedema report significantly worse physical and mental health outcomes, lower health related quality of life, a higher percentage of anxiety and depression, along with significantly increased emergency room visits and hospitalizations compared to patients without angioedema in the United States.^3,4 Similarly, these patients report significantly higher work and activity impairment than those without angioedema.⁵ Both patients and physicians report being free of angioedema as an important treatment goal in CSU.⁶

As previously reported, data from the Phase 2b trial showed that treatment with barzolvolimab resulted in rapid, significant, and durable improvements in angioedema. Relief from angioedema symptoms began as early as Week 1 and deepened over 52 weeks of treatment. Furthermore, newly presented data show that barzolvolimab treatment led to robust and sustained reductions in angioedema symptoms at Week 76, demonstrating prolonged off-treatment benefits. Up to 64% of patients who had angioedema at baseline were angioedema-free 7 months after the last dose.

Two Phase 3 trials of barzolvolimab in CSU are ongoing and enrollment is complete. 1,939 patients were enrolled, the largest program conducted in antihistamine-refractory CSU, including patients with advanced therapy experienced/refractory CSU. The studies included 43 countries across 500 sites. Topline data are anticipated in Q4 2026, supporting a planned BLA submission in 2027.

¹Kolkhir P, et al. Nat Rev Dis Primers. 2022 Sep 15;8(1):61 ²Weller, et al. Dermatol Ther, 2025. ³Balp M, et al. Burden of angioedema in patients with chronic spontaneous urticaria in EU5 and US, EADV Congress 2023. ⁴Balp M, et al. Characterization of chronic spontaneous urticaria among patients in EU5, US and Japan. EADV Congress 2023. ⁵Soong W, et al. World Allergy Organ J. 2025. ⁶Bernstein J, et al. Frequency of angioedema in chronic spontaneous urticaria patients: Report from the Urticaria Voices study, GA²LEN Global Urticaria Forum 2024.

About Barzolvolimab

Barzolvolimab is a humanized monoclonal antibody with a novel mechanism of action that targets mast cells by binding with high specificity to a unique part of the KIT receptor and potently inhibiting its activity. The KIT receptor is abundantly expressed by mast cells and critical for their function and survival. Mast cells are drivers of inflammatory responses such as hypersensitivity and allergic reactions and, in certain inflammatory diseases, such as chronic urticarias, mast cell activation plays a central role in the onset and progression of the disease. Based on data from robust, randomized, placebo controlled Phase 2 studies, barzolvolimab has significant potential as a first-in-class and best-in-disease treatment option for patients with chronic spontaneous urticaria (CSU), cold urticaria (ColdU) and symptomatic dermographism (SD). Barzolvolimab is currently being studied in Phase 3 studies in CSU and ColdU/SD and Phase 2 studies in prurigo nodularis (PN) and atopic dermatitis (AD), with additional indications planned for the future.

About the Phase 2 CSU Study

The randomized, double-blind, placebo-controlled, parallel group Phase 2 study evaluated the efficacy and safety profile of multiple dose regimens of barzolvolimab in patients with CSU who remain symptomatic despite antihistamine therapy, to determine the optimal dosing strategy. 208 patients were randomly assigned on a 1:1:1:1 ratio to receive subcutaneous injections of barzolvolimab at 75 mg every 4 weeks, 150 mg every 4 weeks, 300 mg every 8 weeks or placebo during a 16-week placebo-controlled treatment period. After 16 weeks, patients then entered a 36-week active treatment period, in which patients receiving placebo or the 75 mg dose were randomized to receive barzolvolimab 150 mg every 4 weeks or 300 mg every 8 weeks; patients already randomized to the 150 mg and 300 mg treatment arms remained on the same regimen as during the placebo-controlled treatment period. After 52 weeks, patients entered a follow-up period for an additional 24 weeks. Barzolvolimab achieved the primary efficacy endpoint of the study—a statistically significant mean change from baseline to Week 12 in UAS7 (weekly urticaria activity score) compared to placebo at all dose levels. For additional information on this trial (NCT05368285), please visit www.clinicaltrials.gov.

About Celldex

Celldex is pioneering new horizons in immunology to deliver life-changing therapies. We are relentless in our pursuit of novel antibody-based treatments that engage the human immune system and directly affect critical pathways to improve the lives of patients with allergic, inflammatory and autoimmune disorders. Visit www.celldex.com.

Forward Looking Statement

This release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, our ability to successfully complete research and further development and commercialization of Company drug candidates, including barzolvolimab (also referred to as CDX-0159) and CDX-622, in current or future indications; the uncertainties inherent in clinical testing and accruing patients for clinical trials; our limited experience in bringing programs through Phase 3 clinical trials; our ability to manage and successfully complete multiple clinical trials and the research and development efforts for our multiple products at varying stages of development; the availability, cost, delivery and quality of clinical materials produced by our own manufacturing facility or supplied by contract manufacturers, who may be our sole source of supply; the timing, cost and uncertainty of obtaining regulatory approvals; the failure of the market for the Company’s programs to continue to develop; our ability to protect the Company’s intellectual property; the loss of any executive officers or key personnel or consultants; competition; changes in the regulatory landscape or the imposition of regulations that affect the Company’s products; our ability to continue to obtain capital to meet our long-term liquidity needs on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials that we have initiated or plan to initiate; and other factors listed under “Risk Factors“ in our annual report on Form 10-K and quarterly reports on Form 10-Q.

All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise.

Company Contacts

Sarah Cavanaugh
Senior Vice President, Corporate Affairs & Administration
(508) 864-8337
[email protected]

Elizabeth Higgins
Executive Director, Investor Relations & Corporate Communications
(857) 404-2088
[email protected]

– Lorundrostat was associated with significant reductions in heart failure risk biomarkers in a proteomic analysis of data from participants with uncontrolled hypertension –

– Coordinated reductions in biomarkers of fibrosis and heart failure suggest lorundrostat may favorably modulate the physiological processes that drive heart failure –

RADNOR, Pa., June 14, 2026 (GLOBE NEWSWIRE) — Mineralys Therapeutics, Inc. (Nasdaq: MLYS), a biopharmaceutical company focused on developing medicines to target hypertension and aldosterone-related adverse outcomes in comorbid conditions such as chronic kidney disease (CKD), obstructive sleep apnea (OSA) and other diseases driven by dysregulated aldosterone, today presented new data on the effect of lorundrostat on heart failure (HF) risk biomarkers. This post hoc analysis of circulating proteomic data from participants enrolled in the Company’s Launch-HTN and Advance-HTN trials was presented in a late-breaking poster presentation at ENDO 2026, the Endocrine Society’s annual meeting in Chicago, Illinois.

“People with uncontrolled hypertension are at particular risk of heart failure, a condition where more effective treatments are still neededⁱ,” said Jon Congleton, Chief Executive Officer of Mineralys Therapeutics. “Aldosterone plays a well-established roleⁱⁱ in driving this disease, and these findings suggest that lorundrostat may act on the biological processes that contribute to heart failure, supporting further evaluation of its therapeutic potential in this setting.”

This analysis characterized the systemic pharmacodynamic (PD) effects of lorundrostat and generated hypotheses regarding its potential modulation of pathways implicated in HF pathophysiology by profiling circulating protein biomarkers at baseline and after 12 weeks from 1,004 participants enrolled in the pivotal Phase 3 Launch-HTN and Phase 2b Advance-HTN trials.

The PD analysis confirmed that lorundrostat was associated with significant increases in renin and decreases in angiotensinogen, reflecting target engagement of the renin-angiotensin-aldosterone system (RAAS). Lorundrostat was associated with significant reductions in 6 of 11 recently published candidate causal risk biomarkers of incident HF, including NT-proBNP, consistent with the hypothesis that RAAS inhibition favorably modulates processes involved in HF risk.ⁱⁱⁱ

Compared to placebo, lorundrostat treatment led to coordinated changes in key biomarkers: reductions in markers of scarring and heart failure risk and increases in markers of hemostasis and protease inhibitor activity. These changes occurred together, suggesting a broad, consistent effect on disease pathways—particularly reducing harmful fibrosis—rather than isolated, random shifts.

These results provide biological plausibility and support further evaluation of the therapeutic potential of lorundrostat in heart failure.

Lorundrostat is currently under review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) target date of December 22, 2026.

About Launch-HTN 

The Launch-HTN trial (NCT06153693) was a global, randomized, Phase 3 double-blind, placebo-controlled clinical trial of adults whose blood pressure remained uncontrolled despite being on two to five antihypertensive medications. Participants were assigned to one of three groups: lorundrostat 50 mg once daily; lorundrostat 50 mg once daily with the option to increase to 100 mg at week six based on prespecified criteria; or placebo. The primary endpoint was change from baseline in systolic blood pressure at six weeks versus placebo, measured by automated office blood pressure monitoring.

About Advance-HTN

The Advance-HTN trial (NCT05769608) was a randomized, Phase 2 double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of lorundrostat for the treatment of uncontrolled hypertension or resistant hypertension, when used as an add-on therapy to a standardized background treatment of two or three antihypertensive medications in adult participants. Participants who met screening criteria had their existing hypertension medications discontinued and started on a standard regimen of an angiotensin II receptor blocker (ARB) and a diuretic, if previously on two medications, or a standard regimen of ARB, diuretic and calcium channel blocker if previously on three to five medications. Participants who remained hypertensive despite the standardized regimen were then randomized into three cohorts and treated for 12 weeks: lorundrostat 50 mg once-daily; lorundrostat 50 mg once-daily with the option to increase to 100 mg once-daily at week four based on prespecified criteria; or placebo. The primary endpoint was the change in 24-hour ambulatory systolic blood pressure at week 12 from baseline for active cohorts versus placebo.

About Hypertension

Having sustained, elevated blood pressure (BP) (or hypertension) increases the risk of heart disease, heart attack and stroke, which are leading causes of death in the United States. In 2022, more than 685,000 deaths in the United States included hypertension as a primary or contributing cause. Hypertension and related health issues resulted in an estimated annual economic burden of about $219 billion in the United States in 2019.

Less than 50% of hypertensive patients achieve their BP goal with currently available medications. Dysregulated aldosterone levels are a key factor in driving hypertension in approximately 30% of all hypertensive patients.

About Lorundrostat

Lorundrostat is an investigational proprietary, orally administered, highly selective aldosterone synthase inhibitor being developed for the treatment of uncontrolled hypertension (uHTN) or resistant hypertension (rHTN), as well as related comorbidities, such as chronic kidney disease, obstructive sleep apnea and other diseases driven by dysregulated aldosterone. Lorundrostat was designed to reduce aldosterone levels by inhibiting CYP11B2, the enzyme responsible for its production. Lorundrostat has 374-fold selectivity for aldosterone-synthase inhibition versus cortisol-synthase inhibition in vitro, an observed half-life of 10-12 hours and demonstrated a 40-70% reduction in plasma aldosterone concentration in participants with hypertension.

Mineralys has now completed six late-stage clinical trials of lorundrostat supporting the efficacy and safety profile while also validating aldosterone as an integral therapeutic target in uHTN and rHTN. This includes two pivotal, registrational trials: the Phase 3 Launch-HTN trial and the Phase 2 Advance-HTN trials of lorundrostat, which support the robust, durable and clinically meaningful reductions in systolic blood pressure by lorundrostat. Lorundrostat was well tolerated in both trials with a favorable safety profile.

About Mineralys

Mineralys Therapeutics is a biopharmaceutical company focused on developing medicines to target hypertension and related comorbidities such as chronic kidney disease, obstructive sleep apnea and other diseases driven by dysregulated aldosterone. Its initial product candidate, lorundrostat, is an investigational, proprietary, orally administered, highly selective aldosterone synthase inhibitor. Mineralys is based in Radnor, Pennsylvania, and was founded by Catalys Pacific. For more information, please visit https://mineralystx.com. Follow Mineralys on LinkedIn, Twitter and Bluesky.

Forward-Looking Statements

Mineralys Therapeutics cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to, statements regarding: the potential therapeutic benefits of lorundrostat; the anticipated timing of the U.S. Food and Drug Administration’s (FDA) review of the Company’s accepted New Drug Application (NDA) and any subsequent regulatory approval of lorundrostat; and the planned future clinical development of lorundrostat and the timing thereof. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: topline results that we report are based on a preliminary analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial and such topline data may not accurately reflect the complete results of a clinical trial; any delays in the FDA’s review of our accepted NDA, including as a result of a government shutdown or reductions in agency funding or personnel, the results of our clinical trials, including the Advance-HTN and Launch-HTN trials, may not be deemed sufficient by the FDA to serve as the basis for regulatory approval of lorundrostat; later developments with the FDA may be inconsistent with the feedback from prior meetings, including whether the proposed pivotal program will support registration of lorundrostat following the FDA’s review of our NDA submission; our future performance is dependent entirely on the success of lorundrostat; potential delays in the commencement, enrollment and completion of clinical trials and nonclinical studies; our dependence on third parties in connection with manufacturing, research and clinical and nonclinical testing; unexpected adverse side effects or inadequate efficacy of lorundrostat that may limit its development, regulatory approval and/or commercialization; unfavorable results from clinical trials and nonclinical studies; results of prior clinical trials and studies of lorundrostat are not necessarily predictive of future results; macroeconomic trends and uncertainty with regard to high interest rates, elevated inflation, tariffs and other trade policies, and the potential for a local and/or global economic recession; our ability to maintain undisrupted business operations due to any pandemic or future public health concerns; regulatory developments in the United States and foreign countries; our reliance on our exclusive license with Tanabe Pharma Corporation to provide us with intellectual property rights to develop and commercialize lorundrostat; and other risks described in our filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in our annual report on Form 10-K, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Contact:

Investor Relations

[email protected]

Media Relations

Melyssa Weible
Elixir Health Public Relations
Email: [email protected]

___________________________

<sub>ⁱ Peikert A, et al. Contemporary treatment options in heart failure with preserved ejection fraction. European Heart Journal – Cardiovascular Imaging. 2024;25(11):1517-1524. https://doi.org/10.1093/ehjci/jeae201


ⁱⁱ Stiefel P, et al. Role of the renin-angiotensin system and aldosterone on cardiometabolic syndrome. Int J Hypertens. 2011;2011:685238. doi:10.4061/2011/685238.


ⁱⁱⁱ Shah, A.M., et al. Large scale plasma proteomics identifies novel proteins and protein networks associated with heart failure development. Nat Commun 15, 528 (2024). https://doi.org/10.1038/s41467-023-44680-3</sub>

– Verekitug, administered once every three months, led to clinically meaningful improvements in nasal polyp score (NPS) in approximately 80% of participants –

– Majority of verekitug-treated participants experienced clinically meaningful improvements across key secondary endpoints, including 72% in nasal congestion and 83% in CRSwNP total symptom score –

WALTHAM, Mass., June 14, 2026 (GLOBE NEWSWIRE) — Upstream Bio, Inc. (Nasdaq: UPB), a clinical-stage company developing treatments for inflammatory diseases, with an initial focus on severe respiratory disorders, today presented new responder analyses from the Phase 2 VIBRANT trial of verekitug in participants with chronic rhinosinusitis with nasal polyps (CRSwNP). The findings were presented during an oral session at the European Academy of Allergy and Clinical Immunology (EAACI) 2026 Congress in Istanbul, Turkey.

As previously reported, verekitug administered every three months achieved a placebo-adjusted reduction in NPS of -1.95 (p<0.0001) at Week 24, after adjustment for concomitant rescue therapy with systemic corticosteroids. This improvement is nearly double the 1.0 point reduction generally considered clinically meaningful.

The new post-hoc responder analyses further demonstrated that the significant majority of verekitug-treated participants achieved clinically meaningful improvements in NPS, the primary endpoint, and across key secondary endpoints in the Phase 2 VIBRANT trial.

“These data underscore the depth and consistency of verekitug’s clinical benefit, with significant improvements in nasal polyp burden, congestion, sense of smell and other key symptoms observed in the overwhelming majority of participants with inadequately controlled, severe CRSwNP,” said Aaron Deykin, Chief Medical Officer and Head of Research & Development at Upstream Bio. “The compelling new analyses add to the increasing and comprehensive body of evidence supporting verekitug’s potential, as the only known antibody targeting the TSLP receptor in clinical development, to deliver differentiated and durable efficacy among biologic therapies for serious respiratory diseases. Together with the convenience of quarterly dosing, we believe this profile could meaningfully advance the standard of biologic treatment. We are excited to be rapidly progressing toward the initiation of Phase 3 trials in both CRSwNP and severe asthma in the first quarter of 2027.”

“For people living with CRSwNP and the physicians who help manage their disease, the goals of treatment extend beyond reducing disease burden to achieving meaningful improvements in nasal congestion, breathing, and sense of smell,” said Joaquim Mullol, MD, PhD, Professor of Research and Head of the Laboratory of Clinical and Experimental Respiratory Immunoallergy at the August Pi Sunyer Biomedical Research Institute, Barcelona, Spain, and a VIBRANT trial investigator. “These findings demonstrate that most participants in the VIBRANT trial achieved clinically meaningful improvements across these important measures. The results support every three-month administration of verekitug as a potentially effective treatment option for patients whose CRSwNP remain inadequately controlled despite standard intranasal corticosteroid therapy.”

About the VIBRANT Trial Responder Analyses

VIBRANT (NCT06164704) was a Phase 2, global, randomized, double-blind, placebo-controlled, parallel group clinical trial that evaluated the efficacy and safety of verekitug over 24 weeks in 81 adults with severe, inadequately controlled CRSwNP, despite treatment with standard-of-care intranasal steroids. The VIBRANT trial demonstrated the efficacy of verekitug administered every 12 weeks in participants with uncontrolled, severe CRSwNP, in improving NPS, sinonasal symptoms, and sinus disease. The new responder analyses evaluated the proportion of participants who achieved predefined thresholds for clinically meaningful improvements across efficacy endpoints at Week 24.

Key findings included:

• Nasal Polyp Score (Primary endpoint): 79% of verekitug-treated participants, or approximately four in five treated, achieved clinically meaningful improvements in NPS at week 24, compared to 24% in the placebo group (odds ratio [95% CI], 12.06 [4.09–35.53] p<0.0001).
• Nasal Congestion Score: 72% of verekitug-treated participants demonstrated at least one point of improvement in nasal congestion, a key secondary endpoint, compared to 39% in the placebo group (odds ratio [95% CI]: 3.99 [1.50–10.59]; p=0.0046).
• Total Symptom Score: 83% of verekitug-treated participants demonstrated at least four points of improvement in total symptom score, a key secondary endpoint, compared to 37% in the placebo group (odds ratio [95% CI]: 8.57 [2.86–25.67]; p<0.0001).
• Difficulty with sense of smell (Loss of smell): 69% of verekitug-treated participants demonstrated at least one point of improvement in sense of smell, a key secondary endpoint, compared to 21% in the placebo group (odds ratio [95% CI]: 8.52 [2.97–24.45]; p<0.0001).
• Lund-Mackay score
  : 78% of verekitug-treated participants demonstrated at least five points of improvement in the Lund-Mackay score, a key secondary endpoint measuring extent of sinus disease by CT scan, compared to 12% in the placebo group (odds ratio [95% CI]: 25.38 [6.86–93.83]; p<0.0001).

As previously reported, verekitug demonstrated significant and clinically meaningful improvements in key secondary endpoints, including 76% (p=0.03) reduction in the need for surgery or systemic corticosteroids compared with placebo. Verekitug was generally well tolerated, demonstrating a favorable safety profile consistent with previous studies, with no serious adverse events observed during the trial.

Upstream Bio designed the VIBRANT trial using endpoints that, pending interactions with regulatory authorities, could produce data to support submissions for product approval. The Company plans to initiate dosing in Phase 3 registrational trials in both CRSwNP and severe asthma in the first quarter of 2027.

A digital version of the presentation can be found on the Publications section of the Upstream Bio website.

About CRSwNP

CRSwNP is a chronic inflammatory disease of the upper airway, marked by inflammation in the nose and sinuses and the presence of nasal polyps. CRSwNP has four main symptoms: runny nose or postnasal drip, nasal congestion, facial pressure and/or pain, and loss of smell and/or taste. Despite available treatments such as corticosteroids, surgery and, more recently, biologics, quality-of-life studies and post-surgical recurrence rates clearly show that many people with CRSwNP have uncontrolled symptoms that impact their daily life and that current treatments are not meeting their needs. It is estimated that CRSwNP affects up to 4% of the general population, of whom 40% have uncontrolled disease.

Nasal polyps are associated with significant disease burden and debilitating symptoms. It is estimated that over 40% of people with severe asthma also have CRSwNP, and that up to 70% of people with CRSwNP also have asthma, demonstrating a strong association between the two conditions.

About the Phase 2 VIBRANT Trial

The Phase 2 VIBRANT trial (NCT06164704) was a global, randomized, placebo-controlled, parallel group clinical trial, which was designed to assess the efficacy and safety of verekitug in adults with CRSwNP who were receiving concurrent intranasal corticosteroid therapy. Participants received either 100 mg of verekitug or placebo subcutaneously every 12 weeks for 24 weeks. The primary endpoint was change in endoscopic nasal polyp score at Week 24, a primary endpoint that has been used in several registrational trials for other biologic treatments for CRSwNP. Secondary endpoints included: nasal congestion score, sinus opacification, difficulty with sense of smell, total symptom score, percentage of participants requiring systemic corticosteroids or nasal polyp surgery, and time to first such interventions up to Week 24.

About Verekitug

Verekitug is a novel recombinant fully human immunoglobulin G1 (IgG1) monoclonal antibody that binds to the thymic stromal lymphopoietin (TSLP) receptor and inhibits proinflammatory signaling initiated by TSLP. It is the only known antagonist currently in clinical development that targets and inhibits the TSLP receptor.

TSLP is a cytokine that is a key driver of the inflammatory response in major allergic and inflammatory diseases, such as asthma, where disruption of TSLP signaling has been clinically validated as an effective therapeutic strategy. TSLP activation is one of the first events in the inflammatory cascade stimulated by allergens, viruses and other triggers, initiating the activation of downstream targets such as IL-4, IL-5, IL-13, IL-17 and IgE. Because TSLP is a target upstream in the inflammatory cascade, blocking the TSLP receptor presents an opportunity for a single treatment to impact the drivers of multiple pathological inflammatory processes across a broad set of diseases.

Verekitug has advanced into three separate global, placebo-controlled, randomized Phase 2 clinical trials, including the positive VIBRANT trial (NCT06164704) in patients with CRSwNP and the positive VALIANT trial (NCT06196879) in patients with severe asthma. The VENTURE trial (NCT06981078) in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD) is ongoing. Additionally, in May 2025, Upstream Bio initiated the VALOUR trial (NCT06966479), a long-term extension study in eligible participants with severe asthma who completed the VALIANT Phase 2 clinical trial.

About Upstream Bio

Upstream Bio is a clinical-stage biotechnology company developing treatments for inflammatory diseases, with an initial focus on severe respiratory disorders. The Company is developing verekitug, the only known antagonist currently in clinical development that targets and inhibits the receptor for thymic stromal lymphopoietin (TSLP), a cytokine which is a clinically validated driver of inflammatory response positioned upstream of multiple signaling cascades that affect a variety of immune-mediated diseases. The Company has advanced this highly potent monoclonal antibody into separate Phase 2 trials for the treatment of chronic rhinosinusitis with nasal polyps (CRSwNP), severe asthma, and chronic obstructive pulmonary disease (COPD). Upstream Bio’s team is committed to maximizing verekitug’s unique attributes to address the substantial unmet needs for patients underserved by today’s standard of care. To learn more, please visit www.upstreambio.com.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “continue,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “predict,” “project,” “seeks,” “should,” “target,” “will” and variations of these words or similar expressions. Any statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, express or implied statements regarding: the clinical development of verekitug for the treatment of severe asthma, CRSwNP and COPD, including the initiation, timing, progress and results of ongoing and planned clinical trials; expectations regarding the planned regulatory interactions with the U.S. Food and Drug Administration on the data from the Phase 2 VALIANT and VIBRANT trials and the outcomes of any such interactions; expectations regarding the timing of Phase 3 initiation in CRSwNP and severe asthma, including the expected initiation of dosing in the first quarter of 2027; expectations for future discussions with regulatory authorities and the potential of the endpoints of the Company’s clinical trials to produce data that could support submissions for product approval; the potential for verekitug to provide clinical benefit and deliver best-in-class efficacy with quarterly dosing convenience; expectations regarding the differentiation, safety, efficacy, tolerability, and/or extended dosing interval of verekitug; and expectations for the size and growth potential of the market for verekitug and the Company’s ability to serve that market. Any forward-looking statements in this press release are based on the Company’s current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Readers are cautioned that actual results, levels of activity, safety, efficacy, performance or events and circumstances could differ materially from those expressed or implied in the Company’s forward-looking statements due to a variety of risks and uncertainties, which include, without limitation, risks and uncertainties related to: Upstream Bio’s ability to advance verekitug through clinical development, and to obtain regulatory approval of and ultimately commercialize verekitug on the expected timeline, if at all; the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the initiation, timing, progress and results of clinical trials; Upstream Bio’s ability to fund its development activities and achieve development goals; Upstream Bio’s dependence on third parties to conduct clinical trials and manufacture verekitug, and commercialize verekitug, if approved; Upstream Bio’s ability to attract, hire and retain key personnel, and protect its intellectual property; Upstream Bio’s financial condition and need for substantial additional funds in order to complete development activities and commercialize verekitug, if approved; regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; Upstream Bio’s competitors and industry; and other risks and uncertainties described in greater detail under the caption “Risk Factors” in Upstream Bio’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, as well as any subsequent filings with the SEC. Any forward-looking statements represent Upstream Bio’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Upstream Bio explicitly disclaims any obligation or undertaking to update any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based except to the extent required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Investor and Media Contact

Meggan Buckwell
Director, Corporate Communications and Investor Relations
[email protected]