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• 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]

• CDX-622 is a novel, bispecific antibody combining mast cell depletion and TSLP inhibition, two independent pathways contributing to multiple I&I diseases
• CDX-622 targets the soluble form of stem cell factor (SCF), depleting mast cells without measurably impacting other KIT functions
• Rapid, profound, dose-dependent, and durable reductions in serum tryptase observed
• Well-tolerated at all dose levels
• First company to demonstrate that neutralizing soluble SCF can selectively inhibit KIT signaling in mast cells
  

HAMPTON, N.J., June 14, 2026 (GLOBE NEWSWIRE) — Celldex (NASDAQ:CLDX) today presented positive results from the Phase 1 healthy participant study of CDX-622, a novel, bispecific antibody that targets soluble SCF and TSLP, at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting in Istanbul, Türkiye. Data demonstrated that CDX-622 induced rapid, durable, dose-dependent reductions in serum tryptase, indicative of mast cell depletion, and was well-tolerated at all dose levels. Building on Celldex’s leadership in mast cell science, the data also demonstrated that neutralizing soluble stem cell factor (SCF) enables the potential for meaningful mast cell inhibition and depletion without impacting other KIT-dependent functions. CDX-622 is currently being studied in a Phase 1b proof of mechanism study in mild to moderate asthma to assess the impact of dual neutralization of SCF and TSLP.

“Celldex continues to drive groundbreaking science and is the first company to directly demonstrate that neutralizing soluble stem cell factor can selectively inhibit KIT signaling in mast cells, a historically challenging target,” said Tibor Keler, Ph.D., Co-founder, Executive Vice President and Chief Scientific Officer at Celldex. “Importantly, the approach of targeting soluble SCF provides a promising anchor mechanism enabling the development of a robust portfolio of bispecific candidates designed to overcome the heterogeneity inherent in the pathophysiology of many inflammatory diseases. Today’s results from CDX-622 highlight the potential of this approach and we look forward to initiating additional proof of concept studies in multiple indications where both mast cells and TSLP play a pathogenic role, focusing next on allergic rhinitis and food allergy.”

“Mast cell targeting strategies are rapidly emerging as powerful therapeutic approaches in inflammatory diseases. Leveraging our expertise in antibody drug development, and building on our success with barzolvolimab, Celldex is committed to driving innovation across the field of mast cell science to bring our leading science to additional patient populations that could benefit from our medicines,” said  Anthony Marucci, Co-Founder, President and Chief Executive Officer at Celldex. “Barzolvolimab and CDX-622 target mast cells through two distinct, highly synergistic platform approaches. Together, these novel candidates build a powerful foundation for a broad portfolio of therapeutics targeting a wide range of inflammatory diseases where mast cells are implicated. We look forward to sharing more about our growing pipeline in the future.”

Data Summary:

CDX-622 is a uniquely engineered novel bispecific antibody that targets soluble SCF and alarmin thymic stromal lymphopoietin (TSLP), two critical pathways that may contribute to the pathology of several allergic and inflammatory disorders with significant unmet medical need. Combined neutralization of SCF and TSLP with CDX-622 is expected to simultaneously reduce tissue mast cells and inhibit Type 2 inflammatory responses, allowing for a complementary dual mechanism approach that may overcome the heterogeneity inherent in the pathophysiology of many inflammatory disorders. CDX-622 has been engineered to disable effector function (AQQ) and enhance half-life (YTE).

• Rapid, profound, dose-dependent, and durable reductions in serum tryptase were observed, indicative of tissue mast cell inhibition and depletion.
  • Tryptase decreases were comparable to KIT-targeting following multiple doses.
  • Biopsy data were consistent with greater impact on mucosal mast cells than skin mast cells.
• CDX-622 exhibited monoclonal antibody-like pharmacokinetics, with extended half-life and good exposure with subcutaneous administration, consistent with good bioavailability.
  • There was no evidence of immunogenicity at any dose.

• CDX-622 was well-tolerated in all study parts and at all dose levels. There were no dose-limiting toxicities or related serious adverse events.
  • The most commonly reported adverse event across the study was Grade 1 headache.
  • There were no changes in hair or skin pigmentation.
  • There was no meaningful impact on hematologic parameters.

• Additionally, in June, Celldex presented new non-human primate data at the European Mast Cell and Basophil Research Network (EMBRN) that validates the approach of targeting soluble SCF to deplete mast cells. The preclinical study evaluated antibodies either targeting soluble SCF or both soluble and the membrane form of SCF. The data showed that targeting soluble SCF effectively depleted mast cells in a manner similar to targeting membrane SCF, but without measurable effect on spermatogenesis or melanogenesis in non-human primates.
• Results support that combined TSLP neutralization and mast cell depletion with CDX-622 may result in broad efficacy in inflammatory diseases where both pathways play a pathogenic role.

About the Phase 1 Study

The Phase 1 trial was a randomized, double-blind, placebo-controlled, dose escalation study designed to assess the safety, pharmacokinetics, pharmacodynamics and immunogenicity of single ascending doses (Part 1) and multiple ascending doses (Part 2) of CDX-622 in healthy participants. The study also included a single ascending dose of CDX-622 administered subcutaneously (Part 3). 32 participants were enrolled in Part 1 across 4 cohorts (8 participants per cohort; n=6 CDX-622, n=2 placebo) and received single ascending intravenous doses of CDX-622 (0.3, 1.0, 3.0 and 9.0 mg/kg) and were observed over a 12-week period. 24 participants were enrolled in Part 2 across 3 cohorts (8 participants per cohort; n=6 CDX-622, n=2 placebo) and received multiple ascending intravenous doses of CDX-622 (1.0, 3.0, and 9.0 mg/kg at weeks 2, 4, and 6) and were observed for 18 weeks. 24 participants were enrolled in Part 3 across 3 cohorts (8 participants per cohort; n=6 CDX-622, n=2 placebo) and received single ascending subcutaneous doses of CDX-622 (290, 580, and 870 mg) and were observed for 12 weeks. Data from Part 1 were previously presented in October 2025.

About 622: CDX-622 is a bispecific antibody that targets two complementary, clinically validated pathways that drive chronic inflammation, potently neutralizing the alarmin thymic stromal lymphopoietin (TSLP) and depleting mast cells via stem cell factor (SCF) starvation. SCF activation of the KIT receptor is required for mast cell survival and plays a key role in their activation, maturation and tissue recruitment. Combined neutralization of SCF and TSLP with CDX-622 is expected to simultaneously reduce tissue mast cells and inhibit Type 2 inflammatory responses to potentially offer enhanced therapeutic benefit in inflammatory and fibrotic disorders.

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 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 Contact

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

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

•  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.

• 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.

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