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Tag: clinical trial

[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients

Basel, 15 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted.

Celldex Presents Data Demonstrating Profound Long Term Improvement in Angioedema in Barzolvolimab Phase 2 Study in Chronic Spontaneous Urticaria at EAACI 2025

HAMPTON, N.J., June 14, 2025 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (NASDAQ:CLDX) today announced data demonstrating that barzolvolimab profoundly improves angioedema at 52 weeks in the Company’s Phase 2 clinical trial in chronic spontaneous urticaria (CSU). Angioedema, characterized by swelling of the deeper dermal layers of the skin and mucous membranes, is a painful, debilitating symptom of CSU that has significant impact on quality of life. It commonly affects the face (lips and eyelids), hands, feet, and genitalia but can also involve the tongue, uvula, soft palate, and pharynx1.

Jasper Therapeutics Reports Positive Data from 180mg Cohort in SPOTLIGHT Study of Briquilimab in Chronic Inducible Urticaria

11 of 12 participants (92%) enrolled in the 180mg cohort achieved a complete response

ROCKET ALERT: Bragar Eagel & Squire, P.C. Announces that a Class Action Lawsuit Has Been Filed Against Rocket Pharmaceuticals, Inc. and Encourages Investors to...

NEW YORK, June 13, 2025 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized stockholder rights law firm, announces that a class action lawsuit has been filed against Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) (NASDAQ:RCKT) in the United States District Court for the District of New Jersey on behalf of all persons and entities who purchased or otherwise acquired Rocket securities between February 27, 2025 to May 26, 2025, both dates inclusive (the “Class Period”). Investors have until August 11, 2025 to apply to the Court to be appointed as lead plaintiff in the lawsuit.

RCKT Investor Notice: Robbins LLP Reminds Shareholders of the Class Action Lawsuit Against Rocket Pharmaceuticals, Inc.

Robbins LLP is Investigating Allegations that Rocket Pharmaceuticals, Inc. (RCKT) Misled Investors Regarding the Viability and Efficacy of RP-A501

Rakuten Medical Selected to Participate in FDA CEO Forum in San Diego

- Engaged with FDA Commissioner on shared commitment to fast, safe, and affordable patient access to innovative therapies SAN DIEGO, June 13, 2025 /PRNewswire/ -- Rakuten...

ROSEN, A LEADING LAW FIRM, Encourages Rocket Pharmaceuticals, Inc. Investors to Secure Counsel Before Important Deadline in Securities Class Action – RCKT

NEW YORK, June 13, 2025 (GLOBE NEWSWIRE) --

WHY: Rosen Law Firm, a global investor rights law firm, announces the filing of a class action lawsuit on behalf of purchasers of securities of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) between February 27, 2025 and May 26, 2025, both dates inclusive (the “Class Period”). A class action lawsuit has already been filed. If you wish to serve as lead plaintiff, you must move the Court no later than August 11, 2025.

Tiziana Life Sciences Expands Phase 2 Clinical Trial of Intranasal Foralumab with Commencement of First Patient Dosing at Weill Cornell Multiple Sclerosis Center

NEW YORK, June 13, 2025 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, today announces that dosing has commenced at the prestigious Weill Cornell Medicine Multiple Sclerosis Center in New York City, in its ongoing Phase 2 clinical trial evaluating intranasal foralumab in patients with non-active Secondary Progressive Multiple Sclerosis (na-SPMS). This fifth site complements existing sites at Yale University, Johns Hopkins University, Brigham and Women's Hospital, and the University of Massachusetts.

InspireMD Announces CE Mark Approval for CGuard® Prime Embolic Prevention System (EPS) Under European MDR for the Prevention of Stroke

MIAMI, June 13, 2025 (GLOBE NEWSWIRE) -- InspireMD, Inc. (Nasdaq: NSPR), developer of the CGuard® Prime carotid stent system for the prevention of stroke, today announced the company has received CE Mark approval under the European Medical Device Regulation (MDR) for the CGuard® Prime EPS.

Biomea Fusion Presents Updated Preliminary Clinical Data for Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory Acute Leukemia at EHA 2025

New clinical results show sustained CRi, deep bone marrow responses, and encouraging survival in FLT3-mutant AML patients, including those previously treated with gilteritinib
New clinical results show sustained CRi, deep bone marrow responses, and encouraging survival in FLT3-mutant AML patients, including those previously treated with gilteritinib

Merry Life Launches Global Phase II Trial of TML-6, The First Drug Targeted at Autolysosome pathway for Therapy of Alzheimer’s Disease

TAINAN, June 12, 2025 /PRNewswire/ -- Merry Life Biomedical Company announces the launch of its global Phase II clinical trial for TML-6, a novel...

Innovent to Present Multiple R&D Results of General Biomedicine Pipeline at the ADA’s 85th Scientific Sessions

SAN FRANCISCO and SUZHOU, China, June 12, 2025 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and...

Blueprint Medicines Announces Data Reinforcing Sustained Clinical Efficacy and Well-Tolerated Safety Profile of Long-Term AYVAKIT®/AYVAKYT® (avapritinib) Treatment at 2025 EHA and EAACI Congresses

-- Breadth of presentations, including one oral and two flash talks, showcase Blueprint Medicines' leadership role in advancing care for patients with systemic mastocytosis...

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