Latest news with #)Therapeutics
Business Insider
8 hours ago
- Business
- Business Insider
Celldex announces data on barzolvolimab from its Phase 2 trial in CSU
Celldex (CLDX) Therapeutics announced data demonstrating that barzolvolimab profoundly improves angioedema at 52 weeks in the Company's Phase 2 clinical trial in chronic spontaneous urticaria, CSU. The data were presented today by Dr. Martin Metz in an oral presentation at the European Academy of Allergy and Clinical Immunology, EAACI, Congress 2025. Celldex previously announced that the Phase 2 study in CSU met its primary and secondary endpoints at 12 weeks with clinically meaningful and statistically significant decreases in UAS7 compared to placebo across multiple dose groups, including improvements in quality of life and angioedema measurements, and demonstrated a favorable safety profile. The data presented further support these results by demonstrating improvements in AAS7 and additional measures of angioedema control over the 52 week treatment period. AAS7 measures the frequency and intensity of angioedema episodes, where higher scores indicate increased angioedema activity. Confident Investing Starts Here:
Business Insider
19 hours ago
- Business
- Business Insider
Sarepta suspends non-ambulatory Elevidys shipments after second death
Sarepta (SRPT) Therapeutics provided a safety update regarding Elevidys, an FDA approved gene therapy for patients with Duchenne muscular dystrophy. The company announced a second reported case of acute liver failure resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after Elevidys infusion, along with post-treatment monitoring, remains the same. Sarepta has also voluntarily paused dosing in the ENVISION clinical study. FDA concurs with this action, the company said. 'The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume,' Sarepta added. The company is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys.
