Clinical Trial News

The EMA's CHMP recommended against renewing Translarna's conditional approval for Duchenne muscular dystrophy (DMD) treatment, citing insufficient evidence of efficacy in a key patient group. PTC Therapeutics plans to request a re-examination, emphasizing Translarna's benefits. Translarna remains available in the EU pending review, with a final decision expected by January 2024.

Recent breakthroughs in Alzheimer's treatment, notably lecanemab's FDA approval and donanemab's Phase 3 trial success, offer new hope for patients and investment opportunities. Despite challenges in access and infrastructure, these developments mark a significant step forward in combating Alzheimer's, promising better outcomes and reducing healthcare burdens.

Study on axi-cel infusion in 62 aggressive B-cell non-Hodgkin lymphoma patients showed a 71.0% complete metabolic response (CMR) at 3 months. Secondary outcomes included a 75.8% objective response rate (ORR) at 3 months and 59.7% CMR at 6 months. Adverse effects were common, with 95.2% experiencing grade 3 or higher effects, including neutropenia and infections like COVID-19. Efficacy and safety were consistent across age and comorbidity subgroups.

Acelyrin Inc's stock (NASDAQ:SLRN) drops 62% to $10.60 after its skin disease drug fails in a trial. Jefferies cuts price target to $15 from $31, calling the data 'painfully disappointing.' Despite a 'strong buy' rating from analysts, SLRN faces volatility and lands on the short sell restricted list.

Acelyrin's stock dropped after disappointing Phase 2b trial results for izokibep in treating Hidradenitis Suppurativa. Despite not meeting the primary endpoint, early HiSCR100 responses and dose-effect data support further evaluation. Analysts maintain a Strong Buy rating for SLRN.

Acelyrin's IL-17A inhibitor, izokibep, did not meet the primary endpoint in a phase 2b/3 trial for hidradenitis suppurativa treatment, with 39% of patients on once-weekly 160 mg achieving HiSCR75 at 16 weeks vs. 29% on placebo. Despite high discontinuation rates and unexpected placebo response, izokibep showed potential in HiSCR100 and has an ongoing phase 3 trial.

Risankizumab showed non-inferiority for clinical remission at week 24 and superiority for endoscopic remission at week 48 compared to ustekinumab in treating Crohn's disease. All secondary endpoints were statistically significant, with a consistent safety profile for risankizumab.

FDA approved updated Pfizer and Moderna Covid vaccines targeting XBB.1.5 for ages 12+, with emergency use for younger children. Availability pending CDC approval. Novavax's vaccine under review. Vaccines aim to protect against current strains as cases and hospitalizations rise.

Vir Biotechnology Inc's shares dropped 54% YTD due to disappointing influenza A and HBV trial results, leading BofA to downgrade the stock to Neutral and lower its price target to $14. Despite a negative enterprise value offering downside protection, near-term HBV trial readouts are unlikely to improve investor sentiment. Key HBV trial results expected in 4Q23 and 2Q24 could impact future share trajectory.

The EMA is reviewing givinostat for Duchenne muscular dystrophy (DMD) treatment, following a similar FDA priority review. Givinostat blocks HDACs to potentially improve muscle repair and strength, based on Phase 3 trial results showing slowed motor function decline and delayed muscle fat build-up in DMD patients.