Pamrevlumab

2024 was a challenging year for the biopharma industry, with companies like Bayer, Bristol Myers Squibb, and Johnson & Johnson cutting hundreds to thousands of jobs. Cassava Sciences announced layoffs of 10 employees, 33% of its workforce, following a failed Phase III trial for its Alzheimer's drug. CytomX Therapeutics cut 40% of its staff to focus on clinical programs. Velia, a San Diego biotech, is shutting down, affecting 47 employees. Regeneron's acquisition of Oxular led to layoffs, with no Oxular employees joining Regeneron. Javara, Ring Therapeutics, Outlook Therapeutics, Editas, Bavarian Nordic, BenevolentAI, Chroma Medicine and Nvelop Therapeutics, Cellectar Biosciences, Carisma Therapeutics, Belharra Therapeutics, National Resilience, AmplifyBio, Agenus, Alligator Bioscience, Idorsia Pharmaceuticals, Kronos Bio, Novartis, Recursion Therapeutics, Medigene, Alector, Bristol Myers Squibb, Sonata Therapeutics, 23andMe, Johnson & Johnson, Merck, Gilead Sciences, Adaptimmune, Sensei Biotherapeutics, Marinus Pharmaceuticals, Orna Therapeutics, Thermo Fisher Scientific, Charles River Laboratories, Aurinia Pharmaceuticals, Viracta Therapeutics, Astellas Gene Therapies, Sana Biotechnology, Sage Therapeutics, Compass Pathways, Spero Therapeutics, ICON, Pfizer, Takeda, SalioGen Therapeutics, Evonik, Medtronic, CareFusion Resources, Turnstone Biologics, Leo Pharma, Astellas Pharma, Prime Medicine, Kaléo, Stryker, Relay Therapeutics, ImmunityBio, Shattuck Labs, Inventprise, bluebird bio, Athira Pharma, AGC Biologics, Oncternal Therapeutics, Biosense Webster, Vesigen Therapeutics, Connect Biopharma, BioMarin, IN8bio, Edwards Lifesciences, DermTech, Repare Therapeutics, Genentech, Tome Biosciences, Aadi Bioscience, Lykos Therapeutics, Evotec, Galera Therapeutics, Grail, Ovid Therapeutics, Lexicon Pharmaceuticals, Acelyrin, Boundless Bio, FibroGen, Ajinomoto Bio-Pharma Services, AN2 Therapeutics, Entero Therapeutics, Precigen, Sumitomo Pharma America, uniQure, Vir Biotechnology, Arbutus Biopharma, HilleVax, and Bayer also announced significant layoffs, reflecting a tough year for the industry.

Biotech stocks like Rapt Therapeutics, Sol-Gel Technologies, and Puma Biotechnology are seeing significant search interest surges, highlighting their focus on innovative treatments for diseases ranging from cancer to skin conditions.

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

Luca Richeldi discussed IPF clinical trial failures at ERS Congress 2024, highlighting the need to learn from past setbacks to improve future trial designs. He noted that not all negative trials are failures and cited the 2012 study as an example. Richeldi analyzed recent phase 3 trials (ziritaxestat, zinpentraxin alfa, pamrevlumab) that faced challenges, emphasizing the importance of larger sample sizes, longer study durations, and handling outliers. He proposed adaptive trial designs, rigorous statistical methods, and focusing on patient-relevant outcomes to enhance future IPF research.

FibroGen, Inc. reported a Q1 2024 EPS of $-0.33272, missing expectations. Key updates include progress on pamrevlumab for pancreatic cancer, with Phase 3 data expected in 2024, and roxadustat's strong performance in China, with a 24% sales increase. FibroGen also highlighted its early-stage oncology pipeline and a strong cash position of $214.7 million, sufficient to fund operations into 2026.

Sarepta Therapeutics received FDA accelerated approval for Elevidys, a gene therapy for DMD in children aged 4-5, pending confirmatory trial results. Bristol Myers Squibb's Camzyos was approved in the EU for treating obstructive HCM. FibroGen's pamrevlumab failed in a phase III IPF study, leading to discontinuation of related trials. Intercept Pharmaceuticals faced FDA rejection for its NASH treatment, prompting restructuring. MoonLake Immunotherapeutics reported positive phase II results for sonelokimab in treating HS.