Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43. Read More
Zevra Therapeutics Inc. will make its case Aug. 2 for its Niemann-Pick type C (NPC) candidate, arimoclomol, when the U.S. FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) meets for the first time. Read More
The disappointments continue for Fibrogen Inc., which is terminating work on its once-promising anti-CTGF monoclonal antibody, pamrevlumab, after reporting missed endpoints in two late-stage pancreatic cancer studies, and cutting its workforce by about 75%. Read More
Macrogenics Inc., agreeing with an independent data monitoring committee, has discontinued dosing patients in the troubled phase II Tamarack study of vobramitamab duocarmazine as a second-line treatment for metastatic castration-resistant prostate cancer. Read More
Radiopharm Theranostics Ltd. said it plans to list on Nasdaq by the end of 2024. The company had earlier announced plans to list in February 2023, but it delayed the listing due to market conditions.
Karen Carey, BioWorld managing editor and chief analyst, takes a look at the numbers from the first half of 2024. She finds the first six months to be healthy for the biopharma market while the rest of the year is populated with question marks that include the U.S. presidential election and potential interest rate cuts. Read More
From no hope to viable treatments, BioWorld is there to cover the breakthroughs in medicine. Listen to Randy Osborne explain why the BioWorld team is ‘always on the lookout for what's next.’ Read More
Biopharmas raising money in public or private financings, including: Arch Biophartners, Chemomab, Day One, LTZ, Novabay, Pinetree, Synthetica. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aspargo, Biontech, Celldex, Clearside, Imunon, Nkgen, Novo Nordisk, Roche Holding, Yamo. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Cortice, CNS Pharmaceuticals, Leveragen, Moderna, Mthera, Neurobo, Neutherx, VBI Vaccines, Verseon. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Amneal, Bio-Thera, Clearside, Dr. Reddy's, Galderma, Kiora, Neurim, Parvus, Stallergenes, Verastem. Read More
