The good news for Sarepta Therapeutics Inc. is bad news for Pfizer Inc. as the phase III study of its mini-dystrophin gene therapy in Duchenne muscular dystrophy (DMD) has missed its primary endpoint. Now Sarepta’s Elevidys (delandistrogene moxeparvovec), a single-dose, adeno-associated virus-based gene transfer therapy for DMD, is barreling toward a June 21 PDUFA date with the U.S. FDA as the near competition shrinks in the rearview mirror. Read More
Urogen Pharma Ltd. shares (NASDAQ:URGN) closed June 13 at $17.50, up $4.78, or 37%, on word of an 82.3% 12-month duration of response data by Kaplan-Meier estimate from its phase III Envision study with UGN-102. The finding was made in in low-grade, intermediate-risk, non-muscle invasive bladder cancer patients who achieved complete response at three months after the first instillation of the drug for intravesical solution. Read More
Following a phase II/III failure, Stamford, Conn.-based Cara Therapeutics Inc. discontinued its only pipeline program, that of oral difelikefalin for moderate to severe pruritis in adults with notalgia paresthetica, placing the company’s future in question. Read More
Santa Ana Bio Inc. launched with combined series A and B rounds that pulled down $168 million for its precision immunology push in inflammatory diseases, with an aim of entering the clinic next year across several fronts. Read More
Chinese artificial intelligence (AI)-driven drug discovery firm Quantumpharm Inc., also known as Xtalpi, began trading on the Hong Kong stock exchange June 13, listing under a new special technology listing track that lured it away from an IPO in the U.S. Read More
The BioWorld Drug Developers Index (BDDI) mirrored the movements of the Nasdaq Biotechnology Index and the Dow Jones Industrial Average, up in May after a decline at the end of April. BDDI ended May up 1.82% after closing in April down 4.8% from the beginning of the year. Read More
The Biosecure Act missed its first chance at a congressional ride June 11 when the U.S. House Rules Committee didn’t include it, as many had expected, on the list of potential riders the House will consider for its version of the National Defense Authorization Act, a must-pass defense spending bill for fiscal 2025. But that doesn’t mean the bill will be stranded by the wayside. Read More
The U.S. Supreme Court unanimously upheld the FDA’s relaxed regulation of the abortion drug mifepristone when it ruled June 13 that the organizations challenging those changes lacked the standing to do so. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Avidity, Biosion, City of Hope, Junshi, Nodthera, PDS, Ultragenyx. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Biosyent, Pfizer, Profound, Scancell, Sibylla. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Ascentage, Belite, Revive. Read More
