Acrivon Therapeutics Inc. provided updated phase II data for checkpoint kinase candidate ACR-368, highlighting promising response rates for biomarker-positive patients with endometrial cancer and raising the possibility of an accelerated pathway in the second-line setting.
Ushering in a new class of antibiotics, the U.S. FDA approved GSK plc’s gepotidacin for use in uncomplicated urinary tract infections. Branded Blujepa, the oral triazaacenaphthylene bacterial topoisomerase inhibitor is indicated for treating female adults and adolescents, 12 and older.
Shares of Unity Biotechnology Inc. (NASDAQ:UBX) sank 28.8% to close at $1.30 March 24 as a top-line readout of its phase IIb Aspire study testing UBX-1325 head-to-head against aflibercept in diabetic macular edema (DME) fell short of statistical noninferiority on the primary analysis endpoint. But that’s not the full story, according to company executives, who have plans for moving the senolytic Bcl-xL inhibitor into late-stage studies.
Black Diamond Therapeutics Inc. is getting $70 million up front and could earn up to $710 million in milestone payments in a deal with Servier for phase I-stage BDTX-4933, a small-molecule candidate targeting both RAS mutations and RAF alternations for treating solid tumors, including non-small-cell lung cancer.
While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with plans for a BLA filing by the end of 2025 for del-zota, an antibody-oligonucleotide conjugate, in Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), based on positive top-line data that analysts say bode well for Avidity’s other late-stage programs targeting rare neuromuscular diseases.
The first treatment for macular telangiectasia type 2 is set to enter the market following U.S. FDA approval of NT-501 (revakinagene taroretcel) from Neurotech Pharmaceuticals Inc., a privately held company that has been quietly advancing its encapsulated cell therapy platform for more than two decades.
For executives of Lexicon Pharmaceuticals Inc., the missed primary endpoint in the phase IIb Progress study testing pilavapadin, its non-opioid candidate, in adults with moderate to severe diabetic peripheral neuropathic pain seemed merely a footnote for what CEO Mike Exton called an “exciting and long-awaited day for the Lexicon team, collaborators and patients.”
Though it’s been used off-label for more than three decades to treat cerebrotendinous xanthomatosis, Mirum Pharmaceuticals Inc.’s chenodiol gained an official U.S. FDA nod Feb. 21 as the first drug approved specifically for treating the rare autosomal recessive lipid storage disease.
As the flurry of executive orders continues to flow from the White House, leaving in its wake chaos and confusion across the overall health care sector, Tim Opler, managing director of the global health care group at investment firm Stifel, is taking the long view and urging the biopharma sector to continue focusing on innovation.
Pliant Therapeutics Inc., which offered few details earlier this week when it voluntarily paused enrollment in the phase IIb Beacon-IPF trial testing bexotegrast in idiopathic pulmonary fibrosis (IPF), has taken an unusual step as it assembles a panel of outside experts to review unblinded data from the study with the goal of providing an independent recommendation.
