Vertex Pharmaceuticals Inc. has tapped into Treefrog Therapeutics SA’s high-throughput stem cell manufacturing technology in a deal worth potentially up to $780 million to help it advance its type 1 diabetes programs, including phase I/II asset VX-880.
Some gene therapies could be big winners under the changes the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to Medicare’s new technology add-on program (NTAP) for its fiscal 2025 inpatient prospective payment system.
Vertex Pharmaceuticals Inc. is acquiring Alpine Immune Sciences Inc. for $4.9 billion in cash to gain Alpine’s lead product, phase III-ready povetacicept, which demonstrates best-in-class potential in patients with immunoglobulin A nephropathy (IgAN). Povetacicept (ALPN-303), or “pove,” holds potential “as a pipeline in a product in a number of other serious renal diseases and cytopenias,” Vertex CEO Reshma Kewalramani said during an April 10 conference call, noting that the deal was “just the right fit with just the right assets at just the right phase of development where Vertex can add value.”
Vertex Pharmaceuticals Inc. has described α1-antitrypsin (SERPINA1) (Z-mutant) polymerization inhibitors reported to be useful for the treatment of α1-antitrypsin (AAT) deficiency.
While some states are beginning to double down on the prices they pay for prescription drugs, the state of Colorado is taking it to a whole new level with its Prescription Drug Affordability Review Board that was empowered to set maximum prices of prescription drugs it considers “unaffordable.”
Vertex Pharmaceuticals Inc. has obtained IND clearance from the FDA for VX-407, an investigational first-in-class small-molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
When founders of Latigo Biotherapeutics Inc. first set out a few years ago to establish a biopharma firm focused in the area of pain, the plan had been to get a head start by in-licensing promising assets in the space. But that proved easier said than done. “With the exception of very early chemical matter” from the Lieber Institute for Brain Development, “we really couldn’t find anything else of quality to bring in, which I think is a testament to how little pain research and investment was ongoing in pharma and academia,” said Sean Harper, co-founding managing director at Westlake Village Biopartners, which founded Latigo in 2020 and led the firm’s $135 million series A round.
Vertex Pharmaceuticals Inc. upped its own ante with pivotal phase III data in its strong suit, cystic fibrosis (CF). Taking on Trikafta, the company’s bestseller, Vertex’s once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza) combo hit all primary and key secondary endpoints in two randomized controlled studies of those ages 12 and older.
Newly approved gene therapies targeting sickle cell disease will be the first focus of the U.S. Centers for Medicare & Medicaid Services’ (CMS) Cell and Gene Therapy Access Model, the agency said Jan. 30.
Despite Vertex Pharmaceuticals Inc.’s mostly positive phase III study results of a non-opioid for treating moderate to severe acute pain, the data for a key secondary endpoint show VX-548 is not as effective as Vicodin (hydrocodone, Abbvie Inc.).