Vertex Pharmaceuticals Inc. has described orexin OX2 receptor agonists reported to be useful for the treatment of amyotrophic lateral sclerosis, obesity, hypertension, retinopathy, multiple sclerosis, narcolepsy, hypersomnia and Parkinson’s disease.
In denying Medicaid patients with sickle cell disease or transfusion-dependent beta-thalassemia access to Vertex Pharmaceuticals Inc.’s fertility preservation program, which is intended to counteract a side effect of the company’s gene-editing therapy, Casgevy, “the federal government now stands as the barrier between thousands of predominantly Black Americans and the necessary medical care that would protect their basic right to have biological children,” Vertex said in a lawsuit filed July 15.
Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel).
The BioWorld Biopharmaceutical Index (BBI) saw an 8.15% increase at the close of May, outperforming the Dow Jones Industrial Average, which rose by 2.64%, and the Nasdaq Biotechnology Index, which was up by 0.7%. The BBI previously showed a strong performance in Q1, followed by a dip for all three indices in April, before all rebounded in May.
Vertex Pharmaceuticals Inc. has divulged orexin OX2 receptor modulators reported to be useful for the treatment of amyotrophic lateral sclerosis, obesity, hypertension, retinopathy, multiple sclerosis, narcolepsy, hypersomnia and Parkinson’s disease.
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.”
