Drug discovery
The conversion of Calquence’s accelerated approval in mantle cell lymphoma comes a day before the drug was listed among the 15 products to be subject to IRA-prescripted price negotiations for Medicare this year.
Concurrently, a preprint from the industry-backed Vital Transformation found a 50% drop in company investments into small-molecule drug development.
The FDA’s guidance on AI in drug development points to potentially life-threatening consequences of the technology, highlighting the importance of providing the regulator with detailed information regarding models’ development and maintenance.
Effectively treating and preventing this common form of dementia will require a cocktail of drugs and a combination of approaches, as well as a drive toward early detection.
Madrigal Pharmaceuticals, X4 Pharmaceuticals and Day One Biopharmaceuticals secured their maiden approvals this year in metabolic dysfunction-associated steatohepatitis, WHIM syndrome and pediatric low-grade glioma. Geron Corporation and ImmunityBio also notched wins.
Even as Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla slowly roll out onto the market, experts question the efficacy of these anti-amyloid antibodies and the amyloid hypothesis overall.
Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.
A slow launch for Alzheimer’s medicine Leqembi, a lackluster pipeline and a challenging drug launch environment are just a few of the factors that have sent Biogen’s shares down this year.
In a deal worth up to $285 million initially for the lead program, Novo Nordisk will gain access to Ascendis’ TransCon technology platform in an effort to find novel GLP-1 candidates with reduced dosing frequency.
Vertex may have pivoted away from the space, but candidates in development by Arrowhead/Takeda, Wave, Korro and others could address the damage underlying alpha-1 antitrypsin deficiency and make today’s treatments a thing of the past.
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