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SpringWorks Therapeutics is the perfect case study for rescuing a discontinued assets. It’s time to repeat the process for every rare disease, experts say.
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To drive true innovation in drug development, executives must not let excitement about the latest shiny object obscure ultimate outcomes.
Heading into the final quarter of a year that has seen dramatic upheaval at the FDA—from the exodus of numerous senior leaders to unclear policy changes and a safety saga that engulfed the gene therapy space—drug approvals appear roughly on par with recent years.
Therapies from industry leaders BioMarin and Ascendis Pharma supply a key hormone that promotes bone growth. In order to move the field forward, challengers are looking to address the underlying cause of the rare, genetic disease.
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Vertex Pharmaceuticals Incorporated and Lonza announced a strategic collaboration to support the manufacture of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapies for people with T1D, currently focusing on the VX-880 and VX-264 programs that are currently in clinical trials.
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After Emma Walmsley steps down as GSK CEO in January, Vertex Pharma’s Reshma Kewalramani will be the sole female CEO at a top-20 pharma company. Still, there are many prominent women in pharma that could someday break through again.
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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.
Driven by the early approval of its updated COVID-19 vaccine, BioNTech far exceeded analysts’ expectations in the third quarter and reported its first quarterly profit in 2024. However, the German biotech also cut its outlook for the year.
Multiple players are exploring whether modalities designed to combat B cell malignancies can be repurposed against lupus, myasthenia gravis and other conditions traced to misdirected immune response.
The Big Pharma companies made a last-ditch effort asking a U.S. appeals court to reconsider their lawsuits against the Inflation Reduction Act’s Medicare drug price negotiations, which they contend infringe on their constitutional rights.
Jefferies analyst Roger Song in an investor note said that Viking Therapeutics’ readout for its investigational therapy VK2735 exceeded expectations, with “class-leading” weight loss. Patients on 100-miligram doses of the pill lost 8.2% of their body weight after 28 days.
The FDA is set to decide on four promising therapies in the next two weeks, including a CAR T for acute lymphoblastic leukemia.
This year has seen several biopharma companies drop Alzheimer’s and Parkinson’s disease programs, but experts say plenty are still chasing these multi-billion-dollar markets.
While some of the initial excitement around immunotherapies has waned, companies—particularly smaller biotechs—are developing newer iterations that will take cancer care to the next level.
In part 1 of the pivotal ESSENCE trial, Novo Nordisk’s weight loss drug Wegovy demonstrated “statistically significant and superior improvement” in liver fibrosis in patients with metabolic dysfunction–associated steatohepatitis.
Lilly CEO Dave Ricks in Wednesday’s third-quarter earnings call acknowledged that the company is at the mercy of wholesaler stocking decisions.