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Disc Medicine’s leadership tried to express optimism that its rare disease therapy bitopertin can be approved based on a Phase 3 trial set to begin shortly. However, analysts are worried that the protocol was developed with former FDA leaders.
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The current state of political affairs in the U.S. does not bode well for the direction of that turn. The country is at real risk of losing its long-held lead in biotech innovation.
The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
The rare disease drugmaker is facing potential competitors for achondroplasia drug Voxzogo. Is a big M&A deal with two approved assets enough to maintain investor interest?
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The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
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Projected to be worth over $38 billion in the global healthcare market by 2032, AI simulations have the potential to streamline clinical trials and help address inequities in underserved patient populations.
Eli Lilly topped the list of the 20 biggest pharmas by market cap with a more than 39% improvement year-to-date in its share price. Other companies have not been so lucky.
Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock.
Trump is rounding out his health cabinet with another controversial figure: one of the authors of the Great Barrington Declaration, which advocated for herd immunity through infection during the COVID-19 pandemic.
BridgeBio’s Attruby wins approval for transthyretin amyloid cardiomyopathy while the FDA accepts Alnylam’s application for Amvuttra in the indication; Cassava’s controversial Alzheimer’s drug flunks Phase III; Amgen’s MariTide fails to impress investors, Donald Trump’s controversial nominations continue.
With Elevidys expansion in hand, Sarepta commits up to $10 billion to develop short interfering RNA–based drugs to build out its pipeline.
With Amgen’s MariTide results at the lower end of investors’ expectation of 20% to 25% weight loss, the much-anticipated readout sent the company’s shares tumbling.
Currently, Eli Lilly’s GLP-1 drugs Mounjaro and Zepbound (tirzepatide) are not on the FDA’s shortage list but compounded pharmacies are still making them. That’s unprecedented.
Alector is kicking off a resource realignment effort that will include a workforce reduction. The biotech expects its current cash position to last it through 2026.
Truqap’s positive clinical data comes after it failed a late-stage study in metastatic triple-negative breast cancer. It helps AstraZeneca position itself as a top player in the prostate cancer space, alongside its Big Pharma colleagues.