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When Doug Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed a fierce passion for the therapeutic area. He will step aside from his role to dedicate more time to his family.
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LB Pharma needed $350 million to advance a promising schizophrenia candidate at a time when the biotech markets were locked up tight. Fortunately, it wasn’t CEO Heather Turner’s first rodeo.
Rare disease drug developers struggle to survive in a biopharma investment market that prioritizes large patient populations. Initiatives like the Orphan Therapeutics Accelerator are attempting to solve what CEO Craig Martin says is not a science problem, but a math problem.
Eli Lilly’s win in a head-to-head trial drove Novo Nordisk’s market cap to pre-Wegovy levels not long after the victor became the first pharma company to top a $1 trillion valuation. It seems one company can do no right, while the other can do no wrong.
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Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.
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Our CEO accidentally started a book club. Now we’re all dreaming of mega pharma mergers.
In the Phase III SERENA-6 trial, camizestrant—in combination with CDK-inhibitors—beat out current standard-of-care treatments in terms of progression-free survival, according to AstraZeneca.
As high prices and supply issues drive consumers to alternative markets for GLP-1s, physicians aren’t too interested in using these therapies to treat conditions like heart disease risk that have existing cheap standards of care.
Eikon’s lead candidate, EIK1001, is being tested for advanced melanoma. The candidate is currently in late-stage development, which the biotech will fund using Wednesday’s series D raise.
SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say.
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.
As FDA seeks to rehire some fired employees, Donald Trump threatens to enact tariffs on pharma companies unless they reshore manufacturing; another lawsuit hits the complex GLP-1 compounding space as Eli Lilly offers expanded Zepbound options; and struggling gene therapy biotech bluebird bio goes private in an attempt to stay solvent.
While many industry players and observers have high hopes for the EPIC Act, some say budgetary headwinds could make it difficult for the current administration to make meaningful repeals or amendments to the IRA.
Kiniksa will now focus its development efforts on the IL-1 blocker KPL-387, which it is testing for recurrent pericarditis with Phase II/III trials planned for mid-2025.
Emalex is gearing up for a New Drug Application for ecopipam in Tourette syndrome later this year.