News
BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
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Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
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Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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Takeda and Arrowhead Pharmaceuticals’ Phase II trial studying fazirsiran, an investigational RNA therapeutic developed for AATD-LD, was effective in Phase II–but so was the placebo.
Day One Biopharmaceutical reported positive topline data from a Phase II trial in pediatric low-grade glioma, confirming earlier results.
Italian biopharma Chiesi Farmaceutici SpA announced Sunday it was acquiring rare disease-focused Amryt Pharma in a deal that could reach up to $1.48 billion in value.
The pharma titan adds an intriguing blood pressure medication to its line up with a buyout of CinCor.
Ipsen bolsters rare disease portfolio with acquisition of liver disease specialist Albireo.
The FDA approved Eisai and Biogen’s lecanemab (Leqembi) Friday afternoon. It is the second anti-amyloid antibody to be approved for Alzheimer’s disease in two years.
PCN-101, a ketamine-based candidate for treatment-resistant depression being developed by Atai’s Perception Neuroscience, fell short in a Phase II trial.
Thursday, Fate Therapeutics announced it terminated the 2020 collaboration agreement with Johnson & Johnson’s biopharma subsidiary Janssen.
Graphite Bio announced it is voluntarily pausing the Phase I/II study of its lead candidate nula-cel in sickle cell disease.
Moderna and CytomX ink a cancer partnership.