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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Erasca announced that it was expanding its existing partnership with Pfizer to assess its ERAS-007 in combination with the Big Pharma’s Ibrance.
Roche and Austria-based Hookipa Pharma tied up a licensing and research and development collaboration valued at more than $950 million focused on KRAS-mutated cancers.
After the Data Monitoring Committee reviewed the case based on protocol modifications implemented earlier this year, it was determined that enrollment in the trial and dosing may continue.
BMS’s blockbuster checkpoint inhibitor Opdivo hit the primary endpoint in the Phase III CheckMate -76K trial for a particularly intractable form of melanoma.
Kyverna Therapeutics filed its first IND application this week to test the efficacy and tolerability of KYV-101, a CAR-T therapy for lupus nephritis.
The FDA’s Obstetrics Reproductive and Urologic Drugs Advisory Committee voted nearly unanimously to withdraw the approval of Covis’ preterm birth drug Makena.
While worthy advances have recently been made in sickle cell disease, companies such as Graphite Bio, bluebird bio, Vertex Pharmaceuticals and Editas Medicine have loftier ambitions.
Massachusetts-based PIC Therapeutics has closed a $35 million Series A financing round centered around the advancement of its lead asset, a novel therapeutic for advanced metastatic breast cancer.
A Phase II trial studying PTC Therapeutics’ PTC518 in Huntington’s disease has been paused in the United States following a request from the FDA for additional data.
Jazz and Zymeworks entered into a $50 million licensing agreement over zanidatamab, Zymeworks’ bispecific antibody targeting HER2.