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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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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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The biopharma’s acute myeloid leukemia hopeful is on partial clinical hold, affecting two Phase III AML studies, Gilead announced Monday. The CD47 candidate also spent four months on hold in 2022.
The companies will evaluate Moderna’s investigational Claudin18.2 mRNA cancer vaccine in combination with CARsgen’s Claudin18.2 CAR T cell candidate.
After sustaining two Phase III fails, Roche and Exelixis finally reported a late-stage win for their combination regimen of Tecentriq and Cabometyx in metastatic castration-resistant prostate cancer.
Following a prior rejection due to manufacturing problems, Regeneron’s higher-dose Eylea won the FDA’s approval Friday, allowing for more infrequent dosing for the treatment of various eye diseases.
The regulator’s greenlight on Friday for Regeneron Pharmaceuticals’ monoclonal antibody Veopoz (pozelimab) makes it the first and only treatment indicated for children and adults with CHAPLE disease.
Boehringer Ingelheim will launch three Phase III studies for its obesity drug candidate; third time is a charm for Ipsen as it gets FDA approval; and Pfizer takes multiple myeloma battle to J&J.
The company is continuing its renal cell carcinoma push for its oral HIF-2α inhibitor Welireg with promising Phase III data showing significant progression-free survival improvements in patients.
In a European Phase I/II clinical trial, the experimental drug for treating Crigler-Najjar syndrome reduced bilirubin below toxic threshold in the liver with a single intravenous injection.
After the company’s investigational eye therapy recently failed two late-stage studies, Kodiak Sciences’ Chief Medical Officer and Chief Development Officer Jason Ehrlich has resigned from his roles.
The companies contend that their updated COVID-19 vaccines for the fall vaccination season can elicit strong immune responses against the virus’ currently dominant and emerging subvariants.