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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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Novartis has been investing heavily in its cardiovascular pipeline this year, forging partnerships with Flagship startup ProFound Therapeutics and China’s Argo Biopharmaceutical, among others.
Regeneron’s antibody duos significantly lowered eye itching and redness, as well as pin prick reactivity, in people with cat and birch allergies. Still, BMO Capital Markets expressed uncertainty about the assets’ “commercial potential in a highly generic market.”
Ori Biotech’s CEO said the prioritization of review by FDA, coupled to the impact of the technology, could shave up to three years off development timelines.
With two late-stage programs set to read out in the next 48 months, Biogen is translating its wealth of experience in multiple sclerosis to lupus—developing a pipeline BMO Capital Markets analysts called “thoughtful.”
The 2025 Bioprocessing Summit revealed knowledge gaps and changing mindsets. As some companies look towards the future, others struggle with the present and past.
Presenting at the World Sleep Congress 2025, the Dublin-based company’s Phase II study bested Takeda drug in both efficacy and safety.
While a win for consumers, the regulatory action did nothing to stem the manufacture of compounded versions of the popular obesity drugs that are made by Novo Nordisk and Eli Lilly. In fact, the FDA seems to be signaling that “some level of compounded product is acceptable,” according to BMO Capital Markets.
Shares of Rapport Therapeutics popped Monday morning after Phase IIa data for RAP-219 exceeded analyst and Wall Street expectations, reducing seizures by almost 78% in patients with drug-resistant focal onset seizures.
Ivonescimab elicited better overall survival in Asian patients with non-small cell lung cancer than in those from North America and European countries, in Western countries narrowly missing the statistical significance threshold the FDA is seeking.
KER-0193 is a modulator of ion channels connected to autism spectrum disorder. The FDA bestowed orphan drug and rare pediatric drug designations on the candidate earlier this year.