Drug Development
Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.
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Analysts, investors and scientists are eager for Biogen’s 2026 BIIB080 readout. Even if successful, executives warn that there are many more steps before the Alzheimer’s therapy could reach the market.
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
After advancing in lockstep through the pandemic, the fortunes of the biotechs have diverged as their use of COVID-19 windfalls has taken shape.
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Beginning this week in Chicago, the American Association for Cancer Research’s annual conference will feature presentations that could have far-reaching implications for breast and blood cancers and more.
Analysts were effusive about Merus’ new HNSCC data, writing that petosemtamab could “become the standard of care” in the first-line setting for this indication.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the importance of target product profiles, particularly when navigating funding challenges, with Ian Fisher, head of development analytics.
From a higher bar for regulatory clearance to pricing limitations, drug development is more expensive than ever. This has led firms to make tough pipeline decisions early in the development process. The result may be costly for all of us.
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide Tryngolza in the third quarter, building up toward a regulatory submission in hypertriglyceridemia by year-end.
In a year when eradicated diseases are on the uptick in America, how will American children survive RFK Jr.’s vaccine scrutiny and inconsistency? Two experts call on pharma and regulatory bodies to rebuild trust.
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Govorestat failed to meet its primary endpoint in a Phase II/III trial for a rare form of Charcot-Marie-Tooth disease, a few months after the FDA rebuffed the same drug in a similar indication.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.