Drug Development
The mid-stage disappointment in Alzheimer’s disease delivers another blow to Neuphoria Therapeutics, which in November last year was forced to launch a strategic business review after a Phase 3 trial in social anxiety disorder failed.
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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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Prothena has already discontinued the development of birtamimab, and anticipates further spending cuts, including workforce reduction.
As the World Health Organization initiates a new agreement for coordinating global responses to future pandemics, the future of vaccine development in the U.S. faces growing challenges, including waning funding and regulatory changes, that threaten next-gen COVID-19 vaccine candidates and pandemic preparedness more broadly.
Vocal skeptics of COVID-19 vaccinations gave mRNA a bad name and government funding for mRNA research is now being cut. On the flip side, at least one CEO said the pandemic also provided “elevated acceleration” for the field, which also holds promise in therapeutics for cancer and rare diseases.
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.