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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Regeneron is aiming to file a regulatory application for DB-OTO by the end of the year.
Moderna’s mRNA-4359, when used with Keytruda, achieves a 24% overall objective response rate in patients with melanoma, with efficacy increasing to 67% in those positive for PD-L1.
The market outlook is full of uncertainty and pitfalls. Experts weigh in on how to mitigate risks.
While the benefits of AI are clear, the amount data sets needed for effective AI integration is proving to be a challenge. This is particularly true for cell therapy companies as they are eagerly seeking ways to reduce development costs. Two experts at Charles River Laboratories provide insights by giving their takeaways from their own AI integrations.
To drive true innovation in drug development, executives must not let excitement about the latest shiny object obscure ultimate outcomes.
Therapies from industry leaders BioMarin and Ascendis Pharma supply a key hormone that promotes bone growth. In order to move the field forward, challengers are looking to address the underlying cause of the rare, genetic disease.
Sanofi’s Orano Med-partnered radioligand therapy AlphaMedix achieved all primary efficacy endpoints, which included a measure of overall response rate, in the mid-stage ALPHAMEDIX-02 study.
The U.S. government remains shut down, with the FDA closed for new drug applications until further notice; cell and gene therapy leaders gather for the annual meeting in Phoenix with the field in a state of flux; Pfizer and Amgen will make drugs available at a discount as President Donald Trump’s tariffs still loom; and new regulatory documents show how Pfizer beat out the competition for Metsera.
Skye Bioscience’s nimacimab fell short of investor and company expectations, but showed encouraging weight-loss results when combined with Wegovy, according to analysts at William Blair.
As industry leaders gather at the annual event in Phoenix, the cell and gene therapy space remains in a state of flux, with M&A activity and regulatory support signaling momentum while commercialization challenges continue to hinder broader investor interest.