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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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BioSpace sat down with Roche Global Head of Neurodegeneration Rachelle Doody, Ph.D. and Alzheimer’s Drug Discovery Foundation Co-founder Howard Fillit, M.D. to discuss the highlights.
Four months after implementing a restructuring initiative to extend cash flow, X4 Pharmaceuticals is celebrating positive top-line results in WHIM syndrome.
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Shares of Horizon Therapeutics are soaring in premarket trading Wednesday after the rare disease company confirmed it is being courted for a potential acquisition by three pharma giants.
Nimbus reported positive Phase IIb results for NDI-034858 in moderate-to-severe plaque psoriasis, which researchers hope will have the efficacy of JAK inhibitors without the safety issues.
Nectin Therapeutics announced dosing of the first solid tumor patient in a Phase I clinical trial while additional investors jumped in to expand the company’s Series A to just over $25 million.
Full data from the Phase III Clarity AD trial show Eisai and Biogen’s lecanemab has a favorable safety profile in patients with Alzheimer’s disease.
BigHat Biosciences will team up with Merck to design candidates for three drug discovery programs backed by the company’s proprietary AI technology platform.
BMS is terminating a seven-year-old gene therapy collaboration to develop potential treatments for congestive heart failure with uniQure N.V. valued at $1 billion.