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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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At the heart of the acquisition is Regulus’ farabursen, an miRNA-targeting oligonucleotide in early-stage development for rare autosomal dominant polycystic kidney disease.
GSK’s dealmaking will be “cautious and disciplined” under the current trade war, but the pharma will focus on looking for “opportunities created” amid these tensions, according to CEO Emma Walmsley. The company also reported a 4% earnings bump for the quarter.
It’s been a fraught road for the proposed merger between Acelyrin and Alumis, with Tang Capital’s Concentra Biosciences in February threatening to upend the deal with a proposed $3-per-share acquisition of Acelyrin.
The drug, a small molecule protein inhibitor, brought in $132 million in the first quarter, missing consensus estimates by 17%.
As Q1 2025 earnings season continues, tariffs remain top of mind for pharma CEOs and investors. Meanwhile, the American Association for Cancer Research’s annual event kicks off this year’s oncology conference season. Plus, will the FDA become politicized under HHS Secretary RFK Jr.?
What a CEO makes can be staggering from the seat of a rank-and-file employee, whose pay is typically in the five-to-six digit range.
Policy uncertainties are impacting biopharma dealmaking from continent to continent, with companies being asked to walk a tightrope on their relations with China.
Nevertheless, Albert Bourla has been lobbying the U.S. government—as high as President Donald Trump himself—to skip the pharmaceutical industry tariffs, which Trump has threatened, in part as a way to shore up U.S. national security.
Despite executing perfectly, Octagon confronted a “scientific no-go,” CEO Isaac Stoner said in his LinkedIn post announcing the company’s impending closure.
The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.