Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial.
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After Friday’s news that Center for Biologics Evaluation & Research Chief Vinay Prasad will leave the FDA—again—at the end of April, stocks for several rare disease drug developers popped. UniQure, in particular, was up 51% in premarket trading on Monday. Prasad in a meeting last Thursday with select journalists called the biotech’s Huntington’s treatment AMT-130 a “failed” therapy, according to STAT News. Shares of Replimune and REGENXBIO—which have suffered rejections during the past year—also rose.
One person who is not impressed with the plethora of rare disease drug rejections of late—H.C. Wainwright said in a note Tuesday that there have been at least five cell and gene therapies they believe could have been approved under prior FDA officials—is Wisconsin Senator Ron Johnson. Tuesday, Bloomberg News reported that Johnson has launched an investigation into these recent denials.
Johnson called the FDA’s request that uniQure conduct a sham surgery-controlled trial of AMT-130 “bureaucratic idiocy,” according to the publication. Meanwhile, uniQure and the FDA appear to be on different pages regarding the design of this prospective trial, with uniQure Chief Medical Officer Walid Abi-Saab referring to a 10-12 hour surgery during which [burr] holes would be drilled in patients’ skulls and Prasad claiming on a media call last week that it would require only “one to three nicks in the scalp.”
In other news, no episode of The Weekly would be complete without our weekly weight loss segment. Roche and Zealand Pharma’s amylin analog fell short of Eli Lilly’s rival candidate eloralintide; AbbVie reported what analysts called “competitive” results, with its amylin analog eliciting nearly 10% weight loss at 13 weeks in a Phase 1 trial; and Regeneron touted a much-needed Phase 3 win for Hansoh-partnered dual GLP-1/GIPR agonist olatorepatide in China. Beyond data, Novo Nordisk and Hims & Hers are together again, with Novo striking a deal to sell its injectable and oral GLP-1 medicines through the telehealth provider.
Elsewhere on the business side of biopharma, experts are reporting a cut-throat atmosphere behind doors on the M&A front as the supply of companies available to buy dwindles.
