Today, a brief rundown of news involving UniQure and Bridge Minds Biosciences, as well as updates from Specific Biologics, Teva Pharmaceuticals and CinRx Pharma that you may have missed.
Following a high-profile back and forth with UniQure, the Food and Drug Administration has agreed to review an approval application for the company’s Huntington’s disease gene therapy. UniQure said Monday the agency is doing a faster-than-usual review and should have a verdict by May 1. The FDA is also tentatively planning to discuss the application in a meeting with external advisors, according to UniQure. Though company shares had fallen 7% by late Monday, Wall Street analysts generally viewed the update as positive for what could be the first disease-modifying treatment for Huntington’s. Joseph Thome, of TD Cowen, wrote in a note to clients that his team expects key doctors in the field to adopt the therapy. He currently models annual sales reaching $1.6 billion in 2039. — Jacob Bell
Elsewhere at the FDA, an application to begin a mid-stage trial for an oral antiseizure medication has been placed on partial clinical hold. The proposed trial is designed to evaluate ascending doses of “BMB-101” — which is meant to activate select receptors that, in turn, inhibit certain channel proteins involved in seizures. According to the medication’s developer, New York-based Bright Minds Biosciences, the primary reason the FDA gave for the partial hold was toxicity identified in mouse testing. Bright Minds said the partial hold does not affect its other ongoing trials. The company also said it believes the mouse findings were “incidental and not drug-related,” and that this toxicity was not seen in any other species. Bright Minds plans to “immediately” request further clarification from the FDA. Company shares lost more than half of their value Monday, closing at $26.20 apiece. — Jacob Bell
Specific Biologics, a Toronto-based developer of gene editing therapies, announced on Monday the closing of a Series A funding round led by Hatteras Venture Partners. Specific said the $35 million haul was greater than originally planned, and saw participation from half a dozen other investors, including a life sciences venture fund managed by the Business Development Bank of Canada. Proceeds will go toward an early stage trial that hopes to, via biological markers, show Specific’s most advanced therapy works as intended in people with certain genetic mutations linked to ALS. — Jacob Bell
Teva Pharmaceuticals on Monday said it will stop early a mid-stage study of a drug it’s been testing in the rare neurodegenerative condition multiple system atrophy. After an interim data check, emrusolmin, a pill it’s developing with Modag GmbH, met the criteria for “futiilty,” meaning there was little chance the therapy would succeed if the study continued. Teva will evaluate the data before determining the path forward for enrusolmin. The outcome is “disappointing,” but “not core to the thesis” for Teva, wrote Dennis Ding, an analyst at Jefferies, in a Monday note to clients. — Gwendolyn Wu
CinRx Pharma, the startup creator behind a biotech AstraZeneca bought in 2023, is taking another swing at cardiovascular drug development through a partnership with Graviton BioScience. In a Monday announcement, the two said they’ve formed a joint venture named CinRock Pharma that’ll advance a pair of experimental drugs that inhibit so-called ROCK2 enzymes. CinRock will test those drugs in pulmonary arterial hypertension, a form of heart failure and other unnamed cardiovascular conditions. CinRock is funded by CinRx and got its drug prospects from Graviton, a Sam Waksal-led startup that previously partnered with Ovid Therapeutics to develop ROCK2-targeting treatments for rare brain disorders. — Gwendolyn Wu