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Ultragenyx Publishes 96-Week Data for Genglycos
Ultragenyx announced the publication of 96-week data from its Phase 3 study of Genglycos, also known as DTX401, for the treatment of glycogen storage disease type Ia in The Journal of Inherited Metabolic Disease. At week 96, participants across the treatment and crossover groups experienced a 61% mean reduction in daily cornstarch intake while maintaining glycemic control. Additionally, 33% of the original treatment group and 42% of the crossover group completely eliminated nighttime cornstarch dosing. The company noted the gene therapy demonstrated an acceptable safety profile, with transaminase elevations managed by corticosteroid regimens.
Ultragenyx's Genglycos Receives FDA Accelerated Approval
Ultragenyx Pharmaceutical announced that the FDA granted accelerated approval for Genglycos, also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). "The approval of Genglycos fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa. The reduced reliance on cornstarch, experienced by patients in our clinical studies, demonstrates this gene therapy's ability to establish the normal breakdown of glycogen to produce glucose during fasting or episodes of metabolic stress. This ability to regulate glucose has alleviated the disease burden and has the potential to mitigate the risk of severe or life-threatening hypoglycemia for these patients," said Eric Crombez, M.D., Chief Medical Officer. "As our first gene therapy approval, Genglycos represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases."
Digital Turbine Shares Rise 24.3% After Earnings
Stock futures were mixed following this week's already sharp gains after another slate of big earnings reports. While SpaceX slipped after its first report as a public company, gains from Booking, Arista, and others weighed on futures. Crude oil futures were also lower amid optimism over a potential U.S.-Iran deal to reopen the Strait of Hormuz.Check out this evening's top movers from around Wall Street, compiled by The Fly.HIGHER AFTER EARNINGS -Digital Turbineup 24.3%Travere Therapeuticsup 12.4%Arista Networksup 9.2%Ultragenyxup 6.4%Bookingup 5.7%Wynn Resortsup 5.5%Paylocityup 3.4%ALSO HIGHER -Sight Sciencesup 9.6% after receiving FDA 510clearance of the Omni Ultra Surgical SystemDOWN AFTER EARNINGS -Teradatadown 18.4%ViaSatdown 9.5%Chemoursdown 9.4%Match Groupdown 9.1%Pinterestdown 9.1%AMDdown 8.7%Lucid Groupdown 7.8%DaVitadown 7.3%SpaceXdown 7.2%Lattice Semiconductordown 6.5%Coupangdown 6.1%
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