Ultragenyx Publishes Positive 96-Week GSDIa Gene Therapy Trial Data
RARE sits 42% above its 52-week low of $18.29.
Summary
Ultragenyx published 96-week data from its randomized, placebo-controlled trial of GENGLYCOS (DTX401) in GSDIa in the Journal of Inherited Metabolic Disease. The trial showed successful crossover treatment, reinforcing the therapy's efficacy and safety profile. This follows the FDA's accelerated approval of GENGLYCOS on August 19 and the $2.7M price announcement on August 24. The publication adds peer-reviewed validation and long-term data, which could support reimbursement and adoption. Watch for the UX111 PDUFA date on September 19 as the next catalyst.
At the time of this announcement, RARE was trading at $25.97 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $2.5B. The 52-week trading range was $18.29 to $39.89. This news item was assessed with positive market sentiment and an importance score of 7 out of 10. Source: Reuters.