U.S. Stock Futures Fall, Ultragenyx Pharmaceutical Shares Down 8% Amid FDA Complete Response Letter for UX111 Gene Therapy

U.S. stock futures fell, with the Dow futures declining around 100 points on Monday. Shares of Ultragenyx Pharmaceutical Inc. RARE dropped 8% to $27.15 in pre-market trading after receiving a Complete Response Letter from the FDA for UX111 AAV gene therapy. Other stocks moving lower include MiNK Therapeutics, Above Food Ingredients, Newegg Commerce, CEL-SCI Corporation, Unusual Machines, Gossamer Bio, Gilat Satellite Networks, and KalVista Pharmaceuticals. Apple Inc. shares fell 1% to $209.22 in pre-market trading amid a management reshuffle.

U.S. stock futures fell on Monday, with the Dow futures declining by around 100 points. Shares of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) dropped by 8% to $27.15 in pre-market trading following the company's receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its UX111 AAV gene therapy, intended to treat patients with Sanfilippo syndrome type A (MPS IIIA) [1].

In the CRL, the FDA requested additional information and improvements related to specific aspects of chemistry, manufacturing, and controls (CMC) and observations from recently completed manufacturing facility inspections. Ultragenyx believes these observations are readily addressable and not directly related to the quality of the product. The company will work with the FDA over the next few months to resolve these issues and expects to resubmit the Biologics License Application (BLA) [1].

Clinical reviews have been ongoing, and the FDA has acknowledged the robustness of the neurodevelopmental outcome data and the supportive biomarker data. The CRL did not note any review issues related to the clinical data package or clinical inspections, and the company is required to include updated clinical data from current patients in the resubmission [1].

The setback is likely to delay the potential approval of UX111 to 2026. Ultragenyx aims to address the CMC observations swiftly to resubmit the BLA as soon as possible. If approved, UX111 would be a significant advancement for patients with MPS IIIA, a rare and fatal lysosomal storage disease with no approved treatment [2].

Other stocks moving lower in pre-market trading include MiNK Therapeutics, Above Food Ingredients, Newegg Commerce, CEL-SCI Corporation, Unusual Machines, Gossamer Bio, Gilat Satellite Networks, and KalVista Pharmaceuticals. Apple Inc. shares fell 1% to $209.22 amid a management reshuffle [1].

References:
[1] https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-receives-complete-response-letter-fda-ux111-aav-gene
[2] https://www.marketscreener.com/quote/stock/ULTRAGENYX-PHARMACEUTICAL-15672076/news/Ultragenyx-Pharmaceutical-Inc-Receives-Complete-Response-Letter-from-FDA-for-UX111-AAV-Gene-Therapy-50496214/