NASDAQ — Healthcare: Manufacturing, Biotechnology: Pharmaceutical Preparations
www.ultragenyx.comThe US9186344 patent for the product DOJOLVI, which relates to a method for treating glycogen storage disease using ketogenic odd carbon fatty acids, is set to expire on July 1, 2025. This expiration could impact the competitive landscape for ULTRAGENYX PHARMACEUTICAL INC, as it may allow for the introduction of generic alternatives to DOJOLVI, potentially affecting future revenue streams.
patentThe clinical trial for Triheptanoin, designated as NCT06067802, is set to commence on July 1, 2025. This trial is significant as Triheptanoin is studied for its potential use in treating glycogen storage disease through the administration of ketogenic odd carbon fatty acids. This event coincides with the expiration of patent US9186344 related to DOJOLVI, underscoring its strategic importance for ULTRAGENYX PHARMACEUTICAL INC and Baylor Research Institute as the market landscape may change with the patent's expiration.
clinical trial startUltragenyx Pharmaceutical Inc announced the expected completion of its Phase 3 clinical trial DTX401-CL301 for Glycogen Storage Disease Type Ia (GSDIa) on February 1, 2026. This trial aims to assess the efficacy and safety of DTX401 in patients aged 8 and older. Participants in the study are randomized to receive either DTX401 or a placebo and are monitored over 48 weeks, with a crossover to DTX401 for those initially receiving placebo. The trial is currently active and has enrolled 49 participants. The results may impact Ultragenyx’s growth prospects as it seeks to expand its treatment portfolio for rare diseases.
clinical trialUltragenyx Pharmaceutical Inc. is expected to complete its clinical trial comparing Setrusumab against a placebo for Osteogenesis Imperfecta on April 1, 2027. This trial is active but not recruiting new participants and has a total enrollment count of 182. Setrusumab is intended to provide treatment for individuals with this genetic disorder affecting bone strength. The trial consists of both Phase 2 and Phase 3 studies, where participants will receive either low or high doses of Setrusumab or a placebo. Patients in extended treatment periods may continue receiving Setrusumab until it becomes commercially available in their region.
clinical trial completionRARE expects to complete the clinical trial for Triheptanoin, designated for children with Primary-Specific Pyruvate Dehydrogenase Complex (PDC) deficiency, by June 30, 2027. The study, which is in Phase 1 and currently recruiting participants, will involve administering Triheptanoin to patients to assess its efficacy and safety. The trial requires patients to attend up to 10 visits over two years, including blood draws. Triheptanoin will be provided at no cost to participants, with a target dose of 1.2-3.9 g per kg body weight. This trial is particularly significant as PDC deficiency is a rare genetic disorder, presenting a potential therapeutic option for affected families.
Clinical Trial CompletionThe clinical trial NCT05933200, sponsored by Ultragenyx Pharmaceutical Inc, is expected to complete on August 1, 2027. The trial compares triheptanoin with even-chain medium-chain triglycerides (MCT) in pediatric patients suffering from long-chain fatty acid oxidation disorders (LC-FAOD). Approximately 60 participants are enrolled in the study, which is currently in the recruiting phase and is designed to assess the efficacy of triheptanoin in enhancing metabolic control events (MCEs) and reducing liver fat fraction. Results could influence treatment methodologies for LC-FAOD, a group of rare genetic disorders affecting fatty acid metabolism.
Clinical Trial CompletionUltragenyx Pharmaceutical Inc is expected to complete a follow-up study of AAV-mediated gene transfer UX111 for MPS Type IIIA on August 1, 2027. This multicenter study, enrolling 41 patients, aims to monitor the long-term effects of UX111, which is a gene therapy previously administered in earlier clinical trials (NCT02716246 and NCT04088734). No investigational product will be provided in this follow-up, but selected participants may receive adjuvant immunomodulatory therapy as determined by clinical oversight. The study was transferred from Abeona Therapeutics, Inc. in August 2022, highlighting its ongoing significance in gene therapy for metabolic disorders.
Clinical Trial CompletionUltragenyx Pharmaceutical Inc is expected to complete its Phase 3 clinical trial of GTX-102 for pediatric patients with Angelman Syndrome by November 1, 2027. The trial, aimed at evaluating the drug's efficacy on cognitive function in subjects with deletion-type Angelman Syndrome, will enroll approximately 120 participants. Currently, the trial is in the recruiting phase, having commenced on December 3, 2024. This study's outcome could have significant implications for Ultragenyx's future product offerings in the rare genetic disorder space.
Clinical Trial CompletionUltragenyx Pharmaceutical Inc is expected to complete its clinical trial of Setrusumab in pediatric patients with Osteogenesis Imperfecta in Japan by January 1, 2028. The trial, which is currently active but not recruiting participants, began on October 25, 2024, and involves a 24-month treatment period followed by an extension phase. Setrusumab is aimed at treating this genetic bone disorder, potentially impacting the company's product pipeline and market presence in Japan. With only six participants enrolled, the trial's results could inform future commercialization plans for the drug in the region.
Expected Trial CompletionUltragenyx Pharmaceutical Inc. is expected to complete the Phase 3 clinical trial for DTX301, a gene therapy for patients with Ornithine Transcarbamylase (OTC) deficiency, on December 1, 2028. DTX301 aims to treat late-onset OTC deficiency in patients aged 12 and older through a randomized, double-blind, placebo-controlled study involving 50 participants. The trial includes a crossover design where patients initially receiving a placebo will switch to DTX301 after 64 weeks. Following this trial, participants will have the opportunity to enter a long-term Disease Monitoring Program. This therapy's successful development could significantly impact the treatment landscape for OTC deficiency, with potential commercial implications for Ultragenyx Pharmaceutical Inc.
Clinical Trial Completion