Treatment with setrusumab (UX143) for at least 6 months resulted in 67% reduction in annualized fracture rate in patients with OI in Phase 2/3 Orbit study
Quantitative data from the Phase 1/2 study of GTX-102 for AS show clinically meaningful improvements in multiple domains as compared to natural history
4 of 5 patients in lowest-dose cohort of Phase 1/2/3 study of UX701 in Wilson disease are tapering off of chelators and/or zinc therapy, including 2 that are now completely off standard therapy
Ultragenyx Analyst Day live webcast available today at
“The data we are presenting today show that these investigational therapies are having meaningful clinical effects on difficult diseases with limited or no approved treatments and are potentially transformative for people living with these diseases if proven safe and effective in Phase 3 studies,” said
Analyst
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Interim Phase 2 data from the Phase 2/3 Orbit study show statistically significant decrease in annualized fracture rates following at least 6 months of treatment
- Data presented at the
American Society for Bone and Mineral Research 2023 Annual Meeting (ASBMR) show that treatment with setrusumab reduced the annualized fracture rate by 67% and this reduction was associated with continuing large and meaningful improvements in bone mineral density (BMD). - Setrusumab was generally well tolerated with no drug related serious adverse events (SAEs) reported and no reports of drug-related hypersensitivity.
- The company plans to provide updated Phase 2 data next year.
GTX-102 antisense oligonucleotide for Angelman syndrome: Data from the extension cohorts in the Phase 1/2 study show clinically meaningful improvements in multiple domains
- Quantitative data show improvements across multiple clinical domains compared to natural history data, where available, and clinical changes were associated with quantitative changes in EEG.
- Long term data showed patients who stopped and restarted treatment reacquired previously gained developmental skills when they were re-dosed with the current regimen.
- There have been no additional treatment-related SAEs, including lower extremity weakness, since
November 2022 . - Data from the dose expansion cohorts on at least 20 patients who have been on therapy for at least 6 months is anticipated in the first half of 2024.
UX701 AAV gene therapy for Wilson disease: Four of five patients in the lowest-dose cohort of the Phase 1/2/3 Cypress2+ study show improvements in tapering standard of care
- Four out of 5 patients in the low-dose Cohort 1 have had reductions in urinary copper and are tapering off of chelators and/or zinc therapy, including 2 of 3 earlier treated patients in the Cohort that are now completely off standard therapy.
- UX701 has been generally well tolerated with no treatment-related SAEs.
- The seamless study is expected to complete dosing of all 3 dose cohorts in Stage 1 at the end of 2023 and these data are expected in the first half of 2024.
Company also provided update on other late-stage gene therapy candidates
- DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): The Phase 3 GlucoGene study was fully enrolled in the first quarter of 2023 and the company plans to provide preliminary data in the first half of 2024.
- UX111 for Sanfilippo syndrome (MPS IIIA): The pivotal Transpher A study has been fully enrolled and the company plans to meet with the FDA in the fourth quarter of 2023.
- DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: The Phase 3 Enh3ance study is expected to complete enrollment in the first half of 2024.
Analyst Day and Webcast Information
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About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit ultragenyx.com.
Ultragenyx Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, business plans and objectives for UX143, expectations regarding the clinical benefit, tolerability and safety of UX143, future clinical and regulatory developments for UX143, the clinical benefit, tolerability and safety of GTX-102, future clinical and regulatory developments for GTX-102, the clinical benefit, tolerability and safety of UX701, future clinical and regulatory developments for UX701, timing for enrollment, dosing and data for Ultragenyx’s investigational therapies and gene therapy candidates and regulatory meetings are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company and
In addition to its
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