The company will hold an investor conference call on April 17 at 8 a.m. ET to discuss new data from its Phase 1/2 clinical trial of GTX-102 in Angelman syndrome patients. To do.
NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced new clinical efficacy and safety data from expanded cohort of Phase 1/2 study Presented preclinical and clinical data. Research findings on GTX-102 for the treatment of Angelman syndrome will be presented virtually at the 76th American Academy of Neurology (AAN) meeting in Denver, April 13-18. GTX-102 is an investigational antisense oligonucleotide delivered by intrathecal administration; UBE3A antisense transcript (UBE3A-AS).
Abstracts can be viewed online at www.aan.com/events/annual-meeting-abstracts. The GTX-102 Emerging Science presentation includes data beyond what is currently available online.
AAN 2024 presentation details:
title: Clinical activity and safety of GTX-102, an investigational antisense oligonucleotide for the treatment of patients with Angelman syndrome.
format: Oral presentation (Abstract #192, Presentation #008)
session: emerging science 2
Presenter: Kemi A. Orgemo, MD, FAAN, Ultragenyx
Date Time: Tuesday, April 16th, 6:12pm – 6:18pm PDT
title: Proteomic profiling of Angelman syndrome for disease-related biomarker discovery
format: Poster (#8.001)
session: Child Neurology and Developmental Neurology: Neurogenetics 2
Presenter: Dr. Rachel Elizabeth Hawtin, Ultragenyx
Date Time: Tuesday, April 16th, 5:30pm-6:30pm PDT
title: Clinical features of creatine transporter deficiency (CTD): final results of the Vigilan observational study
format: Poster (#8.002)
session: Child Neurology and Developmental Neurology: Neurogenetics 2
Presenter: Melanie Brandabur, MD, Ultragenyx
Date Time: Tuesday, April 16th, 5:30pm-6:30pm PDT
Conference call and webcast information
Ultragenyx will host a conference call on Wednesday, April 17, 2024 at 8:00 a.m. ET to discuss new efficacy and safety data from its Phase 1/2 clinical trial of GTX-102 being presented at the conference. will be discussed.
A live and replayed webcast of the call will be available on the company's website at https://ir.ultragenyx.com/events-presentations. To join the live call, please register by clicking on the link below (registration link). Dial-in details will be provided.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing new products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company aims to address diseases with high unmet medical need and distinct biology for treatment, where there is typically no approved therapy to treat the underlying disease. We have built a diverse portfolio of approved treatments and product candidates.
The company is led by a management team with experience in the development and commercialization of rare disease drugs. Ultragenyx's strategy is premised on time- and cost-effective drug development, with the goal of bringing safe and effective treatments to patients with the utmost urgency.
For more information about Ultragenyx, please visit the company's website at www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Other than the historical information contained herein, the matters discussed in this press release contain forward-looking statements regarding future operating and financial performance, GTX-102's business plans and objectives, and expectations regarding the tolerability and safety of GTX-102. Contains statements regarding Ultragenyx's expectations and projections. GTX-102, and future clinical and regulatory developments of GTX-102, 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 significant risks and uncertainties. This may cause our clinical development programs, collaborations with third parties, and future results, performance and achievements to differ materially from those expressed or implied by the forward-looking statements. Such risks and uncertainties include the uncertainties of clinical drug development, the unpredictability and lengthy process of obtaining regulatory approval, the Company's ability to successfully develop GTX-102, and expectations regarding This includes the company's ability to achieve its expected development goals within the expected time period. , risks related to adverse side effects, risks related to dependence on third party partners to perform certain activities on the Company's behalf, market opportunities for the Company's products and product candidates being smaller than expected, manufacturing risks, other treatments; the sufficiency of existing cash, cash equivalents and short-term investments, the Company's future operating and financial performance, the timing of clinical trial activities and reporting of results therefrom, and the Ultragenyx products; matters that may affect the availability or commercial potential of new drug candidates; Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
For a more detailed description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements and risks associated with Ultragenyx's business generally, see Ultragenyx's Form filed in the United States. See 10-K Annual Report. Periodic reports filed with the Securities and Exchange Commission (SEC) on February 21, 2024 and thereafter filed with the SEC.
In addition to SEC filings, press releases, and public conference calls, Ultragenyx uses its investor relations website and social media to publicly disclose important information about the company, including information that may be important to investors. We comply with legal disclosure obligations. Regulation FD. Financial and other information about Ultragenyx is posted periodically and is available on Ultragenyx's Investor Relations website (https://ir.ultragenyx.com/) and the LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).
contact address
Ultragenics Pharmaceutical Co., Ltd.
Investor
Higa Joshua
+1-415-475-6370
ir@ultragenyx.com
media
carolyn wang
+1-415-225-5050
media@ultragenyx.com