Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting

Company will host investor call on April 17 at 8:00 a.m. ET to discuss new data from GTX-102 Phase 1/2 clinical study in patients with Angelman syndrome

NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that preclinical and clinical data, including new clinical efficacy and safety data from the expansion cohorts in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome, will be presented at the 76th Annual American Academy of Neurology Meeting (AAN) being held April 13-18 in Denver and virtually. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).

Abstracts are available to view online at www.aan.com/events/annual-meeting-abstracts. The GTX-102 emerging science presentation will include 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 (abstract #192, presentation #008)
Session: Emerging Science 2
Presenter: Kemi A. Olugemo, M.D., FAAN, Ultragenyx
Date/ Time: Tuesday, April 16, 6:12-6:18 p.m. MDT

Title: Proteomic Profiling of Angelman Syndrome for Disease-Associated Biomarker Discovery
Format: Poster (#8.001)
Session: Child Neurology and Developmental Neurology: Neurogenetics 2  
Presenter: Rachael Elizabeth Hawtin, Ph.D., Ultragenyx
Date/ Time: Tuesday, April 16, 5:30-6:30 p.m. MDT

Title: Clinical Characteristics 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, M.D., Ultragenyx
Date/ Time: Tuesday, April 16, 5:30-6:30 p.m. MDT

Conference Call and Webcast Information
Ultragenyx will host a conference call at 8:00 a.m. ET on Wednesday, April 17, 2024, to discuss the new efficacy and safety data from the GTX-102 Phase 1/2 clinical study being presented at the conference.

The live and replayed webcast of the call will be available through the company’s website at https://ir.ultragenyx.com/events-presentations. To participate in the live call, please register by clicking on the following link (registration link) and you will be provided with dial-in details.