Benzinga | Dec 1, 2021 04:34PM EST

Ultragenyx, GeneTx Report Will Participate In FAST Global Summit, Gala Dec. 4

GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), companies partnered in the development of GTX-102, today announced multiple presentations at the 2021 FAST Global Summit & Gala, highlighting additional supportive data from the initial five patients treated in the Phase 1/2 study of GTX-102, an investigational treatment for Angelman syndrome. The hybrid event will take place December 2-4 with additional information available at the FAST Global Summit & Gala website.

Details for the presentations are as follows:

* Translational Research in a Large Animal Model of Angelman Syndrome Friday, December 3, 2:10 -- 2:35 PM CT Scott Dindot, Ph.D. Associate Professor of Genomics and EDGES Fellow at Texas A&M University and Executive Director of Molecular Genetics at Ultragenyx Pharmaceutical

* Development of Rare Disease Therapies: Overcoming Challenges Saturday, December 4, 9:35 -- 9:55 AM CT Emil Kakkis, M.D., Ph.D. CEO of Ultragenyx Pharmaceutical Inc.

* GTX-102 Phase 1/2 Clinical Trial Update, Development of an ASO for Angelman Syndrome Saturday, December 4, 10:25 -- 10:45 AM CT Scott Stromatt, M.D. Chief Medical Officer at GeneTx Biotherapeutics Elizabeth Berry-Kravis, M.D., Ph.D. Professor of Pediatrics, Neurological Sciences, and Biochemistry at Rush University Medical Center in Chicago

Initial results from the originally treated five patients in the United States were first presented at the 2020 FAST Global Summit. The new, supportive data for these patients are from two exploratory efficacy endpoints:

* Vineland-3 Adaptive Behavior Scales: a semi-structured interview administered by a clinician to the caregiver to assess adaptive behavior including communication, daily living skills, socialization, and motor skills in individuals with intellectual and developmental disabilities. The Vineland-3 is completed at baseline, at last dose, and at one or more later time points during the follow-up period.

* Electroencephalogram (EEG) Readings: additional quantitative analysis of EEG findings in four of the five patients to assess delta waves and delta power, which are abnormal in patients with Angelman syndrome at baseline.

The companies recently began enrolling and dosing treatment-naive patients in an amended protocol of the Phase 1/2 study. A status update highlighting additional progress with the amended study will be provided around year-end 2021.