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Design Therapeutics to Present Preclinical Data Highlighting the Potential of its GeneTAC™ Small Molecule, DT-216, for the Treatment of Friedreich Ataxia at ICAR 2022
Company On-track to Report Initial Data from Friedreich Ataxia Phase 1 Trial of DT-216 in the Fourth Quarter of 2022 CARLSBAD, Calif., Oct. 17, 2022 (GLOBE
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Company On-track to Report Initial Data from Friedreich Ataxia Phase 1 Trial of DT-216 in the Fourth Quarter of 2022\nCARLSBAD, Calif., Oct. 17, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that preclinical data for the company’s novel GeneTAC™ small molecule, DT-216, as a potential treatment for Friedreich ataxia (FA) will be presented during an oral session at the International Congress for Ataxia Research (ICAR) 2022. The data were included in the company’s Investigational New Drug (IND) application for DT-216, which is currently being evaluated in a Phase 1 clinical trial. The conference will be held in Dallas, Texas from November 1-4, 2022. FA is a devastating multisystem degenerative disease caused by a mutation characterized by a GAA repeat expansion in the frataxin (FXN) gene that impairs FXN transcription and reduces gene expression. Reduced FXN transcription results in mitochondrial and cellular dysfunction and leads to all FA disease manifestations, including neurological deficits such as loss of balance and coordination, cardiomyopathy, arrhythmias, diabetes and other serious symptoms. DT-216 is a GeneTAC™ small molecule designed to specifically target the GAA repeat expansion mutation and restore endogenous FXN transcription. The preclinical data support the potential for DT-216 to restore FXN gene expression, improve mitochondrial function and address the root cause of FA. Key findings of the presentation include: DT-216 dose-dependently increased FXN in peripheral white blood cells from multiple FA donors and in multiple FA patient cell models Administration of DT-216 increased FXN mRNA by approximately 10-fold in peripheral blood mononuclear cells (PBMCs) collected directly from FA patients (N=23 donors with >100 to > 1500 GAA repeats)Administration of DT-216 at ~ 10nM, the 90% maximal effective concentration (EC90) for the molecule, for 14 days in FA patient-derived neurons restored FXN protein to levels comparable to non-FA neurons DT-216 improved mitochondrial respiration in FA B-lymphoblastoid cells and patient-derived cardiomyocytes as measured using a Seahorse XFp Analyzer Design is currently evaluating DT-216 in a Phase 1 clinical trial in adult patients with FA. The...