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Design Therapeutics Announces FDA Clearance of Investigational New Drug Application for First GeneTAC™ Molecule for Friedreich Ataxia
Phase 1 Trial of DT-216 in Patients with Friedreich Ataxia to Be Initiated Soon Topline Data from Phase 1 Trial Expected in the Second Half of 2022 CARLSBAD,
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Phase 1 Trial of DT-216 in Patients with Friedreich Ataxia to Be Initiated Soon Topline Data from Phase 1 Trial Expected in the Second Half of 2022 CARLSBAD, Calif., Feb. 28, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today announced that its Investigational New Drug Application (IND) for its lead candidate, DT-216, for the treatment of Friedreich ataxia (FA), was cleared by the U.S. Food and Drug Administration (FDA). The company is preparing to initiate a Phase 1 clinical trial of DT-216 to assess the safety, tolerability, pharmacokinetics, and frataxin (FXN) levels in patients with FA. Study enrollment is expected to begin in the coming weeks. FA is a devastating multisystem degenerative disease caused by a mutation of the GAA repeat expansion of the FXN gene that impairs transcription and reduces gene expression. Low FXN levels result in all FA disease manifestations, including neurological deficits such as loss of balance and coordination, cardiomyopathy and arrhythmias, as well as diabetes and other serious symptoms. DT-216 is a GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation and restore FXN gene expression. “The IND clearance for DT-216 is an important milestone for the company, further validating the therapeutic potential of our GeneTAC™ platform, a new class of medicines being developed for serious degenerative diseases caused by a single gene defect. GeneTAC™ molecules, designed based on decades of pioneering research into the mechanisms of gene transcription, are capable of dialing up or dialing down the expression of disease-causing genes and therefore have the potential to address the root cause of genetic diseases,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “DT-216 is the first in a pipeline of GeneTAC™ molecules, which can be designed and selected for desirable pharmaceutical properties such as tolerability, broad tissue distribution, including the CNS, efficient manufacturing, and convenient administration. We are eager to begin clinical development of DT-216 for people with FA, for whom there are no disease-modifying treatments available today.” Design’s IND was supported by a comprehensive package of...