Design Therapeutics Outlines Progress Across GeneTAC Platform

Author: Happy Mohamed | March 19, 2024 04:02pm

New Drug Product for Friedreich Ataxia (FA) DT-216P2 with Favorable Nonclinical Pharmacokinetic and Injection Site Safety Profile; Complete GLP Studies by Year-end 2024 to Start Patient Trials in 2025
 

IND Cleared for DT-168 for Treatment of Fuchs Endothelial Corneal Dystrophy (FECD) with Phase 1 Development to Start in 2024; Observational Study Underway to Confirm Patient Characteristics and Evaluate Potential Endpoints

New Program Unveiled for Huntington's Disease (HD) Targeting Reduction of Mutant Huntingtin with a GeneTAC™ Small Molecule

Cash and Securities of $281.8 Million at Year-end 2023 Support Five-Year Operating Runway and Advancement of Up to Four Programs to Clinical Proof-of-Concept

Conference Call and Webcast to be Held Today at 4:30pm ET

CARLSBAD, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today provided an update on the progress of its platform of GeneTAC™ portfolio assets and reported fourth quarter and full year 2023 financial results.

"At Design, our vision is to develop a portfolio of first or best-in-class small molecules capable of treating a host of degenerative diseases by working with a patient's natural genome to restore cellular health. In recent months we have undertaken important work to evaluate the scope of our GeneTAC™ platform, which we believe has the potential to deliver clinical proof-of-concept in up to four programs, subject to research and development results, under our current cash runway through the next five years," said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics.

Dr. Shah continued, "Our lead program in FA has a new drug product, DT-216P2, that we have designed to have an improved pharmacokinetic and injection site safety profile, which positions us to resume clinical development for this debilitating, degenerative, neuro-muscular genetic disease. This work builds on encouraging data in FA patients from our previous clinical trial. In addition, our FECD program, DT-168, which has the potential to be the first effective treatment addressing the root cause of this degenerative corneal disease, now has an IND that was cleared by the FDA. Finally, we are highlighting an exciting new program for Huntington's Disease, an indication of tremendous unmet medical need, which further demonstrates the depth and versatility of our differentiated approach to genomic medicines."

Pipeline Updates and Anticipated Upcoming Milestones

• Friedreich Ataxia (FA) Design has developed a new drug product, DT-216P2, for patients with FA that demonstrates an improved pharmacokinetic (PK) profile and a favorable injection site safety profile in nonclinical studies. When compared to the prior formulation, DT-216P2 demonstrated greater than 10-fold exposures that are more sustained over time. DT-216P2 also appears suitable for intravenous or subcutaneous routes of administration.
  
  
  
  Design previously reported Phase 1 data using its prior formulation that showed increased levels of frataxin (FXN) mRNA in peripheral blood cells and skeletal muscle, confirming activity in patients with FA. Based on these findings, Design is advancing DT-216P2 for FA, with plans to complete GLP studies by year-end 2024 to start patient trials in 2025.

• Fuchs Endothelial Corneal Dystrophy (FECD) The U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for DT-168 in FECD. To evaluate the potential impact of DT-168 on this degenerative corneal disease, the company is conducting an observational study designed to confirm disease characteristics and progression and evaluate potential endpoints prior to initiating an interventional treatment trial. The observational study is expected to enroll 200 patients with a follow-up of two years. Design expects to initiate Phase 1 development for DT-168 in 2024.
• Huntington's Disease (HD) Design announced a new program in HD that targets allele-selective reduction of mutant huntingtin (HTT) gene expression with a GeneTAC™ small molecule. In preclinical studies, the company's HD GeneTAC™ candidate molecules selectively dial-down the expression of the mutant HTT gene by over 50% in the brain striatum with systemic administration. The company is working toward selection of a development candidate in anticipation of a future IND submission.
• Myotonic Dystrophy Type-1 (DM1) Design is also advancing its preclinical characterization of several lead molecules for the treatment of DM1, a multi-system genetic disorder. The company's DM1 GeneTAC™ small molecules potently dial-down the expression of the mutant DMPK gene in DM1 patient cells, eliminating foci and restoring normal splicing. Design is working toward selection of a development candidate in anticipation of a future IND submission.

Posted In: DSGN