Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

CARLSBAD, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc.. 

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. 

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.

Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity

Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity. Front. Pharmacol., 19 March 2024, Sec. Neuropharmacology, Volume 15, 2024, doi:10.3389/fphar.2024.1342965   

Real-life activity monitoring is feasible and well tolerated by patients. Accelerometer-based measures can quantify disease progression in FRDA over 1 year, providing objective information about patient’s motor activities and supporting the usefulness of these data as complementary outcome measure in interventional trials.