Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, today announced the presentation of key findings from the Phase III trial of Sodium Dichloroacetate (DCA) for Pyruvate Dehydrogenase Complex Deficiency (PDCD) at the Mitochondrial Medicine 2025 conference in St. Louis.
“We are pleased to have Dr. Peter Stacpoole share the latest data from our clinical trial at the Mitochondrial Medicine conference,” said Dave Penake, CEO Saol Therapeutics. “We would like to thank patients, investigators and clinical trial sites who participated in the clinical trial. Our team is working with the FDA on the review of this product, with an FDA action date of August 27th, 2025.”
Key findings from the trial include:
- DCA was well-tolerated, even upon chronic administration for over three years. The most commonly reported adverse reaction occurring in >5% of patients was gastrointestinal disorders (n=2, 5.9%).
- The primary efficacy endpoint, the Observer Reported Outcome (ObsRO) survey, did not demonstrate statistical significance in the double-blind portion of the trial. Longer duration of therapy, including the open label extension (OLE), demonstrated a statistically significant improvement in motor function in the ITT population (p=0.002).
- DCA significantly (p=0.006) decreased plasma lactate concentrations.
- DCA treatment improved survival (p=0.027) compared to appropriately matched controls.
Saol Therapeutics, Dr. Peter Stacpoole, and the other investigators plan to submit the results of the Phase III clinical program for publication.
