~ Saol Therapeutics, a clinical-stage pharmaceutical company, made a significant announcement at the Mitochondrial Medicine 2025 conference in St. Louis today. The company shared key findings from their Phase III trial of Sodium Dichloroacetate (DCA) for Pyruvate Dehydrogenase Complex Deficiency (PDCD).

According to Saol Therapeutics CEO Dave Penake, the company is pleased to have Dr. Peter Stacpoole present the latest data from their clinical trial at the conference. He also expressed gratitude towards the patients, investigators, and clinical trial sites who participated in the study. Penake added that Saol's team is currently working with the FDA on reviewing DCA and has an FDA action date of August 27th, 2025.

The key findings presented from the Phase III trial include:

- DCA was well-tolerated by patients, even after being administered for over three years. The most commonly reported adverse reaction was gastrointestinal disorders.

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- The primary efficacy endpoint, Observer Reported Outcome (ObsRO) survey, did not show statistical significance in the double-blind portion of the trial. However, longer duration of therapy in the open label extension (OLE) demonstrated a significant improvement in motor function.

- DCA significantly decreased plasma lactate concentrations.

- Treatment with DCA showed improved survival compared to matched controls.

Saol Therapeutics, along with Dr. Peter Stacpoole and other investigators involved in the study, plan to submit their Phase III clinical program results for publication.

Sodium Dichloroacetate (DCA), also known as SL1009, is an investigational product that could potentially be used with a proprietary dose-determining genetic test to treat PDCD - a rare and life-threatening genetic disorder that affects children. PDCD can lead to chronic energy deficit, lactic acidosis, developmental problems and early childhood death.

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DCA has received Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, making it eligible for Priority Review and a Priority Review Voucher. The FDA action or PDUFA date for DCA is August 27, 2025. Saol Therapeutics, in collaboration with Medosome Biotec, has also filed a Humanitarian Device Exemption (HDE) application for the dose-determining genetic test that will be used as a companion diagnostic for patients treated with DCA.

For PDCD patients who were not part of the clinical trial, Saol Therapeutics is currently conducting an expanded access program (EAP) for eligible individuals. More information about this program can be found on Clinicaltrials.gov under NCT06931262.

In conclusion, Saol Therapeutics' presentation of key findings from their Phase III trial of DCA for PDCD at the Mitochondrial Medicine 2025 conference has provided valuable insights into the potential benefits of this investigational product. With promising results and various designations from the FDA, DCA could potentially become a much-needed treatment option for children with PDCD.

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