Saol Therapeutics Announces Poster Presentation at the UMDF Mitochondrial Medicine 2025 Conference
Key findings from the Phase III trial of Sodium Dichloroacetate (DCA) for Pyruvate Dehydrogenase Complex Deficiency (PDCD) were presented.
ROSWELL, Ga. and DUBLIN and HAMILTON, Bermuda, June 19, 2025 /PRNewswire/ -- 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.
SL1009, Sodium Dichloroacetate (DCA)
DCA is an investigational product that, if approved, will be used with a proprietary dose-determining genetic test to treat an orphan pediatric-onset mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). PDCD is a rare and life-threatening genetic disorder that can cause chronic energy deficit leading to lactic acidosis, profound developmental problems and early childhood death.
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 is August 27, 2025. Saol, in collaboration with Medosome Biotec, also filed the Humanitarian Device Exemption (HDE) application for the dose-determining genetic test that will serve as a required companion diagnostic for patients treated with DCA.
For PDCD patients who did not participate in the clinical trial, Saol is now conducting an expanded access program (EAP) for eligible PDCD patients. More information can be found at Clinicaltrials.gov, NCT06931262.
About Saol Therapeutics
Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland, and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit www.saolrx.com.
Saol Therapeutics Contact
Senior Vice President, Strategy
Brian Nappi
bnappi@saolrx.com
ST-P207-3011-01
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SOURCE Saol Therapeutics