#United States #St. Louis #Saol Therapeutics #PDDC #DCA

Saol Therapeutics Updates on Sodium Dichloroacetate

Saol Therapeutics, a clinical-stage pharmaceutical company, recently announced significant findings from its Phase III trial of Sodium Dichloroacetate (DCA) aimed at treating Pyruvate Dehydrogenase Complex Deficiency (PDCD). The crucial data was presented at the Mitochondrial Medicine 2025 Conference held in St. Louis, Missouri. CEO Dave Penake expressed gratitude toward the patients, investigators, and trial sites that contributed to the successful results.

Phase III Trial Results

The pivotal trial revealed several important findings concerning the safety and efficacy of DCA:

• - Tolerability: DCA was found to be well-tolerated over a chronic administration period exceeding three years. The most frequently noted side effect was gastrointestinal disorders, reported in about 5.9% of patients.
• - Efficacy Endpoint: The primary efficacy endpoint—an Observer Reported Outcome (ObsRO) survey—did not achieve statistical significance during the double-blind phase. However, an observable trend of improvement in motor function was noted with extended therapy, showing significant results in the open-label extension.
• - Plasma Lactate Levels: DCA treatment led to a significant reduction in plasma lactate concentrations, with a p-value of 0.006.
• - Improved Survival Rates: Results indicated that DCA administration improved survival rates compared to appropriately matched control groups, with a p-value of 0.027.

These pivotal findings shed light on the beneficial effects DCA may have in slowing the progression of PDCD, a rare genetic disorder causing chronic energy deficits and associated complications in children.

Regulatory Progress

The company is actively collaborating with the FDA as it prepares for a review of DCA. This includes a PDUFA action date set for August 27, 2025. DCA also qualifies for various designations such as Fast Track, Orphan Drug, and Rare Pediatric Disease, enhancing its potential for expedited approval.

Moreover, in conjunction with Medosome Biotec, Saol Therapeutics has filed for a Humanitarian Device Exemption that pertains to the genetic test necessary for determining the correct dosage of DCA for patients suffering from PDCD.

An expanded access program is currently underway, enabling eligible PDCD patients who didn't participate in the clinical trial to access treatment under specific conditions. Interested patients can find more information through ClinicalTrials.gov, with the trial identifier NCT06931262.

About Saol Therapeutics

Founded with the mission of addressing unmet medical needs, Saol Therapeutics operates with clinical development centers in Roswell, Georgia, Dublin, Ireland, and Hamilton, Bermuda. Their focus includes CNS disorders and orphan diseases such as PDCD. The company’s commitment seeks to empower patients and healthcare providers through innovative treatment options.

For more information on the company and its initiatives, visit www.saolrx.com.