Saol Therapeutics Shares Exciting DCA Trial Results at Conference
Saol Therapeutics Presents Key Findings on Sodium Dichloroacetate
Saol Therapeutics, a clinical-stage pharmaceutical company, has shared notable findings from its Phase III trial of Sodium Dichloroacetate (DCA) aimed at treating Pyruvate Dehydrogenase Complex Deficiency (PDCD). This presentation occurred during the Mitochondrial Medicine 2025 conference, showcasing the collaborative efforts of various stakeholders in the clinical trial process.
Insights from the Phase III Trial
During the presentation, CEO of Saol Therapeutics, Dave Penake, expressed gratitude towards patients, investigators, and trial sites for their invaluable contributions. The trial aimed to explore Sodium Dichloroacetate's effectiveness in treating PDCD, a rare and serious genetic disorder.
Adverse Reactions and Tolerability
One of the key outcomes indicated that DCA was well-tolerated by participants over an extended period, with a treatment duration exceeding three years. Gastrointestinal disorders were the most frequently reported adverse reactions, affecting 5.9% of the participants.
Assessing Efficacy Through Observer-Reported Outcomes
While the primary efficacy endpoint—the Observer Reported Outcome (ObsRO) survey—did not yield statistically significant results in the double-blind portion of the trial, there was a significant improvement in motor function during the open-label extension of the trial. This finding highlighted the long-term benefits of continued treatment.
Statistical Improvements Observed
Notably, DCA treatment led to a significant reduction in plasma lactate levels, coupled with a commendable improvement in survival rates compared to matched control groups. These statistics underscore the therapeutic potential of DCA for patients suffering from PDCD.
Future Directions for Saol Therapeutics
Looking ahead, Saol Therapeutics, along with Dr. Peter Stacpoole and other investigators, plans to submit the findings for publication, contributing to the growing body of knowledge regarding DCA's role in treating mitochondrial diseases.
DCA and Its Significance
DCA is an investigational product that could become pivotal in treating PDCD if approved. It works in conjunction with a genetic test designed to guide dosing, tailoring treatment to meet the unique needs of each patient. PDCD can lead to severe energy deficiencies causing serious complications, including developmental delays and life-threatening situations.
Fast Track and Collaborative Efforts
DCA has received important designations from the FDA, including Fast Track Designation and Orphan Drug Designation, ensuring it is eligible for Priority Review. Additionally, Saol Therapeutics, collaborating with Medosome Biotec, has applied for a Humanitarian Device Exemption (HDE) for the genetic test that will serve as a critical companion diagnostic for DCA treatment.
Expanded Access Program for PDCD Patients
For those patients with PDCD who did not participate in the clinical trial, Saol is actively conducting an expanded access program (EAP) to provide treatment options. This initiative aims to ensure eligible patients can access this potentially life-changing treatment, emphasizing Saol's commitment to patient care.
About Saol Therapeutics
Saol Therapeutics operates with a dedication to addressing central nervous system disorders and orphan diseases. With facilities in multiple locations, including Roswell, GA, Dublin, and Hamilton, Bermuda, the company is focused on improving therapeutic options for patients and their healthcare providers. They strive for innovation while ensuring the safety and efficacy of their products.
Saol Therapeutics Contact
For inquiries, Brian Nappi serves as the Senior Vice President of Strategy at Saol Therapeutics.
Frequently Asked Questions
What is Sodium Dichloroacetate used for?
Sodium Dichloroacetate is being investigated as a treatment for Pyruvate Dehydrogenase Complex Deficiency, a rare genetic disorder.
How long did the Phase III trial last?
The Phase III trial involved chronic administration of DCA for over three years, assessing its long-term efficacy and safety.
What were the main findings of the trial?
The trial found DCA to be well-tolerated, led to significant improvements in motor function, reduced plasma lactate levels, and improved survival rates.
What designations has DCA received from the FDA?
DCA has received Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation for its potential therapeutic uses.
How can patients access DCA if they didn't participate in the trial?
Saol Therapeutics has initiated an expanded access program for eligible PDCD patients who were not part of the clinical trial, ensuring they can access potential treatment options.
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