Saol Therapeutics Announces Major Clinical Milestones Across Multiple Development Programs

SL1009 targets a 2025 FDA decision for Pyruvate Dehydrogenase Complex Deficiency (PDCD), while SL1002 advances toward Phase III trials for osteoarthritis knee pain and spasticity

ROSWELL, Ga. and DUBLIN and HAMILTON, Bermuda, April 17, 2025 /PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, today announced multiple updates on its rapidly advancing clinical development portfolio.

SL1009, Sodium Dichloroacetate (DCA)
DCA is an investigational product, that if approved, will be used with a proprietary dose-related genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). PDCD is a rare and life-threatening genetic disorder that causes 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 recently notified Saol of a 90-day extension to the NDA review, moving the PDUFA date from May 27, 2025, to August 27, 2025. No concerns were raised by the FDA about the adequacy of the filing or the need for an advisory committee meeting. Saol, in collaboration with Medosome Biotec, also filed the Humanitarian Device Exemption (HDE) application for the dose-related genetic test that will serve as a required companion diagnostic for patients treated with DCA.

"We are working with the FDA to secure approval for this product this year," said Dave Penake, CEO. "Our entire organization is united in the mission to bring hope to patients and families who have been waiting for this breakthrough therapy."

For PDCD patients who did not participate in the clinical trial, Saol is now advancing an expanded access program (EAP). More information can be found at Clinicaltrials.gov, NCT06931262.

Further, Saol is preparing for a Type C meeting with the FDA to discuss the clinical development program for the use of DCA in the treatment of congenital lactic acidosis (CLA). Congenital lactic acidosis is caused by inborn errors of metabolism, including PDCD, often presenting early in life, leading to lactic acid accumulation, with high mortality.

SL1002
SL1002 is an investigational, novel perineural chemoneurolytic injection that utilizes Saol's proprietary CYCLOPHLEX™ technology. It is currently being advanced in development for the treatment of chronic knee pain related to osteoarthritis and for limb spasticity, both in the adult population (18+).

"SL1002 continues to be a program with enormous potential," said Penake. "Our recent FDA End of Phase II meeting provided a clear regulatory path for our osteoarthritis pain program. We are eager to initiate our pivotal program this year. Meanwhile, our spasticity data was recently presented by our investigators and received positive feedback from thought leaders in the field. As we further explore SL1002's therapeutic potential, it is becoming increasingly clear that this program has far-reaching possibilities to treat patients with a variety of neuromuscular disorders."

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
[email protected]

SOURCE Saol Therapeutics