Saol Therapeutics Submits NDA for SL1009 to U.S. FDA

Saol Therapeutics Makes Significant Progress in Developing Treatment for Rare Pediatric Disease

ROSWELL, Ga. and DUBLIN and HAMILTON, Bermuda, Dec. 3, 2024 – Saol Therapeutics, a clinical-stage pharmaceutical company dedicated to developing treatments for rare and orphan diseases, has announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for SL1009. This Sodium Dichloroacetate Oral Solution (DCA) is targeted for the treatment of Pyruvate Dehydrogenase Complex Deficiency (PDCD), a rare pediatric mitochondrial disorder, utilizing a proprietary genetic test. SL1009 has been awarded several designations by the FDA, including Orphan Drug, Fast Track, and Rare Pediatric Disease, making it eligible for Priority Review and a Priority Review Voucher.

Dave Penake, Chief Executive Officer of Saol Therapeutics, emphasized the importance of this NDA submission, stating, "This submission represents a crucial step toward providing a treatment option for children with PDCD in the U.S., where no approved therapies currently exist. We are eager to continue our ongoing dialogue with the FDA to address the significant needs of children affected by PDCD."

The NDA for SL1009 is supported by comprehensive data from two key studies. The first, a Phase 3 double-blind placebo-controlled crossover study (SL1009-01), assessed the drug's efficacy and safety. The primary endpoint was measured using a daily Observer Reported Outcomes (ObsROmotor) survey to evaluate changes in motor functions, while a key secondary endpoint measured the reduction in plasma lactate levels as compared to placebo. The patients were dose-stratified post-randomization based on their GSTZ1 genotype, identified through a proprietary genetic test designed to minimize adverse events like peripheral neuropathy.

The second study, SL1009-02, was a survival analysis comparing outcomes of DCA-treated patients from the Phase 3 study with an external cohort of untreated PDCD patients matched by age and sex. The objective was to evaluate the long-term survival benefit of the treatment.

Peter Stacpoole, MD, the initial study sponsor for SL1009-01, expressed his gratitude for the clinicians and families who participated in the trials, stating, "The NDA submission marks a monumental milestone, reflecting years of dedicated effort."

Saol Therapeutics plans to present detailed data from these studies at an upcoming medical conference, underlining the potential impact of SL1009 for PDCD patients.

SL1009, if approved, would be the first FDA-sanctioned treatment for PDCD available as an oral solution. PDCD is caused by genetic mutations in the Pyruvate Dehydrogenase Complex (PDC), leading to its inhibition by pyruvate dehydrogenase kinases (PDK). DCA works by inhibiting PDK, thereby stimulating residual PDC activity and enhancing mitochondrial energy production. The dosing regimen is personalized based on a genetic test that categorizes patients as "fast" or "slow" metabolizers, aiming for optimal efficacy and safety.

PDCD is a disorder that impairs carbohydrate oxidation, primarily affecting the nervous system and skeletal muscles, resulting in decreased ATP production and energy failure. The condition is associated with symptoms such as lethargy, poor feeding, rapid breathing, and various neurological and neuromuscular dysfunctions. It is estimated that in the U.S., 300-500 patients receive treatment at expert centers, with the overall prevalence potentially reaching up to 2,000. Currently, there are no FDA-approved treatments for PDCD.

About Saol Therapeutics
Saol Therapeutics is a private pharmaceutical company with operations in Roswell, GA, Dublin, Ireland, and Hamilton, Bermuda. The company focuses on developing therapies for central nervous system disorders and rare diseases, aiming to provide innovative treatment options for patients and healthcare providers.

For further details, Saol Therapeutics continues to pursue advancements in therapeutic solutions to meet the needs of patients with rare and challenging conditions.