Saol Therapeutics announced that the pivotal Phase 3 clinical trial evaluating Sodium Dichloroacetate for the treatment of a rare, pediatric, ultra-orphan mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency has achieved its targeted enrollment.
ROSWELL, Ga. and DUBLIN and HAMILTON, Bermuda, Oct. 4, 2022 /PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the pivotal Phase 3 clinical trial evaluating Sodium Dichloroacetate (DCA) for the treatment of a rare, pediatric, ultra-orphan mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD) has achieved its targeted enrollment. Saol is one of the trial collaborators, with topline results anticipated early in the second half of 2023.
“There are many challenges to overcome when enrolling patients in clinical trials for rare diseases”, said Saol Therapeutics CEO David Penake. “The hard work of the individual study sites, patient advocacy groups, genetic testing companies, and genetic counseling partners allowed us to exceed our enrollment target of 30 patients. Our team will now prepare for a rapid NDA submission should the treatment prove to be successful.”
This investigator-initiated, NIH and FDA funded trial, conducted in close partnership with University of Florida, Saol Therapeutics and Medosome Biotec, represents the culmination of years of work for Dr. Peter Stacpoole, Principal Investigator and Professor of Medicine at University of Florida.
“I began working on a treatment for PDCD in the 1980s after a family in Connecticut contacted me to see if I could help their child, so it is extremely rewarding to finally reach this milestone”, said Stacpoole.
Philip E. Yeske, Ph.D., Science & Alliance Officer at United Mitochondrial Disease Foundation, commented “The mitochondrial disease patient community is deeply appreciative of the time, effort, and capital that Saol and its collaborators are investing into this project. An over-enrolled clinical trial is a testament to the desire of the PDCD patient families to play an active role in therapeutic development.”
Study Design
This trial evaluates daily administration of DCA in children with a confirmed pathological mutation in the Pyruvate Dehydrogenase Complex or in a pyruvate dehydrogenase phosphatase gene. The primary efficacy outcome measure is based on a novel Observer Reported Outcome (ObsRO) survey developed specifically for this trial.
The study design is a 9-month double-blind crossover comparison between DCA and placebo, during which the ObsRO is completed daily by a parent/caregiver, followed by an open label phase of DCA administration.
More information on the clinical trial and participating institutions can be found at Phase 3 PDCD Trial.
About DCA (SL-1009)
DCA has the potential to be the 1st approved medication for the mitochondrial disease PDCD and will be available as an oral solution. Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. However, PDC is also inhibited by pyruvate dehydrogenase kinases (PDK), that may be over-expressed in PDCD. DCA inhibits PDKs to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. DCA dosing is based on a proprietary genetic test that dichotomizes subjects into “fast” and “slow” drug metabolizers, providing individualized dosing.
About Pyruvate Dehydrogenase Complex Deficiency (PDCD)
PDCD is a mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure. It affects only a few hundred individuals in the U.S. It is a common cause of congenital lactic acidosis, a life-threatening condition that may occur as early as the neonatal period. Patients may also exhibit extreme tiredness (lethargy), poor feeding, and rapid breathing (tachypnea)and other signs of neurological and neuromuscular dysfunction, such as developmental delay, low muscle tone (hypotonia), abnormal eye movements and seizures. Signs and symptoms usually begin soon after birth but may appear later in childhood.
There are currently no FDA-approved therapies for PDCD.
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.
Medosome Biotec, LLC is a privately held, preclinical and early-stage clinical pharmaceutical company with operations in Alachua, FL. The Company focuses on pediatric diseases with an emphasis on developing and offering genetic tests for diagnosing rare diseases and providing personalized dosing of pharmaceutical drugs. For more information, visit www.mdbiotec.com
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