Relief Therapeutics with Acer Therapeutics Claims Acer-001 To Be A Potential Alternative For The Treatment Of Urea Cycle Disorders at GDMI Conference 2022
The two companies presented a poster presentation at the recent GMDI Conference.
Acer Therapeutics and its collaboration partner, Relief Therapeutics, today announced the presentation of data evaluating the bioavailability, bioequivalence and taste attributes of ACER-001 (sodium phenylbutyrate) compared to sodium phenylbutyrate (BUPHENYL®) powder during poster sessions at the recent Genetic Metabolic Dieticians International (GMDI) Conference on May 5, 2022, in Las Vegas, Nevada.
The study was conducted to identify and quantify the intensity of perceived flavor attributes of ACER-001 relative to NaPBA powder in two taste assessment studies that enrolled trained healthy panelists. The companies in their poster presentation says, ‘ACER-001 was shown to have overall lower flavor intensity scores than NaPBA powder when administered within 5 minutes of preparation.’
“Despite available treatments, there remains an unmet medical need for additional management options for the treatment of UCDs. We look forward to the Prescription Drug User Fee Act (PDUFA) target action date on June 5, 2022 and potential approval by the U.S. Food and Drug Administration (FDA) of Acer’s new drug application for ACER-001 for the treatment of UCDs. Assuming a favorable decision from the FDA, we plan to submit a Marketing Authorization Application in the EU for the product during 2022.”Raghuram (Ram) Selvaraju, Chairman of Relief
The primary urea cycle disorders (UCDs) result from an inherited defect in one of the 6 enzymes or 2 transporters of the urea cycle. A defect in any of the urea cycle enzymes leads to the accumulation of ammonia, resulting in deleterious effects on the central nervous system, including brain damage, coma, and death.
“We are pleased to present results from these two studies at another renowned genetic metabolic conference.”Adrian Quartel, MD, CMO of Acer
Adrian added, “These data further support our belief that, if approved, ACER-001 could offer an alternative to current therapies that may lead to meaningful clinical outcomes in patients with Urea Cycle Disorders (UCDs).”
As reported, ‘ACER-001 was bioequivalent to NaPBA powder under both fasting and fed conditions. Higher levels of PBA and PAA were observed when ACER-001 was administered under fasting versus fed conditions –A similar reduction in the PK of NaPBA powder under fed conditions was observed between Study 1 and Study 2. The adverse events in these studies showed no major safety signals and were similar to NaPBA. These studies suggest investigating administration of nitrogen scavengers under fasting conditions is warranted, which may ultimately provide lower dose options and increased dosing flexibility.’