Acer Therapeutics and Relief Therapeutics Announce Presentation of ACER-001 Data at the Genetic Metabolic Dieticians International Conference - Seite 2

The data presented concluded that ACER-001 was bioequivalent to sodium phenylbutyrate (BUPHENYL ^® ) powder under both fed and fasting conditions. Higher levels of phenylbutyrate (PBA) and phenylacetate (PAA), a conjugate base of phenylacetic acid, were observed when ACER-001 was administered under fasting versus fed conditions. A similar reduction in the PK of sodium phenylbutyrate (BUPHENYL ^® ) powder under fed conditions was observed between the fasted and fed studies. Adverse events in these studies showed no major safety signals and were similar to sodium phenylbutyrate (BUPHENYL ^® ). These studies suggest investigating administration of nitrogen scavengers under fasting conditions, which may ultimately provide lower dose options and increase dosing flexibility.

Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders ²
The second poster presented at GMDI details results from two Phase 1, open-label, repeated measures, taste assessment studies of polymer coated sodium phenylbutyrate (ACER-001) and sodium phenylbutyrate (BUPHENYL ^® ) powder. The studies included healthy panelists who were required to complete a training program for a minimum of six months that educated panelists on the identification, description, and quantification of sensory attributes of products.

The objective of the two taste assessment studies was to identify and quantify the intensity of perceived flavor attributes of ACER-001 relative to sodium phenylbutyrate (BUPHENYL ^® ) powder. Results from both studies concluded that ACER-001 was shown to have overall lower flavor intensity scores than sodium phenylbutyrate (BUPHENYL ^® ) powder when administered within five minutes of preparation.

About UCDs
The urea cycle is a series of biochemical reactions that occur primarily in the liver, which converts toxic ammonia produced by the breakdown of protein and other nitrogen-containing molecules in the human body into urea for excretion ³ . UCDs are a group of disorders caused by genetic mutations that result in a deficiency in any one of the six enzymes or two of the amino acid transporters, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. ⁴ Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.