EQS-News

Relief Provides Update on Progress and Plans with ACER-001 for the Treatment of Urea Cycle Disorders - Seite 2

Based on the timelines outlined above and pending a positive decision by regulators, ACER-001 could be launched in both the U.S. and Europe during 2022.

ACER-001 is also being developed for the treatment of Maple Syrup Urine Disease (MSUD).

Jack Weinstein, Chief Financial Officer and Treasurer of Relief, said, "It is great to see the progress being made with ACER-001. We are excited to continue moving forward with the Acer team to develop and commercialize this product candidate around the globe to address important unmet needs for patients with UCDs, a rare and debilitating set of diseases. Following on the important steps being made by our partner in the U.S., we expect to be able to move forward rapidly in Europe."

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ABOUT UREA CYCLE DISORDERS (UCDS)

UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. 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.^1,2

The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for UCDs include nitrogen scavengers, RAVICTI^(R) and BUPHENYL^(R), in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.³