“Many UCDs patients currently taking nitrogen scavengers may struggle with elevated ammonia levels potentially associated with consequences of treatment non-compliance,” said
Details of the presentations are as follows:
2022 SIMD 43rd Annual Meeting
Title: The Pharmacokinetics of Taste-Masked Sodium Phenylbutyrate (Acer-001) for the Treatment of Urea Cycle Disorders Under Fasting and Fed Conditions in
Presenter: Dr.
Poster #: 84
Date/Time:
Title: Taste-Masked Coating of Sodium Phenylbutyrate (Acer-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders
Presenter: Dr.
Poster #: 18
Date:
Additionally, Acer is sponsoring a lunchtime symposium at SIMD on
2022
Title: ACER-001: a Potential Alternative to Sodium and Glycerol Phenylbutyrate for Treatment of Urea Cycle Disorders
Presenter: Dr.
Poster #: 26
Date:
Title: Taste-masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders
Presenter: Dr.
Poster #: 10
Date/Time:
Additionally, Acer is sponsoring a breakfast symposium at GMDI on
Parties interested in the ACER-001 program for UCDs may sign up for updates at:
https://www.acertx.com/rare-disease-research/acer-001-for-urea-cycle-disorders-ucds/
About 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 patients with 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 patients with UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate and sodium phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports3, 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 aversive taste and odor4, frequency with which medication must be taken, required number of pills, and the high cost of the medication.
About ACER-001
ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). ACER-001’s multi-particulate dosage formulation for oral administration is designed to minimize the aversive taste and odor4 of sodium phenylbutyrate while quickly dissolving in the stomach. The ACER-001 NDA for UCDs is currently under FDA review with a PDUFA target action date of
About
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In
About
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate, RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the
References
Ah Mew N , et al. Urea cycle disorders overview. Gene Reviews.Seattle, Washington :University of Washington ,Seattle ; 1993.- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders.
Orphanet Journal of Rare Diseases . 2012;7(32). - Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
- Peña-Quintana L, et al. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017
Sep 6 ;11:1489-1496.
Acer Forward-Looking Statements
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CORPORATE CONTACTS
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