Acer Therapeutics Unveils Data Identifying Ideal Attributes for Treating Urea Cycle Disorder at Society for Inherited Metabolic Disorders Annual Meeti

Acer Therapeutics Inc. recently presented data at the 44th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), which focused on healthcare providers' preferences for treating Urea Cycle Disorders (UCDs). The survey results showed that healthcare providers identified taste and odor as the most important factors that affect the overall prescription and patient adherence to UCD treatments, specifically nitrogen-binding medications like sodium phenylbutyrate and glycerol phenylbutyrate. Acer Therapeutics Inc. is a company that acquires, develops, and commercializes therapies for rare, serious, and life-threatening illnesses with significant unmet medical needs.

Dr. Robert Steiner, a professor at the University of Wisconsin School of Medicine and Public Health, shared that using medication that binds with nitrogen (like sodium phenylbutyrate or glycerol phenylbutyrate) can be effective in treating UCDs, but only if patients take their medication regularly. Unfortunately, up to 25% of dangerous hyperammonemic crises in UCD patients may happen because patients don't stick to their prescribed medication and/or diet, and some types of nitrogen-binding medication can make adherence more difficult. Dr. Steiner believes that new treatment options are necessary as a result.

At the 2023 SIMD Meeting, Acer shared data on UCD treatment preferences gathered from healthcare providers. The poster presentation is now available to view on Acer's website using the following link: https://www.acertx.com/wp-content/uploads/2023/03/2023-SIMD-DCE-Poster.pdf

In this article, Poster #97 talks about a study that was presented at an event called SIMD. The research was done online and used a method called discrete choice experiment or DCE. Its goal was to find out what people wanted in their medications for Urea Cycle Disorder, specifically nitrogen-binding medicines like sodium phenylbutyrate and glycerol phenylbutyrate. By doing this survey, they were able to identify the most important factors that could affect patients taking and sticking to their treatment plans.

Out of the 51 healthcare providers who were surveyed, the majority expressed their discontent with the current available treatment choices. When asked to rate their satisfaction level, with 1 being the least satisfied and 9 being the most satisfied, the average rating was 5.4 with a standard deviation of 1.7. The survey results revealed that for both prescribing and patient adherence, taste and odor were identified as the most crucial factors. As a result, the authors suggested that by enhancing the patient experience, such as masking taste and odor, or improving other aspects of the medication, could enhance adherence and support improved outcomes in UCDs.

According to Adrian Quartel, MD, FFPM, who is Acer's CMO, OLPRUVA™5 brings together the well-known effectiveness of sodium phenylbutyrate with an inventive double-coating that makes the medication easier to swallow. He also mentions that Acer is proud to provide a new, reasonably priced treatment choice for those with UCDs, and that the company is devoted to collaborating with the UCD community to help individuals affected by the disorder live full, uninterrupted lives.

New evidence was presented at two recent conferences, SIMD and GMDI, revealing improved palatability of ACER-001 (sodium phenylbutyrate) for treating Urea Cycle Disorders. Researchers found that a taste-masked coating of ACER-001 improved the overall flavor intensity scores compared to sodium phenylbutyrate (BUPHENYL®) powder. These findings were from two Phase 1 studies which measured taste assessments of ACER-001 suspension and BUPHENYL® powder. ACER-001 is now marketed in the U.S. as OLPRUVA™ (sodium phenylbutyrate) after receiving FDA approval in December 2022. Results from both studies indicate that the introduction of ACER-001 has significantly improved the palatability of sodium phenylbutyrate for patients with UCDs.

An innovative treatment option for Urea Cycle Disorders (UCDs) has been approved by the FDA. This new option goes by the brand name OLPRUVA™ and is formulated with sodium phenylbutyrate. OLPRUVA™ is a prescription medication that is used alongside certain therapies including dietary changes, to manage the symptoms of UCDs in adults and children who weigh 44 pounds (20kg) or more, and have a body surface area (BSA) of 1.2 m2 or greater, and deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). As always with new medication, full Prescribing Information and Important Safety Information can be found in the Patient Information.

Acer Therapeutics is a company that works on finding, developing, and selling treatments for severe and rare diseases that have no cure. In the United States, they have OLPRUVA™, a medication made from sodium phenylbutyrate, which is used to treat people who have urea cycle disorders (UCDs) that result from a lack of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer also has other drugs that are being developed to treat life-threatening diseases, such as Maple Syrup Urine Disease (MSUD), vascular Ehlers-Danlos syndrome (vEDS), post-traumatic stress disorder (PTSD), prostate cancer, and Vasomotor Symptoms (VMS). For more detailed information, visiting their website at www.acertx.com is recommended.

The Acer press release includes statements regarding the risks and uncertainties involved in their launch activities and survey data. These statements are considered forward-looking and are only estimates. The company is still in the early stages of commercializing their product OLPRUVA™ for treatment of UCDs, and they still lack development in sales, marketing, and distribution capabilities. There is no guarantee that their investigational products, including OLPRUVA™ for other indications, will be approved by health authorities or available commercially. The results and performance of the company may differ from what is projected due to various factors like financing availability, drug development risks, and regulatory approval processes. The company claims no responsibility to update these statements based on events or circumstances that occur after the original release date. The full filings of the company with the Securities and Exchange Commission are available for viewing at http://www.sec.gov.

If you want to get in touch with Acer Therapeutics Inc., you can reach out to Jim DeNike, who serves as their contact for corporate and investor relations. You can connect with him by sending an email to [email protected] or by dialing their telephone number, which is +1-844-902-6100.

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