Acer Therapeutics develops and commercializes therapies specifically for patients with serious rare and life-threatening diseases, utilizing targeted pharmacological approaches to address unmet medical needs. The company focuses on conditions such as Maple Syrup Urine Disease and Vascular Ehlers-Danlos Syndrome, providing critical treatment options for patients who lack effective therapies.
Funding
$1M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Patients with Urea Cycle Disorders (UCDs) require lifelong management to prevent the buildup of toxic ammonia in the blood, which can lead to severe neurological damage and death. Existing treatments, such as sodium phenylbutyrate powder, can be unpalatable and inconvenient to administer, impacting patient compliance and quality of life. Current options may not adequately address the need for convenient and accurate dosing, especially for those managing their condition on the go.
Solution
Zevra Therapeutics offers OLPRUVA, a prescription medicine containing sodium phenylbutyrate, formulated as a palatable, premeasured, and portable oral suspension for long-term management of UCDs. OLPRUVA's unique dual-coating formulation improves palatability, while single-dose envelopes ensure accurate dosing and ease of administration. Designed for individuals weighing 44 pounds (20 kg) or greater with a body surface area (BSA) of 1.2 m2 or greater, OLPRUVA helps the body eliminate excess nitrogen, preventing dangerous ammonia buildup. Unlike traditional powder formulations, OLPRUVA's convenient packaging and taste-masked formulation promote better adherence to prescribed treatment regimens, supporting improved ammonia control and overall patient well-being.
Target Audience
The primary target audience includes adults and children (weighing 44 pounds (20 kg) or greater with a BSA of 1.2 m2 or greater) diagnosed with Urea Cycle Disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic synthetase (AS), as well as their caregivers and healthcare providers.
Features
- Palatable dual-coating formulation designed to mask the taste of sodium phenylbutyrate
- Premeasured single-dose envelopes for accurate and convenient administration
- Portable and discreet packaging for on-the-go ammonia control
- Indicated for long-term management of UCDs in patients weighing 44 pounds (20 kg) or greater with a BSA of 1.2 m2 or greater
- Does not require syringes for administration
- Designed to be taken with food