A new therapeutic option to address debilitating rare diseases
Urea cycle disorders (UCDs) and maple syrup urine disease (MSUD) are rare genetic metabolic disorders for which current treatment options are limited, or non-existent in the case of MSUD. Both diseases greatly affect patients’ quality of life. True to our mission to provide patients with therapeutic relief from serious illnesses with high unmet medical needs, we partnered with Acer Therapeutics, a U.S.-based biotech company with strong capabilities in accelerated development of therapies for rare disease, for the worldwide development and commercialization of OLPRUVA™ (sodium phenylbutyrate) for oral suspension (formerly known as ACER-001).
OLPRUVA™, a proprietary powder formulation of sodium phenylbutyrate (NaPB), is designed to be both taste-masked and immediate release. These characteristics could make it a compelling alternative to existing NaPB-based treatments for UCD, as the unpleasant taste associated with NaPB is cited as a major impediment to patient compliance with those treatments.
Learn more about OLPRUVA™ (sodium phenylbutyrate) for oral
suspension
In March 2021, Relief signed a collaboration and license agreement with Acer Therapeutics Inc. (“Acer”) for the worldwide development and commercialization of ACER-001. In August 2023, Relief and Acer entered into a new exclusive definitive licensing agreement for the development and commercialization of OLPRUVA™ for the treatment of certain UCDs and other potential indications. This agreement supersedes the March 2021 collaboration and license agreement between the companies.
Under the terms of the revised agreement, Acer retains development and commercialization rights for OLPRUVA™ in the U.S. and other countries worldwide, excluding geographical Europe. Relief will receive a 10 percent continuing royalty calculated on the net sales of OLPRUVA™ in the Acer territory up to a cumulative amount of $45 million. At the same time, Relief retains development and commercialization rights for OLPRUVA™ in geographical Europe, which includes the European Union, Liechtenstein, San Marino, Vatican City, Norway, Iceland, Principality of Monaco, Andorra, Gibraltar, Switzerland, United Kingdom, Albania, Bosnia, Kosovo, Montenegro, Serbia and North Macedonia.
Under the terms of the initial collaboration and license agreement, Acer received $35 million cash payments from Relief. With the execution of the revised license agreement in August 2023, Acer provided Relief with a non-contingent $10 million upfront cash payment and shall provide an additional non-contingent $1.5 million cash payment in August 2024.