RARE Daily

Relief and Acer Sign Option Agreement for Exclusivity on Treatment for UCDs

January 26, 2021

Rare Daily Staff

Swiss biotech Relief Therapeutics and rare disease drug developer Acer Therapeutics said they have signed an option agreement providing exclusivity for the right to negotiate a potential collaboration and license agreement for worldwide development and commercialization for ACER-001 for the treatment of urea cycle disorders and Maple Syrup Urine Disease.

Urea cycle disorders (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.

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.

Maple Syrup Urine Disease (MSUD) is a rare but serious inherited condition whereby the human body cannot process certain amino acids, causing a harmful build-up of substances in the blood and urine. The human body breaks down protein foods such as meat and fish into amino acids. Other than a highly restricted diet of branched-chain amino acid free synthetic foods and formula, there are no currently approved treatments for MSUD.

Under the terms of the option agreement, Acer will receive a $1 million non-refundable payment from Relief in return for exclusivity until June 30, 2021 to negotiate and enter into a definitive collaboration and license agreement between the two companies for the development of ACER-001. Relief will also make a $4.0 million loan to Acer.

If the collaboration and license agreement is executed by the end of June, Acer will receive $15 million in cash (inclusive of the $1 million payment and offset by a repayment of the $4 million loan from Relief). In addition, Relief will agree to pay up to $20 million in U.S. development and commercial launch costs for the UCDs and MSUD indications. Further, Acer will retain development and commercialization rights in the U.S., Canada, Brazil, Turkey and Japan. The companies will split net profits from Acer’s territories 60:40 in favor of Relief. Relief will also license the rights for the rest of the world, where Acer will receive from Relief a 15 percent net sales royalty on all revenues received in Relief’s territories. Acer could also receive a total of $6 million in milestones based on the first European marketing approvals for UCDs and MSUD.

“This potential collaboration could provide important resources and additional expertise to help bring ACER-001 to patients worldwide suffering from debilitating diseases like UCDs and MSUD,” said Chris Schelling, Acer founder and CEO.

For Relief Therapeutics, which is mainly focused on respiratory therapies with a compound in late stage development for Covid-19, the potential partnership with Acer is its first initiative to build a pipeline of drugs beyond its Covid-19 therapy.

ACER-001 is a powder formulation of sodium phenylbutyrate (NaPB). The formulation is designed to be both taste-masked and immediate release. ACER-001 is being developed using a microencapsulation process for the treatment of various inborn errors of metabolism, including UCDs and MSUD. If ACER-001 is approved, its taste-masked properties could make it a compelling alternative to existing NaPB-based treatments, as the unpleasant taste associated with NaPB is cited as a major impediment to patient compliance with those treatments. The U.S. Food and Drug Administration has granted orphan drug designation to ACER-001 for the MSUD indication.

Photo: Chris Schelling, Acer founder and CEO

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