Actimed Licenses European Rights to ACM-002 for Treating ALS

Actimed Therapeutics has obtained the rights to develop and commercialize ACM-002 (S-oxprenolol) for the treatment of amyotrophic lateral sclerosis (ALS) in Europe, the company announced.

The rights, licensed from patent owner Charité University Medicine Berlin, cover all major European markets, including France, Germany, the U.K., Italy, and Spain. Actimed now owns global rights to ACM-002 for this indication.

“Given the world-leading expertise in muscle wasting disorders available to the company, we believe it is an ideal partner to further the development of S-oxprenolol in ALS, where there remains a high unmet medical need,” Bettina Büttner, PhD, said in a press release. Büttner is technology manager at BIH Innovations, the joint technology transfer office of Charité and the Berlin Institute of Health (BIH).

ACM-002 is composed of a medication often used to treat high blood pressure, chest pain, and irregular heartbeat. It targets three biological mechanisms involved in muscle waisting: anabolism, or the building of complex molecules out of smaller ones; catabolism, which is the part of the metabolism responsible for breaking those complex molecules into smaller ones; and  fatigue/appetite.

Importantly, ACM-002 is soluble in fat and can cross the blood-brain barrier easier than other medications of the same class. The blood-brain barrier is a natural protective layer that regulates the transport of molecules between the brain’s blood vessels and brain tissue.

The company initially licensed and started exploring this molecule for the treatment of cachexia — the changes in appetite and metabolism that cause extreme weight loss and muscle waisting in cancer patients.

But data has shown that ACM-002 also might be a good treatment candidate for ALS.

When animal models of ALS carrying a SOD1 mutation were treated with this compound, they lived 33% longer than animals receiving a placebo — 56 versus 42 days. Animals also experienced less muscle loss, as well as fewer  reductions in body lean mass, body fat mass, and overall weight.

Notably, animals receiving a ACM-002 at a dose of 20 mg/kg per day also significantly outlived those given a greater dose of riluzole (30 mg/kg day), an approved medication for ALS, showing the potential of this new medication.

“… we are delighted to sign this additional licensing agreement with Charité [which] gives us global rights to S-oxprenolol in both potential indications,” said Robin Bhattacherjee, CEO of Actimed Therapeutics. This “position will significantly enhance our options to develop this molecule in cachexia and now ALS.”