Actimed Plans to Advance Treatment for Muscle Wasting in ALS

Actimed Plans to Advance Treatment for Muscle Wasting in ALS
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Actimed Therapeutics is developing S-oxprenolol to prevent muscle wasting and body mass loss due to amyotrophic lateral sclerosis (ALS), using proceeds from a recent license agreement with Faraday Pharmaceuticals.

Under the agreement, Faraday acquired global rights to develop and commercialize S-oxprenolol for muscle-affecting disorders other than ALS, particularly cancer-related cachexia, a wasting syndrome.

Actimed received an upfront payment of $550,000, and is eligible for a $2.7 million near-term milestone payment, plus additional revenue as more milestones are reached.

“We are pleased to partner with Actimed on S-oxprenolol as we are committed to developing and advancing therapies that will prevent and treat cardiac and skeletal muscle loss associated with multiple morbidities. S-oxprenolol is highly complementary to our existing portfolio and will further strengthen our product pipeline,” Steve Hill, Faraday’s CEO, said in a press release.

Most immediately, earnings will be used to move Actimed’s lead candidate ACM-001 (S-pindolol) into clinical testing in cancer patients with cachexia — changes in appetite and, especially, body metabolism that cause extreme weight loss and muscle wasting that cannot be corrected through diet.

“This deal enhances our ability to speed up and further advance the development of our lead candidate ACM-001 (S-pindolol) towards Phase 2 clinical studies for cancer cachexia in patients with non-small cell lung cancer and colorectal cancer,” said Robin Bhattacherjee, the company’s CEO .

“We are also pleased to retain rights to S-oxprenolol for ALS, an indication where patients suffer similar life-threatening muscle-wasting effects,” Bhattacherjee added.

S-oxprenolol 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 that diminishes appetite.

The treatment is reported to reach the brain and spinal cord more easily than other medications of the same class.

S-oxprenolol was initially developed to treat cachexia in cancer patients, as at least half of these people are estimated to develop this wasting syndrome as their disease advances.

But a study in mouse models of ALS showed its potential as a treatment candidate for ALS. In one experiment, its use lessened muscle and weight loss, and extended the animals’ lifespan by 33% compared with mice given a placebo.

In another, mice treated with S-oxprenolol at a daily dose of 20 mg/kg also significantly outlived those given a higher daily dose (30 mg/kg) of riluzole, an approved ALS medication.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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