Actimed Therapeutics Announces US FDA Grant of Orphan Drug Designation to S-oxprenolol (ACM-002) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

London, UK – 4^th September 2024. Actimed Therapeutics Ltd (“Actimed”), a UK based clinical stage specialty pharmaceutical company focused on bringing innovation to the treatment of cancer cachexia and other muscle wasting disorders, announces that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to S-oxprenolol (ACM-002) for the treatment of amyotrophic lateral sclerosis (ALS).

ALS (also known as Lou Gehrig’s disease) is a progressive, fatal neuromuscular disease that affects nerve cells in the brain and spinal cord, eventually leading to a loss of muscle control and paralysis. ALS prevents motor neurons sending impulses from the brain and spinal cord to the muscles, causing muscle atrophy and weakness and has a mean survival of 3.5 years. Voluntary muscle action is progressively lost and in late-stage ALS patients may have difficulty communicating, swallowing, moving, and breathing. There is currently no cure for ALS.

Cachexia, a complex wasting disease, may occur in the early stages of ALS and has a significant impact on quality of life, proving terminal in some patients. In pre-clinical ALS models, S-oxprenolol has demonstrated significant benefits including slowing disease progression, improvement in survival, attenuation of muscle loss, motor neuron protection, reduction in loss of body mass and improvements in lean body mass¹.

Robin Bhattacherjee, Chief Executive Officer of Actimed Therapeutics commented “We are pleased to receive Orphan Drug Designation for S-oxprenolol in ALS, where loss of body mass and muscle wasting can have a significant impact on survival. ALS is a devastating orphan disease of high, unmet medical need and the early non-clinical data we have generated for S-oxprenolol in this indication suggest that it could have a potential role in meeting some of the important needs of this under-served patient population”.

FDA Orphan Drug Designation is granted to investigational therapies which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides several benefits to drug developers, including certain development cost benefits in the US, increased FDA interaction and eligibility for seven-year market exclusivity following approval.

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About Actimed Therapeutics 

Actimed Therapeutics is a clinical stage specialty pharmaceutical company focused on bringing innovation to the treatment of muscle wasting disorders to transform the care of an underserved and vulnerable patient population.

The lead area of focus for Actimed is specifically in cachexia. Cachexia is a wasting disease that is associated with cancer and other serious chronic illnesses and with significant morbidity and mortality. A significant number of cancer patients suffer from cachexia² and it is estimated that cachexia is responsible for up to 20% of all cancer deaths³. A recent meta-analysis demonstrated that cachexia was associated with an 82% higher relative risk of mortality in patients with NSCLC versus no cachexia⁴.

Despite its prevalence and devastating clinical effects, there is no globally approved drug for the treatment or prevention of cancer-related cachexia. 

The lead product of Actimed, S-pindolol benzoate (ACM-001.1) targets multiple pathways that drive cachexia and has generated promising proof of concept Phase 2a clinical data in cachexia patients. Actimed is currently preparing for further clinical studies in cancer cachexia having received an Investigational New Drug (IND) approval from FDA for this programme in August 2023.

Actimed also owns the global rights to its second asset, S-oxprenolol, which is being developed by the Company for the muscle wasting seen in amyotrophic lateral sclerosis (ALS) where loss of body mass and muscle wasting may impact survival⁵. Actimed has licensed the global rights to develop and commercialise S-oxprenolol for cancer cachexia and any other indications outside of ALS to US company Faraday Pharmaceuticals.