Actimed's S-oxprenolol Gets FDA Orphan Drug Status for ALS

Actimed Therapeutics Ltd, a London-based clinical-stage pharmaceutical company specializing in innovative treatments for cancer cachexia and other muscle-wasting conditions, recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to their product S-oxprenolol (ACM-002) for amyotrophic lateral sclerosis (ALS).

ALS, often referred to as Lou Gehrig’s disease, is a progressive and fatal neuromuscular disorder that impacts nerve cells in the brain and spinal cord. This leads to muscle control loss and eventual paralysis. The disease hampers motor neurons' ability to relay impulses from the brain and spinal cord to the muscles, resulting in muscle atrophy and weakness. The average survival rate for ALS patients is 3.5 years. As the disease advances, patients struggle with communication, swallowing, movement, and breathing. Currently, there is no cure for ALS.

Cachexia—a complex syndrome causing extreme weight loss and muscle wasting—can manifest in the early stages of ALS, significantly deteriorating the quality of life and proving fatal for some patients. Pre-clinical models of ALS have shown that S-oxprenolol offers several benefits, including slowing disease progression, improving survival rates, reducing muscle loss, protecting motor neurons, minimizing body mass loss, and enhancing lean body mass.

Robin Bhattacherjee, Chief Executive Officer of Actimed Therapeutics, expressed satisfaction with the FDA's Orphan Drug Designation for S-oxprenolol in ALS. He highlighted the severe impact of body mass loss and muscle wasting on ALS patients' survival. Bhattacherjee emphasized that ALS is a devastating disease with a high unmet medical need and that early non-clinical data suggest S-oxprenolol could potentially address significant aspects of this under-served patient population's needs.

The FDA awards Orphan Drug Designation to investigational therapies aimed at treating, diagnosing, or preventing rare diseases or conditions affecting fewer than 200,000 people in the United States. This designation offers several advantages to drug developers, including development cost benefits, increased interactions with the FDA, and eligibility for seven-year market exclusivity post-approval.

Actimed Therapeutics focuses on developing treatments for muscle wasting disorders, particularly cachexia, a condition often seen in cancer and other chronic illnesses. Cachexia, which is linked with high morbidity and mortality, affects a substantial number of cancer patients. It is estimated to account for up to 20% of all cancer deaths. Research has shown an 82% higher relative risk of mortality in non-small-cell lung cancer patients with cachexia compared to those without.

Despite its prevalence and severe impact, there is no globally approved drug for preventing or treating cancer-related cachexia. Actimed's lead product, S-pindolol benzoate (ACM-001.1), targets multiple pathways driving cachexia and has shown promising Phase 2a clinical data in cachexia patients. The company is planning further clinical studies in cancer cachexia, having received an Investigational New Drug (IND) approval from the FDA in August 2023.

Additionally, Actimed owns the global rights to S-oxprenolol for treating muscle wasting in ALS. The company has licensed global rights to develop and commercialize S-oxprenolol for cancer cachexia and other indications outside ALS to Faraday Pharmaceuticals, a US-based firm.