FDA Grants Orphan Drug Status to Actimed's ALS Candidate

The US Food and Drug Administration (FDA) has granted orphan drug status to Actimed Therapeutics' S-oxprenolol (ACM-002) for treating amyotrophic lateral sclerosis (ALS). This decision allows the UK-based pharmaceutical company to receive tax credits for clinical trials conducted in the US and, if the therapy gets approval, the potential for seven years of market exclusivity.

According to Actimed, S-oxprenolol shares similar pharmacological properties with S-pindolol, an anabolic/catabolic transforming agent. Actimed's primary product, S-pindolol benzoate, is currently under investigation for treating cachexia, a syndrome characterized by metabolic weight loss. With S-oxprenolol, Actimed aims to leverage S-pindolol's mechanistic approach in addressing the body mass and muscle wasting typically observed in ALS patients.

ALS, the most prevalent form of motor neuron disease, results in muscle wasting as the degeneration of motor neurons disrupts the transmission of electrical signals to the muscles. This condition leads to significant muscle atrophy and weakness, severely impacting patient quality of life.

Currently, the FDA has approved six therapies for ALS. However, the agency has also laid out a five-year plan to accelerate the development of additional treatments and improve access to these medicines. The ALS treatment landscape has faced its share of challenges, including the Phase III trial failure of Amylyx Pharmaceutical’s candidate and the decision by Ionis/Biogen to halt the development of their ALS drug.

Preclinical models of ALS have shown that S-oxprenolol can reduce muscle loss, improve lean body mass, and decrease overall body mass loss, among other benefits. Oxprenolol, typically used as a beta blocker for heart conditions such as angina and high blood pressure, has not had its mechanism of action in ALS fully disclosed by Actimed. However, a related compound, S-pindolol, enhances anabolism and muscle growth through partial beta-2 receptor agonism.

Actimed is actively conducting trials for S-pindolol in cancer cachexia, a type of the syndrome associated with cancer patients. The compound has shown promising results in a Phase IIa study, and additional clinical trials are in the pipeline.

Although Actimed has not yet provided detailed plans for a clinical program specifically for S-oxprenolol, the company has licensed the global rights to the drug for cancer cachexia and other non-ALS indications to Faraday Pharmaceuticals, a US-based company. The agreement, finalized in 2021, included an upfront payment of $550,000 and $126.2 million in milestone payments.

Actimed’s CEO, Robin Bhattacherjee, expressed optimism about the early non-clinical data for S-oxprenolol, suggesting it could play a crucial role in addressing the unmet needs of ALS patients. He emphasized the potential of S-oxprenolol to significantly improve the quality of life for individuals affected by this debilitating disease.

In summary, the FDA’s orphan drug designation for S-oxprenolol marks a significant step for Actimed Therapeutics in its quest to develop effective treatments for ALS. By focusing on the muscle-wasting aspect of the disease, Actimed hopes to provide new therapeutic options for patients who currently have limited choices. The ongoing research and future clinical trials will determine whether S-oxprenolol can fulfill its promise and become a viable treatment for ALS.

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