Oxprenolol

ALS prevents motor neurons from sending impulses from the brain and spinal cord to the muscles, causing muscle atrophy and weakness. Image credit: Shutterstock / CC7. The US Food and Drug Administration (FDA) has granted an orphan drug designation to Actimed Therapeutics’ S-oxprenolol (ACM-002) for the treatment of amyotrophic lateral sclerosis (ALS). The UK-based pharma company will now be in line for tax credits for US-based clinical trials, and if the therapy is approved, the potential for seven years of market exclusivity in the designated indication, as per a 4 September press release. Actimed says S-oxprenolol shares “a similar pharmacology” to S-pindolol, which is an anabolic/catabolic transforming agent. S-pindolol benzoate, Actimed’s lead product, is being investigated for the treatment of the metabolic weight loss syndrome called cachexia. With S-oxprenolol, Actimed is targeting S-pindolol’s mechanistic approach in the wasting syndrome to address the loss of body mass and muscle wasting seen in ALS. ALS is the most common form of motor neuron disease. Muscle wasting is seen in patients as motor neuron degeneration means muscles no longer receive electrical signals. See Also: ESC 2024: Beta blocker treatment paradigm shifts for myocardial infarction Novavax wins FDA emergency approval for updated Covid-19 vaccine There are currently six FDA-approved therapies for ALS, though the agency has developed a five-year plan to accelerate the development of more treatments and improve medicine access. There have been a few recent setbacks in the space, including Amylyx Pharmaceutical’s Phase III failure and Ionis/Biogen’s decision to discontinue the development of their ALS candidate. In preclinical ALS models, S-oxprenolol has demonstrated an ability to attenuate muscle loss, improve lean body mass, and reduce the loss of overall body mass, amongst other benefits. Oxprenolol is a beta blocker usually used for the treatment of heart conditions such as angina and high blood pressure. While Actimed has not disclosed the mechanism of action in ALS, the related compound S-pindolol increases anabolism and muscle by partial beta-2 receptor agonism. Actimed is currently conducting trials for S-pindolol in cancer cachexia, a sub-type of the syndrome found in cancer patients. The compound has demonstrated positive proof of concept data in a Phase IIa study, with further clinical trials planned according to the company. Actimed has yet not unveiled details of a clinical programme for S-oxprenolol. Nonetheless, the company has already licensed the drug’s global rights for cancer cachexia and non-ALS indications to US company Faraday Pharmaceuticals. The deal, signed in 2021, included an upfront payment of $550,000 and $126.2m in milestone payments. Actimed’s CEO Robin Bhattacherjee said: “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.”

London, UK – 19th October 2023. 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, today announces the appointment of Martin Walton as Chief Financial Officer. Since 2009, Martin has been an active Venture Capital and Private Equity investor, portfolio manager, and advisor in life sciences working with several of the largest investors in the sector. He has held a number of executive positions (Executive Chairman, CEO, CFO), completing transactions with over 25 companies (spinouts, financing rounds, M&A, IPOs, licensing deals, divestitures and trade sales) and raising over £1bn in investment and co-investment capital. He is Chairman and CEO of Bradshaw Consulting Limited, a strategic advisory group assisting companies and shareholders in creating, generating and realising value from investments in the life sciences sector. Robin Bhattacherjee, Actimed CEO, commented: “I am delighted to welcome Martin to Actimed, who brings with him a set of highly complementary skills and extensive industry experience. This will be invaluable as we progress the business and ensure that Actimed is well financed to deliver on its ambitious plans and objectives.” Originally CEO of Excalibur Fund Management, Martin is a co-founder of LSE-listed Arix Bioscience plc (LSE: ARIX), of Arthurian Life Sciences Limited and of Excalibur Medicines Ltd. He is currently an investor/Director at Interrad Medical LLC, developer of the SecurAcath catheter securement device (now centrally purchased as the standard of care in the NHS), of AIM-listed Reneuron Plc (AIM: RENE), a UK-based leader in exosomes technologies, and of the Liverpool Life Sciences Accelerator Partnership. In addition, he has extensive Governance, Oversight, Audit Committee and Risk Committee experience as well as specific experience in start-up, growth strategies (organic and acquisition), financing, turnaround and consolidation strategies in the healthtech, biotech and medtech sectors. Martin initially spent 25 years in global investment banking and asset management, in UK, USA and Europe, culminating in a position as Vice Chair responsible for Wholesale and Commercial Banking for Europe and Asia-Pacific at Toronto-Dominion Bank. Martin Walton, Actimed CFO commented: “I am excited to be joining Actimed at this important point in the company’s journey to develop the first globally approved product for cancer cachexia. In particular, having gained Investigational New Drug (IND) approval for the Phase 2b/3 clinical study of S-pindolol benzoate (the “IMPACT” clinical trial programme) to treat cachexia, the Company will now move to the next stage of its development. Much work remains to be done, but I believe Actimed has the product, people and capabilities to deliver significant benefits to this under-served patient population and achieve substantial commercial success.” *** 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. Actimed was founded in 2017 by Stefan Anker and Andrew Coats, two eminent physicians in muscle wasting research, together with Yann Colardelle, a professional in communications and medical education who has been involved in research and education in cachexia for many years. 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 cachexia1 and it is estimated that cachexia is responsible for up to 20% of all cancer deaths2. A recent meta-analysis demonstrated that cachexia was associated with an 82% higher relative risk of mortality in patients with NSCLC versus no cachexia3. 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 phase 2b/3 clinical studies in cachexia in Non-Small Cell Lung Cancer (NSCLC) and Colorectal Cancer (CRC) - the IMPACT programme: IMProving cancer cachexia with ACTAs (Anabolic/Catabolic Transforming Agents). Actimed received an Investigational New Drug (IND) approval from the 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 survival⁴. 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. FOR MORE INFORMATIONActimed Therapeuticswww.actimedtherapeutics.com MEDiSTRAVA ConsultingFrazer Hall, Erica HollingsworthTel: +44 (0)203 928 6900Email: actimed@medistrava.com WG Partners LLPNigel Barnes, Satheesh Nadarajah, Robin Stürken Tel: +44 (0) 203 705 9330 Email: olga@wgpartners.co.uk ___________________________[1] Anker M et al., J. Cachexia, Sarcopenia and Muscle; 2019: 10: 22 – 242 Argilés JM et al, Nat Rev Cancer 2014; 14:754-623 Bonomi P. et al. The mortality burden of cachexia in patients with non-small-cell lung cancer: A meta-analysis; International Conference of Sarcopenia, Cachexia and Wasting Disorders, June 17 – 18 2023, Stockholm, abstract 2-18, page 139 ⁴ Wolf J et al., PMID 28184974 DOI: 10.1007/s00115-117-0293