Muscular Atrophy

The EMA’s human medicines committee recommended 114 medicines for approval in 2024, marking the highest number of positive opinions issued in 15 years, according to the regulator’s latest report . Of the 114 recommendations, 46 were for new active substances, the EMA’s chief medical officer Steffen Thirstrup said at a press conference on Thursday. Cancer remained the therapeutic area with the most approvals in 2024, followed by immunology, rheumatology, blood and coagulation disorders, and endocrinology, he added. Thirstrup highlighted several positive opinions issued last year that he said helped address unmet needs, including one for Merck’s Welireg for a rare condition called von Hippel-Lindau disease that is characterized by tumors and cysts that can turn cancerous. The agency also recommended ARS Pharmaceuticals’ EURneffy, an EpiPen alternative that’s an adrenaline nasal spray for severe allergic reactions. Although the active substance itself is not new, the green light marks a “major contribution to patients” because the spray format is “much more handy and can be handled by a non-medic,” Thirstrup said. Looking ahead, label expansions for weight loss drugs — as well as new candidates being advanced to offset their muscle loss or sarcopenia side effects — could be key areas of focus. One ongoing discussion at the Committee for Medicinal Products for Human Use centers on whether cardiometabolic indications related to obesity should be added as new indications for GLP-1s or if the clinical data should just be added on to the product label so prescribers are aware of the latest research, Thirstrup said. In December, CHMP said a label expansion of Eli Lilly’s blockbuster diabetes drug Mounjaro into obstructive sleep apnea isn’t necessary. Meanwhile, several companies are testing candidates designed to preserve muscle when used in conjunction with GLP-1 drugs, including Scholar Rock’s myostatin-focused approach and Veru’s oral selective androgen receptor modulator. “I would not exclude that some obesity products could have an action [in other areas such as cancer cachexia] but we need to have, first of all, a clear, identified disease entity and how do we define cachexia and muscle wasting. And then we need the company to demonstrate an effect on desirable endpoints,” Thirstrup said.

Fabio Dorigotti brings over 25 years of clinical development experienceJoins Actimed following retirement of Frank Misselwitz London, UK – 16 January 2025. Actimed Therapeutics, a UK based clinical stage speciality pharmaceutical company focused on bringing innovation to the treatment of cancer cachexia and other muscle wasting disorders, announces the appointment of Dr Fabio Dorigotti as Chief Medical Officer (CMO) following the retirement of previous CMO, Frank Misselwitz MD. Prior to joining Actimed, Dr Dorigotti was CMO of Swiss biopharmaceutical company Quercis Pharma. He brings over 25 years of pharmaceutical industry experience spanning the full breadth of the clinical development process. Robin Bhattacherjee, Actimed CEO, commented: “I am very pleased to welcome Fabio as our new Chief Medical Officer as we focus on advancing our lead candidate ACM001.1 (S-pindolol benzoate) for cancer cachexia into late-stage clinical development. Fabio brings a wealth of relevant knowledge and experience and a proven track record of success. I would also like to take this opportunity to thank our retiring CMO Frank Misselwitz for his invaluable contribution to Actimed over the last several years and am delighted that he will retain his role as a Non-Executive Director on the Board of the company.” During his career, Dr Dorigotti has held inhouse roles of Head of Global Medical Affairs for CSL Vifor and Medical Affairs Director at Astrazeneca, and as Chief Strategy Officer and President of Global Clinical for Worldwide Clinical Trials, a full-service global contract research organization (CRO). He brings a strong track record of successful clinical development and product marketing approvals, alongside a deep understanding of FDA, EMA and other regulatory agencies. Dr Dorigotti commented: “Joining Actimed as CMO provides me with an exciting opportunity to bring my skills and experience to bear in the further development of products that could fill an unmet medical need and benefit patients with serious muscle wasting disorders. I look forward to collaborating with the excellent team at Actimed to help drive the further progress of this exciting candidate and the company.” *** About Actimed Therapeutics Actimed Therapeutics is a clinical stage speciality 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 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, with further clinical studies in preparation. Actimed is currently preparing for further clinical studies in cancer cachexia having received an Investigational New Drug (IND) approval from FDA for S-pindolol benzoate in August 2023 for the treatment of cancer cachexia. Actimed also owns the global rights to its second asset, S-oxprenolol (ACM-002), 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 was granted Orphan Drug Designation to S-oxprenolol for the treatment of ALS by the FDA in 2024. 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 Frazer Hall, Erica HollingsworthTel: +44 (0)203 928 6900Email: actimed@medistrava.com ___________________________[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 139⁴ Wolf J et al., PMID 28184974 DOI: 10.1007/s00115-117-0293

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older. The decision, which allows patients with the rare muscle-wasting disorder to access Agamree on the NHS in Scotland, comes one month after the National Institute for Health and Care Excellence (NICE) recommended the drug for use on the NHS in England, Wales and Northern Ireland. Estimated to affect 2,500 males in the UK at any one time, DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles. The disease causes progressive muscle degeneration and weakness, and its major milestones are the loss of ambulation and self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. Agamree is a dissociative anti-inflammatory drug that works in a similar way to existing corticosteroids, the current standard of care for children and adolescents with DMD, but without the same safety concerns. The SMC’s decision follows the Medicines and Healthcare products Regulatory Agency’s approval of the therapy in January 2024, which was supported by results from the pivotal VISION-DMD study and three open-label studies. In VISION-DMD, patients treated with Agamree on average maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on average experienced growth stunting. The trial also showed that patients who switched from prednisone to Agamree after 24 weeks were on average able to resume growing in height over the remainder of the study. Additionally, unlike corticosteroids, Agamree did not result in a reduction of bone metabolism or a significant reduction in bone mineralisation in the spine after 48 weeks in the clinical studies. Commenting on the latest authorisation for the therapy, Santhera’s chief executive officer, Dario Eklund, said: “I am delighted that the team has secured this latest approval, which will ensure Scottish patients can benefit from this important treatment for DMD. “We will work closely with NHS Scotland, along with all the key regulatory and health authorities in all our approved markets, to ensure patients have access to Agamree.”