Faraday Pharmaceuticals, Inc.

London, UK – 4th 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. *** 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. FOR MORE INFORMATIONActimed Therapeuticswww.actimedtherapeutics.com MEDiSTRAVA ConsultingFrazer Hall, Erica HollingsworthTel: +44 (0)203 928 6900Email: actimed@medistrava.com ___________________________ 1Suzuki et al, JCSM 2018; 9, 183-212. (Poster 5-17, page 200)2 Anker M et al., J. Cachexia, Sarcopenia and Muscle; 2019: 10: 22 – 243 Argilés JM et al, Nat Rev Cancer 2014; 14:754-624 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 1395Wolf J et al., PMID 28184974 DOI: 10.1007/s00115-117-0293

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.”

SEATTLE, July 16, 2024 (GLOBE NEWSWIRE) -- Faraday Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on the prevention of heart failure by reducing myocardial damage following acute ST-elevation myocardial infarction (STEMI) through the reduction of reperfusion injury during percutaneous coronary intervention (PCI), today announced it will host a virtual KOL webinar on Tuesday, July 30, 2024, at 11:00 AM ET. To register for the webinar, click here. The webinar will feature Dr. Cheerag Shirodaria (Oxford University Hospitals), Dr. Marc Bonaca (University of Colorado Anschutz), and Dr. Antonio Gutierrez (Duke University Hospital and Durham VA Medical Center), who will discuss the unmet need and current treatment landscape in preventing ischemia-reperfusion injury and highlight the effects of STEMI on heart failure and other patient outcomes. The webinar will also review data from the development of FDY-5301, an investigational product being evaluated by Faraday in minimizing cardiac damage following acute STEMI. The company’s pivotal Phase 3 Iocyte AMI-3 trial of FDY-5301 in anterior STEMI patients undergoing primary percutaneous intervention is now fully enrolled with topline data anticipated in 2H 2025. A live question and answer session will follow the formal presentations. About Cheerag Shirodaria, BSc, MBBS, MD, FRCP, MBA Dr. Cheerag Shirodaria is Chief Development Officer at Caristo Diagnostics and a Cardiologist at Oxford University Hospitals in the UK. He is an interventional cardiologist who is also an expert in cardiac imaging, particularly cardiac MRI (CMR) and coronary CT angiography (CCTA), having completed his doctoral thesis in Oxford on novel vascular imaging techniques using CMR. He was heavily involved in the design and execution of the phase 2 STEMI trial involving FDY-5301 and is a member of the Executive Steering Committee of the IOCYTE-AMI trial. As co-founder and former CEO of Caristo, he has developed and commercialized novel imaging AI imaging tools to predict future cardiometabolic risk. Dr Shirodaria obtained his medical degree from St Bartholomew's Hospital Medical College, his medical doctorate from the University of Oxford and his MBA from London Business School. About Marc Bonaca, MD, MPH Dr. Marc Bonaca is a cardiologist and vascular medicine specialist serving as executive director of CPC and Director of Vascular Research & Professor of Medicine at the University of Colorado Anschutz. Dr. Bonaca earned his medical degree from the University of Connecticut and his MPH at Harvard. He completed his medical residency, cardiology fellowship, and vascular medicine fellowship at Brigham and Women’s Hospital and a dedicated research fellowship at the TIMI Study Group. He became faculty at BWH and Harvard Medical School and an investigator at TIMI. He directed pharmacovigilance at TIMI and was an investigator on several large outcomes trials including TRA2P-TIMI 50, PEGASUS-TIMI 54, DECLARE-TIMI 58, and REAL TIMI 63B. In 2018, he joined the faculty at the University of Colorado as Professor of Medicine and the William R. Hiatt Endowed Chair in Cardiovascular Research. He is the Executive Director of CPC, an affiliated non-profit Academic Research Organization. At CPC, he has led several clinical trials with CPC as the lead ARO, including VOYAGER PAD, PREVENT HD, and ASPEN, and is leading several ongoing trials, including BRIGHT and EVOLVE MI, a 4000-patient pragmatic multinational acute coronary syndrome trial. Dr. Bonaca’s research focus is on ischemic risk with atherosclerotic vascular disease, risk prediction, and risk modification using pharmacologic and biologic therapies. He has extensive experience designing and conducting large, multicenter randomized clinical trials, and analyzing registries and real-world datasets. His areas of interest include PAD, PVD, and diabetes, with a focus on the breadth of risk including ischemic limb outcomes, microvascular complications, and major adverse cardiovascular events. He is also investigating the cardiac, vascular, and thrombotic complications associated with novel oncologic therapies. At CPC, he has focused on building a robust faculty and operational group dedicated to high quality, efficient trials leveraging health networks, informatics, and decentralized design. About J. Antonio T. Gutierrez, MD Dr. Antonio Gutierrez’s clinical interests include diagnostic and interventional coronary angiography, peripheral angiography and percutaneous intervention, and vascular medicine. His research interests are focused on peripheral artery disease. He has been involved in multiple clinical trials involving patients with atherosclerotic disease and has published several peer-reviewed articles and reviews. Dr. Gutierrez received his Bachelor of Arts from the College of the Holy Cross in 2000, Doctorate of Medicine from Case Western Reserve University School of Medicine in 2007, and Masters of Health Science in Clinical Research from Duke University School of Medicine in 2012. He completed his residency in Internal Medicine at Duke University Medical Center in 2010 and was appointed Chief Medical Resident of the Durham VA Medical Center in 2011. Dr. Gutierrez completed his General Cardiology and Vascular Medicine Fellowships at Brigham and Women’s Hospital and a Clinical Research Fellowship at the TIMI Study Group. Most recently Dr. Gutierrez matriculated his Interventional Cardiology Fellowship at Duke University Medical Center. Dr. Gutierrez is currently an Assistant Professor of Medicine at Duke University Hospital and Staff Physician at the Durham VA Medical Center. About STEMI Acute STEMI is a leading cause of cardiovascular death and remains a primary cause of the development of heart failure. Standard treatment of a STEMI involves PCI, during which a catheter is inserted into the artery to remove the blockage and restore blood flow. Following a STEMI, one of the critical factors influencing patient outcomes is reperfusion injury, which occurs when the oxygen-rich blood supply returns to the ischemic heart muscle. About FDY-5301 FDY-5301 is an elemental reducing agent containing sodium iodide for which Faraday has obtained method of use patent protection in major markets worldwide. Faraday has selected FDY-5301 for investigation in the belief that its properties are well-suited to mitigate ischemia-reperfusion injury (IRI). In preclinical IRI models, FDY-5301 reduced tissue damage, infarct size, and inflammation. FDY-5301 functions as a catalytic neutralizer of hydrogen peroxide, a prominent reactive oxygen species implicated in the IRI cascade leading to cardiomyocyte death, and also acts as an immunomodulating agent. A Phase 2 clinical trial of FDY-5301 in STEMI patients demonstrated that it was well-tolerated and provided encouraging signals of potential efficacy in minimizing cardiac damage. Results from that trial — known as Iocyte AMI — were reported in the January 15, 2022, issue of the International Journal of Cardiology. About Faraday Pharmaceuticals, Inc. Faraday Pharmaceuticals® is a clinical-stage biopharmaceutical company focused on the prevention of heart failure by reducing myocardial damage in acute STEMI. The company was founded by Dr. Mark Roth of the Fred Hutch Cancer Center and is backed by an investor group led by ARCH Venture Partners and Polaris Partners. The company’s lead program, FDY-5301, is in a pivotal Phase 3 trial and is designed to reduce IRI in acute STEMI, a leading cause of death and a primary cause of the development of heart failure. The company is headquartered in Seattle. For more information, visit www.faradaypharma.com or follow the company on LinkedIn. Contact:Brian BlackmanChief Financial Officerbblackman@faradaypharma.com Contact:PJ KelleherLifeSci Advisors, LLC+1-617-430-7579pkelleher@lifesciadvisors.com