RYR1

Alongside its rebrand, RyCarma Therapeutics unveiled a new pipeline program to develop its lead candidate, ARM210, in heart failure with reduced ejection fraction. Armgo Pharma is making a fresh start this new year, as the Boston-based biotech announced Tuesday that it will henceforth be known as RyCarma Therapeutics.The new name draws from the company’s nearly two decades of work in ryanodine receptor (RyR) biology, which it has used to develop a new class of small-molecule drugs dubbed Rycals, with the help of RyR technology licensed exclusively from Columbia University and stemming from the research of RyCarma founder Andrew Marks, M.D.Alongside the rebrand, RyCarma also unveiled a new pipeline program to develop its lead candidate, ARM210, in heart failure with reduced ejection fraction. If ultimately approved, per the company, it stands to become the first heart failure therapy that not only improves heart function but also addresses the common symptom of skeletal muscle weakness. ARM210 is also being tested in the much rarer setting of ryanodine receptor 1-related myopathies (RYR1-RM), which comprise neuromuscular diseases caused by mutations in the RYR1 gene.In another set of changes for the biotech, RyCarma also named a handful of new executives, including the appointments of CEO Adam Rosenberg and of Jonathan Alspaugh and Sanjay Jalota to serve as chief strategy officer and senior vice president of regulatory, respectively.Marks said in a statement that he was “thrilled” with the new direction of the company he founded in 2004.“The past decade has seen an incredible convergence of structural, genetic and physiological data on RyR biology," he added. "RyCarma is at the forefront of advancing therapeutics that target RyR to treat serious and devastating diseases like RYR1-RM and heart failure." RyCarma has previously dabbled in applying its Rycals to cardiac conditions: In its early days, one candidate reached phase 2 trials in heart failure and arrhythmias before being scrapped in favor of a focus on neuromuscular disorders.In 2021, the company then known as Armgo brought in $35 million from Forbion and other VC backers that it said would be used to test ARM210 in a phase 2 trial of catecholaminergic polymorphic ventricular tachycardia, a rare inherited cardiovascular disease that’s characterized by abnormal heart rhythm. At the time, the biotech pitched ARM210 as a potential “pipeline-in-a-product.”As of this week, however, only the heart failure and RYR1-RM programs are listed as ARM210’s ongoing projects on RyCarma’s site.Editor's note: This story was updated to correct a mention of Armgo's founding year. It was founded in 2004, not 2006.

BOSTON--( BUSINESS WIRE )-- RyCarma Therapeutics, Inc. , formerly known as ARMGO Pharma (the “Company” or “RyCarma”), a clinical-stage biotech company developing first-in-class small molecule therapeutics for cardiovascular and skeletal muscle diseases, today unveiled its new leadership and company name change from “ARMGO Pharma, Inc.” to “RyCarma Therapeutics, Inc.” and introduced a new program in heart failure. The name RyCarma references the Company’s foundational work and leadership in ryanodine receptor (RyR) biology and in establishing the therapeutic potential of Rycals ® , a novel class of small molecule drugs. RyCarma’s new program focuses on developing its lead candidate ARM210 in heart failure with reduced ejection fraction, which affects millions of people worldwide. ARM210 is also currently being investigated in ryanodine receptor 1-related myopathies (RYR1-RM), a group of rare neuromuscular diseases caused by mutations in the RYR1 gene. The Company also introduced several new members of its leadership team, who bring a track record of success in leading biotech companies and guiding novel biological discoveries through drug development. Adam Rosenberg, Chief Executive Officer, most recently served as the chief executive officer of Aliada Therapeutics and Sionna Therapeutics and brings decades of experience leading innovative biotechnology companies. Also joining the leadership team are Jonathan Alspaugh, President & Chief Strategy Officer, who brings 15 years guiding biotech and biopharma companies on financial and strategic matters and Sanjay Jalota, Senior Vice President of Regulatory, an experienced regulatory strategist steering both early- and late-stage compounds through development. “RyCarma will expand on our long-term focus on the central role of RyRs in human biology, building on the pioneering work of our founder Dr. Andrew Marks, a globally recognized leader in RyR science, who cloned the receptor and described its structure at atomic resolution,” said Adam Rosenberg, Chief Executive Officer of RyCarma. “Despite other therapeutic advancements in heart failure, there continues to be serious unmet need with high levels of mortality and hospitalizations. Our experienced team is committed to translating our expertise with the Rycal mechanism into innovative treatments for patients.” RyCarma’s lead candidate ARM210 is designed to repair leaky ryanodine receptors, ion channels that regulate the intracellular flow of calcium that is essential for muscle contraction. In a Phase 1b trial in patients with RYR1-RM, ARM210 showed improvement of fatigue and proximal muscle strength, and was well-tolerated with no dose-dependent safety signals or serious adverse events. For patients with heart failure, ARM210 has the potential to be the first therapy that directly repairs leaky RyRs in both cardiac and skeletal muscle, potentially improving heart function as well as skeletal muscle weakness, a common symptom that is not addressed by existing treatment options. “I am thrilled with the new strategic direction of RyCarma, the additions to the leadership team and the potential for Rycals first-in-kind mechanism,” said Dr. Andrew Marks. “The past decade has seen an incredible convergence of structural, genetic and physiological data on RyR biology. RyCarma is at the forefront of advancing therapeutics that target RyR to treat serious and devastating diseases like RYR1-RM and heart failure.” About RyCarma Therapeutics RyCarma (formerly ARMGO Pharma) is a clinical-stage biotech company developing first-in-class small molecule therapeutics for cardiovascular and skeletal muscle diseases. RyCarma’s drug discovery platform is built from deep expertise on ryanodine receptors (RyRs), channels that regulate the intracellular flow of calcium ions in nearly all cell types. RyCarma holds an exclusive license from Columbia University for its RyR technology based on the research of the Company’s founder, Dr. Andrew Marks. RyCarma’s lead program, ARM210, is in development for the treatment of heart failure and ryanodine receptor 1-related myopathies (RYR1-RM). For more information, visit www.rycarma.com .

AMSTERDAM, March 26, 2024 /PRNewswire/ -- Norgine B.V. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of NPJ5008 (dantrolene sodium hemiheptahydrate) for the treatment of malignant hyperthermia. The European Commission (EC) will now review the CHMP's recommendation. If approved, the centralized marketing authorization would be valid in all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway and would be the first new treatment in Europe for malignant hyperthermia in over forty years. Submissions to the UK MHRA and Swiss Medic are ongoing. Dr David Gillen, Chief Medical Officer of Norgine, added: "We are delighted to have received a positive CHMP opinion for NPJ5008. Malignant hyperthermia (MH) is thankfully a rare medical emergency following anaesthesia. Nevertheless, it is clear that effective management of MH is critically important to improve clinical outcomes. NPJ5008 will allow faster administration compared to the existing dantrolene formulation and therefore, we believe, better outcomes for all European patients with MH." For further information please contact: [email protected] Follow us @norgine Notes to Editors: About Malignant Hyperthermia Malignant hyperthermia (MH) is a rare life-threatening pharmacogenetic disorder of skeletal muscles that presents as an exaggerated hypermetabolic response to volatile anaesthetic agents and depolarizing muscle relaxants.[1] About NPJ5008 NPJ5008 is a novel formulation of dantrolene that has been developed by Norgine to shorten preparation and administration times versus DANTRIUM IV (dantrolene sodium). Each vial of NPJ5008 contains 120mg of dantrolene and is reconstituted in 20 mL of water for injections for intravenous administration.[2] About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References: Kaur et al. Mo Med. 2019;116(2):154–159. Ng Kwet Shing RH, et al. Eur J Anaesthesiol. 2024;41:1–10. Logo: