Congenital Structural Myopathy

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 .

A 20-year-old biotech working on small molecule treatments for cardiovascular and skeletal muscle diseases has rebranded and plans to enter Phase 2 in heart failure later this year. Formerly known as ARMGO Pharma, the Boston biotech will move forward as RyCarma Therapeutics, the Forbion-backed company told Endpoints News . Advancing the nimble biotech are CEO Adam Rosenberg, and president and chief strategy officer Jonathan Alspaugh. Rosenberg was previously CEO of Aliada Therapeutics, a neuroscience biotech recently bought by AbbVie for $1.4 billion, and is chair of VectorY Therapeutics , Seamless Therapeutics and Ambagon Therapeutics . Alspaugh was previously president of Aeglea , which is now Spyre. ARMGO was formed in 2004 based on research conducted by Columbia University professor Andrew Marks, who’s worked for decades in the cardiovascular space. The biotech builds off Marks’ work in the field of ryanodine receptors. “When we’ve spoken to cardiologists about the mechanism here, they actually get really excited because of how easy it is to actually articulate,” Alspaugh said in an interview. The company’s small molecules are allosteric modulators of the ryanodine receptor. “The receptors are leaky due to cellular stress or genetic aberrations, and this stabilizes the channel and allows it to return to normal function,” he said. “This, in contrast to something like SGLT2 inhibitors — which are amazing drugs for heart failure patients, [but] cardiologists still don’t fully understand how they work.” The goal is to get ARM210 into a Phase 2 trial at the end of this year that tests the drug in patients with heart failure with reduced ejection fraction, according to Alspaugh. The exact details of the trial are still being ironed out, Rosenberg said in the same interview. The biotech is also testing ARM210 in patients with ryanodine receptor 1-related myopathies, which are rare neuromuscular diseases. Rosenberg said his father, a doctor himself, has hypertrophic cardiomyopathy. “I’m unfortunately quite aware of the impact of cardiovascular disease,” he said. “When heart failure patients present, they don’t typically come in and say, ‘My heart is weak.’ They say, ‘I am weak.'” Alspaugh said the company is hopeful that ARM210 can help with weakness on multiple fronts. “This would be the first mechanism that could actually address impairments of the cardiac muscle and the skeletal muscle,” he added. RyCarma had a 14-year partnership with Servier. The companies eventually took an experimental medicine into multiple Phase 2 clinical trials, but the European pharma ended the collaboration a few years ago, Rosenberg said. Though it’s been around for two decades, RyCarma has raised only about $50 million to date, mainly from Forbion, Pontifax and Kurma Partners, said Rosenberg, who added that the biotech employs fewer than 10 people and has been capital-efficient. It also helped that Servier covered direct costs, supported research and funded three Phase 2 trials with the prior molecule, dubbed ARM036, according to Rosenberg. RyCarma recently did a bridge financing of about $12.5 million from its Series B investors, Alspaugh said. That will back the biotech “through this year,” he said. The rebrand includes “arm” as a nod to its original name. Those are the initials of Marks, the scientific founder.

Novartis is buying gene therapy and neuroscience biotech Kate Therapeutics in a deal worth $1.1 billion in upfront and milestone payments, the Swiss pharma confirmed to Endpoints News on Thursday. And Novartis is not done yet. It is still on the lookout for bolt-on M&A opportunities — but don’t expect them to go higher than $3 billion. “That’s not that we couldn’t do a little bit bigger, but that’s the majority of the available target companies, if you will,” Novartis CFO Harry Kirsch told the media ahead of its investor event in London on Thursday. CEO Vas Narasimhan added that a lot of M&A from the recent past has been in the $1 billion to $2 billion range, pointing to its acquisitions of Mariana Oncology and KateTx. Over the past eight years, it made $5 billion to $10 billion deals only “occasionally” because of valuation considerations and “financial discipline,” he added. Novartis will take on KateTx’s pipeline of preclinical candidates in Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1. The San Diego biotech’s gene therapy tech features adeno-associated virus capsids that deliver their payloads into specific tissues to avoid off-target issues such as hepatic side effects. In June 2023, it said that it was partnering with Astellas on a neuromuscular disease called X-linked myotubular myopathy. Novartis revealed its purchase of KateTx ahead of its Meet Novartis Management event in London that was intended to reassure investors its pure-play strategy is bearing fruit now and in the next several years. A key part of its growth strategy is more M&A as “replacement power,” with the company facing the loss of exclusivity for key drugs such as Entresto , Kirsch said. When asked if changes to the Federal Trade Commission could lead to Novartis raising its M&A budget, Narasimhan said the FTC is not a major driver of its decisions. As a part of its reorg, the company zeroed in on four therapeutic spaces: cardiovascular-renal-metabolic, immunology, neuroscience and oncology. “Preclinical, Phase 1, Phase 2 area — that’s the opportunity where we can step in” and Novartis can add value to the acquired company, Narasimhan said, adding that it’s challenging to “find synergies, harder to justify” with later-stage candidates. Narasimhan said that when it bought MorphoSys for $2.9 billion, its lead oncology candidate , pelabresib, was in an ongoing Phase 3 trial. “We saw the opportunity in an area of myelofibrosis that we’ve been in for a long period of time, and we took a bet on an ongoing clinical trial,” he said, adding that if it had waited for the late-stage readout, the purchase price would have been “double at least.” But that bet on MorphoSys led to the company taking an $800 million impairment hit just months after the deal closed due to emergent malignancies. “In this case, it didn’t go our way,” he said, nonetheless noting the company’s overall M&A track record . As for pelabresib, the company’s next update should come in the second half of next year. Narasimhan said it is monitoring patients until the middle of next year. “We are also planning to conduct additional studies to look at certain subpopulations of myelofibrosis patients where pelabresib could have a better benefit-risk profile. So I think all of that will take us at least until 2027 — it might take longer, depending on the safety topic,“ he added.