Category: News

• Trial to investigate the safety and efficacy of ARM210 for the treatment of Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
• Lead molecule ARM210 repairs ‘leaky’ Ryanodine Receptor (RyR) channels to restore intracellular calcium signaling in cardiac and musculoskeletal disorders

ARDSLEY, N.Y., June 21, 2023 – ARMGO^® Pharma, Inc. (ARMGO), a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs known as Rycals^®, announced today the enrollment of the first patient in a Phase 2 proof of concept clinical trial using its Rycal ARM210 (also known as S48168), for the treatment of Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). The trial, performed at the Amsterdam University Medical Center (Amsterdam UMC), Netherlands and the Mayo Clinic, Rochester MN, USA will investigate the safety and efficacy of ARM210 in CPVT.

CPVT is a rare genetic heart disease causing arrhythmia. It affects 1:10,000 people with a fatality rate of 30-50% by the age of 40 if left untreated. CPVT is caused by dysregulation of intracellular calcium handling in cardiomyocytes resulting mainly from mutations in Ryanodine Receptor 2 (RyR2), an intracellular calcium-release channel. Such mutations render RyR2 channels leaky, leading to inappropriate channel opening during periods of exercise or stress, resulting in severe and often fatal arrhythmias.

ARM210 is a potential disease modifying therapy for CPVT as it repairs leaky RyR2 channels. By binding and stabilizing the leaky channel, ARM210 can restore normal function, as demonstrated in animal models and in high resolution structures of mutant RyR2. In addition to CPVT, this unique mechanism of ARM210 has potential use in other RyR-mediated cardiac and skeletal muscle diseases, such as the genetic muscle disorder Ryanodine Receptor 1-Related Myopathy (RYR1-RM).

“This trial represents an important milestone for ARMGO and the CPVT community, potentially providing a disease modifying treatment for CPVT and validating ARM210’s unique mechanism of action for Ryanodine Receptors” stated Gene Marcantonio, M.D. Ph.D., Chief Executive Officer of ARMGO Pharma. “We are looking forward to advancing this important clinical program in partnership with the leading CPVT experts Arthur Wilde, M.D., Ph.D. and Michael Ackerman, M.D., Ph.D. and we are confident in the success of this trial. In addition, we are building our portfolio to strengthen ARMGO’s position as a pioneer of Rycal-based treatments for Ryanodine Receptor-related disorders.”

ARMGO was granted orphan drug designation as well as rare pediatric disease designation by the FDA in 2020 for the use of ARM210 as a potential treatment for patients with CPVT. The phase 2 trial is supported in part by an Orphan Products Development (OPD) grant from the FDA (1R01FD007279). Further information about the trial can be found at https://clinicaltrials.gov/ with identifier – NCT05122975.

About ARMGO Pharma

ARMGO Pharma, Inc., is a privately held biopharmaceutical company dedicated to applying targeted mechanism-based science to the development of novel small-molecule therapeutics to treat cardiac, and musculoskeletal disorders characterized by leaky Ryanodine Receptor (RyR) calcium channels. Leaky RyRs are caused by genetic mutations as well as post-translational modifications of RyR channels. The company’s proprietary drugs, known as Rycals, are a new class of oral agents that repair calcium leaks through the RyR. ARMGO Pharma has an exclusive, worldwide license from Columbia University for its RyR technology based on the research of founding scientist Andrew R. Marks, M.D.

For more information, please visit http://www.armgo.com or contact:

Media contact

Zyme Communications
Sarah Jeffery
Tel: +44 (0)7771 730919
E-mail: sarah.jeffery@zymecommunications.com

ARMGO Pharma Inc.
E-mail: info@armgo.com

• Series B investment led by Forbion and joined by Pontifax and Kurma Partners
  
• Investment will fund clinical studies of ARMGO’s lead molecule ARM210, an oral treatment, in development for cardiac and skeletal muscle diseases 

New York, USA – December 20 2021: ARMGO Pharma Inc (“ARMGO”), a leading novel small molecule therapeutics company developing treatments for cardiac, musculoskeletal, and neurological disorders today announced that it has completed a $35 million financing led by Forbion. Proceeds will fund further clinical development of its lead asset, ARM210, for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) as well as other cardiac and skeletal muscle indications. 

Forbion is a dedicated European life sciences venture capital firm that manages over €1.8 billion of investments and works closely with entrepreneurs to build life sciences companies that will change the future of medicine. In the financing, Forbion was joined by further top VC investors Pontifax and Kurma Partners. 

Dr. Geert-Jan Mulder and Dr. Dmitrij Hristodorov from Forbion, Dr. Iyona Rajkomar from Pontifax and Dr. Peter Neubeck from Kurma will join the ARMGO Board of Directors.  

The investment will fund Phase 2 clinical studies, commencing later this year, to evaluate ARM210 for the treatment of CPVT, a rare form of ventricular tachycardia and sudden death caused by mutations in the ryanodine receptor 2 (RyR2) which controls Ca2+ homeostasis in cells. Based on the work of ARMGO’s founder, Professor Andrew Marks from Colombia University, it is known that mutated RyR2 channels become leaky and lead to a severe form of ventricular arrhythmia. The current cornerstone therapy for CPVT is beta-blockers that reduce heart rate but do not repair the leaky channels that cause the arrythmia seen in most cases of CPVT. Beta-blockers leave a significant proportion of patients with residual arrhythmic burden that increases the risk for sudden cardiac death. Regardless of their efficacy, beta-blockers also cause strong side effects that significantly reduce the quality of life of these patients. To address both unmet needs, ARM210, a potentially disease-modifying, once daily oral pill, that repairs leaky RyR2 channels and restores physiological Ca2+ homeostasis will be tested in CPVT patients. ARM210 has successfully passed extensive preclinical development and human safety studies and is considered efficacious and well tolerated.  

ARMGO will initially aim to develop ARM210 in CPVT to provide a positive clinical proof-of-concept for the mechanism of action which will further de-risk the development of ARM210 in other diseases that are driven by dysregulated Ca2+ homeostasis. These studies will build on an ARM210 Phase 1b trial in patients with mutations in RyR1 (RyR1 related myopathy) ongoing at the National Institute of Health. Proceeds from the financing will be allocated to select further cardiac and skeletal muscle indications, ultimately building a pipeline-in-a-product for ARM210.  

Gene Marcantonio MD PhD, President and Chief Medical Officer of ARMGO, commented:
“We are pleased to have Forbion leading this financing round alongside Pontifax and Kurma Partners. They are all experienced healthcare investors that see the potential of ARMGO’s approach to provide disease-modifying treatments for patients by restoring the physiological Ca2+ homeostasis in affected tissues. We are excited to drive development of ARM210 in multiple indications and for the potential to reach significant milestone achievements within the next two years.”  

Geert-Jan Mulder and Dmitrij Hristodorov from Forbion commented: “We are pleased to lead this financing round and look forward to working together with Gene Marcantonio, his team and our co-investors in restarting and building ARMGO to reach potential clinical value inflection points over the coming years. ARMGO has done an excellent job in de-risking ARM210 through preclinical and early clinical development activities and it is now ready to be tested in patients.” 

Dr. Peter Neubeck of Kurma added: “We are excited to join the ARMGO team, Forbion and Pontifax in advancing ARMGO’s unique approach of modulating ryanodine receptors to treat high unmet need diseases with CPVT being an ideal initial clinical proof-of-concept indication.” 

-Ends- 

About Forbion www.forbion.com 

Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands, Germany and Singapore. Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space. 

Forbion manages well over EUR 1.8 billion across multiple fund strategies that cover all stages of (bio-)pharmaceutical drug development. Forbion’s current team consists of 20 life sciences investment professionals that have built an impressive performance track record since the late nineties with successful investments in over 70 companies. The firm is a signatory to the United Nations Principles for Responsible Investment. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients. Its investors include the EIF, through its European Recovery Programme (ERP), LfA, Dutch Venture Initiative (DVI), AMUF and EFSI facilities and KfW Capital through the Programme, “ERP – Venture Capital Fonds investments”. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.  

About Kurma Partners www.kurmapartners.com 

Founded in July 2009, Kurma Partners is key European player in the financing of innovation in healthcare and biotechnology, from pre-seed to growth capital, notably through Kurma Biofund I through III and Kurma Diagnostics, as well as via strategic partnerships with prestigious European research and medical institutions. Innovative projects from Academia and research institutions, aspiring to meet unmet medical need is an important pillar of the firm’s investment strategy, a second is venture capital financings of innovative young companies in Biotech and MedTech; all with a strong focus on innovative drug development approaches. 

ARDSLEY, N.Y., December 17, 2019 –ARMGO Pharma, Inc., a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs known as Rycals®, announced today the start of a clinical trial using its Rycal ARM210 (also known as S48168), for the treatment for patients with Ryanodine Receptor Type 1 Related Myopathies (RYR1-RM). The trial is being performed in collaboration with the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institute of Nursing Research (NINR) at the National Institutes of Health (NIH). In 2018, the FDA granted orphan drug designation to ARMGO for ARM210 as a potential treatment for patients with RYR1-RM.  

ARM210 is a potential disease modifying therapy for genetic diseases, that targets the Ryanodine Receptor calcium channel (RyR), an intracellular calcium-release channel that becomes leaky in these and other diseases. Intracellular calcium leaks via mutant RyR1 channels impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.  

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential disease modifying therapy for RYR1-RM. Muscle biopsies of the patients in this trial have been previously shown to respond biochemically to ARM210 in vitro. This trial will evaluate the safety and explore the biochemical effect of oral administration of ARM210 in these same patients.  

“This trial represents an important achievement for the Rycal® ARM210 program as we hope to provide effective treatment for patients with RYR1-RM, for which there are no approved therapies,” said Gene Marcantonio MD PhD, President and Chief Medical Officer of ARMGO Pharma. “We are looking forward to continuing the progress of the ARM210 clinical program, building on success in RYR1-RM patients to address other genetic mutations of RYR 1 and 2.” Further information can be found at https://clinicaltrials.gov/ Identifier: NCT04141670. 

ARDSLEY, N.Y., September 5, 2018 –ARMGO Pharma, Inc., a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs known as Rycals®, announced today that the  U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ARM210 (also known as S48168), a potential treatment for patients with Ryanodine Receptor Type 1 Related  Myopathies (RYR1-RM). In 2015, the FDA granted orphan drug designation and rare pediatric disease designation to ARM210 as a potential treatment for Duchenne Muscular Dystrophy (DMD).  

ARM210 targets the Ryanodine Receptor calcium-release channel(RyR), an intracellular calcium release channel that becomes leaky in a number of diseases including DMD and RYR1-RM, contributing to muscle damage and loss of function. RYR1-RM comprise a group of rare skeletal muscle diseases due to mutations in the ryr1 gene, which lead to leaky channels. These leaks both impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle,  causing the symptoms in RYR1-RM. 

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential therapy for RYR1-RM.  

“This designation represents an important achievement for the Rycal® ARM210 program and highlights the unmet need to provide effective treatment for patients with RYR1-RM,” said Gene Marcantonio MD Ph.D., President and Chief Medical Officer of ARMGO Pharma. “We are looking forward to continuing  the progress of the ARM210 clinical program, including upcoming studies in RYR1-RM patients.” 

About Orphan Drug Designation  

Orphan Drug Designation is granted by the FDA to drug candidates intended to treat diseases affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor to apply for FDA  orphan research grants, waiver of Prescription Drug User Fee Act filing fees, tax credits for clinical research costs, and a seven-year period of market exclusivity upon approval of the drug.  

About ARMGO Pharma 

ARMGO Pharma, Inc., is a privately held biopharmaceutical company dedicated to applying targeted mechanism-based science to the development of novel small-molecule therapeutics to treat cardiac,  musculoskeletal, and neurological disorders. The company’s proprietary drugs, known as Rycals®, are a  new class of oral agents that repair the calcium leak through the RyR, which is located on the sarcoplasmic/endoplasmic reticulum. ARMGO Pharma has been awarded an exclusive, worldwide license from Columbia University for its RyR technology. Development and commercial rights for  ARMGO’s Rycal drugs in cardiovascular and skeletal muscle indications outside of the US and Japan are exclusively licensed to Les Laboratoires Servier (Servier). Development of ARM210 has been supported through a research collaboration with Servier, and an award from the Muscular Dystrophy  Association (MDA USA).