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BASEL, Switzerland, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Noema Pharma, a clinical-stage biotech company targeting debilitating central nervous system (CNS) disorders, today announced the appointment of Volker Knappertz, M.D., as Executive Vice President of Research & Development. “We are excited to welcome Volker to Noema. Volker is a recognized leader in drug development with a proven track record building and leading Research and Development organizations and obtaining market approvals for novel drugs, including Epidiolex for the treatment of rare developmental childhood onset forms of epilepsy,” said Ilise Lombardo, M.D., Chief Executive Officer of Noema Pharma. “His experience and expertise across CNS development will be pivotal as Noema enters a transformational year with readouts of multiple Phase 2 studies across our pipeline.” “I am thrilled to join Noema and look forward to the continued clinical development across the company’s multiple indications including seizures in Tuberous Sclerosis Complex, pain in Trigeminal Neuralgia, Tourette syndrome and vasomotor symptoms plus CNS-mediated symptoms of menopause,” said Dr. Knappertz. “Noema’s commitment to patients and innovative approach to CNS disorders is proven by their late-stage clinical pipeline and I look forward to working with the team to bring these first-in-disease therapeutics to patients in need.” Dr. Volker Knappertz has spent over 25 years in drug development and has been involved in numerous Investigational New Drug (IND) filings and product approvals during his career. Prior to joining Noema, Dr. Knappertz served as Executive Vice President of Research & Development (R&D) at Aurinia Pharmaceuticals, where he led the entirety of R&D across multiple programs targeting autoimmune diseases. Dr. Knappertz also served as Executive Vice President of R&D and Chief Medical Officer at GW Pharmaceuticals (acquired by Jazz Pharmaceuticals). During his time at GW, Dr. Knappertz was instrumental in driving the transformation of GW from a botanical pharmaceutical company to a fully integrated biopharmaceutical company following the FDA approval of Epidiolex for the treatment of rare developmental childhood onset forms of epilepsy. In addition, he played a role in several drug approvals and major business development deals during his tenure in leadership positions at Teva Pharmaceuticals, AstraZeneca and Bayer. Dr. Knappertz received his doctorate and Medical Degree from Cologne University and is a US board certified neurologist. He has served as Adjunct Professor in Neurology at the University of Pennsylvania and Heinrich-Heine University in Düsseldorf. About Noema PharmaNoema Pharma is a clinical-stage biotech company advancing a portfolio of transformative, first-in-disease therapeutics targeting neuroscience-based conditions with high unmet need. Noema has four programs currently in active Phase 2 clinical trials evaluating seizures in Tuberous Sclerosis Complex, pain in Trigeminal Neuralgia, Tourette syndrome and vasomotor symptoms plus CNS-mediated symptoms of menopause with readouts expected in 2025. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including EQT Life Sciences, Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com. Investor ContactArgot Partners Maeve Conneighton 212-600-1902noema@argotpartners.com

NMD Pharma publishes new data on the role of ClC-1 inhibition in Charcot-Marie-Tooth in the Annals of Clinical and Translational Neurology Results from the ESTABLISH1 observational study confirms that neuromuscular junction (NMJ) dysfunction is an underappreciated disease characteristic in patients with Charcot-Marie-Tooth disease (CMT) types 1 and 2 affecting muscle function in patientsAdditionally, results from preclinical studies provide proof-of-mechanism for recovery of muscle function with ClC-1 inhibition using NMD670 in CMT mouse models Aarhus, Denmark, 18 December 2024 – NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today published new data in the journal Annals of Clinical and Translational Neurology with collaborators from the Aarhus University Hospital, University of Missouri, The Ohio State University, and Aarhus University. The peer-reviewed paper, entitled ‘Neuromuscular Transmission Deficits in patients with CMT and ClC-1 Inhibition in CMT animal Models,’ provides evidence that targeting the neuromuscular junction (NMJ) with ClC-1 inhibitors, such as NMD670, could enhance muscle function in patients with CMT, a rare neuromuscular disease. Clinical features of CMT include motor signs and symptoms such as muscle weakness, muscle atrophy, and fatigue, as well as sensory deficits that, in combination, cause substantial reduction in quality of life. Preclinical studies have previously implicated NMJ transmission deficits in muscle dysfunction in CMT. This study describes results from ESTABLISH1, an observational clinical study in CMT Type 1 and 2 patients and healthy patients in the US and Denmark, which confirms that patients with CMT, regardless of genotype, have notable NMJ transmission dysfunction that correlates with disease severity. In addition, the paper describes that the treatment of mouse models of CMT Type 1 (CMT1A) and CMT Type 2 (CMT2D) following partial inhibition of ClC-1 chloride channels and the application of nerve-stimulated muscle force, was shown to improve muscle contractile function with the addition of the novel small molecule ClC-1 inhibitor NMD670. W. David Arnold, MD, Principal Investigator who led the international ESTABLISH1 observational study, commented: “With no currently approved therapies to provide a cure or alleviate symptoms, data from the ESTABLISH and preclinical studies represents a significant milestone in the development of a treatment. Here, the data shows that patients with CMT, regardless of genotype, are characterized by notable NMJ transmission dysfunction, and that the level of dysfunction directly correlates with the level of disease severity, further confirming the potential therapeutic value of ClC-1 inhibition to address the muscle weakness and fatigue associated with CMT.” Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, added: “This data provides further evidence of the crucial role of neuromuscular junction dysfunction in CMT patients. The impaired connectivity between the motor neuron and skeletal muscle at the neuromuscular junction in CMT patients presumably occurs because the axons are in a process of degeneration or are newly formed or forming neuromuscular junctions from axons nearby degenerated connections. It is very promising that NMD670 improves muscle contractile function in animal models of CMT, and we have already started evaluating the effect of NMD670 in a Phase 2 clinical trial which we initiated in November 2024.” The full manuscript is available online through the following link: Neuromuscular transmission deficits in patients with CMT and ClC‐1 inhibition in CMT animal models - Grønnebæk - Annals of Clinical and Translational Neurology - Wiley Online Library On 18 November, NMD Pharma announced that it had dosed the first CMT disease patient in its Phase 2 clinical trial of NMD670. NMD Pharma has three ongoing global clinical trials investigating NMD670 across rare neuromuscular diseases characterized by a high degree of patient impact and need, including a Phase 2 study in adults living with spinal muscular atrophy type 3 and a Phase 2b study in generalized myasthenia gravis patients. Further information on the study can be found here: Study Details | Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease | ClinicalTrials.gov Exploring SynapTic ABnormaLitIeS in Hereditary neuropathies -END- Contacts NMD Pharma A/SDan Brennan, SVP Corporate and Commercial StrategyE-mail: contact@nmdpharma.com ICR HealthcareMary-Jane Elliott / Ashley Tapp / Lindsey Neville E-mail: NMDPharma@icrhealthcare.comTel: +44 (0)20 3709 5700 About NMD PharmaNMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of rare neuromuscular disorders and age-related neuromuscular diseases with high levels of patient unmet. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at http://www.nmdpharma.com. About NMD670NMD670 is NMD Pharma’s lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle’s responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. This novel treatment approach has resulted in clinical evidence of CIC-1 inhibition in myasthenia gravis and preclinical evidence in spinal muscular atrophy, Charcot-Marie Tooth disease and sarcopenia. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of gMG. About Charcot-Marie-Tooth disease (CMT)CMT encompasses a group of hereditary sensory and motor neuropathies that cause damage to peripheral nerves and their neuromuscular junction. Damage caused by CMT worsens slowly over time and can result in substantial reductions in mobility, independence and quality of life due to muscle weakness, fatigue and atrophy across skeletal muscle groups such as the legs, feet, arms, hands and potentially leading to diaphragm weakness and paralysis. CMT is often broadly grouped into demyelinating (CMT type 1) and axonal (CMT type 2) based on nerve conduction studies, but there are more than 160 subtypes of CMT based on their genetic causes, clinical features and progression patterns. CMT affects approximately 136,000 individuals in the United States and 3.2 million worldwide, with first symptoms typically appearing during adolescence or early adulthood.

Proceeds will be used to enable key late-stage clinical trial readouts for assets across several underserved neurological diseases with high unmet medical need. EQT Life Sciences has led the extension, and joins a consortium of prominent global biotechnology investors. The investment builds on EQT Life Sciences' previous successful partnership with neurology-focused repeat entrepreneur and Noema Pharma CEO Ilise Lombardo, MD STOCKHOLM, Dec. 11, 2024 /PRNewswire/ -- EQT Life Sciences is pleased to announce that the LSP 7 fund has invested in Noema Pharma ("the Company"). The clinical-stage biotech company headquartered in Basel, Switzerland is developing a pipeline of first-in-disease therapeutics that address central nervous system (CNS) disorders. The Series B extension brings the total capital raised in the round to CHF 130 million, including prior investments from Forbion, Jeito Capital, Sofinnova Partners, Gilde Healthcare, Polaris Partners, Invus and UPMC Enterprises. CNS disorders are a significant area of unmet medical need, affecting hundreds of millions of people worldwide who often face inadequate treatment options with limited effectiveness and significant side effects. Many of the conditions targeted by Noema Pharma have historically been difficult to treat due to their complex nature and the lack of effective therapies. By leveraging its diverse portfolio of neurological assets and innovative clinical pipeline, Noema Pharma is strongly positioned to address these debilitating disorders and provide much-needed hope and transformative solutions to patients living with these life-altering challenges. The Company's portfolio includes four active Phase 2 trials, with key readouts from all studies anticipated in 2025. Proceeds from the financing will drive the continued development of Noema Pharma's clinical-stage assets, including its lead candidate basimglurant (NOE-101), a mGluR5 inhibitor currently in Phase 2 trials for severe pain in trigeminal neuralgia (TN) and seizures in tuberous sclerosis complex (TSC). Additionally, the Company is advancing gemlapodect (NOE-105), a PDE10a inhibitor in Phase 2 for Tourette syndrome and childhood onset fluency disorder (COFD or stuttering), as well as NOE-115, a broad-spectrum monoamine modulator in a Phase 2 trial for vasomotor symptoms and additional symptoms of menopause. Ilise Lombardo, MD, CEO of Noema Pharma, stated: "We are thrilled to welcome EQT Life Sciences as a lead investor and to have Felice join our Board. Their support and expertise will be invaluable as we progress our clinical programs and strive to make a meaningful impact on patients' lives." Felice Verduyn-van Weegen, Partner at EQT who is joining the Company's Board of Directors, commented: "Noema Pharma's innovative approach to CNS disorders aligns very well with our investment strategy and we are excited to support their late-stage clinical pipeline and transformative therapies. Having worked with Ilise Lombardo on a previous successful investment, her exceptional leadership as a repeat entrepreneur further reinforces our confidence in Noema's potential to deliver meaningful impact to patients in need." Contact EQT Press Office, [email protected] This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED