Muscle Spasticity

Announcement follows FDA's February 2025 decision to grant radiprodil Breakthrough Therapy designation Company remains on track to initiate Phase 3 pivotal trial of radiprodil in mid-2025 NEW YORK, March 17, 2025 /PRNewswire/ -- GRIN Therapeutics, Inc., a leader in the development of therapies to treat serious neurodevelopmental disorders, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation for its investigational drug, radiprodil, for the treatment of GRIN-related neurodevelopmental disorder (NDD). Radiprodil is designed as a potent negative allosteric modulator selectively targeting the N-methyl-D-aspartate receptor subtype 2B (NR2B or GluN2B). The company is on track to initiate a pivotal Phase 3 trial for radiprodil in mid-2025 for the treatment of GRIN-related NDD with GoF mutations and has an ongoing global open-label clinical trial of radiprodil for the treatment of tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II. "We are making rapid progress in our efforts to bring a first-ever treatment for GRIN-related neurodevelopmental disorder to patients. Supported by our promising clinical data, the FDA's decision to grant Orphan Drug designation to radiprodil is the latest milestone in that effort," said Michael A. Panzara, MD, MPH, Chief Medical Officer at Neurvati Neurosciences and GRIN Therapeutics. "As we plan to launch our pivotal Phase 3 clinical trial to evaluate the impact of radiprodil on seizures, behavioral abnormalities and functional outcomes associated with GRIN-related NDD in the coming months, we are very grateful to the patients and their families who continue to support this important research effort." Orphan Drug designation is designed to support innovation and research that can lead to more new treatments for diseases that affect fewer than 200,000 people in the United States. The designation grants drug developers the potential for seven years of market exclusivity for their drug after approval, during which time the FDA will generally not approve an application for the same drug targeting the same disease or condition. In February 2025, the FDA granted radiprodil Breakthrough Therapy designation for the treatment of seizures associated with GRIN-related NDD in patients with GoF mutations. "We look forward to continuing to engage with the FDA through the means offered by Breakthrough Therapy designation, as we advance our Phase 3 program with a goal of bringing radiprodil to patients," said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Senior Vice President, Development, Regulatory, and Quality at Neurvati Neurosciences and GRIN Therapeutics. "We have heard from so many clinicians and patients about the urgent unmet need in the treatment of GRIN-related disorders and our entire team is dedicated to advancing this development program through late-stage clinical research and regulatory review as rapidly as possible." About GRIN-related neurodevelopmental disorder GRIN-related neurodevelopmental disorder is a family of rare, genetically defined pediatric neurodevelopmental disorders caused by mutations in GRIN genes. While symptoms of GRIN-related neurodevelopmental disorder can present as early as infancy, a diagnosis is often not confirmed until age two or later when a child fails to reach developmental milestones. Individuals may experience developmental delay, intellectual disabilities, epilepsy, muscular hypotonia, movement disorders, spasticity, feeding difficulties and behavioral problems. There are currently no approved therapies for GRIN-related neurodevelopmental disorder. About Radiprodil Radiprodil is an investigational, selective and potent negative allosteric modulator of the N-methyl-D-aspartate (NMDA) receptor subtype 2B (NR2B or GluN2B). In nonclinical studies, radiprodil has been shown to potently and selectively modulate GluN2B. Radiprodil has also demonstrated an antiseizure effect in a number of in vitro and in vivo preclinical seizure and epilepsy models and specifically in models characterized by an enhanced GluN2B NMDA transmission, which can occur with gain-of-function (GoF) mutations in GRIN-related neurodevelopmental disorder. In vitro analysis of brain tissues extracted from both tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) lesions has shown enhanced GluN2B NMDA expression supporting the potential ability of radiprodil to control seizures in these conditions. About GRIN Therapeutics GRIN Therapeutics is dedicated to the research and development of precision therapeutics for pediatric neurodevelopmental disorders with the goal of bringing hope to patients and caregivers. Late last year, GRIN Therapeutics reported promising topline data from a Phase 1b/2a clinical trial (the Honeycomb Trial) evaluating radiprodil in GRIN-related neurodevelopmental disorder in patients with GoF variants, leading to the decision to advance to a Phase 3 trial. The company has an additional ongoing clinical trial to evaluate radiprodil for the potential treatment of tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II. GRIN Therapeutics is an affiliate of Neurvati Neurosciences, a portfolio company of Blackstone Life Sciences (BXLS). For more information, please visit About Neurvati Neurosciences Neurvati Neurosciences, a portfolio company of Blackstone Life Sciences, identifies and advances the development of high-potential drug candidates across the neuroscience landscape. Neurvati employs a collaborative model that establishes fit-for-purpose affiliate companies, aligning dedicated resources with long-term strategic capital to catalyze innovative treatment options in areas of unmet need. Neurvati's team of experienced operators and drug developers seeks opportunities to challenge current treatment paradigms and make a difference for patients suffering from a wide range of neurological and psychiatric disorders. For more information, please visit About Blackstone Life Sciences Blackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines and medical technologies that improve patients' lives and currently has more than $12 billion in assets under management. Corporate Contact Elliott Ruiz +1 201.674.5417 [email protected] SOURCE GRIN Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The study also found high satisfaction with TDD for pain relief in patients with complex chronic pain EDINA, Minn., Feb. 26, 2025 /PRNewswire/ -- Nura Pain Clinics, a subsidiary of the Capitol Pain family of practices and the Twin Cities' foremost multidisciplinary pain management clinic, announces the results of a recent study on patient satisfaction using spinal targeted drug delivery (TDD) for pain relief, indicating it may be a safer, more effective alternative to long-term opioid use. The study was published in Neuromodulation, the preeminent journal in the area of neuromodulation, providing readers with state of the art clinical, translational and basic science research in the field. The independent study investigated which components of TDD therapy lead to the best outcomes and highest patient satisfaction. Nura researchers analyzed survey data from 560 active TDD patients, focusing on factors like medication dosage, pain relief, diagnosis, catheter tip location, side effects, mental clarity, physical functioning, and health care use. The data demonstrated: Good to excellent pain relief (63.8%) High satisfaction with TDD (80.7%) Improvement in physical functioning (75.0%) Better quality of life (89.7%) An impressive 78.5% of respondents reported complete discontinuation or substantial reductions in systemic opioid use "The results of this study confirm that intrathecal drug delivery can be a safe and effective alternative to long-term use of opioids for patients with refractory chronic pain who have exhausted other interventions," said Dr. David Schultz, lead author and chief medical officer of Nura Pain Clinics. TDD therapy, administered through a pain pump, is a safe, proven and effective way to manage chronic pain, cancer pain or severe spasticity with fewer side effects and a high degree of clinician control as compared to oral medication. "Our findings indicate that patients who received higher-dose intrathecal medication admixtures experienced better pain relief and higher satisfaction -- without any increase in side effects, mental impairment, or therapy complications," said Dr. Schultz. The team at Nura is at the forefront of innovation to provide comprehensive care through the seamless coordination of medication management, physical therapy and behavior therapy within a single medical practice. Nura's multidisciplinary approach is a critical advantage for patients who otherwise may receive fragmented pain management by multiple providers at multiple locations. For more complex chronic pain problems, Nura offers a full range of implantable pain control options, including spinal cord stimulation and targeted spinal drug delivery devices. This approach allows providers to focus on pinpointing and treating the source of pain while reducing the need for opioids. The full study is available in Neuromodulation. For more information on Nura Pain Clinics and its focus on pain management, visit nuraclinics.com. About Nura Pain Clinics Nura (formally MAPS Medical Pain Clinics) was founded in 1995 by David Schultz, MD, a pioneer in interventional pain management. In his work as a practicing anesthesiologist in the 1990s, Dr. Schultz realized the need to treat chronic pain patients with a more comprehensive approach and went on to establish the region's first multidisciplinary pain management clinic, coordinating high tech interventional procedures with in-house physical therapy and psychology services. Nura is nationally recognized as a center of excellence for implantable pain control and one of the leading research centers in the U.S. for trials and implants of neuromodulation devices. Nura's mission is to improve the lives of people living with the most complex chronic pain through exceptional care. About Capitol Pain Institute Capitol Pain Institute (CPI) is a leading interventional pain management platform with clinics and ambulatory surgical centers across the United States. CPI offers a customized and multi-disciplinary approach to chronic pain treatment, with a particular focus on advanced procedures such as spinal cord stimulator implants. CPI partners with physicians and practices that share a common goal of providing high-quality, compassionate care at a reasonable cost. The company is also committed to enhancing the communities in which they serve and investing in the advancement of the field of pain management through innovation and evidence-based medicine. CPI was founded by Dr. Matthew Schocket in Austin, Texas, in 2007. For more information, visit CapitolPain.com. MEDIA CONTACT Hyedi Nelson Bellmont Partners 651-757-7054 [email protected] SOURCE Nura Pain Clinics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

A French biotech focused on developing genetic medicines for neurological conditions has secured €27 million in a Series B raise to support its lead program through an early clinical trial. Paris-based EG 427 will use the proceeds to advance its candidate, dubbed EG110A, for the treatment of neurogenic detrusor overactivity (NDO) in patients with spinal cord injury. NDO develops when a muscle in the bladder contracts too much, leading to urinary incontinence. EG110A is currently in a Phase 1b/2a dose-ranging study which is aiming to enroll 16 patients in the US with spinal cord injury and NDO, according to a Thursday release . The company expects “significant readouts” later this year, CEO Philippe Chambon told Endpoints News in an interview. The main treatment for an overactive bladder due to loss of CNS control is botox, but this acts at the muscle level and doesn’t address the root driver of the problem, Chambon said. He added that the driver is the type C sensory neuron, which is involved in an “abnormal reflex loop” that triggers involuntary bladder contractions. The biotech’s candidate carries a transgene that stops certain vesicles from forming in the type C neurons, and so cutting the reflex loop, Chambon said. The treatment uses a non-replicating herpes viral vector developed with the company’s so-called HERpes Modular Expression System (HERMES) technology. While the company is initially focusing on spinal cord injury, many other patients experience NDO, including those with Parkinson’s and multiple sclerosis. The Phase 1b/2a study could thus pave the way for development of its candidate across a number of indications which together affect millions of patients, according to the company. The biotech will also use some of the Series B to advance its preclinical pipeline, where it is exploring programs that could target chronic pain, focal epilepsy and spasticity. The fundraise was co-led by Andera Partners and Bpifrance, with support from others including specialist fund SCI Ventures. Chambon said it marks the company’s first institutional round. In 2023, EG 427 secured €18 million in a Series A backed entirely by family office investors. The company was founded back in 2019 as a spinout of technology developed by a consortium of universities.