mGluR5

Allyx continues to advance clinical research of its first-in-class oral compound for neurodegenerative diseasesNEW HAVEN, Conn., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics has announced that the first patient has been dosed with its lead compound, ALX-001, in a new clinical study assessing safety, pharmacokinetics and potential therapeutic response in patients with Parkinson’s disease. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy. Allyx Therapeutics is a clinical-stage biotechnology company working to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. “Allyx Therapeutics is moving forward with strong momentum to develop ALX-001 as the first-ever disease-modifying small molecule for neurodegenerative diseases, with two concurrent safety studies in patients with Parkinson’s disease and with Alzheimer’s disease, adding important information to the body of knowledge for this novel therapeutic approach,” said Tim Siegert, Ph.D. chief operating officer and co-founder of Allyx Therapeutics. The 28-day Parkinson’s disease study (NCT06309147) is assessing the safety of ALX-001 dosed twice daily at either 50 mg or 100 mg versus placebo in adults between 21 and 80 years of age, and will also investigate dopamine transporter levels in the brain measured with single photon emission computed tomography as an early marker of therapeutic response to a treatment that targets synapse restoration. The study is being conducted by the Duke Clinical Research Institute (DCRI) and is supported with grant funding awarded to Allyx Therapeutics from The Michael J. Fox Foundation for Parkinson’s Research. “I’m excited to collaborate with the Allyx team as we advance understanding of how Parkinson’s disease affects the brain and what more we might be able to do to provide a real impact for patients. Investigating the safety of this disease-modifying small molecule is a crucial first step, and one I’m proud to be a part of,” said Laurie H. Sanders, Ph.D., associate professor of Neurology at Duke University School of Medicine and DCRI. Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in ongoing studies. The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) programs, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research, among others. About ALX-001 ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer's and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx scientific founder Stephen Strittmatter at Yale School of Medicine. Allyx Therapeutics obtained an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale School of Medicine. About Allyx Therapeutics Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com. About the Duke Clinical Research Institute The DCRI, part of the Duke University School of Medicine, is the largest academic clinical research organization in the world. Our mission is to develop, share, and implement knowledge that improves global health through innovative clinical research. The institute conducts multinational clinical trials, manages major national patient registries, and performs landmark outcomes research. The DCRI is a pioneer in cardiovascular and pediatric clinical research and conducts groundbreaking clinical research across multiple other therapeutic areas, including infectious disease, neuroscience, respiratory medicine, and nephrology. ContactMedia: Eliza Schleifstein917.763.8106Eliza@schleifsteinpr.com Investors: Tim Siegert203-691-6543tsiegert@allyxthera.com

-- Top-line results from Phase 2B study expected in first quarter of 2025 -- Aug. 13, 2024 -- Noema Pharma AG, a clinical-stage biotech company targeting debilitating neuroscience-based disorders with first-in-disease therapeutic approaches, today announced the completion of enrollment in the GALENE Phase 2B trial, a double-blind, placebo-controlled trial evaluating the efficacy of NOE-101 for seizure control in tuberous sclerosis complex (TSC). "We are pleased to have completed enrollment in our Phase 2B study of NOE-101,” said Ilise Lombardo, MD, Chief Executive Officer for Noema. “NOE-101 has the ability to decrease neuronal excitability with the potential to address important therapeutic needs for patients with TSC with a novel mechanism of action. With the trial fully enrolled, we look forward to reporting top-line data in the first quarter of next year.” Dr. Mustafa Sahin, MD, Associate Chief of Research, Department of Neurology; Managing Director, Rosamund Stone Zander Translational Neuroscience Center and Director, Translational Research Program at Boston Children's Hospital added, “TSC is a debilitating disease characterized by serious, life-threatening seizures that have long-term impact on brain development. Since current antiepileptic therapies have limited efficacy and significant side effects with over 60% of patients refractory to current treatments, the need for new treatments remains strong.” Dr. Sahin added, “This need underscores the importance of studying medications like NOE-101 with distinct mechanisms of action.” The Phase 2B GALENE study (NOE-TSC-201) is a multicenter, 30-week randomized, double-blind, placebo controlled, cross-over study evaluating the efficacy and safety of NOE-101 adjunctive to ongoing antiseizure therapy in patients with uncontrolled seizures associated with TSC. The primary endpoint will assess improvement in seizure frequency for the NOE-101 treatment and placebo treatment periods. Patients who complete the GALENE study may continue to a 52-week open-label extension. NOE-101, or basimglurant, is a potent inhibitor of metabotropic glutamate receptor 5 (mGluR5). It is also currently under clinical investigation for trigeminal neuralgia. Tuberous sclerosis complex is a rare genetic multisystem disorder caused by mutations in TSC1 and TSC2 genes. This disorder causes uncontrolled benign tumor growth in vital organs of the body including the brain, skin, heart, eyes, kidneys, and lungs and is the leading cause of genetic epilepsy. Approximately 80,000 people in the US and EU are diagnosed with TSC, and about 60 percent of patients do not respond to current treatment options. Their treatment-resistant seizures are often serious or life threatening. Noema Pharma is a clinical-stage biotech company advancing a portfolio of transformative therapeutics utilizing first-in-disease approaches targeting orphan and large neuroscience-based indications with high unmet need. Noema has active Phase 2 clinical trials evaluating pain in trigeminal neuralgia, seizures in tuberous sclerosis complex, Tourette syndrome and vasomotor symptoms due to menopause. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com. The content above comes from the network. if any infringement, please contact us to modify.

With Growing Body of Clinical Evidence, ALX-001 is Ready to Commence Phase 2 StudiesNEW HAVEN, Conn., June 11, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics, a clinical-stage biotechnology company working to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases, announced that the first Alzheimer’s disease patient has been dosed with its lead compound, ALX-001, in a clinical study. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy that is ready for Phase 2 clinical development. “Administering the first dose in patients is a watershed moment for a new therapeutic, and we are pleased to begin research in Alzheimer’s disease patients as we continue to advance clinical development of ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases,” commented Stephen Bloch, M.D., chief executive officer and co-founder of Allyx Therapeutics. Individuals with Alzheimer’s disease are being enrolled in a 28-day study which will evaluate the safety of ALX-001 dosed twice daily at either 50 mg or 100 mg versus placebo in adults between 50 and 80 years of age. This is the second phase of an ongoing study which previously assessed multiple ascending doses in healthy volunteers (NCT05804383) and is being conducted at Yale School of Medicine with support from the National Institute on Aging. “Every neuroscientist hopes for the moment that an asset they believe in and fight for advances to the critical stage of testing in patients,” stated Stephen Strittmatter M.D., Ph.D., scientific founder of Allyx Therapeutics and professor and chair of Neuroscience at the Yale School of Medicine. “The unique mechanism of action of ALX-001 and its promising early data have brought us to this point, and bolster our hope that it has the potential to be a difference-maker in Alzheimer’s disease.” ALX-001 is ready to proceed to phase 2 clinical development, and is not restricted by completion of ongoing studies. Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in studies supported by highly reputable third parties. The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) programs, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research, among others. About ALX-001ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer's and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx scientific founder Stephen Strittmatter at Yale School of Medicine. Allyx Therapeutics obtained an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale School of Medicine. About Allyx Therapeutics Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com. Contact Media: Eliza Schleifstein917.763.8106Eliza@schleifsteinpr.com Investors: Tim Siegert203-691-6543tsiegert@allyxthera.com