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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

July 24, 2024 -- ConSynance Therapeutics, Inc., a clinical-stage biopharmaceutical company developing first-in-class therapies for rare central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational drug CSTI-500 for the treatment of Prader-Willi Syndrome (PWS) in children and adolescents. “Receiving RPDD from the FDA is a significant regulatory milestone for ConSynance as we prepare for the Phase 2 study of CSTI-500 for PWS,” said Shuang Liu, PhD, Founder and CEO of ConSynance. “PWS is a rare neurodevelopmental disorder characterized by hyperphagia and severe temper outbursts, among other neuropsychiatric behaviors. CSTI-500 targets the imbalance of three key neurotransmitters, serotonin, dopamine and norepinephrine associated with these behaviors. CSTI-500 stands out as the first drug candidate for PWS designed to address both hyperphagia and severe temper outbursts, providing a potential breakthrough and comprehensive treatment solution for this challenging disorder.” The FDA defines rare pediatric diseases as conditions affecting fewer than 200,000 individuals in the United States, which are serious or life-threatening and primarily impact those under 18 years of age. This designation makes ConSynance eligible to receive a transferable Priority Review Voucher if CSTI-500 receives FDA approval for PWS. Priority Review Vouchers have recently sold for approximately $100 million, highlighting their significant value. More About CSTI-500: CSTI-500 is a phase 2-ready, first-in-class, orally administered Triple Monoamine Reuptake Inhibitor (TRI). It is designed to optimally increase the levels of three critical neurotransmitters, serotonin, dopamine, and norepinephrine in the synaptic clefts of neurons. Human and animal data suggest that PWS is associated with low levels of these neurotransmitters. Deficiencies in these neurotransmitters are also implicated in many other neuropsychiatric disorders, as evidenced by the approval of selective serotonin reuptake inhibitors, selective norepinephrine reuptake inhibitors, and dopamine norepinephrine reuptake inhibitors in conditions like binge eating disorder (BED), obsessive compulsive disorders (OCD), attention deficit hyperactivity disorder (ADHD), among others. CSTI-500 has demonstrated brain target engagement in healthy volunteers via positron emission tomography (PET). These data, along with the corresponding pharmacokinetics data, predict the efficacy of CSTI-500 in PWS and other neuropsychiatric indications, and enable a personalized dosing approach to ensure optimal safety and efficacy. CSTI-500 has been studied in nearly 100 humans across three Phase 1 clinical trials, including 10 PWS patients, setting the stage for a Phase 2 study expected to commence in 2025. About PWS: PWS is a rare, complex genetic neurodevelopmental disorder that affects approximately 1 in 15,000 live births. Initially characterized by severe hypotonia and failure to thrive, it evolves into a life-threatening hyperphagia beginning in early childhood, compounded by other debilitating neuropsychiatric behaviors, notably severe temper outbursts. Despite significant challenges faced by PWS patients and their families, there are currently no approved therapies that address the core dysfunctional brain networks associated with abnormal levels of monoamines and consequent neuropsychiatric behaviors in PWS. Normalizing these neurotransmitters may restore functional neural circuits, alleviating hyperphagia, temper outbursts, among other behavioral symptoms that significantly impact the patients and their families. About ConSynance Therapeutics: ConSynance is a New York-based biopharmaceutical company dedicated to developing innovative therapies for rare CNS disorders. ConSynance is a member of the PWS Clinical Trial Consortium and engaged in an ongoing partnership with Harmony Biosciences to develop HBS-102 (formerly known as CSTI-100) for the treatment of a variety of rare neurological diseases. These partnerships underscore the company's collaborative approach and its drive to bring effective therapies to patients in need. The content above comes from the network. if any infringement, please contact us to modify.

SEOUL, South Korea I July 09, 2024 I S.BIOMEDICS (KOSDAQ: 304360) announced positive one-year assessment data from the first 3 participants (first low dose cohort) of Phase I/IIa clinical trial for Parkinson’s disease with TED-A9, hESC (human embryonic stem cell)-derived midbrain dopaminergic progenitors. The data demonstrates that 3 participants showed safety and efficacy of TED-A9 in a one-year follow-up. The clinical trial was conducted at Severance Hospital of Yonsei University in South Korea led by Prof. Jin-Woo Chang, a neurosurgeon and Prof. Phil Hyu Lee, a neurologist. For the 3 participants who received initial low dose (3.15 million cells), MRI and CT scans after one year revealed no adverse effects related to the cell transplantation or surgery. Moreover, the MDS-UPDRS Part III (off) evaluation, which objectively measures motor functions, showed a mean decrease of 12.7 points, from baseline 61.7 to 49.0 one-year post treatment. The MDS-UPDRS Part III (off) evaluation indicated significant improvement in their motor abilities. Improvement was also observed in symptoms such as wearing off and freezing of gait. DAT brain imaging (FP-CIT-PET), conducted one-year post transplantation, revealed an increase in dopamine transporters (DAT), suggesting the potential engraftment of dopamine neurons, which correlated with improvements in the patients’ Parkinson’s symptoms. “Although this clinical evaluation only targets the first 3 low-dose patients, but not all 12 subjects, no adverse issues related to transplant surgery or cell safety were observed in one-year post transplantation. Importantly, the clinical results demonstrated very promising efficacy,” said Prof. Dong-Wook Kim at Yonsei University College of Medicine and CTO of S.BIOMEDICS. “The results are believed to align closely with the findings from our preclinical in vitro and in vivo studies. We are excited that TED-A9 could be a fundamental treatment that directly replaces dopaminergic neurons lost in patients with Parkinson’s disease.” Assessment data of further participants (first high dose cohort) will be announced in September or October of 2024 after a one-year follow-up. About TED-A9 and Phase I/IIa clinical trial TED-A9 is an investigational cell therapy designed to replace ventral midbrain-specific dopaminergic cells lost in patients with Parkinson’s disease. These ventral midbrain-specific dopaminergic cells are derived from hESCs by exclusively utilizing small molecules only. TED-A9 represents a significant advancement in the field, offering highly purified dopamine cells derived from hESCs. Through surgical procedure, these hESC-derived dopaminergic progenitor (precursor) cells (TED-A9) are transplanted to three segments of the putamen; the anterior, middle, and posterior sections, with three tracks per each putamen. Bilateral putamina received cell transplantation in a single surgical procedure, with cells injected at three points within each track. After transplantation, the expectation is that the transplanted dopaminergic progenitor cells will mature into dopaminergic neurons which will supply the dopamine that Parkinson’s patients are lacking, restoring the motor function of patients. The Phase I/IIa clinical trial is conducted on 12 participants who have been diagnosed with Parkinson’s disease for more than 5 years and exhibited motor complications such as wearing-off, freezing of gait or dyskinesia. The participant’s age was between 50 and 75 years old. TED-A9 were administered to 6 participants in the low-dose group (3.15 million cells) and to another 6 participants in the high-dose group (6.30 million cells). An initial cohort of three patients was enrolled at a low dose to assess initial safety, including dose-limiting toxicity (DLT) evaluation, over a period of up to 3 months post-transplantation. There were no safety concerns within this timeframe. Thus, an additional 3 patients were enrolled at a high dose for evaluation over another 3-month period post-transplantation. As no safety issues arose during this extended period, the clinical trial continued by adding three further patients to each of the low-dose and high-dose groups, totaling 12 patients. The final participant was administered with TED-A9 on February 2024. The primary objective of the Phase I/IIa trial is to assess the safety and exploratory efficacy of TED-A9 transplantation over two years post-transplant. Safety will be monitored for additional 3 years, making it a total 5 years. About S.BIOMEDICS Established in 2005, S.BIOMEDICS is at the forefront of stem cell therapy, focusing on regenerative medicine powered by data-driven biology. Based on two core platform technologies, S.BIOMEDICS currently develops seven cell therapy programs, targeting non-curable diseases. Its leading programs are under clinical stage accelerating the journey of cell medicine as shown below: For more information about S.BIOMEDICS, visit www.sbiomedics.com . S.BIOMEDICS is listed on the Korea Exchange (KOSDAQ: 304360) and is also the founder and controller of S.THEPHARM ( www.sthepharm.com ), a corporation specializing in anti-aging products such as HA-Filler. More Information about the Phase 1/2a clinical trial for Parkinson’s disease is available at ClinicalTrials.gov (NCT05887466) . SOURCE: S.Biomedics