ConSynance Therapeutics Announces U.S. FDA Grants Rare Pediatric Disease Designation to CSTI-500, a Potential First-in-Class Therapy for Prader-Willi Syndrome

RENSSELAER, N.Y., July 24, 2024 (GLOBE NEWSWIRE) — 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. For more information, please visit www.ConSynance.com Forward-Looking Statements: This press release contains forward-looking statements based on ConSynance Therapeutics Inc.’s current expectations and beliefs. These statements are subject to various risks and uncertainties, and actual results may differ. ConSynance Therapeutics Inc. accepts no obligation to update or revise any forward-looking statement, except as required by law. Contact Information:Sarah Sheldrick, PhDConSynance Therapeutics, [email protected]