Iama Therapeutics Srl

GENOA, Italy--(BUSINESS WIRE)-- IAMA Therapeutics, a pharmaceutical company focused on the discovery, development, and commercialization of novel medicines for children affected by brain disorders, today announced that the first subject was treated with IAMA-6, an orally administered small molecule therapeutic targeting NKCC1. IAMA-6, IAMA’s lead program, is being evaluated for the treatment of autism and epilepsy in a Phase 1 first-in-human (FIH), randomized, double-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study. “We are proud to announce the commencement of the first-in-human trial aimed at establishing a safe dose range for the continued clinical development of our lead program, IAMA-6,” stated Andrea P. Malizia, CEO of IAMA Therapeutics. “This treatment, proven to be safe and well-tolerated in preclinical studies, holds the promise of making a genuine and meaningful impact on children and families grappling with central nervous system disorders, including autism and epilepsies. This significant milestone brings us one step closer to fulfilling our commitment to address the unmet medical needs in this patient population." Initial interim data from this study is expected in June 2024. About the IAMA-6 Phase 1 Clinical Trial The phase 1 clinical trial is a randomized, double blind, placebo controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study. The study will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and food effect of IAMA-6 administered orally to healthy adults. About IAMA-6 IAMA-6, the lead clinical program of IAMA, is an orally administered small molecule therapeutic designed to directly target and inhibit NKCC1-associated neuronal hyperexcitability. The elevated activity of NKCC1 is implicated in various pathological conditions, underscoring the significant potential of NKCC1 inhibition in the treatment of both idiopathic and secondary forms of autism (autism spectrum disorder, or ASD), refractory epilepsy, Dravet Syndrome, and other neurological disorders. With the capability to be applied across multiple central nervous system (CNS) indications, IAMA-6 has demonstrated its safety and favorable tolerability in pre-clinical studies. About IAMA Therapeutics IAMA Therapeutics pushes the boundaries of neuroscience drug discovery to develop new therapeutic opportunities and make a difference in children affected by epilepsy, neurodevelopmental and cognitive disorders. IAMA Therapeutics is developing IAMA-6 for potentially treating drug-resistant epilepsies and autism spectrum disorders, in addition to other chloride modulators targeting neuropsychiatric conditions characterized by cognitive impairment. For more information, please visit

GENOA, Italy--(BUSINESS WIRE)-- IAMA Therapeutics, a pharmaceutical company focused on the discovery, development, and commercialization of novel medicines for children affected by brain disorders, today announced that the Italian Medicines Agency (AIFA) has accepted the Clinical Trial Application (CTA) to initiate a Phase 1 first-in-human (FIH), randomized, double-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and food effect of IAMA’s lead program IAMA-6, an orally administered small molecule therapeutic targeting NKCC1 for the treatment of autism and epilepsy. “We are proud to announce the first regulatory clearance for our lead program, IAMA-6, which has been shown to be safe and well-tolerated in preclinical studies,” said Andrea P. Malizia, Chief Executive Officer of IAMA Therapeutics. “Children living with central nervous system disorders, including autism and epilepsies, face significant unmet medical needs. This exciting milestone for IAMA-6 brings us one step closer to achieving our goal of making a meaningful impact on the individuals affected by these diseases and the families who care for them.” IAMA-6 has shown encouraging in vitro and in vivo proof of concept results in autism spectrum disorders and drug-resistant epilepsies. The compound is designed to directly target and inhibit NKCC1 and neuronal hyperexcitability. The Company expects to begin dosing healthy adults in the Phase 1 study in January 2024. About IAMA Therapeutics IAMA Therapeutics pushes the boundaries of neuroscience drug discovery to develop new therapeutic opportunities and make a difference in children affected by epilepsy, neurodevelopmental and cognitive disorders. IAMA Therapeutics is developing IAMA-6 for potentially treating drug-resistant epilepsies and autism spectrum disorders, in addition to other chloride modulators targeting neuropsychiatric conditions characterized by cognitive impairment. For more information, please visit .

Study results support the potential for IAMA-6 as a disease-modifying therapy for drug-resistant epilepsies GENOA, Italy--(BUSINESS WIRE)-- IAMA Therapeutics, a pharmaceutical company focused on the discovery, development, and commercialization of novel medicines for children affected by brain disorders, today announced that it will present a poster titled “Selective NKCC1 Inhibitors for the Treatment of Refractory Epilepsy” at the American Epilepsy Society (AES) 2023 Annual Meeting, taking place December 1-5 in Orlando, FL. “The altered expression of chloride importer NKCC1, which can lead to an imbalance in brain excitation and inhibition, is implicated in several neurodevelopmental and neurological conditions, including drug-resistant epilepsies like Mesial Temporal Lobe Epilepsy (MTLE) and Dravet syndrome,” said Annalisa Savardi, Team Leader Neuropharmacology at IAMA Therapeutics. “In the upcoming presentation, preclinical data show that IAMA’s lead program IAMA-6, a small molecule targeting NKCC1 for the treatment of autism and epilepsy, effectively reduces epileptic activity in an animal model of MTLE and, in preliminary data, reduces the number of seizures in a model of Dravet syndrome. Importantly, IAMA-6 restores correct inhibition directly in brain preparations from patients with MTLE.” “These exciting preclinical results demonstrate the potential of IAMA-6 for treating drug-resistant epilepsies and signify a step forward for our pipeline of small molecule therapies for CNS disorders,” said Andrea P. Malizia, Chief Executive Officer of IAMA Therapeutics. “We look forward to sharing more with the healthcare providers, scientists, advocates, and other industry professionals dedicated to better outcomes for people with epilepsy at AES 2023.” AES 2023 Annual Meeting Format: Poster Presentation Poster Title: Selective NKCC1 Inhibitors for the Treatment of Refractory Epilepsy Poster Session #: 1.275 Date: Saturday, December 2, 2023 Time: 12:00 PM - 2:00 PM About IAMA Therapeutics IAMA Therapeutics pushes the boundaries of neuroscience drug discovery to develop new therapeutic opportunities and make a difference in children affected by epilepsy, neurodevelopmental and cognitive disorders. IAMA Therapeutics is developing IAMA-6 for potentially treating drug-resistant epilepsies and autism spectrum disorders, in addition to other chloride modulators targeting neuropsychiatric conditions characterized by cognitive impairment. For more information, please visit