NKCC1

STALICLA’s precision psychiatry study highlights strong EEG-based target engagement of STP1 treatment in defined subgroup of patients with autism and numerical improvement in core symptoms June 27, 2024 – STALICLA SA, a Swiss neuro precision biotech company dedicated to developing precision medicine-based treatments for neuropsychiatric and neurodevelopmental disorders, today announced the publication of a landmark phase 1b study with STP1, a novel combination therapy tailored for the treatment of a clinically and biologically defined subgroup of patients with autism spectrum disorder (ASD), named ASD Phenotype 1 (ASD-Phen1). The results of the study were published in the peer-reviewed journal Biomedicines, in an article titled “Safety, Tolerability, and EEG-Based Target Engagement of STP1 (PDE3,4 Inhibitor and NKCC1 Antagonist) in a Randomized Clinical Trial in a Subgroup of Patients with ASD”. Lynn Durham, CEO of STALICLA, highlighted: “This study marks a significant milestone in the advancement of precision medicine for ASD. It is a first-of-its-kind stratification-based approach for clinical development in neurodevelopmental disorders, demonstrating the potential of precision medicine in ASD.” The randomized, double-blind, placebo-controlled phase 1b clinical trial evaluated the safety and tolerability of STP1, a combination of ibudilast and bumetanide, in ASD-Phen1 patients. The clinical trial (registered at clinicaltrials.gov NCT04644003), involved two 14-day treatment phases of ASD-Phen1 patients receiving STP1 or placebo. The results showed that STP1 was well-tolerated with no significant adverse effects reported. Significant and dose-related reductions in gamma power were observed in the whole brains of patients taking STP1, particularly in regions associated with executive function and memory. Additionally, STP1 increased alpha 2 power in frontal and occipital regions, while improving habituation and neural synchronization to auditory chirps. Dr. Craig A. Erickson, Associate Professor, UC Department of Psychiatry and Behavioral Neuroscience, Cincinnati Children’s Hospital Medical Center, and the principal investigator of the study, remarked: “The electrophysiological signals from this study are remarkable and represent the strongest early trial target engagement signals our lab has seen in the autism field.” Dr. Laura Pérez-Cano, Head of Discovery at STALICLA and co-author of the study, added: “These findings not only highlight the potential of STP1 as a therapeutic option for ASD-Phen1 patients but also underscore the importance of combining biologically-based patient stratification with quantifiable outcome measures such as EEG that can then be correlated with behavioral measures.” By focusing on a biologically defined subgroup of ASD patients, STALICLA is moving closer to making personalized treatment options a reality for patients with ASD. This approach has the potential to revolutionize the treatment of ASD and other neuropsychiatric conditions. STALICLA is a global, clinical-stage biotechnology company focused on advancing precision medicine for brain disorders. The company has developed a unique neuro precision development platform, DEPI, supported by clinical validation in a first indication: Autism Spectrum Disorder. Its lead neurodevelopmental disorder asset, STP1, is entering Phase 2 trials. Its lead neuropsychiatry asset, STP7 (Mavoglurant), fully funded by the US government, will soon be Phase 3 ready for Cocaine Use Disorder. ‎ The content above comes from the network. if any infringement, please contact us to modify.

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 .