IAMA Therapeutics

IAMA Therapeutics is a clinical-stage pharmaceutical company, established in 2021 and headquartered in Genoa, Italy. The company emerged from a decade of research conducted at the Italian Institute of Technology (IIT) by the 'Brain Development and Disease' and 'Molecular Modeling and Drug Discovery' research groups, led by founders Laura Cancedda and Marco De Vivo. This foundational research, supported by Fondazione Telethon, focused on neurodevelopmental disorders and led to the discovery of a new class of drug candidates.

The company's core business revolves around discovering and developing small-molecule drugs that target cation-chloride cotransporters to address central nervous system (CNS) disorders. Its business model is centered on advancing these proprietary drug candidates through clinical trials to eventually commercialize them, providing new treatments for patients with neurological conditions. The company has successfully raised a total of $25.9M in capital over two funding rounds. A Series A round in April 2022 brought in €8 million from Claris Ventures and CDP Venture Capital. This was followed by a €15 million Series B round in April 2025, led by Indaco Venture Partners, to accelerate clinical development.

IAMA's lead product candidate is IAMA-6, an orally administered, selective inhibitor of the NKCC1 cotransporter. The NKCC1 protein is involved in transporting chloride ions into neurons; in many brain disorders, its dysregulated activity leads to an ion imbalance and neuronal hyperexcitability. By selectively inhibiting NKCC1, IAMA-6 aims to restore the natural chloride balance and reduce the pathological neuronal activity seen in conditions like epilepsy and autism. This mechanism has shown the potential to alleviate cognitive and behavioral symptoms in preclinical models. IAMA-6 entered a first-in-human Phase 1 clinical trial in January 2024 to evaluate its safety, tolerability, and pharmacokinetics. The company is targeting a range of orphan brain disorders, including drug-resistant epilepsy, Dravet syndrome, and autism spectrum disorders.

Keywords: IAMA Therapeutics, clinical-stage pharmaceutical, neurodevelopmental disorders, central nervous system, NKCC1 inhibitor, IAMA-6, epilepsy treatment, autism spectrum disorder, cation-chloride cotransporters, small-molecule drugs, orphan brain disorders, Laura Cancedda, Marco De Vivo, Italian Institute of Technology, neuronal hyperexcitability, chloride transporter modulation, drug discovery, Genoa Italy, refractory epilepsy, pediatric neuropsychiatric conditions, CNS drug discovery, clinical trials, selective inhibitor