What Iama Therapeutics does

IAMA Therapeutics S.r.l. is a clinical-stage biopharmaceutical company headquartered in Genoa, Italy, that develops first-in-class small molecule therapeutics selectively inhibiting the cation-chloride cotransporter NKCC1 to treat central nervous system disorders. The company's lead asset, IAMA-6, is an oral selective NKCC1 inhibitor that has completed a Phase 1 first-in-human trial (NCT06300398) demonstrating safety and favorable pharmacokinetics, and is positioned to enter Phase 2 in drug-resistant epilepsy, autism spectrum disorder, Fragile X syndrome, and Dravet syndrome. A second pipeline compound, IAMA-299, is in earlier-stage development. The underlying technology is a proprietary discovery platform combining medicinal chemistry, atomistic molecular simulation, and structure-based drug design, developed in partnership with the Istituto Italiano di Tecnologia (IIT) under an exclusive worldwide license granted in January 2022.

The company's business model is a pre-commercial licensing-and-co-development model targeting large pharmaceutical partners. As a clinical-stage biotech with no marketed products, IAMA does not yet generate product revenue; instead, its go-to-market involves strategic licensing of IAMA-6 and pipeline assets, with future revenue expected from upfront payments, milestone payments tied to clinical and regulatory progress, and royalties on commercial sales. Clinical operations are executed through service agreements with Evotec (Phase 1 execution and CMC) and translational research collaborations with Sapienza University, Brigham and Women's Hospital, Northwestern University, Psychogenics, and Telethon Foundation. The investor base includes Indaco Venture Partners, CDP Venture Capital, Claris Ventures, and Fondazione Enea Tech e Biomedical, with a €15 million financing round closed in April 2025 specifically earmarked for Phase 2 advancement.

Name

Iama Therapeutics

Legal name

IAMA Therapeutics S.r.l.

Website

https://iamatherapeutics.com

Company type

Private

Founded year

2022

Operating status

Operating

Headcount range

1–10 employees

Short description

IAMA Therapeutics is a clinical-stage biopharmaceutical company developing first-in-class oral selective NKCC1 inhibitors for CNS disorders including drug-resistant epilepsy, autism spectrum disorder, and Fragile X syndrome, with lead asset IAMA-6 advancing to Phase 2 trials.

Ownership category

akta.pro rank

Headquarters

HQ city

Genoa

HQ country

Italy

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Pharmaceutical Development and Licensing: As a clinical-stage biopharmaceutical company, IAMA Therapeutics' primary revenue model will involve licensing its drug candidates to larger pharmaceutical companies, receiving milestone payments tied to clinical development progress, regulatory approvals, and commercial sales royalties.
2. Co-development and Partnership Agreements: Strategic partnerships with pharmaceutical companies for joint development and commercialization, potentially generating upfront payments, research funding, and success-based milestone revenues.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels4 records

Core offering

IAMA Therapeutics is a clinical-stage biopharmaceutical company that discovers and develops novel oral small-molecule therapeutics selectively inhibiting the NKCC1 cation-chloride cotransporter to restore neuronal chloride homeostasis in central nervous system (CNS) disorders. Its lead candidate, IAMA-6, targets autism spectrum disorder, Fragile X syndrome, drug-resistant epilepsy, and Dravet syndrome, and has completed Phase 1 first-in-human clinical trials. A second pipeline candidate, IAMA-299, is in earlier development for related CNS indications.

Product overview

IAMA Therapeutics is a clinical-stage biopharmaceutical company advancing a pipeline of novel small molecule therapeutics targeting selective cation-chloride cotransporters, particularly NKCC1, for central nervous system disorders. The company's lead program is IAMA-6, a first-in-class oral selective NKCC1 inhibitor currently completing Phase 1 clinical trials for autism and epilepsy, with plans advancing toward Phase 2. A second pipeline compound, IAMA-299, is in earlier preclinical development. The company leverages a proprietary discovery platform combining medicinal chemistry, structure-based drug design, and computational modeling in partnership with the Istituto Italiano di Tecnologia (IIT) to develop treatments for conditions characterized by chloride imbalance including epilepsies, autism spectrum disorders, and other neurodevelopmental disorders.

Differentiator

Problem solved

Functional benefit

Products and services

• IAMA-6 First-in-class oral selective small-molecule NKCC1 inhibitor designed to restore inhibitory neurotransmission and rebalance neuronal excitability in neurodevelopmental and CNS disorders. Lead clinical candidate for autism spectrum disorder, Fragile X syndrome, drug-resistant epilepsy, and Dravet syndrome. Phase 1 first-in-human clinical trial completed with favorable safety and pharmacokinetic profile.
• IAMA-299 Second pipeline compound in development targeting cation-chloride cotransporters for CNS disorders. Specific indications and development stage are not publicly detailed in available sources.

Quantifiable outcome

• Successfully completed Phase 1 first-in-human clinical trial demonstrating safety, tolerability, and favorable pharmacokinetic profile
• +2 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Seven partnerships are on record, tiered core and secondary.

• EvoteccoreTechnology or Integration · 2 October 2023Multi-phase strategic provider agreement where Evotec provides integrated preclinical and clinical development services including INDiGO platform, toxicity, safety, manufacturing, and clinical trial execution support. Evotec conducted the Phase 1 SAD/MAD first-in-human clinical trial of IAMA-6 in healthy adult volunteers under the agreement.
• Sapienza University of RomesecondaryStrategic or Co-development Partner · 10 July 2023Sponsored research agreement with Professor Eleonora Palma and Dr. Gabriele Ruffolo for translational studies investigating functional efficacy of selective chloride modulators in reducing neuronal excitability in human tissue from patients with epilepsies and neurodevelopmental disorders including Dravet syndrome, Focal Cortical Dysplasia, and Rett syndrome.
• Istituto Italiano di Tecnologia (IIT)coreStrategic or Co-development Partner · 1 January 2022Foundational research partnership and license agreement with IIT, the company's spin-out origin. Laura Cancedda and Marco De Vivo serve as Principal Investigators at IIT. The agreement grants exclusive worldwide rights to research, develop, manufacture, and commercialize selective NKCC1 inhibitors discovered at IIT. This collaboration provides access to high-performance computing and molecular modeling capabilities including access to one of Europe's most advanced supercomputers.
• PsychogenicssecondaryStrategic or Co-development PartnerPreclinical study collaboration for drug-resistant epilepsy and Dravet syndrome. Service agreement initiated in 2022 for preclinical development studies, with expanded agreement in January 2023 for preclinical studies in Dravet syndrome.
• Brigham and Women's HospitalsecondaryStrategic or Co-development PartnerResearch collaboration supporting the NKCC1 inhibitor program and clinical development of IAMA-6.
• Northwestern UniversitysecondaryStrategic or Co-development PartnerResearch collaboration supporting the NKCC1 inhibitor program and clinical development of IAMA-6.
• Telethon FoundationsecondaryStrategic or Co-development PartnerResearch funding and collaboration support. Dr. Annalisa Savardi was awarded postdoctoral fellowship from Jerome Lejeune Foundation as principal investigator in project on NKCC1 inhibition in Down syndrome and co-morbidities. Telethon Foundation supported preclinical studies on NKCC1 inhibitors.

Scale indicators5 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Marinus Pharmaceuticals: Clinical-stage CNS biopharma focused on seizure disorders (ganaxolone for CDKL5 deficiency disorder, drug-resistant epilepsies). Closely comparable to IAMA in indication focus, stage of development, and small-cap biopharma positioning, prior to its 2025 acquisition by Lundbeck.
• Ovid Therapeutics: Clinical-stage biopharma developing therapeutics for rare neurodevelopmental disorders including Fragile X syndrome and Angelman syndrome. Directly comparable to IAMA's neurodevelopmental indication focus and orphan CNS strategy.
• Praxis Precision Medicines: Clinical-stage CNS company developing therapies for epilepsy and other CNS disorders, with programs in developmental and epileptic encephalopathies. Comparable to IAMA in target indications and clinical-stage profile.
• Stoke Therapeutics: Clinical-stage biopharma using antisense oligonucleotides to treat severe neurodevelopmental diseases including Dravet syndrome. Comparable to IAMA in rare CNS indication focus, partnership-driven commercial model, and disease-modifying mechanism strategy.
• Zogenix (now UCB): Developed FINTEPLA for Dravet syndrome and Lennox-Gastaut syndrome before being acquired by UCB in 2022. Directly comparable to IAMA in Dravet syndrome indication overlap and pediatric CNS drug development.

Broad incumbents

• Sage Therapeutics: CNS-focused biopharma developing CNS therapeutics including Zurzuvae (zuranolone) for postpartum depression, with CNS pipeline breadth overlapping IAMA's CNS focus. Larger and broader CNS portfolio than IAMA's selective NKCC1 focus.
• Jazz Pharmaceuticals: Global biopharma with significant CNS franchise acquired via GW Pharmaceuticals (Epidiolex for epilepsies including Dravet). Broad incumbent competitor in IAMA's target indications with substantially greater commercialization resources.
• Lundbeck: Global CNS specialty pharmaceutical company with extensive epilepsy and CNS disorder portfolio. Notably the new IAMA CEO Torsten Madsen's former employer; represents both a potential partner/ acquirer and a broad incumbent in IAMA's CNS space.
• Cerevel Therapeutics: CNS-focused biopharma developing therapies for schizophrenia, epilepsy, and Parkinson's disease, acquired by AbbVie in 2024. Comparable CNS franchise approach; demonstrates exit pathway for CNS-focused biotechs that achieve clinical success.

Emerging players

• Hoba Therapeutics: Early-stage CNS biopharma developing novel pain and CNS therapeutics, previously led by the current IAMA CEO Torsten Madsen. Comparable in size, stage, and CNS therapeutic focus.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/iamatherapeutics
• Xx.com/IAMABiotech

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds2 records

Investors5 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Iama Therapeutics