atai Life Sciences has been awarded a multi-year, milestone-driven grant worth up to $11.4 million by the National Institute on Drug Abuse (NIDA) to fund the optimization and early-stage development of its novel 5-HT2A/2C receptor agonists for opioid use disorder (OUD). The UG3/UH3 grant structure represents a competitive, milestone-based funding mechanism reserved for promising translational research.
Addressing Critical Unmet Medical Need
The program targets a significant global health challenge, as OUD affects approximately 16 million people worldwide with annual costs exceeding $750 billion. More than 120,000 opioid-related deaths occur annually worldwide. Current OUD treatments often require daily administration, creating a significant treatment burden for patients.
"This grant award underscores atai's and NIDA's shared dedication to providing meaningful treatment options for those struggling with OUD," stated Glenn Short, Ph.D., Chief Scientific Officer of atai. "This is the first external validation of atai's AI-driven polypharmacology drug discovery approach and its 5-HT2A/2C agonist program."
Novel Therapeutic Approach
atai's approach aims to develop compounds that modulate serotonin receptors implicated in addiction while specifically avoiding 5-HT2B activity, which has been linked to cardiac valvulopathy – an important safety consideration. The company's discovery program has identified a new class of differentiated 5-HT2A/2C receptor agonists that demonstrate non-hallucinogenic potential, as evidenced by their inability to fully substitute for traditional psychedelics in pre-clinical studies.
The goal is to identify clinical candidates that maintain therapeutic activity against opioid use disorder while minimizing hallucinogenic effects. These novel compounds could support lasting abstinence while reducing the daily treatment burden seen with current OUD therapies.
Development Timeline and Funding Structure
The grant provides non-dilutive funding to advance atai's 5-HT2A/2C agonist program, supporting lead optimization, translational proof-of-concept studies, and the toxicology and manufacturing work needed to file an Investigational New Drug (IND) application. This funding effectively de-risks early development without equity dilution.
If early-stage development milestones are met, atai plans to progress the program into a first-in-human Phase 1 study. The federal backing represents meaningful validation of atai's scientific approach and accelerates their timeline toward clinical trials.
Strategic Significance
"The commitment of Federal support to advance innovative and differentiated research in OUD highlights the devastating toll this crisis continues to take on individuals, families, and communities," said Srinivas Rao, M.D., Ph.D., Chief Executive Officer and Co-founder of atai. "This investment demonstrates the urgent need for evidence-based prevention, treatment, and long-term recovery solutions that can truly make a difference."
The grant not only provides capital but positions atai as an emerging leader in the non-hallucinogenic 5-HT2A agonist space, an area gaining significant attention for neuropsychiatric applications. This recognition helps establish atai as a leader in the non-hallucinogenic 5-HT2A agonist drug space and accelerates the timetable to bring these innovative treatments to patients suffering from addiction.
