atai Life Sciences Awarded Grant from the National Institutes of Health

Rhea-AI Summary

atai Life Sciences (NASDAQ: ATAI) has secured a significant $11.4 million UG3/UH3 grant from the National Institute on Drug Abuse (NIDA) to develop novel treatments for opioid use disorder (OUD). The grant will fund the development of non-hallucinogenic 5-HT2A/2C receptor agonists, marking the first external validation of atai's AI-driven polypharmacology drug discovery approach.

The funding will support lead optimization, translational proof-of-concept studies, and preparations for an IND application. The company aims to develop compounds that maintain therapeutic efficacy while minimizing hallucinogenic effects and avoiding cardiac risks. This initiative addresses a critical global health crisis, with OUD affecting 16 million people worldwide and causing over 120,000 annual deaths, with associated costs exceeding $750 billion annually.

Positive

• Secured significant non-dilutive funding of $11.4 million from NIH/NIDA
• First external validation of atai's AI-driven drug discovery approach
• Addresses massive market opportunity with OUD affecting 16 million people globally
• Potential to develop improved treatment option without hallucinogenic effects

Negative

• Early-stage development with significant clinical and regulatory hurdles ahead
• Success dependent on meeting specific development milestones
• Competitive market with existing OUD treatments

Insights

Pharmaceutical Research Analyst

ATAI secured $11.4M NIH grant to develop non-hallucinogenic treatments for opioid addiction, validating their AI drug discovery approach.

atai Life Sciences has secured a significant milestone-driven grant of up to $11.4 million from the National Institute on Drug Abuse (NIDA), marking the first external validation of the company's AI-driven polypharmacology drug discovery approach. This non-dilutive funding will accelerate the development of their novel 5-HT2A/2C receptor agonists designed to treat opioid use disorder (OUD) without hallucinogenic effects.

The UG3/UH3 grant structure is particularly noteworthy as it's a competitive, milestone-based funding mechanism reserved for promising translational research. The funding will support critical development stages including lead optimization, proof-of-concept studies, and the necessary toxicology and manufacturing work to file an Investigational New Drug (IND) application – effectively de-risking early development without equity dilution.

This program targets a significant unmet need, as OUD affects approximately 16 million people globally with annual costs exceeding $750 billion. Current OUD treatments often require daily administration, creating a significant treatment burden. 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 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. While this program remains pre-clinical, the federal backing represents meaningful validation of atai's scientific approach and accelerates their timeline toward first-in-human trials.

5-year UG3/UH3 grant will fund the optimization and early-stage development of atai’s novel 5-HT2A/2C receptor agonists with non-hallucinogenic potential for opioid use disorder

NEW YORK and AMSTERDAM, Sept. 18, 2025 (GLOBE NEWSWIRE) -- atai Life Sciences (NASDAQ: ATAI) (“atai” or “Company”), a clinical-stage biopharmaceutical company on a mission to develop highly effective mental health treatments to transform patient outcomes, today announced that it has been awarded a multi-year, milestone-driven grant worth up to $11.4 million by the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH). The UG3/UH3 grant will fund the optimization and early-stage development of atai’s novel 5-HT2A/2C receptor agonists with non-hallucinogenic potential for opioid use disorder (OUD).

“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. This recognition helps to 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.”

“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 provides non-dilutive funding to advance atai’s 5-HT2A/2C agonist program, focused on developing novel compounds that modulate serotonin receptors implicated in addiction. The funding will support lead optimization, translational proof-of-concept studies, and the toxicology and manufacturing work needed to file an Investigational New Drug (IND) application. The goal is to identify clinical candidates that maintain therapeutic activity against opioid use disorder while minimizing hallucinogenic effects and avoiding 5-HT2B activity, which has been linked to cardiac valvulopathy. If early-stage development milestones are met, atai plans to progress the program into a first-in-human Phase 1 study.

OUD affects 16 million people globally, costing >$750 billion annually.^1,2 More than 120,000 opioid-related deaths occur annually worldwide.^1,3 atai’s novel 5-HT2A/2C receptor agonists with non-hallucinogenic potential could support lasting abstinence while reducing the daily treatment burden seen with current OUD therapies.

About atai’s Discovery Program
As part of atai’s ongoing commitment to developing innovative mental health treatments, the company’s discovery program has identified a new class of differentiated 5-HT2A/2C receptor agonists. These novel compounds demonstrate non-hallucinogenic potential, as evidenced by their inability to fully substitute for traditional psychedelics in pre-clinical studies. atai is actively optimizing these compounds and evaluating their therapeutic potential across a range of preclinical models, with the goal of developing safer, polypharmacologic targeted treatments for mental health conditions including substance use disorders, anxiety and depression.

About atai Life Sciences
atai is a clinical-stage biopharmaceutical company on a mission to develop highly effective mental health treatments to transform patient outcomes. Our pipeline of psychedelic-based therapies includes BPL-003 (intranasal mebufotenin benzoate) for treatment-resistant depression (TRD), which is being advanced through a strategic investment with Beckley Psytech Limited; VLS-01 (buccal film DMT) also for TRD; and EMP-01 (oral R-MDMA) for social anxiety disorder. All three programs are in Phase 2 clinical development. We are also advancing a drug discovery program to identify novel, non-hallucinogenic 5-HT2AR agonists for TRD. These programs aim to address the complex nature of mental health providing commercially scalable interventional psychiatry therapies that can integrate seamlessly into healthcare systems. For the latest updates and to learn more about our mission, visit www.atai.com or follow us on LinkedIn.

Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). The words “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “anticipate,” “initiate,” “could,” “would,” “project,” “plan,” “potentially,” “preliminary,” “likely,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements include express or implied statements relating to, among other things: our business strategy and plans; the timing, potential success, and plans for atai’s 5-HT2A/2C receptor agonists and the plans and objectives of management for future operations, research and development and capital expenditures.

Forward-looking statements are neither promises nor guarantees, but involve known and unknown risks and uncertainties that could cause actual results to differ materially from those projected, including, without limitation, the important factors described in the section titled “Risk Factors” in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”), as such factors may be updated from time to time in atai's other filings with the SEC. atai disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by applicable law..

The grant reported in this press release is awarded by the National Institute on Drug Abuse of the National Institutes of Health under number 1UG3DA064403-01. The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. 

Contact Information
Investor Contact:
IR@atai.com

Media Contact:
PR@atai.com

References (to be cross-validated)
1. Dydyk AM, Jain NK, Gupta M. Opioid Use Disorder. StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 [cited 2024 May 24]. PMID: 31985959
2. Murphy SM. The cost of opioid use disorder and the value of aversion. Drug Alcohol Depend. 2020 Dec 1;217:108382. PMCID: PMC7737485
3. Jones G, Ricard JA, Lipson J, Nock MK. Associations between classic psychedelics and opioid use disorder in a nationally-representative U.S. adult sample. Sci Rep. 2022 Apr 7;12(1):4099. PMCID: PMC8990065

FAQ

What is the value and purpose of ATAI's new NIH grant?

ATAI received an $11.4 million UG3/UH3 grant from NIDA to develop non-hallucinogenic 5-HT2A/2C receptor agonists for treating opioid use disorder.

How many people are affected by opioid use disorder globally?

Opioid use disorder affects 16 million people globally, causing over 120,000 deaths annually and costing more than $750 billion per year.

What makes ATAI's approach to treating OUD unique?

ATAI is developing novel non-hallucinogenic 5-HT2A/2C receptor agonists using an AI-driven polypharmacology approach, aiming to maintain therapeutic benefits while avoiding hallucinogenic effects and cardiac risks.

What are the next development steps for ATAI's OUD treatment?

ATAI will conduct lead optimization, translational proof-of-concept studies, and toxicology work to prepare for an IND application, followed by potential Phase 1 trials if milestones are met.

How will this grant impact ATAI's drug development timeline?

The non-dilutive funding will accelerate the development timeline for bringing these innovative OUD treatments to patients, supporting various stages from optimization through potential Phase 1 studies.