Neuphoria Therapeutics announced significant progress across its clinical pipeline, highlighted by a strategic partnership with Merck that has advanced to Phase 2 clinical trials for Alzheimer's disease treatment. The company also reported continued momentum with its lead candidate BNC210 in late-stage development for anxiety disorders.
The Burlington, Massachusetts-based clinical-stage biotechnology company received a US$15 million milestone payment from Merck in March 2025, following Merck's initiation of a Phase 2 clinical trial evaluating MK-1167, a partnered α7 nicotinic acetylcholine receptor positive allosteric modulator (PAM), for treating symptoms of Alzheimer's disease dementia.
"2024 and the first quarter of 2025 were transformational for our pipeline," said Spyros Papapetropoulos, M.D., Ph.D., President and CEO of Neuphoria. "Our internal BNC210 program entered Phase 3 in Social Anxiety Disorder with robust enrollment, and our partnered α7 nicotinic acetylcholine receptor positive allosteric modulator program with Merck for the treatment of the symptoms of Alzheimer's disease dementia advanced to Phase 2."
BNC210 Clinical Development Progress
Neuphoria's lead candidate BNC210 is advancing in multiple neuropsychiatric indications. The company initiated the Phase 3 AFFIRM-1 trial in July 2024, evaluating 225mg BNC210 for the acute, as-needed treatment of anxiety in social anxiety disorder (SAD). The trial is progressing as planned with enrollment on track for topline results anticipated in Q3 2025.
The company previously reached an agreement with the U.S. Food and Drug Administration (FDA) on the design of BNC210's registrational program in SAD during an End-of-Phase 2 meeting held in Q3 2023. Results from the Phase 3-enabling PREVAIL Phase 2 study of BNC210 in SAD were published in Psychiatry Research in February 2025.
BNC210 has demonstrated rapid-onset, broad and meaningful anti-anxiety effects in completed clinical trials across multiple anxiety conditions, including:
- Panic attacks
- Generalized anxiety disorder (GAD)
- Social anxiety disorder (SAD)
Importantly, these effects were achieved without evidence of sedation, impairments in cognition, or addiction potential—common limitations of existing anxiety treatments.
For post-traumatic stress disorder (PTSD), Neuphoria held a successful End-of-Phase 2 meeting with the FDA in July 2024, establishing a potential registrational path for BNC210. The company plans to initiate a Phase 2b dose-ranging study in Q4 2025, following the release of topline results from the ongoing Phase 3 AFFIRM-1 trial in SAD.
This PTSD study will evaluate a lower dose of BNC210 (450 mg BID) compared to the 900mg BID used in the previous ATTUNE Phase 2b trial. Based on pharmacokinetic and pharmacodynamic analyses, the company expects the lower dose to provide similar efficacy while improving safety and tolerability.
Clinical data supporting BNC210's potential in PTSD was published in NEJM Evidence in December 2024, demonstrating clinically meaningful improvements across several key PTSD symptoms.
Strategic Partnerships Strengthen Financial Position
The $15 million milestone payment from Merck extends Neuphoria's cash runway to Q3 2026. Under the agreement, Neuphoria remains eligible to receive up to $450 million in additional milestone payments plus royalties on net sales of any licensed medicines.
"We started 2025 strong and with a focus on efficiently advancing our pipeline and increasing shareholder value through leveraging capital generated through our long-term partnerships with Merck and Carina Biotech," said Dr. Papapetropoulos.
The company also received a milestone payment of AUS$1 million in November 2024 from Carina Biotech for BNC101, a partnered legacy oncology program. This agreement could yield up to approximately AUS$117 million in additional payments if Carina successfully develops and markets the therapy.
Mechanism of Action and Therapeutic Potential
BNC210 is an oral, proprietary, selective negative allosteric modulator of the α7 nicotinic acetylcholine receptor. It represents a first-of-its-kind, broad-spectrum anti-anxiety experimental therapeutic designed to restore neurotransmitter balance in relevant brain areas.
The drug candidate's mechanism provides rapid relief from stress and anxiety symptoms without the common drawbacks of current treatments, including sedation, cognitive impairment, and addiction potential.
In addition to BNC210 and the Merck-partnered MK-1167, Neuphoria's pipeline includes next-generation α7 nicotinic acetylcholine receptor programs and Kv3.1/3.2 preclinical programs, both in the lead optimization development stage.
The company successfully re-domiciled from Australia to the United States in December 2024 and completed a private placement in May 2024 that enabled continued execution of its late-stage clinical programs.
As Neuphoria advances its diverse pipeline targeting significant unmet needs in neuropsychiatric disorders, the company appears well-positioned with both internal development programs and strategic partnerships to support its clinical and commercial objectives through 2025 and beyond.
