Axsome Therapeutics Acquires Subtype Selective GABA-A Receptor Positive Allosteric Modulator AZD7325 for the Treatment of Epilepsy

Rhea-AI Summary

Axsome Therapeutics (NASDAQ: AXSM) acquired exclusive global rights to AZD7325, an oral GABAA α2,3 subtype-selective positive allosteric modulator, from AstraZeneca via purchase of 100% of Baergic Bio on Nov 6, 2025.

AZD7325 completed Phase 1 studies in over 700 patients with a favorable safety profile and showed anti‑convulsant effects in preclinical seizure models. Axsome plans Phase 2 trial‑enabling activities in 2026. Deal terms include a $0.3M upfront to Baergic shareholders, potential development/regulatory milestones of $2.5M (first indication) plus $1.5M per additional indication, up to $79M in sales‑based milestones, and tiered mid‑to‑high single‑digit royalties; AstraZeneca will receive a single‑digit million upfront, milestones, sales milestones, and royalties.

Positive

• Exclusive worldwide development and commercial rights to AZD7325
• AZD7325 completed Phase 1 studies in >700 patients with favorable safety
• Company plans Phase 2 trial‑enabling activities in 2026
• Acquisition structured via 100% equity purchase of Baergic Bio

Negative

• AZD7325 remains early stage (only Phase 1 complete) with development risk
• Contingent payments include up to $79M in sales milestones plus royalties
• AstraZeneca and Baergic stakeholders eligible for milestone and royalty payments

Insights

Portfolio Strategy Expert

Acquisition expands Axsome's CNS portfolio with an early‑stage epilepsy program and modest upfront obligations.

Axsome gains exclusive worldwide rights to AZD7325, a subtype‑selective GABAA α2,3 positive allosteric modulator, via acquisition of Baergic Bio and an amended license with AstraZeneca. This adds an oral, mechanism‑differentiated asset to an existing late‑stage CNS pipeline and transfers development, commercial, and manufacturing control to Axsome.

Key deal economics are explicit and modest: a $0.3 million upfront to Baergic Bio shareholders, potential development/regulatory payments of $2.5 million for the first indication and $1.5 million for subsequent indications, up to $79 million in sales‑based milestones, and a tiered mid‑to‑high single‑digit royalty to AstraZeneca; AstraZeneca also receives a single‑digit million cash upfront. These terms limit near‑term cash risk while preserving upside through milestones and royalties.

Watch execution risks and timelines: Axsome plans to begin Phase 2 enabling activities in 2026, so the near term will focus on preclinical/regulatory enabling work and trial design. Monitor announced study start dates, IND/CTA filings, and further milestone payments over the next 12–24 months.

Clinical Development Expert

AZD7325 has Phase 1 human data and preclinical anti‑convulsant signals, enabling Phase 2 entry after enabling work.

The asset completed Phase 1 and has been studied in over 700 patients with a reported favorable safety and tolerability profile; preclinical models showed anti‑convulsant effects. That factual profile supports a Phase 2 program focused on efficacy in epilepsy rather than de‑risking basic safety.

Dependencies include successful completion of Phase 2 enabling activities and trial design that link the α2,3 subtype selectivity to meaningful clinical endpoints in epilepsy. The stated plan to start enabling activities in 2026 creates a clear near‑term milestone to watch, along with any disclosed IND/CTA submissions and Phase 2 start dates over the following 12–36 months.

Axsome obtains exclusive global rights to AZD7325, a novel oral selective GABA_A α2,3 receptor positive allosteric modulator

Deepens Axsome’s broad and innovative neuroscience portfolio with a synergistic early-stage program for epilepsy

NEW YORK, Nov. 06, 2025 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced that it has entered into an agreement to obtain exclusive global rights to AZD7325, a novel oral GABA_A receptor α2,3 subtype-selective positive allosteric modulator (PAM), licensed from AstraZeneca AB (NASDAQ: AZN). AZD7325 has completed Phase 1 trials. Axsome intends to evaluate AZD7325 as a potential treatment for epilepsy and plans to begin Phase 2 trial-enabling activities in 2026.

“This transaction adds AZD7325, an earlier stage molecule with a differentiated mechanism of action, to our leading late-stage CNS portfolio,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “We are excited to further deepen our research pipeline in a complementary way, and to extend our clinical efforts into epilepsy, an area where there is an urgent need for innovative new treatment options for patients.”

AZD7325 has demonstrated anti-convulsant effects in preclinical seizure models. In clinical studies in over 700 patients to date, AZD7325 has demonstrated a favorable safety and tolerability profile.

Axsome will receive worldwide commercial, development, and manufacturing rights to AZD7325. The transaction was effectuated through Axsome’s acquisition of a 100% equity interest in Baergic Bio, Inc., a subsidiary of Avenue Therapeutics, Inc. (OTC: ATXI), and concurrent amendment to the License Agreement between Baergic Bio and AstraZeneca. Under the terms of the Baergic Bio Share Purchase Agreement, Baergic Bio shareholders will receive a $0.3 million upfront payment and are eligible to receive development and regulatory milestone payments of $2.5 million for the first indication and $1.5 million for each indication thereafter, up to $79 million in potential sales-based milestones, and a tiered mid-to-high single-digit royalty on potential global net sales of AZD7325. Under the terms of the License Amendment, AstraZeneca will receive a cash upfront payment in the single digit millions and is eligible to receive development and regulatory milestone payments for each indication, sales-based milestones, and a tiered mid-to-high single-digit royalty on potential global net sales of AZD7325.

About Epilepsy

Epilepsy is a chronic and debilitating neurological disorder that affects approximately 3.4 million people in the U.S., with about 150,000 new cases diagnosed each year.^1-3 It is characterized by recurrent, unprovoked seizures, or sudden and uncontrolled surges of electrical activity in the brain that can cause involuntary movements, sensory disturbances, loss of awareness, or convulsions.^3,4 People living with epilepsy often face stigma, barriers to education and employment, higher rates of comorbid psychiatric conditions, and increased risk of premature mortality, contributing to reduced quality of life and social isolation.^5-7 Despite currently available treatment options, more than one-third of patients do not respond to treatment.⁸

About Axsome Therapeutics

Axsome Therapeutics is a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) conditions. We deliver scientific breakthroughs by identifying critical gaps in care and develop differentiated products with a focus on novel mechanisms of action that enable meaningful advancements in patient outcomes. Our industry-leading neuroscience portfolio includes FDA-approved treatments for major depressive disorder, excessive daytime sleepiness associated with narcolepsy and obstructive sleep apnea, and migraine, and multiple late-stage development programs addressing a broad range of serious neurological and psychiatric conditions that impact over 150 million people in the United States. Together, we are on a mission to solve some of the brain’s biggest problems so patients and their loved ones can flourish. For more information, please visit us at www.axsome.com and follow us on LinkedIn and X.

Forward Looking Statements

Certain matters discussed in this press release are “forward-looking statements”. The Company may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the commercial success of the Company’s SUNOSI^®, AUVELITY^®, and SYMBRAVO^® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials, and/or data readouts, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of the Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration (“FDA”) or other regulatory authority approval of, or other action with respect to, the Company’s product candidates, including statements regarding the timing of any NDA submission; the Company’s ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the Company’s ability to successfully resolve any intellectual property litigation, and even if such disputes are settled, whether the applicable federal agencies will approve of such settlements; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company’s license agreements; the acceptance by the market of the Company’s products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the commercialization of SUNOSI, AUVELITY, and SYMBRAVO and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic and other factors, including general economic conditions and regulatory developments, not within the Company’s control. The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

Investors:
Ashley Dong
Director, Investor Relations
(929) 687-1614
adong@axsome.com

Media:
Darren Opland
Director, Corporate Communications
(929) 837-1065
dopland@axsome.com

References

1. CDC. Epilepsy Facts and Stats. https://www.cdc.gov/epilepsy/data-research/facts-stats. Accessed September 2025.
2. Cleveland Clinic. Epilepsy. https://my.clevelandclinic.org/health/diseases/17636-epilepsy. Accessed September 2025.
3. Institute of Medicine (US) Committee on the Public Health Dimensions of the Epilepsies; England MJ, Liverman CT, Schultz AM, et al., editors. Epilepsy Across the Spectrum: Promoting Health and Understanding. Washington (DC): National Academies Press (US); 2012. https://www.ncbi.nlm.nih.gov/books/NBK100589
4. National Institute of Neurological Disorders and Stroke. Epilepsy and Seizures. https://www.ninds.nih.gov/health-information/disorders/epilepsy-and-seizures. Accessed September 2025.
5. de Souza JL et al. The Perceived Social Stigma of People with Epilepsy with Regard to the Question of Employability. Neurol Res Int. 2018;2018:4140508. https://pubmed.ncbi.nlm.nih.gov/29862075
6. Mula M et al. Psychiatric Comorbidities in People with Epilepsy. Neurol Clin Pract. 2021 Apr;11(2):e112-e120. https://pubmed.ncbi.nlm.nih.gov/33842079
7. Thurman DJ et al. The burden of premature mortality of epilepsy in high-income countries: A systematic review from the Mortality Task Force of the International League Against Epilepsy. Epilepsia. 2017 Jan;58(1):17-26. https://pubmed.ncbi.nlm.nih.gov/27888514
8. Chen Z, et al. Treatment Outcomes in Patients With Newly Diagnosed Epilepsy Treated With Established and New Antiepileptic Drugs: A 30-Year Longitudinal Cohort Study. JAMA Neurol. Mar 1;75(3):279-286. https://pubmed.ncbi.nlm.nih.gov/29279892

FAQ

What did Axsome (AXSM) acquire on November 6, 2025?

Axsome acquired exclusive global rights to AZD7325 by buying 100% of Baergic Bio and amending the license from AstraZeneca.

How far along is AZD7325 development for epilepsy under AXSM?

AZD7325 completed Phase 1 studies in over 700 patients; Axsome plans Phase 2 trial‑enabling activities in 2026.

What are the financial terms of Axsome's AZD7325 deal (AXSM)?

Baergic shareholders get $0.3M upfront, development milestones of $2.5M (first indication) and $1.5M thereafter, up to $79M sales milestones, plus tiered mid‑to‑high single‑digit royalties; AstraZeneca receives a single‑digit million upfront, milestones, sales milestones, and royalties.

Will AXSM manufacture AZD7325 after the acquisition?

Axsome obtained worldwide development and manufacturing rights to AZD7325 under the transaction.

What clinical evidence supports AZD7325 as an epilepsy candidate?

Preclinical seizure models showed anti‑convulsant effects and clinical Phase 1 data in >700 patients demonstrated a favorable safety and tolerability profile.