Exploring a New Frontier in Neuroscience

Original sponsored article published in the Boston Business Journal in August 2025. Republished with permission.

Long considered the world’s leading biotech hub, Boston and Cambridge are home to organizations and institutions responsible for some of today’s most cutting-edge scientific innovations. Alkermes is no exception, with its earliest beginnings in Cambridge. After nearly four decades and with most of our R&D and corporate workforce now based in Waltham, we’re proud of our Massachusetts roots and the contributions we’re making to further cement the area’s legacy of changing lives for the better.

In the field of neuroscience, we have more knowledge and technological capabilities available than ever before. Harnessing the understanding and application of the orexin pathway is one of the next great frontiers. With possible indications for sleep disorders and beyond, this emerging drug class has the potential to usher in transformative options across a range of therapeutic areas. While several companies have efforts underway, Alkermes’ relentless pursuit of great science and our neuroscience heritage has positioned us as a leader in this new space. This is an exciting time for patients and will further add to our local legacy of innovation.

Central nervous system (CNS) drug development is particularly complex, and Alkermes is well positioned to bring our decades of expertise in this work to bear, having developed and registered multiple small molecule CNS medicines. With our singular focus on neuroscience, Alkermes is setting the pace during what will be a pivotal few years for the field.

Promise of the Orexin Class to Address Unmet Need in Sleep Disorders

Sleep is an essential pillar of health, with known impacts across physical and mental health, cognitive function, and growth and development. Quality wakefulness, a related measure, is important for alertness, cognitive function, attention and mood.¹ For people living with chronic sleep disorders such as narcolepsy and idiopathic hypersomnia – neurologic disorders that are challenging to diagnose – maintaining a “normal” wake/sleep cycle is all but impossible. The burden of disease for those living with these disorders is high^2,3 and there remains a need for new treatment options.

Orexins are neuropeptides produced in the brain that regulate vital functions, including wake and sleep states, energy homeostasis, reward systems and cognition. Our lead investigational orexin 2 receptor (OX2R) agonist, ALKS 2680, is being evaluated in three phase 2 studies as a once-daily treatment option for people living with narcolepsy type 1 (NT1), narcolepsy type 2 (NT2) and idiopathic hypersomnia (IH). Alkermes is at the forefront of development, as ALKS 2680 is currently the only OX2R agonist with patient data across a range of doses that supported phase 2 initiation in NT1, NT2 and IH.

Unlocking a Wider Range of CNS Therapies

Beyond sleep disorders, we are actively exploring additional areas of clinical utility for novel orexin pharmacology. Project Saturn, our orexin portfolio expansion strategy, is researching the effects of orexin across multiple symptomatic domains, including wakefulness, mood, cognition, fatigue and attention. We’re currently pursuing multi-faceted preclinical research to evaluate the effects of other OX2R agonists as either monotherapy or combined with other mechanisms with the goal of extending the spectrum of pharmacologic activity. This work spans rare neurodegenerative and neurodevelopmental diseases to broader neuropsychiatric disorders, all seeking to leverage the OX2R agonist pathway’s broad, unrealized clinical potential. Progress is accelerating as we work to bring two of our investigational compounds into the clinic this year.

An Approach Guided by Patient Needs

As Alkermes continues to move into new areas of innovation, we’re led by strong patient-focused drug development processes, rooted in our fundamental understanding of the unmet needs facing patients living with complex brain disorders. We’re pursuing the greatest opportunities to apply our deep neuroscience and medicinal chemistry expertise towards R&D that we believe can have the biggest impact on delivering important new medicines across a broad range of disorders affecting the brain.

Unlocking the potential of orexin agonists represents a significant opportunity to advance the field of medicine and make a real impact in the lives of patients in Massachusetts and beyond – our purpose since the beginning.

References:

^{1 Centers for Disease Control and Prevention.  About sleep. Retrieved April 22, 2025, from https://www.cdc.gov/sleep/about/index.html} 

^{2 Flores NM, Villa KF, Black J, Chervin RD, Witt EA. The humanistic and economic burden of narcolepsy. Journal of Clinical Sleep Medicine. 2016;12(3):401-407.}

^{3 Tadrous R, O’Rourke D, Mockler D, Broderick J. Health‐related quality of life in narcolepsy: A systematic review and meta‐analysis. Journal of Sleep Research. 2021;30(6):e13383.}

Certain statements set forth in this article constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning the potential therapeutic looking statements due to various risks, assumptions and uncertainties, including those described under the heading “Risk Factors” in the company’s Annual Report on Form 10-K for the year ended Dec. 31, 2024 and in subsequent filings made by the company with the U.S. Securities and Exchange Commission (SEC), which are available on the SEC’s website at www.sec.gov, and on the company’s website at www.alkermes.com in the “Investors – SEC filings” section. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. Except as required by law, the company disclaims any intention or responsibility for updating or revising any forward-looking statements.