Leading a New Era of Patient-Inspired Neuroscience

Originally published as a sponsored article in FiercePharma in January 2024. Republished with permission.

The need for innovation in neuroscience remains urgent. Patients, families and communities around the world carry the massive burden of suffering caused by diseases and disorders of the central nervous system. Despite scientific progress in understanding the brain’s complexities, much remains to learn about its structure, function, and role in neurological and psychiatric disorders.

Alkermes enters 2024 as a standalone, focused neuroscience company poised for continued growth, following a transformational year for our business. With an established commercial portfolio providing real-world insight into conditions facing patients with serious mental illness, and a research pipeline built around our core medicinal chemistry and neurobiology capabilities, we are uniquely positioned to innovate. Our singular vision is to develop new, differentiated medicines and bring them to patients as expeditiously as possible, representing a real evolution of Alkermes’ focus.

CNS Foundation and Future

Our origins began in drug formulation and delivery, to improve the pharmacology of existing drug molecules – through formulation, prodrug chemistry and advanced delivery technologies – and address unmet patient need. The science was complex and the logic simple: that we could improve treatment options for patients suffering from serious chronic diseases. As our experience and scientific capabilities grew, we evolved deliberately into a developer of proprietary new medicines for people living with complex, difficult-to-treat psychiatric and neurological disorders. Today, we proudly commercialize internally developed medicines for the treatment of schizophrenia, bipolar I disorder, alcohol dependence and opioid dependence.

We continue our pursuit of great science to develop novel medicines for neurological conditions needing innovation to advance the state of the art and make a meaningful difference to patients.

Patient-Inspired Science Fueling Advances in Rare Disease

Narcolepsy, characterized by excessive daytime sleepiness, is a chronic neurological disorder affecting the brain’s ability to regulate sleep-wake cycles. An estimated 200,000 people in the U.S. live with this condition, which takes an immense toll on everyday life.¹

Therapeutics for narcolepsy focus on symptom management. Even with the use of these current medicines, the management of symptoms remains a profound challenge for patients, their families and providers. Many patients report that medications are not improving their complete range of symptoms.²

In contrast, we are focused on researching and developing therapies with the potential to modify the disease itself. Narcolepsy is caused by dysfunction in neurocircuitry related to the natural sleep-wake cycle, which relies on a neuropeptide called orexin. Our development program in this space is based on the strong biological rationale for the use of orexin agonists to address the root cause of disease.

This fall, at the 2023 World Sleep Congress in Brazil, we shared initial data from a phase 1 study evaluating ALKS 2680, our investigational lead orexin agonist, in healthy volunteers, and proof-of-concept data from the first cohort of four patients with narcolepsy type 1 (NT1). These data support our design goal for ALKS 2680 – to increase daytime wakefulness with a once-daily oral dose. We continue advancing this important research program, with a phase 2 study planned for the first half of this year, while we continue gathering phase 1 data from patients living with narcolepsy type 2 and idiopathic hypersomnia (IH).

Core to our mission at Alkermes, we believe in incorporating the patient voice across the development process. We have conducted research to better understand the lived experience of people affected by narcolepsy and IH, and our planned phase 2 program will include patient-reported outcome measures to assess how potential treatment options may impact the real-world challenges they face. Ultimately, this information may help inform future studies and regulatory interactions.  

Pipeline for Growth

Our deep neuroscience expertise allowed Alkermes to develop a distinct proprietary commercial portfolio and fuels our ability to thoughtfully invest in R&D in areas with unmet need where we believe we can make a lasting difference.

Neurological and psychiatric disorders are complex to manage, can significantly impair daily functioning, and can deprive people of their independence and aspects of life often taken for granted. As just one example, there are many conditions for which excessive, debilitating fatigue is a hallmark – with narcolepsy being the most well-known – and compelling need exists for continued development. We believe our narcolepsy research program provides a foundation to explore additional pipeline opportunities targeting the brain’s wakefulness circuitry, and we will continue following the science toward potential innovative new treatment options.

From our roots in new formulations of existing drugs to reduce treatment burden and improve tolerability, to today’s focus on developing innovative new medicines using advanced molecular design, our past successes energize our pursuit of new medicines for psychiatric and neurological disorders. This is a challenging area of drug development, fraught with risk and frequent failure. But with this comes potential for significant benefit, measured by the profound impact new treatment options can have on patients, families and communities.

References:

¹ Global Narcolepsy Drugs Market, Forecast 2019-2025.  Allied Market Research

² Bhattarai, J., & Sumerall, S. (2017). Current and Future Treatment Options for Narcolepsy: A Review. Sleep science (Sao Paulo, Brazil), 10(1), 19–27. https://doi.org/10.5935/1984-0063.20170004