Alkermes (ALKS) investor relations material

Key clinical data and insights

• Recent NT2 study provided unprecedented data on orexin agonist dose response, tolerability, and safety, debunking prior assumptions about dose-limiting side effects at higher doses.

• Patient-level data revealed variability in response, with some NT2 patients showing no MWT improvement but normal ESS, highlighting the complexity of responder identification.

• Split dosing is being explored to extend efficacy throughout the day, with modeling suggesting it may benefit both responders and some non-responders.

• No dose-limiting adverse events were observed up to 18 mg, allowing for greater flexibility in dose selection and commercial use.

• Secondary endpoints such as cognition and fatigue are being analyzed, with early extension data suggesting persistent benefits similar to NT1 results.

Strategic and commercial considerations

• Acquisition of Avidel was initiated before NT2 data, aiming to establish presence in sleep centers and leverage synergies for future launches.

• Market modeling anticipates growth in narcolepsy treatment rates with new, well-tolerated therapies, and the potential for both orexin agonists and oxybates to coexist.

• Combining orexin agonists with sodium oxybate is considered a valuable clinical experiment, as each addresses different aspects of narcolepsy management.

• Flexibility in payer negotiations is enhanced by offering both branded treatments, supporting bundled approaches for refractory patients.

Forward-looking statements and pipeline

• Phase three for NT2 will proceed before IH split dose data, with confidence in modeling based on robust NT2 results.

• IH studies will use ESS and IHSS as primary endpoints, with MWT as a secondary measure to assess variability.

• Confidence in orexin agonists for broader indications, such as ADHD and Alzheimer's adjunct, is bolstered by NT2 data showing efficacy in patients with baseline orexin tone.

• Human data for backup orexin programs expected after completion of SAD and MAD studies, with translational studies likely in 2026.