Alkermes (ALKS) Goldman Sachs 46th Annual Global Healthcare Conference summary

Strategic evolution and scientific focus

• Transitioned from drug delivery and royalty business to proprietary products with a $1B top line, now focused on advancing up the value chain through novel molecule development.

• Emphasis on neuroscience, particularly the orexin pathway, leveraging in-house chemistry expertise to design complex molecules targeting sleep-wake regulation.

• Current macro environment favors innovative molecules with high pricing power and less payer control, supporting sustained profitability and pipeline growth.

Orexin agonist program and clinical development

• Orexin agonists show promise in directly enhancing wakefulness by mimicking natural neuropeptides, with robust dose-dependent efficacy in NT1, NT2, and IH populations.

• Phase 1B demonstrated clear, dose-dependent increases in wakefulness, informing the design of ongoing phase II Vibrance studies across multiple patient groups.

• Vibrance-1 (NT1) is expected to bracket optimal dosing for phase III, while Vibrance-2 (NT2) explores higher, variable doses due to disease heterogeneity.

• Primary endpoints include Maintenance of Wakefulness Test (MWT), Epworth Sleepiness Scale, and cataplexy rates, with secondary analyses on patient-reported outcomes and cognition.

Safety, tolerability, and patient experience

• Adverse events have been mild to moderate and transient, mainly insomnia and polyuria, with no discontinuations in phase 1B; ongoing monitoring for visual disturbances and other potential effects.

• Patient and physician excitement is high due to the potential for superior efficacy and improved quality of wakefulness compared to current treatments.

• Post-double-blind dosing flexibility in phase II will inform real-world dose optimization and patient preferences for phase III.