Navigating a Defining Moment in Biotech to Advance Global Growth in 2026 and Beyond

The biopharmaceutical industry is at a pivotal moment. Scientific advances are accelerating, but so are patient expectations and technological advancements, while access to treatment is increasingly being impacted by rapidly-shifting regulatory policies and priorities. These global realities are reshaping what it takes to advance a therapy from discovery to delivery.

For Acadia, these factors serve to clarify our priorities for 2026 and beyond: build a global neuroscience and rare disease company designed for sustained growth, which turns scientific promise into meaningful innovation for underserved neurological and rare disease communities around the world.

That focus means staying disciplined in where and how we invest. We are prioritizing neurological and rare diseases where unmet need remains high, advancing a pipeline built for long-term impact, and strengthening the capabilities required to operate at global scale. Equally important, we are grounding our decisions in scientific rigor, responsible use of data and technology, and close engagement with patients and caregivers, ensuring that innovation is shaped not only by what is possible, but by what truly matters to the communities we serve.

Building Long-Term Growth in Neurological and Rare Diseases

We are building sustainable growth across a focused portfolio in neurological and rare diseases. Our growth is built on the strong foundation of our core commercial franchise, made up of approved treatments for Parkinson’s disease-related hallucinations and delusions and for Rett syndrome. These therapies have established our credibility as a company willing to take on some of the most complex conditions.

From that solid footing, we are advancing a robust late-stage pipeline that targets large, underserved populations. This includes programs in Alzheimer’s disease psychosis, Lewy body dementia (LBD) with psychosis, and major depressive disorder – conditions that collectively affect millions of people and remain inadequately treated. These programs represent significant opportunities to apply precision medicine innovation at scale in neurological disorders. For example, we are embedding exploratory biomarkers in both our Alzheimer’s disease psychosis and LBD trials to help understand whether certain patient subsets respond better to treatment. These biomarker results could help inform our trial design – and ensure that any product we launch can be as specific and targeted to the appropriate patient populations as possible.

At the same time, we are investing in an innovative early-stage pipeline that positions us for long-term impact. Our disclosed programs span essential tremor, tardive dyskinesia, Rett syndrome and Fragile X, Huntington’s disease, and SYNGAP1-related disorders, alongside multiple undisclosed programs. Together, these reflect a deliberate balance between near-term opportunity and future innovation.

Supporting this growth are four core drivers of transformation critical to our strategy:

• Precision Medicine, including an increasing focus on biomarkers from the earliest stages of development.
• Data Innovation, involving transforming how we discover and develop medicines through digital tools, artificial intelligence, and machine learning.
• Globalization, building targeted capabilities beyond the United States (including in markets across Europe, Japan, and Canada) to meet patient needs at global scale.
• Patient Empowerment, creating a connected ecosystem of patients, caregivers, and advocates to turn lived experience into actionable insight.

How Are We Turning Transformation Drivers into Action?

Biotech leadership is earned through decisive execution, not aspiration alone. We are converting ambition into real-world action and measurable impact.

A clear example is our decision to develop a new powder-for-oral-solution formulation of our approved treatment for Rett syndrome, which will be available on a limited basis starting in the first quarter of 2026 with broader availability anticipated in the second quarter. This innovation, which provides caregivers with greater flexibility to modify the taste and volume of their loved one’s dose, was informed directly by feedback from patients, caregivers, and healthcare providers about what would make a meaningful difference in daily care. By responding to the community’s feedback, we aim to offer greater flexibility and choice while maintaining the therapeutic integrity patients rely on.

We are also investing in strong data governance frameworks and fostering a culture of accountability, where every colleague understands their role in maintaining data integrity and quality. As we increasingly harness technology and data, this foundation is essential – not only to enable responsible, AI-driven innovation, but to preserve trust with patients, regulators, and partners.

Meeting the Moment Through Science, Technology, and Transparency

The external environment for the biopharmaceutical industry is changing rapidly, and Acadia is meeting this moment head-on. Pricing pressures continue to intensify, requiring discipline and focus. Patients and families are demanding greater transparency and treatments that reflect their lived experiences, not just clinical endpoints. Advances in genetics, biomarker science, and artificial intelligence are also transforming what is possible in understanding and addressing disease.

These forces are not separate from our strategy. They are shaping it. We are building a pipeline and organization designed to thrive in this reality, integrating patient insight, precision science, and responsible innovation.

Across our pipeline, momentum is building. We have eight disclosed programs and multiple undisclosed programs, with five additional Phase 2 or Phase 3 study initiations expected by the end of 2026, and four Phase 2 or Phase 3 study readouts anticipated by the end of 2027.

Artificial intelligence is accelerating this progress. In commercial operations, AI-powered analytics help our teams better target engagement. In R&D, clinical command centers monitor trials in real time to identify and mitigate risk. In regulatory functions, AI-enabled medical writing expands internal capacity and accelerates document development. Across these applications, technology is supporting better decisions, faster learning, and greater impact.

A Transformative Year Ahead

Looking ahead, 2026 represents a transformative year for Acadia. We expect key regulatory opinions that will mark important steps in our global expansion efforts, including for our Rett syndrome treatment. We also anticipate continued progress across our late-stage neurological disease programs, including emerging therapies for Alzheimer’s disease psychosis.

More broadly, this year will focus on deepening our mission: transforming scientific possibility into real-world impact.

The road ahead will not be simple. The science is complex, the challenges are real, and the stakes are high. But that is exactly why we are here. By staying focused on patients, investing in our core capabilities, and executing with discipline and purpose, I believe Acadia is well positioned to become a biotech powerhouse – one defined not just by growth, but how we can truly be the difference in people’s lives.