Corporate Presentation May 13, 2015

Corporate Presentation

May 13, 2015

Forward-Looking Statement

This presentation contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. Each of these statements is based only on current information, assumptions and expectations that are inherently subject to change and involve a number of risks and uncertainties. Forward-looking statements include, but are not limited to statements about (i) the plans for, including timing and progress of, clinical development, clinical trials and commercialization for our product candidates, including NUPLAZID™ (pimavanserin); (ii) the timing of any submission or application for, or receipt of, regulatory clearances and approvals, any potential approval of NUPLAZID as a first-in-class drug for PDP or potential approval for other indications; (iii) the benefits to be derived from and efficacy of our product candidates, including the clinical benefits of NUPLAZID, in PDP, ADP, schizophrenia or other neurological or psychiatric indications, the potential advantages of NUPLAZID versus existing antipsychotics, the potential for NUPLAZID to represent a new class of psychosis medicine, and the expansion opportunities for NUPLAZID; (iv) estimates regarding the prevalence of PD, PDP, ADP or schizophrenia; (v) the potential market for any of our product candidates, including NUPLAZID; and (vi) our estimates regarding our cash position or capital requirements.

In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward looking statements. Given the risks and uncertainties, you should not place undue reliance on forward-looking statements. For a discussion of the risks and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2014, as well as our subsequent filings with the SEC. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them for future events.

ACADIA: A CNS Focused Biopharmaceutical Company

•

Building a leading U.S. specialty CNS franchise

•

NUPLAZID™ (pimavanserin), a differentiated and potential new class of

psychosis therapy

‒ Selective serotonin inverse agonist preferentially targeting 5-HT_2A receptors

‒ Potential to be first and only drug approved in U.S. for Parkinson’s disease psychosis (PDP) – NDA planned for 2H 2015

‒ Demonstrated strong efficacy and favorable safety profile in Phase III PDP trial

‒ FDA granted Breakthrough Therapy designation in 2014

‒ Opportunity for pimavanserin to expand into broad range of neurological and psychiatric indications

•

Worldwide commercialization rights to NUPLAZID

•

U.S. patents go into 2028

Pipeline

COMPOUND/

PROGRAM INDICATION IND-TRACK PHASE I PHASE II PHASE III

REGULATORY APPROVAL COMMERCIALIZATION RIGHTS NUPLAZID™ (pimavanserin) Parkinson’s Disease Psychosis Alzheimer’s Disease Psychosis Schizophrenia ACADIA

Adrenergic Chronic Pain Allergan

Muscarinic Glaucoma Allergan

Parkinson’s Disease Psychosis

An Unmet Medical Need

• Characterized by hallucinations and

delusions

• Chronic disorder; worsens over time

and severely impacts daily living

• Afflicts about 40% of the 1 million

Parkinson’s patients in the U.S.

• Leading cause of nursing home

placement of Parkinson’s patients

Current Antipsychotics Not Approved for PDP and Increase

Mortality and Morbidity

• Can counteract PD dopamine replacement therapy resulting in a worsening

of motor symptoms

• Significant side effects are problematic for frail elderly population; sedation, stroke, hematological disorder, cardiovascular events, and cognitive impairment

• Not approved by the FDA for PDP

• Black box warning:

“Increased mortality in elderly patients with dementia-related psychosis. Elderly patients with dementia-dementia-related

psychosis treated with antipsychotic drugs are at an increased risk of death.”

NUPLAZID: Differentiation From Atypical Antipsychotics

NUPLAZID’s selective, non-dopaminergic profile enables treatment of PDP without compromising motor control

5-HT_2A D₂ H₁  NUPLAZID™  ― ― ― Seroquel     Zyprexa     Risperidone   ―  Clozapine    

NUPLAZID: Key Findings From Phase III -020 Study

• 6-week double blind placebo-controlled study in 199 PDP patients randomized to 40 mg of NUPLAZID or placebo (1:1)

• Highly significant and clinically meaningful improvement in psychosis

• Significant improvement on all additional efficacy measures: nighttime sleep, daytime wakefulness and caregiver burden

• Favorable safety and tolerability profile - no worsening of motor function

-8 -6 -4 -2 0 1 15 29 43 SAPS -PD Impr ov eme n t (LSM ± SE) Study Day Placebo 40 mg NUPLAZID p = 0.001 p = 0.037

-020 Study: NUPLAZID Demonstrated Highly Significant

Reduction in Psychosis

Change from Baseline to Day 43 (LSM + SE)

SAPS-PD (primary endpoint)

-020 Study: NUPLAZID Showed Highly Significant Efficacy as

Assessed by Different Raters and Scales

Endpoint Rater p-value*

SAPS-PD Centralized Independent

0.001

0.001

_0.001

_<0.001

-020 Study: NUPLAZID Improved Nighttime Sleep and

Daytime Wakefulness

(ITT, N=185; change from baseline) Nighttime Sleep

(ITT, N=185; change from baseline)

SC OP A I mpr ov emen t (L SM  SE ) SC OP A I mpr ov emen t (L SM  SE )

-020 Study: NUPLAZID Reduced Caregiver Burden

Caregiver Burden

NUPLAZID PDP Program

Open-Label Safety Extension Studies

•

~800 patient years of exposure in PDP

– > 250 patients treated ≥ 1 year

– > 100 patients treated ≥ 2 years

– Longest patient exposure > 9 years

•

Favorable long-term safety and tolerability profile observed to date in

fragile, elderly patients

Parkinson’s Disease Psychosis

Patient Profile

• Average age around 74 years

• Around 60/40 split between men and

women

• Over 70% suffer comorbid sleep

disturbances

• Almost 90% have caregiver support

Parkinson’s Disease Psychosis

ACADIA Market Research

•

Treating physicians surveyed were dissatisfied with use of atypical

antipsychotics for PDP patients:

– Safety and tolerability issues

– Black-box warning

– Impact on motor function

•

Physicians’ top-ranked attributes for PDP product:

– “Does not negatively impact motor symptoms”

– “Resolves psychosis fully”

– “Low incidence of side effects”

•

These top-ranked attributes compare favorably with the profile we have

observed with NUPLAZID in the clinic

PDP: U.S. Payer Landscape

• ACADIA has conducted foundational access and reimbursement research with key decision makers for payers covering 168 million lives:

• Payers responded favorably to NUPLAZID’s target product profile. Most valued attributes:

– Reduction in psychotic symptoms

– Safety and tolerability profile Payer Landscape Medicare Part D Standard Medicaid Medicare Part D LIS Commercial

PDP-Treating Physicians Landscape

• 11,000 PDP-Treating Physicians:

- Neurologists comprise the largest group

- Psychiatrists

- Long-term care physicians

• 135 Sales Reps will be hired at approval

Commercial Preparations for Planned Launch of NUPLAZID

Executing on Plan

•

Building out ACADIA commercial organization:

– Preparing commercial infrastructure and systems, including supply chain distribution

– Planning to hire 135 sales representatives at approval

•

Completed extensive market research to understand:

– PDP market landscape

– Physicians’ current treatment behaviors, view of unmet medical need and prescribing patterns

– PDP patients’ and caregivers’ needs

•

Conducted national and regional scientific advisory boards

•

Launching PDP disease awareness campaign

Pimavanserin Life Cycle Management

PDP Provides Strategic Entry Into Other Indications

Psychiatric

Neurological

Psychosis

Alzheimer’s

Lewy Body Dementia

Schizophrenia Depression

Mania

Pimavanserin has the potential to be a transformative advancement in the treatment of psychosis

Alzheimer’s Disease Psychosis (ADP)

Neurology Expansion Opportunity for Pimavanserin

• ADP afflicts 25% to 50% of the 5.2 million Alzheimer’s disease patients in U.S.

• No drug approved by FDA for ADP

• Current antipsychotics have black box warning for use in elderly demented patients

• MOA and safety profile of pimavanserin potentially attractive for ADP

• Development and regulatory synergies with PDP

Phase II ADP Trial (-019 Study): Design

Patient Pathway From Screening to Treatment Period

Phase II Efficacy, Tolerability and Safety Study

Location Nursing homes at Biomedical Research Centre for Mental Health, Kings College

Patients Target enrollment of 200 ADP patients

Type of design Randomized, double-blind, placebo-controlled Key efficacy endpoints NPI-NH, CMAI-SF, ADCS-CGIC

12-Week Blinded Treatment Period

Baseline 4-Week Visit 6-Week Key Endpoint 12-Week Cognitive Endpoint Screening NPI-NH 2-Week Visit

BPST Run-In 40 mg PIM or PBO (1:1)

9-Week Visit

Schizophrenia

Psychiatric Expansion Opportunity for Pimavanserin

• A debilitating lifelong disease afflicting 1% of population

• Current therapies are sub-optimal • Pimavanserin profile may allow for an

improved schizophrenia therapy

— Phase II PoC demonstrated

advantages of co-therapy

— Potential use as stand-alone

maintenance therapy to improve compliance

0.0 5.0 10.0 15.0 20.0 25.0 30.0 PANSS Im pr ov emen t

Pimavanserin Co-Therapy

Phase II Schizophrenia Trial

Pimavanserin co-therapy (PIM/RIS) demonstrated equivalent efficacy with less weight gain

0.0 0.5 1.0 1.5 2.0 2.5 3.0 W eigh t Chang e (kg )

Change in Mean PANSS Score from Baseline

Mean Change in Weight from Baseline

p = 0.05 p = 0.007

RIS LD PIM/RIS RIS HD

Corporate Information

Profile:

• Based in San Diego • 130 employees

Financial:

• Cash position at

March 31, 2015(1)_{: $298M}

ACADIA

Key Priorities

•

NUPLAZID - Parkinson’s disease psychosis

– Submit NDA to FDA in 2H 15

– Submit MAA to EMA around six to nine months following NDA submission

– Execute on commercial preparations for successful launch of NUPLAZID in U.S.

•

Pimavanserin - Alzheimer’s disease psychosis

– Complete enrollment in Phase II study around the end of the year

•

Pimavanserin life cycle management

– Initiate Phase II study with pimavanserin in PD patients with sleep disturbances following NDA submission

– Initiate Phase II study with pimavanserin in patients with schizophrenia around the end of the year

ACADIA: A CNS Focused Biopharmaceutical Company

•

Building a leading U.S. specialty CNS franchise

•

NUPLAZID™ (pimavanserin), a differentiated and potential new class of

psychosis therapy

‒ Selective serotonin inverse agonist preferentially targeting 5-HT_2A receptors

‒ Potential to be first and only drug approved in U.S. for Parkinson’s disease psychosis (PDP) – NDA planned for 2H 2015

‒ Demonstrated strong efficacy and favorable safety profile in Phase III PDP trial

‒ FDA granted Breakthrough Therapy designation in 2014

‒ Opportunity for pimavanserin to expand into broad range of neurological and psychiatric indications