Jefferies Healthcare Conference June 2, 2015 Creating the Next Generation of CNS Drugs
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. 2
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 3
Pipeline COMPOUND/ REGULATORY COMMERCIALIZATION INDICATION IND-TRACK PHASE I PHASE II PHASE III PROGRAM APPROVAL RIGHTS Parkinson’s Disease NUPLAZID™ (pimavanserin) Psychosis Alzheimer’s Disease ACADIA Psychosis Schizophrenia Adrenergic Chronic Pain Allergan Muscarinic Glaucoma Allergan 4
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 • No drug approved by FDA for PDP 5
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-related psychosis treated with antipsychotic drugs are at an increased risk of death.” 6
NUPLAZID: Differentiation From Atypical Antipsychotics 5-HT 2A D 2 H 1 ― ― ― NUPLAZID™ Seroquel Zyprexa ― Risperidone Clozapine NUPLAZID’s selective, non-dopaminergic profile enables treatment of PDP without compromising motor control 7
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 Source: Cummings et al., Lancet (2014) 383 8
-020 Study: NUPLAZID Demonstrated Highly Significant Reduction in Psychosis SAPS-PD (Primary Endpoint) SAPS-PD Improvement (LSM ± SE) 0 -2 -4 -6 p = 0.037 Placebo p = 0.001 40 mg NUPLAZID -8 1 15 29 43 Study Day CGI-S CGI-I Change in CGI-Severity Score 0 4 CGI-Improvement (LSM ± SE) (LSM ± SE) -0.5 3.5 -1 3 p = 0.022 p = 0.010 Placebo Placebo p < 0.001 40 mg NUPLAZID 40 mg NUPLAZID p = 0.001 -1.5 2.5 1 15 29 43 1 15 29 43 Study Day Study Day 9
-020 Study: NUPLAZID Improved Nighttime Sleep, Daytime Wakefulness and Reduced Caregiver Burden Daytime Wakefulness Nighttime Sleep 0.5 0.5 SCOPA Improvement (LSM SE) SCOPA Improvement (LSM SE) 0 0 -0.5 -0.5 -1 -1 -1.5 -1.5 -2 -2 p = 0.045 p = 0.001 Placebo Placebo -2.5 -2.5 p = 0.012 40 mg NUPLAZID 40 mg NUPLAZID -3 -3 1 15 29 43 1 15 29 43 Study Day Study Day Caregiver Burden Caregiver Burden Improvement 2 0 (LSM SE) -2 -4 Placebo p = 0.002 40 mg NUPLAZID -6 1 15 29 43 Study Day 10
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 • Differentiation from off-label use of antipsychotics 11
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 with 74% requiring daily care Source: Based on ACADIA market research with over 800 PDP-treating physicians and over 700 PDP patient chart audits 12
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 Source: Based on ACADIA market research with over 800 PDP-treating physicians and over 700 PDP patient chart audits. 13
PDP: U.S. Payer Landscape • ACADIA has conducted foundational access and reimbursement research with key decision makers for payers covering 168 million lives: Payer Landscape Medicare Commercial Part D Standard Medicare Medicaid Part D LIS • Payers responded favorably to NUPLAZID’s target product profile. Most valued attributes: – Reduction in psychotic symptoms – Safety and tolerability profile – No worsening of motor function – Delaying institutionalization 14
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 around approval 15
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 around 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 16
PDP Disease Awareness Campaign • Neurology journal and digital placements creating over 500,000 impressions • PDP educational website targeting physicians • Educational programs with experts • Strong presence at neurology/psychiatry medical meetings 17
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