Corporate Presentation - Acadia Pharmaceuticals
Transcript of Corporate Presentation - Acadia Pharmaceuticals
Corporate Presentation
Creating the Next Generation of CNS Drugs
January 12, 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, 2013, 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.
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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
– Potential to be first and only drug approved in U.S. for Parkinson’s disease
psychosis (PDP)
– Demonstrated strong efficacy and favorable safety profile in Phase III PDP trial
• Broad opportunity for pimavanserin to expand into range of neurological
and psychiatric indications
– Potential to transform treatment paradigm in large underserved indications
• Worldwide commercialization rights to NUPLAZID
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NUPLAZID (Pimavanserin) Potential New Class of Psychosis Therapy
• Selective serotonin inverse agonist preferentially targeting 5-HT2A receptors
• Small molecule NCE discovered and developed by ACADIA
• FDA granted Breakthrough Therapy designation in 2014
• Completed Phase III PDP Program:
- NDA submission planned for Q1 15
- MAA submission planned six to nine months after NDA submission
• Conducting Phase II study in Alzheimer’s disease psychosis (ADP)
• Major life cycle expansion opportunities in neurological and psychiatric indications
• U.S. patents go into 2028
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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
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Current Antipsychotics Not Approved for PDP and Increase Mortality and Morbidity
• Can counteract PD dopamine replacement therapy resulting in a worsening of motoric 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.”
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NUPLAZID: Differentiation From Atypical Antipsychotics
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NUPLAZID’s selective, non-dopaminergic profile enables treatment of PDP without compromising motor control
5-HT2A D2 H1
NUPLAZID™ ― ― ―
Seroquel
Zyprexa
Risperidone ―
Clozapine
NUPLAZID: Key Findings From Phase III -020 Study
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• 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
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-6
-4
-2
0
1 15 29 43
SAP
S-P
D Im
pro
vem
en
t (
LSM
± S
E)
Study Day Placebo 40 mg NUPLAZID
p = 0.001
p = 0.037
-020 Study: NUPLAZID Demonstrated Highly Significant Reduction in Psychosis
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-2.73
-5.79
-7
-6
-5
-4
-3
-2
-1
0
Placebo 40 mg PIM
SAP
S-P
D I
mp
rove
me
nt
Change from Baseline to Day 43 (LSM + SE)
SAPS-PD (primary endpoint) (ITT, N=185; change from baseline)
-020 Study: NUPLAZID Showed Highly Significant Efficacy as Assessed by Different Raters and Scales
Endpoint Rater p-value*
SAPS-PD Centralized Independent
0.001
SAPS H+D Centralized Independent
0.001
CGI-I Investigator 0.001
CGI-S Investigator <0.001
*ITT, MMRM, OC
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-020 Study: NUPLAZID Improved Nighttime Sleep and Daytime Wakefulness
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-3
-2.5
-2
-1.5
-1
-0.5
0
0.5
1 15 29 43
Study Day
Placebo 40 mg NUPLAZID
p = 0.045 p = 0.001
-3
-2.5
-2
-1.5
-1
-0.5
0
0.5
1 15 29 43
Study Day
Placebo 40 mg NUPLAZID
p = 0.012
Daytime Wakefulness (ITT, N=185; change from baseline)
Nighttime Sleep (ITT, N=185; change from baseline)
SCO
PA Im
pro
vem
en
t (L
SM
SE)
SCO
PA Im
pro
vem
en
t (L
SM
SE)
-020 Study: NUPLAZID Reduced Caregiver Burden
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-6
-5
-4
-3
-2
-1
0
1
2
1 15 29 43
Car
egi
ver
Bu
rde
n Im
pro
vem
en
t (L
SM
SE)
Study Day Placebo 40 mg NUPLAZID
p = 0.002
Caregiver Burden (ITT, N=185; change from baseline)
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
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Parkinson’s Disease Psychosis Patient Profile
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• 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
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.
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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
– No worsening of motor function
– Delaying institutionalization
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Payer Landscape
Medicare Part D
Standard
Medicaid Medicare Part D LIS
Commercial
PDP-Treating Physicians Landscape
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• 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
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Pimavanserin Life Cycle Management PDP Provides Strategic Entry into Other Indications
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Psychiatric Neurological
Psychosis Parkinson’s 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
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• 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 initiated in late 2013
Phase II ADP Trial (-019 Study): Design
Patient Pathway From Screening to Treatment Period
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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
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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
PAN
SS Im
pro
vem
en
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
We
igh
t C
han
ge (
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 RIS LD PIM/RIS RIS HD
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Meltzer et al., Schizophrenia Research (2012)
Pipeline
COMPOUND/ PROGRAM
INDICATION DISCOVERY 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
ER Neuroprotection
and Chronic Pain ACADIA
Nurr-1 Parkinson’s Disease ACADIA
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Corporate Information
Profile:
• Based in San Diego
• 95 employees
Financial:
• Cash position at
September 30, 2014(1): $338M
(1) Reflects cash, cash equivalents and investment securities
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ACADIA Key Priorities
• NUPLAZID - Parkinson’s disease psychosis
– Submit NDA to FDA in Q1 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 by end of 2015
• Pimavanserin life cycle management
– Initiate Phase II study with pimavanserin in PD patients with sleep disturbances in H1 15
– Initiate Phase II study with pimavanserin in patients with schizophrenia in H2 15
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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
– Potential to be first and only drug approved in U.S. for Parkinson’s disease
psychosis (PDP)
– Demonstrated strong efficacy and favorable safety profile in Phase III PDP trial
• Broad opportunity for pimavanserin to expand into range of neurological
and psychiatric indications
– Potential to transform treatment paradigm in large underserved indications
• Worldwide commercialization rights to NUPLAZID
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Creating the Next Generation of CNS Drugs