Jefferies Healthcare Conference June 2, 2015 Creating the Next - - PowerPoint PPT Presentation

▶
jefferies healthcare conference
SMART_READER_LITE
LIVE PREVIEW

Jefferies Healthcare Conference June 2, 2015 Creating the Next - - PowerPoint PPT Presentation

Apr 15, 2023 453 likes •725 views

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

slide-1
SLIDE 1

Jefferies Healthcare Conference

June 2, 2015

Creating the Next Generation of CNS Drugs

slide-2
SLIDE 2

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

  • f, 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

slide-3
SLIDE 3

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-HT2A 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

slide-4
SLIDE 4

Pipeline

COMPOUND/ PROGRAM INDICATION IND-TRACK PHASE I PHASE II PHASE III REGULATORY APPROVAL COMMERCIALIZATION RIGHTS

NUPLAZID™ (pimavanserin) Parkinson’s Disease Psychosis ACADIA Alzheimer’s Disease Psychosis Schizophrenia Adrenergic Chronic Pain Allergan Muscarinic Glaucoma Allergan

4

slide-5
SLIDE 5

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

slide-6
SLIDE 6

Current Antipsychotics Not Approved for PDP and Increase Mortality and Morbidity

  • Can counteract PD dopamine replacement therapy resulting in a worsening
  • f 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

slide-7
SLIDE 7

NUPLAZID: Differentiation From Atypical Antipsychotics

NUPLAZID’s selective, non-dopaminergic profile enables treatment

  • f PDP without compromising motor control

5-HT2A D2 H1  NUPLAZID™  ― ― ― Seroquel     Zyprexa     Risperidone   ―  Clozapine    

7

slide-8
SLIDE 8

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

slide-9
SLIDE 9
  • 020 Study: NUPLAZID Demonstrated Highly Significant

Reduction in Psychosis

9

  • 8
  • 6
  • 4
  • 2

1 15 29 43 SAPS-PD Improvement (LSM ± SE) Study Day

SAPS-PD (Primary Endpoint)

Placebo 40 mg NUPLAZID p = 0.001 p = 0.037

2.5 3 3.5 4 1 15 29 43

CGI-Improvement (LSM ± SE) Study Day

CGI-I

Placebo 40 mg NUPLAZID p = 0.001 p = 0.010

  • 1.5
  • 1
  • 0.5

1 15 29 43

Change in CGI-Severity Score (LSM ± SE) Study Day

CGI-S

Placebo 40 mg NUPLAZID p = 0.022 p < 0.001

slide-10
SLIDE 10
  • 020 Study: NUPLAZID Improved Nighttime Sleep, Daytime

Wakefulness and Reduced Caregiver Burden

  • 6
  • 4
  • 2

2 1 15 29 43 Caregiver Burden Improvement (LSMSE) Study Day

Caregiver Burden

Placebo 40 mg NUPLAZID p = 0.002

10

  • 3
  • 2.5
  • 2
  • 1.5
  • 1
  • 0.5

0.5 1 15 29 43

Study Day

Nighttime Sleep

Placebo 40 mg NUPLAZID p = 0.045 p = 0.001

SCOPA Improvement (LSMSE)

  • 3
  • 2.5
  • 2
  • 1.5
  • 1
  • 0.5

0.5 1 15 29 43

Study Day

Daytime Wakefulness

Placebo 40 mg NUPLAZID p = 0.012

SCOPA Improvement (LSMSE)

slide-11
SLIDE 11

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

slide-12
SLIDE 12

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

slide-13
SLIDE 13

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
  • bserved 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

slide-14
SLIDE 14

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 Payer Landscape

Medicare Part D Standard Medicaid Medicare Part D LIS Commercial

14

slide-15
SLIDE 15

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

slide-16
SLIDE 16

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

slide-17
SLIDE 17

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

slide-18
SLIDE 18

Pimavanserin Life Cycle Management

PDP Provides Strategic Entry Into Other Indications

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

18

slide-19
SLIDE 19

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 ongoing

19

slide-20
SLIDE 20

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

20

slide-21
SLIDE 21

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

21

slide-22
SLIDE 22

0.0 5.0 10.0 15.0 20.0 25.0 30.0

PANSS Improvement

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

Weight Change (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

Meltzer et al., Schizophrenia Research (2012)

22

slide-23
SLIDE 23

Corporate Information

Profile:

  • Based in San Diego
  • 130 employees

Financial:

  • Cash position at

March 31, 2015(1): $298M

(1) Reflects cash, cash equivalents and investment securities

23

slide-24
SLIDE 24

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

24

slide-25
SLIDE 25

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-HT2A 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

25

slide-26
SLIDE 26

Creating the Next Generation of CNS Drugs