ACADIA Pharmaceuticals : Corporate Presentation

Published: 2024-11-11 01:55:03 ET ACAD

Acadia

Corporate

Presentation

November 2024

Forward-Looking Statements

This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "outlook," "potential" and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward- looking statements contained in this presentation, include, but are not limited to, statements about: (i) our business strategy, objectives and opportunities; (ii) plans for, including timing, development and progress of commercialization or regulatory timelines for, NUPLAZID, DAYBUE and our product candidates; (iii) benefits to be derived from and efficacy of our products, including the potential advantages of NUPLAZID and DAYBUE and expansion opportunities for NUPLAZID and DAYBUE in other indications, and for DAYBUE in jurisdictions outside the U.S. and Canada; (iv) estimates regarding the prevalence of the diseases targeted by our products and product candidates; (v) potential markets for any of our commercial products; and (vi) our estimates regarding our future financial performance, cash position, profitability or capital requirements. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to: our dependency on the continued successful commercialization of NUPLAZID and DAYBUE and our ability maintain or increase sales of NUPLAZID or DAYBUE; the costs of our commercialization plans and development programs, and the financial impact or revenues from any commercialization we undertake; our ability to obtain necessary regulatory approvals for our product candidates and, if and when approved, market acceptance of our products; our dependence on third-party collaborators, clinical research organizations, manufacturers, suppliers and distributors; the impact of competitive products and therapies; our ability to generate or obtain the necessary capital to fund our operations; our ability to grow, equip and train our specialized sales forces; our ability to manage the growth and complexity of our organization; our ability to maintain, protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties 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, 2023 as well as our subsequent filings with the Securities and Exchange Commission from time to time, including our quarterly report on Form 10-Q for the period ended November 7, 2024. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.

Acadia Overview

Commercial

Two growing commercial franchises in DAYBUE and

NUPLAZID

Reported $250.4 million

in total revenue in Q3

Quarterly revenues now annualizing to more than $1 billion

R&D

Two late-stage assets with strong early-stage pipeline

Ongoing P3 trial of ACP-101 in Prader-Willi syndrome

Ongoing P2 / P3 program of ACP-204 in Alzheimer's disease psychosis

Multiple early-stage programs focused on rare and neuropsychiatric diseases

Financial

18% YoY revenue growth in 3Q24

Cash balance of $565.3M as of September 30, 2024

DAYBUE (trofinetide) is only approved in the U.S. by the FDA and in Canada by Health Canada for the treatment of Rett syndrome in adults and pediatric patients two years of age and older.

NUPLAZID (pimavanserin) is only approved in the U.S. by the FDA for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis.

Rett Syndrome

~5,000~6,000 - 9,000

diagnosed patients

prevalent population

in the US

Debilitating Symptoms of Rett Syndrome1

Fine and gross

Loss of independence

motor impairment

and require 24/7 support

Loss of verbal and

G.I. symptoms, including

nonverbal communication

severe constipation

Hand stereotypies

Seizures

1Acadia market research, Neul JL et al, Annal Neurol. 2010;68;944-50 and and https://www.rettsyndrome.org/about-rett-syndrome/what-is-rett-syndrome/.

DAYBUE for the Treatment of Rett Syndrome

First and only FDA-approved drug for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. Received priority review, orphan drug and fast track drug designations.

1Acadia owes Neuren Pharmaceuticals 1/3 of the value at the time it is used or sold by Acadia

A twice-daily liquid medication that can be orally- administered or via gastrostomy tube

Approval based on improvement in point scales measuring severity of disease including RSBQ (caregiver assessed) and CGI-I (clinician rated)

Most common adverse reactions include diarrhea and vomiting

Method-of-use patent to 2036

Received a Priority Review Voucher on approval1

Acadia owns worldwide rights

DAYBUE Update

Number of Patients Receiving Paid Shipments in the Quarter

Persistency Based on Shipments

Key 3Q Trends

# of Paid Shipments

60%

of all DAYBUE

patients have now been on treatment 10 months or more

Persistency rates of

50%

or higher after

12 months

Approximately

~800

unique

prescribers

DAYBUE Initiatives to Drive Penetration

Focus on Real-World Experience

Translating clinical endpoints to tangible real-world benefits, including stories from HCPs and Caregivers of patients' improved:

Quality of Life

Alertness

Mood

Engagement

Purposeful

Use of Hands

Verbal & Non-Verbal

Communication

Key Drivers

Key Market Data

NUPLAZID for the Treatment of Hallucinations and Delusions Associated with Parkinson's Disease

• ~50% of people with PD may develop

hallucinations and/or delusions at some point

during the course of their disease1

NUPLAZID is the first and only FDA-

• ~130,000 patients each year are PD patients

approved drug for the treatment of

hallucinations and delusions

treated with an Atypical Antipsychotic2

associated with Parkinson's disease

psychosis.

Debilitating Symptoms

• Composition of matter patent to 2030

Seeing things that others don't

Paranoia

• Formulation patents to 2038

Hearing sounds, music or voices

False beliefs

1 Elin B Forsaa , et al. A 12-year population-based study of psychosis in Parkinson disease Arch. Neurol.. 2010; Aug;67(8):996-1001

2 Acadia estimate as of June 2024 based on claims data

NUPLAZID 3Q Drivers

$159.2M

in Net Product Sales

Up 10% year-over-year;

highest ever quarterly sales

Real-World Evidence1-3 Findings:

Label Change Clarifying:

Mosholder AD, Ma Y, Akhtar S, et al. Mortality among Parkinson's disease patients treated with pimavanserin or atypical antipsychotics: an observational study in Medicare beneficiaries. Am J Psychiatry. 2022;179(8):553-561.

Layton JB, Forns J, McQuay LJ, et al. Mortality in patients with Parkinson's disease-related psychosis treated with pimavanserin compared with other atypical antipsychotics: a cohort study. Drug Safety. Published online December 14, 2022.

doi:10.1007/s40264-022-01260-6.

Published online December 14, 2022. doi:10.1007/s40264-022-01260-6.

Deep CNS Pipeline

Program

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Marketed

NUPLAZID®

Parkinson's Disease

(pimavanserin)

Psychosis

DAYBUE

Rett Syndrome

(trofinetide)

ACP-1011

Hyperphagia in

Prader-Willi Syndrome

ACP-2041

Alzheimer's Disease

Psychosis

ACP-25911

Rett Syndrome; Fragile

X Syndrome

ASO Programs1

SYNGAP1; Rett

syndrome; Undisclosed

Multiple Undisclosed

Neuropsychiatric and

Programs

Rare Disorders

Acadia has worldwide rights for all assets

1 The safety and efficacy of these investigational agents have not been established. There is no guarantee these investigational agents will be filed with or approved by any regulatory agency.

Disclaimer

ACADIA Pharmaceuticals Inc. published this content on November 11, 2024, and is solely responsible for the information contained herein. Distributed by Public, unedited and unaltered, on November 11, 2024 at 06:54:02.743.