Acadia_Pharmaceuticals_JPM2025_FINAL.pdf

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1、43rd Annual JP Morgan Healthcare ConferenceCatherine Owen AdamsChief Executive OfficerJanuary 14,2025This 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 statement

2、s 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.Fo

3、rward-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 candi

4、dates;(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

5、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,u

6、ncertainties,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

7、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 nece

8、ssary 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 genera

9、te 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

10、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,

11、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 September 30,2024.The forward-looking statements contain

12、ed herein are made as of the date hereof,and we undertake no obligation to update them after this date,except as required by law.This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our indu

13、stry.This data involves a number of assumptions and limitations,and you are cautioned not to give undue weight to such estimates.Projections,assumptions and estimates of the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk.The tra

14、demarks included herein are the property of the owners thereof and are used for reference purposes only.Forward-Looking Statements22025 expected to be first full year of$1 billion+revenueFiling of DAYBUE MAA with EMA with expected approval in Q1 2026Anticipated timelines for last patient in and top

15、line results for:ACP-101 Phase 3 study in Prader-Willi SyndromeACP-204 Phase 2 study in Alzheimers Disease PsychosisPlans to initiate a Phase 2 of ACP-204 in Lewy Body Dementia with Psychosis Plans to host first R&D day in company history in mid-2025Announced Today3MAA:marketing authorization applic

16、ationEMA:European Medicines Agency2025-2026 Anticipated MilestonesCorporate2026Q1Q2Q3Q42025Filed MAA with EMA Managed Access Programs available in select EU countriesPhase 3 LAST PATIENT INPhase 2 in LBDP FIRST PATIENT INR&D Day(MID-2025)Annual Sales$1BPotential ApprovalQ1Q2Q3Q4ExpectedApproval in E

17、MATwo late-stage pipeline programs anticipated to have topline results in 2026ACP-204(ADP/LBDP)ACP-101(PWS)4Phase 2 in ADP LAST PATIENT INPhase 2 in ADP TOPLINE RESULTSPhase 3 TOPLINE RESULTSNeuro PsychBuilding Long Term Growth in CNS&Rare DiseaseNeuro RareADPACP-204(new 5-HT2A)Essential TremorACP-7

18、11(selective GABAA-3 modulator)TRD/MDD/OtherACP-211(NMDA receptor antagonist)PWS ACP-101(Intranasal carbetocin)Rett/Fragile XACP-2591(cGP analogue)5LBDPACP-204(new 5-HT2A)PRECISION MEDICINE|DATA INNOVATION|GLOBALIZATION|PATIENT EMPOWERMENTPOWERED BYCore FranchiseCore PipelineExpansion AreasExpansion

19、 to other Rare Disease areas under evaluationEndocrineMetabolicCardiovascularImmunologyNephrologyNeuropsychiatric Franchise6Disease Awareness Campaign7Overview of Parkinsons Related Hallucinations and Delusions1M patients with Parkinsons Disease(PD)in U.S.Around 50%may develop hallucinations and/or

20、delusions at some point during course of their disease1Low awarenessMarket research indicated that at the start of 2024 less than 10%of caregivers and patients were aware that hallucinations and delusions are associated with PD2130,000 PD patientsare treated with an atypical antipsychotic annually3S

21、eeing,hearing or experiencing things that others dontBelieving things that are not trueSYMPTOMS1 Elin B Forsaa,et al.A 12-year population-based study of psychosis in Parkinson disease Arch.Neurol.2010;Aug;67(8):996-10012 Source:Acadia confidential market research3 Acadia estimate as of June 2024 bas

22、ed on claims data 8NUPLAZID for the Treatment of Parkinsons Related Hallucinations and DelusionsNUPLAZID is the first and only FDA-approved drug for the treatment of hallucinations and delusions associated with Parkinsons disease psychosis.9CLINICALLY PROVENOver 82,000 patients treatedMost studied R

23、x in PD-Psychosis,with no negative impact on motor function or cognition1GROWTH ACCELERATING10%YTD net sales growth Q1-Q3 in 2024DTC campaign driving increased referrals in Q4 2024OPPORTUNITY REMAINS20%share of 130,000 patients on atypical antipsychoticsComposition of matter to Oct.2030;formulation

24、to Feb.2038ANTICIPATED$325M IN INCREASING ANNUAL CASH FLOW FUELING CORPORATE GROWTH1 J Wu et al,Global research trends and hotspots in Parkinsons disease psychosis:a 25-year bibliometric and visual analysis,Frontiers in Aging Neuroscience,November 2024,DOI 10.3389/fnagi.2024.1480234NUPLAZID Commerci

25、al Strategy&OutlookPull through interested consumers from branded DTC campaignActivate ConsumersFurther leverage real world evidence to drive prescriber decisions Drive Market ShareLeverage AI and data to call on the right prescribers at the right timeMaximize field force efficiencyNUPLAZID 2025 OUT

26、LOOKAccelerating sales growth Increasing market shareAdditional data publicationsAdditional real world evidence out to 5 years10DAYBUE119,000-12,000 prevalent population in EU3Overview of Rett SyndromeDebilitating Symptoms of Rett Syndrome15,500-5,800 diagnosed patients in U.S.2with a prevalent popu

27、lation of 6,000-9,000Fine and gross motor impairmentLoss of verbal and nonverbal communicationHand stereotypies Loss of independence and require 24/7 supportGI symptoms including severe constipationSeizuresTypically caused by mutations in the MECP2 gene disrupting the function of MECP2 protein cruci

28、al for brain development and function121 Acadia market research,Neul JL et al,Annal Neurol.2010;68;944-50 and https:/www.rettsyndrome.org/about-rett-syndrome/what-is-rett-syndrome/.2 Based on Acadia Pharmaceuticals analysis of claims data as of 2024 claims data.3 Based on Acadia internal estimates.D

29、AYBUE is the first and only FDA-approved drug for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.GROWING CLINCIAL EXPERIENCEOver 1,600 patients treated to date66%of active patients have been on treatment 10 monthsFAVORABLE ACCESS ENVIRONMENT90%of DAYBUE fami

30、lies pay 200 patients received ACP-204 in Phase 1 trialslower hERG vs.pimavanserin;No signs of QT prolongationWide dose range established supporting potential for 2x pimavanserin 34 mg equivalentSteady state PK achieved in 5 days vs pimavanserin 12 daysRESULTS TO DATEPlanned publications throughout

31、2025ACP-204 FEATURES A COMBINATION OF STRUCTURAL CHANGES VS PIMAVANSERIN,AND REDUCED OFF-TARGET EFFECTS ALONG WITH EQUAL OR INCREASED POTENCYMitigate or eliminate QT prolongationEnable doses higher than pimavanserin 34 mg equivalent Improve time to onset of action24ACP-204 TARGET PRODUCT PROFILERADI

32、ANT Phase 2/Phase 3 Pimavanserin experience supports P2/P3 programGlobal,placebo-controlled,double-blind Phase 2 enrolling25Q1:2026:Phase 2 in ADP last patient in expectedMid-2026:Phase 2 in ADP topline results expectedQ1Q2Q3Q420252026SEAMLESS ENROLLMENTPHASE 2TWO PHASE 3 STUDIESN=318,double-blind,r

33、andomized 1:1:1of similar size and designACP-204(60mg)Placebo6-week treatment periodACP-204(30mg)ACP-204(60mg)Placebo6-week treatment periodACP-204(30mg)ACP-204(60mg)Placebo6-week treatment periodACP-204(30mg)Primary Endpoint for Phase 2:SAPS-H+D total score change from baseline to Week 6Q1Q2Q3Q4Pha

34、se 2 in ADP LAST PATIENT INPhase 2 in ADP TOPLINE RESULTSPhase 2 in ADP CONTINUED ENROLLMENT1 Simuni T,Chahine LM,Poston K,et al.A biological definition of neuronal-synuclein disease:towards an integrated staging system for research.Lancet Neurol.2024 Feb;23(2):178-190.doi:10.1016/S1474-4422(23)0040

35、5-2.PMID:38267190.2 Cummings et al 2018.3 Based on IQVIA data and Acadia internal estimates.LBD is a progressive brain disorder that affects thinking,movement,mood,and behaviorLewy Body Dementia(LBD)Associated with abnormal deposits of alpha-synuclein in the brain1No therapies approved for LBD with

36、psychosisand some traditional antipsychotics that are commonly used in other diseases can be harmful1 million people in the U.S may be living with LBD;50%-75%of people with LBD experience psychosis(LBDP)2Approximately 200,000 patients living with LBD are being treated with antipsychotics 326Plan to

37、Initiate Phase 2 Study in LBDP in Q3 2025Phase 2 program will enroll both PDD and DLB patients with psychosisRelapse data from pimavanserin withdrawal study suggest potential utility of targeting 5HT2A%of patients experiencing relapse:5.3%(1 of 19)patients receiving pimavanserin55.0%(11 of 20)receiv

38、ing placebo Alpha-synuclein and other biomarkers will be evaluated to characterize patient population27Parkinsons Disease Dementia(PDD)Lewy Body Dementiawith Psychosis(LBDP)Dementia with Lewy Bodies(DLB)ACP-711 for Essential Tremor28Essential Tremor1.https:/www.mayoclinic.org/2.Gerbasi ME,Nambiar S,

39、Reed S,et al.Front Neurol.2022.doi:10.3389/fneur.2022.891446.3.Shanker V.BMJ.2019.doi:10.1136/bmj.l4485.4.Louis ED,Ottman R.Tremor Other Hyperkinet Mov(N Y).2014.doi:10.7916/D8TT4P4B.Shaking or trembling movements of the hands(and beyond)1Associated with physical and cognitive impairments,social avo

40、idance,and other challenges that significantly impact patients lives2-3 7 million people affected in the United States 1 million currently receiving Rx4Can be debilitating,with significant impact on quality of life2Innovation neededonly approved pharmaceutical treatment is more than 50 years oldAlig

41、ns with Acadias customerfacing footprintand focus on movement disorder specialists/centers of excellence29ACP-711 is a Natural Fit with Acadias Commitment to Innovation in CNSGABAA 3 subunit is expressed in key regions involved in essential tremor(cerebellar dentate,thalamus,and cortex)1-4ACP-711 is

42、 a selective GABAA 3 positive allosteric modulator(PAM)with potential to treat ET2025 activities planned to support Acadias desired Phase 2 trialInclusion of elderly cohort in ongoing Phase 1 to inform future development,given importance of treating the whole patient populationPhase 2 trial planned

43、for 202630Highly selective activity for alpha-3 subunit1.Waldvogel HJ,Munkle M,van Roon-Mom W,et al.J Chem Neuroanat.2017.doi:10.1016/j.jchemneu.2017.04.006.2.Sperk G,Kirchmair E,Bakker J,et al.J Comp Neurol.2020.doi:10.1002/cne.24910.3.Stojanovic T,Capo I,Aronica E,et al.J Comp Neurol.2016.doi:10.1

44、002/cne.23923.4.Schaefer SM,Vives Rodriguez A,Louis ED.Expert Rev Neurother.2018.doi:10.1080/14737175.2018.1413353.%modulation relative to reference%modulation relative to referenceLog SAN711(M)Log SAN711(M)High activityLow activityNo activityIn Closing31LPI:Last Patient InAcadia Believes More is Po

45、ssibleAdvancing breakthroughs in neuroscience and rare diseases to elevate life.Growing pipeline in CNS and rare disease with late-stage readouts expected in 2026Hosting first-ever R&D Day in mid-2025R&D322025 expected to be first full year of$1 billion+U.S.total revenuesStrong growth for both brand

46、s;initial revenues from Managed Access Programs in select EU countriesTWO GROWING BRANDSCOMMERCIALFINANCIALStrong balance sheet with positive,growing cash flow to reinvest for growthLATE-STAGE PIPELINEACP-101(P3-PWS)LPI expected Q4:2025ACP-204(P2-ADP)LPI expected Q1:2026PRECISION MEDICINE|DATA INNOVATION|GLOBALIZATION|PATIENT EMPOWERMENTPOWERED BY43rd Annual JP Morgan Healthcare ConferenceCatherine Owen AdamsChief Executive OfficerJanuary 14,2025