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1、SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.1Sarepta Therapeutics,Inc.(NASDAQ:SRPT)JPMorgan Healthcare Conference San Francisco,CaliforniaJANUARY 8,2024DOUG INGRAMPresident and CEOSAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.2FORWARD-LOOKING STATEMENTS This presentation contains forward-l
2、ooking statements.Any statements that are not statements of historical fact may be deemed to be forward-looking statements.Words such as“believe,”“anticipate,”“plan,”“expect,”“will,”“may,”“intend,”“prepare,”“look,”“potential,”“possible”and similar expressions are intended to identify forward-looking
3、 statements.These forward-looking statements include statements relating to future operations,financial performance and projections,including our expected financial results;potential solutions and market opportunities with our RNA technologies,gene therapy and gene editing;the potential benefits of
4、our technologies and scientific approaches;the potential benefits of PMO and PPMO;the potential of gene therapys applicability across disease;the potential of ELEVIDYS to benefit all patients living with Duchenne;our goal to become a life-transforming big biotech;and expected milestones and plans,in
5、cluding our understanding that,for ELEVIDYS,the FDA will evaluate a labeling expansion to the fullest extent possible based on a review of the data and will do so rapidly,our belief that our confirmatory trial for ELEVIDYS confirms its benefits and our accelerated approval should be converted to tra
6、ditional approval,announcing clinical data for SRP-5051 in 2024,and our other 2024 priorities,including moving our LGMD programs forward,including SRP-9003,driving innovation in manufacturing and other R&D goals.These forwardlooking statements involve risks and uncertainties,many of which are beyond
7、 our control and are based on our current beliefs,expectations and assumptions regarding our business.Actual results and financial condition could materially differ from those stated or implied by these forwardlooking statements as a result of such risks and uncertainties,and such risks and uncertai
8、nties could materially and adversely affect our business,results of operations and trading price.Potential known risk factors include,among others,the following:the FDA may not approve a supplement to expand the approved label for ELEVIDYS;the FDA may not convert accelerated approval into traditiona
9、l approval;we may not be able to comply with all FDA requests,including post-approval commitments and requirements,in a timely manner or at all;the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business;our data for our different programs,inc
10、luding PPMO and gene therapy-based product candidates,may not be sufficient for obtaining regulatory approval;our product candidates,including those with strategic partners,may not result in viable treatments suitable for commercialization due to a variety of reasons,including the results of future
11、research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates;success in preclinical testing and early clinical trials,especially if based on a small patient sample,does not ensure that later clinical
12、trials will be successful;the expected benefits and opportunities related to our agreements with our strategic partners may not be realized or may take longer to realize than expected due to a variety of reasons,including any inability of the parties to perform their commitments and obligations unde
13、r the agreements,challenges and uncertainties inherent in product research and development and manufacturing limitations;if the actual number of patients living with Duchenne and LGMD is smaller than estimated,our revenue and ability to achieve profitability may be adversely affected;our dependence
14、on our manufacturers to fulfill our needs for our clinical trials and commercial supply,including any failure on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet product demand,may impair the availability of products to successfully support various pr
15、ograms,including research and development and the potential commercialization of our gene therapy product candidates;we may not be able to successfully scale up manufacturing of our product candidates in sufficient quality and quantity or within sufficient timelines;we are subject to uncertainty rel
16、ated to reimbursement policies;we may not be able to execute on our business plans and goals,including meeting our expected or planned regulatory milestones and timelines,clinical development plans,and bringing our product candidates to market,for various reasons including possible limitations of ou
17、r financial and other resources,manufacturing limitations that may not be anticipated or resolved for in a timely manner,regulatory,court or agency decisions,such as decisions by the United States Patent and Trademark Office;and those risks identified under the heading“Risk Factors”in Sareptas most
18、recent Annual Report on Form 10-K and most recent Quarterly Report on Form 10Q filed with the Securities and Exchange Commission(SEC)and in its other SEC filings.For a detailed description of risks and uncertainties Sarepta faces,you are encouraged to review Sareptas filings with the SEC.We caution
19、investors not to place considerable reliance on the forwardlooking statements contained in this presentation.The forwardlooking statements in this presentation are made as of the date of this presentation only and,other than as required under applicable law,Sarepta does not undertake any obligation
20、to publicly update its forwardlooking statements.SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.3The vision set forth in 2017 was clear:Build a leading biotech enterprise grounded in a patient-first,science-driven philosophySAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.4Where we were as we en
21、tered 20171 TECHNOLOGY PLATFORMRNAPRE-PROFITABILITY2016REVENUEPIPELINEON-MARKET THERAPIESApprox.$5M61Market Cap:$1.50B12/30/16SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.5The ingredientsA passionate missionA well-articulated strategyThe ability to attract like-minded professionals A culture th
22、at challenges convention and rewards execution1234SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.6Transformational change*Based on estimated expected QTD Non-GAAP net profit1 TECHNOLOGY PLATFORMWhere we were as we entered2017RNAPRE-PROFITABILITYPIPELINEON-MARKET THERAPIES61PROFITABLE*PIPELINEON-M
23、ARKET THERAPIES$1.145B40+4RNAGENE THERAPYGENE EDITINGWhere we are today in20243 TECHNOLOGY PLATFORMSMarket Cap:$1.50B(12/30/16)Market Cap:$9.02B(12/29/23)REVENUE*2016REVENUEApprox.$5M*2023 estimated net product revenue(unaudited)SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.7DuchenneDuchenne mus
24、cular dystrophy affects approximately 1 in 3,500-5,000 newborn males worldwide1Duchenne is a rare,fatal neuromuscular genetic disease inherited in an X-linked recessive pattern2Muscle weakness becomes increasingly noticeable by 3 to 5 years of age,and most patients use a wheelchair by the time they
25、are 10 to 14 years old2During adolescence,cardiac and respiratory muscle deterioration lead to serious,life-threatening complications31.National Institutes of Health.Genetics Home Reference.Duchenne and Becker muscular dystrophy;https:/ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy
26、.Accessed Jan 2020.2.McDonald CM,Abresch RT,Duong T,et al.Long-term effects of glucocorticoids on function,quality of life,and survival in patients with Duchenne muscular dystrophy:a prospective cohort study.Lancet.2018;3(391):451-461.3.Passamano L,Taglia A,et al.Improvement of survival in Duchenne
27、Muscular Dystrophy:retrospective analysis of 835 patients.Acta Myologica.2012;31(1):121-125.HUDSON&JACOBLiving with Duchennemuscular dystrophySAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.8Robust launch of ELEVIDYS generates Q4 net product revenue of$131M*and full-year revenue of$200M*RNA-based
28、PMO revenue exceed 2023 annual guidance of$925MQ4 net product revenue total$234M*and full-year total$945M*Q4 QTD and YTD 2023 estimated net product revenue(unaudited)SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.9Robust total net product revenue for Duchenne franchise(PMOs and gene therapy)02004
29、00600800620020202120222023$Millions$381M$456M$612M$844M$5.4M$155M$301MELEVIDYSPMOs$945M*$200M*$1.145B*2023 estimated net product revenue(unaudited)115%7-year CAGR SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.10ELEVIDYS(delandistrogene moxeparvovec-rokl):Expanding the oppor
30、tunity for treatment*Accelerated Approval(AA)granted by FDA on June 22,2023,for Duchenne patients aged 4 through 5 years.Granted FDA Approval*Strong launch trajectory continuesSeeking label expansionSAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.11*ELEVIDYS:Clinical development plansSTUDY 301125
31、patientsAges 4-7,ambulatoryDouble-Blind,Placebo-ControlledNCT05096221STUDY 103 58 patientsAges 3+,ambulatory and non-ambulatoryOpen-LabelNCT04626674 Expression and safety Enrollment completed for 4 cohorts;enrollment underway for 5th cohort Genetic mutation inclusion criteria varies by cohortExons 1
32、-79Exons 18-44,46-79 Pivotal Phase 3 study Primary endpoint:NSAA Excludes mutations 1 to 17,45STUDY 303148 patientsAmbulatory and non-ambulatoryDouble-Blind,Placebo-ControlledNCT05881408 Phase 3 study 128 weeks,2-part study Primary outcome(Part 1):Change from baseline at Week 72 in the total score o
33、f PUL*Study underway*PUL=performance of upper limb Exons 18-79*ELEVIDYS is contraindicated in patients with any deletion in exons 8 and/or 9.SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.12Key secondaryOther timed secondaryResults favor treatment with ELEVIDYS on all endpointsEMBARK achieved sta
34、tistical significance on all pre-specified key secondary endpointsNSAATTR*SV95C10MWR*100MWR*Ascend 4*Favors ELEVIDYSLSMean Difference (95%CI)P-valueStandardized test statistic*(95%CI)*Timed function tests sign reversed to align favorable directions among effect endpoints*Blue lines plot standardized
35、 test statistic(+/-1.96)after dividing LSMean(95%CI)by standard errorPrimarySAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.13Functional benefits of ELEVIDYS are not limited to a particular age groupFavors ELEVIDYSLSMean Difference (95%CI)P-valueStandardized test statistic(95%CI)*Timed function te
36、sts sign reversed to align favorable directions among effect endpoints*Blue lines plot standardized test statistic(+/-1.96)after dividing LSMean(95%CI)by standard errorKey secondaryOther timed secondaryNSAATTR*SV95C10MWR*100MWR*Ascend 4*PrimarySAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.14Summ
37、aryWe believe the data from EMBARK exceeded the threshold for substantial evidence of effectiveness and the risk/benefit of ELEVIDYS remains favorableWe are pleased with the consistency,the magnitude of response and the clinical meaningfulness of the results from EMBARK and from the body of evidence
38、 supporting ELEVIDYSThe data support ELEVIDYS as a disease-modifying therapy and therefore we believe all patients with Duchenne can benefit from treatmentFollowing positive discussions with FDA leadership,they are committed to evaluating a labeling expansion to the fullest extent possible based on
39、a review of the data and will do so rapidlyNo new safety signals were observedSAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.15Path forward for ELEVIDYSELEVIDYS trials remain ongoing as Sarepta pursues label expansion20232024Submitted efficacy supplement seeking broad label(without restriction to
40、 age or ambulatory status)DEC 2023Submitted Priority Review request DEC 2023Submitted PMR seeking conversion from AA to traditional approval DEC 2023FDA response(Day 74 Letter)expected on filingEARLY MARCH 2024PDUFA/Action dateAUGUST 2024SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.162024 prior
41、ities Serve the Duchenne community with approved therapies Announce clinical data for next-generation RNA-based therapy/PPMO(SRP-5051)to treat Duchenne Execute R&D productivity,including a novel new capsid and approaches to clearing pre-existing antibodies Drive innovation in manufacturingSAREPTA TH
42、ERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.17Market leading gene therapy portfolio in 6 LGMDs1.Taghizadeh E,Rezaee M et al.J Cell Physiol.2019;234(6):7874-7884.Steady stream of gene therapy candidates covering 70%of known LGMD patients1SarcoglycanopathiesNon-SarcoglycanopathiesLGMD2D/R3SRP-9004LGMD2C/R
43、5SRP-9005LGMD2B/R2SRP-6004LGMD2L/R12SRP-9006LGMD2A/R1SRP-9010LGMD2E/R4 SRP-9003MHCK7-Sarc.AAVrh74tMCK-Sarc.AAVrh74MHCK7-Sarc.AAVrh74MHCK7DysferlinAAVrh74tMCKANO5AAVrh74tMCKCAPN3AAVrh74Dual VectorTRYNITYLiving with limb-girdlemuscular dystrophyClinicalClinicalClinicalPreclinicalPreclinicalPreclinical
44、SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.182024 will be monumentalSAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.19Transformational change,unparalleled opportunity*2023 estimated net product revenue(unaudited)1 TECHNOLOGY PLATFORMWhere we were as we entered2017RNAPRE-PROFITABILITYPIPELIN
45、EON-MARKET THERAPIES61PROFITABLE*PIPELINEON-MARKET THERAPIES$1.145B40+4RNAGENE THERAPYGENE EDITINGWhere we are today in20243 TECHNOLOGY PLATFORMSMarket Cap:$1.50B(12/30/16)Market Cap:$9.02B(12/29/23)REVENUE*2016REVENUEApprox.$5M*Based on estimated expected QTD Non-GAAP net profitSAREPTA THERAPEUTICS
46、,INC.2024.ALL RIGHTS RESERVED.201 TECHNOLOGY PLATFORMTransformational change,unparalleled opportunityWhere we were as we entered2017RNAPRE-PROFITABILITYPIPELINEON-MARKET THERAPIES61PROFITABLE*PIPELINEON-MARKET THERAPIES$1.145B40+4RNAGENE THERAPYGENE EDITINGWhere we are today in20243 TECHNOLOGY PLATF
47、ORMSMarket Cap:$1.50B(12/30/16)Market Cap:$9.02B(12/29/23)*2023 estimated net product revenue(unaudited)REVENUE*2016REVENUEApprox.$5MBy 2030:Sarepta is poised to become a big biotech,focusing on cutting-edge genetic medicine to improve the human condition*Based on estimated expected QTD Non-GAAP net
48、 profitSAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.21There is more to do.But now let us pause and bask in the glow of our achievement.Let us pause in gratitude.Let us pause in celebration.Let us watch as dawn rises and brings in the day.“BUDDY CASSIDY Living with Duchenne and an FDA advisory committee member for ELEVIDYSMay 2023From the words of someone who drives our mission and passion SAREPTA THERAPEUTICS,INC.2024.ALL RIGHTS RESERVED.22Sarepta Therapeutics,Inc.(NASDAQ:SRPT)JPMorgan Healthcare Conference San Francisco,CaliforniaJANUARY 8,2024DOUG INGRAMPresident and CEO