Abeona JPM 2025 Presentation Final 011025.pdf

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1、Corporate Presentation January 20252Note regarding forward-looking statementsThis presentation of Abeona Therapeutics Inc.(the“Company”)contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933,as amended,and Section 21E of the Securities Ex

2、change Act of 1934,as amended,and that involve risks and uncertainties.Such forward looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of1995.We have attempted to identify forward-looking statements by such terminology as“may,”“will,”“b

3、elieve,”“anticipate,”“expect,”“intend,”“hope,”and similar expressions(as well as other words or expressions referencing future events,conditions or circumstances),which constitute and are intended to identify forward-looking statements.Actual results may differ materially from those indicated by suc

4、h forward-looking statements as a result of various important factors and numerous risks and uncertainties,including,but not limited to,the timing and outcome of the FDAs review of our resubmitted Biologics License Application for pz-cel,including the FDAs assessment of our responses to the items id

5、entified in the Complete Response Letter relating to pz-cel received from the FDA;potential market opportunities and commercial launch strategies for pz-cel,if approved;the FDAs grant of a Priority Review Voucher upon a potential approval of pz-cel;continued interest in our rare disease portfolio;th

6、e timing of studies or study manuscript submissions;our ability to enroll patients in clinical trials;the outcome of future meetings with the FDA or other regulatory agencies,including those relating to our preclinical programs;our ability to achieve or obtain necessary regulatory approvals;the impa

7、ct of any changes in government,financial markets,and global economic conditions;risks relating to the decline in market price of our common stock after receipt of the Complete Response Letter;risks associated withdata analysis and reporting;and other risks disclosed in the Companys most recent Annu

8、al Report on Form 10-K and subsequent periodic reports filed with theSecurities and Exchange Commission.The Company undertakes no obligation to revise forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation,whether as a result of

9、new information,future developments,or otherwise,except as required by the federal securities laws.All trademarks,service marks,and trade names of the Company or its affiliates used herein are trademarks,service marks,orregistered trademarks of the Company.Any other product,company names,or logos me

10、ntioned herein are the trademarks and/or intellectual property of theirrespective owners.This presentation discusses pz-cel and other investigational products that have not been approved by the FDA.Nothing in this presentation should be construed as pre-approval promotion of pz-cel or any other prod

11、uct.3Abeona is poised for growthAnticipated value inflection milestones in 2025 and drivers for long-term growthDifferentiated clinical profile,transformative treatment potential in Recessive Dystrophic Epidermolysis Bullosa(RDEB)April 29,2025 PDUFA date Eligible for Priority Review Voucher US comme

12、rcial launchPreclinical POC in multiple eye diseasesProprietary AAV capsids designed to improve tropism profiles for variety of diseasesBLA submitted for MPS IIIA in December 2024(Ultragenyx)Compelling early clinical data in Rett Syndrome(Taysha)Prademagenezamikeracel(pz-cel):AAV-based gene therapy

13、platform:Out-licensed gene therapies:4Recessive dystrophic epidermolysis bullosa(RDEB)overview Rare and debilitating monogenic skin disease Caused by mutations in both copies ofCOL7A1 gene,resulting in lack of collagen VII,a protein necessary to keep skin intact Leads to fragile skin that blisters e

14、asily RDEB wounds can lead to serious life-threatening complications Patients suffer from years of large painful wounds,itch and infection High risk of squamous cell carcinoma(SCC)2,systemic infection&sepsis 76%likelihood of death by age 403 Lifelong burden on patients and caregiversRDEBtreatment go

15、als1 Bruckner,et al.Orphanet J Rare Dis.2020 Jan 3;15(1):1.;2 South,et al.J Invest Dermatol.2023 Nov;143(11):2108-2119.;3 Tang,et al.Orphanet J Rare Dis.2021 Apr 13;16(1):175.of patients body wounded130%Provide multi-year durable wound healing Provide long-term pain and itch relief Reduce risk of SC

16、C5Designed to deliver durable benefit for RDEB patientsPz-cel is the only investigational therapydesigned to deliver long-lasting healing and pain reduction via a single application,even in tough large,chronic RDEB wounds.61.Phase 3 VIITAL study:43 treated wounds vs matched control wounds in 11 pati

17、ents with RDEB in a multicenter,randomized,intrapatient-controlled study.2.Phase 1/2a study:38 treated wounds in 7 patients with RDEB in an open-label,single-arm study.Pz-cel offers uniquely differentiated value proposition as seen in clinical trialsPatient-reported improvements in itch and blisteri

18、ng2Favorable long-term safety profile1,2Demonstrated significant pain reduction in wounds1Healing in even tough large,chronic RDEB wounds1,2Multiple years of wound healing after single application27Healed wounds at Week 24 were confirmed 2 weeks later to be included.Complete wound healing defined as

19、 re-epithelialization with no drainage or erosion and presence of only minor crusting.1.Pain assessed using Wong-Baker FACES rating scale(0-10).Pain reduction calculated as difference between baseline and postbaseline pain scores.Abbreviations:N,number of total wounds with non-missing healing improv

20、ement category;N1,total number of wounds with non-missing pain reduction score.Significant wound healing and reduction in pain1reported with pz-cel81.4%65.1%16.3%16.3%7.0%0.0%50%Healing75%HealingComplete Healing*pz-celControl3572837Co-primaryendpointN=434343434343Wound healingfrom baseline at Week 2

21、4-3.07-0.90-4-3-2-10Mean pain reductionCo-primary endpointP0.0001P0.0001Pain reduction from baseline at Week 24P20 cm2,open for 5 years(median)Met both co-primary endpoints for wound healing and pain reduction at six months Pz-cel was well-tolerated with no serious treatment-related adverse events o

22、bserved,consistent with past clinical experience8Example RDEB wounds before&after pz-cel treatment Source:Phase 3 VIITAL study patient;individual results varyWound healing scoring was Investigator-assessed per predefined criteria.Wounds scored as at least 75%healing at week 24Upper left thighUpper l

23、eft thighBaseline:Week 24:9Example RDEB wounds before&after pz-cel treatmentSource:Phase 3 VIITAL study patient;individual results varyWound healing scoring was Investigator-assessed per predefined criteria.Complete wound healing was defined as re-epithelialization with no drainage or erosion and pr

24、esence of only minor crusting.E9B4B4E9Upper trunkUpper trunkB4 scored as at least 75%healing,E9 scored as complete wound healing at week 24Baseline:Week 24:10Example RDEB wounds before&after pz-cel treatmentSource:Phase 3 VIITAL study patient;individual results varyWound healing scoring was Investig

25、ator-assessed per predefined criteria.B3 scored as at least 75%healing at week 24Upper backUpper backB3B3Baseline:Week 24:11Example RDEB wounds before&after pz-cel treatmentSource:Phase 3 VIITAL study patient;individual results varyWound healing scoring was Investigator-assessed per predefined crite

26、ria.Complete wound healing was defined as re-epithelialization with no drainage or erosion and presence of only minor crusting.Wounds scored as at least 75%healing and complete wound healing at week 24Baseline:Week 24:Left flankLeft flank12Multi-year wound healing and pain reduction after single app

27、lication of pz-cel0%10%20%30%40%50%60%70%80%90%100%6 mo1Y2Y3Y4Y5Y6Y7Y8Y50%75%#of wounds=Source:So et al.Orphanet Journal of Rare Diseases(2022)17:377 38383832212710155%treated wounds with50%or 75%healing%treated wounds with pain(n/N)53%0%16%5%8%0%0%0%0%03 mo6 mo1Y2Y3Y4Y5Y6YTime after treatment(20/38

28、)(0/38)(6/38)(2/38)(3/38)(0/26)(0/15)(0/15)(0/5)BaselinePhase 1/2a study key patient characteristics:Baseline wounds remained open for average of 11 years Average treated body surface area 100 cm213Multi-year wound healing after one-time pz-cel applicationSource:Phase 1/2a study patient(So et al.Orp

29、hanet Journal of Rare Diseases(2022)17:377);individual results varyWound healing scoring was Investigator-assessed per predefined criteria.BaselineDay 7Year 2Year 5Upper backUpper backUpper backUpper back14Collagen VII protein expression observed for 24 months at a pz-cel treated siteSource:Phase 1/

30、2a study patientSiprashvili 2016,Eichstadt 2019Year 2Year 13 monthsAnchoring fibril expression 2 years after pz-cel treatmentCollagen VII expression 2 years after pz-cel treatmentNo anchoring fibrilsNo collagen VII expressionYear 2Year 13 monthsBaselineBaseline15clPz-cel cumulative clinical trial ex

31、perienceFavorable long-term safety profileDurable wound healing and reduction in pain&itchStudy#pz-cel patients#wounds treatedPhase 1/2a742Phase 31157Phase 3b321Total21120 Treated multiple,different anatomical locations,including anterior or lateral,posterior,extremities and trunk2 Treated large wou

32、nd areas (up to 240 cm2)1,2No serious adverse events(SAEs)related to pz-cel(most common AE was pain related to surgery)1,2No squamous cell carcinoma(SCC)at treated sites1,2with longest follow-up time of 11 years(occurrences in non-treated sites)2No positive replication competent retrovirus(RCR)2 Wou

33、nd healing and reduction of pain&itchseen with pz-cel treatment,even in tough large,chronic RDEB wounds Sustained wound healing with a mean of 5.9 years of follow-up from Phase 1/2a study11.Phase 3 VIITAL study:43 treated wounds vs matched control wounds in 11 patients with RDEB in a multicenter,ran

34、domized,intrapatient-controlled study.2.Phase 1/2a study:38 treated wounds in 7 patients with RDEB in an open-label,single-arm study.16Robust US commercial opportunity1,500 pz-cel treatment opportunities based on current prevalent pool1.Claims Analysis 2024,2.Internal US.Payor Research,3.3Q2024 Krys

35、tal Financial Report1,300 US DEB patients1;750 RDEB pz-cel eligible patientsAnticipate 2 treatment cycles per patient,on average,to cover impacted body areasPayer willingness to cover RDEB lives2,3Pricing expected per each treatment cycle,and in line with value of a one-time gene therapy offering mu

36、lti-year clinical benefit$500Mest.peak annual US revenue85-90%est.gross margin after initial ramp17Partnering with high-volume EB Centers of Excellence(COEs)to drive successful pz-cel launch Ongoing commercial readiness activities:OperationsEarly engagement Initiated onboarding process with multiple

37、 EB COEs to become pz-cel Qualified Treatment Centers(QTCs)Developing comprehensive patient assistance and provider support programs Standing up specialty distributor and pharmacy distributionchannels Increasing commercial manufacturing capacity Driving medical awareness through scientific exchange

38、and peer-to-peer education Engaging commercial payers*representing 80%of RDEB lives and Medicaid programsScaled launch Commercialization team with robust cell and gene therapy launch experience Launch at geographically dispersed QTCs to maximize patient treatment access Site readiness to start treat

39、ing patients 2-3 months post-approval Drive demand through high pz-cel awareness across COEs to enable referralsLaunch year goals:Onboard that see 30%of pz-cel eligible patientsTreatper center per month5 EB COEs 1 to 2 patientsEnsure broad and timely market access*Pre-approval information exchange18

40、“I think its much more important to focus on after a wound closes,does it remain closed for the next 2 years with incidental trauma or activities of daily life?I think thatll be the most important measurement.”US KOL“I wouldnt have any concerns referringthey would have to be admitted but being in th

41、e hospital isnt new to them.”“I am hopeful and excited I have questions on the before and after process,but when can I get it?”“These are clinically meaningful endpoints,especially the 50%wound healing.”Physicians,patients,and payers confirm unmet need in RDEB and enthusiasm for pz-cel“These before&

42、after photos of pz-cel patients are extremely compelling.”Regional PayerCOE PhysicianCommercial Payer31-year-old Patient19Building strong payer support across reimbursement pathways for pz-cel1.Medicare Severity Diagnosis-Related GroupCommercial&Medicaid Pre-approval engagement ongoing Targeting key

43、 payers representing 80%of RDEB covered lives Positive early engagement builds confidence to secure broad coverage and reimbursementAnticipated Access Inpatient hospital payment methodologies are established Payers appreciate high unmet need and pz-cel clinical value Payers expect similar coverage t

44、o existing gene therapiesMedicare Product-Specific Coding:ICD-10-PCS pz-cel procedure code granted for genetically engineered autologous cell therapy Favorable DRG Payment:MS-DRG1reimbursement for pz-cel(Pre-MDC DRG 018)mapped to highest inpatient hospital reimbursement levels for cell and gene ther

45、apies Additional Potential Payments:New Technology Add-on Payment(NTAP)and Outlier payment methodologies to be pursued for additional Medicare reimbursementRDEB Payer Coverage MixCommercial55-60%Medicaid30-35%Medicare10%20Ramping manufacturing capacity to support 200+annual pz-cel treatments by 2H 2

46、027At launch Starting 1H 2026By 2H 20271.Leased adjacent building space in October 2024.50 pz-cel treatments/annum120 pz-cel treatments/annum200+pz-cel treatments/annumRamping up operating capacity within existing GMP facilityCreation of additional manufacturing space121Patient intakePz-cel patient

47、journeyBiopsy sample procurementPz-cel manufacturingPz-cel surgical procedureMonitoring/discharge5432122Patient hub providing support throughout the pz-cel treatment journeyReimbursement SupportPatient NavigatorTransportation&Lodging SupportCopay AssistanceCaregiver and Patient Education&SupportPipe

48、line of differentiated cell and gene therapiesDISCOVERYPRECLINICALPHASE 1/2 PHASE 3 Pz-celRDEBABO-503X-linked retinoschisis(XLRS)ABO-504Stargardt diseaseABO-505Autosomal dominant optic atrophy(ADOA)UX111*Sanfilippo syndrome type A(MPS IIIA)TSHA-102*Rett syndromeTSHA-118*Infantile batten disease(CLN1

49、 disease)PARTNEREDPartnered with*Ultragenyx Pharmaceutical Inc,*TayshaGene Therapies,Inc.BLA submitted,Apr 29,2025 PDUFABLA submitted*24Novel and differentiated approaches for genetic ophthalmic conditions with high unmet medical needProprietary AAV capsids Novel therapeutic approachesDesigned for i

50、mproved tissue/cell tropismand reduced immune responses for distinct routes of administration including ocular,systemic and direct CNSAAV204 identified for exceptional ability to penetrate retinal cell layers after injection directly into vitreousLicensing opportunities for Abeona proprietary capsid

51、s in various ocular conditionsDeveloping therapies for higher prevalence ophthalmic disorders including Stargardt disease,X-linked retinoschisis(XLRS),and autosomal dominant optic atrophy(ADOA)XLRS and ADOA programs leverage AAV204 capsid combined with para-retinal injection,a potentially safer rout

52、e compared to subretinal administrationStargardt diseaseasset producesfull length ABCA4 proteinby using proprietary AAV-based Cre-Lox system to overcome packaging capacity limits of traditional AAV genome 25ABO-503 for X-linked retinoschisisKey findings ABO-503 treatment of very young mutant mice si

53、gnificantlyprevents formation of cavities and preserves photoreceptor function compared to untreated mutant mice Robust RS1 expression observed in photoreceptor cells near injection site and in adjacent inner retina in mutant mice 3 months after treatment with ABO-503 RS1 expression associated with

54、improved cone photoreceptor density and increased thickness of photoreceptor outer nuclear layer Full-field flicker electroretinogram(ERG)analysis showed significant improvement in cone photoreceptor function Proprietary AAV204 capsid allows for less invasive para-retinal injection route to achieve

55、high macular transduction rateFirst-in-human opportunity in 1H 2026Imaging of mutant mice 2 months after treatmentUntreatedABO-503MaculaPara-retinal injection of AAV204 in non-human primates26Current capital resources provide runway through potential launch into 2026,with potential future income str

56、eams to extend further*As of Sept 30,2024$110M in cash resources*Sale of pz-cel Priority Review Voucher(PRV),if awarded by FDA Revenues from commercial sales of pz-cel,if approved by FDACapital resources into 2026Potential future income streams272025 milestones and catalystsPZ-CELPotential FDA appro

57、val in RDEB(April 29,2025 PDUFA)Potential PRV grantActivate pz-cel treatment sitesBuild referral networks to pz-cel treatment centersPursue pz-cel ex-US opportunity post-FDA approvalPIPELINERS1 preclinical data presentation at scientific congressComplete RS1 IND-enabling workContinue to assess external assets to expand pipeline