Plus Therapeutics, Inc. · NASDAQ:PSTV
Plus Therapeutics — Cytori Therapeutics — Biotech Showcase 2016
35 slides from Plus Therapeutics's conference presentation, Jan 12, 2016
Slide 1: Cytori Therapeutics: Enhancing lives through novel cell therapies
Cytori Therapeutics NASDAQ: CYTX Enhancing lives through novel cell therapies Biotech Showcase I January 2016
Slide 2: Forward Looking Statements and Disclaimers
Forward Looking Statements and Disclaimers 33% 15% 11% 22% 19% OTHER USA EU JAPAN ASIA-PACIFIC This presentation contains certain ‘forward-looking statements’ about Cytori Therapeutics, Inc. All statements, other than statements of historical fact, that address activities, events or developments tha
Slide 3: Summary
Summary Unique cell therapy platform- clear commercial model, late-stage lead indication and growing development pipeline Completed corporate restructuring- better positioned to achieve key milestones 2016Substantial clinical data- indicates Cytori Cell Therapy provides symptomatic benefit, improved
Slide 4: Cytori Cell Therapy: Late-Stage Clinical Pipeline
Enrolling2 Cytori Cell Therapy: Late-Stage Clinical Pipeline ECCI-50 Enrollment Complete ECCO-50 Preclinical3 DCCT-10 Enrolling ECCS-50 Enrolling1 ECCS-50 Pre-Clinical Phase I/II Phase III Market (Estimate) >$1B >$500M >$3B >$75M >$50M 1 Cytori-funded, Investigator-initiated trial2 Japan Govt Sponso
Slide 5: Cytori 3-Step Bedside Process
Cytori 3-Step Bedside Process TIME 30 Min 120 Min 5 - 30 Min PROCESS HARVESTSmall Volume Liposuction(100-360 mL) PROCESSCelution® SystemTissue Processing, Cell Isolation & Dose Preparation DELIVERCytori® Cell TherapyTM Delivery Bedside Manufactureproprietary consumables,software, and reagents Ad
Slide 6: Cytori Cell Therapy: Cellular Composition
Cytori Cell Therapy: Cellular Composition Cells sourced from autologous adipose tissueTherapeutic manufactured in bedside GMP processProcess yields a diverse, unique cell populationAll consumables are clinical grade Stem & Stromal Cells Leukocytes Tissue Macrophages Endothelial VascularSMCs A clinic
Slide 7: Cytori Cell Therapy: Mechanism of Action
Cytori Cell Therapy: Mechanism of Action Cytori Cell Therapy beneficially modulates multiple key pathologic processes leading to anticipated sustained reduction in pain and disability and improved quality of life Promotes angiogenesisNormalization of vessel architectureImproved vasomotor function1-5
Slide 8: Lead Indication: Scleroderma
Lead Indication: Scleroderma
Slide 9: Scleroderma Pathophysiology
Scleroderma Pathophysiology Scleroderma or Systemic SclerosisRare autoimmune conditionAffects Women: Men, 4:1US Prevalence: 50,000 patients>90% of patients have hand disabilityFibrosis, pain, and edema result in diminished mobility and hand function even with standard medical careSevere vasomotor sy
Slide 10: Scleroderma: Market Overview
Scleroderma: Market Overview Current Standard of CareNo therapies approved for treatment of hand dysfunction in scleroderma patientsExisting 1st and 2nd line treatments for treatment of Raynaud’s Phenomenon or other aspects of scleroderma are often inadequate and poorly toleratedExisting 3rd line tr
Slide 11: Scleroderma: Treatment Approach
Scleroderma: Treatment Approach AmbulatoryProcedure roomLocal or mild conscious sedationSingle administration ECCS-500.5cc injection to each NVBNo bandage \ 11 1/10/16
Slide 12: Pilot/Phase I SCLERADEC I Trial
Pilot/Phase I SCLERADEC I Trial SCLERADEC I Study size 12 Randomization Open label Administration Single administration (~4m cells/finger) Sites Single site (IIS) - Marseille, France Endpoints Cochin Hand Function ScaleRaynaud’s Condition ScoreScleroderma Health Assessment QuestionnairePain Modified
Slide 13: ECCS-50 Treatment led to improvement in hand function, Raynaud's phenomenon, and pain
ECCS-50 Treatment led to improvement in hand function, Raynaud’s phenomenon, and pain SCLERADEC I Improved PROs over 24 months Granel et al. Ann Rheum Dis 2014; Guillaume-Jugnot et al. Rheumatology 2015mean ± std err Key Observation:Concordant reduction (~50% ) in four key symptomatic patient report
Slide 14: SCLERADEC I - Other Endpoints
SCLERADEC I- Other Endpoints Granel et al. Ann Rheum Dis 2014; Guillaume-Jugnot et al. Rheumatology 2015All data presented as mean ± std err Sustained improvement in hand strength & skin stiffness 130% improvement in pinch strength30% improvement in grip strength p=0.014 23% improvement in mRSS of t
Slide 15: Reduction in digital ulcers, improved microvascular architecture
Reduction in digital ulcers, improved microvascular architecture All data presented as mean ± std errGranel et al. Ann Rheum Dis 2014; Guillaume-Jugnot et al. Rheumatology 2015Image at top reproduced with permission of the nonprofit International Scleroderma Network at sclero.org SCLERADEC I- Other
Slide 16: Scleroderma: Ongoing Clinical Trials
Scleroderma: Ongoing Clinical Trials STAR (Phase III) SCLERADEC II (Phase III) Study size 80 40 Randomization 1:1, active: placebo 1:1 (dose from Pilot, placebo) Crossover Placebo, crossover at 48 weeks Placebo, crossover at 24 weeks (cryo) Sites Up to 20 in USA 6 France Primary Endpoint Cochin Hand
Slide 17: Scleroderma: Anticipated Development Timeline
Scleroderma: Anticipated Development Timeline 2015 2016 2017 2018 2019 3 4 1 2 3 4 1 2 3 4 1 2 3 4 1 2 3 4 STAR Enrollment, 12 Month Follow-Up & Data Analysis FDA Original PMA Submission & Panel Approval Reimbursement Scleradec II Enrollment,6 Month Follow-Up & Data Analysis Reimbursement EMA MAA Su
Slide 18: Managed Access Program Overview
Managed Access Program Overview SituationScleroderma rare disease, orphan designationSCLERADEC-I results show positive ECCS-50 risk-benefit ratioTwo pivotal trials enrollingClear unmet patient needs and verified broad EMEA demand for ECCS-50OpportunityProvide ECCS-50 access to patients unable to par
Slide 19: Managed Access Program Objectives
Managed Access Program Objectives ❶ Provide ethical and compliant access to Cytori Cell TherapyTM, ECCS-50, for hand scleroderma patients prior to EMA marketing authorization ❷ Increase awareness of and facilitate a positive experience with Cytori Cell TherapyTM among healthcare providers in advance
Slide 20: Scleroderma Anticipated Managed Access Program Timeline
Scleroderma Anticipated Managed Access Program Timeline 2015 2016 2017 2018 2019 2020 2021 3 4 1 2 3 4 1 2 3 4 1 2 3 4 1 2 3 4 1 2 3 4 1 2 SCLERADEC-II French Clinical Study EMA Conditional MAA Submission(based on SCLERADEC-II) Reimbursement Applications EMA Full MAA Submission (based on SCLERADEC-I
Slide 21: Pipeline Indications
Pipeline IndicationsKnee OsteoarthritisUrinary IncontinenceRadiation/Nuclear Burn
Slide 22: Knee Osteoarthritis
Knee Osteoarthritis OsteoarthritisProgressive loss of joint functionImbalance between anabolic (cartilage-forming) and catabolic (cartilage-destroying) processes driven by synovial inflammation EpidemiologyOA is the most common form of arthritis13.9% of adults >25 years33.6% (12.4 million) >65 years
Slide 23: Knee Osteoarthritis: Market Overview
Knee Osteoarthritis: Market Overview 2014E Market Size: $2B Knee OA Treatment Modality AAOS Guideline US Japan ROW Hyaluronic Acid (HA) Injection * Not recommended $900M $700M $400M Situation: OA affects 10% of global population and clear unmet need Few non-surgical treatments recommended by AAOSTre
Slide 24: Rationale for use of Cytori Cell Therapy in OA
Rationale for use of Cytori Cell Therapy in OA The pathophysiology of osteoarthritis (persistent synovial inflammation leading to cartilage destruction) overlaps with other clinical indications in which Cytori Cell Therapy shown to have impactCombination of veterinary, preclinical, in vitro, and pil
Slide 25: ACT-OA Enrolled Phase II Trial
ACT-OA Enrolled Phase II Trial Phase II (ACT-OA) Study Size 94 Randomization 1:1:1 (low dose, high dose, placebo) Crossover None Sites 12 US Primary Endpoint KOOS - pain on walking Secondary Endpoints KOOS, pain/function questionnaires, pain meds, SF-36, MRI Follow-Up 48 weeks Status Enrolled24 wk d
Slide 26: Osteoarthritis Potential Development Path
Osteoarthritis Potential Development Path *Pending 48 week data, funding, and FDA approval 26 2015 2016 2017 2018 2019 2020 3 4 1 2 3 4 1 2 3 4 1 2 3 4 1 2 3 4 1 2 3 4 ACT-OA Follow-Up & Data Analysis *ACT-OA II Enrollment, 12 Month Follow-Up& Data Analysis FDA Original PMA Submission & Panel Approv
Slide 27: Stress Urinary Incontinence Program: Japan ADRESU Trial
Stress Urinary Incontinence Program: Japan ‘ADRESU’ Trial ObjectivesApproved, reimbursed therapy for SUI in men following radical prostatectomySignificant unmet need for patients whose symptoms are not responding to conservative methods SupportIIS with support from Cytori and substantial funding via
Slide 28: Radiation/Nuclear Burn Program: USA
Radiation/Nuclear Burn Program: USA ObjectivesDevelopment medical countermeasure for mass casualty event involving thermal burn ± radiation exposure Develop proof of concept clinical data for use of Cytori Cell Therapy in wound healing SupportFunded by $106MM contract from Biomedical Advanced Resear
Slide 29: Corporate Information
Corporate Information
Slide 30: Cytori Cell Therapy: Global Patent Estate
Over 80 patents issued worldwide; over 55 applications pending Cytori Cell Therapy: Global Patent Estate Goal: Protect Cytori’s proprietary methods and devices for manufacturing Cytori Cell Therapy, as well as methods of using Cytori Cell Therapy in the treatment of scleroderma, osteoarthritis, SUI
Slide 31: Capitalization Summary
Capitalization Summary Select Data – as of 12/31/15 Cash ~ $19MM* Senior term loan ~ $17.7MM Common Shares outstanding ~ 195MM Outstanding options, RSAs and warrants ~ 13MM Fully dilutive share count ~ 208MM Market capitalization ~ $35MM 31 * As of September 30, 2015 1/10/16 Q2 2014- Corporate & man
Slide 32: Financial & Operational Performance
Financial & Operational Performance * Based on revised guidance of $22 million in operating cash burn for 2015** Based on annualized figures from YTD September 30, 2015 financials 32 1/10/16
Slide 33: Summary
Summary Unique cell therapy platform- clear commercial model, late-stage lead indication and growing development pipeline Completed corporate restructuring- better positioned to achieve key milestones 2016Substantial clinical data- indicates Cytori Cell Therapy provides symptomatic benefit, improved
Slide 34: Review Cytori Corporate Milestones
Review Cytori Corporate Milestones 2016 Milestones 1st Half EU MAP program launch24 WK ACT-OA interim data evaluation2 YR follow up data SD-I presentedFull STAR enrollmentFull SD-II enrollment 2nd Half 48 WK ACT-OA data evaluationJapan & MAP progress reportedSD-II data evaluation 34 2017 Milestones
Slide 35: Thank You
Thank You