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CEO Chris Calhoun presents at the Biotech Showcase on January 13, 2014
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Cytori Corporate Overview NASDAQ: CYTX
January 13, 2014
Safe Harbor Statement
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 that we intend, expect, project, believe or anticipate will or may occur in the future are forward-looking statements. Such statements are based upon certain assumptions and assessments made by our management in light of their experience and their perception of historical trends, current conditions, expected future developments and other factors they believe to be appropriate. The forward-looking statements included in this presentation involve known and unknown risks that relate to Cytori’s future events or future financial performance and the actual results could differ materially from those discussed in this presentation. Risks and uncertainties that may cause Cytori's actual results to differ materially from those discussed in the presentation can be found in the "Risk Factors" section of Cytori’s Form 10-K, Forms 10-Q and other filings with the United States Securities and Exchange Commission. We would advise reading our most recent annual report on Form 10-K and quarterly report on Form 10-Q filed with the United States Securities and Exchange Commission for a more detailed description of these risks. The forward-looking statements contained in this presentation represent Cytori’s estimates and assumptions only as of the date of this presentation and Cytori undertakes no duty or obligation to update or revise publicly any forward-looking statements contained in this presentation as a result of new information, future events or changes in Cytori’s expectations.
Overview
Transformative technology platform – Cell therapy regulated as a device – Business model: single use consumables
Clinical experience in thousands of patients Strong IP protection Near term value drivers
– U.S. government contract milestones (BARDA) – Phase II U.S. cardiovascular trial data – International approvals and revenue growth
Proprietary Formulation of Cells • Autologous
(Patient’s own cells)
• Point-of-care availability (no offsite manufacturing)
• Patented Technology
• Virtually ‘off the shelf’ – ideal for physician workflow
Easily Accessible in Hospital & Clinic
System: • Low six figure ASP (current generation) • Next-generation system: smaller, faster, cheaper
& higher cell yields
Per-procedure consumable: • $8,000-$12,000 for vascular indications • $2,000-$3,000 for soft tissue • >80% GM
Cell therapies offered via device & single-use consumable… allows for favorable pricing, reimbursement, and margins
Commercial Model & Economics
Broad & Deep Intellectual Property DEVICES CURRENT
DEVICES NEXT GENERATION
COSMETIC & RECONSTRUCTIVE SURGERY
CARDIOVASCULAR THERAPIES PIPELINE THERAPIES
US: (6) CELUTION DEVICE (‘484) CELUTION DEVICE PLUS ADDITIVES (‘420) STEMSOURCE DEVICE (‘115) CELUTION DEVICE PLUS SENSORS FOR CLINICALLY SAFE OUTPUT (‘670) BEDSIDE COMPREHENSIVE DEVICE (‘059) CELUTION DEVICE CD31 POSITIVE CELLS (‘276) JAPAN: (2) CELUTION DEVICE (‘952) CELUTION FOR CLINICALLY SAFE OUTPUT (‘556) KOREA: (3) CELUTION DEVICE (‘995) STEMSOURCE DEVICE (‘812) CELUTION DEVICE (‘139) INDIA: (1) CELUTION DEVICE (‘706) AUSTRALIA: (2) CELUTION DEVICE (‘135) STEMSOURCE DEVICE (‘901) CHINA: (1) CELUTION DEVICE (‘689) OLYMPUS-CYTORI PATENTS NEXT GENERATION DEVICE - JAPAN CENTRIFUGAL CONTAINER (‘020) DIGESTIVE ENZYMES (‘861)
US: (1) CELUTION & FUTURE GENERATIONS (‘075) CHINA: (1) CELUTION & FUTURE GENERATIONS (‘241) HONG KONG (1) CELUTION & FUTURE GENERATIONS (‘322) INDIA: (1) CELUTION WITH CENTRIFUGE OR FILTER (‘529) AUSTRALIA: (1) CELUTION WITH CENTRIFUGE OR FILTER (‘937) SINGAPORE: (1) CELUTION & FUTURE GENERATIONS (‘683) ISRAEL: (1) CELUTION WITH CENTRIFUGE OR FILTER (‘800) MEXICO: (1) CELUTION & FUTURE GENERATIONS (‘348) KOREA: (1) CELUTION WITH CENTRIFUGE OR FILTER (‘305) JAPAN: (1) CELUTION BUSINESS METHOD
US: (7) CELUTION FOR MIXING ADRCS PLUS FAT (‘488) CELUTION OR NEXT GEN DEVICES FOR SOFT TISSUE DEFECTS (‘684) ADRCS PLUS FAT PLUS ADDITIVES (‘795) ADRCS PLUS FAT (‘672) ADRCS PLUS FAT COMPOSITION (‘121) CURRENT CELUTION DEVICE PLUS FAT (‘947) ADRC’S PLUS FAT PLUS BUFFER (‘834) JAPAN: (1) CELUTION AND NEXT GEN DEVICES FOR MIXING ADRCS PLUS FAT (‘041) KOREA: (3) ADRCS PLUS FAT (‘454) CELUTION OR NEXT GEN DEVICES FOR SOFT TISSUE DEFECTS (‘508) ADRCS PLUS FAT METHOD (‘666)
EUROPE: (2) ADRCS FOR CARDIAC (‘382) OPPOSED DEVICE FOR RESTORING BLOOD FLOW (‘575) OPPOSED AUSTRALIA: (1) ADRCS FOR CARDIAC (‘858) ADRCS FOR REMODELING (‘046) SINGAPORE: (1) ADRCS FOR RESTORING BLOOD FLOW(‘309) CHINA: (1) ADRCS FOR RESTORING BLOOD FLOW (‘104) HONG KONG: (1) ADRCS FOR RESTORING BLOOD FLOW (‘085) RUSSIA: (1) CELUTION FOR RESTORING BLOOD FLOW (‘924) SOUTH AFRICA: (1) ADRCS FOR CARDIAC (‘446) MEXICO: (1) CELUTION FOR RESTORING BLOOD FLOW (‘775) ISRAEL: (1) ADRCS FOR CARDIAC (‘354) CANADA: (1) ADRCS FOR RESTORING BLOOD FLOW(‘510) JAPAN: (2) CELUTION FOR RESTORING BLOOD FLOW(‘787) CELUTION FOR REMODELING (‘155)
US: (4) CELUTION FOR BONE (‘043) CELUTION OUTPUT PLUS PROSTHETIC FOR BONE RELATED DISORDERS (‘716) CELUTION FOR TREATING WOUND HEALING (‘580) ADRC’S FOR RENAL (‘229) EUROPE: (2) CELUTION FOR ACUTE TUBULAR NECROSIS (‘834) ADRCS FOR WOUND HEALING (‘833) JAPAN: (3) ADRCS FOR WOUND HEALING (‘699) CELUTION OUTPUT PLUS PROSTHETIC FOR BONE RELATED DISORDERS (‘119) CELUTION FOR PERIPHERAL VASCULAR DISEASE (‘511) INDIA: (1) ADRCS FOR WOUND HEALING (‘580) KOREA: (1) ADRCS FOR WOUND HEALING (‘909) CARDIAC CONTINUED KOREA (1) ADRCS FOR VENTRICULAR DILATION (‘170)
• More than just “stem cells” – Device output yields multiple cell types – Benefit from multiple mechanisms-of-action
Adipose Derived Regenerative Cells
Primary Mechanisms • Improve blood flow • Modulate inflammation • Wound healing
Commercial Research Market
Areas of Focus
Soft Tissue Cardiac
• Completed proof-of-concept trial • Enrolling U.S. Phase II HF trial • Data in 2014
• Japan approval • EU ‘Vascular’ approval • Clinical experience in
thousands of patients
• Up to $106 MM in development funding
• Commercial preparedness contract with U.S. Government
• PMA for burns
Current therapy does not address damage to the heart
Cardiac Cell Therapy
Damage leads to: reduced function
left ventricular failure . . .
Death
Cardiac Cell Therapy
Ischemic Heart Disease: Market Size
Ischemic heart disease in the United States • Direct & indirect costs of heart failure estimated at $19.5B (2009) • $11.7B paid to Medicare beneficiaries (2006) • Medical costs of IHD expected to double 2013 to 2030 • Estimated incidence is 200,000 – 500,000 patients/year in the U.S. alone
Ischemic heart disease in Europe • IHD expected to cost EU economies €60B/yr • Estimated that 34.4M and 12.1M disability adjusted life years are lost in Europe
and the EU respectively each year due to IHD
Few, if any, new pharmaceutical approaches for IHD
US Regulatory Path for Heart Failure
Development Pipeline
Chronic
Pilot trial: • Cells shown to be safe; no adverse effects on rhythm at 6 & 18 months • Statistically significant stabilization in VO2Max vs placebo Phase II ATHENA trials: • 90 patients, multi-center, double blind, placebo-controlled • Advance to pivotal in 2015
Pilot Complete U.S. Phase II
(ATHENA) Pivotal 2015
PRECISE Trial Showed Proof-of-Concept
Objective Safety and feasibility of intramyocardial injection in patients not amenable to revascularization
Design Double-blind, randomized, parallel group, placebo-controlled Size 27 patients (Spain, Netherlands, Denmark; 21 Treated, 6 Placebo) Procedure
• Electromechanical mapping (NOGASTAR®) • ≤ 15 intramyocardial injections of cells (median dose = 42 x 106 per
subject) or placebo (indistinguishable solution) in the area with inducible ischemia via MYOSTAR® Injection Catheter
Summary Results • No safety issues
• No adverse effects on rhythm at 6 and 18 months
• VO2Max: improvement (p<0.05 vs. placebo)
– Supported by NYHA improvement trend
• MRI: reduction in infarct size (p<0.05 vs. placebo)
• Mortality [36 months]: placebo 33% (2/6) , ADRC 14% (3/21)
PRECISE Trial: Data
12
14
16
18
20
Baseline 6 months 18 months
VO2 M
ax
Change in VO2Max at 6 and 18 Months
ADRCs
Placebo
PRECISE Trial: Exercise Tolerance
ATHENA Trials (USA)
• Heart failure due to ischemic heart disease
• Phase II trial design based on PRECISE
• Prospective, randomized, double-blind, placebo-controlled trials – 90 patients: 30 patients at low dose, 30 patients at high dose, 30
placebo control – Up to 10 centers
• First data readout targeted for 2014
• Potential for pivotal trial initiation in 2015
Safety • Treatment emergent Serious Adverse Events (SAEs) • Arrhythmia assessment via 24-hour Holter monitoring • MACE defined as Cardiac Death or Hospitalization for Heart Failure
Efficacy • VO2 Max (treadmill) (6 mo) • LVEF, LVESV/LVEDV assessed by Echo (6 mo) • Perfusion defect by Rest/Pharmacologic Stress SPECT (6 mo) • Heart failure symptoms, angina, and quality of life (3, 6, 12 mo):
• NYHA and CCS classifications • Minnesota Living with Heart Failure Questionnaire (MLHFQ) • SF-36 Health Survey
ATHENA Trial
BARDA Contract
Contract objective: National preparedness contract worth up to $106 million in reimbursed development funding to create a treatment for thermal burns combined with radiation
Clinical development: » PMA-based pathway for traditional burns » Development from preclinical to FDA approval fully funded » Cytori’s first U.S. soft tissue indication
Commercialization & Procurement » Cytori maintains commercial rights for approved label » Government can make direct purchases or facilitate orders
from hospitals for preparedness purposes » Procurement potential is above and beyond contract funding
BARDA Contract
Discussions Begin 2010
Award Announcement Fall, 2012
3 Objectives Trigger Option 1 & 3 Q1, 2014
3 Proof-of-concept Objectives Ongoing
Cell viability
Animal model POC
Next Gen device feasibility
$32.6 M Option 1
$23.4M Option 3
Commercial Acquisition at BARDA’s Discretion
$45.5M Option 2
Option 1 Objective Triggers Option 2 Q4, 2015
Pre-Award, White Papers, Proposal, Negotiation
$4.7M Phase 1 Proof of Concept
Commercialization
Emphasis: Researchers performing independently funded studies » 50+ studies worldwide completed, in process or planned using Celution®
Rationale: New indications & approvals expand pipeline and opportunities I. BARDA contract worth up to $106 MM (Sep. 2012) II. Lorem Vascular: $24 MM equity, $500 MM milestones (Nov. 2013) III. Japan DIET passes new Regenerative Medicine Law to Fast Track Technology
Growth: International regulatory approvals and partnerships I. Japan Class I clearance achieved II. Europe: Vascular delivery & ischemic tissue muscle approvals III. Asia Pacific:
» Australia & Singapore approvals
Objec&ve • evaluate the safety and feasibility of ADRC injected into tears of the
hamstring muscle Trial Design: • Randomized, double-‐blind, placebo-‐controlled • Part A: 10 paBent open-‐label, safety, feasibility & dose-‐escalaBon
– Low Dose: 5 paBents, High Dose: 5 paBents • Part B: 60 paBent, double-‐blind, placebo-‐controlled safety, feasibility &
dosing study – Low Dose: 20 paBents, High Dose: 20 paBents, Placebo: 20 paBents
• Safety & funcBonal end-‐points
21
RECOVER Trial (US)
Lorem Vascular Partnership
Celution® System Commercialization rights Territories: Australia, China, Hong Kong, Malaysia, & Singapore Indications: All indications w/initial focus on cardiac, diabetes and renal
Agreement Terms
Equity: $24 million; 8 million shares at $3.00 per share Milestones: Up to $500 million in commercial milestones Supply agreement: Cytori receives transfer margin on long-term agreement
Partner
Lorem Vascular: Newly formed company dedicated to Celution® products Distribution Plan: Combination of direct sales and distributors
Upcoming Milestones
ATHENA
– Complete enrollment in the U.S. ATHENA trial
– Report six-month outcomes from the U.S. ATHENA trial
– Publish PRECISE chronic ischemic heart failure trial long-term data
BARDA
– Achieve proof-of-concept objectives
– Qualify for up to $56 MM in further development funding
– Define & Initiate US Burn Pilot Trial (IDE)
COMMERCIAL
– Growth in product & contract revenues in 2014
– Obtain product registration for the Celution® System in China RECOVER
– Initiate enrollment in US trial (Part A) for hamstring injury
Cytori Corporate Overview NASDAQ: CYTX
Thank you!