Date post: | 05-Dec-2014 |
Category: |
Health & Medicine |
Upload: | cytori-therapeutics-inc |
View: | 1,140 times |
Download: | 0 times |
Marc Hedrick, MDPresident
2
Safe Harbor Statement
This presentation may contain certain ‘forward-looking statements’. 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 are also subject
to a number of material risks and uncertainties. We caution investors not to
place undue reliance on the forward-looking statements contained in this
presentation.
We would advise reading our annual report filed with the United States
Securities and Exchange Commission on Form 10-K for a more detailed
description of these risks.
Cytori- Brief Summary
• Estimated 4-5,000 patients treated world wide
• Completed 3 clinical trials, 2 CV trials in progress
• Growing product portfolio
• Commercial growth
• Expanding regulatory approvals
• Pipeline focus
– Cardiovascular disease
– Soft tissue repair
3
Adipose Derived, Point of Care Cell Therapy
4
Cell Type Frequency
Endothelial cells
(CD34+/CD31+)
7%
CD34+/CD31-/CD45- cells
(CFU-F)
38%
1-5%
Smooth muscle cells 9%
Leukocytes 22%
Tissue Macrophages 23%
Culture = ADSCs
Adipose Enzymatic Digestion
(no enzyme, no cells)
No Culture = ADRCs/SVF
Publication Heat Map
5
ADRCs ADSCs
Organ System Preclinical
Clinical Preclinical
Clinical
Cardiovascular 6 3 17 0
Pulmonary 1 2 4 0
Orthopedic 5 1 30 1
Muscular 1 0 6 0
Central Nervous System 0 0 11 0
Peripheral Nervous System 2 1 7 0
Hepatobiliary/Pancreatic 0 0 10 2
Gastrointestinal 0 1 3 5
Immunologic (Autoimmune, GVHD)
0 0 5 7
Genitourinary 1 1 11 0
Renal 1 0 2 0
Subtotal 17 9 106 15
Plastic and Reconstructive 4 9 17 1
Dental 0 0 6 2
Subtotal 4 9 24 3
Grand Total 21 18 130 18
• No Adipocytes
• Multipotent cells
• Endothelial cells
• Vasc. smooth muscle cells
• Tissue resident macrophages
• Perivascular cells
Key Attributes of Adipose Derived Regenerative Cells
Device & Therapeutic
6
0
15
30
45
Total Nucleated Cells in Adipose
0
0.25
0.5
Multipotent Cells in AdiposeMultipotent Cells in BM
Mixed Cell Population High Cell Number Potency
million/100cc
Cardiovascular Trials- Effectiveness of ADRCs in Ischemia
7
APOLLO: ST Segment
Elevation Myocardial Infarctions
Objective • Safety and feasibility study in ST-elevation myocardial infarction (STEMI) Cells delivered via intra-
coronary route
Study design • Double-blind, placebo controlled
Sample size • N = 14
Randomization • 3:1 randomization (ADRC: Placebo)
Outcome assessment
• Primary at 6m / Long-term at 36m LVEF, wall thickness, wall motion, perfusion defect, pro-BNP
Patient selection
• STEMI 2- 12 hours duration• Successful PCI• Ejection Fraction 30-50%
Core laboratories
• All SPECT, MRI, 2D-Echo, 3D-Echo images were evaluated by independent, blinded core laboratories
Safety oversight
• An independent, blinded, Data and Safety Monitoring Board conducted scheduled and ad-hoc safety review of the study
PRECISE: No Option Chronic
Myocardial Ischemia
Objective • Safety and feasibility in chronic ischemia patients Cells delivered via a NOGA™ catheter patients not eligible
for percutaneous or surgical revascularization
Study design • Double-blind, placebo controlled
Sample size • N = 27
Randomization • 3:1 randomization within each cohort (21 active, 6 placebo)
Outcome assessment
• Primary at 6m / long-term at 36m LVEF, wall thickness, wall motion, perfusion
defect, VO2 Max, pro-BNP
Patient selection
• NYHA Class II-III
• Ejection Fraction < 45%
• Inducible ischemia on SPECT
Core laboratories
• All SPECT, MRI, 2D-Echo, 3D-Echo images were evaluated by independent, blinded core
laboratories
Safety oversight
• An independent, blinded, Data and Safety Monitoring Board conducted scheduled and ad-hoc safety review of the study
Cardiovascular Trials- APOLLO (AMI)
8
24.4 cc/ -72.2% improvementTime
Placebo ADRC Tx
Change in mV02 from baseline to 6 and 18 months
The PRECISE Trial
ADRCs
PlaceboBaseline 6 mo 18 mo
Transplantation
20.0
18.0
16.0
14.0
19.0
15.5 15.3
16.6
17.117.2
P < 0.05 P < 0.05
Cardiovascular Trials- PRECISE (Chronic Ischemia)
9
mV
O2
(L/m
in)
Cardiac Development Status
10
Clinical Data
PRE-IDE Q4 ‘11
Initiate 2012
Regulatory Reimbursement
Clinical Data
CE Mark Application Filed 2011
Regulatory
(2012)Reimbursement
Clinical Data
Pilot complete
Pivotal began 2011
Regulatory Reimbursement
EU Chronic Myocardial Ischemia
EU Acute Myocardial Infarction
US Chronic Myocardial Ischemia ATHENA
Cell Enriched Fat Grafting
11
Cell Enriched Grafting for Breast Reconstruction
12
RESTORE 2: Reconstruction in BCT (lumpectomy and RTx)
Study design • Prospective, single-arm, EU study of cell enriched fat grafting after BCT
• Baseline performance study, no control group• Seven centers, 70 patients
Co-primary
endpoints
• Patient satisfaction with functional and cosmetic results and
improvement in overall breast deformity at 12 mo• Physician satisfaction with functional and cosmetic results and
improvement in overall breast deformity at 12 mo
Secondary endpoints
• Change in breast volume and shape (MRI)• Improvement in breast deformity• Number of treatments required to achieve positive changes in
selected endpoints• Improvement in patient Quality of Life scores
Patient selection
• Quadrantectomy for ≤ T2N0M0, up to 3 cm• Min of 12 mo from last treatment to enrollment, no recurrence• Mild to moderate breast damage with ≥ 2/3 of the breast
mound remaining
Pre Tx
Cell Enriched Grafting for Breast Reconstruction
12 months
Post Op
13
100cc defect60 GySingle treatment150 cc wet CEFG
150cc defectNo RTTwo treatments
150/100 cc wet CEFG
Cell Enriched Grafting for Breast Reconstruction
14
Co-primary Endpoints
Endpoint Outcome
Physician and patient satisfaction with overall treatment
• Physician- 78% (6 mo), 85% (12 mo)• Patient- 70% (6 mo), 75% (12 mo)
Secondary Endpoints
Endpoint Outcome
Change in breast volume &
contour (MRI)
• MRI volumetric analysis ineffective in these lesions
• Improved or much improved- 77% (6 mo), 83% (12 mo)
Improvement in soft tissue (Lent Soma and cutaneous scoring)
• Improvement in fibrotic indices but not in pain and coloration
Adverse Event profile • Safe, no cancer recurrences, benign lipid cysts were reported as adverse events in 10 patients (14.9%)
Number of treatments required • 1.35 (24 of 67 had second procedure)
Improvement in patient Quality
of Life scoresNo improvement detected with SF-36. This instrument is now
known to lack sensitivity in breast reconstruction. New instrument (breast-Q) is more sensitive in this population.
Resource utilization Average LOS was 7 hours-2 days, inpatient treatment 41/68 (60%) at baseline 19/24 (79%) at optional 6-month treatment
Breast Reconstruction Development Status
15
2006
• RESTORE 1 Initiated in Japan
2007
• CE Mark for general processing
• RESTORE 1 data reported
2008
•Cytori begins RESTORE 2 trial in EU
2009
• RESTORE 2 enrollment complete
2010
• Breast reconstruction added to CE Mark
2011
• UK NIC identifies cost-effective-ness
• Complete RESTORE 2 data reported
Urethral Lumen
• 90 yr old Female
• 2 year chronic wound
• Spine exposure
• Radiation Tx > 40 yrs
• Single treatment
• Complete healing
Dr. AkitaNagasaki, JapanWHO Center for Advanced Nuclear & Radiation Medicine
Pre-Treatment 1 year Post-Treatment
Day 0 Day 14 Day 21 Day 68 Day 89
Wound Healing
Key Milestones 2012
17
Initiate ATHENA trial in U.S. – done
CE Mark for Chronic myocardial ischemia
Publication of key EU breast and CV data
Breast reconstruction technology evaluation in UK
Japanese and other non US regulatory approvals
Expand our commercial scope via partnerships