Download ACP-01 - Hemostemix

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WWW.HEMOSTEMIX.COM
TSXV: HEM
SAFE HARBOR
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COMPANY OVERVIEW
Clinical-stage cell-therapy company
Platform technology derived from simple draw of autologous
(patient’s own) blood for treatment of vascular diseases
• 
• 
• 
• 
Strong evidence of angiogenic (blood-vessel forming) capacity in humans
Shown to be safe in 45 clinical trial patients and >400 compassionate use patients
Strong intellectual property estate
Established manufacturing system with low COGS
Ongoing international multicentre phase-2 trial for critical limb
ischemia (CLI).
• 
• 
Severe disease with large number of patients (large market) with poor treatment
options (strong market need)
CLI is gateway indication to other ischemic indications
Industry-experienced CEO and management team
• 
Demonstrated success in designing and running a phase-2 CLI cell therapy trial
COMPANY OVERVIEW
Trading
TSXV: “HEM”
Stock Listings
Headquarters
Calgary, AB
Current Share Price (CA$)
$0.84
Fiscal Year End
April 30
Shares Outstanding
65.6 million
Accounting Firm
MNP LLP
Market Cap
$55 million
Legal Counsel
HLF LLP
30-Day Average Trading Volume
17,000
Investor Relations
Stonegate Capital
Partners
Warrants
1.4 million
Avg Price = $1.75
Options
5.5 million @ $0.10
0.6 million @ $0.80
Insider ownership
(Officers & Directors)
26.7%
Cash @ June 30, 2015
$1.5 million
2015 Annual Cash Burn
$3.3 million
(As at July 31, 2015)
MANAGEMENT TEAM
Dr. Ina Sarel
VP R&D &
Manufacturing
Dr. Elmar Burchardt
Developed HEM’s GMP cell manufacturing system
•  Director of R&D at Theravitae
•  Former Project Manager at ProNeuron Biotechnologies
•  15+ years as a scientist and project manager in cell
therapy companies
•  PhD from Boston University
President & CEO
Dr. Hardean Achneck
Medical Doctor with 20+ years in the biopharma industry
•  Former VP of Regenerative Medicine at Pfizer
•  Designed and ran phase II CLI trial at Aastrom
•  Involved in 40+ cell therapy clinical studies with
hundreds of patients in North America, Europe and Asia
•  MD & PhD from University of Hannover; second PhD
from University of Witten/Herdecke
VP Clinical Research &
Operations
Dr. Rahul Sarugaser
VP Corporate
Development
10+ years in medical research, and designing and
running clinical trials
•  Former Assistant Professor of Surgery & Pathology at
Duke University
•  Conducted clinical studies on endothelial progenitor cells.
•  MD from Yale University
Buy and sell-side experience funding clinical stage companies
•  Director, Business Development at CCRM for 4+ years
•  Built industry network of ~120 companies; executed 16 JV partnerships
•  Prior experience at GE Healthcare, and in Venture Capital
•  PhD in stem cell research from University of Toronto; MBA from
University of Oxford
CRITICAL
LIMB
ISCHEMIA
DISEASE PROFILE
Chronic, inadequate blood
flow to limbs, resulting in
tissue loss and gangrene1,2
INCIDENCE & PREVALENCE
Millions
of Patients Globally
NO treatment
for most critical patients
United States & Canada Estimates (2015):
UP
TO
UP
TO
25
30%
% Mortality
Rate
Amputation
Rate
By 1 year post-diagnosis
1http://www.ucdmc.ucdavis.edu/vascular/diseases/cli.html
2 http://www.vascularweb.org/vascularhealth/Pages/peripheral-artery-disease-
%28-pad-%29-.aspx
4.2
1.2 Million
Million
Current CLI Patients
New CLI Patients Each Year
CRITICAL
LIMB
ISCHEMIA
Critical Limb Ischemia
& Buergers Disease
Ventricular Device Assist
Tip of the Iceberg
$2B Market
(orphan indication)
Ischemic Dilated Cardiomyopathy
Congestive Heart Failure
Acute Myocardial
Infarction
Peripheral
Arterial
Disease
Gateway
TO BROADER
Ischemic Diseases Market
$20B+ Market
LEAD CLINICAL
PRODUCT
ACP-01 for Critical Limb Ischemia
1
Extract and enrich desired cell
population from patient’s blood
2
Inject cell population to form
new blood vessels in dying tissue
selfdonor
ACP-01
Offers Hope for
CLI Patients Facing Amputation
uses patient’s own cells, no immune
rejection, no observed safety issues
simple
cell harvest
via blood draw
quick
7 days from
draw to admin
Shipping:
Blood shipped to
manufacturing facility
Isolation:
Cells are selected and
enriched from blood
enrich
& collect
desired
cells
ACP-01
Technology Platform
Collection:
isolate
desired
cells
Simple 250 ml
blood draw
Manufacturing:
Cells are incubated
with proprietary
cocktail of factors
package
cells into
syringes
collect
patient’s
blood
inject cells
back into
patient
Shipping:
Product shipped for
injection back into patient
Progressive healing of chronic toe ulcer in
CLI patient after treatment with ACP-01
Pre-Treatment
ACP-01
Case Study
Day 17
Day 33
Day 90
O N G O I N G
ACP-01
TARGET PATIENTS
DATA COLLECTION
CLINICAL TRIAL
PHASE 2
33 control
6
MONTH
MINIMUM
FOLLOW-UP
66 treated
international
multicentre
prospective
controlled
randomized
double-blind
Diagnosed with CLI
No treatment options
Controlled levels in
blood sugar, blood
pressure, cholesterol
adverse events
treatment failure1
pain
quality of life
No previous amputations
above the ankle on target limb
1Major
CANADA
S AFRICA
USA
TAIWAN
amputation, 2x wound size, new gangrene or death
O N G O I N G
ACP-01
CLINICAL TRIAL
PHASE 2
33 control
66 treated
CLINICAL ENDPOINTS
defined by
“TREATMENT FAILURE”
•  Mortality
or
•  Major amputation
6
MONTH
MINIMUM
FOLLOW-UP
international
multicentre
prospective
controlled
randomized
double-blind
or
•  Doubling of wound size
or
•  De novo gangrene
CANADA
S AFRICA
USA
TAIWAN
WHY USE
“TREATMENT FAILURE”
•  Each category contributes in
approximately equal proportions
to treatment failure
•  Treatment failure is much more
likely to occur during 6 month
interval than limb amputation
resulting in high event rates
•  Larger dataset enables greater
differentiation between treated
and control groups to determine
significance
•  Trials using time to treatment
failure rather than “incidence of
amputation” are smaller and
more cost-effective
TIMELINE
15/Q3
16/Q1
16/Q2
Interim
Data
Phase-2 trial for CLI
US FDA IND clearance
15/Q4
16/Q3
16/Q4
17/Q1
17/Q2
Complete
✔
Mechanistic MRI study
(Israel/Germany)
Add US manufacturing site
Automation of manufacturing
process to further reduce COGS
è
IND & CTA submission for
additional indication/s
Phase-2 trial(s) for additional
indication(s)
è
COMPARABLE COMPANIES
Company
Ticker Mkt Cap* Cell/source
Approach
Phase
Indication(s)
Hemostemix
HEM
$58M
ACP/peripheral blood
Autologous
2
Pluristem
PSTM
$193M
MSC/placenta
Allogeneic
1, 2
Critical limb ischemia (CLI),
Intermittent claudication (IC)
Vericel
VCEL
$86M
MSC/bone marrow
Autologous
2, 3
Dilated cardiomyopathy (DCM),
Critical limb ischemia (CLI) (halted)
ReNeuron
RENE
$90M
NSC/central nervous
system
Allogeneic
1
Critical limb ischemia (CLI)
Caladrius
CLBS
$103M
HSC/bone marrow
Autologous
3
ST-elevated myocardial infarction (STEMI)
Capricor
CAPR
$81M
Cardiac/heart
Allogeneic
1, 2
Cytori
CYTX
$83M
MSC/adipose
Autologous
2
*As at market close July 3, 2015
Critical limb ischemia (CLI)
Dilated cardiomyopathy (DCM),
Acute myocardial infarction (AMI)
Chronic heart failure
COMPARABLE TRANSACTIONS
Licensor
Licensee
Technology Disease Area/Indication(s)
Upfront
Deal Value*
Pluristem
Cha
PLX-PAD
Bio&Diosteck
Critical Limb Ischemia
2
Cell
therapy
$10M
$10M
2013
Pervasis
Shire
Vascugel
Cardiovascular
2
Cell
therapy
Undisclosed
$200M
2012 Worldwide
Capricor
Janssen
CAP-1002
MI, heart failure, DCM,
ischemic heart disease
2
Cell
therapy
$13M
$338M
2014 Worldwide
Athersys
Chugai
MultiStem
Ischemic stroke
2
Cell
therapy
$10M
$200M
2015 Japan
Pfizer
Medicines
Co.
ApoA-1
Milano
Cardiovascular
2
Biologic
$10M
$420M
2010 Worldwide
Resverlogix
Shenzen
Hepalink
RVX-208
Cardiovascular
2
Small
molecule
$0
$429M
2015
Portola
Novartis
Elinogrel
Cardiovascular
2
Small
molecule
$75M
$575M
2009 Worldwide
Isis
Pharmaceuticals
Bayer
ISIS-FXIRx
Thrombosis
2
Antisense
drug
$100M
$475M
2015 Worldwide
*Deal value = Upfront + Milestone Payments
Stage
Modality
Date Territory
South
Korea
China,
Taiwan
INVESTMENT OPPORTUNITY
Strong human evidence of angiogenic (blood-vessel forming)
capacity
• 
• 
• 
• 
Platform technology derived from simple draw of autologous blood
Shown to be safe in 40 clinical trial patients and >400 compassionate use
patients
Robust intellectual property portfolio
Established manufacturing system with low COGS
Product addresses large market
• 
• 
• 
Ongoing phase-2 clinical trial for CLI
CLI is a severe disease with large number of patients (large market) with
poor treatment options (strong market need)
CLI is gateway indication to other ischemic diseases
Industry-experienced CEO and management team
• 
• 
Demonstrated success in designing and running a phase-2 CLI cell therapy
trial
Designed state-of-art trial to enable successful endpoint readout
SUMMARY
Early
Studies
Lead
Product
Indicate Potential
Efficacy
Phase-2
CT
ACP-01 for CLI:
Significant unmet need
Cost-Effective
& Scalable
Angiogenic
Platform
Technology
Team
One Protocol
Canada, U.S., S. Africa, Asia
IP
Experienced
Anecdotal Human
No Safety
Efficacy data Issues Observed
Growing
Patent Portfolio
Focus
Long-Term Focus:
Exploit Platform & Expertise
for Other Ischemic Indications
APPENDIX
BOARD OF DIRECTORS
Victor Redekop
Director
R Lee Buckler
Director
35+ years international executive
experience
Founder & Managing Director of
Cell Therapy Group
Officer, Director & Principal,
Simmons Edeco
Co-founder & CEO of
RegenerativeMedicineJobs.com
Co-founder of a gas transmission
company and other international
oil and gas ventures
Past roles with Progenitor Cell
Therapy, STEMCELL
Technologies, and International
Society for Cell Therapy (ISCT)
Director of several public and
private companies, including
Nations Energy, Bankers
Petroleum, and BNK petroleum.
Chartered accountant
Co-Chair, Alliance for
Regenerative Medicine’s
Communications and Education
Committee
Founding Chair of ISCT’s
Commercialization Committee
CW (Bill) Baker
Chairman
40+ years diverse senior executive
experience including biotech,
resource, manufacturing, sports
management and service sectors.
Has served on the Board of several
companies and associations.
Founding shareholder and
Chairman of the Company since
2006.
Elmar Burchardt
David Wood
President & CEO
Director
Medical Doctor with 20+ years in
the biopharma industry
CEO, CFO, and Director of
DataMiners Capital Corp
Former VP of Regenerative
Medicine at Pfizer
Founder & President, Zenith
Appraisal and Land Consulting
and Double Check Consulting
Designed and ran phase II CLI
trial at Aastrom
Involved in 40+ cell therapy clinical
studies with hundreds of patients in
North America, Europe and Asia
MD & PhD from University of
Hannover; second PhD from
University of Witten/Herdecke
Director of several public and
private companies, including
Nations Energy
Professional Appraiser (Appraisal
Institute of Canada)
SCIENTIFIC ADVISORY BOARD
Mark Jay Zucker
MD, JD, FACC, FACP
Director, Harvey & Georgina Weinstein National Heart
Transplant Center (Beth Israel Medical Center)
Norman CW Wong
MD, FRCP (C)
PhD
Faculty of Medicine Professor, University of Calgary
Chair-elect, Tissue Engineering and Regenerative
Medicine International Society
(European Chapter).
Past Associate VP Research & International, University of
Calgary
Head, Ludwig Boltzmann Institute for
Experimental and Clinical Traumatology
Past faculty member, Mount Sinai School of Medicine
and Loyola University Medical School
Past Assistant Dean of Research, University of Calgary
Advisory Board Vice-Chair & Board Director, New Jersey
Organ & Tissue Sharing Network
Past Director, Libin Gene Therapy Unit at the University
of Calgary
Associate Professor, Vienna University of Technology
Coordinator, Austrian Cluster for Tissue
Regeneration
25+ years in heart and lung transplantation
Consultant for leading biopharma companies, including Eli
Lilly, Merck Frost, Solvay Pharma, GlaxoSmithKline,
Abbott Labs
Clinical Professor of Medicine, University of Medicine and
Dentistry of New Jersey Medical School
Co-founder & CSO, Resverlogic Corp (TSX:RVX)
Heinz Redl
CEO, Trauma Care Consult
Principal or co-investigator on 40+ trials including cellbased therapies (BioHeart myoblast and Baxter RENEW
trials)