january 2015 - replicel · –2015 clinical trial starts (tendon, dermatology and hair) –2015...
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Safe Harbor Statements
Statements included in this presentation that do not relate to present or historical conditions are “forward looking statements”. Forward-looking statements are projections in respect
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factors are based on information currently available to our Company, including information obtained from third-party industry analysts and other third party sources. In some instances,
material assumptions and factors are presented or discussed elsewhere in this presentation in connection with the statements or disclosure containing the forward-looking
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uncertainties include:
• negative results from any of our clinical trials;
• Failure to advance research and development programs into clinical trials;
• the effects of government regulation on our business;
• the viability and marketability of our cell replication technologies;
• our failure to successfully implement our marketing plan;
• the development of superior technology by our competitors;
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• the risk that publications on which certain data in this presentation are based are withdrawn or invalidated;
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Readers should consult all of the information set forth herein and should also refer to the risk factor disclosure outlined in the Company’s annual report on Form 20-F for the fiscal
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laws of such jurisdiction. This presentation may not be relied upon in connection with the purchase of any security. Securities of the Company, if offered, will only be available to
parties who are “qualified investors”, pursuant to applicable securities laws.
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RepliCel Overview
• Product portfolio – large commercial applications– Two distinct cell therapy platforms built on hair follicle expertise
– A unique dermal injector device
– Strong IP covering process/use of hair follicle cells in a variety of indications
• Strong near-term clinical pipeline
* funded by Shiseido
• Near-term milestones– 2015 clinical trial starts (tendon, dermatology and hair)
– 2015 injector device approval
– Data read outs for tendon and skin trials 12 months post-initiation
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Chronic tendinosis RCT-01 1/2 Canada
Aging and sun damaged skin RCS-01 1 2015 Germany
Pattern Baldness RCH-01 2 2015 Germany
Pattern Baldness RCH-01 TBD 2015 Japan *
RepliCel Overview
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Business Model: Create high-value assets for pre-commercial licensing to partners
Strategy: minimize overhead, maximize R&D output, execute deals, manage partnerships
Technology: Cell therapies. Injection device.
Markets: Orthopedics. Dermatology. Baldness.
Near-term Value Creation:
First deal (2013): $35M+ deal with Shiseido Company
Next: geographic licensing for tendon, skin & injector device
Contract manufacture (Innovacell)
Shiseido-RepliCel joint-steering committee
Fibroblast platform (RCS, RCT)
Dermal Sheath Cup Cells (RCH)
Dermal injector (RCI)
Chronic tendinosis RCT-01
Aging & sun-damaged skin RCS-01
Pattern baldness RCH-01
2015 trial launches
2016 Q1 data readouts
2015/6 geographic licenses
1. Condition is diagnosed
2. Biopsy taken from scalp
3. Cells isolated from hair follicle
4. Cells grow (5-8 weeks)
5. Cells mixed with carrier and frozen
6. Cells injected back into patient
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Platform
NBDSFibroblasts(RCT-01, RCS-01)
Dermal Sheath Cup Cells (RCH-01)
Proprietary delivery device
30
RepliCel’s Position in the Regen Med Industry
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Pre-Clinical | Early-Stage Trial | Mid-Stage Trials | Late-Stage Trials | On Market
Isolated Target Cells
Adult Stem Cells
Target Cells
Induced Pluripotent Stem Cells
Adult Stem Cells
Induction Differentiation
Target Cells
Embryonic Stem Cells
Re
gulato
ry Bu
rde
n
High
Low
Expanded Target Cells
Allogeneic & Autologous
Differentiated cell products
RepliCel represents a lower technical, clinical & regulatory risk than many companies in the sector
NBDS
DP
DSC
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Platform Technology from Hair Follicles
• Non-bulbar dermal sheath (NBDS) fibroblast cells are prolific type I collagen production (5x dermal fibroblasts)1
• Active fibroblasts promote tendon healing and dermal matrix rejuvenation
• RCT-01 Phase 1/2 (Tendon), RCS-01 Phase 1 (Skin Aging)
Dermal Sheath Cup Cells (DSC)
Fibroblast Platform (NBDS)
• DSC cells are responsible for the regulation of the volume of dermal papillae (DP) cells
• DP cells are responsible for the thickness and growth of a hair fibre
• RepliCel’s RCH-01 Phase 2 (Pattern Baldness)
• Shiseido’s RCH-01 Trial (Pattern Baldness) 1 Source: Jun Kyu Oh, et al, JSID, 2012
Chronic Tendinosis – Fibroblast Deficit
• Chronic tendinosis
– Cycle of incomplete healing & re-injury
– Chronic degeneration of the tendon
– Problem: deficit of active fibroblasts
• Standard of Care
– Physiotherapy, PRP, ultrasound, acupuncture, dextrose injections and surgery
• Incidence (North America 21-60 yr old)
– Achilles: 485,000 (0.24%)
– Elbow: 6,200,000 (3.0%)
– Knee: 412,000 (0.20%)
• RepliCel Treatment
– Injection of fibroblasts into the injury
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Phase 1 Chronic Achilles Tendinosis - 63 Yr-old Male
• 3-years of chronic pain
• Failed eccentric loading, casting & platelet rich plasma
• Chronic tendinosis: unorganized tissue formation
• Pain reduction
• Tendon thickness reduction
• Organized tissue formation
• Healing complete: return to normal tendon structure
Ultrasound Image Before Treatment – Day 1
Ultrasound Image After Treatment – 6 Months
Source: D. Connell et al, JBJS 2012, Note: JBJS has issued an expression of concern about this publication due to proper clearances. Both Connell and RepliCel believe these allegations are without merit and working to resolve the allegations.
Phase 1 Chronic Achilles Tendinosis
Past Clinical: Phase 1 Achilles Tendinosis1
(D. Connell et al., Royal National Orthopaedic Hospital)
Treatment – Adipose derived dermal fibroblasts
• 24 patients (unilateral disease)
– 12 treated, 12 controlled
– Mean age 45.2 years (20 male, 12 female)
– VISA questionnaire & VAS scores @ 6 months
• VISA median values (p<0.001)
– Cell group improved 127%
– Control improved 11%
• VAS median values (p<0.001)
– Cell group decreased 66%
– Control decreased 20%
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Function
Pain Measurement
1 Source: D. Connell et al, JBJS 2012
Chronic Tendinosis – Fibroblast Deficit
• Phase 1/2 Trial for Achilles Tendinosis (UBC) cleared by Health Canada
– Primary endpoint - safety through 6 months - then
• volunteers will be solicited for an additional 18 months follow-up
• next-phase study application will be submitted to Health Canada
– 28 patients, 21 treated & 7 placebo
– Estimated 3 months to complete enrollment
– Secondary endpoint – efficacy at 6 months
• Function, pain, tendon appearance & biomechanics, blood flow, activity scale, quality of life measured at 1, 2, 4, & 6 months
– Treatment: fibroblast cells from hair follicle sheath
• Abundant, healthy and easily accessible
• Prolific producers of type I collagen
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Aging and Sun Damaged Skin – Fibroblast Deficit
• Skin damage caused by aging, UV exposure, lifestyle factors
• As we age fibroblasts in the skin produce less collagen causing skin to lose its elasticity and structure
• Injection of collagen-rich fibroblasts is designed to reverse skin damage
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YOUNG SKIN OLDER SKINHistology Histology
Collagen Fibroblast FibroblastCollagen
RCS-01 Proposed Phase 1 Trial 2014
• Treatment: Injection of RCS-01 fibroblasts
– Prolific producers of type I collagen
• 28 healthy volunteers (22 treated, 6 placebo)
• Primary endpoint – local safety & tolerance
• Secondary endpoints
– Efficacy at 6 and 12 months
– 16 patients biopsied for gene expression of skin aging markers
– 12 patients biopsied for histopathology analysis
• Assess structural characteristics underlying the skin
• Assess molecular markers associated with skin aging
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BEFORE
AFTER
New collagen growth
RCS-01 Injection
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Dermal Sheath Cup Deficit: Pattern Baldness
• Human scalp follicles develop with a fixed amount of dermal papillae (DP) cells (approx. 500)
• Total # of DP cells determines the # and thickness of hair fibers
• Diminishing volume of active DP cells leads to pattern baldness
• Drug treatments do not increase DP cells
• Only dermal sheath cup (DSC) cells regulate the volume of DP cells and therefore hair fiber growth
• RepliCel solution: deliver missing dermal sheath cup cells (collected from back of scalp)
DP
DSC
Current Standard of Care
• Micro-transplant surgery is the current gold standard
– Highly dependent on surgeon’s skill
• Rogaine (minoxidil) – topical solution suitable for men and women
– Approx. 8-16% density increase from baseline (at 12 months)
– Treatment maintenance required or gains are lost
• Propecia (finasteride) – oral pill not suitable for women
– Approx. 7-14% density increase from baseline (at 12 months)
– Treatment maintenance required or gains are lost
• Replicel Dermal Sheath Cup phase 1 averaged 11.8% density increase at 6 months for its responders (5%)
– Safety endpoint achieved; no adverse events
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RCH-01: Phase 1 Safety - Six Month Results
• 63% of subjects responded with increases in density greater than the 6-month 5% target1
– Mean change in total hair density at 6-months = 6.1% vs 5.0% target
• 70% of responders were above 10% - average 14.3%1
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19.6%
19.2%
17.2%
13.6%
12.0%
11.2%
10.6%
9.6%
6.2%
5.1%
2.1%
1.9%
1.1%
-3.7%
-4.6%
-6.2%
Change in total hair density after 6 months
≥5% responders (n=10)Average = 11.8%
≥10% responders (n=7) (70%)Average = 14.3%
1 Source: RepliCel’s 6-month interim analysis results from Phase I trial
RCH-01: Phase 2 Pattern Baldness
• Planned Phase 2 Trial for Pattern Baldness 2015 (Charité, Freiburg and Münster)
– 160 participants
– 396 RCH-01 treatment group sites (vs. 16 in P1), with 244 placebo sites
– Designed to measure dosing and treatment frequency
– Primary commercial endpoint – efficacy at 52 weeks after final injection
• efficacy measured as total density, hair thickness, cumulative hair and treatment response rate
– Secondary endpoints – 52 weeks
• local tolerance, systemic safety
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Screening
Inclusion Criteria:
• Male pattern baldness
RCH-01 Group 2: Day 1 + day 91 injections, 4 sites, 3 doses, 1 placebo (n=66)
Placebo Group 1: Single placebo injection, 4 sites, all placebo (n=14)
Placebo Group 2: Day 1 + day 91 repeat injections, 4 sites, all placebo (n=14)
N = 160
RCH-01 Group 1: Single injection, 4 sites, 3 doses, 1 placebo (n=66)
3rd Party Validation & Value Creation
• 2013 licensing partnership with Shiseido (July 2013)
– Geographic license for pattern baldness only for Japan, China, Korea and ASEAN nations
– $35 Million ($4M upfront, $31M in sales milestones and royalties)
– Shiseido is the 4th largest cosmetic company WW 2013
• Joint product and clinical development; shared data
• Shiseido funds own facility and trial
– Shiseido opened new cell-processing facility May 2014
– Completing tech transfer & comparability/validation runs
– Capable of handling all aspects of the RCH-01 technology
• Biopsies, cell isolation and expansion, packaging and shipment
• Actively pursuing additional geographic partners for other indications
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Injector Devices
Generation 1 Injector
• Dermal injector
Generation 2 Injector (RCI-02)
• Devices for RCS and RCH
• Programmable depth and dose
• Built-in freezing replaces anaesthetic (Pelletier Element)
• Treatment specific interchangeable heads
• Prototype build stage
• CE mark, 510K pathway
• Focused licensing program into dermal filler market
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Dermal Filler Market
PRODUCT # OF PROCEDURES
GROWTH RATE
PROCEDUREFEE
APPROX. PRODUCT
COST
APPROX. PRODUCTMARKET
Botulinum Toxin
3,766,148 15.6% $392 $10-17/unit $2+ billion
Hyaluronic Acid 1,872,172 31.5% $552 $200-$800/syringe
$1.2+ billion
Others 253,335
TOTAL 5,891,655
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2013 statistics provided by the American Society of Plastic Surgeons
• $1.9 billion spent on physician fees for skin rejuvenation procedures in 2013
Potential Therapeutic Applications
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Rotator Cuff
Tennis Elbow
Patellar
Achilles
Hair Loss
Aged and Damaged Skin
Golfer’s Elbow
Periodontal
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Pro-Forma Financial Highlights
in $CAD
STOCK LISTING TSXV: RPOTCQB: REPCF
Recent Share Price (CDN) $0.35
52 week high/low (CDN) $0.99 - $0.30
Market Cap. (approx.) $20.0 M
Cash & Equivalents (09/31/14) $3.3 M
Current Monthly Burn Rate $350,000
Shares Outstanding (Basic/FD) 54.9/67.4M
Share Ownership- Founders/Officers/Insiders 43.5%
Anticipated Trial Costs- RCH-01 (24 months)- RCH-01 Japan (24 months)- RCT-01 (9 months)- RCS-01 (3 months)
$2 - $2.5 M$0 M$1 M$0.5 M
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Upcoming Development and Strategic Milestones
Program Event Est. Timing
RCT-A-01 – Tendon Initiate Phase 1/2 trial in chronic Achilles tendinosis
RCS-01 – Skin Initiate Phase 1 trial in sun damaged and aging skin Q1 2015
RCH-01 – Hair Initiate Phase 2 trial in pattern baldness H1 2015
RCH-01 – Hair Shiseido initiate trial in pattern baldness H1 2015
RCI-02 – Injector Generation 2 device CE mark approval filing H2 2015
RCT-A-01 – Tendon Phase 1/2 data on chronic Achilles tendinosis 12 months
RCS-01 – Skin Phase 1 data on sun damaged and aging skin 12 months
RCT-P-01 – Tendon Initiate Phase 1/2 study in chronic patellar tendinosis H2 2015
RCH-01 – Hair Phase 2 data on pattern baldness 2016
Corporate Licensing 2015/2016
RepliCel Life Sciences Inc.
OTCQB: REPCFTSXV: RP
David M HallCEO & President604.248.8730dhall@replicel.com
R. Lee BucklerVP Business Development
604.248.8693 lee@replicel.com
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