january 2015 - replicel · –2015 clinical trial starts (tendon, dermatology and hair) –2015...

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Cells for Healing Biotech Showcase January 2015

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Cells for Healing

Biotech Showcase

January 2015

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

of future events or our future financial performance. In some cases, you can identify forward-looking statements by terminology such as “may”, “should”, “intend”, “expect”, “plan”,

“anticipate”, “believe”, “estimate”, “predict”, “potential”, or “continue”, or the negative of these terms or other comparable terminology. Forward-looking information presented in such

statements or disclosures may, among other things, include: the potential of our products, including its potential for success with women; forecasts of expenditures; the sources of

financing; expectations regarding our ability to raise capital; our business outlook; plans and objectives of management for future operations; and anticipated financial performance.

Various assumptions or factors are typically applied in drawing conclusions or making the forecasts or projections set out in forward-looking information. Those assumptions and

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

information. You are cautioned that the following list of material factors and assumptions is not exhaustive. The factors and assumptions include, but are not limited to:

• no unforeseen changes in the legislative and operating framework for the business of our Company;

• a stable competitive environment; and

• no significant event occurring outside the ordinary course of business such as a natural disaster or other calamity.

These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause our or our industry’s actual results, levels of activity or

performance to be materially different from any future results, levels of activity or performance expressed or implied by these forward-looking statements. These risks and

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;

• the failure of consumers and the medical community to accept our technology as safe and effective;

• the risk that publications on which certain data in this presentation are based are withdrawn or invalidated;

• risks associated with our ability to obtain and protect rights to our intellectual property;

• risks and uncertainties associated with our ability to raise additional capital; and

• other factors beyond our control.

Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity or performance. Further,

any forward-looking statement speaks only as of the date on which such statement is made, and, except as required by applicable law, we undertake no obligation to update any

forward-looking statement to reflect events or circumstances after the date on which such statement is made or to reflect the occurrence of unanticipated events. New factors emerge

from time to time, and it is not possible for management to predict all of such factors and to assess in advance the impact of such factors on our business or the extent to which any

factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statement.

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

year ended December 31, 2013 and other periodic reports filed from time-to-time with the Securities and Exchange Commission on Edgar at www.sec.gov and with the Canadian

Securities Commissions on Sedar at www.sedar.com.

Not an Offer to Purchase or Sell Securities. This presentation is for informational purposes and is not an offer to sell or a solicitation of an offer to sell any securities in the

Company, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities

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.

2

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

3

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

4

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

5

Platform

NBDSFibroblasts(RCT-01, RCS-01)

Dermal Sheath Cup Cells (RCH-01)

Proprietary delivery device

30

RepliCel’s Position in the Regen Med Industry

6

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

7

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

8

9

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%

10

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

11

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

12

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

13

BEFORE

AFTER

New collagen growth

RCS-01 Injection

14

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

15

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

16

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

17

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

18

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

19

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

20

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

21

Rotator Cuff

Tennis Elbow

Patellar

Achilles

Hair Loss

Aged and Damaged Skin

Golfer’s Elbow

Periodontal

22

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

23

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 & [email protected]

R. Lee BucklerVP Business Development

604.248.8693 [email protected]