Medgenics Investor Presentation 06/2012

Medgenics Presentation

June 13, 2012
Andrew L. Pearlman, Ph.D. President & CEO

NYSE Amex: MDGN
AIM: MEDU, MEDG

Forward-Looking Statements:
This presentation includes certain estimates and other forward-looking statements
within the meaning of Section 21E of the Securities Exchange Act of 1934, as
amended, including statements with respect to anticipated operating and financial
performance, clinical results, potential partnerships, licensing opportunities and
other statements of expectation. Words such as “expects,” “anticipates,”
“intends,” “plans,” “believes,” “assumes,” “seeks,” “estimates,” “should” and
variations of these words and similar expressions, are intended to identify these
forward-looking statements. While we believe these statements are accurate,
forward-looking statements are inherently uncertain and we cannot assure you that
these expectations will occur and our actual results may be significantly different.
These statements by the Company and its management are based on estimates,
projections, beliefs and assumptions of management and are not guarantees of
future performance. Important factors that could cause actual results to differ from
those in the forward-looking statements include the factors described in the
Company’s filings with the U.S. Securities and Exchange Commission. The
Company disclaims any obligation to update or revise any forward-looking
statement based on the occurrence of future events, the receipt of new information,
or otherwise.

2

Truly Personalized Medicine:
 Innovative med-tech company developing sustained
protein therapies for chronic diseases utilizing
proprietary “Biopump” technology.
 “Biopump” provides for continuous protein production
and delivery from patient’s own skin.
 Designed to be better, safer and cheaper, replacing
scores of injections, in $130b protein market.
 Potentially offering major advantages in treating a wide
range of chronic diseases starting with anemia, hepatitis
and hemophilia.
 Proof of concept shown in patients: 6 months to 3 years
sustained treatment.

(1) RNCOS—Global Protein Therapeutic Market Analysis (Ed. 3, May 2010) 3

Experienced Management Team:
Extensive experience in healthcare industry, founded, operated and led firms to M&A
totaling billions of dollars.
Board of Directors: SAB/Advisors: Management:
Andrew L. Pearlman PhD Clinical & Regulatory: Andrew L. Pearlman PhD
Pres/CEO >25yrs Biomed Allen Nissenson MD – past Pres. Founder President & CEO
RPA, CMO DaVita Corp Clarence “Butch” Dellio
Eugene Bauer MD, Exec Chm. Anatole Besarab MD – World Chief Operating Officer
Former Dean, Stanford Med Sch, authority renal anaemia, Dir. RPA Xoma, Neosil
Connetics, Peplin Stephen Fishbane MD – World Stephen Bellomo MSc
Isaac Blech-Biotech investor authority renal anaemia VP Product Development &
Celgene, ICOS, Nova Bruce Bacon MD – Leading Hep C IP; COO Medgenics Israel
authority- past Pres.AASLD
Nir Shapir PhD  
Gary Brukardt, former CEO Nezam Afdhal MD – Leading Hep C VP R&D Development
Renal Care Group (sold for $3.5B) authority – Chief of Hepatology, Beckman-Coulter
Harvard
Andra E. Miller PhD – Former FDA Ehud Shoshani MD
Alastair Clemow PhD VP Clinical Affairs
J & J, Geliflex, Prolor cell/gene-therapy group leader
Quintiles Israel
Stephen Ettinger DVD – World
Joel Kanter, founding investor renowned veterinary expert Phyllis Bellin MBA
Dean Hautamaki MD – Chairman VP Admin
I-Flow, Prospect Medical, Prolor Citibank
Dept of Medicine , SMH
Stephen McMurray MD RPA,
Fresenius, DaVita
4

Key Considerations:
 Proprietary Biopump, an autologous tissue-based platform technology for
the sustained production and delivery of therapeutic proteins.
 3 lead products address markets >$16B/yr in anemia, hepatitis and
hemophilia.
 EPODURE: Anemia/EPO - Completing Phase I/II in Israel, cleared for Phase IIa trial
in dialysis patients in Israel commencing in Q2; IND cleared for Phase IIb in USA;
 INFRADURE: Hepatitis/Interferon alpha – awaiting clearance of Phase I/II trials in
Israel to treat hepatitis-C in Q2; filed for Orphan Drug Designation in hepatitis D; and
 HEMODURE: Hemophilia/FVIII - being developed as a sustained Factor VIII therapy
for the prophylactic treatment of hemophilia.
 Reimbursement: aiming at replacement value of current therapy

 Clinically demonstrated: one treatment can relieve anemia for 6-36 months.

 US FDA Clearance for Phase IIb Trial in dialysis patients with anemia

 IP protection: 30+ issued and 70+ pending patents.

5
(1) R&D Pipeline News, La Merie Business Intelligence, March 3, 2011

Lead Products
 EPODURE (anemia) Sustained EPO therapy ($9.2B/yr market) could
replace $15-30,000/yr/patient in injections, potentially offering:
 Superior treatment at lower cost; 6-36+ months sustained EPO therapy -
avoid peak overdose risks, improve compliance and reliability.
 Improved hemoglobin control, directly address current key issues in anemia:
• FDA hemoglobin safety, CMS reimbursement bundling

 INFRADURE (hepatitis) Sustained IFN-a therapy ($2.7B/yr mkt) could
replace $35-85,000/yr/patient for current therapies, potentially offering:
 Effective treatment with greatly increased compliance, reduced side effects –
tolerable and safer, with unmatched treatment interval of 6+ months.
 Cost effective alternative for most patients with hepatitis C, hepatitis B, and others,
potentially replacing costly triple-treatments and new oral drugs.
 Filed for Orphan Drug Designation in hepatitis D – potential expedited approval

 HEMODURE (hemophilia) Sustained FVIII therapy ($4.4B/yr mkt) could
replace >$100-250,000/yr/patient injections, potentially offering:
 Prophylactic treatment – rather than “rescue” injections.
 > 6-12 months sustained FVIII therapy from single treatment.
6
 Improved QOL at a lower cost.

Biopump Method:
1. Harvest the tissue by needle biopsy from
under patient’s skin.

2. Process tissue into a drug producing
Biopump in 10-14 days by controlled Biopump and a toothpick
transfer of desired gene.

3. Measure each Biopump’s continuous 10 Harvests
protein production level.
4 Implants

4. Implant required number of Biopumps
under patient’s skin.

5. Reversible by simple ablation, excision.
7

The Biopump Therapeutic Syst

DermaVac

Microorgan

Biopump

8
Cryo Bank

Repeat Bolus Injections vs. Biopump:
Protein
Injection overshoot – Adverse side effects
concentration
EPO: Cardiovascular Risk
in serum
IFN-a: Severe flu symptoms

Therapeutic
window
.. ..

Biopump Sustained Clinical Dose

# of Days
Injection
undershoot Missed injection
Injected dose in (No Effect)
range
9

Biopump Platform: EPODURE in vitro: for anemia
Sustained EPO high level production for 6+ months
EPODURE long term in vitro EPO secretion

10000
Skin 1
Skin 2

1000
IU / Biopump / day

100

10

1
6 9 16 25 36 46 66 80 101 122 143 164 185
T ime (Days)

Ti
Time to Implant
in Patient
10

EPODURE Replaces Injections, Elevates Hemoglobin Level
Up to 36 Months of Continuous Anemia Relief:

Estimated baseline
100 days after last
injection

EPO Injections EPODURE

11

Phase I/II Interim Study Conclusions:
 Presented at ASN 2010, 2011 by leading authorities.*

 EPODURE is safe and doseable; no antigenic response. EPO serum
level always stayed within normal physiological range.
 Clinical feasibility demonstrated.

 Single EPODURE administration can raise and maintain hemoglobin
(Hb) levels for up to 36 months without any injection of ESAs.
 Elevated, maintained Hb 3+ mo in 13/17 patients, 6+ mo in 8/17
“We believe EPODURE may have significant
potential to become an effective interventional
treatment – a paradigm shift.”
Allen Nissenson ,MD, CMO of DaVita Corp and past president of RPA

Anatole Besarab, MD, Director of Clinical Research, Division of
Nephrology and Hypertension, Henry Ford 12
Hospital - Detroit, MI

Biopump Platform: INFRADURE in vitro: for Hepatitis
Sustained IFN-a high level production for 6+ months

INFRADURE Long term in-vitro production
10000

1000
IFN ng/Biopump/day

100

10

1
6 9 16 27 37 48 62 76 97 118 139 160 181 202 223 244
Days from harvesting

Ti
Time to Implant
in Patient

13

One Platform → Multiple Alliances:
Business model – Revenues before product approval.
Platform: Same low-cost core technology – multiple deal opportunities.
Major Opportunities: Each >$1B/year, no protein factory needed.
Other Opportunities:
 Niche applications – rapid route to product approval; high value-
added.
 New proteins/markets.
Timing: Typical deals at Phase I/II or Phase II.
Early Revenue Source:
 Pre-approval milestone payments, typically $100M+.
 Royalties on product sales, or transfer price.
14

Valuation metrics – Recent Comparables
Licensing Deals Companies

BMS
+ +
Inhibitex AMEX: PBTH
Ph III Hep C $11B Ph II Hep C $2.5B Cap $354m Phase II
Nov 2011 Jan 2012 Feb 13, 2012

+
SangamoBioSciences
AMEX: PLX
Discovery Hemophilia $213m Cap $546m Phase III
Feb 2012 Feb 13, 2012

*For comparison purposes only. Not a form of expressed or implied outcome* 15

Recent Achievements in 2012
First Quarter
 Positive meeting with NIH RAC (Recombinant DNA Advisory
Committee) for Phase IIb Trial in dialysis patients with Anemia
Second Quarter
 Approval to initiate Phase IIa clinical study of EPODURE in
patients on dialysis in Israel
 Filed for FDA Orphan Drug Designation for INFRADURE to
treat hepatitis D
 US FDA Clearance to initiate Phase IIb Trial in dialysis patients
with Anemia
 Launched US Biopump GMP processing center in California,
produced EPODURE Biopumps meeting all release criteria
16

Anticipated Milestones thru 2012:
 Anemia:
 Launch Phase IIa EPODURE Israel study in dialysis patients
 Launch of Phase IIb EPODURE U.S study in dialysis patients
 Hepatitis C: Approval and launch of first INFRADURE
clinical trials in Israel
 Phase I/II in relapsed responding patients
 Phase I/II in treatment naïve patients
 Obtain FDA Orphan Drug Designation for INFRADURE in
hepatitis D
 Partnering: Pursue strategic alliances; active discussions
continue with potential partners. 17

Route to Revenues:
Regulatory:
 FDA Clearance for Phase IIb EPODURE anemia trial (May 2012)
 Filed for Orphan Drug INFRADURE for hepatitis D (April 2012
), potential for expedited approval route using small pivotal trial.
 QA designed in: automated processor using sealed cassettes.
Clinical Pathway to Approvals:
 Delivery of well-known proteins now in routine clinical use.
 Better compliance – always on board.
 Better safety: own protein, no peak overdose or under-dose
between injections, ability to reverse or stop treatment
Scale Up:
 Reliable method >10,000 Biopumps made.
 Closed system: Biopumps shipped to/from remote clinical sites to
central GMP processing facilities
 Planning upgrades, automated bioprocessor – early development.
18

Pipeline for Biopump Platform:
Condition Protein Development stage 2010 Sales ($b)*

Anemia Erythropoietin At Phase II 9.2
Hepatitis Interferon Alpha Phase I/II (launching Q3) 2.7
Hemophilia Factor VIII Preclinical 4.4
Growth Retardation Growth hormone Future Candidate 3.0
Multiple Sclerosis Interferon Beta Future Candidate 6.5
Diabetes Insulin Future Candidate 15.5
Arthritis IL-1Ra Future Candidate 20.9
Wound Healing PDGF-BB Future Candidate NA
Obesity Peptide YY3-36 Future Candidate NA
Chronic Pain IL-10 Future Candidate NA
Cancer Recovery G-CSF Future Candidate 5.4

(1) R&D Pipeline News, La Merie Business Intelligence, March 3, 2011
19

Value Proposition:
Game changer – Potential major win for:
Patients Physicians Payors Pharma
Partners
 Replaces frequent  Billable procedure  Reduces costs  Blockbuster opportunities
Injections
 Improved patient flow  Fewer claims  No multi-$B protein
 Improves quality of life manufacturing plant
 Increased patient  Preventive
 Prevents side effects compliance & treatment  Superior value
 More reliable treatment reliability proposition to capture
market share
 Safer, better outcomes
 Much more affordable

20

Key Take-Aways:
 Disruptive platform technology for >$130B protein
market, multiple partnering opportunities, strong IP portfolio.
 Strong value proposition: Potentially better, safer, less costly.
 Lead products >$16B focusing on anemia (EPODURE)
hepatitis (INFRADURE) and hemophilia (HEMODURE).
 Successfully demonstrated in patients: 6-36 months from a
single treatment in patients, FDA Clearance for Phase II study
 Significant partnering potential; active discussions.
 Experienced, proven team.
 2012 milestones: Launch Phase IIb anemia trial in
U.S., Launch 3 trials in Israel (Ph IIa anemia, Ph I/II hepatitis
C); Obtain Orphan designation for INFRADURE in hepatitis D. 21

Medgenics BioMed
Presentation
September 2011
Andrew L. Pearlman, Ph.D. President & CEO
NYSE Amex: MDGN
AIM: MEDU, MEDG

Medgenics Investor Presentation 06/2012

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