medgenics investor presentation 06/2012
DESCRIPTION
Investor presentation for Medgenics (AMEX:MDGN)TRANSCRIPT
Medgenics Presentation
June 13, 2012Andrew L. Pearlman, Ph.D. President & CEO
NYSE Amex: MDGNAIM: MEDU, MEDG
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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.
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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)
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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 Pres/CEO >25yrs Biomed
Eugene Bauer MD, Exec Chm. Former Dean, Stanford Med Sch, Connetics, Peplin
Isaac Blech-Biotech investorCelgene, ICOS, Nova
Gary Brukardt, former CEO Renal Care Group (sold for $3.5B)
Alastair Clemow PhDJ & J, Geliflex, Prolor
Joel Kanter, founding investor I-Flow, Prospect Medical, Prolor
Stephen McMurray MD RPA, Fresenius, DaVita
Clinical & Regulatory:Allen Nissenson MD – past Pres. RPA, CMO DaVita CorpAnatole Besarab MD – World authority renal anaemia, Dir. RPAStephen Fishbane MD – World authority renal anaemiaBruce Bacon MD – Leading Hep C authority- past Pres.AASLDNezam Afdhal MD – Leading Hep C authority – Chief of Hepatology, Harvard Andra E. Miller PhD – Former FDA cell/gene-therapy group leaderStephen Ettinger DVD – World renowned veterinary expertDean Hautamaki MD – Chairman Dept of Medicine , SMH
Andrew L. Pearlman PhD Founder President & CEO
Clarence “Butch” Dellio Chief Operating Officer Xoma, Neosil
Stephen Bellomo MSc VP Product Development & IP; COO Medgenics Israel
Nir Shapir PhD VP R&D Development Beckman-Coulter
Ehud Shoshani MDVP Clinical AffairsQuintiles Israel
Phyllis Bellin MBA VP AdminCitibank
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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.
(1) R&D Pipeline News, La Merie Business Intelligence, March 3, 2011
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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. Improved QOL at a lower cost.
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Biopump and a toothpick
4 Implants
10 Harvests
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 transfer of desired gene.
3. Measure each Biopump’s continuous protein production level.
4. Implant required number of Biopumps under patient’s skin.
5. Reversible by simple ablation, excision.
Biopump Method:
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DermaVac
Microorgan
Biopump
The Biopump Therapeutic System
Cryo Bank
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Repeat Bolus Injections vs. Biopump:Protein concentration in serum
Injection overshoot – Adverse side effects EPO: Cardiovascular Risk
IFN-a: Severe flu symptoms
# of Days
Injection undershoot
(No Effect)
Therapeutic window
Biopump Sustained Clinical Dose
Injected dose in range
....
Missed injection
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Biopump Platform: EPODURE in vitro: for anemia Sustained EPO high level production for 6+ months
1
10
100
1000
10000
6 9 16 25 36 46 66 80 101 122 143 164 185
IU / B
iop
um
p / d
ay
Time (Days)
EPODURE long term in vitro EPO secretion
Skin 1
Skin 2
Ti
Time to Implant in Patient
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EPODURE Replaces Injections, Elevates Hemoglobin Level Up to 36 Months of Continuous Anemia Relief:
EPO Injections EPODURE
Estimated baseline 100 days after last injection
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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 Hospital - Detroit, MI
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Biopump Platform: INFRADURE in vitro: for HepatitisSustained IFN-a high level production for 6+ months
1
10
100
1000
10000
6 9 16 27 37 48 62 76 97 118 139 160 181 202 223 244
IFN
n
g/B
iop
um
p/d
ay
Days from harvesting
INFRADURE Long term in-vitro production
Ti
Time to Implant in Patient
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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.
One Platform → Multiple Alliances:
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Valuation metrics – Recent Comparables
Licensing Deals
*For comparison purposes only. Not a form of expressed or implied outcome*
Ph III Hep C $11BNov 2011
Companies
Feb 13, 2012
AMEX: PLX
Feb 13, 2012
Cap $354m Phase II
Cap $546m Phase III
AMEX: PBTH
Ph II Hep C $2.5BJan 2012
Inhibitex
Discovery Hemophilia $213m
SangamoBioSciences
Feb 2012
BMS+
+
+
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Recent Achievements in 2012First 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
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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.
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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.
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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
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Value Proposition: Game changer – Potential major win for:
Reduces costs
Fewer claims
Preventive
Replaces frequent Injections
Improves quality of life
Prevents side effects
More reliable treatment
Safer, better outcomes
Much more affordable
Billable procedure
Improved patient flow
Increased patient compliance & treatment reliability
Blockbuster opportunities
No multi-$B protein manufacturing plant
Superior value proposition to capture market share
Patients Physicians Payors Pharma Partners
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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.
Medgenics BioMedPresentation
September 2011Andrew L. Pearlman, Ph.D. President & CEO
NYSE Amex: MDGNAIM: MEDU, MEDG