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Company Presentation

Company Presentation. March 2014. Forward Looking Statement.

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Company Presentation

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  1. CompanyPresentation March 2014

  2. Forward Looking Statement 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.

  3. Company Overview • History • Founded in 2000in Misgav, Israel • Traded on NYSE-MKT (MDGN) and LSE-AIM (MEDU and MEDG) • Technology • >$100 million invested in ex-vivo gene therapy platform • 107 patents filed (49 issued) around platform • Leadership • Chairmen Sol Barer, Founder and former Chairmen and CEO of Celgene • New management team joined September 2013 • Location • R&D center – Misgav, Israel • U.S headquarters – Wayne, PA

  4. New Management Team Has Proven Track Record of Success • President, Shire Specialty Pharmaceuticals- Responsible for all aspects of the $2.5 billion business e.g.., commercial, R&D, manufacturing, etc. • President, Life Sciences, Safeguard Scientifics – Lead Life Sciences PE team, responsible for both investment and management of portfolio companies • Multiple senior operational leadership positions at Astra Merck/AstraZeneca Mike Cola CEO • Responsible for >20 FDA approvals in multiple therapy areas • Corporate Vice President of Science and Technology, Johnson and Johnson - Multiple senior R&D leadership positions at Astra Merck/Merck KGa/AstraZeneca • Head of R&D, Appletree Partners – Responsible for both investment, overall R&D strategy, and management of portfolio companies in PE fund Garry A Neil, MD Global Head of R&D • Vice-President Commercial Assessment for Shire Specialty Pharmaceuticals - Responsible for both corporate and business unit M&A and white space strategies • Principal, Devon Park Bioventures - Responsible for all aspects of venture investing e.g., negotiation, board governance, financing for $125M Life Sciences VC fund • Associate Principal, McKinsey & Company – Worked on strategic issues for multiple Top 25 pharmaceutical companies; specialized in BD/M&A, and payer/reimbursement issues John H. Leaman, MD CFO

  5. Investment Highlights • BioPump™ offers sustained production and delivery of therapeutic proteins using ex-vivo gene therapy and the patient’s own tissue • Demonstrated clinically relevant production of therapeutic proteins in 28 patients in Phase 1/2 EPODURE study • Multiple BioPump programs in rare and orphan disease markets to initiate in 2014 • Strong financial position with more than $22M in cash as of December 31, 2013

  6. BioPump™ System Technology Overview

  7. The BioPump™ System Unlocking the potential of gene therapy Harvest tissue from under patient’s skin with minimal pain via needle biopsy into a sealed cassette BioPump Process 1 Harvest Process tissue into drug producing BioPump by controlled transfer of desired gene using safe “gutless” Adeno vector technology ex-vivo 2 Process Measure each BioPump’s continuous protein production level – patient’s personalized protein – to calibrate therapy 3 Measure 4Implant Implant required number of BioPumps under patient’s skin 5 Titrate Change dose by adding or removing BioPumps

  8. BioPump™ Offers Sustained and Consistent Protein Delivery Superior protein kinetics with BioPump approach • Repeated infusion of proteins results in peaks and troughs • Peak levels are well above therapeutic range resulting in potential side effects • Trough levels are below the therapeutic range resulting in lack of efficacy • BioPump maintains protein levels within the therapeutic window at steady state Peak BioPump Therapeutic window Trough # of Days Injection Injection Injection Missed Injection

  9. Advantages of BioPump™ Approach Continuous and autologous production • Patients become their own “bioreactor” producing protein with unique autologous glycosylation patterns • Autologous proteins generally have several advantages: • Longer duration in vivo • Greater cell uptake • Less immunogenicity • Maintenance of proteins within therapeutic window mimics more closely the natural physiological state versus injected/infused protein therapies

  10. Advantages of BioPump™ Approach Improved safety, tolerability and compliance • Patients not exposed to risk of immune response associated with systemic viral vector used in gene therapy • Reduced chance of developing neutralizing antibodies leading to reduced efficacy and potential anaphylactic reactions • Changing dose is possible through addition or removal of BioPump • Implantation ensures compliance, better outcomes, and provides complete control of the product

  11. Advantages of BioPump™ Approach Capital efficient, de-risked path for drug development • Exogenous gene therapy on “tummy tuck” tissue is performed to test human protein expression levels in vitro • Severe combined immunodeficient (SCID) mice provide predictive in-vivo model for protein expression and duration • In-vitro (human) and in-vivo (SCID mouse) models provide pre-clinical POC, greatly de-risking clinical trials for approximately $100K and less than 6 months • Using patient as their own bioreactor eliminates need for large investment in manufacturing and scale up for clinical trial material

  12. BioPump™ System EPODURE Clinical Development Plan

  13. EPODURE™ Clinical Programs 1st Generation EPODURE BioPump™ displayed safety and efficacy in phase 1/2 trial; insufficient durability of expression for commercially viable product • Demonstrated capability to produce endogenous protein at clinically relevant levels over a several month period • BioPump showed physiologically relevant levels of erythropoietin (EPO) production with concomitant increase in hemoglobin in renal patients • EPODURE showed excellent safety and tolerability profile • No serious adverse events reported • Decision to delay EPODURE Phase II trial based upon improved pre-clinical performance of 2nd Generation BioPump

  14. 1st Generation BioPump Demonstrated Clinically Relevant Protein Production Levels For One Month Chronic Kidney Disease Patients (n=13) One month of erythropoietin production translates into 4-5 months of anemia treatment given lifespan of red blood cells (90-110 days) Therapeutic level of EPO

  15. 2nd Generation BioPump™ Demonstrated Physiologically Relevant Protein Production Levels for Greater Than 6 months In-vivo SCID Mice In-vivo SCID mouse models have been highly predictive of human efficacy Therapeutic level of EPO Note: Comparative data from two separate experiments; 1st Generation (n=5); 2nd Generation (n=5)

  16. BioPump™ System Strategy Update

  17. 2nd Generation BioPump™ Has Significant Advantages Over Current Protein Replacement Therapies • Current protein and enzyme replacement therapies are made exogenously in bioreactors with various expression systems and infused into patients • High burden to patient to travel to a clinic or use home infusion creates compliance issues leading to poor outcomes • Peak to trough kinetics and non native glycosylation patterns create reduced efficacy and increased immunogenicity • Increased immunogenicity can eventually lead neutralizing antibodies that eliminate the drug before any clinical benefit

  18. Improvement Shown in 2ndGeneration BioPump™ System Opens New Market Opportunities • Unique Proteins and Peptides (>Phase 1 development) 122 • Filtered-out Molecules ~500 • Acute treatments • Chronic • Treatments with limited potential significant improvement with BioPump 30 • Bio-better molecules • Further prioritization • High Potential Targets • Therapeutic Proteins and Peptides

  19. Prioritization • Further prioritize disease areas by: • Unmet need • Market opportunity • Technical feasibility • Several orphan products identified as highly attractive targets • Targets include group of products with proof of concept data but failed further development due to a lack of ability to produce the drug substance or insufficient therapeutic half-lives • Preclinical work has begun on the highest potential targets: • Synthesize cDNA for viral vector production • Create in-vitro models using human “tummy tuck” tissue • Create in-vivo models with SCID mice • Preclinical models should be very predictive of performance in human subjects based on experience with EPODURE • Targets cannot be disclosed at this time until program specific IP is secured

  20. Upcoming Corporate Milestones • Activities • Timing • Initiate multiple programs in rare and orphan disease • Pre-clinical proof-of-concept data • 2H 14 • Further validate BioPump platform • Initiate EPODURE trial with 2nd Generation BioPump™ System • EPODURE trial results • 1H 14 • 2H 14 • Leverage BD to expand on company’s strategy • Build on company’s expertisein ex-vivo gene therapy and protein/peptide drug development • TBD

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