Strategic Vision. Jörn Aldag Chief Executive Officer

Transcription

1 AGENDA Strategic Vision 2. uniqure s Modular Platform & Cross Referencing 3. Gene Therapy for Liver Disease and Metabolic Disorder 4. Breakthroughs in Lysosomal Storage Disease Break 5. Gene Therapy for CNS Disorders 6. Gene Therapy for Cardiovascular Disease 7. Key Technologies for AAV-based DNA Delivery & Foundation for Success 8. Closing Remarks 1

2 Strategic Vision Jörn Aldag Chief Executive Officer

3 uniqure Delivering on the Promise of Gene Therapy Starting the Next Phase of Development GENE THERAPY INDUSTRY READY FOR PRIME TIME EXPONENTIAL GROWTH PREVIOUS YEARS FOCUS THE NEXT 5 YEARS PATIENTS FIRST BENEFITING FROM THE WORLD AROUND US 2015 A BREAKTHROUGH YEAR 3

4 Gene Therapy Industry Ready For Prime Time Recent Successes and Failures + RPE65 PH 3 DATA + SANFILIPPO B PH 1 / 2 1 ST LSD POC + GLYBERA APPROVAL / LAUNCH / PRICING + POC SICKLE CELL DISEASE / BETA THAL + MULTIPLE PRECLINICAL SUCCESSES (INCL. S100A1) + HEMOPHILIA B DURABLE EFFECT IN HUMAN LIVER + JEAN ERICKSON 1 ST CLINICAL BENEFIT IN PARKINSON S AVALANCHE - MACULAR DEGENERATION - CELLADON - HEART FAILURE - CEREGENE - PARKINSON S - SEVERAL HEMOPHILIA TRIALS - TARGETED GENETICS - CF PH 2 + RESTORED IMMUNE SYSTEMS IN 17 SCID + PATIENTS STARTING IN 2004 Before - FAILED HEMOPHILIA FIX TRIAL - FAILED X-SCID TRIAL IN

5 Gene Therapy Exponential Growth Clinical Trials and Market Cap of Listed Companies Exploded Over Past 10 Years $5.6bn $0.1bn OPEN CLINICAL TRIALS (clinicaltrials.gov) # PUBLIC COMPANIES 2005: TGEN, OXB; 2015: QURE,AAVL, BLUE, ONCE, AGTC, ARWR, CLDN, RGNX, SGMO, ABEO PUBLIC MARKET CAP (as of 10/12/2015) 5

6 uniqure in 2013 EMPLOYEES PARTNERS > ACADEMIC COLLABORATIONS LOCATIONS > AMSTERDAM, NL 45 CLINICAL PROGRAMS CASH MARKET CAPITALIZATION (EU-approved) US$ 0.4m < US$ 50m 6

7 Has Also Grown Exponentially 2015 EMPLOYEES PARTNERS LOCATIONS 190 > ACADEMIC COLLABORATIONS > BMS, CHIESI > 4D THERAPEUTICS (NEXT GEN VECTORS) > SYNPROMICS (NEXT GEN PROMOTERS) > TREEWAY FOR ALS > AMSTERDAM, NL > LEXINGTON, MASS. US > HEIDELBERG, GERMANY CLINICAL PROGRAMS CASH MARKET CAPITALIZATION Hemophilia B (Commercial) Parkinson s Sanfilippo B (proforma) US$ 250m US$ 400m 7

8 uniqure Past Years Focus Vector, Promoter, Administration, Manufacturing Technologies Continued to Evolve Vector POTENCY > Proprietary AAV5 > Rational design - in house next gen optimization > Directed evolution with 4D Mol. Therapeutics Promoter SPECIFICITY > Solve efficiency/ size dilemma > Partnership with Synpromics Administration FIT FOR PURPOSE > In-house development for CVD > CNS administration with UCSF Manufacturing SAFETY, SCALE, QUALITY > Largest industrial scale manufacturing > Safe-by-design 8

9 uniqure The Next 5 Years Delivering on the Promise of Gene Therapy GLYBERA LAUNCHED, SANFILIPPO B PH 1 / 2 SUCCESSFUL, HEMOPHILIA B PH 1 / 2 EXPECTED EOY Changing the World of Medicine with Gene-based Therapies BECOME A FULLY INTEGRATED GENE THERAPY COMPANY WITH UP TO 2 MARKETED DRUGS* IN 2020 THE LEADING GENE THERAPY CLINICAL PORTFOLIO PROPRIETARY PROGRAMS FOCUSED ON RARE AND OTHER GENETIC DISEASE PARTNERING PROGRAMS IN LARGER INDICATIONS *beyond Glybera 9

10 Patients First From Technology Platform to Therapeutic Areas uniqure Technology Basis of Success Vectors, Promoters, Manufacturing, Development, Regulatory LIVER / METABOLIC Therapeutic Focus Area CNS Therapeutic Focus Area CARDIOVASCULAR Therapeutic Focus Area 10

11 Patients First From Technology Platform to Therapeutic Focus Areas uniqure Technology Basis of Success Vectors, Promoters, Manufacturing, Development, Regulatory LIVER / METABOLISM Therapeutic Focus Area CNS Therapeutic Focus Area CARDIOVASCULAR Therapeutic Focus Area Deya Corzo, MD SVP, R&D Liver / Metabolism Lexington, MA Charles Richard, MD, PhD SVP, R&D - CNS Lexington, MA Prof. Patrick Most, M.D., PhD Managing Director - Cardiovascular Heidelberg, Germany 11

12 uniqure Strategy PATIENTS FIRST BUILDING A PORTFOLIO OF RARE DISEASE PRODUCTS ACROSS 3 THERAPEUTIC FOCUS AREAS FAST TO MARKET LEVERAGE OUR REGULATORY AND CLINICAL EXPERTISE PARTNERSHIPS FOR LARGER PATIENT POPULATIONS (E.G. BMS) TECHNOLOGY LEADERSHIP CONTINUE TO DEVELOP NEXT GENERATION TECHNOLOGIES INTERNAL AND EXTERNAL GROWTH 12

13 Benefiting from the World Around Us Academic and Industry Partnerships to Accelerate Growth ACADEMIA INDUSTRY 13

14 2015 : A Breakthrough Year for uniqure Delivering on the promise of gene therapy to patients suffering from rare and other debilitating diseases CLINICAL DATA FROM MULTI-BN $ PARTNERSHIP WITH BMS 1 ST GENE THERAPY LAUNCH 3 THERAPEUTIC FOCUS AREAS +$220 MILLION HUMAN POC TOP LINE DATA SANFILIPPO B HUMAN TRIAL DESIGNATION OF 3 ADDITIONAL COLLABORATION TARGETS IN EUROPE A GREAT EXPERIENCED LEADERSHIP TEAM SECURED YTD HEMOPHILIA B EXPECTED AROUND END OF YEAR 14