Delivering gene therapy to patients

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1 Delivering gene therapy to patients

2 FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, will, would, could, should, continue, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. The forward-looking statements contained in this presentation reflect uniqure s current views with respect to future events, and uniqure assumes no obligation to update any forward-looking statements except as required by applicable law. These forward-looking statements include, but are not limited to, statements regarding the risk of cessation or delay of any of the ongoing or planned clinical studies and/or development of our product candidates, the risk of delay or failure to successfully commercialize or obtain further regulatory approval of Glybera, and the risk that our collaborations with Chiesi or our other collaboration partners will not continue or will not be successful. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors described under the heading Risk Factors in uniqure s form 20-F and the prospectus dated February 5, 2014, both documents filed with the Securities and Exchange Commission. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, except as required by law. 2

3 3 Strategy of Leadership in Curative Treatments Maximize patient benefit through single treatment interventions uniqure involved in 2 key gene therapy breakthroughs Fully integrated gene therapy value chain 1. AAV delivery 2. Manufacturing 3. Development /regulatory 4. Commercial (with partner Chiesi) Strategy > Secure lead across value chain > Build strong portfolio of programs, internal and for partnering > Source best in class programs from academia and biotech

4 What disease is this? Lifetime cost Lifetime days in hospital > US$ 10 m Min. 2 i-v. infusions per week > > 6,000 treatments over 6 decades 4

5 Hemophilia Spontaneous bleeds, joint damage, compliance What is Hemophilia? > Rare, genetic disease affecting men > Lack of / dysfunctional Factor IX or FVIII leading to spontaneous bleeds > Often affecting joints, leading to severe disabilities Significant medical need > Frequent infusions of FIX /FVIII > Infusions are painful, time-consuming, require venous access > Compliance > In-spite of current best standard of care patients still have bleeds 5

6 AAV8/FIX Single Intervention Reduces Need for Prophylactic Treatment (1) Sustained, dose dependent effect over > 4 years after a single intervention: 4/6 high dose patients did not require further FIX treatment; 4/7 on prophylaxis could stop prophylaxis % Expression of normal Low-dose Mid-dose High-dose Presented in New England Journal of Medicine 12/2011 Oral Presentation Patient Patient 10 Disease Severity Mild Moderate Severe 1) Third party trial conducted by St. Jude s Children Research Hospital and UC London 6

7 uniqure has Full Freedom to Operate Gene cassette licensed from St. Jude s Hospital, AAV5 exclusive license from NIH AAV8* ) + FIX gene** ) AAV5** ) + FIX gene** ) 7 * ) Baxter IP ** ) uniqure IP

8 Dose (gc/kg) AAV5/FIX Phase I/II Dose Escalation Study High ( ) Mid ( ) Low ( ) AAV8 mammalian ST Jude ST Jude ST Jude AAV5 insect cell uniqure uniqure Cross Referencing St. Jude FIX safety data Porphyria AAV5 safety data Population > 10 patients (severe bleeding phenotype) Severe (< 1% FIX) on prophylactic therapy Moderate ( 2% FIX) on-demand therapy > Recruitment on-going Objectives > Assess safety/tolerability > Define FIX expression level Key efficacy assessments > Factor IX activity > FIX product consumption > Annual bleeding rate > Health related quality of life Timelines > Study begin Q4/14, Q1/15 > Study results Q4/15, interim Q2 / Q3 8

9 AAV5/FIX - Efficacy in Primates Human AAV5/FIX expression levels in macaques similar to those achieved in human AAV8 clinical study (UCL/St Jude s) Linear dose response levels of human AAV5/FIX in dose escalating GLP tox study Preclinical data suggests AAV5 comparable to AAV8 Key AMT-060 in Rhesus Monkeys Porphyria Porphyria AMT-060 in Cynomolgus Monkeys Porphyria 9

10 Summary of uniqure s Hemophilia B program uniqure has all required elements in place Full freedom to operate Strong AAV5 safety given from Porphyria trial Efficacy of FIX cassette proven over > 4 years since first treatment Gene Cassette FIX Cassette excl. license from St. Jude Scalable manufacturing Direct path to pivotal trial Trial initiation: 12/2014 Vector AAV5 excl. license from NIH Manufacturing Robust, scalable, proprietary 10

11 Hemophilia A - AAV5-FVIII PoC Studies Successful uniqure FVIII construct largely equivalent to AAV5 FIX, changed gene Core issue in Hem A is oversized FVIII gene Our approach > Package 2 ends of the gene in different vectors > Restoration of full genomes upon concatemer production Result > AAV-FVIII injection results in intact FVIII mrna production > Functional FVIII protein produced upon injection in mice > High FVIII activity can be obtained in vivo 11

12 Hemophilia Development Timelines Hemophilia B preliminary safety and efficacy data mid 2015 Preclinical CTA/IND Phase I Hemophilia B Proof of Concept (mice and monkeys) Preclinical Hemophilia A CTA/IND

13 High Value of Market for Coagulation Factors and Continued Growth Market: Market Volume (in US$ bn) US$ 8 bn Growth 6% p.a. Hemophilia A Hemophilia B 13

14 Higher Benefit to Patients, Saving Health Care Cost Push lifetime cost for hemophilia gene therapy below US$ 10 m Prophylaxis US$ 10 m Gene Therapy Duration of effect paramount for pricing > Proven expression in animals > 10 years > Proven expression in humans > 4 years Drug Related Cost US$ 8.5 m US$? m Annuity based payments? > Preferred pricing model is performance based > Requires ease of measurement Non-Drug Related US$ 1.5 m US$? m Carlsson, et al., Henrad et al., company estimates 14

15 1. Hemophilia 2. Gene Therapy Leadership 3. The Portfolio

16 From natural to next generation AAV TROPISM Natural AAV Next Gen. AAV AAV 1 Exclusive for LPLD in Muscle AAV 2 AAV 5 Exclusivity for LIVER / CNS AAV 6, 8, 9 Super-Mutant development 4D Therapeutics Partnership Directed evolution Selected for > Potency > IP / FTO Available

17 Building the world s largest dedicated AAV manufacturing unit in Boston, US Amsterdam, NL > 2 x 50 L > EMA approved facility Lexington, MA, USA > 2 x 500 L (scalable to 2 x 2000 L) > Copy/scale of existing technology > Estimated time of completion for GMP batches H

18 1. Hemophilia 2. Gene Therapy Leadership 3. The Portfolio

19 Product Strategy uniqure Validation Program PRECLINICAL I II III MARKET GLYBERA EU GLYBERA US uniqure Core Programs HEMOPHILIA B HEMOPHILIA A CONGESTIVE HEART FAILURE (S100A1) Option Programs ACUTE INTERMITTENT PORPHYRIA SANFILIPPO B / other LSDs PARKINSON S DISEASE HUNTINGTON s mirna 19

20 InoCor A Solution for Congestive Heart Failure Entered Cardiovascular space through acquisition of InoCard GmbH Gene therapy (InoCor) against chronic heart failure > PoC in large animal > InoCard licensed S100A1 gene and peptide therapies > S100A1/HF program started 2001 / total of 8.5 m invested Huge unmet medical need > 5-year mortality 50% > Rapid progression > No causative treatment > No-option disease WW > 20 m pts today, x today 20

21 InoCor Unique Therapeutic Profile S100A1: one target unique efficacy 21 Adapted from Pleger ST, Raake P, Katus HA, Most P. Circulation Research (2014) 114: Targeting the heart failure calcium signalosome

22 InoCor Superior Data AAV-S100A1 AAV-control InoCor s USPs 22

23 Glybera Roll-out Plan 1 st wave > Chiesi hold EU rights, plus some selected countries > Successively country-by country > 23 30% net royalty to uniqure > Pricing dossier submissions in launch countries (Germany, Italy, UK, etc.) ongoing 2 nd wave > Distribution agreements in non-us, non-chiesi territory > E.g. Japan, Israel, ME, Korea, Australia 3 rd wave > FDA approval sought in 2018 > uniqure commercialization in US uniqure Territories Chiesi Territories 23

24 Milestones 12 Months Hemophilia B > Trial initiation year end > Preliminary data mid 2015 Hemophilia A > Primate data Glybera > EU launch Q / Q > Clarity on pricing in initial launch countries 24

25 The Leader in Gene Therapy

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