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RNA Treat Foundation Therapeutics 1 Forward looking statements This presentation contains forward-looking statements that disclosed in our forward-looking statements, and you should not involve substantial risks and uncertainties. All statements, other place undue reliance on our forward-looking statements. Actual than statements of historical facts, contained in this results or events could differ materially from the plans, presentation, including but not limited to, statements regarding intentions and expectations disclosed in the forward-looking our strategy, future operations, future pre-clinical and clinical statements we make. The forward-looking statements contained trial plans and related timing of trials and results, research and in this presentation reflect our current views with respect to development, future financial position, future revenues, future events, and we assume no obligation to update any projected costs, prospects, therapeutic potential of our products, forward-looking statements except as required by applicable law. plans and objectives of management, are forward-looking These forward-looking statements are subject to a number of statements. The words “aim,” “anticipate,” “believe,” “estimate,” risks, uncertainties and assumptions, including those that may “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” be described in greater detail in the annual report filed on Form “potential,” “will,” “would,” “could,” “should,” “continue,” and 20-F for the year ended December 31, 2016 that we have filed similar expressions are intended to identify forward-looking with the U.S. Securities and Exchange Commission (the “SEC”) statements, although not all forward-looking statements contain and any subsequent filings we have made with the SEC. We have these identifying words. included important factors in the cautionary statements included in that annual report, particularly in the Risk Factors Forward-looking statements represent our management’s beliefs section, and subsequent filings with the SEC that we believe and assumptions only as of the date of this presentation. We could cause actual results or events to differ materially from the may not actually achieve the plans, intentions or expectations forward-looking statements that we make. ProQR Therapeutics – R&D Day 2017 2 Our mission and strategy Build strong RNA Treat foundation therapeutics Genetic • Experienced team with • Elegant & highly targeted approach Diseases proven track record • Emergent RNA field with 5 • Top-notch science and approved products collaborators • Established modality through >20 • ProQR was founded to • Broad IP estate years of experience (delivery, find a treatment for CF safety, manufacturing, etc.) • Less than 10% of genetic diseases have a treatment Created Build a Creating treatments for valuable sustainable • pipeline business severe rare diseases where we can have a big • Diversified product pipeline • Best-in-class products • Focused on high unmet • Achievable commercial impact medical need and accelerated strategy in rare diseases development pathways • Drive shareholder value ProQR Therapeutics – R&D Day 2017 3 ProQR: 2012 - now Strong foundation under solid company Track record of success Advance- ments to show for it Dinko Valerio Rapidly Top notch advanced Scientific company collaborators Henri Termeer In-house Track record Strong experts in of success team many disciplines James Shannon ProQR Therapeutics – R&D Day 2017 4 ProQR: 2012 - now Strong foundation under solid company Management team Supervisory board Advance- ments to show for it Daniel de Boer Noreen Henig Gerard Platenburg Dinko Valerio James Shannon Chief Executive Officer Chief Medical Officer Chief Innovation Officer Chairman Rapidly Top notch advanced Scientific company collaborators Smital Shah René Beukema Robert Cornelisse Antoine Papiernik Alison Lawton Chief Financial Officer Chief Corp. Development Chief People Officer & General Counsel & Organization In-house Track record Strong experts in of success team many disciplines David Rodman Paul Baart Chief Development Strategy Officer ProQR Therapeutics – R&D Day 2017 5 ProQR: 2012 - now Strong foundation under solid company 150 employees in EU and US work on discovering and developing therapies Advance- ments to show for it Rapidly Top notch advanced Scientific company collaborators In-house Track record Strong experts in of success team many disciplines ProQR Therapeutics – R&D Day 2017 6 ProQR: 2012 - now Strong foundation under solid company Rapidly advanced entrepreneurial company Advance- ments to In 2 years from foundation show for it • 2014: to Nasdaq IPO, raising ~$200M • 2015: In 3 years from IP to start Rapidly Top notch global clinical trial advanced Scientific company collaborators • 2016: In 4 years to clinical PoC in lead product In-house Track record Strong experts in • Unique operating model with of success team many product focused biz units disciplines enabling rapid development of company and pipeline ProQR Therapeutics – R&D Day 2017 7 ProQR: 2012 - now Strong foundation under solid company Top notch science and collaborators Advance- ments to show for it Rapidly Top notch advanced Scientific company collaborators In-house Track record Strong experts in of success team many disciplines ProQR Therapeutics – R&D Day 2017 8 ProQR: 2012 - now Strong foundation under solid company Positioned for success QR-010 (CF) with positive clinical data Advance- • ments to • Phase 1b top line data expected in show for it September 2017 • Phase 2 to start in 2018 • QR-110 (LCA 10) IND clearance for clinical trial • IND cleared, first patient to be dosed soon, trial Rapidly Top notch completed in 2018 advanced Scientific company collaborators • QR-313 (DEB) clinical trial to start in 2018 • Clinical trial to start and readout in 2018 • Discovery pipeline that has the potential to In-house Track record Strong experts in deliver new IND every year of success team many • Ophthalmology, Axiomer® disciplines • Cash position (end Q1 2017): € 52.1M • Runway into Q3 2018 ProQR Therapeutics – R&D Day 2017 9 Therapeutic Strategy Patient centric Well understood causality Local delivery Best-in-class high impact Single gene defect leading to Feasible delivery route products for patients in need disease manifestation to target organ Genetic rare diseases RNA therapy Accelerated development Limited treatment options, Highly specific approach for a Accelerated development strategies viable commercial strategy wide range of mutations to treat patients faster ProQR Therapeutics – R&D Day 2017 10 Key programs in ProQR pipeline Cystic Inherited Debilitating Highlights Fibrosis blindness skin disorders Innovation pipeline QR-010 for F508del QR-110 for LCA10 QR-313 for DEB Axiomer® cystic fibrosis Pipeline Pipeline • Novel RNA editing Positive clinical data in • QRX-411 for Usher syndrome • QRX-323 for DEB platform technology NPD biomarker study • QRX-421 for Usher syndrome • QRX-333 for DEB • Direct ADAR to make • Phase 1b study top line • QRX-504 for FECD • QRX-343 for DEB specific edits in RNA data expected in • QRX-1011 for Stargardt’s • >20.000 G>A mutations September 2017 Disease ProQR Therapeutics – R&D Day 2017 11 QR-010 Cystic fibrosis due to F508del mutation Presenter: Noreen Henig ProQR Therapeutics – R&D Day 2017 12 Cystic fibrosis due to F508del mutation F508del High unmet Lung & Limited life expectancy of Most common mutation medical need other organs 27 years affects ~65,000 patients ProQR Therapeutics – R&D Day 2017 13 Cystic Fibrosis is a heterogeneous population Stop codon mutations • More than 2,000 CFTR mutations known Gating • F508del patients mutations • Variable drug response within F508del population • Expected that several different therapies are needed to treat different subgroups Homozygous F508del of F508del population • Beyond QR-010 for F508del • RNA therapies have potential to target Heterozygous F508del stop-codon mutations ProQR Therapeutics – R&D Day 2017 14 Cystic Fibrosis is a heterogeneous population Stop codon mutations • More than 2,000 CFTR mutations known Gating • F508del patients mutations • Variable drug response within F508del population • Expected that several different therapies are needed to treat different subgroups Homozygous F508del of F508del population • Beyond QR-010 for F508del • RNA therapies have potential to target Heterozygous F508del stop-codon mutations ProQR Therapeutics – R&D Day 2017 15 QR-010 for F508del cystic fibrosis Differentiating product profile INHALED DRUG for lung delivery and systemic uptake SINGLE AGENT for F508del to treat underlying cause of disease CONVENIENT AT HOME DOSING 3 times a week or less in under 15 minutes AIMS TO STOP PROGRESSION OF DISEASE or prevent disease and improve quality of life ProQR Therapeutics – R&D Day 2017 16 QR-010: RNA target comes with advantages CFTR • Single agent • Low treatment burden Chloride • No predicted drug- drug interactions RNA • No predicted carcinogenicity DNA • No predicted teratogenicity ProQR Therapeutics – R&D Day 2017 17 QR-010 for F508del cystic fibrosis Patients are ill because of absence of CFTR function. QR-010 has shown to restore CFTR function QR-010 has been found safe and well tolerated in ongoing clinical trials Smart development program to reduce development time to approval Inhalation delivers effectively to lung and distributes to other organs QR-010 restores CFTR function in CF patients ProQR Therapeutics – R&D Day 2017 18 Bringing QR-010 to CF patients Two Clinical Trials by mid-2017 PHASE
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