Development of differentiated immuno-oncology therapeutics by using multivalent Nanobodies

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1 Nanobodies Innovative therapeutics Development of differentiated immuno-oncology therapeutics by using multivalent Nanobodies Immune Checkpoint Inhibitors Europe Sandra Li - 17 November 2016

2 Forward looking statements Certain statements, beliefs and opinions in this presentation are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this presentation regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this presentation as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its parent or subsidiary undertakings or any such person s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this presentation or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this presentation. 2

3 Ablynx Powerful platform generating potentially innovative medicines CORPORATE Platform technology and late-stage clinical development company >350 staff in Ghent, Belgium TECHNOLOGY Pioneer in next generation antibody-derived drugs Nanobodies >500 patent applications and granted patents; critical know-how Validation through multiple partnerships with top tier pharma companies PRODUCTS >45 wholly-owned and partnered programmes 8 Nanobodies in clinical development First potential launch in 2018 PARTNERS AbbVie, Boehringer Ingelheim, Eddingpharm, Genzyme, Merck &Co., Inc., Merck KGaA, Novartis, Novo Nordisk and Taisho Pharmaceuticals > 380M cash received; > 7Bn in potential milestones; and royalties FINANCIALS 289M in cash at 30 th June M of issued Convertible Bonds maturing in

4 Hybrid business model fuels the pipeline >45 programmes, 8 Nanobodies in clinical development Product Indication Target Pre-clinical Phase I Phase II Phase III Filing caplacizumab attp vwf vobarilizumab RA IL-6R SLE IL-6R + ALX-0171 RSV RSV Up to 17 programmes Immuno-Oncology Various ozoralizumab RA TNFα Japan RA TNFα Greater China ALX-0761 Psoriasis IL-17A/IL-17F BI Oncology VEGF/Ang2 BI Chronic kidney disease CX3CR1 NA Inflammation CXCR2 ALX-0141 Bone disorders RANKL Greater China >15 whollyowned and partnered programmes Various 4

5 Unique technology What are Nanobodies?

6 Nanobodies Derived from heavy-chain only antibodies Camelid heavy-chain only antibodies are stable and fully functional Nanobodies represent the next generation of antibody-derived biologics C H 1 V H V HH 12-15kDa V L V HH Ablynx s Nanobody C H 2 C H 3 C L C H 2 C H 3 small and robust easily linked together sequence homology comparable to humanised/human mabs nano- to picomolar affinities Conventional antibodies Heavy chain only antibodies able to bind and block challenging targets multiple administration routes manufacturing in microbial cells 6

7 Ablynx s drug discovery engine Rapid generation of novel biologics Immunise llamas with antigen and/or Use proprietary synthetic Nanobody phage libraries Wide range of highly diverse Nanobodies with nM affinities Formatted Nanobodies Cloned into microbial systems and produced through fermentation ~12-18 months 7

8 Ablynx s Nanobodies Platform advantages Mix and match Multiple delivery routes Multi-specific/multivalent Nanobodies that address multiple targets in a single drug molecule flexible GS linker lengths Inhalation Ocular Oral-to-topical High-yield, highconcentration, low-viscosity, microbial production Manufacturing Able to bind and block challenging targets Nanobodies against ion channels and GPCRs Customised half-life extension Hours/days/weeks Albuminbinding Nanobody Fc 8

9 Unique modularity of Nanobodies Building a custom therapeutic GS-linker (from C- to N-term): custom linker length for maximum efficacy Bi-paratopic Nanobody: binding multiple identical or different epitopes on same target 12-15kDa anti-target A anti-target B anti-hsa payload Multi-specifics: Individual binding arms with tailored affinity Half-life extension Possibility to extend from hours to ~20 days) Microtubulin or DNA inhibitors, toxins 9

10 Unique modularity of Nanobodies Multi-specific Nanobodies versus combination mabs More difficult for mabs to bind to different targets simultaneously One tri-specific Nanobody is 4x smaller than a mab mab 2 Tri-specific Nanobody mab 3 mab 1 mab Target 1 Target 2 Target 3 Multi-specific Nanobodies may block multiple targets simultaneously Target 3 Target 2 Target 1 10

11 Nanobodies in Immuno-Oncology Applying the unique formatting flexibility of Nanobodies

12 Immuno-oncology Changing the cancer treatment paradigm Huge market potential Proven substantial survival impact Market expected to grow to >$43bn by 2020* I/O drugs expected to treat 60% of cancers* Multiple targets Increasing number of targets Combination therapies are the next generation Multi-specific Nanobodies Bind multiple targets (2, 3, 4 or 5) with one Nanobody molecule Potential to increase efficacy and avoid escape mechanisms Technology allows rapid exploration of combinations Manufacturing simplicity and cost-effectiveness Merck & Co., Inc. and Ablynx Collaboration Targeting multiple immune-checkpoint modulators Up to 17 fully-funded Nanobody programmes Focus on multi-specific combinations First in vivo pre-clinical milestone achieved in October 2015 *BofA Merrill Lynch July 2015 Nature Reviews

13 Ablynx s anti-gitr Nanobody programme Target background GITR is a member of the TNF receptor super family expressed on multiple immune cell types Unique mechanism of action and strong pre-clinical tumour model data promote tumour regression through differential effects on T Eff and T Reg enhances anti-tumour immunity through effects on other immune cells synergizes with chemotherapy, radiation, cancer vaccines, checkpoint inhibitors, etc. Several anti-gitr mabs in phase I Merck & Co., Inc.; Leap Therapeutics; others 13

14 Questions CONTACT DETAILS Sandra Li, Scientist Exploratory Pharmacology Sandra.Li@ ablynx.com