Corporate Presentation
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Nanobodies® creating better medicines Corporate presentation February 2017 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 2 Ablynx Rapid growth since its foundation in 2001 Today R&D – late stage 2007 o > €370M in equity and debt R&D – early stage o > €400M cash from partners o > 45 R&D projects o €70M private equity o > 1,500 patients treated o €85M IPO (Euronext) o 8 Nanobodies in the clinic 2002 o 11 R&D projects st Technology platform o 1 Nanobody expected to be o 1 Nanobody in the clinic launched in 2018 o €5M seed financing o 3 partners o 8 partners o No products o 144 employees o ~ 400 employees o No partnerships st o €235M in cash at 31 Dec 2016 o 10 employees 2001 Foundation Evolving into a commercial stage biotech company 3 3 Broad product 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 Immuno-Oncology Various programmes ozoralizumab RA TNFα Japan RA TNFα Greater China ALX-0761/M1095 Psoriasis IL-17A/IL-17F BI 836880 Oncology VEGF/Ang2 BI 655088 Chronic kidney disease CX3CR1 NA Inflammation CXCR2 ALX-0141 Bone disorders RANKL Greater China >15 wholly- owned and Various partnered programmes Filing in EU based on Phase II TITAN data 4 Four key pillars of value First marketed product expected 2018 Caplacizumab ALX-0171 Vobarilizumab Immuno- (anti-vWF) (anti-RSV) (anti-IL-6R) oncology • First-in-class treatment for • Potential breakthrough • Novel potential best-in- • Up to 17 programmes acquired thrombotic treatment delivered by class treatment for partnered with Merck & thrombocytopenic purpura inhalation for respiratory rheumatoid arthritis (RA); Co in deal worth up to • Acute, life threatening, syncytial virus infections also being studied in €5.7Bn in milestones plus ultra-rare blood clotting (RSV) systemic lupus royalties disorder • RSV can be very serious erythematosus (SLE) • First in vivo pre-clinical • No indicated therapeutic in infants, the elderly and • Partnership with AbbVie milestone achieved drug currently available the immune-compromised • RA - two successful • First clinical studies • Filed for approval in • No approved therapeutic Phase IIb studies; planned for 2017 Europe in H1 2017 drug currently available preparing for regulatory • Up to 9 clinical studies meetings and potential • Phase III results expected • Phase IIb topline results expected to start over expected in H2 2018 Phase III; exploring new next three years in H2 2017; potential BLA partnership options filing in H1 2018 • Multi-billion $ market • Market expected to grow • SLE – Phase II topline to >$43bn by 2020 • Wholly-owned; potentially • Wholly-owned results expected in H1 Ablynx’s first marketed 2018 product in 2018 • Large, multi-billion $ • Self-commercialisation markets strategy being executed • Peak sales potential of > €400M 5 Key events in 2016 Significant progress across the entire business Study results Pipeline development Corporate • ALX-0171 (anti-RSV): excellent • Bi-specific anti-VEGF/Ang2: Phase I • Caplacizumab (anti-vWF): safety and encouraging efficacy start by BI - €8M milestone publication of Phase II data from in Phase I/IIa study (53 TITAN study in the NEJM hospitalised infants with a RSV • Anti-CX3CR1 (GPCR): Phase I start infection) by BI - €8M milestone • Caplacizumab (anti-vWF): announced commitment to self- • Vobarilizumab (anti-IL-6R): • Anti-CXCR2 (GPCR): Phase I start by Novartis commercialize in North America excellent safety and superior and Europe efficacy versus tocilizumab in • Caplacizumab (anti-vWF): start of 3- • Financing: successfully raised Phase IIb RA monotherapy study year follow-up study for patients (251 RA patients) completing the Phase III HERCULES €74M through oversubscribed private placement of new shares • Vobarilizumab (anti-IL-6R): study excellent safety and efficacy in • Merck & Co., Inc.: extension (for • Vobarilizumab (anti-IL-6R): nd Phase IIb RA combination Completed recruitment of 300 patients the 2 time) of ion channel (+MTX) study (345 RA patients) in Phase II SLE study collaboration - €1M payment • Caplacizumab (anti-vWF): >100 • Novo Nordisk: discovery patients already recruited in Phase III milestone with multi-specific HERCULES study Nanobody - €1M milestone • AbbVie: declined to opt-in and license vobarilizumab in RA 3 successful studies in a 4 clinical trial starts, 3 of Important corporate total of ~650 patients which were with partners developments 6 Ablynx Diversified shareholder base – January 2017 • Ordinary shares listed on Euronext Brussels (ABLX) • Sponsored Level I ADRs on the US OTC market (ABYLY) • 61.1M shares outstanding • 2.5M outstanding warrants (in number of shares) Breakdown of share capital % of Institutional Shareholders by Geography (representing 87% of total S/O) France Van Herk Investments B.V. (NL) 2% 10% Other Scandinavia 4% Fidelity Management Research 1% 9% LLC (FMR LLC) (US) The Bank of America (US) Netherlands 14% US 5% 38% Perceptive Advisors (US) 5% GAM International (UK) 4% 64% Belgium 3% Boehringer Ingelheim (DE) 21% Other shareholders UK 20% 7 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 V H 12-15kDa VHH CH1 VHH VL Ablynx’s Nanobody • small and robust CL CH2 • easily linked together CH2 • sequence homology comparable to humanised/human mAbs CH3 CH3 • nano- to picomolar affinities • able to bind and block challenging targets Conventional Heavy chain only • multiple administration routes antibodies antibodies • manufactured in microbial cells 8 Ablynx’s Nanobody drug discovery engine Rapid generation of novel biologics Immunise llamas with antigen and/or Wide range of highly Formatted Cloned into microbial diverse Nanobodies with Use proprietary synthetic Nanobodies systems and produced 0.1-10nM affinities Nanobody phage libraries through fermentation ~12-18 months 9 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 Able to bind and block Manufacturing challenging targets Customised half-life extension High-yield, high- Hours/days/weeks concentration, Nanobodies low-viscosity, against ion Albumin- microbial channels and binding production GPCRs Nanobody Fc 10 Key value drivers Ablynx SOTP valuation – analyst average • Average target price: €15/share • Average target equity value: €880M* • Average value per programme: €29M €106M Cash minus costs Other 3% partnerships €59M 12% Merck IO 7% caplacizumab 50% €439M ALX-0171 15% €128M vobarilizumab 13% €117M *Current number of outstanding shares: 60.9M 12 Caplacizumab (anti-vWF) – wholly-owned Potential first-in-class treatment of acquired thrombotic thrombocytopenic purpura (aTTP) Caplacizumab – anti-vWF Nanobody First-in-class potential for the treatment of aTTP • Total annual market potential ~€800M1 • Feb 2017: filed in Europe for approval • Phase III HERCULES topline results in H2 2017 • Phase I results in Japanese subjects in H2 2017 • 2018: anticipated first launch in Europe and BLA submission in USA • 2019: anticipated launch in USA • Forecast peak sales of >€400M1 • Ablynx to lead commercialisation in USA, Canada and Europe • Orphan Drug Status (EU/USA) – IP protection to 2035 aTTP: acquired thrombotic thrombocytopenic purpura 1 USA, Canada, EU and Japan 14 Caplacizumab unique mode of action Rapidly stops formation of microclots Caplacizumab blocks the platelet – ULvWF interaction