2017 half year results - ablynx ablynx 2017 half year results presentation participants on the call...
TRANSCRIPT
Nanobodies®
creating better medicines
Webcast presentation – 24 August 2017
2017 Half Year Results
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Ablynx 2017 half year results presentation
Participants on the call
Dr Robert K. Zeldin
CMO
Dr Edwin Moses
CEO
Wim Ottevaere
CFO
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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 including, without limitation, the
timing for completion of certain milestones in our agreements and the amount of any milestone payments
we may receive, our expected net cash burn, the progress of our key pre-clinical and clinical development
programmes, the timing of the commercialization of, and the estimated market potential and projected
peak sales for, our product candidates, the anticipated duration of the patent protection we receive for our
product candidates, and our ability to create value from the development and commercialisation of our
product candidates. 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.3
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Ablynx 2017 half year results presentation
Agenda
• Welcome and introduction
• Financial highlights
• Operational performance
• Outlook
• Q&A
• Conclusion
Financial highlights
Half Year results 2017
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Financial summary
Half year results 2017
€ million HY 2017 HY 2016 Change
Revenue 34.7 53.1 35%
Grant income - 0.4 100%
Total revenue and grant income 34.7 53.5 35%
R&D expenses (50.5) (49.0) 3%
G&A expenses (8.9) (6.5) 37%
Operating result (24.8) (2.0) > 100%
Financial income 3.1 28.4 89%
Financial expenses (3.7) (3.5) 6%
Profit/(loss) for the period (25.3) 22.8 > 100%
Net cash flow (30.9) (19.0) (1) 62%
Cash (4) at 30 June 204.5 (2) 288.7 (3) 29%
(1) excluding €71.4 million net proceeds from the private placement of new shares (1 June 2016)
(2) including €1.6 million in restricted cash
(3) including €1.3 million in restricted cash
(4) cash, cash equivalents, restricted cash and other financial assets at the end of the period
Following the initiation of the collaboration with Sanofi, and a review of the timing of other
milestone payments, net cash burn guidance for full year 2017 is lowered to €65-75 million
Product pipeline
Important progress in key pre-clinical and clinical development
programmes
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Broad product pipeline
>45 programmes, 7 Nanobodies® in clinical development
IndicationProduct Target
aTTP
RSV
vobarilizumab
Pre-clinical Phase I Phase II Phase III
caplacizumab vWF
ALX-0171
ALX-0141 RANKL
ALX-0761/M1095
RSV
Bone disorders Greater China
IL-17A/IL-17F
ozoralizumab TNFα
Greater China
Filing
Japan
RA
SLE
RA
Psoriasis
Immuno-Oncology
>20 wholly-owned and partnered programmes
Up to 17
programmes
IL-6R
IL-6R
TNFαRA
Various
Oncology VEGF/Ang2BI 836880
Chronic kidney
diseaseCX3CR1BI 655088
Filing in EU based on Phase II TITAN data
Phase III data
late Q3 2017
SLE Phase II
data H1 2018
Catalysts
+
ALX-1141/M6495 Osteoarthritis
Up to 8
programmesVariousImmuno-Inflammation
ADAMTS-5
Phase IIb
data H2 2018
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Caplacizumab – anti-vWF Nanobody
First-in-class potential for the treatment of aTTP
• Estimated annual market potential ~€800M1
• Feb 2017: filed in Europe for approval
• Jul 2017: received Fast Track designation from the FDA
• Phase III HERCULES topline results expected in late Q3 2017
• Phase I results in Japanese healthy volunteers expected in Q4 2017
• 2018: anticipated first launch in Europe and BLA submission in USA
• 2019: anticipated launch in USA
• Forecast peak sales of >€400M1
• Ablynx expected to lead commercialisation in USA, Canada and
Europe
• Orphan Drug Status (EU/USA) – Patent protection up to 2035
aTTP: acquired thrombotic thrombocytopenic purpura
1 USA, Canada, Europe and Japan
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Caplacizumab in aTTP
Phase III HERCULES and follow-up study on track
• 145 patients with aTTP recruited in just 20 months
• Last patient completed the HERCULES study in August
• Topline results expected in late Q3 2017
• >80% roll-over of eligible HERCULES patients into the 3-year follow-up study
Phase III topline data:
primary endpoint: time to confirmed platelet count normalisation
key secondary endpoints: composite efficacy endpoint; recurrence of aTTP during
study period; refractory aTTP; time to normalisation of organ damage markers
safety: summary of all (serious) adverse events
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Caplacizumab in aTTP
Preparations for launch
• Medical Science Liasons (MSLs) already in place in Germany, France and the
UK – additional recruitment ongoing
• Multiple presentations and publications at both the international and national
level
• Numerous interactions with national bodies to enable rapid availability of
caplacizumab to patients
• Launch of Ablynx sponsored TTP website – UnderstandingTTP.com
• Selection of key service partners
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ALX-0171 – inhaled anti-RSV Nanobody
Potential breakthrough for the treatment of RSV infections
• Multi-billion dollar market
• No widely used therapeutic available
• ALX-0171 – administered by inhalation
• Infants – Phase IIb RESPIRE study: – started in January 2017
– first 3 safety cohorts complete (36 infants) – positive DMC
recommendation
– parallel dose part initiated (144 infants)
– results anticipated in H2 2018
• Adults – preparations underway to file for regulatory
approval to start clinical development in RSV-infected
haematopoietic stem cell transplant patients
• Patent protection up to 2037
RSV: respiratory syncytial virus; DMC: Data Monitoring Committee
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ALX-0171 development plan
Key potential near term milestones
2017 2018
Phase IIb – ongoing hospitalised infants with RSV (N=180)
topline results
Phase II in Japanhospitalised infants with RSV
Phase IIRSV-infected haematopoietic
stem cell transplant patients
CTN
IND / CTA
CTN: Clinical Trial Notification; IND: Investigational New Drug; CTA: Clinical Trial Application
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Vobarilizumab – anti-IL-6R Nanobody
Potential novel treatment for RA and SLE
• Opportunity in multi-billion dollar markets
• RA:– very encouraging results from two Phase IIb RA studies
(N=~600)
– open-label extension study ongoing
• SLE– chronic multi-system inflammatory disease with large unmet
medical need
– Phase II study ongoing (N=~300); topline data in H1 2018
• We will wait for the SLE data and AbbVie’s opt-in
decision before deciding on our future strategy
RA: rheumatoid arthritis; SLE: systemic lupus erythematosus
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Vobarilizumab in SLE
* complement score omitted due to mechanism of action of vobarilizumab
Phase II STEADY study on trackR
AN
DO
MIS
AT
ION
1:1:1:1:1
Placebo
ALX-0061 dose 1, Q4W
312 subjects
ALX-0061 dose 2, Q4W
ALX-0061 dose 2, Q2W
ALX-0061 dose 3, Q2W
Primary endpoint at Week 24:
modified (m)BICLA* response
Secondary endpoints:
(m)BICLA, (m)SRI, (m)SLEDAI-2K
and BILAG over time; patient’s and
physician’s global assessment; flare
rate; corticosteroid reduction
Other assessments:
pharmacokinetics,
pharmacodynamics,
safety/tolerability, immunogenicity
• Subjects with moderate-to-severe active, seropositive SLE
• Worldwide, randomised, double-blind, placebo-controlled 48 week dose-range finding
study
• Patient recruitment completed in December 2016; topline data expected in H1 2018
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Vobarilizumab in SLE
Rationale for approach
Ball et al., Lupus 2013; Tackey et al., Lupus 2004; Mao et al., Clin Rheumatol. 2014; Wallace et al., Ann Rheum Dis 2017; Illei et al., Arthritis &
Rheumatism 2010
• IL-6 levels are elevated in patients with SLE
• Inhibition or knock-out of IL-6R in pre-clinical models had a strong impact on the
SLE phenotype
• Clinical studies targeting both IL-6 and IL-6R have shown encouraging
preliminary efficacy data in SLE, though safety appears to have presented
problems
• We have been able to learn from previous studies in designing the STEADY
trial, particularly with regard to enrollment criteria
• Vobarilizumab has shown in the RA studies that it targets IL-6R effectively and
has a favourable safety profile
Strategic partnerships
>€450M cash received and >€10Bn in potential milestones
plus royalties
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Strategic collaboration with Sanofi
*GBI research Dec 2015
Up to 8 programmes initially focused on immuno-inflammatory diseases
• Deal signed in July 2017
• Up to 8 different targets or target combinations
• €23M upfront payment received plus research funding
• Potential additional option exercise fees
• Up to €2.4Bn in potential future milestones plus royalties
+
Initial focus on immune-mediated inflammatory diseases
• Chronic disorders characterised by dysregulation of immune pathways
• Associated with significant morbidity, mortality and reduced quality of life
• Growing need for novel treatments to modify disease state
• Affect 5–7% of western populations*
• Market expected to grow to $74Bn in 2022*
IBD
RA
Psoriasis
COPD
Lupus
asthma
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Strategic partnerships
Progress year-to-date
• Co-discovery and co-development collaboration signed in November 2011
‒ €20M upfront and €15M milestone payments received so far
• Progress year-to-date
Merck KGaA accepted the pre-clinical package prepared by Ablynx for an anti-
ADAMTS-5 Nanobody in osteoarthritis in May 2017, triggering a €15M milestone
payment
• Merck KGaA plans to start the single ascending dose part of the Phase I
study in healthy volunteers in H2 2017
• Co-discovery and co-development collaboration signed in September
2008
‒ €10M upfront and €4.5M milestone payments received so far
• Progress year-to-date
Encouraging Phase Ib data with anti-IL-17A/F bi-specific Nanobody in patients with
moderate to severe psoriasis reported
• Merck KGaA partnered with Avillion to advance a potential Phase II study
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Strategic partnerships
Progress year-to-date
• Immuno-oncology collaboration signed in 2014 and expanded in 2015
‒ includes up to 17 programmes with a focus on multi-specific combinations
‒ €33M upfront and €6M milestone payments received to date
‒ currently ~40 FTEs fully funded at Ablynx
‒ up to €5.7Bn in potential future milestones plus royalties
• Progress year-to-date
Merck & Co., Inc., started an IND-enabling toxicology study with a bi-specific
Nanobody, triggering a €2.5M milestone payment
Merck & Co., Inc., completed a second in-vivo proof-of-concept study with a mono-
specific Nanobody
• Potential start of first clinical study in H1 2018
Outlook
Potential value enhancing events
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Outlook
Focus on sustainable value creation
• HERCULES Phase III data anticipated to be available in late Q3 2017
• Results from Phase I with caplacizumab in Japanese healthy volunteers planned
in Q4 2017
• Preparations for commercialisation of caplacizumab ongoing
• In Q4 2017, we expect to file for regulatory approvals
– to start a trial with ALX-0171 in Japanese infants infected with RSV
– to study the use of ALX-0171 in adults who have undergone a stem cell transplant and
have become infected with RSV
• The STEADY trial with vobarilizumab in SLE will report data in H1 2018
• The RESPIRE trial with ALX-0171 in RSV infected infants should report data in H2 2018
• Cash burn for full year 2017 is now forecast to be €65-75 million
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