ablynx financial presentation half year results 2014
DESCRIPTION
Webcast presentation Ablynx financial results for the first six months ending June 2014TRANSCRIPT
Nanobodies® -
Inspired by nature
Ablynx Half-Year Results 2014
28 August 2014
Webcast presentation
www.ablynx.com
Participants on the call
2
Dr Dominique Tersago
Chief Medical Officer
Dr Antonin de Fougerolles
Chief Scientific Officer
Dr Edwin Moses
Chief Executive Officer
Wim Ottevaere
Chief Financial Officer
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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. 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.
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H1 2014 results – agenda
Welcome and introduction
Strong first half of the year
Outlook
Q&A
Conclusion
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Significant progress during H1 2014
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• Clinical proof-of-concept (POC) achieved with anti-
vWF Nanobody, caplacizumab, in patients with
acquired TTP
• Pre-clinical POC achieved and positive Phase I
results from 2 inhalation studies with ALX-0171 (anti-
RSV)
• Initiated bioavailability study with sc formulation of
ALX-0061 (anti-IL-6R)
• Merck Serono completed SAD Phase I study in
healthy volunteers and initiated Phase Ib study in
patients with psoriasis with the anti-IL-17A/F bi-
specific Nanobody (ALX-0761)
• Opt-out option exercised for ALX-0751; Merck Serono
now solely responsible for further development
(Ablynx to receive potential milestones and royalties)
• Development of ALX-0962 (anti-IgE) and BI 1034020
(anti-Abeta) stopped
• Signed major research collaboration and
licensing agreement with Merck & Co in
immuno-oncology with €20M upfront, €10.7M
research funding, up to €1.7bn in milestones
and royalties
• Raised €41.7M through a private placement
of new shares; Fidelity (US) became a >3%
shareholder
• Free float increased to ~90%
• Awarded €2.1M grant from IWT (in August) to
support development of novel Nanobodies for
ocular applications
• Won Transaction of the Year at the 2014
European Mediscience Awards
Partnering & Corporate Product Pipeline
Nanobodies® -
Inspired by nature
Financial performance
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H1 2014 – financial summary
7
H1 2014 (€M) H1 2013 (€M) % change
Revenues 22.2 12.9 72%
R&D income 21.8 12.0 82%
Grants 0.4 0.9 (56%)
Operating expenses (29.8) (23.8) 25%
R&D (24.5) (19.3) 27%
G&A (5.3) (4.5) 18%
Operating result (7.6) (10.9) 30%
Net financial result 1.3 0.4 225%
Net result (6.3) (10.5) 40%
Net operational cash burn (3.9) (21.6) (82%)
Cash at 30th June 196.0 (1) (2) 72.0 (3) (4) 172%
(1) not including proceeds from private placement announced on 30th June 2014 (€41.7M raised) (2) including €2.0M restricted cash (3) including proceeds from private placement announced on 28th February 2013 (€31.5M raised) (4) including €2.3M restricted cash
Nanobodies® -
Inspired by nature
Pipeline update
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Broad pipeline – current status
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Immunology/
Inflammation
Fu
lly o
wn
ed
Therapeutic area Product name Target
Inflammation/
Immunology/
Infection
Haematology
Oncology
Pulmonology
Discovery
ALX-0061
Pre-clinical Phase I Phase II Phase III
IL-6R
caplacizumab vWF
ALX-0171
Various
Inflammation/
Immunology
Neurology
Various
ozoralizumab TNFα
ALX-0141 RANKL
Pulmonology
ALX-0761
NA
NA
NA
NA
NA
RSV
Co
Co
NA
NA
Various
Various
Various
Various
Fu
lly p
art
nere
d
NA
Oncology/Neurology
Immunology Potential to evolve into at least 4 co-co programmes
Bone disorders in Greater China
IL-17F/IL-17A
Various
Various
Immuno-oncology Various
Validated targets (clinic)
1st in class
Ocular NA
Oncology ALX-0751
Various
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Phase II programmes – caplacizumab
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June 2014
Anti-vWF (caplacizumab) – bivalent Nanobody – wholly-owned
• Phase II proof-of-concept achieved in patients with acquired TTP
• preparations to start a Phase III study in 2015 initiated
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Caplacizumab – Phase II TITAN design and schedule
Random
isation
Primary endpoint: time to confirmed normalisation of
platelet count
Secondary endpoints: plasma exchange frequency and volume;
relapse; exacerbations; mortality; major clinical
events (stroke, MI, organ dysfunction); recovery
from signs/symptoms.
1:1
PEX
PEX
X caplacizumab
1 year follow-up
1 year follow-up
Inclusion criteria:
subjects with
acquired TTP
requiring plasma
exchange (PEX)
Exclusion criteria:
• severe infection / sepsis
• pregnancy
• bone marrow
transplantation
• disseminated
intravascular
coagulation
• known congenital TTP
Long-term endpoints:
relapse; non focal neurological
symptoms..
Target – 110 subjects
Actual – 75 patients
placebo
30 days
30 days 30 days
30 days
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TITAN primary endpoint
N = 36 N = 39
Overall Hazard Rate Ratio for Caplacizumab vs. Placebo
(95% CI), N = 75 2.197 (1.278, 3.778)
Stratified Log-rank Test p-value 0.013
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Caplacizumab Placebo
Median days to confirmed platelet response – subjects with
no prior plasma exchange (95% CI)
3.00 (2.74, 3.88)
N = 34
4.92 (3.21, 6.59)
N = 35
25th & 75th percentile 2.72 & 4.31 3.01 & 11.37
Median days to confirmed platelet response – subjects with
one prior plasma exchange (95% CI)
2.44 (1.92, 2.97)
N = 2
4.31 (2.91, 5.68)
N = 4
25th & 75th percentile 1.92 & 2.97 3.37 & 5.23
The group of patients treated with caplacizumab in conjunction with the standard of care
achieved confirmed platelet normalisation at more than twice the rate of the group
receiving the standard of care plus placebo
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TITAN key secondary endpoints
Caplacizumab
N = 36
Placebo
N = 39
Exacerbations up to 30 days after end of daily plasma
exchange for the complete population 3 (8%) 11 (28%)
Complete remission up to 30 days after end of daily plasma
exchange as measured by confirmed platelet response and
absence of exacerbation
29 (81%) 18 (46%)
Proportion of subjects with exacerbation and/or relapse
at 1 month follow-up after study drug treatment was completed 13 (36.1%) 13 (33.3%)
Deaths 0 2
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These secondary endpoints illustrate the potential protective effect of caplacizumab
treatment in the acute phase of TTP
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Caplacizumab – next steps
Complete full analysis of TITAN study and consult with KOLs and regulatory
authorities
Complete a Phase I trial to demonstrate bioequivalence between the liquid and
lyophilised formulations
Perform market access and pricing study
Continue preparations to start a Phase III study in 2015
Continue to evaluate various partnering and commercialisation options
Potential presentation of complete Phase II data set at ASH conference
(Dec 2014)
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In 2015, caplacizumab will be the first Nanobody to enter Phase III clinical
development
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anti-IL-6R (ALX-0061) – monovalent half-life extended
• initiated bioavailability Phase I study with subcutaneous formulation
• preparations well underway for Phase II studies in RA and SLE in 2015
Phase I programmes – key events
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anti-RSV (ALX-0171) – inhaled trivalent
• positive results from 2 Phase I studies in healthy
volunteers and subjects with hyper-reactive airways
• encouraging results from a neonatal lamb model
April 2014 May 2014 July 2014
anti-IL-17A/F (ALX-0761) – bi-specific half-life extended
• Phase I SAD study in healthy volunteers completed by Merck Serono
• Phase Ib study initiated by Merck Serono in patients with psoriasis
anti-Abeta (BI 1034020) – biparatopic half-life extended
• programme stopped by Boehringer Ingelheim
Nanobodies® -
Inspired by nature
Partnerships update
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Broad platform exploitation and cash generation
>€330M in non-dilutive cash received from collaborators to date
~€3Bn in potential future milestones plus royalties
• Global licensing deal with AbbVie for ALX-0061 (anti-IL-6R) in RA and SLE: $175M upfront and
total potential value of $840M plus royalties
• Strategic discovery alliance with Boehringer Ingelheim (8 pre-clinical programmes on-going)
• 4 deals with Merck Serono: 10 programmes (1 Phase I) on-going in inflammation, immunology,
oncology, immuno-oncology, neurology and osteoarthritis
• 2 discovery deals with Merck & Co: ion channel deal in neurology; immune-onco deal with €20M
upfront, €10.7M research funding and total potential milestones of up to €1.7bn plus royalties
• Licensing deal with Eddingpharm in Greater China for ALX-0141 (anti-RANKL) in bone
disorders
• Target based discovery deal with Novartis
Nanobodies® -
Inspired by nature
Outlook
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News flow in 2014
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• Phase I results from safety
and PK studies with ALX-
0171 (anti-RSV)
• Phase II results caplacizumab
in acquired TTP
• Completion SAD Phase I in
healthy volunteers with ALX-
0761 (Merck Serono)
• Phase I results from s.c.
study with ALX-0061 (anti-IL-
6R)
• Phase I results from bio-
equivalence study with
caplacizumab (anti-vWF)
• Potential additional
collaborative deals
• Continue discussions with
potential partners in a
number of areas
• Start of Phase I study with
s.c. ALX-0061 (anti-IL-6R)
• Start of Phase Ib study with
ALX-0761 in patients with
psoriasis (Merck Serono)
• Start Phase I/II infant study
with ALX-0171 (anti-RSV)
• Start of bio-equivalence
study with caplacizumab
(anti-vWF)
• Preparation to start Phase II
in RA and SLE with ALX-0061
and Phase III with
caplacizumab in 2015
Business
development
Completion of
clinical trials
Start of
clinical trials
3 trial readouts achieved
2 readouts remaining
3 clinical trials initiated
1 clinical trial to be initiated
cash generative
partnerships
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Value creation – clinical data expected from patient studies
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ALX-0061 Phase IIb s.c. (anti-IL-6R) in subjects with RA
Licensed to AbbVie (worldwide)
ALX-0761 Phase Ib (anti-IL-17A/F) in subjects with psoriasis
Licensed to Merck Serono (worldwide)
ALX-0171 Phase I/II (anti-RSV) in infants with an RSV infection
Wholly-owned clinical asset
caplacizumab Phase II (anti-vWF) in subjects with acquired TTP
Wholly-owned clinical asset
caplacizumab Phase III (anti-vWF) in subjects with acquired TTP
Wholly-owned clinical asset
ALX-0171 Phase II (anti-RSV) in infants with an RSV infection
Wholly-owned clinical asset
ALX-0141 Phase I/II results from patient study in China
Licensed to Eddingpharm (Greater China)
Nanobodies® -
Inspired by nature
Q&A
Nanobodies® -
Inspired by nature
Ablynx Half-Year Results 2014
28 August 2014
Webcast presentation