pdf version of the presentation

Full Year Results 2013 & Outlook
27 February 2014
Webcast presentation
Nanobodies® Creating better medicines
Participants on the call
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Dr Edwin Moses
Dr Antonin de Fougerolles
Chief Executive Officer
Chief Scientific Officer
Wim Ottevaere
Dr Dominique Tersago
Chief Financial Officer
Chief Medical 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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Agenda
Welcome and introduction
2013 - a transformational year!
Outlook 2014
Q&A
Conclusion
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2013 has been transformational for Ablynx
Business
Development
Clinical Trials
•
Strong Phase IIa results for
the anti-IL-6R Nanobody
•
Continued Phase II
recruitment for TTP with
caplacizumab (anti-vWF)
•
•
Merck Serono and BI
initiated Phase I trials
(indications: RA and
Alzheimer’s)
•
•
•
Started additional Phase I
studies with anti-RSV
Nanobody
AbbVie: deal for ALX-0061
worth up to $840M in
upfronts and milestones
plus double-digit royalties
Merck Serono: signed the
4th discovery collaboration
which could generate
>€100M in cash over the
next 6.5 years
Eddingpharm: licensing of
anti-RANKL Nanobody in
Greater China
Corporate
Development
•
Strengthened management
team and Board of
Directors
•
Raised €31.5M through a
private placement of new
shares
•
17 million VC-owned
shares successfully placed
•
Free float increased from
53% at the end of 2012 to
85% today
Evolving into one of Europe’s leading
biopharmaceutical companies
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Financial performance
Nanobodies® Inspired by nature
Financial results 2013
FY 2013 (€M)
FY 2012 (€M)
% change
35.9
26.7
34%
R&D income
33.2
25.6
30%
Grants
2.8
1.1
155%
(53.7)
(56.3)
(4%)
R&D
(43.7)
(46.9)
(7%)
G&A
(10.0)
(9.4)
6%
0.1
(0.2)
Operating result
(17.7)
(29.8)
Net financial result
(1.8)
1.3
Net result
(19.5)
(28.5)
Net cash inflow (burn)
137.6 (1)
(21.1)
Cash at December 31st
200.4 (2)
62.8 (3)
Revenues
Operating expenses
Other operating
income/(expenses)
(1) incl.
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41%
32%
219%
proceeds from private placement (€31.5M) and AbbVie upfront payment ($175M)
(2) incl
€2.3M restricted cash
(3)
incl €2.7M restricted cash
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Pipeline update
Nanobodies® Inspired by nature
Fully partnered
50% CoCo
Fully owned
Broad pipeline – current status
Therapeutic area
Product name
Target
Haematology
Inflammation/
Immunology/
Infection
caplacizumab
vWF
ozoralizumab
Various
TNFα
Oncology
Various
Various
Bone disorders
ALX-0141
RANKL
RSV
Pulmonology
ALX-0171
Various
Various
Oncology
ALX-0751
Inflammation/
Immunology
NA
NA
NA
Oncology/Neurology
Immunology
Various
Various
Immunology
ALX-0761
ALX-0061
Bone disorders
ALX-0141
Neurology
BI 1034020
NA
Oncology
NA
Pulmonology
NA
Various
NA
NA
Immuno-oncology
Various
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Discovery Pre-clinical Phase I
Phase II
Phase III
Filing
ex Greater China
Potential to evolve into at least 4 co-co programmes
IL-17F/IL-17A
IL-6R
RANKL
in Greater China
Validated targets (clinic)
1st in class
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Phase II programmes – key events
Feb 2013
Sept 2013
Jan 2014
Anti-IL-6R (ALX-0061) – monovalent Nanobody with T1/2 extension
• Phase II proof-of-concept achieved in patients with RA in Feb 2013
• global exclusive licensing deal signed with AbbVie in Sept 2013
Anti-vWF (caplacizumab) – bivalent Nanobody
• Phase II recruitment halted in Jan 2014
• Proof-of-concept data analysis in H1 2014
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ALX-0061 – compelling POC results in RA patients
• Treatment showed strong efficacy and was well tolerated at all doses
• No increase of adverse events upon extension of treatment
• No anti-drug antibodies were detected
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ALX-0061 – global licensing deal with AbbVie
Ablynx received $175 million upfront; $665 million of potential total
milestones plus double-digit royalties
Ablynx will perform and fund subcutaneous Phase I study (2014) and Phase
II studies in RA and SLE (start in 2015)
AbbVie will pay a fee if they exercise the right to license ALX-0061 after the
completion of Phase II studies
• Phase II results in RA expected in 2016
• Phase II results in SLE expected in 2018
AbbVie is responsible for Phase III development, registration and global
commercialisation
Ablynx retains option to co-promote in Benelux
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Caplacizumab – 1st in class opportunity in acquired TTP
As a result of slow recruitment it was decided to stop the TITAN study and
analyse the data early
Potential Phase II POC results in June 2014
Positive data would allow further exploration of partnering opportunities
Phase III trial could start in 2015
• planning a simpler study protocol compared to the Phase II trial
Orphan drug status for the treatment of TTP in the US and Europe
We believe approximately 10,000 patients present each year
• there is no specific drug treatment available, only multiple plasma exchanges
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Phase I programmes – key events
Anti-IL-17A/F (ALX-0761) – bi-specific Nanobody with T1/2 extension
• Phase I study initiated by Merck Serono in June 2013
June 2013
July 2013
Oct 2013
Anti-RSV (ALX-0171) – 1st inhaled trivalent Nanobody
• additional Phase I studies initiated in July 2013
Anti-RANKL (ALX-0141) – bivalent Nanobody with T1/2 extension
• exclusively licensed to Eddingpharm for Greater China in Oct 2013
Alzheimer’s (BI 1034020)
• Phase I study initiated by Boehringer Ingelheim in Oct 2013
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Pre-clinical programmes – update post year-end 2013
Anti-RSV (ALX-0171) – 1st inhaled trivalent Nanobody
• encouraging pre-clinical POC results in neonatal lamb model (treatment at viral peak)
- daily inhalation of ALX-0171 resulted in a strong reduction of viral titres in lung fluid and
a marked reduction in inflammation in the lungs and viral lesions
- treatment with ALX-0171 also appeared to significantly improve various clinical signs
and symptoms such as behavioural activity and general well-being
M e a n v ir a l t it e r s in B A L f lu i d
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0
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Pre-clinical programmes – update post year-end 2013
Anti-IgE (ALX-0962) – bivalent Nanobody
• novel dual mode of action demonstrated in pre-clinical studies
- IgE neutralisation and displacement
• however, after completing pre-clinical studies and business case analysis we do not
believe we have a material competitive advantage over the current standard of care
(Xolair®)
• strategic decision to stop further development and invest resources elsewhere
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Value creation – clinical data expected from patient studies
ALX-0061 Phase
IIb sc (anti-IL-6R) in
patients with RA
Licensed to AbbVie
2013
Caplacizumab
Phase II (anti-vWF)
in patients with
acquired TTP
clinical
ALX-0061 Phase IIa Wholly-owned
asset
iv (anti-IL-6R)
in patients with RA 
ALX-0171 Phase
I/II (anti-RSV) in
infants with RSV
infection
ALX-0761 Phase
IIa (anti-IL-17A/F)
in patients with RA
Wholly-owned clinical
asset
Licensed to Merck
Serono
Two partnered
Phase I/II
programmes in
cancer
Licensed to AbbVie
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Partnerships update
Nanobodies® Inspired by nature
Broad platform exploitation and cash generation
•
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 ongoing) and a collaboration in Alzheimer’s (Phase I)
•
4 discovery 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; cancer immunotherapy
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
>€320M in non-dilutive cash received from collaborators to date
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Merck & Co – new alliance in immuno-oncology (Feb. 2014)
New alliance, which builds on the existing ion channel collaboration in
neurology:
• opportunity in the emerging field of cancer immunotherapy
• focused on the discovery and development of Nanobody-candidates against
immune checkpoints
• exploiting the ability to rapidly generate multi-specific Nanobodies
The deal comprised: €20 million upfront; €10.7 million in research
funding; €1.7 billion in potential total milestones plus royalties
Following a three-year research term at Ablynx, Merck & Co will be
responsible for further development of the Nanobody-candidates, product
registration and commercialisation
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Rapid and proven formatting to make multi-specifics
Pardoll – Nature Rev Cancer 2012
Rapid formatting of
monovalent building
blocks
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Proven
experience
Interfere with multiple
pathways at once
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Outlook
Nanobodies® Inspired by nature
Expected news flow for 2014
Expected
Clinical Data
Start of
Clinical Trials
Business
Development
•
Potential Phase II POC
results with caplacizumab in
acquired TTP
•
Start of Phase II paediatric
study with ALX-0171 (antiRSV)
•
Potential milestone
payments from on-going
collaborations
•
Phase I results from sc
study with ALX-0061 (antiIL-6R)
•
•
Potential additional
collaborative deals
•
Phase I results from safety
and PK studies with ALX0171 (anti-RSV)
Preparation for start of
Phase II RA and SLE
studies with ALX-0061
(anti-IL-6R) in 2015
•
Phase I results from BI for
Nanobody for use in
Alzheimer’s disease
Start of Phase I with ALX0061 (anti-IL-6R) sc
formulation
•
Start of up to four partnered
Phase I studies with
Nanobodies
•
•
Phase I results from Merck
Serono for ALX-0761 (antiIL-17A/F)
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
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Q&A
Nanobodies® Inspired by nature
Full Year Results 2013 & Outlook
27 February 2014
Webcast presentation
Nanobodies® Creating better medicines

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