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* Proof-of-concept in the Phase II TITAN study was achieved with significant
reductions in time to platelet count normalisation and recurrences while on
treatment with caplacizumab
* Based on these results, Ablynx is on track to file for conditional approval
of caplacizumab in Europe in the first half of 2017
* A confirmatory international Phase III study in patients with acquired TTP
is ongoing and will be used to support a BLA submission in 2018 in the USA
* Ablynx intends to lead the commercialisation of caplacizumab in Europe and
the USA

GHENT, Belgium, 11 February 2016 - Ablynx[Euronext Brussels: ABLX; OTC: ABYLY]

today announced that the results of the Company's worldwide Phase II TITAN
study[1]with caplacizumab for patients with acquired thrombotic
thrombocytopenic purpura (aTTP) have been published in today's issue of the
New England Journal of Medicine (NEJM).

"Caplacizumab has the potential to become an important new component in the
standard of care for patients with acquired TTP" saidProfessor Flora
Peyvandi, Principal Investigator for the TITAN study at IRCCS
Maggiore Hospital Foundation, University of Milan, Italy, and lead author of
the NEJM paper.
"The results from the Phase II TITAN study showed that caplacizumab acts
quickly to control the critical acute phase of the disease and protects
patients until immunosuppressive treatments take effect."

Dr Robert K. Zeldin, Chief Medical Officer of Ablynx, commented:
"The publication of the TITAN data in this high-impact clinical journal[2]is a
further validation of the potential of caplacizumab in the treatment of
acquired TTP. This publication is the culmination of over a decade of work by
Ablynx and its external collaborators. We are on track to file for
conditional approval of caplacizumab in Europe in 2017 and to complete
enrolment of the confirmatory Phase III study before the end of 2017. We look
forward to making caplacizumab available for patients with this devastating

About caplacizumab and the TITAN study results

Caplacizumab is a highly potent and selective bivalent anti-von Willebrand
Factor (vWF) Nanobody® that received Orphan Drug Designation in the USA and
EU in 2009. Caplacizumab inhibits the interaction between ultra-large vWF and
platelets by targeting the A1 domain of vWF. It thereby prevents platelet
aggregation and the formation of micro-clots during the acute, critical phase
of acquired TTP.

Caplacizumab's clinical effect was demonstrated in the Phase II TITAN study in
75 patients with aTTP:

* As indicated by a nearly 40% reduction in median time to platelet count
normalisation (p = 0.005). Treatment with caplacizumab reduced the use of
daily plasma exchange (PEX) and prevented further consumption of platelets
in microthrombi and small blood vessel occlusion.
* As shown by the low number of recurrences requiring re-initiation of daily
plasma exchange during treatment with caplacizumab (N=3) vs. placebo (N=11)

These results will serve as the basis for filing for conditional approval in
Europe in H1 2017. Caplacizumab could be the first drug specifically approved
for the treatment of acquired TTP.

More information on caplacizumab, including the NEJM paper, can be found on

About aTTP

aTTP is an ultra-rare, acute, auto-immune blood clotting disorder, affecting
up to 11 per million people worldwide. It has a sudden onset caused by
impaired activity of the ADAMTS13 enzyme (typically<10% of that in normal
plasma), leaving ultra-large vWF molecules un-cleaved (vWF is an important
protein involved in the blood clotting process). These ULvWF molecules
spontaneously bind to blood platelets, resulting in severe thrombocytopenia
(very low platelet count) and micro-clot formation in small blood vessels
throughout the body[3].

aTTP is associated with major morbidities in the brain (e.g. stroke), heart
and kidney and impacts life expectancy and quality of life. Mortality is high
at 10-20%[4], typically occurring within 2 weeks after initial diagnosis.
Moreover, about 36% of patients have recurrences[5]after treatment with the
current standard of care, which consists of daily PEX and
immune-suppressants, and these recurrences have the potential to cause
further organ damage and poorer longer term outcomes.

Phase II TITAN clinical study

The NEJM paper, titled "Caplacizumab Treatment for Acquired Thrombotic
Thrombocytopenic Purpura" (Peyvandiet al
., NEJM 2016: published 11 February 2016), reported data from the worldwide
Phase II TITAN clinical trial which was a single-blinded, randomised,
placebo-controlled study. In total, 75 patients were randomised on a 1:1
basis to active drug or placebo, with all patients receiving the current
standard of care. Those patients in the active drug treatment arm immediately
received an intravenous bolus dose of 10 mg caplacizumab and then a 10 mg
subcutaneous dose of the drug daily until 30 days had elapsed after the final
plasma exchange. Patients in the control arm received placebo at the same
time points.

The TITAN study was conducted at 56 study centres worldwide, with
investigators from countries including Italy, England, Switzerland, the USA
and Austria.

Phase III HERCULES clinical study

The worldwide Phase III HERCULES study is a multinational, double-blind,
placebo-controlled study evaluating the efficacy and safety of caplacizumab,
in conjunction with the standard of care, in patients with aTTP. The study is
expected to enrol 92 patients at clinical sites across 17 countries. The
primary endpoint is time to platelet count normalisation. Other clinically
relevant endpoints include: the prevention of recurrence of the presenting
TTP episode after stopping daily PEX; the effect on biomarkers of organ
damage; severe morbidity associated with tissue ischemia and; mortality.
Recruitment for this study is expected to be completed by the end of 2017,
followed by an anticipated BLA filing in the USA in 2018.

About Ablynx

Ablynxis a biopharmaceutical company engaged in the development ofNanobodies®,
proprietary therapeutic proteins based on single-domain antibody fragments,
which combine the advantages of conventional antibody drugs with some of the
features of small-molecule drugs. Ablynx is dedicated to creating new
medicines which will make a real difference to society. Today, the Company
has approximately40 proprietary and partnered programmesin development in
various therapeutic areas including inflammation, haematology,
immuno-oncology, oncology and respiratory disease. The Company has
collaborations with multiple pharmaceutical companies including AbbVie,
Boehringer Ingelheim, Eddingpharm, Genzyme, Merck&Co., Inc., Merck KGaA,
Novartis, Novo Nordisk and Taisho Pharmaceuticals. The Company is
headquartered in Ghent, Belgium. More information can be found

For more information, please contact


Dr Edwin Moses
t: +32 (0)9 262 00 07
m: +32 (0)473 39 50 68

Marieke Vermeersch
Associate Director Investor Relations
t: +32 (0)9 262 00 82
m: +32 (0)479 49 06 03

Follow us on Twitter @AblynxABLX

Ablynx media relations
Instinctif Partners:

Sue Charles, Daniel Gooch
London office
t: +44 (0)20 7866 7905

Belgium/Dutch and French language
Jim Rusagara
Brussels office
t: +32 (0)2 626 9500

[1]Top line data from the TITAN study were communicated inJune 2014
and were subsequently presented atASH 2014and ISTH 2015 (results frompost hoc analysisandADAMTS13 activity to guide treatment duration)
[2]The most recent (2014) impact factor for NEJM is 55.873, the highest among
general medical journals
[3]Veyradier, NEJM 2016: "von Willebrand Factor - A new target for TTP
[4]Allford et al, BJH 2003; Kremer Hovinga, Blood 2010; Benhamou,
Haematologica 2012
[5]George et al, EJB 2008

pdf format of the press release


This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Ablynx via Globenewswire


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