Du är här

2015-09-29

Ablynx: ABLYNX INITIATES A MULTINATIONAL PHASE III STUDY WITH CAPLACIZUMAB IN PATIENTS WITH ACQUIRED TTP, A RARE BLOOD CLOTTING DISORDER

* Caplacizumab is a first-in-class anti-von Willebrand factor (vWF) Nanobody®
in development for the treatment of acquired thrombotic thrombocytopenic
purpura (TTP)
* Acquired TTP is an acute, potentially life-threatening ultra-rare blood
clotting disorder with a high unmet medical need, that affects about 11 per
million people worldwide
* Caplacizumab demonstrated promising clinical efficacy in patients with
acquired TTP in the Phase II TITAN study[1]
* Ablynx is on track to file for conditional approval of caplacizumab in
Europe in 2017 for the treatment of acquired TTP

GHENT, Belgium, 29 September 2015 - Ablynx[Euronext Brussels: ABLX; OTC:
ABYLY]

today announced the initiation of a multinational, double-blind
placebo-controlled Phase III "HERCULES" study evaluating efficacy and safety
of caplacizumab, its wholly-owned anti-vWF Nanobody, in acquired TTP. The
study is expected to enrol 92 patients at clinical sites across 17 countries.

Acquired TTP is an ultra-rare acute blood clotting disorder that leads to the
formation of microvascular thrombosis (blood clots in small blood vessels)
and organ damage throughout the body, including the brain and the heart.
Mortality remains high at 10-20% and about 36% of patients suffer from
relapses after initial treatment even with the current standard-of-care,
which consists of plasma exchange (PE) plus immune-suppressive treatment. In
addition, the organ damage caused by a TTP episode may result in poor longer
term outcomes.

There remains a high unmet medical need to immediately inhibit the formation
of microvascular thrombi, thereby reducing the risk of further organ damage.
Maintenance of this platelet-protective effect is required until the
underlying auto-immune activity has been resolved. Caplacizumab is being
developed to address this unmet need and its clinical effect has been
demonstrated in the Phase II TITAN study.

The Phase III "HERCULES" study will evaluate the efficacy and safety of
caplacizumab in patients with acquired TTP when administered in addition to
the standard-of-care. The primary endpoint is time to platelet count
normalisation, a measure of prevention of further microvascular thrombosis.
Other clinically relevant endpoints include the prevention of recurrence of
the presenting TTP episode after stopping daily PE, the effect on biomarkers
of organ damage, severe morbidity associated with ischemia, and the mortality
rate.

Dr Edwin Moses, CEO of Ablynx, commented:

"The ability of caplacizumab to rapidly inhibit the formation of small blood
clots, resulting in the more rapid restoration of normal platelet counts and
an important reduction in exacerbations, was well demonstrated in the Phase
II TITAN study. Based on the clinical effect seen in this TITAN study, we are
planning to submit caplacizumab for conditional approval to the European
Medicines Agency (EMA) in 2017. We now look forward to enrolling 92 patients
into our Phase III "HERCULES" study which we plan to have completed by the
end of 2017 to support a BLA filing in the United States in 2018. In parallel
with the clinical development and regulatory preparations, we are committed
to preparing to lead the commercialisation of caplacizumab in Europe and the
United States to make this product available for patients suffering from this
potentially life-threatening disorder."

About thrombotic thrombocytopenic purpura (TTP)

TTP exists in two forms: a congenital and an acquired form, with the latter
accounting for>90% of the patients. The condition is characterised by severe
thrombocytopenia (low blood platelet count), haemolytic anaemia (abnormal
break down of red blood cells) and signs and symptoms of tissue ischemia
(insufficient blood supply to organs) including stroke or myocardial
infarctions. The ischemic damage may result in both acute complications as
well as poor longer term outcomes. In the majority of patients, it is an
autoimmune condition where auto-antibodies are generated to the enzyme
ADAMTS13, which is responsible for ultra large vWF (ULvWF) cleavage. As a
result of impaired ADAMTS13 activity (typically<10% than in normal plasma),
these ULvWF molecules spontaneously bind to platelets, resulting in ULvWF
mediated platelet string formation in the small blood vessels.

About caplacizumab

Caplacizumab is a highly potent and selective bivalent anti-vWF Nanobody that
received Orphan Drug Designation in the US and EU in 2009. It could be the
first drug specifically approved for the treatment of acquired TTP.

Caplacizumab inhibits the interaction between vWF and platelets by targeting
the A1 domain of vWF and thus has the potential to immediately block the
ULvWF mediated platelet interactions and the formation of the string-like
clots in the blood of patients with acquired TTP.

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 more than30 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 Serono, Novartis and Taisho
Pharmaceutical Co., Ltd. The Company is headquartered in Ghent, Belgium. More
information can be found onwww.ablynx.com.

For more information, please contact

Ablynx:

Dr Edwin Moses
CEO
t: +32 (0)9 262 00 07
m: +32 (0)473 39 50 68
e: edwin.moses@ablynx.com

Marieke Vermeersch
Associate Director Investor Relations
t: +32 (0)9 262 00 82
m: +32 (0)479 49 06 03
e: marieke.vermeersch@ablynx.com

Follow us on Twitter @AblynxABLX

Ablynx media relations
Instinctif Partners:

Sue Charles, Daniel Gooch
London office
t: +44 (0)20 7866 7905
e: ablynx@instinctif.com

Belgium/Dutch and French language
Jim Rusagara
Brussels office
t: +32 (0)2 626 9500
e: ablynx@instinctif.com
---------------------------------------

[1]Phase II study results published inJune 2014
http://hugin.info/137912/R/1793634/617554.pdf
; results from the post-hoc analysis presented atASH 2014
http://hugin.info/137912/R/1878224/662002.pdf
andISTH 2015

pdf format of the press release
http://hugin.info/137912/R/1954990/711633.pdf

---------------------------------------

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

HUG#1954990

Författare Hugin

Tala om vad ni tycker

Tala om vad ni tycker

Ni är just nu inne på en betaversion av nya aktiespararna. Lämna gärna feedback på vad ni tycker i formuläret nedan.