ABLYNX'S FIRST-IN-CLASS WHOLLY-OWNED ANTI-vWF NANOBODY, CAPLACIZUMAB, MAY HAVE THE POTENTIAL TO REDUCE MORBIDITY AND MORTALITY ASSOCIATED WITH ACQUIRED TTP
(Thomson Reuters ONE) -
Additional data from post-hoc analyses of the Phase II TITAN study to be
presented at the European Congress on Thrombosis and Haemostasis (ECTH)
GHENT, Belgium, 29 September 2016 - Ablynx [Euronext Brussels: ABLX; OTC: ABYLY]
announced that it will today present additional data from post-hoc analyses of
the Phase II TITAN study of its wholly-owned Nanobody®, caplacizumab, in
patients with acquired thrombotic thrombocytopenic purpura (aTTP), at the first
European Congress on Thrombosis and Haemostasis (ECTH), being held from 28-30
September 2016 in The Hague, The Netherlands. Shortly after the conference, the
presentation will be available on the Ablynx website under the R&D portfolio
section.
Acquired TTP is an acute, ultra-rare, life-threatening blood clotting disorder
in which uncontrolled platelet aggregation and microclot formation cause small
blood vessel occlusions throughout the body[1], resulting in thrombotic
complications and widespread organ damage[2]. Despite the current standard-of-
care treatment of daily plasma exchange (PEX) and immunosuppression, episodes of
aTTP are still associated with a mortality rate of up to 20%, with most deaths
occurring within 30 days of diagnosis[3]. Furthermore, patients are at risk of
acute thromboembolic complications (e.g. stroke, venous thrombosis and
myocardial infarction) and of recurrence of disease. In addition, some patients
are refractory to therapy[4], which is associated with a very poor prognosis for
survival of an acute episode of aTTP. Data from the French Reference Center for
Thrombotic Microangiopathies showed that 50% of patients who died from an acute
episode were refractory to treatment[5].
Post-hoc analyses of the Phase II TITAN study data were performed to assess the
impact of caplacizumab on a composite endpoint of major thromboembolic
complications and aTTP-related mortality, as well as on refractoriness to
standard treatment. The results demonstrate that a clinically meaningful lower
proportion of subjects treated with caplacizumab experienced one or more major
thromboembolic events, or died, as compared to placebo (11.4% versus 43.2%). In
addition, fewer caplacizumab-treated patients, compared to those who received
placebo, were refractory[6] to treatment (5.7% versus 21.6%; and 0% versus
10.8%, respectively depending on the definition of refractoriness(4)). There
were two deaths in the placebo group and both those patients were refractory to
treatment; no deaths were reported in the caplacizumab group.
A Phase III confirmatory study of caplacizumab is currently ongoing which
includes prospectively defined assessments of these clinically relevant
endpoints.
Professor Flora Peyvandi, Principal Investigator for the TITAN study at IRCCS
Maggiore Hospital Foundation, University of Milan, Italy, commented: "Acquired
TTP is a very severe disease with high unmet medical need. Any new treatment
option would need to act fast to immediately inhibit the formation of micro-
clots in order to protect the patient during the acute phase of the disease and
so have the potential to avoid the resulting complications. The topline results
and the subsequent post-hoc analyses of the Phase II TITAN data demonstrate that
caplacizumab has the potential to reduce the major morbidity and mortality
associated with acquired TTP, and confirm our conviction that it should become
an important pillar in the management of acquired TTP."
About caplacizumab
Caplacizumab is a highly potent and selective bivalent anti-von Willebrand
Factor (vWF) Nanobody that received Orphan Drug Designation in the United States
and Europe in 2009. Caplacizumab blocks the interaction of ultra-large vWF
multimers (ULvWF) with platelets and, therefore, has an immediate effect on
platelet aggregation and the ensuing formation and accumulation of the
microclots that cause the severe thrombocytopenia and organ and tissue damage in
aTTP. This immediate effect protects the patient from the manifestations of the
disease while the underlying disease process resolves.
The efficacy and safety of caplacizumab in conjunction with the standard of care
(PEX) were evaluated in the Phase II TITAN study in 75 patients with aTTP.
Caplacizumab was well-tolerated and the primary endpoint of reduction in time to
platelet normalisation was met (p=0.005). Treatment with caplacizumab resulted
in a nearly 40% reduction in time to platelet count normalisation as compared to
placebo (i.e., a faster reversion of thrombocytopenia with consequent reduced
use of PEX). Moreover, during treatment, caplacizumab reduced aTTP recurrences
by 71% compared to placebo when administered as an adjunct to standard of
care[7].
These Phase II results will serve as the basis for filing for conditional
approval of caplacizumab in Europe in early 2017. The confirmatory Phase III
HERCULES study is currently ongoing and will support the BLA filing in the
United States. Results from this Phase III study are expected by the end of
2017.
Caplacizumab has the potential to be the first therapeutic specifically approved
for the treatment of acquired TTP.
About aTTP
aTTP is an acute, ultra-rare, life-threatening, 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 ULvWF molecules uncleaved (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, leading to
thrombotic complications and widespread organ damage.
Up to 20% of patients die from an initial aTTP episode with most deaths
occurring within 30 days of diagnosis. In addition to the acute risks of the
disease, patients experiencing an episode of aTTP may suffer long-term
consequences such as cognitive deficits, depression, and arterial
hypertension[8], and are at risk for recurrence. The recurrence rate ranges from
10-84%[9] and typically occur within 1-2 years[10], but recurrences have been
reported up to 30 years after the initial episode[11].
About Ablynx
Ablynx is a biopharmaceutical company engaged in the development of Nanobodies®,
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 than
45 proprietary and partnered programmes in 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 on
www.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(at)ablynx.com
Marieke Vermeersch
Director IR & Corporate Communications
t: +32 (0)9 262 00 82
m: +32 (0)479 49 06 03
e: marieke.vermeersch(at)ablynx.com
Follow us on Twitter (at)AblynxABLX
Ablynx media/analyst relations
FTI Consulting:
Julia Phillips, Brett Pollard, Mo Noonan, Matthew Moss
t: +44 20 3727 1000
e: ablynx(at)fticonsulting.com
--------------------------------------------------------------------------------
[1] Veyradier, NEJM 2016: "von Willebrand Factor - A new target for TTP
treatment?"
[2] Scully et al, Br J Hem 2012; Sarode et al, J Clin Apher 2014; Chaturvedi et
al, Am J Hem 2013
[3] Benhamou, Y.et al., Haematologica 2012
[4] Defined as: 'failure of platelet response after 7 days despite daily plasma
exchange treatment' or 'absence of platelet count doubling after 4 days of
standard treatment, and LDH>upper limit of normal
[5] Benhamou, Y. et al, Journal of thrombosis and haemostasis 2015
[6] Peyvandi et al, notes to editor NEJM 2016
[7] Press release June 2014; Manuscript in the NEJM, Feb 2016; presentation at
EHA 2016
[8] Deford et al, Blood 2013
[9] Thejeel et al, American journal of hematology 2016
[10] Kremer Hovinga et al, Blood 2010
[11] Falter et al, Hamostaseologie 2013
pdf format of the press release:
http://hugin.info/137912/R/2045290/763966.pdf
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq 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
Unternehmensinformation / Kurzprofil:
Datum: 29.09.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 497742
Anzahl Zeichen: 10246
contact information:
Town:
Ghent/Zwijnaarde
Kategorie:
Business News
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