ADDITIONAL CLINICAL DATA FROM THE PHASE II STUDY WITH ABLYNX'S ANTI-vWF NANOBODY, CAPLACIZUMAB, PRESENTED AT THE AMERICAN SOCIETY OF HEMATOLOGY ANNUAL MEETING
(Thomson Reuters ONE) -
* Caplacizumab is a potential first-in-class therapeutic with Orphan Drug
status
* Caplacizumab, in addition to plasma exchange and immunosuppressive therapy,
has the potential to become an important new treatment for acquired
thrombotic thrombocytopenic purpura (TTP)
* Additional data suggest that caplacizumab prevents consumption of platelets,
thereby protecting patients from further formation of microvascular thrombi
* Strong Phase II clinical proof-of-concept data published in June 2014
Additional Phase II results will be discussed during a webcast on Thursday 11
December at 15h CET, 9 am EST
The webcast may be accessed on the home page of Ablynx's website at
www.ablynx.com or by clicking here
To participate in the Q&A, dial +32(0)2 402 30 92 with confirmation code 7878101
GHENT, Belgium, 8 December 2014 - Ablynx [Euronext Brussels: ABLX] today
announced additional clinical results, including some post hoc analyses, from
the worldwide Phase II TITAN trial with its anti-von Willebrand Factor (vWF)
Nanobody®, caplacizumab, in patients with acquired thrombotic thrombocytopenic
purpura (TTP). The results were presented today (abstract #229) by Professor
Peyvandi, Principal Investigator for the TITAN trial, at the American Society of
Hematology (ASH) Annual Meeting in San Francisco, CA.
TTP is a rare disorder of the blood coagulation system that causes formation of
microthrombi that block the small blood vessels leading to tissue ischemia.
The results of the Phase II study suggest the potential protective effect of
caplacizumab against microvascular thrombosis and related tissue ischemia during
the acute phase of a TTP episode and further demonstrate that caplacizumab is
generally well tolerated, with an increase in mild bleeding events which were
readily managed and did not require vWF or Factor VIII substitution.
The majority of patients had very low platelet counts at the start of the study,
had experienced their first TTP episode and had ADAMTS13[1] activity below 10%
(a measure of the underlying disease activity). All patients received the
standard of care for treating acquired TTP, i.e. daily plasma exchange (PE), and
the majority of patients also received immunosuppressive therapy
(corticosteroids).
Only 8% (n=3) of caplacizumab treated patients experienced an exacerbation while
on treatment as compared to 28% (n=11) of patients on placebo. It is however
important to note that 22% (n=8) of caplacizumab patients experienced a relapse
event in the first month after stopping caplacizumab treatment, with all but one
of those patients having ADAMTS13 activity below 10% around the time when drug
treatment was stopped. This suggests that, in order to maintain the Nanobody's
protective effect, some patients may require longer treatment with caplacizumab
until their underlying disease is resolved.
Exploratory analysis of biomarkers of tissue damage in these TTP patients
illustrated the detrimental consequences of microvascular thrombosis in TTP,
with 49% of all patients at the start of the study having an above normal value
of Troponin I or T, markers of cardiac injury. Similar observations have been
reported in the literature[2]. Importantly, in patients who were treated with
caplacizumab, these biomarkers returned more rapidly to the normal range,
suggesting that the inhibition of microthrombi formation by caplacizumab may
result in reduced cardiac injury.
Commenting on the full analysis of the worldwide Phase II TITAN trial with
caplacizumab, Professor Flora Peyvandi, Principal Investigator, at IRCCS
Maggiore Hospital, University of Milan, Italy said:
"The detailed findings of this study confirms our conviction that caplacizumab
has the potential to become an important tool in the management of acquired TTP.
The results show that caplacizumab is able to control the acute phase of the
disease and buys time for the later onset of the immunosuppressive treatment
which is often needed to fully resolve a TTP episode."
Dr Dominique Tersago, Chief Medical Officer of Ablynx, added:
"These results strengthen our belief in caplacizumab's mechanism of action as a
potential novel treatment for acquired TTP. We will now work diligently to
ensure that this product could become available to patients at the earliest
opportunity."
About caplacizumab
Caplacizumab is a bivalent anti-vWF Nanobody which is highly potent and
selective. It received Orphan Drug Designation in the US and EU in 2009 and
could be the first drug specifically approved for the treatment of acquired TTP
as an adjunct to plasma exchange.
Von Willebrand factor (vWF) is a blood glycoprotein involved in haemostasis, a
complex process that causes the bleeding process to stop. vWF's primary function
is to bind to other proteins, including glycoprotein Ib in the initiation of
platelet adhesion.
vWF is implicated in TTP where ultra-large, multimeric precursors of vWF (UL-
vWF) are present in the blood of patients leading to the formation of unwanted
characteristic string-like clots in small blood vessels. In healthy subjects,
UL-vWF are immediately cleaved into smaller, regular-sized multimers by the
ADAMTS13 enzyme. In patients with TTP, activity of the ADAMTS13 enzyme is
impaired (<10%) due to the formation of auto-antibodies against the enzyme. In
some cases, ADAMTS13 activity is normal (>10%) but UL-vWF are present as a
result of massive endothelial stimulation with consequent release of UL-vWF
multimers in amounts exceeding the system's ability to degrade them.
Caplacizumab inhibits platelet binding to UL-vWF and thus has the potential to
prevent the formation of these string-like clots in the blood of patients with
acquired TTP. Importantly, caplacizumab selectively prevents thrombus formation
in high-shear blood vessels and is expected not to interact with haemostasis in
normal, healthy blood vessels.
About Phase II TITAN trial
The worldwide Phase II TITAN clinical trial was a single-blinded, randomised,
placebo-controlled study which recruited from January 2011 to January 2014. In
total, 75 patients were randomized on a 1:1 basis with one active drug treatment
arm and one placebo arm. All patients received the current standard of care
which is primarily multiple plasma exchanges. The protocol for the study was
adapted in September 2013, to also allow one day of plasma exchange prior to
study enrolment. 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.
About TTP
TTP is a rare disorder of the blood coagulation system that causes extensive
microscopic thromboses in small blood vessels throughout the body. It is a
potentially life-threatening disorder characterised by thrombocytopenia,
haemolytic anaemia and microvascular thrombosis causing variable degrees of
tissue ischemia and infarction. TTP exists in two forms: a congenital and an
acquired form, with the latter accounting for >90% of the patients. There are
currently no drugs specifically approved for the treatment of TTP. The standard
of care for the acquired form of TTP is multiple daily plasma exchanges (PE)
until confirmed platelet normalisation which occurs when the patient's platelet
count returns to normal, as well as immunosuppressant treatment. Daily PE
requires lengthy hospital stays and may be associated with clinical
complications. Additionally, a significant number of patients will subsequently
suffer a relapse after recovering from a first TTP episode. There are believed
to be approximately 10,000 TTP-related events in the US and top 15 European
markets per year.
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
30 proprietary and partnered programmes in development in therapeutic areas
including inflammation, haematology, oncology and respiratory disease. The
Company has collaborations and significant partnerships with pharmaceutical
companies including AbbVie, Boehringer Ingelheim, Merck & Co, Merck Serono and
Novartis. 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: +44 (0)7771 954 193 /
+32 (0)473 39 50 68
e: edwin.moses(at)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(at)ablynx.com
Follow us on Twitter (at)AblynxABLX
Ablynx media relations Consilium Strategic Communications:
Mary-Jane Elliott, Jonathan Birt, Amber Bielecka, Lindsey Neville
t: +44 203 709 5700
e: ablynx(at)consilium-comms.com
--------------------------------------------------------------------------------
[1] In healthy subjects, ultra-large, multimeric precursors of vWF (UL-vWF) are
immediately cleaved into smaller, regular-sized multimers by the ADAMTS13
enzyme. But in the majority of patients with acquired TTP, activity of this
ADAMTS13 enzyme is impaired (activity <10%) due to the formation of auto-
antibodies against the enzyme.
[2] Journal of Thrombosis and Haemostasis, Hughes et al, 2009
pdf format of the press release:
http://hugin.info/137912/R/1878224/662002.pdf
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Source: Ablynx via GlobeNewswire
[HUG#1878224]
Unternehmensinformation / Kurzprofil:
Datum: 08.12.2014 - 16:00 Uhr
Sprache: Deutsch
News-ID 357915
Anzahl Zeichen: 11851
contact information:
Town:
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