YASTEST
Additional
results from the Phase III HERCULES study showed that treatment
with caplacizumab resulted in:
-
38% relative reduction in the
number of days of plasma exchange (PEX)
-
41% relative reduction in the
volume of plasma used
-
65% relative reduction in the
number of days in the intensive care unit (ICU)
-
31% relative reduction in the
number of days in hospital
Conference call and webcast
today at 4 pm CET/10 am
ET
GHENT, Belgium,
12 December 2017 (1.40 pm CET) - Ablynx NV
[Euronext Brussels and Nasdaq: ABLX] today announced additional
results from the Phase III HERCULES study with caplacizumab, the
Company's anti-von Willebrand factor (vWF) Nanobody® being
developed for the treatment of acquired thrombotic thrombocytopenic
purpura (aTTP). These new data relate to additional pre-specified
secondary endpoints and demonstrate that treatment with
caplacizumab resulted in a clinically meaningful reduction in the
use of PEX and length of stay in the ICU and the hospital.
The number of days of PEX during
the overall treatment period was 38% lower in the caplacizumab
group compared to the placebo arm (5.8 days versus 9.4 days)
resulting in a 41% reduction in the volume of plasma used (21.3L on
caplacizumab compared to 35.9L in the placebo group). For those
patients admitted to the ICU, the number of days in intensive care
was reduced by 65% for patients treated with caplacizumab compared
to placebo (3.4 days versus 9.7 days, respectively). The overall
duration of hospitalisation in the caplacizumab group was reduced
by 31% compared to the placebo group (9.9 days versus 14.4 days,
respectively).
The data were presented today by
Professor Marie Scully of the University College London Hospitals,
an investigator in the HERCULES trial, as part of the late-breaking
abstracts session at the 59th Annual
Meeting of the American Society of Hematology (ASH) in Atlanta, GA,
USA. The presentation is available on the Ablynx website under
Events & Presentations.
Dr Robert K.
Zeldin, Chief Medical Officer at Ablynx, commented:
"The reduced use of plasma
exchange and shorter time in the ICU and hospital further
demonstrate the potential positive impact of caplacizumab on the
quality of life of patients with aTTP, together with providing the
opportunity for considerable cost savings. These data build on the
previously reported topline results which demonstrated that
patients treated with caplacizumab experienced a faster resolution
of their acute aTTP episode with a significantly shorter time to
platelet count response and a significant reduction in
recurrences."
"These efficacy data together with
caplacizumab's safety profile demonstrate that it has the potential
to address the high unmet medical need in the treatment of aTTP and
to have an important impact on the lives of affected patients. We
look forward to working together with regulatory authorities to
make caplacizumab available for patients suffering from this
devastating disease."
Investor
conference call and webcast information
Ablynx will host a conference call
and webcast today at 4.00 pm CET/10.00 am ET. The live webcast and
replay will be available via this link. If you wish to participate
in the Q&A session, please dial +32(0)2 400 69 26 or
+1 646 828 8193 and use confirmation code 9477994.
About
HERCULES
The HERCULES study recruited 145
patients and is the largest randomised, double-blind,
placebo-controlled study conducted in patients with aTTP. Patients
with an acute episode of aTTP were randomised 1:1 to receive either
caplacizumab or placebo in addition to daily PEX and
immunosuppression. Patients received a single intravenous bolus of
10mg caplacizumab or placebo followed by a daily subcutaneous dose
of 10mg caplacizumab or placebo for 30 days after the last daily
PEX. If at the end of this treatment period there was evidence of
persistent underlying disease activity (indicative of an imminent
risk for recurrence), treatment could be extended for additional
seven-day periods up to a maximum of 28 days and was to be
accompanied by optimisation of immunosuppression. Patients were
followed for a further 28 days after discontinuation of
treatment.
Positive topline results from the
Phase III HERCULES study, meeting its primary and first two key
secondary endpoints, were announced on 2 October 2017. Treatment
with caplacizumab in addition to standard-of-care resulted in a
significantly shorter time to platelet count response (p<0.01),
a significant reduction in aTTP-related death, recurrence of aTTP,
or at least one major thromboembolic event during study drug
treatment (p<0.0001), and a significantly lower number of aTTP
recurrences in the overall study period (p<0.001). Analysis of
the third and fourth key secondary endpoints showed the potential
of caplacizumab to prevent refractory disease and positively impact
normalisation of organ damage markers (lactate dehydrogenase,
cardiac troponin I and serum creatinine).
Based on the topline data, the
safety profile of caplacizumab is consistent with its mechanism of
action and the Phase II TITAN study results. The number and nature
of treatment-emergent adverse events (TEAEs) were similar between
the treatment groups. Serious TEAEs were more common in the placebo
group, driven by the percentage of patients experiencing a
recurrence of aTTP. Consistent with the mechanism of action of
caplacizumab, the percentage of subjects with any bleeding-related
TEAE was higher in the caplacizumab treatment group than in the
placebo treatment group (66.2% vs. 49.3%). Most bleeding-related
TEAEs were mild or moderate in severity. There were three deaths in
the placebo group and none in the caplacizumab group during the
study drug treatment period.
A three-year follow-up study
(NCT02878603) of patients who have completed the HERCULES study is
in progress and will further evaluate the long-term safety and
efficacy of caplacizumab and repeated use of caplacizumab, as well
as characterising the long-term impact of aTTP.
About
caplacizumab
Caplacizumab is a bivalent
anti-vWF Nanobody that received Orphan Drug Designation in Europe
and the United States 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 micro-clots that cause the severe
thrombocytopenia, tissue ischemia and organ dysfunction in aTTP.
This immediate effect of caplacizumab has the potential to protect
the patient from the manifestations of the disease while the
underlying disease process resolves.
The efficacy and safety of
caplacizumab in addition to daily PEX and immunosuppression were
evaluated in the Phase II TITAN study and in the Phase III HERCULES
study.
In February 2017, based on the
Phase II TITAN study results, a Marketing Authorisation Application
(MAA) was submitted to the European Medicines Agency (EMA) for
approval of caplacizumab in aTTP. In July 2017, Ablynx received
Fast Track designation from the Food and Drug Administration (FDA)
for caplacizumab for the treatment of aTTP. The positive
results from the Phase III HERCULES study are expected to further
support the MAA, as well as a planned Biologics License Application
(BLA) filing in the United States in 2018. If approved by
regulatory authorities, caplacizumab would be the first therapeutic
specifically indicated for the treatment of aTTP.
About
aTTP
aTTP is a rare, acute,
life-threatening, autoimmune blood clotting disorder. It is caused
by impaired activity of the ADAMTS13 enzyme, 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 clot formation in small blood vessels
throughout the body[1], leading to
ischemia and widespread organ damage[2].
Despite the current
standard-of-care treatment consisting of 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, myocardial infarction) and of
recurrence of disease. Some patients are refractory to
therapy1, which is
associated with a poor prognosis for survival of an acute episode
of aTTP. Long term, patients are at increased risk for
hypertension, major depression, and premature death[4].
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; Merck & Co., Inc., Kenilworth, New Jersey, USA;
Merck KGaA; Novartis; Novo Nordisk; Sanofi 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@ablynx.com
Lies Vanneste
Director Investor Relations
t: +32 (0)9 262 01 37
m: +32 (0)498 05 35 79
e: lies.vanneste@ablynx.com
Follow us on Twitter @AblynxABLX
Ablynx media
relations:
Consilium Strategic Communications
Mary-Jane Elliott, Philippa Gardner, Sukaina Virji
t: +44 (0)20 3709 5700
e: ablynx@consilium-comms.com
Disclaimer
Certain statements, beliefs and opinions in this press release 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 press
release 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 press release 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 press release 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
press release.
[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] Deford
et al., Blood 2013
pdf version of the press
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Source: Ablynx via Globenewswire
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