YASTEST
GHENT, Belgium,
21 December 2017 - Ablynx NV [Euronext
Brussels and Nasdaq: ABLX] today announced that the single and
multiple dose Phase I study demonstrated comparable PK of
caplacizumab in Japanese and Caucasian subjects. Caplacizumab is
the Company's wholly-owned anti-von Willebrand factor (vWF)
Nanobody® being developed for the treatment of acquired thrombotic
thrombocytopenic purpura (aTTP).
The Phase I single centre study
enrolled 60 healthy Japanese and Caucasian subjects and consisted
of single ascending dose and multiple dose parts. At all doses
studied, the PK of caplacizumab in the Japanese population were
similar to those observed in Caucasians. Caplacizumab was
well-tolerated in all groups and its safety profile was consistent
with its mechanism of action.
Dr Robert K.
Zeldin, Chief Medical Officer at Ablynx, commented:
"We are very pleased with these results as they enable us to bridge
available clinical data for caplacizumab between Japanese and
Caucasian populations. This is a very important step in making
caplacizumab available to Japanese patients suffering from aTTP. We
look forward to discussing these results, together with the recent
Phase III HERCULES study results of caplacizumab, with the Japanese
Regulatory Agency and aligning on a path forward for regulatory
submission in Japan."
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
demonstrated in the Phase II TITAN and Phase III HERCULES studies.
In the HERCULES study, 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). Importantly, treatment with caplacizumab
resulted in a clinically meaningful reduction in the use of PEX and
length of stay in the ICU and the hospital, compared to the placebo
group. In addition, caplacizumab has the potential to prevent
refractory disease and have a positive impact on the normalisation
of organ damage markers (lactate dehydrogenase, cardiac troponin I
and serum creatinine). Caplacizumab has a favourable safety
profile, consistent with its mechanism of action. No deaths were
reported during the study drug treatment in the caplacizumab group
in both the TITAN and HERCULES studies, while for the placebo
group, two deaths were reported in the TITAN study and three deaths
in the HERCULES study.
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.
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
release
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
Ablynx NV (NASDAQ:ABLX)
Graphique Historique de l'Action
De Mai 2024 à Juin 2024
Ablynx NV (NASDAQ:ABLX)
Graphique Historique de l'Action
De Juin 2023 à Juin 2024