ABLYNX RECEIVES FAST TRACK DESIGNATION FROM THE FDA FOR CAPLACIZUMAB FOR THE TREATMENT OF ACQUIRED TTP
(Thomson Reuters ONE) -
GHENT, Belgium, 26 July 2017 - Ablynx [Euronext Brussels: ABLX; OTC: ABYLY]
today announced that the U.S. Food and Drug Administration (FDA) has granted
Fast Track designation for caplacizumab, the Company's first-in-class anti-von
Willebrand factor (vWF) Nanobody® being developed for the treatment of acquired
thrombotic thrombocytopenic purpura (aTTP).
The FDA's Fast Track programme is designed to facilitate the development and
expedite the review of drugs that treat serious conditions and fill an unmet
medical need. Drugs that receive this designation are eligible for more frequent
interactions with the FDA, priority review and rolling review of the Biologics
License Application (BLA). The purpose of this FDA programme is to get important
new drugs to the patient earlier.
aTTP is a life-threatening, autoimmune blood clotting disorder manifested by
extensive clot formation in small blood vessels throughout the body, leading to
thrombocytopenia, ischemia and widespread organ damage especially in the brain
and heart. Currently no products have been authorised for the treatment of aTTP
and despite standard-of-care treatment with plasma exchange (PEX) and
immunosuppressive therapy, patients remain at risk for thrombotic complications,
recurrences and death.
The potential of caplacizumab to address this unmet need has been demonstrated
in the Phase II TITAN study which supports the Marketing Authorisation
Application (MAA) submitted to the European Medicines Agency (EMA) in February
2017. Caplacizumab is currently being further evaluated in the randomised,
double-blind, placebo-controlled Phase III HERCULES study. Results from this
Phase III study will be reported in the second half of 2017 and are expected to
further support the MAA, as well as a planned BLA filing in the United States in
2018.
Dr Robert K. Zeldin, Chief Medical Officer at Ablynx, commented:
"The designation of Fast Track status by the FDA is recognition of the high
unmet medical need in patients with aTTP and the potential for caplacizumab to
improve outcomes in this very severe disease. We look forward to continuing to
work with the FDA and accelerating the development of caplacizumab as
potentially the first therapeutic specifically indicated for the treatment 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 conjunction with the standard of care
of PEX and immunosuppression, were evaluated in the Phase II TITAN study in 75
patients with aTTP. Caplacizumab was well-tolerated and the primary endpoint was
met (p=0.005), with caplacizumab treatment resulting in a 39% reduction in time
to platelet count normalisation as compared to placebo (i.e., a faster reversion
of thrombocytopenia with consequent reduced use of PEX)[1]. Moreover, during
treatment, caplacizumab reduced recurrences of aTTP by 71% compared to
placebo[2]. 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%)(2). In addition, fewer caplacizumab-treated patients, compared to
those who received placebo, were refractory to treatment (5.7% versus
21.6%)[3]. There were two deaths in the placebo group and both of those patients
were refractory to treatment; no deaths were reported in the caplacizumab group.
The randomised, double-blind, placebo-controlled Phase III HERCULES study
(NCT02553317) will evaluate the efficacy and safety of caplacizumab in patients
with aTTP 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. Key secondary endpoints include a composite
endpoint consisting of TTP-related mortality, recurrence of TTP and major
thromboembolic events during study drug treatment, as well as the prevention of
recurrence of TTP during the study period, refractoriness to treatment, and the
effect on biomarkers of organ damage. Results from this Phase III study are
expected in the second half of 2017 and these results are expected to support a
BLA filing in the United States in 2018. In February 2017 a MAA has been
submitted to the EMA for approval of caplacizumab in aTTP[4]. If approved by
regulatory authorities, caplacizumab will be the first therapeutic specifically
indicated for the treatment of aTTP.
A three-year follow-up study (NCT02878603) of patients who have participated in
the HERCULES study is also in progress and will further evaluate the long-term
safety and efficacy of caplacizumab and repeated use of caplacizumab, as well as
characterizing the long-term impact of aTTP.
About aTTP
aTTP is a rare, acute, life-threatening, blood clotting disorder. It has a
sudden onset 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[5], leading to ischemia and widespread organ
damage[6].
Despite the current standard-of-care treatment with 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[7]. Furthermore, patients are
at risk of acute thromboembolic complications (e.g. stroke, myocardial
infarction) and of recurrence of disease. Some patients are refractory to
therapy(3), which is associated with a poor prognosis for survival of an acute
episode of aTTP. Long term, patients are at increased risk of hypertension,
major depression, and premature death[8].
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(at)ablynx.com
Lies Vanneste
Director Investor Relations
t: +32 (0)9 262 01 37
m: +32 (0)498 05 35 79
e: lies.vanneste(at)ablynx.com
Follow us on Twitter (at)AblynxABLX
Ablynx media/analyst relations
FTI Consulting:
Julia Phillips, Brett Pollard, Mo Noonan
t: +44 20 3727 1000
e: ablynx(at)fticonsulting.com
--------------------------------------------------------------------------------
[1] Press release June 2014; Manuscript in the NEJM, Feb 2016; Manuscript in the
JTH, Apr 2017
[2] Peyvandi et al., notes to editor NEJM 2016
[3] Defined as: 'failure of platelet response after 7 days despite daily plasma
exchange treatment'
[4] Press release February 2017
[5] Veyradier, NEJM 2016: "von Willebrand Factor - A new target for TTP
treatment?"
[6] Scully et al., Br J Hem 2012; Sarode et al., J Clin Apher 2014; Chaturvedi
et al., Am J Hem 2013
[7] Benhamou, Y. et al., Haematologica 2012
[8] Deford et al., Blood 2013
pdf version of the press release:
http://hugin.info/137912/R/2122808/809506.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: 26.07.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 553872
Anzahl Zeichen: 10369
contact information:
Town:
Ghent/Zwijnaarde
Kategorie:
Business News
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