ABLYNX ANNOUNCES POSITIVE TOPLINE RESULTS FROM THE PHASE III HERCULES STUDY OF CAPLACIZUMAB FOR THE TREATMENT OF ACQUIRED TTP
(Thomson Reuters ONE) -
REGULATED INFORMATION - INSIDE INFORMATION
* Caplacizumab meets primary endpoint and key secondary endpoints
* Statistically significant reduction in time to platelet count response,
with at any given time patients treated with caplacizumab 50% more
likely to achieve platelet count response
* 74% relative reduction in the percentage of patients with aTTP-related
death, a recurrence of aTTP, or at least one major thromboembolic event
during the study drug treatment period
* 67% relative reduction in the percentage of patients with aTTP
recurrence during the overall study period
* No caplacizumab-treated patients had refractory disease
* Trend to faster normalisation of organ damage markers
* Safety profile consistent with Phase II TITAN results and mechanism of
action
* Data will be used to drive the registration process for caplacizumab in
Europe and the USA
Conference call and webcast today at 4pm CET/10am ET
GHENT, Belgium, 2 October 2017 - Ablynx [Euronext Brussels: ABLX; OTC: ABYLY]
today announced positive topline 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).
Treatment with caplacizumab in addition to standard-of-care resulted in a
statistically significant reduction in time to platelet count response (p<0.01),
the primary endpoint of the study and a measure of prevention of further
microvascular thrombosis. Patients on caplacizumab were 1.5 times more likely to
achieve platelet count response at any given time point, compared to patients
treated with placebo.
The Phase III HERCULES study also met the first two key secondary endpoints.
Treatment with caplacizumab resulted in a 74% reduction in the percentage of
patients with aTTP-related death, recurrence of aTTP, or at least one major
thromboembolic event during study drug treatment (p<0.0001), with recurrences
being the driver for achievement of this endpoint. In addition, the proportion
of patients with a recurrence of aTTP in the overall study period (including the
28 day follow-up after discontinuation of study drug treatment) was 67% lower in
the caplacizumab arm compared to the placebo arm (p<0.001), demonstrating the
durability of the treatment effect.
Analysis of the third key secondary endpoint showed that no patients treated
with caplacizumab were refractory to treatment compared to three patients
treated with placebo (p=0.057).
The analysis of the fourth key secondary endpoint showed a trend to faster
normalisation of the organ damage markers (lactate dehydrogenase, cardiac
troponin I and serum creatinine) in patients treated with caplacizumab.
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. One patient in
the caplacizumab group died in the follow-up period after completing the study
drug treatment and this was assessed by the investigator not to be related to
study drug.
Caplacizumab is wholly-owned by Ablynx and today's reported data will be used to
support the registration process for caplacizumab in Europe and the USA.
Dr Robert K. Zeldin, Chief Medical Officer at Ablynx, commented:
"Patients with aTTP are at risk for significant morbidity and early death. We
believe these positive Phase III study results confirm the potential for
caplacizumab to address the high unmet medical need in the treatment of aTTP and
to have a meaningful impact on the lives of affected patients. We continue to
analyse the data and look forward to sharing the results with regulatory
authorities and the scientific community."
"We thank the study participants and their families as well as the investigators
and staff who contributed to this study."
Dr Edwin Moses, CEO of Ablynx, commented:
"I am delighted by this outcome as it reinforces all our beliefs in the
potential for caplacizumab to change the lives of patients affected by aTTP.
This is a very important milestone for the Company as it further validates our
Nanobody platform and demonstrates our ability to discover and develop medicines
that make a real difference for society. These results strengthen our resolve to
obtain marketing approval as quickly as possible so that caplacizumab rapidly
becomes available to patients suffering from this severe disease for which there
is currently no approved drug available."
Professor Marie Scully, leading TTP specialist from the University College
Hospital in London and Investigator in the HERCULES study commented:
"The results of this landmark trial constitute a complete game changer for
patients with aTTP. They will revolutionise how we manage the acute phase of the
disease, which is when patients are at highest risk for organ damage, recurrence
and death."
Investor conference call and webcast information
Ablynx will host a conference call/webcast today at 4pm CET, 10am ET. The
webcast may be accessed by clicking here. To participate in the Q&A, please dial
+32 (0)2 620 01 38, using confirmation code 4097559. Shortly thereafter, a
replay of the webcast will be available on the Company's website:
http://www.ablynx.com/news/events-presentations/.
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 standard-of-care treatment (i.e. daily plasma exchange
[PEX] and immunosuppression). Patients received a single intravenous bolus of
10mg caplacizumab or placebo followed by daily subcutaneous dose of 10mg
caplacizumab or placebo until 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, the treatment could be
extended for additional seven-day periods up to a maximum of 28 days. Patients
were followed up for a further 28 days after discontinuation of treatment.
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.
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[1]. In July 2017, Ablynx received
Fast Track designation from the Food and Drug Administration (FDA) for
caplacizumab for the treatment of aTTP.[2] 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[3], leading to ischemia and widespread organ
damage[4].
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[5].
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 for
hypertension, major depression, and premature death[6].
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 relations:
Consilium Strategic Communications
Mary-Jane Elliott, Philippa Gardner, Sukaina Virji
t: +44 (0)20 3709 5700
e: ablynx(at)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] Press release February 2017
[2] Press release July 2017
[3] Veyradier, NEJM 2016: "von Willebrand Factor - A new target for TTP
treatment?"
[4] Scully et al., Br J Hem 2012; Sarode et al., J Clin Apher 2014; Chaturvedi
et al., Am J Hem 2013
[5] Benhamou, Y. et al., Haematologica 2012
[6] Deford et al., Blood 2013
pdf version of the press release:
http://hugin.info/137912/R/2138418/818605.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: 02.10.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 561971
Anzahl Zeichen: 15635
contact information:
Town:
Ghent/Zwijnaarde
Kategorie:
Business News
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