- Proof-of-concept in the Phase II TITAN study was achieved with significant reductions in time to platelet count normalisation and recurrences while on treatment with caplacizumab
- Based on these results, Ablynx is on track to file for conditional approval of caplacizumab in Europe in the first half of 2017
- A confirmatory international Phase III study in patients with acquired TTP is ongoing and will be used to support a BLA submission in 2018 in the USA
- Ablynx intends to lead the commercialisation of caplacizumab in Europe and the USA
GHENT/ZWIJNAARDE, Belgium, Feb. 11, 2016 (GLOBE NEWSWIRE) -- Ablynx [Euronext Brussels: ABLX; OTC: ABYLY] today announced that the results of the Company's worldwide Phase II TITAN study[1] with caplacizumab for patients with acquired thrombotic thrombocytopenic purpura (aTTP) have been published in today's issue of the New England Journal of Medicine (NEJM).
"Caplacizumab has the potential to become an important new component in the standard of care for patients with acquired TTP" said Professor Flora Peyvandi, Principal Investigator for the TITAN study at IRCCS Maggiore Hospital Foundation, University of Milan, Italy, and lead author of the NEJM paper. "The results from the Phase II TITAN study showed that caplacizumab acts quickly to control the critical acute phase of the disease and protects patients until immunosuppressive treatments take effect."
Dr Robert K. Zeldin, Chief Medical Officer of Ablynx, commented: "The publication of the TITAN data in this high-impact clinical journal[2] is a further validation of the potential of caplacizumab in the treatment of acquired TTP. This publication is the culmination of over a decade of work by Ablynx and its external collaborators. We are on track to file for conditional approval of caplacizumab in Europe in 2017 and to complete enrolment of the confirmatory Phase III study before the end of 2017. We look forward to making caplacizumab available for patients with this devastating disease."
About caplacizumab and the TITAN study results
Caplacizumab's clinical effect was demonstrated in the Phase II TITAN study in 75 patients with aTTP:
- As indicated by a nearly 40% reduction in median time to platelet count normalisation (p = 0.005). Treatment with caplacizumab reduced the use of daily plasma exchange (PEX) and prevented further consumption of platelets in microthrombi and small blood vessel occlusion.
- As shown by the low number of recurrences requiring re-initiation of daily plasma exchange during treatment with caplacizumab (N=3) vs. placebo (N=11).
These results will serve as the basis for filing for conditional approval in Europe in H1 2017. Caplacizumab could be the first drug specifically approved for the treatment of acquired TTP.
More information on caplacizumab, including the NEJM paper, can be found on Ablynx's website.
About aTTP
aTTP is associated with major morbidities in the brain (e.g. stroke), heart and kidney and impacts life expectancy and quality of life. Mortality is high at 10-20%[4], typically occurring within 2 weeks after initial diagnosis. Moreover, about 36% of patients have recurrences[5] after treatment with the current standard of care, which consists of daily PEX and immune-suppressants, and these recurrences have the potential to cause further organ damage and poorer longer term outcomes.
Phase II TITAN clinical study
The TITAN study was conducted at 56 study centres worldwide, with investigators from countries including Italy, England, Switzerland, the USA and Austria.
Phase III HERCULES clinical study
About Ablynx
For more information, please contact
Dr Edwin Moses
CEO
t: +32 (0)9 262 00 07
m: +32 (0)473 39 50 68
e: edwin.moses@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@ablynx.com
Follow us on Twitter @AblynxABLX
Ablynx media relations:
Instinctif Partners
Sue Charles, Daniel Gooch
London office
t: +44 (0)20 7866 7905
e: ablynx@instinctif.com
Belgium/Dutch and French language
Jim Rusagara
Brussels office
t: +32 (0)2 626 9500
e: ablynx@instinctif.com
[1] Top line data from the TITAN study were communicated in June 2014 and were subsequently presented at ASH 2014 and ISTH 2015 (results from post hoc analysis and ADAMTS13 activity to guide treatment duration)
[2] The most recent (2014) impact factor for NEJM is 55.873, the highest among general medical journals
[3] Veyradier, NEJM 2016: "von Willebrand Factor - A new target for TTP treatment?"
[4] Allford et al, BJH 2003; Kremer Hovinga, Blood 2010; Benhamou, Haematologica 2012
[5] George et al, EJB 2008
pdf format of the press release http://hugin.info/137912/R/1985216/727994.pdf
HUG#1985216