TiGenix Receives Positive Feedback from the FDA on Cx601 Global Phase III Trial Protocol
(Thomson Reuters ONE) -
PRESS RELEASE
TiGenix Receives Positive Feedback from the FDA on Cx601 Global Phase III Trial
Protocol
Leuven (BELGIUM) - 7(th) March, 2017, 7:00 am CET - TiGenix NV (Euronext
Brussels and Nasdaq: TIG), an advanced biopharmaceutical company focused on
developing and commercializing novel therapeutics from its proprietary platform
of allogeneic stem cells, announced today that it has received positive feedback
from the U.S. Food and Drug Administration (FDA) on an improved protocol for its
global Phase III trial for the treatment of complex perianal fistulas in Crohn's
disease patients.
On August 2015, TiGenix received the FDA endorsement of a Special Protocol
Assessment (SPA) for a pivotal Phase III study for Biologics License Application
(BLA) in the U.S. Since the granting of the SPA, further confirmatory positive
results have been obtained from the European Phase III study of Cx601 (ADMIRE-
CD), the largest randomized study performed so far in Crohn's disease patients
with complex perianal fistulas. Subsequently, TiGenix submitted a Marketing
Authorization Application to the European Medicines Agency in March 2016.
Following the publication of this additional data, TiGenix submitted a series of
technical improvements to be considered by the FDA regarding the previously
endorsed protocol.
Based on the minutes from a meeting with the FDA on January 19, 2017, the Agency
has agreed to an improved protocol for the global Phase III trial of Cx601. With
these current amendments, the FDA has agreed that the BLA could be filed based
on the efficacy and safety follow-up of patients assessed at week 24, instead of
week 52. Furthermore, the FDA has agreed to accept fewer patients than
originally planned in the study, and has endorsed a broader target population
that will ultimately facilitate the recruitment process. With these adjustments,
the study will benefit from an expedited recruitment process that should lead to
shorter timelines, an earlier filing, and the possibility of an earlier approval
in the U.S. As a result of these modifications, the trial design is even more
similar to the European ADMIRE-CD than it was before.
"We are pleased to have had the opportunity to meet with the FDA and receive
the Agency endorsement for our proposals for this improved protocol," said Dr.
Maria Pascual, VP Regulatory Affairs & Corporate Quality of TiGenix. "We
continue to be focused and committed to bringing Cx601 to U.S. patients as soon
as possible. We believe that this revised protocol will allow us to file for
approval one year earlier than we had originally planned," concluded Dr.
Pascual.
The global pivotal Phase III trial for the U.S. registration of Cx601 is
expected to begin in the first half of 2017. In parallel, TiGenix is exploring
further expedited pathways to accelerate the submission and review process for
its future BLA.
###
For more information
Claudia D'Augusta
Chief Financial Officer
T: +34 91 804 92 64
claudia.daugusta(at)tigenix.com
About TiGenix
TiGenix NV (Euronext Brussels: TIG) is an advanced biopharmaceutical company
focused on developing and commercializing novel therapeutics from its
proprietary platforms of allogeneic, or donor-derived, expanded stem cells. Our
lead product candidate from the adipose-derived stem cell technology platform is
Cx601, which is in registration with the European Medicines Agency for the
treatment of complex perianal fistulas in Crohn's disease patients. Our adipose-
derived stem cell product candidate Cx611 has completed a Phase I sepsis
challenge trial and a Phase I/II trial in rheumatoid arthritis. Effective July
31, 2015, TiGenix acquired Coretherapix, whose lead cellular product candidate,
AlloCSC-01, is currently in a Phase II clinical trial in Acute Myocardial
Infarction (AMI). In addition, the second product candidate from the cardiac
stem cell-based platform acquired from Coretherapix, AlloCSC-02, is being
developed in a chronic indication. On July 4, 2016, TiGenix entered into a
licensing agreement with Takeda, a large pharmaceutical company active in
gastroenterology, under which Takeda acquired the exclusive right to
commercialize Cx601 for complex perianal fistulas outside the United States.
TiGenix is headquartered in Leuven (Belgium) and has operations in Madrid
(Spain).
About Cx601
Cx601 is a suspension of allogeneic expanded adipose-derived stem cells (eASC)
locally injected. Cx601 is an investigational agent being developed for the
treatment of complex perianal fistulas in Crohn's disease patients with
inadequate response to at least one conventional or biologic therapy including
antibiotics, immunosuppressants, or anti-TNF agents. Crohn's disease is a
chronic inflammatory disease of the intestine and patients can suffer from
complex perianal fistulas for which there is currently no effective treatment.
In 2009, the European Commission granted Cx601 orphan designation for the
treatment of anal fistulas, recognizing the debilitating nature of the disease
and the lack of treatment options. Cx601 has met the primary end-point in the
Phase III ADMIRE-CD study in Crohn's disease patients with complex perianal
fistula, a randomized, double-blind, placebo-controlled trial run in Europe and
Israel and designed to comply with the requirements laid down by the EMA.
'Madrid Network' issued a soft loan to help finance this Phase III study, which
was funded by the Secretary of State for Research, Development and Innovation
(Ministry of Economy and Competitiveness) within the framework of the INNTEGRA
plan. The study's primary endpoint was combined remission, defined as clinical
assessment at week 24 of closure of all treated external openings draining at
baseline despite gentle finger compression, and absence of collections >2cm
confirmed by MRI. In the ITT population (n=212), Cx601 achieved statistically
significant superiority (p=0.024) on the primary endpoint with 50% combined
remission at week 24 compared to 34% in the placebo arm. Efficacy results were
robust and consistent across all statistical populations. Treatment emergent
adverse events (non-serious and serious) and discontinuations due to adverse
events were comparable between Cx601 and placebo arms. The 24-weeks results have
been published by The Lancet, one of the most highly regarded and well known
medical journals in the world. The Phase III study has completed a follow-up
analysis at 52 weeks confirming its sustained efficacy and safety profile. Top
line follow-up data showed that in the ITT population Cx601 achieved statistical
superiority (p=0.012) with 54% combined remission at week 52 compared to 37% in
the placebo arm. The 52-week data also showed a higher rate of sustained closure
in those patients treated with Cx601 and in combined remission at week 24
(75.0%) compared to patients in the placebo group (55.9%). Based on the positive
24-weeks Phase III study results, TiGenix has submitted a Marketing
Authorization Application to the EMA in early 2016. TiGenix is preparing to
develop Cx601 in the U.S. after having reached an agreement with the FDA through
a special protocol assessment procedure (SPA) in 2015. On July 4, 2016 TiGenix
entered into a licensing agreement with Takeda, a pharmaceutical company leader
in gastroenterology, whereby Takeda acquired an exclusive right to commercialize
Cx601 for complex perianal fistulas in Crohn's patients outside of the U.S.
Forward-looking information
This press release may contain forward-looking statements and estimates with
respect to the anticipated future performance of TiGenix and the market in which
it operates. Certain of these statements, forecasts and estimates can be
recognised by the use of words such as, without limitation, "believes",
"anticipates", "expects", "intends", "plans", "seeks", "estimates", "may",
"will" and "continue" and similar expressions. They include all matters that are
not historical facts. Such statements, forecasts and estimates are based on
various assumptions and assessments of known and unknown risks, uncertainties
and other factors, which were deemed reasonable when made but may or may not
prove to be correct. Actual events are difficult to predict and may depend upon
factors that are beyond the Company's control. Therefore, actual results, the
financial condition, performance or achievements of TiGenix, or industry
results, may turn out to be materially different from any future results,
performance or achievements expressed or implied by such statements, forecasts
and estimates. Given these uncertainties, no representations are made as to the
accuracy or fairness of such forward-looking statements, forecasts and
estimates. Furthermore, forward-looking statements, forecasts and estimates only
speak as of the date of the publication of this press release. TiGenix disclaims
any obligation to update any such forward-looking statement, forecast or
estimates to reflect any change in the Company's expectations with regard
thereto, or any change in events, conditions or circumstances on which any such
statement, forecast or estimate is based, except to the extent required by
Belgian law.
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: TiGenix via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 07.03.2017 - 07:00 Uhr
Sprache: Deutsch
News-ID 528495
Anzahl Zeichen: 11103
contact information:
Town:
Leuven
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 258 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"TiGenix Receives Positive Feedback from the FDA on Cx601 Global Phase III Trial Protocol"
steht unter der journalistisch-redaktionellen Verantwortung von
TiGenix (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
