SOUTH SAN FRANCISCO, Calif.,
Aug. 30, 2016 /PRNewswire/
-- Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) today
announced that fostamatinib, its oral spleen tyrosine kinase (SYK)
inhibitor, met the primary endpoint in the first of two
double-blind studies in the FIT Phase 3 clinical program for the
treatment of adult chronic/persistent immune thrombocytopenia
(ITP). The study (n=76) showed that 18% of patients receiving
fostamatinib achieved a stable platelet response compared to none
receiving a placebo control (p=0.0261). A stable platelet
response was defined as achieving greater than 50,000 platelets per
uL of blood on at least four of the last six scheduled visits
between weeks 14 and 24 of treatment. The results from the second
FIT Phase 3 study are expected in October/November 2016.
The most frequent adverse events were gastrointestinal-related,
and the safety profile of the product was consistent with prior
clinical experience, and no new or unusual safety issues were
discovered.
"These data demonstrate the potential benefit of fostamatinib
for chronic ITP patients who are in need of new treatment options,"
said Raul Rodriguez, president and
chief executive officer of Rigel. "We believe that fostamatinib has
significant commercial potential given that it has a unique
mechanism of action that may work where other products have
failed."
"We are very encouraged by these results," said Anne-Marie
Duliege, M.D., executive vice president and chief medical officer
of Rigel. "Consistent with the prior clinical study of fostamatinib
in ITP, this FIT Phase 3 study demonstrated that fostamatinib
provided a robust and enduring benefit for those patients who
responded to the drug candidate."
Patients who met the primary endpoint of this study typically
had an increase in platelet counts to a level above 50,000/uL
within the initial weeks of treatment, providing early feedback as
to whether it was a viable option for treating their ITP.
In general, the clinical goal of ITP treatment is to raise
platelet counts to more than 50,000/uL. Patients who met the
primary endpoint in this study had their platelet counts increase
from a median of 16,000/uL at baseline to a median of more than
100,000/uL at week 24, a robust response that potentially allows
patients to remain above 50,000/uL more consistently.
All of the patients from this study who met the stable platelet
response endpoint enrolled in the long-term, Phase 3 extension
study and continued to maintain their platelet levels for months
past the initial study period of 24 weeks. These data affirm
similar results observed in two patients from the Rigel Phase 2
study of fostamatinib in ITP who have been taking fostamatinib for
more than seven years and have maintained stable platelet levels
over this extended time period.
Fostamatinib's clinical safety profile includes more than 5,000
patient years of data across multiple autoimmune indications and
has a well-defined and manageable safety profile, providing data
that it may be suitable for long-term maintenance therapy in
chronic ITP.
If these results are reproduced in the second Phase 3 study and
are supported by the results of a planned interim analysis of the
Phase 3 extension study, the company expects to submit a New Drug
Application with the U.S. Food and Drug Administration in the first
quarter of 2017. Further results from the FIT Phase 3 studies
and long-term extension will be presented at future medical
meetings.
FIT Phase 3 Program
The FIT program consists of two
identical multi-center, randomized, double-blind,
placebo-controlled studies of approximately 75 adult patients each.
The patients have been diagnosed with persistent or chronic ITP,
and have blood platelet counts consistently below 30,000/uL of
blood. The patients all had experience with at least one other
ITP treatment such as steroids, Rituxan, splenectomy and/or TPO
mimetics. Patients were randomized in a 2:1 ratio to receive
either fostamatinib or placebo twice a day to be taken for up to
six months. Study subjects remained on treatment for up to 24
weeks. The primary efficacy endpoint of this program is a
stable platelet response defined as achieving platelet counts at or
above 50,000/uL of blood for at least four of the last six clinic
visits of the study. Patients were subsequently offered to
enroll in an open-label, Phase 3, long-term extension study, which
is ongoing.
Fostamatinib and ITP
In patients with ITP, the immune
system attacks and destroys the body's own blood platelets, which
play an active role in blood clotting and healing. There are
approximately 50-60 thousand adult patients in the U.S. living with
primary chronic ITP. ITP patients can suffer extraordinary
bruising, bleeding and fatigue as a result of low platelet
counts. Further, people suffering with chronic ITP live with
increased risk of severe bleeding events that can result in serious
medical complications or even death. Current therapies for ITP
include steroids, blood platelet production boosters (TPOs) and
splenectomy. While these treatment options can be effective
in treating ITP symptoms, given the heterogeneity of the disease,
each has significant limitations. It can be difficult to predict
which approved treatments are going to be effective.
Fostamatinib is an oral investigational drug with a unique
mechanism of action designed to inhibit SYK kinase, a key
player in the immune process that leads to platelet destruction in
ITP. The U.S. Food and Drug Administration has granted Orphan
Drug designation to fostamatinib for the treatment of patients with
ITP. Unlike other therapies that modulate the immune system
in different ways or stimulate platelet production, fostamatinib
may address the underlying autoimmune basis of ITP by impeding
platelet destruction. Fostamatinib potentially offers a
compelling addition to the treatment options available for ITP
patients.
Conference Call and Webcast Presentation Today at 8:00AM
Eastern Time
Rigel will hold a live conference call and
webcast today at 8:00am Eastern Time (5:00am Pacific Time). Participants can access the
live conference call by dialing 855-892-1489 (domestic) or
720-634-2939 (international) and using the Conference ID number
72149873.
The conference call and accompanying slide presentation will
also be webcast live and can be accessed from Rigel's website at
www.rigel.com. The webcast will be archived and available for
replay after the call via the Rigel website.
About Rigel (www.rigel.com)
Rigel
Pharmaceuticals, Inc. is a clinical-stage biotechnology
company dedicated to the discovery and development of novel,
targeted drugs in the therapeutic areas of immunology, oncology and
immuno-oncology. Rigel's pioneering research focuses on signaling
pathways that are critical to disease mechanisms. The company's
current clinical programs include fostamatinib, an oral spleen
tyrosine kinase (SYK) inhibitor, which is in Phase 3 clinical
trials for immune thrombocytopenia (ITP); a Phase 2 clinical trial
for autoimmune hemolytic anemia (AIHA); and a Phase 2 clinical
trial for IgA nephropathy (IgAN). In addition, Rigel has two
oncology product candidates in Phase 1 development with partners
BerGenBio AS and Daiichi Sankyo.
This press release contains "forward-looking" statements,
including, without limitation, statements related to Rigel's
clinical development plans, including the timing, design and nature
of planned clinical trials and the timing and nature of results of
those trials, as well as the potential activity of fostamatinib
with respect to ITP. Any statements contained in this press release
that are not statements of historical fact may be deemed to be
forward-looking statements. Words such as "planned," "will," "may,"
"expect," and similar expressions are intended to identify these
forward-looking statements. These forward-looking statements
are based on Rigel's current expectations and inherently involve
significant risks and uncertainties. Actual results and the timing
of events could differ materially from those anticipated in such
forward looking statements as a result of these risks and
uncertainties, which include, without limitation, the availability
of resources to develop Rigel's product candidates, Rigel's need
for additional capital in the future to sufficiently fund Rigel's
operations and research, the uncertain timing of completion of and
the success of clinical trials, risks associated with and Rigel's
dependence on Rigel's corporate partnerships, as well as other
risks detailed from time to time in Rigel's reports filed with
the Securities and Exchange Commission, including its Annual
Report on Form 10-Q for the year ended June 30, 2016.
Rigel does not undertake any obligation to update forward-looking
statements and expressly disclaims any obligation or undertaking to
release publicly any updates or revisions to any forward-looking
statements contained herein.
Contacts:
Raul Rodriguez
Phone: 650.624.1302
Email: invrel@rigel.com
Jessica Daitch
Chandler Chicco Agency
Phone: 917.816.6712
Email: jessica.daitch@inventivhealth.com
Logo - http://photos.prnewswire.com/prnh/20030226/RIGLLOGO
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/rigels-fostamatinib-meets-primary-endpoint-in-phase-3-study-in-chronic-itp-300319730.html
SOURCE Rigel Pharmaceuticals, Inc.