SOUTH SAN FRANCISCO, Calif.,
Oct. 20, 2016 /PRNewswire/
-- Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) today
announced results for the second of two double-blind studies in the
FIT Phase 3 clinical program for fostamatinib, an oral spleen
tyrosine kinase (SYK) inhibitor, in adult chronic/persistent immune
thrombocytopenia (ITP). The primary endpoint in the study was
a stable platelet response, defined as platelet counts greater than
50,000/uL of blood on at least four of the last six scheduled
clinic visits between weeks 14 and 24 of treatment. In the FIT 2
(Study 048) Phase 3 study, the fostamatinib response rate was 18%,
consistent with the recently reported FIT 1 (Study 047) Phase 3
study. In Study 048, one patient in the placebo group (4%) achieved
a stable platelet response; therefore the difference between those
on treatment and those on placebo did not reach statistical
significance (p=0.152) and the study did not meet its primary
endpoint. When the data from both studies are combined, however,
this difference is statistically significant (p=0.007). Data
from both FIT Phase 3 studies and the open-label extension study
demonstrates the consistent benefit of fostamatinib in ITP.
Stable Platelet
Responders / Total Patients
|
|
FIT 1 – Study
047
|
FIT 2 – Study
048
|
Combined
|
Fostamatinib
|
9/51
|
18%
|
9/50
|
18%
|
18/101
|
18%
|
Placebo
|
0/25
|
0%
|
1/24
|
4%
|
1/49
|
2%
|
|
|
p=0.026
|
|
p=0.152
|
|
p=0.007
|
"We believe that the totality and consistency of data from the
FIT Phase 3 program, which included two Phase 3 studies and one
long-term extension study, strongly supports a clear treatment
effect, with a sustained clinical benefit of fostamatinib," said
Raul Rodriguez, president and chief
executive officer of Rigel. "We are encouraged by these
results and believe that the risk/benefit ratio for fostamatinib is
positive for patients with chronic/persistent ITP, a population
with a serious unmet medical need. As a result, we will
continue to pursue this opportunity. Our next step is to seek
feedback from the FDA."
In the combined dataset for Study 047 and Study 048, patients
who met the primary endpoint had their platelet counts increase
from a median of 18,500/uL of blood at baseline to more than
100,000/uL at week 24 of treatment. These patients benefited
substantially and typically did so within weeks of initiating
treatment, providing early feedback as to whether fostamatinib may
be a viable option for treating their ITP. In the combined
data sets, the frequency of patients who achieved a stable platelet
response was statistically superior in the fostamatinib group
versus the placebo group in all subgroup analyses: prior
splenectomy or not; prior exposure to TPO agents or not; platelet
counts below or above 15,000/uL of blood at baseline, demonstrating
that the effect of fostamatinib is consistent across various
clinical and treatment backgrounds.
The most frequent adverse events were gastrointestinal-related,
and the safety profile of the product was consistent with prior
clinical experience, with no new or unusual safety issues
uncovered.
FIT Phase 3 Long-Term Extension Study 049
Patients from both the 047 and 048 Phase 3 studies were given
the option to enroll in a long-term open-label extension study
(Study 049) and receive treatment with fostamatinib. As of
June 2016, 118 patients had been
enrolled in this study. All the patients who responded to
fostamatinib in the parent studies enrolled in Study 049 and had a
median platelet count of 96,000/uL of blood in this study. These
patients have been exposed to fostamatinib for a median of 13
months through the combined parent and 049 trials.
In addition, there were 43 placebo non-responders from the 047
and 048 studies that enrolled in the 049 study. 36 of these
patients had at least 12 weeks of follow-up. Of these, 6
patients (17%, p=0.01) achieved a prospectively defined stable
platelet response in the 049 study.
"Given the heterogeneity of ITP, it is currently almost
impossible to predict how patients will respond to available
therapies, which is why it is so important for physicians and
patients to have treatment options," said James B. Bussel, M.D., professor of pediatrics,
pediatrics in obstetrics and gynecology, and pediatrics in medicine
at Weill Cornell Medicine, and the principal study investigator on
the FIT Phase 3 program. Dr. Bussel is also a member of
Rigel's advisory/scientific board. "This heterogeneity means
that treatments that work by different mechanisms can make
important contributions in certain patients, such as those who
might be especially responsive to fostamatinib because of its
unique mechanism of action. The FIT Phase 3 studies have both
demonstrated that fostamatinib provided a robust and enduring
benefit for those patients who responded to the drug."
Statement on Financial Position
Rigel expects to
report that it ended the third quarter of 2016 with approximately
$85.3 million in cash, cash
equivalents, and short-term investments, which Rigel expects will
be sufficient to fund its operations through the end of 2017.
In this forecast, Rigel has allocated substantial funds to continue
efforts in preparation of the potential commercial launch of
fostamatinib in ITP. Rigel is also continuing to evaluate
ex-U.S. partnerships for fostamatinib and other partnering
opportunities. As a result of the recent research
restructuring, Rigel believes that it has created greater
flexibility for its cash runway moving forward.
About the 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,
have failed at least one prior therapy for ITP, and have platelet
counts consistently below 30,000/uL of blood. Patients were
randomized in a 2:1 ratio to receive either fostamatinib or placebo
orally twice a day for up to 24 weeks. The primary efficacy
endpoint of this program is a stable platelet response defined as
achieving platelet counts greater than 50,000/uL of blood for at
least four of the six scheduled clinic visits between weeks 14 and
24 of treatment. Patients were subsequently offered to enroll
in an open-label, long-term Phase 3 extension study, which is
ongoing.
About 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. ITP
patients can suffer extraordinary bruising, bleeding and fatigue as
a result of low platelet counts. Current therapies for ITP
include steroids, blood platelet production boosters (TPOs) and
splenectomy. However, a significant portion of patients do not do
well on existing therapies. As a result, there remains a
significant medical need for additional treatment options for
patients with ITP.
Fostamatinib is an oral investigational candidate 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 FDA 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 cause of ITP by impeding platelet
destruction.
Conference Call and Webcast 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 1-855-892-1489
(domestic) or 1-720-634-2939 (international) and using the
Conference ID number 98782744.
The conference call 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. The company completed and reported
results from two Phase 3 clinical studies of fostamatinib in
chronic immune thrombocytopenia (ITP) in August and October 2016. Rigel is also conducting a Phase 2
clinical trial with fostamatinib in 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, as well as Rigel's cash position as of
September 30, 2016 and sufficiency of
cash resources. 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, change in
Rigel's estimated cash position based on the completion of its
financial closing procedures, 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 Quarterly Report on
Form 10-Q for the quarter 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
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