SOUTH SAN FRANCISCO, Calif.,
Oct. 3, 2017 /PRNewswire/
-- Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) today
announced that the company recently completed enrollment of
Stage 1 of its Phase 2, open-label, multi-center, two-stage study
of its investigational drug fostamatinib for the treatment of
patients with warm antibody AIHA. On a top-line, preliminary basis,
the Phase 2 study has achieved the pre-specified primary efficacy
endpoint for Stage 1. A response was defined as achieving a
hemoglobin level of greater than 10 g/dl and at least a 2 g/dl
increase from baseline.
This Phase 2 study, also known as the SOAR study, is evaluating
the safety and efficacy of fostamatinib in patients with warm
antibody AIHA who have previously received at least one treatment
for this disease, but did not have a meaningful benefit and are
still anemic. The SOAR study utilizes an open-label, Simon
two-stage design to evaluate fostamatinib at 150 mg BID (twice
daily) in patients with warm antibody AIHA, a disease for which
there is no available treatment.
Stage 1 of the SOAR study has enrolled 17 patients who have had
at least one post-baseline hemoglobin measure. Of the 17 patients,
4 responded during the 12-week evaluation period and an additional
2 patients met the response criteria in the extension study after
12 weeks of dosing, for a response rate of 35% (6/17) on
fostamatinib (these data are preliminary and require further
verification). During the trial, 2 of the 17 patients
withdrew early from the study due to non-safety-related reasons and
will be replaced per the study protocol. A comprehensive analysis
of the Phase 2 data will continue and will be presented at a future
scientific conference.
The safety profile was consistent with the existing fostamatinib
safety database, which comprises over 5,000 patient-years of
exposure. Two deaths were reported during the trial due
to non-treatment related serious adverse events (SAEs) as
determined by the investigators (one patient with skin necrosis and
infection, and an elderly patient with pneumonia who was
immunosuppressed due to prior CLL and steroids). A third
patient experienced a non-treatment related SAE as determined by
the investigator, recovered and continued on treatment. Having met
the Stage 1 primary efficacy endpoint, Rigel intends to begin
enrollment for Stage 2 of this study in which 20 patients will be
enrolled under the same protocol.
"Many patients with AIHA suffer from severe, debilitating
disease that negatively affects their quality of life," said
David J. Kuter, M.D., the director
for the Center of Hematology at Massachusetts General Hospital and
the lead investigator of the SOAR study. "There are no
FDA-approved medications for the treatment of AIHA, which means
that those living with the condition are in need of new and
effective therapeutic options."
About AIHA
Autoimmune hemolytic anemia (AIHA) is a
rare, serious blood disorder where the immune system produces
antibodies that result in the destruction of the body's own red
blood cells. AIHA affects approximately 40,000 adult patients in
the US and can be a severe, debilitating anemia. To date, there are
no disease-targeted therapies for AIHA, despite the tremendous
medical need that exists for these patients.
About Rigel (www.rigel.com)
Rigel
Pharmaceuticals, Inc. is a biotechnology company dedicated to
discovering, developing and providing novel small molecule drugs
that significantly improve the lives of patients with immune and
hematological disorders, cancer and rare diseases. Rigel's
pioneering research focuses on signaling pathways that are critical
to disease mechanisms. The company's current clinical programs
include clinical trials of fostamatinib, an oral spleen tyrosine
kinase (SYK) inhibitor, in a number of indications. Rigel has
submitted and the FDA has accepted for review, an NDA for
fostamatinib in patients with chronic or persistent immune
thrombocytopenia (ITP). In addition, Rigel has product candidates
in development with partners BerGenBio AS, Daiichi Sankyo and
Aclaris Therapeutics.
Forward Looking Statements
This release contains
forward-looking statements relating to, among other things, Rigel's
belief that fostamatinib may benefit patients with AIHA and the
timing and nature of results of Rigel's clinical trials. 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," "hope" 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 top-line data Rigel has reported
is based on preliminary analysis of key efficacy and safety data,
and such data may change following a more comprehensive review of
the data related to the clinical trial and such top-line data may
not accurately reflect the complete results of the trial, and the
FDA may interpret Rigel's findings differently, which could result
in the FDA not approving any submitted NDA; 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 studies; market competition, 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, 2017. 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.
Contact: Raul Rodriguez
Phone: 650.624.1302
Email: invrel@rigel.com
Media Contact: Jessica Daitch
Phone: 917.816.6712
Email: jessica.daitch@inventivhealth.com
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SOURCE Rigel Pharmaceuticals, Inc.