SOUTH SAN FRANCISCO, Calif.,
May 16, 2019 /PRNewswire/
-- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), today announced
that it has enrolled the first patient in a pivotal Phase 3
clinical trial of fostamatinib disodium hexahydrate (fostamatinib)
in warm antibody autoimmune hemolytic anemia (AIHA). The clinical
trial protocol calls for approximately 80 patients in a 24-week
study with topline results projected for early 2021. This disorder
affects an estimated 40,000 patients in the U.S., for whom no
approved treatment options currently exist.
"Enrolling the first patient in our Phase 3 clinical trial
of fostamatinib in warm AIHA is an important milestone in our
efforts to develop the first FDA-approved therapy for this
disease," stated Raul Rodriguez,
president and CEO. "With a clear unmet need, warm AIHA is a very
attractive market that is synergistic with our current commercial
infrastructure and provides a significant potential opportunity to
have a positive impact on patient lives in a second
indication."
The Phase 3 clinical trial of fostamatinib is a
placebo-controlled study of approximately 80 patients with primary
or secondary warm AIHA who have failed at least one prior
treatment. The primary endpoint will be a durable hemoglobin
response by week 24, defined as Hgb > 10 g/dL and > 2 g/dL
greater than baseline and a durability of response measure, with
the response not being attributed to rescue therapy. Enrollment is
expected to take approximately 12 months. Additional details
regarding the Phase 3 clinical trial, including clinical trial
sites, can be found by visiting the clinicaltrials.gov website at
the following link.
In Rigel's SOAR Phase 2 clinical trial evaluating the safety and
efficacy of fostamatinib in patients with warm AIHA who did not
receive a meaningful benefit from at least one previous treatment,
9 out of 21 evaluable patients (43%) achieved the primary efficacy
endpoint at week 24. One additional patient was a late responder at
week 30, for a total of 10 out 21 evaluable patients (48%) that
achieved a response. The primary endpoint was defined as a
hemoglobin level of greater than 10 g/dl and at least a 2 g/dl
increase from baseline. The safety profile was consistent with the
existing fostamatinib safety database, including diarrhea and
hypertension as the most common adverse events. The open-label
extension period of the SOAR Phase 2 study is ongoing.
Currently, fostamatinib is commercially available in the U.S.
under the brand name TAVALISSE® (fostamatinib disodium
hexahydrate), which is the first and only spleen tyrosine kinase
(SYK) inhibitor indicated for the treatment of thrombocytopenia in
adult patients with chronic ITP who have had an insufficient
response to a previous treatment.
Rigel also announced the resignation of Anne-Marie Duliege,
executive vice president and chief medical officer, effective
August 31, 2019. The Company
has initiated an external search for its next chief medical officer
with a focus on expertise in the areas of hematology, oncology, and
rare diseases, as well as clinical trial and regulatory approval
experience at commercial stage companies.
Mr. Rodriguez stated, "On behalf of the entire company, I want
to thank Anne-Marie for her significant contributions during these
past few years at Rigel. She played an integral role in gaining our
first product approval in the U.S. and positioning us for a
potential European approval."
About AIHA
AIHA is a rare, serious blood
disorder in which 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 U.S. and can be a
severe, debilitating disease. To date, there are no
disease-targeted therapies approved for AIHA, despite the unmet
medical need that exists for these patients.
About 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. Common symptoms of ITP are excessive
bruising and bleeding. People suffering with chronic ITP may
live with an 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. However, not all
patients are adequately treated with existing therapies. As a
result, there remains a significant medical need for additional
treatment options for patients with ITP.
About TAVALISSE
Indication
TAVALISSE® (fostamatinib disodium hexahydrate)
tablets is indicated for the treatment of thrombocytopenia in adult
patients with chronic immune thrombocytopenia (ITP) who have had an
insufficient response to a previous treatment.
Important Safety Information
Warnings and Precautions
- Hypertension can occur with TAVALISSE treatment. Patients with
pre-existing hypertension may be more susceptible to the
hypertensive effects. Monitor blood pressure every 2 weeks until
stable, then monthly, and adjust or initiate antihypertensive
therapy for blood pressure control maintenance during therapy. If
increased blood pressure persists, TAVALISSE interruption,
reduction, or discontinuation may be required.
- Elevated liver function tests (LFTs), mainly ALT and AST, can
occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT
or AST increase to >3 x upper limit of normal, manage
hepatotoxicity using TAVALISSE interruption, reduction, or
discontinuation.
- Diarrhea occurred in 31% of patients and severe diarrhea
occurred in 1% of patients treated with TAVALISSE. Monitor patients
for the development of diarrhea and manage using supportive care
measures early after the onset of symptoms. If diarrhea becomes
severe (≥Grade 3), interrupt, reduce dose or discontinue
TAVALISSE.
- Neutropenia occurred in 6% of patients treated with TAVALISSE;
febrile neutropenia occurred in 1% of patients. Monitor the ANC
monthly and for infection during treatment. Manage toxicity with
TAVALISSE interruption, reduction, or discontinuation.
- TAVALISSE can cause fetal harm when administered to pregnant
women. Advise pregnant women the potential risk to a fetus. Advise
females of reproductive potential to use effective contraception
during treatment and for at least 1 month after the last dose.
Verify pregnancy status prior to initiating TAVALISSE. It is
unknown if TAVALISSE or its metabolite is present in human milk.
Because of the potential for serious adverse reactions in a
breastfed child, advise a lactating woman not to breastfeed during
TAVALISSE treatment and for at least 1 month after the last
dose.
Drug Interactions
- Concomitant use of TAVALISSE with strong CYP3A4 inhibitors
increases exposure to the major active metabolite of TAVALISSE
(R406), which may increase the risk of adverse reactions. Monitor
for toxicities that may require a reduction in TAVALISSE dose.
- It is not recommended to use TAVALISSE with strong CYP3A4
inducers, as concomitant use reduces exposure to R406.
- Concomitant use of TAVALISSE may increase concentrations of
some CYP3A4 substrate drugs and may require a dose reduction of the
CYP3A4 substrate drug.
- Concomitant use of TAVALISSE may increase concentrations of
BCRP substrate drugs (e.g., rosuvastatin) and P-Glycoprotein (P-gp)
substrate drugs (e.g., digoxin), which may require a dose reduction
of the BCRP and P-gp substrate drug.
Adverse Reactions
- Serious adverse drug reactions in the ITP double-blind studies
were febrile neutropenia, diarrhea, pneumonia, and hypertensive
crisis, which occurred in 1% of TAVALISSE patients. In addition,
severe adverse reactions occurred including dyspnea and
hypertension (both 2%), neutropenia, arthralgia, chest pain,
diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache,
syncope, and hypoxia (all 1%).
- Common adverse reactions (≥5% and more common than placebo)
from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea,
dizziness, ALT and AST increased, respiratory infection, rash,
abdominal pain, fatigue, chest pain, and neutropenia.
Please see www.TAVALISSE.com for full Prescribing
Information.
To report side effects of prescription drugs to
the FDA, visit www.fda.gov/medwatch or call
1-800-FDA-1088 (800-332-1088).
TAVALISSE is a registered trademark of Rigel
Pharmaceuticals, Inc.
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
hematologic disorders, cancer and rare diseases. Rigel's pioneering
research focuses on signaling pathways that are critical to disease
mechanisms. The company's first FDA approved product is TAVALISSE®
(fostamatinib disodium hexahydrate), the only oral spleen tyrosine
kinase (SYK) inhibitor, for the treatment of adult patients with
chronic immune thrombocytopenia who have had an insufficient
response to a previous treatment. Rigel's current clinical programs
include a Phase 3 study of TAVALISSE in autoimmune hemolytic anemia
(AIHA) and Phase 1 study of R835, a proprietary molecule from its
interleukin receptor associated kinase (IRAK) program. In addition,
Rigel has product candidates in clinical development with partners
BerGenBio ASA, Daiichi Sankyo, Aclaris Therapeutics, and
AstraZeneca.
1 The product for this use or indication is
investigational and has not been proven safe or effective by any
regulatory authority.
Forward Looking Statements
This release contains forward-looking statements relating to,
among other things, the utility of fostamatinib in other
indications, including warm autoimmune hemolytic anemia and other
indications; Rigel's belief that fostamatinib may be an important
alternative for patients with ITP or AIHA; the potential
opportunity for fostamatinib to obtain approval in the EU; the
management and advancement of Rigel's clinical programs; and the
design, timing and 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," "anticipate," 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, risks and uncertainties associated
with the commercialization and marketing of TAVALISSE; risks that
the FDA, EMA or other regulatory authorities may make adverse
decisions regarding fostamatinib; risks that TAVALISSE clinical
trials may not be predictive of real-world results or of results in
subsequent clinical trials; risks that TAVALISSE may have
unintended side effects, adverse reactions or incidents of misuses;
the availability of resources to develop Rigel's product
candidates; market competition; 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 March 31, 2019.
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: David Burke
Phone: 650.624.1232
Email: dburke@rigel.com
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SOURCE Rigel Pharmaceuticals, Inc.