SOUTH
SAN FRANCISCO, Calif., Dec. 2, 2024
/PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a
commercial stage biotechnology company focused on hematologic
disorders and cancer, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation to R289 for
the treatment of patients with previously-treated transfusion
dependent lower-risk myelodysplastic syndrome (LR-MDS).
R2891, Rigel's potent and selective dual inhibitor of
IRAK1 and IRAK4, is being studied in an ongoing Phase 1b study evaluating the safety, tolerability,
pharmacokinetics and preliminary activity in patients with LR-MDS
who are relapsed or refractory to prior therapies.
"We are pleased that R289 has been granted Fast Track
designation, which underscores the significant unmet need for
patients with transfusion dependent lower-risk MDS," said
Raul Rodriguez, Rigel's president
and CEO. "By targeting inflammatory signaling, we believe that R289
has the potential to meaningfully improve the lives of those living
with this disease."
"Lower-risk MDS affects a primarily elderly patient population
that faces progressive cytopenias, particularly anemia, and
treatment options for transfusion-dependent patients are limited,"
said Lisa Rojkjaer, M.D., Rigel's chief medical officer. "This
designation is based on initial data from the ongoing Phase
1b study and highlights the potential
of R289 to be a new therapeutic option for these patients. We look
forward to working closely with the FDA to advance the clinical
development of R289."
Fast track is a process designed to facilitate the development
and expedite the review of drugs to treat serious conditions and
fill an unmet medical need. A drug that receives Fast Track
designation may benefit from more frequent interactions with the
FDA over the course of drug development. In addition, the Fast
Track program allows for eligibility for Accelerated Approval and
Priority Review, if relevant criteria are met.
About R289
R289 is a prodrug of R835, an IRAK1/4 dual inhibitor, which
has been shown in preclinical studies to block inflammatory
cytokine production in response to toll-like receptor (TLR) and
interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs
play a critical role in the innate immune response and
dysregulation of these pathways can lead to various inflammatory
conditions. Chronic stimulation of both these receptor systems is
thought to cause the pro-inflammatory environment in the bone
marrow responsible for persistent cytopenias in lower-risk MDS
patients.2
About Rigel
Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology
company dedicated to discovering, developing and providing novel
therapies that significantly improve the lives of patients with
hematologic disorders and cancer. Founded in 1996, Rigel is based
in South San Francisco,
California. For more information on Rigel, the Company's
marketed products and pipeline of potential products,
visit www.rigel.com.
- R289 is an investigational compound not approved by the
FDA.
- Sallman DA et al. Unraveling the Pathogenesis of MDS:
The NLRP3 Inflammasome and Pyroptosis Drive the MDS Phenotype.
Front Oncol. June 16, 2016.
DOI: https://doi.org/10.3389/fonc.2016.0015
Forward-Looking
Statements
This press
release contains forward-looking statements relating to, among
other things, the potential benefits of
Fast Track
designation for R289
for the treatment of patients with lower-risk myelodysplastic
syndrome (LR-MDS), its potential as a therapeutic, the existence of
patients with an unmet medical need for such therapy, the potential
for such therapy to meaningfully improve the lives of such
patients, and Rigel's
ability to further develop its clinical stage
product candidates, including the progress of
current and potential future clinical trials of
R289. Any statements
contained in this press release that are not statements of
historical fact may be deemed to be forward-looking statements and
as such are intended to be covered by the safe harbor for
"forward-looking statements" provided by the PSLRA. Forward-looking
statements can be identified by words such as "plan", "potential",
"may", "look to", "expects", "will" and similar expressions in
reference to future periods. Forward-looking statements are neither
historical facts nor assurances of future performance. Instead,
they are based on Rigel's current beliefs, expectations, and
assumptions and hence they inherently involve significant risks,
uncertainties and changes in circumstances that are difficult
to predict and many of which are outside of Rigel's
control. Therefore, you should not rely on any of these
forward-looking statements. 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, Fast Track
designation may not result in a more expedited development or
regulatory review process, and such a designation does not increase
the likelihood that R289 will receive marketing approval in
the United States; Fast Track
designation does not change the standards for regulatory approval;
the FDA may later decide that R289 no longer meets the conditions
for qualification or decide that the time period for FDA review or
approval will not be shortened; risks and uncertainties associated
with the commercialization and marketing of R289; risks that the
FDA or other regulatory authorities may make adverse decisions
regarding R289; risks that clinical trials may not be predictive of
real-world results or of results in subsequent clinical trials;
risks that R289 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 September
30, 2024 and subsequent filings. Any forward-looking
statement made by us in this press release is based only on
information currently available to us and speaks only as of the
date on which it is made. Rigel does not undertake any obligation
to update forward-looking statements, whether written or oral, that
may be made from time to time, whether as a result of new
information, future developments or otherwise, and expressly
disclaims any obligation or undertaking to release publicly any
updates or revisions to any forward-looking statements contained
herein, except as required by law.
Contact for Investors &
Media:
Investors:
Rigel Pharmaceuticals,
Inc.
650.624.1232
ir@rigel.com
Media:
David
Rosen
Argot Partners
646.461.6387
david.rosen@argotpartners.com
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SOURCE Rigel Pharmaceuticals, Inc.