SOUTH
SAN FRANCISCO, Calif., Jan. 9, 2025
/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 Orphan Drug designation to R289
for the treatment of myelodysplastic syndromes (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.
"Receiving Orphan Drug designation for R289 supports the
development of this therapeutic candidate for the treatment of MDS
and highlights the significant unmet medical need that exists for
these patients," said Raul
Rodriguez, Rigel's president and CEO. "Orphan Drug and Fast
Track designations, along with encouraging initial data from our
ongoing Phase 1b study in patients
with lower-risk MDS, represent significant milestones in the
advancement of R289 as a potential new treatment option."
The FDA Office of Orphan Products Development grants orphan drug
designation to support the development of medicines for rare
disorders that affect fewer than 200,000 people in the U.S. Under
the Orphan Drug Act, orphan drug designation qualifies a company
for incentives including tax credits, exemptions from certain FDA
fees for clinical trials and the potential for seven years of
market exclusivity following drug approval.
R289 was previously granted Fast Track designation by the
FDA for the treatment of patients with previously-treated
transfusion dependent lower-risk MDS.
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.2016.00151
Forward Looking Statements
This press release contains forward-looking statements relating
to, among other things, the possible advantages of Orphan Drug and
Fast Track designations, the potential benefits of R289 as a
therapeutic for MDS and LR-MDS, the existence of patients with an
unmet medical need for such therapy, certain potential benefits
associated with orphan drug designation, and Rigel's ability to
further develop its clinical stage product candidates, including
the encouragement of initial data from, and progress of, current
clinical trials of R289, and the potential for future trials. 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 and Orphan Drug
designations 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 and
Orphan Drug designations do 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.