- Preliminary fourth quarter 2023 total revenue of
approximately $35.7 million which
includes record TAVALISSE® net product sales of
$25.7 million and
REZLIDHIA® net product sales of $3.9 million
- Strategic alliance with MD Anderson to advance REZLIDHIA in
AML and other cancers
- Collaboration with CONNECT to evaluate REZLIDHIA in a Phase
2 clinical trial in glioma
SOUTH
SAN FRANCISCO, Calif., Jan. 8, 2024
/PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL)
today provided a business update including preliminary total
revenue for the fourth quarter of 2023, ongoing activity from the
commercial portfolio, including TAVALISSE® (fostamatinib
disodium hexahydrate) tablets and REZLIDHIA®
(olutasidenib) capsules, and upcoming catalysts for 2024.
"2023 was a year of significant growth across our commercial
hematology-oncology portfolio. We grew TAVALISSE net product
sales nearly 24% over 2022 and successfully achieved more than
$10 million of REZLIDHIA
revenue during its first full year of launch. This
enabled us to generate more than $104
million of total net product sales this year," said
Raul Rodriguez, Rigel's president
and CEO. "Also, our recent collaborations with MD Anderson and
CONNECT will allow us to evaluate the potential of REZLIDHIA as a
possible therapy in a broad range of IDH1-mutant cancers. As we
head into the future, we are focused on commercial execution, the
advancement of our hematology-oncology pipeline, and our plan to
reach financial breakeven."
Commercial and Preliminary Financial Update
In the
fourth quarter of 2023, a total of 2,671 bottles of TAVALISSE were
sold in the U.S., of which, 2,463 bottles were shipped directly to
patients and clinics, representing the highest daily bottles
shipped to patients and clinics in a quarter since launch. While
Rigel is still determining final results for the fourth quarter of
2023, it expects to report net product sales of TAVALISSE of
$25.7 million for the fourth quarter
compared to $21.9 million for the
same period of 2022.
In the fourth quarter of 2023, a total of 308 bottles of
REZLIDHIA were sold in the U.S., of which, 278 bottles were shipped
directly to patients and clinics. While Rigel is still determining
final results for the fourth quarter of 2023, it expects to report
net product sales of REZLIDHIA of $3.9
million for the fourth quarter compared to $0.9 million for the same period of 2022.
Overall, Rigel expects to report net product sales of
$104.3 million in 2023, representing
36% growth over 2022.
Contract revenues for the fourth quarter of 2023 are expected to
be approximately $6.1 million,
consisting of $6.0 million in
contract revenue from collaborations and $0.1 million in government contract revenue.
Contract revenue from collaborations is expected to include
$3.7 million of revenue from Grifols
S.A., related to delivery of drug supplies and earned royalties, as
well as $2.2 million of revenue from
Kissei Pharmaceutical Co., Ltd. and $0.1
million of revenue from Medison Pharma Trading AG, related
to delivery of drug supplies.
For the fourth quarter of 2023, Rigel expects to report total
revenue of approximately $35.7
million.
Rigel expects to report cash, cash equivalents, and short-term
investments of approximately $56.9
million as of December 31,
2023, compared to $58.2
million as of December 31,
2022.
The above information is preliminary, has not been audited, and
is subject to change upon the audit of Rigel's financial statements
for the year ended December 31, 2023.
Rigel expects to provide complete fourth quarter and full year 2023
financial results in March 2024.
Q4 Business Update
- Rigel and The University of Texas
MD Anderson Cancer Center (MD Anderson) recently
announced a multi-year strategic development collaboration to
expand the evaluation of REZLIDHIA (olutasidenib) in
acute myeloid leukemia (AML) and other hematologic cancers. Under
the strategic collaboration, Rigel and MD Anderson will evaluate
the potential of olutasidenib to treat newly diagnosed and relapsed
or refractory (R/R) patients with AML, higher-risk myelodysplastic
syndromes (MDS), and advanced myeloproliferative neoplasms (MPN),
in combination with other agents. The collaboration will also
support the evaluation of olutasidenib as monotherapy in
lower-risk MDS and maintenance therapy in post-hematopoietic stem
cell transplant (HSCT) patients. Rigel will provide $15 million in time-based milestone payments and
study material over the five-year collaboration.
- Rigel and CONNECT recently announced a collaboration
to evaluate REZLIDHIA (olutasidenib) in combination with
temozolomide as maintenance therapy in patients with high-grade
glioma (HGG) harboring an isocitrate dehydrogenase-1 (IDH1)
mutation. Under the collaboration, CONNECT will include
olutasidenib in CONNECT's TarGeT-D, a molecularly guided Phase 2
umbrella clinical trial for HGG. The Rigel-sponsored arm will study
post-radiotherapy administration of olutasidenib in combination
with temozolomide followed by olutasidenib monotherapy as
maintenance treatment in newly diagnosed pediatric and young adult
patients (<39 years old) with IDH1 mutation positive HGG,
including diffuse intrinsic pontine glioma (DIPG), an aggressive
brain tumor with limited treatment options. Rigel will provide
funding up to $3 million and study
material over the four-year collaboration.
- In December 2023, Rigel presented
four posters highlighting data from the Company's commercial
and clinical-stage hematology-oncology portfolio at the 65th
American Society of Hematology Annual Meeting and Exposition.
Included was a poster, Abstract #2888, reporting post hoc analyses
in a subset of patients with mIDH1 R/R AML or MDS that were
R/R to HSCT, ivosidenib, or venetoclax. The analyses suggest
that olutasidenib alone or in combination with azacitidine may
induce complete remissions in these patients.
- Rigel continues to advance its open-label, Phase 1b clinical trial of R2891, an
investigational, potent, and selective IRAK1/4 inhibitor, in
patients with lower-risk myeloid dysplastic syndrome (LR-MDS) who
are refractory/resistant to prior therapies. The primary endpoint
for this trial is safety with key secondary endpoints including
preliminary efficacy and evaluation of pharmacokinetic
properties. Rigel is currently enrolling patients in the third
cohort.
About ITP
In patients with ITP (immune
thrombocytopenia), 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 (TPO-RAs), and
splenectomy. However, not all patients respond to existing
therapies. As a result, there remains a significant medical need
for additional treatment options for patients with ITP.
About AML
Acute myeloid leukemia (AML) is a rapidly
progressing cancer of the blood and bone marrow that affects
myeloid cells, which normally develop into various types of mature
blood cells. AML occurs primarily in adults and accounts for about
1 percent of all adult cancers. The American Cancer Society
estimates that in the United
States alone, there were about 20,380 new cases, most in
adults, in 2023.2
Relapsed AML affects about half of all patients who, following
treatment and remission, experience a return of leukemia cells in
the bone marrow.3 Refractory AML, which affects
between 10 and 40 percent of newly diagnosed patients, occurs when
a patient fails to achieve remission even after intensive
treatment.4 Quality of life declines for patients
with each successive line of treatment for AML, and well-tolerated
treatments in relapsed or refractory disease remain an unmet
need.
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.5
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 (eg, rosuvastatin) and P-Glycoprotein (P-gp)
substrate drugs (eg, 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.TAVALISSEUSPI.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
REZLIDHIA®
INDICATION
REZLIDHIA
is indicated for the treatment of adult patients with relapsed or
refractory acute myeloid leukemia (AML) with a susceptible
isocitrate dehydrogenase-1 (IDH1) mutation as detected by an
FDA-approved test.
IMPORTANT SAFETY INFORMATION
WARNING:
DIFFERENTIATION SYNDROME Differentiation syndrome, which
can be fatal, can occur with REZLIDHIA treatment. Symptoms may
include dyspnea, pulmonary infiltrates/pleuropericardial effusion,
kidney injury, hypotension, fever, and weight gain. If
differentiation syndrome is suspected, withhold REZLIDHIA and
initiate treatment with corticosteroids and hemodynamic monitoring
until symptom resolution.
|
WARNINGS AND PRECAUTIONS
Differentiation
Syndrome
REZLIDHIA can cause differentiation syndrome. In
the clinical trial of REZLIDHIA in patients with relapsed or
refractory AML, differentiation syndrome occurred in 16% of
patients, with grade 3 or 4 differentiation syndrome occurring in
8% of patients treated, and fatalities in 1% of patients.
Differentiation syndrome is associated with rapid proliferation and
differentiation of myeloid cells and may be life-threatening or
fatal. Symptoms of differentiation syndrome in patients treated
with REZLIDHIA included leukocytosis, dyspnea, pulmonary
infiltrates/pleuropericardial effusion, kidney injury, fever,
edema, pyrexia, and weight gain. Of the 25 patients who experienced
differentiation syndrome, 19 (76%) recovered after treatment or
after dose interruption of REZLIDHIA. Differentiation syndrome
occurred as early as 1 day and up to 18 months after REZLIDHIA
initiation and has been observed with or without concomitant
leukocytosis.
If differentiation syndrome is suspected, temporarily withhold
REZLIDHIA and initiate systemic corticosteroids (e.g.,
dexamethasone 10 mg IV every 12 hours) for a minimum of 3 days and
until resolution of signs and symptoms. If concomitant
leukocytosis is observed, initiate treatment with hydroxyurea, as
clinically indicated. Taper corticosteroids and hydroxyurea after
resolution of symptoms. Differentiation syndrome may recur with
premature discontinuation of corticosteroids and/or hydroxyurea
treatment. Institute supportive measures and hemodynamic monitoring
until improvement; withhold dose of REZLIDHIA and consider dose
reduction based on recurrence.
Hepatotoxicity
REZLIDHIA can cause hepatotoxicity,
presenting as increased alanine aminotransferase (ALT), increased
aspartate aminotransferase (AST), increased blood alkaline
phosphatase, and/or elevated bilirubin. Of 153 patients with
relapsed or refractory AML who received REZLIDHIA, hepatotoxicity
occurred in 23% of patients; 13% experienced grade 3 or 4
hepatotoxicity. One patient treated with REZLIDHIA in combination
with azacitidine in the clinical trial, a combination for which
REZLIDHIA is not indicated, died from complications of drug-induced
liver injury. The median time to onset of hepatotoxicity in
patients with relapsed or refractory AML treated with REZLIDHIA was
1.2 months (range: 1 day to 17.5 months) after REZLIDHIA
initiation, and the median time to resolution was 12 days (range: 1
day to 17 months). The most common hepatotoxicities were elevations
of ALT, AST, blood alkaline phosphatase, and blood bilirubin.
Monitor patients frequently for clinical symptoms of hepatic
dysfunction such as fatigue, anorexia, right upper abdominal
discomfort, dark urine, or jaundice. Obtain baseline liver function
tests prior to initiation of REZLIDHIA, at least once weekly for
the first two months, once every other week for the third month,
once in the fourth month, and once every other month for the
duration of therapy. If hepatic dysfunction occurs, withhold,
reduce, or permanently discontinue REZLIDHIA based on
recurrence/severity.
ADVERSE REACTIONS
The most common (≥20%) adverse
reactions, including laboratory abnormalities, were aspartate
aminotransferase increased, alanine aminotransferase increased,
potassium decreased, sodium decreased, alkaline phosphatase
increased, nausea, creatinine increased, fatigue/malaise,
arthralgia, constipation, lymphocytes increased, bilirubin
increased, leukocytosis, uric acid increased, dyspnea, pyrexia,
rash, lipase increased, mucositis, diarrhea and transaminitis.
DRUG INTERACTIONS
- Avoid concomitant use of REZLIDHIA with strong or moderate
CYP3A inducers.
- Avoid concomitant use of REZLIDHIA with sensitive CYP3A
substrates unless otherwise instructed in the substrates
prescribing information. If concomitant use is unavoidable, monitor
patients for loss of therapeutic effect of these drugs.
LACTATION
Advise women not to breastfeed during
treatment with REZLIDHIA and for 2 weeks after the last dose.
GERIATRIC USE
No overall differences in effectiveness
were observed between patients 65 years and older and younger
patients. Compared to patients younger than 65 years of age, an
increase in incidence of hepatotoxicity and hypertension was
observed in patients ≥65 years of age.
HEPATIC IMPAIRMENT
In patients with mild or moderate
hepatic impairment, closely monitor for increased probability of
differentiation syndrome.
Click here for Full Prescribing Information,
including Boxed WARNING.
To report side effects of prescription drugs to the FDA, visit
www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).
REZLIDHIA is a registered trademark of Rigel Pharmaceuticals,
Inc.
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.
- The American Cancer Society. Key Statistics for Acute Myeloid
Leukemia (AML). Revised January 12,
2023. Accessed Feb. 15, 2023:
https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html
- Leukaemia Care. Relapse in Acute Myeloid Leukaemia (AML).
Version 3. Reviewed October 2021.
Accessed Feb 15, 2023:
https://media.leukaemiacare.org.uk/wp-content/uploads/Relapse-in-Acute-Myeloid-Leukaemia-AML-Web-Version.pdf
- Thol F, Schlenk RF, Heuser M, Ganser A. How I treat
refractory and early relapsed acute myeloid leukemia. Blood
(2015) 126 (3): 319-27. doi:
https://doi.org/10.1182/blood-2014-10-551911
- 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, expected commercial and financial results for
the fourth quarter and fiscal year ended December 31, 2023, expectations related
to the potential and market opportunity of olutasidenib
as therapeutics for R/R AML and other conditions, the
commercialization of fostamatinib or olutasidenib in the U.S. and
international markets, and Rigel's ability to further
develop its clinical stage product candidates and Rigel's
partnering and collaboration efforts, including the progress of
Phase 1b clinical trial of R289 for
the treatment of lower-risk myeloid dysplastic syndrome,
olutasidenib's evaluation in acute myeloid leukemia
(AML) and other hematologic cancers, and in newly diagnosed
pediatric and young adult patients with high-grade glioma (HGG)
harboring an isocitrate dehydrogenase-1 (IDH1) mutation. Any
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Forward-looking statements can be identified by words such as
"plan", "potential", "may", "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 our 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, risks and
uncertainties associated with the commercialization and marketing
of fostamatinib or olutasidenib; risks that the FDA, European
Medicines Agency, PMDA or other regulatory authorities may make
adverse decisions regarding fostamatinib or olutasidenib; risks
that clinical trials may not be predictive of real-world results or
of results in subsequent clinical trials; risks that fostamatinib
or olutasidenib 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, 2023 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
212.600.1902
david.rosen@argotpartners.com
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SOURCE Rigel Pharmaceuticals, Inc.