SOUTH SAN FRANCISCO, Calif.,
May 5, 2021 /PRNewswire/
-- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today
reported financial results for the first quarter ended March 31, 2021, including sales of
TAVALISSE® (fostamatinib disodium hexahydrate) tablets,
for the treatment of adults with chronic immune thrombocytopenia
(ITP) who have had an insufficient response to a previous
treatment.
"We continued to make significant progress in achieving
important corporate and clinical milestones in the first
quarter, which is a true testament to the dedication of our team to
bring meaningful treatment options to those patients who need them
the most," said Raul Rodriguez,
Rigel's president and CEO. "Following our recent announcement of
positive topline data from the Phase 2 trial of fostamatinib in
hospitalized patients with COVID-19 and our partnership with Eli
Lilly for our RIP1 inhibitor program, we are excited by the
prospects that the rest of 2021 holds for Rigel."
Business Update
In April
2021, Rigel reported positive topline data from the
multi-center, Phase 2 clinical trial evaluating the safety of
fostamatinib, Rigel's oral spleen tyrosine kinase (SYK) inhibitor,
for the treatment of hospitalized patients with COVID-19. The trial
met its primary endpoint of safety, and showed broad and consistent
improvement in numerous efficacy endpoints including mortality,
ordinal scale assessment, and number of days in the ICU. The trial
was conducted in collaboration with the National Heart, Lung, and
Blood Institute (NHLBI), part of the National Institutes of Health
(NIH), and Inova Health System. The NHLBI is expected to
publish a full analysis of the trial data in a peer-reviewed
journal. Rigel is discussing these results with health authorities,
including the U.S. Food and Drug Administration (FDA), and intends
to apply for Emergency Use Authorization (EUA) for fostamatinib for
the treatment of hospitalized COVID-19 patients.
Rigel's Phase 3 clinical trial to further evaluate fostamatinib
in hospitalized patients with COVID-19 is currently enrolling.
Rigel was awarded $16.5 million from
the U.S. Department of Defense's (DOD) Joint Program Executive
Office for Chemical, Biological, Radiological and Nuclear Defense
(JPEO-CBRND) to support this Phase 3 clinical trial. The study is
designed to evaluate fostamatinib for prevention of progression to
severe disease in hospitalized patients with COVID-19 without
respiratory failure that have certain high-risk prognostic factors.
This multi-center, double-blind, placebo-controlled study will
randomly assign patients to either fostamatinib plus standard of
care (SOC) or matched placebo plus SOC (1:1). Treatment will be
administered orally twice daily for 14 days. There will be a
follow-up period to day 60. The primary endpoint of this study is
the proportion of subjects who progress to severe/critical disease
within 29 days. In addition, Rigel's COVID-19 program
includes an investigator-sponsored trial currently being conducted
by Imperial College London.
Rigel's FORWARD study, a Phase 3 pivotal trial of TAVALISSE in
patients with warm autoimmune hemolytic anemia (wAIHA), has
enrolled 72 of 90 patients as of May 5,
2021. If approved, TAVALISSE has the potential to be the
first to market therapy for patients with wAIHA. The FDA has
granted Fast Track designation as well as Orphan Drug designation
to TAVALISSE for the treatment of wAIHA.
In the first quarter of 2021, 1,599 bottles of TAVALISSE were
shipped to patients and clinics, an increase of 14% year over year.
Net product sales for the first quarter decreased 2% year over year
to $12.4 million. During the
quarter, the company experienced typical first quarter
reimbursement issues such as the resetting of co-pays and the
Medicare donut hole, along with physician and patient access issues
created by the COVID-19 pandemic. Incrementally, the company's
net product sales were negatively impacted by a significant 235
bottle decrease in bottles remaining in its distribution channels
compared to Q4 2020.
In April 2021, Rigel received a
$125 million upfront cash payment
from its collaboration with Eli Lilly and Company (Lilly),
following the expiration of the waiting period under the Hart-Scott
Rodino Antitrust Improvements Act of 1976. In February 2021, Rigel and Lilly entered into a
global exclusive license agreement and strategic collaboration to
develop and commercialize Rigel's R552, a receptor-interacting
serine/threonine-protein kinase 1 (RIP1) inhibitor, for all
indications including autoimmune and inflammatory diseases.
Rigel and Lilly are currently focused on the planning for the
initiation of a Phase 2 clinical trial. Pursuant to the
collaboration, Lilly will also lead all clinical development of
central nervous system (CNS) penetrating RIP1 inhibitors. Rigel is
eligible to receive up to an additional $835
million in potential development, regulatory and commercial
milestone payments, as well as tiered royalties that will vary
depending upon Rigel's clinical development investment.
Rigel is also advancing the development of its IRAK1/4 program,
where it intends to pursue both hematology/oncology and rare immune
disease opportunities. Rigel has begun discussions with the FDA
regarding initiating a Phase 2 clinical trial in low-risk
myelodysplastic syndrome (MDS) and is also in discussions regarding
academic medical collaborations in this indication. In rare immune
diseases, the company is exploring opportunities including
palmoplantar pustulosis (PPP), hidradenitis suppurativa (HS), and
others.
Rigel's partner Kissei Pharmaceutical Co., Ltd. (Kissei) has
completed enrollment of its Phase 3 clinical trial of fostamatinib
in adult Japanese patients with chronic ITP. In October 2018, Rigel and Kissei entered into an
exclusive agreement to develop and commercialize fostamatinib in
all current and potential indications in Japan, China,
Taiwan, and the Republic of
Korea.
Financial Update
For the first quarter of 2021, Rigel
reported net income of $39.5 million,
or $0.23 per basic share and
$0.22 per diluted share, compared to
net income of $21.2 million, or
$0.13 per basic and diluted share,
for the same period of 2020.
In the first quarter of 2021, total revenues were $81.0 million, consisting of $12.4 million in TAVALISSE net product sales,
$65.6 million in contract revenues
from collaborations and $3.0 million
in government contract revenue.
Contract revenues from collaborations of $65.6 million for the first quarter of 2021
consisted of $60.6 million in revenue
related to Rigel's license agreement with Lilly, $4.0 million in revenue related to the grant of
non-exclusive license of a certain patent to an unrelated
third-party company, and $1.0 million
in revenue for the delivery of drug supply under its collaboration
agreement with Grifols. Government contract revenue was related to
the income that Rigel recognized pursuant to the agreement it
entered into in January 2021 with the
DOD to support Rigel's ongoing Phase 3 clinical trial of
fostamatinib in hospitalized patients with COVID-19.
Rigel reported total costs and expenses of $39.3 million in the first quarter of 2021,
compared to $34.7 million for the
same period in 2020. The increase in costs and expenses was
primarily due to increases in personnel-related costs, stock-based
compensation expense, and research and development costs related to
its ongoing Phase 3 clinical trial in hospitalized patients with
COVID-19 and development of its IRAK 1/4 inhibitor program,
partially offset by the decrease in research and development costs
due to the completion of a Phase 1 clinical trial for its RIP1
inhibitor program.
As of March 31, 2021, Rigel had
cash, cash equivalents and short-term investments of $39.3 million, compared to $57.3 million as of December 31, 2020. Cash, cash equivalents
and short-term investments as of March 31,
2021, does not include the $125.0
million upfront payment received from Lilly in April 2021.
Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live
conference call and webcast today at 4:30pm
Eastern Time (1:30pm Pacific
Time).
Participants can access the live conference call by dialing
(877) 407-3088 (domestic) or (201) 389-0927 (international). The
conference call and accompanying slides will also be webcast live
and can be accessed from the Investor Relations section of the
company's website at www.rigel.com. The webcast will be archived
and available for replay after the call via the Rigel website.
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 AIHA
Autoimmune hemolytic anemia (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 45,000 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. Warm antibody
AIHA (wAIHA), the most common form of AIHA, is characterized by the
presence of antibodies that react with the red blood cell surface
at body temperature.
About COVID-19 & SYK Inhibition
COVID-19 is the
infectious disease caused by Severe Acute Respiratory Syndrome
Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the
upper and lower respiratory tract and can lead to acute respiratory
distress syndrome (ARDS). Additionally, some patients develop other
organ dysfunction including myocardial injury, acute kidney injury,
shock resulting in endothelial dysfunction and subsequently micro
and macrovascular thrombosis.1 Much of the underlying
pathology of SARS-CoV-2 is thought to be secondary to a
hyperinflammatory immune response associated with increased risk of
thrombosis.2
SYK is involved in the intracellular signaling pathways of many
different immune cells. Therefore, SYK inhibition may improve
outcomes in patients with COVID-19 via inhibition of key Fc gamma
receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers
of pathology, such as inflammatory cytokine release by monocytes
and macrophages, production of neutrophil extracellular traps
(NETs) by neutrophils, and platelet aggregation.3,4,5
Furthermore, SYK inhibition in neutrophils and platelets may lead
to decreased thromboinflammation, alleviating organ dysfunction in
critically ill patients with COVID-19.
For more information on Rigel's comprehensive clinical program
in COVID-19, go to:
https://www.rigel.com/pipeline/proprietary-programs/covid-19
About R552
The investigational candidate, R552,
is an orally available, potent and selective inhibitor of
receptor-interacting serine/threonine-protein kinase 1 (RIP1). RIP1
is believed to play a critical role in necroptosis. Necroptosis is
a form of regulated cell death where the rupturing of cells leads
to the dispersion of their inner contents, which induces immune
responses and enhances inflammation. In preclinical studies, R552
prevented joint and skin inflammation in a RIP1-mediated murine
model of inflammation and tissue damage. The safety and efficacy of
R552 has not been established by the FDA or any healthcare
authority.
About R835
The investigational candidate, R835, is an
orally available, potent and selective inhibitor of IRAK1 and IRAK4
that has been shown preclinically to block inflammatory cytokine
production in response to toll-like receptor (TLR) and the
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 a variety of
inflammatory pathological conditions. R835 treatment demonstrates
amelioration of clinical symptoms in multiple rodent models of
inflammatory disease including psoriasis, arthritis, lupus,
multiple sclerosis and gout. The safety and efficacy of R835 has
not been established by the FDA or any healthcare authority.
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.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 and TAVLESSE are registered trademarks 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 hematologic
disorders, cancer and rare immune 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) tablets,
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. The
product is also commercially available in Europe (TAVLESSE) and Canada (TAVALISSE) for the treatment of
chronic immune thrombocytopenia in adult patients.
Fostamatinib is currently being studied in a Phase 3 clinical
trial for the treatment of warm autoimmune hemolytic anemia
(wAIHA)6; a Phase 3 clinical trial for the treatment of
hospitalized patients with COVID-196; an
NIH/NHLBI-sponsored Phase 2 clinical trial for the treatment of
hospitalized patients with COVID-19, in collaboration with Inova
Health System; and a Phase 2 clinical trial for the treatment of
COVID-19 being conducted by Imperial College London.
Rigel's other clinical programs include its interleukin
receptor-associated kinase (IRAK) inhibitor program, and a
receptor-interacting serine/threonine-protein kinase (RIP1)
inhibitor program in clinical development with partner Eli Lilly
and Company. In addition, Rigel has product candidates in
development with partners AstraZeneca, BerGenBio ASA, and Daiichi
Sankyo.
1. Berlin DA, Gulick RM, Martinez FJ. Severe Covid-19. N Engl J
Med 2020
2. Becker RC. COVID-19 Update: COVID-19 associated coagulopathy.
Journal of Thrombosis and Thrombolysis May
15, 2020. DOI:
https://doi.org/10.1007/s11239-020-02134-3
3. Hoepel W. et al. Anti-SARS-CoV-2 IgG from severely ill COVID-19
patients promotes macrophage hyper-inflammatory responses. bioRxiv
July 13, 2020. DOI:
https://doi.org/10.1101/2020.07.13.190140
4. Sung P-S and Hsieh S-L (2019) CLEC2 and CLEC5A: Pathogenic Host
Factors in Acute Viral Infections. Front. Immunol. 10:2867. DOI:
https://doi.org/10.3389/fimmu.2019.02867
5. Behnen M. Immobilized Immune Complexes Induce Neutrophil
Extracellular Trap Release by Human Neutrophil Granulocytes via Fcγ
RIIIB and Mac-1. The Journal of Immunology July 2014. DOI:
https://doi.org/10.4049/jimmunol.1400478
6. 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
commercial success of TAVALISSE in the U.S. and TAVLESSE in
Europe; Rigel's intention to apply
for EUA for fostamatinib for the treatment of hospitalized COVID-19
patients; Rigel's ability to achieve development, regulatory and
commercial milestone payments, as well as tiered royalties;
expectations related to the market opportunity for fostamatinib as
a COVID-19 therapeutic; Rigel's ability to further develop its
clinical stage and early stage product candidates and programs; and
Rigel's partnering efforts. 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 "potential", "may",
"expects", 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 Annual
Report on Form 10-K for the year ended December 31, 2020. In addition, the COVID-19
pandemic may result in further delays in Rigel's studies, trials
and sales, or impact Rigel's ability to obtain supply of TAVALISSE.
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.
Investor Contact:
Jodi Sievers - Rigel
Pharmaceuticals
Phone: 650.624.1232
Email: ir@rigel.com
Media Contact:
Emily Correia - Syneos Health
Phone: 508-314-3157
Email: emily.correia@syneoshealth.com
RIGEL
PHARMACEUTICALS, INC.
|
STATEMENTS OF
OPERATIONS
|
(in thousands,
except per share amounts)
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
March 31,
|
|
|
2021
|
2020
|
|
|
(unaudited)
|
|
|
|
|
Revenues:
|
|
|
|
Product sales,
net
|
$
12,376
|
$
12,680
|
|
Contract revenues
from collaborations
|
65,642
|
43,081
|
|
Government
contract
|
3,000
|
—
|
|
Total
revenues
|
81,018
|
55,761
|
|
|
|
|
Costs and
expenses:
|
|
|
|
Cost of product
sales
|
316
|
155
|
|
Research and
development (see Note A)
|
16,826
|
16,149
|
|
Selling, general and
administrative (see Note A)
|
22,121
|
18,430
|
|
Total costs and
expenses
|
39,263
|
34,734
|
|
|
|
|
Income from
operations
|
41,755
|
21,027
|
|
Interest
income
|
1
|
358
|
|
Interest
expense
|
(485)
|
(142)
|
Income before income
taxes
|
41,271
|
21,243
|
Provision for income
taxes
|
1,771
|
-
|
Net income
|
$
39,500
|
$
21,243
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
|
|
Basic
|
$
0.23
|
$
0.13
|
|
Diluted
|
$
0.22
|
$
0.13
|
|
|
|
|
Weighted average
shares used in computing net loss per share, basic and
diluted
|
|
|
|
Basic
|
169,800
|
168,469
|
|
Diluted
|
176,069
|
168,568
|
|
|
|
|
|
|
|
|
Note
A
|
|
|
|
|
|
|
Stock-based
compensation expense included in:
|
|
|
|
Selling, general and
administrative
|
$
2,053
|
$
1,330
|
|
Research and
development
|
586
|
694
|
|
|
$
2,639
|
$
2,024
|
|
|
|
|
|
|
|
|
|
SUMMARY BALANCE
SHEET DATA
|
|
(in
thousands)
|
|
|
|
|
|
|
March
31,
|
December
31,
|
|
|
2021
|
2020
(1)
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
$
39,345
|
$
57,327
|
|
Total
assets
|
215,993
|
110,378
|
|
Stockholders'
equity
|
78,298
|
34,026
|
|
|
|
|
(1)
|
Derived from audited
financial statements
|
|
|
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SOURCE Rigel Pharmaceuticals, Inc.