SOUTH SAN FRANCISCO, Calif.,
March 2, 2021 /PRNewswire/ -- Rigel
Pharmaceuticals, Inc. (Nasdaq: RIGL) today reported financial
results for the fourth quarter and full year ended December 31, 2020, 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.
"Our team has shown its resilience as we continue to execute on
our mission to serve patients who have diseases where few or no
approved treatment options exist," said Raul Rodriguez, Rigel's president and CEO.
"Despite the challenges brought on by 2020, we successfully
expanded our global ITP reach and positioned ourselves for
potential success in wAIHA, announced a major collaboration with
Lilly to develop RIP1 inhibitors, and launched a
comprehensive COVID-19 clinical program which has gained the
support from the NIH, DOD, and several universities. Importantly,
we also continued to explore opportunities in immunology, and more
recently heme-onc, with our IRAK 1/4 inhibitor
program."
Business Update
In February
2021, Rigel and Eli Lilly (Lilly) announced a global
strategic collaboration to co-develop and commercialize R552,
Rigel's receptor-interacting serine/threonine-protein kinase 1
(RIP1) inhibitor, for all indications, including autoimmune and
inflammatory diseases. In addition, Lilly will lead the development
and commercialization of all RIP1 inhibitors in central nervous
system (CNS) indications. Under the terms of the agreement,
Lilly will pay an upfront cash payment to Rigel of $125 million and is eligible to receive up to
$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 launched a Phase 3 clinical trial to evaluate fostamatinib
for the treatment of hospitalized COVID-19 patients. The Phase 3
trial is designed to evaluate the safety and efficacy of
fostamatinib in hospitalized COVID-19 patients without respiratory
failure that have certain high-risk prognostic factors. This
multi-center, double-blind, placebo-controlled, adaptive design
study is expected to enroll over 300 evaluable patients that will
be randomly assigned 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.
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 Rigel's Phase 3 clinical trial of
fostamatinib in hospitalized COVID-19 patients.
The Phase 2 clinical trial of fostamatinib in hospitalized
COVID-19 patients sponsored by the National Heart, Lung, and Blood
Institute (NHLBI), part of the National Institutes of Health (NIH),
in collaboration with Inova Health System has currently enrolled
57 patients. This is a
double-blind, placebo-controlled Phase 2 clinical trial that is
randomly assigning fostamatinib plus standard of care (SOC) or
matched placebo plus SOC (1:1) to approximately 60 evaluable
patients who are a 5 to 7 on the 8-point ordinal scale (requiring
supplemental oxygen via nasal canula or non-invasive ventilation,
requiring mechanical ventilation or extracorporeal membrane
oxygenation). The primary endpoint of this study is cumulative
incidence of serious adverse events (SAE) through day 29. The
NHLBI and Rigel expect to report topline data from this clinical
trial in April 2021.
Imperial College London's ongoing Phase 2 clinical trial of
fostamatinib in hospitalized COVID-19 patients is a two-stage open
label, controlled trial with patients randomized (1:1:1) to
fostamatinib, ruxolitinib, or standard of care. The study has
currently enrolled 106 patients. Treatment will be administered
twice daily for 14 days and patients will receive a follow-up
assessment at day 14 and day 28 after the first dose. The primary
objective will be to determine the efficacy of fostamatinib and the
efficacy of ruxolitinib compared to standard of care to reduce the
proportion of hospitalized patients progressing from mild or
moderate to severe COVID-19 pneumonia.
Enrollment is progressing in Rigel's FORWARD study, a Phase 3
pivotal trial of TAVALISSE in warm autoimmune hemolytic anemia
(wAIHA). The study has enrolled 66 of 90 patients targeted for
enrollment. Rigel has reached agreement with the U.S. Food and Drug
Administration (FDA) on the durable response measure for the
primary efficacy endpoint of the study as well as the inclusion of
additional secondary endpoints. The company recently announced
that the FDA had granted Fast Track designation to TAVALISSE for
the treatment of wAIHA. The FDA previously granted TAVALISSE Orphan
Drug designation for this indication. If approved, TAVALISSE may be
the first-to-market therapy for patients with wAIHA.
Rigel continues to advance the development of its IRAK1/4
program, which includes R835, an orally available, potent and
selective inhibitor that inhibits both IRAK1 and IRAK4. The company
is currently identifying therapeutic opportunities in the areas of
hematology/oncology and rare immunology diseases.
In November 2020, Rigel and its
partner Medison Pharma announced that Health
Canada approved the new drug submission (NDS)
for TAVALISSE for the treatment of thrombocytopenia in
adult patients with chronic ITP who have had an insufficient
response to other treatments. Medison is also anticipating a
decision on a New Drug Application (NDA) in Israel in Q2 2021. In July 2020, Rigel and its partner Grifols S.A.
announced the launch of TAVLESSE in Germany and the United Kingdom following approval by the
European Commission in January 2020.
Currently, TAVALISSE is in a Phase 3b
clinical trial in Japan with
Rigel's partner Kissei. This trial is required for approval by the
Pharmaceuticals and Medical Devices Agency (PMDA) in Japan.
Financial Update
For the fourth quarter of 2020, Rigel
reported a net loss of $19.2 million,
or $0.11 per share, compared to net
loss of $17.2 million, or
$0.10 per share, in the same period
of 2019.
In the fourth quarter of 2020, total revenues were $18.5 million, consisting of $17.8 million in TAVALISSE net product sales and
$697,000 in contract revenues from
collaborations. TAVALISSE net
product sales of $17.8 million
increased by 28% from $13.8 million
in the fourth quarter of 2019. Contract revenues from
collaborations of $697,000 for the
fourth quarter of 2020 consisted of $500,000 from Grifols related to an option for
commercialization in additional territories and $197,000 in revenues earned from the performance
of certain research and development services from Rigel's
collaboration agreement with Grifols.
Rigel reported total costs and expenses of $37.3 million in the fourth quarter of 2020,
compared to $32.7 million for the
same period in 2019. The increase in costs and expenses was due to
the increase in research and development costs primarily related to
the continued work on Rigel's Phase 1 clinical trial in IRAK 1/4
inhibitor program, as well as its recently launched Phase 3 clinical trial for
hospitalized COVID-19 patients, partially offset by the decrease in
costs on Rigel's ongoing Phase 3 clinical trial in wAIHA.
For the full year ended December 31,
2020, Rigel reported a net loss of $29.7 million, or $0.18 per share, compared to a net loss of
$66.9 million, or $0.40 per share, for the same period of 2019.
Rigel reported total revenues of $108.6
million for the year ended December
31, 2020, compared to $59.3
million in the same period of 2019. Total revenues for
the year ended December 31, 2020
consisted of $61.7 million in
TAVALISSE net product sales and $46.9
million in contract revenues from collaborations. TAVALISSE
net product sales of $61.7 million
increased by 41% from $43.8 million
in 2019. The increase in contract revenues from collaborations
related to revenue from the upfront fee previously received in
2019, as well as the milestone payment received from Grifols in the
first quarter of 2020 upon EC approval of the MAA for fostamatinib
in Europe, and the
$2.1 million in contract revenues for
the achievement of a milestone in accordance with the amended
license and collaboration agreement with Daiichi-Sankyo, partially
offset by the developmental and commercial milestones
from its various collaborative
partners in 2019.
Total costs and expenses for the year ended December 31, 2020, were $137.6 million, compared to $128.4 million, for the same period of 2019. The
increase in total costs and expenses was primarily related to the
increases in research and development costs due to the completion
of Rigel's Phase 1 clinical trial in RIP1 inhibitor program,
ongoing work on recently launched Phase 3 clinical trial for
hospitalized COVID-19 patients, continued work on Rigel's Phase 1
clinical trial in IRAK 1/4 inhibitor program and ongoing Phase 3
clinical trials in wAIHA, as well
as increases in personnel-related costs, partially offset by
decreases in various third-party costs due to the COVID-19
pandemic.
As of December 31, 2020, Rigel had
cash, cash equivalents and short-term investments of $57.3 million, compared to $98.1 million as of December 31, 2019.
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.
About R5526
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 R8356
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 trial
for the treatment of warm autoimmune hemolytic anemia
(wAIHA)6; an NIH/NHLBI-sponsored Phase 2 trial for the
treatment of hospitalized COVID-196 patients, in
collaboration with Inova Health System; and a Phase 2 trial for the
treatment of COVID-19 being conducted by Imperial College
London. Additionally, Rigel has launched a Phase 3
clinical trial of fostamatinib for the treatment of hospitalized
COVID-19 patients.
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.
- Berlin DA, Gulick RM, Martinez FJ. Severe Covid-19. N Engl J
Med 2020
- 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
- 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
- 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
- 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
- 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 ability to
achieve development, regulatory and commercial milestone
payments, as well as tiered royalties; Rigel's expected operating
results for the year ending and as of December 31, 2020, including net sales and cash,
cash equivalents and short-term investments; expectations related
to the market opportunity for fostamatinib as a COVID-19
therapeutic; Rigel's ability to further develop its clinical stage
product candidates; 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 Quarterly Report on Form 10-Q
for the period ended September 30,
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.
Rigel Investor Contact:
Phone: 650.624.1232
Email: ir@rigel.com
RIGEL
PHARMACEUTICALS, INC.
|
STATEMENTS OF
OPERATIONS
|
(in thousands,
except per share amounts)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
December 31,
|
|
Year Ended
December 31,
|
|
|
2020
|
2019
|
|
2020
|
2019
|
|
|
(unaudited)
|
|
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
Product sales,
net
|
$
17,753
|
$
13,829
|
|
61,696
|
$
43,772
|
|
Contract revenues
from collaborations
|
697
|
1,571
|
|
46,925
|
15,516
|
|
Total
revenues
|
18,450
|
15,400
|
|
108,621
|
59,288
|
|
|
|
|
|
|
|
Costs and
expenses:
|
|
|
|
|
|
|
Cost of product
sales
|
321
|
178
|
|
895
|
906
|
|
Research and
development (see Note A)
|
15,138
|
14,247
|
|
60,101
|
52,885
|
|
Selling, general and
administrative (see Note A)
|
21,818
|
18,312
|
|
76,598
|
74,588
|
|
Total costs and
expenses
|
37,277
|
32,737
|
|
137,594
|
128,379
|
|
|
|
|
|
|
|
Loss from
operations
|
(18,827)
|
(17,337)
|
|
(28,973)
|
(69,091)
|
Interest
income
|
19
|
464
|
|
582
|
2,532
|
Interest
expense
|
(429)
|
(327)
|
|
(1,353)
|
(335)
|
|
|
|
|
|
|
|
Net loss
|
$
(19,237)
|
$
(17,200)
|
|
$
(29,744)
|
$
(66,894)
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.11)
|
$
(0.10)
|
|
$
(0.18)
|
$
(0.40)
|
|
|
|
|
|
|
|
Weighted average
shares used in computing net loss per share, basic and
diluted
|
169,039
|
167,619
|
|
168,754
|
167,400
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Note
A
|
|
|
|
|
|
|
|
|
|
|
|
|
Stock-based
compensation expense included in:
|
|
|
|
|
|
|
Selling, general and
administrative
|
$
1,242
|
$
934
|
|
$
5,223
|
$
6,453
|
|
Research and
development
|
388
|
477
|
|
2,072
|
2,662
|
|
|
$
1,630
|
$
1,411
|
|
$
7,295
|
$
9,115
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
SUMMARY BALANCE
SHEET DATA
|
|
|
|
|
(in
thousands)
|
|
|
|
|
|
|
|
|
|
|
|
|
December
31,
|
|
|
|
|
|
2020
|
2019
|
|
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
$
57,327
|
$
98,078
|
|
|
|
|
Total
assets
|
110,378
|
147,569
|
|
|
|
|
Stockholders'
equity
|
34,026
|
53,815
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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SOURCE Rigel Pharmaceuticals, Inc.