Full Press Release Details
Rigel Pharmaceuticals Provides Business
SOUTH SAN FRANCISCO, Calif., January 10, 2022 - Rigel Pharmaceuticals,
Inc. (Nasdaq: RIGL) today provided a business update including preliminary 2021 full-year total revenue, the status of the Phase 3 study
in COVID-19, and upcoming catalysts in 2022.
"Despite the challenges the global pandemic continued to present
in 2021, our team responded with creative solutions to navigate the evolving landscape and now look forward to several catalysts that
have the potential to be transformative for the company in 2022," said Raul Rodriguez, Rigel's president and chief executive officer.
"We've made strategic investments designed to accelerate TAVALISSE ITP sales in the U.S., prepare for pivotal readouts in
two Phase 3 studies that have the potential to expand the market for fostamatinib into new indications such as wAIHA and COVID-19, and
advance our clinical-stage IRAK1/4 and RIPK1 programs into Phase 2 development."
Commercial and Preliminary Financial Update
In the fourth quarter of 2021, a total of 1,814 bottles of TAVALISSE (fostamatinib
disodium hexahydrate) were sold in the U.S., of which 1,785 were shipped directly to patients and clinics, representing the highest daily
bottles shipped to patients and clinics in a quarter since launch. For the full year ended December 31, 2021, 6,787 bottles of TAVALISSE
were shipped directly to patients and clinics, representing an increase of 8% compared to 2020. While Rigel is still in the process of
determining final results for the fourth quarter of 2021, Rigel expects to report net product sales of $17.6 million compared to $17.7
million for the same period of 2020.
Contract revenues for the quarter ended December 31, 2021, are expected
to be approximately $2.8 million, consisting of $1.8 million in revenue from collaborative partners and $1.0 million in government contract
revenue from the U.S. Department of Defense (DOD).
For the fourth quarter of 2021, Rigel expects to report total revenue
of approximately $20.4 million.
The company expects to report cash, cash equivalents, and short-term
investments as of December 31, 2021, of approximately $124.9 million compared to $57.3 million as of December 31, 2020.
The above information is preliminary, has not been audited, and is
subject to change upon the audit of the company's financial statements for the year ended December 31, 2021.
Phase 3 Trial in wAIHA
Rigel's FORWARD study, a Phase 3 pivotal trial of TAVALISSE in patients with wAIHA, is complete. Rigel expects to report topline data
from the 24-week study in mid-2022 and proceed with regulatory filings if the data is positive. If approved, TAVALISSE has the potential
to be the first-to-market therapy for patients with wAIHA in 2023.
Rigel's Phase 3 clinical trial evaluating fostamatinib in high-risk
patients hospitalized with COVID-19 has enrolled 231 of the targeted 308 patients to date. In December, Rigel expanded the inclusion
criteria to include patients with more severe disease (NIAID Ordinal Scale 6) to more accurately reflect the clinically predominant patient
population hospitalized with COVID-19 and help speed enrollment. In collaboration with the United States Food and Drug Administration
(FDA) and DOD, Rigel has also updated the primary endpoint for the study from progression to severe disease within 29 days, to the number
of days on oxygen through day 29.
"As a study endpoint, reducing the number of days a patient
spends on supplemental oxygen has proven to be clinically meaningful, is a good measure for recovery, and has been widely used in other
large NIH sponsored trials in the recent past," said Wolfgang Dummer, M.D., Ph.D., Rigel's chief medical officer. "The
findings of the NIH/NHLBI phase 2 study provided evidence of broad, consistent and clinically meaningful improvement in mortality, time
to sustained recovery, and the number of days on oxygen, for patients treated with fostamatinib in addition to standard of care. We look
forward to confirming these findings in a larger patient population in our Phase 3 study."
This endpoint allows for closer comparison of the results with earlier
results from the NIH/NHLBI Phase 2 trial with fostamatinib and various other NIH-sponsored trials, such as ACTIV-4, which uses a similar
outcome measure as a primary endpoint. Rigel expects to complete enrollment and report topline data in mid-2022.
In addition to Rigel's Phase
3 study, fostamatinib is also being evaluated as a treatment for COVID-19 in two ongoing studies, the
NIH/NHLBI-funded Phase 3 ACTIV-4 Host Tissue trial and a Phase 2 open-label clinical trial conducted by Imperial College London.
Rigel expects to advance R2899, a potent and selective
IRAK1/4 inhibitor, into an open-label Phase 1b/2 clinical study in patients with low-risk myeloid dysplastic syndrome (MDS) in Q1 2022.
R289 blocks inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1
receptor family (IL-1R) 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 patients1.
Partnered with Eli Lilly
R5529, a potent and selective RIPK1 inhibitor, will advance
into Phase 2 development in psoriasis in the 1H 2022 with partner Lilly. RIPK1 is implicated in
a broad range of key inflammatory cellular processes and plays a key role in TNF signaling, especially in the induction of pro-inflammatory
necroptosis. The program also includes RIPK1 compounds that cross the blood-brain barrier (CNS-penetrants) to address neurodegenerative
diseases such as Alzheimer's disease and ALS. Rigel is completing early discovery work on a potential candidate that Lilly may
advance into clinical development.
Progress on Global Expansion in ITP
In December 2021, partner Kissei reported positive topline results
for a Phase 3 study in ITP in Japan and is preparing a new drug application (NDA) for submission
to Japan's Pharmaceuticals and Medical Devices Agency (PMDA). In October 2018, Rigel entered into an exclusive license and supply
agreement with Kissei to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan and
the Republic of Korea. Fostamatinib has Orphan Drug Designation in Japan and Korea.
In patients with ITP, 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 receptor agonists) and splenectomy. However, not all patients are adequately
treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that destroy 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.2 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.3
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 (Fc R)
and c-type lectin receptor (CLR) mediated drivers of pathology such as pro-inflammatory cytokine release by monocytes and macrophages,
production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.4,5,6,7 Furthermore, SYK inhibition
in neutrophils and platelets may lead to decreased thrombo-inflammation, alleviating organ dysfunction in critically ill patients with
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
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.
Rigel Pharmaceuticals, Inc., is a biotechnology company dedicated to developing, and commercializing novel small molecule drugs that