Mirati Therapeutics Reports Fourth Quarter Financial Results

On February 28, 2019 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the fourth quarter and full-year ended December 31, 2018 (Press release, Mirati, FEB 28, 2019, View Source [SID1234533854]).

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"Mirati made great progress in 2018 with significant advances in our sitravatinib and KRAS (MRTX849) programs. We remain focused on both programs as we anticipate enrolling second-line NSCLC patients in our Phase 3 randomized trial with sitravatinib in combination with nivolumab in the first half of 2019. We look forward to seeing data from our collaboration with BeiGene, with initial proof of concept data from the clinical trials studying sitravatinib in combination with tislelizumab, in certain tumor types, expected in the second half of 2019. We’re also excited to see MRTX849 advance in the Phase 1/2 trial toward initial clinical efficacy data this year," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer. "We are well funded to execute our plans following a successful financing in January and look forward to a pivotal year for Mirati."

Financial Results for the Fourth Quarter and Full Year 2018

Cash, cash equivalents, and short-term investments were $222.8 million at December 31, 2018, compared to $150.8 million at December 31, 2017. In January 2019, we completed a public equity offering with net proceeds of $107.9 million.

License and collaboration revenues for the fourth quarter of 2018 were $3.5 million and for the year ended December 31, 2018 were $12.9 million. There were no license and collaboration revenues during the same periods of 2017. License and collaboration revenues relate to the Collaboration and License Agreement between the Company and BeiGene, Ltd., which became effective January 7, 2018.

Research and development expenses for the fourth quarter of 2018 were $26.8 million, compared to $15.2 million for the same period in 2017. Research and development expenses for the year ended December 31, 2018 were $93.9 million compared to $58.1 million for the same period in 2017. The increase in research and development expenses for both periods is due to an increase in expense associated with sitravatinib and our KRAS inhibitor program, as well as an increase in salaries and related expense, including an increase in share-based compensation expense. The increase in sitravatinib expense is due to increased costs to support the expansion of existing and new clinical trials and the increase in KRAS inhibitor program expense relates primarily to increased manufacturing expenses for our lead clinical compound, MRTX849. The Company recognized research and development-related share-based compensation expense of $2.1 million during the fourth quarter of 2018 as compared to $0.7 million for same period in 2017 and $7.2 million during the year ended December 31, 2018 as compared to $3.2 million for the same period in 2017.

General and administrative expenses for the fourth quarter of 2018 were $6.4 million, compared to $3.0 million for the same period in 2017. General and administrative expenses for the year ended December 31, 2018 were $21.7 million compared to $13.5 million for the same period of 2017. The increase is due primarily to an increase in share-based compensation expense, and to a lesser extent increases in salaries and related expense, professional and consulting fees and patent filing fees. The Company recognized general and administrative-related share-based compensation expense of $2.1 million during the fourth quarter of 2018 as compared to $0.6 million for same period in 2017 and of $8.6 million during the year ended December 31, 2018 as compared to $3.6 million for the same period in 2017.

Net loss for the fourth quarter of 2018 was $28.3 million, or $0.87 per share basic and diluted, compared to net loss of $17.9 million, or $0.67 per share basic and diluted for the same period in 2017. Net loss for the year ended December 31, 2018 was $98.4 million, or $3.19 per share, compared to $70.4 million, or $2.78 per share, for the same period of 2017.

About Sitravatinib

Sitravatinib is a spectrum-selective kinase inhibitor that potently inhibits receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET. As an immuno-oncology agent, sitravatinib is being evaluated in combination with nivolumab (OPDIVO), an anti-PD-1 checkpoint inhibitor, in patients whose cancers have progressed despite treatment with a checkpoint inhibitor. Sitravatinib’s potent inhibition of TAM and split family RTKs may overcome resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment, enhancing antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation.

Sitravatinib is also being evaluated as a single agent in a Phase 1b expansion clinical trial emphasizing enrollment of patients whose tumors harbor specific mutations in the CBL protein. When CBL is inactivated by mutation, multiple RTKs, including TAM, VEGFR2 and KIT, are dysregulated and may act as oncogenic tumor drivers in NSCLC and melanoma. Sitravatinib potently inhibits these RTKs and is being investigated as a treatment option for cancer patients with CBL mutations.

About MRTX849

MRTX849 is an investigational, orally-available small molecule, that is designed to potently and selectively inhibit a form of KRAS which harbors a substitution mutation (G12C). KRAS G12C mutations are present in approximately 14% of NSCLC adenocarcinoma patients, 4% of colorectal cancer patients, and subsets of other types of cancer. Tumors characterized by KRAS G12C mutations are commonly associated with poor prognosis and resistance to therapy, and patients with these mutations have few treatment options. MRTX849 is being evaluated in a Phase 1/2 trial treating patients with molecularly-identified KRAS G12C-positive advanced solid tumors.

Fate Therapeutics to Webcast Conference Call Reporting Fourth Quarter and Full Year 2018 Financial Results

On February 28, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that the Company will host a conference call and live audio webcast on Tuesday, March 5, 2019 at 5:00 p.m. ET to report its fourth quarter and full year 2018 financial results and provide a corporate update (Press release, Fate Therapeutics, FEB 28, 2019, https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-webcast-conference-call-reporting-fourth-4 [SID1234533844]).

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In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 3374407. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

Nektar Therapeutics Reports Fourth Quarter and Year-End 2018 Financial Results

On February 28, 2019 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Nektar Therapeutics, FEB 28, 2019, View Source [SID1234533843]).

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Cash and investments in marketable securities at December 31, 2018 were $1.9 billion as compared to $353.2 million at December 31, 2017.

"2018 was a remarkable year for Nektar accentuated by new collaborations with leading pharmaceutical companies that validate the depth and innovation of our pipeline," said Howard W. Robin, President and Chief Executive Officer of Nektar. "We entered 2019 in an exceptionally strong financial position. We are working closely with the FDA during the ongoing review of our NDA for NKTR-181 while simultaneously preparing for a potential commercial launch later this year. We continue to design and execute on our broad registrational program for NKTR-214 (bempegaldesleukin1) with our partner Bristol-Myers Squibb. NKTR-358 is advancing in the ongoing clinical study in lupus patients and two new studies in additional auto-immune conditions are planned to start in 2019.NKTR-262, our TLR agonist is being evaluated in a Phase 1/2 study and finally, NKTR-255, our next I-O candidate is slated to move into the clinic first in patients with multiple myeloma and then in combination with CAR-T therapy."

Summary of Financial Results

Revenue for the fourth quarter of 2018 was $39.8 million as compared to $95.5 million in the fourth quarter of 2017. Revenue in the fourth quarter of 2017 included a total of $60.0 million of non-recurring revenue related to a new sublicense agreement, a contract settlement agreement and the recognition of deferred revenue from several collaboration agreements. Revenue for the year ended December 31, 2018 was $1.2 billion as compared to $307.7 million in 2017 and included the recognition of $1.06 billion of license revenue from the Bristol-Myers Squibb collaboration agreement.

Total operating costs and expenses in the fourth quarter of 2018 were $140.1 million as compared to $119.5 million in the fourth quarter of 2017. Total operating costs and expenses for 2018 were $505.4 million as compared to $367.4 million in 2017. Total operating costs and expenses increased primarily as a result of increased research and development (R&D) expense.

R&D expense in the fourth quarter of 2018 was $108.9 million as compared to $81.4 million for the fourth quarter of 2017. R&D expense for the year ended December 31, 2018 was $399.5 million as compared to $268.5 million in 2017. R&D expense was higher in the fourth quarter and full year 2018 as compared to the same periods in 2017 primarily because of expenses for our pipeline programs, including the continued development of bempegaldesleukin in Phase 2 and registrational studies and related manufacturing costs, costs related to the NKTR-181 New Drug Application and NKTR-181 pre-commercial manufacturing, Phase 1 clinical studies of NKTR-358, the Phase 1 study of NKTR-262 in combination with bempegaldesleukin and IND-enabling activities for NKTR-255.

General and administrative (G&A) expense was $23.8 million in the fourth quarter of 2018 as compared to $12.3 million in the fourth quarter of 2017. G&A expense for 2018 was $81.4 million as compared to $52.4 million in 2017. G&A expense was higher in the fourth quarter and full year 2018 as compared to the same periods in 2017 primarily due to an increase in non-cash stock-based compensation expense.

Net loss for the fourth quarter of 2018 was $98.2 million or $0.57 basic and diluted loss per share as compared to a net loss of $33.8 million or $0.21 basic and diluted loss per share in the fourth quarter of 2017. Net income for the year ended December 31, 2018 was $681.3 million or $3.78 diluted earnings per share as compared to a net loss of $96.7 million or $0.62 basic and diluted loss per share in 2017.

2018 and Year-to-Date Business Highlights

● In February 2019, Nektar presented clinical data from first-line Stage IV urothelial carcinoma patients enrolled in the PIVOT-02 study of bempegaldesleukin with nivolumab at the 2019 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium.
● In December 2018, Nektar and Gilead entered into a research collaboration to explore the potential of NKTR-255, an IL-15 agonist, in virology. The collaboration will evaluate NKTR-255 in combination with antiretroviral therapies in Gilead’s portfolio.
● In December 2018, Nektar and Bristol-Myers Squibb initiated PIVOT-10, a potential registrational study evaluating bempegaldesleukin with nivolumab in cisplatin-ineligible urothelial carcinoma patients whose tumors express PD-L1 (Combined Positive Score < 10).
● In December 2018, Nektar and Bristol-Myers Squibb initiated PIVOT-09, a Phase 3 study of bempegaldesleukin with nivolumab versus tyrosine kinase inhibitor (TKI) monotherapy in patients with advanced metastatic renal cell carcinoma.
● In November 2018, Nektar presented data for first-line Stage IV melanoma patients from the ongoing PIVOT-02 study at the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting.
● In November 2018, Nektar entered into a new clinical collaboration with Pfizer Inc. to evaluate several combination regimens in multiple cancer settings including metastatic castration-resistant prostate cancer (mCRPC) and squamous cell carcinoma of the head and neck (SCCHN). Under the collaboration, Pfizer will initiate a Phase 1b/2 clinical trial to evaluate the anti-cancer activity of the combined agents, avelumab, talazoparib and bempegaldesleukin and separately avelumab, enzalutamide and bempegaldesleukin.
● In September 2018, Nektar and Bristol-Myers Squibb initiated the Phase 3 study of bempegaldesleukin with nivolumab as compared to nivolumab monotherapy in participants with previously untreated unresectable or metastatic melanoma.
● In July 2018, the U.S. Food and Drug Administration filed and accepted a New Drug Application (NDA) for NKTR-181, a first-in-class opioid analgesic, to treat chronic low back pain in adult patients new to opioid therapy. In February 2019, Nektar received notification from the FDA that the review period for NKTR-181 has been extended by three months. The new Prescription Drug User Fee Act (PDUFA) date is now August 29, 2019. The FDA extended the action date to allow time to review data from two additional preclinical studies that Nektar conducted which were requested by the FDA early on in our review process. The new preclinical data are supportive of the company’s abuse liability package included in the NDA filing for NKTR-181.
● In June 2018, Nektar presented data from the ongoing PIVOT study for bempegaldesleukin with nivolumab at the 2018 ASCO (Free ASCO Whitepaper) Meeting. Pre-specified efficacy criteria were achieved in three tumor types: first-line melanoma, first-line renal cell carcinoma and first-line urothelial cancer.
● In May 2018, Nektar began dosing patients with systemic lupus erythematosus in a new Phase 1b multiple ascending dose study of NKTR-358, an IL-2 regulatory T cell stimulator, designed to correct the underlying immune system dysfunction found in patients with immune disorders.
● In April 2018, Nektar announced a new clinical collaboration agreement with Takeda to evaluate bempegaldesleukin with TAK-659, a dual SYK and FLT-3 inhibitor in liquid and solid tumors. The first of these studies, a Phase 1b study in patients with Non-Hodgkin Lymphoma, was initiated in January of 2019.
● In April 2018, Nektar presented preclinical data for its immuno-oncology pipeline at the 2018 AACR (Free AACR Whitepaper) Annual Meeting. Preclinical data presented by Nektar researchers and collaborators demonstrated that bempegaldesleukin combines with multiple modalities including TLR agonism and adoptive cell therapy.
● In April 2018, Nektar began dosing patients in the REVEAL Phase 1/2 study, which will evaluate NKTR-262, Nektar’s wholly-owned novel toll-like receptor 7/8 agonist, in combination with bempegaldesleukin. The dose-escalation phase of the study is continuing.
● In February 2018, Nektar and Bristol-Myers Squibb entered into a global development and commercialization agreement to evaluate the full potential of bempegaldesleukin with nivolumab in more than 20 indications in 9 tumor types.

The company also announced upcoming presentations and speaking engagements at the following scientific congresses during the first half of 2019:

ASCO-SITC Clinical Immuno-Oncology Symposium, San Francisco, CA

● Oral presentation (Abstract #28): "Phase Ib: Preliminary clinical activity and immune activation for NKTR-262 (TLR 7/8 agonist) plus NKTR-214 (CD122-biased agonist) in patients (pts) with locally advanced or metastatic solid tumors (REVEAL Phase Ib/II Trial)"
o Presenter: Dr. Adi Diab, MD Anderson Cancer Center
o Session: Oral Abstract Session B
o Session Date & Time: March 1, 2019, 1:00 p.m.-2:15 p.m. Pacific Time
26th International Molecular Medicine Tri-Conference, San Francisco, CA

● Presentation Title: "Advanced Cytokine Engineering for Immunotherapy"
o Presenter: Steven Doberstein, Ph.D., Nektar Therapeutics
o Session: Emerging Immuno-Oncology Targets Session
o Session Date & Time: March 12, 2019, at 11:25 a.m. Pacific Time
Keystone Symposia Cancer Immunotherapy: Mechanistic Insights to Improve Clinical Benefit, Whistler, BC, Canada

● Presentation Title: "Intratumoral expansion of CD8+ T cells and depletion of Tregs after treatment with NKTR-214, a first-in-class, CD122-preferential IL-2 pathway agonist"
o Presenter: Willem Overwijk, Ph.D., Nektar Therapeutics
o Session: Immune Checkpoints: Basic Mechanisms and Novel Targets
o Session Date & Time: March 13, 2019, 8:00 a.m.-11:00 a.m. Pacific Time
CHI’s Fourth Annual Immuno-Oncology Summit Europe, London, UK

● Presentation Title: "Exploratory Studies up to IND with NKTR-255, a Memory T-Cell Stimulating Cytokine"
o Presenter: Saul Kivimae, Ph.D., Nektar Therapeutics
o Session: Importance of Cytokines/Innovative Approaches with Clinical Benefit
o Session Date and Time: March 19, 2019, at 16:45 Greenwich Mean Time
ICI-IO Combinations Summit, Boston, MA

● Presentation Title: "Supercharging the Tumor Microenvironment: Lessons from NKTR-214 and OPDIVO"
o Presenter: Willem Overwijk, Ph.D., Nektar Therapeutics
o Session Date and Time: March 20, 2019, at 11:30 a.m. Eastern Time
American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019, Atlanta, GA

● Abstract 2256/Poster Board 15: "Combination of neoantigen DNA plasmid vaccine VB10.NEO and NKTR-214, a CD122-biased immunostimulatory cytokine, induces strong neoantigen-specific T cell responses and sustained tumor regression in pre-clinical models," Granum, S., et al.
o Session: Clinical Research – Combination Immunotherapies 1
o Session Date and Time: April 1, 2019, 1:00 p.m.-5:00 p.m. Eastern Time
o Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 19

● Abstract 3210/Poster Board 20: "A potential immunotherapeutic approach for the treatment of osteosarcoma," Wahba, A., et al.
o Session: Immunology – Combination Immunotherapies 2
o Session Date & Time: Tuesday, April 2, 2019, 8:00 a.m.-12:00 p.m. Eastern Time
o Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 2

● Abstract 3265/Poster Board 15: "NKTR-255, a polymer-conjugated IL-15 enhances anti-tumor NK cell responses and synergizes with monoclonal antibodies to provide long-term survival in human lymphoma model," Miyazaki, T., et al.
o Session: Immunology – Novel Immunomodulatory Agents
o Session Date and Time: April 2, 2019, 8:00 a.m.-12:00 p.m. Eastern Time
o Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 25
15th Annual PEGS Boston Summit, Boston, MA

● Keynote: "Harnessing Potent Cytokine Agonist Pathways by Polymer Engineering to Develop Novel Immune Therapeutic Agents"
o Presenter: Loui Madakamutil, Ph.D., Nektar Therapeutics
o Session: Latest Developments in Agonist Immunotherapy – Cytokines and OX40 Targets
o Presentation Date and Time: Thursday, April 11, 2019 1:50 p.m. Eastern Time
6th Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference (ITOC6), Vienna, Austria

● Poster P2.12: "Mechanism of Action of NKTR-214, a first-in-class, CD122-preferential IL-2 pathway agonist"
o Presenter: Willem Overwijk, Ph.D., Nektar Therapeutics
o Session: Emerging Concepts/Novel Agents
o Session Dates and Times: April 11, 2019, at 18.20 Central European Time and April 12, 2019, from 14.00-14.30 Central European Time

Conference Call to Discuss Fourth Quarter and Year-End 2018 Financial Results
Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time today, Thursday, February 28, 2019. This press release and a live audio-only webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Thursday, March 28, 2019.

To access the conference call, follow these instructions:

Dial: (877) 881-2183 (U.S.); (970) 315-0453 (international)
Passcode: 4988768 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call.

Oncolytics Biotech® Presents Biomarker Data in Second-line Pancreatic Cancer at AACR

On February 28, 2019 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the publication of an abstract demonstrating a biomarker for predicting clinical response in patients treated with pelareorep (Press release, Oncolytics Biotech, FEB 28, 2019, View Source [SID1234533842]). This analysis was conducted in patient samples from REO 024; a study of pelareorep and Keytruda in combination with chemotherapy in patients with second-line pancreatic cancer. Detailed results will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place March 30 through April 3 in Atlanta, Georgia.

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"With a simple blood draw, this biomarker data allows physicians to understand which patients are likely to respond to treatment, allowing for the design of clinical studies that are cheaper, faster and more likely to succeed," said Dr. Matt Coffey, President and CEO of Oncolytics Biotech. "Our biomarker data that we will present at AACR (Free AACR Whitepaper) is immediately applicable to future clinical studies. Investigators should now be able to predict which patients will respond to pelareorep in combination with a checkpoint inhibitor. This biomarker will be evaluated in our clinical studies with checkpoint inhibitors, including both of our multiple myeloma studies, the AWARE-1 breast cancer study, and importantly, in the same setting this data was produced, our phase two second-line pancreatic study in combination with Keytruda."

"Using patient blood samples from our REO 024 study in second line pancreatic cancer, T cell receptor sequencing was performed with Adaptive Biotechnologies’ immunoSEQ Assay to measure the diversity or clonality of the T cell population," said Dr. Rita Laeufle, CMO of Oncolytics Biotech. "Results from this analysis demonstrate that higher clonality after one three-week cycle of treatment can identify patients likely to respond to combination treatment of pelareorep and a checkpoint inhibitor."

The results suggest that those patients with a statistically significant change in their T cell population demonstrate a clinical benefit from pelareorep treatment. High T cell clonality correlates with progression free survival at baseline (HR=0.05, p=0.01). Moreover, high clonality correlates with overall survival at both baseline (HR=0.124, p=0.01) and after one cycle of treatment (HR=0.08, p=0.01). This research highlights the potential utility of measuring T cell clonality as a predictive and prognostic biomarker of pelareorep therapy.

The abstract, authored by Dr. Grey Wilkinson, a translational scientist at Oncolytics Biotech, and his colleagues, in collaboration with Northwestern University, UT Health San Antonio and Adaptive Biotechnologies can be found online at View Source!/6812/presentation/4866. Full details from the poster presentation will be announced after it is presented.

Poster Board Number: 1
Presentation Number: 2272
Title:
Exploratory analysis of T cell repertoire dynamics upon systemic treatment with the oncolytic virus pelareorep in combination with pembrolizumab and chemotherapy in patients with advanced pancreatic adenocarcinoma
Date:
Monday, April 1
Lecture Time: 1:00 p.m. ET – 5:00 p.m. ET
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 20
Speakers: Grey Wilkinson
Session Category:
Clinical Research

Session:
Current Developments in Non-invasive Biomarkers for Assessment of Cancer 3

About Pelareorep
Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

Rigel Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Company Update

On February 28, 2019 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL), reported financial results for the fourth quarter and full year ended December 31, 2018 and provided a business update (Press release, Rigel, FEB 28, 2019, View Source [SID1234533841]).

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Recent Highlights

· Fourth quarter 2018 total revenues were $37.9 million; including $30.6 million in collaboration revenues and $7.3 million in net product sales of TAVALISSE (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment

·Full year 2018 total revenues were $44.5 million, including $13.9 million in net product sales of TAVALISSE

· As of December 31, 2018, cash, cash equivalents, and short-term investments were $128.5 million

· On January 23, 2019, Rigel entered into an exclusive license and supply agreement with Grifols, S.A. (Grifols) to commercialize fostamatinib in Europe and Turkey

"Since the successful U.S. launch of TAVALISSE in May of 2018, our momentum has continued to build with a growing number of physicians utilizing our product as an early treatment option," stated Raul Rodriguez, president and CEO. "Our recent commercial collaborations with European and Japanese partners, Grifols and Kissei, lay the groundwork for us to advance fostamatinib globally and to access the worldwide ITP market which is estimated to be $1.8 billion annually. These recent collaborations, along with growing TAVALISSE sales, have also provided additional funds to strengthen our balance sheet. While we remain focused on building our commercial business, we will continue the expansion of our pipeline with the upcoming initiation of our Phase 3 trial in warm autoimmune hemolytic anemia."

Financial Update

For the fourth quarter of 2018, Rigel reported net income of $3.2 million, or $0.02 basic and diluted net income per share, compared to a net loss of $25.9 million, or $0.18 basic and diluted net loss per share, in the same period of 2017.

For the fourth quarter of 2018, Rigel reported total revenues of $37.9 million, consisting of $30.6 million in collaboration revenues and $7.3 million in net product sales of TAVALISSE. There were no net product sales nor contract revenues from collaborations in the fourth quarter of 2017.

Contract revenue from collaborations of $30.6 million in the fourth quarter of 2018 were related to the portion of the $33.0 million upfront fee recognized as revenue upon delivery of license rights to Kissei Pharmaceutical Co., Ltd. (Kissei) for the development and commercialization of fostamatinib in all current and potential indications in Japan, China, Taiwan and the Republic of Korea.

Rigel reported total costs and expenses of $35.3 million in the fourth quarter of 2018, compared to $26.2 million for the same period in 2017. The increase in costs and expenses was primarily due to the increase in personnel costs as Rigel expanded its customer-facing and medical affairs teams, third party costs to support Rigel’s ongoing commercial efforts for TAVALISSE, as well as research and development cost for its warm autoimmune hemolytic anemia (AIHA) and other preclinical studies.

For the year ended December 31, 2018, Rigel reported a net loss of $70.5 million, or $0.44 per share, compared to a net loss of $78.0 million, or $0.62 per share, for the same period of 2017.

Rigel reported total revenues of $44.5 million for the year ended December 31, 2018, compared to $4.5 million in 2017. Total revenues in 2018 consisted of $30.6 million in collaboration revenue related to Rigel’s collaboration agreement with Kissei and $13.9 million in net product sales of TAVALISSE. Contract revenues from collaborations in 2017 consisted of a $3.3 million payment Rigel received from BerGenBio ASA as a result of advancing BGB324, an investigational and orally available small molecule AXL kinase inhibitor, to a Phase 2 clinical study, and a $1.2 million payment Rigel earned pursuant to its license agreement with a third party. There were no product sales for the year ended December 31, 2017.

Total costs and expenses for the year ended December 31, 2018 were $117.2 million, versus $84.1 million for the full year 2017, primarily related to an increase in personnel and third-party costs associated with the launch of TAVALISSE.

As of December 31, 2018, Rigel had cash, cash equivalents and short-term investments of $128.5 million, compared to $115.8 million as of December 31, 2017.

Business Update

Revenue from sales of TAVALISSE grew approximately 50% in the fourth quarter of 2018 compared to the third quarter of 2018, driven in part by continued use of the product as an early treatment option in steroid refractory patients and strong continuation of therapy among patients.

In January 2019, Rigel announced that it had entered into an exclusive license and supply agreement with Grifols to commercialize fostamatinib disodium hexahydrate in all potential indications in Europe and Turkey.

With this agreement, Rigel is positioned to access the three largest markets for ITP (U.S., EU, and Japan), an indication with a global market that is estimated to be over $1.8 billion annually. This collaboration partners fostamatinib with one of the largest intravenous immunoglobulin (IVIG) manufacturers globally that has established relationships with European hematologists and hematologist/oncologists, as well as a distribution infrastructure across the EU. Fostamatinib is on track for potential EMA approval by the end of 2019, which could enable a product launch in initial European markets as early as 2020.

Under terms of the commercial license agreement, Rigel received a $30.0 million upfront cash payment, with the potential for $297.5 million in payments related to regulatory and commercial milestones, which includes a $20.0 million payment upon EMA approval of fostamatinib for the treatment of chronic ITP. Rigel will also receive stepped double-digit royalty payments based on tiered net sales which may reach 30% of net sales of fostamatinib. In return, Grifols receives exclusive rights to fostamatinib in human diseases in Europe and Turkey, including chronic ITP, autoimmune hemolytic anemia (AIHA), and IgA nephropathy (IgAN). In the event that, in 2021, after the second anniversary of the agreement, fostamatinib has not been approved by the EMA for the treatment of ITP in Europe, Grifols will have the option during a six-month time-frame to terminate the entire agreement which would terminate all their rights to ITP, AIHA, and all other indications. In this limited circumstance, Rigel will pay Grifols $25.0 million and regain all rights to fostamatinib in the Grifols territories. Rigel retains the global rights to fostamatinib outside the Grifols and Kissei territories.

In addition to growing its commercial business, Rigel has been working to broaden its pipeline by expanding the potential use of fostamatinib in other indications and developing new molecules. Rigel is on track to initiate a pivotal Phase 3 trial of fostamatinib in warm AIHA in the first half of 2019. Additionally, the Phase 1 trial of R8351, Rigel’s investigational IRAK 1/4 inhibitor, is expected to report initial data in the second half of 2019.

About ITP

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 (TPOs) 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 with ITP.

About AIHA

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 40,000 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.

About R8351

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 (IL-1R) family receptor 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 conditions. R835 is active 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.

Conference Call and Webcast with Slides Today at 5:00PM Eastern Time

Rigel will hold a live conference call and webcast today at 5:00pm Eastern Time (2:00pm Pacific Time).

Participants can access the live conference call by dialing 855-892-1489 (domestic) or 720-634-2939 (international) and using the Conference ID number 2257425. The webcast, with slide presentation, can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

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 is a registered trademark of Rigel Pharmaceuticals, Inc.