On February 27, 2020 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported full-year 2019 financial results (Press release, CytomX Therapeutics, FEB 27, 2020, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-full-year-2019-financial-results" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-full-year-2019-financial-results" rel="nofollow">View Source [SID1234554911]).

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As of December 31, 2019, CytomX had cash, cash equivalents and short-term investments of $296.1 million.

"During 2019, CytomX made substantial progress in advancing our clinical stage pipeline of innovative Probody therapeutics from Phase 1 platform proof of concept into Phase 2 clinical studies. We took major steps forward with our proprietary programs and in our partnerships and set the stage for significant updates throughout 2020," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "We also strengthened our product development capabilities with the recruitment of deeply experienced leadership, positioning ourselves to maximize the potential of our lead, wholly-owned assets, CX-072 and CX-2009, and of our entire portfolio. My colleagues and I look forward to continued pipeline momentum throughout the coming year as we maintain our focus on the discovery, development and ultimate commercialization of potentially transformative cancer treatments."

Business Highlights and Recent Developments

Initiation of Combination Phase 2 Study of CX-072, An Anti-PD-L1 Probody Therapeutic

In the fourth quarter of 2019, CytomX initiated the PROCLAIM (Probody Clinical Assessment In Man) CX-072-002 Phase 2 study evaluating the efficacy and tolerability of the anti-PD-L1 Probody CX-072, in combination with the anti-CTLA-4 antibody Yervoy (ipilimumab), in patients with relapsed or refractory melanoma. Stage 1 of this two-stage trial will enroll up to 40 patients with initial data anticipated in 2020. Additional information is available at ClinicalTrials.gov using the identifier NCT03993379.
Initiation of Phase 2 Study of CX-2009, An Anti-CD166 Probody Drug Conjugate

In the fourth quarter of 2019, CytomX initiated the PROCLAIM CX-2009 Phase 2 expansion study of CX-2009 monotherapy (7 mg/kg, administered every three weeks) in up to 40 patients with hormone receptor (ER, PR) positive, HER2-negative breast cancer. Initial data from this trial is anticipated in 2021. Additional information on this trial is available at ClinicalTrials.gov using the identifier NCT03149549.
Initiation of Part 2a of Ongoing Study by Bristol-Myers Squibb of BMS-986249, An Anti-CTLA-4 Probody Therapeutic

Initiation by Bristol-Myers Squibb of a Phase 2 randomized cohort expansion in its ongoing first-in-human Phase 1/2a trial of the anti-CTLA-4 Probody BMS-986249, a Probody version of the anti-CTLA-4 antibody Yervoy (ipilimumab), in combination with Opdivo (nivolumab) in patients with metastatic melanoma. The advancement of BMS-986249 into this study triggered a milestone payment of $10 million from BMS to CytomX. Additional information is available at ClinicalTrials.gov using the Identifier NCT03369223.
Ongoing Dose Escalation Phase 1 Study of CX-2029, An Anti-CD71 Probody Drug Conjugate, within AbbVie Alliance

Continued patient enrollment by CytomX in the dose escalation phase of PROCLAIM-CX-2029 Phase 1/2 study, partnered with AbbVie, evaluating CX-2029 as monotherapy in patients with solid tumors. Initial data from Phase 1 dose escalation arm is anticipated in 2020 with proof-of-concept data from the first cohort expansion studies in specific tumor types anticipated in 2021. Additional information is available at ClinicalTrials.gov using the Identifier NCT003543813.
Phase 1 Study Initiation by Bristol Myers Squibb of BMS-986288, An Anti-CTLA-4 Probody Therapeutic

In September 2019, Bristol-Myers Squibb initiated the dose escalation phase of a Phase 1/2a clinical study of a second anti-CTLA-4 Probody, BMS-986288, based on a modified version of Yervoy, administered as monotherapy and in combination with Opdivo in patients with selected advanced solid tumors. Additional information is available at ClinicalTrials.gov using the Identifier (NCT03994601).
ImmunoGen Collaboration

In December 2019, CytomX obtained exclusive worldwide development and commercial rights to ImmunoGen’s preclinical epithelial cell adhesion molecule (EpCAM)-targeting program that was developed utilizing CytomX’s Probody technology and ImmunoGen’s drug conjugate technology.
Probody T-Cell Bispecific Program

CytomX’s most advanced program in the T-Cell Bispecific (TCB) modality is an Epidermal Growth Factor Receptor-CD3 TCB which is partnered with Amgen. CytomX anticipates advancing a lead candidate for this program during 2020.
Clinical Development Team Appointments

In October 2019, the Company announced the appointment of Amy C. Peterson, M.D., as executive vice president and chief development officer. In this new role, Dr. Peterson has oversight of a multi-disciplinary team focused on advancing all aspects of CytomX’s clinical development and product registration activities.
In February 2020, the Company announced the appointment of Alison Hannah, M.D., as senior vice president and chief medical officer. In this role, Dr. Hannah oversees CytomX’s clinical development activities.
Anticipated 2020 Milestones

PROCLAIM-CX-072 (Anti-PD-L1)

Data is anticipated from the expansion arms of the Phase 1/2 trial of CX-072 as monotherapy in multiple selected tumor types.
Initial data is anticipated from Stage 1 of the Phase 2 study of CX-072 in combination with Yervoy.
PROCLAIM-CX-2009 (Anti-CD166)

Data is anticipated from the CX-2009 Phase 1 dose escalation and dose ranging studies.
PROCLAIM-CX-2029 (Anti-CD71)

Initial data is anticipated by CytomX and its partner, AbbVie, from the Phase 1 dose escalation stage of the PROCLAIM CX-2029 Phase 1/2 study.
Full Year 2019 Financial Results

Cash, cash equivalents and short-term investments totaled $296.1 million as of December 31, 2019, compared to $436.1 million as of December 31, 2018.

Revenue was $57.5 million for the year ended December 31, 2019, compared to $59.5 million for the year ended December 31, 2018. The net decrease in revenue of $2.0 million for 2019 compared to 2018 was primarily due to a decrease in revenue of $13.1 million from AbbVie under the CD71 Co-Development and Licensing Agreement with AbbVie Unlimited Company (AbbVie), as well as decreases in revenue under our agreements with Amgen, Pfizer and ImmunoGen, partially offset by an increase in revenue from Bristol-Myers Squibb due to the accelerated recognition of revenue related to the cessation of research on certain targets under our agreement with Bristol-Myers Squibb in the first quarter of 2019.

Research and development expenses increased by $27.8 million during the year ended December 31, 2019 compared to the corresponding period in 2018. The increase was largely attributed to an increase in personnel-related expenses; expenses relating to the acquisition of technical know-how during the first quarter of 2019; license fees paid to the University of California, Santa Barbara (UCSB) in connection with an amendment to our license agreement with UCSB in the second quarter of 2019; and an upfront license fee paid to ImmunoGen in the fourth quarter of 2019 for the EpCAM program.

General and administrative expenses increased by $3.3 million during the year ended December 31, 2019 compared to the corresponding period in 2018. The increase was primarily due to an increase in personnel-related expenses due to an increase in headcount.

Teleconference Scheduled Today at 5:00 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:00 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 1686972. An archive of the webcast will be available on the CytomX website from February 27, 2020, until March 6, 2020.

On February 27, 2020 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products reported updated results from the combination arms of the Company’s ongoing Phase 1/2 study investigating ADXS-503 in patients with non-small cell lung cancer (NSCLC) at the Immuno-oncology 360° Conference in New York, New York (Press release, Advaxis, FEB 27, 2020, View Source [SID1234554909]). The trial is evaluating ADXS-503, part of the Company’s ADXS-HOT cancer-type specific immunotherapy program which leverages Advaxis’ proprietary Lm technology platform to target hotspot mutations that commonly occur in specific cancer types as well as other proprietary, tumor-associated antigens, alone and in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy.

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Key findings presented by Dr. Andres Gutierrez, Chief Medical Officer of Advaxis, include:

●Demonstrated substantial tumor shrinkage in the first two evaluable patients in the combination arm, Part B; with ne patient who achieved a partial response (PR) and the other patient who achieved stable disease (SD) with 25% reduction in a target lesion
●These new data build upon previous results presented at the IASLC 2020 Targeted Therapies of Lung Cancer Meeting from seven evaluable patients demonstrating:

○50% (3 of 6) of evaluable patients from the monotherapy arm, from Part A, showed stable disease (SD)
○ADXS-503 monotherapy and in combination with pembrolizumab appeared safe and tolerable in this heavily pretreated population of patients with no dose limiting toxicities observed

"The first two patients treated in the combination arm of ADXS-503 and pembrolizumab were older patients with up to four prior lines of therapy" said Dr. Jonathan W. Goldman, Director of Clinical Trials in Thoracic Oncology at UCLA. "These data are very encouraging as they preliminarily demonstrate the ability of ADXS-503 to enhance or restore sensitivity to checkpoint inhibitors as we had hypothesized during the design of the study based on preclinical data." He added, "We will follow the durability of the responses to therapy and look forward to moving into Part C in ADXS-503 later this year, which will enroll patients for first line therapy in combination with pembrolizumab."

Dr. Andres A. Gutierrez, Chief Medical Officer and EVP at Advaxis , said, "These initial signals of anti-tumor activity in non-small cell lung cancer correlate with the clinical benefit observed with ADXS-PSA and pembrolizumab in our KEYNOTE-046 study in advanced prostate cancer, highlighting the potential synergistic effects of Lm immunotherapies and checkpoint inhibitors."

The Phase 1/2 clinical trial of ADXS-503 will seek to establish the recommended dose, safety, tolerability and clinical activity of ADXS-503 administered alone and in combination with a checkpoint inhibitor in approximately 50 patients with NSCLC, in at least five sites across the U.S. The two dose levels with monotherapy in Part A, (1 X108 and 5 X108 CFU) have been completed and Part B in combination with a checkpoint inhibitor is currently open to enrollment.

About ADXS-HOT

ADXS-HOT is a program that leverages the Company’s proprietary Lm technology to target hotspot mutations that commonly occur in specific cancer types. ADXS-HOT drug candidates are designed to target acquired shared or "public" mutations in tumor driver genes along with other proprietary cancer-testes and oncofetal tumor-associated antigens that also commonly occur in specific cancer types. ADXS-HOT drug candidates are an off-the-shelf treatment, designed to potentially treat all patients with a specific cancer type, without the need for pretreatment biomarker testing, DNA sequencing or diagnostic testing.

About KEYNOTE-046

KEYNOTE-046 (NCT02325557) is a Phase 1/2 open-label, multicenter, dose-determination and expansion trial that evaluates the safety, tolerability and preliminary clinical activity of ADXS-PSA as monotherapy (Part A; n=14 [13 treated]), and in combination with KEYTRUDA (Part B; n= 37) in heavily pretreated patients with progressive and refractory metastatic castration-resistant prostate cancer (mCRPC).

On February 27, 2020 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Nektar Therapeutics, FEB 27, 2020, View Source [SID1234554908]).

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Cash and investments in marketable securities at December 31, 2019 were approximately $1.6 billion as compared to $1.9 billion at December 31, 2018.

"Nektar’s progress over the past year has established a strong foundation for growth, with a robust portfolio of clinical-stage immuno-oncology and immunology candidates addressing multiple therapeutic areas," said Howard W. Robin, President and CEO of Nektar. "Our amended joint development plan with Bristol-Myers Squibb for bempegaldesleukin in combination with Opdivo expands the active registrational program for the doublet to five indications, including new Phase 3 studies in the adjuvant melanoma setting and muscle invasive bladder cancer. It also provides a path forward in first-line lung cancer and enhances our ability to pursue new combinations in additional indications."

Mr. Robin continued, "We also advanced NKTR-255, a novel IL-15 agonist that stimulates NK cells and memory T cells, into the clinic in combination with ADCC therapies. With NKTR-358, we have an opportunity to address the underlying immune imbalance associated with multiple autoimmune and chronic inflammatory diseases. Our partner Eli Lilly is on track to initiate a Phase 2 study in lupus, advance ongoing Phase 1b clinical trials in psoriasis and atopic dermatitis, and start an additional Phase 2 study in a new autoimmune indication this year."

Summary of Financial Results

Revenue in the fourth quarter of 2019 was $33.9 million as compared to $39.8 million in the fourth quarter of 2018. Revenue for the year ended December 31, 2019 was $114.6 million as compared to $1.2 billion in 2018 and was lower primarily due to the recognition of $1.06 billion of license revenue from the Bristol-Myers Squibb collaboration agreement in the second quarter of 2018.

Total operating costs and expenses in the fourth quarter of 2019 were $143.5 million as compared to $140.1 million in the fourth quarter of 2018. Total operating costs and expenses for 2019 were $554.7 million as compared to $505.4 million in 2018. Total operating costs and expenses increased primarily as a result of increases in research and development (R&D) expense and general and administrative (G&A) expense.

R&D expense in the fourth quarter of 2019 was $110.4 million as compared to $108.9 million for the fourth quarter of 2018. R&D expense for the year ended December 31, 2019 was $434.6 million as compared to $399.5 million in 2018. R&D expense was higher in 2019 as compared to 2018 primarily because of the continued clinical development of bempegaldesleukin, including the registrational studies in melanoma, bladder cancer and renal cell carcinoma, and manufacture of Phase 2 drug supply for NKTR-358, which were partially offset by lower bempegaldesleukin and NKTR-181 manufacturing costs.

G&A expense was $27.1 million in the fourth quarter of 2019 as compared to $23.8 million in the fourth quarter of 2018. G&A expense for 2019 was $98.7 million as compared to $81.4 million in 2018. G&A expense was higher in the fourth quarter and full year 2019 as compared to the same periods in 2018 primarily due to non-cash stock based compensation expense, limited commercialization readiness activities for NKTR-181, as well as other costs related to personnel, facilities and outside services.

Net loss for the fourth quarter of 2019 was $112.2 million or $0.64 basic and diluted loss per share as compared to a net loss of $98.2 million or $0.57 basic and diluted loss per share in the fourth quarter of 2018. Net loss for the year ended December 31, 2019 was $440.7 million or $2.52 diluted loss per share as compared to net income of $681.3 million or $3.78 diluted earnings per share in 2018.

2019 and Year-to-Date Business Highlights:

·In February 2020, Nektar announced the publication of preclinical bempegaldesleukin data in two manuscripts in Nature Communications showing how bempegaldesleukin works synergistically with multiple immune-based therapies to enhance T-cell-mediated tumor control.

·In January 2020, Nektar and Bristol-Myers Squibb announced a new joint development plan that expands the ongoing registrational program for bempegaldesleukin plus Opdivo (nivolumab) from three ongoing registrational trials in first-line metastatic melanoma, first-line cisplatin-ineligible metastatic urothelial cancer and first-line metastatic renal cell carcinoma (RCC) to include two additional registrational trials in adjuvant melanoma and muscle-invasive bladder cancer. In addition, a Phase 1/2 study will be initiated to evaluate bempegaldesleukin plus nivolumab in combination with axitinib in first-line RCC in order to support a future registrational trial. Bristol-Myers Squibb will also independently conduct and fund a Phase 1/2 study in first-line non-small-cell lung cancer with bempegaldesleukin and nivolumab.

●In January 2020, Nektar made the strategic business decision to withdraw its New Drug Application (NDA) for NKTR-181, an investigational medicine in development for chronic pain and make no further investment into the program.

●In December 2019, Nektar presented results from preclinical studies of NKTR-255, its IL-15 agonist, at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting highlighting the candidate’s potential in the treatment of hematological malignancies by restoring both NK cell and memory CD8 T cell compartments in patients.

In November 2019, Nektar presented updated results from the first-in-human Phase 1a study of NKTR-358 at the 2019 Annual Meeting of the American College of Rheumatology supporting development of the candidate as a first-in-class T regulatory cell stimulator for the treatment of autoimmune and other chronic inflammatory conditions.

●In November 2019, Nektar presented new data from the Stage IV front-line melanoma cohort in the PIVOT-02 study at the 2019 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. At a median time of follow-up of 18.6 months, median progression free survival had not yet been reached.

●In October 2019, Nektar announced that its partner Eli Lilly initiated two Phase 1b studies of NKTR-358, one in patients with psoriasis and one in patients with atopic dermatitis.

●In October 2019, Nektar announced the initiation of a first-in-human, Phase 1 clinical study evaluating NKTR-255 as monotherapy for patients with relapsed or refractory non-Hodgkin lymphoma or multiple myeloma.

·In September 2019, Nektar presented clinical data from its PIVOT-02 study for bempegaldesleukin in combination with Opdivo (nivolumab) at the 2019 CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) demonstrating the promising clinical activity of the combination in patients with advanced or metastatic triple-negative breast cancer, particularly in patients with PD-L1 negative baseline tumors.

●In August 2019, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for bempegaldesleukin in combination with Opdivo (nivolumab) for the treatment of patients with previously untreated unresectable or metastatic melanoma.

●In June 2019, Nektar presented biomarker and clinical data from the ongoing PIVOT-02 study for bempegaldesleukin in combination with Opdivo (nivolumab) at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. Clinical data presented included 12-month follow-up for the Stage IV first-line melanoma patient cohort and showed a deepening and durability of response over time.

●In April 2019, Nektar presented positive preclinical data on its immuno-oncology pipeline candidates, bempegaldesleukin and NKTR-255, at the 2019 AACR (Free AACR Whitepaper) Annual Meeting.

●In March 2019, Nektar presented preliminary immune activation, safety and clinical activity data from the ongoing dose-escalation stage of the REVEAL study at the 2019 ASCO (Free ASCO Whitepaper)-SITC Meeting. The REVEAL Phase 1/2 study is evaluating the safety and efficacy of NKTR-262, a novel TLR agonist, in combination with bempegaldesleukin.

●In February 2019, Nektar presented clinical data from first-line Stage IV urothelial carcinoma patients enrolled in the PIVOT-02 study of bempegaldesleukin with Opdivo (nivolumab) at the 2019 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium.

The company also announced upcoming presentations at the following scientific congresses:

Society of Toxicology (SOT) 59th Annual Meeting, Anaheim, CA

●Presentation: "Bempegaldesleukin (NKTR-214), a novel IL-2 based immunotherapy, demonstrates superior nonclinical safety compared to that reported for recombinant human IL-2 (rhIL-2)", Leung, S., et al.

oSession: Safety Assessment: Pharmaceutical—Drug Development
oDate: Wednesday, March 18th, 10:45 a.m. – 12:30 p.m.

●Presentation: "Toxicology Species Selection for Preclinical Safety Assessment of TLR7/8 Prodrug Agonist", Gunther, J., et al.

oSession: Safety Assessment: Pharmaceutical—Drug Development
oDate: Wednesday, March 18th, 10:45 a.m. – 12:30 p.m.

American Chemical Society National Meeting

●Presentation: "NKTR-262: Discovery of a novel TLR 7/8 agonist prodrug that demonstrates synergistic anti-tumor effect in combination with NKTR-214, a CD-122 preferential IL-2 pathway agonist", Anand, N., et al.

oSession: MEDI: Tissue Specific Delivery: TLR Agonists
oDate: Tuesday, March 24th, 10:10 a.m. – 10:45 a.m.

Conference Call to Discuss Fourth Quarter and Year-End 2019 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, Thursday, February 27, 2020.

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 March 27, 2020.

To access the conference call, follow these instructions:

Dial: (877) 881-2183 (U.S.); (970) 315-0453 (international)

Passcode: 2507828 (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.

On February 27, 2020 Lipocine Inc. (NASDAQ:LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported the closing of a registered direct offering of 10,084,034 Class A Units, each consisting of one share of its common stock and one half of a common warrant to purchase one share of its common stock, at a price of $0.595 per Class A Unit, for total gross proceeds to the Company of approximately $6 million, before deducting placement agent fees and other estimated offering expenses (Press release, Lipocine, FEB 27, 2020, View Source [SID1234554907]). The Company intends to use the net proceeds from this offering for working capital and general corporate purposes.

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Roth Capital Partners acted as sole agent for the offering.

The offering was made pursuant to a shelf registration statement on Form S-3 (File No. 333-220942) (including a prospectus) previously filed with and declared effective by the U.S. Securities and Exchange Commission (the "SEC"). A prospectus supplement describing the terms of the offering has been filed with the SEC.

A copy of the prospectus supplement and the accompanying prospectus relating to these securities may be obtained by contacting Roth Capital Partners, 888 San Clemente Drive, Suite 400, Newport Beach, CA 92660, (800) 678-9147. Electronic copies of the prospectus supplement and the accompanying prospectus are also available free of charge on the website of the SEC at www.sec.gov.

This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, nor will there be any sale of the securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On February 17, 2020 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the fourth quarter and full year ended December 31, 2019, 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 (Press release, Rigel, FEB 27, 2020, View Source [SID1234554906]).

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"We are entering 2020 with a clear plan to drive shareholder value for Rigel," said Raul Rodriguez, Rigel’s president and CEO. "We see substantial opportunities for TAVALISSE to meet patient needs in the growing adult chronic ITP market, particularly as an early line therapy. Utilization in these less refractory patients continues to grow with the support of ongoing physician education and data generation. To expand the range of TAVALISSE indications, we are conducting a Phase 3 trial in warm AIHA and expect to complete patient enrollment midyear. In addition, we are extremely excited about the potential of our early stage candidates and are currently exploring partnership opportunities that would enable Rigel to realize near-term value while also participating meaningfully in the upside of these programs."

Business Update

At the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in December 2019, the company presented post-hoc data analysis from its Phase 3 clinical program of TAVALISSE in adult patients with chronic ITP. In this analysis, 32 patients received fostamatinib as a second-line therapy, of which, 78% (25/32) achieved ≥1 platelet count of ≥50,000/µL (without rescue therapy). Adverse events were manageable and consistent with those previously reported with fostamatinib. These data highlight the potential benefit of using TAVALISSE in earlier lines of therapy.

In February 2020, Rigel received a $20.0 million payment from its collaborative partner Grifols, S.A. (Grifols). The payment is comprised of $17.5 million for the European Commission’s approval of the marketing authorization application for fostamatinib for the treatment of chronic immune thrombocytopenia in adult patients who are refractory to other treatments and a $2.5 million creditable advance royalty payment based on the terms of the collaboration agreement. Fostamatinib will be marketed in Europe under the brand name TAVLESSE (fostamatinib). Grifols is planning to launch the product in the second quarter of 2020.

Enrollment is ongoing in Rigel’s Phase 3 pivotal trial in warm AIHA, FORWARD (Fostamatinib Research in Warm Antibody AIHA Disease). A total of 34 patients have been randomized to date. The trial remains on track to complete enrollment in mid-2020.

In February 2020, Rigel’s partner Kissei Pharmaceuticals Co., Ltd. (Kissei) was granted orphan drug designation from the Japanese Ministry of Health, Labour and Welfare for R788 (fostamatinib) in chronic idiopathic thrombocytopenic purpura.

Financial Update

For the fourth quarter of 2019, Rigel reported a net loss of $17.2 million, or $0.10 per share, compared to net income of $3.2 million, or $0.02 per share, in the same period of 2018.

In the fourth quarter of 2019, total revenues were $15.4 million, consisting of $13.8 million in net product sales and $1.6 million in contract revenues from collaborations. Net product sales of $13.8 million increased by 90% compared to $7.3 million in the fourth quarter of 2018. This increase reflects the expanding patient and prescriber base for TAVALISSE and the growing persistency rate for refills at month 4, which is approximately 54%.

Contract revenues from collaborations of $1.6 million for the fourth quarter ended December 31, 2019 consists of a $1.5 million fee earned pursuant to an amendment of the license and collaboration agreement with Aclaris Therapeutics, Inc. (Aclaris) in October 2019, as well as deferred revenue from Rigel’s collaboration with Grifols related to the performance of certain research and development services.

Rigel reported total costs and expenses of $32.7 million in the fourth quarter of 2019, compared to $35.3 million for the same period in 2018. The decrease in costs and expenses was primarily due to decreases in personnel-related expenses and various third-party costs.

For the full year ended December 31, 2019, Rigel reported a net loss of $66.9 million, or $0.40 per share, compared to a net loss of $70.5 million, or $0.44 per share, for the same period of 2018.

Rigel reported total revenues of $59.3 million for the year ended December 31, 2019, consisting of $43.8 million in net product sales and $15.5 million in revenues related to Rigel’s collaboration agreements with Grifols, Kissei, Aclaris, and Impact Biomedicines, Inc.

Total costs and expenses for the year ended December 31, 2019, were $128.4 million, compared to $117.2 million, for the same period of 2018. The increase in total costs and expenses was primarily due to the increases in third party costs related to Rigel’s ongoing pivotal Phase 3 study in warm AIHA, personnel-related costs, on-going commercialization of TAVALISSE in adult chronic ITP, and research and development costs related to its Phase 1 study in RIP.

As of December 31, 2019, Rigel had cash, cash equivalents and short-term investments of $98.1 million, compared to $128.5 million as of December 31, 2018. Rigel previously announced that in September 2019, we entered into a $60.0 million term loan credit facility with MidCap Financial. At closing,

$10.0 million was funded to Rigel in an initial tranche. The facility also gives Rigel the ability to access an additional $50.0 million, of which $40.0 million is subject to the achievement of certain customary conditions.

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 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 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 R5521

The investigational candidate, R552, is an orally available, potent and selective inhibitor of receptor-interacting protein kinase (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 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 trademark of Rigel Pharmaceuticals, Inc.