On February 20, 2020 Reata Pharmaceuticals, Inc. (Nasdaq: RETA) ("Reata," the "Company," or "we"), a clinical-stage biopharmaceutical company, reported financial results for the fourth quarter and full year ended December 31, 2019, and provided an update on the Company’s business and product development programs (Press release, Reata Pharmaceuticals, FEB 20, 2020, View Source;source=gmail&ust=1582354855075000&usg=AFQjCNHkerl-gpEGVj67JhlxLel2BJQtHw [SID1234554566]).

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"In 2019, we made significant progress toward our goal of becoming a global, fully-integrated biopharmaceutical company," said Warren Huff, Reata’s Chief Executive Officer and President. "We announced positive, pivotal data from our lead franchises, chronic kidney disease (CKD) and neurology, and are actively preparing for commercial launch in the United States and abroad for bardoxolone methyl (bardoxolone) in CKD caused by Alport syndrome, and omaveloxolone in Friedreich’s ataxia, two severe and life-threatening diseases without approved therapies. We also successfully capitalized the Company to fund the development of our pipeline assets and advance our lead drug candidates and early-stage drug candidates into new indications with high unmet need."

Reata 2019 Pipeline Highlights

Reported positive data from the pivotal part 2 portion of the Phase 2 MOXIe study of omaveloxolone in patients with Friedreich’s ataxia.
Reported positive data from year one of the pivotal Phase 3 portion of the CARDINAL study of bardoxolone in patients with CKD caused by Alport syndrome.
Launched the pivotal, global Phase 3 FALCON study of bardoxolone in patients with CKD caused by autosomal dominant polycystic kidney disease (ADPKD).
Completed enrollment in the pivotal Phase 3 CATALYST study of bardoxolone in patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH).
Reata 2019 Corporate Highlights

Reacquired the development, manufacturing, and commercialization rights concerning our proprietary Nrf2 activator product platform, including bardoxolone and omaveloxolone, originally licensed to AbbVie, Inc. (AbbVie) for territories outside of the United States and excluding, for bardoxolone, certain Asian countries previously licensed to Kyowa Kirin Co., Ltd. (KKC).
Completed an underwritten public offering of 2,760,000 shares of Class A common stock at a price to the public of $183.00 per share, resulting in gross proceeds of $505 million.
Fourth Quarter Financial Highlights

Cash and Cash Equivalents

At December 31, 2019, we had cash and cash equivalents of $664.3 million, as compared to $337.8 million at December 31, 2018.

GAAP and Non-GAAP Net Loss

The net loss according to generally accepted accounting principles in the U.S. (GAAP) for the fourth quarter of 2019 was $186.9 million, or $5.91 per share, on both a basic and diluted basis, as compared to a GAAP net loss of $25.6 million, $0.86 per share, on both a basic and diluted basis, for the same period of the year prior. For the twelve months ended December 31, 2019, the GAAP net loss was $290.2 million, or $9.54 per share, on both a basic and diluted basis, as compared to a GAAP net loss of $80.5 million, or $2.91 per share, on both a basic and diluted basis, for the same period of the year prior.

The increase in GAAP net loss is driven primarily by a Reacquired license right expense of $124.4 million related to the amended and restated license agreement we entered into with AbbVie in October 2019 (Reacquisition Agreement) to reacquire the development, manufacturing, and commercialization rights concerning our proprietary Nrf2 activator product platform, as well as increases in stock based compensation expense related to the growth of our personnel.

The non-GAAP net loss for the fourth quarter of 2019 was $50.3 million, or $1.59 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $22.8 million, $0.77 per share, on both a basic and diluted basis, for the same period of the year prior. For the twelve months ended December 31, 2019, the non-GAAP net loss was $139.4 million, or $4.58 per share, on both a basic and diluted basis, as compared to a non-GAAP net loss of $70.0 million, or $2.53 per share, on both a basic and diluted basis, for the same period of the year prior.

The non-GAAP net loss excludes stock-based compensation and reacquired license rights expense. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in the press release.

Revenue

Revenue was $2.7 million in the fourth quarter of 2019, as compared $8.5 million for the same period of the year prior. Revenue for the fourth quarter of 2019 included $0.7 million from the AbbVie collaboration agreement, $1.2 million from the KKC license agreement, and $0.8 million from other sources. Revenue was $26.5 million for the twelve months ended December 31, 2019, as compared to $53.6 million, for the same period of the year prior. Revenue for the twelve months ended December 31, 2019 included $20.6 million from the AbbVie collaboration agreement, $4.7 million from the KKC license agreement, and $1.2 million from other sources.

GAAP and Non-GAAP Research and Development (R&D) Expenses

GAAP R&D expenses were $40.2 million for the fourth quarter of 2019, as compared to $25.3 million, for the same period of the year prior. GAAP R&D expenses were $128.1 million for the twelve months ended December 31, 2019, as compared to $97.3 million, for the same period of the year prior.

Non-GAAP R&D expenses were $36.7 million for the fourth quarter of 2019, as compared to $24.3 million, for the same period of the year prior. Non-GAAP R&D expenses were $119.4 million for the twelve months ended December 31, 2019, as compared to $93.3 million, for the same period of the year prior.

The non-GAAP R&D expenses exclude stock-based compensation expense. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP R&D expenses appearing later in the press release.

GAAP and Non-GAAP General and Administrative (G&A) Expenses

GAAP G&A expenses were $22.3 million for the fourth quarter of 2019, as compared to $7.9 million, for the same period of the year prior. GAAP G&A expenses were $58.3 million for the twelve months ended December 31, 2019, as compared to $32.7 million, for the same period of the year prior.

Non-GAAP G&A expenses were $13.4 million for the fourth quarter of 2019, as compared to $6.2 million, for the same period of the year prior. Non-GAAP G&A expenses were $40.6 million for the twelve months ended December 31, 2019, as compared to $26.1 million, for the same period of the year prior.

The non-GAAP G&A expenses excludes stock-based compensation expense. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP G&A expenses appearing later in the press release.

Operating Capital Requirements

We believe our existing cash and cash equivalents will be sufficient to enable us to fund our operations through the end of 2021.

Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including non-GAAP R&D expenses, non-GAAP G&A expenses, non-GAAP operating expenses, non-GAAP net loss and non-GAAP net loss per common share – basic and diluted. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The Company defines non-GAAP R&D expenses as GAAP R&D expenses less stock-based compensation expense, non-GAAP G&A expenses as GAAP G&A expenses less stock-based compensation expense, non-GAAP operating expenses as GAAP operating expenses less stock-based compensation expense and reacquired license rights expense, non-GAAP net loss as GAAP net loss plus stock-based compensation expense and reacquired license rights expense and non-GAAP net loss per common share – basic and diluted as GAAP net loss per common share – basic and diluted plus stock-based compensation expense and reacquired license rights expense. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of reacquired license rights expense because the Company believes its impact makes it difficult to compare its results to prior periods and anticipated future periods.

Because management believes certain items such as stock-based compensation expense and reacquired license rights expense can distort the trends associated with the Company’s ongoing performance, the following measures are often provided, excluding special items, and utilized by the Company’s management, analysts, and investors to enhance consistency and comparability of year-over-year results, as well as to industry trends, and to provide a basis for evaluating operating results in future periods: non-GAAP net loss; non-GAAP net loss per common share – basic and diluted; non-GAAP R&D expenses; non-GAAP G&A expenses; and non-GAAP operating expenses.

The Company believes the presentation of these non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with these non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between these non-GAAP measures and the most directly comparable GAAP measures is provided later in this press release.

On February 20, 2020 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported that it will report its fourth quarter and year end 2019 financial results after market close on Thursday, February 27, 2020 (Press release, Rigel, FEB 20, 2020, View Source [SID1234554584]). Rigel senior management will follow the announcement with a live conference call and webcast at 4:30pm Eastern Time (1:30pm Pacific Time) to discuss the financial results.

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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 for 90 days after the call via the Rigel website.

On February 20, 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) reported that the loss of one copy of the miR15a/miR16-1 gene cluster promoted initiation and progression of multiple myeloma in mice, according to results published online in Blood Cancer Discovery, the latest journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, AACR (Free AACR Whitepaper), FEB 20, 2020, View Source [SID1234554626]).

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Multiple myeloma is a cancer of antibody-producing cells called plasma cells. It is preceded by a premalignant condition known as monoclonal gammopathy of undetermined significance (MGUS), in which abnormal plasma cells are present but do not expand. A hallmark of both MGUS and multiple myeloma is the detection of an M spike, which indicates the accumulation of an abnormal secreted antibody in the patient’s blood.

"The risk of progression from MGUS to malignant multiple myeloma is approximately 1 percent each year, but the factors that contribute to progression are not well understood," said Marta Chesi, PhD, associate professor of medicine at the Mayo Clinic. "The purpose of our study was to model genetic risk factors that may contribute to initiation and progression of multiple myeloma. This understanding could eventually allow us to identify the mechanisms that increase the risk of progressing to multiple myeloma."

One copy of chromosome 13 is deleted in approximately half of patients with MGUS and multiple myeloma; however, the significance of this deletion on prognosis and disease progression remains controversial. Chesi and colleagues hypothesized that individual genes on chromosome 13 may promote disease initiation and/or progression.

The authors examined the impact of two genetic loci found on human chromosome 13 – RB1 and MIR15A/MIR16-1 – on disease initiation and progression. Both RB1 and MIR15A/MIR16-1 are considered tumor suppressor genes due to their roles in regulating cellular proliferation. The RB1 protein is known to be inactivated in many cancers, including multiple myeloma. A previous study demonstrated that deletion of MIR15A/MIR16-1 enhances proliferation of human B cells, and deletion of the gene in mice promotes development of another blood cancer, chronic lymphocytic leukemia.

In this study, the authors deleted a single copy of either Rb1 or miR15a/miR16-1 in wild-type mice and in a transgenic mouse model of multiple myeloma they previously developed. The authors found that deletion of one copy of Rb1 did not affect disease initiation or progression. In contrast, deleting one copy of miR15a/miR16-1 in wild-type mice significantly accelerated the development of an M-spike. Furthermore, the deletion of one copy of miR15a/miR16-1 in mice with multiple myeloma significantly enhanced the aggressiveness of the disease and led to increased expression of genes that promote cellular proliferation.

The authors also analyzed a genetic dataset of multiple myeloma patients and found that deletion of one copy of MIR15A/MIR16-1 in patient tumors was associated with increased expression of the same cellular proliferation genes that were upregulated in mice.

"Losing one copy of the MIR15A/MIR16-1 gene appears to promote tumor cell proliferation in both mice and patients," said Chesi. "For many years, we thought that deletion of chromosome 13 was just a byproduct of other genetic changes in the tumor and that it did not directly affect disease progression. Our study now demonstrates that deletion of chromosome 13, and specifically deletion of MIR15A/MIR16-1, appears to alter the biology of the tumor.

"However, the fact that the entire chromosome 13, and not just MIR15A/MIR16-1, is lost in many cases of MGUS or multiple myeloma suggests that other genes on this chromosome are also likely to be important for pathogenesis," added Chesi. In particular, Chesi is interested in studying DIS3, another gene located on chromosome 13 that is frequently mutated in multiple myeloma. The authors were not able to assess its contribution in this study due to the lack of relevant mouse models.

Another limitation of the study is that it was not possible to delete Rb1 or miR15a/miR16-1 only in myeloma cells due to technical restrictions of their mouse model. As a result, these genes were deleted in all cells, including immune cells, which could have led to indirect effects on disease progression, explained Chesi. However, Chesi added that results from additional control experiments indicate that the observed results are unlikely to be indirect, as transplanted tumors behaved similarly in both wild-type and miR15a/miR16-1-deleted mice. This study was sponsored by grants from the National Institutes of Health. Chesi declares no conflict of interest.

On February 20, 2020 Pacira BioSciences, Inc. (Nasdaq: PCRX) reported financial results for the fourth quarter and full-year of 2019 and provided 2020 financial guidance (Press release, Pacira Pharmaceuticals, FEB 20, 2020, View Source;991.htm [SID1234554551]).

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"By every measure, 2019 was an outstanding year for Pacira. We are delighted to report record revenues for EXPAREL with our sixth consecutive quarter of more than 20 percent growth," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "Demand has continued to mount within the anesthesia community as EXPAREL-based nerve and field blocks take hold as institutional protocol. Additionally, we enhanced our non-opioid pain management product portfolio with the addition of iovera° and we are seeing great interest from the marketplace around this innovative system."

"Looking forward, we intend to capitalize on this momentum with robust topline growth that will drive substantial operating leverage and cash flow, providing significant financial flexibility to invest in future growth opportunities. Our mission for 2020 remains steadfast as we continue to propel our global leadership in non-opioid pain management and deliver multiple milestones, including the publication of new data in C-section, label-expansion initiatives for pediatrics and lower extremity nerve block, and the advancement of regulatory activities in Europe, Canada, and China," added Mr. Stack.

2019 Full-Year and Fourth Quarter Financial Highlights

Full-year revenues of $421.0 million and fourth quarter revenues of $122.4 million.

Full-year EXPAREL (bupivacaine liposome injectable suspension) net product sales of $407.9 million and fourth quarter EXPAREL net product sales of $116.9 million.

Full-year iovera° net product sales were $7.9 million and fourth quarter iovera° net product sales of $3.2 million. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.

Full-year GAAP net loss of $11.0 million or $0.27 per share (basic and diluted).

Full-year non-GAAP net income of $70.7 million or $1.67 per diluted share.

Fourth quarter GAAP net loss of $4.9 million or $0.12 per share (basic and diluted).

Fourth quarter non-GAAP net income of $23.8 million or $0.56 per diluted share.

Recent Highlights

Launch of national regional anesthesia training initiative with Envision Physician Services.
In January 2020, Pacira announced a collaboration with Envision Physician Services to train anesthesiology clinicians on ultrasound-guided regional anesthesia techniques utilizing long-acting local anesthetics like EXPAREL via a series of interactive workshops held across the country. The program supports the ongoing efforts by both organizations to advance the delivery of high-quality, patient-centered care.

EXPAREL achieves primary and key secondary endpoints in Phase 4 CHOICE study in cesarean section patients. In January 2020, Pacira announced that its Phase 4 study of EXPAREL in patients undergoing Cesarean section achieved its primary endpoint with a statistically significant reduction in total postsurgical opioid consumption while maintaining pain scores through 72 hours (P≤0.001). EXPAREL demonstrated statistical significance for the key secondary endpoint of a reduction in the incidence and severity of itching for 72 hours after surgery (P≤0.05). Full study results will be submitted for publication in the peer-reviewed medical literature later this year.

Phase 3 PLAY study of EXPAREL in pediatric patients achieves positive results. In December 2019, Pacira announced positive results from its Phase 3 PLAY study of EXPAREL administered as a single-dose infiltration in pediatric patients undergoing spinal or cardiac surgeries. Overall findings were consistent with the pharmacokinetic and safety profiles for adult patients with no safety concerns identified at a dose of 4 mg/kg. These results will provide the foundation for the company’s supplemental New Drug Application submission in the first half of 2020 to the U.S. Food and Drug Administration (FDA) seeking expansion of the EXPAREL label to include children aged six and over.

Fourth Quarter 2019 Financial Results

Total revenues were $122.4 million in the fourth quarter of 2019, a 29% increase over the $95.1 million reported for the fourth quarter of 2018.

EXPAREL net product sales were $116.9 million in the fourth quarter of 2019, a 24% increase over the $94.4 million reported for the fourth quarter of 2018.

Fourth quarter iovera° net product sales were $3.2 million. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.

Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $1.7 million in the fourth quarter of 2019, compared to $0.3 million in 2018.

Fourth quarter royalty revenue was $0.6 million compared to $0.4 million in 2018.

Total operating expenses were $120.7 million in the fourth quarter of 2019, compared to $82.9 million in the fourth quarter of 2018.

Research and development (R&D) expenses were $19.7 million in the fourth quarter of 2019, compared to $14.2 million in the fourth quarter of 2018. The company’s R&D expenses include $8.7 million and $6.5 million of product development and manufacturing capacity expansion costs in the fourth quarters of 2019 and 2018, respectively.

Selling, general and administrative (SG&A) expenses were $54.2 million in the fourth quarter of 2019, compared to $44.6 million in the fourth quarter of 2018.

GAAP net loss was $4.9 million, or $0.12 per share (basic and diluted) in the fourth quarter of 2019, compared to GAAP net income of $8.3 million, or $0.20 per share (basic and diluted), in the fourth quarter of 2018.

Non-GAAP net income was $23.8 million, or $0.57 per share (basic) and $0.56 per share (diluted), in the fourth quarter of 2019, compared to non-GAAP net income of $19.8 million, or $0.48 per share (basic) and $0.47 per share (diluted), in the fourth quarter of 2018.

Pacira had 41.8 million basic weighted average shares of common stock outstanding in the fourth quarter of 2019.

For non-GAAP measures, Pacira had 42.6 million diluted weighted average shares of common stock outstanding in the fourth quarter of 2019.

Full-Year 2019 Financial Results

Total revenues were $421.0 million in 2019, a 25% increase over the $337.3 million reported in 2018.

EXPAREL net product sales were $407.9 million in 2019, a 23% increase over the $331.1 million reported in 2018.

Full-year iovera° net product sales were $7.9 million. Pacira began recognizing sales of iovera° in April 2019 after completing its acquisition of MyoScience, Inc., a privately held medical technology company.

Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $3.2 million in 2019, compared to $1.3 million in 2018.

Full-year royalty revenue was $2.1 million compared to $1.9 million in 2018.

Total operating expenses were $410.5 million in 2019, compared to $321.4 million in 2018.

Research and development (R&D) expenses were $72.1 million in 2019, compared to $55.7 million in 2018. The company’s R&D expenses include $29.7 million and $28.5 million of product development and manufacturing capacity expansion costs in 2019 and 2018, respectively.

Selling, general and administrative (SG&A) expenses were $200.8 million in 2019, compared to $177.3 million in 2018.

GAAP net loss was $11.0 million, or $0.27 per share (basic and diluted) in 2019, compared to a GAAP net loss of $0.5 million, or $0.01 per share (basic and diluted) in 2018.

Non-GAAP net income was $70.7 million, or $1.70 per share (basic) and $1.67 per share (diluted), in 2019, compared to non-GAAP net income of $43.5 million, or $1.06 per share (basic) and $1.04 per share (diluted), in 2018.

Pacira ended 2019 with cash, cash equivalents, short-term and long-term investments ("cash") of $356.7 million. Cash provided by operations was $70.5 million in 2019, compared to $48.9 million in 2018.

Pacira had 41.5 million basic weighted average shares of common stock outstanding in 2019.

For non-GAAP measures, Pacira had 42.4 million diluted weighted average shares of common stock outstanding in 2019.

2020 Outlook
Pacira announces its full-year 2020 financial guidance as follows. Pacira expects:

Total revenues to be between $485 million and $500 million

EXPAREL net product sales to be between $465 million and $475 million;

iovera° net product sales to be between $15 million and $20 million;

Non-GAAP gross margins to be between 76% and 78%;

Non-GAAP research and development (R&D) expense to be between $60 million to $70 million;

Non-GAAP selling, general and administrative (SG&A) expense to be between $180 million and $190 million; and

Stock-based compensation to be between $35 million and $40 million.
See "Non-GAAP Financial Information" and "Reconciliations of GAAP to Non-GAAP 2020 Financial Guidance" below.
Today’s Conference Call and Webcast Reminder
The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Thursday, February 20, 2020, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 8765839. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 8765839. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.
Non-GAAP Financial Information
This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income, non-GAAP net income per share, non-GAAP cost of goods sold, non-GAAP gross margins, non-GAAP research and development (R&D) expense and non-GAAP selling, general and administrative (SG&A) expense, because such measures exclude milestone revenue; acquisition-related charges, product discontinuation costs and other expense; stock-based compensation; amortization of debt discount; amortization of acquired intangible assets; an income tax benefit and step-up in basis of inventory in connection with the acquisition of MyoScience, Inc.; and loss on investment and other non-operating income.

These measures supplement Pacira’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, gross margins, R&D expense and SG&A expense outlook for 2020 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at Pacira and its future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP

measures, and a reconciliation of our GAAP to non-GAAP 2020 financial guidance for gross margins, R&D expense and SG&A expense.

On February 20, 2020 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and leader in predictive target discovery, reported its plan to initiate a Phase 1/2 study evaluating a triple combination of Compugen’s COM701, an investigational anti-PVRIG antibody, in combination with Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor Opdivo (nivolumab) and BMS-986207, Bristol-Myers Squibb’s investigational anti-TIGIT antibody (Press release, Compugen, FEB 20, 2020, View Source [SID1234554585]).

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The triple combination study is designed to evaluate the blockade of the three immune checkpoint pathways – PVRIG, TIGIT and PD-1, and will accelerate the clinical evaluation of Compugen’s science-driven DNAM axis hypothesis in various advanced solid tumors. The study is expected to commence in the second half of 2020, following the clearance of a new Investigational New Drug Application by the U.S. Food and Drug Administration. Compugen will be the study sponsor with Opdivo and BMS-986207 supplied by Bristol-Myers Squibb.

"We are excited to expand our collaboration with Bristol-Myers Squib with this biomarker-informed triple combination study to accelerate the clinical evaluation of COM701," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "The triple combination regimen allows us to ultimately test our science-driven hypothesis that the dual inhibition of the DNAM axis with PVRIG and TIGIT blockers, together with the inhibition of the PD-1 pathway, will enable robust activation of T cells leading to anti-tumor immune responses in cancer patients who are non-responsive or refractory to PD-1 blockers."

"The initial encouraging signals of anti-tumor activity observed in heavily pretreated patients in the monotherapy dose escalation arm of our ongoing Phase 1 study, paired with our strong scientific rationale and preclinical data, support to our decision to evaluate whether the combination of these three immune checkpoint inhibitors improve patient outcomes and broaden the patient population that will respond to immunotherapies," added Dr. Cohen-Dayag.

Under the existing collaboration with Bristol-Myers Squibb, COM701 is being investigated as a monotherapy and in combination with Opdivo in an ongoing Phase 1 study. Following the Companies’ joint decision to move forward with a triple combination study, Compugen will complete the dose escalation arm of the dual combination of COM701 with Opdivo under its ongoing Phase 1 study. Future studies evaluating COM701 in combination with a PD-1 inhibitor in specific tumor types will be assessed at a later date. As previously indicated, Compugen plans to present initial data from the combination dose escalation study of COM701 with Opdivo in the second half of 2020. Compugen will continue to advance the biomarker informed monotherapy expansion arm of the ongoing COM701 Phase 1 study, as planned.

The planned open-label Phase 1/2 trial is designed to evaluate the safety, tolerability and antitumor activity of COM701 in combination with Opdivo and BMS-986207. The study will evaluate a safe and tolerable dose of the combination during dose escalation and antitumor activity in selected tumor types in the expansion cohorts (ovarian cancer, endometrial cancer and a biomarker-driven arm of tumor types with high expression of PVRL2). Dose levels for Opdivo and BMS-986207 combinations have already been determined through prior testing by Bristol-Myers Squibb, allowing for dose escalation of COM701 with fixed doses of Opdivo and BMS‑986207.

About COM701

COM701 is a humanized antibody that binds with high affinity to PVRIG, a novel immune checkpoint discovered computationally by Compugen, blocking the interaction with its ligand, PVRL2. Blockade of PVRIG by COM701 has demonstrated potent, reproducible enhancement of T cell activation, consistent with the desired mechanism of action of activating T cells in the tumor microenvironment to generate anti-tumor immune responses. PVRIG and TIGIT, also discovered by Compugen’s computational discovery platform in 2009, constitute parallel immune checkpoint pathways that counteract DNAM, a costimulatory molecule on T cells and NK cells. As such, preclinical data suggest that the inhibition of PVRIG together with TIGIT and/or PD-1 has the potential to further enhance anti-tumor immune response and improve patient outcomes in a broad variety of tumor types.

COM701 is being evaluated as a monotherapy and in combination with Opdivo (nivolumab), Bristol-Myers Squibb’s PD-1 inhibitor in a Phase 1 open-label clinical trial in patients with advanced solid tumors. Primary end points of the trial are safety and tolerability; secondary endpoints include preliminary anti-tumor activity, pharmacokinetics and pharmacodynamics in patients with selected tumor types. Data from the monotherapy dose escalation study (n=13) presented at SITC (Free SITC Whitepaper) 2019 showed that COM701 is well-tolerated and demonstrated preliminary signs of anti-tumor activity in heavily pretreated patient population Additional information is available at www.clinicaltrials.gov (NCT03667716).

Conference Call and Webcast Information

Compugen management will hold a conference call today, February 20, 2020, at 8:30 AM ET. To access the conference call by telephone, please dial 1-888-407-2553 from the United States, or +972-3-918-0610 internationally. The call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.