On August 5, 2021 NGM Biopharmaceuticals, Inc. (NGM) (Nasdaq: NGM), a biotechnology company focused on discovering and developing transformative therapeutics for patients, reported financial results for the period ending June 30, 2021 (Press release, NGM Biopharmaceuticals, AUG 5, 2021, View Source [SID1234585883]).

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"With six disclosed pipeline programs, five of which are in the clinic, and additional undisclosed preclinical and research programs, we continue to make meaningful progress towards achieving our mission to translate complex powerful biology with urgency and rigor to deliver life-changing medicines for patients," said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM Bio. "In our sizable oncology portfolio alone, we continue to advance multiple programs that we believe have the potential to benefit patients with a variety of solid tumors. We were pleased to dose our first patient with NGM707 in a Phase 1/2 study this quarter and look forward to presenting interim results from our Phase 1a/1b dose-finding study of NGM120 at ESMO (Free ESMO Whitepaper) later this quarter."

"We also continued to make important strides in ophthalmology, with the recent completion of enrollment in our Phase 2 CATALINA study exactly one year after initiation. Our strong balance sheet positions us well to continue progressing our ambitious clinical and research efforts."

Key Second Quarter and Recent Highlights

Retinal diseases

Completed enrollment in the Phase 2 CATALINA study of NGM621 in patients with geographic atrophy. NGM completed enrollment in the Phase 2 CATALINA study, a multi-center, randomized, double-masked, sham-controlled clinical trial to evaluate the safety and efficacy of intravitreal, or IVT, injections of NGM621 every four weeks or every eight weeks in 320 patients with geographic atrophy in one or both eyes secondary to age-related macular degeneration. The primary efficacy endpoint is the rate of change in geographic atrophy lesion area, as measured by fundus autofluorescence, or FAF, imaging, over 52 weeks of treatment. NGM anticipates reporting topline data from the CATALINA study in the second half of 2022. Upon completion of a proof-of-concept study in humans, Merck has a one-time option to license NGM621 and its related molecules as well as the additional one-time option to license NGM621 and its related molecules together with all other ophthalmology compounds included within the scope of our ongoing collaboration with Merck.
Cancer

Continued enrollment in a Phase 2 placebo-controlled component of the ongoing Phase 1/2 PINNACLES study testing NGM120 as a first-line treatment in combination with gemcitabine and Abraxane (paclitaxel protein bound) in patients with metastatic pancreatic cancer. In March 2021, NGM initiated a multi-center, randomized, single-blind (sponsor unblinded), placebo-controlled component of NGM120 in combination with gemcitabine and Abraxane as a first line treatment in patients with metastatic pancreatic cancer as part of the ongoing Phase 1/2 trial. This Phase 2 component of the Phase 1/2 study is designed to enroll approximately 60 patients and will assess the efficacy, safety and tolerability of NGM120 or placebo in combination with gemcitabine and Abraxane against both cancer and cancer-related cachexia endpoints. The Phase 1a/1b dose-finding portion of the study is still ongoing, and NGM expects to report interim results from that portion of the study at the European Society for Medical Oncology in the third quarter of 2021.
Initiated the Phase 1 portion of a Phase 1/2 Study of NGM707 for the treatment of advanced solid tumors. The Phase 1 portion (n≅60) of the study includes a monotherapy dose escalation arm (Part 1a) and a dose-finding arm in combination with KEYTRUDA (pembrolizumab) (Part 1b). The Phase 2 portion (n≅120) of the study will employ a basket design that will include expansion cohorts of patients treated with NGM707 monotherapy (Part 2a) or NGM707 in combination with KEYTRUDA (Part 2b).
Liver and metabolic diseases

Reported topline data from the Phase 2b ALPINE 2/3 study of aldafermin in patients with NASH and liver fibrosis stage 2 or 3, or F2 or F3, in May 2021. The 24-week study assessed the efficacy, safety and tolerability of 0.3 mg, 1 mg and 3 mg doses of aldafermin compared to placebo. The primary objective of the ALPINE 2/3 study was to evaluate a dose response on liver fibrosis improvement by ≥ 1 stage with no worsening of steatohepatitis at week 24. The study did not meet its primary endpoint evaluating a dose response at 24 weeks on liver fibrosis improvement by >1 stage with no worsening of NASH (p=0.55), analyzed using a dose response-driven statistical analysis plan (Multiple Comparison Procedure Modeling, or MCP-Mod). The study achieved statistical significance versus placebo on certain secondary endpoints, including NASH resolution (at the 3 mg dose) and multiple non-invasive measures of NASH, including liver fat content reduction by MRI-PDFF, ALT, AST and Pro-C3 (at the 1 mg and 3 mg doses). Aldafermin was generally well tolerated with an overall safety profile similar to placebo. As previously disclosed, NGM plans not to pursue Phase 3 clinical development of aldafermin in F2/F3 NASH.
Continued enrollment in Phase 2b ALPINE 4 study of aldafermin in patients with compensated NASH cirrhosis (liver fibrosis stage 4, or F4). The 48-week study is designed to enroll approximately 150 patients and will assess the efficacy, safety and tolerability of 0.3 mg, 1 mg and 3 mg doses of aldafermin compared to placebo. The primary objective of the ALPINE 4 study is to evaluate a dose response at 48 weeks on liver fibrosis improvement by ≥ 1 stage with no worsening of steatohepatitis.
Merck continued enrollment in its Phase 2b study of MK-3655 in patients with NASH and F2 or F3 liver fibrosis. In November 2020, Merck initiated a global Phase 2b multicenter study of MK-3655 for the treatment of patients with F2 or F3 NASH. The 52-week randomized, double-blind study is designed to enroll approximately 320 patients and will assess the efficacy, safety and tolerability of 50 mg, 100 mg and 300 mg once monthly doses of MK-3655 compared to placebo. The primary objective of the Phase 2b study is NASH resolution without worsening of fibrosis after 52 weeks. Merck licensed MK-3655 following NGM’s completion of a proof-of-concept study. NGM retains an option, at the initiation of the first Phase 3 clinical trial for MK-3655, to either receive milestone and royalty payments or to co-fund development and participate in a global cost and revenue sharing arrangement of up to 50% for MK-3655.
Corporate

Announced appointment of Roger M. Perlmutter to Board of Directors. On June 8, 2021, NGM announced that the stockholders of the company elected Roger M. Perlmutter, M.D., Ph.D. to the company’s board of directors. Dr. Perlmutter brings decades of expertise and renowned leadership in drug discovery and development with global healthcare companies including Merck and Amgen. Dr. Perlmutter is currently Chairman, President and Chief Executive Officer at Eikon Therapeutics, Inc.
Amended collaboration with Merck. In June 2021, NGM and Merck announced that they will continue their research, discovery and development collaboration with a narrower scope, focused primarily on retinal and cardiovascular and metabolic (CVM) targets of interest to Merck. Merck will continue to advance MK-3655, which is currently in a global Phase 2b clinical trial in patients with F2 or F3 NASH. Merck retains its option to license NGM621 and its related molecules, which is currently in the NGM-led Phase 2 CATALINA clinical study in patients with geographic atrophy. Merck will provide approximately $120 million in research and development, or R&D, funding to NGM through March 2024, plus additional potential license option payments. NGM gained worldwide rights to its disclosed oncology portfolio, including NGM120, NGM707 and NGM438, as well as all undisclosed preclinical and research assets falling outside of the amended collaboration’s narrower scope.
Second Quarter 2021 Financial Results

NGM reported a net loss of $36.7 million for the quarter ended June 30, 2021, compared to a net loss of $25.6 million for the same period in 2020.
Related party revenue from our collaboration with Merck was $16.8 million for the quarter ended June 30, 2021, compared to $19.8 million for the same period in 2020. Related party revenue decreased $3.0 million in the quarter ended June 30, 2021 as compared to the prior year due to the derecognition of a $4.6 million contract asset that was associated with our previous collaboration agreement with Merck.
R&D expenses were $43.6 million for the quarter ended June 30, 2021, compared to $38.5 million for the same period in 2020. R&D expenses increased $5.1 million in the quarter as compared to the prior year, primarily due to increases in external expenses driven by our ongoing clinical studies of NGM621 and NGM120 and preclinical studies of NGM438, and increases in personnel-related and internal and unallocated R&D expenses. These increases were partially offset by decreases in expenses for our manufacturing activities and our clinical trials of aldafermin and in external expenses related to our other development programs.
General and administrative expenses were $9.8 million for the quarter ended June 30, 2021, compared to $6.8 million for the same period in 2020. The $3.0 million increase in general and administrative expenses in 2021 was primarily attributable to increases in personnel-related expenses driven by increased headcount, as well as external expenses to support our operations.
Cash, cash equivalents and short-term marketable securities were $390.6 million as of June 30, 2021, compared to $295.2 million as of December 31, 2020.

On August 5, 2021 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that financial results for the second quarter ended June 30, 2021 (Press release, Puma Biotechnology, AUG 5, 2021, View Source [SID1234585882]). Unless otherwise stated, all comparisons are for the second quarter of 2021 compared to the second quarter of 2020.

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Product revenue, net consists entirely of sales revenue from NERLYNX, Puma’s first commercial product. Net NERLYNX revenue in the second quarter of 2021 was $48.9 million, compared to $48.8 million in the second quarter of 2020. Net NERLYNX revenue in the first six months of 2021 was $94.7 million, compared to $97.4 million in the first six months of 2020.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $5.1 million, or $0.13 per share, for the second quarter of 2021, compared to net income of $3.4 million, or $0.09 per basic share and $0.08 per diluted share, for the second quarter of 2020. Net income for the first six months of 2021 was $11.3 million, or $0.28 per share, compared to a net loss of $13.5 million, or $0.34 per share, for the first six months of 2020.

Non-GAAP adjusted net income was $13.1 million, or $0.32 per share, for the second quarter of 2021, compared to non-GAAP adjusted net income of $14.0 million, or $0.36 per basic share and $0.35 per diluted share, for the second quarter of 2020. Non-GAAP adjusted net income for the first six months of 2021 was $35.4 million, or $0.88 per basic share and $0.87 per diluted share, compared to non-GAAP adjusted net income of $6.0 million, or $0.15 per share, for the first six months of 2020. Non-GAAP adjusted net income excludes stock-based compensation expense. For a reconciliation of GAAP net income (loss) to non-GAAP adjusted net income and GAAP net income (loss) per share to non-GAAP adjusted net income per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the second quarter of 2021 was $0.1 million, compared to net cash provided by operating activities of $16.2 million in the second quarter of 2020. Net cash provided by operating activities for the first six months of 2021 was $15.6 million, compared to net cash provided by operating activities of $4.7 million for the first six months of 2020. At June 30, 2021, Puma had cash, cash equivalents and marketable securities of $108.9 million, compared to $93.4 million at December 31, 2020.

"We were pleased to achieve sequential growth in NERLYNX sales in the second quarter of 2021. Importantly, this marks the first quarter that we have shown sequential unit growth since 2019," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "We also recently received an important label expansion, which adds dose escalation of NERLYNX for HER2-positive early stage and metastatic breast cancer patients. Utilization of this recommended dosage schedule has shown improved overall tolerability of NERLYNX, thereby enabling patients and physicians to optimize treatment and reduce therapy-related tolerability issues. We look forward to increasing awareness of this within the breast cancer community."

Mr. Auerbach added, "We anticipate the following key milestones over the next 12 months: (i) reporting top line data from the randomized cohort of the Phase II SUMMIT trial of neratinib in hormone receptor positive breast cancer that has a HER2 mutation (Q4 2021); (ii) conducting a pre-NDA meeting with the FDA to discuss accelerated approval of neratinib in HER2-mutated hormone receptor positive breast cancer

(Q4 2021); (iii) reporting data from the Phase II TBCRC-022 trial of the combination of Kadcyla plus neratinib in patients with HER2-positive breast cancer with brain metastases who have previously been treated with Kadcyla (H2-2021/H1 2022); (iv) reporting Phase II data from the SUMMIT trial of neratinib in non-small cell lung cancer patients with EGFR exon 18 mutations (H1-2022); (v) conducting a meeting with the FDA to discuss the potential for an accelerated approval pathway for neratinib in non-small cell lung cancer patients with EGFR exon 18 mutations who have previously been treated with an EGFR tyrosine kinase inhibitor (2022); (vi) reporting Phase II data from the SUMMIT trial of neratinib in cervical cancer patients with HER2 mutations (H1-2022); and (vii) receiving regulatory decisions for the extended adjuvant HER2-positive early stage breast cancer indication in additional countries (2021)."

Revenue

Total revenue consists of product revenue, net from sales of NERLYNX, license revenue and royalty revenue. For the second quarter of 2021, total revenue was $53.4 million, of which $48.9 million was net product revenue, $0.2 million was license revenue received from Puma’s sub-licensees and $4.3 million was royalty revenue. This compares to total revenue of $70.6 million in the second quarter of 2020, of which $48.8 million was net product revenue, $20.7 million was license revenue received from Puma’s sub-licensees and $1.1 million was royalty revenue. For the first six months of 2021, total revenue was $151.6 million, of which $94.7 million was net product revenue, $50.3 million was license revenue received from Puma’s sub-licensees, which included a $50 million upfront payment for providing development, manufacturing and commercial rights to NERLYNX in Greater China to Pierre Fabre, and $6.6 million was royalty revenue. This compares to total revenue for the first six months of 2020 of $121.8 million, of which $97.4 million was net product revenue, $22.7 million was license revenue received from Puma’s sub-licensees, and $1.7 million was royalty revenue.

Operating Costs and Expenses

Total operating costs and expenses were $70.0 million for the second quarter of 2021, compared to $63.5 million for the second quarter of 2020. Operating costs and expenses for the first six months of 2021 were $148.1 million, compared to $128.9 million for the first six months of 2020.

Cost of Sales

Cost of sales was $12.0 million for the second quarter of 2021, compared to $9.4 million for the second quarter of 2020. Cost of sales was $41.5 million for the first six months of 2021, of which $20.0 million was for a termination fee paid to a former sub-licensee for the return of commercial rights to NERLYNX in Greater China, compared to cost of sales of $18.5 million for the first six months of 2020.

Selling, General and Administrative Expenses

Selling, general and administrative (SG&A) expenses were $39.4 million for the second quarter of 2021, compared to $29.4 million for the second quarter of 2020. SG&A expenses for the first six months of 2021 were $67.7 million, compared to $60.3 million for the first six months of 2020.

The $7.4 million year-over-year increase for the first six months resulted primarily from an increase in stock-based compensation of approximately $10.9 million, partially offset by decreases in payroll and related costs of approximately $0.8 million, professional fees and expenses of approximately $0.6 million, travel and meetings costs of approximately $0.9 million, and other expenses of approximately $1.2 million.

The $10.9 million increase in stock-based compensation expense consisted of a $13.6 million incremental expense resulting from a modification to the term of Mr. Auerbach’s warrant and an increase of $2.6 million from new grants, partially offset by decreases of approximately $4.2 million for stock awards that have fully vested and $1.1 million from stock awards forfeited.

Research and Development Expenses

Research and development (R&D) expenses were $18.6 million for the second quarter of 2021, compared to $24.7 million for the second quarter of 2020. R&D expenses for the first six months of 2021 were $38.9 million, compared to $50.1 million for the first six months of 2020.

The $11.2 million year-over-year decrease for the first six months resulted primarily from decreases in stock-based compensation expense of approximately $6.3 million, clinical trial expenses of approximately $2.8 million, internal R&D expenses of approximately $1.6 million and consultant and contractors’ costs of approximately $0.5 million.

Total Other Income (Expenses)

Total other income was $11.5 million for the second quarter of 2021 compared to $3.7 million of total other expenses for the second quarter of 2020. Total other income was $7.9 million for the first six months of 2021 compared to total other expenses of $6.4 million for the first six months of 2020. The $14.3 million year-over-year increase for the first six months resulted primarily from a net reduction in accrued legal verdict expense of approximately $14.9 million, partially offset by a decrease in interest income of approximately $0.4 million and other immaterial fluctuations.

Conference Call

Puma Biotechnology will host a conference call to report its second quarter 2021 financial results and provide an update on the Company’s business and outlook at 1:30 p.m. PDT/4:30 p.m. EDT on Thursday, August 5, 2021. The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international). Please dial in at least 10 minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available shortly after completion of the call and will be archived on Puma’s website for 90 days.

On August 5, 2021 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the second quarter of 2021 (Press release, Y-mAbs Therapeutics, AUG 5, 2021, View Source [SID1234585881]).

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"We are very pleased with our second quarter 2021 results. The commercialization of DANYELZA (naxitamab-gqgk) is progressing throughout the U.S. as we continue to see new sites gaining experience with DANYELZA. In addition, the DANYELZA BLA submission has recently been accepted by the National Medical Products Administration of China ("NMPA"), and we continue to advance our deep pipeline. We ended the quarter with a cash balance of $233.6 million, so we believe we are well positioned to elevate our business to new levels," stated Thomas Gad, founder, Chairman and President.

Dr. Claus Moller, Chief Executive Officer, continued, "We submitted the European Marketing Authorization Application to the European Medicines Agency (the "EMA") for omburtamab in April. In parallel, after our Type B meeting with the FDA in June, we believe we now have a clearer path towards the resubmission of the omburtamab BLA to the FDA. Pending a positive Type B meeting with the FDA in the third quarter, we hope to initiate rolling resubmission of the omburtamab BLA by the end of the year."

Recent Corporate Developments

After the close of the first quarter, on July 6, 2021, Y-mAbs announced that SciClone Pharmaceuticals had submitted the Biologics License Application to the National Medical Products Administration ("NMPA") of China for DANYELZA for the treatment of patients with relapsed/refractory high-risk neuroblastoma.

On June 25, 2021, Y-mAbs announced that 177Lu-omburtamab-DTPA for the treatment of medulloblastoma had received a positive opinion on Orphan Medicinal Product Designation by EMA.

On June 23, 2021, Y-mAbs announced updated timelines for resubmission of the BLA for omburtamab for neuroblastoma, with an anticipated initiation of a rolling BLA submission by the end of 2021.

On May 19, 2021, Y-mAbs announced an exclusive distribution agreement with Adium Pharma S.A. for DANYELZA and omburtamab in Latin America.

On April 27, 2021, Y-mAbs announced the submission to the EMA of a European Marketing Authorization Application for omburtamab for the treatment of pediatric patients with central nervous system/leptomeningeal metastasis from neuroblastoma.

On April 12, 2021, Y-mAbs announced data from GPA33-SADA, which in a xenograft model of colorectal cancer showed radioactivity uptake with a tumor to blood ratio of 122 measured 24 hours after injection. We expect to file an IND for GPA33-SADA next year.
Financial Results

Y-mAbs reported net loss of $22.9 million, or ($0.53) per basic and diluted share, for the quarter ended June 30, 2021, compared to a net loss of $40.4 million, or ($1.01) per basic and diluted share, reported for the quarter ended June 30, 2020. The decreased net loss was caused by the DANYELZA revenues in 2021 and the reduced R&D expenses for the quarter ended June 30, 2021 compared to June 30, 2020.

For the six months ended June 30, 2021, Y-mAbs reported a net income of $10.5 million, or $0.25 per basic share and $0.23 per diluted share, compared to the net loss of $66.6 million, or ($1.67) per basic and diluted share, reported for the six months ended June 30, 2020. The net income was primarily caused by the sale of a priority review voucher and the DANYELZA revenues in the first six months of 2021.

Revenues

Y-mAbs reported net revenues of $11.0 million for the quarter ended June 30, 2021, consisting of $9.0 million of DANYELZA revenues and $2.0 million of licensing revenue.

Revenues were $16.3 million for the six months ended June 30, 2021 and consisted of $14.3 million from the sales of DANYELZA and $2.0 million of licensing revenue.

No revenues were reported for the quarter ended and six months ended June 30, 2020.

Operating Expenses

Research and Development
Research and development expenses were $19.8 million for the three months ended June 30, 2021, compared to $30.1 million for the three months ended June 30, 2020, a decrease of $10.3 million. The decrease in research and development expenses was primarily due to the following:

$13.3 million decrease in milestone payments and license acquisition costs driven by the $13.3 million SADA agreement entered into in 2020 which included an upfront payment of $2.0 million, $3.3 million in equity issuances to Memorial Sloan Kettering ("MSK") and Massachusetts Institute of Technology ("MIT"), $7.4 million for the issuance of shares to two non-employees, and $0.6 million for milestone payments which were deemed probable for the period ending June 30, 2020.This decrease was partially offset by a $3.0 million increase in personnel costs.
Research and development expenses were $41.4 million for the six months ended June 30, 2021, compared to $48.7 million for the six months ended June 30, 2020, a decrease of $7.3 million. The decrease in research and development expenses was primarily due to:

$13.3 million decrease in milestone payments and license acquisition costs driven by the $13.3 million SADA agreement which included an upfront payment of $2.0 million, $3.3 million in equity issuances to MSK and MIT, $7.4 million for the issuance of shares to two non-employees, and $0.6 million for milestone payments which were probable for the period ending June 30, 2020; and
$3.5 million decrease in outsourced manufacturing expenses.
These decreases were partially offset by increases of:

$5.5 million in personnel costs;
$1.3 million in outsourced manufacturing;
$0.5 million in clinical trials; and
$1.0 million in premises expenses.
Selling, General, and Administration

Selling, general, and administrative expenses were $13.5 million for the three months ended June 30, 2021, compared to $10.4 million for the three months ended June 30, 2020, an increase of $3.1 million. The increase in selling general and administrative expenses primarily reflects a $3.1 million increase in personnel costs due to the continued hiring of our commercialization team.

Selling, general, and administrative expenses were $25.4 million for the six months ended June 30, 2021, compared to $18.5 million for the six months ended June 30, 2020, an increase of $6.9 million. The increase in selling general and administrative expenses primarily reflects a $6.4 million increase in personnel costs due to the continued hiring of our commercialization team.
Cash and Cash Equivalents

The Company had $233.6 million in cash and cash equivalents as of June 30, 2021, compared to $114.6 million as of December 31, 2020. The increase of $119.0 million was primarily attributable to the following:

The completion of the sale of our DANYELZA priority review voucher in January 2021. Y-mAbs netted $62.0 million after paying 40% of the net proceeds from the sale to MSK pursuant to the terms of the license agreement with MSK, and
$107.7 million in net proceeds raised in our public offering in February 2021.
These increases were partially offset by the net cash used in operational activities of $50.6 million for the six months ended June 2021.

Webcast and Conference Call

The Company will host a conference call on Friday, August 6, 2021 at 9 a.m. Eastern Time. To participate in the call, please dial 855-327-6838 (domestic) or 604-235-2082 (international) and reference the conference ID 10015973. A webcast will be available at: View Source

On August 5, 2021 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the second quarter of 2021 and provided an update on progress toward achieving key corporate objectives, as well as commercial, clinical and pipeline development milestones (Press release, Exelixis, AUG 5, 2021, View Source [SID1234585880]).

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"In the second quarter of 2021, Exelixis delivered a 59 percent year over year increase in CABOMETYX net product revenue growth and record total revenues, based on the broad adoption of the CABOMETYX and OPDIVO combination regimen as a preferred treatment in first-line renal cell carcinoma"

"In the second quarter of 2021, Exelixis delivered a 59 percent year over year increase in CABOMETYX net product revenue growth and record total revenues, based on the broad adoption of the CABOMETYX and OPDIVO combination regimen as a preferred treatment in first-line renal cell carcinoma," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "Following this strong commercial performance, we are raising our net product and total revenue guidance for 2021. In parallel, we also made significant progress across our development activities, with the filing of a supplemental New Drug Application for cabozantinib in differentiated thyroid cancer based on the COSMIC-311 study, and announcements of top-line results from both the COSMIC-312 trial in first-line advanced hepatocellular carcinoma and the expanded Cohort 6 of the COSMIC-021 trial in patients with advanced prostate cancer. In addition, on the pipeline front, we broadened the development program of XL092 with a new clinical collaboration with Bristol Myers Squibb and initiated the Phase 1 trial for XB002, our first antibody-drug conjugate. I’d like to thank the entire Exelixis team for a very strong first half of 2021 and look forward to providing further updates on our progress in the second half of the year."

Second Quarter 2021 Financial Results

Total revenues for the quarter ended June 30, 2021 were $385.2 million, compared to $259.5 million for the comparable period in 2020.

Total revenues for the quarter ended June 30, 2021 included net product revenues of $284.2 million, compared to $178.7 million for the comparable period in 2020. The increase in net product revenues was primarily related to an increase in sales volume that was driven by strong uptake for the combination therapy of CABOMETYX (cabozantinib) and OPDIVO (nivolumab) following approval by the U.S. Food and Drug Administration (FDA) in January 2021.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $100.9 million for the quarter ended June 30, 2021, compared to $80.7 million for the comparable period in 2020. The increase in collaboration revenues was primarily related to increases in development cost reimbursements earned, and higher royalty revenues for the sales of cabozantinib outside of the U.S. generated by Exelixis’ collaboration partners, Ipsen Pharma SAS (Ipsen) and Takeda Pharmaceutical Company Limited (Takeda), which was partially offset by a decrease in the recognition of milestone-related revenues.

Research and development expenses for the quarter ended June 30, 2021 were $148.8 million, compared to $114.9 million for the comparable period in 2020. The increase in research and development expenses was primarily related to increases in license and other collaboration costs, personnel expenses and stock-based compensation expense, which was partially offset by decreases in clinical trial costs.

Selling, general and administrative expenses for the quarter ended June 30, 2021 were $98.5 million, compared to $59.8 million for the comparable period in 2020. The increase in selling, general and administrative expenses was primarily related to increases in personnel expenses, marketing costs, corporate giving and stock-based compensation expense.

Provision for income taxes for the quarter ended June 30, 2021 was $28.8 million, compared to $13.9 million for the comparable period in 2020, primarily due to an increase in pre-tax income.

GAAP net income for the quarter ended June 30, 2021 was $96.1 million, or $0.31 per share, basic and $0.30 per share, diluted, compared to GAAP net income of $66.8 million, or $0.22 per share, basic and $0.21 per share, diluted, for the comparable period in 2020.

Non-GAAP net income for the quarter ended June 30, 2021 was $117.9 million, or $0.38 per share, basic and $0.37 per share, diluted, compared to non-GAAP net income of $79.4 million, or $0.26 per share, basic and $0.25 per share, diluted, for the comparable period in 2020. Non-GAAP net income excludes stock-based compensation, adjusted for the related income tax effect.

Cash, cash equivalents, restricted cash equivalents and investments were $1.7 billion at June 30, 2021, compared to $1.5 billion at December 31, 2020.

Non-GAAP Financial Measures

To supplement Exelixis’ financial results presented in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Exelixis presents non-GAAP net income (and the related per share measures), which excludes from GAAP net income (and the related per share measures) stock-based compensation expense, adjusted for the related income tax effect for all periods presented.

Exelixis believes that the presentation of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors. In particular, Exelixis believes that these non-GAAP financial measures, when considered together with its financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare Exelixis’ results from period to period, and to identify operating trends in Exelixis’ business. Exelixis has excluded stock-based compensation expense, adjusted for the related income tax effect, because it is a non-cash item that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. Exelixis also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate its business and to make operating decisions.

These non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. Exelixis encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP financial information and the reconciliation between these presentations, to more fully understand Exelixis’ business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

2021 Financial Guidance

Exelixis is providing the following updated financial guidance for fiscal year 2021:

Total revenues (1)

$1,300 million – $1,400 million

Net product revenues (1)

$1,050 million – $1,150 million

Cost of goods sold

Approximately 5% – 6% of net product revenue

Research and development expenses (1)(2)

$650 million – $700 million

Selling, general and administrative expenses (1)(3)

$375 million – $425 million

Effective tax rate

20% – 22%

Cash and investments (4)(5)

$1.7 billion – $1.8 billion

(1)

Guidance updated on August 5, 2021 from previously provided guidance on May 6, 2021.

(2)

Includes $45 million of non-cash stock-based compensation expense.

(3)

Includes $60 million of non-cash stock-based compensation expense.

(4)

This cash and investments guidance does not include any potential new business development activity.

(5)

Cash and investments is composed of cash, cash equivalents, restricted cash equivalents and investments.

Cabozantinib Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $284.2 million during the second quarter of 2021, up 25 percent over the prior quarter, with net product revenues of $275.6 million from CABOMETYX and $8.6 million from COMETRIQ (cabozantinib). Exelixis earned $24.9 million in royalty revenues during the quarter ended June 30, 2021, pursuant to collaboration agreements with our partners, Ipsen and Takeda.

Announcement of Phase 1b Results from Cohort 6 of COSMIC-021 Trial in Patients with Metastatic Castration-Resistant Prostate Cancer (CRPC). In May, Exelixis announced results from the metastatic CRPC cohort 6 of COSMIC-021, the phase 1b trial of cabozantinib in combination with atezolizumab in patients with locally advanced or metastatic solid tumors. The analysis included 132 patients, 101 of whom had high-risk disease, defined as measurable visceral and/or extra-pelvic lymph node metastases. Based on these promising results, Exelixis intends to discuss the data with the FDA to determine next steps toward a potential regulatory submission for the combination regimen for patients with high-risk metastatic CRPC and plans to present detailed results at a medical meeting in the second half of 2021.

Cabozantinib Presentations at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO 2021) Demonstrate Consistent Results in Renal Cell Carcinoma (RCC). In June, cabozantinib was the subject of multiple RCC data presentations at ASCO (Free ASCO Whitepaper) 2021, which included: a post-hoc exploratory analysis demonstrating consistent efficacy benefits across subgroups in the phase 3 CheckMate -9ER pivotal trial with CABOMETYX in combination with OPDIVO compared with sunitinib as a first-line treatment for advanced RCC; results from another post-hoc analysis of the CheckMate -9ER trial demonstrating that CABOMETYX in combination with OPDIVO resulted in a statistically significant and clinically meaningful increase in quality-adjusted survival (Q-TWiST) compared with sunitinib; and positive phase 2 results from an investigator-sponsored trial evaluating cabozantinib in combination with nivolumab in patients with advanced or metastatic non-clear cell RCC.

Detailed Results from Phase 3 COSMIC-311 Pivotal Trial of Cabozantinib in Patients with Previously Treated Radioactive Iodine (RAI)-Refractory Differentiated Thyroid Cancer (DTC) Also Presented at ASCO (Free ASCO Whitepaper) 2021. In June, Exelixis and Ipsen announced detailed results from the phase 3 COSMIC-311 pivotal trial of cabozantinib in patients with previously treated RAI-refractory DTC, presented at ASCO (Free ASCO Whitepaper) 2021. Results from the trial, which met the primary endpoint of demonstrating significant improvement in progression-free survival (PFS) assessed by a blinded independent radiology committee, were recently published in The Lancet Oncology and served as the basis for the supplemental New Drug Application (sNDA) submitted to the FDA in June.

Announcement of Top-line Results of Phase 3 COSMIC-312 Pivotal Trial in Patients with Previously Untreated Advanced Hepatocellular Carcinoma (HCC). In June, Exelixis and Ipsen announced that COSMIC-312, the ongoing phase 3 pivotal trial evaluating cabozantinib in combination with atezolizumab versus sorafenib in patients with previously untreated advanced HCC, met one of the primary endpoints, demonstrating significant improvement in PFS at the planned primary analysis. A prespecified interim analysis for the second primary endpoint of overall survival (OS), conducted at the same time as the primary analysis for PFS, showed a trend favoring the combination of cabozantinib and atezolizumab but did not reach statistical significance. The trial will continue as planned to the final analysis of OS; results are anticipated in early 2022. Exelixis plans to present the trial results at a future medical meeting and intends to discuss the results with the FDA to determine next steps toward a potential regulatory submission for the combination regimen for patients with previously untreated advanced HCC.

FDA Accepts sNDA for CABOMETYX for Patients with Previously Treated RAI-Refractory DTC. In August, Exelixis announced that the FDA accepted the company’s sNDA for CABOMETYX as a treatment for patients 12 years and older with DTC who have progressed following prior therapy and are RAI-refractory (if RAI is appropriate). The FDA granted Priority Review designation and assigned a Prescription Drug User Fee Act goal date, or target action date, of December 4, 2021.

Pipeline Highlights

FDA Accepts Investigational New Drug Application (IND) and Phase 1 Trial Initiated for Tissue Factor-Targeting Antibody-Drug Conjugate (ADC) XB002 in Patients with Advanced Solid Tumors. In April, Exelixis announced that the FDA accepted its IND to evaluate the safety, tolerability, pharmacokinetics and preliminary antitumor activity of XB002 in patients with advanced solid tumors, and in June, a phase 1 trial was initiated. As a next-generation tissue factor-targeting ADC, XB002 has the potential for an improved therapeutic index and may provide a favorable safety profile compared with earlier-generation tissue factor-targeting ADCs. Exelixis in-licensed XB002 from Iconic Therapeutics, Inc. in December 2020 under the companies’ May 2019 collaboration agreement.

Exelixis and Bristol Myers Squibb (BMS) Enter Clinical Trial Collaboration and Supply Agreement to Evaluate XL092 in Combination with Immuno-oncology Therapies in Advanced Solid Tumors. In June, Exelixis announced a clinical trial collaboration and supply agreement with BMS for STELLAR-002, a new phase 1b trial evaluating XL092 in combination with immuno-oncology therapies in advanced solid tumors. The objective of the study is to evaluate the safety, tolerability and efficacy of XL092, Exelixis’ novel next-generation tyrosine kinase inhibitor, in combination with: nivolumab; nivolumab and ipilimumab (YERVOY); and nivolumab and bempegaldesleukin, an investigational CD122-preferential IL-2–pathway agonist developed by Nektar Therapeutics (Nektar). The trial is anticipated to begin enrolling patients in the second half of 2021. Nektar will supply bempegaldesleukin to BMS through their existing global development and commercialization collaboration, which is evaluating nivolumab in combination with bempegaldesleukin.

Corporate Updates

Exelixis Expands its Biotherapeutics Portfolio with Acquisition of Anti-Müllerian Hormone Receptor 2 (AMHR2) Program from GamaMabs Pharma SA (GamaMabs). In May, Exelixis entered into an asset purchase agreement with GamaMabs under which Exelixis will, upon the closing of the asset purchase and subject to certain conditions, acquire all rights, title and interest in GamaMabs’ antibody program directed at AMHR2, a novel oncology target with relevance in multiple forms of cancer. Exelixis believes applying its ADC capabilities to GamaMabs’ panel of antibodies against AMHR2 could yield potential additions to the company’s biotherapeutics portfolio.

Exelixis Files Lawsuit to Enforce Its Intellectual Property Rights for CABOMETYX against Abbreviated New Drug Application (ANDA) Filers. In June, Exelixis filed a patent lawsuit against Teva Pharmaceuticals Development, Inc. and Teva Pharmaceuticals USA, Inc. (individually and collectively referred to as Teva), along with Teva Pharmaceutical Industries Limited, following receipt of two Paragraph IV certification notice letters from Teva informing Exelixis that it had filed an ANDA with the FDA requesting approval to market a generic version of CABOMETYX tablets. Teva’s notice letters included a Paragraph IV certification with respect to three of Exelixis’ Orange Book-listed patents: U.S. Patent Nos. 9,724,342 (formulations), 10,034,873 (methods of treatment) and 10,039,757 (methods of treatment), which expire in 2033, 2031 and 2031, respectively. Teva’s notice letter did not provide a Paragraph IV certification against any additional CABOMETYX patents. Exelixis is seeking, among other relief, an order that the effective date of any FDA approval of the ANDA would be a date no later than the expiration of all of U.S. Patent Nos. 9,724,342, 10,034,873 and 10,039,757, the latest of which expires on July 9, 2033, and equitable relief enjoining Teva and Teva Pharmaceutical Industries Limited from infringing these patents.

Basis of Presentation

Exelixis has adopted a 52- or 53-week fiscal year that generally ends on the Friday closest to December 31st. For convenience, references in this press release as of and for the fiscal periods ended July 2, 2021, January 1, 2021 and July 3, 2020 are indicated as being as of and for the periods ended June 30, 2021, December 31, 2020, and June 30, 2020, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the second quarter of 2021 and provide a general business update during a conference call beginning at 5:00 p.m. EDT / 2:00 p.m. PDT today, Thursday, August 5, 2021.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 7296685 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on August 7, 2021. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 7296685. A webcast replay will also be archived on www.exelixis.com for one year.

On August 5, 2021 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing potential new medicines for patients with sarcomas, pediatric cancers, and other hematologic and solid cancers, reported important corporate events and its financial results for the second quarter ended June 30, 2021.

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"The second quarter and recent weeks have continued a period of substantial development for Salarius, highlighted by significant progress in our clinical programs leading to multiple data readouts in 2021 and 2022. Importantly, we ended the quarter with $33.1 million in cash and cash equivalents which, based on our current operating plan, we believe is sufficient to support the achievement of these readouts," stated David Arthur, Chief Executive Officer of Salarius Pharmaceuticals.

Mr. Arthur continued, "We have added the Fox Chase Cancer Center as our ninth sarcoma clinical trial site and are now actively recruiting and treating patients with seclidemstat in clinical trials across five patient groups, three in high unmet need sarcomas and two in high unmet need hematologic cancers. In addition, the data unveiled during ASCO (Free ASCO Whitepaper) 2021 demonstrated exactly what we wanted to see, and our Ewing sarcoma program is now providing the opportunity to explore seclidemstat in combination with chemotherapy agents where synergistic drug activity has been demonstrated. This combination with a commonly used second- or third-line chemotherapy, offers the opportunity for introducing seclidemstat earlier in the treatment continuum while improving patient outcomes and achieving objective responses. In the near future, we plan to initiate additional clinical programs in cancer indications where LSD1 overexpression is known to be a factor. In summary, we are pleased with how Salarius is positioned for the second half of 2021 and beyond."

Recent Business and Corporate Highlights:

MD Anderson Cancer Center in Houston, TX activated and is enrolling patients in an investigator-initiated trial in hematologic cancers, a Phase 1/2 study to determine safety, tolerability, maximum tolerated dose and overall response rate of seclidemstat in combination with azacytidine as a treatment for Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML).
In roughly 1 in 3 patients, MDS or CMML can progress to a rapidly growing cancer of bone marrow cells called Acute Myeloid Leukemia (AML) which the American Cancer Association estimates had more than 19,000 new cases diagnosed in the U.S. in 2020.
Fox Chase Cancer Center in Philadelphia, Pennsylvania added as an active clinical trial site in the ongoing dose-expansion stage of the Phase 1/2 sarcoma trial.
Nine sites now actively recruiting for three patient groups across Ewing sarcoma and FET-rearranged sarcomas.
Initial patients enrolled across both Ewing sarcoma and FET-rearranged sarcoma cohorts.
Investor event showcased key opinion leaders (KOLs) in cancer research that we believe supports LSD1 inhibition and the potential of seclidemstat as a differentiated treatment for Ewing sarcoma, FET-rearranged sarcomas, and hematologic or blood cancers.
Clinical trial data presented at ASCO (Free ASCO Whitepaper) showed seclidemstat has a manageable safety profile, has favorable pharmacokinetics that support twice-daily oral dosing, and established recommended Phase 2 dose at 900 mg BID.
Evidence of preliminary anti-tumor activity in advanced, heavily pre-treated patient populations.
Data supports continued exploration of seclidemstat in both Ewing sarcoma and FET-rearranged sarcomas.
Financial Highlights:

Three-month period ended June 30, 2021, net loss per common share – basic and diluted – for continuing operations of $0.07, compared to $0.13 for the same period last year.
Total working capital of $36.8 million as of June 30, 2021
Mr. Arthur concluded, "Key opinion leaders in cancer research recently noted that seclidemstat specifically inhibits the scaffolding properties of LSD1. This capability could prove effective against a broad range of cancers, including solid tumors, potentially further differentiating seclidemstat from other LSD1 inhibitors. Our ultimate aim as a company is to maximize the potential of seclidemstat as a treatment for cancer and we look forward to expanding the scope of our research into additional high unmet need patient populations. We are looking forward to updating investors and patients throughout 2021 and beyond."

Three-Month Financial Results:
For the three-month period ended June 30, 2021, Salarius’ reported net loss was $3.1 million, or $0.07 per basic and diluted share, compared to a net loss of $1.8 million, or $0.13 per basic and diluted share for the same period in 2020. The loss before other income for the three-month period ended June 30, 2021, increased by $1.2 million compared to the loss before other income for the same time span last year, primarily due to lower grant revenue and overall higher operating expenses.

Net cash used for operating activities during the three-month period ended June 30, 2021 totaled $3.2 million, an increase of approximately $0.7 million compared to the same span last year due to higher overall spending during the second quarter 2021.

Conference Call Information:
Salarius Pharmaceuticals will host a conference call and live audio webcast on Thursday, August 5, 2021, at 10:00 a.m. ET, to discuss its corporate and financial results for the second quarter 2021. Interested participants and investors may access the conference call by dialing either:

An audio webcast will be accessible via the Investors Events and Presentations section of the Company’s website View Source An archive of the webcast will remain available for 90 days beginning at approximately 11:00 a.m. ET, on August 5, 2021.

(Press release, Salarius Pharmaceuticals, AUG 5, 2021, View Source [SID1234585879])