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EXELIXIS ANNOUNCES THIRD QUARTER 2020 FINANCIAL RESULTS AND PROVIDES CORPORATE UPDATE

On November 5, 2020 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the third quarter of 2020 and provided an update on progress toward fulfilling its key corporate objectives, as well as commercial and clinical development milestones (Press release, Exelixis, NOV 5, 2020, View Source [SID1234570072]).

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"In the third quarter of 2020, the Exelixis team built the foundation to accelerate revenue growth with CABOMETYX (cabozantinib) in 2021," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "Based on the positive results from the CheckMate -9ER phase 3 pivotal trial evaluating cabozantinib in combination with nivolumab in previously untreated patients with advanced renal cell carcinoma, we and Bristol Myers Squibb completed our respective regulatory filings for the combination in August. In September, the first detailed results from the trial were presented during a Presidential Symposium of the ESMO (Free ESMO Whitepaper) Virtual Congress 2020. And then just last month, we announced the FDA accepted the filings, granted Priority Review designation and assigned an action date of February 20th of next year. Exelixis is launch-ready and prepared to immediately support this important new combination regimen, pending FDA approval."

Dr. Morrissey continued: "As we continue working to maximize the clinical and commercial potential for CABOMETYX, we’re moving quickly in parallel to build a diversified pipeline behind it. In October, we presented the preclinical profile and initial clinical pharmacokinetic data for XL092, our next-generation oral tyrosine kinase inhibitor that builds on the experience and target profile of cabozantinib with improved characteristics, including a shorter pharmacokinetic half-life. Encouraged by the data we’ve seen to date, we expanded the phase 1 study to evaluate XL092 in combination with atezolizumab in multiple solid tumors, with enrollment now underway. The XL092 program is an important component of our growing pipeline, as well as an opportunity to drive growth into new and potentially larger indications with unmet medical need. We further strengthened our pipeline during the quarter through business development activities, with two additional collaboration and license agreements focused on the discovery and development of novel antibody-drug conjugates. Our continued efforts to expand the breadth and depth of our discovery pipeline beyond small molecules, along with a focused investment in the development of cabozantinib and XL092, have the potential to drive top-line growth significantly for Exelixis, and provide new treatment options for the patients we serve."

2020 Financial Results
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November 5, 2020
Third Quarter 2020 Financial Results

Total revenues for the quarter ended September 30, 2020 were $231.1 million, compared to $271.7 million for the comparable period in 2019.

Total revenues for the quarter ended September 30, 2020 included net product revenues of $168.6 million, compared to $191.8 million for the comparable period in 2019. The decrease in net product revenues was due to a decrease in sales volumes driven by decreases in prescriptions, which were in line with market trends, and lower customer inventory.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $62.5 million for the quarter ended September 30, 2020, compared to $79.9 million for the comparable period in 2019. The decrease in collaboration revenues was primarily related to a decrease in the recognition of milestone related revenues, which was partially offset by 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).

Research and development expenses for the quarter ended September 30, 2020 were $176.8 million, compared to $97.3 million for the comparable period in 2019. The increase in research and development expenses was primarily related to increases in clinical trial costs, license and other collaboration costs and personnel expenses. The increase in clinical trial costs was primarily due to costs associated with expanding clinical trial programs for cabozantinib, which includes CONTACT-02, COSMIC-313, COSMIC-312 and COSMIC-021. The increase in license and other collaboration costs was primarily due to an increase in upfront license fee payments from recent business development activities with two additional collaboration and license agreements focused on the discovery and development of novel antibody-drug conjugates (ADCs). The increase in personnel expenses was primarily due to an increase in stock-based compensation expense attributable to the performance-based restricted stock units (PSUs) granted in 2019 that became probable of achievement during the third quarter of 2020 and an increase in headcount to support Exelixis’ expanding discovery and development efforts.

Selling, general and administrative expenses for the quarter ended September 30, 2020 were $88.2 million, compared to $51.3 million for the comparable period in 2019. The increase in selling, general and administrative expenses was primarily related to increases in personnel expenses and marketing costs. The increase in personnel expenses was primarily due to an increase in stock-based compensation expense attributable to the PSUs granted in 2019 that became probable of achievement during the third quarter of 2020 and an increase in administrative headcount to support Exelixis’ commercial and research and development organizations.

Provision for (benefit from) income taxes for the quarter ended September 30, 2020 was $(6.0) million, compared to $25.2 million for the comparable period in 2019, primarily due to the change in pre-tax income (loss).

GAAP net income (loss) for the quarter ended September 30, 2020 was $(32.0) million, or $(0.10) per share, basic and diluted, compared to GAAP net income of $97.5 million, or $0.32 per share, basic and $0.31 per share, diluted, for the comparable period in 2019. The change in GAAP net income was primarily related to an increase in operating expenses and a decrease in total revenues.

Non-GAAP net income for the quarter ended September 30, 2020 was $11.2 million, or $0.04 per share, basic and diluted, compared to non-GAAP net income of $107.6 million, or $0.35 per share, basic and $0.34 per share, diluted, for the comparable period in 2019. Non-GAAP net income excludes stock-based compensation, adjusted for the related income tax effect.
Exelixis Third Quarter 2020 Financial Results
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November 5, 2020
Cash and investments were $1.5 billion at September 30, 2020, compared to $1.4 billion at December 31, 2019.

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 (loss) (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.

2020 Financial Guidance
Cabozantinib Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $168.6 million during the third quarter of 2020, with net product revenues of $159.6 million from CABOMETYX and $9.0 million from COMETRIQ (cabozantinib). Based upon cabozantinib-related revenues generated by Exelixis’ collaboration partner Ipsen in the third quarter of 2020, Exelixis earned $19.9 million in royalty revenues.
Exelixis Third Quarter 2020 Financial Results
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Initiation of CONTACT-03 Phase 3 Pivotal Trial of Cabozantinib in Combination with Atezolizumab in Previously Treated Metastatic Renal Cell Carcinoma (RCC). In July 2020, Exelixis announced the initiation of CONTACT-03, a global phase 3 pivotal trial of cabozantinib in combination with atezolizumab in patients with inoperable, locally advanced or metastatic RCC who progressed during or following treatment with an immune checkpoint inhibitor as the immediate preceding therapy. CONTACT-03 is part of a clinical trial collaboration between Exelixis and F. Hoffmann-La Roche Ltd. that includes two additional ongoing phase 3 pivotal trials – CONTACT-01 in patients with metastatic non-small cell lung cancer who have been previously treated with an immune checkpoint inhibitor and platinum-containing chemotherapy and CONTACT-02 in patients with metastatic castration-resistant prostate cancer (mCRPC) who have been previously treated with one novel hormonal therapy.

Completion of Patient Enrollment for EXAMINER Phase 4 Trial of Cabozantinib in Metastatic Medullary Thyroid Cancer. In July 2020, Exelixis announced the completion of patient enrollment in EXAMINER, the phase 4 trial evaluating the safety and efficacy of the 60 mg tablet formulation of cabozantinib compared with the 140 mg capsule formulation, which is marketed as COMETRIQ, for the treatment of patients with progressive, metastatic medullary thyroid cancer. EXAMINER is a post-marketing requirement from the U.S. Food and Drug Administration (FDA) and the European Commission. The trial was designed to enroll up to 250 patients, and top-line results from the trial are anticipated later this year.

Submission of Supplemental New Drug Application (sNDA) to and Acceptance by the FDA for Cabozantinib in Combination with Nivolumab for Advanced Renal Cell Carcinoma. In August 2020, Exelixis announced the submission of an sNDA to the FDA for cabozantinib in combination with Bristol-Myers Squibb Company’s (BMS) nivolumab for patients with advanced RCC. The sNDA submission was based on the positive results of the CheckMate -9ER phase 3 pivotal trial, which met its primary endpoint of significantly improving progression-free survival (PFS) and secondary endpoints of overall survival (OS) and objective response rate. In October 2020, Exelixis and BMS announced that the FDA had accepted Exelixis’ sNDA and BMS’ supplemental Biologics License Application (sBLA), granted Priority Review to both applications and assigned a Prescription Drug User Fee Act goal date, or target action date, of February 20, 2021.

Completion of Patient Enrollment for the COSMIC-312 Phase 3 Pivotal Trial in Previously Untreated Hepatocellular Carcinoma (HCC). In August 2020, Exelixis announced the completion of patient enrollment in COSMIC-312, a global phase 3 pivotal trial evaluating cabozantinib in combination with atezolizumab versus sorafenib as a treatment for patients with previously untreated advanced HCC, providing the patient population for the event-driven analyses of the study’s endpoints. Separately, patient enrollment remains open in China in order to enroll a sufficient number of patients to enable local registration, if supported by the clinical data. The co-primary endpoints of the trial are PFS and OS for the combination of cabozantinib and atezolizumab versus sorafenib. Based on current event rates, Exelixis anticipates announcing top-line results in the first half of 2021.

Presentation of Positive Results from CheckMate -9ER Phase 3 Pivotal Trial during Presidential Symposium I at the European Society for Medical Oncology Virtual Congress 2020 (ESMO 2020). In September 2020, Exelixis and BMS announced the first presentation of results from the CheckMate -9ER phase 3 pivotal trial as part of Presidential Symposium I at ESMO (Free ESMO Whitepaper) 2020, in which cabozantinib in combination with nivolumab demonstrated significant improvements across all efficacy endpoints, including OS, in previously untreated advanced RCC, with a favorable tolerability profile versus sunitinib.

Validation of Regulatory Filings for Cabozantinib plus Nivolumab in the European Union (EU). In September 2020, Exelixis, BMS and Ipsen announced that the European Medicines Agency (EMA) had validated BMS’ and Ipsen’s type II variation applications for cabozantinib plus nivolumab, which confirmed the submissions were complete and began the EMA’s centralized review process. Like BMS’ and Exelixis’ own regulatory filings in the
Exelixis Third Quarter 2020 Financial Results
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November 5, 2020
United States, BMS’ and Ipsen’s EU filings were based on positive data from the CheckMate -9ER phase 3 pivotal trial.

Presentation of Positive Results from Two RCC Cohorts of the COSMIC-021 Trial of Cabozantinib in Combination with Atezolizumab at ESMO (Free ESMO Whitepaper) 2020. In September 2020, Exelixis presented positive phase 1b clinical trial results for the combination of cabozantinib and atezolizumab in patients with locally advanced or metastatic solid tumors at ESMO (Free ESMO Whitepaper) 2020. Data from the clear cell RCC and non-clear cell RCC expansion cohorts of the COSMIC-021 trial were presented as part of the GU Proffered Paper Session and as part of a poster presentation, respectively, and showed that cabozantinib in combination with atezolizumab demonstrated promising preliminary efficacy and a favorable safety profile.

Announcement of Submission of Supplemental Application for Cabozantinib in Combination with Nivolumab in Japan for the Treatment of Unresectable, Advanced or Metastatic RCC. In October 2020, Exelixis announced that its collaboration partner Takeda, and Ono Pharmaceuticals Co., Ltd. (Ono), submitted a supplemental application to the Japanese Ministry of Health, Labour and Welfare for Manufacturing and Marketing Approval of cabozantinib in combination with nivolumab for the treatment of patients with unresectable, advanced or metastatic RCC. The application is also based on the results from the CheckMate -9ER study. Takeda has licensed the exclusive rights to cabozantinib from Exelixis for development and commercialization in Japan, while Ono jointly develops and commercializes nivolumab, in collaboration with BMS, in Japan, South Korea and Taiwan. Takeda previously received approval in March 2020 to market single-agent cabozantinib for the treatment of patients with curatively unresectable or metastatic RCC in Japan.

Pipeline Highlights

Presentations of Data for XL092 and AUR102 at the 32nd EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) Symposium on Molecular Targets and Cancer Therapeutics. In October 2020, Exelixis presented the preclinical profile and initial clinical pharmacokinetics (PK) for XL092 at the ENA Symposium. The poster discussion presentation included preclinical results demonstrating robust target and tumor growth inhibition, as well as increased efficacy for XL092 when combined with an immune checkpoint inhibitor. PK data from the ongoing phase 1 trial suggested a significantly shorter PK half-life for XL092 as compared to cabozantinib. Also at the ENA Symposium, Aurigene presented promising preclinical data for AUR102, its novel inhibitor of cyclin-dependent kinase 7, including potent anti-tumor activity in a large panel of cancer cell lines. Exelixis has an exclusive option for AUR102 under its July 2019 exclusive collaboration, option and license agreement with Aurigene. Exelixis’ option window extends up until the time of Investigational New Drug application (IND) acceptance; AUR102 could be the subject of an IND later this year.

Enrollment of First Patient in Phase 1 Trial Cohort Evaluating XL092 in Combination with Atezolizumab in Patients with Advanced Solid Tumors. In October 2020, Exelixis announced enrollment of the first patient into the dose-escalation cohort of the combination arm of the phase 1 trial evaluating the safety, tolerability, PK and preliminary anti-tumor activity of XL092 alone and in combination with atezolizumab in patients with advanced solid tumors. Initiated in February 2019, the dose-escalation evaluation of the XL092 monotherapy arm of the phase 1 trial is ongoing. Once the recommended doses of both single-agent XL092 and XL092 in combination with atezolizumab are established, the trial will begin to enroll expansion cohorts for patients with clear cell and non-clear cell RCC, hormone-receptor positive breast cancer and mCRPC.

Corporate Updates

FDA Approves TECENTRIQ (Atezolizumab) Plus COTELLIC (Cobimetinib) and ZELBORAF (Vemurafenib) for Previously Untreated BRAF V600 Mutation-Positive Advanced Melanoma. In July 2020, the FDA approved the sBLA submitted by Genentech, Inc. (a member of the Roche Group) (Genentech), for TECENTRIQ plus COTELLIC
Exelixis Third Quarter 2020 Financial Results
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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 6945239 to join by phone.

A telephone replay will be available until 8:00 p.m. EST on November 7, 2020. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 6945239. A webcast replay will also be archived on www.exelixis.com for one year.
Exelixis Third Quarter 2020 Financial Results
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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 6945239 to join by phone.

Guardant Health Reports Third Quarter 2020 Financial Results

On November 5, 2020 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary blood tests, vast data sets and advanced analytics, reported financial results for the quarter ended September 30, 2020 (Press release, Guardant Health, NOV 5, 2020, View Source [SID1234570071]).
Recent Highlights
•Revenue of $74.6 million for the third quarter of 2020, an increase of 23% over the corresponding period of 2019
◦Precision oncology revenue of $60.4 million, an increase of 16% over the corresponding period of 2019
◦Development services and other revenue of $14.2 million, an increase of 63% over the corresponding period of 2019
•Reported 16,950 tests to clinical customers and 3,071 tests to biopharmaceutical customers in the third quarter of 2020, representing an increase of 28% and a decrease of 42%, respectively, over the third quarter of 2019
•Received FDA-approval for Guardant360 CDx, becoming the first liquid biopsy test for comprehensive tumor mutation profiling across all solid cancers
•Launched enhanced version of Guardant360 including tumor mutational burden (TMB) and expanded homologous recombination deficiency (HRD) and fusion gene set
•Study published in Nature Medicine shows evidence of Guardant360 liquid biopsy accelerating clinical trial enrollment compared to tissue biopsy
"Our strong performance in the third quarter is a testament to our team’s dedication, as they stay focused on executing for our customers and on our pipeline programs," said Helmy Eltoukhy, PhD, co-founder and CEO. "While we expect to continue to see impacts related to the pandemic in the fourth quarter, we are confident that the fundamentals of our business are firmly intact. With the FDA approval of Guardant360 CDx behind us and as we look forward to 2021, I am more confident than ever in the opportunity that lies ahead for Guardant to transform patient care across the cancer care continuum."
Third Quarter 2020 Financial Results
Revenue was $74.6 million for the three months ended September 30, 2020, a 23% increase from $60.8 million for the three months ended September 30, 2019. Precision oncology revenue grew 16% driven predominantly by an increase in average selling price. There were 16,950 clinical tests and 3,071 biopharmaceutical tests performed during the third quarter of 2020. Development services and other revenue increased 63% primarily related to the timing of project related milestones for companion diagnostic development programs.
Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $53.4 million for the third quarter of 2020, an increase of $11.0 million from $42.3 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 72%, as compared to 70% for the corresponding prior year period.
Operating expenses were $127.6 million for the third quarter of 2020, as compared to $59.8 million for the corresponding prior year period, an increase of 113%.
Net loss attributable to Guardant Health, Inc. common stockholders was $77.7 million for the third quarter of 2020, as compared to $12.8 million for the corresponding prior year period. Net loss per share attributable to Guardant Health, Inc. common stockholders was $0.78 for the third quarter of 2020, as compared to $0.14 for the corresponding prior year period.
Non-GAAP adjusted EBITDA was $14.6 million loss for the third quarter of 2020, as compared to $9.0 million loss for the corresponding prior year period.
Cash, cash equivalents and marketable securities were $1.1 billion as of September 30, 2020.
2020 Guidance
Guardant Health is not providing 2020 financial guidance due to the continued uncertainties from the impact of COVID-19.
Webcast Information
Guardant Health will host a conference call to discuss the third quarter 2020 financial results after market close on Thursday, November 5, 2020 at 1:30 PM Pacific Time / 4:30 PM Eastern Time. A webcast of the conference call can be accessed at View Source The webcast will be archived and available for replay for at least 90 days after the event.
Non-GAAP Measure
We believe that the exclusion of certain income and expenses in calculating non-GAAP Adjusted EBITDA can provide a useful measure for investors when comparing our period-to-period core operating results, and when comparing those same results to that published by our peers. To derive Adjusted EBITDA, we remove from GAAP results the impact of income (expenses) attributable to material non-cash items, specifically stock-based compensation and fair value remeasurements due to the subjectivity, management judgment, and market fluctuations involved around these amounts. We exclude certain other items because we believe that these income (expenses) do not reflect expected future operating expenses. Additionally, certain items are inconsistent in amounts and frequency, making it difficult to perform a meaningful evaluation of our current or past operating performance.
This non-GAAP financial measure is not intended to be considered in isolation from, as substitute for, or as superior to, the corresponding financial measure prepared in accordance with GAAP. There are limitations inherent in non-GAAP financial measures because they exclude charges and credits that are required to be included in a GAAP presentation, and do not present the full measure of our recorded costs against its revenue. In addition, our definition of the non-GAAP financial measures may differ from non-GAAP measures used by other companies.
Definition of Non-GAAP Adjusted EBITDA
"Adjusted EBITDA" is defined as net loss attributable to Guardant Health, Inc. common stockholders before: (i) interest income,(ii) interest expense (iii) provision for (benefit from) income taxes, (iv) depreciation and amortization expense, (v) other (income) expense, net, (vi) stock-based compensation expense, (vii) adjustments relating to non-controlling interest and contingent consideration and, if applicable in a reporting period, and (viii) acquisition-related expenses, and other non-recurring items.

Puma Biotechnology Reports Third Quarter 2020 Financial Results

On November 5, 2020 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the third quarter ended September 30, 2020 (Press release, Puma Biotechnology, NOV 5, 2020, View Source [SID1234570070]). Unless otherwise stated, all comparisons are for the third quarter of 2020 compared to the third quarter of 2019.

Product revenue, net consists entirely of sales revenue from NERLYNX, Puma’s first commercial product. Net NERLYNX revenue in the third quarter of 2020 was $49.3 million, compared to $53.5 million in the third quarter of 2019. Net NERLYNX revenue in the first nine months of 2020 was $146.7 million, compared to $152.9 million in the first nine months of 2019.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $31.5 million, or $0.79 per share, for the third quarter of 2020, compared to a net loss of $16.9 million, or $0.44 per share, for the third quarter of 2019. Net loss for the first nine months of 2020 was $45.0 million, or $1.14 per share, compared to a net loss of $64.4 million, or $1.67 per share, for the first nine months of 2019.

Non-GAAP adjusted net loss was $23.9 million, or $0.60 per share, for the third quarter of 2020, compared to non-GAAP adjusted net loss of $4.7 million, or $0.12 per share, for the third quarter of 2019. Non-GAAP adjusted net loss for the first nine months of 2020 was $17.9 million, or $0.45 per share, compared to non-GAAP adjusted net loss of $18.6 million, or $0.48 per share, for the first nine months of 2019. Non-GAAP adjusted net loss excludes stock-based compensation expense. For reconciliations of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash provided by operating activities for the third quarter of 2020 was $1.7 million, compared to net cash used in operating activities of $7.3 million in the third quarter of 2019. Net cash provided by operating activities for the first nine months of 2020 was $6.4 million, compared to net cash provided by operating activities of $20.8 million in the first nine months of 2019. At September 30, 2020, Puma had cash, cash equivalents and marketable securities of $109.0 million, compared to cash, cash equivalents and marketable securities of $111.6 million at December 31, 2019.

"During the third quarter Puma continued to be negatively impacted by the challenges presented by COVID-19. Given these challenges, we were pleased that we were able to achieve revenues that were within the previously stated third quarter guidance range of net NERLYNX revenues," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "Although we anticipate that COVID-19 may continue to impact our revenues going forward, we remain focused on and committed to providing support to patients battling breast cancer. Our team has continued to work remotely and continues to respond to any COVID-related challenges, and we are pleased with the accomplishments made by the team during this pandemic. During the third quarter, we announced the publication of updated interim results of the Phase II CONTROL trial in Annals of Oncology as well as the publication of overall survival results from the Phase III ExteNET Trial in patients with HER2-positive, hormone receptor-positive, early stage breast cancer in Clinical Breast Cancer, which we believe further reinforces the benefits of neratinib."

Mr. Auerbach added, "We anticipate the following key milestones over the next 12 months: (i) reporting Phase II data from the SUMMIT trial of neratinib in hormone receptor positive breast cancer patients with HER2 mutations in the fourth quarter of 2020; (ii) reporting additional data from the Phase II CONTROL trial in the fourth quarter of 2020; (iii) reporting Phase II data from the SUMMIT trial of neratinib in bile duct cancer patients with HER2 mutations in the first quarter of 2021; (iv) reporting Phase II data from the SUMMIT trial of neratinib in non-small cell lung cancer patients with EGFR exon 18 mutations in the first half of 2021; (v) conducting a pre-NDA meeting with the FDA to discuss accelerated approval of neratinib in HER2 mutated hormone receptor positive breast cancer and HER2 mutated cervical cancer in the first half of 2021; (vi) 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 in the first half of 2021; (vii) 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 been previously treated with an EGFR tyrosine kinase inhibitor in 2021; and (viii) receiving regulatory decisions for an extended adjuvant HER2-positive early stage breast cancer indication in additional countries in 2021."

Revenue

Total revenue consists of product revenue, net from sales of NERLYNX, Puma’s first commercial product, license revenue and royalty revenue. For the third quarter of 2020, total revenue was $50.8 million, of which $49.3 million was net product revenue and $1.5 million was royalty revenue from Puma’s sub-licensees. This compares to total revenue of $56.4 million in the third quarter of 2019, of which $53.5 million was net NERLYNX revenue, $2.8 million was license revenue, and $0.1 million was royalty revenue from Puma’s sub-licensees. For the first nine months of 2020, total revenue was $172.6 million, of which $146.7 million was net product revenue, $22.7 million was license revenue, and $3.2 million was royalty revenue from Puma’s sub-licensees. This compares to total revenue of $209.3 million for the first nine months of 2019, of which $152.9 million was net product revenue, $56.2 million was license revenue, and $0.2 million was royalty revenue from Puma’s sub-licensees.

Operating Costs and Expenses

Total operating costs and expenses were $62.9 million for the third quarter of 2020, compared to $70.8 million for the third quarter of 2019. Operating costs and expenses in the first nine months of 2020 were $191.8 million, compared to $239.7 million in the first nine months of 2019.

Cost of Sales

Cost of sales was $10.0 million for the third quarter of 2020 and $28.4 million for the first nine months of 2020, compared to $9.4 million for the third quarter of 2019 and $26.7 million for the first nine months of 2019.

Selling, General and Administrative Expenses

Selling, general and administrative (SG&A) expenses were $29.6 million for the third quarter of 2020, compared to $31.4 million for the third quarter of 2019. SG&A expenses for the first nine months of 2020 were $89.9 million, compared to $110.4 million for the first nine months of 2019. The $20.5 million year-over-year decrease resulted primarily from decreases in stock-based compensation expense of approximately $9.4 million, professional fees and expenses of approximately $7.0 million and travel and meetings expense of approximately $4.4 million.

Research and Development Expenses

Research and development (R&D) expenses were $23.3 million for the third quarter of 2020, compared to $30.0 million for the third quarter of 2019. R&D expenses for the first nine months of 2020 were $73.5 million, compared to $102.6 million for the first nine months of 2019. The $29.1 million year-over-year decrease resulted primarily from decreases in clinical trial expense of approximately $16.5 million, stock-based compensation expense of approximately $9.3 million and consultant and contractor expenses of approximately $3.4 million.

Total Other Income (Expenses)

Total other expenses were $19.4 million for the third quarter of 2020 and $25.8 million for the first nine months of 2020, compared to total other expenses of $2.5 million for the third quarter of 2019 and $34.0 million for the first nine months of 2019. The $16.9 million increase in total other expenses for the three months ended September 30, 2020, compared to the three months ended September 30, 2019, was largely attributable to a legal verdict expense of $15.9 million. During the third and fourth quarters of 2020, we obtained additional data, previously unavailable, from the claims report relating to our class action lawsuit, which asserted damages in the amount of $50.5 million. While we intend to challenge some of these claims, we have increased our estimate of the legal accrual to $24.8 million. This resulted in the additional $15.7 million legal expense during the third quarter of 2020. The $8.2 million year-over-year decrease in total other expenses resulted primarily from decreases in interest expense of approximately $1.4 million, legal verdict expense of approximately $0.4 million and debt extinguishment loss of approximately $8.1 million and an increase in other income of approximately $0.2 million, partially offset by a decrease in interest income of approximately $1.9 million.

Conference Call

Puma Biotechnology will host a conference call to report its third quarter 2020 financial results and provide an update on the Company’s business and outlook at 1:30 p.m. PST/4:30 p.m. EST on Thursday, Nov. 5, 2020. 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.

BioMarin Announces Third Quarter 2020 Total Revenues of $477 Million

On November 5, 2020 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the third quarter ended September 30, 2020 (Press release, BioMarin, NOV 5, 2020, View Source [SID1234570069]).
Net Product Revenues for the third quarter of 2020 increased to $460.7 million, compared to $450.9 million in the third quarter of 2019. The increase in Net Product Revenues was primarily attributed to the following:
•Palynziq Net Product Revenues increased by $22.0 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy;
•Aldurazyme Net Product Revenues increased by $18.1 million due to higher sales volume to Genzyme;
•Naglazyme and Vimizim Net Product Revenues decreased by an aggregate of $33.7 million primarily due to timing of orders placed from Latin America as well as the impact of missed infusions resulting from the COVID-19 pandemic.
The increase in GAAP Net Income for the third quarter of 2020, compared to the same period in 2019 was primarily due to the following:
•an increase in the benefit from income taxes of $800.8 million primarily due the completion of an intra-entity transfer of certain intellectual property (IP) rights to an Irish subsidiary where the Company’s Ex-US regional headquarters are located and has significant manufacturing and commercial operations, to better align ownership of IP rights with how the business operates resulting in a tax benefit of $835.1 million based on the fair value of the transferred IP rights; and
•decreased research and development (R&D) expense primarily resulting from decreased clinical manufacturing costs for BMN 307 and lower clinical activity spend for valoctocogene roxaparvovec gene therapy programs; partially offset by
•an increase in Cost of Sales of $91.8 million primarily attributed to the $81.2 million reserve of valoctocogene roxaparvovec pre-launch inventory due to delays in anticipated regulatory approvals; and
•higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec.
Non-GAAP Income for the third quarter of 2020 increased to $98.7 million, compared to Non-GAAP Income of $78.1 million for the same period in 2019. The increase in Non-GAAP Income for the quarter, compared to the same period in 2019, was attributed to decreased R&D expense and higher gross profit, excluding the $81.2 million pre-launch inventory charge, partially offset by higher SG&A expense.
As of September 30, 2020, BioMarin had cash, cash equivalents and investments totaling approximately $1.8 billion, which includes net proceeds of $535.8 million from the Company’s May 2020 convertible debt offering, as compared to $1.2 billion on December 31, 2019. On October 15, 2020, the Company’s 1.50% senior subordinate convertible notes matured and were settled in cash for approximately $375.0 million.
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Commenting on third quarter 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "While the impact of COVID-19 continued through the third quarter, BioMarin employees remained focused on our mission to serve patients and ensure the steady supply of our critically-important medicines. In these continued unpredictable times, the essential nature of our products to the people who rely on them remains constant."
Mr. Bienaimé continued, "In the third quarter we received unexpected news on the status of our application with valoctocogene roxaparvovec for hemophilia A from health authorities, which resulted in a delay in potential approval timelines. However, we remain confident in our valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A. We continue to work with the health authorities to align on next steps toward approval. Our 134-subject Phase 3 study with valoctocogene roxaparvovec will complete one-year of observation in all subjects later this month, and we anticipate sharing top-line results comprising 1 to 2 years of follow-up from that study, in the first quarter of 2021. We also plan to submit the complete one-year Phase 3 data to the EMA in the second quarter of 2021."
"Vosoritide for the treatment of achondroplasia is advancing as planned with applications under review in both the U.S. and Europe with potential regulatory approvals anticipated in 2021. The significant unmet medical need for children with achondroplasia has enabled BioMarin to build a multi-pronged dossier of clinical studies. It includes the highly statistically significant placebo-controlled Phase 3 results, long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, and the ongoing study of newborns through 5 years, which is nearing enrollment completion. The positive results from our vosoritide clinical programs bolster our confidence in the potential for this drug to be the first pharmacological treatment to address the underlying cause of achondroplasia. Interest in our clinical studies with vosoritide has been extremely robust, demonstrating that many families are keen to seek early treatment for their children."
Mr. Bienaimé concluded, "Despite the impact from COVID-19 and the timing set-back on the potential approval of valoctocogene roxaparvovec, we remain confident in our business. BioMarin fundamentals are strong, driven by our global base business of essential medicines and cash position, but our people and pursuit and development of innovative therapies will always be our most important assets."

Caladrius Biosciences Provides Corporate Update and Reports 2020 Third Quarter Financial Results

On November 5, 2020 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease, reported financial results for the three and nine months ended September 30, 2020 (Press release, Caladrius Biosciences, NOV 5, 2020, View Source [SID1234570068]).

"Amid the continuing global impact of COVID-19, our team continues to rise to the occasion, addressing a multitude of challenges tied to the pandemic," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "To date in 2020, we have extended our cash runway through the end of 2021, while continuing to deliver on a number of key initiatives in support of our clinical programs including the initiation of our study of CLBS119 for the treatment of COVID-19 induced lung damage and finalizing preparations for the start to our newly-named Phase 2b FREEDOM Study of CLBS16 in coronary microvascular dysfunction."

"We are excited about what lies ahead in 2020 and expect to build on this momentum as we continue to advance our clinical development pipeline and strive to achieve a number of important development milestones throughout the balance of the year," concluded Dr. Mazzo.

Product Development and Financing Highlights

HONEDRA (formerly CLBS12) development in Japan continues to progress

The Company’s open-label, registration-eligible study in Japan of HONEDRA (formerly CLBS12), continues to progress toward enrollment completion, although enrollment has been slowed by the impact of the COVID-19 pandemic in Japan. HONEDRA is a SAKIGAKE-designated product candidate for the treatment of critical limb ischemia ("CLI"); a disease with limited treatment options – most of which have minimal impact – and a higher combined incidence and mortality rate than all cancers but lung cancer. As previously reported, the Buerger’s Disease (an "orphan-sized" type of CLI) cohort has concluded with 4 out of 7 (~60%) patients achieving a positive outcome, an outstandingly positive result for these patients who normally see continued progression leading to amputation. The Company remains encouraged by the patient pre-screening pipeline that has been identified for the arteriosclerosis obliterans ("ASO") cohort, which is the primary arm of the study, and anticipates trial enrollment to conclude in the first quarter of 2021, leading to top line data for the full study in late 2021 or early 2022.

CLBS14 remains poised to enter a single confirmatory phase 3 clinical trial

The Company’s Phase 3 protocol for its RMAT-designated product candidate, CLBS14, for the treatment of no-option refractory angina ("NORDA") remains ready to initiate pending sufficient funding to run the program to completion. Based on an abundance of very strong data from previous Phase 1, 2, and 3 studies, Caladrius remains confident in the potential for clinical success once the program is executed.

CLBS16 to be studied in Phase 2b trial for the treatment of coronary microvascular dysfunction

Caladrius recently completed and announced the results of its ESCaPE-CMD Phase 2a study of CLBS16 for the treatment of coronary microvascular dysfunction ("CMD"), a disease that continues to be underdiagnosed and potentially afflicts millions annually – a vast majority of whom are female – with no current treatment options. Data from the Phase 2a trial showed a positive therapeutic effect with a statistically significant improvement in angina frequency, coronary flow reserve, Canadian Cardiovascular Society Angina Class and Seattle Questionnaire score, as well as an acceptable safety profile. The Company is committed to raising awareness of this growing women’s health crisis and plans to initiate a rigorous Phase 2b FREEDOM trial, with the first patient expected to be enrolled by the

end of 2020. The double-blind, randomized, placebo-controlled Phase 2b trial will evaluate the efficacy and safety of delivering autologous CD34+ cells (CLBS16) in subjects with CMD and without obstructive coronary artery disease.
CLBS119 for the repair of COVID-19-induced lung damage in COVID-19 survivors

Caladrius is committed to helping patients and communities combat the public health crisis of COVID-19 by leveraging its proprietary CD34+ cell technology to potentially repair COVID-19-induced lung damage. COVID-19 appears to damage the vasculature of the lungs and Caladrius believes the repair of that vasculature will prove necessary for patients to achieve a full recovery. Experience to date indicates that a large portion of COVID-19 survivors who required ventilatory support will suffer long-term, debilitating lung damage. While many developmental therapies responding to the COVID-19 pandemic are appropriately targeting the SARS-CoV-2 virus itself, or the manifestations of the acute phase of the illness, Caladrius is unaware of a therapy that has demonstrated the ability to repair COVID-19-induced lung damage. With consistent clinical and pre-clinical evidence that CD34+ cells can repair multiple organs, including models of severe lung inflammation, the Company sought and received FDA authorization for its investigational new drug ("IND") application for the study of CLBS119, a CD34+ cell therapy for the repair of COVID-19-induced lung damage. The planned 10-12-patient open-label, proof-of-concept clinical trial, is designed to evaluate the safety and efficacy of a single administration of CLBS119 for the treatment and repair of COVID-19-induced lung damage in adults. The study was recently initiated and patients who are experiencing hypoxia due to prior infection with SARS-CoV-2 and who require supplemental oxygen are now being screened for participation at NYU Langone Health.

Secures new capital to support cash runway through the end of 2021

As previously disclosed, in July 2020, Caladrius raised $2.0 million in a private placement priced at the market under Nasdaq rules. Caladrius has now successfully raised approximately $30 million in net proceeds year-to-date in 2020.

Third Quarter 2020 Financial Highlights

Research and development expenses were approximately $3.0 million for both the three months ended September 30, 2020 and the three months ended September 30, 2019. Research and development in both periods focused on the advancement of our ischemic repair platform. More specifically, R&D expense comprised (i) costs associated with investigational new drug application and planning for commencement of a pilot study of CLBS119, (ii) execution expenses for our ongoing registration-eligible study for CLBS12 in critical limb ischemia in Japan, and (iii) expenses for both the completion of our ESCaPE-CMD study of CLBS16 in coronary microvascular dysfunction and planning for the follow on Phase 2b study.

General and administrative expenses were approximately $2.3 million for the three months ended September 30, 2020, compared to $2.1 million for the three months ended September 30, 2019, representing an increase of 12%.

Overall, net losses were $5.3 million for the three months ended September 30, 2020, compared to $4.9 million for the three months ended September 30, 2019.

Balance Sheet Highlights

As of September 30, 2020, Caladrius had cash, cash equivalents and marketable securities of $40.3 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations through 2021.

Conference Call

Caladrius will hold a conference call on Thursday, November 5, 2020, at 4:30 p.m. ET to discuss the financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. The conference call will also be webcast live under the Investors section on the Company’s website at www.caladrius.com.