Alector to Present at the BofA Securities 2021 Virtual Health Care Conference

On May 6, 2021 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported that Shehnaaz Suliman, M.D., MBA, M.Phil., president and chief operating officer of Alector, will participate in a fireside chat at the BofA Securities 2021 Virtual Health Care Conference on Thursday, May 13, 2021, at 11:45 a.m. ET (Press release, Alector, MAY 6, 2021, View Source [SID1234579348]).

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A live webcast of the fireside chat will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 30 days following the event.

NantHealth Reports 2021 First Quarter Financial Results

On May 6, 2021 NantHealth, Inc. (NASDAQ-GS: NH), a provider of enterprise solutions that help businesses transform complex data into actionable insights, reported financial results for its first quarter ended March 31, 2021 (Press release, NantHealth, MAY 6, 2021, View Source [SID1234579347]).

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"Last month, we successfully completed a $137.5 million financing transaction and will use a portion of the proceeds to retire our existing convertible notes," said Ron Louks, Chief Operating Officer, NantHealth. "We intend to use the balance of the proceeds to, among other things, support our growth initiatives, which include further developing our existing SaaS offerings, expanding our recently acquired OpenNMS business and investing in our data services and cloud capabilities.

"Turning to our 2021 first-quarter financial results, as expected revenues declined largely due to the accounting for amortization of services related to expired or renewed contracts at the end of the 2020 fourth quarter. While we remain committed to managing our overhead costs, we have continued the ramp up of our investment in research and development to improve and expand the innovative solutions we bring to our customers.

"Looking ahead, we see growing opportunities to expand and diversify our business within and beyond healthcare. Earlier this week, we launched Eviti Connect for autoimmune diseases, extending our proven payer solution beyond oncology, and announced plans to further develop the Eviti platform to allow us to expand into additional disease states, over time. In addition, we are expanding OpenNMS offerings to include a new cloud-based platform to supplement our already robust on-premise platform, which will both broaden the solutions we offer to our healthcare customers and attract new customers across a variety of industries."

Software and Services Highlights:

Clinical Decision Support (Eviti):
In May, launched Eviti Connect for autoimmune diseases, bringing the Company’s payer solution for oncology to a disease type that affects more than 50 million in the U.S. alone. The Company developed a new, flexible platform that will enable it to expand into disease areas beyond oncology and autoimmune
Launched Eviti Connect 8.4, which included:
New functionality enables drug customization per payer or line of business providing more flexibility to meet diverse policies within the payer organization
New payer communication capability that reduces delays in treatment plan reviews
In May, Eviti Connect won the MedTech Breakthrough award for "Best MedTech Overall Software," recognized for the platform’s innovative ability to break through digital health and technology markets
Payer Engagement (NaviNet and Population Health Management):
Continued customer renewals, securing three multi-year agreements in Q1 with over $700K of annual contract value
AllPayer, the Company’s direct-to-provider solution, had its seventh consecutive quarter of growth
Enhanced NaviNet essential workflows to support automation and promote greater usability
Open Authorizations now allows health plans to configure rules defining which services do not require prior authorization. This increases transparency for providers and saves time by eliminating unnecessary requests
New Referral Submission API enables providers to automate management of referral requests through NaviNet, making it easier to guide patients to optimal quality and cost referral sites
Announced Population Health Management, a new product offering that integrates multiple data sources into one platform, providing a more complete view of the patient. This enables payers and providers to engage proactively with patients, increasing the quality of care while reducing costs
Network Monitoring and Management (OpenNMS):
Deployed a solution for a global energy customer that scales the monitoring and analysis of network communications by aggregating the data streams (netflow) to greatly improve scalability and usability of the solution
Initiated a Zero-touch Appliance service pilot with a large national retailer
Business and Financial Highlights

For the 2021 first quarter:

Total net revenue was $16.2 million compared with $18.2 million in Q1 of 2020. Within total revenue, total software-related revenue was $16.2 million compared with $18.1 million in prior-year first quarter.
Gross profit was $9.1 million, or 56% of total net revenue, compared with $11.0 million, or 60% of total net revenue, for the prior-year period.
Selling, general and administrative (SG&A) expenses increased to $12.5 million from $12.4 million in the 2020 first quarter.
Research and development (R&D) expenses increased to $5.0 million from $3.6 million.
Net loss from continuing operations attributable to NantHealth, net of tax, was $15.4 million, or $0.14 per share, compared with $8.9 million, or $0.08 per share, in the 2020 first quarter.
Non-GAAP net loss from continuing operations attributable to NantHealth was $9.6 million, or $0.09 per share, compared with $6.1 million, or $0.06 per share, for the first quarter of 2020.
At March 31, 2021, cash and cash equivalents totaled $10.8 million.
Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the first quarter ended March 31, 2021. The conference call will be available to interested parties by dialing 844-309-3709 from the U.S. or Canada, or 281-962-4864 from international locations, passcode 8364209. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

Use of Non-GAAP Financial Measures

This news release contains references to Non-GAAP financial measures, including adjusted net loss and adjusted net loss per share, which are financial measures that are not prepared in conformity with United States generally accepted accounting principles (U.S. GAAP). The Company’s management believes that the presentation of Non-GAAP financial measures provides useful supplementary information regarding operational performance, because it enhances an investor’s overall understanding of the financial results for the Company’s core business. Additionally, it provides a basis for the comparison of the financial results for the Company’s core business between current, past and future periods. Other companies may define these measures in different ways. Non-GAAP financial measures should be considered only as a supplement to, and not as a substitute for or as a superior measure to, financial measures prepared in accordance with U.S. GAAP. Non-GAAP per share numbers are calculated based on one class of common stock and do not incorporate the effects, if any, of using the two-class method.

BeiGene Reports First Quarter 2021 Financial Results

On May 6, 2021 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, reported recent business highlights, anticipated upcoming milestones, and financial results for the first quarter of 2021 (Press release, BeiGene, MAY 6, 2021, View Source [SID1234579346]).

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"We continue to make excellent progress across the board in advancing our key strategic objectives, including enhancing our commercial scale in China, working to broaden access to our medicines around the world through new regulatory filings, advancing our pipeline, including potentially first-in-class compounds and wholly owned combinations, and expanding our capabilities in biologics manufacturing. We are encouraged by the performance of our commercial portfolio since the implementation on March 1 of three innovative oncology products in the National Reimbursement Drug List in China," said John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene. "In addition, we closed our license and collaboration agreement with Novartis, receiving a $650 million up-front payment, and recently announced positive interim results from our global Phase 3 head-to-head ALPINE trial in chronic lymphocytic leukemia, which we believe further demonstrates the differentiated and potentially best-in-class profile for BRUKINSA. We are confident in our strong position to deliver on our mission of providing high quality medicines to billions of more people around the world who need them."

Recent Business Highlights and Upcoming Milestones

Commercial Operations

Generated $106.1 million in global product revenue in the three months ended March 31, 2021, representing a 104% increase from $52.1 million in the comparable period of the prior year. Product sales in the first quarter grew over the prior year as well as sequentially compared to the prior quarter due to continued progress in our product launches, including significantly increased patient demand following the inclusion of tislelizumab, BRUKINSA, and XGEVA in the NRDL, effective March 1, 2021, which more than offset the net effect of price reductions as a result of NRDL inclusion;
First quarter product revenue grew sequentially despite a negative adjustment of $24.2 million as a result of the normal process in China of compensating distributors for products previously sold at the pre-NRDL price during the quarter that remained in the distribution channel, due to the first inclusion of tislelizumab, BRUKINSA, and XGEVA in the NRDL. The majority of the compensation related to tislelizumab;
Inclusion in the NRDL led to significant increases in the number of formal hospital listings for tislelizumab, BRUKINSA, and XGEVA to approximately 4x, 8x, and 6x their respective levels prior to NRDL inclusion; and
Sales of BRUKINSA in the United States continued steady growth despite the continued impact from COVID-19, including the vaccine rollout, on patient treatment plans.
Development Programs

BRUKINSA (zanubrutinib), a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects, approved in the United States, China, Canada, and other international markets in selected indications and under development for additional approvals globally.

Announced positive results from a planned interim analysis of the ongoing Phase 3 ALPINE trial (NCT03734016) comparing BRUKINSA against ibrutinib in adults with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL);
Received approval and launched BRUKINSA in Canada for the treatment of adult patients with Waldenström’s macroglobulinemia (WM), based on the Phase 3 ASPEN trial comparing BRUKINSA and ibrutinib; and
Continued to advance BRUKINSA in new markets. BRUKINSA is now commercially available in Israel for patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. To-date, more than 30 marketing authorization applications in multiple indications have been submitted outside of the United States and China, covering the European Union (EU) and more than 20 other countries.
Expected Milestones for BRUKINSA

Announce topline results from the Phase 3 SEQUOIA trial (NCT03336333) comparing BRUKINSA with bendamustine plus rituximab in patients with treatment-naïve CLL/SLL as early as 2021;
Present interim results from the Phase 3 ALPINE trial (NCT03734016) at a major medical conference in 2021 and announce additional results in 2022;
Continue to expand BRUKINSA’s registration program globally in new geographies and indications, including potential approvals in 2021 for certain patients with MCL in the Middle East, South America, Canada, Australia, and Russia; and with WM in the United States, EU, China, and Australia; and
Complete enrollment in the pivotal global Phase 2 ROSEWOOD trial (NCT03332017) comparing BRUKINSA and obinutuzumab versus obinutuzumab alone in patients with R/R follicular lymphoma (FL) in 2021.
Tislelizumab, a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages; approved in China in selected indications and under development for additional approvals globally.

Entered into and closed a collaboration and license agreement with Novartis Pharma AG granting Novartis rights to develop, manufacture and commercialize tislelizumab in North America, Europe, and Japan, and received a $650 million up-front payment;
Announced the acceptance of a supplemental Biologics License Application (sBLA) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for treatment in the second- or third-line setting of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have progressed on prior platinum-based chemotherapy;
Initiated patient enrollment in the global Phase 2 clinical trial (NCT04716634) of tislelizumab in combination with HUTCHMED (China) Ltd.’s fruquintinib in solid tumors;
Completed enrollment in the Phase 3 trial (NCT04005716) of tislelizumab with or without platinum chemotherapy plus etoposide in patients with untreated extensive-stage small cell lung cancer; and
Announced clinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 from the planned interim analysis of the Phase 3 RATIONALE 303 trial (NCT03358875) of tislelizumab compared to docetaxel as second- or third-line therapy for patients with locally advanced or metastatic NSCLC in an oral presentation.
Expected Milestones for Tislelizumab

Submit the first biologics license applications (BLA) outside of China in 2021 in collaboration with Novartis;
Submit sBLAs in China for MSI-H/dMMR solid tumors in the first half of 2021, and for second-line esophageal squamous cell carcinoma (ESCC) in mid-2021;
Receive approvals in first-line non-squamous NSCLC and second/third-line hepatocellular carcinoma (HCC) in China in 2021;
Present clinical data at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting, including posters on:
– the RATIONALE 302 trial (NCT03430843) of tislelizumab versus chemotherapy as a second-line treatment for advanced unresectable ESCC; and

– the Phase 2 clinical trial (NCT03736889) of tislelizumab as monotherapy in patients with previously treated, locally advanced unresectable or MSI-high/MRD solid tumors;

Announce topline results of the Phase 3 trial (NCT03924986) of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NPC) in 2021; and
Complete enrollment in the Phase 3 trial (NCT03957590) of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC in 2021.
Pamiparib, an investigational selective small molecule inhibitor of PARP1 and PARP2

Expected Milestones for Pamiparib

Receive approval in China for the treatment of patients with germline BRCA mutation-associated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy, in the first half of 2021;
Announce topline results from the Phase 3 trial (NCT03519230) of pamiparib as a maintenance treatment in patients with platinum-sensitive recurrent ovarian cancer (OC) in 2021 or the first half of 2022; and
Present clinical data at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting, including posters on:
– the Phase 2 trial (NCT03575065) in China of pamiparib in patients with locally advanced or metastatic HER2-negative breast cancer with germline BRCA mutation; and

– the Phase 2 clinical trial (NCT03427814) of pamiparib versus placebo as maintenance therapy in patients with inoperable locally advanced or metastatic gastric cancer that responded to platinum-based first-line chemotherapy.

Ociperlimab (BGB-A1217), an investigational TIGIT monoclonal antibody with competent Fc function

Initiated patient enrollment in the following trials:
– the Phase 2 AdvanTIG-202 trial (NCT04693234) of ociperlimab in combination with tislelizumab in patients with previously treated recurrent or metastatic cervical cancer; and

– the Phase 2 AdvanTIG-203 trial (NCT04732494) of ociperlimab in combination with tislelizumab versus tislelizumab in combination with placebo for the second-line treatment of patients with unresectable, locally advanced, recurrent or metastatic ESCC whose tumors have high PD-L1 expression.

Expected Milestones for Ociperlimab

Present clinical data at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting on the Phase 1 dose-escalation study (NCT04047862) of ociperlimab in combination with tislelizumab in patients with advanced solid tumors;
Initiate patient enrollment in the global Phase 3 AdvanTIG-302 trial (NCT04746924) of ociperlimab in combination with tislelizumab for the first-line treatment of patients with locally advanced, unresectable, or metastatic NSCLC whose tumors have high PD-L1 expression and do not harbor EGFR-sensitizing mutations or ALK translocations, in the first half of 2021; and
Initiate patient enrollment in the global Phase 3 AdvanTIG-301 trial (NCT04866017) of ociperlimab in combination with tislelizumab and concurrent chemoradiotherapy, in patients with previously untreated, locally advanced unresectable NSCLC in 2021.
Early-Stage Programs

Announced that the first patient was dosed in a Phase 1 clinical trial (NCT04649385) of BGB-15025, an investigational hematopoietic progenitor kinase 1 (HPK1) inhibitor that is designed to be a potent and highly selective small molecule oral inhibitor of HPK1 and is among the first HPK1 inhibitors to enter the clinic, representing a novel immuno-oncology approach; and
Continued to advance our early-stage clinical pipeline of internally-developed product candidates, including BGB-11417 (BCL-2 inhibitor in Phase 1 development for cancer), BGB-A445 (non-ligand competing OX40 monoclonal antibody in Phase 1 development in combination with tislelizumab for solid tumors), and BGB-10188 (PI3Kδ inhibitor in Phase 1 development in combination with BRUKINSA or tislelizumab for cancer).
Expected Milestones for Early-Stage Programs

Initiate a Phase 1 trial (NCT04771130) for BGB-11417, BeiGene’s investigational BCL-2 inhibitor, in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) in 2021. BGB-11417 has been designed to be a potent and selective small molecule Bcl-2 inhibitor; and
Initiate the Phase 2 portion of the Phase 1/2 trial (NCT03744468) of BGB-A425 in the first half of 2021.
Collaboration with Amgen

Received acceptance of a sBLA and priority review in China of BLINCYTO (blinatumomab) for the treatment of children with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.
Other Collaboration Programs

Sitravatinib, an investigational tyrosine kinase inhibitor of receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, MER), split family receptors (VEGFR2, KIT) and RET, licensed from Mirati Therapeutics Inc. (Mirati), in Asia (excluding Japan), Australia, and New Zealand.

Announced clinical data at AACR (Free AACR Whitepaper) on the combination of tislelizumab with sitravatinib, being jointly developed with Mirati, in two oral presentations from two cohorts of a Phase 1b trial (NCT03666143), in patients with unresectable or metastatic melanoma who were refractory or resistant to PD-1/L1 inhibitors, and in patients with advanced platinum-resistant ovarian cancer (PROC); and
Completed enrollment in the Phase 1/2 trial (NCT03941873) of sitravatinib as monotherapy and in combination with tislelizumab in patients with locally advanced or metastatic hepatocellular carcinoma (HCC) or gastric/gastroesophageal junction cancer (GC/GEJC).
Expected Milestones for Sitravatinib

Initiate a Phase 3 trial of sitravatinib in combination with tislelizumab in squamous and non-squamous NSCLC in 2021.
Manufacturing Operations

Announced approval from the NMPA to begin manufacturing commercial supply of tislelizumab at our state-of-the-art biologics facility in Guangzhou, China. At over one million square feet (100,000 square meters) and 8,000 liters of biologics capacity approved for commercial supply, this wholly owned facility has begun production and distribution of commercial supply of tislelizumab for the China market. An additional phase of construction currently in progress to bring total capacity to 64,000 liters is expected to be completed by the end of 2022.
COVID-19 Impact and Response

The Company expects that the worldwide health crisis of COVID-19 will continue to have a negative impact on its operations, including commercial sales, regulatory interactions, inspections, filings, and clinical trial recruitment, participation, and data read outs. There remains uncertainty regarding the future impact of the pandemic globally. The Company is striving to minimize delays and disruptions, and continues to execute on its commercial, regulatory and clinical development goals globally.
Corporate Developments

Announced that Julia Wang has been appointed Chief Financial Officer, effective June 30, 2021. Ms. Wang will succeed Howard Liang, Ph.D., who previously announced his intention to retire from BeiGene and who will stay on through June 30 to ensure an orderly transition; and
Continued to work on our filing for a proposed public offering and listing of the Company’s ordinary shares on the Science and Technology Innovation Board (STAR Market) of the Shanghai Stock Exchange, which is expected to be completed in 2021, subject to market conditions, shareholder approval, and regulatory approvals.
First Quarter 2021 Financial Results

Cash, Cash Equivalents, Restricted Cash, and Short-Term Investments were $4.8 billion as of March 31, 2021, compared to $4.7 billion as of December 31, 2020, representing an increase of $162.1 million.

In the three months ended March 31, 2021, cash provided by operating activities was $125.1 million, which included $650.0 million received as an upfront payment from the collaboration agreement with Novartis; capital expenditures were $42.4 million; cash used for upfront license payments was $8.5 million; and cash provided by financing activities was $107.4 million, consisting primarily of bank loans and the exercise of employee share options.
Revenue for the three months ended March 31, 2021 was $605.9 million, compared to $52.1 million in the same period of 2020.

Product revenues totaled $106.1 million for the three months ended March 31, 2021, compared to $52.1 million in the same period of 2020, and comprised:
– Sales of tislelizumab in China of $48.9 million, compared to $20.5 million in the prior year period;

– Sales of BRUKINSA of $22.1 million, compared to $0.7 million in the prior year period;

– Sales of XGEVA, the first product transferred to BeiGene from the Amgen collaboration, in China of $14.5 million. BeiGene commenced sales and marketing in China in July 2020;

– Sales of Bristol Myers Squibb (BMS) in-licensed products in China of $20.3 million, compared to $30.8 million in the same period of the prior year. The reduction in the current year period is due primarily to the lack of product sales of ABRAXANE following the suspension by the NMPA and voluntary recall by BMS in March 2020; and

Collaboration revenue for the three months ended March 31, 2021 was $499.8 million, resulting primarily from the partial recognition of the upfront payment of $650.0 million from Novartis. There was no collaboration revenue for the prior year period.
Expenses for the three months ended March 31, 2021 were $535.7 million, compared to $425.8 million in the same period of 2020.

Cost of Sales for the three months ended March 31, 2021 were $32.7 million, compared to $14.1 million in the same period of 2020. Cost of sales increased due to increased product sales of tislelizumab, BRUKINSA and XGEVA, and were partially offset by lower sales of BMS in-licensed products.
R&D Expenses for the three months ended March 31, 2021 were $320.7 million, compared to $304.3 million in the same period of 2020. The increase in R&D expenses was primarily attributable to continued increases in spending on our ongoing and late-stage pivotal clinical trials, the preparation for additional regulatory submissions, and manufacturing costs related to development programs and pre-commercial activities. Upfront fees related to in-process R&D for in-licensed product candidates decreased $34.5 million to $8.5 million for the three months ended March 31, 2021, compared to $43.0 million for the same period of 2020. R&D-related share-based compensation expense was $21.9 million for the three months ended March 31, 2021, compared to $20.4 million for the same period of 2020.
SG&A Expenses for the three months ended March 31, 2021 were $182.1 million, compared to $107.1 million in the same period of 2020. The increase in SG&A expenses was primarily attributable to increased headcount and increased external expenses related to the growth of our global commercial organization, as we continue to build our worldwide footprint. SG&A-related share-based compensation expense was $23.9 million for the three months ended March 31, 2021, compared to $17.9 million for the same period of 2020.
Net Income for the three months ended March 31, 2021 was $66.5 million, compared to a net loss of $363.7 million in the prior year period. For the three months ended March 31, 2021, basic and diluted earnings per share were $0.06 and $0.05, respectively, and basic and diluted earnings per American Depositary Share (ADS) were $0.73 and $0.69, respectively. For the three months ended March 31, 2020, net loss per share was $0.36 per share, or $4.70 per ADS.

[1] Research and development expense for the first quarter ended March 31, 2021 and 2020 includes upfront fees related to in-process research and development of in-licensed assets totaling $8.5 million and $43.0 million, respectively.

Exelixis Announces First Quarter 2021 Financial Results and Provides Corporate Update

On May 6, 2021 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the first 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, MAY 6, 2021, View Source [SID1234579345]).

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"The Exelixis team made significant progress across all components of our business in the first quarter of 2021, and delivered top-line revenue growth following the U.S. approval of CABOMETYX in combination with OPDIVO for first-line renal cell carcinoma in late January," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. "We also executed on a number of key priorities throughout the quarter, including initiating phase 1 clinical development of XL102, our novel small molecule CDK7 inhibitor, and successfully filing an Investigational New Drug application with the FDA for XB002, our first antibody-drug conjugate. We continue to focus on key 2021 development and regulatory milestones, and expect to report top-line results in the second quarter from the phase 3 COSMIC-312 pivotal trial evaluating the combination of cabozantinib and atezolizumab as a first-line treatment in advanced hepatocellular carcinoma, and file up to three supplemental New Drug Applications for cabozantinib across multiple indications by year-end. I look forward to providing updates on our progress throughout the year as we further our efforts to build a leading multi-product oncology company aiming to deliver new therapies and treatment options for the patients we serve."

First Quarter 2021 Financial Results

Total revenues for the quarter ended March 31, 2021 were $270.2 million, compared to $226.9 million for the comparable period in 2020.

Total revenues for the quarter ended March 31, 2021 included net product revenues of $227.2 million, compared to $193.9 million for the comparable period in 2020. The increase in net product revenues was primarily related to an increase in sales volume that was partially 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 of 2021.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $43.0 million for the quarter ended March 31, 2021, compared to $33.0 million for the comparable period in 2020. The increase in collaboration revenues was primarily related to 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), and increases in development cost reimbursements.

Research and development expenses for the quarter ended March 31, 2021 were $159.3 million, compared to $101.9 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, clinical trial costs, personnel expenses and stock-based compensation expense.

Selling, general and administrative expenses for the quarter ended March 31, 2021 were $102.4 million, compared to $62.9 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, which was partially offset by a decrease in the Branded Prescription Drug Fee.

Provision for (benefit from) income taxes for the quarter ended March 31, 2021 was $(3.6) million, compared to $11.4 million for the comparable period in 2020, primarily due to the change in pre-tax income (loss).

GAAP net income for the quarter ended March 31, 2021 was $1.6 million, or $0.01 per share, basic and $0.00 per share, diluted, compared to GAAP net income of $48.6 million, or $0.16 per share, basic and $0.15 per share, diluted, for the comparable period in 2020.

Non-GAAP net income for the quarter ended March 31, 2021 was $28.5 million, or $0.09 per share, basic and diluted, compared to non-GAAP net income of $59.4 million, or $0.19 per share, basic and 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 $1.6 billion at March 31, 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 maintaining the following previously provided financial guidance for fiscal year 2021:

Total revenues

$1,150 million – $1,250 million

Net product revenues

$950 million – $1,050 million

Cost of goods sold

Approximately 5% – 6% of net product revenue

Research and development expenses (1)

$600 million – $650 million

Selling, general and administrative expenses (2)

$375 million – $425 million

Effective tax rate

20% – 22%

Cash and investments (3)(4)

$1.6 billion – $1.7 billion

(1)

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

(2)

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

(3)

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

(4)

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 $227.2 million during the first quarter of 2021, up 13 percent over the prior quarter, with net product revenues of $223.6 million from CABOMETYX and $3.6 million from COMETRIQ (cabozantinib). Exelixis earned $23.8 million in royalty revenues during the quarter ended March 31, 2021, pursuant to collaboration agreements with our partners, Ipsen and Takeda.

FDA Approves CABOMETYX in Combination with OPDIVO for Advanced Renal Cell Carcinoma (RCC). In January 2021, Exelixis announced the FDA approved its supplemental New Drug Application (sNDA) for CABOMETYX in combination with Bristol Myers Squibb’s OPDIVO as a first-line treatment of patients with advanced RCC. The approval is based on 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 (ORR), with a favorable tolerability profile versus sunitinib.

Cabozantinib Data Presentations Demonstrate Broad Anti-Tumor Activity at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium (ASCO GU 2021). In February 2021, cabozantinib was the subject of multiple data presentations at ASCO (Free ASCO Whitepaper) GU 2021, held virtually from February 11-13. Presentations included: updated trial results with extended follow-up and patient-reported outcomes from the CheckMate -9ER trial demonstrating continued superior PFS, OS and ORR versus sunitinib across both the subgroup of 75 patients with sarcomatoid histology and the full study population, as well as significantly improved health-related quality of life; positive results from the SWOG S1500 trial ("PAPMET") of cabozantinib versus sunitinib in metastatic papillary RCC; positive findings from an international retrospective study of cabozantinib in RCC patients with brain metastases; and positive final results from the phase 1 trial sponsored and conducted by NCI-CTEP, including seven expansion cohorts, evaluating cabozantinib in combination with either nivolumab or nivolumab plus ipilimumab in patients with refractory metastatic genitourinary tumors.

FDA Grants Breakthrough Therapy Designation to Cabozantinib for the Treatment of Patients with Previously Treated Radioactive Iodine-Refractory Differentiated Thyroid Cancer (DTC). In February 2021, Exelixis announced that the FDA granted Breakthrough Therapy Designation to cabozantinib as a potential treatment for patients with DTC who progressed following prior therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). The FDA’s Breakthrough Therapy Designation aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases. The designation was based on an interim analysis of the COSMIC-311 phase 3 pivotal trial, in which cabozantinib demonstrated significant improvement in PFS.

Exelixis’ Partner Ipsen Receives European Commission (EC) Approval for CABOMETYX in Combination with OPDIVO as First-Line Treatment for Patients with Advanced RCC. In March 2021, Exelixis announced its partner Ipsen received approval from the EC for CABOMETYX in combination with OPDIVO as a first-line treatment for advanced RCC. The approval allows for the marketing of CABOMETYX in combination with OPDIVO in this indication in all 27 member states of the European Union, Norway, Iceland and Liechtenstein. The EC approval was based on the positive results of the phase 3 CheckMate -9ER pivotal trial.

Enrollment Completed in Phase 3 COSMIC-313 Pivotal Trial of Cabozantinib in Combination with Nivolumab and Ipilimumab Versus Nivolumab and Ipilimumab in Previously Untreated Advanced RCC. In March 2021, Exelixis announced that COSMIC-313, the phase 3 pivotal trial evaluating the triplet combination of cabozantinib, nivolumab and ipilimumab versus the combination of nivolumab and ipilimumab in patients with previously untreated advanced intermediate- or poor-risk RCC, completed enrollment. COSMIC-313 is a multicenter, randomized, double-blind, controlled phase 3 pivotal trial that enrolled approximately 840 patients globally. The primary endpoint of the trial is PFS, and additional endpoints include OS and ORR.

Cabozantinib Data Presentations at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. Cabozantinib will be the subject of 20 presentations at this year’s meeting, which has again adopted a virtual format as a result of the COVID-19 pandemic. Data presentations will include results from the COSMIC-311 and CheckMate -9ER phase 3 pivotal trials, as well as updates from externally sponsored studies.

Pipeline Highlights

Initiation of Phase 1 Clinical Trial Evaluating XL102 as a Single Agent and in Combination with Other Anti-Cancer Agents in Patients with Advanced or Metastatic Solid Tumors. In January 2021, Exelixis announced the initiation of a phase 1 clinical trial evaluating the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of XL102, both as a single agent and in combination with other anti-cancer agents, for the treatment of patients with inoperable, locally advanced or metastatic solid tumors. XL102 (formerly AUR102) is a potent, selective and orally bioavailable inhibitor of cyclin-dependent kinase 7 (CDK7) that Exelixis in-licensed from Aurigene Discovery Technologies Limited in December 2020 under the companies’ July 2019 collaboration agreement.

FDA Accepts Investigational New Drug Application (IND) for Tissue Factor-Targeting Antibody-Drug Conjugate (ADC) XB002 in Patients with Advanced Solid Tumors. In April 2021, 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. As a next-generation tissue factor-targeting ADC, XB002 (formerly ICON-2) 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.

Corporate Updates

Exelixis Outlines Key Priorities and Anticipated Milestones for 2021. In January 2021, Exelixis announced its key priorities and anticipated milestones for 2021, including: the commercial launch of CABOMETYX in combination with OPDIVO as a first-line treatment of patients with advanced RCC; potential sNDA submissions for CABOMETYX in DTC, hepatocellular carcinoma and metastatic castration-resistant prostate cancer; progress and enrollment in the COSMIC and CONTACT clinical studies evaluating cabozantinib as a single agent or in combination with immune checkpoint inhibitors (ICIs); expanded clinical development activities for XL092; and multiple INDs for preclinical assets. Exelixis presented the details of its key priorities and anticipated milestones at the 39th Annual J.P. Morgan Healthcare Conference, which was held virtually from January 11-14.

Exelixis and Adagene Inc. (Adagene) Enter into Collaboration and License Agreement to Develop Novel Masked ADC Therapies with Improved Safety and Efficacy Profiles. In February 2021, Exelixis and Adagene announced a collaboration and license agreement under which Exelixis will utilize Adagene’s SAFEbody technology platform to generate masked versions of monoclonal antibodies (mAbs) from Exelixis’ growing preclinical pipeline for the development of SAFEbodies or other innovative biologics against Exelixis-nominated targets. Under the terms of the agreement, Exelixis received an exclusive, worldwide license to develop and commercialize any potential ADC products generated by Adagene with respect to an initial target, as well as a second target Exelixis may nominate during the collaboration term.

Exelixis Enters into Exclusive License Agreement with WuXi Biologics (WuXi Bio) to Support Further Expansion of its Growing Oncology Biologics Pipeline. In March 2021, Exelixis announced an exclusive license agreement with WuXi Bio to support the continued expansion of Exelixis’ oncology biologics pipeline. Under the terms of the agreement, Exelixis received an exclusive license to a panel of mAbs to a validated target, discovered based on WuXi Bio’s integrated technology platforms, for the development of ADC, bispecific, and certain other novel tumor-targeting biologics applications.

Exelixis Enters Clinical Trial Collaboration and Supply Agreement with Merck KGaA, Darmstadt, Germany (Merck KGaA) and Pfizer Inc. (Pfizer) to Evaluate XL092 and Avelumab (BAVENCIO) in Various Forms of Locally Advanced or Metastatic Urothelial Carcinoma (UC). In March 2021, Exelixis announced a clinical trial collaboration and supply agreement with Merck KGaA and Pfizer for the ongoing phase 1b dose escalation study STELLAR-001 (previously called "XL092-001"), adding three new cohorts that will evaluate the safety and tolerability of XL092, Exelixis’ novel next generation tyrosine kinase inhibitor, in combination with avelumab, an anti-PD-L1 ICI, in patients with locally advanced or metastatic UC.

Exelixis Expands its Biotherapeutics Portfolio with Acquisition of Anti-Müllerian Hormone Receptor 2 (AMHR2) Program from GamaMabs Pharma SA (GamaMabs). In May 2021, 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 Receives Notice of Paragraph IV Certifications. In May 2021, Exelixis received a notice letter from Teva Pharmaceuticals USA, Inc. (Teva) regarding an Abbreviated New Drug Application Teva submitted to the FDA, requesting approval to market a generic version of CABOMETYX tablets. Teva’s notice letter 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 intends to continue to vigorously defend its cabozantinib intellectual property estate.

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 April 2, 2021, January 1, 2021 and April 3, 2020 are indicated as being as of and for the periods ended March 31, 2021, December 31, 2020, and March 31, 2020, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the first 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, May 6, 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 6139476 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on May 8, 2021. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 6139476. A webcast replay will also be archived on www.exelixis.com for one year.

Ascendis Pharma A/S Announces Presentations for TransCon™ PTH at Upcoming Medical Conferences

On May 6, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported presentations featuring TransCon PTH at two upcoming medical conferences: the 48th annual meeting of the European Calcified Tissue Society (ECTS), taking place virtually May 6-8, 2021 and the 23rd European Congress of Endocrinology taking place virtually May 22-26, 2021 (Press release, Ascendis Pharma, MAY 6, 2021, View Source [SID1234579344]).

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"We are excited to share the 26-week open-label extension (OLE) data from our phase 2 PaTH Forward Trial in adult patients with hypoparathyroidism with the European medical community," said Mark Bach, M.D., Ph.D., Senior Vice President of Endocrine Medical Sciences at Ascendis Pharma.

"These data provide evidence indicating that TransCon PTH has the potential to be a first line therapy as a hormone replacement in patients with hypoparathyroidism. The study was selected for oral presentations by both ECTS and ECE meeting organizers which we think underscores the significance of these results," continued Dr. Bach.

ECTS 2021: Presentation Details

Oral Presentation
Title Date/Time
Week 26 Results from the PaTH Forward Open-Label Extension Trial Support TransCon PTH as a Potential Hormone Replacement Therapy for Patients with Hypoparathyroidism Plenary Oral Presentations 2: Novel Aspects of Osteoporosis and Treatments
May 7, 2021
16:30 – 17:30 CEST
e-ECE 2021: Presentation Details

Oral Presentation
Title Date/Time
TransCon PTH as a Potential Hormone Replacement Therapy for Patients with Hypoparathyroidism: PaTH Forward Open-Label Extension Trial Week 26 Results Oral Communications 6: Calcium and Bone
May 25, 2021
14:30 – 15:30 CEST
The posters will be available on the Ascendis website under Selected Publications in the Pipeline section: View Source If you are a healthcare provider who would like more information, please contact: [email protected].

About TransCon Technology

TransCon refers to "transient conjugation." The proprietary TransCon platform is an innovative technology to create new therapies that are designed to optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and can be used systemically or locally.