On April 28, 2022 Gilead Sciences, Inc. (Nasdaq: GILD) reported its results of operations for the first quarter of 2022 (Press release, Gilead Sciences, APR 28, 2022, View Source [SID1234613140]).

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"Gilead’s performance in the first quarter reflects the strength and diversity of our business with both our HIV and oncology therapies contributing to year-over-year growth," said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. "Biktarvy delivered strong 18% year-over-year revenue growth, and oncology sales increased by 60% year-over-year, driven by increased demand for Trodelvy and our cell therapy products. As we continue to advance our broad oncology portfolio, we look forward to providing more new options for people living with cancer."

First Quarter 2022 Financial Results

First quarter 2022 revenue increased 3% to $6.6 billion compared to the same period in 2021, primarily due to increased demand for Biktarvy (bictegravir 50mg/emtricitabine 200mg ("FTC")/tenofovir alafenamide 25mg ("TAF")) and Veklury (remdesivir 100mg for injection), partially offset by the impact of the loss of exclusivity for Truvada (FTC/tenofovir disoproxil fumarate 300mg ("TDF")) in the United States and unfavorable pricing dynamics for hepatitis C virus ("HCV") products.
Diluted Earnings Per Share ("EPS") decreased to $0.02 for the first quarter of 2022 compared to $1.37 for the same period in 2021. The decrease was primarily the result of a $2.7 billion in-process research and development ("IPR&D") impairment related to assets acquired by Gilead from Immunomedics in 2020.
Non-GAAP diluted EPS increased 4% to $2.12 for the first quarter of 2022 compared to $2.04(1) for the same period in 2021, primarily reflecting higher product sales.
As of March 31, 2022, Gilead had $6.8 billion of cash, cash equivalents and marketable debt securities compared to $7.8 billion as of December 31, 2021.
During the first quarter of 2022, Gilead generated $1.8 billion in operating cash flow, which includes the cash outflow related to the $1.25 billion legal settlement.
During the first quarter of 2022, Gilead made a $725 million collaboration opt-in payment to Arcus Biosciences, Inc., repaid $500 million of debt, paid dividends of $945 million and repurchased $352 million of common stock.
________________________________

(1)

Non-GAAP diluted EPS has been recast due to an update to our non-GAAP policy in the first quarter 2022, resulting in a $0.04 reduction of previously-reported non-GAAP diluted EPS for the first quarter of 2021. Refer to Non-GAAP Financial Information section below for further information.

Product Sales Performance

First quarter 2022 product sales increased 3% to $6.5 billion compared to the same period in 2021. Total product sales, excluding Veklury, increased 2% to $5.0 billion in the first quarter of 2022 compared to the same period in 2021, primarily reflecting higher demand for Biktarvy, our cell therapy products and Trodelvy (sacituzumab govitecan-hziy), partially offset by unfavorable pricing dynamics in HCV.

HIV product sales increased 2% to $3.7 billion in the first quarter of 2022 compared to the same period in 2021, primarily reflecting higher demand for Biktarvy and favorable pricing dynamics partially offset, as expected, by the loss of exclusivity of Truvada in the United States.

Biktarvy sales increased 18% year-over-year in the first quarter of 2022, primarily due to higher demand.
Descovy (FTC 200mg/TAF 25mg) sales increased 4% year-over-year in the first quarter of 2022, primarily driven by increased demand and favorable pricing, partially offset by unfavorable channel inventory dynamics.
Truvada sales decreased 72% year-over-year in the first quarter 2022, as expected, primarily due to the loss of exclusivity in the United States in October 2020.
HCV product sales decreased 22% to $399 million in the first quarter of 2022 compared to the same period in 2021, primarily driven by lower net price and fewer patient starts.

Hepatitis B virus ("HBV") and hepatitis delta virus ("HDV") product sales increased 7% to $235 million in the first quarter of 2022 compared to the same period in 2021. Vemlidy (TAF 25mg) sales increased 10% in the first quarter of 2022 compared to the same period in 2021, primarily driven by higher demand in geographies outside the United States. Hepcludex (bulevirtide) contributed $11 million in the first quarter of 2022, as launch activities continued across Europe.

Cell therapy product sales increased 43% to $274 million in the first quarter of 2022 compared to the same period in 2021.

Yescarta(axicabtagene ciloleucel) sales increased to $211 million in the first quarter of 2022, primarily driven by demand for relapsed or refractory large B-cell lymphoma ("LBCL") in the United States and Europe and follicular lymphoma in the United States.
Tecartus(brexucabtagene autoleucel) sales were $63 million in the first quarter of 2022, primarily driven by growing adoption in Europe for mantle cell lymphoma and for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia in the United States.
Trodelvy sales increased 103% to $146 million in the first quarter of 2022 compared to the same period in 2021, primarily reflecting uptake in the second line setting for the treatment of metastatic triple-negative breast cancer in the United States and Europe as well as metastatic urothelial cancer in the United States.

Veklury sales increased by 5% to $1.5 billionfor the first quarter of 2022 compared to the same period in 2021. Veklury revenue generally reflects COVID-19 related rates of infections, hospitalizations and vaccinations, as well as the availability, uptake and effectiveness of alternative treatments for COVID-19.

First Quarter 2022 Product Gross Margin, Operating Expenses and Effective Tax Rate

Product gross margin was 78.2% for the first quarter of 2022 compared to 78.5% for the same period in 2021, primarily driven by a change in product mix and restructuring costs for the closing of a New Jersey manufacturing site in 2022, partially offset by lower inventory reserve adjustments. Non-GAAP product gross margin was 87.4% for the first quarter of 2022 compared to 86.5% in the same period in 2021, primarily driven by lower inventory reserve adjustments.
Research and development ("R&D") expenses for the first quarter of 2022 were $1.2 billion compared to $1.1 billion in the same period in 2021. Non-GAAP R&D expenses for the first quarter of 2022 were $1.2 billion compared to $1.0 billion in the same period in 2021. The increase in R&D and non-GAAP R&D expenses primarily reflect increased clinical activities for Trodelvy.
Selling, general and administrative ("SG&A") expenses were $1.1 billion for the first quarter of 2022 and for the same period in 2021. Non-GAAP SG&A expenses for the first quarter of 2022 were $1.1 billion compared to $1.0 billion in the same period in 2021.
The effective tax rate ("ETR") for the first quarter of 2022 was 107.9% compared to 23.9% for the same period in 2021, primarily driven by the $2.7 billion IPR&D impairment. Non-GAAP ETR for both the first quarter 2022 and the same period last year was 18.4%.
Guidance and Outlook

For the full-year, we have updated our EPS guidance to primarily reflect the $2.7 billion IPR&D impairment. We now expect EPS between $3.00 and $3.50, compared to $4.70 and $5.20 previously. There is no change to other guidance shared on February 1, 2022:

Total product sales between $23.8 billion and $24.3 billion.
Total product sales, excluding Veklury, between $21.8 billion and $22.3 billion.
Total Veklury sales of approximately $2.0 billion.
Non-GAAP earnings per share between $6.20 and $6.70.
This financial guidance excludes the impact of any expenses related to potential acquisitions or business development transactions that have not been executed, fair value adjustments of equity securities and discrete tax charges or benefits associated with changes in tax related laws and guidelines as Gilead is unable to project such amounts. A reconciliation between GAAP and non-GAAP financial information for the 2022 guidance is provided in the accompanying tables. Also see the Forward-Looking Statements described below. The financial guidance is subject to a number of risks and uncertainties, including uncertainty around the duration and magnitude of the COVID-19 pandemic. While the pandemic can be expected to continue to impact Gilead’s business and broader market dynamics, the rate and degree of these impacts as well as the corresponding recovery from the pandemic may vary across Gilead’s business.

Key Updates Since Our Last Quarterly Release

Viral Diseases

Received a Complete Response Letter from FDA related to vial compatibility issues for the New Drug Application of investigational lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced ("HTE") people with multi-drug resistant HIV-1 infection.
Presented one-year data from studies of investigational lenacapavir at the 29th Conference on Retroviruses and Opportunistic Infections ("CROI") with results from each of the Phase 2/3 CAPELLA trial in HTE people living with multi-drug resistant HIV and Phase 2 CALIBRATE trial in treatment-naive people living with HIV demonstrating high rates of virologic suppression at one-year.
Presented five-year results from two Phase 3 studies of Biktarvy at CROI which reinforced Biktarvy’s sustained efficacy and durable viral suppression with zero cases of treatment failure due to emergent resistance observed.
Announced data demonstrating in vitro activity of Veklury against ten SARS-CoV-2 variants, including Omicron. Additionally, interim results from the Phase 2/3 CARAVAN trial of Veklury in pediatric patients aged 28 days to less than 18 years hospitalized with COVID-19 were presented at CROI.
Oncology

Announced results from the Phase 3 TROPiCS-02 study of Trodelvy in patients with HR+/HER2- metastatic breast cancer who had been heavily pre-treated. The study met its primary endpoint, demonstrating a statistically significant improvement in progression-free survival compared to physician’s choice of chemotherapy. Additionally, at the first interim analysis, a trend in improvement for overall survival ("OS") was observed, a key secondary endpoint. No new safety concerns were noted. The company will discuss the study data with regulators and the study will continue to follow patients for OS and detailed results will be presented at an upcoming medical conference. Trodelvy has not been approved by any regulatory agency for the treatment of HR+/HER2- metastatic breast cancer, and its safety and efficacy have not been established for this indication.
Received FDA approval for Yescarta for the treatment of adult patients with LBCL that is refractory to first-line chemoimmunotherapy or that relapse within 12 months of first-line chemoimmunotherapy. Additionally, the National Comprehensive Cancer Network updated its Clinical Practice Guidelines for B-cell Lymphomas to include Yescarta as a Category 1 recommendation for "Relapsed disease <12 mo or Primary refractory disease" under diffuse large B-cell lymphoma.
Announced that FDA has lifted the partial clinical hold on studies evaluating investigational magrolimab in combination with azacitidine for the treatment of myelodyspastic syndrome and acute myeloid leukemia.
Corporate

Announced that the company’s Board of Directors declared a quarterly dividend of $0.73 per share of common stock for the second quarter of 2022. The dividend is payable on June 29, 2022, to stockholders of record at the close of business on June 15, 2022. Future dividends will be subject to Board approval.
Announced $24 million in grants to support 116 organizations in 41 countries as part of Gilead’s Zeroing In: Ending the HIV Epidemic program. Grantee organizations will focus on advancing comprehensive HIV innovation programs, digital health innovations, and/or community outreach and education.
Received FDA approval for commercial production at Kite’s new CAR T-cell therapy manufacturing facility in Frederick, Maryland.
Purchased approximately 27 acres of additional land in Oceanside, California to potentially support further manufacturing operations.
Certain amounts and percentages in this press release may not sum or recalculate due to rounding.

Non-GAAP Financial Information

The information presented in this document has been prepared in accordance with U.S. generally accepted accounting principles ("GAAP"), unless otherwise noted as non-GAAP. Management believes non-GAAP information is useful for investors, when considered in conjunction with Gilead’s GAAP financial information, because management uses such information internally for its operating, budgeting and financial planning purposes. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Gilead’s operating results as reported under GAAP. Non-GAAP financial information generally excludes acquisition-related expenses including amortization of acquired intangible assets and inventory step-up charges, and other items that are considered unusual or not representative of underlying trends of Gilead’s business, fair value adjustments of equity securities and discrete and related tax charges or benefits associated with changes in tax related laws and guidelines. Although Gilead consistently excludes the amortization of acquired intangible assets from the non-GAAP financial information, management believes that it is important for investors to understand that such intangible assets were recorded as part of acquisitions and contribute to ongoing revenue generation.Non-GAAP measures may be defined and calculated differently by other companies in the same industry. Reconciliations of the non-GAAP financial measures to the most directly comparable GAAP financial measures are provided in the accompanying tables.

Beginning in the first quarter of 2022, consistent with recent industry communications from the U.S. Securities and Exchange Commission ("SEC"), Gilead no longer excludes acquired IPR&D expenses from its non-GAAP financial measures. Acquired IPR&D expenses reflect the initial costs of externally-developed IPR&D projects, acquired directly in a transaction other than a business combination, that do not have an alternative future use, including upfront and other payments related to various collaborations and the initial costs of rights to IPR&D projects. Prior period non-GAAP financial measures are revised to conform to the new presentation.

Conference Call

At 1:30 p.m. Pacific Time today, Gilead will host a conference call to discuss Gilead’s results. A live webcast will be available on View Source and will be archived on www.gilead.com for one year.

On April 28, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, reported that the BTK inhibitor BRUKINSA (zanubrutinib) has been approved in Uruguay for the treatment of adult patients with previously treated mantle cell lymphoma (MCL), relapsed or refractory marginal zone lymphoma (MZL), and Waldenström’s macroglobulinemia (WM) (Press release, BeiGene, APR 28, 2022, View Source [SID1234613173]). BeiGene and Adium entered into an exclusive distribution agreement for Adium to commercialize BRUKINSA in Latin America.

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"Tolerability of treatments for B-cell malignancies is an important consideration with BTK inhibition, and BRUKINSA was designed with that in mind to maximize BTK occupancy and minimize off-target binding. Today, we have a new treatment option for patients with MCL, MZL and WM in Uruguay, providing hope for improved treatment outcomes," said Dr. Karina Cicinelli, Corporate Medical Director at Adium.

"We are proud of the progress BeiGene has made in Latin America over the past year, with this approval in three indications in Uruguay following recent launches in Brazil, Chile and Ecuador. With Adium’s established commercial presence in Latin America, we hope patients with MCL, MZL, and WM will soon have access to this important treatment option," commented Eduardo Molinari, Senior Director of New Market Development in Latin America at BeiGene.

About BRUKINSA

BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

BRUKINSA has previously been approved for three indications in the United States: for the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (Nov. 2019)*; for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) (Aug. 2021); and for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (Sept. 2021)*.

BRUKINSA is supported by a broad clinical program which includes more than 3,900 subjects in 35 trials across 28 markets. To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the United States, China, the EU and Great Britain, Canada, Australia and additional international markets. Currently, more than 40 additional regulatory submissions are in review around the world.

* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

BeiGene Oncology

BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 2,900 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 14,500 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the United States, China, the EU and U.K., Canada, Australia and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab as well as the PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including with Mirati Therapeutics, Seagen, and Zymeworks.

In January 2021 BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD1 antibody tislelizumab in North America, Europe, and Japan. Building upon this productive collaboration, including a biologics license application (BLA) under FDA review, BeiGene and Novartis announced an option, collaboration and license agreement in December 2021 for BeiGene’s TIGIT inhibitor ociperlimab that is in Phase 3 development. Novartis and BeiGene also entered into a strategic commercial agreement through which BeiGene will promote five approved Novartis Oncology products across designated regions of China.

On April 28, 2022 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported it has received approval from the Spanish Agency for Medicines and Health Products (AEMPS) Competent Authority and from the CElm Provincial de Sevilla Ethics Committee in Spain for the Company’s potentially pivotal study of Berubicin for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer (Press release, CNS Pharmaceuticals, APR 28, 2022, View Source [SID1234613189]).

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"We continue to build momentum in our potentially pivotal study of Berubicin, and importantly advance toward bringing a much needed treatment option to patients. We have significantly bolstered our international presence and we are grateful to Spain for joining us in support of this trial. We are committed to driving this development program forward and are executing on all fronts to build momentum," commented John Climaco, CEO of CNS Pharmaceuticals.

Dr. Juan M Sepúlveda Sánchez, MD, 12 de Octubre University Hospital, Madrid, added, "There remains a significant unmet medical need for patients with GBM. We are pleased to bring this important clinical program to Spain and help to further evaluate Berubicin’s potential to meet that need. I look forward to working alongside the rest of the clinical team to advance CNS Pharmaceuticals’ potentially pivotal study and importantly, progress Berubicin through the clinic with the hope of bringing a valuable treatment options to patients and physicians."

Berubicin is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier currently being evaluated in a potentially pivotal global study evaluating its efficacy and safety in the treatment of GBM. The potentially pivotal global trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy. Approximately 243 patients with GBM after failure of standard first line therapy will be randomized in a 2:1 ratio to receive Berubicin or lomustine for the evaluation of Overall Survival, the primary endpoint of the study. Overall Survival is a rigorous endpoint that the U.S. Food and Drug Administration (FDA) has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm.

A pre-planned, non-binding futility analysis will be performed after approximately 30 to 50% of all planned patients have completed the primary endpoint at 6 months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis.

The FDA recently granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with the FDA to expedite the development and review process. As previously announced, the Company also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA.

For more information about the potentially pivotal Berubicin trial, visit clinicaltrials.gov and reference identifier NCT04762069.

About Berubicin

Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata Pharmaceuticals, Inc. Berubicin, was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center.

On April 28, 2022 NEC reported (Press release, NEC, APR 28, 2022, View Source [SID1234613084])

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1. Consolidated Financial Results for the Year ended March 31, 2022 (April 1, 2021 – March 31, 2022)
(1) Consolidated Operating Results
(2) Consolidated Financial Position
(3) Consolidated Cash Flows

2. Dividends
3. Consolidated Financial Results Forecast for the Fiscal Year Ending March 31, 2023 (April 1, 2022 – March 31, 2023)

1. Non-consolidated Financial Results for the Year Ended March 31, 2022 (April 1, 2021 – March 31, 2022)
(1) Non-consolidated Operating Results
(2) Non-consolidated Financial Position

*This consolidated financial results falls outside the scope of audit to be performed by certified public accountants or an audit firm.

*Explanation concerning the appropriate use of the financial results forecast and other special matters (Adjusted profit (loss)) "Adjusted operating profit (loss)" is an indicator for measuring underlying profitability in order to clarify the contribution of acquired companies to the NEC Group’s overall earnings. It is measured by deducting amortization of intangible assets recognized as a result of M&A and expenses for acquisition of companies (financial advisory fees and other fees) from operating profit (loss). Also, "Adjusted net profit (loss) attributable to owners of the parent" is an indicator for measuring underlying profitability attributable to owners of the parent. It is measured by deducting adjustment items of operating profit (loss) and corresponding amounts of tax and non-controlling interests from net profit (loss) attributable to owners of the parent. (Cautionary statement with respect to forward-looking statements) The forward-looking statements such as operating results forecast contained in this statements summary are based on the information currently available to NEC Corporation ("the Company") and certain assumptions considered reasonable. Actual operating results may differ significantly from these forecasts due to various factors.

For details, please refer to "3. Cautionary Statement with Respect to Forward-Looking Statements" on page 16. (How to obtain supplementary financial materials and information on the financial results briefing) On April 28, 2022, the Company will hold a financial results briefing for the institutional investors and analysts. Presentation materials will be posted on the company website after the release of financial results, and the presentation video and Q&A summary will be also posted on the company website promptly after the financial results briefing. In addition to the above, the Company periodically holds briefings on business and operating results for the individual investors. Presentation materials and Q&A summary will be posted on the company website promptly after the briefing. For the schedule and details, please check the company website.

On April 28, 2022 XBiotech (NASDAQ: XBIT) reported that the French National Agency for the Safety of Medicines and Health Products [L’Agence nationale de sécurité du médicament et des produits de santé (ANSM)] approved the launch of a multicenter randomized clinical study for XBiotech’s candidate cancer treatment Natrunix in combination with trifluridine/tipiracil for the treatment of colorectal cancer (Press release, XBiotech, APR 28, 2022, View Source [SID1234613124]). The French National Cancer Institute (INCA) has also awarded a grant to fund all clinical costs for the study.

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Investigators will combine Natrunix and trifluridine/tipiracil as a new candidate therapy for metastatic colorectal cancer in subjects that have failed earlier treatment with oxaliplatin, irinotecan, and fluoropyrimidine. The study will randomize patients to receive the Natrunix plus chemotherapy or placebo plus chemotherapy and is designed to seamlessly proceed to a Phase III study based on achievement of certain early efficacy milestones.

Headed by Dr. François Ghiringhelli and Dr. Come Lepage, the clinical program will include over 20 participating clinical centers and enroll at least 160 subjects. Dr. François Ghiringhelli is Professor in Medical Oncology and Director of the INSERM research team at the Georges-Francois Leclerc Cancer Centre, and Dr. Come Lepage, Prof. Department Gastroenterology and Digestive Oncology, University Hospital Dijon, Dijon, France. The study design was developed by the lead investigators in collaboration with XBiotech.

The first portion of the study will be an open label, dose escalation (3:3). The Phase II portion will be a multicenter, randomized, double blind, placebo-controlled, non-comparative trial that will enroll 160 subjects. The main objective of the phase II study is to evaluate the efficacy of Natrunix + trifluridine/tipiracil in comparison with placebo + trifluridine/tipiracil with respect to 6-month overall survival in patients with refractory metastatic colorectal cancer. Secondary efficacy measures in the phase 2 portion will include progression free survival, median overall survival, tolerance, quality of life, serum markers of inflammatory cytokines, and tumor markers by immunohistochemistry. With successful completion of the primary endpoint in the Phase II portion, the study will continue into a phase III trial with the number of additional patients enrolled based upon results from the Phase II.

Natrunix is a therapeutic monoclonal antibody discovered, manufactured and undergoing clinical development by XBiotech. The antibody blocks the activity of interleukin-1 alpha (IL-1α). Malignant tumors "trick" the body into producing IL-1α, which has multiple roles in supporting tumor growth and spread. IL-1α expression is also induced by cytotoxic chemotherapy. IL-1α is a potent activator of new blood vessel formation (upregulating VEGF and tumor neoangiogenesis); it mediates breakdown of connective tissue through stimulating matrix metalloproteinase production; facilitates metastasis (enhancing adhesion and migration across blood vessels); and mediates systemic illness (fatigue, anorexia, and anxiety) through activating the hypothalamic-pituitary-adrenal axis. Using Natrunix to block IL-1α in combination therapy could inhibit tumor growth and spread, reduce unwanted effects of chemotherapy, and improve outcomes.

Colorectal cancer is one of the most common forms of cancer in Europe and the United States, with the American Cancer Society’s estimating over 151,000 new cases and over 52,000 deaths in the United States alone.

About True Human Therapeutic Antibodies
XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.