On February 4, 2021 Illumina, Inc. (NASDAQ:ILMN) reported an agreement with the Belgian Society of Medical Oncology (BSMO) which is running a new national pilot to evaluate the use of comprehensive genomic profiling (CGP) in 864 patients with advanced metastatic cancer (Press release, Illumina, FEB 4, 2021, View Source [SID1234574631]). The BALLETT (Belgian Approach for Local Laboratory Extensive Tumor Testing) study will recruit patients from 12 participating sites to be offered CGP from one of nine next-generation sequencing laboratories across Belgium. Data from CGP will be used to determine the best therapeutic options for patients through access to targeted medicines with the aim of improving clinical outcomes in advanced cancer. The study will begin next month.

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CGP uses NGS to analyze hundreds of genes and biomarkers in tissue and blood samples and detect those that are clinically relevant in driving cancer growth. Illumina will provide its CGP panel, TruSight Oncology 500 (TSO500), as well as NovaSeq 6000 and NextSeq sequencing platforms for the study. Clinical interpretation of the sequencing data will be carried out using PierianDx Clinical Genomic Workspace solution and OncoDNA Clinical Decision Support Platform OncoKDM.

"Belgium is a leader in the application of new technologies to make precision healthcare a reality. Through this new study, we want to ensure that patients across Belgium can receive the right treatment for their particular cancer at the right time," said Dr. Sylvie Rottey, Chair BSMO.

"CGP reveals the unique molecular profile of a patient’s cancer which empowers their oncology team to propose the most effective course of treatment suited to them," said Phil Febbo, MD, Chief Medical Officer at Illumina. "We are very pleased to be working with the BSMO and the different cancer institutes across Belgium to examine the value of CGP in a real-world setting in terms of its ability to improve patient outcomes through more targeted treatment options."

Doctor Brigitte Maes of the Jessa Hospital in Belgium, Coordinator of the BALLETT study, said, "As part of Belgium’s broad approach to advancing precision medicine the study will generate valuable insights into the value of CGP versus currently reimbursed sequencing approaches. For example, in addition to genetic mutations that drive cancer formation, CGP will also identify cancers driven by the tumor mutational burden (TMB) biomarker which can guide patients towards immunotherapy treatments. This means that the study will give access to additional treatments which may not have been considered through more traditional diagnostic testing."

Genomic data together with de-identified clinical data from the study will be used to populate a newly-established national genomic tumor database, collated with oversight from Belgium’s Scientific Institute for Public Health, Sciensano, designed to advance precision medicine and patients’ access to novel effective therapies.

On February 4, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the fourth quarter and full-year ended December 31, 2020 and provided full-year 2021 financial expense guidance (Press release, Neurocrine Biosciences, FEB 4, 2021, View Source [SID1234574648]).

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"In 2020, we served more patients with tardive dyskinesia than ever before despite the pandemic weighing on the development of the overall market. We are pleased with the recently updated guidelines from the American Psychiatric Association that now recommend first-line treatment for tardive dyskinesia with a VMAT2 inhibitor, which we hope will benefit even more patients as the vast majority of patients living with tardive dyskinesia remain undiagnosed," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "In 2021, we plan to initiate eight mid-to-late stage clinical studies and look forward to important data read-outs for NBI-1065844 in the negative symptoms of schizophrenia and valbenazine for the treatment of chorea associated with Huntington’s Disease."

Fourth Quarter and Full-Year Net Product Sales and Revenues Highlights:

INGREZZA net product sales for the fourth quarter and full-year 2020 were $240 million and $993 million, respectively, representing an increase of 1% and 32% versus respective 2019 comparable periods
INGREZZA inventory adjusted net product sales for the fourth quarter of 2020 were $258 million representing 4% sequential growth vs. the third quarter of 2020
INGREZZA end of fourth quarter 2020 days-on-hand channel inventory decreased by $18 million relative to the third quarter
INGREZZA new prescriptions increased in the fourth quarter of 2020 vs. the third quarter of 2020
Refill and persistency rates continued to be strong for existing INGREZZA patients
ONGENTYS launched in the United States in late September 2020 and net product sales for the fourth quarter of 2020 were approximately $1 million reflecting growing uptake throughout the quarter
Elagolix royalties received from AbbVie on combined fourth quarter 2020 net sales of ORILISSA (elagolix tablets) and ORIAHNNTM (elagolix, estradiol and norethindrone acetate capsules and elagolix capsules) totaled $6 million
Financial Highlights:

Fourth quarter 2020 GAAP net income and diluted earnings per share were approximately $348 million and $3.58, respectively, compared with approximately $34 million and $0.35, respectively, in the fourth quarter of 2019, primarily driven by a non-cash tax benefit of $296 million related to the release of substantially all of the Company’s valuation allowance against its deferred tax assets on December 31, 2020
Fourth quarter 2020 non-GAAP net income and diluted earnings per share were approximately $88 million and $0.91, respectively, compared with approximately $102 million and $1.05, respectively, in the fourth quarter of 2019 driven by:
Increased Research and Development (R&D) expense primarily due to increased investment across our expanded pipeline programs and higher headcount costs
Increased Selling, General and Administrative (SG&A) expense primarily due to increased investment in marketing initiatives and higher headcount costs
2020 full-year GAAP and non-GAAP net income of $407 million and $402 million, respectively, represents year-over-year growth of approximately 10 times and 41%, respectively
Total debt outstanding decreased by $136 million to $381 million after repurchase of approximately 26% of debt outstanding during the fourth quarter of 2020. The total aggregate repurchase price of $187 million was paid in cash and the transaction resulted in an $18 million loss recognized during the fourth quarter of 2020.
At December 31, 2020, the Company had cash, cash equivalents and debt securities available-for-sale of $1.0 billion
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.

Income Tax Benefit:
The Company’s fourth quarter financial results include the reversal of substantially all of the valuation allowance recorded against the deferred tax assets of the Company. This reversal resulted in the recognition of a non-cash income tax benefit in the fourth quarter 2020 of $296 million, or $3.05 earnings per diluted share. The Company has performed a continuing evaluation of its deferred tax asset valuation allowance on a quarterly basis. The Company has now concluded that, as of December 31, 2020, it is more likely than not that the Company will generate sufficient taxable income within the applicable net operating loss and R&D carryforward periods to realize substantially all of its deferred tax assets. This conclusion, and the resulting reversal of the deferred tax asset valuation allowance, is based upon consideration of a number of factors, including the Company’s strong financial performance over the past few years and its forecast of future profitability.

After recognizing the valuation allowance reversal, the Company’s net deferred tax assets totaled $319 million at December 31, 2020, net of a valuation allowance of $50 million. The ability to recognize the remaining deferred tax assets that continue to be subject to a valuation allowance will be evaluated on a quarterly basis to determine if there are significant events that would affect the Company’s ability to utilize these deferred tax assets. As a result of this reversal, the Company will begin recording federal and state tax expense on its earnings beginning in the first quarter of 2021. No federal cash tax is expected in 2021 based upon a net operating loss position of approximately $500 million entering 2021.

Recent Events

In October 2020, the U.S. Food and Drug Administration (FDA) requested additional non-clinical data to support the Investigational New Drug Application (IND) we submitted in August 2020 in support of a Phase II clinical study for NBI-921352 in patients with SCN8A Developmental Epileptic Encephalopathy (SCN8A-DEE). Based on feedback received in January 2021, we plan to initiate a Phase II clinical study in adolescent patients (aged 12 years and older) with SCN8A-DEE in the third quarter of 2021, and the study protocol will be amended to include younger pediatric patients (aged 2-11 years) with SCN8A-DEE as soon as the FDA has reviewed and approved additional non-clinical information. We are also advancing clinical plans to initiate a Phase II clinical study of NBI-921352 for the treatment of adult focal epilepsy in 2021. In addition, in October 2020, we announced the FDA granted us Rare Pediatric Disease Designation for NBI-921352 for the treatment of SCN8A-DEE.
In November 2020, the Company announced the initiation of a Phase II study of NBI-827104 (formerly ACT-709478) in Epileptic Encephalopathy with Continuous Spike and Wave during Sleep (CSWS), a rare pediatric epilepsy. NBI-827104 was licensed from Idorsia and is a potent, selective, orally active and brain penetrating T-type calcium channel blocker.
In February 2021, the Mitsubishi Tanabe Pharma Corporation (MTPC) reported positive top-line results from the J-KINECT Phase III Study, designed to evaluate the efficacy and safety of valbenazine in tardive dyskinesia. Detailed results from this trial will be presented at a future medical conference. With positive data in hand, a marketing authorization with the Ministry of Health and Welfare is planned for 2021 in Japan. In addition, MTPC submitted filings for marketing authorization in South Korea, Thailand, Singapore, Indonesia, and Malaysia in 2020.
In February 2021, the Company notified Voyager Therapeutics, Inc. (Voyager) of the Company’s termination of the NBIb-1817 (VY-AADC) development program in Parkinson’s disease (the Program). The Company will work to transfer the rights to the Program to Voyager by August 2, 2021.
GAAP and Non-GAAP expense guidance range reflects increased investment in:
R&D expense including meaningful investments in collaboration programs (specifically with Idorsia, Xenon and Takeda) and the planned initiation of eight mid-to-late-stage clinical studies
INGREZZA and ONGENTYS marketing costs
GAAP-only guidance includes approximately $125 million of share-based compensation. GAAP-only guidance does not include any potential milestones or in-process research and development costs associated with current collaborations or future business development activities.
2021 Expected Milestones and Key Activities

Program

Indication

2021 Milestones / Key Activities

Valbenazine

Chorea in Huntington’s Disease

Phase III Top-Line Data Expected in Q4 2021

Tardive Dyskinesia

Marketing Authorization with Ministry of Health and Welfare in Japan

Neurological Indication

Initiate Phase III Registrational Study

Psychiatric Indication

Initiate Registrational Study

Crinecerfont

Congenital Adrenal Hyperplasia (Adult)

Continue Phase III Registrational Study Enrollment

Congenital Adrenal Hyperplasia (Pediatric)

Initiate Phase III Registrational Study

NBI-1065844

Negative Symptoms of Schizophrenia

Phase II Top-Line Data Expected in Q1 2021

NBI-1065845

Treatment Resistant Depression

Initiate Phase II

NBI-1065846

Anhedonia in Depression

Initiate Phase II

NBI-827104

Rare Pediatric Epilepsy: CSWS

Continue Phase II Enrollment

Neurological Indication

Initiate Phase II

NBI-921352

Focal Onset Seizure in Adults

Initiate Phase II

Rare Pediatric Epilepsy: SCN8A-DEE

Initiate Phase II

Conference Call and Webcast Today at 4:30 PM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 800-895-3361 (US) or 785-424-1062 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

On February 4, 2021 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Quest Diagnostics, FEB 4, 2021, View Source [SID1234574669]).

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"In a year dominated by the pandemic, Quest brought critical COVID-19 testing to our country, and delivered record revenues, earnings and cash from operations for the fourth quarter and full year 2020," said Steve Rusckowski, Chairman, CEO and President. "Declines in our base business recovered rapidly throughout the summer and fall; however the recovery stalled at the end of November and into December due to the surge in COVID-19 infections across the country. Continued high demand for COVID-19 testing drove our performance through the second half of the year.

"In light of the company’s strong financial position, we have increased our dividend and our share repurchase authorization while maintaining flexibility to pursue our M&A strategy," Mr. Rusckowski continued. "Given the ongoing uncertainty regarding the trajectory of the virus and its impact on COVID-19 testing trends as well as further recovery of our base business, we are providing an outlook for the first six months of 2021. We expect to provide additional updates as the year progresses."

Mr. Rusckowski continued: "The pandemic has tested our nearly 50,000 employees and they have responded as heroes, by developing COVID-19 tests, building test capacity, innovating new testing models with our retail partners, transporting specimens, delivering results and of course, supporting our customers. I’m proud of what we have achieved and optimistic about what we can accomplish in 2021."

For further details impacting the year-over-year comparisons related to operating income, operating income as a percentage of net revenues, income from continuing operations attributable to Quest Diagnostics, and diluted EPS, see note 2 of the financial tables attached below.

The sum of reported and adjusted diluted EPS for the four quarters of 2020 did not equal the total for the year ended December 31, 2020 due to quarterly fluctuations in the company’s earnings and weighted average common shares outstanding throughout the year as a result of the impact of COVID-19 and the temporary suspension of repurchases under the company’s share repurchase program.

Note on Non-GAAP Financial Measures

As used in this press release the term "reported" refers to measures under accounting principles generally accepted in the United States ("GAAP"). The term "adjusted" refers to non-GAAP operating performance measures that exclude special items such as restructuring and integration charges, certain financial impacts resulting from the COVID-19 pandemic, amortization expense, excess tax benefits ("ETB") associated with stock-based compensation, a gain on remeasurement of an equity interest, costs associated with donations, contributions and other financial support through Quest for Health Equity, the company’s recently announced initiative with the Quest Diagnostics Foundation to reduce health disparities in underserved communities, the gain on the sale and leaseback of a property, and other items.

Non-GAAP adjusted measures are presented because management believes those measures are useful adjuncts to GAAP results. Non-GAAP adjusted measures should not be considered as an alternative to the corresponding measures determined under GAAP. Management may use these non-GAAP measures to evaluate our performance period over period and relative to competitors, to analyze the underlying trends in our business, to establish operational budgets and forecasts and for incentive compensation purposes. We believe that these non-GAAP measures are useful to investors and analysts to evaluate our performance period over period and relative to competitors, as well as to analyze the underlying trends in our business and to assess our performance. The additional tables attached below include reconciliations of non-GAAP adjusted measures to GAAP measures.

Conference Call Information

Quest Diagnostics will hold its quarterly conference call to discuss financial results beginning at 8:30 a.m. Eastern Time today. The conference call can be accessed by dialing 888-455-0391 within the U.S. and Canada, or 773-756-0467 internationally, passcode: 7895081; or via live webcast on the company’s website at www.QuestDiagnostics.com/investor. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 866-461-2735 for domestic callers or 203-369-1352 for international callers. No passcode is required. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on February 4, 2021 until midnight Eastern Time on February 18, 2021. Anyone listening to the call is encouraged to read the company’s periodic reports, on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.

On February 4, 2021 AstraZeneca reported that British drugmaker and researchers from the UCL Division of Infection & Immunity will collaborate on two projects which will aim to contribute to the development of new cancer treatments (Press release, AstraZeneca, FEB 4, 2021, View Source [SID1234574705]).

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The new research collaborations will investigate immune checkpoints – key biochemical pathways that regulate the body’s immune responses.

Although immune checkpoints help to keep the body’s immune response at normal levels by not harming healthy cells, they can also block specialist immune cells from attacking and destroying cancer cells.

Over the last decade, the emergence of checkpoint inhibitor drugs have revolutionised cancer treatment and demonstrated benefit in clinical results for patients with solid tumours.

Meanwhile, the two AstraZeneca/UCL projects will focus on increasing understanding of immune checkpoint mechanisms and how to manipulate them.

The ultimate aim of the projects will to be to aid the development of new immunotherapy approaches.

AstraZeneca will provide a number of compounds for the projects, while UCL will use unique preclinical models and an array of molecular and cell biology techniques to study these pathways.

"These collaborations with AstraZeneca will bring together some of the very best minds in immuno-oncology," said Dr Kathryn Walsh, executive director, office of the Vice-Provost (Enterprise), UCL.

"Working together, experts from both institutions will push the boundaries of our understanding of the role of the body’s immune system. In the future, these insights will play a valuable role in how we will be able to develop new treatments to help patients with solid tumours," she added.

On February 4, 2021 West Pharmaceutical Services, Inc. (NYSE: WST), a global leader in innovative solutions for injectable drug administration, reported that it will release fourth-quarter and full-year 2020 financial results before the market opens on Thursday, February 18, 2021, and will follow with a conference call to discuss the results and business expectations at 9:00 a.m. Eastern Time (Press release, West Pharmaceutical Services, FEB 4, 2021, View Source [SID1234574615]). To participate on the call, please dial 877-930-8295 (U.S.) or 253-336-8738 (International). The conference ID is 4095168.

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A live broadcast of the conference call will be available at the Company’s website, www.westpharma.com, in the "Investors" section. Management will refer to a slide presentation during the call, which will be made available on the day of the call. To view the presentation, select "Presentations" in the "Investors" section of the Company’s website.

An online archive of the broadcast will be available at the site three hours after the live call and will be available through Thursday, February 25, 2021, by dialing 855-859-2056 (U.S.) or 404-537-3406 (International). The conference ID is 4095168.