On February 22, 2022 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, reported a partnership with Medidata, a Dassault Systèmes company, to evaluate the use of its Synthetic Control Arm (SCA) solution in a planned Phase 2 trial of Rhenium-186 NanoLiposome (186RNL) in recurrent glioblastoma (GBM), a type of brain cancer (Press release, Cytori Therapeutics, FEB 22, 2022, View Source [SID1234608799]).

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SCAs are especially advantageous in indications such as recurrent GBM where the standard of care control treatment is considered undesirable by some patients and physicians. The SCA enables study designs that have a higher than usual probability of assignment of prospective patients to the investigational therapy. This enhances patient enrollment and retention and potentially reduces clinical trial costs while upholding the scientific integrity of the trial.

"Medidata has developed a pioneering capability and helped to validate the use of synthetic controls in clinical drug development," said Norman LaFrance, M.D., Chief Medical Officer and SVP of Plus Therapeutics. "Although a recent advancement, the U.S. Food and Drug Administration has already agreed to recognize a Phase 3 clinical trial design incorporating a synthetic control arm in a registrational randomized control arm in recurrent GBM."

About the Synthetic Control Arm

Synthetic control arm – a type of external control – is formed by carefully selecting patients from Medidata’s extensive repository of historical clinical trials to match the baseline demographic and disease characteristics of the patients treated with the new investigational product. Case studies have shown that SCAs can effectively mimic a classic randomized control and, therefore, can be used to accurately interpret the treatment effects of an investigational product.

SCAs can help enhance the scientific validity of single-arm trials and, in certain indications, enhance randomized clinical trials to expose fewer prospective patients to control and/or ineffective or existing standard-of-care treatments that might not provide a benefit to the patient. This is done while still providing highly valid scientific evidence. These factors can influence a patient’s willingness to participate in a trial where there is a very poor prognosis and perceived inadequate standard of care.

On February 22, 2022 Sysmex Inostics, a global leader in the liquid biopsy revolution for oncology, reported that it will introduce HNSCC-SEQ, a highly sensitive Plasma-Safe-SeqS and Next Generation Sequencing (NGS) assay service for head and neck squamous cell carcinomas (HNSCC), at the annual 2022 Molecular Medicine Tri-Conference being held Monday, February 21st through Wednesday, February 23rd in San Diego, California (Press release, Sysmex Inostics, FEB 22, 2022, View Source [SID1234608815]). The assay has a turn-around-time of 7-10 days and is available to researchers and clinicians.

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Sysmex Inostics senior director of medical affairs, Dr. Fred Jones stated, "We see a huge opportunity for researchers and clinicians with our HNSCC-SEQ and HPV-SEQ assays being used in tandem to appropriately identify patients’ tumor mutational drivers." Jones added, "We know the human papillomavirus (HPV) fuels a growing percentage of head and neck cancers, but there is also an unmet need to track HNSCC tumors via circulating tumor DNA (ctDNA) that are HPV-negative – that’s where HNSCC-SEQ comes in. This HNSCC panel helps researchers and clinicians identify patients quickly and accurately for the appropriate therapy and avoiding over-treatment."

HNSCC develop from the mucosal tissue in the oral cavity, pharynx, and larynx and are the most common malignancies that arise in the head and neck regions.¹ HNSCC-SEQ was designed for HPV-negative patients and can be used to detect novel therapeutic targets and frequently occurring driver mutations for treatment response monitoring. HNSCC-SEQ delivers high-sensitivity mutation detection in HNSCC with a limit of detection of 0.05% MAF.2

HNSCC-SEQ can identify head and neck cancer mutational drivers from the genes: CDKN2A, EGFR, ERBB2, FGFR3, HRAS, KRAS, NOTCH1, PIK3CA, PTEN, and TP53, many of which are actively being pursued as therapeutic targets.3

Dr. Jones will discuss how Plasma-Safe-SeqS technology, including HNSCC-SEQ, can aid cancer drug development, treatment guidance and monitoring, in addition to post-treatment recurrence monitoring during the 2022 Molecular Medicine Tri- Conference’s C4B- Clinical Biomarkers & Companion Diagnostics presentation track in session room Indigo 206 on Tuesday, February 22nd at the Hilton San Diego Bayfront. More information about the presentation can be viewed here.

2022 Multidisciplinary Head and Neck Cancers Symposium
Additionally, Dr. Ari Rosenberg, Assistant Professor of Medicine at the University of Chicago will present findings from his study using Sysmex Inostics HPV-SEQ test at the 2022 Multidisciplinary Head and Neck Cancers Symposium being held February 24th through 26th in Phoenix, Arizona. The poster titled ‘Dynamic changes of cell-free HPV DNA in locoregional viral-associated oropharyngeal cancer receiving response-adaptive treatment’ will be presented Thursday, February 24th. More information can be viewed here.

HNSCC-SEQ and HPV-SEQ are available as a testing service provided by the Sysmex Inostics CLIA lab in Baltimore. MD.

On February 22, 2022 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the results of an additional, more finely detailed, analysis of the integration site of the cytochrome P450 2B1 gene from the augmented HEK293 cell clone that is used in PharmaCyte’s pancreatic cancer product candidate known as CypCaps (Press release, PharmaCyte Biotech, FEB 22, 2022, View Source [SID1234608833]).

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Previous studies that PharmaCyte conducted showed that the cytochrome P450 2B1 gene in the augmented HEK293 cell clone was located on human chromosome 9. As PharmaCyte announced on December 8, 2021, the flanking sequence around the site of integration was sequenced in its entirety. In this new study, PharmaCyte has been able to confirm the previously elucidated structure of the integrated transgene sequence using more data points.

These studies also set the stage for a next step analysis to determine the genetic stability of the cytochrome P450 2B1 gene at the DNA level after multiple rounds of cell growth. This new study was completed by comparing the original Research Cell Bank cells with cells from the Master Cell Bank, and the analysis confirmed that the cytochrome P450 2B1 and the surrounding sequence has remained stable with no changes detected at the DNA level.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced, inoperable pancreatic cancer, we encourage you to watch PharmaCyte’s documentary video complete with medical animations at: View Source

On February 22, 2022 Hovione, the leader in spray drying and particle engineering, reported a strategic partnership with Zerion Pharma to market and commercialize Dispersome, Zerion´s proprietary solubility enhancement technology platform (Press release, Zerion, FEB 22, 2022, View Source [SID1234609347]).

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Zerion´s innovative Dispersome technology builds on a new concept of increasing drug solubility by using natural protein-based excipients to formulate APIs into amorphous solid dispersions (ASDs) by spray drying. By combining Dispersome technology with Hovione’s unique spray drying capabilities, experience in development, scale-up and GMP manufacturing, Hovione further strengthens its leadership in amorphous solid dispersions and shows its commitment to innovative solutions to overcome one of the most prevalent challenges faced by the industry: low drug solubility.

"We are extremely pleased to have entered into this partnership with Zerion whom we recognize as a very innovative company in the field of oral drug formulations. By joining forces, Zerion and Hovione will be able to bring Dispersome to the market faster. We look forward to applying the technology in collaboration with our customers and partners in our effort to develop novel drugs with strong benefits to patients.", comments Jean-Luc Herbeaux, Hovione´s Chief Operating Officer. "Hovione will continue to pursue opportunities to enhance its offering around core areas of expertise such as particle engineering and inhalation by partnering with companies and research institutions developing innovative drug formulation technologies, like Dispersome".

Zerion´s CEO, Ole Wiborg, sees a strong synergy in the partnership and expects it to generate new pharma partnerships. "We consider Hovione the global leader in the field of producing amorphous solid dispersions by spray-drying. More importantly for us, this leadership has resulted in Hovione being, over the last 5 years, the main commercial manufacturer of novel FDA-approved drugs formulated as amorphous solid dispersions. Incorporating these competencies in our offering to the pharma industry both validates the strength of our Dispersome platform and provides us and our pharma partners immediate access to the highest quality in upscaling and commercial GMP manufacturing." says Ole Wiborg.

The announcement of this partnership follows Hovione’s communication on a further expansion with an expected investment of $170 million in assets worldwide and shows the company’s commitment to also expand its technology platforms. Hovione is investing both in new assets and innovative technologies to meet customer demand for integrated and differentiated services in drug substance manufacturing, particle engineering and most recently drug product manufacturing.

On February 22, 2022 Exact Sciences Corp. (Nasdaq: EXAS), a leader in advanced cancer diagnostics, reported that the company generated revenue of $473.8 million for the fourth quarter ended Dec. 31, 2021 and $1,767.1 million for the full year ended Dec. 31, 2021 (Press release, Exact Sciences, FEB 22, 2022, View Source [SID1234608800]).

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"Cologuard and Oncotype DX are off to a great start in 2022, with strong momentum from the fourth quarter carrying over to the new year," said Kevin Conroy, chairman and CEO of Exact Sciences. "We have a team of talented people dedicated to defeating cancer. You’ll see the results of their dedication this year as we test more patients and share evidence supporting our pipeline of innovative tests across the cancer continuum."

Fourth Quarter 2021 Financial Results

For the three-month period ended Dec. 31, 2021, as compared to the same period of 2020 (where applicable):

Total revenue was $473.8 million, an increase of 2 percent
Total revenue, excluding COVID-19 testing, increased 16 percent
Screening revenue was $277.7 million, an increase of 11 percent
Precision Oncology revenue was $149.0 million, an increase of 27 percent
COVID-19 testing revenue was $47.1 million, a decrease of 52 percent
Gross margin including amortization of acquired intangible assets was 70 percent, and non-GAAP gross margin excluding amortization of acquired intangible assets was 75 percent
Net loss was $220.6 million or $1.28 per share, compared to a net loss of $418.3 million or $2.67 per share
EBITDA was $(175.1) million and adjusted EBITDA was $(122.2) million
Cash, cash equivalents, and marketable securities were $1,030.5 million at the end of the quarter
Screening includes laboratory service revenue from Cologuard tests and revenue from Biomatrica products. Precision Oncology includes laboratory service revenue from global Oncotype products and therapy selection products, including oncomapTM and oncomapTM ExTra, formerly known as Oncotype MapTM and GEM ExTra, respectively.

2022 Outlook

The company anticipates revenue of $1,975-$2,027 million during 2022, assuming:

Screening revenue of $1,340-$1,367 million, including $40-$42 million from PreventionGenetics,
Precision Oncology revenue of $595-$610 million, and
COVID-19 testing revenue of $40-$50 million
Non-GAAP Disclosure

In addition to the company’s financial results determined in accordance with U.S. GAAP, the company provides non-GAAP measures that it determines to be useful in evaluating its operating performance. The company presents EBITDA, adjusted EBITDA, as well as non-GAAP gross margin and non-GAAP gross profit. EBITDA and adjusted EBITDA consist of net loss after adjustment for those items shown in the table below. The company defines non-GAAP gross profit and non-GAAP gross margin as GAAP gross profit and GAAP gross margin, respectively, excluding amortization of acquired intangible assets. The amortization of acquisition-related intangible assets used in the calculation of non-GAAP gross profit and non-GAAP gross margin pertain only to the amortization associated with developed technology acquired and recorded through purchase accounting transactions. The amortization of these intangible assets will recur in future periods until such intangible assets have been fully amortized. The company believes that these non-GAAP measures are useful in evaluating the company’s operating performance. The company uses this non-GAAP financial information to evaluate ongoing operations and for internal planning and forecasting purposes. Non-GAAP financial information, when taken collectively, may be helpful to investors because it provides consistency and comparability with past financial performance. However, non-GAAP financial information is presented for supplemental information purposes only, has limitations as an analytical tool and should not be considered in isolation or as a substitute for financial information presented in accordance with U.S. GAAP. For example, non-GAAP gross margin and non-GAAP gross profit exclude the amortization of acquired intangible assets although such measures include the revenue associated with the acquisitions. For a reconciliation of these non-GAAP measures to GAAP, see below "EBITDA and Adjusted EBITDA Reconciliations" and "Non-GAAP Gross Profit and Non-GAAP Gross Margin Reconciliations."

Fourth Quarter Conference Call & Webcast

Company management will host a conference call and webcast on Tuesday, February 22, 2022, at 5 p.m. ET to discuss fourth quarter and full year 2021 results. The webcast will be available at www.exactsciences.com. Domestic callers should dial 888-330-2384 and international callers should dial +1-240-789-2701. The access code for both domestic and international callers is 4437608.

An archive of the webcast will be available at www.exactsciences.com. A replay of the conference call will be available by calling 800-770-2030 domestically or +1-647-362-9199 internationally. The access code for the replay of the call is 4437608. The webcast, conference call, and replay are open to all interested parties.

About Cologuard

The Cologuard test was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. The Cologuard test is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2021) and National Comprehensive Cancer Network (2016). The Cologuard test is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use the Cologuard test if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. The Cologuard test is not a replacement for colonoscopy in high-risk patients. The Cologuard test performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. The Cologuard test performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again. Medicare and most major insurers cover Cologuard. For more information about Cologuard, visit www.cologuardtest.com. Rx only.

About Oncotype DX

The Oncotype DX portfolio of breast, colon and prostate cancer tests applies advanced genomic science to reveal the unique biology of a tumor in order to optimize cancer treatment decisions. In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS. In prostate cancer, the Oncotype DX Genomic Prostate Score test predicts disease aggressiveness and further clarifies the current and future risk of the cancer prior to treatment intervention, and the Oncotype DX AR-V7 Nucleus Detect test helps determine which patients with metastatic castration-resistant prostate cancer (mCRPC) are resistant to androgen receptor (AR)-targeted therapies. The Oncotype DX AR-V7 Nucleus Detect test is performed by Epic Sciences at its centralized, CLIA-certified laboratory in San Diego and offered exclusively by Exact Sciences. The Oncotype MAP Pan-Cancer Tissue test is a rapid, comprehensive tumor profiling panel that aids therapy selection for patients with advanced, metastatic, refractory, or recurrent cancer. With nearly 1.5 million patients tested in more than 90 countries, the Oncotype tests have redefined personalized medicine by making genomics a critical part of cancer diagnosis and treatment. To learn more about Oncotype tests, visit www.OncotypeIQ.com, www.MyBreastCancerTreatment.org or www.MyProstateCancerTreatment.org.

About Exact Sciences’ Therapy Selection Program

Exact Sciences’ therapy selection program includes two comprehensive genomic profiling (CGP) tests to help physicians identify the genomic mutations driving advanced cancers, leading patients to better care through targeted cancer treatments. The oncomap ExTra test, formerly known as GEM ExTra, detects damage in tumor genes and provides a complete biological picture of certain refractory, rare, or aggressive cancers. With an extensive panel of approximately 20,000 genes and 169 introns, the oncomap ExTra test is one of the most comprehensive genomic (DNA) and transcriptomic (RNA) panels available today. The oncomap ExTra test provides physicians, academic medical centers, and biopharma researchers with vital interpreted information to understand changes to a patient’s tumor genomic profile and recommend therapeutic treatment plans.2 For patients with advanced and metastatic cancer, the company offers the oncomap test, formerly known as Oncotype MAP, a rapid, comprehensive tumor profiling panel, which delivers results in three to five business days and allows physicians to understand a patient’s tumor profile and recommend actionable targeted therapies or clinical trials.

About PreventionGenetics

Founded in 2004 and located in Marshfield, Wisconsin, PreventionGenetics is a CLIA and ISO 15189:2012 accredited laboratory. PreventionGenetics delivers clinical genetic testing of the highest quality at fair prices with exemplary service to people around the world. PreventionGenetics has 25 PhD geneticists on staff and provides tests for nearly all clinically relevant genes including the powerful and comprehensive germline whole genome sequencing test, PGnome and whole exome sequencing test, PGxome. PreventionGenetics was acquired by Exact Sciences in December 2021.