On February 22, 2022 Code Biotherapeutics, Inc. (Code Bio), a biotechnology company pioneering targeted non-viral delivery of genetic medicines, reported a collaboration and option agreement with Takeda to leverage Code Bio’s proprietary targeted 3DNA non-viral genetic medicine delivery platform to design and develop gene therapies for rare disease indications (Press release, Takeda, FEB 22, 2022, View Source [SID1234608801]).

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Through the agreement, Takeda and Code Bio will design and develop a targeted gene therapy leveraging Code Bio’s 3DNA platform for a liver-directed rare disease program, plus conduct additional studies for central nervous system-directed rare disease programs. Takeda has the right to exercise options for an exclusive license for four programs. Under the terms of the agreement, Code Bio will receive double-digit million dollars in upfront, near-term milestone and research funding payments. Code Bio is also eligible to receive future development and commercial milestone payments plus tiered royalties with a potential total deal value over the course of the partnership of up to $2 billion if milestones for all four programs are achieved. Takeda and Code Bio will collaborate on research activities up to candidate selection. After option exercise, Takeda will assume responsibility for further development and commercialization.

"We are proud to collaborate with Takeda, a world leader in the fields of genetic medicine and rare diseases," said Code Bio Chairman, Chief Executive Officer, and Co-Founder Brian P. McVeigh. "This collaboration will further enhance our capabilities by bringing together Takeda’s expertise in gene therapy and rare diseases with our expertise in delivering genetic medicines as we drive forward on our mission to bring meaningful treatments and cures to patients suffering from serious and life-threatening genetic diseases."

Code Bio’s targeted 3DNA non-viral genetic medicine delivery platform is designed to overcome key limitations of other genetic medicine delivery approaches and fully unlock the potential of genetic medicines. The synthetic, multivalent design of the 3DNA platform enables cell specific targeting, the delivery of large genetic payloads, and the potential for re-dosability. Extensive preclinical data have demonstrated that targeted formulations are highly specific and effective at delivering genetic cargo to targeted cells and tissues with no evidence of off-target effects or safety concerns related to the platform seen.

"We aim to provide functional cures to patients with rare genetic and hematologic diseases through next-generation gene therapy programs," said Madhu Natarajan, Head of the Rare Diseases Drug Discovery Unit at Takeda. "Code Bio’s 3DNA platform will allow us to build upon the foundation we have established through our internal capabilities and external partnerships and will hopefully enable us to develop re-dosable and durable gene therapies that will be superior to current approaches."

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.

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 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated oncology therapeutics, reported that it will report fourth quarter and full year 2021 financial results on Tuesday, March 1, 2022, after the close of U.S. markets (Press release, CytomX Therapeutics, FEB 22, 2022, View Source [SID1234608798]). Following the announcement, the Company will host a conference call and webcast at 5:00 p.m. ET / 2:00 p.m. PT to discuss the results and provide a corporate update.

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Participants may access the live webcast of the conference call from the Events and Presentations page of CytomX’s website at View Source An archived replay of the webcast will be available on the Company’s website until March 8, 2022.

On February 22, 2022 Statera Biopharma, Inc. (NASDAQ: STAB) (the "Company" or "Statera Biopharma"), a leading biopharmaceutical company creating next-generation immune therapies that focus on immune restoration and homeostasis, reported unaudited, preliminary revenue and other financial results for its Fiscal Year ended December 31, 2021 (Press release, Cleveland BioLabs, FEB 22, 2022, View Source [SID1234608797]).

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Revenues for the year ended December 31, 2021 were $1,487,036 representing an increase of 100%, from $0 for the same period in 2020. The increase in revenues was due to the acquisition of ImQuest Life Sciences, Inc. and its subsidiaries (ImQuest) in June 2021 by Old Cytocom. ImQuest is a research and development company focused specifically on cancer, inflammation and infectious disease treatments. The Company reported no revenue in 2020.

Cost of revenues for the year ended December 31, 2021 was $488,314, representing an increase 100% for the same period in 2020. The increase was due to the acquisition of ImQuest. Cost of revenues as a percentage of revenue was 33% for the year ended December 31, 2021.

Operating costs for the year ended December 31, 2021 were $31,587,009, representing an increase of $20,086,619, or 191%, from $10,501,668 for the 2020. The increase in operating costs was principally due to increases in research and development expense (an increase of $6,566,403 or 125% year over year) and general and administrative expense (an increase of $14,441,905 or 276% year over year). The increase in research and development expense was the result of increased costs for the expansion in 2021 of clinical trial programs for Crohn’s disease and COVID-19. The increase in general and administrative expense reflects the costs incurred for the Merger and Old Cytocom’s acquisition of ImQuest, legal and other fees incurred to raise additional capital in 2021, increases in employee compensation, benefits and stock based compensation, and insurance expense.

Other expense for the year ended December 31, 2021 was $4,328,823, representing an increase of $2,736,630, or 172%, from other expense of $1,592,193 for the same period in 2020. The change is due to an increase of $5,458,954 in interest and other non-operating expense, offset by an increase of $2,722,324 in gains on extinguishment of debt.

Net loss attributed to the Company for the year ended December 31, 2021 was $34,892,762, representing an increase of $22,798,701, or 189%, from $12,093,861 for the same period in 2020. The increase in net loss is principally due to the increase in operating expense for the reasons described above.

Michael K. Handley, the Company’s CEO, stated, "We have taken numerous steps to further the development of our clinical stage pipeline that has us well-positioned to achieve numerous milestones in 2022. Following the recent submission to the FDA of our Phase 3 clinical trial protocol for STAT-201 in the treatment of pediatric Crohn’s Disease, we plan to use proceeds from our recent registered direct offering to initiate patient enrollment in the second quarter. Additionally, use of proceeds will include the enrollment of patients with acute COVID-19 infection in our STAT-205 study, from which we are targeting to have preliminary data this year."