On April 19, 2021 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported that in March, the first patient was infused in the CD19-Specific Rapid Personalized Manufacturing ("RPM") CAR-T Phase I Trial, being conducted by Eden BioCell, its joint venture with TriArm Therapeutics (Press release, Ziopharm, APR 19, 2021, View Source [SID1234578181]).

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The patient was treated at National Taiwan University Hospital under the direction of lead investigator, Dr. Shang-Ju Wu.

The patient’s T cells, collected from the patient via apheresis, were genetically engineered utilizing the Company’s non-viral Sleeping Beauty transposon transposase system and infused two days after gene transfer.

"We are excited to be conducting this important trial for this experimental treatment," said Dr. Jay Zhang, Chief Executive Officer of TriArm Therapeutics.

The Company and Eden BioCell will provide updates regarding the experimental treatment and other patient data in the second half of the year at appropriate venue(s), including scientific conferences, publications and / or bespoke events that the Company may convene.

About the Trial "Infusion of CD19-Specific Chimeric Antigen Receptor T-cells Produced by Rapid Personalized Manufacture for Patients with Advanced Lymphoid Malignancies"
This is a single center phase I, open-label dose-escalation trial, for patients with relapsed CD19+ leukemias and lymphomas. Up to 24 patients will be enrolled in this trial. The primary endpoint of the trial is to evaluate the safety and tolerability of autologous CD19-specific T cells manufactured using the RPM process.

On April 19, 2021 Oncotelic Therapeutics, Inc. ("Oncotelic" or the "Company") (OTCQB:OTLC) reported a presentation at AACR (Free AACR Whitepaper)-2021 (American Association for Cancer Research/ Annual Meeting) entitled: Combination therapy of anti-sense oligonucleotide targeting TGF-β2 (TASO) and IL-2 (Proleukin) has anti-cancer effect in solid cancer (Press release, Oncotelic, APR 19, 2021, View Source [SID1234578197]).

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TGF-β (transforming growth factor-beta) is an essential cytokine for tumor proliferation and metastasis. The expression of TGF-Β correlates with malignancy of various cancers and involves immunosuppression and angiogenesis of a tumor. IL-2 is a major cytokine to proliferate T cells and NK cells which are major players of cancer immunity. However, the toxicity of high dose IL-2 limits its use in cancer therapy. Combination treatment of TGF-β inhibitor and IL-2 would have an anti-tumor effect by immune cells through diminishing immunosuppression by TGF-β and enforcement of immune cells by IL-2. Trabedersen is an anti-sense oligonucleotide targeting human TGF-β mRNA. It is shown that Trabedersen is well tolerable in cancer patients and effective reagent to treat pancreatic cancer, melanoma, and glioblastoma. Proleukin is the only approved IL-2 reagent to treat Renal cell carcinoma and Melanoma.

Trabedersen and Proleukin activated human PBMC (Peripheral Blood Mononuclear Cell) are treated to several solid cancer cell lines, such as Breast cancer, Pancreatic cancer, Melanoma, Lung cancer, and Colon cancer to see the cytotoxicity effect of combination therapy of Trabedersen and Proleukin. NSG mouse (NOD Scid Gamma mouse, NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) which are humanized by human PBMC engraftment and the tumor growth of Melanoma and TNBC (Triple Negative Breast Cancer cell) cell line are monitored.

The combination treatment of Trabedersen and low dose Proleukin decreased cancer cell viability in vitro experiment in solid cancer cell lines. Melanoma and TNBC tumor growth was delayed in humanized NSG mouse model by Trabedersen and low dose IL-2 combination therapy and tumor growth delay was statistically significant to Trabedersen alone or IL-2 alone group. Tumor infiltrating lymphocyte population was increased in Trabedersen treated group and FoxP3+ regulatory T cell population in blood and tumor microenvironment was decreased by treatment of Trabedersen.

TGF-β inhibitor (Trabedersen) and low dose IL-2 (Proleukin) combination treatment is expected to be an effective regimen in solid cancer treatment than individual treatment by alteration of tumor environment. Modulation of the dose of Proleukin expects to help reduce the toxicity of IL-2 and increase the anti-cancer effect by combination with Trabedersen.

Oncotelic has previously announced the regulatory approval from the Ministry of Food and Drug Safety of Korea for the phase 1b clinical trial of a patented OT-101/IL-2 combination. This phase 1b clinical trial will confirm the safety and effectiveness of OT-101/IL-2 in solid cancer patients in cooperation with the UK global pharmaceutical company Clinigen Group. The study will be conducted together with Autotelic BIO- a partner of Mateon on OT-101/IL-2 combination.

OT-101 has received orphan drug designation for glioblastoma, melanoma, and pancreatic cancer. Furthermore, FDA recently granted Rare Pediatric Designation for OT-101 against diffuse intrinsic pontine glioma (DIPG). OT-101 is also effective against coronavirus including COVID-19 and being deployed against the COVID-19 epidemic.

OT-101 has demonstrated robust efficacy against pancreatic cancer, glioblastoma, and melanoma during phase 2 clinical trials. The demonstration that OT-101 will synergize with IL-2 further demonstrate its utility as adjunct to other immunotherapies. Interleukin-2 (IL-2, Aldesleukin, PROLEUKIN) Immunotherapy is cancer treatment that stimulates the body’s immune system to fight cancer, such as melanoma.

On April 19, 2021 Perrigo Company plc (NYSE; TASE: PRGO), a leading global provider of Quality, Affordable Self-Care Products, reported that its Board of Directors declared a quarterly dividend of $0.24 per share, payable on June 22, 2021 to shareholders of record on June 4, 2021 (Press release, Perrigo Company, APR 19, 2021, View Source [SID1234578182]).

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On April 19, 2021 NorthShore University HealthSystem (NorthShore) and Sema4, a patient-centered health intelligence company, reported the availability of a system-wide data-driven genomics program that utilizes predictive analytics to help clinicians and patients prevent, detect, and treat diseases at an early stage, when they are most treatable (Press release, NorthShore University HealthSystem, APR 19, 2021, View Source [SID1234578198]). As part of the program, NorthShore clinicians and patients will have access to Sema4’s industry-leading, information-rich genomic solutions for hereditary cancer, cardiovascular diseases, pharmacogenomics, and rare diseases.

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"Unlocking the power of genomics in patient care requires us to make information available in a way that clinicians and patients can easily act upon over time," said Peter Hulick, MD, director of the Mark R. Neaman Center for Personalized Medicine at NorthShore. "The partnership between NorthShore and Sema4 will allow us to get even better at delivering on the promise of genomic insights to prevent, detect, and manage diseases through one of the largest and most comprehensive personalized medicine programs in the country."

Advanced Clinical Offerings

By combining clinical information with genomic analysis, Sema4’s technology platform structures and evaluates data sets to draw insights on how genetic variants will impact patients’ chances of developing disease and to determine the most appropriate treatment options, helping their doctors administer more personalized, holistic care plans. NorthShore will have the opportunity to benefit from disease network models developed by Sema4. For example, disease models for cancers will provide more comprehensive views of cancer patients, their tumor progression, and how best to diagnose or treat them.

Amidst the pandemic, as evidence reflects people are delaying preventative care, these more personalized genomic insights are particularly relevant in helping patients and physicians make informed decisions about prioritizing care.

"By narrowing the significant gap between available biomedical data and health and disease information, we are enabling NorthShore clinicians to make real-time use of the latest insights that allow for highly informed decisions at the point-of-care and improved health outcomes," said Eric Schadt, PhD, Founder and Chief Executive Officer of Sema4. "NorthShore has done what few health systems in the country have – brought the potential of genomics from the research labs to the front lines of care. Through the implementation of Sema4’s innovative health intelligence platform in a clinical-care setting, we are together advancing industry standards."

Improved Patient and Clinician Experiences

In addition to guiding clinicians, the program makes it easier for NorthShore patients to understand the implications of genomic findings. Novel digital tools and resources will uncover personalized insights that shape the care of multiple conditions throughout the lifetimes of patients. Patients also have an opportunity to consent to share de-identified data to improve research programs focused on expanding the understanding of genomic-guided care.

The strategic alliance between NorthShore and Sema4 will significantly expand on NorthShore’s existing clinical genomics offerings, which were already the largest in routine primary care in the U.S. More than 1,000 physicians and approximately 300,000 NorthShore patients will have access to Sema4’s offerings.

"Precision medicine represents the next major transformation in health care, allowing us to predict and prevent disease, to identify the right treatment at the right time, and to empower patients and clinicians to make better choices about health," said J.P. Gallagher, President and Chief Executive Officer of NorthShore. "This advanced genomics and health intelligence initiative with Sema4 builds on our long history of pioneering technology and genomic advancements to improve patient care – and aligns with our strong commitment to improving the accessibility of our best-in-class care to address documented disparities in health care services, disease prevalence, and outcomes."

Addressing Disparities in Care

Due to NorthShore’s intentional focus on increasing access to advanced clinical programs, the program is launching in conjunction with an investment to engage culturally diverse communities. In addition to understanding and addressing barriers to access, NorthShore, via a gift from the Swedish Hospital Foundation, will subsidize genetic testing costs for patients who do not have the financial resources to pay for testing.

Earlier this month, NorthShore began offering the program to patients of Swedish Hospital, a State of Illinois Safety Net Hospital located in a federally designated Medically Underserved Area and a member of the NorthShore family. Through the program, the system expects to gain insights about the views of diverse patient populations towards personalized medicine and barriers to receiving genetic information and testing.

On April 19, 2021 Oncocyte Corporation (NASDAQ: OCX), a molecular diagnostics company with a mission to provide actionable answers at critical decision points across the cancer care continuum, reported that it has completed its acquisition of Chronix Biomedical, a privately held molecular diagnostics company developing blood tests for use in cancer treatment and organ transplants (Press release, Oncocyte, APR 19, 2021, View Source [SID1234578183]). The acquisition includes the intellectual property and technology for Chronix’s TheraSure-Copy Number Instability (CNI) Monitor test for immune therapy monitoring. The Chronix CNI test is a patented, novel blood-based test that is differentiated from current methods because it requires no upfront tumor tissue sample. With the acquisition, Oncocyte also gains the organ transplant technology and the associated patent portfolio developed by Chronix.

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"We believe the acquisition of Chronix will provide Oncocyte a distinct competitive advantage as the first and only company to potentially offer a continuum of tests, from selecting patients for immune therapy treatment, to monitoring the effectiveness of the treatment," said Ron Andrews, President and Chief Executive Officer of Oncocyte. "Once available, we believe the CNI test will allow physicians to begin monitoring patients for treatment efficacy more quickly given the CNI test requires only a blood sample. In contrast, getting a result from the emerging tests for monitoring can take significantly longer as these tests start with the time consuming and costly genetic sequencing of a patient’s tissue sample. According to our voice of customer work, many later stage cancer biopsies do not provide enough tissue available to complete the analysis. These challenges are particularly amplified in lung cancer, where most later stage patients have very limited tissue from a fine needle biopsy sample and thus, quite often, have too little sample to perform tissue-based testing, including personalized MRD testing. Even in cases where sufficient tissue is available, preserving precious sample to enable other tests will be an added benefit of Oncocyte’s fully blood-based approach. Assuming the data published to date continues to be validated in future studies, the CNI test will be able to deliver timely results on progression using blood only, with no need for tissue biopsies, at the second treatment cycle. This time frame will deliver valuable information to physicians much faster than currently seen with imaging or tissue-informed technologies."

The addition of the CNI test to Oncocyte’s portfolio of diagnostic products marks the Company’s entry into blood-based immune-therapy monitoring, a market that is estimated at $3 billion in the United States alone. Ownership of Chronix’s intellectual property portfolio helps establish the foundation for Oncocyte to potentially build additional applications to address an estimated $6 billion U.S. recurrence monitoring market. Further, as approximately 40%-60% of patients fail to see a sustained response to immune checkpoint inhibitors, it is estimated that by 2025, more than $60 billion a year could be misspent on treatments that may never benefit patients.

The acquisition, which includes Chronix’s lab in Germany and its EU-based development and business team, establishes a footprint for the continued development and potential commercial launch of Oncocyte’s proprietary tests in the European market. The Company expects to launch the CNI test as a pharma service in Europe from Germany by the end of the current quarter. After the tech transfer to its U.S. facilities, Oncocyte plans to launch the test for research use only in domestic immune therapy clinical trials during the fourth quarter of this year. The anticipated first indication for use will be in lung cancer, followed by expansion to other cancer types. Further, as the path to reimbursement is already established, Oncocyte plans to demonstrate the comparability of the CNI test to existing reimbursed tests.

Also included in the acquisition are tests and a patent estate for the use of digital PCR to detect transplant rejection in recipients as well as enabling the absolute amount of tumor or donor cell-free DNA, which eliminates the influence of changes in cell-free DNA caused by unrelated reasons making the technique more specific for monitoring. The Company plans to explore the use of the patented methodologies and combine with digital PCR for development of super-sensitive detection of tumor-derived DNA in blood to monitor for long-term recurrence as well as explore licensing this technology and patent estate for transplant to potential partners.

Dr. Ekkehard Schuetz, former CEO and Chief Medical Officer of Chronix and now General Manager and CMO of Oncocyte Europe said, "By becoming part of Oncocyte, we believe our CNI monitoring test, together with DetermaIO, has the potential to become a much more powerful tool for oncologists as they will have a comprehensive solution for two of the biggest challenges in immune therapy: better identifying patients who are most likely to respond to treatment, and then monitoring for treatment efficacy and the development of resistance. The combination of Oncocyte’s proprietary immune therapy selection test and their commercial experience with Chronix’s rich portfolio of tests and IP make the deal clearly a case of the sum of the parts being greater than the whole."

More about CNI and the CNI Monitor Test

Cancer modifies the normal genome of cells by accumulating mutations and variation in the number of copies of genes in the genome.
Chronix’s CNI test measures the collective burden of tumor derived copy number variation (CNV) across the genome in blood as a sensitive, specific and quantifiable measure of tumor burden, i.e., response to therapy or prognosis of cancer.
CNI is a novel approach to measuring cell-free tumor DNA in blood that does not rely upon sequencing the tumor biopsy specimen (tumor naive vs. tumor informed).
The proprietary CNI test quantitatively measures the amount of CNV present in blood that has been shed by dying tumor cells.
The CNI Monitor test monitors the change in CNI over time and is suitable for monitoring patients being treated with chemotherapy, targeted therapy or immunotherapy.
Multiple publications have demonstrated the accuracy of CNI in identifying response or resistance to therapy prior to the second or third cycle of treatment, including a study in Clinical Cancer Research, and the value to assess the prognosis of cancer published for Head & Neck Cancer.
About the Merger and Principal Transaction Terms

Under the amended merger agreement, Oncocyte delivered closing consideration and paid off assumed liabilities approximating $4.25 million in cash and $3 million of Oncocyte common stock (or approximately 591,000 shares) and agreed to pay an additional $2.5 million of liabilities by July 2022. The agreement also provides for Oncocyte to pay a revenue share on the net collected revenues for certain tests and services for specific periods, and to pay a combination of cash or Oncocyte common stock of up to $14 million if certain milestones are achieved over a ten-year timeframe, subject to offset by Oncocyte for liabilities paid in excess of $8.25 million. Oncocyte issued the shares in a private transaction and intends to register the resale of the shares. Additional information regarding the terms of the transaction will be provided in Oncocyte’s Current Report on Form 8-K expected to be filed with the Securities and Exchange Commission on or about April 19, 2021.