On January 14, 2020 John Theurer Cancer Center (JTCC) at Hackensack University Medical Center in New Jersey reported that were part of the 15-site international ZUMA-2 trial, a phase II study of CAR T-cell therapy in a heavily pretreated patient population who had exhausted all standard treatment options for mantle cell lymphoma (MCL) (Press release, John Theurer Cancer Center, JAN 14, 2020, View Source [SID1234553198]).

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After a single infusion of KTE-X19, 93% of patients with relapsed/refractory MCL responded and 67% achieved a complete response (CR) rate (i.e., no remaining disease). These unprecedented results were presented at the 2019 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, held in December in Orlando, Florida — the world’s leading conference for hematologic cancers and blood disorders.

With CAR T-cell therapy, white blood cells called T cells are removed from the patient, modified in the laboratory to make them recognize a protein (CD19) on lymphoma cells, then expanded to much larger numbers and returned to the patient intravenously, where they can expand further to detect and destroy cancer cells anywhere in the body. Two forms of CAR T-cell therapy — aci-cell (Yescarta) and tisa-cell (Kymriah) — are currently approved by the U.S. Food and Drug Administration to treat recurrent and persistent B-cell lymphoma in adults, as well as acute lymphoblastic leukemia in children and young adults up to age 25. However, CAR T-cell therapy is not yet approved to treat MCL, an aggressive type of non-Hodgkin lymphoma which becomes resistant to therapy over time and is associated with very poor outcomes.

The ZUMA-2 study included MCL patients who had failed to respond to both chemoimmunotherapy and BTK inhibitors, such as ibrutinib or acalabrutinib. The 93% response rate and 67% CR rate were observed regardless of the number of prior therapies or extent of disease. Interestingly, 40% of patients who initially had a partial response or stable disease achieved a CR within 3 to 12 months, showing the effectiveness of a "living drug" that continues to work over time.

Responses were also durable, with one-year estimates of progression-free survival (the time before the cancer continued to grow) and overall survival being 71% and 86%, respectively. At the time of analysis, the median duration of response had not yet been reached, and for those who achieved a CR, 78% of patients remained in remission. For the first 28 patients who were treated with axi-cel and had the longest follow-up time (median 27 months), 43% of the responders remained in remission — a rate totally unprecedented for this population.

"This will be a true game-changer in the management of patients with mantle cell lymphoma," said Andre Goy, M.D., M.S., Chairman and Director of JTCC, Lymphoma Division Chief, and a renowned lymphoma expert who led JTCC’s participation in the ZUMA-2 study.

Side effects were generally manageable and as expected with CAR T-cell therapy, including low blood cell counts, cytokine release syndrome (a release of inflammatory proteins associated with the immune response), and nervous system side effects that were all reversible.

On January 14, 2020 John Theurer Cancer Center (JTCC) at Hackensack University Medical Center in New Jersey are reported the first to report poor adherence to genomic profiling guidelines for four biomarkers of metastatic colorectal cancer used to predict response to therapy, choose the most effective treatment, and improve outcomes — showing that only 40% of patients had their tumors tested (Press release, John Theurer Cancer Center, JAN 14, 2020, View Source [SID1234553197]). The study was published online in the December 6, 2019 issue of JCO Precision Oncology, a publication of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).

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"Despite guidelines that recommend testing for four common biomarkers in patients with metastatic colorectal cancer, overall testing rates were suboptimal," noted hematologist-oncologist Stuart L. Goldberg, M.D., Chief, Division of Outcomes and Value-Based Care at JTCC, Associate Professor of Medicine at Seton Hall School of Medicine, and senior author of the study. "We expected there to be better awareness of the guidelines with time and increased testing rates over the years, but that did not turn out to be the case."

Genomic testing is recognized in national guidelines as essential to guide appropriate therapy selection in metastatic colorectal cancer. The presence or absence of mutations in genes such as RAS (KRAS and NRAS) and BRAF and genetic changes called microsatellite instability (MSI) can predict how well a patient will respond to metastatic colorectal cancer therapies such as cetuximab, panitumumab, and immunotherapy with pembrolizumab. Starting in 2009 and over the ensuing decade, ASCO (Free ASCO Whitepaper) and the National Comprehensive Cancer Network (NCCN) began publishing guidelines recommending routine genomic testing for KRAS, NRAS, BRAF, and MSI status in patients with metastatic colorectal cancer.

Studies published earlier have reported that adherence to testing guidelines was suboptimal, but current testing rates have not been assessed. Investigators at John Theurer Cancer Center retrospectively reviewed the COTA Real World Data database to identify patients with metastatic colorectal cancer diagnosed between 2013 and 2017. Among the 1,497 patients identified, testing for biomarkers according to the guidelines for RAS, BRAF, and MSI were 41%, 43%, and 51%, respectively. RAS and BRAF testing were more likely to be done in academic medical centers compared with community hospitals. Of 177 patients who received cetuximab or panitumumab — drugs indicated for the treatment of people with normal RAS genes — only 28% had undergone tumor testing for RAS status.

"When biomarker testing is underutilized, it places patients at risk of receiving ineffective therapies and may delay or prevent them from receiving appropriate therapeutic options," explained lead author Martin E. Gutierrez, M.D., Chief of Thoracic Oncology and Director of Drug Discovery and the Phase I Unit at JTCC. "Adherence to guideline-recommended biomarker testing would potentially reduce exposure to expensive and ineffective therapies, resulting in more rationale care and improved patient outcomes."

The investigators noted that possible barriers to biomarker testing may include availability of tumor tissue, turnaround time, physician knowledge, cost and insurance issues, patient preferences, and patient eligibility for therapy based on overall health.

On January 14, 2020 AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, reported that it will be featured as a presenting company at the annual Biotech Showcase conference taking place January 13-15 at the Hilton San Francisco Union Square in San Francisco, California (Press release, AIVITA Biomedical, JAN 14, 2020, View Source [SID1234553196]). Dr. Hans S. Keirstead, AIVITA’s Chairman and CEO, will present at the following time and location:

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Date: Tuesday, January 14, 2019
Time: 10:00 AM
Track: Franciscan D (Ballroom Level)

Biotech Showcase is an annual investor and networking conference that provides private and public biotechnology and life science companies with an opportunity to present and meet with investors and pharmaceutical executives.

Dr. Keirstead will be presenting details on AIVITA’s platform cancer technology, a next generation immunotherapy targeting the tumor-initiating stem cells, with strong clinical data evidencing 72% survival at 2-years, and 54% survival at 5-years post-treatment. AIVITA is currently conducting three clinical studies investigating its platform immunotherapy in patients with ovarian cancer, glioblastoma and melanoma.

CLINICAL TRIAL DETAIL

OVARIAN CANCER

AIVITA’s ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous tumor-initiating cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA’s AVOVA-1 trial patients can visit: www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA’s glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the tumor-initiating cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA’s melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with AIVITA’s tumor-initiating cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.

On January 14, 2020 ArcherDX, Inc., reported it has received Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA) for its Personalized Cancer Monitoring (PCM) technology, a bespoke, minimally-invasive and highly-sensitive product intended for early-stage cancer treatment monitoring and recurrence surveillance (Press release, ArcherDX, JAN 14, 2020, View Source [SID1234553195]). ArcherDX’s technology enables healthcare providers across community and academic care settings access to genomic information in their laboratory, saving critical time and allowing world-class, cost-effective care locally.

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The goal of the Breakthrough Devices Program is to provide patients and health care providers with timely access to medical devices by speeding up their development, assessment, and review, while preserving standards, consistent with the FDA’s mission to protect and promote public health.

The program will provide ArcherDX with enhanced communication with the FDA regarding technology validation and clinical trial protocols and could expedite the review process.

Jason Myers, ArcherDX Chief Executive Officer and co-founder said, "Fewer than 1% of cancer patients receive any genomic profiling for the monitoring of their cancer. Millions of individuals who undergo cancer treatment and the millions more who survive and achieve remission, need a sensitive, personalized means of detecting relapse earlier. We believe our bespoke product can improve both therapy monitoring and recurrence surveillance and given the substantial need, we look forward to additional collaborative interaction with regulators to deliver our PCM product to patients as soon as possible."

As part of an on-going collaboration, TRACERx1 investigators, led by Professor Charles Swanton, Group Leader, UCL and the Francis Crick Institute, and Dr. Christopher Abbosh, Principal Clinical Fellow, UCL, are utilizing ArcherDX’s technology to detect low-volume minimal residual disease at high levels of sensitivity to help achieve TRACERx’s goal of a more personalized approach to developing cancer treatments.

The PCM Breakthrough designation follows Breakthrough Device Designation for ArcherDX’s STRATAFIDE which, upon approval, would be the first pan-solid tumor device employing genomic sequencing testing technology to accept both tissue and blood that can be used in local hospital or regional reference laboratories.

On January 14, 2020 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported that the Company’s Board of Directors has approved a reverse stock split of its shares of common stock at a ratio of 1-for-55 (Press release, Phio Pharmaceuticals, JAN 14, 2020, View Source [SID1234553194]). The reverse stock split will become effective at 12:01 a.m. Eastern Time on January 15, 2020 and the Company’s common stock will open for trading on The Nasdaq Capital Market on a post-split basis on January 15, 2020 under the Company’s existing trading symbol, "PHIO." At such time, the Company’s common stock will also commence trading with a new CUSIP number, 71880W303.

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At the Company’s Special Stockholder Meeting held on January 10, 2020, Phio’s stockholders approved a reverse stock split within a range of 1-for-2 and 1-for-70. Thereafter, the Board of Directors determined to fix the ratio for the reverse stock split at 1-for-55.

The reverse stock split is being implemented to increase the per share trading price of the Company’s common stock for the purpose of ensuring a share price high enough to comply with the minimum $1.00 bid price requirement for continued listing on The Nasdaq Capital Market. At the effective time of the reverse stock split, every fifty-five (55) shares of Phio common stock issued and outstanding will be combined into one (1) share of common stock issued and outstanding, with no change to the par value of $0.0001 per share. This will reduce the Company’s outstanding common stock from approximately 36.8 million shares to approximately 0.67 million shares. No fractional shares of common stock will be issued as a result of the reverse stock split and instead holders of Phio common stock will receive a cash payment in lieu of fractional shares to which they would otherwise be entitled. The shares underlying the Company’s outstanding equity awards and warrants will also be adjusted accordingly.

The Company has retained its transfer agent, Computershare Trust Company, N.A. ("Computershare"), to act as its exchange agent for the reverse split. Shareholders with shares held in certificate form will receive from Computershare instructions regarding the exchange of their certificates. Shareholders that hold shares in book-entry form or hold their shares in brokerage accounts are not required to take any action and will see the impact of the reverse stock split reflected in their accounts, subject to brokers’ particular processes. Beneficial holders of Phio common stock are encouraged to contact their bank, broker, custodian or other nominee with questions regarding procedures for processing the reverse stock split.

Additional information regarding the reverse stock split is available in the definitive proxy statement filed with the U.S. Securities and Exchange Commission on November 22, 2019 by the Company.