On January 27, 2020 Human Longevity, Inc. (HLI), an innovator in providing data-driven health intelligence and precision health to physicians and patients, reported the publication of a groundbreaking study in the journal Proceedings of the National Academy of Sciences (PNAS) (Press release, Human Longevity, JAN 27, 2020, View Source [SID1234553593]). The study titled, "Precision medicine integrating whole-genome sequencing, comprehensive metabolomics, and advanced imaging," showed that by integrating whole-genome sequencing with advanced imaging and blood metabolites, clinicians identified adults at risk for key health conditions. Data from 1190 self-referred individuals evaluated with HLI’s multi-modal precision health platform, Health Nucleus, show clinically significant findings associated with age-related chronic conditions including cancer, heart disease, diabetes, chronic liver disease, and neurological disorders — leading causes of premature mortality in adults.

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"The goal of precision medicine is to provide a path to assist physicians in achieving disease prevention and implementing accurate treatment strategies," said C. Thomas Caskey, MD, FACP, FACMG, FRSC, chief medical officer for Human Longevity, Inc., lead author of the study, and a member of the National Academy of Sciences. "Our study showed that by employing a holistic and data-driven health assessment for each individual, we are able to achieve early disease detection in adults."

Study highlights include:

Approximately 1 in 6 adult individuals (17.3%) had at least one pathogenic genetic variant, and when integrated with deep phenotyping (imaging, blood test, etc.), 1 in 9 (11.9%) had genotype and phenotype associations, supporting the clinical diagnosis of a genetic disorder.
Additional highly actionable findings in this self-referred cohort, most of which were not previously known, include:
Insulin resistance and/or impaired glucose tolerance (34.2%)
Elevated liver fat (29.2%)
Cardiac structure or function abnormalities such as valvular disorders (16.2%)
Significant calcified coronary artery plaque (calcium score > 100) (11.4%)
Elevated liver iron (9.3%)
Cardiac arrhythmias such as atrial fibrillation (6.1%)
Cardiac conduction disorders (4.8%)
Early stage tumors, most malignant (1.7%)
A lack of phenotype and genotype associations were observed in 5.8% of individuals with pathogenic genetic variants, further suggesting that the identification of pathogenic genetic variant(s) by sequencing alone is not sufficient for a definitive diagnosis, highlighting the importance of a multi-modal assessment.
Genomics and metabolomics associations revealed 5.1% of heterozygous carriers with phenotype manifestations, affecting serum metabolite levels, suggesting that some genetic carriers may not be completely asymptomatic.
"This study shows that the definition of ‘healthy’ may not be what we think it is and depends upon a comprehensive health evaluation," said J. Craig Venter, PhD, founder, Human Longevity, Inc. and a member of the National Academy of Sciences. "The data underscore Human Longevity’s innovative approach to helping clinicians with early detection and personalized treatments, potentially achieving better health outcomes for patients."

"Our traditional approach to the annual health assessment has been very superficial and will need to be replaced by data-driven measures that will be made possible as costs continue to decline for whole-genome sequencing, advanced imaging, especially MRI, and specialized blood analytics," said David Karow, MD, PhD, president and chief innovation officer, Human Longevity, Inc.

ABOUT THE STUDY

The study cohort was composed of 1190 self-referred participants who enrolled at Health Nucleus with a median age of 54 y (range 20 to 89+ y, 33.8% female, 70.6% European). A multidisciplinary team, including cardiologists, radiologists, primary care physicians, clinical geneticists, genetic counselors, and research scientists, integrated deep phenotype data with genome data for each study participant. Participants were enrolled in the study between September 2015 and March 2018.

On January 27, 2020 OncoImmune, Inc. reported that clinical data from its Phase IIa clinical trial of CD24Fc are being presented at the 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Meeting, taking place in Orlando, Florida in February (Press release, ONCOIMMUNE, JAN 27, 2020, View Source [SID1234553592]). The Phase IIa data will be presented by the study’s Principle Investigator, Dr. John Magenau of the University of Michigan’s Department of Medicine, at 11:15 am on February 21. Dr. Pan Zheng, the Chief Medical Officer of OncoImmune, Inc., will present Phase III clinical trial design in a poster session on February 19-20th.

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CD24Fc is OncoImmune’s first-in class fusion protein that selectively represses inflammation induced by tissue injury while preserving innate immune response to pathogens. The Phase IIa study is a randomized, double blind, placebo controlled, multi-center study to investigate adding CD24Fc to standard of care tacrolimus and methotrexate in acute graft-versus host disease (GVHD) prophylaxis for allogeneic hematopoietic stem cell transplantation (HCT) with matched unrelated donors in treatment of leukemia and myelodysplastic syndrome. The trial included three CD24Fc dose cohorts: 240 mg at day -1, 480 mg at day -1, and the multi-dose cohort of 480-240-240 mg at day -1, day 14 and day 28. CD24Fc has received orphan drug designation from both the US FDA and European Medical Agency (EMA) for GVHD prophylaxis.

The presentation, entitled, "Mitigating Damage Response with CD24 Fusion Protein for Prevention of Acute Graft-Versus Host Disease," compares safety and efficacy data of CD24Fc when used in combination with standard of care GVHD prophylaxis compared to placebo and historical controls. The results demonstrate that CD24Fc was safe and well tolerated in the patient population. More importantly, patients receiving CD24Fc performed significantly better than placebo and historical controls in 180 day grade III-IV GVHD-free survival, the planned primary endpoint for the Phase III trial. These data thus provided strong support for the primary endpoint and dosing regimen of the upcoming phase III clinical trial. Moreover, significantly better relapse free survival (RFS) was observed over placebo control and historical controls. Overall survival (OS) was also significantly improved when compared with a matched historical control. Furthermore, a significant, dose-dependent reduction of mucositis was observed.

"We are very excited by the data observed in the Phase IIa clinical trial. In addition, we have completed enrollment of an open label Phase II expansion study where the drug continues to perform very well with clear signs of clinical efficacy," said Dr. Pan Zheng. "HCT is a curative therapy for refractory leukemia patients but hampered by GVHD, leukemia relapse and conditioning toxicity. As suggested by our preliminary data, CD24Fc shows significant promise in all three of these outcomes and would likely be transformative for the HCT field," she continued.

On January 27, 2020 Immunomic Therapeutics, Inc. reported that it will present data on its investigational nucleic acid platform, UNITE (UNiversal Intracellular Targeted Expression), that elicits potent immune responses and inhibits tumor growth when used with its investigational vaccine, ITI-4000, in mice (Press release, Immunomic Therapeutics, JAN 27, 2020, View Source [SID1234553591]). In preclinical studies, ITI-4000, a DNA vaccine targeting EBNA1, demonstrated robust activation of anti-tumor CD4 and CD8 T cells in vivo and promoted tumor infiltration with activated TNFα-producing CD8 T cells. Intratumoral IL-12-producing dendritic cells and iNOS-producing macrophages were also induced by the vaccine. This data will be presented at the SITC (Free SITC Whitepaper)-ASCO Clinical Immuno-Oncology Symposium in Orlando, Florida.

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"ITI-4000 is an innovative and novel approach to an EBV-tumor vaccine," says Rich Ambinder, M.D., Ph.D., Director of the Division of Hematologic Malignancies and Professor of Oncology at Johns Hopkins, Immunomic’s Scientific Advisory Board member, and expert in viral-driven malignancies.

Current treatment strategies for nasopharyngeal carcinoma are limited to radiation and chemotherapy, demonstrating a need for new, targeted therapies. Most nasopharyngeal carcinomas express Epstein-Barr nuclear antigen 1 (EBNA1), a sequence-specific DNA-binding protein involved in maintenance of the episomal virus genome that is required for EBV latency and associated transformation. Targeting EBNA1 allows for an immunotherapeutic approach by exploiting the potential of the immune system to recognize tumor cells through their expression of this viral antigen. We designed a DNA vaccine encoding EBNA1 using the UNITE platform (ITI-4000). The UNITE platform is based in part on lysosomal targeting technology which results in enhanced antigen presentation and a balanced T cell response. We report that the ITI-4000 induced IFNγ- and TNFα-producing effector memory CD4 T and CD8 T cells, with complete rejection of EBNA1-expressing tumors observed in 50% of mice. Mice rejecting tumors were protected from rechallenge with CT26-EBNA1, demonstrating that antigen-specific memory was induced in these animals. These pre-clinical data suggest that ITI-4000 has the potential to be used as an immunotherapeutic agent against EBV-associated cancers.

Poster Session B

Poster Title: The Effect of Lysosomal-associated membrane protein-1-targeting of Epstein–Barr virus nuclear antigen 1 (EBNA1) elicits potent immune responses and inhibits tumor growth in preclinical studies

Date and Time: 02/07/2020, 11:30 AM – 1:00 PM; 02/07/2020, 6:00 PM – 7:00 PM

Abstract ID: #74

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, is thought to work by encoding the Lysosomal Associated Membrane Protein, an endogenous protein in humans. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release, and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy, and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

On January 27, 2020 GT Medical Technologies, Inc., a company dedicated to improving the lives of patients with brain tumors, reported that the U.S. Food and Drug Administration (FDA) has cleared an expanded indication for GammaTile Therapy (Press release, GT Medical Technologies, JAN 27, 2020, View Source [SID1234553566]). Patients with newly diagnosed malignant brain tumors are now eligible to receive the FDA-cleared surgically targeted radiation therapy (STaRT).

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"We are pleased to offer GammaTile Therapy to patients who are newly diagnosed with malignant brain tumors, in addition to patients with recurrent brain tumors," said Matthew Likens, president and CEO of GT Medical Technologies. "This is a significant step forward that expands our ability to improve the lives of patients with brain tumors. Patients receiving GammaTile Therapy immediately after the removal of a brain tumor will have the peace of mind that they are accelerating their radiation treatment and targeting residual tumor cells where treatment is most needed to help prevent recurrence."

Approximately 700,000 Americans are living with some type of brain tumor each year.1 Despite the efforts of the most skilled brain tumor specialists throughout the world, outcomes for patients with brain tumors have improved very little over the past 30 years. During this time, there have been only four FDA-approved drugs and two FDA-cleared devices available to patients and physicians for the treatment of brain tumors. GammaTile Therapy, which became available to patients with recurrent brain tumors in January 2019, is the most recent treatment cleared or approved by the FDA for the treatment of brain tumors.

GammaTile Therapy is the only radiation therapy specifically designed for use in the brain and offers advantages for patients undergoing surgery for brain tumors. GammaTile begins targeting residual tumor cells immediately at the time of tumor removal surgery, rather than waiting several weeks for surgical wound healing before beginning treatment. GammaTile protects healthy brain tissue while delivering a targeted dose to any remaining tumor cells. The unique design also limits side effects typically associated with radiation therapy, including hair loss. Additionally, the burden of radiation treatment is reduced for patients treated with GammaTile Therapy. These patients receive their course of radiation while going about their daily lives, requiring no additional trips to the hospital or clinic for radiation therapy.

In a clinical study, GammaTile Therapy gave the average patient approximately ten extra months without a local recurrence with extended overall survival.2 Clark C. Chen, M.D., Ph.D., head of the Department of Neurosurgery at the University of Minnesota Medical School, presented data from his first patients treated with GammaTile at the 2019 Society of Neuro-Oncology (SNO) Annual Meeting. Consistent with data published in a peer-reviewed article, Chen reported that local control was achieved in approximately 90% of patients who underwent gross total resection. This impressive result was achieved without an increase in wound complications or length of hospital stay. "GammaTile Therapy is an important addition to the armamentarium of treatments available against brain cancer," said Dr. Chen. "It has the potential to improve the quality of life as well as clinical outcome. I am encouraged by the FDA’s decision to expand the indications for this therapy."

GammaTile Therapy became available to patients in January 2019 and is being used in top cancer treatment centers across the United States. Dr. Vincent DiNapoli, neurosurgeon and director of the Brain Tumor Center at The Jewish Hospital in Cincinnati, Ohio, has been treating patients with GammaTile Therapy since August 2019. "Having been encouraged by the outcomes for recurrent brain tumor patients treated with GammaTile Therapy in my practice, I am excited by the FDA’s decision to expand this technology to the many patients who can benefit from the treatment during their initial diagnosis," Dr. DiNapoli said. "This could potentially create a more meaningful impact on patient outcomes, treatment compliance, and quality of life."

About GammaTile Therapy

GammaTile Therapy is an FDA-cleared, Surgically Targeted Radiation Therapy (STaRT) that is designed to delay tumor regrowth for patients with brain tumors. Roughly the size of a postage stamp, GammaTile Therapy features a bioresorbable, conformable, 3D-collagen tile and uniform radiation source, Cesium-131, provided by Isoray, Inc. (NYSE: ISR). GammaTile Therapy is placed at the end of excision surgery to immediately target residual tumor cells while limiting the impact of radiation on healthy brain tissue.

On January 27, 2020 NeoImmuneTech, Inc., a clinical-stage T cell-focused biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for the combination of NeoImmuneTech’s Hyleukin-7 (NT-I7) and Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of patients with relapsed/refractory (R/R) advanced solid tumors (Press release, NeoImmuneTech, JAN 27, 2020, View Source [SID1234553590]). This IND clearance allows NeoImmuneTech to initiate a Phase 1b/2a basket study evaluating this combination in patients with both checkpoint inhibitor (CPI)-treated and CPI-naïve R/R advanced solid tumors.

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"As a company, we are striving to develop a safe and effective immunotherapy for patients with R/R advanced solid tumors, including those who have not been responsive to single-agent CPIs," said NgocDiep Le, MD, PhD, Chief Medical Officer and Executive Vice President of NeoImmuneTech. "This clearance marks the next step towards our goal of combining Hyleukin-7 with advanced cancer therapeutics such as pembrolizumab."

The goal of this study is to establish a recommended dosing regimen and explore the preliminary anti-tumor activity of the combination in patients with both CPI-treated and CPI-naïve R/R tumors. The results of this study will be used to further clinical development of this combination in select tumor types. The study will be led by Aung Naing, MD, FACP, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center.

"We are constantly looking for better and safer treatment options for our patients, as only a fraction of them can benefit from currently available immunotherapies," said Dr. Naing. "This agent has the potential to augment and broaden the reach of current treatments such as pembrolizumab, due to its T cell amplifying mechanism of action. We hope this study yields results helpful for our patients in dire need."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA.

About Hyleukin-7

Hyleukin-7 (NT-I7), the only clinical-stage long-acting human IL-7, is uniquely positioned to address unmet medical needs in immuno-oncology. IL-7 is a fundamental cytokine for T-cell development and for sustaining immune response to chronic antigens (as in cancer). Hyleukin-7’s favorable PK/PD and safety profiles make it an ideal combination partner for immunotherapy standard of care (SOC) such as Checkpoint Inhibitor and CAR-T therapies. Hyleukin-7 is being studied in multiple clinical trials in solid tumors, and being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.