On June 22, 2022 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for VBI-1901, a bivalent gB/pp65 immunotherapeutic vaccine candidate for the treatment of glioblastoma (GBM) (Press release, VBI Vaccines, JUN 22, 2022, View Source [SID1234616181]). In June 2021, the FDA also granted Fast Track Designation for VBI-1901 for the treatment of recurrent GBM in patients with first tumor recurrence.

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"This orphan drug designation is another significant milestone for our VBI-1901 program, and it underscores the urgency of our effort to develop meaningful new treatment options for patients with this devastating cancer," said Jeff Baxter, President and CEO of VBI. "As recently presented at ASCO (Free ASCO Whitepaper), we continue to see strong tumor response data and improvements in overall survival data compared to historical controls in the Phase 2a study of VBI-1901. With this orphan drug status, we look forward to working closely with the FDA and clinical investigators to build on that data, advancing the potential of this program to be a valuable part of the fight against GBM."

Though classified as a rare disease, GBM is the most common primary brain cancer with approximately 14,000 new cases diagnosed in the United States each year, and a low median overall survival of 15-18 months after diagnosis of primary GBM.1 Glioblastomas are stage IV brain tumors – they are an exceptionally aggressive form of brain cancer, with high recurrence rates and a five-year survival rate around 10%.2 Standard of care in the frontline setting includes surgical resection, chemotherapy, and radiation therapy. There is no effective standard of care in the recurrent setting – median overall survival in this patient population is approximately eight months.3

About FDA Orphan Drug Designation

The FDA’s Office of Orphan Products Development grants orphan drug designation to investigational drugs and biologics intended to prevent, diagnose, or treat rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for qualified clinical trials, exemptions from certain FDA fees, and the potential for seven years of post-approval marketing exclusivity.

About FDA Fast Track Designation

The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to: 1) treat serious or life-threatening conditions, and 2) demonstrate the potential to address unmet medical needs. A therapeutic that receives Fast Track Designation is eligible for some or all of the following: 1) more frequent meetings with FDA to discuss the development plan and data needed to support approval, 2) more frequent written communication from FDA relating to the design of the proposed clinical trials and use of biomarkers, 3) Accelerated Approval and Priority Review, if relevant criteria are met, and 4) Rolling Review, which means the company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, instead of waiting until all sections of the application are completed.

Fast Track Designation was granted to VBI-1901, adjuvanted with granulocyte macrophage colony-stimulating factor (GM-CSF), for the treatment of first-recurrent GBM in June 2021.

About VBI-1901 and GBM

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 14,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

To learn more about VBI’s ongoing Phase 1/2a study and the INSIGhT trial, visit clinicaltrials.gov (Respective Identifiers: NCT03382977 and NCT02977780).

On June 22, 2022 TriSalus Life Sciences, an immunotherapy company on a mission to extend and improve the lives of patients with liver and pancreatic tumors, reported that highlighted a series of updates from clinical and pre-clinical studies that help support the company’s immunotherapy platform approach for treating liver and pancreatic tumors (Press release, TriSalus Life Sciences, JUN 22, 2022, View Source [SID1234616180]).

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Since acquiring its first therapeutic candidate in 2020, TriSalus has initiated two Phase I clinical trials and numerous pre-clinical studies with leading cancer institutes across the U.S. These studies are evaluating the company’s immunotherapy platform, which integrates an investigational class C TLR9 agonist, SD-101, and the proprietary Pressure-Enabled Drug Delivery (PEDD) method of administration to overcome critical treatment barriers that can prevent immunotherapeutic uptake in hard-to-treat tumors.

"Patients with liver and pancreatic tumors can experience poor outcomes due to tumor-induced immunosuppression and high intratumoral pressure that often prevent therapeutics, such as immunotherapy drugs, from reaching their targets. Existing treatment approaches, including direct needle injections and intravenous immunotherapy alone, often fail to address these barriers in a comprehensive way. By engineering new approaches to meet these challenges, we may be able to improve the treatment paradigm for liver and pancreas tumor patients," said Steven C. Katz, MD, FACS, Chief Medical Officer at TriSalus.

Dr. Katz continued, "The TriSalus immunotherapy platform combines the well-studied, investigational immunotherapy, SD-101, administered via PEDD, an innovative vascular delivery method leveraged by FDA-cleared devices. Pre-clinical and early clinical data from studies examining this novel approach are promising and give us hope that we will be able to offer liver and pancreas tumor patients a better chance of responding more reliably to immunotherapy. While our approach is highly innovative, the delivery technology has been used in over 16,000 cases and SD-101 has been studied through Phase 2 trials in other indications. As such, we are building upon reassuring safety data for both SD-101 and PEDD and hope that, by integrating the two into a single platform, we can enable better responsiveness to systemic immunotherapy."

Clinical Development Update for Liver and Pancreas Immunotherapy Platform

Pressure-Enabled Regional Immuno-Oncology (PERIO-01) Clinical Study

In June, the Pressure-Enabled Regional Immuno-Oncology (PERIO-01) clinical trial’s Principal Investigator, Dr. Sapna Patel, an Associate Professor and Director of the Uveal Melanoma Program and Melanoma Fellowship Program at the University of Texas MD Anderson Cancer Center, delivered an oral presentation at the 20th Congress of the International Society of Ocular Oncology. During this presentation, Dr. Patel discussed the innovative approach being taken in the PERIO-01 trial and reviewed early data that supports the safety of SD-101 and immunologic effects within liver tumors at various dose levels.

The PERIO-01 clinical trial (NCT04935229) seeks to evaluate whether the TriSalus immunotherapy platform approach can improve the performance of systemic checkpoint inhibitors in patients with uveal melanoma with liver metastases, a rare form of eye cancer that frequently spreads to the liver. The clinical trial is underway at leading academic medical centers, including MD Anderson Cancer Center, Columbia University Irving Medical Center, Thomas Jefferson University Hospital, Massachusetts General Hospital, and University of Pittsburgh Medical Centers. Since enrollment of the first patient in the PERIO-01 trial, TriSalus has fully enrolled patients at four dose levels in Cohort A, which is evaluating the safety of SD-101, and started enrollment for Cohort B to evaluate the safety of SD-101 in combination with nivolumab, a checkpoint inhibitor.

Early data from the PERIO-01 study, also presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Tumor Immune Microenvironment Workshop, suggest SD-101 delivered via PEDD has enabled immunologic activity within the liver metastases’ tumor microenvironment in metastatic uveal melanoma patients. This data bolsters evidence that suggests this integrated approach may support better immune checkpoint inhibitor performance in uveal melanoma patients and other intrahepatic indications, within the liver and potentially at other disease sites. These clinical data resonate well with a newly published pre-clinical study in Cancer Gene Therapy that suggests administering a class C TLR9 agonist via PEDD can improve responsiveness to systemic checkpoint inhibition for liver tumors through elimination of a highly immunosuppressive cell type known as myeloid derived suppressor cells (MDSC).

Pressure-Enabled Regional Immuno-Oncology (PERIO-02) Clinical Study

In May, TriSalus announced the first patient enrolled in its second immunotherapy platform study, PERIO-02 (NCT05220722). The trial is evaluating SD-101 in adults with locally advanced, metastatic, or unresectable hepatocellular carcinoma and intrahepatic cholangiocarcinoma. Since then, TriSalus has fully enrolled patients in the first dose level of Cohort A, which is evaluating the safety of SD-101 in this patient population. The trial was initiated at MD Anderson Cancer Center with additional sites planned. Cohort B is expected to open over the summer and will test SD-101 via PEDD in combination with systemic pembrolizumab, a checkpoint inhibitor. Importantly, testing the therapeutic platform of SD-101 delivered via PEDD across multiple indications to enable systemic checkpoint inhibition has the potential to provide data to support the broad application of this innovative approach for patients with liver or pancreas tumors.

Pre-Clinical Studies Leveraging Proprietary Infusion Technology

TriSalus’ clinical leadership team also recently presented new pre-clinical data at the Society of Interventional Radiology (SIR) 2022 Annual Scientific Meeting. In one abstract presentation, TriSalus highlighted data that suggests the PEDD method was more effective in delivering SD-101 into liver tissue than direct needle injection. To further support these findings, TriSalus is collaborating with Massachusetts General Cancer Center on an investigator-initiated clinical trial (NCT05128032) to evaluate whether PEDD improves the delivery of radioactive microspheres during radioembolization treatment for liver cancer, compared to a standard microcatheter.

In a second abstract presentation at SIR 2022, TriSalus highlighted new data on the development of a trans-venous approach for the regional treatment of pancreatic tumors, called pancreatic retrograde venous infusion (PRVI). This pre-clinical data is the first of its kind to demonstrate that the PRVI approach can improve uptake and tumor response for pancreatic tumors. Unlike the liver, using the arterial systemic for drug delivery in the pancreas poses significant anatomic challenges. TriSalus has developed a novel approach to leverage the PEDD method in venous branches using a new FDA-cleared device designed for highly targeted intra-pancreatic delivery of SD-101.

Expanded Strategic Research Collaborations

TriSalus has also announced several strategic collaborations with top-tier cancer institutions and research centers to further efforts to develop innovative treatment options. In January, TriSalus announced a three-year strategic research collaboration with the University of Colorado Anschutz Medical Campus to advance research of immuno-oncology treatments for patients with liver and pancreatic tumors, and in April, the company shared it is developing a new immunotherapeutic research laboratory on the campus of Lifespan Health System to further develop its therapeutic platform. These announcements build upon the strategic research collaboration established in 2021 with the University of Texas MD Anderson Cancer Center to evaluate TriSalus’ platform approach for the treatment of liver and pancreatic tumors.

On June 22, 2022 The Menarini Group ("Menarini") and Radius Health, Inc. ("Radius") (NASDAQ: RDUS) (collectively, the "Companies") reported that Menarini, with support from Radius, has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for elacestrant in patients with ER+/HER2- advanced or metastatic breast cancer (Press release, Radius, JUN 22, 2022, View Source [SID1234616179]).

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As part of the submission, the Companies have requested Priority Review with the FDA. If Priority Review is granted, the Companies anticipate that the FDA would conduct an 8-month review, incorporating a 6-month priority designation review.

The NDA submission is based on positive phase 3 data from the EMERALD study that was previously announced on October 20, 2021. EMERALD met both of its primary endpoints, which were progression-free survival (PFS) in the overall population and PFS in the estrogen receptor 1 (ESR1) mutation subgroup as compared to standard of care (SoC) with the options of fulvestrant or an aromatase inhibitor.

Elacestrant is the first and currently only investigational oral SERD to show positive topline results in a pivotal trial for the treatment of ER+/HER2- advanced or metastatic breast cancer in postmenopausal women, and men. Notably, these results showed elacestrant is also active in patients whose tumors harbor an ESR1 mutation, one of the key resistance mechanisms that develops in later treatment lines of metastatic breast cancer.

Following the completion of EMERALD, data from the study was presented at the San Antonio Breast Cancer Symposium (SABCS) on December 8, 2021, published in the Journal of Clinical Oncology (JCO) on May 18, 2022, and further subset analyses were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 6, 2022.

Elcin Barker Ergun, the Chief Executive Officer of Menarini, commented, "We are excited about the potential for elacestrant to be approved for treatment of patients with advanced or metastatic ER+/HER2- breast cancer, which constitutes about 70% of breast cancer and remains an area of significant unmet medical need." Barker Ergun continued, "Elacestrant has shown statistically significant efficacy over current standard of care medications both for overall population and in patients whose tumors harbor an ESR1 mutation, one of the most difficult to treat mechanisms of acquired resistance that develops in the later stages of metastatic/advanced breast cancer."

Chhaya Shah, SVP of Clinical and Regulatory at Radius, commented, "We enrolled and completed the EMERALD trial in a high-quality manner, delivered positive topline results, and prepared the submission of the NDA to the FDA. The submission is a significant milestone for both companies, and we appreciate the strong, collaborative effort of many hard-working employees at Radius and Menarini, investigators, patients, and their families. Together we look forward to advancing elacestrant and providing the opportunity to benefit patients."

Nassir Habboubi, Global Head of Pharma R&D of Menarini Group, added, "The Menarini and Radius teams have done an excellent job working together since our partnership began in July of 2020." Habboubi continued, "We plan to test elacestrant in earlier treatment lines, combination trials, and metastatic breast cancer that has metastasized to the brain. These details are to be communicated by us throughout 2H 2022 and 1H 2023."

With the submission of the NDA, based on the original agreement of the Companies, Menarini takes over activities and will be responsible for registration and commercialization. Menarini plans to use its fully owned subsidiary in the U.S., Stemline Therapeutics, to commercialize elacestrant if approved by the FDA.

About Elacestrant (RAD1901) and EMERALD Phase 3 Study

Elacestrant is an investigational selective estrogen receptor degrader (SERD), out-licensed to Menarini Group, which is being evaluated for potential use as a once daily oral treatment in patients with ER+/ HER2- advanced breast cancer. In 2018, elacestrant received fast track designation from the FDA. Preclinical studies completed prior to EMERALD indicate that the compound has the potential for use as a single agent or in combination with other therapies for the treatment of breast cancer. The EMERALD Phase 3 trial is a randomized, open label, active-controlled study evaluating elacestrant as second- or third-line monotherapy in ER+/HER2- advanced/metastatic breast cancer patients. The study enrolled 477 patients who have received prior treatment with one or two lines of endocrine therapy, including a CDK 4/6 inhibitor. Patients in the study were randomized to receive either elacestrant or the investigator’s choice of an approved hormonal agent. The primary endpoint of the study was progression-free survival (PFS) in the overall patient population and in patients with estrogen receptor 1 gene (ESR1) mutations. Secondary endpoints included evaluation of overall survival (OS), objective response rate (ORR), and duration of response (DOR).

On June 22, 2022 The U.S. National Science Foundation reported a new funding opportunity of nearly $29 million through the Improving Undergraduate STEM Education: Hispanic-Serving Institutions Program, also known as the HSI program (Press release, National Science Foundation, JUN 22, 2022, View Source [SID1234616178]). Through this new solicitation, NSF introduces the HSI Program Network Resource Centers and Hubs, or HSI-Net, which seeks to establish two centers and up to five hubs to develop the infrastructure needed to generate and disseminate new knowledge, successful practices and effective design principles arising from research on and work at HSIs.

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Through the HSI-Net, NSF’s goal is to create a robust national ecosystem consisting of multi-sector partners supporting and sharing knowledge and successful models of undergraduate STEM education at HSIs, building institutional capacity, and effectively broadening participation of students that are historically underrepresented in STEM.

"Enhancing the quality and accessibility of STEM education is far too complex of a challenge for a one-size-fits-all solution and it is imperative that we engage in creative and meaningful partnerships with HSIs at speed and at scale to meet the STEM needs of our nation," said NSF Director Sethuraman Panchanathan. "If we are to successfully advance America’s future STEM workforce, we must enhance educational opportunities, build capacity and increase student participation, retention and career sustainability at our nation’s academic institutions. NSF’s investments in this program will do just that."

NSF’s investment in the HSI-Net will establish:

The HSI Center for Community Coordination, designed to create coordination mechanisms, foster communication, organize collaborations and strengthen connections between HSIs in the U.S. and its territories and its stakeholders, including current and new awardees.

The HSI Center for Evaluation, Research, and Synthesis, designed to develop tools and approaches to support the HSI community in culturally responsive evaluation practices, assessments, research and synthesis efforts.

Up to five HSI Program Resource Hubs, designed to support innovative initiatives addressing key areas of need in the HSI community and to effectively serve the HSI STEM community and its stakeholders.

Together, these centers and hubs will promote excellence in research that impacts the HSI community, including STEM education and broadening participation research. They will conduct activities designed to support and foster collaborations and partnerships in the HSI community, expand student and faculty development and growth, and promote the advancement of historically underrepresented groups in STEM.

"The HSI program is designed to serve the diverse HSI community with unique needs and their students and faculty," said Erika T. Camacho, director of the HSI program. "Through the creation of the HSI-Net, we are supporting community transformation efforts at HSIs to collectively contribute to research on HSIs and their capacity to address equity in education and intentionally serve their students to better prepare them to address future challenges. The HSI-Net will allow us to further impact the STEM research enterprise and workforce for the success and prosperity of our nation."

Since its founding in 2017, the HSI program has made a significant impact on the quality and accessibility of undergraduate STEM education for Hispanic and Latino students. The program now supports more than 150 projects that enable institutions to better serve students from groups historically underrepresented in the sciences and expand the pathways available to them for graduate education and STEM careers.

As part of the White House Initiative on Advancing Educational Equity, Excellence, and Economic Opportunity for Hispanics, the NSF HSI program team will host a workshop at the upcoming White House Initiative Latino Economic Summit to be held at Malcom X College, 1900 W. Jackson Boulevard, Chicago, June 23, 9:30 a.m. CDT. This session will offer insight into NSF’s funding priorities for advancing excellence in undergraduate STEM education and investments that enhance educational opportunities, build capacity and increase student uptake and career pathways throughout the nation’s Hispanic-serving institutions.

NSF anticipates that approximately up to $29 million will be available for continuing or standard awards in response to the HSI-Net solicitation, subject to the availability of funds.

On June 22, 2022 Leidos (NYSE: LDOS), a FORTUNE 500 science and technology leader, reported that the Defense Enclave Services (DES) contract awarded by the Defense Information Systems Agency (DISA) was upheld by the Government Accountability Office (GAO) after a thorough and disciplined review (Press release, Leidos, JUN 22, 2022, View Source [SID1234616176]). The single-award, indefinite delivery, indefinite quantity contract was initially awarded in February 2022 and holds a total estimated value of $11.5 billion if all options are exercised. It includes a four-year base period of performance followed by three two-year option periods.

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"We are pleased the Government Accountability Office has affirmed the Defense Information Systems Agency’s award," said Gerry Fasano, Leidos Defense Group President. "Leidos is deeply committed to DISA’s critical mission, and never stopped preparing for the program’s success. Our robust and continuous preparation has positioned us to start delivering benefits to the user base on or ahead of the current planned schedule, and we look forward to leveraging our decades of technological expertise to support mission success."

Through the DES contract, Leidos will support DISA’s mission by consolidating enterprise IT services at a global scale and by providing standardized, responsive and cost-effective solutions. The program involves a wide range of IT services for more than 370,000 users spanning 22 Department of Defense (DoD) agencies and field activities with over 500 sites both in the U.S. and abroad. This work will focus on mission value and user experience, while improving cybersecurity, network availability, and reliability for Fourth Estate agencies.

DISA is headquartered at Fort George G. Meade in Maryland and supports joint warfighters, national level leaders, and other mission and coalition partners across the full spectrum of military operations with information sharing capabilities, command and control solutions and global enterprise infrastructure.