On May 12, 2020 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported several recent peer-reviewed publications that utilized the company’s GeoMx Digital Spatial Profiler (DSP) to discover spatially-resolved biomarkers (Press release, NanoString Technologies, MAY 12, 2020, View Source [SID1234557617]). GeoMx DSP is an integrated system comprised of hardware, software, and reagents, capable of being read out using either the nCounter Analysis System or Next-Generation Sequencing (NGS).

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The first of these papers entitled, "Multiplex digital spatial profiling of proteins and RNA in fixed tissue," was published in the journal Nature Biotechnology (View Source). In this paper, a team of scientists from NanoString provide the most complete description to date of the GeoMx DSP’s capabilities to spatial profile proteins and RNA in formalin-fixed paraffin-embedded (FFPE) tissue.

The study describes extensive validation of the performance of each multiplexed panel using FFPE cell lines and compares performance to standard methods such as traditional immunohistochemistry (IHC) and in situ hybridization (ISH). The publication also includes case studies of the use of GeoMx DSP across a variety of tissue types and disease areas, illustrating the platform’s flexibility in selecting regions of interest (ROI) for analysis. Examples of key applications enabled by the light directed, non-destructive, ROI selection include auto-segmentation of tumor and tumor microenvironment, hypothesis-free gridding of the entire tissue samples, contour mapping across tissue substructures, and rare cell profiling. In addition, this is the first peer-reviewed publication to detail the performance achieved when GeoMx DSP is read out using NGS, including data spatially profiling 1,400+ genes using a prototype version of the Cancer Transcriptome Atlas.

"Spatial biology is emerging as a fundamental area of research in both discovery and translational science," said Joe Beechem, Chief Scientific Offer and SVP of R&D for NanoString. "Together these papers demonstrate the power and flexibility of GeoMx DSP to span the entire continuum of research — discovering new biology, identifying disease biomarkers, and potentially enabling future diagnostic tests."

Two recent publications from David Rimm and colleagues at Yale University further highlight the translational capabilities of the GeoMx DSP platform. The first publication, "Digital quantitative assessment of PD-L1 using digital spatial profiling" was published in the Nature journal Laboratory Investigation (View Source). This publication highlights the dynamic range of digital counts, high correlation to IHC and quantitative IHC, and the reproducibility of the GeoMx DSP platform. GeoMx DSP was found to generate data comparable to well established industry standard IHC assays which include the FDA approved PD-L1 assay, lab-developed test assays and quantitative immunofluorescence.

Dr. Rimm’s group second paper titled, "Biomarkers associated with beneficial PD-1 checkpoint blockade in Non-Small Cell Lung Cancer (NSCLC) identified using high-plex digital spatial profiling" was published in the journal Clinical Cancer Research. The authors used GeoMx DSP to discover twelve spatially informed biomarkers that are predictive of response to PD-1 checkpoint blockade and associated with clinical outcomes in NSCLC patients. The researchers concluded that the ability to reliably quantify multiple targets from a single tissue has the potential to make GeoMx DSP ideally suited for developing companion diagnostic assays. (View Source).

"The robust quantification and molecular compartmentalization capabilities were critical in identifying immune compartment specific biomarker candidates for predicting response to PD-1 checkpoint blockade," said David Rimm, MD, PhD, Professor of Pathology and Medicine, Yale University. "These data provide confidence for discovering novel bioclassifiers as well as developing clinical applications, from prognostic assays to companion diagnostics, using the GeoMx DSP system."

On May 12, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel, an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of bone marrow transplant (Press release, Gamida Cell, MAY 12, 2020, View Source [SID1234557616]). The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001). Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalizations.1

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Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.2 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect their quality of life.3 Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of stem cells while preserving the cells’ functional therapeutic characteristics.

"I’m very encouraged by the data from this rigorous, Phase 3 study that was conducted at more than 50 centers around the world, as there is a significant need for new bone marrow transplant graft modalities," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "These results have the potential to substantially move the field forward and represent an important step toward making stem cell transplantation more accessible and more successful for patients with lethal blood cancers. Shortening the time to engraftment is clinically meaningful, as it can reduce a patient’s time in the hospital and decrease likelihood of infection."

"Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has the potential to be the first FDA-approved bone marrow transplant graft. We are very pleased with the results of the Phase 3 data reported today, which move us one step closer toward bringing potentially curative therapies to patients. We expect to begin to submit our biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of this year," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We deeply appreciate the participation of patients in this trial and the support we have received from investigators and their teams."

Topline Phase 3 Data

The international, multi-center, randomized Phase 3 study (NCT02730299) was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. The primary endpoint was time to neutrophil engraftment. The intent-to-treat analysis included 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma and was conducted at more than 50 clinical centers in the United States, Latin America, Europe and Asia. The demographics and baseline characteristics were well-balanced across the two study groups.

The study achieved its primary endpoint (p<0.001). In the intent-to-treat analysis, the median time to neutrophil engraftment was significantly shorter for patients who received omidubicel (12 days; 95% CI: 10-15 days) compared to the comparator group (22 days; 95% CI: 19-25 days). Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, 96 percent of patients who received omidubicel achieved successful neutrophil engraftment, compared to 88 percent of patients in the comparator group.

"We are pleased with the outcome of this global, well-designed study in patients with life-threatening blood cancers who were in need of a bone marrow transplant and did not have an available matched donor," said Ronit Simantov, M.D., chief medical officer of Gamida Cell. "Importantly, these data confirmed the results from our earlier Phase 1/2 clinical study and demonstrated that patients who received omidubicel had more rapid recovery of neutrophils, which are key infection-fighting white blood cells."

The data reported today are consistent with results from a multi-center, Phase 1/2 study in 36 patients with advanced hematologic malignancies, which showed that patients treated with omidubicel demonstrated more rapid neutrophil engraftment compared to a concurrent cohort of 146 patients treated with standard umbilical cord blood as reported by the Center for International Blood and Bone Marrow Transplant Research.4 In the Phase 1/2 study, the median time to engraftment was 11.5 days (95% CI: 9-14 days) for omidubicel recipients compared to 21 days (95% CI: 20-23 days) for the CIBMTR cohort (p<0.001).

Gamida Cell expects to report full efficacy and safety results at a medical conference later this year.

Conference Call Information

Gamida Cell will host a conference call today, May 12, 2020, at 8:30 a.m. ET to discuss the Phase 3 study results. A live webcast of the conference call can be accessed in the "Investors" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 1-866-930-5560 (domestic toll-free), 1-409-216-0605 (international) and refer to conference ID number 5454076. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel, the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In both Phase 1/2 and Phase 3 clinical studies, omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.5 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On May 12, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will present at the Bank of America 2020 Health Care Conference on Thursday, May 14 at 4:20 p.m. ET (Press release, Sangamo Therapeutics, MAY 12, 2020, View Source [SID1234557615]).

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The audio presentation will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

On May 12, 2020 INOVIO (NASDAQ:INO) reported that Dr. J. Joseph Kim, President and CEO, along with other members of management will present in a fireside discussion at the RBC Capital Markets Global Healthcare Conference on Tuesday, May 19, 2020 at 2:30 p.m. Eastern Time (Press release, Inovio, MAY 12, 2020, View Source [SID1234557613]).

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This fireside discussion will be held virtually and a live webcast may be accessed by visiting Inovio’s website at View Source Archived versions of the presentations will be made available through the INOVIO Investor Relations Events page.

On May 12, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported two poster presentations at the virtual 23rd Annual Meeting of the American Society of Gene & Cell Therapy ("ASGCT"), being held May 12-15, 2020 (Press release, Mustang Bio, MAY 12, 2020, View Source [SID1234557612]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are extremely pleased with the strides forward that our researchers have made in gaining greater insights into our innovative CS1 chimeric antigen receptor (CAR) T cell therapy (MB-104), which we previously licensed from City of Hope. We commend them on their poster presentations at ASGCT (Free ASGCT Whitepaper) and look forward to learning more as they continue their research to optimize our clinical trials."

Details on the poster presentations are as follows:

Title: CS1 Targeted CAR-T Cells (MB-104) for the Treatment of Multiple Myeloma Shows Antitumor Activity Sparing Normal T-Cells Despite the Common Expression of CS1
Session: Cell Therapies
Abstract number: 421
Date and Time: Tuesday, May 12, 2020, 5:30 PM-6:30 PM ET
Room: Exhibit Hall C & D
Authors: Nathan Gumlaw, Aviva Joseph, James Edinger, Ekta Patel, Research and Translational Sciences, Mustang Bio, Worcester, MA

This poster describes researchers’ investigation into the impact of MB-104 on CS1 positive and negative cells in vitro, as well as T cells due to shared CS1 antigen expansion. The researchers demonstrated MB-104 does not confer biologically significant fratricide and can be successfully manufactured as evident by viability, growth kinetics and fold expansion, despite the shared antigen expression between tumor cells and T cells. CS1 positive T cells are present in culture during the expansion of MB-104, suggesting absence of fratricide. Finally, MB-104 can induce potent anti-tumor cell lysis and proliferates in response to tumor cells but not primary T cells expressing CS1. Taken together, their results demonstrate MB-104 is a novel CS1-targeting CAR T that shows potent anti-tumor cell lysis but spares normal T cells, despite the shared CS1 antigen expression.

Title: Development of an Immunohistochemistry Assay for the Detection of CS-1 Expression in Multiple Myeloma Patients
Session: Pharmacology/Toxicology Studies or Assay Development
Abstract number: 897
Date and Time: Wednesday, May 13, 2020, 5:30 PM-6:30 PM ET
Room: Exhibit Hall C & D
Authors: Bethany Biron Girard, James Edinger, Ekta Patel, Translational Sciences, Mustang Bio, Worcester, MA

This poster details a study in which researchers evaluated commercially available CS1 antibodies for IHC and identified the best clone with high specificity for CS1 to improve screening subjects for CS1 positive tumor expression prior to treatment and correlate efficacy with antigen expression. The researchers, for the first time, developed and optimized a robust immunohistochemistry assay for the assessment of CS1 expression in bone marrow core biopsy samples and plasmacytoma solid tumor samples from multiple myeloma ("MM") patients, which can be used for enrollment into Mustang’s CS1 CAR T clinical trials.

For more information, including abstracts, please visit the ASGCT (Free ASGCT Whitepaper) meeting website at View Source