On April 25, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, reported the results of a new study demonstrating the potential impact of access to omidubicel on health disparities in allogeneic hematopoietic stem cell transplant in a poster presentation at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022 (Press release, Gamida Cell, APR 25, 2022, View Source [SID1234612923]).

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The study, titled "Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant Access and Outcomes for Patients with Hematologic Malignancies in the US," leveraged a decision-tree model to project allo-HCT access and clinical outcomes in a hypothetical population of 10,000 allo-HCT–eligible patients in the U.S. with hematologic malignancies without an available match-related donor. The study concluded that broad use of omidubicel will extend access for allo-HCT-eligible patients, decrease time to transplant and improve clinical outcomes, notably among racial and ethnic groups with worse status quo outcomes. Projected increases in one-year overall survival ranged (with 20% omidubicel use among allo-HCT–eligible patients) from 2.5% for whites patients to 6.3% for Black patients. The study also concluded that higher levels of modeled omidubicel uptake were associated with greater improvements in clinical outcomes and greater reductions in racial disparities.

Previous studies indicate that non-white patients have a lower likelihood of finding an appropriate match in the U.S. public donor registries, with Black patients have a 16-20% chance of finding an appropriate match. Given that an allogeneic stem cell transplant is intended as a curative option, if patients cannot find an appropriate match they will not have access to allogeneic stem cell transplant, a potentially curative treatment. The Phase 3 study of omidubicel demonstrated the ability of the therapy to be used as a donor source for racially and ethnically diverse patients with 40% of patients enrolled in the study being non-white.

"Today, minority groups comprise only about 30% of all allogeneic hematopoietic stem cell transplant transplants, indicating that lack of access to a matched donor is a significant barrier to treatment in the current landscape," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "This study is encouraging in that it projects that broad access to omidubicel has the potential to open up allo-HSCT as an effective treatment for more patients and address the barriers that have contributed to this alarming health disparity. These data are particularly encouraging as we continue to advance our rolling BLA submission to the FDA and move closer to bringing the therapy to more patients in need."

Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.

In addition to this poster, two oral presentations and four additional poster presentations on omidubicel and a poster presentation on GDA-201, the company’s leading NK cell therapy program, will be shared during the conference. All poster presentations will be publicly available at www.ASTCT.org. Details below:

Title (Oral Presentation): Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation (Part of Best Abstract Award Session)
Presenting Author: Paul Szabolcs, M.D., Division of Blood and Marrow Transplantation and Cellular Therapy, UPMC Children’s Hospital of Pittsburgh, Pittsburg, PA
Session Title: Tandem Meetings Best Abstracts Session in the Scientific Track
Session Date / Time: Monday, April 25, 2022, 6:00 PM – 6:15 PM MT, SPCC, Ballroom D
Title (Oral Presentation): Allogeneic HSCT with Omidubicel demonstrates sustained clinical improvement versus standard myeloablative UCBT: Final results of a Phase III randomized multicenter study
Presenting Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute
Session Title: Oral Abstract – Session L – Consider the Source: Stem Cell Grafts and Donors
Session Date / Time: Tuesday, April 26, 2022, 3:15 PM – 3:30 PM MT
Title: (Poster): Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT) With Omidubicel: Long-Term Follow-Up From A Single Center (ASH Encore)
Lead Author: Chenyu Lin, M.D., Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, NC
Title (Poster): Total Costs of Care and Complication Rates Among Patients with Hematologic Malignancies Who Receive Allogeneic Hematopoietic Cell Transplants in the US
Lead Author: Richard Maziarz, M.D., Professor of Medicine, Medical Director Adult Blood and Marrow Stem Cell Transplant Program, Oregon Health and Science University, Portland, OR
Title (Poster): Hospitalization and Healthcare Resource Use of Omidubicel Vs Umbilical Cord Blood (UCB) for Hematological Malignancies in a Global Randomized Phase III Clinical Trial Setting
Lead Author: Navneet Majhail, M.D., Taussig Cancer Institute, Department of Hematology and Oncology, Cleveland Clinic, Cleveland, OH
Title (Poster): HRQOL following transplantation with omidubicel versus UCB in patients with hematologic malignancies: Results from a Phase III randomized , multicenter study
Lead Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute
Title (Poster): Transcriptional and Metabolic Profiling of Nicotinamide-Enhanced Natural Killer (NAM-NK) Cells (GDA-201)
Lead Author: Dima Yackoubov, Scientist, Gamida Cell
About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status and orphan drug designation by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). For more information on clinical trials of omidubicel, please visit the Gamida Cell website.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we are able to enhance, expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.

On April 25, 2022 Turnstone Biologics Corp., a clinical-stage biotechnology company developing next-generation tumor-infiltrating lymphocyte ("TIL") therapy and viral immunotherapy designed to treat and cure solid tumors, reported a research collaboration with the University of Montreal Hospital Research Centre [Centre de recherche du Centre hospitalier de l’Université de Montréal, ("CRCHUM")], to drive advancements in TIL therapy using tumor-reactive T-cells (Press release, Turnstone Biologics, APR 25, 2022, View Source [SID1234612922]). The goal of the collaboration is to identify genome alterations and tumor-specific antigens from different tumor types and to further streamline the process for identification, direct selection and expansion of Turnstone’s tumor-reactive TILs, with the potential to expand this work into future clinical trials at CRCHUM.

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Dr. Simon Turcotte, associate professor of surgery at the Université de Montréal and lead of CRCHUM’s adoptive T-cell cancer immunotherapy program, has agreed to spearhead the collaboration research efforts and plans to provide key access to tumor and blood derivatives essential to conduct this research. Dr. Turcotte completed his medical and surgical training at the Université de Montréal, his post-doctoral research training on TIL immunotherapy at the National Cancer Institute, Bethesda, MD, in the laboratory of Dr. Steven A. Rosenberg, and a hepatobiliary and pancreatic surgical oncology fellowship at the Memorial Sloan Kettering Cancer Center in New York, NY.

"Considering all the different types of T-cell-based immunotherapies being studied to treat advanced solid cancers, TILs have shown the best potential for efficacy to date. This collaboration between CRCHUM and Turnstone Biologics, the first of its kind in Canada, engages us on the exciting path of developing next-generation TILs aimed at better recognizing cancer cells," said Dr. Turcotte. "By bringing together CRCHUM’s unique capabilities with Turnstone’s technology platform, and by applying our own complementary cell therapy research efforts, we see significant potential to improve treatment outcomes for cancer patients."

"Our TIL platform has transformative potential across a wide range of solid tumors," said Stewart Abbot, Ph.D., Chief Scientific Officer, Turnstone. "Establishing this collaboration with Dr. Turcotte and CRCHUM is a tremendous opportunity to advance our vision to unlock the full potential of TILs. We continue to seek innovative technologies and the field’s best minds in support of our development programs. We believe Dr. Turcotte’s extensive expertise and our combined scientific team will allow us to accelerate the development of our tumor-reactive selected TIL product candidates and lead to more effective medicines."

On April 25, 2022 Laboratoires DELBERT reported the signature with Sanofi on March 31st, 2022 of the acquisition of Teralithe 250mg, divisible tablet and Teralithe LP 400mg, prolonged-release tablet (lithium carbonate) in France (Press release, Sanofi, APR 25, 2022, View Source [SID1234612921]).

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Laboratoires DELBERT (View Source) is a French Pharmaceutical company specialized in the « Revival of essential medications » : Marketing discontinuation, stock shortages …affect more and more essential medications especially in crisis periods with the consequence of narrowing the therapeutic arsenal of doctors that may force well-controlled patients to switch to other therapies.

With this acquisition Laboratoires DELBERT reinforces the execution of their strategy in order to ensure safe and continuous availability of Essential Medications to patients and reinforce their CNS portfolio.

"This acquisition demonstrates our ongoing commitment to invest in Major Therapeutical Interest Medications (MITM). Maintaining these medications available for patients in need is our priority," said Marc Childs, & Thierry Hoffmann co-founders of Laboratoires DELBERT, « Laboratoires DELBERT built up over the time a portfolio of MITM that is anchored in 3 specific therapeutic areas : Antiinfectives, CNS and Oncology. »

Laboratoires DELBERT will reach over 30m€ sales by 2022 with double digit growth every year and already commercializes 14 products (out of which 13 princeps) for hospitals/specialists prescriptions.

Laboratoires DELBERT financed this acquisition thanks to very strong relationship with its financial partners who strongly believe in their values focused on patients access to MITM.

On April 25, 2022 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 updated interim data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHLs") and chronic lymphocytic leukemia ("CLL") (Press release, Mustang Bio, APR 25, 2022, View Source [SID1234612920]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center ("Fred Hutch").

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The data, which were presented by Mazyar Shadman, M.D., M.P.H., Associate Professor and physician at Fred Hutch and University of Washington, at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy ("ASTCT") and Center for International Blood & Marrow Transplant Research ("CIBMTR"), demonstrated high efficacy and a very favorable safety profile in all patients (n=25). Five dose levels were used during the study, and complete responses were observed at all dose levels. Durable responses were observed in a wide range of hematologic malignancies including follicular lymphoma ("FL"), CLL, diffuse large B-cell lymphoma ("DLBCL"), and Waldenstrom macroglobulinemia ("WM"). An overall response rate ("ORR") of 96% and complete response ("CR") rate of 72% was observed in all patients across all dose levels. Additionally, two patients had been previously treated with CD19-directed CAR T therapy and subsequently relapsed, and both responded to treatment, one patient with FL with a CR and the other with DLBCL with a partial response.

CAR T expansion was observed across all dose levels. At the 28-day evaluation, a favorable safety profile was observed in all 25 patients. No patients experienced grade 3 or 4 cytokine release syndrome or immune effector cell‐associated neurotoxicity syndrome ("ICANS"), and none of the FL patients experienced ICANS of any grade (n=18).

"We are pleased that in this single institution study, we observed a favorable safety profile and a high rate of complete and durable responses, which make MB-106 suitable for outpatient treatment. Additionally, the responses from patients treated previously with CD19-directed CAR T cell therapy show the potential of MB-106 as an immunotherapy option for these patients. Enrollment in this study remains open to patients with CD20+ B-NHLs and CLL, including patients with prior CAR T treatment," said Dr. Shadman.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "MB-106 continues to demonstrate highly promising clinical activity. In particular, the 100% response rates of WM patients as well as of NHL patients previously treated with CD19-directed CAR T cell therapy underscore the potential for MB-106 to treat these patient populations with high unmet needs. The possible outpatient administration of this therapy makes it potentially even more compelling. We are excited to advance our CD20-targeted CAR T cell therapy program with the launch of a multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL under Mustang’s IND and plan to dose the first patient this quarter."

Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody, and MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the planned multicenter phase 1/2 clinical trial to be initiated shortly under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on the trial can be found at View Source using the identifier NCT03277729.

On April 25, 2022 Merus N.V. (Nasdaq: MRUS) ("Merus", "the Company", "we", or "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported publication of a preclinical report on petosemtamab (Peto, MCLA-158: LGR5 x EGFR Biclonics) in the journal Nature Cancer (Press release, Merus, APR 25, 2022, View Source [SID1234612919]). The publication will be available at 11:00 a.m. ET today. The report describes the use of the company’s Biclonics platform to perform a large-scale functional screen of bispecific antibodies resulting in selection of Peto, a bispecific antibody targeting the epidermal growth factor receptor (EGFR) and the leucine-rich repeat containing G protein-coupled receptor (LGR5). Peto displayed potent growth inhibition of colorectal cancer (CRC) organoids, blockade of metastasis initiation and tumor outgrowth in preclinical models of different tumor types. Peto specifically triggered EGFR degradation in organoids expressing LGR5, while showing minimal toxicity towards normal LGR5-expressing organoids.

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In October 2021, Merus reported early, interim clinical data in an ongoing trial of Peto in patients with head and neck squamous cell carcinoma (HNSCC).

"This publication demonstrates the potential of our Biclonics platform to generate large numbers of diverse panels of antibodies, undertake high throughput functional screening of in-format bispecifics, and identify drug candidates that possess specific biology and characteristics for therapeutic applications," said Cecile Geuijen, Ph.D., Senior Vice President and Chief Scientific Officer. "We are encouraged by the preclinical and clinical data we have obtained to date, and look forward to the further clinical development of Peto."

The results published in Nature Cancer further describe Peto’s results in preclinical models of solid tumors, including the following highlights:

Peto exhibits unique therapeutic properties such as potent growth inhibition of KRAS mutant CRC organoids, blockade of metastasis initiation, and inhibition of tumor outgrowth in preclinical models of different tumor types
Peto shows superior growth inhibition relative to cetuximab, an EGFR inhibitor used for treatment of metastatic CRC and HNSCC, in subcutaneous xenografts generated from inoculation of C31M, a patient-derived CRC organoid bearing a KRAS G12D mutation
Unlike cetuximab, Peto triggers EGFR internalization and degradation through LGR5
Peto shows in vivo anti-tumor activity in other tumor types that express LGR5 such as esophageal squamous cell carcinoma, gastric carcinoma and HNSCC
Peto is currently enrolling in a phase 1 open-label, multicenter study in patients with solid tumors. Merus is planning a clinical update for the second half of 2022.

Merus is the sponsor of the clinical trial, investigating Peto, and certain preclinical work described in the Nature Cancer paper was generated in conjunction with the suppresSTEM consortium, among other institutions and organizations cited in the publication.

About Petosemtamab (Peto, MCLA-158)
Peto is an ADCC-enhanced human IgG1 Biclonics designed to bind to cancer stem cells (CSCs) expressing leucine-rich repeat-containing G protein-coupled receptor 5 (Lgr5) and epidermal growth factor receptor (EGFR). In preclinical models, Peto binding triggers EGFR degradation in LGR5+ CSCs and is designed to have two different mechanisms of action. The first entails blocking of growth and survival pathways in cancer initiating cells. The second exploits the recruitment and enhancement of immune effector cells to directly kill cancer initiating cells that persist in solid tumors and can cause relapse and metastasis.