On February 9, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, reported the results of a Phase 3 clinical study of omidubicel presented in an oral session at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR), or the TCT Meetings. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies (Press release, Gamida Cell, FEB 9, 2021, View Source [SID1234574813]).

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This clinical data set was from the international, multi-center, randomized Phase 3 study of omidubicel that 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. This is the first presentation of these data in a peer-reviewed conference. The full presentation is available on the Gamida Cell website.

"The results of this global Phase 3 study of omidubicel in patients with hematologic malignancies show that omidubicel resulted in faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital, all of which are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "The comparator, a transplant with umbilical cord blood, has been historically shown to result in low incidence of graft versus host disease (GvHD) in relation to other graft sources, and in this study, omidubicel demonstrated a GvHD profile similar to the comparator. Moreover, previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study receiving their transplant more than 10 years ago. The data presented at this meeting indicate that omidubicel has the potential to be considered a new standard of care for patients who are in need of stem cell transplantation but do not have access to a matched donor."

Details of Phase 3 Efficacy and Safety Results Shared at the TCT Meetings

Patient demographics including racial and ethnic diversity and baseline characteristics were well-balanced across the two study groups. The study’s intent-to-treat analysis included 125 patients aged 13–65 years with a median age of 41. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. Patients were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and a key milestone in a patient’s recovery from a bone marrow transplant. 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).

All three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel in relation to patients randomized to the comparator group (intent-to-treat). Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 60.5 days, compared to 48.0 days for the comparator (p = 0.005). The details of these data were first reported in December 2020.

Previously unpublished data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the study’s primary and secondary endpoints. There was no statistically significant difference between the two patient groups related to grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results will form the basis of a Biologics License Application (BLA) that Gamida Cell expects to submit to the U.S. Food and Drug Administration (FDA) in the second half of 2021.

"We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "Sharing the results of the Phase 3 study of omidubicel with the transplant community is a major moment for Gamida Cell, and we are forever grateful to the patients who participated in this study, their caregivers, and the work of the investigators and their teams."

About Omidubicel

Omidubicel 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). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). 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 established by the FDA or any other health authority.

On February 9, 2021 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported strong results of operations for the fourth quarter and full-year 2020, exceeding the goals for net sales and adjusted earnings per share (Press release, Qiagen, FEB 9, 2021, View Source [SID1234574812]).

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Net sales in the fourth quarter of 2020 rose 38% (+36% at constant exchange rates, CER) to $571.2 million, and faster than the outlook for at least 32% CER growth. Adjusted earnings per share (EPS) grew 42% to $0.68 ($0.68 CER) against the outlook for about $0.64-0.65 CER. Net sales for full-year 2020 increased 23% (+23% CER) to $1.87 billion, and exceeded the outlook for about 22% CER sales growth. Adjusted EPS for 2020 rose to $2.15 ($2.17 CER), and above the outlook for about $2.13-2.14 CER.

"Our results for the fourth quarter represented the best quarterly performance of 2020, and once again above the outlook we had set for sales and earnings growth over 2019. We experienced further improvements in non-COVID areas of the portfolio along with very strong demand for product groups addressing the critical demand for COVID-19 testing," said Thierry Bernard, Chief Executive Officer of QIAGEN N.V.

"Our QIAGENers continue to display the highest level of dedication in supplying key products for much-needed testing workflows to support the global response to the COVID-19 pandemic while also executing on our strategic focus on five pillars of growth to drive mid-term growth. Teams are working on regulatory submissions and expanding the use of key solutions in our COVID-19 portfolio that also have applications once the pandemic has subsided. We are particularly pleased to help customers overcome key testing bottlenecks with solutions such as our QIAprep&amp solution, a PCR workflow that is being implemented in labs around the world. We are also making progress on ramping up production capacity for our automated RNA extraction kits, as overall sales of sample technologies were higher. Another top priority is to ensure that our QIAstat-Dx and NeuMoDx tests continue to accurately detect emerging viral variants. As we focus on delivering COVID-19 solutions, we are seeing increasing demand in other areas of our portfolio. These include better trends for the QuantiFERON TB test, our portfolio for oncology testing, universal NGS solutions and DNA sample technologies. Based on the combination of ongoing demand for COVID-19 solutions and improving customer trends in other areas, we feel confident in achieving our outlook for sales and earnings in 2021. QIAGEN is committed to remaining at the forefront of the fight against COVID while expanding our business for post-pandemic growth."

On February 9, 2021 Taiga Biotechnologies, a cell-based immunotherapy company, reported that a colorectal cancer patient at the Davidoff Cancer Center, Beilenson Hospital, Rabin Medical Center, Petach Tikva, was the first recipient of a treatment designed to treat drug-resistant solid tumors in the company’s recently approved Israeli trial. (NCT04640246) (Press release, Taiga Biotechnologies, FEB 9, 2021, View Source [SID1234574811]).

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"The initiation of this study, under the aegis of the Israeli Ministry of Health, represents a significant milestone in the clinical development of the TBX-3400 program. We now have four clinical programs that have begun in the past 18 months," said Dr. Yosef Refaeli, Chief Executive Officer at Taiga Biotechnologies. "We hope to generate critical data that will enable us to focus the development of TBX-3400 in the human clinical space," said Dr. Brian C. Turner, Chief Scientific Officer.

"We are eager to explore the ability of TBX-3400 to treat multiple tumor types in patients whose disease no longer responds to commonly used therapies," said Professor Salomon M. Stemmer, head of the research unit at the Davidoff Center and Primary Investigator of the study. "This novel approach for liberating the patient’s own immune system against the tumor may also hold promise for many different types of malignancy in both adults and children," said Dr. Jerry Stein, head of Bone Marrow Transplantation Unit, Schneider Childrens’ Medical Center, who is overseeing production of TBX-3400 for the Israeli clinical trial.

About TBX-3400

Taiga has developed a proprietary recombinant protein that can passively enter cells without the need for binding and uptake by a cell surface receptor, which can transiently alter intracellular levels of a critical protein involved in survival and proliferation called "MYC." Normally, white blood cells of the immune system that come into contact with a tumor within a patient are crippled by multiple suppressive signals generated by the tumor. Treating these immune cells with the special protein enables them to break free from the cancer-derived dampening signals. Treated cells are called "TBX-3400." To make TBX-3400 for a cancer patient, blood-derived white blood cells are incubated with Taiga’s recombinant protein in a certified lab under the most exacting standards of Good Manufacturing Practice. The cells are rinsed in a machine and then formulated in a standard solution before they are infused back into the patient in the ambulatory clinic. This approach results in a transient increase in immune function by allowing the immune cells to target the cancer. This treatment may be beneficial for a variety of solid tumors and infectious diseases. Viruses such as hepatitis B, like cancers, can escape from the immune system by triggering suppressive signals in white blood cells. Cells that are treated with the patented protein are temporarily liberated, allowing them to fight the virus infected cells.

Summary of TBX-3400 clinical programs

As mentioned above, Taiga has begun several clinical programs in the past year and a half.

Taiga has successfully completed a first-in-human safety and early efficacy pilot study in adults, covered by the regulations of the Japanese Ministry of Health, Labour and Welfare. These patients did not experience any significant treatment-related adverse events and the treatment was well tolerated. In addition, early signs of efficacy were observed in several of the subjects treated.

Taiga has an active Investigational New Drug (IND) on file with the US FDA (NCT03385486). The US study (TBX-3400-001) is enrolling patients with stage III or IV melanoma which has become refractory or resistant to immune checkpoint inhibitors. The study is now open at two major medical centers in the US.

Taiga is also conducting a pilot study on the use of TBX-3400 to treat patients with chronic hepatitis B virus (HBV) associated hepatocellular carcinoma (HCC) in Thailand. This study is currently recruiting patients and is measuring both tumor markers as well as Hepatitis B viremia following the immunotherapy.

On February 9, 2021 BostonGene Corporation, a biomedical software company committed to defining optimal precision medicine-based therapies for cancer patients, and Weill Cornell Medicine, reported a publication in Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, BostonGene, FEB 9, 2021, View Source [SID1234574810]). The manuscript "Clinical and biological subtypes of B-cell lymphoma revealed by microenvironmental signatures" highlights the tumor microenvironment as a critical component of B-cell lymphoma biology and the effects of different microenvironments on diffuse large B-cell lymphoma (DLBCL) clinical behavior, establishing a significant opportunity for the development of novel and personalized therapeutic strategies for this disease.

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In this research study, the microenvironment subtypes of over 4,600 DLBCL patients were classified using curated and refined transcriptional signatures encompassing key microenvironment and cancer cells activities and processes. This analysis revealed four distinct DLBCL microenvironments (LMEs), each with its own set of unique biological and clinical properties. The LMEs were also found to correlate with different clinical outcomes and prognoses, and downstream preclinical mechanistic studies demonstrated that the LMEs could be applied in clinical decision-making for DLBCL patients.

"This study was designed to evaluate the role of the tumor microenvironment in DLBCL biology," said Leandro Cerchietti, M.D., Associate Professor of Medicine and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine. "The results revealed distinct DLBCL microenvironments with unique therapeutic vulnerabilities that can be utilized for optimization of DLBCL treatment strategies."

"The data demonstrate that this novel classification platform provides a roadmap for the therapeutic exploitation of the tumor microenvironment in DLBCL patients," said Nathan Fowler, MD, Chief Medical Officer at BostonGene. "Together with Weill Cornell Medicine, we look forward to identifying new treatment strategies to ultimately improve the clinical outcomes of these patients."

On February 9, 2021 Brooklyn ImmunoTherapeutics LLC ("Brooklyn"), a biopharmaceutical company focused on exploring the role that cytokine-based therapy can have in treating patients with cancer, reported the initiation of an open-label Phase 2 clinical trial of IRX-2 in combination with pembrolizumab (Keytruda) and chemotherapy in triple negative breast cancer (TNBC) (Press release, Brooklyn ImmunoTherapeutics, FEB 9, 2021, View Source [SID1234574809]). The trial will be conducted at the Providence Cancer Institute in Portland, Oregon.

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IRX-2 is an allogeneic, cell-derived biologic with multiple active cytokine components, including IL-2, that act on various parts of the immune system associated with activation of the entire tumor microenvironment.

"In a Phase 1b clinical trial in early stage breast cancer, IRX-2 was well-tolerated as a single agent with no Grade 3 or 4 toxicities in patients and encouraging signs of activity including upregulation of PD-L1," said principal investigator David Page, M.D., medical oncologist and assistant member, Earle A. Chiles Research Institute, a division of Providence Cancer Institute. "These results provide a strong rationale for studying IRX-2 in combination with pembrolizumab, an immunotherapy that targets PD-1, and standard of care chemotherapy in triple negative breast cancer, a disease for which there is an urgent need for new treatment options."

"IRX-2 is currently being studied in a range of cancer indications, both as a single agent and in combination with other anti-cancer therapies, including checkpoint inhibitors," said Lynn Sadowski Mason, Executive Vice President, Clinical Operations of Brooklyn ImmunoTherapeutics. "We believe that the unique combination of cytokines in IRX-2 can improve outcomes for patients with solid tumor cancers based on its ability to activate the immune system in the tumor microenvironment. We look forward to the results of this clinical trial and to exploring potential new indications in the future."

About the Phase 2 Trial

The Phase 2 randomized, open-label trial is designed to assess the efficacy and safety of IRX-2 in patients with TNBC. Approximately 30 patients in total are expected to be enrolled. Patients with locally confirmed TNBC who have previously untreated locally advanced non-metastatic TNBC are eligible. Patients will receive alternating regimens of Pembrolizumab plus chemotherapy and subcutaneous IRX-2 injections twice a day for 10 days as neoadjuvant therapy prior to surgery. The primary efficacy endpoint is pathological Complete Response (pCR), and patients will be evaluated following definitive surgery. Please refer to www.clinicaltrials.gov (NCT04373031) for additional clinical trial details.

This study is being supported in part by a research grant from the Investigator-Initiated Studies Program of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.