On January 24, 2019 Immunomic Therapeutics, Inc. reported that its’ founder and CEO, William Hearl, Ph.D., will present an overview of the company and its UNITE technology platform at the 2019 BIO CEO & Investor Conference on February 12, 2019 at 2:00 pm EST in the Hudson/Empire Room at the Marriott Marquis in New York City (Press release, Immunomic Therapeutics, JAN 24, 2019, View Source [SID1234532885]).

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On January 24, 2019 ArQule, Inc.’s (Nasdaq: ARQL) partner, Basilea Pharmaceutica Ltd. (SIX: BSLN), reported that it entered into a collaboration with Roche (SIX: RO, ROG) to explore a combination of derazantinib (BAL087) and Roche’s PD-L1-blocking immune-checkpoint inhibitor, atezolizumab (Tecentriq), in patients with urothelial cancer. Basilea expects to start a biomarker-driven multi-cohort phase 1/2 study in mid-2019 (Press release, ArQule, JAN 24, 2019, View Source [SID1234532887]).

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The planned study will assess the safety, tolerability and efficacy of the derazantinib-atezolizumab combination in patients with advanced urothelial cancer and confirmed FGFR genomic aberrations. Basilea will be the sponsor of the study, and Roche will provide clinical supply of atezolizumab.

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Peter Lawrence, President and Chief Operating Officer of ArQule, said, "This new combination trial with Tecentriq represents an important step in the development of derazantinib (BAL087, formerly ARQ087) and has the potential to expand meaningfully its therapeutic utility. We look forward to further progress and updates from Basilea."

Derazantinib was licensed to Basilea Pharmaceutica in April 2018 in the US, EU, Japan and the rest of world excluding Greater China. Under the terms of the license agreement, ArQule is eligible to receive up to $326 million in regulatory and commercial milestone payments.

On January 24, 2019 Merck KGaA, Darmstadt, Germany, a leading science and technology company, which operates its healthcare business in the U.S. and Canada as EMD Serono, reported that it has entered into an exclusive licensing agreement with Vertex Pharmaceuticals Incorporated, Boston, USA (NASDAQ: VRTX), for two DNA-dependent protein kinase (DNA-PK) inhibitors – M9831 (formerly known as VX-984) and an additional pre-clinical compound – in the field of gene editing for six specific genetic disease indications (Press release, Merck KGaA, JAN 24, 2019, View Source [SID1234532888]). Merck KGaA, Darmstadt, Germany will receive an upfront payment in addition to milestones and royalties on future net sales and retains the rights to both assets in all other disease areas, including oncology, with the ability to develop both these compounds in-house, or to license them to future partners in the gene editing field. Vertex has the option to add indications to the license grant. Both molecules were acquired in a licensing agreement from Vertex in 2017, and are part of the company’s broad portfolio of DNA Damage Response (DDR) inhibitors.

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"This transaction illustrates our determination to maximize value creation from our pipeline," said Belén Garijo, Member of the Executive Board and CEO Healthcare, Merck KGaA, Darmstadt, Germany. "We are rapidly advancing our leading-edge DDR portfolio in oncology and are delighted to see the potential benefit of DNA-PK in genetic diseases through the enhancement of CRISPR/Cas9-mediated gene editing."

Merck KGaA, Darmstadt, Germany is investing significant resources into the promising area of DDR, and has considerable expertise and experience in developing DDR molecules, with the objective of becoming one of the leading players in this therapeutic area. The company is currently investigating four DDR molecules, including two ATR inhibitors, an ATM inhibitor and an investigational small-molecule of DNA-PK. DNA-PK is a key enzyme that could potentially enhance the efficacy of many commonly used DNA-damaging agents such as radiotherapy and chemotherapy.

Pre-clinical studies have shown that DNA-PK inhibitors can enhance CRISPR/Cas9-mediated gene editing. CRISPR/Cas9 is a technology used to modify genetic sequences and is being investigated for the treatment of various genetic disorders. This collaboration licenses two compounds to study the potential DNA-PK-inhibitor-mediated enhancement of gene editing for the treatment of six genetic diseases included in the license grant to Vertex.

On January 24, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that new data from its clinical programs will be presented at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR) taking place in Houston, Texas, February 20 – 24 (Press release, Gamida Cell, JAN 24, 2019, View Source [SID1234532889]).

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During the meeting, new data will be presented from the ongoing Phase 1 study of NAM-NK, an investigational, cell-based cancer immunotherapy, in patients with non-Hodgkin lymphoma and multiple myeloma. Additionally, there will be two presentations on NiCord, an investigational universal bone marrow donor source, including an oral presentation describing translational data from the completed Phase 1/2 study of NiCord in patients with high-risk hematologic malignancies, or blood cancers. Initial data from a Phase 1/2 study of NiCord in patients with severe aplastic anemia will also be presented.

"We are pleased with our continued progress with NiCord, our late-stage development program in bone marrow transplantation, and we are encouraged by the clinical profile of NAM-NK that is emerging from this first Phase 1 clinical study," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "Collectively, the data that will be presented next month reinforce our belief that our proprietary nicotinamide, or NAM, cell expansion technology has the potential to deliver transformative treatments to patients."

Details about the presentations are as follows:

Time: Wednesday, February 20, 2019, 9:00 a.m. – 5:00 p.m. CT (poster displayed) and 6:45 p.m. – 7:45 p.m. CT (presentation)
Title: First-in-Human Phase I Study of Nicotinamide-Expanded Related Donor Natural Killer Cells for the Treatment of Relapsed/Refractory Non-Hodgkin Lymphoma and Multiple Myeloma
Poster Number: 242
Lead Author: Veronika Bachanova, M.D., Ph.D., associate professor of medicine, division of hematology, oncology and transplantation, University of Minnesota
Location: George R. Brown Convention Center, Level 3, Hall B

Time: Wednesday, February 20, 2019, 9:00 a.m. – 5:00 p.m. CT (poster displayed) and 6:45 p.m. – 7:45 p.m. CT (presentation)
Title: Ex Vivo Nicotinamide-Expanded (NAM-Expanded) Unrelated Cord Blood Transplantation (UCB) for Refractory Severe Aplastic Anemia Results in Rapid Engraftment and Expedites Immune Recovery
Poster Number: 295
Lead Author: Joseph Clara, M.D., Hematology Branch, National Heart, Lung, and Blood Institute
Location: George R. Brown Convention Center, Level 3, Hall B

Time: Saturday, February 23, 2019, 4:45 p.m. – 5:00 p.m. CT (oral presentation)
Title: Rapid and Robust CD4+ and CD8+ T-, NK-, B-Cell, Dendritic Cell, and Monocyte Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation
Abstract Number: 69
Lead Author: Jaap-Jan Boelens, M.D., Ph.D., Chief, Pediatric Stem Cell Transplantation and Cellular Therapies Service, Memorial Sloan Kettering Cancer Center
Location: Hilton Americas Houston, Grand Ballroom G

Abstracts are available on the 2019 TCT Meetings of ASBMT and CIBMTR website.

About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. NAM-NK addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1

About NiCord
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 clinical study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.2 NiCord is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 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 NiCord. For more information on clinical trials of NiCord, please visit www.clinicaltrials.gov.

NAM-NK and NiCord are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On January 23, 2019 Vedanta Biosciences, a clinical-stage company developing a new category of therapies for immune-mediated diseases based on rationally-defined consortia of human microbiome-derived bacteria, reported a publication in Nature that revealed a newly discovered mechanism underlying antitumor immunity that involves human microbiota-driven induction of interferon-gamma-producing (IFNy+) CD8+ T cell accumulation in the gut and tumors (Press release, Vedanta Biosciences, JAN 23, 2019, View Source [SID1234532834]). Led by Vedanta’s scientific co-founder Kenya Honda, M.D., Ph.D., of Keio University School of Medicine, the research also led to the identification and selection of a defined consortium of human microbiome-derived bacterial strains that harnesses this mechanism of antitumor activity and cooperatively potentiates responses to checkpoint inhibitor therapies and immune challenges in general. Based on this research, Vedanta is advancing VE800, a proprietary clinical candidate designed to enhance immune responses against cancer. Vedanta plans to initiate clinical studies in 2019 to evaluate VE800 in combination with Bristol-Myers Squibb’s checkpoint inhibitor OPDIVO (nivolumab).

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"This research demonstrates that specific, human microbiome-derived bacteria assembled rationally into consortia can cooperatively enhance the responses to immune checkpoint inhibitors, which supports our hypothesis that modulating the gut microbiota could be a powerful tool for potentiating immune responses that help fight cancer and infection," said Bernat Olle, Ph.D., Chief Executive Officer of Vedanta Biosciences. "This work also builds upon Dr. Honda’s previous groundbreaking research on the role of the human microbiome in modulating a range of immune responses and provides a robust scientific foundation for our proprietary lead cancer candidate, VE800."

The authors of the Nature paper sought to understand the previously poorly-characterized relationship between the human microbiota and intestinal IFNy+ CD8 T cells, which are critical to innate and adaptive immune responses. In preclinical models, they were able to establish that the number and frequency of these immune cells in the gut depend on the presence of a gut microbiota and are plastic, with specific members of the microbiota promoting their intestinal accumulation in an inducible and reversible manner. The authors went on to identify specific commensal bacterial strains from healthy human donors that spurred the production of IFNy+ CD8+ T cells.

Through rigorous selection, the authors isolated a defined consortium of commensal bacteria derived from the human microbiome that proved most effective at inducing rapid and persistent accumulation of IFNy+ CD8+ T cells. Mice colonized with the defined bacterial consortium demonstrated enhanced therapeutic efficacy in a range of tumor models when given in conjunction with PD-1 or CTLA4 immune checkpoint inhibitors. The strains identified are primarily rare, low-abundance components of the human microbiome, representing a significant opportunity for amplification as a therapeutic strategy.

The research demonstrates for the first time that human microbiome-derived bacterial consortia that cooperatively enhance the responses of immune checkpoint inhibitors can be identified. The authors addressed the challenge of reducing a complex community of human microbiome bacteria down to a few, rationally-defined members that can induce a robust immune potentiation response necessary for an effective cancer immune therapy, and directly linking their activity to pathways that promote antitumor immunity.

The Nature paper also found that human stool samples showed considerable variability in their ability to induce colonic IFNy+ CD8+ T cells. Vedanta’s development process is designed to bypass this variability by using pure, clonal cell banks of well-characterized bacterial strains isolated from healthy humans to produce defined consortia of uniform composition. This eliminates the need to rely on direct sourcing of fecal donor material of inconsistent composition. Vedanta sources bacteria from a vast, extensively characterized collection of 80,000 bacterial isolates obtained from human donors from four continents, which is believed to be the largest collection of human-gut associated bacteria. It then designs high-throughput assays to screen product candidates against a given disease target.

About VE800

VE800 is Vedanta Biosciences’ proprietary oral immuno-oncology product candidate. It consists of a rationally-defined bacterial consortium that activates cytotoxic CD8+ T cells, a type of white blood cell that is the predominant effector in cancer immunotherapy. In preclinical studies, VE800 has been shown to enhance the ability of these T cells to infiltrate tumors, thereby promoting suppression of tumor growth and enhancing survival. Data also suggest that VE800 may enhance the effects of checkpoint inhibitors. Vedanta is evaluating VE800 alone and in combination with checkpoint inhibitors as a potential treatment for patients with advanced or metastatic cancers. In December 2018, Vedanta entered into a clinical trial collaboration to evaluate Bristol-Myers Squibb’s programmed death-1 (PD-1) immune checkpoint inhibitor OPDIVO (nivolumab) in combination with Vedanta’s VE800, in patients with advanced or metastatic cancers. Clinical trials are expected to begin in 2019.