On February 19, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported an agreement with Editas Medicine, Inc., a leading genome editing company, to evaluate the potential use of Editas Medicine’s CRISPR technology to edit NAM-NK cells, which are natural killer cells that have been expanded using Gamida Cell’s proprietary nicotinamide-based, or NAM, technology (Press release, Gamida Cell, FEB 19, 2019, View Source [SID1234533505]). Through this agreement, the companies aim to discover optimized NAM-NK cells that could be used to improve the treatment of hematologic malignancies (blood cancers) and solid tumors.

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"We are encouraged by the early data generated in the Phase 1 study of NAM-NK as an investigational therapy for patients with non-Hodgkin lymphoma and multiple myeloma, and we are pleased to have the opportunity to accelerate and broaden our NAM-NK research efforts through this agreement," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "By leveraging the collective expertise of the Gamida Cell and Editas Medicine teams, we hope to enhance the efficacy of NAM-NK cells through CRISPR editing and potentially bring life-changing immunotherapy treatments to patients."

"Natural killer cells are increasingly recognized as a potential breakthrough approach to treating various cancers. This agreement with Gamida Cell enables us to combine our industry-leading genome editing platform with Gamida Cell’s proprietary NAM-NK cells in an effort to develop best-in-class cellular medicines," said Charles Albright, Ph.D., chief scientific officer of Editas Medicine.

Under the terms of the agreement, Gamida Cell and Editas Medicine will engage in joint research to evaluate unnamed targets by combining Gamida Cell’s proprietary NAM-based cell expansion technology with Editas Medicine’s CRISPR technology. The research initiative is focused on exploring the potential to edit NAM-NK cells to further optimize their tumor-killing properties, and compare the function of the edited and unedited cells in inducing NK cell tumor clearance.

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

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

On February 19, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, reported that Professor Persis Amrolia, Consultant in Bone Marrow Transplant at Great Ormond Street Hospital (GOSH) and NIHR Research Professor of Transplantation Immunology at University College London (UCL) Great Ormond Street Institute of Child Health, presented updated data from the ongoing Phase 1 CARPALL trial of AUTO1 in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 1st European CAR T Cell Meeting held in Paris, France, February 14-16, 2019 (Press release, Autolus, FEB 19, 2019, View Source [SID1234550832]).

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Enrolled patients had a median age of 9 years with a median of 4 lines of prior treatment. Seventeen patients were enrolled, and 14 patients received an infusion of CAR T cells. Ten of 14 patients had relapsed post allogeneic stem cell transplant. Eight patients were treated in second relapse, 5 in >second relapse and 3 had relapsed after prior blinatumomab or inotuzumab therapy. Two patients had ongoing CNS disease at enrollment.

Updated safety results
This data set confirms that AUTO 1 induces no severe cytokine release syndrome (CRS) (Grade 3-5). Nine patients experienced Grade 1 CRS, and 4 patients experienced Grade 2 CRS. No patients required tociluzumab or steroids. As previously reported, one patient experienced Grade 4 neurotoxicity; there were no other reports of severe neurotoxicity (Grade 3-5). The mean cumulative exposure to AUTO1 CAR T cells in the first 28 days as assessed by AUC was 1,721,355 copies/µg DNA. Eleven patients experienced cytopenia that was not resolved by day 28 or recurring after day 28: 3 patients Grades 1-3 and 8 patients Grade 4. Two patients developed significant infections, and 1 patient died from sepsis while in molecular complete response (CR).

Updated efficacy results
With a single dose of CAR T cells at 1 million cells/kg dose, 12/14 (86%) achieved molecular CR. Five patients relapsed with CD19 negative disease. Event free survival (EFS) based on morphological relapse was 67% (CI 34-86%) and 46% (CI 16-72%) and overall survival (OS) was 84% (CI 50-96%) and 63% (CI 27-85%) at 6 and 12 months, respectively.

CAR T cell expansion was observed in all responding patients (N=12), with CAR T cells comprising up to 84% of circulating T cells at the point of maximal expansion. The median persistence of CAR T was 215 days.

The median duration of remission in responding patients was 7.3 months with a median follow-up of 14 months. Five of 14 patients (37%) remain in CR with ongoing persistence of CAR T cells and associated B cell aplasia.

"AUTO1 combines a high molecular CR rate with excellent persistence and a good safety profile in pediatric acute B cell leukemia patients," said Professor Amrolia.

About AUTO1
AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety – while maintaining similar levels of efficacy – compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells’ abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights from UCL Business plc (UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.

For information about the CARPALL trial, visit View Source

About Pediatric Acute Lymphoblastic Leukemia (ALL)
According to the American Cancer Society, ALL is the most common cancer diagnosed in children, with approximately 3,400 new cases diagnosed in the United States each year. Pediatric ALL occurs when the bone marrow makes too many immature lymphocytes, which are a type of white blood cell. The current standard of care for pediatric ALL patients is combination chemotherapy. Although pediatric patients typically respond well to first-line treatment, 10 to 20% of total patients relapse with chemotherapy-resistant disease, leading to a significant unmet need in pediatric patients with high-risk relapsed or refractory ALL.

On February 19, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. based pharmaceutical company with a platform to develop and accelerate the launch of pharmaceutical products and innovative therapeutics in China, U.S., and throughout the world, reported that the National Medical Products Administration (NMPA) has approved the Company’s Clinical Trial Application (CTA) to allow for a confirmatory clinical trial to evaluate the efficacy and safety of ibritumomab tiuxetan injection (ZEVALIN) (Press release, CASI Pharmaceuticals, FEB 19, 2019, View Source [SID1234533487]). Ibritumomab tiuxetan (ZEVALIN) is a U.S. Food and Drug Administration (FDA)-approved product indicated for the treatment of patients with relapsed or refractory, low-grade or follicular B-cell non-Hodgkin’s lymphoma (NHL) and is currently marketed in the U.S. by Spectrum Pharmaceuticals, Inc. (Spectrum). The Company previously acquired greater China rights to ZEVALIN from Spectrum and intends to launch ZEVALIN in China as soon as the confirmatory trial is completed and marketing approval is received from the NMPA.

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The Company currently is addressing certain requirements provided by the Center for Drug Evaluation (CDE), a part of the NMPA; upon satisfying those requirements, the Company will start the ZEVALIN clinical development program in China.

Ibritumomab tiuxetan (ZEVALIN) is also indicated for the treatment of patients with previously untreated follicular non-Hodgkin’s Lymphoma who achieve a partial or complete response to first-line chemotherapy. Ibritumomab tiuxetan (ZEVALIN) builds on the combined therapeutic effects of a monoclonal antibody directed against the CD20 antigen, augmented with a beta-emitting radioisotope. Rituximab and rituximab biosimilars target the same CD20 antigen.

Wei-Wu He, Ph.D., the Company’s Executive Chairman commented, "We are extremely pleased with NMPA’s approval to start the confirmatory clinical trial for ZEVALIN in China. This is another important milestone for CASI as the approval further demonstrates our team’s regulatory strength in working with the NMPA and the effective cross-border operations of our teams in the U.S. and China."

On February 19, 2019 Incyte Corporation (Nasdaq:INCY) reported that it will present at the following investor conferences during the month of March (Press release, Incyte, FEB 19, 2019, View Source [SID1234533470]):

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Cowen and Company 39th Annual Health Care Conference onMonday, March 11, 2019 at 12:00 pm (EDT) in Boston; and
Barclays Global Healthcare Conference on Wednesday, March 13, 2019 at 9:00 am (EDT) in Miami
The presentations will be webcast live and can be accessed at www.incyte.com in the Investors section under "Events and Presentations." Investors interested in listening to the live webcast should log on before the start time in order to download any software required.

On February 19, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT) reported it will report its audited 2018 fourth quarter and full-year financial results and provide a corporate update on February 25, 2019 (Press release, Corcept Therapeutics, FEB 19, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announce-audited-financial-results-provide [SID1234533488]). The company will host a conference call that day at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time).

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Conference Call Information

To participate, dial 1-888-220-8451 from the United States or 1-323-794-2588 internationally approximately ten minutes before the start of the call. The passcode will be 6598298.

A replay will be available through March 11, 2019 at 1-888-203-1112 from the United States and 1-719-457-0820 internationally. The passcode will be 6598298.