On April 6, 2017 Kite Pharma, Inc., (Nasdaq:KITE) reported that it won the Clinical Trial Result of the Year award for ZUMA-1, its pivotal CAR-T trial of axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma (NHL) at the Clinical and Research Excellence (CARE) Awards (Press release, Kite Pharma, APR 6, 2017, View Source [SID1234518500]). The award recognizes clinical achievements in the pharmaceutical industry and contribution to the advancement of therapies for unmet medical needs.

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"This award is an important acknowledgement of the ZUMA-1 trial, the first multicenter CAR-T therapy trial in aggressive NHL, and the potential of CAR-T therapy to significantly transform the treatment of cancer," said Arie Belldegrun, M.D., FACS, Chairman, President and Chief Executive Officer of Kite. "We thank the Kite research and development team for their dedication and congratulate them on this well-deserved recognition for their central role in advancing the development of this potentially paradigm changing treatment."

The study met the primary endpoint of objective response rate (ORR) recorded after a single infusion of axicabtagene ciloleucel with 82 percent (p < 0.0001). At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response (CR). These results from 101 patients demonstrate the treatment effect of axicabtagene ciloleucel in diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL), which are types of aggressive NHL.

The most common grade 3 or higher adverse events included anemia (43 percent), neutropenia (39 percent), decreased neutrophil count (32 percent), febrile neutropenia (31 percent), decreased white blood cell count (29 percent), thrombocytopenia (24 percent), encephalopathy (21 percent) and decreased lymphocyte count (20 percent). As compared to the interim analysis, grade 3 or higher cytokine release syndrome (CRS) decreased from 18 percent to 13 percent and neurologic events decreased from 34 percent to 28 percent. There were three deaths throughout the course of the trial not due to disease progression, of which two events, were deemed related to axicabtagene ciloleucel. There were no cases of cerebral edema.

Full data from the primary analysis of ZUMA-1 were most recently presented at the 2017 American Association of Cancer Research Annual Meeting in Washington, D.C. in April. In December 2015, axicabtagene ciloleucel received Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for DLBCL, TFL, and PMBCL. Kite completed its submission of a Biologics License Application (BLA) for axicabtagene ciloleucel with the FDA in March 2017 and, if approved, plans to commercially launch axicabtagene ciloleucel in 2017.

The awards, presented by Informa’s Pharma Intelligence, were announced at a ceremony in Boston on April 5, 2017.

ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

On Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, reported that two abstracts on luspatercept have been accepted for presentation at the 14th International Symposium on Myelodysplastic Syndromes (MDS) in Valencia, Spain on May 3-6, 2017 (Press release, Acceleron Pharma, APR 6, 2017, View Source [SID1234518497]).

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The oral presentation will include data from a Phase 2 study with luspatercept in lower-risk MDS populations, including first-line ring sideroblast-negative and -positive subpopulations. Luspatercept is being developed under a global partnership with Celgene.

Oral presentation

Title:
Luspatercept Response in New Subpopulations of Patients with Lower-Risk Myelodysplastic Syndromes (MDS): Update of the PACE Study (Abstract #17-0245)
Session: Oral Session 3 (Valencia Conference Center, Auditorium 1)
Date: Saturday, May 6th
Time:
9:15 a.m. CEST
Poster presentation

Title:
Pharmacokinetics and Exposure-Response Relationship of Luspatercept in Patients with Anemia Due to Lower-Risk MDS: Preliminary Results from Phase 2 Studies (Abstract #17-0112)
Session: 637. Myelodysplastic Syndromes – Clinical Studies: Poster I (Valencia Conference Center)
Date: Thursday, May 4th to Saturday, May 6th
The luspatercept clinical poster and slides from the oral presentation will be available immediately following the presentations at the conference in the "Science" section on Acceleron’s website, www.acceleronpharma.com.

About Luspatercept

Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept regulates late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Phase 3 clinical trials are underway to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the "MEDALIST" study) and in patients with beta-thalassemia (the "BELIEVE" study). For more information, please visit www.clinicaltrials.gov.

On April 4, 2017 Michigan State University (MSU) researchers presented promising cancer therapy results at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) International Meeting in Washington, DC. This novel technology, AdVCA0848, activates the stimulator of interferon genes (STING) pathway to delay tumor growth in a B16 melanoma model, promoting beneficial anti-tumor responses (Press release, Venn Therapeutics, APR 5, 2017, View Source [SID1234519737]).

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Supported by the immuno-oncology company Venn Therapeutics, this research (session MS.CL06.01 and abstract number 2994) is led by Dr. Andrea Amalfitano, Ph.D., D.O., Osteopathic Heritage Foundation Endowed Professor of Pediatrics, Microbiology and Molecular Genetics at MSU, and Dr. Chris Waters, Associate Professor of Microbiology and Molecular Genetics at MSU.

Data showed that a single intra-tumoral treatment with AdVCA0848 rapidly inhibited tumor growth and significantly improved animal survival when compared to repeat dosing of an anti-PD1 checkpoint inhibitor. AdVCA0848 also generated high intracellular concentrations of STING agonists that were sustained over several days, reducing the need for multiple treatments. Previously published studies demonstrated that AdVCA0848 induces the production of multiple inflammatory cytokines, likely supporting the reported anti-tumor responses while showing little to no evidence of systemic toxicity.

"This research begins to demonstrate that AdVCA0848 is both a safe and potent stimulator of the STING pathway, and this unique combination promotes safe induction of impressive anti-tumor responses in vivo," said Dr. Amalfitano. "We are excited to further develop this novel immunotherapy and advance it rapidly to human clinical trials."

In general, these results provide evidence that AdVCA0848 is a novel, off the shelf STING agonist that can potentially improve anti-tumor responses in melanoma patients, either as a standalone therapeutic or in combination with currently approved therapies.

"These data are very encouraging. This research highlights a strong STING asset, as the characteristics of AdVCA084 overcome many hurdles that STING-targeting small molecules currently face," said Dr. Edith Janssen, Associate Professor, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, and Chair of the Scientific Advisory Board for Venn Therapeutics. "We are excited to take this asset into clinical trials for further development and improve therapeutic outcomes for cancer patients."

This research is made possible in part through sponsored research and licensed technology partnerships with Michigan State University.

"Partnerships with companies like Venn Therapeutics allow us to work together to move bright ideas to the marketplace and therapies to patients even faster," said Anne C. DiSante, CLP, Associate Director of MSU Technologies. "That’s why translational research is remarkable: we can work collaboratively nationally, even internationally, to enhance human health and well-being."

To learn more about research underway at the MSU Department of Microbiology and Molecular Genetics, visit View Source

Venn Therapeutics is an immuno-oncology company focused on developing novel, best-in-class drugs that transform tumor-resident innate immune cells to an anti-tumor phenotype reversing the immunosuppressive microenvironment found within tumors. Venn believes this approach will expand the number of patients who can be effectively treated with immuno-oncology modalities. For more information visit www.VennTherapeutics.com

The MSU Innovation Center combines innovation, technology transfer, start-up support, and a portfolio of dedicated business and community partnerships to bring cutting-edge ideas to the marketplace. Composed of Business-CONNECT, MSU Technologies and Spartan Innovations, the MSU Innovation Center stewards ideas from concept to product, launching more than 150 discoveries into patented products and start-up businesses annually. Learn more at View Source