publication date: Nov. 1, 2019

Drugs & Targets

AACR Project GENIE begins five-year research project with $36M in industry funding

Project GENIE (Genomics Evidence Neoplasia Information Exchange), an initiative by the American Association for Cancer Research, is launching a five-year, $36 million research collaboration with nine biopharmaceutical companies to obtain clinical and genomic data from an estimated 50,000 de-identified patients.

The patients are treated at institutions participating in AACR Project GENIE. The additional clinical data furthers the project goals of advancing precision oncology and powering clinical decision making through open and transparent data sharing.

The nine biopharmaceutical companies participating in the collaborative project are:

  • Amgen Inc.

  • AstraZeneca

  • Bayer HealthCare Pharmaceuticals Inc.

  • Boehringer Ingelheim

  • Bristol-Myers Squibb Company

  • Genentech, member of the Roche Group

  • Janssen Research & Development, LLC

  • Merck

  • Novartis

AACR Project GENIE is a publicly accessible international cancer registry of real-world data assembled through data sharing between 19 cancer centers across the world. Through the efforts of strategic partners Sage Bionetworks and cBioPortal, the registry aggregates, harmonizes, and links clinical-grade, next-generation cancer genomic sequencing data with clinical outcomes obtained during routine medical practice from cancer patients treated at these institutions.

Currently, AACR Project GENIE’s registry contains clinical-grade cancer genomic sequencing data from nearly 71,000 patients. These data are linked to a limited set of clinical data, such as age, sex, primary diagnosis, and type of tumor sample analyzed (primary or metastatic).

The new collaboration will greatly expand the scope and accelerate the speed of clinical data collection.

In the first two years, the project will add prior cancer treatments, tumor pathology and clinical outcomes to the clinical data already linked with the genomic profiles of nearly 8,000 bladder, breast, colorectal, lung, pancreatic and prostate cancer patients treated at three of the institutions participating in AACR Project GENIE: Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center and Vanderbilt-Ingram Cancer Center.

In years three through five, this data collection will be expanded to as many cancer types as possible from all active participating institutions.

“Recognizing the importance of the outputs of this project to the broader research and patient communities, and in alignment with the guiding principles of openness, transparency, and inclusion, all data generated will be made publicly available 12 months following data lock,” Shawn M. Sweeney, director of the AACR Project GENIE Coordinating Center, said in a statement.


European Commission approves Astellas’ Xospata indication for relapsed or refractory AML

The European Commission has approved Astellas’ oral once-daily therapy Xospata (gilteritinib) as a monotherapy for the treatment of adult patients with relapsed or refractory (resistant to treatment) acute myeloid leukemia with a FLT3 mutation. Gilteritinib has the potential to improve treatment outcomes for AML patients with two forms of the most common mutation—FLT3 internal tandem duplication and FLT3 tyrosine kinase domain mutation.

This approval is based on results from the phase III ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. Patients treated with gilteritinib had significantly longer overall survival than those who received salvage chemotherapy.

Median OS for patients who received gilteritinib was 9.3 months, compared to 5.6 months for patients who received salvage chemotherapy (Hazard Ratio = 0.64 [95% CI 0.49, 0.83], P=0.0004). Rates of one-year survival were 37% for patients who received gilteritinib, compared to 17% for patients who received salvage chemotherapy.

The EC marketing authorization for gilteritinib in relapsed or refractory FLT3mut+ AML is applicable to the European Union member countries, and is also valid in Iceland, Norway and Liechtenstein. Gilteritinib was designated an orphan medicinal product and also received accelerated assessment from the European Medicines Agency earlier this year, which reduced the timeframe for approval.

Patients’ FLT3mut+ status can change over the course of AML treatment, even after relapse. Due to the poor outcomes associated with FLT3mut+ AML, patients’ FLT3 mutation status may be confirmed to help inform the best treatment approach.


FDA grants Cytotron Breakthrough Device Designation for breast, liver and pancreatic cancers

FDA granted Shreis Scalene Sciences Breakthrough Device Designation for the Cytotron, a CE-marked, whole-body therapeutic medical device.

The Center for Devices and Radiological Health granted the designation.

The company’s designation request stated that “The Cytotron is intended to be used to cause degeneration of uncontrolled growth of tissues. It is indicated for treating protein-linked, abnormally regenerating disorders such as neoplastic disease, by selectively targeting and enabling tissue apoptosis, allowing extended progression-free survival, with pain relief, palliation, improved quality and dignity of life. It is indicated for the treatment of solid tumors of the breast, liver, and pancreas.”

Shreis, while actively pursuing collaborations for clinical trials in the current proposed indications for use, intend to also submit a request for Breakthrough designation in other solid tumors such as adult and pediatric brain tumors, lung cancer, and other life-limiting diseases.


MD Anderson, Ziopharm Oncology to expand TCR-T Program

Ziopharm Oncology Inc. and MD Anderson Cancer Center established a research and development agreement relating to Ziopharm’s Sleeping Beauty immunotherapy program to use non-viral gene transfer to stably express and clinically evaluate neoantigen-specific T-cell receptors in T cells.

“This new agreement is a launch point to expand our TCR library and execute two new clinical trials; a trial for utilizing TCRs from the library targeting hotspot mutations in KRAS, TP53 and EGFR, and a second trial for personalized TCRs targeting patient-specific neoantigens,” Ziopharm CEO Laurence Cooper said in a statement.

Under the agreement, Ziopharm commits to fund an additional $20 million for this expanded work in the TCR-T program through 2023, as well as certain milestone payments for clinical development or regulatory approval in the U.S., European Union, Japan and the rest of the world. The funding for this new agreement was included within the budget forecast provided by Ziopharm in its second quarter 2019 financial results news release and webcast commentary.

MD Anderson will receive low, single-digit royalties on net sales in the U.S. and international markets, as well as warrants for Ziopharm common stock which vest upon achievement of clinical milestones. According to institutional guidelines, MD Anderson has implemented an Institutional Conflict of Interest Management and Monitoring Plan to manage this research.

This new agreement expands the relationship between Ziopharm and MD Anderson, established under a 2015 research agreement related to CD19-specific CAR T. Earlier this month, FDA cleared an IND application for a phase I clinical trial to evaluate CD19-specific CAR T, manufactured and infused within two days of gene transfer using Ziopharm’s rapid personalized manufacture, as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas. Ziopharm has approximately $20 million of pre-funded R&D at MD Anderson under the prior agreement, which may now be used under the new agreement, for both the CAR T or TCR-T initiatives.

Ziopharm has entered a lease agreement with MD Anderson to access laboratory and office space within the institution’s campus. This new facility will serve as home for Ziopharm’s expanded Houston office, under the direction of Eleanor de Groot, of GM Cell Therapy, and Drew Deniger, head of Ziopharm’s TCR-T cell therapy program.

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