On December 03, 2015 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported that it has completed enrollment in its Phase 2b clinical trial of its ultra-orphan radiotherapeutic candidate Azedra for the treatment of malignant or recurrent pheochromocytoma and paraganglioma in accordance with the Special Protocol Assessment (SPA) agreement with U.S. Food and Drug Administration (FDA) (Press release, Progenics Pharmaceuticals, DEC 3, 2015, View Source [SID:1234508392]).

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Progenics will allow enrollment to continue through the end of December so that patients already identified as potential candidates may have the opportunity to participate in the trial. The Company expects to report top-line data in the second half of 2016.

"Achieving the number of enrolled patients required under the SPA is a major milestone for Progenics and the Azedra program, a novel therapeutic candidate that has the potential to be the first approved therapy in the U.S. for the treatment of malignant or recurrent pheochromocytoma and paraganglioma," said Mark Baker, Chief Executive Officer of Progenics. "The trial, if positive, will provide the basis for a regulatory submission to the FDA, and we look forward to building on the impressive data reported to date as we advance Azedra as a treatment option for these very rare and life threatening tumors."

The open-label trial is designed to evaluate the efficacy and safety of the administration of two therapeutic doses of Azedra in patients with malignant relapsed/refractory pheochromocytoma or paraganglioma, ultra-orphan cancers with limited treatment options. The primary objective of the study is to determine the clinical benefit of Azedra based on the proportion of study participants with a reduction of all antihypertensive medication by at least 50% for at least six months. The SPA requires that 25% of 58 evaluable patients achieve the primary endpoint.

Azedra has received Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA.

About Azedra

Azedra (Ultratrace iobenguane I-131) is a radio-therapeutic with Breakthrough Therapy Designation and FDA Fast Track status currently being studied in a phase 2b registrational trial under a Special Protocol Assessment with the U.S. Food and Drug Administration using a surrogate marker registrational endpoint.

About Pheochromocytoma and Paraganglioma

Pheochromocytomas and paragangliomas are rare neuroendocrine tumors that arise from cells of the sympathetic nervous system. When pheochromocytomas are located outside the adrenal glands, they are called paragangliomas. Standard treatment options for these tumors include surgery, palliative therapy and symptom management. Malignant and recurrent pheochromocytomas and paragangliomas may result in unresectable disease with a poor prognosis, representing a significant management challenge with very limited treatment options and no approved anti-tumor therapies.

On December 3, 2015 bluebird bio, Inc. (Nasdaq: BLUE) and ViroMed Co., Ltd. reported that they have entered into an exclusive license agreement to research, develop and commercialize chimeric antigen receptor (CAR) T cell therapies using ViroMed’s proprietary humanized antibody to an undisclosed cancer target for solid tumors (Press release, bluebird bio, DEC 3, 2015, View Source [SID:1234508390]).

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Under the terms of the agreement, ViroMed will provide bluebird bio exclusive rights to its novel humanized antibody to the target, and bluebird bio will leverage its proprietary lentiviral gene therapy platform and CAR T capabilities to develop CAR T therapies against the target. Financial terms of the agreement include a $1 million upfront payment and subsequent milestone payments to ViroMed, which together could total over $48 million per licensed product if certain development and regulatory milestones are achieved. ViroMed is also eligible to receive tiered royalties on product sales. bluebird bio will conduct and fund clinical development as well as regulatory and commercial activities.

"Over the course of 2015, bluebird has continued to expand its immuno-oncology research and preclinical programs, building a broad pipeline of multiple targets in solid and hematologic malignancies. We are pleased to enter this agreement with ViroMed and add their novel target to our growing pipeline," said Rob Ross. M.D., head of oncology, bluebird bio. "We believe that this target, combined with our lentiviral vector and manufacturing expertise, position us as a leader in delivering potentially transformative T cell-based immunotherapies to patients."

"CAR T technology has been gaining worldwide attention in recent years as an innovative technology backed by highly promising results from recent clinical studies," said Seung Shin Yu, Ph.D., director of new business planning, ViroMed. "bluebird bio owns critical technologies for the development of CAR T therapeutics as well as manufacturing capabilities and know-how for commercialization, making them an ideal partner for us. This agreement is a testament to ViroMed’s competitive edge in innovative gene therapy technologies like CAR T technology."

On December 3, 2015 Kite Pharma, Inc. (Nasdaq:KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, and GE Global Research (NYSE:GE), the centralized research and development hub for GE, reported that they have formed a strategic research collaboration to develop a next-generation, functionally integrated and automated manufacturing system for engineered T cell therapy (Press release, Kite Pharma, DEC 3, 2015, View Source [SID:1234508387]).

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Kite and GE Global Research aim to change the paradigm of engineered T cell therapy manufacturing by accelerating the development of automation technologies that have the potential to reduce cost, improve speed, and minimize variability. As a result, automated manufacturing technologies could enable more widespread availability of engineered T cell therapies.

This collaboration brings together complementary capabilities from each company, including Kite’s scientific leadership in T cell biology and manufacturing and GE Global Research’s deep expertise in the design and development of innovative manufacturing applications for the biopharmaceutical industry.

"We are excited to announce a strategic collaboration with GE Global Research, a world leader in medical technologies and a company that shares our commitment to the future of T cell-based immunotherapy," said Arie Belldegrun, M.D., FACS, Chairman, President and Chief Executive Officer of Kite. "GE Global Research is the ideal partner to realize our vision to advance engineered T cell manufacturing to a functionally integrated and fully automated process. This unique partnership further positions us to deliver on the promise of T cell-based cancer immunotherapy for patients worldwide."

Michael Idelchik, Vice President for Advanced Technologies at GE Global Research, said, "Cell-based immunotherapy has tremendous potential and could fundamentally change the way various diseases are treated. Together with Kite, a scientific pioneer in the field, we are focused on advancing innovation, delivering end-to-end manufacturing solutions, and building a robust ecosystem that supports widespread adoption of these important therapies and the industry as a whole."

Under the terms of the agreement, Kite and GE Global Research will each contribute resources and relevant expertise to the partnership. Further terms of the agreement are not being disclosed.