On May 02, 2016 Kite Pharma, Inc. (Nasdaq:KITE) ("Kite"), a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) granted orphan drug designations to Kite’s lead product candidate, KTE-C19, for the treatment of primary mediastinal B cell lymphoma (PMBCL), mantle cell lymphoma (MCL), follicular lymphoma (FL), acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL) (Press release, Kite Pharma, MAY 2, 2016, View Source [SID:1234511757]). Kite previously received orphan drug designation for KTE-C19 for the treatment of diffuse large B cell lymphoma (DLBCL) in both the U.S. and the EU, as well as orphan drug designations in the EU for PMBCL, MCL, FL, ALL, and CLL.

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"Kite has now secured Orphan Drug Designations in both the U.S. and the EU for the leading indications in hematological malignancies. This is an important regulatory milestone as we further our development of KTE-C19 in advanced hematological cancers, including the planned initiation of clinical studies in CLL and FL patients in 2017," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "We are encouraged by the progress of our four ongoing ZUMA clinical trials and our ability to manufacture clinical supply of KTE-C19 in our own Santa Monica facilities. We look forward to working closely with the FDA to bringing this potentially transformative therapy to patients with a significant unmet need."

Orphan drug designation is granted by the FDA’s Office of Orphan Products Development (OOPD) to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The OOPD provides incentives for sponsors to develop products for rare diseases which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same biologic for the same indication for seven years, except in limited circumstances.

On May 2, 2016 BioInvent International (BINV) reported it has initiated a Phase I/IIa study that will evaluate the safety and tolerability, and explore the preliminary efficacy, of TB-403 for the treatment of relapsed or refractory medulloblastoma, a rare, life-threatening brain tumor that mainly affects children (Press release, BioInvent, MAY 2, 2016, View Source [SID:1234511754]).

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Today’s study initiation follows the earlier announced partnership between BioInvent, its TB-403 project partner Oncurious, and the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC) in the US. NMTRC is a collaboration of 25 US academic medical centers, teaching hospitals and other entities, with the purpose of facilitating and conducting collaborative research activities and investigations of new treatments for neuroblastoma, medulloblastoma and other pediatric cancers.

Headquartered at the Helen DeVos Children’s Hospital in Grand Rapids, MI, USA, NMTRC is the key clinical trial partner for this Phase I/IIa study. The study aims at recruiting a minimum of 27 patients, with first results expected to be reported in 2017.

TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF) which is expressed in several types of cancer, including medulloblastoma. A paper in Cell in February 2013 (Cell, 152, 1065-76, 2013), highlighted for the first time that PlGF plays a role in the growth and spread of medulloblastoma. The paper was based on pre-clinical research conducted by Prof Rakesh Jain from the Massachusetts General Hospital at Harvard Medical School (Boston) and the team of Prof Peter Carmeliet from Vesalius Research Center, VIB Katholieke Universitiet, Leuven, Belgium.

Treatment with TB-403 in pre-clinical models for medulloblastoma has demonstrated beneficial effects on tumor growth and survival. TB-403 has been shown to have a favorable safety profile in previous clinical trials in healthy volunteers and adult patients with various types of solid tumors.

The drug candidate TB-403 is jointly owned by BioInvent and the Belgian biopharma company Oncurious NV.

Michael Oredsson, CEO of BioInvent commented, "The initiation of this study is a key step in the development of this new targeted potential treatment for children with devastating brain cancers. We are looking forward to be working with NMTRC’s experienced investigator network to complete this study in a timely manner."

Dr Patrik De Haes, Executive Chairman of Oncurious, commented, "We are pleased to announce the initiation of this important Phase I/IIa study with TB-403, a key corporate milestone for Oncurious. Given the encouraging pre-clinical data generated with TB-403 in Leuven and Boston, we are hopeful that this novel antibody, targeting placental growth factor, will provide an improved treatment option for children afflicted with this lethal brain tumor."

Giselle Sholler, MD MSC, of NMTRC, commented, "We are delighted to be starting this study, which is very much in line with our mission to bring forward new therapies that could improve the quality of life and survival of children with medulloblastoma. We believe that with its unique mode of action TB-403 could offer an important solution to address this tremendous unmet medical need."

On May 02, 2016 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported that it has granted an exclusive license to Bayer for the development and commercialization of therapeutic antibodies combining the Company’s prostate specific membrane antigen (PSMA) antibody technology with Bayer’s targeted thorium conjugate technology (Press release, Progenics Pharmaceuticals, MAY 2, 2016, View Source [SID:1234511743]).

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"While there have been recent advances in the treatment of prostate cancer, there remains a great need for therapies that can more specifically target primary and metastatic prostate cancer," said Mark R. Baker, Chief Executive Officer of Progenics. "This license agreement with Bayer further validates the value of our PSMA antibody technology in the development of targeted cancer treatments. We are pleased that Bayer recognizes the potential of our technology."

PSMA is a protein that has been found to be amplified on the surface of >95% of prostate cancer cells and is a validated target for the detection of primary and metastatic prostate cancer. Antibody-thorium conjugates are comprised of a radioactive alpha emitter, thorium-227, linked to an antibody, in this case PSMA. The thorium conjugates bind to the surface of tumor cells and emit alpha particles that destroy tumor cells by inducing DNA double-strand breaks, with no need for uptake into the cells for efficacy.

Per the terms of the license agreement, Progenics will receive an upfront fee and could receive additional potential clinical and regulatory development milestones. If approved, Progenics is entitled to single digit royalties and sales milestone payments.

On May 2, 2016 BIND Therapeutics, Inc. (NASDAQ:BIND), a biotechnology company developing targeted and programmable therapeutics called ACCURINS, reported that it has elected to file a voluntary petition under Chapter 11 of the Bankruptcy Code in the U.S. Bankruptcy Court for the District of Delaware (Press release, BIND Therapeutics, MAY 2, 2016, View Source [SID:1234511739]).

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"We believe this decision is in the best interests of the company and its stockholders," said Andrew Hirsch, president and chief executive officer, BIND Therapeutics. "The protections afforded by Chapter 11 provide for an orderly process and additional time that enables us to pursue the strategic and financial alternatives that are in process. The filing minimizes the impact from the recent demand by our lender, Hercules Technology III, L.P, for accelerated repayment of our outstanding loan. Our current cash and assets exceed the loan amount, and we are current on our regularly scheduled repayment obligations. Through this process, we expect to be able to maintain ongoing financing activities and collaborator obligations while moving our R&D initiatives and pipeline forward."

BIND intends to continue to manage and operate its business under the jurisdiction of the Bankruptcy Court and in accordance with the applicable provisions of the Bankruptcy Code and the orders of the Bankruptcy Court. The Company is working with an investment bank to review financial and strategic alternatives with the goal of maximizing stockholder value. Potential alternatives to be explored further and evaluated during the review process may include raising additional capital, a strategic collaboration with one or more parties, or the licensing, sale or divestiture of some, or all, of the Company’s proprietary technologies.

BIND plans to continue its development and collaboration activities in accordance with its current innovative medicines strategy throughout this process.

On May 2, 2016 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported data from the first cohort of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery (Press release, Celsion, MAY 2, 2016, View Source [SID:1234511730]).

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In the first three patients dosed, GEN-1 plus standard chemotherapy produced excellent results, with no dose limiting toxicities and highly promising efficacy signals leading to successful surgical outcomes. The three patients were treated at the University of Alabama at Birmingham.

"These results, while early, are very impressive and speak to the potential of GEN-1 to improve patient outcomes in ovarian cancer," said Ronald D. Alvarez, M.D., Professor, Division of Gynecologic Oncology at the University of Alabama at Birmingham. "In particular, we see improvements across a number of important and meaningful measures used to assess ovarian cancer, which reinforce our confidence in this IL-12 immunotherapy approach and provide a strong rationale for continued development of GEN-1 for the treatment of ovarian cancer."

OVATION Study – First Cohort Results

Of the three patients treated in the first cohort, two patients demonstrated stable disease (SD) and one patient demonstrated a complete response (CR), as measured by RECIST criteria.
All patients had successful resections of their tumors, with two patients having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed, and one patient with an optimal R1 resection.
One patient demonstrated a pathological complete response (pCR). pCRs are typically seen in less than 7% of patients receiving neoadjuvant chemotherapy followed by surgical resection, and have been associated with a median overall survival (OS) of 72 months, which is more than three years longer than those who do not experience a pCR&supl;.
All patients experienced a dramatic > 96% drop in their CA-125 protein levels as of their most recent study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells. A 50% reduction in CA-125 levels is considered meaningful. All patients’ CA-125 levels were below the standard cutoff level of 35 U/mL.
"These results build on the impressive clinical findings we observed in the GOG Study as well as the translational data from this same study reported earlier this year. As we move closer towards initiating a Phase I/II trial to evaluate the synergistic anti-cancer effects of GEN-1 together with Avastin and Doxil, these results also provide strong validation for the potential of GEN-1 in ovarian cancer," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "We will continue to evaluate the maturing dataset from the OVATION study, and may evaluate ways to leverage insights from this trial to help accelerate the clinical development of this highly promising therapy and inform the design of future studies."

The OVATION Study will continue into 2016 at higher doses of GEN-1 with the goal to identify a safe, tolerable and therapeutically active dose of GEN-1 by recruiting and maximizing an immune response. Celsion has initiated four clinical sites at the University of Alabama at Birmingham; Oklahoma University Medical Center; Washington University in St. Louis and the Medical College of Wisconsin. The trial is designed to enroll three to six patients per dose cohort and will evaluate safety and efficacy and attempt to define an optimal dose. Future studies of GEN-1 will include a Phase I/II study combining GEN-1 with Avastin and Doxil. With the second cohort fully enrolled, Celsion expects to complete the OVATION Study this year.

GOG Study – GEN-1 Data

Celsion also recently announced clinical data from a dose-escalation study evaluating GEN-1 plus Doxil in platinum resistant ovarian cancer (the GOG Study). In the GOG Study, at the highest dose level, GEN-1 plus Doxil produced an objective response rate (ORR) of 29%. This compares favorably to the data from the Phase 3 AURELIA trial in platinum-resistant ovarian cancer, which demonstrated that Avastin plus chemotherapy produced an ORR of 27%. Historical data for trials evaluating Doxil monotherapy in platinum resistant ovarian cancer suggest an ORR of only 8% to 12%.

Translational data from the GOG Study reported in January 2016 is highlighted below:

Intraperitoneal administration of GEN-1 in platinum-resistant ovarian cancer patients resulted in a significant increase in IL-12 levels in peritoneal fluid samples. IL-12 levels were quantifiable in 91% of evaluable fluid samples collected post GEN-1 treatment. None of the evaluable pre-treatment peritoneal fluid samples had any detectable IL-12 levels.
The IL-12 levels were detectable for at least seven days after GEN-1 treatment.
In comparison to peritoneal fluid, the IL-12 levels in plasma samples (systemic exposure) following GEN-1 treatment were not detectable or were very low in quantity.
Significant increases in levels of IFN-γ, a key downstream mediator of IL-12 action, were observed in peritoneal fluid but not in plasma samples. At least a 5-fold increase above pre-treatment level in IFN- γ was observed in most samples, with the highest increase observed at 120-fold. Similar results were observed with TNF-α levels, with the highest increase observed at 77-fold over pre-treatment control.
About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy in patients with peritoneally metastasized ovarian cancer, and recently completed a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer.