On January 07, 2016 Kite Pharma, Inc. (NASDAQ:KITE) reported that it has entered into a Cooperative Research and Development Agreement (CRADA) with the NCI for the research and clinical development of a fully human anti-CD19 chimeric antigen receptor (CAR) product candidate for the treatment of B-cell lymphomas and leukemias (Press release, Kite Pharma, JAN 7, 2016, View Source [SID:1234508683]). Under the CRADA, Kite will collaborate with James (Jim) N. Kochenderfer, M.D., an investigator in the Experimental Transplantation and Immunology Branch of the NCI, to evaluate this product candidate in a Phase 1 clinical study this year.

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In addition, the newly established CRADA will focus on the development of next-generation CAR programs directed against other novel antigens for the treatment of B-cell lymphomas and leukemias. Kite will also continue to advance multiple CAR and T cell receptor (TCR) programs under its existing CRADA with the Surgery Branch of the NCI, led by Dr. Rosenberg, a recognized pioneer in immuno-oncology and special advisor to Kite.

"We are delighted to expand our partnership with the NCI by working with the Experimental Transplantation and Immunology Branch. Our close collaboration with the Surgery Branch over the past three years has enabled us to advance the science behind T cell therapy and to expand our clinical product candidate portfolio at Kite. The new CRADA is a natural step in our life cycle management strategy to further improve the success of CAR therapy for patients with advanced B-cell malignancies," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer.

"Dr. Kochenderfer is a world renowned expert in T cell therapy who pioneered innovative CAR T therapies for patients with aggressive non-Hodgkin lymphoma," added David Chang, M.D., Ph.D., Chief Medical Officer and Executive Vice President of Research and Development. "His seminal research in anti-CD19 CAR T cell therapy has been instrumental for our lead product candidate, KTE-C19, which is currently in four Kite-sponsored clinical trials in B-cell malignancies. We look forward to expanding our successful collaboration on next generation technologies with Jim."

On January 7, 2016 Celsion Corporation (NASDAQ: CLSN), reported new translational data from its Phase 1b study of GEN-1 in patients with platinum-resistant ovarian cancer (Press release, Celsion, JAN 7, 2016, View Source [SID:1234508682]). GEN-1 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. The new data demonstrate that intraperitoneally-administered GEN-1 produces an immunologically distinct IL-12 protein that is localized at the tumor site and lasts for up to one week after a single treatment. Furthermore, concomitant increases in IFN-γ and TNF- α indicate that the IL-12 produced following treatment with GEN-1 treatment is immunologically active. Celsion Corporation is a fully integrated oncology company focused on the development of a portfolio of innovative cancer treatments, including directed chemotherapies, immunotherapies and RNA- or DNA-based therapies for the treatment of cancer and other difficult-to-treat diseases.

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"We now have clear clinical evidence that treatment of platinum-resistant ovarian cancer with GEN-1 produces a sustained, localized immune response, which then translates to the compelling activity and tolerability profile we have seen in early clinical studies," said Khursheed Anwer, Ph.D., executive vice president and chief scientific officer of Celsion. "These data also provide additional evidence of GEN-1’s ability to overcome the limitations associated with recombinant IL-12, which has a very short half-life and results in high systemic levels of IL-12, leading to potentially severe toxicities. We plan to build on these data and advance our understanding of GEN-1’s mechanism of action further with additional translational research as part of our ongoing OVATION Study."

The Phase 1B dose escalating study enrolled 16 patients with platinum-resistant ovarian cancer and evaluated the safety, tolerability and efficacy of GEN-1 in combination with pegylated doxorubicin as well as the effect of intraperitoneal injection of GEN-1 on IL-12 and tumor cytokine levels. Patients received pegylated liposomal doxorubicin on day 1 and GEN-1 on days 1, 8, 15 and 22. This treatment regimen was repeated every 28 days in the absence of disease progression or toxicity.

Celsion reported clinical findings from the Phase 1b study at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting in June 2015 demonstrating an overall clinical benefit of 57% for all treatment arms. The overall clinical benefit observed at the highest dose cohort in this difficult-to-treat patient population was 100% (PR=33% and SD=67%) in all six evaluable patients. A maximum tolerated dose was not reached and studies evaluating higher doses of GEN-1 are currently underway.

The new translational data reported today 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.

A publication of this data is being prepared for submission for major scientific review which will detail all cytokine levels observed in this study.

Celsion intends to collect additional translational data, including cellular responses in primary tumor tissue and peritoneal ascites, in its ongoing OVATION Study, a Phase I dose escalation study in newly diagnosed ovarian cancer patients in the neoadjuvant setting.

"With these remarkable findings we feel confident that the data we expect to collect from the OVATION Study will provide further evidence that GEN-1, our gene-mediated immunotherapy, has significant potential in oncology generally and in ovarian cancer specifically," noted Michael H. Tardugno, Celsion’s chairman, CEO and president. "Our clinical development program for ovarian cancer is now well positioned with compelling clinical and biological data, fully supporting our thesis for combining GEN-1 with Doxil and Avastin to treat platinum-resistant ovarian cancer patients in a study we expect to launch later this year."

On January 7, 2016 BIND Therapeutics, Inc. (NASDAQ: BIND), a clinical-stage nanomedicine company developing targeted and programmable therapeutics called ACCURINS, reported the presentation of complete data from its phase 2 clinical trial of BIND-014, a PSMA-targeted ACCURIN containing docetaxel, in patients with chemotherapy-naive metastatic castration-resistant prostate cancer (mCRPC) who either were or were not exposed to anti-androgens (abiraterone acetate and/or enzalutamide) (Press release, BIND Therapeutics, JAN 7, 2016, View Source [SID:1234508681]). BIND-014 was clinically active and well-tolerated and the study met its primary endpoint with 71 percent of patients achieving rPFS of at least 6 months. The complete data are being presented on January 7, 2016 during a poster session at the 2016 Genitourinary Cancers Symposium held in San Francisco.

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"We are encouraged by the safety and activity profile of BIND-014 in this phase 2 trial, which support its potential to be a nanomedicine alternative superior to conventional docetaxel," said Hagop Youssoufian, M.D., M.Sc., chief medical officer, BIND Therapeutics. "Although these data are encouraging, recent advances with anti-androgen therapy limit our ability to compare BIND-014 data to historical benchmarks. At this time, we are not planning further development of BIND-014 in mCRPC given the evolving treatment landscape. We anticipate additional BIND-014 data from both the iNSITE 1 trial in squamous histology non-small cell lung cancer and the iNSITE 2 trial in multiple tumor types during the first quarter of 2016, which we expect to determine our next steps in the clinical development of BIND-014."

In addition to the primary endpoint, data presented from the trial included measurements of safety and tolerability, objective response rates (ORR), prostate-specific antigen (PSA) response, changes in circulating tumor cells (CTC) and overall survival (OS). Key results include:

BIND-014 administered at 60 mg/m² on day 1 of a 21-day cycle was clinically active and well-tolerated when administered to patients (n=42) with chemotherapy-naive mCRPC, including the 74% of patients with prior exposure to abiraterone acetate and/or enzalutamide.

Median rPFS of 9.9 months (95% CI, 7.1 – 12.6) was achieved (n=42 with 8 censored).
A confirmed ORR of 21% was observed in patients with measurable disease (n=19).
A 50% reduction in PSA was observed in 30% of PSA evaluable patients (n=40).
CTC conversion from ≥ 5 cells/7.5 mL blood at baseline to < 5 cells/7.5 mL blood was observed in 50% of patients.
Median OS was 13.4 months (95% CI, 9.9 – 18.6 months [n=42 with 10 censored]).

On January 7, 2016 C4 Therapeutics reported that it has launched from Dana-Farber Cancer Institute with the closing of a $73 million Series A round of financing (Press release, C4 Therapeutics, JAN 7, 2016, View Source;series-a.html [SID:1234508695]).

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The Company is developing novel treatments in the field of targeted protein degradation using proprietary Degronimid technology. The Degronimid platform technology was pioneered since 2010 by researchers in the Bradner Lab at Dana-Farber Cancer Institute and described in a seminal paper published in the Journal Science in June 2015. Degronimids represent a new class of small molecule targeted protein degradation (TPD) therapeutics that target disease-causing proteins and facilitate their rapid destruction and clearance from the cell. The Company has executed a license agreement with Dana-Farber that provides worldwide exclusivity for all applications of the Degronimid technology.

The groundbreaking Degronimid platform utilizes an innovative, all-chemical solution for potent, targeted and rapid protein degradation. Degronimids are novel chemical adapters that are conjugated with selective small molecules designed to recruit the cell’s ubiquitin/proteasome system (UPS) in order to "naturally" degrade targeted proteins. Degronimids offer a promising solution for the removal of previously undruggable proteins, including those that are known to develop resistance to inhibitors.

"We are building a word-class team of pioneers in the field of targeted protein degradation who are dedicated to our mission of developing a new class of therapeutics that will have a profound effect on many diseases," said Marc Cohen, Co-founder and Executive Chairman of C4 Therapeutics. "The use of Degronimids as a strategy for targeting traditionally undruggable proteins and overcoming resistance is extremely promising. By removing the need for ongoing target inhibition from the therapeutic equation, TPD therapeutics represent a new paradigm in drug development. C4 is a company that will develop many drugs over time in proprietary and partnered development programs."

C4 Therapeutics was co-founded by Ken Anderson, M.D., Kraft Family Professor of Medicine, Harvard Medical School, Director of the Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics at Dana-Farber, and a world-renowned expert in protein degradation and multiple myeloma; Nathanael Gray, Ph.D., Professor of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Principal Investigator at Dana-Farber and an expert in kinase inhibitors and drug discovery; software and biotech entrepreneur Marc Cohen; and James "Jay" E. Bradner, M.D., former Associate Professor of Medicine, Harvard Medical School and former Investigator at Dana-Farber. With Dr. Bradner’s new role at Novartis Institutes for BioMedical Research, he will no longer have involvement with the Company. C4 will be located at Mass Innovation Labs in Kendall Square, Cambridge. Cobro Ventures led the Series A round, with additional investments from Cormorant Asset Management, The Kraft Group, EG Capital Group, and angel investors. Other investors include Roche and Novartis.

"Drug discovery research around the ubiquitin/proteasome system is an exciting and growing field, and the discovery of Degronimids represents the first all-chemical solution to ligand-mediated protein degradation," said Dr. Nathanael Gray. "The Dana-Farber Cancer Institute has a strong track record of fostering the advancement of groundbreaking discoveries in numerous oncology applications. By licensing these technologies to companies such as C4, we are supporting the further development of highly promising therapeutic candidates for the betterment of human health."
In conjunction with the Series A financing, C4 announced the appointment of Jason Fisherman, M.D., as Chief Executive Officer of the Company and Member of the Board of Directors. Dr. Fisherman has an extensive track record of building companies as a venture investor at Synthesis Capital and Advent International, a global private equity firm, where his team led or managed over 35 investments in biotechnology, medical technology and healthcare information services. Prior to joining Advent International, Dr. Fisherman had over ten years of drug research and clinical development experience in biopharmaceutical companies, academia, and at the National Cancer Institute. Dr. Fisherman received a B.A. in Molecular Biophysics and Biochemistry from Yale, an M.D. from the University of Pennsylvania and an M.B.A. from the Wharton School of the University of Pennsylvania.

"We are pleased to have Dr. Fisherman join our growing team and believe that C4 will greatly benefit from his extensive financial, business and clinical development expertise," said Cohen. "Dr. Fisherman has 30 years of professional experience within the healthcare and life science industry and will help build our leading Degronimid pharmacology platform."

About C4 Therapeutics
C4 Therapeutics is developing a new class of targeted protein degradation (TPD) therapeutics for the treatment of a broad range of diseases. Our Degronimid platform incorporates highly selective small molecule binders to target disease-causing proteins and facilitate their rapid destruction and clearance from the cell through the natural ubiquitin/proteasome system (UPS). Because of this distinctive mechanism, Degronimids are capable of hitting many more targets, including those previously thought to be undruggable, while reducing the potential for drug resistance. The broad applicability of Degronimids, and our chemical biology platform designed for accelerated validation, have the potential to make an unprecedented impact across many diseases through multiple industry collaborations as well as proprietary programs.