On November 4, 2016 Celsion Corporation (NASDAQ:CLSN), a clinical stage oncology drug development company, reported that the United States Patent and Trademark Office (USPTO) has granted two patents: Patent No. 9,468,687 B2 – Immuno Gene Therapy for Treatment of Cancer and Hyperproliferative Diseases, which expands the use of GEN-1 into additional cancer treatment modalities in combination with other chemotherapeutics and Patent No. 9,144,546 – Nucleic Acid-Lipopolymer Compositions, which expands and extends previous patent claims on the making of and composition of formulations consisting of our PPC delivery polymer and nucleic acids (Press release, Celsion, NOV 4, 2016, View Source [SID1234516252]).

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These new patents further strengthen coverage of GEN-1, the Company’s DNA-based immunotherapy in development for the localized treatment of ovarian cancer and glioblastoma multiforme (GBM), which is already covered by a composition of matter patent in the United States.

"Issuance of these patents further strengthens Celsion’s growing position as a leader in the development of gene-based immunotherapies addressing some of the most difficult-to-treat cancers, such as ovarian cancer and GBM by covering the use of GEN-1 for treating solid tumors as a monotherapy and in combination with chemotherapy," said Michael H. Tardugno, chairman, president and CEO. "GEN-1 leverages our proprietary TheraPlas technology platform, harnessing the power of IL-12 immunotherapy with a targeted delivery system engineered to overcome the limitations associated with the development of other dosage forms of IL-12 therapies."

About GEN-1
GEN-1 is being evaluated in an ongoing Phase 1b dose-escalating clinical trial (the "OVATION Study") combining GEN-1 with the standard of care for the treatment of newly diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debunking surgery. The OVATION Study is designed to enroll three to six patients per dose cohort at escalating doses of GEN-1 with the goal to identify a safe, tolerable and therapeutically active dose of GEN-1 by recruiting and maximizing an antitumor immune response.

As previously reported, all six patients in the first two cohorts of the OVATION Study experienced a clinically meaningful response, ranging from stable disease to one pathologically confirmed complete response. In addition, all patients sustained decreases of 90% or greater of the prospective indicator of the presence of ovarian cancer cells, CA-125 protein as well as highly impressive pathologically responses, which is associated with prolonged survival. The first three cohorts each enrolled three patients. Enrollment in the fourth and final cohort is underway, and Celsion expects to report full data from the OVATION Study by the first quarter of 2017. Future studies of GEN-1 will include a Phase I/II study combining GEN-1 with Avastin and Doxil for the treatment of recurrent ovarian cancer.

On November 4, 2016 AVEO Oncology (NASDAQ:AVEO) reported financial results for the third quarter ended September 30, 2016 (Press release, AVEO, NOV 4, 2016, View Source;p=RssLanding&cat=news&id=2219710 [SID1234516250]).

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"In the last 18 months, we have made important progress in moving forward both elements of our strategy, including our oncology pipeline, for which we have retained significant North American rights, and our non-oncology pipeline, which is being advanced through partnerships with disease-area experts," said Michael Bailey, president and chief executive officer. "We anticipate a milestone rich calendar in the months ahead, spanning both our proprietary and partnered programs, that we expect will help define AVEO’s long-term potential in multiple areas of unmet medical need."

Mr. Bailey added: "For our lead oncology asset, tivozanib, we are well underway in three simultaneous paths. We expect an approval decision in first-line renal cell cancer (RCC) in Europe and initial results from the Opdivo combination TiNivo study in the first half of 2017, followed by, in the first quarter of 2018, pivotal top-line data from our U.S.-registration-directed Phase 3 TIVO-3 study in RCC. During 2017, we also expect to see progress in our partnered pipeline, serving to highlight the value-potential of these programs which are being developed at little or no cost to AVEO."

Potential Corporate Milestones through the First Half of 2017

Regulatory decision for tivozanib in the European Union and associated milestone payment by EUSA Pharma;
IND and proof-of-concept milestone payments for tivozanib in acute macular degeneration by Ophthotech;
Partnership for AV-353, a first-in-class opportunity to address a major unmet need in pulmonary arterial hypertension;
Initial safety results from the Phase 1 portion of the Phase 1/2 AVEO-sponsored TiNivo study of tivozanib in combination with Bristol-Myers Squibb’s Opdivo (nivolumab);
Development progress and milestone payments for AV-380 in Cachexia by Novartis;
Manufacturing tech transfer milestone payment for AV-203 by CANbridge.
Third Quarter and Recent Highlights

Initiation of Phase 1/2 TiNivo Trial Evaluating Tivozanib in Combination with Bristol-Myers Squibb’s Opdivo (nivolumab) in Advanced RCC. In August 2016, AVEO announced the initiation of a Phase 1/2 clinical trial of AVEO’s oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI), tivozanib, in combination with Bristol-Myers Squibb’s anti-PD-1 therapy, Opdivo (nivolumab), in advanced RCC, named the TiNivo Trial. The trial is being led by the Institut Gustave Roussy in Paris under the direction of Professor Bernard Escudier, MD, Chairman of the Genitourinary Oncology Committee. The Phase 1 trial is designed to evaluate whether tivozanib’s unique specificity and associated safety profile can overcome the tolerability issues that have challenged TKI-PD1 combinations to date. Initial safety results from the Phase 1 portion of the Phase 1/2 are expected to be available to the companies in the first half of 2017.
Ongoing Review of the Marketing Authorization Application (MAA) in Europe for Approval of Tivozanib as a First-Line RCC Treatment Option. EUSA Pharma, to which the Company licensed European and additional rights outside North America, is working to submit responses to the European Medicines Agency (EMA) Day 120 List of Questions before year-end. The MAA, which is based on tivozanib’s existing dataset, including the Phase 3 TIVO-1 study of tivozanib in first-line RCC, seeks approval for tivozanib as a first-line treatment for advanced RCC under the EMA’s centralized review process. Following the response, EUSA Pharma expects to receive the EMA Day 180 List of Outstanding Issues, the last review stopping period prior to a recommendation from the Committee for Medicinal Products for Human Use (CHMP) and the final approval decision from EMA. If approved, tivozanib’s distinct safety profile has the potential to offer a new, well tolerated alternative to currently approved VEGF TKIs, which, in clinical studies, have been associated with challenging tolerability necessitating significant dose reductions and interruptions.
Continued Execution of the Pivotal Phase 3 TIVO-3 Study of Tivozanib in RCC. In May 2016, AVEO announced the commencement of enrollment and patient treatment for the Company’s pivotal TIVO-3 trial, a randomized, controlled, multi-center, open-label study to compare tivozanib to sorafenib in subjects with refractory advanced RCC. The study continues to be on track to reach a top line readout in the first quarter of 2018. The Phase 3 trial is expected to enroll approximately 322 patients with recurrent or metastatic RCC who have failed at least two prior regimens, including VEGF-TKI therapy (other than sorafenib). Eligible patients may also have received checkpoint inhibitor therapy in earlier lines of treatment. Patients will be randomized 1:1 to receive either tivozanib or sorafenib, with no crossover between arms.

The TIVO-3 trial, together with the TIVO-1 trial, is designed to support a first- and third-line indication for tivozanib in the U.S. TIVO-3 would also provide a unique data set, in that it is expected to include the first randomized Phase 3 results showing treatment with a VEGF TKI following prior PD1 therapy, and is designed to support approval of the first VEGF TKI specifically labeled for third-line treatment.
Third Quarter 2016 Financial Highlights

AVEO ended Q3 2016 with $30.8 million in cash, cash equivalents and marketable securities as compared with $34.1 million at December 31, 2015.
Total collaboration revenue in Q3 2016 was approximately $1.0 million compared with $15.2 million Q3 2015.
Research and development expense was $4.4 million in Q3 2016 compared with $4.5 million for Q3 2015.
General and administrative expense was $2.1 million in Q3 2016 compared with $2.2 million for Q3 2015.
Net loss for Q3 2016 was $5.0 million, or a loss of $0.07 per basic and diluted share, compared with net income of $7.9 million, or income of $0.14 per basic and diluted share for Q3 2015.
Financial Guidance

We believe that our $30.8 million in cash resources could allow us to fund our planned operations into the fourth quarter of 2017. Furthermore, we expect that these cash resources, together with certain anticipated operational milestone payments from our collaboration partners, could allow us to fund our U.S. tivozanib development strategy through at least pivotal Phase 3 TIVO-3 top-line data as well as our tivozanib-PD-1 inhibitor combination trial.

On November 4, 2016 AbbVie (NYSE: ABBV), a global biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to veliparib, an oral poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) inhibitor, being investigated in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC) (Press release, AbbVie, NOV 4, 2016, View Source [SID1234516249]).

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PARP is a naturally-occurring enzyme in the body that repairs damage to DNA in cells. While this repair is a useful process to maintain the integrity of healthy cells, the same process may also help repair DNA in cancer cells, causing them to survive.3 Researchers are investigating whether veliparib, a PARP inhibitor, in combination with DNA damaging therapies, such as chemotherapy or radiation, may lessen the repair of DNA damage in cancer cells, eventually causing some cells to die.3

NSCLC is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.2 Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.2 Squamous cell carcinoma accounts for about 25 to 30 percent of NSCLC cases.2 It is usually found in the middle airways of the lungs and is often linked to a history of smoking.2

"Lung cancer is the leading cause of cancer-related deaths in the U.S. and can be difficult to treat, particularly when diagnosed in later stages. This Orphan Drug Designation for veliparib recognizes the significant unmet need in patients with advanced squamous non-small cell lung cancer," said Michael Severino, M.D., executive vice president of research and development and chief scientific officer, AbbVie. "AbbVie is committed to the ongoing development of veliparib in solid tumors to help advance the care of people living with cancer."

AbbVie is currently investigating the efficacy and safety of veliparib in combination with chemotherapy or radiation for the treatment of advanced squamous NSCLC, including in Phase 3 studies. Veliparib is not currently approved to treat any form of NSCLC.1

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.4

About Non-Small Cell Lung Cancer
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer in the U.S., accounting for approximately 80 to 85 percent of diagnosed cases.2 There are three main subtypes of NSCLC: adenocarcinoma, squamous cell (epidermoid) carcinoma and large cell (undifferentiated) carcinoma.2 Currently, about 25 to 30 percent of NSCLC cases worldwide are squamous tumors.2 Survival in people with lung cancer can vary depending on the stage, or extent, of the cancer when it is diagnosed.2

About Veliparib
Veliparib is an investigational oral poly (adenosine diphosphate [ADP]–ribose) polymerase (PARP) inhibitor being evaluated in multiple tumor types.5,6 PARP is a naturally-occurring enzyme in the body that repairs damage to DNA in cells. While this repair is a useful process to maintain the integrity of healthy cells, the same process may also help repair DNA in cancer cells, causing them to survive.3

Discovered and developed by AbbVie researchers, veliparib is being studied in combination with chemotherapy or radiation to help determine whether it can prevent DNA repair in cancer cells to possibly increase the effectiveness of common DNA-damaging therapies, such as chemotherapy or radiation.5 Veliparib is currently being studied in more than a dozen cancers, including in Phase 3 studies in advanced squamous and non-squamous non-small cell lung cancer (NSCLC), ovarian cancer and breast cancer.7 Veliparib is an investigational medicine and its efficacy and safety have not been established by the U.S. Food and Drug Administration (FDA) or any other health authority.

On November 4, 2016 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that its in-house developed anticancer agent Halaven (eribulin mesylate, "eribulin") has been recommended by the U.K. National Institute for Health and Clinical Excellence (NICE) as a treatment for patients with locally advanced or metastatic breast cancer who have received at least two chemotherapeutic regimens for advanced disease (prior therapy may have included an anthracycline or a taxane, and capecitabine) in NICE’s Final Appraisal Determination (FAD) (Press release, Eisai, NOV 4, 2016, View Source [SID1234516247]). 1 Eribulin is the first breast cancer treatment to be recommended by NICE since 2007.
2 Following the issue of the FAD by NICE, eribulin will be eligible for reimbursement for this indication via the National Health Service in England (NHS England).

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The Appraisal Committee considered that the models suggest Eribulin offers a mean overall survival benefit of more than 3 months. According to the FAD, "In light of the short life expectancy at this stage of breast cancer, the committee considered this overall survival benefit to be substantial. The committee concluded that eribulin met the end-of-life criteria objectively and robustly and that it can be considered a life-extending, end-of-life treatment." Eribulin was approved in Europe in March 2011 as a treatment for patients with locally advanced or metastatic breast cancer who have received at least two chemotherapeutic regimens for advanced disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting. The agent was launched in the U.K. in April 2011. Furthermore, eribulin was approved in June 2014 for an expanded indication to include patients with locally advanced or metastatic breast cancer who have progressed after at least one chemotherapeutic regimen for advanced disease (prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting, unless patients were not suitable for these treatments).

Eisai regards oncology as a key therapeutic area and is aiming to discover revolutionary new medicines with the potential to cure cancer. Eisai remains committed to providing further clinical evidence and expanding patient access for eribulin, and by maximizing the value of the drug, seeks to contribute further to addressing the diverse needs of, and increasing the benefits provided to, patients with cancer, their families, and healthcare providers.

About Halaven (eribulin mesylate, "eribulin") Eribulin is the first in the halichondrin class of microtubule dynamics inhibitors with a novel mechanism of action. Structurally eribulin is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai. Eribulin is believed to work by inhibiting the growth phase of microtubule dynamics which prevents cell division. In addition, recent non-clinical studies showed that eribulin is associated with increased vascular perfusion and permeability in tumor cores.3 Eribulin promotes the epithelial state and decreases the capacity of breast cancer cells to migrate.4 Eribulin was first approved in November 2010 in the United States as a treatment for patients with metastatic breast cancer who have received at least two chemotherapeutic regimens for the treatment of metastatic disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting. Eribulin is currently approved for use in the treatment of breast cancer in approximately 60 countries worldwide, including Japan and countries in Europe, the Americas and Asia. In Japan, eribulin has been approved to treat inoperable or recurrent breast cancer and was launched in the country in July 2011. In addition, eribulin has been approved in countries in Europe and Asia indicated as a treatment for patients with locally advanced or metastatic breast cancer who have progressed after at least one chemotherapeutic regimen for advanced disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting, unless patients were not suitable for these treatments.
Regarding soft tissue sarcoma, eribulin was approved in the United States for the treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracycline-containing regimen in January 2016, approved in Japan for the treatment of soft tissue sarcoma in February 2016, and approved in Europe for the treatment of adult patients with unresectable liposarcomas who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease in May 2016. Applications seeking approval for use in the treatment of soft tissue sarcoma are currently under review in countries including Switzerland, Australia, Brazil and Malaysia.

2. About NICE’s New Approach to the Appraisal and Funding of Cancer Drugs The former Cancer Drugs Fund (CDF) was established in 2009 as a means to improve patients’ access to new cancer drugs including those which were "Not Recommended" by NICE. These drugs would be evaluated and listed in the CDF with their costs reimbursed through the fund. However, due to a severe increase in financial burden, a new scheme for NICE appraisal and funding of cancer drugs, including a new CDF, came into operation on July 29, 2016. All novel cancer drugs that had been newly approved would undergo NICE appraisal while cancer drugs that were listed in the former CDF would be eligible for reappraisal by NICE under the new scheme depending on the judgment of each company. The NICE appraisal process consists initially of an Appraisal Consultation Document, the issue of a Final Appraisal Determination, and ultimately the setting of Final Guidance. While cancer drugs that are "Recommended" are eligible for reimbursement through the NHS England, drugs that are "Not Recommended" require an Individual Funding Request (IFR) to be deliberated on a case-by-case basis, and therefore their use is greatly limited. Cancer drugs that could possibly be recommended but are judged to lack sufficient evidence can be given a "Limited Recommendation under the CDF", with provisional access secured under the CDF for a maximum of two years. After receiving this designation, NICE conducts a reappraisal based on the new evidence, and ultimately determines whether to either "Recommend" or "Not Recommend" the drug.

On November 4, 2016 Cancer Research UK and Cancer Research Technology (CRT), the charity’s commercial arm, reported an agreement with TYG oncology Ltd (TYG) to take its new antigen-specific, active checkpoint control cancer vaccine TYG100, into clinical trials in cancer patients with advanced solid tumours (Press release, Cancer Research Technology, APR 4, 2016, View Source [SID1234523176]).

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TYG100 is among a range of new treatments that use a patient’s own immune system to target hormones that encourage the growth and spread of solid tumours. It works by triggering the production of antibodies that specifically target two members of the gastrin hormone family* that help to fuel tumour growth.

Under the agreement, Cancer Research UK and TYG will share the cost of the process development and production of TYG100 for the clinic.

Cancer Research UK’s Centre for Drug Development (CDD) will then sponsor and manage a Phase I clinical trial of TYG100 in cancer patients with advanced solid tumours, mainly to evaluate drug safety and toxicity. It will take place across the Experimental Cancer Medicine Centres (ECMC) network, a nationwide initiative funded by Cancer Research UK and the UK’s four Health Departments.

The collaboration forms part of Cancer Research UK’s Clinical Development Partnership (CPD) scheme, meaning that at the end of the trial, TYG oncology has the option of either retaining rights to the new treatment or transferring them to Cancer Research UK to continue development through a new partner.

Fred Jacobs, president of TYG oncology, said: "We are very pleased to be working in partnership with the clinical experience and expertise from Cancer Research UK to advance TYG100. Cancer Research UK’s sponsorship of the Phase I clinical trial is a significant technical and financial validation of our novel therapeutic vaccine. TYG100 will be attacking some of the most difficult and deadliest solid tumours, including pancreatic and gastro-oesophageal cancers."

Dr Nigel Blackburn, Cancer Research UK’s director of drug development, said: "We hope that next generation cancer vaccines like this one will build on the widespread success of existing hormonal treatments – such as those targeting oestrogen in breast cancer and testosterone in prostate cancer. It’s a very promising area of research and we’re excited to be working with TYG oncology to take this to the next stage."

"Our Clinical Development Partnerships are a novel approach that allows companies to benefit from Cancer Research UK’s extensive drug development resources in a way that is mutually beneficial and that ultimately helps new treatments reach patients more quickly."