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United Therapeutics Corporation Agrees to Sell Priority Review Voucher to AbbVie for $350 Million

On August 19, 2015 United Therapeutics Corporation (NASDAQ: UTHR) reported that it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PPRV) to a subsidiary of AbbVie Inc. (NYSE: ABBV) (Press release, United Therapeutics, AUG 19, 2015, View Source [SID:1234507296]). United Therapeutics received the PPRV when Unituxin was approved by the U.S. Food and Drug Administration (FDA) for the treatment of neuroblastoma, a rare pediatric disease.

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Upon closing of the transaction, United Therapeutics will receive $350 million in cash in exchange for the PPRV. The voucher was awarded by the FDA under a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

"We are very pleased to monetize our PPRV, and hope that this transaction will encourage others to join us in focusing development efforts on rare pediatric diseases," said Roger Jeffs, Ph.D., United Therapeutics’ President and Co-CEO.

The transaction is subject to customary closing conditions and clearance under the Hart-Scott-Rodino Antitrust Improvements Act.

About the Rare Pediatric Disease Priority Review Voucher Program

The program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases. A PPRV may be issued to the sponsor of a rare pediatric disease product application and would entitle the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval. The sponsor receives the PPRV upon approval of the rare pediatric disease product application and it can be sold without limitation.

Pfizer, Provectus Biopharmaceuticals Awarded US Patent Protecting Use of PV-10 as Part of Combination Therapy for Cancer

On August 18, 2015 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.pvct.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus"), reported that it has received a patent from the US Patent and Trademark Office, U.S. Patent number 9,107,887 (Press release, Provectus Pharmaceuticals, AUG 18, 2015, http://www.pvct.com/pressrelease.html?article=20150818.1 [SID:1234507294]). The patent protects the use of PV-10 in combination with certain other types of drugs in the treatment of melanoma and cancers of the liver.

Specifically, the patent covers the use of PV-10 in combination with systemic inhibitors of immune system down-regulation, such as anti-CTLA-4, PD-1 and PD-L1 antibodies, along with enhancers of immune system up-regulation, such as IL-2 and interferon-gamma. Pre-clinical testing of PV-10 used in combination with these important classes of drugs demonstrated potential importance for treatment of advanced cancers.

Provectus believes that the intellectual property covered by the patent will aid it in realizing financial rewards if clinical research demonstrates that PV-10 used in combination with one or more of these classes of drugs improves patient outcomes.

Dr. Eric Wachter, CTO of Provectus, said, "This is a significant milestone in the development of PV-10 as an anti-cancer agent, and builds on our research into PV-10 used on its own. Medical science and our own preliminary studies suggest that it may benefit patients when used in conjunction with these other drugs."

Peter Culpepper, CFO and COO of Provectus, noted, as just one example, a poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) [SITC] 29th Annual Meeting in November 2014. The poster, presented by Dr. Shari Pilon-Thomas of the Moffitt Cancer Center, titled "Efficacy of Intralesional Injection with PV-10 in Combination with Co-Inhibitory Blockade in a Murine Model of Melanoma," showed that combination of PV-10 with anti-CTLA-4, PD-1 and PD-L1 antibodies led to improved tumor response and enhanced anti-tumor immunity of T-cells.

Pfizer Inc. is a joint owner of the patent.

Study Suggests Non-Invasive Stereotactic Body Radiation Therapy Is Feasible For Treating Large Non-Small Cell Lung Cancer Tumors

On August 18, 2015 Varian Medical Systems reported that non-invasive stereotactic body radiation therapy (SBRT), which precisely delivers a high-dose beam of radiation to target tumors while minimizing dose to the surrounding healthy tissue, is effective and well-tolerated by patients with inoperable non-small cell lung cancer (NSCLC) tumors that are larger than 5 cm but had not spread from the lung to the lymph nodes or outside of the chest (i.e. "early stage" or "node negative"), according to a study published in the International Journal of Radiation Oncology Biology Physics (Press release, InfiMed, AUG 18, 2015, View Source [SID:1234507292]).1

The study involved the use of SBRT for the treatment of frail patients with large inoperable lung tumors and without lymph node involvement. SBRT, which makes it possible to complete treatments in fewer sessions than conventional radiation therapy, has not typically been used to treat large tumors. Results from SBRT were compared with literature on outcomes from conventional lung surgery. The research suggests that non-invasive SBRT may be a viable treatment alternative to conventional surgery for some patients with larger lung tumors.

"Our study shows that lung SBRT can be used to safely treat localized node-negative inoperable NSCLC tumors larger than 5 cm, with low rates of recurrence at the primary tumor site and with minimal side effects," said Gregory M. M. Videtic, MD, CM, FRCP, from the Department of Radiation Oncology at the Cleveland Clinic Taussig Cancer Institute, and professor of medicine at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University.

Prior to the emergence of lung SBRT, frail medically compromised patients with node-negative inoperable NSCLC were often treated with external beam radiation therapy which delivers lower doses over a higher number of treatment sessions. However, these patients often experienced a high rate of disease recurrence along with significant side effects. Lung SBRT has become routine for treating small NSCLC tumors, typically less than 3 cm, because of its high rate of local control and limited toxicity. The research by Dr. Videtic and his colleagues is one of the few studies on the use of lung SBRT in larger tumors.

In their retrospective study, Dr. Videtic and his team evaluated the outcomes of 40 patients with node-negative medically inoperable NSCLC whose primary tumors were greater than 5 cm and who were treated with SBRT between December 2003 and June 2014.

The study reviewed patients’ outcomes at 18 months after treatment. Local control, which means there was no evidence of disease at the original tumor site, was achieved in 91.2 percent of the cases. The percentage of patients who experienced distant failure where cancer had spread to other parts of the body was 32.5 percent. When these results were compared to published surgical studies, lung SBRT appeared to have similar rates of local control and similar rates of distant failure.

Disease-free survival in patients who had no lung cancer present at 18 months after treatment was 34.6 percent. The overall survival rate at 18 months, including disease-free patients and those who still had evidence of lung cancer, was 59.7 percent. "The overall survival rates are lower in medically inoperable patients receiving lung SBRT compared to operable surgical patients," said Dr. Videtic. "However, the lower survival rate in medically inoperable patients may be due to the presence of other non-cancer related conditions, such as chronic obstructive pulmonary disease, commonly found in inoperable patients."

The percentage of SBRT patients who were free of side effects was 70.5 percent. Side effects observed from SBRT included mild chest wall pain and modest inflammation in lung tissue. In two severe cases, patients experienced excessive fluid build-up in the lung and a lung collapse due to inflammation that blocked the airways.

The research team concluded: "Lung SBRT for medically inoperable node negative NSCLC tumors larger than 5 cm provides excellent local control with limited toxicity. With appropriate patient selection, SBRT is safe and efficacious for larger tumors."

"While this is a retrospective study that included only a fairly small number of patients who were followed for less than two years, the results look promising and certainly warrant additional investigation," said Dee Khuntia, Varian’s vice president for medical affairs.

Amgen And University Of California, Berkeley Announce Multi-Year Partnership To Assist Cancer Patient Advocacy Organizations In Measuring Effectiveness Of Programs

On August 18, 2015 Amgen (NASDAQ:AMGN) and the Center for Social Sector Leadership at the University of California (UC), Berkeley’s Haas School of Business reported a multi-year partnership to offer a graduate-level course on measuring outcomes of cancer patient advocacy education and support programs (Press release, Amgen, AUG 18, 2015, View Source;p=RssLanding&cat=news&id=2080267 [SID:1234507289]). The MBA course, Social Impact Metrics, is designed to advance the ability of nonprofit organizations to measure the effectiveness of their programs, which is critical in an increasingly challenging donor environment. The overall goal of the initiative is to create a set of measurement best practices that can be adopted across the cancer nonprofit community and beyond.

Amgen has awarded four cancer patient advocacy groups – Bladder Cancer Advocacy Network, Cancer Support Community, Chris4Life Colon Cancer Foundation and Critical Mass – with $35,000 sponsorships to measure the impact of an educational or support initiative. The Berkeley MBA students and faculty will work with the winners to evaluate a specific metrics challenge, recommend a solution, and provide an implementation plan during the semester-long course from August to December 2015.

"Establishing meaningful metrics is critical for nonprofits to improve their impact on the lives of those they serve and demonstrate results to funders," said Colin Boyle, deputy director of University of California, San Francisco Global Health Services and the Haas faculty member who will be teaching this course.

The partnership was formed based on the results of a survey of cancer nonprofit organizations conducted by Amgen in 2013. The study found that creating robust metrics can be a challenge in the social impact field, even as foundations, nonprofits, social enterprises and corporations seek to develop stronger and more significant measures of effectiveness.

"This partnership, like other experiential learning opportunities at UC Berkeley, gives our students a chance to develop and implement solutions in the real world," said Nora Silver, faculty director for the Center for Social Sector Leadership (formerly known as the Center for Nonprofit and Public Leadership). "These types of experiences will prove invaluable to our students as they learn how to forge evidence-based solutions that address current challenges for nonprofits."

"Amgen and other healthcare companies provide substantial support to patient advocacy organizations with the hope that, together, we are making a difference in meeting the needs of patients and families," said Raymond C. Jordan, senior vice president of Corporate Affairs at Amgen. "By measuring the impact of these programs, we can learn how to be more effective in achieving our shared goal of improving the lives of people with cancer. Amgen is excited to support this unique initiative that establishes multi-disciplinary collaboration between the pharmaceutical, academic and patient advocacy communities."

About the Winning Patient Advocacy Projects

Bladder Cancer Advocacy Network’s (BCAN) Patient Webinar Series launched in 2012, to address a variety of education topics and features bladder cancer experts from highly-regarded medical institutions. BCAN is looking to measure the impact this series has on patients, caregivers and family members.

Cancer Support Community (CSC) seeks to better measure the impact of the CSC Cancer Support Helpline, which reaches thousands of patients, family members and health care professionals each year. Launched in April of 2012, licensed mental health professionals provide telephone support interventions that help patients and caregivers identify and address needs and link them to vital information and community resources in order to help them to live better lives, reduce distress and better engage with their health care team.

Chris4Life Colon Cancer Foundation’s clinical trial database, Blue Data, is designed to provide a simple and user-friendly interface that allows patients to accelerate the process of identifying appropriate clinical trials for participation and simplifying the screening process. Chris4Life seeks to measure how patients are engaging with the database in order to improve upon its function and use in order to increase the number of patients who participate in clinical trials.

Critical Mass is looking to measure the impact of its "Mission Control" localized search engine of programs and services specifically curated for adolescents and young adults with cancer. By analyzing the data captured from its current users, Critical Mass seeks to improve the efficient identification of relevant resources to ensure that every adolescent and young adult diagnosed with cancer finds the resources they need, when they need them.

About the Course and Award Criteria

The course will consist of a research project conducted by Berkeley MBA students to assess best practices in nonprofit programming and metrics. The research project is expected to be submitted for publication to a social impact journal. The students’ efforts will not only benefit the sponsorship awardees, but also the patient advocacy community at large that can adopt and learn from the case study solutions upon publication.

The call-for-sponsorships was announced in March 2015, and applications were due by May 29, 2015. To qualify for the award, the nonprofits must have demonstrated a commitment to measuring the impact of their educational or patient support initiatives and have had a specific program or initiative they wished to measure more effectively, along with meeting other criteria for consideration. The selection committee considered a range of criteria, including the variety and mix of programs, relevance to the broader cancer and nonprofit communities and feasibility of executing the metrics solution within the allotted timeframe.

8-K – Current report

On August 18, 2015 The Wistar Institute and BioTime, Inc. (NYSE MKT: BTX) reported that Wistar and BioTime’s subsidiary OncoCyte Corporation, have expanded their collaborative relationship to develop a simple, non-invasive, blood-based test designed to aid physicians in the early detection of lung cancer (Filing, 8-K, BioTime, AUG 18, 2015, View Source [SID:1234507286]). This expanded collaboration follows earlier clinical trials, the interim results of which were presented at the May 2015 American Thoracic Society (ATS) International Conference. Wistar is an international biomedical research leader in cancer, immunology and infectious diseases. OncoCyte is a developer of novel, non-invasive liquid biopsy products for the early detection of cancer.

In October 2013, OncoCyte entered into a Sponsored Research Agreement with The Wistar Institute, a National Cancer Institute-designated cancer center, to develop and test potential lung cancer biomarkers identified by Dr. Louise Showe, Ph.D., professor in Wistar’s Molecular and Cellular Oncogenesis Program. Under the new expanded agreement, OncoCyte and Wistar will continue their collaboration with the goal of developing a highly sensitive and specific diagnostic test for the early detection of lung cancer. Critical to the success of the next phase of the research and development program will be the analysis of an expanded patient sample set, the transition of sample analysis to a platform capable of commercial scale operations, confirmation of mRNA and miRNA expression, and completion of diagnostic test verification activities.

"I look forward to continuing this productive relationship between my lab at Wistar and our collaborators at OncoCyte," said Dr. Showe, professor, Wistar’s Molecular and Cellular Oncogenesis Program; associate director, Center for Systems and Computational Biology; scientific director, Genomics Facility; and scientific director, Bioinformatics Facility. "Lung cancer takes a terrible toll on life and productivity every year. We hope we can impact that toll in some meaningful way, through the ongoing studies."

"We look forward to building on the initial success of our partnership with The Wistar Institute, and are excited about our progress to date," said William Annett, Chief Executive Officer of OncoCyte. "As we continue to develop our liquid biopsy for the early detection of lung cancer, we are enthusiastic about the opportunity to have a major impact on the lives of those that suffer from lung cancer."

Interim Results from Initial Agreement

In May 2015, OncoCyte and The Wistar Institute announced the interim results of a large, clinical study conducted by Dr. Showe and funded by OncoCyte. The clinical interim results from a blood-based diagnostic test for non-invasive detection of lung cancer were presented at the American Thoracic Society (ATS) International Conference. These results from the assayed samples demonstrated a high level of observed sensitivity and specificity of a simple blood-based test designed to aid physicians in the early detection of lung cancer. Performance of the classifier was evaluated using several criteria, including Receiver Operating Characteristic (ROC) area under the curve (AUC) analysis, and yielded an AUC of 0.88 (sensitivity of 76% with a specificity of 88%) in the test set.

Dependent on achieving successful scientific and technical results at this stage of development, OncoCyte and Wistar will proceed to final validation of the test with the goal of completing that work in 2016 to enable OncoCyte to commercially launch the lung diagnostic test.

OncoCyte has exercised options to obtain exclusive licenses to any inventions, discoveries or technology developed in the course of the collaborative research and expects to finalize definitive license agreements with Wistar in the near future.

Lung cancer remains a primary cause of cancer-related death in part because there is no effective diagnostic test to screen patients for lung cancer at an early stage. Annual screening for lung cancer in certain high-risk patients was recently recommended by the United States Preventive Services Task Force (USPSTF), an independent panel of experts in primary care and prevention that systematically reviews the evidence of effectiveness and develops recommendations for clinical preventive services. The Task Force recommended screening using low-dose computed tomography (CT) scans. Although low-dose CT scans have demonstrated high sensitivity in detecting early-stage lung cancer in large clinical studies, it also has a relatively high false-positive rate of approximately 25%. False positives can lead to unnecessary costs and side effects due to the need for highly-invasive diagnostic procedures such as bronchoscopies and lung biopsies.

Large-scale screening of patients at high risk for lung cancer, an estimated seven to ten million patients per year in the U.S., could reduce overall lung cancer mortality through earlier detection. However, the high number of false-positive low-dose CT tests could lead to significant unnecessary costs to the U.S. health care system as a result of associated follow-up testing. Physicians, payers, and patients may therefore welcome a simple to use, low-cost, blood-based test that can help guide patient-management decisions by noninvasively ruling out the presence of cancer.