On October 12, 2016 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported strategic leadership appointments to support the advancement of its portfolio of DNA-based cancer immunotherapies and infectious disease vaccines (Press release, Inovio, OCT 12, 2016, View Source;to-Advance-its-DNA-Immunotherapy-Product-Portfolio/default.aspx [SID:SID1234515766]). Inovio has an extensive pipeline of clinical-stage cancer immunotherapies highlighted by VGX-3100, which is entering phase III this year; and DNA vaccines in development for Zika, MERS and HIV, among others.

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Inovio adds two oncologists and a plasmid manufacturing expert reporting to Inovio’s Chief Medical Officer, Dr. Mark Bagarazzi:

Dr. Ildiko Csiki will serve Inovio as Vice President, Clinical Development, Oncology, responsible for advancing Inovio’s cancer programs. Prior to joining Inovio, Dr. Csiki had been clinical lead and senior director of clinical research at Merck & Co. where she guided Merck’s global solid tumor development program and was lead for several registration studies. Dr. Csiki also served as GSK’s director of clinical development and lead physician for the follicular lymphoma program. Dr. Csiki earned her MD and PhD degrees from Vanderbilt University School of Medicine and her BS in Biology and BA in Psychology from the University of Arkansas.

Dr. Jeffrey Skolnik, Vice President, Clinical Development, Oncology, will also direct Inovio’s cancer immunotherapy programs. Dr. Skolnik was previously vice president of clinical research at TetraLogic Pharmaceuticals, where he oversaw all global clinical assets. He also served as a medical director at GSK and AstraZeneca. Dr. Skolnik earned his MD at New York University, with honors in pathology, and his undergraduate degree from the University of Pennsylvania.

Robert J. Juba Jr. has been promoted to Vice President, Biological Manufacturing and Clinical Supply Management, and is responsible for ensuring the provision of Inovio’s SynCon plasmid DNA therapeutic and prophylactic vaccine candidates for clinical use. He has 22 years of experience in the pharmaceutical industry managing cGMP processes and operations, with extensive technical expertise in bacterial vaccine manufacturing. He led plasmid manufacturing at VGXI, Inc. and held several positions at Merck in bulk vaccine manufacturing operations and strategy. He holds Masters and BS degrees in Chemical Engineering from the Massachusetts Institute of Technology.
Inovio has also bolstered its manufacturing and business development functions with two additional staff reporting to Dr. Niranjan Sardesai, Inovio’s Chief Operating Officer:

Daniel Jordan will serve as Inovio’s Vice President, Device Manufacturing Operations. Previously he was vice president of U.S. and Canadian operations at Verisk Analytics, a 3M company. He has over 25 years of medical device manufacturing operations experience in early growth to mature organizations. Most recently he served as executive director of global operations at Teleflex Medical, a diversified medical device manufacturer. He earned an MBA in Finance from Weber University and a BS in Business Management at San Diego State University.

Dr. Paul Stead, Inovio’s Vice President, Business Development, will lead business development and partnering activities. He was vice president, business development at Nimbus Therapeutics, a company developing novel treatments for metabolic and immunological diseases, and oncology. For more than 20 years he served GSK in roles of increasing responsibility including business development, competitive intelligence and discovery chemistry. He holds an MBA from Lehigh University, a PhD in pharmaceutical sciences from the University of Nottingham, and a BS in Pharmacy from the University of Bath.
Dr. J. Joseph Kim, Inovio’s President & CEO, said, "We welcome these talented and experienced technical and business leaders to Inovio as we work to bring new breakthrough cancer medicines and vaccines to patients. It is gratifying that Inovio’s corporate culture, validated platform, and important product pipeline are attracting such accomplished executives to help us execute our product development and commercialization strategy."

On October 12, 2016 Transgenomic, Inc. (NASDAQ: TBIO), and privately-held Precipio Diagnostics, LLC reported entry into a merger agreement, pursuant to which Precipio will become a wholly owned subsidiary of Transgenomic, and Transgenomic will be renamed Precipio, Inc (Press release, Transgenomic, OCT 12, 2016, View Source [SID:SID1234515801]). In connection with the merger, it is anticipated that the original Precipio security holders will receive between 62% and 80% of the outstanding shares of the combined company, depending on the relative amount of outstanding liabilities of the parties at closing and prior to the investment of new capital. The merger is expected to close in 2016, pending approval by Transgenomic shareholders and other closing conditions set forth in the merger agreement. Simultaneous to the merger, the combined company will receive an investment of up to $7 million from a syndicate led by BV Advisory Partners in a private placement of preferred convertible securities, and $3.0 million of outstanding debt of each company is expected to convert into this same class of preferred convertible securities. This comprehensive transaction will provide the Company with a clean balance sheet and sufficient capital to achieve its planned expansion.

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Transgenomic has filed to complete a reverse stock split of between one-for-ten and one-for-thirty before the merger closes, and the company’s outstanding debt is expected to convert into common and preferred shares. The companies expect that shares of the combined company will be listed on the NASDAQ exchange and trade under the "PRPO" ticker (subject to filing and approval by NASDAQ). The merger agreement provides that, Ilan Danieli, Precipio founder and Chief Executive Officer, will serve as the Chief Executive Officer of the combined company. BV Advisory Partners is acting as advisor to the transaction.

Paul Kinnon, Transgenomic President and Chief Executive Officer, said "In recent years we have transitioned from a provider of conventional life science tools and diagnostic services into an innovative biotechnology enterprise focused on advancing precision medicine. We have done this through our revolutionary ICE COLD-PCR (ICP) technology, which enables accurate, non-invasive tumor profiling using circulating DNA in patient plasma. We have established a solid platform for commercialization of ICP, with leading global distributors and a solid pipeline of potential agreements with partners and customers. This is a good time to join forces with Precipio, which shares our commitment to accurate and timely advanced cancer diagnostics and has established an impressive infrastructure of academic experts and a growing customer base, validated by successful case studies. I look forward to working with my new colleagues to ensure a successful transition."

Ilan Danieli, Precipio founder and Chief Executive Officer, said "We are proud of Precipio’s progress in building a growing platform that provides unique services to cancer patients and their physicians by providing a demonstrated superior level of diagnostic accuracy, ensuring that patients receive the best possible treatment. Cancer misdiagnosis is an all too common and underappreciated problem, which frequently has a negative impact on patient treatment, and may cause needless loss of life. We provide both primary and second opinion screening, and our network of leading academic cancer researchers and advanced diagnostic technologies have proven to be an invaluable resource for patients and physicians. Our entire team is committed to ensuring that our services are made widely available. To that end we will continue building out our sales team to accelerate adoption and revenue growth. We believe Transgenomic’s ICP technology and commercial infrastructure fit well with our values and our business model, and look forward to this next stage of growth, as we work together to integrate our teams, technologies and services."

Keith Barksdale, Founder of BV Advisory Partners, commented, "Transgenomic and Precipio have complementary strengths with the potential to be a dynamic and strong competitor in the rapidly growing market for advanced cancer diagnostics. ICP is a revolutionary mutation detection technology that is now available through global distributors, and adoption by drug researchers and developers is ramping up. The technology is also available to help guide cancer diagnosis and treatment through Transgenomic’s CLIA laboratory. Precipio’s platform of leading academic cancer experts provides superior diagnostic accuracy level to oncologists and their patients; it represents a unique resource that can benefit from and leverage the power of ICE COLD-PCR. We look forward to working with the combined company going forward to help assure its growth and success."

Transgenomic’s ICE COLD-PCR offers major advantages over current sequencing technologies. It delivers at least a 100-fold improvement in sensitivity compared to standard methodologies, allowing detection of both known and previously unknown genetic alterations in any exon of any gene using a single assay. It is robust, easy to use and easily implemented, requiring minimal disruption to established sequencing workflows. It is available as ICEme Kits that deliver up to a 500-fold increase in mutation detection compared to most current methods, with levels of detection routinely achievable down to 0.01%. This ultra-high sensitivity enables detection of low level mutations and allows accurate patient monitoring as well as stratification of cancer sub-populations. ICEme Kits are compatible with most patient samples, including tissue, blood, plasma, urine and other biofluids. The kits are simple to use and work with most of the genomic analysis platforms available in laboratories today. They are easily customizable for use with single mutations or multiple mutations in combination. The current menu includes approximately 20 clinically relevant, actionable mutations that are associated with important cancers. The ICP range of mutation targets is being expanded on an ongoing basis.

ICE COLD-PCR was originally developed by the laboratory of Dr. Mike Makrigiorgos at the Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology exclusively to Transgenomic.

On October 11, 2016 Celsion Corporation (NASDAQ:CLSN), a leading oncology drug development company, reported a collaboration with the Children’s Research Institute to conduct a clinical study of ThermoDox, Celsion’s heat activated liposomal encapsulation of doxorubicin, in combination with magnetic resonance-guided high intensity focused ultrasound (MR-HIFU) to treat relapsed or refractory solid tumors in children and young adults (Press release, Celsion, OCT 11, 2016, View Source [SID:SID1234515733]). This investigator-sponsored Phase I clinical study is being partially funded by the National Institutes of Health and is expected to commence in the fourth quarter of 2016.

"Even with the use of intensive therapy, the prognosis for children diagnosed with metastatic sarcoma and recurrent solid tumors remains poor and has not improved over the past three decades," stated Dr. Nicolas Borys, Celsion’s chief medical officer. "Recent advances in the use of non-invasive MR-HIFU coupled with novel therapies such as ThermoDox have demonstrated the clear potential to overcome the challenges to treating pediatric malignancies by enabling safer, more tolerable targeted therapies with the potential to change cancer treatment paradigms."

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The trial targeting treatment of childhood sarcomas will be carried out as a multi-disciplinary collaboration among Celsion, the research groups of Dr. AeRang Kim, MD, PhD at the Children’s National Medical Center – Department of Hematology/Oncology, and Dr. Brad Wood and Dr. Rosandra Kaplan at the National Institutes of Health.

"Celsion’s experience in combining ThermoDox with HIFU, a non-invasive next generation heating technology, supports this very important research in childhood cancers. From a safe dose, ThermoDox’s proven ability to deliver high concentrations of an effective chemotherapy directly to a heated tumor makes it an ideal candidate for a trial involving children and young adults," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "This study will further define ThermoDox’s potential in combination with ultrasound-induced hyperthermia, and highlight potential applications of ThermoDox in combination with a broad range of heating technologies that could address an even larger population of patients."

ThermoDox is currently in late stage clinical trials in primary liver cancer and recurrent chest wall breast cancer. It is positioned for use with multiple heating technologies, and has the potential for applications in the treatment of other forms of cancer including metastatic liver and non-muscle invading bladder cancers.

About ThermoDox
Celsion’s most advanced program is a heat-mediated, tumor-targeting drug delivery technology that employs a novel heat-sensitive liposome engineered to address a range of difficult-to-treat cancers. The first application of this platform is ThermoDox, a lyso-thermosensitive liposomal doxorubicin (LTLD), whose novel mechanism of action delivers high concentrations of doxorubicin to a region targeted with the application of localized heat at 40°C, just above body temperature. ThermoDox has the potential to address a broad range of cancers.

Celsion’s LTLD technology leverages two mechanisms of tumor biology to deliver higher concentrations of drug directly to the tumor site. In the first mechanism, rapidly growing tumors have leaky vasculature, which is permeable to liposomes and enables their accumulation within tumors. Leaky vasculature influences a number of factors within the tumor, including the access of therapeutic agents to tumor cells. Administered intravenously, ThermoDox is engineered with a half-life to allow significant accumulation of liposomes at the tumor site as these liposomes recirculate in the blood stream. In the second mechanism, when an external heating device heats tumor tissue to a temperature of 40°C or greater, the heat-sensitive liposome rapidly changes structure and the liposomal membrane selectively dissolves, creating openings that can release a chemotherapeutic agent directly into the tumor and into the surrounding vasculature. Drug concentration increases as a function of the accumulation of liposomes at the tumor site, but only where the heat is present. This method damages only the tumor and the area related to tumor invasion, supporting more precise drug targeting.

On October 11,2016 Kite Pharma, Inc. (Nasdaq:KITE) reported that it will hold an Investor Day on October 18, 2016 in New York where the company will share updates on its advancing pipeline of chimeric antigen receptor (CAR) and T cell receptor (TCR) product candidates, next generation research and development programs, and KTE-C19 launch readiness. The event and live webcast will begin at 12:00pm Eastern Time (Press release, Kite Pharma, OCT 11, 2016, View Source [SID:SID1234515737]). The live audio webcast can be accessed through the Events and Presentations section under the Investors tab of Kite’s website at www.kitepharma.com. Following the live audio webcast, a replay will be available on Kite’s website for approximately 30 days.

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About KTE-C19

Kite Pharma’s lead product candidate, KTE-C19, is an investigational therapy in which a patient’s T cells are engineered to express a CAR to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. KTE-C19 has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

On October 11, 2016 Nymox Pharmaceutical Corporation (NASDAQ:NYMX) reported successful new study results from the long-term repeated injection group from the U.S. Phase 3 trials for fexapotide, the Company’s lead compound in late stage development for enlarged prostate (BPH) and for localized prostate cancer (Press release, Nymox, OCT 11, 2016, View Source;fvtc=4&fvtv=6907 [SID:SID1234515739]). The aim of the study was to determine the safety and clinical benefit fexapotide can provide to men who were given a second injection of fexapotide for their prostate enlargement (BPH). In the new study long-term outcomes were determined in 344 patients who were given a single repeat fexapotide treatment after initial blinded treatment with fexapotide or placebo. Patients were followed for 2 to 6.5 years (mean 4.2 years) after initial treatment. All treatment failures were included in the analysis. Results have now shown that there was long-term statistically significant symptomatic improvement (mean improvement of 6.5 points in the AUA BPH Symptom Score) compared to Phase 3 patients who received placebo alone (p<.001). Repeat injection was found to be safe with no significant drug related toxicities or side effects found in the study.

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"These prospective long-term study results in reinjected patients clearly demonstrate that fexapotide leads to clinically meaningful long-term symptomatic improvements in BPH patients with minimal treatment, and without the worrisome and bothersome toxicities of conventional BPH treatments such as retrograde ejaculation, and increased cancer risk," said Dr. Paul Averback MD, CEO of Nymox. "Our earlier reported Phase 3 studies have shown that fexapotide reduces the long-term need for surgery by up to 82-95% compared to approved conventional BPH treatments. Data indicates that fexapotide shows significant efficacy against prostate cancer as a therapeutic, and in addition has been shown in Phase 3 to reduce the risk of prostate cancer when fexapotide is used to treat BPH. This is in comparison to some conventional BPH treatments in routine clinical use today which on the other hand increase prostate cancer risk, and which have these many other well known undesirable side effects," he said.

Nymox’s lead drug fexapotide has been in development for over a decade and has been tested by expert clinical trial investigative teams in over 70 distinguished clinical trial centers throughout the US, and has been found after 7 years of prospective placebo controlled double blind studies of treatment of 995 U.S. men with prostate enlargement to not only show clinically meaningful and durable relief of BPH symptoms, but also to show a major reduction in the incidence of prostate cancer, compared to placebo and compared to the known and expected normal incidence of the disease. The same clinical program conducted at the same highly regarded treatment centers under rigorous trial scrutiny and performed strictly at arms-length by top teams of clinical investigators across the country, has also shown in a long-term blinded placebo crossover group study an 82-95% reduction in the number of these patients who required surgery after they received crossover fexapotide in the trial, as compared to patients who did not receive fexapotide but instead received crossover conventional approved BPH treatments (p<.0001). The aim of the crossover study was to determine the clinical benefit fexapotide can provide to men who initially were double blind randomized to and received placebo, remained blinded as to their placebo treatment, and who subsequently required additional medical and/or surgical treatment. In that study long-term outcomes were determined in 391 patients who were given double blind placebo injections, followed by crossover to other treatments at the patients’ discretion. The numbers of blinded placebo patients who subsequently received surgical treatment during the next 2-3 years for their BPH symptoms were then prospectively analyzed.

For the earlier fexapotide Phase 3 long-term cancer incidence analysis, the men in the study received fexapotide or placebo for the treatment of their prostate enlargement (BPH) symptoms. All men were thoroughly evaluated at expert urological testing investigational centers to exclude any prostate cancer prior to qualifying for enrollment in the studies. The participants were followed for up to 7 years (median of 5 years) after treatment. The study analyzed all cases of prostate cancer that were subsequently diagnosed. The expected rate of new prostate cancer in the U.S. general male population in this age group is in the 5-20% range after 7 years. In the BPH population in published large trials of drugs for the prevention of prostate cancer, the incidence of new prostate cancer cases after 4-7 years has been reported in major studies to be 20-25%. The data analysis from the Nymox fexapotide study showed a statistically significant and very low incidence of 1.3% for prostate cancer in this comparable fexapotide treated BPH population. By comparison, for example in a population of patients with erectile dysfunction treated with PDE5 inhibitor drugs after 4 years the rate of subsequent prostate cancer was 19.5% (and 22.7% in controls) as recently reported in a large U.S. study published in the Journal of Urology (Volume 196; 3, 2016). The quoted study was in a population of middle aged and elderly men without prostate cancer, similar to the Nymox study population.

Nymox has completed and fully financed the execution of seven Phase 3 U.S. BPH clinical protocols, including 2 prospective randomized multicenter single injection double blind clinical trials; 2 U.S. repeat injection clinical trials; and 3 U.S. blinded long-term clinical trial extension studies. In addition, a number of Phase 3 safety and clinical pharmacology studies and analyses have been completed. The Company expects to file for approvals in the next 1-2 quarters. The Company also expects to report further analyses and results when available in the near future. The Company will publish the findings of the fexapotide clinical trials in peer review medical journals as well as in presentations at medical and urological meetings