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

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 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 Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology Company focused on the treatment of primary and metastatic liver cancers, reported that five new clinical trial sites in the U.S. have been activated in the Company’s global FOCUS Phase 3 clinical trial for patients with hepatic dominant ocular melanoma (the FOCUS Trial) (Press release, Delcath Systems, OCT 11, 2016, View Source;p=RssLanding&cat=news&id=2210776 [SID:SID1234515730]). These accredited centers join several other prestigious centers as active participants in the FOCUS Trial. Currently, Delcath now has a total of 8 cancer centers in the U.S. open for patient enrollment in the FOCUS Trial.

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The new participating centers are:

Ohio State University Comprehensive Cancer Center
MD Anderson Cancer Center
Thomas Jefferson University Sidney Kimmel Cancer Center
University of Chicago Comprehensive Cancer Center
University of Maryland Greenebaum Cancer Center
"We are pleased to add these respected cancer research institutions to our FOCUS Trial, and to expand the regional coverage available to patients in the U.S. with ocular melanoma liver metastases," said Jennifer K. Simpson, Ph.D., MSN, CRNP, President and CEO of Delcath. "Interest in the FOCUS Trial has been strong, and we expect to achieve our goal of activating approximately 30 centers in both the U.S. and Europe in the coming months."

About the FOCUS Trial
The FOCUS Trial is a global Phase 3 clinical study evaluating the safety, efficacy and pharmacokinetic profile of the Company’s Melphalan/HDS system versus best alternative care in 240 patients with ocular melanoma liver metastases. The FOCUS Trial’s primary endpoint is a comparison of overall survival between the two study arms; secondary and exploratory endpoints include progression-free survival, overall response rate and quality-of-life measures. The FOCUS Trial is being conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). The SPA provides agreement that the Phase 3 trial design adequately addresses objectives that, if met, would support the submission for regulatory approval of Melphalan/HDS.