On May 17, 2018 Provectus and POETIC reported preclinical PV-10 data from pediatric cancer research that will be presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting in Chicago, IL (Press release, Provectus Pharmaceuticals, MAY 17, 2018, View Source [SID1234526789]).

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PV-10 induced cell death in pediatric solid tumor cell lines derived from relapsed pediatric neuroblastoma, Ewing sarcoma, rhabdomyosarcoma, and osteosarcoma with a measurable therapeutic window compared to normal cells. Western blot analyses of cells treated with PV-10 indicated induction of apoptosis. Drug combination studies showed synergy with radiation and agents that target mitosis. Xenograft studies showed significant reduction of tumor burden in PV-10-treated mice compared to control animals, with a corresponding increase in overall survival.

Provectus Biopharmaceuticals, Inc. (OTCQB: PVCT, www.provectusbio.com) ("Provectus" or the "Company") is a clinical-stage biotechnology company developing PV-10 as the first small molecule oncolytic immunotherapy for adult and pediatric solid tumor cancers. The Pediatric Oncology Experimental Therapeutics Investigators’ Consortium ("POETIC") is a group of North American academic medical centers developing new pediatric cancer therapies.

Findings will be highlighted in a poster presentation on Saturday, June 2 from 8:00 to 11:30 a.m. CDT in Hall A as part of the Pediatric Solid Tumors topic during the Pediatric Oncology session. The POETIC poster title is "In vitro and xenograft anti-tumor activity, target modulation and drug synergy studies of PV-10 against refractory pediatric solid tumors," the poster board number is 230, and the abstract number is 10557.

This preclinical pediatric cancer research was led by Aru Narendran, MD, PhD and researchers at the POETIC Laboratory for Pre-Clinical and Drug Discovery Studies at the University of Calgary (Canada), together with Tanya Trippett, MD, Director of POETIC and researchers at Memorial Sloan Kettering Cancer Center.

Dr. Trippett said, "POETIC’s mission is to promote the early clinical development of promising therapies for the treatment of children, adolescents, and young adults with cancer. We hope our collaboration with Provectus on PV-10 leads to an opportunity to improve cancer care for children around the world."

Ed Pershing, Chair of Provectus’ Board of Directors, said, "We are thrilled to work alongside POETIC and its member institutions in Canada and the U.S. in an effort to jointly advance options for pediatric cancer patients around the globe."

About PV-10

Provectus’ lead investigational cancer drug product, PV-10, the first small molecule oncolytic immunotherapy, can induce immunogenic cell death. PV-10 is undergoing clinical study for adult solid tumor cancers, like melanoma and cancers of the liver, and preclinical study for pediatric cancers such as Ewing sarcoma, neuroblastoma, osteosarcoma, and rhabdomyosarcoma.

About the Pediatric Oncology Experimental Therapeutics Investigators’ Consortium

The Pediatric Oncology Experimental Therapeutics Investigators’ Consortium (POETIC) was founded in February 2003 by Dr. Tanya Trippett at Memorial Sloan Kettering Cancer Center and Dr. Lia Gore at the University of Colorado Cancer Center. POETIC is composed of ten large academic medical centers in North America with a major emphasis on comprehensive cancer care and research that provide the collaborative and research strength needed to complete intensive phase I and II studies. Each of the institutions is uniquely suited to complete early studies in the pediatric and adolescent populations. POETIC’s assets include membership in NCI-designated Comprehensive Cancer Centers, on-site NIH-funded pediatric and/or general clinical translational research centers (CTRCs/CTSAs), and active collaborations with developmental therapeutics programs for adults at a majority of its member institutions. The availability of strong basic science and translational research programs at the institutions allows focus on the development and evaluation of new therapeutic strategies for patients with cancer and related disorders. POETIC’s pediatric oncology studies focus on the biologic basis for anti-cancer therapy, and in particular, attempt to explore and evaluate novel agents and/or combinations of therapies early in clinical development as well as new approaches to targeted delivery. For additional information about POETIC, please visit the Consortium’s website at www.poeticphase1.org.

On May 17, 2018 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported that the first patient was enrolled in a Phase 3 clinical trial in China of pamiparib (BGB-290), an investigational PARP inhibitor, in patients with platinum-sensitive recurrent ovarian cancer (Press release, BeiGene, MAY 17, 2018, View Source;p=RssLanding&cat=news&id=2349570 [SID1234526756]).

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"We are pleased to announce the initiation of this Phase 3 trial of pamiparib in China as a potential maintenance therapy in patients with platinum-sensitive recurrent ovarian cancer. This trial is designed to provide important confirmatory clinical data that could enable registration in the maintenance setting, as well support our planned initial regulatory submission for the treatment of patients with advanced ovarian cancer who carry a germline BRCA1/2 mutation," commented Amy Peterson, M.D., Chief Medical Officer for Immuno-Oncology of BeiGene.

"In China there are currently no approved PARP inhibitors, despite the multiple approvals of PARP inhibitors in other regions of the world and in a variety of settings. Our development program in ovarian cancer is designed to address the limited treatment options that currently exist for these patients in China," commented Lai Wang, Ph.D., Senior Vice President and Head of China Development of BeiGene.

The Phase 3 randomized, double-blind, placebo-controlled, multi-center trial is designed to evaluate the efficacy of maintenance therapy with pamiparib versus a placebo in patients with recurrent ovarian cancer who achieved a complete response or partial response after platinum-based chemotherapy, as measured by progression-free survival (PFS) determined by independent review. Secondary objectives include PFS per RECIST version 1.1 determined by investigator, overall survival, objective response rate, duration of response, time to response, safety, and tolerability. Approximately 215 patients are planned to be enrolled in this trial at 15-20 cancer centers in China.

"As we look to improve the current 30 to 40 percent five-year survival rate for patients with advanced ovarian cancer, I look forward to evaluating pamiparib as a potential new maintenance therapy. We are excited to build upon the knowledge base we have of pamiparib from its Phase 1 and 2 studies as well as from other PARP inhibitors in ovarian cancer," said Professor Ding Ma, M.D., Director of Obstetrics and Gynecology, Tongji Medical College of Huazhong University of Science and Technology; and Principal Investigator of the trial.

For more information about the trial, patients and physicians should email [email protected].

About Ovarian Cancer in China

In China, over 50,000 women are diagnosed with ovarian cancer and more than 22,000 die from the disease each year1. More than 70 percent of patients are diagnosed with advanced disease2. The standard therapy for ovarian cancer consists of surgery followed by postoperative platinum-based chemotherapy. An estimated 85 percent of patients with epithelial ovarian cancer who achieve a full remission following first-line therapy will develop recurrent disease3.

About Pamiparib

Pamiparib (BGB-290) is an investigational inhibitor of PARP1 and PARP2 which has demonstrated pharmacological properties such as brain penetration and PARP–DNA complex trapping in preclinical models. Pamiparib is being evaluated in pivotal clinical trials in China. It is currently in global clinical development as a monotherapy, and in combination with other agents, including BeiGene’s investigational anti-PD1 antibody, tislelizumab (BGB-A317), for a variety of solid tumor malignancies.

On May 17, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, and the Stanford University School of Medicine reported a strategic collaboration to support the advancement of Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD) gene therapy research (Press release, Rocket Pharmaceuticals, MAY 17, 2018, View Source;p=RssLanding&cat=news&id=2349565 [SID1234526774]). Rocket’s lentiviral vector (LVV)-based gene therapy program for FA is currently in clinical trials with academic partners in the U.S. and Europe. The LVV-based gene therapy program for PKD is currently in preclinical development in Europe.

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Under the terms of the agreement, Stanford will serve as a lead clinical trial research center in the United States for a planned upcoming registrational trial for FA, and would also be the lead site for PKD clinical trials. Maria Grazia Roncarolo, M.D., director of the Stanford Center for Definitive and Curative Medicine and co-director of the school’s Institute for Stem Cell Biology and Regenerative Medicine, will lead the school’s effort. The center is a joint initiative of the School of Medicine, Stanford Health Care and Stanford Children’s Health and is focused on bench to bedside development of innovative cell- and gene-based therapies.

Gaurav Shah, M.D., Chief Executive Officer and President of Rocket, commented, "Rocket is delighted to expand the reach of our gene therapy program in the U.S. and prepare for our registrational trial. We are committed to developing FA and PKD programs in collaboration with outstanding gene therapy centers and pioneers in the field. This collaboration with the Stanford Center for Definitive and Curative Medicine is a critical step within our overall strategy of building relationships with gene therapy experts, with investigators who have dedicated their careers to improving the care of patients afflicted with these disorders, and within the broader FA and PKD communities."

"This project will also evaluate the introduction of conditioning regimens for both FA and PKD, where we hope to develop best-in-class gene therapy approaches for both clinical indications. The regulatory design and preparation for our registrational trial for FA is ongoing and we remain on track to advance this program to a registration study in 2019. Our PKD program continues to advance in preclinical studies with an Investigational Medicinal Product Dossier (IMPD) expected to be filed in early 2019," continued Dr. Shah.

For more information about this news on the Stanford website, please visit View Source

On May 17, 2018 Ipsen (Euronext: IPN; ADR: IPSEY) reported that the European Commission (EC) has approved Cabometyx (cabozantinib) 20, 40, 60 mg for the first-line treatment of adults with intermediate- or poor- risk advanced renal cell carcinoma (aRCC) (Press release, Ipsen, MAY 17, 2018, View Source [SID1234650566]). This approval allows for the marketing of Cabometyx (cabozantinib) in this indication in all 28 member states of the European Union, Norway and Iceland.

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"Today’s EC approval is a step forward for advanced kidney cancer patients in Europe who will be able to access a new oral first-line treatment option that offers significant improvement over the standard of care", said Harout Semerjian, Executive Vice President, Chief Commercial Officer, Ipsen. "Ipsen remains committed to improving patients’ lives by continuing to develop new therapies and expanding the potential of Cabometyx across different indications."

Giuseppe Procopio, M.D., Head of the Genitourinary Unit at Fondazione Istituto Nazionale Tumori Milan, stated: "The value of treatment with Cabometyx has been corroborated by the data generated in clinical trials, and since 2016 physicians have also witnessed the potential of it when treating patients following VEGF-targeted therapy. For both of these reasons, physicians will be pleased to soon have access to this new first-line treatment option for intermediate- or poor- risk advanced RCC patients."
Today’s decision is based on the CABOSUN trial, which demonstrated that cabozantinib significantly prolongs progression-free survival (PFS) compared to sunitinib in treatment-naive aRCC patients with intermediate- or poor-risk. Cabozantinib is the first and only monotherapy to demonstrate superior clinical efficacy over sunitinib in treatment-naïve aRCC patients with intermediate- or poor-risk.
The detailed recommendations for the use of this product are described in the Summary of Product Characteristics (SmPC), available here (View Source).
About the CABOSUN study
On May 23, 2016, Exelixis announced that CABOSUN met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in PFS compared with sunitinib in patients with intermediate- or poor-risk aRCC per IMDC (International Metastatic RCC Carcinoma Database Consortium) criteria as determined by investigator assessment. CABOSUN was conducted by The Alliance for Clinical Trials in Oncology as part of Exelixis’ collaboration with the NCI-CTEP. These results were first presented by Dr. Toni Choueiri at the meeting of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016, and published in the Journal of Clinical Oncology (Choueiri, JCO, 2018).i
On June 19 2017 Exelixis announced that the analysis of the review by a blinded independent radiology review committee (IRC) has confirmed the primary efficacy endpoint results of investigator-assessed progression-free survival (PFS) from the CABOSUN randomized phase 2 trial of cabozantinib as compared with sunitinib in patients with previously untreated advanced renal cell carcinoma (RCC) with intermediate- or poor-risk disease per the International Metastatic Renal Cell Carcinoma Database Consortium (IMDC) criteria. Per the IRC analysis, cabozantinib demonstrated a clinically meaningful and statistically significant reduction in the rate of disease progression or death as measured by PFS. The incidence of adverse events (any grade) and the incidence of grade 3 or 4 adverse events between cabozantinib and sunitinib were comparable.
CABOSUN is a randomized, open-label, active-controlled phase II trial that enrolled 157 patients with aRCC determined to be intermediate- or poor-risk per IMDC criteria. Patients were randomized 1:1 to receive cabozantinib (60 mg once daily) or sunitinib (50 mg once daily, four weeks on followed by two weeks off). The primary endpoint was PFS. Secondary endpoints included overall survival and objective response rate. Eligible patients were required to have locally advanced or metastatic clear-cell RCC, ECOG performance status 0-2, and had to be intermediate- or poor-risk per IMDC criteria (Heng, JCO, 2009).ii Prior systemic treatment for RCC was not permitted.
About advanced Renal Cell Carcinoma
With the incidence predicted to rise 22% by 2020, renal cell carcinoma (RCC) threatens to become one of the fastest growing cancers in the world.iii Targeted therapies including tyrosine kinase inhibitors (TKIs) of the VEGF receptor (VEGFR) introduced a decade ago, significantly transformed the treatment landscape of aRCC.iv
The American Cancer Society’s 2017 statistics cite kidney cancer as one of the top ten most commonly diagnosed forms of cancer among both men and women in the U.S.v Clear cell RCC is the most common type of kidney cancer in adults.vi If detected in its early stages, the five-year survival rate for RCC is high. For patients with advanced- or late-stage metastatic RCC, however, the five-year survival rate is only 12% with no identified cure for the disease.vii Approximately 30,000 patients in the U.S. and 68,000 globally require treatment.viii
The majority of clear cell RCC tumors have lower than normal levels of a protein called von Hippel-Lindau, which leads to higher levels of MET, AXL, and VEGF.ix–x These proteins promote tumor angiogenesis (blood vessel growth), growth, invasiveness, and metastasis.xi, xii, xiii, xiv MET and AXL may provide escape pathways that drive resistance to VEGFR inhibitors. xii – xv
About CABOMETYX (cabozantinib)
Cabometyx is an oral small molecule inhibitor of receptors, including VEGFR, MET, AXL and RET. In preclinical models, cabozantinib has been shown to inhibit the activity of these receptors, which are involved in normal cellular function and pathologic processes such as tumor angiogenesis, invasiveness, metastasis and drug resistance.
In February of 2016, Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. This agreement was amended in December of 2016 to include commercialization rights for Ipsen in Canada. On April 25, 2016, the FDA approved Cabometyx tablets for the treatment of patients with advanced RCC who have received prior anti-angiogenic therapy and on September 9, 2016, the European Commission approved Cabometyx tablets for the treatment of advanced RCC in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy in the European Union, Norway and Iceland. Cabometyx is available in 20 mg, 40 mg or 60 mg doses. The recommended dose is 60 mg orally, once daily.
On December 19, 2017, Exelixis received approval from the FDA for Cabometyx for the expanded indication of treatment of advanced RCC.
On May 17, 2018, Ipsen announced that the European Commission approved Cabometyx for the first-line treatment of adults with intermediate- or poor- risk advanced renal cell carcinomain the European Union, Norway and Iceland.

On May 17, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a clinical-stage biopharmaceutical company focused on the development of specialized CAR-T cell based therapies, reported the pricing of a global offering of 1,800,000 ordinary shares, comprised of 523,913 ordinary shares in the form of American Depositary Shares (ADSs) offered in the United States, Canada and certain countries outside of Europe at a price per ADS of $26.28, and 1,276,087 ordinary shares in Europe and certain countries outside of the United States and Canada in a concurrent private placement at a price per share of €22.29 (the "global offering") (Press release, Celyad, MAY 17, 2018, View Source [SID1234532516]). Each ADS represents the right to receive one ordinary share. The price per ADS was determined based on an exchange rate of $1.1789 per euro. The gross proceeds to Celyad from the global offering are expected to be approximately $47.3million (approximately €40.1 million), before deducting underwriting commissions and estimated offering expenses.
In addition, Celyad has granted the underwriters a 30-day option to purchase up to an additional 270,000 ordinary shares, which may be in the form of ADSs, on the same terms and conditions. The closing of the global offering is expected to occur on May 22, 2018, and is subject to customary closing conditions.

Celyad’s ADSs are currently listed on the NASDAQ Global Select Market under the symbol "CYAD" and Celyad’s ordinary shares are currently listed on Euronext Brussels and Euronext Paris.

Wells Fargo Securities, LLC and Bryan, Garnier & Co. are acting as joint bookrunning managers for the offering. Bank Degroof Petercam NV is acting as a co-manager for the private placement and LifeSci Capital LLC is acting as a co-manager for the global offering. Kempen & Co NV is Celyad’s advisor in connection with the offering.

The securities are being offered pursuant to an effective shelf registration statement that was previously filed with, and declared effective by, the U.S. Securities and Exchange Commission (SEC). A preliminary prospectus supplement dated May 15, 2018 relating to and describing the terms of the offering was filed with the SEC on May 16, 2018. The final prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities can also be obtained for free from Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 375 Park Avenue, New York, New York, 10152, at (800) 326-5897 or email a request to [email protected] or Bryan, Garnier & Co., Beaufort House, 15 Saint Botolph Street, London EC3A 7BB, United Kingdom, or by telephone at +44 20 7332 2500, or by email at [email protected].

This press release does not constitute an offer to sell nor a solicitation of an offer to buy, nor shall there be any sale of securities in any state or jurisdiction in which such an offer, solicitation or sale is or would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.