On May 30, 2018 Kyowa Hakko Kirin Co., Ltd. (Tokyo: 4151 President and COO: Masashi Miyamoto, "Kyowa Hakko Kirin") reported that it has been notified that the U.S. Food and Drug Administration (FDA) is extending its review of the Biologics License Application (BLA) for mogamulizumab for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy (Press release, Kyowa Hakko Kirin, MAY 30, 2018, View Source [SID1234526937]). In a notice received from the FDA, the Prescription Drug User FeeAct (PDUFA) target action date has been extended to September 4, 2018.

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During the review, Kyowa Hakko Kirin submitted additional documentation related to the manufacturing section of the BLA, and FDA subsequently decided that it constituted a major amendment that requiring a 3 month extension to the original target action date, to provide time to complete review of the submission.

The Kyowa Hakko Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.

About Mogamulizumab
Mogamulizumab is an investigational humanized monoclonal antibody (mAb) directed against CC chemokine receptor 4 (CCR4), which is frequently expressed on leukemic cells of certain hematologic malignancies including CTCL (cutaneous T-cell lymphoma). Mogamulizumab was produced using Kyowa Hakko Kirin’s proprietary POTELLIGENT platform, which is associated with enhanced antibody-dependent cellular cytotoxicity (ADCC).

In August 2017, the FDA announced that mogamulizumab has been granted Breakthrough Therapy designation status for the treatment of MF and SS in adult patients who have received at least one prior systemic therapy. In late November 2017, the FDA accepted the BLA for filing and granted mogamulizumab Priority Review.

About Mycosis Fungoides (MF) and Sézary Syndrome (SS)
MF and SS are the two most common subtypes of CTCL, a rare type of non-Hodgkin’s lymphoma, which is characterized by localization of malignant T lymphocytes to the skin, and depending on the stage, the disease may involve skin, blood, lymph nodes, and viscera.

On May 30, 2018 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported that an overview of the company’s business strategy and commercial and development-stage programs will be given at the Jefferies 2018 Global Healthcare Conference being held at the Grand Hyatt in New York (Press release, Spectrum Pharmaceuticals, MAY 30, 2018, http://investor.sppirx.com/news-releases/news-release-details/spectrum-pharmaceuticals-present-corporate-update-jefferies-2018 [SID1234526952]). The company presentation is on Wednesday, June 6, 2018, at 10:30 AM EDT.

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On May 30, 2018 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that Rick Fair, President and CEO, is scheduled to present a corporate overview at the Jefferies 2018 Global Healthcare Conference on Wednesday, June 6 at 2:00 p.m. EDT in New York, NY (Press release, Bellicum Pharmaceuticals, MAY 30, 2018, View Source [SID1234526971]).

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A live webcast of the presentation may be accessed from the News & Events section of the Bellicum website. An archived version of the webcast will be available for replay for at least two weeks following the event.

On May 30, 2018 Oragenics, Inc. (NYSE American: OGEN), a leader in the development of new antibiotics against infectious diseases and effective treatments for oral mucositis (OM), reported positive results from its interim safety analysis as requested by FDA on patients from its Phase 2 clinical trial of AG013 for the treatment of OM (Press release, Oragenics, MAY 30, 2018, View Source [SID1234526953]). The study provides information that, we believe, likely indicates that the overall incidence of severe OM is less than would be anticipated in the general population.

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The Phase 2 trial is a double-blind, placebo-controlled, 2-arm, multi-center trial in which approximately 200 patients will be randomized in a 1:1 ratio to receive either placebo or AG013. Safety was evaluated on the basis of treatment-emergent adverse events, vital signs, weight, physical examinations, clinical laboratory assessments and the presence of AG013 in whole blood. Tolerability measures (taste, consistency and smell) were collected from the patient diaries. In addition, the reasons for study treatment discontinuation were also summarized. To date, 24 patients have been randomized and 19 patients included in the safety evaluation.

Following review of the data by an independent Data Safety Monitoring Board (DSMB), it was concluded that the clinical trial can proceed with no changes to the study. The data analysis indicated that the distribution of adverse events were similar between AG013 and placebo. The serious adverse events reported were consistent with those commonly reported in a head and neck cancer population receiving traditional chemoradiation therapy treatments and included fevers, neutropenia, anemia, nausea and vomiting, infections and oral (mouth and throat) pain. There were no reports of bacteremia or sepsis. Of patients that discontinued participation in the clinical study, 4 patients experienced adverse events, including 3 patients who developed nausea and vomiting, 2 patients that were non-compliant with the study procedures and 3 patients developed severe OM.

The purpose of the Phase 2 study (NCT03234465) is to evaluate the efficacy, safety and tolerability of topically administered AG013 compared to placebo for reducing the incidence and severity of OM in patients undergoing traditional chemoradiation for the treatment of head and neck cancer. Key measures include as measured by the duration, time to development, and overall incidence of OM (World Health Organization scale used) during the active treatment phase, beginning from the start of chemoradiation therapy until 2 weeks following its completion.

"We are pleased with the conclusions reached by the DSMB. Of particular interest was the low number of study discontinuations due to the development of severe OM which resulted in patients seeking alternative treatments for their condition. We are also pleased to note that we believe the overall incidence of severe OM is less than would be anticipated in the general population. While recognizing the preliminary nature of this phase of the study, such a finding might well suggest mitigation of severe OM by AG013," said Alan Joslyn, Oragenics’ President and Chief Executive Officer. "Given the clearance by the DSMB, we will proceed with patient enrollment for our AGO13 clinical trial, which we hope to accelerate by the addition of clinical sites in the U.S. and Europe. We expect to report top-line results of the completed phase 2 trial in late 2019."

"The DSMB’s findings are completely in line with the safety and tolerability signals observed in patients receiving induction chemotherapy for head and neck cancer and reported in the Phase 1b trial and pave the way for completion of a robust Phase 2," said Dr. Stephen Sonis, an expert in the field and advisor to Oragenics. "The unique delivery platform by which AG013 delivers hTTF1 represents an innovative approach with the potential to favorably impact the incidence and course of this devastating side effect of radiation therapy for patients being treated for head and neck cancer."

AG013 is an ActoBiotics therapeutic candidate formulated as a convenient oral rinsing solution and designed by our strategic collaboration partner ActoBio Therapeutics, Inc., a wholly owned subsidiary of Intrexon Corporation (NYSE: XON) to deliver the therapeutic molecule Trefoil Factor 1 to the mucosal tissues in the oral cavity. Trefoil Factors are a class of peptides involved in the protection of gastrointestinal tissues against mucosal damage and play an important role in subsequent repair. AG013 received Fast Track designation from the U.S. Food and Drug Administration in November 2016.

Under an Exclusive Channel Collaboration Agreement with ActoBio Therapeutics, Inc., a wholly owned subsidiary of Intrexon Corporation, Oragenics has an exclusive, worldwide license, to develop and commercialize AG013 to treat OM in cancer patients

On May 30, 2018 Horizon Pharma plc (NASDAQ:HZNP), reported that the company will participate in the Goldman Sachs 39th Annual Global Healthcare Conference on Tuesday, June 12, 2018 and will present at 10:40 a.m. PT (Press release, Horizon Pharma, MAY 30, 2018, View Source [SID1234526939]).

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The presentation will be webcast live and may be accessed by visiting Horizon’s website at View Source A replay of the webcast will be available for the event.