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 Nordic Nanovector ASA (OSE: NANO) reported its results for the first quarter 2018 (Press release, Nordic Nanovector, MAY 30, 2018, View Source [SID1234553502]). A presentation by the company’s senior management team will take place today in Oslo at 08:30 CET, see details below.

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Tone Kvåle, CFO and Interim CEO, commented: "We have been focusing our efforts, since the beginning of the year, on getting PARADIGME approved and started. While our progress with the start-up of this pivotal study has been encouraging, we saw reason in April to announce adjustments to the timelines for its duration and are now targeting the first data read-out from the study in the first half of 2020.

"We remain convinced of the significant potential of Betalutin based on the promising clinical data generated to-date. We also believe that PARADIGME provides a robust trial design to generate the data needed to support our regulatory and commercialisation strategy for Betalutin to become an important new treatment option for 3L FL patients."

Operational Highlights Q1’18

• Start-up activities ongoing for pivotal Phase 2b PARADIGME trial investigating Betalutin as a potential new treatment for patients with third-line relapsed/refractory follicular lymphoma (3L R/R FL)

o As at May 29th, 23 sites in 8 countries are open for enrolment

• Malene Brondberg appointed as Vice President, IR and Corporate Communications

Events after Q1’18

• Luigi Costa stepped down as CEO

o Tone Kvåle appointed as Interim CEO in addition to existing role as CFO

o Search for new CEO underway

• PARADIGME timelines revised with first results targeted for 1H 2020 (previously 2H 2019)

o Start-up activities and site initiations progressing

• Clinical development of Humalutin postponed for the foreseeable future as resources are re-focused on PARADIGME and the Betalutin development programme

Financial Highlights Q1 ‘18

(Figures in brackets = same period 2017 unless otherwise stated)

• Revenues in the first quarter 2018 amounted to NOK 0 (NOK 0.1 million).

• Total operating expenses for the quarter were NOK 82.3 million (NOK 65.8 million).

o Research and development (preclinical, clinical, medical affairs, regulatory and CMC activities) expenses accounted for 76.2 % of total operating expenses in the first quarter 2018 (72.2 %).

• Operating loss for the quarter was NOK 82.3 million (loss of NOK 65.7 million).

• Comprehensive loss for the quarter was NOK 90.7 million (loss of NOK 55.8 million).

• Cash and cash equivalents as at 31 March 2018 amounted to NOK 641.5 million, down from NOK 756.6 million at the end of 31 December 2017.

Outlook

Nordic Nanovector aspires to become a leader in the field of targeted therapies for haematological cancers by developing, manufacturing and commercialising innovative therapies to address major unmet medical needs and advance cancer care.

Betalutin, the company’s most advanced product candidate, has a highly differentiated, competitive, clinical profile for R/R FL, based on the promising results from the LYMRIT 37-01 Phase 1/2a clinical study. The company’s pivotal Phase 2b PARADIGME trial with Betalutin in 3L R/R FL is underway with initial data read-outs from the study targeted for 1H 2020 and subsequent filing in 2020 for marketing approval.

Nordic Nanovector intends to maximize the value of Betalutin across other stages of FL, NHL and other haematological cancer indications.

The company is confident that Betalutin could become an attractive and convenient therapeutic option, which, based on detailed market research, has the potential to be commercially successful.

Current cash resources are expected to be sufficient to reach data read-out from PARADIGME in 1H 2020.

Presentation and webcast – First quarter 2018 results and webcast

A presentation by Nordic Nanovector’s senior management team will take place at 8:30 am CEST on 30 May at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: NYLAND

The presentation will be recorded as a webcast and will be available at www.nordicnanovector.com in the section: Investors & Media

The results report and the presentation will be available at www.nordicnanovector.com in the section: Investors & Media/Reports and Presentation/Interim Reports/2018 from 7:00 am CEST the same day.

Results presentation in Norwegian

As announced in April, a separate presentation of the results in Norwegian, to be hosted by Nordic Nanovector’s CFO and Interim CEO, and its VP IR & Corporate Communications, will take place on Thursday, 31 May 2018 at 8:30 am CEST at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: VIPPETANGEN

To attend the meeting please email – [email protected]

The presentation will NOT be recorded as a webcast

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