BioMarin to Participate in Goldman Sachs 40th Annual Global Healthcare Conference on Tuesday, June 11 in Rancho Palos Verdes, CA

On June 5, 2019 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Henry Fuchs, M.D., President, Worldwide Research & Development, will present at the Goldman Sachs 40th Annual Global Healthcare Conference on Tuesday, June 11 at 8:40am PT/11:40am ET in Rancho Palos Verdes, California (Press release, BioMarin, JUN 5, 2019, View Source [SID1234536911]). To access the live webcast, please visit the investor section of the BioMarin website, www.biomarin.com. A replay will also be archived on the site for at least one week following each event.

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Bavarian Nordic Reports Positive Result from ongoing Phase 2 Trial Evaluating BN-Brachyury in Chordoma

On June 5, 2019 Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) reported that the Data and Safety Monitoring Board (DSMB) confirmed a partial response in one of the first chordoma patients recruited and treated with the combination of BN-Brachyury and radiation treatment at the first evaluation timepoint (Press release, Bavarian Nordic, JUN 5, 2019, View Source [SID1234536930]). As this meets the initial pre-defined threshold of activity for the first stage of the Phase 2 trial, recruitment will be expanded to enroll another 19 patients, while the first 10 patients continue to be treated and evaluated.

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The proof-of-concept Phase 2 trial was designed to determine if the combination of BN-Brachyury and radiation therapy, the current standard of care, results in a clinically meaningful objective response rate (ORR), measured as a percentage of patients with a decrease in tumor size within 12 months of radiation therapy. This is a timeframe during which historical controls show an ORR of less than 5% with radiation alone.

The first stage of the study enrolled 10 patients between November 2018 and January 2019. The study will now advance into stage 2, expanding enrollment to a total of 29 patients with an overall goal of achieving 4 patients with objective responses, corresponding to an ORR of ~14% for all patients enrolled for the study to be considered successful.

"We are excited to share these first Phase 2 data from our current immuno-oncology pipeline, and also the first data suggesting clinical activity of combining our targeted immunotherapy with standard of care," said Paul Chaplin, President and Chief Executive Officer of Bavarian Nordic. "We are encouraged by the rapid progression of this trial, which may offer final results sooner than anticipated and hope this initial finding is confirmed in a larger number of patients and that BN-Brachyury may improve treatment options for patients with chordoma."

For more information on how to take part in this trial, please visit the website of the Chordoma Foundation, who is working to advance new therapies to improve the lives of chordoma patients, and is also actively supporting Bavarian Nordic in the study: View Source

About chordoma
Chordoma is a rare cancer that universally overexpresses brachyury and occurs in the base of the skull and spine. There are approximately 1,000 new cases of chordoma diagnosed in the U.S. and E.U. annually, and 10,000 people living with the disease. Current treatments have resulted in limited success against chordoma, with a historical objective response rate of less than 5% with radiation alone.

About BN-Brachyury
Bavarian Nordic’s novel immuno-oncology candidate, BN-Brachyury, targets a key prognostic indicator of several common (e.g. colorectal, prostate, small cell lung, and triple negative breast cancer) and rare or orphan (e.g. chordoma, thyroid, neuroendocrine) cancers. Brachyury is a transcription factor that is believed to play a prominent role the metastasis and progression of tumors. Expression of brachyury is highly correlated with metastatic disease, poor overall survival, multi-drug resistance, and decreased survival rates. BN-Brachyury utilizes a prime-boost vaccination regimen that has been optimized to include the gene for brachyury and other molecules known to increase immune activation. Patients will receive a primer of MVA-BN Brachyury followed by booster doses of the recombinant fowlpox virus. A previous phase 1 trial demonstrated that MVA-BN-Brachyury could safely target brachyury and induce brachyury-specific T-cell immune responses.

BN-Brachyury has received orphan drug status from the FDA.

NANOBIOTIX APPOINTS NEW CHIEF MEDICAL OFFICER AS THE COMPANY
EVOLVES AFTER ACHIEVEMENT OF EUROPEAN MARKET APPROVAL

On June 4, 2019 NANOBIOTIX (Euronext: NANO – ISIN: FR0011341205), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported it has appointed Edwina Baskin-Bey, M.D. as its new CMO (Press release, Nanobiotix, JUN 4, 2019, View Source [SID1234536862]).

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Dr. Baskin-Bey brings over 18 years of oncology clinical development and basic science experience in academia and industry, most recently serving as CMO of North Carolina-based oncology start-up, Innocrin Pharmaceuticals. As a member of the Executive Team at Innocrin, Dr. Baskin-Bey led development and implementation of the overall clinical and corporate strategy from phase I to phase III. She was responsible for the Medical, Regulatory, Pharmacovigilance, Clinical Operations, Biostatistics, and Data Management functions.

Dr. Baskin-Bey joined Innocrin from Janssen Oncology of Johnson & Johnson, where she was charged with leading both early-and late-stage oncology global development programs for in-licensed products from Aragon and Tesaro, apalutamide and niraparib. Previously, she worked for more than six years at Astellas Pharma. At Astellas, BV, Netherlands Headquarters, Dr. Baskin-Bey, was responsible for several early-stage oncology programs, most notably, she co-led with the alliance partner, Medivation, the global development strategy for the in-licensed product enzalutamide, leading to FDA approval, EMA approval, and label expansions. Also while at Astellas, she served at the UK Headquarters as head of European Medical Affairs, where she was responsible for the launch of enzalutamide in Europe.

Dr. Baskin-Bey currently serves as an independent board member for Catalyst Clinical Research, LLC, in North Carolina. Dr. Baskin-Bey obtained her bachelor’s degree from Hunter College in New York, and earned her medical degree at Mount Sinai/New York University (NYU) School of Medicine. She trained in general surgery and oncology for seven years at The Mayo Clinic in Rochester, MN, and in basic science research principles at the National Institute of Health (NIH).

Dr. Baskin-Bey succeeds accomplished Nanobiotix CMO, Elsa Borghi, M.D. Dr. Borghi will remain with the company in a key leadership role focused on early development and innovation

NantKwest Announces Updated Clinical Results for Nanatinostat (VRx-3996), a Novel HDAC Inhibitor Being Developed in Partnership with Viracta Therapeutics

On June 4, 2019 NantKwest Inc. (Nasdaq:NK) reported that its strategic partner Viracta Therapeutics presented updated clinical data on its HDAC inhibitor, nanatinostat (VRx-3996) at the Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, IL, from May 31st – June 4th, 2019 (Press release, NantKwest, JUN 4, 2019, View Source [SID1234536879]).

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In April 2017, NantKwest announced that it was the lead investor in Viracta’s Series B financing round. Concurrent with the financing, NantKwest secured an exclusive license with commercialization rights to nanatinostat for use in combination with natural killer (NK) cell therapies, including NantKwest’s NK cell platforms.

Nanatinostat is a Class 1 histone deacetylase (HDAC) inhibitor currently in phase Ib/II clinical trials (NCT03397706). In preclinical studies, nanatinostat has been shown to reactivate silenced transgenes in tumor cells thereby turning them into preferential targets for NK cell killing, while also serving to broadly stimulate a patient’s immune system, offering the potential for improved clinical responses in cancer patients.

The activity of HDAC inhibitors are believed to be based on the upregulation of natural killer group 2D (NKG2D) ligand expression on cancer cells, which serve as "eat-me" signals for NK cells and can drive NK proliferation, activation and cancer cell killing.

NantKwest is preparing to initiate clinical trials that include nanatinostat in combination with its haNK and t-haNK cell therapy platforms, which we believe will work synergistically to enhance the efficacy of the company’s NK cell therapies and further distinguish us in the market.

Interim results presented at the 2019 annual ASCO (Free ASCO Whitepaper) meeting from the phase Ib portion of the ongoing phase Ib/II clinical trial of nanatinostat was in combination with the antiviral valganciclovir for the treatment of relapsed/refractory Epstein Barr Virus (EBV)-associated lymphomas. Three doses of the combination were evaluated with responses seen at all dose levels. Both drugs are taken orally and can be administered in an out-patient setting.

EBV-associated cancers are known to be an extremely difficult cancer to treat. Early clinical data with the combination of nanatinostat and anti-viral therapy in EBV-associated lymphoma from the phase Ib/II study has provided encouraging efficacy signals. Currently, there are no approved treatments for EBV-associated lymphomas that specifically target the virus.

The combination therapy produced an objective response rate (ORR) of 58%, a complete response rate (CR) of 33% and a disease stabilization rate (DSR) of 75%. Based on these encouraging results, Viracta anticipates advancement of the clinical trial to the phase II stage that will evaluate intermittent dosing of nanatinostat (4 days on and 3 days off) in combination with daily valganciclovir.

Patrick Soon-Shiong, M.D., Chairman and CEO of NantKwest commented, "EBV-associated lymphomas represent a heterogenous group of cancers that are often aggressive and poorly responsive to available therapy. In this phase Ib stage, we are pleased to see encouraging objective responses including a 33% complete response rate in relapsed/resistant EBV positive cancer patients. We look forward to our continued partnership with Viracta to transition to phase II clinical trials, while also moving nanatinostat forward in combination with NantKwest’s haNK and t-haNK cell therapies."

Ivor Royston, M.D., Viracta’s CEO added, "Our ASCO (Free ASCO Whitepaper) presentation and clinical trial update highlights the potential of our proprietary ‘Kick & Kill’ therapeutic approach to treat a wide range of EBV positive cancer patients, with responses seen across all doses in both T cell and B cell lymphomas in our ongoing phase Ib/II study. "This data enable us to move forward with the phase II portion of our study with a well-tolerated dose combination of nanatinostat with valganciclovir, which we expect to initiate in the third quarter of 2019."

About Nanatinostat

Nanatinostat (VRx-3996) is a histone deacetylase (HDAC) inhibitor that is being investigated in a range of clinical indications. Nanatinostat is selective for Class 1 HDACs, including isoforms targeted in Viracta’s "Kick & Kill" therapeutic approach. Viracta is investigating nanatinostat in a phase Ib/II clinical study [NCT03397706] in combination with an antiviral valganciclovir for the treatment of EBV-associated cancers. Both drugs are taken orally and can be given on an out-patient basis. Recently, the nanatinostat plus valganciclovir combination therapy received Orphan Drug Designation (ODD) from the U.S. Food & Drug Administration (FDA) for three sub-types of EBV-associated cancers: post-transplant lymphoproliferative disorder (PTLD), plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma.

About EBV-Associated Cancers

Approximately 95% of the world’s adult population is infected with Epstein-Barr Virus (EBV). Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of lymphatic cells for the duration of the patients’ life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV lymphomas. In addition, EBV is also associated with a variety of solid tumors, including nasopharyngeal carcinoma and gastric cancer.

TG Therapeutics to Present at the Jefferies 2019 Healthcare Conference

On June 4, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, will present at the Jefferies 2019 Healthcare Conference, being held at the Grand Hyatt Hotel in New York City (Press release, TG Therapeutics, JUN 4, 2019, View Source [SID1234536895]). The presentation is scheduled to take place on Wednesday, June 5, 2019 at 9:00 AM ET.

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A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source