On April 16, 2019 NeoImmuneTech, Inc. (NIT), a T cell-focused therapeutics company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Hyleukin-7TM, a T cell amplifier, in development for the treatment of Idiopathic CD4+ Lymphocytopenia (ICL) (Press release, NeoImmuneTech, APR 16, 2019, View Source [SID1234535151]). Hyleukin-7 also received ODD from the European Medicines Agency in 2017, and it is the first and only agent that has obtained ODD for ICL.

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ICL was first defined in 1992 by the Centers for Disease Control and Prevention, and is a rare disease in which patients present persistently low CD4+ T lymphocyte counts without human immunodeficiency virus (HIV) infection or any other cause of immunodeficiency.

"Patients with ICL frequently suffer from severe and recurrent opportunistic infections and are at high risk for developing certain types of cancer. Currently, no specific treatment for ICL exists. As such, there is high medical need for therapies that can increase CD4+ T cells in ICL patients," said NgocDiep (Diep) Le, M.D., Ph.D., NIT’s Executive Vice President and Chief Medical Officer. "We are delighted that the FDA recognized the potential of Hyleukin-7 as an innovative and transformative treatment for ICL and look forward to conducting a clinical trial in this patient population."

In the phase 1 trial in healthy subjects and multiple ongoing dose-escalation trials in cancer patients, Hyleukin-7 showed a well-tolerated safety profile and dose-dependent increases of CD4+ and CD8+ T lymphocyte counts. NIT has been also actively conducting and planning multiple proof-of-concept clinical trials to develop Hyleukin-7 as an immune-oncology (IO)-enabling drug in combination with other IO therapeutics.

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval and commercialization of a therapeutic agent.

About Hyleukin-7TM
Hyleukin-7TM (rhIL-7-hyFc, NT-I7), an immuno-oncology agent, is a T cell growth factor composed of a covalently linked homodimer of engineered Interleukin-7 (IL-7) molecule, biologically fused with the proprietary long-acting platform – hyFc. IL-7 is known to be a critical factor for T cells homeostasis, acting to increase both the number and functionality of T cells. Hyleukin-7 amplifies and reinvigorates persistent T cell immunity in the treatment of patients with cancer and lymphopenia, thus providing unique opportunities for immuno-oncology (IO) combination strategies. Hyleukin-7 is being developed as an "IO enabling" therapy to harness T cell immunity in combination with current cancer treatments such as anti-PD-(L)1 agents or chemo/radiotherapy as well as next generation IO therapeutics.

On April 16, 2019 KSQ Therapeutics reported the appointment of Beni B. Wolf, MD, PhD, as Chief Medical Officer (Press release, KSQ Therapeutics, APR 16, 2019, View Source [SID1234535150]). Dr. Wolf brings nearly 20 years of experience in the biopharmaceutical industry as a senior clinical leader in oncology drug development, with expertise advancing new oncology programs in the clinic and optimizing patient selection to enable rapid proof of concept and registration. He joins KSQ at a time when the company prepares to advance its first therapeutic program into the clinic in 2020, followed by additional oncology programs that address optimal therapeutic targets based on its CRISPRomics technology.

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Most recently, Dr. Wolf was Senior Vice President, Clinical Development at Blueprint Medicines, where he advanced three oncology programs for novel kinase inhibitors from investigational new drug (IND) applications to clinical proof-of-concept. During his four-year tenure, the clinical progress on Blueprint’s pipeline of oncology programs resulted in three breakthrough designations, plenary presentations for three different clinical programs at major oncology meetings, multiple publications in high-impact peer-reviewed cancer journals, and a range of other data presentations to the oncology and biotechnology communities. Dr. Wolf also played a leadership role in the global registration strategies for these kinase inhibitor programs, including preparation of the regulatory submission for the most advanced kinase inhibitor as a treatment for advanced gastrointestinal stromal tumors.

"Ben has an impressive track record of driving the clinical development of innovative oncology medicines, and his expertise is ideally suited to KSQ as we prepare to advance our oncology product candidates towards clinical testing in cancer patients," said David Meeker, MD, Chief Executive Officer of KSQ. "We are delighted to bring Ben’s exceptional experience across all aspects of drug development to help us realize the potential of KSQ’s emerging pipeline of cancer medicines that we believe can advance cancer treatment based on the insights from our CRISPRomics technology."

Previously, Dr. Wolf served as Senior Medical Director for Merrimack Pharmaceuticals where he advanced preclinical, translational and clinical development of a novel cancer antibody, leading all aspects of drug development, biomarker/companion diagnostic development, clinical planning, publications and patent submissions. Prior to working at Merrimack, he served in clinical and medical director roles at ImmunoGen, Amgen and Genentech. Dr. Wolf started his industry career as a discovery scientist at Genentech. He earned a BS from Union College. Dr. Wolf received his MD and PhD (Biochemistry) from the University of Virginia, and board certification in Internal Medicine and Medical Oncology after completing medical training at the University of California at San Diego. He has authored more than 30 peer-reviewed publications and multiple patents related to drug discoveries.

"I believe that KSQ’s CRISPRomics approach holds tremendous potential for developing high-impact cancer medicines that can set new standards in the field of oncology," said Dr. Wolf. "I share the passion of the KSQ team to rapidly advance new cancer treatments that make a meaningful impact on the lives of cancer patients."

On April 16, 2019 Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported that it will host its annual Science Day for analysts and investors on Tuesday, May 7, 2019 at 8:00 a.m. ET in Cambridge, MA (Press release, Moderna Therapeutics, APR 16, 2019, View Source [SID1234535149]).Moderna also announced that it will report its first quarter 2019 financial results before the market opens on Wednesday, May 8, 2019 and, subsequently, host a conference call to discuss these results and provide a corporate update.

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Science Day:

Moderna’s Science Day will feature presentations from Stephen Hoge M.D., president and Melissa Moore Ph.D., chief scientific officer of Moderna’s mRNA Research Platform with a focus on the Company’s newest advances from its commitment to basic and applied sciences. Moderna will also host a tour of the Norwood manufacturing facility in the afternoon.

First Quarter 2019 Financial Results:

Moderna’s first quarter 2019 financial results and corporate update conference call will begin at 8:00 a.m. ET on May 8, 2019. To access the live conference call, please dial 866-922-5184 (domestic) or 409-937-8950 (international), and refer to conference ID 8273939.

Both events will be webcast live under "Events & Presentations" in the Investors section of the Moderna website at View Source The archived webcasts will be available on Moderna’s website approximately two hours after each event and will be available for 30 days following the events.

On April 16, 2019 Blue Earth Diagnostics, a leading molecular imaging diagnostics company, reported that Axumin (fluciclovine (18F)) has recently been added to the European Association of Urology (EAU) 2019 Clinical Practice Guidelines in Oncology for Prostate Cancer (PCa) (Press release, Blue Earth Diagnostics, APR 16, 2019, View Source [SID1234535148]). The EAU PCa Guidelines assist clinicians in making informed decisions, taking into consideration the scientific data available and individual circumstances of patients. These updated EAU PCa Guidelines state that "18F – Fluciclovine has been approved in the US and Europe, and therefore is currently the only PCa-specific radiotracer widely commercially available."

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Axumin is the first and only novel molecular imaging agent approved in the European Union for use in PET imaging to detect and localize recurrent prostate cancer. Axumin is commercially available in Italy, France, Norway, the Czech Republic, The Netherlands, United Kingdom and Austria with further European countries set to follow soon.

Jonathan Allis, D. Phil., CEO of Blue Earth Diagnostics said "We are delighted that Axumin has been included in the EAU Guidelines as it is an important recognition of the clinical value of our product. Inclusion in these guidelines can help facilitate increased access, in concurrence with our commitment to maximize access to Axumin to patients and clinicians in Europe."

Prostate cancer is a leading cause of cancer death in men in Europe, with around 450,000 new cases diagnosed each year1.

The European Association of Urology (EAU) is the leading authority within Europe on urological practice, research and education, with a mission to raise the level of urological care throughout Europe and beyond.

On April 16, 2019 Genmab A/S (Nasdaq Copenhagen: GEN) reported that worldwide net sales of DARZALEX (daratumumab) as reported by Johnson & Johnson were USD 629 million in the first quarter of 2019 (Press release, Genmab, APR 16, 2019, View Source [SID1234535147]). Net sales were USD 352 million in the U.S. and USD 277 million in the rest of the world. Genmab will receive royalties on the worldwide net sales of DARZALEX under the exclusive worldwide license to Janssen Biotech, Inc. to develop, manufacture and commercialize DARZALEX.

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About DARZALEX(daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.1 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United Stated, Europe and Japan. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).1,2,3,4,5

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.