On August 9, 2019 SHINE Medical Technologies LLC reported it has received a $15-million award from the Department of Energy’s National Nuclear Security Administration (DOE/NNSA) (Press release, Shine Medical Technologies, AUG 9, 2019, View Source;pk_kwd=15-million-doe-nnsa-award-for-production-of-medical-isotope [SID1234540974]). The award was made by the agency as part of its effort to establish a reliable, U.S.-produced supply of molybdenum-99 (Mo-99), the most commonly used medical isotope, without the use of highly enriched uranium. The cooperative agreement with the DOE/NNSA requires SHINE to provide $15 million of matching funds.

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Medical isotopes are used in heart stress tests, cancer staging and therapeutics, and other medical applications. Chronic global shortages of Mo-99 routinely and significantly affect the diagnosis and treatment of patients in the United States and around the world.

SHINE broke ground in May on an isotope production facility in Janesville, Wisconsin. It will be the first facility of its kind in the world, using the company’s patented technology to produce Mo-99 and other isotopes used in more than 40 million procedures every year. When the facility becomes operational after the company receives approval to operate from the Nuclear Regulatory Commission (NRC), the plant will be capable of producing the majority of U.S. demand for Mo-99. The NRC’s review is expected to take up to 24 months from its acceptance of SHINE’s application.

"We are grateful for DOE/NNSA’s award as SHINE continues its progress toward production of Mo-99 and other important medical isotopes," said Greg Piefer, SHINE’s founder and CEO. "SHINE’s recently-submitted operating license application to the NRC brings us one step closer to that goal. The recent DOE/NNSA award directly supports SHINE’s continued advancement as we work to supply lifesaving medical tracers and therapeutics to an underserved, growing global market."

About Medical Isotopes
Medical isotopes are radioisotopes that are used in the diagnosis and treatment of disease. Molybdenum-99 (Mo-99) is a radioisotope that decays into the diagnostic imaging agent technetium 99m (Tc-99m). The workhorse of nuclear medicine, Tc-99m is used in more than 40 million medical imaging procedures each year, primarily in stress tests to diagnose heart disease and to stage cases of cancer. SHINE was founded to deploy a safe, cost-effective and environmentally friendly technology to produce a variety of medical isotopes, including Mo-99. Roughly one percent of all Mo-99 in the world decays every hour, meaning it must be produced continuously. Current production is limited to only a handful of government-owned nuclear research reactors, the majority of which are overseas.

On August 9, 2019 NEC Corporation (NEC; TSE: 6701) and CYTLIMIC Inc. reported that financing of 1.3 billion yen (around US$ 12 million at current rate) was completed through the achievement of the milestones, including the first IND filing of CYT001*, pursuant to the agreement with current shareholders; i.e., NEC Corporation, NEC Capital Solutions Limited, Fast Track Initiative, Inc., and SMBC Venture Capital Co., Ltd. In total, 2.3 billion yen (around US$ 21 million) has been raised since the establishment of CYTLIMIC in December 2016 (Press release, NEC, AUG 9, 2019, View Source [SID1234538959]).

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In recent years, Immune-Checkpoint Inhibitors (ICI), such as anti-PD-(L)1 drug, have been expanding their applications; However, a combination of ICI with other drugs are still needed for more than 70% of patients who don’t respond to ICI alone. Among those combination drugs, cancer vaccine is considered as a promising approach to turn "Cold tumors to Hot ones" and to make ICI work. CYT001 is designed to effectively turn "Cold tumors to Hot ones" with the AI-designed optimized shared-antigen peptides, and an optimized combination adjuvant of Poly ICLC (Hiltonol) and LAG-3Ig (Eftilagimod alpha or IMP321). The recent financing allows CYTLIMIC to conduct further clinical trials for the POC establishment of CYT001.

On August 9, 2019 Lion TCR Pte. Ltd., a clinical-stage Biotech company focused on development of innovative engineered T cell immunotherapy against viral-related cancer and chronic Hepatitis B infection, reported on 29 July 2019 that it has signed an agreement with Dalian Lvshun District Government at the 10th Singapore-Liaoning Economic and Trade Council Meeting (SLETC) to develop cell manufacturing and research facilities for cancer treatment (Press release, Lion TCR, AUG 9, 2019, View Source [SID1234538769]). The 5,000-sqm facility, built by Dalian Municipal Government, will support Lion TCR’s development and commercialisation of cell therapy products in China in addition to Lion TCR’s facility at the China-Singapore Guangzhou Knowledge City. Lion TCR shall utilize GMP (Good Manufacturing Practice) certified entities for the production of advanced cellular immunotherapy products for clinical trials and for future commercial products. The facility design and process will comply with both China CDE and internationally recognized GMP guidelines.

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The 10th SLETC was chaired by Mr. Masagos Zulkifli, Singapore’s Minister for the Environment and Water Resources, and Mr. Tang Yijun, Liaoning Governor, the Co-Chairmen of SLETC. At the council meeting, 11 Singapore companies including Lion TCR signed project agreements to provide greater connectivity, sustainability, technology and healthcare in Liaoning. Singapore is Liaoning’s third largest foreign investor with a total investment of about S$19 billion (95 billion yuan). Minister Masagos told the Party Secretary and Mayor of Dalian City at their meeting: "Singapore looks forward to deepening cooperation with Dalian in environmental protection, new energy, interconnection, biomedical, petrochemical, marine fishery, food processing, sustainable development and other fields."

Mr. Stephen Lim, CEO of Lion TCR commented that "Lion TCR looks forward to using this Dalian GMP cell therapy facilities as a manufacturing base for our China commercialisation needs. As a stand-alone building is required for cell therapy GMP application in China, this offer to build with option for Lion to purchase by the Dalian Government is a great help to Lion as it frees our immediate cashflow for continual research and on-going clinical trials."

Mr. Masagos Zulkifli, Singapore’s Minister for the Environment and Water Resources, Singapore Co-Chair of Singapore-Liaoning Economic and Trade Council (fourth from left); Mr. Stephen Lim, CEO of Lion TCR (third from left); Dr. Victor Li, Chairman of Lion TCR (third from right).

On August 9, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Ivan Bergstein, M.D., Stemline’s CEO, will present at the 2019 Wedbush PacGrow Healthcare Conference on Tuesday, August 13, 2019 at 4:15 PM ET at the Parker New York Hotel (Press release, Stemline Therapeutics, AUG 9, 2019, View Source [SID1234538582]). A live webcast of the presentation can be viewed on the company’s website at www.stemline.com.

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About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

On August 9, 2019 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the second quarter ended June 30, 2019 (Press release, Mustang Bio, AUG 9, 2019, View Source [SID1234538581]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The second quarter of 2019 marked the exciting New England Journal of Medicine publication of positive Phase 1/2 data from our partner, St. Jude Children’s Research Hospital (St. Jude), which demonstrated the curative potential of MB-107, a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID). We look forward to transferring the MB-107 IND from St. Jude to Mustang in the fourth quarter of this year. Additionally, we are delighted that the U.S. Food and Drug Administration (FDA) accepted our first Investigational New Drug (IND) application to initiate a multi-center Phase 1/2 clinical trial of MB-102 (CD123 CAR T) in acute myeloid leukemia (AML), blastic plasmacytoid dendritic cell neoplasm (BPDCN) and high-risk myelodysplastic syndrome (MDS)."

Dr. Litchman continued, "Having raised a total of $69 million in the first half of 2019, we look forward to continuing to advance the development of our gene and CAR T cell therapy product candidates in the second half of 2019 and potentially reporting additional CAR T data in the fourth quarter."

Financial Results:

·As of June 30, 2019, Mustang’s cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $83.1 million, compared to $41.1 million as of March 31, 2019 and $34.6 million as of December 31, 2018, an increase of $42.0 million for the quarter and an increase of $48.5 million year-to-date.

·Research and development expenses were $6.8 million for the second quarter of 2019, compared to $3.6 million for the second quarter of 2018. Non-cash, stock-based compensation expenses included in research and development were $0.3 million for second quarter of 2019, compared to $0.6 million for the second quarter of 2018.
·Research and development expenses from license acquisitions totaled $0.2 million for the second quarter of 2019, compared to $0 million for the second quarter of 2018.
·General and administrative expenses were $3.2 million for the second quarter of 2019, compared to $1.7 million for the second quarter of 2018. Non-cash, stock-based compensation expenses included in general and administrative expenses were $1.6 million for the second quarter of 2019, compared to $0.4 million for the second quarter of 2018.
·Net loss attributable to common stockholders was $10.4 million, or $0.29 per share, for the second quarter of 2019, compared to $5.1 million, or $0.19 per share, for the second quarter of 2018.

Recent Corporate Highlights:

·In April 2019, Mustang announced that it had entered into a $20 million debt financing agreement with Horizon Technology Finance Corporation. Fifteen million of the $20 million loan was funded upon closing. The remaining $5 million may be funded upon Mustang achieving certain predetermined milestones. In connection with the debt financing, Mustang issued Horizon warrants to purchase up to 288,184 shares of its common stock at an exercise price of $3.47 per share.

·Also in April 2019, the New England Journal of Medicine published St. Jude data from a Phase 1/2 clinical trial of a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID. Data demonstrated that the lentiviral gene therapy achieved normalization of T-cell numbers in all eight newly diagnosed infants with XSCID to date and disseminated infections resolved completely in all affected infants. Seven of the eight infants treated have developed normal IgM levels to date. Four of those seven infants have discontinued monthly infusions of intravenous immunoglobulin (IVIG) therapy to date. Three of those four infants who discontinued monthly IVIG infusions have responded to vaccines to date.
·In May 2019, Mustang completed an underwritten public offering, including a full over-allotment option exercise, that raised gross proceeds of $31.6 million, excluding underwriting discounts, commissions and other offering-related expenses.
·Also in May 2019, the FDA granted Orphan Drug Designation to MB-108 (oncolytic virus C134) for the treatment of malignant glioma, a type of brain cancer with a median survival of less than 18 months.
·In July 2019, the FDA granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of AML.
·In August 2019, Mustang announced that the FDA had approved its IND application to initiate a multi-center Phase 1/2 clinical trial of MB-102 (CD123 CAR T) in AML, BPDCN and high-risk MDS .