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GAITHERSBURG, Md., May 11, 2017 (GLOBE NEWSWIRE) -- Novavax, Inc. (Nasdaq:NVAX), today announced that Dr. James F. Cummings has been promoted to Vice President, Clinical Development and Translational Medicine. Dr. Cummings joined Novavax as Senior Director of Clinical Development in September 2015, with a specific focus on advancing Novavax’ programs within emerging infectious disease.  Previously, Dr. Cummings served as the Director for Global Emerging Infections Surveillance and Response System (GEIS) at the Armed Forces Health Surveillance Branch (AFHSB), Director, Translational Medicine Branch & Division of Regulated Activities at Walter Reed Army Institute of Research (WRAIR) and Consultant to the U.S. Army Surgeon General for all Medical Research and Development. “In his short tenure at the company, James has shown his ability to constructively organize, motivate and manage complex development programs,” said Stanley C. Erck, President and CEO. “His demonstrated expertise in product development and leadership capabilities earned James the appointment of Project Lead for our nanoparticle influenza vaccine candidate development program, and promotion to Vice President. I am thrilled to congratulate James for his successes here at Novavax.” About Novavax Novavax, Inc. (Nasdaq:NVAX) is a clinical-stage vaccine company committed to delivering novel products to prevent a broad range of infectious diseases. Its recombinant nanoparticles and Matrix-M™ adjuvant technology are the foundation for groundbreaking innovation that improves global health through safe and effective vaccines.


JERSEY CITY, N.J., May 08, 2017 (GLOBE NEWSWIRE) -- SCYNEXIS, Inc. (NASDAQ:SCYX), a biotechnology company delivering innovative anti-infective therapies for difficult-to-treat and often life-threatening infections, today reported financial results for the quarter ended March 31, 2017, and provided an update on recent operational and clinical developments. Clinical Development Program with Oral Formulation of SCY-078 On Track "In the first quarter of 2017, we continued to advance the development program for the oral formulation of SCY-078, our lead development program and the first representative of a novel triterpenoid antifungal family," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. “Although the recent regulatory action is causing some delay in the development of our intravenous formulation, we are progressing our oral development program as planned while we address the clinical hold on the IV formulation. We remain committed to advancing SCY-078 as a treatment for increasingly urgent global health threats. As recently presented at ECCMID, there is a growing body of SCY-078 clinical and nonclinical research highlighting the potential versatility of this compound against multiple invasive, drug-resistant and difficult-to-treat fungal infections, including Candida auris.” First Quarter 2017 Financial Results Cash and cash equivalents and short-term investments totaled $54.9 million as of March 31, 2017, with net working capital of $52.2 million. Research and development, net expenses decreased to $4.0 million in the first quarter of 2017, compared to $4.7 million in the first quarter of 2016. The decrease of $0.7 million, or 15%, for the three months ended March 31, 2017 was primarily driven by a decrease of $0.6 million in clinical development expenses and a decrease of $0.7 million in chemistry, manufacturing and controls (CMC) expenses, offset in part by a $0.3 million increase in preclinical development and a $0.3 million increase in professional services. Selling, general and administrative expenses decreased to $2.1 million in the first quarter of 2017, compared to $2.5 million in the first quarter of 2016. The decrease of $0.5 million, or 19%, was primarily the result of a decrease of $0.6 million in consulting and professional services. Total other income increased to $1.1 million in the first quarter of 2017 due to a $1.5 million non-cash gain recorded on the adjustment in the fair value of the warrant liability, offset in part by a $0.3 million increase in interest expense. Net loss for the first quarter of 2017 was $4.9 million, or $0.19 per share.  This compares to a net loss for the first quarter of 2016 of $7.2 million, or $0.52 per share. About SCYNEXIS, Inc. SCYNEXIS, Inc. is a biotechnology company committed to positively impacting the lives of patients suffering from difficult-to-treat and often life-threatening infections by delivering innovative anti-infective therapies. The SCYNEXIS team has extensive experience in the life sciences industry, discovering and developing more than 30 innovative medicines over a broad range of therapeutic areas. The Company's lead product candidate, SCY-078, is the first representative of a novel intravenous and oral triterpenoid antifungal family and is in Phase 2 clinical development for the treatment of several fungal infections, including serious and life-threatening invasive fungal infections.  For more information, visit www.scynexis.com. Forward Looking Statement Statements contained in this press release maybe, "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited, to: risks inherent in SCYNEXIS' ability to successfully develop SCY-078, including SCYNEXIS' ability to resolve the FDA's concerns to lift the clinical hold on the IV formulation of SCY-078 on a timely basis, if at all, and obtain FDA approval for SCY-078; the expected costs of studies and when they might begin or be concluded; and SCYNEXIS' reliance on third parties to conduct SCYNEXIS' clinical studies. These and other risks are described more fully in SCYNEXIS' filings with the Securities and Exchange Commission, including without limitation, its most recent Annual Report on Form 10-K under the caption "Risk Factors" and other documents subsequently filed with or furnished to the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. SCYNEXIS undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.


Proof of concept study of sequential ONCOS-102 then pembrolizumab treatment in patients with advanced melanoma refractory to check-point inhibitors Oslo 10 May 2017: Targovax ASA ("Targovax" or "the Company"; OSE: TRVX), a clinical stage company focused on developing immuno-oncology therapies to target solid tumors, announces that the first patient has been recruited in the Company's ONCOS-102 study in advanced or unresectable melanoma patients who have had progression following checkpoint inhibitor. The study is conducted at Memorial Sloan Kettering Cancer Center in New York. Anne-Kirsti Aksnes, VP Clinical Development at Targovax said; "This study will allow us to assess in more detail the immune activating properties of ONCOS-102 already observed in earlier studies. The study also addresses a major medical need, as the majority of patients with advanced melanoma do not respond clinically to checkpoint inhibitors. Moreover, we are delighted to do this collaboration with one of the top cancer research centres in the world." ONCOS C824 is an open label, Phase Ib combination study in 12 patients with advanced melanoma, whose disease has progressed despite prior treatment with pembrolizumab (Keytruda®, Merck &Co) or nivolumab (Opdivo®, Bristol Myers Squibb) and who will be sequentially administered ONCOS-102 followed by pembrolizumab. The primary objective of the study is to assess safety and immune activation at lesions and in peripheral blood. The secondary objective is to assess clinical response rate. For further information, please contact: Targovax is a clinical stage company focused on developing and commercializing novel immuno-oncology therapies to target, primarily, treatment-resistant solid tumors. Immuno-oncology is currently one of the fastest growing therapeutic fields in medicine. The Company's development pipeline is based on two novel proprietary platforms: The first platform, ONCOS, uses oncolytic viruses, an emerging class of biological therapy. ONCOS exclusively uses an adenovirus that has been engineered to be an immune activator that selectively target cancer cells. In phase I it has shown to immune activate at lesional level which was associated with clinical benefit. We expect proof of concept data for this platform in 2017 from a clinical trial of lead product ONCOS-102 in patients with refractory malignant melanoma. The second platform, TG peptides (TG), solely targets tumors that express mutated forms of the RAS protein. Mutations to this protein are common in many cancers and are known to drive aggressive disease progression and treatment resistance. There is a high unmet medical need for therapies that are effective against tumors that express these mutations. The TG platform's therapeutic potential stems from its ability to enable a patient's immune system to identify and then destroy tumors bearing any RAS mutations. In early 2017, key proof of concept data for the TG platform from a clinical trial of TG01 in resected pancreatic cancer patients showed encouraging overall survival and will give guidance for the future clinical development of this platform. Targovax's development pipeline has three novel therapeutic candidates in clinical development covering six indications. Both platforms are protected by an extensive portfolio of IP and know-how and have the potential to yield multiple product candidates in a cost-effective manner. Additionally, we have other products in early stages of development. In July 2016, the Company listed its shares on Oslo Axess. In March 2017, the shares were upgraded to Oslo Børs, the main Oslo Stock Exchange. This information is subject to the disclosure requirements pursuant to section 5-12 of the Norwegian Securities Trading Act.


Proof of concept study of sequential ONCOS-102 then pembrolizumab treatment in patients with advanced melanoma refractory to check-point inhibitors Oslo 10 May 2017: Targovax ASA ("Targovax" or "the Company"; OSE: TRVX), a clinical stage company focused on developing immuno-oncology therapies to target solid tumors, announces that the first patient has been recruited in the Company's ONCOS-102 study in advanced or unresectable melanoma patients who have had progression following checkpoint inhibitor. The study is conducted at Memorial Sloan Kettering Cancer Center in New York. Anne-Kirsti Aksnes, VP Clinical Development at Targovax said; "This study will allow us to assess in more detail the immune activating properties of ONCOS-102 already observed in earlier studies. The study also addresses a major medical need, as the majority of patients with advanced melanoma do not respond clinically to checkpoint inhibitors. Moreover, we are delighted to do this collaboration with one of the top cancer research centres in the world." ONCOS C824 is an open label, Phase Ib combination study in 12 patients with advanced melanoma, whose disease has progressed despite prior treatment with pembrolizumab (Keytruda®, Merck &Co) or nivolumab (Opdivo®, Bristol Myers Squibb) and who will be sequentially administered ONCOS-102 followed by pembrolizumab. The primary objective of the study is to assess safety and immune activation at lesions and in peripheral blood. The secondary objective is to assess clinical response rate. For further information, please contact: Targovax is a clinical stage company focused on developing and commercializing novel immuno-oncology therapies to target, primarily, treatment-resistant solid tumors. Immuno-oncology is currently one of the fastest growing therapeutic fields in medicine. The Company's development pipeline is based on two novel proprietary platforms: The first platform, ONCOS, uses oncolytic viruses, an emerging class of biological therapy. ONCOS exclusively uses an adenovirus that has been engineered to be an immune activator that selectively target cancer cells. In phase I it has shown to immune activate at lesional level which was associated with clinical benefit. We expect proof of concept data for this platform in 2017 from a clinical trial of lead product ONCOS-102 in patients with refractory malignant melanoma. The second platform, TG peptides (TG), solely targets tumors that express mutated forms of the RAS protein. Mutations to this protein are common in many cancers and are known to drive aggressive disease progression and treatment resistance. There is a high unmet medical need for therapies that are effective against tumors that express these mutations. The TG platform's therapeutic potential stems from its ability to enable a patient's immune system to identify and then destroy tumors bearing any RAS mutations. In early 2017, key proof of concept data for the TG platform from a clinical trial of TG01 in resected pancreatic cancer patients showed encouraging overall survival and will give guidance for the future clinical development of this platform. Targovax's development pipeline has three novel therapeutic candidates in clinical development covering six indications. Both platforms are protected by an extensive portfolio of IP and know-how and have the potential to yield multiple product candidates in a cost-effective manner. Additionally, we have other products in early stages of development. In July 2016, the Company listed its shares on Oslo Axess. In March 2017, the shares were upgraded to Oslo Børs, the main Oslo Stock Exchange. This information is subject to the disclosure requirements pursuant to section 5-12 of the Norwegian Securities Trading Act.


Discontinues Clinical Development of DTX101, an AAVrh10 Factor IX Gene Therapy Product Candidate for Moderate/Severe-to-Severe Hemophilia B Initial data from Phase 1/2 clinical trial of DTX301, Dimension’s lead AAV8 vector IMD product candidate for OTC Deficiency, expected 2H 2017 CAMBRIDGE, Mass., May 10, 2017 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a key organ for human metabolism, today reported financial results for the first quarter ended March 31, 2017, and provided a corporate update. The company announced its decision to discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of May 2017, and the observation that the data would not meet the company’s minimum target product profile for continued development or future commercialization. Dimension plans to present full study findings, including results from ongoing immune and biomarker analyses, at a future scientific conference. “We are disappointed with the outcome of our DTX101 program, addressing an important disease with significant unmet need; however, our Phase 1/2 open-label clinical study did not demonstrate an ability to achieve a minimum target product profile for continued development or future commercialization,” said Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “We deeply appreciate the participation by the investigators and staff, patients and caregivers who all contributed to the conduct and execution of this Phase 1/2 clinical trial. Further, Dimension remains committed to the hemophilia community through continued investment in the Company’s ongoing IND-enabling activities for DTX201 for hemophilia A, in collaboration with Bayer, and the follow-up of the six patients dosed with DTX101 in the Phase 1/2 clinical trial through an extension study that will monitor all patients for a total of five years.” Dr. Jenkins continued, “We remain excited about the opportunities around our IMD portfolio, which, unlike DTX101, utilizes the AAV8 capsid, and look forward to initial data later this year with DTX301 for OTC deficiency.” The company does not believe the outcome of the DTX101 program will affect the ongoing Phase 1/2 clinical development of DTX301, Dimension’s lead AAV8-based gene therapy, in OTC deficiency, or current vector design for the company’s other investigational AAV therapeutic programs in development. While the company remains focused on the development of our IMD programs testing AAV8 based vectors, it will be undertaking a comprehensive portfolio prioritization review to thoroughly examine resources and the opportunities to focus efforts, which review is expected to be completed by the end of the second quarter of 2017. •  Continued to advance robust portfolio of IMD candidates utilizing the capsid serotype AAV8 - DTX301 for OTC deficiency, DTX401 for GSDIa, DTX501 for PKU, DTX701 for Wilson disease, and DTX601 for citrullinemia type I: •  DTX201: Currently in IND-enabling studies in collaboration with Bayer for the treatment of moderate/severe to severe hemophilia A. Dimension Therapeutics, Inc. (NASDAQ:DMTX) is a leader in discovering and developing new therapeutic products for people living with devastating rare and metabolic diseases associated with the liver, based on the most advanced mammalian adeno-associated virus (AAV) gene delivery technology. Dimension is actively progressing its broad pipeline, which features programs addressing unmet needs for patients suffering from inherited metabolic diseases, including OTC deficiency, GSDIa, citrullinemia type 1, PKU, Wilson disease, and a collaboration with Bayer in hemophilia A. Dimension has initiated a phase 1/2 clinical trial with DTX301 for the treatment of OTC deficiency. The company targets diseases with readily identifiable patient populations, highly predictive preclinical models, and well-described, and often clinically validated, biomarkers. Founded in 2013, Dimension maintains headquarters in Cambridge, Massachusetts. This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the potential productivity of Dimension’s ongoing collaborations, timing and likelihood of achievement of Dimension’s upcoming development milestones, including timing of disclosure of data, the expected progress of Dimension's portfolio and programs, timing and likelihood of regulatory filings and approvals, and our ability to develop and advance product candidates into, and successfully complete, clinical studies. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Dimension’s product candidates, including its candidate, DTX301, will not achieve development milestones, including patient enrollment, dosing of patients, release of initial data, or regulatory filings; and the risks described under the caption "Risk Factors" in Dimension Therapeutics’ Quarterly Report on Form 10-Q for the period ended March 31, 2017, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics’ additional filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Dimension Therapeutics undertakes no duty to update this information unless required by law. This selected financial information should be read in conjunction with the unaudited, condensed consolidated financial statements and notes included in the Company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2017.


EXTON, Pa. and GERMANTOWN, Md., May 09, 2017 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc. (NASDAQ:FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, and Intrexon Corporation (NYSE:XON), a leader in synthetic biology, announced today that two abstracts will be presented in oral sessions at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) from May 10-13, 2017 in Washington, D.C.   The schedule for each presentation is as follows: Session: Pharmacology, Toxicology and Assay Development Presentation: Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) Presenter: Anna Malyala, PhD, Director, New Product Development, Fibrocell Science Abstract ID: 301 Presentation on Thursday, May 11, 2017, 4:45 - 5:00 p.m. ET Room: Maryland ABC Room Session: Somatic Stem Cell Therapies Presentation: Development of an Autologous Gene-Modified Cell Therapy for the Treatment of Linear Scleroderma Presenter: Darby Thomas, PhD, Director, Rare Diseases, Human Therapeutics Division, Intrexon Corporation Abstract ID: 740 Presentation on Saturday, May 13, 2017, 11:00 – 11:15 a.m. ET Location: Delaware AB Room About FCX-007 FCX-007 is Fibrocell's clinical-stage, gene therapy product candidate for the treatment of RDEB, a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7 and is being developed in collaboration with Intrexon Corporation. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution. The FDA has granted Orphan Designation to FCX-007 for the treatment of Dystrophic Epidermolysis Bullosa, which includes RDEB. In addition, FCX-007 has been granted Rare Pediatric Disease Designation and Fast Track Designation by the FDA for treatment of RDEB. Fibrocell is in pre-clinical development of FCX-013, its gene therapy candidate for the treatment of linear scleroderma, a form of localized scleroderma. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System®, a biologic switch activated by an orally administered compound to control future protein expression once the initial fibrosis has been resolved. FCX-013 is designed to be injected under the skin at the location of the fibrosis where the genetically-modified fibroblast cells will produce a protein to break down excess collagen accumulation. The patient takes an oral compound to facilitate protein expression. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further production of the subject protein by FCX-013. Fibrocell has received Orphan Drug Designation from the FDA for FCX-013 for the treatment of localized scleroderma. Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue.  Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase 1/2 trial for the treatment of recessive RDEB. Fibrocell is in pre-clinical development of FCX-013, its product candidate for the treatment of linear scleroderma.   Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology.  For more information, visit http://www.fibrocell.com or follow Fibrocell on Twitter at @Fibrocell. Intrexon Corporation (NYSE:XON) is Powering the Bioindustrial Revolution with Better DNA™ to create biologically-based products that improve the quality of life and the health of the planet.  Intrexon’s integrated technology suite provides its partners across diverse markets with industrial-scale design and development of complex biological systems delivering unprecedented control, quality, function, and performance of living cells.  We call our synthetic biology approach Better DNA®, and we invite you to discover more at www.dna.com  or follow us on Twitter at @Intrexon, on Facebook, and LinkedIn. Fibrocell, the Fibrocell logo and Fibrocell Science are trademarks of Fibrocell Science, Inc. and/or its affiliates.  All other names may be trademarks of their respective owners. Intrexon, Powering the Bioindustrial Revolution with Better DNA, and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners. This press release contains, and our officers and representatives may from time to time make, statements that are “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified as forward-looking statements for this purpose and include, among others, statements relating to: Fibrocell’s expectations regarding the timing of the completion of adult patient enrollment, dosing and reporting of results for the Phase I portion of its Phase I/II clinical trial of FCX-007; the expected initiation of a toxicology/biodistribution study and submission of an IND for FCX-013 in 2017; and the potential advantages of Fibrocell’s product candidates. Forward-looking statements are based upon management’s current expectations and assumptions and are subject to a number of risks, uncertainties and other factors that could cause actual results and events to differ materially and adversely from those indicated herein including, among others: uncertainties and delays relating to the initiation, enrollment and completion of pre-clinical studies and clinical trials; whether pre-clinical study and clinical trial results will validate and support the safety and efficacy of Fibrocell’s product candidates; unanticipated or excess costs relating to the development of Fibrocell’s gene therapy product candidates; Fibrocell’s ability to obtain additional capital to continue to fund operations; Fibrocell’s ability to maintain its collaboration with Intrexon Corporation; and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K filing. As a result, you are cautioned not to place undue reliance on any forward-looking statements. While Fibrocell may update certain forward-looking statements from time to time, Fibrocell specifically disclaims any obligation to do so, whether as a result of new information, future developments or otherwise. Some of the statements made in this press release are forward-looking statements.  These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business.  Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.


CULVER CITY, Calif.--(BUSINESS WIRE)--NantCell and NantKwest Inc. (NASDAQ:NK), two pioneering, next generation, clinical-stage immunotherapy companies focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases, today announced that the U.S. Food & Drug Administration (FDA) has authorized an Investigational New Drug (IND) Application for the NANT Cancer Vaccine for clinical trial enrollment for pancreatic cancer patients (ClinicalTrials.gov NCT03136406). The NANT Cancer Vaccine is the first combination immunotherapy protocol to orchestrate the delivery of metronomic low-dose radiation and chemotherapy with molecularly-informed, tumor-associated antigen vaccines and natural killer cells, to activate the innate and adaptive immune system and to induce immunogenic cell death. By inducing immunogenic cell death and protecting as well as enhancing the innate and adaptive immune system, the NANT Cancer Vaccine seeks to attain long-term sustainable remission of multiple tumor types with lower toxicity and higher efficacy than current standards of care. “Abraxane, a nanoparticle albumin-bound (Nab) paclitaxel, was the first protein-based drug to alter the survival of metastatic pancreatic cancer in over 20 years,” noted Patrick Soon-Shiong, MD, Chairman and CEO of NantKwest. “But we were not content just with the approval of Abraxane as being sufficient to transform this disease. In January 2016, we announced our Cancer Breakthroughs 2020 journey towards developing effective personalized cancer treatments to further harness the human body's innate immune system as a paradigm change to treating patients with cancer. Today's FDA clearance is a further step in our 25-year quest to develop this cancer vaccine that seeks to induce immunogenic cell death and orchestrate the innate and adaptive immune system of the patient through the delivery of molecularly informed, biological platforms. To our knowledge this is the first clinical study whereby protein nanoparticles (Nab) delivering low dose metronomic chemotherapy is combined with molecularly informed (GPS Cancer) tumor associated antigens activating dendritic and T cells by adenoviral and yeast vectors, and orchestrated with both endogenous (IL-15) and exogenous (off the shelf) activation of NK cells. This NANT Cancer Vaccine will be studied in patients suffering from all types of cancers and at all stages of disease in the coming 12 months, a Cancer Breakthroughs 2020 goal," Dr. Soon-Shiong added. “Cancer has historically been one of the most complex challenges that the medical community has tried to combat,” said John Lee, MD, FACS, Senior Vice President of Clinical Development at NantKwest. “Receiving authorization from the FDA for the IND application for the NANT Cancer Vaccine is a testament to our novel immunotherapy approach. Today marks an important milestone for cancer care and reinforces the need for a paradigm shift in the way we approach this deadly disease. NantKwest and NantCell are actively working to initiate the clinical trial across investigational centers and we look forward to offering the NANT Cancer Vaccine regimen to pancreatic cancer patients.” “Cancer cells often go undetected by the immune system; recently, it was found that cancer cells place a brake on the immune system by checkpoint receptors. This discovery has brought forth a new revolution of immuno-oncology with the launch of multiple checkpoint inhibitors. We realize, however, that checkpoint inhibition alone is insufficient to fully activate the immune system to combat the cancerous tumor,” stated Leonard S. Sender, M.D., Senior Vice President of Medical Affairs for Pediatric, Adolescent and Young Adult Oncology at NantKwest. “NANT Cancer Vaccine is a unique, multi-agent protocol aimed at orchestrating all the components of the immune system needed to combat cancer.” This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, statements concerning or implying the timing and conduct of our clinical studies, the anticipated safety and efficacy of our NK cell therapy and the accomplishment and timing of related regulatory determinations and filings. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks and uncertainties include, but are not limited to, the rate of subject enrollment in our clinical studies; the number of subjects that will need to be enrolled in the trial; difficulty obtaining and maintaining regulatory approvals; our limited experience in conducting clinical studies and significant issues regarding our clinical studies, including, but not limited to, the successful opening and the continued participation of clinical sites and their ongoing adherence to protocols, assumptions regarding enrollment rates, timing and availability of subjects meeting inclusion and exclusion criteria, changes to protocols or regulatory requirements, the ability to comply with and meet applicable laws and regulations, unexpected adverse events or safety issues and the sufficiency of funding and adverse events affecting our ability to manufacture and supply cell therapy for our clinical studies. There can be no assurance that data from any of our clinical studies will be sufficient to support an application for marketing in any country or that any such application will ever be approved. These and other risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our Annual Report on Form 10-K for the fiscal year ended December 31, 2016. These forward-looking statements speak only as of the date hereof, and NantKwest, Inc. disclaims any obligation to update these statements except as may be required by law. The NANT Cancer Vaccine is the first combination immunotherapy protocol to orchestrate the delivery of metronomic low dose radiation and chemotherapy with molecularly informed tumor associated antigen vaccines and natural killer cells, to activate the innate and adaptive immune system and to induce immunogenic cell death. By inducing immunogenic cell death and protecting as well as enhancing the innate and adaptive immune system, the NANT Cancer Vaccine seeks to attain long-term sustainable remission of multiple tumor types with lower toxicity and higher efficacy than current standards of care. NantKwest (NASDAQ:NK) is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the body’s first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells. NantKwest’s unique NK cell-based platform, with the capacity to grow active killer cells as a biological cancer therapy, has been designed to induce cell death against cancer or infected cells by three different modes of action: (1) Direct killing using activated NK cells (aNK) that release toxic granules directly into the cell through cell to cell contact, (2) Antibody-mediated killing using haNKs, which are NK cells engineered to incorporate a high affinity receptor that binds to an administered antibody, enhancing the cancer cell killing effect of that antibody, and (3) Chimeric Antigen Receptor (CAR) activated killing using taNKs, which are NK cells engineered to incorporate CARs to target tumor-specific antigens found on the surface of cancer cells. Our aNK, haNK® and taNK TM platform addresses certain limitations of T cell therapies including the reduction of risk of serious “cytokine storms” reported after T cell therapy. As an “off-the-shelf” therapy, NantKwest’s NK cells do not rely on a patient’s own often compromised immune system. In Phase 1 clinical trials in patients with late stage cancer, NantKwest’s NK cells have been successfully administered as an outpatient infusion therapy without any reported severe side effects, even at doses of 10 billion cells. By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs that include a Phase 2 trial for a rare form of melanoma and the planned initiation of a clinical trial of NK cells targeted to breast cancer, we believe NantKwest is uniquely positioned to be the premier immunotherapy company and transform medicine by delivering living drugs in a bag and bringing novel NK cell-based therapies to routine clinical care. For more information please visit http://www.nantkwest.com and follow Dr. Soon-Shiong on Twitter @DrPatSoonShiong. NantCell, a wholly-owned subsidiary of NantWorks, LLC, is an immuno-oncology company focused on the discovery of innovative antibody, T cell and NK cell based treatments by developing molecularly targeted therapeutics, based on the proteomic profile of the patient's tumor, independent of the cancer's anatomical type. NantCell's mission is to make obsolete the standard method of clinical trial design of "trial and error" and replace it with a level of quantitative predictability based on both the genomic and proteomic profile performed a priori. The Company will tap into comprehensive “omic” analytic tools and "big data" generated from supercomputing to develop molecularly designed drugs in this era of genomics and proteomics and identify patients and their tumor signature at the most granular cellular, DNA and protein levels. Patients entering clinical trials would be identified after a comprehensive “omic” analysis from tissue to cell to DNA to RNA to protein to peptide to drug, and tested based on this molecular profile to maximize clinical outcome and minimize side effects. Through these integrated diagnostic methods, the company is pursuing the vision of treating the biology of cancer rather than the anatomy, and driving the immune system inherited by all to defeat cancer. For more information please visit www.nanthealth.com and follow Dr. Soon-Shiong on Twitter @DrPatSoonShiong. NantHealth, Inc., a member of the NantWorks ecosystem of companies, is a next-generation, evidence-based, personalized healthcare company enabling improved patient outcomes and more effective treatment decisions for critical illnesses. NantHealth's unique systems-based approach to personalized healthcare applies novel diagnostics tailored to the specific molecular profiles of patient tissues and integrates this molecular data in a clinical setting with large-scale, real-time biometric signal and phenotypic data to track patient outcomes and deliver precision medicine. For nearly a decade, NantHealth has developed an adaptive learning system, which includes its unique software, middleware and hardware systems infrastructure that collects, indexes, analyzes and interprets billions of molecular, clinical, operational and financial data points derived from novel and traditional sources, continuously improves decision-making and further optimizes our clinical pathways and decision algorithms over time. For more information please visit www.nanthealth.com and follow Dr. Soon-Shiong on Twitter @DrPatSoonShiong.


News Article | September 25, 2017
Site: en.prnasia.com

PROVIDENCE, Rhode Island, Sept. 25, 2017 /PRNewswire/ -- EpiVax, Inc., a leader in the fields of immunogenicity assessment, vaccine design, and immune-engineering today announced the launch of EpiVax Oncology Inc., a company focused on the development of personalized therapeutic cancer vaccines. EpiVax has provided $500,000 in seed funding and has provided exclusive access to enabling technologies to the new precision immunotherapy venture. Gad Berdugo MSc Eng., MBA will lead EpiVax Oncology as Chief Executive Officer. Gad brings over 25 years of biotechnology operational, strategic partnering and financial experience gained at Abbott, Baxter, and Lazard, as well as emerging cancer biotechnology companies. EpiVax Oncology's approach to precision immunotherapy is based on mutated, tumor-specific epitopes, termed Neo-Epitopes, which generate highly potent specific T cell responses against a wide range of tumor types. The company uses genetic profiling (via NGS sequencing) to generate therapeutic vaccines customized for each cancer patient's unique tumor load. EpiVax Oncology vaccines can be used in combination with checkpoint inhibitors or potentially as single agents. EpiVax Oncology's key competitive advantage is centered on a streamlined, validated, commercial-grade Neo-Epitope in silico discovery platform called Ancer™. The Ancer™ platform is powered by EpiVax's EpiMatrix™ and JanusMatrix™ predictive algorithms and exclusively licensed to EpiVax Oncology for the field of cancer. Ancer™ identifies highly immunogenic, Class I and Class II, CD4 and CD8, T cell Neo-Epitopes while selecting out Treg-like epitopes. Candidate Neo-epitopes are qualified and ranked using a proprietary process designed to strengthen vaccine efficacy and decrease the incidence of immune-related adverse events. In head-to-head comparisons, Ancer™ is significantly better at Neo-Epitope identification than public or academic platforms. "Ancer™ is setting EpiVax Oncology apart from emerging companies such as Neon Therapeutics, Gritstone Oncology and BioNtech. EpiVax Oncology will leverage EpiVax's world class excellence in computational immunology, genomics and vaccine design, built over the last 20 years, to create safe, effective, and affordable immunotherapies. We are committed to delivering on the promise of precision medicine," said Gad Berdugo. CLINICAL DEVELOPMENT: EpiVax Oncology is initially preparing for Phase 1b clinical trials in bladder cancer and melanoma using Ancer™-derived Neo-Epitopes formulated in a proprietary clinically-tested delivery vehicle. The first trials are planned to start in 2018. CAPITAL RAISE: EpiVax Oncology is a fully independent company currently conducting a $2M bridge capital raise to prepare for an IND as well as a $10M to $25M Series A financing to fund the first-in-man clinical trials. ABOUT EPIVAX: EpiVax, Inc. is a privately-held biotechnology company focused on the development of vaccines and immunotherapies mainly for infectious diseases and autoimmunity. Led by Annie S. De Groot, M.D., an internationally recognized vaccine design thought leader, and William Martin, chief architect of the EpiMatrix Epitope Discovery Platform, EpiVax has enjoyed significant success in the fields of immunology and bioinformatics.


Despite substantial morbidity associated with respiratory syncytial virus (RSV) infection, there is no licensed vaccine. MEDI-559 is a live attenuated intranasal vaccine candidate being developed for prevention of lower respiratory illness due to RSV in young children. This randomized, placebo-controlled study evaluated safety of MEDI-559 in healthy, RSV-seronegative children. MEDI-559 or placebo was administered on 3 occasions, 2 months apart. Primary safety was based on solicited symptoms (SSs) and adverse events (AEs) collected for 28 days after each dose. Nasal wash samples were collected 3 times after each dose (days 7-10, 12-18, 28-34) and at sick visits. Serum was collected for measuring antibody immune responses to RSV prior to first vaccination and 28 days post final dose. Long-term safety was monitored for 365 days from first dose. SSs were mild and frequent (MEDI-559 84%; placebo 91%); most common SSs were runny/stuffy nose, cough, and irritability/fussiness. AEs occurred in 67% MEDI-559 and 57% placebo recipients: most common AE was upper respiratory tract infection (MEDI-559 35%; placebo 23%). Higher incidence of medically attended lower respiratory illness within 28 days after dosing occurred in the MEDI-559 arm compared to placebo (none associated with vaccine virus shedding). There was no evidence of enhanced RSV disease. Vaccine virus was detected only in MEDI-559 recipients; shedding occurred in 56%subjects, primarily post dose 1. A functional immune response was observed in 59% and 9% MEDI-559 and placebo recipients, respectively, by an RSV microneutralization assay. Vaccine take, assessed by proportion that shed vaccine-type virus or had a seroresponse against RSV, was seen in 95% MEDI-559 subjects. MEDI-559 is therefore biologically active and immunogenic in this seronegative pediatric population. Although the frequency of SSs and AEs was not considered clinically significant, the increase in medically attended lower respiratory illnesses in the vaccine group warrants expanded safety studies. ClinicalTrials.gov NCT00767416.


Leon F.,Clinical Development
Journal of Immunological Methods | Year: 2011

This article reviews the multiple uses of flow cytometry in the diagnosis, monitoring and research of celiac disease, the most prevalent chronic autoimmune gastrointestinal disease. The phenotyping of intraepithelial lymphocytes (IELs) is of clinical relevance in the diagnosis of the disease given the characteristic features of elevated CD3+ IELs (αβ and γδ TcR) and the decrease in CD3- IELs. IEL biomarkers are also useful in the assessment of the response to the gluten-free diet and, importantly, in the diagnosis of the severe complications of celiac disease: refractory celiac disease and enteropathy-associated T-cell lymphoma. Novel applications of flow cytometry for the detection of anti-transglutaminase antibodies (a validated biomarker of celiac disease) and of gluten (the triggering antigen of the autoimmune process) are also discussed. The assessment of diagnostic and prognostic biomarkers by flow cytometry in celiac disease is performed routinely in a growing number of centers and it is an example of the versatility of this technique and its applicability to the research and clinical study of solid tissues. © 2010 Elsevier B.V.

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