Biostatistics

Biostatistics

SEARCH FILTERS
Time filter
Source Type

News Article | April 24, 2017
Site: www.eurekalert.org

Older patients enrolled in team-based primary care practices in Quebec had similar rates of hospital readmission, and lower rates of emergency department visits and death after hospital discharge, compared with those in traditional fee-for-service practices, found a study published in CMAJ (Canadian Medical Association Journal). "Our study showed that the newer team-based primary care delivery model in Quebec was associated with some better post-discharge outcomes among older or chronically ill patients, notably lower rates of emergency department visits and death," writes Dr. Bruno Riverin, Department of Epidemiology, Biostatistics & Occupational Health, McGill University, Montréal, Quebec, with coauthors. Hospital readmissions cost the Canadian health care system $1.8 billion each year (excluding physician costs) and many older or chronically ill patients are at increased risk of complications in the weeks after discharge. The large study looked at data on 312 377 older or chronically ill patients in Quebec who were admitted to hospital between November 2002 and January 2009 (620 656 admissions). The researchers found that about 1 in 4 older or chronically ill patients who had been in hospital for any cause returned within 30 days (for either readmission or an emergency department visit). "Patients enrolled in team-based primary care practices had a 5% lower 30-day risk of emergency department visits not associated with readmission, and significantly fewer patients died in the early period after hospital discharge compared with patients enrolled in traditional primary care practices," write the authors. They hypothesize that health care professionals in these team-based practices are better able to coordinate care for the sickest patients, which helps reduce hospital readmission and death. The study was conducted by researchers at McGill University; Montreal Children's Hospital; McGill University Health Centre, Direction de la santé publique du CIUSS du Centre-Sud-de-l'Île-de-Montréal, Montréal, Quebec; and University of Pittsburgh, Pittsburgh, Pennsylvania.


News Article | April 17, 2017
Site: www.eurekalert.org

Chapel Hill, NC - HIV cure research to date has focused on clearing the virus from T cells, a type of white blood cell that is an essential part of the immune system. Yet investigators in the Division of Infectious Diseases at the University of North Carolina School of Medicine have found the virus persists in HIV-infected macrophages. Macrophages are large white blood cells found in tissues throughout the body including the liver, lung, bone marrow and brain. The discovery of this additional viral reservoir has significant implications for HIV cure research. These findings were published in Nature Medicine on Monday, April 17. "These results are paradigm changing because they demonstrate that cells other than T cells can serve as a reservoir for HIV," said Jenna Honeycutt, Ph.D., lead-author and postdoctoral research associate in the UNC Division of Infectious Diseases. "The fact that HIV-infected macrophages can persist means that any possible therapeutic intervention to eradicate HIV might have to target two very different types of cells." Last spring, this laboratory lead by J. Victor Garcia, Ph. D., professor of medicine, microbiology and immunology at UNC School of Medicine, demonstrated the ability of tissue macrophages to support HIV replication in vivo in the total absence of human T cells. But how macrophages would respond to antiretroviral therapy (ART) and whether macrophages represented a reservoir for HIV after treatment were unknown. Macrophages are myeloid lineage cells that have been implicated in HIV pathogenesis and in the trafficking of virus into the brain. Using a humanized myeloid-only mouse (MoM) model devoid of T cells, Garcia and his team showed that ART strongly suppresses HIV replication in tissue macrophages. Yet when HIV treatment was interrupted, viral rebound was observed in one third of the animals. This is consistent with the establishment of persistent infection in tissue macrophages. "This is the first report demonstrating that tissue macrophages can be infected and that they respond to antiretroviral therapy," Honeycutt said. "In addition, we show that productively infected macrophages can persist despite ART; and most importantly, that they can reinitiate and sustain infection upon therapy interruption even in the absence of T cells -- the major target of HIV infection." Now that Garcia and his team know HIV persists in macrophages, the next step will be to determine what regulates HIV persistence in tissue macrophages, where in the body persistently infected macrophages reside during HIV treatment and how macrophages respond to possible therapeutic interventions aimed at eradicating HIV from the body. The UNC School of Medicine team collaborated with scientists in UNC's Department of Biostatistics, the Theoretical Division at Los Alamos National Laboratory, Veterans Affairs San Diego Healthcare System, and the Departments of Medicine and Pathology at the University of California at San Diego. This study was funded by the National Institute of Mental Health and the National Institute of Allergy and Infectious Diseases of the U.S. National Institutes of Health. The mission of UNC's Institute for Global Health & Infectious Diseases is to harness the full resources of the University and its partners to solve global health problems, reduce the burden of disease, and cultivate the next generation of global health leaders. Learn more at http://www. .


Successful clinical trials to create drugs and vaccines for next pandemic disease will rely on building capacity, community engagement, and international collaboration before and during outbreak WASHINGTON - Mobilization of a rapid and robust clinical research program that explores whether investigational therapeutics and vaccines are safe and effective to combat the next infectious disease epidemic will depend on strengthening capacity in low-income countries for response and research, engaging people living in affected communities, and conducting safety trials before an epidemic hits, says a new report from the National Academies of Sciences, Engineering, and Medicine. Using key lessons learned from the Ebola epidemic in West Africa, the report outlines how to improve the speed and effectiveness of clinical trial research while an epidemic is occurring, especially in settings where there is limited health care and research infrastructure. The research and development of therapeutics and vaccines is a long, complex, and expensive process and cannot be compressed into the course of a rapidly progressing outbreak. The development of a drug "from bench to bedside" is estimated, on average, to take at least 10 years and cost $2.6 billion, with less than 12 percent likelihood of eventual licensing. Therefore, making progress on the research and development of products - such as therapeutics and vaccines - before an epidemic breaks is the only way to ensure that promising candidates are ready for trials once an outbreak occurs, said the committee that carried out the study and wrote the report. In addition, clinical trials could be more rapidly planned, approved, and implemented during an outbreak if promising products are studied through Phase 1 or Phase 2 safety trials in advance of an outbreak and if emergency response planning includes clinical research considerations and clinical researchers in the discussions from the beginning. The 2014-2015 Ebola epidemic was the longest and most deadly Ebola outbreak since the virus was first discovered in 1976, resulting in 28,616 cases and 11,310 deaths in Guinea, Liberia, and Sierra Leone. In August 2014, the World Health Organization declared the epidemic a public health emergency of international concern. Researchers discussed how to conduct clinical trials on potential Ebola therapeutics and vaccines in West Africa, and ultimately, several teams conducted formal clinical trials in the Ebola-affected countries during the outbreak. The clinical trial teams overcame immense logistical obstacles encountered while trying to design and implement trials in West Africa in the midst of a rapidly spreading epidemic of a highly dangerous contagious disease. However, none of the therapeutic trials ended with conclusive results on product efficacy, although limited evidence from the trial for the ZMapp treatment did trend toward a possible benefit. Given the resources, time, and effort put into these trials, they were not as successful as they could have been. The results of the vaccine trials were more fruitful. Two Ebola vaccine candidates have data that suggest they may be safe and produce an immune response, and one is most likely protective, but further data are needed. Planning and conducting clinical research during the Ebola epidemic also required confronting a number of ethical issues, such as whether it was ethical to conduct clinical trials at all in the midst of a public health emergency and whether the research activities drew effort away from providing clinical care to the most people possible. There was also disagreement among researchers over how clinical trials should be designed during the Ebola epidemic, particularly whether trials should use randomization and concurrent control groups. Randomized controlled trials are generally the preferred research design, because they allow researchers to directly compare the outcomes of similar groups of people who differ only in the presence or absence of the investigational agent. However, many argued that randomized controlled trials would be unethical during the Ebola epidemic, as this trial design would deprive patients of an agent that could potentially prevent or treat Ebola, given the high mortality rate and lack of known and available treatment options. The committee concluded that randomized controlled trials are both ethical and the fastest and most reliable way to identify the relative benefits and risks of investigational products, and except in rare circumstances, every effort should be made to implement them during epidemics. The issues that influenced choices about trial design during the Ebola epidemic - such as community mistrust, the feasibility of a standard-of-care-only arm, the high and variable mortality rate, limited product availability, and the potential conflicts between research and care - are likely to recur in future epidemics. Nevertheless, the perceived ethical or logistical hurdles that these issues present are not sufficiently compelling to override the benefits of randomized trials. Rather, randomized trials may be the most ethical trial design, because they offer the fastest route to identifying beneficial treatments while minimizing the risks of exposure to potentially harmful investigational agents. To improve the national and international clinical trial response to the next epidemic, the committee focused on three main areas - strengthening capacity, engaging communities, and facilitating international coordination and collaboration - both in the period of time before an outbreak strikes and during the epidemic itself. The committee found major capacity challenges that hindered and slowed the research response to the Ebola epidemic, and recommended developing sustainable health systems and research capabilities, improving capacity to collect and share clinical and epidemiological data, facilitating the mechanisms for rapid ethics reviews and legal agreements before an epidemic occurs, and incorporating research systems into emergency preparedness and response systems for epidemics. Affected communities had considerable fear, mistrust, and misunderstanding of national and international response and research staff. Community members feared going to health care facilities for the treatment of Ebola, rumors spread that Ebola was deliberately brought to the region by foreigners, and initial response efforts did not take into account community traditions and beliefs. For example, mandatory cremation policies countered deeply held religious beliefs. Successful clinical research is dependent on a community's understanding of, engagement in, and sense of involvement and respect in the process of planning and conducting research, the committee found. Community engagement should be prioritized during epidemic responses and be a continuous and evolving effort, starting at the onset of the epidemic. Research and response efforts were also greatly affected by the relationships among international stakeholders and their ability to coordinate and collaborate. For example, there were a few Ebola-specific therapeutic candidates with suggestive efficacy available at the beginning of the outbreak that could have been investigated in clinical trials, but the mechanism to prioritize which should be studied first was limited. The committee recommended the establishment of an international coalition of stakeholders to work between epidemics that would advise and prioritize pathogens to target for research and development, develop generic clinical trial design templates, and identify teams of clinical research experts who could be deployed to assist with research during an outbreak. The committee also highlighted seven critical steps to launching successful clinical trials when the next epidemic first strikes and before it peaks. The steps are to collect and share patient information and establish standards of care, engage communities and establish mutual trust, integrate research efforts into response and facilitate stakeholder coordination, prioritize vaccines and therapies and select trial designs, negotiate contracts, consult with regulators, and perform independent ethics reviews. The study was sponsored by the U.S. Department of Health and Human Services' Office of the Assistant Secretary for Preparedness and Response, National Institutes of Health, and U.S. Food and Drug Administration. The National Academies of Sciences, Engineering, and Medicine are private, nonprofit institutions that provide independent, objective analysis and advice to the nation to solve complex problems and inform public policy decisions related to science, technology, and medicine. The National Academies operate under an 1863 congressional charter to the National Academy of Sciences, signed by President Lincoln. For more information, visit http://national-academies. . A roster follows. Copies of Integrating Clinical Research Into Epidemic Response: The Ebola Experience are available from the National Academies Press at http://www. or by calling 1-800-624-6242. Reporters may obtain a copy from the Office of News and Public Information (contacts listed above). Gerald T. Keusch, M.D.* (co-chair) Professor of Medicine and Global Health Boston University Schools of Medicine and Public Health Boston Keith McAdam, M.D. (co-chair) Emeritus Professor of Clinical and Tropical Medicine London School of Hygiene and Tropical Medicine London Abdel Babiker, Ph.D. Professor of Epidemiology and Medical Statistics Medical Research Council Clinical Trials Unit at University College London London Susan S. Ellenberg, Ph.D. Professor of Biostatistics Perelman School of Medicine University of Pennsylvania Philadelphia Roger J. Lewis, M.D., Ph.D.* Professor and Chair of the Department of Emergency Medicine Harbor-UCLA Medical Center Los Angeles Alex London, Ph.D. Professor of Philosophy, and Director of the Center for Ethics and Policy Carnegie Mellon University Pittsburgh Michelle M. Mello, Ph.D.* Professor of Law Stanford University School of Medicine and School of Law Stanford, Calif. Olayemi Omotade, M.D. Professor of Pediatrics and Child Health Institute of Child Health University College Hospital University of Ibadan Ibadan, Nigeria Fred Wabwire-Mangen, Ph.D. Associate Professor of Epidemiology and Public Health Makerere University School of Public Health Kampala, Uganda


- Data from GEN-003 Phase 2 trial indicate initial course of injections sustains clinical and virologic efficacy for at least 24 months - - End of Phase 2 meeting successfully completed for GEN-003; expect to be Phase 3-ready by end of 2017 - CAMBRIDGE, Mass., May 04, 2017 (GLOBE NEWSWIRE) -- Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing T cell-directed vaccines and immunotherapies, today reported financial results for the first quarter of 2017 and announced several positive clinical developments on GEN-003, the company’s candidate immunotherapy for the treatment of genital herpes. In a Phase 2 study, GEN-003 demonstrated sustained reductions compared to baseline in the genital lesion rate (percent of days with genital lesions) and the viral shedding rate (percent of days with detectable virus) in genital herpes patients 24 months after dosing across multiple dose groups (see detailed data below). In addition, Genocea has now successfully completed its End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) and continues to expect GEN-003 to be ready for Phase 3 development by the end of 2017. Chip Clark, president and chief executive officer of Genocea commented on the results: “These long-term durability data reinforce our conviction that GEN-003 could become the cornerstone treatment for patients with genital herpes. We believe the data suggest a single course of treatment of GEN-003 could offer significant clinical, virologic and convenience benefits to patients generally, and especially those dissatisfied with current treatments, for at least 2 years with no maintenance dosing. Given our successful End of Phase 2 meeting with the FDA, we continue to plan a Phase 3 program design consistent with previous guidance. We believe that these new data, together with the body of positive clinical results to date and these FDA discussions, give us momentum to advance our pioneering product candidate to Phase 3 readiness this year.” “Since our last earnings announcement,” Mr. Clark added, “we have also progressed our immuno-oncology programs. Together with our advisory board of prominent immuno-oncology experts, we reviewed and refined our development plans for GEN-009, our lead neoantigen cancer vaccine candidate, and we remain confident in our plans to file an IND for this differentiated vaccine candidate this year. Furthermore, we recently presented data on our ATLAS™ antigen identification platform at two important oncology conferences – the Keystone Symposium on immuno-oncology and at AACR 2017 – as well as at the World Vaccine Congress, where we were invited to give an oral presentation on the potential benefits that ATLAS can bring to neoantigen identification and immune response profiling for immuno-oncology.” GEN-003 Phase 2 Clinical Results The 24-month clinical results announced today come from an extension of a Phase 2 dose-optimization study that started in 2014 and enrolled 310 subjects from 17 institutions in the United States. Subjects were randomized to one of six dosing groups of either 30 µg or 60 µg of each of two protein antigens paired with one of three Matrix-MTM adjuvant doses (25 µg, 50 µg, or 75 µg). A seventh group received placebo. Subjects received three doses of GEN-003 or placebo at 21-day intervals and no maintenance doses were given. Baseline viral shedding and genital lesion rates were established for each subject in a 28-day observation period prior to the commencement of dosing. This 28-day observation period was repeated immediately after the completion of dosing and at six, 12, and 24 months following dosing. After the 28-day observation period immediately after dosing, patients in the placebo arm were rolled over across the six active dose groups under a separate protocol. Throughout the trial, GEN-003 continued to demonstrate a safety profile appropriate for its therapeutic setting, as determined by the trial’s independent Drug Monitoring Committee. Genocea has already advanced the two most promising doses (60 µg per protein combined with either 50 or 75 µg of adjuvant) from this Phase 2 trial into an ongoing Phase 2b efficacy trial, from which positive 6-month, placebo-controlled clinical efficacy data were announced in January 2017. The efficacy of GEN-003 at these two dose levels over the course of this Phase 2 extension study is as follows: Notes: * Mean rate reduction vs. pre-dosing baseline levels. Statistical analysis performed using a Poisson mixed effect model with empirical variance: Magaret, Amalia, "Models for HSV shedding must account for two levels of overdispersion" ((January 2016). UW Biostatistics Working Paper Series. Working Paper 410) ** Per prospectively defined clinical trial protocol, descriptive results only, no statistical testing performed. Financial Guidance Genocea expects that its existing cash, cash equivalents and investments are sufficient to support its operating expenses, capital expenditure requirements and debt obligations into the first quarter of 2018, without assuming any receipt of proceeds from potential business development partnerships or equity financings. This guidance assumes commencing Phase 3 trials for GEN-003 for genital herpes around the end of 2017 and filing an IND for GEN-009 for cancer by the end of the year, however it is Genocea’s strategy to secure additional sources of financing in advance of starting GEN-003 Phase 3 clinical trials. Conference Call Genocea will host a conference call and webcast today at 9:00 a.m. ET. The conference call may be accessed by dialing (844) 826-0619 for domestic participants and (315) 625-6883 for international callers and referencing the conference ID number 13446635. A live webcast of the conference call will be available online from the investor relations section of the Company's website at http://ir.genocea.com. A webcast replay of the conference call will be available on the Genocea website beginning approximately two hours after the event, and will be archived for 30 days. About Genocea Biosciences, Inc. Genocea is harnessing the power of T cell immunity to develop life-changing vaccines and immunotherapies. While traditional immunotherapy discovery methods have largely used predictive methods to propose T cell targets, or antigens, Genocea has successfully developed ATLAS™, its proprietary technology platform, to identify clinically relevant antigens of T cells based on actual human immune responses. Genocea used ATLAS to identify the antigens in its lead clinical candidate, GEN-003, an investigational immunotherapy to treat genital herpes, and is currently using ATLAS in immuno-oncology applications to develop neoantigen cancer vaccines and to identify T cell response signatures of cancer patients treated with checkpoint blockade therapies to potentially improve clinical practice. Genocea expects GEN-003 to be ready to begin Phase 3 development and to file an IND for its neoantigen cancer vaccine candidate GEN-009 by the end of 2017. For more information, please visit the company's website at www.genocea.com. Forward-Looking Statements Statements herein relating to future business performance, conditions or strategies and other financial and business matters, including expectations regarding clinical developments, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act. Genocea cautions that these forward-looking statements are subject to numerous assumptions, risks and uncertainties, which change over time. Factors that may cause actual results to differ materially from the results discussed in the forward-looking statements or historical experience include risks and uncertainties, including Genocea's ability to progress any product candidates in preclinical or clinical trials; the ability of ATLAS to identify promising oncology vaccine and immunotherapy product candidates; the scope, rate and progress of its preclinical studies and clinical trials and other research and development activities; anticipated clinical trial results; current results may not be predictive of future results; even if the data from preclinical studies or clinical trials is positive, regulatory authorities may require additional studies for approval and the product may not prove to be safe and efficacious; Genocea's ability to enter into future collaborations with industry partners and the government and the terms, timing and success of any such collaboration; risks associated with the manufacture and supply of clinical and commercial product; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; Genocea's ability to obtain rights to technology; competition for clinical resources and patient enrollment from drug candidates in development by other companies with greater resources and visibility; the rate of cash utilized by Genocea in its business and the period for which existing cash will be able to fund such operation; Genocea's ability to obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity or debt financing or otherwise; general business conditions; competition; business abilities and judgment of personnel; the availability of qualified personnel and other factors set forth under "Risk Factors" in Genocea's Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and other filings with the Securities and Exchange Commission (the "SEC"). Further information on the factors and risks that could affect Genocea's business, financial conditions and results of operations is contained in Genocea's filings with the SEC, which are available at www.sec.gov. These forward-looking statements speak only as of the date of this press release and Genocea assumes no duty to update forward-looking statements.


- Data from GEN-003 Phase 2 trial indicate initial course of injections sustains clinical and virologic efficacy for at least 24 months - - End of Phase 2 meeting successfully completed for GEN-003; expect to be Phase 3-ready by end of 2017 - CAMBRIDGE, Mass., May 04, 2017 (GLOBE NEWSWIRE) -- Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing T cell-directed vaccines and immunotherapies, today reported financial results for the first quarter of 2017 and announced several positive clinical developments on GEN-003, the company’s candidate immunotherapy for the treatment of genital herpes. In a Phase 2 study, GEN-003 demonstrated sustained reductions compared to baseline in the genital lesion rate (percent of days with genital lesions) and the viral shedding rate (percent of days with detectable virus) in genital herpes patients 24 months after dosing across multiple dose groups (see detailed data below). In addition, Genocea has now successfully completed its End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) and continues to expect GEN-003 to be ready for Phase 3 development by the end of 2017. Chip Clark, president and chief executive officer of Genocea commented on the results: “These long-term durability data reinforce our conviction that GEN-003 could become the cornerstone treatment for patients with genital herpes. We believe the data suggest a single course of treatment of GEN-003 could offer significant clinical, virologic and convenience benefits to patients generally, and especially those dissatisfied with current treatments, for at least 2 years with no maintenance dosing. Given our successful End of Phase 2 meeting with the FDA, we continue to plan a Phase 3 program design consistent with previous guidance. We believe that these new data, together with the body of positive clinical results to date and these FDA discussions, give us momentum to advance our pioneering product candidate to Phase 3 readiness this year.” “Since our last earnings announcement,” Mr. Clark added, “we have also progressed our immuno-oncology programs. Together with our advisory board of prominent immuno-oncology experts, we reviewed and refined our development plans for GEN-009, our lead neoantigen cancer vaccine candidate, and we remain confident in our plans to file an IND for this differentiated vaccine candidate this year. Furthermore, we recently presented data on our ATLAS™ antigen identification platform at two important oncology conferences – the Keystone Symposium on immuno-oncology and at AACR 2017 – as well as at the World Vaccine Congress, where we were invited to give an oral presentation on the potential benefits that ATLAS can bring to neoantigen identification and immune response profiling for immuno-oncology.” GEN-003 Phase 2 Clinical Results The 24-month clinical results announced today come from an extension of a Phase 2 dose-optimization study that started in 2014 and enrolled 310 subjects from 17 institutions in the United States. Subjects were randomized to one of six dosing groups of either 30 µg or 60 µg of each of two protein antigens paired with one of three Matrix-MTM adjuvant doses (25 µg, 50 µg, or 75 µg). A seventh group received placebo. Subjects received three doses of GEN-003 or placebo at 21-day intervals and no maintenance doses were given. Baseline viral shedding and genital lesion rates were established for each subject in a 28-day observation period prior to the commencement of dosing. This 28-day observation period was repeated immediately after the completion of dosing and at six, 12, and 24 months following dosing. After the 28-day observation period immediately after dosing, patients in the placebo arm were rolled over across the six active dose groups under a separate protocol. Throughout the trial, GEN-003 continued to demonstrate a safety profile appropriate for its therapeutic setting, as determined by the trial’s independent Drug Monitoring Committee. Genocea has already advanced the two most promising doses (60 µg per protein combined with either 50 or 75 µg of adjuvant) from this Phase 2 trial into an ongoing Phase 2b efficacy trial, from which positive 6-month, placebo-controlled clinical efficacy data were announced in January 2017. The efficacy of GEN-003 at these two dose levels over the course of this Phase 2 extension study is as follows: Notes: * Mean rate reduction vs. pre-dosing baseline levels. Statistical analysis performed using a Poisson mixed effect model with empirical variance: Magaret, Amalia, "Models for HSV shedding must account for two levels of overdispersion" ((January 2016). UW Biostatistics Working Paper Series. Working Paper 410) ** Per prospectively defined clinical trial protocol, descriptive results only, no statistical testing performed. Financial Guidance Genocea expects that its existing cash, cash equivalents and investments are sufficient to support its operating expenses, capital expenditure requirements and debt obligations into the first quarter of 2018, without assuming any receipt of proceeds from potential business development partnerships or equity financings. This guidance assumes commencing Phase 3 trials for GEN-003 for genital herpes around the end of 2017 and filing an IND for GEN-009 for cancer by the end of the year, however it is Genocea’s strategy to secure additional sources of financing in advance of starting GEN-003 Phase 3 clinical trials. Conference Call Genocea will host a conference call and webcast today at 9:00 a.m. ET. The conference call may be accessed by dialing (844) 826-0619 for domestic participants and (315) 625-6883 for international callers and referencing the conference ID number 13446635. A live webcast of the conference call will be available online from the investor relations section of the Company's website at http://ir.genocea.com. A webcast replay of the conference call will be available on the Genocea website beginning approximately two hours after the event, and will be archived for 30 days. About Genocea Biosciences, Inc. Genocea is harnessing the power of T cell immunity to develop life-changing vaccines and immunotherapies. While traditional immunotherapy discovery methods have largely used predictive methods to propose T cell targets, or antigens, Genocea has successfully developed ATLAS™, its proprietary technology platform, to identify clinically relevant antigens of T cells based on actual human immune responses. Genocea used ATLAS to identify the antigens in its lead clinical candidate, GEN-003, an investigational immunotherapy to treat genital herpes, and is currently using ATLAS in immuno-oncology applications to develop neoantigen cancer vaccines and to identify T cell response signatures of cancer patients treated with checkpoint blockade therapies to potentially improve clinical practice. Genocea expects GEN-003 to be ready to begin Phase 3 development and to file an IND for its neoantigen cancer vaccine candidate GEN-009 by the end of 2017. For more information, please visit the company's website at www.genocea.com. Forward-Looking Statements Statements herein relating to future business performance, conditions or strategies and other financial and business matters, including expectations regarding clinical developments, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act. Genocea cautions that these forward-looking statements are subject to numerous assumptions, risks and uncertainties, which change over time. Factors that may cause actual results to differ materially from the results discussed in the forward-looking statements or historical experience include risks and uncertainties, including Genocea's ability to progress any product candidates in preclinical or clinical trials; the ability of ATLAS to identify promising oncology vaccine and immunotherapy product candidates; the scope, rate and progress of its preclinical studies and clinical trials and other research and development activities; anticipated clinical trial results; current results may not be predictive of future results; even if the data from preclinical studies or clinical trials is positive, regulatory authorities may require additional studies for approval and the product may not prove to be safe and efficacious; Genocea's ability to enter into future collaborations with industry partners and the government and the terms, timing and success of any such collaboration; risks associated with the manufacture and supply of clinical and commercial product; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; Genocea's ability to obtain rights to technology; competition for clinical resources and patient enrollment from drug candidates in development by other companies with greater resources and visibility; the rate of cash utilized by Genocea in its business and the period for which existing cash will be able to fund such operation; Genocea's ability to obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity or debt financing or otherwise; general business conditions; competition; business abilities and judgment of personnel; the availability of qualified personnel and other factors set forth under "Risk Factors" in Genocea's Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and other filings with the Securities and Exchange Commission (the "SEC"). Further information on the factors and risks that could affect Genocea's business, financial conditions and results of operations is contained in Genocea's filings with the SEC, which are available at www.sec.gov. These forward-looking statements speak only as of the date of this press release and Genocea assumes no duty to update forward-looking statements.


ROCKVILLE, Md., May 04, 2017 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (Nasdaq:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that preclinical data from studies supported by REGENXBIO at the University of Pennsylvania’s Gene Therapy Program and Center for Advanced Retinal and Ocular Therapeutics and at the Johns Hopkins Wilmer Eye Institute will be shared in one presentation and four posters at upcoming conferences including the Retinal Cell and Gene Therapy Innovation Summit, the Association for Research in Vision and Ophthalmology (ARVO), and the American Society of Gene and Cell Therapy (ASGCT). These data support further clinical research regarding the use of REGENXBIO’s investigational gene therapy RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). “RGX-314 has the potential to be a one-time treatment for people with wet AMD by delivering high expression of anti-VEGF antibodies through the use of our NAV AAV8 vector. We are pleased to share additional positive preclinical results, which were generated by our development partners at the University of Pennsylvania and Johns Hopkins, which support our active IND,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “REGENXBIO is on track to begin enrollment in the RGX-314 Phase I clinical trial by mid-2017 and to provide an interim trial update by the end of 2017.” Details of the upcoming presentation and posters are as follows: Title: Preclinical gene therapy studies to select RGX-314 doses to treat wet age-related macular degeneration Presenter: Jean Bennett, PhD, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA Session date/time: Friday, May 5, 9:20 a.m. – 9:30 a.m. EDT Session title: Gene Therapy, Outcome Measures, and Novel Therapies, Session 1: Preclinical Aspects — Vector Design/Animal Models Room: Holiday 1-3, Hilton Baltimore, Baltimore, MD Posters at Association for Research in Vision and Ophthalmology Title: RGX-314, an AAV8 expressing an anti-VEGF protein, strongly suppresses subretinal neovascularization and vascular leakage in mouse models  Authors: Ji-kui Shen1, Yuanyuan Liu1, Seth D. Fortmann1, Stephen Yoo3, Karen Kozarsky2, Jiangxia Wang1, Peter A. Campochiaro1. 1Ophthalmology, Johns Hopkins Wilmer Eye Inst, Baltimore, Maryland, United States; 3REGENXBIO Inc, Rockville, Maryland, United States Session date/time: Sunday, May 7, 8:30 a.m. – 10:15 a.m. EDT  Session title: Cytokines; Growth factors; Antiangiogenic drugs  Room: Exhibit/Poster Hall, Baltimore Convention Center, Baltimore, MD Abstract number: B0230  Title: Subretinal delivery of RGX-314 AAV8-anti-VEGF Fab gene therapy in NHP Authors: Anna Tretiakova1, Tomas S. Aleman3, Arkady Lyubarsky3, Elaine J. Zhou4, Erik Wielechowski1, Gui-Shuang Ying2, Erin Bote1, Leah Makaron1, Stephen Yoo5, Jean Bennett3,6, Albert M. Maguire3,6, James Wilson1. 1Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States; 2Center for Preventative Ophthalmology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania, United States; 3Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, United States; 4Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States; 5REGENXBIO, Rockville, Maryland, United States; 6Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States Session date/times: Wednesday, May 10, 11:00 a.m. – 12:45 p.m. EDT Session title: Gene editing and gene therapies Room: Exhibit/Poster Hall, Baltimore Convention Center, Baltimore, MD Abstract number: B0164 Title: Normal parameters of the full field ERG recorded with bipolar electrodes in Cynomolgus Macaque (Macaque fascicularis)  Authors: Arkady Lyubarsky1,2, Erik Wielechowski3, Tomas S. Aleman4, Albert M. Maguire1,4, Gui-Shuang Ying4, Erin Bote3, Leah Makaron3, James Wilson3, Jean Bennett1,4, Anna P. Tretiakova3. 1Center for Advanced Retinal and Ophthalmic Therapeutics, SOM Univ. of Pennsylvania, Philadelphia, Pennsylvania, United States; 2Vision Research Center, University of Pennsylvania, Philadelphia, Pennsylvania, United States; 3Gene Therapy Program, University of Pennsylvania SOM, Philadelphia, Pennsylvania, United States; 4Scheie Eye Institute, University of Pennsylvania SOM Ophthalmology, Philadelphia, Pennsylvania, United States Session date/times: Thursday, May 11, 8:30 a.m. – 10:15 a.m. EDT  Session title: Retinal Function – ERG studies  Room: Exhibit/Poster Hall, Baltimore Convention Center, Baltimore, MD Abstract number: B0441 Additional information on the meeting can be found on the ARVO website: http://www.arvo.org Poster at American Society of Gene and Cell Therapy Title: Safety of RGX-314 AAV8-anti-VEGF Fab Gene Therapy in NHP Following Subretinal Delivery Authors: Tomas S. Aleman1, Anna P. Tretiakova2, Arkady L. Lyubarsky1, Jessica I. W. Morgan2, Elaine J. Zhou3, Erik Wielechowski2, Gui-Shuang Ying4, Erin Bote2, Leah Makaron2, Stephen Yoo5, Jean Bennett1, Albert M. Maguire1, James M. Wilson2. 1Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA,2Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA,3Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA,4Center for Preventative Ophthalmology and Biostatistics, University of Pennsylvania, Philadelphia, PA,5REGENXBIO, Rockville, MD. Session date/times: Thursday, May 11, 5:15 p.m. – 7:15 p.m. EDT Session title: Neurologic Diseases (including Ophthalmic and Auditory Diseases) II Room: Exhibit Hall A & B South, Marriot Wardham Park Hotel, Washington, DC Abstract number: 427 Additional information on the meeting can be found on the ASGCT website: http://www.asgct.org Penn has licensed certain Penn-owned AAV intellectual property to REGENXBIO, including rights related to RGX-314. Dr. Wilson is an advisor to REGENXBIO and is a founder of, holds equity in, and receives sponsored research support from REGENXBIO. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Licensees are applying the NAV Technology Platform in the development of a broad pipeline of product candidates in multiple therapeutic areas. This press release contains “forward-looking statements,” within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, REGENXBIO’s research, development and regulatory plans in connection with its NAV Technology Platform and gene therapy treatments. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could cause actual results to differ materially from those projected by such forward-looking statements. All of REGENXBIO’s development timelines could be subject to adjustment depending on recruitment rate, regulatory agency review and other factors that could delay the initiation and completion of clinical trials. Meaningful factors which could cause actual results to differ include, but are not limited to, the timing of enrollment, commencement and completion of REGENXBIO’s clinical trials; the timing and success of preclinical studies and clinical trials conducted by REGENXBIO and its development partners; the ability to obtain and maintain regulatory approval of REGENXBIO’s product candidates and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing REGENXBIO’s product candidates; REGENXBIO’s ability to obtain and maintain intellectual property protection for REGENXBIO’s product candidates and technology; REGENXBIO’s growth strategies; REGENXBIO’s competition; trends and challenges in REGENXBIO’s business and the markets in which REGENXBIO operates; REGENXBIO’s ability to attract or retain key personnel; the size and growth of the potential markets for REGENXBIO’s product candidates and the ability to serve those markets; the rate and degree of market acceptance of any of REGENXBIO’s product candidates; REGENXBIO’s ability to establish and maintain development partnerships; REGENXBIO’s expenses and revenue; regulatory developments in the United States and foreign countries; the sufficiency of REGENXBIO’s cash resources and needs for additional financing; and other factors discussed in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of REGENXBIO’s Annual Report on Form 10-K for the year ended December 31, 2016. In addition to the risks described above and in REGENXBIO’s filings with the Securities and Exchange Commission, other unknown or unpredictable factors also could affect REGENXBIO’s results. There can be no assurance that the actual results or developments anticipated by REGENXBIO will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, REGENXBIO. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. REGENXBIO cautions investors not to rely too heavily on the forward-looking statements REGENXBIO makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). REGENXBIO undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.


News Article | May 23, 2017
Site: www.eurekalert.org

Boston, MA - Patients admitted to major teaching hospitals are less likely to die compared with patients admitted to minor teaching or non-teaching hospitals, according to a large national study from Harvard T.H. Chan School of Public Health. The study will be published online May 23, 2017 in JAMA (Journal of the American Medical Association). (Link to study after embargo lifts: http://jamanetwork. ) "While we know that teaching hospitals fulfill an important mission around teaching and research, we have known less about the quality of care they provide," said Ashish Jha, K.T. Li Professor of Health Policy at Harvard Chan School, director of the Harvard Global Health Institute, and senior author of the study. "We find that across a very wide range of medical and surgical conditions, patient receiving care at teaching hospitals have superior outcomes." Previous studies have compared outcomes at U.S. teaching hospitals -- those affiliated with medical schools, at which medical students are trained -- with outcomes at non-teaching hospitals, and those studies have suggested that patients generally fare better at teaching hospitals. But many of the seminal studies on the topic are decades old. For the new study, researchers analyzed data for 21.5 million hospitalizations of Medicare beneficiaries at 4,483 hospitals across the U.S -- 250 major teaching hospitals, 894 minor teaching hospitals, and 3,339 non-teaching hospitals -- between 2012 and 2014. The study looked at 30-day mortality rates for 15 common medical conditions, such as pneumonia, congestive heart failure, and stroke, and for six surgical procedures, including hip replacement, coronary artery bypass grafting, and colectomy. The 30-day mortality rate was 8.1% at major teaching hospitals, 9.2% at minor teaching hospitals, and 9.6% at non-teaching hospitals, the study found. The overall 30-day mortality difference between major teaching hospitals and non-teaching hospitals was 1.5%. This pattern -- with major teaching hospitals showing better 30-day outcomes -- persisted even after the researchers adjusted for patient characteristics such as age and severity of illness, and hospital characteristics such as size and profit status. Major teaching hospitals also had lower 7-day and 90-day mortality rates -- by 0.3% and 1.6%, respectively--than non-teaching hospitals. Speculating as to why teaching status was linked with lower mortality, the authors said it's possible that teaching hospitals have greater experience treating particular conditions or may be earlier adopters of technologies and treatments that yield better outcomes for patients. But they said more research is necessary to understand the mechanisms behind the association. The study examined mortality rates only among the Medicare population, so it wasn't possible to determine whether the findings are generalizable among non-elderly populations. "Academic medical centers provide a unique environment, with 24-hour availability of specialty services, advanced technologies, and some of the most expert physicians in the country," said Laura Burke, instructor of health policy and management at Harvard Chan School, emergency physician at Beth Israel Deaconess Medical Center, and lead author of the study. "This seems to pay off for patients. While obviously not all patients can receive care in major teaching hospitals, understanding which strategies and resources are particularly important to patient outcomes, and how they can be replicated among non-teaching hospitals, is critically important to improve care for all patients." Other Harvard Chan School researchers involved in the study included Austin Frakt, visiting associate professor, and E. John Orav, associate professor in the Department of Biostatistics. Funding for the study came from the Association of American Medical Colleges. The funders had no role in the design or execution of the project and no input into the writing of the manuscript. "Association between teaching status and mortality in US hospitals," Laura G. Burke, Austin B. Frakt, Dhruv Khullar, E. John Orav, Ashish K. Jha, JAMA, online May 23, 2017, doi: 10.1001/jama.2017.5702 Visit the Harvard Chan School website for the latest news, press releases, and multimedia offerings. Harvard T.H. Chan School of Public Health brings together dedicated experts from many disciplines to educate new generations of global health leaders and produce powerful ideas that improve the lives and health of people everywhere. As a community of leading scientists, educators, and students, we work together to take innovative ideas from the laboratory to people's lives--not only making scientific breakthroughs, but also working to change individual behaviors, public policies, and health care practices. Each year, more than 400 faculty members at Harvard Chan School teach 1,000-plus full-time students from around the world and train thousands more through online and executive education courses. Founded in 1913 as the Harvard-MIT School of Health Officers, the School is recognized as America's oldest professional training program in public health.


News Article | May 10, 2017
Site: globenewswire.com

DURHAM, N.C., May 10, 2017 (GLOBE NEWSWIRE) -- Argos Therapeutics Inc. (NASDAQ:ARGS), an immuno-oncology company focused on the development and commercialization of individualized immunotherapies based on the Arcelis® precision immunotherapy technology platform, today provided an update on the ADAPT trial, a randomized, active controlled, open-label, multi-center Phase 3 trial of Rocapuldencel-T in combination with sunitinib/standard-of-care for the treatment of newly diagnosed metastatic renal cell carcinoma (mRCC), following a meeting with the FDA. As previously reported, the Company has continued to conduct the ADAPT trial notwithstanding the recommendation by the Independent Data Monitoring Committee in February 2017 to terminate the trial for futility. In making this determination, Argos considered, among other factors, the degree of maturity of the data set, the mechanism of action of Rocapuldencel-T, which involves the induction of a long-term memory immune response, and the IDMC's assessment of the safety profile of Rocapuldencel-T. Of note, at the time of the IDMC's February interim analysis, the median duration of follow-up was 20.0 months and more than half the patients in both treatment groups were still alive. The Company submitted information related to its analysis of the interim data to the FDA and met with the FDA to discuss the future direction of the ADAPT trial and the Rocapuldencel-T development program. Participating in the meeting along with representatives from the Company were Robert Figlin, MD, Chairman of Hematology Oncology and Professor of Medicine, Cedars Sinai Medical Center; Nizar Tannir, MD, Professor and Deputy Chairman, Department of Genitourinary Medical Oncology, MD Anderson Cancer Center; and Gary Koch, PhD, Professor of Biostatistics, University of North Carolina. The FDA agreed with the Company’s plan to continue the trial in accordance with the current protocol to 290 events, the pre-specified number of events at which the analysis of overall survival, the primary endpoint, is to be conducted. The Company believes that 290 events will have occurred by late 2017 or early 2018. The Company also proposed to submit, and the FDA agreed to review, a protocol amendment to increase the pre-specified number of events for the primary analysis of overall survival beyond 290 events, which the Company believes could enhance its ability to detect whether Rocapuldencel-T has a delayed treatment effect. The Company can extend the study past 290 events without needing to enroll additional patients. As previously reported, the Company has analyzed interim data from a predefined subset of patients who demonstrated an immune response to Rocapuldencel-T at 48 weeks, whose immune response is consistent with the mechanism of action of the therapy and correlates with survival, suggesting that the treatment is biologically active. Analysis of the data from the ADAPT trial, including immune response data, remains ongoing. The Company expects to provide further updates on the future direction of the ADAPT trial and the Rocapuldencel-T program following further analysis of the data from the trial and further discussions with the FDA. “We are pleased to be able to continue the ADAPT trial,” noted Robert Figlin, MD, principal investigator for the trial. “We believe that Rocapuldencel-T may offer patients and their physicians an important new option for the treatment of mRCC, a disease that remains an area of high unmet medical need. By amending the protocol to extend the ADAPT trial, we believe we can potentially increase the likelihood of detecting a treatment effect, if one exists, given that immunotherapy is expected to result in a delayed treatment effect. We appreciate the collaborative efforts of the FDA as we seek to determine the potential utility of Rocapuldencel-T in the treatment of this difficult disease.“ “We remain committed to the clinical development of Rocapuldencel-T, and look forward to providing additional updates on the ADAPT trial and the Rocapuldencel-T development program moving forward,” noted Jeff Abbey, CEO of Argos. “We appreciate the continued commitment of the investigators and patients in the ADAPT trial as we continue to explore the potential benefit of this unique therapy.” Arcelis® is a precision immunotherapy technology that captures both mutated and variant antigens that are specific to each patient's individual disease. It is designed to overcome immunosuppression by producing a specifically targeted, durable memory T cell response without adjuvants that may be associated with toxicity. The technology is potentially applicable to the treatment of a wide range of different cancers and infectious diseases, and is designed to overcome many of the manufacturing and commercialization challenges that have impeded other personalized immunotherapies. The Arcelis® process uses only a small disease sample or biopsy as the source of disease-specific antigens, and the patient's own dendritic cells, which are optimized from cells collected by a single leukapheresis procedure. The proprietary process uses RNA isolated from the patient's disease sample to program dendritic cells to target disease-specific antigens. These activated, antigen-loaded dendritic cells are then formulated with the patient's plasma, and administered via intradermal injection as an individualized immunotherapy. Argos Therapeutics is an immuno-oncology company focused on the development and commercialization of individualized immunotherapies for the treatment of cancer and infectious diseases using its Arcelis® technology platform. Argos' most advanced product candidate, Rocapuldencel-T, is being evaluated in the pivotal ADAPT Phase 3 clinical trial for the treatment of metastatic renal cell carcinoma (mRCC). In addition, Rocapuldencel-T is being studied in a Phase 2 investigator-initiated clinical trial as neoadjuvant therapy for renal cell carcinoma (RCC). Argos is also developing a separate Arcelis®-based product candidate, AGS-004, for the treatment of human immunodeficiency virus (HIV), which is currently being evaluated in an investigator-initiated Phase 2 clinical trial aimed at HIV eradication in adult patients. Any statements in this press release about Argos' future expectations, plans and prospects, including statements about the ADAPT trial and the interim data from the trial, Argos' anticipated discussions with the FDA, clinical development of Argos' product candidates and future expectations and plans and prospects for Argos and other statements containing the words "believes," "anticipates," "estimates," "expects," "intends," "plans," "predicts," "projects," "targets," "may," "potential," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether Argos' cash resources will be sufficient to fund its continuing operations for the periods anticipated and through completion of the trial; the impact of the planned analysis of the data and discussions with the FDA on the development of Rocapuldencel-T; the impact of the recommendation of the IDMC on the continuation of the ADAPT trial; whether preliminary or interim clinical data such as the interim data referenced in this release will be indicative of the final data from a clinical trial; whether results obtained in clinical trials will be indicative of results obtained in future clinical trials; whether Argos' product candidates will advance through the clinical trial process on a timely basis; whether the results of such trials will warrant submission for approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether Argos' product candidates will receive approval from regulatory agencies on a timely basis or at all; whether, if product candidates obtain approval, they will be successfully distributed and marketed; whether Argos can successfully establish commercial manufacturing operations on a timely basis or at all; and other factors discussed in the "Risk Factors" section of Argos' Form 10-K for the year ended December 31, 2016, which is on file with the SEC, and in other filings Argos makes with the SEC from time to time. In addition, the forward-looking statements included in this press release represent Argos' views as of the date hereof. Argos anticipates that subsequent events and developments will cause Argos' views to change. However, while Argos may elect to update these forward-looking statements at some point in the future, Argos specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Argos' views as of any date subsequent to the date hereof.

Loading Biostatistics collaborators
Loading Biostatistics collaborators