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July 29, 2013 Breaking News and Market Intelligence for the Clinical Trials Industry CRA acquires Comprehensive division…2 A CenterWatch Publication PhRMA and EFPIA endorse trial data sharing principles…3 Survey: Pharma/biotech companies do better job than most providing career development, flexibility P harmaceutical and biotechnology companies, increasingly concerned about the high cost and challenge of replacing skilled employees, have done a better job than companies from other industries investing in their workers’ careers and adopting policies to keep them engaged, according to a new survey released by Randstad Pharma, a global staffing and HR services firm. Pharma/biotech companies received a higher rating from employees (52%) compared to companies in other industries (35%) for creating career development and training programs for their workers. And 60% of pharma/biotech workers reported their employers provide regular performance reviews, compared with only 44% of employees polled across all industries. The Pulse on Study Conduct…6 The survey identified career advancement programs as one of the most important issues for employees: 29% of workers in the pharma/biotech sector rated training and professional development as one of top three areas they value in their workplace. In addition, a higher percentage of pharma/biotech employees (48%) said their companies offered flexible hours or working arrangements, compared to 41% across all industries. One-third of pharma/biotech employees want this type of flexibility in the workplace, making it the top-ranked benefit among pharma/biotech respondents. “Clients in this space are investing more in these areas that are important to employees,” said Greg Coir, president of Randstad page 4 » WCG Academy to offer protocol-specific training T he WIRB-Copernicus Group (WCG) has launched a new partnership to provide state-of-the-art, customized online training for clinical research professionals that includes competency exams to measure site staff understanding of specific study protocols. WCG has partnered with UL Quality, Compliance and Learning (formerly EduNeering)—which has built a cloudbased platform used by the FDA to train and certify its inspectors in quality and compliance—to form the WCG Academy. The academy gives sponsors and CROs online tools to train investigative site staff and ensure their understanding of the protocol throughout a clinical study. Courses, however, are not lim- ited to site personnel and can be used across the clinical research enterprise. The Academy’s protocol training offers both generalized and customized, studyspecific training on protocols for site personnel. Study-specific protocol training includes optional competency tests and measures of understanding. Additionally, study-specific protocol training includes FAQs and checklists site personnel can consult to follow protocol requirements. “This is especially important, since site personnel may have a high turnover rate during the course of a multi-year trial,” said Donald Deieso, Ph.D., executive chairman of the WCG. “Having online courses and Volume 17, Issue 30. © 2013 CenterWatch. All rights reserved. page 5 » Drug & Device Pipeline News…7 CenterWatch has identified 22 drugs and devices that have entered a new trial phase this week. Trial Results…8 CenterWatch reports on results for four drugs. Biotech Review…9 Biotech briefs from BioWorld Today. CWMarketplace…10 Industry service provider profiles. CenterWatch Information Services The CenterWatch Monthly A monthly newsletter featuring in-depth stories on the clinical trials industry and study lead opportunities. Annual subscriptions start at $399. JobWatch A web-based service listing clinical research jobs, career resources and a searchable resume database. Clinical Trials Data Library A valuable online resource providing access to comprehensive data and charts on the life sciences and clinical research industry. Clinical Trials Listing Service™ An international listing service of actively enrolling clinical trials to support sponsors and CROs in their patient enrollment initiatives. Market Intelligence Services Custom surveys for organizations to gain competitive insight into the market and their business. Drugs in Clinical Trials Database A searchable database of 4,000+ detailed profiles of new drugs in development. CenterWatch also prepares custom drug intelligence reports covering a variety of medical conditions. Medical Writing Solutions Comprehensive medical writing services for the biotechnology, medical device and pharmaceutical industries. CenterWatch Publications CenterWatch publishes a wide range of CME-accredited training guides, directories, brochures and drug intelligence information. Visit http://store.centerwatch.com. CenterWatch Main and Editorial Offices 10 Winthrop Square, Fifth Floor, Boston, MA 02110 Tel (617) 948-5100 Fax (617) 948-5101 [email protected] / [email protected] CWWeekly July 29, 2013 2 of 10 Industry Briefs Study Conduct ■■ Tempe, Ariz.-based Clinical Research Advantage (CRA), a wholly owned network of clinical trial sites, has expanded both its geographic and therapeutic reach by acquiring the late phase division of Comprehensive Clinical Development (CCD). CRA said the acquisition will allow it to offer pharmaceutical sponsors and CROs an across-the-board solution for their diverse clinical research needs. Founded in 1992, CRA includes 63 sites operating under CRA and Radiant Research. Both brands conduct stage II-IV clinical trials. CRA focuses on family practice and general medicine, while Radiant conducts multi-specialty and consumer trials. CRA acquired Radiant Research in 2012. CCD conducts psychiatric clinical trials, specifically in schizophrenia, bipolar disorder, depression and Alzheimer’s disease. CCD has participated in the development of every major psychiatric drug that has been developed over the past 10 years. “By adding Comprehensive Clinical Development to the CRA family of trial sites we are able to increase our capabilities to meet the needs of the growing pharmaceutical marketplace,” said Mark S. Hanley, chief executive officer of CRA. Based in Miramar, Fla., CCD was founded in 1998 and operates four independent sites in Atlanta, Ga., Cerritos, Calif., Holliswood, N.Y., and Washington, D.C. The combined companies will continue to function under the names CRA and CCD, but will be looking to integrate and streamline their operations where appropriate. well as technical support, for the development and production of pharmaceuticals, diagnostics, and drug/device combination products from discovery through Phase IIb. The majority of VxP Pharma’s labs are in the U.S., plus facilities in Europe and Asia. All VxP sites have been fully audited by CRO Solutions/VxP Pharma, as well as by global pharmaceutical and biotechnology clients. Most sites have also been audited by the FDA and other regulatory agencies. ■■ ResearchDx, a contract diagnostics organization (CDO), has purchased and will relocate to a 30,000-square-foot facility in Irvine, Calif. The facility will support continued expansion of in vitro diagnostic (IVD) development services to the pharmaceutical, biotechnology, diagnostic development and clinical laboratory industries. According to ResearchDx, new healthcare initiatives toward personalized medicine and the corresponding need for IVD tests to come to market alongside pharmaceuticals as companion diagnostics (CDx) is changing the way drug companies target diseases and treatments for patients. R&D Trends ■■ Venture capitalists invested $6.7 billion in 913 deals in the second quarter of 2013, according to the MoneyTree Report from PricewaterhouseCoopers (PwC) and the National Venture Capital Association (NVCA), based on data provided by Thomson Reuters. Quarterly VC investment activity rose 12% in terms of dollars and 2% in the number of deals compared to the first quarter of 2013 when $6 billion was invested in 896 deals. The biotechnology sector saw increases in both dollars and number of deals. Companies receiving VC funding for the first time, as well as those companies in the early stage of development, experienced jumps in dollars invested. “As the exit window continues to open, we’ll continue to see VCs shifting their focus back to companies in the earlier stages of development,” said Mark McCaffrey, global technology partner and software leader at PwC U.S. “In particular, startups that are able to drive innovation by developing disruptive technologies that are easy to deploy and deliver ongoing value to the user will be of great interest to venture capitalists.” Biotechnology was the second largest sector for dollars invested with $1.3 billion going into 103 deals, up 41% in dollars and 4% in deals from the prior quarter. Medical device investing of $543 million into 71 deals represented a 1% decline in both dollars and deals. Investments in the life sciences sector overall (biotechnology and medical devices) rose 25% in dollars and 2% in deals, purely on the strength of biotech investing. Early stage dollar investments rose to their highest level in six quarters, rising 63% in dollars and 18% in deals, to $2.5 billion going into 480 deals. The average early stage deal was $5.2 million, up significantly from $3.7 million in the prior quarter. Seed/early stage deals accounted for 57% of total deal volume in Q2, compared to 51% in the first quarter. First-time financing (companies receiving venture capital for the page 3 CROs/Service Providers ■■ CRO Solutions has launched VxP Pharma, a wholly owned subsidiary headquartered in Indianapolis. VxP Pharma is comprised of the combined services of the independently owned and operated “niche” CROs of CRO Solutions. VxP Pharma will offer pharmaceutical development services, as CWWeekly (ISSN 1528-5731) Cheryl Appel Rosenfeld Editor-in-Chief Tracy Lawton, Stephanie Hill Drug Intelligence Melissa Nazzaro Advertising Holly Rose Production Send news submissions to Cheryl Appel Rosenfeld Tel (617) 948-5172 Fax (617) 948-5101 [email protected] © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. » © 2013 CenterWatch, LLC. All rights reserved. No part of this publication may be distributed or reproduced in any form or by any means without the express written consent of the publisher. Permission requests can be obtained via fax at (617) 948-5101 or emailed at [email protected]. Single-user annual subscriptions are $249. Reprints and discounted multi-reader or corporate subscription rates are available. Email [email protected] or call (617) 948-5100. CWW1730 CWWeekly July 29, 2013 3 of 10 Industry Briefs (continued from page 2) first time) dollars increased 24% to $1.1 billion going into 302 companies in Q2, a 10% increase in the number of deals from Q1. The life sciences sector experienced a dramatic increase in first-time financing, more than doubling in dollars from Q1 to $328 million, with 33 life sciences companies receiving VC funding for the first time in Q2, up from only 22 companies in Q1. ■■ Wisconsin-based nonprofit Aurora Health Care’s Research Institute will launch a quarterly peer-reviewed research journal The Journal of Patient-Centered Research and Reviews, with the first edition expected in early 2014. The journal will be available online with limited print copies. It will serve as a resource for Aurora researchers to publish original research and reviews for the medical community, and will be geared to a multi-specialty audience in medicine, healthcare and basic science. It will be indexed to major databases, including Medline, and searchable in archives such as PubMed, hosted by the U.S. National Library of Medicine. Dennis Baumgardner, M.D., was named editor and will lead a scientific editorial board. The journal will be accessible to academic medical centers, universities, medical institutions and the public. Ethics/Regulatory ■■ The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the Pharmaceutical Research and Manufacturers of America (PhRMA) have endorsed joint Principles for Responsible Clinical Trial Data Sharing: Our Commitment to Patients and Researchers. “Biopharmaceutical companies commit to enhance these efforts by making additional information available to the public, patients who participate in clinical trials and to qualified researchers,” said Christopher Viehbacher, president of EFPIA and CEO of sanofi. “The data-sharing commitments reflect EFPIA member companies’ strong support for sharing clinical trial data to benefit patients and foster scientific discovery in a way that maintains patient privacy, the integrity of regulatory systems and incentives to invest in biomedical research,” said Richard Bergström, director general of EFPIA. Companies will dramatically increase the amount of information available to researchers, patients and the public: Patientlevel trial data, study-level trial data, full clinical study reports and protocols for medicines approved in the U.S. and E.U. will be shared with scientific and medical researchers upon request and subject to terms necessary to protect patient privacy and confidential commercial information; researchers will be encouraged to publish their findings; companies will work with regulators to provide a factual summary of trial results to participating patients; and the synopses of clinical study reports submitted to the FDA, EMA or national authorities of E.U. member states will be made available after approval. At a minimum, results from all phase III trials and results of significant medical importance should be submitted for publication. Implementation begins January 2014. Sponsors ■■ ■■ Vivus and New York-based First Manhattan (FMC), an investment advisory firm and the owner of 9.9% of Vivus’ outstanding shares, entered a settlement agreement in their proxy contest. Five members of the Vivus board resigned, and six FMC nominees were appointed. The board was expanded from nine to 11 members. Anthony P. Zook was named Vivus’ new CEO and was appointed to the board. Zook previously was executive vice president, global commercial operations for AstraZeneca, and president of Medimmune. He succeeds Leland Wilson, who will depart as CEO but has agreed to serve in an advisory role to ensure a seamless leadership transition. Vivus’ board also appointed Michael Astrue chairman. He previously was CEO of Transkaryotic Therapies and chairman of the Massachusetts Biotechnology Council. Vivus sells obesity drug Qsymia in the U.S. “We intend to move quickly on our four main goals: expand use of Qsymia through targeted patient and physician education, find the right partner for Qsymia, quickly create a pathway for approval in Europe and eliminate expenses not essential to expanding use of Qsymia,” said Astrue. Bristol-Myers Squibb will open a North America capability center in January 2014 and create 579 life sciences jobs by 2017 in Tampa, Fla. The Tampa Bay region is gaining reputation as a hub for R&D in life sciences; over 600 life sciences companies employing more than 13,200 operate in Tampa. The world’s local central lab. Global reach. Local expertise. © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1730 CWWeekly July 29, 2013 4 of 10 Features (continued from page 1) Survey Pharma, a division of Randstad Holdings that focuses on the clinical side of the pharma/biotech industry. “They are listening. They are allowing for more flexibility.” The Randstad Pharma survey was designed to measure employee attitudes and perceptions about their companies along with factors driving employee engagement and retention. The survey, conducted online in March and April, surveyed 3,282 adults currently employed full time in a range of industries; the survey included 161 respondents from the pharma/biotech sector. While the survey found pharma/biotech companies have done a better job than companies in other industries in addressing employee concerns about the workplace environment, it also showed companies in this sector need to provide more opportunities for career advancement to keep their skilled employees from moving to other organizations. Half of respondents (49%) said they feel they must change jobs in order to advance their careers, and 37% indicated they will seek a job in a different company within the next six months. Another 43% said they would be likely to accept an enticing offer from another company. “For the most part, these are highly motivated people who are looking for career pro- “For the most part, these are highly motivated people who are looking for career progression. If their current employer is not offering professional development and career path, we see that as a motivating factor for people wanting to make a change more so than compensation.” —Greg Coir, president, Randstad Pharma gression,” said Coir. “If their current employer is not offering professional development and career path, we see that as a motivating factor for people wanting to make a change more so than compensation.” Coir said pharma/biotech companies have made greater investments, compared to companies in other industries, in training programs and professional development, and have allowed more flexible working arrangements, as they have recognized the significant cost and detriment of losing good talent. The independent Center for American Progress estimates replacing an employee costs approximately one-fifth of that worker’s annual salary. In addition, Coir said other costs of losing an employee in the pharma/biotech sector are difficult to quantify. For example, when an experienced study manager in a highly specialized clinical trial leaves, the study might stop until a replacement can be found. There is a high demand for talent in this space, said Coir, particularly in clinical operations, biometrics, safety and medical writing. “If you lose an employee, it’s really challenging to find someone with the right skill set.” The survey results suggested the need for more targeted retention strategies and investments to keep employees engaged in their jobs. In addition to flexible work arrangements and professional development programs, respondents said they want high-performing workers to receive bonuses and promotions (30%). And while 47% of employees in this sector said their companies have formal systems in place to recognize or reward top employees, compared to 25% in all industries, only 39% said their companies offered promotions or bonuses to high-performing employees. “While we see positive momentum within the industry,” Coir said, “companies must continue to put an emphasis on engagement strategies.” —Karyn Korieth eCOA FORUM © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1730 CWWeekly July 29, 2013 5 of 10 Features (continued from page 1) WCG Academy briefing materials available 24/7 will assure greater continuity of critical knowledge.” Deieso said ensuring the competency of those who design, manage and conduct clinical trials is critical to protecting human subjects and meeting the scientific objectives of clinical research. “Unfortunately, there are too many incidents in which a trial fails because site personnel didn’t understand the protocol,” he said. “Trials are conducted by human beings. Human beings need to have the requisite knowledge to do well. If they don’t have the knowledge, then the trial is jeopardized.” As drug sponsors and CROs adopt online site training programs to reduce costs and increase compliance, other vendors also have integrated protocol-specific training into their offerings. Firecrest Clinical, for example, which was acquired by global CRO Icon, offers customized training modules that allow users to learn study-specific information needed for the day-to-day conduct of a clinical trial. HealthCarePoint, which recently launched online education courses in partnership with CDISC, offers tools for CROs and sponsors to develop their own protocol-specific training. Other CROs and vendors offer similar training modules. The WCG Academy provides global site staff access to more than 100 online courses; those offered as part of the Good Clinical Practice (GCP) curriculum are authored or reviewed by the FDA through a partnership between the agency and UL. Courses, which are offered in 17 languages, provide train- “We feel this partnership will foster transformation in clinical research by improving the quality of clinical trial conduct and ensuring human safety.” —Lynne Budnovitch, general manager, UL Quality, Compliance and Learning ing on FDA ethics and safety requirements, research updates and best clinical practices. The system also can distribute study protocols directly to sites. Once the study begins, the WCG Academy provides site staff with a simple checklist of critical protocol elements they can reference at any time with questions; for example, if a patient is a few days late for a visit, staff can refer to an online FAQ to determine the proper course of action. The online resource also allows training for site personnel hired after the clinical study has begun, keeps track of when site staff need to renew training and provides refresher courses to avoid any regulatory exposure, study delays or operational inefficiencies. “We can also tell if someone hasn’t done well and has to repeat it,” said Deieso. “The important thing is not that they had to repeat it, but that they have the requisite knowledge.” The creation of the WCG Academy, which was announced last month, is part of a plan to bring together disparate parts of the ethical review process to create greater efficiency in clinical research. WCG companies include Western Institutional Review Board (WIRB), Copernicus Group Institutional Review Board (CGIRB) and IRBNet, a research compliance software application supporting IRBs in the U.S. Earlier this year, the WCG formed an exclusive partnership with Mytrus, a clinical technology and services company, to offer an internet-based application that replaces paper-based processes for obtaining patient consent for clinical trial participation. “We feel this partnership will foster transformation in clinical research by improving the quality of clinical trial conduct and ensuring human safety,” said Lynne Budnovitch, general manager of UL. Deieso said the WCG Academy is an important part of his company’s plan to build a “one-stop” integrated solution for the ethical oversight process. Sponsors and CROs that use WIRB-Copernicus ethical review boards can negotiate a contract that also includes Mytrus’ electronic informed consent process and training through the WCG Academy. “This would be instead of having several contracts. For example, if you were going to train on a protocol, it would be good to have the IRB who approved the protocol to author and check the course to be sure that it’s consistent precisely with what was approved,” Deieso said. “We want our group of companies to make one circle of all the knowledge-related services in a clinical trial. We hope to make an impact.” —Karyn Korieth Are your monitors running out of yellow s1ckies? > Get unstuck. Use coordinators. Trained. Experienced. Dedicated. © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. 513.247.5500 CWW1730 CWWeekly July 29, 2013 The Pulse on Study Conduct M 6 of 10 By Elizabeth Weeks-Rowe y position in study start-up requires I evaluate a diverse group of investigative sites for study consideration, from large academic institutions that cover several city blocks and dedicated research sites with the latest in high-tech diagnostic equipment to the small, yet effective, single investigator private practice. This process affords the opportunity to engage with investigators and site staff and discover the reasons they succeed, or struggle, with the trials they conduct. The unanimous goal of site staff during the selection process is to positively represent their site and capabilities to sponsor representatives. Sometimes sponsors presume larger, dedicated research organizations with the staff and expertise to rapidly expedite contract and essential documents—and the budget to employ dedicated recruitment staff for rapid patient recruitment—are the logical first choice. Alternatively, renowned academic institutions often are headed by Key Opinion Leaders who helped develop protocol content, so these site selection visits sometimes are mere formalities and their participation is nearly guaranteed. This leaves the smaller site hybrid of private practice/investigative site as a secondary, or even tertiary, choice, which can leave it feeling overlooked and less important compared to its larger counterparts. Recently, I conducted a site selection teleconference with an investigator being considered for participation in a respiratory study. After the protocol discussion with the investigator and his staff, I began the most crucial part of the meeting, the one-on-one discussion with the lead study coordinator. The majority of site logistical and clinical information is obtained here, and so it can have a great impact on the site selection decision, depending on the quality of data obtained. This particular investigator was in private practice in a small town in the Northwest, and his staff consisted of his medical assistant, study coordinator and receptionist. I have learned over the years that establishing an easy dialogue with the study coordinator offers far more insight into the inner workings Key questions to ask sites at study start-up 1. Has the site had any prior FDA audits/483 letters? 2. How long does it take for a contract and budget to be executed? 3. How long does it take for IRB approval of a study? 4. Does the investigator have experience as a PrincipaI Investigator and experience in the indication under study? 5. Does the investigator have a backup? 6. Does the site have any competing studies and how will it manage them all? 7. Does the site have an experienced study coordinator as well as backup? of a site than an impersonal “follow your checklist” exchange in which facts, not foundation, are gathered. I make it a point to identify a commonality, which will serve as the impetus for the dialogue I require to discover the facts. This particular study coordinator was not participating in the banter. She was extremely businesslike and a bit defensive, which led me to forego friendly exchange for the checklist procedure. I was curious about her atypical behavior, but I had a job to do and proceeded accordingly. Our exchange went something like this: Me: “Please could you give me the name and contact information for those individuals responsible for contracts and budgets.” Study coordinator: “That would be me.” Me: “OK. Then may I have the name and contact information for the individual responsible for regulatory documents?” Study coordinator: “Also me.” Me: “Then may I have the name and contact information for the data manager?” Study coordinator: “Again, that would be me.” After a pause, she volunteered insight into her initial attitude: Study coordinator: “Look, I will save you a lot of question time. I do everything: contracts, regulatory, data entry, phlebotomy, patient care, specimen processing, spirometry. My backup is the medical assistant. We don’t have a recruiter © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. or extra staff. We aren’t a big site like the others you visit. It is just us, and we do the best we can.” I began to see a picture of a small investigative site that conducted only several studies a year, competing and sometimes failing against bigger institutions. I could see an experienced and proficient study coordinator who was a little defensive explaining the site’s small structure, perhaps as a result of past experiences with individuals whose idea of the ideal site was quite narrow and who did not understand that the quality of investigators and data produced has nothing to do with how large the institution, how shiny the equipment or how lauded the investigator. I told the study coordinator my experience with smaller sites, for the most part, had been extremely positive. Using discretion in the manner and number of studies they conducted often led to steady enrollment and quality data. I also explained that, in my experience, having one or two individuals responsible for everything often was more consistent and efficient than sites with an army of individuals responsible for key activities, which required more correspondence, more follow-up and, sometimes, less efficiency. Her response made me smile: “Oh” was all she said. And then I could see her mood brighten, and the resulting conversation led to an informative exchange during which I gleaned an accurate picture of the site’s experience and process. This ultimately led to site selection. In research, as in life, it is best not to judge a book by its cover—or by its size, equipment or reputation. Elizabeth Blair Weeks-Rowe, LVN, CCRA, has spent more than a dozen years in a variety of clinical research roles including CRA, CRA trainer, CRA manager and business development director. She has written and edited newsletters for several CROs, created training curriculum for CRA/clinical research educational and training programs, and is a contributing writer to several research publications. She currently works in relationship development/study start-up in the CRO industry. Email [email protected]. CWW1730 CWWeekly July 29, 2013 7 of 10 Drug & Device Pipeline News Company Drug/Device Medical Condition Status Sponsor Info Genmab HuMax-TFADC multiple solid tumors IND filed with the FDA (609) 430-2481 www.genmab.com Ablynx ALX-0171 pediatric respiratory syncytial virus Phase I trials initiated +32 (0)9 262 00 00 [email protected] Arrowhead Research ARC-520 chronic hepatitis B Phase I trials initiated (626) 304-3400 [email protected] Pulmatrix PUR0200 chronic obstructive pulmonary disease Phase Ib trials initiated (781) 357-2333 [email protected] Invion INV102 (nadolol) chronic bronchitis Phase II trials initiated enrolling 136 subjects in the U.S. +61 7 3295 0500 http://invion.com.au/ Invion INV103 mild systemic lupus erythematosus Phase II trials initiated enrolling 32 subjects in the U.S. +61 7 3295 0500 http://invion.com.au/ Merrimack MM-111 Pharmaceuticals advanced gastric, esophageal and gastroesophageal junction cancers Phase II trials initiated (617) 441-1000 http://merrimackpharma.com/ OncoGenex OGX-427 Pharmaceuticals metastatic bladder cancer Phase II trials initiated enrolling (425) 686-1500 180 subjects in the U.S. and Europe www.oncogenex.com Akebia Therapeutics AKB-6548 anemia Phase IIb trials initiated (513) 985-1920 www.akebia.com MediciNova MN-166 progressive multiple sclerosis Phase IIb trials initiated (858) 373-1500 [email protected] Cubist bevenopran Pharmaceuticals chronic pain, opioid-induced constipation Phase III trials initiated enrolling 1,800 subjects (781) 860-8660 www.cubist.com Imprimis Impracor Pharmaceuticals acute osteoarthritis knee pain Phase III trials planned in the U.S. Josh Berg, (858) 704-4041 http://imprimispharma.com Tesaro niraparib ovarian cancer Phase III trials initiated enrolling 360 subjects www.tesarobio.com University of Louisville adult stem cells severe refractory angina Phase III trials initiated in the U.S. http://louisville.edu/research/ Astellas Astagraf XL immunosuppressant Approved by the FDA (800) 888-7704 www.us.astellas.com Janssen Biotech Simponi Aria rheumatoid arthritis Approved by the FDA (800) 526-7736 www.janssenbiotech.com GlaxoSmithKline Mekinist unresectable or metastatic melanoma Approved by Health Canada http://us.gsk.com/ GlaxoSmithKline Tafinlar unresectable or metastatic melanoma Approved by Health Canada http://us.gsk.com/ ArmaGen Technologies AGT-182 mucopolysaccharidosis type II Orphan Drug designation granted by the FDA (310) 917-1275 [email protected] OXiGENE ZYBRESTAT ovarian cancer Orphan Drug designation granted by the EMA www.oxigene.com Topo Target Belinostat malignant thymomas Orphan Drug designation granted by the EMA +45 39 17 83 92 [email protected] Verastem VS-6063 (defactinib) mesothelioma Orphan Drug designation granted by the FDA (617) 252-9300 www.verastem.com © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1730 CWWeekly July 29, 2013 8 of 10 Trial Results Oncology ■■ ■■ Amgen released results from a phase II study of XGEVA (denosumab) in adults and skeletally mature adolescents diagnosed with giant cell tumor of bone (GCTB). In the international, open-label, phase II study enrolling 282 patients, there were three cohorts: patients with surgically unsalvageable GCTB (Cohort 1), patients with salvageable GCTB whose surgery was associated with severe morbidity (Cohort 2) and patients who transferred from a previous XGEVA GCTB study (Cohort 3). All three cohorts received subcutaneous XGEVA 120mg every four weeks with loading doses on days eight and 15. In Cohort 1, 96% (163/169) of patients had no disease progression after a median follow-up of 13 months. In Cohort 2, 74% (74/100) of patients required no surgery and 62% (16/26) of patients who had surgery underwent a less morbid procedure than planned. XGEVA was approved June 13 by the FDA for the treatment of adults and skeletally mature adolescents with GCTB that is unresectable or where surgical resection is likely to result in severe morbidity. Bayer HealthCare issued results from a phase III study of Xofigo in castration-resistant prostate cancer (CRPC) patients with symptomatic bone metastases. The randomized, double-blind, placebo-controlled, international study enrolled 921 patients in 100 centers in 19 countries. Patients were stratified based on their baseline alkaline phosphatase (ALP) level, current bisphos- phonate use and whether or not they had received docetaxel prior to enrollment. Treatment consisted of up to six intravenous injections separated by four weeks. Xofigo improved overall survival (OS) at the prespecified interim analysis (HR=0.695, [95% CI: 0.552-0.875], p=0.00185); median OS was 14.0 months with Xofigo (95% CI: 12.1-15.8) vs. 11.2 months with placebo (95% CI: 9.0-13.2). These findings were supported by an exploratory analysis performed before patient crossover with an additional 214 events in which Xofigo showed improvement in OS (HR=0.695, [95% CI: 0.581-0.832]); median OS was 14.9 months in the Xofigo arm (95% CI: 13.9-16.1) v. 11.3 months in the placebo arm (95% CI: 10.4-12.8). These data supported the FDA approval of Xofigo injection in May. Hematology ■■ Isis Pharmaceuticals reported results from a phase II trial of ISIS-APOCIII for the treatment of patients with high to severely high triglycerides on stable doses of fibrates. The double-blind, randomized, placebo-controlled, 13-week study enrolled 26 patients who received either a 200mg or 300mg dose of ISIS-APOCIII, or placebo, via weekly subcutaneous injections. All patients were on stable doses of fibrates with average baseline levels of fasting triglycerides between 282mg/dL and 457mg/dL. Patients treated with ISISAPOCIII experienced reductions of up to 70% in apolipoprotein C-III (apoC-III) and up to 64% in triglycerides. In addition, patients treated with ISIS-APOCIII experienced an up to 52% increase in high-density lipoprotein cholesterol (HDL-C), the ‘good’ cholesterol, and an up to 77% reduction in apoC-III-associated very low-density lipoprotein (VLDL) particles. Isis also is evaluating ISIS-APOCIII in this phase II study as a monotherapy in patients with severely high triglycerides. Immunology ■■ Inovio Pharmaceuticals has released results from two phase I trials (HVTN 070 and HVTN 080) of Pennvax-B preventative HIV DNA vaccine, HVTN 070 without electroporation and HVTN 080 with electroporation. Both trials were multicenter, randomized clinical trials, with 070 enrolling 120 patients and 080 enrolling 48 patients. Robust T-cell responses were generated in 89% of subjects who received three vaccinations of Pennvax-B, which consists of 1mg of each of three DNA plasmids (encoding for HIV gag, pol and env proteins) along with 1mg of IL-12 DNA plasmid, followed by intramuscular electroporation with Inovio’s CELLECTRA device. Three or four vaccinations with a 2mg dose of each Pennvax-B plasmid plus 1.5mg of IL-12 DNA generated fewer responses when delivered without electroporation. Six months after vaccination, T-cell response rates remained strong and persistent in the subjects who received only three doses delivered by CELLECTRA EP. Of 24 positive CD4 or CD8 Tcell responders following the third in month three, 79% (19/24) showed persistent CD4 or CD8 T-cell responses at month nine. Clinical Research Conference - 2013 West OCTOBER 27-30, 2013 • THE VENETIAN/Palazzo • LAS VEGAS COMPREHENSIVE: Sponsor Operations • Site Operations • Regulatory Compliance Contracts • Budgets & Billing • Quality Practical Tips Based on Real-Life Examples 90+ Sessions & Workshops in Six Tracks ATTENDEES RATE MAGI CONFERENCES: More Educational (79%) Better Networking (78%) More Practical (87%) Better Value (81%) Save $100 with Discount Code CW152 © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. www.magiworld.org CWW1730 CWWeekly July 29, 2013 9 of 10 Biotech Review ■■ GlaxoSmithKline sent three top global executives to China to deal with an expanding scandal that has seen four executives detained and one confess on national TV. Abbas Hussein, president of global business outside of the U.S., has been tasked with managing the unfolding scandal in China. Hussein said GSK China executives “appear to have acted outside of our processes and controls, which breaches Chinese law. We have zero tolerance for any behavior of this nature.” GSK committed to fully support Chinese authorities’ efforts to reform the medical sector, and it intends to look at its business model in China and proposes lowering drug costs to be more affordable. GSK’s China operations have not had a general manager since Mark Reilly left for the U.K. July 5 for what were supposed to be routine meetings. He has not yet returned. The company’s vice president of finance for China, Steve Nechelput, has been barred from international travel by national authorities. GSK said Nechelput is not under investigation. Authorities are investigating transactions worth $488 billion involving 700 travel agencies. GSK was first notified of the investigation June 27, but it had been underway for six months. The four GSK executives detained by police are all Chinese nationals. A GSK spokesperson would not comment on the status of individuals involved in the investigation. One of the four, VP and operations manager Liang Hong, admitted on national TV to using travel agencies to fake business expenses, pay bribes and increase drug sales. “It means he is hoping for leniency in his own case, perhaps a lighter prison sentence or a suspended sentence,” said Richard Cassin, a lawyer and corruption expert who also publishes the FCPA Blog. “The drug company allegedly arranged for payments to doctors and medical officials in exchange for them buying or recommending the drug company’s products.” In January, China’s top judicial authorities issued an updated interpretation on the enforcement of bribery laws and set out specific punishments for certain industries and provided reduced sentences for suspects who confess. The life sciences industry was singled out. A number of systemic factors make corruption more widespread in China’s healthcare industry, including a fragmented distribution system that leads to large unwieldy sales forces and the low pay of doctors and health officials who supplement their income with benefits from drug makers. Corporate travel agency abuse also is common; many provide legitimate services, but using travel agencies is a way for employees to circumvent company procedures. ■■ ■■ Stockholders in Novadel Pharma of Bridgewater, N.J., approved the sale of substantially all of the company’s assets to Suda of Australia. However, not enough stockholders gave approval for liquidation and dissolution of the company and the proposal did not pass. Novadel will therefore pursue other options. London-based Redx Pharma is moving toward the launch of a third research division, securing a conditional offer from the U.K.’s Regional Growth Fund (RGF) for a unit specializing in metabolic disease. That will complement centers specializing in oncology and anti-infectives, formed with the company in late 2010 around a drug modification and repurposing platform spun out of AstraZeneca. Those two centers employ about 100 staff each, and Neil Murray, CEO of Redx Pharma, said the metabolic unit is expected to be a similar size. Murray said no private money is required because the company is close to signing its first out-licensing deal and has others in negotiation. The new division will be called Redx Metabolic, but Murray said its scope will be wider, covering cardiovascular and inflammatory diseases. If the RGF © 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. grant is confirmed, Redx Metabolic will be up and running by March 2014, with about 300 employees. If that rate of growth is unusual for a biotech startup yet to land its first deal, Murray said Redx is positioning itself in the space left vacant by recent pharma restructurings. ■■ ■■ Biogen Idec of Weston, Mass., said in a report by Reuters News the death of a patient who had taken its multiple sclerosis drug, Tecfidera, was unlikely to be linked to the compound. Biogen said it was made aware of the death of a 59-year-old woman who had been treated with Tecfidera, approved by the FDA in March, and that it was actively gathering facts about the case. In May, Biogen delayed the European launch of Tecfidera until the second half of this year to clarify the drug’s regulatory data protection. Analysts seemed generally unconcerned, with Credit Suisse analyst Ravi Mehrotra calling news stories “a storm in a tea cup.” Quest PharmaTech of Edmonton, Alberta, entered a long-term strategic relationship with AD Biotech of Chuncheon, South Korea. AD Biotech has provided $2 million in clinical development funding to Quest and will provide up to $10 million more in return for Quest common shares and future revenue-sharing. The deal enables Quest to fund its clinical development programs and gives it access to AD Biotech’s antibody, protein transduction domain and manufacturing technologies. The stories included in Biotech Review have been provided to CenterWatch with full permission of BioWorld Today and its publisher, AHC Media LLC, 3525 Piedmont Road, Building 6, Suite 400, Atlanta, GA 30305. Call (800) 688-2421 or (404) 262-5476 for more information, www.bioworld.com. ©2013 AHC Media LLC CWW1730 CWWeekly July 29, 2013 10 of 10 CWMarketPlace CWMarketPlace is a monthly section featuring a range of clinical research service providers who have Industry Provider Profile Pages posted on CenterWatch.com. 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