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Chimerix raises $103M Panelists: Positives amid setbacks in for brincidofovir push; cell therapy, regenerative medicine space potential use widens By Nuala Moran, Staff Writer
By Randy Osborne, Staff Writer
LONDON – There are storm clouds on the horizon for cell therapy and regenerative More than half the gross proceeds of medicine, with a number of trial failures in the past few weeks and the mounting Chimerix Inc.'s $103.8 million from a problems in getting reimbursement for high-cost products that are edging to market, public offering will go toward advancing adding to the huge scientific and development challenges that the sector faces.
the oral nucleotide analogue lipid- Recent news has included negative data in Athersy Inc.'s phase II trial of its allogeneic conjugate drug brincidofovir, the cell therapy in ulcerative colitis, announced April 29, and Cytomedix Inc.'s May 5 company said in its 424B5 filing with the decision to ax its Aldagen subsidiary after its autologous stem cell therapy failed in a Voltarra seeks ‘lightning' Lysogene raises $23M States signal on ‘right strike with zoledronic for Sanfilippo syndrome to try': Washington, acid derivative in knee OA A gene therapy we have a problem By Marie Powers, Staff Writer
By Cormac Sheridan, Staff Writer
By Mari Serebrov, Washington Roundup
Launched last year with a portfolio Lysogene SAS raised €16.5 million Sending a clear signal to Washington, of early stage small molecules and (US$22.6 million) in a series A round Louisiana is poised to become the second late-stage clinical assets assembled to continue its clinical development state to enact a law recognizing the through a pair of acquisitions, Voltarra of SAF-301, a gene therapy treatment right of terminally ill patients, who have Pharmaceuticals Inc. has moved like for Sanfilippo syndrome type A, a rare run out of options, to try investigational THE BIOWORLD BIOME Keeping insulin around Recent start-up Cour Stock movers, p. 2
By Anette Breindl, Science Editor
lands former Pfizer Other news to note, p. 2, 4,6, 8, 9, 10
Logic suggests that raising insulin levels R&D chief as chairman through stabilizing insulin should lead In the clinic, p. 10, 11
to improved glucose control. But in By Michael Fitzhugh, Staff Writer
practice, mice lacking IDE, the enzyme Pharma: Other news to note, p. 12
Cour Pharmaceutical Development that degrades insulin, have impaired Co. Inc., a small company developing Pharma: In the clinic, p. 12
glucose control. nanotechnology-based immunotherapies Now scientists from Harvard University for acute inflammation and autoimmune have developed a pharmacological IDE disease, has appointed Pfizer R&D To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY OTHER NEWS TO NOTE STOCK MOVERS 5/21/2014
Stock in $
Change in %
Adamas Pharmaceuticals Inc., of Emeryville, Calif., received
a $25 million milestone payment from Forest Laboratories
Nasdaq Biotechnology Holdings Ltd., of New York, following the FDA's acceptance
of the new drug application for MDX-8704, a fixed-dose Chimerix Inc.
combination of memantine HCl extended-release capsules and donepezil HCl, in development as a once-daily therapy to treat La Jolla Pharmaceuticals moderate to severe dementia of the Alzheimer's type in the U.S. Biotechs showing significant stock changes Wednesday The companies are collaborating on the development of MDX-8704, for which Forest holds exclusive U.S. commercialization rights. (See BioWorld Today, March 5, 2014.) company's new drug application is filed.
Alnylam Pharmaceuticals Inc., of Cambridge, Mass.,
Cognition Therapeutics Inc., of Pittsburgh, said its small-
said preclinical results using RNAi therapeutics targeting molecule Abeta receptor agonist program for Alzheimer's aminolevulinic acid synthase-1 (ALAS-1) to treat hepatic disease was selected for funding by the National Institute porphyrias, including acute intermittent porphyria (AIP), on Aging NIH Alzheimer's Disease Development Program. were published in the Proceedings of the National Academy of The cooperative agreement program will provide the firm Sciences. In the paper, Alnylam scientists and collaborators with an estimated $1.4 million in funding over four years to at the Icahn School of Medicine at Mount Sinai in New York support critical drug development activities aimed at securing documented results from a mouse model of AIP showing that investigational new drug status.
RNAi therapeutics targeting ALAS-1 can completely block Cytokinetics Inc., of South San Francisco, highlighted three
the abnormal production of toxic intermediates of the heme recently published manuscripts detailing tests of tirasemtiv, its biosynthesis pathway that cause the symptoms and disease skeletal muscle activator and the lead candidate in its skeletal pathology of AIP. In addition, preliminary comparative studies muscle contractility program. The papers, published in PLOS showed that ALAS-1 siRNA administration was more effective ONE, Muscle & Nerve, and the American Journal of Respiratory than heme administration in the treatment of an acute attack. and Critical Care Medicine, described positive early stage data The paper provides proof of concept for an RNAi therapeutic to related to tirasemtiv's impact on a mouse model, dosing in a treat AIP, according to the company, which said it is advancing phase I first-in-human trial, and whether a structural analogue its development candidate, ALN-AS1, designed to knock of tirasemtiv could improve contractile weakness of diaphragm down ALAS-1, and expects to file an investigational new drug muscle fibers that develops in mechanically ventilated critically application by early next year. ill patients.
Antiop Inc., of Lexington, Ky., has signed an agreement to work
Edison Pharmaceuticals Inc., of Mountain View, Calif., said
with Reckitt Benckiser Pharmaceuticals Inc., of Richmond, Va.,
Japanese regulators granted orphan status to EPI-743 for the to accelerate production and worldwide marketing of intranasal treatment of Leigh syndrome. The drug, a small-molecule para- naloxone, its drug for treating opioid overdose. Reckitt Benckiser benzoquinone designed to augment endogenous glutathione and Antiop will co-develop the nasal naloxone spray, which is biosynthesis, is in phase IIb testing in children with the entering its final trial in June, through potential FDA approval. inherited neuromuscular disorder in the U.S., and a phase IIb/III Antiop CEO Daniel Wermeling said he is confident the company trial is being conducted in Japan in conjunction with Dainippon
will meet eligibility requirements for FDA priority review after the Sumitomo Pharma Co. Ltd., of Osaka, Japan.
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partnership is still in place and progressing. "Our job as entrepreneurs is to engage other big pharma companies in the process of [development and stroke trial. But does that mean the sector is in trouble? commercialization] and pull them into a different way of Not according to Greg Bonfiglio, managing partner of Proteus thinking about our treatments," Caplan said. "Big pharma Venture Partners. "I think the field is progressing very nicely," he has to understand we have a cornucopia of molecules that are told BioWorld Today, calling recent events "a natural correction" getting clinically significant results."to a space that "as a whole, is doing quite well."Bonfiglio posed the question during a panel convened to PRICING: BAR "GETS HIGHER EVERY TIME'Such clinically significant data will be the key to getting discuss the state of the industry at this week's World Stem Cells reimbursement for cell therapy products at a time when health & Regenerative Medicine meeting: "Is it a natural downtick, or care budgets are shrinking around the world, said Michael is it an ominous sign something not good is going to happen?" May, CEO of Canada's Center for the Commercialisation of Regenerative Medicine (CCRM) in Toronto.
For Alain Vertes, managing director of Nxr Biotechnologies, Under constrained budgets, the high price of cell therapies who was head of F. Hoffman-La Roche Ltd.'s regenerative presents a huge barrier to market. "You have to displace medicines unit at the point the pharma decided to quit the field, existing technologies, and the bar gets higher every time," May the current problems are a "small accident." Looking to history, said.
for example, 15 of the first 16 clinical studies of cell therapies failed. In the case of acute myocardial infarction, there are about 300 cell therapy trials in progress. But, May said, the standard of "It couldn't have been worse; what we are seeing now is nothing care currently means there is not much headroom. in comparison," he said. The fundamentals of the sector are strong, and there is increasing understanding of the basics of According to a CCRM analysis, the most a payer would the technology.
reimburse for an acute myocardial infarction treatment is $32,000, which is well below the current threshold for a cell The value of Athersys' shares halved when the negative data therapy product.
from the trial of its off-the-shelf stem cell product partnered with Pfizer Inc. were published. (See BioWorld Today, April 29, However, in the case of sepsis, the lack of approved treatments and high mortality rates mean there is much more headroom and payers would be willing to pay $150,000. However, Tim Allsopp, head of external research in regenerative medicines at Pfizer's Neusentis R&D unit in Cambridge, UK, "So you start to see perhaps that acute myocardial infarction said the program is still alive. "It's an ongoing collaboration. We is not where we should be investing," May said. While it may still believe in the partnership, and it was a pretty well designed represent a large market, it will not be easy to get paid for a cell trial," Allsopp said. The treatment was safe and well tolerated.
therapy treatment.
The current position is "quite positive" even though there was CCRM is now examining reimbursement issues at the earliest disappointment on both sides in terms of the data, which came stages of selecting products to take into its manufacturing from an eight-month follow-up of the 128-patient study. Further process development program.
analyses are ongoing, including a subcohort that received a Approaching reimbursement, as the next activity in the repeat dose.
sequence following approval, certainly caused problems for "It's disappointing there was not a stronger effect, but we Tigenix NV, the first company to have an autologous cell should reserve judgement until the final data," Allsopp said.
therapy product approved under the EMA's centralized route.
Allsopp also reported progress in Pfizer's second cell therapy Chondrocelect, for treating knee cartilage injuries, gained program, in which it is collaborating with researchers at approval in 2009, but to date it is only reimbursed in Belgium, University College London, who have generated retinal the Netherlands and Spain. pigment epithelial cells from human embryonic stem cells. "We didn't prepare ourselves in advance," said Maria Pascual, There is UK regulatory approval for a trial in age-related vice president of clinical operations and regulatory affairs at macular degeneration, and Allsopp said Pfizer has signed a Leuven, Belgium-based Tigenix.
manufacturing contract with Roslin Cells Ltd., of Edinburgh, In April, the company threw in the towel and signed over marketing and distribution rights in return for royalties. Now "The product is being manufactured, and it will soon be in the Tigenix is making sure reimbursement is factored into the first patients," Allsopp said.
development of its other products. (See BioWorld Today, April 4, Arnold Caplan, professor of biology at Case Western Reserve University and chief scientific officer at Orthocyte Corp., "You have to prepare for launch before you start phase III," welcomed the fact that the Athersys/Pfizer cell therapy Pascual told delegates. // To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY advisory group for a solid organ transplant study and have several opportunities to meet with that group over the summer to help lead us to what could be a successful study design that SEC, pricing 7.3 million shares at $14.22 each in a deal set to will be clinically relevant," Berrey said.
close next week.
Cowen & Co. analyst Phil Nadeau is optimistic about Chimerix, of Durham, N.C., aims to use about $60 million brincidofovir launching in 2016, based on data from from the public offering to help fund the recently begun phase SUPPRESS. He pointed out in a research report that the III study with brincidofovir for the treatment of adenovirus phase II trial garnered a 73 percent reduction in CMV events. infection, as well as another in kidney transplant patients and "However, there is reason to believe that the reduction will be possibly more.
even greater in phase III," Nadeau wrote. "In phase II, 50 of the Brincidofovir began dosing in the SUPPRESS phase III trial 230 subjects with CMV reactivation had it prior to the first day last year for the prevention of cytomegalovirus (CMV) infection of brincidofovir dosing. In phase III, brincidofovir can be dosed in hematopoietic cell transplant recipients. The compound is prior to engraftment, as early as day one, and therefore few a broad-spectrum antiviral designed to block the replication patients should have reactivation prior to dosing." of double-stranded DNA viruses, including CMV, and deploys Brincidofovir's net could stretch even wider. The in the conjugate cidofovir, a drug currently marketed as compassionate-use program has provided "a wealth of intravenous Vistide by Foster City, Calif.-based Gilead Sciences information on its use to treat a wide range of viral infections, Inc. for treating CMV retinitis in AIDS patients.
in a variety of patient populations," Nadeau wrote. This year, For now, the standard of care in CMV is Valcyte (ganciclovir, the firm will mine the database for new opportunities, which Roche AG), which brings, like Vistide, toxic side effects and could include the John Cunningham (JC) virus, glioblastoma doesn't work to prevent the infection. Valcyte only comes into (apparently linked to CMV, according to some research, and play after stem cell treatments have engrafted, which typically where there have been recent publications establishing a link takes two to four weeks.
to CMV) and human papillomavirus-related papillomatosis. "Interestingly, brincidofovir has been used to treat eight In March, the firm found itself spotlighted in the mainstream patients with JC virus, and has cleared the virus from two," press and scorched in social media for its refusal to let dying Nadeau wrote.
7-year-old Josh Hardy have brincidofovir on a compassionate-use basis. Chimerix solved its ethical conundrum with an open-label Morgan Stanley & Co. LLC and J.P. Morgan Securities LLC are pilot study in immunocompromised patients with adenovirus acting as joint book-running managers for the offering, with infections, making the pivotal phase III study an extension of the Cowen and Co. LLC serving as co-lead manager. William Blair & pilot experiment. (See BioWorld Today, March 13, 2014.) Co. LLC and Canaccord Genuity Inc. are co-managing the deal. Chimerix has granted the underwriters a 30-day option to buy "The adenovirus trial pilot portion was initiated for the main about 1 million more shares.
part to provide access while we were finalizing the study design for the pivotal trial," explained CEO Michelle Berrey during the Shares of Chimerix (NASDAQ:CMRX) gained $1.98, or 13.9 conference call with investors on first quarter earnings. "So percent, to close Wednesday at $16.20. // that pilot portion does not have to have a certain number of patients. It's really to provide access while the study discussions OTHER NEWS TO NOTE are ongoing. We had initially discussed around 20 patients in Glycomimetics Inc., of Gaithersburg, Md., said Pfizer Inc., of
the pilot portion. We are leaving that number open." New York, made a $15 million payment under the companies' Chimerix ended the first quarter with almost $100 million in 2011 collaboration to develop rivipansel (GMI-1070). Under cash, which Piper Jaffray analyst Joshua Schimmer estimated the deal, Pfizer plans to initiate a phase III trial of rivipansel in would last "deep into 2015." With the new money, the firm vaso-occlusive crises in sickle cell patients, which will trigger stands in even better shape to push brincidofovir along. Plans an additional $20 million milestone payment to Glycomimetics for phase III trials for blocking CMV in solid organ transplants upon dosing of the first patient in the trial. (See BioWorld Today, (primarily renal) and treating adenovirus infection are expected Oct. 12, 2011.) to be finalized with the FDA and European regulators by the Hemispherx Biopharma Inc., of Philadelphia, disclosed
end of this year. Enrollment in SUPPRESS should finish around a preliminary-stage agreement required to manufacture the same time, with top-line results expected by mid-2015. Ampligen in Argentina in order to serve Latin American markets "The solid organ transplant study was our number two trial," should Ampligen be approved in Argentina. The agreement Berrey said. "We were trying to get up and running in the first is a prerequisite to the company starting the manufacture of half of 2014, obviously, with the focus on adenovirus and the stability lots, followed by stability testing, necessary to gain opportunity that we had to initiate that trial." approval to manufacture Ampligen. In June 2010, Hemispherx Though the start of the solid organ transplant experiment inked a sales, marketing, distribution and supply agreement likely won't come until next year, "we are initiating our medical with GP Pharm SA, of Barcelona, Spain, for Latin America.
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THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY demonstrated efficacy, but then stalled in the pipeline due to side effects or poor bioavailability.
VOLT01 is the first in line. ZA, administered intravenously, slows lightning – a strategy underpinning the company's name – into the loss of bone mass but is associated with a raft of post-dose phase III trials for its lead candidate. VOLT01 is a derivative of side effects, including flushing, fever, joint pains and muscle zoledronic acid (ZA), a bisphosphonate originally marketed by aches. The VOLT01 formulation is designed to tamp down those Novartis AG in a variety of indications, including osteoporosis, side effects without sacrificing efficacy.
Paget's disease and bone cancers, under the trade names The market could be sizable. The Novartis drug had peak sales Reclast in the U.S., Aclasta in Australia and Zometa. of $2.1 billion in 2011, falling back to less than $1 billion last Bethlehem, Pa.-based Voltarra is collaborating with the year, according to Cortellis Competitive Intelligence.
Menzies Research Institute at the University of Tasmania in Selection of KOA as the drug's initial indication involved a Hobart, Australia, on the multicenter trial, which is assessing bit of serendipity. Several years ago, Menzies conducted a the efficacy and safety of VOLT01 in knee osteoarthritis (KOA).
randomized, placebo-controlled study of ZA vs. placebo in CEO Richard Becker, whose resume includes the oncology patients with KOA, an off-label indication, with the trial partly groups at Bayer Pharmaceuticals, Novartis and Merck & Co. funded by Novartis, Becker explained. Results, published in Inc., formed Voltarra in September 2013 by acquiring the assets 2011, showed that a single 5-mg infusion of ZA in patients with of IMC Biotechnology Inc., which was developing early stage KOA reduced pain and led to shrinkage of bone marrow lesions spleen tyrosine kinase inhibitors and anti-tumor necrosis factor in the study, known as ZAP, which enrolled 59 patients. drugs, and Renascence Pharmaceuticals, which had a portfolio However, the Menzies researchers wanted to follow a larger of late-stage product reformulations. The former CEOs of those group for a longer time period. In June 2013, shortly before companies, IMC's Renee Stewart and Renascence's Ketan Voltarra was formed, the group launched a randomized, Desai, are Voltarra's only other full-time employees, serving as multicenter phase III study, known as ZAP2, comparing ZA to chief scientific officer and chief medical officer, respectively.
placebo. The ongoing trial, funded by grants and involving four Voltarra's early stage assets are on the back burner for now, sites in Australia, is designed to enroll 264 patients with KOA, while the company focuses on moving to market derivatives with efficacy as the primary endpoint.
of compounds that advanced through phase II or beyond, Jefferies 2014 Global Healthcare Conference JUNE 2-5, 2014
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Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY improvements in behavior, attention and sleep were evident in the other three patients. Brain atrophy, measured by nuclear magnetic resonance imaging, appeared stable in two patients neurodegenerative disorder arising from impaired breakdown but increased in two others, including the youngest patient. A of the cell surface and extracellular matrix component heparan follow-up observation trial is ongoing. Meanwhile, Lysogene will embark on a slew of activities in Also called mucopolysaccharidosis III, it is the most prevalent advance of the pivotal studies, including embarking on a and severe of four related lysosomal storage disorders that manufacturing campaign, engaging with drug regulators and have devastating effects on patients' intellectual and motor patient groups, and selecting and preparing clinical trial sites. development and that lead to an early death. It plans to seek a pre-investigational new drug application So far, just four Sanfilippo type A patients have received the meeting with the FDA with a view to including U.S. sites in the therapy in an open-label phase I/II trial. "The next step for us development program. Manufacturing will be carried out at the is to prepare for pivotal studies," Karen Aiach, CEO and co- UCL Partners Gene Therapy Consortium at University College founder of Lysogene, told BioWorld Today. The company aims to London. Aiach's co-founder, Olivier Danos, previously was begin the pivotal program by the end of 2015. In rare diseases, director of that unit and established the AAV facility there. this kind of trajectory is not uncommon. "We even know of The funding round was led by Paris-based Sofinnova Partners, some groups who are aiming to enter directly into phase I/II/III which was also the company's seed investor. Other participants studies in rare diseases with innovative therapies," she said. in the new investment include Bpifrance, through its Innobio The Paris-based firm also plans to commence development of a fund, and Novo Seeds, the early stage investment arm of Novo second gene therapy treatment in an undisclosed indication. It aims to file a clinical trial application by the end of 2016. Sofinnova Partners was attracted to the company on the SAF-301 comprises an adeno-associated viral (AAV) vector basis of the therapeutic potential of its lead program, not its serotype 10 carrying two genes, SGSH and SUMF1, which technological profile as a gene therapy. "For this disease, we encode N-sulfoglucosamine sulfohydrolase and sulfatase- think this is the best approach," Rafaèle Tordjman, managing modifying factor 1, respectively. The former is part of the partner at Sofinnova, told BioWorld Today. "It's not more high- heparan sulfate breakdown pathway and is absent in patients risk than usual. Obviously gene therapy is challenging," she with Sanfilippo syndrome type A. said. But the technical challenge is offset by the more limited In the SAF-301 trial, the construct was injected directly into clinical development requirements. The eventual European the patients' brains via a stereotactic procedure. Each received approval of Uniqure BV's Glybera (alipogene tiparvovec) to treat a dose of 7.2 x 1011 viral genomes, delivered over a two-hour familial lipoprotein lipase deficiency is further encouragement, period from a 12-needle device. "It's mainly a neurological notwithstanding the tortuous approval process the dossier disease, with very mild peripheral symptoms," Aiach said. The went through. "It's probably not the easiest or best case, limited scale of the study means that whatever conclusions because it's not a lethal disease," Tordjman said. can be drawn must necessarily be tentative. "The primary There is no approved therapy for any of the Sanfilippo endpoint was safety," she said. "The second objective of the syndromes as yet. Dublin-based Shire plc has completed a study was to explore potential future efficacy endpoints." The phase I/II trial of an enzyme replacement therapy for type A, construct appeared safe and well tolerated and, despite being SHP610, which it plans to move into a phase IIb trial. Uniqure, on immunosuppressive therapy, the patients did not exhibit an of Amsterdam, the Netherlands, is collaborating with the increase in infection. Pasteur Institute in Paris on a gene therapy approach for SUMF1 plays a role in the post-translational modification and Sanfilippo syndrome type B. A phase I/II trial is ongoing. // catalytic activation of sulfatase enzymes. The company plans to dispense with that aspect of the therapy in the upcoming OTHER NEWS TO NOTE pivotal study – it is present in patients in any case. Imaginab Inc., of Los Angeles, said it formally launched
A report of the trial, titled "Intracerebral Administration Imaginab Japan KK, a wholly owned subsidiary based in Tokyo, of Adeno-Associated Viral Vector Serotype rh.10 Carrying aimed at better meeting the commercial needs of several Human SGSH and SUMF1 cDNAs in Children with significantly clinical development and partnering opportunities, Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/ the company said. The subsidiary will be led by Shintaro II Trial," was published online on May 5, 2014, in Human Gene Therapy. Three of the four patients treated were between 5.5 and 6 years of age, at which point the condition is relatively BIOWORLD™ IS ON TWITTER advanced. All were able to walk, but they had abnormal cognitive abilities and exhibited brain atrophy. The fourth was Stay connected—follow us on Twitter! 2 years and 8 months and, according to the study authors, "was the most likely to display neurocognitive benefit." Modest To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY Regulatory front brincidofovir to a dying 7-year-old boy, whose immune system had been compromised following a round of cancer treatments. The issue for Chimerix was the cost involved, which could delay drugs and devices.
its ongoing development program and, thus, the approval and Unanimously passed by the Louisiana Legislature, the bill was marketing of the drug for other patients. (See BioWorld Today, sent to Gov. Bobby Jindal Tuesday for his signature. HB 891 mirrors the legislation Colorado Gov. John Hickenlooper signed South Plainfield, N.J.-based PTC Therapeutics Inc. ended up in into law Saturday. Other states are following suit. The Missouri court when a patient sued for access to its investigational drug. General Assembly is working on a similar bill, and "right-to-try" While PTC won in district court, it lost on appeal. Rather than will be on the November ballot in Arizona.
draining limited resources in court battles and compassionate The state legislation does nothing to extend compassionate use programs, "the right thing is to get the drug approved use beyond what the FDA already permits, but it shows there and commercially available as rapidly as possible," PTC CEO is a problem with getting access to promising therapies, Rep. Stuart Peltz told BioWorld Today. That way the new treatment is Harold Rogers (R-Ky.) said at Tuesday's House subcommittee available to all patients, not just a select few.
hearing on ways to speed the development of new cures. (See One solution would be for the U.S. to adopt something like BioWorld Today, May 21, 2014.) the EU's conditional approval program, which grants access Citing the lengthy approval process of investigational drugs to some investigational therapies once clinical trials are and devices, the Colorado and Louisiana legislation noted that completed, Peltz said. However, PTC was denied conditional patients with terminal illnesses don't have the luxury of waiting approval in the EU of its ataluren, a protein restoration therapy until a drug or device receives FDA approval.
designed to enable the formation of a functioning protein in Although the legislation recognizes a right to try, it does patients with genetic disorders caused by a nonsense mutation. not force manufacturers to provide investigational drugs or The company is appealing. (See BioWorld Today, May 14, 2014.) devices. It also doesn't require third-party payers to cover the PTC's 48-week, 220-patient confirmatory phase III trial for unapproved therapies. And it shields health care providers ataluren is on track to complete enrollment in a few months, and drug- and devicemakers from liability associated with the with top-line data expected in mid-2015. The process of compassionate use of an investigational product.
developing ataluren from the idea stage has taken 15 to 16 When the experts testifying at the subcommittee hearing years, Peltz said. During that time, the biotech has had to raise were asked about the right-to-try bills, Garry Neil, head of money, develop the clinical path for the novel drug and educate global R&D at Medgenics Inc., said the cost of providing regulators about the disease and the science behind the drug.
an investigational therapy for compassionate use can be CMS FINALIZES RULE prohibitive for small companies.
After withdrawing the most controversial parts of a proposed Sara Radcliffe, executive vice president of the health section rule revising the Medicare Part D prescription drug program, at the Biotechnology Industry Organization, added that the Centers for Medicare & Medicaid Services (CMS) has compassionate use is a difficult, complex issue. The written finalized the rule.
testimony she submitted detailed the growing delays in The final rule, which is to be published in Friday's Federal development that are making compassionate use a front- Register, gives CMS enhanced tools to fight fraud and abuse burner issue with patients.
in the prescription drug program. To ensure that Part D drugs For instance, the duration of the clinical phase of drug are only prescribed by qualified individuals, the rule requires approvals has steadily lengthened from an average of 4.6 years prescribers to enroll in Medicare by June 1, 2015. It also gives in 1990-1994 to an average of 7.1 years in 2005-2009. That CMS the authority to revoke a physician's Medicare enrollment increase has been accompanied by rising protocol complexities for abusive prescribing practices and patterns.
and declining enrollment and retention rates.
Another provision will broaden public access to privacy-protected "Confronting the problem of increasing costs and durations of Part D data, such as unencrypted, prescriber, plan and pharmacy clinical trials is a daunting task," Radcliffe said in her testimony. identifiers contained in prescription drug event records.
While industry is committed to partnering with Congress, The nearly 700-page proposed rule CMS released for comment the FDA, NIH, patients, academia and other stakeholders to in January also would have reduced the number of Part D improve the efficiency of clinical trials to reduce the barriers to plans a sponsor could offer, set standards on requirements new therapies, patients who are out of options don't have time to participate in preferred pharmacy networks, clarified to wait for those talks to produce results. Many of them have noninterference provisions and lifted the protected class turned to social media to pressure drug- and devicemakers to designation for antidepressants, immunosuppressants and, in grant them compassionate use.
the future, antipsychotics.
Chimerix Inc., of Durham, N.C., was recently the target of a The agency backed away from those provisions after it was social media firestorm to get it to provide its antiviral candidate To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY Editor's note: This is an outtake from BioWorld's Bench Press, a weekly addition to the daily news in which BioWorld takes a look at translational medicine. For more science news like this, look inhibitor that they used to probe IDE's role more specifically for the attachment every Monday morning, or visit BioWorld's than knockout mice could. dedicated science portal, The BioWorld Biome: Our Habitat for All In their studies, they showed that in both lean and obese mice, Things Science at IDE regulated not just insulin, but also glucagon and amylin, two hormones that control blood sugar levels. When mice were Regulatory front fed glucose, acute treatment with IDE improved their blood sugar levels. "These findings demonstrate the feasibility of modulating IDE swamped with more than 7,500 comments and Congress activity as a new therapeutic strategy to treat type 2 diabetes threatened to pass a bill preventing it from implementing any and expand our understanding of the roles of IDE in glucose Part D provision included in the rule. (See BioWorld Today, and hormone regulation," the authors concluded. Their work March 4, 2014, and March 12, 2014.) appeared in the May 22, 2014, issue of Nature. ‘SAMENESS' TOOL NEEDEDThe FDA will accept applications next month for a multiyear grant of up to $1 million to develop a mathematical algorithm or model to use in determining similarity or sameness of biosimilars and generic versions of complex drugs.
In providing a "coherent picture" of the molecular structure of complex macromolecules or drug substances, the project to be funded by the grant could help determine the extent of preclinical and clinical studies that will be needed to develop copies of the drugs.
Initially, "the model will be used to qualitatively and quantitatively determine the degree of sufficiency of in vitro chemical and biological characterization assays, with respect to demonstrating similarity (or sameness) among multiple batches of the reference product," the FDA said.
Details about the project and grant are available on the NIH's grants webpage. // OTHER NEWS TO NOTE Inhibikase Therapeutics Inc., of Atlanta, said it received FDA
orphan drug designation for imatinib to treat progressive
multifocal leukoencephalopathy (PML), a rare side effect of
small-molecule and antibody drugs given to patients with autoimmune diseases such as arthritis and multiple sclerosis and a disease that occurs in 1 percent to 3 percent of clinical AIDS patients. Imatinib, the active ingredient in the firm's lead drug, IkT-001Pro, (and also the active ingredient in Novartis AG's Gleevec), is a host-directed protein kinase inhibitor that disrupts the ability of JC virus, which is activated in cases of PML.
Lanthio Pharma BV, of Groningen, the Netherlands, said it
was awarded an Innovation Credit loan of up to €3.6 million
(US$4.9 million) by Dutch government agency RVO. The loan is risk bearing and will be matched by Lanthio's investors and is expected to cover up to 35 percent of development costs through to completion of phase II trials. It will support the development of LP2, a lanthiopeptide designed to selectively activate the angiotensin type 2 receptor of the renin angiotensin system, for treatment of idiopathic pulmonary fibrosis.
To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY and 2009 and has advised a number of other start-ups. "When I looked at Cour and compared it to other start-ups, they have an enormous amount of data, an impressive patent veteran Catherine Mackey as chairman of its board.
portfolio, and have only taken nondilutive funding," Mackey Founded in 2012, the Elmhurst, Ill.-based Northwestern noted. "They've built a very solid foundation. As a platform, it's University spinout is still preclinical. But it already has two yet- not a one-trick pony." to-be-named big pharma collaborators in place and anticipates Indeed, Cour, which gained its name after company co-founders adding a third by the end of the year, Cour CEO and president, voted over dinner to incorporate the ideas of courage and heart, John Puisis, told BioWorld Today.
may have many tricks ahead. "There are many discoveries yet to Cour's immune-modifying nanoparticles (IMPs), based on work be made," Puisis said. "We're still identifying immune pathways done at the University of Sydney and Northwestern University, we want to explore further, and there are lot of opportunities are derived from biodegradable polymer polylactic-co-glycolic- left to be realized there." // acid (PLGA) and have the potential to address a variety of inflammatory conditions.
OTHER NEWS TO NOTE The negatively charged particles bind to the positively charged Neurotez Inc., of Ridgewater, N.J., said it executed an exclusive
MARCO receptor of monocytes, directing them to the spleen for license deal covering its Leptin derivatives for the treatment destruction through apoptosis, thus diminishing inflammation. of Alzheimer's disease and other cognitive disorders with GCA
Inflammatory monocytes are prevented from entering sites Therapeutics Ltd., of New York. Under the terms, GCA gets
of injury or inflammation, reducing tissue damage and rights to clinically develop and commercialize Leptin products accelerating repair and recovery. in mainland China, exclusive of Hong Kong, and Taiwan, and The company's tolerizing immune-modifying nanoparticles will assume all development and regulatory responsibility. In (TIMPs) also employ PLGA nanoparticles, but load them with exchange, Neurotez will be eligible for gross sales milestones of antigen-specific proteins that induce immune tolerance. In the up to $102.5 million, plus royalties on gross sales. The license case of specially programmed TIMPs, the modified particles also has certain minimum annual sales thresholds following can be used to deliver toleragenic signals to antigen-presenting first commercial sales in mainland China.
cells, resulting in long-term immune tolerance. Nuvilex Inc., of Silver Spring, Md., said it, along with the
At the heart of the technology are proprietary modifications Translational Drug Development (TD2) in Scottsdale, Ariz., to the nanoparticles that determine the immune target and started preparing for a U.S.-based preclinical and clinical mechanism of action, while the particle itself biodegrades and studies on the pain and accumulation of fluid in the abdominal is then cleared by the body.
cavity, two commonly occurring symptoms associated with So far, Cour has filed patents to encapsulate hundreds of advanced pancreatic cancer. TD2 will study the effectiveness of epitopes that modulate cell response. The highly specific TIMPs Nuvilex's pancreatic cancer treatment in relieving the pain and are at the core of its pharma partnerships in type 1 diabetes, fluid accumulation known as ascites. The treatment combines celiac disease and a third yet-to-be-named autoimmune the Cell-in-the-Box live-cell encapsulation technology with disorder. It's diabetes partner has an exclusive option on its cancer agent ifosfamide and will be studied to see if it can investigational TIMP, while its celiac partner has an exclusive improve the quality of life of pancreatic cancer patients. evaluation option.
Preclinical studies are set to commence shortly.
Supported by the stable foundation of its partnerships, Cour Supernus Pharmaceuticals Inc., of Rockville, Md., said
plans to advance its internal IMPs in several cardiovascular United Therapeutics Corp., of Silver Spring, Md., paid the
indications, including acute myocardial infarction, acute firm a $2 million milestone upon United's launch of Orenitram encephalitis syndrome and, once lead optimization is complete, (treprostinil) extended-release tablets for the treatment of ischemic stroke and inflammatory bowel disease. pulmonary arterial hypertension in the U.S. Orenitram uses So far, Puisis said, the company has raised enough seed capital a Supernus technology platform. In addition to the launch to fund its needs. "Our goal is to see how these collaborations milestone, Supernus is eligible for royalties on net sales and play out and determine our capital needs going forward," may be entitled to additional milestones. Orenitram gained he said. As the company further explores applications for its approval late last year. (See BioWorld Today, Dec. 24, 2013.) platform and tackles the strategic thinking necessary to build a stable foundation, it was just the right time to bring Mackey on board, he said.
BIOWORLD™ IS ON TWITTER Mackey, an experienced corporate director, brings 30 years Stay connected—follow us on Twitter! of operating experience in the biopharma and agricultural industries. She served as senior vice president of worldwide R&D and site director for La Jolla Laboratories between 2001 To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY VOLT03 will move to market as a neutraceutical, with quick sales – albeit more modest than a prescription drug – creating a revenue stream to help offset the remainder of the VOLT01 ‘PARTNERING CERTAINLY WOULD BE PREFERRED' clinical development program.
As Voltarra came together, Desai contacted the Menzies team Voltarra also is in the middle of a pre-seed round that will likely to discuss comparative data on VOLT01 and the Novartis drug stay south of $1 million, Becker said, with plans for a larger to determine whether the company's findings were consistent series A on tap for later in the year. Once regulatory filings for with results they had seen in ZAP. Not only were the data VOLT01 are under way, the company will decide whether to in line, but the Menzies researchers approached the four seek a series B.
sponsoring institutions and the country's regulatory agency, the "Partnering certainly would be preferred," Becker said. "I'm Therapeutic Goods Administration, seeking to add a third arm going to need a partner to help from the marketing and sales to the trial, Becker said.
end anyway. If I can find one to help with the clinical end, as The proposal was accepted, and VOLT01 was added to the two- well, that would be ideal, but I'm not counting on that." // year randomized, multicenter, placebo-controlled, double-blind trial, conducted under the auspices of Medicines Australia.
OTHER NEWS TO NOTE "The hopes are, whoever wins – and I'm assuming it's not going Wilex AG, of Munich, Germany, said a collaboration agreement
to be placebo – the compound would be moved forward to the with UCB SA, of Brussels, Belgium, for product candidates
Therapeutic Goods Administration for potential registration" in WX-554 and WX-037 and three preclinical antibody programs KOA, likely in 2015, Becker told BioWorld Today. was terminated by mutual agreement due to an extensive In the meantime, the trial includes a pre-planned six-month restructuring at Wilex, which the company disclosed in January. interim analysis after the study is fully enrolled, which Becker The strategic alliance, inked early in 2009, was worth €10 estimated will occur in the first quarter of 2015. At that point, million up front (US$13.5 million) and a potential €10 million "we are going to know how VOLT01 has performed vs. Aclasta" in two near-term milestones to Wilex, which will return to in terms of safety and efficacy, he said. Though long-term UCB all rights and transfer all intellectual property, data and monitoring will continue in the study, Voltarra plans to package documents generated in connection with the programs. An the early data and request face-to-face meetings with the agreement regarding an antibody program for non-oncology FDA and the EMA to seek guidance on the design of a global indications that was signed last year was terminated. Wilex registration study. will receive a final payment from UCB for R&D expenses, and Provided the company can conduct those meetings by mid- UCB agreed to waive repayment of the €2.5 million shareholder 2015, Becker predicted the phase III registration program could loan granted in December 2010 on completion of the transfer. be completed by 2017, with subsequent regulatory filings Termination of the agreement does not affect UCB's role as a steering the product toward approval in 2018. Wilex shareholder. (See BioWorld Today, Jan. 12, 2009.) In the U.S., Voltarra expects to file its new drug application for VOLT01 under the 505(b)(2) pathway, "which saves a boatload of money, since we don't have to go back and reinvent Amgen, of Thousand Oaks, Calif., said The New England Journal
the wheel," Becker said, noting that the drug's preclinical, of Medicine published results from a phase I study suggesting phase I and tox studies are complete. The company hopes that inhibiting thymic stromal lymphopoietin (TSLP) could to file concurrently with the EMA, using essentially the same benefit the treatment of asthma. TSLP is a cytokine thought data package. The company has filed provisional and non- to be a key driver of allergic inflammation. Results from the provisional patents and expects to hold a broad patent estate 31-patient proof-of-concept study showed treatment for 12 for VOLT01 to prevent and treat osteoarthritis.
weeks with AMG 157, a monoclonal antibody that inhibits the To fund its ambitious program, Voltarra has a complex activity of TSLP, resulted in statistically significant reductions curcumin, dubbed VOLT03, that is sitting "in the parking in early asthmatic responses and late asthmatic responses lot" while other companies conduct proof-of-concept studies in the airways following allergen challenges in patients with with similar compounds across applications in oncology, allergic asthma. The data also showed statistically significant inflammatory indications and neurodegenerative diseases. decreases in baseline markers of inflammation in the airways. Approximately 80 trials with curcumin alone or in combination Overall, adverse events were similar across treatment and with other interventions are under way, according to Cortellis placebo groups, with no serious adverse events. The compound, Clinical Trials Intelligence, with most in phase II or III.
in joint development with Astrazeneca plc, of London, through
"We're just sitting back to see if one of those trials is positive," its biologics arm Medimmune, is in phase II development Becker admitted. "Then, potentially, we can move into that in asthma. The phase I findings also were presented at the space with our product, which is also reformulated to make the American Thoracic Society international conference in San curcumin more soluble." To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY wash), improvements in total mucus weight (the amount of mucus produced by the nose) and also symptom diary score Antibe Therapeutics Inc., of Toronto, said it submitted a
compared to placebo. The results are being presented during clinical trial application to Health Canada for lead drug ATB- a poster discussion session at the American Thoracic Society 346, proposing a phase I study to assess the safety, tolerability conference in San Diego.
and pharmacokinetics of single and multiple-ascending doses Intelgenx Corp., of Saint Laurent, Quebec, and Redhill
in healthy subjects. The trial is slated to start this summer. ATB- Biopharma Ltd., of Tel Aviv, Israel, reported data from a
346 is a hydrogen sulfide-releasing derivative of naproxen.
comparative bioavailability study with migraine candidate Atyr Pharma Inc., of San Diego, said it completed a phase I
Versafilm, an oral thin film formulation of rizatriptan for acute study for its lead program, Resokine IV, which marked the first migraines. Results of the study are subject to final quality ever administration of a physiocrine to humans. The double- assurance and an independent study report by the Canadian blinded, placebo-controlled trial was conducted in healthy clinical research organization that conducted the trial, but the male and female subjects in the European Union to assess companies said the data will support the planned submission the safety and tolerability of Resokine IV, in development in Europe for the product during the third quarter. as a potential therapeutic for rare immune disorders. The Nymox Pharmaceutical Corp., of Hasbrouck Heights,
company said the data from 32 healthy subjects in four N.J., reported results from the Brief Male Sexual Function dose cohorts support the basic premise of Resokine IV as Questionnaire in its recently completed NX03-0040 study of an immunomodulator rather than a classic interleukin or NX-1207 in low-grade localized prostate cancer, indicating that interferon with systemic immunostimulating properties upon targeted treatment with the drug at either dose – 2.5 mg or administration. Among their various homeostatic functions, 15 mg – had no significant effect on reported sexual function some physiocrines act as extracellular signaling molecules score post-treatment. Nymox also is testing NX-1027 in benign to orchestrate immunohomeostasis in response to stress and prostatic hyperplasia and recently completed the second other physiological changes. Physiocrines comprise naturally pivotal trial in that indication.
occurring proteins derived from tRNA synthetases that play Summit plc, of Oxford, UK, said SMT C1100, an oral small-
fundamental roles in the function of human physiology and molecule utrophin modulator for the treatment of Duchenne restoring pathophysiological states to a healthier state. muscular dystrophy (DMD), successfully met its primary Celsion Corp., of Lawrenceville, N.J., said the FDA has green-
endpoint of safety and tolerability in a phase Ib trial in patients lighted its planned pivotal phase III trial of Thermodox, its with the disease. The dose-escalating trial was conducted in 12 heat-activated liposomal encapsulation of doxorubicin, in patients with DMD, between 5 and 11 years old. The nonplacebo- combination with radio frequency ablation (RFA) in primary controlled study also measured levels of creatine kinase (CK), an liver cancer, or hepatocellular carcinoma. The trial will enroll enzyme associated with muscle fiber damage that is elevated in 550 patients globally and evaluate Thermodox in combination boys with DMD, and results found that In the majority of patients with RFA vs. standardized RFA alone. The primary endpoint there was a reduction in CK levels during dosing with SMT C1100. for the trial is overall survival. An independent data monitoring Those data are consistent with nonclinical in vivo efficacy studies committee will conduct two interim analyses. Celsion said it in the mdx model of DMD that showed SMT C1100 reduced CK expects to launch the study before the end of June.
levels after only 15 days. The company plans to review those Gilead Sciences Inc., of Foster City, Calif., reported results from preliminary data, which are expected to lead to a revision of
a placebo-controlled, phase IIa challenge study of GS-5806,
future clinical trial plans in order to determine the optimal way, an investigational oral RSV fusion inhibitor, in healthy adult either through dietary means or drug formulation changes, to patients intranasally infected with respiratory syncytial virus address the drug uptake differences between DMD patients and (RSV). The trial achieved its primary and secondary endpoints healthy volunteers. The next patient study is now expected to of lower viral load (the amount of virus detected in the nasal start in the fourth quarter. IS YOUR COMPANY FEATURED IN THIS ISSUE? Promote it on your website or in your investor kit! For high-quality reprints of articles about your company, please contact an Account Manager at (770) 810-3144.
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Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at THURSDAY, MAY 22, 2014 BIOWORLD™ TODAY PHARMA: OTHER NEWS TO NOTE Netherlands, Sweden, Norway, Denmark, Ireland and Italy.
Daiichi Sankyo Co. Ltd., of Tokyo, began a three-year research
Abbvie Inc., of Chicago, said the FDA granted Humira
project with Sanford-Burnham Medical Research Institute, of La (adalimumab) orphan drug designation for the treatment of Jolla, Calif., to investigate cardiovascular-metabolic diseases. non-infectious intermediate, posterior or pan-uveitis, or chronic Terms of the agreement, which will deploy Sanford's screening non-infectious anterior uveitis, a group of rare but serious capabilities, were not disclosed.
inflammatory diseases of the eye. Abbvie is investigating Novo Nordisk A/S, of Bagsvaerd, Denmark, said the FDA
the efficacy and safety of Humira for the treatment of non- tentatively scheduled an advisory committee meeting for Sept. infectious uveitis, and the clinical program is in phase III 11 to discuss the new drug application (NDA) for liraglutide 3 mg for the treatment of obesity. The NDA was submitted to the Baxter International Inc., of Deerfield, Ill., said the FDA
FDA last December.
extended the PDUFA date for its review of the biologics PHARMA: IN THE CLINIC license application for Hyqvia (immune globulin infusion 10 percent [human] with recombinant human hyaluronidase), the Boehringer Ingelheim GmbH, of Boehringer, Germany,
company's investigational subcutaneous treatment for patients presented results from the phase III VIVACITO study at the with primary immunodeficiency. The FDA is requiring additional American Thoracic Society meeting in San Diego, showing time to review supplemental data that Baxter provided as part that the fixed-dose combination of tiotropium and olodaterol, of the ongoing process for approval. The PDUFA date has been delivered via the Respimat inhaler, demonstrated a statistically extended by three months, which is the standard extension significant improvement over both monotherapy treatment (p period. As part of the FDA's extended review, Baxter now < 0.0001) and placebo (p < 0.0001) in the primary endpoint, expects to participate in a Blood Products Advisory Committee defined as the forced expiratory volume in one second. meeting, which the agency has scheduled for July 31. Hyqvia Secondary endpoints included additional tests measuring was approved by the European Commission in 2013 and is breathing over 24 hours. The overall incidence of adverse available in several European countries, including Germany, the events was comparable among treatment groups.
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