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  • 1. BIOTECH’S MOST RESPECTED NEWS SOURCE FOR 20 YEARS FRIDAY VOLUME 21, NO. 98 MAY 21, 2010 PAGE 1 OF 7 Synthetic, but “not from Scratch” Stock Plunges Success Reported in Booting Santhera’s Idebenone Falls Up Cell Via Artificial Genome Short in Second Pivotal Trial By Anette Breindl By Cormac Sheridan Science Editor BioWorld Today Correspondent For 15 years, scientists at the J. Craig Venter Institute Santhera Pharmaceutical Holdings AG has reached have been pursuing the goal of controlling a real cell with what CEO Klaus Schollmeier called a “dead-end street” in its a synthetic genome. And in the May 21 , 2010, edition of Sci- Friedreich’s ataxia (FRDA) program, following the second ence, they reported success. Phase III trial failure of idebenone (Catena, Sovrima). “This is the first self-replicating species we have on Twelve months after the failure of the drug to reach this planet whose parent is a computer,” J. Craig Venter statistical significance on a six-month Phase III trial in the told reporters at a press conference announcing the U.S., the compound suffered what looks like an even more findings, adding that although the synthetic cell’s cyto- comprehensive setback in a European Phase III trial in plasm was contributed by a regular bacterium, “we call it FRDA, a progressive neuromuscular degenerative condi- synthetic because the cell is totally derived from a syn- tion characterized by loss of muscle control and heart thetic chromosome, made with four bottles of chemicals enlargement. on a chemical synthesizer, starting with information in a Investors reacted accordingly. computer.” The stock (ZURICH:SANN) closed Thursday at CHF14. 10 See Genome, Page 3 See Santhera, Page 4 Collins: Funding NIH Paves Way Financings Roundup for Biotech Drug Development NeuroTherapeutics Pharma Gets By Donna Young $43M Series B for Epilepsy, Pain Washington Editor By Trista Morrison WASHINGTON – While the needle-in-the-haystack work Staff Writer done in the pursuit of the 1989 discovery of the gene Despite the difficult venture capital markets, Neu- responsible for cystic fibrosis laid the groundwork for the roTherapeutics Pharma Inc. (NTP) raised a whopping $43 Human Genome Project, the research and development million in Series B financing to advance its epilepsy and efforts that followed to find treatments has created a pain compounds into clinical trials. roadmap that is paving the way for others to follow, the The round ranks among the biggest biotech venture heads of the National Institutes of Health (NIH) and Cystic raises so far this year, and it is particularly impressive Fibrosis Foundation (CFF) said Thursday during a Capitol given that NTP is not yet in the clinic. Brian Williams, vice Hill briefing. president of corporate development and finance for The work of the NIH and groups like CFF is derisking Chicago-based NTP, said his experience during the drug development for biotechs and pharmas, and there- fundraising was that many venture investors are gravitat- fore, must continue to be adequately funded, said NIH ing toward low-priced, later-stage assets, creating intense Director Francis Collins, who co-discovered the cystic competition for scarce early stage funding. fibrosis gene, known as CFTR, a ATP-binding cassette Even so, NTP managed to attract new investors Fidelity See NIH, Page 5 See Financings Roundup, Page 6 INSIDE: OTHER NEWS TO NOTE: NICOX, QLT, QUINTILES ........................................4 CLINIC ROUNDUP: ANTIGENICS, GENENTECH, INSPIRE, JENNEREX ................7 To subscribe, please call BIOWORLD® Customer Service at (800) 888-3912; outside the U.S. and Canada, call (404) 262-5547. Copyright © 2010 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 2. FRIDAY, MAY 21, 2010 BIOWORLD® TODAY PAGE 2 OF 7 O T H E R N E W S T O N O T E Biosimilars and Health Care Legislation: New Opportunities The recent health care legislation contains provi- • A.P. Pharma Inc., of Redwood City, Calif., said that it sions that will provide an eventual pathway for FDA- has been notified by Nasdaq that the company’s minimum approval of generic biologics. But many questions closing bid price had fallen below the required minimum $1 remain, from access by a generics company to the bio- for 30 consecutive trading days. The company has until logics formula to marketing dynamics that will shape the Nov. 15 to regain compliance. competitive landscape for follow-on biologics. • Ablynx NV, of Ghent, Belgium, said a research col- In this all new BioWorld Today audio conference Ed laboration, which forms part of its license agreement for Korwek and Paul Wotton will provide insight and under- Nanobodies to tumor necrosis factor alpha (TNF-alpha) standing of how the recent health care legislation will with Pfizer Inc., of New York, has been extended for affect the pathway for approved biosimilars, including a another year. Details of the extension were not disclosed. detailed explanation of the changes that must be made The original deal, signed in November 2006 with Wyeth and how soon will this impact your business and regula- Pharmaceuticals (now part of Pfizer) had a potential value tory activity. of $212.5 million plus royalties on product sales. Scheduled for Tuesday, May 25, 2010, at 1-2:30 p.m. • BioSante Pharmaceuticals Inc., of Lincolnshire, Ill., EST, registration is $325. Call 1-800-688-2421 to register! said that results of an evaluation of its 2A/Furin technology Mention conference code T10617. by Novartis Pharma AG, of Basel, Switzerland, in its indus- trial antibody CHO expression system were published in the Journal of Applied Microbiology & Biotechnology. The results demonstrated that the 2A/Furin technology was integrated successfully into a plasmid-based, industrial-scale, CHO Stock Movers antibody cell line development process, with minimal opti- 05/20/10 mization required. Use of 2A/Furin could enhance further the distribution of high-expressing clones and improve the average productivity of clones. Company Stock Change • Critical Outcome Technologies Inc., of London, NASDAQ Biotechnology -4. 1% Ontario, reported positive results from the first phase of its HIV integrase inhibitor discovery program. The results pro- Amgen Inc. -4.2% vided validation of the CHEMSAS drug discovery technol- Biodel Inc. -1 1.2% ogy, it said. The company has used its technology to dis- Celldex Therapeutics Inc. -14% cover several small-molecule scaffolds that have an entirely Dynavax Technologies Corp. -1 1.5% new binding mode and interaction with the active site of the viral enzyme. The company has filed composition-of-matter Inhibitex Inc. -16.3% patents and intends to proceed with the next phase of the (Biotechs showing significant stock changes Thursday) project that consists of optimizing a small series of poten- tial lead candidates based on the scaffolds. SUBSCRIBER INFORMATION BioWorld® Today (ISSN# 1541-0595) is published every business day by AHC Media LLC, 3525 Piedmont Road, Building Six, Suite 400, Atlanta, GA 30305 U.S.A. Opinions expressed are not necessarily those Please call (800) 888-3912 to subscribe of this publication. Mention of products or services does not constitute endorsement. BioWorld® and or if you have fax transmission problems. BioWorld® Today are trademarks of AHC Media LLC, a Thompson Publishing Group company. Copyright Outside U.S. and Canada, call (404) © 2010 AHC Media LLC. All Rights Reserved. No part of this publication may be reproduced without the 262-5476. Our customer service hours written consent of AHC Media LLC. (GST Registration Number R128870672). are 8:30 a.m. to 6:00 p.m. EST. Glen Harris, (404) 262-5408 ATLANTA NEWSROOM: Managing Editor: Glen Harris. Assistant Managing Editor: Jennifer Boggs. Jennifer Boggs, (404) 262-5427 Senior Staff Writer: Karen Pihl-Carey. Senior Production Editor: Ann Duncan. Anette Breindl, (518) 595-4041 Staff Writer: Randy Osborne. Trista Morrison, (858) 901-4785 WASHINGTON BUREAU: Washington Editor: Donna Young. Staff Writer: Catherine Hollingsworth. Donna Young, (202) 739-9556 WEST COAST BUREAU: Staff Writer: Trista Morrison. Catherine Hollingsworth, (301) 576-0667 EAST COAST BUREAU: Science Editor: Anette Breindl. Senior Vice President/Group Publisher: BUSINESS OFFICE: Senior Vice President/Group Publisher: Donald R. Johnston. Donald R. Johnston, (404) 262-5439 Director of Product Management: Jane Cazzorla. Internet: http://www.bioworld.com Marketing Coordinator: Sonia Blanco. Account Representatives: Bob Sobel, Chris Wiley, Scott Robinson. DISPLAY ADVERTISING: For ad rates and information, please call Stephen Vance at (404) 262-5511 or email him at stephen.vance@ahcmedia.com. REPRINTS: For photocopy rights or reprints, call our reprints department at (404) 262-5547. PRESS MATERIALS: Send all press releases and related information to newsdesk@bioworld.com.
  • 3. FRIDAY, MAY 21, 2010 BIOWORLD® TODAY PAGE 3 OF 7 Genome Indeed, Church noted that “printing out a copy of an Continued from page 1 ancient text isn’t the same as understanding the language.” In 2007, Venter and his team had transplanted a natural But the philosophical and ethical implications of the M. mycoides genome into the cytoplasm of a close relative, work are nevertheless likely to be enormous. Mycoplasma capricolum. In 2008, they reported synthesiz- Philosophically, bioethicist Arthur Caplan commented ing the genome of another related bacterium, Mycoplasma that “the demonstration that life can be created from non- genitalium. (See BioWorld Today, June 29, 2007, and Jan. 25, living parts, albeit those harvested from a cell,” puts an end 2008.) to the long-held philosophical doctrine that life cannot be The new work combines both feats: Venter and his explained mechanistically, known as vitalism. team first synthesized the genome of M. mycoides, deleting But others dispute that, at least for the time being, not- 14 genes and adding what they termed “watermarks” to be ing that the cytoplasm used to start the cell line came from able to distinguish their creation from a natural genome. a regular, living bacterium. Venter himself said that he did They then transplanted the genome into the cytoplasm of not consider the synthetic cell to be “life created from M. capricolum. The rebooted cell and its descendants were scratch,” but did consider it “synthetic life.” phenotypically pure M. mycoides. Venter´s team intends to use the booting-up technique Venter said that the work’s biggest stumbling blocks to make many different synthetic chromosomes and see- were not the ones his team expected when they started the ing which ones can boot up cytoplasms, thus defining the work. “When we started this out, we thought the synthesis “minimal genome” that can support life. would be the biggest problem,” he told reporters. The work also allows for the mixing of genes from dif- But as technology progressed, synthesizing and ferent species on a much greater scale than current tech- assembling long stretches of DNA proved to be fairly nologies. Both endeavors are certain to influence how life is straightforward – occasional glitches, such as a single base viewed, and perhaps defined. pair error that thwarted the team for several months before Solving the world´s energy and pollution problems are it was detected, notwithstanding. not the only possible use of such novel organisms, of But booting up another cell’s cytoplasm after trans- course, and first author Daniel Gibson acknowledged that planting an artificially created genome proved to be a the technology could be used to create bioweapons, much greater challenge than anticipated. When the team though he also said that “It’s hard today to find a technol- succeeded in synthesizing the entire genome of M. ogy that’s not dual-use.” mycoides in 2008, they more or less assumed the hard part According to the J. Craig Venter Institute, the work has was over. Venter told reporters at the 2008 press confer- been reviewed by “White House offices including the Office ence announcing the synthesis that he would be “disap- of Homeland Security and Office of Science and Technology pointed” if they did not manage to boot up a cytoplasm Policy, the National Science Advisory Board for Biosecurity, using the synthetic genome within the same year. (See . . . the Department of Energy, the National Institutes of BioWorld Today, Jan. 25, 2008.) Health and others,” as well as by an independent bioethics At Thursday’s press conference, Venter acknowledged panel for more than a decade. that more than two years later, for reasons that are still In the end, Gibson said, his team believes the benefits of unknown, “we have not yet succeeded in booting up [the M. the technology outweigh the risks: Its availability represents genitalium] chromosome.” The team found success only a “linear increase” in the ability to do harm, but an “exponen- when it switched from working with the genome of M. gen- tial increase” in the ability to do good, they wrote. ■ italium, which they had picked for its small size, to that of M. mycoides, which, with roughly 1 million base pairs, is twice as big. If the cytoplasm that started the synthetic cell was not O T H E R N E W S T O N O T E synthetic, neither are the DNA sequences are made from scratch: Harvard geneticist George Church noted, in a com- • Grifols SA, of Barcelona, Spain, said that it has mentary in Nature, that watermarks aside, “the semi-syn- reached an agreement with Pharmalink AB, of Stockholm, thetic mycobacterium is not changed from the wild state in Sweden, to acquire various forms of intellectual property any fundamental sense.” associated with the treatment of post-polio syndrome And so, though Venter and his team hope that they can (PPS). The acquisition is expected to be finalized in the next ultimately create bacteria to address energy and environ- few weeks and will include documentation, know-how and mental challenges and help in vaccine design, on the most Swedish regulatory approvals under the trade name Xepol. practical level, so far the resulting organism is nothing The acquisition also includes U.S., European and Japanese more than what they could have picked up faster and patents, which will effectively give Grifols exclusive rights cheaper from goats, in which M. mycoides resides as a com- to the treatment method. Currently, there are no therapies mensal species. approved for the treatment of PPS. To subscribe, please call BIOWORLD® Customer Service at (800) 888-3912; outside the U.S. and Canada, call (404) 262-5547. Copyright © 2010 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 4. FRIDAY, MAY 21, 2010 BIOWORLD® TODAY PAGE 4 OF 7 Santhera Idebenone is undergoing clinical trials in several other Continued from page 1 indications, data from which should become available in (US$12.24), down 43 percent on Wednesday’s close of the coming weeks and months. First up is a Phase II trial in CHF24.75. Leber’s hereditary optic neuropathy, followed by a Phase II In the previous U.S. study, called IONIA (Idebenone trial in Melas syndrome. Interim data from a Phase III trial in effects On Neurological ICARS Assessments), patients who Duchenne’s muscular dystrophy is expected before year- received the drug did show a consistent improvement on end. the primary neurological endpoint, based on a change from The company exited 2009 with CHF53.3 million in cash baseline in the International Cooperative Ataxia Rating and is funded beyond 201 1. It has two other clinical-stage Scale (ICARS), and on a secondary neurological endpoint, projects at present. Fipamezole, which is partnered in the Friedreich’s Ataxia Rating Scale. North America with Mississauga, Ontario-based Biovail However, an unexpectedly high placebo response elim- Corp., will shortly enter a Phase III trial for treatment of inated any possibility of the study attaining statistical sig- dyskinesia associated with levodopa therapy in Parkinson’s nificance. (See BioWorld Today, May 20, 2009.) disease. Omigapil will shortly enter a Phase II/III trial in con- This time around, the picture is even more gloomy. genital muscular dystrophy. “There’s no placebo effect. We simply saw no difference,” Santhera’s Phase III miss is the second major clinical Schollmeier told BioWorld Today. “In this trial, basically disappointment for Europe’s drug developers this month. everything was flat.” Newron Pharmaceuticals SpA, of Milan, Italy, previously The Liestal, Switzerland-based firm had several rea- reported the failure of ralfinamide in a Phase IIb/III trial in sons to be more optimistic about a positive outcome to its 41 1 patients with neuropathic lower back pain. That drug European study, called MICONOS (Mitochondrial Protec- also failed to demonstrate any significant difference with tion with Idebenone In Cardiac Or Neurological Out- respect to placebo. ■ come Study). At 12 months duration, the trial, which had three drug treatment arms as well as a placebo arm, was twice as long as the Ionia trial. It also recruited more patients – 232 vs. O T H E R N E W S T O N O T E 70. Moreover, those who were included were older and had more severe disease than those in the U.S. study. • NicOx SA, of Sophia Antipolis, France, said that it has As well as the ICARS-based primary endpoint and a sec- decided to delay initiation of an in-house Phase II study of ondary endpoint based on the Friedreich’s Ataxia Rating NCX 6560, until it receives clarification of naproxcinod’s Scale, the study also included a responder analysis, based regulatory position. To conserve cash, the company said it on measuring the proportion of patients improving their plans to seek alternative funding options to develop NCX ICARS scores, and a set of cardiac endpoints. 6560, which achieved positive results in a first-in-man clin- Although the cardiac analysis is ongoing, there was, ical study in 2009. The company will continue to seek dis- the company said, no difference between drug and placebo cussions with the FDA, following a May 12 FDA advisory on the key cardiological secondary endpoint, based on a panel that rejected naproxcinod and called for additional combination of anatomical and functional parameters. outcome studies. The European Medicines Agency also is A meta-analysis, based on using data from both Phase reviewing the marketing authorization application for III studies, plus a previous Phase II trial called Nicosia naproxcinod. (NIH Collaboration With Santhera In Ataxia), did show posi- • QLT Inc., of Vancouver, British Columbia, said its tive and statistically significant trends in patients in the board has approved an increase in the number of common combined mid- and high-dose group and those in the high- shares that it may purchase over a 12-month period that dose group only. began Nov. 3, 2009, from 2,731 ,534 common shares, rep- However, the outcome is confusing, particularly when resenting 5 percent of the outstanding shares to 4,700,060 a Phase II trial delivered a positive result. shares, or 10 percent of the public float. The price will be The company has one final shot at getting more clarity. the market price of the shares at the time of acquisition. Data from extension studies will become available over the • Quintiles Transnational Corp., of Research Trian- summer. “We will complete the extension studies. To get a gle Park, N.C., and Nycomed GmbH, of Zurich, Switzer- hold of the longitudinal data of up to three years might be land, have entered an agreement under which the privately helpful,” Schollmeier said. However, an additional clinical held Swiss firm has transferred a UK-based Healthcare trial “is not an option,” he said. Solutions Team to Quintiles. The transfer follows The drug is already approved in Canada, where the Nycomed’s decision to out-license a product to another response from patients and physicians has been “over- pharmaceutical company in the UK. After the team’s trans- whelmingly positive,” he said. It is also available in a small fer to Quintiles, it was contracted back to Nycomed to com- number of European countries on a named-patient basis. plete the work. To subscribe, please call BIOWORLD® Customer Service at (800) 888-3912; outside the U.S. and Canada, call (404) 262-5547. Copyright © 2010 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 5. FRIDAY, MAY 21, 2010 BIOWORLD® TODAY PAGE 5 OF 7 NIH “They can really look at the novel high-risk areas of Continued from page 1 medical research to really effectively move forward solu- transporter protein. tions,” Anderson said. The international effort to understand the molecular The “great progress” being made in cystic fibrosis cause of cystic fibrosis has yielded not just drugs to treat drug R&D, stands as a “proof of principle of what we the symptoms of the disease but investigational com- ought to be able to try to do for many other diseases,” pounds aimed at attacking the basic defect involved, said Collins said. CFF CEO Robert Beall. But, he said, the biomedical community at large is grap- “This clearly has changed this disease from one of pling with how it can extrapolate the success of the cystic hopelessness and despair, to one of really hope and opti- fibrosis story to “paint an even broader picture in a part- mism and excitement amongst the entire CF community, nership with the private sector in a new paradigm of how patients, parents and caregivers alike,” he said. we are going to get to those much hoped for interventions Beall noted that there currently are more than 30 ther- that will do something about diseases for which we cur- apies under investigation to treat cystic fibrosis, including rently have inadequate therapeutics.” four Phase III products – two CFTR modulators and two Collins noted that the NIH’s mission is not only to products aimed at restoring airway surface liquid. Those pursue the fundamental knowledge on which to build drugs have been funded in large part by CFF's therapeutics therapeutic ideas, but also to apply that knowledge to discovery program, which has invested about $660 million extend healthy life and reduce the burdens of illness and in drug discovery since its founding. disability. He said that CFF, which generally spends about $70 He said that the NIH’s “profound impact” on the health million per year on drug development, took a gamble and of the nations has all too often been taken for granted, put $40 million into one discovery project. likely “because these are changes that have happened That gamble paid off with two viable candidates, Beall somewhat gradually.” explained. But the success of the Human Genome Project and the “We felt the biggest risk was not to take this opportu- tools it has provided has “rocketed upward” the scientific nity,” he insisted. discoveries that are leading to new therapies and cures, Under an agreement with CFF, Cambridge, Mass.-based Collins said. Vertex Pharmaceuticals Inc. is now developing those com- The discovery of gene variations involved in the causes pounds, known as VX-770 and VX-809, both CFTR modula- of rare diseases has gone from “very few” to about 2,500, tors, Beall said. and for common diseases, there have been almost 1 ,000 The so-called potentiator drugs are designed to correct DNA-level contributing factors identified, he noted. the function of the defective CFTR protein allowing chlo- “Talk about a plethora of target opportunities,” Collins ride and sodium to move properly in and out of cells lining said. “There’s never been anything like this.” the lungs and other organs. Collins said his goal at the NIH is to “take the hope that Phase II results of VX-770 demonstrated that the com- is attached to the promise of all of these developments and pound in patients with at least one copy of the G551D gene turn that into real results.” mutation had improvements in biological measures of But getting across the bridge of translating that basic CFTR function, or nasal potential difference and sweat chlo- science into therapies will take an integrated partnership ride, and forced expiratory volume. of government, nonprofits and the private sector and con- Vertex initiated two Phase III studies of VX-770 last sistent funding from Congress, he said. year, Beall said. (See BioWorld Today, May 28, 2009.) Collins noted that one major source of funding the NIH Thanks to CFF’s aggressive investment in innovative is awaiting is from the health reform provision that created research and comprehensive care, the median survival age the Cures Acceleration Network (CAN), which authorized for patients with cystic fibrosis has risen from about 5 $500 million aimed at cutting the time between discovery years to 37, said Margaret Anderson, executive director of and development of drugs and therapies through new the nonprofit FasterCures, who noted that CFF pioneered grant-making mechanisms at the agency. the concept of venture philanthropy. The CAN provision gives Collins “DARPA-like” flexible Because of their close relationships with the patient authorities to manage projects, in which he could draw on communities, groups like CFF “have very unique ability to resources more quickly to be able to continue a project or move very quickly to address emerging translational and kill failing projects “instead of continuing to pour money clinical opportunities, and they have the capacity to lever- into them, which is critical,” Collins said. age public investment to catalyze and jumpstart innova- But, he lamented, Congress has yet to appropriate the tion,” Anderson insisted. funds. Patient advocacy groups also can serve as reliable “Now it’s up to appropriators to decide whether in FY 1 1 sources of funding, she added. they will have the funds to support it,” Collins said. ■ To subscribe, please call BIOWORLD® Customer Service at (800) 888-3912; outside the U.S. and Canada, call (404) 262-5547. Copyright © 2010 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 6. FRIDAY, MAY 21, 2010 BIOWORLD® TODAY PAGE 6 OF 7 Financings Roundup linked to vision loss, and gabapentin can cause dizziness Continued from page 1 and sleepiness. Biosciences, MPM Capital, SR One (GlaxoSmithKline plc’s “Coming in with a completely novel mechanism offers venture arm) and Pfizer Inc. Existing investors Novo Ven- them hope, because you’re going about the problem in a tures and Thomas McNerney & Partners, who put $5 million different manner,” Williams added. into the company’s Series A round back in 2006, also par- NTP also is focusing on neuropathic and acute pain, as ticipated. Williams said the syndicate provides “the best of well as other CNS disorders. both worlds,” referring to the mix of traditional and corpo- The firm is aiming to file an investigational new drug rate investors, yet he noted that NTP did not give up any application by the end of the year and get into the clinic future rights or options to its products. early next year. With lean, virtual operations and no wet What brought so many new investors on board? labs, the Series B funding is expected to carry NTP-2014 Williams credited a “completely unique” mechanism of through Phase II trials in multiple indications as well as sup- action for treating central nervous system disorders that port the development of additional compounds. has produced encouraging preclinical safety and efficacy In other financing news: data, even outperforming blockbuster comparators like • MethylGene Inc., of Montreal, completed its previ- gabapentin (now generic, but originally Pfizer Inc.’s Neu- ously announced C$9. 1 million (US$8.9 million) nondilutive rontin). financing through which it conducted a court-supervised NTP was formed in 2006 by a group of angel investors corporate reorganization, transferring all of its assets and and academic researchers. The company secured its Series liabilities to a newly incorporated firm with the same name. A financing and operated virtually until 2008, when the MethylGene’s work on its cancer pipeline will continue as venture backers decided it was time to ramp up the man- before. agement team. They hired Williams, President and CEO • NeoStem Inc., of New York, has converted Stephen Collins, Vice President of Research and Develop- redeemable Series C preferred stock by RimAsia Capital ment Stephen Wanaski, and Vice President of Operations Partners LP. That means the company will no longer be Holli Carlson. All four hailed from Ovation Pharmaceuticals required to pay the annual dividend and expects to recog- Inc., which marketed several CNS drugs and boasted two nize cash savings of $408,875 per year. ■ Phase III programs for epilepsy. Ovation was later acquired for $900 million by H. Lund- beck AS, which subsequently gained approval of epilepsy O T H E R N E W S T O N O T E drug Sabril (vigabatrin), an irreversible inhibitor of gamma- aminobutyric acid transaminase. (See BioWorld Today, Feb. 10, 2009, and Aug. 24, 2009.) • To-BBB Technologies BV, of Leiden, the Netherlands, When the Ovation team came on board, NTP was is entering into a pilot study with Janssen Pharmaceutica focused on one novel CNS target. Yet Williams noted that as NV, of Beerse, Belgium, a subsidiary of Johnson & Johnson, to optimization increased efficacy, it was actually taking them enhance delivery of drugs to the brain for central nervous further and further from their intended target. NTP was system diseases. Drug development for CNS disorders is able to identify the new target and mechanism of action hampered by the blood-brain barrier, which prevents the causing the efficacy and reposition its efforts. delivery of many drug candidates to disease targets in the The company is keeping the details of its approach brain, and to-BBB said its G-Technology is a safe technology confidential for now, but Williams said lead compound NTP- for drug delivery to the brain. Proof of concept with the G- 2014 is an oral, small molecule that works through a known Technology is demonstrated in several disease models, system to decrease the excessive firing in the brain associ- including pain, brain tumors and viral encephalitis. ated with a variety of CNS disorders. And while other drugs can decrease firing too much, resulting in sedation or even cognitive decline, NTP-2014 works in a localized portion of the brain, only during times of excessive neuronal dis- charge. ADVERTISE HERE The initial indication for the drug is epilepsy, which ...and reach high-level Williams noted affects 1 percent of the population, with biotechnology professionals every day! more new cases diagnosed each year than multiple sclero- sis, cerebral palsy, muscular dystrophy and Parkinson’s dis- For advertising opportunities in ease combined. Yet Williams said a “significant” number of BioWorld Today, please contact patients are refractory or unresponsive to the available Stephen Vance at (404) 262-5511 treatments, and even those who do respond are saddled or stephen.vance@ahcmedia.com with side effects. Lundbeck’s Sabril, for example, has been To subscribe, please call BIOWORLD® Customer Service at (800) 888-3912; outside the U.S. and Canada, call (404) 262-5547. Copyright © 2010 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 7. FRIDAY, MAY 21, 2010 BIOWORLD® TODAY PAGE 7 OF 7 the intravenous administration of JX-594 to patients with metastatic cancer. The data demonstrated clear dose- C L I N I C RO U N D U P related delivery to solid tumors, cancer-targeted replica- tion and gene expression, and anticancer effects of JX-594 • Antigenics Inc., of Lexington, Mass., said that data delivered via I.V. Necrotic responses were more commonly from a multicenter Phase I/II trial of Oncophage (vitespen) observed in patients treated at higher doses. In addition, for recurrent high-grade glioma was presented at the Inter- intravenous infusion was safe and well-tolerated. Those national Conference on Brain Tumor Research and Therapy. data were presented at the American Society of Gene and Data from 32 evaluable patients suggested that vaccina- Cell Therapy annual meeting in Washington. tion with Oncophage may improve overall survival in • VIA Pharmaceuticals Inc., of San Francisco, and patients with recurrent high-grade glioma. An overall the Montreal Heart Institute announced the publication of median survival of 44 weeks after tumor resection was clinical trial data from a study of VIA-2291 , a 5-Lipoxyge- observed. Approximately 70 percent of the evaluable nase inhibitor in the May 19, 2010, issue of Circulation: Car- patients survived beyond 36 weeks, and 41 percent sur- diovascular Imaging. Those newly published data strongly vived up to or longer than one year. Oncophage was well supported the evaluation of VIA-2291 in larger outcome tri- tolerated, with no serious adverse events attributable to als. The study met its primary endpoint by demonstrating a the vaccine. statistically significant, dose-dependent inhibition of • Genentech Inc., of South San Francisco, and Biogen whole blood stimulated leukotriene LTB4 production at 12 Idec Inc., of Cambridge, Mass., said data from the Phase III weeks (P < 0.0001) demonstrating greater than 80 percent PRIMA study showed that continuing Rituxan (rituximab) inhibition in 90 percent of patients. for two years in patients who responded to initial treat- ment with Rituxan plus chemotherapy, doubled the likeli- hood of progression-free survival compared to those who stopped treatment (based on a hazard ratio of 0.50, 95 per- cent CI, 0.39; 0.64; p =/< 0.0001). The study enrolled patients with previously untreated advanced follicular lym- phoma. After two-years of follow-up, 82 percent of patients who received Rituxan maintenance were in remission com- pared to 66 percent of patients who did not. No new safety signals were observed. More detailed results will be pre- OBESITY sented at the American Society of Clinical Oncology annual meeting in June. Rituxan currently is approved in non- Hodgkin’s lymphoma, chronic lymphocytic leukemia and REPORT rheumatoid arthritis. • Genetix Pharmaceuticals Inc., of Cambridge, Mass., and the National Institute of Health and Medical Research in France presented additional data from a pilot study of Lenti-D treating X-linked adrenoleukodystrophy (ALD). Data showed continued stable expression of ALD protein and disease stabilization at three years in two ALD This brand new report from BioWorld Today, and patients. Data on a third were insufficient to determine dis- co-published with Medical Device Daily, shows how ease stabilization. Genetix plans to initiate larger clinical studies in ALD in both the U.S. and Europe in 201 1. a burgeoning disease, a strong public awareness, • Inspire Pharmaceuticals Inc., of Durham, N.C., committed government endeavors and a lack said that its scientists and collaborators presented data of new drug and device approvals have created the on denufosol tetrasodium, an investigational therapy for perfect “unmet need” recipe for opportunity. cystic fibrosis, during an oral presentation at the Ameri- can Thoracic Society 2010 International Conference in Order this new Call New Orleans. The data suggested that denufosol, an inhaled ion channel regulator, has properties that allow it report today from 1-800-688-2421 or 1-404-262-5476 to reach and improve lung function in the small airways, BioWorld— it’s just $299 plus Visit which may support its potential as an early intervention www.bioworld.com/bioobesity therapy. $17.95 in S&H. • Jennerex Inc., of San Francisco, reported positive results from its Phase I dose-escalation study evaluating To subscribe, please call BIOWORLD® Customer Service at (800) 888-3912; outside the U.S. and Canada, call (404) 262-5547. Copyright © 2010 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com

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