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Slide 1: BIOTECH’S MOST RESPECTED NEWS SOURCE FOR OVER 17 YEARS WEDNESDAY SEPTEMBER 2, 2009 VOLUME 20, NO. 169 PAGE 1 OF 7 Celgene Likes PTC’s GEMS, Too PTC Gets $12M Up Front in Roche Deal Worth up to $1.9B By Trista Morrison Staff Writer PTC Therapeutics Inc. is expected to announce two drug discovery deals on Wednesday, a potential $1.9 billion whopper with Roche AG and an option exercise by Celgene Corp. for which terms were not disclosed. Both deals center on PTC’s Gene Expression Modulation by Small-molecules (GEMS) technology platform, which identifies small molecules that regulate post-transcriptional control mechanisms. In the Roche deal, PTC will get $12 million up front as well as research funding to find small molecules that hit four jointly selected central nervous system disease targets. Each target could eventually bring up to $239 million in research, development, regulatory and commercial See PTC, Page 3 FDA Panel: More Clolar Data Needed for Approval in AML By Donna Young Washington Editor SILVER SPRING Md. – An FDA advisory panel Tuesday said more data were needed to validate the efficacy and safety of drugs from Genzyme Corp. and Vion Pharmaceuticals Inc. to treat elderly patients with acute myeloid leukemia (AML), stating that the firms’ single-arm Phase II studies were insufficient on which to base approval. Shares of Genzyme (NASDAQ:GENZ) closed at $55.39, down 32 cents, while shares of Vion (OTC BB:VION) plunged 23 percent, or 77 cents, to close at $2.60. In a 9-to-3 vote, the FDA Oncologic Drugs Advisory Committee (ODAC) said Genzyme should be required to conduct a randomized controlled trial before regulators consider approving Clolar (clofarabine) as a first-line treatment for adults 60 years or older with AML with at least See FDA, Page 4 One Down, One Left: Acadia’s Pimavanserin Fails in Phase III By Jennifer Boggs Assistant Managing Editor Any expectations raised in May when Acadia Pharmaceuticals Inc. impressed investors with a potentially lucrative North American partnership for Phase III-stage pimavanserin were dashed when the drug failed to hit its primary endpoint in the first of two pivotal studies in Parkinson’s disease psychosis. News sent the San Diego-based firm’s stock (NASDAQ: ACAD), which had run up in the weeks ahead of the data, plunging 66 percent, or $3.84, to close Tuesday at $2. Top-line data from the six-week, 298-patient trial showed improvements in both treatment arms, according the Scale for the Assessment of Positive Symptoms, but Acadia said a higher-than-expected response in the placebo arm prevented the study from meeting statistical See Acadia, Page 5 Pain Deal Still in Place Icagen’s Phase II Asthma Win: Added Lure for Pfizer Buyout? By Randy Osborne Staff Writer Partner-forsaken Icagen Inc.’s upward stock ride on top-line Phase IIa news in allergic asthma with senicapoc gives the firm a leg up during its ongoing exploration of strategic alternatives, and just might lure pain collaborator Pfizer Inc. to the table for takeover talks. “I wouldn’t rule that out as a possibility,” said Richard D. Katz, chief financial officer of Research Triangle Park, N.C.-based Icagen. Research funding for pain research from Pfizer (soon to merge with Wyeth, of Madison, N.J.) was slated to end this month, but the deal was extended for six weeks, Katz said. “We’re in the process of discussing with them a See Icagen, Page 6 INSIDE: NOVAVAX TURNS TO FUNDING AS VLP VACCINE PASSES MIDSTAGE..............2 CLINIC ROUNDUP..........................................................................................3 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2009 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
Slide 2: WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 2 OF 7 Second Phase II Trial Positive Novavax Will Need More Cash to Push VLP Vaccine Farther By Catherine Hollingsworth Staff Writer Novavax reported positive results from a second Phase II trial of its trivalent seasonal influenza virus-like particle (VLP) vaccine candidate. The Rockville, Md.-based company has a Spain-based partner, ROVI Pharmaceutical Laboratories SA, that is helping to fund the VLP vaccine studies through Phase III and ultimately toward an anticipated regulatory approval in Europe. But the company will need additional funding to satisfy FDA requirements, Rahul Singhvi, president and CEO of Novavax, told BioWorld Today. In the U.S., Novavax has said that it is seeking support from the Health and Human Services Department’s Biomedical Advanced Research and Development Authority. The company also is considering a potential partner as it seeks U.S. approval of the VLP product, Singhvi indicated. In its most recent earnings report, Novavax reported that it had close to $38 million in cash and no long-term debt on its balance sheet. The latest Phase II study of 221 patients showed that the VLP-based vaccine was well tolerated and induced robust immune responses against the three influenza strains (H3N2, H1N1 and B). The three strains were matched to the strains recommended for influenza vaccines during the past flu season, 2008-2009. The positive outcome of the second Phase II study supported moving ahead with larger head-to-head trials of VLP and egg-based vaccines, the first of which is scheduled to start this fall in elderly adults, Novavax said. Phase III studies are expected next year. The previous Phase II study also was positive. While the first Phase II study involved a 2005-2006 formulation, the second study showed that the product worked against strains targeted for the 2008-2009 flu season, Singhvi explained. Novavax has now shown that the VLP vaccine candidate could work in different formulations, he said. Novavax hopes that its vaccine candidate, which is made in cell cultures, will stand apart from those that are made in egg cultures, which can have a lengthy cultivation period. Novavax’s vaccine candidate has the potential to be made faster for annual flu season. Another distinction is that Novavax’s VLP vaccine candidate includes three viral proteins important for inducing a broad immune response: two surface proteins, hemagglutinin (HA) and neuraminidase (NA), and a core matrix protein, M1. Most seasonal vaccines consist almost entirely of HA with little or no NA and M1 , according to the company’s website. The HA protein induces an antibody that neutralizes or blocks the growth of the virus; NA induces antibodies that prevent cell-to-cell transmission of virus down the respiratory tract, potentially reducing the severity of influenza disease; and cell-mediated immune responses to M1 may lead to destruction of cells already infected. VLPs are recombinant structures that mimic the size and shape of a virus. They are incapable of replication but can induce potent immune response, the company said. Rodman & Renshaw analyst Elemer Piros wrote in a research note that “Novavax’s VLP vaccine technology is truly revolutionary compared to the traditional egg-based vaccines.” He added that the company’s developing seasonal vaccine business “may be overlooked,” noting that it is applying its technology toward developing vaccines for respiratory syncytial virus, shingles, HIV, and severe acute respiratory syndrome as well as pandemic H1N1 vaccine. Its VLP-based vaccine against the H1N1 flu vaccine is advancing into preclinical development and could enter the clinic later this year. The company’s drug candidate for RSV also is expected to be in the clinic next year, Singhvi said. Shares in the Rockville, Md.-based company (NASDAQ: NVAX) rose 61 cents, to close at $6.65 Tuesday. ■ SUBSCRIBER INFORMATION Please call (800) 688-2421 to subscribe or if you have fax transmission problems. Outside U.S. and Canada, call (404) 262-5476. Our customer service hours are 8:30 a.m. to 6:00 p.m. EST. Glen Harris, (404) 262-5408 Jennifer Boggs, (404) 262-5427 Anette Breindl, (518) 595-4041 Trista Morrison, (858) 901-4785 Donna Young, (202) 739-9556 Catherine Hollingsworth, (301) 576-0667 Senior Vice President/Group Publisher: Donald R. Johnston, (404) 262-5439 Internet: http://www.bioworld.com 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 of this publication. Mention of products or services does not constitute endorsement. BioWorld® and BioWorld® Today are trademarks of AHC Media LLC, a Thompson Publishing Group company. Copyright © 2009 AHC Media LLC. All Rights Reserved. No part of this publication may be reproduced without the written consent of AHC Media LLC. (GST Registration Number R128870672). ATLANTA NEWSROOM: Managing Editor: Glen Harris. Assistant Managing Editor: Jennifer Boggs. Senior Staff Writer: Karen Pihl-Carey. Senior Production Editor: Ann Duncan. Staff Writer: Randy Osborne. WASHINGTON BUREAU: Washington Editor: Donna Young. Staff Writer: Catherine Hollingsworth. WEST COAST BUREAU: Staff Writer: Trista Morrison. EAST COAST BUREAU: Science Editor: Anette Breindl. BUSINESS OFFICE: Senior Vice President/Group Publisher: Donald R. Johnston. Senior Marketing Product Manager: Chris Walker. Marketing Coordinator: Sonia Blanco. 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Slide 3: WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 3 OF 7 PTC Continued from page 1 milestone payments, plus double-digit royalties, and Roche has an option to add four more targets under the same terms. All-told that’s $1.9 billion in potential milestones. PTC’s senior vice president of corporate development, Cláudia Hirawat, noted that the payments are spread across the development and commercialization continuum with no particular emphasis on the front or back end. Concurrent with the new Roche deal, PTC announced that Celgene exercised its option to partner on a GEMS oncology target. Two years ago, Celgene made a $20 million equity investment in PTC in exchange for options on two targets, and the exercise of one of them will trigger an undisclosed stream of discovery, development, regulatory and commercial milestone payments as well as royalties. (See BioWorld Today, Sept. 14, 2007.) Roche and Celgene are only the latest to notice the sparkle of PTC’s GEMS. The company previously inked a $212 million hepatitis C deal with Schering-Plough Corp., a $345 million cardiovascular deal with CV Therapeutics Inc. (now part of Gilead Sciences Inc.) and a $1.2 billion deal with Pfizer Inc. (See BioWorld Today, March 21 , 2006, June 13, 2006, and Jan. 9, 2007.) Stuart Peltz, president and CEO of PTC, explained that the attraction of GEMS lies in its ability to provide control over difficult, intractable or uncharacterized protein targets. While the middle of an RNA molecule serves as a blueprint for making proteins, the untranslated ends of the molecule determine how much protein gets made. By focusing on the untranslated ends, PTC’s GEMS platform churns out drugs that can increase or decrease the amount of protein produced. In addition to providing a new approach to tough targets, GEMS can be used to replace temperamental biologics with convenient small molecules, Peltz said. PTC has its own GEMS molecule, PTC299, moving through Phase II trials for breast cancer, solid tumors, Kaposi’s sarcoma associated with human immunodeficiency virus infection and brain tumors associated with neurofibromatosis type 2. PTC299 targets vascular endothelial growth factor (VEGF), placing it into a crowded field that includes Avastin (bevacizumab, Genentech/Roche) and loads of experimental drugs, but Hirawat said PTC’s drug works “upstream of what everyone else is doing” and thus far hasn’t elicited typical VEGF inhibitor side effects. Yet the crown jewel of PTC’s pipeline has nothing to do with GEMS. Ataluren (formerly PTC124) is based on PTC’s nonsense suppression technology, which allows cellular machinery to read through nonsense mutations in genetic disorders, resulting in production of a functional protein. Ataluren is in a pivotal Phase IIb trial for muscular dys- trophy and a pivotal Phase III trial for cystic fibrosis. A proof-of-concept trial in hemophilia also is under way, and Peltz said PTC is “just nailing down” a fourth program, which likely will focus on a metabolic disorder. PTC inked a $437 million partnership with Genzyme Corp. on the drug last year but retained U.S. and Canadian rights. (See BioWorld Today, July 18, 2008.) Moving forward, “we think this is going to be a pretty exciting next couple of years,” Peltz said. In a time when many biotechs are struggling and experts claim you can’t build a fully integrated pharmaceutical company, PTC is well funded and moving the first of its internally discovered candidates toward the commercial realm. ■ C LINIC RO UNDUP • Ablynx NV, of Ghent Belgium, has begun a Phase II study for its antithrombotic Nanobody ALX-0081 , a first-inclass nanobody targeting von Willebrand Factor. The openlabel, randomized study is designed to evaluate the safety and efficacy of multiple doses of ALX-0081 vs. the GPIIb/IIIa inhibitor ReoPro in patients undergoing percutaneous coronary intervention. Patients with unstable angina or patients with stable angina with at least two factors indicating high risk will be included in the study. • Antisoma plc, of London, said the ATTRACT-1 Phase III trial of its tumor vasculature disrupting agent ASA404 in non-small-cell lung cancer (NSCLC) has reached its enrollment target of 1 ,200 patients. The trial is the single pivotal registration study for the drug as a first-line treatment for squamous and nonsquamous NSCLC, and is being conducted by Novartis AG, of Basel, Switzerland, Antisoma’s development and commercialization partner for ASA404. The company expects results will be available in time to support potential marketing applications in 201 1. • Kowa Co. Ltd., of Tokyo, presented data showing that pitavastatin is noninferior to atorvastatin and simvastatin at usual therapeutic doses in patients with primary hypercholesterolemia or combined dyslipidemia, as measured by reduction of low density lipoprotein cholesterol (LDL-C) from baseline. LDL-C target attainment data were similar when comparing pitavastatin to atorvastatin and simvastatin, although pitavastatin demonstrated significantly higher LDL-C target attainment compared to simvastatin in the lower dose study-arm (pitavastatin 2 mg vs. simvastatin 20 mg) (p = 0.047). Continued gradual increases in high density lipoprotein cholesterol were observed over the long-term, supported by data from a 52 week extension study. Pitavastatin also demonstrated a favorable safety and tolerability profile to 52 weeks. Data were presented at the European Society of Cardiology meeting in Barcelona, Spain. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2009 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
Slide 4: WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 4 OF 7 FDA Continued from page 1 one unfavorable baseline prognostic factor. A larger ODAC panel in the afternoon voted 13 to 0 that Vion also be required to conduct a randomized trial of Onrigin (laromustine) to establish the safety and efficacy of the drug as a single agent for remission induction in patients 60 years or older with de novo, poor-risk AML. AML, the most common type of acute leukemia, affects elderly adults very differently than younger patients with the disease. Elderly patients have higher risk factors and have lower response rates, with shorter overall survival. Genzyme and Vion argued that their drugs would fill an unmet need for elderly patients, a population for which two-thirds typically go untreated because of the poorer outcomes. In Genzyme’s CLO243 study, 51 of 1 12 patients, or 45.5 percent, achieved overall remission, defined as either complete remission or complete remission with incomplete platelet recovery. Of the 51 responders, 38, or 74.5 percent, achieved OR after one cycle of Clolar and 13, or 25.5 percent, achieved OR after two cycles. In Vion’s single-arm CLI-043 study, 27 of 85 patients, or 31.8 percent, achieved OR. In the company’s CLI-033 supportive single-arm study, 38.2 percent of patients achieved OR. Across all 140 elderly de novo poor-risk AML patients in both studies, 34.3 percent achieved OR. Although the ODAC panelists agreed that the remission rates were impressive, they were unconvinced that the Genzyme’s and Vion’s single-arm studies were sufficient to show that the drugs’ benefits outweighed the risks or that it was clear that the medications were any better than chemotherapy. Richard Pazdur, director of the FDA’s Office of Oncology Drug Products, noted that the agency had urged both companies to conduct randomized controlled trials before submitting their applications – advice that went unheeded by Genzyme and Vion. Clolar already is marketed in the U.S. The FDA in 2004 granted accelerated approval for the drug as treatment for pediatric patients with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens. The company has a commitment to complete a Phase III study of the drug in pediatric patients to gain full approval in that population. Pazdur, however, appeared to lob a warning shot across the bow by calling out the fact that the company has yet to start that trial six years after the approval. Genzyme officials said the company is nearing completion of its Phase I/II development program of Clolar in combination with chemotherapy and plans to start the Phase III randomized controlled trial in newly diagnosed children in 2010. But panelist Thomas Fleming, a professor of biostatistics at the University of Washington in Seattle, scolded Genzyme, asserting that the “congressional intent” of acceler- ated approval was for a “fairly timely” completion of followup studies. Michael Vasconcelles, group vice president and global therapeutic area head of transplant and oncology at Genzyme, said the early combination safety studies, which will serve as the basis of the randomized Phase III pediatric trial, were initiated “immediately after” the accelerated approval was granted and have been ongoing for the past four years. Mark Enyedy, head of oncology and multiple sclerosis at Genzyme, told reporters after the meeting that the company has had a “strong track record” of fulfilling postapproval commitments. “Genzyme is one of the companies in the industry that has completed those postapproval commitments,” he contended. Genzyme submitted its supplemental application for AML in elderly patients to the FDA in November 2008 based on its single-arm Phase II CLO243 trial of Clolar in older adult patients with previously untreated AML who were deemed unfit for conventional treatment with seven days of continuous infusion cytarabine plus three days of an anthracycline or anthracenedione, known as 7+3 induction chemotherapy. The company also submitted data from a supportive multicenter, single-arm study, known as BIOV-121 , in a similar population. Mark Hayes, group vice president of regulatory affairs at Genzyme, said the company had proposed a randomized controlled trial and submitted a special protocol assessment to the FDA for study CLO342 in March 2006 of Clolar in combination with low-dose cytarabine vs. low-dose cytarabine alone in previously untreated older adults with AML. But, he said, a panel of outside AML experts who reviewed the study protocol for Genzyme could not agree on an appropriate comparator arm for the proposed study population and recommended a single-arm, single-agent clinical study instead. But Pazdur noted that the FDA has heard similar arguments before from drugmakers. “This is a common, common comment,” he said. Pazdur argued that companies have several options for comparator arms, including using investigational drugs. Genzyme currently has an ongoing Phase III randomized, double-blind, placebo-controlled study comparing Clolar plus intermediate-dose cytarabine vs. intermediatedose cytarabine alone in adult patients 55 years or older with relapsed or refractory AML after receiving up to two prior induction regimens. But panelists noted that the population in that trial is very different in age and disease type as the population Genzyme is seeking approval for in its supplemental application. Vion’s application primarily relies on the findings of a single-arm study and a post-hoc subset of patients from a second single-arm study. The company also submitted data from a randomized controlled trial, which was placed on clinical hold due to excess death in the Onrigin arm. ■ To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2009 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
Slide 5: WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 5 OF 7 Acadia Continued from page 1 significance. Mean reductions in SAPS scores were 5.8 points in the 10-mg pimavanserin arm, 6.7 points in the 40mg arm and 5.9 points in the placebo group. The company plans further analysis, but executives said they were at a loss to explain the high placebo response. While the drug missed the SAPS endpoint in an earlier Phase II study, “we got a very nice separation between the treatment and placebo [groups],” Acadia CEO Uli Hacksell told BioWorld Today. During the firm’s conference call, executives said a previous four-week study had shown a change from baseline of only 1. 1 points in the placebo study, and investigators were expecting a similar trend in the Phase III trial, which was 90 percent powered for a 5-point difference between treatment and placebo. SAPS is the recommended endpoint to describe psychosis in Parkinson's disease, a condition involving hallucinations and delusions that affects about 40 percent of the estimated 1.5 million U.S. patients with Parkinson’s, Hacksell said. All patients enrolled in the study remained on their stable doses of dopamine drugs, and the study met its secondary endpoint of motoric tolerability, as measured by the Unified Parkinson's’ Disease Rating Scale, showing that it did not worsen motor symptoms associated with the disease. Pimavanserin also was found to be safe and well tolerated. Despite the disappointing data, Acadia remains confident that the drug has “considerable potential” in PDP, Hacksell said. And the company is holding out hope that the second Phase III trial of the selective 5-HT2A inverse agonist, which is about a year behind the first, won’t be thwarted by a similarly high placebo response. That trial is designed with the same endpoints but is testing 10-mg and 20-mg doses of pimavanserin. But a success in that case still won’t give Acadia a clear win. “We do believe we need two pivotal trials” before it can submit a regulatory filing, Hacksell said. That means Acadia will have to consult with partner Biovail Corp. Though Acadia funded both Phase III studies, terms of the May licensing deal call for Toronto-based Biovail to pick up the tab for further development and commercialization activities involving pimavanserin in PDP and any other neurological and psychiatric indications the companies decide to pursue. As Piper Jaffray analyst Edward Tenthoff put it in a research note: “Without funding from Biovail, we believe pimavanserin development will be halted once studies in PDP are complete.” But even if the second PDP study fails, Hacksell said the drug has shown promise in other indications, including Alzheimer’s disease psychosis, schizophrenia and insomnia. The companies already selected ADP as the second indication to explore and “we’ll decide on a path forward after we analyze the data” from the first PDP study, he added. Nevertheless, the first Phase III failure marked a setback for the firm, which had been riding high since pleasantly surprising analysts and investors with its potential $395 million Biovail deal. That partnership, coming before the release of Phase III data, sent the company’s stock soaring 134 percent and seemed to quell some of the doubt surrounding pimavanserin’s efficacy following mixed Phase II data. (See BioWorld Today, May 5, 2009.) It also offered some reassurance for investors skeptical about the drug’s potential in the PDP market. There are no FDA-approved drugs specifically for PDP, but there are generic atypical antipsychotics that are used off-label, with limited efficacy. The most efficacious of those is clozapine, but it carries several well-known side effects. Acadia, which earned $30 million up front from Biovail in its May licensing deal, had about $66.2 million in the bank as of June 30. The firm said it expects to end this year with more than $40 million, enough to carry it into the first half of 201 1. Beyond pimavanserin, it has an ongoing collaboration with Allergan Inc., of Irvine, Calif., for a Phase II-stage drug for chronic pain and a Phase I program in glaucoma. And it’s getting ready to start advancing additional programs from its internal pipeline, starting with ACP-106, a selective 5-HT2A inverse agonist that is in investigational new drug application-track development. ■ F INANCINGS RO UNDUP • Alizé Pharma, of Lyon, France, has raised €3 million (US$4.27 million) of funding. A new investor, SHAM, an insurance company, led the round. Existing investors, Octalfa SAS and CEMA Inc. also participated. Including this new round, Alizé Pharma Group has raised €4.8 million from those three investors since its creation in 2007. The funds raised will allow Alizé Pharma to finalize the optimization and formulation of a first drug candidate for AZP01 and to file a clinical trial application to undertake the clinical development of its Asparec product. • Immunomedics Inc., of Morris Plains, N.J., filed papers with the SEC saying it planned to raise $151. 1 million by selling stock and warrants. The company said it may issue any combination of up to 20 million shares of common stock and 3 million warrants. Last week the company’s shares jumped 277 percent when epratuzumab had a 24.9 percent treatment advantage over placebo in patients with systemic lupus erythematosus in a Phase IIb dosing study. (See BioWorld Today, Aug. 28, 2009.) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2009 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
Slide 6: WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 6 OF 7 Icagen Continued from page 1 renewal that would extend for another year,” he said. As of June 30, Icagen had about $24 million in cash and cash equivalents, enough to operate “for at least the next 12 months,” the company said in its quarterly financial filing, but will need to raise money to operate beyond that time. Shares of Icagen (NASDAQ:ICGN) closed Tuesday at $1.43, up 47 cents, or 49 percent, on word that the KCa3. 1 channel blocker significantly reduced asthma over placebo in patients given an inhaled allergen. Senicapoc fizzled in Phase III against sickle cell anemia two years ago, with Icagen stopping the trial after a monitoring board found the study unlikely to yield positive data. “We had efficacy in terms of many of the secondary endpoints, but didn’t hit the primary endpoint, the control of crises,” Katz said, adding that senicapoc was “clearly demonstrating biologic activity.” The targeted channel is expressed on red blood cells and white blood cells. Dehydration of red blood cells was the problem Icagen sought to solve in sickle cell disease. “In terms of effects on white blood cells [in immune disorders], the channel becomes very important in controlling the influx of calcium, even though it’s a potassium channel,” Katz said. Results in the 34-patient, UK asthma trial were determined by the drop in FEV1 , the volume of air that can be forcibly exhaled in one second. Improvement in the average FEV1 decline was 29 percent (p = 0.06). In the maximum decline, the number was 18 percent (p = 0. 15), the area under the curve amounted to 28 percent (p = 0.08) of FEV1. A secondary endpoint, the fraction of exhaled nitric oxide (a measure of airway inflammation, typically high in asthmatic patients), dropped by 24 percent (p = 0. 10) among drug patients compared to those on placebo. Another secondary endpoint – early asthmatic response – didn’t change, a finding in line with preclinical results. Senicapoc was well tolerated, with no serious adverse events. Icagen plans to offer full results at an upcoming scientific conference. Meanwhile, the company has finished enrollment in a 69-patient U.S. trial testing senicapoc in exercise-induced asthma, with the same FEV1 endpoint. Data are expected in the fourth quarter of this year. In June, Icagen signed J.P. Morgan to help figure out next steps. Senicapoc’s 2007 failure in sickle cell was followed by the loss of the related deals with McNeil Consumer & Specialty Pharmaceuticals, a subsidiary of New Brunswick, N.J.-based Johnson & Johnson. Shortly afterward, Bristol-Myers Squibb Co., of New York, backed away, deciding not to pursue development of the lead compound discovered in a long-standing deal targeting atrial fibrillation. Better news followed in the same year, when New Yorkbased Pfizer inked a potential $1 billion-plus deal to develop sodium ion channel modulators for pain, one of the pharma giant’s stated high-priority therapeutic areas. (See BioWorld Today, Aug. 15, 2007.) Outside of oncology, pain is considered by some as Pfizer’s biggest zone of interest. The firm “doesn’t jump to mind when you think of the major asthma companies,” Katz conceded, but said he “would be surprised if they didn’t have ongoing efforts in inflammatory indications, if not asthma” itself. Other large and “modest-sized” would-be partners have expressed an interest in senicapoc, Katz told BioWorld Today. “I’m not concerned that pharma is shying away from asthma,” he added, especially since the drug – unlike most existing therapies – is orally delivered. Partnering likely will wait until the next Phase II data become available and maybe later, when the “multi-thousand-patient” Phase III asthma studies require deeper pockets, Katz said. ■ O THER N EWS T O N OTE • Affitech A/S, of Copenhagen, Denmark, appointed Robert Burns as CEO and Alexander Duncan as senior vice president of research and development. Burns was previously CEO of Celldex Therapeutics Inc., while Duncan hails from AstraZeneca plc. The appointments follow Affitech’s merger with Pharmexa A/S. (See BioWorld Today, March 5, 2009.) • Applied NeuroSolutions Inc., of Vernon Hills, Ill., appointed Craig S. Taylor as its president, CEO and a member of its board. Previously a partner at Adams Street Partners LLC, Taylor served as associate director of business development for G.D. Searle (now part of Pfizer) and was a senior research biochemist at Abbott Laboratories. • Basilea Pharmaceutica Ltd., of Basel, Switzerland, said that the FDA considers the response submitted by the sponsor Johnson & Johnson Pharmaceutical Research & Development LLC, of Raritan, N.J., as a complete, class 2 response. The submission addresses the FDA’s November 2008 letter concerning the ceftobiprole new drug application for complicated skin and skin structure infections. Ceftobiprole is being developed through an exclusive worldwide collaboration between Basilea Pharmaceutica International Ltd., also of Basel, and Cilag GmbH International, a J&J company. (See BioWorld Today, Dec. 1, 2008.) • Bristol-Myers Squibb Co., of New York, completed its tender offer for shares of Medarex Inc., of Princeton, N.J. As of the expiration of the offering period, Medarex shareholders tendered about 120.4 million shares, which, along with the 2.9 million shares owned by BMS since 2005, represented about 90.7 percent of the shares outstanding. The two firms agreed to the $2.4 billion acquisition in July. (See BioWorld Today, July 24, 2009.) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2009 AHC Media LLC. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
Slide 7: WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 7 OF 7 O THER N EWS T O N OTE • Cellectis SA, of Romainville, France, and Monsanto Co., of St. Louis, entered into a nonexclusive research and commercial license agreement for broad use of Celectis’ meganuclease technology in plants. Cellectis will receive an up-front payment of €3 million (US$ 4.2 million), Monsanto will make an equity investment of €1 million to allow Cellectis to scale the technology for agriculture. Cellectis also will be eligible to receive fees for the development of each meganuclease, success-based milestones and may receive royalties on certain traits commercialized by Monsanto. Further financial details were not disclosed. • Champions Biotechnology Inc., of Baltimore, entered a deal with an undisclosed large biotech company to evaluate an oncology therapeutic using Champion’ Biomerk Tumorgraft platform, which is designed to enable identification of the most promising development path for a compound and also identify potential gene pathways of response and resistance, as well as prognostic molecular biomarkers. Terms were not disclosed. • Clinical Data Inc., of Newton, Mass., sold the equipment and property associated with its wholly owned subsidiary – Germantown, Md.-based Avalon Pharmaceuticals Inc. – to Blacksburg, Va.-based Intrexon Corp. for $1.5 million cash. Clinical Data retains Avalon’s oncology drug candidates, intellectual property and biomarker discovery platform, while Intrexon gets access to a bioassay platform to complement its transgene engineering platform. • Medivir AB, of Stockholm, Sweden, selected MIV-71 1 as a candidate drug for bone diseases. MIV-71 1 is a smallmolecule protease cathepsin K inhibitor distinguishable from MIV-710, and both drugs are being prioritized over MIV-701 for osteoporosis, osteoarthritis, rheumatoid arthritis and metastatic bone disease. • Metabasis Therapeutics Inc., of San Diego, said its president, CEO and chief scientific officer, Mark Erion, resigned as an officer, effective Oct. 31. Erion, who also resigned his position on the board, will consult with the company after that date on matters related to the licensing or sale of its pipeline and advanced discovery programs or other strategic alternatives. Erion is joining Whitehouse Station, N.J.-based Merck & Co. Inc. as vice president and worldwide basis franchise head of diabetes and obesity. David Hale, Metabasis’ chairman, was appointed executive chairman. • MiddleBrook Pharmaceuticals Inc., of Westlake, Texas, has cut its sales managers and field sales representatives by 25 percent and reduced corporate staff by about 20 percent. As a result, the firm expects to achieve approximately $15 million in annual savings. The company said it now anticipates that its total operating expenses for 2009 will range between $78 million and $83 million, vs. its previous guidance of operating expenses ranging between $83 million and $88 million. It will continue to maintain sales coverage of the physicians with the highest potential to prescribe Moxatag (extended-release amoxicillin) Tablets, 775 mg. • Neurobiological Technologies Inc., of Emeryville, Calif., has decided to liquidate the company’s assets and to dissolve the company. The company intends to distribute the majority of its available cash to its stockholders. A stockholders meeting will be held to vote on the plan. As of June 30, the company reported having cash, cash equivalents and short-term investments of $24 million, • Nuvo Research Inc., of Missisauga, Ontario, has entered into a cooperative drug development project with the Fraunhofer Institute for Cell Therapy and Immunology IZI in Leipzig, Germany, for the preclinical and clinical development of WF10 as a potential treatment for allergic rhinitis. The Development Bank of Saxony in Germany will provide financial support for the project, which will be conducted in Leipzig through Nuvo Research GmbH, a Nuvo subsidiary. • Selexis SA, of Geneva, and NKT Therapeutics Inc., of Waltham, Mass., entered a research services deal under which Selexis will screen antibody variants and generate Chinese hamster ovary-based production cell lines using its Integra-D2M • Shire plc, of Basingstoke, UK, said its final two Phase III trials for enzyme replacement therapy velaglucerase alfa met their endpoints in Type I Gaucher’s disease and the company’s rolling new drug application at the FDA has been completed. (See BioWorld Today, Aug. 4, 2009.) • SkyePharma plc, of London, said that additional clinical work will be required to provide more data on dosing for its lead development product, Flutiform (fluticasone propionate/formoterol fumarate), for persistent asthma in patients 12 years of age and older. The need for additional clinical work was confirmed in a meeting with the FDA, the company said. Due to the additional work, the board believes that it is unlikely that Flutiform will be approved in the U.S. before the second half of 201 1. In the meantime, the FDA’s review of product is continuing. • Stemedica Cell Technologies Inc., of San Diego, has been granted a license by the California Food & Drug Branch to manufacture stem cells for human clinical trials. The license recognizes Stemedica as being compliant with California law and the applicable provisions of the Code of Federal Regulations. • Zealand Pharma AS, of Glostrup, Denmark, said it is moving its new generation drug candidate for Type II diabetes into preclinical development. The dual glucagon-GLP1 agonist, ZP2929, has the potential to significantly decrease body weight and reduce the risk of diabetesrelated complications, the company said. 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