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  • BIOTECH’S MOST RESPECTED NEWS SOURCE FOR OVER 17 YEARS WEDNESDAY VOLUME 20, NO. 169 SEPTEMBER 2, 2009 PAGE 1 OF 7 Celgene Likes PTC’s GEMS, Too FDA Panel: More Clolar Data PTC Gets $12M Up Front in Needed for Approval in AML Roche Deal Worth up to $1.9B By Donna Young By Trista Morrison Washington Editor Staff Writer SILVER SPRING Md. – An FDA advisory panel Tuesday PTC Therapeutics Inc. is expected to announce two said more data were needed to validate the efficacy and drug discovery deals on Wednesday, a potential $1.9 billion safety of drugs from Genzyme Corp. and Vion Pharmaceu- whopper with Roche AG and an option exercise by Celgene ticals Inc. to treat elderly patients with acute myeloid Corp. for which terms were not disclosed. leukemia (AML), stating that the firms’ single-arm Phase II Both deals center on PTC’s Gene Expression Modula- studies were insufficient on which to base approval. tion by Small-molecules (GEMS) technology platform, Shares of Genzyme (NASDAQ:GENZ) closed at $55.39, which identifies small molecules that regulate post-tran- down 32 cents, while shares of Vion (OTC BB:VION) scriptional control mechanisms. plunged 23 percent, or 77 cents, to close at $2.60. In the Roche deal, PTC will get $12 million up front as In a 9-to-3 vote, the FDA Oncologic Drugs Advisory well as research funding to find small molecules that hit Committee (ODAC) said Genzyme should be required to four jointly selected central nervous system disease tar- conduct a randomized controlled trial before regulators gets. Each target could eventually bring up to $239 million consider approving Clolar (clofarabine) as a first-line treat- in research, development, regulatory and commercial ment for adults 60 years or older with AML with at least See PTC, Page 3 See FDA, Page 4 One Down, One Left: Acadia’s Pain Deal Still in Place Pimavanserin Fails in Phase III Icagen’s Phase II Asthma Win: By Jennifer Boggs Added Lure for Pfizer Buyout? Assistant Managing Editor By Randy Osborne Any expectations raised in May when Acadia Pharma- Staff Writer ceuticals Inc. impressed investors with a potentially lucra- Partner-forsaken Icagen Inc.’s upward stock ride on tive North American partnership for Phase III-stage pima- top-line Phase IIa news in allergic asthma with senicapoc vanserin were dashed when the drug failed to hit its pri- gives the firm a leg up during its ongoing exploration of mary endpoint in the first of two pivotal studies in Parkin- strategic alternatives, and just might lure pain collaborator son’s disease psychosis. Pfizer Inc. to the table for takeover talks. News sent the San Diego-based firm’s stock (NASDAQ: “I wouldn’t rule that out as a possibility,” said Richard ACAD), which had run up in the weeks ahead of the data, D. Katz, chief financial officer of Research Triangle Park, plunging 66 percent, or $3.84, to close Tuesday at $2. N.C.-based Icagen. Top-line data from the six-week, 298-patient trial Research funding for pain research from Pfizer (soon showed improvements in both treatment arms, according to merge with Wyeth, of Madison, N.J.) was slated to end the Scale for the Assessment of Positive Symptoms, but this month, but the deal was extended for six weeks, Katz Acadia said a higher-than-expected response in the said. placebo arm prevented the study from meeting statistical “We’re in the process of discussing with them a See Acadia, Page 5 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
  • WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 2 OF 7 Second Phase II Trial Positive strains targeted for the 2008-2009 flu season, Singhvi Novavax Will Need More Cash explained. Novavax has now shown that the VLP vaccine candidate could work in different formulations, he said. to Push VLP Vaccine Farther Novavax hopes that its vaccine candidate, which is By Catherine Hollingsworth made in cell cultures, will stand apart from those that are Staff Writer made in egg cultures, which can have a lengthy cultivation Novavax reported positive results from a second Phase period. Novavax’s vaccine candidate has the potential to be II trial of its trivalent seasonal influenza virus-like particle made faster for annual flu season. (VLP) vaccine candidate. Another distinction is that Novavax’s VLP vaccine can- The Rockville, Md.-based company has a Spain-based didate includes three viral proteins important for inducing partner, ROVI Pharmaceutical Laboratories SA, that is help- a broad immune response: two surface proteins, hemag- ing to fund the VLP vaccine studies through Phase III and glutinin (HA) and neuraminidase (NA), and a core matrix ultimately toward an anticipated regulatory approval in protein, M1. Most seasonal vaccines consist almost entirely Europe. of HA with little or no NA and M1 , according to the com- But the company will need additional funding to satisfy pany’s website. FDA requirements, Rahul Singhvi, president and CEO of The HA protein induces an antibody that neutralizes or Novavax, told BioWorld Today. blocks the growth of the virus; NA induces antibodies that In the U.S., Novavax has said that it is seeking support prevent cell-to-cell transmission of virus down the respira- from the Health and Human Services Department’s Bio- tory tract, potentially reducing the severity of influenza medical Advanced Research and Development Authority. disease; and cell-mediated immune responses to M1 may The company also is considering a potential partner as it lead to destruction of cells already infected. seeks U.S. approval of the VLP product, Singhvi indicated. VLPs are recombinant structures that mimic the size In its most recent earnings report, Novavax reported and shape of a virus. They are incapable of replication but that it had close to $38 million in cash and no long-term can induce potent immune response, the company said. debt on its balance sheet. Rodman & Renshaw analyst Elemer Piros wrote in a The latest Phase II study of 221 patients showed that research note that “Novavax’s VLP vaccine technology is the VLP-based vaccine was well tolerated and induced truly revolutionary compared to the traditional egg-based robust immune responses against the three influenza vaccines.” strains (H3N2, H1N1 and B). The three strains were matched He added that the company’s developing seasonal vac- to the strains recommended for influenza vaccines during cine business “may be overlooked,” noting that it is apply- the past flu season, 2008-2009. ing its technology toward developing vaccines for respira- The positive outcome of the second Phase II study sup- tory syncytial virus, shingles, HIV, and severe acute respira- ported moving ahead with larger head-to-head trials of VLP tory syndrome as well as pandemic H1N1 vaccine. and egg-based vaccines, the first of which is scheduled to Its VLP-based vaccine against the H1N1 flu vaccine is start this fall in elderly adults, Novavax said. Phase III stud- advancing into preclinical development and could enter the ies are expected next year. clinic later this year. The company’s drug candidate for RSV The previous Phase II study also was positive. While the also is expected to be in the clinic next year, Singhvi said. first Phase II study involved a 2005-2006 formulation, the Shares in the Rockville, Md.-based company (NASDAQ: second study showed that the product worked against NVAX) rose 61 cents, to close at $6.65 Tuesday. ■ 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) 688-2421 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) © 2009 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 Senior Marketing Product Manager: Chris Walker. Internet: http://www.bioworld.com Marketing Coordinator: Sonia Blanco. Account Representatives: Bob Sobel, Randee Papush. 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) 262-5479. PRESS MATERIALS: Send all press releases and related information to newsdesk@bioworld.com.
  • WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 3 OF 7 PTC trophy and a pivotal Phase III trial for cystic fibrosis. A Continued from page 1 proof-of-concept trial in hemophilia also is under way, and milestone payments, plus double-digit royalties, and Roche Peltz said PTC is “just nailing down” a fourth program, has an option to add four more targets under the same which likely will focus on a metabolic disorder. PTC inked a terms. $437 million partnership with Genzyme Corp. on the drug All-told that’s $1.9 billion in potential milestones. PTC’s last year but retained U.S. and Canadian rights. (See senior vice president of corporate development, Cláudia BioWorld Today, July 18, 2008.) Hirawat, noted that the payments are spread across the Moving forward, “we think this is going to be a pretty development and commercialization continuum with no exciting next couple of years,” Peltz said. particular emphasis on the front or back end. In a time when many biotechs are struggling and Concurrent with the new Roche deal, PTC announced experts claim you can’t build a fully integrated pharmaceu- that Celgene exercised its option to partner on a GEMS tical company, PTC is well funded and moving the first of its oncology target. Two years ago, Celgene made a $20 mil- internally discovered candidates toward the commercial lion equity investment in PTC in exchange for options on realm. ■ 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.) C L I N I C RO U N D U P Roche and Celgene are only the latest to notice the sparkle of PTC’s GEMS. The company previously inked a • Ablynx NV, of Ghent Belgium, has begun a Phase II $212 million hepatitis C deal with Schering-Plough Corp., a study for its antithrombotic Nanobody ALX-0081 , a first-in- $345 million cardiovascular deal with CV Therapeutics Inc. class nanobody targeting von Willebrand Factor. The open- (now part of Gilead Sciences Inc.) and a $1.2 billion deal label, randomized study is designed to evaluate the safety with Pfizer Inc. (See BioWorld Today, March 21 , 2006, June and efficacy of multiple doses of ALX-0081 vs. the GPIIb/IIIa 13, 2006, and Jan. 9, 2007.) inhibitor ReoPro in patients undergoing percutaneous Stuart Peltz, president and CEO of PTC, explained that coronary intervention. Patients with unstable angina or the attraction of GEMS lies in its ability to provide control patients with stable angina with at least two factors indi- over difficult, intractable or uncharacterized protein tar- cating high risk will be included in the study. gets. • Antisoma plc, of London, said the ATTRACT-1 Phase While the middle of an RNA molecule serves as a blue- III trial of its tumor vasculature disrupting agent ASA404 in print for making proteins, the untranslated ends of the non-small-cell lung cancer (NSCLC) has reached its enroll- molecule determine how much protein gets made. By ment target of 1 ,200 patients. The trial is the single pivotal focusing on the untranslated ends, PTC’s GEMS platform registration study for the drug as a first-line treatment for churns out drugs that can increase or decrease the amount squamous and nonsquamous NSCLC, and is being con- of protein produced. In addition to providing a new ducted by Novartis AG, of Basel, Switzerland, Antisoma’s approach to tough targets, GEMS can be used to replace development and commercialization partner for ASA404. temperamental biologics with convenient small molecules, The company expects results will be available in time to Peltz said. support potential marketing applications in 201 1. PTC has its own GEMS molecule, PTC299, moving • Kowa Co. Ltd., of Tokyo, presented data showing through Phase II trials for breast cancer, solid tumors, that pitavastatin is noninferior to atorvastatin and simvas- Kaposi’s sarcoma associated with human immunodefi- tatin at usual therapeutic doses in patients with primary ciency virus infection and brain tumors associated with hypercholesterolemia or combined dyslipidemia, as meas- neurofibromatosis type 2. ured by reduction of low density lipoprotein cholesterol PTC299 targets vascular endothelial growth factor (LDL-C) from baseline. LDL-C target attainment data were (VEGF), placing it into a crowded field that includes Avastin similar when comparing pitavastatin to atorvastatin and (bevacizumab, Genentech/Roche) and loads of experimen- simvastatin, although pitavastatin demonstrated signifi- tal drugs, but Hirawat said PTC’s drug works “upstream of cantly higher LDL-C target attainment compared to sim- what everyone else is doing” and thus far hasn’t elicited vastatin in the lower dose study-arm (pitavastatin 2 mg vs. typical VEGF inhibitor side effects. simvastatin 20 mg) (p = 0.047). Continued gradual Yet the crown jewel of PTC’s pipeline has nothing to do increases in high density lipoprotein cholesterol were with GEMS. Ataluren (formerly PTC124) is based on PTC’s observed over the long-term, supported by data from a 52 nonsense suppression technology, which allows cellular week extension study. Pitavastatin also demonstrated a machinery to read through nonsense mutations in genetic favorable safety and tolerability profile to 52 weeks. Data disorders, resulting in production of a functional protein. were presented at the European Society of Cardiology Ataluren is in a pivotal Phase IIb trial for muscular dys- 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
  • WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 4 OF 7 FDA ated approval was for a “fairly timely” completion of follow- Continued from page 1 up studies. one unfavorable baseline prognostic factor. Michael Vasconcelles, group vice president and global A larger ODAC panel in the afternoon voted 13 to 0 that therapeutic area head of transplant and oncology at Gen- Vion also be required to conduct a randomized trial of zyme, said the early combination safety studies, which will Onrigin (laromustine) to establish the safety and efficacy of serve as the basis of the randomized Phase III pediatric the drug as a single agent for remission induction in trial, were initiated “immediately after” the accelerated patients 60 years or older with de novo, poor-risk AML. approval was granted and have been ongoing for the past AML, the most common type of acute leukemia, affects four years. elderly adults very differently than younger patients with Mark Enyedy, head of oncology and multiple sclerosis the disease. Elderly patients have higher risk factors and at Genzyme, told reporters after the meeting that the com- have lower response rates, with shorter overall survival. pany has had a “strong track record” of fulfilling postap- Genzyme and Vion argued that their drugs would fill an proval commitments. “Genzyme is one of the companies in unmet need for elderly patients, a population for which the industry that has completed those postapproval com- two-thirds typically go untreated because of the poorer mitments,” he contended. outcomes. Genzyme submitted its supplemental application for In Genzyme’s CLO243 study, 51 of 1 12 patients, or 45.5 AML in elderly patients to the FDA in November 2008 based percent, achieved overall remission, defined as either com- on its single-arm Phase II CLO243 trial of Clolar in older adult plete remission or complete remission with incomplete patients with previously untreated AML who were deemed platelet recovery. Of the 51 responders, 38, or 74.5 percent, unfit for conventional treatment with seven days of continu- achieved OR after one cycle of Clolar and 13, or 25.5 per- ous infusion cytarabine plus three days of an anthracycline cent, achieved OR after two cycles. or anthracenedione, known as 7+3 induction chemotherapy. In Vion’s single-arm CLI-043 study, 27 of 85 patients, or The company also submitted data from a supportive 31.8 percent, achieved OR. In the company’s CLI-033 sup- multicenter, single-arm study, known as BIOV-121 , in a sim- portive single-arm study, 38.2 percent of patients achieved ilar population. OR. Across all 140 elderly de novo poor-risk AML patients in Mark Hayes, group vice president of regulatory affairs at both studies, 34.3 percent achieved OR. Genzyme, said the company had proposed a randomized Although the ODAC panelists agreed that the remission controlled trial and submitted a special protocol assessment rates were impressive, they were unconvinced that the Gen- to the FDA for study CLO342 in March 2006 of Clolar in com- zyme’s and Vion’s single-arm studies were sufficient to show bination with low-dose cytarabine vs. low-dose cytarabine that the drugs’ benefits outweighed the risks or that it was alone in previously untreated older adults with AML. clear that the medications were any better than chemotherapy. But, he said, a panel of outside AML experts who Richard Pazdur, director of the FDA’s Office of Oncol- reviewed the study protocol for Genzyme could not agree ogy Drug Products, noted that the agency had urged both on an appropriate comparator arm for the proposed study companies to conduct randomized controlled trials before population and recommended a single-arm, single-agent submitting their applications – advice that went unheeded clinical study instead. by Genzyme and Vion. But Pazdur noted that the FDA has heard similar argu- Clolar already is marketed in the U.S. The FDA in 2004 ments before from drugmakers. granted accelerated approval for the drug as treatment for “This is a common, common comment,” he said. Pazdur pediatric patients with relapsed or refractory acute lym- argued that companies have several options for compara- phoblastic leukemia after at least two prior regimens. The tor arms, including using investigational drugs. company has a commitment to complete a Phase III study Genzyme currently has an ongoing Phase III random- of the drug in pediatric patients to gain full approval in that ized, double-blind, placebo-controlled study comparing population. Clolar plus intermediate-dose cytarabine vs. intermediate- Pazdur, however, appeared to lob a warning shot dose cytarabine alone in adult patients 55 years or older across the bow by calling out the fact that the company has with relapsed or refractory AML after receiving up to two yet to start that trial six years after the approval. prior induction regimens. Genzyme officials said the company is nearing com- But panelists noted that the population in that trial is pletion of its Phase I/II development program of Clolar in very different in age and disease type as the population Gen- combination with chemotherapy and plans to start the zyme is seeking approval for in its supplemental application. Phase III randomized controlled trial in newly diagnosed Vion’s application primarily relies on the findings of a children in 2010. single-arm study and a post-hoc subset of patients from a But panelist Thomas Fleming, a professor of biostatis- second single-arm study. The company also submitted data tics at the University of Washington in Seattle, scolded Gen- from a randomized controlled trial, which was placed on zyme, asserting that the “congressional intent” of acceler- 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
  • WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 5 OF 7 Acadia Alzheimer’s disease psychosis, schizophrenia and insom- Continued from page 1 nia. The companies already selected ADP as the second significance. Mean reductions in SAPS scores were 5.8 indication to explore and “we’ll decide on a path forward points in the 10-mg pimavanserin arm, 6.7 points in the 40- after we analyze the data” from the first PDP study, he mg arm and 5.9 points in the placebo group. added. The company plans further analysis, but executives Nevertheless, the first Phase III failure marked a set- said they were at a loss to explain the high placebo back for the firm, which had been riding high since pleas- response. While the drug missed the SAPS endpoint in an antly surprising analysts and investors with its potential earlier Phase II study, “we got a very nice separation $395 million Biovail deal. That partnership, coming before between the treatment and placebo [groups],” Acadia CEO the release of Phase III data, sent the company’s stock soar- Uli Hacksell told BioWorld Today. ing 134 percent and seemed to quell some of the doubt sur- During the firm’s conference call, executives said a pre- rounding pimavanserin’s efficacy following mixed Phase II vious four-week study had shown a change from baseline data. (See BioWorld Today, May 5, 2009.) of only 1. 1 points in the placebo study, and investigators It also offered some reassurance for investors skeptical were expecting a similar trend in the Phase III trial, which about the drug’s potential in the PDP market. There are no was 90 percent powered for a 5-point difference between FDA-approved drugs specifically for PDP, but there are treatment and placebo. generic atypical antipsychotics that are used off-label, with SAPS is the recommended endpoint to describe psy- limited efficacy. The most efficacious of those is clozapine, chosis in Parkinson's disease, a condition involving halluci- but it carries several well-known side effects. nations and delusions that affects about 40 percent of the Acadia, which earned $30 million up front from Biovail estimated 1.5 million U.S. patients with Parkinson’s, Hack- in its May licensing deal, had about $66.2 million in the sell said. bank as of June 30. The firm said it expects to end this year All patients enrolled in the study remained on their sta- with more than $40 million, enough to carry it into the first ble doses of dopamine drugs, and the study met its sec- half of 201 1. ondary endpoint of motoric tolerability, as measured by the Beyond pimavanserin, it has an ongoing collaboration Unified Parkinson's’ Disease Rating Scale, showing that it with Allergan Inc., of Irvine, Calif., for a Phase II-stage drug did not worsen motor symptoms associated with the dis- for chronic pain and a Phase I program in glaucoma. And ease. Pimavanserin also was found to be safe and well tol- it’s getting ready to start advancing additional programs erated. from its internal pipeline, starting with ACP-106, a selective Despite the disappointing data, Acadia remains confi- 5-HT2A inverse agonist that is in investigational new drug dent that the drug has “considerable potential” in PDP, application-track development. ■ 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. F I N A N C I N G S RO U N D U P That trial is designed with the same endpoints but is testing 10-mg and 20-mg doses of pimavanserin. • Alizé Pharma, of Lyon, France, has raised €3 million But a success in that case still won’t give Acadia a clear (US$4.27 million) of funding. A new investor, SHAM, an win. insurance company, led the round. Existing investors, “We do believe we need two pivotal trials” before it can Octalfa SAS and CEMA Inc. also participated. Including this submit a regulatory filing, Hacksell said. new round, Alizé Pharma Group has raised €4.8 million That means Acadia will have to consult with partner from those three investors since its creation in 2007. The Biovail Corp. funds raised will allow Alizé Pharma to finalize the opti- Though Acadia funded both Phase III studies, terms of mization and formulation of a first drug candidate for AZP- the May licensing deal call for Toronto-based Biovail to pick 01 and to file a clinical trial application to undertake the up the tab for further development and commercialization clinical development of its Asparec product. activities involving pimavanserin in PDP and any other neu- • Immunomedics Inc., of Morris Plains, N.J., filed rological and psychiatric indications the companies decide papers with the SEC saying it planned to raise $151. 1 million to pursue. by selling stock and warrants. The company said it may As Piper Jaffray analyst Edward Tenthoff put it in a issue any combination of up to 20 million shares of com- research note: “Without funding from Biovail, we believe mon stock and 3 million warrants. Last week the com- pimavanserin development will be halted once studies in pany’s shares jumped 277 percent when epratuzumab had PDP are complete.” a 24.9 percent treatment advantage over placebo in But even if the second PDP study fails, Hacksell said the patients with systemic lupus erythematosus in a Phase IIb drug has shown promise in other indications, including 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
  • WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 6 OF 7 Icagen develop sodium ion channel modulators for pain, one of Continued from page 1 the pharma giant’s stated high-priority therapeutic areas. renewal that would extend for another year,” he said. (See BioWorld Today, Aug. 15, 2007.) As of June 30, Icagen had about $24 million in cash and Outside of oncology, pain is considered by some as cash equivalents, enough to operate “for at least the next 12 Pfizer’s biggest zone of interest. The firm “doesn’t jump to months,” the company said in its quarterly financial filing, mind when you think of the major asthma companies,” Katz but will need to raise money to operate beyond that time. conceded, but said he “would be surprised if they didn’t Shares of Icagen (NASDAQ:ICGN) closed Tuesday at have ongoing efforts in inflammatory indications, if not $1.43, up 47 cents, or 49 percent, on word that the KCa3. 1 asthma” itself. channel blocker significantly reduced asthma over placebo Other large and “modest-sized” would-be partners in patients given an inhaled allergen. have expressed an interest in senicapoc, Katz told BioWorld Senicapoc fizzled in Phase III against sickle cell anemia Today. “I’m not concerned that pharma is shying away from two years ago, with Icagen stopping the trial after a moni- asthma,” he added, especially since the drug – unlike most toring board found the study unlikely to yield positive data. existing therapies – is orally delivered. “We had efficacy in terms of many of the secondary Partnering likely will wait until the next Phase II data endpoints, but didn’t hit the primary endpoint, the control become available and maybe later, when the “multi-thou- of crises,” Katz said, adding that senicapoc was “clearly sand-patient” Phase III asthma studies require deeper pock- demonstrating biologic activity.” ets, Katz said. ■ 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. O T H E R N E W S T O N O T E “In terms of effects on white blood cells [in immune disor- ders], the channel becomes very important in controlling the influx of calcium, even though it’s a potassium chan- • Affitech A/S, of Copenhagen, Denmark, appointed nel,” Katz said. Robert Burns as CEO and Alexander Duncan as senior vice Results in the 34-patient, UK asthma trial were deter- president of research and development. Burns was previ- mined by the drop in FEV1 , the volume of air that can be ously CEO of Celldex Therapeutics Inc., while Duncan hails forcibly exhaled in one second. Improvement in the aver- from AstraZeneca plc. The appointments follow Affitech’s age FEV1 decline was 29 percent (p = 0.06). In the maximum merger with Pharmexa A/S. (See BioWorld Today, March 5, decline, the number was 18 percent (p = 0. 15), the area 2009.) under the curve amounted to 28 percent (p = 0.08) of FEV1. • Applied NeuroSolutions Inc., of Vernon Hills, Ill., A secondary endpoint, the fraction of exhaled nitric appointed Craig S. Taylor as its president, CEO and a mem- oxide (a measure of airway inflammation, typically high in ber of its board. Previously a partner at Adams Street Part- asthmatic patients), dropped by 24 percent (p = 0. 10) ners LLC, Taylor served as associate director of business among drug patients compared to those on placebo. development for G.D. Searle (now part of Pfizer) and was a Another secondary endpoint – early asthmatic response – senior research biochemist at Abbott Laboratories. didn’t change, a finding in line with preclinical results. • Basilea Pharmaceutica Ltd., of Basel, Switzerland, Senicapoc was well tolerated, with no serious adverse said that the FDA considers the response submitted by the events. Icagen plans to offer full results at an upcoming sponsor Johnson & Johnson Pharmaceutical Research scientific conference. & Development LLC, of Raritan, N.J., as a complete, class 2 Meanwhile, the company has finished enrollment in a response. The submission addresses the FDA’s November 69-patient U.S. trial testing senicapoc in exercise-induced 2008 letter concerning the ceftobiprole new drug applica- asthma, with the same FEV1 endpoint. Data are expected in tion for complicated skin and skin structure infections. the fourth quarter of this year. Ceftobiprole is being developed through an exclusive world- In June, Icagen signed J.P. Morgan to help figure out wide collaboration between Basilea Pharmaceutica Inter- next steps. Senicapoc’s 2007 failure in sickle cell was fol- national Ltd., also of Basel, and Cilag GmbH International, a lowed by the loss of the related deals with McNeil Con- J&J company. (See BioWorld Today, Dec. 1, 2008.) sumer & Specialty Pharmaceuticals, a subsidiary of New • Bristol-Myers Squibb Co., of New York, completed Brunswick, N.J.-based Johnson & Johnson. its tender offer for shares of Medarex Inc., of Princeton, Shortly afterward, Bristol-Myers Squibb Co., of New N.J. As of the expiration of the offering period, Medarex York, backed away, deciding not to pursue development of shareholders tendered about 120.4 million shares, which, the lead compound discovered in a long-standing deal tar- along with the 2.9 million shares owned by BMS since geting atrial fibrillation. 2005, represented about 90.7 percent of the shares out- Better news followed in the same year, when New York- standing. The two firms agreed to the $2.4 billion acquisi- based Pfizer inked a potential $1 billion-plus deal to tion 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
  • WEDNESDAY, SEPTEMBER 2, 2009 BIOWORLD® TODAY PAGE 7 OF 7 said it now anticipates that its total operating expenses O T H E R N E W S T O N O T E for 2009 will range between $78 million and $83 million, vs. its previous guidance of operating expenses ranging between $83 million and $88 million. It will continue to • Cellectis SA, of Romainville, France, and Mon- maintain sales coverage of the physicians with the highest santo Co., of St. Louis, entered into a nonexclusive potential to prescribe Moxatag (extended-release amoxi- research and commercial license agreement for broad use cillin) Tablets, 775 mg. of Celectis’ meganuclease technology in plants. Cellectis • Neurobiological Technologies Inc., of Emeryville, will receive an up-front payment of €3 million (US$ 4.2 Calif., has decided to liquidate the company’s assets and to million), Monsanto will make an equity investment of €1 dissolve the company. The company intends to distribute million to allow Cellectis to scale the technology for agri- the majority of its available cash to its stockholders. A culture. Cellectis also will be eligible to receive fees for stockholders meeting will be held to vote on the plan. As of the development of each meganuclease, success-based June 30, the company reported having cash, cash equiva- milestones and may receive royalties on certain traits lents and short-term investments of $24 million, commercialized by Monsanto. Further financial details • Nuvo Research Inc., of Missisauga, Ontario, has were not disclosed. entered into a cooperative drug development project with • Champions Biotechnology Inc., of Baltimore, the Fraunhofer Institute for Cell Therapy and Immunology entered a deal with an undisclosed large biotech company IZI in Leipzig, Germany, for the preclinical and clinical to evaluate an oncology therapeutic using Champion’ Bio- development of WF10 as a potential treatment for allergic merk Tumorgraft platform, which is designed to enable rhinitis. The Development Bank of Saxony in Germany will identification of the most promising development path for provide financial support for the project, which will be con- a compound and also identify potential gene pathways of ducted in Leipzig through Nuvo Research GmbH, a Nuvo response and resistance, as well as prognostic molecular subsidiary. biomarkers. Terms were not disclosed. • Selexis SA, of Geneva, and NKT Therapeutics Inc., • Clinical Data Inc., of Newton, Mass., sold the of Waltham, Mass., entered a research services deal under equipment and property associated with its wholly which Selexis will screen antibody variants and generate owned subsidiary – Germantown, Md.-based Avalon Phar- Chinese hamster ovary-based production cell lines using maceuticals Inc. – to Blacksburg, Va.-based Intrexon its Integra-D2M Corp. for $1.5 million cash. Clinical Data retains Avalon’s • Shire plc, of Basingstoke, UK, said its final two Phase oncology drug candidates, intellectual property and bio- III trials for enzyme replacement therapy velaglucerase alfa marker discovery platform, while Intrexon gets access to met their endpoints in Type I Gaucher’s disease and the a bioassay platform to complement its transgene engi- company’s rolling new drug application at the FDA has neering platform. been completed. (See BioWorld Today, Aug. 4, 2009.) • Medivir AB, of Stockholm, Sweden, selected MIV-71 1 • SkyePharma plc, of London, said that additional as a candidate drug for bone diseases. MIV-71 1 is a small- clinical work will be required to provide more data on dos- molecule protease cathepsin K inhibitor distinguishable ing for its lead development product, Flutiform (fluticasone from MIV-710, and both drugs are being prioritized over propionate/formoterol fumarate), for persistent asthma in MIV-701 for osteoporosis, osteoarthritis, rheumatoid arthri- patients 12 years of age and older. The need for additional tis and metastatic bone disease. clinical work was confirmed in a meeting with the FDA, the • Metabasis Therapeutics Inc., of San Diego, said its company said. Due to the additional work, the board president, CEO and chief scientific officer, Mark Erion, believes that it is unlikely that Flutiform will be approved in resigned as an officer, effective Oct. 31. Erion, who also the U.S. before the second half of 201 1. In the meantime, the resigned his position on the board, will consult with the FDA’s review of product is continuing. company after that date on matters related to the licensing • Stemedica Cell Technologies Inc., of San Diego, or sale of its pipeline and advanced discovery programs or has been granted a license by the California Food & Drug other strategic alternatives. Erion is joining Whitehouse Branch to manufacture stem cells for human clinical trials. Station, N.J.-based Merck & Co. Inc. as vice president and The license recognizes Stemedica as being compliant with worldwide basis franchise head of diabetes and obesity. California law and the applicable provisions of the Code of David Hale, Metabasis’ chairman, was appointed executive Federal Regulations. chairman. • Zealand Pharma AS, of Glostrup, Denmark, said it is • MiddleBrook Pharmaceuticals Inc., of Westlake, moving its new generation drug candidate for Type II dia- Texas, has cut its sales managers and field sales represen- betes into preclinical development. The dual glucagon-GLP- tatives by 25 percent and reduced corporate staff by 1 agonist, ZP2929, has the potential to significantly about 20 percent. As a result, the firm expects to achieve decrease body weight and reduce the risk of diabetes- approximately $15 million in annual savings. The company related complications, the company said. 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
  • New from the Publishers of BioWorld® Today Market-Leading Biotechnology Drugs 2009: Blockbuster Dynamics in an Ailing Economy Giving you the insight needed to assess, and access, the dynamics that facilitate success in the biotech market. The new BioWorld's Market-Leading Biotechnology Drugs 2009 concisely delivers precise acumen on the history, trends, value and projection of the ever-increasingly imperative biotech drug therapeutics market that has emerged to the forefront of government initiatives, business agendas and public concern and is impacting many aspects of industry, economics and life. Viable business intelligence in this report includes: ✧ Which new technology is ordained to lead the fight against disease in the twenty-first century? ✧ Which drugmakers are projected to comprise the new list of large-cap BIOW ORL ® D biotechs in 2015? M BIOT ARKET-L ECH EAD ✧ Which biotech drug has the potential to be the first-ever drug to BLO CKB NOL OGY ING UST DRU generate $10 billion in annual revenue? IN A ER D NA GS: ILIN YNA GE MIC ✧ What is the prevailing trend that will define the biotech drug CON OM Y S market in the next decade? ✧ Which Big Biotech is undergoing FOB competition before its patents even expire, putting $4 billion in sales at risk? ✧ What are the 41 drugs that accounted for $35 billion in 2008 revenuethat will lose patent exclusivity through 2012? ✧ Which drugmakers are expected to comprise the new list of large-cap biotechs in 2015? ✧ And much more! Drugs are winning the containment battle, but disease is winning the war and the dynamics are constantly changing. Keep up-to-date with BioWorld's Market-Leading Biotechnology Drugs 2009. This report is focused, nimble, efficient, meticulous, precise and value-packed. Order BioWorld's Market-Leading Biotechnology Drugs 2009 risk-free for 30 days for only $199 (plus $17.95 s&h). Call us at 1-800-688-2421 or 1-404-262-5476 or go to www.bioworld.com/biodrugs. You may review this product risk-free for 30 days. If you do not wish to keep the product, please return the product, together with the invoice marked "Cancel" to AHC at 4401 Freidrich Lane, Suite 205 Austin, TX 78744-1849. Shipping and handling charges for returned items are the responsibility of the customer. If you have any questions or concerns you may contact us at 800-688-2421 or by e- mailing customerservice@ahcmedia.com. ORDER TODAY! Call: 1-800-688-2421 or 1-404-262-5476 and mention priority code S09447. Online: Go to www.bioworld.com/biodrugs E-mail: orders@bioworld.com