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CNDO 11-12-11 BioWorld Insight

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Coronado Biosciences featured in a story entitled "Form 10 as IPO Alternative? Coronado Would Do It Again." December 12, 2011

Coronado Biosciences featured in a story entitled "Form 10 as IPO Alternative? Coronado Would Do It Again." December 12, 2011

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  • 1. ®MONDAY VOL.19, NO. 50 Your Weekly Source for Market and Financial ViewsDEC. 12, 2011 PAGE 1 OF 32In Geron’s Wake Money Raised By BiotechDeals, Data Spark Stem Cell In 2011 Vs. 2010Interest, But Hurdles Remain Jan. 1 – Dec. 9, 2011: $22,777.24MBy Trista Morrison Jan. 1 – Dec. 9, 2010: $18,987.47MEditor SAN DIEGO – Geron Inc.’s exit from the embryonic stem 11,000cell field last month shocked many in the biotech industry 10,000 9,792– but not the regenerative medicine experts attending the 8,634 9,000recent 2011 Stem Cell Meeting on the Mesa. 7,863 Some attendees muttered that Geron had never been 8,000able to effectively balance the conflicting risk-reward 7,000 6,700profiles of its stem cell business and its cancer business. Millions 6,000Others whispered that the writing had been on the wallsince the departure of longtime CEO Thomas Okarma in 5,000 4,351 4,425February. Still others wondered if perhaps the preliminary 4,000data from Geron’s Phase I spinal cord injury trial – theworld’s first of a human embryonic stem cell therapy – 3,000weren’t as good as had been hoped. 2,000 Geron has maintained the latter isn’t the case – the 1,000company blamed its withdrawal from the stem cell spaceon capital scarcity and uncertain economic conditions. (See 0BioWorld Today, Nov. 16, 2011 .) Public Public/ Private Of f erings Other* Biotechs Regardless of the reason, regenerative medicine * Includes financings of public biotech firms with the exceptions ofinsiders don’t view Geron’s move as an indication that public offerings and certain investments from corporate partners.the stem cell industry is on shaky ground – at least, notany shakier than it’s ever been. If anything, data and deal- “has been sorted out,” he said, and advances in researchflow indicate increasing interest in stem cells, although tools have improved the consistency of embryonic stemmaintaining that momentum will require creativity on cell work as well. On the embryonic side, Geron also blazedseveral fronts. a regulatory trail with the FDA by getting into the clinic. “The greatest obstacle now is to deliver compellingKicking into Gear clinical proof of concept data,” said Gil Van Bokkelen, CEO of Mahendra Rao, director of the National Institutes of Athersys Inc. and chairman of the Alliance for RegenerativeHealth’s Center for Regenerative Medicine, noted that the Medicine. “That’s what investors are looking for; that’sstem cell field has been significantly derisked over the pastfew years. Most of the technical risk with adult stem cells See Stem Cells, page 4 BIOTECH SHORT SELLING UP SLIGHTLY, BUT NOT ALL BAD ...................... 2 THIS WEEK IN FORM 10 AS IPO ALTERNATIVE? CORONADO WOULD DO IT AGAIN ........ 3 MONEY RAISED BY BIOTECH IN 2011 ................................................... 4 BIOWORLD WEEK IN REVIEW ................................................................................ 5 WORD ON THE STREET, WEEK IN WASHINGTON ..................................... 6 INSIGHT PHASE I, II & III CLINICAL TRIALS UPDATE: NOVEMBER 2011 .... 7, 11, 15 CONFERENCE DATA ...................................................................... 19-25 NON-U.S. CLINICAL TRIALS DATA; FDA ACTIONS ......................... 26, 28 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com
  • 2. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 2 OF 32A Difference of Opinion Despite continued volatility in the market, theBiotech Short Selling Up measurements of short activity have returned to normal levels.Slightly, But Not All Bad There has, however, been a slight widening of the shortBy Brian Orelli interest as a percent of equity over the last two and a halfBioWorld Insight Contributing Writer years between biopharma and Nasdaq stocks, with the Short sellers are generally considered blasphemers by biopharma stocks closer to 1 .5 percentage points higherbiotech firms. And for good reason; short sellers borrow than the Nasdaq stocks, up from the previously mentionedshares in order to sell them, which puts negative pressure on mean of about 1 percentage point. Grimaldi offered up athe stock. The only way shorts make money is if shares go couple of potential explanations: “It may mean that they’redown. betting against the sector or there may just be a larger But biotechs don’t have much to complain about, according number of companies where it would be profitable to putto data compiled by BMO Capital Markets. Short sellers are on a short position.”only slightly more active in the biopharma sector than they Not every short position is made by an investor whoare for the entire Nasdaq. Looking at the short interest as a believes the shares are overvalued. Short sales are usedpercent of equity float since the beginning of 2007, the 302 to hedge long positions in either the individual stock, thepublicly traded biopharmas used in the study had a mean of sector, or the entire investor’s portfolio. They can also beabout 6.6 percent of their available shares sold short, which is used as a tax deferral strategy.only about a percentage point higher than the short interest Shorts also increase liquidity of shares, which can befor all the Nasdaq stocks. helpful for biotechs. Higher liquidity, for instance, helps Considering the large number of binary events in biotech– data read-outs, FDA decisions, etc. – Annette Grimaldi, See Shorts, page 6managing director at BMO, thought the absolute differencemight have been larger. One possible reason is that the binaryevents also detract short investors because the losses on ashort position are theoretically limitless, unlike a long positionwhere the most an investor can lose is the initial investment. BioWorld is now The means of the short interest ratio – dividing the shortinterest position by the average daily trading volume – were on Twitter!nearly identical at 7.8 and 7.6 for Nasdaq and biopharmastocks, respectively. In other words, it would take almost eightdays for the shorts to cover all their outstanding short sales at Stay Connected,the average trading volume. Follow Us on Twitter! Both the short interest as a percent of equity float andthe short interest ratio spiked in the middle of 2008 duringthe peak of the recession. That’s not surprising as both www.twitter.com/bioworldmeasurements describe how safe it is to put on a shortposition. As the numbers increase, it becomes harder tounwind the short. BIOWORLD® INSIGHT (ISSN 1541-0579) is published every Monday by AHC Media, 3525 Piedmont Road, Building Six, Suite 400, Atlanta, GA 30305. Opinions expressed are not necessarily those of this publication. Mention of products or services does not con- stitute endorsement. BIOWORLD® and BIOWORLD® INSIGHT are trademarks of AHC Media, a division of Thompson SUBSCRIBER INFORMATION Media Group LLC. Copyright © 2011 AHC Media. All Rights Reserved. No part of this publication may be repro- Please call (800) 888-3912 to duced without the written consent of AHC Media. (GST Registration Number R128870672) subscribe or if you have fax trans- ATLANTA NEWSROOM: Executive Editor: Lynn Yoffee. mission problems. Outside U.S. and Managing Editor: Jennifer Boggs. Canada, call (404) 262-5476. Our customer service hours are 8:30 Editor: Trista Morrison. a.m. to 6:00 p.m. EST. Database Editor: Karen Pihl-Carey. BUSINESS OFFICE: Senior Vice President/Group Publisher: Donald R. Johnston. EDITORIAL Lynn Yoffee: (404) 262-5408 Senior Editor: Michael Harris. Trista Morrison: (858) 901-4785 Managing Editor: Amanda Lanier. Donald R. Johnston: (404) 262-5439 Product Marketing Manager: Sarah Cross. Internet: http://www.bioworld.com Account Representatives: Matt Hartzog, Chris Wiley. DISPLAY ADVERTISING: For ad rates and information, please call Stephen Vance at (404) 262-5511. or e-mail 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. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 3. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 3 OF 32Weighing Pros, Cons Unlike in an IPO, Coronado didn’t have investmentForm 10 as IPO Alternative? bankers trotting management around to meet with institutional investors and raise awareness. “With Form 10,Coronado Would Do It Again you essentially have to do it yourself,” Sandage explained,By Trista Morrison adding that he and chief operating officer Noah BeermanEditor have been “on the road nonstop” talking to investors and Four months ago, Coronado Biosciences Inc. embarked on analysts, trying to get the word out.a route to going public not often seen in the biotech industry: For now, trading volume among Coronado’s largelythe Form 10 pathway. (See BioWorld Today, July 20, 2011 .) retail base is low, but Sandage isn’t overly concerned, noting In essence, the Form 10 pathway offers an alternative to the that “IPOs don’t trade that much after the first week or twoinitial public offering (IPO) or reverse merger, seeking to avoid either.” And although Coronado’s price has fallen, Sandagethe market volatility and high transaction costs of the former is encouraged by the fact that the firm was recently pickedas well as the trading restrictions and investor baggage of the up by Roth Capital Partners with a buy rating and a $27 pricelatter. A private firm looking to use the pathway to go public target. He’s also optimistic that more analyst coverage andsimply files a Form 10 with the SEC to become a public reporting interest from institutional investors will come once the firmcompany, files an S-1 to register its shares, and applies to trade transfers to Nasdaq, a process that is already underway.those shares on a market. (See BioWorld Insight, July 25, 2011 .) Beerman also believes Coronado’s stock price Coronado got started down the Form 10 road because will benefit from the achievement of some near-termsuch filings are required for private companies with 500 or milestones. The company recently filed an investigationalmore stockholders of record. The rule most often affects large new drug application for lead program CNDO-201 , pigprivate tech firms like Facebook, but Coronado’s angel-backed whipworm eggs, and a Phase I trial is underway. Safetybusiness model made it one of the rare few private biotechs data are expected in the first quarter of 2012, and a Phase IIto hit the stockholder threshold. Although a bill is pending in trial for Chron’s disease is expected to start in the secondthe House that may raise that threshold, under current law, quarter. Meanwhile Coronado’s second program, whichCoronado had to become a reporting company. involves ex-vivo activation of NK cells, is poised to start a Coronado did not, however, have to start publicly Phase I/II trial in acute myeloid leukemia early next year.trading its shares – but the company was interested in It remains to be seen whether or not other biotechsaccessing the public markets. President and CEO Bobby follow in Coronado’s footsteps and use the Form 10Sandage said Coronado had considered a reverse merger pathway to go public. They may soon have yet anotherand even went as far as to identify a suitable shell company IPO alternative: a bill that has passed the House and isto merge into, but new restrictions from the SEC made that now in the Senate would raise the Regulation A exemptionoption less appealing. Meanwhile, although five biotechs to $50 million, providing privately held biotechs with thehave jumped into the IPO queue in the last few months, the opportunity to raise money and start trading their shareswindow remains far from what most would consider open. without filing a Form 10 to become a public reporting(See BioWorld Today, Nov. 11 , 2011 .) company. (See BioWorld Insight, Nov. 21 , 2011 .)  Hence Coronado opted for the Form 10 route. Sandage saidthe process is not well understood and “even with experiencedSEC lawyers we were sort of figuring things out as we wentalong,” but he believes it was the right decision and would doit again with future start-ups. Coronado debuted on the OTCQB last month at $11 per ADVERTISEshare. That price was market-driven, determined not by ateam of investment bankers negotiating with institutionalinvestors, but simply by what a buyer was willing to pay and HEREa seller was willing to accept. Interestingly, Coronado’s pricewas higher than that of three-quarters of the biotechs to price ...and reach high-levelIPOs this year, even though the Burlington, Mass.-based firm is biotechnology professionals every week!barely in the clinic. Since its pricing, however, Coronado’s shares For advertising opportunities in(OTCQB:CNDO) have dropped 34 percent to $7.25, as of mid BioWorld Insight, please contactlast week. That’s not necessarily unusual for a newly publicbiotech – several members of this year’s IPO class have Stephen Vance at (404) 262-5511fallen just as hard, although as a group they are down about or stephen.vance@ahcmedia.com8 percent. The drop does, however, illustrate some of thechallenges of taking the Form 10 route. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 4. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 4 OF 32 Another sign of momentum can be seen in private Money Raised By Biotech: financing for stem cell and regenerative medicine firms. Jan. 1 - Dec. 9, 2011 Four such firms have completed private offerings this year, according to data from Roth Capital Partners, and three of them brought in at least $30 million. Additionally, 19% 43% three firms in the field – Argos Therapeutics Inc., TVAX ($4,351M) ($9,792M) Biomedical Inc. and Verastem Inc. – have filed to go public, and Advanced BioHealing was in the initial public offering queue as well before its acquisition. Not Out of the Woods So is the financial situation for regenerative medicine companies really that much worse than for biotech as a whole, as Geron’s retraction indicated? It’s somewhat hard to believe, sitting in the brand-spanking-new $127 million laboratories of the Sanford Consortium for Regenerative Medicine, listening to so many examples of progress and success. But according to Geoff Whitehead, principal with Flagship Ventures, the answer is yes. The outlook for stem cell firms is not quite as dire on 38% Public Offerings 43% ($9,792M) the private equity side. Venture capitalists are looking ($8,634M) for transformative ideas, large markets and compelling Public/Other 38% ($8,634M) entrepreneurs, so the fact that a lot of stem cell work is early Private Biotechs 19% ($4,351M) stage and hard to value “doesn’t really factor into things,” Whitehead said. Flagship has three regenerative medicine firms in its portfolio. But on the other hand, Whitehead noted that “earlyStem Cells stage VCs like to invest in pioneers,” and the stem cell field’sContinued from page 1 pioneers don’t have the best track record thus far. He thenwhat pharma is looking for. Once that data gets delivered, it ticked off a dozen reasons why it would be easier for thewill change the mindset in a big way.” average VC to say “no” to a stem cell investment than “yes.” In the adult stem cell space, data is starting to trickle Stem cell start-ups do have the benefit of alternativeout. Morrie Ruffin, managing director of the Alliance for options like grant funding – the California Institute ofRegenerative Medicine, noted that while the media were busy Regenerative Medicine (CIRM) has been doling out moneyreporting on Geron, many missed the fact that Mesoblast for several years now. But Edward Lanphier, presidentLtd.’s allogeneic adult stem cell product Revascor significantly and CEO of Sangamo BioSciences Inc., noted that the onlyreduced cardiovascular deaths in a randomized, placebo- companies able to go public these days are in Phase III, andcontrolled Phase II trial, and Aastrom Biosciences Inc.’s “is CIRM going to take someone to Phase III? I don’t think so.autologous expanded cell therapy ixmyelocel-T significantly There’s a need for venture capital. I’m not optimistic that it’sreduced treatment failures in a randomized, placebo- going to be met, but there’s a need.”controlled Phase II critical limb ischemia trial. (See BioWorld Even if a stem cell company does get public, thatToday, Nov. 16, 2011 .) doesn’t mean their fortunes will improve. Lisa Walters- There’s been good news on the partnering front as well. Hoffert, managing director with Roth, presented an analysisEvery panel at the conference paid homage to Mesoblast’s showing that just nine public stem cell and regenerative$350 million stem cell deal with Cephalon Inc. (now part of medicine firms have raised money this year – less than $140Teva Pharmaceutical Industries Ltd.), which carried a whopping million altogether, and with a hefty average 67.5 percent$130 million up front, as well as to Shire plc’s $750 million buy- warrant coverage. Since their offerings, the firms have seenout of regenerative medicine firm Advanced BioHealing Inc. their shares fall an average of 24 percent, and only one(See BioWorld Today, May 19, 2011, and Dec. 9, 2010.) company – StemCells Inc. – is in the green. “Things move forward at a certain rate and then kick into Further, despite the oft-mentioned Mesoblast andgear,” said Robert Preti, president and chief scientific officer Advanced BioHealing deals, business development in theof Progenitor Cell Therapy LLC, which itself was recently the space has yet to catch fire.subject of some business development activity, having been “We spend a lot of time talking to pharma,” saidacquired by NeoStem Inc. NeoStem also acquired stem cell firm Sangamo’s Lanphier. “As it relates to cell therapy, there isAmorcyte Inc. earlier this year. (See BioWorld Today, July 18, 2011 .) See Stem Cells, page 5 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 5. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 5 OF 32 WEEK IN REVIEW Financings Dendreon Corp. raised $125 million by monetizing its share of royalties on Victrelis (boceprevir). EMBL Ventures GmbH closed a $53.5 million fund for German life science companies. Forbion Capital Partners closed its second FCF 1 Co-Investment Fund at about $50 million. Gilead Sciences Inc. priced $3.7 billion worth of senior unsecured notes in a public offering. Deals Biogen Idec Inc. entered a $300 million joint venture with Samsung for biosimilars. Medicis Pharmaceutical Corp. acquired the assets of Graceway Pharmaceuticals LLC for $455 million. Molecular Partners AG signed an immunology discovery deal with Janssen Biotech Inc. Momenta Pharmaceuticals Inc. acquired Virdante Pharmaceuticals Inc.’s protein sialylation programs. . . . And More See BioWorld Today for coverage of the San Antonio Breast Cancer Symposium. Affymax Inc.’s peginesatide got a thumbs up from an FDA advisory committee. Curemark LLC reported positive Phase III data with CM-AT in children with autism. Genentech Inc./Roche AG’s pertuzumab had good Phase III breast cancer data. Theratechnologies Inc. is halting its chronic obstructive pulmonary disease muscle wasting program and downsizing by 60 percent. Unigene Laboratories Inc.’s parathyroid hormone was dropped by partner GlaxoSmithKline plc.Stem Cells “high-risk work” that neither grant-funded researchers norContinued from page 4 companies can stomach. Nonprofits should also focus on the translational work that academics aren’t interested ininterest, but there is a higher bar. Whether it’s safety, efficacy, because it won’t result in a published paper – the “work thatcost of goods analysis . . . you’ve got to paddle your own canoe is repetitive but essential to derisk the program to a pointa lot longer.” where investors or pharma can pick it up,” she said. Matthias Steger, global head of research and technology Meanwhile, history (and Geron) has proven thepartnering with Roche AG, provided further perspective. traditional drug development business model isn’t aWhile he refuted the notion that big pharma is not involved particularly good fit for stem cell firms. Whether public orin stem cells, pointing to Roche’s own significant investment, private, the firms that have managed to stay in businesshe cautioned that big pharma “can just not afford running are those that have, more often than not, found alternativeinto big failures. We have to sort out the science. It would be a sources of funding.mistake to rush. I think we are making progress, but we have Sangamo, for example, has outlicensed its zinc fingerto be a little bit patient.” protein technology in non-core fields like agriculture and research tools. Cytori Therapeutics Inc. is bringing inJust Keep Paddling revenue by selling its Celution adult stem cell processing The trick will be for stem cell firms to figure out how they system in Europe, where it has received a CE Mark, while itcan afford to patiently wait for the catalyst that captures big navigates the U.S. regulatory path. Organovo Inc. uses itspharma’s attention, or the proof-of-concept data that drive platform to create disease models for partners, as well aspublic market interest. in its own drug development. Neostem generates revenues The experts at the conference said it will require an from a cell therapy manufacturing business, a stem cellunprecedented level of public-private partnership. “It can’t bank and several initiatives in China, which help supportbe industry alone, or academics alone, or government alone the cost of developing its cell therapy pipeline.– there must be collaboration,” said NIH’s Rao. He added The approaches are unconventional, but Greg Lucier,that there are far more stem cell trials being conducted by chairman and CEO of Life Technologies Corp., thinksacademics than by industry, but the former have “no idea how that’s what the regenerative medicine industry needs. “Ito take it to the next level.” That’s where government needs to do believe this is an area where we should be pushing thefocus its efforts, he said. frontiers,” he said. “We’re positive, we’re confident and we Susan Solomon, CEO of the New York Stem Cell are investing in this area. The horizon we see is near termFoundation, added that nonprofits can play a role by doing the and incredibly bright.”  To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 6. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 6 OF 32 WORD ON THE STREET “If you’re a great company with great promise, there’s money out there.” – Geoff Whitehead, principal with Flagship Ventures, on funding difficulties for stem cell firms “The world of pharma is waking up [to the fact] that this is going to be the next frontier.” – Greg Lucier, chairman and CEO of Life Technologies Corp., on stem cells “You want to have Track 1 , 2, 3, 4 and 5 – if you only have Track 1 and Track 5, no one wants to give it a shot.” – William Hicks, partner with Mintz Levin, on the need to have multiple avenues to going public “The hurdle rates for companies in development are being set higher than they have been in the past. Having said that, we love being in this industry.” – Anthony Marucci, president and CEO, Celldex Therapeutics Inc. WEEK IN WASHINGTON The FDA’s latest proposed user fee structure for biosimilars will charge 10 percent of the new drug application fee, plus a development fee. The U.S. Patent and Trademark Office and China’s State Intellectual Property Office will expedite their patent examinations, which should allow applicants to obtain corresponding patents faster in each country.Shorts While shorts might be best ignored from an industryContinued from page 2 perspective, Grimaldi thinks individual firms shouldfirms sell more shares in at-the-market (ATM) offerings, a probably consider why investors are shorting the shares.public offering alternative that has slowly gained traction over “Especially large short interest is a signal that differs fromthe past few years. Inhibitex Inc. raised $20 million through what management is saying. For that particular company,an ATM last month. (See BioWorld Insight, Sept. 7, 2009.) they should look to see if there are any underlying messages And short sellers can actually have a positive effect on in that position.”shares if a company produces positive news. A short squeeze China Biologic Products Inc., Cumberland Pharma-occurs as the short sellers cover their sales by buying back ceuticals Inc., MannKind Corp., BioTime Inc., and Oncolyticsshares, amplifying the upward movement of the stock in Biotechnology Inc. currently top the list of companies withresponse to the good news. the highest short interest.  READING A HAND-ME-DOWN? If you’re reading this issue, but not a subscriber, please call us to start receiving your very own copy. Or, for even more convenience, subscribe to our online version or have it e-mailed directly to you! Authorized copying privileges and multi-user access for your entire office are available at discounted rates. Simply call 1-800-688-2421 or 1-404-262-5476 for more information! To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 7. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 7 OF 32 FDA Approvals In November Company Drug Indication Bristol-Myers Squibb Co. Erbitux Head and neck cancer EUSA Pharma Inc. Erwinase Acute lymphoblastic leukemia Incyte Corp. Jakafi Myelofibrosis IntelGenx Corp. CPI-300 Major depressive disorder Millennium Velcade Updated label for multiple myeloma Pacira Pharmaceuticals Inc. Exparel Postsurgical pain relief Regeneron Pharmaceuticals Inc. Eylea Wet age-related macular degeneration Spectrum Pharmaceuticals Inc. Zevalin Removed bioscan requirment for non-Hodgkin’s lymphoma Transcept Pharmaceutical Inc. Intermezzo Insomnia Phase I Clinical Trials Update: November 2011 Company Product Description Indication Status (Date)# (Location) CANCER Bavarian Nordic CV-301 Off-the-shelf immu- Metastatic breast A trial with 26 heavily pretreated patients A/S (Kvistgard, notherapy or ovarian cancer who received monthly vaccinations Denmark) showed a median time to progression of 2.5 months in the breast cancer group, and median overall survival was 13.7 months (11/10) Celldex CDX-1127 A co-stimulatory Malignant solid Started a Phase I study (11/9) Therapeutics Inc. molecule on T cells tumors or hema- (Needham, Mass.) tologic cancers CureVac GmbH CV9201 mRNA-based can- Non-small-cell Phase I/IIa data showed the drug was well (Tuebingen, cer vaccine lung cancer tolerated and biologically active (11/8) Germany) CytRx Corp. (Los INNO-206 A tumor-targeting Sarcoma Phase Ib/II trial data determined a maxi- Angeles) conjugate of doxo- mum tolerated dose of the drug deliver- rubicin ing doxorubicin at the equivalent of three and a half times standard doxorubicin doses (11/1) Hutchison Epitnib An orally active Cancer Started the first-in-human Phase I trial MediPharma Ltd. (HMPL-813) small molecule (11/7) (London) inhibitor targeting the epidermal growth factor receptor Infinity IPI-145 Oral inhibitor of Advanced Started two Phase I trials (11/1) Pharmaceuticals phosphinositide-3- hematologic Inc. (Cambridge, kinase delta and malignancies Mass.) gamma Lentigen Corp. LG631 Gene therapy Cancer Started a Phase I trial (11/15) (Gaithersburg, Md.) Senesco SNA01-T Intravenous infusion Multiple Initiated patient dosing in its Phase Ib/IIa Technologies Inc. myeloma study (11/4) (Bridgewater, N.J.) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 8. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 8 OF 32 Company Product Description Indication Status (Date)# (Location) Silence Atu027 siRNA drug Solid tumors Phase I data showed 37% of patients had Therapeutics plc stable disease after treatment (11/9) (London) CARDIOVASCULAR Cardiome Vernakalant Oral therapy Atrial fibrillation Recently completed an additional multi- Pharma Corp. ple rising-dose Phase I study and data (Vancouver, British showed the drug was well tolerated (11/7) Columbia) and Merck & Co. Inc. (Whitehouse Station, N.J.) Nile Cenderitide Subcutaneous Chronic heart Phase I data showed that weight-based Therapeutics Inc. infusion failure doses achieved target PK levels when (San Mateo, Calif.) delivered by subcutaneous pump, and that it was well tolerated, bioavailable and reduced PK variability compared to a fixed-dose regimen (11/17) Pozen Inc. (Chapel PA32540 A combination of Gastrointestinal Phase I data showed it was associated Hill, N.C.) 325 mg aspirin and risk in patients with greater platelet inhibition when 40 mg immediate- who require dual dosed 10 hours apart from Plavix com- release omeprazole antiplatelet ther- pared to synchronous administration of apy and gastro- aspirin, clopidogrel and delayed-release protection omeprazole at day seven (11/15) CENTRAL NERVOUS SYSTEM Acadia AM-831 A small molecule Schizophrenia FDA cleared it to begin a Phase I trial (11/8) Pharmaceuticals that combines mus- Inc. (San Diego) carinic m1 partial agonism with dopa- mine D2 and sero- tonin 5-HT2A antagonism Anavex Life Anavex 2-73 The first of a new Alzheimer’s Completed a Phase I single ascending Sciences Corp. class of oral drugs disease dose trial of Anavex, showing it was well (Hoboken, N.J.) tolerated below the 55 mg to 60 mg dose with only mild adverse events (11/15) Cara CR845 A peptide-based Acute postopera- Started its first Phase I trial of an oral for- Therapeutics Inc. kappa opioid ago- tive pain mulation (11/23) (Shelton, Conn.) nist Neuralstem Inc. Stem cell Stem cell therapy Amyotrophic Dosed its first patient in the trial (11/28) (Rockville, Md.) therapy used in the cervical lateral sclerosis region of the spine NeuroDerm Ltd. ND0611 Administered sub- Parkinson’s Met all primary and secondary endpoints (Ness Ziona, Israel) cutaneously by a disease in a Phase I/II trial (11/10) dermal patch Probiodrug AG PQ912 A glutaminyl Alzheimer’s Phase I data demonstrated it was safe and (Halle, Germany) cyclase inhibitor disease well tolerated (11/15) Selecta SEL-068 A nicotine vaccine Smoking cessa- Started a Phase I trial (11/22) Biosciences Inc. tion and relapse (Watertown, Mass.) prevention To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 9. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 9 OF 32 Company Product Description Indication Status (Date)# (Location) DIABETES Akebia AKB-9778 A human protein Diabetic macular Started a Phase I trial (11/22) Therapeutics Inc. tyrosine phospha- edema and dia- (Cincinnati) tase beta inhibitor betic retinopathy Concert CTP-499 Controlled-release Diabetic Phase I data showed it was well tolerated Pharmaceuticals version; analogue nephropathy at single doses up to and including 1 ,800 Inc. (Lexington, of 1-([S]-5- mg (11/15) Mass.) hydroxyhexyl)-3,7- dimethylxanthine Convergence CNV2197944 A calcium channel Chronic pain Initiated a Phase I trial (11/22) Pharmaceuticals blocker Ltd. (Cambridge, UK) DARA DB959 A peroxisome pro- Type II diabetes Phase Ib data showed a safety profile BioSciences Inc. liferator activated comparable to placebo when given as (Raleigh, N.C.) receptor-delta/ once-daily doses for seven days and was gamma agonist safe and well tolerated throughout the 40-fold dose range tested (11/2) Isis ISIS-GCGRRx Antisense drugs Type II diabetes Started Phase I studies (11/3) Pharmaceuticals and ISIS- Inc. (Carlsbad, GCCRRx Calif.) PhaseBio Glymera A recombinant glu- Hyperglycemia Phase I/IIa data demonstrated statistically Pharmaceuticals cagon-like peptide-1 in Type II significant reductions in fasting glucose Inc. (Malvern, Pa.) analogue diabetes glycemic load following meal tolerance testing and in average daily glucose (11/16) INFECTION AVIR Green Hills deltaFLU Intranasal flu Influenza The vaccine was well tolerated and signif- Biotechnology vaccine icantly increased influenza-specific anti- AG (Vienna, bodies in a Phase I/II trial (11/8) Austria) Inhibitex Inc. INHX-189 Monotherapy; Hepatitis C virus Data showed INX-189 dosed at 100 mg (Atlanta) nucleotide poly- once daily for seven days in combination merase inhibitor with ribavirin resulted in a median HCV RNA reduction from baseline of -0.379 log10 (11/30) Inovio SynCon Avian influenza Avian flu A single intradermal electroporation Pharmaceuticals vaccine boost of its vaccine generated hemagglu- Inc. (Blue Bell, Pa.) tination inhibition titers against six differ- ent unmatched strains of H5N1 and it generated a fourfold or greater rise in HAI titers in 50% of boosted subjects in a Phase I study (11/18) Novacta NVB302 An oral treatment Hospital- Started dosing the first healthy volun- Biosystems Ltd. acquired teers in a Phase I trial (11/3) (London) Clostridium difficile infections To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 10. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 10 OF 32 Company Product Description Indication Status (Date)# (Location) MISCELLANEOUS Aeolus AEOL 10150 A broad-spectrum Acute radiation Pilot study results of AEOL 10150 and Pharmaceuticals catalytic antioxi- syndrome G-CSF drug Neupogen confirmed that it Inc. (Mission Viejo, dant designed to does not interefer with the positive Calif.) neutralize reactive effects of Neupogen on the hematopoiet- oxygen and nitro- ic syndrome (11/2) gen species Akebia AKB-6548 Oral hypoxia- Anemia associat- Phase Ib/IIa data showed it was well toler- Therapeutics Inc. inducible factor- ed with chronic ated and enhanced erythropoiesis (11/16) (Cincinnati) prolyl hydroxylase kidney disease inhibitor AlloCure Inc. AC607 A bone marrow- Acute kidney Phase I data showed it was safe and well (Burlington, Mass.) derived mesenchy- injury tolerated, and treated subjects experi- mal stem cell enced a lower incidence of AKI, reduced therapy length of hospital stay and reduced hos- pital readmission rates compared to a cohort of historical controls (11/14) Alnylam ALN-TTR01 An RNAi therapeu- TTR-mediated Phase I data showed statistically signifi- Pharmaceuticals tic targeting trans- amyloidosis cant reductions in serum TTR protein lev- Inc. (Cambridge, thyretin els in ATTR patients (11/22) Mass.) Bellicum CaspCIDe Technology used to Graft-vs.-host Results of the first clinical study showed Pharmaceuticals eliminate donor T disease it worked quickly and effectively without Inc. (Houston) cells compromising the function of the cell (11/4) Chimerix Inc. CMX001 A lipid-antiviral- Kidney disease Phase I/II data showed it was generally (Durham, N.C.) conjugate designed safe and well tolerated when adminis- to deliver cidofovir- tered to renal transplant and hematopoi- diphosphate etic stem cell transplant patients (11/14) Geron Inc. (Menlo hESC therapy Human embryonic Acute spinal Company is stopping recruitment to its Park, Calif.) stem cell therapy injury Phase I trial due to economic conditions (11/16) Pluristem PLX-PAD Cells derived from Critical limb Phase I data showed that it met all the Therapeutics Inc. the firm’s PLX ischemia endpoints, demonstrating a safe immuno- (Haifa, Israel) (PLacental eXpand- logic profile at all dosage levels and ed) platform showing potential efficacy (11/4) Provesica Ltd. XEN-D0501 An antagonist of Overactive Phase I data showed it was safe and well (Cambridge, UK) TRPV1 bladder tolerated (11/28) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 11. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 11 OF 32 Phase II Clinical Trials Update: November 2011 Company Product Description Indication Status (Date)# (Location) AUTOIMMUNE Adeona Trimesta Oral estriol candi- Multiple It will be tested in a Phase II trial evaluating Pharmaceuticals date sclerosis its potential effect on cognitive dysfunc- Inc. (Ann Arbor, tion in female MS patients (11/14) Mich.) Avanir AVP-923 An NMDA antago- Neuropathic Enrolled the first patient in a Phase II trial Pharmaceuticals nist and sigma-1 pain in multi- expected to enroll 400 patients worldwide Inc. (Aliso Viejo, agonist ple sclerosis (11/4) Calif.) Galapagos NV GLPG0634 JAK1 inhibitor Rheumatoid Achieved the primary endpoint of signifi- (Mechelen, arthritis cant improvement in the signs and symp- Belgium) toms of RA in a Phase IIa trial (11/28) KAI KAI-4169 Long-acting intrave- Secondary Phase II data showed it was effective, with Pharmaceuticals nous calcium sens- hyperparathy- 33% and 49% reductions in parathyroid Inc. (South San ing receptor agonist roidism hormone (PTH) from baseline in the 5-mg Francisco) and 10-mg dose groups, respectively (11/14) Opexa Tovaxin A T-cell therapy Relapsing- Phase IIb data showed it was well tolerated, Therapeutics remitting mul- with no serious adverse events, and dem- Inc. (The tiple sclerosis onstrated encouraging results in the reduc- Woodlands, Texas) tion of the annualized relapse rate and improvement in disease progression (11/15) SuppreMol SM101 A soluble version of Lupus Started dosing in a Phase IIa trial (11/29) GmbH the Fc gamma recep- (Martinsried, tor IIb Germany) CANCER Biothera Inc. Imprime PGG An immunomodula- Non-small-cell Completed enrollment of 90 patients in its (Eagan, Minn.) tor drug lung cancer Phase II trial in combination with Erbitux (11/18) Celldex CDX-110 Rindopepimut EGFRvIII- Phase II data showed final overall survival Therapeutics positive glio- of 24.6 months from diagnosis, compared Inc. (Needham, blastoma to 15.2 months for a historic control (11/22) Mass.) Exelixis Inc. Cabozantinib MET and VEGFR2 Hormone Initiated a Phase II trial (11/22) (South San inhibitor receptor-posi- Francisco) tive breast can- cer with bone metastases Galena NeuVax Vaccine Breast cancer Phase II data showed that it may provide a Biopharma Inc. meaningful clinical benefit in patients with (Lake Oswego, less aggressive forms of the disease (11/7) Ore.) Jennerex Inc. JX-594 Oncolytic virus Advanced liver Started the Phase IIb trial in patients who (San Francisco) cancer failed prior therapy with Nexavar (11/7) KAEL-GemVax GV1001 Telomerase peptide Non-small-cell Interim Phase II data demonstrated an 80% Co. Ltd. (Seoul, lung cancer immune response rate (11/16) South Korea) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 12. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 12 OF 32 Company Product Description Indication Status (Date)# (Location) Ligand Melphalan Captisol-enabled Cancer Phase II data showed that it met the Pharmaceuticals propylene glycol- requirements for establishing bioequiva- Inc. (San Diego) free melphalan lence to Alkeran (11/3) MediGene AG EndoTAG-1 A composition of Hormone- Phase II trial started (11/9) (Martinsried, paclitaxel combined receptor- Germany) with neutral and positive, HER2- positive lipids negative breast cancer Peregrine Bavituximab A phosphatidylser- Locally Company reported a 23.2-month median Pharmaceuticals ine-targeting mono- advanced or overall survival from a single-arm Phase II Inc. (Tustin, Calif.) clonal antibody metastatic trial of bavituximab in combination with breast cancer carboplatin and paclitaxel, compared with a published study showing median OS of 16 months with carboplatin and paclitaxel alone (11/23) YM BioSciences Nimotuzumab Anti-EGFR antibody Recurrent dif- Phase II data showed it was well tolerated Inc. (Mississauga, fuse intrinsic when administered to pediatric patients Ont.) pontine glioma (11/1) CARDIOVASCULAR Aastrom Ixmyelocel-T A patient-specific Critical limb Phase II data showed patients in the treat- Biosciences Inc. multicellular therapy ischemia ment arm had a 62% reduction in risk rela- (Ann Arbor, Mich.) tive to placebo in the primary endpoint of time to first occurrence of treatment failure (11/15) Mesoblast Ltd. Revascor Stem cell treatment Congestive Phase II data showed it did not provoke any (Melbourne, heart failure adverse immune responses and there was Australia) a 78% reduction in the rate of serious cardi- ac events (11/16) Resverlogix RVX-208 A small molecule Coronary Started dosing in its Phase IIb study (11/3); Corp. (Calgary, designed to stimu- artery disease the study is fully enrolled with 176 subjects Alberta) late endogenous (11/29) ApoA-I production Sanofi SA (Paris) REGN727/ A fully human anti- Elevated low- Phase II data showed that after 12 weeks of and Regeneron SAR236553 body targeting pro- density lipo- treatment patients achieved mean LDL-C Pharmaceuticals protein convertase protein reductions ranging from about 30% to Inc. (Tarrytown, subtilisin/kexin cholesterol more than 65%, depending on the dosing N.Y.) type 9 regimen; the control arm had a mean reduction of 10% (11/11) Theravance Inc. TD-4208 Inhaled long-acting Chronic Phase IIa data showed it met the primary (South San muscarinic antago- obstructive endpoint, with both study doses demon- Francisco) nist pulmonary strating a statistically significant mean disease change from baseline in peak forced expi- ratory volume in one second compared to placebo (11/16) Trophos SA TRO40303 A mitochondria pore Cardiac isch- Treated the first patient in a Phase II study (Marseille, France) modulator emia-reperfu- (11/1) sion injury in acute myocar- dial infarction To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 13. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 13 OF 32 Company Product Description Indication Status (Date)# (Location) Verona Pharma RPL554 A mixed phosphodi- Chronic Demonstrated bronchodilator effects in a plc (London) esterase 3/4 inhibi- obstructive Phase IIa trial (11/11) tor pulmonary disease CENTRAL NERVOUS SYSTEM BioDelivery BEMA Bupre- Formulation of Opioid Started a confirmatory study, which will Sciences norphine/ buprenorphine and dependence select the final doses to be used in the piv- International Nalaxone naloxone using otal bioequivalence study compared to Inc. (Raleigh, N.C.) BDSI’s BioErodible Suboxone (11/11) MucoAdhesive drug delivery technology Clinuvel Scenesse Afamelanotide; pho- Erythropoietic Phase II data showed it appeared to be Pharmaceuticals toprotective drug protoporphyria effective in preventing severe pain result- Ltd. (Melbourne, ing from sun exposure (11/4) Australia) Cytokinetics Inc. CK-2017357 A fast skeletal mus- Amyotrophic Started a second cohort of an ongoing (South San cle troponin activator lateral Phase II trial (11/2); opened enrollment in its Francisco) sclerosis third Phase II trial (11/30) Knopp Dexpram- A small-molecule Amyotrophic Phase II data showed the highest dose of Biosciences LLC ipexole modulator of mito- lateral the drug reduced mortality by 68% in the (Pittsburgh) chondrial bioener- sclerosis second stage of the trial (11/22) getics NeurogesX Inc. NGX-1998 A topical liquid for- Postherpetic Phase II top-line data showed that the trial (San Mateo, Calif.) mulation of high- neuralgia met its primary endpoint of a percentage concentration change from baseline vs. placebo (11/9) capsaicin Rexahn Serdaxin A neuroprotective Major depres- Phase IIb data showed it did not demon- Pharmaceuticals and antidepressant sive disorder strate efficacy compared to placebo over Inc. (Rockville, candidate an eight-week treatment period (11/7) Md.) Supernus SPN-810 Molindone hydro- Impulsive Started a Phase IIb trial (11/29) Pharmaceuticals chloride aggression Inc. (Rockville, associated Md.) with attention deficit hyper- activity disorder INFECTION Alnylam ALN-RSV01 RNAi therapy Respiratory Completed enrollment in a Phase IIb trial Pharmaceuticals syncytial virus (11/30) Inc. (Cambridge, Mass.) Inviragen Inc. DENVax A dengue vaccine Dengue fever Started a Phase II trial (11/29) (Fort Collins, Colo.) based on an attenu- ated DEN-2 virus Medivir AB TMC435 An oral HCV prote- Chronic hepa- Phase IIb data showed that TMC435 once (Huddinge, ase inhibitor titis C virus daily in addition to pegylated interferon Sweden) and ribavirin produced superior rates of sustained virologic response compared to standard of care (11/3) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 14. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 14 OF 32 Company Product Description Indication Status (Date)# (Location) SEEK (London) FLU-v Vaccine Influenza The vaccine was well tolerated and effec- tive against all influenza strains in a Phase II challenge trial (11/8) Sinovac Biotech Vaccine Enterovirus 71 vac- Hand, foot and The vaccine had good immunogencity and Ltd. (Beijing) cine mouth disease a favorable safety profile with no adverse events in a Phase II trial (11/10) MISCELLANEOUS Alexion Soliris Eculizumab Atypical hemo- Pivotal Phase II data demonstrated that Pharmaceuticals lytic uremic ongoing treatment sustained the suppres- Inc. (Cheshire, syndrome sion of complement-mediated thrombotic Conn.) microangiopathy, maintained or further improved longer-term renal function and enhanced quality of life (11/11) Ampio Ampion A low molecular Nasal inflam- A double-blind study in 20 patients Pharmaceuticals fraction of commer- mation showed it was safe and effective for reduc- Inc. (Greenwood cial albumin ing nasal inflammation (11/11) Village, Colo.) FibroGen Inc. (San FG-4592 A hypoxia-inducible Chronic kidney Phase IIb data showed that 96% of patients Francisco) factor prolyl hydrox- disease had an increase in hemoglobin (Hb) of at ylase inhibitor least 1 g/dL, and 93% had an Hb response; treatment was well tolerated (11/16) Kythera ATX-101 Formulation of Submental fat Data from a long-term follow-up Phase IIa Biopharma- deoxycholate, an trial showed more than 90% of initial ceuticals Inc. endogenous com- responders sustained or improved their (Los Angeles) pound that pro- response (11/8) motes the natural breakdown of dietary fat ProFibrix BV Fibrocaps A dry powder topi- Liver resection Phase II data showed a 50% reduction in (Leiden, the cal fibrin sealant surgery mean time to hemostasis, the primary end- Netherlands) developed from a point, compared to active control (11/16) mixture of fibrino- gen and thrombin RegeneRx RGN-259 Sterile, preservative- Central corne- Phase II data showed it met statistical sig- Biopharma- free eye drop al fluorescein nificance after a challenge-controlled ceuticals Inc. staining adverse environment in the reduction from (Rockville, Md.) baseline compared to placebo (11/7) Repros Androxal Oral therapy that Low Phase II data showed that 25 mg of Therapeutics stimulates the pitu- testosterone Androxal showed statistically equivalent Inc. (The itary gland efficacy to topical Androgel (11/9) Woodlands, Texas) Ritter RP-G28 A drug designed to Lactose Last patient completed treatment in a Pharmaceuticals stimulate colonic intolerance Phase II study (11/18) Inc. (Los Angeles) growth Unigene Oral PTH Oral parathyroid Osteoporosis Phase II data showed it achieved the prima- Laboratories Inc. hormone analogue ry endpoint of increasing bone mineral (Boonton, N.J.) density at the lumbar spine (11/10) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 15. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 15 OF 32 Phase III Clinical Trials Update: November 2011 Company Product Description Indication Status (Date)# (Location) AUTOIMMUNE Genzyme Corp. Lemtrada Alemtuzumab Relapsing- Phase III data showed it met both of its (Cambridge, Mass.) remitting co-primary endpoints (11/15) multiple sclerosis NPS Natpara NPSP558; a bioengi- Hypopara- Phase III data showed that 53% of Pharmaceuticals neered replica of thyroidism Natpara-treated patients achieved the pri- Inc. (Bedminster, human parathyroid mary endpoint vs. 2% of placebo-treated N.J.) hormone patients (11/8) Nuron Biotech NU100 A recombinant Relapsing- Started a pivotal, Phase III trial (11/2) Inc. (Exton, Pa.) human interferon remitting multi- beta-1b ple sclerosis CANCER Amgen Inc. Xgeva Denosumab Bone metasta- Phase III data showed that Xgeva signifi- (Thousand Oaks, ses of prostate cantly prolonged bone metastasis-free Calif.) cancer survival by 4.2 months, delayed bone metastasis by 3.7 months and reduced symptoms of bone metastasis by 33% (11/17) Celgene Revlimid Lenalidomide Castrate- Company is terminating the pivotal Phase International resistant III MAINSAIL trial because the combina- Sarl (Boudry, prostate cancer tion of docetaxel and prednisone with Switzerland) Revlimid did not show a statistically sig- nificant improvement in overall survival compared to docetaxel and prednisone plus placebo (11/28) Exelixis Inc. Cabozantinib A dual inhibitor of End-stage cas- Company was unable to secure an SPA (South San MET and VEGFR trate resistant and plans to start a Phase III trial by the Francisco) prostate cancer end of the year with pain as the primary efficacy endpoint; a second Phase III trial to evaluate overall survival time will launch in 2012 (11/2) Medivation Inc. MDV3100 An oral androgen Prostate cancer A independent data monitoring commit- (San Francisco) receptor antagonist tee found Phase III interim data to be posi- tive and recommended the trial be stopped so the drug could be offered to men receiving placebo (11/4) Novocure Inc. TTF therapy Tumor treating Recurrent glio- Phase III data showed overall survival was (Anaheim, Calif.) fields therapy blastoma 9% vs. 7% and 8% vs. 1% for the TTF group multiforme compared to the standard of care at two and three years of follow-up, respectively (11/22) Talon Marqibo Vincristine sulfate Newly Enrolled the first patient in the Phase III Therapeutics Inc. liposomes injection diagnosed study of Marqibo (11/30) (San Mateo, Calif.) aggressive non-Hodgkin’s lymphoma To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 16. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 16 OF 32 Company Product Description Indication Status (Date)# (Location) CARDIOVASCULAR Almirall SA Aclidinium Fixed-dose combi- Chronic Began a Phase III trial (11/17) (Barcelona, Spain) bromide and nation via the obstructive pul- and Forest formoterol Genuari inhaler monary disease Laboratories Inc. fumarate (New York) Gilead Sciences Ranexa Ranolazine Chronic angina Started a Phase III trial (11/8) Inc. (Foster City, Calif.) Inspiration OBI-1 An intravenous Congenital Started patient enrollment in the seconf Biopharma- recombinant por- hemophilia A of two pivotal studies (11/28) ceuticals Inc. cine Factor VIII prod- (Laguna Niguel, uct Calif.) Omthera Epanova Triglyceride- High Completed enrollment in a pivotal, Phase Pharmaceuticals lowering therapy triglycerides III trial (11/30) Inc. (Bedminster, N.J.) Santarus Inc. (San Rhucin Recombinant Hereditary A retrospective analysis showed that Diego) human C1 inhibitor angioedema median time to primary endpoint of onset of symptom relief was 60 minutes and median time to minimal symptoms was 240 minutes (11/7) CENTRAL NERVOUS SYSTEM Alexza Adasuve Staccato loxapine Schizophrenia Phase III data showed a 40% or greater Pharmaceuticals or bipolar I decrease from baseline in the total PANSS- Inc. (Mountain disorder Excited Component score; another analy- View, Calif.) sis showed a statistically significant improvement over placebo using the Clinical Global Impression-Improvement scale (11/9) Alkermes plc Vivitrol Naltrexone Opioid Data showed sustained efficacy in those (Dublin) dependence receiving Vivitrol in combination with psychosocial treatment for 18 months (11/10) Nabi Biopharma- NicVAX Smoking cessation Smoking A second Phase III trial failed to meet its ceuticals Inc. vaccine cessation primary endpoint of abstinence from cig- (Rockville, Md.) arettes; the vaccine was well tolerated (11/8) Targacept Inc. TC-5214 Nicotinic channel Major depres- Failed to meet its primary endpoint of (Winston Salem, modulator to nor- sive disorder change on the Montgomery-Asberg N.C.) malize cholinergic Depression Rating Scale following eight tone weeks of treatment in a Phase III trial (11/9) Vivus Inc. Qnexa Phentermine/topira- Obesity Results from the 56-week EQUIP study in (Mountain View, mate 1 ,267 patients showed an average weight Calif.) loss of 14.4% of initial body weight, plus improvements in blood pressure, glucose, triglycerides and cholesterol (11/7) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 17. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 17 OF 32 Company Product Description Indication Status (Date)# (Location) DIABETES GlaxoSmithKline Albiglutide A once-weekly glu- Type II diabetes Top-line data from the first of eight Phase plc (London) cagon-like peptide-1 III studies failed to meet the primary end- agonist point of noninferiority to Victoza, though it did demonstrate a statistically signifi- cant reduction in HbA1c from baseline (11/17) NephroGenex Pyridorin Pyridoxamine dihy- Type II diabetes FDA and company reached an agreement Inc. (Research drochloride on the design of a Phase III program (11/9) Triangle Park, N.C.) INFECTION Anacor AN2690 Tavaborole; a topical Onychomycosis Completed enrollment of a targeted 600 Pharmaceuticals antifungal product patients in the first of two Phase III trials Inc. (Palo Alto, (11/11) Calif.) Emergent Biothrax Anthrax vaccine Anthrax Started a pivotal immunmogenicity and BioSolutions Inc. adsorbed safety study of a three-dose vaccination (Rockville, Md.) schedule (11/18) Santarus Inc. (San Budesonide 9 mg taken once Mild or moder- Analysis of pooled data from its two Diego) MMX daily ate active ulcer- Phase III studies indicated it was statisti- ative colitis cally superior to placebo (11/2) Pharmasset Inc. PSI-7977 Nucleotide analogue Hepatitis C virus Started a pivotal Phase III program evalu- (Princeton, N.J.) ating an all-oral, interferon-free regimen of PSI-7977 and ribavirin (11/2) MISCELLANEOUS Affymax Inc. (Palo Peginesatide A synthetic, pegylat- Chronic kidney Phase III data showed that once-monthly Alto, Calif.) and ed peptidic com- disease peginesatide maintained Hb levels in CKD Takeda pound designed to patients on dialysis with anemia (11/14) Pharmaceutical bind to and stimu- Co. Ltd. (Osaka, late the erythropoie- Japan) tin receptor AMAG Feraheme Ferumoxytol Iron deficiency Data from a head-to-head study compar- Pharmaceuticals anemia and ing Feraheme to iron sucrose demonstrat- Inc. (Lexington, chronic kidney ed overall that ferumoxytol 1 .02 g Mass.) disease delivered as two injections had a favor- able safety profile and comparable effica- cy to 1 g of iron sucrose (11/15) Bionovo Inc. Menerba Estrogen receptor Menopausal hot The first patient was randomized and will (Emeryville, Calif.) beta selective drug flashes begin dosing in a Phase III pivotal trial (11/17) BioSante LibiGel Testosterone gel Female sexual Additional data showed the drug increas- Pharmaceuticals dysfunction es free testosterone in the serum of post- Inc. (Lincolnshire, menopausal women to the normal range Ill.) for premenopausal women (11/17) Emisphere SMC021 Oral calcitonin can- Osteopororis It failed to reduce the occurrence of new Technologies didate vertebral fractures in a three-year osteo- Inc. (Cedar Knolls, porosis study (11/15) N.J.) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 18. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 18 OF 32 Company Product Description Indication Status (Date)# (Location) Eyetech Inc. (Palm Macugen Pegaptanib sodium Neovascular Post-hoc analysis of the Phase III study Beach Gardens, age-related showed no apparent association between Fla.) macular treatment with Macugen and the occur- degeneration rence of sustained elevated intraocular pressure (11/8) NPS Gattex Teduglutide Short bowel Phase III data showed that nine patients Pharmaceuticals syndrome discontinued STEPS 2 due to adverse Inc. (Bedminster, events, and while gastrointestinal side N.J.) effects were the most common culprit, three patients developed cancer, with two cases proving fatal (11/1) Trimel CompleoTRT A bioadhesive intra- Hypogonadism Dosed the first patient in a pivotal Phase Pharmaceuticals nasal testosterone III study (11/1) Corp. (Toronto) gel product Vertex Kalydeco Ivacaftor, VX0-770 Cystic fibrosis Pivotal data showed that children who Pharmaceuticals received drug experienced rapid and sus- Inc. (Cambridge, tained improvements in lung function Mass.) and other key measures of disease, including weight gain and a reduction in sweat chloride throughout the 48-week trial compared to placebo (11/4) Vivus Inc. Avanafil A phosphodiester- Erectile A new analysis from a Phase III study (Mountain View, ase-5 inhibitor dysfunction found it was effective in as early as 15 Calif.) minutes after dosing in 80% of all study- wide sexual attempts (11/15) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. SPA = Special Protocol Assessment. BR A N D NE W ! Only BioWorld Today, the most respected news source to the industry’s executive suite for more than 20 years, can bring your future into view with the brand new State of the Industry Report 2011! The deals, mergers & acquisitions, the The innovations, the R&D, the cutting edge discoveries that are leading the pack to Wall Street In-depth analysis into emerging markets; drug The State of the Industry Report— defining the year—mapping your future! Order Today! ONLINE: CALL: EMAIL: www.bioworld.com/biotechindustry 1-800-888-3912 or 1-404-262-5547 orders@bioworld.com *Mention priority code S11406/01137 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 19. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 19 OF 32 American Association for the Study of Liver Diseases (The 62nd annual meeting was held Nov. 4-8 in San Francisco.) Company Product Description Indication Status (Date)# (Location) Boehringer BI201335 Next-generation Hepatitis C virus Phase IIb data of BI201335 in combination Ingelheim GmbH protease inhibitor with pegylated protease inhibitor, in com- (Ingelheim, bination with pegylated interferon and Germany) ribavirin, showed its potential to shorten treatment duration to 12 weeks and to improve the likelihood of viral cure com- pared to pegylated interferon and ribavi- rin alone (11/9) Bristol-Myers BMS-790052 Dual, all-oral direct- Hepatitis C virus Phase II data show they achieved unde- Squibb Co. (New acting antiviral regi- tectable viral load at 12 weeks post-treat- York) men of NS5A ment in 90% of genotype 1b HCV patients replication complex who had previously not responded to inhibitor daclatasvir peginterferon alfa and ribavirin (11/9) and NS3 protease inhibitor asunaprevir ChemoCentryx CCX354 Orally-active small Rheumatoid Phase II data showed it was safe and well Inc. (Mountain molecule that spe- arthritis tolerated and had clinical and biological View, Calif.) cifically targets and activity at 200 mg once daily (11/10) inhibits the chemo- kine receptor known as CCR1 Cytheris SA CYT107 A recombinant Hepatitis C virus Interim Phase I/IIa data showed treatment (Paris) human interleukin-7 with CYT107 plus interferon and ribavirin resulted in a broad immune response associated with HCV viral clearance (11/9) Gilead Sciences Viread Tenofovir disoproxil Chronic hepati- Phase III data showed it maintained long- Inc. (Foster City, fumarate tis B virus term viral suppression of HBV and is asso- Calif.) ciated with a reduction in liver fibrosis and a reversal of cirrhosis (11/8) Jennerex Inc. JX-594 Engineered oncolyt- Advanced liver Phase II data showed a statistically signifi- (San Francisco) ic virus cancer cant benefit in overall survival for the high-dose group vs. the low-dose group (11/8) Merck & Co. Inc. Victrelis Protease inhibitor; Chronic An interim analysis from the open-label (Whitehouse boceprevir hepatitis C virus study testing Victelis in combination with Station, N.J.) peginterferon alfa and ribavirin showed that 38% achieved a sustained virologic response (11/8) NasVax Ltd. (Nes Monoclonal Oral anti-CD3 mono- Nonalcoholic Phase IIa data showed that it promoted Ziona, Israel) antibodies clonal antibodies steatohepatitis regulator y T cells, decreased liver enzymes and alleviated insulin resistance (11/8) Santaris Pharma Miravirsen MicroRNA-targeted Hepatitis C virus Phase IIa data showed that given as a A/S (Hoersholm, drug four-week monotherapy treatment it pro- Denmark) vided robust dose-dependent antiviral activity with a mean reduction of 2 to 3 logs from baseline (11/8) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 20. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 20 OF 32 Company Product Description Indication Status (Date)# (Location) Scynexis Inc. SCY-635 An oral cyclophilin Hepatitis C virus Data demonstrated it reactivated the (Research Triangle inhibitor body’s natural defense mechanism, mak- Park, N.C.) ing it capable of inhibiting replication of the virus (11/8) Tibotec TMC435 Once-daily therapy; Chronic geno- Phase IIb data showed when administered Pharmaceuticals a HS3/4A protease type-1 hepatitis in combination with peginterferon alpha- (unit of Johnson & inhibitor C virus 2a and ribavirin, the drug demonstrated a Johnson; New significantly higher sustained virologic Brunswick, N.J.) and response rate compared to placebo plus Medivir AB PR (11/8) (Huddinge, Sweden) Transgene SA TG-4040 Vaccine Hepatitis C virus Phase II data showed when administered (Strasbourg, in combination with pegylated interferon France) alpha plus ribavirin, it was twice as effec- tive in eliciting a complete early virologic response as standard therapy alone (11/8) Vertex VX-222 Polyermase inhibitor Genotype-1 Phase II data of VX-222 with Incivek, Pharmaceuticals chronic hepati- pegylated interferon and ribavirin Inc. (Cambridge, tis C virus showed that 90% of patients had unde- Mass.) tectable HCV levels in the blood 12 weeks after the end of treatment (11/8) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. Deals and breakthroughs in biotechnology know no borders … shouldn’t your news service do the same? Bringing global intelligence and analysis right to your desktop each week. Visit us at www.bioworld.com or call 1-800-888-3912 to order or request a sample issue today! To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 21. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 21 OF 32 American College of Rheumatology and the Association of Rheumatology Health Professionals (The ACR/ARHP meeting was held Nov. 5-9 in Chicago.) Company Product Description Indication Status (Date)# (Location) 4SC AG (Planegg- Vidofludimus Designed to target Rheumatoid Phase IIb data demonstrated substantial Martinsried, IL-17 and dihydro- arthritis anti-inflammatory activity of vidofludi- Germany) orotate dehydroge- mus, with an ACR20 improvement of nase 50.8% vs. 44.8% in the control group (11/8) Abbott (Abbott Humira Anti-TNF drug; adali- Active nonra- Phase III data showed that more than Park, Ill.) mumab diographic twice as many patients receiving Humira axial spondylo- compared to those receiving placebo arthritis achieved the primary endpoint of 40% improvement (11/8) Abbott (Abbott Humira Anti-TNF drug; adali- Ankylosing Five-year results from an open-label Park, Ill.) mumab spondylitis extension show it resulted in a mean change from baseline of -0.6 in Bath Ankylosing Spondylitis Metrology Index (11/9) Amgen Inc. Prolia Denosumab; a RANK Postmenopausal Phase III data showed that up to six years (Thousand Oaks, ligand inhibitor osteoporosis of continued Prolia treatment through Calif.) both trials produced cumulative bone mineral density gains of 15.2% at the lum- ber spine and 7.5% at the total hip, com- pared with baseline (11/8) Ardea Lesinurad URAT1 inhibitor Gout Phase IIb data showed a combination of Biosciences Inc. lesinurad with allopurinol had consistent, (San Diego) sustained reductions in serum urate lev- els, with 90% of combination therapy patients at week 44 reaching the medical- ly recommended target of sUA less than 6 mg/dL (11/8) BioCryst BCX4208 Next generation Gout Phase IIb data show that patients with Pharmaceuticals purine nucleoside gout who have failed to reach serum uric Inc. (Research phosphorylase acid level of <6 mg/dL on allopurinol Triangle Park, N.C.) inhibitor alone met the primary endpoint (11/9) Celgene Apremilast Oral Ankylosing Phase II data show it was associated with International immunotherapy spondylitits a trend to greater mean improvement Sarl (Boudry, from baseline compared with placebo in Switzerland) all parameters (11/9) Centocor Ortho CNTO 136 Sirukumab; a subcu- Rheumatoid Phase II data showed treatment signifi- Biotech Inc. taneous human anti- arthritis cantly improved rheumatoid arthritis (Horsham, Pa.) body directed signs and symptoms (11/8) against interleukin 6 Genentech Inc. Actemra Tocilizumab Rheumatoid Phase III data showed Actemra as a mono- (South San arthritis therapy resulted in 34.8% of patients Francisco) achieving a DAS28 score at 24 weeks, comparable to the 40.4% rate in patients taking Actemra plus methotrexate (11/8) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 22. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 22 OF 32 Company Product Description Indication Status (Date)# (Location) Neovacs SA Kinoid Immunotherapy Lupus Data from a 28-patient study show that (Paris) Kinoid reduced the gene dysregulation associated with the overexpression of INF-alpha (11/9) Novartis AG (East ACZ885 Canakinumab Active systemic Pivotal Phase III data showed that 45% of Hanover, N.J.) juvenile idio- children were able to substantially reduce pathic arthritis their use of oral corticosteroids within 28 weeks of beginning treatment (11/8) Savient Krystexxa Pegloticase Gout A post-hoc analysis of pivotal Phase III Pharmaceuticals data and an open-label extension con- Inc. (East firmed that risk of infusion reactions and Brunswick, N.J.) anaphylaxis could be mitigated by the monitoring of serum uric acid prior to each infusion and discontinuing Krystexxa in patients whose sUA levels rise above 6mg/dL (11/8) UCB SA (Brussels, Cimzia Certolizumab pegol; Rheumatoid Findings from a post-hoc analysis sug- Belgium) pegylated anti-TNF arthritis gest that disease activity response rates drug of Cimzia plus methotrexate as early as week 12 helped predict the effect on struc- tural joint damage in patients with mod- erate to severe disease at one year (11/8) Vertex VX-509 JAK3 inhibitor Rheumatoid Phase IIa data showed it significantly Pharmaceuticals arthritis improved rheumatoid arthritis as mea- Inc. (Cambridge, sured by the proportion of people who Mass.) achieved at least a 20% improvement in the signs and symptoms (11/8) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 23. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 23 OF 32 American College of Allergy, Asthma and Immunology (The annual meeting was held Nov. 3-8 in Boston.) Company Product Description Indication Status (Date)# (Location) Baxter HyQ A combination Bacterial Phase III data showed patients with pri- International Inc. immunoglobulin infections mary immunodeficiencies had a reduced (Deerfield, Ill.) product rate of serious bacterial infections when treated with HyQ (11/8) CSL Behring LLC Berinert C1-esterase inhibitor Hereditary Data showed median time of 0.46 hours (King of Prussia, angioedema to the onset of symptom relief and 15.5 Pa.) hours to resolution (11/8) Dyax Corp. Kalbitor Ecallantide; A plas- Hereditary Clinical data showed it remained effective (Cambridge, Mass.) ma kallikrein inhibi- angioedema across multiple episodes of acute HAE tor attacks with no apparent increase in adverse events (11/8) Sunovion Ciclesonide Nasal aerosol; a cor- Perennial Phase III data showed statistically signifi- Pharmaceuticals ticosteroid formu- allergic rhinitis cant improvements in reflective and Inc. (Marlborough, lated with a instantaneous nasal symptoms after the Mass.) hydrofluoroalkane first six weeks compared to placebo, as propellant well as tolerability over 26 weeks (11/8) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. American Heart Association (The scientific sessions were held Nov. 12-16 in Orlando.) Company Product Description Indication Status (Date)# (Location) Amarin Corp. plc AMR101 A prescription-grade High Phase III data showed patients who were (Dublin, Ireland) omega-3 fatty acid triglycerides on statin therapy experienced a signifi- cant reduction in triglyceride levels and other lipid parameters, as well as inflam- matory biomarkers, when treated with AMR101 (11/17) Genzyme Corp. MIPO Mipomersen Low-density Positive results from a randomized, dou- (Cambridge, Mass.) lipoprotein ble-blind, placebo-controlled Phase II trial cholesterol showed that weekly treatment reduced LDL-C by 47% compared to 2% with place- bo (11/17) Regado REG1 Anticoagulant sys- Cardiovascular Phase IIb data suggested it provided near- Biosciences Inc. tem; reversible anti- disease ly complete Factor IXa inhibition with a (Basking Ridge, thrombotic agent dose of 1 mg/kg of pegnivacogin and, N.J.) when followed by 75% and 100% reversal doses of anivamersen, resulted in numeri- cally lower rates of ischemic events com- pared with heparin (11/16) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 24. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 24 OF 32 American Association for Cancer Research, National Cancer Institute and European Organization for Research and Treatment of Cancer (The AACR-NCI-EORTC international conference was held Nov. 12-16 in San Francisco.) Company Product Description Indication Status (Date)# (Location) ACT Biotech Inc. Telatinib An oral VEGF inhibi- Gastric cancer Phase II data showed patients treated (San Francisco) tor with telatinib and Xeloda and cisplatin who responded with a large decrease of a specific angiogenesis biomarker were found to have a lower risk of disease pro- gression and death compared with patients that had smaller decreases (11/15) Cerulean Pharma CRLX101 A camptothecin Non-small-cell Phase I/IIa data showed that median pro- Inc. (Cambridge, nanoparticle lung cancer gression-free survival time was 4.4 Mass.) months for 21 of the 38 patients (11/15) Enzon EZN-3042 An LNA oligonuclo- Advanced solid Phase I data demonstrated good tolerabil- Pharmaceuticals etide targeting sur- malignancies ity and antitumor activity (11/15) Inc. (Piscataway, vivn mRNA N.J.) Exelixis Inc. Cabozantinib An inhibitor of MET, Metastatic cas- Phase I data suggested that a daily start- (South San RET and VEGFR2 tration-resistant ing dose of 40 mg resulted in high rates Francisco) prostate cancer of bone scan response assessed by com- puter-aided detection in men with CRPC and bone metastases; interim data on pain relief and related reduction in nar- cotic analgesic use showed 56% of patients decreased their dose by at least 30% (11/15) Geron Corp. GRN1005 Lead LRP-directed Brain metasta- Phase I data demonstrated single-agent (Menlo Park, Calif.) peptide-paclitaxel ses from solid clinical activity in both indications (11/16) conjugate tumors and malignant glioma MethylGene Inc. MGCD265 Oral Met/VEGF Advanced solid Phase I data showed the compound was (Montreal) receptor tyrosine cancers tolerable, with no treatment-related seri- kinase inhibitor ous adverse events, and showed early signs of clinical benefit (11/15) Myrexis Inc. (Salt MPC-3100 Heat-shock protein Cancer Phase I data showed that it was generally Lake City) inhibitor safe and well tolerated (11/15) Oncolytics Reolysin Intravenous therapy Advanced pan- Interim Phase II data of Reolysin in combi- Biotech Inc. creatic cancer nation with gemcitabine showed that all (Calgary) but one of 12 patients reported symptom- atic improvement, including seven with stable disease for 12 weeks or longer, for a clinical benefit rate of 58% (11/15) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 25. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 25 OF 32 Company Product Description Indication Status (Date)# (Location) Oncothyreon Inc. PX-866 Irreversible, pan-iso- Advanced Two Phase I/II trials showed no dose-lim- (Seattle) form phosphati- cancer iting toxicities with the combination of dylinositol-3-kinase docetaxel and PX-866 generally well tol- inhibitor erated; best response was stable disease in 21 of 28 patients and progressive dis- ease in seven patients, for a disease con- trol rate of 75% (11/15) Pieris AG PRS-050 Anticalin; an anti- Advanced solid Phase I data showed it was well tolerated, (Freising- VEGF targeted pro- tumors with no maximum-tolerated dose Weihenstephan, tein therapeutic reached, and it demonstrated biological Germany) activity, with no immunogenicity (11/16) Syndax Exemestane Hormone therapy Postmenopausal A pharmacodynamic analysis in a subset Pharmaceuticals estrogen-recep- of patients demonstrated an association Inc. (San Francisco tor positive of the pharmacodynamic marker lysine and Waltham, breast cancer hyperacetylation with clinical outcome Mass.) (11/15) TetraLogic TL32711 Smac mimetic candi- Solid tumors Phase I data suggested it caused potent Pharmaceuticals date and lymphomas and sustained suppression of inhibitor of (Malvern, Pa.) apoptosis protein in patient peripheral blood mononuclear cells and tumor biop- sies over seven days at tolerable dose lev- els (11/15) Ziopharm Zymafox Palifosfamide Non-small-cell Phase Ib data showed that Zymafos in Oncology Inc. lung cancer combination with etoposide and carbopl- (New York) atin can be given at a maximum-tolerated dose of 130 mg/m2, with a dose-limiting toxicity of neutropenic fever (11/15) Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. Available in print and PDF with our The ALL NEW BioWorld Executive interactive point Compensation Report 2012 has the & click table of contents. figures you’ve been waiting for… The salary, bonus, stock options, market capitalization & total compensation packages for 673 key biotech executives in more than 190 companies! Order today! Visit us at bioworld.com to purchase your copy today! To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 26. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 26 OF 32 Non-U.S. Clinical Trials & Regulatory Actions: Nov. 2011 Company Product Description Indication Status (Date)# (Location) AUTOIMMUNE DuoCort Pharma Plenadren Hydrocortisone, Adrenal European Commission granted approval AB (Helsingborg, modified-release insufficiency (11/8) Sweden) tablet Pfizer Inc. (New Tofacitinib An oral JAK inhibitor Moderate to EMA validated and began review of tofaci- York) severe active tinib (11/22) rheumatoid arthritis CANCER Amgen Inc. Vectibix Panitumumab; Wild-type KRAS Gained an expansion to the European (Thousand Oaks, EGFR-targeted can- metastatic label, allowing it to be used as a first-line Calif.) cer drug colorectal treatment in combination with FOLFOX cancer and as a second-line treatment in combi- nation with FOLFIRI (11/16) BioAlliance Lauriad Clonidine Lauriad To prevent European Commission granted orphan Pharma SA (Paris) radiotherapy- designation (11/3) incuded oral mucositits in head and neck cancer patients Celgene Revlimid Lenalidomide Myelodysplastic Swiss regulators approved it for use in International syndromes patients with transfusion-dependent ane- Sarl (Boudry, mia due to myelodysplastic syndromes Switzerland) associated with a deletion 5q cytogenic abnormality with or without additional cytogenic abnormalities (11/22) Celldex CDX-110 Rindopepimut Glioblastoma EMA granted orphan drug designation Therapeutics (11/1) Inc. (Needham, Mass.) CARDIOVASCULAR Amsterdam Gene therapy Consists of an ade- Hemophilia B Received orphan drug designation from Molecular no-associated viral the EMA (11/17) Therapeutics NV vector containing (Amsterdam, the the human factor IX Netherlands) gene Dyax Corp. Kalbitor Ecallantide Hereditary Companies decided to withdraw the MAA (Cambridge, Mass.) angioedema because European regulators indicated and Sigma-Tau the data were not sufficient to prove a Group (Rome) positive risk-benefit (11/15) Novartis AG Rasitrio Aliskiren, amlodip- High blood Gained European approval (11/29) (Basel, ine and hydrochlo- pressure Switzerland) rothiazide CENTRAL NERVOUS SYSTEM Aquinox AQX01125 Anti-inflammatory Asthma Began two Phase IIa trials in the UK (11/11) Pharmaceuticals drug Inc. (Vancouver, British Columbia To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 27. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 27 OF 32 Company Product Description Indication Status (Date)# (Location) Avanir Nuedexta Dextromethorphan Pseudobulbar Submitted an application to the EMA seek- Pharmaceuticals hydrobromide and affect ing approval (11/1); EMA accepted the MAA Inc. (Aliso Viejo, quinidine sulfate filing (11/18) Calif.) Pfizer Inc. (New vyndaqel Tafamidis Transthyretin European Commission approved it to York) familial amyloid treat adult patients with Stage I symptom- polyneuropathy atic polyneuropathy (11/18) DIABETES AstraZeneca plc Onglyza Saxagliptin; DPP-4 Diabetes Gained a European label expansion (11/29) (London) and inhibitor Bristol-Myers Squibb Co. (New York) Zealand Pharma Lyxumia Lixisenatide; once- Type II diabetes A European marketing authorization A/S (Copenhagen, daily GLP-1 agonist application has been submitted by its Denmark) partner Sanofi SA for Lyxumia (11/4) INFECTION Gilead Sciences Tenofovir A gel HIV The National Institute of Allergy and Inc. (Foster City, Infectious Diseases requested the Calif.) Microbicide Trials Network discontinue the use of tenofovir gel as there was no difference when compared to placebo (11/29) MISCELLANEOUS Alexion Soliris Eculizumab Atypical hemo- European Commission extended the indi- Pharmaceuticals lytic uremic syn- cation for Soliris to include pediatric and Inc. (Chesire, drome adult patients (11/30) Conn.) Pierre Fabre Joncia Milnacipran Fibromyalgia Received marketing authorization in Medicament Australia (11/22) (Castres, France) Regeneron Eylea Aflibercept Wet age-related Started a Phase III trial in China (11/29) Pharmaceuticals macular degen- Inc. (Tarrytown, eration N.Y.) and Bayer HealthCare (Leverkusen, Germany) Notes: CHMP = Committee for Medicinal Products for Human Use; EMA = European Medicines Agency; MAA = Marketing authorization application; MRP = Mutual Recognition Procedure. Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 28. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 28 OF 32 FDA Submissions, Approvals & Other Actions: Nov. 2011 Company Product Description Indication Status (Date)# (Location) AUTOIMMUNE Cipher Cip- Formulation of Acne Submitted an amendment to its pend- Pharmaceuticals isotretinoin isotretinoin ing NDA; it includes additional clinical Inc. (Mississauga, information requested by the FDA Ont.) (11/28) Horizon Pharma Lodotra A modified delayed- Rheumatoid FDA accepted the filing and will Inc. (Deerfield, Ill.) release formulation arthritis review its NDA (11/30) of low-dose predni- sone Regeneron Arcalyst Rilonacept injection To prevent gout FDA has accepted the company’s sBLA Pharmaceuticals flares (11/23) Inc. (Tarrytown, N.Y.) CANCER Bristol-Myers Erbitux Cetuximab; mono- Late-stage head FDA approved it for use with chemo- Squibb Co. (New clonal antibody and neck cancer therapy (11/8) York) EUSA Pharma Inc. Erwinase Asparaginase Acute lymphoblas- FDA approved it (11/21) (Oxford, UK) Erwinia chrysanthe- tic leukemia mi Genentech Inc. Avastin Bevacizumab Breast cancer FDA revoked the approval in the (South San breast cancer indication because evi- Francisco) dence showed the benefits did not outweigh the risks (11/21) Millennium Velcade Bortezomib Multiple myeloma FDA approved an sNDA, updating the (Cambridge, Mass.) label to include additional long-term overall survival data (11/3) Onyx Carfilzomib A next-generation Relapsed and FDA accepted the new drug applica- Pharmaceuticals proteasome inhibitor refractory multiple tion (11/30) Inc. (South San myeloma Francisco) Pfizer Inc. (New Mylotarg Gemtuzumab ozo- Acute myeloid leu- FDA has removed approval upon the York) gamicin kemia company’s request after a confirmato- ry trial failed to demonstrate clinical benefit and revealed safety concerns (11/29) Spectrum Zevalin Ibritumomab tiux- Non-Hodgkin’s Received FDA approval to remove the Pharmaceuticals etan lymphoma pre-treatment bioscan requirement Inc. (Henderson, for Zevalin (11/22) Nev.) SymBio Treakisym Bendamustine Non-Hodgkin’s lym- Started a Phase II trial in Japan (11/15) Pharmaceuticals hydrochloride phoma and mantle Ltd. (Tokyo) cell lymphoma CARDIOVASCULAR Amarin Corp. plc AMR101 A prescription-grade High triglycerides FDA has accepted its NDA (11/29) (Dublin, Ireland) fish oil To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 29. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 29 OF 32 Company Product Description Indication Status (Date)# (Location) Chelsea Northera Droxidopa; an oral Symptomatic neu- FDA accepted for filing its NDA (11/18) Therapeutics synthetic precursor rogenic orthostatic International Ltd. of norepinephrine hypotension (Charlotte, N.C.) CENTRAL NERVOUS SYSTEM IntelGenx Corp. CPI-300 A high-strength for- Major depressive Gained FDA approval (11/15) (Saint Laurent, mulation of bupro- disorder Quebec) pion hydrochloride Otsuka Aripiprazole Depot formulation Schizophrenia FDA agreed that an NDA for mainte- Pharmaceutical nance treatment is sufficiently com- Co. Ltd. (Tokyo) plete for review (11/28) Pacira Exparel Bupivacaine lipo- Postsurgical pain Gained FDA approval (11/1) Pharmaceuticals some suspension, relief Inc. (Parsippany, 1 .3% N.J.) QRxPharma Ltd. MoxDuo Morphine/ Pain FDA plans to review the NDA with a (Sydney, Australia) oxycodone PDUFA data of June 25, 2012 (11/9) Transcept Intermezzo Zolpidem tartrate Insomnia FDA approved it for use as needed to Pharmaceutical treat insomnia characterized by mid- Inc. (Point dle-of-the-night waking followed by Richmond, Calif.) difficulty returning to sleep (11/28) and Purdue Pharmaceutical Products LP (Stamford, Conn.) Vivus Inc. Qnexa Phentermine/topira- Obesity FDA accepted for filing and review the (Mountain View, mate NDA (11/4) Calif.) DIABETES Alimera Sciences Iluvien Fluocinolone ace- Diabetic macular Received a second complete response Inc. (Atlanta) tonide intravitreal edema letter with the FDA saying the risks of insert adverse events in two Phase III trials were not offset by the benefits; the agency requested two additional tri- als (11/14) Furiex Alogliptin/ Fixed-dose combi- Type II diabetes Submitted an NDA (11/29) Pharmaceuticals metformin nation Inc. (Morrisville, N.C.) and Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 30. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 30 OF 32 Company Product Description Indication Status (Date)# (Location) INFECTION Santen Inc. DE-109 Sirolimus Chronic/refractory FDA granted orphan drug designation (Emeryville, Calif.) anterior non-infec- for sirolimus (11/11) tious uveitis, non- infectious intermediate uve- itis, non-infectious panuveitis and non- infectious uveitis affecting the poste- rior segment of the eye MISCELLANEOUS Incyte Corp. Jakafi Ruxolitinib; JAK Myelofibrosis FDA approved it (11/17) (Wilmington, Del.) inhibitor Regeneron Eylea Aflibercept Wet age-related FDA granted approval (11/22) Pharmaceuticals macular Inc. (Tarrytown, degeneration N.Y.) Shire plc (Dublin, Vpriv Velaglucerase alfa; Gaucher’s disease Submitted regulatory filings in the UK) an enzyme replace- U.S. and Europe for the production of ment therapy Vpriv (11/23) Summit Corp. SMT C1100 A small-molecule Duchenne’s FDA granted orphan drug status (Oxford, UK) drug that up-regu- muscular (11/29) lates production of a dystrophy naturally occurring protein called utro- phin to replace missing dystrophin Notes: BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment. Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 20 1 1 AHC Media. Reproduction is strictly prohibited.
  • 31. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 31 OF 32 The Week’s Biggest Gainers and Losers 10 BIGGEST U.S. WINNERS FOR THE WEEK 10 BIGGEST U.S. LOSERS FOR THE WEEK (By Percent) (By Dollars) (By Percent) (By Dollars) Generex Biotech 266.67 Affymax 2.39 Unigene -42.70 Regeneron Pharma -4.90 Bio-path Holdings 242.86 Celgene 2.37 Alexza Pharma -39.22 Pharmacyclics -3.24 Affymax 45.52 Seattle Genetics 1.68 Poniard Pharma -21.11 Onyx Pharma -3.18 Alseres 37.50 Oncothyreon 1.39 Pharmacyclics -20.73 Alexion Pharma -1.92 Dara Biosciences 27.45 Flamel Tech 1.07 XenoPort -18.43 InterMune -1.35 Flamel Tech 26.23 Alkermes 0.97 Titan Pharma -16.54 Poniard Pharma -1.03 Zalicus Inc 21.05 Auxilium Pharma 0.88 Galena Biopharma -14.93 Momenta Pharma -0.92 Peregrine Pharma 20.62 Bio-path Holdings 0.85 Complete Genomics -14.04 Myriad Genetics -0.91 Oncothyreon 20.26 Raptor Pharma 0.81 Idera Pharma -13.60 BioMarin Pharma -0.90 Synta Pharma 19.55 Pacira Pharma 0.81 Epicept -13.33 XenoPort -0.89 BioWorld Stock Report For Public Biotechnology CompaniesCompany Symbol Close Close %Change Vol Company Symbol Close Close %Change Vol 12/2 12/9 WK YTD (000) 12/2 12/9 WK YTD (000)3S Bio SSRX 12.18 11.53 -5.34 -23.03 291 Cardiovascular Bio CVBT 0.4 0.35 -12.50 2.94 424Aastrom Biosci ASTM 2.12 2.09 -1.42 -18.36 1239 Catalyst Pharma CPRX 1.07 1.13 5.61 17.71 639Acadia Pharma ACAD 0.98 1.05 7.14 -14.63 438 Celgene CELG 61.21 63.58 3.87 6.98 17107Accentia Biopharma ABPI 0.3 0.33 10.00 -56.00 268 Cell Therapeutics CTIC 1.11 1.19 7.21 219.89 14550Access Pharma ACCP 1.44 1.44 0.00 -41.22 100 Celldex Therap CLDX 2.9 2.89 -0.34 -30.70 970AcelRx Pharma ACRX 2.85 2.73 -4.21 -40.00 5 CEL-SCI CVM 0.3 0.32 6.67 -61.55 4548Achillion ACHN 6.85 7.52 9.78 85.22 4862 Cerus CERS 2.99 3.11 4.01 25.40 3881Acorda Therap ACOR 22.53 23.22 3.06 -15.59 1624 Chelsea Therap CHTP 5.45 5.15 -5.50 -32.15 2934Adamis ADMP 0.22 0.22 0.00 7.32 300 Cleveland Biolabs CBLI 2.94 3.08 4.76 -57.16 960Adolor ADLR 4.6 4.77 3.70 284.68 4195 Codexis CDXS 5.85 5.72 -2.22 -46.04 1107Adventrx Pharma ANX 0.62 0.6 -3.23 -77.01 1674 Columbia Labs CBRX 2.16 2.19 1.39 0.00 2252Aegerion Pharma AEGR 16.63 16.91 1.68 19.34 358 CombiMatrix CBMX 1.74 1.55 -10.92 -30.49 136AEterna Zentaris AEZS 1.64 1.74 6.10 -0.57 9573 Complete Genomics GNOM 3.49 3 -14.04 -59.84 2084Affymax AFFY 5.25 7.64 45.52 14.89 35197 Compugen CGEN 4.43 4.35 -1.81 -12.65 239Agenus AGEN 2.43 2.37 -2.47 -60.89 159 Corcept CORT 3.2 3.28 2.50 -16.33 511Albany Molecular AMRI 2.34 2.3 -1.71 -60.88 1412 CorMedix CRMD 0.35 0.33 -5.71 -81.25 39Alexion Pharma ALXN 66.98 65.06 -2.87 61.52 6022 Cornerstone Therap CRTX 5.49 5.52 0.55 -2.30 109Alexza Pharma ALXA 1.02 0.62 -39.22 -50.79 13856 Cubist Pharma CBST 38.1 37.79 -0.81 75.12 3767Alimera Sciences ALIM 1.23 1.4 13.82 -86.51 1520 Curis CRIS 3.65 3.78 3.56 87.13 1377Alkermes ALKS 14.86 15.83 6.53 28.70 3755 Cytokinetics CYTK 1.04 1.01 -2.88 -51.67 525Allos Therapeutics ALTH 1.27 1.3 2.36 -71.86 2585 CytRx CYTR 0.35 0.33 -5.71 -67.00 2494Alnylam Pharma ALNY 6.81 7.09 4.11 -28.24 987 Dara Biosciences DARA 1.02 1.3 27.45 -61.42 191Alseres ALSE 0.08 0.11 37.50 -15.38 41 Dendreon DNDN 8.62 7.96 -7.66 -77.52 22292AMAG Pharma AMAG 18.4 19.05 3.53 4.84 805 DepoMed DEPO 4.82 4.73 -1.87 -27.01 1847Amarin AMRN 7.78 7.27 -6.56 -12.30 8463 DiaDexus DDXS 0.17 0.15 -11.76 -48.28 663Amgen AMGN 58.1 58.59 0.84 5.51 95571 Discovery Labs DSCO 1.69 1.7 0.59 -48.95 652Amicus FOLD 3.21 3.43 6.85 -28.39 267 DURECT DRRX 1.3 1.29 -0.77 -63.66 1319Ampliphi Biosci APHB 0.09 0.09 0.00 -64.00 119 Dusa Pharma DUSA 3.77 4.08 8.22 65.85 500Amylin Pharma AMLN 10.5 10.45 -0.48 -29.82 7809 Dyax DYAX 1.29 1.36 5.43 -34.93 1812Anacor Pharma ANAC 5.42 5.59 3.14 4.10 226 Dynavax DVAX 3.2 3.2 0.00 3.23 9546Anthera Pharma ANTH 6.48 6.66 2.78 36.76 789 Emergent BioSol EBS 17.57 17.42 -0.85 -26.44 867Ardea Biosciences RDEA 19.93 19.05 -4.42 -27.51 542 Emisphere Tech EMIS 0.37 0.33 -10.81 -86.08 267Arena Pharma ARNA 1.68 2 19.05 16.96 19743 Endocyte ECYT 10.4 11 5.77 42.30 372Ariad Pharma ARIA 11.9 11.63 -2.27 124.52 10782 Entremed ENMD 1.54 1.51 -1.95 -70.28 111ArQule ARQL 5.54 5.71 3.07 -1.38 1013 Enzo Biochem ENZ 2.27 2.33 2.64 -57.09 574Array BioPharma ARRY 2.12 2.3 8.49 -22.82 1456 Enzon Pharma ENZN 7.01 7.03 0.29 -42.38 909Aryx ARYX 0.01 0.01 0.00 -85.71 59 Epicept EPCT 0.3 0.26 -13.33 -69.41 109Astex Pharma ASTX 1.67 1.69 1.20 -24.89 1164 Exelixis EXEL 4.21 4.45 5.70 -48.20 6628Auxilium Pharma AUXL 17.51 18.39 5.03 -13.30 3253 Flamel Tech FLML 4.08 5.15 26.23 -26.01 352Avanir AVNR 2.37 2.3 -2.95 -42.64 10150 Forest Labs FRX 29.89 29.66 -0.77 -7.23 9587Avax Tech AVXT 0.04 0.04 0.00 -11.11 588 Furiex Pharma FURX 17.3 16.63 -3.87 15.09 529AVEO Pharma AVEO 16.88 16.96 0.47 15.37 1156 Galena Biopharma GALE 0.67 0.57 -14.93 -78.41 3442AVI BioPharma AVII 0.69 0.71 2.90 -67.13 2497 Generex Biotech GNBT 0.09 0.33 266.67 11.90 2397Bellus Health BLUS 0.04 0.04 0.00 -74.19 495 Genomic Health GHDX 28.07 28.09 0.07 28.38 864BG Medicine BGMD 4.22 3.85 -8.77 -53.61 114 GenVec GNVC 2.75 2.76 0.36 -51.58 143BioCryst Pharma BCRX 2.94 2.66 -9.52 -48.15 571 Geron GERN 1.7 1.59 -6.47 -69.48 4071Biodel BIOD 0.61 0.67 9.84 -65.10 756 Gilead Sciences GILD 39.4 39.01 -0.99 7.58 47914Biodelivery Sci BDSI 1.05 1.08 2.86 -70.81 801 GTx GTXI 3.02 2.87 -4.97 3.99 578Biogen Idec BIIB 113.24 112.95 -0.26 69.09 5638 Harvard Bio HBIO 4.21 4.02 -4.51 -0.99 432BioMarin Pharma BMRN 34.53 33.63 -2.61 23.78 5716 Human Genome HGSI 7.57 7.54 -0.40 -68.84 26280Bio-path Holdings BPTH 0.35 1.2 242.86 233.33 53 Idenix Pharma IDIX 8.22 8.13 -1.09 62.93 3291BioSante Pharma BPAX 2.52 2.45 -2.78 47.59 7997 Idera Pharma IDRA 1.25 1.08 -13.60 -62.76 160Cadence Pharma CADX 4.45 3.91 -12.13 -48.14 1735 ImmunoCellular IMUC 1.43 1.45 1.40 6.62 76Cardiome CRME 2 2.02 1.00 -69.16 704 ImmunoGen IMGN 12.23 12 -1.88 27.39 2368 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. 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  • 32. MONDAY, DEC. 12, 20 1 1 BIOWORLD® INSIGHT PAGE 32 OF 32Company Symbol Close Close %Change Vol Company Symbol Close Close %Change Vol 12/2 12/9 WK YTD (000) 12/2 12/9 WK YTD (000)Immunomedics IMMU 3.32 3.39 2.11 -5.83 1194 Tranzyme TZYM 3.24 3.05 -5.86 -23.75 35Incyte INCY 14.24 13.6 -4.49 -18.90 10132 Trinity Biotech TRIB 10.1 10.42 3.17 17.87 240Infinity Pharma INFI 9.49 9.78 3.06 61.65 478 Trius Therap TSRX 7.46 7.23 -3.08 100.00 684Inhibitex INHX 15.51 14.74 -4.96 466.92 10629 Unigene UGNE 0.89 0.51 -42.70 -23.88 1659InterMune ITMN 18.65 17.3 -7.24 -53.13 4912 United Therap UTHR 41.67 42.02 0.84 -33.65 5246Ironwood Pharma IRWD 11.94 12.28 2.85 18.65 1184 Vanda Pharma VNDA 4.82 4.85 0.62 -48.29 618ISIS Pharma ISIS 7.27 7.13 -1.93 -29.75 2614 Ventrus Biosciences VTUS 8 8.01 0.12 21.00 43Keryx Biopharma KERX 2.65 2.59 -2.26 -44.54 5155 Vermillion VRML 1.28 1.22 -4.69 -83.56 465Lexicon LXRX 1.2 1.27 5.83 -14.77 2977 Vertex Pharma VRTX 29.25 29.37 0.41 -16.89 15038Ligand Pharma LGND 11.67 12.27 5.14 37.71 575 Vical VICL 4.69 4.58 -2.35 123.41 5188MannKind MNKD 3.04 2.92 -3.95 -64.08 2334 ViroPharma VPHM 23.55 23.78 0.98 35.11 4743Map Pharma MAPP 13.61 13.73 0.88 -17.59 392 Vivus VVUS 10.52 10.68 1.52 12.07 6584Maxygen MAXY 5.65 5.59 -1.06 41.88 432 XenoPort XNPT 4.83 3.94 -18.43 -54.50 1603MDRNA MRNA 0.15 0.14 -6.67 -90.97 10134 Zalicus Inc ZLCS 0.95 1.15 21.05 -29.45 6164Metabolix MBLX 4.94 4.33 -12.35 -63.61 834 Zogenix ZGNX 1.84 1.69 -8.15 -70.19 736Micromet MITI 6.52 7.01 7.52 -15.24 3246Momenta Pharma MNTA 16.42 15.5 -5.60 3.20 3772Myrexis MYRX 2.73 2.79 2.20 -34.35 983Myriad Genetics MYGN 21.29 20.38 -4.27 -11.12 2245Nabi Biopharma NABI 1.78 1.81 1.69 -69.06 1790Nanogen NGEN 0.01 0.01 0.00 17.65 409Nektar Therap NKTR 4.92 5.29 7.52 -59.18 7692Neurocrine Biosci NBIX 6.85 7.45 8.76 -3.12 1473 LONDON STOCK EXCHANGENeurogesX NGSX 1.01 1.07 5.94 -83.07 1033 Company Symbol 12/2 12/9 %WK %YTD VolumeNovaBay NBY 1.14 1.05 -7.89 -37.13 774Novavax NVAX 1.46 1.43 -2.05 -43.92 3350NPS Pharma NPSP 6.03 6.23 3.32 -22.32 3489 Antisoma ASM 1.7 1.73 1.76 -94.76 2127NuPathe PATH 2.2 2.24 1.82 -75.28 126 Ark Therapeutics AKT 3.6465 3.8 4.21 -72.95 1664Omeros OMER 3.93 4.04 2.80 -51.67 348 Asterand ATD 2.75 2.45 -10.91 -86.83 254OncoGenex Pharma OGXI 11 11.57 5.18 -29.67 138 Oxford Biomedica OXB 4.97 4.87 -2.01 -56.71 1583Oncothyreon ONTY 6.86 8.25 20.26 152.29 5461 Phytopharm PYM 7.5 8.49 13.20 -21.68 433Onyx Pharma ONXX 43.81 40.63 -7.26 9.96 7742 Puricore PURI 24 25.9 7.92 93.28 35Opko Health OPK 5.1 4.82 -5.49 29.92 5911 Renovo Group RNVO 15.92 15.755 -1.04 -45.48 1963Optimer Pharma OPTR 11.71 12.12 3.50 5.39 4208 SkyePharma SKP 44.5 41.5 -6.74 -51.72 68OraSure Tech OSUR 9.5 9.39 -1.16 57.29 1446 Vernalis plc VER 19 19 0.00 -78.16 569Orexigen OREX 1.71 1.74 1.75 -78.68 1441 Note: Prices are denoted in pence.Osiris Thera OSIR 4.76 4.94 3.78 -37.78 271Oxis International OXIS 0.04 0.04 0.00 -70.37 107Pacific Biosciences PACB 2.73 2.67 -2.20 -83.22 1306Pacira Pharma PCRX 7.81 8.62 10.37 22.79 1136Pain Therapeutics PTIE 3.83 3.69 -3.66 -44.84 563Palatin Tech PTN 0.54 0.53 -1.85 -58.91 199 TORONTO STOCK EXCHANGEPeregrine Pharma PPHM 0.97 1.17 20.62 -46.82 12005 Company Symbol 12/2 12/9 %WK %YTD VolumePharmacyclics PCYC 15.63 12.39 -20.73 98.56 7134Pharmasset VRUS 128.44 129 0.44 193.58 10278Pharmos PARS 0.06 0.06 0.00 -40.00 24 Adherex Technol AHX 0.45 0.35 -22.22 677.78 13Poniard Pharma PARDD 4.88 3.85 -21.11 -83.41 159 Allon Therap NPC 0.19 0.2 5.26 -37.50 592Pozen POZN 3.93 3.98 1.27 -39.42 712 BELLUS Health BLU 0.04 0.04 0.00 -77.78 580Progenics Pharma PGNX 7 7.63 9.00 41.04 849 Bioniche Life Sci BNC 0.49 0.48 -2.04 -14.29 173Protalix Biothera PLX 6.13 5.37 -12.40 -46.19 2201 Cangene CNJ 1.8 1.81 0.56 -64.51 226Protein Design PDLI 6.3 6.09 -3.33 -2.09 9599 Isotechnika ISA 0.12 0.11 -8.33 -33.33 116Qiagen QGEN 14.53 14.34 -1.31 -26.27 8174 Lorus Therap LOR 0.2 0.2 0.00 207.69 32QLT Inc. QLTI 6.84 6.74 -1.46 -5.87 410 Medicago MDG 0.53 0.57 7.55 -14.93 926Raptor Pharma RPTP 5.38 6.19 15.06 70.05 2824 MethylGene MYG 0.32 0.31 -3.13 10.71 445Regeneron Pharma REGN 58.28 53.38 -8.41 59.92 6782 Oncolytics Biotech ONC 4.28 4.09 -4.44 48.73 263Repligen RGEN 3.31 3.3 -0.30 -30.19 47 Protox Therap PRX 0.34 0.31 -8.82 -63.53 132Rexahn Pharma RNN 0.49 0.51 4.08 -55.65 907 Resverlogix RVX 1.19 1.4 17.65 -42.15 625Rigel Pharma RIGL 7.77 7.67 -1.29 0.92 1104 SemBioSys Gen SBS 0.06 0.06 0.00 -70.00 65Sagent Pharma SGNT 20.98 20.96 -0.10 9.22 355 Tekmira Pharma TKM 1.7 1.66 -2.35 78.49 240Sangamo Biosci SGMO 2.98 2.77 -7.05 -59.62 1151 Thallion Pharma TLN 0.11 0.14 27.27 27.27 273Santarus SNTS 2.94 3.09 5.10 -6.08 948 Theratechnologies TH 2.24 2.34 4.46 -47.18 1053Savient Pharma SVNT 2.22 2.13 -4.05 -81.03 6339 YM BioSciences YM 1.4 1.61 15.00 11.03 141SciClone Pharma SCLN 4.51 4.54 0.67 7.08 782Seattle Genetics SGEN 16.41 18.09 10.24 17.93 7356 Note: Prices are denoted in Canadian dollars.Sequenom SQNM 4.17 3.89 -6.71 -52.21 12835SIGA Tech SIGA 2.06 2.43 17.96 -82.64 4269Soligenix SNGX 0.03 0.03 0.00 -84.21 NOTES:Somaxon Pharma SOMX 0.74 0.7 -5.41 -78.19 665 Trading volumes for Nasdaq, Amex and NYSE are recorded as the totalSpectrum Pharma SPPI 14.04 13.83 -1.50 95.34 11638 number of shares traded (in thousands) on a weekly basis (cumulativeStemCells STEM 1.81 1.65 -8.84 -37.50 185Sucampo Pharma SCMP 3.79 4.07 7.39 6.27 109 Monday through Friday); the weekly and YTD % changes are from IPOSynta Pharma SNTA 4.04 4.83 19.55 -21.08 869 completion, where applicable.Talon Thera TLON 0.49 0.46 -6.12 -4.17 86 Average Percent Change Week: +2.54%Targacept TRGT 7.58 7.48 -1.32 -71.97 2257Telik TELK 0.29 0.26 -10.34 -67.70 181 Range: -42.70% to +266.67%; Number Of Companies: 203Tengion TNGN 0.4 0.47 17.50 -81.50 595 (does not include LSE or TSX; not market weighted)Theravance THRX 23.43 23.34 -0.38 -8.65 1658 Average Percent Change YTD: -11.39%Titan Pharma TTNP 1.33 1.11 -16.54 -9.02 99Transcept Pharma TSPT 8 7.27 -9.13 -3.71 1776 Range: -90.97% to +466.92%; Number Of Companies: 203Transgenomic TBIO 1.35 1.27 -5.93 78.87 937 (does not include LSE or TSX; not market weighted) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. 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