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

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CNDO 11-12-11 BioWorld Insight

  1. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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.

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