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  1. 1. BIOTECH’S MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARSWEDNESDAY VOLUME 22 , NO. 41MARCH 2, 201 1 PAGE 1 OF 8‘Buy American’ FDA OKs Narrower IndicationDaiichi Sankyo Bolsters Pipe for Forest Lab’s COPD Drugwith $935M Plexxikon Buy By Marie PowersBy Tom Wall BioWorld Today Contributing WriterStaff Writer After a long and winding road, the FDA has at last given In the latest acquisition of a U.S. biotech with promising Forest Laboratories Inc. the green light to market Daliresponcology offerings by a Japanese company, Daiichi Sankyo (roflumilast) in the U.S. to reduce the risk of chronicCo. Ltd., of Tokyo, bought Plexxikon Inc., of Berkeley, Calif., obstructive pulmonary disease (COPD) exacerbationsand its promising melanoma drug PLX4032, for $805 million in patients with severe COPD associated with chronicup front and near-term milestones of $130 million linked to bronchitis and a history of such flare-ups, which mayapproval of PLX4032. include breathlessness, chronic cough and excessive In recent years, Japanese drugmakers have made a production of phlegm leading to medical intervention,series of major U.S. oncology buys, including Eisai Co.’s $205 including hospitalization.million September 2006 purchase of Ligand Pharmaceuticals In 2009, New York-based Forest licensed the drug –Inc.’s three-drug cancer portfolio, a $325 million buyout of marketed as Daxas in the European Union and CanadaMorphotek in March 2007 and, earlier this year, $200 million – from privately held Nycomed GmbH, of Zurich,in research funding to start-up H3 Biomedicine; Takeda Switzerland, in a deal potentially worth more than $500Pharmaceutical Co.’s $8.8 billion purchase of Millennium million. (See BioWorld Today, Aug. 11 , 2009.)Pharmaceuticals Inc. in April 2008; and Astellas Pharma Nycomed had initially submitted the new drug See Plexxikon, Page 2 See Forest, Page 4Novavax, VaxInnate Get Financings RoundupWelcome Shot in the Arm Investors to the Rescue asBy Mari Serebrov Tengion Pulls in $31M PIPEWashington Editor By Jennifer Boggs WASHINGTON – Two government contracts that could be Assistant Managing Editorworth up to nearly $200 million each will inoculate Novavax After a roller coaster February, in which shares ofInc. and VaxInnate Inc. against some of the financial risks Tengion Inc. rose on buyout speculation only to fall afterinvolved in getting their recombinant influenza vaccines the surge in trading activity scared off a potential acquirer,through clinical development. investors of the East Norriton, Pa.-based regenerative The cost-reimbursable, milestone-based contracts, medicine firm have stepped up with some much-neededawarded by the Department of Health and Human Services financing.(HHS), are for three years with a possible two-year extension. Tengion is bringing in $31 .4 million in a PIPE transactionNovavax, of Rockville, Md., will receive $97 million over in exchange for issuing about 1 1 . 1 million shares of commonthe first three years, which is enough to bring its seasonal stock and warrants to purchase about 10.5 million sharesinfluenza recombinant virus-like particle (VLP) vaccine to certain investors. One of those investors is Medtronicthrough late-stage trials to FDA licensure, John Trizzino, Inc., a Minneapolis-based medical device company thatNovavax senior vice president of business development, picked up, in exchange, a right of first refusal on thetold investors Tuesday. preclinical Neo-Kidney Augment program through Oct. 31, If the extension is granted, the total contract would 2013. See Influenza, Page 5 See Financings Roundup, Page 6INSIDE: OTHER NEWS TO NOTE: GERON, INSMED, IRX THERAPEUTICS, KENPHARM .....7 CLINIC ROUNDUP: SEATTLE GENETICS, SYNDAX, TOBIRA THERAPEUTICS ...........8 To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at
  2. 2. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 2 OF 8PlexxikonContinued from page 1Inc.’s $4 billion acquisition of OSI in May 2010 and several Coming Thursday indeals leading up to it. BioWorld Perspectives: The pattern is not a coincidence, Plexxikon CEO K.Peter Hirth told BioWorld Today, but rather how Japanese ‘All My Clones,’companies must compete because of a shortage of biotech BioWorld’s Biotech-Themed Soap Operainnovation in Japan. “It’s particularly true in oncology,” Hirth said. “The In this week’s “All My Clones,” read about Cappuccinoinnovation gap in Japanese pharma in the last 10 years is just Pharmaceuticals’ plans to investigate placebos andmind boggling. There is not enough innovation happening some unethical actions by board members. If youin Japan by itself. They have to buy it. Biotech did not take off missed previous episodes of BioWorld’s biotech-themedin Japan. It has been a big challenge for the Japanese.” soap opera, look to the top of the story for a link to the PLX4032 captured the attention of the cancer world “All My Clones” archives.during the past year and a half. Most recently, an interimanalysis of a Phase III trial released in January showed both BioWorld Perspectives is a weekly e-zine thatan overall survival benefit and a progression-free survival offers unique viewpoints on developments withinbenefit for patients taking the drug as a first-line agent. (See the biotechnology industry. And the best part is, it’sBioWorld Today, Jan. 20, 2011 , and Nov. 8, 2010.) completely free. Opt-in at The 675-patient BRIM 3 study was the first wherePLX4032, which also goes by the name RG7204, was usedas a first-line agent for BRAF mutation positive metastaticmelanoma. Patients were randomized to receive either oralPLX4032 twice daily, or the current standard of care, whichconsists of intravenous dacarbazine every three weeks. Stock MoversHirth said at the time the data were so strong that the study 03/01/1 1was terminated early, and patients in the control arm have Company Stock Changebeen given the option to cross over onto PLX4032. Plexxikon, a privately held firm, and development Nasdaq Biotechnology -$6.44 -0.7%partner Roche AG, planned to file for market approval Novavax Inc. +$0.36 +13.9%in the U.S. and Europe this year, along with a filing for thecompanion diagnostic also being co-developed by the Repros Therapeutics Inc. +$1 .29 +28.7%partners. It is an extremely quick progression. The Phase I RXi Pharmaceuticals Corp. -$0.47 -27.7%trial of PLX4032 began in December 2006. The results of that Vivus Inc. -$0.83 -10.9%trial, which were published in 2010, prompted Plexxikon andRoche to move the drug directly into pivotal testing. The firstpatient was enrolled in the BRIM3 study in January 2010. (Biotechs showing significant stock changes Tuesday) See Plexxikon, Page 3 SUBSCRIBER INFORMATION BioWorld® Today (ISSN# 1541-0595) is published every business day by AHC Media, 3525 Piedmont Road, Building Six, Suite 400, Atlanta, GA 30305 U.S.A. Opinions expressed are not necessarily those Please call (800) 688-2421 to subscribe or if of this publication. Mention of products or services does not constitute endorsement. BioWorld® and Bio- you have fax transmission problems. Outside World® Today are trademarks of AHC Media, a Thompson Publishing Group company. Copyright © 2011 U.S. and Canada, call (404) 262-5476. Our AHC Media. All Rights Reserved. No part of this publication may be reproduced without the written con- customer service hours are 8:30 a.m. to 6:00 sent of AHC Media. (GST Registration Number R128870672). p.m. EST. Lynn Yoffee, (404) 262-5408 ATLANTA NEWSROOM: Managing Editor: Lynn Yoffee. Assistant Managing Editor: Jennifer Boggs. Jennifer Boggs, (404) 262-5427 Senior Staff Writer: Karen Pihl-Carey. Senior Production Editor: Ann Duncan. Staff Writer: Tom Wall. Anette Breindl, (518) 595-4041 Trista Morrison, (858) 901-4785 WASHINGTON BUREAU: Washington Editor: Mari Serebrov. Mari Serebrov, (703) 678-7376 WEST COAST BUREAU: Staff Writer: Trista Morrison. Tom Wall, (404) 262-5417 EAST COAST BUREAU: Science Editor: Anette Breindl. BUSINESS OFFICE: Senior Vice President/Group Publisher: Donald R. Johnston. Senior Vice President/Group Publisher: Director of Product Management: Jane Cazzorla. Donald R. Johnston, (404) 262-5439 Marketing Manager: Sarah Cross. Internet: Account Representatives: Matt Hartzog, Chris Wiley, Scott Robinson. DISPLAY ADVERTISING: For ad rates and information, please call Stephen Vance at (404) 262-5511 or email him at REPRINTS: For photocopy rights or reprints, call our reprints department at (404) 262-5479. PRESS MATERIALS: Send all press releases and related information to
  3. 3. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 3 OF 8Plexxikon for pancreatic cancer. (See BioWorld Today, Sept. 11, 2006, andContinued from page 2 March 23, 2007.) Plexxikon has an agreement to co-promote PLX4032 In January of this year, Eisai Inc., of Woodcliff Lake, N.J.,in the U.S. with Roche’s U.S. commercial oncology the U.S. pharmaceutical operation of Tokyo-based Eisai Co.unit, Genentech. Daiichi Sankyo will retain the U.S. co- pledged as much as $200 million in research funding topromotion rights for PLX4032. Hirth said that he expects start-up H3 Biomedicine, of Cambridge, Mass., which hopesco-promotion will be, “pretty straightforward. It will just to develop oncology treatments genetically tailored tobe shared. Everybody knows everybody, and everybody is patients’ cancers. (See BioWorld Today, Jan. 31 , 2011 .)comfortable.” Takeda, of Osaka, acquired Millennium Pharmaceuticals Hirth said Plexxikon will continue on with similar Inc., of Cambridge, Mass., and its lucrative Velcade multiplestaff and budget. “They like the way we have developed myeloma product in April 2008 for $8.8 billion, renamingprojects, and they expect to get a number of additional the company Millennium: The Takeda Oncology Co. (Seedevelopment candidates,” he said, describing the 40-plus BioWorld Today, April 11, 2008.)person Plexxikon team as “good size, working well, a fine- Astellas, of Tokyo, bought OSI, of Melville, N.Y., for $4tuned racing engine.” billion in May 2010, boosting its oncology franchise and PLX4032, an orally active kinase inhibitor that targets adding OSI’s blockbuster cancer drug Tarceva (erlotinib) asan activating BRAF mutation, is also an example of the well as a U.S. sales force, a pipeline of small molecules andpower of personalized medicine. The drug is being co- discovery capabilities. (See BioWorld Today, May 18, 2010.)developed with a diagnostic test to identify patients whose A series of smaller deals led up to the OSI acquisition.tumors carry the V600 mutation. Such mutations account In December 2007 Astellas bought Agensys Inc., of Santafor about half of all melanoma cases. Monica, Calif., and a pipeline of early clinical-stage anticancer The extremely rapid hurtle down the clinical antibodies for $537 million; licensed prostate cancer drugdevelopment path also means that the conclusions about MDV3100 in a $765 million deal with Medivation Inc., ofPLX4032’s safety and efficacy are based on relatively San Francisco, in October 2009; and in December 2009 gotfew patients, and relatively few clinical events. Such acute myeloid leukemia drug AC220 in a potential $390small numbers and statistics can be shaky, but Plexxikon million deal with Ambit Biosciences Corp., of San Diego.executives have said that because of the strength of the If U.S. oncology biotechs have attracted attention fromdata, they are not concerned that the sample size is small. Japanese companies, so too, have selective kinase inhibitors Melanoma has proved a tough disease for drug been in the spotlight of late. (See BioWorld Insight, Feb. 28,developers in the past, with notable failures such as Genta 2011 .)Inc.’s Bcl-2 targeting antisense drug Genasense (oblimersen) Gilead Sciences Inc.’s recent buyout of Calistogaand tyrosine kinase inhibitor Nexavar (sorafenib) from Pharmaceuticals Inc. for $375 million in cash and up toOnyx Pharmaceuticals Inc. and Bayer AG. But there have $225 million in milestones for rights to PI3K delta isoformbeen bright spots in the space of late, with Bristol-Myers CAL-101 underscores the enthusiasm for small-moleculeSquibb Co.’s ipilimumab, acquired through its acquisition of kinase inhibition. A week earlier AVEO Pharmaceuticals Inc.Medarex Inc., showing promising survival data in a pivotal signed a potential $1 billion-plus partnership for tivozanib,study. (See BioWorld Today, June 8, 2010, and Nov. 8, 2010.) a Phase III-stage kinase inhibitor targeting VEGFR 1 , 2 and 3. Joji Nakayama, CEO of Daiichi Sankyo Co. Ltd., said (See BioWorld Today, Feb. 17, 2011 , and Feb. 23, 2011 .)in a statement that his company sees the acquisition of Kinase inhibitors have produced blockbusters likePlexxikon as an opportunity to accelerate the building of Gleevec (imatinib, Novartis AG) and near-blockbusters likethe company’s oncology franchise. Nexavar (sorafenib, Onyx Pharmaceuticals Inc. and Bayer Other major Japanese pharma firms, including Eisai, AG) and Sutent (sunitinib, Pfizer Inc.). ■Takeda and Astellas, have preceded Daiichi Sankyo with the“buy American” oncology strategy. Eisai in September 2006 purchased a three-drug cancer Clinic Roundupportfolio – Ontak (denileukin diftitox), for cutaneous T-celllymphoma; Targretin (bexarotene) gel and capsules, also for • Cytheris SA, of Paris, said a Phase IIa study of itsCTCL; and Panretin (alitretinoin) gel for Kaposi’s sarcoma, an HIV candidate, CYT109 (interleukin-7), provided evidenceAIDS-related skin cancer – from Ligand Pharmaceuticals Inc., of of efficacy and showed that interleukin-7 stimulated theSan Diego, for $205 million plus royalties. Eisai next acquired expansion of CD4 and CD8 T cells in peripheral blood.Morphotek, Inc., of Exton, Pa., in March 2007 in a $325 million Those results showed that interleukin-7 was important inbuyout. One of a few firms at the time that could make fully stimulating T-cell repopulation of lymphoid tissue in thehuman monoclonal antibodies in cell culture, Morphotek had GI tract. Cytheris presented the data at the 2011 Conferencea pair of clinical-stage drug candidates, MORAb 003, in Phase on Retroviruses and Opportunistic Infections being held inI/II trials for ovarian cancer, and MORAb 009, in Phase I trials Boston this week. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at
  4. 4. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 4 OF 8Forest warnings, but the FDA did not require a black-box warningContinued from page 1 for the drug.application for roflumilast in July 2009 with a proposed The company expects Daliresp to be available toindication as a maintenance treatment of COPD associated wholesalers in the second calendar quarter.with chronic bronchitis in patients at risk of exacerbations. Patients with COPD have largely depended on inhalersLast spring, however, FDA reviewers voiced concerns over to treat symptoms of the disease, so the approval ofsuicides linked to the drug. (See BioWorld Today, April 6, 2010.) Daliresp “is good news for [Forest] and validates the Subsequently, the FDA’s Pulmonary-Allergy Drugs company as differentiated from a regulatory perspective,”Advisory Committee reversed its earlier endorsement of Leerink Swann Healthcare Equity Research analyst Seamusthe drug, voting against the drug’s approval. (See BioWorld Fernandez wrote in a research note.Today, April 8, 2010.) Jefferies & Co. analyst Corey Davis was even Thus, it was hardly a surprise when the agency issued more ebullient, describing Daliresp as “Forest’s mosta complete response letter for roflumilast. (See BioWorld underappreciated pipeline drug” and predicting “blockbuster-Today, May 19, 2010.) like potential” for the drug. “We’ve seen the label and it looks In the midst of the FDA’s review, Forest had submitted great,” Davis wrote in a research note. “While there area new application – too late in the game to be considered some warnings, there are no black-box warnings, no [riskoriginally by the FDA panel – for the narrower indication evaluation and mitigation strategy], and as we expected, useand a new warning about neuropsychiatric events. At the is being restricted to the most severe patients.”time, analysts suggested the changes would pass muster Though Forest has not disclosed pricing information,with the FDA, which turned out to be the case. Davis expects Daliresp to be priced competitively with Officials at Forest were traveling and did not respond to Spiriva (tiotropium bromide, Pfizer Inc.).requests for an interview. “Because of Daliresp’s unique mechanism of action, we Daliresp, an oral tablet taken once daily, becomes the don’t see it competing directly vs. the inhalers, and as anfirst FDA-approved selective phosphodiesterase-4 inhibitor. oral med, we see great utility as a potential combo agent,”The compound’s specific mechanism of therapeutic action Davis wrote. Although the “consensus” 2016 Street estimatein COPD is not well defined, but is thought to be related to for Daliresp is $292 million, compared to Jefferies’ $523the effects of increased intracellular cyclic AMP in lung cells. million estimate, “with Forest likely to go ‘whole hog’ on Forest said the efficacy and safety of Daliresp were the Daliresp launch, and use of the drug likely to moveevaluated in eight clinical studies including 9,394 adult upstream into milder COPD patients, we think our estimatespatients. Of these, 4,425 were treated with Daliresp 500 mcg. could prove to be conservative,” he added. Two one-year trials enrolled patients with severe COPD Although shares of Forest (NYSE:FRX) gained more thanassociated with chronic bronchitis, at least one COPD $1 before the market opened Tuesday, sending the stockexacerbation in the previous year and at least a 20 pack- from Monday’s close of $32.40 to $33.64 at Tuesday’syear smoking history. In these trials, long-acting beta- opening bell, the stock lost ground during another brutalagonists and short-acting anti-muscarinics were used by day for the market, closing at $32.02, down 38 cents. ■44 percent and 35 percent of patients treated with Dalirespand 45 percent and 37 percent of patients treated withplacebo, respectively. Other News To Note One of these trials randomized 1 ,525 patients (765 onDaliresp) and the other randomized 1 ,571 patients (772 on • Genzyme Corp., of Cambridge, Mass., completedDaliresp). Daliresp reduced the rate of moderate or severe the sale of its pharmaceutical intermediates businessexacerbations, compared to placebo, by 15 percent in one to International Chemical Investors Group (ICIG). Thetrial and by 18 percent in the other. acquired business, which includes all but the drug The most common adverse reactions, occurring in less delivery technologies portion, has been renamed Cordenthan or equal to 10 percent of patients receiving Daliresp Pharma Switzerland LLC and will operate as part of ICIG’sin the eight trials, included diarrhea, nausea, weight loss, pharmaceutical business within the Corden Pharma group.headache, back pain, influenza, insomnia, dizziness and Financial terms are not material to Genzyme and weredecreased appetite. Daliresp was associated with an not disclosed. As part of the agreement, Corden Pharmaincrease in psychiatric adverse reactions (5.9 percent of contracted with Genzyme to provide materials neededpatients treated with Daliresp vs. 3.3 percent treated with for the production of eliglustat tartrate, an investigationalplacebo). During clinical trials, the company said one patient treatment for Gaucher disease Type 1 that’s in Phase IIItreated with Daliresp committed suicide and two attempted trials, and for the manufacture of other treatments. Thesuicide, compared to one patient treated with placebo who sale completes the three divestitures announced last yearconsidered suicide. as part of Genzyme’s effort to sharpen its focus on core The risk of suicide is mentioned among the label businesses. (See BioWorld Today, July 22, 2010.) To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at
  5. 5. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 5 OF 8Influenza LGLS will be responsible for conducting clinical trialsContinued from page 1 and getting the vaccines licensed in these areas. Novavaxbe $179. 1 million. The additional funding would support will provide technology transfer and manufacturing supportthree Phase I and II trials of a pandemic H5N1 VLP vaccine, for a manufacturing facility LGLS is building in South Korea.Trizzino added. Novavax will receive an up-front payment with potential VaxInnate, of Cranbury, N.J., will receive $117.9 million milestones, Singhvi said. It also will receive double-digitover the first three years, which can be extended for royalty payments from LGLS’ commercial sales of VLPa total contract value of $196.6 million. The five-year vaccines in South Korea and the emerging markets. Termsfunding would enable the privately held company to take of those payments were not disclosed.its seasonal and pandemic influenza vaccines up through At VaxInnate, the HHS contract will “put the company onPhase III development. more solid footing” as it begins the long process of clinical “This gives us the financial security to carry out this trials for its pandemic and seasonal flu vaccines, Sethkind of long-haul development program,” Alan Shaw, Rudnick, a physician, venture partner at Canaan Partners,VaxInnate’s chief scientific officer, told BioWorld Today. “It and board member of VaxInnate, told BioWorld Today.allows us to breathe.” Canaan invested in VaxInnate four years ago to support For Novavax, news of the HHS contract came on the the development of a vaccine technology with the flexibilityheels of a licensing agreement with LG Life Sciences to adapt quickly to unexpected changes in the flu.(LGLS), of Seoul, South Korea, that could open markets in The HHS funding will allow the company to hire 20several emerging countries. The back-to-back news is additional scientists and technical staff, a 44 percent“transformational for the company,” Novavax President and increase to its 45-person work force, Shaw said, which willCEO Rahul Singhvi told investors. “Today, we are from every help with increased reporting requirements and trials.perspective – scientific, financial and commercial – a much Using VaxInnate’s Toll-like receptor (TLR) technology,stronger and more competitive company in the vaccine initial trials will evaluate the components and combinationsarena.” of what will ultimately be several different pandemic flu That strength was reflected in the value of Novavax vaccines and a seasonal quadrivalent flu vaccine.(NASDAQ:NVAX) shares, which jumped nearly 14 percent, or The TLR technology genetically fuses vaccine antigens36 cents, Tuesday, closing at $2.96. to the bacterial protein flagellin, producing a synthetic Piper Jaffray’s Edward Tenthoff maintained his product that can be quickly and inexpensively reproduced.overweight rating for Novavax but increased the share Unlike traditional egg-based vaccines, VaxInnate’s vaccinesprice target from $3 to $4. can be frozen and stored for long periods of time, Shaw The HHS contracts, awarded through its Biomedical explained.Advanced Research and Development Authority, are part Because the vaccine is synthetic, VaxInnate doesn’tof a national pandemic vaccine preparedness strategy have the burden of growing the influenza virus needed forthat includes the advanced development of new types of traditional vaccines.influenza vaccines. The strategy also calls for expanding The technology “sort of short circuits many of theand diversifying domestic influenza vaccine production issues” involved with egg-based vaccines, Shaw added.and establishing and testing stockpiles of pre-pandemic For instance, using its technology, VaxInnate can turnvaccine. around a new strain in less than three months and produce Under its contract, Novavax plans to conduct a dose- millions of doses in a single six-day production run.confirming Phase II trial of its seasonal vaccine in 500- VaxInnate already completed a series of Phase I/II trials600 subjects in the last half of the year. A Phase III study using VAX 125 and VAX128, prototype vaccines for bothis expected to begin late next year, with a biologic license seasonal and pandemic Type A1 flu vaccines.application submitted to the FDA in 2014, said Trizzino. The most recent trial, involving three forms of the The company also will develop a detailed plan for a VAX128 vaccine, confirmed the safety and efficacy of themanufacturing facility that has the capability to produce vaccine in elderly subjects, who are less responsive to flufinished vaccine within 12 weeks and at least 50 million vaccines due to the effects of aging on the immune system.doses within six months of an influenza pandemic (See BioWorld Today, May 7, 2009.)declaration. Under its contract with HHS, VaxInnate will be able to As for the licensing agreement, Novavax granted broaden the flu strains its technology can address, RudnickLGLS an exclusive license to manufacture, develop and noted. ■commercialize influenza vaccines using its recombinantVLP technology in South Korea and a nonexclusive license BioWorld is now on Twitter!in Africa, Central America, South America and other parts Stay Connected, Follow Us on Twitter!of Asia. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at
  6. 6. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 6 OF 8Financings Roundup capital and other general corporate purposes.Continued from page 1 Piper Jaffray & Co. served as sole lead placement agent, Shares of Tengion (NASDAQ:TNGN) fell 23 cents while Leerink Swann LLC served as co-placement agent.Tuesday to close at $2.49. In other financings news: The stock traded as high as $5.51 last month – trading • NeuroPhage Pharmaceuticals Inc., of Cambridge,volume peaked at 25 times the average in mid-February – Mass., said it secured $12.4 million in Series B least in part on rumors that the firm was in acquisition The funding will be used for Phase I trials of its Alzheimer’snegotiations. candidate, NPT001 . Mérieux Développement was the lead Only days later, Tengion confirmed that it had been in investor, with Shire LLC participating as a new investor.talks about a stock-for-stock merger with an undisclosed NeuroPhage’s total capital raised since 2007 is $19.6publicly traded company, but said the potential acquirer million.ended negotiations due to the unexpected increase in • RXi Pharmaceuticals Corp., of Worcester, Mass.,trading price and volume. priced a public offering of 6 million units at $1 .35 each for Tengion had about $12.5 million on its balance sheet gross proceeds of $8. 1 million. Each unit consists of oneas of Sept. 30, which it said would carry the firm into April. share of common stock, a 13-month warrant to purchaseIf a possible sale or alternative financing transaction had 0.50 of a share of stock at an exercise price of $1 .70 eachnot come through, the company might have had to look at and a five-year warrant to purchase 0.50 of a share at anwinding down operations only a year after it succeeded in exercise price of $1 .87 per share. The RNAi firm expects netgoing public. proceeds to total about $7.3 million and said those funds Though it had to take a haircut on the price, Tengion’s will be used for general corporate purposes. Lazard Capitalinitial public offering (IPO) was a major coup for the life Markets LLC is acting as sole book-running manager, andsciences industry, as the first true early stage biotech to Roth Capital Partners LLC is acting as co-manager. Sharessqueeze out of the current IPO window. Unfortunately, the of RXi (NASDAQ:RXII) fell 47 cents, or 27.7 percent, to close$30 million offering was less than the firm had hoped to Tuesday at $1 .23. ■raise, forcing it to return to investors for more cash. (SeeBioWorld Today, April 12, 2010.) And Tengion isn’t alone. Other new market entrantshave had to seek cash shortly after completing IPOs. Clinic RoundupAnthera Pharmaceuticals Inc. and AVEO PharmaceuticalsInc., for instance, both completed PIPE financings about six • Neuralstem Inc., of Rockville, Md., dosed its firstmonths after going public. (See BioWorld Today, Sept. 22, subject in a Phase Ia trial of NSI-189, for major depressive2010, and Nov. 1 , 2010.) disorder. The company said that NSI-189 stimulates new Proceeds from Tengion’s PIPE are expected to fund neuron growth to restore brain physiology. In the trial,R&D activities, primarily related to its Neo-Urinary Conduit healthy volunteers will be given a single oral dose of NsI-and Neo-Kidney Augment programs. Both are based on the 189. After the maximum tolerated single dose is determined,company’s platform, which involves isolating progenitor Phase Ib will commence with escalating daily doses for 28cells from a patient’s tissue sample, then expanding those days in patients with major depressive disorder.cells and placing them on a bioabsorbable scaffold within • NPS Pharmaceuticals Inc., of Bedminster, N.J., hasa bioreactor to grow a new organ. That new organ is then randomized its last patient in a Phase III trial of NPS558implanted into the patient. for hypoparathyroidism. NPS558 is a bioengineered form The Neo-Urinary Conduit is the lead program. It’s of human parathyroid hormone. The study, designateddesigned to use smooth muscle cells to grow tissue for REPLACE, includes a total of 135 patients. Top line results arebladder cancer patients who need urinary diversion after expected by the end of the year.bladder removal. Tengion has initiated a five-person trial • Poniard Pharmaceuticals Inc., of San Francisco,and, in November, completed the one-month postoperative received approval from the Chinese State Food and Drugclinical assessment for the first patient. That study is aimed Administration to conduct two Phase III trials of its leadat providing safety data and helping investigators optimize product candidate, picoplatin, in the treatment of second-the surgical technique. line small-cell lung cancer (SCLC) and second-line ovarian In preclinical development, Tengion has Neo-Kidney cancer. The approval will allow Poniard to include ChineseAugment, which is designed to improve renal function and clinical sites in global registration trials within those diseaseprevent or delay dialysis or transplant. Other early stage settings. Picoplatin, a platinum-based chemotherapeuticprograms include Neo-GI Augment for intestinal issues and agent, has demonstrated promising clinical activity in SCLCNeo-Vessel Replacement as a graft alternative. and ovarian cancer, the company said. Shares of Poniard Remaining funds from the offering, set to close prior to (NASDAQ:PARD) gained 5 cents, or 14.3 percent, to close atMarch 4, will go toward debt repayment and for working 40 cents Tuesday. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at
  7. 7. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 7 OF 8 • Shareholders of Insmed Inc., of Richmond, Va., Other News To Note approved a stock conversion and a 1-for-10 reverse stock split of the company’s common stock. As a result, 91 .7 • Geron Corp., of Menlo Park, Calif., reported million outstanding shares of Series B preferred stock werepreclinical study data showing positive effects of GRNCM1 , immediately converted into common stock. Insmed intendsthe company’s cardiomyocyte product derived from to effect the reverse stock split as of the close of businesshuman embryonic stem cells, in a small animal model of Wednesday. In December, the company was given untilacute heart damage. Administering GRNCM1 by injection June 13 to comply with Nasdaq’s $1 minimum bid price rule.into the heart resulted in greater resistance to induced Shares of Insmed (NASDAQ:INSM) lost 4 cents Tuesday, toarrhythmias, halted adverse cardiac remodeling and close at 50 cents.preserved mechanical function compared to controls. The • IRX Therapeutics Inc., of New York, signed aresults suggested that GRNCM1 positively affects cardiac research collaboration with an undisclosed research-function through several mechanisms, leading to overall based pharmaceutical firm in which tumor specimens fromincreased cardiac output and decreased arrhythmias in the subjects treated with IRX-2 and control subjects will beacute infarct setting. Geron is developing GRNCM1 for the analyzed by the collaborating company using gene analysistreatment of myocardial disease. The data were presented technology. The goal is to characterize the action of IRX-at the Keystone Symposium on Mechanisms of Cardiac 2 immunotherapy at the molecular level and assess theGrowth, Death and Regeneration in Colorado. potential effects of the drug on the tumor microenvironment. IRX-2 was tested in a Phase II trial enrolling patients with newly diagnosed head and neck squamous cell cancer who Wondering What You received IRX-2 prior to surgery. • KemPharm Inc., of North Liberty, Iowa, filed an Missed in BioWorld Insight? answer and counterclaims against Shire LLC, a subsidiary of Shire plc, of Dublin, Ireland. The counterclaims were Reverse Merger Interest Dims filed in response to a lawsuit filed in Virginia on Sept. as IPO Window Creaks Open 30, 2010, by Shire against KemPharm and Travis Mickle, It became the exit du jour two years ago when the KemPharm’s president and CEO, alleging breach of contract initial public offering window slammed shut, but now and intentional interference with contract in relation to the reverse merger seems to have regained its previous Mickle’s prior employment with New River Pharmaceuticals, less-than-favorable reputation. To find out whether that which Shire acquired in 2007. KemPharm’s answer denies reputation is warranted, BioWorld Insight takes a look the allegations of Shire’s complaint, raises affirmative back at some of the highly touted reverse merger deals defenses and makes claims for unfair competition, antitrust of 2007 – 2008 and finds out where they are now. violations and other relief. The antitrust claims assert that the Shire lawsuit is baseless and designed to interfere with Genzyme Aces in Hand May KemPharm’s business. (See BioWorld Today, Feb. 21 , 2007.) Ward Off Pharma Culture War • MediGene AG, of Martinsried, Germany, completed the Sanofi-Aventis SA said it intends to preserve the transfer of exclusive European rights to Eligard (leuprolide biotech culture at Genzyme Corp. after the acquisition, acetate) for the treatment of hormone-dependent prostate but can suit coats and lab coats really get along? As the cancer to Astellas Pharma Europe Ltd., of London, part large-cap biotech players fold one by one, Genzyme of Astellas Pharma Inc. The transaction triggered a second may have the cards to stay in the game. milestone payment to MediGene, in the amount of €15 million (US$20.65 million). Effective March 1 MediGene also will Calistoga Buyout Spotlights receive a 2 percent share in net European sales of Eligard. The Selective Kinase Perception transfer of rights terminated MediGene’s costs and obligations Kinase inhibition has been a biotech staple for the associated with future supplies of Eligard to Astellas. past decade, but the trick has been trying to create small • Oncothyreon Inc., of Seattle, reported preclinical molecules selective enough to avoid off-target effects. data demonstrated the effect of its PX-866, a small-molecule Recent deals such as Gilead Sciences Inc.’s buyout of compound, and its metabolite as an irreversible inhibitor Calistoga Pharmaceuticals Inc. suggest the industry is of phosphatidylinositol 3-kinase (PI3K). The data were making impressive headway. presented at an American Association of Cancer Research special conference on targeting PI3K/mTOR signaling. Take BioWorld Insight for a test drive. Call (404) Oncothyreon is conducting a broad development program 262-5476 or (800) 688-2421 and mention editor Trista of PX-866, including trials evaluating the compound as Morrison for a free trial subscription. a single agent and in combination with other agents in multiple cancer types. To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at
  8. 8. WEDNESDAY, MARCH 2, 201 1 BIOWORLD® TODAY PAGE 8 OF 8 Other News To Note Clinic Roundup • Pfizer Inc., of New York, completed its purchase of • Seattle Genetics Inc., of Bothell, Wash., and Millennium,King Pharmaceuticals Inc., of Bristol, Tenn., for $14.25 of Cambridge, Mass., a unit of the Takeda Pharmaceuticalper share, or about $3.6 billion. King is now a wholly owned Co. Ltd., initiated a Phase I trial of brentuximab vedotin insubsidiary of Pfizer. (See BioWorld Today, Oct. 13, 2010.) combination with chemotherapy for the treatment of newly • Pluristem Therapeutics Inc., of Haifa, Israel, and diagnosed systemic anaplastic large cell lymphoma patients.the Berlin-Brandenburg Center for Regenerative Therapies, The dose-escalation trial will evaluate brentuximab vedotin,in cooperation with the Charite-University of Medicine in an antibody-drug conjugate, when administered sequentiallyBerlin, reported positive results from a preclinical study or in combination with multi-agent, front-line chemotherapydemonstrating that local administration of Pluristem’s regimens. The study is expected to enroll up to 60 patients atPLX cells following muscle injury resulted in significant multiple centers in Europe and the U.S.improvement in the recovery of muscle function when • Syndax Pharmaceuticals Inc., of Waltham, Mass.,compared with the control group. Study data indicated will sponsor a Phase II study of its breast cancer candidate,PLX cells can be used in accidental injuries, such as athletic entinostat, in postmenopausal women with operable tripleinjuries, as well as intentional injuries, such as the incision negative breast cancer, with the goal of reprogrammingof skeletal muscle during surgery. Based on the findings, tumors to express estrogen receptor, thereby becomingPluristem plans to move ahead with clinical trials for muscle sensitive to hormonal therapy. In a preclinical study ininjury indications, which will be conducted in addition to animals, it was established that entinostat induces re-the company’s anticipated Phase II/III trial using PLX-PAD for expression of extrogen receptor and aromatase in triplethe treatment of critical limb ischemia. Earlier this year, the negative breast cancer cell lines. Those cells are thencompany raised $36 million in a public offering to finance sensitive to aromatase inhibitor. That study was publishedthat trial. (See BioWorld Today, Jan. 28, 2011 .) in the March 1 , 2011 , issue of Cancer Research. • Precision Therapeutics Inc., of Pittsburgh, and • Tobira Therapeutics Inc., of Princeton, N.J., saidMed BioGene Inc., of Vancouver, British Columbia, signed that single-dose cenicriviroc had no dose-limiting toxicity,a commercialization, license and research reimbursement predictable pharmacokinetics and good oral bioavailabilityagreement providing Precision with exclusive global rights in a Phase I trial for HIV. The drug was given to healthyto develop and commercialize LungExpress Dx, a gene volunteers in doses from 10 mg to 800 mg. Data wereexpression-based test designed to improve staging and presented at the 2011 Conference on Retroviruses andbetter identify mortality risks for patients with early stage Opportunistic Infections being held in Boston this week.non-small-cell lung cancer following surgical removal of The company plans to initiate Phase IIb studies in thetheir tumors. second quarter. B IO W ORLD P ERSPECTIVES A free, weekly e-zine offering unique viewpoints on developments within the biotechnology industry. Sign-up today and get a fresh outlook on topics that you can’t find elsewhere! Go to and click on “Perspectives”! To subscribe, please call BIOWORLD® Customer Service at (800) 688-2421; outside the U.S. and Canada, call (404) 262-5476. Copyright © 2011 AHC Media. Reproduction is strictly prohibited. Visit our web site at