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BIoTeCH'S MoST ReSPeCTeD NeWS SoURCe FoR MoRe THaN 20 yeaRS VolUMe 22, No. 20 two chinas emerging as biopharmas Money Raised By Biotech: 2014 vs. 2013shift toward innovative drugs Jan. 1 – May15, 2014: $15,199.04M Jan. 1 – May16, 2013: $7,124.49M By Marie Powers, Staff Writeralthough innovation is the topic of much conversation in China's biopharma industry, Western companies are likely to observe that the notion of innovative drug development in China is more hype than reality. however, participants at the Chinabio partnering forum, held in Suzhou earlier this month, offered compelling evidence that Chinese biotechs are, indeed, transforming cutting-edge discoveries into drug pipelines, with partnering opportunities very much welcomed.
no one disputes that the opportunities for drugmakers in China are enormous. Characterizing the country as a BrIC nation, along with Brazil, russia and India, is a misnomer at this point, according to ray Stevens, co-founder of ruiyi Inc. and professor in the departments of molecular biology and chemistry at the Scripps research Institute. Stevens contended that China "is ahead of every other BrIC national by 10 years." that assessment isn't far off. a recent thomson reuters white paper, "overcoming Clinical Challenges in BrIC markets," citing feb. 18 data from Cortellis Clinical trials Intelligence, indicated that more than 4,200 clinical trials were started in * Includes financings of public biotech firms with the exceptions of public offerings and certain investments from corporate partners.
China over the past five years, compared to approximately 1,200 in Brazil, 2,600 in russia and 2,300 in India over the same period. In fact, China is the only BrIC nation seeing an increase in the number of clinical trials initiated each year, according to the white paper. forecasts suggest that China will become the second largest pharmaceutical market in the world CompanIeS jumpIng on potentIal in 2017, overtaking japan.
SgC modulatorS payoff China's potential is attracting droves of returnees – Western- BIoteCh SeCtor ContInueS to Stall educated Chinese nationals – including tens of thousands of aS general marketS alSo tumBle scientists with advanced degrees. many also have work experience at big pharmas or biotechs and are eager to apply money raISed By BIoteCh In 2014 their knowhow to the Chinese market.
Word on the Street Chinese bioteChs in hunt for partners
Consider Shenogen pharma group. after eight years of effort,
Week In WaShIngton the Beijing-based company advanced its lead compound, Sng- phaSe I, II & III ClInICal trIalS update 162 (lcaritin), into a phase II study in China. Shenogen was co-founded by two lab mates: kun meng, chairman and Ceo, amerICan aSSoCIatIon for CanCer who completed his postdoctoral training at harvard university reSearCh; pharma data and the Washington university School of medicine, and Charlie Wang, chief science officer, who completed postdoctoral non-u.S. ClInICal trIalS update; To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email bioworld.sales- [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected]. Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght companies jumping on potential regulating cgmp, Ironwood's chief scientific officer, mark Currie, told BioWorld Insight. "In our mind, there are very sGc modulators payoff few regulators that have this kind of utility," he said.
By Michael Fitzhugh, Staff Writer Currie and his team now are advancing preclinical molecules that target sgC in a bid to address indications merck & Co. Inc.'s new billion-dollar collaboration with beyond the gastrointestinal space. they expect to initiate Bayer ag threw a spotlight on drugs capable of amping up their first sgC clinical study in the first half of 2015, concentrations of the messenger molecule cyclic guanosine prioritizing evaluation of application in pah and other monophosphate (cgmp) to impact cardiovascular and gastrointestinal diseases. now Bayer, Ironwood pharmaceuticals Inc. and others are leveraging expertise By looking at ways to optimize the half-life and volume of built on the way to gaining fda approval for drugs in the distribution of molecules targeting sgC, Currie's team is on space to expand their use.
the path to finding ways to deliver a controlled day-long lowering of blood pressure and, in other cases, diminish the only two fda-approved drugs capable of modulating fibroblast proliferation in the treatment of pulmonary, liver cgmp, Bayer's adempas (riociguat) and Ironwood's linzess and kidney fibroses. they're also looking at ways to cross (linaclotide), do so in different ways. Both impact targets in the blood-brain barrier to impact alzheimer's disease and the guanylate cyclase family and both are showing potential improve memory, he said.
for interesting new applications.
adempas targets soluble guanylate cyclase (sgC), the only sharing risks and rewards
known nitric oxide receptor in the human body. nitric oxide despite the promise of sgC modulators, major development activates soluble guanylate cyclase, which in turn kicks off efforts and clinical programs will be required to realize their production of cgmp, relaxing arteries and thereby increasing potential. that's where the Bayer-merck deal comes in. Both blood flow and decreasing blood pressure. the fda companies have been working on sgCs for years. approved adempas to treat two forms of pulmonary underscoring Bayer's desire to defray the major costs it will hypertension in october 2013, making it the first drug in its face developing adempas and other sgCs for new class to be approved to treat pulmonary arterial indications, this month it struck a deal giving merck full hypertension (pah) and the first drug of any class to be commercialization rights for adempas outside the americas, shown to be effective for patients with chronic half the profits for its phase II sgC, vericiguat, and thromboembolic pulmonary hypertension.
optionally half of any profit from several other sgCs the Ironwood's linzess (linaclotide), on the other hand, companies plan to co-develop. In exchange, merck will selectively activates guanylate cyclase C (gC-C), a receptor shoulder half the development costs for those new drugs.
found almost exclusively in the membrane of cells lining the for the potential rewards adempas and the other sgCs inner surface of intestinal tissue. In august 2012, it became offer, merck agreed to pay Bayer $1 billion up front and up the first and only gC-C activator approved by the fda to to an additional $1.1 billion if adempas and the other sgCs treat chronic idiopathic constipation (CIC) and irritable hit certain sales milestones. In addition to contributing its bowel syndrome with constipation (IBS-C), easing cash and one phase I compound, the Whitehouse Station, abdominal pain in the latter, and netting 2013 sales of n.j.-based company also brings serious credibility in the about $119 million. now Ironwood is pursuing an expanded cardiovascular market to the table. (See BioWorld Today, indication for linzess in opioid-induced constipation (oIC).
the company is also developing another gC-C activator, Importantly for both, Bayer's vericiguat, an investigational IW-9179, to treat functional dyspepsia and gastroparesis. But there's much more opportunity to be realized in bioworld insight pRacticaL infoRMation
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Send all press releases and related information to donald r. johnston, (770) 810-3118 // peter Winter, (770) 810-3142 // donald r. johnston (Senior director, editorial), amanda lanier (managing lynn yoffee, (770) 810-3123 editor), Sarah Cross (marketing director), matt hartzog, paul marino & greg rouse (account representatives) atLanta newsRooM lynn yoffee (executive editor), jennifer Boggs (managing editor), peter Winter (editor), karen pihl-Carey (database editor) monday, may 19, 2014 BIoWorld™ InSIght Biotech sector continues to stall little over 0.5 percent in may. however, companies in the group have seen their share values go on a wild ride during as general markets also tumble this period of uncertainty for the sector. By Peter Winter, Editor leading rnai therapeutics company alnylam pharmaceuticals Inc., for example, saw its share value drop With the general markets sliding last week over concerns 15 percent in april only to regain most of it back so far this about an uncertain economy, biotech companies followed month on the strength of reporting positive top-line results suit and by close of market thursday, the BioWorld Blue from its ongoing phase I trial of aln-at3, a subcutaneously Chip Biotech Index had dropped almost 1 percent since the administered rnai therapeutic targeting antithrombin in start of the month. this was, however, slightly better than development for the treatment of hemophilia and rare the 1.5 percent decline in the nasdaq Composite Index over bleeding disorders. the same period, while the dow jones Industrial average has so far held its own and remained unchanged.
among the leading decliners in the group was aegerion pharmaceuticals Inc., of Cambridge, mass., whose shares leading the decliners so far in may has been Seattle have tumbled 25 percent after posting a wider loss than genetics Inc., whose shares have fallen 8.7 percent despite analysts were expecting in its first quarter financials. the reporting solid revenue growth in its first quarter results company did post $27 million in net product sales for its with net product sales of its adcetris (brentuximab vedotin) juxtapid (lomitapide) capsules, approved to treat patients antibody-drug conjugate for treating hodgkin's lymphoma with homozygous familial hypercholesterolemia (hofh), a and anaplastic large-cell lymphoma coming in at $38.7 genetic disorder that causes extremely high cholesterol million compared to $33.9 million for the same period in levels. the amount represented a growth of 24 percent over 2013. despite the fact that most analysts believe the net product sales from the u.S. business in the prior quarter.
company's prospects are very good going forward investors are still looking for more. It is a story that has been reflected news of acorda therapeutics Inc.'s complete response letter across the whole spectrum of the sector – despite a for the new drug application (nda) related to plumiaz generally positive first quarter for public biotech companies, (diazepam) for epileptic cluster seizures was enough to send the reports have failed to bring investors back to the fold. its shares down about 14 percent so far this month. the (See BioWorld Blue Chip Index, below.) company also said the nasal spray version of the benzodiazepine therapy is unlikely to win approval this year. the BioWorld growth Index, which includes companies with (See BioWorld Today, may 5, 2014.) market caps in the range of $1 billion to $3 billion and a résumé that typically includes a strong drug pipeline and the dive in share prices permeated across the board for all partnered products in late-stage clinical trials, has seen its biotech companies. the BioWorld emerging Biotech Index, value drop almost 8 percent since the end of the first which tracks selected small-cap companies that have quarter, but similar to the Blue Chip Index has only fallen a To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght Money Raised By Biotech: Jan 1 - May 15, 2014 we've observed in the clinic, not just in animal studies, that every time we saw activity, we saw it first at metastasized sites. We're thinking that, if you combine our drug with some chemotherapy, it can wipe out metastasized sites as well as the primary tumor site, which would be a nice combination. But we can only do one or two trials at a time with our resources. a partner can do multiple trials." ‘the quality of the sCienCe is outstanding'
Suzhou alphamab Co. ltd. is another model of innovative drug discovery. founded in 2008, the company has 90 employees, including more than 65 scientists in its r&d operation. alphamab is advancing biosimilars as its bread and butter but also building a pipeline of novel oncology drugs. for example, Public Offerings kn-014 is a genetically engineered therapeutic protein that inhibits various isotopes of vegf, pIgf and pdgf, with the goal Private biOtechs of treating lung, bladder, colorectal and other metastatic cancers, as well as wet age-related macular degeneration. kn-010 is a bispecific antibody that binds different epitopes on cancer cells to prohibit cancer cell growth and proliferation through a variety of mechanisms. designed for improved efficacy against tumors while preventing resistance, the drug is being training at princeton university and served as an associate developed to treat breast cancer and other solid tumors.
professor at harvard medical School. Wang also is a professor of pathology at Creighton university.
founded last year, mabspace Biosciences Co. also is focusing on antibodies for therapeutic and diagnostic applications. using jun Bao, the company's senior vice president, chief business immune tolerance breaking technology, the company has officer and acting chief financial officer, holds a phd from the identified three therapeutic antibodies, mSB-001, mSB-002 and university of kansas. Bao's resume includes tenure at mSB-003, and one diagnostic application, mSB-004, that it is glaxosmithkline plc in China and at u.S. firms onyx seeking to partner. mSB-001 is designed as a best-in-class pharmaceuticals Inc., Icos Corp. – acquired in 2006 by Cialis antibody that targets the tumor microenvironment for treating (tadalafil) joint venture partner eli lilly and Co. – and Cell gastric and lung cancers. mSB-002 is a first-in-class antibody therapeutics Inc. (See BioWorld Today, oct. 18, 2006.) with the potential to treat tissue fibrosis and cancer, and mSB- Shenogen is targeting first-in-class drug development. Its Sng- 003 is an antibody-targeted immunotherapy for lung cancer, 162 is a naturally derived traditional Chinese medicine known as renal cancer and melanoma.
yingyang huo, a perennial grass whose name translates as and ruiyi has a bi-coastal strategy in its effort to develop novel "horny goat weed." the small molecule, which targets the drugs for the Chinese market. the company is headquartered in estrogen receptor er-alpha 36, is in a phase II study in China, la jolla, Calif., but has its discovery efforts and research facility with applications in liver and breast cancer as well as leukemia. in the Zhangjiang hi-tech park in Shanghai. earlier this year, Shenogen was started with just $100,000 in seed money, ruiyi netted a $15 million series B round from its existing according to Bao, and now has 80 employees. the company's investors, which include 5am ventures, versant ventures, business acumen and scientific prowess last year helped apposite Capital, Sr one, merck Serono ventures and aravis Sa. Shenogen attract a $20 million series C from a syndicate that (See BioWorld Asia, april 2, 2014.) included Qiming venture partners llC, of Shanghai, and legend ruiyi is using its intermembranous Conformation antigen Capital, of Beijing, an affiliate of tech company lenovo, along presenting System, or iCapS, technology to leverage therapeutic with China Investment Wealth venture fund and Shenzhen specificity across a set of targets from the large g protein- venture, a venture arm of the municipal government. lead coupled receptor (gpCr) family. although nearly one-third of investors from Shenogen's series B, including Idg venture and approved drugs modulate gpCrs, traditional drug discovery lapham group Inc., also joined. (See BioWorld Asia, nov. 6, methods have been stymied in exploring many of the selective targeting gpCrs – especially those with small extracellular Bao attended Chinabio seeking partners.
domains. ruiyi's iCaps changed that equation, enabling the "We'd like to find one or two partners for the market outside purified, isolated, conformationally correct presentation of China," he told BioWorld Insight, "and in China we are also open functional gpCrs, optimized to identify selective antibody for collaborations." the fact that Sng-162 has shown activity in inhibitors or activators with greater specificity, resulting in the solid tumors as well as leukemia is intriguing, Bao said, "and To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght riemser pharma gmbh acquired keocyt, which markets
pharmaceutical products to treat rare diseases.
Zalicus inc. said horizon discovery group will acquire its
aeterna Zentaris inc. entered an at-market issuance sales
combination high-throughput screening platform and related agreement with mlv & Co. llC.
assets for $8 million.
agile therapeutics inc. filed to raise up to $65 million in an Ipo. . . and More
Celator pharmaceuticals inc. entered a $15 million loan
abeona therapeutics has been granted fda orphan drug
agreement with hercules technology growth Capital Inc.
designations for its lead investigational therapies for treatment Chase pharmaceuticals Corp. raised $21 million in a series B
of Sanfilippo syndromes a and B.
financing round.
alk-abello a/s said partner Merck & Co. inc. launched
imaginab inc. completed a $21 million series B financing round.
ragweed allergy immunotherapy tablet ragwitek in Canada.
invivo therapeutics holdings Corp. closed a public offering for
beryllium has finalized its consolidation of assets from
gross proceeds of approximately $16.1 million.
emerald Bio and decode genetics and emerged as a drug gemmus pharma inc. said it closed a $3.3 million series B
discovery company.
financing round.
daiichi sankyo Co. ltd. discontinued a phase III trial for
Marinus pharmaceuticals inc. filed to raise up to $63 million in
nimotuzumab after four treatment-related deaths were observed, all in the nimotuzumab arm.
radius health inc. postponed its Ipo citing poor market
health Canada has suspended the establishment license of biolyse pharma Corp.'s facility in St. Catharines, ontario,
because of concerns with the manufacturing process.
sorrento therapeutics inc. expects to generate $25 million in
gross proceeds from a public offering.
Merck & Co. inc. said the fda approved Zontivity (vorapaxar)
to reduce thrombotic cardiovascular events in patients with a
sorbent therapeutics inc. said it closed a $6.5 million tranche
history of heart attack or with peripheral arterial disease.
of a $15 million series d financing.
provectus biopharmaceuticals inc. said its shares have been
Zs pharma inc. is seeking to raise up to $86 million from an
approved for listing on the nySe mkt.
woRd on the stReet allergan inc. has rejected the unsolicited april 22 acquisition
proposal from Valeant pharmaceuticals international inc.
"I think [cancer immunotherapy] is very exciting and ultimately will be beneficial to patients, but I do think it's blocked out a aslan pharmaceuticals pte ltd. signed an agreement with Csl little bit of the sun at aSCo again."
ltd. to develop an anti-Il-13 receptor monoclonal antibody,
CSl334, for severe to moderate asthma.
ron squarer, Ceo, array Biopharma Inc.
bristol-Myers squibb Co. is testing pd-1 checkpoint inhibitor
"this study characterizes the extent to which our genetic nivolumab in non-small-cell lung cancer with Celldex
makeup influences biomarkers in our blood, and it helps to therapeutics inc.'s Cd27-targeting varlilumab.
define their general characteristics. With its help, we can begin to understand very comprehensively to what extent our Jubilant biosys ltd. and orion Corp. are collaborating to
physiological status is controlled by genetics and by discover small-molecule inhibitors in the neuroscience nongenetic factors." therapeutic area.
nicole soranzo, group leader at the Wellcome
Mabvax therapeutics inc., a privately held cancer immuno-
trust Sanger Institute in hinxton, Cambridgeshire, therapy company, is merging with publicly traded telik inc.
on a massive study exploring the ways in which Medimmune is testing its anti-pdl-1 immune checkpoint
common genetic variants influence the levels of inhibitor, medI4736, in combination with incyte inc.'s oral
various metabolites in human blood indoleamine dioxygenase-1 inhibitor. "the decision to divest our ophthalmic business is part of our Merck & Co. inc. signed a deal to sell nine of its ophthalmic
ongoing strategy to sharpen our commercial focus and improve products to japan-based santen pharmaceutical Co. ltd.
our operational effectiveness." prometic life sciences inc. increased its ownership in nantpro
Jay galeota, president of hospital and specialty
care at merck & Co. Inc., on its decision to sell shire plc is acquiring lumena pharmaceuticals inc. for $260
nine of its ophthalmic products to japan-based million up front plus undisclosed milestones.
Santen pharmaceutical Co. ltd.
To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght week in washinGton residual uncertainty and assumptions about analytical quality and similarity – as especially relevant to the development of the ema and the fda released a draft joint proposal to facilitate multi-arm, multicompany clinical trials for pediatric the federal trade Commission (ftC) is once again proposing a treatments of gaucher disease. the proposed approach would study of patent assertion entities, also known as patent trolls, determine the safety and efficacy of several new medicines at and their impact on innovation and competition. the ftC first the same time, reducing the total number of children enrolled proposed the study last year. after reviewing 70 comments on since all the therapies would be evaluated against one control the study plan, the commission revised the study to sharpen its group. If successful, this concept could be used to develop focus and reduce the burden on participants.
multiple drugs for other rare diseases in a reduced time frame the ema issued an update on joint u.S.-european efforts to and with limited enrollment.
tackle the rising tide of antimicrobial resistance, detailing the fda released the latest installment in its series of draft progress in converging the ema and fda regulatory biosimilar guidances. It discusses the design and use of clinical requirements in the development of new antibacterial drugs. pharmacology studies. pharmacokinetic and the move to merge regulation in that area sits under the pharmacodynamic data are required as part of the fda's umbrella of the transatlantic taskforce on antimicrobial stepwise approach to demonstrating biosimilarity to the resistance, established between the european Commission reference drug. the draft guidance identifies three key and the u.S. department of health and human Services, to act concepts – exposure and response assessment, evaluation of together in fighting drug-resistant pathogens. //
cakewalk. locals cite challenges with importing cells, gaining access to radioactivity and interfacing with the China food and drug administration (Cfda), along with mundane tasks such potential development of more effective antibodies and other as making bank deposits, which can require a half-day of standing in line. the perception of drug development also is the company's lead candidate, ryI-008, is an anti-Il-6 different in China, where biosimilars remain the standard and monoclonal antibody with potential uses in autoimmune domestic investors view phase II as early stage.
diseases and cancer, with rheumatoid arthritis as a proposed for that reason, ruiyi's lead compound uses well-established initial indication. (See BioWorld Today, may 17, 2013.) biology that is sufficiently innovative to help the Cfda gain ruiyi's Stevens was lured to China by the energy and experience working with the company's technology. "once we entrepreneurship in the nation's science community. after have that one going, we can follow suit with our gpCr teaching students from China for more than a decade and antibodies, which are innovative," Stevens said. hearing the hype about their home country, he wanted to pursuing innovative drug discovery and development in China answer three questions: Is the science real? Is it high quality? requires commitment, he added.
and how would it lead to innovative drug development? "everything is changing rapidly, so if you want to do drug "I've been coming to China for 20 years, and I used to see a lot development here, you can't do it part-time," he maintained. of empty buildings," Stevens told BioWorld Insight. after three "you commit or you don't. If you care about quality, you have to years on the ground in Shanghai, he's convinced the science "is be fully invested." very real." his students at the ihuman Institute at Shanghaitech In some ways, however, China reminds Stevens of California, university – a research university modeled after the which was the "wild, wild West" in the early days of biotech. massachusetts Institute of technology and developed from the ground up by the Shanghai municipal government and Chinese "no one has really hit it big yet in China" in terms of innovative academy of Sciences – already are publishing papers in Nature drug development, he said, "but our investors know this is and Science. where they need to be. We know a breakthrough drug is going to happen." "the quality of the science is outstanding," Stevens said, adding that the opportunity to conduct drug development is better in until it does, patience is the key. China than almost anywhere else in the world, including the "don't come to China because you think that's where the u.S. "putting ruiyi in China was exactly the right thing to do," he money is," Stevens advised. "Instead, come to China if you have a really good business model. there's double-digit growth potential and a large population of treatment-naïve patients ‘you haVe to be fully inVested'
with unmet medical needs. But you need long-term thinking, that's not to say innovative drug development in China is a not just one or two years." //
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monday, may 19, 2014 BIoWorld™ InSIght and marketing partner in japan, also is working on sgCs and has built an extensive patent position in the space, but with very different chemistry than Ironwood's, Currie said.
sgC it's developing in two phase IIb studies in an indication "I think it's a natural progression as you see advances in it's calling "worsening chronic heart failure," is included in these area that once you're able to bring clear benefit to the deal. merck's lengthy experience selling cardiovascular patients," he said, "there will continue to be innovations." //
treatments in the u.S. market could be a boon for Bayer, noted Bayer Ceo marijn dekkers, during a conference call discussing the deal. "the significant expertise that merck has built as a top five global player in the cardiovascular area, and its strong presence in the large u.S. market, make merck a partner of choice for our current and future sgC program," he said.
market caps averaging about $500 million, has seen its Without doubt, having a partner to foot the cost of what value drop about 1 percent in may.
dekkers called "a significant and comprehensive" phase III despite a rocky start to the month, the overall declines for trial of vericiguat next year will help, too.
biotech companies have not been as dramatic as predicted. for vericiguat and other potential investigational sgC there will still be some turbulence going forward but with modulators, Bayer will lead the commercialization outside investors squarely focused on the upcoming 50th meeting of the americas while merck will lead commercialization in the the american Society of Clinical oncology later this month americas. Both companies will have the option to this could be enough to smooth the ride for biotech stocks co-promote adempas and the follow-on sgC modulators in given the number of promising results from important cancer each others' territories. therapies that drug developers are scheduled to report. //
meanwhile, leverkusen, germany-based Bayer already is looking for ways to expand riociguat's use. It's preparing to run phase II trials studying the drug's impact in pulmonary hypertension (ph) associated with interstitial idiopathic NEGOTIATE LUCRATIVE BIOPHARMA LICENSING DEALS pneumonia, diffuse systemic sclerosis, cystic fibrosis and the BIOPHARMACEUTICAL ROYALTY rare raynaud's disease.
the partners also are testing two more compounds in phase I, an unnamed Bayer sgC stimulator in development as a ESSENTIAL BENCHMARKS FOR DEALMAKING treatment for resistant hypertension and merck's mk 8892 New data from BioWorld reveal that biotechnology companies are now
for ph and heart failure. a second Bayer compound is part garnering higher royalty rates from pharmaceutical partners, higher
of the deal, but is still preclinical.
than other biotech partners.
BioWorld Data's new resource, Biopharmaceutical Royalty
More in play
Rates Analysis: Essential Benchmarks for Dealmaking, analyzed hundreds of licensing deals. According to the Innovation alone won't make drugs targeting guanylate Royalty Rates Analysis, the average royalty rate for deals cyclases successful, of course. In pah, for example, with a pharma licensee is 14.9% at the high end and 11.7% at adempas already has competition from actelion the low end.
pharmaceuticals ltd.'s opsumit (macitentan), an oral This critical negotiation and analysis tool contains: version of actelion's tracleer (bosentan) that was approved • More than 320 licensing deals with all deal terms and key background information on the drug or drug technology in october 2013 for pah. united therapeutics Corp.'s • Median royalty rates and up-front licensing fees, details that can be used remodulin (treprostinil) is creating competition for as benchmarks and comparators adempas, too. But clearly there's plenty of commercial and • Covers hot disease topic areas and more: Alzheimer's, cancer, clinical interest carrying cgmp modulators ahead. cardiovascular, diabetes, hepatitis and pain management new york-based Synergy pharmaceuticals Inc. is developing AVAILABLE IN PRINT AND PDF plecanatide, a drug targeting gC-C to treat the same indications as linzess. the company intends to initiate Visit us at www.bioworld.com or call 1-800-477-6307 or pivotal phase III trials with IBS-C patients in the second half 1-770-810-3144 to purchase your copy today! of 2014. It's additionally developing a gC-C agonist it calls Sp-333 for the treatment of oIC and ulcerative colitis. (See 2013 Thomson Reuters BioWorld Today, nov. 13, 2013.)tokyo-based astellas pharma Inc., Ironwood's development To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght fda approvals in april
Company
alk-abello a/S (Copenhagen) and merck & Co. Inc. ragweed allergic rhinitis (Whitehouse Station, n.j.)Biogen Idec Inc. (Cambridge, mass.) dyax Corp. (Cambridge, mass.) acute hereditary angioedema genmab a/S (Copenhagen) and glaxosmithkline plc arzerra Chronic lymphocytic leukemia (london)greer laboratories Inc. (lenoir, n.C.) allergic rhinitis kaleo Inc. (richmond, va.) phase i clinical trials update april 2014 results from a pilot study of 20 polymer treatment patients showed statistically inc. (new york)
significant improvement in ulcerations and pain phase I data confirmed safety in therapeutics ag
a compound aimed relapsed dosing started in its phase I/II trial 4/17/14 at restoring normal platinum- of apr-246; it will enroll about function to tumor 180 patients in a two-part design suppressor protein arno therapeutics
enrolled the first patient in a inc. (flemington,
progestin hormone castration- phase I/II trial resistant prostate cancer vaccine consisting final phase I data demonstrated therapeutics inc.
of a fully human robust antibody and t-cell responses and evidence of clinical benefit in patients with very specificity for the advanced cancers and suggest that CdX-1401 may predispose receptor deC-205 patients to better outcomes on subsequent therapy with checkpoint inhibitors To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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Cleveland biolabs
phase I trial characterized the inc. (Buffalo, n.y.)
orally administered cancers drug's safety, profiled its pharmacokinetics, and assessed it for preliminary evidence of antitumor activity in the liver deciphera
pan-raf inhibitor Started a phase I trial pharmaceuticals
llC (lawrence,
kan.)
development
received approval from the fda Center for
and the tfda to begin phase I a synthetic double- Solid tumors, Started a phase I trial stranded rna in a inc. (Watertown,
stable lipid particle myeloma or helix biopharma
a targeted antibody Stage Iv received fda approval to start a Corp. (aurora,
naturally occurring metastatic plant enzyme non-small-cell lung cancer immatics
a phase I trial met its primary endpoints of safety and gmbh (tuebingen,
germany)
innate pharma sa
Began the cohort expansion (marseille, france) checkpoint inhibitor portion of an open label phase I trial karyopharm
Started a phase I trial of selinexor 4/22/14 therapeutics inc.
in children with high-risk malignancies resistant to standard chemotherapy peregrine
opened enrollment of a trial of bavituximab in combination with inc. (tustin, Calif.)
yervoy (ipilimumab, Bristol-myers Squibb Co.) precision biologics neo-102
a reformulation of phase I/IIa data showed preliminary signs of activity based on stabilization of disease colorectal cancer sorrento
phase I/II data from six patients therapeutics inc.
showed clinically meaningful improvement in quality of life, achieving, on average, a 20% reduction in their pain intensity To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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tocagen inc. (San
Interim data of patients treated amiretrorepvec and grade glioma with the combination showed it to flucytosine; gene be safe and well tolerated with therapy candidate minimal treatment-related toxicity Xbiotech inc.
advanced cancer results from a phase I/II study showed an excellent safety profile, antitumor therapy while overall, patients' constitutional symptoms improved phase I/II data showed it met its therapeutics inc.
primary endpoint of safety Capstone
dosing was started in the phase Ia 4/9/14 therapeutics inc.
lipoprotein/non- trial high-density lipoprotein cholesterol reduction Cytori therapeutics Cytori Cell
Chronic ischemic data from the preCISe trial phase 4/15/14 inc. (San diego)
I/IIa feasibility trial demonstrated statistically significant differences regenerative cells in cardiac functional capacity extracted from a between treated and placebo patient's own fat tissue using Cytori's Celution system CENTRAL NERVOUS SYSTEM
Cynapsus
Interim data from its pilot study therapeutics inc.
sublingual strip indicated that a higher load of drug on the strip does result in a higher amount of drug entering the bloodstream glenmark
Is entering human trials pharmaceuticals sa
ltd.)
prothena Corp. plc
Started a phase I trial antibody targeting Vernalis plc
an a2a antagonist results from a phase Ib/II proof- of-concept study showed it attention deficit achieved a statistically significant hyperactivity improvement in the number of correct scores in permp-p measure (p = 0.019) compared to placebo To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght final phase Ib data demonstrated 4/23/14 that while octreotide significantly ltd. (tel aviv, Israel)
inhibited the secretion of insulin and glucagon in humans and showed a significant and sustained increase of plasma glucose levels, the company's dg3173 had much less of an effect on insulin and glucagon release halozyme
recombinant and a type 1 diabetes the primary endpoint of non- therapeutics inc.
inferiority of a1C levels at six months was met for the ConSIStent 1 trial sernova Corp.
Contains insulin- Interim results from a phase I/II (london, ontario) study demonstrated that in the first group of patients the implanted Cell pouch is showing longer-term safety and biocompatibility targets galectin-3 the first patient in cohort 2 of its therapeutics inc.
phase I trial was successfully dosed with 4 mg/kg, which is double the dose given in cohort 1; all eight patients have received their first infusion in cohort 2 hepatera ltd.
Inhibits the entry Chronic hepatitis Initiated a trial investigating it for mechanism of hBv B virus and the effects of myrcludex in combination with pegylated interferon and the use of the entry inhibitor as pre-treatment before interferon therapy is initiated INFLAMMATORY
ablynx nV (ghent,
Started dosing in healthy volunteers in a phase I trial evaluating a subcutaneous formulation a small molecule Started a phase I trial inc. (Cambridge,
mass.)
allena
phase I data demonstrated a statistically significant difference inc. (newton, mass.)
in the reduction of urinary oxalate oxalate degrading levels in 30 healthy subjects when the treatment was compared with placebo (p = 0.0002) To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght Started a phase I trial in healthy postmenopausal women ltd. (nottingham,
teriparatide product galectin inhibitor the first phase I cohort showed a therapeutics inc.
therapeutic effect on fibrosis, inflammation and cellular injury kamada ltd. (ness
Company is starting a small phase 4/4/14 alpha-1 antitrypsin I/II proof-of-concept study lpath inc. (San
Started a study of brain injury lysophosphatidic induced by blast overpressure nitto denko Corp.
dosing of healthy volunteers in a sirna that inhibits liver and other phase I study has completed the cause of fibrosis, organsas well as a targeted lipid nanoparticle delivery system oxford biomedica
a lentiviral vector- neovascular wet Completed the planned plc (oxford, uk)
recruitment of 21 patients into its prothena Corp. plc
Interim data from an ongoing phase I study showed that neod001 administered intravenously once per month was generally safe and well tolerated at the doses studied questcor
h.p. acthar gel repository Clinical results showed it could be 4/22/14 a potentially useful therapy for inc. (anaheim hills,
inducing remission of proteinuria phase I/II data showed that the therapeutics inc.
severe glabellar trial met its primary efficacy and stemcells inc.
human neural stem Spinal cord Completed enrollment of the a modified form of positive results from its phase I inc. (lexington,
recombinant human trial in russia showed that two doses of 2,500 Iu and 5,000 Iu were found to be safe and well tolerated notespublic 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, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght phase ii clinical trials update april 2014 therapeutic vaccine allergic asthma failed in a phase IIb trial ag (Schlieren,
Switzerland)
galapagos nV
a selective jak1 gSk2586184 met the primary (mechelen, Belgium) endpoint in phase IIa psoriasis and glaxosmithkline study, with patients given the drug achieving a 75% or greater improvement from baseline in their psoriasis area Severity Index scores at week 12 gene signal
a phase IIa study demonstrated international sa
that topical application of gS-101 reduced the size of psoriatic lesions and inflammation compared to placebo geneuro sa
multiple sclerosis the one-year open-label extension 4/18/14 of its phase IIa study in 10 patients targets the mSrv- was completed, with results showing that the treatment was well tolerated over the long term and that pharmacodynamic responses could be observed phosphagenics ltd. tretinoin
Completed enrollment in a phase transdermal delivery technology tpm synthetic biologics trimesta
oral estriol; oral, topline data from the phase II trial 4/30/14 inc. (rockville, md.)
demonstrated that trimesta given multiple sclerosis with first-line rrmS therapy Copaxone (glatiramer acetate injection) resulted in reduced annualized relapse rate and improved cognitive function at 12 months of treatment as compared to placebo plus Copaxone CANCER
ambit biosciences
flt3-Itd positive Started a phase II cohort of a Corp. (San diego)
phase I/II study of quizartinib in combination with either 5-azacitidine or low-dose cytarabine To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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dendreon Corp.
phase II Stand data suggested recurrent prostate tumor-specific t-cell responses cancer appear to be enhanced and sustained when provenge is given following androgen deprivation therapy halozyme
pancreatic cancer halted its phase II trial following therapeutics inc.
an unexpected imbalance in thromboembolic event rate between treatment and control groups; fda placed a clinical hold on patient enrollment and dosing oncosec Medical
Cutaneous t-cell Company is relaunching a phase II 4/8/14 inc. (San diego)
progenics
Comprises a fully Completed enrollment in the human monoclonal chemotherapy-naïve cohort in its inc. (tarrytown, n.y.)
antibody selectively phase II trial of pSma adC; that targeting prostate- cohort of 36 patients, all of whom specific membrane progressed on hormonal therapies, antigen linked to a was added to the phase II study chemotherapeutic following positive response to pSma adC in patients in the chemotherapy-experienced setting neratinib; a pan- metastatic breast phase II data showed that the biotechnology inc.
her tyrosine kinase cancer neratinib-containing regimen graduated, based on a high probability of success in phase III with a signature of her2-positive/ hr-negative; neratinib achieved an estimated pCr rate of 55.6% compared to the control arm, which had an estimated pCr rate spectrum
Captisol-enabled met the primary endpoints in a propylene glycol- pivotal phase II trial inc. (henderson,
a small molecule Completed enrollment in gog- that targets toll- 3003 (nCt01666444), a epithelial ovarian, randomized, placebo-controlled fallopian tube or phase II trial of vtX-2337 in primary combination with pegylated peritoneal cancer liposomal doxorubicin To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght to prevent atrial genetically screened the first inc. (Westminster,
patient in its phase IIb/III trial patients with heart failure or left ventricular dysfunction glycomimetics inc. gmI-1070
phase II data showed it improved (gaithersburg, md.) crisis in people efficacy outcomes independent of with sickle cell ISIS-apoCIIIrx an antisense drug high triglycerides phase II data showed patients treated achieved average inc. (Carlsbad, Calif.)
reductions of as much as 71% in apolipoprotein C-III a component of very low density lipoprotein, up to 64% in triglycerides, and average increases of up to 52% in high-density lipoprotein cholesterol CENTRAL NERVOUS SYSTEM
alder
a randomized, double-blind, inc. (Bothell, Wash.)
concept trial showed it met the primary endpoint of the study by targets calcitonin significantly reducing mean migraine days per month vs. placebo during weeks five to eight In a subgroup of patients with articular injection associated pain in moderate to severe oak, there inc. (greenwood
were statistically significant osteoarthritis of improvements in WomaC pain (p = 0.005) and function scores (p = 0.04) over 20 weeks brainstorm Cell
fda approved commencement of 4/29/14 therapeutics inc.
mesenchymal stem lateral sclerosis its phase II trial Civitas therapeutics Cvt-301
reported positive results from a inc. (Chelsea, mass.)
phase IIb trial of Cvt-301 showing the study met its primary endpoint Cytokinetics inc.
failed in a phase IIb trial lateral sclerosis To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght phase IIa data showed that at 10 lateral sclerosis weeks following completion of llC (pasadena,
dosing clinical measurements of alS progression remained the same in two of eight patients, while the rates of degradation of those clinical measurements had slowed in five of the remaining six treated patients early data from a phase II study therapies inc. (new
reveal that low doses of the drug show it acts as a potent 5-ht2a receptor antagonist, and that, as the dose is increased, it gradually engages other key brain receptors with regional selectivity the phase II Imaging trial, biotechnology ltd.
ImagIne, did not meet its primary endpoint of a statistically significant reduction in the levels of beta-amyloid plaques DIABETES
adocia sas (lyon,
phase IIa data showed that in comparison to humalog (eli lilly and Co.) commercial insulin, it met the primary endpoint and showed a significant increase in Biochaperone lispro bioavailability in the first half-hour compared to humalog missed the primary endpoint in a selective inhibitor of disease phase II trial of Ctp-499 in inc. (lexington,
patients with diabetic kidney disease, but the 48-week results showed some promise in a longer treatment duration islet sciences inc.
remogliflozin a selective Sglt2 two 12-week phase IIb studies (raleigh, n.C.) and and nonalcoholic showed that at week 12, twice- bhV pharma inc.
daily dosing of remogliflozin (research triangle etabonate produced a statistically significant trend in dose response for change from baseline with changes in hba1c ranging from -1.0 to -1.4%; once-daily dosing demonstrated a trend in dose response for change from baseline in hba1c above the lowest dose with changes in hba1c ranging from -0.5 to -0.8% To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght phase II data showed it met the primary endpoint of reducing inc. (the
mealtime insulin use as well as Woodlands, texas) several secondary endpoints noxxon pharma ag noX-e36
phase IIa proof-of-concept data showed statistically significant reductions in urinary albumin excretion and improved glycemic control an orally ingestible type 2 diabetes phase IIa data showed that the oral insulin appeared to be safe and well tolerated for the dosing regimen tested dosed the first subject in a phase II 4/28/14 study designed to treat inc. (austin, texas)
omnipod infusion pump GASTROINTESTINAL
athersys inc.
Stem cell product product failed to moderate the severity of disease in a phase II ulcerative colitis study of 128 patients; it did not show statistically significant improvement compared to placebo Chronic hepatitis the first cohort of eight patients research Corp.
was fully enrolled and dosed in a (pasadena, Calif.) gilead sciences inc. Sovaldi
hepatitis C virus phase II data of 400 mg of Sovaldi 4/11/14 (foster City, Calif.) with the nS5a inhibitor ledipasvir 90 mg, with and without ribavirin twice-daily, showed 100% of treatment-naïve genotype 3 patients who got 12 weeks of the combo plus ribavirin, and 64% of the same patients without ribavirin achieved Svr 12 weeks after treatment Medivir ab
protease inhibitor phase II data from cohort 2 in the hepatitis C virus interferon-free CoSmoS study showed that 93% of prior null responder and treatment-naïve patients achieved sustained virologic responses 12 weeks after the end of treatment with simeprevir and Sovaldi (sofosbuvir, gilead Sciences Inc.) To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght a hypoxia-inducible anemia Completed enrollment in its therapeutics inc.
ongoing 200-patient phase IIb (Cambridge, mass.) auris Medical ag
phase IIb data demonstrated it (Basel, Switzerland) aspartate (nmda) was well tolerated and safe and receptor antagonist established proof of concept Celgene Corp. and
formerly known as anemia in end- Interim phase IIa data showed acceleron pharma
aCe-011; targets dose-dependent increases in inc. (Cambridge,
hemoglobin in patients on an antisense drug phase II data showed that of seven 4/30/14 infants, five are alive without the inc. (Carlsbad, Calif.)
need for permanent ventilation; in another phase II study in children with Sma encouraging preliminary results were also observed in two additional functional tests: the six-minute walk test and the upper limb module test ohr pharmaceutical Squalamine
an anti-angiogenic Wet form age- Completed enrollment of its phase 4/30/14 inc. (new york)
small molecule that related macular II trial with 142 patients multiple growth factors abnormal dermal the first patient has enrolled in a Corp. (marlborough,
targets connective tissue growth factor scioderm inc.
a topical therapy Completed enrollment in a phase lesions in patients IIb study (Sd-003) to evaluate the safety and efficacy of Sd-101; the study enrolled a total of 48 subjects, ages 6 months and older RESPIRATORY
anergis sa
Birch pollen allergy Birch pollen the long-term efficacy trial is fully 4/24/14 enrolled, including 196 patients enrolled that participated in the 2013 field-based phase IIb trial of allert dbV technologies
a patient enrolled in the phase II sa (Bagneux,
araChIld study who received 18-month epicutaneous immunotherapy with viaskin remained desensitized to peanut allergy after one year off- treatment, with a strict peanut diet To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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novavax inc.
phase II data in 720 healthy (gaithersburg, md.) women of childbearing age vaccine candidate showed it was well tolerated with no serious adverse events; it also demonstrated significant increases in rSv-f antibody levels across all doses and formulations, as well as clear increases in rSv neutralizing antibodies theravance inc.
Started a dose-ranging phase IIb obstructive pulmonary disease notespublic 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, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght phase iii clinical trials update april 2014 atopic dermatitis enrolled the first patient in a pair of pivotal phase III trials inc. (palo alto,
biogen idec inc.
peginterferon beta- relapsing two-year phase III data from the (Cambridge, mass.) multiple sclerosis advanCe trial demonstrated favorable results on relapse rates, magnetic resonance imaging findings and disease progression when dosed every two weeks Catalyst
reached patient enrollment target 4/2/14 for its pivotal phase III partners inc. (Coral
gables, fla.)
CANCER
agenus inc.
Companies are terminating the (lexington, mass.) therapeutic that phase III magrIti study after it missed both the first and second glaxosmithkline plc
Stimulon adjuvant co-primary endpoints (london)
ambit biosciences
an oral, once-daily relapsed/ Initiated the phase III inc. (San diego)
selective inhibitor refractory acute Quantum-r study comparing myeloid leukemia quizartinib as a monotherapy to chemotherapy regimens in patients with the fmS-like tyrosine kinase-3 (flIt3)-Itd mutation amgen inc.
phase III secondary endpoint data 4/7/14 on overall survival showed a "strong trend" despite a statistical miss; the trend in favor of t-vec achieved a "p" value of 0.051 Celsion Corp.
final trial results from a (lawrenceville, n.j.) 701-patient trial suggest that it may significantly improve overall survival, compared to a control, in patients whose lesions undergo the radiofrequency ablation treatment for 45 minutes or more heron therapeutics Sustol
formerly apf530; to prevent Started a phase III label expansion 4/1/14 inc. (redwood City,
induced nausea and vomiting To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght a drug designed to metastatic failed to perform better than block production of castrate-resistant standard chemotherapy in the inc. (Bothell, Wash.)
pivotal Synergy trial; adding custirsen to docetaxel and prednisone did not significantly industries ltd.
improve overall survival for the oncolytics biotech
the phase III reo 018 study inc. (Calgary,
testing reolysin in combination with carboplatin and paclitaxel showed a statistically significant improvement (p = 0.0072); an analysis of overall survival also demonstrated a statistically significant improvement in the reolysin arm vs. control arm (p = 0.0146) oncothyreon inc.
Started a phase III trial (Seattle) and Merck
locally advanced kgaa (darmstadt,
small-cell lung cancer Sorafenib tablets locally recurrent results from the phase III deCISIon trial demonstrated that inc. (South San
nexavar tablets significantly extended the time patients lived bayer healthcare
thyroid carcinoma without their disease worsening inc. (Whippany, n.j.)
sorrento
metastatic breast the first patient was dosed in the therapeutics inc.
polymeric micelle pivotal trial, trIBeCa (trIal designed to evaluate bioequivalence between Cynviloq and abraxane) CARDIOVASCULAR
amgen inc.
protein convertase Cholesterol phase III data confirmed phase II subtilisin/kexin results showing evolocumab brought down low-density lipoprotein cholesterol by 50-60% armetheon inc.
oral anticoagulant prosthetic heart reached an agreement with the (Sunnyvale, Calif.) fda on an Spa for the final pivotal trial of tecarfarin biogen idec inc.
positive top-line results of the kids 4/11/14 (Cambridge, mass.) factor vIII fc fusion a-long phase III trial showed it and swedish
was generally well tolerated and orphan biovitrum
no neutralizing antibodies that might interfere with its activity were detected To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght once-daily factor launched a global, pivotal phase Xa anticocagulant III trial to determine whether inc. (South San
extended preventive treatment can prevent blood clots in the leg and lung that can occur in hospitalized cardiovascular disease patients regado biosciences reg1
Company is expanding enrollment 4/4/14 inc. (Basking ridge,
in the phase III trial comparing reg1 to angiomax (bivalirudin, the medicines Co.) after achieving intervention, or its 1,000-patient enrollment CENTRAL NERVOUS SYSTEM
alkermes plc
favorable phase III data demonstrated statistically significant reductions from baseline in positive and negative Syndrome Scale total scores at week 12, compared to placebo dextromethorphan pseudobulbar phase III data showed that 19% of 4/29/14 hBr and quinidine affect treated patients achieved inc. (aliso viejo,
remission from symptoms after one week; 50% of patients achieved remission by the end of the 12-week trial a breath-powered Severe migraine migraine relief for some patients began as early as 15 minutes inc. (aliso viejo,
following treatment, and a statistically significant greater number of treated patients experienced headache relief compared to placebo at all time points from 30 minutes through two hours Cytokinetics inc.
a novel skeletal results from BenefIt-alS lateral sclerosis showed that of the 711 patients enrolled in the trial, 605 were randomized to double-blind treatment with either tirasemtiv or placebo for 12 weeks; the trial did not achieve its primary efficacy endpoint,, but did result in a statistically significant and potentially clinically meaningful reduction in the decline of slow vital capacity To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght formulation of the attention-deficit Began enrollment in a phase III pharmaceuticals &
development inc.
(subsidiary of
highland
therapeutics Inc.;
toronto)
navidea
technetium tc 99m pain navidea is testing lymphoseek injection in a head-to-head study inc. (dublin, ohio)
with radiolabeled sulfur colloid to see which drug causes patients less pain enrolled the first patient in a sciences
international inc.
(raleigh, n.C.)
evoke pharma inc.
enrolled the first patient in the recurrent diabetic phase III trial INFECTION
abbvie inc.
ritonavir; fixed- hepatitis C virus phase III data showed patients dose combination achieved sustained virologic response rates of 91.8% and 95.9% in the 12-week and co-formulated with 24-week treatment arms, 267) 25 mg, dosed once daily, and dasabuvir (aBt-333) 250 mg with or without rBv, dosed twice daily durata began enrollment of a therapeutics inc.
phase IIIb trial to evaluate the efficacy and safety of dalvance for infections caused injection, in a single 1,500-mg by susceptible dose infused over 30 minutes hepatitis B virus Started a phase III trial technologies Corp.
combines hepatitis (Berkeley, Calif.) B surface antigen with a toll-like receptor 9 agonist To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght Completed the last licensure- biosolutions inc.
enabling study in its Biothrax post-exposure prophylaxis program; it met both its primary and secondary endpoints aBt-450, aBt- three direct-acting Chronic genotype pivotal phase III SapphIre-I and antiviral regimen, 1 hepatitis C virus II study data showed patients inc. (Watertown,
boosted protease achieved sustained virologic mass.) and abbvie
inhibitor aBt-450/ response rates 12 weeks post- treatment of 96.2% (n = 455/473) inhibitor aBt-267 and 96.3% (n = 286/297), nucleoside polymerase inhibitor aBt-333 gilead sciences inc. Sovaldi
positive top-line results from a (foster City, Calif.) chronic hepatitis phase III trial of Sovaldi with C virus infection ribavirin showed it met its primary efficacy endpoint of superiority compared to a predefined historical control sustained virologic response rate Medivir ab
Chronic genotype two phase III trials are recruiting 1 hepatitis C virus patients to study simeprevir in combination with sofosbuvir in treatment-naïve and treatment-experienced patients with and without cirrhosis Initiated separate multinational, placebo-controlled trials to inc. (Cheshire,
evaluate the safety and efficacy of eculizumab in patients generalized myasthenia gravis phase III data showed that therapeutics inc.
subjects who switched from placebo to migalastat after month six turned up a statistically significant reduction in kidney interstitial capillary gl-3 at month 12 (p = 0.013), and those who stayed on migalastat for 12 months showed a durable reduction To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght additional phase III data showed improved clinical outcomes; inc. (Chesterbrook,
histolyticum (CCh) adverse events were mostly pa.) and swedish
localized to the penis, non-serious orphan biovitrum
and resolved without intervention before the next injection biomarin
phase IIIb Spark data showed it pharmaceutical inc.
met its primary endpoint (San rafael, Calif.)
and Merck kgaa
(darmstadt,
germany)
intercept
phase III poISe trial data showed 4/15/14 it met its primary endpoint (p < inc. (new york)
lipocine inc. (Salt
oral testosterone Completed enrollment of its Soar 4/30/14 (Study of oral androgen replacement) pivotal phase III study phase III data supported the safety 4/8/14 liposome injectable arthroplasty and efficacy to achieve a femoral inc. (parsippany,
nerve block in patients; there was a 24% reduction in total opioid use reached its enrollment goal of therapeutics inc.
120 subjects in the first of two head-to-head studies comparing androxal to the leading u.S. testosterone replacement therapy revo biologics inc. atryn
Started a phase III program in pregnant women during the 24th to 28th week of pregnancy Xoma Corp.
Based on its meeting with the (Berkeley, Calif.) fda, plans are being finalized for a gevokizumab phase III program expected to include two double-blind, placebo-controlled clinical studies RESPIRATORY
theravance inc.
Started a phase III study testing the combination treatment, ff/vI 100/25 mcg once daily, compared glaxosmithkline plc
fluticasone furoate disease to vI 25 mcg once daily, administered via the ellipta notesSpa = Special protocol assessment.
public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.
the date indicated refers to the BioWorld Today issue in which the news item can be found.
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Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght american association for cancer Research april 2014 phase I data showed complete remissions in three of seven inc. (Cambridge,
targeting candidate evaluable patients mass.)
bergenbio a/s
a small-molecule phase Ia data showed the drug to 4/10/14 inhibitor of the axl be safe and well tolerated in doses receptor tyrosine up to 1.5 g daily with a predictable pharmacokinetics profile and long plasma half-life, allowing for different dosing options bind therapeutics
phase I data demonstrated a inc. (Cambridge,
greater dose intensity by about 50% at once weekly for three containing cytotoxic weeks dosing (Q1W), as compared to once every week dosing described in a previous study Calithera
phase I data showed that a 100- biosciences inc.
mg dose resulted in peak plasma levels of drug at or above 300 nm and generated a greater than 80% inhibition of glutaminase in platelets four hours after dosing deciphera
pan-raf inhibitor; Cancer Started a phase I trial a small-molecule llC (lawrence,
kan.) and eli lilly
and Co.
(Indianapolis)
delmar
data from its ongoing phase I/II trial showed that the drug is well inc. (vancouver,
tolerated, with no drug-related British Columbia) serious adverse events detected in 26 patients treated to date immunogen inc.
a targeted antibody Cancer Clinical activity was seen starting at doses of 3.3 mg/kg, and its dose-limiting toxicity was the reversible ocular side effects (blurred vision and keratitis) reported with antibody-drug conjugates (adCs) immunomedics inc. Immu-130
phase I data showed it was colorectal cancer therapeutically active in all three conjugated to the trials, but a more frequent dosing schedule was more active in metabolite, Sn-38 To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght phase I data showed that ISa101 pharmaceuticals bV
produced strong immune responses in almost all of the advanced cervical carcinoma patients treated tumor infiltrating data showed evidence of clinical biotechnologies inc.
efficacy from a phase II trial Merck & Co. inc.
responses were observed in (Whitehouse Station, patients with pd-l1 negative tumors; although the preliminary findings for both tumor types suggest that higher pd-l1 expression was associated with higher overall response rates Merrimack
phase I data demonstrated that feraheme was well tolerated inc. (Cambridge,
mass.)
Mirna therapeutics mrX34
Interim safety data from an open- inc. (austin, texas)
microrna mimic of primary liver label phase I study showed that naturally occurring cancer the drug has a manageable safety tumor suppressor noxxon pharma ag noX-h94
lexaptepid pegol; a anemic cancer data from a pilot phase IIa study showed significant increases in designed to bind hemoglobin levels (greater than 1 g/dl) in five of 12 patients (42%) in response to monotherapy oncoethix sa
Interim results from an ongoing molecule inhibitor phase I trial showed linear pharmacokinetics and excellent pfizer inc. (new
phase II paloma-1 data showed it 4/8/14 doubled progression-free survival receptor-positive in an open-label phase II trialbreast cancer provectus
a 10% solution of data showed significant decrease 4/8/14 in melanoma cells in patients' inc. (knoxville,
injected tumors 7-14 days after intralesional pv-10 treatment that was accompanied by similar decrease in uninjected bystander tumors phase II data showed an biotechnology inc.
estimated pCr rate of 55.6% compared to the control arm, which had an estimated pCr rate To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght an ongoing phase Ib/II university of texas md anderson Cancer inc. (South San
Center-sponsored trial of vosaroxin in combination with decitabine in older patients showed that the combination has been found to be effective and well tolerated a cyclin-dependent Chronic In 62 patients, patient response was correlated with mitochondrial inc. (Salt lake City)
response from pre-treatment specimens to peptides known to interact with proteins that promote cell survival: Bim, noxa, Bad, Bmf and hrk Viralytics ltd.
Interim results from the ongoing phase II Calm trial showed antitumor activity in non-injected metastatic tumors in patients participating in the trial, and investigators reported partial or complete reduction of non- injected tumors in multiple patients who had been on treatment at least eight weeks notespublic 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.
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Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght pharma clinical and fda action update april 2014 (location)
abbvie Inc. (north
locally advanced Started a global phase III trial or metastatic squamous non-small-cell lung cancer new results showed that those peptide 1 receptor who previously had been exposed agonist exenatide to exenatide had an advantage of 5.6 points over their controlled-tested counterparts when using the blinding mdS-updrS motor subscale 12 drugs targeted opened a phase II trial testing 12 (london) and pfizer drugs in a single study fda granted orphan drug Bayer healthcare Began enrollment in a phase III trial studying Xofigo injection in combination with abiraterone acetate and prednisone/ resistant prostate prednisolonecancer a polo-like kinase 1 acute myeloid fda and european Commission granted orphan designation germany)Boehringer the Chmp of the ema issued a positive opinion recommending approval of pradaxa Â; fda approved it in patients who have been treated with a parenteral anticoagulant for five to 10 days, and to reduce the risk of recurrent dvt and pe in patients who have been previously treated Combination tablet; type 2 diabetes fda accepted for filing an nda co-transporter-2 germany) and eli peptidase-4 inhibitor To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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Bristol-myers Squibb daclatasvir
Submitted an nda; phase III data 4/8/14; replication complex hepatitis C virus showed that dCv plus aSv given inhibitor and a nS3 in a 24-week regimen gained protease inhibitor Svr12 among treatment-naïve patients (90%), peg-interferon/ribavirin non-responders (82%) and peg-interferon/ribavirin ineligible or intolerant (82%) Bristol-myers Squibb atazanavir a protease inhibitor hIv Co. (new york) and gilead Sciences Inc. cobicistat (foster City, Calif.) daiichi Sankyo Co. oral, once-daily to prevent stroke Began enrolling patients into the direct factor Xa phase III enSure-af study Started a phase Ib/II study to assess the safety and potential growth factor eye efficacy of the eye drops in two doses vs. placebo eisai Co. ltd. (tokyo) Zonegran phase III data showed it was well tolerated and efficacious eli lilly and Co. fda approved Cyramza as a metastatic gastric single-agent treatment endothelial growth cancer or factor receptor 2 gastroesophageal junction adenocarcinoma forest laboratories phase IIb data showed Inc. (new york) and statistically significant gedeon richter plc bipolar I disorder improvements were observed in (Budapest, hungary) the 1.5 mg/day group relative to placebo at six weeks for the primary endpoint, the montgomery-asberg depression rating Scale total score, and the key secondary endpoint, the Clinical global Impressions – Severity score glaxosmithkline plc tanzeum subcutaneous injection glaxosmithkline plc ellipta received marketing authorization 4/18/14 pulmonary disease, including chronic bronchitis and emphysema To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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Ipsen Sa (paris)
an injectable form phase III data demonstrated a of botulinum toxin statistically significantly higher proportion of responders in muscle tone improvement vs. placebo janssen Biotech Inc. Sylvant janssen research & prezista once-daily, fixed- dose antiretroviral (raritan, n.j.; part of combination tablet johnson & johnson) containing protease inhibitor prezista (darunavir) and cobicistat european Commission approved therapy combining mellitus (Beerse, Belgium) canagliflozin and immediate-release metformin hydrochloride in a topical botulinum Clinical data showed significant pharmaceuticals Inc. reductions in both urinary urgency (pittsburgh, pa.) and urinary frequency merck & Co. Inc. hepatitis C virus phase II data showed sustained (Whitehouse Station, mk-8742 protease inhibitor viral responses with 12-week dosing in genotype 1 treatment- replication complex naïve patients reached 94% to 98%, even without ribavirin merck & Co. Inc. (Whitehouse Station, n.j.)novartis ag (Basel, the data monitoring committee receptor neprilysin failure with unanimously recommended early reduced ejection closure of the paradIgm-hf phase III study, indicating patients with chronic heart failure with reduced ejection fraction (hf-ref) who received lCZ696 lived longer without hospitalization than those who received standard care novartis ag (Basel, lymphoma kinase-positive metastatic non-small-cell lung cancer To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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pfizer Inc. (new
top-line phase III data showed that tofacitinb, as a 5-mg or 10-mg dose taken as a pill twice daily, met the primary endpoints of statistically significant superiority over placebo roche ag (Basel, received a positive opinion from rheumatoid arthritis phase III data showed a reduction 4/29/14 (vaccines division of of 56% of dengue disease cases Sanofi Sa; paris)Sanofi pasteur mSd gardasil european Commission granted marketing authorization for a 2-dose schedule at 0 and six months in children ages 9 to 13 Sanofi Sa (paris) fda expanded the approved age reduced diphtheria tetanus, indication of adacel for active toxoid and acellular diphtheria and booster immunization for the pertussis vaccine prevention of tetanus, diphtheria and pertussis as a single dose in subjects, ages 10 through 64 teva pharmaceutical generic lovaza omega-3-acid ethyl hypertri- approved in the u.S.
(jerusalem)teva pharmaceutical pridopidine enrolled the first patient in the pride-hd study, a phase II trial (jerusalem)teva pharmaceutical duoresp a new dry-powder the european Commission has inhaler containing a chronic granted marketing authorization fumarate dehydrate disease twi pharmaceuticals generic extended-release phase I data demonstrated it was 4/30/14 laboratories Inc. rapidly absorbed and exhibited a (maple grove, minn.) notesBla = Biologics license application; Cma = Continuous marketing application; fda = food and drug administration; Ind = Investigational new drug application; nda = new drug application; pdufa = prescription drug user fee act; Spa = Special protocol assessment.
public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.
the date indicated refers to the BioWorld Today issue in which the news item can be found.
To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght non-u.s. clinical trials and regulatory actions april 2014 european independent board of sa (paris)
experts in charge of the safety profile of the relive phase III trial unanimously recommended continuing the study without modification Cytrx Corp. (los
received orphan medicinal doxorubicin with a product designation from the european Commission that binds specifically to albumin erytech pharma sa graspa/ery-
Was granted orphan drug designation by the fda e-therapeutics plc
the uk phase Ia trial showed it was well tolerated at all six dose escalations with no serious diverse events adjunct treatment patient enrollment was initiated in 4/10/14 japan in a phase III trial inc. (Cambridge,
regeneron
the first phase II study in japanese 4/2/14 antibody targeting patients met its primary endpoint inc. (tarrytown, n.y.)
and sanofi sa
(paris)
CENTRAL NERVOUS SYSTEM
Coronado
trichuris suis ova autism spectrum launched in jerusalem a biosciences inc.
randomized, double-blind, (Burlington, mass.) placebo-controlled, study of 60 patients ages 6 to 17 intelgenx Corp. (St. versafilm
Initiated a comparative laurent, Quebec) bioavailability clinical study and redhill
comparing versafilm to the biopharma ltd. (tel-
european reference drug To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght methylnaltrexone Submission to the ema has been accepted for review ltd. (raleigh, n.C.)
and progenics
pharmaceuticals
inc. (tarrytown, n.y.)
INFECTION
Chongqing Zhifei
aC-hib vaccine vaccine against the Cfda gave the green light to biological products
Chongqing to launch its joint Co. ltd. (hong
meningococcus and vaccine against group a and C meningococcus and haemophilus influenzae type B influenzae type B targets microsomal Chronic plans to begin phase I testing in ltd. (mumbai, India)
diseases and pain management to prevent graft european Commission has granted an orphan drug inc. (Cheshire,
organ transplantation ema's Committee for orphan medicinal products adopted a inc. (Cambridge,
positive opinion topical version of approved in luxembourg; inc. (San diego)
approved in Spain hospira inc. (lake
european data showed it appeared 4/29/14 to be as safe as other epoetin alfa products ema determined that the firm's inc. (new york)
chronic kidney disease thrombogenics nV jetrea
Was approved in malaysia (leuven, Belgium) notesBla = Biologics license application; Chmp = european Committee for medicinal products for human use; Cma = Continuous marketing application; ema = european medicines agency; fda = food and drug administration; Ind = Investigational new drug application; maa = marketing authorization application; nda = new drug application; pdufa = prescription drug user fee act; Spa = Special protocol 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, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght fda submissions, approvals & other actions april 2014 fda said its complete response pharmaceutical inc.
submission to the november 2013 (lexington, mass.) partial clinical hold showed it is safe to proceed; it was placed on partial clinical hold following the death of a patient who progressed to liver failure approximately one month following discontinuation of CudC-427 dosing genmab a/s
fda approved a supplemental (Copenhagen) and Bla for the use of arzerra in glaxosmithkline plc
combination with chlorambucil for the treatment of previously untreated patients with Cll for whom fludarabine-based therapy is considered inappropriate kite pharma inc.
fda granted orphan drug engineered t-cell product designed to target Cd19 expression pharmacyclics inc.
Submitted a supplemental nda, (Sunnyvale, Calif.) based on data from the phase III leukemia or small reSonate study, pyC-1112, a lymphocytic head-to-head comparison of Imbruvica vs. arzerra (ofatumumab, genmab a/S and glaxosmithkline plc) in 391 patients fda granted orphan drug inc. (Salt lake City)
CARDIOVASCULAR
biogen idec inc.
Coagulation factor hemophilia B fda approved it for the control (Cambridge, mass.) IX, fc fusion protein and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia B dyax Corp.
ecallantide; peptide acute hereditary Won fda approval to treat (Cambridge, mass.) inhibitor of plasma angioedema patients older than 12 years To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght (location)
the Medicines Co.
to control mild or fda accepted the filing of a Bla (parsippany, n.j.) for hemostatic agent fibrocabs CENTRAL NERVOUS SYSTEM
impax
resubmitted an nda with updated safety and stability (division of Impax laboratories Inc.; intelgenx Corp.
fda acknowledged receipt of the companies' response to the Quebec) and redhill
complete response letter and has biopharma ltd. (tel
requested further clarifications aviv, Israel)
kaleo inc.
pozen inc. (Chapel
fda issued a complete response friendly aspirin qrxpharma ltd.
fda's anesthetic and analgesic release dual opioid severe acute pain drug products advisory (oxycodone and Committee voted to recommend DIABETES
Mannkind Corp.
fda's endocrinologic and (valencia, Calif.) metabolic drugs advisory Committee recommended approval psivida Corp.
diabetic macular the fda set a pdufa date of (Watertown, mass.) Sept. 26 for Iluvien following and alimera
intravitreal insert resubmission of the nda sciences inc.
(atlanta)
INFECTION
Cubist
fda committee recommended pharmaceutical inc.
approval, and a study specifically (lexington, mass.) in adolescents and teens before the label is extended for pediatric use inc. (lexington,
complicated intra-abdominal infections To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght fda committee recommended therapeutics inc.
second-generation, skin and skin edmond pharma srl erdosteine
a small-molecule fda granted orphan drug emergent
fda has granted orphan drug biosolutions inc.
(rockville, md.)
enanta
oral, interferon-free Chronic genotype Submitted an nda inc. (Watertown,
mass.) and abbvie
inc. (Chicago)
gilead sciences inc. Cobicistat
fda accepted the company's (foster City, Calif.) refiling of two ndas boosting, agent that increases blood levels of the protease inhibitors atazanavir and darunavir, and the integrase inhibitor elvitegravir fda said the nda "should be therapeutics inc.
fileable" with existing data (Cambridge, mass.) RESPIRATORY
alk-abello a/s
an allergen extract ragweed allergic fda approved the Bla for (Copenhagen) and ragwitek as an immunotherapy Merck & Co. inc.
for short ragweed pollen-induced (Whitehouse Station, allergic rhinitis, with or without conjunctivitis, confirmed by positive skin test or in vitro testing for pollen-specific Ige antibodies for short ragweed pollen greer laboratories
under-the-tongue allergic rhinitis fda approved oralair to treat inc. (lenoir, n.C.)
allergic rhinitis with or without conjunctivitis notesBla = Biologics license application; Cma = Continuous marketing application; fda = food and drug administration; Ind = Investigational new drug application; nda = new drug application; pdufa = prescription drug user fee act; Spa = Special protocol assessment.
public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.
the date indicated refers to the BioWorld Today issue in which the news item can be found.
To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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monday, may 19, 2014 BIoWorld™ InSIght 10 BiGGest u.s. winneRs foR the week 10 BiGGest u.s. LoseRs foR the week by percent
by dollars
by percent
by dollars
regeneron pharma 16.02 -32.34 puma Biotech -25.00 portola pharma -24.46 ani pharma accentia Biopharma -22.45 agios pharma THE EMERGING BIOSIMILARS DRUG DEVELOPMENT MARKET… OPPORTUNITY OR THREAT?THE BIOSIMILARS GAME: AVAILABLE IN PRINT AND PDF A scorecard for opportunities, threats and
Visit us at www.bioworld.com or call 1-800-477-6307 or • Provides in-depth analysis of the various markets 1-770-810-3144 to purchase (31 countries covered) • Includes market regulatory strategies• Profiles 139 key players developing biosimilars• More than 75 major biosimilar deals are profiled• Data on 276 biosimilars now in the pipeline• And much more… 2013 Thomson Reuters. All rights reserved. Thomson Reuters and the Kinesis logo are trademarks of Thomson Reuters.
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Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght BiowoRLd stock RepoRt foR puBLic BiotechnoLoGy coMpanies syMBoL cLose cLose syMBoL cLose cLose acceleron pharma accentia Biopharma aBpI 0.0049 0.0038 -22.45 Crmd 1.74 1.55 -10.92 25.00 612 -4.30 -19.58 5446 -6.47 -33.33 2128 -2.64 -55.24 4859 dara Biosciences 9.28 -29.10 12694 depo 10.57 10.93 aeterna Zentaris -8.33 -14.44 1119 -2.88 -21.97 1482 albany molecular -2.82 -17.77 4835 adhd 15.85 16.82 -5.07 -33.16 8282 eglt 11.75 11.35 -3.40 -5.42 172 alimera Sciences alSe 0.04 0.03 -25.00 50.00 25 -3.12 -38.95 6223 -9.27 -30.46 13082 ambit Biosciences -3.48 -45.68 9240 five prime thera applied genetic tech agtC -9.43 -29.26 2470 galena Biopharma gale -6.87 -56.25 9028 aryX 0.0105 0.01 3.89 -60.55 13626 6.48 -29.03 -28.08 549 -4.24 -38.25 3085 4.07 -48.83 22688 4.86 -44.06 10214 Bio-path holdings -4.72 -22.97 3488 CpXX 2.65 2.47 -6.79 -18.21 47 -4.22 -18.53 2900 -27.02 -38.39 11880 Cell therapeutics Intercept pharma 19.07 310.46 1935 -4.88 132.93 10167 4.22 -44.94 22001 5.47 -38.00 17653 -32.34 -38.94 1930 karyopharm thera kythera Biopharma kyth Cleveland Biolabs -0.70 -21.67 5463 mgnX 19.01 19.09 To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
Copyright 2014 Thomson Reuters. Reproduction is strictly prohibited. Visit our web site at www.bioworld.com monday, may 19, 2014 BIoWorld™ InSIght BiowoRLd stock RepoRt foR puBLic BiotechnoLoGy coMpanies syMBoL cLose cLose syMBoL cLose cLose tetralogic pharma tetraphase pharma ttph -4.75 -34.69 1058 myrX 0.18 0.17 -5.56 -5.56 99 the medicines Co. -3.47 -22.48 2110 neurocrine Biosci ngSX 0.008 0.0093 16.25 132.50 30 transcept pharma newlink genetics 0.86 -15.69 -30.08 1417 rare 36.55 34.48 -5.66 -18.39 822 ugneQ 0.0008 0.0005 -37.50 -16.67 43 -14.56 -10.10 2354 oncogenex pharma ogXI -5.30 -56.45 1090 vrml 3.04 2.95 -2.96 25.00 103 vSar 28.02 27.53 -7.65 -46.81 9351 oxis International 14.02 -34.96 1903 -0.27 -46.06 8256 pacific Biosciences paCB -7.42 -38.37 5220 pain therapeutics peregrine pharma London stock exchanGe 5/9 5/16 %Wk %ytd volume 305 289.25 -5.16 -6.69 horizon discovery hZd 179.925 169.975 -5.53 -20.76 oxford Biomedica progenics pharma 33.37 -21.01 -28.85 protalix Biothera 237 -1.25 106.90 note: prices are denoted in pence.
toRonto stock exchanGe -16.02 -10.66 1227 5/9 5/16 %Wk %ytd volume regado Biosciences rgdo regeneron pharma regn Bioniche life Sci 0.18 -10.00 -43.75 0.57 0.00 -25.00 oncolytics Biotech onC rvX 0.66 0.66 0.00 37.50 theratechnologies note: prices are denoted in Canadian dollars.
trading volumes for nasdaq, amex and nySe are recorded as the total Seattle genetics number of shares traded (in thousands) on a weekly basis (cumulative monday through friday); the weekly and ytd % changes are from Ipo completion, where applicable. average percent Change week: -0.27%
range: -37.50% to +38.05%; number of Companies: 288 (does not include lSe or tSX; not market weighted) average percent change ytd: -2.34% range: -72.25% to +310.46%; number of Companies: 288 (does not include lSe or tSX; not market weighted) To subscribe, call (770) 810-3144 or (800) 477-6307 if calling from the U.S. and Canada or email [email protected]. For customer service inquiries call (215) 386-0100 or (800) 336-4474 if calling from the U.S. and Canada or email [email protected].
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Source: http://ihuman.shanghaitech.edu.cn/wp-content/uploads/2014/06/BWIS05192014.pdf