cndo 11-12-11 bioworld insight

Jan. 1 – Dec. 9, 2011: $22,777.24MJan. 1 – Dec. 9, 2010: $18,987.47M

* Includes financings of public biotech firms with the exceptions of public offerings and certain investments from corporate partners.

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BIOTECH SHORT SELLING UP SLIGHTLY, BUT NOT ALL BAD ...................... 2FORM 10 AS IPO ALTERNATIVE? CORONADO WOULD DO IT AGAIN ........ 3MONEY RAISED BY BIOTECH IN 2011 ................................................... 4WEEK IN REVIEW ................................................................................ 5WORD ON THE STREET, WEEK IN WASHINGTON ..................................... 6PHASE I, II & III CLINICAL TRIALS UPDATE: NOVEMBER 2011 .... 7, 11, 15CONFERENCE DATA ...................................................................... 19-25NON-U.S. CLINICAL TRIALS DATA; FDA ACTIONS ......................... 26, 28

THIS WEEK IN BIOWORLD INSIGHT

Money Raised By Biotech In 2011 Vs. 2010

MONDAY

DEC. 12, 2011VOL.19, NO. 50

PAGE 1 OF 32

9,792

8,634

4,351

6,700

7,863

4,425

01,0002,0003,0004,0005,0006,0007,0008,0009,000

10,00011,000

PublicOfferings

Public/Other*

PrivateBiotechs

Mill

ions

See Stem Cells, page 4

In Geron’s Wake

Deals, Data Spark Stem Cell Interest, But Hurdles RemainBy Trista Morrison

Editor

SAN DIEGO – Geron Inc.’s exit from the embryonic stem cell fi eld last month shocked many in the biotech industry – but not the regenerative medicine experts attending the recent 2011 Stem Cell Meeting on the Mesa.

Some attendees muttered that Geron had never been able to effectively balance the confl icting risk-reward profi les of its stem cell business and its cancer business. Others whispered that the writing had been on the wall since the departure of longtime CEO Thomas Okarma in February. Still others wondered if perhaps the preliminary data from Geron’s Phase I spinal cord injury trial – the world’s fi rst of a human embryonic stem cell therapy – weren’t as good as had been hoped.

Geron has maintained the latter isn’t the case – the company blamed its withdrawal from the stem cell space on capital scarcity and uncertain economic conditions. (See BioWorld Today, Nov. 16, 2011 .)

Regardless of the reason, regenerative medicine insiders don’t view Geron’s move as an indication that the stem cell industry is on shaky ground – at least, not any shakier than it’s ever been. If anything, data and deal-fl ow indicate increasing interest in stem cells, although maintaining that momentum will require creativity on several fronts.

Kicking into GearMahendra Rao, director of the National Institutes of

Health’s Center for Regenerative Medicine, noted that the stem cell fi eld has been signifi cantly derisked over the past few years. Most of the technical risk with adult stem cells

“has been sorted out,” he said, and advances in research tools have improved the consistency of embryonic stem cell work as well. On the embryonic side, Geron also blazed a regulatory trail with the FDA by getting into the clinic.

“The greatest obstacle now is to deliver compelling clinical proof of concept data,” said Gil Van Bokkelen, CEO of Athersys Inc. and chairman of the Alliance for Regenerative Medicine. “That’s what investors are looking for; that’s

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See Shorts, page 6

A Difference of Opinion

Biotech Short Selling UpSlightly, But Not All BadBy Brian Orelli

BioWorld Insight Contributing Writer

Short sellers are generally considered blasphemers by biotech fi rms. And for good reason; short sellers borrow shares in order to sell them, which puts negative pressure on the stock. The only way shorts make money is if shares go down.

But biotechs don’t have much to complain about, according to data compiled by BMO Capital Markets. Short sellers are only slightly more active in the biopharma sector than they are for the entire Nasdaq. Looking at the short interest as a percent of equity fl oat since the beginning of 2007, the 302 publicly traded biopharmas used in the study had a mean of about 6.6 percent of their available shares sold short, which is only about a percentage point higher than the short interest for all the Nasdaq stocks.

Considering the large number of binary events in biotech – data read-outs, FDA decisions, etc. – Annette Grimaldi, managing director at BMO, thought the absolute difference might have been larger. One possible reason is that the binary events also detract short investors because the losses on a short position are theoretically limitless, unlike a long position where the most an investor can lose is the initial investment.

The means of the short interest ratio – dividing the short interest position by the average daily trading volume – were nearly identical at 7.8 and 7.6 for Nasdaq and biopharma stocks, respectively. In other words, it would take almost eight days for the shorts to cover all their outstanding short sales at the average trading volume.

Both the short interest as a percent of equity fl oat and the short interest ratio spiked in the middle of 2008 during the peak of the recession. That’s not surprising as both measurements describe how safe it is to put on a short position. As the numbers increase, it becomes harder to unwind the short.

Despite continued volatility in the market, the measurements of short activity have returned to normal levels.

There has, however, been a slight widening of the short interest as a percent of equity over the last two and a half years between biopharma and Nasdaq stocks, with the biopharma stocks closer to 1 .5 percentage points higher than the Nasdaq stocks, up from the previously mentioned mean of about 1 percentage point. Grimaldi offered up a couple of potential explanations: “It may mean that they’re betting against the sector or there may just be a larger number of companies where it would be profi table to put on a short position.”

Not every short position is made by an investor who believes the shares are overvalued. Short sales are used to hedge long positions in either the individual stock, the sector, or the entire investor’s portfolio. They can also be used as a tax deferral strategy.

Shorts also increase liquidity of shares, which can be helpful for biotechs. Higher liquidity, for instance, helps

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Weighing Pros, Cons

Form 10 as IPO Alternative?Coronado Would Do It AgainBy Trista Morrison

Editor

Four months ago, Coronado Biosciences Inc. embarked on a route to going public not often seen in the biotech industry: the Form 10 pathway. (See BioWorld Today, July 20, 2011 .)

In essence, the Form 10 pathway offers an alternative to the initial public offering (IPO) or reverse merger, seeking to avoid the market volatility and high transaction costs of the former as well as the trading restrictions and investor baggage of the latter. A private fi rm looking to use the pathway to go public simply fi les a Form 10 with the SEC to become a public reporting company, fi les an S-1 to register its shares, and applies to trade those shares on a market. (See BioWorld Insight, July 25, 2011 .)

Coronado got started down the Form 10 road because such fi lings are required for private companies with 500 or more stockholders of record. The rule most often affects large private tech fi rms like Facebook, but Coronado’s angel-backed business model made it one of the rare few private biotechs to hit the stockholder threshold. Although a bill is pending in the House that may raise that threshold, under current law, Coronado had to become a reporting company.

Coronado did not, however, have to start publicly trading its shares – but the company was interested in accessing the public markets. President and CEO Bobby Sandage said Coronado had considered a reverse merger and even went as far as to identify a suitable shell company to merge into, but new restrictions from the SEC made that option less appealing. Meanwhile, although fi ve biotechs have jumped into the IPO queue in the last few months, the window remains far from what most would consider open. (See BioWorld Today, Nov. 11 , 2011 .)

Hence Coronado opted for the Form 10 route. Sandage said the process is not well understood and “even with experienced SEC lawyers we were sort of fi guring things out as we went along,” but he believes it was the right decision and would do it again with future start-ups.

Coronado debuted on the OTCQB last month at $11 per share. That price was market-driven, determined not by a team of investment bankers negotiating with institutional investors, but simply by what a buyer was willing to pay and a seller was willing to accept. Interestingly, Coronado’s price was higher than that of three-quarters of the biotechs to price IPOs this year, even though the Burlington, Mass.-based fi rm is barely in the clinic.

Since its pricing, however, Coronado’s shares (OTCQB:CNDO) have dropped 34 percent to $7.25, as of mid last week. That’s not necessarily unusual for a newly public biotech – several members of this year’s IPO class have fallen just as hard, although as a group they are down about 8 percent. The drop does, however, illustrate some of the challenges of taking the Form 10 route.

Unlike in an IPO, Coronado didn’t have investment bankers trotting management around to meet with institutional investors and raise awareness. “With Form 10, you essentially have to do it yourself,” Sandage explained, adding that he and chief operating offi cer Noah Beerman have been “on the road nonstop” talking to investors and analysts, trying to get the word out.

For now, trading volume among Coronado’s largely retail base is low, but Sandage isn’t overly concerned, noting that “IPOs don’t trade that much after the fi rst week or two either.” And although Coronado’s price has fallen, Sandage is encouraged by the fact that the fi rm was recently picked up by Roth Capital Partners with a buy rating and a $27 price target. He’s also optimistic that more analyst coverage and interest from institutional investors will come once the fi rm transfers to Nasdaq, a process that is already underway.

Beerman also believes Coronado’s stock price will benefi t from the achievement of some near-term milestones. The company recently fi led an investigational new drug application for lead program CNDO-201 , pig whipworm eggs, and a Phase I trial is underway. Safety data are expected in the fi rst quarter of 2012, and a Phase II trial for Chron’s disease is expected to start in the second quarter. Meanwhile Coronado’s second program, which involves ex-vivo activation of NK cells, is poised to start a Phase I/II trial in acute myeloid leukemia early next year.

It remains to be seen whether or not other biotechs follow in Coronado’s footsteps and use the Form 10 pathway to go public. They may soon have yet another IPO alternative: a bill that has passed the House and is now in the Senate would raise the Regulation A exemption to $50 million, providing privately held biotechs with the opportunity to raise money and start trading their shares without fi ling a Form 10 to become a public reporting company. (See BioWorld Insight, Nov. 21 , 2011 .)

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Money Raised By Biotech: Jan. 1 - Dec. 9, 2011

Public Offerings 43% ($9,792M)

Public/Other 38% ($8,634M)

Private Biotechs 19% ($4,351M)

38%($8,634M)

43%($9,792M)

19%($4,351M)

Stem CellsContinued from page 1

what pharma is looking for. Once that data gets delivered, it will change the mindset in a big way.”

In the adult stem cell space, data is starting to trickle out. Morrie Ruffi n, managing director of the Alliance for Regenerative Medicine, noted that while the media were busy reporting on Geron, many missed the fact that Mesoblast Ltd.’s allogeneic adult stem cell product Revascor signifi cantly reduced cardiovascular deaths in a randomized, placebo-controlled Phase II trial, and Aastrom Biosciences Inc.’s autologous expanded cell therapy ixmyelocel-T signifi cantly reduced treatment failures in a randomized, placebo-controlled Phase II critical limb ischemia trial. (See BioWorld Today, Nov. 16, 2011 .)

There’s been good news on the partnering front as well. Every panel at the conference paid homage to Mesoblast’s $350 million stem cell deal with Cephalon Inc. (now part of Teva Pharmaceutical Industries Ltd.), which carried a whopping $130 million up front, as well as to Shire plc’s $750 million buy-out of regenerative medicine fi rm Advanced BioHealing Inc. (See BioWorld Today, May 19, 2011 , and Dec. 9, 2010.)

“Things move forward at a certain rate and then kick into gear,” said Robert Preti, president and chief scientifi c offi cer of Progenitor Cell Therapy LLC, which itself was recently the subject of some business development activity, having been acquired by NeoStem Inc. NeoStem also acquired stem cell fi rm Amorcyte Inc. earlier this year. (See BioWorld Today, July 18, 2011 .)

Another sign of momentum can be seen in private fi nancing for stem cell and regenerative medicine fi rms. Four such fi rms have completed private offerings this year, according to data from Roth Capital Partners, and three of them brought in at least $30 million. Additionally, three fi rms in the fi eld – Argos Therapeutics Inc., TVAX Biomedical Inc. and Verastem Inc. – have fi led to go public, and Advanced BioHealing was in the initial public offering queue as well before its acquisition.

Not Out of the WoodsSo is the fi nancial situation for regenerative medicine

companies really that much worse than for biotech as a whole, as Geron’s retraction indicated? It’s somewhat hard to believe, sitting in the brand-spanking-new $127 million laboratories of the Sanford Consortium for Regenerative Medicine, listening to so many examples of progress and success. But according to Geoff Whitehead, principal with Flagship Ventures, the answer is yes.

The outlook for stem cell fi rms is not quite as dire on the private equity side. Venture capitalists are looking for transformative ideas, large markets and compelling entrepreneurs, so the fact that a lot of stem cell work is early stage and hard to value “doesn’t really factor into things,” Whitehead said. Flagship has three regenerative medicine fi rms in its portfolio.

But on the other hand, Whitehead noted that “early stage VCs like to invest in pioneers,” and the stem cell fi eld’s pioneers don’t have the best track record thus far. He then ticked off a dozen reasons why it would be easier for the average VC to say “no” to a stem cell investment than “yes.”

Stem cell start-ups do have the benefi t of alternative options like grant funding – the California Institute of Regenerative Medicine (CIRM) has been doling out money for several years now. But Edward Lanphier, president and CEO of Sangamo BioSciences Inc., noted that the only companies able to go public these days are in Phase III, and “is CIRM going to take someone to Phase III? I don’t think so. There’s a need for venture capital. I’m not optimistic that it’s going to be met, but there’s a need.”

Even if a stem cell company does get public, that doesn’t mean their fortunes will improve. Lisa Walters-Hoffert, managing director with Roth, presented an analysis showing that just nine public stem cell and regenerative medicine fi rms have raised money this year – less than $140 million altogether, and with a hefty average 67.5 percent warrant coverage. Since their offerings, the fi rms have seen their shares fall an average of 24 percent, and only one company – StemCells Inc. – is in the green.

Further, despite the oft-mentioned Mesoblast and Advanced BioHealing deals, business development in the space has yet to catch fi re.

“We spend a lot of time talking to pharma,” said Sangamo’s Lanphier. “As it relates to cell therapy, there is

See Stem Cells, page 5



W E E K I N R E V I E W

Financings

Dendreon Corp. raised $125 million by monetizing its share of royalties on Victrelis (boceprevir).EMBL Ventures GmbH closed a $53.5 million fund for German life science companies.Forbion Capital Partners closed its second FCF 1 Co-Investment Fund at about $50 million.Gilead Sciences Inc. priced $3.7 billion worth of senior unsecured notes in a public offering.

Deals

Biogen Idec Inc. entered a $300 million joint venture with Samsung for biosimilars.Medicis Pharmaceutical Corp. acquired the assets of Graceway Pharmaceuticals LLC for $455 million.Molecular Partners AG signed an immunology discovery deal with Janssen Biotech Inc.

Momenta Pharmaceuticals Inc. acquired Virdante Pharmaceuticals Inc.’s protein sialylation programs.

. . . And More

See BioWorld Today for coverage of the San Antonio Breast Cancer Symposium.Affymax Inc.’s peginesatide got a thumbs up from an FDA advisory committee.Curemark LLC reported positive Phase III data with CM-AT in children with autism.Genentech Inc./Roche AG’s pertuzumab had good Phase III breast cancer data.Theratechnologies Inc. is halting its chronic obstructive pulmonary disease muscle wasting program and downsizing by 60 percent.Unigene Laboratories Inc.’s parathyroid hormone was dropped by partner GlaxoSmithKline plc.

interest, but there is a higher bar. Whether it’s safety, effi cacy, cost of goods analysis . . . you’ve got to paddle your own canoe a lot longer.”

Matthias Steger, global head of research and technology partnering with Roche AG, provided further perspective. While he refuted the notion that big pharma is not involved in stem cells, pointing to Roche’s own signifi cant investment, he cautioned that big pharma “can just not afford running into big failures. We have to sort out the science. It would be a mistake to rush. I think we are making progress, but we have to be a little bit patient.”

Just Keep PaddlingThe trick will be for stem cell fi rms to fi gure out how they

can afford to patiently wait for the catalyst that captures big pharma’s attention, or the proof-of-concept data that drive public market interest.

The experts at the conference said it will require an unprecedented level of public-private partnership. “It can’t be industry alone, or academics alone, or government alone – there must be collaboration,” said NIH’s Rao. He added that there are far more stem cell trials being conducted by academics than by industry, but the former have “no idea how to take it to the next level.” That’s where government needs to focus its efforts, he said.

Susan Solomon, CEO of the New York Stem Cell Foundation, added that nonprofi ts can play a role by doing the

“high-risk work” that neither grant-funded researchers nor companies can stomach. Nonprofi ts should also focus on the translational work that academics aren’t interested in because it won’t result in a published paper – the “work that is repetitive but essential to derisk the program to a point where investors or pharma can pick it up,” she said.

Meanwhile, history (and Geron) has proven the traditional drug development business model isn’t a particularly good fi t for stem cell fi rms. Whether public or private, the fi rms that have managed to stay in business are those that have, more often than not, found alternative sources of funding.

Sangamo, for example, has outlicensed its zinc fi nger protein technology in non-core fi elds like agriculture and research tools. Cytori Therapeutics Inc. is bringing in revenue by selling its Celution adult stem cell processing system in Europe, where it has received a CE Mark, while it navigates the U.S. regulatory path. Organovo Inc. uses its platform to create disease models for partners, as well as in its own drug development. Neostem generates revenues from a cell therapy manufacturing business, a stem cell bank and several initiatives in China, which help support the cost of developing its cell therapy pipeline.

The approaches are unconventional, but Greg Lucier, chairman and CEO of Life Technologies Corp., thinks that’s what the regenerative medicine industry needs. “I do believe this is an area where we should be pushing the frontiers,” he said. “We’re positive, we’re confi dent and we are investing in this area. The horizon we see is near term and incredibly bright.”

Stem CellsContinued from page 4



W E E K I N W A S H I N G T O N

W O R D O N T H E S T R E E T

“If you’re a great company with great promise, there’s money out there.” – Geoff Whitehead, principal with Flagship Ventures, on funding diffi culties for stem cell fi rms

“The world of pharma is waking up [to the fact] that this is going to be the next frontier.” – Greg Lucier, chairman and CEO of Life Technologies Corp., on stem cells

“You want to have Track 1 , 2, 3, 4 and 5 – if you only have Track 1 and Track 5, no one wants to give it a shot.”– William Hicks, partner with Mintz Levin, on the need to have multiple avenues to going public

“The hurdle rates for companies in development are being set higher than they have been in the past. Having said that, we love being in this industry.” – Anthony Marucci, president and CEO, Celldex Therapeutics Inc.

The FDA’s latest proposed user fee structure for biosimilars will charge 10 percent of the new drug application fee, plus a development fee.

The U.S. Patent and Trademark Offi ce and China’s State Intellectual Property Offi ce will expedite their patent examinations, which should allow applicants to obtain corresponding patents faster in each country.

fi rms sell more shares in at-the-market (ATM) offerings, a public offering alternative that has slowly gained traction over the past few years. Inhibitex Inc. raised $20 million through an ATM last month. (See BioWorld Insight, Sept. 7, 2009.)

And short sellers can actually have a positive effect on shares if a company produces positive news. A short squeeze occurs as the short sellers cover their sales by buying back shares, amplifying the upward movement of the stock in response to the good news.

ShortsContinued from page 2

While shorts might be best ignored from an industry perspective, Grimaldi thinks individual fi rms should probably consider why investors are shorting the shares. “Especially large short interest is a signal that differs from what management is saying. For that particular company, they should look to see if there are any underlying messages in that position.”

China Biologic Products Inc., Cumberland Pharma-ceuticals Inc., MannKind Corp., BioTime Inc., and Oncolytics Biotechnology Inc. currently top the list of companies with the highest short interest.

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FDA Approvals In NovemberCompany Drug Indication

Bristol-Myers Squibb Co. Erbitux Head and neck cancer

EUSA Pharma Inc. Erwinase Acute lymphoblastic leukemia

Incyte Corp. Jakafi Myelofibrosis

IntelGenx Corp. CPI-300 Major depressive disorder

Millennium Velcade Updated label for multiple myeloma

Pacira Pharmaceuticals Inc. Exparel Postsurgical pain relief

Regeneron Pharmaceuticals Inc. Eylea Wet age-related macular degeneration

Spectrum Pharmaceuticals Inc. Zevalin Removed bioscan requirment for non-Hodgkin’s lymphoma

Transcept Pharmaceutical Inc. Intermezzo Insomnia

Phase I Clinical Trials Update: November 2011Company (Location)

Product Description Indication Status (Date)#

CANCER

Bavarian Nordic

A/S (Kvistgard, Denmark)

CV-301 Off-the-shelf immu-notherapy

Metastatic breast or ovarian cancer

A trial with 26 heavily pretreated patients who received monthly vaccinations showed a median time to progression of 2.5 months in the breast cancer group, and median overall survival was 13.7 months (11/10)

Celldex

Therapeutics Inc.

(Needham, Mass.)

CDX-1127 A co-stimulatory molecule on T cells

Malignant solid tumors or hema-tologic cancers

Started a Phase I study (11/9)

CureVac GmbH

(Tuebingen, Germany)

CV9201 mRNA-based can-cer vaccine

Non-small-cell lung cancer

Phase I/IIa data showed the drug was well tolerated and biologically active (11/8)

CytRx Corp. (Los Angeles)

INNO-206 A tumor-targeting conjugate of doxo-rubicin

Sarcoma Phase Ib/II trial data determined a maxi-mum tolerated dose of the drug deliver-ing doxorubicin at the equivalent of three and a half times standard doxorubicin doses (11/1)

Hutchison

MediPharma Ltd.

(London)

Epitnib (HMPL-813)

An orally active small molecule inhibitor targeting the epidermal growth factor receptor

Cancer Started the first-in-human Phase I trial (11/7)

Infinity

Pharmaceuticals

Inc. (Cambridge, Mass.)

IPI-145 Oral inhibitor of phosphinositide-3-kinase delta and gamma

Advanced hematologic malignancies

Started two Phase I trials (11/1)

Lentigen Corp.

(Gaithersburg, Md.)LG631 Gene therapy Cancer Started a Phase I trial (11/15)

Senesco

Technologies Inc.

(Bridgewater, N.J.)

SNA01-T Intravenous infusion Multiple myeloma

Initiated patient dosing in its Phase Ib/IIa study (11/4)



Company (Location)


Silence

Therapeutics plc

(London)

Atu027 siRNA drug Solid tumors Phase I data showed 37% of patients had stable disease after treatment (11/9)

CARDIOVASCULAR

Cardiome

Pharma Corp.

(Vancouver, British Columbia) and Merck & Co. Inc.

(Whitehouse Station, N.J.)

Vernakalant Oral therapy Atrial fibrillation Recently completed an additional multi-ple rising-dose Phase I study and data showed the drug was well tolerated (11/7)

Nile

Therapeutics Inc. (San Mateo, Calif.)

Cenderitide Subcutaneous infusion

Chronic heart failure

Phase I data showed that weight-based doses achieved target PK levels when delivered by subcutaneous pump, and that it was well tolerated, bioavailable and reduced PK variability compared to a fixed-dose regimen (11/17)

Pozen Inc. (Chapel Hill, N.C.)

PA32540 A combination of 325 mg aspirin and 40 mg immediate-release omeprazole

Gastrointestinal risk in patients who require dual antiplatelet ther-apy and gastro-protection

Phase I data showed it was associated with greater platelet inhibition when dosed 10 hours apart from Plavix com-pared to synchronous administration of aspirin, clopidogrel and delayed-release omeprazole at day seven (11/15)

CENTRAL NERVOUS SYSTEM

Acadia

Pharmaceuticals

Inc. (San Diego)

AM-831 A small molecule that combines mus-carinic m1 partial agonism with dopa-mine D2 and sero-tonin 5-HT2A antagonism

Schizophrenia FDA cleared it to begin a Phase I trial (11/8)

Anavex Life

Sciences Corp.

(Hoboken, N.J.)

Anavex 2-73 The first of a new class of oral drugs

Alzheimer’s disease

Completed a Phase I single ascending dose trial of Anavex, showing it was well tolerated below the 55 mg to 60 mg dose with only mild adverse events (11/15)

Cara

Therapeutics Inc. (Shelton, Conn.)

CR845 A peptide-based kappa opioid ago-nist

Acute postopera-tive pain

Started its first Phase I trial of an oral for-mulation (11/23)

Neuralstem Inc.

(Rockville, Md.)Stem cell therapy

Stem cell therapy used in the cervical region of the spine

Amyotrophic lateral sclerosis

Dosed its first patient in the trial (11/28)

NeuroDerm Ltd.

(Ness Ziona, Israel)ND0611 Administered sub-

cutaneously by a dermal patch

Parkinson’s disease

Met all primary and secondary endpoints in a Phase I/II trial (11/10)

Probiodrug AG

(Halle, Germany)PQ912 A glutaminyl

cyclase inhibitorAlzheimer’s disease

Phase I data demonstrated it was safe and well tolerated (11/15)

Selecta

Biosciences Inc. (Watertown, Mass.)

SEL-068 A nicotine vaccine Smoking cessa-tion and relapse prevention

Started a Phase I trial (11/22)



Company (Location)


DIABETES

Akebia

Therapeutics Inc.

(Cincinnati)

AKB-9778 A human protein tyrosine phospha-tase beta inhibitor

Diabetic macular edema and dia-betic retinopathy

Started a Phase I trial (11/22)

Concert

Pharmaceuticals

Inc. (Lexington, Mass.)

CTP-499 Controlled-release version; analogue of 1-([S]-5-hydroxyhexyl)-3,7-dimethylxanthine

Diabetic nephropathy

Phase I data showed it was well tolerated at single doses up to and including 1 ,800 mg (11/15)

Convergence

Pharmaceuticals

Ltd. (Cambridge, UK)

CNV2197944 A calcium channel blocker

Chronic pain Initiated a Phase I trial (11/22)

DARA

BioSciences Inc.

(Raleigh, N.C.)

DB959 A peroxisome pro-liferator activated receptor-delta/gamma agonist

Type II diabetes Phase Ib data showed a safety profile comparable to placebo when given as once-daily doses for seven days and was safe and well tolerated throughout the 40-fold dose range tested (11/2)

Isis

Pharmaceuticals

Inc. (Carlsbad, Calif.)

ISIS-GCGRRx and ISIS-GCCRRx

Antisense drugs Type II diabetes Started Phase I studies (11/3)

PhaseBio

Pharmaceuticals

Inc. (Malvern, Pa.)

Glymera A recombinant glu-cagon-like peptide-1 analogue

Hyperglycemia in Type II diabetes

Phase I/IIa data demonstrated statistically significant reductions in fasting glucose glycemic load following meal tolerance testing and in average daily glucose (11/16)

INFECTION

AVIR Green Hills

Biotechnology

AG (Vienna, Austria)

deltaFLU Intranasal fluvaccine

Influenza The vaccine was well tolerated and signif-icantly increased influenza-specific anti-bodies in a Phase I/II trial (11/8)

Inhibitex Inc.

(Atlanta)INHX-189 Monotherapy;

nucleotide poly-merase inhibitor

Hepatitis C virus Data showed INX-189 dosed at 100 mg once daily for seven days in combination with ribavirin resulted in a median HCV RNA reduction from baseline of -0.379 log10 (11/30)

Inovio

Pharmaceuticals

Inc. (Blue Bell, Pa.)

SynCon Avian influenza vaccine

Avian flu A single intradermal electroporation boost of its vaccine generated hemagglu-tination inhibition titers against six differ-ent unmatched strains of H5N1 and it generated a fourfold or greater rise in HAI titers in 50% of boosted subjects in a Phase I study (11/18)

Novacta

Biosystems Ltd.

(London)

NVB302 An oral treatment Hospital-acquired Clostridium difficile infections

Started dosing the first healthy volun-teers in a Phase I trial (11/3)



Company (Location)


MISCELLANEOUS

Aeolus

Pharmaceuticals

Inc. (Mission Viejo, Calif.)

AEOL 10150 A broad-spectrum catalytic antioxi-dant designed to neutralize reactive oxygen and nitro-gen species

Acute radiation syndrome

Pilot study results of AEOL 10150 and G-CSF drug Neupogen confirmed that it does not interefer with the positive effects of Neupogen on the hematopoiet-ic syndrome (11/2)

Akebia

Therapeutics Inc.

(Cincinnati)

AKB-6548 Oral hypoxia-inducible factor-prolyl hydroxylase inhibitor

Anemia associat-ed with chronic kidney disease

Phase Ib/IIa data showed it was well toler-ated and enhanced erythropoiesis (11/16)

AlloCure Inc.

(Burlington, Mass.)AC607 A bone marrow-

derived mesenchy-mal stem cell therapy

Acute kidney injury

Phase I data showed it was safe and well tolerated, and treated subjects experi-enced a lower incidence of AKI, reduced length of hospital stay and reduced hos-pital readmission rates compared to a cohort of historical controls (11/14)

Alnylam

Pharmaceuticals


ALN-TTR01 An RNAi therapeu-tic targeting trans-thyretin

TTR-mediated amyloidosis

Phase I data showed statistically signifi-cant reductions in serum TTR protein lev-els in ATTR patients (11/22)

Bellicum

Pharmaceuticals

Inc. (Houston)

CaspCIDe Technology used to eliminate donor T cells

Graft-vs.-host disease

Results of the first clinical study showed it worked quickly and effectively without compromising the function of the cell (11/4)

Chimerix Inc.

(Durham, N.C.)CMX001 A lipid-antiviral-

conjugate designed to deliver cidofovir-diphosphate

Kidney disease Phase I/II data showed it was generally safe and well tolerated when adminis-tered to renal transplant and hematopoi-etic stem cell transplant patients (11/14)

Geron Inc. (Menlo Park, Calif.)

hESC therapy Human embryonic stem cell therapy

Acute spinal injury

Company is stopping recruitment to its Phase I trial due to economic conditions (11/16)

Pluristem

Therapeutics Inc.

(Haifa, Israel)

PLX-PAD Cells derived from the firm’s PLX (PLacental eXpand-ed) platform

Critical limb ischemia

Phase I data showed that it met all the endpoints, demonstrating a safe immuno-logic profile at all dosage levels and showing potential efficacy (11/4)

Provesica Ltd.

(Cambridge, UK)XEN-D0501 An antagonist of

TRPV1Overactive bladder

Phase I data showed it was safe and well tolerated (11/28)

Notes:

Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.

# The date indicated refers to the BioWorld Today issue in which the news item can be found.



Phase II Clinical Trials Update: November 2011Company (Location)


AUTOIMMUNE

Adeona

Pharmaceuticals

Inc. (Ann Arbor, Mich.)

Trimesta Oral estriol candi-date

Multiple sclerosis

It will be tested in a Phase II trial evaluating its potential effect on cognitive dysfunc-tion in female MS patients (11/14)

Avanir

Pharmaceuticals

Inc. (Aliso Viejo, Calif.)

AVP-923 An NMDA antago-nist and sigma-1 agonist

Neuropathic pain in multi-ple sclerosis

Enrolled the first patient in a Phase II trial expected to enroll 400 patients worldwide (11/4)

Galapagos NV

(Mechelen, Belgium)

GLPG0634 JAK1 inhibitor Rheumatoid arthritis

Achieved the primary endpoint of signifi-cant improvement in the signs and symp-toms of RA in a Phase IIa trial (11/28)

KAI

Pharmaceuticals

Inc. (South San Francisco)

KAI-4169 Long-acting intrave-nous calcium sens-ing receptor agonist

Secondary hyperparathy-roidism

Phase II data showed it was effective, with 33% and 49% reductions in parathyroid hormone (PTH) from baseline in the 5-mg and 10-mg dose groups, respectively (11/14)

Opexa

Therapeutics

Inc. (The Woodlands, Texas)

Tovaxin A T-cell therapy Relapsing-remitting mul-tiple sclerosis

Phase IIb data showed it was well tolerated, with no serious adverse events, and dem-onstrated encouraging results in the reduc-tion of the annualized relapse rate and improvement in disease progression (11/15)

SuppreMol

GmbH

(Martinsried, Germany)

SM101 A soluble version of the Fc gamma recep-tor IIb

Lupus Started dosing in a Phase IIa trial (11/29)

CANCER

Biothera Inc.

(Eagan, Minn.)Imprime PGG An immunomodula-

tor drugNon-small-cell lung cancer

Completed enrollment of 90 patients in its Phase II trial in combination with Erbitux (11/18)

Celldex

Therapeutics

Inc. (Needham, Mass.)

CDX-110 Rindopepimut EGFRvIII-positive glio-blastoma

Phase II data showed final overall survival of 24.6 months from diagnosis, compared to 15.2 months for a historic control (11/22)

Exelixis Inc. (South San Francisco)

Cabozantinib MET and VEGFR2 inhibitor

Hormone receptor-posi-tive breast can-cer with bone metastases

Initiated a Phase II trial (11/22)

Galena

Biopharma Inc.

(Lake Oswego, Ore.)

NeuVax Vaccine Breast cancer Phase II data showed that it may provide a meaningful clinical benefit in patients with less aggressive forms of the disease (11/7)

Jennerex Inc.

(San Francisco)JX-594 Oncolytic virus Advanced liver

cancerStarted the Phase IIb trial in patients who failed prior therapy with Nexavar (11/7)

KAEL-GemVax

Co. Ltd. (Seoul, South Korea)

GV1001 Telomerase peptide Non-small-cell lung cancer

Interim Phase II data demonstrated an 80% immune response rate (11/16)



Company (Location)


Ligand

Pharmaceuticals

Inc. (San Diego)

Melphalan Captisol-enabled propylene glycol-free melphalan

Cancer Phase II data showed that it met the requirements for establishing bioequiva-lence to Alkeran (11/3)

MediGene AG

(Martinsried, Germany)

EndoTAG-1 A composition of paclitaxel combined with neutral and positive lipids

Hormone-receptor-positive, HER2-negative breast cancer

Phase II trial started (11/9)

Peregrine

Pharmaceuticals

Inc. (Tustin, Calif.)

Bavituximab A phosphatidylser-ine-targeting mono-clonal antibody

Locally advanced or metastatic breast cancer

Company reported a 23.2-month median overall survival from a single-arm Phase II trial of bavituximab in combination with carboplatin and paclitaxel, compared with a published study showing median OS of 16 months with carboplatin and paclitaxel alone (11/23)

YM BioSciences

Inc. (Mississauga, Ont.)

Nimotuzumab Anti-EGFR antibody Recurrent dif-fuse intrinsic pontine glioma

Phase II data showed it was well tolerated when administered to pediatric patients (11/1)

CARDIOVASCULAR

Aastrom

Biosciences Inc.

(Ann Arbor, Mich.)

Ixmyelocel-T A patient-specific multicellular therapy

Critical limb ischemia

Phase II data showed patients in the treat-ment arm had a 62% reduction in risk rela-tive to placebo in the primary endpoint of time to first occurrence of treatment failure (11/15)

Mesoblast Ltd.

(Melbourne, Australia)

Revascor Stem cell treatment Congestive heart failure

Phase II data showed it did not provoke any adverse immune responses and there was a 78% reduction in the rate of serious cardi-ac events (11/16)

Resverlogix

Corp. (Calgary, Alberta)

RVX-208 A small molecule designed to stimu-late endogenous ApoA-I production

Coronary artery disease

Started dosing in its Phase IIb study (11/3); the study is fully enrolled with 176 subjects (11/29)

Sanofi SA (Paris) and Regeneron

Pharmaceuticals

Inc. (Tarrytown, N.Y.)

REGN727/SAR236553

A fully human anti-body targeting pro-protein convertase subtilisin/kexin type 9

Elevated low-density lipo-protein cholesterol

Phase II data showed that after 12 weeks of treatment patients achieved mean LDL-C reductions ranging from about 30% to more than 65%, depending on the dosing regimen; the control arm had a mean reduction of 10% (11/11)

Theravance Inc.

(South San Francisco)

TD-4208 Inhaled long-acting muscarinic antago-nist

Chronic obstructive pulmonary disease

Phase IIa data showed it met the primary endpoint, with both study doses demon-strating a statistically significant mean change from baseline in peak forced expi-ratory volume in one second compared to placebo (11/16)

Trophos SA

(Marseille, France)TRO40303 A mitochondria pore

modulatorCardiac isch-emia-reperfu-sion injury in acute myocar-dial infarction

Treated the first patient in a Phase II study (11/1)



Company (Location)


Verona Pharma

plc (London)RPL554 A mixed phosphodi-

esterase 3/4 inhibi-tor

Chronic obstructive pulmonary disease

Demonstrated bronchodilator effects in a Phase IIa trial (11/11)


BioDelivery

Sciences

International

Inc. (Raleigh, N.C.)

BEMA Bupre-norphine/Nalaxone

Formulation of buprenorphine and naloxone using BDSI’s BioErodible MucoAdhesive drug delivery technology

Opioid dependence

Started a confirmatory study, which will select the final doses to be used in the piv-otal bioequivalence study compared to Suboxone (11/11)

Clinuvel

Pharmaceuticals

Ltd. (Melbourne, Australia)

Scenesse Afamelanotide; pho-toprotective drug

Erythropoietic protoporphyria

Phase II data showed it appeared to be effective in preventing severe pain result-ing from sun exposure (11/4)

Cytokinetics Inc.


CK-2017357 A fast skeletal mus-cle troponin activator


Started a second cohort of an ongoing Phase II trial (11/2); opened enrollment in its third Phase II trial (11/30)

Knopp

Biosciences LLC (Pittsburgh)

Dexpram-ipexole

A small-molecule modulator of mito-chondrial bioener-getics


Phase II data showed the highest dose of the drug reduced mortality by 68% in the second stage of the trial (11/22)

NeurogesX Inc.

(San Mateo, Calif.)NGX-1998 A topical liquid for-

mulation of high-concentration capsaicin

Postherpetic neuralgia

Phase II top-line data showed that the trial met its primary endpoint of a percentage change from baseline vs. placebo (11/9)

Rexahn

Pharmaceuticals

Inc. (Rockville, Md.)

Serdaxin A neuroprotective and antidepressant candidate

Major depres-sive disorder

Phase IIb data showed it did not demon-strate efficacy compared to placebo over an eight-week treatment period (11/7)

Supernus

Pharmaceuticals

Inc. (Rockville, Md.)

SPN-810 Molindone hydro-chloride

Impulsive aggression associated with attention deficit hyper-activity disorder

Started a Phase IIb trial (11/29)

INFECTION

Alnylam

Pharmaceuticals


ALN-RSV01 RNAi therapy Respiratory syncytial virus

Completed enrollment in a Phase IIb trial (11/30)

Inviragen Inc.

(Fort Collins, Colo.)DENVax A dengue vaccine

based on an attenu-ated DEN-2 virus

Dengue fever Started a Phase II trial (11/29)

Medivir AB

(Huddinge, Sweden)

TMC435 An oral HCV prote-ase inhibitor

Chronic hepa-titis C virus

Phase IIb data showed that TMC435 once daily in addition to pegylated interferon and ribavirin produced superior rates of sustained virologic response compared to standard of care (11/3)



Company (Location)


SEEK (London) FLU-v Vaccine Influenza The vaccine was well tolerated and effec-tive against all influenza strains in a Phase II challenge trial (11/8)

Sinovac Biotech

Ltd. (Beijing)Vaccine Enterovirus 71 vac-

cineHand, foot and mouth disease

The vaccine had good immunogencity and a favorable safety profile with no adverse events in a Phase II trial (11/10)

MISCELLANEOUS

Alexion

Pharmaceuticals

Inc. (Cheshire, Conn.)

Soliris Eculizumab Atypical hemo-lytic uremic syndrome

Pivotal Phase II data demonstrated that ongoing treatment sustained the suppres-sion of complement-mediated thrombotic microangiopathy, maintained or further improved longer-term renal function and enhanced quality of life (11/11)

Ampio

Pharmaceuticals

Inc. (Greenwood Village, Colo.)

Ampion A low molecular fraction of commer-cial albumin

Nasal inflam-mation

A double-blind study in 20 patients showed it was safe and effective for reduc-ing nasal inflammation (11/11)

FibroGen Inc. (San Francisco)

FG-4592 A hypoxia-inducible factor prolyl hydrox-ylase inhibitor

Chronic kidney disease

Phase IIb data showed that 96% of patients had an increase in hemoglobin (Hb) of at least 1 g/dL, and 93% had an Hb response; treatment was well tolerated (11/16)

Kythera

Biopharma-

ceuticals Inc. (Los Angeles)

ATX-101 Formulation of deoxycholate, an endogenous com-pound that pro-motes the natural breakdown of dietary fat

Submental fat Data from a long-term follow-up Phase IIa trial showed more than 90% of initial responders sustained or improved their response (11/8)

ProFibrix BV

(Leiden, the Netherlands)

Fibrocaps A dry powder topi-cal fibrin sealant developed from a mixture of fibrino-gen and thrombin

Liver resection surgery

Phase II data showed a 50% reduction in mean time to hemostasis, the primary end-point, compared to active control (11/16)

RegeneRx

Biopharma-

ceuticals Inc.

(Rockville, Md.)

RGN-259 Sterile, preservative-free eye drop

Central corne-al fluorescein staining

Phase II data showed it met statistical sig-nificance after a challenge-controlled adverse environment in the reduction from baseline compared to placebo (11/7)

Repros

Therapeutics

Inc. (The Woodlands, Texas)

Androxal Oral therapy that stimulates the pitu-itary gland

Low testosterone

Phase II data showed that 25 mg of Androxal showed statistically equivalent efficacy to topical Androgel (11/9)

Ritter

Pharmaceuticals

Inc. (Los Angeles)

RP-G28 A drug designed to stimulate colonic growth

Lactose intolerance

Last patient completed treatment in a Phase II study (11/18)

Unigene

Laboratories Inc.

(Boonton, N.J.)

Oral PTH Oral parathyroid hormone analogue

Osteoporosis Phase II data showed it achieved the prima-ry endpoint of increasing bone mineral density at the lumbar spine (11/10)

Notes:





Phase III Clinical Trials Update: November 2011Company (Location)


AUTOIMMUNE

Genzyme Corp.

(Cambridge, Mass.)Lemtrada Alemtuzumab Relapsing-

remitting multiple sclerosis

Phase III data showed it met both of its co-primary endpoints (11/15)

NPS

Pharmaceuticals

Inc. (Bedminster, N.J.)

Natpara NPSP558; a bioengi-neered replica of human parathyroid hormone

Hypopara-thyroidism

Phase III data showed that 53% of Natpara-treated patients achieved the pri-mary endpoint vs. 2% of placebo-treated patients (11/8)

Nuron Biotech

Inc. (Exton, Pa.)NU100 A recombinant

human interferon beta-1b

Relapsing-remitting multi-ple sclerosis

Started a pivotal, Phase III trial (11/2)

CANCER

Amgen Inc.

(Thousand Oaks, Calif.)

Xgeva Denosumab Bone metasta-ses of prostate cancer

Phase III data showed that Xgeva signifi-cantly prolonged bone metastasis-free survival by 4.2 months, delayed bone metastasis by 3.7 months and reduced symptoms of bone metastasis by 33% (11/17)

Celgene

International

Sarl (Boudry, Switzerland)

Revlimid Lenalidomide Castrate-resistant prostate cancer

Company is terminating the pivotal Phase III MAINSAIL trial because the combina-tion of docetaxel and prednisone with Revlimid did not show a statistically sig-nificant improvement in overall survival compared to docetaxel and prednisone plus placebo (11/28)

Exelixis Inc. (South San Francisco)

Cabozantinib A dual inhibitor of MET and VEGFR

End-stage cas-trate resistant prostate cancer

Company was unable to secure an SPA and plans to start a Phase III trial by the end of the year with pain as the primary efficacy endpoint; a second Phase III trial to evaluate overall survival time will launch in 2012 (11/2)

Medivation Inc.

(San Francisco)MDV3100 An oral androgen

receptor antagonistProstate cancer A independent data monitoring commit-

tee found Phase III interim data to be posi-tive and recommended the trial be stopped so the drug could be offered to men receiving placebo (11/4)

Novocure Inc.

(Anaheim, Calif.)TTF therapy Tumor treating

fields therapyRecurrent glio-blastoma multiforme

Phase III data showed overall survival was 9% vs. 7% and 8% vs. 1% for the TTF group compared to the standard of care at two and three years of follow-up, respectively (11/22)

Talon

Therapeutics Inc.

(San Mateo, Calif.)

Marqibo Vincristine sulfate liposomes injection

Newly diagnosed aggressive non-Hodgkin’s lymphoma

Enrolled the first patient in the Phase III study of Marqibo (11/30)



Company (Location)


CARDIOVASCULAR

Almirall SA

(Barcelona, Spain) and Forest

Laboratories Inc.

(New York)

Aclidinium bromide and formoterol fumarate

Fixed-dose combi-nation via the Genuari inhaler

Chronic obstructive pul-monary disease

Began a Phase III trial (11/17)

Gilead Sciences

Inc. (Foster City, Calif.)

Ranexa Ranolazine Chronic angina Started a Phase III trial (11/8)

Inspiration

Biopharma-

ceuticals Inc. (Laguna Niguel, Calif.)

OBI-1 An intravenous recombinant por-cine Factor VIII prod-uct

Congenital hemophilia A

Started patient enrollment in the seconf of two pivotal studies (11/28)

Omthera

Pharmaceuticals


Epanova Triglyceride-lowering therapy

High triglycerides

Completed enrollment in a pivotal, Phase III trial (11/30)

Santarus Inc. (San Diego)

Rhucin Recombinant human C1 inhibitor

Hereditary angioedema

A retrospective analysis showed that median time to primary endpoint of onset of symptom relief was 60 minutes and median time to minimal symptoms was 240 minutes (11/7)


Alexza

Pharmaceuticals

Inc. (Mountain View, Calif.)

Adasuve Staccato loxapine Schizophrenia or bipolar I disorder

Phase III data showed a 40% or greater decrease from baseline in the total PANSS-Excited Component score; another analy-sis showed a statistically significant improvement over placebo using the Clinical Global Impression-Improvement scale (11/9)

Alkermes plc

(Dublin)Vivitrol Naltrexone Opioid

dependenceData showed sustained efficacy in those receiving Vivitrol in combination with psychosocial treatment for 18 months (11/10)

Nabi Biopharma-

ceuticals Inc.

(Rockville, Md.)

NicVAX Smoking cessation vaccine

Smoking cessation

A second Phase III trial failed to meet its primary endpoint of abstinence from cig-arettes; the vaccine was well tolerated (11/8)

Targacept Inc.

(Winston Salem, N.C.)

TC-5214 Nicotinic channel modulator to nor-malize cholinergic tone

Major depres-sive disorder

Failed to meet its primary endpoint of change on the Montgomery-Asberg Depression Rating Scale following eight weeks of treatment in a Phase III trial (11/9)

Vivus Inc.

(Mountain View, Calif.)

Qnexa Phentermine/topira-mate

Obesity Results from the 56-week EQUIP study in 1 ,267 patients showed an average weight loss of 14.4% of initial body weight, plus improvements in blood pressure, glucose, triglycerides and cholesterol (11/7)



Company (Location)


DIABETES

GlaxoSmithKline

plc (London)Albiglutide A once-weekly glu-

cagon-like peptide-1 agonist

Type II diabetes Top-line data from the first of eight Phase III studies failed to meet the primary end-point of noninferiority to Victoza, though it did demonstrate a statistically signifi-cant reduction in HbA1c from baseline (11/17)

NephroGenex

Inc. (Research Triangle Park, N.C.)

Pyridorin Pyridoxamine dihy-drochloride

Type II diabetes FDA and company reached an agreement on the design of a Phase III program (11/9)

INFECTION

Anacor

Pharmaceuticals

Inc. (Palo Alto, Calif.)

AN2690 Tavaborole; a topical antifungal product

Onychomycosis Completed enrollment of a targeted 600 patients in the first of two Phase III trials (11/11)

Emergent

BioSolutions Inc.

(Rockville, Md.)

Biothrax Anthrax vaccine adsorbed

Anthrax Started a pivotal immunmogenicity and safety study of a three-dose vaccination schedule (11/18)

Santarus Inc. (San Diego)

Budesonide MMX

9 mg taken once daily

Mild or moder-ate active ulcer-ative colitis

Analysis of pooled data from its two Phase III studies indicated it was statisti-cally superior to placebo (11/2)

Pharmasset Inc.

(Princeton, N.J.)PSI-7977 Nucleotide analogue Hepatitis C virus Started a pivotal Phase III program evalu-

ating an all-oral, interferon-free regimen of PSI-7977 and ribavirin (11/2)

MISCELLANEOUS

Affymax Inc. (Palo Alto, Calif.) and Takeda

Pharmaceutical

Co. Ltd. (Osaka, Japan)

Peginesatide A synthetic, pegylat-ed peptidic com-pound designed to bind to and stimu-late the erythropoie-tin receptor

Chronic kidney disease

Phase III data showed that once-monthly peginesatide maintained Hb levels in CKD patients on dialysis with anemia (11/14)

AMAG

Pharmaceuticals

Inc. (Lexington, Mass.)

Feraheme Ferumoxytol Iron deficiency anemia and chronic kidney disease

Data from a head-to-head study compar-ing Feraheme to iron sucrose demonstrat-ed overall that ferumoxytol 1 .02 g delivered as two injections had a favor-able safety profile and comparable effica-cy to 1 g of iron sucrose (11/15)

Bionovo Inc.

(Emeryville, Calif.)Menerba Estrogen receptor

beta selective drugMenopausal hot flashes

The first patient was randomized and will begin dosing in a Phase III pivotal trial (11/17)

BioSante

Pharmaceuticals

Inc. (Lincolnshire, Ill.)

LibiGel Testosterone gel Female sexual dysfunction

Additional data showed the drug increas-es free testosterone in the serum of post-menopausal women to the normal range for premenopausal women (11/17)

Emisphere

Technologies

Inc. (Cedar Knolls, N.J.)

SMC021 Oral calcitonin can-didate

Osteopororis It failed to reduce the occurrence of new vertebral fractures in a three-year osteo-porosis study (11/15)



Company (Location)


Eyetech Inc. (Palm Beach Gardens, Fla.)

Macugen Pegaptanib sodium Neovascular age-related macular degeneration

Post-hoc analysis of the Phase III study showed no apparent association between treatment with Macugen and the occur-rence of sustained elevated intraocular pressure (11/8)

NPS

Pharmaceuticals


Gattex Teduglutide Short bowel syndrome

Phase III data showed that nine patients discontinued STEPS 2 due to adverse events, and while gastrointestinal side effects were the most common culprit, three patients developed cancer, with two cases proving fatal (11/1)

Trimel

Pharmaceuticals

Corp. (Toronto)

CompleoTRT A bioadhesive intra-nasal testosterone gel product

Hypogonadism Dosed the first patient in a pivotal Phase III study (11/1)

Vertex

Pharmaceuticals


Kalydeco Ivacaftor, VX0-770 Cystic fibrosis Pivotal data showed that children who received drug experienced rapid and sus-tained improvements in lung function and other key measures of disease, including weight gain and a reduction in sweat chloride throughout the 48-week trial compared to placebo (11/4)

Vivus Inc.


Avanafil A phosphodiester-ase-5 inhibitor

Erectile dysfunction

A new analysis from a Phase III study found it was effective in as early as 15 minutes after dosing in 80% of all study-wide sexual attempts (11/15)

Notes:



SPA = Special Protocol Assessment.

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American Association for the Study of Liver Diseases(The 62nd annual meeting was held Nov. 4-8 in San Francisco.)

Company (Location)


Boehringer

Ingelheim GmbH

(Ingelheim, Germany)

BI201335 Next-generation protease inhibitor

Hepatitis C virus Phase IIb data of BI201335 in combination with pegylated protease inhibitor, in com-bination with pegylated interferon and ribavirin, showed its potential to shorten treatment duration to 12 weeks and to improve the likelihood of viral cure com-pared to pegylated interferon and ribavi-rin alone (11/9)

Bristol-Myers

Squibb Co. (New York)

BMS-790052 Dual, all-oral direct-acting antiviral regi-men of NS5A replication complex inhibitor daclatasvir and NS3 protease inhibitor asunaprevir

Hepatitis C virus Phase II data show they achieved unde-tectable viral load at 12 weeks post-treat-ment in 90% of genotype 1b HCV patients who had previously not responded to peginterferon alfa and ribavirin (11/9)

ChemoCentryx

Inc. (Mountain View, Calif.)

CCX354 Orally-active small molecule that spe-cifically targets and inhibits the chemo-kine receptor known as CCR1

Rheumatoid arthritis

Phase II data showed it was safe and well tolerated and had clinical and biological activity at 200 mg once daily (11/10)

Cytheris SA

(Paris)CYT107 A recombinant

human interleukin-7Hepatitis C virus Interim Phase I/IIa data showed treatment

with CYT107 plus interferon and ribavirin resulted in a broad immune response associated with HCV viral clearance (11/9)

Gilead Sciences


Viread Tenofovir disoproxil fumarate

Chronic hepati-tis B virus

Phase III data showed it maintained long-term viral suppression of HBV and is asso-ciated with a reduction in liver fibrosis and a reversal of cirrhosis (11/8)

Jennerex Inc.

(San Francisco)JX-594 Engineered oncolyt-

ic virusAdvanced liver cancer

Phase II data showed a statistically signifi-cant benefit in overall survival for the high-dose group vs. the low-dose group (11/8)

Merck & Co. Inc.

(Whitehouse Station, N.J.)

Victrelis Protease inhibitor; boceprevir

Chronic hepatitis C virus

An interim analysis from the open-label study testing Victelis in combination with peginterferon alfa and ribavirin showed that 38% achieved a sustained virologic response (11/8)

NasVax Ltd. (Nes Ziona, Israel)

Monoclonal antibodies

Oral anti-CD3 mono-clonal antibodies

Nonalcoholic steatohepatitis

Phase IIa data showed that it promoted regulatory T cells, decreased liver enzymes and alleviated insulin resistance (11/8)

Santaris Pharma

A/S (Hoersholm, Denmark)

Miravirsen MicroRNA-targeted drug

Hepatitis C virus Phase IIa data showed that given as a four-week monotherapy treatment it pro-vided robust dose-dependent antiviral activity with a mean reduction of 2 to 3 logs from baseline (11/8)



Company (Location)


Scynexis Inc.

(Research Triangle Park, N.C.)

SCY-635 An oral cyclophilin inhibitor

Hepatitis C virus Data demonstrated it reactivated the body’s natural defense mechanism, mak-ing it capable of inhibiting replication of the virus (11/8)

Tibotec

Pharmaceuticals

(unit of Johnson & Johnson; New Brunswick, N.J.) and Medivir AB

(Huddinge, Sweden)

TMC435 Once-daily therapy; a HS3/4A protease inhibitor

Chronic geno-type-1 hepatitis C virus

Phase IIb data showed when administered in combination with peginterferon alpha-2a and ribavirin, the drug demonstrated a significantly higher sustained virologic response rate compared to placebo plus PR (11/8)

Transgene SA

(Strasbourg, France)

TG-4040 Vaccine Hepatitis C virus Phase II data showed when administered in combination with pegylated interferon alpha plus ribavirin, it was twice as effec-tive in eliciting a complete early virologic response as standard therapy alone (11/8)

Vertex

Pharmaceuticals


VX-222 Polyermase inhibitor Genotype-1 chronic hepati-tis C virus

Phase II data of VX-222 with Incivek, pegylated interferon and ribavirin showed that 90% of patients had unde-tectable HCV levels in the blood 12 weeks after the end of treatment (11/8)

Notes:



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American College of Rheumatology and the Association of Rheumatology Health Professionals

(The ACR/ARHP meeting was held Nov. 5-9 in Chicago.)Company (Location)


4SC AG (Planegg-Martinsried, Germany)

Vidofludimus Designed to target IL-17 and dihydro-orotate dehydroge-nase


Phase IIb data demonstrated substantial anti-inflammatory activity of vidofludi-mus, with an ACR20 improvement of 50.8% vs. 44.8% in the control group (11/8)

Abbott (Abbott Park, Ill.)

Humira Anti-TNF drug; adali-mumab

Active nonra-diographic axial spondylo-arthritis

Phase III data showed that more than twice as many patients receiving Humira compared to those receiving placebo achieved the primary endpoint of 40% improvement (11/8)

Abbott (Abbott Park, Ill.)

Humira Anti-TNF drug; adali-mumab

Ankylosing spondylitis

Five-year results from an open-label extension show it resulted in a mean change from baseline of -0.6 in Bath Ankylosing Spondylitis Metrology Index (11/9)

Amgen Inc.


Prolia Denosumab; a RANK ligand inhibitor

Postmenopausal osteoporosis

Phase III data showed that up to six years of continued Prolia treatment through both trials produced cumulative bone mineral density gains of 15.2% at the lum-ber spine and 7.5% at the total hip, com-pared with baseline (11/8)

Ardea

Biosciences Inc.

(San Diego)

Lesinurad URAT1 inhibitor Gout Phase IIb data showed a combination of lesinurad with allopurinol had consistent, sustained reductions in serum urate lev-els, with 90% of combination therapy patients at week 44 reaching the medical-ly recommended target of sUA less than 6 mg/dL (11/8)

BioCryst

Pharmaceuticals

Inc. (Research Triangle Park, N.C.)

BCX4208 Next generation purine nucleoside phosphorylase inhibitor

Gout Phase IIb data show that patients with gout who have failed to reach serum uric acid level of <6 mg/dL on allopurinol alone met the primary endpoint (11/9)

Celgene

International


Apremilast Oral immunotherapy

Ankylosing spondylitits

Phase II data show it was associated with a trend to greater mean improvement from baseline compared with placebo in all parameters (11/9)

Centocor Ortho

Biotech Inc.

(Horsham, Pa.)

CNTO 136 Sirukumab; a subcu-taneous human anti-body directed against interleukin 6


Phase II data showed treatment signifi-cantly improved rheumatoid arthritis signs and symptoms (11/8)

Genentech Inc.


Actemra Tocilizumab Rheumatoid arthritis

Phase III data showed Actemra as a mono-therapy resulted in 34.8% of patients achieving a DAS28 score at 24 weeks, comparable to the 40.4% rate in patients taking Actemra plus methotrexate (11/8)



Company (Location)


Neovacs SA (Paris)

Kinoid Immunotherapy Lupus Data from a 28-patient study show that Kinoid reduced the gene dysregulation associated with the overexpression of INF-alpha (11/9)

Novartis AG (East Hanover, N.J.)

ACZ885 Canakinumab Active systemic juvenile idio-pathic arthritis

Pivotal Phase III data showed that 45% of children were able to substantially reduce their use of oral corticosteroids within 28 weeks of beginning treatment (11/8)

Savient

Pharmaceuticals

Inc. (East Brunswick, N.J.)

Krystexxa Pegloticase Gout A post-hoc analysis of pivotal Phase III data and an open-label extension con-firmed that risk of infusion reactions and anaphylaxis could be mitigated by the monitoring of serum uric acid prior to each infusion and discont inuing Krystexxa in patients whose sUA levels rise above 6mg/dL (11/8)

UCB SA (Brussels, Belgium)

Cimzia Certolizumab pegol; pegylated anti-TNF drug


Findings from a post-hoc analysis sug-gest that disease activity response rates of Cimzia plus methotrexate as early as week 12 helped predict the effect on struc-tural joint damage in patients with mod-erate to severe disease at one year (11/8)

Vertex

Pharmaceuticals


VX-509 JAK3 inhibitor Rheumatoid arthritis

Phase IIa data showed it significantly improved rheumatoid arthritis as mea-sured by the proportion of people who achieved at least a 20% improvement in the signs and symptoms (11/8)

Notes:





American College of Allergy, Asthma and Immunology(The annual meeting was held Nov. 3-8 in Boston.)

Company (Location)


Baxter

International Inc.

(Deerfield, Ill.)

HyQ A combination immunoglobulin product

Bacterial infections

Phase III data showed patients with pri-mary immunodeficiencies had a reduced rate of serious bacterial infections when treated with HyQ (11/8)

CSL Behring LLC

(King of Prussia, Pa.)

Berinert C1-esterase inhibitor Hereditary angioedema

Data showed median time of 0.46 hours to the onset of symptom relief and 15.5 hours to resolution (11/8)

Dyax Corp.

(Cambridge, Mass.)Kalbitor Ecallantide; A plas-

ma kallikrein inhibi-tor

Hereditary angioedema

Clinical data showed it remained effective across multiple episodes of acute HAE attacks with no apparent increase in adverse events (11/8)

Sunovion

Pharmaceuticals

Inc. (Marlborough, Mass.)

Ciclesonide Nasal aerosol; a cor-ticosteroid formu-lated with a hydrofluoroalkane propellant

Perennial allergic rhinitis

Phase III data showed statistically signifi-cant improvements in reflective and instantaneous nasal symptoms after the first six weeks compared to placebo, as well as tolerability over 26 weeks (11/8)

Notes:



American Heart Association(The scientific sessions were held Nov. 12-16 in Orlando.)

Company (Location)


Amarin Corp. plc

(Dublin, Ireland)AMR101 A prescription-grade

omega-3 fatty acidHigh triglycerides

Phase III data showed patients who were on statin therapy experienced a signifi-cant reduction in triglyceride levels and other lipid parameters, as well as inflam-matory biomarkers, when treated with AMR101 (11/17)

Genzyme Corp.

(Cambridge, Mass.)MIPO Mipomersen Low-density

lipoprotein cholesterol

Positive results from a randomized, dou-ble-blind, placebo-controlled Phase II trial showed that weekly treatment reduced LDL-C by 47% compared to 2% with place-bo (11/17)

Regado

Biosciences Inc.

(Basking Ridge, N.J.)

REG1 Anticoagulant sys-tem; reversible anti-thrombotic agent

Cardiovascular disease

Phase IIb data suggested it provided near-ly complete Factor IXa inhibition with a dose of 1 mg/kg of pegnivacogin and, when followed by 75% and 100% reversal doses of anivamersen, resulted in numeri-cally lower rates of ischemic events com-pared with heparin (11/16)

Notes:





American Association for Cancer Research, National Cancer Institute and European Organization for

Research and Treatment of Cancer(The AACR-NCI-EORTC international conference was held Nov. 12-16 in San Francisco.)

Company (Location)


ACT Biotech Inc.

(San Francisco)

Telatinib An oral VEGF inhibi-tor

Gastric cancer Phase II data showed patients treated with telatinib and Xeloda and cisplatin who responded with a large decrease of a specific angiogenesis biomarker were found to have a lower risk of disease pro-gression and death compared with patients that had smaller decreases (11/15)

Cerulean Pharma


CRLX101 A camptothecin nanoparticle

Non-small-cell lung cancer

Phase I/IIa data showed that median pro-gression-free survival time was 4.4 months for 21 of the 38 patients (11/15)

Enzon

Pharmaceuticals

Inc. (Piscataway, N.J.)

EZN-3042 An LNA oligonuclo-etide targeting sur-vivn mRNA

Advanced solid malignancies

Phase I data demonstrated good tolerabil-ity and antitumor activity (11/15)

Exelixis Inc.


Cabozantinib An inhibitor of MET, RET and VEGFR2

Metastatic cas-tration-resistant prostate cancer

Phase I data suggested that a daily start-ing dose of 40 mg resulted in high rates of bone scan response assessed by com-puter-aided detection in men with CRPC and bone metastases; interim data on pain relief and related reduction in nar-cotic analgesic use showed 56% of patients decreased their dose by at least 30% (11/15)

Geron Corp. (Menlo Park, Calif.)

GRN1005 Lead LRP-directed peptide-paclitaxel conjugate

Brain metasta-ses from solid tumors and malignant glioma

Phase I data demonstrated single-agent clinical activity in both indications (11/16)

MethylGene Inc.

(Montreal)MGCD265 Oral Met/VEGF

receptor tyrosine kinase inhibitor

Advanced solid cancers

Phase I data showed the compound was tolerable, with no treatment-related seri-ous adverse events, and showed early signs of clinical benefit (11/15)

Myrexis Inc. (Salt Lake City)

MPC-3100 Heat-shock protein inhibitor

Cancer Phase I data showed that it was generally safe and well tolerated (11/15)

Oncolytics

Biotech Inc.

(Calgary)

Reolysin Intravenous therapy Advanced pan-creatic cancer

Interim Phase II data of Reolysin in combi-nation with gemcitabine showed that all but one of 12 patients reported symptom-atic improvement, including seven with stable disease for 12 weeks or longer, for a clinical benefit rate of 58% (11/15)



Company (Location)


Oncothyreon Inc.

(Seattle)PX-866 Irreversible, pan-iso-

form phosphati-dylinositol-3-kinase inhibitor

Advanced cancer

Two Phase I/II trials showed no dose-lim-iting toxicities with the combination of docetaxel and PX-866 generally well tol-erated; best response was stable disease in 21 of 28 patients and progressive dis-ease in seven patients, for a disease con-trol rate of 75% (11/15)

Pieris AG (Freising-Weihenstephan, Germany)

PRS-050 Anticalin; an anti-VEGF targeted pro-tein therapeutic

Advanced solid tumors

Phase I data showed it was well tolerated, with no maximum-tolerated dose reached, and it demonstrated biological activity, with no immunogenicity (11/16)

Syndax

Pharmaceuticals

Inc. (San Francisco and Waltham, Mass.)

Exemestane Hormone therapy Postmenopausal estrogen-recep-tor positive breast cancer

A pharmacodynamic analysis in a subset of patients demonstrated an association of the pharmacodynamic marker lysine hyperacetylation with clinical outcome (11/15)

TetraLogic

Pharmaceuticals

(Malvern, Pa.)

TL32711 Smac mimetic candi-date

Solid tumors and lymphomas

Phase I data suggested it caused potent and sustained suppression of inhibitor of apoptosis protein in patient peripheral blood mononuclear cells and tumor biop-sies over seven days at tolerable dose lev-els (11/15)

Ziopharm

Oncology Inc.

(New York)

Zymafox Palifosfamide Non-small-cell lung cancer

Phase Ib data showed that Zymafos in combination with etoposide and carbopl-atin can be given at a maximum-tolerated dose of 130 mg/m2, with a dose-limiting toxicity of neutropenic fever (11/15)

Notes:



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Non-U.S. Clinical Trials & Regulatory Actions: Nov. 2011Company (Location)


AUTOIMMUNE

DuoCort Pharma

AB (Helsingborg, Sweden)

Plenadren Hydrocortisone, modified-release tablet

Adrenal insufficiency

European Commission granted approval (11/8)

Pfizer Inc. (New York)

Tofacitinib An oral JAK inhibitor Moderate to severe active rheumatoid arthritis

EMA validated and began review of tofaci-tinib (11/22)

CANCER

Amgen Inc.


Vectibix Panitumumab; EGFR-targeted can-cer drug

Wild-type KRAS metastatic colorectal cancer

Gained an expansion to the European label, allowing it to be used as a first-line treatment in combination with FOLFOX and as a second-line treatment in combi-nation with FOLFIRI (11/16)

BioAlliance

Pharma SA (Paris)Lauriad Clonidine Lauriad To prevent

radiotherapy-incuded oral mucositits in head and neck cancer patients

European Commission granted orphan designation (11/3)

Celgene

International


Revlimid Lenalidomide Myelodysplastic syndromes

Swiss regulators approved it for use in patients with transfusion-dependent ane-mia due to myelodysplastic syndromes associated with a deletion 5q cytogenic abnormality with or without additional cytogenic abnormalities (11/22)

Celldex

Therapeutics

Inc. (Needham, Mass.)

CDX-110 Rindopepimut Glioblastoma EMA granted orphan drug designation (11/1)

CARDIOVASCULAR

Amsterdam

Molecular

Therapeutics NV

(Amsterdam, the Netherlands)

Gene therapy Consists of an ade-no-associated viral vector containing the human factor IX gene

Hemophilia B Received orphan drug designation from the EMA (11/17)

Dyax Corp.

(Cambridge, Mass.) and Sigma-Tau

Group (Rome)

Kalbitor Ecallantide Hereditary angioedema

Companies decided to withdraw the MAA because European regulators indicated the data were not sufficient to prove a positive risk-benefit (11/15)

Novartis AG (Basel, Switzerland)

Rasitrio Aliskiren, amlodip-ine and hydrochlo-rothiazide

High blood pressure

Gained European approval (11/29)


Aquinox

Pharmaceuticals

Inc. (Vancouver, British Columbia

AQX01125 Anti-inflammatory drug

Asthma Began two Phase IIa trials in the UK (11/11)



Company (Location)


Avanir

Pharmaceuticals

Inc. (Aliso Viejo, Calif.)

Nuedexta Dextromethorphan hydrobromide and quinidine sulfate

Pseudobulbar affect

Submitted an application to the EMA seek-ing approval (11/1); EMA accepted the MAA filing (11/18)


vyndaqel Tafamidis Transthyretin familial amyloid polyneuropathy

European Commission approved it to treat adult patients with Stage I symptom-atic polyneuropathy (11/18)

DIABETES

AstraZeneca plc

(London) and Bristol-Myers


Onglyza Saxagliptin; DPP-4 inhibitor

Diabetes Gained a European label expansion (11/29)

Zealand Pharma

A/S (Copenhagen, Denmark)

Lyxumia Lixisenatide; once-daily GLP-1 agonist

Type II diabetes A European marketing authorization application has been submitted by its partner Sanofi SA for Lyxumia (11/4)

INFECTION

Gilead Sciences


Tenofovir A gel HIV The National Institute of Allergy and Infectious Diseases requested the Microbicide Trials Network discontinue the use of tenofovir gel as there was no difference when compared to placebo (11/29)

MISCELLANEOUS

Alexion

Pharmaceuticals

Inc. (Chesire, Conn.)

Soliris Eculizumab Atypical hemo-lytic uremic syn-drome

European Commission extended the indi-cation for Soliris to include pediatric and adult patients (11/30)

Pierre Fabre

Medicament

(Castres, France)

Joncia Milnacipran Fibromyalgia Received marketing authorization in Australia (11/22)

Regeneron

Pharmaceuticals

Inc. (Tarrytown, N.Y.) and Bayer

HealthCare (Leverkusen, Germany)

Eylea Aflibercept Wet age-related macular degen-eration

Started a Phase III trial in China (11/29)

Notes:

CHMP = Committee for Medicinal Products for Human Use; EMA = European Medicines Agency; MAA = Marketing authorization application; MRP = Mutual Recognition Procedure.





FDA Submissions, Approvals & Other Actions: Nov. 2011Company (Location)


AUTOIMMUNE

Cipher

Pharmaceuticals

Inc. (Mississauga, Ont.)

Cip-isotretinoin

Formulation of isotretinoin

Acne Submitted an amendment to its pend-ing NDA; it includes additional clinical information requested by the FDA (11/28)

Horizon Pharma

Inc. (Deerfield, Ill.)Lodotra A modified delayed-

release formulation of low-dose predni-sone


FDA accepted the filing and will review its NDA (11/30)

Regeneron

Pharmaceuticals


Arcalyst Rilonacept injection To prevent gout flares

FDA has accepted the company’s sBLA (11/23)

CANCER

Bristol-Myers


Erbitux Cetuximab; mono-clonal antibody

Late-stage head and neck cancer

FDA approved it for use with chemo-therapy (11/8)

EUSA Pharma Inc.

(Oxford, UK)Erwinase Asparaginase

Erwinia chrysanthe-mi

Acute lymphoblas-tic leukemia

FDA approved it (11/21)

Genentech Inc.


Avastin Bevacizumab Breast cancer FDA revoked the approval in the breast cancer indication because evi-dence showed the benefits did not outweigh the risks (11/21)

Millennium

(Cambridge, Mass.)Velcade Bortezomib Multiple myeloma FDA approved an sNDA, updating the

label to include additional long-term overall survival data (11/3)

Onyx

Pharmaceuticals

Inc. (South San Francisco)

Carfilzomib A next-generation proteasome inhibitor

Relapsed and refractory multiple myeloma

FDA accepted the new drug applica-tion (11/30)


Mylotarg Gemtuzumab ozo-gamicin

Acute myeloid leu-kemia

FDA has removed approval upon the company’s request after a confirmato-ry trial failed to demonstrate clinical benefit and revealed safety concerns (11/29)

Spectrum

Pharmaceuticals

Inc. (Henderson, Nev.)

Zevalin Ibritumomab tiux-etan

Non-Hodgkin’s lymphoma

Received FDA approval to remove the pre-treatment bioscan requirement for Zevalin (11/22)

SymBio

Pharmaceuticals

Ltd. (Tokyo)

Treakisym Bendamustine hydrochloride

Non-Hodgkin’s lym-phoma and mantle cell lymphoma

Started a Phase II trial in Japan (11/15)

CARDIOVASCULAR

Amarin Corp. plc (Dublin, Ireland)

AMR101 A prescription-grade fish oil

High triglycerides FDA has accepted its NDA (11/29)



Company (Location)


Chelsea

Therapeutics

International Ltd.

(Charlotte, N.C.)

Northera Droxidopa; an oral synthetic precursor of norepinephrine

Symptomatic neu-rogenic orthostatic hypotension

FDA accepted for filing its NDA (11/18)


IntelGenx Corp.

(Saint Laurent, Quebec)

CPI-300 A high-strength for-mulation of bupro-pion hydrochloride

Major depressive disorder

Gained FDA approval (11/15)

Otsuka

Pharmaceutical

Co. Ltd. (Tokyo)

Aripiprazole Depot formulation Schizophrenia FDA agreed that an NDA for mainte-nance treatment is sufficiently com-plete for review (11/28)

Pacira

Pharmaceuticals

Inc. (Parsippany, N.J.)

Exparel Bupivacaine lipo-some suspension, 1 .3%

Postsurgical pain relief

Gained FDA approval (11/1)

QRxPharma Ltd.

(Sydney, Australia)MoxDuo Morphine/

oxycodonePain FDA plans to review the NDA with a

PDUFA data of June 25, 2012 (11/9)

Transcept

Pharmaceutical

Inc. (Point Richmond, Calif.) and Purdue

Pharmaceutical

Products LP

(Stamford, Conn.)

Intermezzo Zolpidem tartrate Insomnia FDA approved it for use as needed to treat insomnia characterized by mid-dle-of-the-night waking followed by difficulty returning to sleep (11/28)

Vivus Inc.


Qnexa Phentermine/topira-mate

Obesity FDA accepted for filing and review the NDA (11/4)

DIABETES

Alimera Sciences

Inc. (Atlanta)Iluvien Fluocinolone ace-

tonide intravitreal insert

Diabetic macular edema

Received a second complete response letter with the FDA saying the risks of adverse events in two Phase III trials were not offset by the benefits; the agency requested two additional tri-als (11/14)

Furiex

Pharmaceuticals

Inc. (Morrisville, N.C.) and Takeda

Pharmaceutical

Co. Ltd. (Osaka, Japan)

Alogliptin/metformin

Fixed-dose combi-nation

Type II diabetes Submitted an NDA (11/29)



Company (Location)


INFECTION

Santen Inc.

(Emeryville, Calif.)DE-109 Sirolimus Chronic/refractory

anterior non-infec-tious uveitis, non-infectious intermediate uve-itis, non-infectious panuveitis and non-infectious uveitis affecting the poste-rior segment of the eye

FDA granted orphan drug designation for sirolimus (11/11)

MISCELLANEOUS

Incyte Corp.

(Wilmington, Del.)Jakafi Ruxolitinib; JAK

inhibitorMyelofibrosis FDA approved it (11/17)

Regeneron

Pharmaceuticals


Eylea Aflibercept Wet age-related macular degeneration

FDA granted approval (11/22)

Shire plc (Dublin, UK)

Vpriv Velaglucerase alfa; an enzyme replace-ment therapy

Gaucher’s disease Submitted regulatory filings in the U.S. and Europe for the production of Vpriv (11/23)

Summit Corp.

(Oxford, UK)SMT C1100 A small-molecule

drug that up-regu-lates production of a naturally occurring protein called utro-phin to replace missing dystrophin

Duchenne’s muscular dystrophy

FDA granted orphan drug status (11/29)

Notes:

BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment.





BioWorld Stock Report For Public Biotechnology CompaniesCompany Symbol Close Close %Change Vol 12/2 12/9 WK YTD (000)

Company Symbol Close Close %Change Vol 12/2 12/9 WK YTD (000)

The Week’s Biggest Gainers and Losers

(By Percent) (By Dollars)

10 BIGGEST U.S. LOSERS FOR THE WEEK

Unigene -42.70Alexza Pharma -39.22Poniard Pharma -21.11Pharmacyclics -20.73XenoPort -18.43Titan Pharma -16.54Galena Biopharma -14.93Complete Genomics -14.04Idera Pharma -13.60Epicept -13.33

Regeneron Pharma -4.90Pharmacyclics -3.24Onyx Pharma -3.18Alexion Pharma -1.92InterMune -1.35Poniard Pharma -1.03Momenta Pharma -0.92Myriad Genetics -0.91BioMarin Pharma -0.90XenoPort -0.89

10 BIGGEST U.S. WINNERS FOR THE WEEK

(By Percent) (By Dollars)Generex Biotech 266.67Bio-path Holdings 242.86Affymax 45.52Alseres 37.50Dara Biosciences 27.45Flamel Tech 26.23Zalicus Inc 21.05Peregrine Pharma 20.62Oncothyreon 20.26Synta Pharma 19.55

Affymax 2.39Celgene 2.37Seattle Genetics 1.68Oncothyreon 1.39Flamel Tech 1.07Alkermes 0.97Auxilium Pharma 0.88Bio-path Holdings 0.85Raptor Pharma 0.81Pacira Pharma 0.81

3S Bio SSRX 12.18 11.53 -5.34 -23.03 291Aastrom Biosci ASTM 2.12 2.09 -1.42 -18.36 1239Acadia Pharma ACAD 0.98 1.05 7.14 -14.63 438Accentia Biopharma ABPI 0.3 0.33 10.00 -56.00 268Access Pharma ACCP 1.44 1.44 0.00 -41.22 100AcelRx Pharma ACRX 2.85 2.73 -4.21 -40.00 5Achillion ACHN 6.85 7.52 9.78 85.22 4862Acorda Therap ACOR 22.53 23.22 3.06 -15.59 1624Adamis ADMP 0.22 0.22 0.00 7.32 300Adolor ADLR 4.6 4.77 3.70 284.68 4195Adventrx Pharma ANX 0.62 0.6 -3.23 -77.01 1674Aegerion Pharma AEGR 16.63 16.91 1.68 19.34 358AEterna Zentaris AEZS 1.64 1.74 6.10 -0.57 9573Affymax AFFY 5.25 7.64 45.52 14.89 35197Agenus AGEN 2.43 2.37 -2.47 -60.89 159Albany Molecular AMRI 2.34 2.3 -1.71 -60.88 1412Alexion Pharma ALXN 66.98 65.06 -2.87 61.52 6022Alexza Pharma ALXA 1.02 0.62 -39.22 -50.79 13856Alimera Sciences ALIM 1.23 1.4 13.82 -86.51 1520Alkermes ALKS 14.86 15.83 6.53 28.70 3755Allos Therapeutics ALTH 1.27 1.3 2.36 -71.86 2585Alnylam Pharma ALNY 6.81 7.09 4.11 -28.24 987Alseres ALSE 0.08 0.11 37.50 -15.38 41AMAG Pharma AMAG 18.4 19.05 3.53 4.84 805Amarin AMRN 7.78 7.27 -6.56 -12.30 8463Amgen AMGN 58.1 58.59 0.84 5.51 95571Amicus FOLD 3.21 3.43 6.85 -28.39 267Ampliphi Biosci APHB 0.09 0.09 0.00 -64.00 119Amylin Pharma AMLN 10.5 10.45 -0.48 -29.82 7809Anacor Pharma ANAC 5.42 5.59 3.14 4.10 226Anthera Pharma ANTH 6.48 6.66 2.78 36.76 789Ardea Biosciences RDEA 19.93 19.05 -4.42 -27.51 542Arena Pharma ARNA 1.68 2 19.05 16.96 19743Ariad Pharma ARIA 11.9 11.63 -2.27 124.52 10782ArQule ARQL 5.54 5.71 3.07 -1.38 1013Array BioPharma ARRY 2.12 2.3 8.49 -22.82 1456Aryx ARYX 0.01 0.01 0.00 -85.71 59Astex Pharma ASTX 1.67 1.69 1.20 -24.89 1164Auxilium Pharma AUXL 17.51 18.39 5.03 -13.30 3253Avanir AVNR 2.37 2.3 -2.95 -42.64 10150Avax Tech AVXT 0.04 0.04 0.00 -11.11 588AVEO Pharma AVEO 16.88 16.96 0.47 15.37 1156AVI BioPharma AVII 0.69 0.71 2.90 -67.13 2497Bellus Health BLUS 0.04 0.04 0.00 -74.19 495BG Medicine BGMD 4.22 3.85 -8.77 -53.61 114BioCryst Pharma BCRX 2.94 2.66 -9.52 -48.15 571Biodel BIOD 0.61 0.67 9.84 -65.10 756Biodelivery Sci BDSI 1.05 1.08 2.86 -70.81 801Biogen Idec BIIB 113.24 112.95 -0.26 69.09 5638BioMarin Pharma BMRN 34.53 33.63 -2.61 23.78 5716Bio-path Holdings BPTH 0.35 1.2 242.86 233.33 53BioSante Pharma BPAX 2.52 2.45 -2.78 47.59 7997Cadence Pharma CADX 4.45 3.91 -12.13 -48.14 1735Cardiome CRME 2 2.02 1.00 -69.16 704

Cardiovascular Bio CVBT 0.4 0.35 -12.50 2.94 424Catalyst Pharma CPRX 1.07 1.13 5.61 17.71 639Celgene CELG 61.21 63.58 3.87 6.98 17107Cell Therapeutics CTIC 1.11 1.19 7.21 219.89 14550Celldex Therap CLDX 2.9 2.89 -0.34 -30.70 970CEL-SCI CVM 0.3 0.32 6.67 -61.55 4548Cerus CERS 2.99 3.11 4.01 25.40 3881Chelsea Therap CHTP 5.45 5.15 -5.50 -32.15 2934Cleveland Biolabs CBLI 2.94 3.08 4.76 -57.16 960Codexis CDXS 5.85 5.72 -2.22 -46.04 1107Columbia Labs CBRX 2.16 2.19 1.39 0.00 2252CombiMatrix CBMX 1.74 1.55 -10.92 -30.49 136Complete Genomics GNOM 3.49 3 -14.04 -59.84 2084Compugen CGEN 4.43 4.35 -1.81 -12.65 239Corcept CORT 3.2 3.28 2.50 -16.33 511CorMedix CRMD 0.35 0.33 -5.71 -81.25 39Cornerstone Therap CRTX 5.49 5.52 0.55 -2.30 109Cubist Pharma CBST 38.1 37.79 -0.81 75.12 3767Curis CRIS 3.65 3.78 3.56 87.13 1377Cytokinetics CYTK 1.04 1.01 -2.88 -51.67 525CytRx CYTR 0.35 0.33 -5.71 -67.00 2494Dara Biosciences DARA 1.02 1.3 27.45 -61.42 191Dendreon DNDN 8.62 7.96 -7.66 -77.52 22292DepoMed DEPO 4.82 4.73 -1.87 -27.01 1847DiaDexus DDXS 0.17 0.15 -11.76 -48.28 663Discovery Labs DSCO 1.69 1.7 0.59 -48.95 652DURECT DRRX 1.3 1.29 -0.77 -63.66 1319Dusa Pharma DUSA 3.77 4.08 8.22 65.85 500Dyax DYAX 1.29 1.36 5.43 -34.93 1812Dynavax DVAX 3.2 3.2 0.00 3.23 9546Emergent BioSol EBS 17.57 17.42 -0.85 -26.44 867Emisphere Tech EMIS 0.37 0.33 -10.81 -86.08 267Endocyte ECYT 10.4 11 5.77 42.30 372Entremed ENMD 1.54 1.51 -1.95 -70.28 111Enzo Biochem ENZ 2.27 2.33 2.64 -57.09 574Enzon Pharma ENZN 7.01 7.03 0.29 -42.38 909Epicept EPCT 0.3 0.26 -13.33 -69.41 109Exelixis EXEL 4.21 4.45 5.70 -48.20 6628Flamel Tech FLML 4.08 5.15 26.23 -26.01 352Forest Labs FRX 29.89 29.66 -0.77 -7.23 9587Furiex Pharma FURX 17.3 16.63 -3.87 15.09 529Galena Biopharma GALE 0.67 0.57 -14.93 -78.41 3442Generex Biotech GNBT 0.09 0.33 266.67 11.90 2397Genomic Health GHDX 28.07 28.09 0.07 28.38 864GenVec GNVC 2.75 2.76 0.36 -51.58 143Geron GERN 1.7 1.59 -6.47 -69.48 4071Gilead Sciences GILD 39.4 39.01 -0.99 7.58 47914GTx GTXI 3.02 2.87 -4.97 3.99 578Harvard Bio HBIO 4.21 4.02 -4.51 -0.99 432Human Genome HGSI 7.57 7.54 -0.40 -68.84 26280Idenix Pharma IDIX 8.22 8.13 -1.09 62.93 3291Idera Pharma IDRA 1.25 1.08 -13.60 -62.76 160ImmunoCellular IMUC 1.43 1.45 1.40 6.62 76ImmunoGen IMGN 12.23 12 -1.88 27.39 2368



Antisoma ASM 1.7 1.73 1.76 -94.76 2127Ark Therapeutics AKT 3.6465 3.8 4.21 -72.95 1664Asterand ATD 2.75 2.45 -10.91 -86.83 254Oxford Biomedica OXB 4.97 4.87 -2.01 -56.71 1583Phytopharm PYM 7.5 8.49 13.20 -21.68 433Puricore PURI 24 25.9 7.92 93.28 35Renovo Group RNVO 15.92 15.755 -1.04 -45.48 1963SkyePharma SKP 44.5 41.5 -6.74 -51.72 68Vernalis plc VER 19 19 0.00 -78.16 569

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Company Symbol 12/2 12/9 %WK %YTD Volume

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Company Symbol 12/2 12/9 %WK %YTD Volume

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NOTES:Trading volumes for Nasdaq, Amex and NYSE are recorded as the total 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: +2.54%Range: -42.70% to +266.67%; Number Of Companies: 203(does not include LSE or TSX; not market weighted)Average Percent Change YTD: -11.39%Range: -90.97% to +466.92%; Number Of Companies: 203(does not include LSE or TSX; not market weighted)

Adherex Technol AHX 0.45 0.35 -22.22 677.78 13Allon Therap NPC 0.19 0.2 5.26 -37.50 592BELLUS Health BLU 0.04 0.04 0.00 -77.78 580Bioniche Life Sci BNC 0.49 0.48 -2.04 -14.29 173Cangene CNJ 1.8 1.81 0.56 -64.51 226Isotechnika ISA 0.12 0.11 -8.33 -33.33 116Lorus Therap LOR 0.2 0.2 0.00 207.69 32Medicago MDG 0.53 0.57 7.55 -14.93 926MethylGene MYG 0.32 0.31 -3.13 10.71 445Oncolytics Biotech ONC 4.28 4.09 -4.44 48.73 263Protox Therap PRX 0.34 0.31 -8.82 -63.53 132Resverlogix RVX 1.19 1.4 17.65 -42.15 625SemBioSys Gen SBS 0.06 0.06 0.00 -70.00 65Tekmira Pharma TKM 1.7 1.66 -2.35 78.49 240Thallion Pharma TLN 0.11 0.14 27.27 27.27 273Theratechnologies TH 2.24 2.34 4.46 -47.18 1053YM BioSciences YM 1.4 1.61 15.00 11.03 141

Immunomedics IMMU 3.32 3.39 2.11 -5.83 1194Incyte INCY 14.24 13.6 -4.49 -18.90 10132Infinity Pharma INFI 9.49 9.78 3.06 61.65 478Inhibitex INHX 15.51 14.74 -4.96 466.92 10629InterMune ITMN 18.65 17.3 -7.24 -53.13 4912Ironwood Pharma IRWD 11.94 12.28 2.85 18.65 1184ISIS Pharma ISIS 7.27 7.13 -1.93 -29.75 2614Keryx Biopharma KERX 2.65 2.59 -2.26 -44.54 5155Lexicon LXRX 1.2 1.27 5.83 -14.77 2977Ligand Pharma LGND 11.67 12.27 5.14 37.71 575MannKind MNKD 3.04 2.92 -3.95 -64.08 2334Map Pharma MAPP 13.61 13.73 0.88 -17.59 392Maxygen MAXY 5.65 5.59 -1.06 41.88 432MDRNA MRNA 0.15 0.14 -6.67 -90.97 10134Metabolix MBLX 4.94 4.33 -12.35 -63.61 834Micromet MITI 6.52 7.01 7.52 -15.24 3246Momenta Pharma MNTA 16.42 15.5 -5.60 3.20 3772Myrexis MYRX 2.73 2.79 2.20 -34.35 983Myriad Genetics MYGN 21.29 20.38 -4.27 -11.12 2245Nabi Biopharma NABI 1.78 1.81 1.69 -69.06 1790Nanogen NGEN 0.01 0.01 0.00 17.65 409Nektar Therap NKTR 4.92 5.29 7.52 -59.18 7692Neurocrine Biosci NBIX 6.85 7.45 8.76 -3.12 1473NeurogesX NGSX 1.01 1.07 5.94 -83.07 1033NovaBay NBY 1.14 1.05 -7.89 -37.13 774Novavax NVAX 1.46 1.43 -2.05 -43.92 3350NPS Pharma NPSP 6.03 6.23 3.32 -22.32 3489NuPathe PATH 2.2 2.24 1.82 -75.28 126Omeros OMER 3.93 4.04 2.80 -51.67 348OncoGenex Pharma OGXI 11 11.57 5.18 -29.67 138Oncothyreon ONTY 6.86 8.25 20.26 152.29 5461Onyx Pharma ONXX 43.81 40.63 -7.26 9.96 7742Opko Health OPK 5.1 4.82 -5.49 29.92 5911Optimer Pharma OPTR 11.71 12.12 3.50 5.39 4208OraSure Tech OSUR 9.5 9.39 -1.16 57.29 1446Orexigen OREX 1.71 1.74 1.75 -78.68 1441Osiris Thera OSIR 4.76 4.94 3.78 -37.78 271Oxis International OXIS 0.04 0.04 0.00 -70.37 107Pacific Biosciences PACB 2.73 2.67 -2.20 -83.22 1306Pacira Pharma PCRX 7.81 8.62 10.37 22.79 1136Pain Therapeutics PTIE 3.83 3.69 -3.66 -44.84 563Palatin Tech PTN 0.54 0.53 -1.85 -58.91 199Peregrine Pharma PPHM 0.97 1.17 20.62 -46.82 12005Pharmacyclics PCYC 15.63 12.39 -20.73 98.56 7134Pharmasset VRUS 128.44 129 0.44 193.58 10278Pharmos PARS 0.06 0.06 0.00 -40.00 24Poniard Pharma PARDD 4.88 3.85 -21.11 -83.41 159Pozen POZN 3.93 3.98 1.27 -39.42 712Progenics Pharma PGNX 7 7.63 9.00 41.04 849Protalix Biothera PLX 6.13 5.37 -12.40 -46.19 2201Protein Design PDLI 6.3 6.09 -3.33 -2.09 9599Qiagen QGEN 14.53 14.34 -1.31 -26.27 8174QLT Inc. QLTI 6.84 6.74 -1.46 -5.87 410Raptor Pharma RPTP 5.38 6.19 15.06 70.05 2824Regeneron Pharma REGN 58.28 53.38 -8.41 59.92 6782Repligen RGEN 3.31 3.3 -0.30 -30.19 47Rexahn Pharma RNN 0.49 0.51 4.08 -55.65 907Rigel Pharma RIGL 7.77 7.67 -1.29 0.92 1104Sagent Pharma SGNT 20.98 20.96 -0.10 9.22 355Sangamo Biosci SGMO 2.98 2.77 -7.05 -59.62 1151Santarus SNTS 2.94 3.09 5.10 -6.08 948Savient Pharma SVNT 2.22 2.13 -4.05 -81.03 6339SciClone Pharma SCLN 4.51 4.54 0.67 7.08 782Seattle Genetics SGEN 16.41 18.09 10.24 17.93 7356Sequenom SQNM 4.17 3.89 -6.71 -52.21 12835SIGA Tech SIGA 2.06 2.43 17.96 -82.64 4269Soligenix SNGX 0.03 0.03 0.00 -84.21 Somaxon Pharma SOMX 0.74 0.7 -5.41 -78.19 665Spectrum Pharma SPPI 14.04 13.83 -1.50 95.34 11638StemCells STEM 1.81 1.65 -8.84 -37.50 185Sucampo Pharma SCMP 3.79 4.07 7.39 6.27 109Synta Pharma SNTA 4.04 4.83 19.55 -21.08 869Talon Thera TLON 0.49 0.46 -6.12 -4.17 86Targacept TRGT 7.58 7.48 -1.32 -71.97 2257Telik TELK 0.29 0.26 -10.34 -67.70 181Tengion TNGN 0.4 0.47 17.50 -81.50 595Theravance THRX 23.43 23.34 -0.38 -8.65 1658Titan Pharma TTNP 1.33 1.11 -16.54 -9.02 99Transcept Pharma TSPT 8 7.27 -9.13 -3.71 1776Transgenomic TBIO 1.35 1.27 -5.93 78.87 937

Tranzyme TZYM 3.24 3.05 -5.86 -23.75 35Trinity Biotech TRIB 10.1 10.42 3.17 17.87 240Trius Therap TSRX 7.46 7.23 -3.08 100.00 684Unigene UGNE 0.89 0.51 -42.70 -23.88 1659United Therap UTHR 41.67 42.02 0.84 -33.65 5246Vanda Pharma VNDA 4.82 4.85 0.62 -48.29 618Ventrus Biosciences VTUS 8 8.01 0.12 21.00 43Vermillion VRML 1.28 1.22 -4.69 -83.56 465Vertex Pharma VRTX 29.25 29.37 0.41 -16.89 15038Vical VICL 4.69 4.58 -2.35 123.41 5188ViroPharma VPHM 23.55 23.78 0.98 35.11 4743Vivus VVUS 10.52 10.68 1.52 12.07 6584XenoPort XNPT 4.83 3.94 -18.43 -54.50 1603Zalicus Inc ZLCS 0.95 1.15 21.05 -29.45 6164Zogenix ZGNX 1.84 1.69 -8.15 -70.19 736

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