BIOTECHS MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARS

THURSDAY SEPTEMBER 13, 2012

VOLUME 23 , NO. 178 PAGE 1 OF 9

Financings Roundup

Emerging Markets: Brazil

Vascular Pharma Makes Splash: Raises $16M, Inks Janssen Deal

By Catherine Shaffer Staff Writer Vascular Pharmaceuticals Inc., of Chapel Hill, N.C., closed two deals encompassing the alpha and the omega of its existence. First, it announced the successful completion of a $16 million Series A equity nancing co-led by Intersouth Partners and MPM Capital to continue development of its monoclonal antibody, VPI-2690B, for diabetic nephropathy. While for many biotech companies, a Series A nancing might be the beginning of an exciting and uncertain future, Vascular Pharmas second announcement on the same day revealed that the edgling company already has its exit strategy in the bag. It signed an agreement with Janssen Biotech Inc., granting the Johnson & Johnson subsidiary the exclusive See Financings Roundup, Page 3

ANVISA: Whats on Paper Not Always Whats Practiced

By Mari Serebrov Washington Editor Drugmakers going into emerging markets are nding that what looks clear on paper may not be so in practice. Thats been industrys experience with the Brazilian National Health Surveillance Agencys (ANVISA) drug regulatory path, Benny Spiewak, senior counsel with Zancaner Costa, Bastos e Spiewak Advogados, told BioWorld Today. On the surface, ANVISAs path looks to be on par with that of the FDAs. But when it comes to implementation, the similarities begin to fade due to the underlying rationale that drives the two agencies. While the FDA takes a pro-safety, pro-health approach, ANVISA tends to look at the bottom line. Its a European Union model with a tropical twist, said Spiewak, who See Brazil, Page 4

AIDS 2012 Vaccine

Analysis Adds to Understanding Of RV144 Vaccine Trial Results

By Anette Breindl Science Editor Novel analyses of the RV144 HIV vaccine trial, also known as the Thai trial, have analyzed viral variants that were able to get past the protection the vaccine offered. Those results, published in Nature and presented at the AIDS Vaccine 2012 conference this week, add to the emerging understanding that targeting certain parts of HIVs envelope protein is likely the best path to an effective vaccine. The RV144 Thai HIV vaccine was perhaps the least successful vaccine ever to resurrect a eld. From the moment that results of the 16,000-patient Phase III study of Sano SAs ALVAC HIV and VaxGen Inc.s AIDSVAX B/E were reported in September 2009, it was clear that the vaccine was not effective enough. It reduced the risk of infection by about 30 percent nowhere near the 90See AIDS, Page 5

NewCo News
NeuroNascent Aims to Reverse Neurodegenerative Damage
By Marie Powers Staff Writer A slew of recent failures in Alzheimers disease (AD) could prove providential for NeuroNascent Inc., which has ambitious plans to halt and potentially reverse a range of neurodegenerative diseases. The Clarksville, Md.-based rm was founded in 2004 by Judith Kelleher-Andersson, president and chief scientic ofcer, who had two decades of experience in drug development and research in neurological disease particularly neurodegenerative disorders including positions at Neuralstem Inc., Centaur Pharmaceuticals Inc. and Cortex Pharmaceuticals Inc. She is the primary inventor on more than 50 U.S. and See NeuroNascent, Page 6

INSIDE:

OTHER NEWS TO NOTE: AMPIO, CERUS, CSL BIOTHERAPIES ...........................2 CLINIC ROUNDUP: NYMOX, ONCOLYTICS, OPEXA, SANTARUS............................8

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Other News To Note

Ampio Pharmaceuticals Inc., of Greenwood Village, Colo., entered a denitive agreement with EthyPharm SA, of Paris, to manufacture Zertane for the treatment of premature ejaculation. The orally disintegrating tablet (ODT) will be manufactured by EthyPharm under patented processes jointly owned by EthyPharm and Ampio, which acquired the ODT formulation technology in December 2011 from Valeant Pharmaceuticals International Inc., of Montreal. The agreement and manufactured drug will be used to complete the investigational new drug application for pivotal trials of Zertane in the U.S., complete the registration package for approval of Zertane in Australia based on completed Phase III European trials and nalize pricing of the drug for launch in other countries and for licensing negotiations with additional potential partners. Cerus Corp., of Concord, Calif., formed partnerships with the American Red Cross and Blood Systems Inc. (BSI) to commercialize Intercept plasma for thrombotic thrombocytopenic purpura in the U.S. The Red Cross and BSI will be responsible for manufacture of Intercept plasma in order to distribute the plasma in compliance with FDA regulations. If Intercept is approved by the FDA, Cerus will provide kits for preparation of Intercept to the two organizations, and recoup the costs from the nal sale of products. CSL Biotherapies, of King of Prussia, Pa., a subsidiary of CSL Ltd., received a contract from the Department of Health and Human Services for pre-pandemic and pandemic vaccine antigens for the national stockpile. The government may request that CSL Biotherapies manufacture and store bulk antigen for inuenza strains believed to have pandemic potential. The contract also provides for CSL to develop virus seeds for other manufacturers. The contract is worth a maximum of about $1 .5 billion. Durata Therapeutics Inc., of San Francisco, said a Qt study of its candidate dalbavancin showed there was

Stock Movers
9/12/1 2
Company Nasdaq Biotechnology Access Pharmaceuticals Inc. GTx Inc. Horizon Pharma Inc. Innity Pharmaceuticals Inc. Oncolytics Biotech Inc. Poniard Pharmaceuticals Inc. 3SBio Inc. StemCells Inc. Tengion Inc. Stock Change -$0.62 +$0. 13 +$0.73 +$0.62 +$1 .64 -$33.00 -$0. 13 +$1 .83 +$0. 19 +$0.23 -0.04% +24.53% +19.26% +15.23% +8.60% -12.09% -23.64% +15.79% +10.00% +12.92%

(Biotechs showing signicant stock changes Wednesday)

no clinically signicant effect on cardiac condition. The compound is being evaluated in two ongoing, Phase III trials for acute bacterial skin and skin structure infections. The data were presented at the Interscience Conference on Antimicrobial Agents and Chemotherapy in San Francisco. Eaton Scientic Systems Ltd., of Beverly Hills, Calif., said it will begin developing a transdermal patch delivery system for Tropine 3 for hot ashes. Eaton plans to contract with a drug delivery system expert for development of the patch and begin testing device technologies in conjunction with clinical studies in the next few weeks.

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BioWorld Today (ISSN# 1541-0595) is published every business day by AHC Media, 3525 Piedmont Road, Building Six, Suite 400, Atlanta, GA 30305 U.S.A. Opinions expressed are not necessarily those of this publication. Mention of products or services does not constitute endorsement. BioWorld and BioWorld Today are trademarks of AHC Media, a Thompson Media Group LLC company. Copyright 2012 AHC Media. All Rights Reserved. No part of this publication may be reproduced without the written consent of AHC Media. (GST Registration Number R128870672). Executive Editor: Lynn Yoffee. Managing Editor: Jennifer Boggs. ATLANTA NEWSROOM: Senior Editor: Michael Harris. Managing Editor: Amanda Lanier. BioWorld Insight Editor: Peter Winter. Database Editor: Karen Pihl-Carey. Senior Production Editor: Ann Duncan. Staff Writer: Marie Powers. WASHINGTON BUREAU: Washington Editor: Mari Serebrov. EAST COAST BUREAU: Science Editor: Anette Breindl, Staff Writer: Catherine Shaffer. EUROPEAN BUREAU: Staff Writers: Sharon Kingman, Nuala Moran, Cormac Sheridan. BUSINESS OFFICE: Senior Vice President/Group Publisher: Donald R. Johnston. Director of Brand Management: Beth Schilling. Marketing Manager: Sarah Cross. Account Representatives: Matt Hartzog, Chris Wiley. DISPLAY ADVERTISING: For ad rates and information, please call Stephen Vance at (404) 262-5511 or email him at stephen.vance@ahcmedia.com. REPRINTS: For photocopy rights or reprints, call our reprints department at (404) 262-5476. PRESS MATERIALS: Send all press releases and related information to newsdesk@bioworld.com.

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Financings Roundup
Continued from page 1 right to acquire Vascular Pharma following completion of its planned Phase II study. Vascular Pharma will receive an up-front payment and milestone payments in exchange for the acquisition rights. Were extremely excited to be able to aggressively move the program forward and to be able to partner with top-tier capital funds as well as biotech to make this program a reality, Vascular Pharma CEO Richard Shey told BioWorld Today. Shey said the company had been formed in late 2005, but until this point had operated very much in a virtual mode. VPI-2690B is designed to target the insulin-like growth factor I (IGF-1) pathway by binding to the alphaVbeta3 receptor, which inhibits IGF-1-mediated smooth muscle cell proliferation in response to hyperglycemia. That smooth muscle cell proliferation is related to plaque development in the vasculature. The VPI-2690B antibody is designed to stick to a different binding site than the commonly-targeted RGD binding site of alphaVbeta3. In animal models, VPI-2690B reversed the development of proteinuria and prevented histological changes in the kidneys of diabetic animals. Those preclinical studies in pig and rat models have been carried out at Chapel Hill School of Medicine, and are being prepared for publication in a peer-reviewed journal. Vascular Pharma is in the process of humanizing the murine antibody and preparing for Phase I trials, to be followed promptly by Phase II studies. Were currently beginning our IND-enabling preclinical program. Were targeting ling our IND by the second half of 2013, commencing clinical trials thereafter, Shey said. Vascular Pharma expects its funding to last through completion of Phase II, getting the company to the point where Janssen may exercise its option. If it does, then the $16 million Series A will be the only equity nancing that the company needs. I wouldnt want to speculate on what Janssen may or may not do, Shey said. Thats one scenario that may unfold. According to Vascular Pharma, 24 million people in the U.S. have diabetes, and another 57 million have prediabetes, at an annual health care cost of $116 billion. The chronic hyperglycemia of diabetes accelerates the formation of atherosclerotic plaques resulting in complications of diabetes including coronary artery disease, peripheral artery disease, diabetic retinopathy and poor renal function. The current standard of care for those vascular complications is statin therapy such as Crestor (rosuvastatin calcium, AstraZeneca plc) and Lipitor (atorvastatin, Pzer Inc.) to lower blood cholesterol levels. But that approach

leaves much to be desired in terms of reversing damage done. Stents and coronary artery bypass grafts also are used, but are subject to high rates of restenosis (greater than 50 percent), and are not able to address disease in smaller arteries. Vascular Pharma previously funded its program through small business technology transfer grants from the Department of Health and Human Services and through a nonequity-based partnership with a pharmaceutical partner. The company is laser-focused on its initial indication of diabetic nephropathy, but Shey said other, related vascular indications are possible for the antibody. Although our current clinical plan that is funded is specically to advance the program in diabetic nephropathy, the drug has shown impressive animal results in other diabetic complications, including accelerated atherosclerosis and diabetic retinopathy, Shey said.

Interest in Diabetic Nephropathy Widespread

According to Shey, there is no head-to-head competition from other products targeting the same cellular pathway or mode of action. There are other players, however, in the eld of diabetic nephropathy. Genkyotex SA, of London, recently raised $26 million to extend a series C nancing to develop its NOX-1 and NOX4 inhibitor, GKT137831 , for diabetic nephropathy. That drug has progressed to a Phase Ib multi-ascending dose study, and Phase II is expected to begin by the end of 2012. (See BioWorld Today, July 11 , 2012.) Noxxon Pharma AG, of Berlin, treated its rst patient in June 2012 in a Phase IIa trial of NOX-E36, an anti-CCL2/ MCP-1 drug. That 12-week trial will evaluate the efcacy of the subcutaneous injections of 0.5 mg/kg of NOX-E36 twice weekly compared to placebo in about 50 patients. Pharma giants Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis, have thrown their hats into the ring, as well. They reported results from a post-hoc analysis of a trial of Tradjenta (linagliptin) in Type II diabetes patients showing a 29 percent reduction in urinary albumin-to-creatinine ratio with linagliptin plus angiotensin-converting enzyme inhibitors (ACEs) and angiotensin receptor blockers (ARBs) vs. ACEs and ARBs alone at 24 weeks. They presented those data at the American Diabetes 72nd Annual Scientic Sessions in Philadelphia in June. In other nancings news: 3SBio Inc., of Shenyang, China, said its board received a nonbinding proposal letter from Chairman and CEO Jing Lou and CPE China Fund LP to acquire the companys outstanding shares in a going private transaction valued at $15 per American Depositary Share in cash, subject to See Financings Roundup, Page 7

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Brazil

Continued from page 1 helps life sciences companies navigate the Brazilian system. He said he hoped that a Food and Drug Law Institute (FDLI) conference held in Sao Paulo this week would be the beginning of a new round of communication between the FDA and ANVISA that will address their regulatory differences so their paper similarities will hold true in practice. Price controls are one of the biggest challenges for companies going into Brazil, as they have to get the price of their drugs approved before getting marketing authorization. Since the government is the most important buyer of medicines in the country, it imposes a price cap that represents a discount of up to 40 percent. This is for starters, Spiewak said. Even with the discounts, not every new drug will be covered by the governments universal health care system. In addition to the pricing structure, drugmakers can face a number of regulatory hurdles in Brazil. For one thing, the government seems to favor generic drugs and has no clear, straightforward policy for innovative drugs, Spiewak said. As a result, Brazil isnt known as a global launching point for new drugs. Other than perhaps a few new drugs from local companies, Spiewak couldnt think of any new drugs that were approved rst in Brazil. Another challenge is that Brazils regulatory path is time-consuming and the level of transparency isnt as high as it could be. On average, the review time is 18 months to 24 months, Spiewak said, adding that theres no accelerated approval for unmet needs. Instead, Brazil views all drugs, except biologics, as the same. Given the incentives for generics, Brazil also has a high level of competition that could lead to cutting corners on safety. For instance, a complex drug, such as a synthetic protein, has the same regulatory generic thresholds as an aspirin. Thats a bit scary, Spiewak said. One of the topics at this weeks FDLI conference was how to best regulate drugs that are more complex than traditional small molecules and even some biologics, J. Michael Nicholas, senior director of life cycle initiatives at Teva Pharmaceuticals Inc., told BioWorld Today. How complex drugs are regulated makes a difference when it comes to follow-ons. In places such as Australia, Canada, Chile, Europe and Mexico, theyre treated more like biologics, so clinical trials are required for approval of followons. The FDA, however, treats proteins like small molecules, so trials arent generally required for generic versions. Whether theyre biologic or synthetic like Tevas multiple sclerosis drug Copaxone (glatiramer acetate injection), proteins shouldnt be lumped in with small molecules, said Nicholas, who addressed the issue at the conference. Because the composition of those drugs is highly dependent on the manufacturing process, safety issues could arise that wouldnt be detected by bioequivalence testing, he added. Those issues could increase in the future as more

synthetic proteins are expected to be developed. And generally, those drugs are intended to treat serious diseases. Until the drugs are fully understood, it makes sense to err on the side of caution and require clinical trials for the follow-ons, Nicholas said.

The Draw of Brazil

Despite the regulatory challenges and unanswered questions, Brazil has become the No. 1 emerging market of choice for many drugmakers, Spiewak said, citing a recent survey in which 85 percent of biopharma executives indicated they were interested in the Brazilian market over other emerging countries. He chalked that preference up to Brazils diverse population of nearly 200 million people and the countrys position as a gateway to other emerging economies, including the rest of Latin America, which is coming into its own as an important geographic focus for drug companies. By 2015, Deutsche Bank estimated Latin America will have four of the top 20 biopharma markets. Most countries in the region are expected to grow their markets by more than 10 percent annually from 2011 to 2015 compared with less than 3 percent for the U.S. and 1 percent to 4 percent for Western Europe. (See BioWorld Insight, June 4, 2012, and July 2, 2012.) Another thing Brazil has going for it is its economy. When it comes to gross domestic product (GDP) based on purchasing power, Brazil ranks second among the four BRIC countries (Brazil, Russia, India and China). With a 2011 percapita GDP of nearly $2.5 trillion, Brazil was No. 75 among the world economies, according to the World Bank. Russia was No. 45, China ranked 94th and India came in at 125.

Other News To Note

Edison Pharmaceuticals Inc., of Mountain View, Calif., said the European Medicines Agencys Committee for Orphan Medicinal Products granted orphan designation to EPI-743 for the treatment of Leigh syndrome. The designation was based on data from a recently completed Phase IIa study, compassionate use in Europe and a U.S. expanded access program. No treatment has been authorized in the European Union for the rare syndrome caused by mutations in mitochondrial respiratory enzymes. Gliknik Inc., of Baltimore, said preclinical data from rheumatoid arthritis and thrombocytopenia models showed its stradomers have clinical therapeutic potential. Laboratory-made clusters of the Fc portion of human antibodies, stradomers are designed to mimic the efcacy of IVIG (intravenous immunoglobulin). The data were recently published online in Arthritis Research and Therapy. Gliknik is advancing its lead stradomer candidate, GL-2045, into contract manufacturing for clinical studies scheduled to begin late next year. (See BioWorld Today, Jan. 15, 2010.)

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AIDS

Continued from page 1 plus percent risk reduction that is typical for most vaccines. But in the words of writer Antoine de St. Exupery, We live not by things, but by the meaning of things. And the meaning of the RV144 trial was that a HIV vaccine is possible. Scientists immediately began to analyze the data from the trial to understand why the vaccine was effective at all, and what it might take to make a vaccine effective enough for widespread use. One region that emerged as an important determinant of how well the vaccine worked was the V1V2 loop of HIVs env protein. People who were protected in the trial often produced antibodies to that part of the virus. And senior author Jerome Kim, who is at the U.S. Military Research Program, told BioWorld Today that a fair number of the broadly neutralizing antibodies that have been identied recently contact the V2 loop of the env protein. In their work, which was published in the Sept. 10, 2012, advance online edition of Nature, the authors used a complementary approach. If antibody analysis considers the immune systems reaction to the vaccine, Kim said, basically, we looked at things from the virus point of view. The reason to look at the viral end of the interaction is that comparing infected and uninfected individuals shows that immune responses are not randomized among vaccines, as the authors wrote in their paper. In other words, those people in which the vaccine prevented HIV infection might have stronger immune responses overall, and the production of antibodies to the V1V2 loop might be a symptom of that overall strong immune response rather than the cause of the vaccines effectiveness. Comparing the HIV genomes of vaccinated vs. unvaccinated people who acquired an infection during the trial circumvents that issue. If the [V2-targeting] antibodies had exerted some pressure, we would see it in the comparison between viruses in individuals receiving vaccine vs. placebo, rst author Morgane Rolland told the audience at a Monday press conference at the AIDS 2012 meeting in Boston. The effectiveness of those antibodies would translate into an absence of certain viral strains in vaccinated patients. For their studies, Rolland, Kim and their colleagues sequenced the genome of nearly 1 .000 HIV strains that were isolated from 44 vaccinated and 66 placebo patients. They found that the V2-targeting antibodies that were induced by the vaccine did indeed protect vaccinated persons. The vaccines efcacy against viral strains that matched the vaccines V2 epitope at two specic amino acid positions was 50 percent to 80 percent, higher than the 30 percent overall efcacy that it demonstrated. The work converges with previous evidence, Kim said, that targeting the V2 loop of the env protein can lead to an efcacious vaccine. The next question, however, is how to

target it in a way that provides broad-spectrum protection. Rolland was blunt about her opinion of how broad such protection could be, even under the best of circumstances. There is so much diversity in HIV that you are never going to have a vaccine that will match viruses for everybody, she said. There are different strategies to do that. But there is not going to be a conserved V2 that would be a central immunogen that would match every possible [viral strain]. Nevertheless, one obvious question arising from the work is whether it is possible to engineer antigens that provide better protection. Kim said there are some preliminary studies suggesting that there are ways to engineer the V2 loop to improve its performance as a vaccine. And among the sessions at the conference were both an oral abstract session dedicated to V1/V2 Antibody Responses and a symposium on New Env Immunogens.

Clinic Roundup
Andromeda Biotech Ltd., of Yavne, Israel, said it completed patient enrollment in its conrmatory Phase III DIA-AID2 trial using DiaPep277 for the treatment of Type I diabetes. The study includes 475 subjects, ages 20 to 45, within six months of diagnosis with residual insulin secreting cells. Patients are treated once every three months with a 1-mg dose of the drug or placebo for a period of two years. The primary outcome is the ability of DiaPep227 to maintain insulin secretion, and results are expected at the end of 2014. DiaPep227 is a peptide of 24 amino acids derived from the sequence of heat-shock protein 60. e-Therapeutics plc, of Oxford, UK, said it started a second Phase I trial testing cancer drug ETS2101 , a drug designed to promote apoptosis. The dose-escalating study is expected to enroll up to 45 patients with a variety of solid tumors. The primary objective is to evaluate the safety of ETS2101 and identify an appropriate dose for Phase II development, while secondary objectives include initial investigation of the drugs activity and pharmacokinetics. Data are expected in 2013. An investigator-led Phase I trial is ongoing testing the drug in brain cancer. KaloBios Pharmaceuticals Inc., of South San Francisco, said it started a Phase II study of KB003, an antiGM-CSF Humaneered monoclonal antibody, in subjects with severe asthma uncontrolled by corticosteroids. The study is expected to enroll about 150 patients, who will be randomized to KB003 or placebo in addition to current therapy for ve months. The primary endpoint will be the change in mean pre-bronchodilator forced expiratory volume from baseline. Secondary endpoints will include asthma exacerbation rate and the degree of asthma symptom control. The study is expected to be completed in the rst quarter of 2014.

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NeuroNascent

Continued from page 1 global patents in small-molecule therapeutics. Kelleher-Andersson next tapped Kathleen Mattis, who served as vice president of nance at Neuralstem, to run the nancial side of NeuroNascent, and the rm raised initial funding from friends and family. In 2005, NeuroNascent whose name refers to new neurons launched operations. The companys discovery platform starts with human neuronal progenitor cells from the adult hippocampus, which are constantly being replenished through the process of neurogenesis. Even when assailed by certain neurodegenerative conditions, the brain attempts to compensate for the neuron loss by promoting further proliferation and differentiation into new neurons, which migrate to regions of the brain exhibiting the neurodegeneration, Kelleher-Andersson explained. Drugs that accelerate the neurogenesis process could potentially halt and even reverse disease progression, she reasoned. To nd such compounds, NeuroNascent needed an oral small molecule that could easily cross the blood-brain barrier. The company licensed several focused smallmolecule libraries, screened the libraries to identify agents with the ability to produce adult neurons from progenitor cells and examined whether the neurogenic agents also were neuroprotective. The company then began to optimize the agents in vitro and move them into animal models. We have to prove the neurogenesis we saw in cell cultures also occurs in animals and these new neurons actually correlate with improved behavior, characteristics such as enhanced memory performance, reduced anxiety and heightened motor function, Kelleher-Andersson told BioWorld Today. Were not chasing a single target but looking to enhance cellular function. Unlike other neurodegenerative drug candidates, NeuroNascents program isnt seeking to affect symptoms. Were actually trying to reverse a decit, not just inhibit it, Kelleher-Andersson said. Were setting the bar extremely high. Meeting that goal requires greater effort in animal models than the typical preclinical study, she added. NeuroNascents lead compound, NNI-362, demonstrated the ability to reverse a cognitive decit in aged mice as well as the capacity to halt motor decits in a neurodegenerative model of Huntingtons disease. In potentially game-changing work, NeuroNascent also examined a Down syndrome model in transgenic mice, using its technology to reverse cognitive decits associated with the disorder. That nding correlated with the increase in neurogenesis in those animals, Kelleher-Andersson said. The lead indications for that therapeutic candidate,

NNI-351 , are depression and post-traumatic stress disorder (PTSD). But NNI-351 also inhibits a protein, Dyrk1a protein, that is overproduced in individuals with Down syndrome, perhaps enabling NNI-351 to increase the production of new neurons in the hippocampus and subsequently increasing learning and memory in Down syndrome transgenic mice. Importantly, those benecial effects modied the disease, rather than simply masking symptoms, in the mouse model, according to Kelleher-Andersson. NeuroNascents AD compound is now in investigational new drug (IND)-enabling studies. NNI-351 has advanced to preclinical studies in depression and PTSD, and a Parkinsons disease (PD) drug is in the optimization phase. All of the compounds are covered for composition of use in patent lings. We think the only way really to affect the 30 million Alzheimers disease patients worldwide is to be neurorestorative as well as neuroprotective, KelleherAndersson emphasized. Thus, the companys platform seeks not only to enhance the growth of additional neurons but also to protect those neurons so they dont die off in the same way, she said. Having agents that are truly neurogenic producing new neurons, not just new connections as well as neuroprotective by enabling those neurons actually to survive is critical for many neurodegenerative diseases, Kelleher-Andersson added. Were in a good place here, if we can get into the clinic. To that end, NeuroNascent is using roughly a 50/50 mix of translational grants and angel funding to complete preIND studies for NNI-362. With the two principals the only full-time employees, we can survive on very little money, Kelleher-Andersson said. Once the IND is led, the company hopes to attract additional angels and, perhaps, pharmaceutical venture funding. Weve spoken with every major neurology player, and were still in communication with a number of these companies, Kelleher-Andersson said. We believe there are enough funding sources out there to help us with this. The push toward treatments for rare diseases may expedite the path for NNI-351 in Down syndrome, as a growing body of research suggests many intellectual disabilities may be treatable. (See BioWorld Insight, Feb. 13, 2012.) Long term, NeuroNascent hopes to partner the AD program and use those resources to move the earlier-stage PD and depression candidates forward on its own. KelleherAndersson is loath to push each asset into a separate company, as we do intend to be acquired, she said. Even though theres a trend toward pre-AD therapeutics, there are still enough pharmaceutical companies out there that are interested in helping patients with existing Alzheimers disease.

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Financings Roundup
Continued from page 3 certain conditions. CPE China Fund is an exempted limited partnership registered in the Cayman Islands and a Chinafocused private equity fund associated with CITIC Private Equity Funds Management Co. Ltd. The buyers intend to nance the acquisition through a combination of debt and equity capital and said an unnamed nancial institution expressed interest in nancing the proposed acquisition. 3SBios board formed a special committee of independent directors to consider the proposal. On Wednesday, the companys shares (NASDAQ:SSRX) gained $1 .85, or 16 percent, to close at $13.44. Sequenom Inc., of San Diego, priced its private offering of $110 million in convertible senior notes due in 2017. Sale of the notes is expected to close Sept. 17. Sequenom granted initial purchasers a 30-day option to buy up to an additional $20 million of the notes to cover overallotments. The convertible notes will bear interest at a xed rate of 5 percent annually and will mature Oct. 1 , 2017, unless earlier converted, redeemed or repurchased. The conversion rate initially will be 216.0644 shares of common stock per $1 ,000 principal amount of notes, equivalent to an initial conversion price of approximately $4.63 per share of common stock. The company plans to use the proceeds to fund the commercialization of its MaterniT21 PLUS laboratory-developed test and for other corporate purposes. On Wednesday, Sequenoms shares (NASDAQ:SQNM) gained 28 cents, closing at $3.91 .

Other News To Note

Indel Therapeutics Inc., of Vancouver, British Columbia, will partner with researchers from the University of British Columbia and Simon Fraser University to identify drug targets and therapeutics for antibiotic-resistant nosocomial pathogens. Genome BC has provided funding for the academic side of the partnership. Islet Sciences Inc., of New York, said it exclusively licensed technology from the University of California, Los Angeles, to commercialize an invention for expanding pancreatic islets. The technology is titled small molecules for islet expansion. Terms were not disclosed. NovaBay Pharmaceuticals Inc., of Emeryville, Calif., reported preclinical data supporting the mechanism of action for lead Aganocide compound NVC-422 against toxins secreted by bacterial such as E. coli. Results showed that 5.5 mM of NCT (N-chlorotaurine), or NVC-422, oxidized and inactivated the toxin Stx2, concluding that long-lived oxidants might act as tools of innate immunity against soluble toxins secreted by common bacteria. Data were presented at the Interscience Conference on Antimicrobial

Agents and Chemotherapy in San Francisco. Phylogica Ltd., of Perth, Australia, licensed its skinrepair Phylomer peptide PYC35 to New York-based Le Metier de Beaute for use in cosmetic products in Hong Kong, the UK and the U.S. Under the nonexclusive licensing agreement, Le Metier is responsible for future costs including formulation, manufacturing and marketing. Phylogica, which retained pharmaceutical rights to PYC35, will receive a signicant royalty on sales of the cosmetic products that contain PYC35, which could generate meaningful near-term revenue. The peptide is derived from the genome of a thermophilic microorganism that dwells in undersea volcanic vents and can endure extreme environmental conditions. Rib-X Pharmaceuticals Inc., of New Haven, Conn., reported data demonstrating the broad-spectrum activity of its RX-04 compounds, including when administered at single low doses. A study conducted in a Streptococcus pneumoniae lung infection model established the potential for that class of compounds to address respiratory pathogens. Studies also showed the activity of those antibiotics across a diverse array of bacterial pathogens, including carbapenem- and colistin-resistant strains of Klebsiella pneumoniae and ve major biodefense pathogens. Those and other data were presented at the Interscience Conference on Antimicrobial Agents and Chemotherapy in San Francisco. Sucampo Pharmaceuticals Inc., of Bethesda, Md., received approval from the UKs Medicines and Healthcare Products Regulatory Agency for Amitiza (lubiprostone) in chronic idiopathic constipation (CIC) and associated symptoms in adults with insufcient response to diet and other nonpharmacological measures. The locally acting chloride channel activator is approved in the U.S. in CIC in adults and in irritable bowel syndrome with constipation in women 18 and older. (See BioWorld Today, May 1 , 2008.) Trius Therapeutics Inc., of San Francisco, reported preclinical data showing that its broad-spectrum, Gram-negative antibacterial agents directed against Gyrase B and ParE had signicant potency in treating challenging Gram-negative bacteria associated with hospital-acquired infections such as E. coli, Pseudomonas aeruginosa, Acinetobacter baumannii and Klebsiella pneumonias, as well as biodefense pathogens such as Yersinia pestis, Francisella tularensis, Burkholderia mallei and Burkholderia pseudomallei. The antibacterial agents exhibited bactericidal activity, low minimum inhibitory concentrations and reduced emergence of bacterial resistance vs. comparators such as ciprooxacin, and Trius reported that the compounds dual-targeting mechanism might impair bacterias ability to develop resistance. Those and other data were presented at the Interscience Conference on Antimicrobial Agents and Chemotherapy meeting in San Francisco.

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THURSDAY, SEPTEMBER 13, 2012

BIOWORLD TODAY

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Clinic Roundup
Nymox Pharmaceutical Corp., of Hasbrouck Heights, N.J., reported favorable results from the most recent safety monitoring committee review for the companys ongoing Phase III pivotal studies of NX-1207 in benign prostatic hyperplasia. The two studies NX02-0017 and NX02-0018 are continuing with patient recruitment. Oncolytics Biotech Inc., of Calgary, Alberta, said it conducted an internal analysis of the blinded combined data for all 80 patients enrolled in the rst stage of its Phase III study testing oncolytic virus Reolysin in combination with carboplatin and paclitaxel for the treatment of head and neck cancers, with data showing a greater than expected median evolving progression-free survival (PFS) of the 80 patients, which comprise the combined control and test groups. On further examination, it was observed that patients for whom only metastatic disease was being measured were responding differently to treatment than patients who had local regional head and neck disease, with a statistically signicantly greater median evolving PFS in the metastatic disease group. Because of the difference in response, Oncolytics said it believes those two patient groups must be considered differently for the purpose of analysis and investigation and has consulted with principal investors and the FDA. Based on those discussions, the rm plans to expand enrollment in the rst stage of the trial to include 160 patients, all of whom have now been enrolled, and intends to introduce an additional segregation to differentiate between patients with local recurrent disease, with or without metastases. That means the expanded rst stage of the REO 018 study will be a separate supportive study to a planned registration study that will take the

place of the original second stage of REO 018, and the company will need additional time for PFS analysis from the expanded rst stage. News of the delay sent shares of Oncolytics (NASDAQ:ONCY) down 12 percent, or 34 cents, to close Wednesday at $2.48. Opexa Therapeutics Inc., of The Woodlands, Texas, said it started a Phase IIb trial of personalized T-cell therapy Tcelna in patients with secondary progressive multiple sclerosis (SPMS). The study, dubbed Abili-T, is expected to enroll 180 patients, with each patient receiving two annual courses of Tcelna treatment, consisting of ve subcutaneous injections per year. The primary efcacy outcome is the percentage of brain volume change at 24 months, while secondary endpoints will measure disease progress, annualized relapse rate and changes in disability. Tcelna, which was rebranded earlier this year from Tovaxin, previously missed its endpoint in a Phase IIb study in 2009 in early relapsing MS, though further analyses of the data demonstrated more promising results. Tcelna has fast-track designation in the SPMS indication. (See BioWorld Today, Sept. 9, 2009.) Santarus Inc., of San Diego, said its 264-patient Phase III study testing rifamycin SV MMX met the primary endpoint of reducing time to last unformed stool (TLUS) in subjects with travelers diarrhea. In the intent-to-treat population, the median TLUS was 46 hours for rifamycin SV MMX vs. 68 hours for placebo (p = 0.0008), and results from the per-protocol population were similar. The drug was generally well tolerated, and the frequency of treatment emergent adverse events was similar to placebo. Rifamycin SV MMX uses MMX colonic delivery technology, which provides coating in the form of gastro-resistant polymers designed to protect the active pharmaceutical ingredient against degradation in the upper gastrointestinal tract and delay the release until the tablet reaches the colon.

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Pharma: Other News To Note

H. Lundbeck A/S, of Copenhagen, Denmark, said the FDA accepted a resubmission of a new drug application by for aripiprazole depot formulation, which is in development for schizophrenia. The agency said the resubmission constituted a complete Class 2 response to its July 26 letter and set a PDUFA date of Feb. 28, 2013. Shire plc, of Dublin, Ireland, said the FDA accepted a supplemental new drug application (sNDA) for Vyvanse (lisdexamfetamine dimesylate) capsules for maintenance treatment of attention decit hyperactivity disorder in children, ages 6 to 17. The application is based on a Phase IIb study of the long-term safety and efcacy of Vyvanse. Shire also has applied for European marketing authorization for Vyvanse in the same indication.

reductions in the rate of hemorrhagic stroke and systemic embolism, in addition to providing greater reductions in major and total bleeding.

Clinic Roundup
Tetraphase Pharmaceuticals Inc., of San Francisco, reported Phase II data at the Interscience Conference on Antimicrobial Agents and Chemotherapy in San Francisco, showing that eravacycline (TP-434) was highly active against drug-resistant bacterial pathogens, demonstrating infection cure rates similar to that of comparator ertapenem for the treatment of complicated intra-abdominal infections and a strong safety prole, with low rates of gastrointestinal side effects. Tetraphase said those data supported Phase III development of the drug in serious infections, including those caused by resistant Gram-negative pathogens. Transcept Pharmaceuticals Inc., of Point Richmond, Calif., said it completed enrollment in its Phase II trial evaluating TO-2061 added as adjunctive therapy for patients with obsessive compulsive disorder (OCD) who have not adequately responded to rst-line treatment with an approved OCD medication. The 168-patient trial will test TO-2061 , a low-dose formulation of 5-HT3 receptor antagonist odansetron, with a primary endpoint measuring the difference between active and placebo treatment arms in the change from baseline according to the YaleBrown Obsessive-Compulsive Scale. The company plans to disclose top-line data in the rst quarter of 2013.

Pharma: Clinic Roundup

Boehringer Ingelheim GmbH, of Ingelheim, Germany, reported ndings at the Asia Pacic Stroke Conference in Tokyo, conrming that, in Asian populations, Pradaxa (dabigatran etexilate) offered considerable benets for the management of patients with atrial brillation. Subanalysis from the RE-LY trial demonstrated consistently superior efcacy of Pradaxa compared to warfarin for that particular patient group. Pradaxa also showed larger risk

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