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The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report
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The Ones to Watch, Apr. - Jun. 2010 -- Pharma Matters Report

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  • 1. Image CopyrIght: REUTERS/Hazir RekaTHE ONES TO WATCHA PHARMA MATTERS REPORT.ApRil-JUnE 2010 Expert review from Thomson Reuters of the most promising drugs changing clinical phase, receiving approval and launched this quarter, based on the strategic data and insight of Thomson Pharma®, the world’s leading pharmaceutical competitive intelligence solution. AWARDED TO THOMSON SCIENT FIC LIMITED (THE SCIENTIFIC BUS NESS OF THOMSON REUTERS)
  • 2. Times are tough for the pharmaceutical industry, according to a recently published report from CMR International. Portfolios are aging as the pharmaceutical pipelines that gushed in the 1990s slow to a trickle: just 7% of sales in 2009 were of medicines launched in the past 5 years (down from 8% in 2008). R&D expenditure may be dwindling too, falling 0.3% last year after several years of growth, albeit slowing in the past few years. Many of the larger companies such as AstraZeneca and Pfizer are slashing research budgets in the hope of getting more bang for their buck. And they may be right to be cautious. The number of medicines failing in late development is on the rise. The CMR International report said that the number of drugs failing at phase III has doubled in the period 2007-2009 compared with 2004-2006. There is some positive news. Twenty-six new molecular entities were launched in 2009, compared with twenty-one in 2008. But even this represents only a little more than half of the NMEs hitting the market in 1997. The report also looked at the treatment areas that companies are focusing on. Anti-cancer drugs are receiving almost one fifth of the R&D pie. This is reflected in this edition of The Ones to Watch, with six of the twenty featured drugs being cancer treatments, including three for prostate cancer. But the remaining drugs also reveal diversity in the pipeline, from the Novartis Vaccines Institute for Global Health’s candidate typhoid vaccine, which has just begun phase I testing, to POZEN and AstraZeneca’s Vimovo™ pill to reduce the risk of gastric ulcers in long-term users of arthritis medication. Other notable agents could make treatment easier for patients, including Gilead’s Quad pill formulation of antiretrovirals for HIV infection and Novartis’s inhalable QVA-149 for the treatment of chronic pulmonary obstructive disorder, both entering phase III studies this quarter. Will companies rest on their laurels and rely on the revenue generated by stalwarts of the medicine cabinet? Not likely, judging by the diverse range of products moving through the pipeline in this edition of The Ones to Watch. It may just be that companies pick their battles more carefully, devoting resources to their most promising candidates. Let’s take a closer look at the five most promising drugs launched or receiving approval, and moving through each of the clinical phases, between April and June 2010. For more information on Thomson pharma visit go.thomsonreuters.com/commercial or email scientific.lifesciences@thomsonreuters.compHARMA MATTERS | THE OnES TO WATCH
  • 3. THE FivE MOST pROMiSing dRUgS lAUnCHEdOR RECEiving AppROvAl dRUg diSEASE COMpAnY Votrient® Advanced renal cell GlaxoSmithKline carcinoma Nesina® Type 2 diabetes Takeda Jevtana® Metastatic hormone sanofi-aventis refractory prostate cancer Provenge® Hormone refractory Dendreon prostate cancer Vimovo™ Arthritis POZEN/AstraZenecaKicking off this edition of The Ones to Watch is a once-daily oral treatmentfor advanced renal cell carcinoma (RCC), the most common form of kidneycancer, which is often resistant to chemotherapy. Votrient® (pazopanib),developed by GlaxoSmithKline (GSK), was approved in June 2010 in theEC for advanced RCC, including for patients with advanced RCC whohad received prior cytokine therapy. The drug has been available for thisindication since 2009 in the US.Votrient® is a second-generation tyrosine kinase inhibitor which targetsthe VEGFR tyrosine kinases, an important subfamily of cancer-promotingreceptor tyrosine kinases. Votrient® also inhibits platelet-derived growthfactor receptor and c-kit, other members of the tyrosine kinase subfamilywhich are key proteins responsible for tumor growth and survival. Phase IIItesting showed that Votrient® reduced levels of angiogenic factors in RCCpatients. It will provide physicians with a new treatment for patients withadvanced RCC, many of whom have run out of treatment options.Earlier drugs, such as Iressa® and Gleevec® inhibited individual receptortyrosine kinases, while more recently developed drugs, such as Nexavar®and Sutent®, have emphasized the advantages of inhibiting multiplereceptor tyrosine kinases, and overcoming redundancies in signalingpathways to more effectively inhibit tumor growth. Votrient® is also beinginvestigated for other cancer indications, including sarcoma, non-small-celllung cancer and breast cancer.Our next treatment reaching the market is Nesina® (alogliptin), an oralonce-daily treatment for type 2 diabetes patients who are unable to controltheir disease through diet and exercise alone. Nesina® is a small moleculedipeptidyl peptidase IV (DPP-4) inhibitor and is the only one that can betaken either alone or in combination with an alpha-glucosidase inhibitor.Developed by Takeda, it was launched in Japan in June 2010.DPP-4 is an enzyme that cleaves incretin hormones. By reducing thedegradation of these hormones, Nesina® increases their effects, resultingin increased insulin secretion and suppression of glucagon secretion. Thisimproves patients’ glucose tolerance and lowers blood glucose levels.Returning to cancer therapies, sanofi-aventis (formerly Aventis) hasreceived approval for its prostate cancer treatment, Jevtana® (cabazitaxel).It was approved by the FDA for the second-line treatment of metastatichormone-refractory prostate cancer (mHRPC) in combination with theimmunosuppressant prednisone in June 2010 and has since been launchedin the US. Jevtana® was submitted for approval in the EU in April 2010. pHARMA MATTERS | THE OnES TO WATCH
  • 4. Jevtana® is an intravenously administered derivative of taxol that suppresses the division of cancer cells by mitosis. The Phase III TROPIC study showed that, in combination with prednisone, it increased overall and progression- free survival by 2.4 and 1.4 months respectively compared with existing prostate cancer drug Novantrone® (mitoxantrone) plus prednisone. There had previously been few treatment options available for men with advanced mHRPC – Jevtana is the first to be approved specifically for this indication, having been reviewed under the FDA’s priority review scheme. The approval comes just before the patent for Taxotere®, sanofi-aventis’ existing drug for this indication, expires. Thomson Pharma Partnering Forecast reports consensus estimated sales of $39 million in 2011, rising to $208 million in 2014. Our next newly-approved drug is also for prostate cancer, this time for an earlier stage of the disease. Provenge® (sipuleucel-T), from Dendreon (formerly Activated Cell Therapy), is the first ever vaccine approved to treat cancer and is also the first of a new therapeutic class known as autologous cellular immunotherapies. It is designed to induce an immune response against prostatic acid phosphatase, an antigen expressed in most prostate cancers. Provenge® was launched in the US for the treatment of asymptomatic or minimally symptomatic hormone-refractory prostate cancer in April 2010, after phase III testing showed that it can help some patients live longer. In the trial, men in the Provenge® group lived an average of 4.1 months longer and had a 22.5% reduction in the risk of death compared with men in the control group. Brean Murray Carret & Co predicts blockbuster status for the vaccine, with peak sales reaching $2.0 billion. Supply will take a while to meet expected demand, however, with only enough Provenge available within the next year to treat 2000 patients. New manufacturing sites will be up and running by mid 2011 and should increase supply to treat 8000 patients in 2012. Sales are predicted to rise steeply, with a Thomson Pharma Partnering Forecast consensus of $1.4 billion in 2014. Our final agent ready for the clinic is Vimovo™ (PN-40020) an oral formulation for the treatment of osteoarthritis, rheumatoid arthritis and ankylosing spondylitis, a form of spondyloarthritis. Many arthritis patients control the symptoms of their disease with pain-relieving NSAIDs (non- steroidal anti-inflammatory drugs) but long-term use can lead to gastric ulcers, with 50% of chronic NSAID users said to be at risk. Vimovo™, developed by POZEN and AstraZeneca, received FDA approval in April 2010 for this sub-group of arthritis patients. The delayed-release pill is an enteric-coated combination of the NSAID naproxen and the proton pump inhibitor esomeprazole (Nexium®), which reduces gastric acid production. Phase III trials showed patients taking Vimovo experienced significantly fewer gastric ulcers, with one of the trials showing a 4.1% incidence of gastric ulcer in those who took Vimovo™ compared with 23.1% of patients receiving enteric-coated naproxen alone.pHARMA MATTERS | THE OnES TO WATCH
  • 5. THE FivE MOST pROMiSing dRUgS EnTERing pHASE iii TRiAlS dRUg diSEASE COMpAnY Quad pill HIV infection Gilead Sciences REOLYSIN® Head and neck cancer Oncolytics Biotech BHR-100 Traumatic brain injury BHR Pharma QVA-149 Chronic obstructive Novartis/Vectura/Sosei pulmonary disease Varespladib methyl Acute coronary Anthera syndrome Pharmaceuticals/ Shionogi/Eli LillyWe begin our range of drugs entering the final stage of clinical testingwith Gilead Sciences’ Quad pill comprising the antiretrovirals Truvuda®(emtricitabine plus tenofovir disoproxil fumarate), elvitegravir and the boostingagent cobicistat for the potential once-daily treatment of HIV infection.A phase III trial of the pill began in treatment-naive patients in April 2010in the US and Puerto Rico, with the aim of comparing its efficacy with thatof another anti-HIV combination therapy, Atripla® (tenofovir disoproxilfumarate, emtricitabine and efavirenz). At that time, a second phase III trialwas planned for North America, South America, Europe and Asia Pacific.Promising phase II trial results found the Quad pill to be non-inferior toAtripla. Also, being smaller, it should be easier to swallow than Atripla andhas the added benefit of not requiring refrigeration.Turning to cancer, second in our list is Oncolytics Biotech’s REOLYSIN®, aninjectable reovirus therapy aimed at the treatment of Ras-mediated cancers.In May 2010, a phase III trial of intravenous REOSYLIN plus chemotherapydrugs paclitaxel and carboplatin for head and neck cancer began in the US,UK and Belgium. The agent is also under investigation for a range of cancersincluding prostate cancer, lung-metastasized sarcoma, non-small-cell lungcancer and metastatic ovarian cancer.REOLYSIN® is Oncolytics Biotech’s proprietary formulation of the naturallyoccurring human reovirus. It replicates in and kills only tumor cells withactivated Ras pathways which are involved in a possible two-thirds of allhuman cancers and more than 90% of metastatic cancers. After tumorcell death, progeny virus particles are released and infect surroundingcancer cells, leaving healthy cells unscathed. Preclinical data suggest thatREOLYSIN® works synergistically with chemotherapy to kill many morecancer cells than either alone.There are no approved drugs for traumatic brain injury (TBI), which anestimated 1.7 million Americans experience each year. It is the leading causeof death and disability among children and young adults worldwide andplays a role in almost half of trauma deaths. BHR Pharma is hoping that itsintravenous infusion formulation of progesterone, BHR-100, will meet thisneed. A phase III trial of BHR-100 began in the US in June 2010. pHARMA MATTERS | THE OnES TO WATCH
  • 6. The global phase III SyNAPSe study, in which patients receive a 5-day infusion of either BHR-100 or placebo, follows promising earlier trials in which progesterone demonstrated a mortality benefit and improved functional outcomes in TBI patients. How progesterone does this is not known, though various mechanisms have been posited, including reducing neuronal cell death, inhibiting inflammation by reducing cytokine release and immune cell migration, and inducing the remyelination of nerve cells. In December 2009, BHR-100 received US Orphan Drug status for moderate- to-severe closed-head TBI. Daily life for chronic obstructive pulmonary disease (COPD) patients could be made easier with QVA-149 (NVF-239), Novartis’s new combination of bronchodilators which only needs to be taken once daily. COPD, a chronic disease characterized by mostly irreversible airflow limitation associated with an abnormal inflammation in the lung, affecting 210 million people worldwide is projected to be the third largest cause of death by 2030. There are no cures available for COPD but bronchodilators have been the mainstay for COPD therapy as they reduce bronchoconstriction and improve breathing. Under license from Vectura and Sosei (formerly Arakis), Novartis is developing the once-daily inhaled dry powder combination of glycopyrronium bromide, a bronchodilator and muscarinic ACh antagonist, and indacaterol, a long-acting beta-2 receptor agonist. A phase III study began in May 2010 in severe or very severe COPD patients comparing QVA- 149 with glycopyrronium bromide alone. Encouraging data from the phase II trials in moderate to severe COPD patients showed that combining the medications was safe and effective. The last drug transitioning to phase III trials in this edition of The Ones to Watch is an oral treatment for coronary artery diseases including acute coronary syndrome (ACS). Varespladib methyl (A-002) is being developed by Anthera Pharmaceuticals, under license from Shionogi and Eli Lilly. It is a methyl ester of varespladib, a potent and highly selective inhibitor of the pro-inflammatory enzyme, secretory phospholipase A2 (sPLA2), elevated levels of which have been implicated in a range of diseases, including ACS. In Anthera’s phase II study in ACS patients, varespladib demonstrated marked improvements in markers of cardiovascular risk. A global phase III trial in high-risk ACS patients – those at risk of further coronary events – began in June 2010, aiming to test the hypothesis that reducing inflammation in ACS patients will improve outcomes by reducing further coronary events, for example. Anthera hopes varespladib will reduce inflammation more than current statin therapies but also believes that varespladib will work synergistically with them. Patients who received both varespladib and statins in the phase II trial had lower LDL cholesterol levels than those who were given varespladib alone. Early data suggest varespladib will also work alongside other cardiovascular drugs such as niacin.pHARMA MATTERS | THE OnES TO WATCH
  • 7. THE FivE MOST pROMiSing dRUgS EnTERingpHASE ii TRiAlS dRUg diSEASE COMpAnY Affitope AD-02 Alzheimer’s disease AFFiRiS/ GlaxoSmithKline Biologicals ACE-031 Duchenne muscular Acceleron dystrophy CB-183315 Clostridium difficile Cubist Pharmaceuticals infection NNZ-2566 Brain injury Neuren ganglioside vaccine Sarcoma Sloan Kettering/ MabVaxOur first drug entering phase II testing is Affitope AD-02, a potentialAlzheimer’s disease (AD) vaccine from AFFiRiS and licensee GSKBiologicals. The peptide-based vaccine targets β-amyloid 40-42 – a majorcomponent of amyloid plaques that are a feature in AD patients’ brains –and is based on AFFiRiS’s proprietary AFFITOME® technology. A Europeanphase II trial began in April 2010 after reports of favorable safety andtolerability of subcutaneous injections in phase I trials.Alzheimer’s disease is predicted to affect 1 in 85 people by 2050. It has fewtreatments, and those that do exist relieve symptoms only marginally.The vaccine is AFFiRiS’s first program to reach phase II trials, and will be akey test of the validity of its AFFITOME® technology. AFFITOME® is basedon molecular mimicry of target molecules, which AFFiRiS hopes will lead tobetter safety by fine-tuning the immune response to only neoepitopes whichoccur on pathological molecules, rather than healthy precursor proteins.In addition, because the antigen is foreign, there is no need for the vaccineto break immune tolerance. And if Affitope AD-02 does not perform asexpected, further AFFITOME® peptides can be tried.Next up is ACE-031 from Acceleron, a myostatin (GDF-8) inhibitor for thepotential subcutaneous treatment of muscle wasting and weakening causedby cachexia, muscular dystrophy and amyotrophic lateral sclerosis. A phaseII study began in children with Duchenne muscular dystrophy (DMD) inApril 2010 after an encouraging phase I trial showed an average increase inmuscle of 1 kilogram in healthy adults two weeks after administration.ACE-031 is a recombinant fusion protein made up of a portion of the humanactivin receptor type IIB (ActRIIB) and a portion of a human antibody. Itinhibits signalling through ActRIIB by acting as a ‘decoy’ version of ActRIIB,removing myostatin and other proteins that limit muscle growth. pHARMA MATTERS | THE OnES TO WATCH
  • 8. DMD occurs once in every 3,500 live births and has no approved treatment. Affecting mainly boys, it is characterized by loss of muscle strength and function caused by a lack or defect in the dystrophin protein, an integral part of muscle fibers. Heart and respiratory muscles weaken and eventually fail – few patients live beyond their late twenties. Turning to infectious diseases, Cubist Pharmaceuticals is hoping that the oral lipopeptide antibiotic CB-183315 will become only the second FDA-approved treatment for diarrhea associated with Clostridum difficile infection. In April 2010, Cubist announced the initiation of phase II trial in the US and Canada comparing CB-183315 with Eli Lilly, Shionogi and ViroPharma’s Vancocin® (oral vancomycin), the only existing approved treatment. However, Vancocin® is relegated to the second-line treatment for severe and relapsed cases because of concerns over the development of antibiotic resistance and the drug’s high cost. The antibacterial is the first product from Cubist’s own drug discovery work to have reached phase II. New treatments for C. difficile are crucial in light of increasing rates of infection, particularly of an emerging highly virulent strain for which Vancocin and oral metronidazole – used to treat C. difficile off-label – are inadequate. Our next agent is another potential treatment for traumatic brain injury. An intravenous formulation of Neuren’s NNZ-2566, a synthetic analog of a neuroprotectant produced by the brain in response to injury, went into phase II trials in April 2010, in collaboration with the US army. The naturally occurring molecule that NNZ-2566 mimics is a small part of the IGF-1 protein and belongs to the class of neuropeptides, which have broad activities and few side effects. Previous research into brain injury treatment has focused on agents that act on just one or two of the cellular pathways that are stimulated by brain injury and cause cell death. NNZ- 2566 has been shown in animal models to act across a range of pathways and reduce expression of genes involved in inflammation, necrosis and apoptosis, as well as reducing the frequency of post-injury non-convulsive seizures, which cause permanent damage to the brain. The drug has Fast Track status from the FDA and Neuren hopes sales of NNZ-2566 could reach over $2 billion in the US alone in the first 10 years. The last agent entering phase II testing in this edition of The Ones to Watch is a candidate vaccine for recurrent sarcoma. Developed by MabVax, under license from the Memorial Sloan-Kettering Cancer Center, the polyvalent ganglioside vaccine targets the GM2, GD2 and GD3 gangliosides which are displayed on the surface of cancer cells. In June 2010, a phase II study was initiated in patients with metastatic sarcoma, with patients receiving either the vaccine alone or in combination with the immunostimulant OPT-821. Sarcomas have poor survival rates and often recur, particularly osteosarcomas, rhabdomyosarcomas, and other non-rhabdomyosarcomas that most often occur in young adults. The antibody vaccine is novel in that it targets multiple gangliosides, rather than a single antigen. Chemotherapy and radiotherapy have only limited success in clearing residual circulating cancer cells, and MabVax is hoping that targeting multiple antigens will mop up remaining cells. In addition, sarcomas share many antigens with neuroblastoma and melanoma, so a single product could treat all three types of cancer.pHARMA MATTERS | THE OnES TO WATCH
  • 9. THE FivE MOST pROMiSing dRUgS EnTERing pHASE i TRiAlS dRUg diSEASE COMpAnY TRV-120027 Acute heart failure Trevena/Ligand Pharmaceuticals bivalent CRM-197 Typhoid and enteric Novartis Vaccines diphtheria toxoid- fever Institute for Global conjugated vaccine Health MK-5172 Hepatitis C virus Merck infection SL-052 Prostate cancer Quest PharmaTech/ SonoLight ALN-TTR01 Transthyretin-mediated Alnylam amyloidosis Pharmaceuticals/Isis PharmaceuticalsThe first of this edition’s drugs entering phase I trials is Trevena’s TRV-120027,the first biased ligand targeting G-protein-coupled receptor (GPCR) signalingpathways associated with beneficial effects to be tested in humans. Theintravenously administered drug is indicated for acute heart failure, which hasfew safe and effective treatments, mortality rates of 20-30% and accounts foraround one million hospitalizations in the US each year.TRV-120027 is the first in a new class of drugs that target GPCRs in amore specific manner. The GPCRs are a receptor family targeted byaround 40% of all modern medicinal products. Uniquely, TRV-120027blocks angiotensin-mediated G-protein signaling while simultaneouslystimulating signaling through AT1R-specific β-arrestin. This interferes withthe functioning of AT1R (angiotensin II type 1 receptor) – which plays animportant role in the pathophysiology of acute heart failure. In preclinicalanimal studies, TRV-120027 improved several key pathologies associatedwith acute heart failure.Trevena, which licensed the drug from Ligand Pharmaceuticals, beganphase I trials in healthy patients in April 2010.Next is a bivalent vaccine against the Salmonella enterica serovars Typhi (S.Typhi) and Paratyphi A (S. Paratyphi A) from the Novartis Vaccines Institutefor Global Health (NVGH). The CRM-197 diphtheria toxoid-conjugatedvaccine is intended to prevent typhoid, which is caused by S. Typhi, andenteric fever, caused by S. Paratyphi A. A phase I trial for S. Typhi comparingthe safety and immunogenicity of the vaccine with that of GSK’s Typherix®began in May 2010 on healthy adults in Belgium.Typhoid affects around 21 million people each year, killing 600,000, largely inthe developing world. NVGH hopes that the vaccine will one day eradicate thedisease. Available vaccines for S. Typhi do not protect infants and young childrenand S. Paratyphi A is a growing problem in Asia and has no vaccine at all.Staying with infectious diseases, Merck & Co, announced in April 2010that it had initiated phase I trials of MK-5172, a potential oral treatment forhepatitis C virus (HCV) infections resistant to first-line treatment. MK-5172is a second-generation HCV NS3/4a protease inhibitor which has shownimpressive resistance characteristics in preclinical tests against a rangeof HCV mutants in comparison with MK-7009, another Merck proteaseinhibitor in clinical development. It has also proved more effective atreducing viral load in infected chimpanzees. pHARMA MATTERS | THE OnES TO WATCH
  • 10. MK-5172 could provide second-line treatment for infections becoming resistant to other phase III NS3 protease inhibitors such as Vertex and Johnson & Johnson’s telaprivir and Merck & Co’s boceprevir. HCV affects 170 million people worldwide, leads to cirrhosis and liver cancer and is the primary cause of the need for liver transplant in Europe and the United States. Resistance to antivirals emerges rapidly and only around half of patients have infections that are treated sustainably. Surgery and radiation for prostate cancer often damage healthy tissue, leading to poorer quality of life post-treatment. Photodynamic therapy – where a drug becomes activated by illumination once it is in the desired location – has the potential to reduce such side effects. Quest PharmaTech (formerly Altachem Pharma), through its subsidiary SonoLight, is developing SL-052, a synthetic, injectable derivative of hypocrellin, a polycyclic quinone produced by a parasitic fungus that grows on bamboo in China. It is activated by sonodynamic or photodynamic therapy to form oxygen radicals that kill tissues. A phase I trial using SL-052 in photodynamic therapy for prostate cancer began in May 2010 after preclinical studies in canine models showed the drug could be delivered selectively to the prostate and destroy tumors. SL- 052 is also under investigation for lung cancer. Rounding off this edition of The Ones to Watch is a potential treatment for transthyretin-mediated amyloidosis (ATTR) a rare, hereditary disease affecting just 50,000 patients worldwide which leads to familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy. ATTR is caused by mutations in the gene for the protein transthyretin, which is produced in the liver. This results in the toxic accumulation of both mutated and normal protein in many tissues, including the peripheral nervous system, heart, and the gastrointestinal tract. Sufferers live around 5-15 years after the onset of the disease and the only treatment is a liver transplant. To meet this clinical need, Alnylam Pharmaceuticals and Isis Pharmaceuticals are developing ALN-TTR01, a siRNA therapy targeting the TTR gene. The drug is delivered using Tekmira Pharmaceuticals’ stable nucleic acid-lipid particles. A phase I trial for amyloidosis began in June 2010 after encouraging preclinical research showed the prevention and regression of TTR deposits from tissues including dorsal root ganglia, sciatic nerve, stomach and intestines. ALN-TTR01 treatment of non-human primates resulted in the dose-dependent and sustained, yet reversible, silencing of the TTR gene and levels of TTR in serum. The companies are also investigating the drug for the potential treatment of type 2 diabetes.pHARMA MATTERS | THE OnES TO WATCH
  • 11. To sign up to our pharma Matters range of publications visit: go.thomsonreuters.com/pharmamatters THE OnES TO WATCH Focuses on the latest phase changes in the pharmaceutical pipeline. MOvERS And SHAKERS Unravels the most significant game-play in the US generics market. THE CUTTing EdgE OF CHEMiSTRY insights into the chemistry advances transforming drug discovery and development. AbOUT THOMSOn pHARMA® Thomson Pharma brings together the best pharmaceutical data owned by Thomson Reuters in a single comprehensive solution containing millions of pieces of information. And it’s not just data. Thomson Pharma extends and deepens its knowledge with unique abstracts, commentaries and analysis prepared by our team of industry experts. You can link at a click between different types of content. no other data source puts so much information at your fingertips. in place of your legacy indexing systems, multiple interfaces, and complex data sources, imagine how Thomson Pharma can simplify your information needs, justify and speed your decision-making, and keep you abreast of the market. AbOUT THOMSOn REUTERS Thomson Reuters is the leading source of intelligent information for professionals around the world. Our customers are knowledge workers in key sectors of the global economy. We supply them with the intelligent information they need to succeed in fields that are vital to developed and emerging economies such as law, financial services, tax and accounting, healthcare, science and media. Our knowledge and information is essential for drug companies to discover new drugs and get them to market faster, for researchers to find relevant papers and know what’s newly published in their subject, and for businesses to optimize their intellectual property and find competitive intelligence. nOTE TO pRESS: To request further information or permission to reproduce content from this report, please contact: paul Sandell phone: + 44 20 7433 4704 Email: paul.sandell@thomsonreuters.com For more information on Thomson Pharma and Thomson Pharma Partnering visit: go.thomsonreuters.com/commercial or email scientific.lifesciences@thomsonreuters.comhealthcare & Science regional officesNorth Americaphiladelphia +1 800 336 4474 +1 215 386 0100Latin America +55 11 8370 9845Europe, Middle East and Africalondon +44 20 7433 4000Asia PacificSingapore +65 6775 5088Tokyo +81 3 5218 6500For a complete office list visit:science.thomsonreuters.com/contactph1003237Copyright © 2010 Thomson Reuters

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