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  • Aegerion Pharmaceuticals Expects Little Growth In 2015 [View article]
    Stick a fork in it.
    Mar 15, 2015. 09:46 PM | Likes Like |Link to Comment
  • What To Make Of The Chaos At Acadia [View article]
    Worst management ever? Lots of bad management in biotech history, but should be nominated. No excuse for what happened including the delay in reporting after saying everything was hunky dory a month ago. Will trade at a lack of credibility discount for a long time.
    StockNews @StockNews_ · 12h 12 hours ago
    $ACAD - Harwood Feffer LLP Announces Investigation of Acadia Pharmaceuticals Inc.
    Mar 13, 2015. 08:18 AM | Likes Like |Link to Comment
  • Sarepta Is Suffering From Overreaction [View article]
    I think Golabki hit the nail on the head. Extended and larger trials are most likely.
    Jan 15, 2015. 12:04 PM | Likes Like |Link to Comment
  • Update: Richmont Is Betting On Its Island Gold Mine - And It Will Very Likely Pay Off [View article]
    Thanks for the update. This one looked like it going sub zero last year when it crashed.
    New found money!
    Jan 9, 2015. 01:51 PM | Likes Like |Link to Comment
  • The 2015 Calendar Of NASH Trial Catalysts: Gilead Adds To Its NASH Arsenal [View article]
    ISIS also has an early stage drug for NASH. Never count them out. And a NASH partnership.

    Isis Pharmaceuticals shares rise 2.8% after company raises outlook

    (7:18 AM ET) NEW YORK (MarketWatch) -- Isis Pharmacueticals Inc. ISIS, +2.27% said Thursday it expects to "significantly" improve on its 2014 guidance, ending the year with a pro forma net operating loss in the mid to high teens and more than $725 million cash. That is a nearly 70% improvement over its original guidance of a pro forma loss in the low $50 million range. The improvement is due to the maturation of its drug pipeline "and the growing validation of antisense as a viable, efficient and effective drug discovery technology," the company said in a statement. Isis' pipeline comprises 33 drugs to treat a range of diseases with a focus on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. Shares rose 2.8% in premarket trade, and have gained 75.3% in the last three months, while the S&P 500 has gained 2.9%.
    Jan 8, 2015. 10:03 AM | 1 Like Like |Link to Comment
  • Isis - Addressing Some Of The Bull Arguments To My Short Thesis [View article]
    Market doc has never been heard from since. His career as a financial writer ended appropriately with this specious article. He deserves all the blame and ignominy that befalls him.

    ISIS hit an all time high today.
    Dec 11, 2014. 10:05 PM | 1 Like Like |Link to Comment
  • Isis - Addressing Some Of The Bull Arguments To My Short Thesis [View article]
    Hope no one listened to this pretend doctor. ISIS up almost 100% since this non scientifically based short hack job.
    Dec 11, 2014. 09:58 AM | 3 Likes Like |Link to Comment
  • Regulus To See Further Declines [View article]
    This is not the only drug in development as you stated. I would not want to be short when the next data point comes out (multi dosing at higher dose). One dose of the Regulus drug versus how many for Harvoni? 84! Double that with cirrhosis. Not a very fair comparison. Insurance companies would love the cheaper drug.
    Nov 19, 2014. 08:27 PM | 1 Like Like |Link to Comment
  • Regulus To See Further Declines [View article]
    Characterization of the in vitro antiviral activity and the preclinical and clinical resistance profile of miravirsen, a novel anti-HCV therapeutic targeting the human factor miR-122.
    Ottosen S1, Parsley TB2, Yang L2, Zeh K3, van Doorn LJ4, van der Veer E4, Raney AK3, Hodges MR3, Patick AK5.
    Author information
    Miravirsen is a β-D-oxy-Locked Nucleic Acid modified phosphorothioate antisense oligonucleotide targeting the liver-specific microRNA-122 (miR-122). Miravirsen demonstrated antiviral activity against HCV genotype 1b replicons with a mean 50% effective concentration (EC50) of 0.67 μM. No cytotoxicity was observed up to the highest concentration tested (>320 μM) in different cell culture models yielding a therapeutic index of ≥297. Combination studies of miravirsen with interferon-α2b, ribavirin, non-nucleoside (VX-222) and nucleoside (2' -methylcytidine) inhibitors of NS5B, NS5A (BMS-790052), or NS3 (telaprevir) indicated additive interactions. Miravirsen demonstrated broad antiviral activity when tested against HCV replicons resistant to NS3, NS5A and NS5B inhibitors with less than 2-fold reductions in susceptibility. In serial passage studies, an A4C nucleotide change was observed in the 5' HCV UTR from cells passaged in the presence of up to 20 μM (40-fold the miravirsen EC50 concentration) at day 72 of passage but not at earlier time points (up to 39 days of passage). Likewise, a C3U nucleotide change was observed in the HCV 5' UTR from subjects with viral rebound after the completion of therapy in a miravirsen Phase 2 clinical trial. An HCV variant constructed to contain the A4C change was fully susceptible to miravirsen. A C3U HCV variant demonstrated overall reductions in susceptibility to miravirsen but was fully susceptible to all other anti-HCV agents tested. In summary, miravirsen has demonstrated broad antiviral activity and a relatively high genetic barrier to resistance. Identification of nucleotides changes associated with miravirsen resistance should help further elucidate the biology of miR-122 interactions with HCV. (The clinical trial study has been registered at under registration no. NCT01200420).

    Copyright © 2014, American Society for Microbiology. All Rights Reserved.
    Nov 19, 2014. 07:57 PM | Likes Like |Link to Comment
  • Regulus To See Further Declines [View article]
    Results for one dose were excellent and it was the lower dose. Company never claimed one dose cure, nor anyone else of any standing or affiliation, but it may end up being the quickest cure. It's also possible it could be used in conjunction with another drug.
    Nov 19, 2014. 01:17 PM | 1 Like Like |Link to Comment
  • Regulus To See Further Declines [View article]
    For one ISIS sold only aprox 20% of their 7.1 million shares. Which always makes sense when you are funding a huge pipeline. You can debate market value after huge run up but remember this is a platform technology. MiR-122 will prove to be an extremely viable target.

    miR-122 Findings from Mt. Sinai

    >>Nov 18, 2014
    Hepatitis C Antiviral Resistance Revealed

    Scientists at the Icahn School of Medicine at Mount Sinai (ISMMS) say recent research has shed light on the response of the hepatitis C virus (HCV) to targeted therapeutics and provided new insights about HCV’s role in cancer development. Their work (“Hepatitis C virus genetics affects miR-122 requirements and response to miR-122 inhibitors”), published in Nature Communications, focused on microRNA genes, a type of regulatory gene, and used whole-genome sequencing of the virus to challenge conventional wisdom about how the virus responds to emerging therapies.

    Their findings, note the researchers, may contribute to more effective development of hepatitis C drugs in the future and to more personalized treatment for patients.

    According to the Centers for Disease Control and Prevention, HCV is widespread, affecting some 3% of the world’s population and more than 3 million people in the United States alone. Recent CDC reports indicate that hepatitis C infections are on the rise among young people and are increasingly the cause of death among baby boomers.

    The vast majority of people who get HCV will suffer chronic infection, which can lead to liver inflammation, cirrhosis, and liver cancer. Highly effective new treatments have been launched recently, but their high prices have caused public outcry and limited widespread use. There is no commercially available vaccine for hepatitis C.

    In this new study, Mount Sinai researchers examined HCV response to an experimental treatment that targets and blocks the supply of a microRNA (miR-122) that the virus needs for infection of human cells. Contrary to expectations, they found that depleting the supply of miR-122 could trigger drug resistance with the emergence of HCV strains able to infect cells with negligible levels of the microRNA. This information could be used for more effective dosing of drugs targeting this gene, as well as for pre-treatment analysis to determine which patients may respond best to this class of drugs.

    “This effort, which was made possible by innovative microRNA analysis, offers significant progress toward precision medicine in treating HCV patients,” said Matthew Evans, Ph.D., assistant professor of microbiology at the ISMMS and a co-author of the study. “There is a critical need for more weapons in our arsenal to fight HCV, particularly for affordable, effective treatment as we try to stay a step ahead of this virus and prevent it from developing the kind of drug resistance we’re seeing in the bacterial realm.”

    In another key finding, the scientists uncovered knowledge that may help answer the longstanding question of how HCV leads to cancer. The study demonstrated that HCV hijacks the miR-122 gene, diminishing its normal activity in liver cells. Since this microRNA is known to be a potent tumor repressor, it is possible that HCV robs cells of their natural defenses against uncontrolled growth. Such an outcome could contribute to cancers that arise from chronic HCV infection.

    “We found that HCV itself reduces miR-122’s activity in the cell, possibly through binding and sequestering miR-122,” wrote the investigators. “Our study provides insight into the interaction between miR-122 and HCV, including viral adaptation to reduced miR-122 bioavailability, and has implications for the development of anti-miR-122-based HCV drugs.”

    “Our study offers broader implications for this class of microRNA genes and their interaction with targets, which may be useful for a number of diseases in addition to hepatitis C,” said Brian Brown, Ph.D., associate professor of genetics and genomic sciences at the ISMMS and a co-author of the study. “We are also intrigued by this new information that may shed light on the link between HCV and the onset of cancer and look forward to future efforts to explore this theory.”<<
    Nov 19, 2014. 12:46 PM | Likes Like |Link to Comment
  • Genzyme and Isis Pharma announce presentation on cardiac benefits of Kynamro [View news story]
    MACE is the place! Results are unequivocal .
    Nov 18, 2014. 07:03 PM | 1 Like Like |Link to Comment
  • Isis earns milestone payment from GSK [View news story]
    It's the 4th drug with GSK and the 2nd undisclosed ocular drug. Undisclosed because ALNY and other RNA companies like to have ISIS do their drug target discovery for them.
    Nov 3, 2014. 12:23 PM | Likes Like |Link to Comment
  • Aegerion Pharmaceuticals misses by $0.06, misses on revenue [View news story]
    Things will get worse. ISIS AHA oral presentation - Decreased Cardiovascular Events in Familial Hypercholesterolemic Patients Treated With Mipomersen
    Sanofi reps are out in force.
    Oct 30, 2014. 04:38 PM | Likes Like |Link to Comment
  • Richmont Mines Has Tremendous Upside Potential Because Of Island Gold Deep [View article]
    Haven't read it all yet but excellent overview. Well done!
    Oct 30, 2014. 12:56 PM | Likes Like |Link to Comment