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truth_told 22 posts  |  Last Activity: Jun 23, 2015 9:01 PM Member since: Jul 18, 2001
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  • truth_told by truth_told Jun 23, 2015 9:01 PM Flag

    Glad that you're still here...
    TT

  • Phase III Trial Ruins Prosensa's Drisapersen
    Sep. 23, 2013 9:30 AM ET | Includes: RNA, SRPT

    Last Friday, an early top-line Phase III data release for Duchenne Muscular Dystrophy (DMD) oligonucleotide therapeutic drisapersen devastated shares of Dutch biotech Prosensa (RNA). After a 70% drop in share price, Prosensa's valuation stands at $207 MM. Prosensa's partner for the development of drisapersen is GlaxoSmithKline (GSK) - and fortunately for GSK shareholders the company is too large to be affected by a small clinical trial failure.

    The Phase III trial - DMD114044, or DEMAND-III -was a 186-patient randomized, double-blind, placebo-controlled study that tested the DMD drug at a 6mg/kg/week dose in 125 patients. The placebo arm contained the other 61 patients. Patients were assessed over a 48-week period.

    The primary endpoint, designed to measure the efficacy of drisapersen, was based on improvements in Six Minute Walk Distance (SMWD) between the 6mg/kg/week drisapersen arm and the control group. The company was specifically looking for a 30+ meter improvement, which would allow the company to report statistically significant improvements in walking distance with 90% power. Unfortunately, drisapersen fell short by quite a bit.

    The difference in 6MWD (mean (CI) 10.33m (-14.65, 35.31), p=0.415) between drisapersen and placebo groups did not reach statistical significance.

    With a p value that large, Prosensa/GSK cannot use this for FDA approval since the drug has not formally established its efficacy as a DMD treatment. In addition to this, we note that the toxicity remains a serious issue that mars the safety profile of the drug. Drisapersen nearly doubled rates of renal adverse events (proteinuria), and caused an injection site reaction in 78% of drisapersen arm patients versus 16% in the placebo arm.

  • Reply to

    Read it and weep shorts...

    by truth_told Jun 22, 2015 11:41 PM
    truth_told truth_told Jun 22, 2015 11:48 PM Flag

    Shorties... I know the truth hurts..accepting truth is what you need to do. "Denial" is tough but if you face reality and "move on" you will be much better in the end.

    Cover my friends or the hammering of facts will continue.

  • "On 20 September 2013, GlaxoSmithKline (GSK) and Prosensa announced that GSK’s Phase III clinical trial (NCT01254019) of Drisapersen, an exon skipping drug for Duchenne muscular dystrophy (DMD), failed to meet the primary endpoint of a statistically significant improvement in the 6 Minute Walking Distance Test (6MWT) compared to placebo.

    Clearly, one major difference between the two chemistries of exon skipping clinic trials is that Eteplirsen has been administrated systemically at up to 50 mg/kg, more than eight times higher than Drisapersen. The limited dose of 6 mg/kg for Drisapersen was largely the result of kidney toxicity whereas no clear toxicity has so far been identified for Eteplirsen. Most preclinical studies have reported higher efficiency of exon skipping with the PMO chemistry than with equivalent amounts of reagent based on the 2OMePS chemistry.4,5,6 It is therefore not surprising that the Eteplirsen trial reported detectable dystrophin by western blot. The Eteplirsen treated patients also showed a benefit of 67 m less decline in 6MWT over the placebo group.16 Stabilization of the motor function in DMD boys participating in Eteplirsen extension study has now being observed over 2 years. "

  • "Glaxo has disclosed some safety data from early-stage drisapersen clinical trials previously, mainly related to moderate cases of proteinuria (excess protein in the urine) and decreased thrombocytes, which play a role in blood clotting. However, the significance of the adverse events attributed to drisapersen, also known as GSK-2402968, escalated with Sunday's disclosure of hospitalized patients.

    Dr. Rohit Batta, global medical leader in Glaxo's neuromuscular rare disease unit, said four Duchenne muscular dystrophy (DMD) patients treated with drisapersen required hospitalization due to thrombocytopenia and that "several" patients with "severe proteinuria" also required hospitalization.

    Batta's remarks were made during a presentation on Sunday, Feb. 24 at The XI International Conference on Duchenne Muscular Dystrophy. The conference was held in Rome and organized by Duchenne Parent Project Onlus, an Italian DMD advocacy group.

    Batta provided no other information about the hospitalized drisapersen patients during his presentation. A Glaxo spokesperson contacted Wednesday confirmed Batta's remarks and added the DMD patients hospitalized were all enrolled in the ongoing phase II and phase III clinical trials of drisapersen. The hospitalizations had occurred "over the last two years" and had been discussed at previous medical meetings.

    Thrombocytopenia is a condition caused by a significant decrease in the number of platelets, which control blood clotting. Patients with thrombocytopenia are at increased risk for uncontrolled bleeding. Proteinuria is the presence of excess protein in urine. Severe proteinuria can be a sign of kidney damage.

  • truth_told truth_told Jun 22, 2015 11:35 PM Flag

    If I was looking into a crystal ball, it would tell me that you have not covered yet.

    If you were looking into a crystal ball, it would tell you how stupid you were not to cover at 12.

  • truth_told truth_told Jun 22, 2015 11:33 PM Flag

    I guess being confined to a wheel chair and dying is better.. which is what would happen to them if they were on Drisa.

    Keep on posting.. just shows how DESPERATE you are.

  • truth_told truth_told Jun 22, 2015 11:31 PM Flag

    absolutely hysterical.

    After you lose your house, please setup a fund so what we can help you out.

  • "The drug did not fail the trial, the trial failed the drug..."

    PS: Whenever you associate a drug with the words "fail" and "failed" in the same sentence... you're out of excuses.

  • They are scampering like rats.. lying like rugs... and have leg less arguments.

    Every day is a new low and a new lie... utterly pathetic

    Words of wisdom for the shorts and Drisa supporters: Sell your shares, beg for forgiveness, tell the Lord your sins and invest in SRPT.

  • 1) Toxic
    2) Toxic
    3) Toxic

    Where are the mothers and patients making movies about Drisa? Where are the investigators claiming Drisa is a "game changer".... ala Etep?

    We know that 100 out of 100 kidneys prefer Etep. How many kids on Etep were hospitalized for adverse reactions due to Etep?

    "0"

    Anyone want to share the number of hospitalizations for kids on Drisa?

    The last thing anyone would want to do is put that useless poison into the veins of a dying kid.

  • Reply to

    De-linking Adcoms...

    by bionerd51 Jun 12, 2015 7:21 AM
    truth_told truth_told Jun 13, 2015 8:14 AM Flag

    The best can be said is that you're no expert.

    Kids are walking, running, skiing & skating that theoretically should be in a wheelchair. Whether ETEP slows, cures whatever.. it's awesome when you're the patient/parent.

  • truth_told truth_told Jun 7, 2015 10:23 PM Flag

    If "safety" is first..then Drisa will never get approved, 100 out of 100 kidneys agree. Additionally, no DMD parent would want their child on it, especially if ETEP is available.. why the heck would you?

  • Reply to

    10 out of 10 kidneys

    by truth_told May 20, 2015 10:30 PM
    truth_told truth_told May 20, 2015 10:45 PM Flag

    Ludicrous?

    Duchenne muscular dystrophy patients treated with GlaxoSmithKline's (GSK - Get Report) experimental drug drisapersen have been hospitalized due to kidney toxicity and low platelet counts, according to a Glaxo scientist who spoke at a research meeting in Rome last Sunday.

    Glaxo has disclosed some safety data from early-stage drisapersen clinical trials previously, mainly related to moderate cases of proteinuria (excess protein in the urine) and decreased thrombocytes, which play a role in blood clotting. However, the significance of the adverse events attributed to drisapersen, also known as GSK-2402968, escalated with Sunday's disclosure of hospitalized patients.

    Dr. Rohit Batta, global medical leader in Glaxo's neuromuscular rare disease unit, said four Duchenne muscular dystrophy (DMD) patients treated with drisapersen required hospitalization due to thrombocytopenia and that "several" patients with "severe proteinuria" also required hospitalization.

    Batta's remarks were made during a presentation on Sunday, Feb. 24 at The XI International Conference on Duchenne Muscular Dystrophy. The conference was held in Rome and organized by Duchenne Parent Project Onlus, an Italian DMD advocacy group.

    Batta provided no other information about the hospitalized drisapersen patients during his presentation. A Glaxo spokesperson contacted Wednesday confirmed Batta's remarks and added the DMD patients hospitalized were all enrolled in the ongoing phase II and phase III clinical trials of drisapersen. The hospitalizations had occurred "over the last two years" and had been discussed at previous medical meetings.

    Thrombocytopenia is a condition caused by a significant decrease in the number of platelets, which control blood clotting. Patients with thrombocytopenia are at increased risk for uncontrolled bleeding. Proteinuria is the presence of excess protein in urine. Severe proteinuria can be a sign of kidney damage.

  • truth_told by truth_told May 20, 2015 10:30 PM Flag

    10 out of 10 kidneys voted for Etep or Drisa.

  • Reply to

    This is a $350 stock... no questions asked

    by truth_told May 20, 2015 9:50 PM
    truth_told truth_told May 20, 2015 10:28 PM Flag

    Exactly.

  • Sarepta Therapeutics Inc (NASDAQ: SRPT) gained more than 60 percent Wednesday on news that it expects to complete an approval application for its lead drug candidate by mid-2015.

    The company's shares closed at $26.44, up $10.06.

    Sarepta is likely to go "significantly higher over the next several months" according to Baird's Brian P. Skorney, who raised his price target 70 percent to $34, maintaining an Outperform rating.

    Skorney said "a sentiment shift/short squeeze" will result as investors come to realize that a regulatory decision in the autumn will determine whether "the stock ends the year sub $10 or over $100."

    Canaccord's Adam Walsh boosted his target more than 84 percent to $30 and upgraded Sarepta to Buy from Hold.

    While significant approval risks remain for the drug, Walsh expects "powerful advocacy groups" may sway regulators in its favor.

    The drug, called eteplirsen, is intended to treat a genetic for of muscular dystrophy that affects children.

    "It's not a question of if, but a question of when" the drug gets approved, Walsh said.

  • Drisa is toxic - poison to be exact. Where is the stunning efficacy data?

    Etep is the only viable DMD drug here - period - end of discussion.

  • Reply to

    This is a $4B-$6B company in 18-24 months

    by truth_told May 19, 2015 9:21 PM
    truth_told truth_told May 19, 2015 9:24 PM Flag

    No but I need help for my tens of thousands of shares.. when I eventually sell.. I'll need a U-Haul to store the loot.

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