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Sarepta Therapeutics, Inc. Message Board

  • zwerp2000 zwerp2000 Mar 1, 2013 2:22 PM Flag

    Part 2

    On October 3rd, 2012, Sarepta Therapeutics released the first 48 weeks of results from a Phase II 12 patient study based out of Nationwide Children’s Hospital in Columbus, Ohio. These 12 boys, ranging in ages 7 through 13, all carried the defective exon 51 gene. Of the twelve, 4 patients received placebo, 4 received 30 mg/kg of eteplirsen, and the final 4 patients received 50 mg/kg of eteplirsen once weekly for 24 weeks.
    The results were shocking. Muscle biopsies done at weeks 12 and 24 showed that the treated population gained dystrophin-positive fibers within the muscle comprising a 47.0% improvement from baseline dystrophin levels. The treated group was thus able to significantly outperform in the trial’s stated primary clinical endpoint – a standardized 6 minute walking test (6MWT). In the 6MWT, the treated group’s out-performance became more significant as time went on. The results from both the muscle biopsies and the 6MWT were both significant enough to convince the oversight committee to ‘unblind’ the trial midway and ensure that the placebo group would get the benefit of eteplirsen as well.
    Although the patient size was extremely small, the results were so astoundingly different between the placebo and eteplirsen groups as to be extremely statistically significant. Statistical significance in medical trials is known as a ‘p-value’, which is a statistical tool that estimates the probability that results are due to chance. The p-value in the combined 30mg & 50mg cohorts was less than 0.001, virtually ruling out randomness as a reason for the trial’s success.
    As exciting as the statistical significance of the primary endpoints was the clean safety data generated throughout the trial. No clinical treatment-related adverse events were observed and no patients were discontinued treatment due to any serious adverse events. This is especially important since a competing treatment currently in trials (Glaxo’s drisapersen) recently saw a number of trial participants hospitalized due to kidney toxicity and low platelet counts.
    The succesful Phase II was not the first time eteplirsen was tested on humans. Since Sarepta began developing the drug, it has been tested in 38 DMD patients across 4 studies. 28 out of 36 DMD patients treated with eteplirsen who received muscle biopsies showed increased dystrophin production (% positive fibers) from baseline, including 20 out of 20 who received at least 10mg/kg/wk (From page 18 of January 2013 presentation)
    In December Sarepta released updated data from the trial, which followed all the patients through week 62. Patients treated with eteplirsen for 62 weeks maintained a statistically significant benefit on the primary clinical endpoint (6MWT), compared to patients who received placebo for 24 weeks and then received eteplirsen for the remaining 38 weeks. Shortly thereafter Lazard raise Sarepta $125 million in a stock offering priced at $25.25 per share. Combined with the money raised from the green shoe, the company ended 2012 with more than $180 million in cash, more than enough funds in the bank to see eteplirsen to market.
    Historically, drug companies like Sarepta would need to plan a Phase III study comprising far more patients than the Phase II before approaching regulators to file an NDA (New Drug Application). However, in the case of young boys reaching puberty and just beginning to suffer the life-threatening effects of DMD, waiting for a comfirmatory study before marketing the drug would amount to a death sentence.
    With the passage of the FDA Modernization Act of 1997 (FDAMA) and amended in 2012 by the FDA Safety & Innovation Act (FDASIA), Congress created a pathway for rare diseases to receive fast track and accelerated

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