Arikayce fails Phase 2 trial


Of the 68 patients who completed the 84-day open label trial, 21 were culture negative at day 168 (the open label segment followed an 84-day double blind trial). Of the 21 culture negative patients, 10 were culture negative at the beginning of the open label segment. The other eleven culture negatives were five patients in the Arikayce arm and six patients who switched from placebo to Arikayce. This implies a cure rate of ~16%.

In the double blind segment of the trial, 11 of 44 Arikayce patients were culture negative by day 84 implying a cure rate of 25%.

In April, the firm submitted its application to the FDA for BTD designation for Arikayce. It is already designated an Orphan Drug, a Qualified Infectious Disease Product and Fast Track for the treatment of nontuberculous mycobacterial lung infections.

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Comments (2)
  • jayemel
    , contributor
    Comments (16) | Send Message
     
    Although this phase 2 study did not prove universal success, for the 22 patients (11 in double blind portion, additional 11 in open label continuation, in sum representing about 25% of the initial enrollment), who achieved negative cultures, this treatment was definitely successful. Remembering that the study cohort was comprised exclusively of patients who had failed conventional therapy for at least 6 months prior to their entry, as well as the minimal downside in terms of adverse effects, the addition of inhaled amikacin to the pharmacopia available for treatment of Pseudomanal pulmonary disease should be most welcome.
    20 May 2014, 04:24 PM Reply Like
  • User 11955571
    , contributor
    Comment (1) | Send Message
     
    Absolutely terrible, what Seeking Alpha and the Fool report without doing their own due diligence!

     

    The Secondary Endpoint was required by the FDA, as they wanted to see culture conversion. With such a short-term study, Insmed did not feel they could meet that as the primary so they created a Primary Endpoint which had not been used. And remember, this NTM population was virgin territory for a clinical trial. As jayemel pointed out, the patients in the study were the most difficult in terms of prior treatment response. The double-blinded P2 results showed a 25% conversion that amazed the researchers. Now with the open extension, they have exceeded 30% culture conversion. So Seeking Alpha and Motley Fool bash results when they do not understand the study and the data. C'mon!
    21 May 2014, 07:59 AM Reply Like
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