By the way, details of the Poster session are already on the ATS website:
Poster Discussion Session: Tuesday, May 20, 8:15 AM - 10:45AM,
Sapphire Ballroom E-F (Level 4), Hilton San Diego Bayfront
Poster Viewing: 8:15-9:15, Discussion: 9:15-10:45
A Randomized, Double-Blind, Placebo-Controlled Study Of Liposomal Amikacin For Inhalation (Arikace®) In Patients With Recalcitrant Nontuberculous Mycobacterial Lung Disease
C27 | DIAGNOSIS AND TREATMENT OF NONTUBERCULOUS MYCOBACTERIA INFECTIONS
The abstract has a few more interesting details:
90 randomized 19% CF, 81% non CF
64% MAC, 36% M-Abs
90 started, 1 died
72 continued into open label
41 of which have already finished
the deadline for poster submission is March 30th (because they are peer reviewed); therefore insmed knows a lot more detail than was discussed yesterday.
insmed is an exhibitor too (booth #2933)
By the way, the abstract refers to 590mg dosage (as did yesterday's PR). Curious: it has always been 560mg until now. typo? hmmm...
Also (keep in mind) by mid-May the FDA MUST respond (with 60 calendar days) to Insmed's request regarding Breakthrough Therap
Fwiw, my delve into this issue leads me to believe that the dosage actually used for the NTM study was 590 mg.
As regards CF - apart from the discarded 280 mg option only the 560 mg dosage has been mentioned. But not since last year:
February 19 2014 -
[ The Phase 3 trial was designed to assess the comparative safety and efficacy of ARIKACE and TOBI in CF patients with Pa. A total of 302 adult and pediatric CF patients with chronic Pa were enrolled at 75 sites in Europe and Canada, and randomized to receive 28 days of ARIKACE treatment delivered once-daily via an investigational eFlow ® Nebulizer System, or TOBI delivered twice-daily via the PARI LC Plus ® Nebulizer System over a 24-week treatment period. ]
March 6 2014 (2013 10-K) -
[ We completed a registrational phase 3 clinical trial of ARIKAYCE for CF patients with Pseudomonas lung infections in Europe and Canada during the second quarter of 2013. The phase 3 trial was a randomized, open label, multi-center study designed to assess the comparative safety and efficacy of once-daily ARIKAYCE administered for approximately 13 minutes via the eFlow Nebulizer System and twice-daily Tobi ... in CF patients with Pseudomonas. A total of 302 adult and pediatric CF patients with chronic Pseudomonas were randomized to receive 28-days of ARIKAYCE treatment or Tobi delivered twice-daily via the PARI LC Plus® Nebulizer System over a 24-week treatment period. ]
It may well be that the precise dosage is now irrelevant.
Approval of Arikace will be a combination approval of Arikace delivered via the PARI inhaler. I believe the inhaler has a reservoir, into which the Arikace will be poured. It strikes me there's no guarantee with any of these devices that all that goes in one end will come out the other.
Take TIP for example - where users are advised to check each capsule after use.
Perhaps the basis of Arikace approval will be the use of the inhaler for a given period of time?
Fud, so do you think that Insmed was directed to up the dosage to 590mg to make the comparison with TOBI on a more even playing field? (TOBI is 300mg x 2 each day). I think that would have been the case. It would not have been a good trial had we used only 280mg and 560mg was a little short. AND you would be correct that the exact dosage inhaled could be a little less than the full amount.
I'm still skeptical as to the dosage amount. To what authority are you referring?
On ClinicalTrials_gov website, ID# NCT01315236, Arikace® for Nontuberculous Mycobacteria, the dosage is 560 mg.
In the March 26th Press Release, and on slide 24 of the latest Investor Presentation, it is 590 mg.
All of Transave/Insmed's previous trials (and pre-clinical work) involved Arikace dosage at 560mg and the recent Phase 3 CF/Pa trial protocol is (also) 560mg as is the Phase 2 (previous Phase 3 befoer the "rat" incident) NTM trial protocol.
Yet the most recent quarterly filing (the 10-K for 2013), the recent PR regarding NTM top-line data and now ALL the abstracts at the ATS website (including the CF/Pa trial updates) refer to 590mg.
For all the millions spent on trials, you'd think they'd get the details right.
Very useful thanks - I missed that post.
Oddly enough, a recent comment by Michael prompted me to finally look into the dosage issue. It isn't a mistake - the NTM study entailed a 590 mg dose.
I'll probably elaborate during the Easter break when we're all bored.