My english is not so good so I'm having trouble understanding why this CF drug from Vertex has such a blockbuster potential? What is the biggest difference between Kalydeco combined with an experimental drug VX-661 and Arikace?
Arikace targets bacteria which thrive in the congested lungs of an individual with CF. That other drug is targeted at actually reversing one of the effects of the CF mutation, so that there is less congestion.
That uncertainty is all that's keeping the share price at anywhere near the current level. Anybody who has been in biotechs for any length of time will be all too aware that promising Phase II results are not always replicated at Phase III.
But having said that, the performance of Arikace in the Phase II multi-cycle extension study WAS rather special.
The average improvement in FEV-1 evidenced in the approx 43 patients 28 days after the third period of therapy was around 5%. During the three-cycle study which supported the approval of Cayston, the changes from predicted FEV-1 for Cayston and Tobi averaged across the entire study period were a 2.05% increase and a 0.66% decrease respectively.
During the Arikace study, the average increase in FEV-1 across the first three cycles was a lot higher than that measured in the off-therapy periods.
And it's well worth noting that the results due mid-year will offer the first evidence of efficacy from a multi-cycle Arikace study where the patients' therapy was interrupted by off-therapy phases of only 28 days (as opposed to the 56-day phases in the extension study).
Yes take look at INSM phase 2 data in CF - very compelling. Like I said before tough not to bet that we have good shot of favorable results plus the hurdle rate for successful outcome is relatively easy to achieve. As for premium it will move much higher based on many dynamics - short interest, valuation including mktr potential in CF and NTM., drug platform for using with other available drugs. Then you also have some takeout premium as their will be interest from large pharma for this drug platform. That is why by the end of this summer we may have a $1B plus market cap here....But lets save all that talk for later - dont want to sound like a pumper...=)
Man, that sent a chill up the basher's spine! I'm pretty sure that our drug for CF is worth at least half of the Vertex drug, but they're not in competition, as our drug is to target infection, besides the fact that we wrapping up phase 3 with 300 patients!