Part II. Excepts are from Seeking Alpha.
3. VX-809 Study:
-Pushed on safety profile of combo versus just VX-770, i.e., drug-drug interaction or PK change from 809 alone and Vertex stated “there is nothing out of the extraordinary.” Hope he meant out of the ordinary!
-Asked the study design since both homo and hetero included. Vertex stated “There is specific subgroups. The majority of the trial is the homozygous patients and then there is one cohort of heterozygous patients and enrollment is obviously separately (sic) into those groups.” This information took me by surprise because the trial information does not specify these different sub-groups, at least not from what I saw on the government study webpage. I also expected the results to be positively skewed because of the inclusion of heter 508s, but that is not possible given separate groups and small number of heter.
-Asked what Phase 3 would look like and said they aren’t sure yet mostly because they have to meet with global regulatory authority. But Kauffman added that the endpoints would likely be the same as 770–I had thought Vertex was pushing for using possible other measures besides SC and FEV1, but doesn’t seem that Vertex is there yet.
4. VX-661 Study:
-My reading of the transcript is that the VX-661 study is doing higher dosing than the initial VX-809 study, i.e. like Phase 2 Part 2 for VX-809, VX-661 doses are escalated.
-As noted above, VX-661 is only for homo 508 Cfers.
5. Vertex was asked whether it was considering purchasing its royalty rights back from the CF Foundation and Vertex demurred.
whewww. Thanks. That makes sense especially given the labeling on VX770 re when to take; so they'll have to make labeling specific on any combo if fat/water soluable issues. From your experience, is there a higher chance of drug/drug interaction because of opposite soluability, i.e., on drug water and one fat? The conference said nothing really of concern with 770/809. Just wondering if the difference b/w 661 and 809 in soluabiity might indicate more of a saftey concern as well?
Brean Murray has not been overly bullish or bearish and they put out the following note saying 300 million in US sales and another 300 million in ex-US. But I think their time frame is too cautious. A 90% penetration can be achieved next year. And sales will double in a few years when R117H and other gating mutations along with pediatric CFers are also treated. All together the sales should add up to around 1.2 B in a few years. This 1.2 B figure was also given by Bernstein's Geoffrey Porges.
Of course, if 809 or 661 works for 508 mutations as well as 770 worked for 551, multi billion sales is possible.
[> Vertex Pharm will be penetrating over 90% of the G551D patient population by
2017 with approval of Kalydeco, yielding over $300 mln in U.S. sales - Brean
Murray (36.94 +2.21)
Brean Murray notes, approval of Kalydeco positions VRTX to launch this novel
cystic fibrosis drug into an ultra orphan sub-group of patients at an
announced price of $294K/year. Given the targeted population, the safety and
efficacy of the drug and the fact that it is orally administered, the firm
believes there will be very little pushback on reimbursement and usage. They
see Vertex penetrating over 90% of the G551D patient population by 2017,
yielding over $300 million in U.S. sales and a similar number ex-U.S. <]
Off the subject of 809/661,but curious about your opinion of CF revenues for 2012. Summer Street Partners reiterated their Buy rating but lowered their price target and projected CF revues of $95m for
2012. Isn't that absurdly low? Projections?
Also, is it clear that mid-year we will see 809 data? If so, is the bar lowered for approval given the aggressiveness of
the 770 approval? Time frame?
Thanks for the info. I hadn't heard that at all and fron conference call it sounded like there weren't any issues with the 770/661 study, but I was wondering why nothing had been said on the format. Are you familiar with this issue? Are there any "solutions" to overcome the water/fat soluability? Why wouldn't Vertex know this much earlier/why would they even consider moving forward with 661/770? I mean wouldn't they know they were fat/water soluable and couldn't dose together along time ago?? Any thoughts much appreciated as this is a twist I have never heard of/have not read any science on. As always, greatly appreciate it.
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