Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Q2 2023 Earnings Call Transcript

Reshma Kewalramani: Colin, I think there are 2 separate questions in there. One about the vanza triple and what do we need to see and then one on DMD. Let me tackle the DMD, DM1 question, I’ll come back to vanza. On the DMD question, I’m going to broaden it out to muscular dystrophies as a whole and I’ll talk about DMD. and DM1. We have programs in DMD that are going through IND-enabling studies now as well as in DM1. We actually have multiple programs in DM1. The lead program is the one that we in-licensed from Entrada. And that program also is already in IND-enabling studies and both of them should have those results in the second half of ’23. And our timing remains to file the IND for both DMD and for DM1 for the lead program in DM1 in the second half of this year.

With regard to the vanza, I think Stuart just covered that. What we’re looking to see in the way the study is designed is vanzacaftor in the Phase III program head-to-head versus TRIKAFTA and the primary endpoint is sweat chloride. And the reason for that is, again, with patients who — with carriers, those with who have 1 CF gene, they have virtually no manifestations of disease. And that is measured assessed by the sweat chloride carrier level is a description of sweat chloride levels. So that’s what we’re measuring. We are, of course, going to have PPFEV1 in there. And as Stuart said, if the profile is, as I described it to be, improvement on sweat chloride levels, we expect it to have real value to patients. I’ll also add that the vanzacaftor triple has a lower royalty burden than the TRIKAFTA combination.

Susie Lisa: Thanks, Reshma. Thanks, Colin. Gary. That brings us to time. Could you close it out, please?

Operator: This concludes the question-and-answer session and the conference is also now concluded. Thank you for attending today’s presentation. You may now disconnect.