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

Reshma Kewalramani: Yes. So Robyn, the bar for neuropathic pain is to have a better overall profile benefit risk taken together than existing therapies. As you know, the existing therapy has limitations in terms of efficacy but there are also limitations on the safety/AE side. And the reason for that is what we use for neuropathic pain is frankly, a recycled medicine that comes from fundamentally central nervous system depression that we are reusing for neuropathic pain because that’s the best we have. So what we’re going to be looking for is improvement in diabetic peripheral neuropathic pain scores, change from baseline and our Phase II dose-ranging proof-of-concept study also has a Lyrica arm for context. So we’ll be able to see the magnitude of the treatment effect as well as a Lyrica arm for context.

Operator: The next question is from David Risinger with Leerink Partners.

David Risinger: Yes. I wanted to change gears, please, to your two AAT candidates. Could you frame the efficacy results to watch in 2024? And potential timeline for those readouts next year?

Reshma Kewalramani: Sure. David, I think you’re asking about the AATD program. And just to ground everyone on that one, this is our program where we have 2 molecules, VX-864 which is in a Phase II study and VX-634 which is making its way through a Phase I study. Our excitement for this particular program and disease comes from the fact that it fits the Vertex strategy like a glove. We are seeking to target both the liver and lung manifestations of this disease. And our small molecule approach is the only one that holds the potential to treat both liver and lung manifestations. And I do believe you need to treat both in order to have a transformative medicine. Our VX-864 study which is in Phase II is a long-term study. It’s a 48-week study.

And there, we are looking at the impact of long-term dosing on both functional AAT levels in the blood and clearance of liver polymer. You might recall that on a post-hoc analysis of our VX-864 Phase II data from a few years ago, we saw a 90-plus percent reduction in serum Z polymer levels which is why we’re so interested in the liver polymer. And in the 634 study, we are going through our first in-human studies. So I expect that we’ll have all the results from both of these trials by sometime next year, so 2024 and I expect that we’ll be able to share the results at that time. Exact timing, we’re going to need to get a few more months under our belt to look at the enrollment dynamics but I do expect we’ll be sharing results by sometime next year.

So it’s a ’24 milestone.

Operator: The next question is from Terence Flynn with Morgan Stanley.

Terence Flynn: Stuart, you mentioned there were about 20,000 patients not on drug at the start of the year that could potentially be eligible. Just wondering where that figure will end, assuming you achieve your new 2023 guidance?

Stuart Arbuckle: Yes, Terence. So we’ve kind of gone away over the last few years of kind of giving detail to the forensic accounting of all the different patient numbers. And so I’m not going to kind of give you an updated estimate at this time. But as Charlie said in his remarks and I set in mine, we’ve continued to make good progress in treating more patients, including in younger age groups and including in other countries where we’ve secured reimbursements and launches. But other than that, we’re not going into more detail at this time. We may — if there’s a substantial change, we may update those numbers as we’ve done in the last couple of years or so at the beginning of next year when we talk about our guidance for the following year.

Operator: Next question is from Mohit Bansal with Wells Fargo.