Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Q1 2023 Earnings Call Transcript

Reshma Kewalramani: Sure. Let me comment on the target product profile a bit, and then I’ll ask Stuart to comment on the market size, both in terms of patient numbers and dollar potential. So Phil, if we recapitulate the results that we saw in Phase 2, which you’ll recall, is substantially similar to what we’re doing in Phase 3. That is to say, a study in abdominoplasty as a form of a soft tissue model and bunionectomy as a form of a hard tissue pain model, that would be a home run for us. What I mean by that is efficacy, pain relief that’s quick and durable, a benefit risk profile that is quite attractive and by mechanism of action, that is to say the way 548 works is on the peripheral nervous system, not centrally no addictive potential. That is an absolute home run. I’m going to ask Stuart to comment on market size in terms of dollars and patients. Stuart?

Stuart Arbuckle: Yes. Thanks, Reshma. So the study program that we’ve got is we designed, which Reshma just commented on, is designed to secure for us a label for moderate to severe acute pain. In contrast to a number of other relatively recent approvals in acute pain, which have been related to postsurgical pain and linked to certain procedure types, that’s not the label we are seeking for. We are seeking a label for moderate to severe acute pain with large, could be post surgical and that’s indeed the nature of our studies, but that is not the label we’re seeking. So we are not sizing the market based on the number of procedures or the number of a surgery, it is for the broad treatment of acute pain, which can’t be controlled with kind of standard NSAIDs and things like that.

When you look at that market opportunity in the U.S. alone, it’s roughly 1.5 billion treatment days of medicine are utilized and despite 90-plus percent of those prescriptions being generic, therefore, very, very cheap, it is a $4 billion market in the US today. So we see a very, very significant commercial opportunity for VX-548 if it has the profile that Reshma described.

Operator: The next question will come from Michael Yee with Jefferies. Please go ahead.

Andrew Tsai: Andrew Tsai on for Michael Yee. Thanks for all the updates. So I wanted to follow up on the acute and chronic pain study. So the first question we have is, do you think acute pain should have read-through to chronic pain and vice versa? And then second question for us is for your Phase 1 CF program with Moderna. We understand this is a SAD data set coming up later this year. But just curious when we can expect to see longer-term that data? Is it possible that could come out later this year or should we be ruling out that scenario? Thank you.

Reshma Kewalramani: Sure. Let me take the mRNA question first, and then I’ll come back around to pain. With regard to the program we have ongoing with Moderna, that’s the VX-522 program. It is a program for the approximately last 5,000 patients or so who simply can’t benefit from CFTR modulators. It is a SAD MAD program with the SAD portion of it going on right now. When we did KALYDECO and TRIKAFTA, for example, our patients used to tell us that they knew from the first day that they were on placebo or active therapy. So I can’t rule out when we will know whether we have efficacy. But in terms of time lines and what you can plan for, we expect to be done with the SAD this year, and we expect to be well into the MAD this year.