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

Reshma Kewalramani: Sure, Mohit. This is Reshma. I think your question is about exa-cel and what do we really see as the real potential? I have very high confidence in the fact that there is significant unmet need. This is a disease that has really no therapy that can offer curative potential, full stop. There is no debate on that. With regard to the potential of our medicine, all of the data that we’ve showed to date show that this medicine has transformative, if not curative potential. And I’m going to ask Stuart to comment on what we’ve heard from patients, from payers, and from stakeholders in general about their level of understanding and enthusiasm for this product. And I’ll also ask Stuart to remind what we see as the market size. What I’ll say is that in the U.S. and Europe, there are 150,000 people with sickle cell and beta-thalassemia. We are not targeting that full set with this busulfan based conditioning exa-cel program, we’re targeting 32,000. Stuart?

Stuart Arbuckle: Yes. As Reshma said, the 32,000, those patients who are sort of the severe end of the spectrum, essentially, patients very similar to those that were enrolled in our clinical trials. These are patients who are having multiple occlusive crises per year for our sickle cell disease population and multiple transfusions in the year for those in the transfusion-dependent thalassemia patient population. So these are patients who — whose lives are very, very significantly impacted by their disease, and it really impacts all aspects of their life, impacts their loved ones, impacts their ability to work, and they have a very, very significant burden of disease and also a very significant burden for the health care system.

So in the discussions that we’ve had with both patients, payers, and physician groups, the unmet need is very, very clearly well understood, the potential for curative onetime therapies is very, very much something that people are looking forward to. I think with any new technology and with any new product, there are always going to be some questions that people will have around things like safety as these are really transformative and innovative therapies. And to some extent, for some people, that’s something that can only get solved over the course of time and with their own live experience. But certainly, the level of enthusiasm we’re seeing from all stakeholders is very, very high, and we’re very optimistic about this being a multibillion-dollar opportunity.

Mohit Bansal: Thank you.

Operator: The next question will come from Geoff Meacham with Bank of America. Please go ahead.

Geoff Meacham: Good afternoon, everyone. Thanks for the question. Just have a couple. Stuart, on the vanzacaftor triple. Just curious on your updated view as to differentiation, obviously, beyond dosing. Just assuming that a patient is doing well on TRIKAFTA, would the argument be to switch or I think you mentioned in the prepared remarks that patients have been — have discontinued TRIKAFTA, what’s the logic and then maybe responding to the new vanzacaftor triple? And then secondly, Charlie, or Reshma, you guys have almost $12 billion in cash, pretty good pipeline and no real urgent need for BD. So should we think about capital investments in areas that are probably need even more of a build-out like gene or cell therapy? I’m just curious about that uses of cash going forward? Thanks.

Reshma Kewalramani: Sure, Geoff. Let me ask Stuart to tackle vanzacaftor first and then I’ll ask Charlie to comment on capital allocation. Stuart?