Roivant Sciences Ltd. Common Shares Study result

Please go ahead. Good morning, and thanks for joining today's call to review the results from the PHocus study of mosliciguat and PH-ILD.

I'm Stephanie Lee with Roivant. Presenting today, we have Matt Gline, CEO of Roivant. For those dialing in via conference call, you can find the slides being presented today, as well as the press release announcing these updates on our IR website at www.investor.roivant.com. We'll also be providing the current slide numbers as we present to help you follow along. I'd like to remind you that we will be making certain forward-looking statements during today's presentation. We strongly encourage you to review the information that we filed with the SEC for more information regarding these forward-looking statements and related risks and uncertainties. With that, I'll turn it over to Matt.

Thank you, Steph, and thank you everyone for joining this morning. These are always the fun ones. Great data is always good to report on, so I appreciate everyone making time for us, and I'm looking forward to taking you through the results from the PHocus study in PH-ILD. I'm going to do a little bit of frame-up and then I'll get to the data. Starting on slide 3. This is a slide that we've been showing, I think since our investor day late last year, laying out that we have a busy couple of years ahead, and I'm happy to report we continue to add check marks to this slide as we move forward.

Obviously, this one is particularly great, first of all, coming so closely on the heels of the approval of what's now LISRAYA in DM, but also I think properly standing mosliciguat up as a third leg of the stool, which feels really awesome. I'm looking forward to talking more about it. I'm not going to spend time on slide 4 because I'm going to go through the data in great detail in just a moment once I get through some background. The one thing I will say is, look, this is a great day. This is a phenomenal data set. It is robust across both the primary endpoint PVR, functional endpoints like six-minute walk, exploratory pre-specified analysis out to 24 weeks showing improvements even beyond what was possible at the primary at 16.

We were not powered for a P-value at 6-minute walk, but we got a really nice one. Just a great data set all along. It is supported by hemodynamic measures and overall consistent with the disease state, obviously, but a picture on safety and tolerability that feels really good. This is a great data set. Obviously, and I will do this again at the end, there are a bunch of people who need to be thanked here, including the Roivant team, the Pulmovant team for running a great study, and always, most importantly, the investigators and the patients who trusted us with their care. That is actually a pretty good segue to. Again, I think many folks on this call are likely familiar with pulmonary hypertension and with PH-ILD, but I just want to spend 2 seconds on background. This is, in short, a terrible disease.

These patients on slide 5 here are very sick. PH-ILD, these are patients with pulmonary hypertension that comes from lung disease. They often have lung function issues associated with underlying lung disease, and then also issues with blood flow to the lung from the pulmonary hypertension. They have horrible shortness of breath on exertion, so really great difficulty in activity to daily life. Lots of negative sequelae, including, frankly, just a ton of mortality. These patients are really sick. Lots of comorbidities. Things like pulmonary circulation disease, congestive heart failure, vascular disease of other kinds. Just a really, really tough prognosis. Now options. Within the last few years, there has been the introduction of inhaled treprostinil, which has been a big step forward for the field here, but we feel strongly that another option is a real benefit and one that delivers this kind of efficacy as well as the tolerability and safety that we have shown here is something we think could make a big difference for these patients.

We are truly thrilled here. PH-ILD on slide 6. I talked a little bit about this just a moment ago, but again, a really bad disease. 1 and a half year median survival despite best available standard of care treatment, which as I said, includes inhaled treprostinil. We said in other settings about 200,000 patients between the U.S. and Europe. It is a big patient population with a lot of people with high need.

Again, other than sort of the really old stuff, really nothing other than treprostinil approved for this patient population. A lot of opportunity to deliver some value. mosliciguat, which we have talked about at length on some earlier calls, we thought from a sort of mechanistic rationale perspective, a really great fit for the disease, especially in the format we have got here, which is on an inhaled basis, where delivered directly to the lungs to activate impaired sGC. We think this could be the first non-treprostinil treatment option. sGC is a key enzyme in the pathway here, essential for vascular homeostasis. We activate both impaired as well as native sGC and restore cGMP production. In short, we are an inhaled vasodilator, and we think that has some real benefits, and then we have potential reduction of fibrosis and inflammation as well.

We think a really good mechanism, and again, obviously borne out with the data. The study here on slide 8 was a large double-blinded multicenter global trial, 135 patients in PH-ILD across close to 90 sites in 20 countries. Primary endpoint was PVR at week 16, with key secondaries including 6-minute walk and NT-proBNP, and then some pre-specified 24-week exploratory endpoint, 6-minute walk, NT-proBNP, and TTCW at week 24 as well, or 20 week TTCW. A robust study, and we will talk more about the data in a second, but obviously set up for hopefully this kind of outcome. We had talked a little earlier in June about the blended baseline characteristics. We show them by arm on slide 9. In short, pretty well-balanced across the arms in most respects, and I think a fair test of the drug.

Not too much else to say about it other than it was a pretty sick patient population in the study, which I think speaks to the quality of the data that we have been able to generate here. I do not want to spend too much time on preamble. I really just want to get to the highlights. By the way, this data was also just presented at ERS in Europe this morning as well. So excited to get it out to the academic community, and I think it hopefully will have made a splash there. My understanding is there were a lot of docs in the room. On slide 11, in short, this is the primary endpoint data. I believe this is the largest reduction in PVR from baseline ever observed in any pulmonary hypertension study, regardless of which type of pulmonary hypertension.

It is in the box on the upper right-hand corner here, but 100% of patients on drug saw a reduction in their PVR. We had a placebo-adjusted change in PVR at 56.3% reduction with a obviously quite low p-value. This is, on an objective endpoint, a commanding outcome and one that we think speaks to the potency of the drug and the effect that we are having in these patients. So a great, obviously primary. This on slide 12, I just said this, but this is we think the deepest or among the deepest PVR reductions ever seen in pulmonary hypertension. It is a very deep reduction, and it speaks to the breadth of outcomes that we are going to talk about today, including on hemodynamic measures and otherwise, that we are able to deliver this kind of reduction in PVR.

I think it hangs in harmony with the rest of the data that we are going to share. On slide 13, this was actually an analysis that I found really interesting, and I think it speaks to the breadth of what we are going to be able to do with mosliciguat from here. We saw a huge benefit relative to placebo in every pre-specified subgroup. There were a whole bunch of them ranging from age and sex subgroups to patients with and without emphysema, patients on or not on anti-fibrotic therapy, patients with different levels of pulmonary fibrosis, patients with different levels of baseline PVR, baseline 6-minute walk, different forms of PH-ILD. Again, across the board, we saw really, really big benefits. Some of these are relatively small subgroups, but really big benefits in PVR. I think this is helpful for a bunch of reasons.

One is it really underscores the robustness of the data for this patient population. The other is it opens the door to all kinds of other potential forms of pulmonary hypertension, et cetera, for mosliciguat. So we think there's a whole lot of opportunity here, and obviously, the robustness of this data across subgroups feels really good. On slide 14, one of the sort of greatest bits of intrigue going into this dataset is we had said it a bunch of times. This was not a study that was powered to deliver in six-minute walk. Six-minute walk is a finicky endpoint, and you just never know what you're going to see with that kind of variability. We had a really great outcome in six-minute walk. We showed a 35-meter change from baseline delta to placebo at week 16. You can see that on slide 14.