Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q4 2022 Earnings Call Transcript

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Ian Clements: Yas, appreciate that.

Yasmeen Rahimi: I really wanted to say that. And then, in regard to my questions, obviously, a lot of investors are eagerly awaiting volixibat readouts. And I think a fundamental question that comes up is what’s going to be the translation? So obviously, we get the news that in PSC, you can move forward being a pivotal study? Like can you maybe help us understand sort of how much that de-risked PSC opportunity and also what that means for you to put more dollars and more into continuing to really be behind volixibat? That’s sort of my question for you guys. Thank you, again.

Chris Peetz: Thank you for the question. I’ll have a couple of comments and then pass it over to Pam to talk a little bit about the interims, how it relates to the final data. I think the thing I’d say at the outset is, there is really strong proof of concept for IBAT in both of these indications already. Some great clinical data from the Maralixibat program and PSC showing that you can reduce bile acids and pruritus in patients that have elevations coming in. So the patient population that’s in this study. So I think we already have really strong critical derisking of knowing that an IBAT can be active in these settings on these end points. Maybe Pam can speak a little bit to the specifics of the study design and the interims need.

Pamela Vig: Yes. Thanks, Yas, for the question. So just to add on to what Chris was saying. In our own PSC experience, we’ve seen that patients who have moderate to pruritis. And this is in an open-label study that with 70% itch reduction and almost 50% reduction in that population. So again, showing that we know what this mechanism can do in the disease and frankly, also seen in Alagille and PFIC across whole set diseases. Now as you look to our statistical assumptions for the studies in the interim, for PSC, the interim includes 45 patients and that is triggered when all patients have reached week 12. So that means some of the patients will be at 24, some will be beyond, but all patients will be week 12. And this is a really great way for us to look at observational differences and that will help us assess if we’re on the right track for the final analysis.

The interim is not powered for significance by design, but it allows us to determine or in the zone for a positive final analysis. And a couple of notes on our kind of confidence in the way that we’ve conducted the design or that we set up the design is that the data in PBC showed statistical significance in their analysis when they looked at baseline over time. And they only had about 20 patients in each of those cohorts. And that was seen in their BID dosing. And importantly, that was seen in the high dose in the BID dosing regimen. So we think given our dose, given our assumptions, given the number of patients we have, we’re really confident about our assumptions and may even be overpowered.

Yasmeen Rahimi: Thanks so much.

Pamela Vig: Did that answer your question?

Yasmeen Rahimi: Yes, that was extremely helpful. Thank you, Pam.

Operator: Thank you for your question. There are no additional questions waiting at this time. So I’ll pass the conference back to Chris for any closing remarks.

Chris Peetz: Great. Thank you, operator. And I just want to say thanks again to everyone for joining today’s call, and hope you have a great day. Goodbye.

Operator: That concludes the conference call. Thank you for your participation. You may now disconnect your lines.

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