Rhythm Pharmaceuticals, Inc. (RYTM) Earnings Call Transcript & Summary

Good day, and welcome to the Rhythm Pharmaceuticals Conference Call. [Operator Instructions] As a reminder, this call may be recorded. I'd now like to turn the call over to Dave Connolly, Head of Investor Relations. Please go ahead.

Thank you, Michelle. Good morning. I'm Dave Connolly here at Rhythm Pharmaceuticals. This morning, we issued a press release announcing that the extension of the PDUFA goal date for setmelanotide in HO was extended from December 20 to March 20. That press release is available on our website. For those of you participating on the conference call, we have a few slides that can be accessed and controlled by going to the Investors section of our website, ir.rhythmtx.com. On the call today is David Meeker, our Chairman, CEO and President; Alicia Fiscus, Senior Vice President and Head of Regulatory; and CFO, Hunter Smith are here today as well available to answer questions. On Slide 3, I'll remind you that this call contains remarks concerning future expectations, plans and prospects, which constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent annual or quarterly reports on file with the SEC. In addition, any forward-looking statements represent our views as of today and should not be relied upon as representing our views as of any subsequent dates. We specifically disclaim any obligation to update such statements. With that, I'll turn the call over to Dr. Meeker, who will begin on Slide 5. And at the conclusion, we will take a few questions this morning from our covering analysts. Dr. Meeker?

Thank you, Dave. Good morning, everyone, and thank you for joining on such short notice. We learned that last evening that the FDA extended the PDUFA goal date in connection with the review of our supplemental NDA for setmelanotide to treat patients with acquired hypothalamic obesity. The agency asked for additional sensitivity analyses in October and then deemed our response to their request to be a major amendment, which allows the FDA reviewers to extend the PDUFA date by 3 months. The PDUFA was extended, as Dave noted, from December 20 this year to March 20, 2026. We will obviously work closely with the reviewers, and we have every competencies. Additional analyses confirm the robustness of setmelanotide's efficacy and support approval. So here's what happened. We received an information request dated October 20 and submitted the response by the due date of October 27, and extension was based on that response. The information request asked for a sensitivity analysis related to our first key secondary end point. The proportion of adult patients with greater than or equal 5% reduction in BMI or pediatric patients with a greater than or equal 0.2 BMI Z-score reduction. Specifically, since this was a global trial, we only record the year of birth for each patient and not the month and day because of data privacy laws. For a child, months make a difference in the calculation of their BMI Z-scores. For the analysis, we have used an imputed date of January 1 as the birth date for each patient when calculating BMI Z-scores, which are age dependent, which is the same approach we have taken for each of our trials. For this information request, the FDA asked us to use an imputed birth date of July 1 for each patient as opposed to January 1 as an additional sensitivity analysis and to rerun that analysis for all patients under [ 18 ]. The results of that reanalysis and how the results compared to the original analysis are shown on Slide 5. The left side of the table shows the setmelanotide and placebo results for the 120 patient cohort and the full 142 patient cohort using the January 1 imputed date. This was our original submission. The right side of the table shows the same data using an imputed date of July 1. As you can see, when the data set was reanalyzed with the adjustment, the results were highly similar in the trial conclusions unaltered. We also have previously submitted as part of the review, a couple of additional sensitivity analysis on the full data set, which had been requested, which showed similar results and no change to the conclusion. The FDA judge this October 27's response to their information requests as constituting a major amendment justifying the extension of the PDUFA date. Importantly, we have not been notified of any significant safety or efficacy concerns. This extension was unexpected and obviously incredibly disappointing, most importantly, for patients with hypothalamic obesity with no approved therapies available. We will continue to work with the FDA to swiftly address any additional questions. And as Dave noted, we'll move now to questions and obviously, happy to take questions here. These notifications we have from the FDA are extremely cryptic. So I don't have a lot of additional information beyond -- if any, beyond what we've laid out here and our goal in laying this out, is try to give you as much transparency and visibility into what happened here. So with that, we'll go to questions.

[Operator Instructions] Our first question comes from Phil Nadeau with TD Cowen.

Thanks for the transparency, very helpful. Two from us. I guess, first, so you had this information request. It seems like it doesn't change the conclusions. Are there any other outstanding information request from the FDA? Or was this the last one? And then second, if memory serves me back in 2022 when you got the label extension for BBS, there was also a request for, I think, a sensitivity analysis that resulted in a 3-month push to the PDUFA, but ultimately the application was approved. Can you remind us what happened back in 2022 and how similar the situation is to that?

Sure. Let me take that 1 first. So yes, we did have exactly the same situation on the BBS file. They asked us for a relatively voluminous request, again, a sensitivity analysis, I'm really analyzing much of the same data set. That was submitted. To be honest with you, we had no further interaction around the data we resubmitted, the review then went forward. And as you know, that we were approved on the new PDUFA date. So it's similar only in the sense that they ask for a fairly -- they asked a big question with a bunch of additional work. And what also happens with these questions, I mean, this -- we generated a very simple table here. And as you can see from the table, it shows there's nothing has changed, that would be easy for them to look at. That's not all they want. They want all of the original data laid out in table form for every single patient, and then they rerun the analysis themselves. So the additional work in time that they need is based on the fact not that they need to review our table, but that they need to review all of the original data and redo the analysis. So yes, similar in that sense. Are there any other requests? So the review is still ongoing. We -- you continue to get questions as you go forward. The way the timing works here, we were just coming up on 1 of the dates in -- formal dates in the review period where they are required to give you some feedback on what our potential post-marketing request, that was November 8, the Saturday. So that would mean ideally today, we would have gotten some of that information. So they were bumping up against a time line for a requirement on their side. But as I said, the review will go forward up until presumably the March 20 date, and they can continue to ask questions on any part of the file as that goes forward. But there's been nothing, as we've gotten to this point, and there's nothing that we see going forward that they would request, but I don't know. I mean we'll see. I mean they can obviously ask additional questions.

Our next question comes from Derek Archila with Wells Fargo.

Just a quick question from us in terms of just the reviewers. Any changes there in terms of like who's been reviewing the application that might have driven this change or this additional request or -- just a question there. And then in terms of -- again, kind of I guess piggybacking off Phil's question, I mean, is there still a possibility that we could get something on the earlier side of approval before the new PDUFA date just given that, is it like such a voluminous data request, as you kind of said with BBS, is this kind of less volume in terms of data that you're submitting?

Yes. So technically, they can approve it at any time. The PDUFA date is just a goal target. So yes, in theory, it could come earlier. I think historically, my understanding is that's not what tends to happen, they tend to use the time available. So I think our expectation going forward is that March 20 would be the date. They did ask -- sorry, when going back to Phil's question, for updated data now that we have this extended PDUFA date, there's -- Japanese patients will complete their full period, so they want that data as it becomes available as well. So they'll use the full time is my guess. Sorry, there was another question there, Derek?

Yes, just any changes in the personnel reviewing the file.

Sorry, yes. So no, not that we're aware of. I mean all the communication comes from the review division Director. That has not changed. And again, as we've highlighted in our other calls, it seems like we're moving along in a pretty straightforward normal situation. It didn't seem to be affected by some of the other changes at the FDA. The only other thing, and again, we can all just speculate on what's going on here. I mean this is a busy division. They reviewed all the obesity submissions. And we know the priority review voucher discussion just came up, and there -- some obesity drugs that look like they may be in there, and that may put additional workload on this. So all of this is pure speculation. And again, I don't know, we only know what we got from my cryptic notification.

Our next question comes from Mike Ulz with Morgan Stanley.