Rhythm Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Michael Ulz - Morgan Stanley - Analyst : Great. Thanks for that introduction. And maybe we could just stop -- start -- sorry, with IMCIVREE. You touched on this a little bit, but your currently approved indications and kind of how to think about the growth drivers today and what that looks like kind of going forward?

Question: Michael Ulz - Morgan Stanley - Analyst : Great. You talked about your increasing confidence just in the sort of market opportunities here in the US for HO. And maybe talk a little bit about what drove that sort of thinking? And is it possible that maybe 10,000 is -- could be conservative maybe?

Question: Michael Ulz - Morgan Stanley - Analyst : Yeah. Makes sense. Maybe could you talk a little bit about Japan? You mentioned that as a potential important opportunity for you and maybe some of the dynamics in that market versus what you see in the US.

Question: Michael Ulz - Morgan Stanley - Analyst : And maybe if we just move back to the US opportunity and maybe just highlight some of the unmet need or how these patients are treated today or what are their options?

Question: Michael Ulz - Morgan Stanley - Analyst : Yeah. So very high unmet need. And you started to touch on some of the data you saw, and maybe you can expand on that a little bit. You've shared results from the Phase II TRANSCEND study, so maybe highlight some of the key takeaways and data points there.

Question: Michael Ulz - Morgan Stanley - Analyst : Some very promising results there. And as you mentioned earlier, you've already filed with the FDA and got an acceptance with the PDUFA date later this year in December. So maybe you can just talk about interactions with the FDA and how that's gone, just given all the sort of changes in the leadership we've seen this year.

Question: Michael Ulz - Morgan Stanley - Analyst : Sounds good. You're also planning an investor event in a couple of weeks to kind of maybe shed a little bit more light on how you're thinking about the opportunity and launch. So maybe just talk a little bit about what things we should expect there.

Question: Michael Ulz - Morgan Stanley - Analyst : Can you talk a little bit about your current sort of infrastructure and how you leverage that and like how much of a lift is this to sort of group in HO?

Question: Michael Ulz - Morgan Stanley - Analyst : Great. And you're also sort of in discussions with Europe and filing there, maybe just sort of give us any updates.

Question: Michael Ulz - Morgan Stanley - Analyst : Makes sense. Maybe I can just go back to Japan, and we spoke about the opportunity there, but you're going to have some data on those 12 patients coming up, I think, early next year, if I remember, -- how do we think about that data? Or any reasons to think that those patients may be responding differently? Or is there any impact of like baseline characteristics or things like that?

Question: Michael Ulz - Morgan Stanley - Analyst : Got it. Maybe we can shift gears a little bit to your next-generation MC4Rs, and you've got two. So maybe at a high level, just walk us through those two programs and what's different about them.

Question: Michael Ulz - Morgan Stanley - Analyst : If I could just ask a follow-up on the hyperpigmentation. AAnd maybe you could just describe what it -- how it presents in -- with setmelanotide and then maybe contrast that to what you saw with bivamelagon. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 09, 2025 / 3:30PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Michael Ulz - Morgan Stanley - Analyst : Yeah. Makes sense. Earlier, you talked about the bivamelagon sort of Phase III trial design, and kind of what you're considering there? I guess maybe talk about when you can start that and what are the next steps to actually getting that started.

Question: Michael Ulz - Morgan Stanley - Analyst : Makes sense. Yes. So a lot of opportunity for your next-gen MC4Rs. And maybe we can shift gears and talk a little bit about Prader-Willi. You've got a Phase II study ongoing. Maybe talk about the design there and what you learned from your prior experience in Prader-Willi.

Question: Michael Ulz - Morgan Stanley - Analyst : Yeah. So you plan to give an update by the end of the year? And will you have how many patients --

Question: Michael Ulz - Morgan Stanley - Analyst : Okay. Yeah, yeah. Goals. But I guess when you eventually share the data, maybe how many patients, how long will it all be followed up post six months? Or what does that look like? And then what are you looking to see to sort of say, okay, move this forward?

Question: Michael Ulz - Morgan Stanley - Analyst : Okay. Great. Looks like we're just about out of time. So why don't we end it there. Thanks so much, David. Appreciate your time.