Rhythm Pharmaceuticals Inc To Discuss Topline Results Transcript

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

Question: Derek Archila - Wells Fargo Securities, LLC - Analyst : Hey. Good morning, and let me be the first to congratulate you on the data here. Well done. So just two questions for us, one for Dr. Phillips and then one for the Rhythm team. So for Dr. Phillips, maybe could you just discuss how common seizures and seizure disorders are in HO patients? And maybe just put some context around the broader health issues HO patients face beyond just the weight gain and the hyperphagia. And then just one simple one for the Rhythm team. Just curious to know what percent of patients in the trial were able to normalize their BMI completely. Thank you.

Question: Derek Archila - Wells Fargo Securities, LLC - Analyst : That is correct. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 07, 2025 / 12:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc To Discuss Topline Results

Question: Derek Archila - Wells Fargo Securities, LLC - Analyst : Excellent. Congrats, again. And thanks for taking the questions.

Question: Phil Nadeau - TD Cowen (Research) - Analyst : First of all, our congratulations on the data. Great day for Rhythm. Two questions from us, as well. First, for Dr. Phillips. Dr. Phillips, could you give us some sense of how you'll integrate IMCIVREE into your treatment paradigm for HO patients now that you've seen the Phase 3 data? That's the first question. And then, second, for Rhythm, we're curious whether there was any correlation in the study between the changes in hyperphagia scores and BMI reduction. Thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 07, 2025 / 12:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc To Discuss Topline Results

Question: Phil Nadeau - TD Cowen (Research) - Analyst : Is it safe to say you'd use it in all your patients with weight gain in --

Question: Phil Nadeau - TD Cowen (Research) - Analyst : Thank you.

Question: Phil Nadeau - TD Cowen (Research) - Analyst : Fair enough. Congratulations again on the data.

Question: Jeremiah Lorentz - BofA Securities, Inc. - Analyst : Hi, this is Jeremiah Lorentz on for Tazeen. Thanks for taking our question, and congrats on the Phase 3 results. We were wondering if you could provide any color on the profile of acquired HO patients that were unable to achieve the greater than 5% BMI reduction at 52 weeks. And I know you had highlighted that there was a case where one patient saw BMI reduction accelerate during the open-label extension portion of the study. Just curious on how you think this phenomenon could potentially affect how healthcare providers are thinking about the treatment trial periods when they utilize in the real world. Thanks.

Question: Jeremiah Lorentz - BofA Securities, Inc. - Analyst : Got it. Are you able to provide any color on the average trial period for patients for, let's say, BBS patients in the real world?

Question: Jeremiah Lorentz - BofA Securities, Inc. - Analyst : Yeah. I guess, how long are KOLs willing to have patients on drugs before they classify them as either a responder and non-responder?

Question: Jeremiah Lorentz - BofA Securities, Inc. - Analyst : Got it. Okay, thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 07, 2025 / 12:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc To Discuss Topline Results

Question: Seamus Fernandez - Guggenheim Securities LLC - Analyst : Great, thanks for the question. So a couple here. I just wanted to ask, I think there was one patient in the open-label period that suffered a cortical stroke. Just wanted to get a better sense of the patient history there, if you could help us understand that, and if that cortical stroke was attributed to a drug or not. And then, as it relates to the question around the patient who experienced the seizure in the study, it may not be sort of a frequent occurrence with setmelanotide or in this patient population, but what were the underlying kind of characteristics of the patients? Was it really related to -- were the seizures related to sort of an underlying epileptic condition that the patient was allowed under the study for just trying to get a little bit more color around those details? And then just my last question, when you look at the progression of these patients to other sort of related conditions -- this is a question for the doctor -- the progression to diabetes and perhaps even the ability to prevent progression to diabetes or reverse it, how would you sort of predict the setmelanotide might actually benefit patients in that regard and really contribute to reducing costs within the system? Thanks.

Question: Seamus Fernandez - Guggenheim Securities LLC - Analyst : Yes, that's perfect. Thanks so much.

Question: Paul Matteis - Stifel Nicolaus and Company, Incorporated - Analyst : Hey, let me get my congrats, and thanks for taking my question. Two data questions and then one regulatory question. So on the data, I appreciate the granularity you guys have shared. I don't know if you have this data point, but can you just talk about median versus mean and any meaningful difference there? Second, on the SAEs that occurred in at least one patient, any more color you can give on what was observed on the drug arm and your confidence that there's no new safety signal that's emerged in the study? And then lastly, David, I appreciate your comments on the kind of uncertain environment on the background FDA set at the beginning of this call. I know you guys are watching this all play out like we are. Do you have any insight into your review division specifically for this sNDA as it relates to the continuity of the leadership or review team levels? Any comments you can make would be helpful. Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 07, 2025 / 12:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc To Discuss Topline Results

Question: Paul Matteis - Stifel Nicolaus and Company, Incorporated - Analyst : It was on the SAEs, so it's probably at least one patient and just any more color you can give.

Question: Whitney Ijem - Canaccord Genuity - Analyst : Hey, guys. Adding my congrats on the data. Quick follow-up on the GLP-1 patients. I know you showed the placebo-adjusted BMI change. What was the actual placebo change? And what was the end of that placebo change? And then one for Dr. Phillips, just a follow-up question on kind of how you plan to use this in your practice. Just to confirm, are there any patients, for whatever reason, you think would not be a good fit for setmelanotide based on the data today? And just how do you think about the potential

Question: Whitney Ijem - Canaccord Genuity - Analyst : Well, specifically, just because someone had already asked that, but just wanted to confirm, are there any patients who you think would not be a fit for any reason?

Question: Whitney Ijem - Canaccord Genuity - Analyst : Noted. All right. All good. Thank you. Congrats again.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Good morning. Thanks for taking the question, and congratulations on the data as well. Maybe just one for me related to the ongoing Japanese cohort in terms of any potential read-through you see from here. Is it fair to assume similar results in the Japanese cohorts? Are there any sort of key differences maybe in the patient population we should consider as we think through that? Thanks.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Thanks, and congrats again.

Question: Dennis Ding - Jefferies LLC - Analyst : Good morning. Thanks for taking our questions, and congrats on the data. Two from us. On the pipeline, the data from the daily oral and the weekly subcu read out over the next 6 to 12 months. How are you going to compare that data to the setmelanotide Phase 3 data? What are you looking for there and what would constitute a success on your end? And then as a follow-up, I had a question on the dosing in the Phase 3. What was the average dose in the drug arm? And how much incremental efficacy do you think is left on the table as you transition from the daily subcu to a daily oral or even the weekly injectable? Thank you.

Question: Dennis Ding - Jefferies LLC - Analyst : I was just trying to compare things.

Question: Dennis Ding - Jefferies LLC - Analyst : Okay, thank you.

Question: Joey Stringer - Needham & Company, LLC - Analyst : Hi. Good morning. Thanks for taking our questions. We have two for Dr. Phillips. Dr. Phillips, you mentioned before that you would recommend setmelanotide to all of your acquired HO patients. I guess, in your view, what would be the potential barriers to a broad uptake of the drug? And secondly, based on the Phase 3 data and in your experience, how compliant in a real-world setting do you think your average HO patient would be if they were on setmelanotide? In general, do you think HO patients would be more or less compliant relative to, say, Bardet-Biedl patients? Thanks.

Question: Joey Stringer - Needham & Company, LLC - Analyst : Sure. It was around the -- in your view, what would be potential barriers to broad uptake of the drug?

Question: Joey Stringer - Needham & Company, LLC - Analyst : Great. Thank you for taking our questions.

Question: Jonathan Wolleben - Citizens JMP Securities, LLC - Analyst : Hey. Congrats on the data, and thanks for the questions. Two for me. The first clarification, on slide 8, you list seven discontinuations due to adverse events, but then on slide 15, you only have four on the setmelanotide. So I'm wondering about kind of what's the difference there. And then what should we think about long-term use for setmelanotide in HO patients in terms of long-term effects? Are you seeing plateauing of BMI? Should we expect normalization or to get back to prior hypothalamic injury levels? And then how does a growing population factor into that as well?

Question: Jonathan Wolleben - Citizens JMP Securities, LLC - Analyst : The AEs had seven discontinuations and then four in the table. So I'm wondering about the other three.

Question: Jonathan Wolleben - Citizens JMP Securities, LLC - Analyst : Yeah, got it. There was just zero listed for placebo on slide 15. So I was wondering about the difference there. But that's helpful color.

Question: Raghuram Selvaraju - H.C. Wainwright & Co., LLC - Analyst : Thanks very much for taking my questions, and congratulations on the data. I just wanted to ask about what your previous interactions with payers have been regarding the prospects of setmelanotide in hypothalamic obesity. And if you would characterize this data set as constituting either a best case scenario or close to a best case scenario, as far as payers are concerned, what you think their response might be to this data set. And in particular, if you have any sense of how they are going to be approaching the deployment and management of this drug in the context of this population, given the heterogeneity of response, but also the prospect of a significant magnitude of BMI change. Thank you.

Question: Raghuram Selvaraju - H.C. Wainwright & Co., LLC - Analyst : Thank you.