Rhythm Pharmaceuticals Inc Q1 2025 Earnings Call Transcript

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

Question: John Woods - Citizens - Analyst : Hey thanks for taking the question and congrats on the update. David, I hope you could talk a little bit more about those non-responders you highlighted. It sounded like several of them were having good responses but dropped out. So what were the reasons for those discontinuations?

Question: John Woods - Citizens - Analyst : Can you talk just a minute about real world persistence, and, understanding of, expectations for adverse events and how that understanding keeps patients on drugs or not, like, what's the real world experience and how does that translate from the clinical data? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 07, 2025 / 12:00PM, RYTM.OQ - Q1 2025 Rhythm Pharmaceuticals Inc Earnings Call

Question: John Woods - Citizens - Analyst : That's helpful. Thanks for the colour.

Question: Derek Archila - Wells Fargo - Analyst : Hey, good morning, and thanks for taking the questions. Congrats on the progress here. Just two quick ones from us just for Part C, for RM 718. I know you highlighted enrollment will complete by the end of the year, but we just wanted to confirm whether you actually plan to share any efficacy data for patients reaching 16 weeks before the end of the year. And then the second question for Hunter, you noticed some changes on compliance during the quarter, so I was wondering if you could just elaborate on that and I guess how we should be thinking about that trending for the rest of the year. Thanks.

Question: Tanzina Ahmed - Bank of America - Analyst : Hi, good morning. Thanks for taking my question. I'm sorry if this was part of Yann's comments. I couldn't hear him clearly, but can you just talk to us about the importance of these continual data updates that you're expecting to make now at conferences going forward? It's our understanding from companies that we've spoken to or doctors that we've spoken to that they think that the data is pretty much perfect. So what additional data do you think is necessary?

Question: Tanzina Ahmed - Bank of America - Analyst : Yeah, and then just on questions around seasonality, I just want to clarify, are you expecting to see any seasonality for the remaining quarters of the year?

Question: Tanzina Ahmed - Bank of America - Analyst : Okay, thank you.

Question: Phil Nadeau - TD Cowen - Analyst : Good morning. Congrats on progress and thanks for taking our questions. A few from us first, David, you mentioned in your prepared remarks that you have an FDA meeting scheduled. Can you discuss what elements of the filing need to be out with the FDA at this point? Any uncertainties or is the meeting more a formality?

Question: Phil Nadeau - TD Cowen - Analyst : Got it the second question is on the Q4. Hunter, you noted that there was somewhat of an impact because of the time to get insurance reauthorizations and the bridging of patients on for drugs. Could you quantify that impact in the quarter? Do you have a sense of how much revenue was hit by that in particular? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 07, 2025 / 12:00PM, RYTM.OQ - Q1 2025 Rhythm Pharmaceuticals Inc Earnings Call

Question: Phil Nadeau - TD Cowen - Analyst : Perfect and then, last question is on the payer mix. Can you remind us what the payer mix is for BBS between Medicare and Medicaid and private pay, and where do you expect that to go for the HO population?

Question: Phil Nadeau - TD Cowen - Analyst : Great, thanks for taking our questions.

Question: Seamus Fernandez - Guggenheim Securities - Analyst : Great, thanks for the question. So, to really kind of focused in on the Bivi Meigon opportunity and how you see this advancing in the pace at which you can kind of bring that product forward should the phase two program deliver the profile that you anticipate, which again if you could just remind us, the profile that you're expecting, I recall David you saying that. Your hope for that product is that it would deliver efficacy comparable to what we've seen in HO, with setmelanotide, but excluding the impacts of MCR1 and the hyperpigmentation. So that's my first question.

Question: Seamus Fernandez - Guggenheim Securities - Analyst : Great, and a follow up to kind of pipeline opportunity and and questions there, and the PWS opportunity, can you talk a little bit more about how you see that opportunity potentially emerging? For MC4R specifically targeted agents, largely because we know that the first effort, kind of missed the mark, but I know you're testing higher doses, so. Trying to get a better sense of the PWS opportunity that you see for the product and if there are additional learnings that have kind of brought you forward to the willingness to kind of pursue a higher dose here. Thanks.

Question: Seamus Fernandez - Guggenheim Securities - Analyst : Great, thanks so much.

Question: Whitney Ijem - Canaccord Genuity - Analyst : Hey, thanks for taking the question. Just wanted to follow up on HO and kind of the launch trajectory versus BBS. I know you've talked a lot about the specifics of the HO patient population that should that hopefully will lead to kind of faster uptake or a different launch trajectory. I think most of those comments have been thinking about the US or contemplating the US. But it seems those same things should be the case in Europe with potential for maybe even. More momentum just given some of the early access progress that's been made on the HO front there. So just curious if you can talk a little bit more about how to think about HO uptake in Europe specifically relative to what we've seen in BBS so far.

Question: Whitney Ijem - Canaccord Genuity - Analyst : Got it. That's helpful. And then thinking about the label, can you just remind us how you are thinking about that currently? I know I think we're expecting an acquired HO specific label, but should we be thinking about BMI requirements or age or time from injury, any other kind of parameters that we should be thinking about?

Question: Whitney Ijem - Canaccord Genuity - Analyst : Awesome, thanks so much.

Question: Dennis Ding - Jefferies - Analyst : Hi, good morning. Thanks for taking the question. Just one for me. So consensus has HO revenue ramping from around $55 million to $60 million in 2026 to a billion plus in 2030. So there's quite a steep ramp in just four or five years of launch. Can you talk about your level of confidence that the launch will be solid and what are the different moving parts that could drive revenue higher or lower than consensus? Thank you.

Question: Dennis Ding - Jefferies - Analyst : Great, thanks for the call.

Question: Leland Gershell - Oppenheimer & Co. Inc - Analyst : Hi, good morning. Great to see the consistency across the age subgroups in the HO study. A couple of questions from us first, question for Hunter, apologies if I if I missed it in the discussion about inventories, but should we expect lumpiness this year as we model quarterly revenue? Should we think about another fourth quarter kind of inventory stocking? And also a question for Jennifer, just on your comments about the about a third of first-time prescribers, turning to repeat prescribers, is that particularly limited by the number of patients who fall under in current indications or are there other factors that may be limiting the a prescribers becoming repeat prescribers. Thank you.

Question: Leland Gershell - Oppenheimer & Co. Inc - Analyst : That's very helpful. Thanks very much.

Question: Selena - Morgan Stanley - Analyst : Hello, this is Selena on for Mike. Thanks for taking our question. For the congenital HO substudy, could you describe the patient mix that you're seeing among the different pituitary deficiency disorders included and any learnings on reliably diagnosing congenital HO? Thank you.

Question: Selena - Morgan Stanley - Analyst : Okay. Thank you.

Question: Joseph Stringer - Needham & Company - Analyst : Hi, thanks for taking our questions. For the instant relaunch of BBS, can you disclose the number of written TRX and reimbursed TRX in the US in the first quarter?

Question: Joseph Stringer - Needham & Company - Analyst : Okay, thank you.

Question: Raghuram Selvaraju - H.C. Wainwright & Co. - Analyst : Thanks very much for taking our questions. Firstly, I was wondering if you could comment on any qualitative differences that you expect to see and implement in the context of the commercial rollout in hypothalamic obesity relative to the other indications for which is already approved. I'm just trying to look here for, any changes in broader marketing and promotional strategy that you might utilize in this specific indication.

Question: Raghuram Selvaraju - H.C. Wainwright & Co. - Analyst : Great. And then secondly, very quickly, in the wake of the rare stone termination, I was just wondering in the context of the broader international strategy, how you intend to go forward in China if you are looking at the possibility of bringing in a replacement partner or if your assessment of the China commercial opportunity you know has kind of signaled an evolution in your thinking about how best to penetrate that opportunity. Thanks.

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

Question: Unidentified Company Representative 1 - - Analyst : Hey, sorry about that. I think I saw you earlier. I appreciate you putting me in. Just going back to Balon really quickly, anything you guys can say about safety, from the study, given that it's pretty far along now, anything just on the idiosyncratic safety side related to live. Or anything else that kind of you're seeing that would give you more comfort in the molecule and then I just wanted to clarify David on the bar, I mean 10% makes sense, but this study is meaningfully shorter than the setmelanotide phase 3. So how do you think about the bar for the shorter phase 2. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 07, 2025 / 12:00PM, RYTM.OQ - Q1 2025 Rhythm Pharmaceuticals Inc Earnings Call