Rhythm Pharmaceuticals Inc To Announce Topline Results Transcript

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

Question: Derek Archila - Wells Fargo Securities LLC - Analyst : Yeah, good morning. Thanks for taking the questions and congrats on the data. So just two questions from us. Can you maybe first just give us a sense of the overall patient drug levels in the trial and whether you think maybe Bivamelagon was leaving some efficacy on the table given the pill burden and maybe some of the noncompliance and then just second question, in terms of what you're going to present at ENDO, I guess is there going to be anything incremental from what we saw today that you'll present new at the meeting?

Question: Derek Archila - Wells Fargo Securities LLC - Analyst : Very helpful. And maybe one follow-up. Just in the trial, can you just discuss maybe some of the different HO etiologies that may have been in the trial? And was the efficacy across maybe those etiologies more or less consistent with Bivamelagon?

Question: Derek Archila - Wells Fargo Securities LLC - Analyst : Got it. Thanks so much and congrats on the data.

Question: Tanzina Ahmed - Bank of America - Analyst : Hey, good morning, David thanks for taking my questions Can you just give us a little bit more color on the localized hyperpigmentation? Do you have a sense of why that's occurring? And does the type of hyperpigmentation differ from the types that you're seeing from Setmelanotide in terms of the darkness itself and how long it takes to resolve, let's say, after a patient might discontinue. And then can you just talk about the doses that you plan on taking forward for Phase III? Do you plan on including the 200 mg. There was clearly a dose response, but how do you offset thinking about needing to titrate to deal with the nausea issue versus needing to map out efficacy thanks.

Question: Tanzina Ahmed - Bank of America - Analyst : Okay. And maybe just a follow-up, David, on the hyperpigmentation. So the level of sun exposure has a factor here. I don't know if these patients might have been on trial during the summer season and that kind of affected any of them?

Question: Dennis Ding - Jefferies LLC - Analyst : Hi, good morning. Thanks for taking our questions and congrats on the data. Big picture life cycle management question. Just wondering how you envision positioning some of the second-gen drugs, whether it's the oral or the weekly or both once they're approved? And how would you incentivize switching from Setmelanotide before generics launch? And how does that kind of strategy change after generic launches? And then my second question is just around your prior comments on noncompliance with the oral. Presumably, the weekly subcu could help with some of that. So talk about your expectations for that weekly update. And is it unreasonable to assume efficacy could look better just on better compliance and perhaps less daily PK variability?

Question: Dennis Ding - Jefferies LLC - Analyst : And what about after a generic is launched and maybe talk a little bit about your expectations around payer reimbursement in that sort of landscape?

Question: Mike Ulz - Morgan Stanley - Analyst : Good morning. Thanks for taking the question and congrats on the data as well. Maybe just could you talk a little bit about timing of the new formulation tablet? What are the next steps there? And when will that be available? And could that be rolled into the pivotal?

Question: Mike Ulz - Morgan Stanley - Analyst : Great, thank you and congrats again.

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Thanks for being your question and adding our congratulations on the results. Just a question as we talk about dosing between Setmelanotide and biva, if you could remind us, to what extent was Setmelanotide dosing weight-based in its Phase III HO study versus here, you have kind of fixed doses that are titrated wondering if, a, that would make it easier potentially for patients who are in the oral less burdensome in terms of having to titrate as they lose weight. Also I want to ask if you could see patients once they lose a certain amount of weight on biva, could they maybe downgrade to a lower dose as a maintenance and that could also lighten the effects on the skin hyperpigmentation?

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Just wondering if over time, patients you could see patients losing weight and moving to quite a lower maintenance dose with maybe a better. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JULY 09, 2025 / 12:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc To Announce Topline Results

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Got it. And then just one follow-up, if I may. We just noticed in the slides, we have four patients on biva with skin pigmentation and none in the either 600 or placebo in the press release, you also mentioned four patients, but it also has that dose included 1 placebo patient in that sentence. I'm just if you could comment on that discrepancy? Thank you

Question: Leland Gershell - Oppenheimer & Co Inc - Analyst : Got it. Okay, great. Thanks very much.

Question: Jonathan Wolleben - Citizens - Analyst : Hey, thanks for taking the question and congrats on the data. Wondering if you could talk a little bit about the open-label extension. What's the longest follow-up you have today? When do you expect to report data? Do you expect to see continue to benefit over time like we saw with Setmelanotide. And then any comments on if patients are staying at the lower dose in the open-label extension or are people migrating back up to the 600-milligram dose? Thanks.

Question: Joseph Stringer - Needham & Company LLC - Analyst : Hi, good morning. Thanks for taking our question. JJust a follow-up on the noncompliance or I guess, suspected noncompliance. You mentioned some of the patients have trouble taking the large pills, which if they're in the 600-mg cohort, they had to do that 3 times per day. So with the new tablet, you have the once-daily 600 mgs, but pill size looks very similar looking at Slide 19, maybe slightly smaller. So I guess my question is, was the suspected noncompliance mostly due to the fact that the patients had to take 3 pills per day? Or was it simply the size of the pills or was it maybe a combination of both? And then how confident are you that the new tablet can address the noncompliance issue? 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. JULY 09, 2025 / 12:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc To Announce Topline Results