Rhythm Pharmaceuticals Inc Q4 2024 Earnings Call Transcript

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

Question: Derek Archila - Wells Fargo - Analyst : Hey, good morning and congrats on the progress. Just two questions from us. Just first, could you comment on whether you're going to share the sad mad portion of the RM 718 study separately or will that kind of be wrapped into the second half 25 update with part C? And then just on in Siri's phase 3 trial on HO, I was wondering if you could just remind us the mix of adults and pediatric patients you expect and ultimately will the split be the same in the Japanese cohort. Thanks.

Question: Seamus Fernandez - Guggenheim Securities - Analyst : Oh, thanks so much for the question. So, David, you're emphasizing a lot the market opportunity NHO being quite firm at the, sort of 5,000 to 10,000 patient range, interested to get a sense of, with success, how you would see uptake in that population just more identifiable. Than the BBS patient population or perhaps even the genetic population, so interested to just get a better understanding there. And then as we've spoken with some thought leaders in the space, there's a little bit of a discussion around sort of the pre-existing obesity set point. As a, potential contributor because of the hormone replacement dynamics of treatment with selannoide. Just interested to know, if you feel like that could have an impact on the phase 3 HO trial results in any direction given the mix of patients that you just talked about in the phase 3 study. Thanks.

Question: Seamus Fernandez - Guggenheim Securities - Analyst : More than that there was a pre-existing set point prior to the injury and that, it could sort of limit the magnitude of benefits depending on the timing of that injury.

Question: Phil Nadeau - TD Cowen - Analyst : Good morning. Thanks for taking our questions too from us as well, diving into the HO pivotal data a bit more deeply in your prepared remarks, you said you expect Se Melannoide to do well on the primary point. I'm sure the question we're going to get from investors is what does management think well is, I guess, in the spectrum of 5% weight loss. which is the regulatory hurdle to 25% BMI reduction, which is what you saw in the The long term extension kind of where in that in that spectrum is quote well. That's the first question then second one for Hunter in terms of Q1 trends, just trying to assemble all the data points you gave us it sounds like there was $4 million of inventory bill in Q4. Is that likely to be destocked in Q1 and therefore is a modestly down quarter of a quarter possible or is that going to be absorbed in the channel and bled out more slowly?

Question: Whitney Ijem - Canaccord Genuity - Analyst : Hey guys, thanks for taking the question. I just wanted to follow up on Seamus's question around prevalence in HO and I guess some of the commentary earlier on the call, just around the prevalence numbers that have been quoted, I think largely being based on cranial pharyngioma data. So is there any work that, I mean, I know there's work, so can you speak to any of the work ongoing, in terms of the prevalence that might come from other ideologies including the congenital form?

Question: Tazeen Ahmad - Bank of America - Analyst : Hi, good morning. Thanks for taking my questions. David, I wanted to ask a little bit more detail about, I know what you said about your thoughts but not really, being able to project. What results you'll get other than knowing that this is an active drug and that it works over time. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 26, 2025 / 1:00PM, RYTM.OQ - Q4 2024 Rhythm Pharmaceuticals Inc Earnings Call But what have doctors told you about what they'd like to see in terms of weight loss in order for them to want to be using it right away in HO patients? And then my second is more of a financial one. You completed your ATM, but you do have this HO data set readout coming up very soon. would you rule out doing a cash raise after that data set reads out, assuming that it's positive? Thanks.

Question: Joseph Stringer - Needham and Company - Analyst : Hi, good morning. Thanks for taking our question. Just on the phase 3 HO trial, the inclusion criteria. Has slightly different age groups compared to the open label phase 2 where phase 3, it's including 4 to 6 year olds and then also patients older than 40. So, can you describe what impact the inclusion of each of these groups of patients could have on the phase 3 be my primary endpoint both in terms of magnitude of effect and overall data variability. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 26, 2025 / 1:00PM, RYTM.OQ - Q4 2024 Rhythm Pharmaceuticals Inc Earnings Call

Question: Leland Gershell - Oppenheimer - Analyst : Hey, good morning. Thanks for the update, and we have two questions. First, I just want to ask, it's early days, but since the label expansion to younger just want to see to what extent expansion of the younger ages is becoming a component of commercial uptake and then also want to ask as you look to enroll patients with Congenital, HO if you could just review with us kind of maybe the similarity in governs in the way those patients are identified, versus your other historical congenital, genetic therapy.

Question: Dennis Ding - Jefferies - Analyst : Good morning. This is Anthea on for Dennis. thank you for taking our questions. 2 from us on the upcoming PAHO trial, could you talk a little bit about glip one use at baseline and if drop-ins are allowed throughout the trial and how you anticipate that to impact efficacy? And then on the bio phase two, do you see any read-throughs from the Seaha trial in terms of efficacy as well as enrollment and the adult pediatric fix? thank you very much.

Question: Dennis Ding - Jefferies - Analyst : Yes, do you see any read throughs from the seminatlannoide trial in terms of efficacy and then also in terms of enrollment and the adult pediatrics.

Question: Dennis Ding - Jefferies - Analyst : Got it thank you.

Question: Ram Selvaraju - H.C. Wainwright - Analyst : So much for taking my questions and congrats on all the progress recently. I just wanted to ask if You could comment a little bit more on the recent impact of the label expansion for ivy and what specific dynamics you're seeing in the US market regarding patient acquisition, patient adherence, and how that compares to the ex-S experience that you've had since that label has been in place for longer outside of the US. And the second question is, if we think about The Trispera relationship that you have announced. To what extent is this likely to be a template or a model for future relationships in ex-S territories? Is this likely to inform to a significant extent your future ex-USBD activities, or is it very much kind of individualized to the Turkish context? thank you.

Question: Unidentified Participant - Citizens JMP - Analyst : Hey, thanks for taking the question. Just wondering, in the phase.

Question: Unidentified Participant - Citizens JMP - Analyst : We did see some patients who dose reduced due to AEs, see a regain of BMI, which, gives us confidence in the drug effect, but wondering in the phase 3 protocol if you're allowing dose reductions for AEs during the 52 week period or if everyone's set after the dose titration period.

Question: Unidentified Participant - Citizens JMP - Analyst : Okay, and one more if I may. You talked a little bit about the Vivimegaon and 718 being having less activity at MC1, wondering, can you talk about the relative activity at MC4 between the next gens and Semiennoide? You say, efficacy is kind of TBD if there's going to be a difference there, but you talk about the specificity of the MC4R receptor between those three candidates.

Question: Unidentified Participant - Citizens JMP - Analyst : Got it. Alright, thanks.