Rhythm Pharmaceuticals Inc at Oppenheimer Healthcare Life Sciences Conference Transcript

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

Question: Leland Gershell - Oppenheimer - Analyst : There is a way for you to submit questions, so please go ahead and do so if you'd like, and I can work those into the discussion. But maybe just to sort of set the stage for those who may be less familiar with Rhythm, maybe, David, if you could just give us an overview of the company's approach to rare genetic obesity disorders and how your product, IMCIVREE, fits into that strategy?

Question: Leland Gershell - Oppenheimer - Analyst : And the melanocortin-4 pathway, obviously, very distinct from the GLP-1 receptor and pathway that I think many people are familiar with. Maybe, David, just run us through briefly kind of the key distinctions and applicability of MC4 modulators versus the GLP-1 agonist.

Question: Leland Gershell - Oppenheimer - Analyst : Great. And you've been commercializing in in some very rare genetic obesity. You were also developing this product for what could be a much larger opportunity in hypothalamic obesity or HO. We're looking forward to data. I believe it's going to be in the first half of this year from the study that you're currently running. 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 12, 2025 / 1:40PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Oppenheimer Healthcare Life Sciences Conference Maybe if you can give a sense of the numbers of patients out there with HO. I believe you've given 5,000 to 10,000. Maybe it could be a bit more than that. We seem to have some optimism for higher numbers from our discussions with physicians, but maybe in terms of the diagnosis and the nature of this disorder versus genetic, this is comes from an injury to the hypothalamus and so patients are presumably much better identified, is that right?

Question: Leland Gershell - Oppenheimer - Analyst : Right, and as you say, an approved drug could certainly change that. But let's talk a bit about the Phase 3 trials. So that's a year-long study, 2 to 1 randomized reading out, as I mentioned, first half. So you're looking at the change in BMI and remind me, (inaudible) was it a 12% difference?

Question: Leland Gershell - Oppenheimer - Analyst : So there's been a lot of investor focus on what this exact difference is going to be. Obviously, we saw, as you mentioned, very strong and consistent data in the prior study. Anything you can share with us in terms of your outlook on maybe beating that number, coming in around that number? Again, I have to ask the question because that's the question I get from lots of people. 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 12, 2025 / 1:40PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Oppenheimer Healthcare Life Sciences Conference

Question: Leland Gershell - Oppenheimer - Analyst : Great, that's terrific. And we did see some encouraging data from early access program in France. So we saw a handful of patients there at ObesityWeek also seemed to be supportive for this opportunity.

Question: Leland Gershell - Oppenheimer - Analyst : Terrific. Yeah, and so let's talk a bit about the current commercial. So you've been selling IMCIVREE now for a few years. You've got Bardet-Biedl, a few other rare disorders, maybe just, David, share kind of what you've learned in that process and how that's been moving? Had a very nice quarterly update for us last month, so it seems like the Bardet-Biedls are really kind of catching fire, but interested to hear your thoughts.

Question: Leland Gershell - Oppenheimer - Analyst : I think it's very important for people to realize that with this type of opportunity as we've seen with Genzyme and other rare disease companies, with Rhythm and the like, is that you have this very long and expansive opportunity. So it seems like we're just barely kind of getting going here, and you're also expanding into another disorder which I'm very close to given other research interests, but Prader-Willi, and you've mentioned making lives easier for the families, the parents. I mean, Prader-Willi is a very disruptive disorder. They would just love to hear your thoughts as you enter a study in Prader-Willi. I know you've looked at [Setmelanotide] before, but maybe just hit the highlights and current development.

Question: Leland Gershell - Oppenheimer - Analyst : Perfect. Yeah, we look forward to those data. Yeah, you have a couple of candidates that are behind, so to speak, IMCIVREE, one's an oral, one's a once weekly injectable, both are differentiated clinically as well. It seems like you're going to be developing kind of both of those for maybe the larger indications going forward. Maybe just share with us your outlook on how you're going to be deploying resources toward those two assets?

Question: Leland Gershell - Oppenheimer - Analyst : Perfect. I think you had about $300 million in your last reported cash runway into 2026. Maybe just as we think about kind of the financial side of Rhythm, your approach toward profitability and additional investments in R&D and very kind of balancing that with commercial spend.

Question: Leland Gershell - Oppenheimer - Analyst : Excellent. So we'll obviously be looking out for the HO data. Any other milestones? Obviously you have, the (inaudible) study looking at some other genetic subtypes and. 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 12, 2025 / 1:40PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Oppenheimer Healthcare Life Sciences Conference

Question: Leland Gershell - Oppenheimer - Analyst : Great, we look forward to all that. And David, thank you very much for joining us. This has been a great discussion and thanks to all of you who've tuned in for this fireside chat with Rhythm Pharmaceuticals. Have a great rest of the conference.