Rhythm Pharmaceuticals Inc at Needham Healthcare Conference (Virtual) Transcript

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

Question: Joey Stringer - Needham & Company - Analyst : David, thank you so much for joining us today.

Question: Joey Stringer - Needham & Company - Analyst : Well, we'll start off with the IMCIVREE launch. We'll get right into it. How would you [handicap] the launch to date in BBS? Is it tracking with internal expectations?

Question: Joey Stringer - Needham & Company - Analyst : In terms of the launch metrics today for IMCIVREE, can you provide a summary of those? 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 17, 2023 / 7:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Joey Stringer - Needham & Company - Analyst : And in terms of the launch to date, what --?

Question: Joey Stringer - Needham & Company - Analyst : Yeah. Important point on that. But in terms of the biggest surprises to date, what have been the biggest positive surprises about the launch to date with your -- or the internal expectations? And on the other side of that, what are some of the headwinds that you've encountered?

Question: Joey Stringer - Needham & Company - Analyst : Great. And in terms of the total prescriptions to date, they've been added at a pretty steady rate since launch. Kind of a two-part question around that. The first one is, what are the types of initial BBS patients that are being added to commercial drug or to the launch to date? And do you expect the -- secondly, do you expect the current rate of [TRx] adds to continue over the next several quarters? 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 17, 2023 / 7:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Joey Stringer - Needham & Company - Analyst : No, that is just in terms of the rate of TRx add quarter-over-quarter --?

Question: Joey Stringer - Needham & Company - Analyst : Just expectations on (multiple speakers) for the next couple of quarters.

Question: Joey Stringer - Needham & Company - Analyst : Yeah. And just to follow-up on you mentioned patients transitioning from trial to commercial drug, can you give us the current dynamic of that in terms of what's the expected switch or contribution of that in the next -- going forward? And maybe looking back, how many of the total TRx -- I think you mentioned it, from the last quarter were switching from trial to commercial priority?

Question: Joey Stringer - Needham & Company - Analyst : In terms of the ease of diagnosis for BBS patient and the identification, if a patient is diagnosed and identified, how easy is it for them to get on drug? And what's the typical process like? How long does it take?

Question: Joey Stringer - Needham & Company - Analyst : And you're still in the first year of launch for BBS, just curious if -- what's been the feedback either qualitatively or quantitatively for persistence and compliance rate so far? 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 17, 2023 / 7:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Joey Stringer - Needham & Company - Analyst : And you mentioned that discontinuation rates are quite up. Just curious for those that have discontinued, is there any singular particular reason why they've discontinued or is it just varied?

Question: Joey Stringer - Needham & Company - Analyst : And it's sort of related to the previous question on persistence rates, what impact do you anticipate the 5% weight loss BMI stopping rule that's in the IMCIVREE label, what effect do you think that will have on persistence rates? And how do you anticipate this will be handled by payers, physicians, and patients?

Question: Joey Stringer - Needham & Company - Analyst : And last question or two on BBS, just want to get your updated thoughts on the European BBS launch compared to the US launch. You mentioned that the metrics are all US-based now. But on one hand it appears that the European BBS patients, more of them are identified and it's a little bit better organized in terms of the community. But on the other hand, the dynamics of say, staggered country-specific reimbursements coming online. So what are some of the other differences that you anticipate for the European BBS launch compared to how the US launch has gone to date?

Question: Joey Stringer - Needham & Company - Analyst : And then lastly on -- for BBS. Can you give us your updated thinking on the BBS prevalence estimates? And you've previously shown on I think a slide in your corporate presentation on how do you bucket that, the BBS prevalence, in terms of segmentation of diagnosed and IDed versus undiagnosed, et cetera. Give us your updated thoughts on what do you guys are thinking for BBS prevalence and patient ID.

Question: Joey Stringer - Needham & Company - Analyst : Yeah, that's very helpful. And now, we'll switch into one of your other lead indications, hypothalamic obesity or HO. So those who have been following the story, they're familiar with the indication but others may not be as familiar in terms of the similarities and differences to BBS, your BBS indication. So can you describe the unmet need in HO and what are the prevalence numbers for this indication?

Question: Joey Stringer - Needham & Company - Analyst : So you mentioned the Phase 2 HO data being released last year, arguably some of the -- well, the best clinical data for IMCIVREE generated to date. Can you just recap the significance of those results, number one? And then number two, what's been the feedback from physicians and the medical community?

Question: Joey Stringer - Needham & Company - Analyst : Yeah. To the point, I know that you just started the Phase 3 HO trial, but I'm going to jump ahead and ask you a question on what would be the similarities or differences between a potential IMCIVREE launch in HO versus say, what we've seen to date in BBS? Would the trajectories be similar, just given some of the dynamics that you talked about?

Question: Joey Stringer - Needham & Company - Analyst : And in terms of the entire HO addressable patients, just based on the Phase 2 data that you've generated and the feedback that you've gotten from physicians and medical community, what's your estimate of the percentage of the overall HO patient population that would be good candidates for IMCIVREE therapy?

Question: Joey Stringer - Needham & Company - Analyst : Phase 3 trial is underway, but can you just remind us of the design of that trial? And what are the main differences between Phase 3 trial and what you had done in 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. APRIL 17, 2023 / 7:00PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Joey Stringer - Needham & Company - Analyst : That's very, very useful overview and discussion on HO. I guess I want to shift slightly back to the other indications, in particular you're leveraging the drug towards younger patient populations. You have an ongoing Phase 3 trial in two-to-six-year olds that's set to read out in second half of this year. Can you just give us -- or at least describe what types of patients are in this particular trial and what the goal of this trial is?

Question: Joey Stringer - Needham & Company - Analyst : Yeah. In terms of the expectations on the weight loss and BMI reduction relative to what was observed in the patients that were six years old and over, should we expect similar type of results on those efficacy measures? Is that fair or is it little more --?

Question: Joey Stringer - Needham & Company - Analyst : Yeah. No, that makes sense. And in terms of the potential label expansion to this particular age group, how would you handicap the impact on the launch trajectory in terms of -- for example, is there a large bolus to the six-year-old BBS patients, for example, that are -- that's going to be there? Or maybe a little bit more detail on what this would mean if we did get that expansion to that two to six population.

Question: Joey Stringer - Needham & Company - Analyst : No. It makes sense. You have an additional program ongoing, Phase 3 EMANATE trial, looking at other MC4R related pathway disorders. Four independent sub-studies, what are the endpoints for those trials and how long you think it will take to enroll into those sub-studies?

Question: Joey Stringer - Needham & Company - Analyst : And in terms of the four independent sub-studies, can you handicap relative patient population? So prevalence for each of those. Maybe just one through four. And give us a sense for what you're thinking about in terms of the number of patients prevalence wise for each of those?

Question: Joey Stringer - Needham & Company - Analyst : And you mentioned you also have the Basket trial, the Phase 2 DAYBREAK trial, which is exploring 10 or so additional related -- MC4R related genes. You said to announce results in the second half of this year from that trial, what are the important efficacy metrics that we should look for? And what would be the bar for success that would give you confidence to sort of proceed to the next step for say, a particular gene or indication in that Basket study?

Question: Joey Stringer - Needham & Company - Analyst : Makes sense. And last question, David, what's your current cash position and your expectations around cash runway and what are the assumptions that we've built into this?

Question: Joey Stringer - Needham & Company - Analyst : Great. Well, thanks so much, David, for the discussion and participating. It was very useful, I think. And thanks again, to everyone for joining on the webcast. Have a good rest of your day and a good rest of the conference.