Rhythm Pharmaceuticals Inc at Stifel Healthcare Conference Transcript

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

Question: Dae Gon Ha - Stifel - Analyst : As I always do, I figured we would start with a broad overview of Rhythm, which is probably redundant, but nevertheless, let's get in. Go ahead, and get started.

Question: Dae Gon Ha - Stifel - Analyst : So let's start with the epi of HO. I think most recently that has perked up in our conversations. Your estimates based on some literature analysis has arrived at 5,000 to 10,000. Craniopharyngiomas, astrocytomas, hamartomas are some of the more major tumor types. But you also group sort of the other in the sort of a smaller subgroup. At this point, what's the latest take or latest estimate? I mean, is it really 5,000 to 10,000 you put out there, or are you just being conservative as patients will most likely come to the floor once a drug is approved?

Question: Dae Gon Ha - Stifel - Analyst : Okay. Are you comfortable putting a little bit bigger of a benchmark in terms of are we talking 2x higher when you think about conservatism 3x higher? Because we've heard some ridiculous numbers that came out of a doc meeting.

Question: Dae Gon Ha - Stifel - Analyst : Good segue way. By the way, congrats on that update this morning. Four new French patients, a gift that keeps giving, it seems. Just briefly remind us what you found there. How does it compare to the Phase 2 11 pediatric patients that you enrolled? I know two of these patients that you announced this morning was congenital, like you mentioned, so quite different from acquired. But from a magnitude standpoint, treatment benefit-wise, what have you seen across these four patients?

Question: Dae Gon Ha - Stifel - Analyst : Right. That 39 number, the 39-patient, 34-week study just piqued my interest a little bit. 34-week treatment duration is slightly shorter than the Phase 3, which is 52 weeks in the maintenance portion. And then 39, given the limited amount of data that you have on the congenital side, I was kind of curious if you can talk a little bit more about the powering. How did you arrive at that 39 number? And is this sufficient when it comes to a slightly shorter trial duration?

Question: Dae Gon Ha - Stifel - Analyst : Okay, okay. So to be clear, you haven't necessarily ran this by the FDA, this sub-study proposal.

Question: Dae Gon Ha - Stifel - Analyst : Yeah. Okay, got you. The epi on this segment of the patient population, you mentioned on the press release, about 1,000 plus. This being a congenital, just kind of curious. You've been having this VOOS assessment that you've been funding, right, to try and uncover patients who might have genetic obesity. Is this congenital HO going to be included in that, or is it already included in that? How should we think about maybe working to expedite uncovering these patients? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 18, 2024 / 4:30PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Stifel Healthcare Conference

Question: Dae Gon Ha - Stifel - Analyst : Is that included in the newborn screening of any kind?

Question: Dae Gon Ha - Stifel - Analyst : Subsequent to the genetic diagnosis, presumably.

Question: Dae Gon Ha - Stifel - Analyst : Got you. So the Phase 3 and now with the sub-study, you mentioned 10% for the main study and then the 12% difference. Just to be clear, is that difference to the baseline or to the placebo arm?

Question: Dae Gon Ha - Stifel - Analyst : Placebo-adjusted.

Question: Dae Gon Ha - Stifel - Analyst : So how are you thinking about --

Question: Dae Gon Ha - Stifel - Analyst : Okay. You just read my mind. That was going to be my question. On the patient enrollment to the Phase 3, can you also just briefly remind us how much are or have been on GLP-1s before? How many are currently receiving GLP-1s? And what is the protocol mandate in terms of adjusting their dose, if you will?

Question: Dae Gon Ha - Stifel - Analyst : Going back to the French real world adult data from obesity week, you mentioned five patients out to six-month data. The fifth patient drew my interest a little bit in the sense that their magnitude or his or her magnitude was not as robust as the other four. And I remember there was a substantial amount of hormonal therapy as well as GLIP-1 therapy that still participated in the trial. Do we know anything about any baseline characteristics or concomitant medications that has led to a little bit of that disparity?

Question: Dae Gon Ha - Stifel - Analyst : Right, the uniformity was striking. So quickly touching on the timing, first-half '25, a little bit of misunderstanding as to why it's first half, not a first Q or second Q. So I know there are some moving pieces to the two portions of the study, right, dose titration and the data readout. Can you just remind us why you're sticking with the first half and what those moving pieces are?

Question: Dae Gon Ha - Stifel - Analyst : Yeah, got you. By the way, if there is any question from the audience, feel free to chime in. Let's quickly pivot to Bardet-Beidl. HO clearly has become the dominating force when it comes to Rhythm, but Bardet-Beidl has been a sort of the tortoise and the hare kind of a situation where it continues to build, build, build. What's your latest assessment of the Bardet-Beidl opportunity, the next growth higher? What can you kind of do in terms of maximizing the growth opportunity in Bardet-Beidl?

Question: Dae Gon Ha - Stifel - Analyst : Right. And then European rollout, what has been sort of your track record, or I guess not track record, but what have you been seeing from their

Question: Dae Gon Ha - Stifel - Analyst : Just to clarify, NIH or NHS?

Question: Dae Gon Ha - Stifel - Analyst : NHS, okay.

Question: Dae Gon Ha - Stifel - Analyst : So let's briefly touch on your pipeline expansion candidates. You now call 54640 bivaelagon, which is a little bit of a tongue twister, and 718. What's your long-term strategy with these two candidates? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 18, 2024 / 4:30PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Stifel Healthcare Conference And clearly, you're evaluating HO for both. Any sense of when we should be expecting those data readouts vis-a-vis the main Phase 3 data readout?

Question: Dae Gon Ha - Stifel - Analyst : Got it. And then just briefly touching on finances, your cash balance and runway projections.

Question: Dae Gon Ha - Stifel - Analyst : All right. Well, with that, David, thanks very much. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 18, 2024 / 4:30PM, RYTM.OQ - Rhythm Pharmaceuticals Inc at Stifel Healthcare Conference