Q2 2025 Rhythm Pharmaceuticals Inc Earnings Call
Now, like to hand the conference over to your speaker today. Dave Connolly investor relations, please go ahead
Thank you Tanya. I'm Dave Connolly here at Rhythm Pharmaceuticals for those of you participating on the conference call our slides can be accessed and controlled by going to the investors section of our website. IR, dox.com this morning we issued our press release. That provides our Q2 Financial results in a business update and that press release is available on our website, our agenda listed on slide 2. Uh our agenda is listed on slide 2 on the call today, our David meager our chairman chief executive officer and president Jennifer Lee Executive Vice President head of North America Hunter Smith Chief Financial Officer and yon mzero Executive. Vice President head of international is on the line joining us from Europe.
On slide 3, I'll remind you that this call contains remarks concerning future expectations, plans, and prospects, which constitute forward-looking statements.
Actual results May differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed on our most recent annual or quarterly reports on on file with the SEC in addition. Any forward-looking statements represent our views as of today and should not be relied upon as representing our views as of any subsequent dates, we specifically disclaim any obligation to update such statements with that. I'll turn the call over to David meager, who will begin on slide 5.
Thank you, Dave. Good morning everybody. Thank you for joining.
Today marks the first earnings call where we can truly start map where we can truly start mapping the long-term future rhythms. Early, startups are beyond the simple struggle to survive often. Don't have the luxury of looking longer term that Rhythm. We have more than survived. And in quarter 2, we laid the foundation for a significant future growth. I'll briefly review those elements on this call.
We have another solid quarter of BBS sales growth. This is important because we are now three years post-launch of an extremely promising, but very challenging opportunity. Our North American International Teams have entered a classic ultra-rare disease community, facing all the challenges that come from a lack of disease awareness, difficulty getting to a diagnosis, or finding an expert, as well as gaining access to the only approved medication.
The projected epidemiology seems right, the patients are benefiting and the Health Care system is working with us. All of that translates to sustainable steady growth, which is what you are seeing this quarter. We expect BBS will be an important part of these quarterly earnings calls for the next 15 years.
In terms of significance, I don't think we have had a more impactful quarter. The Phase 3 readout of Setmelanotide and the acquired hypothalamic obesity in the Phase 3 readout of the first of our two Next Generation compounds sets us on course for our next phase of growth. Although we previously reported those results, I will briefly review them; they're worth revisiting.
We had a productive meeting with the FDA the first in-person meeting in 5 years and we are on track to complete us and European regulatory filings in Q3, we will update you upon acceptance of the filings.
Finally, we're very well capitalized following our recent, oversubscribed 189 million.
On slide 6, I remind you again of the meaningful opportunities ahead of us BBS with an estimated 5,000 patients in the US and similar numbers in Europe, acquired, hypothalamic obesity with 5 to 10,000 patients in the US. And as noted, a growing level of confidence in the upper range of that number was similar numbers. Estimated for Europe, in Japan opportunity looks equally promising.
Finally, we look forward to the emanate readout in the first quarter of next year. Importantly, we have the time to fully develop these opportunities, set, Milani, composition of matter patent is up in 2032. But importantly, the formulation patents extended 2034 in the US. Our next Generation compounds. Will extend that protection to 2040 Plus
On slide 7. We want to share a little more color as to what the patients are experiencing 30 patients or their caregivers who participated in our phase 3 trial of set latitude in a quiet hypothermic obesity, participated in a qualitative 1-hour interview. These results were presented at Endo last month. I encourage you to read the representative quotes on the slide. I'm not going to read them, but these individuals who may have been living a relatively normal life prior to their injury brain tumor, most of these cases. So
We're confronted with rapid weight gain, increased hunger, and a severe preoccupation with food, all accompanied by a lack of control. Once on treatment, they could, as they described it, feel good and find joy in their lives again.
On slide 8, you can see that of the 30 patients participating in the interviews, they almost all experienced an increase in hunger, an increase in fatigue, and a decrease in their physical activity. This disease is not about simply adding a few additional kilograms.
5 9, the set, monotype phase 3, acquired hypothalamic obesity results we reported out in April.
We're hugely validating both in terms of the underlying biology; this is a disease driven by impaired MC4R signaling.
and,
Effect of stimulant, a functional analog of the endogenous hormone alpha-MSH, which had a consistent and meaningful impact on the primary and key secondary endpoints.
As shown here, the placebo adjusted difference was 19.8% reduction in BMI importantly, this result was consistent across all age groups and in both male and female patients.
We are equally excited to get the results of the phase 2 biomg trial. These were the first results in patients and we are learning.
As shown on slide, 10, the placebo cohort, gained weight. There was a clear dose response and the 600 mg cohort decreased their BMI by more than 9%
On slide 11 as you remember, we made our best attempt to draw an Apples. To Apples comparison with a settlement type data at 12 and 16 weeks from The Phase 2 and 3 trials, and acquired hypoclin, obesity, and patients, aged 12 and above
As you can see, the patients decreased their BMI by 9.7% and 10.1% at 12 and 16 weeks, respectively, as compared to 9.3% for the 600 milligram dose cohort at 14 weeks in an intent-to-treat analysis.
We expect 600 milligrams will be our Target dose going into phase 3 trials. We will request an end of phase 2 meeting with the FDA and request scientific advice from the chmp of the EMA to share the data and gain alignment on the design of the phase 3 trial and a path to registration.
Finally algar filled our CSO. And I had the privilege of joining on this team is the improved meeting in Prague. He will describe in Greater detail, but it is a unique event focused on mc4r pathway diseases. While the meeting was more genetically, while the prior meetings were more genetically focused. This meeting had significant discussions about ho and a sharing of some of the early real world treatment experience in Europe.
The fact that approximately 150 Physicians from around Europe, would attend a rhythm, sponsored meeting speaks to the quality of the science, which was shared and the level of trust yawn and his team have built with that community.
Violence, slide 13 tonight. The number of the upcoming Milestones remain on track for us. And EMA filings this quarter for settlement Titan. Ho our goal is to disclose preliminary results from the phase 2 prayer to Willie trial before the end of the year, we aim to complete enrollment of the 718 weekly Phase 2 study in, Ho patients,
26. We'll also release data Topline data from the Japanese cohort from our phase 3, Alpha acquired ho trial in q1 and we will release Topline data from the emanate trial in q1. We aim to complete enrollment of A congenital ho trial in the first half of 2026. And finally, we will initiate our phase 3 study. We build melon and acquired ho in 2026 and we will further refine the timing. Once we have feedback from regulators,
That I don't know. I'll turn the call over.
Thank you, David. I'm going to be starting today on slide 15.
June, 2025 marked the schedule and launch of in BBs.
Continue to grow at a steady pace. We have delivered consistent and steady progress in establishing a serious and robust presence. This includes the first and only therapy that addresses the underlying cause of hyperphagia.
The pathological hunger that leads to abnormal food-seeking behaviors and severe obesity in patients with rare MC4R pathway diseases like Bardet-Biedl syndrome.
We had a strong second quarter, and we continue to see solid growth in new prescriptions and new patient starts, driven by our fine-tuned patient identification efforts.
We are seeing steady growth in new first-time, prescribers and repeat prescribers. Are writing prescriptions for second patients and more following up. Positive, first experience with emcy,
With the label expansion down to 2 years of age received late last year, we are now seeing more patients younger than 18. Come on, therapy, the last 2 quarters.
An importantly, our teams are preparing to launch in Cy and hypothalamic obesity, pending FDA approval.
I'll touch on each of these positive themes from the quarter.
Next slide.
First prescribers.
In the second quarter, we saw a continued growth in the number of MCB prescribers for VBS patients.
We recorded a 38% growth in the cumulative number of BBS prescribers from Q2 2024 to Q2, 22,025.
And the cumulative number of BBS prescribers from q1 2025 to Q2 of 2025.
Next slide.
The FDA approved. Label expansion down to 2 years of age, enabled us to renew engagement across Physicians who treat younger patients.
We leverage the expanded indication to amplify a strong message that Emy due to its efficacy. And safety can be used in patients as young as 2 years of age, differentiating, mc4r diseases and their early onset obesity from the population with General obesity.
This focused messaging resulted in a growth in prescriptions, coming from both the Pediatric and Adolescent patients in Q2.
40% of prescriptions in the quarter were for patients, under 12 years of age up from 27% in q1.
and,
27% of prescriptions in the quarter were for patients between 12 and less than 18 years of age, up from 23% in Q1.
These positive Trends stem from a combination of patients younger than 6 potentially waiting on their label expansion.
As well as moving older. Children. Adolescence onto treatment.
Though we saw positive contribution of prescriptions from younger patients due to our focused messaging around the label expansion. We expect this label expansion to be a minimal incremental opportunity for us, moving forward.
Over these last three years, BBS' commercial performance has continued to be strong.
With improved understanding throughout the Computing, community of the impact of early onset. Hyperfont differentiated from General obesity.
Physicians are engaged in diagnosing patients and writing prescriptions for EIMI.
Payers also appreciate the difference. And while we Face similar challenges faced, by any rare disease, therapy pairs are providing coverage of incorrect for these patients. And most importantly patients are starting and staying on therapy and seeing benefits.
Next slide.
We are excited about the next stage of Rhythm's potential growth and hypothalamic obesity leveraging what we have learned and put in place since the BBS launch.
As David outlined. We are confident that the number of us patients with acquired pipeline obesity is in the upper bound of our 5 to 10,000 prevalence estimates.
We're approaching this as a specialty launch focused on endocrinologists both adults and Pediatrics.
Our ongoing physician profiling and patient, identification efforts are underway and we remain excited to launch upon approval. We will look forward to providing you more details on the ho launch Readiness efforts of September 24th. During our in-person event in Boston, which will also be webcast it
Stay tuned for registration details and feel free to contact Dave Conley with that. I'll turn the call over to you on.
Thank you, Jennifer. I begin on slide 20 and we are pleased to report that in is now available for BBS and orom cd part efficiencies, either as fully reimburse therapy or an ambition sales in more than 20 countries outside the United States.
This includes 2 countries where we have achieved 3 EMA approval paid Early Access for patients with hypothalamic obesity.
We are seeing a steady increase in the overall number of patients on MC3 in the international region. As we are very pleased with the results of the second quarter.
The main goals drivers for the international region. This quarter where IMC cells in approved, indications DBS and Pyar. Deficiencies as they made up the larger increase in patient numbers and paid Early Access for each of patients in France and Italy which drove the largest percentage increase in sequential quarterly growth.
Reimburse. How each of patients now account for Meaningful? Percentage of total reimburse patients in the international region?
As a reminder, in France and in Italy, these early access programs allow patients to gain access to federal reimbursement before the approval in Europe.
Both programs are progressing well and seeing increases in patient and therapy.
And the patients appear to be be benefiting as well.
Previously and use this quarter. We are seeing patients from palande and the Czech Republic.
And in Japan, we are building out our team with a focus on regulatory medical Affairs marketing and Market access.
Next slide.
On slide 21 are more details on the third improved meeting where approximately 150 Physicians scientists and researchers gathered to learn from 1. Another
attendees came from 19 countries, including Japan.
Reasons support this conference, but the scientific agenda is built by a panel of leading experts co-chaired. This year by Professor Jesus orente from Spain and Professor Sada faruki from the UK.
The scientific agenda is built on primary lectures, peer-to-peer scientific exchange and sharing of best practices.
The scope of the agenda has grown over the years too. Initially, it focused on genetic pathway diseases and BBs. And now, it includes ho.
there were also 43 poster presentations, and 3 impactful workshops
Participants attended 2 of these 3 workshops which covered Optimal Care for rare MC, for a password diseases, multiple linear care and treatment perspectives for patients. You more than 6 and co-morbidities and Communications for patients with acquired ho,
End of the poster presentations. The committee. Selected 3 winners.
The results from a European retrospective study and monogenic obesity so that I can hyper your questionaire as a screening tool for monogenic obesity and an assay for variance in the asip gene.
As the only medical and scientific conference focused on mc4 password diseases, improved as turned into an important opportunity for so many experts to get together, nearly 4% of attendees were practicing and looking at lists and more than 25% were pediatricians and the feedback was overwhelmingly positive.
important, themes emerge, as discussions, point this year, the uniqueness of mc4 pathway diseases
the early onset of obesity in these patients at a young age as a key for diagnosis and of course, High PreCure
These face-to-face discussions are helping these physicians to change their clinical practice when it comes to how they identify, diagnose, and treat these patients.
With improved and many additional efforts, reason is playing an important part in growing the International Community of MC4R pathway disease. Experts.
And now I will turn the call over to enter.
Thank you, John.
Today's business update is positive based on a strong quarter for global commercial Revenue, successful data readouts, as well as an upsized and oversubscribed Equity offering in July.
Let's start with the balance sheet on slide 23. We ended the second quarter of 2025 with 291 million in cash on hand. And last month we completed the equity offering in which we sold approximately 2.4 million, shares of common stock at 85% per share for resulting in net proceeds to rhythm of 1899.2 million.
We are grateful to have received so much support from many existing but also several new long-only and Healthcare dedicated investors in this transaction and on an ongoing basis.
We note here that we paid 40 million dollars. To LG chem in July, the 2nd of 2 tons is associated with the licensing agreement. For biva malagon. That was announced in January of 2024.
This cash payment in July is not reflected in our cash on hand at June 30th. The remaining obligations to LG are post approval, milestones and royalties the fixed consideration component of the agreement is fully satisfied.
Rhythms cash on hand combined with the net proceeds from last month's, stock offering forecasted revenues from the anticipated launch of obsidian acquired ho as well as ongoing revenue from approved indications.
And and currently planned R&D, and Commercial activity provides cash. Runway of at least 24 months.
This level of liquidity indicates that rhythms balance sheet is the strongest strongest in its history.
On slide 24.
Global revenue for the second quarter was 48.5 Million, an increase of 29% quarter over quarter.
66% of Q2 revenue, or $32 million, was generated in the United States, and 34% or $16.5 million was generated outside the United States.
Quarter over quarter, the global number of patients on therapy. Reimbursed patients on therapy increased by approximately 12%.
Us Revenue, increased 7.6 million or 31% over the prior quarter. The number of reimbursed patients on therapy in the US. Continue to grow at Mid single digit percentage rates.
Drop well, dropped below. 10 days.
In the second quarter, the difference between product shipped to our Specialty, Pharmacy, exceeded product dispense to patients by approximately 0.5 million.
A month, a more modest but positive effect on Revenue in the quarter.
The Specialty Pharmacy, carried approximately 10 days of inventory on hand at June 30th.
Excluding these inventory factors sequential us Revenue growth for patient demand and Q2 was approximately 3 million or roughly 10.5% from 28.5 to 31.5 million.
Outside the United States quarter over quarter growth was approximately 3.2 million or 24%.
Appreciation of the Euro and other currencies contributed approximately 1.2 million of this increase.
Geographically, revenue growth was primarily driven by increased sales in France, the UK, and Italy, as well as increased name patient sales in various countries. The latter of which is more variable quarter to quarter.
Looking at growth by indication. As Jan mentioned, our approved indications of BBS tomc and lepar provided the larger increase in patient numbers in the quarter.
Yet Early Access programs for patients with hypothalamic obesity in France and Italy drove a higher percentage increase in patient growth.
Reimburse, hypothalamic obesity patients. Now account for a meaningful percentage of total reimburse patients in rhythms international region.
On slide 25.
In comparison to Q2 2024, net product revenues increased $19.4 million, or 67%. U.S. sales were 83.9%, in line with levels we have historically experienced.
Cost of sales, this quarter was 11.4% of that product revenues. We generally expect cost of sales to be between 10 to 12% of net product revenues for the foreseeable future with variation, due to how our inventory balances are changing and the corresponding capitalization of Labor and overhead costs.
R&D expenses were $42.3 million for Q2 compared to $30.2 million in the same quarter last year. Sequentially, R&D expenses increased by $5.3 million, or 14%, over Q1 2025. This increase was primarily due to CMC work related to formulation for Biva Malagon and auto-injector development for RM7 18.
Increased headcount and stock compensation also contributed to this increase in expenses; clinical trial costs were relatively flat quarter over quarter.
SG&A expenses were $45.9 million for Q2 2025, as compared to $36.4 million.
Sequentially sgna expenses increased by 6.9 million, or approximately 18% compared to q1 2025.
Increased spending in sgna from q1 to Q2 is due to increase headcount, and, and marketing costs.
Uh, the headcount costs are inclusive of stock compensation.
For the second quarter of 2025, there were 63.7 million weighted average common shares outstanding.
Cash used in operations was approximately 22 million during the second quarter. Our gaap EPS for the second quarter of 2025 was a net loss for basic and diluted share of 75 cents including 2 cents per share from a crew. Dividends on convertible preferred stock of 1.3 million.
On slide 26, there's a little more detail on operating expenses for the quarter and guidance for the full year.
For the second quarter, operating expenses of $8.2 million include a total of $15.9 million in stock-based compensation. Non-GAAP operating expense guidance for the full year of 2025 remains unchanged. We anticipate approximately $285 million to $315 million in non-GAAP operating expenses, comprised of non-GAAP SG&A expenses of $135 million to $145 million, and non-GAAP R&D.
Expenses of 150 to 170 million with that. I'll turn the call back over to David.
Thanks, Hunter. So, I think as you've heard now, we're pleased—really pleased—to report out a good quarter.
Incredibly excited about the future ahead of us. So, with that, we'll open it up for questions.
Certainly, as a reminder to ask a question, please press *1, 1 on your telephone and wait for your name to be announced.
To withdraw your question. Please press star 1 1, again, in the interest of time, please let yourselves to 1 question please. Stand by while we compile our Q&A roster,
And our first question will be coming from tazeen Ahmad of the Bank of America. Your line is open,
Is, is it an exploratory study or is this um, a high conviction study? Because there is a history of of uh set meant its high being looked at in this indication before. And I think people would just appreciate getting a sense about how you're feeling about um what data would be good data and what the next step would be if it is good data, thanks.
Yeah, thanks Christine. Um, yeah, I I characterize this as exploratory. Um, as you noted, I mean, our original, uh, our initial study done, um, back in 2019, quote, unquote was um, negative and meaning it did not show up positive result, but was we've explained that was a difficult trial design and we thought the dose was too low. The timing was too short and there was good reasoning based on the underlying biology of the greater Willie to believe that the mc4r pathway plays an important role. So, uh, the current trial open label study, um,
The dosing as in our last study had a maximum dose of 2.5. This study goes up to all patients are dose, escalated to 5 milligrams as tolerated and the duration. Um, to that trial, patients, were on for a maximum of sort of 4 to 8 weeks um on a certain dose. Um, this trial will go out 6 months and we'll look at the data at that point. Uh, what would be good? And the reason I characterize it exploratory, I've I've characterized and we'll continue to characterize it as a, you know, 50/50 a very legitimate 50/50. And the reason for that is, I think, um, we have high conviction about the underlying biology and the importance of the mc4 pathway in the disease. But we know the disease is
Challenging and, you know, a lot of a lot of drugs have failed. And, and there's a behavioral component to this disease, which can often create noise or obscure a potential beneficial effect. So those are, you know, things that give me pause. And, and this is why I would characterize this as exploratory
How many patients' worth of data?
yeah, so we
Um so the the trials that we can roll up to 30, it's an open label trial. We won't enroll.
Up to a full 30 patients. Um, our goal is to get north of 10 patients, so 10 to 20 patients, and, you know, hopefully have a meaningful ability to say something meaningful by the end of the year. Again, the, and as he's like this, um, you know, you don't start talking after 2 or 3 patients. There's just too much noise in the system, and you can't be confident in what you're seeing. So our goal is to say something by the end of the quarter, which hopefully will be based on data we can have confidence in, and so, by the end of the year, apologies.
okay, thank
And 1 moment for our next question.
Our next question, will be coming from Mike of Morgan Stanley. Mike, your line is open. Yep. Uh, good morning. Thanks for taking the question and congratulations on. All the progress, maybe just a quick follow-up on the prayer. Willie question, uh, appreciate the color on the number of patients. But can you give us a sense on, you know, what sort of level of follow-up You're Expecting?
Thanks.
Uh, so follow up again with all these many diseases. But certainly in rare diseases, if patients are benefiting, you keep them on treatment. You don't tend to run even an early-stage study and just stop at the end of it. That's challenging for these patients, and it doesn't make sense. Some of the most valuable data you gain is in the long term.
Follow-up of these patients and your your ultimate submission is a totality of the evidence approach. So you you may have your phase 3 but it's strongly supported. Perhaps by, you know, 1 year to 2 year data, uh, out of your early early, treated patients. So, um, these patients, you know, 6 months is the point at which we will look at the data and and begin to, uh, try to determine what we have, but those patients will continue on Beyond 6 months and they'll continue on indefinitely. As long as we believe that there is an effect and we are proceeding with the overall clinical development for prayer of will.
Got it. That's helpful. Maybe I could just ask a quick follow-up. You know? Assuming
You know, if the data is positive, you know, how do you think about some of your next Generation mc4s? Like biva megalon, is that something you consider taking forward in this indication as well? Thanks,
Yeah, um, again, it's been, you know, I think, uh, historically, we've said that, you know, most, if not all of our subsequent development work would be done with our next Generations. It just makes sense for multiple reasons, potentially better drugs, long longer patent license, Etc. However, um, if the data is, um, you know, compelling and we're convinced, um, the possibility of going immediately was set manitite is absolutely on the table. And so we'll see how we do with a timing getting 718. Uh, you know, up to this initial proof of concept period, And how that matches up with the proof of concept data, we get on prayer, Willie, uh, and our current trial, and then we'll make final decisions. But we'll certainly be. If we're going forward, we will for certain be doing it with our next gen. I think the 1 or both of our next Generation molecules, the question is, do we go rapidly with 7 at a time?
Thanks very much.
And our next question will be coming from fail Nadu. A TD Cohen. Your line is open filled.
Good morning. Congrats on the productive quarter, uh, a follow-up from us on Prairie, too. Just circling back on what is is good data? Uh, it seems like there's a few elements to the data. We'll be looking at BMI decreases reductions in hunger, as well as the consistency across the the patient population. David could you give us some sense, uh, as to how you, what you want to see to to move forward? What would be good data in terms of weight loss effects on hunger and and consistency?
Yeah, yeah, thanks. So I decided I didn't mention that or answer that earlier. Um, so I, I think the primary end point here. I mean, aside from safety and tolerability is a a weight. Um, as we all know, um, slo's drug was approved on a hyper fasia endpoint and that, you know, it was a huge breakthrough for the community because it was the first drug approved and it did in a sense to find a pathway for hyper fasia as an endpoint to be approved. But and we know our drug, um, by definition, the way the biology works is we provide a society signal so we decrease the hyperphysics expenditures. So if we get weight loss, um, BMI decrease some almost by definition, we should have an improvement in hyper fasia the magnitude we're looking for here is different. Um,
And our other, um, mc4r pathway diseases. And that's I think because of the overlay of all the other challenges,
but nothing gives you weight loss in this disease. So anything 5% or greater, um, is approval based on the FDA guidelines, um, for obesity drugs.
So, that would be, uh, that would be our Target and that's at a year. So our goal would be to have confidence that we were seeing a change in BMI that either was at or, you know, moving consistently and steadily toward at minimum a 5% decrease in BMI, um, the 1 cavitt, which was a 10 point that SLO got approved on, uh, it's an uncontrolled study and so those kind of um, patient reported outcomes are a little more challenging to interpret perhaps in that setting, but we will have that data as well.
Great. And um, 1 quick housekeeping question, for for Hunter, if I might, uh, in terms of Opex, your guidance for the non- go. Non-gaap, uh, upbringing expenses is very clear, but in terms of stop comp, uh there's 15.9 million in Q2. I think you have like 30 million in stock comp for all of.
2024. So how should we think about stock comp uh, going forward in the in the second half of 2025?
I think it's a, I think it's a fair question. Phil obviously, we've seen a significant increase in stock comp, for the due to the change in the price of the equity of Rhythm. And, um, so um, I don't think we're in a position to give full year guidance. But obviously, you know, an increase of, you know, essentially,
3 million quarter over quarter is um you know significant then beyond our Direct Control because it's just driven by the stock price. So
Got it. So, so this is a good Baseline to to use as we think of. It's, it's a, it's a fair Baseline as we move forward. Yeah, perfect. Thank you.
And our next question will come from David Arcola of Wells, Fargo, your line is open there.
Hey, good morning. Uh, Derek on uh, just uh had 1 question for David here and then 1 for Hunter. So, David just will you be providing updated estimates for ho prevalence during the commercial day in September. And I guess what gets you confident that? They're at the higher end of the range as he said in the prepared remarks and then just a quick 1 for Hunter, you know, just in terms of the growth that you've been reporting X us for the past 2 quarters, you know, how should we be thinking about that? Moving forward. Thanks.
Um, so Derek. I'm gonna plead, uh, needing a little more time. We haven't defined the exact agenda, our goal would to give you as best sense. We can about our current understanding of ho. Obviously, a lot of Works being done. Jennifer's teams doing a lot of work. Uh, now in the field, I think on the Epic side of it, as we've said, we've we've moved from sort of our initial estimates of 5 to 10 to quote, unquote, being more confident that we're uh we're at the higher end of that 5 to 10,000 and it's it's comprised of a number of things. I mean you start out with as you do in bra diseases you got whatever is out there in the literature. Um we've done claims data work now in the US and um
This feels about right? And so, um, so I'm not sure it's a long way of saying I'm not sure, we're going to update our assessment, um, at that point we'll give you, you know, we can reconfirm where we are. But, um,
Uh, but we are learning a lot and we'll try to give you a sense at that day, where we are. Um, in terms of what the field teams are learning, probably, that's the biggest piece, which will be new.
And Derek with respect to, uh, with respect to revenue growth. I think, you know, we, we did highlight the currency effect during the quarter, which was responsible for about 36% of the growth. So 1.2 million of 3.2. Um, so that's obviously something that, uh, we can't we can't predict and I certainly wouldn't model. Um, and that, but separate from that, um, I would say we we have had a strong run in the past, 2 quarters in international, um,
Q3 in general, can be a little quieter, uh, in terms of new patients starts in Europe, um, just the vacation effect that people have and that has an effect on growth and not named patient. Sales are also less predictable, some countries take a shipment for a few months at a clip and there was certainly some effect of that in Q2. Um, so you know, it's not as clear when those types of countries come back in for another set of shipments. But, um, overall we're we're pleased with, uh, growth and International.
um,
and uh, we expect to continue.
Great, thank you very much, guys.
Thanks sir.
And as a reminder, in the interest of time, please limit yourselves to 1 question.
Our next question will be coming from Karen Johnson of Goldman Sachs, your line is open.
Good morning everyone. Um, maybe on the other clinical update expected later this year, you don't have that first station enrolled in part C. Could you provide any Clarity on the major of the data? You could possibly share later this year. Recognizing, um, that enrollment is going to continue into next year and then on the um, age show use, I think you said that there are sign meaningful xus utilization. But do you have any visibility on whether there are ho patients getting into every awful label here in the US?
Thanks.
Yeah. Um, so on, on the part C uh piece of this what we've said and you know, we've moved our, my goal originally is, you know, or many of, you know, um, was to say something about 718 by the end of the year, it's taken us longer, uh, to get up and running, um, and we are up and running now, but um, that's delayed us a bit. So we moved the, you know, completing enrollment, um, to quote unquote, first quarter. So that means that it's extremely unlikely, that we will have anything
Say about it is an open label study but that we will say anything about 718 by the end of the year it's more likely that we'll be into 2026.
And regarding the um ho off label usage within the US. I would say that. Right now we do have a couple um it's like a handful very minimal just in terms of what we have received from a RX perspective uh to date in that indication
It's fair to say in rare diseases, in the US off, label use is. Um, people are the good news is, they're very Allegiance, payers very allegiance to the, uh, to the label. But on the flip side, there isn't a kind of off labels use. You might see in some other diseases.
Thank you.
And our next. Next question, will be coming from Paul, Ms of people. Your line is open, Paul.
Hey, good morning, thanks for taking my question. Um, just 1 question, 718 you guys did a good job at the business knowledge on study and I'm preparing us for the caveats to comparison and some of the demographic differences between trials that will sort of inform, how you can stack up these drugs for for the 7118 study. Um, I know you're getting started with BHL portion now, but what are you expecting for the patient next. Um, and what are some of the things you should keep in mind as you sort of gauge whether or not this is matching?
See if your other drugs. Thank you.
that's the part that hopefully, this trial will sort this sort out and give us a good feeling for
Do you think you've maxed out the efficacy of this mechanism at this point or can greater exposure actually drive more benefits?
I I do think we've maxed it out. I mean we've now we've done enough, we've treated enough different populations, I just you know, I'm not convinced there's occasional patients who may need a higher dose and we don't dose based on weight and obviously there's a very big difference from a, you know, a 50 kilogram, pediatric patient, 200 kg adult patient. And so, you know, those are the kind of differences where dose May on the margin making issue. But I think your basic question is have we maxed out? Yeah I I think we've likely maxed out so this is
Fair enough. Thank you.
And our next question.
Will be coming from a seam. Miss Fernandez of Guggenheim, your line is.
No, thanks for the question. So um you know David I think in the past we've talked about the opportunity for rhythm to become, you know, quite a bit more important uh in the overall team of the you know sort of specialty Market. Can you just help us understand a little bit better? Uh, the opportunity that you see you've talked about BBS is a 15 year opportunity for growth. Um, aho, you know, in the mix, how do you think about the opportunity? There you're talking about 10,000 patients but it seems like over time.
You expand the market opportunity. We could see numbers north of that over time and obviously, the company potentially becoming more important from a strategic perspective. So, just wanted to get a better sense of how you're thinking about the overall launch characteristics. Uh, in aho and the markets that you're going to most urgently urgently reach into, um, but the opportunities that you see beyond just the, you know, sort of standard uh Japan uh, Europe and uh us opportunity. Thanks so much.
Yeah, thanks. Yeah. That's that is a bit of a theme of uh today's call in the sense of you know, how does how does Rhythm grow? So you started where I would start is um BBS and we have you know by now a lot of confidence in the BBS numbers. Uh
It will grow over time. And I think the biggest variable for me is is not so much. Will we get to some projected Peak kind of Revenue and maybe, you know, these kind of rare disease opportunities often don't peek. Um, but they just tend to grow which is why I picked 15 years out of the year. Of course. Um, I don't have any insight that's going to be 15. But what I do know about rare diseases and this is from my past. History is they do go for decades and they do tend to continue to grow for decades and they grow both inside the markets where you started. But then you also continue to add markets and, you know, we've been very focused from the beginning of being Global. Um, and we realized that, you know, it was going to be hard and you start slow and Beyond
Highlighted this morning we have you know a new patient or patient you know there are a handful of patients in the Czech Republic and in Poland that's how it works. And you get, you know, you start with 1 or 2. And those first patients are incredibly important because they signal a willingness of the system to start paying and to work with you and the like. And so and it just builds over time. So that's BBS. Acquired. Ho bigger, epidemiology. I mean, we had questions this morning and we'll get continue to get a lot of questions about how big could this be? I think where we are now. A lot of confidence in our current projections. Could it be bigger? Absolutely. Um, will it have some of the same Dynamics as these kind of ultra rare diseases and AO sits a little bit in the middle? It's absolutely a rare disease quite rare 5 to 10 thousand. Puts it in the very rare category but it's very specialty like given the concentration more patients diagnosed you know, an attentive specialty up by definition. So um aho may have a slightly different
Ramp, if you will, uh, steeper than BBS because of those factors. But the other aspects of AHR are going to be very similar, which is think about steady growth over time. Don't think about inflecting, you know, this is something that will explode out of the gates. By some of these quote-unquote special opportunities do, that's going to be more rare. But it will be steeper and it will grow for a very long time. And we may be wrong in the epidemiology, meaning that it could be larger, and given enough time, I think that's likely. And then finally,
Know back to hoe again, opening up new countries. I mean we're in Japan, but we're still early in terms of assessing Asia and and you know, other markets like that. So we will get there. Um, but that's how you you grow an opportunity like this.
Thank you and 1 moment for our next question, which will be coming from Dennis? Ding of Jeff? Dennis, your line is open.
Thanks for taking my question. I had 1 on Trader Willie. Um, so just given the availability of vat and the fact that you're faced to, um, is being done at a single Center. Uh, what sort of guidance are you giving, Dr. Miller in terms of voodoo enroll in the study versus maybe who she uses vicat, uh, specifically like what types of patients would go onto the study. Um, and you think that would make it more difficult potentially for set Millennia type to show, uh, and efficacy signal there. Thanks. Yeah. And Dennis, it's a really good question. Um, so, uh, the guidance is this inclusion criteria and inclusion criteria is, it's, um, prayer to Willie patients 6 and above. Um, there's no exclusion for the use of vaccard. So if patients are stable on that drug, they're allowed in that trial and we will have some of those patients. Um, 1 is we were interested in what that combination would look like and 2. Um, it's standard of care now and, you know, that's the world we be moving into to develop this drug.
So, so that's not an interesting. I think, you know,
Uh, so that's the guidance I think who she's she's enrolling, there's a group of patients um who uh, so diabetics for example, uh, it's more challenging to use white card in that population. I mean, it inhibits insulin release and so it can make your diabetes worse and so um, we I already know we have some patients with diabetes in this um, you know, open label study and and that's um yeah. Your your point is could those be more challenging patients and we know
My definition yet. Diabetics can be more challenging particularly in a weight loss study, and they have other stuff going on, which makes them difficult to manage. So, yeah, that's it. It's going to be much more of a mix and, you know, we'll have to analyze it with that context.
Okay, got it understood. Thanks.
Thank you. 1 moment. Next question.
Next question will be coming from ragi Ram. Salvo of HC, rain writing company. Your line is open.
Thanks very much for taking my question, this pertains to CMC. I was just wondering if you could comment on the status of the development of the smaller pill for bevel and also the key objectives in your auto injector development work for RM 718 including but not limited to the possibility of developing a formulation that might be doable. Uh, less frequently than once weekly. Thank you.
Yeah. Um, so in terms of the smaller pill, I mean that's not a big challenge right now in the sense that the bigger challenge which is the CMC group has, I think, surmounted was getting the formulation changed. So, our current...
2000 milligram pill we can now get 600 milligrams in a single pill. So basically, a 90% drug load in that single pill going down to 400 and 200 milligram pills with 90% drug low. Just means you're going to have a smaller pill and that that technically in a sense that's done.
Um, the auto injector goal. Uh, it's for a weekly formulation. We do not have any plans at this point, um, everything's possible and that's a natural path to continue to try to extend your, your frequency of injection. But for the moment, um, this is all completely aimed at our weekly, uh, program,
Thank you.
You.
And our next question will be coming from fizzy khurshida.
Your line is open.
Hi. This is Heidi. Jacobson on Professor kashid. Thanks for taking our question. Can you share any updated? Thoughts on the phase 3 study. Design for Viper. Malagon in acquired ho, including dose study size and what is left to get done. Before that study can get rolling, I think
Yeah.
Um, so what's left to get done is, um, we we need to submit, uh, or submit a, a meeting request to the, uh, FDA and EMA. And then we, we submit a briefing package with a synopsis or proposed trial design that they react to, we may or may not get a meeting or a call. We'll see what happens with that. But
But that's step one in terms of the regulatory process. I think in terms of design.
Standard in terms of their responses to this kind of thing. So we may well end up with a study that looks more like marker and Howe trial.
Those, those are the things we're thinking about.
Got it. Thank you.
And I would now like to turn the conference back to David meaker for closing remarks.
Okay well thanks everyone again for turning in. Um I said we're we're really pleased. Um, where we are, we're making good progress.
I had a lot to do, so we look forward to our next update. Thank you.
And this concludes today's conference call, thank you for participating. You may now disconnect