Q1 2025 Rhythm Pharmaceuticals Inc Earnings Call
Speaker Change: Good day and thank you for standing by. Welcome to the Rhythm Pharmaceuticals First Quarter 2025 earnings conference call. At this time, all participants are in a listen-only mode.
Speaker Change: Please be advised that today's conference is being recorded. After the speaker's presentation, there will be a question and answer session. To ask a question, please press star 1-1 on your telephone and wait for your name to be announced.
Speaker Change: To withdraw your question, please press star one one again. I would not like to hand a conference over to your speaker today. David Connolly, Investor Relations at Rhythm Pharmaceuticals Inc.
Speaker Change: Thank you, Josh. 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 Investor section of our website, ir.rhythmtx.com
Speaker Change: This morning, we issued our press release that provides our Q1 2025 financial results and a business update, and that press release is available on our website.
Speaker Change: Our agenda is listed on slide 2. I'm a call today our David Meeker, our chairman Chief Executive Officer of President
Speaker Change: Jennifer Lee, Executive Vice President, Head of North America, Hunter Smith, Chief Financial Officer, and Yann Mazabraud, Executive Vice President, Head of International, is on the line, and joining us from Europe .
Speaker Change: On slide three, I'll remind you that this call contains remarks concerning future expectations, plans and prospects
which constitute forward-looking statements.
Speaker Change: Actual results made different 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 file with the SEC.
Speaker Change: 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 date.
Speaker Change: Re-specifically displaying any obligation to update such statements. With that, I'll turn my call over to David Meeker, we'll begin on Slide 5.
David Meeker: Thank you, Dave. So good morning and thank you for joining
David Meeker: So, we're about one month since our call highlighting the top line results on day three trial and acquired hypophilamical obesity [inaudible]
David Meeker: We remain on track for Q3 filing, including having an in-person type-d meeting scheduled with the FDA. I will say the FDA is responsive and I would characterize our interactions as completely normal.
David Meeker: We have reviewed all of the data, which we will present at upcoming meetings. The more we dig into the data, the more convinced we and I are that set melanotype has the potential to transform the life of both the patient and their families. I will remind you of the top line data with a little additional color in the next few slides.
David Meeker: Commercially, with BVS, we had a very good first quarter, and the U.S. demand, as reflected in vials, dispensed to patients, continues to grow. The team, as Jennifer will describe, is making good progress on multiple fronts addressing the challenges of this complex disease.
David Meeker: The growth and demand is obscured this quarter by the inventory shifts which Hunter will walk you through
Speaker Change: The international team continues to execute on its country-by-country BBS launch strategy and the H.O. contribution to revenues predominantly from France and Italy continues to grow supporting our view of the unmet need and the level of interest in satellite attitude. Thank you very much.
Thank you.
Speaker Change: As we have highlighted, we're looking forward to the Bibamelagon Phase 2 readout in Q3 and having something to say about the ongoing DWS study, Brewer Willie, and the 718 weekly study in H.O. by the end of the year. And we remain well capitalized with a projected cash runway in 2027.
Speaker Change: On slide six, this is data from a publication by Professor Mueller from Germany, a renowned expert in the field of hypothalamic obesity. It provides some numbers behind what we are learning is we get to know this community and review the data from this trial in the field.
Speaker Change: The medical complexity and severity that these patients and their families are dealing with is unlike any disease I have worked on in my industry career [inaudible]
Speaker Change: There are diseases with higher mortality rates but very few with higher medical complexity.
Speaker Change: and you can see that from the numbers on the slide. 3.7 hospitalizations in the first two years after the injury, of which 23% required an ICU admission, 12 visits to their general practitioner and an average 20 visits to them.
Speaker Change: Specialist, and even more strikingly, the average number of prescriptions written per month is 5.5. The average number of unique medications prescribed in the first two years is 22 and 89% of these patients require three or more therapies for a neuroendocrine dysfunction. The average number of patients require three or more prescriptions for a neuroendocrine dysfunction. The average number of patients require three or more prescriptions for a neuroendocrine dysfunction.
Speaker Change: As the data from this trial shows us, including the exit interviews with patients and caregivers, the untreated hypothetical obesity with its associated hyperphasia and fatigue represent a significant part of the medical burden they are dealing with.
Speaker Change: The fact that patients were willing to commit to this 52-week placebo controlled trial, given the incremental burden of testing and clinic visits, which are part of any trial, speaks to the motivation of this community to find solutions. We have rhythm are highly motivated.
Thank you.
Speaker Change: On slide 7, now I'll provide you a little additional color on the results of the Phase 3 study. First, the pop line, and we're showing again, with a 16.5% reduction in BMI in the 7-malanetide cohort as opposed to a 3.3% increase in BMI in the placebo group, for a placebo-adjusted difference over 19.
0.8 percent.
Speaker Change: As we showed you last time, moving to slide 8, there was no difference in effect between patients under 18 and those over 18 [inaudible]
Speaker Change: So we have broken this out further, so on slide nine. [inaudible]
Speaker Change: We did stratify patients between three age groups, breaking out the under 18 to those between 12 and 18 and those less than 12 [inaudible]
Speaker Change: Adults may be a relatively homogeneous population, but there's a big difference [inaudible]
Speaker Change: and a four-year-old and a 17-year-old. Here, you can see the three age cohorts and remarkably, they are similar again with placebo-adjusted BMI percent changes ranging from 19.2 percent to 21 percent.
And slide 10.
Speaker Change: A hallmark of the trials of semiland tide in this disease has been the consistency of response, and as we showed you last call, 80% of the patients lost more than 5% suggesting some response.
Speaker Change: As always, the patients are greatest interests, at least to me, are the apparent non-responders, and I gave three patient examples. I asked all the patients who did not reach the 5% but had other data suggesting a response to drug. A more complete summary of that analysis is as follows.
Speaker Change: There were 17 out of 81 set melanotide treated patients who were not considered responders by the person reaching 5% or more in this analysis.
Speaker Change: Eight of these patients discontinued treatment prematurely and have their data imputed.
Speaker Change: Three of these eight patients actually had reductions in BMI percent, BMI greater than equal to 5 percent at their last time point assessed but were counted as non-responders due to the conservative nature of the multiple imputation method which uses the placebo patient data to generate the imputation values.
Speaker Change: Of the nine non-responders who did complete the trial, six of the nine patients either had a response greater than 5% at some point during the trial and or for the pediatric patients had a BMIZ score decrease greater than equal to 0.2, which is clinically significant.
Thank you.
Two of the three patients with no response.
Speaker Change: and no other explanation had drug blood levels consistent with significant non-compliant.
Speaker Change: So in summary, the consistency we saw in the phase 2 trial, the consistency response we're seeing in the real world French experience is evident here. [inaudible]
Speaker Change: This further analysis sports the thesis that the biology in this disease is driven by impaired signaling through the M.C.4-R pathway with a consequent deficit in alpha-millanocyte stimulating
Speaker Change: The Consistency of the Response to Semi-Lanatine, we see across this highly heterogeneous and medically complex patient population suggests we are helping to correct a fundamental biologic deficit.
David Meeker, David Meeker, David Connolly,
Speaker Change: Let's find 11. Remind you of the disposition in this trial, the vast majority of the 143 patients enrolled consisting of 120 in the pivotal cohort, 11 supplemental and 12 in the Japanese cohort have moved to the open label extension trial. [inaudible]
Speaker Change: A total of 120 patients remain on treatment, 108 patients who transitioned to the open label extension, and the 12 Japanese patients remain in the blinded portion of the study
Speaker Change: And importantly, there were no new safety signals related to septum line of tide observed, which of course is in line with septum line of tide well established and well understood safety profile. Consistent with prior experience, septum line of tide is also generally well tolerated in the study.
Speaker Change: and on slide 12, which you've seen before, to remind you of the very significant opportunity in the choir H.O. The unmet need is there, these patients are accessible, they're diagnosed, and
and this set-in-line tie data looks extremely promising. [inaudible]
Speaker Change: We believe we can make a significant difference in this disease.
So with that, I'll turn the call over to Jennifer [inaudible]
Thank you, David.
Speaker Change: Beginning on slide 14, we are pleased to report that the demand from Sivri as the only treatment that addresses the root cause of hyperphasia and obesity associated with bottle beetle syndrome remained strong in the first quarter of 2025.
Speaker Change: Overall, we have seen continued increasing demand for M. Zivri with a consistent number of new prescriptions received over the past several quarters, resulting in ongoing growth in patients on reimbursed therapy.
Speaker Change: During the first quarter, we did see an increase in the number of patients that transitioned to new insurance plans, which happens at the beginning of every year.
Speaker Change: This resulted in an increase in the number of patients that temporarily transitioned from receiving commercial drug to receiving free drug from our bridge program.
Speaker Change: I am pleased to report that we secured access for the vast majority of these patients and exited to one with the overall percentage of bridge patients back to normal levels experience at the end of 2024.
Speaker Change: On the next slide, I will update you on two additional positive trends relating to improved Medicaid coverage and improvements in the breadth and depth of prescribers.
[inaudible]
Speaker Change: We continue to see increased breaths and depths of prescribers. In Q1 we saw the highest number of total prescribers within the quarter. [inaudible]
Speaker Change: since the launch of them severed and saw a 13% growth in the total number of prescribers this quarter compared to Q4 of 2024.
Speaker Change: This growth was driven by an increase in new prescribers which is an important driver of the business since launch to date approximately a third of new prescribers become repeat prescribers.
Speaker Change: This demonstrates that when a physician is educated to look for the clinical manifestations of BBS and their patients, they begin to find more BBS patients [inaudible]
Second positive trend is Medicaid access on the next slide.
Thank you.
Speaker Change: The last time we shared this metric was a year ago, when the breakdown between states with positive coverage from civil rights versus no coverage was approximately 85 to 15.
Speaker Change: We'll continue to work persistently to expand access, and in recent months we have secured in civil-specific policies in three additional states.
Speaker Change: We now have in Sivri-specific policies or positive coverage decisions firm Sivri in states that account for greater than 95% of covered Medicaid lives, leaving less than 5% in states with no positive coverage firm Sivri today.
Speaker Change: This success and securing access firms every from Medicaid, as well as commercial payers, will serve as a strong foundation for our next potential launch in acquired hypothalamic obesity.
Speaker Change: We are excited by the potential to bring in Sivri to HO patients. Since our market research, both with physicians and patients and caregivers, points to a significant need for a therapy for this devastating disease.
Speaker Change: We know endocrylonologists remain critical for patients with Hexlamic obesity, enabling us to focus our efforts on this one specialty, which is the same specialty that accounts for about half of prescribers for BBS.
Speaker Change: Waking, lack of energy, and hyperphasia were outlined by endos to have a significant negative impact on patients' day-to-day lives, with current treatment options insufficient in addressing these symptoms for patients.
Speaker Change: As expressed by one end, we need a treatment that is going to be more effective, well-tolerated, and safe to continue long-term because this problem is not going to go away [inaudible]
Speaker Change: These patients are going to be on this treatment for their lifetime. Once you withdraw the treatment, they will regain the weight and more.
Speaker Change: When shared a blinded target product profile, all physicians outlined they would prescribe
Speaker Change: All my next and my last slides are research with patients and caregivers illustrates just how significant the impact of hunger and hyperphasia, decreased energy or fatigue, and weight gain are for patients [inaudible]
Speaker Change: As seen in the first quote, I was just feeling like a different person after the tumor and the hunger rules your life.
Yann Mazabraud: These patients know what life was before the onset of these symptoms associated with H.O. and they want their lives back. With that, let me hand it over to Yann.
and David Connolly. Thank you.
Yann Mazabraud: Thank you, Jennifer. We've been very available to remember taxes, name, patient sales, or Yann Mazabraud programs now in more than 15 countries outside the United States.
Yann Mazabraud: We are seeing steady global growth in the number of patients.
Yann Mazabraud: on pace therapy at the end of the World Cup. Patience and treatment, essentially by country.
Sullivan, C.S. Cotton, Jeff Lynch, Yann Mazabraud, J.B.
Yann Mazabraud: Patient Population, a better identify in Europe than anywhere. Remain an important contributor in the international.
Yann Mazabraud: BBS Aweller, the main driver of the new, our type 9 page. We say it grows in BBS and Rainbow Strapi.
and Germany and France.
Yann Mazabraud: Also, more patients with hypotalanic obesity are being accessed to incinerary through early access programs and can also name themselves instinctively.
Yann Mazabraud: Of course, we are seeing it of course with the pay the DNA approval for our friends [inaudible]
Bosch, The Quieter, and Conjunitor, Academic Ability
The early access programming directs growths.
Yann Mazabraud: We are now seeing a bit of Spain, and even where our first
I've started, sir.
Next slide.
David Connolly, David Connolly, David Connolly,
Yann Mazabraud: We are looking forward to the future of European medical care.
at which we have ten procedures including five four roles.
Yann Mazabraud: the first ever joint conference of the European Society of Technology.
and the European Technology Institute in Copenhagen.
The 30-second press conference will take place in Malagasy.
Yann Mazabraud: We are particularly grateful for what is present, because we will ward data from the French
Yann Mazabraud: Recall, in the fall of 2024, Francis presented real world data demonstrating the potential efficacy of settler side in eight patients with acquired ecotalanic obesity. What's even?
Yann Mazabraud: The 12.1,000 BMI-regions are pre-borns and factations were greater than 21 BMI-regions are pre-borns on settler data.
Yann Mazabraud: Next week, we will see that that's a quick topic with a choir of content, so that's a good idea. We'll reach to it for more on-site.
Yann Mazabraud: as well as details on how our success and support programs are considered.
Thank you. Bye.
and Kathy Vilsack.
Surgenity, career, it's awesome. If you need in the daybreak file, Rhythmic.
as representative patients.
H-I-P-E-V-E-R
Altogether there, we have ten presentations.
Yann Mazabraud: at these two conferences, plus two symposia focused on career and support pathways.
Yann Mazabraud: This level of activity has two different things. Okay, this is the level of support and the very strong interest we've got from European experts.
No, I would put it up.
David Meeker: Yeah, so you were breaking up a little bit, yes. I think we lost some of your communication there. I'm just going to read briefly from your script just so everybody has the last part. I think
Speaker Change: I'll summarize briefly. Things are going incredibly well in Europe . As Yann highlighted, hopefully people got that in the BBS launch of progressing across countries. The end of FB meeting which is coming up and this is what he's speaking to at the end is we're excited about that and we have multiple. We'll talk about that.
Speaker Change: Abstracts that are being presented there and just to summarize what he said but make sure everybody heard it. So we previously presented data on the data from the initial eight patients in the French real-world experience and next week we'll see data from 24 patients with acquired or congenital obesity who have reached at least three months or more in several antitide.
Speaker Change: And then there also be a presentation of real world evidence of incivory for German BDS patients which points will point to the benefit of reducing hyperfasional obesity as well as details on how this specialized nursing support program tributes to patient and caregiver satisfaction.
Speaker Change: and then there'll be a publication or an abstract on the genetic rare MC4 pathway indications studied in daybreak come.
Speaker Change: including representing findings on patients with this PHIP variant, which is one of the variants which have particularly strong results in our face.
Speaker Change: Our initial phase two day break study and we look forward to doing more work there so that that will be there. And I think I'll just comment that on the H.O. data, the 24 patients you're going to see, we're in barcode now, so we can't present that data but you won't be surprised. There's nothing about that data set that's in consistent with what we've seen so far. [inaudible]
Speaker Change: So with that, I'll turn it over to Hunter. Thank you, David. Thank you, Yann. Rhythm had a very good first quarter. Let me walk you through the components on slide 23
Hunter Smith: Dravenood from global sales of them, Sivri was 37.7 million in the first quarter of 25. The number of patients on reimbursed therapy increased 14% globally during the quarter.
Speaker Change: The biggest, the biggest driver of the Q4 to Q1 change in revenue was an inventory swing of 8.3 million at the specialty pharmacy that dispenses him every to patients in the US.
Speaker Change: As we discussed in our Q4 call, the specialty pharmacy had purchased approximately 4.1 million more than it dispensed the patients in that quarter. This resulted in an increase in the specialty pharmacy's inventory to more than 22 days on hand, effectively pulling forward orders from Q1 into Q4.
Speaker Change: In the first quarter of 2025, this ordering and shipment pattern more than reversed itself, the specialty pharmacy ordered 4.2 million less than it dispensed to patients, reducing inventory days on hand to less than nine, typically the specialty pharmacy inventory runs between 10 and 15 days on hand. [inaudible]
Speaker Change: The value of them, Severi, dispense to patients in the US, the best measure of demand for them, Severi, increased 1.1 million sequentially over Q4 Major drivers of this included a 4% price increase taken in January and increased the number of reimburse BBS patients on therapy
Speaker Change: and increasing the number of patients on bridge therapy at the start of the quarter as Jennifer described earlier that resolved itself by the end of the quarter and a modest increase in patient compliance.
Speaker Change: Outside the United States, sequential revenue growth is strong at 3.1 million, primarily driven by France, Germany and Italy
Speaker Change: Taken together, the 8.3 million inventory swing, the increase of 1.1 million in product dispense to patients in the US, and the 3.1 million increase XUS [inaudible]
Speaker Change: resulted in a net revenue decrease of 4.1 million compared to the fourth quarter of 2024, reflecting timing difference from inventory shipment patterns in what was an otherwise strong period of growth.
Speaker Change: Looking ahead, we don't expect such significant swings in a civilian inventory of the specialty pharmacy as we experienced in the last two quarters. But if we do experience it, we will continue to highlight any significant impacts as we have done in the past.
Speaker Change: Now move to Slide 24 with our financial snapshot from Q1 2025.
Speaker Change: In comparison to Q1 2024, net product revenues increased $11.7 million, or 45% over the first quarter of 2024
Speaker Change: for some additional color on the P&L, grossed to that, for US sales was 84.2%, generally in line with 85% GTN, we've reported previous Fortress.
Speaker Change: R&D expenses were $37 million for Q1 2025, compared to $128.7 million during the first quarter of last year. When we incurred R&D costs of $92.4 million associated with the acquisition of Bivomelagon from LG Count.
Speaker Change: On a sequential basis, R&D expenses are down by approximately 10% from the 41.2 million we reported in the fourth quarter of 24 due to decreased spending on the PD average phase three trial, they break and emanate trials and decreased costs associated with the RM718 phase one trial.
Speaker Change: Also, we are seeing lower costs related to post-rial access due to a more efficient design for our open label extensions [inaudible]
Speaker Change: SGNA expenses were 39.1 million for the first quarter of 2025 as compared to 34.4 million for the first quarter of 2024. Sequentially SGNA expenses increased modestly by less than 3% compared to Q4.
Speaker Change: For the first quarter of 25, the weighted average common shares outstanding with 63.1 million, compared to 60.1 million for the first quarter of 24.
Speaker Change: and 61.6 million weighted average common shares outstanding in Q4. The increased quarter over quarter primarily reflects the approximately 1.3 million shares sold under the ATM program in December and January which we announced in conjunction with our Q4 earnings.
Speaker Change: Cash used in operations was approximately 40.4 million compared to 19 million in the fourth quarter. This change represents an expected seasonal increase that occurs in the first quarter each year with the payment of annual bonuses company-wide.
Speaker Change: In addition, the 6.3 million cash consideration paid to re-acquire the rights to him so we in China was recorded this quarter [inaudible]
Speaker Change: Looking at cash, love going forward, the final going forward into Q2. Thank you very much.
Speaker Change: Just to remind everyone, the final 40 million portfolio, final 40 million portion of the license to be payable to LG as part of the the Melagon Transactions.
Speaker Change: will be paid during the coming quarter. It was dispensed in the first quarter of 2024 as previously mentioned. [inaudible]
Speaker Change: On slide 25, we ended the quarter with 314.5 million in cash on hand, which we believe will be sufficient to cover all planned operations into 2027.
Speaker Change: As we touched on earlier, U.S. revenues were affected by an inventory swing from Q4 to Q1, which decreased the percentage, the U.S. percentage of overall product revenue to 65% in Q1 from 74% in Q4 . . . . . . . .
Speaker Change: We noted previously about reacquiring our rights in China for $6.3 million during Q1. This transaction was recorded as a reduction of license revenue with $5 million due to the termination of our licensing agreement with RareStone and a reduction of deferred revenue on our balance sheet of $1.3 million.
Speaker Change: Our Gap EPS for the first quarter of 2025 was a net loss for basic and diluted share of 81 cents. This includes 8 cents per share for the rarestone repayment and 2 cents per share for of accrued dividends on convertible preferred stock of 1.3 billion.
Lassie on side, 26. Our Outbacks guidance remains unchanged. [inaudible]
Speaker Change: We anticipate approximately 285 to 315 million in non-GAAP topics comprised of non-GAAP SG&A expenses of 135 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 for Q&A.
Thanks, Hunter. So, summary, yeah.
Speaker Change: Again, hopefully what you're hearing, Rhythm continues to grow by all metrics, I'll give you one additional metric, we're about 300 people now employed at Rhythm from Rhythm Pharmaceuticals,
Speaker Change: to manage the diverse set of activities we have going on. And as I look across everything, our commercial efforts as you've heard, BBS launching, progressively around the world, global expansion efforts and then our development efforts, we're in a really good place.
Speaker Change: I've been in this industry again, as you all know, for a very long time, much of that time spent in clinical development, and I have to say...
Speaker Change: I don't know if I've had another phase three set of data that has been more rewarding in terms of the fun you have analyzing that data said and realizing the impact that it is having it will have hopefully for a much larger number of patients going on.
Speaker Change: We feel good about where we are, look forward to your questions and that will turn it over to Q and A.
Speaker Change: Thank you. As a reminder to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again.
Speaker Change: Our first question comes from John Wolleben with Citizens You May Proceed
Speaker Change: Hey, thanks for taking the question and regrets on the update. David, I hope you can talk a little bit more about those non-responders you highlighted. It sounded like several of them were having good responses but dropped out, so what were the reasons for those discontinuations?
David Connolly, David Connolly, David Connolly,
Speaker Change: Yeah, I mean, again, there were a variety, and they were across both placebo and treated. They mix from just, you know, patients withdrawing because they couldn't keep up with the clinical trial, either themselves or the family managing it, so that was about half of the group. And then the other, including some of the small number of the placebo group or due to reactions, so GI injection site overall. [inaudible]
Speaker Change: You know, the patient who had the seizure disorder preexisting had uncontrolled seizures during the trial and ended up actually dying of uncontrolled seizures at the end of the trial. That patient did not complete so that's not patient. [inaudible]
Speaker Change: I think as I've explained what I was trying to explain with when patients drop out their data does get imputed and the way you know companies are guided to handle that is done in the most conservative way which is not surprising the FDA wants to make sure that they're not in any way.
Speaker Change: Getting to a robust result here, so the imputation is done by using data from the placebo group in terms of their trajectory over the course of the trial and that value is used to create the or generate the imputation values. [inaudible]
Obviously the
Speaker Change: placebo patient group here, gained weight. So if you're using data from that data set, it's going in the opposite direction of what might support evidence of an effect. So that's why I was just highlighting that three of the patients out of the aid who dropped out had already reached a point of response at the point they dropped out but the imputation then took them down below that 5%. [inaudible]
David Connolly, David Connolly, David Connolly,
Can you talk a minute about real-world persistence? Um, um,
Speaker Change: and understanding of expectations for adverse events and how that understanding keeps patients on drugs or not, like what's the real world experience and how that translates from the clinical data?
Speaker Change: Yeah, I mean, you know, we can talk this more. I mean, what we talked about generally across all of our
Patient populations treated on our commercial experiences [inaudible]
Speaker Change: You know globally with a number around 30% ish of patients who discontinue. In the H.O. world and we've gotten this question, are we gonna see fewer discons there? [inaudible]
Speaker Change: The numbers still relatively small, but I think the answer is yes. I mean, the discontinuation rate in this trial, again, was less than 10% off the total 143 patients that were enrolled. Compliance looks like it was extremely high overall. And so, you know. [inaudible] I'm not sure. I'm not sure. I'm not sure. I'm not sure.
Speaker Change: Part of my goal in giving you the background around the medical complexity is, yeah, that's the world they're living in, but despite all of that, the importance of continuing Sentinel Antide and the relief they're getting from that seems to be incredibly high. So...
Speaker Change: I think we're going to do better, maybe quite well in the H.O. population with regard to both discons and compliance.
Dr. Topple, thanks for the color.
Thanks.
Speaker Change: Thank you. Our next question comes from Derek Archila with Wells Fargo, he may proceed
Speaker Change: Good morning, and thanks for taking the questions and congrats on the progress here. Just two quick ones from us. Just for part C for RN718, I know you highlighted enrollment will complete by the end of the year, but we just wanted to confirm whether you actually plan to share any efficacy data for patients reaching 16 weeks.
Speaker Change: before the end of the year. And then the second question for Hunter, you noted some changes on compliance during the quarter so I was wondering if you could just elaborate on that and I guess how we should be thinking about that trending for the rest of the year. Thanks.
Speaker Change: I'll take the first one and turn it over to Hunter.
Speaker Change: So yeah, part C is just getting underway, as you know, it's...
Speaker Change: As we say, and it's true obviously, it's hard to guide until you know the trial is really up and running and you're enrolling at a more consistent rate here, so we're just at the beginning. The reason we're guiding to hopefully being able to say something by the end of the year, so we're not targeting necessarily a specific number of patients. So for example, for the DIVA Melagon trial, that's a blinded for our trial 28 patients, you got to enroll 28 and got them to the end. But in the open label effort here for 7.8, it's also true for Prader
Speaker Change: Willie, with the open label, we just want to get to a robust number which is why my hope and expectation is is that we will have enough patients treated for a long enough period of time that we will have something to say by the end of the year. [inaudible]
Speaker Change: Derek, on compliance, we generally have seen compliance sort of in the low 80% zip code, and we had a very strong compliance rate in Q4, which was a bit of a tailwind in Q4, and it returned to a more normalized level in Q1. [inaudible]
Anderson, okay, thank you.
Thank you
Speaker Change: Our next question comes from Tazeen Ahmad, with Bank of America, he may proceed to the end of the day.
Tazin Ahmad: Hi, good morning. Thanks for taking my question. I'm sorry if this was part of Yann's comments like
Speaker Change: couldn't hear him clearly, but can you just talk to us about the importance of these? [inaudible]
Tazin Ahmad: Continue with data updates that you're expecting to make. Now, a conference is going forward. Thank you very much.
Speaker Change: You know, what's our understanding from companies that we've spoken to or doctors that we've spoken to, that they think that the data is pretty much perfect. So what additional data do you think is necessary?
Speaker Change: Yeah, I'm sorry, I'll take that one. Also, look, that's sure if your connections back solid. Yeah, no, it's a fair question. I think, you know, my view of particularly the rare disease world is
Speaker Change: Every patient is incremental and nuanced, in a sense, particularly when you're early on. So, you know, the French patient data that we presented last fall was there was only eight patients, but it was incredibly important data. I think we all realized because it highlighted a group that we hadn't studied in our phase two, meaning these are all adults. [inaudible]
Speaker Change: and, with the mean age of 30, and they were also out about 11 years from the time of their insults. So it addressed...
Speaker Change: Based on small numbers, an important question there which now this phase three trial is also highlighted or answered I think which is it doesn't matter whether you're an adult or pediatric patient you're going to respond well [inaudible]
Speaker Change: What is the value of now having a 24-patient update as opposed to an eight? Again, it's just, you know, 24 is a lot more than eight, it's still a small number of patients So it's not that we're going to have every quarter an update on how many patients are treated in France and Italy and some of these other countries which you may have missed I mean we do have...
Speaker Change: You know, individual patients, some sort of name patients settings and a few other countries as well treated for H.O. So we'll do that for a little while but not endlessly. So I don't have to answer your question [inaudible]
Speaker Change: Yeah, and then just on, clashes around seasonality. I just want to clarify, are you expecting to see any seasonality for the remaining quarters of the year? [inaudible]
I mean, Jennifer, you want to take that?
Jennifer Lee: I think overall Q1 is probably the biggest impact based off of what we outlined in terms of those insurance changes that happen just across the board for any patient population.
Speaker Change: and the ability for us to work through with the new pair to regain insurance, that that is what really hit us from a Q1 perspective.
Speaker Change: I think Q4, we don't have a real control in terms of what the Specialty Pharmacy orders, so that may be something that happens in the very last couple of orders of the month and December , but beyond that, we haven't really experienced a lot of other seasonality throughout the year. Just add to that, if we look at our pattern of...
Speaker Change: Prescriptions written and approvals for reimbursement received with the exception of the impact on change in the calendar year. We don't see a significant difference between the quarters. [inaudible]
Okay, thank you [inaudible]
Thank you.
David Connolly, David Connolly, David Connolly, David Connolly,
Speaker Change: Our next question comes from Phil Nadeau with Deity Cowan, you may proceed.
Phil Nadeau: Good morning, congrats on the progress and thanks for taking our questions. A few from us. First, David, you mentioned in your prepared remarks that you have an FTA meeting schedule. Can you discuss what elements of the filing need to be earned out with the FTA at this point? Any uncertain teasers the meeting more formality? Yes, please.
It's...
I can just say... [inaudible]
Phil Nadeau: For the past five years, I'm thrilled anytime we can get more meaningful interactions with the FDA, meaning just they're busy and have a lot to do. What I was excited about here is we've got an in-person meeting [inaudible]
Phil Nadeau: This will be my first in-person meeting in about five years, so looking forward to that. In terms of why the meeting yet, this is a you invariably have they get called different things but a meeting before a filing and it is to clarify what we're presenting in the filing, how we look to present the data, make sure they're all aligned with how that's going to come through. Thank you.
So, that's it.
Got it
The second question is on the Q4.
Hunter Smith: Hunter, you noted that there was somewhat of an impact because of the time to get insurance.
Reauthorizations, and the bridging
Speaker Change: of Patience on Unfoldering. Could you quantify that impact in the quarter? Do you have a sense of how much revenue was hit by that in particular? [inaudible]
It was about a $70,000 effect. It was about a $70,000 effect.
Speaker Change: Perfect. And then the last question is on the paramix. Can you mind us what the paramix is for the S-between Medicare and Medicaid and private pay and where do you expect that to go for the H.O. population?
and Yann Mazabraud.
Speaker Change: Approximately equal split between the two with Medicare being a smaller portion in terms of impact of the BBS patient population, and we are currently still evaluating the HO opportunity to better understand what that parent makes looks like.
Great. Thanks for taking my questions.
Thank you.
Speaker Change: Our next question goes from Samus Fernandez with Guggenheim Securities, he may proceed to the next question.
David Connolly, David Connolly, David Connolly,
Great, thanks for the question. [inaudible]
Speaker Change: You know, two really kind of focused in on the Bibamelagon opportunity and how you see this advancing in the pace at which you can kind of bring that product forward should the face to program, deliver the profile that you anticipate, which again if you could just remind us.
Speaker Change: The profile that you're expecting, I recall, David, you saying that? [inaudible]
Speaker Change: Your hope for that product is that it would deliver efficacy comparable to what we've seen.
N.H.O.
Speaker Change: You know, with several out of the tide, but excluding the, you know, the impacts of MCR1 and the hyperpigmentation, so that's my first question.
Speaker Change: Yeah, so you put the right emphasis on my hope, you know, see what the data shows, we have, you know, now answering that question and these answers involved a little bit but they've evolved since I've seen the phase three data. I think given the efficacy of semilanetide [inaudible]
Speaker Change: as shown in phase 3. The threshold here is really to be better than 10%.
Speaker Change: So I was, you know, what's clinically meaningful, that's certainly clinically meaningful. Does it have to be the same as, you know, what we saw with...
Semolina Tide, meaning if we ended up short of the 16.5% [inaudible]
Speaker Change: Without the placebo correction part, does that mean you don't have a drug? I say no, I mean, I think you can have...
Speaker Change: Different formulations, oral versus subcue Many patients might prefer an oral I think you know the GLP one world is a good example of where you know everybody's getting tied up and knots about you know two or three percent differences and GLP one outcomes one clinically makes absolutely no difference at all I mean they're all robust you know results to responses that drug so internally I think our threshold would be we're looking for something north of 10% I think if we get that we have a drug [inaudible]
and then how quickly we can develop it. [inaudible]
Speaker Change: We are developing new formulations which are going to be critical, most specifically for the pediatric population, we'll have both a liquid and a chewable tablet.
Speaker Change: So that's going to be a little bit gating in terms of getting forward. I can't put quite a timeline on that, but what I will say is if we have positive data, we will move aggressively to, you know, get into a phase three efforts specifically in H.O. [inaudible]
Speaker Change: In parallel, we'll look at our strategies for doing DDS in Palm Sea, but we will certainly move quickly to get into H.O. And maybe we have to stagger a little bit in terms of, you know, how he's come into that, you know, depending on availability of product.
Speaker Change: Great, and you know, follow-up to kind of pipeline . . .
Opportunity and questions there. And...
Speaker Change: The PWS opportunity, can you talk a little bit more about how...
You see that opportunity potentially emerging for MC4R.
Speaker Change: specifically targeted agents, largely because we know that the first effort kind of missed the mark.
Speaker Change: I have kind of brought you forward to the willingness to kind of pursue a higher dose here. Thanks.
Speaker Change: Yeah, so let me just remind you a bit on the original study so yes, I think we used too low a dose but it wasn't just dosing in that first trial it was too short a trial and it was a complicated design with crossover and the like so as we've said and I've said I don't think we learned much of anything from that original effort. [inaudible]
Number one, two.
Speaker Change: I think we maybe have some increased level of confidence in the biology here. I mean, D
Speaker Change: This pathway is absolutely part of what is impaired in patients with freight or welling. Now, that's a very complex disease. There's a lot of other things going on, other genes which are deleted and maybe impacting things like behavior and the like. [inaudible]
Speaker Change: makes it more challenging to study. So, with the context that, you know, this is a tough disease and I've always thought, you know, you can have a drug which works and maybe your trial fails just because the disease and the background is so, so challenging. [inaudible]
That's dead.
Yeah, I-
Speaker Change: You know, I put our sort of general probability success here, which is kind of a non-answer, you know, sort of the 50-50 range, but...
It's a legitimate 50-50, and...
Speaker Change: I have to say, so what are we looking for? The bar is lower and greater willy, so I think, you know, H.O., we came in and, you know, we're...
Speaker Change: Yeah, the phase two date, we're looking for something north of 10 we got ended up with 16 placebo adjusted of almost 20 That's not what we're looking for in Prairie or Willie Prairie or Willie Um.
Speaker Change: Nothing helps those patients lose weight. I mean, Osflano's drug just got approved for decreasing their hunger.
Speaker Change: But there's a big unmet medical need, which remains there. So anything that moves the needle there, that's going to be interesting and something I think we'll seriously look forward to take forward.
Great, thanks so much.
Thank you.
Michael Higgins, Philip Nadeau, David Connolly,
Speaker Change: Arnach's question comes from Whitney Ijem, with Kenneth Gordon-Nurie, you may proceed.
Whitney Adjim: Hey all, thanks for taking the question. Just wanted to pop up on H.O. and kind of the launch trajectory versus BBS. I know you've talked a lot about the specifics of the H.O.P. patient population that should lead, that hopefully will lead to kind of fast rep taker or a different launch trajectory. I think most of those comments have been...
Whitney Adjim: Thinking about the US, or contemplating the US, but it seems those same things should be the case in Europe with potential for maybe even
Whitney Adjim: More momentum, just given some of the early access, progress that's been made on the H.O. Front there. So just curious if you can talk a little bit more about how to think about H.O. uptake in Europe specifically relative to what we've seen in BBS so far.
Yann Mazabraud: Yeah, Yann, you want to give it another shot and we'll just see how your connection is. If not, I'll take it back.
Yeah, it would fly [inaudible]
Thank you. Thank you.
The first is that, as I've stated,
The engagement with the...
and...
Israel Yann, Yann, Yann
Speaker Change: I'm going to stop you just because we're losing too much of what you said there [inaudible]
So I'll give you my...
Speaker Change: Answer, Whitney. There's a lot of things about Europe , rare diseases in general, H.O. specifically, which are of course advantageous from a launch standpoint and that the centers of excellence aspect of this patients tend to be more concentrated. That said, I think the dynamic in Europe , which is going to be similar to the U.S. is...
Heavily concentrated in the endocrinologist, these patients they have...
Speaker Change: You know, very high percentage of them have two or three insufficiency and they are chronically managed by endocrinologists and so to that extent it will be very similar between the US and Europe in terms of our approach to that market and then you know the the rate of which they come on I don't know if it's going to be different I mean they have different reasons for you know why make it faster slow and slower either in the US or Europe and I think you know what we've seen you know PBS for example I'm not been so different and I think that maybe the case here I do think
Speaker Change: As we've said, and we'll continue to say, H.O. is a very different opportunity than BBS, so some of that will be reflected in our launch trajectory of course.
Michael Higgins, Philip Nadeau, David Connolly,
Speaker Change: That's helpful. And then thinking about the label, can you just remind us how you are thinking about that? Currently, I know I think we're expecting an acquired H.O. specific label, but should we be thinking about BMI requirements or age or time from injury, any other kind of parameters that we should be thinking about?
Speaker Change: So, don't know, we have had zero interaction on our label to date, so I don't have any insight there. In general, FTA labels your drug based on how your inclusion exclusion criteria are written in the trial. [inaudible]
Speaker Change: The VMI criteria, FDA is moving away from that, so I'm actually not expecting that to be part of it, meaning the threshold of, you know, 30 being a magical number whereby, you know, above and below at your obese or not obese [inaudible]
Speaker Change: So I don't expect hopefully that that will not be of it because it's not helpful and the other thing for H.O. which I think they will understand. This is about the change in trajectory. It's not about an absolute level of weight or BMI. It's about. [inaudible]
Speaker Change: You were at a level, quote unquote, relative normality before you had your injury or your insult, and then following the injury, then you have a rapid acceleration of your weight gain increase in your pain.
Speaker Change: So, long story short, I don't think it's going to be there. Age-wise, we went down to four, we've gone down to, we're approved for two and above for a BBS in Palm Sea. I think, you know, we just trial only studied four, so four and above and we had a four-year-old in the trial, so I would expect minimally we'll get a four and above in terms of age, so not really an age restriction on what we're seeing here.
Speaker Change: And then, we have Hunger in the Label in Europe .
Speaker Change: We have tried to get it in here that we have the ability to promote. We have data on hunger in the label. We'd like to get it in the indication statement. To me, you know, that's been more about our inclusion, exclusion criteria. It's a key secondary in this trial. We hit hunger. We're going to go back and try again. And I think that's, you know, it's an important part of the indication statements. We'll work hard to get it. But, you know, to be seen. We'll work hard to get it.
Awesome, thanks so much.
Great, thank you.
Speaker Change: Our next question comes from Dennis Stain with Jeffries, you may proceed.
Thank you very much. Thank you.
Speaker Change: So consensus is HO revenue ramping from around $55-$60 million in 2026 to
Speaker Change: A billion plus in 2030. So there's quite a steep ramp in just four or five years of launch. Can you talk about your levels confidence that the launch will be solid? And what are the different moving parts that could try revenue higher or lower than consensus? Thank you. Thank you.
Speaker Change: Yeah, Dennis, thanks. So I think consensus and ourselves, and we're all continuing to work a problem, none of us know exactly. So I'm not going to guide on the revenue piece of it specifically. I'll reinforce the elements, try to answer the other parts of your question, fundamentally different. And, you know, I've talked before, the numbers here, 5 to 10,000 in the US and we're numbers in Europe . Thank you.
Speaker Change: aren't arguably that much larger than BBS, right, 4 to 5,000 in BBS. But of that 5 to 10,000, a much, much higher percentage of those patients are diagnosed. They're visible, they have complex, and they're actively being managed by specialists, and that specialist is endocrinologist.
in about 50% of the patients of the writers.
Speaker Change: Scriptwriters in the U.S. for PBS are happening to be endocrinologists. So this is a world we're in.
Speaker Change: H.O. will be even more concentrated in the endocrinology world and it's Jennifer and her team continue to work to develop the plans for launch, we will approach this in a different way than we did with BBS. In a rare disease world where the majority of patients are not diagnosed, you're trying to build a system that helps them get to a diagnosis. You don't find the patients by knocking on doors, you're really blinded by...
Speaker Change: Working with a system and then a patient gets to the diagnosis and often they find us, some of them we find them.
and this, which is much more specially like. [inaudible]
Speaker Change: If they're concentrated in a specialty, you can cover that specialty. And our goal will be in a teared way to cover certainly those endocrinologist which is significant number where the majority of these patients sit. So all of that says it should be a different ramp than we're seeing with BBS. [inaudible]
Speaker Change: Now that said, it's still a $375,000 a year price point. There'll be no change in price.
Speaker Change: That puts a natural drag on any launch you have a prior authorization as you go through and so there'll be a balance between some of this. We have a huge advantage of having been out there around BBS. It's not like we're new to this.
Speaker Change: Community, they know us, increasingly people know the drug, and increasingly people are seeing the importance of MC4 pathway as being a, you know, this is a critical fundamental underlying biology. So all of that, you know, and how that nets out, I don't know, but I'll summarize and leave it with, you know, yes, it's going to be different trajectory, I can't comment on, you know, whether that those numbers in consensus are going to be the ones we're going to hit. [inaudible]
David Meeker, David Connolly, David Connolly,
Great. Thanks for the call.
Thank you [inaudible]
Speaker Change: Our next question comes from Leland Gershell with Oppenheimer, he may proceed
Leland Gershow: Good morning, great to see the consistency across the age subgroups [inaudible]
Speaker Change: in the HSO study. A couple of questions from us. First question for Hunter. Plodges if I missed it in the discussion about inventories, but should we expect lumpiness this year as we model quarterly revenue? Should we think about another fourth quarter kind of inventory stocking? And I also have a couple of questions for Jennifer. Just on your comments about the, about the steward of
Speaker Change: For some prescribers, turning into repeat prescribers, is that principally limited by specific number of patients who fall under insuries for indications or their other factors that may be limiting the prescribers becoming repeat prescribers? Thank you.
Speaker Change: A normal pattern, you know, SPs do tend to do a little bit of stocking during the fourth quarter [inaudible]
Speaker Change: It's a way they try to manage there. We believe it's a way they try to manage their gross margins because they think there may be price increases coming in the new year. It's not isolated to Rhythm, obviously it's a very common factor across the industry. [inaudible]
You know, some of the lumpiness occurs by things as [inaudible]
Speaker Change: Arcane as the fact that the quarter ended on a Monday, which is the day, Monday 31st of March, which is the day when SPs usually place their order, but they don't receive it until the next day. This is the day when SPs usually place their order, but they don't receive the order, but they don't receive it until the next day.
Speaker Change: So, that type of thing, you know, if you would like to try and forecast that level of minutiae, I-
Speaker Change: Wish you luck, I can't do it, but again, overall we don't think the moves in days on hand are going to be this significant. We think in general they will sit in sort of the 10 to 15 day range on average.
Speaker Change: and the rest around that will just be the vagaries of timing and to some extent ways they may try to manage their own gross margin.
Thank you.
Speaker Change: follow on to what David has been outlining. I think for an indication like VBS, we sort of have smaller.
Speaker Change: Little bread crumbs where the teams are really scouring to get to a physician who may have this type of patient population and educating them so that they are aware so that when that patient comes to them, they are able to get that patient to a diagnosis. That's it.
Speaker Change: For H.O. in contrast, I think the breadcrumbs are much larger, that will lead us to the right position more quickly in terms of being able to educate and engage with that physician population. [inaudible]
Speaker Change: The, once a physician actually gets a patient to a diagnosis, then they realize that this is something that they should be aware of as this patient with all these different symptoms comes to them and it just becomes more of a piece of
Speaker Change: These patients could be anywhere and sometimes they go to a physician who has already had experience with a BBS patient and other times they're in the hands of someone who that is the first patient diagnosed. I will say that once that physician diagnoses and has a great experience on them's history, then they are more likely to also prescribe this drug for those particular patient populations. So it's a bit of a mix of that in terms of where the patients land in terms of care and also the positive experience.
Speaker Change: Williams after touching and seeing what emceevery can do for the BBS population that results in a repeat prescriber. [inaudible]
Speaker Change: And so, just to reinforce, again, what Jennifer said, why don't the one third of patients all prescribe another drug? As a rule, as she said, if you get one patient on you have that experience, you will believe her, then it's more a question of do you have other patients?
That's very helpful. Thanks very much.
Thank you
Title Microsoft Office Word Document MSWordDoc Word.Document.8
Speaker Change: Our next question comes from Michael Ulz, with Morgan Stanley , he may proceed
Speaker Change: Hello, this is Selina on from Mike. Thanks for taking our question. For the congenital H.O. sub-study, could you describe the patient mix that you're seeing among the different two-day deficiency disorders included in any learnings on reliably diagnosing congenital H.O. Thank you.
Speaker Change: Yeah, I know we're way too early to answer that in any kind of meaningful way. I mean, I think the list we've talked about of, you know, SOD, Sepuloptic Displasia, Petritory Stock Interruptions, and from multiple pituitary deficiency.
Speaker Change: I mean, we're going to see all of those. Right now, I would say the SO deep to a Terry Stock maybe interruption, maybe to the more comfortable. We're so early, I just can't answer that in a meaningful way about how it'll break out across the different quote unquote categories.
Thank you.
Thank you.
Speaker Change: Our next question comes from Joseph Stringer with the Neededman Company he may proceed.
Joseph Stringer: Hi, thanks for taking our questions. For the intensive relaunch of BBS, can you disclose the number of written TRX and reimbursed TRX in the US in the first quarter?
Joseph Stringer: Yeah, we're not breaking those out, Joey. I mean, we did in the beginning just to give everybody a sense because we didn't have revenues that spoke meaningfully to where we are. I think now we're very much relying on the revenues, which are more holistic view of how the market's doing.
Joseph Stringer: That's right, so we gave it color around it, but we're not gonna break out the exact numbers. [inaudible]
Okay, thank you [inaudible]
But yeah, thanks.
Speaker Change: Thank you, and as a reminder, to ask a question, please press star 1-1 on your telephone. Our next question comes from Raghuram Selvaraju with AT Rainwright. You may proceed.
Raghuram Selvaraju: Thanks so much for taking our questions. Firstly, I was wondering if you could comment on any qualitative differences that you expect to see.
and implement. [inaudible]
Raghuram Selvaraju: in the context of the commercial rollout in hypothalamic obesity relative to the other indications where it's already approved. I'm just trying to look here for any changes in broader marketing and promotional strategy that you might utilize in this specific indication.
I think that...
Raghuram Selvaraju: The trajectory is really based off of how quickly you can identify the patients because usually and is the case here about BBS and H.O. There was a need for an additional therapy. [inaudible]
Raghuram Selvaraju: We have done market research. We've gathered feedback from our field teams who have started engaging with positions just around.
Raghuram Selvaraju: that are understanding the H.O. market. The pieces that, you know, make us feel very positive about the opportunity is that consistently, the, the, the, the, the,
Raghuram Selvaraju: The comments expressed about the unmet need in this particular population is so incredibly high we hear that from patients and caregivers as well.
Raghuram Selvaraju: as I outlined, just in terms of our blinded TPP, which we went out with slightly less efficacy than what we actually saw for a top-line data perspective, a reaction to emphsivary efficacy as well as overall profile has been...
Raghuram Selvaraju: Very overwhelming in terms of positive feedback from the physician population.
Speaker Change: So those two pieces are, you know, sort of right in terms of the need that exists and the other comment that David had outlined, you know, for H.O. these patients because of all of the other clinical manifestations due to what they went through from a treatment perspective. [inaudible]
They are in the hands of these endocrinologists.
Speaker Change: You know, the vast majority of them, like 80% of them are still seeing endocrinologists so that specialty call and that ability to go to that particular physician set with the data that we have to be able to hone us down to those even within this specialty. [inaudible]
is much higher.
Speaker Change: Then, like I said, the breadcrumbs that we started with in BBS were there was no ICS.
Speaker Change: DC-10 code, they're scattered amongst various different physicians. So I think those dynamics make it a more interesting and exciting potential launch for us.
Thank you.
Speaker Change: Great, and then, secondly, very quickly, in the wake of the rare stone termination, I was just wondering in the context of the broader international strategy, how you intend to go forward in China, if you are looking at the possibility of bringing in a replacement partner or if...
Speaker Change: Your assessment of the China commercial opportunity has kind of signaled an evolution and you're thinking about how best to penetrate that opportunity. Thanks.
Speaker Change: Yeah, no, thanks. So, when we were thrilled to have that, you know, the rights to China back, it's just-
Speaker Change: Still good about having global rights here, number one, number two. We absolutely are expanding globally. I'm really excited about the Japan opportunity. I'm going to talk about that on this fall.
Speaker Change: That's going to be a very meaningful opportunity, H.O. I'm China itself. We learned a lot in our partnership with the Rare Stone and it was incredibly helpful.
you know, in that sense, in that.
Speaker Change: You know, they did some good work. Other work had been done. The genetic side of this opportunity is probably less in China, not that it's not there. But HO is absolutely there. And I don't have numbers for you today, but we know it's there. So we will have a China strategy. Today, I wouldn't say we're committed any particular course of action, meaning we go alone, which I think is...
Speaker Change: We can go alone in Japan with more challenging perhaps to go alone in China so that would suggest we'd need a partner but all that's to come but I think you know China and Asia you know in a larger sense you know Taiwan
These are on.
So.
Speaker Change: Those are meaningful opportunities and we'll work our way through that and keep thinking about what's the right strategy for China.
Thank you.
Thank you
[inaudible]
Paul Enties: Our next question comes from Paul Nadeau with Steeple You May Proceed.
Paul Enties: Hey, sorry about that. I think I saw it this year earlier. I appreciate you putting me in. Just going back to business in Oregon really quickly. Anything you guys can say about safety from the study given that it's pretty far along now, anything just on the idiosyncratic safety side related to liver or anything else that kind of you're seeing that would give you more comfort in the molecule. And then I just wanted to clarify, David, on the bar, I mean 10% makes sense, but this study is is menopely shorter than the semolina time phase three. So
Paul Enties: Are, how do you think about the bar for this shorter face, too?
Speaker Change: Yeah, that's a great question, Paul. Thank you. So on the safety side, again, you know, it's blinded, but obviously you follow the safety and a blinded sense and
Speaker Change: All I will say there's no significant safety signals here that we're concerned about. You know, I don't have fine detail in terms of looking at individual patient's LFT data, but there was a...
Speaker Change: Sirius Adversive and Related to LFTs, we would see that so we're not seeing any of that so I would categorize our blinded view of all that is quite reassuring number one.
Speaker Change: And then two, your question on the bar, it's a good one, which I always...
Speaker Change: I don't like being boxed in, you know, particularly around data. There's always contacts around data and particularly in a rare disease. I mean, the means are...
Speaker Change: I'm not at all interested in the mean value so much, I'm very much interested in patient-by-patient and-
Speaker Change: You know, if the mean doesn't get to 10%, but you can explain the couple patients that were dragging the mean down then okay you still got a drug so we'll look at the data that way. You know one way of looking at this is our phase two data if you remember with semilanetide was 16 weeks.
Speaker Change: You know, they did hit, you know, almost all of those patients had 10% or greater or were on track to lose 10% or greater. So I think 10%'s not an unreasonable target here in terms of using set as the bar, semi-identity as the bar, but they'll be context that we'll have to take into consideration. [inaudible]
and it's 16 weeks is relatively short. [inaudible]
Thanks, Hans, thanks a lot.
Speaker Change: Thank you. I would not like to turn the call back over to David Meeker for any closing remarks.
Speaker Change: Okay, well thanks everybody, long call this morning, a lot of questions, we'll appreciate that and we look forward to our next update for the next update.
Speaker Change: Thank you. This concludes the conference. Thank you for your participation. You may now disconnect.
No one
Michael Higgins, Philip Nadeau, Hunter Smith, David Connolly, David Connolly
Michael Higgins, Philip Nadeau, David Connolly, Susan Phillips, Rhythm Pharmaceuticals Inc