Good morning, everyone, and welcome to the Goldman Sachs healthcare conference. We're thrilled to have David Meeker, the CEO of Rhythm, here today joining us. Before we get started, we are required to make certain disclosures and public experiences about Goldman Sachs' relationships with companies that we discuss. The disclosures relate to investment banking relationships, compensation received, or 1% or more ownership. We are prepared to read aloud disclosures for any issue or upon request. However, these disclosures are available in our most recent reports available to you as clients on our firm portals. The views stated by non-Goldman Sachs personnel do not necessarily reflect those of Goldman Sachs. Okay. With that done, thanks for joining us, David.
Thank you, Corinne.
Maybe we can just get started on the recent data that you guys reported in HO. It demonstrated a significant improvement in BMI across both pediatric and adult patients. Maybe you could just start by level setting with the highlights from that data.
Sure. Needless to say, we were thrilled with the results. We were optimistic coming out of the strong phase II, but that was only 18 patients and an open label. This phase III was 120 patients, randomized two to one, 80/40 roughly. The primary endpoint hit robustly with a placebo-adjusted difference of 19.8%. There were two things about that. One, very strong, obviously, on the impact of setmelanotide itself on BMI, but also, very importantly, it reinforced what all the KOLs have been saying, is that nothing really works in this and that these patients continue to gain weight. If they just had some drug that could keep them from gaining weight, that would be a victory. The placebo group gained 3.3%, the setmelanotide group decreased by 16.5%. That was the headline.
If I'm going to go a little deeper here, there were a few other things. What's always been striking to us, and it was true in the phase II, I mean, we've been happy with the percentage change, but we've also tried to stay a little bit out of the GLP-1 wars of the 2% difference is clinically meaningful, and people are so focused on that. What's really remarkable about this data set has been the consistency, again. Pretty much, and it's consistent, that concept, the consistency of response, is very supportive of this underlying biology, which we think is we're treating people with a deficit in this alpha-MSH stimulating hormone. When you correct a hormonal deficiency, take hypothyroidism, you should have a very high percentage of response. I mean, you're just replacing what's missing there.
The consistency that we saw below the headline data was we had stratified for three age groups, six to 12, 12-18, and 18 and above. There had been some early concerns before the French data came out last fall, maybe it works better in kids and adults. Almost unbelievably, it was identical across all three, 19%-20% placebo-adjusted changes for all three age strata. Those were, as I said, we felt really good about the efficacy data there. We can talk about the GLP-1 data also if you want, but overall, the headline was strong.
Yeah. You mentioned the consistency of the data. I guess you talk a lot about some of the individual patient stories. For the few patients who did not respond, could you just give us a little bit of color on that?
Sure. We had 80% of the patients lost 5% or more. If you take 80% of the 80%, there were about 17 patients who did not make the 5% at the end. Eight of those 17 patients did not complete the trial for one reason or another, so they had to have their data imputed. There is a way the regulators work, FDA specifically. It is a very conservative method. You do these simulations, and that is how you impute the data. The data that informs the simulations is drawn from a placebo group. If you have a placebo group that is gaining weight, and then somebody drops out of the trial, and you impute their data using the trajectory from the placebo, then obviously you take a hit there. Eight of those patients fell in that group, one.
Of the remaining nine, six of them either had passed the 5% mark and fell back for one reason or another, or they were kids who, despite not reaching 5%, did have a greater than 0.2% change in their BMIV score, which for a growing child is clinically meaningful. I think overall, it was very, very consistent with the fact that most patients respond, not 100%. That got us down to three. Of the three patients who did not fall in one of those two groups, two out of the three, we have documented noncompliance in that we tested trough levels at the midpoint in the trial, trough drug levels, midpoint in the trial, and at the end of the trial. Both of those values are below level of quantification for those patients.
Okay. Helpful. Maybe on the safety and tolerability side, could you speak to what you saw in the study and comment on some of the SAEs that popped up?
Yeah. So overall, the safety of setmelanotide was first approved in 2020. It's been out in the Bardet-Biedl population since 2022. So. And we've treated probably well over 2,000 patients. So there's a very strong safety database behind the drug. What we saw in this population was very consistent with what we've seen across. It's GI-related side effects for the most part, hyperpigmentation, where 50%-60% recorded as an adverse event, but 100% of the patients probably have some change because it's an on-target, off-target mechanism. The serious adverse events were interesting. There was a distinct imbalance there. We had 27% roughly SAEs in the treated group versus 7% in the placebo group. As you broke those down, and I've now, which I didn't have at the time of our original earnings call or original call following the top-line data readout.
I've been through every one of those cases in depth. It's a series of one-offs, and there's really not a good pattern there. For example, there was a couple of COVID infections in that group. We didn't have any, there was none in the placebo for serious adverse events, but the percentage of COVID infections overall was exactly the same, 11% and 5%. It matched very much the randomization 2%-1% . There were three infections there: pyelonephritis, appendicitis, and cholecystitis, which, again, there's nothing about the drug or anything. I mean, each one of them had a reason why they might have developed that infection. I think the one thing that, and I think this will be part of our label and that we will want to make sure people look at, this is a population that's incredibly medically complex.
They have literally one to two pages of medications they're on between all of the different hormonal replacements that they're on. They may have a seizure disorder. They may have depression. They may have, so again, there's a plethora of medications they're on. It's also a group that has never been able to lose weight like this. As you start losing weight, if you don't pay attention to the other medications and their dosing, and the most specific one, and there was a handful, there's two that stand out. One is this diabetes insipidus. I think now it's being pushed to call it arginine vasopressin deficiency. That's the hormone itself. That's the hormone that allows you to concentrate your urine. It's injured in the hypothalamus and the pituitary. It interacts between the two of those is how it works.
If you have diabetes insipidus, unable to concentrate, you have to take your hormone regularly, daily, and you've sort of modulated. The parents and families adjust these or patients adjust themselves a bit. You have to hit fluid targets. You have to keep drinking a lot. Anything that throws that off, if you got a GI, so it could be GI-related stuff related to the drug, but often just GI stuff in general, your sodium will bounce out of whack. We had a handful of patients who got admitted for that. The other significant challenge for these patients is adrenal insufficiency. That's another, again, hormone that's very commonly 80%+ of these patients had the diabetes insipidus, and a similar percentage had adrenal insufficiency. Anytime anything happens, they need to get covered with what they call stress dose steroids.
It is to make sure that when we get sick, our cortisol levels rise, and that is what allows us to deal with that insult. If you do not do it, of course, it can be a quite serious problem. Those were things that complicated. I think my point is the only link to why there may have been an imbalance in this series of one-offs on the serious adverse events is that their background meds may have gotten a little bit off as they were going through these changes with weight loss.
With that in mind, do you anticipate that those patients who have some of these additional complications will still be candidates for setmelanotide in the real world?
100%. As I said, the percentage of those patients are 80%+ of this total population, number one. Number two, the other thing we told us was over time, they were requiring less medication, and it was becoming easier to manage. It is the change itself. It is not the fact that you have it and you forever cannot do it. You just have to be alert as you are managing through these initial changes. If you remember, this drug, based on our phase II experience and consistent with hormone replacement, you do not endlessly lose weight. One of the things about, I think, GLP-1s and concern is, could you tip over into an anorexic kind of world where you are just not? We just have not seen this. You do not develop an aversion to food. You just know when you are full. You leave it.
You may leave food on the table, but you're.
Can you speak to the compliance you saw in the study, and how do you anticipate that data can translate into real-world use?
It was high. If you remember, we had about 10%, almost exactly 10% dropouts overall, 14 out of 143 patients enrolled in the full data set, which was very good, much better than we've seen in our other trials. That was split about half and half between the placebo group and the treated group. The compliance was 80%+ . I do think this is a group of patient population that is used to taking a lot of medications, as we've been told. Another injection really isn't that big a deal. They're paying a lot of attention to these other meds. It's not like you've got something that's intruding in your life. It's just you have your meds. This is now one of them. You have your hormonal replacement. This is now one of them. I think for multiple reasons.
The benefit relative to what they are living with, I think that was, I'll just make the editorial comment that given the complexity and all they have going on in their life, the importance of this medicine and these changes to their quality of life was pretty dramatic. You might wonder whether you can move the needle when you're just adding another med on two pages of medicines, right? This is the issue. It's a big issue for them.
Okay. So then as you think about filing for approval and going through the regulatory process, what are the most important things you want to make sure get into the label?
Yeah. So all the data, I think it'll comfortably go on the label. I don't think there'll be a lot of debate or negotiation there. The piece that we've tried before, we'll try again. Hope springs eternal here. It will be helpful to get hyperphagia into the label. And we have it in Europe, as people know. In the indication statement, we have data on hunger in the label. We can vote on it, and it's not part of it. It will be helpful, for example, as we continue to deal with Medicare. Medicare does not cover today. Anything in the label that would further differentiate us from a sort of interchangeable chronic weight management drug will be extremely helpful. That's probably our biggest push.
Okay. Then as you kind of move towards that, you also are presumably preparing for commercial launch. What are some of the key commercial tasks you need to undertake between now and when you get to the approval?
Yeah. I mean, this is interesting for so many reasons. I'll go back to Bardet-Biedl. Bardet-Biedl syndrome, as you and I have discussed, is really a classic ultra-rare disease. 4,000, 5,000 patients in the U.S., most of them majority undiagnosed. They're spread out. They don't tend to concentrate in any. They may see a specialist to get to a diagnosis, but then they get sent back to their primary care because there's nothing for that, no reason for that specialist necessarily to keep them. So Bardet-Biedl, we've talked about the steady cadence. The reason it's steady is you just have to do the work each quarter. You find them, bring them through the reimbursement process and the like. You don't build a structure to knock on all the doors because you couldn't, and it wouldn't make sense.
You try to help build a healthcare system, a community that is better at diagnosing the patients. And then as many patients as you find, they will find you. We see this each quarter in our, there's 25%, 30% of physicians writing scripts, amazingly, still three years later, who we don't call on, we don't interact with. That's the they're finding us part of the equation. This is different. HO is different. The numbers, at least our current understanding, 5,000-10,000, maybe not so different. It's still, by every definition, an ultra-rare indication, but very well diagnosed is our belief based on the fact that it's a recognizable clinical presentation. They have an insult. There's a before and after. 80%+ of these patients have pituitary insufficiency.
That is really, I think, the key driver is that because of that, they do stay with a specialist. They need to be seeing an endocrinologist to manage this complex endocrine background. We are looking at this. It is ultra-rare, but as a specialty kind of launch. What do you do in specialty? Specialty is a little bit of the dream for pharma/bio-pharma in the world in the sense that you have big unmet needs, high-value transformative medicines, and they are cared for by specialists. Endocrinologist, and we have begun to do the work. We are early on in the claims work and the like, and we are validating that claims work with the people in the field. There are about 10,000 endocrinologists, 2,500-5,000 of those probably have well over 80%+ of the patients based on our current understanding. We will cover them.
We will build a salesforce that is capable of knocking on all those doors. It is now a bit of a reach and frequency kind of game. One of the things, and we have heard this with other companies' challenges, all specialists, but certainly including endocrinologists, who are a bit overwhelmed with the one demand, I think, is jamming up that part of the equation. When you have a new therapy for a big, frustrating, unmet medical need, you can get access. Our early experience is people want to hear about this, and they want something for these patients. We are getting in.
Perfect. Okay. Maybe we can move on to talk about some of the next-generation agents in HO. You've got an oral and a weekly that are currently in development. Maybe let's just start with high-level how you think about that franchise. What are some of the advantages you hope to bring for patients with the oral and the weekly programs?
Yeah. The initial obvious one is just more convenient, daily oral versus daily injectable or weekly injectable. The other is our current setmelanotide does hit the MC1 receptors. We have the hyperpigmentation. It's not 5%-6% of the total number of patients we treat in the real world discontinue because of the hyperpigmentation. There's probably a little higher number that they may not call that out as their primary reason for discontinuing, but they're not thrilled by it. It's not a huge percentage of the patient population, but it's meaningful. There's no value to it. If we could eliminate that. Both drugs were designed with the idea of, let's eliminate the hyperpigmentation. We'll be looking for that in our phase, this first experience in patients.
The developmental strategy behind them is we'll take, if it works, we'll take both of them into HOs. The wonderful thing, if you will, about the hypothalamic obesity indication is because it's so sensitive from a clinical development standpoint, it's a gift. If we had to develop this for the first time in Bardet-Biedl, much more challenging and trying to figure out, are you seeing it? Is it real? Not real. This should be pretty black and white. We'll get I don't know if I answered that question.
Yeah.
Yeah.
It sounds like you're about to go for it, but I was going to say we anticipate near-term clinical data from Biva. How should we think about that readout? What do you kind of see as a threshold to move forward with the program?
Yeah, my favorite question. I spent months answering. What do I think is going to be the percent change for the HO phase III trial? For the Biva trial, what do we expect to see? It's a phase II, 28 patients, and four groups. Seven patients per cohort, three dose groups, and a placebo group. Each cohort is extremely small. I've been really trying to highlight for people, they should not be focused on the means. The means may look great, but with only seven patients, I'm not looking at the means. I'm going to look at seven individual patients for each cohort. If five look good and two do not, then we're going to try to figure out why the two did not respond or whatever. If we have a good reason for that, obvious might be compliance.
You're not going to throw the drug out because the mean looks low. You're going to go with the five that are telling you it's working and looking good. That's just how we'll analyze the data. Thresholds. The way we've been answering this, and it's a little bit the way we tried to answer it earlier, which is in this population, and now that setmelanotide set the bar, 16.5% in the phase III itself, 10% or greater is a drug. Now, it's 10% or greater in an apples- to- apples. The reason I say that is, and we've seen this in other situations, I don't think you need to be as good necessarily. If you are significantly more convenient, you have other features. At 10%+ , many patients may be perfectly happy with that. One.
Two, you get to 10%, not because everybody got to 10%, but because some did much better than 10% and some did not. I think just conceptually in thinking about it, 10% or greater is how that should be the threshold. Now, that is at a year. We are going to be reading this out at 14 weeks. What we will try to do, and it is doable, is when we present the data or put out the high-level data, to provide as much context as we can so people can see the apples- to- apples. What did the phase II look like? The phase II was a 14-week, sorry, 16-week trial. Fourteen, 16, it is close, but actually two weeks at full dose is meaningful. Again, we will try to help people understand that.
We can also take a cut of the phase III data at 14, 16 weeks or 12 and 16, I guess we have other data points. We will try to frame it and, again, give people as much of an apples- to- apples as we can. The other difference in this trial is that it is 12 and above, so we do not have the six to 12. If you go back to our original phase II, we had several patients who were under 12 who did extraordinarily well in that first 16 weeks. They lost 30%+ . Our phase II number was 14.5% in that phase II trial, and it was driven disproportionately by, like I said, a few of these kids who were in the 30% range at that early time point. Anyway, these are the things that, again, small data sets.
What we want to know is we want to know, do we have a drug? This is not we're not setting up the future label here. We just want to know, do we have a drug? Do we want to invest and go to phase III? I'm pretty hopeful that this trial will tell us. If it doesn't work, we'll know. I mean, no response is no response. Then we'll be done.
To your point, you've got a lot of data with setmelanotide, including in preclinical models. So as you look at what Biva has shown, where you can look at apples- to- apples thus far, how did the two agents compare?
Yeah. So I mean, in LG Chem, who we acquired the molecule from, as I've said many times, they did more preclinical models than we've done. And we've repeated some of this now internally. You get identical results. Now, the challenge there is, of course, it's a little hard. When we do 718 or weekly and the setmelanotide, those two molecules are very similar. And almost milligram- to- milligram, they're very similar. Whereas for Biva, you've got a different molecule, oral route of administration. You can get identical results. It's a little harder to take your preclinical dosing and extrapolate perfectly to your clinical setting. People ask me, how do you handicap all of these? I know both 718 and Biva are very good MC4R agonists. All things being equal, they should work. I think the biggest variable, the unknown, which introduces the uncertainty is dosing.
Do we have the right dosing?
Okay. Maybe we can also go through some of the same questions on 718. You can talk about the study that's ongoing later this year.
Yeah. 718 weekly, as I said, very similar. It's a seven amino acid cyclic peptide, setmelanotide, and eight amino acid cyclic peptide. We built it off what we knew about setmelanotide. So a lot of reasons to feel good about the molecule itself. We finished the SAD and MAD part of that. It's fine. We've got the PK we want. Safety is all acceptable, of course. We're getting that trial up and running. What I've said, and still the goal is, the goal is it's an open label study, is to have something to say by the end of the year. I'm hedging now a little bit because we're definitely slower getting it up. It's just been harder getting sites open again. We can talk about that. It's just we're a little bit behind what we want to do.
We're just starting on the 718. I'm still hopeful we can say something by the end of the year, but it may bleed into Q1.
Why has it been harder to get sites up and running? I mean, it's not your first rodeo, right?
It is not our first rodeo, but it is we've had challenges in the past getting and it's just I can tell you it's tough at these clinical sites.
Where is the break? Is it like getting IRB stuff or is it?
It's a mix of things. I mean, your key person in any clinical trial is your study coordinator. And so just depending on how many trials they're running, for better or worse, we're working with sites that are running more than one, just Rhythm stuff, of course. Contracting offices, they work at their own pace. You'd think in today's world with funding being what it is, they'd be thrilled to have a biopharma knocking on the door wanting to do stuff. But it doesn't translate. So those are the stuff. There's nothing unusual about it. It drives me crazy, but it is what it is. And we're working on it.
Once you get the sites up and running, given your experience, how would you anticipate pace of patient enrollment? Is that things smooth out at the time?
Yeah, it should be good. The other piece is we made it a little bit intense. We had a very intense pharmacokinetic requirement. Patients needed to be in the hospital with prolonged periods of multiple blood draws. That is a problem. We have now mended or are mending the trial to much lighten up on that dramatically.
Okay. One of the advantages of these programs from a Rhythm perspective is the patent. Could you just talk to us about the patent on the two?
Yeah. So incredibly important. Let me just start with setmelanotide again because I want to make sure people understand that. Composition of matter for setmelanotide 2032, what you've heard me now, let's talk about it a little bit more. We had not so much in the beginning is our formulation patents for setmelanotide go out to 2034. When you get a drug approved, the FDA regulators, FDA specifically, puts out a guidance document. Now, going forward, that office has been, we'll see what happens. Anyway, historically, they put out a guidance document that tells the generic what it would need to do to develop a drug for that. That guidance document basically takes you through our formulation patents. We are feeling, Rhythm's opinion, like we may be in a reasonably good position there. That would give us coverage to 2034.
The other thing that not everybody, when they're modeling this, they tend to model endocomposition of matter or whatever with a cliff. That does not happen in a rare disease world. It does not happen because, A, the competitor, the generic entry, tends not to compete on price because you cannot make it up on volume. If you cut it, they tend to price it similarly. Companies compete for share. The incumbent in that world has a pretty big advantage in terms of. Anyway, we do not think the setmelanotide world, there would be a cliff. Back to the two new molecules, they are incredibly important because they both, with patent term extension, take to 2040+ .
Okay. Perfect. Maybe last question on the two and the franchise here. As you think about the decisions you have to make around going forward, are they contingent at all on each other? Will you make the decision?
No.
Great.
Independently.
That was the answer I expected. Okay. Maybe on other data this year, you've started to discuss a phase II and Prader-Willi syndrome. Maybe talk about the confidence where you got confidence in pursuing that indication knowing it's been something of a graveyard for drug development.
It has been a graveyard. I mean, it's just that we have tremendous respect for the challenges there. As you know, we ran a trial in 2018, predated me. That trial was negative. Quotes for everybody. It was complex and wrong design in the sense that it was earlier on in development. We were being, as a company, very careful in our dose escalation. The highest dose study was 2.5 mg, and it was for four and eight weeks in a crossover design. In that net, it was uninterpretable to me. We had a couple of placebo patients who lost a lot of weight. If you look at the top dose, the 2.5 mg and the patients who were on for eight weeks, there was a response in a handful of patients, small number. Not uninteresting.
As I said, from a trial design, you could say, well, it doesn't prove anything. It doesn't prove anything. There is hope. Second, biologically, it makes sense. The MAGEL2 gene is in the part of the chromosome that's deleted or imprinted here. MAGEL2, we studied that gene. We studied it in our phase II DAYBREAK study. In the three or so patients who we, based on their genetics, we knew that their variant was a loss of function variant, they had a good response. There is a mouse model. You knock it out, mouse gets obese. You treat that mouse model with an MC4R agonist, they get better. That biology is absolutely clear. It is absolutely present in Prader-Willi. The challenge of the disease is it's not just the hunger and the weight gain.
It's the behaviors which are really challenging to deal with and a little bit independent. The hunger, hyperphagia may make the behaviors worse. If you improve the hyperphagia, you don't necessarily make the behaviors better. That is a big confounder. What we're running now is an open label trial with Dr. Miller at the University of Florida. We decided not to do a control. Lots of criticism might be, well, you won't know anything without control. Actually, I think conversely, you have to run a pretty big trial for that control group really to help you here. The value of working with somebody like Dr. Miller, who just knows her patients extremely well, not all investigators know the patients the way she does. She takes care of them.
I think working with her, her ability to interpret what we're seeing, I think our goal is we should come out of this with being pretty confident it works or it doesn't work. That's the belief and our hope. The bar here arguably is lower. Nothing has caused weight loss. LANO got approved, but reduction in hyperphagia, some behavioral improvements, but not weight loss. It's not totally clear how diazoxide relief is. It doesn't work through MC4R. Different pathway. I think in a world where nothing's been able to show weight loss, if we can show 5% or greater weight loss, that's a drug. We would take that forward.
Okay. You're going up to the 5 mg dose with setmelanotide in this.
For six months, yeah.
Yep. Maybe just talk about where you've used 5 mg in patients before and how you think about the safety and tolerability that you could see emerge at that higher dose.
Yeah. In normal volunteers, we've gone to 7 mg. We did a three-month study comparing our full setmelanotide weekly and a daily setmelanotide. Went up to 5 mg in that study. There's no, I think, safety issue. We have gone individually. There's been a handful of patients in our previous world, some Bardet-Biedl patients, some patients in the original old VASCA study who felt like they needed more. And we've gone up to the 5 mg.
Are you allowing background diazoxide on the trial?
We are. And so there will be some patients there.
As you think about the next steps, if you see a signal with the open-label phase II, do you move forward into phase III from there? Do you wait to see one of the oral or weekly? What are kind of the next steps in this patient population?
Yeah. That's a really great important question. The answer is depends. But it's shifted a little bit in our minds, my mind certainly. I think originally, as we've said, we've been very clear that we do all our new development work with the next gens. Prader-Willi's unique and a big enough unmet need. If we see something that's meaningful there, by far the fastest route to approval is with setmelanotide. It would be a supplemental NDA. We could move, in theory, pretty quickly. So that's the defense part. We'll see how the data looks. But if we feel like it's making a meaningful difference, we'll go.
Okay. As you think about what a phase III would have to look like, you obviously have done a couple of phase IIIs in rare disease populations. You have the diazoxide trials to look at. Any key parameters you would anticipate being required for the phase III?
Everybody says the same things. I mean, we'll use the HQCT, which monitors their hyperphagia and the like. The tools that Soleno uses, everybody uses. I mean, we'll for sure be incorporating those. As I said, we need to see changes in weight loss. The traditional measures there would be BMI. Body composition is really important. We haven't been as consistent getting that in some of our other trials. If we went forward, for sure, we'll be clear on that.
Okay. I guess can you just give us a kind of sizing of the addressable market unmet need in this patient population?
For Prader-Willi?
For Prader-Willi.
Yeah. I mean, the numbers out there for the U.S. population is 10,000- 20,000. So pick 15,000, whatever. I think, again, a very well-diagnosed population. Part of the value that's been ascribed to Soleno in a very short period of time is that they're coming out with the first approval, which was really a huge step for the field, their ability to get approved there. Into a world where there's nothing.
Yeah. Okay. You currently have said your cash runway is into 2027. As you think about kind of sources of capital and funding all of these potential programs, maybe first, what's included in that guidance? We've talked about a number of additional phase IIIs. How do you think about kind of sources of additional funding?
Yeah. So we took some money down from the ATM, which allowed us to give new guidance into 2027. That includes all of the existing clinical trials and our preparations for the HO launch. What's not in there is new clinical development work with the idea that if it was positive, phase III stuff is not in there for that, which includes some investments in CMC. There are things that planning for success arguably we would need to raise for. A big variable in that guidance is revenues and how well we do. I think Hunter and team have taken a conservative view of that. We are not terribly aggressive in this guidance. If we do better there than our needs, as Hunter likes to say, we are not solving for a big number necessarily here.
When we have a number of different ways we could attack it, equity, of course. Stock's in a good position. We could do equity. It ranges full range. I mean, we've got a royalty with HealthCare Royalty and their partners. I think I've expressed a willingness in the past to be supportive of something else if we wanted to go there. We're also maturing as a company. Debt becomes an option at some point.
Okay. In the final minute, I guess, anything else that you view as significant value drivers that we could unlock with Rhythm over the next, let's call it 12-24 months?
Yeah. I think if people, I mean, Rhythm's at a really exciting time. And this pathway is fully validated now. MC4R pathway. I mean, in the beginning, it was just like, can you get your now it's like, okay, the drug's approved. You've got a good MC4R agonist. Maybe you have some next generation agonist as well. But this pathway, which historically always was viewed as important, I think it fell out of favor a little bit because people couldn't drug it. Now it's back in favor. People are beginning to peel that onion and to say, okay, how many different quote unquote indications might this pathway be important in because it's this thing? You've got the phase II DAYBREAK studies. You've got other causes of injury to the hypothalamus we've talked about.
We talked about the congenital form, traumatic brain injury, which hopefully will be part of our current label and the indication. The point being is I think there's a really rich area out there to be explored now that the pathway has been validated and we have a drug.
Perfect. That fits us perfectly at time. Thank you, David.
Thank you, Corinne.
Great job.
Yes.