Rhythm Pharmaceuticals, Inc.

Welcome to the Rhythm Pharmaceuticals first quarter 2022 earnings conference call. My name is Hilda and I will be your operator for today's call. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session. During the question and answer session and at any time, if you have a question, please press 01 on your touchstone phone. I will now turn the call over to Mr. David Connolly, Investor Relations and Corporate Communications. You may begin.

Thank you, and good morning, everybody. I'm Dave Connolly, head of IR and corporate communications here at Rhythm Pharmaceuticals. For those of you participating via the conference call, the accompanying slides can be accessed and controlled by going to the events section of the investors page on our website at ir.rhythmtx.com. This morning, we issued a press release that provides our first quarter 2022 financial results and business update, which is available on our website. And as listed on slide two, today here with me in Boston for the conference call are David Meeker, Chair, President, Chief Executive Officer of Rhythm, Jennifer Chen, Executive Vice President, Head of North America, Linda Shapiro, our Chief Medical Officer, Hunter Smith, our Chief Financial Officer, and Yann Mazzebro, Executive Vice President, Head of International, is on the phone joining us from France. With slide three, I'll remind you this call contains remarks concerning future expectations, plans, and prospects, which constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent annual or quarterly report on file with the SEC. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent dates. We specifically disclaim any obligation to update such statements With that, I'll turn the call over to David, who will begin on slide five.

Thank you, Dave, and good morning, everyone. Thank you for tuning in this morning, and we look forward to updating you on the progress we've made in quarter one. But before I do that, I'd like to start on slide five with a bit of an unusual start here. This is a cartoon of our biology. Many of you know this slide well. As we all know, it's been a particularly difficult moment in the markets generally, and it's been particularly difficult for small and mid-cap biotechnology companies. And I think at moments like these, it's worth looking at fundamentals. They're always important, but particularly so at these kind of moments. So I wanted to spend a couple minutes just reviewing Rhythm's fundamentals. So number one, there's a clear unmet medical need that we're pursuing here. Patients who have A genetic variant that impairs the MC4 pathway suffer from hyperphagia, decreased energy expenditure, and consequent early onset obesity, and all the comorbidities associated with that. Two, the biology is incredibly strong, as highlighted on this slide. It's been well studied. The pathway that we're pursuing, the MC4R pathway, the endogenous ligand, the alpha-MSH, which interacts with the MC4 receptor, When it engages, it decreases the appetite, increases energy expenditure, and you get a reduction in weight. And we've shown in, again, multiple trials that that's associated with other benefits as well. Third, we have a precision medicine, a solution to this problem. The set melanotide is an analog for alpha-MSH, and when it engages the receptor, you get all those benefits. And we are essentially a replacement therapy. It's very simple conceptually and biologically. And we're working in a world where other approaches to try to manage this problem have not been reliably successful. Bariatric surgery, you can get weight loss, but do you get it reliably and sustainably? And that's the same for other approaches to weight loss management. Forward de-risk. Most things fail in this industry, and we've been fortunate enough to get set melanotide through the regulatory process in both FDA and EMEA. We have an approved drug with Another one or two indications imminent coming up. And so from a risk standpoint, this is a company that's passed one of the very major hurdles that we all aspire to. Five, and it's not an endless list here. We know what we need to do commercially. We know what we need to do clinically. And we know what we need to do financially. And we have the team to do it. Now what you're gonna hear as we continue to update you, we're executing and as I said, I feel really good about the fundamentals that we're standing on. So with that, let's go to the quarter. So slide six, we're on track. Let's talk about the US first. We're very much looking forward to our Purdue for data on June 16th. We've used the time well as you can imagine. We've continued active patient identification and disease education efforts. Jennifer will highlight and provide a little more color around that effort. The current commercial opportunity is playing out exactly as we had hoped. We have tens of patients on therapy. We continue to learn more about the market access situation. We are able to educate payers, and those interactions are laying a strong foundation for a BBS launch. And really importantly, as we've highlighted in the past, we continue to get to interact with patients who have consented in to our patient services group, now called InTune, and that's hugely valuable insights as we think about how we can provide the best service for that patient, how we can help them manage through the early part of beginning a therapy like this, the daily administration, the early side effects and the like, that all can be significantly benefited by that strong interaction. The international markets are an incredibly important part of our whole story. I'll talk a little bit more about that in the next slides coming up. But suffice it to say that as we look at Europe, we've highlighted this many times, for all rare diseases, and it's certainly the case in the areas that we're working, Europe is better organized, Singapore healthcare system, Patients get referred, center of excellence gets set up. True KOLs, thought leaders emerge out of that. They have the opportunity to see many patients. They can do research. And so, again, the end result of that is as a starting point, there tend to be many more patients identified. And once you do get approval through the healthcare system for access, the process of then getting patients on to therapy is much more straightforward. And then in our third bucket, we have a broad clinical development program, and it's a thing. We put in a tremendous amount of work to get these trials up and running, and they're now running. M&A, Daybreak, the hypothalamic obesity trial, pediatrics, weeds, weekly formulation trials, all ongoing. We're generating a lot of data, and we're publishing that data, and we just had abstracts released at PEAS, Pediatric Endocrine Society, over the weekend, and we announced a on Monday, new additional abstracts, which will be presented at ENDO, and Linda will highlight those in more detail. And finally, as you know, we're very much looking forward to providing updates, the results on our hypokalamic study and our MC4R rescuable interim data, and that will happen mid-year. So next slide, number seven. So internationally, and as I said, We've highlighted that first commercial patients started in March in France and were underway in their early access program. And with that early access program, we're about a year ahead of where we would be if we did not have that in France. Germany all along has been a real education and a very positive development here. These products, weight loss drugs in general, are viewed as lifestyle products and restricted. We were able to get an exemption from Annex II. And that was just published in the past couple days in the National Gazette, so that's confirmed. Reimbursement dossier is now being submitted, and we look forward to having our first commercial patients in the next couple months in Germany. In the UK, a nice recommendation expected in June, and we will get it. This is not one where, you know, are we going to be approved and able to go forward in the UK? No, we're going to go forward. Final details to be worked out. But that, you know, we've progressed to that stage where we can be extremely confident. Similarly in Italy, final stages of price negotiation. Feel remarkably good about that. That's evolved perhaps even more favorably than we had hoped. Netherlands earlier, but active. And then Spain and Sweden were in the process of being associated. So We're working our way through Europe with a team of about 20 people, highly experienced, and Jan's on the phone. Again, if there's additional questions, we can go there. And so on the clinical side, as you know, we've updated our M&A and Daybreak strategies slightly, and Linda will dive into that in greater detail. But I'll just say up front, we feel that the adjustments we've made to emanate, we have a flat-out better trial with a higher probability of success. And I'll remind you that we're working in an area where we continue to learn. We continue to learn more about not just us, but us and our partners, the world at large, more about individual variants and allows us to think about classifying them, looking at ones that were in that boost category, but now maybe with a little better understanding you could categorize them as more likely to be toward the pathogenic, likely pathogenic end of the spectrum. And so as we've redesigned it, again, narrowing it down, focusing on those, it does give us that better trial. The numbers, so from a total market opportunity, the numbers have decreased, but I'll remind you here, so no change to the SH2B1, SRC1 numbers. And the heterozygous, POMC, and leptin receptor numbers at about 10,000 puts us at an aggregate opportunity, U.S. only, of 50,000 plus. And the HET, leptin, receptor, and POMC worlds will in fact be a less confusing commercial opportunity. These patients are better and more clearly defined based on their genetics and therefore will be easier to manage through the overall process. So that and that feel really good about where we are with M&A and Daybreak. So with that, I'll turn it over to Jennifer.