Rhythm Pharmaceuticals, Inc.

Good day and thank you for standing by. Welcome to the Redland Pharmaceuticals third quarter 2023 earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, it will be a question and answer session. To ask a question during the session, you will need to press star 101 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 101 again. Please be advised, today's conference is being recorded. I would now like to hand the conference over to your speaker today, David Connolly, Investor Relations and Corporate Communications. Please go ahead.

Thank you, Victor. I'm Dave Connolly here at Rhythm Pharmaceuticals. For those of you participating on the conference call, our slides can be accessed and controlled by going to the Investors section on the Investors page of our website at ir.rhythmtx.com. This morning, we issued our press release that provides our third quarter 2023 financial results and a business update, which is available on our website. As listed on slide two, here with me today in Boston are David Meeker, Chair, Chief Executive Officer and President of Earthen Pharmaceuticals, Jennifer Chen, Executive Vice President, Head of North America, Hunter Smith, our Chief Financial Officer, and Jan Mazerow, Executive Vice President, Head of International, is joining us on the line from Europe. On slide three, I'll remind you that this call contains remarks concerning future expectations, plans, and prospects, which constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our most recent annual or quarterly reports on file with the SEC. In addition, any forward-looking statements represents 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 Meeker, who will begin on slide five.

Thank you, Dave, and good morning, everyone. Thank you for joining the call. So we're pleased to report out another very strong quarter with continued execution across all parts of our business. The two near-term drivers of Rhythm Value building the BBS commercial opportunity globally and enrollment into our Phase 3 HO trial remain on track. I'm incredibly impressed by the performance of our North American and international organizations. BBS and the monogenic forms of MC4R pathway diseases we are approved to treat are, as we have discussed multiple times, highly meaningful rare disease opportunities. But they do fall in the ultra-rare category, with all of the challenges these patients face getting to a diagnosis and then accessing the appropriate therapies. The sheer volume of noise around the management of obesity as a disease and the use of GLP-1 specifically has both aided the cause, healthcare providers are looking more closely at patients who present with obesity and doing the appropriate workups, and hindered the cause, where the availability of powerful therapies such as GLP-1 medications has led many to believe that all obesity is the same and GLP-1 represents a universal solution. As we know, that is not correct. Obesity is not one disease, but many diseases, and as with most forms of medical therapy, the more targeted and specific the solution, the better. The medical community continues to learn more about the factors which control hunger, the role of the different pathways, and the effect of different drugs play in modulating those pathways. Our story remains relatively simple. We're replacing or supplementing a hormonal signal, which is deficient. So if you're managing a patient, why wouldn't you start there? Revenues in the quarter came in at $22.5 million, showing exactly the steady growth we had hoped to see. We're pleased with the script volume in the U.S., where the team is doing a great job educating on the MC4 pathway and the value of precision therapy. And we're incredibly excited to cross the 100-patient threshold in Europe, where in addition to the usual challenges facing rare disease communities, market access across the different healthcare systems can be particularly challenging. We have experienced teams in all geographies who are remarkably skilled at navigating these challenges. So although it will never be easy, patient by patient, we find solutions. As you know, we do not provide financial guidance, but for those of you building models, my experience working in rare diseases with many similarities to our current world has taught me it never gets easier. The revenue trajectory does not inflect, but these opportunities continue to grow over time. HO Phase III enrollment continues on track with two-thirds of the patients screened. That's our proxy for enrollment as almost none of these patients screened fail. The majority of patients needed to complete enrollment have their screening visits already scheduled, and we still have sites who are just opening or scheduled to open and who are eager to get their patients enrolled. We did get early access approval for HO in the third quarter in France based on the 18 patient Phase II data alone. This is an incredible recognition of both the unmet medical need in this population and the potential significant impact treatment may have. The first patient, as Jan will outline, should be treated before year end. Finally, we look forward to providing additional updates at our R&D day, particularly with regard to our next generation weekly formulation, the Daybreak Trial, and our pediatric results. So moving to slide six, we recently had a separate analyst call following a very successful TOSS meeting in Dallas with multiple presentations, which are outlined here. What's particularly gratifying about these meetings, it's the opportunity to meet with the community in person and feel, literally, the growing interest and learning more about the different forms of obesity, including MC4R pathway diseases. On slide seven, I want to take you through all of the 12-month HO data that we reviewed on the last call. Fourteen patients entered the long-term extension, and the results for all 14 are shown in this slide. We saw a robust mean 25% BMI decrease across the 12 patients who had 12 months of data. And I remind you that this is a blend, this trial is a blend of ages with 11 of 12 of the patients, pediatric patients. And the pediatric patients are growing where you would expect the BMI to actually increase with that growth. The two panels on the right are the two patients who are off treatment for some period of time. The short message here is if you take the therapy, most patients respond. And when you stop treatment, i.e., you stop this hormonal replacement therapy, your BMI weight increases. Finally, there's growing interest in the quality of the weight loss, meaning losing fat mass is good, but losing large amounts of lean mass is not good. Our early results suggest we do pretty well in that category, as shown on the DEXA scan results, which we presented on the poster. And one other graphic on the poster shows the shift in obesity class experience by the patients, with three of the pediatric patients actually getting back into the normal BMI range for their age. Slide 8, this is just to remind you of the trial design where we are targeting 120 patients, and as noted, two-thirds of the patients have been screened, and the majority of the balance of patients needed to complete the trial have already scheduled their screening visits. And importantly, and I think this is, you know, it's kind of still early here, but quite noteworthy, of the patients treated, we've had almost no trial discontinuations. So finally on slide nine, this slide is to remind you that we are working on meaningful opportunities. You could build a very profitable company around BBS and the POMC and LEPR monogenic opportunities we are approved for today. However, we are investing significantly in R&D because those opportunities that we are pursuing are even greater. And as you know, for example, the HO opportunity itself represents a large, well-identified patient population with no approved therapy. And with that, I'll turn the call over to Jennifer.