Rhythm Pharmaceuticals, Inc.

Good day and thank you for standing by. Welcome to the Rhythm Pharmaceuticals third quarter financial results conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question, you will need to press star one on your telephone. Please be advised, today's conference is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to your speaker today, David Connolly with Rhythm Pharmaceuticals. Please go ahead.

Thank you, and good morning. I'm David Connolly, Head of IR and Corporate Communications here at Rhythm Pharmaceuticals. For those of you participating via 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 a third quarter 2021 Financial Results and Business Update, which is available on our website. As listed on slide two, with me today here in Boston for the conference call are David Meekers, Chair and President and Chief Executive Officer of Rhythm, Linda Shapiro, our Chief Medical Officer, Jennifer Chen, Executive Vice President, Head of North America, and Hunter Smith, our Chief Financial Officer. With 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 factors, including those discussed in our most recent annual 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 date. 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. We're very pleased to report another strong quarter of execution for Rhythm, and I'll remind you that we're building Rhythm piece by piece, gene by gene, and are, like I said, very pleased with the progress to date. The first slide is to highlight four areas of high focus for us. One is, of course, the commercial experience with NCIVRI. Second is looking forward to the anticipated Varded Beetle and Ahlstrom's launch in 2022. Third is to build out our global presence, working with healthcare systems, first and foremost in Europe, but we will increasingly look beyond there. And fourth, to continue to advance a robust clinical development program, which has the ability to meaningfully increase the addressable patient population. Now, quarter three is a quarter with multiple highlights, again, with continued execution. First, the U.S. Incivary launch is progressing and meeting expectations. As we said, we expected to have tens of patients on treatment, and that view of the world has continued to hold. We were pleased with the net sales, clearing $1 million for the quarter. But more importantly, we're really happy about the learnings that we're beginning to accumulate from this initial commercial experience, which will lay the foundation for our subsequent Bardic Beetle launch. And in the international markets, Europe specifically, reimbursement dossiers have been filed in major markets and some of the smaller markets as well. And we're in advanced discussions with the three major markets, Germany, France, and the U.K., Second, as we advance toward BVS launch in mid-2022, as you know, the regulatory submissions have been filed in both the US and Europe. In the US, we've fully hired a highly experienced field team, and Jennifer will provide you with more color around that. So a year ahead of anticipated launch, we feel like we are in a good place, so I'm looking forward to that event. Third, we had a very strong presence at medical meetings with 22 presentations at three major medical conferences. Positive data was presented from a variety of different clinical data sets with world-renowned KOLs presenting several annotated data. In a noteworthy category, we have our first data on health-related quality of life and additional data on hunger and weight scores across a number of different genetic diseases. Linda will provide a little more color on that in her presentation. And finally, the clinical development programs remain on track with M&A-favorite, pediatric, and emergency trials advancing to first patient in, and I will provide a little more color on our testing program in the next slide or two. Before we get to that, on the next slide, slide number seven, I just want to remind you there's an efficiency to rare disease community building. which we are taking advantage of. And in the rare disease setting, you often have a limited number of centers of excellence, a limited number of KOLs, and they serve multiple purposes. And so you can focus your efforts very much around these sites. Not only do they run the trials, serve as key opinion leaders, they're also the same physicians, healthcare providers, who diagnose and actually treat these patients. What we're doing is pursuing a hub-and-spoke model where that center of excellence, the clinical trial site, is at the center, and then we will work with a surrounding set of treaters, interested parties who are potential referral sites, referring into that site, and over time some will become their own center of excellence depending on the level of interest. Moving to slide eight, here's a brief update on our testing program. We've had several generations of our genetic screening panel. In May, we went from a 40-gene panel that we reported out on to reporting out on an 80-gene panel. And that panel includes all the genes of interest to rhythm in terms of genes we believe are closely linked to the MC4 pathway. but it also includes the number of genes that would be of interest to the obesity treater at large, meaning other genes that might help inform what is the underlying driver of that patient's obesity. So what this pie graph shows is on the order of 55% of patients who present with a history of early onset obesity and hunger are likely to test positive for one of the genes of interest, meaning a gene that would qualify that patient for entry into the Emanate trial or the Daybreak trial, which is an additional 31 genes we've yet to explore but have some reason to believe they may be related to the pathway, or be potentially eligible for commercial therapy with incivory. Now, of note, in that 55%, our 1.96, almost 2% of the population who are biallelic for Bardot-Beetle or the ALMS1 gene, which is the gene that is responsible for Ahlstrom's syndrome. So that percentage is a noteworthy percentage. There's much more to be learned. We don't know that all of those patients, that their genetic defect is driving their underlying obesity or that they will even go on to develop Bardot-Beetle, but it opens up a whole other avenue for for better understanding this disease, and rhythm's gonna play a leading role in driving that exploration. With that, we'll move on to slide nine. Slide nine is just to remind you again that we do have a broad pipeline, and it underscores the fact that I think we have a legitimate pipeline in a product, and Linda will provide a little more color on our progress across this portfolio in her section. And with that, I'll turn it over to Linda.