Rhythm Pharmaceuticals Q1 2023 Earnings Call Transcript

Quartr
Provided by Quartr
May 2, 2023

There are 12 speakers on the call.

Operator

Good day, and thank you for standing by. Welcome to the Rhythm Pharmaceuticals Q1 2023 Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Dave Connolly, Executive Director of Investor Relations and Corporate Communications.

Operator

Please go

Speaker 1

ahead. Thank you, Benny. I'm Dave Conley here at Rhythm Pharmaceuticals. For those of you

Operator

.rhythmtx.com.

Speaker 1

This morning, we issued a press release that provides our Q1 2020 Three financial results and a business update, which is available on our website. And as listed on Slide 2 is our agenda. Here with me today in Boston are David Meeker, Chair, Chief Executive Officer and President of Irvin Pharmaceuticals Jennifer Chen, Executive Vice President, Head of North America Hunter Schmidt, our Chief Financial Officer and Jan Mazibro, Executive Vice President, Head of International He's on the line joining us from Europe. And I'll remind you that this call contains on Slide 3, I'll remind you that this call contains remarks 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. In addition, any forward looking statements represent our views only as of today.

Speaker 1

It With that, I'll turn the call over to David Baker, who will begin on Slide 5.

Speaker 2

Thank you, Dave, and thank you all for joining this morning as we report another strong quarter. As we will keep reiterating, this company is built on strong, well understood biology, that is the impairment in the MC4 pathway, which governs calorie intake, I. E. The hunger and energy expenditure, a clear unmet medical need in the patients who suffer with these rare diseases and a simple solution. Incybri is a replacement therapy restoring function in that pathway.

Speaker 2

The near term value in Rhythm resides in our ongoing global launch of Incybri A global program working in a challenging space continue to execute. We are well capitalized with funding into 2025 with Incipri revenues now beginning to make a meaningful contribution So looking at Slide 5, these are our 3 strategic pillars. Commercial launches are off to a strong start. Our conviction in the value we are bringing within Sivri, particularly with regard to the less well appreciated and less well understood hyperphagia part of the disease continues to grow. Obesity is not one disease, it is many diseases.

Speaker 2

Our educational efforts helping healthcare providers recognize these diseases and their need for a targeted solution are making a difference. Our personalized approach to supporting patients and their healthcare providers is working. We see each patient for who they are, we meet them where they are and we don't give up. In the U. S, scripts continue to be written by a growing number of physicians and we are making continued progress in expanding payer approvals, particularly in patients covered by Medicaid as Jennifer will Internationally, we have launched Incyvary for DBS in Germany, the largest European market, following the second exemption from the German Federal Joint Committee, which recognized is a therapy for a devastating rare genetic disease and not a lifestyle medication.

Speaker 2

As Jan will describe, the team is moving and we look forward to updating you on the progress. The HO trial has initiated with the first patients treated. We expect to complete enrollment as we had previously guided in Q1 of 2024. We look forward to providing updates on the 12 month data at a medical meeting in Q4. And we are continuing to make progress on our M and A trial enrolling, pediatric trial finishing, the weekly switch study finishing and Daybreak Part 1 data to be presented later this year.

Speaker 2

The shinvento integration has gone extremely well as we work towards candidate selection for the lead indication of congenital hyperinsulin. We will provide further updates on all of these programs in the Q4. On Slide 6, Slide 6 is our biology slide, which we will probably show on every call. And this is to remind you that this is a differentiated pathway, which when impaired requires a targeted solution. These diseases are quite distinct From general obesity, the early onset obesity is severe and the obesity is lifelong.

Speaker 2

The common thread across these diseases is Hyperphagia, that is insatiable pathologic hunger drive that leads to abnormal food seeking behaviors. On Slide 7, You see the 2 foundational opportunities, BBS and HO affect meaningful numbers of patients with a BBS prevalence of 4000 to 5000 And an HO prevalence of 5000 to 10000 in the U. S. With comparable numbers in Europe. The major difference between the BBS and HO opportunity is that the vast Majority of HO patients are diagnosed and actively engaged with the healthcare system, specifically the doctors we are working The additional opportunities represented in the Phase 3 M and A trial offer significant potential upside from there.

Speaker 2

As a reminder, no therapies are The GLP-one question is important. Earlier generations of GLP-one did not show benefit. While While we do not have trial data on the newer GLP-one or combo therapies, anecdotally, as Doctor. Abuzaha described on our first HO call last year, She believes 20% or so of HO patients may have some response to GLP-1s and the magnitude of that response might be on the order of 10% or less. These drugs are different.

Speaker 2

They work through different receptors on different pathways. It makes sense that some patients may have some response to other medications, including GLP-1s Phase 2 cohort is how consistently semilimtide work in those patients who are compliant with a therapeutic dose. The 18 patients had a mean BMI decrease 14.5% at 16 weeks associated with meaningful decreases in their hunger scores. The consistency of those results strongly suggest Before deciding on the need for additional therapy. The obvious and most simple of those interventions is a diet and exercise program, which these patients have universally failed when driving the presence of an MC4 pathway defect, but find greater success once function in that pathway is restored.

Speaker 2

Now moving to Slide 8, we are excited to have our Phase 3 trial underway with the design as shown here on Slide 8. As a reminder, this trial in HO is a double blind randomized controlled trial enrolling 120 patients randomized 2:one treatment and placebo. Patients will be dose escalated over 4 to 8 weeks and then followed for 52 weeks with the primary endpoint of percent BMI reduction as compared to baseline. It is challenging it is a challenging time to be running clinical trials, but the team has done a great job working with our CRO and we expect to have all of our Overall, we have worked with over 100 clinical sites in 15 countries, which in addition to testing our therapy creates awareness, Bill's experience with the therapy and most importantly helps build a community of patients and physicians working to improve the lives of patients living with these MC4 pathway diseases. With that, I will turn the call over to Jennifer.

Speaker 3

Thank you, David. I will be starting on Slide 11 today. We are pleased with the continued demand and uptake we are seeing with our U. S. Launch of emcipri for VBS.

Speaker 3

At launch, We felt good about our starting point and our strategic plan to identify patients, to engage with physicians and educate them on the hyperphagia and severe obesity Three full quarters into launch, we are excited by our progress and our team is thrilled by the success stories we're hearing from patients and their treating physicians. We continue to hear from patients, caregivers and physicians experiencing the benefits of not only weight loss, but also improvements in social activity and engagement, better sleep and more confidence. Since Obsivory was approved for BBS by the FDA On June 16, 2022, and through the end of the Q1 of 2023, We have received more than 300 new prescriptions for VBS patients with more than 100 of them in Q1. The more than 300 new prescriptions since approval comes from more than 175 physicians. Importantly, we have received payer approval for more than 160 of these prescriptions since launch.

Speaker 3

The demand for AMZIVRIA is strong. Physicians are writing prescriptions, patients are experiencing benefit on drugs and payers are increasingly recognizing the value of this therapy. Next slide. Looking at the prescribers of ensifery, endocrinology, both pediatric and adult We remain the top specialty at a combined 44% since launch. Pediatricians remain second, accounting for 20% of prescribers.

Speaker 3

Approximately 27% of all Incybri prescribers since launch are new to Rhythm, meaning that our territory managers had not called on them directly prior to writing a prescription. That share increased in Q1 versus prior quarters as 37% of prescribers who wrote in the Q1 of 2023 were new to Rhythm. This trend continues to give us confidence in our non personal promotion efforts, which supplements our field team by educating a broader physician and patient population. Next slide. For our payer mix for BBS prescription, the majority come from commercial plans and Medicaid and a small purpose or less than 10% come from Medicare.

Speaker 3

We have mentioned in the past that commercial coverage for IMCIVRI is good with payers representing the vast majority of covered lives have a policy in place to cover IMCIVRI. We are also pleased with Medicaid coverage and the progress we are making in securing approval. I have outlined on prior calls that there is variation in covered status As some states cover MCIFRII, some states do not and others decide on a case by case basis through the appeals process. In an effort to provide more granularity on Medicaid coverage, we outline on the next slide Some data based on Medicaid covered lives relative to IMCIVRI coverage. Next slide.

Speaker 3

According to Medicaid, there were approximately 85,000,000 individuals enrolled in Medicaid in all 50 states, plus Puerto Rico and the District of Columbia as of December 2022. Looking at the left hand side of the pie chart, Approximately 75% of Medicaid covered lives are in states with a positive emcivory policy in place or in a state where we have been able to get at least one positive coverage decision in the absence of an IMCIVRI policy. Within this latter category, which represents about half of the 75%, there are some states where we have been able to consistently gain positive coverage position, whereas other states could be mixed with 1 or more approvals along with 1 or more denials. Now moving to the right hand side of the pie chart, The remaining 25% of Medicaid covered size is a mix of states with no policy yet for MCIF coverage and 1, we have not yet had a prescription for Amsivri that would trigger a coverage decision or 2, We have received a prescription and we're still working to secure access. Or finally, 3, where we have received a prescription and have not been successful in gaining access for Infantivary through the appeals process.

Speaker 3

This last category represents less than 10% of covered slides. We remain committed in our payer education and outreach efforts to help them recognize CVS as a distinct disease that requires a targeted therapeutic approach and we continue to work persistently to explore reimbursement opportunities for all of our patients. For example, even when we have denial through the appeals process, we have had success in gaining Medicaid coverage through EPSDT for early and periodic screening, diagnostic and treatment benefits. This program provides comprehensive and preventative health care services for all children under age 21 who are enrolled in Medicaid. So this dynamic is constantly evolving.

Speaker 3

Next slide. The age breakdown of BBS patients For whom we have prescriptions is here. Adults account for approximately 50% of prescriptions received since launch, while prescriptions for adults for children and adolescents continue to account for the other half. And nearly all or 97% of patients with prescription This allows our team to work side by side with patients and their families to help them gain insurance coverage and to support them through our education efforts from initiation and maintenance on therapy. Next slide.

Speaker 3

Based on the information available to us today, We know there are physicians who have prescribed Incybri for 1 or more patients who have additional BBS patients for whom they have yet to prescribe, as well as physicians with DBS patients who may require additional education to prescribe Incyvary. Our territory managers are actively engaging with each physician to increase a sense of urgency to treat the hyperphagia and obesity In parallel, genetic testing, use of ICD-ten codes to narrow our physician targets, As well as digital non personal promotion efforts, all of which we began well ahead of last June's launch, have driven our patient identification We are excited by the progress of these ongoing efforts and the opportunity that remains for ensivary moving forward. With that, let me hand it over to Jan.

Speaker 4

Thank you, Jennifer, and good morning. Slide 18, please. Last week, we announced the launch of IMCYR in Germany for the treatment of obesity and control of hunger associated with DBS With federal reimbursement, as you can see on the slide, the German Federal Joint Committee or GBA rules that Insevary for BBS is eligible for full reimbursement by statutory health insurances. The GBA unanimously voted to exclude Imsilary For the patients with DBS from its lifestyle exemption list, as it did previously for Bialylic, POMC, PCSK1 and Lipar Deficiencies, And exactly on time with regard to our plans. As David said, this is a very important recognition of the severity of BBS And further reinforces the distinction between general obesity and rare MC4R pathway diseases.

Speaker 4

Next slide. Germany holds a unique place in the history of rhythm that is very favorable to us and septalenatide. Our first Patients were treated at the Charite University Hospital in Berlin, where the local experts have the longest experience in the world treating patients with sepalentide, more than 10 years. We are very well positioned in Germany with an experienced team on the ground engaging with healthcare authorities, payers, Our General Manager in Germany comes from Alaylam, has successfully led many orphan drugs and high Launches in Germany and he is leading a team of 6 people dedicated to the launch. Genetic testing is well established in Germany And our own programs are supplementing it.

Speaker 4

Based on our interaction with the centers of excellence, we believe about half of the patients diagnosed with BDS in Germany I've already been genotyped. This is quite important as genetic confirmation is required under our level. We also have strong starting points. German BBS TRIPON guidelines are currently in development and will be published soon. There is a very well organized patient advocacy group dedicated to BBS And also 2 existing academic registries for rare renal diseases and rare ophthalmological diseases.

Speaker 4

Next slide. Rare disease launches are difficult to forecast, especially the 1st 12 to 18 months. In Germany, in particular, we do anticipate a methodical Patient by patient approach. However, we are confident that BBS in Germany represents a significant opportunity for M Series. Our team has already engaged with physicians in 18 major hospitals across the country in an effort to identify patients with BBS and prepare the launch and have set up a significant number of medical education and disease awareness activities.

Speaker 4

We estimate that the prevalence for BBS in Germany is approximately 1200. We believe that there are about 800 patients diagnosed and of those 800, we have identified physicians caring for more than 250 of them Next slide. This slide is a reminder of how important the European market is to

Speaker 2

Sorry? You still there, yes?

Speaker 4

Okay. I'm back. Sorry, I had a Wi Fi issue. So I was saying that the slide is a reminder of how important the European market is to our global commercial strategy. For rare genetic diseases, we know that European countries More than the U.

Speaker 4

S. With single payroll healthcare systems, government funded genetic testing, rare disease organizations, center of excellence and referral networks. For Biomedic, POMC and EPAR deficiencies, we know that there are about 100 patients identified in the EU for the UK. And for BBS, more than 1500 patients are identified across those same countries. For In 9 countries in addition to the U.

Speaker 4

S, we are launched in the UK, Germany, Italy and the Netherlands and we have achieved an ambition sales in France, Austria and Turkey and early access in Argentina. In summary, we are very pleased with the progress we are making in Europe in terms of market access. Next and last slide, please. We are also very pleased with the level of support we are receiving from key experts in MC4 RP3 disease, including barbie beetle syndrome. Europe is home to many of the world's leading experts and Rhythm is fortunate to enjoy a strong and long lasting relationship with many of them.

Speaker 4

In March, Professor Sadaf Farooqi from Cambridge UK, one of the world's leading experts in MC4R pathway diseases and Professor Phil Bils from London, who helped The final with Diagnose BBS, both led Aurizm's sponsor of disease education webinar with Angela Schroeder, who is on this with BBS And who is the BBS Patient Liaison Officer for BBS UK Clinics. On this webinar, the speakers explored hyperphagia, The genetics of rare MC4 pathway diseases with a focus on BGS and how to best care for these patients with a multidisciplinary approach. We are delighted to have more than 125 physicians from 17 countries join the webinar live, which speaks to the high level of interest in rare MC4 disease among European physicians. Thank you. And with that, Hunter.

Speaker 5

Turning to Slide 24, with the launch of Emcivri in And additional global markets coming online this year, Rhythm is growing into a global commercial rare disease company. And as we do, We approach all our operations and investments in our commercial, R and D and operational programs with a financial discipline that governs our decision making and focuses on building long term value for our shareholders. We are grateful for your support. Let me review the highlights of the Q1 P and L. As mentioned, we recorded $11,500,000 in net product revenue during the Q1 versus $1,500,000 during the Q1 last year, which was prior to FDA approval for BBS, Compared to $8,800,000 in net product revenue from Incyprix in the Q4 of 'twenty two, that marks an increase of $2,700,000 or more than 30% quarter over quarter.

Speaker 5

This growth is driven primarily by IMCIVRI sales for BBS in the United States. Cost of sales during the Q1 was $1,400,000 or approximately 12 Net product revenue, which is consistent with Q4 2022. Cost of sales consisted of approximately $600,000 in royalties due to Ipsen Under our original licensing agreement for septalatinib, approximately $200,000 of amortization, previously capitalized sales based milestones as well as Product costs associated with increased sales of commercial product. R and D expenses were $37,900,000 for the Q1 of 2023. This compares to $32,500,000 during the Q1 of last year.

Speaker 5

Compared to $23,500,000 in Q4 of 2022, This quarter quarter increase of $14,400,000 is driven by several factors. First, there were $5,400,000 in costs and fees associated with the SIBENTO acquisition. The remainder of the quarter over quarter increase was due to a $6,000,000 net increase in clinical trial expenses. These are mainly startup costs associated with the HO Phase 3 trial and a substantial increase in activity associated with the M and A Phase 3 study. Also in Q4, 2022, Rhythm received a $2,500,000 credit during the closeout of our GoID study during that quarter, which reduced R and D expenses.

Speaker 5

Overall, clinical trial costs are expected to be higher on a period basis during study startup and after all trial sites have opened. Lastly, in Q1, there was a $2,100,000 increase in clinical supply costs for these studies and for other programs. SG and A expenses were $24,600,000 for the Q1 of 2023 compared to $21,400,000 in the same quarter last year. This increase was largely due to the impact of $2,600,000 in higher headcount costs, including stock compensation. Quarter over quarter, SG and A declined Nearly $1,700,000 or nearly 7 percent from $26,300,000 in the Q4 of 'twenty two.

Speaker 5

The decrease in SG and A versus Q4 is due primarily to lower marketing expenses in the U. S. For the Q1, common shares outstanding were 56 We closed the quarter of 2023 well capitalized at $295,000,000 in cash on hand sufficient to fund all planned activities into 2025. This cash guidance includes the impact of projected milestones associated with the Shinvento acquisition. We'll touch on a few other aspects of the quarter.

Speaker 5

Of the Q1 net product revenue of $11,500,000 83 percent of this revenue was generated from U. S. Sales of INSEBRY as compared to 85% in the Q4 As mentioned, dollars 5,400,000 of operating expenses represented consideration associated with the Simbento acquisition, which was included in this quarter. We accounted for this transaction as an asset acquisition. Q1 operating expenses included Total stock based compensation of $6,400,000 as compared to $5,300,000 in the Q4 of 2022.

Speaker 5

And our non GAAP operating expense guidance for 2023, which we disclosed last quarter, remains unchanged at $200,000,000 to 220,000,000 This guidance excludes the non cash impact of stock based compensation. And with that, I'll turn the call over to David.

Speaker 2

Thank you, Hunter. So in summary, we're excited about the progress we have made and we look forward to multiple data readouts In addition to continuing to update you on our global commercial launch with BBS in the upcoming quarters. And so with that, we'll open it up for questions.

Operator

Thank you. Our first question comes from the line of Phil Nadeau from TD Cowen. Please proceed with your question.

Speaker 6

Good morning and congrats on the progress and thanks for taking our question. A couple of commercial questions. First, in terms of reimbursement for BBS in the U. S, Are there any new trends in terms of either faster or easier reimbursement as the launch continues? Or is it that the patients are spread among so many insurance plans that it's still each plan evaluating their 1st patient?

Speaker 2

Yes. So, Jennifer, yes?

Speaker 3

Thanks for the question. So, We're continuing to build the relationships, but as you outlined, there are just so many different payers. So the scripts come in is dependent on if we've interacted with the payer before or not. I would say that in terms of scripts that we've received Through a payer where we've been able to gain reimbursement that process also because we know what the process looks like for that particular payer is quicker. We are also seeing trends through our education efforts that we have a higher percentage of Payer approval at the prior authorization stage, which is also a good sign.

Speaker 3

So once again, it's really 1 on 1 with these payers as they come in that our teams interact with and overall the average time in In terms of gaining reimbursement still remains within that 1 to 3 months period of time.

Speaker 6

In the slides, there was a note that one of the opportunities for expansion in BBS in the U. S. Are among those physicians We're treating BBS patients, but are not yet ready to prescribe, emcevary. What are the objections those physicians have? What do they need to do or what do you need to do to convince them that they should be prescribing Insevary?

Speaker 3

Yes. So I would say that The gating factors could be either on the patient level or the physician level. For both of them, some of these patients may not be of age and within our label. For patients specifically, the physician may have written a script, but the patient could be lost to follow-up. There may be concerns just in terms of injections or a parent just wants You get a bit more education in terms of the safety profile before putting their child on a chronic lifelong therapy, and many other reasons.

Speaker 3

From an HCP perspective, sort of similarly, they may require some additional education to truly appreciate and understand the difference In terms of the hyperphagia and the early onset obesity that these patients have, In rare diseases, it's not uncommon for a physician to just have one patient, so there may be additional education needs. So there's a lot of different reasons, but I will say that our teams are really on ground interfacing with the customers to try to

Speaker 2

And Phil, just to reinforce what Jennifer said, the hyperphasic component of this, in terms of the opportunity for Creating that sense of urgency, not surprisingly, I mean healthcare providers themselves, I think just don't understand many of them don't understand The full impact of this on both the patient and the family. And so there's still a little bit of we understand you're hungry, but they don't understand the pathology And severity of that hyperphagia component. So I think as Jennifer said, we are making good progress there, but that is a clear opportunity as we go forward.

Speaker 6

Great. And then one last question from us. In terms of German reimbursement, can you remind us where you are in negotiating the final price

Speaker 4

So we are Still in the midst of the PONCILIPA pricing negotiation, and so far the dialogue has been very positive To date, the medical benefit assessment has been positive as well. BBS price negotiation will start in a few months and the process takes Approximately 6 to 9 months. So more to come for the German price for VDS.

Speaker 6

Perfect. Thanks again for taking our questions.

Speaker 2

Thank you.

Operator

All right. One moment please for the next question. All right. Our next question comes from the line of Derek Archila from Wells Fargo. Please go ahead.

Speaker 7

Hey, good morning, everyone, and congrats on the progress. Thanks for taking the questions here. Just a couple from us. So I know you said that the BBS launch and these types of rare disease launches can be lumpy, but I guess can you provide some more color on what's Specifically deriving the acceleration that we saw in new patient adds from 4Q to 1Q here. And is that something that's Translate going forward into the following quarters.

Speaker 7

And then second question is, do we have enough data yet to really understand The discontinuation rate for IMCivri and bartobetal patients in real clinical practice and just understanding how that's trending right now. And I might have one follow-up. Thanks.

Speaker 2

Thanks, Jared. Jennifer?

Speaker 3

Sure. I would say that overall, just in terms of The level of demand and interest for this specific therapy for BBS has been really overwhelming and great to See as well as clearly there was a need in this patient population as we're hearing the benefit that the patients are actually receiving. Once again, a motivating factor for teams overall. There were existing opportunities that still remain in terms of the patients that were identified through all of our cross functional team efforts and still remaining opportunity just in terms of pull through to scripts. And in the meantime, I feel that in any disease, but particularly in this one, we have identified specific Ways of going about to really have targeted approaches of Patient find and identification, 1, to get to physicians or patients that have already been diagnosed and lost in the system as well as Trying to expedite the path to get patients who are symptomatic to a diagnosis.

Speaker 3

So all of these efforts are ongoing, which Continue to fuel the increase just in terms of number of scripts that we have received. When we say that rare diseases are I would say that in terms of some of these efforts, you can't predict quarter by quarter that's going to be the exact same. So I wouldn't necessarily linearize or just make the same assumptions quarter over quarter at this point of time, but I would just say that there still remains quite an opportunity just in terms of growth within this patient population for IMZIVRI. To the next question, I think around

Speaker 2

May I just add one quick follow on to that, Derik. So I think what we can say at this And this is what Jennifer said is that we are well beyond whatever pent up demand existed in the system and the like and that you're seeing now a quarter on quarter As we would expect, stability and sort of ongoing strength in the overall opportunity, if you will. And again, I'll reference back to the number of physicians who we had not been in contact with who and that pool is growing. That's again what we would see and speaks to overall health of I think a rare disease opportunity. But again, don't trend as Jennifer said, I think That's not.

Speaker 2

It could be less or more in any given quarter, but our confidence that this thing is real and working is very high.

Speaker 3

And there was another question just regarding discontinuations. We continue to be quite pleased. As I outlined in the past, there was a lot of effort, CrossFlex Health team effort really focused to make sure that the patients were able to go and tolerate the titration phase. So through that Process and education, we still remain very, very happy just in terms of the level of maintenance of patients through that phase with The number of discounts relating to nausea or vomiting being extremely low. We do have some Continuation, for various different reasons, including a very low number relating to hyperpigmentation, But there's other reasons that a patient may continue that are also opportunities for follow-up.

Speaker 3

And I think one of the pieces that continues to be one factor that increases the compliance of this therapy It's a hyperphagia because people feel the impact, they also feel the impact of stopping therapy. And Once again, we also hear when patients stop therapy that the hyperphagics comes back and there may be interest to come back on to therapy. So very happy overall just in terms of the low discount rate.

Speaker 7

Got it. And maybe just one follow-up here on the prelim data that you're going to put I guess, will you be kind of doing an in-depth kind of presentation on framing those opportunities? And I don't know if you've kind of Got it. So what those opportunities look like from a commercial perspective? Are they more like a Pompey or more like a Barta Beetle or something different?

Speaker 7

Thanks.

Speaker 2

Thanks, Derek. We haven't guided and not prepared to guide today. I think what I have said and Reiterated, I expect to report out on 5 ish plus or minus genes where we have enough data. The DAYBREAK trial is designed as an exploratory trial. It's It's done exactly what we wanted it to do and allows us to sort a relatively large number of genes that we Knew had some link to the pathway with the goal of trying to understand which were the ones that had the strongest link versus the others.

Speaker 2

I will say that some of those which we Discontinued earlier on, we're extremely rare and we just weren't able to enroll. So back to your question about the POMC smaller opportunity kind of thing, but Others have a much higher frequency and more in the order of SHTV-one and SRC-one that we're pursuing on our M and A trial again. So More to come on that, but the expectation we should set is it will be around 5 plus or minus genes where we have enough meaningful data to report out.

Speaker 5

Got it. Thanks so much. Okay.

Speaker 2

Next question.

Operator

Thank you. Our next question comes from the line of Corrine Jenkins from Goldman Sachs. Your line is now open.

Speaker 8

Yes. Hi, good morning, everyone. Maybe a couple from us. So of the roughly 140 patients that don't currently have Reimburse product and including the 40 in particular that had a prescription as of year end 2022, what portion do you expect to ultimately get on reimbursed drug Versus what portion do you think may just remain on free drug from here?

Speaker 3

So within the 140 that you mentioned, That includes patients that are still within the pending category that we're still working Through 2 reimbursements, as well as patients that we have put on to our free drug program. I will say that in terms of our free drug program, we have outlined in the past that For reimbursement, Medicare patients have not been A patient population that we've been able to gain reimbursement for at this particular time, so that's You know approximately less than 10% of scripts to date. The commercial coverage has always been very strong. The Caveat here is as with other rare diseases, there are very small commercial self insured plans where cost of a therapy like ensivory can be challenging. So we do have patients that are on small Self insured plans as well that are on path as well as, as I went through today, some Medicaid patients as well.

Speaker 3

The caveat that I will say here is even though they're on our free drug program, I think the word that you're going to keep on hearing is that We have a persistent just in terms of still working on those patient populations as well, whether it's Further education with the payer, or just ongoing help for that patient itself. We have been successful in moving some of these patients off into commercially insured path. So It's constantly evolving from that perspective, and we are really just still starting and We continue to engage with all of our customers on that point.

Speaker 2

So thanks, Jeff. And maybe just to, Prem, just to add to that. At this point, I think the number I would think about and Jennifer said we're still learning and it's early, etcetera, but about 20% plus or minus of the Total scripts are patients who are likely to be on PAP and again reminding you that of that 20% plus or minus you have Approximately 10%, a little less than 10% that are the Medicare and they go straight there. But most encouragingly, and this has been true in our prior experiences as well, that Patients who go on PAP don't necessarily stay on PAP and we continue to find ways to move them over and patients themselves actually don't want to be on PAP. They'd much rather be on a more stable, if you will, situation where they are being paid through the system.

Speaker 8

Thanks. That's helpful. And then maybe on the clinical side, you highlighted you expect to complete enrollment for the study in HL in 1Q 2024. Are there any factors that could shift that timeline either to be more rapid or delayed for any reason?

Speaker 2

Yes, many. Probably on both sides. I mean, I think what we've shared and there's 2 parts to this. One is just the So administrative issues getting sites up and going. We've identified the sites that we need.

Speaker 2

You continue to look for other maybe Outstanding sites to have is back up if something happens at a site, but in general, we have the sites we need. So it's just a matter of working through contracting with these sites and the IRB approvals And the like, again, as we said, we're in a queue, not just with other BC trials, we're in a queue with any trial going on at that site. So that's been slower than we would have liked. Again, our goal is to have all these sites open and operational enrolling by the end of Q3. So that's one and that could vary.

Speaker 2

I think we have quite a comfortable guidance there, but No, you don't know what you don't know. And then the second is on terms of patient interest. Patient interest is high. So we have our investigator meetings coming In May, first one, the U. S, some follow-up a week or 2 later in Berlin for the European sites.

Speaker 2

And Again, what we've heard and what I expect to see there is a high level of interest and also I would think some competitive enrollment. Again, We know there's patients out there eager to get in and these sites are going to know that if all 25 sites are enrolling, there aren't that many patients per site That will be have eligible slots and some of the sites are clearly aiming for much higher than even distribution. So we'll see how it goes. But I think I'm Pretty confident that one's rolling and running.

Speaker 8

Great. Thank you.

Operator

Thank you. One moment please. All right. Our next question comes from the line of Dae Gon from Stifel. Please go ahead.

Speaker 9

Hey, good morning guys. Thanks for taking our questions and congrats on the progress as well. Just reverting back to Derek's question, just Hitting it head on, just apologies if I've missed it. But the discontinuation rates, David, you were at other Conference recently talking about mid single digits. I just wanted to confirm that number is still true.

Speaker 9

And then in terms of the two questions I had, 1, when you think about the patient dispositions across the three dose levels that are in the label, can you comment on any kind of I guess Jennifer kind of went into Education to get patients into sort of the more tolerant dose, but any color you can provide across the 3 doses? What kind of disposition we should be Going forward and what work is being done to keep patients off of the 1 milligram and more skewed towards the 3 milligram arm? And then secondly on the strategies for the reimbursement. David, you spoke previously a number of times, Gaochai, as sort of the analog we should be thinking about for BBS going forward. But just harkening back to your rare disease experience, what kind of reimbursement rate Should we be expecting eventually?

Speaker 9

I mean is this something that can near into 80%, 90% or hovering in the 70%? And what work needs to be done for Emcevery to get there? Thank you so much.

Speaker 2

Thanks, Pecan. So first on the discontinued patient rates. We previously, as you noted, said mid, I would characterize we're drifting up a little bit there, not Surprisingly, as we get more patients on for longer periods of time, so it characterizes with the high level single digits. But As Jennifer said, and this is what's most encouraging overall is, 1, I think we've done much, much better than we did in the clinical trials for the reasons she outlined, the close contact with the patient through our rhythm 2nd is recent patients are discontinuing about half are related to side effects of the drug. Again, opportunity to just continue to educate and set And the other are personal issues specific to that individual patient, some of which may resolve over time And, know the patients being willing to come back on therapy and we have several of those examples, so including others that we're working.

Speaker 2

With regard to dosing, the whole strategy behind dosing here is we start low and we're going a little slower than we did in the trial. Not Surprisingly, if you go a bit more gradually, this early tolerability is better, number 1. Number 2, The vast majority of patients are getting to 3 mgs. I would say, the balance of the patient population is early and So working their way there. So my expectation is that the truly vast majority of patients will be at or close to 3 mgs.

Speaker 2

Younger patients, very young patients may in fact achieve their desired level of benefit at a lower dose level. But Go back to our Phase 3 trial where we had a number about half the patients were under age of 18 and the other half were over and then on BBS. Again, that was those patients were literally virtually all on 3 mgs. So if you have tolerability issues, you go down, Go a little slower, but you continue to dose yourself back up. You don't just go down and stay as a rule.

Speaker 2

Your third question was just on the Negotiate analog, I mean, I just referenced that to remind people that these opportunities in rare diseases, they may ramp somewhat more gradually. You don't have a hockey stick as a rule, But you tend to have them for a long time and negotiate 30 plus years from its original approval is still $1,000,000,000 plus opportunity. So again, We'll see where it goes. But VBS has many of the elements of what you want to see in a rare disease opportunity in terms of the overall size of the opportunity, The ability to diagnose that it's syndromic in this case and the strength of the community that's emerging, the patient community and physician community. So So we'll see where it goes.

Speaker 2

That was the analogy there. And with regard to reimbursement, I don't expect to see in the U. S. Specifically, any decrease in price, including when we expand into HO, Obviously, you don't necessarily take the same price increases that you might take in other parts of our industry here. So there is an It's a decrease if you don't take a price increase in terms of inflationary adjustments.

Speaker 2

But in terms of actual price, Yes, we don't expect any of these are rare diseases and we think we're very fairly priced. Great. Thanks for taking our questions. Yes. Thank you.

Operator

Thank you. One moment please as we compile the Q and A roster. Our next question comes from the line of Michael Higgins from Ladenburg Thalmann. Your line is now open.

Speaker 2

Thanks, operator, and congrats all these guys on the continued progress. Just want to follow-up on the H. O. Trial, the pivotal that's enrolling. If you can give us some feedback on how the pace of enrollment, the pace of screening failure rates and the pace of site enrollment are coming in versus expectations?

Speaker 2

Thanks. Yes. Thanks, Michael. Early, we just started. We'll figure out we will give you metrics on how that trial is evolving.

Speaker 2

Today, the communication is we're up and running, but again, It is much too early to have any sense there. I will say, again, probably my opinion As opposed to sort of observed on hard data here, but there are enough patients and enough interest out there that the prescreening of patients Should be pretty good. So patients who actually come to the site to be formally screened, I'm not expecting a high screen failure rate there To be determined, but again, I think this is one of those situations where you're not desperate to enroll just anybody and therefore you can end up No, I'm getting higher screen failure rates, but we'll see. But I can't give you more information today. Okay.

Speaker 2

I appreciate that. And also, you've noted that you're looking for 3 data readouts in the second half. I'm curious if you can provide feedback as to the order of those events And if they come in before or after the Obesity Society meeting in mid October? Thanks. Yes, I think and you're correct in highlighting that they are We've guided to Q4.

Speaker 2

I think that's all I can do today, But we will get that information. Okay. Appreciate it. Thanks guys. Thank you, Michael.

Speaker 2

Thanks, Martin.

Operator

Thank you. One moment, please. All right. Our next question comes from the line of Joseph Springer from Needham and Company LLC. Your line is now open.

Speaker 10

Hi, good morning. Thanks for taking our questions. Just wanted to get your updated thoughts on the European VVS launch and how it would could compare to the U. S. Launch to date.

Speaker 10

It looks like European VVS patients, More of them are identified and the community is more organized, but you have the dynamics of the staggered reimbursement process. So How do you anticipate your PN launch playing out and perhaps maybe using Germany as an example, You have the 250 patients ID'd. Could we expect a similar rate of TRx add relative to what has been seen in the U. S. To date.

Speaker 2

Yes. I think, Joey, I'll turn it over to Jan, One second, just to highlight, you are correct that the European is better experienced or situation is better organized and more identified, but as Jan highlighted may have a different pace. So Jan?

Speaker 4

Yes. Maybe I will start with the German launch versus U. S. And I will end on the overall European situation. So first, Germany and the U.

Speaker 4

S, So you're right. There are similarities between the two countries and the most important one is the decentralized health care and decentralization of the care. And as I've said in my presentation, we already know more than almost 20 large hospitals where they are diagnosed with BBS patients And where patients will be treated. There is a main difference, which is really the pace of starting the treatment. The German physicians are well known to be more conservative than the average, And we know that it will be patient by patient decisions like for any other rare disease.

Speaker 4

So that's Germany versus the U. S. And then back to the overall question of Europe. It's a bit early to speak in terms of trajectories For Insulin Europe, 1st, because Germany is our first important launch and second, most The important European country will launch at the end of the year, Italy, Netherlands, Spain, etcetera, and at the end of 2024 for the UK. So it's still a bit early.

Speaker 2

Thanks, John. Joey, is that covered?

Speaker 10

Great. Thank you for taking our questions.

Operator

Thank you. All right. Our next question comes from the line of All right. Our next question comes from the line of Jeff Hung from Morgan Stanley. Your line is now open.

Speaker 11

For the low number of patients discontinuing due to hyperpigmentation, do you have a sense from those patients how their hyperphagia was? Do they happen to have lower hyperphagia than other BBS patients so the hyper pigmentation overrides that? And then I have a follow-up.

Speaker 2

Sarah, Jennifer, just to highlight again, the number of patients stopping because of hyperpigmentation is extremely low. So one that's been reassuring, but I don't know on those specific cases.

Speaker 3

I don't think it's necessarily correlated with the hyperphased in terms of The reasons for the discount, I think that it's patient by patient just in terms of How problematic the hyper pigmentation is for that particular patient. And once again, I think as the patients discount and they feel the resurgence Of the hyperphagia itself, they can also be at a decision point once again just in terms of really deciding whether To discontinue or to reinitiate therapies. Our teams are there regardless to support them as they Go on their path forward.

Speaker 11

Okay, thanks. And then I know it's a bit early, but with the recommended weight loss monitoring after 1 year of treatment, Do you have a rough sense for the proportion of patients that started on commercial drug fairly early in the launch that have already seen at least 5% loss in body weight or BMI? Just trying to get a gauge the potential impact of discontinuations based on this recommendation. Thanks.

Speaker 3

Yes. So as you outlined, I would say that for the most part, just in terms of the 3 off period, The payers are following our guidance or label, and so it's a bit early just in terms of really reaching that With that said, we feel very good just in terms of the positive Feedback and the compliance and persistence on therapy to date, which speaks to the benefit. And I think that in general, it will be interesting just in terms of what Clinical benefit is outlined. I think for the most part, like, peers just want to be reassured that these patients are actually receiving Clinical benefit while being on therapy and that's something that once again our patients continue to Monitor baseline themselves versus on therapy, so that can also be translated to the physicians who

Speaker 2

And just to remind you, Jeff, the label, as Jennifer referenced, is it's 12 months is for BBS is when the label suggests you should reevaluate. Payers, of course, can choose to do so earlier, but As Jennifer said, people tend to be sticking to the label and we're less than a year out for March. Great. Thank you.

Operator

All right, thank you. We do not have any other questions. So I would now like to Turn the conference back to David Meeker for closing remarks.

Speaker 2

Great. Well, thank you everyone again for tuning in this morning and we very much look

Operator

Thank you. So this concludes today's conference call. Thank you for participating. You may now disconnect.

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Rhythm Pharmaceuticals Q1 2023
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