Rhythm Pharmaceuticals Q3 2026 Earnings Call Transcript
Rhythm Pharmaceuticals (NASDAQ:RYTM) reported third-quarter financial results on Tuesday. The transcript from the company’s third-quarter earnings call has been provided below.
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Summary
Rhythm Pharmaceuticals reported strong Q3 2026 revenue of $92 million, driven by a successful U.S. launch of Imcivree for acquired hypothalamic obesity (HO), marked by robust patient demand and a growing prescriber base.
The company announced the initiation of its phase 3 bivameligon HO study, targeting a 52-week trial with BMI as the primary endpoint, and plans to start screening patients by year-end.
Rhythm Pharmaceuticals amended its bivameligon licensing agreement with LG Chem, reducing royalty obligations and aligning interests. The agreement involves payments totaling $350 million over four years.
The launch in Japan for acquired HO commenced following approval by the Ministry of Health, Labor and Welfare, with expectations that Japan will become a significant market.
Financials were bolstered by a new $750 million term loan facility, enhancing liquidity to support bivameligon development and global commercial execution.
The company expressed confidence in its strategic focus on genetic indications, HO, and Prader Willi syndrome, with upcoming phase 3 studies planned for 2027.
Management noted positive payer engagement and reimbursement trends, with 75% of approvals coming by the prior authorization stage and expectations for further policy adoptions.
Full Transcript
OPERATOR
Ladies and gentlemen, thank you for standing by. Welcome to Rhythm Pharmaceuticals third quarter 2026 earnings 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 during the session, you would need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised and to withdraw your question, please press star 11 again.
We ask that you please limit to one question. Please be advised that today’s conference is being recorded. I would now like to turn the call over to Dave Connelly, Head of Investor Relations. Please go ahead.
David Connolly (Vice President, IR and Corp Comms)
Thank you, Michelle. 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 of our website, ir.rhythmtx.com. Last night we issued a press release announcing the amendment to our bivameligon licensing agreement. And this morning we issued our press release that provides our Q3 2026 financial results and a business update. Both press releases are available on our website.
Our agenda is listed on slide 2. On the call today are David Meeker, our Chairman, Chief Executive Officer and President; Jennifer Lee, Executive Vice President, Head of North America; Hunter Smith, Chief Financial Officer; and Jan Mazabro, Executive Vice President, Head of International, is on the line joining us from Europe on slide 3. 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 reports on file with the SEC. In addition, any forward-looking statements represent our views 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.
David Meeker (Chair, President and CEO)
Thank you, Dave. Good morning and thank you for joining today. Rhythm continues to execute on multiple fronts and we are excited to update you on our progress. So let’s start on slide 5. We are very pleased to announce a strong third quarter driven by an excellent start to the US launch. Start forms are growing as expected with encouraging market access translating to strong revenue performance of $92 million for the quarter. Jennifer will provide more details.
We are excited about launching in Japan, with the first commercial patients already on treatment. We have every expectation that Japan will become our second largest market. Jan will provide more color there. I will say more about our clinical development plans and the recently announced amendment to our LG Chem deal over the next couple slides. Slide 6 is a reminder of our three major strategic pillars. The first pillar is the genetic indications where we have multiple approvals and we will be looking to expand the list of genes we are investigating, more to come on that in 2027. Second is HO, where we have a growing conviction in the magnitude of this opportunity globally as you will hear more about today. Third is Prader Willi syndrome, a devastating genetic disease with only one approved therapy in the US and a desperate need for more treatment options.
Let me spend a minute on why PWS is so strategically important to Rhythm and why we believe we can make a meaningful difference in this disease. I often draw the analogy to where the cystic fibrosis community was at the time the first disease-modifying treatments were being approved. Like CF, PWS patients are almost universally diagnosed at birth. The newborn is hypotonic, floppy, which leads to early genetic testing. Average survival is age 30 which speaks to the severe unmet medical need and providing care for these patients is challenging for their families, the healthcare system and society as a whole.
The PWS community is well organized with a strong patient organization, specialized clinics staffed by experienced clinicians providing these patients with greater access to the healthcare system than many patients with HO, for example, who still don’t have a diagnosis. It is a complex genetic disease with multiple genes involved, but central to the disease is impaired signaling through the MC4R pathway. The six-month phase 2 data Dr. Miller presented at Endo in June showing a reduction in hyperphagia scores, anxiety scores and BMI values supports the potential role of an MC4R precision agonist in the management of this disease.
Now I will extend the CF analogy one step further. Vertex recognized with their first approval in CF that they had a unique ability to make a major difference in this disease, so they literally doubled and tripled down on trying to optimize outcomes. Our strategy will be similar. We will run our first phase 3 trial with bivameligon, but with the expectation, given the size and importance of this opportunity, we would develop both of our next generation therapies in this disease.
We expect, as previously indicated, the decision to start with bivameligon has some practical aspects more specifically related to CMC and availability of supply. We simply have an ability to start earlier than we would with 718. Furthermore, as with CF, we expect a monotherapy to work and be approved, but given the complexity of the disease, optimal outcomes will be achieved by combination therapies. We are not prepared to discuss our portfolio at this time.
We will do that in 2027, but the message today is simply that we are working to develop the best possible to MC4R diseases based on our understanding of the biology and that will include proprietary combination approaches. Moving to slide seven, we’re excited to initiate our phase 3 bivameligon HO study. This is a 52-week comparably sized study to the original Transcend study with BMI as the primary endpoint. We hope to begin screening patients at the first sites by the end of the year and expect to dose the first patients in January. With a decision to proceed with bivameligon in PWS, we will now engage with regulators with the goal of initiating a six-month phase 3 study in PWS with hyperphagia as measured by HQCT as a primary endpoint in 2027.
I want to provide a shout out to the Rhythm teams who have not been idle on the development front. In preparation for the phase 3 studies, we needed to complete a bioequivalence study with a new formulation, a food effect study, a drug-drug interaction study and a hepatic impairment study, all of which have been completed satisfactorily in the past year, further de-risking bivameligon as a core asset as we move into phase 3 trials. Finally, a brief word about a recently announced amendment of the LG Chem deal. Our decision to amend was driven by two factors. First, our growing belief in the overall opportunity related to MC4R pathway diseases giving us the confidence to buy down the existing royalty and secondly, the original structure, despite our best efforts to align the two companies’ interests, left us with an economic disincentive to fully develop bivameligon as that would drive disproportionate royalty burden relative to developing 718.
In the current structure we are fully aligned and can simply, as we would want to do, follow the science and develop the best possible treatment for the indication. I remind you that the patent protection for both biva and 718 extends into the 2040s providing sustainable long-term value for shareholders. Hunter will provide more details on the LG Chem transaction. I’ll now turn the call over to Jeff.
Jennifer Lee (Executive Vice President, Head of North America)
Thank you, David. I’ll begin on slide 9. I’m pleased to report on the strong performance we’re seeing in the U.S. launch of Imcivree for acquired hypothalamic obesity in the third quarter, the second full quarter of launch. Patient demand continues to be strong and, consistent with what we experienced our first quarter of launch, we’re seeing growth in a broadening base of prescribers. On the patient access side, reimbursement trends are highly encouraging, reflecting our ongoing payer engagement leading to an increase in Imcivree policy adoption.
On slide 10, I’ll start with metrics related to patient demand. Since launch on March 19 and through the first two quarters, we have received more than 700 patient start forms for acquired HO, including start forms for 71 clinical trial patients. Consistent with the first quarter, the patient age distribution remains evenly split among older and younger than 21, with 20% of patient start forms from patients between 4 and 12, pointing to the urgency that comes with caring for children.
We are pleased with the distribution of time from hypothalamic injury among patients with prescriptions. Consistent with the prior quarter, approximately 15% of prescriptions have come from patients injured within the last two years; hence they were diagnosed and treated early in their disease journey. More than a third of patient start forms have come in for those whose hypothalamic injury was 10 years ago or more. These early dynamics are encouraging and demonstrate meaningful demand across a broad range of patients and a long-term growth opportunity in both incident and prevalent patients.
Slide 11 outlines how we are approaching the opportunity, balancing the institutional medical centers where patients begin their journey and then following the patients through their care. The first point of contact for patients with hypothalamic injury comes at a major medical center or large healthcare organization where there is a concentration of new patients each year. Upon launch, we identified 43 of these priority accounts, and as we had more engagement in the field, we continue to see a growth in the number of these identified accounts.
Approximately 30% of patients remain engaged with the care teams at these larger centers, while approximately 70% of patients transition beyond their original point of care. The total number of accounts and percentage of patients within these accounts will continue to evolve throughout the launch. With that said, our HCP tiering provided to our sales teams are based off of where the volume of potential patients are, and our commercial approach balances physician engagement at these institutional accounts as well as the physicians across other care settings, reflecting where patients enter care and where they are receiving ongoing follow-up care.
We remain focused on driving earlier diagnosis and treatment of acquired HO patients within a year of onset of their acquired hypothalamic obesity symptoms, while working to ensure prevalent patients who have been living with acquired HO for several years also get to an accurate diagnosis and treatment. Onto slide 12, we continue to see strong growth and a broadening base of prescribers. Launch to date, we have received start forms for more than 500 prescribers, including approximately 200 first-time acquired HO prescribers during the third quarter. Of the HO prescribers to date, approximately 25% of them have written two or more Imcivree prescriptions for acquired HO, an incremental increase from the 20% repeat prescriber metric at the end of the second quarter.
This points to a positive experience with prescribing Imcivree, but also reflects the early stage of the launch and the opportunity that remains as many of these HCPs have more than one potential acquired HO patient under their care. Overall, 80% of prescribers are endocrinologists, evenly split between adult and pediatric endos, further supporting that acquired HO is a specialty opportunity. Slide 13: We’ve made significant progress with payers during the third quarter, and the early access trends and speed of approvals for reimbursement are highly encouraging. Payers are recognizing the differentiation and severity of acquired HO. Of approvals launched to date, 75% of them have come by the prior authorization stage, and over 80% of approvals have come within 60 days of receiving the start form. We are encouraged with the strong progress in securing positive payer policies.
As of the end of Q3, we have policies in place for acquired HO that affect approximately 60% of Medicaid covered lives and approximately 75% of commercial covered lives, and we expect that ongoing policies are going to be put in place throughout the end of the year. Importantly, these advances in payer coverage are resulting in positive access experiences for physicians and patients early in launch. Onto slide 14. Lastly, for me, a brief update on BBS. We continue to grow the BBS business during the third quarter through the efforts of our dedicated team of territory managers now exclusively focused on BBS. BBS remains an important contributor to Rhythm’s success. Now let me hand it over to Yan.
Yan
Sorry. Thank you, Jennifer. I will begin on slide 16. We are very pleased to have launched IMCIVREE for acquired HO in Japan on October 14th following approval by the Ministry of Health, Labor and Welfare, or MHLW, and inclusion on Japan’s National Health Insurance Drug Price List. This launch represents an important milestone for patients and families in Japan. IMCIVREE is the very first approved therapy specifically for acquired HO, a serious disease for which there were previously no approved treatment options.
It is also an important milestone for Rhythm Pharmaceuticals and reflects several years of intense and focused work. We made the decision to establish a presence in Japan because we saw significant unmet medical need with an estimated patient population of 5,000 to 8,000 patients and a specialist community that was highly engaged and focused on improving the diagnosis and treatment of acquired HO. Our first discussions with the Japanese regulatory authorities began in 2023.
Twelve Japanese patients participated in the TRANSCEND clinical trial and, through close collaboration across our global and Japanese teams, we were able to move efficiently from clinical development through regulatory review, pricing and now commercial launch. The MHLW approval for IMCIVREE came less than six months after the approval in the United States, five months and five days to be more precise. Our commitment to pursuing approval to benefit the lives of patients living with acquired HO in Japan is a point of pride for Rhythm Pharmaceuticals and very much appreciated in Japan, especially by the healthcare authorities.
We are excited to provide further details today as this is a meaningful and durable long‑term opportunity for Rhythm Pharmaceuticals. Slide 17. A successful rare disease launch is focused on disease awareness, medical education and scientific engagement. We believe this foundation is developing very well in Japan. In September, the Japanese Endocrine Society and an MHLW‑funded research group published official guidelines for the diagnosis and treatment of acquired HO. These guidelines are important because they describe the significant burden of acquired HO, recognize that conventional approaches to obesity are generally inadequate for these patients, and provide a more consistent clinical framework for disease recognition, diagnosis and treatment.
Also in October, the Japanese Endocrine Society, the Japanese Society for Pediatric Endocrinology and the Japanese Society for Hypothalamic and Pituitary Tumors issued a joint statement on the safe and appropriate use of setmelanotide for acquired HO. The TRANSCEND publication in the New England Journal of Medicine, which included four Japanese authors, further supports awareness of both the disease and the clinical evidence among Japanese physicians.
These data were presented and very well received in June in Kyoto at the annual meeting of the Japanese Endocrine Society. The approval and the launch, combined with these new treatment guidelines, support from leading medical societies and growing clinical experience in Japan, provide a substantially stronger foundation for identifying and treating patients with acquired HO. Moving to slide 18, we began building our Japanese organization in April 2025 because we believed success in this market would require deep local capabilities and sustained engagement with physicians and patients. Today we have more than 50 employees in Japan. This includes 16 hospital affairs leads, a position similar to the territory managers in the U.S., and six scientific affairs managers supporting medical education and scientific community engagement.
This is a very experienced team with deep rare disease background and a strong understanding of the Japanese healthcare system. Our targeting strategy reflects the way patients with acquired HO move through the healthcare system. We have identified and targeted 350 priority hospitals where a significant majority of patients with brain tumors are treated and, by extension, where patients with acquired HO are. This concentration allows us to take a disciplined, high‑touch approach.
Our teams are focused on increasing disease awareness and helping institutions build the processes required to initiate and maintain treatment. At the same time, we are working to ensure physicians and institutions have the knowledge and support required to identify the appropriate patients and provide a high‑quality treatment experience. Slide 19. Japan represents a large, accessible and durable rare disease opportunity, and our significant pre‑launch physician engagement and patient identification work gives us a strong starting point. To date our field teams have engaged with 300 of the 350 priority hospitals and, through this work, we have already identified more than 1,000 patients who may be eligible for IMCIVREE. Rhythm Pharmaceuticals is forging new ground in Japan, introducing a new disease to the market with an integrated medical and commercial model initially focused on high‑volume brain tumor centers.
We have built a scalable approach to expand patient identification and treatment access across Japan. Our approach begins with intensive, high‑touch engagement in these priority centers with the goal of replicating successful practices throughout Japan. While confident in the long‑term opportunity, there are structural features of the Japanese market that we expect will influence the early launch trajectory. During the first year of a new drug launch, prescriptions are limited to a two‑week supply.
This means patients must return to the hospital to consult with their physicians every two weeks to continue receiving treatment. This requirement applies during the first year of the product’s launch in Japan. We expect the restriction to be lifted in November 2027, after which eligible patients may receive prescription supply for up to three months. We anticipate a greater commercial contribution from Japan in the second and third years of launch.
The first year will be focused on building institutional experience, supporting patient identification and establishing accessible treatment pathways. As physicians gain experience and the prescription supply restriction is lifted, we expect the launch to accelerate in years two and three and continue building over time. Lastly, on slide 20, I will provide a brief update on Europe. We estimate that there are approximately 10,000 patients with acquired HO in Europe. With paid early access programs, physicians in France and Italy have treated well over 100 patients with HO, generating positive experience and real‑world evidence. Following European Commission authorization earlier this year, we have continued to advance the country‑level market access processes required for launch.
In Germany, the G‑BA process, which is our final step prior to launch, is nearing completion. We have also submitted reimbursement dossiers in France, Italy, the United Kingdom, Spain and the Netherlands. Our experience commercializing IMCIVREE for BBS and other rare MC4R pathway diseases provides a strong foundation for these discussions. Payers and many national experts are already familiar with setmelanotide, the MC4R pathway and Rhythm Pharmaceuticals’ rare disease approach.
We continue to expect country‑level launches for acquired HO in Europe during 2027, beginning with Germany and with the timing in each of the additional markets dependent on the reimbursement process. With the launch now underway in Japan and market access activities advancing well in Europe, we are establishing the next phase of IMCIVREE’s international growth and expanding our ability to bring this therapy to patients living with acquired HO around the world.
With that, I will turn the call over to Hunter.
Hunter Smith (Chief Financial Officer)
Thank you, Yan. Rhythm exited the third quarter in a strong financial position and we are encouraged by the early U.S. launch execution. The success of the launch is reflected in our quarterly revenue performance and underscores the quality of our business execution and increases our confidence in Rhythm’s potential sustainable long‑term value creation. I’ll provide more details on our quarterly performance shortly, but I’ll start with the details on the BIVA Melligon license amendment and our financing on slide 22.
Our amended license agreement for BIVA represents a significant investment in Rhythm’s MC4 agonist franchise. The original license with LG Chem completed in 2024 has been a tremendous success. The data generated in the Phase 2 trial in acquired HO deepened our conviction in VivaMelligon’s value potential and reinforced our belief that it can serve as a foundational asset in our MC4 agonism franchise. With this amendment, we are investing additional cash to increase our stake in Bivamelligon and hence our entire MC4R portfolio.
We project that our cross‑portfolio royalty obligations could have been 5% to 7% under the previous agreement. Pending future indication approvals for Bivamelligon, we are buying down this royalty obligation to 1% flat assuming U.S. approval for Bivamelligon. In exchange for this reduced royalty, we will pay LG $350 million in installments over the next four years separately, in addition to the $205 million in development and commercial milestones due to LG Chem under the original agreement.
Under the amended agreement, those are now payable on MC4 portfolio sales and LG Chem will receive an additional commercial milestone of $200 million if we achieve $3 billion in sales in a calendar year. When it comes time to pay that milestone, we would be happy to make that payment. Fixed payments totaling $350 million to LG Chem begin with the first payment of $70 million due 15 days from closing. Three additional payments will follow: $100 million in September 2027, $100 million in September 2028, and $80 million in September 2029. While the payments will be made in the annual installments as I’ve described, the present value of the aggregate amount will be recognized in the fourth quarter as research and development expenses.
Please note that for your models, next year at this time we anticipate two global pivotal trials evaluating Vivamelligon, one in HO and one in PWS, will be well underway. This transaction represents a strategic investment in our core business and our growing confidence in its potential. On slide 23, to finance this investment and strengthen our balance sheet, we entered into a five‑year term loan facility providing Rhythm access to as much as $750 million. We are pleased to continue and expand our partnership with Healthcare Royalty Partners, which dates back to the first half of 2022 when setmelanotide was approved for BBS and before we read out positive data from the Phase 2 trial of setmelanotide in HO. Also, we are now pleased to partner with Blackstone, a top‑tier global investor whose insights and collaboration were critical to catalyzing this transaction.
We believe the terms of this loan facility are competitive. The term loan commitment totals $500 million with an additional $250 million available by mutual option. The structure gives us access to capital without requiring us to draw the full amount, so we can match future borrowing decisions to funding requirements and continued development of the business. We will draw $250 million initially. Borrowings carry interest at SOFR plus 4.6%. The facility has a five‑year term, no scheduled principal amortization before maturity, no equity component, and no exit fee.
Funded amounts are subject to a 1% original issue discount and market‑standard non‑call and prepayment penalty provisions. As part of this transaction, we also amended our existing revenue interest financing agreement with Healthcare Royalty. These amendments remove certain debt‑like covenants and default provisions, limit HCR’s lien to backup security interest, and modify the agreement’s material adverse event provisions. We are a far different company today than we were in 2022 and these changes reflect that improvement. Taken together, the amended license agreement and financing strengthen our ability to invest in Viva, support our global commercial execution, and continue to advance the broader MC4 portfolio and our pipeline.
This transaction represents a bullish investment in Vivamelligon and in Rhythm’s future. On slide 24, we continue to be well capitalized with significant financial flexibility. We finished the quarter with approximately $321 million in cash, cash equivalents, and short‑term investments. This quarter‑closed cash on hand does not include any of the proceeds from the term loan nor the initial payment of $70 million to be paid to LG this quarter. As we’ve been saying for some time, we expect this cash amount to be sufficient to fund planned operations for at least 24 months.
On slide 25, we saw continued strength in our global commercial business during Q3. Global net product sales of IMCIVREE totaled $92.1 million, which represents 29% sequential growth over the second quarter. Patient demand remained strong during the third quarter, with the number of reimbursed patients on therapy globally increasing by 20%. During the third quarter, $67.5 million, or 73%, of product revenue was generated in the United States. The U.S. HO launch was the primary driver of sequential quarterly revenue growth and accounted for the significant majority of the increase in reimbursed patients.
On slide 26, I’ll walk you through the components of the sequential quarter‑over‑quarter revenue change. Global net revenue increased from $71.3 million in the second quarter to $92.1 million in the third quarter. We saw $15.1 million in revenue growth attributable to increased product demand in the U.S. More than 90% of that growth was attributable to the strong demand from patients with acquired HO. With the growing demand for IMCIVREE, product shipments to Rhythm Specialty Pharmacy outpaced patient dispensers by $4.5 million, resulting in a $1.3 million inventory effect on second quarter revenue.
Inventory on hand at the specialty pharmacy increased from 20 to 21 days. U.S. gross‑to‑net during the quarter was 86%, in line with Q2. International revenue increased $4.4 million sequentially to $24.7 million in the third quarter. The majority increase was attributable to the absence of a $3.8 million retroactive charge associated with the French Contribution M mechanism recognized during the second quarter. Excluding this item, the underlying international business increased quarter over quarter, with growth from patient demand offset by the inherent variability of shipments to named‑patient sales markets.
Our international business remains strong, as evidenced by continued growth of almost 10% in patients on reimbursed therapy during the third quarter. On slide 27 is a financial snapshot of the third quarter compared to the third quarter of 2025. Cost of goods sold was 13.2% of product revenue, primarily driven by cost of materials and royalty payments. R&D expenses were $52.5 million for the third quarter, compared to $46 million in the same period last year. Sequentially, the increase was $9.1 million versus the second quarter. The increase was primarily driven by increased personnel costs, development costs for Bivomeligon studies, and a development milestone associated with our CHI program.
SG&A expenses were $66.8 million for the third quarter. Sequentially, SG&A expenses decreased slightly by approximately 1% compared to the second quarter, largely reflecting timing around certain activities related to the U.S. and global HO launches. Weighted average common shares outstanding were approximately 69 million for the third quarter. GAAP EPS was a net loss for basic and diluted share of $0.61, including $0.01 per share from accrued dividends on convertible stock of $1.0 million.
Cash used in operations was approximately $3 million during the quarter. We ended the third quarter with $320 million in cash, cash equivalents, and short‑term investments. As I mentioned previously, looking ahead to slide 28 for the final quarter of this year, we are updating our annual OPEX guidance. We now anticipate approximately $360 million to $380 million in non‑GAAP operating expenses for 2026, comprised of non‑GAAP R&D of $165 million to $175 million and non‑GAAP SG&A of $195 million to $205 million. Guidance has changed very little. The midpoint of R&D has been reduced by $15 million due to research and formulation development activities planned to take place in late 2026 now likely occurring in early 2027.
As a reminder, non‑GAAP operating expenses exclude stock‑based comp and milestone payments associated with licensing agreements. Therefore, our guidance will not be affected by the recognition of the upfront payments due to LG Chem. With that, I’ll turn the call back over to David.
David Meeker (Chair, President and CEO)
Thank you, Hunter. So hopefully you’ve heard again this morning, really excited about where we are, confirming, I think after two‑fourths of launch, the potential of HO. We made great progress there and expect that to continue and very much looking forward to accelerating development of our next‑generation assets. As I said in my comments, updating you about future developments in the pipeline in 2027. So with that, we’ll open it up for questions.
OPERATOR
Thank you. As a reminder, to ask a question please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. Again, as a reminder, please limit to one question. Our first question comes from Phil Nadeau with TD Cowen. Your line is now open.
Phil Nadeau (Analyst at TD Cowen)
Good morning. Congrats on a really strong quarter. Thanks for taking our question. Our question, I think, is the obvious one: what you’re expecting for patient dynamics over the next quarter or two? It does seem like you’re adding about 100 PSFs per month. Is that sustainable? Where do you think you are in patient recruitment at this point in the launch? Thank you.
Jennifer Lee (Executive Vice President, Head of North America)
So, thanks for the question. I think overall, I would just say that we’re still early in the launch and there’s so much opportunity that remains. I was happy to see that overall, in terms of what we reported out, Q2 versus Q3, that volume in terms of RXs has not slowed down. And that remains the case even early into Q4. There’s still, in terms of opportunities, I would say probably the lowest‑hanging fruit is physicians who have a diagnosis patient. This is the quickest to convert, especially if they already have experience treating this patient with other therapies and they realize that they need something more. Just in terms of retreatment, we have educated a lot of physicians along the way who have identified a potential AHO patient that they suspected. And so they need to go through that validation process as that patient comes in for the next visit and then ultimately have that discussion around IMCIVREE treatment.
We also have physicians who are used to and already have patients who are on another therapy. And this was probably because IMCIVREE wasn’t available at that time, and they want to wait and see how the patient does before they think about RXing for IMCIVREE specifically, as well as a lot of other physicians that are still on our list that we need to educate. So, you know, all of this is going to take time to unlock with some opportunities quicker than others, but I feel very confident in terms of our ability to continue on with that disease as well as product education moving forward.
Phil Nadeau (Analyst at TD Cowen)
That’s very helpful. Thank you.
OPERATOR
Thank you. And the next question is going to come from Derek Archila with Wells Fargo. Your line is open.
Jennifer Lee (Executive Vice President, Head of North America)
Sure. So from an access perspective, we are very happy. I think, once again, all of that groundwork done to educate the payers to differentiate our patient population, that just flowed through in terms of what we’re seeing on the access side for acquired HO and potentially for any future approvals as well. I would say that the speed in terms of the approvals from Rx receipt to actually getting payer approval has been quicker than what we saw during the BBS launch.
For that reason, however, I will say that where we are, even though the percentage of covered lives is still a work in progress throughout the rest of this year, as P&T meetings happen, we’re already at a similar pace of approval times that we are with BBS. So I don’t expect that timeline in terms of approvals to dramatically increase as we, you know, increase the number of acquired HO-specific policies that we have in place. We’ve had the ability to get approvals even without a policy in place specific for acquired HO because of all that, once again, background work.
So I would say that the pace would stay the same just in terms of Rx to approvals moving forward. Thank you.
OPERATOR
Next question. And the next question comes from Tazeen Ahmed with Bank of America. Your line’s open.
Tazeen Ahmed (Analyst at Bank of America)
Hi, guys. Good morning. Thanks for taking my question. How should we be thinking about the, I guess the slope of the launch, given all the dynamics that you’ve mentioned? You’ve been careful to say that we should expect to see steady uptake. But once it becomes easier to get insurance payments, once those turnaround times normalize to where you expect it to be, and once more physicians are educated on the drug itself, should we expect to see any acceleration from the rate that we’re seeing right now?
Jennifer Lee (Executive Vice President, Head of North America)
Yeah. So I think that the reality is just what it looks like, just in terms of a lot of different rare diseases and the different states in terms of where those physicians are, it just takes time to go from even suspecting and then diagnosing and ultimately, especially in a realm where they may have been used to prescribing another drug because IMCIVREE wasn’t available, making that turn in terms of their actual experience with our drug and that process changing.
The other reality is that these physicians are extremely busy. Their practices are so busy. And just in terms of that ability to go through those steps, through the busy practice of that patient, coming in iteratively will also take some time. So that’s why we outlined just in terms of that steady pace. But once again, confidence just in terms of what this launch will look like as we’re just very early.
David Meeker (Chair, President and CEO)
Yeah. And Tazeen, just to build a little bit on what Jennifer said, the steadiness and I think the key part of her answer is there’s elements of a rare disease launch in this, which is patient by patient. There’s individual factors which may impact how quickly an individual patient is able to get to a diagnosis, get seen by their physician and get on treatment. What is strikingly different and why, you know, the opportunity here is so much different than BBS is that the EPI is fundamentally different and the concentration of patients fundamentally different.
So steady as it goes, but with a very different, you know, potential, you know, longer term, potentially. Next question.
OPERATOR
And the next question comes from Michael with Morgan Stanley. Your line’s open.
Michael (Analyst at Morgan Stanley)
Good morning. Thanks for taking the question and congrats on the strong quarter as well. Maybe just another follow up on the HO launch. Can you talk about the level of awareness you’re seeing among the endocrinologists sort of since the launch? You’re a little bit two quarters into the launch and just wanted to get a sense of where that is and how that could evolve going forward. Thanks.
Jennifer Lee (Executive Vice President, Head of North America)
I will say that when we started our education, our disease education efforts, which started even before our approval, there was a strikingly large opportunity to actually educate the physicians on what acquired HO actually was. And so that still remains the case. Just in terms of that ability to differentiate and outline that the weight gain in this patient population is not attributable just due to things around other hormones being administered to these patients, differences in lifestyle just because of a surgical procedure, et cetera, et cetera.
I think we have made progress for sure in terms of our efforts across our teams through different forums in addition to the one-on-one. But there’s a lot more physicians for us to educate to once again make them understand that similar to other hormone deficiencies, the cause of acquired HO is based off of the injury that is caused through that procedure and management of the tumor. So more opportunity to continue on though. Very happy, at least with the progress that we’ve made today.
David Meeker (Chair, President and CEO)
Mike, I’d contrast this again with Prader-Willi, where it’s a genetic diagnosis. There’s no challenge in a sense of getting to a diagnosis in almost all patients, whereas this is a clinical diagnosis and is generally, you know, some of these features can be seen in other settings, other diseases. And so getting to a diagnosis is different. And that’s the work that’s done and that’s the rare disease effort. And so tremendous opportunity to continue to increase awareness, as she said.
Michael (Analyst at Morgan Stanley)
Thank you. Congrats again.
OPERATOR
Thank you. And the next question is going to come from Paul Matthias with C4. Your line’s open
Matthew (Analyst at C4)
Hi there, this is Matthew on for Paul. Thank you so much for taking our question. I guess I wanted to dig into for the acquired HO population, what has the retention rate on drug been and how does that compare relative to your early BBS launch? Thank you so much.
Jennifer Lee (Executive Vice President, Head of North America)
The retention of our acquired HO patients, it is early in the launch. We expect that the patients that are probably the most motivated may start early on. The retention has been strong to date and the vast majority have remained on drug. I will say that our confidence in this particular patient population is a bit different in terms of that retention piece of what we expect compared to BBS, primarily because one, this perceived lack of efficacy is a bit different because of the strength in terms of that BMI reduction that we saw in our clinical study.
In this particular patient population, for example, other factors may include the fear or the burden of injections. And this patient population is more used to having injectable therapies due to their other hormone deficiencies. So there are differences, I would say holistically in terms of this particular population where we feel that their retention may be a bit different than BBS, but it’s early and it’s something that we’re going to continue to monitor as we move forward.
OPERATOR
Thank you. Next question. And our next question will come from Priyanka Grover with JP Morgan. Your line’s open.
Priyanka Grover (Analyst at JP Morgan)
Hi guys. Congrats on the quarter. So one thing you mentioned in the beginning of the call is Japan may be the second largest market for you guys considering EU has an estimated 10,000 patients for acquired HO. Can you provide more color on the major confidence in the Japanese market? Thanks, Jan.
Yan
Yes, for sure. So first, as you just said, the prevalence is meaningful. Two, we have in Japan a very well organized healthcare system. Three, we have a national coverage with a good price and those are extremely positive aspects, especially for a rare disease where patients sometimes are difficult to find. And I think that the 1,000 patients that we have already identified tells our efforts are paying and how many patients are there.
David Meeker (Chair, President and CEO)
And Priyanka, I mean Europe, obviously to Yan’s point, about a single country, that 10,000 in Europe is spread across countries with very different dynamics. So the accessibility of a slightly smaller EPI to 8,000 as opposed to the accessibility of the full 10,000 in Europe, it’s just fundamentally different. So all those reasons would say, yeah, Japan’s a tremendous opportunity for us.
Priyanka Grover (Analyst at JP Morgan)
Thanks for answering my question.
OPERATOR
Thank you. And the next question is going to come from Whitney Ijam with Canaccord. Your line is open.
Whitney Ijam (Analyst at Canaccord)
Hey guys. Congrats on the quarter, another one on Japan and sorry if I missed this but can you talk about where you landed on price and I guess the trajectory, how we should be thinking about the trajectory of price over time in Japan, is it kind of more similar to US or Europe as we think about kind of annual price changes? And then just the second part of that question. Do you plan to provide any patient start form or any other of those types of metrics as we watch the Japanese launch?
Yan
Yes, thank you. So first of all we are pleased with the price we achieved. One, it’s consistent with rare disease prices and also it’s consistent with what we were expecting and what we have forecasted. The price is public and it’s $2,200 per vial which is 60% to the US WAC, which is also very good compared to other U.S. rare disease drugs. And the second questions or the related questions in terms of long term price, it’s a price which is set for the next 10 years.
So of course there are some capping processes as you can imagine. But we are also very comfortable with where we are with MHLW estimated peak and how this price should be maintained in the upcoming years.
OPERATOR
Thank you. And our next question will come from Samantha at Citi. Your line is open.
Samantha (Analyst at Citi)
Hi, good morning. Congratulations on the quarter and thanks for taking the question. I have one on Prader-Willi. I’m wondering if you could just talk a bit about the decision to run a six-month hyperphagia-focused study. Will you include BMI changes in this initial study or will that be a separate BMI-focused study? And I’m wondering what you think you need from both an FDA approval perspective but also a payer utilization perspective in terms of hyperphagia versus BMI or both for utilization.
Thank you.
David Meeker (Chair, President and CEO)
Yeah, thanks, Samantha. So let me try to fill in a little bit on the Prader-Willi here. So we will run a six-month hyperphagia study. As I said in my initial comments, that is based on when we see that change. It’s a very important part of this disease. It fits with the mechanism of our drug. We do the satiety signal, patients get reduced hyperphagia, and that translates into changes in the HQ-CT, as we’ve seen in the six-month data we presented.
Number one, when Solano, now Neurocrine, when their drug VICAT was approved by the FDA, as is often the case with first approvals, they set a bit of a precedent and a template for others to follow. And I think we’ve heard indirectly that it’s probably a reasonable assumption to make that a six-month hyperphagia study is acceptable and, if positive, is approvable. So BMI changes, weight—we know from our other development efforts in the MC4R pathway diseases those are 12 months.
The regulators want to see sustainability of that BMI change over 12 months. So if that was the primary, the expectation is that would have been a 12-month. So from a very practical standpoint, this is a shorter and more direct route for approval. Your second question was, will we collect BMI in this? Absolutely. Will BMI change in six months? Yes, we’ve seen that already in the six-month, and so we would expect those positive results to be reflected in the label. Now it won’t be in a primary indication, but it will. Like I said, we do expect that to be in the label, and from a payer standpoint—and again I think VICAT’s a good example—in rare diseases payers tend to be very, they stick to the label.
Right. They don’t tend to interpret. You don’t tend to get step edits for unapproved therapies and the like. And so if you get an approved drug in Prader-Willi, again we can expect that it will be used, and they would not differentiate on whether hyperphagia was our primary endpoint or BMI is, in my opinion.
OPERATOR
Thank you. And the next question will come from Seamus Fernandez with Guggenheim. Your line is open.
Seamus Fernandez (Analyst at Guggenheim)
Oh great. So David, on the PWS side of things, it does seem like your conviction is quite a bit higher for the overall portfolio opportunity. But interested to just better understand maybe from you and Hunter the buy-down opportunity that you saw with the royalty and what the drivers of your expectations are for what looks like a very robust assumption for peak sales potential. How much did PWS weigh into that? Or was this kind of primarily your views of the global HO market opportunity and sort of the switch opportunity that you see moving from setmelanotide over to the new portfolio of assets?
David Meeker (Chair, President and CEO)
Yeah, thanks, Seamus. It was really, as I said in my comments, I mean the two major drivers here were, you know, number one, as you know, we’re running the phase 3 bivomelagon in HO. It will be the first next-generation product approved in HO. It doesn’t have hyperpigmentation, it’s an oral—lots of advantages. We think it will drive an incremental opportunity in the HO space, which we already think is large. And so that just economically said, if we can lower, you know, our royalty burden overall in that context, that made sense.
But the second factor, back to your question of strategy and how do we think about developing it: the original agreement did have a part of it which said if we develop bivomelagon in new indications, there’s an incremental royalty associated with that decision—for example, Prader-Willi. If we were to develop it in Prader-Willi, then yes, that would have carried a higher burden, and we didn’t want to be constrained by that. We’re starting with bivomelagon because it’s available and, as I said, supply issues are driving that near-term decision.
But we very much like having both next-generation assets available, particularly for our two major options, but in general to be able to make decisions around the portfolio fully driven by the science. So, turn over. Hunter, thoughts on that?
Hunter Smith (Chief Financial Officer)
Yeah, I’ll just add one thing, Seamus, which is we did multiple scenarios to analyze the economics of this deal and the opportunity here, and we based our analysis on essentially the potential reduction in royalties against the risk-adjusted sell-side consensus. So we were comfortable doing the transaction on that basis. If we were to outperform that consensus, either because probability of success goes up or the top-line unadjusted revenue goes up, that represents greater upside to the transaction.
We think we’re shaving about 5%. You have to make a couple of assumptions on what was going to be approved, but, you know, 5% against sell-side consensus yields a pretty good payback relatively quickly.
Seamus Fernandez (Analyst at Guggenheim)
Great. And if you don’t mind, just a quick follow-up on that. Was this initiated by Rhythm or was this kind of a competitive buy-down situation, just because we know there are other royalty players out there?
Hunter Smith (Chief Financial Officer)
It was initiated by Rhythm.
Seamus Fernandez (Analyst at Guggenheim)
Perfect. Thanks so much.
OPERATOR
Thank you. And our next question is going to come from Bosn Rodwin with Leerink. Your line is now open.
Bosn Rodwin (Analyst at Leerink)
Good morning, and thank you for taking our question, and congrats on the quarter. One question on HO. Were you able to identify more patients with suspected HO diagnosis than the 2,000 identified earlier in the launch? And how does Rhythm help with this process and the conversion from suspected to confirmed diagnosis? Do you guys provide help with the brain scans like the way you did with the genetic testing for kids for PWS and the monogenic indication?
That’s it. Thank you.
Jennifer Lee (Executive Vice President, Head of North America)
So we continue to—you know, the more we’re out there, the more physicians we educate, the more physicians are suspecting and getting patients to an HO diagnosis. So that number has continued to rise since we released that back in September over a year ago. And we’re using more, different metrics now post launch relating to Rx volume and such, just to be more holistic in terms of patients identified and ultimately getting to Rx. And that will be the metric that we use moving forward.
In terms of our help, I would say that it’s just education. It’s not necessarily—you know, these patients don’t have a genetic testing that can be supported through our URO program, but it is the combination of, do they have injury? And once again, we were really happy that our label is broader than just tumors. You know, so do they have an injury that resulted in hypothalamic dysfunctions, which can be measured by the need for different hormonal treatments?
And do they have weight gain because of this, and associated with that timing? So there are just a couple of different pieces of education to make that physician put those pieces together for that particular patient to get to that diagnosis.
David Meeker (Chair, President and CEO)
And it’s fundamentally very different, obviously, from BBS. I mean, I think when Jennifer’s teams are out there talking to physicians who might potentially see a BBS patient—just given the epidemiology and the nature of that rare disease opportunity—the odds are each individual physician, the probability they may see a BBS patient is low. When we’re talking to endocrinologists, the teams that are out there, the probability is high, and the clinical diagnosis—the clinical phenotype that drives that diagnosis, as Jennifer just described—is highly recognizable.
This isn’t one where they have to stretch to try to remember or do a special test to figure out if that disease is present. These are very obvious parts of the presentation, so the diagnosis is readily made once you know what you’re looking for. Next question.
OPERATOR
Thank you. And the next question is going to come from Lisa Walter with RBC Capital. Your line’s open.
Lisa Walter (Analyst at RBC Capital)
Oh, good morning. Thanks for taking our question. Given this is the second full quarter of the HO launch, just curious if you can share which payer group most patients are coming from, whether that be Medicare or the commercial population. And could we also expect any seasonality in the first quarter, as some patients may change insurers or care providers in the new year? Any color here would be helpful. Thanks so much.
Jennifer Lee (Executive Vice President, Head of North America)
Sure. So just in terms of the payer background, I would say that where we are right now in terms of percentage of Rxs that come from commercial and Medicaid payers is very much similar to the BBS, with the majority of those being commercial and Medicaid. I think what is interesting and a positive is that there are a bit more commercial payers on the HO side versus Medicaid payers. But, you know, we expect that there are going to be a lot of things that evolve over time—perhaps potentially the age mix of patients, which also may impact the payer mix and such—and it’s one thing that we will continue to monitor moving forward.
From an insurance perspective, this is a dynamic that happens every single year just in terms of, you know, for any drug out there. They may be impacted as patients are going through an insurance change. And we have experienced in the beginning of the first quarter, or in the first quarter, a bit of a bump up in terms of our patients on our bridge or free drug program, as we may have to temporarily bridge them onto this free drug therapy until we’re able to get reimbursement on their new insurance plan.
So that’s a dynamic that has happened year over year, and I imagine is the same for any drug as well.
OPERATOR
Thank you. And the next question is going to come from John Wolbin with Citizens. Your line’s open.
Kathryn (Analyst at Citizens)
Hi, this is Kathryn on for John. I’m just going to follow up on the PWS, and can you just discuss a little bit about differentiation on hyperphagia of bivomelagon versus other PWS programs, and kind of any learning from earlier programs as far as what we can expect as far as the placebo rate and what is clinically meaningful as far as benefit over placebo? Thank you.
David Meeker (Chair, President and CEO)
Sure. You know, Prader-Willi—again, I don’t need to remind all of you—it’s just an incredibly challenging disease. And, you know, there’s a reason almost all therapies, with the exception of VICAT, and there have been many, multiple attempts, failed to date. So, you know, we’re very respectful of that, number one. Number two, the placebo rate—there is a placebo rate for hyperphagia, absolutely. And part of the reason we will be running a six-month trial, although a shorter trial might be acceptable from a regulatory standpoint, is to make sure that we move well beyond that period of time, which might be—I’d say once you get out beyond three to four months—that placebo effect should absolutely have worn off. So I think a six-month trial, we feel confident that will show that.
The magnitude of the difference, that threshold has been set again by the VICAT approval—that was on the order of five points or less. So, you know, obviously we haven’t engaged with regulators yet and that could evolve. I expect them to more or less follow precedent there, and that would be our threshold of what we’d be looking to achieve.
OPERATOR
Thanks so much. Thank you. Thank you. And our next question comes from Capit Patel with Wolfe Research. Your line’s open.
Capit Patel (Analyst at Wolfe Research)
Yeah. Hey, good morning, and thanks for taking the questions. For the launch, can you break down the new HO start forms by month for the third quarter and sort of characterize how that pace has been heading into October, October–November? And then second, for the PWS study for the phase 3, have you finalized what the restriction or the minimum baseline HQ-CT score might be for that phase 3? Thank you.
David Meeker (Chair, President and CEO)
Yeah, yeah. We don’t break out by month, so I think the quarter in a rare disease, particularly early in launch, I think is a very high level of discrimination. And what Jennifer said is going into the fourth quarter, yeah, the pace seems to be very much holding up. And so we’re seeing no slowdown as we go into the fourth quarter, but we won’t break out by month. Sorry. On the Prader-Willi question was—yeah.
Capit Patel (Analyst at Wolfe Research)
What the baseline HQ-CT score—
David Meeker (Chair, President and CEO)
Might be for that day. Yeah, yeah. I mean, historically companies have used an HQCT of 13. I’m not sure. We haven’t engaged with regulators yet and we haven’t made a final decision internally. But I would say in general we’re not going to try to create new ground here. We’re going to follow, you know, established precedent in terms of, you know, trial designs that have at least structurally been performed previously. So.
OPERATOR
Thank you. And the next question will come from Joseph Stringer with Needham. Your line is open.
Eddie
Good morning, this is Eddie on for Joey. Thanks for taking our question. Just another one on the start forms. Just wondering if the delta between the patient start forms and initiation of therapy of the 60 days, is that something kind of a baseline we should expect going forward? And how has this changed since the launch has been progressing? And how might this be the same or different for other geographies? Appreciating as the launch is still pretty early in Japan and Europe.
David Meeker (Chair, President and CEO)
So maybe just on those metrics, they are very US-specific. There’s a completely different dynamic in, obviously, Japan — you get one payer — and then Europe, country by country. But I think Jennifer maybe just comment on our average and how does that distribution work for a different patient group?
Jennifer Lee (Executive Vice President, Head of North America)
Yeah, so I would say that overall, just in terms of the pace of approvals, I don’t expect that to dramatically increase. As I outlined, it is a pretty similar time frame as what we are seeing also on the BBS side. So from that perspective I feel very good that we’re already at that pace. And we also have others that are continuing on just in terms of the pending and we’re still working through to get to approvals overall. So I don’t expect that. I do expect, and there’s no reason to believe, that we won’t be at a similar percentage of covered lives with IMCIVREE policies as we are right now with BBS. So that piece will continue to evolve as the P&T meetings ultimately happen at various different payers and we get those in place.
David Meeker (Chair, President and CEO)
These rare disease therapies almost always have a prior auth. Now, we’re happy to have a number of situations where they don’t require a prior auth, but in general they require the prior auth. So there’s a built-in inherent delay from the time the script gets written until the patient gets on therapy. And then, as Jennifer said, if you end up in a multiple appeals process, then obviously you stay in the queue and that timeline extends over, so your average longer than 60 days perhaps.
But what’s really rewarding and promising about this is that we very quickly got to a point where we’re getting relatively rapid approvals.
Eddie
Thank you.
OPERATOR
Thank you. And I would now like to turn the call back over to David Meeker for closing remarks.
David Meeker (Chair, President and CEO)
Thank you. So again, thanks all for tuning in. Continue, as I said in my opening, to make great progress and are really excited about what’s going to come up in 2027. Next time we’ll talk, we’ll be well into the first quarter there. So thanks again.
OPERATOR
This concludes today’s conference call. Thank you for participating and you may now disconnect.
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