Rhythm Pharmaceuticals, Inc. (RYTM): Business Model Canvas [Dec-2025 Updated]

You're digging into how Rhythm Pharmaceuticals, Inc. actually makes money and builds value, and honestly, it boils down to mastering the MC4R pathway for rare obesity. As someone who's looked at balance sheets for two decades, I see a focused operation: they are driving global sales of IMCIVREE, which brought in $137.5 million in net product revenue for the first nine months of 2025, while aggressively funding R&D for pipeline growth, all supported by a solid $416.1 million cash cushion as of Q3 2025. This canvas breaks down exactly how their specialized patient support, key partnerships like the one with LG Chem for bivamelagon, and high-touch sales force translate that science into revenue streams-dive in below to see the whole nine-block structure.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Key Partnerships

Specialty pharmacies and distributors for drug delivery

In the United States, Rhythm Pharmaceuticals sells its product to one specialty pharmacy. The product moves through a third-party logistics, or 3PL, distribution agent; this agent does not take title to the product. For the first quarter of 2025, U.S. product revenue saw a net decrease of $7.2 million compared to the fourth quarter of 2024. This was due to an $8.3 million decrease in inventory at the specialty pharmacy that dispenses IMCIVREE, offset by a $1.1 million increase in product dispensed to patients. For the third quarter of 2025, revenue generated in the United States was $38.2 million, which represented 74% of the total product revenue for that quarter.

Clinical research organizations (CROs) for global trials

Rhythm Pharmaceuticals utilizes external organizations to support its global clinical development programs. As of late 2025, key trials involving external support include the ongoing Phase 2 trial of setmelanotide in Prader-Willi syndrome (PWS), which planned to complete enrollment in the second half of 2025. Furthermore, the Phase 1, Part C trial evaluating RM-718 in acquired hypothalamic obesity was anticipated to complete enrollment in the first quarter of 2026. The Phase 2 trial for bivamelagon involved 28 patients in the initial study design.

LG Chem for the in-licensed oral MC4R agonist, bivamelagon

Rhythm Pharmaceuticals in-licensed bivamelagon from LG Chem, Ltd in January 2024. The financial structure of this partnership involved significant upfront payments and future contingent payments.

In the Phase 2 trial for bivamelagon in acquired hypothalamic obesity, the 600mg cohort (n=8) achieved a -9.3% BMI reduction from baseline at 14 weeks (p-value=0.0004). The placebo cohort (n=7) increased BMI by 2.2% over 14 weeks.

Patient advocacy groups for rare disease awareness

Rhythm Pharmaceuticals actively engages with patient advocacy organizations to support research and awareness efforts for rare neuroendocrine diseases.

• On March 18, 2025, Rhythm announced a research collaboration with the Raymond A. Wood Foundation.
• The Raymond A. Wood Foundation is a patient advocacy organization for survivors of craniopharyngioma and hypothalamic-pituitary brain tumors.
• The collaboration is set to study the impact of fatigue on persons with craniopharyngioma.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Key Activities

You're looking at the core engine of Rhythm Pharmaceuticals, Inc. (RYTM) as of late 2025, which is all about driving IMCIVREE (setmelanotide) adoption while aggressively pushing the pipeline forward. These activities are what keep the lights on and fund the next breakthrough.

Global commercialization of IMCIVREE (setmelanotide)

The commercial engine is definitely gaining steam. You saw strong sequential growth through the year, which is what you want to see for a rare disease product. For the third quarter of 2025, net product revenues from global sales of IMCIVREE hit $51.3 million. That's a solid jump from the $33.3 million seen in Q3 2024.

The US market is the primary driver, bringing in $38.2 million, which accounted for 74% of the total product revenue in Q3 2025. Outside the US, revenue was $13.1 million, making up the remaining 26%. To be fair, the international piece saw a sequential dip of 21% from Q2 2025, but the overall trajectory is up. The number of patients on reimbursed therapy in the US grew 14% in Q1 2025 compared to the prior quarter. Plus, in October 2025, they locked down a final reimbursed price with the French Economic Committee for Health Products (CEPS) for BBS and POMC and LEPR deficiencies.

Here's a quick look at the revenue progression leading up to that Q3 number:

IMCIVREE, known as Civri in Europe, is approved and available in about 14 countries there. That's a key part of the global commercialization effort.

Research and development (R&D) for pipeline expansion

Rhythm Pharmaceuticals is definitely spending to build out beyond the current indication. For the nine months ending September 30, 2025, Research and Development (R&D) expenses totaled $125.3 million. Just for the third quarter of 2025, R&D spend was $46.0 million, up year-over-year from $37.9 million in Q3 2024. The full-year 2025 Non-GAAP Operating Expense guidance suggests R&D expenses will land between $150 million to $165 million for the year.

The R&D focus is on lifecycle management and new targets. You're looking at several near-term milestones that will define the next phase of growth:

• Disclose preliminary results from the Phase 2 trial evaluating setmelanotide in Prader-Willi syndrome in the fourth quarter of 2025.
• Phase II data for the oral formulation of setmelanotide expected in the third quarter of 2025.
• Advancing investigational MC4R agonists bivamelagon and RM-718.

The composition of matter patent for setmelanotide expires in 2032, so developing these new formulations is critical for lifecycle management.

Regulatory submissions for new indications like acquired hypothalamic obesity

This is a major activity, as success here unlocks a significant new patient population. Rhythm submitted a supplemental New Drug Application (sNDA) to the FDA for setmelanotide for acquired hypothalamic obesity (AHO) on August 20, 2025, following positive Phase 3 TRANSCEND trial data. The FDA granted Priority Review, setting the Prescription Drug User Fee Act (PDUFA) goal date for a decision on December 20, 2025. They also validated the Type II variation submission to the European Medicines Agency (EMA) for the same indication.

The Phase 3 TRANSCEND trial data is the foundation for this push. The key efficacy number you need to watch is the -19.8% placebo-adjusted body mass index (BMI) reduction achieved by the overall patient group (N=120) at 52 weeks. For adult patients 18 years and older (n=49), the result was a -19.2% placebo-adjusted BMI reduction at 52 weeks.

Key regulatory milestones for AHO include:

Manufacturing and supply chain management for a sterile injectable product

Managing the supply chain for a sterile injectable like IMCIVREE is a non-negotiable, high-stakes activity. While specific 2025 manufacturing cost figures aren't public, the financial health supporting these operations is clear. As of September 30, 2025, Rhythm reported cash, cash equivalents, and short-term investments of approximately $416.1 million. This cash position, combined with their current operating plans, is expected to fund operations for at least 24 months. The company anticipates total Non-GAAP Operating Expenses for the full year 2025 to be in the range of $295 million to $315 million. This financial buffer is what allows them to manage the complex logistics of a commercial sterile product while funding pipeline development. It's defintely a core operational support function.

Finance: draft 13-week cash view by Friday.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Key Resources

You're looking at the core assets Rhythm Pharmaceuticals, Inc. (RYTM) relies on to drive its commercial and clinical strategy as of late 2025. These aren't just line items; they are the foundation of their market position in rare neuroendocrine diseases.

Proprietary melanocortin-4 receptor (MC4R) agonist platform

Rhythm Pharmaceuticals, Inc. anchors its value in its proprietary platform targeting the MC4R pathway, which is central to energy homeostasis. This resource isn't just one drug; it's a pipeline of assets designed to treat rare obesity and related disorders. The platform includes the already commercialized setmelanotide and the next-generation oral candidate, bivamelagon.

The clinical validation for the platform is substantial, particularly in acquired hypothalamic obesity (AHO), which Rhythm estimates affects between 5,000 to 10,000 people in the U.S. alone. The data from the Phase 3 TRANSCEND trial for setmelanotide showed a 19.8% placebo-adjusted reduction in BMI for AHO patients. Furthermore, the oral candidate, bivamelagon, showed promise in its Phase 2 trial, with the 600mg cohort (n=8) achieving a 9.3% BMI reduction from baseline.

The platform's key components as of late 2025 include:

• Setmelanotide (IMCIVREE) for syndromic/monogenic obesity.
• Setmelanotide for acquired hypothalamic obesity (sNDA decision expected December 20, 2025).
• Bivamelagon, an oral MC4R agonist, with Phase 2 data supporting potential for a next-generation therapy.
• RM-718, another pipeline asset.

IMCIVREE (setmelanotide) drug and patent portfolio

IMCIVREE (setmelanotide) is the currently commercialized asset, generating net product revenues of $51.3 million in Q3 2025. Rhythm Pharmaceuticals, Inc. has strategically consolidated its control over this asset, reacquiring the global franchise rights for China, Hong Kong, and Macau in March 2025. This move ensures Rhythm owns the entire global commercial opportunity for setmelanotide.

The intellectual property surrounding IMCIVREE provides a significant barrier to entry for competitors. The patent protection is robust, with the last outstanding exclusivity set to expire in 2031, and the estimated generic launch date is not until July 04, 2034. Here's a look at some of the key U.S. drug patents protecting the asset:

Strong cash position of approximately $416.1 million as of Q3 2025

Financial stability is a critical resource, and Rhythm Pharmaceuticals, Inc. ended Q3 2025 in a very strong position. As of September 30, 2025, the company reported cash, cash equivalents, and short-term investments totaling approximately $416.1 million. This robust balance sheet is a direct result of strong IMCIVREE sales growth and a successful equity offering of $189 million in July 2025. Honestly, this cash position is expected to be sufficient to fund planned operations for at least 24 months, giving the management team runway to execute on the anticipated HO launch and pipeline development without immediate financing pressure.

For context on the financial strength:

• Cash as of September 30, 2025: $416.1 million.
• Cash as of December 31, 2024: $320.6 million.
• Cash used in operations during Q3 2025: Approximately $27 million.

Specialized rare disease commercial and medical affairs teams

Successfully bringing a therapy like IMCIVREE to market, especially in rare diseases, requires a highly specialized commercial and medical infrastructure. Rhythm Pharmaceuticals, Inc. has built and expanded these teams specifically for the rare disease landscape. The company was actively expanding its existing commercial teams with the hiring of experienced individuals in preparation for the anticipated launch of IMCIVREE for acquired HO later in 2025. This specialized human capital is essential for navigating the complex diagnosis and reimbursement pathways for ultra-rare conditions.

The scale of the organization supporting these key resources as of October 2025 was approximately 364 employees across North America, Europe, and Asia. The management team, including CEO David Meeker, has an average tenure of about 5.4 years, indicating institutional knowledge in this niche area.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Value Propositions

You're looking at the core value Rhythm Pharmaceuticals, Inc. (RYTM) delivers to its customers-the patients and prescribers dealing with severe, rare forms of obesity. This isn't about general weight loss; it's about providing targeted, first-in-class mechanisms for conditions that standard treatments often fail to address.

First-in-class therapy for severe hyperphagia and obesity in specific rare genetic diseases

Rhythm Pharmaceuticals, Inc. (RYTM) offers IMCIVREE (setmelanotide) as an indicated therapy to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS), POMC, PCSK1 or LEPR deficiency, as determined by an FDA-approved test demonstrating pathogenic, likely pathogenic, or of uncertain significance (VUS) variants in those genes. This is the foundation of their initial commercial value proposition.

The commercial traction shows this value is being realized:

• Net product revenues from global sales of IMCIVREE reached $51.3 million for the third quarter of 2025.
• Net product revenue for the second quarter of 2025 was $48.5 million.
• The number of patients on reimbursed therapy increased by 14% in the first quarter of 2025 compared to the fourth quarter of 2024.
• In Q3 2025, revenue generated in the United States was $38.2 million, representing a 19% sequential increase.

Significant BMI reduction in patients with acquired hypothalamic obesity (HO)

The value proposition extends significantly into the acquired Hypothalamic Obesity (HO) space, where setmelanotide has demonstrated robust efficacy in the Phase 3 TRANSCEND trial, a condition where previous anti-obesity medicines often failed to show consistent, sustained weight loss. You need to see the numbers to appreciate the clinical impact here.

Here's a breakdown of the key efficacy data presented in 2025:

These results show statistically significant BMI reductions across patient groups. The therapy also showed meaningful improvements in cardiometabolic health and patient-reported outcomes.

Potential for an oral next-generation therapy (bivamelagon)

Rhythm Pharmaceuticals, Inc. (RYTM) is developing bivamelagon, an investigational oral melanocortin-4 receptor (MC4R) agonist, to offer a more convenient dosing option for HO patients. The Phase 2 trial data suggests this oral therapy delivers efficacy consistent with setmelanotide, which is an injectable. The goal is to transform the patient experience through an oral formulation.

At 14 weeks in the Phase 2 trial for acquired HO, the BMI reductions were statistically significant compared to placebo, which saw a 2.2% increase in BMI.

• 600mg cohort achieved a 9.3% BMI reduction (p=0.0004).
• 400mg cohort achieved a 7.7% BMI reduction (p=0.0002).
• Patients on the 600mg dose achieved a mean reduction greater than 2.8 points in their \'most\' hunger scores on a TEN-point scale.

Rhythm plans to seek input from U.S. and EU regulatory authorities on a Phase 3 trial design for bivamelagon.

Comprehensive patient support programs for complex, rare conditions

For complex, rare conditions like those treated by Rhythm Pharmaceuticals, Inc. (RYTM), access and affordability are critical components of the value proposition, often managed through robust Patient Support Programs (PSPs). While specific RYTM PSP metrics aren't detailed in the latest filings, industry trends show the high demand for these services.

Industry data from a Q1 2025 survey of pharmaceutical executives highlights the focus areas for PSPs:

• 80% of executives reported that copay assistance was the most used and popular PSP offered.
• 69% suggested that patient access and affordability programs (PAPs) were the most utilized.
• PAPs were cited by 78% as leading to a positive patient experience.

Without this type of financial assistance, nearly a third of patients struggle to afford their medications. This support structure is defintely key to ensuring patients with rare diseases can start and stay on therapy. Finance: review Q3 2025 SG&A spend against patient enrollment growth by end of month.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Customer Relationships

You're looking at how Rhythm Pharmaceuticals, Inc. builds and maintains relationships with the specialized, often geographically dispersed, patient and physician base for its precision medicines. For a company focused on rare neuroendocrine diseases, this relationship block is defintely where the commercial engine runs.

High-touch, personalized patient support and access programs are centered around the Rhythm InTune support program. This program is designed to provide resources, education, and information tailored to the unique needs of individuals living with rare MC4R pathway diseases, such as Bardet-Biedl syndrome (BBS). The success of these relationships directly impacts revenue realization, which for the third quarter of 2025 reached $51.3 million in global net product revenue from IMCIVREE sales. This revenue growth was supported by a 10% sequential increase in the number of patients on reimbursed therapy globally during that quarter.

Direct engagement with specialized physicians and centers of excellence is critical for driving adoption in a niche market. While specific 2025 metrics on the number of active centers of excellence aren't public, historical data shows the focus on onboarding new prescribers. For instance, in 2023, first-time prescribers averaged 48 new prescribers per quarter. The commercial team's efforts are clearly translating to market penetration, with US revenue for Q3 2025 hitting $38.2 million, a 19% sequential increase. The focus remains on getting the medicine to patients, as evidenced by the fact that IMCIVREE is now available for BBS and/or POMC/LEPR deficiencies in more than 25 countries outside the United States.

Educational outreach to geneticists and endocrinologists is heavily supported by presenting clinical data at major medical congresses. This scientific engagement builds credibility with the specialists who diagnose and manage these rare conditions. For example, Rhythm presented four datasets at ObesityWeek® 2025 and highlighted data from the ENDO 2025 meeting regarding setmelanotide efficacy in acquired hypothalamic obesity (HO). This data dissemination is key to driving the diagnosis and subsequent prescription of their therapies, especially as they await an anticipated FDA decision on the sNDA for HO by December 20, 2025.

Building long-term trust with the rare disease community involves ensuring access across geographies and supporting patients through complex treatment journeys. The company is committed not only to developing medicines but also to helping patients get the education, awareness, and access they need. The international footprint is expanding, with a final reimbursed price agreement reached with the French Economic Committee for Health Products (CEPS) for IMCIVREE in October 2025. This global push, alongside the ongoing support provided by programs like Rhythm InTune, solidifies the relationship with the patient community.

Here's a quick look at the commercial scale supporting these relationships as of late 2025:

The company's strong cash position of approximately $416.1 million as of September 30, 2025, provides the necessary runway to continue funding the specialized sales force and patient support infrastructure required for these high-touch relationships. Finance: draft 13-week cash view by Friday.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Channels

You're looking at how Rhythm Pharmaceuticals, Inc. gets its therapies, like IMCIVREE (setmelanotide), to the patients who need them, especially as they push for the acquired hypothalamic obesity (AHO) indication. The channels strategy is a mix of specialized logistics, a targeted field force, and heavy medical engagement.

Specialized pharmacy networks for drug distribution

Rhythm Pharmaceuticals relies on a focused distribution model, particularly in the United States, utilizing a specialty pharmacy to dispense IMCIVREE to patients. This is a critical channel for managing a rare disease product.

Here are some figures related to that distribution and market reach:

The company is actively working on securing reimbursement, having agreed to a final reimbursed price with the French Economic Committee for Health Products (CEPS) in October 2025 for BBS and POMC and LEPR deficiencies. That's how you lock in the channel for a specific market.

Direct sales force targeting rare disease specialists in the US and EU

The commercial channel relies on a direct engagement model to reach the limited number of rare disease specialists. The overall company size gives you a sense of the scale of this effort, though it's not purely sales headcount.

As of October 2025, Rhythm Pharmaceuticals Inc. had approximately 364 employees across 6 continents. This team supports the commercialization efforts in North America, Europe, and GCC countries.

Physician engagement metrics show the channel's traction:

• Cumulative IMCIVREE prescribers increased by 38% from Q2 2024 to Q2 2025.
• Cumulative IMCIVREE prescribers increased by 9% from Q1 2025 to Q2 2025.
• US prescribers for IMCIVREE increased by 13% from Q4 2024 to Q1 2025.

Geographically, revenue split for Q3 2025 highlights the US channel's current dominance:

• United States product revenue (Q3 2025): $38.2 million (74% of total product revenue).
• Outside United States product revenue (Q3 2025): $13.1 million (26% of total product revenue).

Global regulatory pathways (FDA, EMA) for market access

Regulatory milestones are the gateway for expanding the commercial channels. The focus in late 2025 is on the AHO indication for setmelanotide.

Key regulatory dates and statuses:

The FDA's Priority Review status for the AHO sNDA, accepted on August 20, 2025, is designed to expedite this critical market access channel.

Digital and in-person medical education programs

Educating healthcare professionals (HCPs) is a core channel activity to drive diagnosis and prescription volume. Rhythm Pharmaceuticals actively presented data at major medical congresses throughout 2025 to support this.

Examples of HCP engagement channels in 2025:

• The Endocrine Society's Annual Meeting (ENDO 2025) in July 2025.
• Obesity Medicine 2025 Annual Conference in April 2025.
• 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in March 2025.
• Pediatric Endocrine Society (PES) 2025 Annual Meeting in May 2025.

For instance, at ENDO 2025, Rhythm hosted a symposium titled: Understanding Acquired Hypothalamic Obesity, a Rare Neuroendocrine Disease, featuring speakers like Dr. Odelia Cooper and Dr. Ilene Fennoy.

The company also hosted an event for investors and analysts on Wednesday, September 24, 2025, in Boston, MA, to review U.S. launch plans for AHO.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Customer Segments

You're looking at the specific patient populations and specialists Rhythm Pharmaceuticals, Inc. (RYTM) targets with its therapies, primarily IMCIVREE (setmelanotide) and pipeline assets.

The core customer base for the currently approved indication is defined by specific genetic conditions requiring treatment for excess body weight and long-term weight maintenance.

• Patients, aged 2 years and older, with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS).
• Patients with monogenic obesity due to genetically confirmed POMC, PCSK1, or LEPR deficiency, as determined by an FDA-approved test demonstrating pathogenic, likely pathogenic, or VUS variants.

The commercial performance reflects the current reach within these established segments. For the third quarter of 2025, Rhythm Pharmaceuticals, Inc. reported net product revenue from global sales of IMCIVREE of $51.3 million. Of this, $38.2 million, representing 74% of product revenue, was generated in the United States.

The potential expansion targets a significant, though still rare, patient group where clinical data suggests strong efficacy.

For patients with acquired hypothalamic obesity (HO), Rhythm Pharmaceuticals, Inc. has a supplemental New Drug Application (sNDA) pending with the FDA, with a Prescription Drug User Fee Act (PDUFA) goal date set for December 20, 2025. The Phase 3 TRANSCEND trial supported this submission, showing a 19.8% placebo-adjusted reduction in body mass index (BMI).

Estimates for the HO population show a substantial unmet need:

The specialized physician segment is crucial for diagnosis and prescription, especially given the complexity of these rare neuroendocrine diseases.

• The broader rare disease community in the U.S. is large, with 8% of U.S. adults reporting they or someone they live with has been told they have a rare disease [cite: 2 (SSRS)].
• The estimated diagnosed BBS population in the United States was 555 in 2020.
• The Bardet-Biedl Syndrome Market Size was projected to be 1.145 USD Billion in 2025.
• In the European Union, setmelanotide requires prescription and supervision by a physician with expertise in obesity with underlying genetic etiology.

The company is also tracking other rare obesity indications, with preliminary results expected from a setmelanotide Phase 2 trial in Prader-Willi syndrome in the fourth quarter of 2025.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Cost Structure

You're looking at the major outlays for Rhythm Pharmaceuticals, Inc. as they push toward a potential launch for acquired hypothalamic obesity later this quarter. The cost structure is heavily weighted toward R&D and commercial readiness, which translates to significant SG&A spend.

The company provided updated financial guidance for the year ending December 31, 2025, focusing on Non-GAAP Operating Expenses, which exclude stock-based compensation and fixed consideration related to in-licensing.

High Research and Development (R&D) expenses are a core cost driver, guided at $150 million to $165 million for FY 2025. This R&D spend supports ongoing work on setmelanotide and bivamelagon.

Significant Selling, General, and Administrative (SG&A) costs are also projected, guided at $145 million to $150 million for FY 2025. This reflects increased headcount and marketing costs associated with the anticipated launch of IMCIVREE in acquired hypothalamic obesity.

The total anticipated Non-GAAP Operating Expenses for the full year 2025 range from $295 million to $315 million.

Here's a look at the breakdown of the major expense categories:

• R&D expenses for the nine months ended September 30, 2025, were $125.3 million.
• SG&A expenses for the nine months ended September 30, 2025, totaled $137.5 million.
• Q3 2025 operating expenses were $98.5 million.
• Q3 2025 stock-based compensation expense was $18.8 million.

The R&D expense for the nine months ended September 30, 2025, was lower than the same period in 2024, primarily due to a net decrease in clinical trial costs.

Manufacturing and cost of goods sold (COGS) for IMCIVREE are not explicitly broken out in the latest guidance figures provided, but revenue from global sales of IMCIVREE for Q2 2025 was $48.5 million.

Clinical trial costs for setmelanotide and bivamelagon pipeline assets are embedded within the R&D expenses. For instance, the year-over-year decrease in R&D expenses for the nine months ended September 30, 2025, was partially attributed to a net decrease in clinical trial costs.

This table summarizes the key guidance and year-to-date actuals for the primary operating expense components as of late 2025:

The increase in SG&A spend from Q2 to Q3 2025 was approximately 14%, or $6.5 million, driven by headcount and marketing costs for the upcoming launch.

Finance: draft 13-week cash view by Friday.

Rhythm Pharmaceuticals, Inc. (RYTM) - Canvas Business Model: Revenue Streams

You're looking at the hard numbers for Rhythm Pharmaceuticals, Inc.'s (RYTM) top-line performance as of late 2025. The revenue engine is currently driven by one key product, IMCIVREE (setmelanotide), and the focus is clearly on expanding its reach and indications.

Net product revenue from global sales of IMCIVREE forms the entirety of the recognized product revenue stream. This stream shows significant year-over-year acceleration, which is what we need to track closely.

Here's a quick look at the top-line performance as of the latest reported period:

The growth is defintely real. For the nine months ended September 30, 2025, total net product revenue was $137.5 million, a substantial jump from $88.3 million for the same period in 2024. That's serious momentum.

Revenue growth is clearly being driven by increased patient demand for the Bardet-Biedl Syndrome (BBS) indication. We see this reflected in the quarter-over-quarter performance, too. For instance, the number of patients on reimbursed therapy increased 14% in the first quarter of 2025 compared to the fourth quarter of 2024. The third quarter of 2025 saw revenue of $51.3 million, up sequentially from the second quarter of 2025.

We can break down the geography for the third quarter of 2025:

• Revenue generated in the United States: $38.2 million, which is 74% of product revenue.
• Revenue generated outside the United States: $13.1 million, representing 26% of product revenue.

The next major potential revenue catalyst is tied to the acquired Hypothalamic Obesity (HO) indication for setmelanotide. The U.S. Food and Drug Administration (FDA) accepted the supplemental New Drug Application (sNDA) for Priority Review, assigning a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2025. This approval would open up a significant new patient population for IMCIVREE.

Keep an eye on these near-term revenue drivers:

• FDA decision on the acquired HO sNDA by December 20, 2025.
• European Medicines Agency (EMA) review for the same indication, which began in August 2025.
• Preliminary results from the setmelanotide Phase 2 trial in Prader-Willi syndrome expected in the fourth quarter of 2025.