What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-08-04 compared with 2026-05-05 · 188 added, 100 removed, 230 unchanged (56% of the section changed)

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the sufficiency of our cash, cash equivalents and short-term investments to fund our operations;

expected milestone payments and the timing thereof;

our ability to hire and retain necessary personnel;

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Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired HO and in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to BBS or pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

The European Commission (EC) has authorized IMCIVREE for the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity (aHO) due to hypothalamic injury or impairment, and for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above.

The United Kingdom’s Medicines & Healthcare Products Regulatory Agency (MHRA) has authorized IMCIVREE for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above.

In addition to the United States, we have achieved market

access or named patient sales of IMCIVREE for BBS or POMC and LEPR deficiencies, or both, in more than 25 countries outside the United States, and we continue to collaborate with authorities to achieve access in additional markets.

The European Commission (EC) has authorized IMCIVREE for the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment, and for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above.

deficiency in adults and children 2 years of age and above.

In addition to the United States, we have achieved market access or named patient sales of IMCIVREE for BBS or POMC and LEPR deficiencies, or both, in more than 25 countries outside the United States, and we continue to collaborate with authorities to achieve access in additional markets.

IMCIVREE’s label expansion to treat patients living with acquired HO was approved by the U.S.

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In addition, on May 5, 2026, we announced that our Japanese New Drug Application (JNDA) for setmelanotide to treat acquired HO was accepted, validated and is now under review by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) .

We anticipate PMDA's decision on the application in the second half of 2026 and, if positive, expect commercial launch by the end of 2026.

We anticipate anticipate launch of IMCIVREE for acquired HO in Japan, pending a decision by Japan's Ministry of Health, Labour and Welfare (MHLW), by year-end 2026.

Acquired hypothalamic obesity is a rare form of obesity that occurs following damage to the hypothalamic region of the brain.

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In July 2025, we announced bivamelagon achieved statistically significant and clinically meaningful BMI reductions at 14 weeks of treatment in a Phase 2 trial in patients with acquired HO.

We also anticipate completing enrollment in Part C of the Phase 1 trial evaluating the weekly RM-718 in patients with acquired hypothalamic obesity in the first quarter of 2026.

We have also completed enrollment in Part C of the Phase 1 trial evaluating the weekly RM-718 in patients with acquired hypothalamic obesity and announced preliminary, promising data on August 4, 2026.

We also are advancing MC4R agonists with the intention to improve the treatment landscape for patients living with Prader‑Willi syndrome (PWS), a rare, complex genetic neurodevelopmental disorder characterized by hyperphagia, severe obesity risk, and significant metabolic, cognitive, and behavioral complications.

PWS affects approximately one in 10,000 to 30,000 individuals worldwide, and approximately 20,000 patients in the United States.

We estimate there are between 12,500 and 16,000 patients living with PWS in the United States and a similar number in Europe, based on updated internal prevalence estimates developed using a bottoms-up methodology analyzing incidence, age-specific survival and claims-based validation.

Further, we estimate that 80% to 90% of PWS patients are living with hyperphagia and obesity, or approximately 8,500 – 12,750 patients.

There are currently limited therapeutic options that effectively reduce the extreme hyperphagia and address low resting energy expenditure associated with PWS.

Based on the central role of MC4R pathway dysfunction in PWS, we believe that targeting this biology represents a compelling therapeutic approach.

On December 11, 2025, we announced positive interim results from our exploratory Phase 2 trial of setmelanotide in patients with PWS.

Setmelanotide demonstrated BMI and hyperphagia reductions at month 3 and month 6, as well as safety and tolerability results consistent with setmelanotide’s well-established clinical profile.

On June 13, 2026, we announced positive preliminary data from a Phase 2 trial evaluating setmelanotide in patients with PWS that demonstrated treatment with setmelanotide was associated with improvements across multiple clinically relevant endpoints, including BMI, BMI-Z and measures for hyperphagia and anxiety.

In addition to this ongoing trial of setmelanotide, we have begun enrolling patients in a Phase 2 trial evaluating RM-718 in PWS.

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Our Phase 3 EMANATE trial did not achieve the primary endpoint in each of its four independent substudies evaluating setmelanotide in genetically caused MC4R pathway diseases, but we did see positive signals in two genetic indications.

We continue to analyze the EMANATE results and evaluate potential clinical development paths to develop one of our product candidates in patients with SRC1 (NCOA1) deficiency and POMC insufficiency, including with our next-generation MC4R agonists bivamelagon or RM-718.

We continue to

analyze the EMANATE results and evaluate potential clinical development paths to develop one of our product candidates in patients with SRC1 (NCOA1) deficiency and POMC insufficiency, including with our next-generation MC4R agonists bivamelagon or RM-718.

In addition, we plan to continue to evaluate the potential for MC4R agonism in the genes and gene families previously identified through the exploratory Phase 2 DAYBREAK trial, including the SEMA3 family, PHIP, TBX3 or PLXNA family.

Our sequencing-based epidemiology estimates show that each of these genetically-defined

MC4R pathway deficiencies are considered rare diseases, according to established definitions based on patient populations.

Our sequencing-based epidemiology estimates show that each of these genetically-defined MC4R pathway deficiencies are considered rare diseases, according to established definitions based on patient populations.

Our epidemiology estimates are approximately 4,600 to 7,500 for U.S.

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Additional recent clinical, regulatory, corporate and commercial updates include:

• On May 5, 2026, we announced that more than 150 patient start forms had been received for IMCIVREE in acquired hypothalamic obesity (HO) in the United States, within the first six weeks following approval by the U.S.

Food and Drug Administration on March 19, 2026;

• On May 5, 2026 we announced revenue from global sales of IMCIVREE was $60.1 million for the first quarter of 2026, an increase of 5% on a sequential basis from the fourth quarter of 2025, primarily driven by sales of IMCIVREE for the treatment of patients with Bardet-Biedl syndrome (BBS) and an increase in the number of patients on reimbursed therapy globally.

In the first quarter of 2026, revenue of $36.9 million, or 61% of product revenue, was generated in the United States, a decrease of 5% on a sequential basis primarily driven by specialty pharmacy inventory and some patients receiving drug under the Company’s bridging program as they transitioned insurance plans during the quarter.

Revenue of $23.2 million, or 39% of product revenue, was generated outside the United States, a sequential increase of $4.9 million or 27%;

• On May 5, 2026, we announced positive data from the Japanese cohort of our Phase 3 TRANSCEND study and that our New Drug Application for setmelanotide to treat acquired HO has been accepted, validated and is now under review by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA).

We anticipate PMDA's decision on the application in the second half of 2026 and, if positive, commercial launch by the end of 2026;

• On May 4, 2026, we announced new data presented at the Pediatric Endocrine Society Annual Meeting demonstrating sustained reductions in BMI and BMI Z‑score through 2.5 years of setmelanotide treatment and observed weight category improvements in pediatric patients (n=10) with acquired hypothalamic obesity and a second presentation demonstrating weight category improvement in the pediatric subpopulation of participants after 1 year of setmelanotide treatment;

• On April 3, 2026, we announced the appointment of Kim Popovits to our Board of Directors and the resignation of Ed Mathers.

• On March 26, 2026, the EMA's CHMP adopted a positive opinion recommending to expand the current marketing authorization for IMCIVREE ® (setmelanotide) to include the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired HO due to hypothalamic injury or impairment;

• On March 19, 2026, the FDA approved a label expansion for IMCIVREE to reduce excess body weight and maintain reduction long term in adults and pediatric patients aged 4 years and older with acquired HO;

• On March 16, 2026, we announced topline results from the Phase 3 EMANATE trial.

None of the trial’s four sub-studies met the primary endpoint, which was the difference in mean percent change in BMI from baseline to Week 52 versus placebo, analyzed in the modified intent-to-treat (ITT) population using prespecified multiple imputation to account for missing values and discontinuations.

Topline results include:

◦ POMC / PCSK1 Hets (N=78):

–4.3% placebo-adjusted reduction in BMI (p=0.15);

◦ LEPR Hets (N=23):

–3.6% placebo-adjusted reduction in BMI (p=0.94);

◦ SRC1 (NCOA1) (N=73):

–4.0% placebo-adjusted reduction in BMI (p=0.12);

◦ SH2B1 (N=121):

–1.7% placebo-adjusted reduction in BMI (p=0.43).

• According to post hoc analyses based on last observation carried forward (LOCF) for missing values, setmelanotide achieved statistically significant and clinically meaningful BMI reductions at Week 52 in the modified intent-to-treat patient populations in the POMC/PCSK1 Hets and SRC1 substudies:

◦ –5.5% least-squares mean difference in BMI in POMC/PCSK1 Hets patients (n=78;

◦ –6.2% least-squares mean difference in BMI in SRC1 (NCOA1) patients (n=73;

• According to post hoc analyses of genetically confirmed patients who reached Week 52 of treatment, setmelanotide achieved statistically significant and clinically meaningful BMI reductions in patients who completed the 52-week trial in the POMC/PCSK1 Hets and SRC1 substudies:

◦ –9.7% placebo-adjusted reduction in BMI in POMC/PCSK1 Hets patients (n=41;

◦ –8.0% placebo-adjusted reduction in BMI in SRC1 (NCOA1) patients (n=29;

• On February 26, 2026, we announced completion of an end-of-Phase-2 meeting with FDA regarding bivamelagon in acquired HO and disclosed encouraging open-label extension data from our Phase 2 trial that showed bivamelagon achieved persistent BMI reductions at six and nine months of therapy.

4, 2026, we announced that more than 400 patient start forms had been received for IMCIVREE for acquired HO from approximately 300 prescribers as of June 30, 2026, since the approval by the FDA on March 19, 2026;

• Revenue from global sales of IMCIVREE was $71.3 million for the second quarter of 2026, an increase of 19% on a sequential basis from the first quarter of 2026.

The number of patients on reimbursed therapy globally increased by greater than 20% as compared to the first quarter of 2026.

Revenue of $51.0 million, or 72% of product revenue, was generated in the United States in the second quarter of 2026, an increase of $14.1 million or 38% compared to the first quarter of 2026.

The sequential quarter over quarter growth in U.S.

revenue was driven primarily by increased patient demand for IMCIVREE in acquired HO, as well as continued growth in demand in Bardet-Biedl syndrome (BBS).

Revenue of $20.3 million, or 28% of product revenue, was generated outside the United States, a sequential decrease of $(2.9) million or (13)% compared to the first quarter of 2026.

The Company incurred a $3.8 million charge associated with a retrospective rebate in France, of which $3.1 million was related to revenue booked in periods prior to the second quarter of 2026.

The number of reimbursed patients on IMCIVREE continued to increase in the second quarter of 2026.

4, 2026, we announced preliminary data from Part C of the phase 2 study of RM-718 in acquired HO patients.

Eleven patients with acquired HO were enrolled in the ongoing, open-label trial.

Key preliminary findings include:

◦ -11.6% reduction in mean BMI from baseline (n=7) at 16 weeks of RM-718;

◦ RM-718 efficacy results at 16 weeks comparable to mean BMI reduction of -10.1% with bivamelagon (600 mg dose;

n=7) at 14 weeks and -10.1% BMI reduction with setmelanotide at 16 weeks (n=64 patients in Phase 2 and 3 trials);

◦ Two (2) mild instances of hyperpigmentation were reported and were limited to the injection site, with no generalized hyperpigmentation observed.

• RM-718 was generally well tolerated, with the most common adverse events being injection site reactions, nausea, and vomiting;

• Eight (8) patients remained on active treatment as of July 16, 2026, including two (2) patients who had not yet reached 16 weeks on therapy.

Two participants discontinued treatment due to adverse events:

injection site induration and nausea.

One patient withdrew from the extension portion of the trial.

4, 2026, we announced that six abstracts, including three oral presentations and three poster presentations have been accepted for presentation at the European Society for Pediatric Endocrinology’s Annual Meeting (ESPE 2026) September 8-10, 2026.

The presentations include:

◦ Efficacy and safety of setmelanotide in pediatric patients with acquired hypothalamic obesity:

final phase 3 trial results;

◦ Impact of setmelanotide on metabolic index scores in pediatric participants with acquired hypothalamic obesity - A Phase 3 trial post-hoc analysis;

◦ Pediatric patients with acquired hypothalamic obesity treated with setmelanotide:

real-world BMI and hunger data for up to 12 months in France;

◦ Long-term weight outcomes of setmelanotide in pediatric participants with Bardet-Biedl syndrome or with POMC or LEPR deficiency and obesity;

◦ The association of clinical features of Bardet-Biedl syndrome in a large population of patients with obesity and a positive genetic test for biallelic BBS variants;

◦ Clinical features associated with pathogenic or likely pathogenic biallelic Bardet-Biedl syndrome variants in a large population of patients with obesity;

• On July 8, 2026, we announced that results from Rhythm’s pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in patients with acquired hypothalamic obesity were published in The New England Journal of Medicine;

• On June 13, 2026, during the Endocrine Society’s Annual Meeting (ENDO 2026) we announced positive preliminary data from a Phase 2 trial evaluating setmelanotide in patients with PWS that demonstrated patients with PWS (N=17) achieved clinically meaningful BMI or BMI z-score reductions, reductions in fat mass with preservation of lean mass, and improvements in hyperphagia and anxiety measures.

Results from the six-month analysis demonstrate that treatment with setmelanotide was associated with improvements across multiple clinically relevant endpoints, as of a data cut off date of May 7, 2026.

Highlights include:

◦ Consistent BMI reductions in pediatric and adult patients at Month 6:

▪ -3.06% mean reduction in BMI (N=17 pts);

▪ -3.11% mean reduction in BMI in adult patients (n=10);

with six achieving >2.5% BMI reduction, and four achieving >4% BMI reduction;

▪ −3.00% mean reduction in BMI in pediatric patients (n=7);

▪ −0.35 mean reduction in BMI z-score from baseline in pediatric patients (n=7);

▪ Five (5) of seven pediatric patients achieved clinically meaningful BMI z-score reduction > 0.2;

◦ Setmelanotide achieved preservation of lean mass and reductions in fat mass across 16 patients with data available from DEXA scans:

▪ +0.74% mean gain in lean mass and -4.19% mean loss in fat mass across 16 patients;

▪ Six (6) of nine adult patients achieved >5% reduction in fat mass;

▪ Five (5) of seven pediatric patients gained ≥2.95% in lean mass;

◦ Clinically meaningful improvement in hyperphagia score observed in patients with moderate to severe hyperphagia, defined as a ≥7-point reduction in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score

▪ Eight (8) of 10 patients who entered trial with moderate to severe hyperphagia ( > 13 at baseline) achieved clinically meaningful improvement of 7 points or better.

◦ Improvement in PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) which measures anxiousness, emotional distress, and behavioral dysregulation.

▪ Of the 15 patients who had a baseline score >11, 10 patients achieved clinically meaningful improvement of ≥11 points;

◦ Safety and tolerability results have been consistent with the well-established profile observed with setmelanotide.

• In conjunction with the presentation of the above data, Rhythm also provided an update to its epidemiology estimates for PWS, including a PWS prevalence in each of the U.S.

and Europe at between 12,500 - 16,000 and an estimate that 8,500 - 12,750 PWS patients are living with hyperphagia and obesity in each of the U.S.

These estimates are based on our internal review and interpretation of the literature and available prevalence data, combined with our analysis of survival rate data.

• Also during ENDO 2026, we had additional data presentations that included long-term setmelanotide data in acquired HO;

one-year bivamelagon data in acquired HO;

post-hoc analysis of patients with acquired HO patients from the Phase 3 TRANSCEND trial with a history of bariatric surgery;

weight category improvements patients with acquired HO achieved with one year of setmelanotide treatment;

real-world efficacy analysis of U.S.

patients with BBS;

and results from the real-world RESTORE study of hyperphagia reduction in BBS patients treated with setmelanotide;

• On May 28, 2026, we announced the publication of a new evidence-based, consensus-driven diagnostic algorithm for Bardet-Biedl syndrome (BBS) in the peer-reviewed American Journal of Medical Genetics;

• On May 12, 2026, we announced six data presentations at The European Congress of Endocrinology (ECE), including real-world efficacy results in patients with acquired HO treated with setmelanotide enrolled in France’s early-access program, results from a study that evaluated hyperphagia severity in adults with Bardet-Biedl syndrome (BBS), results that demonstrated setmelanotide treatment in patients with acquired HO led to significant improvements in multiple metabolic index scores, and a study that highlighted the persistent and under-recognized health burden of acquired HO across life stages, drawing on the experiences of individuals with childhood-onset craniopharyngioma (CO-CP) from the Netherlands, Germany, and the UK.

We currently anticipate the following, near-term milestones:

• Announce six-month results from the ongoing exploratory Phase 2 trial of setmelanotide in PWS in the second quarter of 2026;

• European Commission decision on the Type II Variation for IMCIVREE in acquired hypothalamic obesity in the second quarter of 2026;

• Announce results from the Phase 1/2, Part C trial evaluating the weekly, MC4R agonist RM-718 in patients with acquired HO mid-year 2026;

• Complete enrollment in the substudy evaluating setmelanotide in congenital HO in the second half of 2026;

• Complete enrollment in the setmelanotide substudy in congenital HO in the second half of 2026;

• Complete enrollment in the Phase 1/2, Part D trial evaluating RM-718 in PWS in the second half of 2026;

• Anticipate launch of IMCIVREE for acquired HO in Japan pending a decision by Japan's Ministry of Health, Labour and Welfare (MHLW) by year-end 2026;

• Initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired HO by year-end 2026;

• Anticipate country-level launches of IMCIVREE for acquired HO in Europe beginning in 2027.

Following IMCIVREE's initial approval in the United States and marketing authorizations in the EU, Great Britain and Canada, we are continuing to pursue a country-by-country strategy to establish market access and reimbursement for IMCIVREE in additional countries.

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We expect we may need to continue to fund our operations through the sale of equity, debt financings or other sources.

We have built our own marketing and commercial sales infrastructure in the United States and are in the process

of building a similar infrastructure in several European markets and the United Kingdom.

We have built our own marketing and commercial sales infrastructure in the United States and are in the process of building a similar infrastructure in several European markets and the United Kingdom.

We may enter into collaborations with other parties for certain markets outside the United States.

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If we fail to raise capital or enter into such other arrangements as, and when needed, we may have to significantly delay, scale back or discontinue the development or commercialization of setmelanotide.

As of March 31, 2026, we had an accumulated deficit of $1.4 billion.

Our net loss was $55.6 million and $49.5 million for the three months ended March 31, 2026 and March 31, 2025.

We expect to continue to incur significant expenses and operating losses for the foreseeable future.

As of June 30, 2026, we had an accumulated deficit of $1.5 billion.

Our net loss was $49.3 million and $46.6 million for the three months ended June 30, 2026 and June 30, 2025.

Our net loss was $104.9 million and $96.1 million for the six months ended June 30, 2026 and June 30, 2025.We expect to continue to incur significant expenses and operating losses for the foreseeable future.

Our expenses may increase in connection with our ongoing activities, as we:

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• continue to operate as a public company.

As of March 31, 2026, our existing cash and cash equivalents and short-term investments were approximately $340.6 million.

We expect that our cash and cash equivalents and short-term investments as of March 31, 2026, will be sufficient to fund our planned operations for at least 24 months.

As of June 30, 2026, our existing cash and cash equivalents and short-term investments were approximately $330.9 million.

We expect that our cash and cash equivalents and short-term investments as of June 30, 2026, will be sufficient to fund our planned operations for at least 24 months.

Financial Operations Overview

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License revenue

In the three months ended March 31, 2025, we recognized a reduction of previously-recognized license revenue of $5.0 million in connection with the termination of our exclusive license agreement with RareStone.

Significant Agreements , to the unaudited condensed consolidated financial statements included under Part I, Item 1 of this Quarterly Report on Form 10-Q.

In the six months ended June 30, 2025, we recognized a reduction of previously-recognized license revenue of $5.0 million in connection with the termination of our exclusive license agreement with RareStone.

See Note 12, Significant Agreements , to the unaudited condensed consolidated financial statements included under Part I, Item 1 of this Quarterly Report on Form 10-Q.

Cost of sales

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The following table summarizes our current research and development expenses:

Three Months Ended

Three months ended June 30, Six months ended June 30,

Research and development summary 2026 2025 2026 2025

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General and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, relating to our full-time employees not involved in R&D or commercial activities.

Other significant costs include rent, information technology, legal fees relating to patent and corporate matters and fees for accounting and consulting services.

Other significant costs

include rent, information technology, legal fees relating to patent and corporate matters and fees for accounting and consulting services.

The following table summarizes our current selling, general and administrative expenses:

Three Months Ended

Three months ended June 30, Six months ended June 30,

Selling, general and administrative summary 2026 2025 2026 2025

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Results of Operations

Comparison of the three months ended March 31, 2026 and 2025

The following table summarizes our results of operations for the three months ended March 31, 2026 and 2025, together with the changes in those items in dollars and as a percentage:

Three Months Ended

March 31, Change

Comparison of the three months ended June 30, 2026 and 2025

The following table summarizes our results of operations for the three months ended June 30, 2026 and 2025, together with the changes in those items in dollars and as a percentage:

Three months ended June 30, Change

2026 2025 $ %

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Product revenue, net $ 71,255 $ 48,502 $ 22,753 47 %

License revenue, net — (5,014) 5,014 (100 %)

Total revenues 71,255 48,502 22,753 47 %

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Product revenue, net .

Product revenue, net increased by $22.4 million to $60.1 million for the three months ended March 31, 2026 from $37.7 million for the three months ended March 31, 2025, an increase of 59%, primarily due to higher volume of product sold both domestically and internationally.

Product revenue, net increased by $22.8 million to $71.3 million for the three months ended June 30, 2026 from $48.5 million for the three months ended June 30, 2025, an increase of 47%, primarily due to higher volume of product sold both domestically and internationally.

The increase was offset by a reduction to product revenue, net of $3.8 million due to an increase in international government rebates, primarily related to growth of sales in France.

We expect our sales of IMCIVREE to continue to increase.

We have achieved market access for IMCIVREE for BBS or POMC and LEPR deficiencies, or both, in more than 25 countries outside the United States, and we continue to collaborate with authorities to achieve access in additional markets.

For the three months ended March 31, 2026, and 2025, a substantial amount of our product revenue, or 61% and 65%, respectively, was generated from sales of our product to patients in the United States.

We have achieved market access or named patient sales for IMCIVREE for BBS or POMC and LEPR deficiencies, or both, in more than 25 countries outside the United States, and we continue to collaborate with authorities to achieve access in additional markets.

For the three months ended June 30, 2026, and 2025, a substantial amount of our product revenue, or 72% and 66%, respectively, was generated from sales of our product to patients in the United States.

Cost of sales.

Cost of sales increased by $3.4 million to $8.9 million for the three months ended June 30, 2026, from $5.5 million for the three months ended June 30, 2025, an increase of 61%, which was driven by an increase in net product revenue in the three months ended June 30, 2026.

Cost of sales is composed of royalty expense due to Ipsen Pharma S.A.S., or Ipsen, on our net product revenue, amortization of our capitalized sales-based milestone payment made to Ipsen upon our first commercial sale in the United States and EU, the cost of product, as well as costs associated with our patient assistance programs.

Specifically, the $3.4 million increase in cost of sales in the three months ended June 30, 2026, from the same period in 2025, was due to $1.1 million of additional royalties due to our growth in net product revenue and $2.3 million attributed to increased product costs associated with higher net product revenue.

We expect cost of sales as a percentage of revenue to continue to be in a range of 10% to 12% in the foreseeable future.

Research and development expense.

Research and development expense increased by $1.1 million to $43.4 million for the three months ended June 30, 2026, from $42.3 million for the three months ended June 30, 2025, an increase of 3%.

The net increase was primarily due to the following:

• an increase of $3.3 million related to personnel costs including $2.2 million in stock-based compensation, and $1.1 million related to salaries, benefits and other compensation costs related to the hiring of additional full-time employees in order to support the growth of our research and development programs,

• an increase of $1.4 million in data analytics primarily related to the company's sponsored genetic testing programs and

• an increase of $0.8 million in pre-clinical expenses due to an increase in CHI-related costs.

The above increases were partially offset by:

• a net decrease of $3.5 million due to phasing of spending associated with chemistry, manufacturing, and controls (CMC) costs for drug formulation and autoinjector development to support our ongoing clinical trials related to RM-718,

• $1.0 million net decrease in clinical trial costs driven primarily by a $3.6 million decrease in clinical trial costs due to the wind-down of various studies including our EMANATE Phase 3, and Phase 3 HO Setmelanotide trial, offset by an increase of $2.0 million related to various Bivamelagon studies.

Selling, general and administrative expense.

Selling, general and administrative expense increased by $21.5 million to $67.4 million for the three months ended June 30, 2026, from $45.9 million for the three months ended June 30, 2025, an increase of 47%.

The increase was primarily due to the following:

• an increase of $18.7 million related to personnel costs including $7.9 million of stock-based compensation, and $10.8 million related to salaries, benefits and other compensation costs related to the hiring of additional full-time employees to support our expanding business operations as well as to establish commercial operations in international regions,

• an increase of $1.9 million related to increased marketing and promotion costs to support continued product revenue growth and our product launch for acquired Hypothalamic Obesity.

Other income (expense), net.

Other expense, net decreased by $0.7 million to $(0.3) million for the three months ended June 30, 2026 from $(1.0) million for the three months ended June 30, 2025.

The decrease was primarily due to the following:

• a decrease of $1.2 million of non-cash interest expense in the three months ended June 30, 2026, associated with the accretion of the LG Chem liability, which was fully settled in July 2025.

The above decrease was partially offset by:

• an increase in other expense of $0.7 million recognized on the change in fair value of the embedded derivative on our deferred royalty obligation.

Comparison of the six months ended June 30, 2026 and 2025

The following table summarizes our results of operations for the six months ended June 30, 2026 and 2025, together with the changes in those items in dollars and as a percentage:

Six Months Ended June 30, Change

2026 2025 $ %

(in thousands)

Statement of Operations Data:

Product revenue, net $ 131,367 $ 86,220 $ 45,147 52 %

License revenue — (5,014) 5,014 (100) %

For the three months ended March 31, 2025, we recognized a reduction of previously-recognized license revenue of $5.0 million in connection with the termination of our exclusive license agreement with RareStone.

Total revenues 131,367 81,206 50,161 62 %

Costs and expenses:

Cost of sales 16,088 9,191 6,897 75 %

Research and development 85,153 79,281 5,872 7 %

Selling, general, and administrative 131,039 85,034 46,005 54 %

Total costs and expenses 232,280 173,506 58,774 34 %

Loss from operations (100,913) (92,300) (8,613) 9 %

Other income (expense), net (3,035) (3,413) 378 (11) %

Loss before income taxes (103,948) (95,713) (8,235) 9 %

Provision for income taxes 989 417 572 137 %

Net loss $ (104,937) $ (96,130) $ (8,807) 9 %

Product revenue, net .

Product revenue, net increased by $45.1 million to $131.4 million for the six months ended June 30, 2026, from $86.2 million for the six months ended June 30, 2025, an increase of 52% primarily due to higher volume of product sold both domestically and internationally.

The increase was offset by a reduction to product revenue, net of $3.8 million due to an increase in international government rebates, primarily related to growth of sales in France.

We expect our sales of IMCIVREE to continue to increase.

We have achieved market access or named patient sales of IMCIVREE for BBS or POMC and LEPR deficiencies, or both, in more than 25 countries outside the United States, and we continue to collaborate with authorities to achieve access in additional markets.

For the six months ended June 30, 2026, and 2025, the majority of our product revenue , or 67% and 65%, respectively, was generated from sales of our product to pharmacies for patients in the United States.

License revenue .

For the six months ended June 30, 2025 , we recognized a reduction of previously-recognized license revenue of $5.0 million in connection with the termination of our exclusive license agreement with RareStone.

See Note 12, Significant Agreements , to the unaudited condensed consolidated financial statements included under Part I, Item 1 of this Quarterly Report on Form 10-Q.

Cost of sales.

Cost of sales increased by $3.5 million to $7.2 million for the three months ended March 31, 2026, from $3.6 million for the three months ended March 31, 2025, an increase of 96%, which was driven by an increase in net product revenue in the three months ended March 31, 2026 .

Specifically, the $3.5 million increase in cost of sales in the three months ended March 31, 2026, from the same period in 2025, was due to $1.1 million of additional royalties due to our growth in net product revenue and $2.4 million attributed to increased product costs associated with higher net product revenue.

Cost of sales increased by $6.9 million to $16.1 million for the six months ended June 30, 2026 from $9.2 million for the six months ended June 30, 2025, an increase of 75%, which was driven by an increase in sales volume and net product revenue in the six months ended June 30, 2026.

Cost of sales is composed of royalty expense due to Ipsen on our net product revenue;

amortization of our capitalized sales-based milestone payment made to Ipsen, upon our first commercial sale in the United States and European Union, the cost of product, as well as costs associated with our patient assistance programs.

Specifically, the $6.9 million increase in cost of sales in the six months ended June 30, 2026, from the same period in 2025 was due to $2.2 million of additional royalties due to our growth in sales and $4.7 million attributed to increased product costs associated with higher sales volume.

We expect cost of sales as a percentage of revenue to continue to be in a range of 10% to 12% in the foreseeable future.

Research and development expense.

Research and development expense increased by $4.8 million to $41.7 million for the three months ended March 31, 2026, from $37.0 million for the three months ended March 31, 2025, an increase of 13%.

Research and development expense increased by $5.9 million to $85.2 million for the six months ended June 30, 2026 from $79.3 million for the six months ended June 30, 2025, an increase of 7%.

The net increase was primarily due to the following:

• an increase of $10.4 million related to personnel costs, including $5.7 million related to salaries, benefits and other compensation costs related to the hiring of additional full-time employees in order to support the growth of our research and development programs, and $4.7 million of stock-based compensation,

• an increase in patents, licenses and other regulatory costs of $0.6 million.

• an increase of $1.2 million in data analytics primarily related to the Company's sponsored genetic testing program,

• an increase of $1.0 million in non-clinical expenses due to an increase in CHI-related costs, and

• an increase of $0.9 million in patents, licenses and other regulatory expenses.

The above increases were partially offset by:

• a net decrease in clinical trial expenses of $1.7 million driven primarily by a decrease in costs due to the wind down or conclusion of various studies including our long-term extension trial, DAYBREAK Phase 2, EMANATE Phase 3, and Phase 3 HO Setmelanotide trial, and

• a net decrease of $1.4 million due to the timing of chemistry, manufacturing, and controls (CMC) costs for drug formulation and autoinjector development to support our ongoing clinical trials.

• a decrease of $4.9 million due to phasing of spending associated with chemistry, manufacturing, and controls (CMC) costs for drug formulation and autoinjector development to support our ongoing RM-718 and bivamelagon trials, and

• a net decrease of $2.6 million in our clinical trial costs due to the completion and wind down of various studies including our long-term extension trial, EMANATE phase 3, HO Phase 3, partially offset by an uptick in Bivamelagon-related clinical trials.

Selling, general and administrative expense.

Selling, general and administrative expense increased by $24.5 million to $63.6 million for the three months ended March 31, 2026, from $39.1 million for the three months ended March 31, 2025, an increase of 63%.

Selling, general and administrative expense increased by $46.0 million to $131.0 million for the six months ended June 30, 2026 from $85.0 million for the six months ended June 30, 2025, an increase of 54%.

The increase was primarily due to the following:

• an increase of $20.1 million related to personnel costs including $7.8 million of stock-based compensation, and $12.3 million related to salaries, benefits and other compensation costs related to the hiring of additional full-time employees to support our expanding business operations as well as to establish commercial operations in international regions, and

• an increase of $3.6 million related to increased marketing and promotion costs to support continued product revenue growth and our product launch for acquired Hypothalamic Obesity.

• an increase of $38.8 million related to personnel costs including $15.7 million of stock-based compensation, and $23.2 million related to salaries, benefits and other compensation costs related to the hiring of additional full-time employees to support our expanding business operations both domestic and internationally,

• an increase of $5.5 million related to increased marketing and promotion costs to support continued revenue growth, and our launch for acquired Hypothalamic Obesity, and

• an increase of $1.8 million in market access and commercial services to support our expanding international business operations.

Other income (expense), net.

Other expense, net increased by $(0.3) million to $(2.7) million for the three months ended March 31, 2026 from $(2.4) million for the three months ended March 31, 2025.

The increases were primarily due to the following:

Other income (expense), net decreased by $0.4 million to $3.0 million for the six months ended June 30, 2026 from $3.4 million of other expense for the six months ended June 30, 2025.

The decrease was primarily due to the following:

• a decrease of $2.3 million of non-cash interest expense in the six months ended June 30, 2026, associated with the accretion of the LG Chem liability, which was paid in July 2025.

The above decrease was partially offset by:

• an increase of $1.0 million for the change in fair value of the embedded derivative of our deferred royalty obligation during as we recognized a loss of $0.6 million during the six months ended June 30, 2026 compared to a gain of $0.4 million during the six months ended June 30, 2025, and

• an increase of $0.6 million in unrealized loss on foreign currency.

• an increase in other expense of $0.3 million recognized on the change in fair value of the embedded derivative on our deferred royalty obligation

• an increase of $0.2 million in interest expense related to our deferred royalty obligation.

These increases were partially offset by:

• a decrease of $1.1 million of non-cash interest expense in the three months ended March 31, 2026, associated with accretion of LG Chem liability, which was paid in July 2025.

Liquidity and Capital Resources

As of March 31, 2026, our cash and cash equivalents and short-term investments were approximately $340.6 million.

The following table provides information regarding our cash flows for the three months ended March 31, 2026 and 2025:

Three Months Ended March 31,

As of June 30, 2026, our cash and cash equivalents and short-term investments were approximately $330.9 million.

The following table provides information regarding our cash flows for the six months ended June 30, 2026 and 2025:

Six Months Ended June 30,

(in thousands)

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The use of cash in all periods resulted primarily from our net loss adjusted for non-cash charges and changes in components of operating assets and liabilities.

Net cash used in operating activities was $44.2 million for the three months ended March 31, 2026 and consisted primarily of a net loss of $55.6 million adjusted for non-cash items of $28.5 million, which consisted of stock-based compensation, non-cash interest expense, non-cash accretion and amortization of short-term investments, depreciation and amortization, non-cash rent expense, the change in the fair value of our embedded derivative asset, and the foreign currency impacts.

Additionally, the change in operating assets and liabilities used net cash of approximately $17.0 million and was primarily driven by net increases in accounts receivable of $8.1 million, primarily due to the increase in product revenue, the change in prepaid expenses and other current assets of $0.5 million, the change in inventory of $3.1 million, the change in deferred revenue of $0.1 million, and the decrease in accounts payable, accrued expenses and other liabilities of $5.6 million.

These net uses of cash were offset by a decrease in other long-term assets of $0.4 million.

Net cash used in operating activities was $40.4 million for the three months ended March 31, 2025 and consisted primarily of a net loss of $49.5 million adjusted for non-cash items of $17.1 million, which consisted of stock-based compensation, non-cash interest expense, non-cash accretion and amortization of short-term investments, depreciation and amortization, rent expense and the change in the fair value of our embedded derivative asset.

The change in operating assets and liabilities used net cash of approximately $7.9 million and was primarily driven by net decreases in accounts payable and accrued expenses of $3.6 million, the change in deferred revenue of $1.3 million, and net increases in prepaids and other current assets of $3.2 million.

These net uses of cash were offset by net increases in other long-term assets of $0.1 million and net increases in accounts receivable and inventory of $0.1 million.

Net cash used in operating activities was $53.3 million for the six months ended June 30, 2026 and consisted primarily of a net loss of $104.9 million adjusted for non-cash items of $58.0 million, which consisted of stock-based compensation, non-cash interest expense, non-cash accretion and amortization of short-term investments, depreciation and amortization, non-cash rent expense, the change in the fair value of our embedded derivative asset, and the foreign currency impacts.

Additionally, the change in operating assets and liabilities used net cash of approximately $6.4 million and was primarily driven by net increases in accounts receivable of $14.3 million, primarily due to the increase in product revenue, the change in inventory of $5.4 million, and the change in deferred revenue of $0.2 million.

These net uses of cash were offset by an increase in accounts payable, accrued expenses and other liabilities of $10.8 million, the change in prepaid expenses and other current assets of $2.3 million, and the change in other long-term assets of $0.4 million.

Net cash used in operating activities was $63.7 million for the six months ended June 30, 2025 and consisted primarily of a net loss of $96.1 million adjusted for non-cash items of $35.9 million, which consisted of stock-based compensation, non-cash interest expense, non-cash accretion and amortization of short-term investments, depreciation and amortization, rent expense, the change in the fair value of our embedded derivative asset, and the change in unrealized gain on foreign currency.

The change in operating assets and liabilities used net cash of approximately $3.4 million and was primarily driven by net increases in prepaid expenses and other current assets of $10.0 million, the change in accounts receivable of $7.1 million, and the change in deferred revenue of $1.3 million.

These net uses of cash were offset by an increase in accounts payable, accrued expenses and other liabilities of $9.6 million, and net decreases in other long-term assets of $5.5 million.

Net cash provided by investing activities

Net cash provided by investing activities was $57.1 million for the three months ending March 31, 2026 and relates to purchases of short-term investments for $42.2 million, offset by gross maturities of short-term investments of $99.3 million.

Net cash provided by investing activities was $24.8 million for the three months ended March 31, 2025 and relates to gross maturities of short-term investments of $58.3 million, offset by purchases of short-term investments for $33.5 million.

Net cash provided by financing activities

Net cash used in financing activities was $5.4 million for the three months ended March 31, 2026, and consisted of proceeds of $7.3 million from the exercise of stock options and the issuance of common stock from our Employee Stock Purchase Plan.

These proceeds were offset by $4.3 million of repayments of our deferred royalty obligation and $8.5 million for the net settlement of equity awards.

Net cash provided by financing activities was $32.5 million for the three months ended March 31, 2025, and consisted of net proceeds of $34.0 million from our ATM equity offering, as well as proceeds of $3.1 million from the exercise of stock options and the issuance of common stock from our Employee Stock Purchase Plan.

Net cash provided by investing activities was $71.1 million for the six months ending June 30, 2026 and relates to purchases of short-term investments for $85.7 million, offset by gross maturities of short-term investments of $156.8 million.

Net cash provided by investing activities was $78.8 million for the six months ended June 30, 2025 and relates to gross maturities of short-term investments of $139.3 million , offset by purchases of short-term investments for $60.5 million .

Net cash (used in) provided by financing activities

Net cash used in financing activities was $6.6 million for the six months ended June 30, 2026, and consisted of $11.2 million of repayments of our deferred royalty obligation and $9.7 million for the net settlement of equity awards.

These uses of cash were offset by proceeds of $14.3 million from the exercise of stock options and the issuance of common stock from our Employee Stock Purchase Plan.

Net cash provided by financing activities was $31.6 million for the six months ended June 30, 2025, and consisted of net proceeds of $34.0 million from our ATM equity offering, as well as proceeds of $6.5 million from the exercise of stock options and the issuance of common stock from our Employee Stock Purchase Plan.

These proceeds were offset by $8.9 million of repayments of our deferred royalty obligation.

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We also expect to incur additional costs associated with operating as a public company.

We expect that our cash and cash equivalents and short-term investments as of March 31, 2026 will be sufficient to fund our planned operations for at least 24 months.

We expect that our cash and cash equivalents and short-term investments as of June 30, 2026 will be sufficient to fund our planned operations for at least 24 months.

Our cash and cash equivalents are maintained at financial institutions in amounts that exceed federally-insured limits.

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Contractual obligations

As of March 31, 2026, there were no other material changes to our principal contractual obligations and commitments as reported in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025.

As previously disclosed in Note 14, Commitment and Contingencies, as of June 30, 2026, we estimate that potential milestone payments of up to $18 million could become payable by the Company during the next 12 months depending on the achievement and timing of specified milestones.

Other than the above, there were no other material changes to our principal contractual obligations and commitments as reported in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025.

Quantitative and Qualitative Disclosures About Market Risk

As of March 31, 2026, there were no material changes to our quantitative and qualitative disclosures about market risks as reported in Part II, Item 7A “Quantitative and Qualitative Disclosures About Market Risks” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025.

As of June 30, 2026, there were no material changes to our quantitative and qualitative disclosures about market risks as reported in Part II, Item 7A “Quantitative and Qualitative Disclosures About Market Risks” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 2. Management’s Discussion and Analysis