RHYTHM PHARMACEUTICALS, INC.

Previous wording

You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q. In addition to historical information, some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and is subject to the “safe harbor” created by those sections. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q, including without limitation statements regarding: the promise or potential of any of our products or product candidates; the marketing, commercialization, and sales of IMCIVREE (setmelanotide), including marketing approval in Japan and the timing thereof; the design, success, cost and timing of our product development activities and clinical trials for setmelanotide, RM-718, bivamelagon, and our other product candidates; our ability to obtain regulatory approval for setmelanotide in further indications, as well as for RM-718, bivamelagon, and our other product candidates; our financial performance, including our expectations regarding our existing cash, operating losses, expenses and sources of future financing; 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; patient enrollments and the timing thereof; the timing of announcements regarding results of clinical trials; our ability to protect our intellectual property; ongoing activities under and our ability to negotiate our collaboration and license agreements, if needed, and the impact of termination; our marketing, commercial sales, revenue generation, and cost of revenue; expectations surrounding our manufacturing arrangements; the impact of the current or future economic conditions on our business and operations and our future financial results; and other statements identified by words such as “anticipates,” “believes,” “could,” “estimates,” “expects,” “intends,” “may,” “might,” “likely,” “plans,” “potential,” “predicts,” “projects,” “seeks,” “should,” “target,” “will,” “would,” or similar expressions and the negatives of those terms are forward-looking statements. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of known and unknown risks, uncertainties, and other important factors, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in such forward-looking statements. We discuss factors that we believe could cause or contribute to these differenc…

Excerpt shortened here; the source contains the full paragraph.

Current wording

You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q. In addition to historical information, some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and is subject to the “safe harbor” created by those sections. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q, including without limitation statements regarding: the promise or potential of any of our products or product candidates; the marketing, commercialization, and sales of IMCIVREE (setmelanotide); the design, success, cost and timing of our product development activities and clinical trials for setmelanotide, RM-718, bivamelagon, and our other product candidates; our ability to obtain regulatory approval for setmelanotide in further indications, as well as for RM-718, bivamelagon, and our other product candidates; our financial performance, including our expectations regarding our existing cash, operating losses, expenses and sources of future financing; 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; patient enrollments and the timing thereof; the timing of announcements regarding results of clinical trials; our ability to protect our intellectual property; ongoing activities under and our ability to negotiate our collaboration and license agreements, if needed, and the impact of termination; our marketing, commercial sales, revenue generation, and cost of revenue; expectations surrounding our manufacturing arrangements; the impact of the current or future economic conditions on our business and operations and our future financial results; and other statements identified by words such as “anticipates,” “believes,” “could,” “estimates,” “expects,” “intends,” “may,” “might,” “likely,” “plans,” “potential,” “predicts,” “projects,” “seeks,” “should,” “target,” “will,” “would,” or similar expressions and the negatives of those terms are forward-looking statements. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of known and unknown risks, uncertainties, and other important factors, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in such forward-looking statements. We discuss factors that we believe could cause or contribute to these differences below and elsewhere in this report, including but not limit…

Excerpt shortened here; the source contains the full paragraph.

Previous wording

We are a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases. We are focused on advancing our melanocortin-4 receptor (MC4R) agonists, including our lead asset, IMCIVREE® (setmelanotide), as precision medicines designed to treat hyperphagia and severe obesity caused by MC4R pathway diseases. While obesity affects hundreds of millions of people worldwide, we are advancing therapies for a subset of individuals who have hyperphagia, a pathological, insatiable hunger and impaired satiety accompanied by persistent and abnormal food-seeking behaviors, decreased energy expenditure and severe obesity due to diseases such as acquired or congenital hypothalamic obesity (HO), Bardet-Biedl syndrome (BBS) or other diseases caused by impaired MC4R pathway signaling. The MC4R pathway is a neuroendocrine pathway in the brain that is responsible for regulating hunger, caloric intake and energy expenditure, which consequently affect body weight. IMCIVREE, an MC4R agonist for which we hold worldwide rights, is the first-ever therapy developed for patients with certain rare diseases that is approved or authorized in the United States, European Union (EU), United Kingdom, Canada and other countries and regions.

Current wording

We are a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases. We are focused on advancing our melanocortin-4 receptor (MC4R) agonists, including our lead asset, IMCIVREE® (setmelanotide), as precision medicines designed to treat hyperphagia and severe obesity caused by MC4R pathway diseases. While obesity affects hundreds of millions of people worldwide, we are advancing therapies for a subset of individuals who have hyperphagia, a pathological, insatiable hunger and impaired satiety accompanied by persistent and abnormal food-seeking behaviors, decreased energy expenditure and severe obesity due to diseases such as acquired or congenital hypothalamic obesity, Prader-Willi syndrome (PWS), Bardet-Biedl syndrome (BBS) or other diseases caused by impaired MC4R pathway signaling. The MC4R pathway is a neuroendocrine pathway in the brain that is responsible for regulating hunger, caloric intake and energy expenditure, which consequently affect body weight. IMCIVREE, an MC4R agonist for which we hold worldwide rights, is the first-ever therapy developed for patients with certain rare diseases that is approved or authorized in the United States, European Union (EU), United Kingdom, Canada and other countries and regions.

Previous wording

IMCIVREE is approved by the U.S. 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 hungerin adults and children 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment, andfor 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.

Current wording

IMCIVREE is approved by the U.S. 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 hypothalamic obesity (“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 HO 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.

Previous wording

IMCIVREE’s label expansion to treat patients living with acquired HO was approved by the U.S. FDA on March 19, 2026, and a similar expansion of its marketing authorization was approved by the European Commission on April 30, 2026, making it the first approved therapy in the United States and the first approved therapy in Europe for patients with this rare disease. These approvals were supported by the positive pivotal Phase 3 TRANSCEND trial of setmelanotide in 142 patients with acquired HO. The global study met its primary endpoint, with a statistically significant -18.4% placebo-adjusted reduction in body mass index (BMI). For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=94) achieved a -15.8% reduction compared with a +2.6% increase among patients on placebo (n=48) at 52 weeks (p<0.0001). Setmelanotide was generally well tolerated in the Phase 3 trial. The most common adverse events (affecting >20% of participants) were skin hyperpigmentation, nausea, vomiting and headache. 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 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.

Current wording

IMCIVREE’s label expansion to treat patients living with acquired HO was approved by the U.S. FDA on March 19, 2026, and a similar expansion of its marketing authorization was approved by the European Commission on April 30, 2026. Japan's Ministry of Health, Labour and Welfare (MHLW)also approved IMCIVREE to treat patients with acquired HOon August 24, 2026, making it the first approved therapy in the United States, Europe and Japan for patients with this rare disease. These approvals were supported by the positive pivotal Phase 3 TRANSCEND trial of setmelanotide in 142 patients with acquired HO. The global study met its primary endpoint, with a statistically significant -18.4% placebo-adjusted reduction in body mass index (BMI). For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=94) achieved a -15.8% reduction compared with a +2.6% increase among patients on placebo (n=48) at 52 weeks (p<0.0001). Setmelanotide was generally well tolerated in the Phase 3 trial. The most common adverse events (affecting >20% of participants) were skin hyperpigmentation, nausea, vomiting and headache.

Previous wording

Acquired hypothalamic obesity is a rare form of obesity that occurs following damage to the hypothalamic region of the brain. This disease most frequently follows the growth or surgical removal of craniopharyngioma, astrocytoma or other rare brain tumors. Additional causes of injury may include traumatic brain injury, stroke, or inflammation. Patients experience accelerated weight gain, a reduction in energy expenditure, and hyperphagia (a chronic pathological condition characterized by insatiable hunger, impaired satiety, and persistent abnormal food-seeking behaviors) leading to accelerated onset of severe obesity. We estimate there are approximately 10,000 people living with hypothalamic obesity in the U.S., 5,000 to 8,000 people living with hypothalamic obesity in Japan, and approximately 10,000 people living with hypothalamic obesity in the E.U.

Current wording

Acquired HO is a rare form of obesity that occurs following damage to the hypothalamic region of the brain. This disease most frequently follows the growth or surgical removal of craniopharyngioma, astrocytoma or other rare brain tumors. Additional causes of injury may include traumatic brain injury, stroke, or inflammation. Patients experience accelerated weight gain, a reduction in energy expenditure, and hyperphagia (a chronic pathological condition characterized by insatiable hunger, impaired satiety, and persistent abnormal food-seeking behaviors) leading to accelerated onset of severe obesity. We estimate there are approximately 10,000 people living with hypothalamic obesity in the U.S., 5,000 to 8,000 people living with hypothalamic obesity in Japan, and approximately 10,000 people living with hypothalamic obesity in the E.U.

Previous wording

In addition to our commercial efforts and inclusive of late-stage development efforts, we are advancing what we believe is the most comprehensive clinical research and development program ever initiated in MC4R pathway diseases, with multiple ongoing and planned clinical trials. Our MC4R pathway program is designed to expand the total number of patients who we believe would benefit from setmelanotide therapy or advance one of our other drug candidates, including RM-718, which is designed to be a more selective MC4R agonist with weekly administration, and bivamelagon, an investigational oral small molecule MC4R agonist in Phase 2 clinical trials. As mentioned above, our Phase 3 trial of setmelanotide in patients with acquired HO met the primary and key secondary endpoints, and we have initiated an additional, independent substudy in patients with congenital hypothalamic obesity as part of that trial.

Current wording

In addition to our commercial efforts and inclusive of late-stage development efforts, we are advancing what we believe is the most comprehensive clinical research and development program ever initiated in MC4R pathway diseases, with multiple ongoing and planned clinical trials. Our MC4R pathway program is designed to expand the total number of patients who we believe would benefit from setmelanotide therapy or advance one of our other drug candidates, including RM-718, which is designed to be a more selective MC4R agonist with weekly administration, and bivamelagon, an investigational oral small molecule MC4R agonist which we are preparing to evaluate further in a Phase 3 clinical trial. As mentioned above, our Phase 3 trial of setmelanotide in patients with acquired HO met the primary and key secondary endpoints, and we have initiated an additional, independent substudy in patients with congenital hypothalamic obesity as part of that trial.

Previous wording

We are advancing our next-generation MC4R agonists in clinical trials. 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 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.

Current wording

We are advancing our next-generation MC4R agonists in clinical trials. 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 have also completed enrollment in Part C of the Phase 1 trial evaluating the weekly RM-718 in patients with acquired HO and announced preliminary, promising data on August 4, 2026.

Previous wording

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. 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 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.

Current wording

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. 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 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 1/2 trial evaluating RM-718 in PWS.

Previous wording

•On Aug. 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;

Current wording

•On November 3, 2026, we announced that more than 700 patient start forms for IMCIVREE for acquired HO had been received from more than 500 prescribers as of September 30, 2026, since the approval by the U.S. Food and Drug Administration (FDA) on March 19, 2026;

Previous wording

•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.

Current wording

•Revenue from global sales of IMCIVREE was $92.1 million for the third quarter of 2026, an increase of 29% on a sequential basis from the second quarter of 2026. The number of patients on reimbursed therapy globally increased by 20% as compared to the second quarter of 2026.

Previous wording

◦U.S.: 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).

Current wording

◦U.S.: Revenue of $67.5 million, or 73% of product revenue, was generated in the United States in the third quarter of 2026, an increase of $16.5 million or 32% compared to the second 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).

Previous wording

◦Ex-U.S.: 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.

Current wording

◦Ex-U.S.: Revenue of $24.7 million, or 27% of product revenue, was generated outside the United States, a sequential increase of $4.4 million or 22% compared to the second quarter of 2026. The number of reimbursed patients on IMCIVREE continued to increase in the third quarter of 2026;

Previous wording

◦Efficacy and safety of setmelanotide in pediatric patients with acquired hypothalamic obesity: final phase 3 trial results;

Current wording

◦Efficacy and Safety of Setmelanotide in Acquired Hypothalamic Obesity: Final Phase 3 Trial Results;

Previous wording

•On Aug. 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:

Current wording

•On September 10, 2026, the Company announced the presentation of six abstracts, including three oral presentations and three poster presentations, at the 64th Annual European Society for Paediatric Endocrinology (ESPE) Meeting. Presentations included;

Previous wording

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

Current wording

◦Long-term weight outcomes with setmelanotide in paediatric patients with Bardet-Biedl syndrome (BBS), POMC deficiency and LEPR deficiency; and

Previous wording

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

Current wording

•Complete enrollment in the setmelanotide substudy in congenital HO in 2027;

Previous wording

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

Current wording

•Begin country-level launches of IMCIVREE for acquired HO in Europe beginning with Germany in 2027 and

Previous wording

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. 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 may enter into arrangements with other parties for certain markets outside the United States. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms, or at all. 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.

Current wording

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. Since our inception, we have raised funds through the issuance of common and preferred equity, the sale of assets, our Revenue Interest Financing Agreement, or RIFA, and as of November 2, 2026 afive-year term loan facility providing access to up to $750 million of capital. 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 may enter into arrangements with other parties for certain markets outside the United States. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms, or at all. 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.

Previous wording

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:

Current wording

As of September 30, 2026, we had an accumulated deficit of $1.5 billion. Our net loss was $41.4 million and $52.9 million for the three months ended September 30, 2026 and September 30, 2025. Our net loss was $146.3 million and $149.0 million for the nine months ended September 30, 2026 and September 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:

Previous wording

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.

Current wording

As of September 30, 2026, our existing cash and cash equivalents and short-term investments were approximately $320.9 million. We expect that our cash and cash equivalents and short-term investments as of September 30, 2026, will be sufficient to fund our planned operations for at least 24 months.

Previous wording

To date, we have generated approximately $553.9 million in product revenue. Our lead product candidate, IMCIVREE, was approved by the FDA in November 2020 for chronic weight management in adult and pediatric patients six years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE became commercially available in the United States in the first quarter of 2021. We recorded our first sales of IMCIVREE in the United States in March 2021 and we made our first sales in the EU in March 2022 under the paid early access program. IMCIVREE was approved by the FDA and the EC in adult and pediatric patients six years of age and older with obesity due to BBS in June and September 2022, respectively.

Current wording

To date, we have generated approximately $646.0 million in product revenue. Our lead product candidate, IMCIVREE, was approved by the FDA in November 2020 for chronic weight management in adult and pediatric patients six years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing. IMCIVREE became commercially available in the United States in the first quarter of 2021. We recorded our first sales of IMCIVREE in the United States in March 2021 and we made our first sales in the EU in March 2022 under the paid early access program. IMCIVREE was approved by the FDA and the EC in adult and pediatric patients six years of age and older with obesity due to BBS in June and September 2022, respectively.

Previous wording

In March 2026, IMCIVREE was approved by the FDA to reduce excess body weight and maintain reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity (aHO).

Current wording

In March 2026, IMCIVREE was approved by the FDA to reduce excess body weight and maintain reduction long term in adult and pediatric patients aged 4 years and older with acquired HO and a similar expansion of its marketing authorization was approved by the European Commission on April 30, 2026. Japan's Ministry of Health, Labour and Welfare (MHLW) also approved IMCIVREE to treat patients with acquired HO on August 24, 2026. Following these initial approvals, sales of IMCIVREE have grown, and we expect will continue to grow.

Previous wording

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.

Current wording

In the nine months ended September 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.

Previous wording

Product revenue, net. 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 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.

Current wording

Product revenue, net. Product revenue, net increased by $40.8 million to $92.1 million for the three months ended September 30, 2026 from $51.3 million for the three months ended September 30, 2025, an increase of 80%, primarily due to higher volume of product sold both domestically and internationally. We expect our sales of IMCIVREE to continue to increase. We have also 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. Additionally, we experienced continued revenue growth in the third quarter of 2026 for acquired HO in the United States, which was approved in March 2026. We expect sales of IMCIVREE for acquired HO to increase and we also continue to collaborate with authorities to achieve access in additional markets for acquired HO. For the three months ended September 30, 2026, and 2025, a substantial amount of our product revenue, or 73% and 74%, respectively, was generated from sales of our product to patients in the United States.

Previous wording

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.

Current wording

Cost of sales. Cost of sales increased by $6.7 million to $12.2 million for the three months ended September 30, 2026, from $5.5 million for the three months ended September 30, 2025, an increase of 121%, which was driven by an increase in net product revenue in the three months ended September 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 $6.7 million increase in cost of sales in the three months ended September 30, 2026, from the same period in 2025, was due to $2.1 million of additional royalties due to our growth in net product revenue and $4.6 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.

Previous wording

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:

Current wording

Research and development expense. Research and development expense increased by $6.5 million to $52.5 million for the three months ended September 30, 2026, from $46.0 million for the three months ended September 30, 2025, an increase of 14%. The net increase was primarily due to the following:

Previous wording

•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,

Current wording

•an increase of $1.0 million related to personnel costs including $0.7 million in stock-based compensation, and $0.3 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,

Previous wording

•$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.

Current wording

•$1.6 million net increase in clinical trial costs driven by an increase in costs related primarily to various Bivamelagon and RM-718 studies, partially offset by a decrease in clinical trial costs due to the wind-down of various studies including our EMANATE Phase 3 and Phase 3 HO Setmelanotide trials,

Previous wording

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:

Current wording

Selling, general and administrative expense. Selling, general and administrative expense increased by $14.3 million to $66.8 million for the three months ended September 30, 2026, from $52.4 million for the three months ended September 30, 2025, an increase of 27%. The increase was primarily due to the following:

Previous wording

•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,

Current wording

•an increase of $13.6 million related to personnel costs including $5.5 million of stock-based compensation, and $8.1 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 domestically and internationally,

Previous wording

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:

Current wording

Other income (expense), net. Other expense, net increased by $(1.3) million to $(1.5) million for the three months ended September 30, 2026 from $(0.1) million for the three months ended September 30, 2025. The increase was primarily due to the following:

Previous wording

•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.

Current wording

•a decrease in other income of $0.6 million recognized on the change in fair value of the embedded derivative on our deferred royalty obligation as we recognized a $0.4 million gain on the change in value during the three months ending September 30, 2025, and we recognized a ($0.2) million loss on the change in value during the three months ending September 30, 2026.

Previous wording

•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.

Current wording

•an increase of $0.2 million of non-cash interest expense in the three months ended September 30, 2026, associated with the deferred royalty obligation.

Previous wording

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:

Current wording

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

Previous wording

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.

Current wording

Product revenue, net. Product revenue, net increased by $86.0 million to $223.5 million for the nine months ended September 30, 2026, from $137.5 million for the nine months ended September 30, 2025, an increase of 63% primarily due to higher volume of product sold both domestically and internationally. We expect our sales of IMCIVREE to continue to increase. We also 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. Additionally, we experienced continued revenue growth in the third quarter of 2026 was for acquired HO in the United States, which was approved in March 2026. We expect sales of IMCIVREE for acquired HO to increase and we also continue to collaborate with authorities to achieve access in additional markets for acquired HO. For the nine months ended September 30, 2026, and 2025, the majority of our product revenue, or 70% and 69%, respectively, was generated from sales of our product to pharmacies for patients in the United States.

Previous wording

License revenue. For thesix 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.

Current wording

License revenue. For the nine months ended September 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.

Previous wording

Cost of sales. 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.

Current wording

Cost of sales. Cost of sales increased by $13.6 million to $28.3 million for the nine months ended September 30, 2026 from $14.7 million for the nine months ended September 30, 2025, an increase of 92%, which was driven by an increase in sales volume and net product revenue in the nine months ended September 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 $13.6 million increase in cost of sales in the nine months ended September 30, 2026 from the same period in 2025 was due to $4.3 million of additional royalties due to our growth in sales and the remaining $9.3 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.

Previous wording

Research and development expense. 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:

Current wording

Research and development expense. Research and development expense increased by $12.3 million to $137.6 million for the nine months ended September 30, 2026 from $125.3 million for the nine months ended September 30, 2025, an increase of 10%. The net increase was primarily due to the following:

Previous wording

•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,

Current wording

•an increase of $11.6 million related to personnel costs, including $6.1 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, and $5.5 million of stock-based compensation,

Previous wording

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

Current wording

•an increase of $1.5 million in data analytics primarily related to the Company's sponsored genetic testing program as well as an increase in costs related to global medical affairs,