What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-05-05 compared with 2025-11-04 · 150 added, 208 removed, 180 unchanged (67% of the section changed)

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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 (including PDUFA goal dates and Type II variation submissions);

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;

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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 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

The European Commission (EC) and the United Kingdom’s Medicines & Healthcare Products Regulatory Agency

(MHRA) have 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.

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.

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

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 due to infection.

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 severe obesity within six to 12 months following tumor resection or other injury.

On April 7, 2025, we announced positive topline results from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for the treatment of acquired hypothalamic obesity.

The global trial, which we believe is the largest and longest placebo-controlled trial to evaluate a therapy for patients with acquired hypothalamic obesity, met its primary endpoint with a statistically significant and highly clinically meaningful reduction in body mass index (BMI) with setmelanotide in both adult and pediatric patients versus placebo.

No new safety signals with setmelanotide were observed, in line with setmelanotide’s well-established and well-understood safety profile.

We completed applications to seek regulatory approval for setmelanotide as a treatment for acquired hypothalamic obesity in the United States and Europe in the third quarter of 2025.

On August 20, 2025, we announced that the U.S.

FDA accepted for filing our supplemental New Drug Application (sNDA) for setmelanotide seeking approval for the treatment of conditions associated with hypothalamic obesity.

The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2025.

Additionally, the European Medicines Agency (EMA) confirmed validation or the Type II variation submissions to the Marketing Authorization Application (MAA) for setmelanotide.

The application review began on August 16, 2025, by the Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission (EC) regarding potential approval.

We also anticipate reading out data from a 12-patient cohort in Japan in the first quarter of 2026 which we believe, if successful, could support registration of setmelanotide in Japan for this disease.

With these planned submissions, we believe setmelanotide has the potential to become the first-ever approved therapy for patients with acquired hypothalamic obesity.

Additional causes of injury may include traumatic brain injury, stroke, or inflammation.

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.

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 from one of our new drug candidates, RM-718, which is designed to be a more selective MC4R agonist with weekly administration, or 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 hypothalamic obesity 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.

We are advancing 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 hypothalamic obesity.

We 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.

Our Phase 3 EMANATE trial, comprising four independent substudies evaluating setmelanotide in genetically caused MC4R pathway diseases is ongoing, and we completed our Phase 2 DAYBREAK trial evaluating setmelanotide in additional genetic indications in 2024.

We are leveraging what we believe is the largest known DNA database focused on obesity - with approximately 100,000 sequencing samples as of December 31, 2024 - to improve the understanding, diagnosis and care of people living

with severe obesity due to certain variants in genes associated with the MC4R pathway.

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 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.

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 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 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.

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.

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

We continue to leverage what we believe is the largest known DNA database focused on obesity - with approximately 120,000 sequencing samples as of December 31, 2025 - to improve the understanding, diagnosis and care of people living with severe obesity due to certain variants in genes associated with the MC4R pathway.

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.

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

patients in initial FDA-approved indications, including obesity due to biallelic POMC, PCSK1 or LEPR deficiencies, and BBS.

Our epidemiology estimates for the two more prevalent indications being studied in our Phase 3 EMANATE trial (SH2B1 and POMC/PCSK1) suggest that approximately 29,000 U.S.

patients in setmelanotide's initial FDA-approved indications, including obesity due to biallelic POMC, PCSK1 or LEPR deficiencies, and BBS.

Our epidemiology estimates for the two more prevalent indications studied in our Phase 3 EMANATE trial (SRC1 deficiency and POMC/PCSK1 insufficiency) suggest that approximately 26,000 U.S.

patients with one of these genetically driven obesities have the potential to respond well to setmelanotide.

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We believe that all these patients face similar challenges as other patients with rare diseases, namely lack of awareness, resources, tests, tools and, especially, therapeutic options.

Additional recent corporate and commercial updates include:

On November 4, 2025, we announced net product revenue from global sales of IMCIVREE® (setmelanotide) of $51.3 million for the third quarter of 2025, an increase of 6% percent on a sequential basis from the second quarter of 2025, primarily driven by sales of IMCIVREE for the treatment of patients with BBS.

In the third quarter of 2025, revenue of $38.2 million, or 74% of product revenue, was generated in the United States, an increase of 19% on a sequential basis.

Revenue of $13.1 million, or 26% of product revenue, was generated outside the United States, a sequential decrease of $3.4 million or 21%.

The decrease in ex-U.S.

revenue was primarily due to an agreement reached with the French Economic Committee for Health Products (CEPS) on final reimbursement pricing for IMCIVREE for BBS and POMC and LEPR deficiencies.

This resulted in a change to the Company’s price estimate associated with the paid, early-access program that began in 2022, reducing product revenue by approximately $3.2 million in the third quarter of 2025, of which approximately $1.7 million related to periods prior to 2025.

revenue also was affected modestly by European seasonality and variability in ordering patterns from named patient sales in certain countries.

On July 11, 2025, we closed a public offering of 2,367,647 shares of its common stock at a public offering price of $85 per share, resulting in net proceeds of approximately $188.7 million, net of underwriting discounts and commissions, and as well as other offering expenses incurred by the Company.

Additional recent clinical and regulatory updates include:

On November 4, 2025, we announced that four abstracts have been accepted for presentation – one oral presentation and three posters – at the Obesity Society’s Annual Meeting at ObesityWeek 2025 this week in Atlanta, GA:

“Efficacy of Setmelanotide in Patients with Acquired HO Previously or Concurrently on GLP-1 Therapy," an oral presentation by Christian Roth, M.D., Seattle Children’s Research Institute, Thursday, Nov.

6, 9:00–9:15 a.m.;

“Cardiometabolic Results from a Phase 3 Trial of Setmelanotide in Acquired Hypothalamic Obesity,” a poster presentation by Jill Garrison, Director, Medical Affairs, Rhythm Pharmaceuticals, Nov.

4, 7:30–8:30 p.m.;

“Patient- and Caregiver-Reported Experience with Acquired Hypothalamic Obesity in the TRANSCEND Trial,” a poster presentation by Dr.

4, 7:30–8:30 p.m.;

“Hyperphagia and the Identification of Genetic Variants in Patients with Early-Onset Obesity,” a poster presentation by Roohi Kharofa, M.D., Cincinnati Children’s Hospital Medical Center, Nov.

5, 2:30–3:30 p.m.

In September, researchers at the University Hospital Essen published results from a prospective, observational study that showed setmelanotide treatment was associated with improvement in measures of metabolic dysfunction-associated steatotic liver disease (MASLD) and kidney function in patients with BBS in the Journal of Clinical Endocrinology & Metabolism.

All patients (N=26) in this study had MASLD at baseline and after six months of setmelanotide treatment, more than 80% of them achieved either resolution of MASLD or stabilization at grade S1.

On August 5, 2025, we announced that we enrolled the first patient with hypothalamic obesity in Part C of our Phase 1 trial evaluating RM-718, a weekly-administered investigational MC4R agonist.

On July 12, 2025, at the Endocrine Society’s Annual Meeting, data from our pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in acquired hypothalamic obesity, the largest randomized, placebo-controlled trial in acquired hypothalamic obesity to date, were delivered in an oral presentation.

Highlights of the presentation included:

● -19.8% placebo-adjusted difference in BMI reduction (N=120);

Statistically significant BMI reductions following setmelanotide treatment were consistently observed across subgroups stratified by age (<12, 12 to 17, <18, and 18 years and older;

ranging from -15.6% to -17.2%) and by sex (-16.3% female;

On July 9, 2025, we announced bivamelagon achieved statistically significant and clinically meaningful BMI reductions at 14 weeks of treatment in our Phase 2 trial in patients with acquired hypothalamic obesity, including:

● -9.3% BMI reduction from baseline in the 600mg cohort (n=8) (p-value=0.0004);

● -7.7% BMI reduction from baseline in the 400mg cohort (n=7) (p-value=0.0002);

Post-hoc analyses showing bivamelagon demonstrated BMI reductions consistent with BMI reductions achieved with setmelanotide therapy as observed in similar patient populations at comparable dosing durations;

Safety and tolerability results were consistent with MC4R agonism and mechanism of action during the placebo-controlled portion of the trial.

We currently expect to achieve the following, near-term milestones:

● Disclose preliminary results from our Phase 2 trial evaluating setmelanotide in Prader-Willi syndrome in the fourth quarter of 2025;

● Receive a decision from the FDA on our sNDA (anticipated to be received by the PDUFA goal date of December 20, 2025) for setmelanotide in conditions associated with acquired hypothalamic obesity;

● Complete enrollment in the Phase 1, Part C trial evaluating the weekly, MC4R agonist RM-718 in patients with acquired hypothalamic obesity in the first quarter of 2026;

● Announce topline data in the 12-patient Japanese cohort of the setmelanotide Phase 3 trial in acquired hypothalamic obesity in the first quarter of 2026;

● Announce topline data in the Phase 3 EMANATE trial evaluating setmelanotide in genetically caused MC4R pathway diseases in the first quarter of 2026;

● Complete enrollment in the setmelanotide Phase 3 trial substudy in congenital hypothalamic obesity in the first half of 2026;

● Pending further feedback from U.S and European regulatory agencies, initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired hypothalamic obesity in 2026.

IMCIVREE first became commercially available to patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency in the U.S.

in the first quarter of 2021 and patients 6 years of age and older with obesity due to BBS during June 2022.

Following marketing authorizations in the EU, Great Britain and Canada, as well as expanded

labels in the U.S., the EU and Great Britain to make IMCIVREE available to patients as young as 2 years of age, we are continuing to pursue a country-by-country strategy to establish market access and reimbursement for IMCIVREE in additional countries.

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.

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

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

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.

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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.

To date, we have not generated sufficient cash flow from product sales and have financed our operations primarily through the proceeds received from our sales of common and preferred stock, royalty interest financing, asset sales, as well as capital contributions from the former parent company, Rhythm Holdings LLC.

From August 2015 through August 2017, we raised aggregate net proceeds of $80.8 million through our issuance of Convertible Preferred Stock.

Since our initial public offering, or IPO, on October 10, 2017, through our underwritten follow-on offerings and through our ATM program through September 30, 2025, we have raised aggregate net proceeds of approximately $1,053.4 million through the issuance of our common stock after deducting underwriting discounts, commissions and offering related transaction costs.

This includes the follow-on offering that we closed on July 11, 2025, for net proceeds of $188.7 million, net of $12.6 million of certain costs including underwriting discounts and commissions as well as other offering expenses incurred by the Company, for total share issuance of 2,367,647.

We also received $100.0 million from the sale of our Rare Pediatric Disease Priority Review Voucher (“PRV”), to Alexion Pharmaceuticals, Inc.

in February 2021.

In June 2022, we entered into the Revenue Interest Financing Agreement (“RIFA”), with entities managed by HealthCare Royalty Partners, collectively referred to as the Investors, and through September 30, 2025 have received cumulative proceeds of $96.7 million, net of certain transaction costs.

On April 1, 2024, we entered into an Investment Agreement with certain affiliates of Perceptive Advisors LLC, or Perceptive, and certain other investors, relating to the issuance and sale of 150,000 shares of a new series of the Company’s Series A Convertible Preferred Stock, par value $0.001 per share, titled the “Series A Convertible Preferred Stock”, or the Convertible Preferred Stock, for an aggregate purchase price of $150.0 million, or $1,000 per share (as disclosed in Note 9, Series A Preferred Stock ).

We received $147.8 million in net proceeds under the Investment Agreement.

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

Since our inception, we have raised funds through the issuance of common and preferred equity, the sale of assets and a Revenue Interest Financing Agreement, or RIFA.

We expect 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.

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As of September 30, 2025 we had an accumulated deficit of $1.3 billion.

Our net loss was $52.9 million and $43.6 million for the three months ended September 30, 2025 and September 30, 2024.

Our net loss was $149.0 million and $217.3 million for the nine months ended September 30, 2025 and September 30, 2024.

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 collaborations 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.

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.

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• continue to operate as a public company.

As of September 30, 2025, our existing cash and cash equivalents and short-term investments were approximately $416.1 million.

We expect that our cash and cash equivalents and short-term investments as of September 30, 2025, will be sufficient to fund our planned operations for at least 24 months.

On October 1, 2025, the U.S.

government shut down and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical government employees and stop critical activities.

If a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business.

It could also our ability to access the public markets and obtain necessary capital in order to fund our operations.

See Risk Factors—Risks Related to Regulatory Approval and Marketing of Setmelanotide and Other Legal and Compliance Matters and Disruptions at the FDA, including those caused by changing presidential administrations and related priorities, funding shortages staffing or other resource limitations or global health concerns could hinder their ability to hire, retain or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved or commercialized in a timely manner or at all, which could negatively impact our business.

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.

Financial Operations Overview

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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 a paid early access program.

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.

In 2024, IMCIVREE was approved by the FDA to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to BBS or POMC, PCSK1, or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 , or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

Also in 2024, the EC and MHRA 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.

We expect our sales of IMCIVREE will continue to grow as we identify and treat more patients with this disease and obtain reimbursement throughout the international markets in which we operate, however, we cannot estimate or predict with certainty the rates at which sales in any of our markets will grow and whether such sales will grow at a higher or lower pace as compared to sales in the United States.

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).

Following these initial approvals, sales of IMCIVREE have grown, and we expect will continue to grow.

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.

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.

Cost of sales

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Most of the manufacturing costs have been recorded as research and development expenses in prior periods.

We expect cost of sales to increase in 2025 as we continue to support our revenue growth.

We expect cost of sales to increase in 2026 as we continue to sell inventory that is produced after we began capitalizing manufacturing costs for IMCIVREE commercial inventory.

Research and development expenses

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• facilities, depreciation, and other expenses, which include rent and maintenance of facilities, insurance and other operating costs.

● acquired in process research and development costs associated with the acquisition of LG Chem, Ltd.’s, or LGC’s proprietary compound bivamelagon in the three months ended March 31, 2024.

We expense research and development costs to operations as incurred.

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Three Months Ended

Nine Months Ended

September 30,

Research and development summary 2026 2025

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Selling, general and administrative expenses

Selling expenses consist of professional fees related to preparation for the commercialization of setmelanotide, as well as salaries and related benefits for commercial employees, including stock-based compensation.

Selling expenses consist of professional fees related to preparation for the continued commercialization of setmelanotide, as well as salaries and related benefits for commercial employees, including stock-based compensation.

As we further implement and execute our commercialization plans to market setmelanotide in new territories and as we explore new collaborations to develop and commercialize setmelanotide, we anticipate that these expenses will materially increase.

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, 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

Nine Months Ended

September 30,

Selling, general and administrative summary 2026 2025

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Results of Operations

Comparison of the three months ended September 30, 2025 and 2024

The following table summarizes our results of operations for the three months ended September 30, 2025 and 2024, together with the changes in those items in dollars and as a percentage:

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

September 30,

(in thousands)

Statement of Operations Data:

Product revenue, net

Costs and expenses:

Cost of sales

Research and development

Selling, general, and administrative

Total costs and expenses

Loss from operations

Other income (expense), net

Loss before income taxes

Provision for income taxes

Product revenue, net .

Product revenue, net increased by $18.0 million to $51.3 million for the three months ended September 30, 2025 from $33.3 million for the three months ended September 30, 2024, an increase of 54%, primarily due to an increase in sales volume.

Additionally, in the third quarter of 2025 we reached an agreement with French authorities on a final reimbursement price for IMCIVREE sales in France, which resulted in an adjustment of $3.2 million reduction to product revenue, net.

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 three months ended September 30, 2025, and 2024, the majority of our product revenue, or 74% and 68%, respectively, was generated from sales of our product to pharmacies for patients in the United States.

Cost of sales.

Cost of sales increased by $1.7 million to $5.5 million for the three months ended September 30, 2025, from $3.8 million for the three months ended September 30, 2024, an increase of 44%, which was driven by an increase in sales volume and net product revenue in the three months ended September 30, 2025 .

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 European Union, the cost of product, as well as costs associated with our patient assistance programs.

Specifically, the $1.7 million increase in cost of sales in the three months ended September 30, 2025, from the same period in 2024, was due to $0.9 million of additional royalties due to our growth in sales and $0.8 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 $8.1 million to $46.0 million for the three months ended September 30, 2025, from $37.9 million for the three months ended September 30, 2024, an increase of 21%.

The net increase was primarily due to the following:

● an increase of $8.0 million related to personnel costs including $4.6 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 $3.4 million of stock-based compensation, and

● an increase of $1.2 million for regulatory costs to support our research and development programs associated with Hypothalamic Obesity.

The above increases were partially offset by:

● a net decrease in clinical trial expenses of $0.7 million driven by a decrease in costs of $4.6 million due to the wind down or conclusion of various studies including our long-term extension trial, DAYBREAK phase 2, EMANATE phase 3, and RM-718 parts A and B, offset by an increase in clinical trial expenses of $3.9 million for ongoing work in our bivamelagon phase 2 and Prader Willi syndrome phase 1 clinical trials.

Selling, general and administrative expense.

Selling, general and administrative expense increased by $17.0 million to $52.4 million for the three months ended September 30, 2025, from $35.4 million for the three months ended September 30, 2024, an increase of 48%.

The increase was primarily due to the following:

● an increase of $14.0 million related to personnel costs including $4.4 million of stock-based compensation, and $9.6 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 $2.6 million related to increased marketing and promotion costs to support continued revenue growth and our anticipated product launch for Hypothalamic Obesity.

Other income (expense), net.

Other income (expense), net remained relatively flat at ($0.1) million for both periods the three months ended September 30, 2025 and September 30, 2024.

The slight increase in other expense was primarily due to the following:

● a decrease of $1.0 million of non-cash interest expense in the three months ended September 30, 2025, associated with accretion of LG Chem liability, which was paid in July 2025, and

● a decrease in other income of $0.8 million due to less gain recognized on the change in fair value of the embedded derivative on our deferred royalty obligation as well as other income, including less gains on foreign exchange.

Comparison of the nine months ended September 30, 2025 and 2024

The following table summarizes our results of operations for the nine months ended September 30, 2025 and 2024, together with the changes in those items in dollars and as a percentage:

Nine Months Ended

September 30,

March 31, Change

2026 2025 $ %

(in thousands)

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Product revenue, net $ 60,112 $ 37,718 $ 22,394 59 %

License revenue

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

Total revenues 60,112 32,704 27,408 84 %

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Provision for income taxes 545 80 465 581 %

Net loss $ (55,639) $ (49,498) $ (6,141) 12 %

Product revenue, net .

Product revenue, net increased by $49.2 million to $137.5 million for the nine months ended September 30, 2025, from $88.3 million for the nine months ended September 30, 2024, an increase of 56% primarily due to an increase in sales volume.

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.

We expect our sales of IMCIVREE to continue to increase.

For the nine months ended September 30, 2025, and 2024, the majority of our product revenue , or 69% and 73%, respectively, was generated from sales of our product to pharmacies for patients in the United States.

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.

License revenue .

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.

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 $5.1 million to $14.7 million for the nine months ended September 30, 2025 from $9.6 million for the nine months ended September 30, 2024, an increase of 53%, which was driven by an increase in sales volume and net product revenue in the nine months ended September 30, 2025 .

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 $5.1 million increase in cost of sales in the nine months ended September 30, 2025, from the same period in 2024 was due to $2.5 million of additional royalties due to our growth in sales and $2.6 million attributed to increased product costs associated with higher sales volume.

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 .

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 $3.5 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.

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 decreased by $71.5 million to $125.3 million for the nine months ended September 30, 2025 from $196.8 million for the nine months ended September 30, 2024, a decrease of 36%.

The net decrease was primarily due to the following:

● a decrease of $92.4 million related to acquired In-Process Research and Development (“IPR&D”) costs associated with the acquisition of LGC’s proprietary compound bivamelagon in the nine months ended September 30, 2024, which did not recur in 2025;

● a net decrease of $1.6 million in our clinical trial costs due to the completion and wind down of our long-term extension trial, DAYBREAK phase 2, EMANATE phase 3, and RM-718 parts A and B long-term extension trial, Pathway Studies, Phase 3 pediatrics trial, and 718 Phase parts A and B, as well as decreased genetic sequencing costs.

The above decreases were partially offset by:

● an increase of $14.2 million related to personnel costs, including $7.7 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 $6.6 million of stock-based compensation, and

● an increase of $7.6 million associated with chemistry, manufacturing, and controls (CMC) costs for drug formulation and autoinjector development to support our ongoing Phase 1 clinical trial of RM-718 and our Phase 2 bivamelagon trial, as well as $1.3 million in other pre-clinical drug research efforts.

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%.

The net increase was primarily due to the following:

• an increase of $7.0 million related to personnel costs including $4.6 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 $2.5 million of stock-based compensation;

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

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.

Selling, general and administrative expense.

Selling, general and administrative expense increased by $31.3 million to $137.5 million for the nine months ended September 30, 2025 from $106.2 million for the nine months ended September 30, 2024, an increase of 29%.

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%.

The increase was primarily due to the following:

● an increase of $26.9 million related to personnel costs including $11.9 million of stock-based compensation, and $15.0 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, and

● an increase of $5.2 million related to increased marketing and promotion costs to support continued revenue growth, and our anticipated product launch for Hypothalamic Obesity.

• 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.

Other income (expense), net.

Other income (expense), net decreased by ($10.9) million to ($3.5) million for the nine months ended September 30, 2025 from $7.4 million of other income for the nine months ended September 30, 2024.

The decrease was primarily due to the following:

● a one-time gain of $8.9 million that was recognized for the settlement of the forward contract recorded with the issuance of Convertible Preferred Stock during the nine months ended September 30, 2024;

which did not recur in 2025, and

● an decrease in other income of $1.2 million primarily due an additional gain in the change in fair value of the embedded derivative on our deferred royalty obligation and other income, including less gains on foreign exchange in 2024.

● Offset by the recognition of an additional $0.7 million of non-cash interest expense in the nine months ended September 30, 2025, associated with an increase in the accretion of the deferred royalty obligation as well as the non-current liability payable to LGC that was paid in July 2025.

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:

• an increase of $0.8 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 September 30, 2025, our cash and cash equivalents and short-term investments were approximately $416.1 million.

On July 11, 2025, we closed a public offering of 2,367,647 of common stock at a public offering price of $85 per share, resulting in net proceeds of approximately $188.7 million, after deducting underwriting discounts and commissions, and other offering expenses payable by us.

The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024:

Nine Months Ended September 30,

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,

(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 $90.3 million for the nine months ended September 30, 2025 and consisted primarily of a net loss of $149.0 million adjusted for non-cash items of $59.0 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.

The change in operating assets and liabilities used net cash of approximately $0.3 million and was primarily driven by net increases in accounts receivable of $13.7 million, the change in prepaids and other current assets of $3.6 million, the change in inventory of $2.5 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 $20.6 million.

Net cash used in operating activities was $95.0 million for the nine months ended September 30, 2024 and consisted primarily of a net loss of $217.3 million adjusted for non-cash items of $121.9 million, which consisted of non-cash stock-based compensation, non-cash interest expense, non-cash accretion and amortization of short-term investments, non-cash accretion of other current liability, gain on settlement of forward contract, depreciation and amortization, non-cash rent expense and the change in the fair value of our embedded derivative liability, totaling $29.5 million.

Our net loss adjusted for non-cash items also includes $92.4 million of acquired IPR&D assets, which are classified as investing activities.

The change in operating assets and liabilities used net cash of approximately $0.3 million, primarily driven by net increases in accounts receivable and inventory of $9.7 million, offset by net decreases in accounts payable and accrued expenses of $6.7 million and net decreases in long-term assets of $3.2 million.

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 provided by investing activities

Net cash used in investing activities was $167.5 million for the nine months ending September 30, 2025 and relates to purchases of short-term investments for $328.3 million, offset by gross maturities of short-term investments of $200.8 million.

Additionally, we made the final payment of $40.0 million to LGC for the Company’s asset purchase of their proprietary compound bivamelagon from January 2024.

Net cash used in investing activities was $69.3 million for the nine months ended September 30, 2024 and relates to purchases of short term investments for $201.9 million and cash used for the purchase of LGC’s proprietary compound bivamelagon (LB54640) for $40.0 million in January 2024, offset by gross maturities of short-term investments of $173.1 million.

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 provided by financing activities was $220.9 million for the nine months ended September 30, 2025, and consisted of net proceeds of $188.7 million from our follow-on offering in July 2025, $34.0 million from our ATM equity offering, and proceeds of $12.7 million from the exercise of stock options and the issuance of common stock from our Employee Stock Purchase Plan.

These proceeds were offset by $14.5 million of repayments of our deferred royalty obligation

Net cash provided by financing activities was $152.9 million for the nine months ended September 30, 2024, and consisted of net proceeds of $147.8 million from the issuance of Series A Preferred Stock as well as 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 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.

These proceeds were offset by $4.6 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 September 30, 2025 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 March 31, 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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If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market our setmelanotide program that we would otherwise prefer to develop and market ourselves.

On November 2, 2021, we entered into a Sales Agreement with Cowen and Company, LLC (“Cowen”), pursuant to which we may issue and sell shares of our common stock, having an aggregate offering price of up to $100.0 million, from time to time through an “at the market” equity offering program under which Cowen acts as sales agent (the “ATM Program”).

On November 2, 2021, we entered into a Sales Agreement (the "Prior Sales Agreement") with TD Securities (USA) LLC (“TD Cowen”), pursuant to which we may issue and sell shares of our common stock, having an aggregate offering price of up to $100.0 million, from time to time through an “at the market” equity offering program under which TD Cowen acts as sales agent (the “ATM Program”).

Between August 10, 2023 and August 21, 2023, we sold approximately two million shares of our common stock in the ATM Program for net proceeds of approximately $48.9 million.

On February 29, 2024, the Company and Cowen entered into Amendment No.

1 to Sales Agreement (the “Amendment”) to increase the aggregate offering price of the shares of common stock that may be issued and sold pursuant to the Sales Agreement to $200,000,000 (excluding the aggregate offering price of shares of common stock issued and sold pursuant to the Sales Agreement prior to February 29, 2024).

In connection with the Amendment, on February 29, 2024, we filed with the SEC a prospectus supplement, dated February 29, 2024, which, combined with the Base Prospectus (together, the “New Prospectus”), amended the Prior Prospectus in its entirety.

The issuances and sales under the Sales Agreement, as amended by the Amendment, will be made pursuant to the Registration Statement and the New Prospectus.

On February 29, 2024, the Company and TD Cowen entered into Amendment No.

1 to Sales Agreement (the “Amendment”) to increase the aggregate offering price of the shares of common stock that may be issued and sold pursuant to the Prior Sales Agreement to $200.0 million (excluding the aggregate offering price of shares of common stock issued and sold pursuant to the Prior Sales Agreement prior to February 29, 2024).

In connection with the Amendment, on February 29, 2024, we filed with the SEC a prospectus supplement, dated February 29, 2024, related to the issuances and sales under the Prior Sales Agreement, as amended by the Amendment.

From December 10, 2024 to December 31, 2024, the Company sold 744,595 shares of common stock in the ATM Program for net proceeds of $41.2 million as of December 31, 2024.

The Company sold an additional 587,510 shares of common stock in the ATM Program from January 1, 2025 through January 21, 2025 for net proceeds of approximately $32.1 million in the quarter ending March 31, 2025.

On February 26, 2026, the Company and TD Cowen entered into a new Sales Agreement (the “Sales Agreement”) pursuant to which the Company may issue and sell shares of its common stock, having an aggregate offering price of up to $200.0 million, through its ATM Program.

The Sales Agreement supersedes the Prior Sales Agreement between TD Cowen and the Company, dated November 2, 2021, as amended on February 29, 2024, and the Prior Sales Agreement terminated upon the effectiveness of the Sales Agreement.

In connection with the Sales Agreement, on February 26, 2026, the Company filed with the SEC a registration statement on Form S-3ASR containing a base prospectus and a prospectus supplement, each dated February 26, 2026, pursuant to which issuances and sales under the Sales Agreement will be made.

Contractual obligations

As of September 30, 2025, 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, 2024.

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.

Quantitative and Qualitative Disclosures About Market Risk

As of September 30, 2025, 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, 2024.

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.

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