Rhythm Pharmaceuticals: A Promising Player in Rare Neuroendocrine Diseases

Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company, recently announced preliminary unaudited net revenues from global sales of IMCIVREE® (setmelanotide) for the fourth quarter of 2024 and the full year of 2024, as well as significant advancements in its pipeline and upcoming milestones. The company's strong performance and strategic initiatives position it as a promising player in the rare neuroendocrine diseases space.



Preliminary Unaudited Fourth Quarter and Full Year 2024 Net Product Revenues
Rhythm Pharmaceuticals expects net revenues from global sales of IMCIVREE to be approximately $42 million for the fourth quarter of 2024, representing a 26% increase on a sequential basis from the third quarter of 2024. For the full year of 2024, net revenues are expected to be approximately $130 million, compared to $77.4 million for the full year of 2023. The sequential quarter-over-quarter increase was due to growth in reimbursed patients on therapy and inventory growth in the United States. U.S. sales of IMCIVREE contributed approximately 74% of fourth quarter preliminary unaudited net product revenues and approximately 73% of full-year 2024 revenues.

Pipeline Advancements and Upcoming Milestones
Setmelanotide
Acquired Hypothalamic Obesity (HO)
Rhythm is on track to report topline data from the pivotal, 120-patient cohorts of its global, Phase 3 trial evaluating setmelanotide in acquired HO in the first half of 2025. The company has completed enrollment in its supplemental, 12-patient Japanese cohort of the global Phase 3 trial evaluating setmelanotide in acquired HO. Data from this supplemental cohort will serve as the basis for a regulatory submission in Japan.

Congenital HO
Rhythm anticipates enrolling the first patients with congenital HO in a 34-week substudy of the ongoing global Phase 3 trial in the first quarter of 2025. This substudy is independent from the pivotal Phase 3 trial in acquired hypothalamic obesity.

Genetically Caused MC4R Pathway Diseases
Rhythm completed enrollment in the Phase 3 EMANATE trial, which is comprised of four substudies: SH2B1, POMC and/or PCSK1, SRC1, and LEPR. The four-substudy design of this trial allows for independent data readouts and potential registration for each genetic cohort. The primary endpoint for each substudy is the difference in mean percent change in BMI from baseline to 52 weeks in the setmelanotide arm compared to the placebo arm. Rhythm anticipates reporting topline data from the Phase 3 EMANATE trial in the first half of 2026.

Prader-Willi Syndrome (PWS)
Rhythm plans to initiate a new, 26-week, open-label Phase 2 trial evaluating setmelanotide in PWS. This trial could provide valuable data on the efficacy and safety of setmelanotide in a new patient population, potentially leading to regulatory approval and commercialization in PWS.



Rhythm Pharmaceuticals' expansion into new markets, such as Japan, contributes to its global strategy and potential revenue growth by increasing patient access, diversifying revenue streams, and securing regulatory approvals in new markets. The company's strong performance and strategic initiatives position it as a promising player in the rare neuroendocrine diseases space, with a robust pipeline and a commitment to transforming the lives of patients and their families living with these conditions.

In conclusion, Rhythm Pharmaceuticals' preliminary unaudited net revenues, pipeline advancements, and upcoming milestones demonstrate the company's strong performance and strategic initiatives in the rare neuroendocrine diseases space. With a promising pipeline and a commitment to transforming the lives of patients, Rhythm Pharmaceuticals is well-positioned to continue its growth and success in the coming years.