Rhythm Pharmaceuticals (RYTM) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Key data presentations and upcoming milestones

• Six-month data from an 18-patient open-label study for Praloretel will be presented at ENDO, including weight, BMI, DEXA, and patient-reported outcomes for each patient.

• Updated one-year data for RM-718 (bivamelagon) will be highlighted at the conference, showing confirmatory results similar to setmelanotide.

• Next steps for RM-718 include starting adult trials by year-end and pediatric trials in Q1, with a chewable tablet formulation.

• Data for RM-718 in HO will be shared next quarter, focusing on comparison to semaglutide.

Commercial launch and market opportunity

• IMCIVREE was approved for HO in March, with a broad label and no major restrictions, targeting a U.S. population of about 10,000.

• Initial launch saw 150 patient start forms in six weeks, with strong physician interest but low disease awareness.

• Sales force targets endocrinologists, focusing on those with more than two patients, aiming to reach 5,000 of the 10,000+ patients.

• Coverage plans are expected to be in place within 6–12 months, with Medicaid and Medicare populations influencing gross-to-net.

Physician and patient dynamics

• Most HO patients are managed by endocrinologists due to pituitary insufficiency post-tumor resection.

• Physician feedback shows high interest but limited prior awareness; availability of therapy is expected to drive diagnosis and education.

• Adherence is anticipated to be high, especially as patients seek to return to normalcy; hyperpigmentation discontinuation rates are expected to be low.