Rhythm Pharmaceuticals (RYTM) investor relations material

Key product and clinical updates

• IMCIVREE (setmelanotide) is approved for three rare neuroendocrine disorders, with a fourth (hypothalamic obesity) under FDA review, PDUFA date set for March 20.

• Recent interim phase II data in Prader-Willi syndrome (PWS) show promising results, with further data expected in the first half of the year.

• Two pipeline compounds, an oral MC4R agonist (bivamelagon) and a weekly injectable (RM-718), are in development for improved dosing and tolerability.

• Setmelanotide demonstrated a 19.8% placebo-adjusted BMI reduction in HO patients, with high responder rates across age groups.

• Phase I study of RM-718 in PWS is underway, with updates on enrollment and timelines expected later in the year.

Commercial and sales trajectory

• BBS is the primary sales driver, with $57 million reported last quarter and 9% average quarter-over-quarter growth last year.

• IMCIVREE is available in 25 countries, with ongoing patient identification and prescription growth.

• U.S. BBS market could reach 1,000 patients and $300 million peak sales, with additional opportunity ex-U.S.

• HO represents a larger market, with an estimated 10,000 U.S. patients and higher long-term sales potential.

• Expanded U.S. sales force from 16 to 42 in anticipation of HO approval, with over 2,000 HO patients identified in key practices.

Market access and launch considerations

• Patient identification for HO leverages claims data and physician profiling, targeting 5,000 endocrinologists.

• Launch trajectory may be affected by physician awareness, appointment delays, and payer reimbursement timelines.

• Medicare reimbursement is currently prohibited for weight loss therapies, potentially impacting HO patient access.

• PDUFA delay was due to an FDA request for supplemental BMI Z score analysis, with no impact on efficacy endpoints.