Rhythm Pharmaceuticals (RYTM) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase summary

Key value drivers and disease focus

• Three main value drivers: approved drug for rare genetic indications, promising results in hypothalamic obesity (HO), and a focus on the POMC signaling pathway.

• Bardet-Biedl syndrome (BBS) and HO represent significant rare disease opportunities, with BBS affecting 4,000–5,000 in the U.S. and HO 5,000–10,000, most of whom are diagnosed.

• Setmelanotide acts as a hormonal replacement, addressing satiety signaling deficits in these populations.

• Early intervention in genetic obesity is critical, as early-onset cases have significantly reduced life expectancy and accumulate comorbidities from a young age.

• The company is exploring additional genetic targets within the same pathway for future expansion.

Clinical development and data

• Phase III trial for HO is fully enrolled, with a very low dropout rate and data expected in the first half of next year.

• Phase II data in HO showed consistent and significant BMI reduction, with all patients responding to treatment.

• The trial is now 99% powered to show a 10% difference due to a larger safety database.

• Variability in response is attributed to patient heterogeneity, but even lower responders showed meaningful changes in body composition.

• 25% of phase III participants had prior GLP-1 use, and 10% were still on GLP-1s during the trial.

Commercial strategy and market access

• BBS launch is progressing slowly but steadily, with a focus on community building, genetic testing, and patient identification.

• U.S. BBS opportunity estimated at $250–$300 million, assuming 1,000 patients at $250,000–$300,000 net per patient.

• European market access is strong, with pricing reflecting rare disease status and launches expanding across major and smaller countries.

• Commercial ramp for HO is expected to be steeper than BBS due to higher diagnosis rates and patient concentration, but will still face rare disease market challenges.

• Sales force will expand to support HO launch, leveraging existing infrastructure and relationships with key opinion leaders and centers of excellence.