Rhythm Pharmaceuticals (RYTM) investor relations material

Platform and product overview

• Focuses on melanocortin-4 pathway biology, with an approved analog drug targeting this receptor.

• Addresses impaired signaling leading to severe obesity, with both genetic and acquired causes.

• Initial approvals targeted rare genetic deficiencies, later expanding to Bardet-Biedl syndrome and acquired hypothalamic obesity.

• Drug is now available in about 25 countries for three genetic indications.

Market penetration and launch dynamics

• Early rare genetic indications had limited commercial potential due to small patient populations.

• Bardet-Biedl syndrome (BBS) has about 5,000 U.S. patients, with steady, long-term growth expected.

• Acquired hypothalamic obesity (HO) is a larger, more concentrated market, estimated at 10,000 U.S. patients.

• HO launch uses a larger sales force (42 reps) targeting endocrinologists, with a steeper expected ramp than BBS.

Early launch metrics and pricing

• Over 150 patient start forms written by 110 physicians within the first six weeks of HO launch, triple the rate of BBS.

• List price is approximately $380,000 per year, with net price around $330,000.

• Uptake reflects broad physician engagement and pent-up demand in the community.