Rhythm Pharmaceuticals (RYTM) Wells Fargo 21st Annual Healthcare Conference summary

Business overview and strategic pillars

• Three main pillars: genetic indications (POMC deficiency, BBS), hypothalamic obesity (HO), and Prader-Willi syndrome, each representing significant rare disease opportunities.

• HO launch is in its second quarter, targeting an estimated 10,000 U.S. patients, with strong early momentum and a focused sales force of 42 reps.

• Prader-Willi syndrome is highlighted as a major unmet need with a clear diagnosis pathway and recognized by payers as a devastating disease.

• Patient identification and awareness are central, with ongoing efforts to educate endocrinologists and leverage claims data for targeted outreach.

• Patient advocacy is growing, with organizations like the Raymond A. Wood Foundation increasing HO awareness.

HO launch progress and market dynamics

• Early adoption is broad-based, with about 400+ scripts written by 300 prescribers, indicating wide engagement rather than concentration in a few centers.

• Main friction point is clinic access due to busy endocrinology practices; payer policies are developing favorably, with many approvals processed via prior authorization.

• Guidelines and published data are critical for physician education and driving adoption, especially as HO is now recognized as a distinct hormonal deficiency.

• The HO market is more accessible than BBS, with a steeper expected ramp and potential for deeper penetration among the 10,000 patient population.

• Steady growth is anticipated, with the possibility of underestimating the true patient population as awareness increases.

Prader-Willi development and future plans

• Phase II data in Prader-Willi showed consistent positive trends across hyperphagia, anxiety, BMI, and body composition endpoints.

• Phase III plans include a six-month hyperphagia study, with potential for additional trials to support broader label claims or combination therapies.

• Trial design will include global sites, careful inclusion/exclusion criteria, and may allow stable use of other approved therapies like VYKAT XR.

• Manufacturing scale-up and formulation improvements are key gating factors for advancing both oral and injectable assets into pivotal trials.

• Prader-Willi is seen as central to future strategy, with investment in both oral and injectable options and consideration of combination approaches.