Rhythm Pharmaceuticals (RYTM) Corporate presentation summary

Strategic focus and pipeline development

• Advancing MC4R agonist therapies across genetic MC4R pathway diseases, hypothalamic obesity, and next-generation assets bivamelagon (oral) and RM-718 (weekly injectable).

• IMCIVREE (setmelanotide) serves as the global commercial foundation, with approvals for Bardet-Biedl syndrome (BBS) and acquired hypothalamic obesity (HO) in the US, EU, and Japan.

• Expanding pipeline includes ongoing and planned trials in congenital HO, Prader-Willi syndrome (PWS), and congenital hyperinsulinism.

• U.S. patent protection for next-generation assets extends into the 2040s.

Commercial execution and financial position

• IMCIVREE available in over 25 countries, supporting long-term growth and global infrastructure.

• Net product revenues have shown steady growth since BBS launch, reaching $71.3M by Q2 2026.

• U.S. launch for acquired HO demonstrated strong patient demand, with over 400 start forms in the first 14 weeks and broad prescriber engagement.

• Early payer engagement resulted in positive coverage policies for 25% of Medicaid and 35% of commercial lives.

• Cash, cash equivalents, and short-term investments of $330.9M as of June 30, 2026, sufficient to fund operations for at least 24 months.

Clinical data and efficacy highlights

• Phase 3 trial of setmelanotide in acquired HO showed a -18.8% placebo-adjusted BMI reduction at 52 weeks (p<0.0001).

• Consistent BMI reductions observed across age groups and in patients with prior or concomitant GLP-1 use.

• Real-world and long-term extension data confirm durable BMI and hunger reductions, with some pediatric patients achieving normal weight.

• Bivamelagon and RM-718 demonstrated statistically significant and clinically meaningful BMI reductions, comparable to setmelanotide.

• Setmelanotide in PWS Phase 2 trial showed mean BMI reduction of -3.06% at six months, with improvements in hyperphagia and anxiety.