Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM

Rhythm Pharmaceuticals — Pediatric Endocrine Society Annual Meeting 2024

12 slides from Rhythm Pharmaceuticals's conference presentation, May 2024

  1. Slide 1: Efficacy and Safety of Setmelanotide in Patients Aged 2 to <6 Years: VENTURE Trial

    Efficacy and Safety of Setmelanotide in Patients Aged 2 to <6 Years With Rare Melanocortin-4 Receptor Pathway Diseases of Obesity: Results From VENTURE, a Phase 3, Multicenter, 1-Year, Open-Label Trial Jesús Argente, MD, PhD1,2; Uzoma Okorie, MD, FAAP3; Charles F. Verge, MB, BS, PhD4; Ilene Fennoy,

  2. Slide 2: Disclosures

    Disclosures • JA has received payment for lectures and advisory boards from Rhythm Pharmaceuticals, Inc. • UO, CFV, IF, and MMK’s institutions have received funding and materials for research from Rhythm Pharmaceuticals, Inc. • SF has received speaker fees from Rhythm Pharmaceuticals, Inc. • CC, CS,

  3. Slide 3: Energy Balance Is Regulated by the Hypothalamic MC4R Pathway

    Energy Balance Is Regulated by the Hypothalamic MC4R Pathway • Under physiologic conditions, the MC4R pathway regulates hunger, satiety, energy expenditure, and body weight, whereas rare variants in genes involved in the MC4R pathway are associated with hyperphagia and early-onset, severe obesity1-9

  4. Slide 4: Study Design and Eligibility Criteria

    Study Design and Eligibility Criteria • Study design (NCT04966741) Open-label Open-label Outcomes Bridging program (ages <6 years) or dose escalation maintenance dose • Weight-based commercial product (ages ≥6 years) measurements • Caregiver Reported Global c Screening QD setmelanotide QD setmelanot

  5. Slide 5: Baseline Characteristics and Disposition

    Baseline Characteristics and Disposition Parameter POMC or LEPR deficiency BBS Total Patient disposition Enrolled patients, n 7 5 12 Age range, years 3-4 2-5 2-5 Patients enrolled Male, n (%) 5 (71.4) 2 (40.0) 7 (58.3) N=12 Race, n (%)* • 3 with POMC deficiency • 4 with LEPR deficiency White 3 (42.9

  6. Slide 6: Coprimary Endpoints: From Baseline to Week 52 in Safety Population

    Coprimary Endpoints: From Baseline to Week 52 in Safety Population (Data as Observed) The proportion of patients achieving a clinically meaningful decrease in Mean percent change in BMI Z score (WHO) of ≥0.2 absolute BMI 83.3% (10 of 12)* −18.4% 95% CI, 58.7-99.8 • 85.7% of patients with POMC • −25.

  7. Slide 7: Mean BMI Z Score Reduction in Safety Population (Data as Observed)

    Mean BMI Z Score Reduction in Safety Population (Data as Observed) BMI-Z (CDC) change from baseline BMI-Z (WHO) change from baseline n= 12 9 12 8 12 11 10 10 11 11 11 11 n= 12 9 12 8 12 11 10 10 11 11 11 11 0.0 -0.2 0.0 -0.2 Mean (SD) BMI Z score (WHO) -1.0 Mean (SD) BMI Z score (CDC) -1.0 -2.0 -2.0

  8. Slide 8: Individual BMI Z Score (CDC) Reductions at Week 52

    Individual BMI Z Score (CDC) Reductions at Week 52 (Data as Observed) POMC (n=3) LEPR (n=4) BBS (n=5) 0 * -0.16 -0.5 BMI Z score (CDC) reduction from baseline -0.48 -0.46 -0.43 † -1 -0.83 -1.13 -1.5 -2 -2.5 -2.36 -2.32 -2.42 -2.56 -3 -3.5 -4 -4.18 -4.5 *Patient was nonadherent and lost to follow-up

  9. Slide 9: Change in %BMI95 From Baseline (Data as Observed)

    Change in %BMI95 From Baseline (Data as Observed) n= 12 9 12 8 12 11 10 10 11 11 11 11 10 0 -10 Mean (SD) change in %BMI95 -20 -30 −33 percentage points -40 -50 -60 0 4 8 12 16 20 24 28 32 36 40 44 48 52 Week %BMI95, percent of the 95th BMI percentile; SD, standard deviation. Pediatric Endocrine Soc

  10. Slide 10: Reduction in Patient Hunger Over Weeks as Observed by Caregivers

    Reduction in Patient Hunger Over 52 Weeks as Observed by Caregivers • Overall, 10 of 11 patients (91%; 6 of 6 with POMC or LEPR deficiency; 4 of 5 with BBS) demonstrated hunger reduction at 52 weeks compared with before the study, as observed by caregivers How hungry has your child acted in the past

  11. Slide 11: Adverse Events

    Adverse Events • All patients had at ≥1 AE and ≥1 TRAE • All AEs were mild (7 patients [58.3%]) or moderate (5 patients [41.7%]) • There were no discontinuations related to AEs, serious AEs, or deaths • There was no evidence of impaired growth or neurocognitive development due to setmelanotide TRAEs

  12. Slide 12: Conclusions

    Conclusions • Setmelanotide was well tolerated and demonstrated consistent, clinically meaningful weight-related reductions in patients 2 to <6 years of age Clinically meaningful Most caregivers Generally well reductions in BMI and reported a reduction tolerated with a safety BMI Z score (CDC and in

Rhythm Pharmaceuticals — Pediatric Endocrine Society Annual Meeting 2024 on 6ix