Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — ObesityWeek 2023
23 slides from Rhythm Pharmaceuticals's conference presentation, Oct 18, 2023
Slide 1: Rhythm at ObesityWeek® 2023
Rhythm at ObesityWeek® 2023 Data presentations in hypothalamic obesity and more as presented at ObesityWeek Oct. 18, 2023 © Rhythm Pharmaceuticals, Inc. All rights reserved.
Slide 2: On Today’s Call
On Today’s Call David Connolly, Executive Director of Investor Relations and Corporate Communications David Meeker, MD, Chair, President and Chief Executive Officer Hunter Smith, Chief Financial Officer ® 2
Slide 3: Forward Looking Statements
Forward Looking Statements This presentation contains certain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, and that involve risks and uncertainties, including without limitation statements regarding the potential, safety, efficacy, and regula
Slide 4: Rhythm at ObesityWeek® 2023
Rhythm at ObesityWeek® 2023 3-Year Impact of Impact of Weight Reduction Setmelanotide Cardiac, Renal, and Setmelanotide on 4-Year Setmelanotide on in Patients with Weight Outcomes Endocrine/Diabetes Metabolic Setmelanotide Metabolic Hypothalamic in Patients with Mellitus Outcomes Syndrome Risk in We
Slide 5: Weight Reduction in Patients with Hypothalamic Obesity Treated With Setmelanotide for 12 Months
Weight Reduction in Patients with Hypothalamic Obesity Treated With Setmelanotide for 12 Months ® 5
Slide 6: Patient Dispositions: Phase 2 to Open-label, Long-term Extension Trial
Patient Dispositions: Phase 2 to Open-label, Long-term Extension Trial Patients enrolled/ setmelanotide full analysis set/ Discontinued study safety population (n=2, both because of AEs) (N=18) Protocol violations Completed study (n=1, nonadherent with study drug per protocol administration) (n=15)
Slide 7: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics Total (n=14) Age, mean (SD), y 13.6 (5.0) Age range, n (%), y Adults >18 2 (14.3) Children 10-18 12 (85.7) Sex, n (%) Female 4 (28.6) Male 10 (71.4) Tumor type, n (%) Craniopharyngioma 11 (78.6) Hypothalamic hamartoma 2 (14.3) Juvenile pilocytic astrocytoma
Slide 8: Setmelanotide Achieved Progressive, Deepening BMI Reduction at 16 Weeks, 6 and 12 Months
Setmelanotide Achieved Progressive, Deepening BMI Reduction at 16 Weeks, 6 and 12 Months in Patients with Hypothalamic Obesity Error bars are the standard deviation. *Includes all patients who received 16 weeks of setmelanotide in the index trial and ≥12 months of treatment in the long-term extensio
Slide 9: Sustained and Deepened BMI Reduction in Hypothalamic Obesity at One Year
Setmelanotide Achieved Sustained and Deepened BMI Reduction in Patients with Hypothalamic Obesity at One Year 0 BMI % change from index trial baseline -5 -10 -15 -20 -25 -30 -35 -40 -17.7% -25.5% mean percent reduction mean percent reduction -45 in BMI at Week 16 in BMI at Month 12 from baseline fro
Slide 10: All Patients Achieved ≥5% BMI Reduction at One Year
All Patients Achieved ≥5% BMI Reduction at One Year Patient who discontinued Patients on treatment at Month 12 Patient lost to follow-up (n=1) treatment because of an AE (n=1) (n=12) 0 5 Reinitiated 5 Dose Off treatment Dose Off treatment treatment reduced increased 0.8 -5 -7.0 0 0 BMI change from b
Slide 11: Body Composition Data Show Greater Decreases in Total Fat Mass vs. Lean Muscle Mass
Body Composition Data Show Greater Decreases in Total Fat Mass vs. Lean Muscle Mass Patient number 1 2 3 4 5 6 7 8 9 10 11 12 Age at baseline 6 9 9 10 11 12 13 14 15 15 16 24 Percent change in BMI from baseline to -35.8 -20.5 -39.1 -23.9 -7 -8.7 -21.6 -29.2 -14.6 -36.6 -42.4 -27.1 Month 12 40 17.5 1
Slide 12: All Patients Achieved a Decrease in Obesity Severity at One Year
All Patients Achieved a Decrease in Obesity Severity at One Year Three of 11 pediatric patients achieved normal weight at one year based on NIH, WHO weight classifications Adults WHO Classification BMI, kg/m2 Pediatric patients (n=11)* BMI percentile6 (n=1) (NIH5) 190 ≥50 50 166 157 158 Obesity clas
Slide 13: Setmelanotide and Hypothalamic Obesity: A Transformative Opportunity for Rhythm
Setmelanotide and Hypothalamic Obesity: A Transformative Opportunity for Rhythm ✓ Unmet medical need is high; no approved therapies 5,000 – 10,000* ✓ MC4R pathway deficiency following patients injury to hypothalamic region Estimated U.S. prevalence ✓ Patients are identified; no genetic testing requi
Slide 14: Hypothalamic Obesity: Expect to Complete Enrollment by the end of 2023 in Ph3 Trial
Hypothalamic Obesity: Expect to Complete Enrollment by the end of 2023 in Ph3 Trial Screening and Dose titration Double-blind, placebo-control Randomization Setmelanotide QD (n=80) 120 patients Open-label randomized to setmelanotide: 2:1 extension placebo Placebo QD (n=40) Up to 52 weeks 8 weeks Sta
Slide 15: Questions
Questions Rhython
Slide 16: Appendix: Additional TOS 2023 presentation summaries
Appendix: Additional TOS 2023 presentation summaries ® 16
Slide 17: Patient Baseline Demographics and Dispositions Related to TOS 2023 Long-term Data Readout in Patients with BBS
Patient Baseline Demographics and Dispositions Related to TOS 2023 Long-term Data Readout in Patients with BBS BBS patients with clinically meaningful, age-appropriate weight response at 1 year (N=35) Not included: • Ongoing with <3 years of setmelanotide treatment (n=4) • Transitioned out of trial
Slide 18: 16% BMI Reduction in Adult Patients with BBS on Setmelanotide Therapy at Three Years
16% BMI Reduction in Adult Patients with BBS on Setmelanotide Therapy at Three Years Error bars are the SD. BBS, Bardet-Biedl syndrome; BMI, body mass index; SD, standard deviation. ® 18
Slide 19: Reduction of 0.7 in Mean BMI Z Achieved in Pediatric Patients at Three Years
Reduction of 0.7 in Mean BMI Z Achieved in Pediatric Patients at Three Years of Setmelanotide Therapy Error bars are the SD. %BMI95, percent of the 95th BMI percentile; BBS, Bardet-Biedl syndrome; BMI, body mass index; SD, standard deviation. ® 19
Slide 20: 4-Year Setmelanotide Weight Outcomes of Patients With POMC and LEPR Deficiency Obesity
4-Year Setmelanotide Weight Outcomes of Patients With POMC and LEPR Deficiency Obesity Key takeaways • The long-term efficacy of setmelanotide was analyzed in patients with POMC and LEPR deficiency who had a Weight measures over 4 years of treatment* clinically meaningful weight response at Year 1 a
Slide 21: Cardiac, Renal, and Endocrine/Diabetes Mellitus Outcomes in Children with Bardet-Biedl Syndrome
Cardiac, Renal, and Endocrine/Diabetes Mellitus Outcomes in Children with Bardet-Biedl Syndrome Key takeaways Prevalence of cardiac, endocrine, diabetes, and renal outcomes • The prevalence of cardiac, endocrine/diabetes, and in the overall population renal outcomes was examined in children (aged ≤1
Slide 22: Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With Bardet-Biedl Syndrome
Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With Bardet-Biedl Syndrome Key takeaways • The change in MetS risk as assessed through the MetS-Z-BMI score was quantified in patients with BBS following 1 year of setmelanotide treatment (N=22) MetS-Z-BMI score* at baseline and week 52
Slide 23: Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With POMC and LEPR Deficiency
Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With POMC and LEPR Deficiency Key takeaways • The change in MetS risk as assessed through the MetS-Z- BMI score was quantified in patients with POMC (n=10 [POMC, n=9; PCSK1, n=1]) and LEPR (n=8) deficiency following 1 year of setmelanoti