Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q2 2022 Earnings Presentation
30 slides from Rhythm Pharmaceuticals's earnings presentation, Aug 2, 2022
Slide 1: Second Quarter 2022 Financial Results and Business Update
Rhythm Pharmaceuticals Second Quarter 2022 Financial Results and Business Update August 2, 2022 ® © 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 Jennifer Chien, Executive Vice President, Head of North America Yann Mazabraud, Executive Vice President, Head of International Hunter
Slide 3: Forward Looking Statements
Forward Looking Statements This presentation contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including witho
Slide 4: David Meeker, MD
David Meeker, M Rhythm
Slide 5: Executing on our Global Strategy: A Precision Medicine Addressing Unmet Medical Needs
Executing on our Global Strategy: A Precision Medicine Addressing Unmet Medical Needs Strong Start to International Market Potential for Meaningful U.S. BBS Launch Access Advancing Label Expansion • IMCIVREE approved for BBS on June • CHMP* positive opinion on BBS; EC • Proof of concept achieved in
Slide 6: Hypothalamic Obesity: A Rare, Acquired Form of Obesity Following Injury to the Hypothalamic Region
Hypothalamic Obesity: A Rare, Acquired Form of Obesity Following Injury to the Hypothalamic Region Craniopharyngioma and other suprasellar brain tumors and treatment – tumor resection surgery and radiation – is most common cause MC4R pathway deficiency following injury to hypothalamic region causes
Slide 7: Clinically Meaningful Reductions Observed in All Patients by 16 Weeks
Clinically Meaningful Reductions Observed in All Patients by 16 Weeks 0 -5 * ** −5% change -7.02 -8.18 -10 Mean % Change BMI -10.03 -10% change -15 -12.70 -12.79 -16.12 -20 -18.39 -21.10 -25 -22.73 -24.46 -30 -35 -37.19 -40 Interim analysis as of the data cutoff date of May 6, 2022, as disclosed on
Slide 8: Setmelanotide and Hypothalamic Obesity: What's Next?
Setmelanotide and Hypothalamic Obesity What’s Next? 5,000 – 10,000* • Planning to present full data patients from 18 patients in Phase 2 trial Estimated U.S. prevalence in fall of 2022 • Request submitted for End of Phase 2 meeting with FDA, ~500* additional cases diagnosed in U.S. each year anticip
Slide 9: Setmelanotide and Hypothalamic Obesity: Potentially a Transformative Impact
Setmelanotide and Hypothalamic Obesity: Potentially a Transformative Impact Hypothalamic Obesity as an Meaningful Opportunity for MC4R Pathway Disease Rhythm • Phase 2 data reinforces the importance of • Patients are identified; no genetic testing the MC4 pathway as a key driver in some required for
Slide 10: Multiple Ongoing Clinical Trials Evaluating Setmelanotide
Multiple Ongoing Clinical Trials Evaluating Setmelanotide Enrollment complete Pediatrics Trial Weekly Formulation Phase 3 Phase 3 Patients aged 2 to <6 years Switch Trial Hypothalamic obesity Phase 3 Trial planned for 1H2023 Phase 3 Trial Phase 2 Trial ® 10
Slide 11: Jennifer Chien: BBS U.S. Launch
Jennifer Chien BBS U.S. Launch ® 11
Slide 12: IMCIVREE Approved in BBS on June 16, 2022; Commercial Launch Efforts Well Underway
IMCIVREE Approved in BBS on June 16, 2022; Commercial Launch Efforts Well Underway Now approved First and only FDA-approved therapy that targets a root cause of hyperphagia and early- onset, severe obesity in patients with Bardet- Biedl syndrome Physicians are writing Initial payer mix Patients with
Slide 13: Foundation Set and Momentum Building at BBS Launch
Foundation Set and Momentum Building at BBS Launch Foundation laid Momentum building at 2021 to 2022 approval for BBS U.S. commercial launch meeting Commercial availability for POMC, PCSK1 and LEPR ENDO 2022 deficiency Continued physician engagement Territory Managers engaging Series of BBS Foundati
Slide 14: Initial Six Weeks of Launch off to a Strong Start
Initial Six Weeks of Launch off to a Strong Start Territory manager engagement with physicians Clinical trial conversions Patient advocacy groups >50* >35* and associations BBS unique prescriptions prescribers CRIBBS registry at Marshfield Clinic Non-personal promotional efforts to reach physicians
Slide 15: Snapshot of Patient Prescriptions Received in First Six Weeks
Snapshot of Patient Prescriptions Received in First Six Weeks Source of prescriptions Age Range % >50* Adult 18+ 29% 22% BBS Adolescent 12-17 34% 78% prescriptions Pediatric 6-11 37% New Physicians Targeted Physicians *As of July 28, 2022 ® 15
Slide 16: BBS IMCIVREE Prescribers by Specialty
BBS IMCIVREE Prescribers by Specialty 7% 17% 27% >35* Pediatrics Endocrinology unique Pediatric Endocrinology prescribers 4% Primary Care 45% All others *As of July 28, 2022 ® 16
Slide 17: More than 80% of Initial BBS Prescriptions Fall Under Commercial and Medicaid Plans
More than 80% of Initial BBS Prescriptions Fall Under Commercial and Medicaid Plans 3% 7% • Pull-through of prescriptions 6% in line with expectations • Many Medicare patients 34% 50% have secondary insurance Commercial Medicaid Medicare Only Medicare + Secondary Federal *As of July 28, 2022 ® 17 Da
Slide 18: Rhythm InTune Support Services
Rhythm InTune Support Services Personalized program to achieve access, set treatment expectations and support patient adherence and continuity of therapy Introduce Set Injection tips Goal Treatment Adherence Discuss therapy IMCIVREE expectations setting support with physician ® 18
Slide 19: Positive Early Signals in Launch
Positive Early Signals in Launch Educate for Educate on Find diagnosed earlier Facilitate Initiation on pathway and patients diagnosis of reimbursement IMCIVREE IMCIVREE patients ® 19
Slide 20: Yann Mazabraud, EVP, Head of International
Yann Mazabraud EVP, Head of International ® 20
Slide 21: Significant Market Opportunity for BBS and POMC, PCSK1 and LEPR Deficiencies in Europe
Significant Market Opportunity for BBS and POMC, PCSK1 and LEPR Deficiencies in Europe POMC, PCSK1 and LEPR Bardet-Biedl Syndrome Deficiency Obesities ~100 >1,500 individuals identified individuals identified in EU4 + UK in EU4 + UK (~20 academic medical centers with >40 BBS patients) Estimated Euro
Slide 22: Market Access for POMC, PCSK1 and LEPR Deficiency
Market Access for POMC, PCSK1 and LEPR Deficiency • Germany Launched, first sales in 2Q22 with exemption from GBA lifestyle drug exclusion list Sweden • France Reimbursed since March 2022 via early access program England Netherlands Germany • United Kingdom Commercial launch set for October 2022 fol
Slide 23: EMA's CHMP Recommends IMCIVREE for Treatment of Obesity and Control of Hunger in Patients with BBS
EMA’s CHMP Recommends IMCIVREE for Treatment of Obesity and Control of Hunger in Patients with BBS EC decision on Marketing Authorization Anticipated in 4Q2022 France Strong Support for IMCIVREE Reimbursed early access program achieved in July 2022 among Leading European BBS Experts United Kingdom P
Slide 24: French ANSM and HAS Grant Pre-marketing Authorization for IMCIVREE for Patient with BBS
French ANSM and HAS Grant Pre-marketing Authorization for IMCIVREE for Patient with BBS Ahead of CHMP 32 days recommendation following and EMA the US FDA authorization approval No restriction to label ® 24
Slide 25: Hunter Smith: 2Q 2022 Financial Results
Hunter Smith 2Q 2022 Financial Results ® 25
Slide 26: 2Q22 Financial Highlights: Cash Runway Extended into 2H 2024 with Revenue Interest Financing Agreement
2Q22 Financial Highlights: Cash Runway Extended into 2H 2024 with Revenue Interest Financing Agreement with HealthCare Royalty Non-dilutive, capped RIFA provides financial flexibility with investment of up to $100M To Rhythm: • $37.5M received upon approval of IMCIVREE for BBS • $37.5M following EC
Slide 27: 2Q2022 Financial Snapshot
2Q2022 Financial Snapshot ($ in millions except as noted, per share data Three months ended Three months ended and shares outstanding) June 30, 2022 June 30, 2021 Product revenue, net $2.3M $0.3M License revenue $6.8 _ R&D expenses $31.5M $25.1M SG & A expenses $22.3M $15.5M Net (loss) $(44.7)M $(35
Slide 28: David Meeker, MD: Conclusion
David Meeker, MD Conclusion ® 28
Slide 29: Rhythm's Strategic Priorities for 2022 and 2023
Rhythm’s Strategic Priorities for 2022 and 2023 Execute on U.S. Achieve access Initiate Phase 3 Expand commercial and launch trial to evaluate IMCIVREE strategy with IMCIVREE for setmelanotide in opportunity BBS launch both BBS and hypothalamic through POMC, PCSK1 obesity additional and LEPR in stud
Slide 30: Questions
Questions Rhythm
Rhythm Pharmaceuticals — Q2 2022 Earnings Presentation on 6ix