Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Q4 2021 Earnings Presentation
29 slides from Rhythm Pharmaceuticals's earnings presentation, Mar 1, 2022
Slide 1: Fourth Quarter / Year End 2021 Financial Results
Rhythm Pharmaceuticals Fourth Quarter / Year End 2021 Financial Results March 1, 2022 ® © Rhythm® Pharmaceuticals, Inc. All rights reserved.
Slide 2: 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 3: On Today's Call
On Today’s Call David Meeker, MD, Chair, President and Chief Executive Officer Linda Shapiro Manning, MD, PhD, Chief Medical Officer Yann Mazabraud, Executive Vice President, Head of International Hunter Smith, Chief Financial Officer ® 3
Slide 4: David Meeker, MD
David Meeker, M Rhythar
Slide 5: Multiple Recent Highlights and Achievements
Multiple Recent Highlights and Achievements Preparing for BBS U.S. launch U.S. IMCIVREE commercial • Severe obesity and hyperphagia availability meeting expectations • No approved therapies • 4Q21 net sales > $1.8M • PDUFA goal date now June 16 • Continued progress with reimbursement from payers • P
Slide 6: The Morris Family: Our Life with BBS
THE MORRIS FAMILY Our Life with BBS ® 6
Slide 7: Rhythm Continues to Advance to BBS Commercial Launch
Rhythm Continues to Advance to BBS Commercial Launch Key takeaways from our virtual BBS Commercial Event held on Feb. 16 Unmet need in BBS Solution Rhythm is ready to • Hyperphagia • Address root cause launch in BBS • Severe obesity • Hunger reduction • Commercial foundation established • Co-morbidi
Slide 8: Roadmap to Identifying Patients with BBS
Roadmap to Identifying Patients with BBS Undiagnosed U.S. prevalence estimated to be >350 1,500 to 2,500 Identified and patients* Suspected diagnosed patients Diagnosed patients Identified and diagnosed * Company estimate based on published prevalence estimates of one in 100,000 in North America. ®
Slide 9: IMCIVREE Commercial Availability Met Expectations in 2021
IMCIVREE Commercial Availability Met Expectations in 2021 $3.2M revenue in 2021 $1.8M revenue in 4Q21 ® 9
Slide 10: Exclusive Licensing Agreement with RareStone Expected to Expand Reach into Asia
Exclusive Licensing Agreement with RareStone Expected to Expand Reach into Asia In December 2021, RareStone was granted an exclusive license to develop and commercialize IMCIVREE in China, including mainland China, Hong Kong and Macau RareStone agreed to: Under the terms of the agreement: • Seek loc
Slide 11: Linda Shapiro, MD, PhD: Regulatory and Clinical Update
Linda Shapiro, MD, PhD Regulatory and Clinical Update ® 11
Slide 12: Rare Disease Day, World Obesity Day and Obesity Care Week
Rare Disease Day, World Obesity Day and Obesity Care Week ® 12
Slide 13: Multiple Trials Evaluating Setmelanotide
Multiple Trials Evaluating Setmelanotide Pediatrics Trial Weekly Switch Trial (patients aged 2 to <6 years) Phase 3 Phase 3 Phase 2 Trial Phase 3 Trial ® 13
Slide 14: Anticipated Clinical Data Readouts coming in 1H2022
Anticipated Clinical Data Readouts coming in 1H2022 Long-term Extension Trial Phase 2 Hypothalamic • 12-month data for SRC1 Obesity Study deficiency obesity • Preliminary data • 12-month data for SH2B1 deficiency obesity • 24-month data for Bardet- Biedl syndrome Exploratory Phase 2 • 24-month data
Slide 15: Hypothalamic Obesity: An Acquired Form of Obesity Following Injury to the Hypothalamic Region
Hypothalamic Obesity: An Acquired Form of Obesity Following Injury to the Hypothalamic Region Craniopharyngioma and its treatment - tumor resection surgery and radiation - is considered the most common cause Clinical and pre-clinical data suggest MC4R pathway deficiency following injury to hypothala
Slide 16: Phase 2 Open-label Study Designed to Evaluate Setmelanotide's Effect on BMI in Hypothalamic Obesity
Phase 2 Open-label Study Designed to Evaluate Setmelanotide’s Effect on Body Mass Index in Patients with Hypothalamic Obesity Primary Endpoint: Proportion of patients who achieve at least 5% reduction from baseline in BMI at ~16 weeks of treatment PATIENTS 6-16 YEARS OLD 1 mg 2 mg 3 mg Primary Scree
Slide 17: Yann Mazabraud: International Update
Yann Mazabraud International Update ® 17
Slide 18: Rhythm's International Organization Designed to Achieve Market Access in Key Markets
Rhythm’s International Organization Designed to Achieve Market Access in Key Markets Experienced team across EU4 + UK + the Netherlands, the Nordics, Turkey and Argentina EU Rare Diseases community better organized • KOLs and Centers of Excellence • Rare diseases national plans and National Centers
Slide 19: Advancing Access to IMCIVREE in Europe and UK
Advancing Access to IMCIVREE in Europe and UK July 2021 European Commission July 2020 Authorization of IMCIVREE® for Submission of Marketing the Treatment of Obesity and Authorization Application to the Control of Hunger Associated European Medicines Agency for October 2021 with POMC, PCSK1 and LEPR
Slide 20: IMCIVREE Featured in EMA's Human Medicines Highlights of 2021
IMCIVREE Featured in EMA’s Human Medicines Highlights of 2021 - 92 positive EMA opinions in 2021 - 8 “outstanding contributions to public health” - Cancer: Abecma, Trodelvy - Gastroenterology/Hepatology: Bylvay - Immunology/Rheumatology/Transplantation: Tavneos, Voxzogo - Metabolism: Imcivree - Neur
Slide 21: First Commercial Sales Expected in Germany in Second Quarter 2022
First Commercial Sales Expected in Germany in Second Quarter 2022 • KOL support • Ongoing exclusion from Annex II (list of “lifestyle” indications and products –not reimbursed by health insurances) • German Federal Joint Committee (GB-A) plenary session on January 20th, 2022 – Strong vote in favor o
Slide 22: First Commercial Sales Expected in France in Second Quarter of 2022
First Commercial Sales Expected in France in Second Quarter of 2022 • KOL support • French Haute Autorité de Santé (HAS) granted paid early access for IMCIVREE – “AP2” granted for POMC & LEPR on January 19th, 2022 • Patients within label (> 6 years old) can be treated with IMCIVREE without limitatio
Slide 23: Additional Progress in Key International Markets on Track
Additional Progress in Key International Markets on Track United Kingdom • Selected for Highly Specialized Technology evaluation; Positive discussion at initial NICE Committee meeting in December 2021 Italy • Reimbursement dossier submitted to Italian Medicines Agency (AIFA) in July 2021 Spain • Rei
Slide 24: Advancing Submissions and Market Access Efforts for BBS in Europe
Advancing Submissions and Market Access Efforts for BBS in Europe CHMP decision expected 2H2022 More than Estimated European 1,500 prevalence estimated to be individuals identified in ~2,500* EU4 + UK patients with BBS at ~20 academic medical centers each with >40 BBS patients * Based on company est
Slide 25: Hunter Smith: 4Q/YE 2021 Financial Results
Hunter Smith 4Q/YE 2021 Financial Results ® 25
Slide 26: Q4 and FY 2021 Financial Highlights
Q4 and FY 2021 Financial Highlights Cash Expected to be Sufficient to Fund Operations into 2H 2023 ($ in millions except as noted, per share data Three months ended Twelve months ended and shares outstanding) December 31, 2021 December 31, 2021 Product revenue, net $1.8 $3.2 R&D expenses $31.6 $104.
Slide 27: David Meeker, MD: Conclusion
David Meeker, MD Conclusion ® 27
Slide 28: Progress Expected 2022
Progress Expected 2022 1H 2022 ✓ Initiate Phase 2 DAYBREAK trial ✓ Initiate Phase 3 “switch study” of weekly formulation ✓ Initiate Phase 3 trial in pediatric patients aged 2-6 years old Initiate Phase 3 EMANATE trial PDUFA for BBS and Alström syndrome June 16, 2022 Initial data from Phase 2 in MC4R
Slide 29: Questions
Questions Rhythar
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