Rhythm Pharmaceuticals, Inc. · NASDAQ:RYTM
Rhythm Pharmaceuticals — Investor Presentation (January 2019)
29 slides from Rhythm Pharmaceuticals's investor presentation, Jan 8, 2019
Slide 1: 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 state
Slide 2: Transforming the Care of Patients with Genetic Obesity
Transforming the Care of Patients with Genetic Obesity The MC4R pathway is the key pathway regulating weight and hunger. Genetic defects in the MC4R pathway cause insatiable hunger, leading to severe and early-onset obesity There are currently no effective or approved treatments for people living wi
Slide 3: Meet Katy: Living with an MC4R Pathway Disorder
Meet Katy: Living with an MC4R Pathway Disorder “It causes extreme unrelenting hunger and excessive eating. As a child the fridge and food was controlled massively but nobody could understand that I was desperately hungry and just wanted to stop that feeling.” INFANCY: “Normal” weight at birth, but
Slide 4: Setmelanotide Rescues the Impaired MC4R Pathway Upstream
Setmelanotide Rescues the Impaired MC4R Pathway Upstream XXXX XXXX XXXX XXXX XXXX XXXX XXXX XXXX ALMS1 BBSx LEPR PCSK1 POMC LEPR deficiency Alström syndrome POMC deficiency Bardet-Biedl syndrome Homozygous, compound heterozygous, heterozygous, epigenetic dysfunction MC4R pathway genes Activation Dow
Slide 5: Setmelanotide: Potential First-in-Class Replacement Therapy for MC4R Pathway Disorders
Setmelanotide: Potential First-in-Class Replacement Therapy for MC4R Pathway Disorders
Slide 6: Overview of Setmelanotide
Setmelanotide: 8 amino acid peptide with high potency (EC50 0.27nM) Administered by once daily subcutaneous injection Retains the specificity and functionality of the naturally-occurring hormone Once-weekly formulation in clinical development Developed in partnership with Camurus Terminal half-life
Slide 7: Basket Study: Foundation for Achieving POC in MC4R Pathway Disorders
Basket Study: Foundation for Achieving POC in MC4R Pathway Disorders Indications enrolled in Phase 2: POC Achieved, Phase 3 initiated: POMC, LEPR, BBS & Alström Syndrome Pre-POC, Phase 2 ongoing: MC4R pathway heterozygous obesity, POMC epigenetic obesity New Indications: Additional genetic targets u
Slide 8: POMC and LEPR Phase 2 Studies: Patients #1
1.5 mg 1.5 mg 1.5 mg 1.0 POMC and LEPR Phase 2 Studies: Patients #1 * Figures represent cumulative weight lost in kgs Not all patients had similar responses (1) Kühnen, et. al, Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist. N Engl J Med. July 2016 . Yellow vertical ba
Slide 9: BBS and Alström Syndrome Phase 2 Studies: Patients #1
Weight Change (kg) Hunger Score (0-10 points) BBS and Alström Syndrome Phase 2 Studies: Patients #1 * Figures represent cumulative weight lost in kgs Not all patients had similar responses (1) Haws et al. Effect of the Melanocortin-4 Receptor Agonist Setmelanotide on Obesity and Hyperphagia in Indiv
Slide 10: Phase 2 BBS and Alström Results Support Advancement to Phase 3
Phase 2 BBS and Alström Results1 Support Advancement to Phase 3 (1) Haws RM et al. Effect of the Melanocortin-4 Receptor Agonist Setmelanotide on Obesity and Hyperphagia in Individuals Affected by Bardet-Biedl Syndrome. ESPE 2018. Abstract RFC6.3. Updated data for pts. 6 & 7 announced by Rhythm in J
Slide 11: Additional Indications in Phase 2 Basket Study
Additional Indications in Phase 2 Basket Study Heterozygous and Epigenetic Obesity Disorders Focus on most debilitated patients MC4R pathway heterozygous deficiency: one loss of function allele POMC epigenetic disorders: disease caused by DNA modifications that can change gene expression without alt
Slide 12: Setmelanotide Has Been Generally Well-Tolerated
Setmelanotide has been evaluated in over 300 obese subjects Setmelanotide has been generally well-tolerated Most AEs are mild and non-mechanism based: Mild injection site reactions Darkening of skin (tanning) and skin lesions, mediated by the closely related MC1 receptor (the natural “tanning” recep
Slide 13: Ongoing Pivotal Phase 3 Trials in POMC, LEPR and BBS/Alström
Ongoing Pivotal Phase 3 Trials in POMC, LEPR and BBS/Alström All pivotal trials share a common design: Patients aged > 6 ~1-year trial duration, with long-term extension Dosage: Daily subcutaneous injection 1o endpoint: categorical analysis of responders(1) Key 2o endpoints include mean weight loss
Slide 14: Building a Community for the Treatment of Rare Genetic Disorders of Obesity
Building a Community for the Treatment of Rare Genetic Disorders of Obesity
Slide 15: Epidemiology Suggests Significant Market Opportunity
Epidemiology Suggests Significant Market Opportunity GENETIC EPIDEMIOLOGY(2) ~13,000 individuals in the U.S. with POMC or LEPR -- a five-fold increase over the clinical epidemiology estimate. CLINICAL EPIDEMIOLOGY Up to 5,000 patients in U.S.(1) * The patient numbers above are based on company estim
Slide 16: Rhythm-Sponsored Initiatives to Expand Addressable Patient Population
Rhythm-Sponsored Initiatives to Expand Addressable Patient Population Identifies individuals with rare genetic disorders of obesity who may be eligible for further study and treatment with setmelanotide . Rhythm-Sponsored Phase 2 Basket Study Designed to facilitate rapid progress to proof of concept
Slide 17: GO-ID Genotyping Initiative
GO-ID Genotyping Initiative Genotyping study of individuals with obesity >2 years of age Identifies individuals with rare genetic disorders of obesity who may be eligible for further study and treatment with setmelanotide . Recruitment via outreach to HCPs caring for patients with severe obesity Exp
Slide 18: Community Building Through the TEMPO Registry
Community Building Through the TEMPO Registry Commitment to understanding the ongoing impact and burden of disease on patients and their caregivers Facilitates enhanced understanding of these conditions in the medical community Builds upon ongoing collaborations with existing patient registries (e.g
Slide 19: Rhythm Achieved Key Milestones in 2018
Rhythm Achieved Key Milestones in 2018 Completed enrollment in POMC & LEPR Phase 3 trials Announced updated positive Phase 2 BBS & Alström results Breakthrough Therapy and EMA Prime designation expanded to include BBS & Alström Enrolled first patient in pivotal Phase 3 study in BBS & Alström Advance
Slide 20: Rhythm Expects Significant Growth in 2019 and 2020
Rhythm Expects Significant Growth in 2019 and 2020 1Q19 Updated interim data for MC4R pathway heterozygous and epigenetic obesity disorders 3Q19 Topline data from both POMC and LEPR Phase 3 studies 4Q19-1Q20 Initial NDA submission filings for setmelanotide in POMC and LEPR 2H19 Complete pivotal enro
Slide 21: Living with the Insatiable Hunger and Obesity that Characterize BBS
Living with the Insatiable Hunger and Obesity that Characterize BBS “As our daughter Lucy grows older, the weight obstacle of BBS looms more ominously before us, hindering much of what we do. Lucy measures most of her activities and much of her happiness around when her next meal is, what food is av
Slide 22: Living with the Insatiable Hunger and Obesity that Characterize BBS
Living with the Insatiable Hunger and Obesity that Characterize BBS “As our daughter Lucy grows older, the weight obstacle of BBS looms more ominously before us, hindering much of what we do. Lucy measures most of her activities and much of her happiness around when her next meal is, what food is av
Slide 23: Rhythm Pipeline
Indication PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 SETMELANOTIDE POMC Deficiency Obesity LEPR Deficiency Obesity Bardet-Biedl Syndrome Alström Syndrome POMC & Other MC4R Pathway Heterozygous Deficiency Obesities POMC Epigenetic Disorders RM-853 (GHRELIN O-ACYLTRANSFERASE INHIBITOR) Prader-Willi Syndrom
Slide 24: Bardet Biedl & Alström Syndrome: Phase 3 Study Design
Setmelanotide 3 mg Placebo Double-blind treatment (Period 1) 14 weeks Dose titration Phase (2 weeks) Long-Term Extension Phase Screening 2-3 weeks Week 0 Primary endpoint assessed Extension study Week 52 38 weeks Setmelanotide 3 mg Open-label treatment (Period 2) Week 14 14 weeks Setmelanotide 3 mg
Slide 25: Phase 3 Study Update
Phase 3 Study Update Protocol Final comments from FDA have been incorporated Study enrollment milestones First patient with BBS enrolled Study enrollment goals Enrollment minimum: 20 BBS patients 6 Alström Syndrome patients Centers of excellence Marshfield Clinic Health System Bob Haws, MD Universit
Slide 26: RM-853: Potent, Orally Available GOAT Inhibitor for PWS
RM-853: Potent, Orally Available GOAT Inhibitor for PWS GOAT is key enzyme involved in producing active ghrelin Blocking GOAT results in: Lower levels of active ghrelin, and Increased levels of DAG, a ghrelin precursor believed to have independent beneficial effects In preclinical studies with high
Slide 27: Cash expected to be sufficient to Fund Operations into 2H '20
Cash expected to be sufficient to Fund Operations into 2H ‘20 SHARES OUTSTANDING as of 9/30/2018 34,382,525 (basic) 36,857,315 million (fully-diluted) $ 272.4 million CASH, CASH EQUIVALENTS AND SHORT TERM INVESTMENTS as of 9/30/2018 Raised $163M in net proceeds in June 2018 follow-on offering
Slide 28: Cash expected to be sufficient to Fund Operations into 2H '20
Cash expected to be sufficient to Fund Operations into 2H ‘20 34,382,525 (basic) 36,857,315 million $ 272.4 million (fully-diluted) Raised $163M in net proceeds in June 2018 follow-on offering
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