Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-38223 RHYTHM PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 46-2159271 (State or ot ...

• IMCIVREE now commercially available in Canada, GCC states; German BBS launch underway • 12-month data planned for fall conference • Additional detail on development programs at 4Q R&D event 6 Investigational New Drug (IND) application on track to be filed by end of 2023 *Includes patent term adjustment and patent term extension. Heterozygous POMC/PCSK1 insufficiency *Estimated prevalence of U.S. patients based on company estimates; does not include ex-U.S. prevalence estimates. 53,000 patients in the U.S. ...

Start Time: 08:00 January 1, 0000 8:51 AM ET Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) Q2 2023 Earnings Conference Call August 01, 2023, 08:00 AM ET Company Participants David Meeker - Chairman, President, and CEO Jennifer Chien - EVP, Head of North America Yann Mazabraud - EVP, Head of International Hunter Smith - CFO David Connolly - Executive Director, IR and Corporate Communications Conference Call Participants Divya Rao - TD Cowen Derek Archila - Wells Fargo Corinne Jenkins - Goldman Sachs Dae Gon Ha ...

® 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 Smith, Chief Financial Officer ® May 2, 2023 On Today's Call Important Notice | --- | --- | |-------|------------------| | | | | | | | | | | | | | | | | | | | | | | | David Meeker, MD | | | | | | | | 4 | ! Rhythm® | • Strong s ...

Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) Q1 2023 Earnings Conference Call May 2, 2023 8:00 AM ET Company Participants David Connolly - Executive Director of Investor Relations and Corporate Communications David Meeker - Chairman, President, and Chief Executive Officer Jennifer Chien - Executive Vice President, Head of North America Yann Mazabraud - Executive Vice President, Head of International Hunter Smith - Chief Financial Officer Conference Call Participants Phil Nadeau - TD Cowen Derek Archila - Well ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-38223 RHYTHM PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 46-2159271 (State or o ...

Financial Performance - The company has generated less than $25.0 million in revenue from product sales since its inception[290]. - Net losses for the years ended December 31, 2022, and 2021 were $181.1 million and $69.6 million, respectively, with an accumulated deficit of $710.1 million as of December 31, 2022[293]. - The company has incurred significant operating losses since its inception and anticipates continued losses for the foreseeable future[289]. - The company may need to raise additional funding to support ongoing operations and product development, which may not be available on acceptable terms[297]. Research and Development - The company expects significant increases in research and development expenses due to ongoing clinical trials for setmelanotide and commercialization efforts[295]. - The company has focused its efforts on the research and development of setmelanotide, which is approved for specific obesity-related conditions[292]. - The company has approximately 60,000 genetic sequencing samples in its database as of December 31, 2022, which aids in estimating patient populations for MC4R pathway deficiencies[324]. - Setmelanotide is being developed as a treatment for obesity caused by genetic deficiencies affecting the MC4R pathway, with clinical trials ongoing[390]. Clinical Trials and Regulatory Challenges - The company has eliminated a fifth sub-study in its Phase 3 EMANATE clinical trial based on FDA feedback, focusing on four independent sub-studies[314]. - The clinical response rate for patients carrying pathogenic or likely pathogenic variants is estimated at 40%, while it is 20% for those with variants of uncertain significance[333]. - The company acknowledges that setbacks in clinical trials could adversely affect its business and financial prospects[317]. - The company faces competition in clinical trials for obesity-related product candidates, which may limit patient enrollment due to overlapping trial sites and limited patient pools[336]. - Challenges in enrolling pediatric patients for setmelanotide trials may lead to significant delays or abandonment of clinical trials, impacting development costs and marketing approvals[338]. - Delays in clinical trials could increase costs and hinder the ability to generate revenue, affecting the overall business[339]. - The company may encounter various delays in clinical trials due to regulatory approvals, patient recruitment, and other operational challenges[340]. - The COVID-19 pandemic has adversely impacted clinical trials, leading to potential delays in approvals, patient enrollment, and site initiation[350]. Market Exclusivity and Competition - Setmelanotide has received seven years of orphan drug exclusivity in the U.S. for chronic weight management in patients aged 6 and older with obesity due to specific genetic deficiencies[375]. - In the EU, setmelanotide has been granted ten years of market exclusivity for treating obesity associated with genetically confirmed loss-of-function biallelic POMC and LEPR deficiencies in adults and children aged 6 and above[375]. - The company faces intense competition in the biotechnology and pharmaceutical industries, with established competitors having greater resources and market presence[418]. - Market acceptance of IMCIVREE is contingent on its ability to demonstrate chronic weight management and provide incremental health benefits compared to existing treatments[416]. Manufacturing and Supply Chain - Manufacturing of setmelanotide relies entirely on third-party suppliers, with ongoing risks related to compliance with regulatory requirements and potential supply chain disruptions[424]. - The company currently has sufficient finished setmelanotide for ongoing clinical trials and commercial supply, but manufacturing delays could impact revenue generation[430]. - The COVID-19 pandemic has disrupted supplier activities, potentially affecting the supply chain for setmelanotide[432]. - The company has entered into long-term commercial supply agreements with PolyPeptide Group and Recipharm Monts S.A.S. for the manufacturing of IMCIVREE[428]. Intellectual Property Risks - The company faces risks related to intellectual property rights, including the potential inability to protect proprietary technology and maintain issued patents, which could adversely affect business operations[440]. - There is uncertainty regarding the validity and enforceability of patents covering setmelanotide, which could lead to significant competitive disadvantages[442]. - The company may be involved in costly and time-consuming litigation to protect its patents, which could distract management and impact financial resources[446]. - The outcome of legal challenges to patents covering setmelanotide is unpredictable, and a loss of patent protection could have a material adverse impact on the business[460]. Regulatory Approval and Compliance - The regulatory approval process for setmelanotide is complex and may delay commercialization, impacting revenue generation[475]. - The FDA may require additional studies or post-market requirements to assess potential serious risks associated with setmelanotide, which could increase commercialization costs[362]. - The company is required to identify patients with unique genetic subtypes for effective treatment, which may necessitate the approval of an in vitro companion diagnostic device[389]. - Regulatory authorities may request withdrawal of setmelanotide from the market or impose additional labeling requirements if undesirable side effects are identified[362]. Commercialization and Market Access - The successful commercialization of IMCIVREE depends on obtaining adequate coverage and reimbursement from governmental authorities and third-party payors[401]. - Pricing negotiations for IMCIVREE in foreign countries can take 6 to 12 months or longer after regulatory approval, affecting market entry[405]. - The EU member states can restrict the range of medicinal products for which national health insurance provides reimbursement, impacting market access[406]. - Health Technology Assessment (HTA) is becoming common in the UK and EU for pricing and reimbursement procedures, influencing the status of medicinal products[409]. Future Considerations - The company has not yet established a full-scale commercial infrastructure for IMCIVREE, which may hinder revenue generation in the near term[411]. - The company may need to engage additional third-party suppliers to meet future manufacturing needs as it scales up production[433]. - The termination of the exclusive license agreement with RareStone Group Ltd. may delay or terminate the development and commercialization of setmelanotide and IMCIVREE in certain markets[434]. - RareStone has materially breached its obligations under the license agreement, including funding and performing key clinical studies, which could significantly impact product development[436].

Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) Q4 2022 Earnings Conference Call March 1, 2023 8:00 AM ET Company Participants David Connolly - Investor Relations and Corporate Communications David Meeker - Chairman, President and Chief Executive Officer Jennifer Chien - Executive Vice President, Head of North America Yann Mazabraud - Executive Vice President, Head of International Hunter Smith - Chief Financial Officer Conference Call Participants Philip Nadeau - Cowen and Company Derek Archila - Wells Fargo Se ...

Rhythm Pharmaceuticals Important Notice | --- | --- | |-------|------------------| | | | | | | | | | | | | | | | | | | | | | | | David Meeker, MD | | | | | | | | 4 | ! Rhythm® | Global commercial strategy execution • >100 reimbursement approvals 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 Int ...

Transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare MC4R pathway diseases by rapidly advancing care and precision medicines addressing the root cause Forward Looking Statements ® ✓ Granted EC marketing authorization for IMCIVREE for BBS; received early access authorization in France ✓ Completed enrollment in Phase 3 trial in pediatric patients ✓ Entered non-dilutive RIFA agreement with HealthCare Royalty for up to $100 million Phase 3 weekly formul ...