Rhythm Pharmaceuticals : RYTM 1Q2024 vFinal

Rhythm Pharmaceuticals

First Quarter 2024 Financial Results

and Business Update

May 7, 2024

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© Rhythm® Pharmaceuticals, Inc. All rights reserved.

On Today's Call

David Connolly, Executive Director of Investor Relations and Corporate Communications David Meeker, MD, Chair, President and Chief Executive Officer

Jennifer Lee, Executive Vice President, Head of North America

Yann Mazabraud, Executive Vice President, Head of International

Hunter Smith, Chief Financial Officer

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Forward-looking Statements

This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the safety, efficacy, and regulatory and clinical design or progress, potential regulatory submissions, approvals and timing thereof of any of our products and product candidates, including setmelanotide and LB54640, including our Phase 3 trial of setmelanotide for patients with hypothalamic obesity in Japan, the United States or in Europe, the potential benefits of setmelanotide for patients with hypothalamic obesity, our expectations surrounding potential regulatory submissions, approvals and timing thereof, including the IND application for RM-718, the Company's business strategy and plans, including regarding commercialization of setmelanotide, expectations surrounding sales and reimbursement of IMCIVREE, our anticipated financial performance and financial position, including estimated Non-GAAP Operating Expenses for the year ending December 31, 2024, the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word such as "expect", "anticipate", "believe", "may", "will", "aim" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the ability to achieve necessary regulatory approvals, risks associated with data analysis and reporting, failure to identify and develop additional product candidates, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, the impact of competition, risks relating to product liability lawsuits, inability to maintain collaborations, or the failure of these collaborations, our reliance on third parties, risks relating to intellectual property, our ability to hire and retain necessary personnel, general economic conditions, risks related to internal control over financial reporting, and the other important factors discussed under the caption "Risk Factors" in our Form 10-K for the year ended December 31, 2023 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this presentation or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise.

Non-GAAP Financial Measures

This presentation and the accompanying oral presentation includes Non-GAAP Operating Expenses, a supplemental measure of our performance that is not required by, or presented in accordance with, U.S. GAAP and should not be considered as an alternative to operating expenses or any other performance measure derived in accordance with GAAP. We define Non-GAAP Operating Expenses as GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing. We caution investors that amounts presented in accordance with our definition of Non-GAAP Operating Expenses may not be comparable to similar measures disclosed by our competitors because not all companies and analysts calculate this non-GAAP financial measure in the same manner. We have not provided a quantitative reconciliation of forecasted Non- GAAP Operating Expenses to forecasted GAAP operating expenses because we are unable, without making unreasonable efforts, to calculate the reconciling item, stock-based compensation expenses, with confidence. This item, which could materially affect the computation of forward-looking GAAP operating expenses, is inherently uncertain and depends on various factors, some of which are outside of our control.

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David Meeker, MD

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Rhythm's Value Drivers

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BBS global commercial execution

Hypothalamic obesity offers a significant expansion opportunity

Advancing next generation assets LB54640 and RM-718 with IP to 2040s

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Results from Phase 2 Study of Setmelanotide for the Treatment of Hypothalamic Obesity Published in The Lancet D&E

16 of 18

patients achieved primary endpoint

of >5% reduction in BMI

(P<0.0001)

−14.5%

mean change

in BMI at 16 weeks

14 of 18

patients achieved

>10% reduction in BMI

-25.5%

mean percent reduction

in BMI at Month 12

from baseline

DOI:https://doi.org/10.1016/S2213-8587(24)00087-1

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Jennifer Lee

BBS U.S. Launch

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Steady Progress with First Quarter 2024 Prescriptions and Approvals for Reimbursement

	~100		~70
	Prescriptions		Approvals for
	received during		reimbursement
	Q1 2024		during Q1 2024

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Increased Depth and Breadth of BBS IMCIVREE Prescriber Base

>425	>30%
Prescribers
launch	>30%
to date*

of prescribers

'new to Rhythm'*

of prescribers have written more than one prescription*

*As of March 31, 2024

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Access and Reimbursement Remains Steady and Consistent

~90%		>85%		>140
of reimbursed		of Medicaid
prescriptions fall		covered lives in		approvals for
under commercial		states with access		reauthorization*
and state Medicaid		achieved for
plans*		IMCIVREE*

*As of March 31, 2024

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