BOSTON, June 03, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced the presentation of the first patient and caregiver reported experiences from qualitative interviews following the completion of a Phase 2 trial that evaluated treatment with setmelanotide in hypothalamic obesity (HO). These results were among six Rhy ...

BOSTON, June 03, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at Goldman Sachs 45th Annual Global Healthcare Conference on Monday, June 10, 2024 at 9:20 a.m. The fireside chat will be webcasted and available unde ...

BOSTON, June 03, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at Goldman Sachs 45th Annual Global Healthcare Conference on Monday, June 10, 2024 at 9:20 a.m. The fireside chat will be webcasted and available unde ...

"BBS is a debilitating rare disease that severely affects quality of life of patients and their families," said Philip Beales, M.D., UCL Great Ormond Street Institute of Child Health. "Hyperphagia – the feeling of extreme hunger that stays with patients all the time – leads to early-onset, life-long, severe obesity that affects many aspects of daily living. Until now there have been no licensed treatments for obesity and hyperphagia caused by BBS." The final NICE recommendation is aligned to the European Ma ...

Rhythm Pharmaceuticals, Inc (NASDAQ:RYTM) Q1 2024 Results Conference Call May 7, 2024 8:00 AM ET Company Participants David Connolly - Executive Director, IR and Corporate Communications David Meeker - Chairman, President, and CEO Jennifer Lee - Executive Vice President, Head of North America Yann Mazabraud - Executive Vice President, Head of International Hunter Smith - Chief Financial Officer Conference Call Participants Derek Archila - Wells Fargo Jeff Hung - Morgan Stanley Dae Gon Ha - Stifel Phil Nadea ...

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, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 222 Berkeley Street 12 th Floor Boston, MA 02116 (Address of Principal Executive Offices) (Zip Code) (857) 264-4280 (Registrant's Telephone Number, Including Area Code) N/A (Former N ...

[Overview and Key Highlights](index=1&type=section&id=Overview%20and%20Key%20Highlights) Rhythm Pharmaceuticals reported strong Q1 2024 results, with **$26.0 million** IMCIVREE net revenue, pipeline advancements, and **$150 million** financing extending operations into 2026 | Metric | Value | | :--- | :--- | | Q1 2024 IMCIVREE Net Revenue | $26.0 million | - The company is on track to submit a supplementary New Drug Application (sNDA) to the FDA in **Q2 2024** to expand IMCIVREE's label to pediatric patients aged **2 to younger than 6**[1](index=1&type=chunk)[3](index=3&type=chunk) - Phase 3 trial for setmelanotide in hypothalamic obesity progressing, with topline data expected in **H1 2025** and first patients in Japan anticipated in **Q2 2024**[1](index=1&type=chunk)[3](index=3&type=chunk) - Secured **$150 million** in convertible preferred stock financing, expected to fund operations into **2026**[1](index=1&type=chunk)[6](index=6&type=chunk) [Business and Operational Updates](index=1&type=section&id=Business%20and%20Operational%20Updates) The company reported strong IMCIVREE commercial performance and anticipates key clinical milestones, including new trial initiations and data readouts through early 2025 [Recent Business Highlights (Q1 2024)](index=1&type=section&id=Recent%20Business%20Highlights%20%28Q1%202024%29) Rhythm achieved significant commercial traction for IMCIVREE, presented positive setmelanotide data, secured **$150 million** financing, and initiated a Phase 1 trial for RM-718 - Approximately **100 new prescriptions** for IMCIVREE for Bardet-Biedl syndrome (BBS) written by U.S. prescribers, with about **70 receiving payor approval** during **Q1 2024**[4](index=4&type=chunk) - Phase 2 study of setmelanotide in hypothalamic obesity published, showing mean BMI reduction of **15% at 16 weeks** and approximately **26% at one year** in long-term extension[6](index=6&type=chunk) - Investment agreement for sale of series A convertible preferred stock for gross proceeds of **$150 million** announced, closing on **April 15, 2024**[6](index=6&type=chunk) - First patients dosed in **Phase 1 clinical trial of RM-718**, an investigational weekly MC4R agonist[6](index=6&type=chunk) [Anticipated Upcoming Milestones](index=2&type=section&id=Anticipated%20Upcoming%20Milestones) Rhythm anticipates key milestones including sNDA submission for IMCIVREE, new trial initiations, and data readouts from multiple studies through H1 2025 - **sNDA Submission:** Plan to submit an sNDA to the FDA for pediatric patients (**2 to <6 years old**) in **Q2 2024**, with potential EMA approval in **Q4 2024**[6](index=6&type=chunk) - **Clinical Trial Initiations:** Begin dosing in Japanese cohort of **Phase 3 hypothalamic obesity trial in Q2 2024** and **Phase 2 SIGNAL trial for LB54640 in Q3 2024**[6](index=6&type=chunk) - **Upcoming Data Readouts:** Phase 2 DAYBREAK study data in **Q3 2024**, Phase 1 RM-718 trial data in **H1 2025**, and top-line data from Phase 3 hypothalamic obesity trial in **H1 2025**[6](index=6&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) Rhythm reported increased Q1 2024 revenue and a higher net loss driven by R&D expenses, with a strong cash position bolstered by recent financing and clear FY2024 guidance [Q1 2024 Financial Results](index=2&type=section&id=Q1%202024%20Financial%20Results) Q1 2024 saw net product revenues more than double to **$26.0 million**, while R&D expenses surged to **$128.7 million** due to an acquisition, resulting in a **$141.4 million** net loss | Financial Metric | Q1 2024 | Q1 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | Net Product Revenues | $26.0M | $11.5M | +126% | | R&D Expenses | $128.7M | $37.9M | +239% | | S,G&A Expenses | $34.4M | $24.6M | +40% | | Net Loss | $(141.4)M | $(52.2)M | +171% | | Net Loss per Share | $(2.35) | $(0.92) | +155% | - Significant year-over-year increase in R&D expenses primarily due to **$92.4 million** in-process R&D costs for **LG Chem's compound LB54640 acquisition**[7](index=7&type=chunk) - Cash, cash equivalents, and short-term investments approximately **$201 million** as of **March 31, 2024**, a decrease from **$275.8 million** at year-end **2023**[5](index=5&type=chunk) [Financial Guidance (FY 2024) and Capital Position](index=3&type=section&id=Financial%20Guidance%20%28FY%202024%29%20and%20Capital%20Position) Rhythm projects FY2024 Non-GAAP Operating Expenses between **$250 million** and **$270 million**, with current cash and **$150 million** financing expected to fund operations into **2026** | FY 2024 Guidance | Amount | | :--- | :--- | | Non-GAAP Operating Expenses | $250M - $270M | - Existing cash as of **March 31, 2024**, combined with **$150 million** from preferred stock, expected to fund operations and capital expenditures into **2026**[9](index=9&type=chunk) [Financial Statements](index=8&type=section&id=Financial%20Statements) Condensed consolidated statements show a **$141.4 million** net loss for Q1 2024, driven by higher R&D, and a balance sheet reflecting **$258.7 million** total assets as of March 31, 2024 Condensed Consolidated Statements of Operations (in thousands) | | Three months ended March 31, 2024 | Three months ended March 31, 2023 | | :--- | :--- | :--- | | **Total revenues** | **$25,967** | **$11,469** | | Total costs and expenses | $165,854 | $64,000 | | Loss from operations | $(139,887) | $(52,531) | | **Net loss** | **$(141,372)** | **$(52,179)** | | **Net loss per share, basic and diluted** | **$(2.35)** | **$(0.92)** | Condensed Consolidated Balance Sheets (in thousands) | | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Cash and cash equivalents | $53,428 | $60,081 | | Short-term investments | $147,771 | $215,765 | | **Total assets** | **$258,651** | **$332,745** | | Total current liabilities | $54,160 | $55,203 | | **Total liabilities** | **$197,070** | **$162,986** | | **Total stockholders' equity** | **$61,581** | **$169,759** | [Product Information: IMCIVREE (setmelanotide)](index=4&type=section&id=Product%20Information%3A%20IMCIVREE%20%28setmelanotide%29) IMCIVREE (setmelanotide) is an MC4R agonist approved for chronic weight management in specific rare genetic obesity disorders, with detailed indications, limitations, and safety information - IMCIVREE indicated for chronic weight management in adult and pediatric patients (**6 years and older**) with obesity due to genetically confirmed **POMC, PCSK1, LEPR deficiency, or Bardet-Biedl syndrome (BBS)**[12](index=12&type=chunk)[13](index=13&type=chunk)[14](index=14&type=chunk) - Drug not indicated for other types of obesity, including **general (polygenic) obesity** or those not related to the **MC4R pathway**[21](index=21&type=chunk) - Key warnings include potential for **increased skin pigmentation** requiring annual full-body skin examinations, and need to monitor **heart rate and blood pressure**[15](index=15&type=chunk)[16](index=16&type=chunk) - Most common adverse reactions: **hyperpigmentation (51%)**, **injection site reaction (39%)**, **nausea (33%)**, and **headache (26%)**[20](index=20&type=chunk) [Other Information](index=4&type=section&id=Other%20Information) This section includes standard disclosures on forward-looking statements, defines Non-GAAP Operating Expenses, and provides conference call and investor relations contact information - Press release contains **forward-looking statements** concerning clinical trial progress, regulatory submissions, and financial performance, subject to risks[25](index=25&type=chunk) - **Non-GAAP Operating Expenses** defined as GAAP operating expenses excluding **stock-based compensation** and **fixed in-licensing consideration**, used for internal planning[26](index=26&type=chunk)[28](index=28&type=chunk) - Company hosted a conference call and webcast on **May 7, 2024, at 8:00 a.m. ET** to review financial results[10](index=10&type=chunk)

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 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 Other ...

Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) Q4 2023 Earnings Conference Call February 22, 2024 8:00 AM ET Company Participants David Connolly - Executive Director, IR and Corporate Communications David Meeker - Chairman, President, and CEO Jennifer Lee - Executive Vice President, Head of North America Hunter Smith - Chief Financial Officer Yann Mazabraud - Executive Vice President, Head of International Conference Call Participants Tazeen Ahmad - Bank of America Corinne Jenkins - Goldman Sachs Derek Archila ...

Exhibit 99.1 Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update -- Fourth quarter 2023 net revenue from global sales of IMCIVREE (setmelanotide) of $24.2 million -- -- Enrollment completed in 120-patient, pivotal cohort of Phase 3 setmelanotide trial in hypothalamic obesity; top-line data on track for 1H2025 -- -- Announced Phase 3 development plans for setmelanotide in hypothalamic obesity in Japan -- -- Spain and Italy authorities approve reimbursement for IMCIVREE to ...