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 ...

[PART I FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%20FINANCIAL%20INFORMATION) This section provides the unaudited condensed consolidated financial statements and management's discussion and analysis of financial condition and results of operations [Item 1. Condensed Consolidated Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements%20(Unaudited)) This section presents Rhythm Pharmaceuticals' unaudited condensed consolidated financial statements, including balance sheets, statements of operations, cash flows, and explanatory notes [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | September 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $64,593 | $127,677 | | Short-term investments | $234,667 | $205,611 | | Total current assets | $329,201 | $354,236 | | Total assets | $354,199 | $382,481 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $50,144 | $39,809 | | Deferred royalty obligation | $104,699 | $75,810 | | Total liabilities | $156,725 | $118,219 | | Total stockholders' equity | $197,474 | $264,262 | | Total liabilities and stockholders' equity | $354,199 | $382,481 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Product revenue, net | $22,504 | $4,284 | $53,194 | $8,094 | | Total revenues | $22,504 | $4,284 | $53,194 | $14,848 | | R&D Expenses | $33,570 | $21,116 | $105,059 | $85,082 | | SG&A Expenses | $30,475 | $21,938 | $85,158 | $65,715 | | Loss from operations | $(43,953) | $(39,267) | $(143,092) | $(137,054) | | Net loss | $(44,163) | $(40,861) | $(143,045) | $(138,626) | | Net loss per share | $(0.76) | $(0.79) | $(2.50) | $(2.73) | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for the Nine Months Ended September 30 (in thousands) | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(106,774) | $(139,428) | | Net cash (used in) provided by investing activities | $(28,305) | $68,804 | | Net cash provided by financing activities | $71,945 | $196,463 | | Net (decrease) increase in cash | $(63,084) | $125,839 | | Cash, cash equivalents and restricted cash at end of period | $64,921 | $185,415 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) - The company is a commercial-stage biopharmaceutical firm focused on its lead asset, IMCIVREE® (setmelanotide), a precision medicine for rare MC4R pathway diseases causing hyperphagia and severe obesity[19](index=19&type=chunk) - As of September 30, 2023, the company had an accumulated deficit of **$853.1 million** and expects operating losses to continue. Management believes existing cash, cash equivalents, and short-term investments of **$299.3 million** are sufficient to fund operations for at least the next twelve months[22](index=22&type=chunk)[23](index=23&type=chunk)[25](index=25&type=chunk) - On February 27, 2023, the company acquired Xinvento B.V. for approximately **$5.7 million**. The acquired In-Process Research and Development (IPR&D) assets were expensed as research and development[87](index=87&type=chunk)[90](index=90&type=chunk) - The company has a Revenue Interest Financing Agreement (RIFA) with HealthCare Royalty Management, LLC, accounted for as a deferred royalty obligation. As of September 30, 2023, the carrying value was **$104.7 million** with an effective interest rate of **15.43%**[115](index=115&type=chunk)[120](index=120&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=36&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management analyzes financial performance, highlighting IMCIVREE revenue growth, increased operating expenses, and a strong liquidity position expected to fund operations into 2026 [Overview and Recent Developments](index=38&type=section&id=Overview%20and%20Recent%20Developments) - The company focuses on advancing IMCIVREE® (setmelanotide) for rare MC4R pathway diseases, approved in the U.S., EU, and Great Britain for specific obesity indications[124](index=124&type=chunk) - Key clinical programs include a pivotal Phase 3 trial for hypothalamic obesity, with enrollment expected to complete in Q4 2023, and ongoing Phase 3 EMANATE and Phase 2 DAYBREAK trials[126](index=126&type=chunk)[129](index=129&type=chunk)[133](index=133&type=chunk) - Near-term milestones for Q4 2023 include completing enrollment in the hypothalamic obesity trial, submitting an IND for RM-718, and announcing data from several ongoing trials[143](index=143&type=chunk) - As of September 30, 2023, the company held approximately **$299.3 million** in cash, cash equivalents, and short-term investments, projected to fund operations into **2026**[146](index=146&type=chunk) [Results of Operations](index=50&type=section&id=Results%20of%20Operations) Comparison of Three Months Ended September 30, 2023 and 2022 (in thousands) | Metric | Q3 2023 | Q3 2022 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Product revenue, net | $22,504 | $4,284 | $18,220 | 425% | | Cost of sales | $2,412 | $497 | $1,915 | 385% | | Research and development | $33,570 | $21,116 | $12,454 | 59% | | Selling, general, and administrative | $30,475 | $21,938 | $8,537 | 39% | | Net loss | $(44,164) | $(40,861) | $(3,303) | 8% | - The **425% increase** in Q3 2023 product revenue was driven by continued sales growth of IMCIVREE, particularly following the FDA approval for BBS in June 2022[167](index=167&type=chunk) - Q3 2023 R&D expenses increased by **59% YoY**, primarily due to a **$5.3 million** increase in clinical trial costs for Phase 2 DAYBREAK, Phase 3 EMANATE, and the hypothalamic obesity trial, alongside higher personnel and preclinical research costs[169](index=169&type=chunk)[171](index=171&type=chunk) Comparison of Nine Months Ended September 30, 2023 and 2022 (in thousands) | Metric | 9M 2023 | 9M 2022 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Product revenue, net | $53,194 | $8,094 | $45,100 | 557% | | License revenue | $0 | $6,754 | $(6,754) | -100% | | Research and development | $105,059 | $85,082 | $19,977 | 23% | | Selling, general, and administrative | $85,158 | $65,715 | $19,443 | 30% | | Net loss | $(143,046) | $(138,626) | $(4,420) | 3% | - For the nine months ended Sep 30, 2023, R&D expenses increased by **23% YoY**, driven by a **$5.7 million** charge for IPR&D assets from the Xinvento acquisition, a **$6.6 million** increase in personnel costs, and a **$4.9 million** rise in clinical trial activity[179](index=179&type=chunk)[181](index=181&type=chunk) [Liquidity and Capital Resources](index=58&type=section&id=Liquidity%20and%20Capital%20Resources) - As of September 30, 2023, the company held cash, cash equivalents, and short-term investments totaling approximately **$299.3 million**[185](index=185&type=chunk) - Net cash used in operating activities for the first nine months of 2023 was **$106.8 million**, a decrease from **$139.4 million** in the same period of 2022, primarily due to higher revenues offsetting increased operating expenses[186](index=186&type=chunk)[188](index=188&type=chunk)[189](index=189&type=chunk) - Net cash provided by financing activities was **$71.9 million** for the first nine months of 2023, including **$48.9 million** from the ATM program, **$24.4 million** from the deferred royalty obligation, and **$3.5 million** from stock option exercises, offset by **$4.8 million** in royalty obligation repayments[193](index=193&type=chunk) - The company anticipates its existing cash and investments will be sufficient to fund operations into **2026**[196](index=196&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=64&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company reported no material changes to its market risk disclosures compared to the prior fiscal year-end - There were no material changes to the company's market risk disclosures as of September 30, 2023, compared to the end of 2022[210](index=210&type=chunk) [Item 4. Controls and Procedures](index=64&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective, with no material changes to internal control over financial reporting during the quarter - Management concluded that as of September 30, 2023, the company's disclosure controls and procedures were effective[212](index=212&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that have materially affected, or are reasonably likely to materially affect, internal controls[213](index=213&type=chunk) [PART II OTHER INFORMATION](index=65&type=section&id=PART%20II%20OTHER%20INFORMATION) This section covers legal proceedings, risk factors, equity security sales, and a list of exhibits filed with the report [Item 1. Legal Proceedings](index=65&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings - As of the filing date, the company is not involved in any material legal proceedings[215](index=215&type=chunk) [Item 1A. Risk Factors](index=65&type=section&id=Item%201A.%20Risk%20Factors) This section details significant risks, including financial position, development and commercialization of setmelanotide, regulatory compliance, and operational challenges [Risks Related to Financial Position and Need for Capital](index=65&type=section&id=Risks%20Related%20to%20Financial%20Position%20and%20Need%20for%20Capital) - The company has a limited operating history, incurred significant operating losses since inception (accumulated deficit of **$853.1 million** as of Sep 30, 2023), and anticipates continued losses[217](index=217&type=chunk)[222](index=222&type=chunk) - Additional funding will be required for clinical trials and commercialization, which may not be available on acceptable terms, potentially delaying or terminating development efforts[224](index=224&type=chunk)[231](index=231&type=chunk) - The Revenue Interest Financing Agreement (RIFA) could restrict IMCIVREE commercialization, limit cash flow, and dedicate a portion of cash flow to service the obligation[233](index=233&type=chunk)[236](index=236&type=chunk) [Risks Related to the Development of Setmelanotide](index=73&type=section&id=Risks%20Related%20to%20the%20Development%20of%20Setmelanotide) - Positive results from earlier clinical trials may not predict later trial outcomes, and success in one indication does not guarantee success in others[239](index=239&type=chunk)[241](index=241&type=chunk) - Target patient populations for MC4R pathway variants are small and not precisely established; smaller actual patient numbers could adversely affect revenue[251](index=251&type=chunk) - Setmelanotide may cause undesirable side effects (e.g., cardiovascular effects, skin darkening) that could delay or prevent regulatory approvals or limit its commercial profile[280](index=280&type=chunk)[284](index=284&type=chunk) - Reliance on third-party CROs for clinical trials means their failure to perform successfully could delay or harm regulatory approvals and commercialization[316](index=316&type=chunk) [Risks Related to the Commercialization of IMCIVREE (setmelanotide)](index=101&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20IMCIVREE%20(setmelanotide)) - Commercial success depends on obtaining and maintaining adequate coverage and reimbursement from government and private payors, a time-consuming and uncertain process[321](index=321&type=chunk)[322](index=322&type=chunk)[324](index=324&type=chunk) - Market acceptance of IMCIVREE is not guaranteed, depending on factors like physician and patient acceptance, administration convenience (SC injection), and competition from other obesity therapies[334](index=334&type=chunk)[335](index=335&type=chunk) - Complete reliance on third-party CMOs for setmelanotide manufacture means any non-compliance with GMP or failed regulatory inspections could delay or halt production and supply[346](index=346&type=chunk)[348](index=348&type=chunk) - The company's termination of its license agreement with RareStone Group for cause could delay or terminate development and commercialization in China, Hong Kong, and Macao[353](index=353&type=chunk)[355](index=355&type=chunk) [Risks Related to Regulatory Approval and Marketing of Setmelanotide and Other Legal Compliance Matters](index=125&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20and%20Marketing%20of%20Setmelanotide%20and%20Other%20Legal%20Compliance%20Matters) - The regulatory approval process for additional indications is expensive, time-consuming, and uncertain, with failure materially impairing revenue generation[395](index=395&type=chunk) - FDA and other agencies actively enforce laws prohibiting off-label promotion; non-compliance could result in significant penalties and harm the business[445](index=445&type=chunk)[447](index=447&type=chunk) - The company is subject to numerous federal, state, and foreign healthcare laws (e.g., Anti-Kickback Statute, False Claims Act); non-compliance could lead to criminal sanctions, civil penalties, and exclusion from government programs[451](index=451&type=chunk) - The evolving global data protection landscape (e.g., GDPR, CCPA/CPRA) means failure to comply with privacy and security laws could result in government enforcement, significant penalties, and reputational damage[458](index=458&type=chunk)[461](index=461&type=chunk)[463](index=463&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=177&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%2C%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases during the period - The company reports no unregistered sales of equity securities or issuer purchases of its equity securities during the period[534](index=534&type=chunk)[536](index=536&type=chunk) [Item 6. Exhibits](index=178&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including amended executive offer letters and Sarbanes-Oxley Act certifications - Exhibits filed include amended and restated offer letters for executives David P. Meeker, Joseph Shulman, Jennifer Chien, and Hunter Smith, as well as CEO and CFO certifications[540](index=540&type=chunk)