Core Viewpoint - Rhythm Pharmaceuticals has received FDA approval for an expanded indication of IMCIVREE (setmelanotide) to treat obesity in children as young as 2 years old, specifically for those with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), PCSK1, or LEPR deficiencies [5][22][29] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, headquartered in Boston, MA [3][7] - The company is committed to advancing care and precision medicines that address the root causes of these diseases [7] Product Information - IMCIVREE is the first and only precision medicine targeting the hypothalamic MC4R pathway, addressing hyperphagia and obesity in patients with BBS and POMC, PCSK1, or LEPR deficiencies [14] - The drug is indicated for long-term weight reduction in patients aged 2 years and older [17][22] Clinical Development - Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases and investigational MC4R agonists [3] - Results from the Phase 3 VENTURE trial indicate significant and sustained reductions in weight and hunger [31] Market Impact - The approval is seen as a significant advancement for the BBS community, providing a targeted treatment option for children suffering from insatiable hunger and obesity [15] - Approximately 4,000 to 5,000 people in the U.S. are affected by BBS, while POMC, PCSK1, and LEPR deficiencies affect around 600 to 2,500 individuals [16]

Core Insights - Rhythm Pharmaceuticals announced positive results from its Phase 3 VENTURE trial for setmelanotide, targeting severe obesity in children under 5 with specific genetic deficiencies [1][2] - The trial showed significant reductions in body mass index (BMI) and hunger levels among participants, indicating the potential effectiveness of setmelanotide in this demographic [2][3] Company Overview - Rhythm Pharmaceuticals is a biopharmaceutical company focused on rare neuroendocrine diseases, with its lead product, IMCIVREE (setmelanotide), approved for chronic weight management in patients with specific genetic obesity conditions [5][6] - Setmelanotide has received regulatory approvals in both the U.S. and the European Union for treating obesity and controlling hunger in genetically confirmed cases of Bardet-Biedl syndrome (BBS) and other deficiencies [4][7] Clinical Trial Results - The Phase 3 VENTURE trial enrolled 12 patients, with 83% achieving a ≥0.2-point reduction in BMI Z-score and an 18% mean percent change in BMI from baseline [2][3] - Caregivers reported a 91% reduction in hunger among patients, alongside a decrease in personal burden [2] Safety and Tolerability - Setmelanotide was generally well tolerated, with no serious adverse events leading to discontinuation or death reported [3] - Common treatment-emergent adverse events included skin hyperpigmentation (75%), vomiting (58%), and nasopharyngitis (42%) [3][16] Regulatory Developments - Rhythm has submitted a supplemental New Drug Application (sNDA) to the FDA to expand the label of IMCIVREE for pediatric patients aged 2 to under 6 years [4] - The FDA has granted Priority Review for the sNDA, with a Prescription Drug User Fee Act (PDUFA) goal date set for December 26, 2024 [4]

Sales Performance - IMCIVREE global sales revenue reached $33.3 million in Q3 2024, a 14% increase sequentially from Q2 2024, with 70% generated in the U.S. and 30% outside the U.S.[145] - Product revenue increased by $10.7 million to $33.3 million for the three months ended September 30, 2024, representing a 48% increase compared to the same period in 2023[175] - Product revenue increased by $35.1 million to $88.3 million for the nine months ended September 30, 2024, representing a 66% increase compared to the same period in 2023[185] - IMCIVREE, the lead product candidate, generated approximately $185.8 million in product revenue since its approval in November 2020[160] Clinical Trials and Research - The Phase 3 pediatrics trial for setmelanotide showed a mean reduction of 3.04 in BMI-Z score and an 18.4% mean reduction in BMI among patients aged 2 to 6 with BBS or POMC/LEPR deficiencies[143] - The company has completed enrollment in its Phase 3 trial for acquired hypothalamic obesity and is advancing multiple clinical trials for new drug candidates RM-718 and bivamelagon (LB54640)[143] - Real-world data indicated that adult patients with acquired hypothalamic obesity achieved a mean BMI reduction of 12.8% at three months on setmelanotide therapy[147] - The Phase 2 DAYBREAK trial showed that 84% of patients on continuous setmelanotide therapy maintained a >5% BMI reduction over 40 weeks[148] - The company is leveraging a DNA database with nearly 80,000 sequencing samples to enhance understanding and treatment of severe obesity related to MC4R pathway variants[144] Financial Overview - As of September 30, 2024, the accumulated deficit was $1.1 billion, with a net loss of $43.6 million for the three months ended September 30, 2024[156] - Existing cash and cash equivalents and short-term investments were approximately $298.4 million as of September 30, 2024, expected to fund operations into 2026[157] - Net cash used in operating activities was $95.0 million for the nine months ended September 30, 2024, compared to $106.8 million in the same period in 2023[196][197] - Net cash provided by financing activities was $152.9 million for the nine months ended September 30, 2024, primarily from the issuance of Series A Preferred Stock[200] - The company raised approximately $791.5 million through common stock offerings since its IPO in October 2017, and $150 million from a recent investment agreement[152][153] Expenses and Cost Management - The cost of sales rose by $1.4 million to $3.8 million for the three months ended September 30, 2024, an increase of 59% driven by higher sales volume[178] - Research and development expenses increased by $4.4 million to $37.9 million for the three months ended September 30, 2024, a 13% increase from the same period in 2023[179] - Selling, general and administrative expenses increased by $4.9 million to $35.4 million for the three months ended September 30, 2024, reflecting a 16% increase compared to the same period in 2023[181] - Research and development expenses rose by $91.7 million to $196.8 million for the nine months ended September 30, 2024, an increase of 87% primarily due to acquired in-process research and development costs[187] - Selling, general, and administrative expenses increased by $21.0 million to $106.2 million for the nine months ended September 30, 2024, reflecting a 25% increase driven by higher salaries and marketing costs[190] Future Outlook and Funding - The company anticipates continued significant expenses and operating losses due to ongoing clinical trials and expansion of commercialization efforts[156] - The company expects to incur significant commercialization expenses related to product sales and marketing as it continues to develop its product candidates[202] - The existing cash and cash equivalents are expected to fund operations into 2026, but additional funding may be required for ongoing research and development activities[203][204] - The company expects to finance cash needs through equity offerings, debt financings, collaborations, strategic alliances, and licensing arrangements due to the current economic volatility[207] - Future funding requirements may be impacted by economic conditions, including rising interest rates and declines in consumer confidence[207] Regulatory and Market Considerations - The FDA accepted the supplemental New Drug Application for IMCIVREE to treat children as young as 2 years old, with a PDUFA goal date set for December 26, 2024[150] - IMCIVREE was approved by the FDA and became commercially available in Q1 2021, but its commercial success remains uncertain[206] - The company may face dilution of stockholder ownership if additional capital is raised through equity or convertible debt securities[208] - The company may need to delay or limit product development if unable to raise additional funds through equity or debt financings[209] - The company has no material changes to its quantitative and qualitative disclosures about market risks as of September 30, 2024[214]

BOSTON, Nov. 06, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in fireside chats at two upcoming investor conferences: Guggenheim Securities Healthcare Innovation Conference on Monday, November 11 at 11:30 a.m. ET Stifel 2024 Healthcare Confere ...

Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) Q3 2024 Earnings Conference Call November 5, 2024 5:00 PM ET Company Participants David Connolly - Head of Investor Relations and Corporate Communications David Meeker - Chairman, President and Chief Executive Officer Jennifer Lee - Executive Vice President and Head of North America Yann Mazabraud - Executive Vice President and Head of International Hunter Smith - Chief Financial Officer Conference Call Participants Philip Nadeau - TD Cowen Corinne Johnson - Goldma ...

Rhythm Pharmaceuticals Third Quarter 2024 Financial Results 。 and Business Update ....... November 5, 2024 · · · · · . . . . . . . . . . . . . . . . · · . . C ●●●●●●●●●●●● ............ © Rhythm® Pharmaceuticals, Inc. All rights reserved. • 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 Inter ...

Rhythm Pharmaceuticals, Inc. (RYTM) came out with a quarterly loss of $0.73 per share versus the Zacks Consensus Estimate of a loss of $0.80. This compares to loss of $0.76 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 8.75%. A quarter ago, it was expected that this company would post a loss of $0.70 per share when it actually produced a loss of $0.55, delivering a surprise of 21.43%.Over the last four quarters, the company ...

Exhibit 99.1 Rhythm Pharmaceuticals Reports Third Quarter 2024 Financial Results and Business Update -- Third quarter 2024 net revenue from global sales of IMCIVREE (setmelanotide) of $33.3 million -- -- Adult patients with acquired hypothalamic obesity (N=8) achieved mean BMI reduction of 12.8% on setmelanotide therapy at three months in French early-access program -- -- Top-line data readout for Phase 3 trial evaluating setmelanotide in 120 patients with acquired hypothalamic obesity on track for 1H 2025 ...

-- Third quarter 2024 net revenue from global sales of IMCIVREE® (setmelanotide) of $33.3 million -- -- Adult patients with acquired hypothalamic obesity (N=8) achieved mean BMI reduction of 12.8% on setmelanotide therapy at three months in French early-access program -- -- Top-line data readout for Phase 3 trial evaluating setmelanotide in 120 patients with acquired hypothalamic obesity on track for 1H 2025 -- -- sNDA to expand IMCIVREE label to include patients as young as 2 years old in approved indicat ...

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