Latest news with #DavidMeeker
Yahoo
14-05-2025
- Health
- Yahoo
Rhythm Pharmaceuticals Announces New Data Presentations on Patients with Acquired or Congenital Hypothalamic Obesity (N=35) Treated with Setmelanotide for up to Nine Months in French Early-access Program
-- New data showed setmelanotide achieved consistent, meaningful weight reduction -- -- Multiple presentations delivered at the first-ever Joint Congress between the European Society for Paediatric Endocrinology and the European Society of Endocrinology (ESPE-ESE); 32nd annual European Congress on Obesity (ECO); and the 2025 annual meeting of the Pediatric Endocrine Society (PES 2025) -- BOSTON, May 14, 2025 (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 the presentation of new, real-world data at two European congresses that show consistent improvements in body mass index (BMI), BMI-z, and hunger scores in a total of 35 patients with acquired or congenital hypothalamic obesity who were treated with setmelanotide for up to nine months. Physicians from Sorbonne University in Paris delivered two oral presentations with data from patients in the early-access programs in France. At the first-ever Joint Congress between the European Society for Paediatric Endocrinology and the European Society of Endocrinology (ESPE-ESE), the presentation detailed real-world data from 30 patients with acquired hypothalamic obesity on setmelanotide therapy for up to nine months. For the second presentation at the 32nd annual European Congress on Obesity (ECO), they also shared data from five patients with congenital hypothalamic obesity, including four patients who reached six months on setmelanotide therapy. 'These real-world data show that patients with acquired or congenital hypothalamic obesity on setmelanotide treatment achieved clinically meaningful weight reductions and reductions in hunger beginning at three months that were sustained and deepened in those patients who reached six and nine months on therapy,' said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. 'There is a significant unmet need for a treatment that can address the weight gain and overwhelming hunger that patients with either acquired or congenital hypothalamic obesity live with, and we believe these data – which are consistent with data from our Phase 2 and 3 clinical trials – are supportive of the potential efficacy MC4R agonists can achieve for these patients.' Real-world setmelanotide weight outcomes in French patients with acquired or congenital hypothalamic obesity The presentations delivered at ESPE-ESE and ECO 2025 detailed clinical outcomes from 30 patients (10 patients younger than 18; 20 patients 18 or older) with acquired hypothalamic obesity who received setmelanotide treatment as part of the pre-approval, early-access program at 14 different treatment centers in France. Highlights include: Of the 20 adult patients with acquired hypothalamic obesity treated with setmelanotide therapy: Across all patients, there was an overall -11.9% mean change in body mass index (BMI) from baseline at month 3; Ten (n=10) who reached month 6 on therapy achieved -19.2% mean change in BMI; and Eight (n=8) who reached month 9 on therapy achieved -23.0% mean change in BMI; and Adult patients reported meaningful decreases in hunger scores after 3 and 6 months of treatment with setmelanotide. For pediatric patients with acquired hypothalamic obesity treated with setmelanotide, BMI z-score (a measure that represents standard deviations of a child's BMI that corrects for age and sex) decreased from baseline at all timepoints analysed. A clinically meaningful reduction in BMI z-score is defined as a ≥0.2-point reduction. Data from these pediatric patients include: Ten (n=10) pediatric patients who reached month 3 on therapy achieved a mean BMI z-score decrease of 0.3 from baseline; Seven (n=7) pediatric patients who reached month 6 on therapy achieved a mean BMI z-score decrease of 0.4 decrease from baseline; and Two (n=2) pediatric patients who reached month 9 on therapy achieved a mean BMI z-score decrease of 0.4 from baseline. The presentation at ECO 2025 also featured data from five patients (4 younger than 18; 1 older than 18) with congenital hypothalamic obesity. Highlights include: Four (n=4) pediatric patients who reached month 3 on setmelanotide therapy achieved a mean BMI z-score decrease of -0.2 from baseline; and Three (n=3) pediatric patients who reached month 6 on setmelanotide therapy achieved a mean BMI z-score decrease of -0.4 from baseline; and One adult patient with congenital hypothalamic obesity achieved a -14.8% BMI reduction baseline at month 6 of setmelanotide therapy. Setmelanotide was generally well tolerated with the most frequent AEs being injection site reactions and skin hyperpigmentation. No new safety signals related to use of setmelanotide were observed, consistent with setmelanotide's well-established and well-understood safety profile. For further information about setmelanotide's safety profile, please see the information below under 'WARNINGS AND PRECAUTIONS'. Additional Congress PresentationsAdditional Rhythm presentations were also shared at ECO and ESPE/ESE. Two presentations will also be shared at the Pediatric Endocrinology Society (PES) annual meeting from May 15-18 in National Harbor, Maryland. ECO 2025Rhythm and its collaborators also presented one encore oral presentation, three encore poster presentations and hosted one satellite symposium at ECO: 'DAYBREAK Trial: Setmelanotide vs placebo in patients with Melanocortin-4 Receptor Pathway Variants' 'Frequency of Bardet-Biedl syndrome variants in a population with early-onset obesity' 'Weight loss at 18 months of setmelanotide in 2 to <6-year-old patients with rare MC4R pathway diseases' 'Body composition improvements with 12 months of setmelanotide in acquired hypothalamic obesity' ESPE-ESE 2025In addition to sharing the acquired hypothalamic obesity data from the French early-access program, Rhythm and its collaborators also presented two poster presentations and two oral presentations and hosted a satellite symposium at the conference. 'Setmelanotide treatment in individuals with obesity and PHIP variants: Results from the DAYBREAK trial' 'Patient and caregiver experiences with setmelanotide treatment in Bardet-Biedl syndrome – real-world evidence and a patient support program' 'Age of onset of hyperphagia and/or obesity as key predictors of a positive genetic test for POMC, PCSK1 or LEPR deficiency or BBS' 'Weight loss at 18 months of setmelanotide in 2 to <6-year-old patients with rare MC4R pathway diseases' PES 2025Rhythm and its collaborators will present two posters at the 2025 Pediatric Endocrine Society (PES) Annual Meeting held May 15-18, 2025 in National Harbor, Maryland: 'Impact of setmelanotide on metabolic syndrome risk score in pediatric patients with acquired hypothalamic obesity' 'Clinical characteristics of 2 to 5-year-old patients with hyperphagia and obesity secondary to melanocortin-4 receptor pathway diseases and 1-year response to setmelanotide' All of the Rhythm-related presentations from ECO, ESPE-ECE, and PES are available here: These presentations are intended for U.S. audiences. Rhythm previously announced that its pivotal Phase 3 trial evaluating setmelanotide for the treatment of acquired hypothalamic obesity met its primary endpoint with a -19.8% placebo-adjusted BMI reduction in 120 patients. The Company remains on track to submit a supplemental New Drug Application to the FDA and a Type II variation request to the European Medicines Agency for setmelanotide for the treatment of patients with acquired HO in the third quarter of 2025. In addition, Rhythm anticipates it will complete enrollment in the Phase 3 trial substudy investigating the use of setmelanotide in congenital hypothalamic obesity in the second half of 2025. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONSMost common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the safety, efficacy, potential benefits of, and regulatory and clinical progress, potential regulatory submissions, approvals and timing thereof of setmelanotide and other product candidates; the clinical design or progress of any of our products or product candidates at any dosage or in any indication, the planned substudy to evaluate patients with congenital hypothalamic obesity and the timing of enrollment for the substudy to our Phase 3 trial evaluating setmelanotide in patients with acquired hypothalamic obesity; the potential benefits of any of the Company's products or product candidates for any specific disease indication or at any dosage, including the potential benefits of setmelanotide for patients with acquired hypothalamic obesity or congenital hypothalamic obesity; our participation in upcoming events and presentations, and the date, time and content thereof and the timing of any of the foregoing. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks, uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption 'Risk Factors' in Rhythm's Quarterly Report on Form 10-Q for the three months ended March 31, 2025 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 release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@ Sign in to access your portfolio
Yahoo
06-05-2025
- Health
- Yahoo
Rhythm Pharmaceuticals Announces Participation in Upcoming Investor Conferences
Setmelanotide Indication In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). About Rhythm Pharmaceuticals Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. The fireside chats will be webcasted and available under 'Events & Presentations' in the Investor Relations section of the Company's website at and webcast replays will be available for 30 days following the presentations. BOSTON, May 06, 2025 (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 three upcoming investor conferences: Story Continues In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication, our participation in upcoming events and presentations, and the date, time and content thereof and the timing of any of the foregoing. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks, uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024 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 release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact: David Connolly Head of Investor Relations and Corporate Communications Rhythm Pharmaceuticals, Inc. 857-264-4280 dconnolly@ Media Contact: Sheryl Seapy Real Chemistry (949) 903-4750 sseapy@
Yahoo
26-02-2025
- Business
- Yahoo
Rhythm Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Business Update
-- Fourth quarter 2024 net revenue from global sales of IMCIVREE® (setmelanotide) of $41.8 million -- -- On track to report topline data from global Phase 3 trial evaluating setmelanotide in acquired hypothalamic obesity in the second quarter of 2025 -- -- Completed enrollment in the Phase 2 trial of oral MC4R agonist bivamelagon in acquired hypothalamic obesity -- -- FDA approved expanded label for IMCIVREE to include children as young as 2 years old -- -- Raised approximately $75 million in gross proceeds under ATM equity offering program; Cash runway extended into 2027 -- -- Management to host conference call today at 8:00 a.m. ET -- BOSTON, Feb. 26, 2025 (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 reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2024. 'Rhythm delivered solid IMCIVREE global sales growth in the fourth quarter and is poised to drive continued growth in 2025,' said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. 'Our Phase 3 trial in acquired hypothalamic obesity (HO) remains on track as we expect to disclose topline data in the second quarter of 2025. We are well capitalized as we strengthened our balance sheet with approximately $75 million in gross proceeds raised under our ATM program, which extends our cash runway into 2027, enabling us to execute on multiple clinical milestones expected in the year ahead.' Fourth Quarter and Recent Business Highlights Revenue from global sales of IMCIVREE was $41.8 million for the fourth quarter of 2024, an increase of 26% percent on a sequential basis from the third quarter of 2024, primarily driven by sales of IMCIVREE for the treatment of Bardet-Biedl syndrome (BBS). In the fourth quarter of 2024, revenue of $31.7 million, or 76% of product revenue, was generated in the United States, an increase of 36.2% on a sequential basis; revenue of $10.1 million, or 24% of product revenue, was generated outside the United States; Today, the Company announced that it raised approximately $75 million in gross proceeds from the sale of approximately 1.3 million shares of common stock at a weighted average price of $56.30 under its 'at the market' equity offering program (the 'ATM Program') during the fourth quarter of 2024 and in January 2025; Today, Rhythm announced that it completed enrollment in the Phase 2 trial evaluating oral MC4R agonist bivamelagon (LB54640) in acquired hypothalamic obesity; Today, Rhythm announced a strategic partnership agreement with Trispera Pharma Solutions in Turkey; On January 10, 2025, Rhythm announced that it: Completed enrollment in its supplemental, 12-patient Japanese cohort of the global Phase 3 trial evaluating setmelanotide in acquired HO. Data from this supplemental cohort will serve as the basis for a regulatory submission in Japan; Completed enrollment in the Phase 3 EMANATE trial of setmelanotide, which is comprised of four substudies: SH2B1 (n=121); POMC and/or PCSK1 (n=79); SRC1 (n=73); and LEPR (n=23). The four-substudy design of this trial allows for independent data readouts and potential registration for each genetic cohort; On December 20, 2024, the Company announced that the U.S. Food and Drug Administration (FDA) approved an expanded indication for IMCIVREE to include children as young as 2 years old with syndromic or monogenic obesity due to BBS or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency; On December 3, 2024, Rhythm announced that the United Kingdom's Medicines & Healthcare products Regulatory Agency (MHRA) has expanded the marketing authorization for IMCIVREE to include the treatment of obesity and control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adult and pediatric patients as young as 2 years old and older; and During the annual meeting of the European Society for Paediatric Endocrinology in November 2024, Rhythm presented new, real-world data that showed four pediatric patients, two with acquired HO and two with congenital HO, achieved >5% weight reduction at three months on setmelanotide. The Company announced plans for a new, 34-week substudy in the ongoing setmelanotide Phase 3 trial in 39 patients with congenital HO aged 4 years and older. Anticipated Upcoming Milestones Rhythm expects to achieve the following near-term milestones: Begin enrolling the first patients with congenital HO in a 34-week substudy of setmelanotide being conducted under the protocol for its global Phase 3 trial in the first quarter of 2025; Begin enrolling the first patients with Prader-Willi syndrome (PWS) in a 26-week, open-label Phase 2 trial evaluating setmelanotide in the first quarter of 2025; Begin enrolling patients with acquired HO in Part C of the Phase 1 trial evaluating the weekly, MC4R agonist RM-718 in the first quarter of 2025; Announce top-line data in the Phase 3 trial evaluating setmelanotide in acquired HO in the second quarter of 2025; Announce topline data from the Phase 2 trial of bivamelagon in acquired HO in the second half of 2025; and Announce top-line data in the Phase 3 EMANATE trial evaluating setmelanotide in genetically caused MC4R pathway diseases in the first half of 2026. Fourth Quarter and Full Year 2024 Financial Results: Cash Position: As of December 31, 2024, cash, cash equivalents and short-term investments were approximately $320.6 million, as compared to $275.8 million as of December 31, 2023. The December 31, 2024 cash balance does not include gross proceeds of approximately $34.7 million from the sale of stock under the Company's ATM program executed during January 2025. Revenue: Net product revenues relating to global sales of IMCIVREE were $41.8 million for the fourth quarter of 2024 and $130.1 million for the full year of 2024, as compared to $24.2 million for the fourth quarter of 2023 and $77.4 million for the full year of 2023. R&D Expenses: R&D expenses were $41.2 million in the fourth quarter of 2024 and $238.0 million for the full year of 2024, as compared to $29.9 million in the fourth quarter of 2023 and $135.0 million for the full year of 2023. The year-over-year increase was primarily due to increased costs associated with the acquisition of bivamelagon from LG Chem and its development with certain additional product manufacturing activity, ongoing clinical trials and increased headcount. This increase was partially offset by decreased costs in other trials. SG&A Expenses: SG&A expenses were $38.1 million for the fourth quarter of 2024 and $144.3 million for the full year of 2024, as compared to $32.4 million for the fourth quarter of 2023 and $117.5 million for the full year of 2023. The year-over-year increase was primarily due to increased headcount, marketing and promotions costs and expenses for professional services and partially offset by inventory-related costs that were capitalized as labor and overhead. Other income (expense), net: Other income (expense), net was ($2.1) million for the fourth quarter of 2024 and $5.2 million for the full year of 2024. Net Loss: Net loss attributable to common stockholders was ($44.6) million for the fourth quarter of 2024 and ($264.6) million for the full year of 2024, or a net loss per basic and diluted share of ($0.72) and ($4.34), respectively, as compared to a net loss attributable to common stockholders of ($41.6) million for the fourth quarter of 2023 and ($184.7) million for the full year of 2023, or a net loss per basic and diluted share of ($0.70) and ($3.20), respectively. Financial Guidance: For the year ended December 31, 2024, the Company had GAAP total operating expenses of $382.3 million, inclusive of $92.4 million associated with the acquisition of the rights to bivamelagon from LG Chem. The Company today reported non-GAAP Operating Expenses for the year ended December 31, 2024 of $250.2 million, which is derived from GAAP total operating expenses of $382.3 million less $39.7 million in stock-based compensation and $92.4 million associated with the acquisition of the rights to bivamelagon from LG Chem. For the year ending December 31, 2025, Rhythm anticipates approximately $285 million to $315 million in Non-GAAP Operating Expenses. Non-GAAP Operating Expenses are derived from: GAAP total operating expenses, inclusive of: SG&A expenses of approximately $135 million to $145 million; R&D expenses of approximately $150 million to $170 million; and Excluding stock-based compensation. Non-GAAP Operating Expenses is defined as GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing (see below under "Non-GAAP Financial Measures" for more details). Based on its current operating plans, Rhythm expects that its existing cash, cash equivalents and short-term investments as of December 31, 2024, and including proceeds from the sale of stock through its ATM program during January 2025, will be sufficient to fund its operating expenses and capital expenditure requirements into 2027. Conference Call InformationRhythm Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to review its fourth quarter and full year 2024 financial results and recent business activities. Participants may register for the conference call here. It is recommended that participants join the call ten minutes prior to the scheduled start. A webcast of the call will also be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at The archived webcast will be available on Rhythm Pharmaceuticals' website approximately two hours after the conference call and will be available for 30 days following the call. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONSMost common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication, including, setmelanotide, bivamelagon, and RM-718; the potential use of setmelanotide in patients with acquired hypothalamic obesity; our expectations surrounding potential regulatory submissions, progress, or approvals and timing thereof for any of our product candidates; the estimated market size and addressable population for our drug products; the announcement of data from our clinical trials, including our Phase 3 trial evaluating setmelanotide for patients with acquired hypothalamic obesity, the substudy evaluating setmelanotide for patients with congenital hypothalamic obesity, the Phase 3 EMANATE trial evaluating setmelanotide in genetically caused MC4R pathway diseases, and the Phase 2 trial evaluating the oral MC4R agonist bivamelagon in acquired hypothalamic obesity; Part C of the Phase 1 trial evaluating RM-718; the open-label Phase 2 trial evaluating setmelanotide in patients with Prader-Willi syndrome; the ongoing enrollment in our clinical trials; existing or future collaboration agreements; the Company's business strategy and plans; our anticipated financial performance and financial position for any period of time, including estimated Non-GAAP Operating Expenses for the year ending December 31, 2025; and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations; and the timing of any of the foregoing. Statements using words 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 Annual Report on Form 10-K for the year ended December 31, 2024 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 press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise. Non-GAAP Financial MeasuresThis press release 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 present this non-GAAP financial measure because we consider it to be an important supplemental measure of our performance and believe it is frequently used by securities analysts, investors, and other interested parties in the evaluation of companies in our industry. Management believes that investors' understanding of our performance is enhanced by including this non-GAAP financial measure as a reasonable basis for comparing our ongoing results of operations. Management uses this non-GAAP financial measure for planning purposes, including the preparation of our internal annual operating budget and financial projections; to evaluate the performance and effectiveness of our operational strategies; and to evaluate our capacity to expand our business. This non-GAAP financial measure has limitations as an analytical tool, and should not be considered in isolation, or as an alternative to, or a substitute for operating expenses or other financial statement data presented in accordance with GAAP in our consolidated financial statements. Rhythm has not provided a quantitative reconciliation of forecasted Non-GAAP Operating Expenses to forecasted GAAP operating expenses because the Company is 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 Rhythm's control. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@ Rhythm Pharmaceuticals, Inc. Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (Unaudited) Three Months Ended December 31, Year Ended December 31, 2024 2023 2024 2023 evenues: Product revenue, net $ 41,830 $ 24,234 $ 130,126 $ 77,428 Total revenues 41,830 24,234 130,126 77,428 Costs and expenses: Cost of sales 3,787 3,233 13,368 9,302 Research and development 41,168 29,892 237,957 134,951 Selling, general, and administrative 38,130 32,374 144,304 117,532 Total costs and expenses 83,085 65,499 395,629 261,785 Loss from operations (41,255 ) (41,265 ) (265,503 ) (184,357 ) Other income (expense): Other income (expense), net (195 ) 39 2,239 190 Gain on settlement of forward contract — — 8,900 — Interest expense (5,447 ) (4,018 ) (20,603 ) (13,892 ) Interest income 3,514 3,819 14,711 13,945 Total other income (expense), net (2,128 ) (160 ) 5,247 243 Loss before income taxes (43,383 ) (41,425 ) (260,256 ) (184,114 ) Provision for income taxes (89 ) 196 346 564 Net loss $ (43,294 ) $ (41,621 ) $ (260,602 ) $ (184,678 ) Accrued dividends on convertible preferred stock (1,340 ) — (3,970 ) — Net loss attributable to common stockholders $ (44,634 ) $ (41,621 ) $ (264,572 ) $ (184,678 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.72 ) $ (0.70 ) $ (4.34 ) $ (3.20 ) Weighted-average common shares outstanding, basic and diluted 61,596,442 59,211,199 60,995,204 57,673,128 Other comprehensive loss: Net loss attributable to common stockholders $ (44,634 ) $ (41,621 ) $ (264,572 ) $ (184,678 ) Foreign currency translation adjustment 977 76 2 (140 ) Unrealized (loss) gain, net on marketable securities, net of tax (412 ) (175 ) (175 ) 366 Comprehensive loss $ (44,069 ) $ (41,720 ) $ (264,745 ) $ (184,452 ) Condensed Consolidated Balance Sheets(in thousands, except share and per share data) December 31,2024 December 31,2023 Assets Current assets: Cash and cash equivalents $ 89,137 $ 60,081 Short-term investments 231,428 215,765 Accounts receivable, net 18,512 14,867 Inventory 18,741 8,624 Prepaid expenses and other current assets 16,382 8,931 Total current assets 374,200 308,268 Property and equipment, net 632 1,341 Right-of-use asset 3,477 781 Intangible assets, net 6,174 7,028 Restricted cash 464 328 Other long-term assets 7,326 14,999 Total assets $ 392,273 $ 332,745 Liabilities, Convertible Preferred Stock and Stockholders' Equity Current liabilities: Accounts payable $ 12,328 $ 4,885 Accrued expenses and other current liabilities 62,658 48,262 Other current liability - LG Chem 37,704 — Deferred revenue 1,286 1,286 Deferred royalty obligation, current 1,541 — Lease liability — 770 Total current liabilities 115,517 55,203 Long-term liabilities: Deferred royalty obligation 108,269 106,143 Lease liability, non-current 3,938 490 Derivative liability — 1,150 Total liabilities 227,724 162,986 Commitments and contingencies Series A convertible preferred stock, $0.001 par value: 150,000 sharesauthorized; 150,000 and 0 shares issued and outstanding at December 31, 2024and December 31, 2023, respectively. Liquidation preference of $150,000 as ofDecember 31, 2024. 142,820 — Stockholders' equity: Common stock, $0.001 par value: 120,000,000 shares authorized; 62,390,654and 59,426,559 shares issued and outstanding at December 31, 2024 andDecember 31, 2023, respectively 61 59 Additional paid-in capital 1,177,045 1,064,302 Accumulated other comprehensive (loss) income (39 ) 134 Accumulated deficit (1,155,338 ) (894,736 ) Total stockholders' equity 21,729 169,759 Total liabilities, convertible preferred stock and stockholders' equity $ 392,273 $ 332,745 Sign in to access your portfolio
Yahoo
13-02-2025
- Business
- Yahoo
Rhythm Pharmaceuticals to Report Fourth Quarter and Full Year 2024 Financial Results on Wednesday, February 26, 2025
BOSTON, Feb. 13, 2025 (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 it will host a live conference call and webcast at 8:00 a.m. ET on Wednesday, February 26, 2025 to report its fourth quarter and full year 2024 financial results and provide a corporate update. To access the live conference call, participants may register here. While not required, it is recommended that participants join the call ten minutes prior to the scheduled start. Also today, Rhythm announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at the upcoming TD Cowen 45th Annual Health Care Conference in Boston on Monday, March 3, 2025, at 1:10 p.m. ET. Live webcasts of both the financial results conference call and the fireside chat will be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at The archived webcast of the financial results conference call will be available on Rhythm's website approximately two hours after it concludes and will be available for 30 days following the call. A replay of the TD Cowen webcast will also be available on the Rhythm website for 30 days following the presentation. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the release of our financial results and our participation in upcoming events and presentations, including the timing of any of the foregoing. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks, uncertainties and other important factors, including those discussed under the caption 'Risk Factors' in Rhythm's Quarterly Report on Form 10-Q for the three months ended September 30, 2024 and 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 release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@
Yahoo
05-02-2025
- Business
- Yahoo
Rhythm Pharmaceuticals to Present at Oppenheimer Annual Healthcare Life Sciences Conference in February
BOSTON, Feb. 05, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a 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 a fireside chat at the upcoming Oppenheimer 35th Annual Healthcare Life Sciences Conference on Wednesday, February 12, 2025 at 8:40 a.m. ET. The fireside chat will be webcasted and available under 'Events & Presentations' in the Investor Relations section of the Company's website at A replay of the webcast will be available on the Rhythm website for 30 days following the presentation. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements our participation in upcoming events and presentations, including the timing thereof. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, risks relating to our net revenues and anticipated financial results for the fiscal year ended December 31, 2024, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic, political and regulatory conditions, and the other important factors discussed under the caption 'Risk Factors' in Rhythm's Quarterly Report on Form 10-Q for the three months ended September 30, 2024 and 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 release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@ Effettua l'accesso per consultare il tuo portafoglio
