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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

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Rhythm Pharmaceuticals (RYTM) presented new real-world data showing positive outcomes for setmelanotide in treating hypothalamic obesity. The data, from French early-access programs, included 35 patients treated for up to 9 months. Key findings for acquired hypothalamic obesity patients showed:
  • Adult patients (n=20) achieved -11.9% mean BMI change at 3 months, improving to -23.0% at 9 months
  • Pediatric patients showed clinically meaningful BMI z-score reductions
  • The drug was generally well-tolerated
The company previously reported successful Phase 3 trial results with -19.8% placebo-adjusted BMI reduction in 120 patients. Rhythm plans to submit regulatory applications in Q3 2025 for acquired hypothalamic obesity treatment approval. Additionally, enrollment for the Phase 3 congenital hypothalamic obesity substudy is expected to complete in H2 2025.
Rhythm Pharmaceuticals (RYTM) ha presentato nuovi dati real-world che mostrano risultati positivi per setmelanotide nel trattamento dell'obesit脿 ipotalamica. I dati, provenienti da programmi di accesso precoce in Francia, includono 35 pazienti trattati fino a 9 mesi. I risultati principali per i pazienti con obesit脿 ipotalamica acquisita sono stati:
  • I pazienti adulti (n=20) hanno registrato una riduzione media del BMI dell'11,9% a 3 mesi, migliorando al 23,0% a 9 mesi
  • I pazienti pediatrici hanno mostrato riduzioni clinicamente significative del punteggio z del BMI
  • Il farmaco 猫 stato generalmente ben tollerato
L'azienda aveva gi脿 riportato risultati positivi nella fase 3 con una riduzione del BMI aggiustata per placebo del 19,8% in 120 pazienti. Rhythm prevede di presentare le domande regolatorie nel terzo trimestre del 2025 per l'approvazione del trattamento dell'obesit脿 ipotalamica acquisita. Inoltre, si prevede il completamento del reclutamento per lo studio sussidiario di fase 3 sull'obesit脿 ipotalamica congenita nella seconda met脿 del 2025.
Rhythm Pharmaceuticals (RYTM) present贸 nuevos datos del mundo real que muestran resultados positivos para setmelanotide en el tratamiento de la obesidad hipotal谩mica. Los datos, provenientes de programas de acceso temprano en Francia, incluyeron a 35 pacientes tratados durante hasta 9 meses. Los hallazgos clave para pacientes con obesidad hipotal谩mica adquirida fueron:
  • Pacientes adultos (n=20) lograron una reducci贸n media del IMC del 11,9% a los 3 meses, mejorando al 23,0% a los 9 meses
  • Los pacientes pedi谩tricos mostraron reducciones cl铆nicamente significativas en la puntuaci贸n z del IMC
  • El medicamento fue generalmente bien tolerado
La compa帽铆a hab铆a informado previamente resultados exitosos en un ensayo de fase 3 con una reducci贸n del IMC ajustada por placebo del 19,8% en 120 pacientes. Rhythm planea presentar solicitudes regulatorias en el tercer trimestre de 2025 para la aprobaci贸n del tratamiento de la obesidad hipotal谩mica adquirida. Adem谩s, se espera que la inscripci贸n para el subestudio de fase 3 sobre obesidad hipotal谩mica cong茅nita se complete en la segunda mitad de 2025.
Rhythm Pharmaceuticals(RYTM)電� 鞁滌儊頃橂秬 牍勲 旃橂鞐� 鞛堨柎 靹疙姼氅滊澕云疙俭鞚措摐鞚� 旮嶌爼鞝侅澑 瓴瓣臣毳� 氤挫棳欤茧姅 靸堧鞖� 鞁れ牅 雿办澊韯�(real-world data)毳� 氚滍憸頄堨姷雼堧嫟. 頂勲瀾鞀� 臁瓣赴 鞝戧芳 頂勲攴鸽灗鞐愳劀 靾橃霅� 鞚� 雿办澊韯办棎電� 斓滊寑 9臧滌洈 霃欖晥 旃橂氚涭潃 35氇呾潣 頇橃瀽臧 韽暔霅橃棃鞀惦媹雼�. 須嶋摑靹� 鞁滌儊頃橂秬 牍勲 頇橃瀽鞐� 雽頃� 欤检殧 瓴瓣臣電� 雼れ潓瓿� 臧欖姷雼堧嫟:
  • 靹膘澑 頇橃瀽(20氇�)電� 3臧滌洈鞐� 韽夑窢 BMI臧 -11.9% 臧愳唽頄堨溂氅�, 9臧滌洈鞐愲姅 -23.0%旯岇 臧滌劆霅�
  • 靻岇晞 頇橃瀽霌れ潃 鞛勳儊鞝侅溂搿� 鞚橂 鞛堧姅 BMI z-鞝愳垬 臧愳唽毳� 氤挫瀯
  • 鞎诫鞚 鞝勲皹鞝侅溂搿� 鞛� 瓴帉臁岇潓
須岇偓電� 鞚挫爠鞐� 120氇呾潣 頇橃瀽毳� 雽靸侅溂搿� 頃� 3靸� 鞁滍棙鞐愳劀 鞙勳暯 雽牍� BMI臧 -19.8% 臧愳唽頃� 靹标车鞝侅澑 瓴瓣臣毳� 氤搓碃頃� 氚� 鞛堨姷雼堧嫟. Rhythm鞚 2025雲� 3攵勱赴鞐� 須嶋摑靹� 鞁滌儊頃橂秬 牍勲 旃橂 鞀轨澑 鞁犾箔鞚� 鞝滌稖頃� 瓿勴殟鞚措┌, 靹犾矞靹� 鞁滌儊頃橂秬 牍勲鞐� 雽頃� 3靸� 頃橃渼 鞐瓣惮鞚� 霌彪鞚 2025雲� 頃橂皹旮办棎 鞕勲霅� 鞓堨爼鞛呺媹雼�.
Rhythm Pharmaceuticals (RYTM) a pr茅sent茅 de nouvelles donn茅es en conditions r茅elles montrant des r茅sultats positifs pour le setmelanotide dans le traitement de l'ob茅sit茅 hypothalamique. Les donn茅es, issues de programmes d'acc猫s pr茅coce en France, comprenaient 35 patients trait茅s jusqu'脿 9 mois. Les r茅sultats cl茅s pour les patients atteints d'ob茅sit茅 hypothalamique acquise sont les suivants :
  • Les patients adultes (n=20) ont obtenu une baisse moyenne de l'IMC de -11,9 % 脿 3 mois, s'am茅liorant 脿 -23,0 % 脿 9 mois
  • Les patients p茅diatriques ont montr茅 des r茅ductions cliniquement significatives du score z de l'IMC
  • Le m茅dicament a 茅t茅 g茅n茅ralement bien tol茅r茅
La soci茅t茅 avait pr茅c茅demment rapport茅 des r茅sultats positifs lors d'un essai de phase 3 avec une r茅duction de l'IMC ajust茅e sur placebo de -19,8 % chez 120 patients. Rhythm pr茅voit de soumettre des demandes r茅glementaires au troisi猫me trimestre 2025 pour l'approbation du traitement de l'ob茅sit茅 hypothalamique acquise. De plus, le recrutement pour la sous-茅tude de phase 3 sur l'ob茅sit茅 hypothalamique cong茅nitale devrait 锚tre achev茅 au second semestre 2025.
Rhythm Pharmaceuticals (RYTM) pr盲sentierte neue AG真人官方-World-Daten, die positive Ergebnisse f眉r Setmelanotid bei der Behandlung von hypothalamischer Adipositas zeigen. Die Daten stammen aus franz枚sischen Fr眉hzugangsprogrammen und umfassen 35 Patienten, die bis zu 9 Monate behandelt wurden. Wichtige Erkenntnisse bei Patienten mit erworbener hypothalamischer Adipositas waren:
  • Erwachsene Patienten (n=20) erreichten eine durchschnittliche BMI-Reduktion von -11,9 % nach 3 Monaten, die sich auf -23,0 % nach 9 Monaten verbesserte
  • P盲diatrische Patienten zeigten klinisch bedeutsame Reduktionen des BMI-Z-Scores
  • Das Medikament wurde im Allgemeinen gut vertragen
Das Unternehmen hatte zuvor erfolgreiche Phase-3-Studienergebnisse mit einer placebo-korrigierten BMI-Reduktion von -19,8 % bei 120 Patienten berichtet. Rhythm plant, im dritten Quartal 2025 Zulassungsantr盲ge f眉r die Behandlung der erworbenen hypothalamischen Adipositas einzureichen. Zudem wird erwartet, dass die Rekrutierung f眉r die Phase-3-Substudie zur kongenitalen hypothalamischen Adipositas in der zweiten H盲lfte des Jahres 2025 abgeschlossen wird.
Positive
  • Strong efficacy data with -23.0% BMI reduction in adult patients at 9 months
  • Consistent positive results across both adult and pediatric populations
  • Successful Phase 3 trial with -19.8% placebo-adjusted BMI reduction
  • Well-established safety profile with no new safety concerns
  • Regulatory submissions on track for Q3 2025
Negative
  • None.

Insights

Positive real-world data reinforces setmelanotide's efficacy in hypothalamic obesity, strengthening RYTM's regulatory submission strategy.

The new data from France's early-access program significantly bolsters Rhythm's position as they prepare for regulatory submissions. The results show consistent and meaningful weight reductions across both acquired and congenital hypothalamic obesity patients treated with setmelanotide, with particularly impressive -23% BMI change at 9 months in adults with acquired hypothalamic obesity.

What's particularly compelling is the consistency between this real-world data and Rhythm's controlled clinical trials. The French data showed pediatric patients achieved clinically meaningful BMI z-score reductions (鈮�0.2-point is considered clinically significant) at -0.3 at 3 months and -0.4 at 6 months. The drug also demonstrated durability of effect, with benefits deepening over time rather than plateauing.

The safety profile remains consistent with no new signals, which is critical for regulatory review. The most common adverse events were injection site reactions and skin hyperpigmentation, both manageable and expected with melanocortin receptor agonists.

Rhythm's regulatory timeline remains on track, with submissions planned for Q3 2025 based on their pivotal Phase 3 trial that showed an impressive -19.8% placebo-adjusted BMI reduction. This real-world data provides complementary evidence supporting setmelanotide's efficacy in a patient population with significant unmet needs.

For a rare disease-focused company like Rhythm, expanding indications for their lead asset is a crucial value driver. These data presentations at major endocrinology conferences elevate setmelanotide's profile among prescribers while building the case for regulatory approval in new indications beyond the drug's currently approved uses.

-- 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.听(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.

鈥淭hese 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,鈥� said听David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. 鈥淭here 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 鈥� which are consistent with data from our Phase 2 and 3 clinical trials 鈥� are supportive of the potential efficacy MC4R agonists can achieve for these patients.鈥�

AG真人官方-world setmelanotide weight outcomes in French patients with acquired or congenital hypothalamic obesity听
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; 听听
    • 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鈥檚 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 鈮�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鈥檚 well-established and well-understood safety profile. For further information about setmelanotide鈥檚 safety profile, please see the information below under 鈥淲ARNINGS AND PRECAUTIONS鈥�.

Additional Congress Presentations
Additional 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 2025
Rhythm and its collaborators also presented one encore oral presentation, three encore poster presentations and hosted one satellite symposium at ECO:

  • 鈥淒AYBREAK Trial: Setmelanotide vs placebo in听patients with Melanocortin-4 Receptor听Pathway Variants鈥�
  • 鈥淔requency of Bardet-Biedl syndrome variants听in a population with early-onset obesity鈥濃�
  • 鈥淲eight loss at 18 months of setmelanotide in听2 to <6-year-old patients with rare MC4R听pathway diseases鈥嬧�
  • 鈥淏ody composition improvements with 12听months of setmelanotide in acquired听hypothalamic obesity鈥嬧�

ESPE-ESE 2025
In 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.

  • 鈥淪etmelanotide treatment in individuals with obesity and PHIP variants: Results from the DAYBREAK trial鈥�
  • 鈥淧atient and caregiver experiences with听setmelanotide听treatment in Bardet-Biedl听syndrome 鈥� real-world evidence and a patient听support program鈥濃�
  • 鈥淎ge of onset of hyperphagia and/or obesity as key听predictors of a positive genetic test for POMC,听PCSK1 or LEPR deficiency or BBS鈥嬧�
  • 鈥淲eight loss at 18 months of听setmelanotide听in 2 to <6-year-old patients with rare MC4R pathway听diseases鈥嬧�

PES 2025
Rhythm 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:

  • 鈥淚mpact of setmelanotide on metabolic syndrome risk score in pediatric patients with acquired hypothalamic obesity鈥�
  • 鈥淐linical 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鈥�

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听European Medicines Agency听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 Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm鈥檚 lead asset, IMCIVREE庐 (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鈥檚 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鈥檚 headquarters is in Boston, MA.

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).

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 鈥済asping syndrome鈥� can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs.

ADVERSE REACTIONS
Most common adverse reactions (incidence 鈮�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 www.fda.gov/medwatch. See section 4.8 of the 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鈥檚 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 鈥渆xpect鈥�, 鈥渁nticipate鈥�, 鈥渂elieve鈥�, 鈥渕ay鈥�, 鈥渨ill鈥� 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 鈥淩isk Factors鈥� in Rhythm鈥檚 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 Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280

Media Contact:
Sheryl Seapy
AG真人官方 Chemistry
(949) 903-4750


FAQ

What were the key results from RYTM's setmelanotide treatment in hypothalamic obesity patients?

Adult patients achieved -11.9% mean BMI change at 3 months, improving to -23.0% at 9 months. Pediatric patients showed clinically meaningful BMI z-score reductions of 0.3-0.4 from baseline.

When will Rhythm Pharmaceuticals submit regulatory applications for setmelanotide in acquired hypothalamic obesity?

Rhythm plans to submit a supplemental New Drug Application to the FDA and a Type II variation request to the EMA in the third quarter of 2025.

What were the safety results for setmelanotide in the RYTM study?

Setmelanotide was generally well-tolerated with most frequent adverse events being injection site reactions and skin hyperpigmentation, consistent with its established safety profile.

How many patients were included in RYTM's French early-access program for setmelanotide?

The program included 35 total patients: 30 with acquired hypothalamic obesity and 5 with congenital hypothalamic obesity.

What was the Phase 3 trial result for RYTM's setmelanotide in acquired hypothalamic obesity?

The Phase 3 trial met its primary endpoint, demonstrating a -19.8% placebo-adjusted BMI reduction in 120 patients.
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