A comprehensive clinical development program
“通过多次赌钱网址平台官网, we are exploring opportunities to extend the reach of our 精密医学 to address the needs of many more patients with early-onset, 严重的肥胖和贪食. Our goal is to deliver new options to these patients who otherwise have no approved therapies that specifically address the underlying cause of their rare MC4R pathway diseases.”
——大卫·米克尔.D.首席执行官
节奏的管道
Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to address the underlying cause of obesities driven by impaired function of the MC4R pathway.
IMCIVREE (setmelanotide) has been approved by the U.S. Food and Drug Administration for chronic weight management in adult and pediatric patients 6 years old and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency or Bardet-Biedl syndrome (BBS). In Europe, IMCIVREE has been authorized by the European Commission and the UK’s Medicines & Healthcare 产品s Regulatory Agency (MHRA) for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, 包括PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE has been generally well-tolerated. 性唤起障碍, 抑郁和自杀意念, and skin pigmentation and darkening of pre-existing nevi may occur. The most common adverse reactions were skin hyperpigmentation, injection site reactions and nausea. For more information, including important safety information, visit IMCIVREE.com.
We are advancing setmelanotide in several additional trials. Here is our investigational pipeline:
We are advancing setmelanotide in several addition trials. Here is our investigational pipeline:
研究
阶段
Heterozygous POMC/PCSK1 deficiency
杂合子LEPR缺乏症
SRC1缺乏SH2B1缺乏
第三阶段
Patients (ages 2 to younger than 6) with obesity due to biallelic POMC, PCSK1或LEPR缺陷或BBS
第三阶段
Additional genes with strong or very strong relevance to MC4R
第二阶段
探索性篮子研究
第二阶段
下丘脑肥胖
第三阶段
Setmelanotide(每周)
开关试验
第三阶段
Patients with BBS naive to setmelanotide therapy
第三阶段
* Patients with obesity due to biallelic or heterozygous POMC, PCSK1 or LEPR deficiency or a clinical diagnosis of BBS with genetic confirmation.
完整的
指示研究正在进行
计划研究
第二阶段
第三阶段
Setmelanotide(每日)
散发
POMC不足肥胖
LEPR功能不全肥胖
SRC1缺乏性肥胖
SH2B1缺乏性肥胖
儿科试验
黎明
Additional genes with strong or very strong relevance to the MC4R pathway
探篮试验
几个群
下丘脑肥胖
Setmelanotide(每周)
开关试验
Patients previously in open-label extension study of daily setmelanotide*
De Novo试验
Patients with BBS naive to setmelanotide therapy
* Patients with obesity due to biallelic or heterozygous POMC, PCSK1 or LEPR deficiency or a clinical diagnosis of BBS.
Opportunity to extend the reach of our 精密医学
The Phase 3 散发 trial and the Phase 2 黎明 trial are evaluating setmelanotide in people with obesity and a variant in certain genes with strong or very strong relevance to the MC4R pathway.
了解更多赌钱网址平台官网
Rare MC4R pathway diseases that present with severe obesity early in life
The 严重的肥胖和贪食 that characterize rare MC4R pathway diseases often present early in life. That is why 节奏 initiated a Phase 3 trial in pediatric patients.
The trial includes patients ages 2 to younger than 6 years old who have obesity due to biallelic proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency or a clinical diagnosis of Bardet-Biedl syndrome (BBS).
欲了解更多信息，请访问 赌钱网址平台官网.政府
Potential to improve convenience for patients and caregivers
节奏 is evaluating a once-weekly formulation of setmelanotide designed to provide similar efficacy and to improve compliance and adherence.
In a completed trial in healthy volunteers living with obesity, data showed that a once-weekly formulation of setmelanotide achieved safety and efficacy results comparable to the daily-dosing formulation.
节奏 is advancing two Phase 3 trials evaluating a once-weekly formulation of setmelanotide:
- Phase 3 switch trial evaluating a weekly formulation of setmelanotide in patients 6 years of age and older with a rare genetic disease of obesity who are currently taking the daily formulation of setmelanotide.
- Phase 3 de novo trial to evaluating the weekly formulation of setmelanotide in patients with BBS who have not previously taken setmelanotide. This trial is being conducted in Europe and the Middle East.
Learn more about 节奏’s clinical trials process
研究者发起的研究
节奏’s areas of interest for investigator-initiated studies are focused on MC4R pathway related diseases, including but not limited to natural history, genotype-phenotype相关性, 能量消耗, hyperphagia and real-world experience with setmelanotide. Studies designed for general obesity or non-rare diseases are not within our scope. For more information, please email (电子邮件保护).
