At Rhythm Pharmaceuticals, we are committed to our clinical trials program designed to develop innovative therapies for people with rare neuroendocrine diseases. These trials help us evaluate safety and efficacy of these potentially life-changing treatments.
If you think you may be eligible, speak with your healthcare provider about the possibility of participating in one of our clinical trials.
IMCIVREE® (setmelanotide)
Disease population*
- POMC and LEPR deficiency obesities
- Bardet-Biedl syndrome
Setmelanotide daily
Disease Population
- Acquired hypothalamic obesity
Disease Population
- POMC insufficiency obesity
- LEPR insufficiency obesity
- SRC1 deficiency obesity
- SH2B1 deficiency obesity
Disease Population
- Variants in certain genes with strong association to the MC4R pathway
Bivamelagon (LB54640)
Disease Population
- Acquired hypothalamic obesity
- An investigational oral, MC4R-specific agonist shown not to cause hyperpigmentation being evaluated as a potential therapy for acquired hypothalamic obesity
RM-718
- A new weekly MC4R–specific agonist designed to not cause hyperpigmentation
Discovery Pipeline
We are evaluating a suite of early-stage assets in order to develop new therapies for congenital hyperinsulinism (CHI).
Understanding clinical trials
Clinical trials are scientific studies where researchers evaluate a treatment, like a new drug, in human volunteers. In most cases, new medicines must pass through clinical trials to show their safety and effectiveness for approval from the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other health regulators.
What you should know about clinical trials
- A patient must meet specific eligibility criteria to participate in a clinical trial, such as age, diagnosis, and treatment history
- Clinical trials can involve risks, and participation should be carefully considered
- There is usually no cost to receive treatment in a clinical trial
- Volunteers in a clinical trial may withdraw at any time
For more information about clinical trials, visit clinicaltrials.gov or speak with your doctor. Questions about Rhythm clinical trials can be sent to [email protected].
Expanded access to investigational drugs
Patients may be able to receive investigational therapies through the expanded access program (EAP) (sometimes called compassionate use), if:
- Patient is unable to participate in clinical trials, and
- Patient has a serious or immediately life-threatening disease or condition with no comparable or satisfactory alternative treatment options, and
- Potential benefit of treatment outweighs the risk
Following the completion of a Phase 3 clinical trial that meets key clinical endpoints, Rhythm may consider making setmelanotide available through our EAP. If a treating physician believes that setmelanotide may be the only therapeutic option for a patient along with the parameters listed above, they can request additional information about Rhythm’s EAP by emailing: [email protected]. Rhythm will acknowledge receipt of such requests within five (5) business days.
Investigator-initiated studies
Rhythm’s areas of interest for investigator-initiated studies are focused on neuroendocrine diseases, including but not limited to natural history, genotype-phenotype correlations, energy expenditure, 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 [email protected].
*In the United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or 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) or BBS.