Rhythm Pharmaceuticals has enrolled the first patient in a pivotal Phase lll clinical trial examining setmelanotide for the treatment of patients with Bardet-Biedl Syndrome (BBS) and Alström Syndrome.

Both BBS and Alström Syndrome are rare genetic disorders and have multiple biological similarities. People suffering from these diseases experience an insatiable hunger and severe obesity early in life.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The pivotal Phase lll trial includes a multinational, open-label, single-arm study design.

It is expected to enrol 30 patients, including around 20 BBS patients and approximately six Alström Syndrome patients, aged six years and older.

“Rhythm’s setmelanotide is an investigational potent, melanocortin-4 receptor (MC4R) agonist designed to treat rare genetic disorders of obesity.”

During the trial’s 52 weeks of the treatment period, patients will receive a daily subcutaneous injection of setmelanotide. The trial will also comprise a 14-week placebo-controlled period.

The trial’s primary objective is a responder analysis after nearly 52 weeks of therapy.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Following the completion of enrolment, Rhythm plans to continue enrolling supplemental patients in order to gather additional safety and efficacy data in people living with BBS and Alström Syndrome.

The trial’s principal investigator Robert Haws said: “Many of our BBS patients struggle with insatiable hunger and severe obesity, which can be a challenging burden while also managing the disorder’s potential effects on vision, kidney function and other organs.”

Rhythm’s setmelanotide is an investigational potent, melanocortin-4 receptor (MC4R) agonist designed to treat rare genetic disorders of obesity.

Setmelanotide is developed to activate MC4R, which is a major biological pathway that independently controls energy expenditure and appetite.