PWS Clinical Trials Update - Part 2

Over the past 3 years we have seen a number of new companies investing in Prader-Willi Syndrome. This is in part because of PWS's orphan indication and the significant unmet medical need, but is also reflective of the steps we have taken as a community to position ourselves for pharma investment. 

On October 30th, 2016 FPWR held its third annual clinical trials panel and shared updates from several PWS clinical trials. The video below is part 2 of 2 recordings from this panel.

In this recording, Dr. Fred Fiedorek, Chief Medical Officer of Rhythm, a biopharmaceutical company, presents on their study results of Setmelanotide, a drug targeting weight and appetite, and currently undergoing a phase 2 study. 



In his presentation, Dr. Fred Fiedorek provides an overview of the status of trials for the drug setmelanotide. Fiedorek noted that unlike diazoxide-CCR, described in a part 1 of our clinical trials update, setmelanotide is a new drug. “We’re doing everything we need to do to evaluate it in all the safety and toxicology studies that are necessary.” In its current formulation, it is administered as a single daily injection. In describing the setmelanotide’s function, Fiedorek stated, “we’re targeting the MC4 receptors [which reside in the hypothalamus] that are in the end of the pathway [.…] That’s where appetite, energy metabolism and ultimately weight […] is controlled in a normal situation.”

Fiedorek then presented information on two trials involving setmalanotide. The first, phase two study, does not include PWS patients, but rather patients of a very rare genetic form of obesity called POMC-Deficiency. Similar to the PWS population, POMC-Deficiency patients also suffer from dysregulation of the hypothalamus, and resulting intense hunger and weight issues. Published results of phase two trials with these patients have shown positive results with regards to weight, hunger, as well as a marked improvement in insulin resistance. As data collection wraps up for a second phase two study of setmelanotide -- this time involving PWS patients -- Fiedorek hopes for comparable results. “Our hope is that […] we’re going to have similar effects on weight and hyperphagia.” Results for this trial are expected by the end of the year.

For updated information on PWS clinical trial opportunities and to sign up for a monthly PWS Clinical Trial Alert, visit the PWS Clinical Trials page.PWS Clinical Trials

Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.