Looking to the Future of PWS Treatment

We are working hard to uncover new ways to treat Prader-Willi syndrome. Our research is informed by emerging developments in the understanding of PWS at the genetic level. We are following these paths in an effort to pave the way for a brighter future for both patients and caregivers.

Our lead therapeutic program is LV-101 (intranasal carbetocin) for the treatment of hyperphagia and behavior associated with Prader-Willi syndrome.

A Phase 2 proof of concept study was conducted in 2014 testing LV-101 versus placebo in PWS.  This randomized, double-blinded, placebo-controlled study showed clinically meaningful improvements in hyperphagia and associated behavioral symptoms.  These data were presented at the 2018 Annual Meeting of the Pediatric Academic Societies and published in JCI Insight.

Based on the positive results seen in the Phase 2 study, Levo Therapeutics conducted a Phase 3 clinical trial of LV-101, referred to as CARE-PWS.  Top-line results from the 8-week placebo-controlled period were announced in August of 2020, and later presented at the 2021 Annual Meeting of the Endocrine Society (ENDO 2021).

In January 2022, we announced that, despite determining that the product was safe for chronic use in patients with PWS, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter following its review of Levo’s New Drug Application (NDA) for LV-101 (intranasal carbetocin) as a treatment for hyperphagia and behavioral distress associated with PWS.  We are working diligently to design another clinical study to evaluate the efficacy of LV-101 in patients with PWS.

LV-101 represents the first of what we hope to be many treatments for this complex syndrome. We are developing additional programs for the treatment of PWS leveraging our understanding of the underlying neurobiological pathophysiology.