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Inversago Pharma Receives Rare Pediatric Disease Designation for INV-101 for the Treatment of PWS

Inversago has received a Rare Pediatric Disease designation for INV-101 for treating PWS. A Phase I study is ongoing now to evaluate safety and efficacy.


Inversago Pharma has been granted a Rare Pediatric Disease (RPD) designation by the FDA for the Company’s lead compound, INV-101, for the treatment of Prader-Willi syndrome (PWS). A Phase I study with INV-101 is currently ongoing to evaluate its safety, tolerability and pharmacokinetics profile. Read Inversago's recent press release here. 

INV-101 is a first-in-class, small molecule CB1 inverse agonist / antagonist being developed by Inversago for the treatment of PWS and non-alcoholic steatohepatitis (NASH). It is specifically designed to interact with CB1 receptors outside of the brain (“peripheral” CB1 receptors); those located in the gastro-intestinal tract, liver, pancreas, fat tissues, muscles, lungs and other organs, thus aiming for a safe and effective therapeutic approach without the known risks of CB1 blockers that act in the brain. Peripheral CB1 blockade is a well-documented pathway, linked to many clinically meaningful metabolic benefits.

The RPD designation is open to drug development programs targeting serious, life-threatening diseases that primarily affect less than 200,000 Americans aged 18 or younger. This program provides the incentive such that once a new drug application is approved under this program, the company is eligible to receive a priority review voucher for any related marketing application. Such a voucher may also be sold or transferred.

Inversago is one of several companies that are part of FPWR's venture philanthropy program. "The goal of the program is to balance early and late stage research investments to accelerate the delivery of new therapeutics for your loved one," said John Walter, CEO FPWR. "Your support has helped advance Inversago’s work from preclinical to an early phase I clinical trial and if successful, a phase II clinical trial in PWS could be launched as early as 2022."

 “We’re thrilled to see the continued development of this promising drug for PWS” said Theresa Strong, Director of Research Programs at FPWR.  “The endocannabinoid pathway targeted by INV-101 has been shown to be altered in PWS, and preclinical data suggests that INV-101 may be well suited to address the metabolic disturbances associated with PWS.”






Topics: Clinical Trials Opportunities

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 future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.