Levo Therapeutics Receives Fast Track Designation From FDA for Intranasal Carbetocin for the Treatment of Prader-Willi Syndrome

CHICAGO, Nov. 18, 2019 /PRNewswire/ -- Levo Therapeutics, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for LV-101 (intranasal carbetocin) for the treatment of Prader-Willi syndrome (PWS). Levo is currently enrolling participants in its Phase 3 clinical study of intranasal carbetocin for the treatment of PWS. The study is a multi-center, randomized, double-blind, placebo-controlled study called CARE-PWS.

The FDA may designate a drug as a Fast Track product if the drug is intended, whether alone or in combination with one or more other drugs, for the treatment of a serious or life-threatening disease or condition, and it demonstrates the potential to address unmet medical needs for the disease or condition. Fast Track designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.

"We are thrilled to receive Fast Track designation from the FDA, which supports the understanding among the PWS community that this syndrome presents serious and life-threatening issues," said Sara Cotter, CEO of Levo Therapeutics. "Levo is committed to developing impactful treatments for patients with PWS, and our Phase 3 study is designed to see whether intranasal carbetocin provides one such treatment. We look forward to finishing enrollment of this important clinical study in the coming months."

The Phase 3 CARE-PWS study is currently enrolling study participants at over 20 clinical study sites throughout the United States and Canada, and Levo anticipates additional study sites opening in Australia soon. Listings of clinical study sites for the CARE-PWS study are kept updated on clinicaltrials.gov (study NCT03649477) and at www.levotx.com/care-pws.

Levo also announced today that it has completed a Series B financing round, co-led by Samsara BioCapital and an undisclosed healthcare investment fund. Proceeds from the financing will be used to support the Phase 3 CARE-PWS Study, to further the development of LV-101, and to advance Levo's pipeline programs.

About CARE-PWS (CARbetocin Efficacy and Safety Study in PWS)

This multi-center, randomized, double-blind, placebo-controlled study has an 8-week period designed to test the effectiveness, safety, and tolerability of LV-101 in participants with PWS.

Effectiveness will be measured using both caregiver-reported and clinician-reported measures of hyperphagia (extreme hunger), obsessive and compulsive behaviors, and anxiety. Safety and tolerability will be measured by adverse events, laboratory tests, and physical exams.

All participants will receive active treatment with LV-101 after the 8-week placebo-controlled period, during a long-term follow-up period of 56 weeks. At Week 8, participants who were randomized to placebo in the placebo-controlled period will be randomized to one of the two LV-101 doses, administered three times per day before meals. The study also includes an optional extension period, where subjects participating in the long-term follow-up period will have an opportunity to continue receiving LV-101.

About Intranasal Carbetocin

Carbetocin is an analog of the naturally-occurring neuroendocrine hormone oxytocin. Carbetocin was designed to have an improved receptor binding profile compared to oxytocin, with greater affinity for the oxytocin receptor and lower affinity for related vasopressin receptors. It is approved in over 90 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery, with an estimated cumulative exposure of over 10 million patients. LV-101 is an investigational intranasal form of carbetocin, intended to be administered to patients with PWS three times each day before meals.

For additional background information, the results of the Phase 2 study of intranasal carbetocin (LV-101) in patients with genetically confirmed PWS can be found online at JCI Insight.

About Prader-Willi Syndrome (PWS)

Prader-Willi syndrome (PWS) is a complex, multisystem neurodevelopmental disorder that occurs in approximately 1 in 16,000 births¹. The underlying cause of PWS is the lack of expression of paternally-inherited imprinted genes on chromosome 15q11-q13. These genetic anomalies lead to a distinctive phenotype that includes mild to moderate levels of intellectual disability, compulsivity, growth hormone deficiency, life-threatening hyperphagia, and high risk of obesity.

About Levo Therapeutics, Inc.

Levo Therapeutics is a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome and related disorders. To learn more about Levo, please visit www.levotx.com.

For further information:

Levo Therapeutics, Inc.
Tel: 847-901-9260
[email protected]

¹Burd L, Vesely B, Martsolf J, Kerbeshian J. Prevalence study of Prader-Willi syndrome in North Dakota. Am J Med Genet. 1990; 37:97-9.

SOURCE Levo Therapeutics, Inc.