Levo Therapeutics Announces Publication of Phase 2 Study Results and Planned Phase 3 Study of Intranasal Carbetocin for the Treatment of Prader-Willi Syndrome

Thursday, June 21, 2018 General News
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CHICAGO, June 21, 2018 /PRNewswire/ -- Levo Therapeutics Inc. announced today that JCI Insight has published the results

of a Phase 2 study of intranasal carbetocin (LV-101) in patients with genetically confirmed Prader-Willi syndrome (PWS). Results from the study suggest that the investigational product LV-101 has marked effects on core features of PWS, including hyperphagia
and obsessive-compulsive symptoms. Although the Phase 2 study was conducted in 2014, today's publication is the first time that these data have been made fully available to the public.

"Levo is excited that these data are now available to the PWS community, as they show the potential of LV-101 to treat this rare genetic syndrome," said Sara Cotter, CEO of Levo Therapeutics. "LV-101 showed meaningful improvements in all primary and secondary endpoints. Building on these promising data, we look forward to bringing intranasal carbetocin back into the clinic later this year."

The publication, entitled Intranasal carbetocin reduces hyperphagia in individuals with Prader-Willi syndrome, can be found online at JCI Insight.

Summary of LV-101 Phase 2 study and data

The Phase 2 clinical study was a 15-day randomized, double-blinded, placebo-controlled, multi-center study of 37 patients with genetically confirmed PWS (17 patients, aged 10-18 years, were randomized to the carbetocin arm and received drug; 20 to placebo). The primary outcome measure of the study was the change from baseline in the Hyperphagia in PWS Questionnaire-Responsiveness (HPWSQ-R) total score in patients receiving LV-101 versus patients receiving placebo. Secondary outcome measures included the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) total score, Clinical Global Impressions of PWS (CGI) and the Food Domain of the Reiss Profile. LV-101 was administered intranasally three times per day before meals.

In the primary efficacy endpoint analysis, patients who received LV-101 had marked and clinically relevant reductions from baseline in the parent/caregiver-rated HPWSQ-R total score at end of treatment (least squares mean change of ?15.6) compared with those who received placebo (–8.9; one-sided p=0.029). Statistically significant improvements were also observed for secondary endpoints CY-BOCS (one-sided p=0.005) and the Food Domain of the Reiss Profile scores (one-sided p=0.01). In addition, secondary analysis of CGI–Improvement total scores indicated a statistically significant treatment effect of carbetocin at study end (one-sided p=0.023). Highly statistically significant correlations were found between the primary endpoint change in HPWSQ-R total score and all supportive secondary endpoints including change in CY-BOCS total score, change in the Food Domain of the Reiss Profile and CGI-I score (r=0.60–0.93; one-sided p?0.0001), indicating that treatment-related improvements in hyperphagia were associated with overall symptom improvement.

The incidence of treatment-emergent adverse events (TEAEs) occurring in at least five percent of either study group was evenly distributed across the carbetocin and placebo groups. The most common TEAE was headache, reported in five patients who received carbetocin and six who received placebo (overall 29.7 percent). No severe TEAEs were observed during the study.

The Phase 2 study was sponsored by Ferring Pharmaceuticals.  In August of 2017, Levo Therapeutics acquired from Ferring the exclusive rights to continue development of intranasal carbetocin for the treatment of PWS.

About intranasal carbetocin (LV-101)

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 80 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery. Carbetocin has a well-established safety profile, with an estimated cumulative exposure of over 10.5 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.

About Prader-Willi syndrome (PWS)

Prader-Willi syndrome (PWS) is a complex, multisystem neurodevelopmental disorder that occurs in approximately one 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: 847-901-9260contactus@levotx.com

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

Cision View original content:http://www.prnewswire.com/news-releases/levo-therapeutics-announces-publication-of-phase-2-study-results-and-planned-phase-3-study-of-intranasal-carbetocin-for-the-treatment-of-prader-willi-syndrome-300670179.html

SOURCE Levo Therapeutics, Inc.



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