Prader-Willi Syndrome (PWS)
Levo Therapeutics is developing small molecule therapeutics for the treatment of the genetic disease Prader-Willi Syndrome. The lead drug candidate, LV-101 (carbetocin), is an oxytocin analogue. There are currently no treatments for Prader-Willi Syndrome.
LV101(Carbetocin)-Prader-Willi Syndrome is caused by a lack of paternal chromosome 15, either through paternal chromosome deletion or a mis-inheritance of dual copies of the maternal chromosome. This genetic abnormality results in hypothalamic dysfunction in the area of the brain responsible for oxytocin production. The decreased oxytocin causes symptoms such as: anxiety, compulsive behavior, false sense of starvation,and intellectual disability. LV-101 is a synthetic oxytocin analogue that has a higher selectivity for the oxytocin receptor over other receptors that oxytocin binds to. This therapeutic aims to compensate for the lower levels of oxytocin produced by Prader-Willi Syndrome patients. Currently, there is no approved Prader-Willi Syndrome treatment.
Intranasal Carbetocin (LV-101)-Currently recruiting for phase III clinical trial
Peer-Reviewed Journal Article:
Intranasal Carbetocin Reduces Hyperphagia in Individuals with Prader-Willi Syndrome
Dykens, EM; et al. JCI Insight. 2018 Jun 21; 3(12)
Development of Intranasal Carbetocin (LV-101) for the Treatment of Hyperphagia and Behavioural Symptoms in Prader-Willi Syndrome
Dykens, EM; Miller, J; Angulo, M; Roof, E; King, K; Cotter, S. 2018 Annual Meeting of the Pediatric Academic Societies
- Small molecule
- Genetic diseases
- Rare Diseases
Levo Therapeutics has a lead drug candidate in phase III clinical trials and no other therapeutics in preclinical development. Levo may be interested in partnering with industry or academia to develop other compounds for the treatment of Prader-Willi Syndrome.