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Prader-Willi syndrome

FDA grants Fast Track designation to Carbetocin for PWS patients

CARE-PWS study is a multi-centre, randomised, double-blind, placebo-controlled study

The FDA has granted Fast Track designation for Levo Therapeutics’ LV-101 (intranasal carbetocin) for the treatment of Prader-Willi syndrome (PWS), a complex, multisystem neurodevelopmental disorder that occurs in approximately 1 in 16,000 births. 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.

Levo is currently enrolling participants at over 20 clinical study sites throughout the US and Canada in its Phase 3 clinical study of intranasal carbetocin for the treatment of PWS. The study is a multi-centre, randomised, double-blind, placebo-controlled study called CARE-PWS. The company anticipates additional study sites opening in Australia soon. The study has an eight-week period designed to test the effectiveness, safety, and tolerability of LV-101 in participants with PWS.

Carbetocin is an analogue 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.

Effectiveness will be measured using both caregiver-reported and clinician-reported measures of hyperphagia (extreme hunger), obsessive and compulsive behaviours, 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 eight-week placebo-controlled period, during a long-term follow-up period of 56 weeks. At week eight, participants who were randomised to placebo in the placebo-controlled period will be randomised 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.

"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 enrolment of this important clinical study in the coming months."

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