Share Price, page-640

Re Prader-Willi syndrome, both Levo Therapeutics and Soleno completed Phase 3 studies in this indication last year and set about preparing NDAs for submission to the FDA.

Unfortunately, Soleno’s drug, DCCR, didn’t meet primary endpoint in its Phase 3 study and in April this year, the FDA informed Soleno that an additional trial would need to be completed.

Levo Therapeutic’s drug, intranasal carbetocin, aims to treat the hyperphagia and behavioural distress caused by Prader-Willi syndrome. Levo submitted its NDA mid-year. In its Phase 3 study, a higher dose tested didn’t meet the primary outcome, as it failed to reduce hyperphagia or obsessive-compulsive behaviours. While a lower dose tested also didn’t reduce obsessive-compulsive behaviours, it did demonstrate statistically significant reduction in hyperphagia, anxiety, and distress over an eight-week treatment period.

Early last month, an FDA Advisory Committee voted 12 to 1 against recommending the approval of carbetocin on the grounds that substantial efficacy wasn't proven. This was despite the fact that there are currently no approved therapies for the condition and that the advisory committee had received more than 100 comments from the Prader-Willi community seeking its support of carbetocin’s approval for the treatment of hyperphagia.

That final decision on carbetocin is expected to come this week (I'll keep an eye out). Most of the time, the FDA’s final decision follows the Advisory Committee’s recommendation.

Rejection by the FDA would likely hit the Prader-Willi community hard following earlier disappointments. Last year, Millendo Therapeutics made the decision to discontinue late stage development of livoletide in Prader-Willi and in 2016, a Phase 3 Prader-Willi trial was abandoned by Zafgen after the death of two trial participants.