Results of HARMONY CTP Phase III trial of Wakix (pitolisant) in narcoleptic patients published in Lancet Neurology- BioProjet Pharma

The Lancet Neurology has published the results from the HARMONY CTP Phase III controlled trial of Wakix (pitolisant), from BioProjet Pharma, in severely affected narcoleptic patients with cataplexy. The trial showed that pitolisant, the first drug promoting histamine neurotransmissions in brain, and previously shown to be active against EDS, was also able to significantly reduce cataplexy when given once-a-day for two months in severely affected patients. Cataplexy attacks (on which psychostimulants like amphetamines or modafinil are not significantly active) were reduced, by up to 75% (50% compared to placebo), even in severely affected patients experiencing nearly 10 attacks per week on average.

The number of patients with very high cataplexy level at the end of treatment (more than 15 attacks per week) were also more than 3 times less frequent with pitolisant than placebo. In addition to cataplexy, EDS was significantly improved when evaluated using either a subjective test (Epworth) or an objective laboratory test (MWT, Maintenance of Wakefulness Test, which measures the delay after which patients fall asleep under a dim light). This wake-promoting effect confirms previous trials with pitolisant.

See: "Safety and efficacy of pitolisant on cataplexy in patients with narcolepsy: a randomised, double-blind, placebo-controlled trial" Prof Zoltan Szakacs et al. The Lancet Neurology Online first Published: 24 January 2017 DOI: http://dx.doi.org/10.1016/S1474-4422(16)30333-7