ConSynance Therapeutics on Tuesday has reported positive results from its Phase I trial of CSTI-500, a first-in-class drug for the rare central nervous system disorder (CNS).

Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder that affects both males and females from birth and throughout their lives. All individuals with PWS have some cognitive impairment that ranges from low normal intelligence with learning disabilities to mild to moderate intellectual disability.

The National Organization for Rare Disease (NORD) , estimates that the incidence of PWS is between 1 in 10,000-30,000 individuals in the general population and about 350,000-400,000 individuals worldwide.

The single-centre, open-label Phase I trial (NCT05504395) of 14 participants reported no severe adverse events and an efficacy profile equitable to CSTI-500’s earlier trial in healthy volunteers.

Additionally, ConSynance, the New York based start-up, reported that the bioequivalence of their newly developed capsule formulation is confirmed by this Phase I pharmacokinetics data.

CSTI-500 is a first-in-class, orally administered New Chemical Entity (NCE) and an optimally balanced triple monoamine reuptake Inhibitor (TRI). It is designed to regulate the reuptake of serotonin, dopamine, and norepinephrine—three vital neurotransmitters involved in many neuropsychiatric and neurological functions, such as feeding behaviours, mood, and sleep.

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Current PWS treatment is geared toward early intervention and treatment of specific symptoms. Common treatments include growth hormone (GH) therapy, treatment of hypogonadism with either testosterone or human chorionic gonadotropin, and sex hormone therapy during puberty.

“CSTI-500 has the potential to significantly improve the lives of PWS patients and their loved ones by addressing two of the most debilitating symptoms – hyperphagia and temper outbursts,” said Shuang Liu, CEO and founder of the New York based start-up.

Pharmaceutical Technology’s analysis of the rapid growth of rare drugs and orphan drug designations found most orphan therapies are currently in Phase II clinical trials (18%), with 16% in market as of 2022.