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ConSynance Therapeutics Receives FDA Rare Pediatric Disease Designation for CSTI-500 in Prader-Willi Syndrome

  • ConSynance Therapeutics received FDA Rare Pediatric Disease Designation for CSTI-500, an investigational drug for Prader-Willi Syndrome (PWS) treatment in children and adolescents.
  • CSTI-500, a first-in-class Triple Monoamine Reuptake Inhibitor (TRI), targets neurotransmitter imbalances associated with hyperphagia and temper outbursts in PWS patients.
  • The RPDD makes ConSynance eligible for a Priority Review Voucher upon CSTI-500's FDA approval, potentially worth approximately $100 million.
  • A Phase 2 study of CSTI-500 is expected to commence in 2025, building on promising Phase 1 data in healthy volunteers and PWS patients.
ConSynance Therapeutics, Inc. has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for CSTI-500, an investigational drug intended for the treatment of Prader-Willi Syndrome (PWS) in children and adolescents. This regulatory milestone supports ConSynance's preparations for a Phase 2 study of CSTI-500, planned to commence in 2025.

Significance of RPDD

The FDA grants RPDD for diseases affecting fewer than 200,000 individuals in the U.S., which are serious or life-threatening and primarily affect those under 18 years of age. Shuang Liu, PhD, Founder and CEO of ConSynance, emphasized the importance of this designation, noting that CSTI-500 is the first drug candidate designed to address both hyperphagia and severe temper outbursts associated with PWS, offering a potential breakthrough treatment.

About CSTI-500

CSTI-500 is a Phase 2-ready, first-in-class, orally administered Triple Monoamine Reuptake Inhibitor (TRI). It is designed to increase the levels of serotonin, dopamine, and norepinephrine in the synaptic clefts of neurons. PWS is associated with low levels of these neurotransmitters, and CSTI-500 has demonstrated brain target engagement in healthy volunteers via positron emission tomography (PET). These data, along with pharmacokinetics data, predict the efficacy of CSTI-500 in PWS and other neuropsychiatric indications, enabling a personalized dosing approach to ensure optimal safety and efficacy. CSTI-500 has been studied in nearly 100 humans across three Phase 1 clinical trials, including 10 PWS patients.

Prader-Willi Syndrome (PWS)

PWS is a rare, complex genetic neurodevelopmental disorder affecting approximately 1 in 15,000 live births. It is characterized by severe hypotonia and failure to thrive, evolving into life-threatening hyperphagia and neuropsychiatric behaviors, including severe temper outbursts. There are currently no approved therapies that address the core dysfunctional brain networks associated with abnormal levels of monoamines in PWS. Normalizing these neurotransmitters may restore functional neural circuits, alleviating hyperphagia, temper outbursts, and other behavioral symptoms.

Financial Implications

The RPDD makes ConSynance eligible to receive a transferable Priority Review Voucher if CSTI-500 receives FDA approval for PWS. Priority Review Vouchers have recently sold for approximately $100 million, highlighting their significant value.
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Reference News

[1]
ConSynance Therapeutics Receives FDA Rare Pediatric Disease Designation for CSTI-500 in Prader-Willi Syndrome
drugs.com · Jul 24, 2024

ConSynance Therapeutics receives FDA Rare Pediatric Disease Designation for CSTI-500 in Prader-Willi Syndrome, aiming to...

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