ConSynance Therapeutics Announces U.S. FDA Grants Rare Pediatric Disease Designation to CSTI-500, a Potential First-in-Class Therapy for Prader-Willi Syndrome
24 Julio 2024 - 9:00AM
ConSynance Therapeutics, Inc., a clinical-stage biopharmaceutical
company developing first-in-class therapies for rare central
nervous system (CNS) disorders, today announced that the U.S. Food
and Drug Administration (FDA) has granted Rare Pediatric Disease
Designation (RPDD) to its investigational drug CSTI-500 for the
treatment of Prader-Willi Syndrome (PWS) in children and
adolescents.
“Receiving RPDD from the FDA is a significant regulatory
milestone for ConSynance as we prepare for the Phase 2 study of
CSTI-500 for PWS,” said Shuang Liu, PhD, Founder and CEO of
ConSynance. “PWS is a rare neurodevelopmental disorder
characterized by hyperphagia and severe temper outbursts, among
other neuropsychiatric behaviors. CSTI-500 targets the imbalance of
three key neurotransmitters, serotonin, dopamine and norepinephrine
associated with these behaviors. CSTI-500 stands out as the first
drug candidate for PWS designed to address both hyperphagia and
severe temper outbursts, providing a potential breakthrough and
comprehensive treatment solution for this challenging
disorder.”
The FDA defines rare pediatric diseases as conditions affecting
fewer than 200,000 individuals in the United States, which are
serious or life-threatening and primarily impact those under 18
years of age. This designation 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.
More About CSTI-500: CSTI-500 is a phase
2-ready, first-in-class, orally administered Triple Monoamine
Reuptake Inhibitor (TRI). It is designed to optimally increase the
levels of three critical neurotransmitters, serotonin, dopamine,
and norepinephrine in the synaptic clefts of neurons. Human and
animal data suggest that PWS is associated with low levels of these
neurotransmitters. Deficiencies in these neurotransmitters are also
implicated in many other neuropsychiatric disorders, as evidenced
by the approval of selective serotonin reuptake inhibitors,
selective norepinephrine reuptake inhibitors, and dopamine
norepinephrine reuptake inhibitors in conditions like binge eating
disorder (BED), obsessive compulsive disorders (OCD), attention
deficit hyperactivity disorder (ADHD), among others. CSTI-500 has
demonstrated brain target engagement in healthy volunteers via
positron emission tomography (PET). These data, along with the
corresponding pharmacokinetics data, predict the efficacy of
CSTI-500 in PWS and other neuropsychiatric indications, and enable
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, setting
the stage for a Phase 2 study expected to commence in 2025.
About PWS: PWS is a rare, complex genetic
neurodevelopmental disorder that affects approximately 1 in 15,000
live births. Initially characterized by severe hypotonia and
failure to thrive, it evolves into a life-threatening hyperphagia
beginning in early childhood, compounded by other debilitating
neuropsychiatric behaviors, notably severe temper outbursts.
Despite significant challenges faced by PWS patients and their
families, there are currently no approved therapies that address
the core dysfunctional brain networks associated with abnormal
levels of monoamines and consequent neuropsychiatric behaviors in
PWS. Normalizing these neurotransmitters may restore functional
neural circuits, alleviating hyperphagia, temper outbursts, among
other behavioral symptoms that significantly impact the patients
and their families.
About ConSynance Therapeutics: ConSynance is a
New York-based biopharmaceutical company dedicated to developing
innovative therapies for rare CNS disorders. ConSynance is a member
of the PWS Clinical Trial Consortium and engaged in an ongoing
partnership with Harmony Biosciences to develop HBS-102 (formerly
known as CSTI-100) for the treatment of a variety of rare
neurological diseases. These partnerships underscore the company's
collaborative approach and its drive to bring effective therapies
to patients in need.
For more information, please visit www.ConSynance.com
Forward-Looking Statements: This press release
contains forward-looking statements based on ConSynance
Therapeutics Inc.'s current expectations and beliefs. These
statements are subject to various risks and uncertainties, and
actual results may differ. ConSynance Therapeutics Inc. accepts no
obligation to update or revise any forward-looking statement,
except as required by law.
Contact Information:Sarah Sheldrick,
PhDConSynance Therapeutics, Inc.sarah@consynance.com