Positive Phase 2 Study Results of Trofinetide in Pediatric Rett Syndrome Published in Neurology, the Medical Journal of the American Academy of Neurology
-- Statistically Significant Improvement Indicates Trofinetide’s
Potential for Treating Rett Syndrome
-- Trofinetide for Rett Syndrome has Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Drug Designation in
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The study, “Double-Blind,
Randomized, Placebo-Controlled Study of Trofinetide in Pediatric Rett
Syndrome” has been published online and will appear in the
“Disease burden is severe for Rett patients and their families, and the
impact of the disorder is life-long,” said
In 2018, ACADIA entered into an exclusive North American license agreement with Neuren for the development and commercialization of trofinetide for Rett syndrome and other indications.
“Rett syndrome is a condition that leads to severe neurological
impairments and is not only debilitating for the person with the
disease, but also very hard on the families and caregivers of the
children, mostly females, who are often unable to speak, walk, eat, and
even breathe normally,” said
ACADIA plans to initiate a 12-week Phase 3 double-blind, randomized, placebo-controlled study evaluating trofinetide in the second half of 2019 following completion of additional manufacturing scale-up activities. This study will evaluate efficacy and safety of trofinetide and placebo in approximately 180 females ages 5 to 20 years with Rett syndrome. Half of the study participants will receive trofinetide and half will receive placebo. The study will use the RSBQ and the CGI-I syndrome specific efficacy measures as co-primary efficacy endpoints. The Phase 3 study will be followed by a nine month open label extension study in which all participants, including those on placebo in the Phase 3 study, will be eligible to receive trofinetide. In the open label extension study, all participants will be followed to evaluate long term tolerability and safety of trofinetide.
“Neuren has successfully led the clinical development of trofinetide to
date, which allows us to further evaluate trofinetide as a potentially
important treatment option for Rett syndrome,” said
Phase 2 Study Design
In the Phase 2 study, 82 females were enrolled and randomized to receive liquid trofinetide or placebo orally or via gastrostomy tube for six weeks: 24 subjects to placebo BID, 15 subjects to 50 mg/kg of trofinetide BID, 16 subjects to 100 mg/kg of trofinetide BID, and 27 subjects to 200 mg/kg of trofinetide BID. Safety and tolerability assessments included adverse events (AEs), clinical laboratory tests, physical examinations, and concomitant medications.
The five core efficacy endpoints included three clinician-completed measures and two caregiver-completed measures.
- The RSBQ, a rating scale in which the subject’s caregiver rates the frequency of symptoms.
- The CGI-I, in which the clinician rates how much the subject’s overall illness has improved or worsened, relative to baseline.
- The RTT-DSC, in which the clinician assesses - on a visual analog scale - the severity of concerns identified for each subject on an individual basis.
- The Motor Behavioral Assessment, a rating scale in which the clinician rates the subject’s current level of function.
- The Caregiver Top 3 Concerns, in which the subject’s caregiver assesses - on a visual analog scale - the severity of concerns identified for each subject on an individual basis.
Trofinetide is a novel synthetic analog of the amino‐terminal tripeptide
of IGF-1 designed to treat the core symptoms of Rett syndrome by
reducing neuroinflammation and supporting synaptic function. In the
central nervous system, IGF-1 is produced by both of the major types of
brain cells – neurons and glia. IGF-1 in the brain is critical for both
normal development and for response to injury and disease. Trofinetide
has been granted Fast Track Status and Orphan Drug Designation in the
U.S. and Orphan Drug Designation in
About Rett Syndrome
Rett syndrome is a debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life. Rett syndrome has been most often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Rett syndrome is caused by mutations on the X chromosome on a gene called MeCP2. There are more than 200 different mutations found on the MeCP2 gene that interfere with its ability to generate a normal gene product. Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births causing problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. Typically, between six to 18 months of age, patients experience a period of rapid decline with loss of purposeful hand use and spoken communication and inability to independently conduct activities of daily living. Symptoms also include seizures, disorganized breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis), and sleep disturbances. Currently, there are no approved medicines for the treatment of Rett syndrome.
ACADIA is a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in central nervous system disorders. ACADIA has developed and is commercializing the first and only medicine approved for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis. In addition, ACADIA has ongoing clinical development efforts in additional areas with significant unmet need, including dementia-related psychosis, schizophrenia inadequate response, schizophrenia-negative symptoms, major depressive disorder, and Rett syndrome. This press release and further information about ACADIA can be found at: www.acadia-pharm.com.
Rettsyndrome.org (RSO) is one of the leading private funders of Rett
syndrome research, investing over
Statements in this press release that are not strictly historical in
nature are forward-looking statements. These statements include but are
not limited to statements regarding the timing of the commencement of
the Phase 3 clinical trial evaluating trofinetide; the likelihood of
success of such clinical trial; the prospects for
ACADIA Pharmaceuticals Inc.
Mark Johnson, CFA
Jon Pilcher, Chief Financial Officer
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