ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Exclusive License Agreement for the North American Development and Commercialization of Trofinetide in Rett Syndrome
-
-Neuren retains all rights to trofinetide outside of
-Neuren to receive US
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“A potential treatment for Rett syndrome is a perfect fit with ACADIA’s
mission to develop novel therapies to improve the lives of patients with
central nervous system disorders,” said
Neuren Executive Chairman Dr.
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 disease1,2.
Trofinetide has been granted
Neuren conducted a Phase 2 double-blind placebo-controlled dose ranging study in girls aged 5 to 15 years with Rett syndrome, in which statistically significant and clinically meaningful improvement was demonstrated on the RSBQ and the CGI-I. This followed positive trends observed in an earlier Phase 2 trial in adolescents and adults aged 16 to 45 years with Rett syndrome3. In addition, Neuren has completed an exploratory study in Fragile X syndrome.
Dr.
Under the terms of the License Agreement, Neuren is eligible to receive
US
About Trofinetide
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 disease1,2. Trofinetide has been granted U.S. FDA Fast
Track Status and Orphan Drug Designation in the U.S. and
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 MeCP24. There are more
than 200 different mutations found on the MeCP2 gene that interfere with
its ability to generate a normal gene product4. Rett syndrome
occurs worldwide in approximately one of every 10,000 to 15,000 female
births5 causing problems in brain function that are
responsible for cognitive, sensory, emotional, motor and autonomic
function. Typically, between six to eighteen 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 living5. 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 approved for the treatment of Rett syndrome1.
About Fragile X syndrome
Fragile X syndrome is the most
common inherited cause of intellectual disability and the most common
known cause of autism. Fragile X syndrome is caused by a single gene
defect on the X chromosome that impacts the FMRP protein, which is
responsible for regulating the synapses of nerve cells. One of every
5,000 males and one of every 4,000 to 8,000 females are estimated to
have the full gene mutation6. Generally males are more
severely affected than females. Clinically, Fragile X syndrome is
characterized by intellectual disability, hyperactivity and attentional
problems, autistic symptoms, anxiety, emotional lability and epilepsy.
Currently, there are no medicines approved for the treatment of Fragile
X syndrome.
About
About
About Rettsyndrome.org
Rettsyndrome.org
is one of the leading private funders of Rett syndrome research. The
organization hosts the largest global gathering of Rett researchers and
clinicians to establish research direction for the future. The mission
of the organization is to accelerate the full spectrum research to cure
Rett syndrome and empower families with information, knowledge and
connectivity. Further information about Rettsyndrome.org can
be found at: rettsyndrome.org.
Forward-Looking Statements
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
This ASX-announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.
References
1
2Vahdatpour, C., et al. (2016). "Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders." Front Neurosci 10: 450.
3Glaze, D. G., et al. (2017). "A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Rett Syndrome." Pediatr Neurol 76: 37-46.
4Chahrour, M. and
5Ip, J. P. K., et al. (2018). "Rett syndrome: insights into genetic, molecular and circuit mechanisms." Nat Rev Neurosci 19(6): 368-382.
6Hagerman, R.J., et al. (2017). “Fragile X syndrome.” Nat Rev Disease Primers 3: 1-19.
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Source:
ACADIA Pharmaceuticals Inc.
Investor Contact:
Elena
Ridloff, CFA
(858) 558-2871
ir@acadia-pharm.com
or
Media
Contact:
Bob Laverty
(609) 558-5570
bob@taftcommunications.com
or
Neuren
Pharmaceuticals Limited
Contact:
Dr. Richard Treagus
Executive
Chairman
+61 417 520 509
rtreagus@neurenpharma.com