Spinogenix Announces Enrollment is Open for its Phase 2 Clinical Trial of SPG601 in Fragile X Syndrome
2024年7月23日 - 9:00PM
Spinogenix, Inc., a clinical-stage biopharmaceutical company
pioneering first-in-class therapeutics that restore synapses to
improve the lives of patients worldwide, today announced that
enrollment is now open for their Phase 2 clinical trial evaluating
SPG601 for the treatment of adult men with Fragile X Syndrome
(FXS), the leading inherited form of intellectual disability and a
known cause of autism. The U.S. Food and Drug Administration (FDA)
recently cleared the Investigational New Drug (IND) application and
granted Orphan Drug Designation for SPG601 for the treatment of
people with FXS.
"Fragile X patients and their families compose an incredible,
yet still underserved community. That is why, during this year’s
National Fragile X Awareness Month, we are especially excited to
announce that we have begun recruiting for our Phase 2 trial
evaluating SPG601 for the treatment of individuals with this
difficult condition. This aligns with our commitment to advancing
research and treatment options to help people with this condition
live better-quality lives, as there are currently no FDA-approved
drugs available for those with FXS," said Craig Erickson, M.D.,
Spinogenix Chief Medical Advisor. “Notably, SPG601 is a novel
once-a-day pill that works by binding to BK channels and increasing
their activation to correct specific synaptic dysfunctions, a
therapeutic approach that we believe can bring much needed hope to
individuals and families struggling with FXS.”
Stella Sarraf, Ph.D., Spinogenix Chief Executive Officer and
Founder, added, "People with FXS and their families face immense
challenges every day. This milestone marks a significant step
forward in our mission to bring new hope to the people and families
affected by this condition. At Spinogenix, we are passionately
working to develop first-in-class therapeutics that restore
synapses to improve the lives of people worldwide and we are
accomplishing this through our trials evaluating SPG601 in FXS and
SPG302 in ALS and Alzheimer’s disease. We look forward to exploring
the potential of our small molecule platform to address a range of
diseases involving synapse loss and dysfunction."
This study is a Phase 2 randomized, double blind,
placebo-controlled, cross over, single dose of SPG601 and matching
placebo in patients with Fragile X syndrome. Enrollment is
currently open at the Cincinnati Children’s Hospital Medical Center
in Cincinnati, Ohio, United States. Additional information on the
Phase 2 trial is available on ClinicalTrials.gov (NCT06413537).
FXS results from the silencing of the Fmr1 gene which
leads to changes in mRNA and protein expression that results in
abnormalities in synaptic function. Some of these abnormalities
have been shown to be a result of a reduction in the amount and
activity of large-conductance, calcium-activated potassium (“BK”)
channels. Many core symptoms of FXS have been linked to deficient
activity of BK channels. In addition to FXS, BK channel expression
and activity have been linked to a range of other conditions
including neurodegenerative, vascular, and sensory disorders.
SPG601 is a novel small molecule BK channel activator that works by
binding to BK channels and increasing their activation to correct
specific synaptic dysfunctions that underlie these conditions.
About Fragile X SyndromeFragile X Syndrome
(FXS) is the leading inherited form of intellectual disability and
a known cause of autism that results from the silencing of
the Fmr1 gene. FXS is an orphan disease affecting
approximately 1 in 4-5000 men and 1 in 6-8000 women globally. In
addition to intellectual disability, FXS patients endure a wide
range of disabling symptoms, including severe anxiety, social
aversion, hyperactivity and attention deficit, sensory
hypersensitivity, aggression, developmental seizures, and others.
Providing care for individuals with FXS often becomes a full-time
commitment for at least one parent and imposes significant
financial strain, with direct family healthcare costs totaling $4.1
billion annually in the United States alone. Despite the
considerable impact of FXS, there are currently no FDA-approved
drugs available for those with the condition.
About Spinogenix Spinogenix is dedicated
to developing transformative therapeutics for conditions involving
the loss or dysfunction of synapses. Our lead clinical-stage
synaptic regenerative candidate is a first-in-class therapeutic
designed to reverse synapse loss and improve cognitive and motor
functions in neurodegenerative and neuropsychiatric diseases such
as ALS, Alzheimer’s disease, and schizophrenia. In parallel, we are
also developing a synaptic function therapeutic designed to improve
behavior in Fragile X Syndrome. More information on Spinogenix can
be found at www.spinogenix.com or follow us on LinkedIn.
Spinogenix Contact
info@spinogenix.com
Media Contact
Kristin Politi, Ph.D.LifeSci
Communicationskpoliti@lifescicomms.com(646) 876-4783