SEONGNAM, South
Korea and BALTIMORE, Oct. 25, 2017 /PRNewswire/ -- 1ST
Biotherapeutics, Inc. (1ST
Bio), a private Korean biotech company, and Neuraly Inc., a startup from Johns Hopkins School of
Medicine, today announced a collaboration to co-develop novel brain
penetrant c-Abl inhibitors for Parkinson's disease. Recent research has shown
that inhibitors to c-Abl could slow the progression of Parkinson's disease.
Parkinson's disease, a chronic and progressive movement disorder, is the second
most common neurodegenerative disease, affecting nearly one million people in the United States.
"While FDA-approved and marketed anticancer drugs that
block c-Abl are available for clinical research studies, these drugs are not
designed to cross the blood-brain barrier and have toxicity issues that could
prevent long-term therapy for neurodegenerative diseases," said Jamie
Jae Eun Kim, founder and CEO of 1ST Biotherapeutics. "The goal of the
collaboration between 1ST Biotherapeutics and Neuraly is to develop novel c-Abl
inhibitor drugs that have desirable properties for CNS therapy such as optimal
blood-brain barrier penetration and improved safety profile. We look forward to
collaborating with the researchers at Neuraly, who have performed important
research into c-Abl's pathogenic role in the neurodegeneration seen in
Parkinson's disease."
The pathology of Parkinson's disease is characterized by the
accumulation and aggregation of a protein called a-synuclein into intracellular
clumps called Lewy bodies. Lewy bodies form in nerve cells, disrupting normal
signaling, and eventually causing neuronal cell death. From postmortem analysis
of patient samples, animal models, and in vitro studies,
hyperactivity of c-Abl, a tyrosine kinase signaling protein, was shown to be
associated with a-synuclein aggregation, neuropathology, and neurobehavioral
deficits (Brahmachari S., et al, Journal
of Clinical Investigation). This study, led by Drs. Han
Seok Ko and Ted Dawson at Johns Hopkins , the
cofounders of Neuraly, and other research suggests that pharmacological
inhibition of c-Abl could be a novel therapeutic intervention for Parkinson's
disease.
"Our research confirmed that inhibiting c-Abl activity had
a direct effect on a-synuclein aggregation and disease progression,"
said Seulki Lee, Ph.D., interim CEO of Neuraly and assistant
professor at Johns Hopkins School of Medicine. "We believe c-Abl is a
promising target for pharmacological intervention, but we need to develop a
neurotherapeutic that can penetrate the blood-brain barrier and has the proper
pharmacokinetics. We look forward to working with 1ST Biotherapeutics, who has
identified small molecule preclinical candidates with improved pharmacological
properties and safety profiles."
Under the terms of the collaboration, 1ST Biotherapeutics will
perform the preclinical development, and Neuraly will test the drug candidates
in various preclinical models of the disease. The goal is to file an
Investigational New Drug application with the FDA next year. 1ST
Biotherapeutics will retain all intellectual property generated in the
collaboration. Financial and other terms were not disclosed.
About Neuraly, Inc.
Neuraly is a private biotech company that is developing
therapeutics for neurodegenerative diseases, including Parkinson's and
Alzheimer's diseases. Neuraly is a startup from Johns Hopkins
University. For more info, please visit https://ventures.jhu.edu/learn-about-jhu-startups/.
About 1ST Biotherapeutics, Inc.
1ST Biotherapeutics (1ST Bio) is dedicated to developing
breakthrough therapies in the areas of immuno-oncology, neurodegenerative
diseases, and orphan diseases. 1ST Bio is focused on discovery and development
of innovative first-in-class small molecule therapeutic candidates with high
likelihood of clinical success. For more info, please visit http://www.1stbio.com/.
SOURCE 1ST Biotherapeutics, Inc.
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