Our Parkinson's Place: 1ST Bio and Neuraly Collaborate to Advance Novel c-Abl Inhibitors for Parkinson's Disease

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Wednesday, October 25, 2017

1ST Bio and Neuraly Collaborate to Advance Novel c-Abl Inhibitors for Parkinson's Disease

October 25, 2017

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.