Companies team on bringing Parkinson’s treatments to brain
Ophidion, Neuronasal aim for non-invasive delivery approaches
- Ophidion and Neuronasal are teaming up to deliver Parkinson's treatments to the brain.
- Ophidion uses its "Trojan horse" technology to bypass the blood-brain barrier.
- Neuronasal employs noninvasive nose-to-brain delivery for enhanced therapeutic access.
Biotechnology company Ophidion is teaming up with Neuronasal, a clinical-stage company focused on nose-to-brain delivery of therapies for central nervous system disorders, to develop ways to deliver treatments for Parkinson’s disease and other conditions to the brain using noninvasive technology.
The companies said the agreement will leverage Ophidion’s noninvasive technology to transport gene-silencing and large-molecule therapeutics to the brain, along with Neuronasal’s patented nose-to-brain delivery of therapies. The goal is to improve brain exposure and clinical outcomes of Parkinson’s treatments.
“We are excited to partner with Neuronasal and advance these therapeutics with our Central Nervous System (CNS)-delivery platform,” Yacoub Habib, PhD, CEO of Ophidion, said in a company press release. “Parkinson’s disease remains a major unmet need, and by tackling multiple targets simultaneously, we believe we can increase the probability of success and speed translation into patients.”
Treatments for neurodegenerative diseases, including Parkinson’s, can be hindered by the blood-brain barrier (BBB), a semipermeable, tight layer of cells that prevents most molecules from reaching the central nervous system (CNS), which comprises the brain and spinal cord.
‘Trojan horse’ technology crosses BBB
Ophidion said its “Trojan horse” carrier technology is designed to cross the BBB and deliver molecules to the CNS through intravenous (into-the-vein) or subcutaneous (under-the-skin) injections. The technology relies on receptors in the BBB and nerve cells to shuttle cargo into the brain.
The company’s approach supports gene-silencing agents, including small interfering RNA (siRNA) and antisense oligonucleotides (ASOs), as well as proteins and antibodies. Both siRNAs and ASOs bind to messenger RNA, the template molecule produced when a gene is read and is necessary for protein production, and can either block protein production or lead to mRNA degradation.
Neuronasal has developed a nose-to-brain route for N-acetylcysteine (NAC), which has been shown to deliver the drug to the brain in a human pilot trial. NAC is a naturally occurring antioxidant approved to prevent liver damage, which can improve motor and mental abilities of people with Parkinson’s, given by intravenous injection or orally in clinical trials. Intranasal NAC delivery is 50 times more efficient in getting to the brain than intravenous delivery. It is currently being assessed in Phase 1 trials, according to the company.
The companies said the collaboration will extend from proof-of-concept to investigational new drug enabling studies in relevant preclinical models, which are necessary to advance clinical trials in humans. It includes upfront and development milestone payments as well as a joint venture establishment to advance the programs through clinical development.
“Neuronasal is excited to add Ophidion’s brain delivery capability to our Parkinson’s disease developments; combining our expertise with Ophidion’s CNS delivery platform will enhance our clinical program for disease-modifying therapies,” said Thomas I. Bradshaw, CEO of Neuronasal.
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