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Option Therapeutics
Option Therapeutics developed a small-molecule prodrug to replicate the effects of intermittent hypoxia on respiratory function in patients with cervical...
Option Therapeutics
Option Therapeutics developed a small-molecule prodrug to replicate the effects of intermittent hypoxia on respiratory function in patients with cervical spinal cord injury. Its single-asset program advanced through a Phase 2a trial that measured respiratory muscle function in chronic tetraplegia and met the primary endpoint of increased minute ventilation after oral dosing in December 2021. The trial received National Institutes of Health grants and enrolled patients at multiple U.S. spinal cord injury centers, with foundational research supplied by founders from the University of Florida and the Malcom Randall VA Medical Center. No additional clinical or corporate updates have been disclosed since mid-2026.
General information
Firm type
Asset Manager
Frequently asked questions
What was Option Therapeutics' lead drug candidate and its mechanism of action?
The company's lead candidate was an oral small-molecule prodrug designed to be a selective adenosine A2A receptor antagonist. It was administered prior to therapeutic intermittent hypoxia sessions to enhance respiratory motor plasticity. The approach aimed to amplify the body's natural neural adaptive response to low-oxygen conditions in patients with chronic spinal cord injuries, specifically targeting cervical injuries that impair breathing. The underlying mechanism was based on decades of preclinical research demonstrating that A2A receptor blockade can potentiate the functional benefits of intermittent hypoxia on phrenic motor output.
What clinical data did Option Therapeutics generate?
In December 2021, the company announced positive results from a randomized, double-blind, placebo-controlled Phase 2a clinical trial in patients with chronic tetraplegia. The trial met its primary endpoint, demonstrating that the lead candidate significantly increased minute ventilation during breathing trials compared to placebo when combined with a single session of acute intermittent hypoxia. The study was conducted at multiple spinal cord injury research centers across the United States and supported in part by National Institutes of Health grants.
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