Science & Technology

Intranasal Drug Delivery

Intranasal drug delivery is a non-invasive approach that directly targets the brain, offering a fast and efficient alternative to traditional drug administration methods. Unlike oral or intravenous delivery, which must navigate systemic circulation and the blood-brain barrier (BBB), intranasal administration leverages the olfactory and trigeminal nerve pathways to bypass the BBB and deliver therapeutics directly to the central nervous system (CNS). This rapid and targeted transport allows for significantly higher drug concentrations in the brain while minimizing systemic exposure and potential side effects.

For the treatment of concussions, neurodegenerative diseases, and rare leukodystrophies, this targeted approach is particularly advantageous. Concussions require immediate intervention to reduce inflammation, oxidative stress, and neuronal damage, making intranasal delivery ideal for fast-acting neuroprotective treatments. In neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and multiple sclerosis, intranasal drug delivery facilitates sustained therapeutic action by efficiently reaching affected brain regions without requiring excessive systemic doses. Similarly, for rare leukodystrophies, where lipid and metabolic dysfunctions compromise brain function, direct-to-brain drug delivery ensures that key compounds reach diseased cells without being diluted or metabolized in peripheral circulation.

By pairing this proven physiological pathway with an advanced self-administered or automatic intranasal device, patients receive precise, reproducible dosing in a way that is both patient-friendly and clinically effective. This dual-device strategy not only enhances drug efficacy but also improves patient compliance, making intranasal drug delivery a transformative solution for brain-targeted therapeutics.

Novel Neurosteroids

Novel neurosteroids, like ONP-002 and ONP-001, represent a breakthrough therapeutic approach for concussions, neurodegenerative diseases, and rare leukodystrophies by leveraging their powerful neuroprotective, anti-inflammatory, and regenerative properties. Unlike traditional treatments that often address only symptoms, neurosteroids work at the cellular and molecular level, modulating brain function to promote recovery and long-term resilience. They act on intracellular steroid receptors within neurons, glial cells, and the blood-brain barrier, triggering gene expression pathways that reduce inflammation, oxidative stress, and neuronal swelling—key contributors to progressive brain damage. Additionally, neurosteroids enhance synaptogenesis and myelin repair, which are critical for restoring cognitive and motor function in neurodegenerative diseases and leukodystrophies.

In the case of concussions or mild traumatic brain injuries (mTBI), neurosteroids provide rapid intervention by stabilizing cell membranes, reducing secondary brain injury, and preventing long-term complications such as post-concussion syndrome or chronic traumatic encephalopathy (CTE). For neurodegenerative conditions like Alzheimer’s, Parkinson’s, and multiple sclerosis, neurosteroids help mitigate disease progression by modulating neuroinflammation, protecting against neurotoxicity, and promoting neuronal survival. In rare leukodystrophies, such as Niemann-Pick Type C, where defective lipid metabolism leads to severe neurological decline, neurosteroids activate transport mechanisms that clear harmful lipid accumulation from brain cells while supporting myelin integrity. With their ability to cross the blood-brain barrier efficiently—especially when delivered intranasally—neurosteroids offer a promising, next-generation therapeutic option that directly targets the root causes of these debilitating conditions, providing both immediate symptom relief and long-term neuroprotection.