ONP-002

ONP-002 is a first-in-class neurosteroid and new chemical entity (NCE) developed for the treatment of concussion (mild traumatic brain injury, mTBI). It is engineered for rapid action and high brain penetration via an intranasal delivery system, which enables direct targeting of the brain after head injury. ONP-002 acts by diffusing intracellularly to induce steroid receptors found in neurons, glia, and the endothelium of the blood-brain barrier. Activation of these receptors leads to gene expression changes that reduce inflammation, oxidative stress, and swelling while promoting macro-autophagy to clear cellular debris—mechanisms believed to prevent long-term neurological damage. Unlike GABAergic compounds, ONP-002 does not cause sedation or fatigue. The drug has demonstrated safety in Phase I trials and is advancing into Phase II clinical studies for acute concussion treatment[1][2][3][4][5][6][8].