NBO Pharma is developing an intranasal formulation of octreotide acetate to treat Idiopathic Intracranial Hypertension (IIH), a condition with no approved drugs. By repurposing the orphan drug octreotide, the company aims to lower intracranial pressure and alleviate headaches and visual symptoms while reducing injection frequency, systemic side effects, and patient discomfort. The intranasal product is currently advancing through clinical trials for IIH patients.
Funding
Funding not disclosed
Founders
Product
Problem
Idiopathic Intracranial Hypertension (IIH) lacks any approved pharmacologic therapy, leaving patients at risk of persistent headaches, papilledema, and progressive vision loss. Existing treatments rely on invasive procedures or off‑label injectable drugs that require frequent dosing and cause systemic side effects.
Solution
NBO Pharma is developing an intranasal formulation of octreotide acetate to deliver the drug directly to central nervous system receptors implicated in IIH. The nasal route enables lower doses, reduces injection frequency, and minimizes systemic exposure while maintaining efficacy in lowering intracranial pressure. Preclinical toxicology studies in rodents and canines have demonstrated safety and CNS exposure, and a Phase 1 trial in healthy volunteers established pharmacokinetics, pharmacodynamics, and tolerability. Ongoing clinical development plans a Phase 2a open‑label trial in IIH patients to assess safety and efficacy, leveraging a 505(b)(2) regulatory pathway and orphan‑drug designations in the US and EU.
Target Audience
Primary customers are neuro‑ophthalmologists, ophthalmologists, neurologists, and headache specialists who treat IIH patients.
Features
- Intranasal delivery platform that provides direct CNS access for octreotide, reducing the need for injectable administration
- Formulation optimized for lower drug amounts while achieving therapeutic intracranial pressure reduction
- Completed GLP toxicology studies in rats and canines confirming safety of repeated intranasal dosing
- Phase 1 PK/PD study in 20 healthy subjects demonstrating dose‑dependent CNS and plasma exposure with favorable safety profile
- Orphan‑drug designation and 505(b)(2) regulatory strategy to accelerate market entry
- Patent portfolio covering formulation, delivery method, and manufacturing processes in the US, EU, and Australia