Intranasal octreotide is in the clinic, with Phase 1 results expected in Q4 2025; Phase 2 is anticipated to begin in Q1 2026 and be completed in Q4 2026.
Compound
Octreotide is approved for non-CNS conditions, but difficult/inappropriate delivery systems for IIH
Target for Compound
Receptors in the Central Nervous System
IN octreotide clinical trial underway
Phase 1 PK & PD (and initial uptake signal) trial in 20 healthy subjects will determine safety, dose(s), & PK/PD (conducted by NBO Pharma)
Proof-of principle in humans
Multiple positive open-label clinical trials with injectable dosage form (conducted by others)
Favorable toxicology results
Successful 3-month GLP Intranasal tox study in rats; Successful 14-day Maximum Tolerated Dose study in canines; ongoing 3-month GLP intranasal tox study in canines (conducted by NBO Pharma)
CNS Delivery in animals
Multi-dose Intranasal delivery in rats demonstrated both CNS and plasma exposure (conducted by NBO Pharma)
Regulatory pathway
505(b)(2) in the US, Hybrid application in the EU; possibility of Accelerated Approval in the US
Orphan drug designations
Approved US July 2021 &
EU December 2021
Patents
Two filed by NBO in the US in Q1 2021; 2 PCTs in Q1 2022; both accepted in Australia 2025; others pending
Effective market # of patients
>25K patients US, >25K patients Europe
Targets for Commercialization
Neuro-Ophthalmologists, Ophthalmologists, Neurologists, & headache specialists
Competition
No other drugs approved or in development for IIH