Status

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