NRO-1

Regenerative Therapy for Ophthalmic Diseases

A Novel

Regenerative Treatment

NRO-1 is a topically delivered, nerve regenerative therapeutic that accelerates nerve regeneration and recovery of functional nerves

Corneal Nerve Degeneration

Dry Eye Disease

Patients with dry eye often have corneal nerve degeneration, resulting in decreased corneal sensitivity, decreased lacrimation, and corneal and conjunctival epithelium disorder.

Post-LASIK Treatment

Corneal nerves are severed during LASIK surgery causing decreased corneal sensitivity, dry eye like symptoms and other complications.

Neurotrophic Keratitis

A rare disease of the corneal epithelium resulting from corneal nerve degeneration and potentially leading to a loss of vision

Retinal Ganglion Cell Degeneration

Glaucoma & Optic Neuropathies

Glaucoma and certain optic neuropathies are neurodegenerative diseases characterized by loss of retinal ganglion cells (RGCs) resulting in vision loss and blindness.

NRO-1 MOA

NRO-1 releases glial cell-derived neurotrophic factor (GDNF), which recovers ocular surface function through regenerating corneal nerves and accelerating corneal wound healing directly

MECHANISM OF ACTION

Corneal Sensitivity Recovery

Corneal Nerve Regeneration

Corneal Epithelium Migration

Corneal Wound Healing

PHARMACOKINETICS

NRO-1 permeates to

the back of the eye via

topical application

NRO-1 can reach the target tissues in the back of the eye with efficacious concentrations with BID dosing

OCULAR DISEASES

NRO-1 promotes axon regeneration and corneal wound healing via release of GDNF

NRO-1 recovers ocular surface function through regeneration of corneal nerves

POST SURGICAL

Topical NRO-1 facilitates axonal regeneration

and recovery of corneal

sensitivity after flap

surgery in rabbits

NRO-1 may decrease complications in patients

after LASIK surgery

REGENERATION

NRO-1 induces neurite elongation / regeneration in rat trigeminal ganglion neurons

NRO-1 can regenerate neurons, providing a disease modifying treatment for degenerative diseases

Based on the results from the Phase 1 SAD and MAD clinical studies, a Phase 2 clinical study of NRO-1 in dry eye disease was initiated in the January 2020.

NRO-1 has shown efficacy in a range of nonclinical studies

Nerve damage is linked to a range of ophthalmic diseases, including dry eye disease, creating a high probability of success for human studies

Phase 2

Dry Eye Disease:

STUDY DETAILS

Phase 1

Clinical Study:

Well tolerated across the entire dose range, all patients completed dosing in both the SAD and MAD studies.

STUDY DETAILS

Study Overview:

Multicenter, randomized, double-asked, vehicle-controlled clinical trial that will evaluate the safety and efficacy of NRO-1in patients with dry eyed disease

120 patients with moderate-to-severe drye disease, including post-surgical patients.

2 dosages of NRO-1 compared to

vehicle (placebo)

Study initiated in Janary 2020, with results available in 2H 2020

Key Results:

Similar safety and tolerability to placebo

No serious or severe adverse events

No clinical chemistry, hematology or ECG safety concerns

Safety profile consistent with preclinical data and supportive of continuing to Phase 2