NRO-1 is a regenerative topical treatment being developed by Neuroptika for degenerative eye diseases such as neurotrophic keratitis.

The investigational therapy has the advantage of easy storage, topical delivery, and reduced pain and irritation compared to Oxervate (cenegermin), which is currently approved by the U.S. Food and Drug Administration for neurotrophic keratitis.

How does NRO-1 work?

In neurotrophic keratitis, nerve cells that serve the cornea of the eye fail to function properly, resulting in the breakdown of the outer corneal layer. In more severe cases, the inner layer of the cornea may also break down, a phenomenon known as stromal melting, leading to the scarring of the cornea and to vision loss.

Glial cell line-derived neurotrophic factor (GDNF) is a protein that promotes the growth of elongated structures from nerve cells called neurites (which may be an axon or a dendrite that is projected out from the nerve cell). Neuronal growth is controlled by a class of proteins called semaphorins, such as semaphorin 3A (Sema 3A), that guide the growth of neurites to the target organs and repeal their growth if needed.

NRO-1 stimulates the release of GDNF, thereby enhancing neurite elongation while inhibiting the activity of Sema 3A. It is hoped that this will promote the regeneration of nerve cells in the cornea of the eye and restore its function.

NRO-1 in clinical trials

A Phase 1, single- and multiple-ascending dose clinical trial completed in April 2019 showed that NRO-1 was well-tolerated across the dose range tested in 36 healthy volunteers. The participants reported no serious or adverse events, and there were no safety concerns.

In January 2020, Neuroptika initiated a Phase 2 trial to further test the treatment in patients with dry eye disease. The company also plans to launch a Phase 2 study of NRO-1 in patients with neurotrophic keratitis in 2020.

Other information

NRO-1 is also being investigated for diseases such as dry eye disease, glaucoma, and Leber’s Hereditary Optic Neuropathy.

The treatment may decrease complications after surgeries such as laser-assisted in situ keratomileuses or LASIK eye surgery.


Last updated: Jan. 27, 2020


Neurotrophic Keratitis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.