Neurotrophic Keratopathy (NK)

Neurotrophic keratopathy (NK) is a progressive debilitating condition that can lead to corneal scarring and vision loss. The primary cause is loss of the sensory nerve supply of the cornea. The innervation adds trophic support to the cornea and is integral to the blink reflex necessary to maintain a protective tear film over the corneal surface. The most common causes of loss of the corneal sensory innervation are viral infections, herpes simplex virus I and herpes zoster virus, diabetes, and trauma and surgery.

The prevalence is commonly reported at 3.5-5/10,000 and approximately 30% of patients with more severe disease are at risk of irreversible scarring of the cornea. The incidence of NK is increasing and is likely underdiagnosed.

While the long-term goal is to restore innervation there is a need to accelerate corneal healing and dampen inflammation as a driver of progressive disease. AP-001 uniquely addresses all these therapeutic needs. Through its broad mechanism of action AP-001 is expected to more rapidly and to a higher degree restore corneal integrity than current therapy, while offering a more patient friendly treatment alternative.

Anida plans to start a first clinical study in NK in 2024 with clinical proof-of-concept expected in 2025.

Retinopathy of Prematurity (ROP)

Retinopathy of prematurity is an eye disorder with high risk of vision loss and blindness affecting premature infants commonly born before gestational week 27 or with a birthweight below 1,500 grams. In the US 30,000 infants/year are at risk and of these 12,000-15,000 are affected by ROP to some degree. The statistic is similar in Europe. In low- and middle-income countries the incidence is higher and globally 300,000 infants are at risk every year.

ROP is increasing globally and in the US the incidence doubled from 2003 to 2019 with now more than 8% of premature infants with a birth weight below 1,500 grams affected, reflecting the improved survival of those with very low birthweights.

With an inverse relationship between age at birth and incidence and severity of ROP, there is an increasing need to develop non-invasive early prophylactic treatments. Current invasive treatment, laser photo-coagulation, or intravitreal injection of an anti-VEGF agent is a last resort to prevent retina loss due to late developing pathological neovascularization.

Inhibition of undesired vessel growth does not solve other major outcomes of ROP including retina neurodegeneration and other causes of impaired vision later developing and also affecting those with disease subthreshold for current standard of care.
Anida’s proposal is an early intervention non-invasive eye drop for infants at risk of developing the condition.

The goal of the program is to create a new standard of care for the treatment of ROP with an early safe treatment reducing the need for late invasive intervention.

A first clinical study is planned for 2025 with completion during 2026.

Future therapeutic opportunities

The activity profile of AP-001 indicates its potential use in other major ocular conditions. The combination of neuroprotection with control of inflammation and oxidative stress indicates a major potential for AP-001 in the treatment of diabetic retinopathy, both non-proliferative as well as proliferative with macular edema.

Development Status

AP-001 therapeutic potential is demonstrated in six established ocular translational models. Eye drop administration for both corneal and retinal indications is demonstrated in large eye species and with confirmed excellent tolerability.

AP-001 efficacy is further demonstrated in six translational CNS models and in hearing loss. 

Regulatory status

The company has orphan drug status for ROP in the US and EU and rare pediatric disease designation for ROP in the US. An IND is planned for 2024 for ROP, with phase I adult clinical trials planned for 2024, as a lead into ROP trials in infants.

Anida has in PIND obtained FDA consent for path into and through first clinical studies in man.