Calpain Therapeutics is developing a library of proprietary compounds to treat ophthalmic diseases representing major causes of blindness and unmet medical needs globally.

Ophthalmic Indications


According to the World health Organization (2010), cataract is the leading cause of reversible blindness, representing 51% of global blindness or approximately 20 million people. The National Institute of Health predicts that by age 80 more than half the people in the United States will have a cataract or have had cataract surgery.

The number of people in the world suffering from cataract is predicted to increase due to growing and ageing populations. While cataract can be treated by surgery, the current surgical capacity on a global scale is insufficient to meet the anticipated future demand.

One of Calpain Therapeutics' compounds is a first-in-class pre-clinical pharmaceutical compound in development for the treatment of cataract.

Delaying cataract onset or progression may have a significant impact on the quality of life of early- and mid-stage cataract sufferers and may reduce the amount of surgeries required with potentially significant savings to community and Government medical programmes worldwide.


The World Health Organisation estimates that Glaucoma is responsible for 12% of all blindness or 4.5 million people globally which may grow to 80 million people by 2020. Glaucoma is a group of age and genetically-related eye diseases which cause restricted vision which progressively worsens leading to irreversible blindness. The exact cause of glaucoma is unknown, but elevated intraocular pressure (IOP) is thought to be the major cause of the optic nerve damage that typifies the disease. 

The primary treatments for Glaucoma are medication and/or surgery that lower intraocular pressure. Unfortunately while these treatments do treat the cause of optic neuropathy they do not work for all patients, especially those with normal intraocular pressure whose symptoms can continue to worsen.  

We are also developing first-in-class pre-clinical drug candidates to treat Glaucoma by conferring a neuroprotective effect on the retinal ganglion cells (RGCs) to reduce loss of vision associated with death of these photoreceptors.  

Conferring protection on the retinal ganglion cells has the potential to have a significant impact on the quality of life of early- and mid-stage glaucoma sufferers, with potentially significant savings to community and Government medical programmes worldwide.

Retinal Diseases

Retinal Vein Occlusion (RVO, 16.4 million), Diabetic Macular Edema (DME, 21 million), Diabetic Retinopathy (DR, 93 million), Age-related Macular Degeneration (AMD, 20-25 million), and certain orphan diseases, are all retinal- and/or optic-nerve based diseases where the aetiology is potentially treatable with one of Calpain Therapeutics’ assets.

Glaucoma and IntraOcular Pressure (IOP) elevation pharmaceutical sales totalled US$4.4 billion in 2017 and are projected to grow to US$5.3 billion in 2022. The blood vessel pathology in RVO, DME, DR and AMD resulting from increased production of the protein vascular endothelial growth factor (VEGF) is currently treated by intravitreal (IVT) injections of anti-VEGF therapy. In 2017, IVT anti-VEGF sales reached US$10.3 billion and are forecast to rise to US$13.9 billion by 2022.

Despite the increasing pharmaceutical sales forecasts, all of the above disease examples have a neurodegenerative aspect that is not treated by IOP-lowering, anti-VEGF or other surgical or pharmacological means. Neurodegeneration leads to irreversible blindness through degeneration of RGCs as well as all other cellular members of the neural retina, including photoreceptors. This loss of the key elements for transmitting visual signals from the retina to the brain represents a significant unmet medical need.

Importantly, the Calpain protein is known to be activated under ischaemia, hypoxia and other trauma conditions in the eye and plays a central role in the degeneration of RGCs, axons and photoreceptors and in retinal pigment epithelium (RPE) dysfunction.