COMPANY OVERVIEW

Developing Treatments to Untreatable Causes of Blindness

Alkeus Pharmaceuticals is a clinical stage biotechnology company focused on discovering and developing treeatments against serious and untreatable diseases of the eye.

The eye is one of the most complex and precious organs of the body : open or closed, it is constantly at work, making it fragile and vulnerable and resulting in hundreds of ocular diseases. Building on years of groundbreaking research in ophthalmology and on well-understood biochemical processes, Alkeus philosophy is to find creative ways to combine this scientific knowledge to invent novel medicines and to bring them from the laboratories to the bedside.

As of 2012, Alkeus most advanced therapeutic program is based on novel molecules to address dry age-related macular degeneration (dry AMD) and Stargardt disease, the two leading causes of blindness in patients over 50 (AMD) and in children (Stargardt). In laboratory experiments, these molecules have shown promise by preventing the formation and accumulation of toxic ocular piments, which resulted in preserved visual function in animal models of macular degeneration.

Alkeus is currently developing a new oral medicine, designated as ALK-001, that can specifically address Stargardt disease. Stargardt disease is a rare genetic disease that leads to poor recycling of vitamin A and accelerated formation of toxic compounds in the eye. Although, extrapolation of animal data to humans can only be proven in well controlled clinical trials, ALK-001 has showed in mice that it could safely prevent vision loss. Alkeus intends to start a clinical trial in patients with Stargardt disease in 2013.

Because Stargardt disease is a rare disease, it makes it hard to find patients: we therefore invite you to register your name in our confidential mailing list if you have been diagnosed with Stargardt. We will keep you informed of future trials in the coming months.

Among its other interests, Alkeus is investigating methods to treat other rare genetic diseases such as Leber Hereditary Optic Neuropathy (LHON), an inherited disease that leads to sudden loss of vision in young adults as well as in autosomal recessive Retinitis Pigmentosa and in Cone Rod Dystrophy, two forms of serious vision loss that often occur early in life.