Development

Pipeline

Remofuscin is being developed for two indications: Stargardt disease and dry age-related macular degeneration. In both indications Remofuscin is entering Phase 2. Stargardt disease is a rare indication with a high medical need.

Stargardt Disease

Disease

Stargardt disease patients accumulate lipofuscin in their retina. Disease onset may already occur in infants or in young adults. Progressing over the years, entire areas of the retina degenerate and this process eventually leads to legal blindness with only peripheral vision remaining. Currently, there is no approved treatment for Stargardt patients.

Stage

Together with its partners, Katairo is currently conducting the STARTT study, a phase 2 'proof-of-concept' clinical study.

STARTT study

The endpoints of this study will show if the effects observed in cells and animals can be confirmed in a human study. This includes first and foremost the removal of lipofuscin. Efficacy and safety endpoints will beĀ  monitored.

Patients may find more detailed information about the STARTT study in the following documents, each for the respective trial site:

2019 May 20 Patient Information STARTT study Southampton

2019 May 20 PatientInformation STARTT study Tuebingen

2019 June 06 Patientinformation STARTT study Nijmegen and Leiden

Dry AMD

Disease

The dry form of age-related macular degeneration is characterized by excessive accumulation of lipofuscin in the retina of elderly patients. Although onset of the disease may occur as early as age 50, most patients with visual problems are over the age of 70. The progression of he disease is associated with growing central retinal atrophy - commonly termed 'geographic atrophy'. This results in vision loss and blindness by legal standards.

Stage

The development of Remofuscin for the treatment of dry AMD is entering Phase 2. Katairo is actively seeking funding for a first clinical study for this indication.