Clinical Status

MacuSight is initially developing sirolimus as a potential next-generation therapeutic for the treatment and prevention of wet age-related macular degeneration (wet AMD) and the treatment of diabetic macular edema (DME), a manifestation of diabetic retinopathy. The company has completed two separate Phase 1 clinical trials of its sirolimus product candidate in patients with wet AMD and DME.

Diabetic Macular Edema:

Results from a prospective study of 50 patients with chronic, refractory DME demonstrated that MacuSight's proprietary formulation of sirolimus was safe and well-tolerated in all doses tested with two different routes of administration (subconjunctival injection and intravitreal injection). The study showed no evidence of increased intraocular pressure or inflammatory response to treatment.

In addition to safety and tolerability data, the study provided an initial assessment of sirolimus' biological activity in DME. At 45 and 90 days following treatment, patients receiving the two lowest doses of sirolimus by subconjunctival injection demonstrated mean improvements in visual acuity over baseline using a standard (ETDRS) eye chart. Additionally, this group of patients also experienced anatomical improvements, with mean decreases in foveal thickness that were consistent with the observed functional improvements in visual acuity. Data for these patients at 180 days following treatment suggest that these improvements were maintained, or even enhanced over time, in many patients.

Wet Age-Related Macular Degeneration:

Preliminary results from this prospective study of 30 wet AMD patients demonstrated that MacuSight's proprietary formulation of sirolimus was safe and well-tolerated in all doses tested with two different routes of administration (subconjunctival injection and intravitreal injection). The trial showed no evidence of increased intraocular pressure, inflammatory response to treatment, or indication of progression of cataracts.

Furthermore, the study provided an initial assessment of sirolimus' biological activity in AMD with patients demonstrating improvements in visual acuity despite extended duration of disease prior to entering the study. Additionally, patients also experienced anatomical improvements as demonstrated by reductions in retinal thickness. These preliminary findings demonstrated that sirolimus administered via subconjunctival injections was as effective, if not more so, than sirolimus administered via intravitreal injections.

MacuSight intends to complete its collection and analysis of all data from this trial and present final findings at the Retinal Physician Symposium in Naples, Florida in May 2008.

PHASE 2 PROGRAM

Based on the positive results from its Phase 1 trials, MacuSight is initiating Phase 2 studies in DME and wet AMD, as well as additional ocular diseases and conditions.

• Lead Program
• Mechanisms of Action
• Safety
• Optimized Administration
• Clinical Status
• Intellectual Property

"Based on the positive results from its Phase 1 trials, MacuSight is initiating Phase 2 studies in DME and wet AMD, as well as additional ocular diseases and conditions."