Drug Pipeline Series: Phase I, Oct 31- Nov 6, 2006

TARGEGEN INITIATES STUDY OF TOPICAL AMD DRUG

TargeGen has begun a single-site Phase I clinical trial involving TG100801, a small-molecule, topically applied, multitargeted kinase inhibitor for the treatment of age-related macular degeneration (AMD) and other debilitating diseases of the eye. The trial, involving approximately 45 subjects, is expected to be completed by the end of the first quarter of 2007. The company hopes to initiate Phase II studies with TG100801 in mid-2007.
TG100801, applied daily in eyedrop form, is designed to suppress disease-related edema, angiogenesis and inflammation. Edema, angiogenesis and inflammation are pathological hallmarks of macular degeneration, diabetic macular edema and proliferative diabetic retinopathy. Currently approved therapies for macular degeneration require repeated injection into the eye and typically act on only VEGF-mediated retinal leakage.
TargeGen believes that TG100801 may offer equal or superior efficacy to injectable agents, while offering patients the greater convenience and potential safety advantages of noninvasive delivery. In published preclinical studies conducted by outside retinal disease experts, TG100801 demonstrated the ability to reduce VEGF-mediated retinal leakage, angiogenesis and inflammation after topical instillation. TG100801 is the second internally discovered TargeGen compound to enter clinical trials.

MACUSIGHT INITIATES WET AMD STUDY

MacuSight has begun a Phase I study of its lead product candidate in patients with wet age-related macular degeneration (AMD). This trial, which will enroll a total of 30 patients, is designed to evaluate the safety and tolerability of MacuSight's proprietary formulation of sirolimus (rapamycin) when administered in various doses through two different types of ocular injections.
Investigators for this randomized, open-label, dose-escalation study will treat patients with a single subconjunctival (just under the lining layer over the white of the eye) or intravitreal (into the back of the eye) injection of MacuSight's sirolimus formulation. The trial will include six treatment arms with patients receiving one of three doses of sirolimus via subconjunctival injection or one of three doses of sirolimus via intravitreal injection. Each administration of sirolimus will provide the patient with exposure to the compound for up to approximately three months.
Sirolimus, originally known as rapamycin, is a highly potent, broad-acting compound that has demonstrated the ability to combat disease through multiple mechanisms of action including immunosuppressive, anti-angiogenic, anti-migratory, anti-proliferative, anti-fibrotic and anti-permeability activity. Based on the versatility associated with these multiple mechanisms of action, MacuSight believes that its sirolimus product may serve as a highly efficacious therapeutic for a wide range of ocular diseases and conditions, including the treatment and prevention of wet AMD, as well as the treatment of diabetic macular edema (DME). The company recently initiated a similar Phase I trial in patients with DME.