Leadership Addition
Dr. Hendrik Scholl, a leading expert in Stargardt disease and age-related macular degeneration, joined as Chief Medical Officer, bringing significant expertise to the clinical program.
Progress in Clinical Trials
The DRAGON 2 trial for Stargardt's disease is fully enrolled with 104 patients, and the PHOENIX trial for geographic atrophy has enrolled over 280 subjects. There is also progress in dosing and enrollment in the U.S., U.K., and Japan.
Significant Clinical Trial Results
The Phase II study with Tinlarebant showed a significant reduction in the growth rate of Stargardt disease lesions and stabilization of visual acuity loss.
Financial Position
R&D expenses decreased to $6.8 million from $8.7 million year-over-year. The company maintains a strong balance sheet with $109 million in cash, providing about 4 years of cash runway.
Regulatory Designations
Tinlarebant has received rare pediatric disease and Fast Track designations in the U.S., and Orphan Drug designation in the U.S., EU, and Japan, highlighting significant unmet medical needs.