The following transcript has been edited for clarity.
Diana V. Do, MD: Hi, today I'm joined by Quan Dong Nguyen, MD, MSc, from Stanford. Quan, you presented some exciting data about an investigational therapy for Stargardt disease, tinlarebant. Tell us the key take-home points.
Quan Dong Nguyen, MD, MSc: Well, Diana, this is quite exciting because the phase 3 DRAGON study looking at tinlarebant (LBS-008; Belite Bio) for adolescent Stargardt disease has shown a significantly positive outcome, showing that tinlarebant, taken as an oral agent at 5 mg daily, has been able to slow down the progression of the lesions significantly compared to placebo in all different aspects, in both the study eye and the fellow eye, using different outcome measures. Overall, there were no associated systemic side effects with it and [adverse visual phenomena were] very minimal. Patients tolerated it very well and were able to do quite well in the study.
The exciting part is we just learned that, as of yesterday [June 12], the manufacturer Belite Bio has filed an application to the US Food and Drug Administration for approval of the drug. So hopefully, very soon we will have the first treatment for patients with Stargardt disease.
Dr. Do: Thank you very much for sharing this latest update on this very innovative therapy that may help our patients.
Dr. Quan: Thank you very much. RP







