Belite Bio, Inc American Depositary Shares Wells Fargo 21st Annual Healthcare Conference

Sure. First of all, thank you for having us. We are Belite Bio. What we have is an oral once-a-day tablet called tinlarebant. We have two indications now. One is Stargardt disease and one is geographic atrophy. Stargardt disease is a gene mutation disease that causes, in almost all cases, legally blind. We have completed our pivotal Phase III study last year, and we have submitted for NDA in the U.S. and Japan. The FDA has confirmed the acceptance of the NDA with priority review, with the PDUFA date being February 12th next year. On GA, we have completed enrollment of the study. It is also a Phase III study globally, and we are just doing the execution right now.

You want to say it?

No, you. Well, I should first, apparently we're very excited and appreciate with the FDA's priority review.

I think FDA has been showing a lot of support throughout the process. We do have a lot of patient have been waiting for this drug for a long time, given there is no treatment. We really look forward to continue to work with FDA and provide the drug to the patient as soon as we can.

Maybe an extra comment. It just underscores the significant unmet medical need that we have for this rare, but not so rare, severe in pediatric disease.

We have had very constructive discussions with the FDA about the design of the DRAGON trial, how the DRAGON trial was conducted, when we submitted our interim analysis to the FDA that led to breakthrough designation. The agency is very aware about the design of the trial, what was the interim result that led to breakthrough designation, and now with our submitted package, including a very comprehensive document that we call confirmatory evidence, because we seek approval based on one trial, and that, again, was pre-discussed with the agency. It is important to underscore how, and I want to praise the FDA here, how educated the agency is about the primary endpoint that we have been using in both trials. It is just a different nomenclature. In geographic atrophy, it is simply called geographic atrophy. It is not so sophisticated.

It is measured on autofluorescence images, and it is again, compared to the optic nerve head in terms of the blackness level. In Stargardt, we call it definitely decreased autofluorescence, meaning very black, again, compared to the optic nerve head. So it is essentially the same outcome measure that is being used for both indications. The FDA already expressed 10 years ago in publicly available papers that if you preserve photoreceptors and you can show that on imaging, that is a good thing. If you cannot preserve them, it is a bad thing. So the FDA is willing and eager to accept efficacy measured precisely on these imaging modalities that are part of standard clinical care and clearly indicate preservation of photoreceptors that eventually, in the long term, correlates with visual function or dysfunction in that case.

Therefore, because the agency clearly understands our primary endpoint and perceives this as an approvable endpoint, we do not anticipate necessarily an advisory board, because typically at an advisory board, it would be about the primary endpoint, and we do not believe that there are so many questions about what is the endpoint, what does the endpoint mean, how it is being measured, what is the reproducibility and so forth.

Yeah. This is We prediscuss with the agency. The agency understands this is a genetically caused disease. It is caused by dysfunction of ABCA4. The question goes to the generalizability of the data, and we believe that given that everybody affected by Stargardt disease is that because there are mutations in the gene, that the patient population 12 to 20 years old can be generalized to all patients out there, regardless of the age of onset. The age of onset shows variability. A latest survey of the literature shows that it can be as early as 1 year of age, and it can be as late as 84 years of age. We still believe that the data of the DRAGON trial of patients of the age of 12 to 20 is generalizability to all patients out there. The second question is about disease stage.