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What are the results of your clinical trials?
Now that the data is out, we’re preparing the NDA documents, and it’s a lot of work. This is about eight years of scientific data—everything from preclinical animal work, to toxicology, to clinical studies. In a nutshell, the entire coming year will basically be our final submissions for regulatory approval.
Right now, there’s no treatment for Stargardt because there’s nothing to stop or protect retinal cells from dying. Preserving retinal cells is the key to treating retinal degenerative disease, because those cells are responsible for vision.
Tinlarebant is the first-ever treatment to show preservation of retinal cells by nearly 40% compared to placebo, in a Phase 3 trial in patients aged 12 to 20. That age group is in the early stages of the disease, and their condition tends to be fast-progressing.
Up until now, any treatment for retinal disease has been a needle directly into the back of the eye. Everything is localized, so the drug doesn’t go systemic. Really, you just monitor local side effects.
But with an oral drug, it’s the opposite, because it’s systemic. For a lifelong treatment, the safety bar is about as high as it gets. You have to monitor the whole body: cardiac, kidney, everything. To even get to Phase 2 or Phase 3, we had to run all sorts of safety tests.
Tinlarebant showed about 36% slowing of lesion growth, and the difference between treatment and placebo showed a statistical significance with a p-value of lower than 0.0001. That’s a first for this disease, and that p-value is about as low as you can get, so it’s very statistically significant. Tinlarebant was even able to demonstrate a robust and consistent treatment effect in both eyes.
Where will Belite conduct its next clinical research?
Patients usually start to feel like they’re losing eyesight pretty early—often in their teenage years.
Our mission has always been to find a treatment for Stargardt and preserve vision, getting the drug approved as soon as possible and getting it out to patients worldwide. We’ve been approached by multinational pharma companies. We also have investors who want us to commercialize Tinlarebant ourselves.
The good thing is, for this disease, there aren’t that many experts. They’re mostly concentrated in large teaching hospitals. These retinal specialists are already on board and most of them have participated in our studies. I’d say that we know 80% of them already. Hendrik Scholl, Belite’s CMO, knows basically everyone in the space.
Really, though, it all comes down to speed, and how quickly we can get the drug to patients who need it.
Understanding Stargardt has been a big game changer. We’ll refine our second study on geographic atrophy, a much more prevalent macular degeneration, after interim data. Honestly, we recruited 530 patients really quickly. That probably means uptake of intravitreal anti-complement injections hasn’t been good, and patients are looking for oral options.
If we’re successful, this could revolutionize degenerative eye disease treatment, because taking a tablet like Tinlarebant is noninvasive. Even if the effect is, say, around 15%, there are basically no competitors in oral treatment.
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Dr. Adil Ali
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