LightBio at H.C. Wainwright Conference: Telariband’s Promising Path

On Tuesday, 20 May 2025, LightBio (NASDAQ:BLTE) presented at the H.C. Wainwright 3rd Annual BioConnect Investor Conference, sharing insights into their lead candidate, telariband. The conference highlighted both the challenges and opportunities for telariband, which is aimed at treating Stargardt disease and geographic atrophy. The FDA has granted breakthrough therapy designation, signaling positive interim results from ongoing trials.

Key Takeaways

• LightBio’s telariband received breakthrough therapy designation from the FDA.
• The company has $157 million available to complete ongoing clinical trials.
• The DRAGON trial is expected to conclude by the end of the year.
• Telariband’s unique oral administration differentiates it from existing treatments.
• Japan is eager for telariband’s market authorization due to the SAKIGAKE designation.

Financial Status

LightBio reported a financial reserve of $157 million, which is sufficient to complete all ongoing clinical trials. This ensures the company can continue its research without immediate financial constraints.

Operational Updates

• Dr. Hendrik Shaw, Chief Medical Officer, emphasized the unique mechanism of telariband, which reduces vitamin A levels and bisretinoid accumulation in the eye.
• LightBio’s DRAGON trial is on track, with the final patient visit scheduled for September 30th, 2025.
• The DRAGON-two trial is progressing, with 16 out of 60 patients enrolled, including 8 in Japan.
• Telariband’s oral delivery method avoids the risks associated with subretinal delivery used in gene therapies.

Future Outlook

Dr. Shaw expressed optimism about telariband’s market potential, particularly in the U.S., where over 50,000 patients suffer from Stargardt disease. The company is also in discussions with regulatory agencies worldwide, including the FDA, Chinese FDA, PMDA, MHRA, and EMA, to facilitate global access.

Q&A Highlights

• Analysts inquired about LightBio’s financial position, confirming that the company has ample resources to complete phase three trials.
• The potential launch strategy was discussed, with the U.S. likely to be the first market, although no final decision was made.
• Dr. Shaw reiterated the urgency of bringing telariband to market to help patients as soon as possible.

Conclusion

For more detailed information, please refer to the full conference call transcript below.

Full transcript - H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025:

Operator: Good afternoon. Welcome to the HC Wainwright CERT Bioconect Investor Conference in Nasdaq. For this session, we will have a fireside chat with doctor Hendrik Shaw, chief medical officer of the LightBio. Thank you for joining us.

Hendrik Shaw, Chief Medical Officer, LightBio: Thank you for having me.

Operator: Doctor Shah, you joined Billai Bio last September. Is that right?

Hendrik Shaw, Chief Medical Officer, LightBio: That’s correct. Could

Operator: you tell us what is unique about Billai Bio that made you join the company?

Hendrik Shaw, Chief Medical Officer, LightBio: Keep in mind that I have been seeing patients affected by Stargardt disease in AMD for more than two decades in Germany, The United Kingdom. I was professor at the Wilmer Institute for more than six years. I was the principal investigator for the largest natural history study ever conducted for Stalgar disease, the PROXA study in The United States, and then continued to see patients as a Chairman in Basel. But I always wanted to be connected with therapy for these patients because these patients inevitably go blind. And I have been collaborating with Belite for more than half a decade in various capacities, and this obviously is a major opportunity to get as close as possible to the first therapy ever for Stargardt disease.

And I took that opportunity and I’m very happy with it.

Operator: Could you tell us well, your lead candidate is called telarabant. Could you tell us, how telarabant’s mechanism of action is differentiated from other competitors?

Hendrik Shaw, Chief Medical Officer, LightBio: Happy to. Maybe first of all, it’s an oral compound. It’s a chemical compound. It addresses the most important part of the pathophysiology of Stagler disease and late dry age related macular degeneration. I think there’s a lot of activity in the field of gene therapy.

I have been very active in that field as well. But specifically for Stargardt disease, there’s a problem, namely that the ABCA4 gene is very large, exceeds the packing capacity of the typical vectors that are being used for gene therapy, namely AAV. And there are more than 2,800 variants in the gene. Plus, when you develop gene therapy for a condition that affects the macula, the only way currently to efficiently transfuse photoreceptors is subretinal delivery. Surgically, that means you have to detach the macula in order to have a treatment effect for macular diseases and this is absolutely counterproductive.

I speak as a vitreoretinal surgeon and always when you detach the macular, you deteriorate the situation and you harm photoreceptors. When it comes to the mechanism of action of tenlariband, it addresses retinol or vitamin A availability in photoreceptors and the RPE. And we need vitamin A in photoreceptors. But if ABCA four is mutated, then we get an accumulation of of ultransretinal, which is part of the visual cycle, and that is toxic itself, plus it forms with other molecules so called bisretinoins that are very toxic. And tinlaraband efficiently reduces the amount of vitamin A in photoreceptors but not too much and efficiently reduces the accumulation of bis regiments at the level of the retinal pigment epithelium and the photoreceptors and this with an excellent safety profile.

And this is unique. The only other compound that may be similar if you will is deuterated vitamin A also called drilled uritinal where by modifying vitamin A you reduce downstream the baseretinoin accumulation of photoreceptors, but you really give very high amounts of vitamin A to the human body to photoreceptors. If you do the math, it equals seventy thousand international units of vitamin A. And keep in mind, the drug is intended to be used for the rest of the life of the patient and giving such high amounts of vitamin A, in my opinion, poses a big risk for patients.

Operator: Thank you. So are there a good number of Stargardt disease patients available in The U. S. That would present an attractive market opportunity for the company?

Hendrik Shaw, Chief Medical Officer, LightBio: The answer is a clear yes. So in the past, it has been difficult to estimate the prevalence of relatively rare diseases such as Stargardt disease because population based studies are not sufficient to allow us to really estimate the prevalence of Stargardt disease. Many papers still cite an opinion from publication of nineteen eighty eight where a Dutch ophthalmologist was asked how prevalent is Stargardt disease and the answer was, Oh, it’s more prevalent than a retinoblastoma but less prevalent than retinitis pigmentosa. Therefore, I estimate the prevalence to be one in eight thousand to one in ten thousand, which is kind of funny that this is still being cited. But there is an opportunity in Stargardt disease.

It’s an inherited disease due to biallelic mutations in ABCA4. And there is no non penetrance, meaning if you carry two mutations in both alleles, you will be affected. And because of that, you can look into genetic databases of the world populations and you can calculate the number of affected individuals. And if you do that, you find that in populations of European descent, the prevalence is one in six thousand five hundred. And if you look into the prevalence in East Asians, you find that the prevalence is pretty much exactly one in eleven to one in twelve thousand.

So it’s actually a pretty high prevalence. And when you do the math and calculate the number of affected patients in The U. S, the number is taking into account so called hypomorphic mutations, then you find the lowest number would be forty three and the highest number fifty nine thousand patients in The U. S. Long story short, more than fifty thousand on average.

And I think this is a big market opportunity.

Operator: Thank you. So I understand the telerobrane is currently being evaluated in two Phase III trials named Dragon and Dragon II trials. And the first Dragon trial is now on track to be completed in the fourth quarter of this year. Right? So can you tell us your expectations for the data readout?

What will be considered positive results?

Hendrik Shaw, Chief Medical Officer, LightBio: Yes. Thank you very much for the question. This is a very important moment for myself because actually at 11:37 today, we received a letter from the FDA of the designation of breakthrough for telariband based on the interim data that were submitted to the agency a couple of weeks ago. So the interim analysis was preplanned and about 75% of the data were available and were presented to the DSMB, an independent data safety monitoring board of the DRAGON trial with the task to provide feedback on safety and efficacy insofar, the trial would fall into a so called promising zone, which would allow to add 30 more subjects in order to boost conditional power. If no subjects would be added, that would mean either futility or an efficacy signal beyond the so called promising zone.

And since the DSMB provided an additional recommendation, namely submit the data for further regulatory review. The only conclusion that can be drawn, in my opinion, is that there must have been a significant efficacy signal that prompted the DSMB to make this recommendation. And we followed the recommendation of the DSMB and have initiated interactions with various regulatory agencies, the FDA, the Chinese FDA, the PMDA, the MHRA, the EMA. And we are currently in discussions with all these agencies with one example that I just mentioned, namely breakthrough designation that was granted actually this late morning today. So therefore, I have high expectations of the final study data that we would have available by the end of this year.

The last patient, last visit is September 30. And I believe with the image analysis by the central reading center, then data cleaning and QC, we should have the data available by the end of the year.

Operator: Well, congratulations on getting the BRICSID therapy designation. Does that mean the pathway to approval could be accelerated? I would hope so. We have feedback from the FDA. We had that before that

Hendrik Shaw, Chief Medical Officer, LightBio: if one trial is sufficient would be a review issue. I would believe it’s the same with interim data given the fact that 75% of the data is already available, was available at the time of the interim analysis and the combination of the efficacy and the safety of tillaraband. Last, the fact that our secondary endpoints could represent so called surrogate endpoints for the primary endpoint would mean that we should be in a strong position to at least discuss with the agency about accelerated approval, but it remains to be seen.

Operator: And meanwhile, the Dragon two trial is still advancing as planned. Right? Can you talk about the status of the Dragon two? And is the is Dragon two is having the exactly same trial design as Dragon one?

Hendrik Shaw, Chief Medical Officer, LightBio: So Dragon two is based on an opportunistic approach due to the PMDA in Japan providing the SAKIGAKE designation with the requirement that Belite Bio would run a Phase Ib clinical trial looking into the pharmacokinetics and pharmacodynamics in Japanese patients, plus enrolling at least 10 Japanese patients into an interventional trial. And that prompted the DRAGON-two trial, although EMA, PMDA and so forth do not require a second trial explicitly. We took that opportunity to launch the trial in Japan and invited centers in The United States and United Kingdom to also participate in the DRAGON-two trial. So if we ever needed a second trial, we would have one. It’s currently running.

The target enrollment is 60 patients, currently 16 are enrolled. Eight patients in Japan have been enrolled, which means we need another two, and we anticipate that to be completed by summer or so at least. And the trial design is almost identical to the DRAGON-one trial. There are two differences. One, it’s a one:one randomization between tinlaraband and placebo, while in DRAGON-one, we have a two:one randomization.

And the second difference is that we somewhat lowered the lower threshold of visual acuity to from two thousand two hundred to 2,400 to allow more patients to be enrolled into the DRAGON-two trial. Otherwise, same intervention, five milligrams per day daily, same primary outcome measures, same secondary outcome measures. So in other words, it’s almost identical.

Operator: Okay. So does that mean later this year, you will have data from the 10 Japanese patients from Dragon two? And combined with data from Dragon, you will be able to submit to PMDA in Japan for potential approval?

Hendrik Shaw, Chief Medical Officer, LightBio: That is correct. The PMDA is I mean, I I think the Sakigake designation speaks for itself. It’s keen on having tillariband being market authorized in in Japan. It remains to be seen if if Japan indeed would be the first country in the world. If if you ask me, I would still bet that it will be The United States, but but but but, clearly, Japan is very welcoming towards an approval for telariband in Japan.

Operator: I guess it remains to be seen whether Beliba will launch the truck in The U. S. First or in ex U. S. Territories, right, given the current most favored nation pricing policy?

Hendrik Shaw, Chief Medical Officer, LightBio: Yes. This is an excellent, excellent question. I think it was a very good plenary session this morning from eight to nine, but this was also discussed. And I like the conclusion that we cannot really conclude anything right now how to position ourselves. I can speak as a physician.

I’ve seen these patients for more than two decades, and my big motivation is to get the drug to the patients as soon as possible. And I think this is path we are currently following, but you’re absolutely right. We will observe closely about future uncertainties being imposed onto market, the world by the Trump administration, and we’ll we’ll try to to find the best path forward.

Operator: Got it. Telarabine is also being evaluated in a phase three PHOENIX trial for geographic atrophy. Do you expect the drug to work equally well in GA as in Stargardt disease?

Hendrik Shaw, Chief Medical Officer, LightBio: We don’t know yet. In Stargardt, the situation is clear, if you will. There’s this gene, APCO4, that is very well characterized. We know exactly what it does. Geographic atrophy is a different beast.

It’s a so called complex disease. There are various pathways that contribute to GA. We know that the photoreceptors and RPE are sick, very similar to Stagler disease. When ABCA4 gene was discovered, it was claimed at the time that this is an AMD gene. I believe that the two diseases are different, but they are so similar.

Plus, it has been established that bisoretinoids play a role in late dry AMD that I feel that tinlaraband must be efficacious in geographic atrophy. Since there are other pathways in late dry AMD that may also contribute, you could argue that tenlarabine may be less efficacious in geographic atrophy. But there is one factor that would speak for the same efficacy namely the pace of geographic atrophy that is typically two to three times faster or larger in geographic atrophy, which means that intervention targeting successfully one of the important pathways because of the relatively fast pace could have a similar treatment effect as observed in Stargardt disease.

Operator: Are there any close competitors in the GA space that you think investors should be aware of, particularly those drugs that are noninvasive like teleripen?

Hendrik Shaw, Chief Medical Officer, LightBio: Yeah. That’s a good addition that that that they are that you that you We know I mean, I’m I’m practicing in Switzerland, in Europe. There is no drug available at all. In The United States, the situation is different. We have two injectables that target the complement system.

I’m proud to say that my group in 02/2008 was the first to show that there is systemic complement activation in AMD. So those drugs make sense. But when you look at the treatment effect, least in the published literature, in the GATHER2 trial, the efficacy signal was fourteen percent reduction after twelve months. And when we look at ciforvir, there were two trials. One actually did not meet its primary endpoint with an efficacy signal of 12% in twelve months.

The other one, the OAX trial met the primary endpoint with an effect signal of 21% based on monthly injections, right? And I mean, it’s a hard sell because the disease will continue to get worse. And you think about the treatment effect of about, let’s say, fifteen percent of taking those two interventions together, which means eighty five percent of progression remains in this still on a monthly injection basis for many, many years. So that this is a hard sell. But still, there is a treatment available in The United States.

In my opinion, tillariband would be truly transformative because it’s an oral treatment that simply needs to be taken as one pill per day. When we consider other systemic interventions, then there is one compound that could be considered similar, namely deuterated vitamin A. This was tested in the so called SAGA trial that was presented at the last academy meeting last October and was observed to fail its primary endpoint. In our opinion, in our interpretation, there was a trend at least that was shown in this deuterated vitamin A. And we feel this kind of supports this intervention, namely targeting biseretinoins in geographic atrophy, right?

So I would say for AMD patients that was a not so nice piece of information that the trial failed, but at least there was a good piece of information that targeting bisretinoins may be or is a good idea, and that’s exactly what we are doing, with tinlariband. I think that’s the current landscape.

Operator: Does BELIBA currently have sufficient capital to complete the, all the ongoing phase three trials?

Hendrik Shaw, Chief Medical Officer, LightBio: The answer is a clear yes. Currently, we have USD 157,000,000 available, and that would allow to complete all the clinical trials that are currently running.

Operator: Okay. That’s all my questions. Questions for our audience? Nope? All right.

Thank you very much.

Hendrik Shaw, Chief Medical Officer, LightBio: Thank you very much.

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