Prepared remarks
Ladies and gentlemen, thank you for joining us. Welcome to the Belite Bio First Quarter 2026 Earnings Call. After today's prepared remarks, we will host a question-and-answer session. If you would like to ask a question, please raise your hand. If you have dialed in to today's call, please press 9 to raise your hand and 6 to unmute. I will now hand the conference over to Julie Fallon. Please go ahead.
Good afternoon, everyone. Thank you for joining us. On the call today are Dr. Tom Lin, Chairman and CEO of Belite Bio; Dr. Hendrik Scholl, Chief Medical Officer; Dr. Nathan L. Mata, Chief Scientific Officer; and Hao-Yuan Chuang, Chief Financial Officer. Before we begin, let me point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially. We encourage you to consult the risk factors discussed in our SEC filings for additional detail. Additionally, today we will be discussing certain non-GAAP financial measures. Reconciliations to the most directly comparable GAAP measures are provided in the press release we issued today. And now I will turn the call over to Dr. Lin. Dr. Lin?
Thank you, Julie. Good afternoon. Thank you for joining our First Quarter 2026 financial results and updates. We have made exciting progress so far this year. We received our Phase 3 clinical study report in Q1 without delay. In April we initiated our NDA rolling submission to the FDA for Stargardt disease. We are on track to complete the submission by the second quarter of this year. As we approach the completion of the rolling submission, we are also preparing for our commercial launch. We have hired all commercial leadership positions and continue to build out our teams in sales, market access, and medical affairs, also building out our commercial infrastructure, as well as engaging with the retinal community to raise awareness of Stargardt disease. We are focused on preparing for a strong launch and look forward to sharing more on our commercial planning in the future. In line with that commitment to bring Tinlarebant to patients around the world, this past quarter we also announced that we have completed enrollment in our Phase 2/3 DRAGON 2 clinical trial evaluating Tinlarebant for Stargardt disease. This trial enrolled 73 adolescent and adult subjects aged 12 to 20 years from Japan, the United States, and the U.K. This is a registration-enabling study to pursue approval in Japan. This is shaping up to be a pivotal year for Belite as we begin our transition to a commercial-stage company. We look forward to providing further updates on our work bringing therapies for retinal degenerative diseases with significant unmet medical needs. I will now turn the presentation over to Hao-Yuan to discuss the financials. Hao-Yuan?
Thank you, Tom. In Q1 2026, our R&D expenses were $15.7 million compared to $9.4 million in Q1 2025. The increase was mainly driven by higher spending on the DRAGON 2 trial, increased API and drug product manufacturing expenses, and higher consultant and professional service fees. On a non-GAAP basis, excluding share-based compensation expenses, R&D expenses in Q1 2026 were $13.8 million compared to $7.4 million in Q1 2025. SG&A expenses in Q1 2026 were $17.0 million compared to $6.1 million in Q1 2025. The increase in SG&A expenses was primarily due to an increase in share-based compensation expenses, professional service fees, wages, and salaries resulting from our team expansion. On a non-GAAP basis, excluding share-based compensation expenses, SG&A expenses in Q1 2026 were $5.7 million compared to $1.5 million in Q1 2025. GAAP net loss for the quarter was $26.9 million compared to $14.3 million in the same period last year. On a non-GAAP basis, excluding share-based compensation expenses, net loss was $13.7 million in Q1 2026 compared to $7.6 million in Q1 2025. Despite the increased investment in R&D and SG&A, our balance sheet remains very strong. Specifically, with proceeds from employee stock option plan and warrant exercises, we ended Q1 with $799 million in cash, cash equivalents, and U.S. Treasury bills, a higher balance than at the end of 2025. This strong cash position gives us ample capital to execute on our goals, including finalizing our NDA application, preparing for commercialization in Stargardt disease, and completing our ongoing clinical trials. With that, I will now turn the call back to the operator for Q&A. Operator?
Questions and answers
We will now begin the question-and-answer session. If you would like to ask a question, please raise your hand now. If you have dialed in to today's call, please press 9 to raise your hand and 6 to unmute. Please standby as we compile the Q&A roster. And your first question comes from the line of Judah Frommer with Morgan Stanley. Your line is open. Please go ahead.
Hi, guys. Thanks for taking the question, and congrats on all the progress here. On DRAGON 2, what confidence do you have based on communication with the FDA that the readout will not be necessary for an approval decision in the U.S.? And then on the flip side of that, if the FDA does imply that they would like to see DRAGON 2 results, what are the chances that it is confirmatory? And how could that play into timelines? Thank you.
Thanks, Judah. That's a great question. We had several meetings with the FDA, including a meeting to discuss the strong positive interim data. It is the FDA's recommendation that we complete the DRAGON 2 study at two years with a possible path to a single-study approval based on the robustness of our data. I should clarify: I think you meant DRAGON 1, not DRAGON 2, when referring to the data applicable to the U.S. filing. We do not believe that the DRAGON 2 data would be applicable to our FDA filing. But even if there is a slight chance that the FDA would request additional data, we could always have DRAGON 2 data available, at least the interim portion, to serve as confirmatory evidence. DRAGON 2 is mostly for Japan regulatory requirements. I hope that answers your question. Regarding building out the commercial infrastructure and how targeted the commercial or field sales team could be given how concentrated the patient population is and where they are seen by centers of excellence, I'll turn that to Hao to address operationally.
We expect to have two teams: one focused on diagnostic promotion to increase disease awareness and genetic testing access, and another focused on promoting the drug. In total, we are thinking about 30 to maybe 40 total team members for that effort. Many retina specialists already have databases of Stargardt disease patients confirmed with genetic testing. We are conducting a lot of surveys right now and expect to provide a market update about what we know and our plans, hopefully in September. That will give a better idea about the whole plan and what is already known in the field. In short, we see that many patients are highly motivated for this treatment and continue to be followed by their physicians. We will focus on the retina specialist community, patient advocacy groups to better understand needs, and also general ophthalmologists and low-vision optometrists as appropriate. Thank you.
And your next question comes from the line of Marc Goodman with Leerink. Your line is open. Please go ahead. A reminder that you may need to unmute locally.
Hey, guys. How are you? Regarding geographic atrophy, can you just talk about how you are thinking about this right now and the timing of the interim and what happens if the GA indication ends up looking really, really strong?
Thanks, Marc. So for GA, right now we are focused on getting FDA approval for Stargardt disease. We are aiming for the interim analysis for GA around the end of the year. Right now, we do not know what the data will look like. If it is strong positive data, that would be a good problem to have, but at this time we do not know the outcome, so we have not given much thought to strategy beyond that. Near the time when we have the data, we will have a better idea. The timing depends on coordination and getting the data ready with the CROs and the sites. It is a much larger data set than for Stargardt disease, so it is more complicated, but we are aiming for the end of the year.
Right. Okay. So you will have a sense of that, right?
Yes. We are aiming for the end of the year, but it depends on coordination with our partners and contractors. We will update you as we get closer and have the data in hand.
And then just back on Stargardt, what is the timeline for Japan again?
Given that we have Sakigake designation in Japan, the PMDA is aiming for approval within three months of the FDA approval. So we are looking on track for that as well.
And your next question comes from the line of Steve Seedhouse with Cantor. Your line is open. Please go ahead.
Great. Thanks, and thanks for the color on the commercial preparations in the U.S. I actually wanted to ask about ex-U.S., particularly Europe. How are you thinking about filing timeline, launch strategy, partnering strategy if relevant? Would love your current thinking on the ex-U.S. opportunity.
Sure. Again, right now we are focusing on the FDA approval. During the submission timeline and the FDA review period we expect to receive questions from the FDA, so we do not want to overstretch ourselves by filing in multiple jurisdictions while focusing on the FDA. Our filing strategy is that the FDA submission will form the basis of our submissions elsewhere, and the rest of the world filings will be consistent with the FDA filing. The timeline for ex-U.S. will be based on our responses and interactions with the FDA. At this point the timeline needs further updates and the FDA will serve as our priority.
I just want to follow up on the GA analysis around year-end as well. Is this the type of situation where you would share data in any scenario, resize the study, or stop the study either for efficacy or futility? Can you talk about some of the possible scenarios for analysis? My assumption is resizing the study is a possible outcome. Will the interim show whether the sample size needs adjustment?
Steve, the decision will be data-driven. The interim analysis will inform whether any sample size adjustments are needed. These are typical possibilities: sharing data, resizing the study, or stopping for efficacy or futility, but we will make those decisions based on the data. We are doing everything we can to bring this treatment to patients around the world—for both GA and Stargardt—but as Tom said, some of these decisions will be data-driven. Stargardt disease in the U.S. will be our first focus, but we will continue to monitor other developments and try to bring the treatment to all patients as soon as possible.
And your next question comes from the line of Greg Swanovic with Mizuho. Your line is open. Please go ahead.
Hey, it’s Greg. Thanks so much for taking my questions. Congrats on progress. I had two questions. One, it has been some time since you had the Phase 3 data in hand. Have you done any additional payer market research in terms of potential pricing bands that would be acceptable? What are your latest thoughts on potential pricing? And second, appreciating that you are ramping up pre-commercial activities, can you give a sense of the level of awareness of Tinlarebant right now with the prescribing community and how much education will be needed once you get to launch?
I will ask Hendrik to discuss awareness and the clinical community's view of the data, and Hao to comment on our commercial research and pricing considerations.
The R&D and retina specialist community is a well-defined group that meets regularly at ARVO, ASRS, and the American Academy meetings. People know about Belite Bio and Tinlarebant. We can certainly improve awareness because our interviews with retinal specialists show enthusiasm for the prospect of a first treatment for this untreatable disease and appreciation for the convenience of an oral treatment. However, the proportion of retina specialists with in-depth knowledge about Tinlarebant and the DRAGON trial needs to be improved. We will be present at the American Society of Retina Specialists meeting in Montreal in July, the Retinal Society meeting in Los Angeles in September, and the American Academy meeting in October, with presentations at all of those meetings. These will be major opportunities to educate the community about this forthcoming treatment for Stargardt disease. We are actively pursuing that.
That was a long question. What was the second half regarding pricing and payer research?
Greg, we have done several pricing projects so far. Payers have been supportive of the price range we are considering and recognize the strong unmet need as the first treatment for Stargardt. It is still early to set the final price, but as a reference we consider the average orphan/new ophthalmology drug price in the U.S. of around $350,000 as a fair reference and potentially up to $500,000 as a possible range when compared with some analogs. We have not finalized pricing, but this is a reasonable assumption range based on our payer research so far.
Okay. Thank you.
And your next question comes from the line of Yi Chen with H.C. Wainwright. Your line is open. Please go ahead.
Thank you for taking my questions. Assuming you get FDA approval in early 2027, can you tell us how quickly you can launch the drug, whether your manufacturing is aligned with that timing, and more importantly, can you provide a rough estimate of how many patients could reasonably be expected to receive Tinlarebant treatment in 2027? Thank you.
Hao, do you want to take that?
Yi, this is a small-molecule drug, so manufacturing, packaging, and distribution are comparatively straightforward. We expect to be able to launch relatively quickly upon approval. We are getting our supply chain and manufacturing ready now. Regarding patient numbers in the first year, we are conducting surveys and will present a more thorough market estimate at a commercial day in September. We are doing everything we can to identify potential patients and warm up the community, but we cannot provide a specific guidance on patient counts on today's call. Regarding operating expenses, it is reasonable to expect OpEx to increase as we prepare for launch. We have had significant team expansion—last year we were around 30 employees and now we are close to 90—so expenses are increasing due to hiring and pre-commercial activities. That said, compared to last quarter that ramp was just starting, so sequential comparisons can be misleading. We ended Q1 with close to $800 million in cash, so we are in a very comfortable cash position. To launch Stargardt in the U.S., you would probably look for about $300 million, and for our existing pipeline over the next three years we expect about $150 million, so in total roughly $450 million. With approximately $800 million in cash, we believe we are well positioned to fund these investments.
Got it. Thank you.
Thank you. There are no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.