Aura Biosciences, Inc. Q1 FY2026 Earnings Call

Good afternoon, and welcome once again to TD Cowan's 7th Annual Oncology Innovation Summit. I'm Phil Nadeau, one of the biotech analysts here at Cowan. It's my pleasure to do a fireside chat with Aura. We have with us today Natalie Hollis, the CEO of Aura. Natalie, congratulations on assuming the role of CEO at Aura. We are sure you had a lot of opportunities. Can you discuss what attracted you to Aura and convince you to take this role in particular?

Certainly, and thanks for having me here, Phil. And I'm delighted to chat about Aura and my awesome new job I'm very excited about. So what attracted me to Aura, this was a unique opportunity. I was in the fortunate position of being able to look at a lot of different opportunities. And what really piqued my interest was the potential, the compelling value proposition of Belsar as a new treatment for a patient population that is really desperately in need of better treatment options. And in a space where there's really no other innovation, even on the distant horizon. So that combination of a really interesting data set and a wide open development space from a competitive perspective makes for what I believe to be a really underappreciated market opportunity for Belsar and early choroidal melanoma. And as an operator, those types of underappreciated opportunities are really exciting. And so it's been a great first month and I'm thrilled to be here.

Maybe more broadly, looking out over the next year, what do you think are the challenges that Aura faces? And you've talked a little bit about the opportunities, but maybe going into a little bit more detail there. So what does Aura need to do to drive our performance over that next year

two-year period. Yeah. So we're in the process of wrapping up enrollment in Compass, which is our registrational study of BELSAR for the treatment of early coroidal melanoma. Very exciting milestone for the company. And with the achievement of that milestone, our attention turns to BLA preparation and getting ready for a potential commercial launch into this patient population that, again, is very much in need of new treatment options. So the next 12 to 24 months includes outstanding execution on completing data collection within the COMPAS study. And then BLA prep, not only on the clinical piece of the BLA, but the CMC piece as well. So that's where the team will be focusing over the next, again, 12 to 24 months. And then beyond the early choroidal melanoma program for BELSAR, we're also excited about the opportunity for indication expansion within the ocular oncology setting with our ongoing studies in choroidal metastases and ocular surface cancers. And with the recent financing, we intend to invest more meaningfully in these programs to get to POC data in those two studies to inform future decisions on how we really build a business in this space. So I would say critical success factors, to answer your question directly, are just continuing to execute on the COMPASS study through top-line data in the back half of 2027, advancing our choroidal metastases and ocular surface programs to ensure a nice steady cadence of data catalysts over the next two years or so, as we work towards achieving our vision of building the world's preeminent ocular oncology company.

To dive into each of those programs in a bit more detail, maybe starting with choroidal melanoma, can you remind us of the highlights of the data from the phase two trial of suprachoroidal Belsar and choroidal melanoma. How does that data compare to the data produced by Belsar and IVT, as well as standard of care for choroidal melanoma?

Yeah, certainly. So as a reminder, in the phase two study of Belsar, we administered ascending doses of Belsar via suprachoroidal injection, followed by laser activation of this VLP di-conjugate drug substance. And in the efficacious dose range of that ascending dose study, at 12 months, we saw 80% tumor control and 90% visual acuity preservation and a highly favorable safety profile. This was a really exciting result because current treatment choices for these patients are dire. Choice one is watch and wait, which can lead to metastatic disease with its attendant very poor outcomes. Option two is radiotherapy, which can control tumor growth, but leads to blindness and upwards of 90% of patients who received the treatment. So to be able to arrest tumor growth, which is considered a functional cure in early choroidal melanoma, while preserving vision is a huge win for these patients. As you mentioned, we had an earlier study in which we administered Belsar via intravitreal delivery, and there we saw some posterior inflammation that impacted the safety profile and the efficacy of the drug. But in shifting to suprachoroidal injection, we were able to largely eliminate the posterior inflammation and produced a safety profile, which was really, really mild, mostly grade one AEs, all of which resolved with no or short-term treatment, no treatment-related SAEs, no grade three to five treatment-related SAEs, or AEs, excuse me. So it was a really encouraging safety profile and demonstrated that in moving from IVT to SC administration, we really optimized the therapeutic profile of Belsar. So we were really pleased with the phase two results and the potential that BELSAR has to shift the treatment paradigm for these patients.

Turning to the phase three trial, for those less familiar, could you remind us of the design of that study? How many arms are going to be tested? How many patients will enroll? Kind of the basics.

Yeah, so this is a three-arm study enrolling approximately 100 patients with early choroidal melanoma with a primary efficacy analysis evaluating an 80 microgram dose of Belsar versus a sham injection. The primary efficacy analysis is a 15-month landmark analysis looking at time to tumor progression. And so as a reminder, in this setting, halting tumor growth is considered a functional cure. A key secondary endpoint is a composite of either time to tumor progression or visual acuity failure, whichever comes first. And while we expect that tumor progression will be the primary driver of efficacy in this analysis, collecting data on vision preservation was really important to this division of the agency and is very important to us as an important differentiator versus radiotherapy. And so having those data on the label will be really important and impactful for us in the commercial setting.

Or has received an SPA for the design of the trial? What elements are aligned in that SPA?

Yeah, so it's an agreement on the design, the size, and the efficacy and safety analyses in this study required to support registration based on the results of Compass. This was a highly collaborative effort between the team and the division far before my time joining the company. But it was a great win because we are heading into BLA filing with a very high degree of clarity on what the bar is going to be for approval. And the size of the study, the approximately 100 patient size study is really driven by the need to collect sufficient data, safety data in this population to support registration. But what it also means is that we are very highly powered on our primary endpoint based on the results from our phase two study. So as I said, enrollment is complete. We are now in data collection, integrity, analysis, and reporting mode and heading towards a potential BLA filing, which is really exciting.

I think the most recent guidance is data from the trial in the second half of 2027 as you work on the data collection portion of the study. Is that still on track? Very much so. And you referenced the very highly powered nature of the primary endpoint of the study. What have you disclosed about the powering of the trial on that primary endpoint?

So in moving from phase two to phase three, we really sort of kept as many variables consistent as possible in terms of exclusion criteria, route of administration dose. And so if we are looking to recapitulate what we saw in the Phase II study in terms of an 80% response rate in terms of arresting of tumor growth, we are over 99% powered on that primary efficacy analysis.

And you mentioned the key secondary endpoint looking at both time-to-tumor progression and visual acuity failure. Can you go into a little bit more detail how important that secondary endpoint is for regulatory approval and then ultimately for commercial adoption?

Yeah, so I think from a regulatory approval, it is a secondary endpoint. This was an important element of the efficacy analysis for the ophthalmology division of FDA. And so it is important from a regulatory perspective. I think it's more impactful ultimately in the commercial setting because, again, radiotherapy does a very good job of arresting tumor growth. Rates are about what we saw in the phase two of Belsar, but with a very high frequency of vision loss. And so if we can achieve similar efficacy while preserving vision, that's a huge win for these patients.

What do you perceive are the key risks to the trial success? I mean, the phase two data seemed to show pretty clearly that tumor growth is arrested, as you said, in 80% of the patients. But as investors, what should we worry about as we look forward to that phase three data release? What could trip up the trial?

Well, I think that the big risk is essentially overcome, which was really around enrollment. And it was a long time to get this study fully enrolled for reasons that we can talk about. But now we're there. We are functionally complete with enrollment. And so now the team's focus shifts from finding patients to keeping patients in the study, making sure that data collection continues, that we are maintaining the integrity of the data, clinical quality, and BLA readiness. So it's very much an in-the-shoot execution time for us, which is exciting. And then in addition to running the clinical study, as I said earlier, we need to get the CMC side of the BLA ready as well. So two key areas of focus as we approach BLA and choroidal melanoma.

You discussed the market. What's the global incidence and prevalence of choroidal melanoma? Are patients diagnosed? How are they treated today?

So for early stage disease, we believe the incidence is about 8,000 patients per year between the U.S. and the EU5. That's based on current diagnosis and treatment paradigms. And as you often see, and I've seen this time and again in other markets, the introduction of a new treatment option that meaningfully moves the needle for these patients, particularly one with a potentially transformative profile like Belsar, these numbers tend to grow. So you find more patients when you have treatment options for them. So these patients are typically diagnosed on routine eye exam, looks like a spot on the retina. The differential diagnosis between a benign freckle or nevi and early choroidal melanoma is often based on assessment of a number of risk factors by retinal specialists. And the decision to watch and wait versus treat with radiotherapy we're finding really varies from physician to physician, because again, the treatment comes with the terrible downside of blinding the patient. And so this is why the potential of Belsar is so transformative. For the first time, there's this third choice by which you can halt tumor growth and preserve vision. And so all that is to say that with that new treatment option available, we could see those

numbers grow over time. How does Belsar compete to some of the other agents in development today? Any notable competitors in the pipeline?

Yeah, in the early choroidal melanoma space, there are no other competitors. There are some really important drugs being studied, or even on the market, in metastatic uveal melanoma that are important treatment options for patients for whom the disease wasn't caught early and they've advanced a metastatic disease. But in the early choroidal melanoma space, Belsar is the only treatment being evaluated. And, and again, the choices are watch and wait, risk metastatic disease and becoming a patient eligible for some of the other therapies that are being studied sort of further downstream in the disease, or treat with radiotherapy and blind the patient.

Great. And you mentioned an instance of 8,000 patients per year. Does that include indeterminate lesions? How do those lesions differ than choroidal melanoma itself? And do you think the treatment paradigm could evolve to include treatment of indeterminate lesions?

Yeah, great question. So the way to think about indeterminate lesions versus small choroidal melanomas, they are all early choroidal melanoma. The sort of nomenclature of indeterminate lesion versus choroidal melanoma is often made sort of based on treatment decisions by the physicians. So if a patient is going to be treated, it's diagnosed as early choroidal melanoma. If it's a watch and wait, nobody likes to have a cancer diagnosis and not have anything done about it. So they end up being called indeterminate lesions. And what we're seeing in the field is that, again, with the introduction of a new treatment option, the nomenclature and the way that physicians are talking about this patient population shifts. All of these patients are early choroidal melanoma. With these risk factors in place, they are all potentially treatable with Belsar. And that's really a lot of the work that our medical affairs and market development different teams are going to be doing as we approach launch, which is really helping to shift that paradigm towards seeing this as one big patient population, which is eligible for therapy.

What are Aura's most recent thoughts on pricing? Any goalposts that you're willing to provide?

Yeah, so we haven't given any guidance. But I think, you know, what I would say is, based on the product profile that is emerging from the phase two data in terms of a high degree of efficacy with a really compelling safety profile, the lack of other treatment options available for these patients, the overall size of the patient population. I would say I think that the analysts that are covering the company have taken a very conservative view on what might be appropriate from a pricing perspective for Belsar. So more to come on that. And certainly it'll be driven by the results of the phase three study, but we're really excited about

the profile we're seeing today. And last question before moving on to some of the other indications, any potential for redosing of Belsar in patients who have recurrence?

Yeah. So it's a great question. So the first thing to point out is these patients already receive a number of injections. This is a course of therapy, so you don't have this redosing immunogenicity problem that you worry about with AAV-based gene therapy, for example. I think the possibility exists, and our long-term follow-up data will provide more insight into what long-term effects look like with treatment of Belsar. There's no reason that one, two, three years down the line, if there's some regrowth that patients couldn't be treated again, there's also no reason that patients can't move on to radiotherapy at this point. So it really is a very attractive first-line option for, again, halting tumor growth without impacting vision.

Turning to choroidal metastases, could you give a brief overview of choroidal metastases, the current treatment paradigm, and maybe even just the very basics? What's different between a choroidal metastases and choroidal melanoma?

Yeah, so choroidal melanoma is a primary melanoma tumor that starts in the choroid. Choroidal metastases are metastases from other primary tumors that appear in the choroid. And some of the primary tumors that we see often metastasize into the choroid are breast and lung cancer. Current standard of care is really grim for these patients. They've got to move to a place where there is external beam radiation and receive daily radiation for four weeks and then go home. So this is a really high treatment burden for patients who are already very sick. Um, so in terms of the opportunity here, um, to treat with Belsar and see, um, see more efficient, effective reduction in tumor size and hopefully preservation of vision, it offers a really compelling treatment option for these patients.

Do you describe the design of the phase two study?

Yeah, it's a standard three plus three dose escalation study. And we're looking at changes in tumor size and potential for visual acuity improvement or loss or prevention of loss of visual acuity. We're starting with an 80 mg single cycle course of therapy. So 80 mg is the top dose that we dosed at phase two. It's the phase three primary efficacy analysis dose in the COMPAS study for choroidal melanoma. And we're only doing one cycle versus the three cycles that we're doing in coroidal melanoma because these patients are sick. And so we wanted to limit the treatment burden. And study is currently enrolling, and we are on track for early data from this study later this year.

Any tumor types or primary tumor locations that you think would be amenable to Belsar in the metastatic arena? And any specific, like, would breast work better than lung or vice versa?

Great question. So inclusion criteria are broad in terms of primary tumor type. But as I said, breast and lung are the most common. We have extensive preclinical data showing good activity against multiple tumor lineages with Belsar. And so I don't think there's any reason to think that breast would respond better than lung, for example, based on the preclinical data. But of course, the clinical data will inform where we go from here.

Can you give us some sense of what you would consider proof of concept? What response rate or what other efficacy data you'd need to see to move forward in any specific?

Yeah, fair question. I think it's a little early to say we'll know when we'll see it. But, you know, we're looking for reductions in tumor size and certainly maintaining vision in this patient population is a really important factor as well.

Moving on to cancers of the ocular surface, can you outline the design of that phase one trial?

Yeah, so this is an interesting program because here we are making a couple of changes to the root of administration. So rather than suprachoroidal injection, this is intratumoral injection to the front of the eye. These are cancers on the surface. And there's a slight difference in the way that the laser is directed rather than a standard retinal laser. This is a laser that just penetrates the anterior surface of the eye. So this first study is really a safety and feasibility study, just given the new route of administration. And then standard of care for these patients is excision of the tumor. And so unlike in choroidal melanoma, where we don't have the opportunity to look at sort of the primary tissue effect of Belsar treatment, in ocular surface cancers, we do. So we can look for evidence of necrosis, secondary immune infiltration, based on that administration of BELSAR.

And what do you hope to see in that initial data to form proof of principle or proof of concept?

Yeah, I think, again, safety that we can deliver reliably and administer the laser in a way that is effective, and then evidence that the drug is doing what we believe it to be doing based on the mechanism of action studies. And then, you know, from there, it'll inform, you know, Do we need to make any tweaks to the route of administration? Are we seeing what we want to see? It's really an exploratory study, whereas in the choroidal metastases study, it's much more similar to how we're treating in early choroidal melanoma. So there's less sort of exploratory elements to that study and really driving right towards safety and efficacy.

This trial is beginning in Australia. What's the rationale for starting in Australia?

Yeah. So you see a higher incidence of cancers of the ocular surface in Australia, given the population's exposure to higher levels of UV radiation. So it was just a good place to start to be able to find a pretty enriched population. Right. And can you give us an overview of the

market? How many patients, incidence prevalence, and I think you mentioned standard of care as excision. Anything else to say about how these patients are typically treated?

Yeah. So this is a group of cancers affecting, this is a bigger population. We estimate about 35,000 patients per year are diagnosed with ocular surface cancers. And the current treatment paradigm is a combination of surgical excisions, which are unpleasant, as you might imagine, or topical off-label chemo, which also has a high degree of safety and tolerability issues associated with it. So patients can be treated, but they're left with lifelong dry eye and discomfort. Some patients lose their vision or end up having the eye enucleated. And so a pretty dire set of treatment options for this type of ocular cancer as well, which is why we really like the potential to build this nice sort of cohesive and efficient business around Belsar in this larger group of ocular cancers.

Should you decide to move forward in ocular surface cancers, what would be the next steps? Would you go on to a phase two? Could you go right into pivotal?

Yeah, great question. I think too early to say because this is a more exploratory study. And we also, as you mentioned, we're running the study in Australia. So we'll take the data, we'll go back to the regulators, have a discussion about the best way to proceed.

And then maybe we'll finish up with just a corporate question. Can you remind us of your balance sheet? What's your current cash

balance and anticipated runway? Yeah. So as I mentioned earlier, we did a follow-on financing about a month ago. With the proceeds of that financing, we had our pro-pharma balance sheet is $355 million as of March 31st, which provides runway into the back half of 2028, so well beyond our expectations for top line data readout from Compass. So we're really pleased that the company is so well financed through a number of catalysts over the next 24 months as we, again, work towards our vision of building the world's best ocular oncology company. That's great. With that, I think

we're out of time. Congratulations again on the new role, and we look forward to the upcoming data.

Great. Thank you so much, Phil.