Aura Biosciences, Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference

Thanks very much, Sean. I'm happy to be here.

Certainly. It's been four months since I joined the company, and I'd say what got me excited on my way in was that Aura represented a late-stage clinical opportunity that was as de-risked clinically and from regulatory perspective as anything that I had seen. The phase II data for our investigational product, bel-sar, in early choroidal melanoma, it's a small end, but I think you can't argue with the magnitude of the effect and the consistency of the finding across the subjects. I was intrigued, but what really got me excited about the opportunity was the true unmet need in the field of early choroidal melanoma, and the untapped commercial opportunity that it presented for us. We can talk more about that as we go on. But it was not only the clinical de-risking, but the opportunity to build a really important company around a special product.

When I joined the company, as I said, we were actually just finishing enrollment in our phase III registration study of CoMpass for treatment of patients with early choroidal melanoma. The evidence around the potential for efficacy and impact was really overwhelming and exciting. I believe, though, that you've got to pick one thing that you're going to be best in the world at and sort of go all in.

From my perspective, there's the opportunity to really impact patients' lives, build a valuable business focused just in ocular oncology. We had previously had a program ongoing in bladder cancer, a phase II study. We committed to the community that we're going to finish that study. We'll follow the enrolled participants through 12 months, but really focus the company's resources and attention in ocular oncology going forward.

Yeah. One of the things that's happening in the field is that this delineation between indeterminate lesions and small choroidal melanomas is really sort of going away, and the group is more broadly being defined as early choroidal melanoma. The reason for that is that the treatment choices for these patients have been dire to date. You receive this diagnosis, you have melanoma growing in your eye, you have two really bad choices. Number one is do nothing, leave it alone, and let it grow. Or we can treat it, but the treatment, which is plaque radiotherapy, is going to put most patients on a basically irreversible path to blindness. So the delineation between indeterminate lesions and small choroidal melanomas was really based on, am I going to treat or not?

If I'm not, I'm not going to say you have melanoma and do nothing. I'll call it an indeterminate lesion. If I am going to treat, I'm not going to blind you because you've got an indeterminate lesion.

Sure. I'm going to call it melanoma.

So what bel-sar represents is a whole new treatment paradigm. What we saw in the phase II data is that we got excellent tumor control. 80% of patients saw cessation of tumor growth, which is considered a functional cure in this disease, and it's our primary efficacy endpoint in the ongoing study. But also importantly, 90% of patients had vision preservation. So now you have the opportunity to treat the tumor without losing your vision. So that is really the opportunity that we have to change what is currently a pretty dire diagnosis for these patients.

Yeah. So CoMpass is a randomized phase III registrational study being conducted under a Special Protocol Assessment with the FDA in patients with early choroidal melanoma. We have delineations around the size of the choroidal melanomas, and importantly, for reasons that we'll talk about, the patients coming into the study need to have documented growth. So they need to have tumors that are growing. There are three arms to the study. The first is the therapeutic arm, which is bel-sar treated at an 80-microgram dose. We have a sham arm, randomized one-to-one between the 80-microgram dose and the sham arm. But then we also have a 40-microgram dose- which was designed in collaboration with the agency to ensure that our investigators were masked to treatment arms.