Opus Genetics, Inc. Common Stock Chardan's 10th Annual Healthcare Conference

Yeah, thanks. I'll start with the economics piece, and then George, maybe you want to take the strategic piece on why this one's different. We do earn a $10 million milestone associated with the approval. We would expect that to come in this quarter. It has up to $130 million total milestones over the course of the arrangement, and it has a very attractive royalty rate as well. The royalty starts off, it's undisclosed, starts off sort of lower teens and then quickly scales up to a higher level, but it's a very attractive royalty for us.

Those are the economics. From a strategic standpoint, it's really interesting because it's going to be, well, it is now, the first approved presbyopia drop that does not have a warning label for potential of a retinal detachment.

It also doesn't have the same night glare issues that you get or nighttime dimming issues with some of the strong miotics. It does that because it fundamentally does not engage the ciliary body. We have a much cleaner safety profile that allows one to increase the drop and hopefully should provide a good treatment option for patients.

Yeah, so good questions. In the pivotal trial, it's an 8-patient pivotal, so I think it's the smallest trial in ophthalmology that's being done for a pivotal. The primary endpoint is going to be proportion of patients that achieve a greater than 7 decibel improvement on microperimetry. Now, in the central 16. Microperimetry is a test where you pointwise test the sensitivity of the retina. You have a grid across the retina that tests 68 points or so. The central 16 tests the center, just like it says, the center 16. The average of those needs to be above 7 decibels. Now, in our prior study, our average was 10 and a half decibels, and so we feel pretty confident in that bar. So right now, the way the trial is structured is that the control for the trial is actually the 6-month period preceding treatment.

The patients serve as their own control. The patients have all completed that 6-month period, and we're currently dosing. Dosing should conclude pretty quickly here with a readout by the end of next year.

Then- Addressable market. Yeah. Sorry about that.

No, no. This is an addressable market of around 150 to 200 patients.

Almost all of those are seen at the University of Pennsylvania, and so the commercial efforts will be quite modest as far as what's required to get to those patients. We would expect a pretty high penetration into this population, given the safety profile of the drug and the pretty dramatic efficacy that we've seen for our patients. The fact that most of these patients are captured at the University of Pennsylvania. It will qualify for a priority review voucher as well, so you can see that the economics can be quite meaningful.

Well, I mean, BEST1, I have been involved with over 100 ophthalmic trials. It is probably the most exciting trial I have been involved with. The fact that the vitelliform material of BEST vitelliform macular dystrophy is disappearing and the function of the retina in exactly the same location is improving is quite a meaningful sort of structure-functional correlation. The second reason it is exciting is to enroll cohort 1 took us maybe six months. Cohort 2 was five patients. We over-enrolled it to 11 patients in about three weeks. To see that kind of excitement from patients and physicians is, I think, a really cool thing. We have currently enrolled cohort 2, 11 patients, originally planned at five. We will read out that data in the second quarter of next year, along with an update of potentially flipping into the pivotal trial.

The way we construct these trials is really an adaptive trial. The protocol that we are using for the current dose escalation would be directly adapted into the pivotal trial, and it really saves a lot of time and money on the execution side. We have already aligned with the FDA on what the endpoint will be. Based on cohort 1, it will be, again, a microperimetry endpoint for the pivotal.