Release Date: August 07, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- FDA has confirmed that the SOL-1 and SOL-R studies are appropriate as registration-enabling studies for AXPAXLI in wet AMD.
- Ocular Therapeutix Inc (OCUL, Financial) has approximately $460 million in cash, providing a cash runway into 2028.
- Enrollment in the SOL-1 study is accelerating and exceeding expectations, with 60 sites activated and over 150 patients in various stages of loading and randomization.
- Initiation of the SOL-R study for wet AMD was completed in just 3 months, with the FDA confirming it as a registrational study.
- Positive results from the Phase I HELIOS study in nonproliferative diabetic retinopathy (NPDR) show that no patients developed vision-threatening complications at 48 weeks after a single AXPAXLI implant.
Negative Points
- The company had to make the difficult decision to reduce headcount in areas not aligned with its vision.
- There is still uncertainty regarding the exact timeline for the completion of patient enrollment in the SOL-1 and SOL-R studies.
- The company has not yet had a formal meeting with the FDA regarding the nonproliferative diabetic retinopathy study.
- The SOL-R study's retreatment criteria are still under discussion with the FDA, leading to potential uncertainties.
- The company has not provided specific updates on the number of patients randomized in the SOL-1 study since the Investor Day.
Q & A Highlights
Q: Congrats on getting the alignment with FDA. Pravin, I wanted to maybe ask you with that now into your SOL-1 study has been recruiting much faster than expected. And at your R&D Day, you talked about how that could also feed into the speed with which the SOL-R study could recruit. Have you had a chance to talk to FDA about whether both studies would need to be submitted at the same time when you do apply for approval or kind of potential rolling submission beyond the table? That's my first question. And then secondly, as you think about the time period between inserts that might be needed for the SOL-1 study, you've talked about an average of maybe every 9 months. Can you talk about how's the experience as the retina position, the variability that physicians or flexibility that physicians may choose to have maybe some dosing more frequently and some dosing less frequently? And where you think at the end of the day, the average dosing frequency for the insert will be?
A: Tazeen, thank you for the question. I appreciate the thoughtfulness of your questions. First of all, let me make it very clear. We are absolutely thrilled with the written FDA response. It validates what we've been saying to the Street all along, which is that our thinking is absolutely in line with the FDA requirements. And now we have this in writing and this absolutely validates everything that we've told you. We also have in writing that these 2 studies will potentially qualify for approval. One is a noninferiority study and the other one is a superiority study. These 2 studies are very thoughtfully, very carefully designed to answer not only the regulatory requirement questions, but also have a commercial impact. Very importantly, as I've been saying all along, the SOL-R study is specifically designed to actually increase the recruitment in SOL-1. Having said that, I must tell you that we are very pleased with the continued pace of recruitment in SOL-1. As you know, the recruitment is not necessarily linear. After you hit a certain critical mass, it becomes exponential. And we clearly are in that phase. We are very pleased with the recruitment of SOL-1. Now in regards to your question about the clinical, the potential clinical use of AXPAXLI. Our goal, as you know, is to get this drug to patients as soon as possible. We believe that when you take the SOL-1 and SOL-R in its totality, it allows physicians the flexibility of dosing up to every 6 months. In some patients, this may be necessary, in a lot of patients, this will not be necessary and patients may be extended further for treatment every 9 months, 10 months or even a year. So the important point here is that to recognize that this disease is a heterogeneous disease. Patients will be treated in a personalized fashion, I believe, as they have -- as they are being with the anti-VEGF today. But the important part is that, given the totality of these 2 studies, it answers many questions while providing a clear regulatory pathway and gives the physicians the flexibility in terms of the personal treatment and having a [indiscernible] treatment regimen with potentially a better long-term outcome.
Q: Congrats on the regulatory update. Maybe, Pravin, if I could start with SOL-1. I think the company previously guided to patient enrollment completing in the trial in the first half of 2025. Do you have an update on this timeline? And then second question is, you've got the FDA feedback. And I know there has been some discussions with EMA. Any update there in terms of the EMA's thoughts on the design of SOL-R and SOL-1?
A: Biren, thank you for your question. So first of all, you're right, we did guide in our previous filings the effective recruitment for SOL-1 being the end of the first quarter of 2025. We did also say that given the pace of enrollment for SOL-1, we will [indiscernible] earlier, we have not changed any official guidance since then. It is too early to do so. When appropriate, we certainly will update you with the proper [indiscernible]. In regards to the EMA, clearly, AXPAXLI is a global drug, and we are in conversations with the EMA. We're very, very pleased with our conversations. And when the time is appropriate, we certainly will update you as to that as well.
Q: It's great to see the news on the FDA feedback here. So my question is, if you can elaborate on the term that you put in the press release that's generally acceptable in the written response, and if you believe there's any remaining risk to both studies satisfying the NDA requirements. And really, if so, if there's anything that you're doing to mitigate that proactively.
A: Look, the term that we use is a term that the FDA uses and it's a term that's generally used. There's really nothing to read into that. The take-home message here is that we are absolutely thrilled with the alignment that the FDA has, and we have that now in writing, and I repeat, in writing. So this is not subject to any interpretation. It is in writing that they're thinking, that their requirements are absolutely aligned with what we've been saying. For example, we have been saying, and this is aligned with the FDA and we have this in writing, that sham is not recommended, that it does not constitute complete masking. It has the ability to introduce bias. And any study done with a sham will be subject to review. That is what we've been saying all along. And now we have that in writing from the FDA in a Type C response. As you know, we don't have any sham in any of our studies. We are not willing to do any of our studies at risk. We believe that we're an absolute alignment with what the FDA requirements are as of just a few days ago.
Q: Congrats on the progress. So with the FDA written feedback, would you expect to also plan to pursue a SPA for SOL-R. And then based on the feedback that you received, are there any changes that you plan to make to the SOL-R study? And can you just confirm how the FDA is viewing the role of the comparator arm? Do you need any sort of statistical significance difference in terms of the Eylea high-dose arm?
A: Colleen, thank you for your question. It's important to state that we requested a Type C meeting and the FDA responded by saying that they know us and our drug well enough to have a written response. As you know, we have a SPA for the SOL-1 study. To answer your question directly, look, we believe we are in complete alignment with the FDA, as we've been saying. We believe that everything that we said, and I've just given you the example regarding sham, is absolutely validated in this written response. In regards to the potential for a SPA, the answer is I honestly don't know at this point. We've just received this written response a few days ago. What I will tell you is that
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
