Ocugen (OCGN) Q2 FY26 earnings call: guidance and Q&A

What are the regulatory plans and timelines for OCU400 in ex-US jurisdictions, particularly how they compare to the US BLA path?

Ocugen has alignment from EMA for the same single trial to be sufficient for approvals. For orphan gene therapies, ex-US approvals typically follow US FDA approval, linking all international timelines to the US FDA process.

“Michael, what we have with RQ400, we got alignment from EMA in addition to FDA with the same that single trial we're doing in the US is good for approvals. And across the globe, for orphan gene therapies, typically they get approval based on U.S. approval.”

asked by Michael Okunewitch · answered by Shankar Musunuri

What are the key differences in trial design between the Phase 3 Armada 3 and the Phase 1/2 Armada trial for OCU410?

Armada 3 is a global Phase 3 trial with one treatment arm (OCU410) versus control, a 2:1 randomization, and 12-month follow-up for the primary endpoint. It will enroll 237 subjects globally. The primary endpoint will be similar to Armada 1, expecting similar trends, but with a larger, global scope.

“Regarding Armada 3, which is our global phase 3 trial for GA, which we just got the approval from FDA, you know, just recently to be initiated this quarter, the phase 3 trial designed for Armada 3 will be one treatment arm with OCU410 versus a control will be two to one randomization allocation and that day to follow each subject up to 12 months.”

asked by Michael Okunewitch · answered by Unknown Speaker

How will Ocugen approach pricing for OCU410ST, considering potential chronic therapies entering the market and the difference between one-time vs. chronic treatment models?

Ocugen's one-time treatments will have a different pricing structure than ongoing chronic therapies. Pricing will be dictated by data, focusing on safety, efficacy, and the potential for disease reversal, not just slowing progression. Differentiators include targeting pediatric patients (3+ years) compared to 12+ for competitors, and the disruptive technology platform allows for pricing based on its own merits rather than comparison to chronic therapies.

“I think the way you look at it is our treatments are one and done treatments potentially. So that will have a different pricing structure than ongoing chronic therapies. Number two, everything will be dictated by data. And if you have a safe one-time treatment, I think our gene therapies, once again, we're still collecting data.”

asked by Michael Okunewitch · answered by Shankar Musunuri

Can you elaborate on the Target Product Profile (TPP) for OCU410ST, specifically the potential for disease reversal and improvement in visual acuity, and if the trial design enriches for this outcome?

The OCU410ST trial population is broader, including early to late-stage patients and pediatric patients (3+ years), and a wider range of lesion sizes. Prior Phase 1 data showed slowing of structural progression and functional benefits, including visual function improvement (gain of fixed letters close to one line). While the primary endpoint for the one-year trial is lesion size, secondary visual function will be monitored, and long-term data from Phase 1 patients will be available.

“So our population was definitely broader than other competitors. Just to highlight, first, we included patients from early to late stage target disease. That's number one. As Shankar just mentioned, too, we include subjects, you know, are younger than, you know, young adults. We included, you know, subject, you know, three plus years of age, so that's a very broad population.”

asked by Whitney Ijem · answered by Unknown Speaker

How is Ocugen thinking about its cash position, given the progress with the GA study, and what levers can be pulled to extend the cash runway further, balancing GA startup vs. commercial prep?

The company has a cash runway into 2028. Levers to extend this include the potential sale of the OCU410ST Priority Review Voucher (PRV) for $100M-$200M, evaluating ex-US business development deals for all programs, and the Janus Henderson warrants (10M warrants at $1.50 strike, potential $15M). They also anticipate a special meeting for share authorization to raise additional equity if needed, aiming to minimize shareholder dilution while bringing products to market.

“So, first of all, I mean, our primary goal is to make sure that we are, you know, minimizing shareholder dilution, but evaluating our opportunities in order to raise capital, just as you said, in order to bring these novel products to patients. So, just first of all, we have cash runway into 2028.”

asked by Whitney Ijem · answered by Unknown Speaker

The Armada 3 trial size for OCU410 was resized from ~300 to 237 patients. Was this requested by the FDA, or was it based on new assumptions regarding effect size or variability?

The adjustment to 237 patients was discussed and aligned with the FDA. It was based on sample size estimation and power calculations, specifically leveraging the 31% reduction seen in the Phase 1/2 Armada 1 trial at the optimal dose. This size provides 95% power for the pivotal trial, with 158 patients in the treatment arm and 6,000 in the control arm.

“Yes, we had a discussion with the agency, the FDA, so all this being aligned and discussed with the FDA. But to answer your question specifically, it was based on all the sample size estimation and also the power calculation we did.”

asked by Charles Wallace · answered by Unknown Speaker

The OCU400 rolling BLA submission guidance shifted from Q3 to Q1 next year. What caused this change, and why submit non-clinical modules later?

The company is ready with CMC and non-clinical modules, having completed PPQs on time. The timeline for submission is based on agency comfort and decision, which they respect. While they can file non-clinical modules earlier after the pre-BLA meeting (following top-line data in Q1 2027), the PDUFA clock only starts once the clinical section is filed, which is planned for Q2 2027. This is a collaborative decision with the FDA, not impacting the overall approval timeline.

“Charles, I think from our perspective already, I mean, I think we're doing very well with our PPQs as we've mentioned. A lot of gene therapy companies stuck with CMC. We're ahead of the game. We used to commercial scale lots in phase three. We completed our PPQs on time. We got non-clinical and PPQ are done.”

asked by Charles Wallace · answered by Shankar Musunuri