Q2 2025 Ocugen Inc Earnings Call
Good morning and welcome to Oyen second quarter 2025 financials results. In business update, please note. That this call is being recorded at this time. All participants lines are in listen-only mode. Following the speaker's commentary, there will be a question and answer session. I'll now turn the call over to Tiffany Hamilton oyens head of corporate Communications. You may begin
Thank you, operator, and good morning, everyone joining me on today's call and webcast is Dr. Chancre museri, oxygens chairman CEO. And co-founder who will provide a business update and an overview of our clinical and operational progress.
Rsh from. And chandran, our chief accounting officer is also on the call to provide a financial update for the quarter. Ended June 30 2025 Dr. Houma Kamar. Chief medical officer and Dr. Ruin aade Chief scientific officer will be available to answer questions following the presentation.
This morning, we issued a press release detailing Associated business, and operational highlights for the second quarter of 2025. We encourage listeners to review the press release, which is available on our website at oyun.com.
This call is being recorded and a replay with the accompanying. Slide presentation will be available on the investor section of the Oyen website for approximately 45 days.
This presentation contains forward-looking statements within the meaning of the private Securities. Litigation Reform, Act of 1995, which are subject to risks and uncertainties. We may in some cases, use terms such as predicts beliefs potential proposed continue estimates and anticipates expects. Plans, intends may, could might will should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements.
Such statements include but are not limited to statements regarding our clinical development activities, related, anticipated timelines.
Such statements are subject to numerous important risk factors.
risk and uncertainties that may cause actual events or results to differ materially from our current expectations,
These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission. The SEC, including the risk factors. Described in the section, entitled risk factors in the quarterly and annual reports that we file with the SEC.
Any forward-looking statements that we make in this presentation speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise, after the date of this presentation.
I will now turn the call over to Dr. Mussouri
thank you Tiffany and thank you all for joining us today. We're very enthusiastic about the progress of our novel modified gene therapy platform.
All three candidates are advancing through the clinic, with two in late stage, and we are on track to meet our goal of three biological licensing applications.
And Market authorisation application filings in the next 3 years. We remain steadfast in our mission to provide a 1-time therapy for life to address considerable unmet. Medical needs that exist for millions of patients facing the terrifying Prospect of losing their vision.
Patients are actively being recruited in the United States and Canada for the novel modifier gene therapy, ocu, 400 phase 3, Limelight, clinical trial for retinitis. Pigmentosa
And we are on target for bla and Ma filings in 2026.
You may have seen our recent social media collaboration with Molly bark, and outspoken, RP, patient. And an advocate to raise awareness for the Limelight clinical trial. It's gratifying to see the increased visibility for the work we are doing, and the response has been very positive.
Notable accomplishments this quarter included, the fda's agreement to proceed with the fees to 3 Guardian 3 pal, confirmatory trials. For oq for 10s, for stargard disease, following rare pediatric disease, designation RPD, and dosing of first patient. Earlier this month,
For OCU410, SD and OCU410 are positive, demonstrating favorable safety and efficacy with improved structural and functional outcomes.
The OCU 40033 Limelight clinical trial is the only global broad RP gene-agnostic trial to address multiple genetic mutations with a single therapeutic approach.
RK400 has obtained multiple designations to assist with regulatory review, including Regenerative Rate of Medicine, Advanced Therapy Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration, as well as Orphan Medicinal Product Designation (MPD) and Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA).
In the second quarter, the EMA granted eligibility to submit the ocu 400 Market. Authorisation application MAA through the centralized procedure based on the current study design and statistical analysis plan due to its now Gene agnostic mechanism for Action, okay? 400 has potential to address more than 100 different mutations associated with RP. In contrast, a traditional gene therapy approach would require development of over 100 products to treat each individual mutation, which is commercially not visible.
Currently.
The only approach gene therapy for RP targets. A single Gene, rpu, 65, which accounts for 1 to 2.
As a result, approximately 298,000 people in the US and Europe with other forms of RP. Remain without any approved treatment options.
In anticipation of the planned bla filing in 2026 where actively ramping up preparation for commercialization process, validation and Manufacturing activities, remain on track for completion this year.
The data and safety monitoring board. Recently, convened and reported no serious. Adverse Events related to oq 400 and recommended continuing the study of dosing as planned.
Star got disease is an inherited retinal disorder primarily caused by mutations in the abca4 gene with over 1200 different mutations. In this Gene identified to date.
It typically presents in childhood or adolescence and affects approximately 100,000 individuals in the United States and Europe combined, and an estimated 1 million people globally.
Despite its prevalence, there is currently no FDA approved Treatment available for stargard disease.
The ocu foral St. Clinical trial is progressing well having achieved key Milestones, receipt of
rare pediatric disease, designation in May
IMD Amendment clearance in June and first patient dosing in July in registration trial.
There is a clear sense of urgency from the agency to bring treatment on options to patients who currently have no approved Therapies.
As we initiate the Phase 23 registration trial, we are potentially accelerating the clinical development of OQ for 10s by 2 to 3 years, potentially delivering an innovative gene therapy to patients in critical need even sooner than originally anticipated.
The guardian 3 clinical trial builds upon encouraging results, and positive data from The Phase, 1 Guardian trial, which included, those range, and dose, escalation study to evaluate the safety and preliminary efficacy of ocq 410.
In The Phase 1 trial evaluable treated eyes. Demonstrated a 48%.
Slower Legion growth at 12 month. Follow-up compared to untreated lies. Additionally, treated eyes, show. A statistically significant with the P value of
0.031 and critically, meaningful Improvement of nearly 2 line or 9 letter gains, and best corrected visual Equity bcva at 12 month, follow-up. When compared to untreated eyes,
Offers a more comprehensive and one-time therapeutic approach.
Aki 410 has the potential to regulate all four pathways related to disease progression: lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of disease with a single subcutaneous injection.
The Phase 1 data at 12 months, demand State 23% slow lesion growth in the treated eye when compared to the untreated fellow eye after a single subretinal injection.
In addition to the structural improvement, Treatise also demonstrated a stabilization or gain in visual function in low light.
Low luminance since visual Equity with a 2 line or 10 letter gains when compared to untreated fellow eyes.
Preliminary results from 6-month intrum analysis.
In 31 subjects, demonstrated a 27% lesion growth.
and preservation of retinal tissue in the treated eyes and compared to
Untreated control ice.
The 27% reduction in lesion growth at 6 months, is more pronounced compared to currently approved, intravitreal therapies monthly and every other month.
P Saito client injections with demonstrated reductions of 13% and 12% respectively.
In addition to improved Legion reduction, a single subretinal injection of ocu, 410 demonstrates greater efficacy in preserving retinal tissue surrounding GA lesions compared to monthly.
And every other month.
Affect plan treatments.
We plan to provide full 12-month data from The Phase 2 study in the first quarter of 2026 and initiate phase 3 in 2026.
During the second quarter, we signed a binding term sheet for our first Regional partnership for occup 400 announced the spin-off of neoart. Into orto 2 significant, static events that we expect will make a considerable impact on Oxygen's financial position.
Aligned with oxygens Business Development strategy, for ocu 400, the company signed a term sheet to negotiate and enter into your licensing agreement with a well-established leader in the pharmaceutical and Healthcare sector in Korea for exclusive Korean rights to occup 400.
We are expecting to close the definitive agreement by September.
This regional partnership approach will allow Ocugen to retain rights and cover larger geographies.
Maximizing global patient reach while generating value for shareholders, we are actively engaged in discussions to explore a range of strategic partnership opportunities for Active 400 and all of our gene therapy candidates.
The proposed re reverse merger with ortho, a wholly owned subsidiary and Charisma, Therapeutics will create a NASDAQ listed late, clinical stage, regenerative Cell Therapy company with the first in class, technology platform focused on Orthopedic diseases.
The combined company will focus on the development of orthosis neocart Technology from the treatment of knee articular cartilage defects.
The Strategic move is intended to create value for oxygen stockholders without dilution of oxygen stock. As Aro selects is planned to be funded with 25 million in private financing under the exchange ratio Formula in the merger agreement artho selects its value is estimated at 135 million.
We believe there's a significant valuation increase opportunity for Orthocon's initiation of the Phase 3 NeoArt clinical trial, given the more than $2 billion market cap of the only other Atlas cartilage implant on the market today, Oxygen. Industry experts believe this market is significantly under-penetrated, and NeoArt could represent a true step forward in the innovation for ACX.
Please also have senior Financial roles at Astro, including CFO of the US, diabetes business. Satish brings more than 30 years of leadership experience across academic research early.
And meets these biotechs in large pharmaceutical companies, including Wyatt and fiser. Satish has his name on countless patterns and in recent years considers himself as a serial entrepreneur, leading biotech companies focused on developing products across a variety of therapeutic indications.
We also made updates to the Retina Scientific Advisory Board to reflect the absolute best guidance in this space. As we move closer to commercialization,
3, renowned retinal surgeons, who work at the Forefront of research and Cutting Edge. Advancements for retinal disease, doctors. Jeff higher.
Peter Kaiser and ashat. Kanani have joined the Saab to help us. Bring meaningful Innovative therapeutic options for patients, living with serious retinal diseases.
Finally, to optimize Ocugen's R&D and clinical efforts and build upon positive momentum, we are pursuing strategic partnerships and developing a commercial strategy with the company's internal expertise in critical functions.
Visit tomorrow, join oxygen in the newly. Created position of Chief development officer and brings over 32 years of global regulatory leadership with the Deep expertise and biotechnology biosimilar.
And complex, regulatory submissions.
Abby, Gupta has been named Executive Vice President and Commercial.
And business development following the retirement of Mike Shine, Abby has more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry.
And Michael blackton is our new VP of manufacturing and Supply bringing over 30 years of experience in quality operations and Manufacturing across the biotechnology and pharmaceutical Industries.
I'm delighted with these leadership changes and confident that we have the best people at the helm to take Ocugen to the next level of our success.
With that. Now, I will turn the call over to Chief accounting officer for machindra to provide an update on our financial results. For the second quarter ended, June 30 2025, romesh,
Thank you, Shankar. The company's cash, cash equivalents, and restricted cash totaled $27.3 million as of June 30, 2025, compared to $58.8 million in 2024. The company had 292.2 million shares of common stock outstanding as of June 30, 2025.
Total operating expenses for the 3 months, and at June 30th 2025, where 15.2 million including 8.4 million, in research and development, expenses and 6.8 million in general, and administrative expenses.
This compares to Total operating expenses for the 3 months, and the June 30th, 2024 of 16.6 million, that included research and development, expenses of 8.9 million, and general and administrative expenses of 7.7 million.
As always, we are constantly exploring strategic and shareholder friendly opportunities to increase our working capital and continue to pursue strategic Partnerships that will drive long-term strategy that concludes my update for the quarter, Tiffany back to you.
Thank you, Rash. We will now open the call for questions.
Operator.
Your first question comes from the line of Michael Okun from Maxim group. Your line is live.
All right. Thank you, everyone. Thanks for taking my questions today, and congrats on all the progress you've made.
Um, yes. So I
I guess to start out here, you’ve been quite busy. You did the Ortho Salic spinout. You did the Korea license agreement in the past couple of months. So are there any other deals that you might be looking to execute? Whether these be, you know, regional license agreements, asset sales, or spinouts? Is there anything that’s kind of been working on in the pipeline or identified as a goal?
Go ahead.
1 moment.
He just dropped wide. I'll put him back in now.
Okay.
Your line is live again. Michael, my apologies.
No worries. Thank you.
Yes, I just wanted to follow up on the star of Phase 23. Now that that started to enroll. Um, could you remind us how many sites you're including and then do you anticipate that the rare nature of the disease? May make it more challenging to enroll versus something like RP or ga
I would let the human answer the question. Yeah, so thank you for the question. Uh, star guard, um, phase, uh, 2/3, pivotal confirmatory trial is already started dozing. And we have uh, not only dosed 1 but quite a few patients have enrolled all together. Uh in the month of July, we do not anticipate any challenges in enrolling, star guard patients because the stats in
United States is 44,000 approximately patients for Star guard. And there is no approved product. Our inclusion exclusion criteria is, uh, very, uh, amenable and palatable to the population in terms of the bcva. And also, this particular ocu 410 is, uh, for all ABC for related, uh, uh, retinopathy as well. Uh, covering all, uh, the mutations that fall under star guard. We have 15 centers activated, and we are going to activate, um, couple of centers to make 15 cents according to the protocol, and we are on track for the enrollment, um, and the ba in 2027,
Thank you very much for the additional caller. And then one last one for me, and I'll hop back into the queue.
Um, I'd like to see if you guys could help us contextualize what a 27% lesion growth in GA might look like from a patient perspective, in terms of delaying loss of vision and preservation of function.
Take that sure, thank you and thanks for, you know, asking this question.
so, uh, as you know, uh, photo receptors, are critical, uh, to maintain the vision in these patients,
So, if you look at it from a translational perspective, like how this 27% in 6 months would translate in the long term, the way we look at, you know, other prior reports or products even, even after a year or 2 years, you see very limited reduction in growth.
So from that perspective, when I look at 27% reduction in religion growth, we see that it will, it will help these patients, you know, uh, basically prevent them losing their their visual function, okay, significantly. And and as you know, this is a modified gene therapy approach. So not only it is going to preserve the photo receptors but also it is going to enhance the function of the photo receptor, which eventually we believe that uh, we lead to the to the gain. In the functional Vision in which patients, okay what what we have noticed in the phase 1, patient like, you know, in terms of low eliminations visual acuity.
So, uh, in some way, I would say we would, we would see the better impact on these patients as this modifier gene therapy product, uh, you know, um, uh, uh, start making changes in the photo receptor function, in these patients, over course of time.
All right, thank you. I appreciate it. And once again, congrats on all the progress you've made.
Thank you.
Your next question comes from the line of Boris. Peeker from tyton Partners, your line is now live.
Great. Uh, thanks for, uh, taking my questions. Maybe I'll start.
400 in RP, I'm just, uh, when the dsmb convened to look at the interim data, was there a utility analysis of which there's just purely safety analysis to continue moving forward.
or any Adverse Events of special and Trust related to the investigational product reported
Great and maybe then if we move on to 410, um, can you talk about the potential interim update? Uh, maybe confirm that kind of estimated timing for that update, as well as what will be analyzed during that update.
Yes. Um, so in terms of our Q4 can, uh, Geographic atrophy, our model trial secondary to dry age micro degeneration, we are going to update, um, the interim analysis, um, data in the fourth quarter, we will be providing structural and functional outcomes um, for for that analysis.
And and boring, just to clarify, we just gave some data at 6 months.
and, and we have total subjects, including
The untreated control group consists of 31 patients, which represents a 31% reduction in lesion growth. The data looks promising compared to currently approved marketed products, which show much lower rates of lesion growth. The second thing is...
Obviously as Autumn stated before.
From the structural changes to functional change, such as an LLV or a regional equity, it takes a little longer. By the time we reach the fourth quarter, we'll have some data from the later time points, like 9 months, and maybe in some patients 12. This should really enlighten us on any changes in the functional benefit to the patients.
Gotcha, I made my last question is on the EMA or the European regulatory front, uh, when will we get some feedback to kind of get a sense of if the pivotal study sufficient to get approval there or not?
Um, so actually on RT, we already have it on Star guard. We should be able to get it, um, by fourth quarter this year.
No question for this Korean partnership. When will we see that $11 million recognized on the, um, income statement?
Uh, that's over the period certain period of time, which will extend up to into 27. So,
It's it's uh in different phases. So it's not at 1 point of time, okay. Got it.
Thanks for clarifying. Well, thank you very much for taking my questions.
You're welcome.
Your next question comes from the line of sa re cat from HC, Ren Wright, your line is live.
Thank you. Um, good morning chancre, uh,
a couple of quick questions from me, um, regarding, um, the Limelight trial, um,
You know, um, can you give us what the current enrollment percentage is? And, um, also, um, when would be, uh, when should we be expecting? Um, Topline data from this, from this study.
So, I'll take the, uh, question.
So we are, um, on track for, um, the enrollment and on track for our ba in 2026 as, uh, this is a blinded study. Uh, we will be releasing, um, just the periodic safety updates but um, the other data would be available after, um, the last patient. Last visit is done.
In 26 in 2026 or yeah.
Okay, thank you for that. Uh, Homer. Um, is there any additional, um, dsmb looks into this study or the next? Um, you know, uh, major data update is, is when you release that efficacy Topline, um, in the in, in early, 20, I mean, in 2026. Yes. So we will have another, um, safety, um, update, um, in the fourth quarter,
Okay, thanks for that. And then, um, Shankar on the author Helix, um,
I'm just trying to understand, you know, would you get any non diluting funding from from this? Uh, spin-off. Um, and, and also on the, on the financial side of things, you know, uh, with um, your current, um, funding uh, how do you plan to um, um, to bring an additional funds so that you can get all these
3 programs, um, to the completion.
I mean, obviously, we're working hard on potential business development opportunities partnership, and, uh, that has tremendous potential to bring in non daily funding. In addition to that, uh, as the Market opens up and we, we will be opportunistic, you know, as needed. Uh, we can also look into some equity and and so those are the things we always want to balance. You know, the shareholder dilution. We take that pretty seriously. So if there are ways we can, you know, minimize that with strategic Partnerships to non dilute funding, we'll try to maximize that as far as our to select is concerned, once the company is formed, um, after that, uh, the company can independently seek any potential government grants, uh, because there may be a need in that area. And and so, they're open to all that.
Thank you, thanks for taking my questions.
Thank you.
Your final question comes from the line of Daniel.
From aided.
Your line is a lot.
Yeah. Hey, good morning, guys. Uh, thanks for taking the question, and congrats on all the progress. I just have a couple from me. Um, first, and I'll keep it to 200. In your press release, you mentioned that you intend to complete the Phase 1 trial in the second half. Um, how would you describe patients' and physicians' interest in this program, given, um, a fairly large number of effective and approved options for these patients?
and when you for the initial data from the program,
Uh, so here. Daniel, um, this s. I, uh, so in terms of, okay, 200 just the progress. Uh, we are, um, successfully dozing in cohort, 3, um, right now and the data is expected, uh, towards later. This year, we would be, um, the safety and efficacy report on. Okay? 200, uh, novel by Logic Tombstone, and transferring the combin infusion protein in terms of the entrust from the investigators actually, it it is good because uh, not only from the safety perspective but also the Dual um, nature of Tombstone and transferring novel recomment infusion protein. And also how the responders and the rescue therapies are there. So this seems like a potential uh not only safe product in terms of intravital administration but also um to prevent the coral new vascularization.
And also uh, to look at the responders and um, you know, non-responders treatment uh in further trial. And also currently uh, the rescue treatment that are being given um with patients of macular edema who have got the approved therapies. So I think, okay, just to clarify further. Um, the currently, there is a significant portion of the population for non-responders, the current treatments.
That's where a 20,200 comes in. We think potentially this can treat both non-responders and responders.
Got it, got it, got it. Thank you. Um, another question on, um, okay, $400, with respect to that licensing agreement in Korea.
Um, Can can you tell us what the regulatory path um or approval path would look like uh for okay. 400 in Korea and um potentially in other Asian markets
Yeah.
Just as, uh, EMA didn't request. Um, I mean waved, a clinical trial. They're willing to take us data, the US clinical trial to give approval. Similarly, there are many markets. If you get, um, FDA or EMA approval, since it's a orphan drug, many countries, we can use our usfda approval to get approvals in other countries. So, Korea is the same path. Um, um, they potentially don't need any further, clinical trials.
And, uh, they can use our FDA approval to get approval to launch the product in Korea.
Similarly, we're looking at our markets, we don't have answers yet, including Japan. Uh, we're still working with the pmda and uh, when we get a definitive response from them, we will update the markets.
Okay. Got it, makes sense and 1 1, last question. In terms of your interactions, uh, with the FDA, how would you describe? Um, you know, the the conversations and in the past few months, um, and what changes, if any of the expected, the recent departure of Dr. Prasad from cber,
Put somebody in place of the super office, uh, in charge with all the changes and we're still getting pretty good response. Number 1, the stargard disease.
All the decisions they made working with us. Um, approving. Uh, our our IMD amendment in a record time, um, and and getting the design, everything lined up in the last 3 months and they're very supportive in addition, any questions we have, any of our programs from RP to stargardt, they're reacting. Very promptly.
So from all perspective, we're not seeing any impact on our programs.
Got it. Okay, all right. Thank you very much for taking my questions.
You're welcome.
This concludes the Q&A portion, I will now turn the call back over to Chairman CEO and co-founder Dr. Shankar luceri
Thank you. Everyone again. Um,
um,
Thank you, operator. Thank you everyone for joining today. The meaningful progress Ocugen is making across its normal modifier gene therapy platform, along with the notable leadership changes and significant external alliances, are evidence of a strong first half of 2025.
And we look forward to providing critical program updates and data in the coming months, have a great day.
This concludes the meeting. You may now disconnect.