Q4 2022 Ocugen Inc Earnings Call

Speaker 1: And that.

Speaker 1: I TR.

Speaker 2: to Ocugen's fourth quarter and full year 2022 financial results and business update call. Please note that this call is being recorded at this time. All participant lines are in a listen-only mode. Following the speaker's commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications.

Speaker 3: You may begin. Thank you. Joining me today are Audrey Linds, Chairman, CEO , and Co-Founder, Dr. Shankar Musanuri, who will provide a business update, and our Chief Accounting Officer and Senior Vice President of Finance, Jessica Crespo, who will provide more detail on our financial results. Earlier this morning, we issued a press release detailing business and operational highlights forwhich we're following in the event of the meeting.

Speaker 3: section of the Occupyton website for approximately 45 days. This presentation contains forward-looking statements within the meaning of the Private Security's litigation reform act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as, predict, believe, potential,

Speaker 3: proposed, continue, estimate, anticipate, expect, plan, intend, may, could, might, will, should or other words that convey uncertainty, a future event, or outcome to identify these forward-looking statements.

Speaker 3: Such statements are subject to numerous important factors, risks to an uncertainty and may cause actual events or results to differ materially from our current expectations. Investors should familiarize themselves with the company's firing for complete details.

Speaker 3: Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation.

Speaker 3: whether as a result of new information, future events, or otherwise after the date of this presentation. Finally, Occident 10-K covering 2022 will be filed soon after today's call. I will now turn the call to Dr. Musanuri.

Speaker 4: Thank you Tiffany, good morning and thank you all for joining us today. I'm excited to share with you the Significant Progress, Occasion made in 2022 and during the first months of 2023 to further advance our diversified pipeline.

Speaker 4: while continuously focusing on patients and pursuing courageous innovation. Fueled by our team's passion, dedication, and visionary mindset, we witnessed progress across all our clinical programs.

Speaker 4: date on our gene therapy program. In December 2022, we announced that our Q400, our investigation drug candidate for the treatment of Retinitis pigmentosa.

Speaker 4: And Libre congenital amrosis was granted expanded orphan drug designations by the US FDA, which supports the therapeutic potential of RQ400 to treat multiple inherited retinal diseases, the single product. RQ400 is symbolic of Apochagin's gene modifier approach that is based on nuclear hormone receptors.

Speaker 4: that regulate diverse physiological functions in the written up. Such as development, metabolism, cellular functions, and thereby establishes homeosteases to potentially restore written up health and function.

Speaker 4: that regulate diverse physiological functions in the written up. Such as development, metabolism, cellular functions, and thereby establishes homeosteases to potentially restore written up health and functions. In the US,

Speaker 4: RP and LCA affect 110,000 and 15,000 people respectively. And globally, these conditions affect approximately 1.6 million people. And at, despite its prevalence, RP and LCA patients have limited treatment options. As current approved, all in development gene therapies.

Speaker 4: focus on individual genes. RQ400 addresses shortcomings of current gene therapy approaches as a broad spectrum gene agnostic approach to genetically diverse and hereditary diseases.

Speaker 4: We have completed enrollment of RP patients in the Phase 12 trial for protocol and continued to enroll patients with LCA. We also established the high dose to be the maximum tolerable dose. We plan to start the Phase 3 clinical trial near the end of 2023.

Speaker 4: OQ200, our biologic product candidate, is a recombinant fusion protein consist of two human proteins, some statin and transferant, and is designed to treat severely site threatening diseases like diabetes, macular edema, diabetic retinopathy and vet AMD.

Speaker 4: of patient populations, experience, limited or no response to current treatments. Acute 200 works with a distinct mechanism for action compared to existing therapies and targets multiple causative pathways such as angiogenesis, oxidation and inflammation.

Speaker 4: and has the potential to offer better treatment to all patients. Yesterday, we submitted an investigational new drug application, IND, with the US Food and Drug Administration to initiate a Phase I clinical trial of OCT200 for treating diabetic, macular edema, DMI.

Speaker 4: This regulatory milestone fulfills the company's commitment to file the I&D for RQ200 within the first quarter of 2023.

Speaker 4: We have brought to further advance our Q200, one of our occasions founding uptomic programs. Another major highlight in 2022 that would like to note is the expansion of our pipeline into cell therapy with Neocard.

Speaker 4: It faced three-release region-grade cell therapy technology that combines the evolutionary advancement in bioengineering and cell processing to enhance the autologous cartilage repair process.

Speaker 4: We are developing near part specifically for the treatment of particular

Speaker 4: cartilage defects in the knee. Current therapies to treat cartilage damage in the knee are suboptimal with varying outcomes due to variable cellular responses. The current standard of care suffers from factors such as pain, reduce knee function, fail you to address cartilage damage, donor tissue availability and open surgery.

Speaker 4: In addition to receiving a regional rate to medicine advanced therapy designation from the FDA, we received a conference from the FDA on the confirmatory Phase III clinical trial design. We have already begun renovating our facility to accommodate CGMP manufacturing for clinical trials. And we are planning to initiate Phase III randomized control study in subjects with particular cartilage defects in the first half of 2024.

Speaker 4: Now, turning to our vaccine portfolio and continued efforts to significantly mitigate the spread of COVID-19. From a public health perspective, we actually monitor the medical need for a more durable vaccine, as more than a million cases of COVID-19 were diagnosed in the US over the last 30 days.

Speaker 4: The International Health Regulations Emergency Committee of the World Health Organization recently held a meeting in January this year to discuss the state of the COVID-19 pandemic which revealed that the global risk of COVID-19 and its ongoing transmission as high.

Speaker 4: This assessment was based on factors regarding circulating SARS-Co2 variants of concern, status of global vaccination and hybrid immunity, and the unexpected and relatively early seasonal return of the flu, which further encumbers already constrained healthcare systems.

Speaker 4: The data back this up, more than 5 million cases were diagnosed and nearly 40,000 people died worldwide in the last 28 days.

Speaker 4: Current COVID-19 vaccines are limited by a lack of durability and the inability to stop transmission.

Speaker 4: As part of our commitment to address current gaps and the fight against COVID-19, we are developing a novel, Nucosal vaccine platform that includes RQ500, a Bivalent COVID-19 in Herald vaccine.

Speaker 4: Acute Fight 10 is seasonal, quadrivalent flu inhaled the vaccine, and Acute Fight 20, a combination quadrivalent seasonal flu and Biivalent COVID-19 inhaled vaccine.

Speaker 4: The 500 series is based on a novel, chat platform designed to reduce transmission and protect against new variants. The Oculus 500 series is designed for annual boosters.

Speaker 4: For the 2020-23 flu season or 50% of the US population above 6 months of age, received a seasonal flu shot, representing a market size of more than 170 million doses.

Speaker 4: The occupier industry of vaccines and the development grants oxygen a distinct product candidate profile status that could significantly impact major global health obstacles and maximize opportunity to sow broader patient markets.

Speaker 4: Regarding your injectable COVID-19 vaccine, already in development, co-vaccine, we successfully completed enrollment for our co-vaccine Phase 2-3 Immunovaging and Broadening Clinical Trial in December , 2020, and reported top line data in January , 2023. This data showed that co-vaccine was well tolerated and demonstrated immunogenicity, with planned to present final data and analysis at mid-year.

Speaker 4: At the beginning of November 2022, we held Occasion's first R&D day. Since the company's inception, to showcase our dynamic pipeline and world-class team to investors, analysts, KWLs, and other key stakeholders of the company.

Speaker 4: During that meeting, we shared the long-term outlook for the company. On this call, I would like to spend some more time recapping our key priorities of the next 12 to 18 months. First, our gene therapy programs.

Speaker 4: where anticipating occup 400 preliminary efficacy data mid-year and plan to start the phase three clinical trial they are end of the year.

Speaker 4: For occuport 10, we are on track to submit INDs to the FDA in the second quarter of 2023 to initiate Phase 12 trials for dry A&D and sub-God disease. With occuport 10, we believe we have a potential one-time theoretical therapy with a single injection.

Speaker 4: dry AMD FX vision in 10 million people in the US and over 266 million people worldwide. Also targeting CVRI diseases, we look forward to the initiation of our up to 200 phase 1 clinical trial with the preliminary data anticipated in the fourth quarter of this year. We plan to complete the CGMP Facility Construction.

Speaker 4: for the manufacturing of Neocard in the fourth quarter in support of initiating a H3 clinical trial and the first half of 2024. For our inhaled vaccine cities, we are planning to file an I&D to initiate clinical trials in the fourth quarter of 2024.

Speaker 4: One overarching and imminent objective for Occasion is to identify synergies and partnership with organizations that can drive the development of our comprehensive pipeline. With that, I'm thrilled to, I'm thrilled about the recent appointment of a new Chief Business Officer, Kwon Wu.

Speaker 4: Who is the seasoned healthcare business executive with more than two decades of experience in business development, strategy and finance?

Speaker 4: We look forward to benefiting from the prospects of quanted leadership and together further evolve as a fully integrated patient centric biotech company.

Speaker 4: Our strategic initiative to identify partnership for our programs is also critical for our operational objective to console capital and extend runway as appropriate. With that, I will now turn the call over to our chief accounting officer.

Speaker 4: Senior Vice President of Finance, Jessica Frispo, to review our Q4 and 2022 financial metrics. Thank you, Shankar, and good morning, everyone. I will now provide a brief overview of our key financial results for the fourth quarter and full year 2022.

Speaker 3: Our research and development expenses for the quarter ended December 31, 2022, were $17.2 million compared to $7.1 million for the fourth quarter of 2021. For the full year ended December 31, 2022, research and development expense was $49.8 million compared to $35.1 million for the year ended.

Speaker 3: December 31, 2021, with the increase primarily driven by the advancement of our product candidates into clinical trials. General and administrative expenses for the fourth quarter ended December 31, 2022 were 6.9 million compared to $7.5 million for the fourth quarter of 2021. General and administrative expenses for the full year 2022 were 35.

Speaker 3: $18.6 million or $7.00 net loss per share for the fourth quarter of 2021.

Speaker 3: Full-year net loss was $81.4 million or $0.38 net loss per share compared to a net loss of $58.4 million for the full year of 2021 or $0.30 net loss per share.

Speaker 3: Our cash cash equivalents and investments totaled $90.9 million as of December 31st, 2022, compared to 95.1 million as of the year and December 31st, 2021. We expect that our cash cash equivalents and investment balance will enable us to fund operations into the first quarter of 2024.

Speaker 3: We're continuously exploring opportunities to increase our working capital and we'll be focused on seeking out partnerships and non-deluded funding as Shankar mentioned during his prayer remarks. That concludes my update for the quarter. Perfect you.

Speaker 2: Thanks, Deb. We'll now open the call for questions. Operator? At the time, if you'd like to ask a question, simply press star followed by the number one on your telephone keypad. Again, that is star one for any questions.

Speaker 5: Our first question will come from the line of you here with Mizzoulo securities. Please go ahead. Hi guys, thanks for taking my question. I was wondering if you could sort of speak a little bit about your, some more about the your COVID program, particularly with co-vaccine.

Speaker 5: Could you sort of just help us understand where how it fits currently, I guess, within the changing landscape where the FDA, I guess, are moving forward with recommendations for the by-veilant, as well as...

Speaker 5: you know, companies developing as you guys are also with the 200 series, you know, in combination with the flu vaccine. That's my first question. And I guess my second question is, on RQ400, could you sort of help us understand, you know, as you...

Speaker 5: What should we expect, I guess, from the Phase II read out in mid-year and how would that help you to move into Phase III in terms of, I guess, trial designs as well as, you know, what sort of primary endpoint you would propose to the FDA.

Speaker 4: And obviously, coaxing is a good vaccine. I mean, it has got solid data. It is given to a few hundred million people across the globe. The theorem will safely profile.

Speaker 4: And unfortunately in US, we needed to do more work to bridge with US demographic and population as started by FDA. So the first trial obviously included immunobrigeing trials, that's what we're completing. And the second one obviously we are anticipating a safety trial because we need to bridge immunobrige too.

Speaker 4: efficacy, the largest efficacy trial done over by partners in India. And the second step is of course the safety bridge. And as we stated earlier, you know, we needed to work with government agencies having more vaccine options is important in this, especially with the fairable safety profile. We conformed in our emerging trial.

Speaker 4: We didn't have any myocarditis, tricarditis, or thrombosis. Any of the adverse events, like you see with other vaccines. And again, consistently confirming the safety data generated by our partners elsewhere.

Speaker 4: So we believe in all this has a space provided.

Speaker 4: We do get some help and funding from the government. So that's where the coaxing is. I mean, as I stated before, there's more work to be done. Obviously, there's a need for multiple tools in the toolkit. The code is not done. It'll be there for many years. And we believe it will be a safety profile.

Speaker 4: which is built on a traditional vaccine platform such as polio and other vaccines. This could be a vaccine in a toolkit which could be beneficial for many patients or many subjects.

Speaker 4: Now, coming to I Q4 00, obviously the landscape is changing. That's why we are in our IQ 500 city, as you stated, with inhalation vaccine, the potential to control transmission and durability. There are two things which are issues to the current vaccines. And we believe the market is going to move into annual bolsters.

Speaker 4: As we stated, there's a large market size for flu. You know, 50% of Americans are taking flu shots every year. So ideally, if we can combine, you know, with our technology we have, which is unique, and chat platform, and designing novel flu, as well as COVID, and also, combination of vaccine will offer a long term as oxygen can contribute into that space.

Speaker 4: Now, for public and perspective. Now, changing gears coming to occupant requests, as we said to you, we're monitoring multiple observation and points as a part of this efficacy analysis.

Speaker 4: And what we are anticipating is we have multiple things we are looking at from structural perspective as well as functional endpoints. Our goal is to identify one end point. I mean ideally the same end point works for multiple mutations is good. But again the data is going to tell that.

Speaker 4: And so we have to wait until we get the data. Our goal is to identify a appropriate endpoint based on the data from this Facebook clinical trial and then propose that endpoint with FDA and eventually EMA and finalize our Facebook design and then move on to Facebook clinical trial. And your question about.

Speaker 4: The phase-trick linkle design, it will be similar to a product which got approved in the US several years ago in the ice space on the inter-pip product. So the design will be similar to that.

Speaker 2: Okay, thank you. Your next question comes from the line of Jennifer Kim with Cantor Fitzgerald. Please go ahead.

Speaker 6: Hi, good morning. Thanks so much for taking my questions. I have a few here. I guess the first one is Oki 200. I saw that

Speaker 6: You're going to get the phase one started out and we're going to see some initial data in the fourth quarter and I'm wondering What what are you looking for in that preliminary data and how should we think about the design of that trial? And then second, I think you mentioned that your runway gets you to the first quarter of 2024

Speaker 6: I'm wondering, how do you think about, I think previously it was run away into the end of this year. And I'm wondering, I guess, how should we think about how you're managing cash at your balancing all these programs and how you were able to extend that? Thanks.

Speaker 6: How do you think about, I think previously it was run way into the end of this year. I'm wondering, I guess, how should we think about how you're managing cash at your balancing all these programs and how you were able to extend that? Thanks. Yes.

Speaker 4: So, after 200, as we announced, it's a dose escalation study in a small population, or goal is to look at the range of doses, and so we can pick one before we go into phase two.

Speaker 4: So, as a part of that, I mean, obviously we're going to look at CST, Central Soft Field thickness as other companies have looked at DME space, and specifically, once again, the primary objective of any case, one-plus-plus-plus trial is safety, as a part of the dose escalation to finalize the dose.

Speaker 4: and we'll be looking at observational endpoints, and one of them is ESD. And now the second question, extending runway into Q1, I'll just answer that. Hi, Jennifer. So in terms of the extension of our runway into the first quarter, we did utilize our ATM a bit, so that has helped us extend.

Speaker 3: our runway into Q1 of 2024. But as Shankar mentioned, I mean, we will need to raise capital in order to progress on all of our programs and we're exploring many different options and including our focus on non-deludive funding as we stated. Maybe if I could...

Speaker 6: Squeeze one more question with the two additional IND filings in the second quarter of this year. Should we think about you?

Speaker 6: Are initiation of those clinical programs? Could that come this year? Or are you thinking more in, I guess, early next year? Thanks.

Speaker 4: So, for that as we stated, yeah, yeah, yeah, again, I just wanted to confirm. The Fortin and Fortin is T. We call it, we separate it out because Fortin targets dry age related macular degeneration.

Speaker 4: Specifically, we'll be targeting late stage patients in geographic atrophy, and that's targeted for filing and second quarter of this year. Similarly, RQ410ST targeting orphan disease, TARGARS, is also targeted for second quarter of this year, IED filing.

Speaker 4: Now, actually, we stated a New York article in the thread with Start-Post-F of next year. We are constructing a CGNP manufacturing facility, and that will be complete by the end of this year. Okay, and can you give us a little more clarity on how much is left on the ATM as of today?

Speaker 3: So, as part of our, I would say, general corporate housekeeping, we've canceled the ATM in connection with converting our automatic shelves into a regular shelf, so you'll see those filings come through today. So we'll put the ATM back up when and if it's appropriate for the appropriate amount. So at this point, it's been canceled.

Speaker 5: Okay, but can you tell us how much of that ATM was actually used overall from the end? Yeah, under that ATM we've filled approximately 14 million in terms of gross proceeds. Okay.

Speaker 3: And we've netted about 13.6 million. Yes, I thank you very much, that is all.

Speaker 2: You're next question comes from the line of Robert LeVolier with Noble Capital Markets. Please go ahead.

Speaker 7: Good morning. Thank you for laying out some of the milestones for the co-vaccine programs. Could you give any time frames for what you would mention the midyear top line or conclusions and full data?

Speaker 7: Could you give some of the timeframes for the Phase 3 and some of the other program starts and milestones?

Speaker 4: Good morning. Are you specifically referring to co-vaccine or other programs?

Speaker 4: Good morning. Are you specifically different to co-vaccine or other programs?

Speaker 4: Are you referring to co-vaccine and other programs? Yes.

Speaker 4: Okay, yeah, as I stated before, the coaxene, we are finishing of the current study and we'll have the final data analysis expecting a media of this year.

Speaker 4: And as I stated before, we may need to do a safety clinical trial. We're still waiting for a VA to respond. That comments on our safety protocol.

Speaker 4: And once we get that, obviously, we, as we stated, we're seeking government funds to conduct that clinical trial. So as far as other phase-trick-linked rights are concerned, there are two more we talked about during this earnings call. One of them is our signature Q400 Modifer Gene Therapy Plac Phone.

Speaker 4: So, where we have completed recruitment in our magnetic pigment dose of portion of the phase 1, 2, clinical trial. And so based on data read, we're anticipating mid-year on efficacy signal. And we're going to get into planning to get into phase 3 by the end of this year.

Speaker 4: personalized medicine. So we are building our own manufacturing facilities, renovating the existing facilities to convert them into CGMP manufacturing for cell therapy production. And we're anticipating that construction will be complete with end of the year. And that will allow us to initiate pastry clinical trial and the first step of...

Speaker 2: Okay, thank you very much. Our next question will come from the line of Sean Lee with HC Wainwright. Please go ahead.

Speaker 5: Good morning, guys. Just a quick question for me. So for the proposed Neoclass study, have you finalized on the endpoint in the study design yet?

Speaker 4: Yeah, I think the study design as we agreed with the BA includes counterplastic control and that's different than prior control they used.

Speaker 4: in the prior studies. The second thing is the end points will include pain and function. The third thing is the end points will include pain and function.

Speaker 7: Compared to the previous phase III that the histogenics ran with Neocard, what would be the key differences?

Speaker 4: The key differences are, as they had a micro fracture at the control.

Speaker 4: The key differences are they had a micro fracture at the control, which is...

Speaker 4: So what we did is we looked into the current standard of care which is conruplasty. So based on that, we actually wanted to use conruplasty and they agreed with that.

Speaker 4: The second thing is we're also going to restrict the lesion size to 3 cm square and really focus on that so that it's not very broad like they did before.

So I think with these changes we anticipate, you know, we'll have better prospects of, you know, relatively easily recruiting patients. That's important because control plastic is standard of care, not micro-factor. And that's really important. That's an important change.

And also focusing on that specific range for a leader in size is very important for us. Great. Thanks for that. One last panel follow up on that. For the new manufacturing facility that you're building, would that be only for supporting materials with a Phase 3 or could you scale that up to potential commercial later as well?

Good questions from. When you build this personalized medicine, initial therapies, this is like a scale out, not scale up. So these are very small, you know.

We can call them bio reactors, we call them tissue engineering process units. So these things are very small, sweet, so this can be used for commercial manufacturing tests. I see, yeah, thanks for that. That's all questions I have.

Your next question will come from the line of the Neil Godlin with Shardon. Please go ahead. Hey, good morning guys. Thank you for taking the question and congrats on all the progress. Just a couple of follow-up one on the cash run away in terms of bundling. Are you expecting to fund the OC500 Series vaccine programs internally or looks?

24. Thanks.

Okay, good morning. The first one is related to Occupyhundred Series, Emulation Vaccines.

So, as I stated before, we are funding the development of those vaccines and taking it to the clinic.

So we're also in parallel working with various government agencies because of the need we believe you know we're trying to secure funding from that. So we do need their support to move them into the clinic. But the development part and preclinical studies we have budgeted for it and we're going to complete that.

And the second step question you had, an RQ410 and an RQ410SD, targeting RIMD and stronger disease, those INDs are scheduled to be filed, planning in second quarter of this year. And as soon as we get a positive nod from FDA, we will start those impatience.

This year. Okay, thank you very much. This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO Dr. Shankar Mucineary. Thank you, operator. I'd like to conclude the call with some additional remarks. I think it's clear that we are staying true to our mission.

as an integrated patient-centric biotechnology company. The targets unmet medical needs. We believe we are in a position of strength and we are poised to execute our goals with our pipeline or the course of 2023. We look forward to keeping you updated on our programs throughout the year. Thanks for your time today and have a great week. Thanks everyone, have a great day. Bye.

Thank you all for joining today's meeting. You may now disconnect.

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