Ocugen, Inc. (OCGN) Q4 2021 Earnings Call Transcript

Ocugen, Inc. (OCGN -5.60%)
Q4 2021 Earnings Call
Feb 25, 2022, 8:30 a.m. ET

Contents:

• Prepared Remarks
• Questions and Answers
• Call Participants

Prepared Remarks:

Operator

Good morning, and welcome to the Ocugen conference call. [Operator instructions] Please note this conference is being recorded. I will now turn the conference over to Ken Inchausti, head of investor relations and communications for Ocugen. Sir, you may begin.

Ken Inchausti -- Head of Investor Relations and Communications

Thank you, operator. I'd like to welcome you to our conference call. With me today are Ocugen's chairman, CEO, and co-founder, Dr. Shankar Musunuri, who will provide a business update; and our chief financial officer and head of corporate development, Sanjay Subramanian, who will provide the financial update.

Earlier this morning, we issued a press release, including a business update and fourth quarter and full-year financial results for 2021. We encourage listeners to review the press release, which is available on our website at www.ocugen.com. This call is also being recorded, and a replay, along with accompanying slide presentation, will be available on the investor section of the Ocugen website for approximately 45 days. As always, I need to advise you that this call will contain certain forward-looking statements.

Such forward-looking statements are subject to risk and uncertainty that could cause actual results to differ materially from expectations, including, among other things, the uncertainties inherent in research and development of our product candidates, risks to our business related to the ongoing COVID-19 pandemic, uncertainty regarding whether and when we will be able to submit a biologic -- biologics license application for Covaxin to the FDA, and whether and when we will receive regulatory approvals for Covaxin in the United States or Canada. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. You should read carefully the risks and uncertainties described in today's press release, as well as the risk factors included in our filings with the SEC. Note that we will intend to file our Form 10-K on March 1st.

I will now turn the call over to Ocugen's chairman and CEO, Dr. Shankar Musunuri.

Shankar Musunuri -- Chairman and Chief Executive Officer

Thank you, Ken. Good morning, everyone, and thank you for joining, and we hope you and your families are safe and well. We are here today to review with you some of the most recent activities and events that transpired in the fourth quarter of 2021 and provide some perspective on the full year. 2021 was a transformational time for Ocugen, especially as we successfully progressed two different assets within our portfolio.

Simultaneously, we expanded and diversified our team to match our growing clinical development, commercial, and R&D needs. This was the year in which Ocugen doubled down on what I called courageous innovation. That phrase speaks to our purpose and commitment to development with the purpose and grit, searching for new solutions to enduring challenges, and working to bring a new COVID vaccine into U.S. and Canada to initiating a clinical trial for a game-changing Emory to gene therapy that could potentially treat multiple genetic mutations with a single product.

I'm proud of this team that has succeeded with our business partners to position us for so much more in 2022. This slide highlights some of the critical milestones for Covaxin in the later part of the year and into 2022. But first, it's important to understand the context for the progress we made. We have seen that the science has evolved and our nations' strategies have changed.

Today, the focus is on how to achieve ongoing protection and closing the gaps among populations who remain unvaccinated. In just one year, so much has changed. At Ocugen, our work for Covaxin had evolved over the months to meet this moment. That led to our IND submission in late October and our EUA submissions in early November.

That work has paid off. Last week, we have received clearance from the FDA to initiate a clinical trial to support a biological licensing application, or BLA. Our clinical program will be a Phase 2/3 immuno-bridging and broadening study. We will provide more details over the coming months as the study progresses.

On the pediatric EUA front, I'm pleased to say that there are discussions involving the data we have submitted, including Bharat Biotech's immunogenicity data published in a preprint server in late December 2021, our own omicron research showing effectiveness against this variant, and a database of more than 36 million teens vaccinated with Covaxin in India. With more than 70% of Americans looking for options based on our latest Harris Poll, we know the need is great. We will continue educating the FDA to give Covaxin a fair shot by having it evaluated by its independent vaccine advisory committee known as VRBPAC. We recognize that many of you have been asking for ongoing regulatory updates and asking why the review can't move faster.

We hear you, and we would note two things. The first is that I'm pleased with the time frame we took to how the clinical hold lifted. That took less than three months. The second is that getting to the critical juncture required significant confidential dialog with the FDA.

Therefore, making public those details would have been detrimental to the review process. So once the review process starts, very little can be disclosed until there is clear guidance from the agency. In Canada, we have supplied a comprehensive set of responses to help Canada from follow-up questions contained in what is known as a notice of deficiency. Remember that Covaxin is now under the new drug submission review, which will potentially lead us to full approval.

We are following up with Health Canada to determine next steps. And speaking of Canada, there is nothing -- there is growing interest from local and provincial government officials about our interest in the manufacturing site run by Liminal BioSciences outside of Belleville, Ontario. Positive discussions with leaders have centered on the value of reestablishing a manufacturing and R&D hub within the region and for supporting that nation's contribution to innovation and public health. One final note about Covaxin.

We're pleased to share with you that we have made good progress toward establishing manufacturing capability at our partner Jubilant HollisterStier in Spokane, Washington. This has involved designing a robust manufacturing process, establishing a means to transport active ingredient and straight adjuvant from India to the U.S., selecting primary packaging components, and establishing quality control strategies that will provide additional assurance that the final product meets rigorous FDA standards and can be safely administered to people. The technology transfer is going well, and we expect to complete qualification manufacturing runs at Jubilant by the middle of this year. Now let's turn to our modifier gene therapy program.

As you know, we submitted in November of 2021 an investigational new drug application for a Phase 1/2 clinical trial for OCU400, our lead candidate in the modifier gene therapy platform for the treatment of retinitis pigmentosa resulting from genetic mutations NR2E3 and rhodopsin. Within 30 days, the FDA accepted our application, and our dose-ranging clinical trial is now recruiting. Details of the study are available on the National Institutes of Health website, clinicaltrials.gov. Our next candidate, OCU410, has IND-enabling studies underway to support a future Phase 1/2 clinical trial.

And our novel biologic, OCU200, a transferrin-tumstatin fusion protein that has potential to help those with diabetic macular edema, diabetic retinopathy, and age-related macular degeneration is progressing well with IND-enabling activities. We are on track to initiate toxicology studies in nonhuman primates. So 2022 is off to a fast start with clinical trials for two product candidates, with the data readouts expected second half of this year, ongoing discussions with the FDA surrounding our pediatric U.S. submission for Covaxin, and continued progress on the remaining assets in our portfolio.

I would like to thank our partners whose efforts are key to our progress. I'm very pleased with the progress we have made and know that we provide updates throughout the year. I will now turn the call over to Sanjay to provide our fourth quarter and full year 2021 financial update. Sanjay?

Sanjay Subramanian -- Chief Financial Officer and Head of Corporate Development

Thank you, Shankar, and good morning, everyone. I will now provide an overview of key financial results for the fourth quarter and full year of 2021. Our research and development expenses for the quarter ended December 31, 2021, were $7.1 million, compared to $1.6 million for the fourth quarter ended December 31, 2020. For the full year, research and development expenses for the year ended December 31, 2021, were $35.1 million, compared to $6.4 million for the year ended December 31, 2020.

R&D expenses for 2021 included a $15 million upfront payment to Bharat Biotech for the additional rate and license to Covaxin in Canada. The remaining increase is attributed to the continued investment in the development activities for Covaxin and our ocular portfolio. General and administrative expenses for the quarter ended December 31, 2021, were $7.5 million, compared to $2.2 million for the fourth quarter ended December 31, 2020. For the full year ended December 31, 2021, G&A costs were $22.9 million, compared to $8 million for the year ended December 31, 2020.

Our increase in G&A expenses relates to increased infrastructure costs to support the growth of the organization. Net loss was approximately $14.6 million, or $0.07 net loss per share for the quarter ended December 31, 2021, compared to a net loss of approximately $3.8 million or $0.02 net loss per share for the previous years' quarter ended December 31, 2020. For the year ended December 31, 2020, Ocugen reported a net loss of approximately $58.4 million, or $0.30 net loss per share, compared to net loss of approximately $34.4 million, or $0.31 net loss per share for the year ended December 31, 2020, which included the in-process R&D expense of $7 million related to the reduction of the carrying value of an asset that was previously reported as held for sale. Our cash, cash equivalents, and restricted cash totaled $95.1 million as of December 31, 2021, compared to $24.2 million as of December 31, 2020.

Earlier this week, we raised net proceeds of $15 million before estimated offering expenses. This further strengthens our balance sheet and extends our runway. That concludes my update. Back to you, Ken.

Ken Inchausti -- Head of Investor Relations and Communications

Thanks, Sanjay. And with that, we will open up the call for questions. Operator?

Questions & Answers:

Operator

Thank you. [Operator instructions] Our first question comes from the line of Swayampakula Ramakanth from H.C. Wainwright. Your line is open.

You may now go ahead.

Shankar Musunuri -- Chairman and Chief Executive Officer

Hi, RK. Good morning, RK.

Operator

We have another question from the line of Zegbeh Jallah from ROTH Capital Partners. Your line is open. Please go ahead.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Good morning, and thanks for taking my questions, and congrats on the progress. I think the first question for me here is if you can just comment on the details around what was required to lift the clinical hold. And you, RK.

Shankar Musunuri -- Chairman and Chief Executive Officer

Zegbeh, good morning. Thanks for the question. Clinical hold, when you submit an IND, typically FDA takes their own time. FDA is extremely busy too.

There's so many applications on the vaccine site. So the typical questions, if they have any clarifications they need and those are the questions they asked and we promptly submitted our responses as I mentioned on our call, and we're able to wrap up this whole process within three months, which is good considering the workload FDA has and everything else going on.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thanks. And can you just clarify? So was it adding additional data that was needed or just a reanalysis? I'm just trying to get a sense of that to kind of predict perhaps the outcome of that pediatric EUA.

Shankar Musunuri -- Chairman and Chief Executive Officer

No. I think -- I mean, let me clarify on the IND hold. Additional data was not -- there's clarification questions mostly. We didn't have to generate any additional data.

Coming to -- so the second question, are you asking about pediatric EUA?

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Yeah. I was just trying to cut and gauge what might be required for the pediatric EUA based on the IND, so if you can just comment on that as well, that would be helpful to the extent that you can.

Shankar Musunuri -- Chairman and Chief Executive Officer

Yeah. So the IND is for long-term biological license and application process, and we're trying to breach the data from the U.S. demographic with the data generated in a large clinical trial in India. So that's the Phase 2/3 immuno-bridging and broadening study we're embarking on.

And that's for BLA. Now the EUA was submitted, emergency use authorization, for pediatric population ages 2 to 18 based on the data generated by our partners based on the immuno-bridging trial, which bridges data to large-scale Phase 3 trial run in adults. So that's under active review by FDA.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thanks, Shankar. And then as a follow-up, we got this question from investors, and they just kind of wanted some clarity on whether the FDA committed to providing an approval on the BLA if the bridging study comes out positive, meaning that you will not need any additional studies beyond the bridging study to support the BLA approval.

Shankar Musunuri -- Chairman and Chief Executive Officer

Yeah. For the BLA approval, Zegbeh, there are two things I think of 21 CFR. One is immuno-bridging. Typically, you do that because you cannot keep on -- that will bridge with a large-scale safety and efficacy trial, typically you do.

That's what other companies have done in U.S. too. They apply for pediatric population. You bring to pediatric to large-scale efficacy and safety file.

Similarly, in this case, for the BLA, we are bridging on the immunogenicity side. That's kind of bridging it to efficacy file. Typically, you also have to conduct a safety study in the U.S. demographic based on 21 CFR, and that bridges the safety of the population in the U.S.

with the data collected from elsewhere. So in addition to this immuno-bridging trial, we foresee doing a safety trial in the U.S. population. So those are the two clinical trials required typically for BLA submission.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Perfect. Thanks, Shankar. And then just the last here. I know this is perhaps still being determined, but was just wondering if there was agreed-upon sample size for the bridging study or for the safety study.

And then where are you regarding next steps that need to be completed prior to the initiation of the bridging study, for example, manufacturing specimens, etc.? And I will squeeze in my last one here, and maybe Sanjay can just comment now with a recent public offering. Can you provide your updated cash runway?

Shankar Musunuri -- Chairman and Chief Executive Officer

OK. So let me take the first few questions. The population for bridging study, typically you need a few hundred subjects for immunogenicity trial. You don't need large population.

So that bridges, you know, neutralizing antibodies. And then there are other antibodies we're going to monitor. In our case, we'll be looking at not only spike. We'll also be looking at nucleocapsid protein and others because this vaccine does provide a broadening immune response, unlike spike-based vaccines.

And the second question you asked is on safety clinical trial. Typically, that requires, you know, more than what we need for immunogenicity bridge, and those discussions are ongoing with the FDA. And once we have the final agreement with them on the number, we'll be providing update to the market. Sanjay, go ahead.

Sanjay Subramanian -- Chief Financial Officer and Head of Corporate Development

Sure. Zegbeh, thanks for the question. Regarding cash, yeah, we're pleased that we did the financing earlier this week. It strengthens our balance sheet even further.

The additional capital will help with the investment in our programs and Covaxin as well as all the ocular programs. As you know, we have the EUA for pediatric use of Covaxin is pending at this point in time. So there's potential depending upon the -- how that progresses, the potential for near-term revenues. So that could obviously change our cash runway.

But if we exclude that or any benefit from that and we have a -- without that, we -- with the capital that we have raised, we have sufficient capital to go well into the first quarter of next year. It gives us ample runway for the investment in our programs and all the trials that we are contemplating to support the programs.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thank you, Sanjay. And I will get back into the queue 'cause I do have questions about OCU400.

Shankar Musunuri -- Chairman and Chief Executive Officer

Sure. Thank you.

Sanjay Subramanian -- Chief Financial Officer and Head of Corporate Development

Thank you, Zegbeh.

Operator

[Operator instructions] We have a follow-up question from Zegbeh Jallah from ROTH Capital Partners. Your line is open. Please go ahead.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

I guess I had the honor of asking all the questions today. So just kind of moving on here. I think the next question as it relates to the Covaxin opportunity is just having you comment, I guess, on the size of the market opportunity as you think about perhaps being kind of a late player to the U.S. market.

And then maybe you can comment a bit on your survey findings as well and perhaps, you know, who you serve it and some of the details around that.

Shankar Musunuri -- Chairman and Chief Executive Officer

Yeah. So the U.S. market, Zegbeh, it's still evolving, right? But we still have the pandemic, which we believe will continue for the next few years. So the -- currently, obviously, the pediatric, that's the reason we applied for pediatric EUA because it's a significant unmet medical need.

In the under age five group, there are no vaccines available. From 5 to 18 age group, there's only one vaccine option available. And the vaccination rates -- the kids who got two doses from 5 to 11 age is about 25%. So to increase the vaccination rates, which are extremely important to prevent hospitalization and death, and in the pediatric populations, majority of the parents are looking for options.

And that's the reason there is a great need for pediatric vaccines, which are safe and effective, and we have provided a significant amount of dataset to agency and requesting them to push for the outcome meeting. Second thing is -- so the second opportunity is booster market. As you know, this pandemic will continue, and people who got the vaccines, eventually, they'll be looking for boosters. People who are unvaccinated, they're also looking for options.

And providing an option such as ours with a broadening immune response and the product, which is built on nontraditional platform technology such as polio vaccine, people can relate to. So some of the vaccine hesitancy folks who are still there or not unvaccinated to date, and they can also get an option such as over vaccines. So there is a need for options and providing a vaccine with solid efficacy and safety and also which is built on a traditional platform will help, we believe, significant value because there are people out there that can really provide options to increase the vaccination rates. And in addition to that, the U.S.

government is doing a good job through the vaccine diplomacy. They're producing vaccines from U.S. companies and donating for global use. Unless global population is vaccinated everywhere of a certain extent, this pandemic is not going to be under control.

Therefore, it's extremely important to having multiple vaccines in the toolkit. Especially the vaccine -- our vaccine has expected shelf life of two years at refrigerated temperature, six months at room temperature. So this vaccine will be an ideal vaccine for stockpiling and for vaccine diplomacy for global distribution.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thanks, Shankar. That's really helpful in terms of how you're thinking you might have an advantage. But I guess what one would ask then is about how you're thinking about perhaps being able to enroll the study within the U.S. and maybe some strategies around how you plan to position that, you know, to kind of help with the enrollment of the study and perhaps even some comments around the timing of how long do you think that could take?

Shankar Musunuri -- Chairman and Chief Executive Officer

So the -- I think the clinical trial information will be posted shortly on clinicaltrials.gov on the Covaxin trial design, and the study will allow not only new population, but it allows subjects who's already vaccinated, but there's a pause. I think they have to have a minimum six months of gap after the last vaccine doses, and they can be enrolled in the study. So we have designed it very creatively, and the details will be posted at clinicaltrials.gov. After this process, Zegbeh, if you have any further questions, we'll be happy to address them.

And again, there's a great need for options in this country. And, I mean, we get a lot of calls and questions from people across the country, and we believe we'll be able to recruit very quickly in this trial.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thanks, Shankar. And then just quickly for OCU400 program we're very interested in. Just kind of want to get a sense of when you think data from that study could become available and how much detail do you think you'll provide at the time of the next update?

Shankar Musunuri -- Chairman and Chief Executive Officer

So OCU400 study is recruiting now. If you go to the clinicaltrials.gov, details are given. So this is a Phase 1/2 trial of two mutations. And in each mutation, we are planning to recruit nine patients each.

And it's a dose escalation. And so we will be able to recruit those patients we have identified in multiple sites. And the process is ongoing, and we're very excited about, you know, completing the recruitment this year and continue to provide updates in the second half of this year. Safety is the main goal for this study.

In addition to that, we'll be looking at efficacy through observation endpoints. So we expect to provide updates in the second half.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thank you. And then the key thing about this program is really the potential to be able to use it in multiple inherited retinal diseases, and so I was just wondering at what point are we able -- are we going to be able to get a sense of its broad efficacy potential? And then can you then comment on what you think the market opportunity could be for this program?

Shankar Musunuri -- Chairman and Chief Executive Officer

Yeah. Absolutely, Zegbeh. Great question. So that's a good thing about this program.

I mean, not only we are going into NR2E3 the master gene mutation itself in our Phase 1/2 clinical trial. We also are looking into rhodopsin mutation, which is a modified approach. So that actually is the -- that concept, once we show activity in those patients, it's working, that opens up the door for entire IRD population potentially. You're talking about targeting 150 mutation within our feet.

Similar to that, the CEP290 mutation, which we are envisioning to initiate in those patients too within the next one year. And that covers LCA, which is Leber congenital amaurosis, an [Inaudible] mutations. So our goal is to have at least three mutations covered, which can represent RP and LCA covering about 175 mutations. Two million patients globally struggle with these diseases.

So that's a enormous opportunity to treat a lot of these patients globally and significant population within the U.S. and E.U. U.S. has about 100,000 to 200,000 patients covering between RP and LCA.

So it's a huge -- big-sized market. And, moreover, it's a -- it would be a great help for these patients to rescue them who are in desperate need. As I mentioned in my past conference calls, many of these patients do become legally blind by the time they're in mid-40s. So it's really important.

There is a sense of urgency needed to rescue these patients. We hope we can be there for them.

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Thanks, Shankar, and congrats on the progress.

Shankar Musunuri -- Chairman and Chief Executive Officer

Thank you.

Sanjay Subramanian -- Chief Financial Officer and Head of Corporate Development

Thanks, Zegbeh.

Operator

Our next question comes from the line of Brian Cheng from Cantor. Your line is open. Please go ahead.

Brian Cheng -- Cantor Fitzgerald -- Analyst

Hey, guys. Thanks for taking my questions, and congrats on the progress. So I have a three-part question for Covaxin, and I hope you can answer them. Can you comment on the manufacturing supply and capacity for Covaxin? And just from the capacity ramp-up perspective, I'm just curious, how we should think about the capacity assuming that you will have the pediatric approval near term? And lastly, when you think about the broad vaccine market in North America, do you expect to look for partners to speed up the production and even reaching out to additional unvaccinated subject? And then I have a follow-up.

Thanks.

Shankar Musunuri -- Chairman and Chief Executive Officer

So, thank you Brian, thank you. So let me start with a capacity question, is that EUA is authorized. Currently, as I mentioned on the call, we are qualifying Jubilant HollisterStier that plant is progressing really well, by midyear it will be completing qualification runs. However, we have a plan, our partners in India, as you know, this is inactivated vaccine, it needs to excel facilities, they have actually scaled up to billion doses a year capacity.

And what we have done in the interim, we established the packaging site in the U.S. and the release testing site, so that we can release the product to our standards through Ocugen in the U.S. And those types are already established. So initial supply will immediately come from India, from our partners, which gets tested in the U.S.

and packaged in the U.S. to release to the public. While the tech transfer is ongoing, and we are planning to build up to 100 million does capacity at Jubilant for good product. However, as I mentioned to you before, there are three aspects of supply we are envisioning; one, the pediatric use, the second part is bolster use in the U.S.

market, the third part, U.S. vaccine global diplomacy. So I mean government purchasing doses for distribution abroad. So we believe with our partners significant capacity and the capacity we're building in the U.S., with our CMOs, we'll be able to supply whatever the needs are.

And second question you have was related to reaching some other states and also the people who are unvaccinated. Currently, the distribution, as you know, is completely done by the government. They purchased the doses, the procurement is 100% done by government, and that they take care of the distribution for now. And they distribute the states and they're using their own network.

So in future, obviously, that may change, if at all, this pandemic ever moves into endemic, and becomes a annual boasters and others are predicting, and therefore time, you know, when it gets to prioritization, then we have some time to work on those details. But for now, you know, all the vaccines are procured by the government and distributed the government. And obviously, we'll be working with some states and other governments, if there is a need, you know, in certain states for this vaccine, because an option many subjects are looking for, we'll be happy to have them up.

Brian Cheng -- Cantor Fitzgerald -- Analyst

And then on partnership, do you think that you will need one eventually to kind of ramp up on the outreach or even ramp up the capacity? You're actually thinking about the product approval to the adult population?

Shankar Musunuri -- Chairman and Chief Executive Officer

Yes. I mean, all those options are no, we'd be opportunistic. I mean, our goal is to maximize, you know, supply of this vaccine as needed. But we'll be exploring all those options.

Brian Cheng -- Cantor Fitzgerald -- Analyst

OK. Thanks, Shankar. And then maybe just one on OCU400. I'm curious if you can talk about or remind us about the market size opportunity with the mutations that you are initially targeting.

And, you know, with regards to the Phase 1/2 study that you're running, can you talk about what will you need to see before expanding to other mutation types?

Shankar Musunuri -- Chairman and Chief Executive Officer

Yes. Typically, I mean, this is a standard clinical trials with the gene therapy in my space to look into rare diseases. Typical, I mean, our trial follows exactly what others have done. It's a Phase 1/2 trial.

The primary objective is safety. And you have to monitor these patients for a year on a -- if it's three months, you have to check for safety. And for every mutation, you look into the clinical trial, you have multiple observational endpoints because then from those observational endpoints, once the study is done after one year, you pick the observation point and make one of them as a primary endpoint into the Phase 3. So that means in this case, Phase 3 primary endpoint, by the time we go to Phase 3 maybe different than rhodopsin.

So, as I mentioned before, rhodopsin truly follows modified approach. So if we show the signal in rhodopsin, it's working the way it's supposed to work, that opens up the door for many, many mutations. I mean, obviously, you know, we also have to be cognizant, is a rare diseases. And therefore, we will methodically do the clinical trials in few mutations, so to cover part of the RP, and cover LCA.

So that, just as I mentioned before, although this decline, you know, cover those 100,000 limitations between RP and LCA. So the market size, you can look into populations, and US, between RP and LCA is 100,000 to 200,000 patients. And globally, it's about 2 million covering those 170 limitations. Currently, there's only one approved product for RPE65-mutantation that I believe has less than 1,000 patients in the US.

So there's a significant unmet medical need, and a significant opportunity. And for the gene therapy pricing, Brian, I think there are a couple of products in the market. And you can take a look at it. And I think other companies are getting there, and they're pricing based on similar models.

Brian Cheng -- Cantor Fitzgerald -- Analyst

And what do you need to see just curious, what will you need to see in terms of efficacy, before you expand into other mutation types, because I noticed that one other slide you're planning to also expand into such an idea as well. So just curious, what kind of bar should which investors expect in terms of perhaps sequential improvement before you expand to other investigated mutation type?

Shankar Musunuri -- Chairman and Chief Executive Officer

Yeah. So on CEP290, Brian, we wanted to start with -- for vaccine, we do have data on CEP290 on the preclinical and in other models, it's the same product, remember that so we don't have to repeat any top study. And so, it's a timing issue. So we're going to actually introduce that into the clinic.

Since the agency has allowed us to go into production. We're confident they'll allow us to go into CEP290. We're also in discussions actually with the EMA. And we do have broad RPA and LCA, indications for the orphan drug designations in the EU.

Therefore, we're also in touch with the EMA. And our goal is to at least complete the Phase 1/2 clinical trials for these three mutations in the US, while we are in communications with EMA, then eventually, when we go into clinical trials, after one year, what you're asking is, what do you want to see, I mean, obviously, CEP290, we don't believe we'll have any issues getting into the clinic with that. I mean, obviously, we'll be monitoring the rescue, disease progression from the -- gene therapy, the single dose and see how it's protecting people who are dose. And based on the data and the disease progression and rescue, based on those endpoints were monitoring, we pick the primary endpoint and go into Phase 3.

And once we and during this process, obviously, we believe coding to four articles is broad. And one for LCA, hopefully, should allow us for potentially broad RPA and LCA indications. So obviously, we'll be collecting a lot of data in Phase 1/2, and carefully moving into cautiously interface three for all three mutations. That's the plan together between US and EU.

So we'll mark the programs that the US and EU that's the plan, eventually, for Phase 3 clinical trials. Typically the Phase 1 for one year, we hope, the Phase 3 will continue to be in the timeframe, because you do have to catch the signal and also show, based on the primary endpoint, adequate duration during the clinical trial.

Brian Cheng -- Cantor Fitzgerald -- Analyst

With that said, look forward to your next update. Thank you, Shankar.

Shankar Musunuri -- Chairman and Chief Executive Officer

Yes. Welcome.

Sanjay Subramanian -- Chief Financial Officer and Head of Corporate Development

Thank you, Brian.

Shankar Musunuri -- Chairman and Chief Executive Officer

Thank you.

Operator

And there are no further questions at this time. Ken, you may continue for closing remarks.

Ken Inchausti -- Head of Investor Relations and Communications

Great. Thank you so much. And we want to thank everybody, who took the time to listen in on the call on both Covaxin and our modified gene therapy program, and we look forward to providing updates in the coming months. Thank you very much and have a great weekend.

Bye.

Operator

[Operator signoff]

Duration: 44 minutes

Call participants:

Ken Inchausti -- Head of Investor Relations and Communications

Shankar Musunuri -- Chairman and Chief Executive Officer

Sanjay Subramanian -- Chief Financial Officer and Head of Corporate Development

Zegbeh Jallah -- ROTH Capital Partners -- Analyst

Brian Cheng -- Cantor Fitzgerald -- Analyst

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