Good morning, and welcome to the Ocugen conference call. At this time, all participants are in a listen-only mode. A question and answer session will follow the presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. 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. You may begin.
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 a financial update. Earlier this morning, we issued a press release, including a business update and third-quarter 2021 financial results. 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, we need to advise you that this call will contain forward-looking statements.
Such forward-looking statements are subject to risks and uncertainties that could cause actual events or 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 the FDA will grant us emergency use authorization for COVAXIN in ages 2- 18, and when we will be able to submit a biologics license application for COVAXIN to the FDA, and whether and when we will receive regulatory approvals for authorizations for COVAXIN in the U.S. 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 and accompanying slide presentation, as well as the risk factors included in our filings with the SEC. Note that we intend to file a Form 10-Q with the SEC today. I will now turn the call over to Ocugen's Chairman and CEO, Dr. Shankar Musunuri.
Thank you, Ken. Good morning, everyone, and thank you for joining, and we hope you and your families are safe and well. Today, we're here to review a rapid succession of milestones from this company I feel privileged to lead. It reminds me of the famous Margaret Mead quote, "Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it's the only thing that ever has." These words remind me that just one year ago, Ocugen was less than 20 individuals. We all had a vision to bring new therapies that could tackle serious diseases and bring new options for people wanting a choice.
We have completed three quarters of 2021, and our Ocugen family has grown significantly, committed to bringing COVAXIN BBV152, our COVID-19 vaccine candidate, to the United States and Canada, along with our lead candidate for blindness diseases, OCU400, which is a part of our modifier gene therapy platform. Today's update is a result of their hard work, along with the efforts of our global partners, Bharat Biotech and CanSinoBio. Thank you all for your contributions. This slide outlines the major events that transpired over the recent months, including the third quarter of 2021. First, we want to congratulate our partners at Bharat Biotech for securing an emergency use listing for COVAXIN by the World Health Organization. This is exciting news. That's a tremendous accomplishment and a critical validator for broadening the global portfolio of COVID-19 vaccines and is noticed by the regulatory authorities around the world.
Closer to home, Ocugen took significant steps to progress COVAXIN BBV152 with the U.S. Food and Drug Administration. Last week, we submitted COVAXIN for emergency use authorization with the FDA for use among those aged 2 to 18 years. We believe there is a significant unmet need within this age group, knowing that there is a lack of choice for different vaccines within the U.S. market. Particularly in ages 2 to 5, there is currently no approved option. We believe the data, as we top-lined on our Friday call, November fifth, press release, make a compelling efficacy and safety case to the agency, and we look forward to furthering our discussions with them.
In late October, we filed an investigational new drug application to support the initiation of a phase III immunobridging study between the U.S. population and the results of Bharat Biotech's phase III clinical trial involving nearly 25,800 participants. This phase III bridging trial is being conducted in support of an upcoming BLA. Our trial has designation OCU-002 and will involve a few hundred subjects. The primary objective will be to compare neutralizing titers between the U.S.-based participants who get two doses of COVAXIN to those who got two doses in the phase III efficacy trial in India. Secondary objectives include measuring the immunogenicity of two doses of COVAXIN over time in those who are of age between 18 and 65, in those over the age of 65, as well as determining its immuno-broadening effect, including in those who previously received an mRNA vaccine.
Such a broadening effect could include antibody responses against multiple antigens, such as spike and nucleocapsid proteins. We're also preparing for the possibility of conducting a safety bridging trial if required. Finally, we are hearing from many people about their interest in participating in this clinical trial if approved. We are very appreciative of their passion and ask that they stay tuned for further developments. To the north, our engagement with Health Canada continues, and responses to deficiencies noted are being prepared. As a reminder, we applied for approval under the interim order, and our application was automatically transferred to a new drug submission process. I now want to update you on our progress with our modifier gene therapy program.
I'm pleased to announce that yesterday, Ocugen filed an investigational new drug application for OCU400, our lead candidate in the modifier gene therapy platform for the treatment of retinitis pigmentosa resulting from genetic mutations in NR2E3 and rhodopsin. This is a proposed safety and dose-finding phase I/II clinical trial involving a small number of patients. We have already successfully completed manufacturing at commercial scale, at 200-liter scale to support clinical studies. As part of the clinical trial, patients will be observed post-dose for at least 12 months. From there, OCU400 could move into a phase III clinical trial, evaluating its ability to address multiple inherited retinal disease mutations. This is the beginning of a new journey, one started by our partner, Dr. Neena Haider from Harvard Medical School, and we look forward to sharing the progress of our trial, if approved, throughout 2022.
Following close behind OCU400 is our next candidate, OCU410. IND-enabling preclinical studies have started to support a future phase I/II clinical trial. OCU410 is designed to address dry age-related macular degeneration. It's the most prevalent chronic form of AMD, accounting for approximately 90% of total AMD cases, and is characterized by slow progressive dysfunction of the retinal pigment epithelium, photoreceptor loss, and retinal degeneration. With about 150 million people suffering from dry AMD around the world and no treatment options available, there is significant unmet medical need. Preclinical data recently presented at the Dry AMD Therapeutic Development Summit in October suggest that OCU410 plays a role in the genes associated with how dry AMD develops over time, and we will continue to explore this area throughout 2022.
We are pleased to share that in order to support the manufacturing of OCU410, we have expanded our arrangement with CanSinoBio to be our partner responsible for chemistry, manufacturing, and controls development and manufacturing. They will now support the CMC development and manufacturing for both the OCU400 and OCU410 programs. Our agreement with CanSinoBio was amended in September to add this program. Rounding out our ocular portfolio, OCU200, our transferrin-tumstatin fusion protein, is still progressing well. We are on track with our preclinical activities to explore further how it can help those with diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. With OCU400 moving into clinic and OCU410 and OCU200 continuing their IND-enabling studies, our focus on ocular therapies remains very strong.
This indeed has been a busy quarter, with much going on to advance COVAXIN and our ocular portfolio, and there is much more ahead. I will now turn the call over to Sanjay to provide our third quarter 2021 financial update. Sanjay?
Thank you, Shankar, and good morning, everyone. I will now provide an overview of key results for the third quarter of this year. Our research and development expenses for the quarter ended September 30, 2021, were $6.3 million, compared to $1.5 million for the third quarter ended September 30, 2020. The increase is primarily driven by COVAXIN development and regulatory activities, OCU400 preclinical activities, as well as employee-related expenses due to an increase in R&D headcount. General and administrative expenses for the quarter ended September 30, 2021, were $4.5 million, compared to $1.7 million for the third quarter ended September 30, 2020. Our increase in general and administrative expenses relates to increased infrastructure costs to support the growth of the organization.
Net loss was $10.8 million or $0.05 net loss per share for the quarter ended September 30, 2021, compared to a net loss of $10.5 million or $0.07 net loss per share for the previous year's quarter ended September 30, 2020, which included a $7 million write-off of an asset held for sale. We ended the quarter with cash equivalents and restricted cash totaling $107.5 million as of September 30, 2021, compared to $24.2 million as of December 31, 2020. That concludes my update. Back to you, Ken.
Thank you, Sanjay. With that, we will open the call for questions. Operator?
Your first question comes from the line of Kenay Naka with Chardan.
Good morning. A couple of questions for you.
Morning, Ken.
Morning, Ken. Yeah, the first question relates to timing, you know, for the adult population in the U.S. As you commence the bridging study, and hopefully that will set you up to file, maybe the middle of next year. You know, should we think about possible approval in Q1 of 2023? Is that kind of an appropriate way to look at the timing?
Yeah. If you follow the path of regulatory pathway other companies have followed, we believe agency will also consider a fast track for us. We were planning to initiate that at the right timing. Based on that, we're planning to file the BLA in the second half of next year. Sometime in 2023 first half, if we do get a fast track designation, that seems to be reasonable.
Okay, great. Under your agreement with Bharat, you know, under a scenario where the U.S. would like to purchase your vaccine to help distribute OUS again because of its many attractive characteristics. Is Bharat precluded from selling vaccine to the U.S. for that purpose, and you exclusively have the right to sell the vaccine to the U.S. government for any purposes?
Yes. We have rights in U.S. and Canada. Any procurement from the U.S. government, yes, we have to go through Ocugen.
Okay, great. Then for the most recent EUA filing for the pediatric, you know, given that you're running the bridging study for adults, you know, how do we think about whether you'll need to run a bridging study for pediatrics as well before you could get any type of approval?
This is again we have filed emergency use authorization. As we outlined in the two-five age group, currently there are no authorized vaccines or approved vaccines, and there is a significant unmet medical need. Those things will be considered when you apply for emergency use authorization. That's the reason, you know, based on compelling data we have in the pediatric population, we decided that the American kids do need a choice. We thought in the two to 18 age group, that's the data we have from our pediatric population, from our partners, we filed EUA. It is purely based on unmet medical need.
Okay. Just two more questions. One, for Canada, you know, how should we think about the potential timing there to get a potential approval, you know, having filed in Q3, you know, with their full or at least anticipating a full review again, as you know, first half 2023 or 2022, I'm sorry, or you know, maybe mid-2022, the appropriate timing for that potential approval?
I think the process for Canada, again, as we stated, there is no equivalent of emergency use authorization that's called interim order. It expired. The file got into NDS, which is New Drug Submission process. Typically it does take some time, and I cannot comment on actual regulatory time, exact date. However, we are closely working with Health Canada review process and addressing any questions come up on the way.
Okay. Congrats on the filing for OCU400. Nice to see those programs will be entering the clinic, you know, around the end of the year. That's great. That's all I have.
Thank you. Thank you, Ken.
Your next question is from Zegbeh Jallah with Roth Capital.
Good morning. Thanks for taking my question and congrats on all the regulatory updates. I think a couple of quick questions for me. The first is just on the pediatric EUA. I was just curious if you're gonna hear any kind of-
Update on an acceptance of the EUA before a decision is made, or your next feedback from the FDA will be about whether or not it is approved, or should you expect something within a week or two from now?
Good morning, Zegbeh. Again, when the review process, you know, they accept it for review, so the process is ongoing. When we get updates, we'll provide updates to the market. At this time, I can't comment anymore.
Okay, you're not expecting to get a letter about an acceptance or anything like that prior to an update on whether or not the EUA is approved.
No. Typically, the EUA goes through the process based on the process other three companies have done. Typically, it goes to AdCom before it gets any nod. That's a typical process. We're working with the agency on that.
The next one here, I know you said, you know, for Health Canada, you can't really comment on the timing or anything, but I think you also said that you haven't gotten any questions or comments from them. Is that correct?
Can you repeat the question, Zegbeh? Sorry.
Yeah. For Health Canada, I was just saying that, so far after your submission, you haven't gotten back any questions or follow-up, or you haven't had to submit additional information or anything like that. You're just waiting to hear back from them.
Zegbeh, this is a normal course of business review process of any NDAs or any submissions you do to any regulatory agencies. The companies, you know, as a part of the active process, we respond back and forth. That's again normal course of business. That's what we are dealing with Canada too.
Oh, thanks. Okay. You have had some back and forth with them. The next one here is just about the potential to expand your agreement with Bharat. I know I think you have the opportunity to do so for more territories, but I was just wondering what it is that you need to see before you make the decision to do so.
Zegbeh, just to make sure I understood your question correctly, you're asking about our opportunity to expand our territories with Bharat for,
Mm-hmm.
for other regions and what would be the
Potential
Catalyst for that? Is that right? Yeah. Zegbeh, again, you know, those options are always open. Again, we're really focusing on the U.S. and Canada at this stage. Again, we'll keep those options open with our partners.
Okay. Moving on to OCU400, really excited to see that IND get submitted. I was just curious as to, you know, any updates about the study design or what we might see, any kind of clinical updates or anything from that program?
Yeah. Once IND gets accepted and goes through the process, Zegbeh, we will be launching the information into clinicaltrials.gov, and I mean, that'll help all the stakeholders and potential patients and everybody else. Again, this is going to be a small trial, just as other orphan designation trials to the retinal space.
Okay. I know you expanded the CanSino agreement to include OCU400. I was just wondering where you are in terms of development. Is CanSino doing anything right now for OCU400? Are you doing, you know, IND-enabling studies? Where in the process are you?
Yes. CanSino has started working on the development just as they did for OCU400. We do have a roadmap from the FDA on OCU410. Currently, the team is in the process of executing those plans.
Perfect. Thanks for the updates.
Thank you, Zegbeh Jallah.
Your next question is from Robert LeBoyer with Noble Capital.
Good morning, Robert.
Hi.
Good morning.
Hi. Congratulations on the results and all the progress you've made in the last quarter.
Thank you, Robert.
My question has to do with the booster shots and the durability of COVAXIN and whether there's data that might show any need or lack of need to have a booster shot 6 months or 12 months after vaccination. I also was wondering if there was any data to show or any discussion of the idea using COVAXIN following one of the messenger RNA vaccines to give broader protection or any benefits that one might have there.
Robert, first question is the longevity effect. Again, our partners are generating some data. When the data is available, it will be published. However, the data to date clearly showed one thing scientifically people have to discuss more about, cellular responses, the T-cell response, which creates a memory, and that's how you get the long protection with any vaccine. Our partners have shown in the published articles in the credible journals, medical journals that COVAXIN elicits very strong cellular responses. That means you have potential memory and you should get long protection. Again, when they generate more data, we're going to continually share that with the markets. The second question is, people who have been vaccinated with mRNA vaccines, are we going to generate the data?
The answer is yes. Our clinical trial, which we did file the IND, the immunobridging trial doesn't exclude anybody who took mRNA vaccines. In fact, it does include people who have taken mRNA vaccines with the caveat that it has to be at least, you know, six months or earlier. Those people will be included in our clinical trial, and we are going to generate the data in those subjects.
Great. Thank you very much.
Thank you, Robert.
Your next question is from Sean Lee with H.C. Wainwright.
Good morning, guys, and thanks for taking my questions. My first question.
Good morning.
This is on the supply agreement with Biovet. Does that cover potential commercial supply if your EUA for pediatric population is approved? How much supply would you have access to? Also in terms of the tech transfer to Jubilant, what's the progress on and expected timeline on that?
Yes. Upon the EUA, our supply agreement does include adequate doses, Sean, and our partners have significantly increased their capacity this year. They don't have many restrictions for export. We'll get adequate supply, whatever is needed, post EUA in the U.S. As far as technology transfer is concerned at Jubilant HollisterStier, the program is going well and we are anticipating completion of you know, establishing that, including process validations in the first half of next year so that we can switch the supply. Initial part of the supply does include even we receive product from Bharat Biotech or partners, it will include U.S. packaging and the testing from the U.S. release site which have been established.
Once the tech transfer is completed next year, would you need additional CMC validations from the FDA to show that the clinical batch and the commercial batch are the same?
Yeah, that's a normal part of the process. When you add additional sites, you always have to show the comparability, and that will be the part of the process.
All right. My next question is on the OCU410. You showed some pretty good preclinical results from that program back in October. I was wondering what's the development timeline on that, and when can we expect that to go into the clinic?
Is it related to 410?
410, yes.
Yeah. No. OCU410, yes, that one, again, we have a roadmap from FDA. We have to follow a typical process as other gene therapy product like we did for OCU400. The two steps which are really important to file the IND for that, one is developing and manufacturing the product. Second part is preclinical toxicology studies as we agreed with the FDA. Again, this is a big program. It goes to you know large populations unlike OCU400, which targets rare diseases. Therefore, the workload for this is little. It'll take a little longer from preclinical tox perspective, everything else. Therefore, we're simply working on it.
At least we have a roadmap from FDA, and we're going to execute it. Our goal is to put that in the clinic in the next 12-18 months.
Great. That's all I have. Thanks for the additional clarity.
Thank you, Sean.
At this time, there are no additional questions. I would like to turn it back over to Ken for closing remarks.
All right. Thank you very much, and thanks everybody for taking the time to join this call this morning. We look forward to providing further updates in the coming months, and we thank you for your time.
Thank you, ladies and gentlemen. This concludes today's conference call. You may now disconnect your lines at this time. Thank you for your participation, and have a wonderful day.