Q2 2023 Ocugen Inc Earnings Call
Speaker 1: Good morning and welcome to AUC QGIN's second quarter, 2023, Financial Results and Business Update. Please note that this call is being recorded at this time. All participant lines are in Listen Only mode.
Following the speaker's commentary, there will be a question and answer session. I will now turn to call over to Tiffany Hamilton, Occupy Jins Head of Corporate Communications. You may begin.
Thank you, operator. Thank you for joining me today.
Our Oxtance Chairman, CEO and co-founder, Dr. Shankar Musinari, who provided business and financial updates. And Dr. Arun Gupadiye, our financial scientific officer, head of research, development and medical, who is also on the call to answer questions during the Q&A. Yesterday afternoon, we issued a press release, detailing business and operational highlights for the second quarter of 2023. We encourage listeners to review the press release, which is available on our website at occigen.com. This call is being recorded and a replay with the accompanying slide presentation will be available on the investor section of the Occigen website for approximately 45 days. This presentation contains forward looking statements within the meaning of the private security litigation reform act of 1995, which are subject to risk and uncertainties. We may, in some cases, use terms such as predicts, believe, potential, proposed, continuous estimates, anticipates, expects, plans, intends, may, could, might, will, should, or other words, they convey uncertainty, a future event or outcomes to identify these forward looking statements. That statements include but are not limited to.
statements regarding our clinical development activities, and related anticipated timelines. Such statements are subject to numerous important risk factors, uncertainties, and 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 Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors, and 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. Finally, Occigent's quarterly report on Form 10Q covering the second quarter of 2023 has been filed. I will now turn the call to Dr. Musanuri. Thank you, Tiffany. Good morning and thank you all for joining us today. The second quarter of 2023 marked a period of continued.
progress toward our regulatory and clinical milestones, which we are delighted dedicated to advancing through end of the year. It is strategic focus on our novel modifier gene therapy and biologic based ophthalmic programs. We expect to begin those in patients across these black forms by the end of this year.
We are on track to initiate the RQ400 Phase III adult trial near the end of 2023-early 2024, subject to outcome of the ongoing Phase I-II trial and discussions with FDA on proposed Phase III trial plan.
We also anticipate a clinical study results update for RQ400 this quarter.
Investigational new drug applications were cleared by FDA for I Q4 10 and I Q4 10 SD for geographic atrophy and stargarde disease respectively. We plan to initiate Phase 12 trials by the end of 2023.
We're also planning to initiate the phase 3 clinical trial for our regenerative cell therapy product candidate, Neocot, in the second half of 2024. This would mean Ocogen would have late stage programs in gene and cell therapies in 2024.
In an effort to conserve our working capital and advance our patient-centric agenda to develop a novel inhaled mucosal vaccine platform, we have submitted multiple proposals to obtain non-dilutive government funding on having discussions with pertinent agencies.
to secure their support for our RQ500 vaccine series. Our first-in-class modifier gene therapy to treat multiple inherited retinal diseases remains unmatched industry-wide. This unique gene-agnostic approach has the potential to address retinal diseases caused by mutations in multiple genes with one product.
Our goal is to build on the innovation of gene therapy and expand its potential to treat a wider population of patients suffering from a host of rare retinal diseases that single gene replacement therapies are unable to address.
In the second quarter, we were honored to present in detail the Mechanism of Action and scientific basis for our modified gene therapy platform to preeminent researchers and medical professionals in attendance at the Association for Research and Vision and Ophthalmology.
and bio-international conferences. As we advance our clinical agenda, we'll continue to identify and secure opportunities to educate stakeholders on the differentiation and potential benefits of this innovative approach to gene therapy.
In April , we announced encouraging and compelling positive preliminary safety and efficacy results from our OCCU400 Phase 1-2 multi-center open-label dose ranging clinical trial in patients afflicted with RP.
We believe the preliminary findings from the study support the potential for our modified gene therapy to be a viable alternative to traditional treatments to the increasing population of patients suffering from these diseases.
Enrollment is ongoing for all defined subjects in the study, adults with LCA, and children between ages of 6 to 17.
Pending positive feedback from the FDA, we aim to initiate our Phase III adult clinical trial at the end of this year or early 2024. We continue to execute our comprehensive strategy to develop OCCU400 and bring it to market by 2026 with the goal of providing desperately needed treatment options for the patient.
for the esteemed 125,000 patients estimated in the US alone that suffer from RP and LCA.
In parallel, we will continue progressing our other modified gene therapy programs to address additional uptal conditions.
We believe that upon successful realization of these goals, Ocogen will have built a vast commercial footprint that may hold significant upside for our shareholders.
and most importantly, meet a critical medical need for patients. Dry-age-related macular degeneration is one of the most prevalent neurodegenerative eye diseases.
affecting approximately 10 million people in the US and nearly 266 million people worldwide.
Dry AMD results in irreversible loss of sight among elderly populations, leading to a lack of functional independence that severely impacts quality of life.
A variety of biotechnology companies, small and large, are working to develop therapies for dry AMD. However, we believe our OCCU410 candidate can offer a less burdensome option for our patients. With OCCU410, the
We are again investigating the potential for our novel modified gene therapy to provide a one-time treatment option that targets all four hallmark conditions of dry AMD, including lipid metabolism, inflammation, oxidative stress, and complement activation.
The current standoff gear only targets the complement factor, requires multiple injections per year and has reported side effects.
We're excited to initiate the phase one, two clinical trial this year because of the significant global unmet medical need.
Moving on to Ocu410ST, we're extremely pleased to receive orphan drug designation from the FDA to address ABCA4 associated retinal diseases such as Stargardt disease, RP19, and cone rod dystrophy, for which there are currently no treatment options. Ocu410ST is a novel modified gene therapy.
delivery of the RAR-related orphan receptor A.
Nuclear hormone receptors are master gene regulators that help maintain homeostasis by regulating diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks.
We believe that by harnessing the power of nuclear hormone receptors, we can develop one-time treatments that can modulate cell activity disrupted by disease-causing gene mutations.
Now, turning to our efforts to develop a series of next generation inhalation vaccines, for which the company intends to submit an IND application in 2024 pending government funding.
in multiple preclinical trials.
Mucosal vaccines have demonstrated vaccine-induced high neutralization titer and effector responses.
In here, mucosal vaccines represent a distinct product candidate profile that could help remedy major global health challenges and maximize our opportunity to serve a broader cross-section of patients.
through a less invasive delivery mechanism with the potential for superior durability when compared with the current intramuscular administration.
Clinical studies using a similar vector of inhaled administration have shown mucosal antibodies, systemic antibodies, and durable immune response up to one year with one-fifth of the dose compared to
traditional intramuscular vaccines.
Greater ease of administration presents the potential for improved vaccination compliance and wider adoption, particularly among traditionally underserved populations and throughout the developing world.
Current COVID-19 vaccines are limited by a lack of durability and marginal ability to prevent transmission.
As a part of our commitment to address barriers to widespread vaccination to protect against COVID-19,
We are developing this inhaled vaccine platform that includes OQ500, a bivalent COVID-19 vaccine, OQ510, a seasonal quadrivalent flu inhaled vaccine, and OQ520, a combination quadrivalent seasonal flu and bivalent COVID-19 inhaled vaccine.
The RQ500 vaccine series is based on a novel chat platform designed to reduce transmission and protect against new variants with potential durability up to one year.
To optimize resources across our diverse and critically needed development programs and maintain shareholder value, our team has been engaging with public health officials and federal government agencies to pursue non-dilutive funding to support the development of our OCCU 500 vaccine series.
We maintain an ongoing dialogue with respective agencies and anticipate receiving further information on the status of our funding requests later this year. Earlier this year, the FDA notified us that they were putting a hold on our RQ200 program and requested additional information related to chemistry, manufacturing, and controls. We are working with the FDA to release the hold and expect the Phase 1 trial to be initiated in Q4, 2023. We believe RQ200 works.
with a distinct mechanism of action compared to existing therapies for the treatment of diabetic macular edema and targets multiple causative pathways such as angiogenesis, oxidation and inflammation as potential to offer better treatment to all patients.
Neocot is our phase 3 ready regenerative cell therapy technology that combines novel
advancement in bioengineering, and self-processing to enhance the autologous cartilage repair process. Manufacturing facility construction for Neo-Cart is on target to be completed by the end of 2023 as planned. A company plans to initiate the phase three trial.
in subjects with articular cartilage defect in the second half of 2024. We are highly dedicated to completing our stated objectives with the strategies we believe will enable O2 to reach several value-enhancing milestones and are planning to file BLAs across all first-in-class platform technologies.
gene therapies, cell therapies, and vaccines in the next three to five years. I will now provide an overview of the key financial results for second quarter 2023.
Our research and development expenses for the quarter ended June 30, 2023 for $14.2 million compared to $9 million for the second quarter of 2022. This included a non-recurring non-cash expense of $4.4 million.
as a result of the impairment of the short-term asset for the advanced payment for the supply of Covaxin as well as the associated loss on the disposal of related fixed assets.
General administrative expenses for the quarter ended June 30, 2023 for 9.6 million compared to 10.6 million for the second quarter of 2022.
10 cents, point 10 dollars net loss per share for the quarter ended June 30, 2023, compared to a net loss of approximately 19.5 million or nine cent net loss per share for the second quarter of 2022. Our cash, cash equivalents and investments total 70.6 million as of June 30, 2023, compared to 90.9 million as of December 31, 2022.
In May, we closed a public offering of 30 million shares of common stock for gross proceeds of 16.5 million. Net proceeds from the offering are being used for general corporate purposes, capital expenditures, working capital, general and administrative expenses, and R&D.
We are continuously exploring opportunities to increase our working capital and will be focused on seeking out corporate partnerships for gene therapies and non-dilutive funding for vaccines.
That concludes my update for the quarter. Tiffany, back to you.
Thank you Shankar. We will now open the call for questions. Operator.
The floor is now open for your questions. To ask a question at this time, please press star 1 on your telephone keypad. If at any point you'd like to withdraw from the queue, please press star 1 again.
We'll now take a moment to compile our roster.
Our first question comes from the line of Jennifer Kim from Cantor Fitzgerald. Please go ahead.
Hi. Good morning. Thank you for taking my questions. I have two. The first is, as you're thinking about cash burn going forward and you're seeking non-dilutive funding opportunities, excluding the one-time impairment expense, is this quarter a good basis as we think about quarterly burn?
And then my second question is on the OCCI-400 program. Can you remind us what you're looking for in that updated data this quarter as sort of the go-no-go for the Phase 3 adult trial? Thanks.
Yeah, Jennifer, good morning. Let me address the first question, then Arun is going to take the second one. Yes, there's a one time impairment charge. There's also a non-cash stock comp charge of 2.6 million. And if you add those two, the cash comes out to be 15.9 million total.
I let Aaron address the other question on our key 400 program. Thank you. Thanks Jennifer. So, yeah, you were right. I think our this quarter update on OK 400 clinical.
Phase 1 to 2 data will guide us, you know, about our Phase 3 study.
That's the data will be used as a basis for going over decision for. Okay, and can you remind us what you're looking for in that data?
So, primarily we are looking at the functional improvement in the patients treated with OQ400 and the focus is going to be the RP patient.
Okay, and then in your discussions with the FDA later on...
For the phase three trial, is that going to focus on our patients or are you also considering inclusion of LCA patients? Thank you. So, to begin with, we'll start with the RPP percent and as we collect the data for LCA patients. Then later we include LCA, but to begin with, yeah, we are planning to go with adult RPE.
All right, thanks for taking my questions. Thank you.
Our next question comes from the line of William Ramkanth from H.C. Wainwright. Please go ahead. It is very difficult for the audience to answer the question.
Thank you. This is RK from HPSA. So a couple of quick questions on the 400 program and then maybe one on the new card. On the 400, so in terms of your discussions with the FDA, is that the...
Thanks RK so we have not initiated our discussion with FDA yet.
So once we complete the data analysis, only then we are going to reach out to FDA.
we complete the data analysis, only then we are going to reach out to FDA. But that is planned for.
this quarter, you know, are related to data update and followed by reaching out to FDA.
Thank you for that. And then on the 4.10 program, you know, in terms of now that you have already been cleared by the FDA, what else needs to get done before you can initiate the Phase 1, 2 study? Just we need to get the site ready and...
in this study both GA as well as Star Guard this year.
So it is more like getting ready with the clinical site.
Got it. Got it. And then on the on the new card program if
Just trying to understand, if the facility just gets completed by the end of 23,
You know you.
What else needs to get done in terms of commissioning the plant and getting the clinical material ready?
to start your program on the Phase III study by second half. Is it just that or is it, you know, you still have to design the protocol and just try and understand what all, because we've been talking about this program for almost a year and a half now.
end of this year. It's on target. And then as you know, there's a GMP facility. It takes a few months for getting the qualification done.
and then they'll be ready to produce Neocot in that facility.
In the interim, obviously, the team is going to prepare CMC and clinical sections, and they'll continue to update those so that they're ready for submission next year.
before they start the clinical trial. And we do have RMAT designation right. I just wanted to remind regenerative medicine advanced therapy designation with FDA. So when we have any questions in the interim, we can always reach out to them and get clarifications.
they start the clinical trial. And we do have RMAT designation right. I just wanted to remind regenerative medicine advanced therapy designation with FDA. So when we have any questions in the interim, we can always reach out to them and get clarifications. Perfect.
Thanks for taking the questions. Our next question comes from the line of Robert LaBoyer from Noble Capital Markets. Please go ahead.
Good morning everyone. My question has to do with the upcoming data presentation.
And I was wondering if you could disclose whether the data to be presented will update the previous data on all patients or whether it will just be in new patients that haven't been reported and wondering if there are any endpoints.
that you could share with us at this point. Thank you. So yeah, definitely we'll be providing detailed update when we kind of present this data to the market. But to address your first part of the question, whether it is going to include the percent to be presented in our previous, you know.
point. Thank you. So, yeah, definitely will be providing detailed, you know, update when we kind of know present this data to the market. But to add this to your first part of the question, whether it is going to include the percent to be presented in our previous, you know.
disclosure yes so we will include those subjects as well as some new subjects which has completed additional follow up visits. So it will be a combination of both.
Great. And you had mentioned corporate partnerships for the gene therapy.
you discuss any type of arrangements
research and development or just marketing or any objectives to the business development activities.
I mean, Robert, this is a, yeah, it's a loaded question. Obviously, we'll be open to when you seek partnerships at this stage as a biotech, big pharma established with the infrastructure and everything else. Obviously, your post target is going to be commercial.
This is, as you know, complex science is involved in these clinical trials. Obviously, when you're going into phase three, once we have the data out, we'll obviously work very hard with any potential partners. And obviously, as you know, if they are interested in commercial development, they would be interested in phase three program too.
So I think we'll keep our options open, whatever can maximize our value for Ocogen, as well as, you know, make sure we have ability to provide market access to patients.
desperately need this part.
this part.
The final question comes from the line of Dale.
Gautaland from Chardin. Please go ahead.
Hey, good morning guys. Thank you for taking that question. Got a couple. One for OCU410 programs. Just wanted to ask strategically how do you see positioned one term, whether it's viewed as a stand alone approach or in combination with anything else.
and the patient population that you'll be targeting. And the second question is for the Inhaled Vaccine Series. Can you comment on your interactions to date regarding the funding and particularly if you can comment on the interest in this program given that there are several other approved options? Thank you. Thank you. So I'll take the first question.
And by like, you know, very nature of this disease, you know, being multi factorial in nature, the various causes which lead to this disease. So our product has potential to target all those pathways which are linked to di-MD pathogenesis. So we believe that this could be a differentiated product and and has potential to to offer a better, you know, a clinical benefit compared to what what we have right now. And then good morning. The second question related to government funding. Again, we have been working with various agencies and we'll provide enough.
So what would be ideal option going into the future? I think there is definitely a need for mucosal vaccines. The scientific community agrees on that. And also that will provide systemic as well as mucosal immunity. So you can actually potentially prevent at the target of viral entry into mucosal system. The second thing is durability. I mean, we believe and scientific community believes that COVID vaccinations are breezy for people early on.
In order to gain the compliance with public, you cannot keep on vaccinating every three months. That's why people get vaccine fatigue. So you need to move into annual vaccination, such as flu, so compliance will go up. So that's the intent. I think, as we stated, there are X-U.S. trials with inhalation vaccine using a similar technology.
sure durability after one year, that's really important. So there are two things, controlling transmission and durability are very important for next-gen vaccines. And we believe our platform technology inhalation vaccines for COVID and flu, they can provide that.
Thank you so much. This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO Dr. Shankar Muhsenuri.
Thank you, operator.
In closing, I'd like to recognize the entire team for their resilient efforts to advance our patient-centric mission. To our shareholders and partners, thank you for your ongoing trust and support. We are already well into the second half of 2023, and the steadfast in our commitment to transparency informed decision-making based on sound scientific principles and a tireless work ethic dedicated to excellence.
in all phases of research, development, and clinical testing. We remain confident that we'll be able to fulfill our mission of developing novel therapies with innovative discovery to bring to market effective treatments for patients suffering from a range of conditions that currently lack treatment options. We look forward to sharing more details on our progress in the coming months.