Q3 2023 Ocugen Inc Earnings Call

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Okay.

Good morning, and welcome to Aki, just third party retrenchments, if we financial results and business update please.

Please note that this call is being recorded at this time.

All participants are in a listen only mode.

Well I don't distinguish commentary there will be a question and answer session.

I will now turn the call over to Tiffany habits, and Okay. Just head of corporate Communications you may begin.

Okay.

Yeah.

Thank you operator, and good morning, everyone.

Joining me on today's call and webcast Dr. Shankar, Missouri, Okay, Who's Chairman CEO and co founder who will provide a business update and an overview of our clinical and operational progress.

Mike O'brien here, our corporate controller is also on the call to provide a financial update for the quarter ended September 32020.

Dr. Arun a party Chief Scientific officer head of research development and medical will be available to answer questions. Following the presentation.

This morning, we issued a press release detailing associated business and operational highlights for the third quarter of 2023.

Encourage listeners to review the press release, which is available on our website at <unk> Dot com.

The call is being recorded and a replay with the accompanying slide presentation will be available on the investors section of the <unk> website for approximately 45 days.

This presentation contains forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties.

We may in some cases use terms such as predict believe potential propose continue estimate anticipates expects plans intends may could might will should or other words that convey uncertainty of future events or outcomes to identify these forward looking statements.

Such statements include but are not limited to statements regarding our clinical development activities and related anticipated timeline.

Such statements are subject to numerous important factors risks and uncertainties that may cause actual events or results to differ materially from our current expectations.

And other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission SEC, including the risk factors described in the section titled Risk factors in our quarterly and annual report.

File with the SEC.

Any forward looking statements that we make in this presentation speak only as of the date of the presentation.

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, <unk> quarterly report on Form 10-Q, covering the third quarter of 2023 has been filed.

I will now turn the call to Dr. Missouri.

Thank you Tiffany and thank you all for joining us today.

Lymphocytes in the press release, we put out this morning, we continue to make significant headway with the development of our pipeline assets, particularly with our first in class ophthalmic gene therapy programs and I'm proud of the momentum we have achieved to date.

Following additional positive and encouraging clinical study results from our remodel modestly of gene therapy based phase one to our Q4 hundred study in September 2023, we believe we have strong clinical evidence to initiate our phase III clinical trial.

Pigmentosa RP patients in early 2024 based on FDA conference.

A dual track strategy, we also intend to expand out our Q4 hundred 50 trial in the second half of 'twenty 'twenty four to include patients with Leber congenital amaurosis LCA contingent upon favorable results from the phase <unk> study.

With enrollment.

For our Q4 10, and <unk> 14 SD programs, we are diligently working to those patients. This quarter, we anticipate clinical updates from our Q4 hundred Q4 come back to you for these studies in the later part of 2024.

Our clinical and regulatory teams continue to work on responses to the FDA regarding the <unk> submission for our Q2 hundred the Companys ophthalmic biological product candidate and we plan to initiate a phase one clinical study in the first half of 'twenty to 'twenty four contingent on the lift of the FDA.

And it is great when I listen to you.

Thanks.

But our region due to cell therapy candidate for knee cartilage repair Neocart, we are on track to complete construction.

Our state of the art Cgmp facility at the end of this year and are planning to complete qualification of the facility in the first half of 2024.

We plan to initiate the phase three clinical trial in the second half of next year.

Last month.

To be selected.

<unk> in a phase one clinical trial 100 by National Institute of allergy and infectious disease to investigate the administration of our COVID-19, mucosal vaccine candidate occupied Hungary.

Safety and Immunogenicity of up to 500 will be evaluated using inhaled and intranasal routes of delivery during the phase one clinical trial in the first half of 2024.

All of these catalysts consider we can safely reiterate the 2020 for the transfer may do for oxygen.

Our mission to introduce critically needed therapies into the market is imminent.

Planned initiation of phase III trials, encompassing gene and cell therapies and the near term.

Our R&D team's dedication and hard work as even the significant progress and compelling results.

<unk> modifiers gene therapy, our Q4 hundred program for RFP and in CF patients.

Towards the phase two trial of primary objective has been to.

Observed safety and Tolerability of the sub retinal administration of our Q4 hundred and subjects as well as immune response and systemic distribution.

Our preliminary signs of efficacy, we focused on a few visual function and functional vision indicators, namely best corrected visual acuity <unk> low luminescence presume equity alone.

In multiply luminescence mobility test empty <unk>.

The details on our current design can be found on clinical trials start out with the identifier code listed.

At the bottom of this slide.

Let me provide situational analysis.

Unmet need and unload resort market for RP in FCS patients and.

An estimated one 6 million people globally are affected by RP and NCA combined in the U S alone. We're looking at about 125000 patients total.

In LCA are classified as inherited retinal diseases.

Group of heterogeneous disorders.

Dana.

These diseases, often lead to a slight loss and ultimately blindness.

That said the earlier homeostasis can be stabilized in patients with either of these diseases, but better.

Through relevant medical meetings and continued engagement with advocacy groups. We aim to create awareness for the prevalence of retinitis pigmentosa and leber congenital amaurosis and potential emerging therapies like our Mt platform.

<unk> objective is to provide treatment of people suffering from vision impairment and blindness caused by RP and MCA for whom currently no therapeutic options exist.

I listed our three exploratory endpoints for visualization stabilization.

And improvement.

<unk> in patients treated with our Q4 hundred on slide four.

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And then I'll let D.

And the 12 accumulate to subjects that have undergone a minimum of six months follow up.

Our Q4 hundred dosing, we observed the following metrics.

This venn diagram demonstrates the age of 12 subjects short is a stabilization means no change from baseline plus minus four level change for <unk> and.

In EMEA and zero less level changed for MLP.

Improvement in all three parameters of <unk> and <unk>.

Which means more levers.

And MLM D Richardson articulate one lex level <unk>.

Demonstrating initial efficacy of our Q4 hundred.

Non responders of listed outside the circles for each group.

To recap what we know from our findings to date is that our Q4 hundred has a favorable safety and tolerability profile in patients post.

Positive trends observed in all set with relationships and stability and improvement factors, which details that.

83% of subjects demonstrated stabilization or improvement in the future lie is around <unk> <unk> MLP scores from baseline.

Percent of subjects showed stabilization or improvement in future lies in MLP scores from baseline.

86% of ROE mutation subjects experienced a stabilization or improvement in MLM discourse from baseline, among which 29% demonstrated three less luminescence level improvement.

Demonstrating the gene agnostic mechanism faction of our Q4 hundred.

The road mutation effect more than 10000 people in the U S alone.

Based on this data we are highly enthusiastic about the future of our Q4 hundred and the vision saving potential it may provide to RP and LCA patients.

The execution of critical elements of our Q4 hundred phase one two trial, including the completion of dosing of RP and LCA patients sets the stage for us to execute a phase III clinical trial for both indications in 2024 upon FDA Congress.

Our Q4 10 hour modified gene therapy candidate for dry age related macular degeneration AMD is a potential one time curative therapy with a single sub retinal injection that targets multiple pathways, causing dry AMD, including the big metropolitan inflammation oxidative stress.

And complement activation.

Unlike other currently market products targeting a single pathway complement activation. We are currently enrolling patients in the phase two <unk> study to assess the safety and efficacy of our Q4 10 for geographic atrophy secondary to dry AMD.

Geographic atrophy, an advanced form of dry age related macular degeneration affects approximately 1 million people in the United States alone.

From a competitive standpoint, we believe Aki for them is differentiated among other therapies available and in development for geographic atrophy and dry AMD by frequency of administration onetime versus multiple injections per year reduced side effects from structural impact strong safety profile.

It's a mechanism a story homeostasis and preserving the conditions that promote self help.

The slight demand straights, how our Q4 time you play this in AAV delivery platform for the retinal delivery of Aurora G. In preclinical studies occupancy and demonstrated efficacy and regulating multiple pathways and one with the disease, including lipid metabolic them reducing.

<unk> formation.

Regulation of inflammation.

<unk> inflammation oxidative stress improving cell survival membrane attack complex complement the story and they complement protein.

On this slide we have captured a proposed program design for our Q4 10.

63, adult subjects 50 or older with geographic atrophy secondary to dry AMD, we will observe the treatment effect of a single unilateral sub retinal injection or Q4 of them, starting with safety and efficacy in patients.

We're employing a three plus three design with the low medium and high dose. In addition to a dose expansion exercise using a one to one to one design randomized subjects to Eva two treatment groups or dose levels, one control group.

Using a similar approach our orphan drug designation <unk> SD modifier to gene therapy platform I'll start disease, Leverages nuclear hormone receptors to modulate selectivity and you flagged this in AAV delivery platform for retinal delivery.

Already our later orphan receptor <unk>.

Our Q4 and delivery and preclinical studies of targeted disease demonstrated a structural and functional improvement.

And the occupancy ft phase one two trial, we intend to treat and investigate 42 subjects 30 of which are adults and 12 of our children its target disease the inclusion criteria.

Look at their own patients between 18 to 65 and pediatrics between $6 17.

Employing a three plus three design with a low medium and high dose cohort. In addition to your dose expansion exercise using a one to one to one design randomized subjects to either two treatment groups per dose levels are one control group.

Our team's diligent efforts resulted in <unk> <unk>.

<unk> <unk> hundred for inclusion in a project Nextgen phase one clinical trial of a mucosal vaccine candidate for COVID-19 likely to be initiated in the first half of 2024.

From our own development efforts, we observed vaccine induced high neutralizing and factor responses. During preclinical studies occupy 100, we believe the inhaled route of administration has the potential to be the Holy Grail for broad and durable protection from severe diseases and can suppress the transmission rate.

As a refresher projects Nexgen multi government agency initiate two overseen by AIG.

He is a $5 billion multi government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19.

And my early.

We will execute the clinical trial for <unk> hundred upon completion of the trial oxygen will process full rights soft reference to the findings. This initiative is a testament to the fact that COVID-19 is still ramping with.

With the emergence of new variants and needs more durable vaccines to treat them in a recent Harris poll.

We are favorably found that 66% of Americans would prefer to have a more vaccine options. The board also upon that 52% of Americans will be more open to getting an intranasal around here versus injectable COVID-19 vaccine in line with the <unk> mission to support immigration and public health.

We look forward to potentially expanding the platform for the flu and other risk briefly wadham diseases and infections.

I would like to bring our pipeline update close by providing a brief update on Neocart <unk> until August regenerative cell therapy.

Would you use this patients on cartilage cells is on track to begin its phase III clinical trial in the second half of 2024, a cgmp facility for manufacturing Neocart is expected to be completed at the end of 2023 and qualification is expected in the first half of 2024.

Our Q2 hundred is an ophthalmic biological product candidates in preclinical development for treating severely sight threatening diseases like diabetic macular edema, diabetic retinopathy and wet age related macular degeneration. We are working on responses and continue to interact with the FDA regarding the clinical hold.

On a rocky to a 100 R&D submission and expect to initiate a phase one clinical study and thus have a currency can be for <unk>.

That I will now turn the call over to our corporate controller, Michael burning gear.

To provide an update on our financial results for the third quarter ended September 32023.

Thank you Shocker.

Our research and development expense for the quarter ended September 32023 were $6 3 million compared to $15 6 million for the third quarter of 2022 general and administrative expenses for the quarter ended September 32023 were $9 1 million compared to $7 5 million.

During the same period in 2022.

Net loss was approximately $14 2 million or <unk> <unk> net loss per share for the.

Quarter ended September 32023, compared to a net loss of approximately $21 9 million or 10.

Net loss per share for the third quarter of 2022.

Net loss was approximately $53 6 million or 22 net loss per share for the nine months ended September 32023, compared to a net loss of approximately $59 4 million or <unk> 28, <unk> net loss per share for the nine months ended September 32022.

Our cash cash equivalents and investments totaled $53 5 million as of September 32023, compared to $90 9 million as of December 31, 2022.

As always we are constantly exploring strategic and shareholder friendly opportunities to increase our working capital and we'll be focused on seeking out corporate partnerships for gene therapies and non dilutive funding for vaccines.

That concludes my update for the quarter Tiffany's back to you.

Thank you, Mike we will now open the call for questions operator.

Well thanks for your questions to ask a question. This time. Please press Star then the number one on your telephone keypad.

Well pause for just a moment to compile the Q&A last June.

We have a question from the line of Mike O'hare.

HPE.

Your line is open.

Hey, good morning, Shanghai and Tim.

Here's RK.

For RK.

Congrats on the progress so I just had a.

Couple of question on 400.

So when could we expect the complete dataset from our phase II.

Study as well, especially for the patients.

So if you can give some color on that would be.

Appreciate it.

Yes, the LCA, we just dose.

So I think it will take.

And the later part of next year.

How about the other patient left in the year.

Group, the RP patients will get it in the first half.

However, we believe we have adequate information and we are working with the regulatory agencies of the Anda Ma.

His III.

Okay.

So speak of the Phase III study.

So.

From what to use that in the press release and the core.

As the phase III will be a single phase III impacted together, both RP patient as well I would say or.

It could be two separate phase III study.

We'll start with RP because that's the data we have right now and then we're going to add LTE arm little later in the clinical trial.

Okay, so that would be in the single phase III or yes.

Okay. Okay.

And the last question on 400 days.

One is for <unk>.

You request a meeting with FDA.

Based on your own proposal.

What which.

<unk> going to be your primary endpoint.

And lift.

But the answer that our CSO better thanks, Tim good so we are considering.

Combinatorial approach and we have we have proposed to the FDA.

And where.

We are going to have a meeting with them this quarter.

Accordingly, once we have alignment that they are good and we will update the market.

Alright, it sounds great. Thanks for taking my question.

Thank you.

Yes.

Okay.

Our next questions comes from the line of Robert <unk> with Noble capital markets. Your line is open.

Good morning, and thanks for taking my question.

I just had a follow up on the phase III for <unk> 400, and.

Wondering if you have any information or could disclose how many patients you expect to be in the trial or what the length of follow up is going to be for the patients.

Good morning, Robert I'll, let <unk> address that.

Yes. So we are planning in the range of <unk> 200 subjects in phase III.

One is to wander into Madison and one year follow up.

Okay. Thank you very much.

Okay.

Just to confirm we did 100 okay.

It's 100 patients Robert Yes.

Again, if you would like to ask a question Press Star then the number one on your telephone keypad.

There are no further questions at this time I will now turn the call over to chairman and CEO, Doug question Carl must annuity.

In closing I'd like to reiterate our unwavering commitment to groundbreaking science and clinical innovations in order to create effective positively impactful therapies that are accessible to patients globally.

As we continue to execute stated plans, we remain focused on delivering long term value for our shareholders.

<unk> and for a prospect to one seeking to be part of our story.

And have a great day.

Thanks, everyone.

This concludes today's conference call you may now disconnect.

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