Q1 2024 Ocugen Inc Earnings Call

Participants

Tiffany Hamilton; Head of Communications; Ocugen Inc

Shankar Musunuri; Chairman of the Board, Chief Executive Officer; Ocugen Inc

Michael Breininger; Principal Financial Officer, Interim Chief Accounting Officer; Ocugen Inc

Arun Upadhyay; Chief Scientific Officer, Executive Officer, Head of Research, Development and Medical; Ocugen Inc

Huma Qamar; Chief Marketing Officer; Ocugen Inc

Arthur He; Analyst; H.C. Wainwright & Co., LLC

Robert LeBoyer; Analyst; Noble Capital Markets

Daniil Gataulin; Analyst; Chardan Capital Markets

Presentation

Operator

Good morning, and welcome to Ocugen's first quarter 2024 financial results and business update. Please note that this call is being recorded. (Operator Instructions)
I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.

Tiffany Hamilton

Thank you, operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's Chairman, CEO and Co-Founder, who provide a business update and an overview of our clinical and operational progress. Michael Breininger, our Corporate Controller, is also on the call to provide a financial update for the quarter ended March 31, 2024. Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research and Development, and Dr. Huma Qamar, Chief Medical Officer, will be available to answer questions following the presentation.
This morning, we issued a press release detailing associated business and operational highlights for the first quarter of 2024. We encourage listeners to review the press release, which is available on our website at Ocugen.com. This call is being recorded and a replay with the accompanying slide presentation will be available on the Investors section of the Ocugen 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 term, such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intend, 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 timelines. Such statements are subject to numerous important factors, risks and uncertainties and may cause actual events or results to differ materially from our current expectations. These other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors in the 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 the presentation, except as required by law, we assume no obligation to update forward-looking statements contained in this presentation whether as result of new information, future events or otherwise after the date of this presentation. Finally, Ocugen's quarterly report Form 10-Q covering the first quarter of 2024 has been filed.
I will now turn the call over to Dr. Musunuri.

Shankar Musunuri

Thank you, Tiffany, and thank you all for joining us today. As detailed in our press release, we are excited to discuss the substantial progress of our game-changing modifier gene therapy platform. By using nuclear hormone receptors to restore homeostasis in the retina, modifier gene therapy has the potential to address multiple inherited retinal diseases as well as diseases -- larger multifactorial diseases such as dry age-related macular degeneration, through a gene agnostic approach.
Just after the close of the first quarter of this year, we announced that the FDA cleared our IND application from OCU400 Phase 3 liMeliGhT clinical trial and the EMA provided acceptability of the US-based trial for submission of a market authorization application. I will discuss the significance of these milestones and the potential for OCU400 in greater depth later in the presentation.
OCU410 and OCU410ST are now in Phase 1/2 clinical trials. Targeting geographic atrophy, secondary to dry age-related macular degeneration and Stargardt disease, respectively. With the clinical updates expected in the third quarter of 2024. Our cell therapy platform is poised to advance that the renovations to our existing facility completed earlier this year. This gives Ocugen the capability to support NeoCart autologous cell therapy, manufacturing for personalized Phase 3 clinical material, contingent upon adequate funding.
Finally, OCU500 as soon advanced into Phase 1 clinical trial via the National Institute of Allergy and Infectious Diseases, NIAID sponsor trial, comparing the administration of OCU500 via two different rounds; inhalation into the lungs and as a nasal spray. With three gene therapy candidates to treat blindness diseases from the clinic and the Phase 3 ready cell therapy candidate. We are confident that 2024 will be a bellwether for Ocugen.
We are thrilled to share significant advancements in our leading modifier gene therapy candidate OCU400 as it makes remarkable strides in clinical development. The green light from the FDA to begin the Phase 3 clinical trial, marks a pivotal milestone. As OCU400 becomes the first gene therapy to progress to Phase 3 trials, it's such a broad retinitis pigmentosa indication.
Until now, there has been only one market product to treat one of the hundred gene mutations associated with RP. Now there is real hope for all RP patients who haven't had a treatment option. Our completion of enrolment and dosing in Phase 1/2 trial demonstrated promising safety and efficacy across various genetic mutations and dosage levels, paving the way for Phase 3 clinical trial.
OCU400 has already received key regulatory approvals, including orphan drug, regenerative medicine advanced therapy and orphan medicinal product designations from both FDA and European Commission for treating inherited retinal diseases. These endorsements highlight OCU400's broad therapeutic potential, and we remain on track to meet 2026 BLA and MAA approval targets.
We expect to begin dosing patients in the Phase 3 liMeliGhT clinical trial in the second quarter of 2024, which will include 150 subjects across two arms, one with patients affected by RHO mutation and one on -- that is gene-agnostic. Luminance dependent navigation assessment, LDNA, the primary endpoint for this study focuses on the proportion of responders in treated and untreated control groups, achieving an improvement of at least 2 two Lux level from baseline in the study eyes.
The secondary endpoint, it will be measured by changing low Luminance visual equity score of 0.3 log more from the baseline. The effectiveness of the treatment will be compared to say, untreated control group for the secondary endpoint as well. Additionally, leveraging a dual-track strategy, we plan to expand the Phase 3 trial in the later half of 2024 to include patients with Leber congenital amaurosis or LCA, contingent on favourable results from Phase 1/2 study.
Let me take a moment to discuss the unmet need and underserved market for RP and LCA patients. An estimated 1.6 million people globally are affected by RP and LCA combined. In the US and Europe alone, our initial target markets, there are nearly 300,000 total patients. RP and LCA are classified as inherited retinal diseases from the group of heterogeneous disorders that affect the retina. These conditions frequently result in visual impairment and ultimately blindness.
By establishing homeostasis in patients affected by these diseases, our aim is to preserve or improve vision by actively sharing our insights at pertinent medical conferences and engaging with advocacy groups, we strive to raise awareness of the desperate need to find a solution for all RP and LCA patients as well as potential of modified gene therapy to revolutionize treatment. To better understand the experience of one of our Phase 1/2 clinical trial patients, I encourage you to watch this video and the patient's section of our website.
Moving on to our development in the treatment of geographic atrophy, secondary to dry age-related macular degeneration, dAMD and Stargardt disease with our OCU410 and OCU410ST programs. These modified gene therapies leverage the nuclear receptor gene RAR-related orphan receptor A, RORA, aiming to provide a potential onetime therapy for life with a single subretinal injection.
OCU410 specifically designed to address multiple pathways implicated in the pathogenesis of dAMD offers a distinct advantage or current treatments that the target only one cause of GA and often requires multiple injections per year, accompanied by various safety concerns. Our approach with OCU410 is to provide a comprehensive and durable solution, a potential onetime therapy for life.
Dry AMD affects about 19 million people in the US and Europe combined, while GA affects about 2 million to 3 million people in the US and Europe, a significant market opportunity. In December 2023, we began the Phase 1/2 ArMaDa clinical trial for OCU410. Considerable progress has been made with the completion of dosing in both the first and second cohort low and medium doses.
Following the successful dosing in this cohort, the Data and Safety Monitoring Board, DSMB will convene later this month to evaluate proceeding with the high-dose cohort in ongoing dose escalation phase of the study. After completion of the third cohort where transition into Phase 2 clinical trial, the expansion phase. A key upcoming event for this trial is a clinical update, which is anticipated in the third quarter of 2024. This data will provide initial insights into the safety and efficacy of OCU410.
OCU410 Phase 1 study is multicentre and open label, focusing on dose-ranging, while Phase 2 is randomized, aiming to expand our understanding of drug's efficacy and safety compared to a control untreated arm with patients randomized in a one-to-one-to-one ratio across two dosage groups and one control group. Participants must be aged 50 or older, have a best corrected visual equity of approximately 24 letters or more using the ETDRS chart and have a total geographic atrophy area between 2.5 and 20.5 millimetre square.
We now turn to our OCU410ST, which received Orphan drug designation from FDA for the treatment of ABCA4 associated retinopathy, including Stargardt disease. Stargardt affects approximately 100,000 people in the US and EU combined.
This modified gene therapy candidate also utilizes that RORA and the Phase 1/2 GARDian trial for Stargardt disease is actively enrolling patients. We have completed dosing in the first cohort, low dose. In April 2024 the Data Safety and Monitoring Board approved the continuation for the medium dose, which we expect will be completed this month. A clinical trial update for OCU410ST is also anticipated in the third quarter of 2024.
Phase 1/2 trial involves up to 42 participants, 30 adults and 12 children, which exhibit mild to moderate disease symptoms. The study uses a three plus three dose escalation design in Phase 1, segmenting subjects into low, medium and high dose cohorts. Following the dose escalation phase, the trial will transition into Phase 2, the expansion phase. This expansion phase will use a one-to-one-to-one design to randomize subjects into two different treatment groups at varying dose levels, body control, untreated group, allowing for a comprehensive assessment of treatment's efficacy across different dosages.
Our work across OCU400, OCU410 and OCU410ST perform our enduring commitment to the success of these modified gene therapies for the benefit of patients faced with the terrible prospect of losing their sight. We are encouraged by the progress in these trials and look forward to sharing further updates as we reach critical milestones and get closer to addressing substantial unmet medical needs.
With that, I'll now turn the call over to our Corporate Controller to provide an update on our financial results from first quarter ended March 31, 2024. Mike?

Michael Breininger

Thank you, Shankar. Our research and development expenses for the quarter ended March 31, 2024, were $6.8 million compared to $10.2 million for the first quarter of 2023.
General and administrative expenses for the quarter ended March 31, 2024, were $6.4 million compared to $8.3 million during the same period in 2023. Net loss was approximately $11.9 million or $0.05 net loss per share for the quarter ended March 31, 2024, compared to a net loss of approximately $17.3 million or $0.08 net loss per share for the first quarter of 2023.
Our cash and cash equivalents totalled $26.4 million as of March 31, 2024, compared to $39.5 million as of December 31, 2023. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital.
That concludes my update for the quarter. Tiffany, back to you.

Tiffany Hamilton

Thank you, Mike. We will now open the call for questions. Operator?

Question and Answer Session

Operator

(Operator Instructions)
Arthur He, H.C. Wainwright.

Arthur He

Hey, good morning. Shankar and team. Congrats on the progress. So I just had a quick one regarding the 400 Phase 3 study. [Rick] for the gene agonistic arm, is there and minimum number of patients for each specific gene mutation to meet the requirement for the BLA application?

Shankar Musunuri

Hey Arthur. Good morning. I let Arun take the response.

Arun Upadhyay

No, we have not previous specified any specific number of patients for each mutation. But our approach is going to be, you know, include as many mutation as possible in gene-agnostic [com].

Arthur He

Okay, thanks. Thanks for the colour. And also my second question is regarding the 200. Could you give us more color on the status and what's your effort to get the clinical hold lifted as soon as possible.

Arun Upadhyay

Yes. So we are working on addressing the CMC question we received from the FDA, and we are planning to address that ASAP and then continue the development of OCU200 further.

Arthur He

Okay, great. Thanks for taking my question.

Shankar Musunuri

Thank you, Arthur.

Operator

Robert LeBoyer, Noble Capital Markets.

Robert LeBoyer

Good morning and congratulations on all the progress during the quarter. My question has to do with the Phase 3 trial coming up and you had mentioned some milestones to start. Are there any timeframes or any particular milestones that we can look forward to in terms of accrual or projected time for data release.

Shankar Musunuri

Yeah, Robert. I'll let Dr. Qamar answer the question.

Huma Qamar

Hi, thank you for your question. In terms of the milestones and you are aware that OCU400 reduces the card approval, we are currently screening patients, and we will be continuously updating the market with dosing updates very soon.

Robert LeBoyer

Okay. Is there any projected time for the first data release?

Huma Qamar

For OCU400 Phase 3 on Phase 1/2. (multiple speaker) Okay. So Phase 1/2, we have already updated that, continuous updates would be coming in the next quarter as well. And as soon as we did see more data on the LCA patients for OCU400 Phase 3, it will be -- there is no timeframe as it will be periodic, and we will be giving periodic updates to the market as soon as it's available.

Shankar Musunuri

And Robert, I think this is a controlled clinical trial for Phase 3. So until the study is done, we cannot reveal any information to the market on the efficacy portion.

Robert LeBoyer

Sure. That's a --

Shankar Musunuri

We'll provide periodic updates as they Dr. Qamar is saying on recruitment and with the trial is going and also new markets and update about on track for our target approval for BLA and MMA in 2026.

Robert LeBoyer

Okay. Yeah, that's probably as much as anyone can say for a rare disease like this. So thank you.

Operator

Daniil Gataulin, Chardan.

Daniil Gataulin

Yeah, hey, good morning, guys, and thank you for the question. Also, the question on OCU410 Phase 3. So you mentioned that as part of DealerTrack strategy, you will be expanding Phase 3 that includes patients with LCA pending the [Phase 1/2 TRC] data. I just wanted to clarify if this will be additive new arm in the trial or if it will be a separate stand-alone trial?
And I guess the second part of the question is what data from LCA Phase 1/2, would you consider favourable that would give you a go-ahead decision for these patients? Thank you.

Shankar Musunuri

Arun will take the call.

Arun Upadhyay

Thanks Shankar. So based on the Phase 1/2 study outcome, our plan is to have the [necessary time] for the LCA because it's a different disease. So once we have the data level, we'll be engaging with the agency and best do agency recommendation which will initiate the trial accordingly.

Daniil Gataulin

Okay, got it. Thank you. And in terms of the Phase 1/2, what would you consider favourable that went into Phase 3.

Arun Upadhyay

Primarily the safety and efficacy.

Daniil Gataulin

Alright, thank you.

Operator

This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO, Dr. Shankar Musunuri.

Shankar Musunuri

Thank you, operator. Our efforts in the first quarter of the year evidence the importance of our gene therapy programs and the need to operate the business to ensure their success. We are optimistic about Ocugen cell therapy in vaccine platforms, meaning that these technologies have great therapeutic and financial potential that pursuing partnership to support our entire pipeline. We look forward to mark this quarter and the rest of the year holds for the Company, our people and the patients we serve. Tiffany?

Tiffany Hamilton

Thanks, everyone. Bye.

Operator

Ladies and gentlemen, that concludes today's call. Thank you all for joining, and you may now disconnect.