Q3 2024 Ocugen Inc Earnings Call
Good morning, and welcome to oxygen third quarter, 'twenty 'twenty, four financial results and business update.
Operator: Good morning and welcome to Ocugen's 3rd Quarter 2024 Financial Results and Business Update. Please note that this call is being recorded at this time. All participant lines are in listen-only mode.
Please note that this call is being recorded at this time all participant lines are in listen only mode.
Operator: Following the speaker's commentary, there will be a question and answer session.
Following the Speakers' commentary there will be a question and answer session.
Tiffany Hamilton: I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications.
Speaker Change: I'll now turn the call over to defend the Hamilton oxygen head of corporate Communications you may begin.
Tiffany Hamilton: You may begin. 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 will provide a business update and an overview of our clinical and operational progress.
Thank you operator, and good morning, everyone.
Speaker Change: Joining me on today's call and webcast as doctors younger machinery oxygen chairman CEO and co founder who will provide a business update and an overview of our clinical and operational progress.
Tiffany Hamilton: Ramachandran, our Chief Accounting Officer, who is transitioning from Mike Breininger, Interim Chief Accounting Officer, is also on the call to provide a financial update for the quarter ended September 30, 2024. Dr. Huma Qamar, Chief Medical Officer, and Dr. Arun Upadhyay, Chief Scientific Officer, will be available to answer questions following the presentation.
Speaker Change: We're measuring the children, our chief Accounting Officer, who was transitioning from Mike Breininger interim Chief Accounting Officer is also on the call to provide a financial update for the quarter ended September 32024.
Speaker Change: Doctor, who moca more chief Medical Officer, and Dr. Arun Party, a chief scientific officer will be available to answer questions. Following the presentation.
Tiffany Hamilton: This morning we issued a press release detailing associated business and operational highlights for the third quarter of 2024. We encourage listeners to review the press release, which is available on our website at ocugen.com.
This morning, we issued a press release detailing associated business and operational highlights for the third quarter of 2024.
Speaker Change: We encourage listeners to review the press release, which is available on our website at <unk> Dot com.
Operator: This call is being recorded and a replay with the accompanying slide presentation will be available on the investor section of the Ocugen website for approximately 45 days.
Speaker Change: This call is being recorded and a replay with the accompanying slide presentation will be available on the investors section of the oxygen web site for approximately 45 days.
Tiffany Hamilton: 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 predicts, believes, potential, proposed, continue, estimates, 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 timelines. 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.
Speaker Change: 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 predicts believe potential proposed.
Speaker Change: <unk> estimates anticipates expects plans intends.
Speaker Change: They could might will should or other words that convey uncertainty of future events or outcomes to identify these forward looking statements.
Speaker Change: Such statements include but are not limited to statements regarding our clinical development activities and related anticipated timelines.
Speaker Change: The statements are subject to numerous important factors risks and uncertainties that may cause actual events or results to differ materially from our current expectations.
Tiffany Hamilton: These and other risks and uncertainties are more fully described in our periodic filing with the Securities and Exchange Commission, the SEC, including 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 it's 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 the presentation.
Speaker Change: These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission the SEC, including risk factors described in the section entitled Risk factors in our quarterly and annual reports that we filed with the SEC.
Speaker Change: 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 the presentation.
Tiffany Hamilton: Finally, Ocugen's quarterly report, Form 10-Q, covering the third quarter of 2024, will be filed next week.
Speaker Change: Finally, <unk> quarterly report form.
Speaker Change: 10-Q, covering the third quarter of 'twenty 'twenty four will be filed next week.
Shankar Musunuri: I will now turn the call over to Dr. Musunuri. Thank you, Tiffany, and thank you all for joining us today. We're excited to share the ongoing progress of our novel modifier gene therapy platform across all three clinical programs, as well as recent announcement regarding our Octi200 biologic candidate, which I will highlight later in the presentation. During the third quarter, we accomplished notable clinical and regulatory milestones, including approval from Health Canada to initiate the RQ400 Phase 3 Limelight clinical trial in Canada and approval from the U.S. FDA for an Expanded Access Program, or EAP, for the treatment of adult patients aged 18 and older with retinitis pigmentosa, or RP, using RQ400.
I will now turn the call over to Dr. Mr. Murray.
Mr. Murray: Thank you Tiffany and thank you all for joining US today, we're excited to share the ongoing progress of our novel modify your gene therapy platform.
Mr. Murray: All three clinical programs as well as recent announcements regarding the rocket 200, biologic candidates, which I will highlight later in the presentation.
Mr. Murray: During the third quarter, we accomplished notable clinical and regulatory milestones, including approval from health, Canada to initiate our Q4 hundred phase III limelight clinical trial in Canada and approval from the U S. FDA for an expanded access program or EAP for the treatment of pain.
Mr. Murray: <unk>, aged 18 and older with retinitis Pigmentosa RP using Q4 hundred these.
Shankar Musunuri: These accomplishments and consistent trial enrollment are bringing the company even closer to providing a potential one-time treatment for life to patients living with RP. Our modifier gene therapy programs leveraging the RORA gene, OCU410 and OCU410ST are advancing through their respective clinical trials as planned. We are currently dosing patients in OCU410 phase 2 Armada clinical trial for the treatment of geographic atrophy, GA, an advanced stage of dry age related macular degeneration, DAMD. We completed the phase one dosing in the phase one to OCU410ST Guardian clinical trial with a favorable safety and tolerability profile. The data safety.
Mr. Murray: These accomplishments and consistent to trial enrollment or.
Mr. Murray: Bringing the company even closer to providing it.
Mr. Murray: One time treatment for life to patients living with RP.
Mr. Murray: Our modified gene therapy programs, leveraging the rotor gene Q4, and our Q4, Denis Deet, alright, advancing through their respective clinical trials as planned.
Mr. Murray: Currently dosing patients in our Q4 10 phase two clinical trial for the treatment of geographic atrophy G. H.
Mr. Murray: In advanced stage of dry age related macular degeneration D M D.
We completed the phase one dosing.
They just want to ask you for SD Guardian clinical trial with a favorable safety and Tolerability profile.
Mr. Murray: The data on safety.
Shankar Musunuri: Monitoring Board, DSMB, approved enrollment for the second phase of the Phase I-II clinical trial. Stargardt disease is the most common inherited retinal disease and there remains a large unmet medical need with no currently approved FDA treatment. I'm encouraged by these achievements and confident in our path forward to achieving our near-term inflection points. This week, we announced the closing of a debt financing that secured 30 million from Avenue Capital Group. This funding is expected to extend our runway into the first quarter of 2026. Dosing is well underway in the Pivotal Phase 3 Limelight clinical trial for RQ400, our lead gene therapy candidate that utilizes NR2A3 gene.
Mr. Murray: Monitoring board the SMB abroad enrollment for the second phase of the phase one two clinical trial.
Its target disease is the most common inherited retinal disease and there remains a large unmet medical need with no currently approved FDA treatments.
Mr. Murray: I'm encouraged by these achievements and confident in our path forward.
Mr. Murray: Our near term inflection points.
Mr. Murray: This week, we announced the closing of a debt financing secured $30 million from Avenue capital Group.
Mr. Murray: This funding is expected to extend our runway into the first quarter of 2026.
Mr. Murray: Dosing is well underway and the pivotal phase III limelight clinical trial for our Q4 hundred.
Mr. Murray: Our lead gene therapy candidate that utilizes <unk> it's regime.
Shankar Musunuri: As previously mentioned, we received Health Canada's approval to initiate the phase three clinical trial for OCU-400 in Canada. Expanding the clinical trial to Canada is a significant opportunity for Ocugen, as it will allow us to reach a broader patient population. encompassing numerous gene mutations associated with RP. We plan to enroll subjects across a maximum of five sites, expediting recruitment and broadening the commercialization potential of the gene agnostic treatment in the United States and Europe. Acu400 also received FDA approval for an EAP for the treatment of adult patients with RP aged 18 and older. The EAP is a meaningful step forward, as it makes OCU400 available to qualifying patients beyond our Phase III clinical trial, offering hope and optimism for a wider population of patients desperate for a therapeutic option.
As previously mentioned, we received health, Canada approval to initiate the phase III clinical trial for our Q4 hundred in Canada expanding.
Mr. Murray: Expanding the clinical trial to Canada is a significant opportunity for oxygen as it will allow us to reach a broader patient population.
Mr. Murray: Boston numerous gene mutations associated with RP, we plan to enroll subjects across a maximum of five sites expediting recruitment and broadening the commercialization potential of these gene agnostic treatment in the United States and Europe.
Mr. Murray: Our Q4 hundred also received FDA approval for an EAA.
Mr. Murray: For the treatment of adult patients with RP, aged 18 and older.
Mr. Murray: The EAP is a meaningful step forward as it makes our Q4 hundred available to qualifying patients beyond our phase III clinical trial, offering hope and optimism, but a wider population of patients this breakthrough therapeutic option.
Shankar Musunuri: This approval also serves to validate the positive safety data generated in the previous Phase I-II ARCQA 400 clinical trial. Furthermore, this is the first phase three gene therapy candidate to treat patients with RP, regardless of mutation. The phase three clinical trial is on track to complete enrollment in the first half of 2025, file the Biologics License Application, BLA, and Market Authorization Application, MAA in Europe in the first half of 2026 and pursue commercialization in 2027.
Mr. Murray: This approval also serves to validate the positive safety data generated in the previous space want to occupy 100 clinical trials.
Mr. Murray: What are their more this is the first phase III gene therapy candidate to treat patients with RP, regardless of mutation the phase III clinical trial is on track to complete enrollment in the first half of 2025 filed the biologics license application BLA and market authorization application MAA in Europe.
Mr. Murray: In the first half of 296 and pursue commercialization in 2027.
Mr. Murray: Let me take a moment to highlight the unmet need and under resort market for RP patients.
Shankar Musunuri: Let me take a moment to highlight the unmet need and under-deserved market for RP patients. There are approximately 300,000 patients in the U.S., Europe, and Canada that are affected by the disease, which is caused by mutations in roughly 100 different genes. The only approved gene therapy on the market and one currently in development each address one mutation associated with the disease. Other candidates in development, including optogenetics, are intended only for a very small patient population. Ocu400 has showcased its potential to provide a totally new category of treatment using its gene agnostic approach. Rather than a one-to-one approach, Modified Gene Therapy targets many genes associated with this underserved disease through the use of master gene regulators, resetting the functional network of the retina and restoring overall health.
There are approximately 300000 patients in the U S Europe, and Canada detour affected by the disease, which is caused by mutations in roughly 100 different genes.
Mr. Murray: The only approved gene therapy on the market and one currently in development each address one mutation associated with the disease.
Mr. Murray: Other candidates in development, including Optogenetics are intended only for a very small patient population.
Speaker Change: Our Q4 hundred has showcased its potential to provide it.
Speaker Change: Totally new category of treatment using X gene agnostic approach.
Speaker Change: Other than one to one approach modified gene therapy targets many genes associated with this.
Speaker Change: <unk> disease through the use of master gene regulators resetting the functional network of the retina and restoring overall health.
Speaker Change: We continue our extensive campaign.
Shankar Musunuri: We continue our extensive campaign to educate key stakeholders about the differentiated mechanism for action of our modifier gene therapy platform and its advantages over current therapies. During third quarter, we had the opportunity to provide updates on our three clinical stage modifier gene therapies to significant investor audiences, as well as industry decision makers during meetings like the cell and gene meeting on the MESA hosted by the Alliance for Regenerative Medicine.
Speaker Change: Key stakeholders about the differentiated Mccann is also actions of our Marty for your gene therapy platform.
Advantages over current therapies.
Speaker Change: Third quarter, we had the opportunity to provide updates on our three clinical stage modify your gene therapies to significant investor audiences as well as industry decision makers during meetings like the cell and gene meeting on the Mesa bolstered by the alliance for regenerative medicine.
Now, let's move on our development.
Shankar Musunuri: Now let's move on to our development in Ocu410 and Ocu410-ST, which aim to treat GA, secondary to D-AMD, and Stargardt disease, respectively, with a single subretinal injection. That could be a one-time treatment for life for patients living with these debilitating blindness disease. Ocu410 is specifically designed to address multiple pathways implicated in the pathogenesis of DAMD and offer a distinct advantages over current treatment options that target only one pathway, the complement system. Currently, approved treatment options require frequent intravitreal injections, about 6 to 12 doses per year, and are accompanied by various safety concerns. For example, roughly 12% of patients develop WIT-AMD following treatment.
Speaker Change: In our Q4, 10, and <unk> SD, which aim to treat GA secondary to AMD and the star got disease, respectively.
Speaker Change: With a single sub retinal injection that could be a one time treatment for life for patients living with this debilitating blindness diseases.
<unk> is specifically designed to address multiple pathways implicated in the pathogenesis of D. M D and offer a distinct advantages over current treatment options.
Speaker Change: <unk> only one pathway the complement system.
Currently approved treatment options required frequent intraocular injections about six to 12 doses per year and are accompanied by video safety concerns for example, roughly 12% of patients develop wet AMD following treatment.
Speaker Change: Our Q4 10 has the potential to regulate all four pathways related to disease progression lipid metabolic some inflammation oxidative stress and the complement system.
Shankar Musunuri: RQ410 has the potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of the disease. With approximately 2 to 3 million GA patients in the U.S. and Europe combined, OcuFortin represents a considerable market that is primed for a new entrance, given the shortcomings with current therapies. Additionally, there is no approved product for GA in Europe. We are currently in Phase 2 of the Phase 1-2 ARMADA clinical trial. and plan to complete dosing in early 2025. A preliminary safety and efficacy update on the RQ410 Phase 1 to Armada clinical trial will be shared at the upcoming clinical showcase next week.
Addressing the underlying causes of the disease.
Speaker Change: With approximately two to 3 million patients in the U S and Europe combined Aki for 10 represents a considerable market that is prime for new entrants given the shortcomings with current therapies. Additionally, there is no approved product for <unk> in Europe.
Speaker Change: We are currently in phase II of the phase one two <unk> clinical trial.
Speaker Change: And plan to complete dosing in early 2025.
Speaker Change: A preliminary safety and efficacy update.
Speaker Change: On the <unk> phase <unk> clinical trial will be shared at the upcoming clinical showcased next week.
Speaker Change: To date nine patients with <unk> have been treated with the low medium and high doses and to argue for 10 phase one study and <unk> demonstrated a favorable safety and tolerability profile to date no serious events SaaS related to <unk> have been reported.
Shankar Musunuri: To date, nine patients with GA have been treated with low, medium, and high doses in the OCUV-410 phase one study, and 410 demonstrated a favorable safety and tolerability profile. To date, no serious events SAEs related to OCUV-410 have been reported. The phase two dose expansion, SSR blinded clinical trial, is recruiting patients and will assess the safety and efficacy of RQ410 in a larger group of patients who will be randomized into one of three groups, a medium dose treatment group, a high dose treatment group, or an untreated control group. Participants must be aged 50 or older, be able to identify 24 letters or more on a BCVA, which is like the charts you read at optometrists' office, and have a total geographic atrophy area between 2.5 and 20.5 square millimeters.
Speaker Change: The phase II dose expansion assessor blinded clinical trial is recruiting patients and we will assess the safety and efficacy of our Q4 10 and the larger group of patients who will be randomized into one of three groups and medium dose treatment group a high dose treatment group.
Speaker Change: Untreated control group.
Speaker Change: Participants must be aged 50 or older.
Speaker Change: <unk> identified 24 letters or more on a B C D, which is like the charge you read at Optometrist office and have a total geographic atrophy area between two five and 25 square millimeters.
Shankar Musunuri: Turning on to Ocufortin-ST, which has received orphan drug designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. Phase one dosing of the phase one to Guardian clinical trial has been completed, and OcuFortin SD demonstrated a favorable safety and tolerability profile. To date, no SAEs related to OcuFot NST have been reported. The Data and Safety Monitoring Board has approved proceeding to phase two of the clinical trial. Sarger disease affects approximately 100,000 people in the U.S. and Europe, and no approved therapy is available. This condition is the most common form of inherited macular dystrophy with symptoms of bilateral central vision loss, typically forming during childhood and gradually worsening over a person's lifetime.
Speaker Change: Joining onto Aki Foughten SD, which has received orphan drug designation from FDA.
Speaker Change: Treatment of ABC year for associated retinopathy, including Star got disease.
Speaker Change: Phase one dosing of the phase one two guardian clinical trial has been completed and argued for Denis Deet demonstrated a favorable safety and Tolerability profile.
Speaker Change: No Ses related to argue for dentists have been reported.
Speaker Change: The data and safety monitoring board has approved proceeding to phase II of the clinical trial.
Speaker Change: approximately 100,000 people in the U.S. and Europe, and no approved therapy is available.
Shankar Musunuri: A preliminary safety and efficacy update on the RQ410ST Phase 1-2 Guardian Clinical Trial will also be featured at the upcoming clinical showcase.
Shankar Musunuri: Lastly, I would like to call attention to our biologic platform, Ocu200, which possesses unique features to treat vascular complications of diabetic macular edema, DME. In recent news, we announced that the FDA cleared the investigational drug application for the phase one clinical trial evaluating Ocu200, a recombinant fusion protein consisting of thumb statin and transferrin for treatment of DME. DME causes blurriness in vision and progressive vision loss as the disease advances. Approximately 746,000 patients in the United States are affected with DME. The condition is becoming more prevalent as the number of people with diabetes in the U.S.
Speaker Change: <unk> <unk> hundred <unk>.
Speaker Change: Recombinant fusion protein consisting of some statin and transfer in for treatment of <unk>.
DMA causes blurriness envision and progressive vision loss as the disease advances approximately 746000 patients in the United States are affected with the Dnb.
Speaker Change: Foundation is becoming more prevalent as the number of people with diabetes in the U S raises making it more competitive to address <unk>.
Shankar Musunuri: rises, making it more imperative to address it. Approximately 30 to 40 percent DME patients are refractive to current anti-VEGF therapies, and we believe that OcuV-200 has the potential to provide a new treatment option for a significant percentage of people living with DME, including non-responders to the current standard of care. We plan to initiate phase one clinical trial of RQ200 this quarter.
Speaker Change: Approximately 30% to 40% DMA patients are refractory to <unk>.
Speaker Change: Current anti VEGF therapies, and we believe <unk> 200 has the potential to provide a new treatment option for a significant percentage of people living with DMD, including non responders to the current standard of care.
Speaker Change: We plan to initiate phase one clinical trial of our Q2 hundred this quarter.
Shankar Musunuri: Our efforts across platforms represent our commitment to treating blindness diseases. focusing on innovative solutions that aim to provide lasting patient benefits. We look forward to sharing further updates as we advance these therapies through clinical development.
Speaker Change: First across platforms represent our commitment to treating Brian this diseases.
Speaker Change: Focusing on innovative solutions that aim to grow a lasting patient benefits. We look forward to sharing further updates as we advance these therapies through clinical development.
Speaker Change: I will now turn the call automation ramachandra to provide an update on our financial results for the quarter ended September 32024 formation.
Ramesh Ramachandran: I will now turn the call over to Ramesh Ramachandran to provide an update on our financial results for the quarter ended September 30, 2024. Ramesh. Thank you, Shankar. The company's cash and restricted cash totaled $39 million as of September 30, 2024, compared to $39.5 million as of December 31, 2023. Total operating expenses for the three months ended September 30, 2024, were $14.4 million and included research and development expenses of $8.1 million and general and administrative expenses of $6.3 million. This compares to total operating expenses for the three months and that September 30, 2023 of $16.1 million that included research and development expenses of $7 million and general and administrative expenses of $9.1 million.
Speaker Change: Shanker, the company's cash and restricted cash totaled $79 million as of September 32024, compared to $39 5 million as of December 31, 2023.
Ramachandra: Operating expenses for the three months ended September 32024.
Ramachandra: $14 4 million and included research and development expenses of $8 1 million and <unk>.
Ramachandra: General and administrative expenses of $6 3 million.
Ramachandra: This compares to total operating expenses for the three months ended September 32023 of $16 1 million that included research and development expenses of $7 million and general and administrative expenses of $9 1 million.
Ramesh Ramachandran: As stated earlier, we recently completed the successful debt financing of $30 million, extending our runway into the first quarter of 2022.
Ramachandra: As stated earlier, we recently completed the successful debt financing of $30 million extending our runway into the first quarter of 2026.
Ramesh Ramachandran: As always, we are proactively exploring shareholder-friendly opportunities to increase our working capital, including partnerships that will drive long-term strategy for our scientific platforms. That concludes my update for the quarter.
Speaker Change: As always we are proactively exploring shareholder friendly opportunities to increase our working capital, including partnerships that will drive long term strategy for a scientific platforms that concludes my update for the quarter Stephanie back to you.
Tiffany Hamilton: Tiffany, back to you. Thank you, Ramesh.
Speaker Change: Thank you Ramesh we will now open the call for questions operator.
Operator: We will now open the call for questions.
Operator: Operator?
Operator: We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again.
Speaker Change: We will now begin the question and answer session. If you have dialed in and would like to ask a question. Please press star one on your telephone keypad to raise your hand and join the queue.
Speaker Change: I would like to withdraw your question simply press Star one again.
Operator: If you are called upon to ask your question and are listening via speakerphone on your device, please pick up your handset to ensure that your phone is not on mute when asking your question. Again, press star 1 to join the call.
Speaker Change: You are called upon to ask a question and are listening via speaker phone device. Please pickup your handset to ensure that your phone is not on mute when asking a question again press star one to join the queue.
Speaker Change: Okay.
Speaker Change: Our first question comes from the line of Jason Mccarthy with Maxim Group. Your line is open.
Jason Mccarthy: Our first question comes from the line of Jason McCarthy with Maxim Group. Your line is open.
Speaker Change: Hi, guys. Thanks for taking my question and congrats on the quarter so at the upcoming clinical showcase.
Jason Mccarthy: Hi guys. Thanks for taking the question and congrats on the quarter. So at the upcoming clinical showcase, we're expecting some of the initial data in GA. Could you just help us understand what we can be looking for from that, the bar for success, and then in particular for gene agnosticism?
Speaker Change: We're expecting some of the initial data.
Speaker Change: Could you just help us understand what we can be looking forward.
Speaker Change: The bar for success and then in particular for gene Agnosticism.
Huma Qamar: Huma? So thanks for the questions. So in the upcoming clinical showcase, we will be presenting the geographic atrophy and our other gene types. Preliminary Safety and Efficacy. Particularly for the GA, we have established the safety at this point. And what we will be showcasing, the efficacy endpoints like geographic atropine lesion and other parameters, would be functional as well as structural. So we will be presenting that data in the upcoming clinical showcase on November.
Speaker Change: Hey, Michael.
Speaker Change: So thanks for the question so in the upcoming clinical showcase we will be presenting the geographic Egypt other gene therapy trials preliminary safety and efficacy, particularly for the G.
Speaker Change: We have.
Speaker Change: Establishing the safety at this point and what we will be showcasing the efficacy endpoints like geographic atrophy lesion and other Panama would be functional as well as structure. So we will be presenting that data in the upcoming clinical showcase on November 12 at Nasdaq.
Speaker Change: Okay, great. Thanks.
Jason Mccarthy: Okay, great, thanks.
Jason Mccarthy: And then for the Stargardt study, when could we expect to see data?
And then for the Star Guard study when could we expect to see data.
Huma Qamar: And then also as a follow-up with a couple small molecule drugs in Stargardt in late-stage development, how do you sort of see those fitting in with OCU410ST if they are approved?
Speaker Change: And then also as a follow up with a couple of small molecule drugs and started up in late stage development.
Speaker Change: How do you sort of see those fitting in with argue for Tennant's key.
Speaker Change: If they are approved.
Speaker Change: Okay. That's a great question currently just to address that there are no approved therapeutic or any treatment approved for <unk>. So there is significant unmet medical need out there.
Huma Qamar: Okay, that's a great question.
Huma Qamar: Currently, just to address that, there are no approved therapeutic or any treatment approved for Stargardt, so there is significant unmet medical need out there. In our upcoming clinical showcase, we will also be presenting the safety as we have already apprised the market to our DSMB press releases, but for efficacy, we will also be presenting that in our upcoming. So stay tuned for all the updates.
Mr. Murray: Our upcoming clinical showcase we will also be presenting the safety as we have already priced the market to our DSM be press releases, but for efficacy. We will also be presenting that in our upcoming clinical showcase so stay tuned for all the updates so Jason I know there are some couple of products in the development.
Huma Qamar: So Jason, I know there are some couple of products in the development. However, you know, we are planning for a treatment option, only treatment option with our. At this time, we're not thinking about any... et al.
However.
We are planning.
Alright.
Mr. Murray: The treatment option only treatment option with our therapy.
Mr. Murray: At this time, we're not thinking about an equal therapies.
Yeah.
Speaker Change: Got it.
Speaker Change: Got it yes.
Jason Mccarthy: Got it, yeah. Thanks a lot.
Jason Mccarthy: And then I just thought I'd bring up, since it's it's topical, if you could just, you know, given the recent issues with, you know, existing gene therapies, Pfizer dropping out, you know, Bluebird coming under fire for safety. Can you just talk a bit about how modifier gene therapies avoid some of those pitfalls impacting the space at large?
Speaker Change: And then I just thought I'd bring up since it's comparable.
Speaker Change: If you could just given the recent issues with.
Speaker Change: Listing gene therapies, Pfizer dropping out.
Speaker Change: Bluebird coming under fire safety.
Speaker Change: Can you just talk a bit about how to modify our gene therapies avoid some of those pitfalls impacting the space at large.
Speaker Change: Yeah, I mean, good question, Jason I think two things that we need to consider when you are.
Shankar Musunuri: Yeah, I mean, good question, Jason. I think two things I think we need to consider when you're doing any gene therapies, first and foremost, product quality is extremely important. And during, you know, the course of last few years with the three parallel clinical trials going on, in ophthalmology space, probably we have the biggest ophthalmology clinical trials in gene therapy. We have not seen any adverse events, SAEs related to our product. The product seemed to be safe and effective. Therefore, that's number one. Quality of the product is very important. We believe we are consistently making high quality.
Speaker Change: Doing any gene therapies first and foremost product quality is extremely important.
Speaker Change: And during.
Speaker Change: The course of last few years.
Speaker Change: Three parallel clinical trials going on.
Speaker Change: Ophthalmology space, probably we have the biggest ophthalmology clinical trial in gene therapy space.
Speaker Change: We have not seen any adverse events.
Speaker Change: Is it related to our product the product seem to be safe and effective.
Therefore.
That's number one quality of the product is very important we believe we are consistently making high quality products number two we target sub retinal surgeries to get these gene therapy.
Shankar Musunuri: Number two, we target subretinal surgeries to get this gene therapy into retina. And so we do have highly skilled, experienced retinal surgeons in our network. And we formed a very good network of these surgeons for all our clinical trials. So that's very important.
Speaker Change: At dinner.
And so we do have highly skilled experienced retinal surgeons in our network and we found a very good network of resurgence for all our clinical trials. So that's really important the procedure is important.
Shankar Musunuri: The procedure is. So those are the two things we're focusing on. And obviously, there are also differences. I just wanted to point out for listeners, systemic gene therapy versus, you know, ophthalmology therapy and our target dose typically in ophthalmology space, because we. directly go into the target and now goes from 10 to the 10 to the 10 to the 11 gene copies. Systemic gene therapy is going to 10 to the 14 and higher. And so there's a significant difference, almost like a thousand fold or 10,000 fold increase. And so there are other things come up when you go to systemic gene therapy.
Speaker Change: Those are the two things we're focusing on and obviously there are also differences I just wanted to point out for listeners.
Speaker Change: Systemic gene therapy versus ophthalmology gene therapy.
Speaker Change: Those typically in ophthalmology space because we.
Speaker Change: Directly go into the targets and our growth from Canada down to the 10 to 11 gene copies systemic gene therapy is going to jump to the 14 and higher.
Speaker Change: And so theres a significant difference almost like 1400 10000 fold increase and so there are other things come up when they go to systemic gene therapies.
Jason Mccarthy: Okay, thanks.
Speaker Change: Okay. Thanks that was helpful.
Jason Mccarthy: That was helpful.
Speaker Change: Okay.
Speaker Change: Our next question comes from the line of Danielle <unk> with Chardan. Your line is open.
Daniil Gataulin: Our next question comes from the line of Daniil Gataulin Wichardon. Your line is open. Good morning, guys. Thank you for taking the question and congrats on the progress.
Hey, good morning, guys. Thank you for taking my question and congrats on the progress.
Daniil Gataulin: I have one question on geographic atrophy programs. Given there are still no approved options for it in Europe, how are you thinking of taking advantage of the opportunity? And have you had any interactions with the regulators regarding your program?
Speaker Change: I have one question on geographic atrophy.
So given there are no still no approved options for us in Europe.
Speaker Change: What are you thinking of taking advantage of the opportunity.
Speaker Change: Have you had any interactions with the regulators regarding your program. Thank.
Huma Qamar: Thank you.
Speaker Change: Thank you.
Dennis: Great question Dennis.
Huma Qamar: Great question, Daniil. Yes, we will start interactions this year, I mean, the next few quarters with the EU. Obviously, the phase two is limited to US. What EU regulators are looking for is some functional improvement or stabilization for geographic atrophy patients. The current therapies which are on the market in the US, they use structural endpoints to get approval. So we'll be definitely looking for functional endpoints, and some of the data will be shared next week.
Dennis: Yes, we will start interactions.
Dennis: This year I mean, the next.
Dennis: A few quarters with the EU, obviously, the phase III is limited to the U S. What EU regulators are looking for is some functional.
Dennis: Improvement or stabilization.
Dennis: For geographic atrophy patients, although current therapies, which are in the market in U S. They used structural endpoints to get approvals.
Dennis: It will be definitely looking for functional endpoints and some of the data will be shared next week natural pace.
Daniil Gataulin: Got it.
Got it.
Daniil Gataulin: And a quick follow-up on the 410SP program. Do you see, given it's a rare disease, do you see any potential of modifying your Phase 2 trial or Phase 2 portion of the trial to make it a pivotal?
Speaker Change: Quick follow up on the form 10 S T program.
Speaker Change: Do you see given given it's a rare disease this into potential of modifying our phase two trial of our phase two portion of the trial to make it a pivotal.
Huma Qamar: Yeah, that's a great question.
Speaker Change: Yeah. That's a great question, that's why after phase one clinical trial, we are taking a pause even though the SMB.
Huma Qamar: That's why after phase one clinical trial, we are taking a pause, even though DSMB gave approval to move forward with the phase two with the previously designed study approved by FDA. We're going to take a pause. We're going to spend a few months for discussions with the FDA because there are some new guidance.
Gave approval to move forward into phase II with the previously designed study approved by FDA, but I'm going to take a pause but are going to spend a few months for discussions with FDA.
Speaker Change: There are some new guidance there is an opportunity for orphan diseases with gene therapies to convert phase III pivotal trial.
Huma Qamar: There is an opportunity for orphan diseases with gene therapies to convert phase two into a pivotal trial for registration trials. So we're going to closely work with agency and seek their advice and guidance before we move forward to phase two slash confirmatory.
Speaker Change: For a registration trial, so we're going to closely work with agency and seek their advice and guidance before we move forward to phase II slash complementary trial.
Daniil Gataulin: Gata, thank you, and again, congrats on the progress.
Speaker Change: Got it thank you and again congrats on the progress.
Speaker Change: Thank you.
Operator: This concludes the Q&A portion.
This concludes the Q&A portion I will now turn the call back over to chairman and CEO and co founder Dr. Shankar machinery.
Shankar Musunuri: I will now turn the call back over to Chairman, CEO, and Co-Founder, Dr. Shankar Musunuri. Thank you, operator. We appreciate the continued interest and involvement of our key stakeholders as we move forward with our transformative initiatives.
Speaker Change: Thank you operator, we appreciate the continued interest and enrollment of our key stakeholders as we move forward with our transformation initiatives. We look forward to closing a successful fourth quarter of 2024, as we continued to solidify <unk> position as a biotechnology leader in ophthalmology.
Shankar Musunuri: We look forward to closing a successful fourth quarter of 2024 as we continue to solidify Ocugen's position as a biotechnology leader in ophthalmology.
Operator: Have a great day.
Speaker Change: Dave.
Speaker Change: Yes.
Operator: Ladies and gentlemen, this concludes today's conference call. You may now disconnect.
Speaker Change: Ladies and gentlemen. This concludes today's conference call you may now disconnect.
Speaker Change: [music].
Speaker Change: Yes.
Speaker Change: Okay.
Speaker Change: [music].
Okay.