Q1 2025 Lineage Cell Therapeutics Inc Earnings Call
Speaker Change: Welcome to the Lineage Cell, therapeutics, first quarter, 2025 conference call. At this time, all participants are in a listen only mode.
Speaker Change: An audio webcast of this call is available on the Investors section of Lineage's website at www.lineagecell.com. This call is subject to copyright and is the property of lineage.
Speaker Change: and recordings, reproductions, or transmissions of this call without the express written consent of Lineage are strictly prohibited.
Speaker Change: As a reminder, today's call is being recorded. I would now like to introduce your host for today's call. I want a home head of investor relations at Lineage. Miss Hone, please go ahead.
Ioana Hone: Thank you, Carly. Good afternoon, and thank you for joining us. A press release reporting our first quarter 2025 financial results was issued earlier today, May 13, 2025, and can be found on the investor section of our website.
Ioana Hone: Please note that today's remarks and responses to your questions reflect management's views as of today only, and will contain forward-looking statements within the meaning of federal security's laws.
Ioana Hone: Statements made during this discussion that are not statements of historical fact should be considered forward-looking statements which are subject to significant risks and uncertainties.
Ioana Hone: The company's actual results or performance may differ materially from the expectations indicated by such forward-looking statements.
Ioana Hone: For a discussion of certain factors that could cause the company's results or performance to differ, we refer you to the forward-looking statement sections in today's press release and in the company's SEC filings, including its most recent annual report on Form 10K and in subsequent SEC filings.
Ioana Hone: We caution you not to place undue reliance on any forward-looking statements, which speak only as of today and are qualified by the cautionary statements and risk factors described in our FCC filing.
Ioana Hone: With us today are Brian Culley, Archief Executive Officer, and Jill Howe Archief Financial Officer. I'll now hand the call over to Brian .
Brian Culley: Thank you Iwana and good afternoon everyone. I appreciate you taking the time to join us on the call.
Brian Culley: I'll start off today with an update on Pantgin and talk about some interesting results reported at the recent ice salarator and Arvo Conferences.
Brian Culley: I also want to ensure everyone is aware of the forthcoming 36-month clinical data which will be presented next month at the CTS meeting.
Brian Culley: I have a brief but important update on a recent manufacturing milestone that gives us the opportunity to broaden our business strategy.
Brian Culley: And after that, I'll say a few words about the DOST study of OPC-1 before Jill reviews our financials and we'll leave plenty of time today for analyst questions.
Brian Culley: Starting with the offergen, this is an allergenetic RPE cell transplant intended for patients suffering from dry AMD with geographic atrophy.
Brian Culley: Unlike the currently approved anti-complement therapies, which require monthly or semi-monthly injections, and only slow disease progression by around 20%.
Brian Culley: Arprigin has shown evidence of both anatomical and functional benefits lasting for up to two years and from a one-time surgical delivery of RPE cells to the sub retinal space.
Brian Culley: Importantly, the benefits we've seen today have not been observed to occur spontaneously in the natural course of the disease, which makes their presence increasingly meaningful with each passing year.
Jill Howe, Brian Culley
Brian Culley: Based on the totality of the data collected to date, we believe there's a tremendous opportunity to provide better outcomes for GA patients than from what is available from existing therapies.
Brian Culley: Opportunity is currently being evaluated in a phase 2A study called the Gillette study that is being paid and run for by our partners, Prof. Genentech.
Brian Culley: The GLET study is exploring various parameters of surgical delivery to help enable a safe and repeatable procedure.
Brian Culley: Overall, the goal of the GLEP study is to refine the surgical procedure and optimize the risk-benefit profile.
Brian Culley: We believe the ongoing Galette study is progressing well because it is an open label study with 90 day endpoints and it has been enrolling continuously for approximately two years.
Speaker Change: Well, we do not have access to data from that trial.
Speaker Change: We note that Genentech has continued to invest in the program by, for example, seeking and obtaining ARMAT designation, opening additional clinical sites, and acquiring proprietary surgical delivery devices designed for delivery to the sub-radinal space.
Speaker Change: These positive steps have occurred against the background of Roche reducing its pipeline, which adds further conviction to our belief that the offer is delivering promising results in this open-label study.
Speaker Change: I want to remind everyone that there is no timeline available yet for a decision by Genentech whether to advance operative into a controlled study.
Speaker Change: We don't know how such a decision would be made or communicated, but we do know there's no regulatory requirement to complete the ongoing GLEP study or announce any GLEP data before initiating such a controlled study so it's possible a larger controlled study could be announced while the GLEP study continues.
And as a reminder,
Speaker Change: Oppregion to the pioneering new technology for which delivery is an important component.
Speaker Change: So we expect that at least some aspect of surgical optimization could continue for a long time.
Speaker Change: At the same time, we're encouraged that Genentech could elect to advance to offer to the next stage of development whenever they're comfortable doing so. And until such a decision is made, we will continue to await further updates from our highly capable partners.
Speaker Change: Well, we all await information from the ongoing Phase 2A, or any subsequent trials of the two additional things I'd like to highlight.
Speaker Change: The first is that Genentech plans to report three-year data from the Phase 1-2A trial which Lineage conducted.
Speaker Change: and which will be presented on Genentex behalf by Dr. Chris Reeman from Cincinnati Eye Institute.
Speaker Change: That presentation will occur to clinical trials at the Summit 2025 Conference on June 21st.
Speaker Change: TTS was founded by Dr. Shad Kanani, Director of Clinical Research at Sierra, I associate, and the event brings together a diverse and global group of experts to discuss the latest data for ongoing clinical trials.
Speaker Change: Dr. Reeman was an investigator on the phase 1, 2, a study of operative and we greatly appreciate his availability to present this new data for Genentech.
Speaker Change: The reason this forthcoming data is important is that the anatomical and functional benefits that were previously reported out to two years following operogen treatment were notable.
Speaker Change: In comparison, the evidence from large clinical trials and natural history registries clearly show that untreated patients or patients on anti-complement therapy continue to lose vision
Speaker Change: As we consider what happens after a third year, we believe any visual benefits or disease stability observed at year three will be even more compelling than at year two.
Speaker Change: because it indicates the difference in visual performance between an operogen treated patient and a patient even on the best available therapy could be widening over time.
Speaker Change: Furthermore, a three-year benefit adds additional support to operative potentially being a one-time treatment which could mitigate compliance issues and we believe what offers striking advantage over the monthly injections required for best available outcomes with anti-complement
Speaker Change: We haven't seen the three-year data from Genentech yet, but we're hopeful it will provide additional conviction in our approach.
Speaker Change: Speaking of conviction, the second offered item I wanted to highlight today is recent news and data updates from competing RPE companies which I think is extremely positive for us.
Speaker Change: Recent Phase I data, disclosed by two separate companies at ARVO last week, provides independent validation that indeed an RPE transplant can drive clinical outcomes well beyond the reach of currently approved therapies.
Speaker Change: Even more importantly, those benefits included indications of a large functional visual benefit with one-time dosing, which supports the treatment regimen that we've envisioned for
Speaker Change: Another reason this is important is that an RPE cell tramped flat.
is a completely new modality and the only approach.
Speaker Change: Credibly demonstrating the potential to improve vision and GA patients, so it's normal for people to want to see more clinical data than Lineage has generated today. And so every time some other company independently reports positive data from an RPE transplant
Speaker Change: Those data points can be aggregated into an increasingly powerful endorsement of our approach.
Speaker Change: At this point, we are aware of four independent reports of functional gains from an RPE transplant.
Speaker Change: From among those companies, we assume investors will try to sort out which among us is best position to win the large addressable and increasingly established GA market.
Speaker Change: Given everything that is required to successfully manufacture and commercialize an allergenic cell therapy product for such a large patient population.
Speaker Change: I feel confident that the three pillars of one, Lineage's manufacturing and scale-up process
Speaker Change: Two, Genentex Device and Drug Development Capability, and three, Rosh's leadership in Ophthalmology Product Commercialization provides us with advantages over competing RPE programs.
Speaker Change: Overall, I'll say that we continue to believe these actions reflect things moving in a very positive direction with the [inaudible]
Speaker Change: I just mentioned Lineage's manufacturing and scale-up capability as one of the three pillars which contribute to a competitive advantage. I want to take just a moment to explain again why I see so much value in our manufacturing platform.
Speaker Change: When comparing allergenic and autologous cell therapies, it's important to recognize.
Speaker Change: But the key advantage of Allagenetic is that it offers the possibility of low-cost manufacturing, of a stable and consistent material with no donor variability and a true off-the-shelf user profile.
Speaker Change: That is, unlike the typically expensive autologous therapies which are made for a single patient and often include complex handling and or logistical and transportation requirements.
Speaker Change: Today, we haven't found any examples of what we consider to be a true allergenic manufacturing process, meaning one which fulfills the intent of low cost and consistent production from a stable and unchanging source of cells.
Speaker Change: We believe these features are a necessary requirement to supply material to a large patient population, but it's difficult to find evidence of anyone satisfying this requirement.
Speaker Change: So, last month we revealed that on two separate occasions we have completed a GMP production run of a product candidate from a single pluripotent cell line.
Speaker Change: which had been the source of a GMP master and GMP working cell bank system.
Given the exponential expansion available from our dual banking system,
Speaker Change: We believe we are the first in our field, specifically in the field of non-cancer allogeneic cell transplantation.
Speaker Change: to have reduced to practice a GMP cell-based production system that is capable of producing millions of doses of product from a single starting cell line.
Speaker Change: and we have also demonstrated that our system is not product or cell line specific because it was performed with more than one cell type and with two different cell lines.
Speaker Change: We see this as a major achievement for our business because generating a GMP banking system that can deliver large-scale and low-cost production of a self-therapy opens up new opportunities for partnerships using cell types not currently in our pipeline.
Speaker Change: And in fact, around the time of disclosing this achievement, we were able to initiate a number of additional partnering discussions.
Speaker Change: And while these new opportunities are still just early stage for now, the willingness of others to open discussions is partly the result of us being able to point to this recent cell-based manufacturing capability.
Speaker Change: The business implication of all this is that if Genentech moves oxygen into a comparative trial we believe we will enjoy not only a lower cost of capital
Speaker Change: But also a strong rationale to repeat our success with additional cell-based programs beyond offergen and OBC1 and ANP1 currently in our pipeline
Speaker Change: For this reason, we believe entering into additional deals can offer an increasingly attractive non-delutive opportunity to expand our business model and create long-term value.
Speaker Change: Moving on, a next we'll provide an update on our second clinical stage, allergenic cell transplant program, OPC-1, which is designed to help increase recovery and mobility for people who have suffered from a spinal cord injury.
Speaker Change: Our approach is to manufacture replacement cells of the spinal cord and deliver them to the site of injury, which is basically the same mechanism that has produced exciting outcomes in dry AMD.
Thank you for tuning in.
Speaker Change: OPC-1 has been tested in 30 individuals with severe spinal cord injuries and the long-term safety and efficacy data we have collected so far is promising and merits further investigation.
Speaker Change: But we have two areas of improvement we want to complete before we would feel ready to move OPC-1 into a later stage trial
Speaker Change: The first area of improvement is delivery. We have initiated a small clinical study called the DOST study to test the safety and performance of a novel delivery device in 6 to 10 patients.
First, it's easier to use and deploy the clinical sites [inaudible]
Speaker Change: But more importantly, second, the new device allows a dose of cells to be administered over four to five minutes without stopping the patient's respiration.
Speaker Change: Previously, it was necessary to stop ventilation when you delivered the cell, so this is a significant enhancement to the procedure.
Speaker Change: This is important because this study will be the first time OPC-1 will be administered to patients with a chronic spinal cord injury.
Speaker Change: Chronic SEI represents an additional and larger potential patient population for this experimental therapy.
Speaker Change: The first clinical site in the dose study is just down the road at UC San Diego Health and we anticipate that UCSD can begin identifying patients for screening this week and allow us to work toward our goal of enrolling our first patient in the dose study in June .
Speaker Change: The second area of OPC-1 improvement is our new manufacturing process, which actually has to subparts.
Speaker Change: We increase the scale, purity, and control of the cells we make, and second we developed a new immediate use formulation which eliminates the lengthy dose preparation steps that were required with the original clinical material.
And I'm Brian Culley. Thank you. Thank you.
Speaker Change: Subject to obtain a clearance from FDA. We plan to introduce the cells we make from this improved process and the new formulation into the ongoing dose to trial.
Speaker Change: In parallel, with these two ongoing enhancements to the OPC-1 program, we also are working on the design of a larger trial with a focus on collecting more specific and clinically relevant end points.
Speaker Change: with a goal of reducing the well-documented challenges of data capture in this patient population.
Speaker Change: When all of these necessary activities have been concluded, the design, the device, and the cells, we believe we, either alone or with a future partner, will be in a position to conduct a larger trial of OPC-1.
Speaker Change: Meanwhile, we still plan to apply for a SRM clinical grant as soon as they begin accepting applications which SRM has indicated should occur fairly soon.
Speaker Change: And lastly, we recently announced that we are proudly collaborating again with the Christopher and Dana Reeve Foundation on the third annual SCI Investors Symposium.
Speaker Change: We've decided to convert this to a virtual conference because that can significantly increase participation for individuals who have difficulty within person events and broaden overall participation
Speaker Change: This year's symposium will be held on last year's momentum and will be available online June 27th, 2025.
Speaker Change: And with that, I will turn things over to Jill for a review of our finances.
Thanks, Brian , and good up to you and everyone.
Jill Howe: As of March 31, 2025, our overall cash position was $47.99.00 [inaudible]
Jill Howe: This capital is expected to support our planned operations into Q1 of 2027, which we believe will allow us to reach multiple events and milestones.
Jill Howe: As we discussed in detail on our last call we continue to have approximately $37 million of warrant capital which could become available to us.
Jill Howe: We continue to pursue and investigate additional sources of funding, such as milestone payments we are eligible for under the Roche Genentech Collaboration Agreement, program grants like the Sturm Grant and additional collaborations which we may elect to enter into.
Jill Howe: With respect to our first quarter financials, we largely came in line with our historic periods.
Jill Howe: Total revenues for the quarter were 1.5 million, a net increase of 0.1 million, compared to 1.4 million, for the same period in 2027. This increase was primarily driven by more collaboration revenue recognized from deferred revenues under the collaboration and license agreement with Roche.
Jill Howe: Operating expenses for the first quarter of our 8 million, a decrease of 0.1 million, as compared to 8.1 million, for the same period in 2024.
Jill Howe: R&D expenses for the first quarter were $3.1 million, an increase of $0.1 million is compared to the same period in 2024.
Jill Howe: The net increases primarily driven by 0.2 million for our creep medical programs, partially offset by 0.1 million for other research and development programs.
Jill Howe: GNA expenses for the first quarter were 4.9 million, but primarily in line with expenses for the same period in 2024.
Jill Howe: Laws through operations for the first quarter was 6.5 million, a decrease of 0.2 million as compared to 6.7 million for the same period in 2024.
Jill Howe: Other income for the first quarter was 2.4 million compared to 2.1 million, but the same period in 2024 and the net increases primarily driven by changes in the fair value of the warrant liability.
Jill Howe: The net loss for the first quarter was 4.1 million or 2 cents per share as compared to a net loss of 6.5 million or 4 cents per share for the same period in 2024.
Jill Howe: As you can see from our financial results, we continue to demonstrate our commitment to fiscal discipline and strike an appropriate balance between our cost of capital and investments we make in our program.
With that, I'll hand the call back to Brian [inaudible]
Brian Culley: Thanks, Jill. So, I will summarize today by saying that we remain confident in the potential for offer-gen to drive positive clinical outcomes in dry AMD and are encouraged by our partners' continued signs of commitment to the program.
Speaker Change: We also believe the independent evidence generated by other RPE cell transplant trials supports and elevates the evidence and we look forward to the three-year clinical data update coming from GenNFAC next month.
Speaker Change: Second, we're planning for success by seeking to capitalize on our investments in cell manufacturing and use recent achievements as a new foundation from which additional programs can be advanced by a funded partnerships and independently.
Speaker Change: And third, we are steadily advancing OPC-1 toward the goal of conducting a planned larger clinical trial in spinal cord injury while ensuring first that the necessary attributes for long-term success are in place with that program.
Speaker Change: As always, we appreciate your support and belief in our vision with that operator we are ready to take any questions from analysts.
Speaker Change: At this time I would like to remind everyone in order to ask a question, press star, then number one on your telephone keypad. We'll pause for just a moment to compile the Q&A roster.
Your first question comes from at my ink. Montami would be Riley's securities.
Speaker Change: In terms of manufacturing to peers and how much of a differentiation you feel that you have in scale-up.
Speaker Change: You know, potentially having redundancies and perhaps even supplying for the demand that
Speaker Change: Yeah, hi, William. So I can speak broadly about our capabilities, but I won't speak to any particular programs.
Speaker Change: but actually it's quite frustrating that we can't easily make direct comparisons. Unfortunately,
Speaker Change: The majority of our peers don't actually describe in sufficient detail exactly what they're capable of. There are some exceptions, some companies have the confidence to explicitly state that they have not.
Achieved this level of capability, but most of them, I think...
Speaker Change: Appeared to evade a difficult question, and that's okay because we know that it is difficult. We've been working on this kind of technology for 20 years, so I do believe it's highly differentiated.
Speaker Change: because of the capability, the learnings, the experience, only a portion of which is subject to patent applications or issued patents. There are a lot of things we've learned that we retain as know-how.
Ultimately,
I think that as-
Therapy, Mature,
and investors look beyond.
Speaker Change: The initial and sometimes exciting phase 1 data and start asking difficult questions about being able to supply material affordably
Speaker Change: to a large patient population, and noting that in some cases that material itself in a single dose
May be millions, tens, or even hundreds of millions of cells.
Speaker Change: One is going to be increasingly forced to demonstrate a production capability from a platform that can deliver that kind of scale.
Speaker Change: And so, if you permit me to just give an illustration,
Speaker Change: And any one of those 100 vials could make a working cell bank of 100 vials.
Speaker Change: and then any one of those vials could make, let's say, a hundred doses of your treatment.
You have been in hand.
Speaker Change: A manufacturing system that's capable of making 1 million vials of your product without going back and resetting your original cell line without changing your process, etc. So I want to be clear that we don't have millions of vials of any of our therapies frozen away waiting to be utilized.
Speaker Change: But what we have done is we have reduced to practice in a GMP environment.
Speaker Change: That system, or that platform, and we've done it with multiple cell lines, and we've done it for multiple cell types.
Speaker Change: So, I do hope that others do come forward so that we can make direct comparisons, we welcome the challenge, but right now we believe we stand alone with this capability at this time.
Speaker Change: That's very helpful. And then just sort of taking a step out and just looking at some of the dynamics that we've seen but the recent with the current administration just thinking about tariffs. You have a manufacturing site in Israel. I was just kind of curious what type of potential tariff impacts we might expect? Yeah.
Speaker Change: and how those might be mitigated if, depending on, you know, the degree of tariffs that we might see come, obviously a little bit of speculation there.
Yo, Brian . I'm happy to take that.
Speaker Change: Question, you know, in general with our manufacturing site in Israel, we don't expect to experience any type of tariff impact. We do however purchase materials way in advance and are able to throw them on site to ensure that we can properly mitigate any type of production issues.
Speaker Change: Yeah, and if you'll permit the William to add, there are a couple other things that are, um...
Speaker Change: Floating in the ether these days, we're really encouraged by comments that the new Commissioner of Marty McCarry has said about cell and gene therapies holding tremendous promise. I think that's a direct quote.
Speaker Change: and I think that the even more recent discussion around MFN simply highlights how important it is to have really wide margins if there's cost pressures and pricing pressures.
Speaker Change: Being able to manufacture your material very affordably will become increasingly important in order to be protected from some of those constraints.
Speaker Change: And we're going to be talking about the the the the the the the the the the the the the the
Speaker Change: I appreciate that extra color. We'll have back in the queue, really appreciate you answering our questions and congratulations on a very productive quarter. Thank you.
Thank you, William.
Your next question comes from Jack Allen with Baird.
Jack Allen: Alright, thanks so much for taking the questions and congrats on all the progress.
Jack Allen: I guess I'll start with one on the manufacturing side of things as well. It seems like you believe you develop a unique capability here and it sounds like you've already seen some interest as it relates to partnerships.
Jack Allen: I'd love to hear a little bit more of your thoughts on how central deals could take shape. You know, is it a technology that you look at out like since, or is it something that you look to kind of utilize as a platform and you know, take small pieces as an enabler of other people's products?
Jack Allen: How are you thinking about the potential partnership formation around this new outcome here?
Speaker Change: Thank you for that question, Jack. I think each opportunity will be specific to itself.
Jack Allen: I think as a general matter, we are not aspiring to become a CDMO.
Jack Allen: We're not aspiring to do some sort of contract fee-for-service manufacturing for others. However,
I think this new capability and being able to...
Jack Allen: Show some clear evidence of that capability, meaning, you know, actual specific file counts and etc.
I think that the-
Jack Allen: The ultimate scope of what a deal, or multiple deals would look like, I think from our perspective,
Jack Allen: Part of the answer is that it's prudent to have a balance or a mix of different deal types. I think that offers diversity and optionality to us as an innovator, but it takes two to strike a deal.
Jack Allen: and it's going to depend on what the scope and need of potential partners is going to look like.
Jack Allen: But what we're most encouraged by is an ability to be able to speak with people not about some prophetic aspiration that we have.
Jack Allen: But, being able to, again, point to a specific example or two where we've achieved something and so perhaps we would do some pilot work.
Jack Allen: Look beyond just a program like OPERGIN and think about how we can expand our capabilities and our success into other areas and own pieces of that action and those other assets.
Jack Allen: in other disciplines where, frankly, we would not be experts in all of those different disease states, but we could be experts in direct differentiation and process development.
Speaker Change: Got it, that's very helpful. And then, as it relates to the OPC-1 program, it seems like you've made some real progress there, and that you're hoping to have the first patient enrolled.
Speaker Change: in June . What might be to expect data from that program? And then a bit of an aside here, but there's been a recent kind of spotlight on the space with 60 minutes highlighting onwards.
Speaker Change: You know, device technology in spinal cord injury. How do you expect OPC-1 to play in with potential device innovations as you know, I know onward has theirs and Neuralink is also working on a device for this population as well.
Speaker Change: Yeah, thank you. I had an opportunity to watch the 60-minute piece. I thought it was almost as good as the CNN piece that included OPC-1 and the recent interview with Dr. Sanjay Gupta.
Speaker Change: The nice advantage of OPC-1 is that it really could partner well with other technologies.
So whether those technologies are-
Electro-stimulation-based
Speaker Change: Mechanical-based, technology like Neuralink, or precision that have brain chip interfaces. All of these things could partner with OPC-1, and I do think that many in the field believe that maximum clinical outcomes.
Meaning The Greatest
Improvements to Mobility and Quality Life
Four Patients
But if we can utilize, for example,
Someone has an injury, maybe there's a procedural...
Step that's beneficial immediately following an accident. Maybe there's...
Some new insights regarding mobilization or immobilization.
Speaker Change: and then maybe shortly thereafter a patient is receiving a transplant of OPC-1, and then maybe they're going into a really well-refined and patient-specific physical therapy course.
Speaker Change: and then maybe they're getting aided by either some stem or some mechanical devices and maybe they ultimately have a chip implanted so they can really maximize the gains provided by those prior modalities.
Speaker Change: Devastating by all of us working together, which is one of the reasons we've pulled together this conference is to imagine what it looks like. So, I think that we're not...
Speaker Change: Compete with those modalities or displays them. I think it's exactly the opposite. I think OPC one can make those modalities better and vice versa. And that's probably the best and highest use of a cell transplant in spinal cord injury.
Speaker Change: That's great, Brian , sorry, to seem more pointed to you. Do you have a sense for when we could get initial data from that dose study of OPC-1?
Speaker Change: The dose study is an open label study, the primary endpoint regarding the success and safety of delivery occurs at 30 days, so I imagine that we would be forthcoming on a regular basis.
Speaker Change: from that Open Label Study. The other component of it is functional assessments.
Speaker Change: Those would necessarily lag. A typical functional assessment would be ticks or maybe more likely 12 months after administering a therapy. So there would probably be sort of two waves of information. The first affirming for investors that are decision to move to this new device.
Speaker Change: that that device is being deployed successfully without unusual adverse events. That would be important announcement. And then I think lagging to that by you know several quarters or more.
Would be any information regarding functional changes in patients unless...
Speaker Change: Those functional changes were extremely striking and notable on a standalone basis.
Speaker Change: It was approximately nine months before we disclosed that to the public because it really is...
Speaker Change: and Astounding Achievement. And we wanted to make sure it was authentic and verified by experts in the field before we went public with those claims. So if we did see something extraordinary in particular with one of the chronic patients,
Speaker Change: You can have some assurance that we would carefully assess that before making it public so as to not get out in front of any excitement too early.
Speaker Change: Great color and congratulations again on all the progress. Thanks so much.
Thank you, Jack
Your next question comes from Michael Okunewitch with Maxim Group.
Thank you.
Michael Okunowicz: Brian , thank you so much for taking my questions today, rats on all the progress.
Thank you, Mike.
So I guess it's a start on the dose study.
Speaker Change: Do you just help us understand what a successful delivery outcome would look like? Is this...
Speaker Change: Pretty much all safety you're looking for different adverse events or there's some metrics that would demonstrate successful delivery and then is there a benchmark that we should be looking at to compare to the prior delivery method.
Speaker Change: Thank you, Michael. It's a very good question. Importantly, we are initially using the same material
Delivered in the same way by the same needle.
Speaker Change: So, many aspects of this, including importantly aspects of it that are internal to the patient, are identical to what was seen before, so we do have a reference point, we do have something
Speaker Change: The external components are all about how that needle is held and the apparatus, the physical apparatus is attached to the patient, which as I described in my remarks.
Speaker Change: Includes the ability to avoid the cessation of respiration and allows us to deliver the cells while the patient is still connected to the respirator and, you know, having supported breathing.
Speaker Change: I do actually think it's a fairly low bar with relatively low risk because the material is the same, the needle is the same, the process is the same, the process meaning that you know where you're aiming your target destination and the dose.
So, I do think that success for us...
Speaker Change: Is largely going to be pointing at the exterior equipment? Is it easy to use? Are there any unexpected complications?
Speaker Change: which pertains to the use of the equipment more so than A.E.'s pertaining to the patient.
Speaker Change: for the reasons I just shared. So I think that really this is about enabling a better superior method of surgical delivery which can be more easily deployed into a large number of clinical sites.
so that a larger clinical trial can be performed.
If we were to utilize the original-
Surgical Delivery Apparatus
Speaker Change: It's very complicated, it's very unwieldy and frankly none of us want to go into a phase two or other design.
Speaker Change: Using that first-generation equipment when we have the ability to very quickly in a small number of patients test a second-generation device which is more easily deployed. We're talking about something about the size of a shoe box.
Speaker Change: So you can imagine comparing that with something that's the size of, you know, a dresser drawer with all of the packaging and goes with it. So it's a pretty low bar, but it affords us a very nice upgrade with respect to usability and convenience and deployment in multiple sites.
Thank you for that. And then...
Speaker Change: You did mention a new immediate use formulation. Is this different from the Thine and Jack's formulation and how solid it is?
No, I often use those terms interchangeably.
Speaker Change: I think going back to the first question that I think my aunt was asking.
Ways That People Prepare and Utilize Cells Are Important Considerations So...
We're aware that the original material that was used
Speaker Change: Patented and Employed, A Thought and Inject Formulation, which allows that product candidate to be immediate use. And for us...
Speaker Change: The reason why this is important is that many of the self therapies...
Speaker Change: Every where you have a step, you have cost, you have tracking, you have the potential for errors.
and using our thought and inject formulations.
Speaker Change: It gives us a greater level of convenience, it reduces cost, and we've even had discussions about people who wanted to see if they could use it for their program, so we've had people who have been asking us questions about licensing and using that technology, so thank you for the question. Again, I mean it used, and Sean and Jack, I think I typically use those interchangeably.
But the point here is to avoid...
Speaker Change: The manipulation and the handling of a complex biologic at the bedside and instead being able to store for many many years this material and use it on demand which obviously helps with the entire supply chain.
Speaker Change: Yes, thank you. I appreciate the additional clarity as well as the additional commentary.
Speaker Change: One more for me if you don't mind and then I'll hop back into the queue. I just wanted to see if the inclusion of the two surgical devices to Rochis' operagen study. Is this something new that Rochis doing?
Speaker Change: Yeah, thank you. That was a small comment I made at the recent Icelerator meeting.
Speaker Change: Note that they were planning to evaluate two proprietary surgical devices. One of those is a dual lumen trans vitriol, sub retinal injection. The other is a next generation.
Speaker Change: Orbit SDS, you may recall that Lineage utilized the first gen, Orbit SDS.
Speaker Change: The reason one of several reasons I think this is important is these are proprietary, so I don't particularly feel like I need to convince anyone that we're the leader in RPE transplanting but
Speaker Change: If you consider that there's a surgical optimization study being conducted and that if there are any improvements
Speaker Change: and Findings and Surgical Delivery. If those are done with a proprietary device, that is just making the moat between us and others that much larger. I feel like it's already quite significant but this is exactly the original vision. This is what we...
Speaker Change: One of the things we had hoped for through the Alliance with Roshnchenentek is they are bringing to this program advanced surgical delivery, know-how tools.
Speaker Change: Technology and ability to test that are beyond what Lineage was prepared or capable of doing at that time. And I think if they are able to increase the risk benefit of that program even farther, it just makes it all the more appealing. So that's what I was able to speak at publicly at that ICelerator conference just a week or two ago.
Speaker Change: Yes, thank you. It's certainly encouraging to see the level of optimization they're putting into the program.
Thank you, Mike.
Speaker Change: Hi, um, maybe just to follow up from the previous question and comments, I was just wondering if you can maybe give us a little more color, I guess, on the potential advantages of these new devices, whether maybe they aim to be a little safer or somehow, I guess apply this as a little better and yeah, whatever else you can share, be interested to hear.
Yeah, thanks, Albert.
Speaker Change: A little bit. The Duolumen Transvitrial, that enables delivery of a saline pre-blapped, followed by the operagen suspension through a simplified procedure and one with a single retinotomy.
Speaker Change: So one of the things that we did in our phase one was you would you would deliver a pre-blab into the sub retinal space.
Speaker Change: and then you would have to follow that with a second delivery of your material and the pre-level is largely present to confirm that you had reached the correct destination. So, if you could do that with a single retinotomy...
Speaker Change: That would be advantageous, so do aluminum but still trans vitriol injectors one approach.
Speaker Change: The other is a next-generation orbit, SDS. So SDS stands for Super Coroidal Delivery System. So, I'm so-so.
Speaker Change: This, when we utilized the original SDS, this utilized a trans-corroidal approach so you would actually cut a small, you would make a small incision into the scleror and you would thread the needle around the back of the eye.
Speaker Change: and deliver the material to the target area, to the sub-retinal space without the need for a retinotomy. So that has certain advantages as well. I'm not familiar with exactly what the...
Attributes of the Next Gen Orbit SDS, what those specifically are.
Speaker Change: But they are designed to be easier to use and more comfortable for the surgeon, so...
Speaker Change: You know, I've often said as a way to analogize what's going on that
Speaker Change: You know, something like laser surgery. When it was first conceived, you know, required a scalpel and it over time became a laser and you know, the technology improved and got easier, more trustworthy over time until it became virtually commonplace.
Speaker Change: The operagen RPE cells being delivered to a sub-retinal space, it's a delicate surgery, and although we had some outstanding outcomes and others are having outstanding outcomes,
Speaker Change: If there are opportunities to make it easier to have higher success rates to have lower AE profiles, that is just going to make an increasingly compelling product profiles.
Speaker Change: So, I don't have all of the details about numbers of patients or timelines. I've shared, you know, substantially everything that I am able to share with you and others today, but I think the most important thing is that
Speaker Change: And we see that as a positive indication that reflects positively on the probability of long-term success of this program and thus that explains why we're looking at other ways to drive long-term growth of the business and not be just the operative company but to go far beyond that.
Speaker Change: because, you know, again, we're very, very good at process development, direct the differentiation and, you know, banking systems.
Speaker Change: But we can't possibly know every indication at the level of expertise that's required. So we're going to need to do some partnerships. The first and best case of that to date is what we did with Russian Genentech and it continues to be going very well, which validates our business and it validates our technology.
Speaker Change: All right, great. Thanks. That action information is helpful and yet seems clear that the oxygen programs could teach to advance and yet looking forward to hearing some about these other potential partnerships.
All right.
Thank you, Albert.
Your final questing comes from Sean McCutcheon with Raymond James.
Thank you. Thank you.
Yang: Hi, good afternoon team, Lee C.D. Hone for Sean. We have two quick questions.
Yang: The first one is wondering your thoughts on the new director for CDR, especially his take on cell therapy and what's your consideration for FDA regulation regarding gene cell therapy.
Speaker Change: The second question is Could you comment please on the title called Injury Trial, a little bit on the design for instance, is it
Speaker Change: Single Arm, and the dosing is a single multiple, as well as preliminary efficacy evaluation. Thank you.
Speaker Change: Thank you, young, for the questions. With respect to Dr. Prasad,
Speaker Change: You know, I'm not too concerned about the comments that I see regarding...
Speaker Change: You know, secondary markers of efficacy and so forth because you know the operogen program probably is is not going to rely. I can't speak for the regulatory strategy, but I presume it's not going to rely on some of the secondary end points.
that are so often used in on calligraphy trials. So,
Speaker Change: You know, I think that having watched Dr. Prasad sitting down doing a, you know, between the ferns conversation with Dr. McCary, I heard a lot about supporting innovation, being flexible and accelerating timelines.
Speaker Change: Where's such opportunities made sense? A very data-driven and rational, no shortcut. In many ways maybe it feels like elevating the bar.
Speaker Change: And frankly, we welcome that. I think I can look no farther than anti-complement and the drama regarding anti-complement that, you know, a 1 in 5,000 or 1 in 10,000 severe adverse event really impaired the uptake of that product.
Speaker Change: You know, because the clinical benefit was, you know, to be fair, quite questionable. I think it was present, but it wasn't very exciting.
Speaker Change: So I think in contrast you look at something like what an RPE transplant is capable of doing and I don't think that there's any short cuts because I think that the clinical magnitude is so significant with respect to your second question on the SCI design.
Speaker Change: The total enrollment would be between 6 and 10 patients. There is a stagger, so the first three patients will be thoracic patients, so those are T1 to T10 injury patients, but they can be chronic or sub-acute.
Speaker Change: When we get to patient 4, that's where we introduce our first cervical patient, which can be C4 to C8, and then we go into open enrollment. So we do have a stagger, and we have to get through that stagger. But I am thinking that it may be the case that chronic patients are...
Speaker Change: It's easier to enroll than subacute. We haven't seen that because we don't have experience yet, but it's part of our belief system. So, it remains to be seen what enrollment looks like, but definitely the first three patients.
Speaker Change: We'll be thoracic injuries and then we go to cervical and then it's open enrollments beginning with patient 5, 5 through 6 or 5 through 10.
Thank you for the call.
You're welcome, y'all. Thank you for the question.
Speaker Change: There are no further questions at this time. I will now turn the call back over to Brian Culley, CEO of Lineage for Closing Remarks.
Speaker Change: Thanks everyone, really no closing remarks. I think we covered everything we were hoping to cover today and, you know, if you didn't get your question answered feel free to follow up myself or Jill at any time.
Speaker Change: Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.
Joseph Pantginis, Jack Allen, Brian Culley, Brian Culley, Brian Culley
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