Lineage Cell Therapeutics Q1 2025 Earnings Call Transcript

Quartr
Provided by Quartr
May 13, 2025

There are 8 speakers on the call.

Operator

Welcome to the Lineage Cell Therapeutics First Quarter twenty twenty five Conference Call. At this time, all participants are in a listen only mode. 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, and recordings, reproductions, or transmissions of this call without the express written consent of Lineage are strictly prohibited. As a reminder, today's call is being recorded.

Operator

I would now like to introduce your host for today's call, Iwana Hone, Head of Investor Relations at Lineage. Ms. Hone, please go ahead.

Speaker 1

Thank you, Carly. Good afternoon, and thank you for joining us. A press release reporting our first quarter twenty twenty five financial results was issued earlier today, 05/13/2025, and can be found on the Investors section of our website. 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 securities laws. 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.

Speaker 1

The company's actual results or performance may differ materially from the expectations indicated by such forward looking statements. For a discussion of certain factors that could cause the company's results or performance to differ, we refer you to the forward looking statements sections in today's press release and in the company's SEC filings, including its most recent annual report on Form 10 ks and in subsequent SEC filings. 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 SEC filings. With us today are Brian Culley, our Chief Executive Officer, and Joe Howe, our Chief Financial Officer. I'll now hand the call over to Brian.

Speaker 2

Thank you, Iwana, and good afternoon, everyone. We appreciate you taking the time to join us on the call. I'll start off today with an update on OpRegen, and talk about some interesting results reported at the recent iCELLERATOR and ARVO conferences. I also want to ensure everyone is aware of the forthcoming thirty six month clinical data, which will be presented next month at the CTS meeting. After OpRegen, I have a brief but important update on a recent manufacturing milestone that gives us the opportunity to broaden our business strategy.

Speaker 2

And after that, I'll say a few words about the dose study of OPC1 before Jill reviews our financials, and we'll leave plenty of time today for analyst questions. Starting with OpRegen, this is an allogeneic RPE cell transplant intended for patients suffering from dry AMD with geographic atrophy. Unlike the currently approved anti complement therapies, require monthly or semi monthly injections and only slow disease progression by around twenty percent, OpRegen 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. 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. Based on the totality of the data collected to date, we believe there's a tremendous opportunity to provide better outcomes for GA patients and from what is available from existing therapies.

Speaker 2

OpRegen is currently being evaluated in a phase 2a study called the GALET study that is being paid and run for by our partners Roche and Genentech. The GALET study is exploring various parameters of surgical delivery to help enable a safe and repeatable procedure. Overall, the goal of the GALET study is to refine the surgical procedure and optimize the risk benefit profile. We believe the ongoing Gillette study is progressing well because it is an open label study with ninety day endpoints and it has been enrolling continuously for approximately two years. While we do not have access to data from that trial, we note that Genentech has continued to invest in the program by, for example, seeking and obtaining RMAT designation, opening additional clinical sites, and acquiring proprietary surgical delivery devices designed for delivery to the sub retinal space.

Speaker 2

These positive steps have occurred against the background of Roche reducing its pipeline, which adds further conviction to our belief that OpRegen is delivering promising results in this open label study. I want to remind everyone that there is no timeline available yet for a decision by Genentech whether to advance OpRegen into a controlled study. 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 GALET study or announce any GALET data before initiating such a controlled study. So it's possible a larger controlled study could be announced while the Galette study continues. As a reminder, OpRegen is a pioneering new technology for which delivery is an important component, So we expect at least some aspect of surgical optimization could continue for a long time.

Speaker 2

At the same time, we're encouraged that Genentech could elect to advance OpRegen to the next stage of development whenever they're comfortable doing so. Until such a decision is made, we will continue to await further updates from our highly capable partners. While we all await information from the ongoing phase 2A or any subsequent trials of OpRegen, are two additional things I'd like to highlight. The first is that Genentech plans to report three year data from the phase one2a trial which Lineage conducted, and which will be presented on Genentech's behalf by Doctor. Chris Reeman from Cincinnati Eye Institute.

Speaker 2

That presentation will occur at the Clinical Trials at the Summit twenty twenty five Conference on June 21. CTS was founded by Doctor. Arshad Khanani, Director of Clinical Research at Sierra Eye Associates, and the event brings together a diverse and global group of experts to discuss the latest data from ongoing clinical trials. Doctor. Riemann was an investigator on the phase one-2a study of OpRegen, and we greatly appreciate his availability to present this new data for Genentech.entech.

Speaker 2

The reason this forthcoming data is important is that the anatomical and functional benefits that were previously reported out to two years following OpRegen treatment were notable. 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 over those two years. 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, because it indicates the difference in visual performance between an OpRegen treated patient and a patient even on the best available therapy could be widening over time. Furthermore, a three year benefit adds additional support to OpRegen potentially being a one time treatment, which could mitigate compliance issues and we believe would offer a striking advantage over the monthly injections required for best available outcomes with anti complement therapy. We haven't seen the three year data from Genentech yet, but we're hopeful it will provide additional conviction in our approach.

Speaker 2

Speaking of conviction, the second OpRegen item I wanted to highlight today is recent news and data updates from competing RPE companies, which I think is extremely positive for us. Recent phase one 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. 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 OpRegen. Another reason this is important is that an RPE cell transplant is a completely new modality and the only approach credibly demonstrating the potential to improve vision in GA patients. So it's normal for people to want to see more clinical data than Lineage has generated to date.

Speaker 2

And so every time some other company independently reports positive data from an RPE transplant like ours, those data points can be aggregated into an increasingly powerful endorsement of our approach. And at this point, we are aware of four independent reports of functional gains from an RPE transplant. From among those companies, we assume investors will try to sort out which among us is best positioned to win the large addressable and increasingly established GA market. Given everything that is required to successfully manufacture and commercialize an allogeneic cell therapy product for such a large patient population, I feel confident that the three pillars of one, Lineage's manufacturing and scale up process, two, Genentech's device and drug development capability, and three, Roche's leadership in ophthalmology product commercialization provides us with advantages over competing RPE programs. Overall, I'll say that we continue to believe these actions reflect things moving in a very positive direction with the OpRegen program.

Speaker 2

I just mentioned Lineage's manufacturing and scale up capability as one of the three pillars which contribute to a competitive advantage, but I want to take just a moment to explain again why I see so much value in our manufacturing platform. When comparing allogeneic and autologous cell therapies, it's important to recognize that the key advantage of allogeneic 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. 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. To date, we haven't found any examples of what we consider to be a true allogeneic manufacturing process, meaning one which fulfills the intent of low cost and consistent production from a stable and unchanging source of cells. 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 2

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, 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, we believe we are the first in our field, specifically in the field of non cancer allogeneic cell transplantation, 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. 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. We see this as a major achievement for our business because generating a GMP banking system that can deliver large scale and low production of a cell therapy opens up new opportunities for partnerships using cell types not currently in our pipeline. And in fact, around the time of disclosing this achievement, we were able to initiate a number of additional partnering discussions.

Speaker 2

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. The business implication of all this is that if Genentech moves OpRegen into a comparative trial, we believe we will enjoy not only a lower cost of capital, but also a strong rationale to repeat our success with additional cell based programs beyond OpRegen and OPC1 and AMP1 currently in our pipeline. For this reason we believe entering into additional deals can offer an increasingly attractive non dilutive opportunity to expand our business model and create long term value. Moving on, and next we'll provide an update on our second clinical stage allogeneic cell transplant program, OPC1, which is designed to help increase recovery and mobility for people who have suffered from a spinal cord injury. 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.

Speaker 2

OPC1 has been tested in thirty 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. But we have two areas of improvement we want to complete before we would feel ready to move OPC1 into a later stage trial. The first area of improvement is delivery. We have initiated a small clinical study called the dose study to test the safety and performance of a novel delivery device in six to ten patients. We believe the device we are testing in this study will be superior to the original delivery system in two ways.

Speaker 2

First, it's easier to use and deploy to clinical sites. 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. Previously it was necessary to stop ventilation when you delivered the cells, so this is a significant enhancement to the procedure. In addition to the safety and performance of the new device we also will be collecting functional assessments on all patients, which gives us the opportunity to investigate any signals of efficacy that may arise. This is important because this study will be the first time OPC1 will be administered to patients with a chronic spinal cord injury.

Speaker 2

Chronic SCI represents an additional and larger potential patient population for this experimental therapy. 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. The second area of OPC1 improvement is our new manufacturing process, which actually has two sub parts. We increased 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. Subject to obtaining 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 2

In parallel with these two ongoing enhancements to the OPC1 program, we also are working on the design of a larger trial with a focus on collecting more specific and clinically relevant endpoints with a goal of reducing the well documented challenges of data capture in this patient population. 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 OPC1. Meanwhile, we still plan to apply for a CIRM clinical grant as soon as they begin accepting applications, which CIRM has indicated should occur fairly soon. Lastly, we recently announced that we are proudly collaborating again with the Christopher and Dana Reeve Foundation on the third annual SCI Investor Symposium. We decided to convert this to a virtual conference, because that can significantly increase participation for individuals who have difficulty with in person events, and broaden overall participation by speakers and attendees.

Speaker 2

This year's symposium will build on last year's momentum and will be available online 06/27/2025. And with that, I will turn things over to Jill for a review of our financials.

Speaker 1

Thanks, Brian, and good afternoon, everyone. As of 03/31/2025, our overall cash position was $47,900,000 This capital is expected to support our planned operations into Q1 of twenty twenty seven, which we believe will allow us to reach multiple events and milestones. As we discussed in detail on our last call, we continue to have approximately $37,000,000 of warrant capital which could become available to us. 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 CIRM grant, and additional collaborations which we may elect to enter into. With respect to our first quarter financials, we largely came in line with our historic period.

Speaker 1

Total revenues for the quarter were 1,500,000 a net increase of $100,000 compared to $1,400,000 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. Operating expenses for the first quarter were $8,000,000 a decrease of $100,000 as compared to 8,100,000.0 for the same period in 2024. R and D expenses for the first quarter were $3,100,000 an increase of 100,000 as compared to $3,000,000 for the same period in 2024. The net increase was primarily driven by $200,000 for our preclinical program, partially offset by $100,000 for other research and development programs.

Speaker 1

G and A expenses for the first quarter were $4,900,000 were primarily in line with expenses for the same period in 2024. Loss from operations for the first quarter was $6,500,000 a decrease of $200,000 as compared to $6,700,000 for the same period in 2024. Other income for the first quarter was $2,400,000 compared to 100,000.0 for the same period in 2024, and the net increase was primarily driven by changes in the fair value of the warrant liability. The net loss for the first quarter was $4,100,000 or $02 per share, as compared to a net loss of $6,500,000 or $04 per share for the same period in 2024. 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.

Speaker 1

With that, I'll hand the call back to Brian.

Speaker 2

Thanks, Jill. So I will summarize today by saying that we remain confident in the potential for OfraGen to drive positive clinical outcomes in dry AMD, and are encouraged by our partners' continued signs of commitment to the program. 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 Genentech next month. 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 via funded partnerships and independently. And third, we are steadily advancing OPC1 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 2

As always, we appreciate your support and belief in our vision. With that, operator, we are ready to take any questions from analysts.

Operator

Your first question comes from Mayank Mamtani with B. Riley Securities.

Speaker 3

Thank you so much for taking our questions. This is William Wood on for Mayank. Congratulations on the very nice quarter. A couple from us. We're actually just curious if you'd be willing to sort of compare your capabilities in terms of manufacturing to peers and how much of a differentiation you feel that you have in scale up, potentially having redundancies and perhaps even supplying for the demand that OpRegen could potentially have.

Speaker 2

Yeah, hi William. I can speak broadly about our capabilities, but I won't speak to any particular programs. But actually it's quite frustrating that we can't easily make direct comparisons. Unfortunately, the majority of our peers don't actually describe in sufficient detail exactly what they're capable of. There are some exceptions.

Speaker 2

Some companies have the confidence to explicitly state that they have not achieved this level of capability, but most of them, I think, appear 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 twenty years, So I do believe it's highly differentiated 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 cell therapy matures and investors look beyond the initial and sometimes exciting phase one data and start asking difficult questions about being able to supply material affordably to a large patient populations and noting that in some cases that material itself in a single dose may be millions, tens, or even hundreds of millions of cells, one is going to be increasingly forced to demonstrate a production capability from a platform that can deliver that kind of scale.

Speaker 2

And so if you permit me to just give an illustration, if you had a master bank of a hundred vials of cells and any one of those 100 vials could make a working cell bank of a hundred vials. And then any one of those vials could make, let's say, a hundred doses of your treatment. You have then in hand a manufacturing system that's capable of making 1,000,000 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, but what we have done is we have reduced to practice in a GMP environment that, system or that platform, and we've done it with multiple cell lines, and we've done it for multiple cell types. So I do hope that others do come forward so that we can make direct comparisons.

Speaker 2

We welcome the challenge, but right now we believe we stand alone with this capability at this time.

Speaker 3

Got it. 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 with the recent with the current administration. Just thinking about tariffs, do you have a manufacturing site in Israel? I was just kind of curious what type of potential tariff impacts we might expect and how those might be mitigated if depending the degree of tariffs that we might see come?

Speaker 3

Obviously, a little bit of speculation there.

Speaker 1

Yeah, Brian, I'm happy to take that question. 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 store them on-site to ensure that we can properly mitigate any type of production issues in the future.

Speaker 2

Yeah. And if you'll permit me, William, to add, there are a couple of other things that are floating in the ether these days. We're really encouraged by comments that the new commissioner Marty Makary has said about cell and gene therapies holding tremendous promise. I think that's a direct quote. And I think that the even more recent discussion around MFN simply highlights how important it is to have really wide margins.

Speaker 2

If there's cost pressures and pricing pressures, being able to manufacture your material very affordably will become increasingly important in order to be protected from some of those constraints.

Speaker 3

Got it. Appreciate that extra color. We'll hop back into queue. Really appreciate you answering our questions, and congratulations on very productive quarter. Thank you.

Speaker 2

Thank you, William.

Operator

Your next question comes from Jack Allen with Baird.

Speaker 4

Great. Thanks so much for taking the questions and congrats on all the progress. I guess I'll start with one on the manufacturing side of things as well. It seems like you believe you developed a unique capability here. And it sounds like you've already seen some interest as it relates to partnerships.

Speaker 4

I'd love to hear a little bit more of your thoughts on how potential deals could take shape. Is it a technology that you'd look to out license? Or is it something that you'd look to kind of utilize as a platform and take small pieces as an enabler of other people's products? How are you thinking about the potential partnership formation around this new outcome here?

Speaker 2

Yeah, thank you for that question, Jack. I think each opportunity will be specific to itself. I think as a general matter, we are not aspiring to become a CDMO. 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 show some clear evidence of that capability, meaning actual specific file counts and etcetera, gives us an opportunity to collaborate with others where we would have, in my vision, some sort of payments, success payments over time and some ownership in an asset.

Speaker 2

I think that the ultimate scope of what a deal or multiple deals would look like, I think from our perspective, 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 And it's going to depend on what the scope and need of potential partners is going to look like. 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, 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 to prove our worth and then increase the value of a deal thereafter. It's still new, it's still breaking, but it's very exciting because it allows us to begin to act on something we've wanted to do for a long time, which is look beyond just a program like OpRegen 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 in other disciplines where frankly we would not be experts in all of those different disease states.

Speaker 2

But we could be experts in directed differentiation and process development.

Speaker 4

Got it, got it. That's very helpful. And then, as it relates to the OPC1 program, seems like you've made some real progress there and that you're hoping to have the first patient enrolled in June. When might we 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 sixty Minutes highlighting onwards device technology spinal cord injury.

Speaker 4

How do you expect OPC1 could play in with potential device innovations? Know ONWARD has theirs and Neuralink is also working on a device for this population as well.

Speaker 2

Yes, thank you. I had an opportunity to watch the 60 piece. I thought it was almost as good as the CNN piece that included OPC1 and the recent interview with Doctor. Sanjay Gupta. The nice advantage of OPC1 is that it really could partner well with other technologies.

Speaker 2

So whether those technologies are electro stimulation based, mechanical based, a technology like Neuralink or Precision that have brain chip interfaces, All of these things could partner with OPC1 and I do think that many in the field believe that maximum clinical outcomes, meaning the greatest improvements to mobility and quality life for patients will come from a combination of different approaches. Regrettably, there are almost disincentives to testing multiple approaches simultaneously. So we all have to show as on a standalone basis that we can move the needle. 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 2

And then maybe shortly thereafter a patient is receiving a transplant of OPC1, and then maybe they're going into a really well refined and patient specific physical therapy course. And then maybe they're getting aided by either some stim or some mechanical devices, and maybe they ultimately have a chip implanted so they can really maximize the gains provided by those prior modalities. I think that's where many people see the vision of spinal cord injury becoming less and less devastating by all of us working together, which is one of the reasons we pulled together this conference, is to imagine what it looks like. I think that we're not certainly going to claim that OPC1 on a standalone basis is going to compete with those modalities or displace them. I think it's exactly the opposite.

Speaker 2

I think OPC1 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 4

That's great color. Brian, sorry, just being more pointed too. Do you have a sense for when we could get initial data from that dose study of OPC1?

Speaker 2

The dose study is an open label study. The primary endpoint regarding the, success and safety of delivery occurs at thirty days. So I imagine that we would be forthcoming on a regular basis from that open label study. The other component of it is, functional assessments. Those would necessarily lag.

Speaker 2

A typical functional assessment would be six or maybe more likely twelve months after administering a therapy. So there would probably be sort of two waves of information. The first affirming for investors that our decision to move to this new device, that that device is being deployed successfully without, unusual adverse events, would be an important announcement. And then I think lagging to that by several quarters or more would be any information regarding functional changes in patients unless those functional changes were extremely striking and notable on a standalone basis. But I will remind you and I'll remind everyone that when we first observed retinal restoration with OpRegen, it was approximately nine months before we disclosed that to the public, because it really is an 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.

Speaker 2

So if we did see something extraordinary in particular with one of the chronic patients, 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 4

Great color, and congrats again on all the progress. Thanks so much.

Speaker 2

Thank you, Jack.

Operator

Your next question comes from Michael Okunowicz with Maxim Group.

Speaker 5

Brian, thank you so much for taking my questions today. Congrats on all the progress.

Speaker 6

Thank you, Mike.

Speaker 3

So I guess just

Speaker 5

to start, on the dose study, can you just help us understand what a successful delivery outcome would look like? Is this pretty much all safety? You're looking for different adverse events or are there some metrics that would demonstrate successful delivery? And then is there a benchmark that we should be looking at to compare it to the prior delivery method?

Speaker 2

Yes, 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, into the same lesion or damaged area. 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 to compare to.

Speaker 2

The external components are all about how that needle is held and the apparatus, the physical apparatus that's attached to the patient, which as I described in my remarks, 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 having supported breathing. 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 is largely going to be pointing at the exterior equipment. Is it easy to use? Are there any unexpected complications which pertain to the use of the equipment more so than AEs pertaining to the patient, for the reasons I just shared?

Speaker 2

So I think that really this is about enabling a better, a 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, it's very complicated, it's very unwieldy, and frankly none of us want to go into a phase two or other design 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 that's about the size of shoe box. So you can imagine comparing that with something that's the size of a dresser drawer with all of the packaging that 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.

Speaker 5

Alright, thank you for that. And then you did mention a new immediate use formulation. Is this different from the thaw and inject formulation and how so if it is?

Speaker 2

No, I often use those terms interchangeably. I think going back to the first question that think my aunt was asking, you know, of the differences and nuances across the different ways that people prepare and utilize cells are important considerations. So we're aware that the original material that was used in the clinical development of OPC1 by other sponsors before we acquired the program, it required hours of dose preparation. So the cells had to be plated and washed, they were being manipulated and counted, and if I recall correctly there was even a dosing error in one of the patients on that study attributable to this dose preparation. So what we have done is developed, patented, and employed a thaw and inject formulation which allows that product candidate to be immediate use.

Speaker 2

And for us immediate use means that we're taking it out of a frozen state, we're putting it into a ThawSTAR device and approximately five minutes later those cells are ready to be injected into the patient without significant manipulation or counting. The reason why this is important is that many of the cell therapies, including some that have been quite successful, have extensive dose preparation or handling or shipping requirements everywhere you have a step. You have cost, you have tracking, you have the potential for error. So by having material that can be used immediately for the patient and using our thaw and inject formulation, 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. We've had people who have been asking us questions about licensing and using that technology.

Speaker 2

So thank you for the question. Immediate use and thaw and inject, I think I typically use those interchangeably, but the point here is to avoid the manipulation and the handling of 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 5

Yes, thank you. I appreciate the additional clarity as well as the additional commentary. 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 Roche's OpRegen study, is this something new that Roche is doing?

Speaker 2

Yeah, thank you. That was a small comment I made at the recent accelerator meeting. Roche, Genentech rather, permitted me to note that they were planning to evaluate two proprietary surgical devices. One of those is a dual lumen transvitriol sub retinal injection. The other is a next generation Orbit SDS.

Speaker 2

You may recall that Lineage utilized the first gen Orbit SDS. 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 if you consider that there's a surgical optimization study being conducted and that if there are any improvements and findings in 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 had, one of the things we had hoped for through the alliance with Roche and Genentech is they are bringing to this program advanced surgical delivery, know how, tools, technology, and ability to test that are beyond what Lineage was prepared or capable of doing at that time.

Speaker 2

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 5

Yeah, thank you. It's certainly encouraging to see the level of optimization they're putting into the program.

Speaker 2

Thank you, Mike.

Operator

Your next question comes from Albert Lo with Craig Hallum Capital Group.

Speaker 6

Hi. Maybe just a 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

Speaker 7

the potential advantages of these new devices, whether maybe they aim to be a little safer or somehow, I guess, apply themselves a little better. Whatever else you can share, I'd be interested to hear.

Speaker 2

Thanks, Albert. A little bit. The dual lumen transvitriol, that enables delivery of a saline pre bleb followed by the OpRegen suspension through a simplified procedure and one with a single retinotomy. So one of the things that we did in our phase one was you would deliver a pre bleb into the sub retinal space and then you would have to follow that with a second delivery of your material. And the pre bleb was largely present to confirm that you had reached the correct destination.

Speaker 2

If you could do that with a single retinotomy, that would be advantageous. A dual lumen, but still transvitriol injector is one approach. The other is a next generation Orbit SDS. So SDS stands for suprachoroidal delivery system. So We utilized the original SDS, this utilized a transcharoidal approach.

Speaker 2

You would actually cut a small you'd make a small incision into the sclera and you thread the needle around the back of the eye and deliver the material to the target area, to the subretinal 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, but they are designed to be easier to use and more comfortable for the surgeon. I've often said as a way to analogize what's going on that you know, something like LASIK surgery, when it was first conceived, it required a scalpel and then over time it became a laser and, you know, the technology improved and got easier, more trustworthy over time until it became virtually commonplace. The OpRegen 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, 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 profile.

Speaker 2

So I don't have all of the details about numbers of patients or timelines. I've shared substantially everything that I am able to share with you and others today, but I think the most important thing is that organizations are looking at ways, continue after years now of work, continuing to look at ways of making things better, 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 for us to drive long term growth of the business and not be just the OpRegen company, but to go far beyond that. Because again, we're very, very good at process development, directed differentiation, and banking systems, but we can't possibly know every indication at the level of expertise that's required. We're going to need to do some partnerships.

Speaker 2

The first and best case of that to date is what we did with RocheGenentech, and it continues to be going very well, which validates our business and it validates our technology.

Speaker 7

Great, thanks. That extra information is helpful, it seems clear that the OpRegen programs continue to advance, and looking forward to hearing some about these other partnership opportunities. All right, thank you.

Speaker 2

Thank you, Albert.

Operator

Your final question comes from Sean McCutcheon with Raymond James.

Speaker 6

Hi, good afternoon team. Lucy Young on We have two quick questions. The first one is wondering your thoughts on the new director for CBER, especially his take on cell therapy And what's your consideration for FDA regulation regarding gene cell therapy? Second question is, could you comment please on the spinal cord injury trial, a little bit on the design, for instance, is it single arm and the dosing is a single or multiple as well as preliminary efficacy evaluation?

Speaker 6

Thank you.

Speaker 2

Thank you, Young, for the questions. With respect to Doctor. Prasad, I'm not too concerned about the comments that I see regarding secondary markers of efficacy and so forth, because the OpRegen program probably 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 endpoints that are so often used in oncology trials. I think that having watched Doctor. Prasad sitting down doing a between the ferns conversation with Doctor.

Speaker 2

McCary, I heard a lot about supporting innovation, being flexible and accelerating timelines, where such opportunities made sense, right? A very data driven and rational, no shortcut. And in many ways maybe it feels like elevating the bar. And frankly, we welcome that. I think I can look no farther than anti complement and the drama regarding anti complement that one in five thousand or one in ten thousand severe adverse event really impaired the uptake of that product because the clinical benefit was, to be fair, quite questionable.

Speaker 2

Think it was present, but it wasn't very exciting. 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 need for shortcuts because I think that the clinical magnitude is so significant. With respect to your second question on the SCI design, the total enrollment would be between six and ten 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 subacute. When we get to patient four, that's where we introduce our first cervical patient, which can be C4 to C8, and then we go into open enrollment.

Speaker 2

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 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. It remains to be seen what enrollment looks like, but definitely the first three patients will be thoracic injuries, and then we go to cervical, and then it's, open enrollment beginning with five through six or five through ten.

Speaker 6

Thank you for the color.

Speaker 2

You're welcome, Young. Thank you for the question.

Operator

There are no further questions at this time. I will now turn the call back over to Brian Cully, CEO of Lineage for closing remarks.

Speaker 2

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 1

Thank you.

Operator

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

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Key Takeaways

  • OpRegen, an allogeneic RPE cell transplant for dry AMD, has shown anatomical and functional benefits lasting up to two yearsfrom a one-time delivery, and Roche/Genentech continue to invest in the program with RMAT designation, additional sites and proprietary surgical devices while a decision on a controlled trial remains pending.
  • Independent phase I data from competing RPE transplant programs presented at ARVO provide external validationthat one-time RPE cell therapy can deliver functional visual gains beyond current anti-complement therapies, bolstering confidence in Lineage’s approach.
  • Lineage achieved a major manufacturing milestone by completing two GMP production runs from a single pluripotent cell bank system capable of producing millions of doses from one starting cell line, a platform demonstrated across multiple cell types that now underpins new partnership discussions.
  • The OPC1 spinal cord injury program will launch a dose-study in June using a novel delivery device that allows continuous respiration and an immediate-use (thaw-and-inject) formulation, with plans to enroll 6–10 patients and later design a larger trial once delivery, cell process and endpoints are optimized.
  • With $47.9 million in cash to fund operations into Q1 2027, about $37 million of potential warrant capital, and ongoing pursuit of milestone payments, grants and collaborations, Lineage underscores fiscal disciplinewhile advancing its cell therapy pipeline.
AI Generated. May Contain Errors.
Earnings Conference Call
Lineage Cell Therapeutics Q1 2025
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