Lineage Cell Therapeutics at H.C. Wainwright: Advancing Cell Therapies

On Monday, 08 September 2025, Lineage Cell Therapeutics (NYSE:LCTX) presented at the H.C. Wainwright 27th Annual Global Investment Conference. CEO Brian Culley outlined the company’s strategic shift from a defensive to an offensive approach in cell therapy, highlighting both opportunities and challenges. The focus was on innovative treatments for conditions like dry age-related macular degeneration (AMD) and type 1 diabetes, while emphasizing the importance of manufacturing scalability and partnerships.

Key Takeaways

• Lineage Cell Therapeutics is advancing cell therapy programs with strategic partnerships, notably OpRegen for AMD with Roche and Genentech.
• The company is prioritizing manufacturing scalability and affordability to maintain a competitive edge.
• New initiatives include the ILT1 program targeting type 1 diabetes and continued progress in spinal cord injury and hearing loss therapies.
• A $670 million deal with Roche and Genentech underscores the financial potential of OpRegen.
• CEO Brian Culley expressed optimism about the future of cell therapy in treating various diseases.

Financial Results

• Lineage secured a $670 million biobucks deal with Roche and Genentech for OpRegen.
• William Demant Invest committed up to $12 million in preclinical investment for the hearing loss program.
• Initial investment in the hearing loss program was under $1 million before partnership funding.

Operational Updates

• The Phase 2 clinical trial for OpRegen, in partnership with Roche and Genentech, is ongoing.
• The hearing loss program is partnered with William Demant Invest.
• The ILT1 initiative aims to address type 1 diabetes by improving islet cell production.
• Lineage has treated 30 spinal cord injury patients with OPC1 therapy.
• A collaboration with Factor is exploring gene editing technologies.

Future Outlook

• Lineage plans to expand its restorative approach to other cell types and indications.
• Manufacturing scalability and affordability remain key focuses.
• The company is exploring future partnerships, including in diabetes.
• Continued investment in OpRegen is anticipated as a lead program.
• Gene editing technologies will be incorporated into future therapies.

Q&A Highlights

• A data update from the Phase 2 trial of OpRegen with Roche/Genentech is expected, though the timeline is uncertain.
• Lineage is concentrating on diseases caused by the loss of specific cell types.
• Ensuring manufacturing and cost efficiency is vital for the commercial viability of cell therapies.

For more detailed insights, readers are encouraged to refer to the full conference call transcript.

Full transcript - H.C. Wainwright 27th Annual Global Investment Conference:

Sarah Nick, Equity Research VP, HC Wainwright: Everyone, and welcome to our next session. I’ll be your moderator, Sarah Nick, an Equity Research VP at HC Wainwright, and it’s my pleasure to introduce our next presenter, Brian Culley, CEO of Lineage Cell Therapeutics, a clinical-stage biotechnology company developing novel cell therapies for neurological and ophthalmic conditions. Brian, the floor is yours.

Brian Culley, CEO, Lineage Cell Therapeutics: Thank you so much, and good morning, everybody. It’s a pleasure to be here. Let’s see where I need to click here. All right, next slide. As we’re a publicly traded company, I may make some forward-looking statements. Please refer to our safe harbor information file. Excuse me, that was ironic. SEC.gov. You can also visit clinicaltrials.gov. All right, so Lineage Cell Therapeutics. I’m going to go very quickly today because I want to squeeze in a couple of new things that you haven’t heard presented from the company before. We had some announcements, but as a general matter, Lineage Cell Therapeutics is dedicated to the manufacture and delivery of specific cell types, and we are doing this in order to address conditions and indications where the loss of a certain cell type gives rise to the disease.

The highest and best use case for this to date for us has been in the setting of dry age-related macular degeneration. This disease, the hallmark is the loss of RPE cells. We manufacture RPE cells in the laboratory and deliver them to patients in order to restore the function that’s lost in that condition. The pipeline, this is part of the news that we have. The pipeline’s a little bit larger. This morning, we announced that we have a new initiative called ILT1. This is our initiative into type 1 diabetes. Specifically, we are looking to address a manufacturing deficiency. Diabetes patients will require a very large number of islet cells in order to provide what appears to be the potential for a functional cure, but to our knowledge, no one has been able to unlock the kind of manufacturing scale required to provide a commercially viable solution.

As we have had success in other programs and we’ve learned some really interesting things on the manufacturing side, this has become an initiative that we’ve become really keen to lay our hand toward. We also recently, more in the middle of the slide here, we also announced that our hearing loss program, where we manufacture auditory neurons to treat the loss of that particular cell type, has been partnered with the William Demant Invest Company, which will provide multiple years of development for that program. A lot of things going really well, you know, and it’s not an accident that we have really been looking to find partnerships to be able to advance this because our platform really provides an opportunity to be very productive. You and me, we have 200 different cell types. That’s about 200 cell types of the human body.

There’s a lot of opportunities to manufacture replacement parts and deliver those to patients and really outperform what small molecules and antibodies are incapable of delivering in the right setting. You have to choose the right setting. All right, how do we do this? First thing we do is we expand the cells. We start with the pluripotent cell line. We expand those cells to great number, and then part two, we then convert those cells into the specific cell. That’s where a lot of our IP is coming from, is our ability to manipulate and control the lineage, i.e., the name of these different cell types in order to make the cell type of interest, so very pure populations of cells. Gene editing is not required. It is optional. If you want to impart certain properties into your cells, you can do that.

For the most part, we haven’t done that. We’re looking to replace exactly the cell that your body already relies on. We have done some very special things in the setting of manufacturing. We have been able to develop a multi-bank system. This is in a GMP environment where our master cell bank, any of those vials can give rise to a working cell bank, and any of those vials can give rise to our product. The multiplicative advantage of that is that you can get into millions of vials. We don’t have millions of vials of our product candidates sitting in freezers, but having reduced this to practice, showing that we can pull a vial and make whatever comes next means that we do have the chain.

We have reduced the practice to chain that can give rise to millions of vials at the point at which we need those millions of vials. That’s a very significant point that I wanted to make today because there’s so much attention in cell therapy, including in especially non-oncology cell therapy around the clinical data. I think that there’s a missing part of this that’s still being caught up, that you don’t have a commercially viable product unless you can affordably produce it. We use this independent banking system, this two-stage system, in order to actually do that. We have reduced this to practice in the lab in a GMP environment. All right, manufacturing is really important to us. This is what we call table stakes. What I mean by that is you’re not playing for the pot unless you actually bring this to the table. You cannot ignore this.

These are the properties that we have brought into our lead program, OpRegen, for dry age-related macular degeneration, where we have been able to show that we can durably improve both the anatomy and the function of patients who are suffering from dry AMD with geographic atrophy. This is partnered with Roche and Genentech. This was a $670 million biobucks deal that we signed a few years ago. Roche and Genentech are running a Phase 2 clinical trial for this program right now. This is one of the leading causes of blindness in this country, dry age-related macular degeneration. There are a couple of approved therapies to treat this, but we believe that they are woefully inadequate and are leaving a lot of opportunities still on the table because they don’t actually improve vision. They just slow the progression of the disease by about 20%.

We have cases of patients that we appear to be what I think can be considered reversing the condition or at least halting its advancement. We do this quite simply by manufacturing the cells that are lost in this disease. We manufacture the retinal pigment epithelial cell, and we transplant that in a one-time injection to the back of the patient’s eye. We saw something. It was sort of a happy accident. We saw this really interesting phenomenon when we put the cells right on the area of atrophy rather than sort of in the neighborhood. We got these extraordinary clinical effects. This frame on the left here, that yellow outline, that shows you where the cells were delivered. Those represent extensive coverage, i.e., we covered the area of atrophy. We covered the wound.

On the right, when we were initially doing this, we tried to stay away from the wound. We wanted to be careful. It was a Phase 1 clinical trial. That shows you what limited coverage looks like. The cells were delivered away from the area of atrophy, which is somewhat defined by the shinier, brighter central area. We’ve taken our patients and broken them into two different groups. What we saw among those patients who got the cells right on the area of atrophy was anatomical improvement that never happens naturally. Human beings cannot regrow their retinal tissue. We saw these anatomical changes that were consistent with a replenishing or a restoration of the tissue. I know a few of you go home and look at high-resolution OCT images. We sort of cartoon them in so you can kind of understand what you’re looking at.

You can obviously see for the patient at baseline did not have continuous layers, critical layers necessary for your vision. After treatment with OpRegen, there was a normalization of that structure. This happened every time we delivered the cells right onto the area of atrophy. We saw this phenomenon. This phenomenon can be quantified, and you can map this and graph this, which our partners Genentech have done and presented at a number of major medical meetings. What they have seen is that when we do cover the area of atrophy, we can see an increase, that’s your upper blue line above baseline, increases in key levels of layers of the retina relative to what is normally expected in this disease condition, which is the loss of retinal integrity. Importantly, very importantly, this was associated with improvements in function, i.e., people saw better.

It’s not just anatomic improvement, but you have a clinically meaningful increase in vision, which has lasted for years from a single administration. That blue line, you will not find that in the natural history of the disease. People get worse with this condition. What you’re seeing there is people had a very rapid increase in just a couple of months, upwards of five, six, seven letters, and it’s persisted for several years. In contrast, the contralateral, i.e., untreated eye, you can see the patients are losing. By the way, those are contralateral eyes in this patient set. If you were to look at a patient population and just track them, that lower line looks much worse. You’re losing about four letters a year, very predictably, out to you’d be minus 11, minus 12 letters.

By the way, also on the leading therapies, you’d be minus 11, 12 letters at three years instead of being plus six. You’re talking about 15, almost 20 letters difference after a single administration. Not 36 pokes in the eye, a single administration of RPE cells. It breaks out and looks even more compelling when you look at extensive coverage of the area of atrophy versus limited coverage. This is all hanging together, the anatomy and function. The ongoing study right now does not have a data report yet, but we’re really excited. We’re hoping for an update at some point from our partners. It is an open-label study. Presumably, they’re seeing the same kinds of effects that we saw, but we don’t know.

We certainly look forward to the partners providing an update on this program because we may be rewriting what the medical texts say about this disease and whether it is truly progressive and degenerative and irreversible. Maybe it isn’t. A key takeaway from this is that when you believe that your lead program is successful and it’s based on certain principles and hallmarks, you want to repeat that success. We are looking at ways that we can apply this restorative approach using other cell types in patients. Coming back to this, the things that we learned in these table stakes that I said at the beginning of the presentation remain intact, and they are important tenets or principles for us in how we develop our programs. You need to have your manufacturing. It’s not just about clinical data. We are doing this technology also in spinal cord injury patients.

You can imagine car crashes, diving boards, everything that you think of, robbing the lives of young people typically from their future because of the loss of mobility. About 30 patients have been treated with this therapy so far. The hallmark, of course, is the loss of mobility. If I turn my hand like that, that doesn’t look like very much, but that’s an ability to manipulate a wheelchair. I can now get around. Even small gains in my upper extremities as a patient with a debilitating injury can have huge impacts in terms of the quality of life. We manufacture a different cell type. Obviously, we’re not putting retina cells into the spinal cord. We’re replacing the cells of the spinal cord. These are oligodendrocyte progenitor cells that are responsible for the myelin. They are the electrical sheath for your nervous system.

These wires here, they wouldn’t do a very good job of carrying current if they weren’t wrapped in the insulation that’s housing them. It’s the same principle here. We’re using the cell type that is lost in a replace and restore strategy. This also is allogeneic, which means this is an off-the-shelf therapy that would be appropriate for everyone. We don’t take cells from the patient, manipulate them, and put them back. That’s profoundly expensive because it’s a custom therapy. This is a therapy that would be fit for all. There are a number of mechanisms of action that we believe could be applicable here, preventing the cavitation, which is the gap, spackling the hole, myelinating the axons necessary for coordinated mobility. Neurotrophic factors are probably supportive in this setting.

What we’ve seen is that patients who receive these cells, this is not a controlled comparative study, but against historical control, seem to be doing better. This was evocative Phase 1 data that people with very severe injuries seem to be having increases in mobility. This is something that we want to get ready to advance into a comparative study to figure out what is the magnitude of benefit that these patients are enjoying from this intervention. If you can move someone a couple of levels from a cervical level four, cervical level six, huge changes in the cost of their therapy and the quality of their lives. Quite importantly, this clinical study was remarkably safe from an AE perspective. 534 AEs, but remember, we’re talking about car crashes. These are cervical injuries. We’re talking about car crashes and things like that.

There are a lot of problems with these individuals. They have problems regulating the entirety of their body. Only one of those 534 AEs was potentially related to OPC1. It was a grade 2 dysesthesia. It’s auto-resolved. We think that we’ve got an opportunity here because we have a very nice safety profile, which might encourage us that even a relatively small increase in mobility could be sufficient to support approval for this kind of intervention. We saw that the cells have been resident. They’re not being rejected. One of the nice things about working in the eye, working in the spinal cord, is you have some immunoprotection. Patients are on very short-term immunosuppression, 60 to 90 days for those two indications. That’s probably more than needs to be. It probably could be even shorter. We didn’t push the limits of it.

We’ve never had a case of rejection of our cells reported from those programs. This has all been fairly recently published just a few years ago. All the publication comes out. I don’t know that anyone has longer follow-up data in cell therapy. This is one of the earliest approaches of its kind. You’re talking 7, 8, 9, up to 13 years of follow-up data. No one’s growing tumors. There are no cysts in these patients. The industry has figured out how to control those problems. Some of the archaic fears of cell transplantation probably need to be revisited because they just have not appeared, certainly at least not in our hands. What has happened in our hands is we’ve gotten much better at controlling how we make the cells.

The clinical data that I just showed you, this is an impurity profile from those cells, the gray bars, upwards of 20% of other stuff that you’re not actually trying to make. The stuff that we make now is represented in orange. You can see how much better. We’re getting nice pure populations. Of course, nowadays, you can bring bioinformatics into that. Look in the top left of the box I marked off here, epithelial cells. Why would you want epithelial cells in your spinal cord? You don’t. That’s actually what led to that evocative clinical data. Now we make a lot cleaner stuff. I don’t think it’s just Lineage. I think we’re world-class at this. As a general matter, non-oncology cell transplantation has really matured as a field. I think you’re seeing it in the hands of a number of companies. We are among the leaders.

You’re seeing a lot of really exciting data because people have figured out how to make the right stuff, control it, scale it. Some have figured out how to scale it. It’s a really exciting and I think emerging branch of medicine relative to CAR-T. CAR-T is amazing, right? Revolutionized oncology. It’s a pillar in there with chemo and surgery. Non-oncology, I think that’s the future. I think that’s the untapped potential. We’re trying to be ready to be there. I’m going to skip past just very quickly. We’ve got this really cool device for spinal cord. It allows you to deliver the cells while the patient is breathing. You don’t have to disconnect the patient from the respirator. However, you have to get the cells in. You can push the cells in over four or five minutes. This is an ongoing study.

For the sake of time, I just want to move past it a little bit faster. I want to get to the end. I want to talk about looking ahead. Part of our strategy is, as we have increased our confidence in our lead program going forward, and as we imagine that we might have a better cost of capital in the future, we want to repeat this success. We want to take this program and we want to apply it into other areas that we see opportunity. We want to go from being defensive to offensive. We want to pick the areas of interest to us. That’s where we’ve done things like our hearing loss program, which when we launched this, because we didn’t have to screen small molecules. I don’t have to create an assay. I don’t have to structure activity relationship.

We just know that the auditory neuron works. We got to figure out how to make an auditory neuron. We were able to decide that we wanted to do this program, right? No wet lab work. Just sitting around with papers saying, "You want to do this?" Within a year, we had started preclinical testing, right? That speed is incredible. We put less than $1 million in that year into this program in order to birth it. Just recently, we announced this partnership with William Demant Invest. They’re going to put in up to, assuming the program continues, they’re going to put in the next $12 million in preclinical investment. We put in $1 million, and then we’ve been able to attract a partnership that’s going to fund some of the early high-risk program.

In an environment where you’ve got kind of a bad biotech tape, the cost of capital is really high. Finding partnerships for early-stage programs is a great way of advancing your business, right? We could imagine that we could have a whole basket of opportunities like this. Really happy with what we’re doing here by having, as I say, a number of different opportunities through partnerships with groups like Roche, groups like William Demant. What else is happening at the company? We have a photoreceptor program. We, not too long ago, uncoupled this from some earlier IPs to get us unencumbered from some third-party rights that we had, which I think increases the value of that program. RND1 is an undisclosed indication that we have. We’re not a gene editing company, but I think the future of this technology will incorporate gene editing and gene engineering.

We have brought in some of that technology through an alliance with Factor. I understand there’s excitement around in vivo editing. There’s also a little scariness around in vivo editing. We’re not doing that. Everything’s ex vivo. We can check what we have before we put it into a patient to assure that we have the stuff that is exhibiting all the right properties and characteristics through our analytical processes. This morning’s announcement around ILT1, type 1 diabetes. I said this at the beginning, but I think it’s a point worth repeating. A challenge with diabetes is you’re talking about needing, let’s say, on the order of 10 to the 14th cells, right? Like 100 trillion cells. The dose for a diabetes patient is probably in the neighborhood of a billion cells per person. In the retina, with macular degeneration, it’s probably in the order of 100,000, right?

You have a massive change in what you’re trying to do. You have to be able to scale that. If you have, you know, there’s not enough T75 flasks in the world to support the entirety of the diabetes population. You have to have different approaches. You have to have banking modalities, and you have to have differentiation modalities that can actually generate affordably the number of cells that are required to treat a meaningful patient population. Otherwise, you’re just doing a science experiment. I don’t know if we’re going to figure it out, but we have a reason to want to try it because we have actually learned some really interesting things. I’m very excited about this new program. Whether this ends up being a future partnership opportunity, whether we enter the diabetes program, diabetes field ourselves, to be determined. I don’t know. We’ll see what happens.

We know that there’s a big problem in this area of the field, and we want to see if we can solve it. I think it’s a fascinating company. There’s a lot of attention. People always ask, you know, what’s going to happen with your lead program? When’s data coming? We don’t know. It’s a really great program. It’s shown three years of exciting clinical effects, and we’ll wait and see. In the meantime, we’re encouraged by the advancement of that program and are investing in a much longer and greater future for the company. Thanks very much for your attention, and I’d be happy to take some questions out there in the back.

Sarah Nick, Equity Research VP, HC Wainwright: Great. Thank you, Sarah.

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