Cell Therapy for Dry AMD and Beyond: Next Steps


Lineage Cell Therapeutics CEO Brian Culley shares a clinical trial update on their leading cell therapy candidate and discusses the important role of the FDA as more players enter the cell and gene therapy space.

At the 2021 Association for Research in Vision and Ophthalmology (ARVO) Virtual Meeting, Lineage Cell Therapeutics presented the latest data from their ongoing clinical trial (NCT02286089) of OpRegen in patients with dry age-related macular degeneration (AMD).

The updated data included a minimum of 4.5 months of follow-up on all 24 treated patients, as well as new results from the 12 patients in Cohort 4. Notably, 83% of those patients' treated eyes were at or above baseline visual acuity at last assessment post-transplant with Lineage's proprietary cell replacement therapy that utilizes allogeneic human retinal pigment epithelium cells injected directly into the subretinal space.

In an interview with GeneTherapyLive, Lineage chief executive officer Brian Culley spoke about the OpRegen program, including the importance of working closely with the FDA on therapy and trial development, as well as other areas of interest where Lineage's cell therapy technology has shown promise.

Interview transcript: (edited for clarity)

Brian Culley: This is the first time that OpRegen has been tested in humans. We have completed 24 patients; the first 12 patients were all legally blind. That's a safety component, as you're not going to do any harm if you've got patients who already have negligible vision, but we had some encouraging early signs and the procedure was well tolerated. We were able to then move into the next 12 patients who had higher levels of baseline vision. In fact, they would be high enough that they could even drive a car, and that's where we saw better efficacy. We think that makes sense because patients that have less advanced disease are more likely to respond to our transplant approach. So, we've completed enrollment in the study, all 24 patients are now in the follow-up period, and every few months we've been putting out interim updates on how the patients are doing. Soon this year, we'll go and talk to the FDA about the next clinical study that will be used with OpRegen.

The FDA has been very responsive to the growing wave of new cell and gene therapies, so sponsors like Lineage and others are really rushing to the clinic because this is a brand new technology which holds tremendous promise. The number of clinical trials for cell and gene therapies has really been growing, so the agency has been really responsive. I think what we're going to see is an increased level of expectation from sponsors. What I mean by that, specifically, is that at one time, you didn't have to do as much analysis of the cells. But I think that the threshold, the requirements to be able to show that you have control over your cell production, to ensure that you're putting the same material in patients every time, which is really the foundational aspect of doing an experiment—to reduce variability—[are going to be greater]. Having control over your process, being able to scale your process, these are all important areas, but I think that the FDA is well aware of them and experienced in them and is communicating not only with Lineage but also others to ensure that these new technologies can reach patients without delay.

I think this is going to be an incredible year; I thought last year was great, and we're going to really try to outdo it. What I mean by that is that we will continue to have very important data updates from the dry AMD program. I'm working to get our spinal cord program back into the clinic; we had achieved a lot of manufacturing improvements to make that commercially viable, so that was a breakthrough achievement. And then we hope to finish enrollment and report data from our oncology program with our dendritic cell vaccines, so that's a lot of clinical data. And then of course, there are always opportunities for partnerships big and small. When you've got, you know, many hundreds of patents out there, we have more technology than we could ever possibly develop on our own, so I think there are a lot of areas for news and growth from Lineage this year.

Forward Looking Statements for Lineage:
This interview is for informational purposes only and is not an offer to sell or a solicitation of an offer to buy any securities of Lineage Cell Therapeutics, Inc. (“Lineage”). This interview includes certain information obtained from trade and statistical services, third-party publications, and other sources. Lineage has not independently verified such information and there can be no assurance as to its accuracy. All statements in this interview, other than statements of historical fact, are forward-looking statements within the meaning of federal securities laws. In some cases, you can identify forward-looking statements by terms such as “may,” ”will,” “expect,” “plan,” “anticipate,” “strategy,” “designed,” “could,” “intend,” “believe,” “estimate,” “target,” or “potential” and other similar expressions, or the negative of these terms. Forward-looking statements involve risks, uncertainties and assumptions that may cause Lineage’s actual results, performance, or achievements to be materially different from those expressed or implied by the forward-looking statements in this interview, including risks and uncertainties inherent in Lineage’s business and other risks described in Lineage’s filings with the Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports filed with the SEC, including Lineage’s most recent Annual Report on Form 10-K and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
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