BiQ Journal: OpRegen 36-Month Results 05/04/26
Lineage Cell Therapeutics (LCTX) recently shared 3-year updated clinical data from its P1/2 trial of OpRegen, evaluating a single administration of OpRegen allogeneic cell therapy in patients with bilateral GA secondary to AMD:
- Mean BCVA Gain (Cohort 4) of +6.2 letters at 36-months vs. +5.5 letters at 24-months.
- BCVA Gain for patients with extensive coverage of +9.0 letters at 36-months vs. +7.4 letters at 24-months.
- Structural improvement of +1.9mm2 at 36-months vs. +2.6mm2 at 24-months.
- In untreated eyes, -3.8mm2 RPE loss at 36-months vs. -2.8mm2 RPE loss at 24-months.
What the data demonstrates is that a single administration of OpRegen leads to long-term, ongoing improvement out to 36 months in treated eyes, while untreated eyes continue to decline. Compare this to currently approved complement inhibitors, such as Syfovre or Izervay, which, at best, only slow the rate of vision decline. Furthermore, these results are from a single administration of OpRegen, whereas currently approved treatments typically require ongoing monthly or bi-monthly injections.
The data also show that patients who receive extensive cell coverage across the lesion achieve greater gains than those with partial coverage. As for safety, no serious treatment-related AEs have been reported. However, there were some side effects that occurred as a result of the surgery itself, including conjunctival hemorrhage, epiretinal membrane, and two cases of retinal detachment. All were mild or repaired with follow-up surgery, with no loss of vision gains. Also, keep in mind these resulted from the surgical procedure, not from the OpRegen cell therapy. Roche, Lineage's partner for OpRegen, has developed a proprietary surgical device intended to both improve lesion coverage and reduce surgery-related AEs.
LCTX has partnered with Roche to develop and commercialize OpRegen. Roche is currently conducting the P2a GAlette trial using a proprietary delivery device intended to facilitate extensive coverage of lesion areas and reduce surgery-related AEs. This device was not used in LCTX's P1/2 trial; therefore, if the proprietary GAlette device performs as expected, patients could potentially see even better results when we see GAlette data late this year or early next year.
While the 36-month data shown above are from a relatively small patient population, this type of vision improvement is unheard of. I find the fact that a single administration of OpRegen can lead to long-term benefits out to at least 36-months is a very bullish signal for Roche's ongoing P2a GAlette trial. If GAlette demonstrates similar or better results, this could make OpRegen an attractive candidate for Accelerated Approval or Breakthrough Therapy Designations.
Geographic Atrophy (GA) affects approximately 1 million people in the US and an estimated 5 to 8 million people worldwide. If OpRegen were to capture only patients refractory to currently approved complement inhibitors, GA would still represent a $1.5B to $2B peak-revenue opportunity. However, given its clinical profile, I believe OpRegen has the potential to capture a much larger share of the market, so I would consider a $2B TAM to be a very conservative estimate. Per terms of the licensing agreement, LCTX is entitled to over $600M in additional clinical and commercial milestones, plus double-digit royalties on sales.
OpRegen is the most advanced asset in LCTX's pipeline; however, the company is also advancing several wholly owned programs for Spinal Cord Injury, Auditory Neuropathy, Corneal Endothelial Disease, Type 1 Diabetes, and Retinitis Pigmentosa--all based on its allogeneic cell therapy platform. The current Market Cap is around $350M, and the pro forma FDEV is around $360M. The current cash balance of $61M, excluding any additional milestone payments or licensing revenue, is sufficient to fund planned operations through the second quarter of 2028.
LCTX is not part of the official BiQ Active Portfolio; however, it is an idea I have shared with BiQ members previously in the BiQ Chat forum.
LCTX share price at time of publication: $1.42
BiQ Adjusted Cost Basis (ACB): $.78
Please note that the BiQ ACB includes any open options positions, which are accounted for using a "worst case scenario" assumption, plus trading profits (and losses) around my core position.
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