ReNeuron has released further follow-up data from the ongoing human retinal progenitor cell (hRPC) trial, which shows a robust sustained averaged response. This data set completes the six-month data on eight patients and extends, for one individual, to 18 months, who showed a good net gain. The next dose level, two million cells in nine patients, remains delayed due to COVID-19. A filing to start a pivotal study is expected in the second half of CY21. Our indicative value remains at £107m.
What is impressive, in our view, is that the therapy, on average, appears to give a clear benefit quickly and then appears stable. In the current one million cell dose cohort, eight patients successfully underwent the procedure; two others had surgical complications, one of whom has fortunately recovered. In our analysis, we continue to look at the successfully implanted patients. With more data points, the net gain in mean visual acuity is now 8.9 letters at six months. One patient so far has 18-month data and displayed a strong response with an impressive 16 letter net gain.
Retinitis pigmentosa (RP) is an inherited, degenerative eye disease caused by one of over 100 different gene mutations. ReNeuron’s hRPC therapy could potentially treat any RP patient, giving a big potential commercial advantage; competing gene therapies only treat specific mutations. ReNeuron has regulatory permission from the FDA to move to a two million cell dose level in its Phase /IIa trial. In the UK, a third trial site, in Oxford, has been approved. However, the COVID-19 situation has so far prevented this cohort from being recruited and treated. In H221, ReNeuron plans to seek approval for a pivotal hRPC study. A pivotal US trial may require a partner, perhaps with a valuable deal. We use a 25% probability of success for hRPC therapy in RP, with launch in 2024.
Our indicative value remains £107m. We will reassess the hRPC valuation as data emerge but note the high deal values in the area with one totalling $250m plus royalties in June. Exosome deals to deliver RNA drugs, with another early stage collaboration announced, are promising but the projects are preclinical. Cash on 31 March 2020 is estimated to be about £8m, pending the year-end results due in July. We envisage a further funding need in FY21.
