The first set of 12-month data from the ongoing retinitis pigmentosa (RP) eye study has been released showing a stable longer-term response, which is excellent. However, the 12-month data are only for three patients so far with mean numbers reported. ReNeuron aims to present more RP eye data over 2020. Stroke data from the CTX PISCES III trial are expected around mid-2021. We retain the indicative value of ReNeuron at £197m.
The US trial (NCT02464436) has now reported data on eight patients to three months, six patients to six months and three at 12 months (Exhibit 1). All these patients had successful surgery, but two others experienced surgical complications and lost sight. The time course data are very supportive (Exhibit 2), with rapid gains in the treated eye followed by a stable mean visual acuity gain of 15 letters using EDTRS (we calculated between six and 12 months). It is interesting the acuity gain seen averages six letters in the untreated eye; this takes longer to develop, from two to three months after treatment. So far, these longer-term data are an average of a few patients (Exhibit 1) with no range or SD reported, but will become more robust as more patient data is gained. The 12-month data on six patients are due in Q320, with all eight currently reported by late 2020.
RP is an inherited, degenerative eye disease caused by one of over 100 different gene mutations. ReNeuron’s human retinal progenitor cell (hRPC) therapy could potentially treat any RP patient giving a big potential commercial advantage. Gene therapies can only treat specific mutations. As a next stage, the current Phase i/IIa US trial will be extended at a higher dose and a new site in the UK is planned. ReNeuron and its clinical advisers believe that the amended protocol will enable the efficacy signal observed so far to be both better delineated and magnified. A pivotal hRPC study is being planned with an application possible in H1 2021. The data so far supports running such a study. Running a larger US trial may require a US partner
We retain our indicative value of £197m or 624p per share. This uses a 25% probability of success for hRP therapy. It is possible that hRPC could become the major valuation contributor over 2020. The CTX stroke indication is a major unmet need, but the PISCES III trial cannot produce data before mid-2021 with our expected launch window being from 2024 onwards.