Stifel Research2 Oct 2019 08:26
ReNeuron has released further efficacy data from the ongoing Phase IIa trial with its hRPC retinal cell therapy candidate for the treatment of retinitis pigmentosa (RP). In our view the data continues to a show a clear efficacy signal, with an average +17.8 letter gain in visual acuity after three months in the treated eyes of six patients, compared to just +6.8 letter gains in the untreated eyes. Although still early days in terms of duration of response and with small patient numbers, this compares favourably with +6 to +11 average letter gains (after 12 months) observed with blockbuster ophthalmology drugs (e.g. Lucentis or Eylea), while gene therapy approaches in RP have yet to demonstrate a meaningful, if any, improvement in visual acuity. While not all patients have responded as dramatically as the first three patients, this was always unlikely to be repeated, and the overall picture remains highly encouraging and we look forward to additional data on October 12th and further updates thereafter. In our view, the results thus far fully support further clinical development of hRPCs, and ReNeuron's still modest EV of approx £60m does not adequately reflect the value being generated by the programme. Reiterate Buy.
Key Points
Further encouraging data. The mean changes from baseline in visual acuity, as measured by the number of letters read on a standard ETDRS eye chart used in clinical trials, are summarised below:
Recall that initial efficacy observed in the first three patients, +23 letters after 2 to 4 months, was profound and unlikely to be repeated. In subsequent patients treated, with greater levels of visual acuity and retinal activity at baseline (and therefore maybe harder to move the needle as dramatically), the visual improvements have been meaningful but less extreme, ranging from +5 to +11 letters in the treated eye, three months post-treatment.
Notable improvements in visual acuity. While the data are still not mature (we'd like to see the majority of patients reach 9-12 month read-outs), and we'd be interested in the response rate (i.e. % patients with >10 or >15 letter gains), we note that much vaunted gene therapies have yet to deliver meaningful improvements in acuity. Spark Therapeutics' approved Luxturna for RPE65-mutation driven RP showed no improvement in acuity, nor so far has Nightstar's NSR-RPGR, while MeiraGTx's AAV-RPE65 showed just a +4.3 letter improvement in nine patients after six months.
We look forward to more detailed data to be presented at the AAO conference on 12th October by a Principal Investigator on the trial, Dr Pravin Dugel, who commented on the data now released: "I believe that we are seeing a clear signal of efficacy in this patient population where any gain in vision, let alone the levels seen in some of these patients, is so hard to come by and so very much appreciated.”
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