Applied Genetic Technologies’ shares more than doubled yesterday on encouraging data from its retinitis pigmentosa gene therapy. But the jump left AGT capitalised at a mere $169m, a relatively tiny sum for a company that is working with a technology generating much investor excitement – and interest from buyers. Biogen, which now owns AGT’s direct rival Nightstarx, walked away from a deal with AGT that included this project in late 2018, helping to explain the low valuation. The data released yesterday were largely a confirmation of earlier findings, but in a few more patients, with some tracked a little longer. 25 subjects with X-linked retinitis pigmentosa caused by mutations in RPGR have been treated with the XLRP project so far; AGT said yesterday that, of the eight patients that have been followed for six months so far, half showed a sustained increase in visual sensitivity. The crucial question now is why some patients are not responding, and on that there is no answer yet. A pivotal trial is to start by the end of the year, but the company needs to figure out how to identify these non-responders before real progress can be made.
|Pipeline for X-linked retinitis pigmentosa caused by RPGR mutations|
|BIIB112 (NSR-RPGR)||Biogen (from Nightstarx)||NCT03116113 (Xirius)||Phase II/III expansion ongoing|
|XLRP (AGTC-501)||Applied Genetic Technologies||NCT03316560||Phase II results due this year; pivotal targeted for YE'20|
|AAV-RPGR||J&J/Meiragtx||NCT03252847||Phase I/II ongoing|