Yes, endpoints such as Best Corrected Visual Acuity won’t be of much help here given the slow moving nature of the disease. Other companies have faced the same problem and have developed novel endpoints.
Rohan did address this question in the webinar last month (~43 min mark). He mentioned that perimetry will be the key and mentioned assessment with electroretinograms and also use of the FDA-accepted multi-luminance mobility test (MLMT) test which was used by Spark during development of Luxturna for RPE65. He said that the endpoints for PYC’s trial have been worked on with clinicians and experts around the world, with a collaboration with the University of Oslo being particularly useful.
I suggest you also look at the outcomes being used in the RP11 Natural History study (link below) which commenced enrolment in July last year. All 5 US sites are currently recruiting.
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