If the status quo continues, ie the rest get improved vision…
Yes, by now there should also be results at 8 weeks for the 3 patients in the MAD 30mcg trial.
It would be great if we see further confirmation of efficacy in this cohort.
But what I am really keen to see is the effect after 2 x 75mcg doses of the treatment in the SAD Part B participants. Will the level of improvement be maintained or will it improve even further?
Will we see a further increase from baseline in retinal sensitivity as measured from baseline, as depicted in this graph in PYC’s 30 August presentation?
I note that Beacon Therapeutics, for whom 12 month microperimetry data was shown in PYC’s graph above, has recently reported 36 month data for their drug, which is a gene therapy to treat X-linked RP. This is a single-treatment, AAV vector-delivered therapy, administered by subretinal injection. As repeat doses are not possible with gene therapies, a sustained duration of response is crucial.
As can be seen from the graph below, although the microperimetry data still showed a clear difference between the treated and untreated eye at 36 months, improvement in the treated eye had receded almost back to baseline.
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(20min delay)