Biotech Values

[DewDiligence]

Re: GENR SPA
Primary endpoint and statistical threshold:

>>However, I find it odd that they don't mention the visual acuity standards or the statistical significance that the FDA is looking for when they agreed to the SPA.<<

GENR’s PR says the primary endpoint is visual acuity (VA) at one year (even though the duration of the trials is two years). This was also the case in the Macugen phase-3 trials.

The specific VA endpoint is almost certainly the same one used in the Macugen trials: avoidance of a loss of 3+ lines relative to baseline on the standard eye chart.

The statistical threshold specified in the SPA is presumably p<0.05 in each of the two phase-3 trials using the Hochberg correction to adjust for the fact that GENR is testing two doses (20mg and 40mg).

Applying the Hochberg correction means that each of the phase-3 trials will be considered statistically significant if:

1. p<0.05 for both the 20mg and 40mg doses;

or

2. p<0.025 for one of the two doses.

Dew