Aldeyra eyes po­ten­tial­ly piv­otal tri­al in re­tini­tis pig­men­tosa af­ter small open-la­bel read­out

A week af­ter Aldeyra’s drug can­di­date was re­ject­ed by the FDA for a rare eye can­cer, the biotech tout­ed da­ta on the as­set in a small, open-la­bel study in a de­gen­er­a­tive reti­nal dis­ease that leads to blind­ness.

The biotech said its in­trav­it­re­al­ly-in­ject­ed ADX-2191, a for­mu­la­tion of the chemo drug methotrex­ate, met the pri­ma­ry goal of safe­ty in a Phase II tri­al in pa­tients with re­tini­tis pig­men­tosa, who cur­rent­ly have no ap­proved treat­ment op­tion. ADX-2191 led to sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in reti­nal func­tion as com­pared to pa­tients’ vi­su­al acu­ity head­ed in­to the study.

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