Shares in Aerpio Pharmaceuticals were more than halved in early trading this morning after it announced the failure of its leading drug candidate.
The midstage test, known as TIME-2b study, was assessing its drug AKB-9778 in diabetic retinopathy, but missed its primary endpoint of a two-step reduction in its diabetic retinopathy severity score (DRSS) score when pitted against a dummy treatment.
The percentage of patients achieving this endpoint for the injectable AKB-9779 twice daily and placebo were 9.6% and 3.8%, respectively (p=0.270).Across all eyes, those achieving this endpoint was 8.6% and 2.7%, for AKB-9778 BID and placebo, respectively (p=0.158).
As ever, when a therapy fails to meet its primary endpoint, the biotech looked to secondaries, where they found changes in Urine Albumin-Creatinine Ratio (UACR), a measure of kidney function, and in intraocular pressure (IOP).
“While we are disappointed in the primary endpoint results of this study, we are nevertheless encouraged by the fact that several other promising findings observed in our prior 3-month Phase 2a trial have been prospectively confirmed in this 1-year trial,” said Stephen Hoffman, M.D., Ph.D., CEO of Aerpio.
“We and our clinical advisors believe that collectively these data support a potentially important role of the Tie2 pathway for the treatment of diabetic complications, as well as for open angle glaucoma. After a full analysis of the study data, we plan to provide an update on the status of the NPDR program. We would like to thank the patients and investigators that participated in this trial.”
The biotech did say that it “plans to advance a topical drop formulation of AKB-9778 into clinical development and expects to initiate a Phase 1b study in the second quarter of 2019 with results anticipated by the end of 2019.”
Shares were down 64% premarket on the news.