Industry News — Clinical Updates

Squalamine Eye Drops Phase 2 Study Results Demonstrate Improvements in VA

Ohr Pharmaceutical, Inc. (New York, NY) announced positive top-line interim results for its double-masked, placebo-controlled phase 2 clinical trial of Squalamine eye drops in wet-AMD patients.

The data demonstrated a positive benefit in visual function across multiple clinically relevant endpoints. This includes a mean change in visual acuity (VA) at the end-of-study visit for interim analysis of +10.4 letters in the Squalamine eye drops plus ranibizumab PRN arm vs +6.3 letters in the placebo eye drops plus ranibizumab as needed (PRN) arm, a 65% additional relative benefit (P = .18).

Improvements in VA were seen as early as 4 weeks, and the relative difference in VA between the 2 treatment arms continued to increase throughout the study.

All patients received an initial ranibizumab injection followed by ranibizumab PRN based on clinical response. Patients in the first treatment arm received Squalamine eye drops twice daily plus ranibizumab PRN. The other treatment arm received standard-of-care treatment, which was placebo eye drops administered twice daily plus ranibizumab PRN.

This planned interim analysis was conducted on the first 62 patients (29 in the Squalamine arm and 33 in the placebo arm) who completed the entire 9 months of the treatment protocol (approximately 50% of the targeted study population). The Squalamine-treated group demonstrated improved best-corrected visual acuity (BCVA) gains relative to the placebo group at all time points evaluated from 4 to 38 weeks.

In the interim analysis group, 48.3% of Squalamine-treated patients showed BCVA gains of ≥ 15 letters (≥ 3 lines) on the Early Treatment Diabetic Retinopathy Study (ETDRS) eye chart, compared with 21.2% in the placebo arm at the end of the study (P = .025). In addition, patients receiving Squalamine drops were more than twice as likely to gain ≥ 4 and ≥ 5 lines of vision compared with patients in the placebo eye drop arm (≥ 4 lines P = .022, ≥ 5 lines P = .059).

VA gains for the placebo eye drop arm were consistent with those observed in previous clinical studies using ranibizumab monotherapy treatment. Squalamine eye drops were well tolerated and had a comparable safety profile to placebo eye drops.

In the interim analysis, there were no significant differences in the frequency of ranibizumab PRN injections, which was the primary endpoint of the study. The mean number of ranibizumab injections was 6.2 for the Squalamine arm and 6.4 for the placebo arm, which included the baseline injection and any injections required up to and including the final study visit for the interim analysis group.

Updated July 10, 2014