Ocular Therapeutix (NSDQ:OCUL) yesterday said its lead drug candidate, OTX-DP, failed to meet a key endpoint the 2nd clinical trial of the eye drug in reducing pain and inflammation after cataract surgery.
The news, coming just a month after OTX-DP met the goals of a 1st Phase III trial, sent OCUL shares down 33.5% to $25.46 apiece in pre-market trading today.
The treatment is designed to deliver sustained dosage over 4 weeks of the drug dexamethasone, using a hydrogel plug inserted into a tear duct. The plug then dissolves and is flushed from the body as tears. In March, Bedford, Mass.-based Ocular said the 1st, 247-patient trial showed that 33.7% of patients treated with OTX-DP showed no inflammatory cells in the eye after 14 days, compared to 14.6% of those receiving a placebo. Some 76.1% of patients receiving the drug reported no pain after 8 days, compared to 36.1% of the placebo arm.
Yesterday Ocular said the 2nd, 240-patient trial met its pain endpoint – absence of pain after 8 days – but missed the inflammation endpoint, absence of inflammatory cells after 14 days. Although 77.5% of patients in the OTX-DP arm reported no pain after 8 days, compared with 58.8% of the control arm, the difference in inflammatory cell counts after 14 days was not statistically significant (39.4% vs. 31.3%), the company said, noting that both endpoints had to be met to consider the study a success.
There were no ocular or treatment-related serious adverse events in either trial and the rate of serious adverse events was lower for the treatment group than for the control group, the company said. The Company expects to be able to use the safety data from these Phase 3 trials to support its other OTX-DP clinical development programs including allergic conjunctivitis.
President & CEO Amar Sawhney said the company plans to meet with the FDA "promptly" to discuss the results and "chart the appropriate path forward." The safety data, he said, "will serve as a foundation for the regulatory submissions for other indications with this product."
Ocular Therapeutix said it plans to pursue other indications for OTP-DX, including allergic conjunctivitis.
"Although the efficacy results for the absence of inflammatory cells in the OTX-DP treatment group met our expectations, the placebo group response was significantly higher than expected. We have begun a thorough analysis of the data from the 2nd Phase 3 trial to fully understand the difference in efficacy between these 2 trials that had essentially the same trial design and similar patient populations," Sawhney said in prepared remarks, noting that a post-hoc analysis of the data from both trials showed a significant difference in the absence or low level of inflammatory cells (66.3% for OTX-DP vs. 42.5% for the placebo arm).
Material from Reuters was used in this report.