OKYO Pharma Limited (NASDAQ:OKYO) saw its stock climb 4.7% on Wednesday following confirmation from the U.S. Food and Drug Administration that the biopharmaceutical company’s proposed primary endpoint for its Phase 2b/3 study of urcosimod in neuropathic corneal pain (NCP) is clinically meaningful.
The FDA validated the company’s selection of the Visual Analogue Scale (VAS) pain reduction at Week 12 as the primary efficacy measure, explicitly recognizing that a 2-point or greater improvement on the VAS constitutes a meaningful treatment effect. In addition to endorsing the primary endpoint, the agency agreed with OKYO’s proposed study design, sample size and powering assumptions.
Regulatory feedback also supported the use of the Ocular Pain Assessment Survey as appropriate supportive evidence of quality-of-life benefit. The FDA indicated alignment with the company’s Chemistry, Manufacturing and Controls - CMC - strategy related to the program.
"This alignment represents an important step forward and underscores the potential to address a significant unmet medical need in patients with neuropathic corneal pain," said Raj Patil, Chief Scientific Officer at OKYO Pharma.
OKYO, which operates from London and New York, plans to begin a 120-patient Phase 2b/3 multiple-dose trial of urcosimod for NCP in the first half of this year. The investigational therapy has previously received fast track designation from the FDA and was granted the first Investigational New Drug application to treat patients with neuropathic corneal pain.
The company focuses its development efforts on therapies aimed at neuropathic corneal pain and inflammatory eye diseases. The FDA’s confirmation of the trial’s primary endpoint and related elements of the protocol represents a regulatory milestone that the company says enables the planned progression to a larger, pivotal study.
Context and next steps
With the agency’s agreement on key design elements and outcome measures, OKYO is positioned to move forward with trial initiation as planned. The upcoming Phase 2b/3 multiple-dose study is designed to enroll 120 patients and will use VAS pain reduction at Week 12 as its primary endpoint, with supportive quality-of-life data collected via the Ocular Pain Assessment Survey.
Investors responded to the regulatory alignment by bidding the stock higher on Wednesday, reflecting market recognition of the cleared path for the planned study. The company will need to execute on initiation and enrollment to advance the program through the pivotal testing phase.