Axitinib injectable suspension phase 1/2a trial enrolls first patients

The first patients have been enrolled in Clearside Biomedical’s phase 1/2a clinical trial of a proprietary suspension of axitinib for suprachoroidal injection (CLS-AX) in patients with wet age-related macular degeneration (AMD).

Clinical sites—all based in the United States—are activated and currently screening patients with AMD for the OASIS trial, said the company in a prepared statement.

Enrollment of the first patients for the OASIS trial is a key milestone as the company executes a strategy to expand its ophthalmology pipeline with innovative and relevant opportunities targeting critical medical needs through the suprachoroidal space (SCS), said George Lasezkay, PharmD, JD, president and chief executive officer.

“We believe that axitinib, a small-molecule tyrosine kinase inhibitor (TKI), could provide safety and efficacy comparable to, or better than, current standard of care,” Dr. Lasezkay added. “And by delivering axitinib as a suspension into the suprachoroidal space using our in-office, non-surgical SCS Microinjector, we may potentially extend the duration of therapeutic action and reduce or relieve the profound treatment burden for wet AMD patients.”

Data from the first cohort of patients are expected in mid-2021, according to the company.

The phase 1/2a open-label, dose-escalation OASIS study of wet AMD patients aims to assess the safety and tolerability of a single dose of CLS-AX administered by suprachoroidal injection. Eligible patients are those who demonstrate stable visual acuity following 2 or more previous injections with an intravitreal anti-VEGF agent.

Enrolled patients initially receive aflibercept at the first visit followed by a single dose of CLS-AX at the second visit 1 month later. The primary endpoint for the trial will assess the safety and tolerability of CLS-AX for the 3 months following the administration of CLS-AX.

Secondary endpoints will evaluate the pharmacokinetics, visual function, ocular anatomy, and the need for additional treatment with intravitreal aflibercept during the 3-month period.

The study design consists of 3 cohorts of 5 patients each (n = 15). Cohort 1 participants will receive the lowest dose, 0.03 mg of axitinib delivered via suprachoroidal injection. Dose escalation will then proceed following review of the safety data by the Safety Monitoring Committee and their recommendation to advance to the next higher dose cohort.

Additional information on the phase 1/2a trial can be found on https://clinicaltrials.gov (NCT04626128).

In preclinical studies, the agent was observed to be well tolerated and showed significant ocular tissue concentrations over time. These characteristics, if demonstrated clinically, may support the potential for suprachoroidal axitinib to reduce or relieve the treatment burden for patients suffering from wet AMD, according to the company.