Abstract 842P
Background
Choroidal melanoma is the most common primary intraocular malignancy in adults. Many subjects with a melanocytic choroidal tumor of indeterminate malignancy (i.e., ‘indeterminate lesion’) or small choroidal melanoma (IL/CM) are monitored clinically or treated with radiotherapy, which may lead to severe and irreversible vision loss. AU-011 (belzupacap sarotalocan) is a virus-like drug conjugate (VDC) currently being investigated as a potential first-line vision-sparing treatment. The dual mechanism of action consists of AU-011 selectively binding to malignant melanoma cells, causing acute necrosis upon light activation and potential long term anti-tumor immunity. The current Phase 2 trial is designed to evaluate the safety and efficacy of AU-011 when administered via suprachoroidal (SC) injection. Trial design using SC administration and interim safety from the open-label dose escalation phase will be presented.
Methods
Phase 2, multicenter trial being conducted at 22 ocular oncology sites in the US. The trial included 6 single- and multiple-dose escalation cohorts to be followed by a randomized confirmatory phase. In dose escalation, adult subjects received up to 3 cycles of 3 weekly AU-011 treatments via SC administration with a maximum dose of 80μg with 2 laser applications.
Results
Preliminary safety results include 17 subjects in the dose escalation phase. The most common adverse events (AE) related to drug or laser were anterior chamber inflammation in 4 subjects, conjunctival hyperemia, eye pain, and punctate keratitis in 2 subjects each. There were no dose-limiting toxicities, treatment-related serious or grade 3/4 AEs, vitritis or serious AEs of vision loss. Two subjects developed 5 serious AEs unrelated to treatment.
Conclusions
Preliminary results indicate AU-011 to have a favorable safety profile, supporting its potential to be a first-line therapy for the treatment of IL/CM, especially in early-stage disease where observation may be commonly employed. Efficacy data including visual acuity and tumor control from the dose escalation phase will be reported later this year.
Clinical trial identification
NCT04417530.
Editorial acknowledgement
Legal entity responsible for the study
Aura Biosciences.
Funding
Aura Bioscience.
Disclosure
H. Demirci: Financial Interests, Personal, Advisory Board: Castle Bioscience; Financial Interests, Institutional, Other, Investigator: Aura Bioscience. A. Narvekar: Financial Interests, Personal, Full or part-time Employment: Aura Biosciences. C. Murray: Financial Interests, Personal, Full or part-time Employment: Aura Biosciences. C. Rich: Financial Interests, Personal, Full or part-time Employment: Aura Biosciences.