Aura Biosciences announced the presentation of updated clinical data from its ongoing phase 1b/2 clinical trial evaluating the safety and efficacy of light-activated AU-011, the company’s lead product candidate for the first line treatment of primary choroidal melanoma, at the American Academy of Ophthalmology (AAO) 2019 Annual Meeting.
“With its ability to provide tumor control and vision preservation, AU-011 holds significant potential as a new targeted therapy for the primary treatment of choroidal melanoma,” Cadmus Rich, MD, Chief Medical Officer and Head of Research and Development of Aura Biosciences, said in a company news release. “The data presented this year by Dr. Duker provided information on Aura’s proprietary technology platform and key insights that will inform the design and conduct of our pivotal phase 3 program for AU-011, which we expect to commence in the second half of 2020. Dr. Schefler’s presentation included an update on ongoing research we are conducting into suprachoroidal delivery which, may allow us to increase the range of tumor sizes that AU-011 can treat. Both of these presentations underscore our long-term vision and commitment to bringing this first-in-class technology to patients for this rare and life-threatening disease.”
Updated Results from the Phase 1b/2 Study Evaluating AU-011
Jay S. Duker, MD, Director New England Eye Center, and Professor and Chair, Tufts Medical Center, gave an oral presentation titled, “Novel Management of Choroidal Melanoma–AU-011,” which highlighted the potential of Aura’s viral like particle technology, updated data from the ongoing open-label phase 1b/2 clinical trial, and described the novel design of the planned phase 3 trial that Aura expects to initiate during the second half of 2020.
The phase 1b/2 clinical data presented at AAO demonstrate that multiple administrations of light-activated AU-011 were well-tolerated. Among the patients evaluated for safety (n=46), the most common treatment-related adverse events (AEs) were expected and included anterior chamber inflammation, posterior chamber inflammation and increase in IOP; all were manageable with standard-of-care treatments and the majority resolved without clinical sequelae. Notably, the posterior inflammation appears to originate within and/or around the tumor which is consistent with AU-011’s mechanism of action of acute tumor necrosis. There was one treatment-related severe AE (vision loss; 2%) in one patient with a juxtafoveal tumor.
Tumor control and vision preservation data continue to be supportive of the planned phase 3 registration trial. In the subset of patients with documented tumor growth prior to trial enrollment (n=17), treatment with AU-011 resulted in tumor control in 15 patients (88%; P=0.0117). The results from this ongoing phase 1b/2 study will inform the design of Aura’s planned pivotal phase 3 program for AU-011.
“The data presented this year at AAO show that AU-011 is well tolerated with early signals of efficacy,” said Dr. Duker. “AU-011 is administered via a simple, two-step procedure which includes an intravitreal injection followed by a laser application; all of which is completed in the physician’s office. If approved, AU-011 represents the first potential new treatment for choroidal melanoma in several decades.”
Exploring Suprachoroidal Delivery for AU-011
Amy C. Schefler, MD, Weill Cornell Medical College and Retina Consultants of Houston, gave an oral presentation highlighting the data from the ongoing phase 1b/2 study with intravitreal administration as well as new preclinical research demonstrating the potential advantages of delivering AU-011 using the suprachoroidal route of administration. Aura recently executed a licensing agreement with Clearside Biomedical for use of Clearside’s suprachoroidal space (SCS) Microinjector for the treatment of intraocular cancers. Aura believes that by delivering AU-011 into the SCS, there is the potential for treating a larger number of patients with a good safety profile and a greater range of tumor sizes. Preliminary preclinical pharmacology data showed that AU-011 administered via the SCS Microinjector achieved full necrosis of tumor cells in all animals following laser activation. Further preclinical studies are currently ongoing and Aura expects to initiate clinical testing using suprachoroidal delivery for AU-011 during the first half of 2020.