Clinical Experience Trial (NORSE ONE)
The first registration clinical trial in our Phase 3 program evaluating ONS-5010 to treat wet AMD is a clinical experience trial. In August 2020 we reported topline results and positive proof-of-concept data for safety and efficacy. In this initial clinical experience trial, we saw the positive trends in efficacy in 3-line visual acuity gains we were looking for, and ONS-5010 was well-tolerated in this study. Importantly, it showed no ocular adverse events related to inflammation. The results support the established design, inclusion criteria and protocol for the ongoing pivotal Phase 3 clinical trial evaluating ONS-5010 for treatment of wet AMD. Our initial clinical experience trial was conducted in Australia and is a superiority trial comparing ONS-5010 with an alternate dosing regimen of ranibizumab (LUCENTIS®).
Pivotal Phase 3 Trial (NORSE TWO)
The second of two registration clinical trials in our Phase 3 program for ONS-5010 to treat wet AMD is our pivotal trial. It is underway in the United States, and in July 2020 completed enrollment of 227 patients who will be treated for 11 months. We expect to complete this pivotal trial in the third quarter of 2021. This pivotal trial also is a superiority trial comparing ONS-5010 with an alternate dosing regimen of ranibizumab (LUCENTIS®). Together with the results of the clinical experience trial and an additional upcoming safety trial that is intended to ensure that a sufficient number of patients have been exposed to ONS-5010, results from our Phase 3 program are expected to be sufficient to form the basis for a new BLA filing with the FDA in late 2021.
Safety Trial (NORSE THREE)
Our third clinical trial investigating ONS-5010 for treatment of wet AMD is an open-label safety study that will be conducted to ensure that an adequate number of safety exposures to ONS-5010 are available for the initial BLA regulatory filing. We expect to initiate this safety trial in the second half of 2020.
Branch Retinal Vein Occlusion
Outlook Therapeutics intends to initiate a registration clinical trial that will evaluate ONS-5010 for use in treating BRVO.
Diabetic Macular Edema
Outlook Therapeutics also intends to commence two registration clinical trials to evaluate ONS-5010 in treating DME.
Regulatory Approval Plans
If our clinical program is successful, it will support our plans to submit for regulatory approval of ONS-5010 in multiple markets. Because there is currently no bevacizumab approved for ophthalmic indications in the United States, we will file ONS-5010 with the FDA as a new BLA under the PHSA 351(a) regulatory pathway, not as a biosimilar.
Outlook Therapeutics has tentatively been granted an ATC code for ophthalmic bevacizumab by the World Health Organization. Also, SME Entity Status has been granted by the EMA.