On 21 February, Ohr Pharmaceuticals announced the closure of its small lab in San Diego, California, US, which focused on its sustained release delivery technology research, just a couple of days after the biotech reported a net loss of approximately $7M for its fiscal quarter ended 31 December, 2016.
According to Ohr, the loss was mainly driven by the high cost in operating expenses. Its shares have been hammered since 2015 when its lead drug candidate Squalamine lactate ophthalmic solution, 0.2% (squalamine), which uses the SKS sustained release technology, failed its Phase II study. A temporary halt of the enrolment of its Phase III study was also reported alongside the financial update.
Squalamine is regarded as a novel potential therapy for neovascular age-related macular degeneration (wet AMD), which develops when fluid leaks from abnormal blood vessels into the macula. The drug is a first-in-class, anti-angiogenic eye solution that has demonstrated safety and effectiveness in wet AMD in early and mid-stage trials. However, squalamine failed to achieve the primary end-goal in its Phase II trial, which was to reduce the frequency of Genentech’s Lucentis (ranibizumab) injections when used as a complementary treatment.
As such, in its current Phase III clinical trial, Ohr aims to investigate the potential of the squalamine combination therapy further, specifically to substantially improve visual acuity in wet AMD patients. However, due to the recent failures of the two high-profile, anti-platelet-derived growth factor (anti-PDGF) inhibitors – Opthotech’s Fovista (pegpleranib sodium) + Lucentis, and Regeneron’s aflibercent + rinucumab – for combination therapy in treating wet AMD, Ohr decided to make a strategic change to its clinical development program.
The company stated that the recent temporary suspension of enrolment of its first Phase III trial is not related to any safety issues. It will carry on with the study for the 200 patients that are currently enrolled for squalamine’s efficacy assessment. This strategic move means that, for now, only a smaller patient population will be measured. However, it allows for the company to begin data collection, and to potentially evaluate and confirm the efficacy of the drug before the end of 2017, which would otherwise be deemed unlikely for the formerly scheduled enrolment number. Still, recommencement of the enrolment is needed for a complete trial to ensure it is in compliance with the protocol prior to submission of a New Drug Application (NDA) to the FDA.
Since squalamine is the only combination drug that is in late-stage development for wet AMD, if it successfully proves its efficacy in its late stage trials, such as vision acuity benefits in patients, it will become the first topical eye drop adjunct therapy to anti-vascular endothelial growth factor (anti-VEGF) injections. Also, due to its patient-friendly formulation of twice daily, self-administered eye drops, and its chemical-based drug characteristics, a lower price is expected. GlobalData therefore anticipates that the drug has potential to achieve a significant market share. The long patent protection further offers a significant advantage to squalamine, as it will cover Ohr’s intellectual property until 2029 in the US. However, Ohr is a small company that lacks sales and marketing experience. As such, once squalamine demonstrates robust safety and efficacy, its commercial success will depend on Ohr’s ability to establish a partnership with a larger pharma company.