Ophthotech Corporation (Nasdaq: OPHT) has announced that it has reached a second $50 million milestone payment from Novartis Pharma AG as part of an ex-US licensing and commercialization agreement for the company’s lead compound, Fovista. According to the company’s press release, Ophthotech has attained $300 million in upfront fees and milestone payments since May 2014, comprising a $200 million upfront fee and $100 million of $130 million in potential clinical enrollment-based milestones. Fovista, Ophthotech’s anti-platelet-derived growth factor (PDGF) compound, is being assessed in combination with anti-vascular endothelial growth factor (VEGF) therapy for the treatment of wet AMD.
Fovista, a pegylated aptamer capable of binding to PDGF-B with high specificity, is designed to inhibit new blood vessel growth. Previously released Phase IIb clinical trial data for the compound supported the continuation of a wet AMD development programme targeting PDGF-B in combination with ranibizumab (Lucentis). In the Phase IIb study Ophthotech had reported that once-monthly intravitreal Fovista plus ranibizumab met a primary endpoint of improving mean visual acuity from baseline to week 24 vs. ranibizumab alone. Visual acuity, measured by ETDRS, showed that patients in the Fovista arm gained 10.6 letters compared to 6.5 letters with ranibizumab alone (p=0.019). According to the company, the prospective, randomized, controlled clinical trial of 449 patients had no significant safety issues. As an aptamer, Fovista is capable of binding to PDGF-B with high specificity thereby inhibiting new blood vessel growth. Aptamers, in development since the early 1990s are well characterized single-stranded oligonucleotides with specific three-dimensional shapes allowing for selection against a broad range of molecular targets. The global phase 3 wet AMD clinical program, expected to have initial, topline data available in 2016.
Under the Ophthotech-Novartis agreement, Ophthotech has granted Novartis exclusive rights to commercialize Fovista in markets outside the United States, with Ophthotech retaining sole rights to commercialize Fovista in the United States. According to Ophthotech, payments under the agreement could total over $1 billion in upfront and milestone payments, excluding future royalties on ex-US Fovista sales. Commenting on the announcement of the original agreement in May 2014, David R. Guyer, M.D., Chief Executive Officer and Chairman of Ophthotech’s Board stated, “As one of the largest ex-US partnering deals ever in the biotechnology industry, this collaboration with Novartis is potentially transformational for Ophthotech. This agreement represents an important achievement for the Company as we continue to execute on a strategy to deliver science-driven retinal products and offer physicians multiple treatment options to improve patient outcome. The collaboration also supports our previously stated plan to partner Fovista outside the United States while we retain sole commercialization rights to Fovista in the United States. The collaboration not only provides a substantial strategic and financial benefit to Ophthotech, it also begins to put in place essential elements designed to expand the reach of Fovista® outside the United States, following potential regulatory approvals.”