- A therapy combining eye care drugs from Novartis and Ophthotech has failed two pivotal Phase 3 studies, the companies announced Monday. (Novartis issued a separate statement.)
- Trial results showed pairing the Swiss pharma's blockbuster drug Lucentis (ranibizumab) with the Opthotech's Fovista (pegpleranib) did not significantly improve visual acuity among patients with wet age-related macular degeneration (AMD) compared to treatment with Lucentis alone.
- Wet AMD is an eye disease characterized by enlarged blood vessels that can leak fluid into the eye, damaging the retinas and causing vision loss. While the dry version of the illness is the most common, wet AMD is considered more harmful.
Fovista works to inhibit platelet-derived growth factor (PDGF), a protein that promotes the development of blood vessels. The outcomes from the Phase 3 trials effectively puts the nail in the coffin for anti-PDGF therapies in treating wet AMD, according to a Dec. 12 note from Jefferies.
That's important, the investment bank explained, because it wards off a potential threat to the superstar of wet AMD treatments: Regeneron's Eylea. Jefferies expects Eylea, which targets the disease by inhibiting a different growth factor, to reach $5.1 billion in peak sales by 2024.
Meanwhile, the trial failures are bad for Novartis and even worse for Opthotech.
More than one-third of all Novartis' ophthalmology revenues come from its licensing deal to market Genentech's Lucentis in the U.S. In the most recent quarter, the drug brought Novartis $456 million in sales. That revenue was down 6% from the same period in 2015, though, due to competitive pressures in the wet AMD market, according to a form filed with the Securities and Exchange Commission.
Where Novartis has a diverse product line to fall back on, Ophthotech only has two candidates in the pipeline, with the other being Zimura, a treatment for inflammation associated with wet AMD.
The two companies' stocks reflected those positions. Novartis remained rather unscathed by the trial failures, with shares falling less than 1% in Monday morning trading. Opthotech shares, on the other hand, plummeted, falling almost 85%.
"The key message from the data is that the proven efficacy of Lucentis monotherapy was not improved by the addition of pegpleranib," Novartis' Chief Medical Officer Vasant Narasimhan said in a Dec. 12 statement.
The primary endpoint for the two Phase 3 trials was improved vision as measured by the increase in letters read in the Early Treatment of Diabetic Retinopthy Study eye chart. Across the studies, which recruited nearly 1,250 participants with wet AMD, patients receiving the combination therapy saw on average 10.24 more letters after 12 months of treatment, versus 10.01 letters for those receiving Lucentis monotherapy.
"We are very disappointed in the results from these trials, particularly for patients afflicted with wet AMD," Ophthotech CEO David Guyer said. "We will continue to analyze the data from these two studies to better understand the trial results."