Avalanche gene therapy safe, modestly effective in AMD patients
- In a 12-month, phase II study, AVA-101 was well tolerated in patients with age-related "wet" macular degeneration (AMD).
- Each of the 32 patients in the study had previously been treated with Lucentis (ranibizumab). In addition, Lucentis was the treatment used as rescue therapy in the study. Their vision improved by an average of 2.2 letters—a significant, though modest, result.
- Avalanche's stock was up more than 25% in after-hours trading after the study data was released to the public. UPDATE: But investors don't appear to be very impressed after taking in the full study. In fact, Avalanche's stock has plummeted a staggering 56% in Tuesday trading, with the study's results deemed inadequate.
Currently, the main treatments used for AMD are from a class known as vascular endothelial growth factor (VEGF) directed antibodies, such as Roche's Lucentis and Regneron's Eylea (aflibercept), which had sales of $4.25 billion and $3.03 billion, respectively, in 2014. AVA-101 also uses the same mode of action as this class, but with a different administation process and dosing schedule.
AVA-101 is injected into the lower retina, instead of the vireous fluid. However, there's a distinct advantage associated with AVA-101: AVA-101 inserts a gene that creates the anti-VEGF protein and keeps producing it, meaning if it's successful than it only needs to be given once. In contrast, Lucentis and Eylea injections have to be given frequently.
There is an agreeement between Avalanche and Regeneron in which Regeneron has a time-limited right to of first negotiation to AVA-101 rights. Avalanche is delivering phase II data to Regeneron, and weighing the possibility of partnership.