Why Avalanche Biotechnologies Is Coming Off A Big Day

Shares of Avalanche Biotechnologies Inc AAVL closed up more than 7 percent following positive commentary by TheStreet.com. According to author Alfredo Fontanini, the company is approaching an important clinical trial catalyst. Results from a phase IIa study of Avalanche's AVA-101 in wet age-related macular degeneration is expected to be released no later than July. Fontanini noted that the data will provide the company with another opportunity to "test investors' love affair" with gene therapy. "Wet age-related macular degeneration (wet AMD) is a chronic disease of the eye that is associated with the proliferation of blood vessels in the retina," Fontanini explained. "Blood and fluids can leak from these new vessels, leading to thickening and bulging of the macular portion of the retina. Wet AMD causes a progressive loss of vision." The current standard of care for wet AMD involves drugs which block VEGF (vascular endothelial growth factor). The two leading anti-VEGF drugs for wet AMD are Regeneron Pharmaceuticals Inc REGN's Eylea and Roche Holding Ltd. RHHBY's Lucentis. Fontanini stated that both Eylea and Lucentis are "major medical breakthroughs" but not without their downside, which presents Avalanche an opportunity to thrive. "Avalanche hopes to demonstrate that treatment with its one-time gene therapy AVA-101 can cure wet AMD, thereby eliminating the need for anti-VEGF therapy," Fontanini wrote. "That's the home run scenario, but not the only way for the company to win. AVA-101 might also find a place in the treatment of wet AMD if it can reduce the frequency of anti-VEGF injections required by wet AMD patients." Despite the positive outlook, Fonantini did state that Avalanche has already communicated with investors that the study is not designed to show a statistically significant difference between AVA-101 and the control arm on the efficacy endpoints. Rather, the primary endpoint of the study is safety.