What happened
Shares of Adverum Biotechnologies (ADVM -1.94%) rose as much as 40% Monday morning after the company reported updated interim results from a phase 1 study evaluating ADVM-022 as a potential treatment for wet age-related macular degeneration (AMD). Wet AMD is a chronic eye disease that causes blurred vision and blind spots.
The company's gene therapy was administered as a high dose or a low dose. All six individuals who received the high dose were rescue injection-free at a median follow-up of 50 weeks. Four of the six individuals who received the low dose were rescue injection-free at a median follow up of 24 weeks. Prior to enrolling in the study, those individuals required an average of 9.3 injections per year.
As of 11:24 a.m. EST, the pharma stock had settled to a 27.2% gain.
Image source: Getty Images.
So what
The standard treatments for wet AMD are anti-VEGF drugs such as Avastin, Lucentis, and Eylea, which slow the growth of blood vessels central to the eye disorder's pathology. These drugs must be injected directly into the eye when used as rescue medications, and can produce several side effects.
The inconvenience of current treatment options makes wet AMD an ideal target for one-time genetic medicines such as gene therapies. While wet AMD is not a genetic disorder, gene therapies aim to deliver the instructions for producing a protective protein within certain eye cells.
The interim results for ADVM-022 suggest that the drug candidate will be a competitor in the industry's wet AMD pipeline, which also includes a gene therapy candidate from Regenxbio and several next-generation monoclonal antibodies from heavyweights such as Roche.
Now what
Adverum Biotechnologies ended September with only $73 million in cash and reported an operating loss of $49 million in the first nine months of 2019. Therefore, investors shouldn't be too surprised if the company pulls the trigger on a public stock offering in the coming days to take advantage of its soaring stock price to raise much-needed funding. Beyond that, more results for ADVM-022 will be presented in 2020, which will now be closely watched by analysts and investors.
