Adverum Biotechnologies Inc (NASDAQ: ADVM) shares were trading higher Monday in reaction to a presentation of data from a Phase 1 study of its experimental gene therapy to treat wet age-related macular degeneration, or AMD.
Efficacy, Safety Established
Adverum said data from six patients in cohort two were treated with a single intravitreal injection of a threefold lower dose of ADVM-022 compared to the first cohort showed that the gene therapy demonstrated robust efficacy and evidence of a dose response.
Four of the six patients in the second cohort remained rescue injection-free at 24 weeks at the low dose, while all six patients in the first cohort remained rescue injection-free at a median follow-up of 50 weeks, the company said.
And for these patients, vision was generally maintained as shown by stable mean best corrected visual acuity compared to baseline, and retinal anatomy improved as shown by mean central subfield thickness, or CST, compared to baseline, according to Adverum.
The company said ADVM-022 was well-tolerated, with no drug-related or procedure-related serious adverse events, no drug-related systemic adverse events and no adverse events meeting the criteria for dose-limiting toxicities.
The presentation was made at the Angiogenesis Exudation and Degeneration 2020.
$50B+ Opportunity Vs. Risks
The update is a positive on CST improvements repeating, Chardan analyst Gbola Amusa said in a note reviewing the presentation.
Yet the analyst expressed concerns over the ocular inflammation signal continuing in the first cohort and appearing in the second.
"The investment controversy to us is therefore balancing the vastness of the market opportunity for a 1x GT (to us, >$50 bn, i.e. the current market's sales times 5-10 years of priced durability) versus the likelihood that ADVM-022 efficacy is not repeated (diminishing with this update) and the likelihood the low-grade inflammation derails the ADVM-022 program," Amusa said.
Roughly doubling the economic contribution of ADVM-022 in Chardan's model, Amusa doubled the price target on the shares to $20 and upgraded the stock from Neutral to Buy.
Adverum shares were adding 20.93% to $12.38 at the time of publication Monday.