What happened
Shares of Abeona Therapeutics Inc. (ABEO), a clinical-stage biotech developing novel gene therapies to treat rare genetic disorders, popped 14% higher on Wednesday morning after an important announcement concerning a rare disease candidate that succeeded in an early stage trial earlier this month. Although the initiation of a pivotal expansion to the study is indeed good news, this morning's pop has fizzled to a gain of 6.0% as of 11:11 a.m. EDT.
So what
Abeona Therapeutics is developing ABO-102 as a potential gene therapy for the treatment of MPS IIIA, a rare metabolic disease caused by a faulty SGSH gene. There are no available treatments for the life-threatening disorder, but results of a small, early-stage clinical trial announced earlier this month suggest Abeona could have the first.
Image source: Getty Images.
MPS IIIA patients seldom survive past their second decade because they can't clear heparin sulfate which leads to liver and brain damage. One year after receiving a single injection of ABO-102, though, two patients demonstrated a 69% heparin sulfate reduction in the fluid that bathes the brain, and dramatically reduced liver volumes as well.
Investors were thrilled with today's announcement because the company has expanded the ongoing trial in a way that could be enough to support the company's first new drug application. Because the highest dose appeared well-tolerated, the highest dose cohort will be used to that will be used to support a new drug application. Since even the lower dose appears highly effective, this candidate has a solid shot at an eventual approval.
Now what
ABO-102 is just one of several irons Abeona has in the fire at the moment. In August the jumped higher when the FDA granted EB-101 a breakthrough therapy designation for the treatment of recessive dystrophic epidermolysis bullosa, a debilitating skin disease. The company will begin a pivotal phase 3 study for this program early next year.
Although the stock has already soared about 337% this year, a recent market cap of about $856 million suggests market-beating long-term gains ahead if either candidate entering pivotal trials succeeds
