What happened

Abeona Therapeutics (NASDAQ:ABEO) jumped 14% on Wednesday after the company released data from its phase 1/2 trial testing ABO-101, a gene therapy treatment for patients with MPS IIIB, commonly referred to as Sanfilippo syndrome Type B.

So what

The data was only from the first patient enrolled in the trial, but it looks promising nonetheless. In addition to being well tolerated, without side effects through the 30 days of follow-up, there were also early signs that the treatment is working.

ABO-101, which is designed to express N-acetyl-(alpha)-D-glucosaminidase (NAGLU) that degrades heparan sulfate and is mutated in patients with Sanfilippo Type B, increased the NAGLU enzyme activity by more than 300-fold over baseline at 30 days after the treatment. The increased activity resulted in reduced heparan sulfate levels of 50% to 69% in the patient's cerebral spinal fluid, urine, plasma and urinary total glycosaminoglycan. The reduction in cerebral spinal fluid is especially significant since issues with Sanfilippo patients are mainly neurological.

Now what

Today's data is certainly good news for Abeona, but investors will need to see more before calling the treatment a success. The company plans to test two more patients at this dose level before testing a higher dose on three more patients.

Doctor examining boy with a stethoscope

Image source: Getty Images.

Despite its nomenclature, ABO-101 isn't Abeona's most advanced gene therapy; ABO-102 for Sanfilippo syndrome Type A is further along, as is EB-101. Abeona is testing the latter for recessive dystrophic epidermolysis bullosa, which is set to enter phase 3 development this year.

Simply due to their ability to generate revenue sooner, investors should be more focused on EB-101 and ABO-102, with ABO-101 as icing on the cake -- or a potential backup plan if things don't work out with the lead therapies.

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