Takeaways From Abeona Therapeutics Initial Gene Therapy Data

Shares of Abeona Therapeutics Inc ABEO were trading higher by more than 11 percent Thursday after the company reported initial data from a 30-day study involving three Sanfillipo Type A patients treated with the company's therapy candidate ABO-102 at low doses (5E12vg/kg).

According to Cantor Fitzgerald's analyst Elemer Piros, Ph.D., the data marks a promising start for the clinical trial.

The analyst wrote:

"A one-time treatment with ABO-102 resulted in mean reductions in CSF gylocosaminoglycan (GAG)-levels of 25.6% + 0.8% and urine GAG decrease of 57.6% + 8.2% within one month. Moreover, these patients had liver and spleen reductions of 17.1% + 1.9% and 17.6% + 7.1%, respectively, as determined by MRI. As a reminder, the three patients were enrolled initially into a MPS IIIA natural history study in which patients had an average liver and spleen enlargement of 116% and 88%, respectively. This enlargement remained unchanged at the time of rollover into the Phase 1/2 study."

Piros added that while the results were encouraging, further confirmation of durability is required. In the meantime, the analyst sees the potential for improvement at the next planned dose of 1E13 vg/kg and the company has already received the FDA's approval to enroll patients.

Shares of Abeona remain Buy rated with an unchanged $21 price target.