Abeona Therapeutics (ABEO -11.5%) is down on a 50% spike in volume following its release of updated data from a Phase 1/2 clinical trial assessing gene therapy ABO-102 in patients with Sanfilippo syndrome type A (MPS III A), a rare inherited disorder characterized by the buildup of sugar molecules in tissues and organs leading to a range of health problems.
To date, 11 patients have received a single intravenous injection of ABO-102 which delivers the missing gene associated with the disease.
Reductions in heparan sulfate, the hallmark sugar molecule in MPS III A, in cerebral spinal fluid were 25.8 – 67.1% at day 30 (3 – 4 patients), 58.7 – 83.3% at day 180 (1 -3 patients) and 65.7 – 69.3% at day 360 (2 patients).
In urine, the reductions in heparan sulfate ranged from 29.2 – 90.3% from day 30 to day 540.
Patients experienced durable biophysical reductions of disease burden, including reductions in liver volume.
On the safety front, no treatment-related serious adverse events were observed.
Investors appeared to have sold on the news since shares were initially down almost 26% before recovering.
Previously: Abeona’s gene therapy candidate for Sanfilippo B shows encouraging effect in first patient; shares up 12% (Feb. 7)
Previously: Abeona rebounds from bearish report-stoked selloff, up 1% (Feb. 15)
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