The Food and Drug Administration (FDA) has granted Orphan Drug designation to APB-102 (Apic Bio, Inc.) for the treatment of genetic superoxide dismutase 1 (SOD1) enzyme amyotrophic lateral sclerosis (ALS).

SOD1 ALS is caused by mutations in the gene that produces the copper zinc SOD1 enzyme, which leads to the degeneration of motor neurons and muscle control typically seen in ALS. APB-102, an investigational single-dose treatment (administered by intrathecal injection), works by silencing misfolded SOD1 in neurons to reduce abnormal protein accumulation.

Findings from published studies have shown, in some instances, APB-102 to silence 93% of the motor neurons of cynomolgus macaques (Macaca fascicularis) and to be safe in terms of no off-target silencing of mRNA relative expression.

The Company expects to submit an Investigational New Drug Application (IND) in 2020.

“It is gratifying that a clinical trial is being planned for this serious neurological disorder caused by SOD1 mutations; it has been 30 years since this mutation was first identified and now is the time to move toward therapy,” commented Robert Brown DPhil, MD, Professor of Neurology at the University of Massachusetts Medical School and scientific co-founder of Apic Bio.

Related Articles

For more information visit