Apic Bio announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to APB-102, a gene therapy soon to be in clinical development for the treatment of genetic SOD1 amyotrophic lateral sclerosis (ALS).

The U.S. FDA Orphan Drug program provides orphan designation to novel drugs that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States). The designation provides sponsors with development and commercial incentives including seven years of market exclusivity in the US, consultation by FDA on clinical study design, potential for expedited drug development, and certain fee exemptions and reductions.

The incidence of ALS is estimated to be 1.5 to 2.5 cases in 100,000 persons in the United States and in Europe, or up to about 30,000 new cases of ALS per year in those areas. It is estimated that 10% of all cases are thought to be inherited as a dominant trait, or otherwise known as Familial ALS (FALS.) Approximately 15 to 20 percent of FALS cases are caused by mutations in the gene that produces the copper zinc superoxide dismutase 1 (SOD1) enzyme, which leads to a progressive degeneration of motor neurons affecting movement and muscle control.

"This orphan drug designation represents an important recognition by the FDA of APB-102’s potential to treat SOD1 ALS, a rare genetic form of ALS," said John Reilly, CEO and Co-Founder of Apic Bio. "Current treatments only offer modest benefits and do not target the genetic cause of the disease, leaving a significant unmet need."

"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.

APB-102 is being developed as a novel, one-time treatment of genetic (SOD1) ALS. Following a single administration given by intrathecal injection, APB-102 is designed to silence misfolded SOD1 in neurons thereby reducing abnormal protein accumulation. In published studies the artificial miRNA achieved, in some instances, 93% silencing the motor neurons of cynomolgus macaques (Macaca fascicularis) and has been shown to be safe in terms of no off-target silencing of mRNA relative expression. APB-102 has the potential to be an important treatment option for patients with SOD1 ALS. The company expects to submit an IND in 2020.

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