Octant, Inc. (“Octant”), a clinical-stage drug discovery company developing correctors for protein misfolding diseases,  announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to OCT-980 for the treatment of retinitis pigmentosa (RP). The designation includes rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), the indication for which OCT-980 is currently being developed.

OCT-980 is an investigational oral small molecule designed to address RHO-adRP at its source by correcting the underlying protein misfolding and restoring proper cellular trafficking of rhodopsin. It is currently being evaluated in a Phase 1/2 clinical study.

“People with RHO-adRP are living with progressive vision loss and no approved treatment options today,” said Sri Kosuri, co-founder and Chief Executive Officer of Octant. “This designation is another step forward as we advance OCT-980. With our Phase 1b/2 study underway at U.S. sites, we are focused on generating the clinical data needed to establish OCT-980 as an oral, disease-modifying option for RHO-adRP.”

Retinitis pigmentosa is a group of inherited retinal disorders that cause progressive vision loss and can ultimately lead to blindness. RHO-adRP is caused by disease-associated variants in the rhodopsin (RHO) gene. Many of these variants result in misfolded and mistrafficked rhodopsin proteins that fail to properly localize within photoreceptor cells, contributing to progressive vision loss.

The FDA grants Orphan Drug Designation to drugs intended to treat rare diseases or conditions. The designation provides certain development incentives, including potential tax credits for qualified clinical trials, exemption from certain FDA application fees, and eligibility for seven years of U.S. market exclusivity following regulatory approval, subject to applicable requirements.