Octant, a clinical-stage drug discovery company developing treatments for protein misfolding diseases, has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for OCT-980 for the treatment of Retinitis Pigmentosa (RP), including rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), the condition for which the investigational drug is currently being developed.

OCT-980 is an investigational oral small molecule designed to correct the underlying protein misfolding and restore the proper cellular trafficking of rhodopsin. The drug is being evaluated in a Phase 1/2 clinical study, including a Phase 1b/2 study underway at sites in the United States.

Sri Kosuri, Co-Founder and Chief Executive Officer of Octant, said patients with RHO-adRP experience progressive vision loss and currently have no approved treatment options. He added that the company is advancing OCT-980 and focusing on generating clinical data to evaluate its potential as an oral, disease-modifying treatment for the condition.

Retinitis pigmentosa comprises a group of inherited retinal disorders characterised by progressive vision loss that 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 rhodopsin proteins that do not reach their proper locations within photoreceptor cells, contributing to progressive vision loss.

OCT-980 is designed to address this mechanism by correcting rhodopsin protein misfolding and supporting its proper cellular trafficking. Its clinical evaluation is intended to assess the investigational treatment’s safety and other clinical characteristics as development progresses.

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, exemptions from certain FDA application fees and eligibility for seven years of US market exclusivity following regulatory approval, subject to applicable requirements.

Octant develops therapeutics for protein misfolding diseases using its Navigator platform, which combines high-throughput synthetic biology, generative chemistry and artificial intelligence and machine learning. The company’s pipeline includes OCT-980 for RHO-adRP and other programmes targeting rare diseases and oncology.