Octant Bio Begins Clinical Testing Oral Treatment for Rhodopsin-Related Retinitis Pigmentosa
Research News
First-of-its-kind small molecule therapy targets protein misfolding at the root of RHO-adRP
Octant Bio has dosed the first healthy volunteer in its Phase 1/2 clinical trial of OCT-980, an investigational oral medication for rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). This milestone marks Octant's transition to a clinical-stage company and represents a novel therapeutic approach for one of the most prevalent forms of inherited retinal disease.
The clinical program consists of two phases: Phase 1a is currently underway in Australia, evaluating the safety, tolerability, and the body's processing of OCT-980 in healthy volunteers. This will be followed by Phase 1b/2 studies in the United States involving patients with genetically confirmed RHO-adRP.
OCT-980 represents a breakthrough in how researchers are thinking about treating retinitis pigmentosa. Rather than replacing defective genes, this small molecule therapy aims to correct the underlying protein misfolding that characterizes many forms of RHO-adRP. When rhodopsin proteins are misfolded, they cannot properly function within photoreceptor cells and cause cellular stress, leading to progressive vision loss and eventual blindness.
By targeting the root cause—helping proteins fold correctly and move to their proper locations within cells—OCT-980 has the potential to improve low-light vision and halt disease progression. As an oral medication, it could offer patients a convenient, non-invasive treatment option for this sight-threatening condition.
"We're committed to generating high-quality clinical data that can support safe, rapid progress of OCT-980 into patient studies," said Sri Kosuri, co-founder and CEO of Octant.
RHO-adRP affects thousands of individuals worldwide and currently has no approved treatments. This form of retinitis pigmentosa is caused by mutations in the rhodopsin gene, which is essential for vision in low-light conditions. Patients typically experience night blindness as an early symptom, followed by progressive loss of peripheral vision that can eventually lead to complete blindness.
More information about the study is available on ClinicalTrials.gov.
