Octant, Inc., a clinical-stage drug discovery company developing corrector therapies for protein misfolding diseases, today announced that the first patient has been dosed in its ongoing Phase 1/2 clinical study evaluating OCT-980, an investigational oral small molecule for the treatment of rhodopsin-associated autosomal dominant Retinitis Pigmentosa (RHO-adRP).
RHO-adRP is an inherited retinal disease caused by mutations in the RHO gene, the most common form of autosomal dominant retinitis pigmentosa in the US and Europe. Most disease-causing RHO variants result in misfolded and mis-trafficked rhodopsin proteins that fail to properly localize within photoreceptor cells, contributing to progressive photoreceptor degeneration and vision loss. There are currently no approved treatments that address the underlying cause of RHO-adRP.
OCT-980 is designed to stabilize misfolded rhodopsin and restore proper cellular trafficking. By directly targeting the underlying protein dysfunction caused by certain RHO variants, OCT-980 aims to improve low-light vision and slow or halt disease progression.
The ongoing Phase 1/2 study of OCT-980 consists of a completed Phase 1a portion in healthy volunteers and an ongoing Phase 1b/2 portion in patients with a genetic diagnosis of RHO-adRP. The Phase 1b/2 is a multi-center study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of OCT-980 in patients, including measures of retinal structure and visual function. More information about the study is available on ClinicalTrials.gov (NCT07408232).
“Patients with RHO-adRP experience progressive vision loss over many years, and new treatment approaches are urgently needed,” said Mark Pennesi, M.D., Ph.D. “The opportunity to evaluate an oral therapy designed to intervene directly in the disease process is particularly exciting. Dosing the first patient is an important step toward understanding whether this approach can preserve vision and ultimately change the course of disease for people with RHO-adRP.”
“Dosing the first patient with RHO-adRP is an important milestone for OCT-980 and the inherited retinal disease community,” said Sri Kosuri, co-founder and Chief Executive Officer of Octant. “We’re excited to now evaluate OCT-980 directly in patients and understand its potential to address the underlying cause of this disease. We’re grateful to the patients, investigators, and clinical teams making this study possible.”
About Octant
Octant is a clinical-stage drug discovery company developing correctors for protein misfolding diseases. Octant’s platform, The Navigator, combines high-throughput synthetic biology, generative chemistry, and AI/ML to discover and develop therapeutics against complex cellular mechanisms in human cells. OCT-980 is a small molecule corrector for RHO-adRP, and Octant’s pipeline includes several other programs across other rare diseases and oncology. For more information visit www.octant.bio.
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