A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)

This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP.

Octant, Inc. is evaluating OCT-980, an oral small molecule corrector for the treatment of patients with rhodopsin (RHO)-associated autosomal dominant retinitis pigmentosa (RHO-adRP). There is no known cure and no currently approved therapy for RHO-adRP, a disease of the retina characterized by progressive loss of vision. This study is comprised of a Phase 1a, to be conducted in Australia; and a Phase 1b/2, to be conducted in the US. The phase 1a study will be a single dose, double-masked, placebo-controlled ascending dose escalation (SAD +/- food effect) to evaluate the safety, tolerability, and PK of OCT-980 in healthy volunteers. The phase 1b/2 study will be open-label, multi-center multiple ascending dose escalation (MAD) to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of multiple-ascending doses of OCT-980 for up to 48 weeks, in participants with a genetic diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RP).

Inclusion Phase 1a (Single Ascending Dose Escalation in Healthy Volunteers) Inclusion: * Be in good general health, as determined by the Investigator * Male or female age 18 to 65 years (inclusive) at the time of signing the informed consent form * Have a body weight \>50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m\^2 (inclusive) at screening * Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples * Participants in Cohort 2b will be asked to participate in an additional food effect cohort and capable of consuming the high-fat meal within the protocol-specified time period and willing to consume 100% of the high-fat meal Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Inclusion: * Participants must have a diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa confirmed by a genetic test that is implemented in accordance with the relevant national requirements * Male or female age 18 to 65 years (inclusive) at the time of signing the informed consent form * Have a body weight \>50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m\^2 (inclusive) at screening * Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) \> 70 * Other than their diagnosis of RHO-adRP, be in good general health, as determined by the Investigator * Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples Exclusion Phase 1a (Single Ascending Dose Escalation in Healthy Volunteers) Exclusion: * Females who are pregnant or breastfeeding * Have evidence of any significant ocular or non-ocular disease/ disorder * Has lifetime history of ocular surgery * Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Exclusion: * Females who are pregnant or breastfeeding * Evidence of any other significant ocular (other than RHO-adRP) and non-ocular disease/ disorder * Lifetime history of ocular surgery * Any prior or current ophthalmologic gene therapy * Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study