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