From Lab to Clinical Trial: First Healthy Volunteer Dosed with OCT-980
Octant, a clinical stage drug discovery company, has recently announced the dosing of the first healthy volunteer in their Phase 1a clinical trial of OCT-980 for Rhodopsin-associated autosomal dominant Retinitis Pigmentosa (RHO-adRP).
In a nutshell:
Octant has started in-human testing of a brand new oral medication that they hope will halt progression of RP associated with certain mutations in the RHO gene that cause protein misfolding. The investigative treatment has now entered a Phase 1/2 clinical trial, beginning with safety testing in healthy volunteers in Australia before moving to genetically confirmed RHO-adRP participants in the US.
What is RP?
Retinitis Pigmentosa (RP) is a form of inherited retinal disease that results in progressive vision loss starting peripherally due to the loss of light-sensing photoreceptor cells in the retina. There are two types of photoreceptors, cones and rods, which differ in structure, function and location. Rods are located in the periphery of the retina and are responsible for night vision and peripheral awareness. Cones are located in the centre of the retina and give you sharp vision and colour detection in good lighting. To learn more, please watch this short animation video on Inherited Retinal Disease.
RHO-adRP is the most common form of autosomal dominant RP, responsible for an estimated 30-40% of cases. It is also among the most severe forms of RP, associated with over 150 different mutations in the RHO gene, found in rod photoreceptor cells. There is currently no approved treatment for RHO-adRP individuals.
What is OCT-980?
OCT-980 is an investigational oral medication that aims to improve low-light vision and halt disease progression in RHO-adRP. OCT-980 is a small molecule corrector therapy designed to address the protein misfolding that occurs as a result of many mutations in the RHO gene, allowing for normal cell processes to occur.
It is a potentially broad treatment for many RHO-adRP mutations, however it is not expected to work universally across every RHO mutation type. This is because mutations can occur in different places in the gene and cause different types of mistakes, disrupting the retina in many different ways, meaning that not all RHO mutations behave identically.
The trial:
The phase 1a portion of this trial is currently underway in Australia. This phase of the trial will evaluate the safety, tolerability and processing of OCT-980 in the bodies of healthy volunteers. Dosing in healthy volunteers is needed for oral medications the first time they are given to humans because we need to understand exactly how the human body processes them and whether there are any safety issues for any body systems, not just the eyes.
The phase 1b portion of the trial will then take place in the US, involving participants with genetically confirmed RHO-adRP.
This phase 1/2 clinical trial hopes to build on the encouraging results seen in preclinical studies, where OCT-980 demonstrated strong efficacy and positive safety profiles.
To learn more, head over to the recording here from last year’s Annual Conference session in which Octant joined us to give a brief overview of Oct-980 at around minute 17 of the video.
If you would like to read more about Octant and OCT-980, please visit their website here. If you would like to read more about the trial specifically, please visit the following link Study Details | NCT07408232 | A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) | ClinicalTrials.gov