ONL1204 is a novel, first-in-class small molecule peptide designed to protect key retinal cells, including photoreceptors, from cell death that occurs in a range of retinal diseases and conditions. Death of these retinal cells is the root cause of vision loss and the leading cause of blindness. ONL expects to advance ONL1204 into clinical trials for retinal detachment patients in 2016.
Retinal detachment occurs when the retina is separated from the underlying layer of cells called the retinal pigment epithelium (RPE). The RPE provides nutritional support to the highly-active photoreceptors in the retina. When there is a detachment, the photoreceptors no longer receive these nutrients and undergo cell death processes that dramatically impact a patient’s vision.
Retinal detachments occur in approximately 50,000 people each year in the United States and affect people of all ages, although risk increases as people reach fifty years of age.
Patients experiencing a retinal detachment are normally treated by surgical reattachment of the retina to reconnect the photoreceptors with the RPE and prevent additional loss of vision. However, these procedures do not address the photoreceptor death and vision loss, which can be significant, that occurs prior to surgery.
ONL1204 will be delivered to patients upon diagnosis and is intended to block photoreceptor cells from dying until surgery can be completed.
ONL Therapeutics co-founder and chief science officer David Zacks said: "When retinal detachments involve the center of vision called the macula, more than a third of patients have final best corrected vision of 20/60 or worse after successful surgery.
"Those are truly poor outcomes from successful surgeries. We are very pleased the FDA has recognized this need and that ONL is the only company to have received an orphan designation for this disease. It reinforces our belief that ONL1204 can play a key role in preventing vision loss in these patients by protecting their photoreceptors."
The FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions that affect less than 200,000 individuals in the US. A drug candidate and its developer must meet several key criteria in order to qualify for, and obtain, orphan drug status.
Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.