The US Food and Drug Administration (FDA) has granted orphan drug designation for ONL Therapeutics’ first-in-class small molecule peptide, ONL1204, for the treatment of retinal detachment.
ONL1204 is designed to protect key retinal cells, including photoreceptors, from cell death that occurs in a range of retinal diseases and conditions.
Retinal detachment occurs when the retina is separated from the underlying layer of cells called the retinal pigment epithelium (RPE), which provides nutritional support to the highly-active photoreceptors in the retina.
It is reported that death of these retinal cells is the root cause of vision loss and the leading cause of blindness.
The company expects to advance ONL1204 into clinical trials for retinal detachment patients later this year.
In the US, retinal detachments occur in about 50,000 people each year and affect people of all ages, although risk increases as people reach 50.
In order to reconnect the photoreceptors with the RPE and prevent additional loss of vision, patients experiencing a retinal detachment are normally treated by surgical reattachment of the retina.
The company noted that 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 centre of vision called the macula, more than a third of patients have a 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 recognised 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."
ONL is a biopharmaceutical firm focused on protecting and improving the vision of patients with retinal disease.
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