ROP treatment receives orphan drug designation

TREM-1 peptide inhibitor granted orphan drug designation for retinopathy of prematurity

Key takeaways:

• The treatment is designed to address retinopathy of prematurity through a ligand-independent mechanism of action.
• Data will be presented at the Association for Research in Vision and Ophthalmology meeting.

The FDA granted orphan drug designation to a triggering receptor expressed on myeloid cells 1 peptide inhibitor for the treatment of retinopathy of prematurity, according to a press release from SignaBlok.

“Current therapeutic options (eye surgery, laser photocoagulation and eye injections of anti-VEGF drugs) are harmful, do not cure, have a high risk of serious complications and are often ineffective, suggesting an unmet need for new therapies,” Alexander B. Sigalov, PhD, president and founder of SignaBlok, told Healio. “SignaBlok addresses this urgent unmet need by introducing a novel, first-in-class, well-tolerable and nature-inspired peptide inhibitor that targets an inflammation-amplifying receptor TREM-1, an emerging target in preventing and treating pathological retinal neovascularization.”

The inhibitor is designed to address the challenge of targeting triggering receptor expressed on myeloid cells 1 (TREM-1), an inflammation amplifier, through a “new, ligand-independent mechanism of action,” the release said. Data on the inhibitor will be presented at the Association for Research in Vision and Ophthalmology meeting in May.

The FDA grants orphan drug status to drugs that show promise for treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S. SignaBlok may now qualify for development incentives including tax credits for clinical testing, prescription drug user fee exemptions and marketing exclusivity for 7 years if the treatment is approved.

“Orphan drug designation for ROP represents a significant milestone in further development of this therapy,” Sigalov told Healio. “Importantly, a unique mechanism of action of our inhibitor significantly reduces the risk of drug failure in clinical trials, creating hope for premature babies affected by ROP.”

For more information:

Alexander B. Sigalov, PhD, can be reached at [email protected].