JNJ-2113 Shows Promise for Psoriasis, Infigratinib for Achondroplasia, and Gildeuretinol Gains FDA Support for Stargardt Disease

Key Insights

• Phase 3 trials of JNJ-2113 demonstrate potential as a first-in-class oral peptide targeting the IL-23 receptor for moderate-to-severe plaque psoriasis in patients aged 12 years and older.
• Infigratinib shows statistically significant increases in annualized height velocity and improved body proportionality in children with achondroplasia in a Phase 2 study.
• Gildeuretinol receives Rare Pediatric Disease and Fast Track designations from the FDA for Stargardt disease, an inherited condition causing vision loss in children and young adults.

New data has emerged regarding potential treatments for various conditions affecting children and young adults, including plaque psoriasis, achondroplasia, and Stargardt disease.

JNJ-2113 for Plaque Psoriasis

Johnson & Johnson's investigational oral peptide, JNJ-2113 (formerly PN-235), has shown promising Phase 3 results for treating moderate-to-severe plaque psoriasis in patients aged 12 years and older. The ICONIC-LEAD (NCT06095115) and ICONIC-TOTAL (NCT06095102) trials evaluated JNJ-2113, a potential first-in-class targeted oral peptide designed to block the IL-23 receptor. The ICONIC-LEAD trial, which enrolled 684 individuals, included patients diagnosed with plaque psoriasis, with or without psoriatic arthritis, for at least 26 weeks, with a total body surface area greater than or equal to 10%, a Psoriasis Area and Severity Index (PASI) greater than or equal to 12, and an Investigator Global Assessment (IGA) greater than or equal to 3.

Infigratinib for Achondroplasia

Phase 2 data published in The New England Journal of Medicine (NEJM) indicates that infigratinib treatment resulted in a statistically significant and sustained increase in annualized height velocity (AHV), positive mean change in baseline in height Z-score, and improved body proportionality in children with achondroplasia. The PROPEL 2 study (NCT04265651) evaluated the safety and efficacy of the oral bioavailable FGFR1–3 selective tyrosine kinase inhibitor in children aged 3 to 11 years. The study enrolled 72 children across 5 sequential cohorts. Achondroplasia is a genetic skeletal condition resulting in disproportionately short stature and medical complications throughout life.

Gildeuretinol for Stargardt Disease

Gildeuretinol, an investigational oral therapy developed by Alkeus Pharmaceuticals for Stargardt disease, has received Rare Pediatric Disease and Fast Track designations. Stargardt disease can result in severe vision impairment in children and young adults, with no treatments currently approved. It is estimated to affect between 30,000 and 87,000 people in the United States. Gildeuretinol is a new chemical entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. The therapy is being studied in the Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) clinical program, which includes four independent studies of oral gildeuretinol: TEASE-1, TEASE-2, TEASE-3, and TEASE-4.