Aldeyra Therapeutics, Inc. announced advancement of new RASP modulators and recent preclinical data in obesity in conjunction with an Investor Roundtable scheduled to begin at 8:00 a.m. ET on June 20, 2024. Pipeline Updates: Following positive biomarker results in adults, including near-normalization of lipid profiles, ADX-629, a first-in-class orally administered investigational RASP modulator, advanced to the pediatric cohort of the Phase 2 clinical trial in Sjögren-Larsson Syndrome, a rare inborn error of metabolism caused by mutations in fatty aldehyde dehydrogenase and characterized by cognitive dysfunction and skin disorders. Top-line results from approximately five pediatric patients are expected in 2025.

Based on new results in a preclinical model of obesity, novel RASP modulator ADX-743 was advanced to Investigational New Drug (IND)-enabling studies. ADX-743 is an analog of ADX-629, which has demonstrated lowering of triglycerides and fatty acids in Phase 1 and Phase 2 clinical trials. Pending additional results, Aldeyra expects to submit an IND application for ADX-743 or an alternative RASP modulator for obesity or hypertriglyceridemia in 2025.

Based in part on superior preclinical results in atopic dermatitis and unprecedented exposure in preclinical RASP modulator pharmacokinetic models, ADX-248 will be advanced in lieu of ADX-246 to Phase 1/2 clinical testing in atopic dermatitis. Phase 1 clinical testing of ADX-248 is expected to begin in the second half of 2024. Novel RASP modulator ADX-631 has initiated preclinical testing in models of retinal disease.

ADX-631 is an analog of ADX-103, which previously demonstrated activity in animal models of the dry form of age-related macular degeneration (dry AMD), retinal inflammation, and diabetic macular edema. Aldeyra expects to submit an IND application for ADX-631 or an alternative RASP modulator for the treatment of dry AMD or geographic atrophy in the first half of 2025.