Aldeyra Therapeutics (ALDX) R&D Day 2025 summary

Pipeline overview and program updates

• RASP platform expanded into central nervous system diseases, adding to ongoing programs in atopic dermatitis, metabolic inflammation, and systemic/retinal immune-mediated diseases.

• Lead candidate reproxalap is at NDA stage for dry eye disease, with a PDUFA date of December 16, 2025.

• ADX-248, a potent, once-daily oral RASP modulator, is in phase I for CNS and immune-mediated diseases, with phase II in atopic dermatitis expected to initiate in H1 2026.

• ADX-2191 is set for pivotal phase III in primary vitreoretinal lymphoma by year-end and phase II/III in retinitis pigmentosa in 1H 2026.

• ADX-246 is in formulation for dry AMD, with IND submission planned for 2026.

Clinical trial data and development milestones

• ADX-629 completed seven phase II trials in immune-mediated diseases, showing safety, anti-inflammatory activity, and improvements in inflammation, lipid profiles, and C-reactive protein.

• ADX-248 demonstrated efficacy in preclinical models of Parkinson’s, ALS, and MS, improving motor function and CNS biomarkers.

• Reproxalap achieved primary endpoint in Phase 3 dry eye chamber trial, with rapid and sustained symptom improvement.

• Phase I trial of ADX-248 is ongoing, with dose escalation and safety/tolerability as primary endpoints; pivotal long-term safety studies expected to complete in 2026.

• Phase 2 trial for ADX-248 in atopic dermatitis and IND submissions for ADX-248 (obesity/hypertriglyceridemia) and ADX-246 (dry AMD) planned for 2026.

R&D strategy and innovation priorities

• Focus on developing first-in-class, systems-based RASP modulators for broad immunology and neuroinflammatory indications.

• Next-generation RASP modulators (ADX-248, ADX-246) show preclinical efficacy in neuroinflammation, Parkinson's, ALS, and atopic dermatitis.

• Emphasis on once-daily oral administration for improved patient compliance.

• Prioritizing atopic dermatitis for ADX-248, with metabolic inflammation and CNS diseases as next targets.