Aldeyra Therapeutics, Inc., a biotechnology leader in immune-mediated disease therapies, has announced encouraging results from its Phase 2 clinical trial evaluating ADX-629 in patients with alcohol-associated hepatitis, and is now refocusing its research and development pipeline on next-generation RASP modulator candidates.
In the recently completed trial reported by Drugs.com, four patients with mild to moderate alcohol-associated hepatitis received oral ADX-629 for one month. The study, though small, yielded statistically significant improvements in key markers of liver health and inflammation, including the Model for End-Stage Liver Disease (MELD) score (P=0.001), triglyceride levels (P<0.0001), and C-Reactive Protein levels (P<0.0001). Importantly, there were no serious adverse events, and none were attributed to the drug. These results build on previous clinical findings in other immune-mediated conditions, such as atopic dermatitis, psoriasis, chronic cough, and asthma, highlighting ADX-629’s potential to improve liver function and reduce inflammation.
Despite these positive outcomes, Aldeyra has decided to conclude further company-sponsored development of ADX-629, pending potential investigator-led studies in Sjögren-Larsson Syndrome, a rare metabolic disorder. This decision stems from the company’s strategic shift to concentrate resources on its more advanced and promising RASP modulators, ADX-248 and ADX-246.
The pipeline update is significant. ADX-248, which demonstrated high drug exposure levels in Phase 1 trials with healthy volunteers, will now replace ADX-743 for the treatment of metabolic inflammation, targeting conditions like obesity and hypertriglyceridemia. An Investigational New Drug (IND) application for ADX-248 is now expected in 2026. Similarly, ADX-246, which showed positive results in an animal model of dry age-related macular degeneration (dry AMD), will replace ADX-631 for this indication, with its IND filing also anticipated in 2026.
Financially, these pipeline optimizations extend Aldeyra’s operational runway into the second half of 2027, supporting ongoing and future development. The company also maintains a robust late-stage portfolio, including reproxalap for dry eye disease and allergic conjunctivitis, and ADX-2191 for rare retinal diseases.
Aldeyra’s approach is distinct: rather than targeting single proteins, its therapies modulate broader protein systems, aiming to optimize multiple biological pathways while minimizing toxicity. This strategy underpins its development of RASP (reactive aldehyde species) modulators, which have shown potential across systemic and retinal immune-mediated diseases.
President and CEO Dr. Todd C. Brady emphasized Aldeyra’s commitment to advancing its innovative pipeline in a fiscally responsible manner, ensuring continued growth while addressing significant unmet medical needs.
As Aldeyra advances next-generation RASP modulators toward the clinic, the company signals its confidence in a diversified and targeted approach to immune-mediated disease, backed by strong early clinical data and prudent financial management.
