Aldeyra Therapeutics Inc., a biotechnology company focused on developing therapies for immune-mediated diseases, announced statistically significant improvements in liver function among patients treated with ADX-629, an investigational RASP modulator. The company also outlined plans to prioritize next-generation candidates ADX-248 and ADX-246 within its RASP modulator pipeline.
Phase 2 Results for ADX-629
In a single-arm, multicenter Phase 2 clinical trial, ADX-629 was administered orally for one month to four patients with mild to moderate alcohol-associated hepatitis.
Compared with baseline, patients demonstrated statistically significant improvement in key markers of liver function and inflammation, including:
- Model for End-Stage Liver Disease (MELD) score: P=0.001
- Triglyceride levels: P<0.0001
- C-Reactive Protein (CRP) levels: P<0.0001
No serious adverse events were reported, and no adverse events were attributed to ADX-629.
“Consistent with earlier trials in atopic dermatitis, psoriasis, chronic cough, and asthma, ADX-629 demonstrated measurable improvements in liver function and inflammation,” said Todd C. Brady, President and CEO of Aldeyra Therapeutics. “These results mark the culmination of ADX-629’s proof-of-concept work as we advance our next-generation RASP modulators for immune-mediated diseases.”
Pipeline Realignment
Following the completion of ADX-629’s clinical program, Aldeyra has redirected focus toward its newer candidates:
- ADX-248: A next-generation RASP modulator for metabolic inflammation, including obesity and hypertriglyceridemia, showing high exposure with once-daily dosing in Phase 1 trials. An Investigational New Drug (IND) application is expected in 2026.
- ADX-246: A RASP modulator for dry age-related macular degeneration (dry AMD), selected following favorable preclinical data. IND submission is also anticipated in 2026.
Clinical development of ADX-629 will be discontinued, except for potential investigator-sponsored studies in Sjögren-Larsson Syndrome, a RASP-mediated metabolic disorder.
Financial Outlook
Aldeyra stated that following these pipeline adjustments, its operational runway—based on current cash, cash equivalents, and marketable securities—has been extended into the second half of 2027.
“In addition to our late-stage, pre-commercial programs in ophthalmic and rare diseases, Aldeyra remains committed to building a sustainable pipeline of novel therapeutics,” added Dr. Brady. “We continue to execute our development strategy in a fiscally disciplined manner to support long-term growth.”
