Relative to Baseline, Signal-Finding Molecule ADX‑629 Demonstrated Statistically Significant Improvement in Liver Function in Patients with Alcohol-Associated Hepatitis
RASP Modulator Product Candidate Pipeline Focused on ADX‑248 and ADX‑246 for Dermatologic, Metabolic, and Retinal Immune-Mediated Diseases
Cash Runway Extended into the Second Half of 2027
LEXINGTON, Mass.--(BUSINESS WIRE)--Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to discovering and developing innovative therapies designed to treat immune-mediated diseases, today announced achievement of statistically significant improvement in liver function in patients treated with ADX-629, an investigational new drug candidate, and focused the RASP modulator product candidate pipeline on next-generation molecules ADX-248 and ADX-246.
ADX-629, a signal-finding RASP modulator for proof-of-concept clinical testing, was administered orally for one month in a single-arm, multicenter Phase 2 clinical trial in four patients with mild to moderate alcohol-associated hepatitis. Relative to baseline, statistically significant improvement was observed in clinically relevant objective markers of hepatic function and inflammation, including the Model for End-Stage Liver Disease (MELD) score (P=0.001), triglyceride levels (P<0.0001), and levels of C-Reactive Protein (P<0.0001). No serious adverse events were reported, and no adverse events were deemed related to ADX-629.
“Consistent with clinical trials in patients with atopic dermatitis, psoriasis, chronic cough, and asthma, the orally administered RASP modulator ADX-629, relative to baseline, demonstrated activity in improving liver function and reducing inflammation in patients with mild to moderate alcohol-associated hepatitis, a chronic and challenging disease that proves difficult to manage in many cases,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “The positive results announced today, which we look forward to sharing in more detail in the future, mark the culmination of our clinical proof of concept with ADX-629, as we focus our pipeline on next-generation RASP modulators ADX-248 and ADX-246 for the treatment of immune-mediated diseases.”
The RASP modulator product candidate pipeline was updated:
Based on Phase 1 clinical trial results generated to date in healthy volunteers, which suggest high levels of exposure following once-daily oral dosing, RASP modulator ADX-248 replaced ADX-743 for the treatment of metabolic inflammation, including obesity and hypertriglyceridemia; anticipated timing for the filing of an Investigational New Drug (IND) application was updated to 2026.
Based on favorable results in an animal model of a dry form of age-related macular degeneration (dry AMD), RASP modulator ADX-246 replaced ADX-631 for the treatment of dry AMD; anticipated timing for the filing of an IND application was updated to 2026.
Clinical development of ADX-629 was discontinued, pending further investigator-sponsored clinical testing in Sjögren-Larsson Syndrome, a RASP-mediated inborn error of metabolism.
As a result of the pipeline updates, projected operational runway based on 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 dry eye disease, allergic conjunctivitis, primary vitreoretinal lymphoma, and retinitis pigmentosa, Aldeyra is committed to developing a robust pipeline of novel therapeutics in a fiscally prudent manner to maintain growth,” continued Dr. Brady.
About Aldeyra
Aldeyra Therapeutics is a biotechnology company devoted to discovering innovative therapies designed to treat immune-mediated diseases. Our approach is to develop pharmaceuticals that modulate protein systems, instead of directly inhibiting or activating single protein targets, with the goal of optimizing multiple pathways at once while minimizing toxicity. Our product candidates include RASP (reactive aldehyde species) modulators ADX‑248, ADX-246, and chemically related molecules for the potential treatment of systemic and retinal immune-mediated diseases. Our late-stage product candidates are reproxalap, a RASP modulator for the potential treatment of dry eye disease and allergic conjunctivitis, and ADX-2191, a novel formulation of intravitreal methotrexate for the potential treatment of primary vitreoretinal lymphoma and retinitis pigmentosa.
Safe Harbor Statement
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Additional factors may be described in those sections of Aldeyra’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, expected to be filed with the SEC in the fourth quarter of 2025, and Aldeyra’s other filings with the SEC.
In addition to the risks described above and in Aldeyra's other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra's results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
