Aldeyra Therapeutics has reshuffled its pipeline, terminating the development of a Phase II asset despite meeting its endpoints in alcohol-associated hepatitis.
The biotech announced the Phase II data showing that ADX-629, a signal-finding RASP modulator, significantly improved markers of hepatic function and inflammation, including the Model for End-Stage Liver Disease (MELD) score, triglyceride levels, and levels of C-Reactive Protein.
Despite this success, the asset will be culled in favour of two next generation candidates ADX-248 and ADX-246.
Aldeyra president and CEO Dr Todd Brady said: “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.”
This is supported by a Phase I study of ADX-248, which has replaced ADX-743, for the treatment of metabolic inflammation, including obesity and hypertriglyceridemia, with Aldeyra anticipating filing an investigational new drug (IND) application with the US Food and Drug Administration (FDA) in 2026.
An animal model of dry age-related macular degeneration (dry AMD) supported the development of ASP modulator ADX-246, an asset that replaced ADX-631, with the IND also expected to be filed next year.
The pipeline updates have extended the projected operational runway based on cash, cash equivalents, and marketable securities has been extended into the second half of 2027.
Dr Brady continued: “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.”
While Aldeyra’s stock initially dropped 8%, from a market open of $5.13 to a low of $4.70 on 28 October, by market close it had recovered, closing the day at $5.14 a share. The company has a market cap of $308.6m.
In April 2025, the FDA refused to approve Aldeyra’s dry eye drug reproxalap, stating in a complete response (CR) letter that the candidate failed to demonstrate efficacy, requesting a further trial to show a positive effect of the treatment. This was the second rejection for the candidate, with the first arriving in 2023.
The 2025 setback hit the company hard, with the stock dropping 72%. The company has brought this back however, with the current stock price similar to that before the CR letter was received.
