Oral hydralazine · Alzheimer’s disease · 505(b)(2)
A 70-year-old medicine. A new question in Alzheimer’s disease.
NeuroNova Therapeutics is developing oral hydralazine as an investigational disease-modifying therapy for Alzheimer’s disease under the FDA’s 505(b)(2) regulatory pathway.
Hydralazine is an investigational treatment for Alzheimer’s disease. It has not been approved by the U.S. Food and Drug Administration or any other regulatory authority for this use. Its safety and effectiveness for Alzheimer’s disease have not been established.
Published mechanism
Two Nature Communications papers from a UT Southwestern laboratory establish the mechanistic rationale: a defined binding target, sirtuin activation, mitochondrial biogenesis, and antioxidant-pathway signaling.
The science →Clinical rationale
A completed 52-week randomized, placebo-controlled trial of oral hydralazine in mild to moderate Alzheimer’s disease, conducted by academic investigators, with a peer-reviewed protocol published in Scientific Reports.
Trial background →Defined regulatory route
Development under the 505(b)(2) pathway, with a completed FDA pre-IND interaction and written Agency responses defining the US program.
The pipeline →What we are doing, and why
NeuroNova Therapeutics is repositioning hydralazine — an oral medicine first approved in the United States in 1953 — as an investigational disease-modifying therapy for Alzheimer’s disease. The mechanistic rationale was established in peer-reviewed work from a laboratory at UT Southwestern Medical Center. A randomized, placebo-controlled trial conducted by academic investigators provides the clinical rationale for a US program, and a completed pre-IND interaction with FDA defines the requirements for a US registrational program.
Alzheimer’s disease progresses through mitochondrial dysfunction, oxidative stress, and impaired protein clearance. The published record on hydralazine engages each of these processes. Whether that translates into clinical benefit is the question a US registrational program is designed to answer — it has not been answered yet.