PathAI’s AIM-MASH AI Assist Receives Historic FDA Qualification for MASH Clinical Trials

The U.S. FDA has granted Drug Development Tool (DDT) biomarker qualification to PathAI’s AIM‑MASH AI Assist for use in MASH clinical trials, making it the first AI‑enabled pathology tool to receive this type of FDA qualification in drug development.13

This qualification allows AIM‑MASH to be used as a qualified biomarker tool in Phase 2 and Phase 3 MASH clinical trials for both enrollment and histologic endpoint assessment, under a clarified regulatory pathway.13

The FDA decision follows earlier European Medicines Agency (EMA) qualification, giving AIM‑MASH the first dual U.S. and EU regulatory status for an AI‑based pathology biomarker in drug development.1

AIM‑MASH is designed to standardize liver biopsy scoring for key MASH features—fibrosis, steatosis, ballooning, and inflammation—aiming to reduce inter‑reader variability, a longstanding source of inconsistency in MASH trial readouts.12

Validation data submitted to FDA showed AIM‑MASH was non‑inferior to average individual pathologist assessments for fibrosis and steatosis, superior to expert manual reads for ballooning and inflammation, and achieved 100% repeatability with improved reproducibility across precision metrics.1

Because small differences in histologic grading can affect perceived drug efficacy, regulators indicated that AIM‑MASH’s higher consistency may improve trial integrity and confidence in histologic endpoints.1

With DDT qualification, pharmaceutical sponsors can integrate AIM‑MASH into IND, NDA, and BLA submissions without revalidating its methodology each time, potentially streamlining development timelines for MASH therapies.13

The tool operates as an AI-assisted pathology solution, enabling a single pathologist, supported by the algorithm, to apply standardized scoring criteria across studies, rather than relying on multiple independent manual readers.12

According to expert commentary cited in the announcement, dual FDA–EMA qualification provides a regulator‑approved path for sponsors globally to use AI‑assisted histology in MASH drug development, supporting broader adoption and more uniform trial design.1

PathAI positions AIM‑MASH and AIM‑MASH+ as research-use-only AI measurement tools that generate CRN‑based MASH activity and fibrosis scores (including continuous scores in AIM‑MASH+) to better capture drug effects and subtle histologic changes in clinical trials.2

Sources:

1. https://hlth.com/insights/news/pathai-secures-fda-qualification-for-aim-mash-the-first-ai-enabled-pathology-tool-for-drug-development-2025-12-10

2. https://www.pathai.com/mash

3. https://markets.businessinsider.com/news/stocks/pathai-s-aim-mash-ai-assist-becomes-first-ai-powered-tool-to-receive-fda-qualification-for-mash-clinical-trials-1035630288