Actinogen Medical (ASX:ACW) has reached a critical regulatory milestone in its bid to bring a novel Alzheimer’s treatment to market, securing agreement with the US Food and Drug Administration (FDA) on the path forward for its lead therapy, Xanamem (emestedastat).
The company confirmed that in a recent Type C meeting, conducted by written response with the FDA’s Neurology-I Division, regulators endorsed Actinogen’s streamlined development plan. The agreement sets out the manufacturing, clinical, and non-clinical requirements needed to support a future US marketing application.
Among the key outcomes were clarity on the regulatory starting materials for Xanamem’s commercial manufacture, the design of an interim analysis for the ongoing XanaMIA trial, and agreement on the structure of a single pivotal Phase 3 trial. The FDA also backed a simplified trial design using a single 10 mg daily dose of Xanamem compared with placebo, along with a limited set of ancillary pharmacology and nonclinical studies.
“This is clear guidance from the FDA that confirms our plans for streamlined development of Xanamem in Alzheimer’s disease,” said Dr Steven Gourlay, Chief Executive Officer and Managing Director of Actinogen. “Importantly, the Agency agreed with our approach for only one additional pivotal trial using a single 10 mg dose design versus placebo to support a marketing application in the US.”
The agreement provides Actinogen with greater certainty as it advances discussions with potential development and marketing partners. It also paves the way for submissions to other regulators, including the European Medicines Agency in 2026 and subsequently the UK Medicines and Healthcare products Regulatory Agency.
Xanamem is a first-in-class, once-daily oral therapy that works by inhibiting the enzyme 11β-HSD1, reducing cortisol inside brain cells. Elevated cortisol levels are strongly linked to the progression of Alzheimer’s disease and are associated with memory loss and damage to brain cells. Actinogen is also investigating Xanamem’s potential in depression, Fragile X syndrome, and other neurological and psychiatric conditions.
The company’s XanaMIA Phase 2b/3 trial is currently underway in Australia and the US, testing Xanamem in 220 patients with mild to moderate Alzheimer’s confirmed by biomarker analysis. Initial results from an interim analysis are expected in January 2026, with final outcomes anticipated later that year.
For Actinogen, the FDA’s green light represents more than regulatory progress. It is validation of the company’s scientific approach in a field where treatment options remain limited and patient need is urgent.