Anavex Life Sciences Corp (AVXL)

[Doc328]

In order to get a basket approval, a company must first run a basket trial. Your example, vemurafenib, was already approved earlier for advanced melanomas with the BRAF-V600 mutations. It was approved in 2019 for patients with BRAF V600-mutant Erdheim-Chester disease (ECD) based on a 2015 basket trial for patients with ECD or non-melanoma BRAF-V600 mutated cancers. The entry criteria was based on the genetic mutation, not the type of cancer or other hematologic disorder. The study included patients with several types of cancer, ECD and Langerhans's cell histiocytosis. The FDA approval, to date, has only been for ECD (was already for melanomas with this mutation).

Basket trials were set up specifically to study rare diseases more efficiently that share a genetic abnormality. The FDA can then approve, based on study results, all the disease studied in the basket, some or as in the case of this drug, one.

Anavex has not initiated a basket trial so any talk of basket approval is premature. Although initially envisioned for cancer research, basket trials can be used for neurodegenerative processes. This is a link to one for a tau agent, TPI-287, for Alzheimer's, 4-repeat PSP or corticobasal degeneration (all having tau tangles)