Anavex sinks after pulling Alzheimer's filing in EU
Shares in Anavex Life Sciences lost more than a third of their value today after the company abandoned a filing for its Alzheimer's disease therapy, blarcamesine, in the EU.
The New York biotech said it had withdrawn the marketing authorisation application (MAA) for the drug as an add-on to current therapies for Alzheimer's, such as cholinesterase inhibitors, that work by boosting neurotransmitter function.
Shares fell 35% after it revealed that feedback from the EMA's human medicines committee, the CHMP, had made it clear that it was unprepared to deliver a positive opinion recommending blarcamesine for EU approval as a treatment for early-stage Alzheimer's.
The panel said it was minded to turn down the application last year, after which Anavex requested a re-examination, limiting the scope of the MAA to people who do not have a mutation in the SIGMAR1 gene, a group that appeared to have strong responses to the treatment in the clinical trial filed in support of the filing.
Blarcamesine's mechanism is thought to lie in activation of the sigma-1 receptor protein, which the company has claimed can help preserve neuronal viability and reduce the formation of amyloid plaques and tau tangles, two histological hallmarks of Alzheimer's.
The CHMP concluded that the study failed to demonstrate the effectiveness of blarcamesine, for the total study population as well as those without SIGMA1R mutations, and had methodological issues which raised concerns about the validity of the results.
It also said the way side effect data were collected did not allow sufficient characterisation of the safety profile of the drug, and was concerned that the provided quality data made it impossible to rule out the formation of nitrosamine impurities that could potentially cause cancer.
In a statement, Anavex said it "remains committed to the ongoing clinical development of blarcamesine and to advancing potential therapeutic options addressing the significant unmet medical needs of patients living with early Alzheimer's disease."
The company also said it would consider the feedback provided by the CHMP and "focus on gathering additional data and conducting further analyses to address the points raised." In January, it said a meeting with the FDA about a possible filing had resulted in "constructive feedback" from the US regulator.
Anavex also tried to develop blarcamesine as a treatment for children with Rett syndrome – a devastating neurological disorder caused by mutations in the MECP2 gene found on the X chromosome – but a negative trial in 2024 prompted the company to prioritise the Alzheimer's programme.
