Optimized Sigma-1 Agonist Method of Responder Selection and Treatment Using Genetics to Predict Who Will Benefit From a Brain-Targeting Drug

This patent describes a way to personalise treatment with drugs that act on the sigma-1 receptor by reading specific genetic markers to predict whether a patient is likely to respond. It is an example of pharmacogenomics, the practice of matching the right medicine to the right patient based on their genes.

The Problem

Many medicines work well for some patients and poorly for others, and brain disorders are a particularly hard place to predict response. Alzheimer’s disease and other neurodevelopmental and neurodegenerative conditions vary enormously between individuals, and trials can be muddied when responders and non-responders are grouped together. If a drug helps a defined subgroup but that subgroup cannot be identified in advance, the benefit can be hidden and patients may be given a treatment that was never likely to help them. A reliable way to pick out probable responders before treatment begins would make both care and clinical studies far more efficient.

What This Invention Does

The patent provides genetic polymorphisms, meaning specific variations in a patient’s DNA, that are associated with an altered response to sigma-1 receptor therapy. By testing a patient for these markers, clinicians can estimate whether that person is likely to respond to a sigma-1 agonist and tailor treatment accordingly. The methods cover detecting the relevant single-nucleotide polymorphisms, using them to select responders, and then treating the selected patients, including for neurodevelopmental and neurodegenerative diseases. In effect, the invention pairs a diagnostic step with a treatment step so the medicine is directed to the people most likely to benefit.

Key Features

• Responder-predicting markers. Specific genetic polymorphisms are linked to how a patient responds to sigma-1 therapy.
• Diagnostic plus treatment. The method combines a genetic test with a tailored treatment decision.
• Focus on brain disorders. The approach targets neurodevelopmental and neurodegenerative conditions.
• Patient selection for trials and care. Identifying responders in advance sharpens both clinical practice and study design.
• Built on sigma-1 biology. The strategy uses the sigma-1 receptor, a target of growing interest in neuroscience.

Who Is Behind It

The applicant is Anavex Life Sciences Corp., a clinical-stage biopharmaceutical company developing sigma-1 receptor agonists for central nervous system disorders. The named inventors are Christopher U. Missling, Mohammad Michel Afshar and Frederic Parmentier. The application is a divisional that traces back to a priority filing from May 2018.

Why It Matters

Personalised medicine is reshaping how neurological drugs are developed and prescribed, and a genetic tool that flags likely responders could improve outcomes while reducing wasted treatment. For a company built around a specific receptor target, owning the markers that predict response is strategically central. Securing the method in Australia protects Anavex’s position as it pursues development and partnering in the region.

Related Concepts

• Sigma-1 receptor – the drug target at the centre of this approach.
• Pharmacogenomics – the science of matching drugs to a patient’s genetics.
• Single-nucleotide polymorphism – the kind of genetic variation used as a marker.
• Alzheimer’s disease – a key condition the therapy is aimed at.
• Personalised medicine – the broader movement this invention supports.

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AU 2026201900 was published in the Australian Official Journal of Patents on 2 April 2026 and is open for public inspection. Patent applications represent inventions that are sought to be protected and do not necessarily reflect commercially available products.