Experimental ALS therapy targeting viral protein receives European patent
Geneuro plans Phase 1 trials in US and Europe in second half of 2027
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- ALS is a progressive neurological condition that damages motor neurons, the nerve cells that control movement.
- The viral protein K Env may contribute to ALS and has been linked to neurodegeneration.
- Geneuro received a European patent for GNK-301, an antibody targeting K Env, with Phase 1 trials planned for the second half of 2027.
Geneuro has received a European patent for GNK-301, its experimental therapy designed to target a viral protein thought to contribute to amyotrophic lateral sclerosis (ALS), and plans to begin Phase 1 clinical trials in the second half of 2027.
The antibody-based therapy was developed through a partnership between Geneuro and scientists at the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH).
It is based on the discovery that HERV-K can produce a protein called K Env, which may play a role in ALS, a progressive disease that damages motor neurons, the nerve cells that control movement.
“The opportunity to partner with the NIH and leading experts across the globe around a biomarker-targeted, potentially disease-modifying drug has the potential to forever change the course of this terrible disease,” Jonathan Javitt, Geneuro’s CEO, said in a press release from NRx Pharmaceuticals, Geneuro’s parent company.
HERV-K may produce toxic protein linked to ALS
HERVs, including HERV-K, are remnants of viruses that infected primates millions of years ago. Their genetic material makes up about 8% of human DNA, but most of it has become inactive during evolution. However, several triggers may disrupt this silencing and reactivate these genes, which can lead to the production of viral proteins such as K Env.
K Env has been detected in the cerebrospinal fluid — the liquid that surrounds the brain and spinal cord — of more than 75% of people with ALS. In preclinical studies, K Env was shown to be toxic to lab-grown human motor neurons and to reduce their electrical activity, while also producing features of ALS-like neurodegeneration in animal models and human motor neuron cultures.
GNK-301 is designed to bind to K Env and block its toxic activity. Preclinical data showed that the therapy protected nerve cells from damage in mice and lab-grown human motor neurons.
The therapy is now being produced according to good manufacturing practice (GMP) standards, which help ensure medicines are consistently produced to appropriate quality standards for use in people. Geneuro plans to begin Phase 1 clinical trials of GNK-301 in the U.S. and Europe during the second half of 2027.
The European patent EP3570878B1, titled “Anti-HERV-K Envelope Antibody and Uses Thereof,” provides patent protection for GNK-301 and its uses for a specified period. Two related U.S. patents, numbers 10,723,787 and 10,981,977, have already been granted. These add to Geneuro’s portfolio of more than 25 issued and pending patents related to HERVs.
Geneuro has previously tested temelimab, an antibody-based therapy that targets the envelope protein of a different retrovirus called HERV-W, in five clinical trials involving more than 400 people with several diseases, including the neurodegenerative condition multiple sclerosis. In MS, temelimab was shown to reduce signs of neurodegeneration. No serious adverse events were reported across the trials.
Geneuro said these findings support the potential for antibody-based therapies targeting HERV envelope proteins to have clinically meaningful effects in people with HERV-mediated neurodegenerative diseases.
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