Wave Life Sciences: FOCUS-C9 trial


Professor Tim Anderson; Neurologist, Department of Medicine, University of Otago Christchurch; Clinical Director New Zealand Brain Research Institute, Christchurch

Dr Julian Bauer; Neurologist Te Whatu Ora Te Toka Tumai Auckland

Current Research

Wave Life Sciences Ltd is a genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.  They have developed an investigation drug WVE-004, and it is specifically designed for MND caused by a mutation in the C9orf72 gene or C9orf72-associated frontotemporal dementia (FTD).

What does the drug do?

WVE-004 uses an approach known as ‘antisense’, where the drug directly interferes with the faulty instructions for making a protein. The C9orf72 gene contains three different sets of RNA instructions. RNA is an acid in the chromosomes of the cells of living things which plays an important part in passing instructions about protein structure from the cell nucleus (where the cell’s DNA is arranged into chromosomes) to the outer part of the cell where proteins are made. In some forms of MND, errors in the C9orf72 gene cause two of the RNA to be faulty and this leads to the production of abnormal proteins that form toxic clumps inside cells. WVE-004 is designed to reduce the production of these toxic proteins.

Phase 1b/2a – FOCUS-C9

The Phase 1b/2a study known as FOCUS-C9 aims to evaluate the safety and tolerability of WVE-004 in people with MND or FTD with a documented mutation in the C9orf72 gene. This study started recruitment overseas last year and is continuing to recruit across Europe, Canada, Australia and now New Zealand. In this study, participants with FTD or MND are given either the active WVE-004 medication or placebo administered into the fluid that surrounds the spinal cord and brain via an injection near the bottom of the back (intrathecal injection) – like a lumbar puncture which takes fluid out but putting fluid in. The principal investigators for New Zealand, are Professor Tim Anderson in Christchurch and Dr Julian Bauer in Auckland. They are recruiting participants from anywhere in New Zealand.

In April 2022 WAVE Life Science released preliminary data reporting that the drug had shown a marked decrease in poly(GP) levels in the cerebrospinal fluid (CSF), Poly(GP) is one of the toxic proteins produced from the faulty RNA in the C9orf72 gene.

Who is eligible to participate

Individuals diagnosed with C9orf72-associated MND or FTD may be eligible. There are several inclusion and exclusion criteria for the trial. If you think you are eligible to participate in this trial please email the New Zealand MND Registry mndregistry@adhb.govt.nz 

Additional resources 

Additional information about this trial is available in the following links.

ALS News today –‘WVE-004 Reduces Toxic Proteins, Early FOCUS-C9 Data Suggests’. Click here to read this article.

MND Research Blog – “Clinical trials to keep an eye out for in 2022”. Scroll down to read the WVE-004 (FOCUS-C9). Find out more here.

ClinicalTrials.gov – This is a registry of all clinical trials. “Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) (FOCUS-C9)”. Click here to read more information about this clinical trial. 

Wave Life Sciences April 2022 – Interim results show some reduction in key biomarker Poly(GP). Further results expected in 2022. Find out more here.

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