Michael Panzara, MD, MPH, Chief Medical Officer at Wave Life Sciences, discusses the company’s clinical trial currently underway that is testing WVE-003 in adults with early stage Huntington disease. 

Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting around 40 years of age. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death. 

Wave Life Sciences is  developing WVE-003, a stereopure antisense oligonucleotide designed to target SNP3, a single nucleotide polymorphism on the mHTT allele. 

The clinical trial [NCT05032196] is currently underway to test dose escalation and dosing frequency in adults with Huntington disease.

Inclusion criteria for the trial include:

  1. Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
  2. Ambulatory, male or female patients aged ≥25 to ≤60 years
  3. Clinical diagnostic motor features of HD, defined as Unified Huntington’s Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
  4. UHDRS Total Functional Capacity Scores ≥9 and ≤13

Exclusion criteria include:

  1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years
  2. Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following:
  3. Received WVE-120101 or WVE-120102 within the last 3 months
  4. Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
  5. Inability to undergo brain MRI (with or without sedation)
  6. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture

For more information about this and other neurologic conditions, visit checkrare.com/diseases/neurology