- Top-line results from a Phase 1/2 clinical trial evaluating Ionis Pharmaceuticals' (NASDAQ:IONS) antisense drug IONIS-HTTrx in people with early-stage Huntington's disease (HD) showed a treatment effect. The data were presented at the 13th Annual CHDI HD Conference in Palm Springs, CA.
- 46 patients with early HD were treated for 13 weeks with four spinal canal injections (10 mg, 30 mg, 60 mg, 90 mg or 120 mg) of IONIS-HTTrx (RG6042) or placebo administered monthly.
- Significant dose-dependent reductions in mutant huntingtin protein (mHTT) were observed in the cerebrospinal fluid (CSF) of treated patients. Reductions up to 60% were observed in those receiving the two highest doses.
- Based on a predictive model, a 40 - 60% reduction in CSF corresponds to an estimated 55 - 85% reduction in mHTT in the brain's cortex and 20 - 50% in the caudate region (near the center of the brain).
- On the safety front, no serious adverse events were observed. Most adverse events were mild and unrelated to the study drug.
- An open-label extension study is ongoing.
- LIcensee Roche (OTCQX:RHHBY) is planning a pivotal study to assess safety and efficacy.
- Huntington's disease is an inherited disorder characterized by the progressive breakdown of nerve cells in the brain. It affects about 30K Americans.
- Ionis will host a conference call this morning at 11:00 am ET.
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