WVE - Wave Life stock rises as WVE-003 shows promise in interim data of Huntington's disease trial

Wave Life Sciences ( NASDAQ: WVE ) said a single dose of 30 mg or 60 mg WVE-003 helped reduce mutant huntingtin (mHTT) protein in cerebrospinal fluid (CSF) in patients with Huntington's disease (HD) who are in the early stages and carry SNP3 in association with their cytosine-adenine-guanine (CAG) expansion, in a phase 1b/2a trial.

HD is a rare, genetic disease which causes progressive degeneration of nerve cells in the brain.

In addition, preliminary data from the study, dubbed called SELECT-HD, showed that wild-type huntingtin (wtHTT) protein was preserved, the company said in a Sept. 20 press release.

"These preliminary data suggest WVE-003 is working as intended: to selectively reduce the toxic mHTT protein while avoiding targeting the healthy, wild-type huntingtin protein, thereby preserving its beneficial effects in the central nervous system," said Ralf Reilmann, member of the SELECT-HD clinical advisory and dose escalation committees.

In the ongoing study, 18 people have been dosed who received different doses of the drug or a place (30 mg n=4; 60 mg n=4; 90 mg n=4; placebo n=6).

Participants enrolled at 30 mg, 60 mg and placebo had adequate follow-up to day 85 for biomarker analysis and were included in the analysis. In the 90-mg group none had reached day 85 so they not included in the analysis.

In the 30 mg and 60 mg groups, the mean reduction in CSF mHTT from baseline was 22% (median reduction 30%) at 85 days after a single dose of WVE-003, the company added.

Wave noted that the difference in the mean reduction in CSF mHTT compared to placebo was 35%

The company said single doses of WVE-003 up to 90 mg appeared generally safe and well-tolerated.

Wave Life added that adverse events (AEs) were balanced across treatment groups, including placebo, and all were mild to moderate.

WVE +3.24% to $3.50 premarket Sept. 20

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