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Update: WAVE Life Sciences Discloses 'Positive' Phase 1b/2a Data From Huntington's Disease Treatment But Shares Fall on Neurofilament Concern
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Update: WAVE Life Sciences Discloses 'Positive' Phase 1b/2a Data From Huntington's Disease Treatment But Shares Fall on Neurofilament Concern
Jun 26, 2024 2:44 AM

05:19 AM EDT, 06/26/2024 (MT Newswires) -- (Updates with the stock move and analyst comments in the fifth, sixth, and seventh paragraphs.)

WAVE Life Sciences ( WVE ) said Tuesday it has seen "positive" results from a Phase 1b/2a trial of WVE-003 to treat people with Huntington's disease by lowering mutant huntingtin protein and preserving healthy, wild-type huntingtin protein.

In the multidose portion of the trial, WVE-003 was "generally safe and well-tolerated, with no serious adverse events reported," the company said.

Wave Life Sciences ( WVE ) also said the "strong data compel a case for accelerated approval for WVE-003."

However, results also showed most WVE-003-treated participants had neurofilament light protein (NfL) levels in the "range of placebo or had NfL levels that increased and returned to the range of placebo."

Shares of the biotechnology company dropped 8.5% at the close on Tuesday.

Despite the positive data, the shares were under pressure mainly due to lingering concerns around fluctuating neurofilament light, according to a note from Truist Securities Tuesday.

"NfL is a cytoskeletal scaffold protein that is only expressed by neurons," Truist analysts, including Joon Lee, said. "Damaged neuronal cells may shed NfL into the [cerebrospinal fluid] and the blood and is considered a biomarker for neuronal health. Some fluctuations in NfL [are] raising some investor concerns."

Huntington's disease is a neurological disorder.

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