Vaccinex' pepinemab disappoints in Huntington's disease study

23 September 2020
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Shares of US biotech firm Vaccinex (Nasdaq: VCNX) tanked more than 60% pre-market on Tuesday, after the company revealed that the mid-stage SIGNAL trial of its pepinemab failed to meet pre-specified co-primary endpoints.

The company announced top-line results from the early manifest treatment arm (Cohort B1, N=179) of the Phase II double-blind, placebo-controlled SIGNAL trial of its lead clinical candidate, pepinemab, in patients with early manifest and prodromal Huntington’s disease (HD).

Maurice Zauderer, president and chief executive of Vaccinex remarked on the trial results: “HD affects multiple regions of the brain and disease progression impacts many critical functions including cognition and motor activity. The results reported today strongly support a cognitive benefit to treatment with pepinemab and indicate that treatment with pepinemab antibody potentially targets cortical centers, including those that govern cognition. The first sign of HD is often chorea and cognitive disturbances generally follow later in disease progression. We believe the data, therefore, suggest that patients at a somewhat more advanced stage of HD may derive the greatest benefit from pepinemab.”

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