By Josée Rose
Voyager Therapeutics said it will assess alternate payloads related to its gene therapeutic program related to a form of amyotrophic lateral sclerosis, also known as Lou Gehrig's disease.
The Massachusetts biotechnology company said the decision, which focuses on its superoxide dismutase 1 program, is because emerging three-month non-human primate data suggested that an alternate payload would be necessary to achieve the desired product profile.
An "alternative payload" aims to improve efficacy and reduce side effects by delivering a more potent payload to cancer cells and minimizing damage to healthy tissues.
As a result, Voyager no longer anticipates filing an investigational new drug application for VY9323 in mid-2025.
"Emerging preclinical data indicate the siRNA payload component of VY9323 does not meet our high standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window," the company said. "We hope that we may be able to identify an alternate payload and find a path forward for this program."
Voyager said it now expects its cash runway to extend into mid-2027. The decision on the ALS program does not impact Voyager's other gene therapy programs, and the company expects to file other investigational new drug applications later in 2025.
Shares of Voyager Therapeutics were down 2.6%, to $5.18 in premarket trading. In the past 52 weeks, the stock has been down about 26%.
Write to josee.rose@wsj.com
(END) Dow Jones Newswires
February 11, 2025 07:43 ET (12:43 GMT)
Copyright (c) 2025 Dow Jones & Company, Inc.
免責聲明：投資有風險，本文並非投資建議，以上內容不應被視為任何金融產品的購買或出售要約、建議或邀請，作者或其他用戶的任何相關討論、評論或帖子也不應被視為此類內容。本文僅供一般參考，不考慮您的個人投資目標、財務狀況或需求。TTM對信息的準確性和完整性不承擔任何責任或保證，投資者應自行研究並在投資前尋求專業建議。