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)
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