BOSTON — VectorY Therapeutics said the U.S. Food and Drug Administration has granted Fast Track Designation to VTx-002, a first-in-class vectorized antibody designed to target TDP-43 pathology in amyotrophic lateral sclerosis.
The designation follows recent clearance of the company’s investigational new drug application and is intended to support the continued advancement of VTx-002 toward the planned PIONEER-ALS Phase 1/2 clinical study. Fast Track status is designed to facilitate development and expedite regulatory review of therapies intended to treat serious conditions with significant unmet medical need.
“ALS is a devastating and relentlessly progressive disease with profound unmet medical need, and patients urgently need new therapeutic options,” said Jim Scibetta, chief executive officer of VectorY Therapeutics. “The FDA’s decision to grant Fast Track designation to VTx-002 underscores the seriousness of ALS and the importance of advancing new investigational approaches. This designation enables closer and more frequent interaction with the FDA as we prepare to advance VTx-002 into the upcoming PIONEER-ALS Phase 1/2 clinical study, with the goal of moving as efficiently and responsibly as possible on behalf of patients.”
Fast Track Designation allows for more frequent communication with the FDA and may make a therapy eligible for Priority Review, if relevant criteria are met, as development progresses.
VTx-002 targets TDP-43 pathology, a key driver in up to 97 percent of ALS cases. In December 2025, VectorY announced that the FDA had cleared its IND application for VTx-002 and outlined plans to initiate the PIONEER-ALS Phase 1/2 study, which is designed to evaluate the safety, tolerability, and pharmacodynamic activity of the therapy in people living with ALS.
VectorY said it plans to enroll the first patient in the PIONEER-ALS study in early 2026.
