Freeline Therapeutics Touts Encouraging Data From Fabry Disease Program
- Freeline Therapeutics Holdings plc (NASDAQ: FRLN) gave an update on its investigational Fabry disease treatment FLT190, currently in a Phase 1/2 trial, saying the candidate proved durable in the second patient tested.
- Fabry disease is an inherited metabolic disorder resulting from the absent or markedly deficient activity of lysosomal enzyme α-galactosidase A, or α-Gal A.
- Patient Two was dosed at the lowest dose cohort of 7.5e11 vg/kg and experienced a sustained and durable response with an increase in expression of plasma α-galactosidase A (α-Gal A), to near-normal levels, from weeks 6 to 16 post-dosing.
- Thus far, the patient remains off enzyme replacement therapy.
- The treatment was well-tolerated with no dose-limiting toxicities or serious adverse events.
- Patient Two has experienced no elevated liver enzymes.
- A third patient will be dosed sometime early next year.
- The biotech also announced that it is working with regulatory authorities to update study protocols for FLT180a for hemophilia B to include additional data collection. It will initiate a Phase 1/2 trial on the investigational candidate Q1 next year.
- Price Action: FRLN shares are down 1.74% at $3.12 during the market session on the last check Wednesday.
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