This Penny Stock Touts Encouraging Long-Term Data For Hemophilia Gene Therapy

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  • Small biotech Freeline Therapeutics Holdings plc FRLN hopes new long-term data will help boost its case.
  • The company announced that the New England Journal of Medicine (NEJM) published positive data, expanding upon previously announced long-term follow-up data for up to 3.5 years from the Phase 1/2 B-AMAZE trial of hemophilia B gene therapy candidate, FLT180a.
  • Hemophilia is an inherited bleeding disorder caused by a deficiency in coagulation factor IX (FIX).
  • Related: Freeline Posts Cases Of Lower FIX Expression, Higher Liver Enzymes From Hemophilia Candidate.
  • Nine of 10 patients maintained their factor IX activity at a median follow-up of 27.2 months, with one patient seeing a response as far out as 42.4 months. Researchers published their findings in the New England Journal of Medicine.
  • Freeline’s study also saw one patient with the highest factor IX levels experienced a severe adverse event of arteriovenous fistula thrombosis.
  • The company said that enrollment in Phase 1/2 B-LIEVE dose confirmation trial will conclude by the end of Q3 enabling pivotal Phase 3 start-up activities in 1H of 2023.
  • Price Action: FRLN shares are down 1.54% at $0.76 during the market session on the last check Thursday.
Posted In: BiotechNewsPenny StocksHealth CareGeneralBriefsgene therapy
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