Beam Therapeutics Temporarily Retreats Clinical Plans For Second Sickle Cell Gene Therapy

  • Last November, the FDA signed off Beam Therapeutics Inc's BEAM Investigational New Drug (IND) application for Phase 1/2 trial for BEAM-101 gene therapy for sickle cell disease.
  • In the most recent earnings release, the company said it is still working on enrolling its first patient for the trial, dubbed BEACON-101, by year-end.
  • The company announced in its Q3 update that it's pushing back plans to file an IND in 2022 for BEAM-102, another sickle cell gene therapy candidate.
  • To speed up the development of BEAM-101, Beam noted that it's working to amend the trial to "streamline and expedite patient enrollment and trial conduct based on the recent regulatory landscape in SCD trials." 
  • As the company explained, patients enrolled in the trial would first have their stem cells retrieved — the cells will be edited ex vivo to make BEAM-101 — receive pre-treatment conditioning before receiving the cells back in a transplant. Only after the first patient's cells are engrafted can investigators begin treating the second patient.
  • Hence, the company wants to give itself more time to work on an improved conditioning regimen with lower toxicity.
  • In the Q3 report, Beam also offered an update on BEAM-201, its base-edited CAR-T therapy, which was placed on clinical hold. Beam has submitted its response to the FDA and will provide an update on the next steps.
  • Cash, cash equivalents, and marketable securities were $1.1 billion.
  • Price Action: BEAM shares are down 10.32% at $38.38 on the last check Monday.
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