Analysts Consider CRISPR Therapeutics' Blood Disorder Trial Data Encouraging, Impressive

  • CRISPR Therapeutics CRSP presented results from its ongoing Phase 1 COBALT-LYM CTX130 (exa-cel) trial, an allogeneic CAR-T cell therapy targeting CD70 for solid tumors and certain hematologic malignancies.
  • CTX130 was well tolerated across all dose levels. There were no cases of Graft versus Host Disease (GvHD), no dose-limiting toxicities (DLTs), and no instances of tumor lysis syndrome (TLS).
  • Needham notes that 95% of beta-thalassemia (TDT) achieved sustained transfusion independence (TI), with the remaining two showing a significantly reduced transfusion burden.
  • An overall response rate (ORR) of 70% with a 30% complete response (CR) rate was reported in patients at dose levels≥3 (300M and 900M cells). 
  • Chardan has updated its model, increasing POS for exa-cel in TDT and SCD to 75% from 65% and 60%, respectively.
  • Needham believes the impressive and durable efficacy observed with CTX001 makes it a competitive therapeutic even if Bluebird Bio Inc's BLUE beti-cel enters the market first. 
  • Read Next: FDA Advisory Committee Backs Bluebird Bio's Blood Disorder Gene Therapy.
  • HC Wainwright says beti-cels' efficacy and safety profile are comparable to CTX001, although the modalities are different.
  • Chardan believes exa-cel is positioned to overtake beti-cel (given its lead in SCD and as BLUE has pulled out of Europe), betting on CRSP's partner Vertex Pharmaceuticals Inc's VRTX global infrastructure.
  • Price Action: CRSP shares are down 13.5% at $55.15 during the market session on the last check Monday.

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