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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