CRISPR Therapeutics (NASDAQ:CRSP) has released encouraging top-line results from its ongoing Phase 1 CARBON study. The study evaluates the efficacy and safety of its wholly-owned allogeneic CAR-T cell therapy, CTX110, that targets CD19+ B-cell malignancies.
CTX110 demonstrates the potential of treating hematological malignancies
Samarth Kulkarni, the CEO of CRISPR Therapeutics, stated that they are delighted with the data, which shows allogeneic therapies’ potential as a treatment option to hematological malignancies. According to Kulkarni, they expect CRISPR-edited allogeneic CAR-T cell therapy to leapfrog autologous CART-T therapy and also offer more benefit to a broader patient population. Kulkarni added that the company has continued enrolling patients in the trial, and they look forward to providing more data readouts for the program in 2021. CRISPR therapeutics also expect to release data readouts for its other allogeneic CAR-T programs CTX130™ and CTX120™ by next year.
Professor Joseph McGuick, who is the division director of Haematological Malignancies and Cellular Therapeutics at the University of Kansas Medical center, is the investigator in the Phase 1 CARBON study. McGuick said that preliminary data from the CTX110 study has demonstrates enhanced dose-dependent efficacy and response rates compared to previous autologous CAR-T trials. Also, CTX110 had a favorable safety profile that will make CAR-T therapies more accessible to patients. Although longer follow-up is necessary, McGuick says that this preliminary data support the potential of CTX110 being an effective off-the-shelf CAR-T for patients having refractory or relapsed B-cell malignancies.
Phase 1 CARBON study enrolled 12 patients
The CARBON trial is a multicentre, open-label study evaluating the efficacy and safety of CTX110 in treating adult patients with refractory or relapsed non-Hodgkin lymphoma that have received two previous therapy lines. Around 12 patients had been enrolled as of September 28, 2020, data cutoff and infused with CTX110. Patients received CTX110 after three days of lymphodepletion using cyclophosphamide and fludarabine. The primary endpoints of the study include overall response rate and safety measured by dose-limiting toxicities incidence. The secondary endpoints include progression-free survival, duration of response, and overall survival.