Caribou Biosciences Reports CB-010 ANTLER Phase 1 Trial Progress

On December 12, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported new 12-month clinical data from cohort 1 in the ongoing ANTLER Phase 1 trial, which show long-term durability following a single infusion of CB-010 at the initial dose level 1 (40×106 CAR-T cells) (Filing, 8-K, Caribou Biosciences, DEC 12, 2022, View Source [SID1234625090]). Cohort 1 results show:

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•6 of 6 patients achieved a complete response (CR) as best response

•3 of 6 patients maintained a durable CR at 6 months

•2 of 6 patients maintain a long-term CR at the 12-month scan and remain on the trial

•18 months is the longest CR maintained to date in ANTLER, achieved by the first patient dosed with CB-010

•CB-010 was generally well tolerated with adverse events consistent with autologous or allogeneic anti-CD19 CAR-T cell therapies

Based on promising initial data, the U.S. Food and Drug Administration (FDA) granted CB-010 both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations. In addition, Caribou has observed an encouraging safety profile for CB-010 at dose level 2 (80×106 CAR-T cells) with no dose-limiting toxicities (DLTs) in the 3 patients treated and is currently enrolling patients at dose level 3 (120×106 CAR-T cells). Caribou expects to provide an ANTLER trial update in 2023.

"With next-generation CRISPR genome-editing technology, the promise of allogeneic cell therapies has advanced significantly, and the early results seen in the ANTLER trial to date are a reflection of

that potential," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "The long-term durability at dose level 1 is comparable to autologous cell therapies and we believe CB-010 has the potential to set a new therapeutic bar for what allogeneic anti-CD19 CAR-T cell therapies can achieve. We are further encouraged by receiving RMAT and Fast Track designations for CB-010 from the FDA, which is a testament to both the encouraging initial ANTLER data and the need for novel therapies for patients with relapsed or refractory B-NHL."

CB-010 is the first allogeneic anti-CD19 CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve the persistence of antitumor activity by limiting premature CAR-T cell exhaustion.

"Patients with relapsed or refractory B cell non-Hodgkin lymphoma are in need of treatments that are immediately available and do not require burdensome or ineffective bridging therapies," said Susan O’Brien, M.D., professor of medicine, Chao Family Comprehensive Cancer Center at University of California, Irvine, CA, and presenting investigator on the ANTLER clinical trial. "The early results from the ANTLER trial are promising and I look forward to enrolling additional patients in this trial to learn more about the potential of CB-010 as an off-the-shelf treatment option for patients with aggressive B-NHL who have a high unmet medical need."

ANTLER is a Phase 1, open-label, multicenter clinical trial (NCT04637763) evaluating the safety and efficacy of the company’s lead allogeneic cell therapy, CB-010, in patients with r/r B-NHL. The trial includes Part A, a 3+3 dose escalation phase designed to evaluate safety of CB-010 at multiple dose levels and establish the recommended Phase 2 dose, and Part B, a dose expansion phase with the primary objective to determine tumor response after a single dose of CB-010. As permitted by the protocol, backfilling patients has begun at doses deemed well tolerated to increase the understanding of CB-010’s safety profile and antitumor activity and provide additional data for establishing a recommended Phase 2 dose.

ANTLER Trial-in-Progress Poster at ASH (Free ASH Whitepaper) 2022
Today at the 64th Annual ASH (Free ASH Whitepaper) meeting, a trial-in-progress poster is being presented to provide details of the design and objectives of the ANTLER Phase 1 trial for CB-010 in r/r B-NHL. Details of the poster presentation are as follows:

Title: A First-in-Human Phase 1, Multicenter, Open-Label Study of CB-010, a Next-Generation CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy with a PD-1 Knockout, in Patients with Relapsed/Refractory B Cell Non-Hodgkin Lymphoma (ANTLER Study)
Presenter: Susan O’Brien, M.D., professor of medicine, Chao Family Comprehensive Cancer Center at University of California, Irvine, CA
Session Name: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster III
Session Date: Monday, December 12, 2022
Presentation Time: 6:00 pm – 8:00 pm CST
Location: Ernest N. Morial Convention Center, Hall D
Abstract number: 4257

The poster presentation will be available for registered attendees on the ASH (Free ASH Whitepaper) website and on Caribou’s website under Scientific Publications (www.cariboubio.com/technology/#pubs) on Monday, December 12, 2022 at 9:00 am CST.

About CB-010
CB-010 is the lead product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 trial. CB-010 is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to boost the persistence of antitumor activity. CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knock out. CB-010 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations. Additional information on the ANTLER trial can be found at www.clinicaltrials.gov using identifier NCT04637763.