GC012F

P1, N=18, Recruiting, Shanghai Changzheng Hospital | Not yet recruiting --> Recruiting

P1/2, N=110, Recruiting, Gracell Biotechnologies Ltd.

Patients received a median of 2 prior lines (range 2 - 3) of therapy including rituximab and anthracyclines. GC012F CAR-T cells were detected in the tumor biopsies, indicating the infiltration of CAR-T cells into the tumor lesions. Overall, based on the safety and efficacy results, CD19-BCMA dual targeting CAR-T product GC012F is a promising therapy for patients with refractory/ relapsed DLBCL.

Patients received a median of 2 prior lines (range 2 - 3) of therapy including rituximab and anthracyclines. This first-in-human trial of GC012F,a CD19-BCMA dual targeting CAR-T product, for thetreatment of r/r B-NHL showed a manageablesafety profileand promising clinical responses.The ORR was 100% at month 3 with 77.8% (7/9) achieving CR. GC012F CAR-Tcells were detectablein thetumor biopsies, indicating theinfiltration of CAR-T cells into thetumor lesions.Study with larger cohortand longer follow-up is ongoing. Keywords: CAR-T

The updated results showed GC012F continues to provide deep and durableresponses,and a very high MRD negativity ratein RRMM pts, including in pts refractory to anti-CD38, PIs and IMiDs. Given the promising results of thecurrent study, further clinical studies will beconducted to confirm theefficacy as well as safety of GC012F therapy for RRMM patients. Keywords: relapsed/refractory, CAR-T, Multiple myeloma

Pts received a median of 2 prior lines (range 2-3) of therapy including rituximab and anthracyclines. This first-in-human trial of GC012F for the treatment of r/r B-NHL showed a manageable safety profile and promising clinical responses. The ORR was 100% at M3 with 77.8% (7/9) achieving CR. GC012F CAR-T cells were detectable in the tumor biopsies, indicating the infiltration of CAR-T cells into the tumor lesions.

The updated results showed GC012F continues to provide deep and durable responses, and a very high MRD negativity ratein RRMM pts, including in pts refractory to anti-CD38, PIs and IMIDs. Based on these promising results of the study of GC012F for RRMM, further clinical studies will be conducted to confirm the efficacy of GC012F. Clinical trial information: NCT04236011; NCT04182581.

Twelve pts received 2 cycles induction therapy of bortezomib, lenalidomide and dexamethasone (VRd), and one patient received 1 cycle bortezomib, epirubicin, and dexamethasone (PAD) and 1 cycle VRd prior to the infusion. GC012F was administered as a single infusion at 3 doses levels (DL) of 1x105/kg, 2x105/kg, or 3x105/kg, after receiving a conditioning chemotherapy consisting of cyclophosphamide and fludarabine...Conclusion In this phase I study for transplant-eligible newly diagnosed high-risk MM, BCMA-CD19 dual FasTCAR-T GC012F showed a very favorable safety profile, high efficacy with 100% ORR and 100% MRD negativity. The promising preliminary results warrants further assessment of GC012F for TE NDMM with more patients and longer follow-up.

Conclusion BCMA-CD19 dual FasT CAR-T GC012F continues to provide deep and durable responses with a favorable safety profile in additional RRMM pts across all dose levels demonstrating a very high MRD negativity rate including in pts refractory to anti-CD38, PI and IMIDs. Based on these promising results GC012F is being studied in earlier lines of therapy as well as additional indications.

All three pts achieved CR by PET-CT at day 28 after GC012F infusion, and response is ongoing at 3-months follow-up in the two assessable patients. Conclusion The preliminary safety and tolerability of GC012F in r/r B-NHL at three different dose levels ranging from 3.7*10 4 CAR-T/kg-3*10 5 CAR-T/kg showed promising early and potent activity in r/r B-NHL pts with 100% CR rate at 1-month which was maintained in pts that were available to be assessed at the 3-month visit including r/r DLBCL pts and pts with bulky disease whose treatment is often challenging. Additional pts and longer follow up are needed to further evaluate these promising early results of GC012F in r/r B-NHL and to determine the RP2D for phase 2.

BCMA-CD19 dual FasT CAR-T GC012F continues to provide deep and durable responses with a favorable safety profile in additional RRMM pts across all dose levels demonstrating a very high MRD negativity rate including in pts refractory to anti-CD38, PI and IMIDs. GC012F is currently being studied in earlier lines of therapy as well as additional indications.

P1/2, N=20, Recruiting, Shanghai Changzheng Hospital | Not yet recruiting --> Recruiting

P1/2, N=20, Not yet recruiting, Shanghai Changzheng Hospital

At data cut off, the median time to follow up was 13.8 mths (6.1-16.4) – with the median duration of response not yet reached. Cytokine Release Syndrome (CRS) and ICANs were graded by ASBMTcriteria: grade 1-2 n=16 (84.2%), grade 3 n=2 (10.5%). Median duration of CRS was 4d (1-8d). No grade 4/5 CRS or Immunce effector cell-associated Neurotoxicity syndrome (ICANS) were observed. 2 deaths occurred on study and were assessed as not related to therapy – one reported previously; one patient was diagnosed with& Conclusion BCMA-CD19 dual FasT CAR-T GC012F showed early, deep and durable responses with a high ORR (94.7%; VGPR or better) including a high MRD negative sCR rate (DL3=100%, n=9) in high risk RRMM pts including those refractory to anti-CD38, PI and IMIDs with a favorable safety profile consistent with previous findings. BCMA-CD19 dual FasT CAR-T GC012F may present a new treatment approach for patients with RRMM including those with high-risk features refractory to standard of care.

BCMA-CD19 dual FasT CAR-T GC012F showed early, deep and durable responses with a high ORR (94.7% - VGPR and better) including a high MRD- sCR rate (DL3=100%, n=9) in high risk RRMM pts including those refractory to anti-CD38, PI and IMIDs with a favorable safety profile consistent with previous findings . BCMA-CD19 dual FasT CAR-T GC012F may present a new treatment approach for patients with RRMM including those with high-risk features refractory to standard of care.