SHANGHAI, June 15,
2024 /PRNewswire/ -- CARsgen Therapeutics Holdings
Limited (Stock Code: 2171.HK), a company focused on innovative CAR
T-cell therapies for the treatment of hematologic malignancies and
solid tumors, announces that the initial results from the ongoing
first-in-human study of CT071 have been presented at the 29th
Annual Congress of the European Hematology Association (EHA).
The preliminary results of Phase I of CT071 (NCT05838131) were
presented as a poster at the 29th EHA Annual Congress on
June 14, 18:00
- 19:00 CEST, which was titled "First-in-human study of
GPRC5D-targeted CAR T cells (CT071) with an accelerated
manufacturing process in patients with relapsed/ refractory
multiple myeloma (RRMM)".[1]
"Multiple myeloma is a common yet incurable hematologic
malignancy with high unmet need. Despite the numerous recent
advances, most patients relapse and become refractory to available
therapies and therefore, novel therapies are needed. GPRC5D, a
protein highly expressed on the surface of malignant plasma cells
with limited expression on normal tissues, represents a promising
target for treating multiple myeloma. CT071 is a fully human
GPRC5D-targeting autologous second-generation CAR T-cell product
manufactured using our expedited CARcelerateTM platform
that shortens the manufacturing time to around 30 hours, supporting
a shorter vein-to-vein time and younger T cells. The preliminary
results of the ongoing study presented at EHA shows that CT071 has
the potential to be the best-in-class GPRC5D targeting CAR-T
therapy," said Raffaele Baffa, M.D.,
Ph.D., Chief Medical Officer of CARsgen Therapeutics. "We are
excited about advancing CT071 and look forward to sharing future
updates with the medical community."
As of February 28, 2024, 10
patients were dosed with CT071—7 patients at 1.0×105
cells/kg and 3 patients at 3.0×105 cells/kg. Among them,
80% had high-risk cytogenetics, 30% had one or more extramedullary
plasmacytomas (EMD), and 40% were at R-ISS stage III. This was a
heavily pre-treated population with a median of 5 prior lines of
therapy, including 90% double-class refractory, 70% triple-class
refractory, 40% penta-drug refractory, 50% having received
autologous stem cell transplantation, and 20% had previously been
treated with BCMA/CD19 dual-targeting CAR T cells. None of the
patients on the study required bridging therapy due to rapid
manufacturing turnaround.
The median follow-up at the time of data cut-off was 4.07 months
(range: 2.8-7.4). There were no Grade 3 or higher cytokine release
syndrome (CRS) events. No immune effector cell-associated
neurotoxicity syndrome (ICANS) was observed. No adverse events of
special interest or dose limiting toxicity (DLT) occurred. Four
patients experienced treatment-related SAE, including pneumonia
(n=1), decreased appetite (n=1) and thrombocytopenia (n=2), and all
recovered.
The overall response rate was 90%, including 5 patients (50%)
with stringent complete response (sCR), 2 patients (20%) with very
good partial response (VGPR), and 2 patients (20%) with partial
response (PR). All the 9 patients with evaluable MRD assessment at
Week 4 achieved MRD negativity (10-6 threshold) ),
including all 5 patients with
sCR/CR. The pharmacokinetic analysis demonstrated
robust cell expansion and persistence, with median Tmax
of 14 days (range: 12-28) and median Cmax of 32280.5
copies/μg gDNA (range: 8372-106060).
About CT071
CT071 is a CAR T-cell therapy candidate
developed utilizing CARsgen's proprietary CARcelerateTM
platform targeting GPRC5D for the treatment of R/R MM or
relapsed/refractory plasma cell leukemia ("R/R PCL"). An
investigator-initiated trial (NCT05838131) is ongoing in
China to evaluate the safety and
efficacy of CT071 for the treatment of R/R MM or R/R PCL. Another
investigator-initiated trial (NCT06407947) is ongoing in
China for the treatment of newly
diagnosed multiple myeloma (NDMM).
About CARsgen Therapeutics Holdings Limited
CARsgen is
a biopharmaceutical company with operations in China and the U.S. and is focused on
innovative CAR T-cell therapies for the treatment of hematologic
malignancies and solid tumors. CARsgen has established a
comprehensive CAR T-cell research and development platform,
encompassing target discovery, innovative CAR T-cell development,
clinical trials, and commercial-scale production. CARsgen has
internally developed novel technologies and a product pipeline with
global rights to address major challenges of CAR T-cell therapies,
such as improving the safety profile, enhancing the efficacy in
treating solid tumors, and reducing treatment costs. CARsgen's
mission is to become a global biopharmaceutical leader that brings
innovative and differentiated cell therapies to cancer patients
worldwide and makes cancer curable.
Forward-looking Statements
All statements in this
press release that are not historical fact or that do not relate to
present facts or current conditions are forward-looking statements.
Such forward-looking statements express the Group's current views,
projections, beliefs and expectations with respect to future events
as of the date of this press release. Such forward-looking
statements are based on a number of assumptions and factors beyond
the Group's control. As a result, they are subject to significant
risks and uncertainties, and actual events or results may differ
materially from these forward-looking statements and the
forward-looking events discussed in this press release might not
occur. Such risks and uncertainties include, but are not limited
to, those detailed under the heading "Principal Risks and
Uncertainties" in our most recent annual report and interim report
and other announcements and reports made available on our corporate
website, https://www.carsgen.com. No representation or warranty is
given as to the achievement or reasonableness of, and no reliance
should be placed on, any projections, targets, estimates or
forecasts contained in this press release.
Reference
[1] J Du, et al. EHA 2024. 2024 Jun; Poster
P941
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