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CARGO Therapeutics Announces Phase 1 Clinical Study of Firi-cel CAR T-Cell Therapy Published in The Lancet

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CARGO Therapeutics announced that data from their Phase 1 clinical study of Firi-cel CAR T-cell therapy has been published in The Lancet. The study focused on patients with relapsed or refractory large B-cell lymphoma (LBCL) who did not respond to CD19 CAR T-cell therapy. Results showed a 68% overall response rate (ORR) and a 53% complete response (CR) rate, with a median follow-up of 23.3 months.

Notably, 36% of patients previously refractory to all therapies achieved CR. The selected dose for the Phase 2 study, DL1, had a 66% ORR and a 52% CR rate, showing an estimated one- and two-year survival rate of 57% and 52%, respectively. The therapy showed no severe cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome.

These results underscore the promising efficacy and safety profile of firi-cel, which has received Breakthrough Therapy Designation from the FDA. The ongoing Phase 2 study, FIRCE-1, is on track for interim analysis in the first half of 2025.

Positive
  • 68% overall response rate (ORR) and 53% complete response (CR) rate in Phase 1 study.
  • 36% of refractory patients achieved complete response (CR).
  • No episodes of severe cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS).
  • Breakthrough Therapy Designation from the FDA for firi-cel.
Negative
  • None.

The publication of Phase 1 clinical study results in The Lancet of firicabtagene autoleucel (firi-cel) represents a significant milestone in the treatment of large B-cell lymphoma (LBCL). As a CD22-directed CAR T-cell therapy, firi-cel addresses a critical unmet need for patients whose disease is relapsed or refractory following CD19 CAR T-cell therapy.

For context, CD22 and CD19 are proteins expressed on the surface of B-cells. Targeting CD22 can be particularly valuable as an alternative therapeutic approach when CD19-targeted treatments fail. The positive overall response rate (ORR) of 68% and complete response (CR) rate of 53% in heavily pretreated patients is promising. Notably, durability of response is a important metric in oncology and the ongoing two-year survival rate of 52% for the selected dose level (DL1) indicates substantial long-term benefits.

Moreover, the Breakthrough Therapy Designation granted by the FDA underscores the therapy’s potential to be a game-changer for patients with limited treatment options. Importantly, the therapy’s safety profile is commendable, with no grade 3 or higher adverse events, aligning it well with the need for effective yet tolerable cancer treatments.

In the broad landscape of oncology, new therapies that provide durable responses in hard-to-treat populations are highly valued. The data presented certainly warrants close attention from both the medical community and investors, as successful Phase 2 and subsequent trials could establish firi-cel as a standard of care for R/R LBCL patients.

The data from this Phase 1 study showcases CARGO Therapeutics' innovative capabilities in the biotech sector. The strong response rates and the durability of firi-cel position the company favorably within the CAR T-cell therapy market, which is currently dominated by CD19-directed therapies. For retail investors, it is important to recognize the strategic implications.

Firstly, the Breakthrough Therapy Designation from the FDA is a valuable asset that can accelerate the regulatory pathway, potentially leading to earlier market entry and sales. This designation often translates into increased investor confidence and potential upward movement in stock prices. However, it's vital to note that the success of the pivotal Phase 2 study will be a key determinant of future financial performance.

Secondly, the progress toward the Phase 2 FIRCE-1 study reflects well on CARGO’s execution capabilities. Successful completion and positive interim results expected in the first half of 2025 could be significant catalysts. It’s also worth noting that the company's ability to maintain a robust safety profile while demonstrating efficacy speaks volumes about its research and development strengths.

While the publication in The Lancet provides a strong validation, investors should also be mindful of the competitive landscape and the inherent risks in biotech investments, such as regulatory hurdles and potential therapy adoption rates. It’s wise to keep an eye on upcoming updates to gauge the company’s ongoing momentum and strategic positioning.

  • Ongoing Phase 1 study informed dose selection for CARGO’s currently enrolling, potentially pivotal Phase 2 clinical study, FIRCE-1 of firicabtagene autoleucel (firi-cel)1, in patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL)
  • Notable patient subset analyses show durability of Complete Response (CR), underscoring promising efficacy in a high-risk, heavily pretreated population with advanced disease
  • Most recent, longer-term follow-up data for Stanford Medicine’s Phase 1 presented at the 2024 European Hematology Association (EHA) Congress, continued to show promising efficacy, durability and safety profile of firi-cel

SAN CARLOS, Calif., July 10, 2024 (GLOBE NEWSWIRE) -- CARGO Therapeutics, Inc. (Nasdaq: CRGX), a clinical-stage biotechnology company positioned to advance next generation, potentially curative cell therapies for cancer patients, today announced that The Lancet has published favorable data from a Phase 1, single-center clinical study (NCT04088890) by Stanford Medicine (Stanford), evaluating firi-cel, a CD22 CAR T-cell therapy CARGO in-licensed for patients with LBCL whose disease is R/R to CD19 CAR T-cell therapy. The clinical data presented in the publication is as of May 22, 2023.

“This first peer-reviewed publication of Stanford’s Phase 1 study data in The Lancet further validates firi-cel’s clinically transformative potential in addressing the high unmet needs of LBCL patients. Currently, approximately 60% of these patients treated with a CD19 CAR T-cell therapy experience disease relapse or progression and have a median survival of less than six months,” said Gina Chapman, President and Chief Executive Officer of CARGO Therapeutics. “We congratulate Stanford and the study’s investigators on these impactful findings, demonstrating the achievement of favorable complete response rate and long-term durability of response in this heavily pretreated, advanced-disease patient population, many of whom were refractory to all prior lines of therapy. We believe that the patient subset analyses further support the study design of our potentially pivotal Phase 2 program, which continues to progress as planned and is on track for interim analysis in first half 2025.”

The Lancet publication titled “CD22-Directed CAR T-Cell Therapy for Large B-Cell Lymphomas Progressing After CD19-Directed CAR T-cell Therapy: A dose-finding Phase 1 study,” demonstrated for all patients treated (n=38), an Overall Response Rate (ORR) and CR rate of 68% and 53%, respectively, at a median follow-up of 23.3 months. Notably, there were no significant differences observed between ORR and CR across subgroups, and of the patients with a history of refractory disease to all prior therapies, 36% (4 of 11) achieved CR.

For Dose Level 1 (DL1), the selected dose for the potentially pivotal Phase 2 study FIRCE-1, the ORR and CR rates were 66% and 52%, respectively. The estimated one- and two-year survival at DL1 was 57% and 52%, respectively. Further for DL1, firi-cel was generally well-tolerated with no episodes of >grade 3 cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and immune effector cell-associated HLH-like syndrome (IEC-HS).

Key baseline and clinical characteristics of the Phase 1 patient population are outlined, including:

  • Median age of 65 years; 50% >65 years of age
  • Median of 4 lines of priory therapies
  • 26 (68%) advanced stage III or IV 
  • 32 (84%) had elevated pre-treatment lactate dehydrogenase (LDH), or high tumor burden 
  • 11 (29%) had history of refractory disease to all prior therapies
  • 37 (97%) had relapsed after CAR T-cell therapy

As reported by CARGO on May 14, 2024, a subsequent abstract and poster were shared at the 2024 European Hematology Association (EHA) Congress reflecting a data cutoff as of February 1, 2024, with a median follow up of 31.4 months. ORR and CR rates for all patients treated were 68% and 53%, respectively. New data for DL1 (n=29) at a median follow-up of 29.8 months include median overall survival (mOS) of 25.7 months (95% CI: 9.2, NE) and estimated 2-year survival remains at 52%. The median PFS, duration of response, and OS have not been reached for patients who achieved a CR. Of the 20 patients achieving CR, there have been no additional patient relapses since the last data cut in November of 2023. Further, no grade 3 or higher cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) events occurred at DL1.

Stanford received Breakthrough Therapy Designation for firi-cel from the FDA for the treatment of adult patients with LBCL whose disease is R/R after CD19-directed CAR T-cell therapy.

The Lancet publication can be viewed by clicking this link.

About Large B-Cell Lymphoma (LBCL)
Lymphoma that affects B lymphocytes are called B-cell lymphomas. B-cells make antibodies to fight infections and are an important part of the human immune system. B-cell lymphomas account for approximately 85% of non-Hodgkin lymphomas (NHL) in the United States. LBCL is an aggressive (fast-growing) lymphoma increasing in prevalence that occurs most commonly in people over the age of 60, though it can occur in childhood.

About Firi-cel

Firicabtagene autoleucel (firi-cel) is CARGO's investigational cell therapy composed of autologous T-cells transduced with a lentiviral vector (m971-BBZ) expressing a CD22-targeting chimeric antigen receptor or CAR. CD22 is a transmembrane protein expressed on normal B-cells and B-cell malignancies. Results of a fully enrolled, ongoing Phase 1 clinical trial conducted by Stanford Medicine (Stanford) in adults demonstrate the safety and antitumor activity of CD22 CAR T-cell therapy in patients with LBCL whose disease is relapsed or refractory (R/R) to CD19 CAR T-cell therapy. Based on initial Phase 1 data, Stanford was granted Breakthrough Therapy Designation from the FDA for the treatment of adult LBCL patients whose disease is R/R after CD19-directed CAR T-cell therapy. CARGO Therapeutics exclusively in-licensed the CD22 CAR T-cell technology from the National Cancer Institute to develop firi-cel. Its unique design is associated with efficacy in the clinic that has not been seen with other CD22 CAR Ts. CARGO believes that firi-cel has the potential to safely and effectively treat LBCL, including patients for whom prior CD19 CAR T-cell therapies have failed.

About CARGO Therapeutics

CARGO Therapeutics, Inc. is a clinical-stage biotechnology company positioned to advance next- generation, potentially curative cell therapies for cancer patients. CARGO’s programs, platform technologies, and manufacturing strategy are designed to directly address the limitations of approved cell therapies, including limited durability of effect, safety concerns and unreliable supply. CARGO is currently evaluating its lead program, firicabtagene autoleucel (firi-cel) (CRG-022), an autologous CD22 chimeric antigen receptor (CAR) T-cell therapy candidate, in a potentially pivotal Phase 2 clinical study in patients with large B-cell lymphoma (LBCL) whose disease relapsed or was refractory (R/R) to CD19 CAR T-cell therapy. CARGO also plans to evaluate firi-cel in patients at earlier stages of disease, including LBCL and other hematologic malignancies. Beyond its lead program, CARGO is leveraging its proprietary cell engineering platform technologies to develop a pipeline of programs that incorporate multiple transgene therapeutic “cargo” designed to enhance CAR T-cell persistence and trafficking to tumor lesions, as well as to help safeguard against tumor resistance and T-cell exhaustion. This includes the CRG-023 program, which incorporates a tri-specific CAR T with CD2 co-stimulation. CARGO’s founders are pioneers and world-class experts in CAR T-cell therapy, and its team has significant experience and success in developing, manufacturing, launching and commercializing oncology and cell therapy products. For more information, please visit the CARGO Therapeutics website at https://cargo-tx.com/.
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1 Firicabtagene autoleucel (firi-cel) is CARGO Therapeutics' autologous CD22 CAR T-cell product candidate. The underlying CAR of which CARGO exclusively in-licensed from the National Cancer Institute was the construct evaluated by Stanford Medicine in a Phase 1 clinical trial in patients with large B-cell lymphoma whose disease relapsed or was refractory to CD19 CAR T-cell therapy. CARGO’s firi-cel Investigational New Drug application included a comprehensive package in which CARGO performed and demonstrated analytical comparability of CRG-022 produced using the intended commercial process to the CRG-022 produced using the process used for the Stanford Phase 1 clinical trials. CARGO cannot assure that the FDA will agree with its claim of comparability and the sufficiency of the data to support it when it files its Biologics License Application.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “target,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: advancement of CARGO’s clinical and preclinical programs; the potential benefits of CARGO’s product candidates, including efficacy, durability and safety profile of firi-cel; and timing of data reports, including the release of interim data from the Company’s ongoing Phase 2 clinical trial of firi-cel. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the company’s ability to obtain necessary capital to fund its clinical programs; the early stages of clinical development of the company’s product candidates; the company’s ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company’s product candidates; the company’s reliance on third-party suppliers and manufacturers, including CROs; the outcomes of any future collaboration agreements; and the company’s ability to adequately maintain intellectual property rights for its product candidates. For a detailed discussion of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to CARGO’s business in general, please refer to the risk factors identified in the Company’s filings with the Securities and Exchange Commission (SEC), including but not limited to its Quarterly Report on Form 10-Q for the quarter ended March 31, 2024 filed on May 14, 2024. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. CARGO’s results for the quarter ended March 31, 2024 are not necessarily indicative of its operating results for any future periods.

Media Contact:
Kimberly Muscara
kimberly@redhousecomms.com

Investor Contact:
Jessica Serra
jserra@cargo-tx.com

Laurence Watts
laurence@newstreetir.com


FAQ

What were the results of the Phase 1 clinical study of Firi-cel CAR T-cell therapy?

The Phase 1 study showed a 68% overall response rate (ORR) and a 53% complete response (CR) rate with a median follow-up of 23.3 months.

What is the significance of the Phase 1 study results for firi-cel?

The results demonstrate promising efficacy and safety, which informed dose selection for the ongoing Phase 2 study and led to FDA Breakthrough Therapy Designation for firi-cel.

When is the interim analysis for the Phase 2 study of firi-cel expected?

The interim analysis for the Phase 2 study, FIRCE-1, is expected in the first half of 2025.

How did the DL1 dose perform in the Phase 1 study?

The DL1 dose showed a 66% overall response rate (ORR) and a 52% complete response (CR) rate, with an estimated one- and two-year survival rate of 57% and 52%, respectively.

What is the median follow-up for the Phase 1 study of firi-cel?

The median follow-up for the Phase 1 study was 23.3 months.

Did the Phase 1 study of firi-cel show any severe side effects?

No, the Phase 1 study showed no episodes of grade 3 or higher cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS).

CARGO Therapeutics, Inc.

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