Adaptimmune Announces U.S. FDA Breakthrough Therapy Designation Granted to Letetresgene Autoleucel (lete-cel) for Treatment of Myxoid/Round Cell Liposarcoma (MRCLS)

Rhea-AI Summary

Adaptimmune Therapeutics (NASDAQ: ADAP) announced that its therapy letetresgene autoleucel (lete-cel) has received FDA breakthrough therapy designation for treating myxoid/round cell liposarcoma (MRCLS) in specific patient conditions. The designation was based on Phase II IGNYTE-ESO trial results, where 42% of patients (27/64) with synovial sarcoma or MRCLS showed responses, including 6 complete and 21 partial responses.

The median duration of response was 12.2 months overall, with 18.3 months for synovial sarcoma and 12.2 months for MRCLS patients. Safety findings showed manageable toxicities, with common adverse events including cytopenias, cytokine release syndrome, and rash.

The breakthrough designation will expedite lete-cel's development and review processes, with Adaptimmune planning to initiate a rolling Biologics License Application later in 2025. The company aims to bring lete-cel to market in 2026 for both synovial sarcoma and MRCLS treatment.

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The Company will present at the Annual J.P. Morgan Healthcare Conference, providing business updates on its sarcoma franchise and other cell therapy pipeline assets

Adaptimmune Allo-T program to be featured at the Biotech ShowCase^(TM) and the
Wuxi Global Forum 2025

Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, today announced that letetresgene autoleucel (lete-cel), has been granted breakthrough therapy designation by the U.S. FDA for the treatment of patients with unresectable or metastatic myxoid/round cell liposarcoma (MRCLS) who have received prior anthracycline-based chemotherapy, are positive for HLA-A*02:01, HLA-A*02:05, or
HLA-A*02:06, and whose tumor expresses the NY-ESO-1 antigen.

More details about the Company's sarcoma franchise, including the lete-cel clinical program and launch progress for TECELRA^® (afamitresgene autoleucel), the Company's first commercial product and the first FDA approved engineered cell therapy for a solid tumor, will be provided during the Company's presentation at the Annual J.P. Morgan Healthcare Conference, taking place in San Francisco, California, on Tuesday, January 14th, 2025, 4:30-5:10 PM PST (Webcast access here).

Breakthrough therapy designation for lete-cel in MRCLS was based on the results in this indication from the Phase II IGNYTE-ESO trial. The Company previously received breakthrough therapy designation for lete-cel for the treatment of synovial sarcoma in 2016. In the Phase II analysis, 27/64 (42%) people with synovial sarcoma or MRCLS had RECISTv1.1 responses by independent review, with 6 complete responses and 21 partial responses. The response rate was 14/34 (41%) for people with synovial sarcoma and 13/30 (43%) for people with MRCLS. The median duration of response (DoR) was 12.2 months (95% CI 6.8, 19.5). In synovial sarcoma, the median duration of response was 18.3 months (95% CI 3.3, -). In MRCLS, the median duration of response was 12.2 months (95%, CI 5.3, -). Safety findings were consistent with the known profile of lete-cel from previous data. All patients experienced treatment-emergent adverse events: cytopenias, cytokine release syndrome (CRS) and rash were the most common adverse events. Overall, toxicities were manageable, and consistent with an acceptable benefit to risk profile. Data from this trial were presented at the Connective Tissue Oncology Society (CTOS) 2024 annual meeting (Link to press release HERE; Presentation HERE).

Adrian Rawcliffe, Adaptimmune's Chief Executive Office: "This designation by the FDA highlights the potential of lete-cel to address a critical need for new treatment options for patients with MRCLS. This is another important milestone in building out our sarcoma franchise, as we aim to bring lete-cel to market in 2026 for the treatment of synovial sarcoma and MRCLS. We look forward to initiating a rolling Biologics License Application for lete-cel later this year for the treatment of both sarcoma indications."

The breakthrough therapy designation is designed to expedite drug development and review processes. The criteria for this designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. With the designation, lete-cel will receive incentives, such as additional interaction and guidance from the FDA, the potential for a rolling submission, and potential priority review of the biologics license application, as well as other opportunities to expedite the development.

In addition to the Company's presentation at J.P. Morgan, Adaptimmune will present the Company's Allo-T program as a spin-out opportunity at the Biotech ShowCase™ and the Wuxi Global Forum 2025 Investor Roundtable. Details can be found below:

Biotech Showcase™ | Investor conference | Co-produced by Demy-Colton and EBD Group
Presentation at 2 p.m. PST on Tuesday, January 14^th, Yosemite C, Hilton Hotel, Union Square.
WuXi Global Forum 2025 - WXPress: for WuXi news and R&D insights
Round Table Presentation at 5 p.m. PST on Tuesday, January 14^th, Tower 3, Hilton Hotel, Union Square

About Adaptimmune
Adaptimmune is a fully integrated cell therapy company working to redefine how cancer is treated. With its unique engineered T cell receptor (TCR) platform, the Company is developing personalized medicines designed to target and destroy difficult-to-treat solid tumor cancers and to radically improve the patient's cancer treatment experience.

Forward-Looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 (PSLRA). These forward-looking statements involve certain risks and uncertainties. Such risks and uncertainties could cause our actual results to differ materially from those indicated by such forward-looking statements, and include, without limitation: the success, cost and timing of our product development activities and clinical trials and our ability to successfully advance our TCR therapeutic candidates through the regulatory and commercialization processes. For a further description of the risks and uncertainties that could cause our actual results to differ materially from those expressed in these forward-looking statements, as well as risks relating to our business in general, we refer you to our Annual Report on Form 10-K filed with the Securities and Exchange Commission for the year ended 31 December, 2023, our Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and other filings with the Securities and Exchange Commission. The forward-looking statements contained in this press release speak only as of the date the statements were made and we do not undertake any obligation to update such forward-looking statements to reflect subsequent events or circumstances.

Investor Relations
Juli P. Miller, Ph.D. - VP, Corporate Affairs and Investor Relations
T : +1 215 825 9310
M : +1 215 460 8920
Juli.Miller@adaptimmune.com

Media Relations
Dana Lynch, Senior Director of Corporate Communications
M: +1 267 990 1217
Dana.Lynch@adaptimmune.com

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/236918

FAQ

What response rates did lete-cel show in the Phase II IGNYTE-ESO trial for MRCLS patients?

In the Phase II IGNYTE-ESO trial, MRCLS patients showed a 43% response rate (13/30 patients), with a median duration of response of 12.2 months.

When does Adaptimmune (ADAP) plan to launch lete-cel for sarcoma treatment?

Adaptimmune plans to bring lete-cel to market in 2026 for the treatment of both synovial sarcoma and MRCLS.

What are the main side effects reported in the lete-cel clinical trials?

The main adverse events reported were cytopenias, cytokine release syndrome (CRS), and rash. The toxicities were described as manageable with an acceptable benefit-to-risk profile.

What benefits does the FDA breakthrough therapy designation provide for lete-cel?

The designation provides additional FDA interaction and guidance, potential for rolling submission, possible priority review of the biologics license application, and other opportunities to expedite development.

What are the patient eligibility criteria for lete-cel treatment in MRCLS?

Eligible patients must have unresectable or metastatic MRCLS, received prior anthracycline-based chemotherapy, be positive for HLA-A*02:01, HLA-A*02:05, or HLA-A*02:06, and their tumor must express the NY-ESO-1 antigen.