Letetresgene autoleucel

Approved for advanced MAGE-A4+synovial sarcoma in adults with certain HLA types who have received prior chemotherapy TECELRA is the first new treatment option for people with synovial sarcoma in more than a decade Adaptimmune to hold webcast at https://www.gowebcasting.com/13428 on August 2, at 8:00 a.m. EDT PHILADELPHIA, PA, USA & OXFORD, UK I August 01, 2024 I Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, today announced U.S. Food and Drug Administration (FDA) accelerated approval of TECELRA ® (afamitresgene autoleucel) for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. TECELRA is the first engineered cell therapy for a solid tumor cancer approved in the U.S., and the first new therapy option in more than a decade for synovial sarcoma, a rare, soft tissue cancer that most commonly impacts young adults. Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer: “The approval of TECELRA is a momentous step in Adaptimmune’s journey to redefine the way cancer is treated and the culmination of a decade of groundbreaking R&D. I want to thank the patients, caregivers, investigators, and clinical teams as well as everyone at Adaptimmune and our partners who made possible this watershed moment for cell therapy and for people with synovial sarcoma. We are committed to advancing our robust clinical pipeline to serve more patients in need and plan to progress lete-cel, the next late-stage investigational treatment in our sarcoma franchise, with a rolling BLA submission to the FDA next year.” The approval of TECELRA was based on results of the SPEARHEAD-1 (Cohort 1) trial, which included 44 patients. The major efficacy outcome was overall response rate (ORR) determined by independent review and supported by duration of response. TECELRA treatment resulted in an ORR of 43% with a complete response rate of 4.5%. The median duration of response was 6 months (95% CI: 4.6, not reached). Among patients who were responsive to the treatment, 39% had a duration of response of 12 months or longer.* Data from the pivotal SPEARHEAD-1 trial were previously published in The Lancet earlier this year. With this approval, Adaptimmune is positioned to make a significant impact on the synovial sarcoma community. HCPs can begin testing patients, Adaptimmune systems are ready to take TECELRA orders, and an integrated support program, AdaptimmuneAssist, is available to enable a seamless and personalized experience through the treatment journey. Adaptimmune plans to have at least six to ten authorized treatment centers (ATCs) up and running this year and to onboard approximately 30 treatment centers within the first two years. These ATCs are recognized leaders in sarcoma research and treatment. Brandi Felser, Chief Executive Officer, Sarcoma Foundation of America: “For decades, therapeutic options for people diagnosed with synovial sarcoma have been limited. With a current five-year survival rate as low as 36%, and for those with metastatic disease at diagnosis, as low as 20%, it is long past time that synovial sarcoma patients have expanded treatment options. Since one third of patients are diagnosed under age 30, improved outcomes can have a tremendous impact. Today, there is a renewed sense of hope for this patient community.” Sandra D’Angelo, MD, Sarcoma Medical Oncologist and Cell Therapist, Memorial Sloan Kettering Cancer Center; SPEARHEAD Trial Principal Investigator: “TECELRA (afami-cel), which uses each patient’s own immune cells to recognize and attack their cancer cells in a one-time infusion treatment, is significantly different than the current standards of care for advanced synovial sarcoma. This approval represents a much-needed new option for people diagnosed with this sarcoma and an important milestone for the use of cell therapies in solid tumor cancers.” TECELRA is contraindicated in adults who are heterozygous or homozygous for HLA-A*02:05P. TECELRA can cause serious side effects, including cytokine release syndrome (CRS), immune effector cell–associated neurotoxicity syndrome (ICANS), prolonged severe cytopenia, infections, secondary malignancies, and hypersensitivity reactions. Most common adverse reactions (incidence ≥20%) were CRS, nausea, vomiting, fatigue, infections, pyrexia, constipation, dyspnea, abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia, back pain, hypotension, diarrhea, edema, low white blood cells, low red blood cells and low platelets. Please see Important Safety Information, including Boxed Warning below. Biomarker tests for human leukocyte antigens (HLA) type and melanoma-associated antigen A4 (MAGE-A4) tumor expression are required prior to treatment with TECELRA. Adaptimmune has partnered with Agilent Technologies for the development, manufacturing, and supply of a companion diagnostic for the MAGE-A4 biomarker, MAGE-A4 IHC 1F9 pharmDx, which also received approval today from the U.S. FDA and is now available. Additionally, the company partnered with Thermo Fisher Scientific to expand the labeling of Thermo Fisher’s companion diagnostic product SeCore TM CDx HLA-A Locus Sequencing System to include TECELRA and to aid in the identification of HLA-A*02:01, A*02:02, A*02:03, and A*02:06-positive patients with synovial sarcoma. For more information about TECELRA visit www.adaptimmune.com . Conference Call Details The Company will host a live webcast to provide additional details tomorrow, August 2, 8:00 a.m. EDT. A live webcast of the conference call and replay can be accessed here: https://www.gowebcasting.com/13428 . Call in information is as follows: 1-844-763-8274 (TOLL FREE US or Canada) or +1-647-484-8814 (International) . About Synovial Sarcoma There are more than 50 different types of soft tissue sarcomas which are categorized by tumors that appear in fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues. 1 Synovial sarcoma accounts for approximately 5 to 10% of all soft tissue sarcomas (there are approximately 13,400 new soft tissue cases in the U.S. each year). 1,2 One third of patients with synovial sarcoma will be diagnosed under the age of 30. 2 The five-year survival rate for people with metastatic disease is approximately 20% and most people undergoing standard of care treatment for advanced disease experience recurrence and go through multiple lines of therapy, often exhausting all options. 1,3 About TECELRA TECELRA® (afamitresgene autoleucel) is a melanoma-associated antigen A4 (MAGE-A4)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices. This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. IMPORTANT SAFETY INFORMATION CONTRAINDICATION: DO NOT use TECELRA in adults who are heterozygous or homozygous for HLA-A*02:05P. BOXED WARNING: Cytokine release syndrome (CRS), which may be severe or life-threatening, occurred in patients receiving TECELRA. At the first sign of CRS, immediately evaluate patient for hospitalization and institute treatment with supportive care. Ensure that healthcare providers administering TECELRA have immediate access to medications and resuscitative equipment to manage CRS. CRS Immune Effector Cell–associated Neurotoxicity Syndrome (ICANS) Monitoring for CRS and ICANS During and Following TECELRA Infusion Prolonged Severe Cytopenia Infections Secondary Malignancies Hypersensitivity Reactions Potential for HIV Nucleic Acid Test False-Positive Results Adverse Reactions Please see full Prescribing Information , including Boxed Warning and Medication Guide . About AdaptimmuneAssist An integrated support program, AdaptimmuneAssist is now available to provide access support for patients, their caregivers, and healthcare providers throughout the TECELRA treatment journey. AdaptimmuneAssist includes connection with a Treatment Navigator, travel and financial support programs for eligible patients, and access to the AdaptimmuneAssist Order Portal (for healthcare providers only). For more information, physicians and patients may call 1-855-246-9232. 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 improve the patient’s cancer treatment experience. Dr. D’Angelo has financial interests related to Adaptimmune. 1. “What is a Soft Tissue Sarcoma?” American Cancer Society. https://www.cancer.org/cancer/types/soft-tissue-sarcoma/about/soft-tissue-sarcoma.html . Accessed June 24, 2024. 2. “Soft Tissue Sarcoma.” Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/21732-soft-tissue-sarcoma . Accessed June 24, 2024. 3. Jami SA, Mobarak SA, Jiandang S, et al. Clinical and strategic outcomes of metastatic synovial sarcoma on limb. Int J Health Sci(Qassim) . 2020;14:38–43. *Per Kaplan-Meier method SOURCE: Adaptimmune

Tecelra is genetically engineered to target melanoma-associated antigen A4 (MAGE-A4). Image: Shutterstock/Christoph Burgstedt Oxford-based cell therapy company Adaptimmune Therapeutics has received accelerated approval from the US Food and Drug Administration (FDA) for Tecelra (afamitresgene autoleucel) for treatment of synovial sarcoma. Tecelra is the first engineered cell therapy for solid tumours approved in the US, and represents the first therapy option against synovial sarcoma in more than a decade. It is indicated to treat adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, and whose tumours express the MAGE-A4 antigen. Additionally, the tumours need to have a certain HLA type— HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive. The approval was granted based on results from the Phase II SPEARHEAD-1 trial (NCT04044768). Amongst 44 patients with synovial sarcoma, the overall response rate (ORR) to treatment was 43% with a median duration of response of six months (95% CI: 4.6, not reached). Continued approval remains subject to verification of clinical benefit in further trials. Synovial sarcoma is a rare, soft tissue cancer with a five-year survival rate of around 36%, which drops to 20% with metastatic disease. It largely effects young adults, with a third of diagnoses occurring in patients under the age of 30. Brandi Felser, CEO of the Sarcoma Foundation of America, described the recent development as, “a renewed sense of hope for this patient community.” The FDA approval follows a series of partnerships made by Adaptimmune. In 2021 the company collaborated with Genentech, a division of Roche, to develop T cell therapies in a five-year deal which could see Adaptimmune receive over $3bn. The company has further partnered with Agilent Technologies to develop the MAGE-A4 ICH 1F9 pharmDx companion diagnostic, which also received FDA approval today (2 August), as well as with Thermo Fisher Scientific to include Tecelra in the labelling for their SeCore CDx HLA-A Locus Sequencing System. See Also: Casgevy: Launch sequence and price analysis of the first marketed CRISPR therapy FibroGen axes 75% of staff after lead cancer therapy fails in Phase III trials As per GlobalData analysis, afamitresgene autoleucel treatment for synovial sarcoma is projected to generate $9mn in revenue for Adaptimmune this year. This is set to reach $97mn by 2030. GlobalData is the parent company of Pharmaceutical Technology . Adaptimmune states that it is now ready to take Tecelra orders and that its integrated AdaptimmuneAssist programme is available to support personalised treatment. The company plans to have at least six treatment centres running this year, increasing to 30 centres within two years. In the 2 August press release announcing the approval, Adrian Rawcliffe, Adaptimmune CEO also outlined the company’s plans for other therapies. He said Adaptimmune plans, “to progress lete-cel , the next late-stage investigational treatment in our sarcoma franchise, with a rolling BLA submission to the FDA next year.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy