dm+d
Unassigned
New Medicines
Advanced synovial sarcoma or myxoid/round cell liposarcoma
Information
New molecular entity
Adaptimmune
Adaptimmune
Development and Regulatory status
Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
Nov 21Adaptimmune intends to file a biologics license application in 2022 [9]
Dec 20Has orphan drug status in EU [6].
Aug 20UK-based Adaptimmune intends to launch melanoma-associated antigen 4 specific T-cell therapy for Synovial sarcoma in USA in 2022. Plans for EU/UK filings not stated but will presumably not be before a US submission [8].
Jul 20EMA awards PRIME status for treatment of synovial sarcoma [5].
Dec 19US FDA grants Regenerative Medicine Advanced Therapy Designation (RMAT) [4].
Sep 19US FDA grants orphan drug status to melanoma-associated antigen 4 (MAGE-A4) specific T-cell therapy for treatment of patients with soft tissue sarcoma [4].
Category
A genetically modified autologous melanoma-associated antigen 4 (MAGE A4) specific T cell therapy. SPEAR® technology works by modifying the complementarity-determining regions (CDRs) of the patient T-cells in order to enhance affinity to the cancer cell.
Soft tissue sarcomas develop from soft tissue cells including smooth muscle cells (leiomyosarcomas), fat cells (liposarcomas), fibrous connective tissue (fibrosarcomas), skeletal muscles (rhabdomyosarcomas), synovium (synovial sarcomas), blood vessels (angiosarcomas), breast ducts (phyllodes tumours) and nerves (nerve sheath tumours) [1]. Around 3,300 people were diagnosed with soft tissue sarcoma in 2010 in the UK [2].
Advanced synovial sarcoma or myxoid/round cell liposarcoma
Intravenous infusion
Further information
Yes
Topic prioritised for potential TA guidance production
Trial or other data
Nov 21PII SPEARHEAD-1 trial is still recruiting (estimated enrollment 90 patients); collection of primary outcome data now expected to complete May 22 [10].
Nov 21Company is expecting PII SPEARHEAD to meet primary endpoints (n=50 aged 19 to 73). An independent review shows overall response rate of 34% (includes 36% of pts with synovial sarcoma and 25% of pts with MRCLS). The disease control rate is 85%, while 75% of pts demonstrated an ongoing response for up to 65.3 weeks post-infusion. Adverse events were observed in 66% of pts, mostly low grade. [9]
Oct 20PII SPEARHEAD trial is recruiting. It intends to support the registration of melanoma-associated antigen 4 (MAGE-A4) specific T-cell therapy for the treatment of advanced synovial sarcoma and MRCLS [6,7].
Feb 20PII SPEARHEAD-1 trial is recruiting [3].
Jul 19PII SPEARHEAD-1 trial to assess antitumor activity of melanoma-associated antigen 4 (MAGE-A4) specific T-cell therapy, in patients with synovial sarcoma and myxoid/round cell liposarcoma (MRCLS) starts (ADP0044-002; NCT04044768). The trial will enrol 60 patients aged 16 to 75 years with inoperable or metastatic synovial sarcoma or MRCLS who have received prior chemotherapy, in the US, Canada, France, Spain and UK (sites are UCLH and The Christie). The primary endpoint is overall response rate; collection of these data is due to complete Jan 22. Subsequently a radiation sub-study was initiated to assess the effect of low-dose radiation on increasing anti-tumour activity and will enrol approximately 10 participants [3,4].