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
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

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].