• FDA grants Fast Track Designation for Daretabart (hu1418K322A), recognising the significant unmet medical need in high-risk neuroblastoma and enabling access to accelerated and rolling review
• FDA clearance of IND application enables initiation of the SHINE Phase II/III clinical trial in relapse and refractory patients with high-risk neuroblastoma
• First commercial-scale GMP batch of Daretabart (hu1418K322A) successfully manufactured
• Programme builds on positive Phase II data which demonstrated overall survival rate of 86.0%

Renaissance Pharma Limited, an Essential Pharma company focused on development stage assets, announces that the US Food and Drug Administration (FDA) has granted Fast Track Designation for Daretabart (hu1418K322A), a novel anti-GD2 monoclonal antibody in development for the treatment of high-risk neuroblastoma (HRNB), a rare paediatric cancer. The designation recognises the significant unmet medical need in HRNB and will support more frequent interactions with the FDA throughout development, as well as eligibility for accelerated and rolling review.

The company also confirms it has received IND clearance from the FDA, enabling initiation of the SHINE Phase II/III clinical trial in relapse or refractory children with HRNB in the United States. The first commercial-scale Good Manufacturing Practice (GMP) batch of Daretabart (hu1418K322A) has also been successfully manufactured for use in the SHINE trial.

Daretabart (hu1418K322A) is being developed by Renaissance Pharma under an exclusive license agreement with St. Jude Children's Research Hospital, a global leader in pediatric cancer research and treatment. The antibody targets GD2, a cell surface antigen highly expressed on neuroblastoma cells. By binding to GD2, Daretabart (hu1418K322A) is designed to enhance immune-mediated tumour cell killing, while incorporating novel structural modifications intended to improve tolerability profile.

The programme builds on encouraging Phase II data evaluating Daretabart (hu1418K322A) as part of first-line therapy and in the post-consolidation setting for patients with HRNB. The study demonstrated a three-year event-free survival (EFS) rate of 73.7% and an overall survival (OS) rate of 86.0%. These results were published in the Journal of Clinical Oncology in December 2021.

The successful manufacture of the first commercial-scale GMP batch marks a key operational milestone and underscores Renaissance Pharma’s commitment to ensuring reliable, high-quality supply as the programme advances. This achievement supports ongoing clinical development and represents an important step towards future commercial readiness.

Simon Ball, Interim CEO of Essential Pharma and Director of Renaissance Pharma Limited said: “Daretabart has the potential to make a real difference for children with high-risk neuroblastoma, a disease where outcomes remain deeply inadequate despite intensive treatment. FDA Fast Track Designation is an important external validation of that potential, and together with IND clearance and our ability to manufacture at commercial scale, reflects the strength and maturity of this programme. Having worked exceptionally hard behind the scenes for a number of months, it is with great excitement that we announce this update today. We are executing at pace, and look forward to sharing data from the SHINE trial as it progresses. Today’s news brings us another step closer to delivering Daretabart as a meaningful new treatment option for children facing this aggressive cancer.”