Telix Pharmaceuticals Limited (ASX: TLX) reports that the London-based Great Ormond Street Hospital (GOSH) has received UK research ethics approval to commence a Phase II academic study of Telix’s investigational product, TLX66 (90Y-DTPA-besilesomab), in children with high-risk leukemia.
Relevant approvals from the Medicines and Healthcare products Regulatory Agency (MHRA), and Administration of Radioactive Substances Advisory Committee (ARSAC) have also been received. The study is independently funded by the generosity of a philanthropic foundation, with GOSH as the sponsor.
The open label Phase II study is being carried out by GOSH to evaluate safety and efficacy of TLX66 as part of a reduced toxicity conditioning regimen in children and adolescents undergoing allogeneic haematopoietic stem cell (HSCT) transplantation.
The independent trial will enroll 25 patients and follows the successful completion of a Phase I study of 10 patients with relapsed refractory leukemia. TLX66 targets CD66, a receptor expressed on specific types of immune/blood cells and has been granted orphan drug designation (ODD) status in Europe for bone marrow conditioning (BMC) for hematopoietic stem cell transplantation (HSCT), a broad clinical indication.
Telix Chief Medical Officer, Dr Colin Hayward, said that in addition to pediatric leukemia, prior Phase I and II clinical studies of TLX66 have demonstrated encouraging efficacy and safety data in multiple myeloma, and in patients with other hematological malignancies currently ineligible for stem cell transplant owing to toxicity.
Telix recently reported positive topline results from the TRALA study of TLX66 at the University of Southampton (UK) for patients with systemic amyloid light chain amyloidosis (SALA), a rare disease with a poor prognosis characterised by abnormal protein deposition in the organs of the body.
We are pleased that Great Ormond Street Hospital, an international centre of excellence in child healthcare, has received ethics approval to use this investigational product in an important academic study of children and adolescents with high-risk leukemia,” Dr Hayward said.
“This disease currently has one of the poorest prognoses amongst pediatric cancers, and we will be closely monitoring progress by the team at GOSH, in line with Telix’s mission to help patients with cancer live longer, better quality lives.”