Shandong Ruzhi Biomedical Technology Co. Ltd. has synthesized compounds reported to be useful for the treatment of neurodegeneration, cardiovascular disorders and cerebrovascular disorders.
DXC-006 (Hangzhou DAC Biotechnology Co. Ltd.) is an antibody-drug conjugate (ADC) that targets CD56, a molecule involved in cell-cell adhesion and cell-matrix adhesion that is overexpressed in neuroblastoma, small-cell lung cancer and multiple myeloma, but less frequently expressed in normal tissues. DXC-006 consists of an anti-CD56 antibody (DXA-006) linked to the topoisomerase 1 (TOP1) inhibitor CPT-116 through a linker.
Essential Pharma Ltd. is paying up to £70 million (US$88.7 million) to acquire Renaissance Pharma Ltd., taking ownership of Hu14.18, a treatment for high-risk neuroblastoma. The antibody has completed a 64-patient phase II trial conducted by St Jude’s Children’s Research Hospital, in which it showed an improvement in overall survival of around 50% compared to currently available therapies.
Researchers at Boston Children's Hospital and Harvard Medical School have explored the possibility of using anaplastic lymphoma kinase (ALK) as a target for the application of ALK.CAR T cells to treat neuroblastoma, the most common and deadliest tumor of infancy.
The GOBACK study analyzed germline susceptibility in children with birth defects and a cancer diagnosis, including a total of 47 probands. Whole-genome sequencing was performed, and researchers evaluated single-nucleotide variants, insertion/deletion variants, as well as copy number variations.
After considering the evidence, the U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 14-6 Oct. 4 that the data from a single external-controlled trial and well-established preclinical animal models present sufficient evidence to demonstrate that US Worldmeds LLC’s DFMO (eflornithine) improves event-free survival in pediatric patients with high-risk neuroblastoma.
For the second time in two weeks, the extent of regulatory flexibility will be at the heart of a U.S. FDA advisory committee meeting. The Oct. 4 meeting of the Oncologic Drugs Advisory Committee (ODAC) comes exactly a week after the Cellular, Tissue and Gene Therapies Advisory Committee voted overwhelmingly that the evidence presented for Brainstorm Cell Therapeutics Inc.’s amyotrophic lateral sclerosis drug, Nurown (debamestrocel), didn’t meet the agency’s flexibility standard. If the FDA’s briefing document for the ODAC meeting is anything to go by, the outcome for US Worldmeds LLC’s eflornithine, also known as DFMO, could be more positive, even though once again the agency is asking if the evidence from a single trial, along with supportive data, is sufficient.
Newco Renaissance Pharma Ltd. has been formed to take a new treatment for neuroblastoma developed at St Jude’s Children’s Research Hospital through to market. The product, Hu14.18, has been in-licensed following a phase II trial conducted at the hospital in which patients with newly diagnosed high-risk neuroblastoma had a three-year event-free survival of 73.7% and an overall survival of 86%.
