MabVax receives orphan drug designation for therapeutic vaccine to treat childhood cancer neuroblastoma

In the U.S., the designation carries significant incentives for the development of drugs to treat rare and difficult diseases. The Company is preparing to commence a Phase 2 clinical trial with the vaccine in 2015. MabVax’s neuroblastoma vaccine is a bivalent vaccine that elicits an antibody response targeting the two most common antigens on neuroblastoma cells with the objective of killing residual cancer cells that can cause recurrence.

Neuroblastoma is a rare extracranial solid tumor cancer in childhood with only about 650 to 800 cases diagnosed each year in North America. Approximately 90% of neuroblastoma patients are younger than 5 years at diagnosis and the cancer is responsible for 12% of all cancer deaths in children less than 15 years of age.

In particular, 60% of children diagnosed with neuroblastoma after 1 year of age have high-risk disease and less than half of these patients are cured even with the aggressive multi-modal regimens currently in use. Patients who relapse typically die from their disease. Children who do survive, especially those who received multimodality therapy, face severe long-term outcomes, such as blindness, hearing loss, paralysis, and learning disabilities.

J. David Hansen, Chief Executive Officer of MabVax, said, "There is a significant medical need to develop new therapies aimed at preventing recurrent disease in this childhood cancer. The orphan drug designation provides MabVax with substantial development incentives such as exclusivity, tax credits, PDUFA fee exemptions, and the opportunity to apply for grants to offset clinical expenses. The designation also allows the Office of Orphan Products Development and MabVax to collaborate more closely to facilitate development of the vaccine."

The results of a Phase I trial with the neuroblastoma vaccine conducted at Memorial Sloan-Kettering Cancer Center were published recently in Clinical Cancer Research. The study report describes a highly encouraging study result with a small cohort of difficult to treat patients who had repeatedly relapsed prior to treatment with MabVax’s vaccine.

The vaccine is intended to be given to patients with relapsed or recurrent high-risk neuroblastoma in remission or with limited residual disease after best available treatment. The vaccine is intended to elicit a targeted immune response that can kill the remaining cancer cells that can cause disease recurrence.

MabVax has already received a $125,000 Phase 1 Small Business Innovation Research Grant (SBIR) from the National Cancer Institute (NCI) to support the manufacture and testing of the neuroblastoma vaccine. The Company is expecting to finalize the manufacturing and testing of the vaccine before the end of 2014.

MabVax has also received a $400,000 grant from the Solving Kids Cancer Foundation/ Neuroblastoma Children’s Cancer Alliance UK for a modified Phase I trial to test the addition of an immunostimulatory agent to the treatment regimen.