Vaccinex is developing antibody candidate drugs for the treatment of neurodegenerative diseases and cancer. The lead program is evaluating pepinemab, a monoclonal antibody targeting SEMA4D, for the treatment of Huntington’s disease (HD). In earlier studies, treatment with pepinemab improved metabolism (increased glucose transport, measured by FDG-PET) of affected neurons in HD patients. Huntington’s disease is an inherited deteriorating chronic illness of the brain (killing neurons), characterized by abnormal movements (chorea), with gradual loss of cognitive and neurological functions. At present, there is no cure or effective treatment for HD. SEMA4D (semaphoring-4D) is a an extracellular signaling molecule involved in neuro-inflammation. Vaccinex’s pepinemab has received Fast Track and Orphan Drug designations by FDA.
 

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In October 2020, Vaccinex expects to release topline results from a 265-patient pivotal clinical trial on the use of pepinemab for the treatment of patients suffering from Huntington’s disease (HD). Endpoints of the study include clinical and behavioral outcomes, as well as measures of brain volume, metabolic activity and inflammation. Based on trial results, Vaccinex plans to seek FDA approval to commercialize pepinemab in the United States. The Company believes that by preventing astrocyte-driven neuro-inflammation, pepinemab might act as a protector of neurons in the brain of HD patients.
Besides HD program, Vaccinex is developing pepinemab in combination with checkpoint inhibitors for the treatment of non-small cell lung cancer, head and neck, colorectal, melanoma and pancreatic cancer. In cancer patients, pepinemab modifies the tumor microenvironment by reducing the number of inhibitory MDSC (myeloid-derived suppressor cells) and stimulating anti-tumor T CD8 killing lymphocytes (improving penetration of tumor mass), resulting in amplification of the anti-tumoral immune response.

Xenetic Biosciences is a biotechnology company developing XCART™, a personalized, patient-specific CAR-T platform anti-cancer technology engineered to target tumor-specific neoantigens. XCART targets the B-cell receptor (BCR) on the surface of B-cell lymphoma cancer cells, which is unique to each patient. The technology is designed to screen for peptides selectively binding to the BCR on the surface of a patient’s lymphoma cancer cells. The specific peptide is engineered into the binding domain of a “chimeric antigen receptor” creating a CAR-T cell therapy drug which only recognizes cancer cells specific to each lymphoma patient. In addition to its cancer program, Xenetic receives royalty payments from partner Takeda for application of Xenetic’s PolyXen® drug delivery technology for the treatment of coagulation disorders.
 

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Recently, Xenetic signed collaboration agreements with Scripps Research, one of the leading academic institutions in the world, and Pharmsynthez. Given that Scripps and Pharmsynthez’s scientists are co-inventors of XCART, Xenetic believes the collaborations will be crucial for the successful development of the technology. Xenetic believes XCART will become a preferred cell therapy for the treatment of B-cell non-Hodgkin’s lymphomas. XCART could be viewed as the reverse of a typical CAR-T, which utilizes “highly specific antibody domains” to recognize a Tumor Antigen. In contrast, XCART targets BCR, which has similarities to a “highly specific antibody domain”. In the case of XCART, “the antibody” is the “Tumor Antigen” instead of the “Medicine” (Reverse CAR-T). Given that XCART will not recognize BCR of normal B lymphocytes, it could potentially have a superior safety profile relative to a typical CAR-T. Based on these competitive advantages, Xenetic expects to attract a global leader in the industry to help develop the technology.