This morning, it was announced that Amphivena Therapeutics, Inc. was granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for AMV564, its lead compound intended for the treatment of Acute Myeloid Leukemia (AML).
The company is developing the novel AMV564 to act as a CD33/CD3-bispecific T cell engaging antibody, and the hope is that it will eventually also act as a therapy for Myelodysplastic Syndromes (MDS) and solid tumors.
AML, also commonly referred to as acute granulocytic leukemia or acute non-lymphotic leukemia, is a cancer that initially presents in the bone marrow and quickly spreads to the blood. It is characterized by the rapid expansion of white blood cells, which obstructs the production of typical blood cells. It can sometimes extend into other parts of the body, including the lymph nodes, liver, spleen, central nervous system, and testicles.
According to the American Cancer Society
, 21,000 new cases of AML were diagnosed in the United States in 2016. Approximately half resulted in death.
“The FDA’s designation of AMV564 as an orphan drug is an important milestone for us that will provide marketing protections and economic benefits at drug approval,” said Eric J. Feldman, M.D., Amphivena’s Senior Vice President of Clinical Development in a press release
. “Given the unique safety and efficacy profile that is emerging in the clinic, we believe our CD33-targeted T cell engager will be an important drug in the armamentarium for leukemia patients who have limited treatment options today.”
AMV564 contains 2 VH and VL amino acid chains that combine to form 4 antigen-binding single chain variable fragments (scFvs). The compound, bivalent for each of the CD33 and CD3 antigens, provides voracity for each target and two times the molecular weight of monovalent constructs.
A Phase 1 clinical study of AMV564 is currently being conducted in patients with relapsed or refractory AML, and a second study in patients with MDS is expected to begin in the first half of 2018. In preclinical studies, the novel compound demonstrated potent activity against AML patient samples that was independent of CD33 expression level, disease stage and cytogenic risk. Nearly all blasts from bone marrow and spleen were eliminated by the antibody in a stringent AML patient-derived xenograft murine model.
The Orphan Drug Designation will provide benefits and incentives to Amphivena, including a period of marketing exclusivity for the first marketing application, assuming the regulatory approval is received for the designated indication.
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