MacroGenics, Inc. (Nasdaq: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MGD006 (also known as S80880), a DART® molecule that recognizes both CD123 and CD3, for the investigational treatment of acute myeloid leukemia (AML).
MGD006 is currently being evaluated in the U.S. and Europe in a Phase 1 dose-escalation study designed to assess the safety and tolerability of the molecule in patients with relapsed/refractory AML or myelodysplastic syndrome (MDS). MacroGenics retains full development and commercialization rights to MGD006 in the U.S., Canada, Mexico, Japan, South Korea and India. Servier participates in the development and has rights to MGD006 in all other countries.
The FDA orphan drug designation provides certain incentives for medications intended for the treatment, diagnosis or prevention of rare diseases. At present, these incentives include seven years of marketing exclusivity for the orphan indication, certain federal grants, tax credits and waiver of certain FDA fees.
"The FDA's decision to grant orphan drug designation for MGD006 in AML is an important regulatory milestone for MacroGenics as we continue to develop this bispecific DART molecule in this difficult-to-treat disease," said Scott Koenig, M.D., Ph.D., President and Chief Executive Officer of MacroGenics. "We believe MGD006 has the potential to be a significant advancement in the treatment of AML, and are pleased that the FDA has recognized the potential of MGD006 to benefit patients in need. We expect to select a dose this year to advance MGD006 into its next phase of clinical development."
MGD006 is a clinical-stage molecule that recognizes both CD123 and CD3. CD123, the Interleukin-3 receptor alpha chain, has been reported to be over-expressed on cancer cells in a wide range of hematological malignancies including AML and MDS.
The primary mechanism of action of MGD006 is believed to be its ability to redirect T lymphocytes to kill CD123-expressing cells. To achieve this, the DART molecule combines a portion of an antibody recognizing CD3, an activating molecule expressed by T cells, with an arm that recognizes CD123 on the target cancer cells.
In December 2016, MacroGenics presented initial clinical experience from the ongoing Phase 1 study of MGD006. Dosing schema and supportive care regimens have been refined to enable therapeutic goals and decrease the effects of cytokine induction by MGD006, an anticipated event resulting from the engagement and activation of T lymphocytes. In addition, the Company continues to characterize the pharmacokinetic properties and clinical activity of MGD006 in AML patients.
About MacroGenics, Inc.
MacroGenics is a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases. The Company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms. The combination of MacroGenics' technology platforms and protein engineering expertise has allowed the Company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies. For more information, please see the Company's website at www.macrogenics.com. DART, MacroGenics and the MacroGenics logo are trademarks or registered trademarks of MacroGenics, Inc.
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