VX15 for the potential treatment of HD also received Fast Track status from FDA in July 2016
ROCHESTER, N.Y., August 23, 2016 (GLOBE NEWSWIRE) – Vaccinex, Inc., a clinical-stage biotechnology company engaged in the discovery and development of therapeutic monoclonal antibodies to treat patients with cancer and neurodegenerative diseases, today announced that its lead antibody product candidate, VX15, which is currently being investigated in a Phase 2 clinical trial for the treatment of Huntington’s disease (the “SIGNAL” trial), has received Orphan Drug Designation by the U.S. Food and Drug Administration (“FDA”).
The SIGNAL trial is based on the company’s prior research of neurodegenerative disease mechanisms, where it was demonstrated in preclinical models that semaphorin 4D (“SEMA4D”) triggers activation of both microglia and astrocytes, the innate inflammatory cells of the central nervous system. The chronic activation of microglia and astrocytes has been implicated as a potentially important disease mechanism in HD, progressive multiple sclerosis (“MS”) and other neurodegenerative disorders. The VX15 antibody is designed to block the functional activity of SEMA4D. The SIGNAL clinical trial builds upon preclinical studies in an animal model of HD and safety data from a Phase 1 dose-escalation clinical trial of VX15 in MS patients that was completed in November 2014.
“We are pleased to receive Orphan Drug Designation from the FDA for VX15 as a potential treatment for Huntington’s disease. SIGNAL is an important proof of concept study for the role of inflammation and, in particular, SEMA4D in neurodegenerative processes. We hope that this study will contribute to treatment of HD, a serious disease for which there are, as yet, no approved disease-modifying therapies,” said Maurice Zauderer, Ph.D., Chief Executive Officer of Vaccinex.
The Orphan Drug Act provides for economic incentives to encourage the development of drugs intended to treat, diagnose or prevent rare diseases and conditions affecting (i) fewer than 200,000 people in the United States or (ii) more than 200,000 people in the United States but for which there is no reasonable expectation for recouping development and commercialization costs based on U.S. sales. Orphan drug designation would provide seven years of market exclusivity in the United States, with certain exceptions, if market approval is granted for VX15. Additional incentives may include tax credits related to clinical trial expenses and waivers from FDA user fees.
About Huntington’s Disease
Huntington’s disease (“HD”) is a hereditary neurodegenerative disease that causes the progressive breakdown of nerve cells in the brain. Symptoms of HD typically begin to manifest in mid-life and worsen over the next 10- to 25-year period. Currently, there are no treatments that can slow or reverse the course of HD. The National Institutes of Health estimates that 30,000 people in the United States have HD. In addition to symptomatic patients, more than 200,000 in the United States carry the gene and are at risk of inheriting the disease.
About Vaccinex, Inc.
Vaccinex, Inc. is a privately held clinical-stage immunotherapy company engaged in the discovery and development of human therapeutic monoclonal antibodies to treat cancer and neurodegenerative diseases, including Huntington’s disease. Vaccinex utilizes its proprietary ActivMAb® Antibody Discovery Technology for rapid, mammalian cell-based antibody selection to build its antibody pipeline and in service to its biopharmaceutical partners. ActivMAb® combines the advantages of rapid and sensitive selection by virus panning and cell sorting in one technology, with intrinsic selection of antibodies that are efficiently expressed and stable in mammalian cells. We believe that recent advances have made this technology very efficient for selection of antibodies against membrane associated proteins, an important class of target molecules for pharmaceutical development. Vaccinex is based in Rochester, New York. For more information and to contact Vaccinex (email@example.com) or visit www.vaccinex.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements reflecting the current beliefs and expectations of management. Words such as “may,” “believe,” “will,” “expect,” “plan,” “anticipate,” “estimate,” “intend” and similar expressions, as well as other words or expressions referencing future events, conditions or circumstances, are intended to identify forward-looking statements. Forward-looking statements contained in this press release include statements about expectations related to a Phase 2 clinical trial for the Company’s lead monoclonal antibody, VX15/2503. Forward-looking statements in this press release involve substantial risks and uncertainties that could cause our performance or achievements to differ significantly from those expressed or implied by the forward-looking statements, including as a result of the inherent challenges in clinical development. All forward-looking statements are based on Vaccinex’s expectations and assumptions as of the date of this press release, and actual results may differ materially. Except as required by law, Vaccinex expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.
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