WASHINGTON, DC — Vanda Pharmaceuticals, Inc., today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate VCA-894A for the treatment of a patient with Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within the IGHMBP2 gene. CMT2S...
Latest News
Nashville, TN – By mapping the cellular and molecular geography of colorectal cancer, Vanderbilt University and Vanderbilt University Medical Center researchers collaboratively have discovered why most colorectal tumors escape detection and destruction by the body’s immune system. Their findings, reported in the journal Cell, could lead to new ways to...
A set of expert consensus-based, quality-of-care indicators identified considerable variability in the quality of pancreatic cancer care among hospitals and may be used to evaluate and identify areas for improvement, according to a new study in the June 9 online issue of the Journal of the National Cancer Institute. Factors...
PARIS – Variant, a company dedicated to developing innovative treatments for inherited eye orphan diseases, announces the decision of the European Commission to grant Orphan Drug Designation (ODD) for VAR002 for the treatment of Leber congenital amaurosis (LCA) and Cone-rod dystrophy (CORD). The decision is based on a positive opinion...
CAMBRIDGE, Mass. – VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for VBI-1901, VBI’s cancer vaccine immunotherapeutic candidate for the treatment of...
Combination therapies including venetoclax and another therapy have displayed promising results against subtypes of acute myeloid leukemia (AML) that are particularly difficult to treat, including relapsed or refractory AML with a specific mutation, high-risk AML and treated secondary AML. Researchers from The University of Texas MD Anderson Cancer Center presented...
Venetoclax is considered a breakthrough treatment for acute myeloid leukemia (AML). But venetoclax combination therapies do not seem to be as useful for treating acute myeloid leukemia that develops as a complication of myeloproliferative neoplasms, a condition known as post-MPN-AML, according to a study recently published in Blood Advances. Post-MPN-AML is...
Among patients with previously treated Waldenström macroglobulinemia (WM), venetoclax appears to be both safe and effective regardless of CXCR4 status, according to research published in the Journal of Clinical Oncology. WM, in which MYD88 and CXCR4 mutations are common, is defined by immunoglobin M secretion in major organs, including the...
San Diego, California – The phase 2 trial PrE0405 met its primary endpoint, achieving a complete response (CR) rate of 85% in 33 patients over the age of 60 with mantle cell lymphoma (MCL) who received bendamustine and rituximab, a standard chemo-immunotherapy treatment, along with venetoclax, which is investigational in...
Encinitas, Calif. — Ventyx Biosciences, Inc. (Nasdaq: VTYX) (“Ventyx”), a clinical-stage biopharmaceutical company focused on advancing novel oral therapies that address a broad range of inflammatory diseases with significant unmet medical need, announced financial results for the fourth quarter and full year ended December 31, 2022, and highlighted recent pipeline...