Thrombocytopenia is a big word that can carry big consequences for those struggling with rare blood disorders, especially among those using heparin to treat and prevent blood clots. But, help is on the way. The National Institutes of Health’s National Heart, Lung, and Blood Institute (NHLBI) recently awarded VERALOX Therapeutics, a small molecule drug discovery and development company, a $319,133 Small Business Innovation Research grant to develop a novel therapeutic for patients suffering from heparin-induced thrombocytopenia and thrombosis (HIT/HITT).
The syndrome is caused by an immune response to heparin and can lead to catastrophic complications, including gangrene, amputations, deep vein thrombosis, pulmonary embolism and/or premature death. The threat is significant because unfractionated heparin (UFH) and low molecular weight heparin (LMWH) are the most widely used anticoagulants in the world with approximately 12 million patients exposed annually in the US alone. Of those patients, 0.5-1.5% develop HIT/HITT every year.
Located in the Frederick Innovative Technology Center Inc. (FITCI), VERALOX is building on established evidence that the enzyme 12-lipoxygenase (12-LOX) and its metabolite, 12-hydroxyeicosatetraenoic acid (12-HETE) are key contributors to the underlying nature of HIT/HITT. VERALOX’s development candidate, VLX-1005, halts aberrant platelet activation and thrombosis in clinically relevant animal models, without impairing normal clotting functions.
The NHLBI SBIR grant allows VERALOX and Dr. Michael Holinstat, Professor of Cardiovascular Medicine and Surgery at the University of Michigan, to continue preclinical characterization of VLX-1005 and conduct early toxicology studies. These efforts represent critical development activities on the path towards human clinical trials. David Maloney, Ph.D., led the drug’s development at NIH before cofounding VERALOX with Matthew Boxer, Ph.D., and Jeffrey Strovel, Ph.D. They recently executed an exclusive world-wide license for the technology from four institutions: NIH/National Center for Advancing Translational Sciences, Eastern Virginia Medical School, University of California Santa Cruz and Thomas Jefferson University.
Maloney says, “Current treatment of HITT includes immediate discontinuation of all heparin products and initiation of Argatroban (a direct thrombin inhibitor and the only US FDA approved treatment for HITT), but this carries a significant risk of severe bleeding, and both mortality and amputation rates remain unacceptably high.” The mortality rate is ~28% and the rate of amputation and gangrene is ~9%. “This project shows potential to significantly lower the risk of devastating morbidity and deadly complications.”
Kathie Callahan Brady is FITCI’s CEO. She’s works closely with clients as they navigate the complicated realm of grant funding and applications. She says winning such a substantial amount affirms the caliber of VERALOX’s work as well as the treatment’s potential to improve so many lives. “Phase I awards normally do not exceed $150,000. So, this is a big deal.” She’s followed VERALOX’s steep climb since the company joined the incubator in March 2018. “Drug discovery can be a long, hard road, but these founders are passionately dedicated to the cause. They have a personal history with people who have suffered through this and they’ve dedicated the bulk of their careers working to this end.”