Veralox Therapeutics Gains EMA Orphan Drug Status for VLX-1005

Veralox Therapeutics, a biotechnology firm focused on pioneering treatments targeting the 12-lipoxygenase (12-LOX) pathway, recently announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its lead candidate, VLX-1005. This small molecule 12-LOX inhibitor is aimed at treating platelet-activating anti-platelet factor 4 (PF4) disorders. VLX-1005 had already received ODD and Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the prevention of thrombosis in patients suffering from heparin-induced thrombocytopenia (HIT). The EMA's decision marks another significant milestone in the regulatory journey of VLX-1005, which has already progressed through Phase 1 trials and is currently in the Phase 2 trial known as ALATHEA.

Dr. Michael Hanna, Chief Medical Officer of Veralox Therapeutics, highlighted the importance of this EMA designation. He emphasized that this regulatory achievement underscores the potential of VLX-1005 in treating severe diseases caused by platelet-activating anti-PF4 disorders like HIT.

HIT is a serious and potentially life-threatening condition that can occur after the administration of the blood thinner heparin. Heparin is widely used in various cardiovascular procedures to prevent blood clots. However, in rare cases, it can trigger an adverse reaction that leads to the activation and consumption of platelets, causing thrombocytopenia (low platelet counts) and promoting clot formation. This condition increases the risk of fatal or severe thromboembolic events. Statistics indicate that between a quarter and a third of HIT patients may succumb to the condition, while a significant number develop new blood clots, leading to complications such as deep vein thrombosis, pulmonary embolism, heart attack, or stroke.

VLX-1005 has completed Phase 1 clinical trials, demonstrating a favorable safety profile and preferred pharmacokinetics in healthy participants. The drug is now being tested in a Phase 2 clinical study. Orphan Drug Designation provides a variety of incentives to advance the development of treatments for rare diseases. These include reduced fees for multiple steps in the drug development process and 10 years of market exclusivity. Additionally, EU member states may offer further incentives for the development of orphan drugs.

HIT is a rare but serious complication that arises from heparin exposure during various surgeries and medical procedures, including kidney dialysis. Given the widespread use of heparin, there are over 300,000 suspected HIT cases annually in the United States alone, with around 50,000 cases confirmed each year. HIT patients face a high risk of severe outcomes, including death, amputation, new thromboses, and major bleeding. The diagnosis and management of HIT are complex, and there remains a significant unmet need for effective therapies due to the limited options currently available.

VLX-1005 is a novel, small molecule inhibitor of 12-LOX designed to treat or prevent thrombosis in adults with HIT. It selectively inhibits platelet 12-HETE production, a process involved in platelet activation and thrombosis. Preclinical studies have shown that VLX-1005 effectively inhibits 12-HETE synthesis and prevents thrombosis without increasing bleeding risk. Phase 1 studies involving 96 healthy participants have indicated that VLX-1005 is safe and well-tolerated, with no deaths or serious adverse events reported. The ongoing Phase 2 study is evaluating VLX-1005 in patients with suspected HIT at 12 clinical sites.

Veralox Therapeutics is dedicated to developing first-in-class therapies that target 12-lipoxygenase, aiming to address serious immune-inflammatory diseases. VLX-1005 is their lead candidate, specifically targeting heparin-induced thrombocytopenia. The company is also working on second-generation orally bioavailable 12-LOX antagonists for potential use in treating type 1 diabetes and other immune-mediated and inflammatory diseases.