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news.The European Commission has granted orphan medicinal product designation for Verastem's VS-5584, a dual mTORC1/2 and PI3K inhibitor, designed to treat patients with mesothelioma.
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Currently, VS-5584 is being evaluated in a Phase I clinical trial in patients with advanced solid tumors and a Phase I combination trial with VS-6063 (defactinib) in patients with relapsed malignant pleural mesothelioma.
VS-6063 is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK).
The open-label, dose escalation and schedule finding Phase I combination trial is designed to evaluate the safety, pharmacokinetics, pharmacodynamics and initial observations of clinical activity.
Around 56 patients are expected to be enrolled in the trial at clinical sites in the UK and US.
Verastem president and chief executive officer Robert Forrester said: "This European orphan designation could help facilitate the regulatory process for development of VS-5584, and if our clinical studies are successful, may allow us to bring this potential new treatment option to patients more rapidly.
"We have dosed the first patient in our first-in-human combination study of VS-5584 and VS-6063 in mesothelioma and we look forward to providing updates as the trial progresses."
The company said that VS-5584 has showed potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2.
In preclinical trials, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models.