Verastem has obtained orphan drug designation from the US Food and Drug Administration (FDA) for its VS-5584 to treat mesothelioma.
VS-5584 is an orally available compound, which has showed potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2.
Mesothelioma is an aggressive form of cancer, which occurs in the mesothelium, the thin layer of tissue that covers the lungs and other organs.
Verastem president and CEO Robert Forrester said: "This is an important regulatory milestone for Verastem and, together with our European orphan medicinal product designation, will facilitate our global development of VS-5584 to help improve the available treatment options for patients suffering from this highly aggressive cancer.
"We look forward to taking full advantage of the opportunities that orphan designation allows in order to bring this potential new treatment option to patients as rapidly as possible."
Recently, the company commenced a Phase I clinical trial to assess the combination of VS-5584 and VS-6063 in patients with relapsed or progressive malignant pleural mesothelioma.
According to the company, the combination clinical study is supported by preclinical work showing the synergistic activity of VS-6063 and VS-5584 in mesothelioma models in vitro and in vivo.
VS-6063 (defactinib) is an orally available compound, developed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK).
In addition, VS-5584 is being assessed in a Phase I trial in advanced solid tumours where the compound was generally well tolerated and preliminary activity has been observed, including in mesothelioma.
Verastem noted that some patients have been on trial for over six months and the maximum tolerated dose of VS-5584 has not been reached.
Image: Micrographs showing mesothelioma in a core biopsy. Photo: courtesy of Robertolyra.