MTX110 is in clinical development for newly-diagnosed diffuse intrinsic pontine glioma (DIPG). DIPG is a high grade childhood brain tumour. The cause is unknown. DIPG develops in a part of the brain stem known as the pons, which controls essential body functions including heartbeat, breathing, eyesight and balance. The symptoms of DIPG may include; abnormal alignment of the eyes, arm and leg muscle weakness, unstable balance and speech difficulties. Because the tumour grows amongst normal nerves cells of the brain stem, it is difficult to treat as they are not surgically resectable. Radiotherapy can temporarily slow the tumour’s growth but is not curative and chemotherapy is often ineffective.
MTX110 contains the anticancer drug panobinostat, which works by inhibiting an enzyme that increases the division and growth of cancer cells. Orally administered panobinostat cannot cross the blood brain barrier and its use in treating DIPG is currently limited. MTX110 contains a special formulation of panobinostat that enables the direct delivery of high concentrations of panobinostat by infusion to tumour sites, which bypasses the blood-brain barrier. This is expected to improve both the safety and efficacy of the treatment while reducing the potential for toxicity and other side effects.
Brigatinib is a medicinal product that is being developed for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) whose disease have progressed following treatment with alectinib or ceritinib. NSCLC is the most common type of lung cancer although a small proportion of NSCLC patients have a rearrangement in the ALK gene. Locally advanced or metastatic cancer means cancer has spread outside the lungs where it started, to other parts of the body and cannot be cured. Current treatment with drugs such as alectinib or ceritinib are effective in slowing the disease and helping patients to live longer, although some patients eventually develop treatment resistance and will require other therapies.