MTX-110 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.
MTX-110 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. MTX-110 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.
Nivolumab in combination with ipilimumab is in clinical development for PD-L1 positive patients with previously untreated unresectable or metastatic urothelial cancer regardless of cisplatin eligibility. Urothelial cancer, a subset of bladder cancer, occurs on the lining of the renal pelvis, ureter, bladder and urethra, and other parts of the urinary system. In some cases,the tumour spreads into the surrounding muscles or other parts of the body which means that it cannot be cured by surgery. Metastatic urothelial cancer occurs when the cancer has spread to other parts of the body, such as the liver or bones. The symptom of urothelial cancer is blood in the urine, but symptoms may only appear once the cancer grows larger or into the deeper layers of the bladder wall for both men and women. Other symptoms may include increased frequency/urgency/pain of urine passing, weight loss, back/lower tummy/bone pain, fatigue and illness.