MorphoSys AG, one of the world's leading biotechnology companies, has announced that its partner Roche has expanded the ongoing SCarlet RoAD phase II clinical trial of gantenerumab for prodromal (early) Alzheimer's disease to a potentially pivotal study. The trial size will be increased from 360 to 770 participants, and a favourable outcome to the trial could be used by Roche to support a marketing application for gantenerumab.

The expansion of the study triggered a clinical milestone payment to MorphoSys, the details of which were not disclosed. MorphoSys stands to receive further developmental milestones as well as royalties on product sales.

“This is a major step forward for a HuCAL antibody program that has genuine blockbuster potential,” commented Dr Marlies Sproll, chief scientific officer of MorphoSys AG. “Gantenerumab is the first investigational antibody development programme for Alzheimer's disease to be clinically tested in a setting for which there is great hope, namely the treatment of early-stage, pre-dementia subjects. A potential path to market for this programme has become much clearer with this transition to a pivotal trial.”

“We believe the greater opportunity to make a difference in patients' lives is in early diagnosis and intervention,” said Luca Santarelli, Head of Neuroscience at Roche. “Our attempt is to utilize a biomarker-driven approach, leveraging both our pharmaceutical and diagnostics divisions to develop a companion diagnostic for gantenerumab to select patients at the prodromal stage, before significant damage to the brain has occurred.”

Gantenerumab is an optimized, fully human antibody that was developed on behalf of Roche by MorphoSys scientists using the Company's proprietary HuCAL technology. Gantenerumab is unique amongst antibodies in development in that it binds to both the N-terminus and mid-section of the 42 amino acid amyloid-& beta; peptide. It has been shown to break down amyloid plaque both in vitro and in vivo. In phase I clinical trials conducted by Roche), the antibody was found to bring about rapid, dose-dependent clearance of plaque from the brains of mild to moderate Alzheimer's disease patients. The ongoing clinical trial is designed to evaluate its effect on cognition and functioning as well as its safety and pharmacokinetics in patients with prodromal, or early-stage, Alzheimer's disease. In this phase of the disease, which can be characterized by measuring certain biomarkers, patients have only mild cognitive impairment and have not yet been diagnosed with Alzheimer's disease. According to recent research, amyloid plaque may accumulate even at this early stage in the brains of sufferers, and may lead to full-blown disease.

MorphoSys's clinical pipeline now comprises one programme in phase III development, seven in phase II and twelve in phase I. Of these, four are proprietary, as yet un-partnered programmes, namely MOR103, which is in a phase I b/II a trial for rheumatoid arthritis and a phase I b trial in multiple sclerosis, MOR208, which is in a phase I trial for chronic lymphocytic leukaemia and MOR202, which is in a phase I/IIa trial for multiple myeloma.

MorphoSys developed HuCAL, the most successful antibody library technology in the pharmaceutical industry. By successfully applying this and other patented technologies, MorphoSys has become a leader in the field of therapeutic antibodies, one of the fastest-growing drug classes in human healthcare.