Treeway, a Netherlands-based, amyotrophic lateral sclerosis (ALS) R&D strategy-focused company, and the Leiden Academic Center for Drug Research (LACDR) at the University of Leiden, the Netherlands, have formed a collaboration focused on the optimization of clinical trial designs and data analysis for ALS through the use of population disease progression models. Both parties aim to obtain a better understanding of the disease by developing ALS physiology-based disease models and test the effect of interventions in the different identified pathways.

The scope of this collaboration encompasses two assignments:

• The development of a population disease progression model for ALS. Currently a phase III clinical trial with Treeway’s drug candidate TW001 is being developed. The availability of a mathematical model characterizing both the average as well as individual time course of disease development will enable optimization of the trial design, as well as a more effective analysis of the study data.
• The development of an in silico ALS physiology-based disease model. This model should provide insight in the complex interaction between neurological and immunological processes, and the cell signaling networks in this. The model can be used to test what-if scenarios and explore the impact of interventions via different pathways.

Inez de Greef, CEO of Treeway, said, “By applying pharmacometric modeling and clinical trial simulations, we can streamline our clinical development programs for new treatments for ALS. In addition, the physiology-based model will provide direction to our discovery efforts.”

Professor Piet Hein van der Graaf, director of the LACDR and head of the department of pharmacology at the University of Leiden, believes the collaboration with Treeway signals an important trend in drug discovery and development.

He said, “Rare and neglected diseases have been ignored by large pharmaceutical companies due to the limited return on high-risk R&D investment. Therefore, collaborations like this between academics and small entrepreneurial biotech companies will be of vital importance to bring new medicines to patients in areas of high unmet medical need like ALS.”