Elan is making a $20 million equity investment in U.S. firm Proteostasis Therapeutics as part of a drug discovery partnership focused on neurodegenerative and dementia-related disorders including Parkinson disease, Huntington disease, multiple sclerosis, amyotrophic lateral sclerosis, and Alzheimer disease. The investment will give Elan a roughly 24% holding in Proteostasis, and the Irish drugs firm will also be granted a right of first negotiation to exclusively license compounds emerging from the partnership. It has also agreed to provide up to $30 million in collaboration funding over five years.
Proteostasis is developing a class of small molecule therapeutics known as proteostasis regulators for treating neurodegenerative, metabolic, genetic, and inflammatory disorders. The candidates are designed to correct perturbed signalling pathways in disease, and restore the correct interaction of the network of proteins controlled by known biological signalling pathways and processes that control protein folding, trafficking, and clearance, which are collectively known as the proteostasis network.
Proteostasis has started the job of fully mapping the proteostasis network, and it is this knowledge that is being exploited for the discovery of new therapeutics. The firm claims such proteostasis regulators offer a new approach to the treatment of diseases associated with loss of cell function due to accumulation, degradation, or aggregation of misfolded proteins.
“Proteostasis’ drug discovery platform is based on an emerging concept in biology, namely that perturbations in the proteostasis network pathways fundamentally underlie a wide variety of diseases, including neurodegenerative diseases as well as orphan conditions such as cystic fibrosis and lysosomal storage disorders, remarks Christopher K. Mirabelli, Ph.D., the firm’s board chairman. “This alliance provides a creative approach to financing our discovery platform and supporting the development of multiple discovery and development programs in parallel.”
“Through an emphasis on protein folding and turnover, the Proteostasis business relationship is both complementary and additive to our internal research effort, which is focused on genetic targets associated with a broad array of neurodegenerative disorders,” comments Dale Schenk, Ph.D., Elan’s CSO, and Ted Yednock, Ph.D., head of research. This initiative enables us to simultaneously expand possible targets and accelerate our progress in bringing science to patients.”
Proteostasis has spent the last few months expanding its IP and knowledge base. In April the firm acquired two exclusive licenses from Harvard University to technologies for enhancing the activity of the ubiquitin-proteasome pathway involved in protein clearance. It claims the acquired technologies, which include novel targets and small molecule compounds, will be relevant to its development of new therapeutics for a range of neurodegenerative and orphan diseases. In 2010 Proteostasis announced a research collaboration with Harvard Medical School researchers that aims to evaluate signaling pathways important for the treatment of neurodegenerative disorders such as Parkinson’s disease and Huntington’s disease.
Also in 2010 the firm inked an exclusive license agreement with New York University covering intellectual property related to components involved in the cellular response to stress, which has been implicated in the mechanism underpinning many common diseases of aging.