Allergan expands its neurological diseases arm, buys Lysosomal Therapeutics

Allergan has purchased an exclusive option right to acquire Lysosomal Therapeutics (LTI), the company focused on innovative small-molecule research and development in the field of neurodegeneration, yielding new treatment options for patients with severe neurological diseases.

LTI-291, LTI’s lead program, aims to stimulate the activity of glucocerebrosidase (GCase) in the brain. In several lysosomal storage diseases, the activity of GCase is reduced due to genetic mutations in the GBA1 gene. Importantly, approximately 5 to 10 percent of patients with Parkinson’s disease (PD) have mutations of the GBA1 gene, and it is well documented that these patients have a more rapid progression of Parkinson’s disease. Thus, it is hoped that activation of GCase by LTI-291 will impact the progression of PD in these patients.

Under the option agreement, Allergan purchased an option right directly from LTI shareholders to acquire LTI following completion of a Phase 1b trial for LTI-291.  In addition, Allergan will provide a separate upfront research and development payment.  Allergan and LTI will establish a joint development committee to oversee the development activities for LTI-291. Additional terms were not disclosed.

“Parkinson’s disease is an area of medicine where significant unmet need exists, particularly in the development and potential breakthrough of disease-modifying treatments for the more than 2 million people suffering with Parkinson’s today,” said David Nicholson, Chief Research & Development Officer, Allergan. “The pioneering work of the LTI team in developing its GCase program harnesses the link between GCase activity and disease progression in patients with a GBA mutation who suffer from Parkinson’s disease. The intention is to provide therapeutic benefit for the affected patients by modulating GCase activity. We look forward to working with the LTI team to further their work in this area and the potential for positive Phase 1 results.”

Dr. Kenneth Marek, President and Senior Scientist at the Institute for Neurodegenerative Disorders of the Michael J. Fox Parkinson Progression Marker Initiative, Clinical Professor of Neurology at Yale University and a member of LTI’s Scientific Advisory Board added, “The key unmet need for Parkinson’s disease patients and families is to slow disease progression. LTI has taken a novel personal medicine approach to Parkinson’s Disease, targeting rapidly-progressing patients who carry a mutant GBA1 gene.  The LTI drug candidate, which is designed to reverse the loss of enzyme activity in patients with the genetic mutation, has a chance to be the first drug in its class.”