PureTech Health and Roche Sign Collaboration Deal Worth $1 Billion-Plus
Boston-based PureTech Health inked a multi-year collaboration deal with Swiss-based Roche to advance PureTech’s milk-derived exosome platform technology for oral dosing of Roche’s antisense oligonucleotide platform.
Under the terms of the agreement, Roche will pay PureTech up to $36 million in upfront payments, research support, and early preclinical milestones. PureTech will also be eligible for development milestones worth more than $1 billion, as well as more sales milestones and royalties for an undisclosed number of products.
PureTech’s platform allows for the oral administration of complex drug payloads, such as nucleic acids, peptides, and small molecules. The exosomes used for this platform are believed to move by way of lymphatic circulation, which opens up the possibility of targeting immune cells in new ways. Also, drugs that can be taken orally generally have a competitive advantage over drugs that require injections or infusions.
“We are excited to accelerate the development of this promising technology from our internal lymphatic and immune cell trafficking programs,” said Daphne Zohar, PureTech’s co-founder and chief executive officer, in a statement. “The expertise and resources that Roche is bringing to the collaboration will help us to potentially address one of the biggest challenges in olignonucleotide-based therapeutic development: oral administration of nucleic acids.”
PureTech’s internal research-and-development programs focus on the brain-immune-gut axis with emphasis on lymphatics and how immune cells travel throughout the lymph system to modulate immunity. The company has three products in the pipeline for central nervous system disorders, including one wrapping up Phase III, five for the immune system, two of which are in Phase II, and two in gastrointestinal, with one in Phase III.
Part of PureTech’s business model is to create and spin out affiliate biotech companies. It has its internal programs, but it also acts as something like a venture capital firm, developing and funding affiliate companies.
In June, PureTech’s affiliate, Karuna Pharmaceuticals, received $8 million from the UK’s Wellcome Trust, which will be used to advance its lead program, KarXT (Karuna-Xanomeline-Trospium) through Phase II for schizophrenia. The key ingredient, xanomeline, had shown efficacy in clinical trials in schizophrenia and Alzheimer’s disease, but there were tolerability issues. KarXT is designed to resolve the tolerability issues by targeting M1/M4 muscarinic receptors in the brain while blocking their activation outside the brain, which was the cause of the adverse events.
“Antipsychotics are the mainstay therapy for the treatment of schizophrenia and have all relied on the same fundamental mechanism of action for the past 60 years,” said Andrew Miller, president and chief executive officer of Karuna, in a statement. “Currently available treatments have limited efficacy—primarily addressing only positive symptoms, such as hallucinations and delusions—and are often associated with serious, often irreversible, side effects, leaving a significant need for new treatments for people living with this devastating disease. With KarXT, we have an opportunity to potentially improve positive, negative, and cognitive symptoms based on prior compelling efficacy data with xanomeline, and without the debilitating side effects of existing therapies. We are delighted that Wellcome is continuing to back this important new therapeutic approach.”
Wellcome Trust had previously awarded the company $3.8 million to fund a Phase I trial, which had successful results.