PureTech Health Announces Collaboration with Roche to Advance Technology for Oral Administration of Antisense Oligonucleotides

On July 20, 2018 PureTech Health plc (LSE: PRTC) ("PureTech Health"), a clinical-stage biopharmaceutical company developing novel medicines focused on the Brain-Immune-Gut (BIG) Axis, reported that it has entered into a multiyear collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., to advance PureTech’s milk-derived exosome platform technology for the oral administration of Roche’s antisense oligonucleotide platform (Press release, PureTech Health, JUL 20, 2018, View Source [SID1234536199]). Under the terms of the agreement, PureTech Health will receive up to $36 million, including upfront payments, research support, and early preclinical milestones. PureTech Health will be eligible to potentially receive development milestone payments of over $1 billion and additional sales milestones and royalties for an undisclosed number of products.

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PureTech’s milk exosome-based technology is uniquely designed to facilitate the oral administration of complex payloads such as nucleic acids, peptides, and small molecules. These exosomes are believed to traffic via lymphatic circulation and could potentially enable the targeting of immune cells in novel ways.

Daphne Zohar, Co-founder and Chief Executive Officer of PureTech Health, said: "We are excited to accelerate the development of this promising technology from our internal lymphatic and immune cell trafficking programmes. The expertise and resources that Roche is bringing to the collaboration will help us to potentially address one of the biggest challenges in oligonucleotide-based therapeutic development: oral administration of nucleic acids."

PureTech Health has been advancing internal research and development projects that focus on the Brain-Immune-Gut (BIG) Axis, with an emphasis on lymphatics and immune cell trafficking to modulate immunity in a tissue-specific manner. These internal pipeline programmes are being consolidated into a separate division of PureTech Health called Ariya. PureTech’s Internal division, which includes the milk-derived exosome technology, has generated compelling pre-clinical data and secured key intellectual property for its lymphatic and immune cell trafficking programmes.

About PureTech’s Milk Exosomes Technology

Milk exosomes represent a significant opportunity to potentially resolve the long-standing challenge of oral bioavailability of macromolecules and complex small molecules. Exosomes, which contain mixtures of lipids, proteins and nucleic acids, play a critical physiologic role in intercellular communication and the transport of macromolecules between cells and tissues. Mammalian-derived exosomes have attractive potential as vehicles for the administration of a variety of drug payloads, especially nucleic acids, since their natural composition will likely provide superior tolerability over the variety of synthetic polymers currently in use. Most sources of mammalian exosomes are not suitable or viable as vehicles for oral administration of drugs due to their lack of stability under the harsh physiologic conditions associated with transit through the stomach and small intestine; however, the milk-derived exosomes that form the basis for PureTech’s internally-developed technology have evolved naturally and specifically to accomplish the task of oral transport of complex biological molecules. The technology is based on research conducted by PureTech Health and its academic collaborators, including Ramesh Gupta, PhD, Agnes Brown Duggan Chair in Oncological Research at the James Graham Brown Cancer Center, and Professor in the Department of Pharmacology and Toxicology at University of Louisville, and exclusively licensed to PureTech Health.