Roche, PureTech team up to develop oral antisense oligonucleotides

Roche has struck a deal with PureTech Health to develop oral antisense oligonucleotides. The pact gives Roche access to PureTech’s milk-derived exosome technology in return for up to $36 million (€31 million) in near-term payments and milestones that could top out above $1 billion.

PureTech licensed the exosome technology from 3P Biotechnologies last year to gain a way to orally deliver biologics, nucleic acids and complex small molecules. The exosomes are designed to encase drugs and protect them from the environment in the gastrointestinal tract, thereby enabling the oral delivery of non-orally bioavailable molecules. Other researchers have explored the use of exosomes for this task, but PureTech thinks its delivery vehicles are more stable than potential competitors.

Roche has bought into the idea. The Swiss pharma will hand over up to $36 million in upfront fees, early preclinical milestones and research payments to get the project going. As products based on the exosome platform progress, Roche could pay out as much as $1 billion in milestones.

The numbers reflect the early-stage nature of the work and the size of the opportunities it could lead to. Antisense oligonucleotides have started to realize their potential to treat a range of diseases by reducing, restoring or otherwise modifying the expression of proteins. But getting molecules to their target sites remains challenging. Sanofi and Ionis’ Kynamro became the first systemically delivered antisense oligonucleotide to win approval in 2016 but oral administration remains an aspiration.

With Roche on board, PureTech thinks it may be able to realize that aspiration.

"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 CEO Daphne Zohar said in a statement.

PureTech unveiled the Roche deal alongside news of internal structural changes. The Boston-based, London-listed company is hiving off its projects focused on the gut-brain axis into a separate division called Ariya.