- Swiss pharma Roche is putting more than $1 billion on the line to access a technology that could allow gene-regulating drugs to be administered via pill or capsule rather than injection.
- Through its latest deal, Roche will pay $36 million upfront to help advance PureTech Health’s milk-derived exosome platform. The platform aims to package up drugs with large or complex payloads in a way that allows them to move through the digestive system without degrading so they can reach the target cell or tissue.
- PureTech is also eligible to receive hundreds of millions of dollars in development milestones, as well as sales milestones and royalties on an undisclosed number of potential products. The collaboration's main goal is to use the exosomes to make Roche's antisense oligonucleotide platform available in an oral form.
Pharma's interest in gene-regulating therapies has only gotten stronger in recent years, spurred by the success of therapies like Alnylam Pharmaceuticals' patisiran. Japan's Takeda Pharmaceutical is one example, entering into a potentially more than $2 billion deal with Wave Life Sciences earlier this year that focuses on nucleic acid therapies for central nervous system disorders.
Roche has been active in the space as well, acquiring Santaris Pharma and its Locked Nucleic Acid platform in 2014 for $250 million upfront, and then in November licensing Ionis Pharmaceuticals' antisense drug for Huntington's disease. The Swiss pharma also has in its pipeline a messenger RNA-based cancer vaccine.
A common problem Roche and other developers in the space are facing, however, is that it's quite difficult to administer these therapies outside of an injection because they often target a specific tissue or region of the body and need to arrive at those locations without being chewed up by the stomach or intestines.
Additionally, patients more often than not prefer oral medications over needles. Creating a gene-editing pill or tablet would therefore give the manufacturer a leg up on competitors — something that will become increasingly important as the market grows more crowded.
That's where PureTech and its platform would ideally provide solutions.
"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," CEO Daphne Zohar said in a July 20 statement.
The resources Zohar mentioned will be particularly important for PureTech, which reported an operating loss of $115 million last year. Collaborations have been a key strategic play for the company, with 20 inked between 2014 and the end 2017. Roche, along with its $36 million upfront payment, surely comes as a welcome addition.