Ann: PYC Investor Update Presentation, page-9

Following the Wall Street Journal article last week, which focused on the type of deals that Pfizer, Novartis and Merck are hunting for, is a Fierce Biotech article this week on GSK’s portfolio intentions.

Although GSK has decided to end its investment in cell and gene therapy, it’s doubling down on genetic targets, based on the fact that drugs based on genetically validated targets have a much higher chance of making it through to market.

One downside to this approach is that half of the novel potential targets identified through genetics can't be targeted with a small molecule or an antibody. GSK believes that the solution lies in oligonucleotides.

As previously mentioned on this forum and by Rohan Hockings, GSK paid an upfront of US$170m to Wave Life Sciences in December to access its oligonucleotide platform. GSK believes that Wave’s chemistry could broaden the use of subcutaneously delivered oligonucleotides beyond the liver, the target organ of most current oligo therapies.

Heading GSK’s oligonucleotide portfolio is bepirovirsen, which was licensed from Ionis in 2019 and is currently in Phase 3 trials as a potential “functional cure” for hepatitis B virus infection.

Bepirovirsen is followed by a drug candidate for nonalcoholic steatohepatitis (NASH), which was licensed from Arrowhead in late 2021 and is about to enter Phase 2.  Arrowhead was paid an upfront of US$120m and will receive an extra $30m on commencement of the Phase 2 trial. $100m will be paid if Phase 2 is successful and there’s another $780m on offer for sales and other milestone payments, with royalties payable as well. Arrowhead has kept the rights to develop and sell the drug in China (NASH is particularly high in China).

The Arrowhead CEO said that his company ran a competitive process for licensing  its NASH candidate and that there were multiple potential partners interested but that GSK was chosen as it had the infrastructure and capabilities best suited to work on and sell the drug.

GSK has made it clear that it hasn’t finished filling its oligonucleotide pipeline with the MD stating

“It's a really important area for us, where the technology of oligos fits so nicely with our expertise in genetics and novel targets, and we're going to be looking to optimize that. You can envision all sorts of business development deals, ranging from single asset acquisitions through to additional platform plays.”

https://www.fiercebiotech.com/biote...its-back-cell-therapies-oligio-strategy-heats

https://www.fiercebiotech.com/biote...continue-ride-oligonucleotide-wave-years-come

https://www.fiercebiotech.com/biote...ng-rnai-pact-target-arrowhead-pharmaceuticals