Tucked away at the end of today’s presentation (Slide 51) is a mention of PYC’s “proof of concept” studies for a Lipid Nanoparticle + Cell Penetrating Peptide conjugate delivery system suitable for the delivery of RNA and DNA therapies. (Refer to PYC ann. Creation of a Novel Gene Therapy Delivery Technology, 8 September).
After earlier stumbles, RNA drug development appears to be alive and well, with focus very much on the delivery system.
Arrowhead Pharmaceuticals (ARWR) has just announced a collaboration with Takeda to co-develop and co-commercialize Arrowhead’s Phase 2 RNAi asset, ARO-AAT for Alpha-1 Antitrypsin-Associated Liver Disease. Under the terms of the deal, Arrowhead is eligible to receive up to US $1.04bn including an upfront payment of US$300m and potential development, regulatory and commercial milestones up to US$740m.
The deal with Takeda follows a US$3.7bn licence and collaboration deal forged between Arrowhead and Janssen in 2018, principally for a chronic hepatitis B infection drug candidate but also including an option for three new unidentified targets.
Previously in 2016, Arrowhead struck a deal with Amgen for RNAi therapies aimed at cardiovascular targets. Signed on the strength of a robust pre-clinical data package, the US$674m deal was the first for drug candidates using Arrowhead’s Targeted RNAi Molecule (TRiM) platform.
In December 2016, Arrowhead jettisoned its previous EX1 drug delivery technology (like TRiM, a GalNAc conjugate delivery platform) and all of its programs based on that technology after monkey deaths in a study prompted the FDA to put the company’s lead Phase 2 clinical trial on hold. The drastic action hit the company hard and pushed the company’s stock to a low of US$1.24. But the move to a next generation platform paid off for Arrowhead, which within just three years saw its stock price climb to US$73 and its market cap grow to over US$7 bn.
Arrowhead now has thirteen RNAi drugs in its pipeline, with eight at clinical stage.
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(20min delay)