Last week, Arrowhead Pharmaceuticals submitted an NDA for its lead drug program, plozisiran for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease which currently has no approved treatments in the U.S. Plozisiran is an RNAi therapy which is dosed subcutaneously every quarter.
Arrowhead uses its platform TRiM technology to develop targeted RNAi therapeutics for genetic diseases caused by excessive protein production (whereas PYC is developing targeted therapeutics for genetic diseases in which there is insufficient protein production).
Yesterday Arrowhead announced a major global licence and collaboration deal with leading antisense pharma, Sarepta Therapeutics.
Sarepta has obtained an exclusive global licence to four clinical-stage and three preclinical-stage programs in muscle, central nervous system, and rare pulmonary disorders. The four clinical programs are all currently at Phase 1/2 stage. In addition, Sarepta and Arrowhead have entered into a discovery partnership pursuant to which Sarepta will nominate, and Arrowhead will deliver, IND-ready constructs for six targets across skeletal muscle, cardiac, and CNS.
Arrowhead will be paid a US$500m upfront. Sarepta will also make a US$325m equity investment in Arrowhead, priced at a 35% premium to the 30-day VWAP prior to the announcement of the agreement. In addition, Sarepta will pay Arrowhead US$250m, paid as annual instalments of US$50m over the next five years, and a further US$350m in near-term milestones. The icing on the cake comes in the form of up to US$10bn in future milestones and royalties on sales.
Sarepta's CEO will also take a Director position on the Arrowhead Board.
As detailed in past posts (see last 2 links below), Arrowhead has been successful in building an extensive pipeline of drugs through licensing and collaboration deals. These include previous deals with Amgen in 2016, Janssen in 2018 and Horizon in 2021.
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