US$158m from a Priority Review Voucher sale is certainly a big jump in value – the last sale was in May for US$108m.
I note that in announcing the sale, the Chief Business Officer at French pharma, Ipsen, stated
The sale of the PRV generates further firepower for our ongoing medicine launches and external-innovation strategy, which has seen multiple additions to our portfolio at every stage of development in 2024.
Ipsen is a US$10 bn market cap company with 11 approved products and a direct commercial presence in more than 30 countries. It focuses on just three therapeutic areas, two of which are rare disease and neurology.
In mid-2021, Ipsen announced a collaborative research, development and commercialization partnership with Exicure, a clinical-stage biotech, to create new oligonucleotide therapies for Angelman syndrome (AS) and Huntington’s disease (HD). The therapies were to use Exicure’s Spherical Nucleic Acids (SNAs) which are proposed to enhance cell penetration, biodistribution and organ persistence properties.
Exicure received $US 20m upfront from Ipsen for the exclusive option to license the SNA-based treatments arising from the partnership, with Exicure to be responsible for the discovery and certain pre-clinical development activities. If Ipsen decided to exercise its option to license, it was to be responsible for further development and commercialization. Aside from the $20 million upfront, Exicure was eligible to receive as much as $1 billion in option exercise fees and milestone payments.
Ipsen chose to terminate the agreement in December 2022 but retained the right to re-license the assets at a later date if desired.
Since then, two oligonucleotide drug candidates for Angelman syndrome, developed by Ultragenyx and Ionis, have successfully passed Phase 2 trials and will imminently commence pivotal trials. Neuren has also successfully passed Phase 2 with its oral drug in AS and Roche is currently conducting a Phase 2 trial as well.
Meanwhile, PYC has been working on a Phelan McDermid syndrome drug candidate for over 2 years now. Two different chemistries/mechanisms of action have been optimised in parallel – one a PPMO and the other a naked RNA drug. The preferred candidate is to be announced before the end of this year.
I note that there are fewer therapies in clinical development for Phelan McDermid syndrome than for Angelman syndrome.
With US$158m firepower to spend on external innovation, a pipeline hole left by the terminated Exicure deal, as well as an obvious interest in differentiated neurological rare disease assets, Ipsen could well be interested in PYC’s preclinical Phelan McDermid syndrome candidate.
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