Amicus spins off gene therapy unit in $600M SPAC deal as CEO becomes chief at Caritas

 Amicus has endured a tough few years of pipeline flops and a roller coaster stock ride, but now it’s riding the special purpose acquisition company wave, spinning off its gene therapy unit in a $600 million deal.

The pact sees Amicus’ gene therapy business bought out by ARYA Sciences Acquisition Corp IV, a SPAC sponsored by Perceptive Advisors. Caritas gets around $400 million in funding to start with, while Amicus adds about $200 million in private funding from “leading biotechnology investors.”

While being spun out, it still remains on a string for Amicus: The biotech will become the largest shareholder in Caritas with around a 36% stake as current John Crowley will lead Caritas as chairman and CEO. In his place, Amicus’ president and chief operating officer Bradley Campbell climbs up the ladder to chief of the biotech.

Caritas, from the Latin word for compassion, will focus on gene therapies, with much of the earlier pipeline from its 2018 $100 million buyout of Celenex. The pipeline is led by two Batten disease programs with clinical proof of concept in CLN6 and CLN3, which it snapped up in that deal three years ago.

It also has six active preclinical programs tackling targets including Fabry disease, Pompe disease and CDKL5 deficiency disorder. Through a collab with Jim Wilson, M.D., Ph.D., and a University of Pennsylvania team, it will also help work on broad range of early rare diseases including Angelman syndrome, Duchenne muscular dystrophy, Rett syndrome, myotonic dystrophy and select other muscular dystrophies. Wilson also becomes a senior scientific and strategic adviser to Caritas.

"This transaction will transform Amicus into a premier rare disease global commercialization and late stage product development company that we believe will benefit all of our stakeholders," said Campbell.

"The separation strengthens the financial profile of Amicus and accelerates our path to profitability, while preserving significant equity ownership in the gene therapy pipeline and commercial rights to the innovative and important Fabry and Pompe gene therapy programs.

“We will be laser focused on maintaining the growth of Galafold and executing on the anticipated global launch of AT-GAA, as we build Amicus into a leading global rare disease biotechnology company and bring our medicines to as many patients as quickly as possible."

This comes after a series of setbacks from the pipeline. Back in February, a phase 3 clinical trial of Amicus’ late-onset Pompe disease prospect missed its primary endpoint.

Yet Amicus looked past the failure of AT-GAA to improve statistically on Sanofi’s market incumbent Lumizyme, zeroing in on details of the data to justify forging ahead with plans to seek approval of the candidate.

And, back in 2017, a drug for rare skin disease epidermolysis bullosa that Amicus spent $847 million on ago failed a key phase 3 trial, throwing a lot of money down the drain.

Things are, however, looking up in Pompe disease: Alongside the SPAC deal, Amicus also announced it has been given FDA standard reviews for its Pompe hopefuls. The biologics license application for cipaglucosidase alfa and the new drug application for the already approved Galafold (miglustat) for AT-GAA, the company’s investigational two-component therapy for the treatment of Pompe disease, have both been handed reviews, with the BLA set for an end of July decision and the NDA for end of May next year.

https://www.fiercebiotech.com/biotech/amicus-spins-off-gene-therapy-unit-600m-spac-deal-as-ceo-moves-over-to-caritas-therapeutics