Amicus spins out gene therapy unit in SPAC deal as it pivots efforts toward Pompe disease program
A rare disease biotech out of Philadelphia is spinning out its gene therapy unit to reverse merge with a blue-chip SPAC.
Amicus Therapeutics will send off its gene therapy component in a new company called Caritas Therapeutics, which will partner with Perceptive’s fourth blank-check outfit and head to Nasdaq, the biotech announced Wednesday morning. Caritas will get about $400 million overall in the deal, including approximately $150 million held in ARYA Sciences Acquisition Corp IV, a $200 million PIPE investment and a $50 million cash investment from Amicus.
“This is a big, bold vision and a massive step forward for next generation biotechnologies for people worldwide living with some of the most devastating rare diseases,” Amicus CEO John Crowley said in a statement. “In a single stroke with the formation and funding of Caritas we will create what will be one of the world’s preeminent next-generation genetic medicines companies.”
The deal is expected to close sometime in the fourth quarter this year, or early next year.
As Caritas gets things going, Amicus will focus on commercializing its Galafold drug for Fabry disease, as well as pushing forward on a label expansion. The biotech is also redoubling its efforts to get its Pompe disease candidate, dubbed AT-GAA, approved and hopes to roll it out globally. On Wednesday, the FDA accepted Amicus’ BLA and NDA for both components of the program, setting PDUFAs dates of May 29, 2022 and July 29, 2022, respectively.
The Pompe disease program will likely run into headwinds with regulators, however, as a Phase III study showed AT-GAA did not pass a head-to-head test against Sanofi’s Lumizyme in the 6-minute walk test, the gold standard for demonstrating efficacy. Researchers saw a p-value of p=0.072 in patients who were both switched from ERT therapy or ERT naïve.
When looking at those populations separately, AT-GAA did hit nominal statistical significance in those who originally switched from Lumizyme at a p-value of p=0.046. But in patients who had never received treatment before, AT-GAA landed with a thud, failing to show superiority on the walk test and hitting a p-value of p=0.57 on forced vital capacity.
Caritas will kick things off with a pipeline of two clinical programs in Batten disease, with the company saying it’s achieved proof of concept in CLN6 and CLN3. There will also be six active preclinical candidates spanning a wide range of rare genetic diseases like Fabry disease, Pompe disease and CDKL5 deficiency disorder.
The spinout is also getting exclusive global rights for about roughly 50 more diseases through a collaboration with gene editing pioneer Jim Wilson and his team at the University of Pennsylvania, the announcement said. Caritas will start with a team of about 115 employees, indicating Amicus expects the new biotech to hit the ground running.
Amicus will be the largest shareholder in Caritas once the SPAC deal closes, owning about 36% of shares. It will also retain co-development and commercialization rights to the Fabry and Pompe gene therapy programs.
In conjunction with the spinout, Amicus also raised $200 million in a private placement from Redmile Group, Avoro Capital Advisors, Perceptive Advisors, Invus, Sphera Healthcare, and Janus Henderson Investors.