Glycogen Storage Disease Type II

Pictured: Astellas headquarters for the Americas in Northbrook, Illinois iStock/JHVEPhoto Astellas is putting the kibosh on a potential $340 million licensing deal it inked last year with Cartesian Therapeutics to develop a treatment for Pompe disease, according to an SEC filing on Thursday. Cartesian said in the filing that it received notification from Astellas stopping the license and development agreement from 2023. The termination will become effective on June 6, 2024. After the termination, Astellas will have no financial commitments or liabilities related to its Xork product candidate. Last year, Cartesian—which merged with Selecta Biosciences—granted Astrellas an exclusive license to its IdeXork tech. From this came the candidate dubbed Xork, an immunoglobulin G protease intended to be developed and commercialized to treat Pompe disease in combination with Astellas’s AT845 candidate, an AAV candidate. Astellas paid $10 million upfront with Cartesian potentially eligible to receive up to $340 million in milestone payments, sales and tiered royalties. The January 2023 deal was originally with Selecta, which merged with Cartesian in November 2023. The SEC document said Cartesian did not incur any early termination penalties due to Astellas’ move. According to the deal announcement last year, Selecta was responsible for the development and manufacturing of Zork and would maintain the right to develop other indications beyond Pompe. “Xork has the potential to expand access to life-changing gene therapies by addressing pre-existing immunity to AAV. Most other IgG proteases in development are derived from common human pathogens, and as a result, there is a high prevalence of pre-existing antibodies against these proteases that can restrict their use,” Selecta CEO Carsten Brunn said at the time. “Xork is differentiated by its low cross-reactivity to pre-existing antibodies in human serum.” The Selecta merger with Cartesian last year included a $60.25 million private financing led by a member of Selecta’s board. The combined company was expected to have over $110 million on hand, which was meant to support the development of the Cartesian pipeline through Phase III of its Descartes-08 T-cell therapy for treating myasthenia gravis. Before the merger last year, Selecta was froze investments in its pipeline and decided to focus its efforts on a candidate for chronic refractory gout. This came as Selecta saw slipping cash flows. Astellas entered into a research and licensing deal with Kelonia Therapeutics in February 2024 to develop new cancer therapies, paying $40 million upfront and $800 million up for grabs for Kelonia in potential milestone payments. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.

Astellas is backing out of a deal for Cartesian's Xork asset 14 months after first signing on. Astellas is putting the kibosh on a licensing deal with Cartesian Therapeutics, deciding it’s no longer interested in using the latter’s Xork asset to help treat Pompe disease.  The decision disclosed Thursday swiftly ends a collaboration announced just over a year ago. The January 2023 deal was technically with Selecta Biosciences, which has since merged with Cartesian.  Astellas paid $10 million upfront to use Xork in conjunction with one of its own gene therapies, AT845. Astellas also offered $340 million in biobucks should the now-terminated collab prove fruitful. The hope was that Xork, a preclinical immunoglobulin protease, would help patients who were otherwise ineligible for AAV-based gene therapies.  But once Selecta merged with Cartesian in November 2023, the focus shifted to the latter’s autoimmune cell therapies, namely Descartes-08, which is in clinical trials to treat myasthenia gravis and lupus, among other conditions. Selecta was teetering in the months leading up to the companies' fusion, laying off 25% of employees in May. Xork survived a subsequent pipeline narrowing, thanks to Astellas' name attached to the program.  Cartesian didn't mention its plans for Xork when the merger was announced and said it didn't incur any early termination penalties following the axed Astellas collab.  Business development has, as of late, been one of Astellas’ favored routes to bolster its pipeline. Most recently, the company’s subsidiary, Xyphos, signed a deal with Kelonia to develop novel immuno-oncology cell therapies. A few months earlier, Xyphos linked up with Phenomic AI to work on an antibody targeting tumor stroma. And in early December, Astellas paid $37 million upfront to Elpiscience Biopharma for its bispecific macrophage engagers. 

Astellas has terminated a licensing pact worth up to $350 million for Cartesian Therapeutics' IgG protease candidate Xork, just over a year after the deal was first announced, the autoimmune disease biotech disclosed Thursday in a US securities filing.The collaboration, inked with Selecta Biosciences in January 2023 prior to its merger with Cartesian, centred on developing Xork with Astellas' experimental adeno-associated virus (AAV)-based gene replacement therapy AT845. The gene therapy, also known as zocaglusagene nuzaparvovec, is currently in early-stage testing for late-onset Pompe disease after the FDA lifted a clinical hold on the treatment last year.The deal gave Astellas exclusive rights to pair Xork with AT845 as a potential pre-treatment to help patients who couldn't receive AAV gene therapies due to pre-existing antibodies against AAV capsids. Astellas paid $10 million upfront and committed up to $340 million more in milestones, plus tiered royalties ranging from low- to high-single digits on commercial sales where Xork is used with AT845.At the time the deal was announced, Selecta CEO Carsten Brunn suggested that Xork differed from most other IgG proteases in development "by its low cross-reactivity to pre-existing antibodies in human serum."However, Selecta ran into business difficulties, announcing in May that it would lay off a quarter of its staff following a review of its portfolio and capital resources. It eventually suspended most of its pipeline to focus on a gout drug. Selecta merged with Cartesian in November with a focus on RNA cell therapies to treat autoimmune disease.Cartesian noted in the SEC filing Thursday that it did not incur any early termination penalties as a result of Astellas' termination of the agreement.