Astellas is set to restart trials for its age-related macular degeneration (AMD) program ASP7317 after improving production and analytical process for the embryonic stem cell-derived candidate.
Astellas added ASP7317 to its pipeline through the acquisition of Ocata Therapeutics in 2015. The cell therapy, derived from pluripotent human stem cells, is being investigated as a potential treatment for people with dry AMD.
But a Phase I trial enrolment was voluntarily put on hold by Astellas last year “due to the time required to study the manufacturing process, quality testing, and establishment of quality standard, as well as to provide order to the FDA,” Astellas CFO Minoru Kikuoka said during his firm’s Q2 2022 results call late last month.
However, trial recruitment is set to start imminently, he said, after the firm changed the manufacturing process for the candidate to improve the ratio of cells with desired characteristics and introduced “cutting-edge” analytical methods for quality testing with a high sensitivity, reproducibility, and specification setting based upon the virus preclinical data and rationale.
“Through these activities, we have established capabilities enabling supply of cells that meet high quality standards.”
With these necessary steps completed, subject screening is anticipated to resume in August, Kikuoka added. “In some cases, similar issues were identified orally in other cell therapy programs leading to smooth responses. We hope that the capability established this time will be utilized in subsequent programs to accelerate the research and development of cell therapy.”
Astellas has built up its cell therapy division through the acquisitions of several small biotechs – Mitobridge and Potenza Therapeutics, along with Ocata – and investment in both native Japan and elsewhere, including a $256 million manufacturing expansion in 2018 and an incubator site in Massachusetts in 2019.
Gene therapy ambitions
The firm, meanwhile, dove fully into the gene therapy side of CGTs in 2019 through the $3 billion acquisition of Audentes. It has sinced invested heavily in its manufacturing network, including a $109 million 135,000 square-foot gene therapy plant in Sanford, North Carolina, which came online during the past quarter.
“This state-of-the-art facility builds on our existing manufacturing network in Tsukuba, Japan, and South San Francisco, California, and will serve as a major driver of our pipeline, partnerships, and technology,” Mathew Pletcher, division head of Gene Therapy Research and Technical Operations at Astellas Gene Therapies, said in June.
“This new facility is a key enabler of our mission to develop safe, effective, and transformative gene therapies as swiftly as possible. As such, Sanford’s manufacturing capabilities will allow us to produce materials for multiple programs in parallel as opposed to in sequence and offer commercial scale manufacturing ability to any future approved therapies. This is critical to rapidly advance our programs and drive the next phase of growth for Astellas”