Krystal Biotech has completed the construction of its new manufacturing facility Ancoris, which will support the clinical and commercial development of KB103, its lead product candidate for the treatment of dystrophic epidermolysis bullosa (DEB).
Ancoris was designed to meet all projected commercial demands of KB103. Located near the company’s headquarters in Pittsburgh, the 4,500-square-foot facility will house state-of-the-art equipment to fulfil the requirements of Good Manufacturing Practice (GMP) standards governing commercial production of products for biopharmaceutical use.
Krystal anticipates that the facility will officially open during the first quarter of 2019, following completion of the first engineering run.
“Our development of internal manufacturing capabilities bolsters our position for commercial readiness as we execute on our vision to bring our therapies to the patient communities in need,” Krish Krishnan, chairman and CEO of Krystal, said in a press release. “We thank the City of Pittsburgh and the Mayor’s office for helping us complete construction in a timely manner and we intend to have a formal inauguration following completion of a trial run.”
The company is planning to build another GMP manufacturing facility in Pittsburgh, expected to be complete in 2020. This larger facility will support the anticipated commercial demands of other products currently under development.
“Having our own GMP facility will enable our STAR-D HSV-1 based platform to be fully integrated and allow us to keep important proprietary process development and associated intellectual property in-house,” said Suma Krishnan, Krystal’s founder and COO. “Ancoris and the second GMP facility we have planned will ensure that we are able to meet the manufacturing demands of all our research and potential commercial programs.”
KB103, developed using Krystal’s STAR-D platform, is a gene therapy candidate that delivers a functional Col7A1 gene directly to skin cells using a modified and safe herpes simplex virus (HSV-1). Mutations in the COL7A1 gene are the underlying cause of DEB, and lead to a defective production of COL7 protein, causing layers of the skin to separate and form skin blisters.
The U.S. Food and Drug Administration granted fast-track designation to KB103 for the treatment of DEB in May 2018. This is expected to expedite and support the development, regulatory review, and approval of KB103.
The investigational treatment is being studied in the GEM-1 Phase 2 trial (NCT03536143) in two children (older than 4 years) and two adults (older than 17) with the recessive form of DEB. Results from the trial are expected in the first half of 2019.
Preliminary data from the two adults, 35 and 28 years old, revealed that topical treatment with KB103 promoted skin wound closure within two weeks, compared with 10 weeks for untreated wounds, and those effects were sustained for more than 3.5 months. No safety issues were reported.