Shire said today it will acquire Lotus Tissue Repair, the privately-held developer of the first and only protein replacement therapy now in preclinical development for the orphan disease dystrophic epidermolysis bullosa (DEB). 

The price was not disclosed, though Shire said it would make an up-front payment as well as payments tied to unspecified safety and development milestones.

Shire’s Human Genetic Therapies business will take over development of Lotus Tissue Repair’s lead product candidate, a recombinant form of human collagen Type VII (rC7) as an intravenous protein replacement therapy for DEB, believed to be prevalent in 1 in 300,000 live births, and its among the more severe versions of EB. No treatment other than palliative care exists for DEB, all forms of which are caused by mutations in COL7A1, the gene responsible for encoding C7.

Lotus Tissue Repair holds an exclusive license to intellectual property related to the manufacture and use of rC7 based on the work of company co-founders, Mei Chen, Ph.D., and David Woodley, M.D.

Founded in 2011, Lotus Tissue Repair closed that year on a $26 million in Series A financing round from venture capital firm Third Rock Investors. Lotus has also joined DEB Research Association (DEBRA) International and DEBRA of America to develop the first international patient-reported registry for EB,

The acquisition marks Shire’s latest effort to strengthen its rare disease effort. Just yesterday, Shire launched a new alliance with ethris aimed at developing RNA-based monoclonal antibody therapeutics for rare diseases.

Shire said the acquisition expands its commitment to treating EB, since its product pipeline includes ABH001, a regenerative medicine product under investigation as a skin substitute therapy for nonhealing wounds in EB patients.

“The acquisition actually has no negative consequences on ABH001. That’s moving ahead at full speed. It’s not being slowed down. We very clearly see these as being complementary in a number of ways,” Philip J. Vickers, Ph.D., senior vp and global head of research and development at Shire Human Genetic Therapies, told GEN.

Last month, Shire disclosed plans through ClinicalTrials.Gov for a Phase III pivotal trial to evaluate the efficacy and safety of ABH001 in patients with EB who have wounds that are not healing. The primary outcome measure will be reduction in wound surface area. Shire told GEN this week it is preparing to launch the Phase III trial, and expects to begin patient enrollment this quarter.

While ABH001 as a skin substitute is not as appropriate for the internal lesions of people with DEB as rC7, Shire HGT hopes it can be used in all forms of DEB.

“It’s possible to envision approaches where patients, for example, who have DEB, could be treated systemically with our product, and for some time perhaps have the lesions treated with the ABH001. That’s a possibility that we can look into,” Dr. Vickers said.  “We’re not focusing initially on a combination product. But we could imagine that you have a systemc approach like the product we’re aiming to bring forward, that we would hope would be useful for the external skin lesions as well as the internal lesions, but with a very different approach from ABH001. You could envision that there could be patients that would gain benefit from both of those therapies. It is possible that down the road, we would look into that.”

Shire HGT’s focus in developing rC7 is DEB, Dr. Vickers added: “But as we now start to use recombinant C7 and understand diferent forms of epidermolysis bullosa, we will be exploring whether or not recombinant C7 will have utility in these other forms of EB.”

Previous articleFat-Burning Fury Occurs in Absence of This Protein
Next articleEnd of an Era: Supreme Court Won’t Hear Stem Cell Case