Shire expands orphan disease repertoire with Lotus acquisition

Transaction nets Shire a product candidate for treating DEB

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DUBLIN, Ireland—Specialty pharmaceutical company Shire plchas announced the signing of an agreement with Lotus Tissue Repair, Inc., underwhich it will acquire the Cambridge, Mass.-based biotechnology company. LotusTissue Repair, a privately held organization, is working on development of thefirst and only protein replacement therapy currently under investigation forthe treatment of dystrophic epidermolysis bullosa (DEB). Though specificfinancial details for the transaction were not disclosed, Shire will acquireLotus Tissue Repair for an upfront payment as well as additional paymentscontingent upon the successful achievement of certain safety and developmentmilestones.
"This acquisition of Lotus Tissue Repair by Shire furtherunderscores the potential of this proprietary rC7 technology to dramaticallychange the treatment landscape for DEB patients and their families," Dr. Markde Souza, founding CEO of Lotus Tissue Repair, said in a press release. "We arethrilled that this program will become part of the innovative pipeline at Shireand the company's growing commitment to this patient community."
Epidermolysis bullosa is a set of rare genetic diseasescharacterized by extremely fragile skin and recurring blister formation as aresult of even minor friction or trauma. DEB is one of the more severedisorders that fall within the disease set, and severe cases of DEB can includeinternal blistering of the mouth, esophagus, upper airway, lowergastrointestinal tract and GU tract. Current incidence estimates are that thedisease occurs in roughly one out of every 300,000 live births.
The recessive form of the disease is extremely painful andpresents with severe blistering and mutilating scarring of hands, feet andjoints. By the time a patient with DEB reaches their 20s or 30s, the diseaseleads to the development of aggressive squamous cell carcinomas in areas ofchronic wounding, which often metastasize and lead to death. There is currentlyno approved treatment for DEB other than palliative care.
"DEB is one the most devastating orphan diseases, severelyimpacting the lives of patients and their families, many of whom have few or notreatment options other than palliative care," Dr. Philip J. Vickers, globalhead of Research and Development, Shire Human Genetic Therapies, said in astatement. "rC7 protein replacement therapy has the potential to provide afirst-in-class disease-modifying treatment for these children. We plan to applyour proven ability to develop protein replacement therapies for rare geneticdiseases to progress rC7 as a possible groundbreaking treatment that offershope to patients with DEB."
Lotus Tissue Repair's lead product candidate is aproprietary recombinant form of human collagen Type VII (rC7), an intravenousprotein replacement therapy for treating DEB. The product is in latepreclinical development, and Shire's Human Genetic Therapies business will bemoving forward with further development of the product candidate. The therapystands to directly address deficiency or dysfunction of C7, a primary cause ofDEB. In preclinical models, rC7 as a protein replacement therapy has shown tobe potent and long-lasting, and is retained in the epidermis and other affectedtissues following intravenous injection.
SOURCE: Shire plc press release

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