Shire plc, the global specialty biopharmaceutical company, has acquired arylsulfatase -A (ASA) an enzyme replacement therapy (ERT) in phase I-II clinical trials for Metachromatic Leukodystrophy (MLD) from the Danish company Zymenex A/S (Zymenex).
MLD is a serious, life-limiting disease in which patients experience progressive irreversible neurological damage. MLD is caused by a deficiency in the enzyme ASA which causes an excess concentration of sulphatide in cells and an ensuing breakdown of myelin. There are approximately 2,000 MLD patients in developed world markets.
The newly acquired ASA product, currently known as Metazym, has completed a phase Ib clinical trial in 12 MLD patients in Europe and an extension to this study is ongoing. The product has received Food and Drug Administration (FDA) approval for its Investigational New Drug (IND) application to initiate a phase II clinical trial and has been granted Orphan Drug Designation in the United States and in the European Union.
Sylvie Grégoire, president, Human Genetic Therapies business, Shire, said, "This product fits very well with Shire's ERT portfolio of treatments for Lysosomal Storage Disorders (LSD). Shire HGT has been committed to MLD and by acquiring this mid-stage clinical program we hope to bring a MLD treatment to patients two years earlier than anticipated."
Shire is making a payment to Zymenex of US$135 million for the acquisition of global rights to the product upon completion of the transaction, which is conditional upon the receipt of customary consents. Zymenex is also providing certain transition services, including know-how transfer, for up to six months after completion. The transaction includes no royalties or other downstream payment obligations.