Orphan drug specialist in $135M deal with Shire

Sale

Zymenex A/S of Denmark has validated its technology platform with the sale of its arylsulfatase A (ASA) enzyme replacement therapy for treating metachromatic leukodystrophy (MLD) to the UK pharmaceutical company Shire plc for $135 million.

The product is currently in Phase II trials in Europe and has orphan drug designation in both Europe and the US. MLD, a lysosomal storage disorder, is a serious, life-limiting disease in which patients experience progressive irreversible neurological damage. It 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 about 2,000 MLD patients in the developed world.

““This deal gives Zymenex a validation of the development of treatment options for other life-threatening diseases,”” said CEO Jens Fogh. The company has other similar projects in its pipeline, and the $135 million will transform the prospects of the 18-strong company.

Zymenex is owned by the Danish VC investors BankInvest Biomedical Venture and Sunstone Capital. ““Zymenex has from the very beginning focused on orphan drugs for lysosomal storage disorders. Market focus and high quality research in all aspects of the development process are the basis for the company’s success,”” claimed Sten Verland from Sunstone Capital, who emphasised that Zymenex has a strong pipeline with the potential to develop further successful enzyme replacement therapies.

MLD is one of 45 diseases within the family of lysosomal storage diseases. The ASA product, 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 approval for its Investigational New Drug application to initiate a US Phase II clinical trial.

Shire will pay Zymenex $135 million for global rights, and Zymenex will also provide certain transition services, including knowledge transfer, for up to six months after completion. There are no royalties or other downstream payment obligations.