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Genzyme General Obtains Rights to Pompe Disease Therapy from Synpac

Posted on: April 20, 2000

Genzyme and Pharming to Fund Commercialization

Cambridge, Mass., USA, Durham, N.C., USA, and Leiden, the Netherlands, April 19, 2000 -

Genzyme General and Synpac (North Carolina) today announced an agreement under which Genzyme will obtain exclusive, worldwide rights to develop and commercialize Synpac’s Pompase™ enzyme replacement therapy for Pompe disease, a lethal, hereditary muscle disorder. In a partnership, Pharming and Genzyme announced their intention to share equally in the program and the funding for the commercialization of Pompase™.

Genzyme will make a $19.5 million initial payment to Synpac, and Pharming will issue a convertible note to Genzyme for an aggregate principal amount of $10 million. Other terms of the agreement were not disclosed. Genzyme and Pharming expect to initiate pivotal clinical trials of Pompase in the second half of this year.

Pompase is a recombinant form of the enzyme human alpha-Glucosidase produced in mammalian cell culture using Synpac’s CHO cell line licensed from Duke University. An initial clinical trial of the product was completed in 1999. Three infants with Pompe disease received treatment with Pompase for three months, and all patients remain on therapy. Results of the trial, which was conducted under the supervision of Dr. Y.T. Chen at Duke Medical Center, recently have been submitted for publication.

“These agreements are consistent with Genzyme’s, Pharming’s and Synpac’s commitment to develop the most effective treatment possible for patients with Pompe disease”, said Henri A. Termeer, chairman and chief executive officer of Genzyme Corp, “We believe Pompase holds great promise for these patients, and we will move rapidly to accelerate its development.’

Leslie Koo, chairman of Synpac, said:“Genzyme is a recognized leader in the development of therapies for rare genetic disorders. We believe that their expertise, commitment and substantial global resources provide the best opportunity to make this therapy available to patients with Pompe disease as quickly as possible.’

Pharming’s president and chief executive officer George J.M. Hersbach commented on the agreement: ‘For Pharming this is a unique opportunity to combine its technology with that of other fascinating developments, both scientifically and business wise. This will ensure that in the end, Pompe patients will receive the best therapy. Both Genzyme and Pharming will significantly strengthen their positions in the development of a therapy for this serious disease, and will continue to equally share costs and profits.”

Genzyme General and Pharming plan to equally fund the commercialization of Pompase and share in the program accordingly. The companies established a joint venture in 1998 to develop human alpha-Glucosidase as an enzyme replacement therapy for Pompe disease. In late 1999, Pharming completed a 36-week phase II clinical trial with transgenic human alpha-Glucosidase at the Sophia Children’s Hospital in Rotterdam, the Netherlands under the supervision of Dr. A.T. van der Ploeg, and the results have been submitted for publication. A phase II clinical trial evaluating the use of human alpha-Glucosidase in infants is ongoing in Essen, Germany. All patients currently on treatment with transgenic human alpha-Glucosidase will continue to receive this product.

Pompe disease is caused by the lack of alpha-Glucosidase activity, an enzyme involved in the breakdown of glycogen into glucose, a primary source of energy for the human body. Patients suffering from this disease show accumulation of glycogen in various tissues, resulting most prevalently in degradation of skeletal, heart and lung muscles. Pompe disease can manifest itself in early and late onset forms. Infants suffering from Pompe disease generally die before reaching 12 months of age. The clinical appearance of the disease varies from rapid progress and cardiac involvement in the infantile form to slow progress and severe respiratory complications in the later onset forms. Pompe disease affects an estimated 5,000 - 10,000 people in the developed world.

The agreement between Genzyme and Synpac is subject to clearance under the Hart-Scott-Rodino Anti-Trust Improvements Act and customary closing conditions. The agreement between Genzyme and Pharming is subject to receipt of corporate approvals.

Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has three therapeutic products on the market and a strong pipeline of products in development focused on the treatment of rare genetic diseases. A division of the biotechnology company Genzyme Corp., Genzyme General has its own common stock intended to reflect its value and track its economic performance.

Pharming Group N.V. focuses on the development, production and commercialization of human therapeutic proteins using the company’s proprietary technology. Pharming’s portfolio comprises products for (rare) genetic disorders, surgical and traumatic bleeding, infectious and inflammatory diseases, tissue and bone repair and blood-related disorders. The company has operations in Belgium, Finland, the Netherlands and the USA,

Synpac (North Carolina), Inc. is a drug development company located in Research Triangle Park.

Pharming
Health Care Products

Pharming B.V.
Niels Bohrweg 11-13
2333 CA Leiden
The Netherlands

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