Leiden, the Netherlands/Cambridge, Mass., October 14, 1998—Pharming Group N.V. (Easdaq: PHAR), the Netherlands biopharmaceutical company, and the American biotechnology company Genzyme General (Nasdaq: GENZ), has created a joint venture to develop and commercialize worldwide the enzyme human alpha-glucosidase as a treatment for Pompe’s disease, a lethal lysosomal storage disorder.
Based on the results of Pharming’s phase I clinical trial conducted to assess safety, tolerability and pharmacokinetics of human alpha-glucosidase in healthy volunteers, Genzyme and Pharming expect to commence phase II/III clinical trials by the end of 1998. In September 1996, Pharming received orphan drug designation for human alpha-glucosidase by the US Food and Drug Administration (FDA), potentially giving the company a seven year market exclusivity in the US following FDA approval.
Under the joint venture agreement, Genzyme will pay the first US $14 million of costs of the product development program. Thereafter, costs and revenues will be 50/50 shred. Upon first approval by the FDA of human alpha-glucosidase, Genzyme will make an additional payment to Pharming of US $7 million. A management team consisting of an equal number of representatives from both companies will manage the joint venture. In July of this year Genzyme also made an equity investment in Pharming of US $14 million.
“The new agreement further validates Pharming’s technology,” says George J.M. Hersbach, president and chief executive officer of Pharming. “The joint venture links Genzyme’s marketing experience to Pharming’s proprietary technologies and product development capabilities and will enable us to accelerate the process of bringing a therapy for Pompe’s disease to market.” Pharming has additional products for genetic disorders in the pipeline, including products for other lysosomal storage disorders.
Pompe’s disease is one of a family of 40 rare diseases known as lysosomal storage disorders. Genzyme’s Cerezyme® and Ceredase® enzyme replacement therapies are the only effective treatments for Gaucher’s disease, one of these disorders. Cerezyme and Ceredase—Genzyme’s lead products, had combined sales in 1997 of US $333 million.
When the two companies first announced their intention to form a joint venture in July this year, Henri A. Termeer, president and chief executive officer of Genzyme Corp, said that Genzyme is convinced that transgenic technology holds great potential for producing proteins to treat enzyme deficiencies underlying lysosomal storage disorders. Termeer further expressed Genzyme’s great enthusiasm about working with Pharming to develop a potentially life-saving treatment.
Pompe’s disease, also known as acid maltase deficiency or glycogen storage disorder type II, is caused by a complete or partial deficiency of the enzyme human alpha-glucosidase. The disease results in a build up of glycogen in various muscles and organs of the body, leading to fatal muscle degeneration. Pharming and Genzyme General believe administration of human alpha-glucosidase to patients suffering from Pompe’s disease could alleviate or eliminate symptoms.
Under the program of the joint venture, collaborations with patient organizations and clinicians will be continued and intensified. In the USA, the Acid Maltase Deficiency Association (AMDA)* represents all US patients suffering from Pompe’s disease. AMDA offers support and information services to patients and their relative and also maintains a patient registry which will be used to notify patients and their physicians of approaching therapy. The Netherlands Association for Neuromuscular Disease* in the Netherlands (VSN) and the Association for Glycogen Storage Disease* in the United Kingdom are equally involved and also maintain registries for patients in their countries. Similar organizations that have been established in other countries may also be involved. Pompe’s disease affects an estimated 5,000-10,000 people in the Western world.
Genzyme recently formed a joint venture with BioMarin Pharmaceutical, Inc., to develop alpha-L-iduronidase as a treatment for Hurler’s syndrome, another lysosomal storage disorder. In addition, Genzyme is conducting a phase I/II clinical trial of alpha-galactosidase as a treatment for Fabry’s disease. The company is also investigating gene therapy approaches to treating lysosomal storage disorders.
Pharming focuses on the development , production and worldwide commercialization of human therapeutic proteins, produced at high levels in the milk of transgenic animals that have been created using the company’s proprietary technology.