Excerpt From Genzyme’s April 17, 2002 Press Release
Pompe Update
Genzyme is moving forward aggressively with its program to develop an effective therapy for Pompe disease. Genzyme has invested significant resources over the past four years pursuing the most promising avenues of Pompe research, including the transgenic enzyme developed in a joint venture with Pharming Group N.V. that began in 1998; the CHO enzyme licensed from Synpac in 2000; the enzyme obtained in the Novazyme acquisition in 2001; and the internally produced CHO enzyme that it began developing last year. Each of these programs has made a major contribution to Genzyme’s understanding of this disease.
To accelerate the progression to regulatory approval for multiple Pompe disease indications, Genzyme recently conducted a comprehensive, blinded pre-clinical analysis comparing all four Pompe enzymes. The analysis showed that Genzyme’s internally developed CHO product, when compared with the Synpac enzyme, provided a similarly robust response profile in terms of glycogen clearance. Due to the significantly greater production yields of the Genzyme CHO enzyme, it offers the clearest and most efficient pathway to commercialization based on both clinical and manufacturing considerations. Genzyme expects to meet soon with U.S. and European regulatory authorities to outline a plan for the clinical development of this product, and it expects to initiate trials in infantile and delayed-onset Pompe patients in the second half of this year. Genzyme plans to initiate pivotal trials for this product by the end of the year.
John Crowley, senior vice president of Genzyme Therapeutics and general manager of Genzyme’s Pompe programs, said: “The robust response associated with CHO enzyme therapy we’ve seen in clinical trials to date has been encouraging. This shift to the internal Genzyme CHO product will help ensure timely availability of significant drug supply for clinical and commercial use. Importantly, by producing this product entirely at our own facilities, Genzyme will be able to leverage its expertise in large scale manufacturing
of enzyme replacement therapies.”
As a result of this decision, Genzyme will not proceed with further clinical development of the CHO therapy licensed from Synpac. Genzyme will continue to supply product to patients participating in the extensions of clinical trials of this product, as it has with patients receiving the transgenic enzyme, until they can be transitioned to the Genzyme CHO product. Genzyme will proceed with the pre-clinical development of Novazyme’s NZ-1001 as a potential next-generation therapy for Pompe disease. Novazyme’s novel carbohydrate engineering technologies-intended to enhance the targeting and uptake of replacement enzymes-will continue to serve as a central part of Genzyme’s efforts to develop improved second-generation versions of its marketed products and optimal products for the treatment of other lysosomal storage disorders.