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From the preliminary S-1:
We are a biopharmaceutical company focused on the development and commercialization of recombinant human insulin-like growth factor-1, or rhIGF-1, for the treatment of short stature, diabetes and other endocrine system disorders. The endocrine system regulates metabolism through the use of hormones, including IGF-1. IGF-1 is a naturally occurring hormone that is necessary for normal human growth and metabolism. A deficiency of IGF-1 can result in short stature, which is characterized by children being shorter than approximately 97.5% of normal children, and can lead, in children and adults, to a range of other metabolic disorders. These metabolic disorders can include lipid abnormalities, decreased bone density, obesity and insulin resistance. We licensed Genentech, Inc.’s rights to develop, manufacture and commercialize rhIGF-1 for a broad range of indications, including for short stature worldwide and diabetes in the U.S. We have Phase III clinical trial data for rhIGF-1 for our first short stature indication, Severe Pediatric IGFD. We intend to submit a New Drug Application, or NDA, to the FDA by early 2005 after completing the transfer and validation of our rhIGF-1 manufacturing process to our contract manufacturer.
The cellular production of IGF-1 is regulated by growth hormone. Growth hormone deficiency, or GHD, leads to inadequate IGF-1 production which results in short stature in children. Growth hormone replacement therapy, which increases IGF-1 levels, can often be used to successfully treat GHD. However, we believe a significant number of individuals have normal growth hormone secretion, but because their cells do not respond normally to growth hormone, they are IGF-1 deficient and suffer from short stature. Individuals with this condition, which we refer to as IGFD, are candidates for rhIGF-1 replacement therapy. Our product candidate, rhIGF-1, is identical to naturally occurring human IGF-1 and performs the same functions in the body.
We have Phase III clinical trial results from the treatment of 65 children with Severe Pediatric IGFD with rhIGF-1 replacement therapy for an average of 3.5 years, with some patients being treated for as many as 10 years. None of the 65 patients discontinued rhIGF-1 treatment due to safety concerns. Of these children, 48 have completed at least one year of rhIGF-1 replacement therapy, which is the generally accepted length of time required to adequately measure growth responses to drug therapy. A highly statistically significant increase in growth rate from 2.6 cm per year prior to treatment to 8.0 cm per year after the first year of rhIGF-1 treatment was demonstrated in these patients (p<0.0001). Compared to pre-treatment growth rates, highly statistically significant increases were also observed during each of the next 5 years of rhIGF-1 treatment (p<0.001). We believe the magnitude of these increases in growth rate were clinically meaningful and comparable to those observed in clinical trials of other approved growth hormone treatments. Highly statistically significant increases in Height SDS were also observed during each of the first 6 years of rhIGF-1 treatment (p<0.0001).
We have had recent discussions with the FDA regarding the use of rhIGF-1 in Severe Pediatric IGFD. We believe that our clinical data supports the submission of an NDA for long-term rhIGF-1 replacement therapy in Severe Pediatric IGFD. We are currently planning a 250-patient Phase III rhIGF-1 clinical trial in children with Pediatric IGFD and plan to initiate additional late stage clinical trials for other indications.
We are currently transferring Genentech’s proprietary, high yield, commercial scale manufacturing process to our contract manufacturer. After the transfer has been completed and the manufacturing process has been validated, we intend to submit an NDA in Severe Pediatric IGFD in early 2005.
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