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Celera, Ipsen to Develop Biomarkers, Molecular Diagnostics for Short Stature

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Celera and Ipsen have entered into a research agreement to develop biomarkers and pharmacogenomic tests for growth failure in patients.
 
Celera brings to the table its genotyping know-how, while Ipsen offers rights to Genentech’s growth hormone Nutropin and Tercica’s IGF-1 drug Increlex.
 
The first phase of the collaboration will focus on discovering and characterizing genetic markers related to short stature, Celera and Ipsen said in a statement.
 
“Assuming the first phase of this collaboration is completed successfully, a key aim thereafter will be to develop diagnostic predictors for use in Ipsen’s clinical trials, which would potentially form the basis for commercial companion diagnostic tests for Ipsen’s short stature therapies,” the companies said in the statement.
 
Celera will receive an undisclosed payment for the first phase of this multi-year collaboration. Although the companies did not reveal the financial details of the collaboration, they noted that future payments to Celera will “depend on the success of the initial phase.”
 
In the first year of the collaboration, the companies will conduct exploratory screening using samples collected from children participating in an observational study sponsored by Ipsen. The identified SNPs will be validated, and if necessary more systematic screening of the human genome will be conducted to identify less obvious polymorphisms related to short stature.
 
In an e-mail to Pharmacogenomics Reporter this week, Didier Véron, Ipsen’s director of public affairs and corporate communications, said that the collaboration will seek to optimize patients’ response to two growth failure treatments in Ipsen’s arsenal. Paris-based Ipsen holds a worldwide license, outside of North America and Japan, to Genentech’s growth hormone Nutropin, as well as a license to the IGF-1 drug Increlex from Tercica.
 
“Given the need to provide short stature children with a more predictable therapeutic outcome … Ipsen with its ongoing clinical studies and market experience would be an ideal partner to develop and validate clinically relevant diagnostic and prognostic assays that will be commercialized by Celera,” Véron wrote in an e-mail.
 
In turn, Ipsen will gain access to Celera’s experience in genomics. “Celera's expertise in the sequencing of the human genome will provide Ipsen with the technologies, including bioinformatics, required to establish associations between genotypes, polymorphisms, and the short stature phenotypes,” Véron said. “Such association should lead to diagnostic and prognostic assays that will be developed by Celera in support of better treatment algorithms for short stature children.”
 
Treating Growth Failure
 
According to the medical literature, the expected height of individuals with growth failure or short stature is based on age, sex, and familial genetic history. However, there is a lack of agreement on what is considered “short stature” according to scientists.
 

“Given the need to provide short stature children with a more predictable therapeutic outcome … Ipsen with its on-going clinical studies and market experience would be an ideal partner to develop and validate clinically relevant diagnostic and prognostic assays that will be commercialized by Celera.”

Researchers have attributed severe shortness to normal factors, such as familial genes, and abnormal factors such as chronic hormone deficiency, disease in a major organ systems, mistreatment, adverse effects of certain drugs, chromosomal deletions, inherited diseases, and birth defects.
 
Genentech’s website for Nutropin tells parents who suspect their child to have growth failure to chart his or her growth compared to other kids of the same age and sex. Nutropin treats growth failure resulting from growth hormone deficiency, which occurs when the pituitary gland of an infant or a child fails to produce growth hormone, or doesn’t secrete enough of the hormone. Blood tests can diagnose the condition.
 
The Hormone Foundation estimates that the number of growth hormone-deficient children varies between 3,500 to 10,000 from country to country. In the United States, the exact prevalence of GHD is unknown, but The Hormone Foundation estimates that approximately 35,000 adults have GHD and 6,000 people are newly diagnosed with the condition each year.
 
According to Véron, only the so-called "organic " deficiency in growth hormones and primary deficiency in IGF-1 are treated by growth hormone and IGF-1 replacement, respectively.
 
“This leaves a large segment of the pediatric short stature population, which does not receive optimal treatment, thus the need to have a better understanding of the genetic components that may result in short stature and how best to treat them,” Veron said, adding that approximately 60 percent of the short stature population does not receive optimal benefit from treatments.
 
The ultimate goal of the collaboration is to define the optimal treatment regimen with growth hormone analogs such as Nutropin and IGF-1-treatments such as Increlex, administered alone or in combination with other treatments. Currently there are no response biomarkers identified within this market.
 
According to David Speechly, senior director of investor relations and corporate communications at Celera, the company will study risk and response markers related to both the disease and treatments. Celera will study candidate genes by sequencing and identify genome variance using arrays.

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