Marked for survival

MONTPELLIER, France—In the category of "good things come insmall packages," genomic and pharmacogenomic company Skuldtech and fellowFrench company AB Science, a Paris-based pharma specializing in the developmentof tyrosine kinase inhibitors, say that from a simple drop of blood, they wereable to identify biomarkers that are predictive of pancreatic cancer survivalassociated with masitinib treatment.

The recently announced discovery comes out of a Phase IIIclinical trial focusing on pancreatic cancer treated with masitinib that wasmanaged by AB Science. The purpose of the trial was to "evaluate thetherapeutic efficiency of a new treatment combining masitinib and gemcitabineas compared with the current standard pancreatic cancer treatment ofgemcitabine only," according to the two companies, but based on Skuldtech'stechnology for associating gene sequencing with the company's proprietarybioinformatics tools and gene profiling studies, they came up with somethingmore. They were able to identify specific transcriptome markers that reportedlycan distinguish between the different populations treated during the Phase IIIstudy and select the predictive markers for higher pancreatic cancer survivalassociated with masitinib treatment.

"AB Science has been the sponsor of the clinical trials andmanaged the Marketing Authorization Application filing. Skuldtech has been incharge of the biomarker identification on the basis of the blood samplescollected during the masitinib Phase clinical trials III," Skuldtech CEO DidierRitter tells ddn. "AB Science andSkuldtech worked closely together to give clinical sense to the biomarkers datagenerated by Skuldtech to meet regulatory authorities requirements for filing.Skuldtech has managed the entire wet lab analysis for biomarkersidentification. Skuldtech identified the biomarkers and validated the selectedgenes. AB Science and Skuldtech worked closely together in order to select thelast set of genes matching the masitinib Phase III clinical results."

This is the first time that Skuldtech and AB Science haveworked together, Ritter notes, but after this biomarker discovery, they havedecided "to collaborate on other masitinib applications."

These newly discovered biological markers "will pave the wayfor market authorization of a companion diagnostic test associated with thisdrug," the companies say. The predictive markers for survival associated withmasitinib treatment and identified in this Phase III clinical trial are thejoint property of AB Science and Skuldtech, and the companies have filedpatents have been filed to protect the markers, which are also associated withthe masitinib Marketing Authorization Application already filed with regulatoryauthorities by AB Science.

If and when masitinib obtains market authorization fortreating pancreatic cancer, however, AB Science will retain all of the rightsrelated to masitinib itself.

"To the best of our knowledge, this is the first time thatnew biological markers have been discovered during a Phase III clinical trial,"Ritter noted in the news release about the discovery. "These markers are bothspecific to the drug being developed and associated with positive patientresponse. Faced with the 'patent cliff' and a challenging economic context, thepharmaceutical industry is seeking new solutions for achieving successful PhaseIII clinical trials, and also new vectors for growth. The concept ofpersonalized medicine or personalization of treatments therefore represents asource of hope both for patients and the medical industry."

Pancreatic cancer is an aggressive cancer with poorprognosis—the survival rate after five years is only 5 percent—and the cancercomes in at about 110,000 new cases per year in industrialized countries.Masitinib is an orally administered tyrosine kinase inhibitor that targets mastcells as well as a limited number of kinases that play key roles in variouscancers. Because it inhibits certain kinases that are essential in someoncogenic processes, it is hoped that masitinib will have an effect on tumorregression, alone or in combination with chemotherapy. Masitinib receivedorphan drug designation in the treatment of pancreatic cancer from both theU.S. Food and Drug Administration and the European Medicines Agency.