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Analytical Chemistry, News | January 23, 2013

BBL Contributes to Successful Phase 3 Trial of BioMarin Orphan Drug

by Aaron Burke

Keratan Sulfate Keratan Sulfate

Bay Bioanalytical Laboratory (BBL), recently acquired by Pacific BioLabs (PBL), provided important biomarker data to the successful Phase 3 trial of GALNS for Morquio Syndrome. “We are very proud of our contribution to the success of this innovative treatment for an orphan disease,” said Bob Cunico, Principal Scientist and founder of BBL.

“BBL’s work on the GALNS trial is a fine example of the sophisticated analytical expertise we can now offer to all of PBL’s clients,” said Tom Spalding, President of PBL. “We are excited to add these capabilities to our existing services in Toxicology and Microbiology.”

In 2007, BioMarin Pharmaceuticals approached BBL to develop and validate a novel LC/MS/MS method to measure plasma and urine concentrations of Keratan Sulfate. Keratan Sulfate is a glycosaminoglycan (GAG) that accumulates in the cells and tissues of patients afflicted with Morquio Syndrome.  The children and young adults with the disease have a defective enzyme (GALNS) and cannot properly process certain GAGs. The GAGs accumulate and cause damage to muscles, bone, and other organs.

BioMarin sought the bioanalytical expertise of BBL to measure Keratan Sulfate in blood and urine to support their preclinical and clinical studies of GALNS. The effort between BBL and BioMarin was extensive and resulted in peer reviewed publications from Bob Cunico and Erik Foehr, a successful IND submission, and now a Phase 3 trial that demonstrated the efficacy of the GALNS treatment. The urinary biomarker data demonstrated a dramatic and statistically significant decrease in urinary keratan sulfate with weekly treatment with GALNS.

The work involved many team members from BBL, including excellent analytical skills of Jayoung Ohh, timely and expert support by the QA group, Kate Lancaster and Samantha McCoy, as well as logistics and lab support by Steve Guthrie and Andrea Parungao.

Pacific BioLabs performs analytical and bioanalytical testing including biomarker discovery and analysis.


Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. (2011.) Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis 3(16):1855-66.

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