BioMarin Sinks Despite Meeting Primary Endpoint

Health and Healthcare

Published: March 21, 2016 9:05 am

Last Updated: January 13, 2020 11:43 am

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) made waves early Monday following the release of late-stage results from its pegvaliase study. The company announced that the pivotal Phase 3 PRISM-2 study of pegvaliase met the primary endpoint of change in blood Phe compared with placebo in preliminary results.

Keep in mind that BioMarin was one of the few companies we pointed out earlier this month that was expecting an FDA decision in March.

The pegvaliase treated group maintained mean blood Phe levels compared to their RDT baseline, whereas the placebo treated group mean blood Phe levels increased compared to their RDT baseline. The treatment effect demonstrated in this study represents roughly a 62% improvement in blood Phe compared to placebo.

In the secondary endpoints of the eight-week RDT, there was no benefit in inattention or mood scores seen in patients treated with pegvaliase compared to placebo. However, in an exploratory sub study of cognitive function in nine patients, the Cambridge Neuropsychological Test Automated Battery (CANTAB) showed trends of improvement favoring pegvaliase.

Cary Harding, M.D., Professor of Molecular and Medical Genetics and Pediatrics at Oregon Health & Science University and investigator for the pegvaliase Phase 3 program, commented:

Treatment with pegvaliase has resulted in dramatic Phe decreases down to within normal levels which have not been achievable in the past with other PKU treatment options. We are grateful to the patients who participated in this important trial. Blood Phe reductions at this level have the potential to have a meaningful impact on the lives of PKU patients.

Barbara Burton, M.D., Professor of Pediatrics-Genetics, Birth Defects and Metabolism at Northwestern School of Medicine and investigator for the pegvaliase Phase 3 program, concluded:

A therapy in development that shows such a substantial reduction in Phe levels could mean that for the first time, PKU patients who cannot comply with dietary protein restriction, can achieve targeted blood Phe levels. This pegvaliase study represents an important advance for PKU adult patients and a potentially meaningful treatment.

Shares of BioMarin closed Friday up 4.7% at $80.94, with a consensus analyst price target of $119.42 and a 52-week trading range of $62.12 to $151.75. Following the release of the results, the stock was down over 4% at $77.50 in early trading indications Monday.

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