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BioMarin Pharmaceutical's CEO Discusses Q2 2012 Results - Earnings Call Transcript

Positive results for one or more of these patient-based trials could be transformative for the company. In particular, GALNS has the ability to quickly bring BioMarin to the next phase of maturity. There is an estimated worldwide population of approximately 3000 patients or 3x the MPS VI market size and all of which are theoretically eligible for treatment. And I’m happy to report that over 1200 patients have already been identified which is more than the entire MPS VI market served by Naglazyme, and combined with our exceeding worldwide commercial infrastructure that is highly leverage-able, that adds significant revenues and contributes substantially to our bottom line.

We continue to have confidence in the success of this program due to the clinical benefits seen in the Phase II extension study which Hank will elaborate on a little bit later, and the robust design of the Phase III trial. The Phase III trials include the enrollment of 176 patients. Selection criteria are baseline [walk] distance less than 325 meters which is [serious] to the design of the Naglazyme and Aldurazyme clinical trials; and no patients with planned surgical procedures during the 24-week duration of the trial. We’re also seeking the mean of two (inaudible) measurements at each time point which we expect to reduce variability in the primary endpoint based on our experience with Aldurazyme and Naglazyme.

Importantly we have two treatment arms in the study and can achieve statistical significance from AP value of 0.025 in either one of the treatment arms or 0.05 in each of the two treatment arms combined. We are further encouraged by the tremendous amount of support and encouragement from our patients and physicians, the [NTS IV Committee] and by the continuous patient benefit seen in the Phase II extension study including improvement in growth (inaudible).

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