BioMarin Pharmaceutical Inc. Q1 2009 Earnings Conference Call Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Salveen Kochnover - Collins Stewart - Analyst : Good afternoon. Thanks for taking my questions. Could you provide us with some color on the first quarter Kuvan new patient additions into the free drug program, and just comments from physicians and patients and how the XUS launch is progressing?

Question: Salveen Kochnover - Collins Stewart - Analyst : Can you maybe just give us a little bit more clarity as to why you think referrals are down versus the fourth quarter?

Question: Salveen Kochnover - Collins Stewart - Analyst : Great. And then is there any chance for an interim look at the Phase II PEG-PAL data post the eight week dosing?

Question: Salveen Kochnover - Collins Stewart - Analyst : And then just one last question. What are the determining factors that you intend to take into consideration on deciding whether to continue with BH4 cardiovascular program going forward?

Question: Salveen Kochnover - Collins Stewart - Analyst : All right. Thank you.

Question: Brian Abrahams - Oppenheimer & Co. - Analyst : Hi. Thank you very much for taking my questions. Question on the Phe handheld device. I was wondering if you could outline it in a little bit more detail some of the steps that are going to be necessary in order to make that available? And what's your sense, based on your market research as to the potential this might have in increasing Kuvan uptake?

Question: Brian Abrahams - Oppenheimer & Co. - Analyst : Great. And then just a quick follow-up. Can you describe in a little bit more detail the reactions that we're seeing with PEG-PAL at the higher doses independent of the Depo-Provera? And based on your dialogue with the FDA, how high is your level of confidence that the Agency will enable you to proceed into Phase II? LSEG STREETEVENTS | www.lseg.com | Contact Us consent of LSEG. 'LSEG' and the LSEG logo are registered trademarks of LSEG and its affiliated companies.

Question: Brian Abrahams - Oppenheimer & Co. - Analyst : Great. Thanks for the additional clarity.

Question: Chris Raymond - Robert W. Baird & Company - Analyst : Yes, thanks. I just want to make sure I understand some of the dynamics here with the Kuvan numbers. Steve, you mentioned, I think I heard you say that unit sales for Kuvan were up. Am I correct, 9% over Q4 '08; is that correct?

Question: Chris Raymond - Robert W. Baird & Company - Analyst : Okay. And so I understand Q1 -- or Q4 had an inventory impact, but is it an accurate statement to say that obviously your sales, your revenue run rate was not up 9.5%, so should we, from our perspective for thinking purposes, assume that maybe that $1 million inventory issue that we heard about in Q4 has been sort of worked out, or are we at sort of a steady state inventory level among the distributors?

Question: Chris Raymond - Robert W. Baird & Company - Analyst : So, remind us, what was the tablet unit growth sequentially in Q4 versus Q3?

Question: Chris Raymond - Robert W. Baird & Company - Analyst : Yes, Q4 versus Q3.

Question: Chris Raymond - Robert W. Baird & Company - Analyst : Okay, okay. So, it essentially looks like they just worked off the inventory from Q4.

Question: Chris Raymond - Robert W. Baird & Company - Analyst : Okay, thanks.

Question: Phil Nadeau - Cowen and Company - Analyst : Good afternoon. Thanks for taking my question. JJ, the first one is for you. After the long-term guidance you gave last quarter, there was a pretty violent reaction in the stock. I'm wondering if that has caused you and the management team to change your long-term plans as far as expenses, and if there are any -- if there is any way that you can actually decrease the expenses that you had projected you were going to have?

Question: Phil Nadeau - Cowen and Company - Analyst : Okay. And just a follow-up to that. Understanding that there are priorities within every company and it's hard to make any definitive statements, but if you did decide to cut costs, were there fertile areas to do that, or do you find that your expense structure is kind of as streamlined as it possibly could be given the projects that you think should be --

Question: Phil Nadeau - Cowen and Company - Analyst : Okay, that's very helpful. Hank, one for you. It seems like the guidance for mid-2010 data for the Phase II PEG-PAL trial is maybe somewhat later than we had thought, maybe a quarter or maybe even two quarters later than we thought. Is there any change in the design that you are thinking about versus what you have suggested in the past, or I guess why is that in mid-2010 rather than like Q1 or earlier?

Question: Phil Nadeau - Cowen and Company - Analyst : Okay.

Question: Phil Nadeau - Cowen and Company - Analyst : Okay. And one last question, just housekeeping question for Jeff. Jeff, do you have the quarterly non-GAAP EPS numbers for 2008, using the new method of computing non-GAAP EPS?

Question: Phil Nadeau - Cowen and Company - Analyst : Do you have the intervening quarters, Q2, Q3 and Q4?

Question: Phil Nadeau - Cowen and Company - Analyst : Okay, great. Thank you. LSEG STREETEVENTS | www.lseg.com | Contact Us consent of LSEG. 'LSEG' and the LSEG logo are registered trademarks of LSEG and its affiliated companies.

Question: Lucy Lu - City Investment Research - Analyst : Great. Thank you. I have a question on the Pompe disease program. What goes into your calculation and consideration in terms of finding a partner versus doing this program yourself? And I also want to know, if you decide to do it yourself, how soon can this program enter clinical testing, and do you need to change your R&D expense guidance for this year? I think it would probably take a lot of resources to do this. Thanks.

Question: Lucy Lu - City Investment Research - Analyst : JJ, it sounds like you're a little more open than before in terms of maybe wanting to take on this program on your own. Am I reading that correctly --

Question: Lucy Lu - City Investment Research - Analyst : Okay. And then the last question the BH4 program. If you were to discontinue that program, how much money can you save from an R&D budget?

Question: Lucy Lu - City Investment Research - Analyst : Okay, thank you.

Question: Joseph Schwartz - Leerink Swann & Company - Analyst : Hi. Thanks for taking my question. I think I heard you say that BPPS referrals were down versus the fourth quarter, but there is a backlog of patients, and I'm wondering now that we're looking towards the summer, do you expect more weakness this year in the summer months? And are all of the patients that were in the investigator-sponsored study that can get commercial therapy already on Kuvan? Thanks.

Question: Joseph Schwartz - Leerink Swann & Company - Analyst : I think there were a couple fairly large investigator-sponsored studies, and you were waiting to begin until those protocols were all figured out, and yet they were going to be using commercial drugs. So, I'm wondering, have those already begun, what stage are there?

Question: Joseph Schwartz - Leerink Swann & Company - Analyst : Okay, great. Thank you.

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : JJ, you said, just kind of following up on Lucy's questions about business development, but you had said earlier this year that there was a potential for a late stage opportunity in licensing this summer, that you guys were still in active negotiations. This is after the Riquent announcement. Is that still a possibility?

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : Okay. And you had said that if something late stage was going to happen, it would happen in the summer, or Q3. Still the same or you don't want to define it that much?

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : Okay. And could you repeat, as part of product sales guidance, you're giving guidance on transfer fees and things that were nonorganic sales. Can you repeat that, please?

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : Aldurazyme and I think there was something with Kuvan.

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : Okay. And none of the other ones have anything like that?

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : Okay, thank you very much.

Question: Josh Schimmer - SCN - Analyst : Hey, thanks for taking the question. For the Morquio program, aside from safety, what efficacy or disease measures will you be evaluating in that Phase I/II study? What activity will you be looking for specifically, and how will you decide what the appropriate dose is to move into Phase III? Thanks.

Question: Josh Schimmer - SCN - Analyst : Great. Thanks so much.

Question: Shiv Kapoor - Morgan Joseph & Co. - Analyst : Hi. I've got two questions. First on business development. It seems like you've got three drugs that are growing pretty fast, and you've got a strong pipeline. You just announced that there are two additional undisclosed drug candidates that could do an -- you could do an IND for pretty soon. I want to know, why do you feel that you need to end license more drugs, and perhaps the question should be, has that need decreased over the past six months?

Question: Shiv Kapoor - Morgan Joseph & Co. - Analyst : So, what I'm hearing is you're not desperate so you won't do a deal until it's very strategically beneficial and leverages your infrastructure.

Question: Shiv Kapoor - Morgan Joseph & Co. - Analyst : Sure. I'll ask the second question. On Naglazyme, can you go over some intra-quarter trends? It seems like you got the marketing approval in Brazil, and although you have been able to sell there before, it seems, according to my calculations, your penetration there is still only 30%, 35%. Have you seen a stronger trend toward the end of this quarter on Naglazyme?

Question: Shiv Kapoor - Morgan Joseph & Co. - Analyst : Okay, thanks.

Question: Andrew Vaino - Roth Capital Partners - Analyst : Hey, thanks for taking my questions. That there weren't too many serious adverse events with --

Question: Andrew Vaino - Roth Capital Partners - Analyst : You mentioned that there were no serious AEs with PEG-PAL. I wonder if you could mention what percentage of patients had antibodies raised to PEG-PAL?

Question: Andrew Vaino - Roth Capital Partners - Analyst : Okay. Thank you.

Question: Steven Willey - Thomas Weisel Partners - Analyst : Hi. Thanks for taking my question. Just one quick question on the GALNS compound that was just moved in the clinic. Can you just update us what the IT is there and maybe just comment on what [Avanade] is doing over in Europe? Thanks. LSEG STREETEVENTS | www.lseg.com | Contact Us consent of LSEG. 'LSEG' and the LSEG logo are registered trademarks of LSEG and its affiliated companies.

Question: Joseph Schwartz - Leerink Swann & Company - Analyst : Thanks for taking the follow-up. At a higher level, I was wondering if you could help us understand how do you assess the risk of pricing controls for your drugs in the current era we're in? And how prepared are you to counter that risk with pharmacoeconomic data?

Question: Joseph Schwartz - Leerink Swann & Company - Analyst : I would think that these drugs would stack up well on a quality adjusted life years basis, because you're adding quality to the early part of a patient's life as opposed to the latter part, as with a cancer drug or the like. Is that correct? And what about the ability to reduce surgeries and things like that? These are very sick patients that often need a lot of other interventions. Do you have data that you're able to use to defend yourself, if that ever came to pass?

Question: Joseph Schwartz - Leerink Swann & Company - Analyst : Great. Thank you.

Question: Carol Werther - Summer Street Research Partners - Analyst : Oh, thanks for taking the question. I was just wondering how -- what different parameters will go into deciding whether you'll go ahead with sickle cell anemia or PAH? The markets are quite different. I guess the length of trial you might have to do quite different. LSEG STREETEVENTS | www.lseg.com | Contact Us consent of LSEG. 'LSEG' and the LSEG logo are registered trademarks of LSEG and its affiliated companies.

Question: Carol Werther - Summer Street Research Partners - Analyst : Do you have all the information now that you need?

Question: Carol Werther - Summer Street Research Partners - Analyst : Okay, thank you.

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : I just wondered, of the $39.4 million in Q1 Naglazyme sales, how much was in the US, Europe, rest of the world, or however you want to break it down geographically?

Question: Leanna Mosotos - Wedbush Morgan Securities - Analyst : Thank you.