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Article Excerpt
PHILIP NADEAU, ANALYST, COWEN & CO.: (audio in progress)-- to Cowen & Company's 29th Annual Healthcare Conference. I am Phil Nadeau, a biotech analyst here at Cowen and it is my pleasure to introduce the next presenting company, BioMarin. We are glad they could make the trip all the way out here from the Bay area. Jeff Cooper, the Senior Vice President and CFO, is here to give about a 20 or 25 minute presentation. I imagine there will be some type of questions in this room after and then immediately following the session, there is another breakout room -- breakout session. Jeff?
JEFF COOPER, SVP & CFO, BIOMARIN PHAMACEUTICALS INC.: Thanks, Phil and good morning. First of all, I'll cover the Safe Harbor statement. That this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical and you can obtain further information in our public filings, our 10-Q, 10-K and 8-K reports.
So giving you an overview of BioMarin, we have a business strategy that targets genetic and serious medical disorders. We primarily focus on orphan drug indications. We are located in the San Francisco Bay Area. We have about 650 employees and we have operations in the US, Europe and South America and in the Middle East.
Our 2009 revenues are projected to be between $307 million and $336 million. We have three approved products in the marketplace. Our first is Naglazyme for MPS VI, which is a lysosomal storage disease, rare disease for which we have worldwide rights, launched the product in the US first in mid-2005.
Aldurazyme in conjunction with our partner, Genzyme, for MPS I. Again, another rare lysosomal storage disease. That was launched in the 2003 timeframe. And our third product is Kuvan that we launched late in 2007 for phenylketonuria, which is a result of high Phe levels, which can cause diseases of the brain, including mental retardation.
We have a number of new products in our development pipeline. The first of which is PEG-PAL, which is an injectable therapy, also for phenylketonuria; GALNS for MPS IVA, which is a lysosomal storage disorder affecting bones and connective tissue that will be moving to the clinic soon; and a number of new IND candidates that I will talk about later in the presentation.
From a financial perspective, our projected 2009 net income is between a loss of $15 million and breakeven on a GAAP basis and $17 million to $35 million on a non-GAAP basis, which excludes non-cash stock compensation expense. I should also note that both our GAAP and non-GAAP net income includes the impact of a $50 million lower charge related to Riquent, which is the deal that we recently did with La Jolla Pharmaceutical.
From a cash perspective, we ended last year with almost $560 million in cash, which will be more than sufficient funds to continue development of our programs and pursue business development opportunities.
One of the things that BioMarin has been successful at is getting products to the market very quickly. It is estimated that the average pharmaceutical time from IND filing to approval is around 10 years and you can see from the chart that we have been able to improve upon that significantly both for Aldurazyme and Naglazyme in which we reached the marketplace in about five years and in the case of Kuvan, a little over three years. So this has resulted in reducing the overall R&D costs for the programs by minimizing the time in development, as well as starting revenue sooner.
In terms of our pipeline, I talked about the three commercial products. I will touch upon those later on in the presentation. We also have a number of programs we are working on in CD, as well as the sickle cell trial that we completed last year. I talked about PEG-PAL for PKU.
In terms of our preclinical program, again, GALNS for MPS IVA. We're also working on a program for Duchenne Muscular Dystrophy, a program for Pompe disease. We also are working on a prodrug that potentially could improve the bioavailability of BH4, which is the compound for Kuvan that potentially could extend the patent life of the program. And finally, IgA protease for IgA nephropathy for the treatment of kidney disease. We also have a couple of other programs that we haven't talked about publicly yet.
So moving on to Kuvan and PEG-PAL for PKU. We believe that our two products potentially have the potential to address the entire spectrum of phenylketonuria patients. We believe there is about 50,000 worldwide. Kuvan, which is currently on the market, was launched in late 2007, is an oral small molecule. We believe that it could benefit up to about half the population, as much as 25,000 individuals.
PEG-PAL, which is a bacterial enzyme substitution therapy that is currently in Phase I, we believe that this product could be helpful for patients who do not respond adequately to Kuvan or for those patients who want to reduce their Phe levels beyond what is possible with Kuvan. This is a program, as I noted, that is in Phase I and is ongoing. So we believe between these two products, potentially we could cover the entire spectrum of patients with phenylketonuria.
In terms of the Phase I study for PEG-PAL, basically it is a safety and pharmacokinetic study of single injections of...
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