BioMarin Pharmaceutical, Inc.
30th Annual JP Morgan Healthcare Conference
January 9, 2012 02:30 pm ET
Jean-Jacques Bienaimé – Chief Executive Officer
Cory Kasimov – JP Morgan
Cory Kasimov – JP Morgan
Good morning, everybody, we’re going to keep it going here. My name is Cory Kasimov; I’m a Senior Biotech Analyst at JP Morgan and it’s my pleasure to introduce our next company here this morning which is BioMarin. Presenting for the company is the CEO, J.J. Bienaimé. A big year coming up from BioMarin with a lot of expected news flow and J.J. will be here to tell us the story. And following his presentation there will be a break out across the hall in the Georgian Room. So with that I’ll turn it over to J.J.
Thank you, Cory. Good morning to everybody. This presentation will contain forward-looking statements. So BioMarin is a company that’s mainly focused on genetic and serious medical disorders. Our strategy has been to go after high or extremely high unmet medical needs and try to have a large impact on a small patient population. We are kind of the opposite of big pharma – we want to make a big difference in a small patient population instead of a small difference in a large patient population. And we want to be first to market or best in class.
And the four products we have on the market today are the only products approved for the indications that they treat, and when you do that, this is how you get price leverage and relative low commercial cost, and also you get fast development timelines. So we have four commercial products helping fund the pipeline. You will see we have a pretty deep pipeline in the company. We have multiple new product opportunities, we have a lot of activities going on this year and a lot of data readout by the end of the year, and we also have new (inaudible) in development. And despite a very rich pipeline we are also very active on the business development front.
But the pipeline is definitely the priority for the company this year. We expect to advance seven programs in the clinic. This is the most we’ve ever had in the history of BioMarin. So we have products at different stages of development: GALNS in Phase III, PEG-PAL approaching Phase III next year and Firdapse in the US approaching Phase III although it’s already approved in Europe. Our (inaudible) stage programs – BMN-701 for Pompe’s Disease; 673, a PARP Inhibitor for Genetically Defined Cancer; 111 for Achondroplasia. We’re studying the healthy volunteer study probably in about a few weeks from now and we have multiple other programs in preclinical and one we just announced at RND Day last year was 190 for Batten’s Disease or a form of Batten’s Disease.
Also excitingly we are potentially, in addition to GALNS which will hopefully be preclinical next year we have three programs advancing to Phase III in 2013 potentially – PEG-PAL, 701 for Pompe and 673 for cancer. We stated a few years ago that we had a goal of finding one IND a year. We’ve been basically true to that which in 2010 we found two INDs. They were through acquisitions, early stage acquisitions but we did, and last year we filed CMP for Achondroplasia that was internally developed at BioMarin. That’s the depiction of our pipeline, trying to project ourselves from where we are today.
If you look at GALNS for MPS IV we should be able to launch the product in 2013/14 and then after that PEG-PAL, 701, and 673 – that’s the 2015-2017 target launch. Following that will be TPP-1 for Batten’s Disease and after that still, again lots of activity going on in the company in this respect. So the most exciting value driver in the short-term is our products GALNS for MPS IVA which is in Phase III at this time. We have very exciting Phase II data that showed endurance benefits with the product. Also we have preliminary growth data that’s very encouraging and the biomechanical markers are suggesting that GALNS does reach the correlation of the patients and improves [contra site] activity which is where the disease is mainly active. And we have safety data that’s consistent with earlier results and other ERT as a (inaudible) therapies product.
But what we’re doing to maximize the launch of the product – we’re doing additional studies in patients under five. We started that and in non-ambulatory patients so that when we launch the product we have a lot of clinical data to cover the entire spectrum of the disease. And we are also planning some supportive analysis to have a comprehensive view of the clinical benefit of the product to support registration. So I’m pleased to inform you that you know, we are on track for enrollment. At the end of December we had enrolled over 91 patients and our goal is to enroll about 160 patients so we have the traditional hockey stick enrollments. So based on this and the fact that we have Brazilian and German sites getting onboard this month, early next month, we anticipate enrollment to be completed by mid-April of this year; and consequently we feel pretty confident we’ll have top line data in Q4 of this year as we announced.
Also we have data that shows that the product increases gross velocity. You have to understand these patients, the MPS IV patients, generally untreated they stop growing around age 7,and here clearly we are making the patients grow again which is pretty dramatic. And I think it increases significantly the chances of regulatory success for the product and increases the value of the product to payers. Also we have more and more long-term urinary keratin sulfate data. This is what you want to decrease in the patients. This is responsible, the accumulation of keratin sulfates either contra site is responsible for the disease and here we have data over two years now, and you see actually we did different doses – 0.1 mg, then 1 mg, 2 mg, then back to 1 mg when we did the first phase of the extension study.
Interestingly enough when we went back to 1 mg from 2 mg the keratin sulfate went back up, which makes sense. We reinitiated the dose at 2 mg after that and that keratin sulfate started going down again which is very good as a demonstration of the efficacy of the product. So we believe that our protocol maximizes the chances of success. Our double blind study going on right now has a 90% power to show a 40 meters’ difference between placebo and the two active arms. Significance could be as low as 27 meters and hopefully we’ll do better than that, so we have three ways to win in terms of showing difference just against on treatment arm or two treatment arms combined.
And (inaudible) criteria is age over 5 but we are doing another study to treat patients under 5. We are shooting for close to 40 sites around the world. It’s also kind of a pre-commercialization study. 85% of the MPS IV patients are outside the US so obviously we want to have approval in the US but it’s very important to give hands-on experience to all the opinion leaders in the world on the product and that’s why we’re doing trials all over Western Europe, lots of South American sites and Southeast Asia so that by the time we launch the product everybody that counts in the disease space, all the clinicians will have hands-on experience with the product.
So we’re anticipating top line results at the end of this year, filing of the DLA in Q1 of next year, anticipated approval in the US in late ’13, early ’14. The opportunity is pretty large. This probably will be our largest enzyme replacement therapy product. The prevalence is probably over 3000 patients in the world. We have identified over 1000 patients already. When Naglazyme was approved we only had identified about 250 patients so that’s a big difference here, and we already have a strong, established worldwide infrastructure. When we launched Naglazyme there was no employee outside the US of BioMarin. We have now over 100 people out there. So this is where I think the P&L leverage is going to happen starting only in 2014 when we launch the product. It’s going to make a big difference on our profitability.
Moving on to PEG-PAL, PEG-PAL has been a long development process. It’s a much more complex product than GALNS but what we have shown is that in 18 out of 21 patients treated with the product on a long-term basis, on a chronic basis, we’ve been able to reduce phenylalanine levels pretty substantially. So we have already established the safe and effective chronic dose for PEG-PAL which is about 1 mg per kg per day. And we have had a very successful Type C meeting with the FDA where it’s clear that a reduction in Phe levels would be an acceptable primary endpoint for the Phase III study; and also the fact that the pivotal trial which we will hopefully start early next year will be able to be done in noncompliant patients, which is the majority of PKU patients.
So again, this is a slide that shows that in all the patients treated so far with 1 mg per kg per week we’ve been able to get the vast majority of the patients back to therapeutic levels of Phe. These patients were at base line above 1200 micromoles on average which is near toxic levels of Phe and we’ve been able to bring them down to below 600, which these are patients that would not respond to KUVAN