Earnings Call Transcript - BMRN Q3 2020

Traci McCarty, Vice President of Investor Relations

Ladies and gentlemen, thank you for standing by and welcome to the BioMarin Third Quarter 2020 Financial Results Conference Call. Hosting the conference call today is from BioMarin, Traci McCarty, Vice President of Investor Relations. Please go ahead, madam.

Jean-Jacques Bienaimé, CEO

Thank you, Traci. Good afternoon and thank you all for joining us on today’s call. We hope you and your families have been well during these uncertain times. It is hard to believe that we are around the month nine of the global COVID-19 pandemic. So like many of you we are ready for a reset, a fresh start in 2021. But our commitments to providing innovative treatments to people with serious conditions keep us focused day-to-day. Indeed, BioMarin faced some challenges in the third quarter, and the results have strengthened our conviction in our mission and renewed motivation to achieve it. The most anticipated milestone of the year, the advancement of ROCTAVIAN gene therapy for the treatment of severe hemophilia, and vosoritide for the treatment of achondroplasia, each reached a key inflection point in their progression during the quarter. There were only 10 applications in the U.S. and Europe that were accepted for review by Health Authorities and are in process for potential approval in 2021. The CHMP opinion is expected in the second half of next year, and the U.S. PDUFA action date is planned for August 20 of next year. The community of parents with children who have achondroplasia who want a treatment option are that much closer to accessing the first potential pharmacological therapy for the condition. With these highly innovative products developed, approved, and commercialized for a variety of rare conditions over the last two decades, this is familiar and gratifying territory for BioMarin.

Jeff Ajer, CFO

Thank you, JJ. Globally, BioMarin's commercial brand contributed $1.24 billion or 8% growth year-to-date in 2020 compared to the first nine months of 2019. Given the challenges posed by the COVID pandemic this year, I'm very pleased with our results and the deep commitment of our commercial and manufacturing teams to our patients around the world.

Henry Fuchs, CMO

Thanks Jeff. JJ conveyed it's been an eventful third quarter for BioMarin. But many of the challenges faced by our R&D organization are part and parcel of the drug development process, augmented by the innovative nature of what we do, never a straight line. With ROCTAVIAN, we did not expect the complete response letter in August, but we have refocused on productive next steps toward approval. We remain encouraged and committed to ROCTAVIAN based on the continued support from our investigators who report the life-changing potential of ROCTAVIAN. With the complete one year Phase 3 data from our 134 subjects study, we like you excitedly await those top-line results in early 2021. In the meantime, we continue to make progress in our discussions with health authorities and understanding what it takes to register ROCTAVIAN. Based on the Phase 3 study, we're confident in hitting the annualized bleeding rate at endpoint at one year. We are also mindful of other critical measures, such as factor VIII levels that will be important to determine success beyond the annualized bleed rate.

Brian Mueller, CFO

Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the third quarter of 2020. As usual, our comprehensive report on the quarter will be available in our upcoming form 10-Q, which we're planning to file tomorrow. Starting with revenues as Jeff noted, we have updated our 2020 product line revenue guidance for the continued impact of COVID-19. Also, I want to remind you that although we did not give specific ROCTAVIAN revenue guidance for 2020, we were planning to launch the product during the third quarter. Therefore, most of the adjustments to our total 2020 revenue guidance, of which the midpoint was lowered by $60 million, was due to the ROCTAVIAN adjustment. Back to COVID-19, the prologue pandemic and specifically slower new patient starts during 2020 may continue into 2021 and restrict revenue growth of our base business. As a result, with the other headwinds we are experiencing through the delay in ROCTAVIAN approval and the previously anticipated impact of the U.S. Kuvan generic competition, we expect our total revenues in 2021 to be roughly flat compared to 2020 revenue.

Hank Fuchs, CMO

Turning to Europe, the EMA recently requested the complete one year results from the full Phase 3 study cohort of 134 subjects to inform their benefit-risk assessment. As a result, we withdrew our application for accelerated assessment yesterday and now plan to resubmit the marketing authorization application filing with complete one-year results in the second quarter of 2021.

Salveen Richter, Analyst

Good afternoon. On ROCTAVIAN, what provides you confidence that the one year Phase 3 expression data or ABR levels will not differ much from the Phase 1, 2 study? And also what do regulatory agencies really want to see from the one-year data and will the FDA accept your data from a partial group? Thank you.

Hank Fuchs, CMO

Hi, Salveen. Complex question. I don't know that we predict what the results of the Phase 3 study will be at 52 weeks or 104 weeks or thereafter given that we started a little bit lower in the Phase 3 than we did in the Phase 1, 2, or so it seems. I think it's that variability that makes it difficult to answer the second part of the question, which is, what do they really want other than more patients followed longer. And so whether the cut that we take at the beginning of the year for the Europeans will contain enough information to get them to the place where they've seen enough duration is going to be discussed with the agency. We're not in a position to predict the outcome of those discussions because they await the data. So I think the data is going to drive our next conversations with agencies>

Cory Kasimov, Analyst

Hey, good afternoon, guys. Hank, another one for you on the vosoritide this time, I appreciate the explanation you had with your view and the FDA desire for two-year data. So I guess I'm wondering, given some of the apparent differences of opinion, at least at this stage between you and the agency. Do you see it as a positive or a negative that they're not planning to convene an advisory panel at this point?

Hank Fuchs, CMO

Thanks, Cory. I don't know how to interpret it either way. Some had told us that they wished there was an advisory committee on ROCTAVIAN. I think that there are other forces that are causing the agency to call advisory committees and not call advisory committees. The good news in this regard is that this division has a history of regulating products. Now, we have to make sure people understand that vosoritide is not growth hormone but they do understand stature and how it relates to things like final adult height and functionality and long-term alleviation of morbidity. The particular discussion topic is not a new one. It's been around for a lot of years. So I think we're encouraged that we have a good plan to address it. That's evidenced by the fact that they've accepted the file for review.

Robyn Karnauskas, Analyst

Hi, thanks for taking the question. What a lot of confusing FDA information this year. And then I just have a couple of questions there. So I think on the last call, you said that you would meet with the agencies. Are you going to not meet with them to figure out if you can narrow the package until you get the one-year data? That's my first question. And then on vosoritide, just why would they put the language in there? It just to me, it felt a little strange. Do you have a sense of why they reminded you they want to your data, or whether or not there's a method in place whereby you can sort of give them additional data as it comes forth? Help me understand those two things if you can? Thanks.

Jean-Jacques Bienaimé, CEO

So Karnauskas, yes, we have met with the FDA. Without going into the substance of the meeting, the conversations are productive, and the discussions are progressing. The big picture conclusion remains the same, which is that they want more longer data. We'll look at the data in January and see how close we come with them to assuage their concerns. On vosoritide, why did they reiterate to your request? Who knows, really, and we'll get into discussion with them about that. But it seems like, consistent with what they said at the advisory. To remind you, I think we started enrollment in the Phase 3 trial at the end of 2016, the advisory committee was in 2018. So, we were pretty far down the road when the subject was kind of put to the advisory committee discussion, and the advisors had a lot of important information and considerations, which for the most part resonated with the plan of our program.

Christopher Raymond, Analyst

Hi, thanks. Maybe just a broader strategic question. So you guys have kind of held things steady, more or less on the business development front for a while now, and a few of your peers seem to be showing maybe a bit more propensity or maybe even urgency to do deals. I guess, JJ, just wonder if maybe you could talk in a broader context, how you're approaching that side of the business now versus years past or how you're viewing things in terms of that environment and maybe more specific to BioMarin's need? Thanks.

Jean-Jacques Bienaimé, CEO

Yes. Well, we have an answer, you obviously deal this year, like DiNAQOR and there's more to come, and we will continue to do that. Later stage deals are way more expensive and riskier. And also if you require, then more resources in financial resources, which obviously, we can gather some. But we still are pretty confident in the approval of ROCTAVIAN and vosoritide. So I would say the need for a significant business development deal, I mean, a business development deal in the short-term, we don’t consider it to be very acute. We’ve said in the past that actually and it before we got a CRM for ROCTAVIAN, as ROCTAVIAN would be launched that it will start generating some significant cash flow, and will allow us then to be able to be more aggressive in the space. But we already have a pretty, we have an expanding pipeline. There is more to come there. And so we don't believe that a large business development deal in the short-term is necessary. Now obviously, if put in a six to nine months from now becomes evident that either ROCTAVIAN will be approved or would be even more delayed or vosoritide, which is very much by the way, then obviously, there will be a different situation. But we’re not there at this time.

Joori Park, Analyst

Hi, I'm Joori Park dialing in for Joe. Thanks for taking our question. My question is on ROCTAVIAN. I was wondering what you have done to ensure that physicians treating the two-thirds of the remaining patients generally abide by the more rigorous on-demand steroid regimen? And do you have any insight into whether they will actually abide by it? Do you have like a medical monitoring group to give you confidence that they'll be doing it or are you going to find out when they un-blind the data?

Jean-Jacques Bienaimé, CEO

Thanks for the question. Yes, our medical group is all over this issue. But I have to say that as soon as the interim analysis results were available, investigators pretty quickly realized that they were on the more let's defer the on-demand steroids than let's jump on the on-demand steroids. As we mined some of the additional data and we share this data with you, we recognize that investigators are starting steroids later in the course of the transaction than in the Phase 1 than the earlier study. We were also finding that there were breakthrough episodes of recurrent transaminitis and factory reductions in the interim analysis cohort. On that basis, we met the investigators where they were already heading, which was to fortify their attention to the details of initiating steroids and the speed at which they taper the corticosteroids, so the monitoring group has been very, very plugged into the investigators reviewing with them as contemporaneously as possible.

Martin Auster, Analyst

Well, thanks for taking the question. I think most of them have been answered, but maybe a follow up on vosoritide. And could you remind us what percentage of patients from the vosoritide basically went into the open label? And then I guess you should be kind of getting that two-year data from the years of exposure? Is there a specific plan or specific requests to furnish that two-year old data to the FDA and if so, can you account for that within the current PDUFA timeframe? Thanks.

Jean-Jacques Bienaimé, CEO

Yes, we can accommodate that data request within the current PDUFA timeframe. We are still in the early stages of the review, gathering initial information requests from both Europe and the United States, and it wouldn't be surprising if additional data are being considered. I don't recall the first part of your question, Martin; I forgot the initial part.

Paul Matteis, Analyst

Great, sorry about that. Thanks so much for taking my question. Hey, just one more on vosoritide to that point. How many patients of two-year data do you think you might have by say, the midpoint of the review? And have you taken any look at some of the long-term data and done any of your own internal analysis to kind of just get a sense of the corroborate the Phase 1, 2?

Jean-Jacques Bienaimé, CEO

Yes, I think that how many patients are pretty straightforward because I think we un-blinded that last patient was out of the one-year in November of 2019. So we'll have all 110 patients of data. Well, 110 patients will have had to have their two-year assessments this month. It should be a pretty fulsome dataset and should be available during the course of the review.

Philip Nadeau, Analyst

Definitely and thanks for taking my question. Hank, another question on vosoritide, in your prepared remarks, you mentioned that there were a number of analyses that you've done to show vosoritide is going to impact adult height. We didn't go into much detail. Could you discuss those maybe in a bit more detail what data analysis you have to show the FDA to possibly change their minds? And I guess a related question in the past and Orphan disorders, the FDA didn't like natural history and differences versus natural history persuasive. Why isn't that the case here?

Hank Fuchs, CMO

I missed the balance of your question. The second part of the question is, why would the natural history be, I forget which side you asked me from unsupported or supported?

Geoff Meacham, Analyst

Hey, guys, thanks for the question. Appreciate it. Just had a few, commercial when you look at the COVID impacts your business I would have thought that logistics would have been figured out by now. But is it just that cases are accelerating and that's more of a disruption or are there still processes that have yet to be worked out to ensure compliance?

Hank Fuchs, CMO

So maybe with the start second question, Greg Guyer, you had talks, can answer the question.

Greg Guyer, Vice President

Yes, thank you. So we have also there are a lot of discussion during the initial filing of VIROX filing and we've had a lot of experience with questions, many, many questions during the inspection or during the review process. I think what's been good is we've had a good dialogue. We've had a bunch of discussion on comparability. The agency accepted that, and we've got no further CMC questions related to those so right now, we're pretty confident in the CMC package that supports ROCTAVIAN.

Jean-Jacques Bienaimé, CEO

In closing, despite the challenges faced in the third quarter due to the impact from COVID-19, and the delay of ROCTAVIAN approval, our base business remain solid, paving the way for substantial non-GAAP income and operating cash flows in 2021. Thank you for your support, and we will now open the call to your questions.

Operator, Operator

Yes. Thank you, sir. Your first question comes from the line of Salveen Richter from Goldman Sachs. Your line is open. Thank you. Your next question comes from the line of Cory Kasimov from JP Morgan. Your line is open. Your next question comes from the line of Robyn Karnauskas, SunTrust. Your line is open. Your next question comes from the line of Christopher Raymond from Piper Sandler. Your line is open. Your next question comes from the line of Joori Park from SVB Leerink. Your line is open. Your next question comes from the line of Martin Auster from Credit Suisse. Your line is open. Your next question comes from the line of Paul Matteis from Stifel. Your line is open. Your next question comes from the line of Philip Nadeau from Cowen & Company. Your line is open. Your next question comes from the line of Geoff Meacham from Bank of America. Your line is open. Your next question comes from the line of Gena Wang from Barclays. Your line is open. Thank you, speakers. I am showing no further questions at this time. I would like to turn the conference back to Mr. JJ, Bienaimé, Chairman, and CEO. Sir, please go ahead.