Q1 2020 Earnings Call

Thank you may and thank you everyone for joining us today to remind you that non confidential presentation contains forward looking statements about the business prospects, the biomarin, including expectation.

Traci McCarty: Thank you everyone for joining us today. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin, including expectations regarding Biomarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in Biomarin's filings with the Securities and Exchange Commission, such as 10Q, 10K, and 8K reports. On the call remotely from Biomarin Management today are JJ Bien-Aimé, Chairman and Chief Executive Officer, Jeff Acher, Executive Vice President We hope to keep this call to one hour and also give everyone the opportunity to ask a question today, so we request that you limit yourself to one during the Q&A portion of the call. Thank you for your understanding. I will now turn the call over to our Chairman and CEO, JJ Bien-Aimé.

<unk> financial performance commercial products potential future product in different areas of therapeutic research and development results may differ materially depending on the progress of Vibrance product program axis of regulatory authority availability of capital future actions in the pharmaceutical market.

Don't Miss by competitors and those factors detailed in Vibrance filings with the Securities and Exchange Commission, such a 10 to 10-K K reports.

On the call remotely from Biomarin management today, our JJ piano named Chairman Chief Executive Officer, Jeff Nature, Executive Vice President Chief Commercial Officer, Robert Baffi, President Global manufacturing technical operations, excuse President worldwide research and development and Brian Mueller acting Chief Financial Officer.

We hope to keep it keeps its called to one hour and also give everyone. The opportunity to ask a question. So we request that you limit yourself to one during the Q and a portion of the call. Thank you for your understanding I will now turn the call it to our chairman and CEO James.

Thank you Tracy a good afternoon in Q4, joining us on today's call.

JJ Bien-Aime: Thank you, Traci. Good afternoon, and thank you for joining us on today's call. We hope you and your families are healthy and coping through these unusual circumstances brought about by the COVID-19 virus. These are unprecedented times, but the essential nature of our medicines to the patients who need them has enabled Biomarin to weather the challenge of COVID-19 quite well. Equally important, I want to emphasize the extraordinary dedication of our employees who have kept operations running smoothly in order to maintain access to our therapies around the world. Our first quarter record results of 502 million dollars in total revenues, or 25% growth over the last year, are a testament to the importance of our therapies and our diversified product base and commercial footprint. Due in part to the sale of Firdap, DapNet income in the first quarter was $81.4 million, exceeding our current full-year guidance rate of $20 to $80 million.

We hope you on your families are healthy and managing through that.

Usually circumstances brought a bite, but by up because he'd nice environments.

So these are unprecedented times, but the essential nature of our medicines to patients we need them has enabled biomarine to weather the challenge of probably 19 quite well.

What is important I want to underscore the extraordinary dedication.

Well kept operation.

Running smoothly in order to maintain access.

Our therapy around the world.

[music] <unk> first quarter record.

So that's $502 billion, a pull revenues for 25% growth.

Here.

Testament to the importance of our therapies and diversified product <unk> and commercial footprint.

Doing parts to the sale of Firdapse GAAP net income into first quarter was $81.4 million.

Exceeding our current 40 or guidance range of 22 $80 million.

JJ Bien-Aime: In the first quarter, we experienced minimal interruptions due to COVID-19, but we do anticipate the potential for more meaningful business disruptions for the remainder of 2020 due to the pandemic. As a result, we have chosen to reduce our full-year total revenue guidance by around 5% or $100 million, while maintaining both GAAP and non-GAAP income for the estimates provided earlier this year. Despite potential near-term impacts to our commercial business from COVID-19, our next blockbusters, BMN-270, Valoxocogin, Roxaparvovec, also known as Varrox for Hemophilia A, Briefing on Vasoetia Choricotrophagia.

In the first quarter, we experienced minimal disruption you could be 19.

But we do anticipate the potential for more meaningful business disruption for the remainder of 2020 due to the pending.

Hi, there we don't we have chosen to reduce our 40 or total revenue guidance by around 5% or full $110, while maintaining both GAAP and non-GAAP income what do you estimate.

Earlier this year.

Despite potential near term impacts to our commercial business Okabe 19.

Blockbusters you meant to 70, so I'm going to talk a gene Fox apart with ex some of those bought walks for hemophilia and it was only tied for you go to page I continue to invest and Jeff.

Where are we deal or we simply a pool brand name for the event to 70 in a moment.

We feel it was are we talking preclinical page in the quarter, we announced that based on recent meetings.

JJ Bien-Aime: In the quarter, we announced that based on recent meetings and successful meetings with health authorities in the US and Europe, we plan to submit marketing applications to the FDA and EMA in the third quarter of this year. It was approved that Rosaritab would be the first medicine for the treatment of achondroplasia in the U.S. and Europe. So we are delighted that this potential therapy is a step closer to regulatory approval. In conclusion, Biomarin employees have risen to the evolving challenges of the COVID-19 pandemic, demonstrating a high level of commitment and dedication to the patient's research. The underlying fundamentals of our business remain strong, and our manufacturing and supply chain are resilient. We have built a durable business on central medicine. Transition the pipeline to address larger rare indications, diversify risk, and position ourselves for substantial success in both the near term and the long term. We are confident in our ability to manage through this ongoing global health crisis while staying grounded in our long-term strategy for success.

Its success will meet seem to health authorities in the U.S. accurate.

We plan to the Midmarket he got teachers to the Andean region in the third quarter of this year.

Oh, It was always side would be the first medicine for the treatment of equal your page in the U.S. and you up.

So we are delighted that these potential therapy, which did step closer to your regulatory.

In conclusion Biomarine I'm pleased to have reason to meet evolving chongjin I'm, just hoping 19 and then Mitch.

Pretty high level of commitment and dedication to the peaks as research.

The underlying fundamentals of our business remains strong and our manufacturing and supply chain resilient.

We have a durable base business the central Medici transitioned the pipeline to address larger rare indication.

Pardon me and positions ourself shoot for substantial success.

In both the near term and an older.

We are confident in our ability to manage through this whole Wayne global health crisis, while staying grounded no long term strategy for success.

JJ Bien-Aime: I'd like to say a few words about Robert Baffi, who has made tremendous contributions to the organization over the last 20 years. During his time at Biomarin, he has manufactured the most complex biological products in the world and built the most advanced commercial-scale gene therapy manufacturing capability. His leadership, technical expertise, foresight, and dedication have played a key role in where we stand today, and we want to acknowledge his many contributions. Thank you, Robert, and we are pleased that he will remain with Biomarin through the review of TMN-270 VAROX and the Valvodoritized Marketing Applications to ensure manufacturing continuity as his successor, Greg Goyer, begins his journey with us in May, coming from Britain. With my respect Thank you all for your continued support, and now I would like to turn the call over to Robert to say a few words. Robert.

I like to see if you're worried about Robert Duffy.

Who has made tremendous contribution.

The organization over the last 20 years.

During this time and borrowing yet manufactured the most complex biological products in the world and feel it's the most advanced solutions shale and you put out the metrics actually kept I've already you know.

[noise] leadership technical expertise foresight and education.

Peter key role in where we stand today and we want your knowledge. His many contributions thank you Robert.

Well, we're pleased that you will remain we probably sooner we view as you mentioned 75 trucks and the south what's already ties marketing applications to ensure manufacturing continuity as his successor, Greg will begin his journey, we that's neat stemming from which Mike.

Thank you all for your continued support and now I would like to turn the call over threat, Robert just if youre it's robin.

Glad you Jay.

The basin has always the core Biomarin success. During my 20 years idea is that sort of technical operations I was still in the call for me if you're guiding principle, so for the culture of innovation.

First let's start with some form and lead decision making.

Well the compliance focus our efforts on patient safety and clinical outcomes and third most teachers know where do you agree its activity and resiliency and all approach the drug development.

Robert Baffi: Thank you, JJ. Innovation has always been at the core of Biomarin's success. During my 20-year tenure as the head of technical operations, I instilled in the company a few guiding principles to foster a culture of innovation. First, let science inform and lead decision-making. Second, let compliance focus our efforts on patient safety and clinical outcomes.

These three time is confused with the towers. So the most dedicated people I've ever worked with has consistently enabled us to take research is rapidly develop.

Navigating the complexity of the regulatory approval process.

Well you effect is differentiated matter to meet the needs of patients.

Biomarin leadership team I shouldn't supported with vision for creating a fully integrated company with technical operations powers clinical studies and commercial demand and there's an integral components, but these are technology development paradigm for sure inventory.

Robert Baffi: And third, let ingenuity create adaptability and resiliency in our approach to drug development. These three tenets, infused with the talents of the most dedicated people I've ever worked with, have consistently enabled us to take research ideas rapidly through development, navigating the complexity of the regulatory approval process in a highly effective and differentiated manner to meet the needs of patients. Biomarin's leadership team has shared in support of the vision for creating a fully integrated company with technical operations, powers, clinical studies, and commercial demands, and is an integral component of strategic technology development paradigms for assuring the timely delivery of an uninterrupted supply of products. Furthermore, innovative and appropriately implemented CMC strategies linked to a faster clinical design allow for rapid development and high success rates that benefit both patients and shareholders alike. As a company, we are going through multiple transitions simultaneously. Challenging in some ways, invigorating in others.

Only delivery of an auto interrupted supply problem.

Furthermore, innovative an appropriately implemented since he says.

Sure. That's the clinical design allows for rapid development and high success rate that benefit both patients and shareholders alike.

As a company we are going through multiple presentations simultaneously.

Challenging in some way invigorated dig in those.

Our transition to profitability. This year provides the resources to develop more innovation innovative therapies.

Transmission gene therapy product leverage is on clinical manufacturing and commercial capabilities have places us squarely OCO for from a good merging technological advancement of precision medicine.

Our transition technical operational leadership provides the opportunity to fill our innovative approach to drug development is a few our growth.

Well that first of all talk with great Diversey V. I couldn't help opium close with the scope of his responsibility and they experience all variety or diversity of his career.

In many ways wireless different journey and shared a lot of commonality with my own and that's not a lot as opposed to biomarin.

I'm confident and convert it does the transition and second cooperations a flow Moran from me to Greg will build on the legacy of science compliant.

For patients that will benefit from the products that will emerge from our efforts for employees and their career abroad shareholders as we become profitable.

Robert Baffi: Our transition to profitability this year provides the resources to develop more innovative therapies. Our transition to gene therapy product leverages our clinical manufacturing and commercial capabilities and places us squarely at the forefront of the emerging technological advancements in precision medicine. Our transition and technical operation leadership provides the opportunity to build on our innovative approach to drug development to fuel our growth. When I first saw Dr. Greg Geier at CV, I could not help but be impressed with the scope of his responsibility and the experiential variety and diversity of his career. In many ways, while a different journey, it shared a lot of commonality with my own, and not to allow those both to become Biomarin. I am confident and committed that the transition and technical operations of Biomarin will be successful.

In terms of licensor of our teams are the manufacturing facility in support of the I'm going to 70 approval.

Police this year that the health products regulatory authority of Ireland conducted on behalf of European Medicines Agency and Preapproval inspection into war.

This inspection involved the detailed review the facility witnessed process and analytical studies are relevant documentation generated in support of validation for such a person.

Following the it's fair to see GMP certification was granted allowing for commercial production and distribution of be amounted to 70, Andy you when the product is approved.

Our president inspectors that facility, but.

It's expected to be completed during Q2, allowing full licensure and the U.S. of the facilities consistent with the August 20 for Hadoop action date.

We have more than 400 doses of commercial beer amounted to 70 ready for potential launch later this year I'm remain very enthusiastic about the prospects for introducing the first gene therapy product for bleeding disorder, the hemophilia community as soon as possible.

Thank you for your support throughout my time at Biomarin and I'd like to turn the call over this.

For the commercial business update yup.

Thank you Robert.

As we begin 2020.

Robert Baffi: From me to Greg, we'll build on the legacy of science, compliance, and ingenuity. For our patients who will benefit from the products that will emerge from our efforts, for our employees and their careers, and for our shareholders as we become profitable. In terms of licensing of our gene therapy manufacturing facility in support of BMN 270 approval, I'm pleased to share that the Health Products Regulatory Authority of Ireland conducted, on behalf of the European Medicines Agency, a pre-approval inspection in Q1. This inspection involved a detailed review of the facility, equipment, process, and analytical studies and relevant documentation generated in support of validation, production, and testing. Following this inspection, a CGMP certification was granted, allowing for commercial production and distribution of BMN270 in the EU when the product is approved.

I'm very pleased with the team's performance.

It's all brands in all regions during the quarter.

Straight you mentioned, we achieved aerohive quarterly revenue on record.

Total revenues of $502 million in the first quarter with net product revenues marketed by Bob ran up 24% to $433 million.

This achievement reflects.

See fundamental strength and growth of our business like near term challenges related to cover that 19, which I will address in a moment.

Onto results in the quarter, it and starting with talent to.

In the U.S. the trend of increasing revenue based on the steadily growing base of patients on commercial therapy, including progression from induction and titration the daily maintenance dosing continued in Q1.

In the early part of the quarter, we did experience a seasonal slowing of new patient enrollment and patient starts somewhat mirroring our historical experience with ku band in the United States.

We are reporting $35 million in pounds the revenue for the first quarter, what's the majority of that revenue coming from the U.S.

Robert Baffi: At present, the inspection of the facility by FDA is expected to be completed during Q2, allowing for the license to sell the facility in the U.S. consistent with the August 21st PDUFA action. We have more than 400 doses of commercial VMA-270 ready for potential launch later this year and remain very enthusiastic about the prospects of introducing the first gene therapy product for a bleeding disorder to the hemophilia community as soon as possible. I thank you for your support throughout my time at Biomarin, and now I'd like to turn the call over to Jeff to discuss the commercial business update. Jeff?

In Europe in the first quarter multiple clinics across Germany continued to actively treating patients with Alan Tse and early uptake signals are encouraging.

During the quarter, we made significant progress in Germany, adding clinics and now have some experience prescribing palins IEC and managing patients through the induction and penetration phase two daily maintenance dosing.

As the number of commercial patients in Germany steadily grows we anticipate meaningful revenue contribution from that you starting this year.

We anticipate finalizing pricing reimbursement negotiations in Germany by mid this year and important steps toward getting pricing reimbursement approvals in other high priority European markets.

Jeffrey Robert Ajer: Do you have it? Thank you, Robert. As we begin 2020, I'm very pleased with the team's performance across all brands and all regions during the quarter. As JJ mentioned, we achieved our highest quarterly revenue on record. The total revenue of $502 million in the first quarter, with net product revenues marketed by Biomarin,

Ku band contributed $122 million and revenues in the quarter or 14% growth year over year with most of that growth coming from the United States.

Mmm revenues grew 9% year over year contributing $137 million on the first quarter driven by an 11% increase in patients year over year. This is reflective of the continued anticipated growth potential we expect for feminism.

Jeffrey Robert Ajer: Up 24% to $433 million.

Jeffrey Robert Ajer: This achievement reflects... the fundamental strength and growth of our business despite near-term challenges related to COVID-19, which I will address in a moment. On to results for the quarter, starting with Palantir. In the U.S., the trend of increasing revenue based on a steadily growing base of patients on commercial therapy, including progression from induction and titration to daily maintenance dosing, continued in Q1. In the early part of the quarter, we did experience a seasonal slowing of new patient enrollments and patient starts, somewhat mirroring our historical experience with Kuvan in the United States.

Turning to Naglazyme revenues totaled $114 million east, 32% year over year growth for the well established brand.

As with them as the impacts from uneven large order patterns makes a quarterly comparisons difficult.

A number of commercial patients on Bagless I'm grew by 6% in the past here and is indicative of the ongoing growth potential for this brand nearly 15 years since being approved.

And finally for Nora contributed $24 million and net product revenues, which represented 97% year over year growth.

These revenues were essentially flat over Q4 and that was driven by a modest yearend inventory build.

Jeffrey Robert Ajer: We are reporting $35 million in Palantik revenue for the first quarter, with the majority of that revenue coming from the U.S. In Europe, in the first quarter, multiple clinics across Germany continue to actively treat patients with Palantir, and early uptake signals are encouraged. During the quarter, we made significant progress in Germany, adding clinics that now have some experience prescribing Palantik and managing patients through the induction and titration phase to daily maintenance dosage. As the number of commercial patients in Germany steadily grows, we anticipate meaningful revenue contributions from the EU starting this year. We anticipate finalizing price and reimbursement negotiations in Germany by mid this year and important steps toward getting price and reimbursement approvals in other high-priority European markets. Two bands contributed $122 million in revenues in the quarter, or 14% growth year over year, with most of that growth coming from the United States.

In the EMEA region in Q4.

Importantly, the growth and bring her revenues compared to prior year reflects an underlying growth of 86% in commercial patients.

We are seeing a net increase in patients benefiting from burn or a treatment due to the success of our disease awareness and patient identification programs.

Taken together, we're pleased with first quarter results and demand for our products and while we experienced minimal financial impact in the first quarter due to cope with 19, we anticipate the potential for a higher degree of impact during the remainder of 2020 as disruptions of day to day operations clinics and.

Capital flow through our business.

Our global commercial teams will continue to adjust to implement innovative approaches to engage with clinics in patients to ensure continuity of access to our medicines, where possible. We're supporting home infusion efforts to help mitigate impact. However, some cobot 19 disruption to new patient starts.

As well as to ongoing infusion center visits from existing patients are expected to continue.

As a result, we are reducing total revenue guidance by 5% at the midpoint due between 18 50 million Didnt 19, 50 million for the full year 2020.

Jeffrey Robert Ajer: BIMISIM revenues grew 9% year-over-year, contributing $137 million in the first quarter, driven by an 11% increase in patients year-over-year. This is reflective of the continued, anticipated growth potential we expect for BIMISIM. Turning to Neclozyme, revenues totaled $114 million, a 32% year-over-year growth for the well-established brand. However, as with Vemisim, the impact of uneven large order patterns makes a quarterly comparison difficult.

Vast majority of todays update a total revenue guidance reflects adjustments to inline brands, including the Miss him Naglazyme and pallets seek and assumes our business will return to normalized demand patterns and the second half of 2020.

Although we did not give BMN to 72020 revenue guidance in February I worked 2020 total revenue guidance did assume some contribution from BMN to 70 in Europe.

Now I'd like to in my remarks, with an update on our hemophilia gene therapy program and introduce you to the intended brand name right Caribbean.

Jeffrey Robert Ajer: The number of commercial patients on Naglozyme grew by 6% in the past year, and this is indicative of the ongoing growth potential for this brand, nearly 15 years since it was approved. And finally, Bernoura contributed $24 million in net product revenues, which represented 97% year-over-year growth. These revenues were essentially flat over Q4, and that was driven by a modest year-end inventory bill in the UUMEA region and Q4.

In previous calls you've heard references to Valtrex, which was an abbreviated form of our I N or international Nonproprietary name Lucky Cogen Harbor back or Alternatively, a program identifier them into 70.

Both the F.D.A.M. may have accepted rock KPN as our brand name and we look forward to adopting rough pay being as we get closer to launch in the meantime, we will cease to use valtrex. So is there not confuse it with our intentions ran it.

Other key launch readiness activities of the continued to progress we have essentially built out the commercial team in the United States and have added key individuals.

Jeffrey Robert Ajer: Importantly, the growth in Brunnera revenues compared to the prior year reflects an underlying growth of 86% in commercial patients. We are seeing a net increase in patients benefiting from Bernoulli treatment due to the success of our disease awareness and patient identification program. Taken together, we are pleased with first quarter results and demand for our products.

Thank you.

The majority of these new employees have substantial and diverse experience in hemophilia.

Our brand campaigns also continue to develop as anticipated and teams a pivot into virtual and digital platforms, allowing for ongoing engagement with the marketplace.

You have face to face interactions.

Obviously certain activities are more amenable to virtual engagements and others and Workovers 19 is selling challenging us most in the short term is with gene therapy educational programs and site readiness.

Jeffrey Robert Ajer: And while we experienced minimal financial impact in the first quarter due to COVID-19, we anticipate the potential for a higher degree of impact during the remainder of 2020, as disruptions of day-to-day operations of clinics and hospitals flow through our business. Our global commercial teams will continue to adjust to implement innovative approaches to engage with clinics and patients to ensure continuity of access to our medicine. If possible, we are supporting home infusion efforts to help mitigate impacts. However, some COVID-19 disruption to new patient starts, as well as to ongoing infusion center visits from existing patients, is expected to continue. As a result, we are reducing total revenue guidance by 5% at the midpoint to between $1850 million and $1950 million for the full year 2020. The vast majority of today's updated total revenue guidance reflects adjustments to inline brands, including Vimazim, Naglazyme, and Palantik, and assumes our business will return to normalized demand patterns in the second half of 2020. Although we did not give BMN 270 2020 revenue guidance in February, our 2020 total revenue guidance did assume some contribution from BMN 270 in Europe.

Fortunately with the team already onboard we anticipate being well prepared the launch if we receive regulatory approval.

In the meantime, we've recently finished some very positive pricing research, which validated payer willingness to embrace rock they'd be in with the current data sets and we'll look forward to providing you with updates on our pricing of launch.

Thank you for your attention and I will now turn the call overhang to provide an R&D update thanks.

Thanks, Jeff.

I'd also like to Echo JJ expression of deep and heartfelt appreciation for the time, Robert it's not with US and also to welcome Greg dire to the organization, where the secret sauce is bottled.

The R&D organizations delighted that our next significant product opportunities can you continue to progress, particularly under the circumstances brought about by Cobot 19.

The ability to come to work, mostly virtually and focus on the advancement of our innovative products today.

<unk>, that's been especially gratifying and welcome distraction from the ongoing pandemic <unk>.

I want to acknowledge and thank our teams for their commitment in contributions during these challenging times.

I've been impressed by your flexibility and your ability to keep the story going while we are dealing with a pandemic.

Starting with rock Canyon, and what have strong intermodal memorable brand name congratulations.

Yeah, Hey is committed to meet the August 20% due to action date.

In Europe, our marketing authorization application filing remains on accelerated assessment at this time however.

Jeffrey Robert Ajer: Now I'd like to end my remarks with an update on our Haemophilia Gene Therapy Program and introduce you to the intended brand name, Roktavian. In previous calls, you have heard references to Valrox, which was an abbreviated form of our INN, or International Non-Proprietary Name, Velocitogein Roxaparvovec, or alternatively, our program identifier, BMN Both the FDA and EMA have accepted Roktavian as our brand name, and we look forward to adopting Roktavian as we get closer to launch. In the meantime, we will cease to use Valrox, so as not to confuse it with our intended brand name. Other key launch readiness activities have continued to progress. We have essentially built out the commercial team in the United States and have added key individuals as first priority.

Beauty procedure is to be extended by at least three months due to covert 19 delays.

Further as is the case with most filings that initially receive accelerated assessment. We believe there is a high possibility that aren't I mean.

We'll revert to a standard review procedures some accelerated assessment.

Based on these assumptions, we expect to see Tempe opinion by late twenties early 21.

We continue to plan to share a three or four your update.

60, 13 vector genome pattillo does as well as the three or update on the 40 13 vector genomes per kilo does in the middle of the year.

Form and has yet to be fine, giving a changing environment for medical meetings.

We haven't data analysis plan in place well move forward with business as usual, but the menu and met then even method for provide any update still fluid at this point so thanks for bearing with us.

Importantly, we do not expect cobot 19 to impact the timelines for completion of the rock TVN phase III trial.

Henry J. Fuchs: The majority of these new employees have substantial and diverse experience in hemophilia. Our brand campaigns also continue to develop as anticipated, and teams have pivoted to virtual and digital platforms, allowing for ongoing engagement with the marketplace in lieu of face-to-face interaction. Obviously, certain activities are more amenable to virtual engagements than others, and where COVID-19 is challenging us most in the short term is with gene therapy, educational programs, and site readiness. Fortunately, with the team already on board, we anticipate being well prepared to launch if we receive regulatory approval. In the meantime, we have recently finished some very positive pricing research, which validated payer willingness to embrace Rahtedion with the current dataset, and we'll look forward to providing you with updates on our pricing at launch. Thank you for your attention, and I will now turn the call over to Hank to provide an R&D update.

That was completed in November of last year, and one of the benefits of being a onetime treatment as that patients do not need received therapy electronic basis. We're also confident that the integrity of the ongoing data collection for the study for this pivotal study is being sufficiently maintain its home health care solutions, a nice aligns nicely with the collection of the primary endpoint.

Annualized bleed rate data.

Turning out of a short time for the treatment of any contemplation as Jay mentioned.

We plan to submit a global marketing applications in the third quarter this year or multi pronged development program, including a long term phase two clinical results in five to eight year old children comprehensive natural history data. The ongoing study of newborns to five years and highly statistically significant placebo controlled phase three trial makes.

For a very comprehensive data package spanning more than five years of treatment with children.

I would say contemplating.

Again, we are the beneficiary unfortunate timing and then our pivotal submission data read outs prior to the pandemic and now much of the work can be concluded remotely.

Henry J. Fuchs: Thanks, Jeff. I'd also like to echo JJ's expression of deep and heartfelt appreciation for the time Robert has spent with us and also to welcome Greg Geyer to the organization where the secret sauce is the bottle. The R&D organization is delighted that our next significant product opportunities continue to progress, particularly under the circumstances brought about by COVID-19. The ability to come to work, mostly virtually, and focus on the advancement of our innovative products to date, Octavian and Vesoritide, has been especially gratifying and a welcome distraction from the ongoing pandemic. I want to acknowledge and thank our teams for their commitment and contributions during these challenging times. I have been impressed by your flexibility and your ability to keep the story going while we are dealing with the pandemic. Starting with Roktavion, and what a strong and memorable brand name. Congratulations, Jeff.

If approved and sort of tied would be the person only medicine designated for the treatment of a contra. Please just in the U.S. and in European Union <unk>.

We continue to look for to publishing the full data from the phase three study later this year and we're pleased to let you know that our late breaker has been accepted an upcoming medical Congress.

Isn't patient will include a one year Gray philosophy, I see scores body proportionality sapiens subgroup analyses.

So stay tuned for more specifics.

As to when and where those data will appear.

The phase two study of US who are tied into your to five year olds referred to as studies to six is proceeding well and we're very pleased that safety data from children ages six months to five years participating in that study will be available as part of our registration package. We are grateful that the timing a key studies is aligned well with a free cobot 19 plans.

Moving to be mm 307, our investigational gene therapy for finicky urea, we're continuing to prepare a new sites to open in order to enroll patients one of the safely do so given the covenant can circumstances were excited about the prospect of the M. In three of seven as it represents a third treatment for penalty urea in our PK you franchise and the second Gen.

Henry J. Fuchs: The FDA is committed to meeting the August 21st PDUFA action date. In Europe, our marketing authorization application filing remains on accelerated assessment at this time. However, the review procedure is to be extended by at least three months due to COVID-19 delays. Furthermore, as is the case with most filings that initially receive an accelerated assessment, we believe there is a high possibility that our MAA will revert to a standard review procedure from an accelerated review procedure.

Therapy development program, leveraging our learnings and capabilities from receiving.

Currently we expect to study the start later, we expect to start the study later in 2020.

The R&D organizations energized by the opportunities before us and 2020.

It's my opinion for severe hemophilia, a and but short type of children, they contemplate advancing towards potential approvals.

Henry J. Fuchs: Based on these assumptions, we expect a CHMP opinion by late 20 or early 21. We continue to plan to share a four-year update of the 6E13 Vector Genome per Kilo dose, as well as the three-year update on the 4E13 Vector Genome per Kilo dose, in the middle of the year. But the form is as yet to be defined, given the changing environment for medical meetings.

Hopeful that these innovative treatments will be available in the very near future. We look forward to updating you on their progress over the coming quarters. Thank you for your continued support and I'll now turn the call over to Brian to review the financials.

Thank you Hey.

Please refer to today's press release summarizing our financial results for full detail on the first quarter of 2020 as usual our comprehensive report on the quarter will be available at our upcoming form 10-Q, which we are on track to file over the next couple of days.

Henry J. Fuchs: We have a data analysis plan in place. We'll move forward with business as usual, but the venue and method for providing the update are still fluid at this point. So, thanks for bearing with us.

Just imagine we are experiencing some modest impacts from the covert 19 pandemic. It as a result, we have updated full year total revenue guidance to between 1.85 billion to 1.95 billion as.

Henry J. Fuchs: Importantly, we do not expect COVID-19 to impact the timelines for completion of the Roktavian Phase III trial. Enrollment was completed in November of last year, and one of the benefits of being a one-time treatment is that patients do not need to receive therapy on a chronic basis. We are also confident that the integrity of the ongoing data collection for this pivotal study is being sufficiently maintained as home health care solutions align nicely with the collection of the primary endpoint annualized bleed rate data. Turning now to Vasoratide for the treatment of achondroplasia, as JJ mentioned, we plan to submit a global marketing application in the third quarter of this year. A multi-pronged development program including long-term Phase II clinical results Again, we're the beneficiary of fortunate timing, and then our pivotal submission data read out prior to the pandemic, and now much of the work can be concluded remotely. If approved, sorotide would be the first and only medicine designated for the treatment of achondroplasia in the U.S. and in the European Union.

As Jeff noted our updated revenue guidance is based on the assumption that our business will return to normalized demand patterns in the second half of the year.

Pardon me well, we lowered our revenue guidance due to the impact of Koby 19 at our commercial business, we were able to analyze our 2020 spend projection and make adjustments that allowed us to maintain our prior GAAP and non-GAAP income guidance, despite the lower revenue.

Moving to operating expenses R&D expense for the first quarter 2020, with 142 million and lower compared to R&D expense for the first quarter of 29 team of 184 million.

Mostly due to less R&D activity for rock TV, given its late stage development as well as pansy following its approvals in the U.S. in Europe.

<unk> expense for the first quarter of 20 was 187 million, which was higher than I've seen expense for the first quarter of 2019 of 162 million the year over year increase was expected with the single largest driver being the commercial preparation for the launch of rock Tasiast and the continued global launch a penalty.

We also incurred some unpredictable foreign currency exchange losses during the month as March at the Cobot pandemic negatively affected similar assets denominated in some of the more volatile global currency.

Turning to bottom line results, we reported GAAP net income of 81 million in the first quarter of 2020 compared to a GAAP net loss of 56.5 million in the first quarter of 29 team.

The improvement in GAAP income was primarily due to higher revenue lower R&D expenses and the gain on sale.

Yes.

With higher revenues and lower R&D expenses non-GAAP income of 117 million into first quarter of 2020 grew substantially as compared to Q1 2019, non-GAAP income of 25 million.

Both of these first quarter 2020 bottom line results gives us a great start towards achieving our 2020 goals of GAAP net income on an annual basis for the first time in company history and considerable growth and non-GAAP income.

I'd also like to touch on a potential tax benefit that we mentioned last quarter that may be recognized in the second half of this year. Our current 2020, GAAP net income guidance of between $20 million to $80 million excludes the potential impact of intra entity intangible asset transfers between biomarin entities.

Henry J. Fuchs: We continue to look forward to publishing the full data from the PACE re-study later this year, and we're pleased to let you know that our late-breaker has been accepted for an upcoming medical congress. The presentation will include one-year growth velocity, Heizzi scores, body proportionality, safety, and subgroup analyses. So stay tuned for more specifics as to when and where those data will appear. The Phase 2 study of Azorotide in 0-5-year-olds, referred to as Study 206, is proceeding well, and we are very pleased that safety data from children ages 6 months to 5 years participating in that study will be available as part of our registration package.

These intangible asset transfers occur we estimate that the tax effect could result in a onetime noncash income tax benefit of greater than $500 million and they mentioned previously you may have seen similar transactions completed by some of our larger peers in recent quarters.

Speaking of total cash and investments we ended the first quarter of 2020 with 1.15 billion compared to $1.17 billion at the end of December 2019.

The modest decrease in total cash and investments during Q1 2020 was largely due to some timing of operating cash flows. However is a significant improvement over the first quarter of 29 team or total cash and investments decreased by $105 million.

Henry J. Fuchs: We're grateful that the timing of key studies has aligned well with a free COVID-19 plan. Moving to BMN-307, our investigational gene therapy for phenylketonuria, we're continuing to prepare new sites to open in order to enroll patients when it is safe to do so given the COVID-19 circumstances. We're excited about the prospect of BMN-307 as it represents a third treatment for phenylketonuria in our PKU franchise and a second gene therapy development program leveraging our learnings and capabilities from Roktavian. However, currently, we expect the study to start later. We expect to start the study later in 2020. The R&D organization was energized by the opportunities before us in 2020, with both Lactavian for severe hemophilia A and Vasorotype for children with achondroplasia advancing towards potential approvals. We are hopeful that these innovative treatments will be available in the very near future. We look forward to updating you on our progress over the coming quarters, and thank you for your continued support. And I'll now turn the call over to Brian to review the financials.

This solid cash position, coupled with vibrant business fundamentals put us in good standing to manage through the continued uncertainty related to covert 19.

In closing the strong performance of the business during the first quarter of 2020, plus our positive financial outlook for the rest of the year indicate that 2020 should be a transformational year for the company and the prospects is valued to come from TV in the store type is there to commercially give us enthusiasm about our future.

Thank you for your support and we will now open the call to your question.

Operator.

Thank you get asked the question via telephone. Please press star one if he would like to address your question. That's the town key please limit yourself to ask one question.

Our first question if some of the line up Robyn Curnow Cartus cost from Suntrust. Your line is now open.

Hi, everyone. Thanks for taking my question and the first off congratulations to grade, but to Rod I was lovely working with you I think you brought up a breath of fresh air Sea working with hasn't seen in all of my buys the camera. So thanks, so much I learned a lot.

I guess in itself with some questions on Roku, TV and I hope I get that correctly. So first of all what gives you confidence that five in the United States that there won't be any more delays people ask me. This non stop and then when you talk about assuming normal operations go on resuming.

Second half is that in the beginning of the second half do you have a timeline for that if it goes into fourth quarter could we see further delays.

Third question isn't what are you hearing as far as like people willing to has the gene therapy procedures died in a coping environment.

Brian R. Mueller: Thank you.

You know as early as fourth quarter or people open to it is separate from the hospitals or what are you hearing on from the ground. Thank you.

Brian R. Mueller: Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the first quarter of 2020. And as usual, our comprehensive report on the quarter will be available in our upcoming Form 10-Q, which we are on track to file over the next couple of days. As Jeff mentioned, we are experiencing some modest impacts from the COVID-19 pandemic, and as a result, we have updated full-year total revenue guidance to between $1.85 billion and $1.95 billion. As Jeff noted, our updated revenue guidance is based on the assumption that our business will return to normalized demand patterns in the second half of the year. Importantly, while we lowered our revenue guidance due to the impact of COVID-19 on our commercial business, we were able to analyze our 2020 spend projection and make adjustments that allowed us to maintain our prior GAAP and non-GAAP income guidance despite the lower revenue.

Got you want you answered a question on the on the no delays with yet.

Okay.

Thank you thank them for the questions.

The confidence from Yankee I guess that we're tracking to our milestones and in some cases, they're accelerating their work.

So having been through a bunch of these recently all signs are pointing.

Favorably.

As far as willingness to dose as you know we completed our dosing in our bell lots clinical trials and we're experiencing a bit of a delay in the peak and you program. So I do expect comparison has an intimate gene therapy clinical trial World I don't know if Jeff major wants to make any comments about anything he's heard about what might happen in the fourth quarter one.

Were approved.

Yeah, I guess you want give your perspective on you know how we launched a product I mean, obviously, if we have to launch today, he would be a little bit challenging, but but we know.

Seeing newsy, we few minutes that the the best I mean this isn't there are starting to reopen in the U.S.

So and around the World, Germany, France, France is what will we opening their schools in a couple of weeks so things are changing not.

Hard to predict.

When things are really going to go back to normal or exposed to normalize as possible.

We cannot anticipate this to happen you know.

At the beginning of a third quarter, but it's a it's hard to tell today, but but Jeff you want to give your perspective on the willingness to patients to be treated and.

Brian R. Mueller: Moving to operating expenses, R&D expense for the first quarter of 2020 was $142 million, and it was lower compared to R&D expense for the first quarter of 2019 of $184 million. Mostly due to less R&D activity for Roktavion, given its late stage of development, as well as Palantzik following its approvals in the U.S. SG&A expense for the first quarter of 20 was $187 million, which was higher than SG&A expense for the first quarter of 2019 of $162 million. The year-over-year increase was expected, with the single largest driver being the commercial preparation for the launch of Roktavion and the continued global launch of Palantir. We also incurred some unpredicted foreign currency exchange losses during the month of March as the COVID pandemic negatively affected some of our assets denominated in some of the more volatile global currencies. During the bottom line results, we reported a gap net income of $81 million in the first quarter of 2020, compared to a gap net loss of $56.5 million in the first quarter of 2019. The improvement in GAAP income was primarily due to higher revenue, lower R&D expenses, and the gain on the sale of the first quarter.

And how our commercial team is going to go about addressing this.

Yeah, It's a great question and of course, nobody can predict the future. So our team has been focused and maybe I'd start with saying Fortunately we have a team in the United States that is comprised largely of people with existing relationships and the hemophilia community.

Those people are busy making connections with clinicians with.

Hemophilia treatment centers and with patient advocacy organizations, all with the objective of continuing to introduce Biomarin as an organization.

To those audiences.

And provide opportunities for gene therapy education, and very important and highly in demand and community and also working on site readiness I think one of the most decisive variables will be the ability and willingness or not.

Certain hospitals to do infusions.

If we are so fortunate to have an approved a rock baby and product in Q4 that will probably be somewhat dependent on the course of the cobot 19 situation.

But we would also note that oh, well, we've seen a lot of disruption to our our infusions of the enzyme products the disruption hasn't been anything close to total and in fact, we've seen less disruption with Brunner, a infusions, which.

Brian R. Mueller: Word. Document.8

Brian R. Mueller: With higher revenues and lower R&D expenses, non-GAAP income of $117 million in the first quarter of 2020 grew substantially as compared to Q1 2019 non-GAAP income of $25 million. Both of these first quarter 2020 bottom line results give us a great start towards achieving our 2020 goals of achieving gap net income on an annual basis for the first time in the company's history and considerable growth in non-gap income. I'd also like to touch on a potential tax benefit that we mentioned last quarter that may be recognized in the second half of this year. Our current 2020 gap net income guidance of between $20 million and $80 million excludes the potential impact of intra-entity intangible asset transfers between Biomarin and Biomarin. If these intangible asset transfers occur, we estimate that the tax effect could result in a one-time, non-cash income tax benefit of greater than $500 million.

Our indicative of the the situation, where hospitals and physicians or can prioritize in but infusions of if they're motivated and then and they find them or to be particularly of import.

So that's our approach for rock baby in pursuing education site readiness, and we'll see where we can do infusions when we get that far I personally haven't gotten a signal that that patients are going to be and Washington confusions one.

As we get that far and that may be reflective of the fact that are you know our our patient engagement is pretty limited in the pre approval setting.

I'll turn that back over to to you Jay Jay if you're on a direct on the resumed normal question.

I mean, I can't get up to try to answer that maybe you want to give your perspective on that was used to normal.

Yep.

Yes.

So robin I don't think that we're saying that that our business will be back to a 100% a normal I'm starting the second half I think we're using that the second half to give some flexibility in terms of the timing of that.

My own senses that we're we're expecting to see the biggest impact to our business in Q2, we would be rebounding in Q3 and with some luck we wouldn't be back to normal prequel good levels of demand and in Q4.

Brian R. Mueller: As I mentioned previously, you may have seen similar transactions completed by some of our larger peers in recent quarters. Speaking of total cash and investment, we ended the first quarter of 2020 with $1.15 billion compared to $1.17 billion at the end of December 2019. The modest decrease in total cash and investments during Q1 2020 was largely due to some timing of operating cash flows. However, it is a significant improvement over the first quarter of 2019, when total cash and investments decreased by $105 million. This solid cash position, coupled with Biomarin's business fundamentals, puts us in good standing to manage through the continued uncertainty related to COVID-19. In closing, the strong performance of the business during the first quarter of 2020 plus our positive financial outlook for the rest of the year indicate that 2020 should be a transformational year for the company. And the prospect of value to come from Roktevian and Besoratine if they are approved commercially gives us enthusiasm about our... Thank you for your support, and we will now open the call to your questions. Operator.

Thank you.

We have our next question from the line of Savi enriched <unk> from Goldman Sachs. Your line is now open.

Good afternoon, and thanks for taking my questions and Robert Good luck with everything I'm glad you must come with Alex launch can you comment on patient screening such as the website. That's sitting at about 400 individuals I believe back in February and an update on the companion diagnostic and then secondly, how should we think about before.

Your phase one.

Turning data and do you anticipate it'll impact payer discussions.

[laughter] you want to answer the companion diagnostic or things that movie going on here and.

Oh, and then you had a question on the phase two.

She also because yeah and the up than that.

I can start with the companion diagnostic enough, where your update and then turn it back you guys for.

The impact on payers or the Hopkins companion diagnostic solving I'm doing great Everything's on track fully expect to have an approvable companion diagnostic and some valorous accrual in the United States fully expect to have it CE Mark for Europe for use in all in Europe, when when when rock Cavium excuse me as group poor.

Your update on one rock TV in the expectation is a main focus is gonna be obviously on bleed control and and secondary emphasis is gonna be on evolving pattern of factor expression and whether there anything or in individual patient said mark.

Who is were vulnerable to loosen factor expression, if that's occurring or not.

So.

As you know the play it's been overseen by a data monitoring committee ER and they have not recorded nothing, particularly one way or the other two us about 30 to a one so we continue to fall. The patients says the protocol described and look forward presenting the data in the middle there.

Operator: Thank you, sir. To ask a question via the telephone, please press star 1. If you would like to withdraw your question, press the pound key. Please limit yourself to asking one question. Our first question is from the line of Robyn Karnauskas from SunTrust. Your line is now open.

So Jeff you want to talk about he is the payers and in fact, I mean to defeat to update impact on like they're off on the parents.

Yeah. Thank you Jane Thanks, I'll being for the question.

For your data point is an important.

JJ Bien-Aime: Hi everyone, thanks for taking my question, and just first off, congratulations to Greg and to Rob. It was lovely working with you; I think you brought a breath of fresh air to working with management teams in all of my biotech universes, so thank you so much. I guess I'm going to start off with some questions on Roktavion. I hope I got that correctly. So, first of all, what gives you confidence that in the United States there won't be any more delays, people ask me this non-stop, and then when you talk about assuming normal operations go, resume in the second half, is that at the beginning of the second half, do you have a timeline for that, if it goes into the fourth quarter, could we see further delays? And thirdly, what are you hearing as far as people willing to have gene Thank you.

Part of the emerging clinical data picture I think I've mentioned on the previous call that or our apparel launch was the gun in Q4 of 2019, So we've got a full.

Pay or they're management team on the ground.

They been they've been conducting calls for the last four months.

And we've been using the data that we have we've been using the three year data from last spring and we've been using the.

Data from that generate one interim data cut that's a data that we have a payers are interested in the question of.

Durability, that's a key question for them I haven't gotten any signals from payers that they think that Ah, but the answer to that question as a binary one based on what we see mid year from a four year data cuts that obviously that will be an important part of the picture and airs care about cook the question of durability.

We have my next question from the line of Josh Schimmer from Evercore ISI. Your line is now open.

Thanks for taking my questions have you started to see any change.

Days receivable in the started the second quarter and how do you expect that to evolve.

But of course, the your and can you comment a little bit on Sunday I insurance coverage shifts you maybe seeing in the U.S. you think that may impact the business if at all.

JJ Bien-Aime: Okay, hi, it's Yon. Do you want to answer the question on no delays with the FDA?

Henry J. Fuchs: Thanks Robin for the questions. The confidence from the FDA is that we're tracking to our milestones, and in some cases, they're accelerating their work. So having been through a bunch of these recently, all the signs are pointing favorably. As far as willingness to dose, as you know, we completed our dosing in our Valrox clinical trials, and we're experiencing a bit of a delay in the PKU program. I do expect that there is some hesitance in the gene therapy clinical trial world. I don't know if Jeff Bader wants to make any comments about anything he's heard about what might happen in the fourth quarter when we're approved.

Hey, Brian you want to answer the new with you, but questions and then see a insurance.

Thanks.

Of course, yeah. Thanks, Josh we're watching that closely including throughout March and April here, we've not yet seen actual disruption to our payments or you know we've got a stable base of customers stable supply chain in relationship from a cash flow standpoint with those customers. So.

We're hopeful that that will continue but you know recognize that there's got to be some uncertainty with a pandemic and its impact on health care system. So we're watching it but no impact to date.

And Josh.

Jack.

So your mix.

Yes, like other companies, where we're very watchful for.

Signals that look like patients are losing or access to private insurance because of being laid off and what happens to those are those families and patience I I would say, we don't have anything that constitutes a signal on that.

Jeffrey Robert Ajer: Yeah, Jeff, you want to give your perspective on, you know, how we would launch the product? I mean, obviously, if we had to launch today, it would be a little bit challenging. But, you know, we're seeing news every few minutes that businesses are starting to reopen in the U.S. And around the world, Germany, France, are reopening their schools in a couple weeks. So things are changing now. You know, it's very hard to predict. When things are really going to go back to normal, or as close to normal as possible, we kind of anticipate this to happen, you know, at the beginning of the third quarter, but it's hard to tell today. But, Jeff, would you like to give your perspective on the willingness of patients to be treated and how our commercial team is going to go about addressing this?

To this point there been a couple of anecdotal reports.

We've collected and where deliberately collecting.

Any signals that we can from our rare connections or hub.

Program. So we've gotten anecdotes of patients calling in thing that they're anxious about the potential lots of insurance, we've seen one patient a family shift from a private to a government plan due to loss for the job and so I would call those anecdotes and not a signal, but we'll keep watching.

If you posted good question. Thank you.

Yeah. My next question from the line of comedy Kasimov from JP Morgan. Your line is now open.

JJ Bien-Aime: Yeah, it's a great question. And, of course, nobody can predict the future. So our team has been focused. And maybe I'd start by saying, fortunately, we have a team in the United States that is comprised largely of people with existing relationships in the hemophilia community. So those people are busy making connections. I think one of the most decisive variables will be the ability and willingness or not of certain hospitals to do infusions. If we are so fortunate to have an approved Roctavian product in Q4, that will probably be somewhat dependent on the course of the COVID-19 situation. But we would also note that while we've seen a lot of disruption to our infusions of the enzyme products, the disruption hasn't been anything close to total.

Hey, good afternoon, guys. Thanks for taking the question and congrats to Robert on your retirement. So my questions for Hank I'm just curious if there.

The reason other than coded why the E.M.A. may revert the rock Kb in M&A to standard review from accelerated assessment, especially since you already successfully completed the a the pre approval inspection I mean was there any new data or new analyses that were requested by the agency anything else you can say there.

I think the key thing about accelerated assessment is that most products fall off accelerated the stuff assessments and I think that yeah. This is also further compounded by the fact that this is an advance medicinal agent.

So I don't think todays comments about accelerated assessment should be interpreted meaningfully differently than when we initially filed the application.

Okay. Thank you.

Yes.

Your next question is from the line of traffic Niekamp from ebay. Your line is open.

JJ Bien-Aime: And in fact, we've seen less disruption with Brunura infusions, which is indicative of the situation where hospitals and physicians can prioritize infusions if they're motivated and they find them to be particularly important. So that's our approach for Roctavian, pursuing education, site readiness, and we'll see where we can do infusions when we get that far. I personally haven't gotten a signal that patients are going to be reluctant to take infusions when we get that far, and that may be reflective of the fact that our patient engagement is pretty limited in the pre-approval setting. I'll turn that back over to you, JJ, if you want to direct the resume to normal questions.

Hey, guys. Thanks for the question.

Just one that's a little bit more about the dynamics in the base business I guess, when you think about the diagnostic path, where new patients on a number of different products via then as then brunner, yeah pounds et cetera.

Obviously, it's a pretty long cycle I just want to ask if you've seen any these physician interactions are coming to converting to virtual I'm just trying to assess the would be the long term disruption to the new starts beyond say you know three quarter third quarter. This year. Thank you.

Got you want to take that one.

Yeah, I'd be happy too.

So relative to the the base business and the dynamics I would say, we anticipate the near term or impact from Misted infusions to be higher than the impact from delayed.

Jeffrey Robert Ajer: No, I mean, I think I kind of tried to answer that. Maybe you want to give your perspective on the resume to normal? Jeff?

Ah patient identification and patient starts.

Probably the ER the disruption to patient starts would be larger for Palin Z at this point because.

Jeffrey Robert Ajer: Yes. So, Robyn, I don't think that we're saying that our business will be back to 100% normal by the second half. I think we're using the second half to give some flexibility in terms of the timing of that. My own sense is that we're expecting to see the biggest impact on our business in Q2. We would be rebounding in Q3, and with some luck, we would be back to normal pre-COVID levels of demand in Q4.

Them assume all of them as Bagless I'm in Brunner APA have a larger starting base of patient so the incremental new patients coming on why the smaller impact.

And there are anecdotal reports.

Clinics are shifting their their interactions with patients to to virtual visits that's something that is obviously outside of.

Biomarin per view, so <unk>, we only have a incidental reports on that and that's not something that we would see to get involved in.

Jeffrey Robert Ajer: Thank you.

Operator: We have our next question from the line of Salveen Richter from Goldman Sachs. Your line is now open. Good afternoon, and thanks for taking my questions. And Robert, good luck with everything.

However, I think for for certain aspects.

Of the identification and patient start pathway, yeah, we do see patients are moving forward.

Jeffrey Robert Ajer: So as you look towards the Valrox launch, can you comment on patient screening, such as the website that stood at about 400 individuals, I believe, back in February, and an update on the companion diagnostic? And then, secondly, how should we think about the four-year phase one, two upcoming data? And do you anticipate it'll impact payer discussions?

So the that process is not ground do a halt exactly but it but it has been disrupted.

Thanks, Jeff for that question I Hope I've I've addressed your concerns.

Next question, if something line of Akash to worry from Wolfe Research. Your line is now open.

Hey, Thanks, so much cob can you give some color on how the FDA may use that you Prophy trial inbound right, you've recently announced what do the possible to have that data with your phase two data as a part of a separate efficacy analysis is getting their label and how long would naturally.

Henry J. Fuchs: Uh, Hank, do you want to answer the companion diagnostic or things that we didn't all hear and, uh... And then you had a question about the phase two updates.

Henry J. Fuchs: I can start with the companion diagnostic and the four-year update and then turn it back to you guys for the impact on payers or the opt-ins. Companion diagnostic, Salveen, doing great. Everything is on track. I fully expect to have an approvable companion diagnostic at the time of Valrock's approval in the United States. I fully expect to have it CE marked for use in Europe when Rocktavian is approved. For a four year update on Rocktavian, the expectation is that the main focus is going to be obviously on bleed control, and secondary emphasis is going to be on the evolving pattern of factor expression and whether there are any things in individual patients that mark who were vulnerable to losing factor expression if that's occurring or not. As you know, the study has been overseen by a data monitoring committee, and they have not reported anything particularly one way or the other to us about study 201, so we continue to follow the patients as the protocol describes and look forward to presenting the data in the middle of the year.

And I just wanted to clarify something you're 2020 guidance included the impact of balance.

Only in Europe, I, just wanted to make sure I I quickly. Thanks.

So.

On the yeah.

Yep Yep Yep.

You know that study started is gonna be delayed because of the pandemic and in any case.

It was ancillary to the initial application and registration the way I think about it is.

In the initial reviews that the agencies are looking at they have.

Some data in patients treated with on demand steroid they have some patients that are treated with.

Ah prophylactic steroid doctors are kind of split some something like prophy something like on demand. We've just dose the 130 patient clinical trial with on demand a steroid use and so therefore, we're thinking about increasing the sample size of the experience that we have.

With Prophy steroid use when those study data are available certainly make them available medically and medical communications and then we'd think about also you know how that might be tied together with a phase one two prophy study to support a label supplement that's pretty far down the road from where we are and I think we have.

Jeffrey Robert Ajer: So Jeff, you want to talk about the payers and the impact, I mean, the Phase 2 update impact or lack thereof?

Jeffrey Robert Ajer: Yeah, thank you JJ, and thanks Salveen for the question. The four-year data point is an important part of the emerging clinical data picture. I think I mentioned on the previous call that our payer launch was begun in Q4 of 2019, so we've got a full payer management team on the ground, and we've been using the data that we have. We've been using the three-year data from last spring, and we've been using the data from the Generate One interim data cut. That's all the data that we have. Payers are interested in the question of durability. That's a key issue for them. I haven't gotten any signals from payers that they think that the answer to that question is a binary one based on what we see mid-year from the four-year data cut. But obviously, that will be an important part of the picture, and payers care about the question of durability.

We're really strong data package at lunch in regard to steroids and how to use them.

It's like any of the guidance the casualty so two big make a key or the original 2020 guidance, which we gave when we put a Q4.

It could at some anticipated revenues and by Rocky in Europe. The revised guidance would you give me today. It's you must know Barack so that you didn't drop because now we anticipate that actually media.

The approval in Europe, we very convenient a bit here early next year. So that's that's pike.

We have although we haven't given where the virus number was just one that you know that.

The new guidance assumes no revenues with my rocks in Europe.

Okay, then right.

We have my next question from the lineup, Chris we mined from Piper Sandler Your line is now okay.

Thanks, I, just wanted to drill down a little bit Tom on pounds IEC.

Jeff I think I heard your commentary and the slowdown in new starts happening at the beginning in the quarter <unk>.

And if memory serves I think there was a bit of a slowdown that started it also in Q4.

And just noting that you know when you look at the product specific guidance changes that this kind of stands out as I think by my math, you know midpoint to midpoint is down 10%, whereas the others that changed were down last.

Operator: We have our next question from the line of Josh Schimmer from Evercore ISI. Your line is now open. Thanks for taking the questions.

Just I wonder if you could just maybe give a little bit more color is this.

Is there something more endemic I guess going on with or without peak. Thanks.

Thanks for the question, Chris So in the Q1 earnings call with pointed to a little bit of a seasonal slowdown in Q4 per pound Zeke in terms of patient starts and ER and patient enrollments and we've historically seen that also ku band.

Brian R. Mueller: Have you started to see any change in the days receivable at the start of the second quarter and how do you expect that to evolve over the course of the year? And can you comment a little bit on the insurance cover?

Jeffrey Robert Ajer: Hey Brian, do you want to answer the day receivable questions and then Jeff the insurance? Next.

That's basically a seasonal.

Brian R. Mueller: Of course, yeah, thanks, Josh. We're watching that closely, including throughout March and April here. We've not yet seen...

The holidays, so from from Thanksgiving in late November through Christmas and new year, we see disruption to clinic activity and slowed a patient enrollments. So we saw a little bit of that in Q4.

unknown: https://www.patreon.com

unknown: and Josh White.

Jeffrey Robert Ajer: Yes, like other companies, we're very watchful for signals that look like patients are losing access to private insurance because of being laid off and what happens to those families and patients. I would say we don't have anything that constitutes a signal on that, at this point. There have been a couple of anecdotal reports that we've collected, and we are deliberately collecting. Any signals that we can from our Rare Connections Hub program. So we've gotten anecdotes of patients calling and saying that they're anxious about the potential loss of insurance. We've seen one patient family shift from a private to a government plan due to the loss of a job. And so I would call those anecdotes and not a signal. But we'll keep watching and keep you posted. Good question. Thank you.

For both Ku band and and pounds. So you can obviously our focus now is on promoting pounds eggs is why we addressed it and similarly in Q1 recall, we've historically seen slowdowns in our business for four Ku band and we're seeing kind of a similar pattern in the early part of Q1, that's not.

Related to the holidays in Q4, that's related to the reset of insurance policies.

Co pays accumulators.

And in some cases patient switching to new policies and and lot of cases, the need for a new prior authorization for patients.

That was really in early Q1 phenomenon by February we were seeing new patient enrollments and starts picking up.

That was really an encouraging sign four pounds. The in in February and March and then as a as the clinics in the United States in Germany began to be shut down in in March from the Covance situation.

Operator: We have our next question from the line of Corey Kosimov from JP Morgan. Your line is now open.

Jeffrey Robert Ajer: Hey, good afternoon guys. Thanks for taking the time to answer the question.

Jeffrey Robert Ajer: Congratulations to Robert on your retirement. So my question is for Hank. I'm just curious, is there a

We're experiencing now a real slow down and patient enrollments and patient starts and I expect that'll continue largely until but clinics or backup and are able to continue their operations again, recalling that with how lindsay.

Henry J. Fuchs: Is there a reason, other than COVID, why the EMA may revert Rockabian MSI?

Henry J. Fuchs: M.A.A. to Standard Review from Accelerated Assessment, especially since you've already succeeded.

Henry J. Fuchs: And that's how we successfully completed the pre-approval inspection. Was there any new data or new analyses that were requested by the agency? Anything else you can say there?

Patient starts are directed to be under the supervision of healthcare professional it can manage a potential adverse events for at least for the first injection.

Henry J. Fuchs: I think the key thing about accelerated assessment is that most products fall off the accelerated assessment, and I think that this is also further compounded by the fact that this is an advanced medicinal product, so I don't think today's comment about accelerated assessment should be interpreted meaningfully differently than when we initially filed the application. Okay, thank you. Your next question is from the line of Jeff Meacham from eBay. Your line is now open.

I hope that answers your question, Chris will talk about anything you know both the patients and the conditions have to be certified that they they can not sufficient get received the product.

And no herself and checked and then.

No the physician can no no about how managing the potential study that's why there's definitely its has been in late March and April a slowdown in the new patients, but we believe it's temporary and when the PK PD reopen GLP ones you an acceleration of.

New patients here.

Operator: Hey guys, thanks for the question. I just wanted to ask a little bit more about the dynamics of the base business. I guess when you think about the diagnostic path for new patients on a number of different products, you know, be it Venezen, Vernaria, Palenzie, etc., it's a pretty long cycle. I just wanted to ask you if you've seen any of these physician interactions coming to life and converting to virtual. I'm just trying to assess

We have our next question from the line of cell NATO from Cowen and company. Your line is now open.

Good afternoon. Thanks for taking my questions are congrats on the progress I also want to ask a question on the guidance looks like the vendors in guidance was cut by about 30 million and magazine by 20 I'm. Just curious one what have you been seeing in April how far apart can those infusions be pushed before efficiency.

I see some impact on on their symptoms.

In two or what proportion of patients worldwide are able to get these infusions that at home and so therefore, maybe won't have much of a disruption to use.

Jeffrey Robert Ajer: The

Jeffrey Robert Ajer: We'll get a long-term disruption to new starts. Beyonce, you know, the third quarter, third quarter of this year. Thank you.

So Jeff.

Tim. Thank you for the question I'm, So and you know that the reduction in guidance on them and Sim and that goes I'm is is proportional to the base of sales what we're talking about so what we're saying is a relatively uniform impact so far.

Jeffrey Robert Ajer: Jeff, do you want to take that one? I'd be happy to.

Jeffrey Robert Ajer: So, relative to the base business and the dynamics, I would say we anticipate the near-term impact from missed infusions to be higher than the impact from delayed Patient Identification and Patient Starts. Probably, the disruption to patient starts would be larger for Palanzec at this point because all of Vimazum, Maglazyme, and Brunnera have a larger starting base of patients. So the incremental new patients coming on board will have a smaller impact. And there are anecdotal reports that clinics are shifting their interactions with patients to virtual visits. That's something that is obviously outside of Biomarin's purview, so we only have incidental reports on that, and that's not something that we would seek to get involved in. However, I think for certain aspects of the identification and patient start pathway, we do see patients moving forward. So that process has not ground to a halt exactly, but it has been disrupted. And thanks, Jeff, for that question. I hope I've addressed your concerns.

Our.

Relative to the size of the business for each of not goes on men and women Sam.

We're seeing more disruption in home and Oh, sorry, more disruption in loss infusions in markets, where a home infusion is not a well established a practice. So we know normally that home infusion.

Where home infusion is.

And established practice that compliance rates are higher and we've seen less disruption in those markets, where patients are home and views that we've got to very diverse global business for both Magals I'm in women's them. So I don't want or try to report high level numbers of what percent.

A patients or or home and views or or not but our tactics have been.

In markets, where tactics to intervene and try to keep our business on track in markets, where we've got home infusion as an established practice, we're trying to push more patients into that channel and that channel is relatively open in markets, where home infusion is not miss that best practice.

Operator: The next question is from the line of Akash Tewari from Wolf Research. Your line is now open. Hey, thanks so much.

Henry J. Fuchs: Can you give some color on how the FDA may use that new PROFI trial on Valrox you recently announced? Would it be possible to have that data pooled with your Phase 1-2 data as part of a separate efficacy analysis in your label? And how long would that take to happen? And I just want to clarify something. Your 2020 guidance included the impact of Valrox, which sails only in Europe. I just wanted to make sure I

We see this is an opportunity in some places to try to get it established and so we've had some success in places like Brazil.

Brazil and Argentina.

In Colombia.

And in Spain for example in pushing patients into home infusion were assigned to establish practice for alternate infusion sites those being maybe a smaller hospital, but it's closer to home less impacted by cobot 19 situation for and.

JJ Bien-Aime: So, uh, how do you want to start on the FD?

Some cases, something like a retail and infusion clinic, where patients can go.

Henry J. Fuchs: Hi Akash. You know, that study's start is going to be delayed because of the pandemic, and in any case, it was ancillary to the initial application and registration. The way I think about it is, in the initial reviews that the agencies are looking at, they have some data on patients treated with on-demand steroids; they have some patients that are treated with prophylactic steroids. Doctors are kind of split, some like prophy, and some like on-demand. We've just dosed 130 patients in a clinical trial with on-demand steroid use, and so we were thinking about increasing the sample size of the experience that we have with prophy steroid use. When those study data are available, we would certainly make them available medically, in medical communications, and then we'd think about also how that might be tied together with the Phase 1-2 prophy study to support a label supplement. That's pretty far down the road from where we are, and I think we have a really strong data package at launch in regard to steroids and how to use them.

And we're seeing some positive effect of that already on our business.

I'm not going to Simon demonstrating our products, where we know.

Patients have not always been.

I'm completely compliant, so probably missing one or or several infusions is.

You know may not be noticeable to the patient or the physician.

For burn or on the other hand every every infusion can be critical and we're fortunately seeing a much higher level of compliance and less disruption was that part of the business, but I hope I've addressed your question there Tim Thank you and actually if I mean, I'd actually is the the virus situation, it's probably a match.

She's going to be helpful.

To actually move a lot of the treatment from the <unk> into its countries that are behind us in western Europe.

For the treatment than infusions from the hospitals into either their homes or places that are closer to the patients which actually overall, it's good news on might actually improve compliance like long term.

Yeah. My next question from the line of Matthew Harrison from Morgan Stanley. Your line is now open.

Great. Good afternoon. Thanks for taking my question just too quickly from me I guess for Hank Hank I didn't see a comment in the press release and I'm sorry, if I missed that maybe you commented during your remarks, but any impact to enrollment.

JJ Bien-Aime: So again, in the guidance, Akash, to make it clear, our original 2020 guidance, which we gave when we reported Q4, included some anticipated revenues from Varrox in Europe. The revised guidance, which is given today, assumes no revenues from Varrox in Europe, because now we anticipate that actually getting approval in Europe will be at the very end of this year or early next year. So that's fine. Although we haven't given where the VAROX number was, we just want to let you know that the new guidance assumes no revenues for VAROX in Europe.

In the last group of patients that you need for the full pivotal study for Val rocks and then.

On PK Eugene therapy. He is there anything you can do by adding some more sites or anything he may be speed up or reduce the delay that you're going to phase here before you start or the basically just the delay until you until you're able to start thanks.

Oh, Hi, net to seven the I'm sorry, your opinion phase three enrollment I think you're asking about we completed in November and so.

We're not presently enrolling anyone further as regards our initial approval. So good news on rock TV in the confirmatory trial are fully enrolled.

Operator: We have our next question from the line of Chris Raymond from Piper Sandler. Your line is now open.

And as far as the sort of the generalized restart we can we have a lot of conversations with investigators and there's reopening happening at different within different ways and in different places at different times, and we're really trying to get a handle on where whereas.

Jeffrey Robert Ajer: Thanks. I just wanted to drill down a little bit on Palantik.

Jeffrey Robert Ajer: So, Jeff, I think I heard your commentary on the slowdown in new starts happening at the beginning of the quarter. As memory serves, I think there was a bit of a slowdown that started in Q4. And just noting that, you know, when you look at the product-specific guidance changes, this kind of stands out as, I think, by my math, it's midpoint to midpoint. Midpoint is down 10%, whereas the others that you changed were down less. I wonder if you could just maybe give it a little bit more color.

Site, it's gonna be able to dose the first patients on the resumption of clinical trials don't exactly have clarity on when that's going to be or where that is gonna be.

Next question, it's on the line of Paul Maddie from Stifel. Your line is now open.

Thanks. This is Alex on for Paul Thanks for taking the question just wanted to see how you're thinking has evolved on paper performance for gene therapy isn't as a model in the U.S. Thanks.

Jeffrey Robert Ajer: Is this... You know, is there something more endemic, I guess, going on with Poundsy? Thanks for the question, Chris. So in the Q1 earnings call, we pointed to a little bit of a seasonal slowdown in Q4 for Palanzec in terms of patient starts and patient enrollments, and we have historically seen that also with QVAN. That's basically a seasonal phenomenon. Dr. John Aucott, Co-Founder & CEO, Pallantheque Medical Center, www.pallanthequemedicalcenter.com. That was really an early Q1 phenomenon. By February, we were seeing new patient enrollments and things picking up. That was really an encouraging sign for Palenzie in February and March. But then as the clinics in the United States and Germany began to be shut down in March due to the COVID situation, we're experiencing now a real slowdown in patient enrollments and patient starts.

Yes, you only address I want.

Yeah. Thanks, Alex it's it's a great question <unk>, we're really going into a new model for reimbursement with rock Kb in and and the payers are very interested in keeping up with gene therapies that are that are coming to the.

<unk> market rock Caveon being perhaps one of the the first meaningful gene therapy brands to hit a they want to make sure that they're not falling behind and it's really the shift from how do you create a reimbursement environment that for a onetime.

Durable treatment in a model that is accustomed to paying for chronic therapies or overtime.

And what what the payers have told US is that one they understand that theres limitations, so far in companies abilities to create pay for performance agreements that don't create problems with.

Government price reporting and I'm sure you understand the issue there Medicaid best prices and M. P's that wouldn't would trigger a cascade of lowering price to government or paid patients. That's a problem they understand that they've also.

Jeffrey Robert Ajer: And I expect that'll continue until the clinics are back up and able to continue their operations again. Recalling that with Palenzie, patient starts are directed to be under the supervision of a healthcare professional that can manage potential adverse events, at least for the first injection. I hope that answers your question, Chris. Yeah, and also for Pauline's speaking of both the patients and...

Advised us biomarin that outcomes based agreements are important to them. It it's a way of shifting the risk from their shoulders back to the manufacturer for both response and durability over the time, they've told us that it's important and they've told us that agreements.

That are limited to.

23% or less of of the purchase price.

JJ Bien-Aime: The clinicians have to be certified that the patient can receive the product and know how to self-inject and that the physician can know about how to manage the potential side effects. That's why there has been, in late March and in April, a slowdown in new patients. But we believe it's temporary, and when the PKU clinic reopens, we hopefully will see an acceleration.

Don't particularly excite them they don't consider that to be a solution. So we've been working on strategies for outcomes based agreements for Rockpile Indian.

We think that Weve potentially you know got some some things that will work for payers were out there talking to payers right right now and probably for competitive purposes I won't go into the details of what that all looks like but I think we we have a potential path forward.

Operator: We have our next question from the line of Phil Nadeau from Cowen & Company. Your line is now open. Good afternoon. Thanks for taking my questions and congrats on the progress. I also wanted to ask a question about the guidance. It looks like the Vimizin guidance was cut by about $30 million and Nagazine by $20 million. I'm just curious, one, what have you been seeing in April? How far apart can those infusions be pushed before patients see some impact on their symptoms? And two, what proportion of patients worldwide are able to get these infusions at home and, therefore, maybe won't have much of a disruption to their lives?

That would address outcomes based agreements desires from payers. Thank you.

Next question is from the line of like banks out from Citigroup. Your line is now open.

Great. Thanks for taking my questions and congrats.

Thank you very much for a job as that from my side a good luck with your next Oh.

Part of your adventures.

No I think.

Just just wanted to get little bit more color on the shape of because you you are thinking in your base case.

If I heard correctly Youre, assuming you should assume Lucy in fourth quarter your assumptions is that fair.

Jeffrey Robert Ajer: So Jeff...

You mean that [laughter].

Jeffrey Robert Ajer: infusions in markets where home infusion is not a well-established practice. So we normally know that in markets where home infusion is an established practice, compliance rates are higher, and we've seen less disruption in those markets where patients are home infused. Now, we've got a very diverse global business for both Naglozyme and Vimazim.

But it could be like to second quarter little bit dollar one third quarter, starting to decode and looks like it could be completely normal or how you're thinking about that anybody has been thank you.

Got you want to.

If you wasn't let's do it but why don't you get started.

Yeah. So thank you Mohit everybody is busy predicting the future here, which is difficult to do in normal circumstances.

Jeffrey Robert Ajer: I don't want to try to report high-level numbers of what percent of patients are home-infused or not, but our tactics have been in markets where we've got home infusion as an established practice, and we're trying to push more patients into that channel. And that channel is relatively open. In markets where home infusion is not an established practice, we see this as an opportunity in some places to try to get it established, and so we've had some success in places like Brazil and Argentina and Colombia and Spain, for example, in pushing patients into home infusion, where it's not an established practice, or alternative infusion sites, those being maybe a smaller hospital that's closer to home, less impacted by the COVID-19 situation, or, And we're seeing some positive effects of that already. On the other hand, every infusion can be critical.

And probably fraught with even more difficult.

Difficulties to do precisely given the current situation, but we started seeing disruptions to our business or the ended the first quarter, which did not impact our financial performance in the first quarter.

But as we began in the second quarter, we we knew that the disruptions that we're seeing would flow through to our business. As described we quickly quickly got and year to start taking mitigating actions mainly on missing fusions as.

As I've described and Ah, we think that those mitigating actions are having an impact. So yes. We're we're anticipating that that we'll see the biggest impact to our business in the second quarter and that's the conditions relative to our business would be improving as we go through.

Through the end of second quarter through the third quarter and our target is to have something that looks like pre cobot 19 levels of demand.

Jeffrey Robert Ajer: And we're fortunately seeing a much higher level of compliance and less disruption with that part of the business. I hope I've addressed your question there, Tim. Thank you.

But we're able to address and facilitate and in the fourth quarter you can tell from the level of our guidance reductions the magnitude of of the anticipated impact to each of our different brand. So that's a that's a way of kind of quantify quantifying our X.

Operator: And actually, if I may add, actually, the virus situation is probably, eventually, going to be helpful to actually move a lot of the treatments from the countries that are behind the U.S. and Western Europe, moving the treatment and infusions from hospitals into either homes or places that are closer to the patients, which is actually good news and might actually improve compliance in the long term.

By patients for each of the different brands relative to the dynamics that I'd been describing so hopefully it's possible for you to connect the dots a little bit between the qualitative and quantitative fees maybe had positive JJ has other I thought I mean, just when I highlight that you did.

Henry J. Fuchs: We have our next question from the line of Matthew Harrison from Morgan Stanley. Your line is now open.

It is indeed very difficult for us to forecast where things are going to go.

Hey, look like things are getting better.

In most parts of the world, but we're not you know we not out of.

Operator: Great. Good afternoon. Thanks for taking the question. Just too quickly from me, I guess, for Hank.

It's complex situation that the virus has created a so again, we assume here the main impacting Q2, some recovery in Q3, and ample hopefully Q4 potentially back to normal but.

Henry J. Fuchs: I didn't see a comment during the press week, and I'm sorry if I missed it. Maybe you commented during your remarks.

It will depend a lot on how quickly the economies are reopening in different parts of the world that also.

You know if some of you know their top that there could be a second wave of the virus that there was in for the 1918 Spanish flu.

Henry J. Fuchs: Any impact on enrollment in...

Henry J. Fuchs: Thank you for the full pivotal study.

Henry J. Fuchs: on PKU gene therapy. Is there anything you can do by adding some more sites or anything?

We are [laughter]. If there is one it will be minor and there will be more therapies available by them that all this is really hard to predict. So here, we don't assume it's significant Seneca second wave of the CRO to virus eating the world in Q4.

Henry J. Fuchs: [inaudible] Hi Matt. 270. I'm sorry, Roktavian Phase 3 enrollment, which I think you're asking about, we completed in November, and so we're not presently enrolling anyone further as regards our initial approval. So good news on Roktavian, the confirmatory trial fully enrolled. And as far as the sort of the generalized restart, we have had a lot of conversations with investigators, and there are reopenings happening in different ways and in different places at different times. And we're really trying to get a handle on where a site is going to be able to dose the first patients when the resumption of clinical trials. Don't exactly have clarity on when that's going to be or where that's going to be. Next question is from the line of Paul Matteis from Stifel. Your line is now open. This is Alex on behalf of Paul. Thanks for taking the questions. I just wanted to see how your thinking has evolved on paper performance for...

Oh, that's always the that doesn't happen. Then then then we'd probably be confident in our guidance here.

The other next question from the line of Tim Lugo from William Blair. Your line is that a little thing.

Thanks for taking my question.

Yeah, we're we're almost two years into the approval Palin Jake here in the U.S. and we obviously have to hear Covanta approaching can you give US you know your updated thoughts on how many of the Ku band patients will be transition on the policy by the kind of generic comes to market here in the U.S. given that we obviously have large.

Sure health care disruption given kind of it.

Yep.

Yeah. So great question, we've seen so far with a with the uptake of Palins IEC in adult patients, we've seen almost 40% of.

Pounds each patients.

Coming from.

Patients that are on commercial therapy, with Ku band and essentially that what that is indicating our PK you patients that were being treated with ku band that we're having a response, but not seeing the magnitude of a response or that they desired and seeking power.

Operator: Paper Performance for Gene Therapy as a Model in the U.S.

Jeffrey Robert Ajer: Jeff, do you want to address that one?

On Zika is a more powerful alternative.

Jeffrey Robert Ajer: Yeah, thanks, Alex. It's a great question. We're really going into a new model for reimbursement with Roktavian, and payers are very interested in keeping up with gene therapies that are coming to the market, Roktavian being perhaps one of the first meaningful gene therapy brands to hit. It's a way of shifting the risk back to the manufacturer for both response and durability over time. They've told us that it's important, and they've told us that agreements that are limited to 23% or less of the purchase price don't particularly excite them. They don't consider that to be a solution. We think that we've potentially, you know, got some things that will work for payers. We're out there talking to payers right now, but probably for competitive purposes, I won't go into the details of what that all looks like. But I think we have a potential path forward that would address outcomes-based agreements and desires from payers. Thank you.

So 40% is a pretty large proportion of our new palins IEC patients coming over from from Ku band, obviously in the United States, We're starting with a pretty big pool of adult patients on Ku band So.

While we're we're seeing a material a shift of that.

Patient base over to Palin Z.

For reasons that I described I wouldn't say the that represents anything near a majority of those patients.

As we go into a loss of exclusivity period later this year.

One of the things that we're encouraged by is.

Even even if we have a ku band patient that Oh that that goes onto a generic form of product. We really haven't lost them forever. We're connected to them through the clinics were connected to them from our hub services with their you know what their opt ins and.

The information our clinical coordinators are connected with them and and how Lindsay remains a viable option going forward for any patient any adult patient that has not seen kind of response, they're looking for on Ku band or a generic version of Ku band. So we're optimistic about.

Operator: The next question is from the line of Mohit Bansal from Citigroup. Your line is now open. Great.

Jeffrey Robert Ajer: Thanks for taking my questions. And congratulations. And thank you very much for Rob as well from my side.

Jeffrey Robert Ajer: Good luck with your next part of your adventures. Now, I just wanted to get a little bit more color on the shape of recovery you are thinking in your base case. If I heard correctly, you are assuming normalcy in the fourth quarter in your assumptions. Is that fair? Or are you assuming that it could be like this second quarter will be down, third quarter starting to recover, and fourth quarter could be completely normal? Or how you're thinking about that would be very helpful.

Our market potential and opportunity in the U.S. per pound the kind of in the medium and longer term.

Thank you.

Next question it sounds a line of Kennen Mackay from RBC capital markets. Your line is now open.

Hi, Thanks for taking my question I want to say congrats to the entire team for the Q1 results that really show the by Marina operationally and clinically and commercially navigating these really unprecedented times ahead of really any of our expectations.

Jeffrey Robert Ajer: Thank you.

Thank you also had another.

JJ Bien-Aime: Jeff, you want to... I'll say a few words on that too later, but why don't you get started?

Keeping question on guidance here.

For your 2020 got him for a negative 5% headwind for them 500 million dollar you're at midpoint.

Jeffrey Robert Ajer: Yeah, so thank you, Mohit. Everybody is busy predicting the future here, which is difficult to do in normal circumstances, and probably fraught with even more difficulties to do precisely, given the current situation. But we started seeing disruptions to our business at the end of the first quarter, which did not impact our financial performance in the first quarter. But as we began the second quarter, we knew that the disruptions that we're seeing would flow through to our business as described. We quickly got in gear to start taking mitigating actions, mainly on misinfusions, as I've described. And we think that those mitigating actions are having an impact. So yes, we're anticipating that we'll see the biggest impact on our business in the second quarter and that conditions relative to our business will be improving as we go through the end of the second quarter and into the third quarter.

And it sounds like expectations on the vast majority about is going to come in Q2 with the exception of maybe a little bit sort of driving onto the rest of year and pertaining to the sort of early removal of Uh huh.

Rock to live in Q4 in Germany, I'm, just trying to sort of manage my my team to expectations should I really be thinking about the vast majority of about $100 million coming.

In Q2, and then lastly, I just feel compelled to give a final congrats to Jeff and anyone else on the people who help come up with the Rocky and then I think that's an awesome brand name and it sounds like a metal song really really like that.

Thank you.

So I think anything I think jet kind of addressed that that's the question again that we do see indeed, the majority of the hit on their revenues. It's Q2, there will be eight in Q3 as compared to what we're expecting because.

For instance of the you know some probably take patients that were supposed to be started that.

Being delayed and then everything gets the day without some miss infusion that will still be probably happening.

Oh, you know in E. They could you maybe able to do you feel that it's hard to predict but actually maybe Brian can give you. Some perspective all this if you want to kick in Brian on this Oh.

Jeffrey Robert Ajer: And our target is to have something that looks like pre-COVID-19 levels of demand that we're able to address and facilitate in the fourth quarter. You can tell from the level of our guidance reductions the magnitude of the anticipated impact on each of our different brands. So that's a way of kind of quantifying our expectations for each of the different brands relative to the dynamics that I've been describing. So hopefully, it's possible for you to connect the dots a little bit between the qualitative and the quantitative. Maybe I'd pause there and see if JJ has other thoughts. No, I mean, just when I...

Yeah.

Yeah of course, it it accident and I think so what we've commented on.

Candidates that that disruption will be worse in Q2.

I don't think we can predict with certainty what how how that will actually translate into ordering patterns. We already point to a you know our annual revenue guidance as the primary indicator for the commercial brand and less so quarter to quarter, because we know that country by country specific ordering patterns you know.

Yes greatly due to timing so so depending on you know whatever level of supply was on hand in these certain territories, where we're experiencing some disruption in infusion, a and whether and when they will order next is well is how that's built into Q3 I don't think we can put our finger on on the exact ordering path.

JJ Bien-Aime: I'd like to highlight that it is indeed very difficult for us to forecast where things are going to go. It looks like things are getting better in most parts of the world, but we're not out of it. This is a very complex situation that the virus has created.

Return, so we would point get to the annual guidance.

[noise], having next question from the line I'm Chino Wow from Barclays. Your line is now.

Hi, This is Peter can from what you know and thanks for taking my question I guess I guess I'm on question on valve on [laughter] switches to a standard review you have made wants to see Oh, the phase three data.

JJ Bien-Aime: So again, we assume here the main impact being Q2, some recovery in Q3, and hopefully, Q4 potentially back to normal. But it will depend a lot on how quickly the economies are reopening in different parts of the world. And also... You know, as some of you know, there are talks that there could be a second wave of the virus, like there was for the 1918 Spanish flu. Hopefully, if there is one, it will be minor, and there will be more therapies available by then, but all this is very hard to predict. So here, we don't assume a significant second wave of the coronavirus seeding the world in Q4. So if that doesn't happen, then we're relatively confident in our guidance.

Make their decision.

Thank you very much.

Hi.

Hi, there.

Yeah, I'm not sure I heard the full question is it possible the repeat it.

Oh sure.

And was in a case where.

Yeah.

It should be and thereby pockets.

And the right review.

Uh huh.

Yeah.

But I mean data to make a decision.

Certainly and imaginable possibility I think it's a little premature to talk about where you know what.

Sort of turns might happen in a review as you know we well historically, we don't give very detailed comments about where we are in review.

Operator: We have our next question from the line of Tim Lugo from William Blair. Your line is now open. Thanks for taking my question. You know, we're almost two years into the approval of Pallan-Zik here in the U.S., and we obviously have generic Kuvan approaching. Can you give us, you know, your updated thoughts of how many of the Kuvan patients will be transitioned onto Pallan-Zik by the time the generic comes to market here in the U.S., given that we obviously have larger healthcare disruptions given COVID?

Because it's very difficult it can be very difficult to interpret and matterson get settled so I think the message up today is because of co bid a while we're on accelerated assessment to review its been delayed and because accelerated assessment is complicated to stay on comp.

Okay that also by the advanced the nature of the therapeutic there may be a third or delay and feel comfortable guiding you to an expectation of an opinion in Q4 Q1 Q4. This year Q1 of next year, but review is certainly not complete and things may change further.

Jeffrey Robert Ajer: Good job.

Jeffrey Robert Ajer: Yeah, so, um, great question. We have seen so far with the uptake of palindzic in adult patients; we've seen almost 40% of palindzic patients coming from patients that are on commercial therapy with QVAN. And essentially, what that is indicating are PKU patients that were being treated with QVAN that were having a response but not seeing the magnitude of a response that they desired and seeking Palenzik as a more powerful alternative. So 40% is a pretty large proportion of our new Palanzec patients coming over from Kuvan.

Thank you very much the good news I would I would say that I mean did that and then now let me flip that around so the good news is we're that to happen we're fully enrolled with a pivotal trial. So it's not like what we're not like the company that put the accelerated approval study into the rate regulators and then have not even so.

Started the confirmatory study, we finish enrollment confirmatory study.

Next question is from the line of Alley, Merlot from Cantor Fitzgerald out your line is now open.

Thanks, so much for sleeping man and congrats on all the progress I'm just a question I'm sorry.

Jeffrey Robert Ajer: Obviously, in the United States, we're starting with a pretty big pool of adult patients on Kuvan. So while we're seeing a material shift of that adult patient base over to Palin Z, for reasons that I've described, I wouldn't say that that represents anything near a majority of those patients.

From your recent discussions with the regulators around the filing.

What was their latest perspective on how much efficacy data you need to have a leap all that covers either under five or whether it's one of the safety data from the ongoing studies, coupled with that sees me and he just over five at the end now for a broadly of off thanks.

Jeffrey Robert Ajer: As we go into a loss of exclusivity period later this year, one of the things that we're encouraged by is even if we have a Kuvan patient that goes on to a generic form of the product, we really haven't lost them forever. We're connected to them through the clinics. We're connected to them from our hub services with their opt-ins and information. Our clinical coordinators are connected to them. And Palanzec remains a viable option going forward for any patient, any adult patient that is not seeing the kind of response they're looking for on Kuvan or a generic version of Kuvan. So I think we're optimistic about our market potential and opportunity in the U.S. for Palanzec in the medium and longer term. Thank you.

Yeah. These kinds of high all even thanks for the question. These conversations are at a much earlier stage than where those kinds of questions get answered, but negotiation is gonna be went which is what's in the package.

And what will be in the package will be the safety data on patients who are older than six months of age.

Ah Ah Ah went up to five years age in addition to the pivotal study.

There are very well aware that and I'm also very well aware that their budgets have been advised them that they should anticipate that huge will commence in very young children at time of the initial licensure, but beyond that we haven't had good conversations about labeling.

Operator: The next question is from the line of Ken and Mackay from RBC Capital Markets. Your line is now open.

JJ Bien-Aime: Hey, thanks for taking the question. And I want to say congrats to the entire team for these two-on results that really show that Biomarin is operationally, clinically, and commercially navigating these really unprecedented times ahead of all of our expectations. So I also had another housekeeping question on guidance.

And maybe by the way you know the first two cohorts of the under five studies are fully enrolled Oh, yeah woman or the third cohort wishes fishes from Asia, you know from burst through six months of age couple of patients have been enrollees been slow down little bit of course by the ER the virus, but the first of course, our food animal.

JJ Bien-Aime: The full year 2020 guidance for a negative 5% headwind sort of implies a $100 million hit here at midpoint. And again, it sounds like expectations are the vast majority of that is going to come in Q2 with the exception of maybe a little bit sort of dragging on through the rest of the year and pertaining to the sort of early removal of Roctavian in Q4 in Germany. I'm just trying to sort of manage my Q2 expectations. Should I really be thinking about the vast majority of that $100 million hit coming in Q2?

Next question is something line of Vinson, Ken I'm from Bernstein. Your line is I'm open.

Thank you very much for squeezing in and congrats on the progress.

<unk> earlier question on insurance coverage.

This is where to shift from commercial and government insurance roughly what net price impact would you like to see if your portfolio and which brought might be more affected if any are the reasons to think that youre your portfolio might actually be somewhat more insulated Andy I guess, the bio pharma sector as a whole.

Jeff you want to try that one.

Yeah. That's a great question Benson Yeah, we we have a we have a government prices, we have 340 be discounts depending on where.

JJ Bien-Aime: And then lastly, I just feel compelled to give a final congratulations to Jeff and anyone else on the team who helped come up with the Roctavian name. I think that's an awesome brand name, and it sounds like a metal song. I really, really like it.

Patients are getting their care and we've got commercial patients and for our portfolio in the United States.

We don't we don't contract for or or discount our commercial portfolio.

JJ Bien-Aime: Thanks. So I think Jeff kind of addressed that question again, that we do indeed think the majority of the hit on revenues is Q2. There will be a hit in Q3 as compared to what we're expecting because, for instance, some palliative patients that were supposed to be started and are being delayed, and then everything gets delayed with that. Some misinfusions that will still be happening, you know, in late Q2 and maybe early Q3, although it's hard to predict. But maybe Brian can give his own perspective on all this. If you want to chip in, Brian, on this, any comments to make?

So if we had a substantial shift in patients moving from.

From commercial plans to like Medicaid or for example.

You could see an individual patients are generating less net revenue.

Based on the the number of patients that we've got in the United States, we'd have to see a pretty big shift.

And.

Patients over to you know the government plans before we'd see a material impact on our on our kind of gross to net for revenues in the United States and then thinking about you know the United States as a part of the diversified global business at the.

Brian R. Mueller: Yeah, of course, JJ. Thanks.

Brian R. Mueller: And I think, what we've commented on Canada is that the disruption will be worse than Q2. I don't think we can predict with certainty how that will actually translate into ordering patterns. We already point to our annual revenue guidance as the primary indicator for the commercial brands and less so quarter to quarter because we know that country by country specific ordering patterns shift greatly due to timing. So depending on whatever level of supply is on hand in these certain territories where we're experiencing some disruption and infusion and whether and when they will order next as well as how that spills into Q3, I don't think we can put our finger on the exact ordering pattern, so we would point you to the annual guidance.

Probably the bigger part of our business in the U.S. will undoubtedly the bigger part is our PK you franchise relative do our.

Our enzyme patience.

So I'm I'm, sorry for musing, while I'm answering I think it's a great question, we haven't seen anything but that.

Even comes close to a signal of you know more than anecdotal reports of a patient here or or or there, losing their private insurance and moving over to a safety net insurance. So yeah I think it's a theoretical.

Thing, but we haven't seen anything close to that yet happening.

Operator: We have our next question from the line of Gina Wang from Barclays. Your line is now open. Hi, this is Peter Iken from Virginia Bank. Thanks for taking our question. I guess I guess on the question about the bell box. If it switches to a standard review, the EMA wants to see the full phase three data to make their decision. Thank you very much.

<unk>.

There are no further questions on time, I turn the call back over to our Tam T.O.J.J.P. enemy for closing statement.

Thank you operator and.

It's just wanted to conclusion say that you know in who's a unprecedented times, then [laughter] innovative treatments that I've been developed by bomb rain and others I've never been more relevance to the people who need them.

Operator: [inaudible] Hank? You there?

Henry J. Fuchs: Yeah, I'm not sure I heard the full question. Is it possible to repeat it? The question was, in a case where EMA review switches to a standardized process, would EMA like to see the full phase 3 data to make their decision? Uh, certainly an imaginable possibility.

Oh it is the healthcare industry that will change the course of the all Green coffee 19 pandemic.

A one of the nice side effect of the viruses that the pricing of drugs is not making the headlines anymore.

For obvious reasons.

A which is a good news for us and somebody in the face of the there you will near term crisis I remain very confident that in bombings ability to deliver on the tremendous opportunity for value creation that we had before us so our underlying fundamentals remain strong and we're well positioned to success, we manage through these uncertain.

Henry J. Fuchs: I think it's a little premature to talk about where, you know, what sort of turns might happen in a review. As you know, we historically don't give very detailed comments about where we are in review. Because it's very difficult, it can be very difficult to interpret, and matters can get settled.

Economic environment.

The critical growth drivers are in place I didn't get the successful based business transition our pipeline to address larger rare indication sent laid the foundation for significant profitability with rock TVN and it was always thought approval.

Henry J. Fuchs: So I think the message of today is because of COVID, while we're on accelerated assessment, the review has been delayed. And because accelerated assessment is complicated to stay on, complicated also by the advanced nature of the therapeutic, there may be a further delay. You feel comfortable guiding you to an expectation of an opinion in Q4, Q1, Q4 this year, Q1 of next year. But, you know, the review is certainly not complete, and things may change further.

The dispositions biomarine for substantial success in both the near term and the long term. So thank you again for your continued support and stay safe Goodbye.

Thank you presented its ladies and gentlemen. This concludes today's conference call. Thank you also participating you may now disconnect presenters. Please save for the post conference.

[music].

Henry J. Fuchs: Thank you very much. The good news, I would say, I mean, and then now, let me flip that around and say the good news is, were that to happen, we're fully enrolled in a pivotal trial, so it's not like we're the company that put the accelerated approval study into the regulators and then have not even started the confirmatory study. We finished enrollment in the confirmatory study. The next question is from the line of Ellie Merle from Cantor Fitzgerald. Your line is now open.

Henry J. Fuchs: Thanks so much for squeezing me in, and congratulations on all the progress. Just a question on SORTIDE, from your recent discussions with the regulators around the filing, I guess, what was their latest perspective on how much efficacy data you'd need to have a label that covers ages under 5, or whether sort of the safety data from the ongoing studies, coupled with phase 3 in ages over 5, would be enough for a broad label? Thanks. These conversations are at a much earlier stage than where those kinds of questions get answered, but negotiation is going to be what's in the package, and what will be in the package will be safety data on patients who are older than six months of age and up to five years of age, in addition to the pivotal study. They're very well aware of that, and they're also very well aware that their advisory committee advised them that they should anticipate that use will commence in very young children at the time of initial licensure, but beyond that, we haven't had concrete conversations about labeling.

Henry J. Fuchs: And let me, by the way, you know, the first two cohorts of the under-5 studies are fully enrolled. The enrollment of the third cohort, which is patients from the age of, you know, from birth to six months of age, CalPERS patients have been enrolled. It's been slowed down a little bit, of course, by the virus, but the first two cohorts are fully enrolled.

Operator: The next question is from the line of Vincent Chen from Bernstein. Your line is now open. Thank you very much for squeezing in, and congratulations on all the progress. To follow up on Josh's earlier question on insurance coverage shifts, if patients were to shift from commercial to government insurance, roughly what net price impact would you expect to see on your portfolio, and which products might be more affected, if any? Are there reasons to think that your portfolio might actually be somewhat more insulated than the, I guess, biopharma sector as a whole?

Jeffrey Robert Ajer: Jeff, do you want to try that one?

Jeffrey Robert Ajer: Yeah, that's a great question, Vincent. You know, we have government prices. We have 340B discounts, depending on where patients are getting their care. And we've got commercial patients. And for our portfolio in the United States... We don't contract for or discount our commercial portfolio.

Jeffrey Robert Ajer: So if we had a substantial shift in patients moving from commercial plans to like Medicaid, for example, you could see an individual patient generating less net revenue. Based on the number of patients that we've got in the United States, we'd have to see a pretty big shift. Patients over to government plans before we'd see a material impact on our, on our kind of gross to net revenues in the United States. And then thinking about the United States as a part of the diversified global business, probably the bigger part of our business in the US, while undoubtedly the bigger part is our PKU franchise relative to our, our enzyme patients. So, I'm sorry for musing while I'm answering. I think it's a great question. We haven't seen anything that... even comes close to a signal of more than anecdotal reports of a patient here or there losing their private insurance and moving over to a safety net insurance. I think it's a theoretical thing, but we haven't seen anything close to that happening yet.

[music].

Operator: There are no further questions at this time. I turn the call back over to our Chairman and CEO, J.J. Venameh, for closing statements.

JJ Bien-Aime: Thank you, Operator. I just want to conclude and say that in these unprecedented times, the necessity of innovative treatments that have been developed by Biomarin and others has never been more relevant to the people who need them. It is the healthcare industry that will change the course of the ongoing COVID-19 pandemic. One of the nice side effects of the virus is that the pricing of drugs is not making the headlines anymore for obvious reasons, which is good news for us, but in the face of this very real near-term crisis, I remain very confident in Biomarin's ability to deliver on the tremendous opportunity for value creation that we have before us. So our underlying fundamentals remain strong The critical growth drivers are in place, having built a successful base business, transitioned our pipeline to address larger, rarer indications, and laid the foundation for significant profitability with Roktevian and Rosaritat approvals. All of that positions Biomarin for substantial success in both the near-term and the long-term. So, thank you again for your continued support, and stay safe. Goodbye.

Operator: Thank you, presenters. Ladies and gentlemen, this concludes today's conference call. Thank you all for participating. You may now disconnect. Presenters, please stay for the post-conference.

unknown: ??? ??? © BF-WATCH TV 2021, ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ???