Q2 2020 Biomarin Pharmaceutical Inc Earnings Call

During the conference call today somebody marine needs Tracy Mccarthy.

Traci McCarty: Operating the conference call today from Biomarin is Traci McCarty, Vice President of Investor Relations. Please go ahead, Traci. Thank you, Laurie, and thank you everyone for joining us today.

<unk> President of Investor Relations. Please go ahead trace.

Thank you all right and thank you everyone for joining us today to remind you. This non confidential presentation contain forward looking statements about the business prospects, a vibrant pharmaceutical inc., including expectations regarding vibrant financial performance commercial products central future products in different areas of therapeutic research and development.

Unknown Executive: To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical Inc., 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, the actions of regulatory authorities, the 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 10-Q, 10-K, and 8-K reports. On the call remotely from Biomarin Management today are JJ Bien-Aime, Chairman and Chief Executive Officer, Jeff Ager, Executive Vice President, Chief Commercial Officer, Robert Baffi, President of Global Manufacturing and Technical Operations, Hank Fuchs, President, Worldwide Research and Development, and Brian Mueller, Executive Vice President and Chief Financial Officer. We hope to keep this call to one hour, so we respectfully request that you limit yourself to one question 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-Aime. Thank you, Traci.

May differ materially depending on the progress of fiber is product program actions of regulatory authorities availability of capital future actions in the pharmaceutical market and development by competitors and those factors detailed and Biomarin filings with Securities and Exchange Commission such a sense you indicate an 8-K reports on.

Paul remotely from Biomarin management's day, or JV enemy, Chairman and Chief Executive Officer, Jeff Ager Executive Vice President Chief Commercial Officer, Robert Baffi President of global manufacturing in technical operations. Thank you President worldwide Research and development and Brian Mueller Executive Vice President and Chief Financial Officer.

We hope to keep this called one hour. So we respectfully request you limit yourself to one question during the Q named portion of the call. Thank you for your understanding I'll now turn the call over to our chairman and CEO JJ piano me [noise].

JJ Bien-Aime: Good afternoon, and thank you all for joining us on today's call. We hope that you and your families have been well during these uncertain times. For Biomarin, we continue to manage the business in new ways to ensure access to our essential therapies around the world. I want to commend our employees for their extraordinary commitment to our mission of providing innovative treatments to people with rare diseases, which has been especially challenging in the COVID-19 world. Our second quarter results of $430 million in total revenues, or 11% growth over last year, is a testament to the importance of our therapies, our diversified product base, and far-reaching commercial food brands. Consistent with our commentary in Q1, we experienced impact from COVID-19 in the second quarter but hope to return to normal demand patterns by the end of 2020, bearing any significant deterioration of the pandemic situation before you ask. We continue to expect to achieve full year revenue and Operating Expenses guidance as communicated on our first quarter call.

Thank you Tracy and good afternoon, and thank you all for joining us on today's call.

We hope that you on your family's that'd be well during these uncertain times.

Right now we continue to manage the business in new ways.

Sure access to our international therapies around the world.

I want to commend our employees for their extraordinary commitment to our mission of providing you know the treatment to people with rare diseases.

Each had been especially challenging.

19 World.

Our second quarter results of $430 million in total revenues or 11% rules work. That's here he did testaments to the importance of our therapies.

Our diversified product base and far reaching commercial food.

Consistent with our commentary in Q1, we experienced impact from Koby 19 in the second floor I hope to return to normal demand patterns by the out 2020.

Sorry, any significant deterioration depend maybe situation before yearend.

We continue to expect to achieve full year revenue.

Operating expenses Dinos has communicated on our first quarter call.

Moving now to the most then you kind of outcomes of the year the advancement of our two product candidate under regulatory review Rock Canyon gene therapy for the treatment was to be hemophilia, a and it was only tied for the treatment of 800 Felicia.

JJ Bien-Aime: Moving now to the most anticipated milestones of the year, the advancement of our two product candidates under regulatory review, Roktevian gene therapy for the treatment of severe hemophilia A and Rosorityte for the treatment of achondroplasia. In June, we were pleased to share the four-year Octavian data updates at the World Federation of Hemophilia's virtual summit. Following a one-time infusion of Octavian, all study participants who received the 2B-marketed dose remained off exogenous factor VIII prophylactic therapy and demonstrated a greater than 90% reduction in bleeding episodes four years after treatment, which is our PDUFA target action date later in August.

Excuse me, we're pleased to share that four year old Stephen data updates at the world situation, where people will be a spiritual summit.

Following a one time infusion from TVN All study participants who received the to be market adores remain.

JJ Bien-Aime: We are going to continue our policy not to comment on the status of the review of our BLA. I want to thank you for bearing with us as we look forward to potential FDA approval later this month. Stay tuned.

Agent.

Moving to a very tied.

JJ Bien-Aime: Two weeks ago, we submitted the marketing authorization application to the European Medicines Agency for the treatment of children with achondroplasia. I want to thank everyone across the organizations who worked on this application under unusual and difficult circumstances due to challenges faced by COVID-19. It seems there is nothing the teams can't accomplish.

Two weeks after two weeks ago, we submitted the marketing authorization application to the European Medicines agency for the treatment our children with a condo pleasure.

I want to thank everyone of course, the organizations, who worked on its application under unusual on difficult circumstances due to challenges faced by covered 19.

It seems there is no say nothing the teams can accomplish.

JJ Bien-Aime: Once the application is validated, we expect the NAA review to begin later this month, and we are on track to submit the results at NDA. The FDA leader in this court. If approved, Rosoratibe would be the first and only medicine designated for the treatment of achondroplasia, the most common form of dwarfism in the U.S. and in East India. These key events, combined with our goal of turning profitable on a cap basis this year, for the first time in the company's history, have had us very busy.

Once the application is validated we expect the any reviewed to begin later this fall.

We are on track to submit the resort that end the to the FDA later in this quarter.

If approved for the retired.

I would be the first and only medicine designated for the treatment of 800 pager. The most common for more dwarfism in the us ending.

In the EU.

This key events combined with our goal of turning profitable on a GAAP basis each year for the first time in the company's history.

It has very busy.

JJ Bien-Aime: It is no coincidence that Biomarin is at a significant inflection point in 2020. We have been growing the company for many years, honing our R&D platform, advancing our manufacturing capabilities, expanding our commercial footprint, and working with health authorities around the world to develop essential medicines for people with rare diseases. We have positioned ourselves for substantial success. Thank you for your continued support. And now, I would like to turn the call over to Jeff to discuss the commercial business update. Thank you, JJ.

It is no coincidence that bottom line is that a significant inflection point in 2020, we have in growing the company for many years, putting our R&D platform advancing our manufacturing capabilities expanding our commercial footprint.

And working with health authorities around the world to develop essential medicines for people with rare diseases.

We are positioned ourselves for substantial success in both the near term and the long term and we want to thank you for your continued support.

Now with our turn the call over to Jeff will discuss the commercial business of the.

Yes.

Thank you Jay Jay as we discussed results from the second quarter 220, pointing I'd like to begin by saying I'm very proud of the team's performance across all regions. During this quarter, which has challenged the entire world with think react and operate differently than ever before.

Jeffrey Robert Ajer: As we discuss results for the second quarter of 2020, I'd like to begin by saying I'm very proud of the team's performance across all regions during this quarter, which has challenged the entire world to think, react, and operate differently than ever before. When we provided our Q1 quarter results to you, the COVID-19 situation was still developing, yet we were already focused on assessing and forecasting near and longer-term impacts from what remains today a dynamic and unpredictable situation. These Q2 results are in line with the revised guidance that we provided previously and reflect the strength and resiliency of our business. They are also a testament to the ability of our teams globally to quickly employ mitigation efforts to reduce the impact of COVID-19 as much as possible.

When we provided our Q1 quarter results you. The cobot 19 situation was still developing yet we were already focused on assessing and forecasting near and longer term impact from what remains today, a dynamic and unpredictable situation.

These Q2 results are inline with the revised guidance that we provided previously and reflect the strength in the resiliency of our business.

They are also a testament to the ability of our teams globally. The quickly employ mitigation efforts to reduce the impact of killed with 19 as much as possible.

Further these results underscore the recognition worldwide that we are providing essential therapies to patients with high unmet medical needs and the prioritizing access to these products is vital to their long term outcomes.

As Jay mentioned, we achieved quarterly total revenues of $430 million and net product revenues marketed by Biomarin of $387 million in the quarter.

Year to date total revenues were $932 million, representing 18% increase over the same period in 2019.

Jeffrey Robert Ajer: Furthermore, these results underscore the recognition worldwide that we are providing essential therapies to patients with high-end medical needs and that prioritizing access to these products is vital to their long-term outcomes. As JJ mentioned, we achieved quarterly total revenues of $430 million and net product revenues marketed by Biomarin of $387 million in the quarter. Year-to-date total revenues were $932 million, representing an 18% increase over the same period in 2019.

On the specifics in the quarter from a brand perspective, and starting first with Pelon Zig.

We are reporting Q2 revenues of $41 million, a 116% increase compared to Q2 last year and year to date total revenues of $75 million, a 142% increase from the first half of 2019.

As expected the majority of that revenue came from the us for patients that had already started palin the therapy prior to covert 19.

They have been stable on their treatment without any significant increase in discontinuations.

Jeffrey Robert Ajer: On to specifics in the quarter from a brand perspective, and starting first with Palanzec. We are reporting Q2 revenues of $41 million, a 116% increase compared to Q2 last year, and year-to-date total revenues of $75 million, a 142% increase from the first half of 2019. As expected, the majority of that revenue came from the U.S. For patients that had already started Palanzec therapy prior to COVID-19, they have been stable on their treatment without any significant increase in discontinuation. The material impact on Palantir has been new patient initiation. The impact was most pronounced in the months of March and April, with an encouraging uptick in May and early June, followed by a regression that correlated with increasing COVID-19 infections. We're still optimistic about our ability to drive additional business with Palantik, subject, in part, to the ability of key clinics to operate in the COVID-19 environment.

The material impact on Palin seekers blended new patient starts the impact was most pronounced in the month of March and April within encouraging uptick in May in early June followed by a regression correlated with increasing cobot 19 infections.

We're still optimistic about our ability to drive additional business with talenti subject in part to the ability of key clinics to operate in the cobot 19 environment.

In Europe in the second quarter. The same dynamic played out in Germany, where patient starts were slow propelling the as expected importantly, despite covered 19, we completed price negotiations in Germany, which was a significant milestone and an important predicate for ongoing negotiations and other high.

Priority European markets.

Two van contributed $123 million on revenue in the second quarter for year to date revenues of $245 million, which represents 11% growth year over year for the first half of the year again with most of that growth coming from the United States.

Moving now to our MPS products as expected Cobot 19 had an impact on both new patient starts as well as continuity of infusions for patients already in therapy.

In Europe, Cobot, 19 impact occurred earlier and impact of the Latin American region occurred later, so the overall impact to the business has been variable by geography and by product.

Jeffrey Robert Ajer: In Europe, in the second quarter, the same dynamic played out in Germany, where patient starts were slow for Palanzig as expected. Importantly, despite COVID-19, we completed price negotiations in Germany, which was a significant milestone and an important predicate for ongoing negotiations and other high priority European markets. Kuvan contributed $123 million in revenue in the second quarter, for year-to-date revenues of $245 million, which represents 11% growth year-over-year for the first half of the year, again, with most of that growth coming from the United States. Moving now to our MPS products, as expected, COVID-19 had an impact on both new patient starts as well as continuity of infusions for patients already in therapy. In Europe, COVID-19's impact occurred earlier, and the impact of the Latin American region occurred later.

As a result, our response has been problem solving specific to the relevant local situations. The overall impact in the current business has been modest and is captured in the current guidance, we still have been able to identify and start new patients on therapy. However, the pace of that activity is slower.

Than prior to the pandemic.

Consistent with that dynamic the Muslim revenues contributed $117 million in the second quarter, a 5% decrease year over year, which is attributed to a combination of covered 19 impact and also order timing that was previously anticipated.

Year to date revenues of 250, more $4 million, which represents modest growth compared to the same period last year. Despite the challenges brought about by the global pandemic demonstrates the essential need for them to patients.

Turning to Naglazyme revenues totaled $81 million in the quarter and 18% decrease year over year and reflective of the same challenges observed with the Muslim.

Jeffrey Robert Ajer: So the overall impact of the business has been variable by geography and by product. As a result, our response has been problem solving specific to the relevant local situations. The overall impact of the current business has been modest and is captured in the current guidance. However, we have still been able to identify and start new patients on therapy. However, the pace of that activity is slower than prior to the pandemic. Consistent with that dynamic, BIMISIM revenues contributed $117 million in the second quarter, a 5% decrease year-over-year, which is attributed to a combination of COVID-19 impact and also order timing that was previously anticipated.

Consistent with vandalism growth of Naglazyme year to date of 6% or $195 million represent solid underlying demand for the 16 year old brand.

And finally, brineura contributed $50 million in that product.

Revenues year to date 26 million of that total added in the second quarter. The brand is maintaining a strong growth trajectory. We've seen in the last year. This contribution and growth are driven by strong adherence to therapy throughout the pandemic and strong 25% growth and patience on commercial.

RP year to date.

Lastly, a few words on launch readiness for rock Cavium.

Following potential approval Rostamian would be the first and only gene therapy available for people with severe hemophilia, a our U.S. stamens prepared and ready having pivoted quickly to optimize virtual and digital interactions with health care teams patient advocacy organizations.

Jeffrey Robert Ajer: Year-to-date revenues of $254 million, which represents modest growth compared to the same period last year, despite the challenges brought about by the global pandemic, demonstrate the essential need for VIMSIM to pay. Turning to Naglazyme, revenues totaled $81 million in the quarter, an 18% decrease year-over-year and reflective of the same challenges observed with Vimazim. Consistent with Vimazim, growth of Naglazyme year-to-date of 6%, or $195 million, represents solid underlying demand for this 16-year-old brand. And finally, Brent Nura contributed $50 million in net product. Revenues year-to-date; $26 million of that total were added in the second quarter.

And payers over the last few months. This is allowed our teams to continue empowering stakeholders with foundational gene therapy education through virtual programming, which has been in high demand.

We hope to be speaking with you soon about launch in pricing details following the potential approval of rock Cavium.

With that I'll. Thank you for your attention and now I'll turn the call over to Hank to provide an R&D update Hank.

Thanks, Jeff.

As Jay mentioned, commenting on the status of the review of our rights Cavium La given how close we are.

Target action date of August 21st Thank you for bearing with.

In Europe, our marketing authorization application filing remains under accelerated assessment at this time, however, and as we previously.

You procedure has been extended by at least three months due to cobot 19 related delays.

However.

And further.

The case with most filings and initially seen accelerated assessment.

Jeffrey Robert Ajer: The brand is maintaining the strong growth trajectory we've seen in the last year. This contribution and growth are driven by strong adherence to therapy throughout the pandemic and strong 25% growth in patients on commercial therapy year-to-date. Lastly, a few words on launch readiness for Roctavian. Following potential approval, Roctavian would be the first and only gene therapy available for people with severe hemophilia A.

We believe there is a high possibility that our M&A will revert to a standard review procedure on accelerated assessment.

Based on assumptions, we expect to see agent.

Later this year early index.

I would be pillar the Rostamian program coming soon is our 134 subject phase three studies completion.

Subjects in this study will be completed 52 weeks of observation by the end of November with data readout dissipated soon thereafter.

Given the strength of the rock TVN annualized bleed rate data observed to date, we're very optimistic that our phase three results will demonstrate superiority over standard of care factor replacement therapies controlling bleeding episodes are primary endpoint.

Henry J. Fuchs: Our U.S. team is prepared and ready, having pivoted quickly to optimize virtual and digital interactions with healthcare teams, patient advocacy organizations, and payers over the last few months. This has allowed our teams to continue empowering stakeholders with foundational gene therapy education through virtual programming, which has been in high demand. We hope to be speaking with you soon about launch and pricing details following the potential approval of Roctavian. With that, I'll thank you for your attention and now turn the call over to Hank to provide an R&D update. Hank?

As most of you know the for full year Octavian results were shared in June.

Mr. John package on the well Federation of various virtual summit.

Demonstrate continued hemostatic efficacy at all factor levels from the high.

On the Investor call following.

Presentation or commissions conveyed increasing confidence in the robust treatment effects observed in many of their claims.

Both professor.

Stories from the individual clinical trial subjects, who have been living.

Differently.

Moving their gene therapy.

Missions of witness firsthand. The contrast in the burden of hemophilia between patients coal trade with senior care prophylaxis and oppose gene transfer.

Thanks.

It's the interest and waiting for decades.

Yes.

Desired driving force for years, we're all eager to offer this is an option for patients.

Henry J. Fuchs: Thanks, Jeff. As JJ mentioned, we'll not be commenting on the status of the review of our Roktavian VLA, given how close we are to the Hadoopa target action date of August. Thank you for bearing with me. In Europe, our marketing authorization application filing remains under accelerated assessment at this time. However, and as we previously communicated, the review procedure has been extended by at least three months.

The four years of data demonstrating dramatically control following treatment gene therapy versus standard of care, but it's been about the dispute the highly innovative and transformational profile.

Importantly, as we think about Billy Joel commercial avian doses 60, 13 vector genome Hello.

Greenbrier results from our four year for 13 secular per Dino packaging of curricula does provides additional comfort about longer term outcomes for bleed control even lower factory.

As part of the virtual value.

Three years post infusion with the 40 does subjects demonstrated in fact activity level of 9.99 per deciliter as measured by that from a genetic assay.

Henry J. Fuchs: [inaudible] We believe there is a high possibility that our MAA will revert to a standard review procedure from the accelerated assessment. Based on these assumptions, we expect to see HMP opinions late this year or early next. Another key pillar of the Rock Damien program coming soon is our 134 subject phase three studies completion. Subjects in this study will have completed 52 weeks of observation by the end of November, with data readout anticipated soon thereafter. Given the strength of the Lactavian annualized bleed rate data observed to date, we are very optimistic that our phase 3 results will demonstrate superiority over standard of care factor replacement therapies in controlling bleeding episodes, our primary endpoint. As most of you know, the full four-year Roctavian results were shared by Professor John Caffey Demonstrating continued hemostatic efficacy at all factor levels from lowest to highest.

And the result was cumulative annualized bleed rate of less than one representing a 93% reduction bleeds from baseline at your free.

Factory this engine accordingly, our service by 96% over three years.

Takeaway for US is slowly decline observed in factory activity levels.

With our potential commercial versus 60 13 May result in control for rock in patients well.

Peers that have been demonstrated.

It's really inspirational and a big reason why we're so passionate about the work we are going for patients.

Turning now to restore tied for the treatment.

As JJ mentioned, we submitted the marketing authorization application European already.

Two weeks ago implanted summit in San Diego later this quarter.

The submissions include extensive data from our four pronged development program, which includes approximately five years of phase two results in 518 year old children.

Comprehensive natural history data.

Highly statistically significant placebo controlled phase three results.

Finally safety data from the ongoing phase two study of newborns through five years.

We continue to look forward to publishing and presenting the full data for the phase three studies that later this year, so stay tuned for that update coming soon.

Moving now to be mm 307, our investigational gene therapy for PK, you were continuing to bear insights and depending on the ongoing impact of serving 19. We believe we can begin dosing days in the phase one two study nicknamed Fearless later in the core third quarter. We're excited about the prospect of VMM three or seven as it represents.

Henry J. Fuchs: On the investor call following the WFH presentation, our commissions conveyed increasing confidence in the robust treatment effect observed in many of their patients. Both professors recounted stories from their individual clinical trial subjects who have been living differently since receiving their gene therapy. The clinicians have witnessed firsthand the contrast in the burden of hemophilia between patients fully treated with standard care prophylaxis and a post-gene transfer hemophilia patient.

Third, peaking new treatment option in our peaking franchise.

Second gene therapy development program, leveraging our learnings capabilities from that statement.

As we prepare for the potential approvals and launches of Octavian in such wallboard sort tied.

Earlier stage pipeline also continues to progress in July we began.

Mm 331 gene therapy credit stereo Egina, we expect benefit from our experience with Octavian environments, we have seven to drive it even more efficient development program lithium in Threeq religion.

Henry J. Fuchs: This community has been waiting for decades for gene therapy. This desire is going to drive you crazy, are all eager to offer their, Four years of data demonstrating dramatic weight control following treatment with gene therapy versus standard of care, it is difficult to dispute the highly innovative and transformational profile of... And most importantly, as we think about the durability potential, commercial Roctavian doses 6E13 VGPK, these 3-year results from our 4-year 4E13 VGPK dose provide additional comfort about longer-term, Leave control at even lower factory levels. Pardon the virtual, but if they'd shut up.

In addition, we're pleased to announce in the second quarter, our preclinical collaboration and license agreement with standpoint, a gene therapy platform.

To develop gene therapies to treat rare genetic part in my office.

The large operative biomarin, given the tremendous unmet need in both indications, where we can benefit from our growing expectation gene therapy development and manufacturing.

Before turning the call over to Brian for the financial update I would also like to their calls.

Colleagues across the organization for your continued commitment to contributions during these challenging but nonetheless.

Extraordinary effort undertaken to submit marketing applications going into something the team accomplished without missing.

I've been impressed by the flexibility of focus.

Okay.

Our programs, we've employed under very challenging.

Now I'll turn the call, we've really been very Yu, Chief Financial Officer, Biosimilar taken away by.

Thank you Hank.

Henry J. Fuchs: 3 years post infusion with the 4E dose, subjects demonstrated an impact activity level of 9.99 IU per deciliters measured by the chromogenic assay, and the result was a cumulative annualized bleed rate of less than one, representing a 93% reduction in bleeds from baseline at year three. Factory usage in the 43rd power was reduced by 96% over. The takeaway for us is that the slowing decline observed in factory activity levels... with our potential commercial dust of 6E13 may result in bleak control, poor rock preservation, Here's the. It's truly inspirational and a big reason why we are so passionate about the work we are doing for patients with penicillia. Tune in now to the Sewer Typhoon.

Please refer to today's press release summarizing our financial results for full details on the second quarter of 2020 as usual our comprehensive report on the quarter will be available on our upcoming form 10-Q, which we're planning to file later today.

Starting with revenues, we've observed that the impact of cobot 19 on our commercial business is overall consistent with our expectations back in April.

As Jeff mentioned, we experienced a modest top line impact from Cobiz 19 pandemic in the second quarter and a continued into the start of the third quarter.

With that much of our business is beginning to stabilize and is on trajectory toward normalized demand path.

We consider this level of Cobiz 19 impact on our business when we revised guidance in April and we remain committed to full year 2020 revenues of between $1.85 billion to 1.95 billion.

Moving on to operating expenses R&D expense for the second quarter was 182 million slightly lower compared to R&D expense for the second quarter, 2019, which was mostly due to reduced R&D activity for Octavian given its late stage of development.

Henry J. Fuchs: As JJ mentioned, we submitted the marketing author... 2 weeks ago and plan to submit to the NDA later this quarter. The submissions include extensive data from our four-pronged development program, which includes approximately five years of phase two results in five to 18-year-old children. Comprehensive Natural History, The Highly Statistically Significant Placebo Control Phase 3 Results And finally, Safety Data from the Ongoing Phase 2 Study of Newborns through 5 Years of Aging. We continue to look forward to publishing and presenting the full data from the Phase 3 study set later this year, so stay tuned for that update coming up. Moving on to BMF 307 and our investigational gene therapy for PKU.

As gene expense for the second quarter, 2020 was 175 million, which is slightly higher than us gene expense for the second quarter of 2019 as expected the year over year increase is due to commercial preparations for the launch of rock Cavium and the continued global launch of pounds.

Turning to bottom line results, we reported a GAAP net loss of 29 billion in the second quarter of 2020 compared to GAAP net loss of 37 million in second quarter of 20 tag team.

Net loss in the second quarter was expected due to the Kogut 19 impact on revenue and the overall timing of our quarterly revenue expenses for the year.

This quarter's net loss was considered when we provided our 2020 GAAP net income guidance earlier in the year.

With higher revenues and essentially flat SGN eight R&D expenses non-GAAP income of 57 million in the second quarter of 2012 also grew substantially as compared to Q2 29 teams. Both of these second quarter 2020 bottom line results keep us on track towards achieving our 2020 go full year GAAP net income.

For the first time in Biomarin history, and considerable growth in non-GAAP income.

With respect to total cash and investments we ended the second quarter of 2020 with 1.7 billion compared to $1.2 billion at the end of 2019, the increasing cash in Q2 was related to our May convertible note offering products $535 million net proceeds executed primarily to pay down.

Henry J. Fuchs: We're continuing to prepare new sites, and depending on the ongoing impact of COVID-19, we believe we could begin dosing in the Phase 1-2 study, nicknamed FEARLESS, later in the third quarter. We're excited about the prospect of BMM-307 as it represents a potential third PKU treatment option in our PKU franchise and a second gene therapy development program leveraging our learnings and capabilities from rock-bottom. As we prepare for the potential approvals and launches of Octavian and subsequently Borten sorotide, our earlier stage pipeline also... In July, we began IND-enabling studies for BMM331 gene therapy for hereditary O&M.

The 375 million of convertible notes due in October of this year.

Beyond the made.

Vertical note issuance and despite the cobot 19 impact on our business, we generated $28 million of positive operating cash flow in the second quarter 2020.

This solid cash position, coupled with Biomarin business fundamental put us in good standing to both managed through the uncertainty related to covert 19, as well as much rock gaming inventory tied should they be commercially approved.

Finally, I would like to discuss the significant one time item that we anticipate will impact GAAP net income in the second half of 2020.

As I mentioned on the Q1 conference call back in April we may recognize a tax benefit related to transfers of intangible asset between biomarin entities. As I mentioned previously you may have seen similar transactions completed by some of our larger peers over the last year.

Henry J. Fuchs: We expect the benefit from our experience with Octavian and BMN 307 to drive an even more efficient development program with BMN 331. In addition, we're pleased to announce in the second quarter our preclinical collaboration and license agreement with Dynacore, a gene therapy platform, to develop gene therapy for Rare Genetic Cardiomyopathy.

Our previous 2020, GAAP net income guidance of between 20 to 80 million noted that excluded the potential impact of these intangible asset transfers.

We now expect these transactions to occur the second half of 2020, which we estimate will result in a onetime noncash income tax benefit of approximately 700 million to 900 million.

In 2020.

Therefore, we have adjusted our full year GAAP net income guidance for this onetime tax benefit and now project that full year 2020, GAAP net income will be between 720 million and 908.

Henry J. Fuchs: These are big opportunities to buy a house where we can benefit from our growing expertise in gene therapy development and manufacturing. Before turning the call over to Brian for the financial... I would also like to acknowledge our colleagues across the organization for your continued commitment and contributions during these challenging but no less busy times. Extraordinary effort undertaken to submit marketing applications during a global pandemic is something no team can accomplish without. I've been impressed by your flexibility and focus, and you've demonstrated to me our progress moving forward under very challenging circumstances. Now I'll turn the call over to our recently appointed and very new Chief Financial Officer, Brian Mueller. Thank you, Hank.

The range acknowledges that we've not yet completed the intangible asset transfers and therefore, the final value cannot be determined that certainty and considering the high value of the underlying biomarin products. The amounts are significant.

Importantly, we note that our base business performance, excluding onetime items remains on track to generate GAAP net income for the full year for the first time and the company's history.

Also as this transaction impacts the income tax line item in our financial statements. We expect excluded from our non-GAAP income for which guidance remains unchanged at 260 $310 billion.

In closing, our second quarter results and trends towards normalization in the second half of the year. In addition to the potential approval of rock TVN indicate that 2020 should be a transformative year for the company on many levels.

Thank you for your support and we'll now open the call your question.

Ladies and gentlemen to ask your question. Please press star one on your telephone.

Brian R. Mueller: Please refer to today's press release summarizing our financial results for full details on the second quarter of 2020. As usual, our comprehensive report on the quarter will be available in our upcoming Form 10-Q, which we are planning to file later today. Starting with revenues, we've observed that the impact of COVID-19 on our commercial business is overall consistent with our expectations back in April. As Jeff mentioned, we experienced some modest top-line impacts from the COVID-19 pandemic in the second quarter that have continued into the start of the third quarter. We note that much of our business is beginning to stabilize and is on a trajectory toward normalized demand patterns. We considered this level of COVID-19 impact on our business when we revised guidance in April, and we remain committed to full year 2020 revenues of between $1.85 billion and $1.95 billion.

If you would like to withdraw your question. Please press the pound.

Your first question is from Joseph Schwartz from SVB, leaving.

Experiment.

Great. Thanks very much.

Just wondering if you're planning to employ any special tactics in order to support a successful launch of rock Caveon. During a pandemic period is there anything.

Based on your.

Wealth of experience launching different products that you think.

You might want to.

Bring out of your bag of tricks or any any new strategies, given the new world We're operating in.

Cash you want to ask the question.

Yeah happy to thanks, Joe for the question.

And this is going to sound perceptibly easy.

Easy to say harder to do in practice, but what we're having to do is to shift from largely not exclusively but largely from live interactions.

To virtual interactions and in the absence of having many more live interactions and we're increasingly turning to digital assets to help us communicate with with different commercial stakeholders.

Brian R. Mueller: Moving on to operating expenses, R&D expense for the second quarter was $182 million, slightly lower compared to R&D expense for the second quarter of 2019, which is mostly due to reduced R&D activity for Roktavion, given its late stage of development. SG&A expense for the second quarter of 2020 was $175 million, which is slightly higher than SG&A expense for the second quarter of 2019.

And I might comment that the virtual interactions of have gone pretty well.

With payer groups in particular.

The payers have have shifted very nicely to virtual interactions.

That that are not too different in quality from weibo interactions.

We've got a team on the ground the commercial and sales organization on the ground.

That are connecting with patient associations, providing.

Brian R. Mueller: As expected, the year-over-year increase is due to commercial preparation for the launch of Roktavion and the continued global launch of Palantir. Turning to bottom-line results, we reported a gap net loss of $29 million in the second quarter of 2020 compared to a gap net loss of $37 million in the second quarter of 2019. The net loss in the second quarter was expected due to the COVID-19 impact on revenue and the overall timing of our quarterly revenue expenses for the year.

Educational opportunities on gene therapy, those virtual and interactions give us an opportunity in some case the cast a broader net than we would if we were doing a live programming event and there's been enough live interactions.

With.

Hemophilia treatment centers and their associated infusion center locations that have allowed us to pursue.

Launch and infusion readiness activities for Ross Cavium in advance of launch with a smaller but perfectly adequate number of treatment sites to be ready at were shortly after launch to facilitate along so that's that's kind of a summary.

Brian R. Mueller: This quarter's net loss was considered when we provided our 2020 Gap Net Income Guidance earlier in the year. With higher revenues and essentially flat SG&A and R&D expenses, non-GAAP income of $57 million in the second quarter of 2020 also grew substantially as compared to Q2 2019. Both of these second quarter 2020 bottom line results keep us on track towards achieving our 2020 goals of full year gap net income for the first time in Biomarin's history and considerable growth in non-gap. With respect to total cash and investments, we ended the second quarter of 2020 with $1.7 billion compared to $1.2 billion at the end of 2019.

Very helpful. Thank you.

Your next question is from Salveen.

Mixture of Goldman Sachs.

Your line.

Good afternoon. Thanks for taking my question on recruiting people could you just walk us through the process for a patient with teeth that gene therapy outside of repair process. So basically how blood test sites have been of excellence and any other factors would come into play.

Yes, so savvy we are.

Our commercial organizations and medical affairs has been.

Working on.

I'm actually.

I'd and helping the patients who the whole process and Jeff Ken can describe that to you.

Yes. Thanks for the question Salveen and in fact, a lot of what happens with the patient journey for Ross avian will be very similar to the kind of patient journey that we've we've become accustomed to dealing with with the launch of.

Brian R. Mueller: The increase in cash in Q2 was related to our May convertible note offering that brought us $535 million in net proceeds. It was executed primarily to pay down the $375 million of convertible notes due in October of this year. Beyond the May 20th convertible note issuance and despite the COVID-19 impact on our business, we generated $28 million of positive operating cash flow in the second quarter of 2020. This solid cash position, coupled with Biomarin's business fundamentals, puts us in good standing to both manage through the uncertainty related to COVID-19 as well as launch rock caving and basaltites should they be commercially viable. Finally, I would like to discuss a significant one-time item that we anticipate will impact gap net income in the second half of 2020.

Burners and limits him for example, so.

Patients already Fortunately have a diagnosis, but they need to go through a diagnostic process to check for a the five.

Sarah negativity.

So there's a co diagnostic in there, but again relative to our enzyme programs. There's also this diagnostics step that we're we're adept at facilitating.

Education about the therapy I mentioned in response to Joe's question. I also noted that like we would do with the enzymes, we're focused on having a number of.

Treatment centers that are educated and ready to initiate therapy upon or shortly after launch so there's there's that step.

And then there's some preparatory step of patients need to take to get ready for infusion in parallel with that then probably the biggest rate limiting step would be.

Brian R. Mueller: As I mentioned on the Q1 conference call back in April, we may recognize a tax benefit related to transfers of intangible assets between Biomarin and, As I mentioned previously, you may have seen similar transactions completed by some of our larger peers over the last year. Our previous 2020 GAAP Net Income Guidance of between $20 to $80 million noted that it excluded the potential impact of these intangible asset trends. We now expect these transactions to occur in the second half of 2020, which we estimate will result in a one-time, non-cash income tax benefit of approximately $700 million to $900 million in 2020. Therefore, we have adjusted our full-year GAAP Net Income guidance for this one-time tax benefit and now project that full-year 2020 GAAP Net Income will be between $720 million and $980 million.

Would be going through the reimbursement approval process and again. This is stuff that we have a team that has been doing this in the United States and are very adept at that we've been doing this for many many years and then getting the infusion scheduled at an vision center.

It's only one infusion.

Again, not too dissimilar with our experience with the enzymes and one thing worth noting is there's a significant patient follow up.

For 52 weeks following infusion and again this looks a lot like the kind of patient support the biomarin provides for other enzyme replacement therapies. So there are a lot of really.

Really new and innovative aspects about rock cavium from a commercial perspective and relative to patients baseline standard of care. There's also a lot of parallels between what we'll need to do with rock Caribbean and what we already do with our base business. So we feel like we're leveraging a lot of competencies here.

Brian R. Mueller: The range acknowledges that we have not yet completed the intangible asset transfers, and therefore the final value cannot be determined with certainty, and considering the high value of the underlying Biomarin products, the amounts are significant. Importantly, we note that our base business performance, excluding this one-time item, remains on track to generate gap net income for the full year for the first time in the company's history. Also, has this transaction impacted the income tax line item in our financials? We expect to exclude it from our non-GAAP income, for which guidance remains unchanged at $260 to $310 billion.

Thank you saw being for the question.

Thank you.

Your next question is from Cory Kasimov JP Morgan Your line is no.

Hey, Thanks, Good afternoon, guys. Thanks for taking my question.

So that the W. W. Each treatment guidelines for hemophilia a were just updated I'm just curious whether this timing poses any potential problem for rock Kb in since it came out pre approval or would you expect them to be updated falling potential commercialization and on that note you have any color in terms of.

Yes, how meaningful guidelines might be on uptake curves for for hemophilia, a or or what we've seen in the path.

I mean could you be were specific as to what changes guidelines.

Great well. They just came out there was it was sent around earlier that World Federation hemophilia updated treatment guidelines and for it to happen now just in front of the approval there's commentary on gene therapy, but obviously nothing specific and so I didnt know.

Operator: In closing, our second quarter results and trends towards normalization in the second half of the year, in addition to the potential approval of Roktavion, indicate that 2020 should be a transformative year for the company on many levels. Thank you for your support, and we'll now open the call to your questions. And ladies and gentlemen, to ask a question, please press star 1 on your telephone keypad. If you would like to withdraw your question, please press the pound sign. Your first question is from Joseph Schwartz from SVB-LELINC. Please go ahead.

Thank you.

You interact with them at all or if they update these guidelines somewhat regular leader with new important new products are approved.

So just trying to get a better understanding what the dynamics might be on that front, whether it's to be.

A potential tailwind if they updated on yields of whatever kind of irrelevant data speak for itself in the study like this.

Again, I'm not aware that does guidelines are creating any kind of repayment for us so hang on for Jeff for you.

Joseph Patrick Schwartz: Great, thanks very much. I was just wondering if you're planning to employ any special tactics in order to support a successful launch of Roktavion during a pandemic period? Is there anything, based on your wealth of experience launching different products, that you think you might want to bring out of your bag of tricks or any new strategies given the new world we're operating in? So Jess, do you want to answer that question?

Airless.

Yes, I can take a first comment on this one and say thank wants to augment.

Thanks for noticing those treatment guidelines Corey impact treatment guidelines in the world of hemophilia, our longstanding they're very important and they do get updated periodically. So I don't think it's a problem that those treatment guidelines have updated in advance.

An approval of ABR Octavian.

I might expect that there would be another cycle and a reasonable period of time, perhaps prompted by the availability of.

Jeffrey Robert Ajer: Yeah, happy to. Thanks, Joe, for the question. And this is going to sound deceptively easy, easy to say, but harder to do in practice.

Gene therapies.

And that an approved gene therapy or therapies would be incorporated in those treatment guidelines in the meantime, I don't think that's an an impediment to the launch of rock they'd be in that as Hank stated in his prepared remarks. This community has been waiting for gene therapy for decades, there's a high.

Jeffrey Robert Ajer: But what we're having to do is to shift from largely, not exclusively, but largely from live interactions to virtual interactions. And in the absence of having many more live interactions, we're increasingly turning to digital assets to help us communicate with different commercial stakeholders. And I might comment that the virtual interactions have gone pretty well. With payer groups, in particular, the payers have shifted very nicely to virtual interactions that are not too different in quality from live interactions. We've got a team on the ground, a commercial and sales organization on the ground that is connecting with patient associations, providing educational opportunities on gene therapy. Those virtual interactions give us an opportunity, in some cases, to cast a broader net than we would if we were doing a live programming event. And there have been enough live interactions with hemophilia treatment centers and their associated infusion center locations that have allowed us to pursue launch and infusion readiness activities for Roctavian in advance of launch with a smaller but perfectly adequate number of treatment sites to be ready at or shortly after launch to facilitate a launch. So that's kind of a summary.

Huge amount of interest in Iraq, PBM and gene therapy in General I think we've got some tailwind in that regard already and without having new guidelines that actually incorporate gene therapy.

Okay, that's what I assume thanks for the other though Jeff.

I think the other thing just to add would be we've got we've dose to 134 patients in a phase three trials, we've got a lot of hands on experience now to share the community.

And we've been in lot of conversations medical symposia et cetera, because there's a great educational opportunity and as Jeff said, so it's almost like a sponge dry sponge for the information. So we were doing a lot of the work that you do in between guideline periods anyway.

Okay.

Thank you guys.

Okay. Next question is from Robin came out from Suntrust Securities. Your line is open.

Hi, guys. Thank you for taking the question it sounds like you're making a lot of progress getting.

All these up and ready for gene therapy.

Two questions on that point. So what are what are your expertise and giving updated thoughts and the eligible patient population I know you. That's 400 before if I believe and you gave some color around that last quarter any additional numbers. They were thought and then on that the viral testing for the virus.

Jeffrey Robert Ajer: Very helpful. Thank you. Your next question is from Salveen Richter of Goldman Sachs. Your line is open.

Salveen Jaswal Richter: Good afternoon, thanks for taking my question. On rock taping approval, could you just walk us through the process for a patient to receive the gene therapy outside of the payer process? So basically, how blood tests, sites of excellence, and any other factors would come into play?

Are there any patients that are getting that done or are there, possibly getting it done getting reimbursed head of the launch or should we expect that that will have to take place as you walk to the reimbursement process can be another step in the delay.

JJ Bien-Aime: Yeah, so Tavi, our commercial organizations and Medicaid Affairs have been working on actually guiding and helping the patients through the whole process, and Jeff can describe that to you. Thanks for the question, Salveen. And in fact, a lot of what happens with the patient journey for Roctavian will be very similar to the kind of patient journey that we've become accustomed to dealing with, with the launch of Brinura and Vimizem, for example. And then there are some preparatory steps that patients need to take to get ready for infusion. In parallel with that, then probably the biggest rate-limiting step would be going through the reimbursement approval process. And again, this is stuff that we have a team that's been doing this in the United States and is very adept at it.

Thanks.

Yes. Good question I mean, I could answer them that I guess is the expert there.

And.

And on the Jeff answer this question is.

I want to preemptive, okay. Thanks, JJ, great questions around and so just to reiterate.

We've modeled what we think is an appropriate.

Eligible patient population at launch of of 2400.

It's worth noting that every six months CDC numbers that estimate.

People with hemophilia in the United States are updated and they bounce around a little bit so those numbers could change over time, but.

And our modeling in estimates will be.

Based on whatever is finally in an approved label for Rockpile avian, but even if there is some variation around that 2400.

I think is a pretty solid number we're sticking with.

And that's just a launch and so a reminder, that with lifecycle management activities, we would hope over.

Jeffrey Robert Ajer: We've been doing this for many, many years. It's only one infusion, again, not too dissimilar to our experience with the enzymes. And one thing worth noting is that there is significant patient follow-up for 52 weeks following infusion. And again, this looks a lot like the kind of patient support that Biomarin provides for our other enzyme replacement therapies. So there are a lot of really, really new and innovative aspects to Roctavian from a commercial perspective and relative to patients' baseline standard of care. But there are also a lot of parallels between what we'll need to do with Roctavian and what we already do with our base business. So we feel like we're leveraging a lot of competencies here.

Over time to broaden that eligible patient population and then regarding your comment about the.

The co diagnostic to test for a by 85 Ciro positivity.

We're waiting for that.

Co diagnostic.

Good.

Probably 10 poorly in line with a rock baby in approval and so to my knowledge, there's not been an opportunity though.

Test for that in advance of of the launch Hank may have more specifics on that point.

All technically correct, what Jeff just said about the companion diagnostic we're planning that we're going to be approved with a requirement of companion diagnostic.

Salveen Jaswal Richter: Thank you, Salveen, for the question. Your next question is from Corey Kazma of J.P. Morgan. Your line is open.

We've been working with Citi RH in our laboratory partner to assure the availability the companion diagnostic a ton of rock saving approval you can't get the companion diagnostic without the command being approved so they're hinge together.

Corey Kazma: Hey, thanks. Good afternoon, guys. Thanks for taking the question. I saw that the WFH treatment guidelines for hemophilia A were just updated, and I'm just curious whether this timing poses any potential problem for Roctavian since it came out pre-approval, or would you expect them to be updated following potential commercialization? And on that note, do you have any color in terms of how meaningful guidelines might be on uptake curves for hemophilia A compared with what we've seen in the past? I mean, could you be more specific as to what's changing those guidelines? Well, they just came out.

The agencies, where the centers work together to time, Matt So it's not possible to be testing somebody for the companion diagnostic prior to its approval.

Okay, and what one follow up any color around important.

400 people through family friends of kind of like a hodgepodge number cause that decrease really rolled in hospitals.

So for this line that number.

General trending upwards or downwards.

Downward direction.

Yes.

Corey Kazma: It was sent around earlier that the World Federation of Hemophilia updated treatment guidelines. And for it to happen now, just in front of the approval, there's commentary on gene therapy, but obviously nothing specific. And so I didn't know if you interact with them at all or if they update these guidelines somewhat regularly or when important new products are approved. So just trying to get a better understanding of what the dynamics might be on that front and whether this would be, you know, a potential tailwind if they updated it on the heels of it. It's kind of irrelevant to let the data speak for itself in a setting like this.

Yes, I think Robin if I, if I heard you correctly youre inquiring about the number of opt ins for.

Further information that we've been collecting overtime and yes, the number of opt ins for further information has been increasing.

Commensurate with the kind of educational.

Activities that we've been doing on a virtual basis and also commensurate with people.

Accessing our our digital assets and opting in for further information. So I don't have specific numbers to share with you today, but.

It's an encouraging indicator of interest and hopefully future demand.

JJ Bien-Aime: Again, I'm not aware that these guidelines are creating any kind of impediment for us, so Hank or Jeff, are you aware of this? Yeah, I can take a first comment on this one and see if Hank wants to add anything. Thanks for noticing those treatment guidelines, Corey. In fact, treatment guidelines in the world of hemophilia are longstanding. They're very important, and they do get updated periodically. So I don't think it's a problem that those treatment guidelines have been updated in advance of the approval of Roctavian. I might expect that there would be another cycle in a reasonable period of time, perhaps prompted by the availability of gene therapies, and that an approved gene therapy or therapy would be incorporated into those treatment guidelines.

Great. Thank you guys. Thanks a lot.

Okay My dad.

One question right and your next question, it's been Matthew Harrison of Morgan Stanley.

Hi, Alex Thanks for taking the question. This is corner on from Matthew So just to quit from US. How are you guys thinking about pricing for rock JV and could you just comment on what kind of in fact generally you expect to see we'll be able to dose patients due to coven and then.

Just quickly on T.K. you what are the steps that you need to complete to start dosing for the PQ excuse me PK you gene therapy studies. Thank you.

Oh, sorry, Jeff.

Jeffrey Robert Ajer: In the meantime, I don't think that's an impediment to the launch of Roctavian because, as Hank stated in his prepared remarks, this community has been waiting for gene therapy for decades. There's a huge amount of interest in Roctavian and gene therapy in general. And I think we've got some tailwinds in that regard already without having new guidelines that actually incorporate gene therapy. Okay, that's what I assumed.

Can talk about pricing.

And Hank about but huge intently highly attrition I just.

Good morning, just took big one comment regarding the Philly dose patients treated just given your current environment and what are these your next question.

We do that went up.

Using using steroids.

Weve potentially latavia.

Hi, Matt.

As you might know some of you them unless you are probably keeping a few weeks ago in the UK.

You see waits for the treatment then management Opcone 19 patients and the study and actually showed that the stimulated is beneficial effect.

Corey Kazma: Thanks for that! I think the other thing just to add would be, you know, we've dosed 134 patients in a phase 3 trial, so we've got a lot of hands-on experience now to share with the community, and we've been in a lot of conversations, medical symposia, etc., because there's a great educational opportunity, and as Jeff says, it's almost like a sponge, a dry sponge Okay, thank you, guys. And your next question is from Robyn Karnauskas from Trust Securities. Your line is open.

I'll now coming 19 management, although I'm not personally recommend you do that but my point here is that.

It appears that if anything.

The need to or the Nike need to use utilizing the early days following treatment with ORKAMBI and should not be an impediment to.

You using rotary again based on this and bank I feel the fact that the steel is actually recommended approval of UK for the Matt maybe 90, so with that.

Well I'll then Jeff you mentioned the pricing question and then I can talk about PQ gene therapy.

Yes, okay. Thanks, JJ, so relative to pricing as we have with our other recent launches.

We'll disclose pricing.

When we have good fortune of having a product launch. So we hope will be soon so stay tuned for that and I'll turn it over the Hank regarding the gene therapy question.

Robyn Kay Shelton Karnauskas: Hi guys, thank you for taking the question. It sounds like you're making a lot of progress getting facilities up and ready for gene therapy. Two questions on that point. So what are your expectations? Do you have any updated thoughts on the eligible patient population? I know you said 400 before, if I believe it. And you gave some color around that last quarter.

The next steps are for clinical trial sites to.

Finished the paperwork that's involved.

To revise can sense for the covered pandemic and so there's are going through revisions and assume sites are up and ready for those single starting we project that will be in the third quarter.

Understood. Thank you.

And your next question is from sale net of Cowen and company. Your line is open.

Robyn Kay Shelton Karnauskas: Any additional numbers there or thoughts? And then on the viral testing for the virus, are there any patients that are getting that done, or are they possibly getting it done and getting it reimbursed ahead of the launch? Or should we expect that that will have to take place as you work through the reimbursement process and be another step in the delay? Thank you. Yes, two good questions. I mean, I could answer them, but Jeff is the expert there.

Good afternoon. Thanks for taking my question just one question on prototyping reimbursement.

Jeff I know you said during your prepared remarks that you've been able to engage with payers.

Virtually can talk a little bit more about what you've been able to show them.

Whether they understand the value of gene therapy in hemophilia.

Maybe lastly.

What type of reimbursement paradigm is going to predominate is there any desire to do alternative.

Reimbursement or is it wouldn't be a onetime upfront fee. Thanks.

Okay. Thanks for the question Phil Yeah.

JJ Bien-Aime: And I'll let Jeff answer those questions. Thanks, JJ. Great questions, Raman.

Our apparel launch actually started in Q4 of of last year under a safe Harbor provision.

Jeffrey Robert Ajer: So just to reiterate, we've modeled what we think is an appropriate eligible patient population at launch of 2400. It's worth noting that every six months, CDC numbers that estimate the number of people with hemophilia in the United States are updated, and they bounce around a little bit, so those numbers could change. And our modeling and estimates would be based on whatever is finally an approved label for Roktavian. But even if there's some variation around that, 2400, I think, is a pretty solid number we're sticking with. And that's just at launch.

Provided by the FDA for these types of situation. So we have a payer team.

That is very experienced same payer team launched pelon, taking the United States and launch printer in the United States. So good working relationships and access with payers.

We were able to put together the standard in the U.S., So called AMCP dossier, which is the basis upon which all payers evaluate new drugs, we've been able to educate them on.

Data related to our investigational program to date, we've been able to talk to them about their they're hemophilia book of business, which has been really positive in the sense that for all payers. Their hemophilia book of business is large it's material they all want.

Jeffrey Robert Ajer: And so a reminder that with lifecycle management activities, we would hope over time to broaden that eligible patient population. And then, regarding your comment about the co-diagnostic to test for AAV5 seropositivity, we're waiting for that to happen. Co-Diagnosed, probably temporally in line with Roktavian approval, and so, to my knowledge, there's not been an opportunity to test for that in advance of the launch. Hank may have more specifics on that point.

To do something about it.

And in combined with the.

The high level of interest on the part of payers in.

Gene therapies in general and how they're going to fit that into their business model. It's been a really nice combination we've gotten a lot of access and had a lot of really productive engagement.

Henry J. Fuchs: Well, I'll technically correct what Jeff just said about the companion diagnostic. We're planning that we're going to be approved with a requirement for the companion diagnostic. We've been working with CDRH and our laboratory partner to assure the availability of the companion diagnostic at the time of Roktavian approval. You can't get the companion diagnostic without the companion being approved.

The the whole outcomes based agreement.

Concept in the United States today has been limited by.

Government price reporting rules related to.

Medicaid and that's kind of limited the options. There is a proposed rule out from CMS that.

Henry J. Fuchs: So they're hinged together, and the agencies where the centers work together to time that. So it's not possible to be testing somebody for the companion diagnostic prior to its approval. Okay, and one follow-up. Any color around the 400?

Intends to address that as a proposed rule and the way it's propose it's not going to be relevant to our raw heavy and launch, but we've been working in the background to try to find a way of having in meaningful outcomes based agreement that stands behind what we expect to be the performance of brought Caribbean.

And that the.

Payers will find meaningful we're intending to go out to launch with that.

Robyn Kay Shelton Karnauskas: You said you identified 400 people through family and friends. It's kind of like a hodgepodge number. Has that increased as you've really drilled in with hospitals and got to know them in preparation for this launch? Is that number consistent, or is it, in general, trending in an upward direction or a downward direction? [inaudible] Yeah, I think, Robyn, if I've heard you correctly, you're inquiring about the number of opt-ins for further information that we've been collecting over time. And yes, the number of opt-ins for further information has been increasing, commensurate with the kind of educational activities that we've been doing on a virtual basis, and also commensurate with people accessing our So I don't have specific numbers to share with you today, but it's an encouraging indicator of interest and, hopefully, future demand. Thank you guys. Thanks a lot.

Well details are confidential at the moment and we expect that that would likely result in a onetime recognition of revenue for.

Rob TVN as opposed to.

Recognition of revenue streamed over time for example.

Okay.

That's helpful. Thank you.

[laughter].

Your next question, it's from Chris Raymond Fibers Pharma your line is open.

Great. Thanks, Hi, This is alley brats on for Chris I.

Another question on rock TVN.

Yeah, you landscape.

We noticed that last month, Ralph took an impairment on spark gene therapy asset on the rationale that hadn't Libra has shifted the goalpost for gene therapy efficacy and safety. So just curious to get your thoughts on that based on your interactions with the physician and patient community ahead of launch.

Operator: And that's a reminder to ask one question at a time. And your next question is from Matthew Harrison of Morgenstern. Hi all, thanks for taking the question. This is Connor on behalf of Matthew.

It yourself that handling for has meaningfully change patients going that's to be honest with rocky dinner or other gene therapy approaches.

Matthew Harrison: So just two quick questions from us. How are you guys thinking about pricing for Rukavian? And could you just comment on what kind of impact you expect to see with being able to dose patients due to COVID? And then, just quickly, on PKU, what are the steps that you need to complete to start dosing for the PKU gene therapy studies? Thank you.

How does that actually comment about the patient level like decision, making on <unk> gene therapy. Thanks.

Thank you want to sell I mean actually we not really I think there was a question. So as I know there was a protect question about a potential impairment to begin my rose, but I don't think we have taken a fair amount Sars I know that's just for clarification purposes.

It's really too.

We did their acquisition of Oh, spark, which has a somewhat different profile.

JJ Bien-Aime: Uh, other stories, and Jeff, uh, can talk about pricing, and Hank about the PKU gene therapy trial initiation. But I just want to make one comment regarding the ability to treat patients with Octavian in a COVID environment. One of the issues and questions we've had related to that was, I know, using steroids. As some of you may know, there was actually a publication a few weeks ago in the UK using steroids for the treatment and management of COVID-19 patients. And that study actually showed that steroid therapy had a beneficial effect on COVID-19 management, although I'm not personally recommending you do that. But my point here is that, if anything, the need or the likely need to use steroids in the early days of treatment with Roctavians should not be an impediment to using Roctavian based on this.

From rotating out in time.

Oh, so anchor Jeff do you want to make some comments.

Well the question.

Well, one comment would be too.

Not a familiar with the impairment either but I would say that the Ada one one data.

Hi, its th didn't particularly exceed expectations I mean, it is one thing that is interesting about the spark status is that can get gene transfer at relatively low you can get hemostatic efficacy or relatively low levels of gene expression. So I think like at two years their data, it's like half of our data two years. So.

Even if we drift downward prognosis accurately the factory debtor, yet, yes, they had bleeding control at half the factory levels that we had.

And so that practice as well if our factory levels Doosan factor of Dallas should be able to maintain hemostatic efficacy for all but I I don't think that their safety profile I think your safety profile, then obviously profile warrants further investigation.

JJ Bien-Aime: But I feel the fact that steroids are actually recommended and approved in the UK for the management of COVID-19. So with that little preamble, maybe Jeff could answer the pricing question, and then Hank can talk about PKUG therapy. Relative to pricing, as we have with our other recent launches, we'll disclose pricing when we have the good fortune of having a product launch, which we hope will be soon, so stay tuned for that, and I'll turn it over to Hank regarding the gene therapy question. Next steps are for clinical trial sites to finish the paperwork that's involved, you know; we've had to revise consents for in the COVID pandemic And so those are going through revisions, and as soon as sites are open and ready for dosing, we'll start, and we project that will be in the third quarter. Understood, thank you.

And as far as patients preferences for humans in hemophilia you know as I said in my comments at the community has been dreaming about having natural factory made endogenously for years and years heme labor as a fantastic advancements it's not for everybody patients have breakthrough bleeding and we think in we think infill gene therapy is going.

To be a really solid offering in this community maybe Jeff you want to entering.

Yeah, I would I would just add that them.

Im LIBOR is certainly change the treatment landscape. It is also broken through the myth that hemophilia patients are.

Unwilling to change therapies so.

If that is kind of broken through the barrier of switching that could also bode well for another.

Big advancement coming to the market, mainly brought pay being a food so fortunate with an approval.

And your next question, it's far from Josh Schimmer of Evercore ISI. Your line is open.

Philip M. Nadeau: And your next question is from Phil Nadeau of Collin & Company. Good afternoon, thanks for taking my question. Just one question on Roktavian reimbursement. Jeff, I know you said during your prepared remarks that you've been able to engage with payers virtually. Can you talk a little bit more about what you've been able to show them?

Great. Thanks for taking my question can you discuss your expectations for Covanta erosion as generic centre in the U.S. what percent on brand do you think you can retain over the mid and long term and white. Thanks.

Yes, you want to go over that.

Hi, Josh Thanks for the question I.

I think just the ground everybody.

We have a loss of exclusivity until then in the United States coming in on October 1st we've anticipated and prepared for this for a long time also.

Philip M. Nadeau: whether they understand the value of gene therapy in hemophilia, and maybe lastly, what type of reimbursement paradigm is going to predominate? Is there any desire to do alternative reimbursements, or is it likely to be a one-time upfront? Okay, thanks for the question, Phil.

Remind and reinforce about 75% of our business in Q ban is coming from the United States. Presently. So this is a big event for us.

Jeffrey Robert Ajer: Yeah, our payer launch actually started in Q4 of last year under a safe harbor provision provided by the FDA for these types of situations. So we have a payer team that is very experienced, the same payer team that launched Palantik in the United States and that launched Brnura in the United States. So, thanks to good working relationships and access to payers, we were able to put together the standard in the U.S., the so-called AMCP dossier, which is the basis upon which all payers evaluate new drugs.

What's unclear Josh is.

How a generic erosion curve applies to a specialty product like QB and there is not very many give analogs to study the ones that we have have complicating factors.

So it's a little bit difficult to guide to I and in in the background. You know that Biomarin provides a lot of patient support services and reimbursements services to patient so.

We've worked really hard to build a value added relationship that is recognized by all of patients their providers and also payors that said, we're expecting a significant erosion of our business.

Jeffrey Robert Ajer: We've been able to educate them on data related to our investigational program to date, and we've been able to talk to them about their hemophilia book of business. Which has been really positive in the sense that for all payers, their hemophilia book of business is large, it's material, and they all want to do something about it. And combined with a high level of interest on the part of payers in gene therapies in general and how they're going to fit that into their business models. It's been a really nice combination.

We don't think it will be the immediate catastrophic type of lost some share, but you might expect for a retail pharmacy product going generic and beyond that I'm afraid we can't offer a lot more specific guidance early so I can.

Hey, guys you covered it.

Jeff This is only impacting us business.

There are no generics no loss of exclusivity at this time in Europe.

Jeffrey Robert Ajer: We've gotten a lot of access and had a lot of really productive engagements. You know, the whole outcomes-based agreement concept in the United States to date has been limited by government price reporting rules related to Medicaid, and that's kind of limited the options. There's a proposed rule out from CMS that intends to address that.

Yes.

Your next question, it's from job mission of Bank of America.

Hey, guys. Thanks for taking my question.

I know the focus obviously is on U.S.

Rock Smart TV and long point ask you in Europe, though.

What do you need to do in advance.

Launch, maybe just help us with some of the nuances.

Jeffrey Robert Ajer: As a proposed rule and the way it's proposed, it's not going to be relevant to our Rocktavian launch, but we've been working in the background to try to find a way of having a meaningful outcomes-based agreement that stands behind what we expect to be the performance of Rocktavian and that the payers will find meaningful. We intend to go out to launch with that. Well, details are confidential at the moment, but we expect that that would likely result in a one-time recognition of revenue for Rocktavian, as opposed to recognition of revenue streamed over time, for example.

Between you and.

In Europe in terms of how peers delivered.

For Hemophilia and then just talk about maybe your visibility.

Thank you.

Perhaps you want to cover that.

Yes, Thanks, Jeff and Great question. So there are a lot of similarities between how.

How hemophilia is treated in Europe and in the United States. One thing to note is there's a greater concentration of care in centers of excellence in the major European markets and as we've worked with stakeholders in Europe to prepare for an eventual.

Philip M. Nadeau: That's helpful, thank you. Your next question is from Chris Raymond of Fiber Sandler. Your line is open. Great, thanks. This is Allie Bratzelon for Chris.

Lunch.

We've been encouraged in major markets to kind of work with.

Con concentrated said centers of excellence, which is actually working really well for us because it.

Christopher Joseph Raymond: Another question on Roctavian and the hemophilia A landscape. We noticed that last month Roche took an impairment on the SPARC gene therapy asset on the rationale that Hemolibra has shifted the goalposts for gene therapy efficacy and safety. So just curious to get your thoughts on this based on your interactions with the physician and patient community ahead of launch. Is it your sense that Hemolibra has meaningfully changed patients' willingness to be dosed with Roctavian or other gene therapy approaches? Or how does that actually come into patient-level decision making on gene therapy? Thanks.

It limits the number of resources that we need to habit launch.

Out in the field working with treatment centers and then as you know the biggest gating factor.

In Europe is going to be related to navigating price on reimbursement approvals.

And as you know from watching our most recent launches of balance peak and bravura, even where there's a a high need that's being addressed those things can take time.

JJ Bien-Aime: I think there was a question about a potential impairment to be taken by Roche, but I don't think they have taken an impairment, as far as I know. That's just for clarification purposes. And that is related to their acquisition of Spark, which has a somewhat different profile from..., from Roktevin at this time. So Hank or Jeff, do you want to make some comments?

Particularly were being slow down from covert 19 related issues on that front in Europe.

The big upside in Europe of course is Theres, a bigger patient population. So overall the patient opportunity in Europe is very large and even if it takes a little what while longer too.

Tap into if we have four so fortunate of having a U.S. launch followed by any launch we would expect more rapid uptake in the United States, but a bigger opportunity in terms of numbers of patients overall from Europe. Thank you Jeff.

Henry J. Fuchs: Well, one comment would be to... I'm not familiar with the impairment either, but I would say that the 8011 data at ISTH didn't particularly exceed expectations. I mean, one thing that is interesting about the SPARC data is that you can get gene transfer at relatively low levels; you can get hemostatic efficacy at relatively low levels of gene expression. So I think, at two years, their data was like half of our data at two years, so even if we drift down a little bit on the prognosis. The factor VIII data, yeah. They had bleeding control at half the factor VIII levels that we had.

Okay. Thank you.

Your next question is from Martin asked true ups credits.

Hi, everyone. This is mark on for Marty. Thanks for taking my questions. So so you mentioned earlier that there are there maybe 2400 immediately address fall hemophilia patients in the U.S. at the time of Octavian launch can you speak to what the processes to expand the label, what those timelines might be and all.

Henry J. Fuchs: And so that prognoses well if our factor VIII levels do in fact drift down, we should be able to maintain hemostatic efficacy for a while. But I don't think that their safety profile; I think their safety profile and efficacy profile warrants further investigation. And as far as patients' preferences in hemophilia, you know, as I said in my comments, the community has been dreaming about having natural factor VIII made endogenously for years and years. HemeLibra is a fantastic advancement. It's not for everybody, but patients have breakthrough bleeding, and we think hemophilia gene therapy is going to be a really solid offering in this community. Maybe Jeff, do you want to add anything?

Ultimately what portion of the population do you think could eventually become eligible for your drug. Thank you.

We are all program here over the next few years to expand the.

The addressable population.

I can just can outline this if you're interested.

We anticipate a very significant increase in the eligible population overtime study right now the number we're giving you is about 14% of U.S. population.

But.

You will be up a population.

That's all commerce not just of your patience.

This could expand to probably for you a 50% of operation over time, but.

Thank you I Skyworks tribe anyway, Thanks, Jeff.

Henry J. Fuchs: Yeah, I would just add that Hemlibra has certainly changed the treatment landscape. It has also broken the myth that hemophilia patients are unwilling to change therapies. So if that kind of breaks through the barrier of switching, that could also bode well for another big advancement coming to the market, namely broctavian if we're so fortunate with an approval. And your next question is from Josh Schimmer of Evercore ISI. Your line is open. Thanks for taking the question. Can you discuss your expectations for Kuvan Erosion as a generic center in the U.S.? What percent of the brand do you think you can retain over the mid and long term, and why?

Yes.

Sure that the two biggest.

Vince to go after immediately in terms of lifecycle our.

Patients, who have preexisting immunity to the cap today, the five and we actually have an ongoing study and we're collecting data on the impact of preexisting immunity on factory expression and bleed control.

That's ongoing sanyal slowly, but its ongoing.

Second major area for investigation, we'd be in.

Patients represent had inhibitors about 25, 35% or the hemophilia patient population has inhibitors to.

To factory they've been excluded from clinical trials.

Josh Schimmer: Jeff, do you want to go over that? Hi Josh, thanks for the question. I think just to ground everybody, we have a loss of exclusivity on Kuban in the United States. Coming on October 1st, we've anticipated and prepared for this for a long time. Also, to remind and reinforce, about 75% of our business in Cuban is coming from the United States presently, so this is a big event for us. What's unclear, Josh, is how a generic erosion curve applies to a specialty product like Cuban coffee. There are not very many good analogs to study, and the ones that we have have complicating factors, so it's a little bit difficult to guide them.

We've talked about patients with pre existing liver disease.

We've talked about are starting to dose adolescents or could be quite a lot of excitement about.

Bringing a drug like Directv into an earlier age population for better preservation of overall health.

Those are some of the lifecycle thoughts must much of which is already underway.

And as.

The first thing to do the first step to achieve label expansion is to finish the label. So that's where we are.

Perfect. Thank you, Jeff I don't know yes.

Thank you Ed for nothing further to add that was complete thank you.

Jeffrey Robert Ajer: And in the background, you know that Biomarin provides a lot of patient support services and reimbursement services to patients, so we've worked really hard to build a value-added relationship that is recognized by all patients, their providers, and also payers. That said, we're expecting a significant erosion of our business. We don't think it will be the immediate catastrophic type of loss of share that you might expect for a retail pharmacy product going generic. And beyond that, I'm afraid we can't offer a lot more specific guidance, or at least I can't. You covered it, Jeff, that this is only impacting the U.S. business, you know; there are no generics, and no loss of exclusivity at this time in Europe. Your next question is from Joss Misham of Bank of America.

So before the next question just you know point of clarification UQM, we're looking to information regarding Roche.

Right up it looks like it took a $40 million write off in their most recent quarter, which is I guess.

Personally.

Our vision cost of a of spark and it looks like if they're finding this is the main reason is a delay in the implementation of the phase two and phase III trial, because it could be 19, which is likely to result in lower sales, we would agree with that.

Next question.

Your next question is attached why are you wont see stretch.

Attaching your line is open.

Hi, I'm sorry, sorry.

Thanks.

Can you walk us through the incentive structure for Octavian.

For doctors, particularly at the 340 be level.

Well, maybe getting a percent of the total hard rock Cavium and how do you think that those in fact this will impact the initial launch for 20000 megawatt.

Jeffrey Robert Ajer: Hey guys, thanks for taking the question. I know the focus, obviously, is on U.S. Valrocks and Roktevian launches. I wanted to ask you, in Europe, though, what you need to do in advance of that launch? Maybe just help us with some of the nuances between the U.S. and Europe. Jeff, do you want to cover that? Yeah, thanks, Jeff. Great question.

I mean I could start.

I mean, all I mean, the majority of our thinking that the majority of.

He will begin treatment centers are our 340 hospitals in the.

Consequently, most patients will be two conceivably be hospitals.

Generally see formerly hospitals get a 21, 22%.

Discount for most drugs Oh, we also understand thus far.

We submitted factories and also read that people are indeed that.

Joss Misham: So there are a lot of similarities between how hemophilia is treated in Europe and in the United States. One thing to note is that there is a greater concentration of care in centers of excellence in the major European markets. And as we've worked with stakeholders in Europe to prepare for an eventual launch, we've been encouraged in the major markets to kind of work with a concentrated set of centers of excellence, which is actually working really well for us because it limits the number of resources that we need to have at launch out in the field working with treatment centers. And then, as you know, the biggest gating factor in Europe is going to be related to navigating price and reimbursement approvals. And as you know from watching our most recent launches of BALANZIC and BRNURA, even where there is a high need that's being addressed, those things can take time. Particularly, we're being slowed down by COVID-19-related issues on that front in Europe.

Discounted be reduced to 70% so we likely to start with 21% we might eventually get would you 70% did not payments are made directly to the treatment centers.

I would say that we don't believe there is a disease that is there to use with TVN because right now, let's say excuse me 5 billion from us.

Oh, you the value of that sure instead of a care.

Centers getting those 20% also discussed before you can get them Antonio from payments are made directly to the centers.

Thank you Ed yes.

That was well stated JJ. Thank you, though the only other thing I would add to that is.

There may be a perception that.

That these treat hemophilia treatment centers are concerned about losing.

Revenue streams that are ongoing over time from existing patients receiving.

Back to replacement therapy chronically.

Our research indicates that.

Hemophilia treatment centers.

Have a revenues 340 be revenue stream from only a portion of the patients under their care.

Jeffrey Robert Ajer: The big upside in Europe, of course, is that there's a bigger patient population. So overall, the patient opportunity in Europe is very large. And even if it takes a little while longer to tap into, if we're so fortunate of having a U.S. launch followed by an EU launch, we would expect more rapid uptake in the United States but a bigger opportunity in terms of numbers of patients overall from Europe. Thank you, Jeff. Okay, thank you. Your next question is from Martin Alster of Credit. Hi, everyone. This is Marc on behalf of Marty.

And that just kind of amplifies Jay's comments, we think that in fact, the threeforty be margin that these treatment centers would likely be able to take from rock Fabian.

I would be significant than who would be good incentive.

That's really helpful. Thank you.

And I would like to turn the pull back a JV animal chairman and CEO for closing comments.

Thank you operator, so now we are over halfway through 2028.

I have continued confidence in.

His ability to deliver on the.

Martin Alster: Thanks for taking my questions. So you mentioned earlier that there may be 2400 immediately addressable hemophilia patients in the US at the time of Octavian's launch. Can you speak to what the process is to expand the label, what those timelines might be? And ultimately, what portion of the population do you think could eventually become eligible for your drug?

Just opportunity for value creation that we have ahead of us.

Our underlying fundamentals remain strong and we're well positioned for achieving our mission. We built a successful based business strategy transition our pipeline to address larger rare indication.

Weve laid the foundation for significant profitability with.

If you then there's always got approval.

Oh this position by me for substantially accomplishment in person near term and long term. So we are we looking forward to the remainder of 2020, we apologize if we can get to your question is because we limited to any to Warner.

JJ Bien-Aime: Thank you. We have a whole program here over the next few years to expand the... The addressable population, you know, Hank and Jeff can outline this if you're interested, uh... but we anticipate a very significant increase in the eligible population over time, starting right now. The number we're giving you is about fourteen percent of the U.S. population, but uh... of the U.S. hemophilia A population, that's all comers, not just severe patients. I think this could expand to probably forty or fifty percent of the population over time, but if you Hank you want to start over describing what we're planning to do, and Jeff can add some comments, Sure.

Yes pressures that we're not.

Last year during the call suites.

We don't do we tried to Tracy Mcgrady and we can as hopefully we can address your question. So thank you all for your continued support and Stacey.

Ladies and gentlemen, this concludes todays conference call. Thank you for participating you may now disconnect with Suntrust. Please stay on the line for your first time frame.

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Henry J. Fuchs: The two biggest segments to go after immediately in terms of life cycle are patients who have pre-existing immunity to the capsid AAV5. And we actually have an ongoing study, and we're collecting data on the impact of pre-existing immunity on factory expression and bleed control. So that's ongoing. It's going a little slowly, but it's ongoing. A second major area for investigation would be in patients who have had inhibitors. About 25-35% of the hemophilia patient population has inhibitors to factory, so they've been excluded from clinical trials.

Henry J. Fuchs: We've talked about patients with pre-existing liver disease. We've talked about starting to dose adolescents. There could be quite a lot of excitement about bringing a drug like Ractavian to an earlier age population for better preservation of overall health.

Henry J. Fuchs: Those are just some of the life cycle thoughts, much of which is already underway, and the first thing to do, the first step to achieve label expansion, is to finish the label. So that's where we are. Perfect. Thank you. Jeff, I don't know. Yeah.

Jeffrey Robert Ajer: Jeff, do you have anything to add? There was nothing further to add. That was complete.

JJ Bien-Aime: Thank you. So before the next question, just a point of clarification. We actually looked into information regarding Roche's write-off. It looks like they took a $400 million write-off in their most recent quarter, which is, I guess, less than 10% of the acquisition cost of SPARC. And it looks like in their filing, they say the main reason is the delay in the implementation of the phase 2 and phase 3 trials because of COVID-19, which is likely to result in lower sales. And we would agree with that.

JJ Bien-Aime: Your next question is from Akash Tewari of Walsh Research. Akash, your line is open. Hi, sorry. So can you walk us through the incentive structure for Octavian for doctors, particularly at the 340B level? Will they be getting a percent of the total cost of Octavian? And how do you think that those incentives will impact the initial launch for 2020 and beyond? Thanks a lot.

JJ Bien-Aime: Uh, I mean, I could start, uh, if you need, I mean, all, I mean, the majority of, uh, I'm just saying that the majority of hemophilia treatment centers are 340B hospitals in the U.S. Consequently, indeed, most patients will be treated in 340B hospitals. Uh, generally, 340 V hospitals get a 21 or 22 percent... Discounts for most drugs, we also understand that for... We saw in the factories and also recently for Himnibra that the discounts have been reduced to 17%. So we'd like to start with 21%; we might eventually get reduced to 17%. These are payments that are made directly to the treatment centers. I would say there is no disincentive there to use Roktavion because right now, let's say we price Roktavion for about... For more information, please visit www.

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JJ Bien-Aime: FEMA.gov. Anything to add, Jeff? That was well stated, JJ. Thank you. The only other thing I would add to that is there may be a perception that these hemophiliac treatment centers are concerned about losing revenue streams that are ongoing over time from existing patients receiving Factor Replacement Therapy. However, chronically, our research indicates that hemophilia treatment centers.

JJ Bien-Aime: [inaudible] That's really helpful, thank you. And I would like to turn the call over to JJ Vianime, Chairman and CEO, for closing comments. Thank you, operator. So now we are over halfway through 2020. I have confidence in Biomarin's ability to deliver on the tremendous opportunity for value creation that we have ahead of us.

JJ Bien-Aime: Our underlying fundamentals remain strong, and we are well-positioned for achieving our mission. We've built a successful base business. We've transitioned our pipeline to address larger, rarer indications, and we've laid the foundation for significant profitability with... Octavian, and Zoetat approvals. All of these position Biomarin for substantial accomplishments in both the near term and the long term.

JJ Bien-Aime: So we are looking forward to the remainder of 2020. We apologize if we couldn't get to your questions, because we limited the Q&A to one hour. Please, if you have questions that were not addressed here during the call, please reach out to Traci McCarty, and hopefully we can address your questions. So thank you all for your continued support, and stay safe. And ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. Presenters, please stay on the line for your post-conference. © BF-WATCH TV 2021, ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? ??? © BF-WATCH TV 2021