Q3 2020 Biomarin Pharmaceutical Inc Earnings Call

See Mccarthy Vice President of Investor Relations. Please go ahead ma'am.

Traci McCarty: Traci McCarty, Vice President of Investor Relations Thank you, Joanna, and thank you all for joining us today. 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, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors. And those factors are detailed in Biomarin's filings with the SEC, including 10Q, 10K, and 8K reports. On the call remotely, well, some are remote and some are sitting here together, are from Biomarin Management today: JJ Piename, Chairman and Chief Executive Officer, Jeff Ager, Executive Vice President, Chief Commercial Officer, Hank Fuchs, President, Worldwide Research and Development, Greg Geyer, Executive Vice President, Chief Technical Officer, and Brian Mueller, Executive Vice President, Chief Financial Officer. We hope to keep this call to one hour, so we respectfully request that Thank you for your understanding. I will now turn the call over to Biomarin's Chairman and Chief Executive Officer, JJ Piename. Thank you, Traci.

Thank you Joann and thank you all for joining us today.

Mind you. This non confidential presentation contain forward looking statements about the business prospects, the biomarin pharmaceutical inc., including expectations regarding financial performance.

Actual product potential future products in different areas of therapeutic research and development results may differ materially depending on the progress the fiber and product program actions of regulatory authorities availability of capital future actions of the pharmaceutical market development by competitors and those factors detailed in <unk> filings with the <unk>.

See including 10-Q, 10-K and 8-K.

On the call remotely well somewhat remote and some are sitting here together.

Our from Biomarin management today, JJ forget M&A, Chairman and Chief Executive Officer, Jeff Eight your executive Vice President Chief Commercial Officer, and compute President worldwide Research and development Bright Guy or executive Vice President Chief Technical Officer, and Brian Mueller Executive Vice President 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 and a portion of the call. Thank you for your understanding I will now turn the call over to Barbara <unk>, Chairman and CEO, Jay maybe out of me. Thank.

JJ Piename: Good afternoon, and thank you all for joining us on today's call. We hope you and your families have been well during these uncertain times. It is hard to believe that we are rounding month 9, of the global COVID-19 pandemic. Like many of you, we are ready for a reset and a fresh start in 2021, but our commitment to providing innovative treatments to people with this has focused day-to-day. Indeed, Biomarin faced some challenges in the third quarter, and the results have been strengthened conviction in our mission and renewed motivation to achieve it. The most anticipated milestone of the year, the advancement of Rotevian gene therapy for the treatment of severe hemophilia, and Rosorita for the treatment of achondroplasia, each reached a key inflection point in their progression during the course of the year.

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

We hope you and your families have been well during these uncertain times.

It's hard to believe that we are rounding miles nine.

Oh, no global Koby 19 pandemic.

Like many of you we are ready to a four week, that's not a crush starting 2021.

But our commitment to providing innovative treatments to people we.

Jews conditions keeps us focused day to day.

If I am going to face some challenges in the third quarter.

And the results have been strengthened.

And you know mission and reuse motivation to achieve it.

Good morning, Mike.

The year the advancement of room can be a gene therapy for the treatment of hemophilia.

Are we talking for the treatment of equal drumbeat, yeah, each region Eightyc infection point.

Their progression during the quarter.

There's always an obligation of the U.S. and Europe were accepted.

JJ Piename: Zoetide applications in the U.S. and Europe were accepted for review by health authorities and are in the process for potential approval in 2021. The CHMP opinion is expected in the second half of next year, and the U.S. PDUFA action date is planned for August 20th of next year. The community of parents with children who have a congenital pancreas and want a treatment option are that much closer to accessing the first potential pharmacological therapy for this condition.

JJ Piename: With six highly innovative products developed, approved, and commercialized for a variety of rare conditions over the last two decades, this is familiar and gratifying territory for Biomarin. Turning to Octavian, one of the most innovative products we have developed. We have transitioned to a more traditional regulatory timeline than originally anticipated for this one-time gene therapy infusion that helps control bleeding in people with severe hemophilia A. However, as is typical for Biomarin, we pursued an accelerated development strategy with Rotavian, which resulted in the significant advancement of the field and greater awareness of gene therapy as an important new therapeutic platform for the treatment of hemopoietic AI.

JJ Piename: And while this platform has been at the research stage for decades, the speed with which Biomarin brought Octavian forward for regulatory review was unprecedented, even for us. Our belief in the therapeutic benefit of Rotavian for our patients has not wavered. To complete one-year phase three data from all 134 patients is paramount to determining next steps, and we look forward to sharing those top line results with you early in 2021. Based on the bleeding control observed to date with Roptivian, we have confidence in the approval and patient interest in this product.

Yes.

And we look forward to sharing boost outline results with you in.

In early 2021.

Based on the building control observed to date with Rotarian, we have confidence in year prudently and patient interest in this product.

JJ Piename: In a moment, Hank will provide more details on our plans over the coming months. Meanwhile, briefly on our third quarter financial results, I have been impressed with the commitment of our commercial and manufacturing organizations in the face of the seemingly never-ending impact of COVID-19. They have not missed a beat. Our ability to function under the limitations imposed by the global pandemic remains a testament to the importance of our therapy. All of those are diversified, product-based, and have far-reaching commercial footprints.

In a moment Hank would provide more details on our clients over the coming months.

We see on the third quarter financial results Ivy impressed with a commitment of our provincial in manufacturing organizations.

In the face of the seemingly never ending impact of COVID-19.

Have not missed the beach.

Our ability to execute under the limitations imposed by the global pandemic remains a testament to the importance of our or therapies.

Are diversified product base and far reaching commercial footprints.

JJ Piename: Chef will provide details of the quarter and updates on 2020 guidance in a moment. We have met the adversity of 2020 with versatility and agility. In what has been the most unpredictable year we have experienced so far at Biomarin, the resilience of our business and the fortitude of our employees have consistently shown. As a result, we remain well-positioned for substantial growth as we focus on the execution of our global opportunities with Octavian and Rosaritide, as well as other key pipeline products. And we want to thank you for your continued support. Now, I would like to turn the call over to Jeff to discuss the commercial business update. Thank you, JJ.

We will provide details of the quarter and updates of 2020 guidance in a moment.

We have met the adversity of 2020 with versatility in agility.

And what has been the most unpredictable year, we have experienced so far at Barbara in the resilience of our business unfortunate you to our employees as consistently show through.

Ah the results will remain well positions.

Or substantial growth as we focus on the execution of our global opportunities with Octavian in Missouri died as well as other key pipeline products and we want to thank you for your continued support.

I would like to turn the call over to Jeff to discuss a commercial business object Jeff.

Thank you J J.

Jeffrey Robert Ajer: Globally, Biomarin commercial brands contributed $1.24 billion, or 8% growth year to date in 2020 compared to the first nine months of 2019. Given the challenges imposed by the COVID pandemic this year, I'm very pleased with our results and the deep commitment of our commercial and manufacturing teams to our patients around the world. Beginning first with our ERT brands for MPS, Femizum, and Naglozyme, I'll share some details. For Naglozyme, quarterly revenue of $76 million represented a decrease compared to Q3 results last year due to COVID-19 impact and order timing, and slightly down year-to-date compared to the same period in 2019. BIMISM results of $148 million in the third quarter followed a similar pattern, with decreased quarterly revenue slightly down year-to-date compared to the same period in 2019.

Liberally Biomarin commercial brand contributed $1.24 billion or 8% growth year to date in 2020 compared to the first nine months of 2019.

Give them challenges opposed by the Covid pandemic this year.

I'm very pleased with our results and the deep commitment of our commercial and manufacturing team store patience around the world.

Getting first with our ERP brands for M. P. S. <unk> I'm also have some details.

<unk> quarterly revenue $76 million represented a decrease compared to Q3 results last year due to COVID-19 impact and order timing.

And slightly down year to date compared to the same period in 2019.

The Muslim the results of $148 million in the third quarter, followed a similar pattern with decreased quarterly revenue slightly down year to date compared to the same period in 2019. These.

Jeffrey Robert Ajer: These solid results, despite the impact of the global pandemic, reflect the essential nature of maglozyme and BIMISM for our patients. In addition, our mitigation efforts around misinfusions in key markets have also contributed to solid results in the quarter. Despite the global pandemic, demand and revenues have stabilized for the third quarter for these two flagship products, although delays and new starts have persisted broadly since the start of the pandemic. Disruption in clinic operations and disruption of patients pursuing diagnoses are the drivers behind this factor.

The solid results despite the impact of the global pandemic reflect the essential nature of magazine I'm in Vimizim to our patient. In addition are mitigation efforts around Miss infusions and key markets have also contributed to solid results in the quarter.

Despite the global pandemic demand and revenues have stabilized for the third quarter for these two flagship products.

Delays and new starts have persisted broadly since the start of the pandemic.

Disruption in clinic operations and disruption of patience pursuing diagnoses or the drivers behind this factor.

The impact in any one quarter has been small yet the cumulative effect on the growth of patience on commercial therapy is now material and has led to the slowing of projected road.

Jeffrey Robert Ajer: The impact in any one quarter has been small, yet the cumulative effect on the growth of patients on commercial therapy is now material and has led to the slowing of projected growth. As a result of these dynamics playing out in our global territories, we have narrowed the range for VIMISM, full-year guidance, to between $515 and $545 million. For NAGLOZYME, we have tightened the range of full-year revenue to between $370 million and $400 million. The impact of COVID-19 coupled with the absence of Roktavian revenue contributions in 2020 result in an adjustment of total revenues for the full year 2020 to between $1.81 to $1.87 billion. Turning now to the peak AU brands, and starting with Palantik, we recognize Q3 revenues of $46 million, a 91% jump from Q3'19 revenues, the majority of which came from U.S. sales, as expected.

As a result of these dynamics playing out in our global territory's we have narrowed the range for Vimizim pull your guidance to between 515 and $545 million for Nag <unk> I'm, we have tightened the range a full year revenue do between 370 million and four.

$100 million.

The impact of COVID-19, coupled with the absence of rock tabby and revenue contributions in 2020 result in an adjustment of total revenues for the full year 2020, you between 1.812 $1.87 billion.

Turning now to the PKU brands and starting with Palin Z. We recognize Q3 revenues of $46 million and 91% jump from Q3 19 revenues the majority of which came from U S sales as expected.

We're pleased to report the patient disconnect.

Jeffrey Robert Ajer: We are pleased to report that patient discontinuation rates remain below the clinical trial experience. New patient starts from the United States have picked up in recent months as additional clinics resume operations and are able to more actively manage patients. COVID-19 has resulted in slowed patient uptake in Germany and delayed entry into other European countries.

Asian discontinuation rates remain below the clinical trial experience new patient starts from the United States have picked up in recent months as additional clinics resume operations and are able to more actively manage patients.

COVID-19 has resulted in slowed patient I've taken Germany and delayed entry into other European countries. We have established revenue in the middle East with a small number of patients starting treatment.

Jeffrey Robert Ajer: We have established revenue in the Middle East with a small number of patients starting treatment. Given the COVID-19 situation, we are pleased with the continued trend of quarter-to-quarter growth of Palanzeek revenue. On October 7th, an SBLA for Palanzec was approved in the U.S. This SBLA allows dosing up to 60 mg daily and adds long-term safety and efficacy data to the label. We view this as a significant positive development in the US. 19% of patients in the clinical trial were using the 60 milligram dose.

Given the COVID-19 situation. We are pleased with the continued trend of quarter to quarter growth apology revenue.

On October 7th and S. B L. A for Palin Zeke was approved in the U S.

This is b L. A allows dosing up to 60 milligrams daily and added longterm safety and efficacy data of the label.

We view this as a significant positive development in the U S 19% of patients in the clinical trial, we're using the 60 milligram dose. So the S. B L. A is expected to impact a meaningful portion of patients in the commercial setting that will benefit from being able to access is higher dose beginning in queue for.

Jeffrey Robert Ajer: So the SBLA is expected to impact a meaningful portion of patients in the commercial setting that will benefit from being able to access this higher dose, beginning in Q4. We reaffirm full year revenue guidance previously provided for Palanzec of between $160 to $190 million. Shifting now to QBAN, global revenues in the third quarter totaled $124 million, representing a 3% increase year over year.

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We reaffirm full year revenue guidance previously provided for Pellens ache between $160 million to $190 million.

Shifting now to Kuban global revenues in the third quarter of totaled $124 million, representing a 3% increase year over year.

After 13 years of exclusivity in the U S. We are heading now into the first quarter with two generic competitors and a plan for material share loss as a result.

Jeffrey Robert Ajer: After 13 years of exclusivity in the U.S., we are heading now into the first quarter with two generic competitors and a plan for material share loss as a result. We are expecting a step down in QBAN revenue in the United States beginning in Q4, which was built into the full year revenue guidance of between $430 million to $480 million previously provided and which we now reaffirm. Kuban has played a crucially important role, enabling Biomarin to be the leader in developing and marketing pharmacologic therapies for PKU, and Kuban remains an important brand in markets outside of the United States, for which we have exclusive rights until late 2024. We are confident in the potential of Palanzec as the next generation treatment for adults and expect that the value of the commercial PKU franchise will continue to rise overall beyond the near-term loss of Q Our commitment to the PKU community, in short, has never been stronger. Finally, an update on Bernoura. Net product revenues were $25 million in Q3, representing 28% growth compared to Q3 of 2019.

We are expecting a step down of Kuban revenue in the United States beginning in queue for which was built into the full year revenue guidance of between 430 million to $480 million.

<unk> previously provided and which we know reaffirm.

Subban has played a crucially important role, enabling biomarin to be the leader in developing and marketing pharmacologic therapies for P. K U and Kuban remains an important brand in markets outside of the United States for which we have exclusive rights until late 20th 24.

We are confident and the potential of pounds Z as the next generation treatment for adults and expect that the value of the commercial PK franchise will continue to rise overall beyond the near term loss of Kuban revenue.

Our commitment to the P. K U community insured has never been stronger bye.

Jeffrey Robert Ajer: The strong performance was driven by relatively undeterred patient uptake in all four regions, and patient compliance has remained high and stable despite COVID-19, reinforcing the urgency of unmet medical need for CLN2 patients and the importance of access to this treatment for improved long-term outcomes. Demand for Bernoulli continues to rise modestly each quarter, and we expect this trend to continue. We are tightening the range of full-year Brenner revenues to between $90 million and $110 million.

Henry J. Fuchs: The filings for Visortide in the U.S. and EU mean that this product could potentially be launched late in 2021. We are excited about the size of the market opportunity, have been working steadily to prepare this program for an eventual launch, and I look forward to providing updates on launch readiness as we get closer to a potential approval next year. In conclusion, in what continues to be a challenging and dynamic global situation, I'm very pleased with the commercial team's execution and performance in the third quarter of 2020 in all four regions. Our ability to react with agility early in the pandemic and put in place solutions to minimize the impact of misinfusions has resulted in relative stability so far, and we are confident in our ability to achieve full-year revenue guidance of more than $1.8 billion. Thank you for your attention, and I will now turn the call over to Hank to provide an R&D update.

Henry J. Fuchs: Thanks, Jeff. J.J. said it's been an eventful third quarter for Biomarin, but many of the challenges faced by our R&D organization are part and parcel of the drug development process, augmented by the innovative nature of what we do. Never a straight line.

Many of the challenges faced by our R&D organization are part and parcel of the drug development process.

Minted by the innovative nature of what we do never a straight line.

Henry J. Fuchs: With Roctavian, we did not expect a complete response letter in August, but we have refocused on productive next steps toward approval. We remain encouraged and committed to Roctavian based on the continued support from our investigators who report the life-changing potential of Roctavian. And with the complete one-year Phase III data from our 134-subject study, the next inflection point in the program, we, like you, excitedly await those top-line results in early 2021. In the meantime, we continue to make progress in our discussions with health authorities about what it takes to register Roctavian.

With rock Cavium, we did not expect the complete response letter in August, but we have refocused on productive next steps toward approval, we remain encouraged and committed to Octavian based on the continued support from our investigators who report the life changing potential of Octavian.

And with the complete one year phase three data from our 134 subject study. The next inflection point in the payload program. We like you excitedly away those topline results in early 2021.

In the meantime, we continue to make progress in our discussions with health authorities and understanding what it takes to register direct Cavium.

Henry J. Fuchs: Based on the powering of the Phase III study, we're confident in hitting the annualized bleeding rate at end point at one year, but we are also mindful of other critical measures, such as factor VIII levels, that will be important to determine success beyond the annualized bleed rate. Assuming the Phase 3 data are supportive, we have a framework for potential timelines subject to ongoing feedback from health authorities. As outlined in August, FDA has indicated a preference for two years of data, resulting in a potential late-22 launch under that scenario. It is important to note that the completion of the one-year Phase 3 study later this month, at the end of November, and some Phase 3 study participants will have been in this study for up to 18 months and others still up to two years. Returning to Europe, the EMA recently requested the complete one-year results from the full Phase III study cohort of 134 subjects to inform their benefit-risk assessment. As a result, we withdrew our application for an accelerated assessment yesterday and now plan to resubmit the marketing authorization application filing with complete one-year results in the second quarter of 2021.

Based on the powering of the Phase three study we are confident in hitting the annualized bleeding rate.

At endpoint at one year, but we are also mindful of other critical measures such as factory levels that will be important to determine success beyond the annualized bleed rate.

Assuming the phase three data are supportive we have a framework for potential timelines subject to ongoing feedback and health authorities as outlined in August FDA has indicated a preference for two years of data, resulting in a potential late 22 launch under that scenario it.

It is important to note that the completion of the one your phase three study later this month and the end of November and some state. Some phase three study participants will have been in this study for up to 18 months and other still up to two years.

Turning to Europe, TDMA recently requested the complete one year results from the full phase three study cohort of 134 subjects to inform their benefit risk assessment.

Henry J. Fuchs: Assuming resubmission in 2Q21, we would anticipate potential approval in early 22 should data be supported. We look forward to seeing and sharing our primary Roctavian findings in Phase 3 in early January and anticipate presenting those results at a scientific venue in 2021. Turning now to sorotide for the treatment of achondroplasia, we are pleased that our new drug application and marketing authorization applications are under review in the United States and in Europe, respectively. Earlier in the week, we were encouraged to have received the Food and Drug Administration's acceptance of our NDA under standard review with a PDUFA action date of August 20, 2021. As we stated in Monday's press release, the FDA reiterated a position raised during the 2018 Advisory Committee recommending two-year placebo-controlled trials in different age groups.

Henry J. Fuchs: And I'd like to share our perspective regarding that comment. Since the start of the program was authorized nearly 10 years ago, we took a collaborative approach with participating investigators whose shared goal was to generate maximum safety and efficacy data while balancing the feasibility challenges associated with exposing young chelatechondroplasia patients to placebo for a prolonged period of time, given the importance of early intervention, the limited window of opportunity, and the lack of revers

On gross offers are really producing reliable method to assess whether bus or tide has indeed had a durable impact and the rate of into contrail bone growth that ultimately increases spinal adult height.

We have endeavored to address the concern for potential acute attenuation of treatment effect in the short term clinical data package is designed to evaluate the short and long term effects of this were tied any contemplation incorporating randomized placebo controlled trials long term follow up studies and extensive real world evidence collection with multiple analytic approaches to confirm the magna.

Henry J. Fuchs: Since then, we have built a robust dossier of data that emphasizes the role of biology, biomarkers, and longer-term clinical data, which we believe supports a strong package on all fronts. In addition, data from the Phase II study program, with now up to five years of long-term follow-up data, as compared to the natural history study data on growth, offers a rigorous and reliable method to assess whether basauritide has indeed had a durable impact on the rate of endochondral bone growth that ultimately increases spinal adult height. We've endeavored to address the concern for potential attenuation of the treatment effect. The sorotide clinical data package is designed to evaluate the short and long-term effects of sorotide in achondroplasia, incorporating randomized placebo-controlled trials, long-term follow-up studies, and extensive real-world evidence collection with multiple analytic approaches to confirm the magnitude and durability of the effects of sorotide, including its effect on final adult height.

Student dirt durability of effect of assorted including its effect on final adults. The FDA has a long history of exercising scientific judgment and reaching its conclusions on the statutory requirement for demonstrating substantial evidence in the approval of orphan drugs and were cautiously therefore optimistic about what lies ahead as the review proceeds.

Henry J. Fuchs: The FDA has a long history of exercising scientific judgment in reaching its conclusions on the statutory requirement for demonstrating substantial evidence in the approval of orphan drugs, and we're cautiously, therefore, optimistic about what lies ahead as the review proceeds. Needless to say, the interest and enthusiasm from families participating in our program and seeking treatment for their children with achondroplasia has been our focus. We want to thank everyone who has participated in Biomarin's development program to date, and we look forward to keeping you apprised of our progress. We're also pleased with the progress being made to identify other serious genetic causes of skeletal dysplasia in order to explore how basaltite may transform the lives of those who are also more severely affected.

Henry J. Fuchs: We expect in the next few days to share news of an investigator-initiated Phase 2 study with a sorotide and selected genetic forms of short stature representing potential new indications. Suffice it to say, we are happy to play a role in the ongoing research to understand the role of the CNPU pathway and other conditions affecting genetic forms of short stature. Stay tuned for more details in a few days. Meanwhile, on BMN-307, our investigational gene therapy for phenylketonuria, we are pleased to announce that dosing has indeed begun in the third quarter, and we have now treated two adults with phenylketonuria in this study. With the 307 study now underway, we continue to advance the next products in our earlier stage pipeline. Last quarter, we began IND-enabling studies with BMN-331 gene therapy for the treatment of hereditary angioedema, which should benefit from an even more efficient development program based on our increasing experience.

Financial update I would also like to acknowledge our colleagues across the organization for their continued commitment and contributions during these challenging but no less busy times, it's been gratifying to see the focus and the flexibility put forth across biomarin in the interest of getting unimportant medicines to the people who need done as expeditiously as possible. During these times I Wanna. Thank you all.

And congratulate you all for your progress now I'd like to turn the call over to Brian R. C. F L take it away Brian.

Thank you Hey, please refer to today's press release summarizing our financial results for full details on the third quarter of 2020.

As usual or comprehensive report on the quarter will be available in our upcoming form 10-Q, which were planning to file tomorrow.

Starting with revenue and just noted we have updated our 2020 product line revenue guidance for the continued Impactive COVID-19.

Henry J. Fuchs: In addition, we also announced last quarter our potential collaboration and license agreement with Dynacor, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathy. These are large opportunities for Biomarin, given the tremendous unmet need in both indications, where we can benefit from our growing expertise in gene therapy development and manufacturing. Before turning the call over to Brian for the financial update, I would also like to acknowledge our colleagues across the organization for their continued commitment and contributions during these challenging but no less busy times. It's been gratifying to see the focus and the flexibility put forth across Biomarin in the interest of getting our important medicines to the people who need them as expeditiously as possible during these times. I want to thank you all and congratulate you all on your progress. Now, I'd like to turn the call over to Brian, our CFO. Take it away, Brian.

Brian R. Mueller: Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the third quarter of 2020. As usual, our comprehensive report on the quarter will be available in our upcoming Form 10-Q, which we're planning to file tomorrow. Starting with revenues, as Jeff noted, we have updated our 2020 product line revenue guidance for the continued impact of COVID-19. Also, I want to remind you that although we did not give specific Roktavian revenue guidance for 2020, we were planning to launch the product during the third quarter. Therefore, most of the adjustments to our total 2020 revenue guidance, of which the midpoint was lowered by $60 million, were due to Roktavian.

Merely to the continued global launch a pound the commercial preparations for the launch of Brac Cavium.

Due in part to cost containment measures. During these uncertain times, we expect that both R&D and SGN expense for the full year 2020 to be lower than previous estimate and we will provide guidance for both of those line items to ranges that are below prior guidance.

With respect to bottom line result, the expected large tax benefit that we discussed over the course of this year was recorded during the third quarter of 2020 totaling $835 million and drove a significant level of GAAP net income in the quarter and year to date 2020 as a reminder, the tax benefit relates to a transfer of certain.

Brian R. Mueller: Back to COVID-19, the prolonged pandemic and specifically slower new patient starts during 2020 may continue into 2021 and restrict revenue growth of our base. As a result, with the other headwinds we are experiencing through the delay in Roktavien approval and the previously anticipated impact of U.S. Kuvan generic competition, we expect our total revenues in 2021 to be roughly flat compared to our revenues in 2020. Moving on to operating expenses, one unique expense item driven by the unanticipated Roctavian approval delay is an $81 million charge to cost of sales during Q3 2020 related to Roctavian pre-launch inventory reserves. However, cost of sales and gross margins beyond those reserves were in line with expectations. R&D expense for the third quarter was $147 million, representing our development of Pessora Tide, Roktavian, and our early-stage pipeline. Q3 2020 R&D expense was lower compared to Q3 2019 due primarily to lower preclinical manufacturing of BMN 307 and lower Octavian R&D given its late stage of development. SG&A expense for the third quarter of 2020 was $179 million, which was slightly higher than SG&A expense for the third quarter of 2019.

2023.

Brian R. Mueller: Due primarily to the continued global launch of Palantir and commercial preparations for the launch of Raktiv. Additionally, due in part to cost containment measures during these uncertain times, we expect that both R&D and SG&A expense for the full year 2020 will be lower than previous estimates, and we have provided guidance for both of those line items to ranges that are below prior guidance. Regarding bottom-line results, the expected large tax benefit that we've discussed over the course of this year was recorded during the third quarter of 2020, totaling $835 million, and drove a significant level of gap net income in the quarter and year-to-date 2020.

For your data and will be F day, except to your data from a partial groups. Thank you.

Brian R. Mueller: As a reminder, the tax benefit relates to a transfer of certain intellectual property rights between Biomarin entities, similar to transactions reported by some of our larger peer firms. We have adjusted our full year GAAP Net Income Guidance for the Tax Benefit and the Roktavian Inventory Reserves to between $760 and $820 million, which also reflects a tighter range than provided for in August because the specific amount of the tax benefit is now. With respect to non-GAAP income, our lower operating expenses more than offset our lower revenue expectations and was the primary driver for updating full year 2020 non-GAAP income guidance to $280 Although we regularly use both GAAP and non-GAAP results and expectations internally to assess our operating performance, we believe that providing non-GAAP income enhances the ability to meaningfully compare the company's results from period to period and to identify operating trends in our principal business.

Hi, shelving.

Kind of complex question.

You know I don't I don't know that week predict what the results of the face three study will be 52 weeks or 104 weeks or thereafter, given that we started a little bit lower in the phase three than we did in the face went to or so it seems and I think it's.

Add variability that makes it difficult to answer the second part of your question, which is what do they really want other than more patients followed longer and so whether the cut that we take at the beginning of the year for the Europeans will contain enough information.

<unk> to get them to the place where they see where they've seen enough duration. You know, it's gonna be discussed with the agency and and we're not in a position to predict the outcome of those discussions because they also they await the data so that I think the data is gonna drive our next conversations with agency.

Fees and we look forward to the day to come in and because we think that the gene expression profile for the material that we put into the clinic is gonna be very strong data.

Okay.

How much. Your next question comes from the line of Great customer some G D more than your line is open.

Brian R. Mueller: As we look ahead to 2021, although we will not provide specific 2021 financial guidance until our typical Q4 earnings call early next year, we do note that while we expect to continue to earn substantial non-GAAP income and positive operating cash flows in 2021, we do expect that we'll revert to a GAAP net loss. Despite the impact of COVID-19 and the Roctavian delay on our near-term financial outlook, we believe that if we're able to launch Roctavian Invisoratide, revenue and profitability growth will resume during 2022 and accelerate in 2023. Lastly, with respect to cash and investment, we ended the third quarter of 2020 with $1.78 billion of total cash and investment, which reflects positive operating cash flows of $86 million and $99 million for the third quarter and nine months ending at the end of September 2020, respectively. And, as expected, we repaid the $375 million of convertible notes that matured subsequent to Q3 in mid-October.

Hey, good afternoon, guys hang another one for you on the sore tied this time I. Appreciate the explanation you had with your view and the F. D. A desire for two year data. So I guess I'm wondering given some of the the apparent differences of opinion at least at this stage between you and and the agency do you see it as a positive or negative that they're not.

Planning to convene an advisory panel at this point.

Thanks, Corie I I don't know how to interpret it either way.

You know some had told us that they wish there as an advisory committee on on Octavian.

You know and I think that there are other forces that are causing the agency to call advisor committees and not call Advisory committees.

Yeah, I'd say the good news in this regard is this division has a history of regulating products.

Now we have to make sure people understand that.

Hi, this is not gross one on but they do understand stature and how it relates to things like final at all tight and functionality and longterm alleviation of morbidity and and and and the particular you know discussion topic is not a new one that's been around for a lotta years and so.

I think we're encourage that we have a good plan to address it and and that's evidenced by the fact that they've accepted to file for review.

Brian R. Mueller: In closing, despite the challenges faced in the third quarter due to the impact of COVID-19 and the delay in rock caving, our base business remains solid, paving the way for substantial non-gap income and operating cash flows in 2020. Thank you for your support, and we will now open the call to your questions. Operator? Operator?

Okay. Thanks appreciate it.

Mhm.

Your next question comes from the line of rubbing kind of Blue Sky I'm curious 90 joking.

Hi, Thanks for taking the question Wow, what alive like confusing FDA information this year.

And I just had a couple of questions there iceberg.

Yeah. So I think on the last call you said that you would meet with you guys are you gonna not meet with them to figure out if you can narrow the package until you get the one your data. That's my first question and then I'm under the store tied just why would they put the language in there I I just give me it felt a little strange you have a sense of you know why.

Operator: Yes, thank you, sir. Ladies and gentlemen, to ask a question via the telephone, you may press star 1, and if you would like to withdraw your question, please press the pound key. Your first question comes from the line of Salveen Richter from Goldman Sachs. Your line is open. Good afternoon.

They reminded you they want to your data on or whether or not there's a method in place whereby you can sort of add additional give them additional data as it comes forth.

Henry J. Fuchs: On Roktavian, what provides you confidence that the one-year Phase III expression data or ABR levels will not differ much from the Phase I-II study? And also, what do regulatory agencies really want to see from the one-year data? And will the FDA accept two-year data? And will the FDA accept two-year data from a partial group? Thank you. Hi, Salveen. Complex question.

Help me understand there's two things you can think.

Henry J. Fuchs: I don't know that we can predict what the results of the Phase 3 study will be at 52 weeks or 104 weeks or thereafter, given that we started a little bit lower in the Phase 3 than we did in the Phase 1-2, or so it seems. And so whether the cut that we take at the beginning of the year for the Europeans will contain enough information to get them to the place where they've seen enough duration is going to be discussed with the agency, and we're not in a position to predict the outcome of those discussions because they also await data. So I think the data is going to drive our next conversations with agencies, and we look forward to the data coming in because we think that the gene expression profile for the material that we put into the clinic is going to be very strong data. Thank you so much. Your next question comes from the line of Corey Kassimov from J.P. Morgan. Your line is open.

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

Henry J. Fuchs: I don't know how to interpret it either way. You know, some people had told us that they wish there had been an advisory committee on Roktavian. You know, and I think that there are other forces that are causing the agency to call advisory committees and not call advisory committees. I'd say the good news in this regard is that this division has a history of regulating products. Now, we have to make sure people understand that serotonin is not a growth hormone, but they do understand Thatcher and how it relates to things like final adult height and functionality and long-term alleviation of morbidity. And this particular discussion topic is not a new one. It's been around for a lot of years.

For for 12 months, where they're followed him just sort of tied for 12 months at the end of that 12 months. They everybody gets put on the sore tired and continues so in point of fact will have 24 months of treatment data coming up in the next few months and we'll have the retrospective data on height change in there.

Running period, which is greater than six months, plus the 12 months, a placebo and and it could be that the agency simply signaling that that is additionally, important to them and again remember that the underlying issue. The itch to scratches is that and that's why I said that they have experience regulating growth products.

Henry J. Fuchs: And so I think we're encouraged that we have a good plan to address it. And that's evidenced by the fact that they've accepted the file for review. Okay, thanks. I appreciate it.

Henry J. Fuchs: Your next question comes from the line of Robyn Karnauskas from Truist. Your line is open. Hi, thanks for taking the question. Wow, what a lot of confusing FDA information this year has been. I just have a couple questions there. I guess first, you know, so I think on the last call, you said that you would meet with the agency. Are you going to meet with them to figure out if you can narrow the package until you get the one-year data? That's my first question. And then on the storage side, just why would they put the language in there? It just, to me, it felt a little strange.

Henry J. Fuchs: Do you have a sense of, you know, why they reminded you they want your data? Or whether or not there's a method in place whereby you can sort of add additional, give them additional data as it comes forth? Help me understand those two things, if you can.

Henry J. Fuchs: Thanks. So, concretely, yes, we have met with the FDA on Galrox and the substance of the meeting. The conversations are productive, and the discussions are progressing, and I think the big picture conclusion remains the same, which is that they want more longer data, and we'll look at the data in January and see how close we come with them to assuaging their concerns. And on Vasorotide, you know, why did they reiterate the two-year request? You know, who knows, really, and we'll get into discussion with them about that, but, you know, it seems consistent with what they've said on the advisory committee. Now, to remind you, I think we started enrollment in the phase three trial at the end of 2016.

Henry J. Fuchs: The advisory committee was in 2018, so we were pretty far down the road when the subject was kind of put to the advisory committee for discussion, and the advisors, you know, had a lot of important information and consideration, which, for the most part, resonated with the plan of our program, and I think that my own personal opinion is that they just reiterated it because it was a subject of discussion at the advisory committee. Now, you know, we continue to follow the patients in the study, and to remind you, what's interesting about the study that we did is that the study has a run-in period before patients are randomized. One-to-one, they receive Vasorotide or placebo, and then they're followed on placebo for 12 months, or they're followed on Vasorotide for 12 months.

Interventions and ultimately these interventions will convey the benefit that we think they do if they do that then.

These drugs will be approved.

Flat.

Yes.

Henry J. Fuchs: At the end of that 12 months, everybody gets put on Vasorotide and continues. So, in point of fact, we'll have 24 months of treatment data coming up in the next few months, and we'll have retrospective data on height change during the run-in period, which is greater than six months, plus the 12 months of placebo, and it could be that the agency is simply signaling that that is additionally important to them. And again, remember that the underlying issue, the itch that scratches is that, and that's why I said that they have experience regulating growth products, which is to the good because they understand many of the scientific considerations. But they are a little bit biased about the story of growth hormone, which is that growth hormone stimulates an initial growth response but then attenuates.

A lot of rotating in Missouri types, I would say the need for a significant business still I mean business development deal in the short term, we don't consider it to be very acute we are seeing in the past that actually an hour before we go to CRM productivity that as rooted in Missouri type would be large study will start generating some.

Significant cash flow and will allow us then to be maybe a bit more aggressive in the space, but we already have a pretty we have an expanding pipeline as there is there is more to come there.

And so we don't believe that a large business development deal in the short term.

Is necessary now obviously if.

Henry J. Fuchs: So they're very sensitive to durability of effect, and this was discussed at the advisory committee quite substantially, and we believe we have put together a great package of preclinical data, biomarker data, and, as I said, the long-term phase two data. So we think we can get there with the package that we've got, and we look forward to interacting with the agency and kind of laying their concerns to rest. Follow-up: you mentioned you met with the agency. Is there a bar they want for variability? Do they give you an exact number, or just let's wait and see, and then we'll chat again?

Okay.

Six to nine months from now.

Becomes EBITDA and either okay, we won't be approved our part will be even more delayed or or was very tight.

Henry J. Fuchs: Robyn, there was a part of your question that I wanted to address that you just reminded me about, which is hard to understand across the board. I think what we see across the board is that the agency has gotten very conservative in the last several months. They've had progressive leadership in the past, and oftentimes when the progressive leadership disappears, they retreat to conservative. And when stressed, they become conservative.

Henry J. Fuchs: And I think now is a stressful time because of the pandemic and the leadership changes. And so you see that. Now, the good news is that we have good studies in the book, and we have really good interventions. And ultimately, these interventions will convey the benefit that we think that they do. If they do that, then these drugs will be approved. Thanks a lot.

JJ Piename: Your next question comes from the line of Chris Raymond from Piper Chandler. Your line is open. Hey, thanks. Maybe just a broader strategic question. So you guys, and maybe for JJ, you guys have kind of held things steady, more or less, on the business development front for a while now. And a few of your peers seem to be showing, you know, maybe a bit more propensity or maybe even urgency to do deals. I guess, JJ, just wondering if maybe you could talk in a broader context, you know, how you're approaching that side of the business now versus years past, or, you know, how you're viewing things in terms of that environment, and maybe more specific to Biomarin, the need.

Trans ammonite us in fact rate reductions.

In the interim analysis cohort and on that basis.

We.

Met the investigators where they were already heading which was to fortify their attention to the details of initiating stewards and.

JJ Piename: Thanks, yeah. Well, we have announced a few early-stage deals this year, like Dynacor, and there are more to come, and we will continue to do that. Uh, later stage deals are way more expensive and riskier, and also we do require, you know, then more resources in terms of financial resources, which obviously we... But we feel pretty confident in the approval of Roktevin and Rosoritye, so I would say the need for a significant business deal, I mean, business development deal in the short term, we don't consider it to be very acute. We have seen in the past that actually, and that was before we got a CRL for Roktevin, as Roktevin and Rosoriteye would be launched, they would start generating some significant cash flow and But we already have a pretty good business, we have an expanding pipeline, and there is more to come there. And so we don't believe that a large business development deal in the short term is necessary.

You said.

You need it for the F D S.

JJ Piename: Now, obviously, if in six to nine months from now it becomes evident that either Roktevin won't be approved, or Park will be even more delayed, or Rosoriteye, which is facing the same issue, which I doubt very much, by the way, then obviously that would be a different situation. We are not there at this time. Your next question comes from the line of Joseph Schwartz from SBB Learing. Your line is open.

Yeah. The city is driving by an ongoing data.

The state is governed by an ongoing get access plan and also by statistical analysis, playing one for Europe, and one for the United States and it's customary to have different statistical analysis, playing some different reasons. So the data is protected by the data access lane in terms of ongoing accessing the data and analyses are governed by physical plans one each for your opinion I'd stick.

<unk>.

Okay, great. Thank you.

Your next question comes from the line of might be all day I'm Patty. Please your nineties open.

Henry J. Fuchs: Hi, I'm Joori dialing in for Joe. Thanks for taking our question. My question is on Ractavian.

Henry J. Fuchs: I was wondering what you have done to ensure that physicians treating the two-thirds of the remaining patients can generate a buy-in for the more rigorous on-demand steroid regimen. And do you have any insight into whether they will actually abide by it? Do you have a medical monitoring group to give you confidence that they'll be doing it? Or are you going to find out when they unblind the data? Thanks for the question. Our medical group is all over this issue. But I have to say that as soon as the interim analysis results were available, investigators pretty quickly realized that they were on the more, let's defer the on-demand steroids, then let's jump on the on-demand steroids. And as we mined some of the additional data, and we've shared this data with you, we recognized that investigators were starting steroids later in the course of the transduction than in the earlier study.

Henry J. Fuchs: And we were also finding that there were breakthrough episodes of recurrent transaminitis and factor VIII reductions in the interim analysis cohort. And on that basis, we met the investigators where they were already heading, which was to reinforce their attention to the details of initiating steroids and the speed at which they tapered corticosteroids. And so the monitoring group has been very, very, very plugged into the investigators, reviewing with them as contemporaneously as possible the most recent ALT measurements and putting the investigators in a position to make quicker decisions about whether there was a need for corticosteroid therapy initiation or adjustment based on the evolving data. So that's another factor that, in addition to what we've observed so far in the Phase III study at the interim analysis And that's even with that more relaxed form of steroids.

How many patients have a two year data do you think you might have by say the midpoint of their review and have you taken any look at some of the long term data and done any of your own internal analyses first natural history to kind of get a sense of the corroborate the phase one too.

Yes, I think to how many patients is pretty straightforward because I think we on blind to that level.

The last question was out of the one year.

Henry J. Fuchs: And if tighter attention to the detail of steroids has a positive impact, it'll make our results in the 134 even better than what we've seen so far. So that's all the more reason why we look so much forward to the analysis in the early part of the year because it's going to provide data to address these questions. Okay, great. Thank you. And then, if I could just ask one more question, when you unblind your phase three data, which is expected in 1Q21, how does it affect your blinding and powering assumptions for your phase two data should you need them for the FDA? The study is governed by an ongoing data access plan and also by a statistical analysis plan, one for Europe and one for the United States, and it's customary to have different statistical analysis plans in different regions So the data is protected by a data access plan in terms of ongoing access to the data, and the analyses are governed by statistical plans, one each for Europe and the United States.

From Cowen and company your line is open.

Henry J. Fuchs: Okay, great. Thank you. Your next question comes from the line of Marty Oster from Credit Suisse. Your line is open.

Henry J. Fuchs: Hey all, thanks for taking the questions. I think most of them have been answered, but maybe a follow-up on Vasorotide. Hank, could you remind us what percentage of patients from Vasorotide Phase 3 went into open label? And then I guess you should be kind of getting that two-year data from the two years of exposure from the patients who rolled over that were on drugs the first year pretty soon. Is there a specific plan or specific request to furnish that two-year OLE data to the FDA? And if so, can you account for that within the current PDUFA date timeframe? Yes, we can accommodate that data request in the current PDUFA timeframe, and we're still in the early stages of the review and gathering some initial information requests from both Europe and the United States, and it wouldn't surprise me if the additional data were part of the consideration.

Henry J. Fuchs: And the proportion of paid...remind me of the first part of your question, Marty? I forgot the first part of your question. Yeah, just asking if you had data on the kind of proportion of patients who rolled over to the OLE, specifically the one-year leadership on drugs. Yeah, yeah, I think it was all but two.

Henry J. Fuchs: Excellent. Thanks. So it was a very, very, very high number. Your next question comes from the line of Paul Matteis from CFL. Your line is open. Call, if you are only present on mute, your line is now open.

Henry J. Fuchs: Oh, great. Sorry about that. Thanks so much for taking my question. Hey, just one more on the sore tied to that point. How many patients with two-year data do you think you might have by say the midpoint of the review? And have you taken any look at some of the long-term data and done any of your own internal analyses versus natural history to kind of just get a sense of what corroborates phase one and two? Yeah, I think the number of patients is pretty straightforward, because I think we unblinded, well, the last patient was out of the one year in November of 2019. So we'll have all 110 patients, if you remember, 110 patients with data, 110 patients will have had their two-year assessments this month. And so it's a pretty comprehensive data set, and it should be available in the course of the review. And how's it going?

Henry J. Fuchs: You know, we haven't actually, we don't take interim looks at the data on an ongoing basis. But, you know, based on the long-term data that we've seen and just, you know, what you hear from investigators, we're encouraged that the effect of the sore tight is not only going to be sustained, but sustained in a in a in a robust manner. That's it, that sounds like it.

Commercially when you look at that could impact your business I did I would've thought that logistics.

Henry J. Fuchs: I mean, that is based on our Phase II study, which has now been going for five years. [inaudible] Yeah, and Hank, it sounds like what you're saying is that the retention rate from one year to two years is very high. Oh, yeah, yeah. I think Marty asked a question earlier, but I forgot.

Henry J. Fuchs: I lost track of the question. But yeah, yeah, I think it was like all the two patients rolled over. So 95, 6% rollover. The compliance rate with the short type is fantastic, and the interest level is fantastic. Thank you. Your next question comes from the line of Phil Nadeau from Cowen & Company. Your line is open. Good afternoon, thanks for taking my question. Hank, another question on basaltite.

Henry J. Fuchs: In your prepared remarks, you mentioned that there were a number of analyses that you've done to show basaltite is going to impact adult height, but you didn't go into much detail. Could you discuss those maybe in a bit more detail? What data and analyses will you have to show the FDA to possibly change their minds? And I guess a related question: in the past, in orphan disorders, the FDA did like natural history and thought differences versus natural history were persuasive. Why isn't that the case here?

And also as a reminder, our routing and manufacturing facility in California as.

He has been.

Approved by the European integrations.

Oh, that's also kind of reassuring for obviously, the EBITDA different questions, but so far the question we got from the FDA.

In part on on on CMBS rotated our.

Pretty pretty straightforward and before we moved out of that Jeff is your answer your question on the growing 19 impact on our base business. Just one thing we'd like you mentioned also is that.

Back to Missouri type you have enough it was great that manufacturing.

Henry J. Fuchs: I might have missed the valency of your question. The second part of your question was why would the natural history be... I forget, which side you asked it from, unsupported or supported? So in the past, it's been supportive. It seems like the FDA, in requiring randomized control data, doesn't think it's supportive here. Why is that?

It doesn't look like we're going to be any respect you want to talk about that the right and the response that we get back from the M&A.

Henry J. Fuchs: Oh, no, I think, well, I think that natural history data is supportive, and I think that that's going to be a key issue of the review, to ensure that it's, you know, sufficiently supportive to address the issue of durability. And that was actually quite a bit of the focus of the pre-NDA meeting that we had with them to convey what we'd done. So in reference to the first half of your question, you know, some of the additional analysis we did was to verify that the natural history data set is representative of the population that was recruited both into the phase three trial and into the phase two trial. Into the prior phase two trial to document that the estimates of growth change over time were similar in the two populations to conduct both sorts of eligibility matching as well as patient level matching to look longitudinally at the data. But A, we have a lot of experience doing this and having drugs registered on the basis of natural history studies.

Henry J. Fuchs: And B, we've had a lot of interaction with the agency. And C, we think we have a really robust data set to address the questions of continued benefit. I think for all those reasons that we have a lot of confidence that the package will get there on its own. And as to whether, you know, the additional data is even required remains to be seen. Did I cover everything, Phil?

It is diligently I would say pursuing diagnoses and identification.

Henry J. Fuchs: Yeah, that was very helpful. Thank you. Hey guys, thanks for the question. Appreciate it. I just had a few.

For the same reasons right, they're reluctant to go to hospitals in some cases, the hospitals are closed or operating with limited capacity. They have other priorities. So the rate of new patient identification and patient start for our enzyme products has slowed down.

Jeffrey Robert Ajer: Commercially, when you look at how COVID impacts your business, I would have thought that logistics would have been figured out by now, but is it just that cases are accelerating and that's more of a disruption? Or are there still processes that have yet to be worked out to just ensure compliance? And then Hank, real quick, it seems like manufacturing is a major focus by FDA now on cell therapy. With Rakhtavian, can you talk about the extent of the interaction so far? And has that, the nature of those discussions changed over time, or is it just all about the phase 3 study? So maybe, actually, let's start with the second question. Greg Geyer, who's our new head of TOPS, can answer that question. Yeah, thank you.

And even for P. K you were clinics have identified patience in their clinic and so patient identification isn't really the issue they're limited capacity for operations has slowed down the rate new patient starts for <unk> and then the third piece of it is particularly in your.

Where the Hell systems have been really focused on addressing the pandemic our ability to gain market access in new countries has been slowed it hasn't stopped but it's been slowed and I expect that that trend will continue going forward for a little while so.

Greg Geyer: So, we obviously had a lot of discussion during the initial filing for the Valrock. We've had a lot of experience with questions, many, many questions during the inspection or during the review process. I think what's been good is that we have had a good dialogue.

Those three separate pieces. Thank you.

Thank you. Your next question comes from the lineup Kanan, Makiya I think I put down like at $90 HM.

Greg Geyer: We've had a bunch of discussions on comparability, and at the end of the day, the agency accepted that, and we've gotten no further CMC questions related to that. So right now, we're pretty confident in the CMC package that supports Rectavian.

Greg Geyer: And as Hank was saying earlier, the focus has primarily been on the clinical side. And also, as a reminder, you know, our Roktegoon manufacturing facility in California has been approved by the European Medical Agency. Oh, that's also kind of reassuring, and so on.

JJ Piename: So, I'm going to let you guys go ahead and ask questions. We'll see if the FDA could have different questions, but so far, the questions we got from the FDA on TMT's work, David, are pretty straightforward. And before we move, I'm going to have Jeff Ajer answer your question on the COVID-19 impact on our base business. But just one thing we'd like to mention also is that, Back to Vasorityte. You haven't asked, but Vasorityte Manufacturing, it doesn't look like we're going to need any inspection. Would you like to talk about that, Frank?

Greg Geyer: Yeah, in the response we got back from the EMA, they asked some specific questions, but what they did say on the inspection side is, due to the great outcome of the Octavian inspection of the same facility that we're going to be making Vasorityte in, they have waived the inspectional need for European Vasorityte applications. So, just some good news that we received yesterday. Okay, so Jeff... Yeah, with respect to COVID-19's impact on our base business, there are really three different factors, and I'll address each one very briefly.

Jeffrey Robert Ajer: The first is the impact of misinfusions. So the early shock of the COVID-19 situation closed down clinics and had patients concerned about going into hospitals to take infusions. They're reluctant to go to hospitals because, in some cases, hospitals are closed or operating with limited capacity. They have other priorities. So the rate of new patient identification and patient start for our enzyme products has slowed down. And even for PKU, where clinics have identified patients in their clinics, and so patient identification isn't really the issue, their limited capacity for operations has slowed down the rate of new patient starts for Palanzec. And then the third piece of it is, particularly in Europe, where the health systems have been really focused on addressing the pandemic, our ability to gain market access in new countries has been slowed. It hasn't stopped, but it's been slowed down, and I expect that that trend will continue going forward for a little while. So those are three separate pieces.

The revenues for Kuban, you know, our our biggest strategy frankly with with P. K U as one of offence. So we have a fabulous drug in <unk> that is approved and we've been promoting very actively in the United States promoting.

[noise] pellens each sentence lunch essentially at the expense of promoting Kuban in the United States fully 35% of our new patient starts on Palin Zeke.

Have come from Kuban transition. So these would be adult patients that are looking for a bigger effect size and the treatment of their P. K U.

Jeffrey Robert Ajer: Thank you. Thank you. Your next question comes from the line of Canon McKay from RBC Capital Markets. Your line is open. Hi guys, this is Vikram from Kennan. Thanks so much for taking our questions. I had one on KUVON.

So we expect that trend to continue of of Kuban transitions to Pellens ache, but it's really about penetrating the adult market with palin seek and looking forward to potentially a gene therapy and the portfolio over over a period of several years.

Jeffrey Robert Ajer: Going into Q4, what are your expectations given the two generics that are going to enter the market, and could you talk about your broader strategy, how you're going to protect the franchise and, broadly, on PKU, are you thinking about maybe defensing the franchise by pricing discounts or any other strategy? Thank you. I'll handle that one.

<unk>.

Thank you so much super helpful. Thank you.

Your next question comes from the line of <unk> for more research. Your line is open.

Gosh, if you're on your feet and you had your nineties now okay, alright, sorry about that so.

Jeffrey Robert Ajer: So, in a general sense, and as I mentioned in the prepared remarks, we have been planning for and expecting a step down in revenues for Kuban in the United States, but not other markets. So, you know, our focus on Kuban as a part of the overall PKU franchise in other markets continues. Inside the United States, as you know, there are limited tools to work with to blunt the impact of generic competitors.

<unk>.

This one's a bit on the nose, but can you walk us through the options at the F. D. A has at this point on before tied in kind of what per cent data current your mind. The first one is the drug approved in August as expected number two the F D.

Yeah, I need two years of follow up and you know maybe you submit that nonrandom I stayed out from your faith three at the end of this year and you can still have an approval maybe by 2021 or the third option, where the F. D. A makes you run an entirely new phase three study that's randomized for two years that could delay the car you know approval.

Jeffrey Robert Ajer: Now, we are fortunate that there are only two generic entries and that we are not, Kuban is not a retail pharmacy subject to, you know, easy and rapid substitution. So, we've been planning for a step down in revenues. It's been difficult, frankly, to model exactly what to expect because there are so few good analogs to look at to guide our expectations.

By three but you're like how likely is that kind of worst case scenario here and is there another option here that maybe we're not appreciating.

Jeffrey Robert Ajer: And we do have some mitigation tactics in place in the United States that we are working on, and you'll forgive me if on this call I don't go into the details as that could be a competitive intelligence issue for others that might be listening. Fully 35% of our new patient starts on Palanzik have come from QBAN transitions. So these would be adult patients that are looking for a bigger effect size in the treatment of their PKU. So we expect that trend to continue as QBAN transitions to Palanzik. Thank you so much.

And then maybe on the financial can you talk about and a scenario where it lets you save Iraq doesn't get approved how much of an EPS bird sorry fire him or an Israeli over the next few years, how much and your margins inflect on your ear can franchise and then how what do you think <unk> will be for the suicide as it started.

To get commercial sale, thanks, a lot.

[noise] sure do you want me to start the first.

Oh, no that'd be all shows online.

Yeah.

It's hard to catch the you know everybody's got a little different calibration around percentages. So I'm always a little reluctant to assign percentages, but I don't think we would have filed.

He thought there was a substantial likelihood that we wouldn't be approved to paducah date, either either with a data all by itself or with the supplemental data sets of the ongoing cuts you know what I think the agency is pretty careful about you know the timing of those sorts of things.

Henry J. Fuchs: Super helpful. Thank you. Your next question comes from the line of Akash Tewari from Wolf Research. Your line is open. Akash, if you are on mute, please unmute; your line is now open. Hey, sorry about that.

Henry J. Fuchs: So thanks for the question. This one's a bit on the nose, but can you walk us through the options that the FDA has at this point on Visorotide and kind of what percent likelihood they'd occur in your mind? The first one is the drug gets approved in August as expected. Number two, the FDA needs two years of follow-up, and, you know, maybe you submit that non-randomized data from your phase three at the end of this year, and you could still have an approval, maybe by 2021. Or the third option, where the FDA makes you run an entirely new phase three study that's randomized for two years that could delay the, you know, approval by three plus years. Like, how likely is that kind of worst-case scenario here?

A key difference here between rotate and just to put it in some perspective. This is that when we had the pre b L. A beating on my T V and we didn't know what the enrollment right for the rest of the face free trial was <unk> and actually it happened pretty quickly so that name for the data are right around the corner in this particular case the agency knows the timing of the availability of the phase three data for.

For the sore or tired and they accepted the application and I think that that can days that the package of data that we do have those are pretty strong package data.

The thing I think you'll left off in the description list of you know what if they're having trouble getting there on the basis of the of the one of your data and the two your longer term follow up in a randomized population. The two O. Six study is a different age population random I studied definitive and quaint and I think that will definitely factor in to consider.

Henry J. Fuchs: And is there another option here that maybe we're not appreciating? And then maybe on the financials, can you talk about in a scenario where, let's just say Valrock doesn't get approved, how much of an EPS road story Biomarin is really over the next few years? How much can your margins inflect on your ERT franchise? And then what do you think the margins will be for Visorotide as it starts to get commercial sales? Thanks a lot.

Gration about what's next because if you see a fair amount of benefit in the patients who are under five years old I think that'll carry a lot of weight as to informing Ah Ah Ah regulatory decision well before further data and the older population would be required.

I think that's the that's the the back stop the big Big definite back stuff.

Henry J. Fuchs: JJ, do you want me to start with the first photo? Let me comment on the three options outlined here. Yeah. You know, hard to catch that, you know, everybody's got a little different calibration around percentages, so I'm always a little reluctant to assign percentages, but I don't think we would have filed if we thought there was a substantial likelihood that we wouldn't be approved on the PDUFA date, either with the data all by itself or with the supplemental data sets of the ongoing cuts. You know, and I think the agency is pretty careful about, you know, the timing of those sorts of things. A key difference here between Roktavian and other drugs is that when we had the pre-BLA meeting for Roktavian, we didn't know what the enrollment rate for the rest of the Phase 3 trial was, and actually, it happened pretty quickly, so that means that the data are right around the corner.

I mean, it's all.

Brian you answer the question on the I guess, you had a christianly gross margin for Missouri.

Henry J. Fuchs: In this particular case, the agency knows the timing of the availability of the Phase 3 data, and they accepted the application, and I think that that shows that the package of data that we do have is a pretty strong package of data. The thing I think you left off in the description list of, you know, what if they're having trouble getting there on the basis of the one-year data and the two-year longer-term follow-up in the randomized population, the 206 study is a different age population randomized study, definitive endpoint, I think that will definitely factor into consideration about what's next, because if you see a fair amount of benefit in the patients who are I think that's the backstop, the big, definite backstop.

Our orphan disease products and the recombinant enzyme that make up our our ERP franchise that fade cost of goods sold will be lower and so we've got 22, 20% to 22% Cogs on the base business today, and we'd expect to as both block caving in bus or tied revenue start to.

Again in the mix that we see our.

Gross margins improved from their debt down into the team.

Although even on our base business I mean, maybe Greg can talk about is there an auto industry is going to actually reduce our cost of goods.

Yeah, Theres tremendous work underway to try to improve our cost of goods sold for all that you are tea brands.

As well as even optimize some of our.

Henry J. Fuchs: I mean, Brian, do you want to answer the question on the, I guess you had a question on the gross margin for Missouri? Yeah, I think it was the margin prospect if there was no rock caving approval based on the current base business. So, first of all, you know, we do plan to continue to have growth from the base business, which is supported by the infrastructure that we have in place today. And, you know, just a reminder that on the operations side, we believe that rock caving approval is a delay, and we were prepared to launch with an SG&A and medical infrastructure here in Q3. And since we believe it's a delay, it's important to retain that capability for when we do hope to get an approval so that we can have the same successful launch trajectory that we were prepared for just a few weeks ago. So, that's the rock caving story.

Hopefully soon launch brands in terms of next generation processes. So a lot of works going on to continue to improve our margins.

In spite of the dynamic nature of our business.

Next question.

Your next question comes from the line of Matt You haven't done from Morgan Stanley. Your line is open.

Hey, great. Good evening, Thanks for taking the question Hank I guess I want to follow up on on one of the things you've already discussed which is.

Which has been sort of tied in and sort of the comments around two year data I mean I wanted to follow up on one of the comments that you made which was that it sounds like you think you'll have.

At least I'll eat follow up for two years I mean, I guess my question is.

You know if the FDA have few for per to your data, let's just say that's how they come out what are the pieces of information that you would provide to them how close would that be to sort of you know fulfilling what they may ask for.

Yes so.

Henry J. Fuchs: And then, you asked if there was not a rock caving approval, you know, I'd point you then to the source type, which with the acceptance of the filings in both the U.S. and Europe and in August to set the date, we'll be hoping to launch the source type in the second half of next year. So, again, that same infrastructure will support margin growth with revenue growth, both from the base business and then, you know, either or or both rock caving and the source type. And then, the last thing I'd say, with respect to your question on the source type margin, what we've said is that we do believe that because rock caving and the source type are different compounds from our legacy base business of these ultra-rare orphan disease products and the recombinant enzymes that make up our ERT franchise, that base cost of goods sold will be lower. And so, you know, we've got 20 to 22% COGS on the base business today, and we'd expect as both rock caving and source type revenues start to get in the mix, we'd see our gross margins improve from there down into the teens.

Let me just remind you of the structure three a one.

Patients were run in on a baseline observational study for at least six months and it's set at least six months number that makes things a little fuzzy in terms of describing things you know some patients with nine months of running that is sufficient for the 12 months you run in data centers 18 months. So.

So at least six months of running data and then at that point patients are randomized to placebo or drug now you know the placebo effect in the trial is pretty small.

It's like 0.16 or something like that so for practical purposes. The run in data and the placebo data. We believe can be pooled because they're not meaningfully different from each other and right. There that creates 18 months of prospective clinical trial grade definitive measurement of height data.

So.

JJ Piename: Although, even on our base business, I mean, maybe Greg can talk about it, there are a lot of initiatives ongoing to actually reduce our cost of goods, too. There's tremendous work underway to try to improve our cost of goods sold for all of the ERT brands, as well as even optimize some of our... Hopefully, soon, we can launch plans in terms of next generation processes. So a lot of work's going on to continue to improve our margins in spite of the dynamic nature of. Next question. Your next question comes from the line of Matthew Harrison from Morgan Stanley. Your line is open. Hey Greg,

Henry J. Fuchs: Good evening. Thanks for taking the time to answer the question. Look, Hank, I guess I want to follow up on one of the things you've already discussed, which is... Which is with Sorotide and sort of the comments around two-year data. I mean, I wanted to follow up on one of the comments that you made, which was that it sounds like you think you'll have at least OLE follow-up for two years. I mean, I guess my question is, you know, if the FDA asks you for two-year data, let's just say that's how they come out, what are the pieces of information that you would provide to them, and how close would that be to sort of, you know, fulfilling what they may ask for? Yeah, so let me just, again, remind you of the structure of 301.

Henry J. Fuchs: Patients were run in based on an observational study for at least six months, and it's that at least six months number that makes things a little fuzzy in terms of describing things. Some patients will have had nine months of run-in data, some patients will have had 12 months of run-in data, some patients will have had 18 months, so at least six months of run-in data. And then, at that point, patients are randomized. [inaudible] Clinical trial grade definitive measurement of height data so at the time the patients cross over to the drug from their 12 month journey through placebo, they will have been observed for 18 or more months. And the patients who are on vasorotide will have been on vasorotide for at least 24 months at that time point.

Female you stay on the line is 13, there's not really a question about that the the the the the question of our scratching <unk> you know how much evidence. So we have that the effect is durable and as I said that that the pretest likelihood that was extraordinarily high and I think the supplemental data if it's even in part of the story is gonna be extremely strong and I don't think that there's.

I don't think there's very much risk that all of a sudden the shorts I'd start slowing down your to your three or four based on what we've seen so far.

Hopefully that gives you a good details on what we can think about looking at if it even comes up.

Your next question comes from the line is you know one of my main three nine is open.

Henry J. Fuchs: So you'll have 18 plus months of an untreated arm, all prospective, and 24 months on the vasorotide arm, all randomized between those two groups to inform the durability of impact. So you can imagine that what we can do is we can look at the first month's height gain, sorry, the first six months of height gain, the second six months of height gain, the third six months of height gain, and the fourth six months of height gain.

I had to take my questions is David off of Gina well one question on the rock avian again, so I'm wondering how likely you can overturn fta's to your fall a requirement estimate on when you're since it's 80% of the patients will likely reached to your follow up and when your date of release, Chris quarter next year and then it seems like other.

Competitive programs like Pfizer's gene therapy as those are required when do you follow up.

So I was wondering if you combine some color on the likelihood of overturning after your decision and how confident are you that.

Henry J. Fuchs: And we can compare that to the first six months of height gain untreated, the second six months, the third six months, and potentially even longer of height gain not stimulated by vasorotide. You know, given how well the natural history data that we collected represents what we observed in our placebo group and how well it reflects the expectation that there's declining growth velocity, we think that that combination of package that says, here's our natural history data that we've prospectively observed for more than 18 months, It goes with this very large natural history data set that's contemporaneous, United States-based population, multi-center, and it can be used as Now, remember, we're not pitching this as pivotal. This is supportive.

You can do that.

Well I don't know that I would use pfizer's strategy as a evidence of what's going to be required for the simple reason that we were putting out similar kinds of discussion points. Prior to are complete response letter and obviously that didn't turn out.

Be an accurate.

The the the agreement that the F D. A had with us changed and so I don't know how one could know that any future agreements Ah wouldn't change in particular, given the sample size, what they're talking about so I'll put that to the side you know, it's a difficult to answer those questions about how likely I think that there are not as many patients.

Henry J. Fuchs: Hopefully, that gives you good details on what we can think about looking at if it even comes up. Thank you. Your next question comes from the line of Gina Wang of Barclays. Your line is open. Hi there, thanks for taking my question. This is David on behalf of Gina. One question on the Roktavian again.

[noise] at the one year data analysis that will have two years of follow up data remember the interim analysis was recorded and may of of 19, and so as that population that's really the leading edge of the study at the two plus years Mark. So you know that that I think is what's underlying.

Henry J. Fuchs: So we're wondering how likely you can overturn FDA's two-year follow-up requirement and submit a one-year data release for the first quarter of next year. And then it seems like other competitive programs like Pfizer's gene therapy are also requiring one-year follow-up. So I'm wondering if you can provide some color on the likelihood of overturning FDA's decision and how confident you are that you can do that. Well, I don't know that I would use Pfizer's strategy as evidence of what's going to be required for the simple reason that we were putting out similar kinds of discussion points prior to our complete response letter, and obviously that didn't turn out to be accurate. The agreement that the FDA So I won't put that to the side. You know, it's difficult to answer these questions about how likely it is.

<unk> the agency's request for more information now you know I I don't know I mean, we could put up the numbers of patients that we have a 24 months a follow up 18 months a follow up in front of the agency and it's you know, it's all gonna depend on the strength of evidence and as I said before they they are they're reluctant and we and Tu Tu Tu Tu IDENT.

<unk>, an explicit specific and point because it really is a combination of consideration of of the a b R. The agency is really hooked on Avr's a primary endpoint of course, we think we're gonna be strong in that regard, but also their concerns for projecting the durability are pretty should get again and I think it bears reminding that they don't have a lot of experience and.

Space you know the cheaper our gene addition programs that they've approved.

Measure the gene product directly and so this is the first experience or having with this and I think it's unfortunately decided to ask for more sample size and longer well after the BLA had been submitted and without any warning, but that it is what it is and as I said before even during these conservative times the recipe.

Henry J. Fuchs: I think that there are not as many patients at the one-year data analysis that will have two years of follow-up data. Remember, the interim analysis was recorded in May 19, and so it's that population that's really the leading edge of the study at the two-plus years mark. So, you know, that I think is what's underlying the agency's request for more information. Now, you know, I don't know.

P. As always you know more data and more time and if you have a good drug more Dana more time things work out.

Henry J. Fuchs: I mean, we could put up the numbers of patients that we have at 24 months of follow-up in front of the agency, and it's, you know, it's all going to depend on the strength of evidence. And as I said before, they are reluctant, and we are, to identify an explicit specific endpoint because it really is a combination of considerations of the ABR. The agency is really hooked on ABR as a primary endpoint. Of course, we think we're going to be strong in that regard. But also, their concerns for projecting durability are pretty significant.

We think we have a good drugs.

Ah help thank you.

Your next that's been comes on the line off Mohit must have T V and 90 shopping.

Good evening. This is coupon from O'hare, thanks for choosing us and I just had a quick question a lot of them have been answered, but how should we think about your disclosures regarding the upcoming meeting with the F. D. A regarding power off would you be sharing with us when you meet with them and what was asked then sort of the topics that were discussed.

And then I guess separately to what extent do you think the availability of alternative therapies impacted the decision to go with the C. R. All thanks.

Henry J. Fuchs: And I think it bears reminding that they don't have a lot of experience in this space. For example, the two prior gene addition programs that they've approved, you don't measure the gene product directly. And so this is the first experience they're having with this, and I think it's unfortunate they've decided to ask for more sample size and longer, well after the BLA had been submitted and without any warning. But it is what it is.

Well like I said, we've had our type anything with the F. B I and we believe that we're making progress. The medium is well attended by the agency and they were quite engaged in verbal and they provided additional information, which really boils down again to wanting to directly observe the durability.

Henry J. Fuchs: And as I said before, even in these conservative times, the recipe is always, you know, more data and more time. And if you have a good drug, more data, more time, things work out. We think we have a good drug. How can you help?

Of the product using the phase three trial, rather than rely on the face one two material, it's really more of a of a different interpretation of the same set of facts than anything else in it and it's a new interpretation of the same set of facts disappointing and surprising but as I said, we have the definitive study.

Henry J. Fuchs: Thank you. Your next question comes from the line of Mohit Bansal of TV. Your line is open. Good evening, this is Keethan from Mohit Bansal TV.

Henry J. Fuchs: Thanks for squeezing us in. Just had a quick question, a lot of them have been answered, but how should we think about your disclosures regarding the upcoming meeting with the FDA regarding Valrox? Would you be sharing with us when you meet with them and what was asked and sort of the topics that were discussed?

Ongoing and as far as the impact of available alternative therapies I think there's been a lot of discussion about that but one has to remember always that you're measuring the potential for benefit above the current available standard of care now if the current condition is completely <unk>.

Henry J. Fuchs: And then, separately, to what extent do you think the availability of alternative therapies impacted the decision to go with the CRL? Thanks. Well, as I said, we've had our type A meeting with the FDA, and we believe that we're making progress. The meeting was well attended by the ANC, and they were quite engaged and verbal, and they provided additional information, which really boils down, again, to wanting to directly observe the durability of the product used in the Phase III trial rather than rely on the Phase I-II material. It's really more of a different interpretation of the same set of facts than anything else, and it's a new interpretation of the same set of facts, disappointing and surprising, but as I said, we have the definitive study ongoing.

Untreated then we think about placebos or natural history comparisons in our case. Our comparison is the best available standard of care number one on the W. H the world better Scream affiliates standard of care recommendation option is recounted decorate replacement therapy delivered prophylactic Lee and that's the control in our study and remind you we demonstrated that uhm compared to patient.

Who have quite substantial breakthrough bleeding on recounted decorate replacement therapy patients you've received the transferring and now in two separate studies have quite substantial reductions in their bleeding posts gene transfer and I think it's that kind of observation that will ultimately animate the uptake of of a treat them like rock television into the community.

As I said before once the agency feels more comfortable with a higher end in a longer duration of a follow up and exactly where that point is is we've talked about is not yet known but the you know the advantages over current therapy or improve bleeding without the without the requirement for.

Henry J. Fuchs: And as far as the impact of available alternative therapies is concerned, I think there's been a lot of discussion about that, but one has to remember always that you're measuring the potential for benefit above the current available standard of care. Now, if the current condition is completely uncared for, [inaudible] And one last thought again, we'll remind the audience here that the phase 3 trial, the one-year, you know, end point APR phase 3 trial, is designed as a superiority trial as compared to standard of care, not a non-inferiority, superiority trial, and this is what we anticipate demonstrating in January. Great, thanks. Your next question comes from the line of Trevor Alred from Oppenheimer. Your line is open. Hey, thanks for squeezing me in. Just two quick ones. One, you've been talking about Valrox. Do you get the sense that there's any read through for this for any gene therapies in the future? Or is this Valrox specific?

Interventions on an ongoing basis, namely prophylactic therapy and from the patients and clinicians that we've talked to this is this transformative.

Yeah, well we might.

The audience here that.

The fish free trial.

Here.

Charles is designer the superiority Charles.

Alright.

Fury superiority trial and this is what we.

There was fitting in January.

Great. Thanks.

Your next question comes from the line Octopi I'll read some Oppenheimer, you're 90 telephone.

Hey, Thanks for squeeze me in just two quick ones. One you you've been talking about of Iraq's do you get the sense that there's any.

Read through for this for any gene therapies in the future as this <unk> specific.

Henry J. Fuchs: And then two, you talked a little bit about Kuvan. I was wondering if you anticipate that generics might have any difficulty entering the market during this time. Thanks. You know, in the read-through, there certainly is a lot of stuff not going on at the Center for Biologics. I guess you can read into that whatever you want to read into it.

And then to you talked a little about Kuban I was wondering if you anticipate the generics might have any difficulty entering the market. During this time.

<unk>.

You know in the read through Uhm, there certainly is a lot of stuff not going on at the center for Biologics [laughter] I guess, you can read into that whatever you want to read a that.

Henry J. Fuchs: My reading of all of that is that things are pretty conservative right now at the agency. And I think, you know, Dr. Marks, who does a great job and is a very serious guy and a very high-caliber person, has talked about some of the communication challenges they've experienced and some of the leadership challenges that they have as they're trying to handle an enormous workload on the cell and gene therapy side, not to mention the Center is handling the COVID vaccine pandemic considerations. So they're massively overworked, and they're massively challenged, and they're trying to make sure that they are perceived to be trustworthy and conservative in their decisions on behalf of, you know, people's health. So I think that's the read-through to be had. You can get into some more scientific details about Roctavian as a product, as I mentioned.

My read of all of that is that things are pretty conservative right now at the agency and I think you know Dr remarks, He does a great job and is very serious guy and very high caliber person has talked about you know, but some of the communication challenges they've experienced then and some of the leadership challenges that they experiences are trying to handle an enormous workload on this.

Helen gene therapy side not to mention the center is handling the covid vaccine Camden <unk> considerations, so they're they're massively over worked and and they're massively challenged and you're trying to make sure that they're perceived to be trustworthy and conservative in their decisions on behalf of People's Health.

So I think that's the that's the read through to the had you can get into some more scientific details about rock payment is a product as I mentioned, it's the first product where you can measure the transferring effect by itself and I think whenever that happens that causes agencies to ask different new questions and when that's done in a conservative and stress environment. It just takes a while for the.

Henry J. Fuchs: It's the first product where you can measure the transgene effect by itself, and I think whenever that happens, that causes agencies to ask different new questions. And when that's done in a conservative and stressed environment, it just takes a while for the agency to get itself comfortable with the issues of consideration. And that's why we maintain confidence that we're going to get there. It's just going to require patience. Jeff, do you want to answer the impact of COVID-19 on dinner? So we're early in this, and we will be watching very closely, but we believe that the likely strategy of generics is one of traditional substitution at the pharmacy level, with the caveat that we're talking about specialty pharmacies here and not retail pharmacies.

You can say to get themselves comfortable with the issues of consideration and that's why we maintain confidence that we're gonna get there. It's just gonna require patience.

Yes, you answer the impact.

In fact, the COVID-19.

Happy too. So we're early in this in and we will be watching very closely but we believe that the the like likely strategy of the generics is one of our traditional substitution at a pharmacy level with the caveat.

That we're talking about specialty pharmacy is here and retail pharmacies. So if if the strategy is one of substitution at specialty pharmacy, I I don't see much of an impact one way or another of the COVID-19 pandemic.

Henry J. Fuchs: So if the strategy is one of substitution at specialty pharmacy, I don't see much of an impact one way or another of the COVID-19 pandemic. [inaudible] Thank you, speakers. I am showing no further questions at this time. I would like to turn the conference back to Mr. J.J. Bien-Aimé, Chairman and CEO. Sir, please go ahead.

If in fact, the strategy of the generics is something different perhaps like a branded generic where they're seeking to get a new patients diagnosed.

Uhm with their product then then I you know if they could be impacted substantially by the pandemic and we don't know for sure which strategy the generic.

JJ Piename: Thank you, Operator. So, in conclusion, with 2020 coming to a close, it is important to have perspective on what has been accomplished to this point, as well as the tremendous opportunities and light ahead for Biomarin. It's been an unpredictable year, but the underlying fundamentals of our business are strong, and we are resolute in our mission. Biomarin employees have demonstrated extraordinary resilience in the face of uncertainty for almost nine months now and counting, and we have confidence in our ability to navigate any new challenges that may arise as we forge ahead.

<unk> pursuing uhm, but but I think it's likely that they would be pursuing the first option of a classic substitution of the pharmacy. Thank you.

Thank you C K I I'm showing no further questions at this time I would like to turn the conference back to Mister G. G. P enemy cannot M. P O. Sir Please go ahead.

Thank you operator or so.

2020.

It is important to have perspective on what has been accomplished at this point.

As well as a tremendous opportunities in my head for awhile right.

I think I have all year, but.

Fundamentals of our business are strong and we originally.

Have demonstrated extraordinary resilience in the face of a certain date for almost nine last year accounting and we are confident in our ability to navigate any new charges that may arise as before that so thank you for your continued support will stay safe.

Operator: So, thank you for your continued support, and stay safe. Bye. Thank you, speakers. Ladies and gentlemen, this concludes today's conference call. Thank you all for joining me. You may now disconnect.

Back.

Thank you speaker, ladies and gentlemen, he concludes today's conference call. Thank you also joining give me now disconnect.

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