Q2 2020 PTC Therapeutics Inc Earnings Call

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Ladies and gentlemen, thank you tend to buy your conference calls have begun momentarily again, thank you to standing by your comp cost I think your momentarily. Thank you.

Operator: Ladies and gentlemen, thank you for standing by. Your conference call will begin momentarily. Again, thank you for standing by.

Operator: Your conference call will begin momentarily. Thank you. Ladies and gentlemen, thank you for standing by and welcome to the PTC Therapeutics 2nd Quarter 2020 Financial Results and Corporate Update Conference Call. At this time, all participants are on listen-only mode. I would now like to turn the conference over to your host, Mr. Alex Kane, Chair of Investor Relations at PTC.

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Unknown Attendee: Thank you. Good afternoon, and thank you for joining us to discuss the PTC Therapeutics second quarter 2020 corporate updates and financial results. Joining me on today's call is our Chief Executive Officer, Stuart Peltz, our Chief Financial Officer, Emily Hill, our Chief Development Officer, Matthew Klein, and our Chief Business Officer, Eric Pauwels. Before we start, let me remind you that today's call will include forward-looking statements based on current expectations. Please take a moment to review the slide posted on our investor relations website in conjunction with this call, which contains our forward-looking statements. Our actual results could materially differ from these forward-looking statements as any and every such risk can materially and adversely affect our business and results of operation. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report, Form 10-Q, and annual report, Form 10-K, filed with the Securities and Exchange Commission, as well as the company's other SEC filings. We will disclose certain non-GAAP information during this call; information regarding our use of GAAP and non-GAAP financial measures, and a reconciliation of GAAP to non-GAAP financial measures is available in today's earnings release.

Ladies and gentlemen, thank you for standing by welcome to the PTC Therapeutics second quarter 2020 financial result in corporate update conference call.

There's some all participants will listen only mode.

I would now trying to concentrate hostess Alice Kane Chief Investor Relations at PTC. Sir. Please go ahead.

Good afternoon, and thank you for joining also discussed with Peachy Therapeutics second quarter 2020, corporate updates in financial results. Joining me on today's call will get cheaper.

Well, our Chief Financial Officer, Humbly Hill, our Chief Development Officer, Matthew Klein, and our Chief business Officer Pals before we start let me remind you that today's call will include forward looking statements based on current expectation.

Take a moment to review the slides posted on our Investor Relations website in conjunction with the call which contains forward looking statements.

Actual results could materially differ from these forward looking statements.

Such which can materially and adversely affect our business in results of operation.

For a detailed description will take couple risks and uncertainties. We encourage you to review the company's most recent quarterly reports on form 10-Q, <unk> Annual report form 10-K filed with Securities and Exchange Commission as well as the company's other actually see filings you build the school in certain non-GAAP information. During this call information regarding our you should GAAP and non-GAAP financial measure.

Unknown Executive: With that said, let me pass the call over to our CEO, Stuart Peltz. Thanks, Alex. Uh, looking back at the second quarter, I'm proud. And FOSDA had an outstanding quarter with greater than 30% year-over-year growth. As you'll hear throughout the remainder of the call, despite the challenging environment due to the global pandemic, PTC has continued to execute in our commercial business and advance key platforms and programs in our pipeline. One of these key platforms is our validated small molecule splicing platform.

A reconciliation of GAAP to non-GAAP is available in today's earnings release.

With that let me pass the call over to our CEO Stuart Peltz still.

Oh.

Thanks, Alex.

Oh looking back at the second quarter I'm proud of continued strong commercial performance.

You shouldn't franchise with nearly 20% growth year over year, excluding Brazil.

Unknown Executive: As many of you know, the most advanced splicing program is RISDAPlan for the Treatment of Spinal Muscular Atrophy, or SMA, with a PDUFA date of August 24th, which is rapidly approaching. We believe that RISDA Plan, which would be the first and only oral treatment for SMA, has the potential to be the most competitive commercial product for this devastating rare disorder. RISDA's plan validates our splicing platform and represents how innovation can generate potential value for all our stakeholders. When we started the SMA Splicing Program, more than a decade ago, It was considered a luncheon.

In Florida had an outstanding quarter with greater than 30% year over year growth.

If you will hear throughout the remainder off the call.

Like the challenging environment local pandemic.

Pete do you have continued to execute in a commercial business.

Key platforms and programs in our pipeline.

One of these key platforms as our validated small molecule spikes in platform.

Many of you know the most likely program is the plan for the treatment of spinal muscular atrophy your estimate.

Good day on August 24, which is rapidly approaching we believe the original plan, which would be the first an older oral treatment for some nice.

Unknown Executive: The dominant belief at the time was that, well, splicing is a great target in principle because it is a mechanism that occurs with most all RNAs and cannot be selectively modulated. Because of our deep understanding and expertise in RNA biology, we knew that RNAs were actually the first end-goal that could form unique structures with catalytic function that could be targeted. We also knew that if successful, targeting RNA would lead to a new platform to discover compounds that could lead to multiple new treatments for patients living with rare dystorphia. It represents a new paradigm for drug use. Over the years, we've built a splicing technology that led to the discovery of... As we highlighted recently in our Splicing Platform Deep Dive, PTC has unique expertise in RNA biology. We have constructed an RNA-centric compound library.

The potential would be the most competitive commercial product for this devastating rare disorder.

Really the plan validate our splicing platform and represents how innovation can generate substantial value for a lot for all our stakeholders.

When we started the or something like two program over a decade ago.

Was considerably.

The dominant believes that the time was that what spikes and there's a great targeting principal it is a mechanism that occurs with both all arnie cannot be selectively modular with small molecule.

Because of our deep understanding that expertise in R&D biology, we knew that our names were actually before and thought that conformed unique structures with cabling function that could be targeted.

We also know that if successful targeting our name would lead to due to a new platform to discover call.

It could be to multiple new treatment option for patients living with rare disorders.

It represents a new paradigm for drug discovery.

Over the years, we don't displacing technology, the blood to the discovery of risk the plant into a novel platform.

Unknown Executive: Proprietary Screening Tool, A Proven Process to Optimize Compounds to Bring Them to the Clinic, And a Fully Integrated Global Commercial Infrastructure to Bring Rare Disorder Therapies to the Clinic. As you may have heard on our recent deep dive, this is a platform that has a number of additional targets moving forward. We anticipate three to five splicing developments. Over the next three to five, the next splicing compound to enter the clinic is PTC 518, our Development Candidate for 100 years, and it is expected to be a first in human trial later this year. In addition to Huntington's disease, there are exciting new splicing programs emerging from our deep pipeline, including SCOT3 and MAP.

Hi.

And our splicing platform die.

Did you see has unique expertise and our anybody else.

We're constructing an earnings centric compound library.

Probably a few screening tool.

A proven process to optimize the telephone bring them into the clinic.

Fully integrated global commercial infrastructure to bring rare disorder therapies for patients.

As you may have heard on our recently God. This is a platform that has a number of additional target moving forward.

We anticipate three to five flexi development candidates over the next three to five year.

The next spikes in compound to enter the clinic, it's PTC five one day, our development candidate for Huntington.

Which is expected to be first in human trial later this year.

Unknown Executive: We're also making progress in other platforms and programs in the second quarter. We strengthened our pipeline with equity. 16923 for PKU

In addition to Huntingtons disease are exciting new splicing programs emerging from our deep pipeline, including Scott three enough though.

Well, we're making progress and other platforms and programs in the second quarter, we strengthened our pipeline would be acquisition would be like two or three for became a year.

Unknown Executive: PTC 923 expands our platform capability with the addition of a late-stage program for inborn errors of metabolism. We believe it has the potential to be the best-in-class treatment for PKU patients. We also recently initiated a Phase 2-3 trial for PTC-299 for COVID-19. We recognize that PTC-299 has a unique dual mechanism that is potentially effective against both stages of the viral infection. We worked with leading academic collaborators and quickly confirmed PTC-299's antiviral activity against SARS-CoV-2 in vitro, moving rapidly into the clinic for COVID-19.

Did you see Nike free expands our platform capabilities through the addition of on late stage programs for inborn errors of metabolism.

I believe it has potential to be the best in class treatment for PK new patients.

We also recently initiated a phase three trial for PTC Tonight nine for corporate banking.

We recognize that could you see tonight not has a unique dual mechanism.

Actually affect up against both stages of the viral infection.

We worked with leading academic collaborators and quickly confirmed PTC Tonight that got viral activity against Sars Koby, two would be true moving rapidly into the clinic for corporate 90.

The clinical trials.

Unknown Executive: The clinical trial is being conducted in two states. We expect Stage 1 to be completed in the second half of 2020 and anticipate reporting top-line results for both stages in the first half of 2020. We have multiple sites in the United States, Brazil, Spain, and Australia, with additional countries expected to initiate in the coming months.

HM stage.

Spec stage, one to be completed in the second half what 2020 and anticipate reporting topline results for both stages in the first half of 2021.

We have multiple sites in the United States, Brazil, Spain, and Australia with additional countries expected to initiate the coming months.

Unknown Executive: Ultimately, a combination of the mechanism, the preclinical data, the well-established safety profiles, and the compound's oral bioavailability gives us great confidence in PTC 299's potential as a treatment for COVID-19. One of the reasons that we took PTC 299 forward is because the global pandemic requires us to work towards a solution. In addition to its obvious effect on the health of our individuals, COVID-19 has impacted many industries, including our own. At PTC, we took early and aggressive steps to mitigate potential risks to our operation. Importantly, despite the challenges presented by COVID-19, we have been able to execute on certain key programs and lessen the impact.

Okay minutely, it's a combination of mechanism the preclinical data the well established safety profile and the compounds oral bioavailability gives us great confidence did you see to 99 potential as a treatment for corporate 90.

One of the reasons that we took PTC to nine like Ford is because Coburn 19 global pandemic requires our industry to work towards low solution. In addition to its obvious effect on the health of our individual covert 19 has impacted many industries and couldn't be now wrong.

At PTC, we took early an aggressive steps to mitigate potential risks to our operations.

Importantly, despite the challenges presented by Copenhagen, Gee, we have been able to execute on certain key programs and lessen the impact than others. As I mentioned earlier, we expect to initiate a first in human phase one trial would be C. Fivea see branded from them.

Unknown Executive: As I mentioned earlier, we expect to initiate a first-in-human Phase 1 trial with PCC 518 for Huntington's disease later this year in our BioE platform. The two potential registrational trials for Vatiquinone, formerly known as PTC 743, remain on track to initiate later this year. We're particularly excited about these upcoming trials as they provide us with near-term opportunities to address two rare disorders with significant unmet medical needs. In addition, as we recently announced, we initiated the phase one study for PTC 857, a second compound from our BioE platform. Before I pass the call to...

He tends to be later this year.

Her body or when you platform.

Two potential Registrational trial.

For a particular known formerly known as PTC. Some for three remain on track to initiate later this year.

We're particularly excited about these upcoming trial as the actually provide us for near term opportunities to address two rare disorders with its significant unmet medical need.

In addition.

Recently announced we initiated a phase one study for PTC eight by sub.

Second call on from our body.

Platform has a schedule.

Before I pass the call over to the team I wanted to touch on recent developments translarna for nonsense mutation Duchenne muscular dystrophy.

Unknown Executive: I want to touch on recent developments with TransLarna for non-transmutation Duchenne muscular dystrophy, the first product we discovered, developed, and commercialized by PTC. Importantly, the EMA confirmed the risk benefit profile of TransLinux with the sixth annual renewal of the Conditional Approval, which is the basis of our sales outside the United States. The CHMP recently recommended revision to the transliner label, removing the statement that efficacy has not been demonstrated in non-ambulatory patients.

The first project, we discovered develop and commercialize like PTC.

Importantly, you make the from the risk benefit profile of trends learn though with the six annual renewal of the conditional approval, which is the basis of our sale.

Outside the United States, let's see some T. recently recommended.

Revision to the translation on label removing the statement efficacy has not been demonstrated in non ambulatory patients.

This change in Naples health care professionals to use their clinical just me treatment decisions for their patients on Translarna loss [laughter]. It also supports our discussion reimbursement authorities on continuing trend planetary.

Unknown Executive: This change enables healthcare professionals to use their clinical judgment to make Treatment Decisions for their Patients on Translino with Los Angeles. It also supports our discussion.

Matthew Klein: Reimbursement Authority. I'm continuing transliteracy for patients who become non-ambulant. We have pioneered therapies for Duchenne muscular dystrophy and remain highly, I'll now turn the call over to key updates on our clinical programs. Next. Thanks, Stu.

For patients become a neighborhood card.

We have pioneered there shouldn't muscular dystrophy remain highly committed to that can be.

I'll now turn the call open to match for key updates on our clinical program.

Yeah.

Thanks to start I want to build on Steve's comments regarding our teams response to the called ignites Nintendo.

Matthew Klein: To start, I want to build on Stu's comments regarding our team's response to the COVID-19 pandemic. Despite the many challenges, we have worked hard to mitigate the impact on ongoing and planned studies. Let me begin with Study 045, the U.S. Translong Distance Study. In developing the protocol for this study, we carefully considered every element of the design to minimize variability that could confound study results. This included the decision to use a single site for the study at UCLA, the method of muscle biopsy collection, and the method of biopsy sample analysis.

Despite the many challenges we've worked hard to mitigate the impact on ongoing and planned study.

Let me begin with study all four or five U.S. trends long I just different studies.

Helping the protocol for the study we carefully considered every element of this is Bob minimize variability that could can sound study results.

This included the decision to use a single site for the study it you see away.

Method of muscle biopsy collection, and the method a biopsy sample analysis.

Matthew Klein: I want to highlight that the protocol specifies that sample analyses will not occur until the end of the study. PTCs and study investigators will be blinded to the results until all analyses are completed. At this time, final study muscle biopsies have not yet been collected from eight remaining boys in the 045 study. Given the evolving COVID-19 landscape in Los Angeles and in other affected regions where study patients reside, we are continuously monitoring the situation to determine when it will be possible to safely obtain the final biopsy. We are also exploring all potential options in order to have a data readout by year's end. Of course, we have ensured that all subjects remain on Transmona until they are able to complete the final study visit. Turning to our BioE class.

That's a highlight that the political specifies that sample analyses will not occur until the end of the study.

See I study investigators will be blinded to the results until all analyses are complete.

At this time final study muscle biopsies I've, not getting collected eight revealing boys and equaled four or five state.

Given the evolving Kobin Nike landscape in Los Angeles, and other affected regions, where study patients for though we're continuously monitoring the situation determine when it will be possible safely obtained the final biopsies.

We're also exploring all potential options in order to have a data readout by years end of course, we even sure all subjects remain on track while until they're able to complete the final study this.

Turning to our buyer platform.

Matthew Klein: We remain on schedule to initiate potential registrational trials of Tiquinone in refractory mitochondrial epilepsy in the third quarter and in pharyngocatastro in the fourth quarter. As we detailed in our BioWeek platform deep dive, the Phase 2-3 Mitochondrial Epilepsy Trial will enroll approximately 60 children with the most common mitochondrial disease subtypes that have refractory seizures as a key component of their pathology. This trial will include a one-month run-in period to ensure eligible subjects have a minimum frequency of observed motor seizures, followed by a six-month parallel arm phase in which subjects will receive either vitiquinone or placebo. We estimate that the refractory mitochondrial epilepsy market is approximately 11,000 to 13,000 patients in the US, EU, Latin America, and Japan.

Remain on schedule to initiate potential registrational trials, the particularly though in refractory mitochondrial let's see the third quarter and increase your capacity work work as we detailed in our biology platform. Besides the phase two three mitochondrial epilepsy trial will enroll approximately 60 children.

But the most common mitochondrial disease sometimes.

Refractory seniors as a key component of the pathology.

This trial will include a one month long answer to ensure eligible subjects have a minimum frequency of observed border features followed by a six month parallel arm C in which subjects will receive either for took went on what placebo.

Yes to me that the refractory mitochondrial epilepsy market is approximately 11 to 13000 patients in the U.S. Yoo Latin America and Japan.

So basically Friedrich attaching trial will be a 48 week double blind placebo controlled trial and will enroll patients couldn't U.S. Yoo, Australia, and Latin America, where we estimate the equity market size be approximately 25000 patients in aggregate.

Matthew Klein: The Phase 3 Fraser-Cataxi trial will be a 48-week, double-blind, placebo-controlled trial and will enroll patients from the U.S., E.U., Australia, and Latin America, where we estimate the F.A. market size to be approximately 25,000 patients in aggregate. We are working closely with site investigator teams and advocacy organizations to ensure that subjects for both of the Tiquanone potential registrational trials will be able to safely travel to and from study sites. In addition, we have adjusted certain elements of the study protocols to minimize any potential disruption that could occur in the event of a second COVID-19 wave. As Stu mentioned, we recently announced that the first patient has been dosed in the Phase 1 trial for PTC 857, the second compound from our BioE platform.

We're working closely with site investigator teams and advocacy organizations to ensure that subjects for both of the pick one on potential registrational trials will be able to seek we traveled to and from study sites.

Additionally, we have adjusted certain elements of the study protocols to minimize any potential disruption I could occur in the event of the second Colgate 19, we.

As you mentioned, we recently announced that the first patient has been dose in the phase one trial for PTC eight buying stuff.

Second compound for my bias required for.

Matthew Klein: This study is progressing well, and we look forward to having data from the single ascending dose and multiple ascending dose studies prior to the end of the year. Turning now to the splicing platform, our Huntington Disease Program remains on track to initiate first-in-human studies this year. These phase one studies will include both single and multiple ascending dose regimens in order to inform safety and pharmacokinetic parameters and inform dose selection to achieve target Huntington mRNA level reduction in the range of 40 to 50%.

This study is progressing well and we look forward to having data from a single ascending dose and multiple ascending dose studies fire TV and to me.

Turning now to the splicing platform or Huntingtons disease program remains on track to initiate first in human studies this year.

These phase one studies will include both single and multiple ascending dose regimens in order to inform safety and pharmacokinetic parameters and inform don't selection to achieve target Huntington M or in a level reduction in the range of 40% to 50%.

Matthew Klein: As you may recall, this strategy of validating target engagement and splicing activity in first-in-human studies was a key component of RISDAPlan's development program. Moving on to our gene therapy platform, due to COVID-19-related delays, we now expect to receive the final CHMP opinion for our AADC deficiency MAA in the first quarter of 2021. Analytical testing by our contract labs, which is needed to respond to standard review process inquiries, has been delayed as contract lab personnel and resources have been diverted to support their COVID-19 related efforts. Turning to the BLA submission for AADC deficiency, the key gating factor remains the study of the surgical use of the intended commercial cannula to deliver the gene therapy product to young patients. These treatment procedures have been delayed by hospital cancellations of elective surgeries due to COVID-19.

As you May recall this strategy validating target engagements and splicing activity in first in human studies with a key component Arista planned development program.

Moving onto our gene therapy platform due to cope with 19 related delays. We now expect received the final see HMP opinion, or our APC deficiency and eight.

In the first quarter of 2021.

Analytical testing by our contract lives, which is needed to respond to standard review process inquiries has been delayed its contract lab personnel and resources I mean diverted to support their covert 19 related efforts.

Turning to the BLE submission for HSBC deficiency, he dating back to remains the study of the surgical use of the intended commercial calculus to deliver gene therapy product to young patients.

He's treatment procedures have been delayed by hospital cancellations of elective surgeries due to covert 19th.

Matthew Klein: These procedures are now scheduled to occur in Q3, and we still expect to be able to initiate the BLA submission for AADC deficiency to the FDA in the second half of 2020, in the absence of additional delay. Finally, I want to share that we have completed the integration of programs from the SENSA Acquisition. We are in the process of completing the necessary non-clinical studies of PTC 923 to support the long-term dosing planned for the Phase III trial in PKU patients. Despite there being two marketed products, there remains a large, unmet medical need globally for PKU patients.

These procedures are now scheduled to occur in Q3, and we still expect to be able to initiate the BLE submission for AG DC deficiency to the FDIC in the second half of Twentytwenty in the absence of additional delays.

Finally, I want to share that we've completed the integration of programs from the sense acquisition. We are in the process of completing the necessary Nonclinical studies of PTC nine to three to support the long term dosing planned for the phase three trial in PK you patients.

Despite there being two marketed products there remains a large unmet medical need globally for PK you patients. We look forward to providing additional insights into the PTC nine to three T.K. you program and a deep dive later this year.

Matthew Klein: We look forward to providing additional insights into the PTC 923 PKU program in a deep dive later this year. As you've heard, despite the impact of COVID-19 on certain clinical programs and regulatory timelines, we have been able to advance a number of key programs and, in some cases, accelerate timelines. I will now pass the call to Eric to provide an update on the commercial business. Thanks, Matt.

As you've heard despite the impact of Koby 19 on certain clinical programs and regulatory timelines, we had been able to advance a number of key programs and in some cases accelerate timeline.

I'll now pass the call to error to provide an update on the commercial business.

Thanks, Matt.

Eric Pauwels: As Stu highlighted, the DMD franchise had strong growth this quarter, with both Enplaza and TransLarna generating significant revenue in Q2. Outside of Brazil, our DMD franchise had an outstanding quarter with revenue growth of nearly 20% year over year. Let me start with TransLarna.

You highlighted the DMD franchise had strong growth this quarter with Bolton Plaza and Translarnas generating significant revenue in Q2.

Outside of Brazil, Our DMT franchise had an outstanding quarter with revenue growth of nearly 20% year over year.

Let me start with trends Laurent.

Eric Pauwels: In our sixth year post-launch, we are exceeding expectations in key markets, with the exception of Brazil, which is one of the countries most severely affected by COVID-19. Importantly, we continue to find new patients in Europe, Latin America, and other key markets despite the challenges of COVID-19. Additionally, as Stu noted, we believe that the revision of the language on the TransLarna label will be positive for patients. We see the update as an opportunity to further educate physicians, caregivers, and patients on the impressive real-world results from the STRIDE Registry and Synergy DMD Natural History Study, highlighting TransLard as having long-term efficacy. Now, I will provide an update on the group purchase order for TransLarder in Brazil. As a reminder, in Brazil, the government is a central payer, by which only a few large group purchase orders are placed annually for the number of patients approved through the judicialization process.

In our six tier hosts launch we are exceeding expectations in key markets with the exception of Brazil, which is one of the countries most severely affected by covert 19.

Importantly, we continue to find new patients in Europe.

Latin America and other key markets, despite the challenges because it 90.

Additionally, as Steve noted, we believe that there were vision of the language on the trends Lorna label will be a positive for patients.

We see the update as an opportunity to further educate physicians caregivers and patients on the impressive real will result from the stride registry and energy DMD Natural history study highlighting Troy Ward is long term efficacy.

No.

Let me provide an update on the group purchase order for trains larder in Brazil.

Binder.

In Brazil, the government is a central pair.

Which only a few large group purchase orders are placed annually for the number of patients approved through the judicial innovation. So.

Due to the impact that cobot by team there was an administrative delays with payers for the centralized order.

Eric Pauwels: Due to the impact of COVID-19, there was an administrative delay with payers for the centralized order. We remain highly engaged with the Ministry of Health and are working to ensure that translanta patients will continue to receive this important treatment. In addition to our ongoing meetings with the Brazilian government, DMD advocacy groups and KOLs are also making their voices heard to advocate for trans-LARNA access in Brazil. These stakeholders are critical to ensure that payers have the most recent information on new and existing patients. Notably, we have seen a substantial increase in newly diagnosed patients in the second quarter. We anticipate an order later this year for both existing and new patients. Now, turning to Implaza.

We remain highly engaged with the Ministry of health and are working to ensure that trends lot of patients will continue to exceed this important treatment.

In addition to our ongoing meetings with the Brazilian government.

The M.D. advocacy groups and K wells are also making their voices heard to advocate for trend Translarna access in Brazil.

These stakeholders are critical to ensure that payers have been most recent information on new and existing patients.

Notably.

We have seen a substantial increase in newly diagnosed patients in the second quarter.

We anticipate an order later this year for both existing and new patients.

Now turning to imply that.

We are hitting our stride U.S. by bringing awareness to the community on the critical importance of in Plaza treatment for all DMD patients.

Eric Pauwels: We are hitting our stride in the U.S. by bringing awareness to the community about the critical importance of in-place treatment for all DMV patients. We continue to see ongoing improvements and greater efficiency supporting the business, resulting in more than a 30% year-over-year increase in second quarter sales. Based on early observations, we anticipate this strong performance will continue into the second half of the year. Our U.S. commercial team, comprised of regional accounts, patient engagement, case, and market access managers I identified new patients at a high rate this quarter, and it's helped accelerate time to commercial therapy. Patients previously on bridge therapy and patient assistance programs transitioned to commercial therapy even more rapidly in the second quarter. These new in-plaza patients included both naive and former prednisone patients, among our existing base of patients. Compliance and adherence remain very high, and discontinuation rates remain low on the plot.

We continue to see ongoing improvements and greater efficiency supporting the business, resulting in more than 30% year over year increase in second quarter sale.

Based on early observations, we anticipate this strong performance will continue into the second half of the year.

Are you have commercial team comprised of regional accounts.

Patient engagement case in market access managers I, then applied new patients at a high rate this quarter and his help accelerate time to commercial therapy.

Patients previously our bridge therapy at patient assistance programs transition to commercial therapy, even more rapidly the second quarter.

These new applaud the patients included both naive and former predators on patients.

Among our existing base of patients.

Compliance and adherence remains very high discontinuation rates remained low and plaza.

Now turning to took study.

Eric Pauwels: Now, turning to Tech Steady, launch activities in Latin America continue to progress well, and we continue to find new patients in Q2. We remain engaged in pricing discussions in Brazil and expect the process to be completed by the end of the year. Also, in Brazil, during the second quarter, we filed with M-VISA for approval of WeLibro. Patient finding and early access programs for weight lever are ongoing, and we continue to anticipate revenue from this product in 2020. To echo both Stu and Matt's comments, COVID-19 has impacted certain aspects of the commercial business.

Launch activities in Latin America continues to progress well and we continue to find new patients in Q2.

We remain engaged in pricing discussions in Brazil, and expect the process to be completed by the end of year.

Also in Brazil during the second quarter, we filed with M pesa for approval for way to lever.

Patient binding and early access program slow we lever our ongoing and we continue to anticipate revenue from this product in 2020.

To Echo books, do and Matts comments.

Cobot 19 hazard packet certain aspects of the commercial business.

We continue to drive key areas of the business forward.

Eric Pauwels: We continue to drive key areas of the business forward. I will now hand the call over to our Chief Financial Officer, Emily Hill, to review our financial progress. Thanks, Eric. I'm proud that PTC is in an excellent position to invest in our growing business and accelerate growth while maintaining fiscal discipline and a strong balance sheet.

I'll now hand, the call over to our Chief Financial Officer, I believe Hill to review our financial progress.

Thanks, Eric.

The PTC is an excellent position to invest in our growing business and accelerate growth, while maintaining fiscal just run on a strong balance sheet.

Emily Luisa Hill: We recently completed a transaction with Royalty Pharma that brought forward $650 million in non-dilutive capital. As a reminder, we retain nearly 60% of the RISD plan royalty stream, up until a $1.3 billion threshold is reached. After the threshold is reached, PTC retains the entirety of the RISD plan royalty.

We recently completed the transaction with royalty pharma that brought forward 650 million and non dilutive capital.

As a reminder, be butane nearly 60% of the rents to blame royalty stream up until a 1.3 billion threshold is reach after the threshold just reached PTC retains the entirety of the rest of plan royalty stream.

Emily Luisa Hill: The steel structure allows us to benefit from RISD's meaningful sales potential. We believe RISDiplam has the potential to be the most competitive commercial product in the SMA market and believe that its market potential exceeds current analyst consensus. Importantly, we also retain all economics associated with the approximately $400 million in remaining RISD milestone payments from ROPE.

The steel structure allows us to benefit from rest of plans meaningful sales potential.

We believe there is the plan has the potential to be the most competitive commercial product in the SMB market and believe it its market potential exceeds current analysts' consensus.

Importantly, we also retain all economics associated with the approximately 400 million at my meaningless to play milestone payments from rush.

As a reminder, we expect a 15 million milestone payment imminently associated with the filing of the M&A with the <unk>.

Emily Luisa Hill: As a reminder, we expect a $15 million milestone payment imminently associated with the filing of the MAA with the EMA. And following the first commercial sale in the U.S., we would receive an additional $20 million milestone payment. And now I want to take a few minutes to highlight the second quarter 2020 financial results, which are summarized in the press release issued earlier today. Starting with our top-line results, we reported $75.2 million in total revenues in the second quarter of 2020, compared to $85.5 million in the second quarter of 2019. As Eric mentioned, IMPLAZA had an outstanding quarter, and TRANSBARMA saw continued growth, with the exception of a delay in the group purchase order in Brazil. TransLarna Net Product Revenues were $38.6 million for the quarter.

And following the first commercial sale in the U.S., we would receive an additional 20 million milestone payment.

And I want to take a few minutes to highlight the second quarter 2020 financial results, which are summarized in the press release issued earlier today.

Starting with our topline results, we reported 75.2 million in total revenues in the second quarter of 2020 compared to total revenues of 85.5 million for the second quarter of 29 team.

As Eric mentioned imply that had an outstanding Florida and trends on the saw continued growth with the exception of a delay in the group purchase order in Brazil.

Translarna net product revenues were 38.6 million for the quarter. This compares to 57.8 million for the second quarter 2019.

Emily Luisa Hill: This compares to $57.8 million for the second quarter of 2019. As I've said, sales for the quarter were impacted by a delay in the Brazil Group Purchase Order, which accounts for the year-over-year decrease in second quarter revenue. From Pflaza, we reported net product revenues of $36.2 million for the second quarter of 2020, compared to $27.6 million for the second quarter of 2019. Growth in net product sales was driven by new patient prescriptions and continued operational improvements and efficiencies in our commercial business. Non-GAAP R&D expenses were $168 million for the second quarter of 2020, excluding $8.6 million in non-cash stock-based compensation expenses. Compared to $54.5 million for the second quarter of 2019, excluding $5.5 million in non-cash stock-based compensation expenses.

As I've said sales for the quarter were impacted by delay of the brazelton purchase order, which accounts for the year over year decrease in second quarter revenue.

From Plaza, we reported net product revenues of 36.2 million for the second quarter up 2020, compared to 27.6 reported for the second quarter of 2018.

<unk> net product sales were driven by new patient prescriptions and continued operational improvements and efficiencies in our commercial business.

Non-GAAP R&D expenses were 168 million for the second quarter 2020, excluding 8.6 million and noncash stock based compensation expense.

Compared to 54.5 million for the second quarter of 2019, excluding 5.5 million a noncash stock based compensation expense.

The increase in R&D expense includes one time charges associated with a refund acquisition on a manufacturing agreement.

Emily Luisa Hill: The increase in R&D expense includes one-time charges associated with a recent acquisition and a manufacturing agreement. Specifically, it includes $53.6 million related to the acquisition of Sensa Pharmaceuticals and $41.2 million related to the MassBio Commercial Manufacturing Agreement for our lead gene therapy program in AADC deficiencies. The majority of these one-time expenses are non-cash charges for the current fiscal year.

Specifically it includes 53.6 million related to the acquisition of sensor pharmaceuticals, and 41.2 million related to the mass bio commercial manufacturing agreement for our be gene therapy program in AIDC deficiency.

The majority of these onetime expenses are noncash charges for the current fiscal year.

Non-GAAP EPS DNA expenses for 45.3 million for the second quarter 2020, excluding 8.3 million and noncash stock based compensation expense.

Emily Luisa Hill: Non-GAAP SG&A expenses were $45.3 million for the second quarter of 2020, excluding $8.3 million in non-cash stock-based compensation expense. Compared to $43.8 million for the second quarter of 2019, excluding $5.4 million in non-cash stock-based compensation expense. The relatively flat year-over-year change in SG&A expense reflects our ability to leverage our existing global infrastructure. Net loss was $181.4 million for the second quarter of 2020 compared to net loss of $41.8 million for the second quarter of 2019. Cash, cash equivalents, and marketable securities totaled $498.9 million as of June 30, 2020, compared to $686.6 million as of December 31, 2019.

Compared to 43.8 million for the second quarter of 2019, excluding 5.4 million a noncash stock based compensation expense.

So relatively flat year over year, changing SGN I expense reflects our ability to leverage our existing global infrastructure.

Net loss was 181.4 million for the second quarter 2020, compared to not lost a 41.8 million for the second quarter of 2019.

Cash cash equivalents, a marketable securities totaled 498.9 million as of June Thirtyth 2020.

Compared to 696.6 million as of December 31st 2019.

Now, including the 650 million in cash received in July upon the closing of the deals royalty pharma unaudited pro forma cash cash equivalents, a marketable securities as of the second quarter would be greater than 1.1 billion.

Emily Luisa Hill: Now, including the $650 million in cash received in July upon the closing of the deals with Royalty Pharma, unaudited pro forma cash, cash equivalents, and marketable securities as of the second quarter would be greater than $1.1 billion. I will now hand the call over to the operator to start our question and answer session.

I'll now hand, the call over to the operator to start and question and answer session.

<unk>.

Thank you, ladies and gentleman, who like to ask a question. Please press Star then one on your Touchtone telephone again, if you like that the question. Please press Star then one one moment please.

Operator: Thank you. Ladies and gentlemen, if you'd like to ask a question, please press star, then 1 on your touchtone telephone. Again, if you'd like to ask a question, please press star, then 1. One moment, please. Our first question comes from Robyn Karnauskas of Truist Security. Your line is open.

Our first question comes from Robyn Karnauskas Truest Securities. Your line is open.

Hi, everyone. Thanks for taking my question. So I'm going ask your question the I get all the time upfront. So can you clarify for us.

Given the last deal you did how important is business developed for you versus say the development of your slicing platform, which I think a lot of people are really excited about help us understand how you prioritize your businesses I think that's the most common question I got I mean, asking on the call.

Robyn Kay Shelton Karnauskas: Hi everyone. Thanks for taking my question. So I'm going to ask a question that I get all the time up front. So can you clarify for us? You know, given the last bill you did, how important is business development for you versus, say, the development of your splicing platform, which I think a lot of people are really excited about? Help us understand how you prioritize your businesses. I think that's the most common question I get, so I'm going to ask it on the call.

Sure. So oh, thanks for the thanks for the call you know obviously, our overarching goal is always to bring.

Innovative therapies to patients with high unmet medical need and I think we.

You can see we rebuilt the company.

You know over the last 22 years, it's done here for the build it to have a strong pipeline over the over the next 20 years and the strategy has really been do you see a terminal discovery capabilities that we've built.

Unknown Executive: Sure. So thanks for the call. You know, obviously, our overarching goal is always to bring innovative therapies to patients with a high amount of medical need. And I think we, you know, as you can see, we've built the company over the last 22 years and will continue to build it to have a strong pipeline over the next 20 years. And the strategy has really been to use the internal discovery capabilities that we've built, as well as business opportunities, in order to bring a number of products that are critical for growth. But that's also true for business development, right? You never know for sure when things are gonna be ready for getting to commercialization, so we look to fill the pipeline as needed through business development. And it's helped us to evolve and develop COREXPERT, such as gene therapy, redox, and inborn errors and metabolism.

As well as business opportunities it continues to grow and the spikes in platform as you see I think has created a subset you know substantial value the plan and I think from the deep dive.

You could see how.

The opportunities out there and available so we plan as part of what we've done this to spend all of its expanded from that so you know the way I look at this is really in the sense shots on goal and that.

No you need a relatively broad pipeline Oh in order to you know, bringing the number of products.

You are critical for growth, but that's also true for business development rights on but you never know for sure when things are going to be ready for Oh.

Getting to commercialization. So we look to fill in the pipeline of needed through business development and it's helpful to evolve and developing additional nucor expertise.

Such a gene therapy, the lead us to the inborn error on somebody tablet for people are important to us as well.

Unknown Executive: So these are important to us as well, and we think there are some near-term value creators, like in inborn errors and metabolism. We'll be starting the registration studies, the BioE programs, we'll be starting the two platforms that are also pivotal studies that can start now that could be for you know in the 2023 time frame, so there's a lot of value that we think we can do in terms of creating value. So the way we're looking at that is we're highly excited about our innovation capability. And we're going to be pursuing them with great zeal. So the therapeutic area is the platform, and the commercial footprint, I think is how we're going to continue to grow both innovation and revenues for the company. Does that help?

Thanks to some near term value creators like him, but the inborn errors of metabolism.

We'll be starting to registration study. So vital you programs will be starting the to what front that also pivotal studies that could be the starting now that could be for the 2023 timeframe. So there's a lot of value that we think we can do in terms of creating value.

So we were looking that that is were highly excited about our innovation capabilities.

And we're going to be pursuing bad with rig that.

Moving forward.

And we look for business development opportunities in the you know and really strategically and in this case you know now that we've built a a pipeline and platforms.

Vertically strengthen if we've seen something.

But that interesting and then the other point is obviously the current platform.

Robyn Kay Shelton Karnauskas: Yeah, I mean, I think the big question is, Is focusing on business development in any way a distraction from developing your current drugs, in particular that from the spicing platform, which is a near-term catalyst? That's a more direct question. Yeah, and I think that's fair.

Yeah.

It's important for the therapeutic area the platform in the commercial footprint I think that's how we're going to continue to grow.

Bofi innovation, but also grew revenues for the company.

Does that help you.

Yeah, I mean, I think the big question. It is focusing on business development and anyway, a distraction from developing your current drugs in particular that from the slicing platform in the near term catalyst.

Yeah, that's a more direct [laughter], yet I think that's there and there's no it doesn't it isn't like where.

Unknown Executive: And the answer is no, it doesn't. It isn't like our pipeline is not yet big enough to say that we're prioritizing A versus B and that we have the resources to be quite focused to make sure, especially now, to really go whole hog through the spike. [inaudible] So I think, you know, we, you know, we're pretty focused on that we're strengthening our current platform. So I don't think you know, that's a very important point. It's not like we're deprioritizing one for the other right now. We're not big enough yet to be able to say that we're going to be doing that. So it's not a distress call.

The other part one is not yet big enough to say that we're prioritizing a versus b and that we have the resources to be quite focus to make sure, especially now to really go a whole hogs really quite see a platform and and and so I don't think it's a diversion.

Oh, because there are obviously different people that are moving forward on this the way we exit constructed the team now or the company in terms of organizational structure isn't sort of pulling from one group to another it's building out seems to be able to him, but handle that and then have the infrastructure for them to be autonomous.

All forward looking up oversight so.

Robyn Kay Shelton Karnauskas: Got it. Thank you so much. Thank you. Our next question comes from Joel Beatty of the City of Atlanta, Philadelphia. Hi, thanks for taking the question. The first one is to follow up on that last question from Robyn. From a business development standpoint, what types of programs would you be looking for that would have the best fit with PTC, you know, at least from a high level?

Well I think no we were pretty focused on that we're strengthening or our current platform. So I don't think you know that's a very important point, it's not like would you prioritize and one for the other right. We're not big enough yet to be able to say that we're going to be doing that.

So it's not it thank you Simon.

Got it thank you so much.

Thank you.

Next question comes from Joel Beatty of Citi Your landfill.

Hi, Thanks for taking my question.

The first one is to fall off on that last question from Robyn.

No. This was felt that standpoint, what types of programs. All would you be looking for that would have the best PTC you know at least from a high level.

Joel Beatty: I guess from a high level. Thanks, Joe. I mean, when we tend to think of it from a high level, it's really to look at within the vertical, you know, if you think about it, we now have either gene therapy, you know, BioE, the splicing platform, right, so it's within the platform that we've built, that will be moving, that will probably, we're not looking to create additional, or embroider the areas of metabolism, we And so, you know, and therefore, and that could also help in our commercial. A question on RISDEPLAM.

I guess from a high level. Thanks.

I mean, when we tend to think about from a high level, it's really to to look at within the the vertical Oh you know if you think about it. We are we now have it was the therapy, but you know bio he.

The splicing platform right. So it's it's within the platform, but we believe that we'll be moving that will probably we're not three but just north or inboards Irina metabolism. We're not looking to go outside of that area. So we're looking carefully for.

You know selective strategic BD opportunities.

You know that focus efforts on the current and future isn't platforms that were we haven't played.

And so could you know therefore and that could also help enhance our commercial footprint.

[noise] Gotta that's helpful.

And on rents to plan some have there been discussions with FDA and a potential indication that anything you can give them that's gives confidence and FDA meeting that put deferred dates.

Unknown Executive: Have there been discussions with FDA on potential indications? Anything you can give to help give confidence to FDA meeting the PDUFA date? How do you anticipate the label could compare with the approved SMA drug? Yeah, so obviously, we're waiting for the BDUPA date, and we strongly believe that it's certainly going to be approved by that time. And we, you know, we strongly believe it's the best in class product.

You do it and how do you anticipate the label could compare with the approved somebody else.

Yeah. So obviously, we're waiting on chemo waiting for the PDUFA date.

We strongly believe that it's certainly going to be approved by that time.

And we you know we strongly believe what gets the best in class product and we think with the most commercial the competitive product out there. We think that obviously from our point of view as can be Oh brought have brought African speed of will be the standard of care. We expect that the label could that be a very broad for all.

Unknown Executive: And we think that, obviously, from our point of view, it's going to be a broad app, will be the standard of care. We expect that the label is going to be very broad for all FFA. And then it's also supported by the other programs, the Jewelfish and Rainbowfish.

At some they types, including one two and three.

Obviously, we have the data for placebo controlled trial Oh, both for somebody type one for those younger babies.

Patients and even then I think even there when you compare that data sets in terms of even in the type ones. The older patients and fees benefit there that wasn't seems like others. So we think it's really quite strong in Minneapolis type two three in the older patients we saw in the into something even a strong data as well and then the cost.

Unknown Executive: So at the end of the day, we think it's going to be a very strong label for both the type ones as well as adolescents and adults. Obviously, the first and only treatment that's an orally bioavailable product, so it's home administration. And in particular, in this environment now, that's a huge benefit. You don't have to go to the hospital to see a physician.

Supported by the other programs the jewel prison Rainbow fish so.

At the end of the day, we think it's going to be a very strong label.

<unk> for both the type ones as well as adolescence and adult.

Obviously, the first and only treatment that's an orally bioavailable products. So its home administration on them and in particular in this environment now that's a huge.

Benefit you don't have to go to the hospitals the position that the strong safety profile.

Unknown Executive: There's a strong safety profile. And we think, you know, we think that it's done quite well. So we're very excited about that. Great, thank you. Thank you. Our next question comes from Althea Young of Kansas. Hi, this is Leon from Lydia. Thanks for taking our call. Maybe just one on ADC.

And we think you know we think that is something nice went quite well. So we're very excited about that.

Great. Thank you [laughter].

Thank you. Our next question comes from Alethia Young of Cantor Your line is open.

I think certainly arent called media. Thanks for taking my call maybe just one on a D.C. can just keep us more color on their remaining starts to fall B. Riley.

Althea Young: Can you just give us more color on the remaining steps to file a BLA and then how confident you are that you can file by year-end? And then another one on the U.S. distribution study. Just wondering if you think the COVID challenges might make it harder to integrate the results given the delay? Sure. So let me remind you that we, you know, we'll talk about the BLA. Submitted.

Now how confident that you can file by year end and another one on the U.S. just trips and study I'm. Just wondering if you think a copy challenges might make it harder to integrate their because given the duly and base. There in the you know that's baked into guidance on that thanks.

Sure.

So let me remind you that we will talk about that.

So they do.

Unknown Executive: So, you know, we're moving through Europe. The key gaining item, as you know, is the surgeries with the commercial cannula. And I'll let you. Would you like to talk a little bit about that, Matt? Yeah, absolutely. So, as Stu said, the key gating item is the surgical procedures with the intended commercial cannula, which is the SmartFlow cannula.

So you know we're moving through Europe the key.

Jamie and I don't as you know, it's the surgeries with the commercial Angela.

And I'll, let 'em.

Matt.

You want to talk a little bit about that Matt.

Yeah, absolutely so as Steve said I'd be really getting items the cost the surgical procedures with the intended commercial cry Hilla, which is the smartflow carrying though now this is CE marked for gene therapy delivery anything you in the U.S. Hudson approved device just not explicitly for the delivery of gene therapy into the prepayments.

Matthew Klein: Now, this is CE marked for gene therapy delivery in the EU. In the US, it's an approved device, just not explicitly for the delivery of gene therapy into the containment. It has been used in clinical trials with a good safety record for gene therapy delivery into the containment of adults. And so really, that last piece is getting some experience in the surgical administration of our gene therapy product with the SmartFlow device. So it's really an assessment that we've been asked to provide of the device and the surgical procedure for the delivery of the gene therapy product into the containment. Once we have those procedures completed, we'll, of course, move forward with final PBLA discussions with the agency and then move forward with preparation for the submission. With regard to the dystrophin study, obviously, we're all frustrated by the delays in the COVID trial. I mean, PTC, as you know, has an incredible longstanding, probably the longest-standing history of being dedicated to developing therapies, specifically Transmarna for DMV patients. We're obviously incredibly excited to receive the sixth annual renewal in Europe.

Has been used in clinical trials with a good safety record for gene therapy delivery to the payment of adult and so really that last piece is getting some experience in the surgical administration on bags multi product with the smartphone device. So it's really an assessment that we've been asked to provide the device and the surgical procedure.

Sure.

So delivery of gene therapy product instead contain.

Once we had those procedures completed well of course move forward side, who are vital P.B.L.A. discussions with with the agency and then move forward with preparation for the submission.

With regard to the Dystrophin study, obviously, where all.

Frustrated by the delays from the clothing trial.

You see as you know has an incredible longstanding quite longest standing history of being dedicated to developing therapies, specifically translarna for DMD patients were obviously incredibly excited to receive the six I know we knew on Europe. The evidence that we're getting to collect on a number a function of long term benefit I see our strides.

Matthew Klein: The evidence that we're continuing to collect on a number of fronts is showing long-term benefit through our stride registry. And now we're at the point that we're just waiting for these last eight patients to come in and get their final biopsies so we can analyze all the results. And clearly, we want to get this study read out by year's end. Obviously, there's some unpredictability due to the pandemic, which is obviously affecting not only the study site in California but also the states where some of the patients live, such as Texas and Arizona. And, of course, first and foremost, we want to ensure the safety of the procedures.

Registry.

And now we're at the point that we're just waiting for these last eight patients to come in and get their final biopsies. So we can analyze all the results thing clearly.

We want to get this study read out by years end, obviously, there's some unpredictability high due to the pandemic, which is obviously affected not only the study site in California, but also in the speech, where some of the team since when such as Texas, Arizona and of course first and foremost we want to make sure. The safety of the procedures were still in the process of sorting out.

Matthew Klein: We're still in the process of sorting out the exact timing of biopsies at UCLA. Unfortunately, it looks like the pandemic numbers may be slowing down, and so we're in constant communication with the site to see when we can get a better idea of the specific timing for those final biopsies. And, of course, most importantly, we're ensuring that these patients don't have any disruption in the supply of Transmarna so that when we're able to get their biopsies, we'll be able to do so in the context of ongoing Transmarna therapy. Thank you. Our next question comes from Joseph Comrie of Cowen & Company. Your line is open.

The exact timing of biopsies that you kill a fortunately it looks like a pandemic numbers, maybe slowing and so we're in constant communication with the site to see when we can get a better idea.

Specific timing could those final biopsies and of course, most importantly.

We're ensuring that these patients don't have any disruption in the supply of Translarna. So that when we're able to get their biopsies, we'll be able to do so in the context of ongoing trend wise.

Thank you. Our next question comes up with Tommy of Cowen and company. Your line is open.

Hi, there. Thank you for taking my questions. The first one on the an acre APC I think you indicated that the he may have some feedback.

Joseph Patrick Schwartz: Hi there, thank you for taking my questions. The first one on the MAA for AADC, I think you indicated that the EMA has some feedback on some additional information that they need from you. If you could just clarify, kind of, what sort of information do they need and did they put you on a stop clock so you have that extra time to respond, or is there a time limit?

Some additional information that they need from you. If you could just call if I kind of what sort of information do they need and ended the put you on a stop clock. So you you have that extra time to respond or is there a time limits and then second if you could just update us on the progress for I entities for PTC after a pretax.

Matthew Klein: And then second, if you could just update us on the progress for INDs, for PTC, for Fragile Taxia, and Angel. Sure. So Matt, do you want to talk a little about the MAA? Yeah, absolutely. Thank you for the question. As we mentioned, we submitted the MAA in January, and based on the standard MAA timelines, we expected the final CHMP opinion in late December of this year. That's just based on the pre-specified timeline from the EMA. During the review process, the agency asked us some pretty standard questions, wanting additional manufacturer-related analyses done on the drug product. These are all done by our external commercial manufacturing organizations. They have been impacted by COVID in a few ways. One is, obviously, a decrease in the number of available personnel.

Angel.

Yes.

Sure. So mark you want to talk a little but the M&A.

Yeah, absolutely. So thank you for the question. So as we mentioned we submitted the any in January and based on the standard Emmy a car lines. We expected the final CHP opinion in late December of this year. That's just based on the pre specified a timeline Co. Committee M&A.

During the during the review process I mean, yes, the some pretty standard questions wanting additional manufacturing related analyses done on the drug products. These are all done by our external commercial.

Capturing organizations of fee they have been impacted by Cobi a in a few ways one is.

Obviously decrease the number available personnel, but also our lead Cmos are involved with other companies and developing solutions for the covert pandemic, so obviously being redirected their resources toward the personnel and other resources towards the cobiz related activities.

Matthew Klein: But also, our lead CMOs are involved with other companies in developing solutions for the COVID pandemic. So, obviously, they've redirected their resources, personnel, and other resources toward COVID-related activities. Therefore, in order to be able to satisfactorily respond to the EMA's questions to us, we were granted a stop clock so that we can then, obviously, come back and address the questions, which are easily addressable once we have the available resources to do so, and plan now for the final opinion in Q1 of 2021. Great, thank you. And then just the update on when we can see the IND for free Zytaxia for that, if there's any. I think we said it was delayed by a quarter.

Therefore in order to be able to satisfactorily.

Respond to the Emmys question in stocks, we were granted to stop clock. So that we getting done obviously come Bakken and address the questions, which which are easily addressable once we have a the available resources to do so and plan now on the final opinion in Q1 2021.

Yes.

Great. Thank you and then just a the update on when we could see the I indeed for free so taxi f. reductions or if there's any uptick.

Oh, I think we said it was delayed Oh.

Unknown Executive: We announced last quarter that we're continuing to work to advance them both, but there have been a number of COVID-related delays for at least a quarter. So, you know what? We really do remain enthusiastic about both of these programs. And we're working to really get it all done.

A corner, we announced last quarter that we're continuing to work to advance them both.

But there's been a network colgate related delays.

For at least the corridor. So no we really do remain enthusiastic about both of these programs.

And we're working them really at all gone. So we can get that done so that's where we're at now so it's similar to what we reported the last time.

Unknown Executive: So that's where we're at now. So it's similar to what was reported the last time. Great, thanks again.

Great. Thanks again.

Thank you. Our next question comes from Brian Abrams of RBC. Your line is open.

Brian Corey Abrahams: Thank you. Our next question comes from Brian Abrahams of RBC. Your line is open. Hi, this is David Cicilline on behalf of Brian.

Hi, This is David centered on for Brian. Thanks for taking my question.

David Neil Lebowitz: Thank you for taking my question. Thank you. Thank you. Thank you. [inaudible] I think your line is not muted, ma'am.

Moving out.

[laughter].

Huh.

[noise] Hello, somewhat I think your line is not beauty.

Ma'am.

Okay go ahead, David sorry.

Operator: Go ahead, David. No worries. Yeah, so just another one on AADC. You know, from your ongoing pre-launch activities, maybe engaging in the changing market, could you just update us on your evolving sense of the clinical pathway to identify AADC patients, and if there are perhaps differences in the U.S. versus EU?

No worries deficit another one on a they see you know from your ongoing prelaunch activities, they've engaging and that's in the market, but it's just update us on your evolving funds the clinical pathway to identify anticipation.

And if there are differences in the U.S. from you I'm, just kind of sense of what under current level of confidences and.

David Neil Lebowitz: And just make a sense of what your current level of confidence is in the epi year, and then have a follow-up after that. Sure. So, um, Eric, you want to talk a little bit about our patient findings?

Pete here and then I've a follow up after this.

Sure. So Eric you want to talk little about about our policy should probably be.

Efforts.

Yeah Sure said I think we have we're continuing to work very hard to identify.

Sure.

Eric Pauwels: Thank you, everyone. Thank you. Thank you. Yeah, sure, Stu.

By Tobin Nike.

We've been raising disease awareness.

That's driving testing, particularly indexes that are patients are at high risk, so actually fall declare click and collect.

Eric Pauwels: I think we have, and we're continuing to work very hard to identify AADC patients. You know, despite COVID-19, we've actually been raising disease awareness and driving testing, particularly in areas that are patients that are at high risk, so through all the clinics and epilepsy clinics. So, patient identification continues to progress well, and as we've said, our goal is to find 300 patients by the time we're prepared to launch in our first key markets globally. Thank you. And then maybe just a quick follow-up on transbarna again.

That's correct.

We've accelerated a lot of our master class argued.

Asian key opinion leaders programs AD boards search Committee symposium is actually published clinical data. So in terms of raising the awareness, we really been so our level of activity.

For awareness and identify patients.

Standard a lot of our activities to a number.

Different countries as well both in Europe Asia Pacific in Latin America and Quickstart.

Yes, it's into that we've been having a lot appeared to Scott.

I really do watch the clinical data to understand.

He therapy like it and to look at proposition. So a patient identification continues to progress well.

As we've said our goal is to it is to find 300 patients and Oh by the time, we're prepared to launch our first the markets globally.

Thank you and then maybe just quick follow up Oh Chung Barnhart jobs are going back to your opening remarks discussing the recent suits them directly ambition.

David Neil Lebowitz: So going back to your opening remarks discussing the recent CHMD recommendation to open the label to patients who become non-ambulatory, I was just wondering if you could provide maybe an update on any other evolving dynamics among physicians in the EU and if you have any perspective on expanding transbarna's label to initiate treatment among non-ambulatory patients. Thank you.

Now to open a the label to know if there's going to become non ambulatory I'll just wanting to provide.

They've been of Didnt do on any other evolving dynamics monk positions in the you and if you have a perspective on expanding fans on as many able to initiation among non ambulatory patients.

Thank you yeah yeah.

Yes. Thanks for that question, you know, where we were really.

Unknown Executive: Yeah, we were really heartened by this and had substantial positive feedback from both physicians and payers on the revision. And this really does allow patients to keep up, have a path to remain on Translant even as they transition to the non-ambulatory, [inaudible] Eric in terms of what Europe is doing. Yeah, first of all, we've had a lot. Thank you for joining us.

Oh heartened by this and.

Substantial positive feedback from both.

Physicians of payers on the revision.

And this so really does allow the patience I keep that have a path to remain upon.

Oh trends largely when this thing crudes listening to the non handbook sorry.

Pardon and so that's actually really important.

In certain countries has already has a willingness to do this.

In terms of non ambulatory that really is sort of country by country as well right now certainly when you think about doesn't make sense from this point of view that if you're gonna go toward from going from memory to non inventory that you'd like to.

Eric Pauwels: Physicians and payers have looked at this as being, you know, a positive step forward. So, immediately after that announcement, a number of patients that might have actually been considered to stop because they went on ambulatory continued treatment. So, this willingness, if you will, to not implement a stopping criteria was critically important.

Good work do you mind Oh.

Maybe a little bit you want to talk a little bit above the.

Eric in terms of Western Europe is doing.

Yeah, I first of all.

Yeah I can immediately after the announcement, hi, physicians and payers I've looked at this is a big you know a positive step coal. So immediately after the after that announced that a number of patients that might have been actually consider or a stock because they went non ambulatory continue treatment. So this.

This willingness if you will.

To not and implemented stopping criteria and it was critically important so we've already actually.

Eric Pauwels: So, we've already actually, since we've already actually seen patients that aren't in, that have been on TransLorna, go on ambulatory and stay, it gives a lot of physicians positive feedback and continues, if you will, this dialogue that they could have right now if they couldn't with certain payers in Northern Europe, in Southern Europe, and even in places like Latin America where payers viewed the label, the So, now there's a lot, there's going to be more in the future, I think, patients and physicians will have a positive risk benefit discussion with payers. And by removing that, the label provides, if you will, a constructive dialogue that can be handled by the physician and the payer.

He actually keep patient it at our net debt if they don't trend, Florida go down inventory and say it gives a lot of the physician positive feedback and continued if you will this dialogue that they could have right. They couldn't with certain pairs in northern Europe odd in southern Europe any.

Even in places like Latin America, where pair reviewed the label the wording of a label as a source soppy barrier or stopping criteria. So now there today.

Much they're going to be now in the future I think patients and physicians will have [laughter] discussion with parents and by removing that no label provides a well if you will a constructive dialogue.

I can be handled by disposition and repair.

Yeah, and then the other thing.

Unknown Executive: And then the other things that we've done, a fair amount of work both in our stride registry and in the non-ambulatory patients following them. And what's really nice is that the results that we saw in the clinical data have been shown in patients, and actually, you can use much harder endpoints now. The publications on the stride data are really quite clear in terms of the substantial improvement on not only walking longer but multiple other time function test measurements. So you see substantial improvement. And also, what we've seen in the non-ambulatory patients, clearly better pulmonary function. I mean, a very clear demonstration when compared to natural history.

A fair amount of work both in our stride registry as well that is a non ambulatory patients following them and what's really nice is that the results that we saw in the clinical data has been shown.

Patient and actually you can use know much harder endpoints.

Those the publications on this Friday, this really quite clear in terms of the.

Substantial improvement.

Not only the walk team longer but multiple over.

Ton function tests Muslims, even see substantial improvement and also what we've seen isn't the non ambulatory occasionally patients clearly better pulmonary function.

I mean, very clear demonstration when compared to the natural history. So we're excited is all about December route where we do try and work to make sure that none of them was torn patients have always had good translarna as well.

Unknown Executive: So we're excited about all of this, and we do try and work to make sure that non-ambulatory patients have a way to get translarna as well. Great, thank you. Thank you. Our next question comes from Raju Kasad of William Blair. Your line is open.

Great. Thank you.

Thank you on next question comes from Roger you could kind of William Blair. Your line is open.

Hi, there this is a sammy on for Rob and I had a question regarding dystrophin study [laughter].

Raju Kasad: Hi there, this is Sami on behalf of Raju. I had a question regarding the dystrophin study. At what point, or what would be the scenario in which you forego the biopsies or a portion of them in order to file that BLA and try to understand which event you're prioritizing? So the, I think, you know, the, so we have 12 out of the 20 that have been done and that have been treated and they've not been, they're still blind, so we don't know the data within there. We just, you know, the study was performed with 20 patients pre and post, and they're still being treated. So we just think that it's better. It's better to have all the patients as possible.

[laughter] point.

Well what would be the scenario in which you wont go that biopsies or question I've done in order to file that'd be a lag [laughter] trying to understand on what should that you're prioritizing.

So the.

I think you know the <unk>. So we have 12 out of the 20 markets and done then but so.

The trees and they've not been.

There's still blinded. So we don't know data within there were just you know this study was the study was performed with 20 patients brings problems.

And there's still be treated so we just think that that's a better.

It's better to have all the patients as possible. If this continues and extends on will we you know there. So we can say maybe we should just look at the things that now.

Raju Kasad: If this continues and extends on, well, we, you know, there is a possibility we could say maybe we should just look at the data now in an interim way and take a look at that. But we'd obviously need to align with the FDA on that before we were to do that. So we don't wanna look at the data before we agree with that. But it's really a question of if we think it's gonna take forever or not.

In the room way and take a look at that.

So, we'd obviously need to align with the yesterday on back up before we were to do that so we're going to look at the data before we agree with that but it's really a question of if we think it's going to take forever or not so that's how I thought.

Unknown Executive: So that's our thought. Thanks, that was really helpful. And just a quick follow-up, how are patient identification efforts for the mitochondrial epilepsy trial going, just to get an idea of how quickly you'll be able to enroll patients in that trial? Sure. Matt, you want to sort of talk a little bit about patient identification for the trial? Yeah, absolutely. So just for some historical background, you know, 743 was really the first drug to be brought into the clinic explicitly for pediatric mitochondrial disease patients and has an enormous amount of brand recognition in the mitochondrial communities globally.

On that.

The thing goes way up when does that just a quick follow up on.

However, patient identification efforts more the mitochondrial epilepsy trial, just to get an idea how quickly you'll be able to enroll patients in that trial.

Sure.

Matt you, one sort of talk little bit about things Mike.

Hello.

Yeah, absolutely so just for some historical background or 743 ways.

Really in the first drugs three brought into the clinic explicitly core pediatric mitochondrial disease patients and has an enormous amount of brand recognition in the mitochondrial communities a globally. How we also have very good working relationships with he patients a foundation spoken to U.S. can you Australia.

Matthew Klein: We also have very good working relationships with patient foundations both in the US, EU, Australia, and Japan, and so we've been relying on this network to help us get the work out that this trial is starting. There's already a great deal of enthusiasm in a number of countries in which the trial is going to be conducted, and we are, you know, really excited to be able to launch the trial and believe that we can rapidly enroll the trial again, COVID permitting. Great, thanks for taking my question. Thank you. Our next question comes from Gina Wang of Barclays. Your line is open. Hi, thanks for taking my question. This is Peter for GMA. I guess two questions for me first on transnarna.

Japan, and so we've been relying on this network to help us get the weren't offset this trial, starting there's already a great deal that's easy hasn't been a number of.

Countries in which the trials calling to be conducted a and we are really excited to be able to launch the trial and believe that weekend.

Rapidly in little bit trial, again coding koby permitting.

Great. Thanks for taking my question.

Thank you next question Constantino Wangle Barclays Your line of Brooklyn.

Hi.

Thanks for taking my question this is Peter but she'd on that.

I guess two questions for me first on trends, Florida.

Unknown Attendee: How much of the quarter of the quarter decline is due to COVID-19 and any color on impacts on new and existing patients and for that half of the year. We expect some growth relative to the first two quarters or largely stable, excluding the Brazil order. Okay, say the first part again. For Transmariner, how much of the photo over quarter decline is due to the pandemic? Okay. Oh, okay. Great. Yeah, so Eric, you want to talk a little bit about? Yeah, sure. I think there's very little decline if we look at the major markets. In fact, I think transnational key markets outside of Brazil continue to grow.

How much up quarter over quarter decline is due to.

Oh, probably nine Jeanette any color on.

On new and existing patients and for back half a year.

He expects some growth relative to first two quarters.

Or largely stable, excluding the Brazil order and I have a quick follow up thank you.

Okay.

The first part again [laughter].

Hi.

Translarnas, how much of the photo of the quarter over quarter decline is due to.

Got it.

Okay, Okay, great Oh.

Yeah, So Eric you want to talk a little bit above that.

Yeah sure I think there's very little decline if we look at the made a major markets contract think treads law all key markets outside of Brazil continues to grow and we've been seen seeing new patient identification.

Eric Pauwels: And we've seen new patient identification and new patients come on to therapy, even in some of the most affected countries, like in southern Europe, like Spain and Italy, where we've had new patients go on to treatment. We've also seen very high adherence rates, compliance rates, very, very low dropout. So essentially, in the main areas, the main regions, we haven't actually seen much impact at all from COVID-19. The main issue and decline are primarily the administrative delay from Brazil in the group purchase order at this time.

New patients come onto a therapy, even in some of the most affected countries like southern Europe, like Spain, and Italy, We've had new patients go on to treatment.

We've also seen very high here in Threeq.

Why is raised very very low dropout.

Essentially in the main area the main region.

We haven't actually seen.

Much impact at all from Cobot 19, the main issue in decline is primarily.

The the administrative delay or from Brazil, and the group purchase order at this time.

Yeah, I think also you might remember Peter.

Eric Pauwels: Yeah, I think also you might remember Peter, in my initial comments, I said out, you know, outside of Brazil, we saw 20% of, Ah, right. Okay. Thanks. Thank you. Um, my second question is, to the extent that you can provide any color on RISD Plan EAP.

First in my comments initially I set out you know outside of Brazil, We thoughts what did you say approximately 40% increase.

Right. Okay. Thanks, Thank you.

Yes.

My second question as to the extent that you can buy any color on.

Well the plant 80.

Unknown Attendee: Would you be able to give some color on how the enrollment rate has been impacted by COVID-19 relative to expectations and any color on, you know, folks switching from this nursing? Thank you. Yeah, sure. I guess I don't think we've ever given numbers on how things are going.

Would you be able to give color on.

Like how the enrollment rate has been impacted by koby nine change relative to expectations and any color on.

Folks switching from listeners. Thank you.

Yeah sure I guess.

I don't think we've ever given numbers on on how things are going but things actually in terms of GMP had gone actually quite well I know that roche's quite satisfied.

Unknown Executive: But things actually have gone quite well in terms of the EAP, and I know that Roche is quite satisfied with how the EAP has been going. We do expect on approval that, you know, we think that, obviously, we're confident in Roche's ability to launch this quickly. And we think, in particular, there'll be a lot of transitions from Spinraza to RISDAPlan. So we think that's gonna be a very strong part of the growth of RISDAPlan over time, as well as obviously the naive, in particular, adolescents. There are so many patients that are not being treated that I think RISDAPlan, as an orally bioavailable agent, is very well suited for these patients.

We have hobby team has been going.

We do expect approval that you know, we think that obviously, we're confident well to the roche's the village.

The launch this quickly and we think in particular, there will be a lot of transmission from Oh from Spinraza.

Two routes to plan. So we think that's going to be a very strong part of the of the growth of the platinum overtime as well is obviously the night.

Ocular so you know the adolescence.

There's so many patients that are not treatment, but I think Bristol plan for orally Bioavailable agent is very well suited for these patients. So we're excited to the buses more Oh lawn crews. We think we anticipate mall are quite quickly.

Unknown Executive: So we're excited about this launch, which we think, and we anticipate will occur quite quickly. Thank you. Our next question comes from Eric Joseph of J.P. Morney, and the line is open. Thanks for taking the question. Just on TransLarna, picking up on your prior comment, please talk a little bit about the extent to which they are interested in results from Study 45 as they look to... (inaudible) I guess, is there any sensitivity on their end on the outcomes of Study 45? How to look at stride registry data

Thank you. Our next question carbon Eric just sort of JP Morgan Your line is okay.

Yeah. Thanks for taking my question just on trends Lorna picking up on your prior comments about.

And with the main health authorities can you talk a little bit about the extent to which they are interested in resulting study 45 as they look to.

Essentially renew conditional approval again and 21.

Let me give some health authorities you know.

I guess is there any sensitivity on their end on 30 45 outcomes versus how to look at stride registry data.

Eric William Joseph: Oh yeah, sure. Thanks for that, Eric. I think one important point there is that it was very clear that the European authorities didn't look at dystrophin as a biomarker that really predicts any efficacy.

Oh, Yeah sure. Thanks for that area I think one important point for you is that it was very clear of that the European authorities.

Look at dystrophin, and say as a vital mark because everybody predicts any efficacy. So it really everything was based on the clinical result can so you know weve kept them are probably not only the clinical data that will have but also the clinical data from the strides in the non ambulatory data so.

They know that data quite well.

And so the.

Unknown Executive: So, really, everything was based on the clinical results. And so, you know, we've kept them apprised not only of the clinical data that we have, but also the clinical data from the strides and the non-ambulatory data. So they know that data quite well. And so, if what the results from study 45 are, I don't, and many other countries outside of the United States.

Yeah.

As a result from WD 45, I don't think will be of any service to India made sense, whether good or bad because I don't think that.

It isn't a biomarker tobacco reason would predict clinical benefit and so they had no interest in it in the past I think you did see from another company when they talked about the result, but they didn't make any headway either so but not using the something that's an approval.

Bio marker, so and that's I think true most other countries outside the United States.

Got it that's that's helpful and.

Unknown Executive: Got it, that's helpful. And maybe a follow up on Inflaza. I'm just wondering if you could help us unpack the dynamic of the strong quarter here. How much is sort of driven by, you know, efficiencies in the prior operation process versus growth in new patient ads among naive patients should we anticipate a similar benefit in the second half of the year? And I guess also, we have just noticed that the phase three limb girl study also completed enrollment recently. Can you just remind us whether this is a potentially labeled expanding trial and what regulators might be looking forward to enabling that? Sure.

Maybe a follow up on when a plaza I'm just wondering if you could help us unpack the.

The dynamic to the strong quarter here, how much is sort of driven by you know efficiencies in the prior off process Firstly.

Growth in new patient adds among naive patients we should be dissipate a similar benefit.

Second half of the year and.

I guess.

Just the also just noted that the the phase three limb girdle study also completed enrollment recently, he just remind us whether this is a potentially we able expanding.

Trial in what regulators might be looking forward to enable us.

Eric William Joseph: So, you know, in terms of Enplaza, obviously, we've been spending the last couple of years gathering data, and the publication, I just think is so clear in terms of demonstrating, I think the YMF plot, the data just shows that it's a superior product relative to prednisone. And I think that there's been a lot of hard work done on that to get to this point. And so I think it's growth. Maybe Eric, you want to talk a little bit about the... [inaudible] Yeah, sure, Eric. Good question.

Sure. So you know in terms of being Plaza, obviously, we've been spending the last couple of years gathering data and the publication I just think so clear in terms of demonstrating.

Why in club or is the superior data just shows that the barrier products.

Relative to the prednisone than I think that's there's been a lot of hard work done on back to to get to this point and so I think that growth maybe Eric you want to live talk little bit about though.

The growth of booked in plaza in terms of patient insights.

Yes, sure [noise], because we're really pleased with the growth right now it would seem Crowne Plaza. This is the majority of the growth right. Now is that we've been able to convert patients that have been on brokerage.

Eric Pauwels: We're really pleased with the growth right now that we've seen from Plaza. The majority of the growth right now is that we've been able to convert patients that have been on birth control and have patient assistance. We've been able to convert them much, much faster to commercial therapy, which is extremely important because that's a free drug. In addition to that, we have continually increased the number of new prescription forms during the quarter, and we saw a nice increase there, an influx of both, a nice combination of naive and former prednisone.

And have those are patient assistance.

We've been able to convert them much much faster to commercial therapy, a which is extremely important because actually drug.

In addition to that we have continually increased the number of new prescription start forms.

During the quarter, we saw nice increase there than influx of both an IDE combination of naive and former prednisone. The other thing is driving is at the base when patients are being our again like prints Warner what seems very high here [laughter] very.

Eric Pauwels: The other thing that's driving it is that the base of patients is, again, like transporters; we're seeing very high adherence, very good compliance rates, and very low dropouts. We've also come into a period of reauthorizations, and our team is staying way ahead of the reauthorization process and with the insurance companies, so that patients can spend less time in that bridge environment of free drugs and more on commercial therapy. So it's a combination of a lot of these different efficiencies that have really driven this growth, and we're seeing that again. It's looking really strong as we're moving into the second half of the year. Yep, I got it. Unknown Speaker. Thank you. Oh, the limb girdle, yeah, my recollection, I can't remember what that was, I don't know if I can remember the shape or not, but, um, yeah, uh, I do think it's a limb girdle.

Very good compliance rates at very low dropout will also come into a period of reauthorization that our team is saying a way ahead of the reality process and when the insurance company.

That patient can spend less time in that bridge environment, we drug and more on personal therapy. So it's a combination of a lot of these different efficiency, that's really driven disk this growth and we're seeing got again, it's looking real strong as as we're moving into the second half of the or.

Got it.

And on limb girdle said.

Oh, the limb girdle.

My recollection, I can't remember that well see it but yeah, Oh I think.

But that's one where I don't think we actually completed the.

Enrollment, yeah, and I think Gibson.

Eric William Joseph: But that's one where I don't think we actually completed the enrollment, um, yeah, and I think it's been, especially with Covid, it's had a substantial uh recruiting challenge. Understood. Thanks for the clarification there. Thanks for taking the questions, guys. Thank you. Our next question comes from Martin Asa of Credit Suisse. Your line is open. Hi, this is Matt Torrell on behalf of Marty.

Especially with coal business and it's been had a.

Let's say too.

Recruiting challenges.

Understood. Thanks for the clarification there.

Thanks for taking my questions that I get it.

Thank you.

Next question comes in Martin.

So credit group your line is open.

Hi, This is not tomorrow on for Marty. Thanks for taking my question regarding the Heightens program, you mentioned that phase one healthy volunteer data will include information on heightened lowering of plasma and I believe you presented some data on mice on the relationship between Huntington lowering in plasma versus the brain.

Martin Asa: Thanks for taking my question. Regarding the Huntington's program, you mentioned that phase one healthy volunteer data will include information on Huntington, lowering, and plasma. And I believe you presented some data on mice on the relationship between Huntington's protein and lowering emplasma relative to various regions of the brain. Does your therapy lower Huntington's protein in non-human primates, and if so, can you describe the relationship you saw in NHP in terms of Huntington's and lowering emplasma relative to various regions of the brain?

Does your therapy, lower hunton protein and non human primates and if so can you describe the relationship you saw and NHP in terms of Huntington, Lauren and plasma relative to various regions of the brain. Thanks a lot.

Unknown Executive: Thanks a lot. Sure. Um, yeah, so you're absolutely right where the, uh, no, it's really just to remind everyone. The Huntington's Program, which you might have seen on the Deep Dive, was that you take what we call the pseudo-intron and trick it into being able to go be part of the messenger RNA, which actually makes the RNA unstable because of a premature stop, and you don't make either the protein or the RNA.

Sure.

Yeah, So you're absolutely right where the.

It's really to remind everyone.

How many tons program, which remixing of the di di was.

That we that you take what we call the suit on him Tron.

And be able to attributed to be able to go to be part of the best since you are in a which which actually makes the ordinary unstable because of a privilege or stop and you don't make either the protein or the are in there. So that's really actually quite important.

Unknown Executive: So that's really quite important. And that's the human form of it. My recollection is that the non-human primate, because it's within the intron, doesn't have that sequence in it, so you wouldn't necessarily see that.

You mean formally.

Oh, My recollection is non human primate because it's within the im trying to both how's that sequence in them. So you wouldn't necessarily see that that's my recollection.

Are you kidding I'm not in the sense of model.

Unknown Executive: That's my recollection of that. Great, thank you so much for the questions. Right. But it is very true that in inpatients, we'll be very much able to measure in the blood the reduction of the RNA and potentially protein, especially in the multiple ascending dose, whether you have time to see the protein go down. And we do know from measurements that blood brain levels are pretty much 50-50. So, you know, what you see in the blood, we anticipate will be similar to what we see in the brain. And that was indeed the case in the animal model.

We do the stuff.

The next would look at the substrate human stuff straighten that case.

Great. Thanks, so much for the question.

Right, but it is very true, but in the in patients were very much. The it wasn't meant to imply the reduction of BRL man, and particularly protein, especially in the multiple ascending dose for the time to see the protein go down.

And we do know from.

Oh measurements that the blood.

Levels are pretty much 50, 50. So we you know what you see in blood, we anticipate won't be similar to what we see embraer and that was indeed the case in the animal model.

Unknown Executive: So it's really quite exciting. It's actually very similar, analogous to what we saw in the SMA program or what we anticipate to see. So you really have an idea from the very early stages of the program that you're on mechanism, on target, you see the effects, and then you go on to measure clinical benefits. Perfect. Thanks again. Thank you. I'm showing no further questions at this time. I would like to turn the call back over to management for any closing remarks. Ah, okay.

So it's really quite exciting is actually very similar to analogous to what we saw him yet for me program or what we anticipate the C. So you have really Oh I'm an idea from the very early stages. The program that you are on mechanism on target you see these effects.

And then you go on to measure clinical done.

Perfect. Thanks again.

Yes.

Thank you I'm showing no further questions at this time.

I was just trying to call back I would imagine for any closing remarks.

Oh, okay well.

Thank you folks for.

Staying on the call on immense asking these questions were obviously very pleased with strong performance.

Unknown Executive: Well, thank you, folks, for staying on the call and asking these questions. We're obviously very pleased with the drug's performance. I'm very proud of how the teams have continued to function in the environment that we're in. You know, as you know, we've been trying to do our part to reduce the consequences of the pandemic. You saw that and we're very excited about 299 and its potential to be part of the solution for COVID-19. We've also, as I talked about before, recently launched our internship program providing opportunities for graduates, which we call the Talent Pipeline Program, or TPP. It offers a one-year internship to provide real-world experience for graduates during the challenge period. Economic Times. All graduates, although all graduates are eligible. We've used this program to reach out to institutions and minority communities. And I'm gratified to say we have about 3,000 applications. So it's been quite successful.

This quarter.

Ross, both commercial and clinical programs I'm very proud on how the team continued to execute.

In the environment that we're going to.

As you know we've been trying to do all part to reduce the consequences that but that mix.

You saw that we're in that we're very excited about through nine magnets and its potential to be part of the solution for corporate 19.

We've also Italy as I talked about before recently Lonestar internship program providing opportunities for graduates.

Which we call the talent pipeline program or TGP.

It offers a one year internship to provide a real world experience for graduates during the challenge.

Economic times.

All graduates, although all graduates are eligible.

We use this program to reach out to institutions minority communities.

And I'm gratified to say, we approximately about a 3000 application.

So it's been quite successful so we're excited to be able to work alongside and mentor, what we hope will be future leaders in the biopharmaceutical community.

Operator: So we're excited to be able to work alongside and mentor what we hope will be future leaders in the biopharmaceutical community. So thanks again for listening, and we hope that. Ladies and gentlemen, this does conclude today's conference. Thank you for participating. You may all disconnect. Have a great day. Thank you for watching!

So thanks again for listening and we hope that once they state.

Ladies and gentlemen, distress concludes today's conference. Thank you participating you may now disconnect probably day.

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