Q1 2020 Earnings Call

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Unknown Executive: Ladies and gentlemen, thank you for standing by, and welcome to the PTC Therapeutics first quarter 2020 financial results and corporate update conference call. At this time, all participant lines are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star one on your telephone.

Unknown Executive: Please, the advice for today's conference is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to your speaker today, Alex Kane, Head of Investor Relations at PTC Therapeutics. Thank you. Please go ahead, sir.

Unknown Attendee: Good afternoon, and thank you for joining us to discuss the PTC Therapeutics first quarter 2020 corporate updates and financial results. I hope that everyone is doing well and staying safe.

[music].

Unknown Attendee: Joining me on today's call is our Chief Executive Officer, Stuart Peltz, our Chief Financial Officer, Emily Hill, as well as Matt Klein and Eric Pauwels, who were recently appointed Chief Development Officer and Chief Business Officer, respectively. 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 the call, which contains our forward-looking statements. Our actual results could materially differ from these forward-looking statements as any and all such risks can materially and adversely affect our business and results of operations. 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 and non-GAAP financial measures is available in today's earnings release. With that, let me pass the call over to our CEO, Stuart.

Stuart Peltz: Thanks, Alec. And thank you for joining us today as we provide an update on the first quarter. I hope that everybody is staying safe and healthy amid these challenging times. I'm incredibly proud of how the PTC team has responded to the COVID-19 crisis. Recognizing the seriousness of COVID-19 and acting, In late February, we understood the threat and set up a COVID-19 task force that included individuals from all areas of expertise of PTC, including physicians, public health experts, and epidemiologists who understood the implications of the viral pandemic.

Stuart Peltz: This team immediately implemented a plan to safeguard the health and safety of our employees and ensure that they had the necessary equipment to work effectively from home. This task force continues to meet and refine processes that allow us to remain productive and safe. We have a second task force that focuses on critical aspects of our business to ensure access to our therapies. This task force is continually assessing issues that could arise with our clinical programs, manufacturing, supply chain, research, as well as the commercial business. We have devised strategies to mitigate potential issues in our businesses and are continuously assessing how well they are functioning. Patient services and engagement teams are now working together to ensure that patients have the necessary access to treatment. We have also created a third COVID-19 task force whose mission is to look at what is next and think through the strategies we should employ as the world begins to open from being locked down. Their job is to strategize multiple scenarios of what will be needed to be successful in different places around the globe.

Stuart Peltz: As a result of these efforts, our teams have adapted and continued to act. Field teams are engaging with physicians, identifying patients, and driving awareness of our commercial products. Given the limited ability for personal interaction, they are doing so remotely using a number of digital approaches.

Stuart Peltz: Our scientists have also continued their work in the laboratory to drive critical research programs forward. We have put safeguards in place, including staggered shifts to allow reduced interaction so that they can work in a safe environment. As all companies in the industry have experienced, ongoing and planned clinical trials have been impacted as hospitals and healthcare providers focus on treating COVID-19 patients and have slowed or closed sites. The effects of COVID have also impacted regulatory filings in our gene therapy programs. Matt will talk more about these.

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Stuart Peltz: Overall, I'm proud of the organization's response to the COVID-19 crisis. We have a strong capital position, with more than $595 million on our balance sheet as of the end of... We're being both strategic and prudent about capital allocation. As an example, because of COVID's potential impact, we have deferred certain capital expenses at our HopeWell facility and now anticipate that GMP manufacturing of clinical material at this facility will begin in early 2021. I also want to highlight other aspects of PTC's business that keep us well positioned both during the crisis and beyond.

[noise], ladies and gentlemen, thank you for standing by and welcome to the P.T.C. Therapeutics first quarter 2020 financial results incorporate update conference call at this time of participant lines on the list.

And only mode. After the secrets presentation, there won't be a question and answer session. That's the question. During the session. You wanted to press dark one on your telephone. Please the advice of today's conference as being recorded if you require any further assistance. Please press start zero I when I like to have a conference on richer speaker today, Alex Kane had an investor relations.

P.T.C. therapeutic. Thank you. Please go ahead sir.

Good afternoon, and thank you for joining us to discuss the P.T.C. Therapeutics first quarter 2020, corporate updates and financial results I hope that everyone is doing well and staying safe.

Unknown Attendee: These include the strong team that we have in place, our global commercial infrastructure, and commercial products that can be delivered and administered to patients at home. And for the first time, SMA patients will have the potential opportunity for at-home therapy. In addition, we have a diverse rare disorder pipeline with multiple upcoming catalysts. Let me now focus on the progress we made during the first. Starting with SMA, we recently disclosed meaningful and positive results for Rizaplam in both the Sunfish Pimple Study for Type 2 and 3 SMA patients and the Firefish Pivotal Study in Type 1 esthemes. We anticipate approval with a broad label later this year. Earlier this week, along with our partners Roche and the SMA Foundation, we shared the positive 12-month results of the FireFish Part 2 Pivotal Study.

Wanting me on today's cause our Chief Executive Officer Stewart helps our Chief Financial Officer, Emily help as well as not Klein in her house, where recently appointed Chief Development Officer, and cheap business officer, respectively.

Before we start.

Me remind me that today's called will include forward looking statements based on current expectations. Please take a moment to review the slide posted on our invest relations website conjunction with the call which contains or forward looking statements are actual results could materially differ from these forward looking statements at any such risk and materially in adversely affect our business results.

Operation.

For a detailed description of clickable risks and uncertainties. We encourage you to review the company's most recent quarterly report form 10, Q. an annual report form 10, K. filed with the Securities and Exchange Commission as well as the company's other I since he finally.

We will disclose certain nongaap information during this call information regarding our use of gap and non got financial measures and a reconciliation of gap to non gap is available in today's earnings release with that let me pass the call over to our C.E.O. Stewart pulse.

Unknown Attendee: The FireFish Part 2 study results are quite exciting and show the importance of increasing SMN protein systemically, demonstrating the achievement of motor functions and developmental milestones such as the ability to roll over, sit, and stand. The study met its primary endpoint of patients sitting without support at 12 months and was highly statistically significant. 12 of 41 patients, or 29%, met the milestone with a p-value of less than 0.0001. Considering the meeting age and enrollment was 5.3 months and that these infants already had progressed disease, these results are particularly exciting. The safety data were consistent with the known safety profile of RISDA plan. Importantly...

Thinks Alex.

And thank you for joining us today as we provide an update on the first quarter I hope that everybody is staying safe and healthy amid these challenging time.

I'm incredibly proud of how the P.P.C.P. has responded to the covert 19 crisis.

Recognizing the seriousness of covert 19, and acting early and late February we understood the threat and set up a covert 19 task force that included individuals from all areas of expertise the P.P.C., including physicians public health experts and epidemiologist that understood the implications of the <unk>.

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This team immediately implemented a plan to safeguard the health and safety of our employees ensure that they had the necessary necessary equipment to work effectively from home.

Stuart Peltz: Unknown Attendee

Unknown Executive: This study also met all its key secondary objectives and This included the CHOP-IN-10, HI-ME-2, and Event-Free Survival. 90% of all infants in this study showed top in 10 improvement from baseline, with a median change from baseline of 20%. Part 2 showed even greater improvements in patients' ability to stand and walk as assessed by the HIEMI-2 than were observed in Part 1 and 12. One patient in FireFish Part 2 reached a bouncing milestone, a key component of developing the ability to walk. After 12 months of treatment with Rizdoplan, 93% of infants were alive, 85% were without permanent ventilation, and 95% of patients maintained the ability to swallow. As Dr. Servais pointed out on the Roche call, this is particularly impressive when compared to other therapies as the bulbile function benefits may reflect a small molecule's ability to reach the brain stem. In contrast, in the natural history of SMA Type 1, the median age of death or permanent ventilation is 13.5 months, and all infants with Type 1 SMA older than 12 months require feeding support.

This task force continues to meet and refine processes that allow us to remain productive unsafe.

We have a second task force that focuses on critical aspects of our business to ensure access to our therapies. This task forces continually assessing issues that could arise with our clinical programs manufacturing supply chain research as well as the commercial business.

We have device strategies to mitigate potential issues in our businesses and our continuously assessing how well they are functioning.

Patient services engagement teams, but not working together to ensure that patients at the necessary access to treatment.

We have also created the third covert 90 task force, whose mission is to look at what his neck and freak through the strategy is we should we should employ as the world begins to open from being locked down.

Their job is to strategize multiple scenarios, what will be needed to be successful in different places around the globe.

As a result of these efforts are teams have adapted and have continued to execute.

Oh teeth are engaging with the positions identifying patience and driving a weirdness of our commercial products.

Given the limited ability for personal interaction they are doing so remotely using the number of digital approaches.

Unknown Executive: Overall, it's clear that these results are highly compelling. As we communicated recently, the FDA extended the PDUFA data for RISDAPLAN to August 24th due to the submission of additional data from Sunfish Part 2. The inclusion of these data in the submission is anticipated to support broad access and reimbursement to RISDAPLAN for the widest range of SMA patients. Importantly, the FDA has identified no substantive issues to date during the review.

Our scientists have also continues to work in the laboratory to drive critical research programs forward.

We have put safeguards in place, including staggered shift to allow reduce interaction so that they can work in a safe environment.

As all companies in the industry have experienced ongoing the plant clinical trials have been impacted as hospitals and health care providers focus on pretty covert 19 patient and I've slowed or close site.

<unk> have also impacted regulatory filings in our gene therapy programs that we'll talk more about these programs.

Unknown Executive: Roche's careful preparation has ensured ample RISDA plan drug supply. Roche is working proactively with its partners in the SMA community and its logistics providers to ensure drug supply upon launch in the current COVID-19 environment. As part of Roche's pre-launch commercial efforts, several early access programs have been initiated. Earlier this year, early access programs were opened in the United States and European countries for Type 1 SMA patients. Recently, it was announced that the program is being expanded to include type 2 SMA patients. Importantly, in response to requests received from type 1 and type 2 patients whose current treatment has been interrupted as a direct consequence of the COVID-19 pandemic, Roche has decided to amend the programs to allow these patients to be able to receive RISDiplas. These requests for the RISDA plan demonstrate that a significant unmet need exists within the SMA population. The need for an oral therapy that is taken at home is shown to be even more critical for patients as they try to navigate through the COVID-19 pandemic.

Overall I'm proud of the organizations response, the corporate 19 crisis real strong capital position with more than $595 million on our balance sheet as at the end the first quarter.

Were being both strategic improved in about capital allocation.

As an example, because a covert potential impact we have different certain capital expenses that our hope well facility.

Anticipate that GMP manufacturing of clinical material at this facility will begin in early 2021.

I also want to highlight other aspects of P.D.C. business to keep us well position within the crisis and beyond. These include the strong team that we have enough place our global commercial infrastructure and commercial products that can be delivered and administered to the patients at home.

And for the first time.

Patience will have the potential opportunity for an at home therapy.

Addition, we have a diverse rare to sort of pipeline with multiple upcoming catalyst.

Let me know focus on the progress we made during the first corridor.

Starting with estimate we recently disclosed the meaningful and positive result for <unk>.

Both the SUNFISH pivotal study for type two and three estimate patients and the fire fish pivotal studying the type one estimate patience.

We anticipate in approval with a broad label later this year.

Unknown Executive: In fact, Risaplan would be the only available SMA therapy that does not require clinic visits for administration, an important concern for patients with respiratory complications. We expect that RizdiPlan will be the most competitive global product for a broad range of SMA patients and anticipate a robust launch following these indications. RIF deployment was the first compound arising from our splicing plant. One advantage of being an aura compound is that it's systemic, so that it is in the blood and can get to all affected tissues. A second advantage is the ability to measure ristoplam's pharmadynamic effect on SMN2 mRNA and SMN protein in blood or other easily accessible tissues.

Earlier this week, along with our partners <unk> Foundation, we shared the positive 12 month results of the firefighters part two pivotal study.

The fire fish part to study results are quite exciting it showed the important for recruiting.

Protein systemically, demonstrating the treatment of motor functions and developmental milestones since his ability to roll over a certain stance.

The study met his primary endpoint a patient sitting without support at 12 month and was highly statistically significant.

12, a 41 patient for 29%.

Let the milestone with a p. value of less than 0.0001 <unk>.

Considering the meeting each other enrollment was 5.3 months.

The safety data were consistent with the no safety profile over is the plan.

Importantly, this study also met all it's key secondary endpoint.

Unknown Executive: As you may recall, we successfully utilized this approach in RISDA Plan Phase 1 studies, where we demonstrated proof of concept of its activity in healthy volunteers. We plan to use this same approach in other splicing programs. PTC 518 is a development candidate from a Huntington program, and IND toxicology studies are ongoing.

This included the top 10 highly too and event free survival.

90% of all fits in this study so cop intend improvement for baseline with a median chain from baseline of 20 points.

Two showed even greater improvements in peace and stability. The standard walk it's assessed by highly too then were observed in part one to 12 months.

One patient and fire fish part to reach the bouncing milestone key component of Diveley developing the ability to walk.

Unknown Executive: Let me now turn to our commercial efforts in the first quarter. We had strong year-over-year first quarter growth with our DMD franchise, with revenues of $68 million. We are reporting $40.5 million in worldwide translander sales and $27.5 million in U.S. emplaza sales.

After 12 months of treatment with risk to plan, 93% of it since we're alive.

5% were without permanent ventilation and 95% of patience maintain the ability to swallow as doctors that they pointed out on the rose call. This is particularly impressive when compared to other therapy says the ballpark function benefits may reflect a small molecules ability to reach the brain stem.

Unknown Executive: In addition, we continue to see positive trends in the weeks following the first quarter. However, Nevertheless, we cannot predict the duration and severity of the COVID impact on our commercial business over the coming months. Therefore, until we have further understanding of the effects of COVID on our revenue, we are withdrawing our 2020 financial guidance at this time. I also want to talk about Analyst Day. It's becoming increasingly clear that hosting an Analyst Day in mid-June in New York City would not be in the best interest of the health and safety of either our employees or guests. We will postpone the meeting and switch to hosting multiple webinars in which we do deep dives into our programs and platforms. We will shortly post the date on which the first deep dive will occur. We believe this will be a productive way to update you on all our programs and platforms.

In contrast in the natural history of the I suddenly type one the meeting each of death or permanent ventilation, it's 13.5 months and all infant type witness I may older than 12 month require feeding support.

Overall, it's clear that these results are highly compelling.

As we communicated recently.

Yeah extended it to do for data for is the point to August 24th.

The the submission of additional data from SUNFISH part too.

The close you know these data into submission is anticipated to support broad access reimbursement to raise the plan for the widest range of estimate patience importantly, the F.D.A. has identified no substantive issues to date during the review.

Roaches careful preparation has the shirt ample room to plant drug supply.

Roaches work you proactively with its partner recently estimate community and this logistic providers to insert drug supply upon launched from the current covert 19 environment.

It's part of ferocious pre launch commercial efforts.

Matt: I'll now turn the call over to Matt and Eric and Emily. For Matt and Eric, we welcome them to the executive team and their first appearance on the quarterly call. Matt will now update you on the status of the clinical programs, Eric will cover more on the commercial business update, and Emily will give a financial update.

Parlor early access programs up and initiate it.

Earlier this year early access programs were open to the United States and European countries for type one estimate patients.

Recently, it was announced that the program is being expanded to include type two assembly patience.

Importantly in response to request received from type one that type two patients whose current treatment has been interrupted as a direct conflict of the covert 19 pandemic roche's decided to amend the programs to allow for these patients to people that receiver is the plan.

Matt: Thanks Kyu. I'm thrilled to join the executive management team at, and I look forward to the continued progress of our clinical programs. It's an exciting time to step into this role with our multiple upcoming clinical catalysts, even in light of the current environment. In terms of timing updates for our clinical programs, I will start with study 045, the U.S. Translunar Distributed... Due to COVID-19, the 045 study site is closed to elective procedures, which has delayed the study completion as a few patients still require final study muscle biopsy. Based on the site's current timelines, we now anticipate reporting top-line data in the third quarter.

These requests for <unk> demonstrate that a significant unmet need exists within the somebody population.

Need for an oral therapy that is taking it home.

To be even more critical for patients as try to navigate through the covert 19 pandemic.

In fact, we use the plan would be the only available estimate therapy that does not require clinic visits for administration and important concerned for patients with respiratory complications.

We expect that <unk> will be the most competitive global product for a broad range of that so many patients and anticipate a robust launch following approval.

Risk the plan was the first compound arising from our splicing platform one advantage it'd be the nor a compound instead, it's systemic so that it is in the blood and can get to all affected tissues. A second Spanish is the ability to measure is the <unk> farmers dynamic effect and I sent it to have marinade and there's some in protein in.

Matt: We expect that, in combination with our existing clinical data, statistically significant results in 045 would be sufficient for accelerated approval in the U.S. As a reminder, this is a single-site, 40-week study that enrolled 20 boys aged 2 to 7 years with nonsense mutations. The primary endpoint is percentage dystrophin change from baseline, as measured by electrochemiluminescence, or ECL. In close collaboration with the FDA, we developed and validated an ECL liquid-based assay that is highly sensitive, highly linear, and particularly well-suited to identify large proteins such as full-length dystrophins. Now, we turn to our splicing platform.

Blood or other easily accessible pitchers.

You may recall, we successfully utilize this approach and reads the plans pays one studies, where we demonstrated proof of concept of his activity.

Healthy volunteers.

We plan to use the same approach and other spicy program.

The same approach will be using P.T.C. five four and eight in Huntington disease P.T.C. Five ornate is is a development candidate for my Huntington program and a 90 toxic <unk> College you studies are ongoing.

Let me know turn to our commercial efforts in the first quarter.

Matt: Detailed the exciting results for RISDAPlant, our first small molecule from this platform. Our next splicing program in Huntington disease remains on track with initiation of clinical studies and healthy volunteers to start prior to the end. These Phase I studies will include both single and multiple ascending dose regimens in order to inform safety and pharmacokinetic parameters. We expect these studies will be relatively straightforward and support dose selection to achieve target Huntington RNA reduction levels in the range of 50%. Moving to our bio e-platform, due to COVID-19, we now expect to initiate potential PTC 743 registrational trials in refractory mitochondrial epilepsy in the third quarter and inphrasia ataxia in the fourth. We will initiate these studies once the planned study sites reopen and are available for clinical trials and, of course, once we can ensure patients can safely travel to and from study sites. The Phase 1 trial of PTC 857, which is being developed for GBA Parkinson's disease as its first indication, remains on track to start in the third quarter. As we have discussed previously, the BioE platform is complementary and synergistic with our other platforms. PTC 743 and PTC 857 target 15-lipoxygenase.

We had a strong year over year first quarter growth with our D.M.D. franchise with revenues of $68 million.

We are reporting $40.5 million at worldwide Translarnas sales and $27.5 million in U.S. in Plaza sales. In addition, we continue to see positive trends in the weeks following the first quarter.

Nevertheless, we cannot predict the duration in severity of the covert impact on our commercial business over the coming months. Therefore until we have further understanding of the effects of covert in on the revenues. We are we're drawing are 2020 financial guidance at this time.

I also want to talk about analyst state is becoming increasingly cleared that hosting it and analysts day in mid June in New York City.

Being the best interest of the health and safety of either our employees for gas.

We will postponed the meeting and switch to host be multiple webinars in which we do deep dives into our programs and platforms will be shortly before posting the data, which the first deep dive will occur. We believe this would be a productive way the update you on all our programs on platforms.

Oh now turn the call over the mat and the Eric and Emily format, and Eric We welcome them to the executive team and their first date debut on the quarterly calls.

Well now update you on the status of the clinical programs.

Eric will cover more on the commercial business update and Emily will give a financial update.

So let me know turn it over to Matt Matt.

Thanks to I'm thrilled to join the executive management team T.C. and I look forward just that continued progress at our critical programs. It's an exciting time to step into this role with our multiple upcoming cynical cat.

Even in light of the current in by him.

In terms of timing updates for clinical programs I will start with this study Oh, four or five D.U.S. transmission or district and study.

Matt: The Enzymatic Hub that Regulates the Inflammation and Oxidative Stress that Underpin Mitochondrial and CNS Disease Pathology. PTC 743 has been tested in over 400 patients, primarily in children with inherited mitochondria, and has demonstrated promising efficacy as well as favorable safety, and Expanded Access and Compassionate Use Studies in Patients with Mitochondrial Disease. PTC 743 has had a clear impact on refractory seizures, reducing the number of patient seizures, as well as seizure-related morbidity, including hospitalization. In addition, in a Phase II trial on Friedrich ataxia, PTC 743 demonstrated a statistically significant impact on disease severity at 24 months. Relative Age and Stage-Matched Natural History Controls as Assessed by the Validated Forest, For PTC 857, the compelling preclinical data and its target mechanism of action strongly support its development for Parkinson's disease. The target of 15-lipoxygenase is a very important upstream modulator of multiple pathways known to be key to Parkinson's disease pathogenesis, including alpha-synuclein oxidation and aggregation and microglial activation, a driving factor in neuroinflammation.

Due to covert 19, the O. four or five study site is closed to elective procedures, which is the latest study completion as a few patients still require final study muscle biopsy.

Based on the sites current timeline as we now anticipate reporting topoint eating the third <unk>.

We expect that in combination with our existing clinical data statistically significant results you know core five would be sufficient for accelerated approval in the U.S.

As a reminder, this is a single site 40 weeks study that are old 20 boys H. 227 years pick nonsense mutation.

The primary endpoint is the percentage district can change from baseline as measured by electric.

<unk>.

Close collaboration with the F.D.A., we developed invalidated U.C.L. liquid based essay that is highly sensitive.

Highly linear and particularly well suited to identify large proteins such as bowling.

Turning out to our spicing platform stewed detailed the exciting results were raised a plant.

Small molecule from this platform.

Next placing program in Huntington disease remains on track with initiation of clinical studies healthy volunteers to start prior to the end.

These fees. One studies will include both single and multiple of sending dose regimens in order to inform safety and farmer kinetic parameters.

We expect these studies will be relatively straightforward and support dose selection to achieve target Huntington already reduction levels in the range of 50 per cent.

Matt: Therefore, targeting 15-lipoxygenase allows us to simultaneously affect multiple pathways, while many current therapies in development target only one of these pathways. Moving on to our gene therapy platform and associated updates, as Sue noted, COVID-19 has impacted our gene therapy program timeline. With respect to the Friedrich ataxia and Angelman syndrome gene therapy programs, COVID-19 has impacted multiple IND-enabling activities.

Shifting <unk> he platform due to Kobe 19, we now expect to initiate potential P.P.C. 743, registrational trials and refractory mitochondrial epilepsy, and the third quarter and and feature attacks yet in the fourth quarter.

We will initiate these studies wants to plan studies sites reopened and are available for clinical trials and of course once we can ensure patients can safely travel to win from studies sites.

Matt: We currently anticipate the IND filings will be delayed at least one quarter, and we plan to provide an update on filing timing as we better understand the ultimate impact of COVID-19 on these programs. Moving on to our AADC deficiency program. Manufacturing activities are continuing and remain on track. The gating factor for the BLA submission remains the study of the use of the commercial cannula in young patients. These treatment procedures have been delayed by hospital cancellations of elective procedures due to COVID-19. Therefore, we now expect the BLA submission for AADC deficiency to the FDA will be in the second half of 2020. As a reminder, the MAA for the AADC Deficiency Program was submitted to the EMA in January.

The phase one trial of P.T.C. 857, which is being developed for G.B.A. Parkinson's disease as its first indication remains on track to start in the third quarter.

As we have discussed previously the bio we platform is complimentary 10 synergistic with our other platforms.

P P.C. seven for three and P.T.C. 857 target 15 my <unk>.

Enzymatic hub that regulates the inflammation in oxidative stress and underpaid mitochondrial and C.N.S. disease pathology.

P. P.C. 743 has been tested in over 400 patients primarily and children with inherited mitochondrial because it has demonstrated promising advocacy as well as favorable safety signals.

Expanded accessing compassionate use studies in patients with mitochondrial C.'s P.P.C. 743 has had a clear impact on refractories seizures, reducing the number of patients seizures as well as seizure related morbidity, including hospitalization.

Matt: And timelines remain in place for the CHMP final opinion by the end of the year. Overall, despite COVID-19-related delays, we still expect a number of exciting and impactful clinical catalysts in the coming years. I will now pass the call to Eric Pauwels to provide an update on the commercial business. Thanks.

In addition in a phase two trial or free took a taxi P.P.C. 743 demonstrated statistically significant impact on disease severity at 24 months.

<unk> age and stage match natural history controls as assessed by the validated far score.

For P.T.C. 857, the compelling pre clinical data and its target and mechanism of action strongly support its development for Parkinson's disease.

Eric Pauwels: To echo Matt's comments, I am delighted to join the executive management team at PTC. With a strong first quarter behind us and important upcoming milestones this year, we will continue to build on our existing portfolio of revenue-generating products, prepare for future launches, and remain selective with business development opportunities. Currently, on the commercial side of the business, our top priority is ensuring that patients on treatment have the necessary drug supply, and we have not seen any significant disruptions today. With personal promotion from our sales team worldwide limited by COVID-19, we continue to engage with health care providers, Patient Advocacy Groups, and Payers by driving DMD disease awareness activities through virtual calls, DMV Masterclass Webinars, and Educational Podcasts, as well as leveraging virtual platforms to host local advisory boards.

They targeted 15 like <unk> seven very important upstream modulator of multiple pathways known to be key to Parkinson's disease path or Genesis, including office, new clean oxidation aggregation and microbial activation driving factor in there right in formation.

For targeting 15, my <unk> allows us to simultaneously effect multiple pathways, while many current therapies in development target only one of these pathways.

Moving onto our gene therapy platform and associated updates as you noted covert 19 has impacted our gene therapy program timeline.

With respect to the free jerk attacks, yet an ancient syndrome cheap therapy programs corporate 19 hasn't packet multiple.

Willing activities.

We currently anticipate the.

Eric Pauwels: Additionally... Social media is playing an even more important role in the current environment, especially for DMD patients seeking information about our products. We had a solid first quarter with TransLarna and Inflaza, both in terms of maintaining our existing patients and in new patient growth. New patient growth was driven in part by diagnostic and educational efforts over the last several months, and we continue to see these efforts positively impacting the business into the second quarter. For TransLarna, we saw continued growth in Q1 and positive trends both in diagnosis and prescriptions globally. In the EMEA region, we have successfully maintained adherence and compliance with patients on TransBarna and have seen minimal disruption. In the LATAM region, we continue to have ongoing discussions with the Ministry of Health in Brazil to secure future purchase orders, and we have seen the number of positive injunctions that will allow patient access to increase. Importantly, we continue to highlight comparisons of the impressive real-world results from the Stride Registry and Synergy DMD Natural History Study, highlighting the long-term efficacy of TransLarner.

Filings will be delayed at least one quarter and we plan to provide an update and finally timing as we better understand the ultimate impact of covert 19 on these programs.

Moving to R.A.D.C. deficiency program manufacturing activities are continuing Ed remain on track that gaiting factor for the B.L.A. submission remains the study of the use of the commercial Cannulate haste. These treat procedures have been delayed it by hospital cancellations of elective procedures to to cope with 19.

Therefore, we now expected B.L.A. submission for H.P.C. deficiency to the F.D. I will be in a second half of Twentytwenty.

As a reminder, the anyway for the A.D.C. deficiency program was submitted to the E.M.A. January.

Timelines remain in place for the C.H.M.P. final opinion by the end of the year.

Overall, despite Kobe 19 related delays, we still expect number of exciting and impactful clinical catalyst in a company months.

Now passed the call to Eric Pals to provide an update on the commercial business.

Thanks.

<unk> comments I am delighted to join the executive management team at P.G.C.

With a strong first quarter behind us.

Important upcoming milestones. This year, we will continue to build on our existing portfolio of revenue generating products prepare for future watches and remain selected with business development opportunities.

Currently on the commercial side at the business our top priority is ensuring that patients on treatments have the necessary drug supply.

And we have not seen significant disruptions today.

With personal promotion from our sales team worldwide limited by covert 19.

Eric Pauwels: Forum Plaza, our U.S. commercial and PTC CARES case management team, has been fully engaged and made a significant impact in the first quarter of 2020, Thriving Enhanced Customer Service and Improving Efficiencies with New Patient Starts. Reauthorization and Patient Adherence and Compliance. We expect these ongoing improvements will continue through the next quarter. In the first quarter of this year, patients moved faster from time of new prescriptions to Commercial Therapy, and patients previously on bridge therapy and patient assistance programs transitioned to commercial therapy more rapidly. In addition, physicians continue to see the differentiated benefits of Implaza, as evidenced by multiple publications, including recent data from Cincinnati Children's Hospital real-world outcomes demonstrating MFLAZA's benefits, such as delaying loss of ambulation, better compared to pregnant women. Now, let's turn to Tech Steady.

We continue to engage with health care providers.

Patient advocacy groups and pairs by driving D.M.D. that he's awareness activities true virtual call.

The Master class Webinars.

And educational podcast as well as leveraging virtual platforms to host local advisory boards.

Additionally.

Social media is playing and even important role in in the current environment, especially for D.M.D. patient seeking information about our products.

We had a solid first quarter with trends liner and and plaza, both in terms of maintaining our existing patients and in new patient grow up.

New patient growth was driven in part by diagnostic an educational efforts over the last several months and we continue to see these efforts positively impacting the business into the second quarter.

Fortran, Florida, we talked continued growth in Q1 and positive trends, both in diagnosis and prescriptions globally.

In the E.M.B.A. region.

We have successfully maintain it here in compliance with patients are trying to Florida and have seen minimal disruption.

In the last time region, we continue to have ongoing discussions with the ministry of health in Brazil to secure future purchase orders.

Eric Pauwels: Our launch activities are still progressing, despite the current environment, with our focus now on pricing discussions in Brazil, which we expect to be completed in the second quarter. TXETI is well different, as the first and only approved at-home therapy in Brazil with slowing disease progression and quality of life indicated in the label. In the first quarter, we saw continued growth in new patient identification and prescriptions in LATAM. As a reminder, Brazil has the largest prevalence of HATTR-amylodosis polyneuropathy patients worldwide.

Then we have seen the number of positive injunctions that will allow patient access to increase.

Importantly, we continue to highlight comparison of the impressive real world results from the stride registry and synergy D.M.D. natural history study highlighting the long term African see I've trained Florida.

Foreign Plaza R.U.S. commercial and P.T. He cares case management team has been fully engaged and made significant impact in the first quarter of 2020.

Having enhanced customer service and improving efficiency mutation starts.

Eric Pauwels: Building on our core capabilities in LATAM, we anticipate a WeLIVRA filing with INVISA in the second half of this year, and we continue to identify new FCS patients for WeLIVRA therapy. Turning to AADC deficiency, pre-launch activities continue to progress. We have adapted to the current environment by implementing virtual education and patient finding initiatives, including conducting multiple masterclasses with over 200 health care providers in attendance from over 20 countries. We also rolled out a program called the Road Less Traveled for finding a path toward early patient diagnosis. We've also held multiple European steering committee meetings. We recently launched social media campaigns utilizing expert videos focusing on symptoms and directing viewers to disease state websites. Finally, patients continue to be identified as our teams push forward with virtual healthcare provider meetings to diagnose new patients in cerebral palsy and epilepsy clinics.

Optimization.

Annotation adherents in compliance.

We expect these ongoing improvements will continue to the next quarter.

In the first quarter of this year patients move faster from time of new prescription.

It's a commercial therapy and patients previously on bridge therapy, and patient assistance programs transition to commercial therapy more rapidly.

In addition, physicians continue to see the differentiated benefits and then plaza continued by multiple.

Publications.

Including the recent data from Cincinnati Children's Hospital real world outcomes, demonstrating in plazas benefit.

The laying lost a baby elation better compared to pregnant so.

Now, let's turn to take Saturday.

Our lunch activities are still progressing despite the current environment with our focus now on pricing discussions in Brazil.

Which we expect to be completed in the second quarter.

Text study as well differentiated as it first and only approved at home therapy in Brazil.

With slowing disease progression and quality of life indicated in the label.

Eric Pauwels: Looking back at the quarter, the home-based administration of our commercial products allowed us to maintain our current base of patients and add new patients, even as the COVID-19 pandemic started to take hold. As a result, in the near term, we anticipate continuity of supply to the existing base of patients. Some continued growth with new patients for both translarna and influenza and continued operational improvement. I will now hand the call over to our Chief Financial Officer, Emily Hill, to review our financial progress.

And the first quarter, we saw continued growth in new patient identification and prescriptions in Latin.

As a reminder, Brazil has the largest prevalence H.H.T.T.R.M. <unk> worldwide.

Building on our core capabilities and watch EM, we anticipate any way libra filing within visa in the second half of this year and continue to identify new S.T.S. stations for why Labour therapy.

Turning to 80 C. deficiency pre launch activities continue to progress we have adapted to the current environment by implementing virtual education and patient finding initiatives.

Including conducting multiple master classes with over 200 health care providers.

In attendance from over 20 countries.

Emily Luisa Hill: Thanks, Eric, and congratulations to Eric and Matt for their promotions. We are happy to have you on the MNIC.

We also ruled out a program called the road less traveled for finding a path towards early patient diagnosis. We've also held multiple European.

Emily Luisa Hill: We're also happy to have reported such a strong first quarter, with our DMD franchise growing 28% year-over-year, and with the positive tailwinds from the first quarter continuing into April. We feel well-prepared to handle the current environment, as we have highlighted this afternoon, but due to lingering uncertainty regarding the duration and the degree of the impact of COVID-19, we are withdrawing financial guidance for 2020. As you have heard reflected throughout today's call, we have given a great deal of thought to costs, timelines, and prioritizations throughout the COVID-19 pandemic. We're fortunate to have a portfolio of home-administered commercial products, diverse assets across multiple platforms, and the ability to shift resources as needed. Strategically, we have identified trade-offs where we can push forward certain programs and be more conservative with capital-intensive programs, such as deferring some capital expenditures for our HOPO Biologics Manufacturing Facility for future commercial products.

Committee meeting.

Recently launched social media campaigns use alighted utilizing expert videos focusing on symptoms.

And directing viewers too Dizzy state website.

Finally patients continued to be identified as our teams push forward with virtual healthcare provider meetings to diagnose new patients in cerebral palsy epilepsy clinic.

Looking back at the quarter the home based administration of our commercial products allowed us to maintain our current base of patience and add new patients even as the covert 19 pandemic started to take home.

As a result, the near in the near term, we anticipate continuity of supplied to the existing base of patients. Some continued growth with new patients for both trend, Florida, Adam Plaza and continued operational improvements.

I will now hand to call over to our Chief Financial Officer, Emily Hill to review our financial progress.

Thanks.

Yeah.

We are happy to have you on the management team are also happy different point of such a strong first quarter with Turkey M.D. fantastic.

Per cent you're over here.

Calvin from the first quarter continuing to April we feel well prepared to handle the current environment.

Like you to lingering uncertainty regarding the duration of the degree.

19, withdrawing financial guidance for 2020.

Emily Luisa Hill: This doesn't impact our production of near-term commercial and clinical programs such as AADC deficiency and Friedrich-Attack-Seazine therapy but allows us to conserve some cash as we move through this uncertain time. Now, I want to take a few minutes to highlight our first quarter 2020 financial results. The press release issued earlier this afternoon summarizes the details of our first quarter 2020 financial results, and I will review these details. Starting with our top-line results, we reported $68.3 million in total revenues in the first quarter of 2020, compared to total revenues of $53.6 million for the first quarter of 2019. TransLarna Net Product revenues were $40.5 million for the quarter. This compares to $35.3 million for the first quarter of 2019. Growth was driven by an increase in net product sales in existing markets, as well as continued geographic expansion into new territories. For FWASA, we reported net product revenues of $27.5 million for the first quarter of 2020, compared to $17.8 million for the first quarter of 2019. Growth was driven by an increase in net product sales to implement the operational improvements and efficiencies noted by Eric earlier.

I get heard reflected throughout the day, given a great deal thoughts of course timeline.

Throughout the 1910 buttons.

Unfortunately, I have a portfolio.

So products diverse.

Across multiple top ones on the ability to shift resources as needed.

Strategically being identified sneak us where we can push forward certain programs and be more conservative capital intensive program.

Different somehow and to transfer our hotel biologics manufacturing facility for future commercial product.

It doesn't impact are near term commercial clinical programs will just H.D.C. deficiency precheck attacking therapy.

I'm sorry.

Through this on certain time.

And I want to take a few minutes highly to first order 2020.

The press release issued earlier this afternoon summarizes the details of our first quarter 2020 financial resources and I will review these details.

Starting with our top line myself, we recorded 68.3 million and total revenues and the first quarter 2020 compared to total revenues of 53.6 million for the first quarter of 2019.

Chance.

40.5 million for the Florida, it's compared to 35.3 million for the first quarter of 19.

By an angry from that.

15, Mark.

Continue to.

And into new territory.

For the plot that we recorded not product revenues of 27.5 million for the first part of 2020 compared to 17.

Okay.

Growth is driven by an increase in that.

Okay and parts the operation prison and efficiency noted by Eric earlier.

No.

Emily Luisa Hill: Non-GAAP R&D expenses were $81.9 million for the first quarter of 2020, excluding $8.2 million in non-cash stock-based compensation expense, compared to $47.9 million for the first quarter of 2019, excluding $4.7 million in non-cash stock-based compensation expense. The increase in R&D expenses reflects costs associated with advancing the gene therapy and BioE platforms and increased investment in research programs, as well as advancement of the clinical pipeline. Non-GAAP SG&A expenses were $51.2 million for the first quarter of 2020, excluding $7 million in non-cash stock-based compensation expense, compared to $36 million for the first quarter of 2019, excluding $4.6 million in non-cash stock-based compensation expense, reflecting continued investment to support our commercial activities, including our expanding commercial portfolio. The net loss was $112.7 million for the first quarter of 2020 compared to a net loss of $72.1 million for the first quarter of 2019.

Sentence, where 81.9 million for the first quarter 2020, flooding 8.2 million and non stop based compensation.

Compared to 47.9 million for the first quarter 2019, excluding 4.7 million in on cash compensation.

The increase in R. and D.C.

See I did with advancing the gene therapy and by Lisa increased investment in research program.

I find.

Non gap.

Chances for 51.2 million for the first quarter of 2020, excluding 7 million a non stop based compensation.

Compared to 36 million for the first quarter 2019, excluding 4.6 million or not.

Confrontation.

Reflecting continued investment to support our commercial activities, including standing commercial portfolio.

Matlock was 112.7.

First quarter 2020, compared to that lots of 72.1 million for the first quarter 2019.

Emily Luisa Hill: Cash, cash equivalents, and marketable securities totaled $595.9 million at March 31, 2020 compared to $686.6 million at December 31, 2019. Before I wrap up, I wanted to share the details of a transaction we completed this week with the former shareholders of Agilid that removed liabilities from our balance. As you are probably aware, as part of our acquisition of the GeneTherapy platform, we owe Jill a two-share cash milestone. We are pleased to have exchanged a subset of those cash milestones for cash and PTC stocks. The future milestones include 40 million in time-based milestones that were owed in August 2020, and 185 cash milestones we expect to pay in 2021 upon the approval of the AADC Deficiency BLA and the receipt of a Priority Review Voucher.

Classical billing and marketable securities totaled 595.9 million March 31st 2020, compared to 680 669 at December 31st 2019.

<unk>.

Wanted to share the details the whole transaction, we compete at this week with a former shareholders eventually.

Remove liabilities for my balance sheet.

You are probably aware as part of her acquisition at the gene therapy.

Ridiculous teacher cash amount so.

Yeah.

Changed some subset of those pass mild sounds for cash from P.G.C. stop.

The future milestone, including 40 million.

<unk>.

Oh, that's 2020 and 185 half Smile John.

2021 upon the equivalent of the A.B.C. deficiency, B.L.A. and never see as a priority review voucher.

Emily Luisa Hill: Almost 95% of former Agilis shareholders elected to exchange the 2021 milestones for approximately 2.8 million shares of PTC stock and an early receipt of the 2020 CAF. This alleviates PTC's significant cash liabilities, especially in 2021, while we advance our gene therapy platform. I will now hand the call over to the operator to start our question and answer session. Operator? As a reminder, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Please stand by while we compile the Q&A roster. Our first question comes from the line of Brian Abrahams from RBC Capital Markets. Your line is now open.

Almost 95% of.

Shareholder selected to exchange 2021 milestones for approximately 2.8 million shares her P.T.P. stuff and an early risky the 2020 cash.

Alleviate people see significant hash liability, especially in 2021, while we advance our gene therapy platform.

I will now all over to the operator beside our question answer session.

<unk>.

As a reminder, task a question you only depressed star one on your telephone to withdraw your question press the pound key please stand by well, we can have a two and a roster.

Our first question comes from the line of Brian Abraham from our B.C. capital markets. Your line is now <unk>.

This is a bird over Brian Tonight.

Unknown Attendee: Hi, this is Burt. I'm here for Brian tonight. Congratulations on all the progress and your handling of the COVID situation. I just wonder if you could talk a little bit about how much progress you've been able to make on the additional study.

<unk> all the progress.

Because the situation I just wonder if you could talk a little bit about how much progress you've been able to make on the additional study of A.D.C. gene therapy.

Unknown Attendee: AADC gene therapy

Unknown Attendee: and Catheter Combination prior to the pandemic, and then I guess what types of data I can get from that.

Combination prior to the pandemic and then I guess what types of.

Unknown Attendee: From that study, do you need to...

Data from that study do you need.

To complete the filing.

Unknown Attendee: Thanks for that!

Yeah.

Unknown Executive: Next question. So yeah, we were making progress with the ANDC clinical trial. Matt, why don't you actually talk through what you were doing, what we were doing, and the next steps?

Question. So yeah, so we were making progress.

Do you see clinical trial met why don't you actually talk for what you were do what we were doing then the next step.

Matt: Thanks, Stu, and thank you for the question. As you mentioned, towards the end of 2019, we received feedback from the FDA on additional data around the use of the intended commercial cannula in young patients. As a reminder, the clinical efficacy of our AADC deficiency program is quite strong. We've demonstrated durable impact with five years of continued effect as well as evidence of continued benefit out to 10 years, and we, of course, have a strong safety profile. Manufacturing also remains on track, and we were able to submit the MAA to the EMA in January. Thus, the key gating item for the BLA was the treatment of young children with the commercial cannula. We had identified two patients that we were prepared to treat before the end of the first quarter, but due to the hospital closure for elective surgeries, those procedures have been pushed back, likely to be done sometime towards the end of Q2, possibly early Q3. And so once we have those surgeries and the data from that, we'll be able to continue to prepare for a BLA submission.

Yep. Thanks to thank you for the question.

As you mentioned, we towards the end of 2019, we had received feedback from the F.D.A. additional data around the use of the intended commercial cannulate young patients.

S reminder, clinical advocacy.

Are 80, C. deficiency program is quite strong we've demonstrated terrible impact with five years continue to affect as well as I have it ends up continued benefit out to 10 years and we of course have it.

Strong C.P. profile manufacturing also remains on track and we were able to submit the M.H.T.M. in January and so the key eating items to the P.L.A. was the treatment of young children with the commercial <unk>. We had identified two patients that we were prepared to treat before the end of the first quarter.

And due to the hospital closure for elective surgeries those procedures have been pushed back likely to be done some times towards the end of Q. Q2, possibly early three and so once we had those surgeries and the data from that you'll be able to continue to have preparations for B.L.A.'s.

Unknown Executive: [inaudible] just having a just, you know, the pandemic fell upon us so that you just take a little time, to move forward.

<unk>.

So we make pretty great.

<unk>.

You know the pandemic fell apart so that it sounds like a little time, but.

The move forward, though.

Great. Thank you so much.

Unknown Executive: Great! Thank you so much.

Our next question concerning the line of leafy a young from Cantor. Your line is now open.

Unknown Attendee: Our next question comes from the line of Alicia Young from Cantor. Your line is now open.

Oh, Hi, there <unk> funny.

Unknown Attendee: Hi there, this is Leon on behalf of Lydia. Thanks for taking our questions. I guess the first one is on COVID-19. I'm just wondering, are you seeing any changes in buying patents for TransLana, and then how do you manage to keep access for patients? And then second, for the FireFish data, how do you think a risk plan matches up to Spinraza and Zalgezma in Type 1 patients? And then for the SoundFish data, is there any plan to present subgroup analysis, particularly around age, from the data?

Question I guess the first one it's I'm copying 19, I'm just wondering I think any changes in buying patterns for transplant and how to manage to keep the access for patients and then second for the fire fished data how do you think mix and match it's up to.

With being brought that Dodger Martin type one patient and then for the South finished data if any Quinn fuel presents subgroup analysis <unk> around h. from the data fat. Thanks.

Unknown Executive: Okay, great. Well, thank you.

Okay, great. Thank you so the.

Unknown Executive: All right. Well, thank you.

Maybe we'll start with the first one would soon.

Eric Pauwels: Okay, maybe we'll start with the first one, which is on TransLargo, and for that matter, on Plaza as well. So I think you can see from the numbers that we had a good first quarter, and I think as both Eric and Emily talked, we saw the numbers continuing on. So the distribution remains ongoing, and I think we feel patients have been able to be in order. Eric, maybe you want to talk a little bit about... How do you see what's coming up for the next quarter or two?

<unk> for that matter <unk> well.

I think you can see from the the numbers, we hadn't actually a good first quarter and I think.

Eric and number when you talk to where you are in we saw the numbers continually non so the distribution remain ongoing and I think we feel patient have been been able to come in the order Eric maybe you want to talk a little bit above.

How do you see what's coming up in Oh for the next quarter to.

Eric Pauwels: Yeah, sure, Stu. And thanks for the question. You know, we're seeing continued growth in the number of patients on both products, with DMV, Translata, and Implaza. And we haven't actually seen any decline in orders. But actually, what happened is that we've actually been able to establish even deeper relationships with our healthcare providers and, in some cases, payers, and advocacy groups during the pandemic. That may tick up a little bit, but it's not very significant.

Yeah sure still enough. Thanks for the question.

You know, we're seeing continued growth in the number of patients are on both products. It with D.M.D. <unk> Plaza, and we haven't seen any actually declining orders.

But actually what happens is this we've actually been able to establish even deeper relationships with our health care providers and in some cases payers and advocacy groups during during the pandemic.

I think our main focus right now is really to provide that current base of patients we have on and plaza trends Lorna a continuity of supply and we haven't seen any major up six it's a question centered around our people asking for multiple months there'd been a few patients out of out for now.

Eric Pauwels: But most importantly, we've been able to really establish our case management team in the U.S. We've been able to establish a strong sort of connection with many of the DMV patients to ensure that they have all the resources they need, and we currently have adequate supply and assure them that we have an adequate supply of both Implaza and Translata.

Supplies of and Plaza, but generally the vast majority of patients have been getting monthly supplies in the quarter that may take up a little bit, but it's not very significant but most importantly, we'd better.

Unknown Executive: Yeah. And so the second question relates to RISD's plan and, in a sense, contextualizes how we feel the risk plan will do relative to the other products out there.

Establish our case management team in the U.S. been able to establish a strong sort of connection with many of the D.M.D. patience to ensure that they have all the resources they need and we currently have adequate and then sure sure them that we have adequate supplies both in plaza.

<unk>.

Yeah.

Unknown Executive: This is really our contention.

So the second question relates to refer to play on the end of the buttons so detection alive.

Unknown Executive: RISD Appliance is the best-in-class molecule with efficacy potential, and we think, you know, from the recent firefish data Part 2 but also Part 1, that this was compelling, compelling efficacy that was seen in that and that occurred even despite the older age when you do comparisons of that, and that we not only did we see that, but we were also able to show durable increases in the SMN protein and changes in the I think we're the only ones who've been able to show it. And not only did we show that, but it was in the periphery as well.

How we feel clambering through a relative to the other products are out there and.

It's really our contention that <unk> I moved the best in class.

Molecule with African potential and we I think you know what you could see from the recent firefighters data both part two but as well part one set of was compelling compelling nothing to do that was seen in that and that occurred even the despite.

The older age when you do comparisons of that and that.

Before we not only did we see that but we're eight ball. So so durable increases and we have some in protein and changes in the r. and they both in the C.N.S. as well as the periphery.

So that's actually really critical I think where the only ones have been able to show.

That we're not only we showed that oh, but it wasn't the periphery and we you know not as much as been talked about how.

Unknown Attendee: And not as much has been talked about how SMA, but it's becoming clearer and clearer, is a whole body disease. While the CNS is critical, it also affects other tissues as well. And then we think another advantage is that it's really the resupply itself that is the treatment. You don't need an interstitial injection for that, nor do you need to take immunosuppression as a consequence of taking the drug. So we think that's really a major advantage. And then we also think that, so far, that's what we've seen.

But it's becoming clearer and clearer as a whole body disease, well see enough critical it also affects other tissues as well and then we took another advantage is that it's really the requirements. So that is the treatment there you don't need the.

Action for that nor do you need to take Immunosuppressant as the consequences of taking the drug. So we think that's really a major advantage and then we also think that so far.

We think that they went back there was no drug related thinking finding and that you know more very proud that we have the <unk>.

Clinical trial program.

Thus far in premium patient enough that my one two and three patients from 225 years old where we've had placebo controlled.

Unknown Attendee: Unknown Attendee.

Unknown Speaker: Unknown Speaker, Unknown Attendee, Unknown Speaker, Unknown Attendee, Unknown Speaker, And then it's really just, it's not approved for all populations, and there's limited data in patients greater than two years old. So, take that and we even think when you compare it to methanursin where, um, where the burden of fecal administration is more difficult in patients, say, with scoliosis, uh, and then you have, you know, continuous accumulation of lumbar punctures.

Trials, there and we also have agreed symptomatic.

Older adults, so I'm not used to treat it. So we really have a a breath of program of the demonstrate the Africa. The on button as shown that both in their own people controlled bumpers trials as well as.

Brooklyn trials and when you can track but.

Oh Gee therapy.

We think that there's little he'll be gone for years in terms of durability.

And even that.

Centrally compounded by the follow on therapy.

The components drug did what I keep pace and well we think that were more safety concerns in terms of both of Liberty Jerry warning systemic corticosteroid button.

Oh, and then you need to be on them, you know suppressed for greater than two months.

And then it's really just it's not approve.

Unknown Speaker: and there have been meningitis and hydrocephalus that's been observed on the safety front and that there's a lack of trial data in patients greater than nine years old. But we think if you take all that into consideration and look at what RISDaplane has done in the whole patient population, again, we think it's the best in class knowledge.

Oh for all populations limit the data.

<unk> 10 years old.

So.

Taking that and we even think when compared to nothing nurse and where where the important enough when the speaker administration more difficult than patients they were going almost.

And then you'll <unk>, you know consumerism stimulation lumbar puncture.

And there have been <unk>.

On the safety of front end up as well.

Lack of trial data in patients grinning nine years old we think if you take all that into consideration and look at what a plane has done in the whole patient population again, we think it's the best in class smaller.

Unknown Speaker: [inaudible]

Unknown Speaker: Yeah, that, you know. I'm sure we'll be having additional posters over time on the data as it accumulates.

And.

One other question.

Sound fish <unk> yeah.

You know I'm sure, we'll be having a decision no poster overtime on I'm the data that accumulates.

Unknown Speaker: Okay, great. Thanks.

Unknown Attendee: Thank you. Our next question comes from the line of Taveen Ahmed from Bank of America. Your line is now open.

Okay.

Thank you all our next question cancer line up to being Ahmed from Bank of America. Your line is now then.

Hi, Good afternoon, guys think so much for taking my question, maybe one on on A.B.C. and then I have a couple more a couple of other programs up there in the past few had been starting forgive us updates on number of patients that you've been able to identify can you give us.

Unknown Attendee: Hi, good afternoon guys. Thanks so much for taking the time to answer my questions. Maybe one on AABC, and then I have a couple more on a couple of other programs. Sue, in the past, you have been starting to give us updates on the number of patients that you've been able to identify. Can you give us a refresh number on how many patients you have as of today? And also whether you still think that they're around, let's say 6,000 patients worldwide. That you hope to find over the course of the next several years.

Refresh number on how many patients you have as of today and also whether you're thinking so is that they're around mistake at 6000 patients worldwide.

That you hope to find over the course of my next several years.

Yeah, great. Thanks for that yeah. So.

Unknown Executive: Yeah, great. Thanks for that. Yeah, so I'd say that, as we said, in January, we had about 200 of greater than 200 patients identified at the JP Morgan meeting.

I'd say that if we as we said in January we had about 200, a greater than 200 pesos.

Identified J.P. Morgan dealing with continuing to search we obviously.

Unknown Executive: We continue to search. We obviously have some issues as a consequence of COVID, and I'll have Eric talk a bit about that. But we're still comfortable with the numbers that we have.

Have some issues than the consequence of Cold Britain, then I'll have a error talk a bit about that but we're still a comfortable with the numbers that we said that we think there and now it's really matter.

Unknown Executive: Transcripts provided by Transcription Outsourcing, LLC.

Unknown Executive: And now it's really a matter of patient identification, and to do so right now in the COVID environment is obviously more difficult than before. But we're still working hard at that. Eric, maybe you could talk a little bit about how we're doing this in the current environment.

Application and could do so in the right now in the covert environment is obviously more difficult than previous but we're still working hard that that Eric made me want to talk a little bit about how we're doing this in the current environment.

Eric Pauwels: Sure. Hi Tazeen. I think right now it's pretty safe to say that, you know, the COVID environment has had a direct impact on patient identification efforts, but we're doing a lot of activities right now and adapting to this new environment. As Stu said, we've identified more than 200 patients at this point, and our goal is to actually get 300 patients identified by the first country launch, whether that's in the US or Europe. We had a number of different initiatives, particularly in Q1, and when things started to slow down a little bit, we shifted, and we increased our web presence and online efforts substantially. We had a master class with about 200 healthcare provider physicians in 20 countries.

Sure.

<unk>.

I think right now it's pretty safe to say that you know the covered environment is how to direct impact on patient identification efforts, but we're doing a lot of activities right now and dappy to this new environment.

As you said you know we've identified more to 200 patients at this point in our goal is to actually get 300 patients identified by the first five country launch whether that's in the U.S. or Europe, we have a had a number of different initiatives, particularly q. wine and when things started to slow down low bit we shifted.

And we increased our web presence in the online effort substantially we had a master class that had about 200 healthcare provider positions in 20 countries. We rolled out. This program that was very very well she called on the road less traveled in it and it it really help sort of healthcare providers.

Eric Pauwels: We rolled out this program that was very well-received called The Road Less Traveled, and it really helped healthcare providers to understand how to diagnose ADD deficiency patients earlier, as well as recognize various symptoms, particularly in areas where we're trying to target right now, which are cerebral palsy clinics and epilepsy clinics. In addition to that, we've actually rolled out quite a few of the European AADC Steering Committee meetings, and we held quite a number of those and had surprisingly really good reception from key opinion leaders in these virtual education classes to help with patient identification. Now, with all that said, it's clear that COVID-19 has had an impact, given that fewer patients are seeing physicians and tests are being done, but we're still doing quite a bit of activity virtually to keep up the educational noise level.

Understand how to diagnose 80, c. deficiency patients earlier as well as recognize very symptoms, particularly in areas, where we're trying to target right now just cerebral palsy clinics Annapolis epilepsy clinics. In addition to that we've actually had rolled out quite a few of the European Agency steering Committee meetings.

And we executed quite a number of doesn't have surprisingly really good.

<unk>.

The opinion leaders virtual education classes.

Help.

With patient identification now with all it said, it's clear that covered 19 has had it as it had an impact given that patient patient fewer patients are seeing position. Some tests are being done by we're still doing quite a bit of activity virtually to keep keep Buffy educational noise level.

Okay. Thanks for that Hmm and then maybe it's still a question on Chancellor now for the study that you're doing to measure dystrophin can you get an idea of what percent of patients have not yet we feed that biopsy and does it make it any meaningful differences.

Unknown Attendee: Okay, thanks for that. And then maybe another question on TransLarna. For the study that you're doing to measure dystrophin, can you give us an idea of what percent of patients have not yet received that biopsy? And does it make any meaningful difference if, let's say, a patient was scheduled to get a biopsy in March or April, but they don't end up getting it until May or June?

Say, a patient was scheduled to get a biopsy and in March or April, but they don't end up getting it until may or June.

Yeah. That's that's a good question you know so we you know.

Unknown Executive: Yeah, that's a good question. I'd say we're waiting to get the last biopsy on approximately 40% of the patients, somewhere around that. They were all scheduled to come in, and this has sort of interrupted that. But we don't think there's a real issue in terms of getting... In terms of the biopsy for treating them longer, we don't think that will be a real problem, so we should be able to, once the sites are back and running and open, be able to get this accomplished. So we don't think that that'll be a big deal.

Probably we've gotten.

I say, we're we're waiting to get the last biopsy on approximately 40 page 40% of the pace.

Somewhere around there there were all scheduled to comment on but some sort of interrupted that we don't think there's a real issue in terms of.

In terms of getting.

In terms of you know a biopsy for training them longer we don't think that won't be a real oh problem right. So we should be about two I'll be able to once the size of back and running an open.

Vehicle thing to be able to get that's accomplished so we don't think that that's what I think that'll be a big deal.

Okay and is it something that you've discussed with L.P.A., that's the way.

Unknown Executive: Okay, and is this something that you've discussed with FDA about this delay?

I think in general they know that sites are.

Unknown Executive: I think in general they know that sites are... are having issues. And so I don't know if we've actually talked to them directly, but they put out things already about what they know about some sites that have just closed down, especially Metropolitan Area. So, and in this particular case, I mean, you might recall from the Sarepta case that they actually had a nine month and an 18 month trial. So we don't look at this as a real issue.

Are having issues.

So I don't know, if we actually talked to them directly but they put out.

Things already about because they know some type of junk close down that specially.

Metropolitan area. So.

Particular case I'm as you might recall from replicates that they actually had a a nine month and the 18 months.

<unk>.

Okay. Thank you.

Unknown Executive: Okay.

Unknown Attendee: Thank you.

Thank you.

Unknown Attendee: Thank you. As a reminder, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Our next question comes from the line of Joel Beatty from Citi. Your line is now open.

Mind or Casco question, you only need to Crestar one on your telephone to withdraw your question press the pound key.

Our next question comes from the line of Joel Beatty from City, you're line is now Ben.

Unknown Attendee: Hi, thanks for the question. When RISD plans launch, should we anticipate that payers will only cover it as a monotherapy, or could there also be situations where payers could be open to paying for it as chemotherapy with Zolgensma or SINRASA?

Hi, six or the question so.

When this to play I'm launches shall we anticipate the parents will only cover as a mental therapy or because they're often these situations that parents could be open to paying for it it's kind of <unk> people intelligence or something rather.

So.

Unknown Speaker: [inaudible] So maybe a couple of points to think about, you know, when you have to remember that, Unknown Speaker. Let's start with Transluc... I mean, there's a plan where...

Well, maybe a couple of points think about you know when you got to remember that.

Let's start with.

Trends <unk> you know there's the plan.

Spend rod, though we don't understand that actually why that was our current necessarily because.

Unknown Executive: Thank you very much. Here's the plan for a very good job in terms of being able to make the appropriate RNA and, therefore, approach. In the case of GENZMA, our guess is that we'll be competing with them in order to get a Type 1 patient, and that, my guess is that it's patient. [inaudible]

They both sort of.

<unk>.

Oh curtains place and this will be some bad.

Transcript then I think what I think then we've shown pretty.

Clearly, but you can actually make all of the I spent man.

A a two hour into into the appropriate.

<unk> to make the <unk> so so <unk>.

<unk> Oh very good job in terms of being able to.

The appropriate our name there for <unk>.

In the case of junk them my guess is that.

Competing with them.

Type one.

Patience and that my Guy that pretty soon.

Feel the need to get additional treatment.

And we know for murder trial that we currently have.

Unknown Speaker: Go from our trial that we currently have.

Unknown Speaker: There are patients from Slojenzema who are now on an open label trial who are now getting a risk diploma as well. So we would anticipate that there will be patients who probably would want to get RISD.pharm as well, especially in the light of we don't know how durable or the variability of how patients do when they get this.

Patients, who Oh ooh from so gentle Oh now Oh no on.

Trial or no.

<unk> as well so we would anticipate the they'll be patient probably wouldn't awhile ago.

Rent appointment, especially in both like though.

<unk> or or.

Okay. So.

When they get this.

What makes sense.

Unknown Attendee: One more question on the plan. Are there certain regions that you anticipate?

Well my question.

Plan.

Are there certain regions that you anticipate you have a little bit more favorable dynamic for more rapid lunch you know that they're another each as you know what type of factors would would help but we should have a rapid launched.

Unknown Attendee: We have a little bit more favorable dynamics for...

Unknown Speaker: More rapid launch than other regions, you know, and what type of factors would help a region have a rapid launch of... Christopher Palmer

<unk>.

Unknown Speaker: Um Well, we think, I mean, I think that Roche will obviously Roche is in charge of, I'm in charge of commercialization, but they're certainly going to want to get moving rapidly in the US and then really throughout the world.

Well, we say I mean, I mean, I think that's Roach will obviously rose in charge of.

Commercialization, but that's certainly going to want to get moving rapidly.

And then really throughout the world.

Unknown Speaker: Unknown Speaker, will want to be done, but the US is the first. The first country that they would go for. The other point, actually, I wanted to make is that

We'll want to be done, but the U.S.

First.

Well first of course countries <unk> role for another point exactly wanted to make and the famous question also was that when I think about the war precedence and where a spin round almost cover about those functions.

Unknown Speaker: When I think about it, there were many...

So I think you know certainly that also goes along with the notes and.

Unknown Executive: Thank you all for joining us today.

Unknown Executive: Great, thank you.

We insist that they separate appointment probably.

Unknown Attendee: Thank you.

<unk>.

Created thinking.

Unknown Attendee: Thank you. Our next question comes from the line of Joseph Thome from Cowan and Company. Your line is now open.

Thank you are next question comes through the line of Joe. So it's all from Cowan in company airline is now.

Unknown Attendee: Hi there, thank you for taking my questions. Just the first one on the GMP manufacturing in early 2021. Can you explain how this possible slate delay would impact any sort of initiation of gene therapy studies if those are tied together? And then my second question is, it looks like there is a study on clinicalchilds.gov for trans LARNA in DMT patients aged six months to two years. Just wondering what you're hoping to show here and is this related to a potential US submission or the ex-US label? Thank you.

And everything you Ferret kicking my questions just first one on the the G.M.P. manufacturing and in early 2021 communities.

Articulate how many houses.

Maybe sleeping late we didn't have any sort of initiate thinking therapy studies. It sends are those are tied together and then.

My second question is it looks like there was a study on clinical trials dot Gov trends Lorna and D.M.D. patients age six months for two years. Just wondering you know what you're hoping to show here and it just related to essentially you at submission or or <unk> legal.

Sure Yeah. So the.

Unknown Executive: Sure, yeah, thanks. So the GMT manufacturing in terms of Hopewell site was really just because when we're thinking about COVID, we've already started making toxicology levels of material at the Bridgewater site. So based on being able to do that, we don't anticipate really being slowing it down at any point.

The.

So the the G.M.T. manufacturing.

The hope while I was really jumped on both when we're thinking about uncovered we've already we're already started making toxicology.

Level material in the bridge waters site told me, we based on being able to do better.

But it really it'd be slow into down it anyway.

Unknown Executive: ....

Unknown Executive: So it allows us, just before the opening of the plant, until we get to 2021 in the current environment we have so that there will be no delays as a consequence of pushing it forward by four months. So we didn't think it was a real issue and it conserved a considerable amount of cash, so we thought we might as well do that since we had already created the tox batches at our Bridgewater site. And for the six months to two years of age, it's really just to get the extension and to be able to bring in We've done the work to get the extension, and we did that, and we've gotten that approval in the US. In Europe, sorry, to expand the label so that people with non-susception can get it earlier. And so that's what we've worked towards. So that's just to continue to get earlier and earlier patients.

Louder than just for the opening the plants are we get 2021 in in the current environment. We have so that it <unk> there'll be no delays as a consequence unfortunate forward bike for a month.

Thinking was a real is shown us concern bug.

Okay. So we thought we might as well do that because we already have created to talk of taxes and.

It's not a prince water site in terms of the six months between your so they just really just to get very expensive, we'll be able to bring we've gone to work together Oh, good and we did that Oh, and we've gotten that actually approval in the U.S.

Oh that was I mean in Europe, sorry to expand the label so that.

People.

The people with.

Gathered earlier.

That's what we've Oh work <unk>, so that's up to continue to get.

Earlier and earlier patients.

Unknown Attendee: Great. And then I could just do one more on AADC. I see you're having some data presentations at ASGCT to outline changes on the Peabody scale and aims and, you know, some functional endpoints. In terms of the regulatory review and maybe what positions are interested in, how are they each going to look at these different efficacy endpoints? Is there one that's most important, or do they look at them kind of holistically?

Right.

Just do one more on a D.C. and I see your data presentation today should see people outline changes on the P. body scale and aims and functional endpoint in terms of the regulatory review and eat what positions are interested in how are they each going to look at these different X.P.C. and point to the one that's that's.

Most important.

I'm kind of hold instantly.

Sure. So you want to check that.

Matt: Sure. So, Matt, do you want to take this?

Matt: Yeah, sure. Thank you, Joseph, for the question. I think when we think about the AADC data package, first, it's very important is the fact that we're able to show on PET scans that there is a significant increase in dopamine production in all the patients. So that's the first very important sign that we're addressing the critical underlying biochemical defects. That's very compelling evidence that we're really targeting the fundamental pathology of the disease. Then, as you look at the data package as a whole, including the primary endpoint of head control, we're able to show that there's benefit in every patient treated. And what we're now seeing with the long-term data, as shown in some of the abstracts you mentioned, is that we're seeing durability of effect out past five years, even close to 10 years in some patients. That's very strong, compelling data. So what we believe is that physicians will appreciate the totality of the data, that one, we're addressing the fundamental biochemical defect in all patients, and two, across the board, we're seeing correction and evidence of correction of the key motor function defects in the disease. And that those effects, those favorable effects, are sustained.

Yeah sure. Thank you chose it for the question.

What we when we think about the A.B.C. data package cursed it's very important.

And we're able to show on pet scan that areas a significant increase in doping meat production in all the patient. So that's the first very important sign that we're addressing the critical underlying biochemical defects. That's very compelling evidence that were really targeting the fundamental pathology of the disease Dang.

As you look at the data package it at whole, including the primary endpoint.

Troll were able to show that there's benefits in every patient treated and what we're now seeing with the long term data as shown in some other the abstracts you mention is that we're seeing durability of effect out past five years, even close to 10 years since on page. That's a very strong compelling data. So what we believe is that.

Issues will appreciate the totality of the data.

One we're addressing the fundamental biochemical detecting all patients and two across the board, we're seeing correction and evidence of correction of the key motor function defects in the disease.

Those effects those favorable effects are sustained.

Unknown Attendee: Great! Thank you so much.

Great. Thank you so much.

Unknown Attendee: Thank you. Our next question comes from the line of Vincent Chen from Bernstein. Your line is now open.

Thank you are next question concerning the line of Vincent Chen from Bernstein. Your line is now open.

Congratulations on progress and thanks for taking the questions. A couple of from me. The first one is just on.

Unknown Attendee: Congratulations on all the progress, and thanks for taking the questions. A couple from me.

Coburn 19 impact I sort of you could elaborate a little it further and what impact you've seen so far as far as slow down related.

Unknown Attendee: The first one is just on, I guess, revisiting the COVID-19 impact. I was wondering if you could elaborate a little bit further on what impact you've seen so far as far as slowdowns are related. Sounds like the franchises have been pretty resilient so far, and I have to imagine the worst has largely been in April. [inaudible] and then the second section.

Don't think the French I should have been pretty originally and so far and after that.

Okay.

The decisions major withdrawal guidance given potential Toby related impacts.

Better sense more whether you're indeed, seeing any slow down and demand for it turned to market product over the course of the last couple of months weather end of Q1 or in in April to date.

Or or pulling gardens, just in case things were to get worse going going forward.

Unknown Executive: Thank you. Thank you.

Yeah on.

Unknown Executive: Well, that's about it, yeah.

Yeah.

Okay cool.

Unknown Executive: Okay, so yeah, that's a good question. Because, you know, it goes to the pulling of guidance is more on, or, you know, or withdrawing it at the moment, really, uncertainty of where things are going in the long term. You just don't know. I would say in the short term, we feel pretty good where we are in the first quarter and even after how things look, in terms of, you know, maintaining the patients already on drugs and even seeing new prescriptions. So it wasn't like we saw a slowdown, and maybe Eric, you know, maybe you want to put a little bit more around this a bit.

A good question.

Go to the the the pulling a guy that more on or you know or withdrawn at at the moment really certain fields.

Where things are you going in there.

The long term you <unk> you just don't know I would say in the short term we feel pretty good. We're we're in the first quarter and even after healthy smart.

The you know maintaining the patience already on broad can you anything new prescription. So it wasn't like we saw slow down and maybe Eric <unk>.

You know, maybe you want to put a a little bit though.

Around the.

Eric Pauwels: Yeah, sure, Stu. Yeah, Vincent, thanks for the question. I mean, in terms of the top line, I mean, we've seen the trends right now, both in Europe and in Flanders, that are still very encouraging. As I mentioned, we haven't had any kind of increase at all, with patients asking for more product. We've been shipping mostly most of the patients on a month-to-month basis, but we continue to see those positive trends in Q2. The existing patient base and new patient growth are still there, although new patient diagnosis and new prescriptions have decreased a little bit now, as we've seen, because patients are not seeing physicians as frequently. But we have protected the base, which is very good, and the trends are good there.

Yeah sure Yeah, Vincent Thanks for the question to I mean in terms of the top line I mean, we've seen that trends right now both in Europe with imply that are very hurricane still.

As I mentioned, we haven't had any kind of increases of increases at all a patient asking for more product. We've been shipping mostly most of the patients on a month to month basis.

But we can see continued to see those positive friends and cute too the existing patient base and new patients grows.

It's still there, although new patient diagnosis and new new prescriptions have decreased a little bit now as we've seen because patients are <unk> not senior position as frequently but we have protected the base, which is very good and the trends are good there we have gone back to me.

Eric Pauwels: We have gone back to make sure that everybody realizes we have an adequate amount of supply, and there have been some requests. In addition to the uptick, there have been a few patients, and we're seeing more and more requests for potentially 90 days of supply for influenza, but it's still a small percentage. I think what we were concerned more about the unknown, and I think, as Stu highlighted, if the pandemic continues, and there's a shift in unemployment and payer mix, or potentially disruption to some of our translarna shipments that might be in various markets. Keep in mind, we have patients in close to 50 countries with partners, as well as direct business. If some of that is disrupted because of the pandemic, at this point in time, we're just trying to be cautious. But there's no indication now, based on our strong results in the first quarter and what we're seeing so far right now, that we're losing patients or that that growth would go down. And Ania, I'll give that back to Stu.

Make sure that everybody realizes we have an adequate amount of supply and and there have been some requests. In addition to you know the.

I'll take it there have been a few patient and we're seeing more and more equestrian potentially 90 days of supply for in Florida, but it's still small percentage I think I think what we were concerned more about as the unknown and I think it's do highlighted but there's if the pandemic continues and there's a shift of unemployed.

I'm in and pair mix or potentially disruption to some of our trends Warner shipments that might be in various markets keep in mind, we have patient and close to 50 countries with partners as well as direct business. If some of that is disrupted because of the pandemic at this point in time, we're just trying to be cautious, but it there's no into.

<unk>.

Based on our strong results in the first quarter and what were seen so far right now.

That that we're losing patience or that that would would go down.

And.

<unk>.

I <unk> and maybe one could fall on on that point.

Unknown Attendee: And maybe one quick follow-up on that point. If you think about the price for MFLAZA in the U.S., how does the price paid for MFLAZA differ between patients on commercial insurance and patients who are on government insurance? Take it another way; if more patients were, if you were to see a meaningful shift in payer mix, how might that impact the net price paid on average?

The it if you think about the price for M. flaws in the U.S., how does a place Kate lots of different between patients on commercial insurance and patience or on government insurance and take another way if if more patients were if you wouldn't be a meaningful shift in here mix.

In fact, the the net price paid on average.

Eric Pauwels: And the simple answer is it's basically a statutory rebate from CMS, which is higher right now. Our current payer mix is about 60-40, with commercial payers being the majority. If that obviously shifted, our gross to net would shift because there would be more patients moving on to Medicaid. But so far, we haven't seen that. It seems to be that DMV families, in general, have pretty good insurance and are covered. And in addition to that, if patients do go from private payers to Medicaid programs, the number of states actually have disability programs for children, and they can move very quickly. So again, I think the only effect there, Vincent, would be the statutory rebates that we would have to pay to CMS if the payer mix shifted.

Yeah on the simple answer is it's basically the statutory rebate from C.M. out, which is which is which is more which is higher.

Which is higher right now our current payer mixes about it you know.

<unk> 60, 40, <unk> with commercial pair thing the majority if obviously that shifted aggress to net would shift because there would be more patients moving on to Medicaid. So far we haven't seen that and it seems to be that C.M.D. families. In general have pretty good insurance and have covered and then.

In addition to that if patients do go from private payers to to Medicaid programs number of the states actually have disability programs for children.

And they can move very quickly. So again I think the only affect their opinion would be the statutory rebates that we would have to pay for T.M. at if there if the pair mixed yet.

Unknown Attendee: I see. And I'd like to ask another question just about TransLarna and the ongoing study. I was just wondering if you could give us a sense for how the powering calculations were done in terms of choosing the number of patients in the ongoing U.S. Dystrophin study. What data did you have to sort of draw upon in terms of estimating the likely effect size? Like, is there data you've collected from other patients treated with TransLarna to see just how much of an increase you see in dystrophin, whether from your studies or from clinical experience, I guess, in territories where TransLarna is used?

I see.

No one just on 10th Lorna and the the ongoing study.

Given for how the powering calculations were done choosing a number of patients in the ongoing.

<unk>.

What did did you have for draw upon in terms of estimating the likely effect size like or are there or is there data you collected from other patients treated with translarna to see just how much.

Dystrophin, whether from your studies or from clinical experience.

In in terms of words were transformed with you.

Unknown Executive: Yeah.

Yeah, Yeah. Thanks for that that's a good question. We we did look at other studies as well and it was based on data that we saw from from syrup done some just making some some assumptions based on that on and what the things like that we got there you might not.

Unknown Executive: Yeah, thanks for that. That's a good question. We did look at other studies as well, and it was based on data that we saw from SIRREPTA and from just making some assumptions based on that and what the assay is like that we got there. Is there anything else you want to add to that?

Is there anything else you want to answer that.

Unknown Executive: Yeah, just that we believed with the 20 subjects in the trial that we would be able to achieve 90% power, which we thought would be sufficient to show the transluant effect. And, of course, we expect a baseline to be, there'd be very, very low levels of dystrophin expression.

Yeah, just that we believe twist their 20 subjects in the trial that we'd be able to cheat you get 90% power, which we thought would be sufficient to show.

And the effect of course, we expect a baseline <unk> there'd be very very low level. So just dropping expression.

Unknown Attendee: Wait, when you say 90% power, did I ask you what the estimated effect size and the variability was?

When you think <unk> alright.

What would we definitely effect size and the variability.

Unknown Executive: Correct, as Stuart said, basically looking at other trial data and running simulations.

Correct.

Basic be looking at other trial date and running simulations.

Unknown Attendee: Okay, thanks. Thanks for taking the time.

Okay. That's the only question.

Oh.

Thank you all our next question comes from the line of G. now Wang's from Barclays airline is now open.

Unknown Attendee: Thank you. Our next question comes from the line of Gina Wang from Barclays. Your line is now open.

Unknown Attendee: Hi, this is Peter Fujino-Lang. Thank you very much for taking the time to answer my question. I guess my first question, my question is on the RISD plan. I guess, please, back to, Do you expect RISD plans to generate meaningful revenue this year, or do you anticipate some patient initiation impacts in COVID? And my second question on the related note is... Um, could you remind us when the EAP was open and how many patients have been sort of enrolled in the U.S. and across the U.S. and whether pre-COVID and post-COVID the EAP enrollment rate has sort of changed? Thank you.

Hi, this is Peter for generating.

Very much for taking a question.

I guess by a first question my question unrealistic plan.

I guess dates back to.

<unk> meaningful revenue despair, or you anticipate patient initiation impact and poet and my second question on the related note.

Could remind us when that he was opened and how many patients I've been sort of envelope then.

Oh, you asked and.

Weather pre Kobe day post Soviet that you can really great has.

Changed.

<unk>.

Unknown Executive: Sure, so maybe the big picture on COVID and revenues. So I think in some ways, people will start thinking about how do I make sure I have a drug supply for patients. So I think in some ways, it might be helpful from a commercial launch perspective.

Sure So maybe the.

Sure.

On on Kobe, Bryant, and and and ER Reverend or something.

So I think on the big picture on a covert I think in some ways.

I think people will start thinking about.

How do I make sure I could have drug supply.

<unk>, so I think in some ways that might be helpful from a commercial lawns perspective.

Emily Luisa Hill: I think in terms of revenues, that would be royalties. Maybe Emily, you want to talk a little bit about that?

I think you we from from rubber windows that would be royalties maybe.

What about that.

Emily Luisa Hill: Sure, I mean, we have the potential for up to $42 million in milestones this year through our collaboration with Roche on the Roots to Plan program. Obviously, we have...

Sure I mean, we well we have the potential for up to 42 million miles John This year on our collaboration focused on the Red Sam program.

Basically we have.

Emily Luisa Hill: Peer Royalties Up to the Mid-Teens. And with the MAA having been submitted earlier this year, and the PDUFA in the US now on August 24th, I'd expect those to ramp up more significantly next year. I think as far as COVID's impact, the rate and ramp of those revenues, there are really some advantages in potentially being the only SMA therapy.

Cared royalties mid teens and square that I may having been submitted earlier this year and do find the U.S. now in August 24th and expect us to ramp up more significantly next year I think that's fine.

Rate and ramp up so.

There's really some advantages.

Centrally being the only estimate therapy that can be delivered at home.

Yeah, I'm in the patient numbers, but well I don't think Rochelle.

Unknown Executive: And then the patient numbers, well, I don't think Rochette has discussed that, but I think what they did say on the call previously, and what they have said, is that there's a lot of interest, and so they are bringing patients in that are already on the EAP, and they're continuing to do that, both in the U.S. as well as setting it up also in Europe as well. A global EAP program, certainly in the U.S. everywhere, and then in countries that allow you in Europe to have these EAP programs.

But I think what they did say on the on call pretty refund what they have <unk>. They are upbringing patient fan.

Already that already.

I'm going to continually do that both in the U.S. as well as offensive seven it off also in Europe as well so it'd be on a global.

Oh global.

Program.

Certainly in the U.S. all over it and then in countries that allow you in Europe to help uneasy N.P. program.

Unknown Attendee: Got it. Thank you very much.

Got it thank you very much.

Unknown Attendee: Thank you. Our next question comes from the line of Raju Prasad from William Blair. Your line is now open.

Thank you are next question costs on the line of Red You Press odd from William Blair. Your line is now fan.

Unknown Attendee: Thanks for taking the questions.

Thanks, raising the question <unk>, what do you anticipate presenting additional fuel efficient Rainbow fish data and you have any general aside from what you've already mentioned give any general commentary on you know how many patients are are lotions, bringing in on the <unk>.

Unknown Attendee: On the RISD plan, when do you...

Unknown Attendee: I anticipate presenting additional jewelfish and rainbow fish data, and do you have any general commentary on how many patients Roche is bringing in on the expansion of the early access program or any details around what types of scenarios that they are expanding it to, is it just if patients are off Spenraza or don't want to go in, they apply, or is there more stringent criteria?

The early access program or any details around.

How what types of scenarios that they are expanding the two in just if if patients are off spinraza or don't want to go in they apply or is there more stringent criteria and then I have one on translation.

Unknown Executive: Sure, yeah, so that's an important point too, is that they are now planning presentations for the, that probably will be an update on the Cure SMA. And then, yes, they have changed the EAP program to allow patients who are in other therapies to get into the EAP program as well. And they've also added Type 2 as well to the program. So, yeah, so patients who can't get, say, intracranial injections or naive patients, they certainly can; it's really based on physician discretion, based on what they think the need is. So COVID is obviously having limited access, so this is really a nice way in which they can get it at home and take it. So that's also adding to the number of patients who are hopefully transitioning to VISTA class. I have a very strong interest in this stuff.

Sure Yeah. So that that's an important 0.2 is that.

They are now planning presentations for those that.

Being updated on the cure right. So.

And then they have changed.

E.S.P. program to allow patients.

To who are who are in another therapies to get into the I.P. program as well.

And they've also adding type two as well to the program. So so yeah, so patients who can't get things going Jackson or not you pay Sherman that they they certainly can it's really based on the recent discretion based on what they think that neither.

So coconut is obviously make unlimited access something really.

Oh wait a second guessing at home and taken from that.

And number pigs, and cool are hopefully trying to <unk>.

Very strong interest and stuff.

Yeah.

You have any oh ideas on how many in the U.S. verse you are getting out in the early axes pull them just.

Unknown Attendee: Do you have any idea of how many in the U.S. versus EU are getting on the Early Access Program?

General percentage wise.

No yeah that haven't been disclosed yet.

Unknown Attendee: No, yeah.

Unknown Attendee: Okay, and then I want to turn to Lorna. And then, obviously, Paul got in and commented on it enough during this call.

Okay, and then on one on friends go on a.

He pulled got in.

Commented on enough on this call, but just wondering you know usually.

Eric Pauwels: But just wondering, you know, usually you have a Q2, Q4 bolus from Latin American Orders. Is the general pattern of weapon recognition going to be similar? Yeah.

Two two q. for ball with.

A Latin American orders is the general pattern.

I'm going to be similar.

Yeah, I think you know.

Eric Pauwels: I think, you know, so I think maybe Eric could comment on that.

Maybe you were can comment on that.

Eric Pauwels: Yes, sure. With regard to Latin America, for TransLarna, as you recall, we had approval from MVISA last year, and we've been building a very nice base of patients. And we saw new patients coming in during the first quarter, not only diagnosing these patients, new prescriptions, but the base of patients that received positive injunctions for therapy continues to increase. So we've seen growth on all those parameters.

Mm.

Yeah sure.

We're yeah, what's your personal Latin America for trends, Florida is you can recall, we had approval from at least the last year and we've been building a very nice base of patients and we saw.

New patients coming in in the first quarter not only diagnosing these patients new prescriptions, but but the the base of patients that receive positive injunctions for therapy continues to increase so we've seen growth on all those parameters.

Eric Pauwels: You know, with regard to that base, we have ongoing negotiations with the Minister of Health and with MVISA to require that we get those group purchase orders. We're working towards securing a group purchase order in the second quarter. You know, we've shifted a lot of our virtual communications right now with the Minister of Health because of COVID-19 to virtual-type meetings, and we continue to have a very strong working relationship with the payers in Brazil. So our goal is, you know, as we've seen continuous increases in patients right now, our goal is to get and secure an order for Q2, barring any kind of exceptional acceleration of the pandemic or some kind of You've got to keep in mind, though, that the rare disease bucket for cost centers at the Minister of Health is relatively small in comparison to the overall health care budget. So we think we can work through some of these things and negotiate and hopefully obtain an order in Q2 and ensure that patients' continuity is there. Again, we're very encouraged by the continued growth, and it's going to continue to put pressure on the Minister of Health to secure those orders.

You know with regards to that.

We we have ongoing negotiations with the ministry of health and with that'd be so that you require that that we get those <unk> purchase orders were working towards securing a good purchase shorter in the second quarter.

You know, we've we've shifted a lot of our virtual communications right now with the Ministry of health because it covered 19 to to a virtual type meetings and we continue to have a very strong working relationship with the payers in Brazil. So our goal is is you know as we've seen continuous number increases.

Patients right now our goal is to get insecure in order for a few too barring any kind of exceptional acceleration of the pandemic or some kind of government diversion of resources.

You got to keep in mind, though that the rare disease bucket for for cost centers at Minister of health is relatively small in comparison to the overall health care budget. So we think we can work through some of these things and negotiate and hopefully obtain in order to Q2 and and ensure that patients continuity or.

Is there were again, we're very encouraged with the it continued growth and it's going to continue to put pressure on the on the minister of house to to secure those orders.

Unknown Attendee: Great, thank you.

Great. Thank you.

Unknown Executive: Thank you. At this time, I am showing no further questions. I would like to turn the call back over to Stu for closing remarks.

[noise]. Thank you know at this time I'm showing no further question I would like to turn the call back over to do for closing remarks.

Unknown Executive: Ah, okay. Well, look, I want to thank all of you for joining the call today. You know, I want to remind you that at the end of March was our 22nd anniversary at PTC. And so as I look back upon our, you know, now our long history, I'm very proud of how the company has evolved. It's gratifying. We all see the progress that we've all made and continue to make in discovering, developing, and commercializing treatments for patients living with rare disorders. I think really as a consequence of the hard work and dedication of the team, we're well positioned to be able to push forward and even thrive, even under these uncertain environments. So again, thank you for joining the call. I look forward to it; I hope to see you all soon.

Oh, okay well.

I want to.

Call today.

Remind you that.

End of March 22nd anniversary in P.G.C., and so as I look back upon.

Hour long history, I'm very proud of public company.

Have evolved describing gratifying.

The the progress that we've all made them to continue to make and discovering developing commercializing treatments <unk>.

For patients living where disorders, and I think really a consequence of the hard work and dedication of a team.

Well.

Be able to push forward.

Right even on the the uncertain environment.

So again, thank you for on joining the color forward I hope.

See you all.

Unknown Executive: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.

Ladies and gentlemen, this complex today's conference call. Thank you for participating you may now disconnect.

Unknown Executive: [inaudible]

[music].

Yeah.

[music].