Q3 2024 PTC Therapeutics Inc Earnings Call
Okay.
Speaker Change: Welcome to the P. T C third quarter 2024 financial result conference call. At this time all participants are in a listen only mode. After the speaker's presentation. There will be a question answer session to ask a question. During the session you will need to press star one one on your <unk>.
Speaker Change: Telephone.
Speaker Change: You will then hear an automated message, but your hand is raised to withdraw your question. Please press star one one again please be advised that today's conference is being recorded I would now like.
Speaker Change: To hand, the conference over to your first speaker today, Jane Hamlin Associate director of Investor never Investor Relations. Please go ahead.
Jane Hamlin: Good afternoon, and thank you for joining us today to discuss PTC therapeutics third quarter 2024, corporate update and financial results.
I'm joined today by our Chief Executive Officer, Dr. Matthew Klein or.
Jane Hamlin: Our Chief Business Officer, Eric Powell, and our Chief financial Officer of paragraph yet.
Jane Hamlin: Today's call will include forward looking statements based on our current expectations.
Jane Hamlin: Please take a moment to review the slide posted on our Investor Relations website in conjunction with the call which contains information about our forward looking statements.
Jane Hamlin: Our actual results could materially differ from these forward looking statements as such statements are subjects to risks that can materially and adversely affect our business and the results of operations.
For a detailed description of applicable risks and uncertainties. We encourage you to review the company's most recent quarterly report on Form 10-Q, and annual report on 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.
Jane Hamlin: Information regarding our use of GAAP to non-GAAP financial measures.
Speaker Change: Reconciliation of GAAP to non-GAAP are available in today's earnings release with that let me pass the call over to our CEO Matthew claims that.
Matthew Claims: Thank you all for joining the call today.
Matthew Claims: I'm happy to share the results of another quarter of outstanding commercial performance and pipeline execution.
Matthew Claims: In the third quarter, we achieved total revenue of $197 million, including $124 million from the DMD franchise.
Matthew Claims: To highlight the $52 million and plaza revenue, which reflects our team's ability to effectively navigate a generic sized and competitive marketplace.
Matthew Claims: With our strong third quarter revenue performance, we closed the quarter with over $1 billion in cash and are raising our 2024 total revenue guidance to $750 million to $800 million.
Matthew Claims: In the third quarter, we submitted to the NDA to the FDA, one for CPA Taryn and one for <unk>.
Matthew Claims: Applications have been accepted for filing.
Matthew Claims: The FDA planned action date for CPO Taryn is July 29, 2025, and action date has not been provided for the trans Lorna NDA due to the regulatory context of the filing.
Matthew Claims: Its secret Taryn, NDA, including the significant and clinically meaningful evidence of efficacy from the phase III affinity study as well as data from the affinity open label extension study demonstrating durability to the CPA taryn treatment benefit.
Matthew Claims: Ability of patients to increase their protein intake, while maintaining control of fee levels.
Matthew Claims: These data support CPA Taryn can provide significant benefit to PKU patients of all age groups and severity subtypes, including classical PKU.
Matthew Claims: The impactful results for the affinity trial were recently published in the prestigious Lancet Journal a testament to the importance of the study findings.
Matthew Claims: In addition to the FDA submission marketing authorization applications for <unk>. Karen are currently under review in the EU and several other countries, including Brazil.
Matthew Claims: We remain on schedule to submit the J NDA in Japan in December of this year.
Matthew Claims: Submissions will support our planned global launch of Cps Chairman in 2025, our launch plans are progressing well as Eric will detail shortly.
Matthew Claims: We remain on track to submit an NDA for particularly known for the treatment of children and adults with Friedrich ataxia in December.
Matthew Claims: This NDA submission will be the fourth approval applications submitted to the FDA. This year, an impressive testament to our team's execution.
Speaker Change: Just a quick one on NDA will be based on the findings are significant benefit on the upright stability subscale of the <unk> disease rating scale and the move at a placebo controlled trial as well as results from two long term extension studies.
Speaker Change: As we recently reported both long term extension studies met their primary endpoint with evidence of statistically significant long term benefit in delaying disease progression.
Speaker Change: Given the extensive safety data collected to date in both children and adults with <unk> has the potential to fill the unmet need for pediatric and adolescent patients as well as provide a promising therapy for adult patients.
Speaker Change: Moving to our PTC 508 program for Huntington's disease. The program received fast track designation from FDA in September reflecting the potential of PTC $5 eight to be the first ever approved disease modifying therapy for HD.
Speaker Change: In addition, we requested two type C meetings with FDA.
Speaker Change: One meeting is to discuss the potential for Huntington lowering to serve as a surrogate endpoint supportive of accelerated approval in the second meeting is to discuss the endpoint strategy for an efficacy trial in HD patients whether that trial has completed a phase III registration trial.
Speaker Change: <unk> as a confirmatory trial in the context of an accelerated approval.
Speaker Change: The FDA has asked to combine the two into a single type C meeting, which has now been scheduled for later in the fourth quarter.
Speaker Change: The ongoing phase III pivotal HD trial continues to progress well and we remain on track to complete the study in the first quarter of 2025 with results expected in the second quarter.
Speaker Change: Turning to our mutual access that program for Alice we remain on schedule to share top line results from the registration directed Cardinal trial in the fourth quarter.
Speaker Change: Lots of if that is the first compound being developed for ALS patients that specifically targets <unk> doses pathway of oxidative stress and cell death demonstrated to be highly relevant kols pathology.
Speaker Change: Given the recent changes in the therapeutic landscape for Alice positive results from the Cardinal study could enable you to lots of staff to address the significant.
Speaker Change: Net need.
Speaker Change: <unk> patients.
Speaker Change: Finally, we remain on schedule for the November 13, FDA action date for the ADC in gene therapy BLA.
Speaker Change: If approved this would be the first ever direct to brain administered gene therapy authorized by FDA and open the door to a whole new approach to treating brain diseases.
Speaker Change: I am very proud of our team's ability to bring this pioneering therapy to patients.
Speaker Change: In summary, we have had a very busy and productive quarter as we continued to execute on all of our planned objectives and achieve our many significant 2024 milestones I will now turn the call over to Eric to discuss our commercial performance Eric.
Eric Powell: Thanks, Matt.
Eric Powell: Global customer facing team had another strong performance in the third quarter delivering $135 million of revenue across our five marketed products, our global DMD franchise had a solid quarter with $124 million of revenue.
Eric Powell: Despite <unk> recent negative opinion on renewal there continues to be strong support from physicians patients and families throughout Europe for <unk> based on the positive totality of evidence in the trials and the real world experience.
Eric Powell: We continue to ensure that transport is made available to boys and young men living with nonsense mutation Duchenne muscular dystrophy in Europe as long as it remains authorized pending review of the <unk> opinion by the European Commission.
Eric Powell: Outside of Europe, our commercial teams have worked effectively with health care professionals as we continue to add new patients and maintain those on existing transponder therapy in Latin America, the Commonwealth of independent States, the middle East and North African countries in <unk>.
Eric Powell: In the third quarter, we obtained first time trends, Florida orders from two new countries in the Middle East and North African region.
Eric Powell: In Brazil, we delivered the remaining 50% of a group purchase to the Ministry of Health.
Speaker Change: As Matt mentioned.
Speaker Change: NDA for Translarna is currently under review by FDA, and our highly experienced U S team is very well positioned to bring trends, Florida to nonsense mutation DMD patients rapidly pending potential FDA approval.
Now turning to the Plaza third quarter net revenue was $52 million.
We continue to work closely with health care professionals payers.
Speaker Change: Specialty pharmacies to dispense the breadth.
Speaker Change: Our PTC Care's team provides white glove personalized services and programs supporting each patients with co pay assistance brand information and timely shipments from our specialty pharmacies greatly enhancing the experience for both new and continuing patients.
Speaker Change: Our customer facing teams are extremely excited about the potential upcoming global launch of <unk> for PKU patients.
Speaker Change: Based on the feedback from patients families and physicians, we believe subjects Aaron has the potential to generate significant revenue, including and over 1 billion opportunity in the U S alone.
Speaker Change: PKU is a large addressable orphan disease population of more than 58000 patients in markets with access and reimbursement and.
And the vast majority of patients are not receiving medical treatment due to the limitations of current options.
Speaker Change: We look forward to providing physicians with this important new differentiated treatment option to address these high unmet needs in PKU patients.
Speaker Change: As Matt mentioned 17, Karen is under review in the U S and Europe, which are important steps in bringing this important new therapy to children and adults living with PKU.
Speaker Change: Further submissions are under review by regulators in Brazil, Canada, Switzerland, and Australia with a filing plan in Japan by the end of this year positioning.
Speaker Change: Out for potential global launch in multiple major markets in 2025 and beyond.
Speaker Change: I will provide some details on our launch efforts, which have been ongoing for several years.
Speaker Change: We have been working closely with key PKU with health care providers, including geneticists.
Speaker Change: Pediatric metabolic specialists.
Speaker Change: Tissues around the world to better understand the unmet needs and build a strong relationship with the PKU patient advocacy community.
Speaker Change: Our teams continue to develop a deeper understanding of the PKU landscape.
Speaker Change: With many of the key treatment centers and prescribers.
Speaker Change: As you know PKU is the most prevalent inborn error of metabolism disease with reliable.
Speaker Change: EMEA allergy via newborn screening available since the early 19 sixties.
Speaker Change: PKU patients receive their care from well defined treatment centers and clinicians around the world.
Speaker Change: We have mapped these treatment centers and health care providers in key markets globally.
Speaker Change: Since the recent publication of affinity data awareness of <unk> has rapidly increased in many of these centers of excellence.
Speaker Change: Highlighting the compounds differentiated efficacy profile seen in clinical trials as a convenient oral treatment getting more patients defeat allow me goal levels and enabling potential diet liberalization.
Speaker Change: Our field based teams have met frequently with PKU stakeholders, who have expressed their excitement about our new treatment option to address their unmet needs potentially available next year.
Speaker Change: Importantly, patients highlight optimism with the ability of <unk> to liberalized diet and this is an important motivator for patients to seek new treatment options earlier from their health care providers.
Speaker Change: Now in addition, we are leveraging our established rare disease global infrastructure, which has a footprint in over 50 countries.
Speaker Change: We will sequence the potential country launches of Cepheid Tara to maximize access in key markets and maintain a narrow pricing and reimbursement corridor globally.
Speaker Change: Our research benchmarking of many rare disease analogs indicates a premium pricing strategy can be obtained based on the differentiated profile of <unk>, Sharon compared to current branded treatment options available, especially in key markets, where PKU treatments are reimbursed and where we are planning our initial launch.
Speaker Change: Sequences upon potential approvals.
Speaker Change: Our customer facing teams understand the complexities of managing rare disease treatments as well as the regulatory and payer access landscape.
Speaker Change: These core capabilities developed at PTC over the last decade, along with <unk> highly differentiated profile should facilitate rapid adoption for PKU stakeholders and reinforce our belief in the potential 1 billion plus opportunity not only globally, but in the U S alone.
Speaker Change: Turning to particular note with the NDA submission planned for December of this year. Our commercial team is excited for the potential opportunity to launch, particularly known for patients in 2025.
Speaker Change: We believe that particular note is well differentiated from current treatment options and to benefit all FAA pitches, and particular pediatric and adolescent patients.
Speaker Change: Kols, who treat us I believe there are significant unmet needs in this devastating disease.
Speaker Change: They tell us that particular unique mechanism of action, it's compelling efficacy data in highly relevant functional parameters for FAA patients along with its favorable safety profile provide a strong rationale for treatment as early as possible in the course of the disease.
Speaker Change: Our experience neurology team is focusing on key projects in preparation for the U S launch of the <unk>.
Speaker Change: Now moving to take steady and way Libre in Latin America, we continue to make good progress across these franchises with growth in both newly identified and treated patients across the region.
Speaker Change: Our geographical expansion continues with a recent approval in positive HDA assessments of Tech study in Mexico.
Speaker Change: During which we have initiated reimbursement discussions.
Speaker Change: In Brazil, we delivered a new government purchase order for Tech study in the third quarter.
Speaker Change: Okay.
Speaker Change: In conclusion, we continued to execute on all fronts based on strong first nine months of revenue this year.
Speaker Change: Matt mentioned, we are updating guidance with a 2024 total revenue guidance of $750 million to $800 million.
Our customer facing team is thrilled by the opportunity to potentially launch for new product.
Lorna: And you see gene therapy trends Lorna.
Speaker Change: Darren and in particular in the U S.
Speaker Change: As well as <unk> globally in 2025.
Speaker Change: I will now turn the call over to Pierre for a financial update.
Speaker Change: Sure.
Pierre: Thank you Eric.
Speaker Change: I will now share the financial highlights of our third quarter of 2024.
Speaker Change: Please refer to the earnings press release issued this afternoon for additional details.
Speaker Change: Beginning with top line results so.
Speaker Change: Total revenue for the third quarter was 197 medium, including DMD franchise revenue of $124 million.
Speaker Change: <unk> net product revenue in the quarter was 72 million, what Im Plaza net product revenue of $52 million.
Speaker Change: Moving to everybody.
Speaker Change: Third quarter Global net revenue of approximately 460 million U S. Dollars was achieved by Roche, resulting in royalty revenue of 61 million for PTC.
Speaker Change: non-GAAP R&D expense was 152 million for the third quarter of 2024, excluding.
Speaker Change: Excluding 9 million in noncash stock based compensation expense compared to 115 million for the third quarter of 2023, excluding $14 million noncash stock based compensation expense.
Speaker Change: non-GAAP SG&A expense was 63 million for the third quarter of 2024, excluding $10 million noncash stock based compensation expense compared to 68 million for the third quarter of 2023, excluding $13 million noncash stock based compensation expense.
Speaker Change: Cash cash equivalence and marketable securities totaled a one <unk> billion as of September 32024, compared to $877 million as of December 31, 2023.
Speaker Change: Our strong financial position provides PTC with the necessary resources to execute on our strategy and achieve our milestones over the next several years, including the anticipated launch of <unk> and other products.
Speaker Change: I will now turn the call over to the operator for Q&A.
Speaker Change: Radar.
Speaker Change: Thank you.
Speaker Change: This time, we will conduct the question and answer session. As a reminder to ask a question you will need to press star one one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one one again, please standby, while we compile the Q&A roster.
Speaker Change: Our first question comes from Kristine <unk> of Cantor Fitzgerald. Your line is now open.
Speaker Change: Hi, This is Rick on for Kristen Thanks for taking our question.
Speaker Change: We've got two for you so.
Speaker Change: So first of all thanks for the update on the type C meeting in Huntington's do you have a sense for when you could update the community on any clarity that might come out of the meeting is there any chance that we'll learn more about a potential path forward here.
Speaker Change: Yeah. Thanks for the question. So the we know Theres a lot of excitement around the discussions we're going to hire with FDA. Both in terms of understanding the endpoints for efficacy trials, but also of course, the discussions around Huntington borrowing as a surrogate endpoint as we've talked about.
Speaker Change: When we think about surrogate endpoints for accelerated approval with the FDA really wants is for us to present, a package of scientific evidence of why lowering Huntington protein is likely to.
Speaker Change: Provide clinical benefit ultimately and are there specific thresholds of lowering that have been associated with that benefit. So we have been able to provide them with the rich scientific literature, including in vitro and in vivo in human studies, which show that if you lower Huntington protein, 20% to 50% that youre likely to have a significant benefit in slowing disease progression and of course as we've shown.
Speaker Change: And our 12 Motivity HD update we are achieving that threshold of Lora Huntington protein.
Speaker Change: We expect to have these meetings with FDA in December of this year.
Speaker Change: And as always as soon as we have clarity, which sometimes comes at the time of the meeting or shortly thereafter or once we receive minutes, we'll be sure to share that with the with the broader <unk>.
Speaker Change: Immunity, including the street as we know Theres a lot of interest in hearing about this path forward for accelerated approval of pivotal HD.
Speaker Change: Excellent and maybe just one more if we have time, we've seen a lot of.
Speaker Change: Regulatory flexibility in the rare disease space recently, so given the FDA resubmission in for.
Trans Lorena: Trans Lorena can you talk about how you think the FDA might sort of weigh the stride registry data and steady flow 41 that were submitted and just kind of looking at these new evidence given sort of how things have been going in terms of regulatory flexibility.
Speaker Change: Sure Rick So as we've talked about before the NDA Resubmission for trends Lorena was based on two things first the evidence of significant benefit in the overall enrolled population of 359 boys in the <unk> study 41.
Trans Lorena: This is where we had significant effect.
Trans Lorena: Six minute walk distance Northstar ambulatory assessment time function test time to loss of 10% in that data set.
Trans Lorena: Broad nonsense DMD population showing significant benefit across all of those endpoints is actually pretty unique with flexibility or not I think this is really the first data set that has an ITT population significant effect across all of these endpoints.
Trans Lorena: In addition, as confirmatory evidence, which the agency likes to see in addition to a single adequate and well controlled trial. We have described registry and what the stride registry is able to demonstrate is these significant benefits, we're seeing on ambulatory function and other aspects of disease in the short term.
Trans Lorena: <unk> 41 are manifesting cumulatively over several years as a delay in loss of ambulation of three and a half years. So.
Trans Lorena: Of course any flexibility the agency can bring to the assessment will be of the packaging would be great, but on its own we have it.
Trans Lorena: Efficacy package with evidence of clinical benefit in both the short term and the long term and nonsense DMD patients, which is quite a unique and compelling package of data.
Speaker Change: Thank you.
Speaker Change: Thank you.
Speaker Change: Our next question comes from Eric Joseph of Jpmorgan. Your line is now open.
Speaker Change: Good evening, thanks for taking the questions.
Speaker Change: One on the Cardinal trial, we noticed recently that you updated.
Speaker Change: The change modify the primary endpoint to use a participant rates combined assessment.
Speaker Change: Sure.
Speaker Change: AOS FRS and also survival can you talk about what some of the motivations were behind the changing to that endpoint and what what the.
Speaker Change: The impact might be from a six quarter.
Speaker Change: Any perspective, and I guess I think you've also now over enrolled.
Speaker Change: This study was that meant to accommodate.
Speaker Change: The change in end point or would you expect to have any sort of sense.
Speaker Change: Slightly overpowered versus prior expectations. Thank you.
Eric Powell: Thanks for the question Eric.
Speaker Change: No.
Speaker Change: Those two are not related to your two questions are not related.
Speaker Change: <unk> is really a way of.
Analyzing the results of the study.
Speaker Change: The outcome of interest remains the ALS FRS score, which is a continuous variable that rates disease progression over the course of the trial. However, it's well known that patients will die over the course of the study and the FDA has recommended in order for us to account for that in the primary analysis they would like.
Speaker Change: US to use their preferred analysis method.
Speaker Change: <unk>, which is known as they joined rank test, which is the statistical test that's able to incorporate the information of decline on the ALS FRS as well as any deaths that occurred during the course of the study. So it's really just a change in the way we're analyzing it to include that in the primary statistical analysis of the <unk>.
Speaker Change: And again, Mr. Kim from feedback from the FDA on the analysis plan as they have said that that is really their preferred analysis method and will certainly if you look back to their reviews of other <unk> drugs Youll see that that is in fact, the tests that they like to use because it's able to incorporate information from all subjects.
Regarding your second question as we've talked about we had a target population for the primary analysis population based on our way to progression observed during the two months running other studies. So this two months running study, where we observed patients and calculate how many points they change on the AOS FRS.
Speaker Change: Scale, we need to enroll a certain number of patients in order to achieve the targeted number of patients in that primary analysis group. So what you are seeing the increasing enrollment was really just because we had a slightly lower yield.
Speaker Change: Screening subjects, who actually met the criteria for the primary analysis population.
Speaker Change: Okay got it.
Speaker Change: Thanks, very much for taking my questions.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question comes from Kelly <unk> of Jefferies. Your line is now open.
Speaker Change: Thank you for taking my questions two questions given the revenue base for this quarter could you help us to set up an expectation for Q4 and maybe also into 2025, what would it be that dynamic.
Speaker Change: Our DMD franchise also birthday.
Speaker Change: That's all it is from everybody's day. Thank you.
Speaker Change: Yeah.
Speaker Change: Yeah. Thank you for the question Kelly.
Speaker Change: So as we said we were.
Speaker Change: Revenue was quite strongest quarter, which allowed us to raise the overall guidance this year to $750 million to $800 million. We expect in the fourth quarter continued contributions from across our portfolio of inline products.
Speaker Change: In terms of both the Dnb franchise revenue as we've talked about in the Plaza continues to perform incredibly well.
Speaker Change: They've done a great job of protecting the brand.
Speaker Change: It's a generic competition.
Speaker Change: And the trends are in our revenue, we expect to be stable over the fourth quarter.
Speaker Change: Certainly as it remains available in Europe, we will have the typical lumpiness that we see quarter to quarter with trends are but overall, we expect strong DMD revenue contribution in the fourth quarter.
Speaker Change: Thank you.
Speaker Change: Thank you one moment for your next question.
Speaker Change: Our next question comes from Jeff Jeffrey Hung of Morgan Stanley. Your line is now open.
Speaker Change: Hi, This is Michael Rehaut answer Jeff hung. Thank you for taking my question and congrats on all the progress in all of the programs.
Speaker Change: <unk>.
Speaker Change: Added the Cardinals topline in <unk>.
Speaker Change: Do you plan to share like baseline characteristics, maybe like time since diagnosis or can you comment on maybe what percent of patients are going into the OLED.
Speaker Change: Yes, Michael Thanks for the question so typically with our topline results, we will get the key baseline characteristics and results with regards to the key endpoints we've seen excellent.
Speaker Change: Continuation of patients as they roll into the O N E.
Speaker Change: Once they finish the Cardinals.
Okay. Thank you and then maybe for the long term extension studies.
Speaker Change: Why do you think you start to see the meaningful separation more in the extension studies, but it's like a rapid impact on Empire as expected or could you.
Speaker Change: <unk> like add some more color on how it particularly known as like influencing the trajectory there.
Speaker Change: I think what we're seeing is a continued benefit in terms of preservation of function as we reported when we ride out the 72 week placebo controlled study and we saw an approximately 50% slowing and progression in the treatment group relative to the placebo group at three years, we're still seeing that 50% benefit 50%.
Slowing the progression as we can.
Speaker Change: Reported any patients who started on particularly on the head.
Speaker Change: A $3 seven point decline, whereas in the natural history cohort there was a seven and a half point decline. So we're.
Speaker Change: Seeing a continuous benefit in terms of preserving function and slowing disease progression.
Speaker Change: Thanks, so much.
Speaker Change: Thank you one moment for our next question.
Speaker Change: Our next question comes from Brian Abrams of RBC capital markets. Your line is now open.
Speaker Change: Hey, good evening, thanks for taking my questions.
Speaker Change: I guess, one on Saturday afternoon, and one on <unk> just step after I'm curious if you could maybe talk a little bit more about the reactions from providers patients and payers to to the lancet data and in particular, maybe elaborating on some of the payer.
Speaker Change: Research communication.
Speaker Change: Given your confidence in the potential for the premium pricing and then on Trans learner. Just wondering if you had any sense of the potential timelines for the next review steps there are posted in the U S and Europe and whether you think we might hear some updates in 2025.
Speaker Change: Yeah sure Brian Thanks for the question. So let me tackle Trans Lora first and then I'll pass the second question.
Eric Powell: Question I'll turn it over to Eric So first on <unk>. So you asked about the U S and the EU.
Eric Powell: Are you aware of course in that period now of approximately 67 days following the CHP opinion, where the European Commission does its assessment of whether or not they want to adopt the opinion. So we don't expect to hear anything further for at least approximately 67 days as we've.
Eric Powell: About turns of auto remains authorized in this period and of course, we're making sure that we continue to provide the drug to patients and make it commercially available while authorization remains in the U S. As we've talked about given the fact that this is a resubmission of the NDA that had initially been filed a protest in 2016, many many years ago.
Eric Powell: Nonetheless, the FTA.
Eric Powell: Is not bound by a clock in terms of doing this review. However, we expect just given what's involved in the evaluation of a resubmission, which is just the portions related to efficacy package.
Eric Powell: We would expect that they'll get to the review in the next.
Eric Powell: The next few months so as soon as we have more information on the specific timeline you can provide any clarity we will.
Speaker Change: Regarding <unk>, let me just make the first comment that obviously, we were quite excited about the lancet publication I think thats, a true testament to have differentiated.
Speaker Change: Taryn is with its dual mechanism of action not only in its ability to act as a precursor to the beach for co factor, but the fact that it has a second chaperone mechanism of action, which allows it to provide benefit to patients who are not being quite responsive, which again is a really important element of why we're seeing such significant effect.
Speaker Change: Across the board in PKU patients have also they already have a variety of genotype.
Speaker Change: The physicians are very excited about this I think they see the lancet publication as being consistent with what they believed which is that this is.
Eric Powell: Transformative therapy that can meet the significant unmet need for PKU patients, let me turn it over to Eric to talk a little bit more about our payer discussions and how we're thinking about.
Eric Powell: Potential pricing.
Eric Powell: Yes, Thanks, Matt and thanks, Brian for the question.
Eric Powell: Yes, you can say, we're really pleased with the publication of affinity we've been able to engage in a lot of peer activity, including market research, but also doing a lot of landscape research and many of the countries where we're sequencing.
Eric Powell: Clearly what payers have told us is that they see a differentiation based on its efficacy profile.
Eric Powell: Seeing that more than eight out of 10 patients can actually achieve therapeutic goals.
Eric Powell: And importantly that sea levels and those goals can be measured rapidly.
Eric Powell: With a very high unmet need we know that there is close to 90% of the population.
Eric Powell: That is not on medical therapy at this time, there is a high unmet need.
Eric Powell: And payers see that and they are willing to pay a premium price both in Europe as well as in the U S. Because they know that they can achieve.
Eric Powell: Are they can actually see the levels of therapeutic.
Eric Powell: Literally in a matter of weeks with top tier tariff.
Eric Powell: Obviously, our pricing strategy is evolving but as we know.
Eric Powell: We've actually looked at a number of different analogs.
Eric Powell: Including many that have been recently launched that are clearly differentiated and we believe that having a premium price over existing branded therapies can be achieved in many of these markets.
Eric Powell: Finally, I'll, just say that pricing strategy really it's not going to be announced until we get much closer to launch.
Eric Powell: And then when we have the final label.
Speaker Change: That's really helpful. Thanks, Eric Thanks, Matt.
Speaker Change: Thank you.
Speaker Change: Our next question comes from Ellie Merle of UBS. Your line is now open.
Ellie Merle: Hey, guys. Thanks for taking my question.
Ellie Merle: Can you talk a little bit more about the potential path forward and the potential range of outcomes in terms of the feedback that you could specifically get from this type C meeting.
Ellie Merle: And if the FDA is not supportive of the use of Huntington line is I'm, sorry, I guess I guess, what would the potential range of outcomes from near D. In terms of what the clinical endpoint study and path forward.
Ellie Merle: For registration could look like.
Ellie Merle: And I guess in that scenario, where that is not supportive of the use of a surrogate.
Ellie Merle: Are you thinking about your strategy around whether you had run.
Ellie Merle: A full phase III on your own or Luxor and potential partners.
Ellie Merle: Yeah.
Speaker Change: Okay. Thanks for the question Gary.
Speaker Change: Obviously, we're optimistic about the ability to have a productive discussion with the agency regarding the potential for Huntington lowering to serve as a surrogate endpoint because it.
Speaker Change: Really meets what they ask for in terms of a surrogate endpoint that is evidence that shows that reductions in Huntington protein can have clinical benefit is a specific threshold associated with that which we're able to achieve and just taking.
Speaker Change: Consideration of the disease itself and it's a monogenetic disease, that's caused by a toxic mutant Huntington protein and we're able to address that I think fits very squarely in the way they have thought about surrogate endpoints and other disorders.
Speaker Change: It isn't implicated protein and a drug targets a protein for example, dystrophin DMD.
Speaker Change: So believe that the agency.
Speaker Change: Has it had an interest in leveraging the accelerated approval pathway for Neurodegenerative disorders lifetime continued disease, where there is significant unmet medical need and establishing efficacy. It takes a bit time. So if there is a safe therapy that has shown the ability to impact a marker of disease that is going to be important.
Speaker Change: To that disease pathology that certainly could open the door.
Speaker Change: For an accelerated approval and again, we believe that.
Speaker Change: PTC $5, eight and lowering Huntington protein certainly fits at.
Speaker Change: At the same time, we're also having a discussion about endpoint strategy for an efficacy trial because what we wanted to do is be in a position to move forward with that efficacy trial as quickly as possible.
Speaker Change: Regardless of their.
Speaker Change: The outcome of that meeting for example, we could meet with FDA and they can say these initial data from the first 30 or so patients are pivoting. She looked very good once you come back with the complete data set.
Speaker Change: <unk> well revisit the surrogate endpoint discussion in the spring that's a possible outcome and if that occurs we still want to be ready to put in place and get started on the efficacy trial, because we're going to need that efficacy trial to be initiated either as part of an accelerated approval or as you say it.
Speaker Change: Situations, where the doors shut on a surrogate endpoint, we want that study up and running so we can get to the finish line as quickly as possible.
Speaker Change: In terms of.
Speaker Change: Conducting this study look we said all along that we have.
Speaker Change: Positioning the company and built a balance sheet to be able to conduct that phase III study and we have the infrastructure and capital to do that.
Speaker Change: Our team has worked incredibly closely with Huntington's disease community, including the patient groups and physicians around the world.
Speaker Change: Who are incredibly enthusiastic that came participating in the study whether that's as an investigator for the patients who want to be in that study either because they didn't have the opportunity for the previous study where they've seen the data now and really want to be part of the Huntington lowering trial of an oral therapy, which is easily administered.
Speaker Change: And has shown to be safe and well.
Speaker Change: All tolerated.
Speaker Change: So we look forward to being able to advance that trial again, we will do it either way, whether we have whether it becomes dull registration trial or the confirmatory start to study in the context of an accelerated approval.
Speaker Change: Great Thanks, and just a.
Speaker Change: My follow up I think you mentioned that you had a question to type C meetings with the FDA, but that the FDA asked the combined.
Speaker Change: Maybe this is just let's just call, but can you share maybe any context around that.
Speaker Change: Maybe what this might suggest.
Speaker Change: Yes.
Speaker Change: This is purely logistical we submitted the meeting request roughly the same time.
Speaker Change: And as you know, it's kind of be the same people who.
Speaker Change: We view discuss and provide their feedback.
Speaker Change: As proud as we are of how well we have been collaborating with the agency in mid Air Division I think for logistical reasons I'd, rather just have one meeting rather than to where the issues have such overlap and it's the same people involved. So we look forward to having that one meeting in December.
Speaker Change: Got it thanks.
Speaker Change: Thank you.
Speaker Change: Our next question comes from Joel Beatty of Baird. Your line is now open.
Speaker Change: Thanks.
Speaker Change: <unk> learned what person.
Speaker Change: Alright.
Speaker Change: The current ex U S sales would not be affected by any potential.
Speaker Change: A commission decision and also how long.
Those sales potentially last into the future.
Speaker Change: Yes sure. Thanks for the question as we've talked about in 2023 roughly 40.
Speaker Change: 6% of Translarna revenue was from Europe itself.
Speaker Change: That would be the revenue that we think that would be at risk in the event that the EC adopts the CHP opinion now we start going forward that there is a possibility in certain countries that we can leverage.
Speaker Change: Inpatient or other individual country pathways, there's still make commercial product available.
Speaker Change: But obviously that will be something that will play out on the other side of an AC adoption, but again its that European revenue that we would see at risk.
Speaker Change: And only that revenue.
And how long would exclusivity.
Speaker Change: Other reasons.
Speaker Change: It varies we have in many regions exclusivity out to 2029.
Speaker Change: And then one last question for Patrick So taxed in the U S. Do you expect to add Tom.
Speaker Change: Hard to know.
Speaker Change: We will submit the package.
Speaker Change: We expect a submitted in December we would expect there to be a priority review.
Speaker Change: Find out at that point, whether or not there'll be an ad com.
Speaker Change: Certainly the FDA is quite familiar with Friedrich ataxia, having already approved therapy for that disease.
Speaker Change: There's obviously a number of therapies in development. So they should have a great deal of understanding of the endpoints and particularly we know they have a very good understanding of the importance of the upright stability subscale with our discussions so.
Speaker Change: No that they would need to have an AD com from the standpoint of us being novel in terms of.
Speaker Change: First in disease therapy, or a first time that they've looked at the same point.
Speaker Change: But if there is an AD com will be ready to support.
Speaker Change: Thank you.
Speaker Change: Our next question comes from Sami Corwin of William Blair. Your line is now open.
Speaker Change: Congrats on the quarter and thanks for taking my question.
Speaker Change: As you look ahead for the launch of Sofia Taryn.
Speaker Change: Are you envisioning specific patients to be early adopters and what are your assumptions in terms of the percentage of patients that may go on each route who would be classic call and based on your conversations with payers.
Speaker Change: Have they kind of disclose that many of their covered patients would have to step through a generic clued math and then I have a follow up.
Speaker Change: Yeah sure let me, let me start and I'll turn it over to Eric as we talked about we see tab.
Eric Powell: Several segments of the population that would be early adopters, including those that are kuban non responders, which would be classical PKU patients or those that tried on <unk> as well as those who have been on VH for and not pad optimal response, so uptime and those are those are two segments that we believe.
Eric Powell: We can.
Eric Powell: Usually access just based on the strength of data from from the Infinity.
Eric Powell: Study and as we heard from Kols and our commercial details last summer.
Eric Powell: Saying they wanted to drive all of their patients, while CPA taryn, regardless of whether they fit into those buckets, but let me pass over to Ara, we can talk a bit more about how payer dynamics can play into that.
Speaker Change: Yes, Jeremy.
Speaker Change: So the question is surely is.
Speaker Change: Is an unmet need market right now what we've seen is 90% of the patients don't get medical treatment they need.
Speaker Change: Tried and failed or discontinue for a variety of reasons and the current standards of care are inadequate and physicians see that but also payers see that so it's not a very difficult conversation when you get when we when we've discussed this with payers around the world in these key markets also with U S payers.
Speaker Change: There is a substantial number of patients that can benefit from treatment from a highly efficacious and oral treatment that can be easily taken second.
Speaker Change: As I mentioned earlier, we could have.
Speaker Change: Premium pricing strategy simply because they.
Speaker Change: They recognize that these patients do not have adequate treatment, they're not control with diet theyre not controlled with <unk>.
Speaker Change: Current treatments.
Speaker Change: And.
Speaker Change: They can actually measure.
What efficacy is very quickly and rapidly. So this isn't something that is a burden in terms of the cost. They can cc level reductions very rapidly. They can measure measure died liberalization. They can also measure how many patients actually get to therapeutic goals. So everything that we've presented has been supported by the.
Speaker Change: Affinity affinity data and.
Speaker Change: Head to head study so far has been well received by the payer community and when we've talked about premium pricing strategy, we definitely know that physicians.
Speaker Change: And patients already know the access pathway, there, we'll be able to get access to treatment quickly and rapidly.
Speaker Change: To your question about step edits I think it's a very simple and easy thing because again fee.
Speaker Change: Fee levels can be measured very quickly so step edits and we can get patients quickly onto subdued Darren through prior authorizations and rapidly through fee key measurements.
Speaker Change: So overall right now the response has been overwhelming by the affinity data and.
Speaker Change: The PKU community right now is very excited to have a new treatment option.
Speaker Change: Great. Thank you and I think the Hemic pointed out the $1 billion opportunity in PKU before and that was with the assumption of modest penetration. So I guess I'm wondering.
Speaker Change: What are your internal assumptions for penetration to breach that 1 billion opportunity anyway.
Speaker Change: Okay.
Speaker Change: Yes, Sir yes.
Speaker Change: Yes, I think right now are I would say first of all we are preparing for global launch alright, So theres going to be contribution everywhere, but we believe that there is a $1 billion opportunity in the U S alone.
Speaker Change: If you look again, if you would.
Speaker Change: Somewhere between 17 to 20000 patients with 90% of them are not medical treatments are very modest penetration, even somewhere between 20% to 30% would get you there.
Speaker Change: Especially if it's supported by premium pricing strategy, which we believe we can get.
Speaker Change: And while we believe a $1 billion launch in the U S alone is an important opportunity we're not ready to guide yet on the product we're going to be as we get closer and closer to launch we will be guiding on product revenue.
Speaker Change: We're excited with this opportunity because of the substantial unmet need.
Speaker Change: And how many patients currently are not medical treatments.
Speaker Change: Got it thank you.
Speaker Change: Thank you.
Speaker Change: Our next question comes from Peyton bonds back.
Speaker Change: TD Cowen Your line is now open.
Speaker Change: Maybe one for Joe and congrats on another strong quarter and thanks for taking our questions I guess on the particular known application for December what's outstanding critical mission and then I know the FDA requested that natural history, when you're designing your analysis.
Speaker Change: Have you sent that to the FDA have been reviewed didn't give any feedback.
Speaker Change: And then I've got a backlog.
Speaker Change: Yes, thanks for the questions Pete and I am not sure I heard the second part of your question about does it.
Speaker Change: The statistical analysis plan is I wish you asked.
Speaker Change: I was asking.
Speaker Change: Whether or not after the readout came in October.
Speaker Change: The fda's view that or you've sent that to the FDA and if maybe give any feedback on it.
Speaker Change: Okay. So in terms of outstanding items were just finishing up all of the summary reports and write ups from the long term extension study and obviously, we got those results in October it takes some time to just write that all up format at the table as.
Speaker Change: As intended and we had had our pre NDA interaction with the agency also in October where we reviewed with them the contents of the package.
Speaker Change: What we are planning to show in terms of long range.
Speaker Change: Long term outcome data that you've seen some of the data.
Speaker Change: And importantly, they had previously agreed to the statistical analysis plan, we were using both for the analysis.
Speaker Change: Of the move in long term extension as well as the long term extension data from the earlier placebo controlled study completed a few years ago. So they know exactly how we're going to analyze the data how we were going to do how we went about doing the matching the endpoint strategy with the primary end point being change in MSR scale over time.
Speaker Change: And so we were fully aligned with them on that and as I mentioned they had seen some of the data we don't expect any additional feedback from the agency.
Speaker Change: Until after we submit the NDA.
Speaker Change: Great that makes a lot of sense and then I guess really quickly pivoting tepes data.
Speaker Change: All the necessary inspections have been done from a manufacturing facility and then assuming approval next week, how quickly could you watch the therapy and how it identification con.
Speaker Change: Yeah. So.
Speaker Change: With the <unk> date of November 13, any inspections are long since done.
Speaker Change: Everything went quite well from an inspection standpoint, and we look forward to.
Speaker Change: From the agency next week when we obviously, we remain optimistic about the potential for approval based on our interactions and we look forward to being able to bring this transformative therapy.
Patients in the U S as quickly as possible.
Speaker Change: Great. Thank you.
Speaker Change: Okay.
Speaker Change: Thank you.
Speaker Change: Your next question comes from Paul Choi of Goldman Sachs. Your line is now open.
Speaker Change: Okay.
Speaker Change: Hi, good afternoon, and congratulations on the quarter. Thank you for taking our questions.
Speaker Change: I wanted to maybe just follow up on with regard to take one on payer discussions here.
Speaker Change: Maybe just comment on what payer feedback has been and just sort of how you maybe potentially sort of thinking about pricing relative Scott claris here for the adult population.
Speaker Change: And secondly, the cash position remains very healthy here and Matt I just wanted to sort of ask what your appetite is currently four.
Speaker Change: Potential external business development versus funding.
Speaker Change: Upcoming multiple launches here in 'twenty five thank you very much.
Speaker Change: Yes, thanks for the questions. Paul obviously, we're a bit early to be having the payer discussions are in particular, we're focused now on getting getting the packaging, but I would say that look.
Speaker Change: There's a clear unmet need for pediatric and adolescent patients and we have a data package in terms of safety and efficacy that supports an important benefit for these patients as we know as well there is.
Speaker Change: Important proportion of patients who are pediatric and adolescent but his diagnosis is increasing in the field patients who are being diagnosed are increasingly young and so that need that unmet need for patients is getting stronger and we look forward to being able to provide a safe and effective therapy for those patients, but as you raise we also believe we can be a provider.
Speaker Change: A therapy for adult patients who have evidence in move Fas benefit adult patients who have evidence on the upright stability scale, which speaks directly to the ability to reduce risk of loss of ambulation, which is incredibly important for <unk> ambulatory <unk> ataxia patients of any age and of course in the long term extension studies of both move.
Speaker Change: As well as the earlier placebo controlled study, which was conducted only in adults. We have evidence of important long term benefit in slowing disease progression.
Speaker Change: Then we also have a very strong safety and Tolerability profile. So I believe we have really a differentiated product that we look forward to thinking about how.
The price and also having discussions with the payers on that.
Speaker Change: The cash position, but we worked very hard both in terms of restructuring.
Speaker Change: The balance sheet towards the end of last year, our efforts to reduce operating expenses and move the company towards cash flow breakeven I think having a strong cash balance not only enable us to.
Speaker Change: Launch the product.
Speaker Change: We would love to be in that situation, where that's happening do you think they have the capital to launch more products, but within a year in the U S and the global launch of Cepheid, Darren but we also of course remain opportunistic about business development.
Speaker Change: We have the cash we have on hand, we also have the ability if we needed to draw down.
Speaker Change: Some of the additional royalty monetization.
Good.
Speaker Change: With royalty pharma, if we saw an opportunity that fits well enter into.
Speaker Change #100: Thank you our portfolio look we're in a position where we are we could be launching many products in the U S. In the short term and so now we need to think about how we could use business development.
Speaker Change #100: Strategic way to help.
Speaker Change #100: Fill out the commercial portfolio ex U S and also think about any other opportunities that can cassette.
Speaker Change #100: Very easily into our existing commercial infrastructure, even development infrastructure.
Speaker Change #101: Great. Thank you.
Speaker Change #102: Thank you.
Speaker Change #103: Our next question comes from Gena Wang of Barclays. Your line is now open.
Speaker Change #104: Thank you.
Gena Wang: Few quick questions. The first one regarding separate Kieran.
For the July 29, 2025, just wanted to confirm that FTE confirm there will be no no.
Speaker Change #106: No Adam.
Speaker Change #106: The second question is regarding the Translarna you mentioned that right now the 67 days.
Speaker Change #106: So can you walk us through like see after 67 days if the decision is.
Speaker Change #106: Ruble what will be the next steps and how long would that take the process well.
Speaker Change #106: We really will see the actual.
Speaker Change #107: Impact on the revenue in Europe and related questions, maybe unrelated Brazil I know you are under independent assessment. They are they have independent approval process backing April any update there regarding the renew process there.
Speaker Change #106: Lastly, very quickly regarding ADC next week, you will get approval hopefully keep our fingers crossed.
Speaker Change #107: How ready are you regarding the.
Speaker Change #108: Manufacturing, how many doses you already stocked and how quick do you think you can get ready to how many patients already identified in the U S.
Speaker Change #110: Jamie. Thank you for the question. So let me walk through them one by one first on the <unk> date for <unk> is July 29, 2025, we do not expect it to DNA com.
Speaker Change #110: There's been no indication of a problem and again, we're in a situation where the endpoint of the trial reduction of phenylalanine is published.
Speaker Change #110: Published as being one of the blood tests that can support full approval.
Speaker Change #110: Obviously the agency understands PKU if they are approved to therapy as support for PQ. So I think this is not one where we would expect to have an ad com.
Speaker Change #110: The clear efficacy in.
And the data package is also.
Speaker Change #110: Very little question, I think that what needed to be brought to an ad com.
Speaker Change #110: Your second question was about <unk> and the timeline in the EU look the 67 days is an approximately 67 days as we learned is the first time, we went through this in the spring.
Speaker Change #110: We expected 67 days to take us from late January to.
Speaker Change #110: March early April and it wasn't until late may or that we actually got confirmation that the AUC had made the decision to not adopt the opinion and some things back to the <unk>. So I think thats the approximate 67 days in <unk>.
Gena Wang: Gena, we can't say for sure what happens on the other side of that guess is kind of dependent with the European Commission says they could adopt the opinion and say, we're going to adopt it and adopt it and for immediate implementation or we're going to adopt it with delay implementation. We're in a situation here it's important to.
Gena Wang: Realize that this is not being this is not come about due to a safety issue. So there is no patient risks here.
Gena Wang: In terms of there being an urgency to get the product off the market.
Gena Wang: So I think that's.
Gena Wang: Also.
Gena Wang: Provide a little bit of flexibility as well in terms of what exactly happens in terms of.
Gena Wang:
Gena Wang: This steps following adoption of the negative opinion.
Gena Wang: And then of course, it's sent back to the <unk> and that's a whole lot to see what those instructions are as well so still a lot of unknown. There to 67 days is approximate and obviously, we'll learn more as we reached.
Gena Wang: Timeline.
Gena Wang: In terms of Brazil. It is an independent assessment and has been ongoing.
Gena Wang: We have not gotten any additional feedback.
Gena Wang: Problem and visa in what we do know is that we had two orders in Brazil bulk orders won in the second quarter one in the third quarter.
Gena Wang: We obviously view that as a good sign.
Gena Wang: That there is obviously a strong interest.
Gena Wang: So the minister of health to ensure that patients in Brazil remain on trends more or less.
Gena Wang: And then in terms of ADC.
We're ready I mean again, we had the EU approval in 2022, we've been we've had approvals.
Gena Wang: The recent approval in Brazil free for NBC deficiency with approval in Taiwan approval in the U K.
Gena Wang: We're doing cross border care, we're in the process of treating patients around the globe. So we have more than adequate supply and again once we.
Gena Wang: See the label will understand what the addressable patient market could look like in the U S. And we have we believe more than enough supply on hand to treat.
Gena Wang: <unk> III patients well through the next one to two years.
Speaker Change #111: Great. Thank you.
Speaker Change #112: Thank you.
Speaker Change #113: Our next question comes from Danielle Brill of Raymond James Your line is now open.
Great. Thank you so much for the question I just have a quick clarification from an earlier question I'm curious what the Translarna NDA submission is technically a resubmission under protest.
Speaker Change #113: Why wouldn't it be a new NDA since you now have new data there.
Speaker Change #113: Oh, sorry, one thanks, so much.
Speaker Change #114: Yeah, Danielle this goes back to 2016 2017 and 2016.
Speaker Change #114: <unk> was filed over protest the agency reviewed it and issued a complete response letter.
Speaker Change #114: Main issue in a complete response letter was the need to conduct an additional adequate and well controlled trial to establish efficacy.
Speaker Change #114: 741 addresses what was in that CRM and therefore this is technically a resubmission of that NDA now fulfilling the deficiency identified that led to the complete response previously in 2000.
Speaker Change #115: In 2016 does that makes sense.
Speaker Change #116: It does thanks, Matt I appreciate the clarification.
Speaker Change #116: Okay.
Speaker Change #116: Okay.
Speaker Change #116: Thank you. Our next question comes from Joe Schwartz.
Speaker Change #117: Leerink partners. Your line is now open.
Speaker Change #118: Hi, Thanks, so much I have a question about the <unk> and one on <unk>. So far we've heard that while some patients think that they've benefited more patients seem to not be blown away by site clearances efficacy. So we're wondering how that might influence how patients might feel about the <unk>.
Speaker Change #119: Just following in his footsteps and works further downstream how do you think this market is evolving now in terms of patient demand and retention for new treatment options as well as their potential to switch or try a combo with citic went out.
Speaker Change #120: Yes. Thanks for the question Joe first of all just going on.
Speaker Change #120: Upstream of at least Mechanistically of where Sky Claris is in terms of its effects on fibrosis pathway, but I would say look I think in the clearly there is no option for pediatric and adolescent patients.
Speaker Change #120: With by Claris, given the age limitations of the label.
Speaker Change #120: And in terms of the adults.
Speaker Change #120: I think it's always hard.
Speaker Change #120: With drugs that slow disease progression in patients perceptions of if they're getting better or not it's always hard to know, but needless to say I think that we would certainly be able to provide a therapeutic option, where we believe we can provide benefit to patients. The data from <unk> show an effect not only in slowing of disease progression, but also a significant effect on the fatigue scale.
Speaker Change #120: Which we know is the number one symptomatic complaint patients with <unk> ataxia.
Do I think that people will be interested in combo therapies I do.
Lot to be sorted out there of course in terms of.
Speaker Change #120: Payers and such but I think look when anytime you have a complex disorder like Friedrich ataxia. It would make sense that you would mean, you likely would need more than one therapy.
Speaker Change #120: And it might be that two therapies could have combined effect.
Speaker Change #120: But look we look forward to being able to offer that.
Speaker Change #120: I think the FAA patients, particularly the kids and adolescents, who desperately need a therapy I mean look if youre going to slow disease progression. The best thing to do is to start.
Early as possible. So that you can enjoy the benefits of slow progression as much as possible.
Speaker Change #120: For adults, we welcome the opportunity to provide them with a safe and effective therapy as well.
Okay. Thank you and then for targeting zero ptosis in AOS can you give us some context for what you're trying to achieve mechanistically is there a particular threshold you're looking to meet based on any preclinical or natural history data that supports your therapeutic hypothesis.
Speaker Change #121: And have you done anything in the Cardinal study too.
Speaker Change #121: Select patients that might best response.
Joe: Yes, it's really really good question Joe obviously this is the first in disease study we have.
Joe: Each lots of statin AOS and I expect we'll learn a lot on the other side about how we tenants.
Joe: Enbridge can could possibly go but look the rationale for fair coast targeting Faruk doses very well established and ALS is a number of different preclinical studies that clearly show that if you target for ptosis.
Joe: Thats by inducing overexpression of GPA explore which is a key enzyme in the <unk> pathway you had significant phenotypic benefit.
Joe: Similarly, if you augment the <unk> process to any number of insults.
Joe: And.
Joe: The accelerated decline in ALS, the whole concept of SB, one in superoxide dismutase speaks directly to being able to affect oxidative stress of irrelevance of oxidative stress in AOS.
Joe: Theres, a great bit of data to support.
Joe: The rationale for chartering ptosis.
Joe: AOS is incredibly complex and aggressive disease.
Joe: We hope to be able to provide a therapy that could slow that progression by targeting what's known to be a key pathologic elements.
Speaker Change #123: Thank you.
Speaker Change #124: Thank you.
Speaker Change #125: This concludes the question and answer session I would now like to turn it back to Doctor Matthew Klein CEO for concluding remarks.
Matthew Klein: Yes. Thank you very much operator, let me just follow on the last question to Joe and just share with everyone that.
Speaker Change #125: As we are.
Speaker Change #125: Getting close to having data for the future lots that cardinal's ALS study. Following this call we're going to not be taking any questions.
Speaker Change #125: Our quiet period for the Cardinal study just because of where we are in terms of.
Speaker Change #125: Being close to having data in the next.
Speaker Change #125: Period of time.
Speaker Change #125: Overall again, thank you everyone for joining the call.
Speaker Change #125: We're incredibly excited about the strength of the quarter. The revenue performance the ability of our teams to continue to execute at an incredibly high level across all parts of the company, whether it'd be on the commercial side on the clinical and the regulatory side and all around.
Speaker Change #125: Look forward to keeping you updated as we still have a number of important milestones this year.
Speaker Change #127: And thank you all for joining the call and have a good evening.
Speaker Change #128: Thank you for your participation in today's conference. This does conclude the program you may now disconnect.
Speaker Change #128: Okay.
Speaker Change #128: [music].
Speaker Change #128: Okay.
Okay.
Speaker Change #128: Yes.
Speaker Change #128: [music].
Speaker Change #128: Okay.
Speaker Change #128: [music].