Q1 2025 uniQure NV Earnings Call
Yeah.
Operator: Good day and thank you for standing by. Welcome to the Uniqure First Quarter 2025 Earnings Conference Call. At this time, all participants are in a listen-only mode.
Speaker Change: Good day and thank you for standing by welcome to the unit. Your first quarter 2025 earnings Conference call. At this time all participants are in a listen only mode. After the speaker's presentation there'll be a question answer session to ask a question. During the session you will need to press star one one on your telephone.
Operator: After the speaker's presentation, there'll be a question and answer session. To ask a question during the session, you'll need to press star 1-1 on your telephone. You will then hear an automated message device in your hand... 2 or 3 are questions, press star 118. Please be advised that today's conference is being recorded.
Speaker Change: the question during the session, you'll need to press star one one on your telephone. You will then hear an automated message advising your hand is raised. So, it's for your question, press star one one again.
Operator: I would now like to turn the conference over to your speaker for today, Kara Russo, Senior Director of Investor Relations. Thank you.
Speaker Change: Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker for today. Cara Russo, Senior Director of Investor Relations, please go ahead.
Kara Russo: Good morning and thank you for joining us for Uniqure's inaugural quarterly earnings call. Earlier this morning, Uniqure released its financial results for the first quarter of 2025. The press release is available on the investors and media section of our website at uniqure.com and our 10-Q will be filed with the SEC later today.
Keara Russo: Thank you. Good morning, and thank you for joining us for Uniqure's inaugural Quarterly Erning Call. Earlier this morning, Uniqure released its financial result for the first quarter of 2025.
Keara Russo: The Francher Least is available on the Investors and Media section of our website at Uniqure.com and our 10Q will be filed with the SEC later today.
Kara Russo: Joining me on the call this morning are Matt Kapisa, Chief Executive Officer, Dr. Waleed Abisab, Chief Medical Officer, and Christian Klempt, our Chief Financial Officer. After our formal remarks, we'll open up the call for Q&A. Please know that we'll be making forward-looking statements during this investor call. All statements, other than statements of historical fact, are forward-looking statements. They are based on management's beliefs and assumptions and on information available to management only as of the date of this conference. our actual results could differ maturely from those anticipated in these forward-looking statements for many reasons, including, without limitation, the factors described in Uniqure's most recent SEC filing.
Keara Russo: Joining me on the call this morning are Matt Capista, Chief Executive Officer, Dr. Wallyd Abisob, Chief Medical Officer, and Christian Clempt, our Chief Financial Officer.
Keara Russo: After our formal remarks, we'll open up the call for Q&A.
Speaker Change: Please know that we'll be making forward-looking statements during this investor call. All statements, other statements of historical fact, are forward-looking statements. They're based on management's beliefs and assumptions, and on information available to management only as a date of discount and cost.
Speaker Change: Our actual results could differ materially from those anticipated in-use for the containment for many reasons, including without limitations the factors described in Uniqure's most recent SEC violence.
Kara Russo: Given these risks, you should not put undue reliance on these forward-looking statements, and we assume no obligation to update these statements, even if new information becomes available in the future.
Speaker Change: Given these risks, you should not first undo your reliance on these four living statements, and we have seen no obligation to update these statements, even if new information becomes available in the future.
Matt Kapusta: Now let me introduce Matt Kavista, Uniqure's CEO. Thanks, Chiara. And good morning, everyone. Thank you for joining our call today.
Now let me introduce my cabbages to Uniqure's CEO .
Matt Kapusta: I'm very pleased to share with you our first quarter 2025 financial results and provide an update on the progress of our four clinical stage gene therapy programs. As we continue to advance our pipeline and drive AMT 130 closer to a planned BLA submission, we are commencing quarterly earnings calls to provide regular updates on our progress.
Matt Kavista: Thanks, Chiara, and good morning, everyone. Thank you for joining our call today.
Speaker Change: I'm very pleased to share with you our first quarter 2025 financial results and provide an update on the progress of our four clinical stage gene therapy programs.
Speaker Change: As we continue to advance our pipeline and drive AMT 130 closer to a planned BLA submission, we are commencing quarterly earnings calls to provide regular updates on our progress.
Matt Kapusta: I'm joined today by Dr. Waleed Abisab, our Chief Medical Officer and Christian Klent, our Chief Financial Officer, who each provide updates on their respective areas. I'd like to begin by reflecting on what has been a truly remarkable last 12 months for Uniqure and why we believe 2025 has the potential to be a transformational year for the company with multiple important value-driving milestones ahead. Last July, we presented compelling two-year data from our Huntington's program, demonstrating AMT130's potential to meaningfully slow disease progression. Then, in November, we announced alignment with the FDA on key elements of an accelerated approval pathway, which include the use of a natural history external control and CU-HDRS as a registrational, intermediate, clinical, and.
Speaker Change: On join today by Dr. Walid Abisab, our Chief Medical Officer, and Christian Clement, our Chief Financial Officer, who each provide updates on their respective areas.
Speaker Change: I'd like to begin by reflecting on what has been a truly remarkable last twelve months for Uniture and why we believe 2025 is the potential to be a transformational year for the company with multiple important value driving milestones ahead.
Speaker Change: Last July , we presented compelling two-year data from our Huntington's program demonstrating AMT-130's potential to meaningfully slow disease progression.
Speaker Change: Then in November , we announced alignment with the FDA on key elements of an accelerated approval pathway, which include the use of a natural history external control and CUHDRF as a Registrational Intermediate Clinical Endpoint.
Matt Kapusta: In 2025, we continue to make strong progress with AMT 130. In April, the FDA granted breakthrough therapy designation, underscoring both the urgent need in Huntington's disease and the strength of our clinical data. In recent weeks, we've had multiple productive interactions with the FDA focused on preparing for a planned BLA submission.
Speaker Change: In 2025, we continue to make strong progress with AMT 130. In April , the FDA granted a breakthrough therapy designation.
Speaker Change: underscoring both the urgent need in Huntington's disease and the strength of our clinical data.
Speaker Change: In recent weeks, we've had multiple productive interactions with the FDA focused on preparing for a planned BLA submission, and we look forward to providing a detailed regulatory update later this quarter after we receive formal meeting minutes.
Matt Kapusta: And we look forward to providing a detailed regulatory update later this quarter after we receive formal meeting minutes. We're also making good progress towards initiating CNC process validation, another important step in support of the planned BLA submission. In addition to the strong progress made with our Huntington's program, we've also expanded our clinical pipeline with the initiation of three additional clinical studies in refractory temporal lobe epilepsy, or TLE, Fabry disease, and SOD1 ALS. In the quarter, we continue to advance enrollment in both our Fabry and SOD1 ALS studies, and we look forward to presenting initial Fabry data in the second half of 2025.
Speaker Change: We're also making good progress toward initiating CMC process validation, another important step into the plan BLA submission.
Speaker Change: In addition to the strong progress made with our Huntington's program, we've also expanded our clinical pipeline with the initiation of three additional clinical studies in refractory temporal lobe epilepsy or TLE, fabric disease, and sub-1ALS.
Speaker Change: In the quarter, we continue to advance enrollment in both our FABRI and SOG-1 ALF studies, and we look forward to presenting initial FABRI data in the second half of 2025.
Matt Kapusta: We've completed enrollment of the first two dose cohorts in our SOD1 ALS trial, and currently have several patients screening for our TLE clinical study.
Speaker Change: We completed a Roman of the first two dose cohorts in our Saaduan ALS trial and currently has several patients screening for our TLE clinical study. We also expect to share preliminary results from the first patient dose in the TLE study at an upcoming scientific meeting later this month.
Matt Kapusta: We also expect to share preliminary results from the first patient dose in the TLE study at an upcoming scientific meeting later this month. Lastly, Uniqure continues to be in a strong financial position with more than $400 million of cash on hand as of March 31st. Last year, we significantly reduced our cash burn with the divestiture of our GMP manufacturing facility and organizational restructuring. We further strengthened our balance sheet with the completion of a targeted $80 million follow-on offering earlier this year. Together, these actions provide us the financial flexibility to advance our pipeline, including the planned BLA submission and launch of AMT 130, and support key data readouts from our other clinical programs. Overall, I'm incredibly proud of the progress our team made in the first quarter.
Speaker Change: Lastly, Uniqure continues to be in a strong financial position with more than $400 million of cash on hand as of March 31st.
Speaker Change: Last year we significantly reduced our cash firm with the divestiture of our GMP manufacturing facility and organizational restructuring.
Speaker Change: We further strengthened our balance with the completion of a targeted $80 million follow-on offering earlier this year.
Speaker Change: Together, these actions provide us the financial flexibility to advance our pipeline, including the Plan BLA submission and Launcher AMT 130, and support key data readouts from our other clinical programs.
Matt Kapusta: We delivered on our key objectives and remain on track to meet the strategic goals outlined for 2025. Our continued focus and disciplined execution positioned us well for what we expect will be a transformative year for Uniqure.
Speaker Change: Overall, I'm incredibly proud of the progress our team made in the first quarter. We delivered on our key objectives and remain on track to meet the strategic goals outlined for 2025.
Speaker Change: or continues focus in Discipline's Execution, position us wealth and what we expect will be a transformative year for Uniqure. I'll now turn the call over to Lolly to provide a more detailed clinical update. Pauline.
Waleed Abisab: I'll now turn the call over to Waleed to provide a more detailed clinical update. Waleed? Thank you, Matt. Good morning and good afternoon, everyone. During the first quarter of 2025, we made meaningful progress across our pipeline of clinical-stage gene therapy. Let me start with AMT130. AMT-130 for the treatment of Huntington's Disease has made significant gains over the last 12 months, beginning in May of last year with the granting of the Regenerative Medicine Advanced Therapy or RMAT designation by the FDA, the first for Huntington's Disease. In July, we announced positive long-term follow-up data on AMT130 supporting dose-dependent slowing of Huntington's disease progression.
Thank you Matt.
Good morning and good afternoon everyone.
Speaker Change: During the first quarter of 2025 we made meaningful progress across our pipeline of clinical stage chain therapies.
Speaker Change: AMD 130 for the treatment of Huntington's disease has made significant gains over the last 12 months beginning in may of last year with the granting of the regenerative medicine advanced therapy or <unk> designation by the FDA the first for Huntington disease.
Speaker Change: In July we announced positive long term follow up data on empty 130, supporting dose dependent slowing of Huntington's disease progression.
Waleed Abisab: In November, we successfully aligned with the FDA on key elements for an accelerated approval pathway, including the use of the Composite Unified Huntington's Disease Rating Scale, or COHRS, functional measure as an intermediate clinical endpoint. In February of this year, we completed enrollment of all 12 patients in the third cohort of the Phase I-II trial, and I will be reviewing the short-term safety data with you in just a moment. In April, AMT 130 was granted breakthrough therapy designation by the FDA, further underscoring the potential promise of this program and the urgent need for disease-modifying treatments for this devastating condition.
Speaker Change: In November we successfully aligned with the FDA on key elements for the accelerated approval pathway, including the use of the composite unified Huntington's disease rating scale or Coa Crs.
Speaker Change: <unk> measure.
Speaker Change: As an intermediate clinical endpoint.
Speaker Change: In February of this year, we completed enrollment of all 12 patients in the third cohort of the phase one two trial.
Speaker Change: Ill be reviewing the short term safety data with you in just a moment.
Speaker Change: And April Anti-war 30 was granted breakthrough therapy designation by the FDA further underscoring the potential promise of this program and the urgent need for disease modifying treatment for this devastating condition.
Waleed Abisab: Most importantly, we believe our interactions with the FDA in both the first and second quarters of this year have been productive.
Speaker Change: Most importantly, we believe our interactions with the FDA in both the first and second quarters of this year have been productive.
Waleed Abisab: We expect to provide a more comprehensive regulatory update, including guidance on the planned DLA submission later this quarter, and additional critical data in the third quarter of this year.
Speaker Change: We expect to provide a more comprehensive regulatory update including guidance on the planned BLA submission later this quarter.
Speaker Change: And additional clinical data in the third quarter of this year.
Waleed Abisab: Now I'd like to move on to core three. Based on our experiences from cohort 1 and 2, which did not include prophylactic immunosuppression, we believe AMT130 is generally safe and well-tolerated at both doses. Code 3, which completed enrollment in February, was designed to evaluate the impact of prophylactic immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab. This cohort includes 12 patients blinded and randomized to receive either the high or low dose of AMT-130. Key observations from Code 3 are as follows. AMT130 continues to be generally well-tolerated at both doses, with no treatment-related serious adverse events reported.
Speaker Change: Now I'd like to move on to cohort three.
Speaker Change: Based on our experiences from cohort, one and two which did not include prophylactic immunosuppression. We believe <unk> is generally safe and well tolerated at both doses.
Speaker Change: Cohort three which completed enrollment in February was designed to evaluate the impact of prophylactic immunosuppression regimen.
Speaker Change: Think of dexamethasone Sirolimus and Rituximab.
Speaker Change: This cohort includes 12 patients blinded and randomized to receive either the high or low dose of MTO authority.
Speaker Change: Key observations from cohort three are as follows.
Speaker Change: And do you want 30 continues to be generally well tolerated at both doses with no treatment related serious adverse events reported.
Waleed Abisab: There were three serious adverse events related to immunosuppression, which were observed. Mania MRSA infection from an abrasion and fever, all of which resolve fully with standard of care intervention. Perioperative changes in CSF-NFL were consistent with previously reported observations, reinforcing their association with the surgical procedure. Based on these results, we believe a short, two-week course of steroids represents an appropriate and optimized immunosuppression strategy to accompany AMT-130. We view this as a favorable outcome and plan to review this data with external advisors in the near future. Importantly, we do not anticipate any impact on the potential timing of a BLA submission.
Speaker Change: There were three serious adverse events related to immunosuppression, which were observed.
Speaker Change: Uh huh.
Speaker Change: RSA infection from an abrasion and fever.
Speaker Change: All of which resolved fully with standard of care interventions.
Speaker Change: Peri operative changes in CSF NFL were consistent with previously reported observations reinforcing their association with the surgical procedure.
Speaker Change: Based on these results we believe a short two week course of steroids represents an appropriate and optimized in your immuno suppression strategy to accompany <unk> hundred 30.
Speaker Change: We view this as a favorable outcome and plan to review this data with external advisors in the near future.
Speaker Change: Importantly, we do not anticipate any impact of the potential timing for BLA submission.
Waleed Abisab: Moving on to Mesial Temporal Lobe Epilepsy, the team remains focused on driving patient enrollment in the Phase I-II trial of AMT260. Following the implementation of FDA-cleared protocol amendments to broaden eligibility, the trial now has 12 active clinical sites, with more expected to be activated throughout the year. We plan to present a case study for the first of those patients, including initial safety and exploratory efficacy data, at the Epilepsy Therapies and Diagnostics Development Symposium on Thursday, May 29.
Speaker Change: Yes.
Speaker Change: Moving onto medial temporal lobe epilepsy. The team remains focused on driving patient enrollment in the phase one two trial of empty.
Speaker Change: Empty to 60.
Speaker Change: Following the implementation of FDA cleared protocol amendments to broaden eligibility. The trial now has 12 active clinical sites with more expected to be activated throughout the year.
Speaker Change: We plan to present, a case study for the first dose patients, including initial safety and exploratory efficacy data at the epilepsy therapies and diagnostics development Symposium on Thursday May 29.
Waleed Abisab: In Fabry disease, we announced a favorable safety review by the Independent Data Monitoring Committee and have now treated a total of four patients in the Phase 1-2 study. we expect to share initial patient data in the second half of this year.
Speaker Change: In Fabry disease, we announced a favorable safety review by the independent data monitoring committee and have now treated a total of four patients in the phase one two study.
Speaker Change: We expect to share initial patient data in the second half of this year.
Waleed Abisab: Similarly, in the Phase 1-2 study of AMT 162 and SOD 1 ALS, following the review of the initial safety data, the IDMCA recommended proceeding with enrollment in the second cohort. The team has now completed enrollment in the second cohort and expects to initiate enrollment in the third cohort in the third quarter of this year.
Speaker Change: Similarly in the phase one two study of AMG 162, and solved one ILS.
Speaker Change: Following the review of the initial safety data the <unk> recommended proceeding with enrollment in the second cohort.
Speaker Change: The team has now completed enrollment in the second cohort and expects to initiate enrollment in the first cohort in the third quarter of this year.
Christian Klempt: Now I will turn the call over to Christian for a financial update. Christian? Thank you, I'll start off with sharing financial highlights of the first quarter of 2020. Please refer to the learnings press release issued this morning and a quarterly filing with the Revenue for the first quarter was 1.6 million dollars compared to 8.5 million dollars in the same period 2002. The decrease of $6.9 million in revenue was mainly from a decrease of $3.3 million of collaboration revenue and a decrease of $4 million in contract manufacturing.
Christian Klent: Now I will turn the call over to Christian for a financial update.
Speaker Change: Sure.
Speaker Change: Thank you.
Speaker Change: I'll start off.
Speaker Change: Highlights for the first quarter of 2021 please.
Speaker Change: Please refer to the earnings press release issued this morning, and our quarterly filings for additional details.
Speaker Change: Revenue for the first quarter was $1 6 million compared.
Speaker Change: Compared to $8 5 million in the same period.
Speaker Change: The decrease of $6 $9 million in revenue was mainly from a decrease of three 3 million.
Speaker Change: Elaboration revenue and a decrease of $4 million in contract manufacturing revenue.
Christian Klempt: Following the divestment of the Lexington facility in July 2024, revenue from contract manufacturing is recorded net, of course, within other cost contract manufacturing revenues were nil in the first quarter compared to 9.1 million dollars for the same period in 2005. again following the divestment of the Lexington facility in July. 4. Cost of contract manufacturing is recorded. Net of revenue within other Research and development expenses were $36.1 million in the quarter compared to $40.7 million during the same period. The majority of the cost reduction was related to a decrease in employee-related expenses and a slowdown. partially offset by a $5.8 million increase in external program spend, primarily related to the submission of a BLA for our Huntington's program.
Speaker Change: Following the divestment of Prolexic uncertainty in July 2020 for revenue from contract manufacturing is recorded net of cost within all of our expenses.
Speaker Change: Contract manufacturing revenues were nil in the first quarter compared to $9 1 million for the same period in 2024.
Speaker Change: Again, following the divestment of the excellent facility in July 2024 cost contract manufacturing is recorded net of revenue of our expenses.
Research and development expenses were $36 1 million in the quarter compared to $47 million the same period in 2024.
Speaker Change: A majority of the cost reduction was related to a decrease in employee related expenses and facility expenses.
Speaker Change: This was partially offset by $5 8 million increase in external program spend primarily related to the submission of the BLA for Huntington's program.
Christian Klempt: Selling, general and administrative expenses were $10.9 million in the quarter compared to $13.9 million during the same period.
Speaker Change: Selling.
Speaker Change: General and administrative expenses were $10 9 million in the quarter compared to $13 9 million jumped the same period in 2020.
Christian Klempt: was again primarily due to a reduction of employee-related Cash, cash equivalents, and investment securities totaled $409 million as of March 31st, 2025, compared to $367.5 million as of December 31st, 2025. The increase is primarily related to the net proceeds of $80.5 million from a first quarter follow-on offer. This strong balance sheet provides Uniqure with the resources, clinical and operational strategy, including the planned U.S. launch of the MT1.
Speaker Change: Decrease was again, primarily due to a reduction of employee related expenses.
Speaker Change: Cash cash equivalents and investment securities totaled $409 million at <unk>.
Speaker Change: For 2025.
Speaker Change: Compared to 367 5 million.
As of December 31st two.
Speaker Change: 2020.
Speaker Change: The increase is primarily related to the net proceeds of $85 million from our first quarter follow on offering.
Speaker Change: This strong balance sheet provides unit cure with the resources.
Speaker Change: Clinical and operational strategy, including the planned U S launch <unk> 30.
Christian Klempt: We expect cash, cash equivalents and investment securities will be sufficient to fund operations into the second quarter, second half of 2027.
Speaker Change: We expect cash cash equivalents and investment securities will be sufficient to fund operations into the second quarter of two.
Matt Kapusta: I now turn the call back over to Matt. Thanks for that update Christian. As you've heard today, we believe 2025 will be a transformative year for Uniqure. We continue to advance a robust pipeline of gene therapies and expect to present data from all of our clinical programs over the next 12 months. We look forward to continuing our productive engagement with the FDA on AMT 130 and expect to update you on our recent interactions, including the expected timing of a BLA submission later this quarter once we receive formal meeting minutes. In addition, we expect to share new data in the third quarter from our ongoing Phase 1-2 study to further support the planned BLA submission.
Speaker Change: 2027.
Matt Kavista: I'll now turn the call back over to Matt.
Matt Kavista: Thanks for that update Christian.
Speaker Change: As you've heard today, we believe 2025 will be a transformative year for unit here, we continue to advance our robust pipeline of gene therapies and expect to present data from all of our clinical programs over the next 12 months.
Speaker Change: Look forward to continuing our productive engagement with the FDA on AMG 130, and expect to update you on our recent interactions, including the expected timing of our BLA submission.
Speaker Change: This quarter once we receive formal meeting minutes. In addition, we expect to share new data in the third quarter from our ongoing phase <unk> study to further support the planned BLA submission.
Matt Kapusta: We're extremely excited about the opportunities ahead of Uniqure and remain focused on delivering innovative therapies that can improve the lives of the patients we serve.
Speaker Change: We're extremely excited about the opportunities ahead of unit year and remain focused on delivering innovative therapies that can improve the lives of the patients we serve.
Matt Kapusta: We look forward to keeping you updated on our continued progress.
Speaker Change: We look forward to keeping you updated on our continued progress.
Operator: With that, we will open the call to take questions from our research analysts. Operator, please proceed. Thank you. As a reminder, if you would like to ask a question, please press star 1 on your You'll hear the automatic message device in your hand is raised. We also ask that you limit yourself to one question and one follow-up. Please wait for your name and company to be announced before proceeding. One moment.
Speaker Change: With that we will open the call to take questions from our research analysts uplift operator. Please proceed.
Speaker Change: Thank you as a reminder, if you would like to ask a question. Please press star one on your telephone.
The automatic message device in your hand as rates. We also ask that you limit yourself to one question and one follow up.
Speaker Change: Please wait for your name and company to be announced before proceeding with your question.
Speaker Change: One moment.
Speaker Change: Thanks.
Luca Esat: And our first question will come from the line of Luca Esat of RBC, your line is open. Oh, great. Thanks so much for taking my question and congrats on all the progress.
Speaker Change: And our first question will come from the line of Luca <unk> of RBC. Your line is open.
Oh, great. Thanks, so much for taking my question and congrats on the progress.
Matt Kapusta: Maybe one for either Matt or Waleed on the new CBER head of the Ultragenics, which is a company with a long history in rare disease, is on record saying that Dr. Prasad may not be the right guy for the job. I guess two questions. One, would you agree with Ultragenics? And two, maybe bigger picture, how confident are you that all the productive conversations that you have had so far with CBER and Huntington will not be flipped upside down by the new leadership? I much appreciate it. Thank you. Yeah, thanks Luca.
Speaker Change: Maybe one for either Matt or will lead.
Speaker Change: The new Seeber head.
Speaker Change: The Altra genetics is a company with a long history in rare disease is on record, saying their doctor Prasad may not be derived guy for the job I guess two question. One would you agree with Walter genetics, and two maybe bigger picture. How confident are you that other productive conversations that you have had so far with seeber on Huntington.
Speaker Change: And we will not be flipped upside down side, the new leadership.
Speaker Change: Any company I much appreciate it thank you.
Matt Kapusta: First, let me just state that honestly I very much look forward to working with Dr. Prasad and and really do appreciate his public service. The reality is that no matter who's the director of CBER, we believe strongly in our in our data and we believe in the end that this is going to carry the day. I also want to point out something really important in terms of the reliance on surrogate biomarkers which has been something that I think has come up over the last couple of days. You have to keep in mind that this is not our approach with AMT 130.
Speaker Change: Yes. Thanks, Luca first let me just state that honestly I very much look forward to working with Dr. Prasad and really do appreciate his public service.
Speaker Change: The reality is that no matter, who is the director of CEVA.
Speaker Change: We believe strongly in our in our data and.
Speaker Change: And we believe in the end this is going to carry the day.
Speaker Change: I also want to point out something really important.
Speaker Change: In terms of the reliance on surrogate Biomarkers, which has been something that I think has come up over the last couple of days you have to keep in mind that this is not our approach with AMG 130.
Matt Kapusta: We're going to be seeking accelerated approval based on three plus years of clinical outcomes and utilizing a clinical measure as a primary endpoint. So honestly I believe that this is a key differentiator for AMT 130 and I mean I remain really encouraged about our path forward. Thank you.
Speaker Change: We're going to be seeking accelerated approval based on three plus years of clinical outcomes and utilizing a clinical measure.
Speaker Change: As a primary endpoint.
Speaker Change: Honestly I believe that this is a key differentiator for AMG 130, and I mean, I remain really encouraged about our path forward.
Speaker Change: Got it thanks, so much.
Operator: One moment for the next question.
Speaker Change: Thank you one moment for the next questions.
Debit Jadipohe: And the next question will be coming from the line of Debit Jadipohe of Guggenheim. Your line is open. Hey, good morning. Thanks for indulging me with two on two questions.
Speaker Change: And the next question will be coming from the line of debit <unk> of Guggenheim.
Speaker Change: Your line is open.
Speaker Change: Hey, good morning.
Speaker Change: For indulging me, but to answer your questions.
Waleed Abisab: The number one, how confident are you on the three year follow up data on the the USDRS and what would you consider as a good outcome? And I have a follow up.
Speaker Change: The number one.
Speaker Change: Confident are you on the three year follow up data on the.
Speaker Change: The USDA RFS and what would you consider as a good outcome.
Speaker Change: A follow up.
Waleed Abisab: Waleed, do you want to answer that one? Sure. As you know, we've been monitoring these patients for a long time. We have not been doing any additional analysis on the data, but it's very clear what we submitted to the FDA included data two years from these patients, but we also have some other patients on the low dose that have been treated even much longer than that. We have no indication at all that we lose any of the efficacy over time. As a result, we believe that the dose-dependent reduction in COHCRS that we observed at the two-year data will be maintained when we evaluate the three-year data.
Lee: Well Lee do you want to answer that one sure.
Speaker Change: As you know we've been monitoring.
Speaker Change: These patients for a long time.
Speaker Change: We have not been doing any additional analyses on the data, but it's very clear when we submitted to the FDA included data two years from these patients, but we also have some other patients on the low dose that have been treated even much longer than that.
Speaker Change: No indication at all that.
Speaker Change: We lose any of the efficacy overtime.
Speaker Change: As a result, we believe that the.
Speaker Change: Dose dependent reduction in <unk> shares that we observed at the two year data will be maintained when we evaluate the three year data so.
Waleed Abisab: So we feel very confident about our results going forward. Got it. Appreciate that.
Speaker Change: We feel very confident about our results going forward.
Speaker Change: Got it I appreciate that and the last question.
Waleed Abisab: And the last question, will the third quarter update include propensity map scoring based on the agreed upon SAP, or will that analysis be withheld for the BLS submission only? Thank you and congrats. Yeah, so the plan is to essentially agree with the FDA and finalize the statistical analysis plan before we lock the database and analyze the data. And at that point, we will be sharing the top-down results of these data. As you know, we have to be careful the degree to which we share from the data because the data would have been not yet reviewed by the FDA.
Speaker Change: Quarter update include propensity matched scoring based on the agreed upon.
Speaker Change: Our little that analysis.
Speaker Change: For the BLA submission and outlook, Thank you and congrats.
Speaker Change: Thank.
Speaker Change: Thank you.
Speaker Change: Yes so.
Speaker Change: <unk>.
Speaker Change: Essentially agree with the FDA on.
Speaker Change: And finalize the statistical analysis plan before.
Speaker Change: Locked the database and analyze the data.
Speaker Change: And at that point.
Speaker Change: We will be sharing the topline results of these data.
Speaker Change: As you know we have to be careful the degree to which we share from the data because the data would have been not yet reviewed by the FDA.
Waleed Abisab: but it will be the analysis that we would have agreed to with. Thank you.
Speaker Change: It will be the analysis that we would have agreed with the FDA.
Operator: One moment for the next question.
Speaker Change: Thank you one moment for the next question.
Paul Matias: And the next question will come from the line of Paul Matias of CFL. Your line is open. Hey, good morning. Thanks for taking my questions. I appreciate it.
Speaker Change: And the next question will come from the line of Paul <unk> of Stifel. Your line is open.
Paul <unk>: Hey, good morning, Thanks for taking my questions I appreciate it.
Matt Kapusta: As it relates to the SAP and the different sort of permutations there, I was wondering if you can kind of walk through your approach to this recent meeting. You know, what are the different things you suggested? That's one. Two, you know, Matt and Waleed, we talked at a conference earlier this year as it relates to the pros and cons of three-year versus two-year national history comparisons with survivorship bias and things like that. So maybe you can just kind of give us an update on where your head is at. And then, you know, three, just in terms of waiting for the meeting minutes, I understand that that is, you know, that's the best practice.
Paul <unk>: As it relates to the S&P and the different sort of permutations. There I was wondering if you can kind of walk through your approach to this recent meeting one of the different things you suggested that's one.
Matt Kavista: Matt and when we talk to it.
Matt Kavista: Conference earlier this year as it relates to the frozen cons of three year versus two year natural history comparisons with survivorship bias and things like that so maybe you can just kind of give us an update on where your head is out and then.
Matt Kavista: In terms of waiting for the meeting minutes I understand that that is that's the best practice.
Waleed Abisab: But are you waiting for the minutes to make sure you fully understand how the meeting went down and that you want to clarify things? Or is this more good housekeeping to not get in front of the FDA? Thanks so much. Yeah, I'll just answer the last one.
Matt Kavista: Waiting for the minutes to make sure you fully understand how the meeting went down and that you want to clarify things or is this more good housekeeping cannot get in front of me, yes. Thanks, so much.
Matt Kavista: Yes, I will just answer the last one and then in terms of the approach to the meeting or two versus three years I'll give that to walid.
Matt Kapusta: And then in terms of the approach to the meeting or two versus three years, I'll give that to Waleed. This is good housekeeping. This is just an appropriate thing to do. Best practice is to make sure you have the complete written responses. So it's really that simple.
Speaker Change: This is good housekeeping. This is just an appropriate thing to do best practice is to make sure you add the complete.
Matt Kavista: Written responses.
Matt Kavista: That's.
Waleed Abisab: And then on the approach to the meeting and two versus three years, Waleed, why don't you address those? So, I mean, look, this has been an ongoing study. We have people who have been also treated for up to four years. The overwhelming majority of patients would have three-year data. And, you know, the assessment is to look at the totality of the data. So we must look at the most relevant data sets. And that's the three-year because that's where the majority of patients are. Of course, we will be looking at the two years. We will be providing the four-year data, but the primary analysis will be based on the three-year data.
Speaker Change: It's really that simple and then on the approach to the meeting and two versus three years, while we why don't you address those.
Matt Kavista: No.
Speaker Change: Look this is this has been an ongoing study we have people who have been also treated for up to four years, the overwhelming majority of patients.
Speaker Change: Would have three year data in and.
Speaker Change: <unk>.
Speaker Change: The assessment is to look at the totality of the data. So we must look at the most relevant.
Speaker Change: Datasets investments three year, because thats, where the majority of patients are of course, we will be looking at the two years, we won't be providing the four year data, but the primary analysis will be based on the three year data and the reason for this is that you want to be able to continue to see D. D.
Waleed Abisab: And the reason for this is that you want to be able to continue to see the promising effects that we've seen at two years are continuing to be manifested at three years and beyond. And so we cannot just look at the earlier part and ignore where the majority of the data are. That's really simply what this is about. Thank you.
Speaker Change: The promising effects that we've seen at two years continuing to be manifested at three years and beyond.
Speaker Change: So we cannot just look at the earlier part and ignore where the majority of the data.
Speaker Change: Thats really simply.
Speaker Change: This is about.
Speaker Change: Thank you.
Patrick Tuchio: And our next question will be coming from the line of Patrick Tuchio of H.C. Wainwright. Your line is open. Thanks. Good morning.
Speaker Change: And our next question will be coming from the line of Patrick GTO.
Speaker Change: H C. Wainwright your line is open.
Matt Kapusta: Just a clarification on, now that you've held the type B meetings on both CMC and the SAP for AMT130, would you anticipate any additional regulatory interactions ahead of the BLA filing?
Speaker Change: Thanks, Good morning, just a clarification on now that you've held the type b meetings on both CMC in the S.
Speaker Change: For AMG 130 would you anticipate any additional regulatory interactions ahead of the BLA filing and then separately regarding the cohort three update I am wondering if you can elaborate on the three <unk>.
Waleed Abisab: And then, separately, regarding the Cohort 3 update, I'm wondering if you can elaborate on the three immunosuppression-related SAEs reported in Cohort 3, and what changes, if any, were made to the protocols or perioperative management. Yeah, I mean, I can, I'll address maybe the first question, you know, we'll, we'll talk about the path forward when we provide a regulatory update, but I think as we stated in the prepared remarks, we've had very constructive interactions with the FDA, we've previously gotten very clear feedback. We obviously look forward to providing the regulatory update and we feel very encouraged about our, about our path forward.
Speaker Change: Freshen related <unk> reported in cohort three and what changes if any were made to the protocols are perioperative management.
Speaker Change: Yes, I mean, I can I'll address maybe the first question.
Speaker Change: We'll talk about the path forward when we provide a regulatory update but I think as we stated in the prepared remarks.
Speaker Change: We've had very constructive interactions with the FDA, we previously gotten very clear feedback.
Speaker Change: We obviously look forward to providing a regulatory update and we feel very encouraged about our about our path forward.
Waleed Abisab: Waleed, do you want to address the, the cohort three? Yeah, sure. So maybe I can take a step back a little bit and kind of, you know, summarize why we did this. So, so starting with, with this, that we believe that the AMT130 is generally safe and well tolerated based on our experience in cohorts one and two. That context, there was no very operative immunosuppression. And what we saw that was some edema and CNS information from the infusion, particularly at the high dose, which manifested within several days of the procedure. But all of these serious adverse events result with either supportive therapy in two out of those four cases, and the other two result after a short course of steroid therapy.
Speaker Change: While we do you want to address the cohort three yes sure. So maybe I can take a step back a little bit and kind of.
Speaker Change: Summarize why we did it so so starting with this that we believe that the MTO and <unk> is generally safe and well tolerated based on our experience in cohorts one and two.
Speaker Change: That context, there was no pairing up with every municipality suppression and what we saw that was.
Speaker Change: Some <unk> in CNS information from the infusion, particularly at the high dose, which manifested within several days of the procedure, but all of these serious adverse events resolved either supportive therapy in two out of those four cases and the other two resolved after a short pause.
Speaker Change: Of steroid therapy.
Waleed Abisab: Now, in core three, we set out to evaluate the effects of a triple regimen, as I said, including steroids, rituximab, and serolimus. And we have not observed any drug-related adverse events attributable to EMT-130. We have observed three serious adverse events related to immunosuppression, mania, which is a well-known adverse event of prolonged corticosteroid therapy, and infection and fever, which probably are related to the triple regimen of immunosuppression. And all of these events fully resolved with standard repair interventions. So overall, we have, to some degree, answered our question. We were quite, we believe we have a good outcome here.
Speaker Change: No.
Speaker Change: Three we set out to evaluate the effects of a triple regimen as I said, including steroids Rituximab Rituxan avid sirolimus.
Speaker Change: Not observed any drug related adverse events attributable.
Speaker Change: <unk> hundred 30, we've observed recent events related to municipal pressure mania, which is a well known adverse event of a prolonged corticosteroid therapy.
Speaker Change: And infection and fever, which probably are related to the triple regimen if immunosuppression.
Speaker Change: And all of these events fully resolved with standard of care interventions.
Speaker Change: So overall.
Speaker Change: Do you have.
Speaker Change: Before degree answered our question we were quite.
Speaker Change: We believe we have a good outcome.
Waleed Abisab: We think the optimal way going forward is most likely going to be a short two-week course of corticosteroids that would be given perioperatively, with EMT-130. We will be double-checking these and discussing with our external advisors. But if you ask me today, my opinion, that would be my advice going forward. Terrific. Thanks so much.
Speaker Change: The optimal way going forward.
Speaker Change: It's most likely going to be a short two week course of corticosteroids that would be given.
Speaker Change: Operator.
Speaker Change: With the <unk>.
Speaker Change: We will be double checking these and discussing with our external advisers, but if you ask me today my opinion that would be the.
Speaker Change: And going forward.
Speaker Change: Bruce.
Speaker Change: Terrific. Thanks, so much sure.
Jenny: Thank you. And our next question will be coming from the line of Joseph Schwartz. Lovely Reap Partners. Your line is open. Hi, this is Jenny on for Joe. Thank you for taking our questions. Just another one on Prasad. We've heard some strong and somewhat contradictory opinions about patient advocacy advocacy groups from him. We've heard him say that the patient voice is probably the most important one at the table, but then we've also heard him express concerns about these groups that take funding from pharmaceutical companies, especially if they've been vocal about lowering regulatory standards for approval.
Speaker Change: Yes.
Speaker Change: Thank you and our next question will be coming from the line of Joseph Schwartz.
Speaker Change: Of Leerink partners. Your line is open.
Speaker Change: Hi, This is Jenny on for Joe. Thank you for taking our questions. Just another one on Prasad referred some strong and somewhat contradictory opinions about patient advocacy advocacy groups from Tim we've heard him say that the patient voice is probably the most important one at the table, but then we've also heard you express concerns.
Speaker Change: These groups that take funding from pharmaceutical companies, especially if they've been vocal about lowering regulatory standards for approval.
Waleed Abisab: In Huntington's, we're aware of the Huntington's Disease Society of America. What kind of relationship do you have with them? Do you know if they've interacted with the FDA? And also would just love to hear your thoughts about the HD community in general, since we know at least some of them were kind of hesitant about expediting development after Tom Anderson and Brown ran a plan. Thank you. Well, do you want to address that? Sure. Thanks, Matt.
Speaker Change: Huntington's, we're aware of the Huntington's disease Society of America, what kind of relationship do you have with them.
Speaker Change: If they have interacted with the FDA and also with just love to hear your thoughts about the HLA community in general since we know at least some of them were kind of.
Speaker Change: About expediting development after termination and Brian <unk>.
Speaker Change: Yes.
Speaker Change: Well Lee do you want to address that sure. Thanks, Matt.
Waleed Abisab: We've been having a very long history working with CHDI and a number of other patient advocacy organizations working in Huntington's Disease. Honestly, we would not be here today, not us, not other ones in the field, if there was no deep commitment from the patients, from the patient advocates, and also from all the experts to generate these very elaborate and very sophisticated natural history that really, really gave us the understanding of the course of the disease, regardless of the stage of Huntington's Disease, so that we can truly compare our trials adequately to that. So without them, honestly, we would not be here today.
We've been having very long history, working with CACI in a number of other patient advocacy organization working in Huntington disease.
Speaker Change: Honestly, we would not be here today, not us not other ones in the fields. If there was no deep commitment from the patients from the patient advocates and also from all be expert.
Speaker Change: Generally these.
Speaker Change: Very elaborate and very soon.
Speaker Change: Sophisticated.
Speaker Change: Natural history that really.
Speaker Change: <unk> really gave us the understanding of the course of the disease.
Speaker Change: Regardless of the stage.
Speaker Change: Huntington disease, so that we can truly compare.
Speaker Change: Our trials to that so so without them honestly would not be here today.
Waleed Abisab: These patient organizations, I find them to be extremely balanced. They work with all organizations, they work with regulators, they work with various pharma and biotech companies, and they make their data available to be used in a scientifically valid manner. I have never seen them favor one group versus the other. I think they're really well respected. And actually, last year, in the late October or early November timeframe, there was a very important meeting that was held by the FDA that invited the patient's organization and heard from a number of them. I think that speaks to the respect that they have for them, and they're really...
Speaker Change: This patient organization I find them to be extremely balanced they work with all.
Speaker Change: Organizations that we work with regulators they work with.
Speaker Change: With various pharma and biotech company and they make their data available to be used in a scientific key.
Speaker Change: Valid manner.
Speaker Change: I have never seen them.
Speaker Change: Favor one group versus the other I think theyre really with respected and actually last year.
Speaker Change: Late October early November timeframe, there was a very important meeting that was held by the FDA.
Speaker Change: Got invited the patient's organization and heard from a number of them I think that speaks to the respect that they have for them.
Speaker Change: And there really.
Waleed Abisab: equitable balance in the way they conduct themselves. So I really feel very fortunate that we have that relationship with them. I don't feel them to be biased at all. And honestly, without them, we would not be here today, not us and not other companies that you've seen recently, who have used the natural Thank you.
Speaker Change: Equitable balance and the way they conduct themselves.
Speaker Change: I really feel very fortunate that we have that relationship with them I don't feel them to be biased at all and honestly without them, we would not be here today, not us and not other companies that <unk> seen recently, who have used the natural history data.
Speaker Change: Okay.
Speaker Change: Thank you.
Operator: Thank you. One moment.
Sylvan Richter: Our next question will be coming from the line of Sylvan Richter of Goldman Sachs. Your line is open. Hi. This is Lydia. I'm from Selby. And thanks so much for taking our questions, and congrats on all the progress. Maybe just on the third cohort of ANT130, do you plan to include these data in the potential BLA filing, even though I think you've noted you don't believe they'll be necessary for the filing? Thanks so much. Yeah. Thank you.
Speaker Change: Thank you one moment.
Speaker Change: Next question will be coming from the line of southern Victor.
Victor: Of Goldman Sachs. Your line is open.
Cynthia Unfreeze: Hi, Cynthia Unfreeze, Dolby and thanks, so much for taking our questions and congrats on the progress maybe.
Speaker Change: Maybe just on the third cohort of A&P. When 30 do you plan to include these data and the potential BLA filing even though I think you've noted we don't believe will be necessary for the filings. Thanks. So much.
Matt Kapusta: Matt, I assume I'm responding to this. I'm going to go. Yeah. Thank you for this question. Yes, of course. At the time of submitting the BLA, all data from all the patients that we have for as long as we have them, as of the cutoff, will be included in the data. So as such, the 12 patients we will be part of the totality of the data for the FDA to review. Thanks so much. Thank you.
Yes, Thank you, Matt I assume I'm responding to this I'm going to go.
Speaker Change: Yes. Thank you for this question, yes of course at the time of submitting the BLA all data from all the patients on behalf for as long as we have them as of the cutoff should be included in the table.
Speaker Change: The 12 patients we will be part of the totality of the data.
Speaker Change: For the FDA to review.
Speaker Change: Thanks, so much.
Joseph Schwartz: Next question will be coming from the line of Joseph Throm of TD Cowan. Your line is open. Hi there. Thank you for taking my question and correcting the progress. You got it.
Speaker Change: Thank you next question will be coming from the line of Joseph <unk> TD Cowen Your line is open.
Speaker Change: Hi, there good morning, Thanks for taking my question and congrats.
Waleed Abisab: Just to clarify, what's the latest data set that the FDA has seen in terms of data cut versus what's been publicly released and have they seen any of that Cohort 3 data with the updated immunosuppression regimen? And then maybe just to be clear, the two-week immunosuppression with steroids that you're suggesting, is this identical to what the initial patients received in the study? Thank you.
Speaker Change: Robert.
Speaker Change: Just to.
Speaker Change: To clarify what's the latest dataset that the FDA has seen in terms of our data cut versus what's been publicly released.
Speaker Change: <unk> seen any of that cohort three data.
Speaker Change: With the updated immunosuppression regimen, and maybe just to be clear the two week immunosuppression with steroids.
Speaker Change: You are suggesting is identical to what.
Speaker Change: What the initial patients received in the study.
Waleed Abisab: So Matt, I'll take those two as well. So the data set that we shared with you back in July of 2024 last year included 21 patients who had crossed the two-year time point that was based on a data cut of March of 2024. When we met with the FDA in November, that is the most recent data that we shared with them, which was based on the June of last year cut, which included three more patients at the high dose. So in total, they saw data from 24 patients at two years, 12 at the high dose and 12 at the low dose.
Matt Kavista: So Matt I'll take that too.
Speaker Change: As well.
Matt Kavista: So the data set that we shared with you back in July of 2024 last year included 21 patients who had crossed the two year time point that was based on a data cut.
Matt Kavista: March of 2024, when he met with the FDA in November.
Matt Kavista: That is the most recent data that we shared with them, which was based on the June.
Matt Kavista: Last year cut which included three more patients at the high dose. So in total they saw data from 24 patients at two years to 12 at the high dose at 12% the low dose.
Waleed Abisab: There has been no additional data shared with them since then, since our meetings with them regarding SAP and the Natural History Protocol and CMC were about those specific topics without any review of data. So they did not see any additional data. They did not see any data from cohorts.
Matt Kavista: There has been no additional data shared with them. Since then since our meetings with them regarding FEP and the natural history protocol and CMC were about.
Matt Kavista: Specific topics without any review of data. So they did not see any additional data they did not see any data from cohort three.
Waleed Abisab: Regarding the two-week steroids, so for first course one and two, there were no systematic perioperative immunosuppression given, including steroids. So this will be the first time that we would be using this regimen. The reason why we're choosing it is because two weeks of steroids is very commonly used in neurosurgical procedures. Two weeks of steroid therapy is also the adverse event profile of that immunosuppression is well understood and considered to be very low. So that's why we're choosing it going forward. Great, thank you. Thank you.
Matt Kavista: Regarding the two week steroid so forth.
Matt Kavista: Cohorts, one and two there were no.
Matt Kavista: Systematic.
Matt Kavista: Operator immunosuppression given.
Matt Kavista: Including steroids.
Matt Kavista: So this will be the first time that we would be using this regimen. The reason why we're choosing it is because two weeks of steroids is very commonly used and new surgical procedures.
Matt Kavista: Two weeks.
Matt Kavista: Of steroid therapy is also.
Matt Kavista: Adverse event profile.
Matt Kavista: Immunosuppression is well understood and considered to be.
Matt Kavista: Very low so.
Matt Kavista: That's why we're choosing it going forward.
Matt Kavista: Great. Thank you.
Ellie Murrell: And our next question will be coming from the line of Ellie Murrell of UBS. Your line is open. Hey guys, thanks for taking the question. I'm just curious if you can update us on the latest in terms of the CMC work and specifically how long you think the CMC requirements will take in order to complete, in order to be ready for filing, since I think in the past you've said the timing of the filing would be more driven by when you'd be able to complete the CMC requirements. And then just a second question, just in terms of the selection of the Natural History Comparator, and the composition of that.
Speaker Change: Thank you and our next question will be coming from the line of Ellie Merle of UBS. Your line is open.
Speaker Change: Hey, guys. Thanks for taking my question just curious if you can update us on how latest in terms of the CMC work and specifically how long do you think the CMC requirements will take them.
Speaker Change: In order to complete in order to be ready for filing since I think in the past you've said the timing of the filing would be more driven by when you would be able to complete the CMC requirements.
Speaker Change: And then just a second question.
Speaker Change: In terms of the selection of the natural history comparator in.
Waleed Abisab: I guess after you met with the FDA and discussed the STATS plan, how confident are you that the natural history will look similar to the natural history that you've used before and when you presented the data? So just in terms of the composition of that. Oh, okay.
Speaker Change: The composition of that I guess after you met with the FDA and discuss the stock plan.
I don't argue that the natural history.
Speaker Change: Well look similar to the natural history that you've used before and when you presented the data suggest in terms of the composition of that thanks.
Waleed Abisab: I'm going to be answering both questions. So, this is Waleed again. So, the CMC, you know, there are a series of, you know, well-agreed and thought-through plans for this. We will be sharing with you, you know, the overall timeline for BLA submission when we provide the regulatory update at the end of the year. All of this is not new to us. Excuse me. As we've said before, we're leveraging the significant experience we have with Hamgenics. We're manufacturing MT130 in the same place by generally the same people. And a lot of this has been leveraged in discussing with the FDA.
Speaker Change: Oh.
Speaker Change: Okay.
Speaker Change: Be answering both questions.
Speaker Change: So this is wahid again.
Speaker Change: So on the CMC.
Speaker Change: A series of <unk>.
Speaker Change: Well agreed and thought through plan for this we will be sharing with you.
Speaker Change: The overall timeline for BLA submission, let me provide a regulatory update at the end of the year. All of this is not new to us excuse me.
Speaker Change: We've said before we're leveraging the.
Speaker Change: The significant experience, we have with Amgen, we're manufacturing empty 130 in the same place.
Speaker Change: But generally the same people and a lot of this is Ben.
Speaker Change: Leverage.
Speaker Change:
Waleed Abisab: So, we'll provide more granular information on this when we provide the regulatory update by the end of this, in the second.
Speaker Change: In discussion discussing with the FDA. So we will provide more granular granular.
Speaker Change: Information on this when we provide deregulatory update by the end of this.
Waleed Abisab: On the selection of natural history, I think we've discussed that we're evaluating a number of natural histories. We've had that discussion with the FDA. We will be updating you again as part of that regulatory update as to where we landed on this. I do not anticipate for this to have any significant difference on what we have been reporting so far, or at least last time we reported it to you back in July of last year. So I think we're still going in the same direction. I don't expect any difference in the outcome. Thank you.
Speaker Change: Second quarter.
Speaker Change: The selection of natural history I think.
Speaker Change: We've discussed that we're evaluating a number of measures we've had that discussion with the FDA.
Speaker Change: We have.
Speaker Change: We will be updating you again as part of that regulatory update as to where we landed on this I do not anticipate for this to have any significant difference on what we have been reporting so far.
Speaker Change: Or at least last time, we reported to you back in July of last year. So I think we're still going in the same direction I don't expect any difference or delta. Thank.
Speaker Change: Thank you.
Speaker Change: Thanks.
WZ Shah Pahe: Our next question will be coming from the line of white ear of the school. Your line is open. Hello. Yeah, hi. Hi. Yeah, sorry. I didn't hear whose name was announced. Sorry.
Speaker Change: Thank you. Our next question will be coming from the line of y ear.
Speaker Change: Mr. <unk> your line is open.
Speaker Change: Hello.
Speaker Change: Yes, hi.
Speaker Change: Hi, Yes, sorry, I didn't hear.
Speaker Change: Please name as announced sorry, yes. So.
Matt Kapusta: Yeah, so a couple of questions from us. Between the CMC meeting and your recent meeting with the FDA, were there any changes in the key personnel? And secondly, you know, given the new CBER director, are you, I know it's early, are you thinking that, have your thoughts on, you know, developing your current pipeline change in any way? And just wanted to make sure. So it's the three year data that will, that will be used for AMT 130 filing. Is that right?
Speaker Change: Yes.
Speaker Change: Questions from us.
Speaker Change: Between the CMC meeting and your recent meeting.
Speaker Change: With the FDA, where there any changes in the key personnel.
Speaker Change: And secondly, given the.
Speaker Change: New Seabury director.
Speaker Change: Are you I know, it's early are you thinking.
Speaker Change: Have your thoughts on developing your current pipeline change in any way and just wanted to make sure that.
Speaker Change: <unk> data that will.
Speaker Change: That will be used for <unk>.
Matt Kapusta: Thank you. Okay, very good.
Speaker Change: 130 filings that right. Thanks.
Matt Kapusta: Hey, Matt, are you back on? I think we're back on. Yeah. So, yeah, so I think you've asked a few questions here. So, the first one was around the CMC and the personnel that we've interacted with at the FDA. We've had no material impact on anybody that is part of the review team that we obviously can see. You know, there's some public record of people that have resigned. There's been at least 30 people that have been involved in our various interactions, and there's been no material changes that we're aware of. Just in terms of your second question about the other pipeline programs, we continue to be very encouraged about the programs that we have.
Speaker Change: Okay very good.
Speaker Change: Matt are you back on.
Speaker Change: We are back on yes, so yes, so I think you've asked a few questions here.
Speaker Change: So the first one was around the CMC and the personnel that we've interacted with the FDA.
Speaker Change: Had no material impact on.
Speaker Change: Anybody that is part of the review team that we've obviously can see.
Speaker Change: There is some public record of people that have resigned theres been.
Speaker Change: At least 30 people that have been involved in our various interactions and there has been no material changes that we are aware of.
Speaker Change: Just in terms of your second question about the <unk>.
Speaker Change: Other pipeline programs, we continue to be very encouraged about the programs that we have we're going to be generating data across those programs over the course of the next.
Matt Kapusta: We're going to be generating data across those programs over the course of the next 6 to 12 months, and we're very excited to do that. And obviously, we're hoping that we're able to determine a path forward and to establish a clinical proof of concept. And then just in terms of the two to three-year data, I think as Waleed mentioned, we'll obviously provide a more detailed update when we provide our regulatory update. I think what Waleed is pointing out is that In the third quarter, we're going to have 27 patients at two years and 24 patients at three years.
Speaker Change: Six to 12 months and we're very excited to do that.
Speaker Change: And obviously, we're hoping that we're able to determine a path forward and to establish a clinical proof of concept.
Speaker Change: And then just in terms of the two.
Speaker Change: Two to three year data I think as Waleed mentioned I will provide will obviously provide a more detailed.
Speaker Change: When we provide our regulatory update I think what <unk> is pointing out is that.
Speaker Change: In the third quarter, and we're going to have 2007 patients at two years in 2000 and for patients at three years.
Matt Kapusta: So, no matter what we look at, it's going to be very important that the, you know, the totality of the data continue to reflect the trends that we continue to see both at 2 years and 3 years.
Speaker Change: No matter, what we look at it is going to be very important.
Speaker Change: The totality of the data continued to reflect.
Speaker Change: The trends that we continue to see both at two years and three years.
Matt Kapusta: Thank you.
Speaker Change: Thank you.
Speaker Change: Okay.
Suzanne Van Vortensen: And our next question will be coming from the line of Susan Van Vortensen of VLK. Thank you. Your line is open. Hi, team. This is Suzanne from Kempen. Thanks for taking my questions.
Speaker Change: Thank you and our next question will be coming from the line of Susan then Watson.
Speaker Change: Okay. Thank you your line is open.
Speaker Change: Hi, Tim This is Alan Mckim from thanks for taking my questions.
Matt Kapusta: I know the focus is very much on the data and the BLA, but can you elaborate a bit on your preliminary thinking on the commercial plans and the rollout, the focus on centers, what you consider key target groups, et cetera?
Speaker Change: I noticed that this is very much on the data on the BLA, but can you elaborate a bit on your preliminary.
Speaker Change: On the commercial plans and the Rollouts and the focus on centers.
Speaker Change: Mike you consider key target groups et cetera, and secondly can you share your current thinking on a potential filing in Europe and or thoughts on a partnership ex U S. Thank you.
Matt Kapusta: And secondly, can you share your current thinking on a potential filing in Europe and or thoughts on a partnership ex-US? Thank you.
Matt Kapusta: Okay, so we will talk in more detail going forward about the, about the commercial strategy. What I can tell you is we're, we're very excited about about the potential here in Huntington's. Obviously, there's no disease modifying treatments that are available for these patients and we believe that being able to demonstrate meaningful slowing of disease progression would be a transformational leap forward for this for this community. Uh, just in terms of prevalence, it's probably 1 of the, the largest genetically defined diseases and we honestly believe that we have a very good shot at being not only 1st to market, but best in class.
Speaker Change: Okay.
Speaker Change: So we will talk in more detail going forward about the about the commercial strategy. What I can tell you is we're very excited about about the potential here in Huntington's, obviously theres no disease modifying treatments that are available for these patients.
Speaker Change: And we believe that being able to demonstrate meaningful slowing of disease progression would be a transformational leap forward for this for this community.
Speaker Change: Just in terms of prevalence, it's probably one of the largest genetically defined diseases.
Speaker Change: And we honestly believe that we have a very good shot at being not only first to market, but best in class. So we're really excited about that.
Matt Kapusta: So, we're, we're really excited about that.
Matt Kapusta: Um, we're right now focused on regulatory interactions with the United States and now that we've established an accelerated path forward, we'll be engaging with regulators in Europe and we'll talk more about. We are preparing for commercialization. As I said, we're very excited about this opportunity. No doubt, strategics are excited about this opportunity as well. This is a needle-moving area for both large biotechs and large pharma companies, so there's certainly a lot of strategic interest in Huntington's, and in the end, we're always going to do what's in the best interest of our shareholders. Thank you.
Speaker Change: We're right now focused on regulatory interactions with the United States and now that we've established an accelerated path forward will be engaging with regulators in Europe, and we will talk more about that.
Speaker Change: We are preparing for commercialization.
Speaker Change: As I said, we're very excited about this opportunity no doubt strategics are excited about this opportunity as well. This is a needle to needle moving area for both large biotech and large pharma companies.
Speaker Change: So there's certainly a lot of strategic interest in Huntington's.
Speaker Change: And in the end, we're always going to do what's in the best interest of our shareholders.
Sammy Corwin: One moment for the next question. And the next question is coming from the line of Sammy Corwin of William Blair. Your line is open. Hey there, thanks for taking my questions and congrats on the progress. I guess as you kind of think about the longer term strategy for AMT 130, how are you thinking about potentially evaluating it in patients with earlier stage or later stage disease? And then I was also curious if you could give us provide any color, I guess, on the number of physicians in the US who would be capable of performing the administration necessary for AMT 130.
Speaker Change: Thank you one moment for the next question.
Speaker Change: And the next question is coming from the line of semi.
Semi Cohen: Cohen of William Blair. Your line is open.
Semi Cohen: Hey, there thanks for taking my questions and congrats on the progress.
Semi Cohen: So as you kind of think about the longer term strategy for AMG 130, how are you thinking about potentially evaluating.
Semi Cohen: Patients with earlier stage or later stage disease, and then I was also curious if you could give us provide any color I guess on the number of physicians in the U S will be capable of performing D administration necessary for anti <unk>. Thank you.
Waleed Abisab: Thank you.
Waleed Abisab: Wally, do you want to answer that? So right now, as you know, in this first trial, we evaluated patients who are early manifest because we believe that this is the best chance to maximize the functions, to preserve as much as functions as possible, but also at the same time, that we need to have enough time to evaluate them to see whether there is an impact on the course of disease. If you take people much earlier, at a pre-symptomatic stage, then it's gonna take you much, much longer to be able to show evidence of stopping or slowing disease.
Speaker Change: Well Lee do you want to answer that.
Semi Cohen: So.
Semi Cohen: So right now.
Semi Cohen: As you know in this first trial.
Semi Cohen: We evaluated patients who are early manifest because we believe that this is the best chance to maximize.
Semi Cohen: B.
Semi Cohen: The functions to preserve as much of functions as possible, but also at the same time that we would need to have enough time to evaluate them to see.
Semi Cohen: Whether there is an impact on the course of the disease, if you take people.
Semi Cohen: Much earlier in a pre symptomatic stage, then it's going to take you much much longer to be able to show evidence of stopping or slowing disease progression.
Waleed Abisab: As we now move forward with this, of course, there will be great interest to try and figure out whether you can intervene earlier or later. And those are really early stages right now that we need to be discussing those with the regulators to see what would be the best path going forward. There's no good reason to believe that you should be excluding patients depending on their stage of the disease. At the same time, we need to be able to generate data to convince us that actually those people are set to benefit from it.
Semi Cohen: As we now move forward with this of course, there will be great interest to try and figure out whether you can intervene earlier or later and those are really early stages right now that we need to be discussing those with the with the regulators to see what would be the best path going forward.
Semi Cohen: No. Good reason to believe that you should be excluding patients depending on the stage of the disease at the same time, we need to be able to generate data to convince us that actually those people are set to benefit from it so.
Waleed Abisab: So I know I'm not giving you a real answer, but this is really early days in our thinking about it, and we need to engage with the regulators to have a much better idea about how we would be approaching for both patients. Those who are earlier and pre-symptomatic and what would be the end point in that case and how long we should treat them, and those who are maybe further advanced. There, I would imagine the limitation would be about being able to reach the deep structures of the brain where we need to inject our therapy in a safe manner.
Semi Cohen: No I'm not giving you a real answer but this is really early early days in our thinking about it and we need to engage with the regulators to have a much better idea about how we'd be approaching for both patients those who are earlier in pre symptomatic and what would be the endpoint in that case and how long we should treat them and those who are maybe further advance.
Semi Cohen: There I would imagine the.
Semi Cohen: The limitation would be about being able to reach the deep structures of the brain, where we need to inject our therapy in a safe manner in other words, if there is not enough and.
Waleed Abisab: In other words, if there's not enough, not significant atrophy that happens such that we will be a bit unsafe to be able to get there. But that usually is a discussion that the neurosurgeon would be able to have.
Semi Cohen: Not significant atrophy that happened such that we will be a bit unsafe to be able to get there, but that's usually is a discussion that the neurosurgery.
Waleed Abisab: So I know it's not a very clean question to answer to your question, but this is our thinking as well. Was there something else? I'm sorry, I might have missed another piece of your question. Yes, yes. How many physicians in the U.S. could be capable of performing the de-administration procedure? Right, so when we looked at this, there are upward of 50 sites who can who can do this. And really, this is not a very complicated procedure. I mean, this is something that it's been used right now for administering, you know, you know, chemotherapy using sort of essentially a guided administration intraparenchymally in the brain using a frame.
Semi Cohen: So I know, it's not a very.
Semi Cohen: Clean question to answer to your question, but this is our.
Semi Cohen: Thinking as of today was there something else I am sorry, I might have missed another piece of your question yes.
Speaker Change: Yes, yes, how many physicians in the U S could be capable of performing the administration procedure.
Speaker Change: Alright, so when we looked at this there are upfront.
Speaker Change: Ft sites, who can who can do this and really this is not a very comp.
Speaker Change: Complicated procedure.
Speaker Change: This is.
Speaker Change: Something that it's been use right now for administering.
Chemotherapy using sort of essentially a.
Speaker Change: Guided administration and shopper activity in the brain.
Waleed Abisab: This is used commonly by neurosurgeon, they should be it's not very complicated. So essentially, in our case, we need to have a center that essentially can do that. But also, we'll have access to an MRI. Because as you know, we do this to maximize the chances if we don't cut corners, this is a one and done. So we need to make sure that whoever is getting that therapy is getting the most out of it. So we're not going navigating blind, we'll do it under MRI guidance, we will look exactly where we're injecting. And the neurosurgeon can adjust the course and make sure that the structures are appropriately filled.
Speaker Change: Using a frame this is used commonly by neurosurgeon they should be it's not very complicated so essentially in our case.
Speaker Change: We need to have a centers.
Speaker Change: Essentially can do that but also will have access to MRI because as you know we do this to maximize the chances that we don't cut corners. This as a one and done so we need to make sure that whoever is getting that therapy. There is getting the most out of it. So we're not going navigating blind, we'll do it under MRI guidance.
Speaker Change: We looked exactly where we are injecting and.
Speaker Change: The neurosurgeon can adjust the course and make sure that the structures.
Waleed Abisab: So those are really the two conditions. But when we look, there's plenty of centers in the US, I said more than 50 that can do it. And yeah, I think that's That's my answer to your question. Sounds good, thanks. Thank you.
Speaker Change: Appropriately filled so those are the really the two conditions, but when we look there's plenty of centers in the U S. I said more than 50 that can do it.
Speaker Change: Yes, I think thats.
Speaker Change: That's my answer to your question.
Speaker Change: Sounds good thanks.
Yonhanzu: And the next question will be coming from the line of Yonhanzu of Wells Fargo. Your line is open. Great, thanks for taking our questions and for the very helpful call. So maybe a couple of follow-up questions on topics already discussed on the length of follow-up for the data in the filing package and also the control arm. On the length of follow-up, I think Based on what I heard, it's maybe a discussion between two or three years, or that might be the range. I just wanted to ask, could you confirm this is not going to be an even longer follow-up than a three-year follow-up for the filing?
Speaker Change: Thank you and our next question will be coming from.
Johan: Non of Johan <unk> of Wells Fargo. Your line is open.
Speaker Change: Great. Thanks for taking our questions and for the very helpful call.
Speaker Change: So maybe a couple of follow up questions on topics already discussed.
Speaker Change: The last follow up for the data in the filing package and also the control arm.
Speaker Change: And the last a follow up I think.
Speaker Change: Based on what I heard.
Speaker Change: Maybe a discussion between two or three year or.
Speaker Change: That might be.
Speaker Change: The range I just wanted to.
Speaker Change: Ask.
Speaker Change: It.
Speaker Change: Could you confirm this is not going to be.
Speaker Change: Even longer follow up than a three.
Speaker Change: Three year follow up for.
Waleed Abisab: And on the external control, it sounds like there shouldn't be much surprise compared to what you have been thinking or what you have reported. But I just wanted to confirm that in terms of the natural history study that you are looking forward to using, is the same track HD in that study and not a different study like enrolled HD? Or perhaps it doesn't matter. Please help with those questions. Thank you.
Speaker Change: For the filing and on the external control.
Speaker Change: And it sounds like there shouldn't be much surprise.
Speaker Change: Paired with what you have been thinking or what you have reported.
Speaker Change: Just wanted to confirm that in terms of the natural history study that.
Speaker Change: You are looking forward to using.
Speaker Change: Is the same.
Speaker Change: Okay.
Track HD.
Speaker Change: That study or not.
Speaker Change: <unk> study like enroll HD or perhaps it doesn't matter.
Speaker Change: <unk> helps.
Speaker Change: With those questions. Thanks.
Waleed Abisab: Sure. On the first question, we have already discussed this last November with the FDA and agreed that the data cutoff of end of June of this year, 2025, will be the data cutoff and we will use the totality of the data to support a BLA sub-measure. At that time, we will have, as Matt said, 27 patients with two years of follow-up, 24 patients with three years of follow-up, eight patients with four years, and actually one patient with five years. But the totality of the data will be received. The primary analysis will be done on the three-year data because that's where the majority of the patients that is going to generate meaningful understanding of the rate of progression of the disease.
Sure.
Speaker Change: On the first question.
Speaker Change: <unk> already discussed this last in November with the FDA and agreed that the data cutoff.
Speaker Change: End of June of this year 2025 will be.
Speaker Change: The data cutoff, we will use the totality of the data.
Speaker Change: To support a BLA submission.
Speaker Change: At that time, we will have.
Speaker Change: That said 2007 patients with two years of follow up 24 patients with three year follow up eight patients with for years and actually one patient with five years.
Speaker Change: The totality of the data will be received the primary analysis will be done on the three year data because that's what the majority of the.
Speaker Change: The patients.
Speaker Change: That is going to generate meaningful understanding of the.
Speaker Change: The rate of progression of disease.
Waleed Abisab: Regarding external control, also we communicated that last time when we met with the FDA, we were asked to evaluate systematically all the available natural history protocols, and EnrollHD is one of the largest ones. Track and PredictHD we've used previously because they control the, you know, stridal volume. But now we've gone through this and systematically, you know, evaluated this. We've had discussions with the FDA and agreed on the one that we will be using as a primary. This will be part of our feedback to you when we provide the regulatory feedback. Again, I think you also answered your question in the question itself.
Speaker Change: Disease.
Speaker Change: Regarding external control.
Speaker Change: Also we communicated that last time, when we met with the FDA. We were asked to evaluate systematically all the available.
Speaker Change: Natural history protocols and enrolled HD is one of the largest ones.
Speaker Change: And predict HD, we've used previously because they control the spread of volume.
Speaker Change: But now we've gone through this.
Speaker Change: Systematically evaluated this we've had discussions with the FDA and agreed on the one that we were using as a primary this would be part of our feedback to you when you provide the regulatory feedback.
Speaker Change: I think you also answered your question.
Waleed Abisab: We said it doesn't matter. Honestly, I don't think it does matter that much between those, the results Enroll, Track, and Predict in terms of the course of the disease provided that you do appropriate matching to your patient population generates virtually the same rate of decline that you see. Incidentally, if I may, you have seen probably recently data from PTC where they've used external control compared to a natural history. In that case, it was EnrollHD. They looked at the rate of decline in COACRS after two years, and it came out at 1.1. If you recall, that's very close to the data that we shared with you last year in July where we used Track and Predict, and we came out to virtually the same number.
Speaker Change: And the question itself, we said it doesn't matter honesty I don't think it does it does matter that much between dose.
Speaker Change: <unk> between enroll track and predict in terms of the course of the disease provided that you do appropriate matching to your patient population generates virtually the same rate of decline that you see.
Speaker Change: Incidentally, if I may you have seen probably recently.
Speaker Change: Data from PTC, where their views.
Speaker Change: External control used compared it to a natural history in that case. It was enrolled <unk>. They looked at the rate of decline in <unk>. After two years and it came out at 1.1, if you recall, that's very close to the data that we shared with you.
Speaker Change: Last year in July, where we used truck and predict and we came up to virtually the same number. So so that should give you enough confidence that in general if you do the appropriate matching of your patient population youre going to end up.
Matt Kapusta: So that should give you and us confidence that in general, if you do the appropriate matching of your patient population, you're going to end up in the same place after two years or in our case, three years. Yes, indeed, that that is what we saw.
Speaker Change: In the same place after two years or not just three years.
Speaker Change: Yes, indeed that is what we saw so thank you for.
Matt Kapusta: So thank you for clarifying all of that.
Speaker Change: Clarifying all of that maybe.
Matt Kapusta: Maybe as a quick follow up, would the team care to share your view on PTC's data and their drug as a potential potential competitive consideration? Thanks.
Speaker Change: Maybe as a quick follow up with the team of care to share.
Speaker Change: Your view on Ptc's.
Speaker Change: Ptc's.
Speaker Change: Data.
Speaker Change: <unk>.
Speaker Change: Their drug as a Po.
Speaker Change: Potential.
Speaker Change: Our competitive consideration.
Waleed Abisab: Well, perhaps Matt can speak about the competitive piece of it from a scientific point of view. I do not think it's appropriate for us to comment on on a competitor's data, especially when. not we don't see the full data set. It's not in a, you know, scientific presentation or a period publication. It's very difficult to do that and I don't think it's appropriate for us to comment.
Speaker Change: Well, perhaps Matt can speak about the competitive piece of it from a scientific point of view I do not think it's appropriate for us to comment on our competitor's data, especially when.
Speaker Change: It's not we don't see the full data set that's not enough.
Speaker Change: Fantastic presentation or a peer reviewed publication.
Matt Kavista: It's very difficult to do that and I don't think its appropriate for us to comment on that and turn it over to you mats.
Matt Kapusta: Turn it over to you, Matt. Yeah, I mean, well, I mean, I would just focus on our data. I think our data that we presented last year showed an approximately 80% slowing of disease progression based on the composite UHDRS. We're very comfortable with our bioavailability of our drug, because we can see real time the filling of the structures when it's administered. And we're also very comfortable with our target, right, which not only suppresses the full-length mutant Huntington protein, but also the short, highly toxic exon 1 fragment, which certainly provides differentiation. So, I mean, we're focused on our data, really encouraged by it, and also believe that our administration and our target makes us potentially best in class.
Speaker Change: Yes.
Matt Kavista: I would just focus on our data.
Speaker Change: I think our data.
Speaker Change: That we presented last year showed an.
Speaker Change: And approximately 80% slowing of disease progression based on the composite UHD Rs.
Speaker Change: We're very comfortable with our bioavailability of our drug.
Speaker Change: Because we can see real time, the filling of the structures when it's administered.
Speaker Change: And we're also very comfortable with our.
Speaker Change: Our target rate, which not only suppresses the full length mutant Huntington protein, but also the.
Speaker Change: Short highly toxic exon, one fragment, which would certainly provides differentiation.
Speaker Change: So I mean, we're focused on our data.
Speaker Change: Really encouraged by it and and also believe that our administration in our target.
Speaker Change: Makes us potentially best in class.
Matt Kapusta: Got it. Thanks and congrats on the progress. Thank you.
Speaker Change: Got it thanks, Thanks and congrats.
Speaker Change: On the progress.
Speaker Change: Thank you.
Operator: If you would like to ask a question, please press star one one on your.
Speaker Change: Thank you if you would like to ask a question. Please press star one on your telephone one moment for the next question.
WZ Shah Pahe: One moment for the next question, and the next question will be coming from the line of WZ Shah Pahe of Guggenheim and it's a follow-up.
Speaker Change: And the next question will be coming from the line.
Speaker Change: Devin shipped.
Speaker Change: Sure.
Speaker Change: Hey, Guggenheim and as a follow up Adam Your line is.
WZ Shah Pahe: There you go, your line is open.
Waleed Abisab: Hey, thanks for letting me back in again. Just another follow up for me. Has there been a discussion on what a future confirmatory study could look like or would you not need one since CUSDRS is not a surrogate endpoint? Well, we. So we have discussed with the FDA back in November last year, and we actually at the time, we had a proposal in place. The FDA said we're not prepared to discuss what would the confirmatory study would look like until we have a chance to review the data that you submit to us as part of the BLA submission that we agreed to with them.
Adam: Hey, Thanks for letting me back on again.
Speaker Change: Just another follow up from me has there been a discussion on what our future content makes this study could look like or would you not need one <unk> is not a sort of at that point.
Adam: Well.
Adam: So.
We have discussed with the FDA back in November.
Adam: Last year and.
Adam: And we actually at the time, we had a proposal in place. The FDA said, we're not prepared to discuss what would be.
Adam: Confirm confirmatory study would look like until we have a chance to review the data that you submit to us as part of the BLA submission that we agreed to.
Waleed Abisab: So at this point, the agency is not ready to discuss this. Our interpretation is that it can be as optimistic as maybe more longer-term data from this study, maybe looking at total functional capacity data from our study at a longer time point, maybe adding another cohort to our study or a completely new independent study, but it would not be placebo-controlled, in our opinion. But again, to some degree, this is... speculation on our part because the agency did not want to discuss that. What we know for sure is that this will not be or it's not expected based on what the agency has told us so far to have to be completed and done and about to start or halfway conducted before we can file for BLA for accelerated approval.
Adam: With them.
Adam: So at this point.
Speaker Change: C is not ready to discuss this our interpretation is that.
Adam: It can be.
Adam: As optimistic as maybe more longer term data from this study may be looking at total functional capacity data from our study at a longer time point, maybe adding another cohort study or a completely new.
Adam: Independent study, but it would not be placebo control in our opinion, but again to.
Adam: To some degree this is.
Adam: Speculation on our part because the agency did not want to discuss what we know for sure is that this will not be it's not expected based on what the agency has told us so far.
Adam: You have to be completed and done and about to start or halfway conducted before we can file for a BLA for accelerated approval. So we don't think its going to.
Waleed Abisab: So we don't think it's going to slow us down at all. And that was clear from our interaction with the FDA.
Adam: Louis down.
Adam: Paul.
That was clear from our interaction with the FDA.
Matt Kapusta: Thank you. At this time I'm not showing any more questions in the queue and I'll turn the call back to Matt Kapusta for closing remarks. Please go ahead. Thank you very much, operator. Really appreciate everybody dialing into the call. Look forward to providing additional updates. very shortly. Thank you so much. This does conclude today's conference call. Thank you all for joining. You may now.
Adam: Thank you.
Adam: At this time I'm not showing any more questions in the queue and I will turn the call back to Matt.
Adam: There.
Matt Kavista: For closing remarks. Please go ahead.
Speaker Change: Thank you very much operator, really appreciate everybody dialing into the call and look forward to providing additional updates.
Very shortly thank you so much.
Speaker Change: This does concludes today's conference call. Thank you all for joining you may now disconnect.
Speaker Change: Okay.
Speaker Change: [music].
Operator: Thanks for watching!
Speaker Change: Okay.
Speaker Change: Yeah.
Speaker Change: [music].
Speaker Change: Okay.
Speaker Change: [music].