Q2 2026 Regenxbio Inc Earnings Call

August 6, 2026

Operator 2: Three, two. Welcome everyone to the Q2 2026 REGENXBIO earnings conference call. My name is Elaine and I will be your conference operator today. All lines have been placed on mute to prevent any background noise, and after the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star then number one on your telephone key. To withdraw your question, press star one again. At this time, I'd like to turn the conference over to Patrick J. Christmas II, Chief Legal Officer of REGENXBIO. Please go ahead.

Operator: Welcome everyone to the Q2 2026 REGENXBIO earnings conference call. My name is Elaine and I will be your conference operator today. All lines have been placed on mute to prevent any background noise, and after the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star then number one on your telephone key. To withdraw your question, press star one again. At this time, I'd like to turn the conference over to Patrick J. Christmas II, Chief Legal Officer of REGENXBIO. Please go ahead.

Speaker #1: All lines have been placed on mute to prevent any background noise, and after the speakers' remarks, there will be a question-and-answer session. If you would like to ask a question during this time, simply press start, then 1.

Speaker #1: go ahead.

Patrick J. Christmas II: Good morning and thank you for joining us today. Earlier this morning, REGENXBIO released financial and operating results for the Q2 ended 30 June 2026. The press release is available on our website at www.regenxbio.com. Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend, and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties.

Speaker #2: Earlier this morning, forward-looking statements regarding our financial outlook in addition to regulatory and forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.

Speaker #2: REGENXBIO released financial and operating results for the second quarter ended June 30, 2026. The press release is available on our website at www.regenxbio.com today.

Speaker #2: Product development plans. These can be identified by words such as "expect," "plan," "will," "may," "anticipate," "believe," "should," "intend," and other words of similar meaning.

Speaker #2: Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties. These risks are described in the Risk Factors and the Management’s Discussion and Analysis sections of REGENXBIO’s Annual Report on Form 10-K for the full year ended December 31, 2025, and comparable Risk Factors sections of REGENXBIO’s quarterly reports on Form 10-Q, which will be on file with the Securities and Exchange Commission and available on the SEC’s website.

Patrick J. Christmas II: These risks are described in the Risk Factors and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended 31 December 2025, and comparable Risk Factor sections of REGENXBIO's quarterly reports on Form 10-Q, which will be on file with the Securities and Exchange Commission available on the SEC's website. Any information we provide on this conference call is provided only as of the date of this call, 6 August 2026, and we undertake no obligations to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company.

Patrick J. Christmas II: These risks are described in the Risk Factors and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended 31 December 2025, and comparable Risk Factor sections of REGENXBIO's quarterly reports on Form 10-Q. Which will be on file with the Securities and Exchange Commission available on the SEC's website. Any information we provide on this conference call is provided only as of the date of this call, 6 August 2026, and we undertake no obligations to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise.

Speaker #2: Any information we provide on this conference call is provided only as the data of this call, August 6, 2026, and we undertake no obligations to update any forward-looking statements we may make on this call on account of new information future events or otherwise.

Speaker #2: Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company.

Patrick J. Christmas II: Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of REGENXBIO.

Speaker #2: Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of REGENXBIO.

Patrick J. Christmas II: Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of REGENXBIO.

Speaker #3: Thank you, Patrick, and good morning, everyone. Thank you for joining us today. The second quarter was another period of positive momentum for REGENXBIO, achieving key milestones across our late-stage pipeline of gene therapies.

Curran M. Simpson: Thank you, Patrick, and good morning, everyone. Thank you for joining us today. Q2 was another period of positive momentum for REGENXBIO, achieving key milestones across our late-stage pipeline of gene therapies. During the quarter, we announced that we completed enrollment in the confirmatory study for RGX-202, reached alignment with FDA on the path to resubmit the BLA for RGX-121, and dosed the first patient in the NAVIGATE trial of sura-vec in diabetic retinopathy, achieving a $100 million milestone payment from AbbVie. We have substantially strengthened our financial position through receipt of over $200 million total, inclusive of the AbbVie milestone payment and new capital, ending the quarter pro forma with more than $310 million.

Curran M. Simpson: Thank you, Patrick, and good morning, everyone. Thank you for joining us today. Q2 was another period of positive momentum for REGENXBIO, achieving key milestones across our late-stage pipeline of gene therapies. During the quarter, we announced that we completed enrollment in the confirmatory study for RGX-202, reached alignment with FDA on the path to resubmit the BLA for RGX-121, and dosed the first patient in the NAAVIGATE trial of sura-vec in diabetic retinopathy, achieving a $100 million milestone payment from AbbVie. We have substantially strengthened our financial position through receipt of over $200 million total, inclusive of the AbbVie milestone payment and new capital, ending the quarter pro forma with more than $310 million.

Speaker #3: During the quarter, we announced that we completed enrollment in the confirmatory study for our GX202, reached alignment with FDA on the path to resubmit the BLA for our GX121, and dosed the first patient in the Navigate trial of CureVac in diabetic retinopathy, achieving 100 million dollar milestone payment from AbbVie.

Speaker #3: We have substantially strengthened our financial position through receipt of over 200 million dollars total, inclusive of the AbbVie milestone payment, and new capital ending the quarter pro forma with more than 310 million dollars.

Speaker #3: This extends our runway into Q4 2027, which includes the expected PDUFA date for our GX2 and 202 and brings us closer to our goal of delivering new, needed medicines to patients and generating our first product revenues.

Curran M. Simpson: This extends our runway into Q4 2027, which includes the expected PDUFA date for RGX-202 and brings us closer to our goal of delivering new, needed medicines to patients and generating our first product revenues. Before I turn the call over to Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer, to provide updates on our clinical and financial progress, respectively, I'd like to highlight a few key program updates. Let's start with RGX-202, our wholly owned potential best-in-class therapy for Duchenne muscular dystrophy. We believe the top-line pivotal data shared in May further establish RGX-202 as a differentiated gene therapy candidate. RGX-202 has uniquely demonstrated a large magnitude of effect relative to baseline and strong correlation between microdystrophin expression and functional improvement at 1 year. This is a comprehensive body of evidence that we believe will support potential accelerated approval.

Curran M. Simpson: This extends our runway into Q4 2027, which includes the expected PDUFA date for RGX-202 and brings us closer to our goal of delivering new, needed medicines to patients and generating our first product revenues. Before I turn the call over to Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer, to provide updates on our clinical and financial progress, respectively, I'd like to highlight a few key program updates. Let's start with RGX-202, our wholly owned potential best-in-class therapy for Duchenne muscular dystrophy. We believe the top-line pivotal data shared in May further establish RGX-202 as a differentiated gene therapy candidate. RGX-202 has uniquely demonstrated a large magnitude of effect relative to baseline and strong correlation between microdystrophin expression and functional improvement at one year. This is a comprehensive body of evidence that we believe will support potential accelerated approval.

Speaker #3: Before I turn the call over to Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer, to provide updates on our clinical and financial progress respectively, I'd like to highlight a few key program updates.

Speaker #3: Let's start with our GX202, our wholly owned potential best-in-class therapy for Duchenne muscular dystrophy. We believe the top-line pivotal data shared in May further establish our GX2 as a differentiated gene therapy candidate.

Speaker #3: Our GX202 has uniquely demonstrated a large magnitude of effect relative to baseline and strong correlation between microdystrophin expression and functional improvement at one year.

Speaker #3: This is a comprehensive body of evidence that we believe will support potential accelerated approval. We recently reported that we had fully enrolled and completed dosing in the confirmatory study of our GX202 ahead of schedule.

Curran M. Simpson: We recently reported that we had fully enrolled and completed dosing in the confirmatory study of RGX-202 ahead of schedule. We have enrolled over 60 patients in the pivotal and confirmatory trials to support a robust safety data set in the planned BLA filing. Momentum for RGX-202 remains strong. Our strengthened cash position enables continued investment in our strategic priorities, including preparing for the US commercial launch of RGX-202. We will soon initiate a randomized, placebo-controlled study in Duchenne named AFFINITY-RISE outside the US to support future global regulatory submissions. We also continue to manufacture intended commercial supply at our FDA-inspected commercial-ready manufacturing facility located in Rockville, Maryland.

Speaker #3: We have enrolled over 60 patients in the pivotal and confirmatory trials to support a robust safety dataset in the planned BLA filing. Momentum for our GX202 remains strong.

Speaker #3: Our strengthened cash position enables continued investment in our strategic priorities. Including preparing for the U.S. commercial launch of our GX202. We will soon placebo-controlled study in Duchenne named AffinityRISE outside the U.S.

Speaker #3: regulatory submissions. We also continue to manufacture intended commercial supply at our FDA-inspected commercial-ready manufacturing facility located in Rockville, Maryland. We remain committed to bringing our GX202 to patients as soon as possible through multiple key milestones, including: initiating the XUS-RCT in the first half of 2027, and submitting the first module of to support future global the BLA to FDA in Q3 2026 with potential U.S.

Curran M. Simpson: We remain committed to bringing RGX-202 to patients as soon as possible through multiple key milestones, including initiating the ex-U.S. RCT in H1 2027 and submitting the first module of the BLA to FDA in Q3 2026, with potential US approval in H2 2027. Moving to RGX-121, following our collaborative discussion with FDA in June, where we aligned on a path forward, we have since held a productive Type A meeting with the agency in July. During the meeting, the FDA confirmed that our available data is sufficient for review under the accelerated approval pathway, and no additional studies, including an RCT, will be required for BLA resubmission. The resubmission will include longer-term efficacy and safety data, including participant imaging that we have submitted and continue to compile and analyze as part of our ongoing monitoring requirements.

Speaker #3: approval in the second half of 2027. Moving to our GX121, following our collaborative discussion with FDA in June, where we aligned on a path forward, we have since held a productive type A meeting with the agency in July.

Speaker #3: During the meeting, the FDA confirmed that our available data is sufficient for review under the accelerated approval pathway, and no additional studies including an RCT will be required for BLA resubmission.

Speaker #3: The resubmission will include longer-term efficacy and safety data, including participant imaging that we have submitted and continue to compile and analyze as part of our ongoing monitoring requirements.

Speaker #3: And we are working to resubmit the BLA in Q3. Beyond our rare programs, we continue to make meaningful progress across our retinofranchise through our strategic collaboration with AbbVie.

Curran M. Simpson: We are working to resubmit the BLA in Q3. Beyond our rare programs, we continue to make meaningful progress across our retinal franchise through our strategic collaboration with AbbVie. Along with dosing the first patient in the phase II-B/III NAAVIGATE study for diabetic retinopathy, we and AbbVie recently presented long-term data in both wet AMD and diabetic retinopathy. These studies highlight the durable safety and efficacy profile that underscores our confidence in the potential commercial success of sura-vec. With the NAAVIGATE study now underway in diabetic retinopathy, near-term focus of the partnership has shifted to the upcoming pivotal readout for sura-vec in subretinal wet AMD, a milestone that is highly anticipated in the field. We are pleased with the continued momentum across the collaboration and look forward to sharing top-line data from the ATMOSPHERE and ASCENT studies in Q4.

Speaker #3: Along with dosing the first patient in the phase 2B/3 Navigate study for diabetic retinopathy, we and AbbVie recently presented long-term data in both wet AMD and diabetic retinopathy.

Speaker #3: These studies highlight the durable safety and efficacy profile that underscores our confidence in the potential commercial success of CureVac. With the Navigate study now underway in diabetic retinopathy, near-term focus of the partnership has shifted to the upcoming pivotal readout for CureVac in subretinal wet AMD, a milestone that is highly anticipated in the field.

Speaker #3: We are pleased with the continued momentum across the collaboration and look forward to sharing top-line data from the Atmosphere and Ascent studies in the fourth quarter.

Speaker #3: Our focus is clear for the remainder of 2026. Execute against our key milestones and bring hope for transformative gene therapies closer to patients in need.

Curran M. Simpson: Our focus is clear for the remainder of 2026: execute against our key milestones and bring hope for transformative gene therapies closer to patients in need. With that, I'll turn it over to Steve.

Speaker #3: With that, I'll turn it over to Steve.

Speaker #2: Thank you, Curran. I'll start with the our GX202 program for the treatment of Duchenne. As Curran referenced, we are incredibly excited by the continued momentum we have seen in the Affinity Duchenne clinical program and look forward to initiating BLA submission this quarter.

Curran M. Simpson: Thank you, Curran. I'll start with the RGX-202 program for the treatment of Duchenne.

Steve Pakola: Thank you, Curran. I'll start with the RGX-202 program for the treatment of Duchenne.

Steve Pakola: As Curran referenced, we are incredibly excited by the continued momentum we have seen in the AFFINITY DUCHENNE clinical program, and look forward to initiating BLA submission this quarter. As a reminder, RGX-202 is the most advanced clinical stage gene therapy program in Duchenne, and both the pivotal and confirmatory studies enrolled ambulatory patients aged one and older. As reported in May, top-line results from our pivotal study demonstrated a highly compelling combination of robust microdystrophin expression, encouraging functional improvement, including in older boys, and a favorable safety profile. These positive results, together with the statistically significant strong correlation observed between microdystrophin expression and NSAA improvement, will serve as a key component of our upcoming BLA submission. As Curran shared, we are also progressing plans to expand the clinical development of 202 to support future regulatory submissions outside the US through the ex-US AFFINITY RISE study.

Speaker #2: As a reminder, our GX202 is the most advanced clinical-stage gene therapy program in Duchenne. And both the pivotal and confirmatory studies enrolled ambulatory patients aged 1 and older as reported in May, top-line results from our pivotal study demonstrated a highly compelling combination of robust microdystrophin expression encouraging functional improvement, including in older boys, and a favorable safety profile.

Speaker #2: These positive results, together with the statistically significant strong correlation observed between microdystrophin expression and NSAA improvement, will serve as a key component of our upcoming BLA submission.

Speaker #2: As Curran shared, we are also progressing plans to expand the clinical development of 202 to support future regulatory submissions outside the U.S. through the XUS AffinityRISE study.

Speaker #2: This global double-mass placebo-controlled randomized trial is designed to enroll approximately 100 patients with a 2:1 active-to-placebo randomization. With enrollment now complete in the confirmatory study, we are excited to initiate this study in the first half of next year.

Steve Pakola: This global, double-masked, placebo-controlled randomized trial is designed to enroll approximately 100 patients with a two-to-one active to placebo randomization. With enrollment now complete in the confirmatory study, we are excited to initiate this study in H1 of next year. We look forward to sharing more as we progress. Turning now to our retina franchise, we continue to be encouraged by the growing body of evidence supporting sura-vec as a differentiated potential one-time gene therapy for retinal disease. Last month at ASRS, we presented multiple data sets that further reinforce the durability, efficacy, and safety profile of the program across both wet AMD and DR. In subretinal wet AMD, we reported long-term follow-up data from our phase I/II study. Results demonstrated sura-vec maintained or improved visual acuity with a meaningful reduction in treatment burden through five years.

Speaker #2: We look forward to sharing more as we progress. Turning now to our retinofranchise, we continue to be encouraged by the growing body of evidence supporting CureVac as a differentiated potential one-time gene therapy for retinal disease.

Speaker #2: Last month, the ASRS, we presented multiple datasets that further reinforced the durability, efficacy, and safety profile of the program across both wet AMD and DR. In subretinal wet AMD, we reported long-term follow-up data from our phase 1-2 study.

Speaker #2: Results demonstrated CureVac maintained or improved visual acuity with a meaningful reduction in treatment burden through 5 years. These results are even more impressive as they reflect a wet AMD population that faced a high burden of chronic anti-VEGF injections prior to receiving one-time gene therapy.

Steve Pakola: These results are even more impressive as they reflect a wet AMD population that faced a high burden of chronic anti-VEGF injections prior to receiving one-time gene therapy. We are very encouraged by these results as we approach top-line data later this year. In DR, we presented new two-and-a-half-year follow-up data from the ALTITUDE study. These results demonstrated sura-vec maintained durable improvements in disease severity, continued prevention of vision-threatening complications, and a favorable long-term safety profile following a single administration. While chronic anti-VEGF injections are approved for DR, real-world data shows that there is a staggeringly low use due to the high treatment burden. We believe the potential to prevent vision-threatening complications with a one-time in-office administration represents an important option for these patients. Finally, this summer we have had the privilege of joining both the Duchenne and Hunter syndrome communities at family and advocacy conferences.

Speaker #2: We're very encouraged by these results as we approach top-line data later this year. In DR, we presented new 2.5-year follow-up data from the altitude study.

Speaker #2: These results demonstrated CureVac maintained durable improvements in disease severity, continued prevention of vision-threatening complications, and a favorable long-term safety profile following a single administration.

Speaker #2: While chronic anti-VEGF injections are approved for DR, real-world data shows that there is a staggeringly low use due to the high treatment burden. We believe the potential to prevent vision-threatening complications with a one-time in-office administration represents an important option for these patients.

Speaker #2: Finally, this summer, we've had the privilege of joining both the Duchenne and Hunter syndrome communities at family and advocacy conferences. These families and advocates inspire us and power our mission every day.

Steve Pakola: These families and advocates inspire us and power our mission every day. Every interaction we have at these events underscore how urgently families are waiting for new treatment options that can meaningfully change the course of these diseases. We are deeply grateful for the community's partnership, support, and enthusiasm for our programs. With that, I will turn the call over to Mitch to review our financial results. Mitch?

Speaker #2: Every interaction we have at these events underscores how urgently families are waiting for new treatment options that can meaningfully change the course of these diseases.

Speaker #2: We're deeply grateful for the community's partnership, support, and enthusiasm for our programs. With that, I'll turn the call over to Mitch to review our financial results.

Speaker #2: Mitch?

Speaker #3: Thank you, Steve, and good morning, everyone. REGENXBIO ended the second quarter of 2026 with cash, cash equivalents, and marketable securities of $106 million. Research and development and general administrative expenses are generally consistent with the same period in 2025, reflecting the continued advancement of our late-stage clinical programs and our operational capabilities to support our planned transition to a commercial-stage organization.

Mitch Chan: Thank you, Steve, and good morning, everyone. REGENXBIO ended the Q2 2026 with cash equivalent, and marketable securities of $106 million. Research and development and general administrative expenses are generally consistent with the same period in 2025, reflecting the continued advancement of our late-stage clinical programs and our operational capabilities to support our planned transition to a commercial stage organization. Subsequent to the quarter, we strengthened our financial position through two important financing events. First, we received the $100 million milestone payment from AbbVie following our first patient dose in the phase IIb/III NAAVIGATE study for DR, reflecting the continued progress within our strategic retinal collaboration. We successfully completed a follow-on public offering of approximately $108 million in net proceeds. We end the quarter pro forma with more than $310 million.

Mitchell Chan: Thank you, Steve, and good morning, everyone. REGENXBIO ended the Q2 2026 with cash equivalent, and marketable securities of $106 million. Research and development and general administrative expenses are generally consistent with the same period in 2025, reflecting the continued advancement of our late-stage clinical programs and our operational capabilities to support our planned transition to a commercial stage organization. Subsequent to the quarter, we strengthened our financial position through two important financing events. First, we received the $100 million milestone payment from AbbVie following our first patient dose in the phase IIb/III NAAVIGATE study for DR, reflecting the continued progress within our strategic retinal collaboration. We successfully completed a follow-on public offering of approximately $108 million in net proceeds. We end the quarter pro forma with more than $310 million.

Speaker #3: Subsequent to the quarter, we strengthened our financial position through two important financing events. First, we received the $100 million milestone payment from AbbVie following our first patient dose in the phase 2B/3 Navigate study for DR, reflecting the continue progress within our strategic retinal collaboration.

Speaker #3: In addition, we successfully completed a follow-on public offering of approximately $108 million in net proceeds. We end the quarter pro forma with more than $310 million.

Speaker #3: These additional resources support both near and long-term strategic priorities including advancing commercial readiness activity across our late-stage programs specific near-term investment to prepare for the potential RGX202 commercial launch in the United States and planned initiation of RGX202 XUS RCT study to support future regulatory opportunities outside the United States.

Mitch Chan: These additional resources support both near and long-term strategic priorities, including advancing commercial readiness activity across our late-stage programs, specific near-term investment to prepare for the potential RGX-202 commercial launch in the United States, and planned initiation of RGX-202 ex-US RCT study to support future regulatory opportunities outside the United States. Including these proceeds, REGENXBIO's cash runway is into the Q4 2027, which enables us to complete multiple milestones, including the top-line data in wet AMD and expected PDUFA date for RGX-202. This cash runway guidance does not include any potential receipt of proceeds associated with additional potential non-dilutive sources of funding, including healthcare royalty agreement, milestone payment associated with our partner programs, or any proceeds from the potential sale of RGX-121 PRV. We find ourselves well positioned to leverage these and other funding options as we advance towards multiple product launches.

Mitchell Chan: These additional resources support both near and long-term strategic priorities, including advancing commercial readiness activity across our late-stage programs, specific near-term investment to prepare for the potential RGX-202 commercial launch in the United States, and planned initiation of RGX-202 ex-US RCT study to support future regulatory opportunities outside the United States. Including these proceeds, REGENXBIO's cash runway is into the Q4 2027, which enables us to complete multiple milestones, including the top-line data in wet AMD and expected PDUFA date for RGX-202. This cash runway guidance does not include any potential receipt of proceeds associated with additional potential non-dilutive sources of funding, including healthcare royalty agreement, milestone payment associated with our partner programs, or any proceeds from the potential sale of RGX-121 PRV. We find ourselves well positioned to leverage these and other funding options as we advance towards multiple product launches.

Speaker #3: Including these proceeds, REGENXBIO's cash runway is into the fourth quarter of 2027, which enables us to complete multiple milestones including the top-line data in wet AMD and expected PDUVA day for RGX202.

Speaker #3: This cash runway guidance does not include any potential receipt of proceeds associated with additional potential non-dilutive sources of funding, including healthcare royalty agreement, milestone payment associated with our partner programs, or any proceeds from the potential sale of RGX121 PRV.

Speaker #3: We find ourselves well-positioned to leverage these and other funding options as we advance towards multiple product launches. With that, I turn the call back to Curran to provide final thoughts.

Mitch Chan: With that, I turn the call back to Curran to provide final thoughts.

Mitchell Chan: With that, I turn the call back to Curran to provide final thoughts.

Speaker #1: Thank you, Mitch. We leave today's call with confidence in our strategy, our execution, and our financial position. With the capital to deliver against multiple catalysts, we remain sharply focused on executing and advancing potentially transformative gene therapies to patients.

Curran M. Simpson: Thank you, Mitch. We leave today's call with confidence in our strategy, our execution, and our financial position. With the capital to deliver against multiple catalysts, we remain sharply focused on executing and advancing potentially transformative gene therapies to patients. We believe the next 18 months will be a defining period for REGENXBIO, and we look forward to sharing our progress. With that, I'll turn over the call for questions. Operator?

Speaker #1: We believe the next 18 months will be a defining period for REGENXBIO, and we look forward to sharing our progress. With that, I'll turn over the call for questions.

Speaker #1: Operator?

Speaker #4: Thank you. We will now begin the question-and-answer session. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and enter the queue.

Operator 2: Thank you. We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and enter the queue. If you would like to withdraw your question, simply press star one again. We will pause for just a moment to compile the Q&A roster. Your question comes from the line of Judah Frommer from MS. Your line is now open.

Operator: Thank you. We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and enter the queue. If you would like to withdraw your question, simply press star one again. We will pause for just a moment to compile the Q&A roster. Your question comes from the line of Judah Frommer from MS. Your line is now open.

Speaker #4: If you would like to withdraw your question, simply press star 1 again. We will pause for just a moment to compile the Q&A roster.

Speaker #4: Your question comes from the line of Judah Frommer from MS. Your line is now open.

Speaker #5: Good morning, guys. Congrats on the progress, and thanks for taking the questions. Two from us. Maybe first, could you just give us a little more color on enrollment for the Affinity confirmatory study?

Judah Frommer: Good morning, guys. Congrats on the progress and thanks for taking my questions. Two from us. Maybe first, can you just give us a little more color on enrollment for the AFFINITY confirmatory study, what demand looked like from patients and investigators there? It does seem like you enrolled relatively quickly. Maybe just feedback from ASRS, kind of general excitement for gene therapy versus alternative modalities that extend treatment. Any particular feedback on subretinal and suprachoroidal delivery versus intravitreal gene therapies, and relative unmet need in wet AMD versus DR for gene therapy? Thanks.

Judah C. Frommer: Good morning, guys. Congrats on the progress and thanks for taking my questions. Two from us. Maybe first, can you just give us a little more color on enrollment for the AFFINITY confirmatory study, what demand looked like from patients and investigators there? It does seem like you enrolled relatively quickly. Maybe just feedback from ASRS, kind of general excitement for gene therapy versus alternative modalities that extend treatment. Any particular feedback on subretinal and suprachoroidal delivery versus intravitreal gene therapies, and relative unmet need in wet AMD versus DR for gene therapy? Thanks.

Speaker #5: What demand looked like from patients and investigators there? It does seem like that enrolled relatively quickly. And then maybe just feedback from ASRS, kind of general excitement for gene therapy, versus alternative modalities that extend treatment.

Speaker #5: Any particular feedback on subretinal and suprachoroidal delivery versus intravitreal gene therapies? And the relative unmet need in wet AMD versus DR for gene therapy? Thanks.

Speaker #3: And just to clarify, Judah, the Affinity confirmatory is speaking to Affinity RISE, the new program, or a different one?

Curran M. Simpson: Just to clarify, Judah, the AFFINITY confirmatory, you're speaking to AFFINITY RISE, the new program, or a different one?

Speaker #5: The confirmatory that I believe will, I think it's set up as the phase 3 portion of Affinity Duchenne. Is that right?

Judah Frommer: The confirmatory that I believe I think it's set up as the phase III portion of AFFINITY Duchenne. Is that right?

Judah C. Frommer: The confirmatory that I believe I think it's set up as the phase III portion of AFFINITY Duchenne. Is that right?

Speaker #3: Okay. Yeah.

Curran M. Simpson: Okay. Yeah. I think

Speaker #5: That's completed in June. The enrollment that completed in June.

Judah Frommer: That completed in June. The enrollment of that completed in June.

Judah C. Frommer: That completed in June. The enrollment of that completed in June.

Speaker #3: Yeah. I think when we think forward to the global study that we just announced today, we certainly think enrollment, just at a 100,000-foot level, will be very positive. We enrolled the 30 patients in our confirmatory study post the pivotal ahead of schedule.

Curran M. Simpson: Yeah. I think when we think forward to the global study that we just announced today, we certainly think enrollment, just at 100,000-foot level, will be very positive. We enrolled the 30 patients in our confirmatory study post the pivotal ahead of schedule. I think that's an indicator of overall patient demand. Globally as well, you see significant uptake of gene therapy ex-US. I think we feel that enrollment in that study is positive. I'll turn it to Steve for his thoughts.

Speaker #3: I think that's an indicator of overall patient demand. And globally as well, you see significant uptake of gene therapy ex-U.S. So, I think we feel that enrollment in that study is positive. I'll turn it to Steve for thoughts.

Speaker #5: Sure. So Curran, you gave the $100,000-foot view. I can sort of give the ground-level view. And as you mentioned, Judah, enrollment was really good, and I think the more data we gathered, the more enthusiasm there has been and, you know, as sites would treat a patient, they would, you know, get more excited as well.

Steve Pakola: Sure. Curran, you gave the 100,000-foot view. I can sort of give the ground-level view. As you mentioned, Judah, enrollment was really good. I think the more data we gathered, the more enthusiasm there has been. As sites would treat a patient, they would get more excited as well. I think a lot of that is obviously the differentiation of 202 that we've talked about in terms of the product itself and the safety profile, as well as the encouraging functional results that we're already seeing. Yeah, I think that really gives us a lot of momentum going into AFFINITY RISE. The other question you had was on 314 and ASRS last week, I think the one-word key message or what was absorbed was durability.

Speaker #5: So I think a lot of that is obviously the differentiation of 202 that we've talked about in terms of the product itself and the safety profile as well as the encouraging functional results that we're already seeing.

Speaker #5: So, yeah, I think that really gives us a lot of momentum going into Affinity RISE. The other question you had was on 314, and ASRS last week, and I think the one word key message or what was absorbed was durability.

Speaker #5: So in both wet AMD and DR, we presented longer-term follow-up. So five-year results from wet AMD and two and a half-year results from DR, and we're seeing great durability and excellent safety with longer-term follow-up.

Steve Pakola: In both wet AMD and DR, we presented 5-year results from wet AMD and 2.5-year results from DR. We are seeing great durability and excellent safety with longer-term follow-up. I think to the follow-up or the additional question on that as far as unmet need that we are seeing compared to some of the other nice advances that have happened in the space in terms of durability. One of the nice things with the greater durability is that we are seeing more and more interest on the one-time option. Big advance or unmet need if you can have that in sustained anti-VEGF. DR, the unmet need and the differentiation is even clearer because in that disease with an asymptomatic nature, you really need a one-time treatment, not repeated injections over time.

Speaker #5: And, you know, I think to the follow-up or the additional question on that, as far as unmet need that we're seeing compared to some of the other nice advances that have happened in the space in terms of durability—you know, one of the nice things with the greater durability is that we're seeing more and more interest in the one-time option.

Speaker #5: So, a big advance or unmet need—if you can have that and sustained anti-VEGF—and DR, the unmet need and the differentiation is even clearer, because in that disease with an asymptomatic nature, you really need a one-time treatment, not repeated injections over time.

Speaker #5: Thanks.

Judah Frommer: Thanks.

Judah C. Frommer: Thanks.

Speaker #4: Your next question comes from Lily Anson-Goh from Lyrinc. Your line is now open.

Operator 2: Your next question comes from Lili Nsongo from Leerink. Your line is now open.

Operator: Your next question comes from Lili Nsongo from Leerink. Your line is now open.

Lili Nsongo: Hi. Good morning, and thank you for the updates. Maybe just a question regarding the patent portfolio. How should we think about the potential impact of patent expiry on Zolgensma royalty stream in the coming years? How should we think about potential revenue stream or royalty rate for Itvisma compared to Zolgensma?

Speaker #6: Hi. Good morning, and thank you for the update. So maybe just a question regarding the patent portfolio. How should we think about the potential impact of patent expiry on Zolgensma royalty stream in the coming years, and how should we think about potential revenue stream or royalty rate for investment compared to Zolgensma?

Speaker #5: Sure, this is Patrick Christmas. I can start. As we've announced, our U.S. patent on Zolgensma has expired, but we do have coverage in about 20 countries outside the United States, where we'll continue to see revenue and royalties on Zolgensma.

Steve Pakola: Sure. This is Patrick Christmas. I can start. As we have announced, our US patent on Zolgensma has expired, but we do have coverage in about 20 countries outside the United States, where we will continue to see revenue and royalties on Zolgensma. In addition, we have coverage on Itvisma, both in the US and worldwide on that product. I think Novartis has announced that they could reach up to $2 billion in sales, so we expect to see continued significant royalties there as well.

Patrick J. Christmas II: Sure. This is Patrick Christmas. I can start. As we have announced, our US patent on Zolgensma has expired, but we do have coverage in about 20 countries outside the United States, where we will continue to see revenue and royalties on Zolgensma. In addition, we have coverage on Itvisma, both in the US and worldwide on that product. I think Novartis has announced that they could reach up to $2 billion in sales, so we expect to see continued significant royalties there as well.

Speaker #5: In addition, we have coverage on Advisma, both in the U.S. and worldwide on that product, and I think I think Novartis has announced that they could reach up to $2 billion in sales and so we expect to see continued significant royalties there as well.

Speaker #6: Thank you.

Lili Nsongo: Thank you.

Speaker #4: Your next question comes from the line of Annabel Samimi from Stephol. Your line is now open.

Operator 2: Your next question comes from the line of Annabel Samimy from Stifel. Your line is now open.

Operator: Your next question comes from the line of Annabel Samimy from Stifel. Your line is now open.

Speaker #7: Hi. Thanks for taking my question. A couple here. So for wet AMD, as you approach data, I know most of these physicians are retinal surgeons and perform vitrectomies, but how many physicians would you say have already been trained on your procedure specifically, and can you remind us how many patients have opted for bilateral treatment?

Annabel Samimy: Hi. Thanks for taking my question. A couple here. For wet AMD, as you approach data, I know most of these physicians are retinal surgeons and perform vitrectomies, but how many physicians would you say have already been trained on your procedure specifically? Can you remind us how many patients have opted for bilateral treatment? Secondly, on DMD, I guess for the timing of the RCT, I think it's going to be in full swing as you are going through review. Given that you could potentially file in H1 2027, I guess suggest that you'll have some of the data and any expectations that FDA is going to wait for the biomarker outcome of that study to make any decisions on your own program. Thanks. Will require the data. Thank you.

Speaker #7: And then secondly, on DMD, I guess for the timing of the RCT, I think it's going to be in full swing as you are going through review.

Speaker #7: So, given that you could potentially file in one H27, I guess it suggests that you'll have some of the data. And any expectations that the FDA is going to wait for the biomarker outcome of that study to make any decisions on your own program?

Speaker #7: Thanks. Or will require the data. Thank you.

Speaker #3: I'll take the second one, and then we'll have Steve speak to the first question. I think on the RCT and the timing, the goal here is obviously to have it up and in active enrollment at the time of review.

Curran M. Simpson: I'll take the second one, and then we'll have Steve speak to the first question. I think on the RCT and the timing, the goal here is obviously to have it up and in active enrollment at the time of review. We think that's important in terms of the overall regulatory strategy. I don't think that there would be an expectation of data being available or specific data from that study playing onto the active review that would be for accelerated approval. I think one of the reasons for that is that we'll have over 60 patients dosed at the time of filing, so our safety database will be pretty extensive from the pivotal study and the confirmatory study that we completed enrollment on. We also, in terms of the timing, will also have roughly half the patients through 12 months of their functional assessment.

Speaker #3: We think that's important in terms of the overall regulatory strategy. I don't think that there would be an expectation of data being available or, you know, specific data from that study playing onto the active review that would be, for accelerated approval.

Speaker #3: And I think one of the reasons for that is that we'll have over 60 patients dosed at the time of filing, so our safety database will be pretty extensive from the pivotal study and the confirmatory study that we completed enrollment on.

Speaker #3: And we also in terms of the timing, we'll also have roughly half the patients through 12 months of their functional assessment. So we think we'll have a very strong data package underpinned by the really positive data we showed with our top-line release earlier this year.

Curran M. Simpson: We think we'll have a very strong data package underpinned by the really positive data we showed with our top-line release earlier this year. I think one thing that we are seeing, though, that I think is really important to our global study is, I believe in terms of obtaining accelerated approval, having a reasonable timeline for completion of a confirmatory study is an element of that. I just don't think it has to be in proximity to the approval of an AA pathway. Steve, I'll let you talk through the

Speaker #3: I think one thing that we are seeing, though, that I think is really important to our global study, is I believe in terms of obtaining accelerated approval, having a reasonable timeline for completion of a confirmatory study is an element of that.

Speaker #3: I just don't think it has to be in proximity to the approval of an AA Stephe, I'll let you talk through the.

Speaker #5: Sure. Hi, Annabel. Thanks. Thanks for the question. So on wet AMD, we've trained quite a lot of surgeons globally now. So over 500 surgeons have actually been trained and I think this speaks the actual evidence of what we've been discussing over time, the scalability of this is really straightforward, not surprising, given these are retina specialists who are used to doing much more complicated procedures.

Steve Pakola: Sure

Steve Pakola: rest of that.

Curran M. Simpson: rest of that.

Steve Pakola: Hi, Annabel. Thanks for the question. On wet AMD, we've trained quite a lot of surgeons globally now. Over 500 surgeons have actually been trained. I think this speaks the actual evidence of what we've been discussing over time. The scalability of this is really straightforward, not surprising given these are retina specialists who are used to doing much more complicated procedures. We're really excited about that as far as moving forward, we and AbbVie, of course. The bilateral question. We're seeing great interest. One issue is technically, to enroll in the bilateral study, you have to meet all of the study requirements. That already will cut out a certain proportion of patients. Although it's a bilateral disease, exactly when patients will meet the criteria in terms of disease activity will change over time. Given those aspects, we're really encouraging.

Speaker #5: So we're really excited about that as far as moving forward. We in AbbVieu, of course, and the bilateral question. So we're seeing great interest one issue is technically, so to enroll in the bilateral study, you have to meet all of the study requirements.

Speaker #5: So that already will cut out a certain proportion of patients. And although it's a bilateral disease, exactly when patients will meet the criteria in terms of disease activity will change over time.

Speaker #5: So given those aspects, you know, we're really encouraging. We hear a lot of anecdotal cases from surgeons of asking, you know, can they have their fellow eye treated if they are already getting repeated injections in both eyes before they saw whatever response they had in the 314 cerebec treated eye.

Steve Pakola: We hear a lot of anecdotal cases from surgeons of asking, can they have their fellow eye treated if they are already getting repeated injections in both eyes before they saw whatever response they had in the RGX-314 sura-vec-treated eye. We haven't given any specific numbers on this. We are AbbVie, but certainly we're encouraged.

Speaker #5: So we haven't given any specific numbers on this. We are AbbVieu, but certainly we're encouraged...

Speaker #7: Great. Thank you.

Annabel Samimy: Great. Thank you.

Speaker #4: Your next question comes from the line of Brian Scornie from Baird. Your line is now open.

Operator 2: Your next question comes from the line of Brian Skorney from Baird. Your line is now open.

Operator: Your next question comes from the line of Brian Skorney from Baird. Your line is now open.

Speaker #8: Hey, good morning, guys. Thanks for taking the question. My question is on Affinity Rise. Actually, I was hoping you could go over some more details around the study design, I sort of heard the highlights of it at the beginning of the call, but how long is the placebo-controlled period?

Brian Skorney: Hey, good morning, guys. Thanks for taking the question. My question's on AFFINITY RISE. Actually, I was hoping you could go over some more details around the study design. I sort of heard the highlights of it at the beginning of the call. How long is the placebo-controlled period, and can you just review the primary and key secondary endpoints and enrollment criteria in terms of the ages of patients? What ex-US regions do you anticipate enrolling in this study?

Speaker #8: And can you just review the primary and key secondary endpoints and enrollment criteria in terms of the ages of patients? And what ex-U.S. regions do you anticipate enrolling in this study?

Speaker #5: Yeah, thanks. Thanks, Brian. So what we've provided that you see in the press release or the high-level aspects of the design, so for XUS requirements, including Europe, not surprising, placebo controlled importantly, we've chosen a two-to-one randomization with active so we think that's a nice positive for families.

Steve Pakola: Yeah, thanks, Brian. What we've provided that you see in the press release are the high-level aspects of the design. For ex-US requirements, including Europe, not surprising, placebo-controlled. Importantly, we've chosen a 2:1 randomization with active, we think that's a nice positive for families and their children as far as odds of getting treatment. As far as the other questions that you asked, we haven't disclosed those in terms of the regions that we're going to go. Also some of the other aspects that you've mentioned, we certainly look forward to getting into more details about the design as we get closer to the initiation of the trial in the H1.

Speaker #5: And their children, as far as odds of getting treatment, you know, as far as the other questions that you asked, we haven't disclosed those in terms of the regions that we're going to go.

Speaker #5: And also, some of the other aspects that you've mentioned, but we certainly look forward to getting into more details about the design as we get closer to the initiation of the trial and the first half.

Speaker #4: Your next question comes from Alex Sternahand from Bank of America. Your line is now open.

Operator 2: Your next question comes from Alec Stranahan from Bank of America. Your line is now open.

Operator: Your next question comes from Alec Stranahan from Bank of America. Your line is now open.

Speaker #8: Hey, guys. Thanks for taking my questions. I guess one on MPS2 and then one follow-up on Affinity Rise. So on the resubmission MPS2, I guess what specific question from the FDA is the longer-term follow-up in imaging answering?

Alec Stranahan: Hey, guys. Thanks for taking my questions. I guess one on MPS II and then one follow-up on AFFINITY RISE. On the resubmission MPS II, I guess what specific question from the FDA is the longer-term follow-up in imaging answering? Do you expect this will meaningfully shift the known clinical profile for RGX-121 in this setting? On AFFINITY RISE, I guess how should we think about crossover from placebo in the study? I guess just thinking about the ethics of keeping patients on placebo for a progressive disease like DMD. Just to clarify, will this study enroll any US patients? Thank you.

Speaker #8: Like, do you expect this will meaningfully shift the known clinical profile for one-to-one in this session setting? And on Affinity Rise, I guess how should we think about crossover from placebo in the study?

Speaker #8: I guess just thinking about the ethics of keeping patients on placebo for a progressive disease like BMD and just to clarify, will this study enroll any U.S.

Speaker #8: patients? Thank you.

Speaker #3: Sure, I'll take the first question, and Steve can walk through the second. On MPS2, if you think about sort of where we started with the program, the initial data provided was six-month data for biomarker, and over the course of the ongoing review, we've now expanded that to two-year biomarker data.

Curran M. Simpson: Sure. I'll take the first question, and Steve can walk through the second. On MPS II, if you think about sort of where we started with the program, the initial data provided was 6-month data for biomarker. Over the course of the ongoing review, we've now expanded that to 2-year biomarker data and 2-year neurocog data as we've updated the filing. What was requested in the Type A was really just a consolidation of that and the safety update that would go along with that in terms of extending out the time. I think the really positive, if you focus back to the CRL, there were a couple of components of the CRL that was issued in February that were really challenging to overcome, which were really the trial design and the inclusion of a control arm.

Speaker #3: And two-year neurocog data, as we've updated the filing. So what was requested in the type A was really just a consolidation of that and the safety update that would go along with that in terms of extending out the time.

Speaker #3: So I think the really positive if you focus back to the CRL, there were a couple of components of the CRL that was issued in February, that were really challenging to overcome, which were really the trial design and the inclusion of a control arm.

Speaker #3: The type A meeting, we feel reset that to now basically an ongoing review of the data that we've provided. So we were not asked to provide additional patient patients being dosed or additional patient data beyond the two-year horizon.

Curran M. Simpson: The Type A meeting, we feel, reset that to now basically an ongoing review of the data that we've provided. We were not asked to provide additional patients being dosed or additional patient data beyond the 2-year horizon. Now I believe we'll see something closer to an evaluation of the benefit risk at that point with all of that data consolidated into one resubmission. I'll let Steve talk through AFFINITY RISE.

Speaker #3: So now I believe we'll see something closer to, you know, an evaluation of the benefit risk at that point with all of that data consolidated into one resubmission.

Speaker #3: I'll let Steve talk through Affinity Rise.

Speaker #5: Yeah, Alex, great question in terms of crossover one aspect is even how we want to get access for these patients in need and certainly patients who commit to getting into a clinical trial.

Steve Pakola: Yeah, Alec. Great question in terms of crossover. One aspect is even how we want to get access for these patients in need and certainly patients who commit to getting into a clinical trial. That's one of the reasons we wanted the 2-to-1 randomization. That still leaves the other third of patients that you're referring to. I think it's reasonable to expect that we would give those boys a chance in a crossover, and then we also get to learn more with more exposures in the trial.

Speaker #5: That's one of the reasons we wanted the two-to-one randomization. But that's still it leaves the other third of patients that you're referring to. So you know, I think it's reasonable to expect that we would give those boys a chance in a crossover and then we also get to learn more with more exposures in the trial.

Speaker #4: Your next question comes from Luca Issi from RBC Capital Markets. Your line is now open.

Operator 2: Your next question comes from Luca Issi from RBC Capital Markets. Your line is now open.

Operator: Your next question comes from Luca Issi from RBC Capital Markets. Your line is now open.

Speaker #7: Oh, great. Hi, Pam. This is Shelby on for Luca. And thanks for taking our question. Maybe on BMD, I believe you said the BLA will include a combined safety data set for over 60 patients and then 12-month functional data for at least half of the pivotal cohort.

[Analyst] (RBC Capital Markets): Oh, great. Hi, team. This is Shelby on for Luca, and thanks for taking our question. Maybe on DMD, I believe you said the BLA will include a combined safety data set for over 60 patients and then 12-month functional data for at least half of the pivotal cohort. Can you quantify how many patients will have that 12-month functional data at the time of BLA initiation this quarter versus at completion in Q1 2027? Does the FDA require kind of a pre-specified minimum for accelerated approval? Any color there much appreciated.

Shelby Hill: Oh, great. Hi, team. This is Shelby on for Luca, and thanks for taking our question. Maybe on DMD, I believe you said the BLA will include a combined safety data set for over 60 patients and then 12-month functional data for at least half of the pivotal cohort. Can you quantify how many patients will have that 12-month functional data at the time of BLA initiation this quarter versus at completion in Q1 2027? Does the FDA require kind of a pre-specified minimum for accelerated approval? Any color there much appreciated.

Speaker #7: So can you quantify how many patients will have that 12-month functional data at the time of BLA initiation this quarter versus at completion in the first quarter of 2027?

Speaker #7: And does the FDA require kind of a pre-specified minimum for accelerated approval? Any color there? Appreciate it.

Speaker #3: Sure. Yeah, I can take the last part of the last question there. There's no pre-specified level of functional data that's required for submission. And to your question around the number of patients, if you roll back to our top-line data, we presented data for nine patients at 12 months.

Curran M. Simpson: Sure. Yeah, I can take the last part of the last question there. There's no pre-specified level of functional data that's required for submission. To your question around the number of patients, if you roll back to our top-line data, we presented data for nine patients at 12 months. By the time the clinical module is submitted, which is the critical path is really defined by the CMC module, as we've talked about before. That does give us the advantage to add additional patients to the clinical module. We're estimating out of the 30 that were treated in the pivotal portion of the study, roughly half of those would be through 12 months. We're not super precise whether it's 14 or 15, and that depends a lot on visits and assessment and QC of the data.

Speaker #3: And so, by the time the clinical module is submitted—which is, the critical path is really defined by the CMC module, as we've talked about before—but that does give us the advantage to add additional patients to the clinical module.

Speaker #3: We're estimating out of the 30 that we're treated in the pivotal portion of the study, roughly half of those would be through 12 months.

Speaker #3: So we're not super precise whether it's 14 or 15 and that depends a lot on visits and assessment and QC of the data, but we obviously feel that we want to present the strongest package at that time and that's one element of this.

Curran M. Simpson: We obviously feel that we want to present the strongest package at that time, and that's one element of this.

Speaker #4: Your next question comes from the line of Ellie Merrill from Berkeley. Please go ahead.

Operator 2: Your next question comes from the line of Ellie Merle from Barclays. Please go ahead.

Operator: Your next question comes from the line of Ellie Merle from Barclays. Please go ahead.

Speaker #8: Hi, this is Joseph on for Ellie. And thank you for taking our questions. So for RGX314, what is your latest perspective on the commercial outlook in the evolving landscape?

[Analyst] (Barclays): Hi, this is Joseph on for Ellie, and thank you for taking our questions. For RGX-314, what is your latest perspective on the commercial outlook in the evolving landscape? In terms of launch preparation, could you characterize your site qualification efforts so far and provide any color on which commercial sites you plan to qualify initially? Thank you.

Joe W. Poen: Hi, this is Joe W. Poen on for Ellie, and thank you for taking our questions. For RGX-314, what is your latest perspective on the commercial outlook in the evolving landscape? In terms of launch preparation, could you characterize your site qualification efforts so far and provide any color on which commercial sites you plan to qualify initially? Thank you.

Speaker #8: And in terms of launch preparation, could you characterize your site qualification efforts so far and provide any color on which commercial sites you plan to qualify initially?

Speaker #8: Thank you.

Speaker #3: I'm sorry, just to clarify, that's for subretinal? What AMD?

Curran M. Simpson: I'm sorry, just to clarify, that's for subretinal wet AMD?

Speaker #8: That's correct.

[Analyst] (Barclays): That's correct.

Joe W. Poen: That's correct.

Speaker #3: Okay. Yeah, I think one aspect just to step back a bit is ultimately on the commercialization effort for subretinal, Abby will have the primary leadership in that role.

Curran M. Simpson: Okay. Yeah, I think one aspect, just to step back a bit, is ultimately on the commercialization effort for subretinal, AbbVie will have the primary leadership in that role. It will not be surprising that many of our clinical sites, which, Steve, correct me if I'm wrong, were well above 100 sites used for enrollment in the US. Some of those sites would be obvious for also commercialization of the product. We're just starting now joint development of the commercial plan with AbbVie, given that we expect a 12-month review cycle on the subretinal BLA filing. That work is just beginning now. It's a bit early to be specific about it, but we have no doubt that the strength of AbbVie's commercial team and their familiarity now with the program over the last three and a half years will get a running start on commercialization.

Speaker #3: It will not be surprising that many of our clinical sites, which Steve correct me if I'm wrong, were well above 100 sites used for enrollment in the U.S., would be some of those sites would be obvious for also commercialization of the product.

Speaker #3: We're just starting now joint development of the commercial plan with AbbVie. Given that we expect a 12-month review cycle on the subretinal BLA filing, so that work is just beginning now.

Speaker #3: It's a bit early to be specific about it, but we have no doubt that the strength of AbbVie's commercial team and their familiarity now with the program over the last three and a half years will get a running start on commercialization.

Speaker #3: On the prospects of the product, we look at what AMD and we look forward to a launch date in which the what AMD market could be in the range of $10 billion.

Curran M. Simpson: On the prospects of the product, we look at wet AMD, and we look forward to a launch date in which the wet AMD market could be in the range of $10 billion. I think historically, we think subretinal has a meaningful place in that market. As Steve mentioned with treatment of the fellow eye, that raises the potential commercial prospects even further. I would suggest that whenever we speak to doctors who are very experienced with this program, they can easily identify out of 10 patients, two or three that are obvious candidates for the subretinal administration because of various factors, availability for monthly injections, disease burden, et cetera. Again, we feel like there's a very meaningful market here, potentially one of the largest opportunities in gene therapy.

Speaker #3: And I think historically, we think subretinal has a meaningful place in that market. And as Steve mentioned with treatment of the Fellow I, that raises the potential commercial prospects even further.

Speaker #3: So I would suggest that whenever we speak to doctors who are very experienced with this program, they can easily identify out of 10 patients, two or three that are obvious candidates for the subretinal administration.

Speaker #3: Because of various factors, availability for monthly injections, disease burden, et cetera. So again, we feel like there's a very meaningful market opportunities in gene therapy.

Speaker #4: Your next question comes from Sean McCutcheon from Raymond James. Your line is now open.

Operator 2: Your next question comes from Sean McCutcheon from Raymond James. Your line is now open.

Operator: Your next question comes from Sean McCutcheon from Raymond James. Your line is now open.

Speaker #8: Hi guys, thanks for the question. Kind of speaking to the AMD readouts in the fourth quarter, can you speak to the expected non-inferiority margin?

Sean McCutcheon: Hi, guys. Thanks for the question. Kind of speaking to the wet AMD readouts in Q4, can you speak to the expected non-inferiority margin in ATMOSPHERE and ASCENT? Would you anticipate it to be significantly narrower than the standard 4.5 letters due to the partially masked nature of the studies? Any commentary on the powering of the studies for that would be helpful. Thanks.

Speaker #8: And atmosphere and ascent, would you anticipate it to be significantly narrower than the standard four-and-a-half letters due to the partially masked nature of the studies?

Speaker #8: And any commentary on the powering of the studies for that end would be helpful. Thanks.

Speaker #3: Great. I'll defer that one to Steve.

Curran M. Simpson: Great. I'll defer that one to Steve.

Speaker #8: Sure. So Sean, you named the non-inferiority margin 4.5 letters and that has been standard and recent history in the FDA has reiterated that. We've never had that challenged by the FDA based on the design and that masking approach has been in there throughout the study.

Steve Pakola: Sure. Sean, you named the non-inferiority margin 4.5 letters, and that has been standard in recent history, and the FDA has reiterated that. We've never had that challenged by the FDA based on the design, and that masking approach has been in there throughout the study. Working with AbbVie, we have very large sample sizes. These are the largest gene therapy programs ever executed. We have 90% power in these studies.

Speaker #8: Working with AbbVie, we have very large sample sizes. These are the largest gene therapy programs ever executed. So we have 90% power in these studies.

Speaker #4: Your next question comes from the line of Paul Choi from Goldman Sachs. Please go ahead.

Operator 2: Your next question comes from the line of Paul Choi from Goldman Sachs. Please go ahead.

Operator: Your next question comes from the line of Paul Choi from Goldman Sachs. Please go ahead.

Speaker #5: Hi, good morning and thanks for taking our questions. And congrats on all the progress. I have two questions. First on 202, can you update us on what the potential cadence of additional data updates might be as you proceed to your BLA filing?

Paul Choi: Hi. Good morning. Thanks for taking our questions, and congrats on all the progress. I have two questions. First, on RGX-202, can you update us on what the potential cadence of additional data updates might be as you proceed to your BLA filing and just what additional longer-term updates you plan to provide from the pivotal portion there. My second question is on RGX-121, with the recent approval and launch of Denali's AVLAYAH. Can you maybe just update us on what you're hearing in the field in terms of receptivity to new therapies here for hunters and how the market might potentially be primed for your and your partners' launch in the future? Thank you.

Speaker #5: And just what additional longer-term updates you plan to provide from the pivotal portion there. And my second question is on 121. And with the recent approval and launch of Denali's Avaya, can you maybe just update us on what you're hearing in the field in terms of receptivity to new therapies here for hunters and how the market might potentially be primed for your and your partner's launch in the future?

Speaker #5: Thank you.

Speaker #3: Yeah, I'll comment first on the 121 question. And I don't have any specific information regarding Denali's launch progress, but I think from our frequent interactions with the patient advocacy groups, we see a very high level of interest in their program.

Curran M. Simpson: Yeah. I'll comment first on the RGX-121 question. I don't have any specific information regarding Denali's launch progress. I think from our frequent interactions with the patient advocacy groups, we see a very high level of interest in their program. I think it's well over 20 years before a new therapy has been available to patients. I think there's excitement. I think, thinking about RGX-121, our goal is to provide potential options for patients to choose their therapy beyond what's already available. I do think indications of strong uptake, which I think is positive for the field. Indications that patients still want choices in the therapy that they choose. I think the obvious benefit of gene therapy in this case being the one-time treatment potential.

Speaker #3: I think it's well over 20 years before a new therapy has been available to patients. And so I think there's excitement. And I think thinking about RGX 121, our goal is to provide potential options for patients to choose their therapy beyond what's already available.

Speaker #3: So I do think indications of strong uptake, which I think is positive, for the field. And indications that patients still want choices in the therapy that they choose and I think the obvious benefit of gene therapy in this case being the one-time treatment potential.

Speaker #3: In terms of additional data updates, on 202, we haven't disclosed any specific updates and probably won't until the BLA filing is complete in Q1 of next year.

Curran M. Simpson: In terms of additional data updates on RGX-202, we haven't disclosed any specific updates and probably won't until the BLA filing is complete in Q1 of next year. I think around that time, once the data has been submitted, we'll obviously consider an update at that point. In the meantime, right now we don't have a specific update planned other than certainly notifying the field and the market regarding submission of the modules and timing around that to confirm that we're on track.

Speaker #3: I think around that time, once the data has been submitted, we'll obviously consider an update at that point. But in the meantime, right now we don't have a specific update planned.

Speaker #3: Other than certainly notifying the field and the market regarding submission of the modules and timing around that to confirm that we're on track.

Speaker #5: Okay, great. Thank you.

Paul Choi: Okay, great. Thank you.

Speaker #3: Thanks.

Curran M. Simpson: Thanks.

Speaker #4: Your next question comes from the line of Yi Chet from HC Wenwright. Your line is now open.

Operator 2: Your next question comes from the line of Yi Chen from H.C. Wainwright. Your line is now open.

Operator: Your next question comes from the line of Yi Chen from H.C. Wainwright. Your line is now open.

Speaker #6: Hey, good morning. This is Katie on for you. Just a couple quick clarification questions. For atmosphere and ascent, it looks like you're looking for top line in the fourth quarter of this year.

[Analyst] (H.C. Wainwright): Hey, good morning. This is Katie on for Yi. Just a couple of quick clarification questions. For ATMOSPHERE and ASCENT, looks like you are looking for top line in Q4 of this year. Will you disclose both trials together with the full non-inferiority margin and injection burden data, or is that something that is mostly AbbVie's call? Is there a milestone tied to the top line versus the 2027 submissions?

Yi Chen: Hey, good morning. This is Katie on for Yi. Just a couple of quick clarification questions. For ATMOSPHERE and ASCENT, looks like you are looking for top line in Q4 of this year. Will you disclose both trials together with the full non-inferiority margin and injection burden data, or is that something that is mostly AbbVie's call? Is there a milestone tied to the top line versus the 2027 submissions?

Speaker #6: Will you disclose both trials together with the full non-inferiority margin and injection burden data, or is that something that's mostly AbbVie's call? And is there a milestone tied to the top line versus the 2027 submissions?

Speaker #3: Yeah, I can cover that. The studies will be disclosed together. They're pretty much right on top of each other in terms of timing. We haven't given specific guidance regarding the data that will be disclosed at that point.

Curran M. Simpson: Yeah, I can cover that. The studies will be disclosed together. They are pretty much right on top of each other in terms of timing. We haven't given specific guidance regarding the data that will be disclosed at that point. That is a joint effort between AbbVie and ourselves regarding the ultimate release. Certainly, you would expect to see primary endpoint disclosure for both studies, and we will be more specific as we get closer around secondaries as well. There is not a milestone associated with top-line data, but there are milestones that we haven't disclosed the specific amounts around for BLA acceptance and then BLA approval. Those are elements that Mitch mentioned in his update, additional non-dilutive financing options that can move our cash runway even further out.

Speaker #3: That is a joint effort between AbbVie and ourselves regarding the ultimate release but certainly you would expect to see primary endpoint disclosure for both studies and we'll be more specific as we get closer around secondaries as well.

Speaker #3: There is not a milestone associated with top line data, but there are milestones that we haven't disclosed specific amounts around for BLA acceptance and then BLA approval.

Speaker #3: And those are elements that Mitch mentioned in his update. Additional non-dilutive financing options that can move our cash runway even further out.

Speaker #6: Great. Thank you guys.

[Analyst] (H.C. Wainwright): Great. Thank you, guys.

Yi Chen: Great. Thank you, guys.

Operator 2: Ladies and gentlemen, that concludes our Q&A session and today's call. Thank you all for joining. You may now disconnect.

Operator: Ladies and gentlemen, that concludes our Q&A session and today's call. Thank you all for joining. You may now disconnect.

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