Cuộc họp công bố kết quả kinh doanh quý 3/2026 của REGENXBIO (RGNX): Dữ liệu Sura-vec và BLA của RGX-202 sắp tới

Các điểm nhấn chính

• REGENXBIO kết thúc quý 3/2026 với 251 triệu USD tiền mặt, tương đương tiền và chứng khoán khả nhượng, so với 241 triệu USD vào ngày 31 tháng 12 năm 2025. Số dư này chưa bao gồm khoản 49 triệu USD thanh toán dàn xếp từ Sarepta Therapeutics nhận được vào tháng 10.
• Ban lãnh đạo dự kiến các thử nghiệm then chốt ATMOSPHERE và ASCENT đánh giá sura-vec trong điều trị thoái hóa điểm vàng thể ướt (wet AMD) sẽ công bố kết quả sơ bộ vào quý 4/2026, tiếp theo là việc đệ trình hồ sơ lên các cơ quan quản lý toàn cầu vào năm 2027.
• Hai thử nghiệm sura-vec đã tiếp nhận hơn 1.300 bệnh nhân tại 14 quốc gia. Cả hai đều đang đánh giá tính không thua kém về thị lực điều chỉnh tốt nhất ở tuần thứ 54.
• REGENXBIO dự kiến nộp đơn BLA hoàn chỉnh, thay vì nộp cuốn chiếu, cho liệu pháp điều trị chứng loạn dưỡng cơ Duchenne RGX-202 vào quý 1/2027 theo lộ trình phê duyệt nhanh. Ban lãnh đạo tiếp tục đặt mục tiêu khả năng được phê duyệt tại Mỹ vào nửa cuối năm 2027.
• Công ty dự kiến nguồn tiền mặt hiện tại sẽ đủ duy trì hoạt động đến quý 4/2027, vượt qua mốc thời gian công bố kết quả wet AMD dự kiến, các đợt nộp BLA theo kế hoạch cho RGX-202 và sura-vec, cũng như ngày PDUFA dự kiến của RGX-202.
• Đơn BLA theo kế hoạch của RGX-202 sẽ bao gồm dữ liệu an toàn từ hơn 60 bệnh nhân, dữ liệu dấu ấn sinh học từ toàn bộ 30 bệnh nhân trong nghiên cứu then chốt và các đánh giá chức năng sau 12 tháng đối với khoảng một nửa nhóm thuần tập then chốt đó.

Dữ liệu tài chính quan trọng

REGENXBIO cũng tiếp tục nhận tiền bản quyền Zolgensma tại một số vùng lãnh thổ ngoài Mỹ và tiền bản quyền Evrysdi tại Mỹ cùng một số thị trường quốc tế. Ban lãnh đạo cho biết đóng góp tiền bản quyền gần đây đến từ việc mở rộng phân phối Evrysdi tại các khu vực pháp lý đủ điều kiện.

Kết quả kinh doanh và hoạt động

Sura-vec trong điều trị thoái hóa điểm vàng thể ướt (wet AMD)

ATMOSPHERE và ASCENT mỗi thử nghiệm đều tiếp nhận hơn 600 bệnh nhân và có lực thống kê đạt 90%. Bệnh nhân được phân bổ ngẫu nhiên theo tỷ lệ 1:1:1 vào nhóm dùng sura-vec liều thấp, sura-vec liều cao hoặc nhóm đối chứng dùng kháng VEGF đã được phê duyệt. ATMOSPHERE sử dụng ranibizumab hàng tháng, trong khi ASCENT sử dụng aflibercept mỗi 8 tuần.

Tiêu chí chính là tính không thua kém về mức độ thay đổi thị lực điều chỉnh tốt nhất ở tuần thứ 54. Thông báo kết quả sơ bộ chung với AbbVie cũng dự kiến sẽ bao gồm gánh nặng tiêm và các kết quả về mặt giải phẫu.

Ban lãnh đạo cho biết chương trình phát triển bao gồm các bệnh nhân mới được chẩn đoán và đã từng điều trị, kể cả những người có mức độ hoạt động bệnh cao. Công ty mô tả mức giảm ít nhất 50% gánh nặng điều trị, với một tỷ lệ đáng kể bệnh nhân không cần tiêm mũi nào, là một mục tiêu lâm sàng quan trọng ở nhóm đối tượng khó điều trị hơn này.

REGENXBIO và AbbVie đã đào tạo hơn 500 chuyên gia võng mạc về kỹ thuật tiêm dưới võng mạc. Công ty cũng nhấn mạnh việc điều trị cho mắt còn lại là một yếu tố khác biệt tiềm năng, lưu ý rằng dữ liệu điều trị hai mắt đã được đưa vào chương trình lâm sàng sau khi đạt được sự thống nhất với FDA.

Sura-vec trong điều trị bệnh võng mạc đái tháo đường

Việc tiếp nhận bệnh nhân đang diễn ra trong nghiên cứu Pha 2b/3 NAVIGATE. Bệnh nhân đầu tiên được dùng liều đã kích hoạt khoản thanh toán cột mốc 100 triệu USD từ AbbVie.

Trong thử nghiệm Pha 2 ALTITUDE, 60% bệnh nhân dùng Mức liều 3 đã cải thiện ít nhất hai mức trên Thang đo mức độ nghiêm trọng của bệnh võng mạc đái tháo đường sau ba năm mà không cần điều trị bổ sung. Mức liều 3 cũng đang được đánh giá trong nghiên cứu NAVIGATE.

RGX-202 trong điều trị chứng loạn dưỡng cơ Duchenne

REGENXBIO đang chuẩn bị hồ sơ BLA hoàn chỉnh cho quý 1/2027. Ban lãnh đạo nêu ba yếu tố khác biệt chính: cấu trúc microdystrophin chứa miền C-terminal, phác đồ ức chế miễn dịch chủ động và quy trình sản xuất sẵn sàng cho thương mại hóa tạo ra capsid đạt xấp xỉ 80% độ tinh khiết.

Công ty cho biết dữ liệu then chốt cho thấy mối tương quan có ý nghĩa thống kê giữa sự biểu hiện microdystrophin và mức cải thiện trong Đánh giá khả năng vận động North Star sau một năm. Ban lãnh đạo coi mối quan hệ này là trọng tâm để hỗ trợ microdystrophin như một tiêu chí thay thế có khả năng hợp lý để dự đoán lợi ích lâm sàng.

Nghiên cứu toàn cầu, ngẫu nhiên và có đối chứng với giả dược AFFINITY RISE dự kiến sẽ bắt đầu vào nửa đầu năm 2027. Nghiên cứu được thiết kế để tiếp nhận khoảng 100 bệnh nhân với tỷ lệ phân bổ ngẫu nhiên 2:1 giữa RGX-202 và giả dược.

RGX-121

REGENXBIO tiếp tục thu thập dữ liệu chẩn đoán hình ảnh và theo dõi dài hạn cho RGX-121. Công ty đang phối hợp với FDA và đối tác NS Pharma nhưng chưa đưa ra mốc thời gian pháp lý cập nhật.

Định hướng của ban lãnh đạo

• Dữ liệu sura-vec trong điều trị wet AMD: Kết quả sơ bộ của ATMOSPHERE và ASCENT dự kiến sẽ có vào quý 4/2026.
• Hồ sơ pháp lý của sura-vec: Các đợt nộp hồ sơ trên toàn cầu được lên kế hoạch vào năm 2027.
• BLA của RGX-202: Kế hoạch nộp hồ sơ hoàn chỉnh dự kiến vào quý 1/2027, thay thế chiến lược nộp cuốn chiếu trước đó.
• Khả năng phê duyệt RGX-202: Ban lãnh đạo tiếp tục đặt mục tiêu vào nửa cuối năm 2027, tùy thuộc vào việc tiếp nhận và xem xét của FDA.
• Khởi động AFFINITY RISE: Dự kiến vào nửa đầu năm 2027.
• Thanh khoản: Các nguồn lực hiện có dự kiến sẽ tài trợ cho hoạt động đến quý 4/2027, mà không tính đến các nguồn tài trợ không gây pha loãng bổ sung.

Rủi ro và các điểm cần theo dõi

• Sura-vec phải chứng minh được tính không thua kém về thị lực đồng thời giúp giảm gánh nặng tiêm kháng VEGF có ý nghĩa lâm sàng.
• Khả năng thương mại hóa sẽ phụ thuộc một phần vào độ bền tác dụng và sự chấp thuận của bên chi trả bảo hiểm. Ban lãnh đạo cho biết các mã thanh toán tiêm dưới võng mạc hiện có và dữ liệu theo dõi 5 năm ở một số bệnh nhân đã củng cố đề xuất giá trị, nhưng vẫn sẽ cần một mã J-code riêng cho sản phẩm.
• RGX-202 vẫn cần sự đồng thuận của FDA về việc gói dữ liệu đủ điều kiện để được phê duyệt nhanh. Ban lãnh đạo kỳ vọng mọi thứ sẽ rõ ràng hơn sau cuộc họp tiền BLA, với việc chính thức được tiếp nhận để xem xét khoảng hai tháng sau khi nộp đơn.
• Mối tương quan chức năng của RGX-202 được báo cáo ban đầu dựa trên 9 bệnh nhân. Phân tích BLA dự kiến sẽ bao gồm dữ liệu chức năng 12 tháng từ 14 bệnh nhân, cùng với gói dữ liệu an toàn và dấu ấn sinh học rộng hơn.
• Độc tính trên gan vẫn là một yếu tố pháp lý then chốt cần cân nhắc đối với liệu pháp gen toàn thân. REGENXBIO cho biết phác đồ ức chế miễn dịch của công ty vẫn được duy trì nhất quán giữa các nghiên cứu then chốt và khẳng định, nhưng đánh giá lợi ích - rủi ro vẫn phụ thuộc vào sự xem xét của FDA.

Điểm nhấn phiên hỏi đáp của các nhà phân tích

• Chiến lược nộp hồ sơ RGX-202: Ban lãnh đạo xác nhận công ty sẽ nộp toàn bộ BLA vào quý 1/2027 thay vì nộp cuốn chiếu. Công ty không cho rằng sự thay đổi này sẽ ảnh hưởng đến tiến trình PDUFA cuối cùng.
• Bằng chứng phê duyệt nhanh: REGENXBIO dự kiến sẽ nhấn mạnh cơ sở dữ liệu về an toàn, việc giảm độc tính trên gan cùng mối tương quan giữa nồng độ microdystrophin và sự cải thiện chức năng. Biên bản cuộc họp với FDA thường có sau khoảng 30 ngày kể từ cuộc họp tiền BLA.
• Hồ sơ cạnh tranh trong điều trị wet AMD: Ban lãnh đạo khuyến cáo không nên so sánh mức giảm số mũi tiêm giữa các thử nghiệm mà không tính đến mức độ nghiêm trọng của bệnh nhân. Các nghiên cứu then chốt của công ty đã chủ ý bao gồm những bệnh nhân từng điều trị và những người có mức độ hoạt động bệnh cao.
• Điều trị hai mắt: Công ty coi việc điều trị cho mắt còn lại là rất quan trọng vì khoảng một nửa số bệnh nhân đã hoặc có thể xuất hiện bệnh ở mắt còn lại. Gói hồ sơ pháp lý của công ty bao gồm một nghiên cứu điều trị hai mắt và các bệnh nhân chuyển tiếp có mắt còn lại được điều trị.
• Độ an toàn và sự khác biệt của RGX-202: Ban lãnh đạo nhấn mạnh phác đồ ức chế miễn dịch không đổi, cấu trúc microdystrophin có miền C-terminal và độ tinh khiết trong sản xuất là những điểm khác biệt. Công ty cho rằng hiệu quả có thể ngày càng trở nên quan trọng nếu các chương trình cạnh tranh cuối cùng cũng đạt được mức độ an toàn tương đương.

Toàn văn cuộc gọi báo cáo kết quả kinh doanh

Toàn văn cuộc gọi công bố kết quả kinh doanh

Phần trình bày của ban lãnh đạo

Operator

[Operator Instructions]

Please be advised that today's conference is being recorded. I will now turn the conference over to Patrick Christmas, Chief Legal Officer of REGENXBIO. Please go ahead.

Patrick Christmas

Good morning and thank you for joining us today. Earlier this morning, REGENXBIO released financial and operating results for the third quarter ended September 30, 2026. The press release is available on our website at www.regenexxbio.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.

These risks are described in the risk factors and the management's discussion and analysis section of REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2025, and comparable factor sections of REGENXBIO's quarterly report on Form 10-Q, which will be on file with the Securities and Exchange Commission and available on the SEC's website.

Any information we provide on this conference call is provided only as of the date of this call, November 5, 2026, and we undertake no obligation 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. Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of REGENXBIO.

Curran Simpson

Thank you, Patrick, and good morning, everyone. Thank you for joining us today. This is an exciting time for REGENXBIO. We are on the cusp of multiple potential regulatory approvals and are preparing for commercialization. Joining me today are Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer. I'll provide an update on our lead programs before turning it over to them for additional details.

I'll begin with sura-vec, our potential first-in-class gene therapy for wet AMD and diabetic retinopathy, developed in collaboration with AbbVie. Ahead of our top-line pivotal readout expected later this year, last month we hosted an investor event featuring 5 leading retina specialists. They discussed the opportunity for sura-vec to transform the wet AMD treatment paradigm.

I want to highlight 3 key takeaways from the event. First, the unmet need in wet AMD remains significant. While anti-VEGF therapies are highly effective, the burden of frequent injections can be difficult for patients to sustain over time. As a result, patients who are unable to maintain consistent treatment schedules may not realize the full potential benefit of today's standard of care and subsequently lose vision.

Second, sura-vec has the potential to benefit a broad spectrum of patients, including those with high disease activity and those with bilateral disease. During our event, the physicians reinforced the advantages of a one-time treatment across the range of wet AMD patients they treat, and pointed to patients with high disease burden as ideal candidates for gene therapy. We and AbbVie incorporated these patients in our clinical studies, giving us a potential advantage in obtaining a broad label and treating these patients commercially.

sura-vec pivotal studies include a broad population of patients, including newly diagnosed and treatment-experienced patients. Additionally, sura-vec is the only late-stage gene therapy program with reported clinical data supporting treatment in both eyes.

Third, sura-vec is well positioned for commercial adoption. The commercial infrastructure for subretinal delivery already exists due to Luxturna and other approved therapies. Further, we know the majority of retina specialists performing these procedures are at high-volume centers, many of whom are included in our pivotal studies. We and AbbVie have already laid a significant foundation, having trained over 500 retina specialists to date.

Together, these factors, combined with AbbVie's global reach and leading eye care capabilities, give us confidence as we proceed towards potential commercial launch. We expect top-line results from both ATMOSPHERE and ASCENT this quarter, followed by global regulatory submissions in 2027.

Looking ahead, we see wet AMD as the tip of the spear in terms of transforming the treatment paradigm for chronic retinal diseases. We are currently enrolling in the Phase 2b/3 NAVIGATE study in diabetic retinopathy. Current anti-VEGF therapy is vastly underused in DR due to the treatment burden for patients who are predominantly middle-aged working adults. We believe a one-time gene therapy has the potential to address this large unmet need and represent another potential blockbuster opportunity for sura-vec.

Turning to RGX-202, we continue to make meaningful progress towards bringing our potential best-in-class gene therapy for Duchenne to patients in the U.S. and beyond. Preparations are well underway to submit the BLA under the accelerated approval pathway in the first quarter of 2027, supporting potential approval in the U.S. in the second half of 2027.

As a reminder, the BLA is expected to include data from both our pivotal and confirmatory studies. Outside of the U.S., momentum for RGX-202 continues, and we remain on track to initiate our ex-U.S. randomized placebo-controlled study named AFFINITY RISE in the first half of 2027. We continue to be highly encouraged by the impressive microdystrophin functional and safety data to date, which demonstrate the potential of RGX-202 to meaningfully improve outcomes for patients with Duchenne.

We have strong support from treating physicians, families, and advocacy organizations. At our FDA-inspected in-house manufacturing innovation center, we continue to manufacture intended commercial supply in anticipation of a potential 2027 commercial launch. We are sharply focused on advancing our late-stage pipeline, executing against upcoming milestones, and making disciplined investment decisions that support these priorities. With that, I'll turn it over to Steve.

Steve Pakola

Thank you, Curran, and good morning, everyone. I'll begin by speaking to our retina franchise, sura-vec, which is being developed in both wet AMD and diabetic retinopathy with our partner, AbbVie. As we approach top-line data later this quarter, let me review the design of our 2 pivotal trials, ATMOSPHERE and ASCENT. Together, these trials have enrolled over 1,300 patients across 14 countries and represent the largest gene therapy trials ever conducted.

Both trials randomized patients 1:1:1 to low-dose sura-vec, high-dose sura-vec, or an on-label anti-VEGF control. The control in ATMOSPHERE is monthly ranibizumab. In ASCENT, the control is aflibercept every 8 weeks. The primary endpoint in both studies is non-inferiority on change in best corrected visual acuity, or BCVA, at 54 weeks. Both studies are powered at 90% with more than 600 patients in each.

In the Phase 1/2 study, at doses similar to those in the pivotal studies, sura-vec-treated patients demonstrated stable to improved visual acuity and had meaningful reductions in anti-VEGF treatment burden through 5 years. Additionally, sura-vec is the only gene therapy in development to demonstrate the potential to treat both eyes. We believe the ability to target the immune-privileged subretinal space is a significant advantage for patients and physicians and could meaningfully differentiate sura-vec from other investigational gene therapies.

We look forward to reporting top-line results by year-end and expect this joint announcement with AbbVie to highlight injection burden and anatomy in addition to the primary endpoint. Turning to diabetic retinopathy, where we continue to advance our pivotal development program. Earlier this year, we dosed the first patient in the Phase 2b/3 NAVIGATE study, which triggered a $100 million milestone payment from AbbVie.

More recently, at the Retina Society Annual Scientific Meeting, we presented encouraging 3-year results from the Phase 2 ALTITUDE trial. The new data highlighted long-term results from Dose Level 3, the same dose being evaluated in NAVIGATE. At 3 years, 60% of patients improved by at least 2 steps on the Diabetic Retinopathy Severity Scale without additional treatment. And 3 of 4 patients who improved 1 step at 1 year without additional treatment went on to improve by at least 2 steps by year 3. These findings reinforce the potential of sura-vec to change the treatment landscape for the millions of patients with non-proliferative diabetic retinopathy.

Turning to RGX-121, we continue to collect and evaluate additional patient imaging and long-term follow-up data with the goal of getting this important treatment to patients. We are working closely with the FDA and with the support of our partner, NS Pharma, and we'll update you on the path forward as we progress.

Within the rare disease franchise, our primary focus remains on our potential best-in-class therapy for Duchenne, RGX-202. RGX-202 was designed to maximize potential benefit through the combination of 3 key elements. 1, our novel microdystrophin. The inclusion of the C-terminal domain makes RGX-202 the closest construct to naturally occurring dystrophin. 2, a proactive immune suppression regimen. And 3, a commercial-ready manufacturing process that delivers industry-leading product purity.

As reported in May, our top-line pivotal results demonstrated robust microdystrophin expression, encouraging functional improvement, and a favorable safety profile. These positive results, together with the statistically significant strong correlation observed between microdystrophin expression and NSAA improvement at 1 year, will be key components of our upcoming BLA submission.

Our BLA is expected to include safety data from more than 60 patients across the pivotal and confirmatory studies. It's important to remember that our analysis plans for the pivotal study were pre-specified and have not changed. The confirmatory study has already completed dosing, and the vast majority of patients in the pivotal and confirmatory studies received RGX-202 using the intended commercial manufacturing process. These are all factors that we believe contribute to the strength of our planned BLA.

Outside the U.S., we continue to make progress towards initiating the AFFINITY RISE global double-masked placebo-controlled randomized trial that is designed to enroll approximately 100 patients with a 2:1 active-to-placebo randomization. We expect this study to initiate in the first half of next year.

I want to thank our investigators and the patients and families who make our progress possible. With that, I'll turn the call over to Mitch to review our financial results.

Mitchell Chan

Thank you, Steve, and good morning, everyone.

REGENXBIO ended the third quarter of 2026 with cash, cash equivalents, and marketable securities of $251 million compared to $241 million as of December 31, 2025. The increase was due to a $100 million milestone payment received from AbbVie in July for the dosing of the first patient in the Phase 2b/3 NAVIGATE study and the net proceeds of $108 million from an underwritten public offering of common stock and pre-funded warrants completed in July 2026. This total excludes a $49 million payment received in October 2026 pursuant to a settlement agreement with Sarepta Therapeutics.

Our cash runway is into the fourth quarter of 2027 beyond multiple milestones, including top-line data in wet AMD, the BLA submissions for RGX-202 and sura-vec in wet AMD, and the expected PDUFA date for RGX-202. This cash runway guidance does not include the receipt of any potential proceeds associated with other potential non-dilutive sources of funding.

We continue to receive Zolgensma royalties in select OUS territories and Evrysdi royalties in the U.S. and certain OUS territories. We find ourselves well positioned as we advance towards multiple potential product launches.

With that, I will turn the call back to Curran to provide final thoughts.

Curran Simpson

Thank you, Mitch. Before we turn to Q&A, I want to reiterate our excitement as we approach key milestones in our lead programs through the remainder of 2026. sura-vec pivotal top-line data expected this quarter could reshape the treatment paradigm for wet AMD. With RGX-202 quickly advancing towards a potential BLA submission early next year, we're making important progress in bringing this important therapy to Duchenne patients next year.

These key near-term milestones are paving the way for our transition to a commercial company via programs with blockbuster potential. With that, I'll turn the call over for questions. Operator?

Operator

[Operator Instructions]

Our first call comes from Annabel Samimy with Stifel.

Phần hỏi đáp

Annabel Samimy

For 314, I want to know how important the fellow eye data is going to be in any of the regulatory reviews that you're going to do, that the FDA has for gene therapy and wet AMD. And then secondly, also for 314, clearly you're approaching the tail end of this program. We have 500-plus physicians that have been trained and they seem to want to use 314, and they seem to know which patients they'll want to use it in. But they've all said that it's going to depend on the payer. So can you talk about the payer work that you've done here, the reception from the payers, and if there is actually reception for gene therapy from the payers. And then I guess lastly, one quick one on 202. I thought the BLA—is the BLA filing still a rolling filing or is it just a straight filing and what impact does that have on any kind of timing of review?

Curran Simpson

Thanks, Annabel. I'll take the last question first just to clarify. The intention is in Q1 we will file the full BLA for RGX-202. So we chose to change the filing strategy from a rolling BLA to just filing the entire BLA because we wanted to have an opportunity this year to discuss in a pre-BLA setting the data that is intended to be submitted. So the entire BLA will go in. That has, in a great way, no impact on the eventual timeline for the PDUFA date because it'll be a single submission.

I think on the payer side, I would characterize a few things around discussions we've had. And this is certainly an area where AbbVie will take a lead role in discussions as we get closer with payers. But I think in general, some things to note are the codes for reimbursement associated with subretinal administration are well established. And being the key element of delivery of the product, there shouldn't be a lot. The only thing we really need ultimately is J-code for the product itself. So the mechanics of payment, I think, are well established, albeit for programs that are more rare disease like Luxturna as an example.

But we feel really optimistic. And one element that is critical to discussions we've had with payers to date is the existence of long-term data, durability data that supports the gene therapy and the benefit from that. We're out with the subretinal administration for wet AMD 5 years now with some patients. And so that will be a really important element as we discuss reimbursement with payers down the road. But we don't see any particular obstacles.

I'll let Steve speak to the fellow eye study. We do think it is absolutely a critical element of our value proposal, and Steve can describe what we've discussed, how that's incorporated in the study, and what we've discussed over time with FDA on this topic.

Steve Pakola

Sure. Hi, Annabel. Thanks for the questions. Yes, the fellow eye aspect actually has always been a critical component for our program, and it's an example of how comprehensive our global program has been. And I think a key aspect is, you know, we're really differentiated here in the ability to treat the fellow eye. So this is one of the benefits of the immune-privileged subretinal delivery, that you can have more comfort from a safety and efficacy standpoint to treat both eyes. And that's very relevant, not just for the FDA, but of course all of the treatment community, the retina specialists, because, you know, roughly half of patients, not surprisingly, do have or will develop fellow eye disease. So a treating clinician wants to know they can not only treat the first eye, but also have the opportunity to have one-time treatment for their patients in the fellow eye.

So fortunately, we always planned for this, and for a lot of the same reasons, the FDA has also cared about this. So it's—it has always been a part of this, and we did align at the end of Phase II meeting for having the package of patients who have been treated in the fellow eye. We've addressed that in 2 separate studies, 1 a bilateral treatment study, and the other is actual patients from our various clinical studies who can roll over and have the fellow eye treated.

Back on your question of the payer and value proposition. I think it's important to highlight AbbVie's work on this and their commitment to the global potential for one-time gene therapy where, you know, the actual evidence of their commitment, 1 is clearly the ACHIEVE Phase 3b study that they have taken on proactively. So this is another step that we think is just going to bolster the value proposition.

Annabel Samimy

Great, thanks for the call. Congratulations.

Steve Pakola

Thank you.

Operator

Our next call comes from the line of Maxwell Skor with Morgan Stanley.

Maxwell Skor

So after the Q1 BLA submission, when and where do you expect to present updated 202 data? What can we expect in this update? Just trying to lay expectations for next year. Thank you.

Curran Simpson

Thanks, Max. We haven't given a specific meeting or event around disclosure of the data, but we do intend to publish the data post-filing of the BLA. I think we've pointed to, in terms of the submission, which will resemble very closely the future publication, we have a safety database that's accumulated between the pivotal study and confirmatory study that is north of 60 patients. We have biomarker data for the full pivotal data set, and then about half of those, which was a 30-patient study, will be through their 12-month functional assessments. So I think that would give the parameters of the data that would be supplied.

One thing I would point out that I think is super critical to our discussions with FDA and the power of the data set that we have is we're the only company that's been able to correlate microdystrophin levels to functional benefit. And that was an element that was missing in the Elevidys AdCom, if you go back to the discussions there. They weren't able to do that. That removes a significant element of risk associated with a single-arm study because the children don't know how much microdystrophin they have, so correlating the level of microdystrophin to functional outcomes is a super powerful element of our value proposition, and I think what we hope will please the reviewers who didn't necessarily see that in the past.

Maxwell Skor

Very helpful. Thank you.

Operator

Our next call comes from the line of Lili Nsongo with Leerink Partners.

Lili Nsongo

Just a little bit regarding the pre-BLA meeting for Duchenne. Going into the meeting, what are some of the points or elements for which you go in with confidence and those points that you would potentially try to gain some clarity on? And also, how long after the meeting do you expect to get and share feedback?

Curran Simpson

I can start with the last part of that. Typically we'll receive minutes from the meeting about 30 days post the meeting itself. I think the objectives of the meeting are basically point by point to discuss with FDA how we feel our program meets the key criteria for accelerated approval. So the key elements of that are we feel that we're in a great position on safety. We've got a large safety database now between the pivotal and the confirmatory study and a significant reduction in liver toxicity demonstrated in our pivotal data set. And hopefully we can show that as well that carries over into our confirmatory study, which hasn't been published yet.

The second part, as I mentioned earlier, is the ability to correlate the microdystrophin levels we've measured in the biopsy samples for the primary endpoint to NSAA in particular, is a 12-month assessment on function. That correlation we feel underpins the fact that the primary endpoint, microdystrophin, is reasonably likely to predict a clinical benefit. And that's, I think, the main message we want to discuss with FDA, again, because that's the main tenet of an accelerated approval pathway. And so we're really excited about our data and excited to engage with FDA. And all of the elements related to the BLA submission are on track for a Q1 submission.

Lili Nsongo

Thank you for the added color.

Operator

Our next call comes from the line of Graig Suvannavejh with Mizuho.

Graig Suvannavejh

I was wondering if you could just provide an update on maybe the competitive landscape in DMD. You do have another gene therapy competitor, I think they just reported yesterday, no conference call, but can you maybe compare and contrast kind of what you see between your program and the Solid program and key areas of differentiation that you think give you the leg up perhaps over that program or any other program? Thanks.

Curran Simpson

Thank you, Graig. Happy to comment on that. I think it starts out with the initial design of the construct, which includes the C-terminus. So from the very beginning, we wanted to bring a differentiated product into clinical development. And the C-terminus in our preclinical data showed a clear benefit from a microdystrophin construct that's devoid of the C-terminus. And we think that's what's carrying over into the clinical study.

The other elements of differentiation I think that are critical is, I'd say second, the manufacturing process, which delivers a capsid at 80% purity, also at a great cost of goods. It's hard to get both, but we've been able to achieve that. We've invested in CMC for over 7 years, and now we're bearing the fruits of that for this program. I think that's a second element, which we believe not only helps with the high microdystrophin levels we're seeing in patients' biopsies, but also on safety.

And then that leads to the last leg of the stool is the immune suppression regimen that's been developed for this program, as I mentioned earlier, has allowed us to drastically reduce the liver toxicity events seen with patients. But more importantly, and I think the long-term value of the program is it allows us to deliver the maximum dose of product to give the Duchenne boys the maximum potential benefit of long-term functional benefit. And I think that was the aim is to match our preclinical data where we saw tremendous restoration of function in the MDX mouse models. We were able to get to that dose because of that IS regimen.

Now we're seeing it in our functional data, which does differentiate it from our competitor. We still to this day haven't seen anything to compare to from our competitor, whereas I think we've been very transparent and proud of our functional data and the fact that it correlates to the microdystrophin levels.

Graig Suvannavejh

Okay, great. Thanks. And if I could just ask a quick follow-up, I think, as related to your reported financials, the royalty revenue I think was just maybe slightly below what or actually was better than we had modeled. Could you just walk through the complexion of the royalty for this particular...

Mitchell Chan

Yes, I'll let Mitch cover that one. Yes, that's a great question. As a reminder, so [ Soliris ], the IP for the U.S. has since expired. There are certain jurisdictions that we continue to receive royalties for Zolgensma, but equally so, Evrysdi has since launched and is launching in a pretty good fashion. The differential that you're seeing right there is the pickup from Evrysdi as we get the royalties from certain jurisdictions including the U.S.

Operator

Our next call comes from the line of Brian Skorney with Baird.

Brian Skorney

Maybe just to follow up a little bit on the last question, just wondering on FDA discussions, obviously the 3 companies are all dialoguing with the agency right now on prophylactic immunosuppression regimens. Sarepta's trying to provide data, effectively switch to more or less your regimen, given the history of some deaths on treatment due to uncontrolled liver tox. You're talking about a submission with the pre-specified regimen and Solid's going to the agency with sort of a lighter regimen with just corticosteroids. So I guess it'd be great to get some granularity on how the agency is actually discussing sort of these different regimens and their concerns over some of the liver tox that we've seen for systemic gene therapy and the burden of evidence needed with and without sirolimus and eculizumab in the clinical practice right now. You know, maybe this is more a Sarepta question, but how prevalent is it becoming among physicians doing systemic gene therapy to just use sirolimus and eculizumab given some of the safety events here? Thanks.

Curran Simpson

Thanks. Yes, I think that safety, in particular for an accelerated approval approach, is absolutely critical. And I think what we've shown in our pivotal data set on safety is highly encouraging. We don't really see liver enzyme elevation through the course of treatment. And again, keeping in mind that we're treating at 2E14 per kilo, which is the maximum dose we wanted to attain based on our preclinical data. So that in return gives patients the best possible chance, I believe, for functional benefit. I think FDA will have this as top of mind. I think our data set will be in line with that.

We do know that other companies are adjusting their IS regimen in the field in clinical studies, etc. We haven't had to do that in any of our studies. We've been consistent throughout our pivotal and confirmatory study patients using the same exact immune suppression regimen. So I feel like that's a strength for our program is the consistency of product, consistency of safety outcomes, and the fact that the patients are all doing well.

I think it's important to maybe look 2 years out, though, and say, what if all these companies, including ourselves, equalize on safety because we learn over time how to deliver AAV more safely? That's where I think we're still going to win because we have the C-terminus, we have the highest purity, I believe, in the field. And what the market will react to then is what will deliver the best efficacy, if safety is sort of the same across programs. And I believe we're in a position to win based on the data we've published and the reaction we're getting, not so much in just the functional assessments, but what we hear from investigators. I can let Steve maybe comment a little bit on what we're hearing in the field as well on this topic.

Steve Pakola

Yeah, I think one of the nice things is, of course, we have actual experience doing this, and as Curran has highlighted, right from the beginning, we've used this regimen. And I think the aspect that comes here is, if you look at the overall benefit-risk equation, certainly patients and patient families want safety and they also want to have their best shot on goal because they literally only have 1 shot. As soon as you take a gene therapy, there's no chance to get a second 1. So you really want to have an optimal chance at success.

And that's where all the differentiators that Curran mentioned come in. And I think on your question of the regulatory view on this, it really is the highest sensitivity in the field, the liver. And I think the differentiation there is so clear, it really adds up to a really strong case for us as far as benefit over and above existing therapy.

Operator

Our next question comes from the line of Luca Issi with RBC.

Luca Issi

This is Cassie for Luca. Curran, as you have elaborated on and highlighted already, the pivotal data showed a highly statistically significant correlation between week 12 microdystrophin expression and NSAA improvement at 1 year in 9 patients. So now as you move towards your pre-BLA meeting, how do you expect the FDA, especially now under new leadership, to weigh this correlation, given you were the first to show such data and evidence is only now in single-digit number of patients? Would there be a threshold or a similar degree of correlation you expect, they expect? And lastly, the correlation was demonstrated on the NSAA as a functional measure. Can you speak to the timed function test if those are also showing a similar picture and if such data will be included in the BLA package? Thanks.

Curran Simpson

Thanks. Great question. We do think that the review team will be excited about the correlation between the 2. I think it ultimately speaks to the drug effect, and I think in a single-arm study, the things that we hear in particular I think [ Kareem Michael ] talking about at FDA where they're looking at updating regulatory frameworks for rare disease, what we're bringing to the table in terms of our data set is directly in line with what we're hearing from FDA leadership.

And that would be a few things. 1 is magnitude of effect. We believe our NSAA data to date shows a dramatic magnitude of effect advantage, particularly in the older patients where they would normally be in a decline phase. And then I think, again, the second is in a single-arm study, the ideal outcome is that you can correlate your biomarker to a clinical benefit, that underscores that your biomarker is reasonably likely to predict clinical benefit. And again, I think that's directly in line with what we're hearing from FDA. So I think as FDA focuses on rare disease, which I believe they are, and as we bring forward the data that we've shown and the additional data that will be part of the BLA submission, those 2 are directly in line with each other.

I'll let Steve talk just a little bit about what we're seeing in some of the other timed function tests.

Steve Pakola

Yes, so one of the strengths of the functional data that we've released at the top-line standpoint is not only the magnitude of effect, but the consistency of effect where we've seen functional benefit in not just the broad NSAA that traditionally people have thought is not so sensitive, but yet we've seen that effect. We've seen that effect across different methods of controlling, and then we've also seen consistent effect across the timed function tests, with a particular focus on time to stand as the traditional 1 that is of most interest.

We'll have more patients, 14 patients, for the BLA, so we'll look for continued nice magnitude of effect and totality of evidence. And since we've seen this strong correlation with NSAA, the broad measure, yes, we'll also be assessing correlation with the timed function tests. We feel good about this overall analysis because of how consistent the magnitude of effect was.

Curran Simpson

I think one thing just to add to Steve's note is in some of the communications FDA has sent our way, they have acknowledged the difference in microdystrophin, that our product is unique and contains the C-terminus. So they're looking at it, I believe, as a unique construct that deserves a unique review.

Operator

Our next question comes from the line of Sean McCutcheon with Raymond James.

Sean McCutcheon

Can you speak to the randomization criteria, patient disposition, and rescue criteria for ATMOSPHERE and ASCENT, how this differs to competitor pivotal studies for extended treatment interval therapies and how that informs expectations for BCVA stability and interpretation of treatment in burden reduction. Thanks.

Curran Simpson

Hi, Sean. Happy to do that. I'll let Steve give the specifics.

Steve Pakola

Sure. Hey, Sean. So you've hit on a lot of the key aspects that certainly were part of our prospective planning. And I think the key aspect is we wanted to have clinically relevant data. So for us, as far as the patients we enrolled and randomized, our goal was to address those patients that the clinicians told us they were eager to be able to treat with gene therapy, a one-time opportunity, and we hear over and over again that the big unmet need is the patients who have high disease activity and therefore have still frequent injections, even with the newer agents, and that's, you know, roughly in the 40% population. So if you want to address that unmet need, you have to actually evaluate it in your Phase II and particularly your pivotal study.

So we include treatment-experienced patients, including those patients with high disease activity. And we also use screening criteria where, yes, you have to show that there's a response to ranibizumab injection, but we also look 1 week after dosing where that's going to allow you to enroll not just patients who are responsive, but also these patients who, yes, they respond at 1 week, but they may have disease recurrence very quickly. And therefore, those are exactly the kind of patients you'd want to enroll.

So the other benefit is we use the same inclusion-exclusion as our Vabysmo pharmacodynamic study and also the same rescue criteria. And that gives us confidence because in that study we saw stable to improved vision, stable disease control, and a very high reduction in treatment burden, including patients not needing any injections at a year.

You mentioned the rescue criteria. So here again, same as the bioreactor study, it's all pre-specified objective based on change in best corrected visual acuity and/or reduction or worsening of fluid or disease activity measurements by OCT. So all these factors give us confidence here. And we powered the studies based on consideration of this patient population. So the goal, again, was having positive studies, but also clear evidence in the patients that we care most about. So really gives an example of how comprehensive our programs are in wet AMD, where they're not just large, they're not just global, but they're also looking at the patients that the clinicians care about.

Operator

Our next call comes from the line of Eliana Merle with Barclays.

Eliana Merle

Just ahead of the Phase 3 wet AMD data, I guess, expected imminently. A common investor question is around sort of the effect size on treatment burden reduction and the proportion of patients that will be injection-free and how to think about that in the context of the broader gene therapy landscape, and we'll be getting other Phase 3 trials from multiple other companies next year as well in the gene therapy space. I guess, given that you have a more severe patient population enrolled, I guess, how are you thinking about what would be a competitive proportion of patients that become treatment-free and then the proportion of like percent treatment reduction and how to think about how this could compare to some of the other trials that might be enrolling, you know, more treatment-naive patients? Thanks.

Curran Simpson

Thanks, Eliana. I'll start with just, I think we're in really good shape in terms of alignment with AbbVie at the outset of the program. And really based off our Phase 1/2 data, we were able to develop a target product profile that is in full alignment between us on those analyses. So I'll let Steve comment a little bit on what expectations we have in terms of sort of what good looks like in the studies.

Steve Pakola

Yes, hi Eliana. That's a great question because context matters here. You can't really go across studies and compare nominally treatment reductions without taking into account how tough to treat the patients are that you're treating in your trial. And as we discussed earlier, we have the opportunity based on earlier results to go after the clinically relevant population. The goal is really to have at least a 50% reduction in treatment burden with a meaningful proportion of those patients not needing any injection. And the key is that if we hit that mark, we're hitting it in a very tough-to-treat population. So that's the evidence that the clinicians need and the patients need in order to make their decision on treatment.

Curran Simpson

I think one other aspect that is important to that, that we've gotten information from polling a number of retinal specialists, is that if the program shows beyond 12-month durability, there's a high level of interest in gene therapy. And just as a reminder, we're out 5 years now on some patients. So I think the element of durability that's associated with sort of value and trust in our program, going to have excellent data at the time of review and eventual disclosure to support all of that.

Eliana Merle

Okay, thanks.

Operator

Our next question comes from the line of Yi Chen with H.C. Wainwright.

Yi Chen

Could you please clarify what decisions the FDA has to make post the pre-BLA meeting to enable the accelerated approval of RGX-202? Thank you.

Curran Simpson

Hi, Yi. Thanks for the question. The main decision and one of the main questions that's the topic of the pre-BLA meeting is, does the data set that we have in hand qualify for review under the accelerated approval pathway. And obviously we'll have, I think, a reasonable understanding of their position on that coming out of the pre-BLA meeting. But the true stamp on that is 2 months post-filing that they accept the BLA for review. And then it really becomes a review process through the next 6 months to go in depth through the data and support that review process.

But I think the key decision point coming up, and we'll get some color on that through the pre-BLA process, is acceptance of the BLA under the AA pathway. And we expect that about 2 months after submission. Thank you.

Operator

I am showing no further questions, and at this time, that does conclude the question-and-answer session. Thank you for your participation in today's conference. This concludes today's conference. Thank you for participating. You may now disconnect.

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