What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-08-06 compared with 2026-05-14 · 131 added, 92 removed, 255 unchanged (47% of the section changed)

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We are developing ABBV-RGX-314 (surabgene lomparvovec, sura-vec) in collaboration with AbbVie as a potential one-time treatment for chronic retinal conditions that cause total or partial vision loss, including wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).

Sura-vec is currently being evaluated in multiple clinical trials, including two pivotal trials (ATMOSPHERE and ASCENT) where enrollment has been completed, one long-term follow-up study and a fellow eye sub-study in patients with wet AMD, all utilizing subretinal delivery.

Additionally, two Phase II clinical trials in patients with wet AMD (AAVIATE) and DR (ALTITUDE) are ongoing along with two corresponding long-term follow-up studies, all utilizing in-office suprachoroidal delivery.

In wet AMD, sura-vec is currently being evaluated in multiple clinical trials utilizing subretinal delivery, including two pivotal trials (ATMOSPHERE and ASCENT) where enrollment has been completed, one long-term follow-up study and a fellow eye sub-study.

In DR, we are actively enrolling a pivotal, two-part Phase IIb/III study (NAAVIGATE) using suprachoroidal delivery.

In addition to these late-stage pivotal programs, sura-vec is being evaluated in two Phase II clinical trials in patients with wet AMD (AAVIATE) and DR (ALTITUDE), which are ongoing along with two corresponding long-term follow-up studies, all utilizing in-office suprachoroidal delivery.

Within the Phase II study in DR, we are also evaluating sura-vec in diabetic macular edema (DME).

Additionally, we have activated U.S.

clinical sites and initiated enrollment of a pivotal trial in DR and expect to dose the first patient in the two-part Phase IIb/III study (NAAVIGATE) in the second quarter of 2026.

Sura-vec uses the NAV ® AAV8 vector to deliver a gene encoding a therapeutic antibody fragment to inhibit vascular endothelial growth factor (VEGF).

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Subretinal Delivery

ATMOSPHERE ® and ASCENT ® are multi-center, randomized, active-controlled trials evaluating sura-vec versus ranibizumab and aflibercept, respectively.

The primary endpoint is non-inferiority based on change from baseline in best-corrected visual acuity (BCVA) at 54 weeks and one year, respectively.

Secondary endpoints include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections in the treatment arms.

Enrollment in the ATMOSPHERE and ASCENT pivotal trials for the treatment of patients with wet AMD using subretinal delivery was completed in October 2025.

These trials are expected to support global regulatory submissions with the U.S.

These trials, which together enrolled over 1,200 participants across more than 200 sites, are expected to support global regulatory submissions including with the U.S.

Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Topline data from these trials are expected to be shared in the fourth quarter of 2026 in partnership with AbbVie, with global regulatory submissions expected in 2027.

Topline data from these trials are expected to be announced in the fourth quarter of 2026 in partnership with AbbVie, with global regulatory submissions expected in 2027.

In July 2026, we presented long-term follow-up data from the Phase I/IIa trial that demonstrated a durable safety and efficacy profile through five years, with participants in Cohorts 3 and 4, who received subretinal sura-vec at doses similar to those being studied in the ATMOSPHERE and ASCENT pivotal trials, demonstrating stable to improved visual acuity and meaningful reductions in anti-VEGF treatment burden, with the exception of one participant in Cohort 4 with polypoidal choroidal vasculopathy refractory to anti-VEGF therapy.

Suprachoroidal Delivery

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Sura-vec for Treatment of DR and DME

In August 2025, we and AbbVie announced an amendment to our collaboration agreement and plans to initiate a pivotal program consisting of a Phase IIb/III trial (NAAVIGATE) as well as a second Phase III trial.

NAAVIGATE is a two-part, multicenter, randomized, masked, sham-controlled Phase IIb/III study to evaluate the safety and efficacy of a one-time, in-office administration of sura-vec in subjects with non-proliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema (CI-DME).

The primary endpoint is >2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) at one year.

We are actively enrolling the Phase IIb/III NAAVIGATE trial and, in June 2026, dosed the first patient in the Phase IIb portion of the trial, upon which we earned a $100.0 million milestone payment that was received from AbbVie in July 2026.

Following an interim analysis of part one (Phase IIb) of the NAAVIGATE trial, we and AbbVie expect to initiate a Phase III expansion, including part two (Phase III) of the U.S.

NAAVIGATE trial and a parallel global trial led by AbbVie.

Concurrent with the August 2025 announcement, we announced positive two-year data from the Phase II ALTITUDE ® trial.

The ALTITUDE trial is a multi-center, open label, randomized, controlled, dose-escalation Phase II trial to evaluate the efficacy, safety and tolerability of sura-vec using suprachoroidal delivery for the treatment of DR.

In November 2023, we announced data showing sura-vec was well tolerated at dose levels 1 and 2 and positive signals of efficacy, including 20.8% of patients exhibiting > 2-step Diabetic Retinopathy Severity Scale (DRSS) improvement without additional DR treatment at one year.

In August 2025, we announced positive 2-year data showing sura-vec was well tolerated in subjects with non-proliferative diabetic retinopathy (NPDR) at dose levels 1, 2, and 3.

The positive two-year data showed sura-vec was well tolerated in subjects with NPDR at dose levels 1, 2, and 3.

There were no drug-related serious adverse events and no intraocular inflammation was observed through two years at dose level 3 (1.0x10 12 GC/eye) (n = 15) with short-course topical prophylactic steroids.

50% of dose level 3 patients achieved at least a two-step improvement without need for any supplemental treatment.

Concurrent with the two-year data announcement in August 2025, we and AbbVie announced an amendment to our collaboration agreement and plans to initiate a pivotal program consisting of a Phase IIb/III trial (NAAVIGATE) as well as a second Phase III trial.

NAAVIGATE is a two-part, multicenter, randomized, masked, sham-controlled Phase IIb/III study to evaluate the safety and efficacy of sura-vec in subjects with NPDR without center-involved diabetic macular edema (CI-DME).

The primary endpoint is > 2-step improvement on the DRSS at one year.

Following an interim analysis of part one (Phase IIb) portion of the NAAVIGATE trial, we and AbbVie will initiate a Phase III expansion, including part two (Phase III) of the U.S.

NAAVIGATE trial and a parallel global trial led by AbbVie.

We have activated U.S.

clinical sites and initiated enrollment of the Phase IIb/III NAAVIGATE trial and expect to dose the first patient in the second quarter of 2026, upon which we are entitled to a $100.0 million milestone payment from AbbVie.

Additionally, 50% of dose level 3 patients achieved at least a two-step improvement without need for any supplemental treatment.

In July 2026, we presented long-term follow-up data from the Phase II ALTITUDE trial that showed, among other findings, that participants at dose level 3 (1.0×10¹² GC/eye) with short-course prophylactic topical steroids, the same dose being evaluated in the Phase IIb/III NAAVIGATE trial, maintained a durable safety and efficacy profile through 2.5 years.

55% of participants achieved >2-step improvement on the DRSS without additional treatment, and 70% of participants experienced no vision-threatening events.

These data are consistent with previously presented two-year dose level 3 NPDR data from the ALTITUDE trial and support the potential of one-time in-office sura-vec to modify the underlying disease and decrease risk of vision-threatening events.

RGX-202 for Treatment of Duchenne

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Notably, five participants for whom functional data has been reported were aged eight or older at dosing, when functional decline is expected.

In October 2025, we announced that enrollment had completed in the pivotal portion of AFFINITY DUCHENNE, which was designed to enroll approximately 30 patients in the U.S.

and Canada, and that we continue to enroll participants in the ongoing confirmatory trial.

In October 2025, we announced that enrollment had completed in the pivotal portion of AFFINITY DUCHENNE (n=approximately 30).

In May 2026, we announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE trial of RGX-202, including primary endpoint (n=30 at Week 12), interim safety (n=31) and interim functional data (n=9 at 12 months):

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• Statistically significant correlation between RGX-202 microdystrophin expression level and functional improvement, supporting validity of surrogate endpoint.

As reported in the May 2026 topline data update, we have enrolled over 20 patients in the confirmatory trial (n=30) evaluating RGX-202 in ambulatory patients aged one year and older, and we expect to complete dosing in all 60 patients across the pivotal and confirmatory trials by mid-2026.

In recent discussions with the FDA, the agency shared that the use of RGX-202 microdystrophin expression as a surrogate endpoint will be based on the correlation analysis with clinical outcomes, which has been clearly demonstrated in our interim data.

As of May 2026, in recent discussions, the FDA shared that the use of RGX-202 microdystrophin expression as a surrogate endpoint will be based on the correlation analysis with clinical outcomes, which has been clearly demonstrated in our interim data.

While the FDA has recommended a randomized controlled trial, it has guided that externally controlled trials may be adequate for demonstrating substantial evidence of effectiveness, especially when the treatment effect is sufficiently large enough to overcome limitations of externally controlled trials.

The FDA offered to review the RGX-202 data and alternative proposals.

We plan to discuss this data with the FDA at a future meeting.

We are also finalizing the trial design for a study of RGX-202 outside the United States to support global regulatory submissions.

Given the positive topline pivotal data, continued favorable safety profile, and statistically significant correlation between microdystrophin and functional improvement, we plan to pursue accelerated approval for RGX-202 and are preparing for a potential commercial launch in 2027.

Given the positive topline pivotal data, continued favorable safety profile, and statistically significant correlation between microdystrophin and functional improvement, we plan to initiate a Biologics License Application (BLA) submission in the third quarter of 2026 under the accelerated approval pathway to support potential FDA approval in the second half of 2027.

In June 2026, we announced we had completed dosing in the confirmatory study of RGX-202 and our plan to include in our planned BLA a safety dataset from the AFFINITY DUCHENNE pivotal and confirmatory studies (n=63) as well as efficacy data from the pivotal portion (n=30), including 12-month functional data for at least half of the total participants in the pivotal study.

We expect to initiate AFFINITY ® RISE, a new, ex-U.S.

randomized, placebo-controlled study to support RGX-202 global regulatory submissions, in the first half of 2027.

We have completed manufacturing the first batches of RGX-202 intended for commercial supply and manufacturing is ongoing to build commercial inventory in advance of a potential commercial launch.

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The FDA did not agree the data set provided substantial evidence of effectiveness to support approval of RGX-121 for the treatment of MPS II.

Following the CRL, we entered into discussions with FDA senior leadership in March 2026 and filed a Formal Dispute Resolution Request which is pending.

We plan to continue to work with the FDA to address the CRL and discuss potential paths forward for the RGX-121 program.

Following the CRL, we entered into discussions with FDA senior leadership in March 2026 and filed a Formal Dispute Resolution Request.

In June 2026, we announced alignment with the FDA regarding the next steps needed for a potential accelerated approval of RGX-121.

During those discussions, the FDA acknowledged that the existing RGX-121 clinical data is sufficient to be considered for the accelerated approval pathway and that the Company does not need to enroll additional patients or conduct additional studies, including the FDA’s previously recommended incorporation of an untreated control arm.

The FDA asked the Company to request a Type A meeting to review existing longer-term biomarker and clinical data and to resubmit the BLA following this meeting.

The FDA stated that it would review our resubmission on an expedited basis, with labeling discussions to begin shortly following the resubmission.

In July 2026, we and the FDA held a positive Type A meeting during which the FDA reaffirmed that no additional studies of RGX-121 are required for BLA resubmission.

We and the FDA aligned on resubmission requirements and we plan to resubmit the RGX-121 BLA in the third quarter of 2026.

The resubmission will include longer-term efficacy and safety data, including participant imaging that has been submitted to FDA and continues to be collected and analyzed as part of ongoing RGX-121 safety monitoring.

A post-approval confirmatory study will be discussed as part of BLA review.

Potential approval of the BLA for RGX-121 could result in receipt of a Rare Pediatric Disease Priority Review Voucher (PRV), assuming the statutory criteria are met.

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Of note, this participant had a background of factors that could have contributed to risk of oncogenic transformation.

For example, the participant underwent unsuccessful stem cell transplant at four months of age, with loss of donor chimerism, and he received chemotherapeutics that may have contributed to DNA damage.

For example, the participant underwent unsuccessful stem

cell transplant at four months of age, with loss of donor chimerism, and he received chemotherapeutics that may have contributed to DNA damage.

The report concluded, based on formal neuropsychologic testing and developmental pediatrician assessment, that the patient’s neurocognitive development was above average, which indicated mitigation of MPS I disease, and the patient continued to do well.

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For additional information regarding the AbbVie Collaboration Agreement, please refer to Note 10, “License and Collaboration Agreements—AbbVie Collaboration and License Agreement” to the accompanying unaudited consolidated financial statements.

In June 2026, we dosed the first patient in the NAAVIGATE trial, resulting in a $100.0 million development milestone payment from AbbVie which was fully recognized as license and royalty revenue in the second quarter of 2026.

The $100.0 million milestone payment was recorded as accounts receivable as of June 30, 2026 and was received from AbbVie in July 2026.

Nippon Shinyaku Collaboration for RGX-121 and RGX-111

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In addition to our internal product development efforts, we also selectively license the NAV Technology Platform and other intellectual property rights to other leading biotechnology and pharmaceutical companies, which we refer to as NAV Technology Licensees.

As of March 31, 2026, our NAV Technology Platform was being applied in two commercial products, Zolgensma ® and Itvisma ® , and the preclinical and clinical development of various other licensed products.

As of June 30, 2026, our NAV Technology Platform was being applied in two commercial products, Zolgensma ® and Itvisma ® , and the preclinical and clinical development of various other licensed products.

Licensing the NAV Technology Platform allows us to maintain our internal product development focus on our core disease indications and therapeutic areas while still expanding the NAV gene therapy pipeline, developing a greater breadth of treatments for patients, providing additional technological and potential clinical proof-of-concept for our NAV Technology Platform and creating additional revenue opportunities.

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Zolgensma and Itvisma Royalties

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma and Itvisma, which are marketed by Novartis Gene Therapies, Inc.

(Novartis Gene Therapies), a wholly owned subsidiary of Novartis AG (Novartis), for the treatment of spinal muscular atrophy (SMA).

Zolgensma and Itvisma are licensed products under our license agreement with Novartis Gene Therapies (the Novartis License) for the development and commercialization of treatments for SMA using the NAV Technology Platform.

In mid-January 2026, licensed patents for Zolgensma under the Novartis License expired in the United States.

Royalty revenue to date consists primarily of royalties on net sales of Zolgensma and Itvisma, which are marketed by Novartis Innovative Technologies Inc.

(formerly, Novartis Gene Therapies, Inc.), a wholly owned subsidiary of Novartis AG (Novartis), for the treatment of spinal muscular atrophy (SMA).

Zolgensma and Itvisma are licensed products under our license agreement with Novartis (the Novartis License) for the development and commercialization of treatments for SMA using the NAV Technology Platform.

In January 2026, licensed patents for Zolgensma under the Novartis License expired in the United States.

We are entitled to continued royalties on net sales of Zolgensma in approximately 20 countries where licensed patents remain active.

Licensed product made prior to patent expiration but sold after expiration may also be subject to royalties.

Patents covering the use of Itvisma have issued in the United States and certain other countries and are licensed to Novartis Gene Therapies under the Novartis License.

We are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

Licensed patents covering the use of Itvisma have issued in the United States and certain other countries, and we are entitled to ongoing royalties on certain net sales of Itvisma in these territories.

Operating Expenses

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We expect to continue to incur significant research and development expenses for the foreseeable future as we continue the development of our product candidates and engage in early research and development for prospective product candidates and new technologies.

The following table summarizes our research and development expenses incurred during the three months ended March 31, 2026 and 2025 (in thousands):

Three Months Ended March 31,

The following table summarizes our research and development expenses incurred during the three and six months ended June 30, 2026 and 2025 (in thousands):

Three Months Ended June 30,

Six Months Ended June 30,

Direct Expenses

ABBV-RGX-314 (sura-vec)

Other product candidates

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Total research and development

Direct expenses related to the development of ABBV-RGX-314 include $12.6 million and $14.7 million for the three months ended March 31, 2026 and 2025, respectively, in net cost reimbursement from AbbVie under our eye care collaboration, which were recorded as a reduction of research and development expenses.

In addition to reimbursement of direct development expenses, net cost reimbursement from AbbVie includes reimbursement of personnel and overhead costs attributable to the development of ABBV-RGX-314, the underlying costs of which are reported as unallocated expenses in the table above.

Direct expenses related to the development of sura-vec include $12.1 million and $24.7 million for the three and six months ended June 30, 2026, respectively, and $17.1 million and $31.7 million for the three and six months ended June 30, 2025, respectively, in net cost reimbursement from AbbVie under our collaboration, which were recorded as a reduction of research and development expenses.

In addition to reimbursement of direct development expenses, net cost reimbursement from AbbVie includes reimbursement of personnel and overhead costs attributable to the development of sura-vec, the underlying costs of which are reported as unallocated expenses in the table above.

We typically utilize our employee and infrastructure resources across our development programs.

As a result, we generally do not allocate personnel and other internal costs, such as facilities and other overhead costs, to specific product candidates or development programs.

result, we generally do not allocate personnel and other internal costs, such as facilities and other overhead costs, to specific product candidates or development programs.

Platform and early research reported in the table above includes direct costs not identifiable with a specific lead product candidate, including costs associated with our research and development platform used across programs, manufacturing support, process and analytical development, early research and development for prospective product candidates and new technologies, and other costs in support of research and development activities.

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We expect that our general and administrative expenses will increase as we continue to develop, and potentially commercialize, our product candidates.

Specifically, we expect general and administrative costs associated with the potential commercialization of our product candidates to increase in future periods as we and our commercial partners prepare for and carry out product launch efforts, in particular for the potential commercialization of our RGX-202 and ABBV-RGX-314 product candidates.

Other Income (Expense)

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Our consolidated results of operations were as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

License and royalty revenue

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Net income (loss)

Comparison of the Three Months Ended March 31, 2026 and 2025

Comparison of the Three Months Ended June 30, 2026 and 2025

License and Royalty Revenue.

License and royalty revenue decreased by $82.0 million, from $87.0 million for the three months ended March 31, 2025 to $5.1 million for the three months ended March 31, 2026.

The decrease was primarily attributable to $70.0 million of upfront license revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025, as well as a decrease in royalty revenues for the first quarter of 2026.

Combined Zolgensma and Itvisma royalties decreased by $11.9 million, from $17.0 million for the first quarter of 2025 to $5.1 million for the first quarter of 2026.

Novartis reported combined Zolgensma and Itvisma sales of $302 million for the first quarter of 2026, as compared to $327 million for the first quarter of 2025.

Zolgensma royalties for the first quarter of 2026 were $4.7 million, a decrease of $12.2 million from the first quarter of 2025.

The decrease was primarily attributable to the expiration of licensed patents in the United States in mid-January 2026.

License and royalty revenue increased by $85.4 million, from $18.5 million for the three months ended June 30, 2025 to $103.8 million for the three months ended June 30, 2026.

The increase was primarily attributable to $100.0 million of license revenue recognized in the second quarter of 2026 upon the achievement of a development milestone under our sura-vec collaboration with AbbVie for the first patient dosed in the NAAVIGATE trial.

The increase in license and royalty revenue was partially offset by a decrease in royalty revenues for the second quarter of 2026.

Combined Zolgensma and Itvisma royalties decreased by $14.6 million, from $18.4 million for the second quarter of 2025 to $3.8 million for the second quarter of 2026.

Novartis reported combined Zolgensma and Itvisma sales of $365 million for the second quarter of 2026, as compared to $297 million for the second quarter of 2025.

Per Novartis, sales growth was driven by continued launch momentum from Itvisma, and Zolgensma sales remained stable.

Zolgensma royalties for the second quarter of 2026 were $1.8 million, a decrease of $16.7 million from the second quarter of 2025, which was primarily attributable to the expiration of licensed patents in the United States in January 2026.

We are entitled to continued royalties on net sales of Zolgensma in approximately 20 countries where licensed patents remain active.

Itvisma royalties for the first quarter of 2026 were $0.3 million.

Itvisma royalties for the second quarter of 2026 were $2.1 million.

Itvisma was approved in the United States in the fourth quarter of 2025, with U.S.

sales commencing in the first quarter of 2026.

Itvisma is now also approved in the UAE, Japan, Qatar and the EU.

Service Revenue.

Service revenue decreased by $0.7 million, from $2.0 million for the three months ended March 31, 2025 to $1.3 million for the three months ended March 31, 2026.

The decrease was primarily attributable to service revenue recognized under our collaboration with Nippon Shinyaku, which decreased from $1.8 million for the first quarter of 2025 to $1.0 million for the first quarter of 2026, largely driven by the performance of RGX-121 development and manufacturing services.

Research and Development Expense.

Research and development expenses decreased by $3.4 million, from $59.5 million for the three months ended June 30, 2025 to $56.1 million for the three months ended June 30, 2026.

The decrease was primarily attributable to the following:

• a decrease of $3.0 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates;

• a decrease of $1.5 million in costs associated with clinical trials and regulatory activities, largely driven by a decrease in expenses for sura-vec and RGX-121 pivotal trials, and partially offset by an increase in pivotal trial expenses for RGX-202.

The decrease in research and development expenses was partially offset by an increase of $0.9 million in costs associated with preclinical activities and other early-stage research and development.

General and Administrative Expense.

General and administrative expenses increased by $1.7 million, from $19.9 million for the three months ended June 30, 2025 to $21.6 million for the three months ended June 30, 2026.

The increase was largely driven by personnel-related costs, commercialization expenses, consulting and other corporate advisory services.

Comparison of the Six Months Ended June 30, 2026 and 2025

License and Royalty Revenue.

License and royalty revenue increased by $3.4 million, from $105.5 million for the six months ended June 30, 2025 to $108.9 million for the six months ended June 30, 2026.

The increase in license and royalty revenue was partially offset by $70.0 million of non-recurring, upfront license revenue recognized under our collaboration with Nippon Shinyaku in the first quarter of 2025, as well as a decrease in royalty revenues for the first half of 2026.

Combined Zolgensma and Itvisma royalties decreased by $26.5 million, from $35.4 million for the first half of 2025 to $8.9 million for the first half of 2026.

Novartis reported combined Zolgensma and Itvisma sales of $667 million for the first half of 2026, as compared to $624 million for the first half of 2025.

Per Novartis, sales growth was driven by continued launch momentum from Itvisma, and Zolgensma sales remained stable.

Zolgensma royalties for the first half of 2026 were $6.5 million, a decrease of $28.9 million from the first half of 2025, which was primarily attributable to the expiration of licensed patents in the United States in January 2026.

We are entitled to continued royalties on net sales of Zolgensma in approximately 20 countries where licensed patents remain active.

Itvisma royalties for the first half of 2026 were $2.4 million.

Itvisma was approved in the United States in the fourth quarter of 2025, with U.S.

sales commencing in the first quarter of 2026.

Itvisma is now also approved in the UAE, Japan, Qatar and the EU.

Cost of License and Royalty Revenues.

Cost of license and royalty revenues increased by $7.6 million, from $3.4 million for the three months ended March 31, 2025 to $11.1 million for the three months ended March 31, 2026.

Cost of license and royalty revenues increased by $3.5 million, from $8.6 million for the six months ended June 30, 2025 to $12.1 million for the six months ended June 30, 2026.

The increase was largely driven by a non-recurring charge of $10.0 million in the first quarter of 2026 related to a settlement with GlaxoSmithKline LLC (GSK) to resolve a dispute over sublicense fee obligations under our license agreement with GSK.

For further information regarding the settlement agreement with GSK, please refer to Note 8, “Commitments and Contingencies—GlaxoSmithKline—GSK Settlement Agreement” to the accompanying unaudited consolidated financial statements.

The increase in cost of license and royalty revenues was partially offset by a decrease in upstream royalties on net sales of Zolgensma, consistent with the decrease in Zolgensma royalty revenues.

Research and Development Expense.

Research and development expenses increased by $4.3 million, from $53.1 million for the three months ended March 31, 2025 to $57.3 million for the three months ended March 31, 2026.

Research and development expenses increased by $0.8 million, from $112.6 million for the six months ended June 30, 2025 to $113.4 million for the six months ended June 30, 2026.

The increase was primarily attributable to the following:

• an increase of $1.3 million in costs associated with clinical trials and regulatory activities, largely driven by RGX-202 pivotal trials;

• an increase of $1.1 million in personnel-related costs due to increased headcount of development personnel, net of a $0.2 million decrease in stock-based compensation expense;

• an increase of $0.9 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates;

• an increase of $1.8 million in personnel-related costs, net of a $0.3 million decrease in stock-based compensation expense;

• an increase of $1.8 million in costs associated with preclinical activities and other early-stage research and development.

The increase in research and development expenses was partially offset by a decrease of $2.1 million in manufacturing-related expenses and other clinical supply costs for our lead product candidates.

General and Administrative Expense.

General and administrative expenses increased by $1.0 million, from $20.3 million for the three months ended March 31, 2025 to $21.3 million for the three months ended March 31, 2026.

The increase was largely driven by personnel-related costs, consulting and other corporate advisory services.

General and administrative expenses increased by $2.7 million, from $40.2 million for the six months ended June 30, 2025 to $42.9 million for the six months ended June 30, 2026.

The increase was largely driven by personnel-related costs, commercialization expenses, consulting and other corporate advisory services.

Liquidity and Capital Resources

Sources of Liquidity

As of March 31, 2026, we had cash, cash equivalents and marketable securities of $150.5 million, which were primarily derived from our royalty monetization with HCR in May 2025 and the up-front payment received under the Nippon Shinyaku Collaboration Agreement in March 2025.

At-the-Market Offering Program

In December 2024, we entered into a Sales Agreement with Leerink Partners LLC (Leerink) pursuant to which we may offer and sell shares of our common stock having an aggregate offering price of up to $150.0 million from time to time through Leerink, acting as our sales agent (the Leerink ATM Program).

As of March 31, 2026, no shares of common stock had been sold under the

Leerink ATM Program.

We intend to use proceeds obtained from the sale of shares under the Leerink ATM Program, if any, for general corporate purposes.

Future Liquidity and Ability to Continue as a Going Concern

As of June 30, 2026, we had cash, cash equivalents and marketable securities of $105.5 million, which were primarily derived from our royalty monetization with HCR in May 2025 and sales of common stock under our at-the-market offering program.

During the three and six months ended June 30, 2026, we sold 2,318,735 shares of

common stock under the ATM Program for aggregate net proceeds of $18.9 million, after deducting commissions and offering expenses.

No shares of common stock were sold under the ATM Program prior to the second quarter of 2026.

In June 2026, we dosed the first patient in the NAAVIGATE trial, resulting in a $100.0 million development milestone payment due to us under our sura-vec collaboration with AbbVie.

The $100.0 million milestone payment was recorded as accounts receivable as of June 30, 2026 and was received from AbbVie in July 2026.

In July 2026, we completed a public offering of 11,671,139 shares of our common stock (inclusive of 1,667,250 shares pursuant to the full exercise by the underwriters of their option to purchase additional shares) at a price of $9.00 per share and 1,111,111 pre-funded warrants to purchase shares of our common stock at a price of $8.9999 per pre-funded warrant, which equaled the public offering price per share of the common stock less the $0.0001 exercise price of each pre-funded warrant.

The aggregate net proceeds received from the offering were approximately $107.8 million, net of underwriting discounts and commissions and estimated offering expenses.

We intend to devote the majority of our current capital to preclinical research, clinical development, seeking regulatory approval of our product candidates and, if approved, commercialization of our product candidates, as well as additional capital expenditures needed to support these activities.

Because of the numerous risks and uncertainties associated with the development and commercialization of gene therapy product candidates, we are unable to estimate the total amount of operating expenditures and capital outlays necessary to complete the development and commercialization of our product candidates.

We expect that our cash, cash equivalents and marketable securities of $150.5 million as of March 31, 2026 will enable us to fund our operating expenses and capital expenditure requirements, and are sufficient to meet our financial commitments and obligations into early 2027.

This estimate is based on our current operating plan, and excludes the potential effects of any future financings or material milestone payments that may be received under our licensing and collaboration arrangements.

We expect that our cash, cash equivalents and marketable securities of $105.5 million as of June 30, 2026, along with the $100.0 million milestone payment received from AbbVie in July 2026 and the $107.8 million in net proceeds received from the public offering of common stock and pre-funded warrants in July 2026, are sufficient to fund operations into the fourth quarter of 2027.

As such, we believe we have the ability to meet our obligations as they become due for at least the next 12 months from the date of this report.

We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our capital resources sooner than expected.

These conditions raise substantial doubt about our ability to continue as a going concern within 12 months from the issuance date of our consolidated financial statements for the three months ended March 31, 2026, which accompany this Quarterly Report on Form 10-Q.

Our ability to continue as a going concern will depend heavily on the successful development, approval and commercialization of our product candidates and our ability to raise additional capital to fund operations.

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Our consolidated cash flows were as follows (in thousands):

Three Months Ended March 31,

Net cash provided by (used in) operating activities

Net cash provided by investing activities

Net cash used in financing activities

Net increase (decrease) in cash and cash equivalents and restricted cash

Six Months Ended June 30,

Net cash used in operating activities

Net cash provided by (used in) investing activities

Net cash provided by financing activities

Net increase in cash and cash equivalents and restricted cash

Cash Flows from Operating Activities

Our net cash used in operating activities for the three months ended March 31, 2026 increased by $109.8 million from the three months ended March 31, 2025, largely driven by the $110.0 million up-front fee received from Nippon Shinyaku in March 2025 and an increase in operating expenses in the first quarter of 2026.

We expect to continue to incur regular net cash outflows from operations for the foreseeable future as we continue the development and advancement of our product candidates and other research programs.

For the three months ended March 31, 2026, our net cash used in operating activities of $76.2 million consisted of a net loss of $90.1 million and unfavorable changes in operating assets and liabilities of $1.5 million, offset by adjustments for non-cash items of $15.4 million.

The changes in operating assets and liabilities include a decrease in accrued expenses and other current liabilities of $16.7 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and sublicense fees, and external research and development services.

The unfavorable changes in operating assets and liabilities were partially offset by a decrease in accounts receivable of $18.2 million, which was driven largely by royalties receivable on net sales of Zolgensma.

Our net cash used in operating activities for the six months ended June 30, 2026 increased by $122.7 million from the six months ended June 30, 2025, largely driven by the $110.0 million up-front fee we received from Nippon Shinyaku in March 2025 and an increase in operating expenses in the first half of 2026.

We expect to continue to incur regular net cash outflows from operations for the foreseeable future, which from time to time may be offset by non-recurring payments received under our license and collaboration arrangements, as we continue the development and advancement of our product candidates and other research programs.

For the six months ended June 30, 2026, our net cash used in operating activities of $138.4 million consisted of a net loss of $67.3 million and unfavorable changes in operating assets and liabilities of $104.8 million, offset by adjustments for non-cash items of $33.7 million.

The changes in operating assets and liabilities include an increase in accounts receivable of $78.5 million, which was driven primarily by the $100.0 million development milestone due from AbbVie as of June 30, 2026 and received in July 2026, and was partially offset by a $19.7 million decrease in royalties due from Novartis.

The unfavorable changes in operating assets and liabilities also include a decrease in accrued expenses and other current liabilities of $15.6 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and sublicense fees, and external research and development services.

Other changes in operating working capital occurred in the normal course of business.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $8.6 million, depreciation and amortization expense of $3.9 million and non-cash interest expense of $3.8 million.

For the three months ended March 31, 2025, our net cash provided by operating activities of $33.6 million consisted of net income of $6.1 million, adjustments for non-cash items of $11.1 million and favorable changes in operating assets and liabilities of $16.4 million.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $8.5 million and depreciation and amortization expense of $4.0 million, partially offset by the accretion of discounts on marketable debt securities during the period.

The changes in operating assets and liabilities include an increase of $39.8 million in deferred revenue, which was driven primarily by the deferred portion of the $110.0 million up-front payment received under our collaboration with Nippon Shinyaku in the first quarter of 2025.

The favorable changes in operating assets and liabilities were partially offset by a decrease in

total accounts payable and accrued expenses and other current liabilities of $18.1 million, which was driven largely by decreases in accrued personnel-related expenses, royalties and external research and development services, as well as a total increase in prepaid expenses and other current assets of $4.3 million, which was driven primarily by increases in prepaid software licenses and net cost reimbursement due from AbbVie under our ABBV-RGX-314 collaboration.

Adjustments for non-cash items primarily consisted of

stock-based compensation expense of $17.7 million, depreciation and amortization expense of $7.6 million and non-cash interest expense of $9.7 million.

For the six months ended June 30, 2025, our net cash used in operating activities of $15.7 million consisted of a net loss of $64.8 million, offset by favorable changes in operating assets and liabilities of $24.0 million and adjustments for non-cash items of $25.1 million.

The changes in operating assets and liabilities include an increase in deferred revenue of $37.7 million, which was driven primarily by the deferred portion of the $110.0 million up-front payment received under our collaboration with Nippon Shinyaku in the first quarter of 2025.

The favorable changes in operating assets and liabilities were partially offset by an increase in prepaid expenses and other current assets of $7.3 million, which was driven primarily by an increase in net cost reimbursement due from AbbVie under our sura-vec collaboration and increases in prepaid clinical trial services and software licenses.

Other changes in operating working capital occurred in the normal course of business.

Adjustments for non-cash items primarily consisted of stock-based compensation expense of $17.2 million and depreciation and amortization expense of $7.9 million.

Cash Flows from Investing Activities

For the three months ended March 31, 2026, our net cash provided by investing activities consisted of $81.7 million in maturities of marketable debt securities, offset by $9.8 million used to purchase marketable debt securities and $1.2 million used to purchase property and equipment.

For the three months ended March 31, 2025, our net cash provided by investing activities consisted of $64.9 million in maturities of marketable debt securities, offset by $19.2 million used to purchase marketable debt securities and $1.0 million used to purchase property and equipment.

For the six months ended June 30, 2026, our net cash provided by investing activities consisted of $148.3 million in sales and maturities of marketable debt securities, offset by $9.8 million used to purchase marketable debt securities and $2.0 million used to purchase property and equipment.

For the six months ended June 30, 2025, our net cash used in investing activities consisted of $230.3 million used to purchase marketable debt securities and $1.4 million used to purchase property and equipment, offset by $136.0 million in maturities of marketable debt securities.

Cash Flows from Financing Activities

For the three months ended March 31, 2026, our net cash used in financing activities primarily consisted of $13.4 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the three months ended March 31, 2025, our net cash used in financing activities primarily consisted of $5.6 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the six months ended June 30, 2026, our net cash provided by financing activities primarily consisted of $19.1 million in proceeds received from sales of common stock under the ATM Program, net of commissions and offering expenses paid during the period, and was partially offset by $15.2 million of royalties paid, net of interest, under our royalty monetization liabilities.

For the six months ended June 30, 2025, our net cash provided by financing activities primarily consisted of $144.5 million in proceeds received from the issuance of the 2025 Royalty Bond and warrants to HCR in May 2025, net of discounts and transaction costs paid during the period, and was partially offset by $10.9 million of royalties paid, net of interest, under our royalty monetization liabilities.

Additional Capital Requirements

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Future Funding Requirements

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.22 billion as of March 31, 2026.

We have incurred cumulative losses since our inception and had an accumulated deficit of $1.19 billion as of June 30, 2026.

Our transition to recurring profitability is dependent upon achieving a level of revenues adequate to support our cost structure, which depends heavily on the successful development, approval and commercialization of our product candidates.

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• the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights, including against Sarepta Therapeutics, Inc., and defending any intellectual property-related claims;

• our current licensing agreements or collaborations remaining in effect, including the AbbVie Collaboration Agreement relating to ABBV-RGX-314 and the Nippon Shinyaku Collaboration Agreement relating to RGX-121 and RGX-111, and our ability to timely achieve any milestones set forth in such agreements or collaborations;

• our current licensing agreements or collaborations remaining in effect, including the AbbVie Collaboration Agreement relating to sura-vec and the Nippon Shinyaku Collaboration Agreement relating to RGX-121 and RGX-111, and our ability to timely achieve any milestones set forth in such agreements or collaborations;

• our ability to establish and maintain additional licensing agreements or collaborations on favorable terms, if at all;

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For information regarding market risk, refer to Item 7A, “Quantitative and Qualitative Disclosures About Market Risk,” included in our Annual Report on Form 10-K for the year ended December 31, 2025.

There have been no material changes to our exposure to market risk during the three months ended March 31, 2026.

There have been no material changes to our exposure to market risk during the six months ended June 30, 2026.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 2. Management’s Discussion and Analysis