Regenxbio's Earnings Call Contradictions: BLA Timing, DMD Data Delays, and Study Design Secrecy Clash

Date of Call: Aug 6, 2026

Guidance:

• Cash runway extends into Q4 2027, supporting milestones including PDUFA date for RGX202.
• Initiate AffinityRISE ex-U.S. RCT study in H1 2027.
• Submit first module of RGX202 BLA to FDA in Q3 2026.
• Potential U.S. approval for RGX202 in H2 2027.
• Resubmit RGX121 BLA in Q3 2026.
• Top-line data for Cerevec (wet AMD) and SuraVac (diabetic retinopathy) expected in Q4 2026.

Business Commentary:

Positive Momentum in Gene Therapy Pipeline:

• Regenexx Bio achieved key milestones in its late-stage gene therapy pipeline, including completing enrollment in the confirmatory study for RGX202 ahead of schedule and aligning with the FDA on the path to resubmit the BLA for RGX121.
• The momentum was driven by the strong differentiation of RGX202, demonstrated by robust microdystrophin expression and functional improvement, which supports potential accelerated approval.

Financial Position and Runway Extension:

• The company strengthened its financial position by receiving over $200 million, inclusive of a $100 million milestone payment from AbbVie and new capital, ending the quarter pro forma with more than $310 million.
• This financial boost extends their cash runway into Q4 2027, enabling continued investment in strategic priorities and commercial readiness for potential product launches.

Progress in Retinal Franchise:

• Regenexx Bio made meaningful progress in its retinal franchise with the strategic collaboration with AbbVie, including dosing the first patient in the Phase 2B-3 Navigate study for diabetic retinopathy.
• The long-term data presented at ASRS highlighted the durable safety and efficacy profile of Cirovec, reinforcing confidence in its potential commercial success.

Regulatory Alignment and BLA Resubmission for RGX121:

• Following collaborative discussions with the FDA, Regenexx Bio aligned on a path forward for RGX121, with the agency confirming that available data is sufficient for review under the accelerated approval pathway.
• The resubmission will include longer-term efficacy and safety data, with plans to resubmit the BLA in Q3 2026, focusing on the benefit-risk evaluation.

Sentiment Analysis:

Overall Tone: Positive

• The Q2 was described as 'another period of positive momentum' with 'continued momentum' across programs. Management expressed being 'incredibly excited' and 'well positioned' with 'confidence in our strategy, our execution, and our financial position.' The next 18 months are viewed as 'a defining period.'

Q&A:

• Question from Judah Frommer (MS): Could you give more color on enrollment for the Affinity confirmatory study? Also, feedback on gene therapy excitement at ASRS, unmet need for AMD vs. DR, and delivery method comparisons?
  Response: Enrollment for the U.S. confirmatory study was completed ahead of schedule. At ASRS, the key message was durability of Cerevec in both wet AMD and DR, driving interest in one-time gene therapy options, especially for DR where repeated injections are burdensome.

• Question from Lily and Sun Goh (Learing): How should we think about patent expiry impact on Zolgensma royalties and potential revenue/royalty rate for Avisma?
  Response: U.S. Zolgensma patent has expired, but coverage remains in ~20 countries outside the U.S. Avisma has U.S. and worldwide patent coverage. Novartis reported up to $2B in sales, with significant royalties expected.

• Question from Annabel Samimi (Stifel): How many physicians have been trained on subretinal procedure for wet AMD? How many patients opted for bilateral treatment? On DMD, will FDA require RCT data for accelerated approval?
  Response: Over 500 surgeons have been trained globally for subretinal administration. Bilateral treatment interest is high but enrollment criteria limit numbers. For DMD, the RCT is for global submissions; FDA accelerated approval is not expected to require RCT data, given extensive safety and functional data from pivotal/confirmatory studies.

• Question from Brian Scorny (Baird): Can you provide more details on AffinityRISE study design, including duration, endpoints, enrollment criteria, and regions?
  Response: AffinityRISE is a global, double-masked, placebo-controlled study with 2:1 active-to-placebo randomization. Specifics on regions, endpoints, and enrollment criteria will be disclosed closer to initiation in H1 2027.

• Question from Alex (Bank of America): What specific FDA questions is the longer-term follow-up for MPS II answering? How should we think about crossover from placebo in AffinityRISE?
  Response: FDA requested consolidation of longer-term biomarker and safety data for MPS II, not additional patient data. Crossover from placebo in AffinityRISE is expected for patients who commit to the trial, though not all will qualify.

• Question from Luca Easy (RBC Capital Markets): Can you quantify how many patients will have 12-month functional data at BLA initiation vs. completion? Does FDA require a pre-specified minimum for accelerated approval?
  Response: At BLA submission (~Q3 2026), roughly half of the 30 pivotal patients (~14-15) will have 12-month functional data, with more potentially added later. FDA does not require a pre-specified level of functional data for submission.

• Question from Ellie Merle (Merkleys): What is the latest commercial outlook for RGX314 in subretinal wet AMD? Can you characterize site qualification efforts?
  Response: Commercial launch for subretinal wet AMD will be led by AbbVie. Many clinical sites (~100+) are potential candidates for commercialization. Launch preparation is just beginning, but AbbVie's experience provides a strong running start. The wet AMD market is a major opportunity.

• Question from Sean McCutcheon (Raymond James): For wet AMD readouts in Q4, can you speak to the expected non-inferiority margin and study powering?
  Response: The non-inferiority margin of 4.5 letters is standard and has not been challenged by FDA. The studies have very large sample sizes (90% power), the largest ever for gene therapy.

• Question from Paul Choi (Goldman Sachs): What is the cadence of additional data updates for RGX202 leading to BLA? How is the market primed for new MPS II therapies post-Denali's Avaya launch?
  Response: No specific data updates are planned until BLA filing in Q1 2027. For MPS II, patient advocacy groups show high interest in new therapies due to the long gap since the last approval, indicating strong market receptivity for gene therapy options.

Contradiction Point 1

AffinityRISE Study Design Disclosure

Conflicting statements on whether detailed study specifics have been disclosed.

Brian Scorny (Baird) - Brian Scorny (Baird)

2026Q2: Specific details on primary endpoints, secondary endpoints, enrollment criteria (age), and exact regions have not been disclosed but will be shared as the trial gets closer to initiation. - [Curran Simpson](CEO)

Can you provide details on the AffinityRISE study design, including its placebo-controlled period, primary/secondary endpoints, age enrollment criteria, and target ex-U.S. regions? - Brian Skorney (Baird)

2026Q2: The company has not disclosed detailed specifics on the study design beyond what was announced... Further details, including regions and specific endpoints, will be shared as the trial initiation approaches... - [Steve Pakola](CMO)

Contradiction Point 2

Commercial Site Qualification for RGX314 (wet AMD)

Contradiction on which sites are prioritized for initial commercial qualification.

Ellie Merle (Merkleys) - Ellie Merle (Merkleys)

2026Q2: Many clinical trial sites (over 100 in the U.S.) are likely candidates for commercial site qualification. - [Curran Simpson](CEO)

What is your latest perspective on the commercial outlook for RGX314 in subretinal wet AMD, and how are site qualification efforts progressing, including which commercial sites you plan to qualify initially? - Ellie Merle / Joseph (Barclays)

2026Q2: Commercial site selection will likely include many of the over 100 clinical sites used in the U.S. trials. - [Curran Simpson](CEO)

Contradiction Point 3

Availability of 12-Month Functional Data for DMD BLA Submission

Contradiction on when 12-month functional data for the pivotal cohort will be available.

Luca Easy (RBC Capital Markets) - Luca Easy (RBC Capital Markets)

2026Q2: At the time of clinical module submission... roughly half of the 30 pivotal patients (about 14-15) will have 12-month functional data. - [Dr. Steve Bacola](CMO)

For DMD BLA, how many patients will have 12-month functional data at BLA initiation (Q3 2026) versus completion (Q1 2027), and does the FDA require a pre-specified minimum for accelerated approval? - Judah Frommer (Morgan Stanley)

20260305-2025 Q4: 12-month functional data on the full pivotal cohort will be available in early 2026, with longer-term data to follow. - [Curran Simpson](CEO)

Contradiction Point 4

Regulatory Pathway Expectations for RGX202

Contradiction on whether the RGX202 BLA submission is expected to be filed in 2026.

Paul Choi (Goldman Sachs), what are your key takeaways from the earnings call? - Paul Choi (Goldman Sachs)

2026Q2: No specific additional data updates are planned until after BLA filing in Q1 2027. - [Dr. Steve Bacola](CMO)

"For RGX202, what is the cadence of additional data updates leading to BLA filing? For RGX121, given the recent launch of Denali's Avexza, what is the field's receptivity to new therapies for Hunter syndrome?" - Eliana Merle (Barclays)

20260305-2025 Q4: The BLA could be filed in 2026 post-meeting. - [Curran Simpson](CEO)

Contradiction Point 5

DMD Confirmatory Trial Enrollment Timeline

Contradiction in projected timeline for completing patient enrollment for the Duchenne muscular dystrophy (DMD) confirmatory study.

Annabel Samimi (Stifel) - Annabel Samimi (Stifel)

2026Q2: For DMD, the ex-U.S. RCT (AffinityRISE) is planned to initiate enrollment during the BLA review process, but data from this study is not expected to be required or influentially considered during the accelerated approval review. - [Dr. Steve Bacola](CMO), [Curran Simpson](CEO)

For wet AMD, how many physicians have been trained on your procedures, and how many patients have opted for bilateral treatment? On DMD, will the FDA require data from the ex-U.S. RCT (AffinityRISE) for decisions during BLA review? - Lili Nsongo (Leerink Partners LLC)

2025Q3: Enrollment for the confirmatory trial... expects to be substantially through enrollment by mid-2026, just in time for the BLA filing. - [Curran Simpson](CEO), [Steve Pakola](CMO)