REGENXBIO Inc. (RGNX) Earnings

Management highlights

### Core Pipeline Milestones - **RGX202 (Duchenne Muscular Dystrophy)**: Fully enrolled and completed dosing in the confirmatory study ahead of schedule. Over 60 total patients have been enrolled across pivotal and confirmatory studies to build a robust safety dataset for BLA submission. Top-line pivotal data from May demonstrated robust microdystrophin expression, encouraging functional improvement (including in older patients), a favorable safety profile, and a statistically significant strong correlation between microdystrophin expression and functional improvement. The ex-U.S. randomized placebo-controlled Affinity Rise trial is planned to initiate enrollment in H1 2027. - **RGX121 (MPS II / Hunter Syndrome)**: Aligned with the FDA on a path for BLA resubmission following a Type A meeting in July 2026. The FDA confirmed existing data is sufficient for accelerated approval review, and no additional clinical studies (including a new RCT) are required for resubmission. Resubmission will include updated longer-term efficacy, safety, and imaging data, and is planned for Q3 2026. - **Retinal Gene Therapy Franchise (Collaboration with AbbVie)**: Dosed the first patient in the Phase 2b-3 NAVIGATE trial of SuraVec for diabetic retinopathy, triggering a $100 million milestone payment from AbbVie. Presented 5-year long-term data for Cerevec in wet AMD (demonstrating maintained/improved visual acuity and reduced treatment burden) and 2.5-year data in diabetic retinopathy (demonstrating durable disease severity improvement and sustained prevention of vision-threatening complications after a single dose). Top-line data from the Atmosphere and Ascent pivotal trials for Cerevec in subretinal wet AMD is expected in Q4 2026. ### Financial Position Update - Strengthened cash position to over $310 million pro forma, extending operating runway through Q4 2027, covering key upcoming milestones including the RGX202 PDUFA date and wet AMD pivotal data readout. Additional non-dilutive funding sources (future partnership milestones, potential sale of the RGX121 PRV, healthcare royalty agreements) are available to extend runway further if needed.

Guidance

- BLA submission for RGX202: First module submission to the FDA planned for Q3 2026, with potential US approval in H2 2027; initiation of the ex-U.S. Affinity Rise RCT planned for H1 2027. - RGX121 BLA resubmission planned for Q3 2026. - Top-line data readout for the Cerevec wet AMD pivotal trials (Atmosphere and Ascent) expected in Q4 2026. - Pro forma cash position of >$310 million extends operating runway into Q4 2027, covering all key upcoming milestones. Guidance does not include potential additional proceeds from future non-dilutive funding sources. - The company maintains its strategic focus on executing key 2026 milestones and advancing transformative gene therapies toward patient access, with the next 18 months expected to be a defining period for the firm.

Segment performance

Regenexx BIO is a clinical-stage biotech company focused on gene therapy development, and has not yet generated any product revenue as of Q2 2026. As of quarter end June 30 2026, the company held $106 million in cash, cash equivalents, and marketable securities. Subsequent to quarter end, the company received a $100 million milestone payment from AbbVie and completed a follow-on public offering generating $108 million in net proceeds, bringing pro forma total cash to over $310 million. Research and development and general administrative expenses were consistent with Q2 2025, aligned with advancing late-stage clinical programs and building capabilities for the planned transition to commercial operations.

Risks & headwinds

- Forward-looking statements (including clinical trial timelines, regulatory outcomes, commercial launch prospects, and cash runway projections) are subject to inherent risks and uncertainties that could cause actual results to differ materially from forecasts. Key risks are detailed in the company's SEC filings (Form 10-K 2025 and subsequent Form 10-Qs). - Clinical development and regulatory approval carry inherent uncertainty: trial enrollment timelines, primary endpoint outcomes, and FDA regulatory decisions may differ from current expectations. - Commercial success of pipeline products depends on unproven market adoption relative to existing and emerging competing therapies, and (for partnered programs) on partner execution of commercialization plans. - The company's cash runway is dependent on the timing and receipt of expected future milestone payments and other potential funding, which may not materialize as expected.

Analyst Q&A

• Q: The confirmatory study for RGX202 enrolled ahead of schedule, what does this indicate about patient demand, and what feedback did you receive on Cerevec at the ASRS conference, particularly around delivery modality and unmet need vs other gene therapy approaches? /

  A: The fast enrollment of the 30-patient confirmatory study reflects strong patient and investigator enthusiasm for RGX202, driven by its differentiated efficacy and safety profile, which the company expects will translate to strong enrollment for the global ex-U.S. Affinity Rise trial. At ASRS, the key takeaway from the clinical community was strong excitement around the demonstrated long-term durability of Cerevec's efficacy and safety. The unmet need for a one-time treatment is particularly clear for diabetic retinopathy, where chronic repeated injections have very low real-world adoption due to treatment burden.

• Q: What is the intellectual property position of your existing royalty streams, and how will patent expiry impact future revenue? /

  A: The U.S. patent for Zolgensma has expired, but the company retains patent coverage and will continue to earn royalties in approximately 20 non-U.S. markets. The company also maintains global and U.S. patent coverage for Avexis, and expects continued significant royalties from that product as Novartis projects sales could reach up to $2 billion.

• Q: Will the FDA require data from the upcoming ex-U.S. Affinity Rise RCT for RGX202 accelerated approval, and how many surgeons are trained for Cerevec delivery, and what is the level of interest in bilateral treatment? /

  A: The Affinity Rise RCT will be active during the FDA review period, but the company does not expect data from this study to be required for the accelerated approval decision. The BLA submission will be supported by a robust dataset from over 60 already dosed patients across the U.S. pivotal and confirmatory studies, with roughly half of patients having completed 12-month functional assessments at filing. Over 500 surgeons globally have already been trained in the Cerevec subretinal delivery procedure, which is scalable because the procedure is within the existing skill set of retinal specialists. While enrollment criteria for bilateral treatment excludes some patients, the company sees strong anecdotal interest from patients for fellow eye treatment.

• Q: Will the Affinity Rise trial include a placebo crossover option for DMD patients, and will it enroll U.S. patients? What question is the RGX121 BLA resubmission addressing for the FDA? /

  A: The company expects to offer a crossover option for placebo patients in Affinity Rise, consistent with ethical standards for a progressive disease like DMD. For RGX121, the original BLA included 6-month biomarker data; the resubmission consolidates existing two-year biomarker, neurocognitive, and safety data that has already been collected. The FDA did not require new patient dosing or additional data beyond the existing two-year horizon, and has removed the prior requirement to add a new control arm to the trial.