REGENXBIO Inc. (RGNX) Q3 2025 Earnings Call Transcript

Access earnings results, analyst expectations, report, slides, earnings call, and transcript.

Overview

The earnings call highlights strong financial performance with a 25% YoY revenue increase and a solid cash position. The strategic partnerships with Nippon Shinyaku and AbbVie, along with optimistic guidance, contribute positively. The Q&A session reveals confidence in regulatory pathways and manufacturing capabilities, despite some uncertainty in pricing and Medicare coverage details. Overall, the company's financial health, strategic partnerships, and optimistic guidance suggest a positive stock price reaction.

Key Financial Performance

Cash, cash equivalents, and marketable securities $302 million as of September 30, 2025, compared to $245 million as of December 31, 2024, representing an increase primarily driven by the $110 million upfront payment from Nippon Shinyaku in Q1 2025 and $145 million in net proceeds from royalty monetization with Healthcare Royalty Partners in Q2 2025, partially offset by cash used for operating activities.

Revenues $30 million for the quarter ended September 30, 2025, compared to $24 million for the quarter ended September 30, 2024, reflecting a 25% year-over-year increase primarily due to development service revenue under the Nippon Shinyaku partnership in Q3 2025.

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Operating Highlights

RGX-202 for Duchenne muscular dystrophy: Enrollment completed in the AFFINITY DUCHENNE pivotal trial. Top-line pivotal data expected in early Q2 2026, with a BLA submission planned for mid-2026. Commercial readiness plans are progressing, with the first batches of RGX-202 produced for commercial supply. Manufacturing capacity is 2,500 doses per year, with a commercial launch anticipated in 2027.

RGX-121 for MPS II: FDA interactions have been productive, and approval is expected by early 2026. Positive 12-month data were delivered to the FDA, and inspections of clinical sites and manufacturing facilities were completed with no observations. Commercial doses are expected to be delivered by early 2026.

Sura-vec for wet AMD and diabetic retinopathy: Enrollment completed in two global Phase III studies for wet AMD, with top-line data expected in Q4 2026. A Phase IIb/III pivotal program for diabetic retinopathy is being initiated. Manufacturing for these programs is conducted in-house.

Expansion of RGX-202 program: Plans to expand the RGX-202 program outside the U.S., starting in Europe.

Manufacturing capabilities: Commercial-ready manufacturing facilities in Rockville are producing high-purity gene therapy products, including RGX-202 and sura-vec. Manufacturing capacity for RGX-202 is 2,500 doses per year.

Financial position: Cash, cash equivalents, and marketable securities totaled $302 million as of September 30, 2025, up from $245 million at the end of 2024. The company expects this cash to fund operations into early 2027, with additional nondilutive financing opportunities available to extend the runway further.

Partnerships: Collaboration with Nippon Shinyaku for RGX-121 and AbbVie for sura-vec programs. These partnerships support development and commercialization efforts.

Focus on large indications: Strategic focus on large commercial opportunities, including Duchenne muscular dystrophy and wet AMD, which represent significant unmet needs.

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Risk or Challenges

Regulatory and Product Development Risks: Forward-looking statements regarding financial outlook, regulatory, and product development plans are subject to risks and uncertainties, which may cause actual results to differ from forecasts. These risks are detailed in the company's filings with the SEC.

Manufacturing and Supply Chain Risks: The company is producing commercial supply batches for RGX-202 and other products. Any disruptions in manufacturing or supply chain could impact the availability of these therapies.

Clinical Trial Risks: Enrollment and execution of pivotal trials for RGX-202, RGX-121, and other programs are critical. Any delays or adverse events could impact timelines and regulatory approvals.

Financial Risks: The company relies on cash reserves and nondilutive financing opportunities to fund operations. Any shortfall in these areas could impact financial stability and operational plans.

Market and Commercialization Risks: The success of RGX-202, RGX-121, and other therapies depends on market acceptance, competitive pressures, and the ability to meet demand. Failure in these areas could impact revenue and strategic goals.

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Guidance & Outlook

RGX-202 for Duchenne Muscular Dystrophy: Enrollment in the AFFINITY DUCHENNE pivotal trial is complete, with top-line pivotal data expected in early Q2 2026. A BLA submission using the accelerated approval pathway is planned for mid-2026. The confirmatory trial is open and continues to enroll patients. Commercial launch preparations are underway, with the first batches of RGX-202 for commercial supply produced. The company anticipates a commercial launch in 2027, targeting the prevalent population.

RGX-121 for MPS II: FDA approval is anticipated by early 2026, with a PDUFA date set for February 8, 2026. The company plans to deliver the first commercial doses of RGX-121 by early 2026. If approved, RGX-121 would be the first and only gene therapy for MPS II, addressing neurodevelopmental decline.

Sura-vec for Wet AMD and Diabetic Retinopathy: Top-line data for the subretinal wet AMD program are expected in Q4 2026. The company is preparing to initiate a Phase IIb/III pivotal program for diabetic retinopathy. The retinal disease franchise benefits from AbbVie's global eye care infrastructure.

Financial Guidance: The company expects its cash balance to fund operations into early 2027, excluding potential nondilutive financing opportunities. These opportunities include the sale of a priority review voucher for RGX-121, development or sales milestones for MPS programs, and milestones from the diabetic retinopathy program under the AbbVie collaboration.

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Shareholder Return Plan

The selected topic was not discussed during the call.

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Key Q&A

Q:When will the next interactions with the FDA for 202 occur, and what is the potential for the accelerated pathway?

A:The next interactions with the FDA for 202 will occur around the vicinity of top-line data in early Q2 2026 and preparing to file the BLA, with a mid-2026 BLA submission planned. The company is optimistic about the accelerated pathway due to significant benefits observed in older patients and a strong safety profile.

Q:What is the cash runway outlook considering nondilutive financing options?

A:If nondilutive financing options, such as the PRV and milestone payments, are factored in, the cash runway could extend well into 2027 or even early 2028, depending on market prices and milestones.

Q:What is the status of enrollment for the confirmatory trial for DMD?

A:Enrollment for the confirmatory trial began at the end of October, and the company expects to be substantially through enrollment by mid-2026. The trial aims to enroll an additional 30 patients, and updates will be provided in the first half of next year.

Q:What are the updates on regulatory interactions for DMD and MPS II?

A:For DMD, the pre-BLA meeting will occur between early Q2 2026 and the BLA filing. For MPS II, the PDUFA date is February 8, and a late-cycle meeting with the FDA is expected. The company has passed facility and clinical site inspections with no observations.

Q:What is the manufacturing capacity at the Rockville site?

A:The Rockville site has a 2,000-liter bioreactor capable of producing up to 2,500 doses of RGX-202 per year. The Hunter program uses less than 5% of the overall capacity, and the site has internal fill/finish capability.

Q:Is the use of FDA natural history as a control in DMD accepted, and how does it support the program?

A:The program uses propensity score matching and other models like CTAP for external natural history matching, which are accepted by the FDA. These methods are prospectively specified in the protocol and support the program's differentiation and efficacy.

Q:What is the company's perspective on recent M&A and licensing activity in gene therapy for wet AMD?

A:The company sees strong interest in gene therapy for wet AMD due to its one-time treatment potential. The partnership with AbbVie validates the program, and retina specialists show significant interest in gene therapy as a pipeline approach.

Q:What are the expectations for a box warning or label restrictions for RGX-202?

A:The company does not expect a box warning for RGX-202 due to its exemplary safety profile, with virtually no liver injury observed, unlike a competitor product with a 40% liver injury rate.

Q:What are the plans for EMA regulatory submissions for RGX-121 and RGX-202?

A:For RGX-121, EMA interactions suggest a preference for a placebo control arm, but named patient sales may be an option. For RGX-202, discussions with EMA have not occurred yet, but differentiation and functional data are expected to support the program.

Q:What is the enrollment status and focus for the suprachoroidal wet AMD program?

A:The suprachoroidal wet AMD program aims to enroll 20 patients at dose level 4. With the subretinal program fully enrolled, enrollment for the suprachoroidal program is expected to accelerate.

Q:What is the pricing strategy and sales team size for RGX-121 and RGX-202?

A:Pricing for RGX-121 will be determined by NS Pharma, with the company receiving meaningful double-digit royalties. Pricing for RGX-202 is undecided, and sales team size has not been specified.

Q:Where is the greatest demand expected for RGX-202 upon commercialization?

A:The greatest demand is expected among patients of all ages, with a focus on the prevalent market of approximately 3,000 eligible patients by 2027. Over time, the focus may shift to the incident market, with early treatment being key.

Q:Review of Unclear Management Responses

A:Management avoided providing specific details on pricing strategy for RGX-202, Medicare coverage timelines for RGX-314, and exact dates for certain regulatory interactions, citing preliminary stages or deferring to partners like AbbVie and NS Pharma.

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Earnings Word Cloud

The most frequently occurring keywords in this quarter's earning call

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REGENXBIO Inc. (RGNX) Q3 2025 Earnings Call Transcript

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The earnings call summary highlights strategic partnerships, promising drug developments, and a solid cash position. The Q&A section reveals proactive strategies for market positioning and safety assurances, with management addressing concerns confidently. Despite some unclear cost details, the overall sentiment is positive due to expected FDA approvals, promising clinical data, and strategic market plans. Adjustments for partnerships and potential milestone payments further boost sentiment.

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The earnings call presents a mixed outlook. Positive factors include strategic partnerships, a strong cash position, and reduced R&D expenses. However, regulatory risks, competitive pressures, and uncertainties in the FDA approval process pose challenges. The Q&A session reveals cautious optimism but also highlights management's avoidance of direct answers on certain regulatory aspects. The stock price is likely to remain stable in the short term, as the market awaits further clarity on FDA approvals and product launches.

RGNX Report

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