REGENXBIO(RGNX) - 2024 Q3 - Quarterly Results

Program Highlights and Milestones REGENXBIO made significant progress in its gene therapy pipeline, advancing programs for Duchenne, retinal diseases, and MPS II - Advancement in Phase I/II AFFINITY DUCHENNE® trial of RGX-202, with pivotal trial initiation and first functional data expected in November 2024[1][3] - Initiated a rolling Biologics License Application (BLA) submission for RGX-121 for MPS II, with completion expected in Q1 2025[1][7] - The End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy has been accelerated to Q4 2024, aiming for a global pivotal program initiation in H1 2025[1][4] - Positive Phase II data for subretinal ABBV-RGX-314 showed a 97% reduction in anti-VEGF treatment burden at nine months in the fellow eye of wet AMD patients, supporting its potential for bilateral treatment[1][5] Neuromuscular Disease: RGX-202 (Duchenne) The RGX-202 program for Duchenne is rapidly advancing, with dosing complete for the pivotal cohort and initiated for younger patients - The last patient in the dose level 2 (pivotal dose) expansion cohort for ages 4-11 has been dosed[3] - The first patient has been dosed in the cohort for ages 1-3. REGENXBIO is the only gene therapy sponsor recruiting U.S. patients in this age group[3] - A Clinical Trials Application (CTA) for RGX-202 has been authorized by Health Canada, with site initiation expected in H1 2025[3] Retinal Disease: ABBV-RGX-314 (Wet AMD & DR) The ABBV-RGX-314 program for retinal diseases is progressing, with accelerated FDA meetings and positive data supporting bilateral treatment - For Diabetic Retinopathy (DR), an End-of-Phase II meeting with the FDA is accelerated to Q4 2024, with a global pivotal trial expected to start in H1 2025[4] - For wet AMD (subretinal delivery), pivotal trials (ATMOSPHERE® and ASCENT™) are on track to support global regulatory submissions in H1 2026[5] - For wet AMD (suprachoroidal delivery), dose level 3 demonstrated an 80% reduction in annualized injection rate at six months. A new cohort is now evaluating a higher dose level[6] Neurodegenerative Disease: RGX-121 (MPS II) REGENXBIO initiated a rolling BLA submission for RGX-121 for MPS II, with completion expected in Q1 2025 - A rolling BLA submission for RGX-121 was initiated in Q3 2024 and is expected to be complete in Q1 2025[7] - Approval of RGX-121 could result in the receipt of a Priority Review Voucher in 2025[7] Financial Results REGENXBIO reported Q3 2024 revenues of $24.2 million, a narrowed net loss, and a strong cash position of $278.6 million - Cash, cash equivalents, and marketable securities were $278.6 million as of September 30, 2024, down from $314.1 million at year-end 2023. The decrease was offset by $131.1 million in net proceeds from a public offering in March 2024[8] Q3 2024 Financial Performance vs. Q3 2023 (in millions, except per share data) | Metric | Q3 2024 | Q3 2023 | Change | | :--- | :--- | :--- | :--- | | Revenues | $24.2 | $28.9 | -16.3% | | R&D Expenses | $54.4 | $58.2 | -6.5% | | G&A Expenses | $19.4 | $23.1 | -16.0% | | Net Loss | ($59.6) | ($61.9) | Improved | | Net Loss Per Share | ($1.17) | ($1.41) | Improved | Financial Guidance REGENXBIO expects its $278.6 million cash balance as of September 30, 2024, to fund operations into 2026 - The company expects its cash balance of $278.6 million (as of Sept 30, 2024) to fund operations into 2026[13] - This cash runway guidance is based on current operational plans and excludes potential milestone payments from partners or licensees[13] Consolidated Financial Statements This section presents REGENXBIO Inc.'s unaudited consolidated financial statements, including balance sheets and statements of operations Consolidated Balance Sheets The Consolidated Balance Sheet details total assets of $519.1 million and total liabilities of $217.7 million as of September 30, 2024 Key Balance Sheet Items (in thousands) | Account | September 30, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Cash and cash equivalents | $56,617 | $34,522 | | Total current assets | $313,396 | $334,971 | | Total assets | $519,114 | $573,970 | | Total current liabilities | $102,725 | $130,272 | | Total liabilities | $217,698 | $262,228 | Consolidated Statements of Operations and Comprehensive Loss The Consolidated Statements of Operations show total revenues of $24.2 million and a net loss of $59.6 million for Q3 2024 Statement of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2024 | Three Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2024 | Nine Months Ended Sep 30, 2023 | | :--- | :--- | :--- | :--- | :--- | | Total Revenues | $24,197 | $28,914 | $62,114 | $68,029 | | Loss from operations | $(62,078) | $(64,960) | $(181,978) | $(204,225) | | Net loss | $(59,597) | $(61,868) | $(175,916) | $(200,605) | | Net loss per share | $(1.17) | $(1.41) | $(3.59) | $(4.60) |