REGENXBIO(RGNX)

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REGENXBIO(RGNX) - 2023 Q4 - Annual Results
2024-02-27 21:10
Financial Performance - REGENXBIO reported revenues of $22.2 million for Q4 2023 and $90.2 million for the full year, down from $31.3 million and $112.7 million in the same periods of 2022, respectively[10]. - The company had a net loss of $62.9 million, or $1.43 per share, for Q4 2023, compared to a net loss of $59.9 million, or $1.38 per share, in Q4 2022; the full year net loss was $263.5 million, or $6.02 per share, compared to $280.3 million, or $6.50 per share, in 2022[15]. - Total revenues for the year ended December 31, 2023, were $90,242 thousand, down 20% from $112,724 thousand in 2022[24]. - License and royalty revenue decreased from $31,345 thousand in Q4 2022 to $22,213 thousand in Q4 2023, representing a decline of 29%[24]. - Net loss for the year ended December 31, 2023, was $263,494 thousand, compared to a net loss of $280,321 thousand in 2022, showing an improvement of 6%[24]. - Comprehensive loss for the year ended December 31, 2023, was $252,522 thousand, compared to $293,153 thousand in 2022, indicating a reduction of 13.8%[24]. - Basic and diluted net loss per share improved from $(6.50) in 2022 to $(6.02) in 2023[24]. - The company reported a total operating expense of $358,370 thousand for the year ended December 31, 2023, down from $375,600 thousand in 2022, a decrease of 4.6%[24]. Cash and Assets - Cash, cash equivalents, and marketable securities totaled $314.1 million as of December 31, 2023, down from $565.2 million a year earlier, with the current balance expected to fund operations into the second half of 2025[10][16]. - Total assets decreased from $833,268 thousand in December 2022 to $573,970 thousand in December 2023, a decline of approximately 31.2%[23]. - Total liabilities decreased from $317,073 thousand in December 2022 to $262,228 thousand in December 2023, a reduction of approximately 17.3%[23]. - Stockholders' equity fell from $516,195 thousand in December 2022 to $311,742 thousand in December 2023, a decline of about 39.5%[23]. Research and Development - Research and development expenses for Q4 2023 were $55.7 million, down from $62.5 million in Q4 2022; full year R&D expenses were $232.3 million, compared to $242.5 million in 2022[13]. - Research and development expenses for the year ended December 31, 2023, were $232,266 thousand, a decrease of 4.2% compared to $242,453 thousand in 2022[24]. Clinical Trials and Future Plans - ABBV-RGX-314 is being evaluated in nine ongoing clinical trials for wet AMD and diabetic retinopathy, with pivotal trials expected to support regulatory submissions in late 2025 to early 2026[4][6]. - In the AAVIATE trial, patients treated with ABBV-RGX-314 showed an 80% reduction in annualized injection rate, with 50% remaining injection-free at six months[6]. - REGENXBIO plans to file a Biologics License Application (BLA) for RGX-202 in 2024, using microdystrophin expression as a surrogate endpoint[11]. - The company expects to share updates on Duchenne and retinal disease programs starting in March 2024, with pivotal dose determination for RGX-202 anticipated in mid-2024[5][11]. - The company anticipates significant data readouts and milestones across all programs in 2024, marking a potential turning point in its journey[3]. General and Administrative Expenses - General and administrative expenses for Q4 2023 were $19.1 million, compared to $21.2 million in Q4 2022; full year G&A expenses were $88.5 million, up from $85.3 million in 2022[14].
REGENXBIO(RGNX) - 2023 Q3 - Earnings Call Transcript
2023-11-09 03:26
REGENXBIO Inc. (NASDAQ:RGNX) Q3 2023 Earnings Conference Call November 8, 2023 4:30 PM ET Company Participants Patrick Christmas - Chief Legal Officer Ken Mills - Chief Executive Officer Steve Pakola - Chief Medical Officer Vit Vasista - Chief Financial Officer Conference Call Participants Gena Wang - Barclays Dane Leone - Raymond James Alec Stranahan - Bank of America Lisa Walter - RBC Capital Markets Brian Skorney - Baird Mani Foroohar - Leerink Partners Operator Good day, and thank you for standing by. W ...
REGENXBIO(RGNX) - 2023 Q3 - Quarterly Report
2023-11-08 21:15
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 47-1851754 (S ...
REGENXBIO(RGNX) - 2023 Q2 - Earnings Call Transcript
2023-08-03 02:21
REGENXBIO Inc. (NASDAQ:RGNX) Q2 2023 Earnings Conference Call August 2, 2023 4:30 PM ET Company Participants Patrick Christmas - Chief Legal Officer Ken Mills - Chief Executive Officer Steve Pakola - Chief Medical Officer Vit Vasista - Chief Financial Officer Conference Call Participants Dane Leone - RJF Alec Stranahan - Bank of America Ellie Merle - UBS Lisa Walter - RBC Andreas Argyrides - Wedbush Securities Caroline Palomeque - Berenberg Capital Markets Operator Good day, and thank you for standing by. W ...
REGENXBIO(RGNX) - 2023 Q2 - Quarterly Report
2023-08-02 20:15
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Registrant as Specified in its Charter) Washington, D.C. 20549 FORM 10-Q (Mark One) (State or other jurisdictio ...
REGENXBIO(RGNX) - 2023 Q1 - Earnings Call Transcript
2023-05-04 02:47
Financial Data and Key Metrics Changes - REGENXBIO ended Q1 2023 with cash, cash equivalents, and marketable securities totaling $474 million, down from $565 million as of December 31, 2022, primarily due to cash used for operating activities [73] - R&D expenses for Q1 2023 were $59 million, an increase from $56 million in Q1 2022, attributed to higher personnel costs and laboratory expenses related to the manufacturing innovation center [74] Business Line Data and Key Metrics Changes - The company is advancing its 5x'25 strategy, aiming to move five AAV therapeutics into pivotal or commercial stages by 2025 [22][25] - The RGX-314 program has dosed over 600 patients across seven trials, demonstrating good tolerability and potential as a one-time treatment for major retinal vascular diseases [29][30] Market Data and Key Metrics Changes - The global market for diabetic retinopathy (DR) is estimated to reach $15 billion in the next decade, with RGX-314 positioned as a potential one-time treatment option [47] - The anti-VEGF market is calculated to be over $14 billion globally, with RGX-314 targeting over 30 million patients with high unmet needs [66] Company Strategy and Development Direction - REGENXBIO is focused on expanding its global reach through collaboration with AbbVie, particularly in the eye care sector, to bring RGX-314 to patients worldwide [44][62] - The company is leveraging its NAVXpress platform for manufacturing, which is operational and designed to meet global regulatory standards [64] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for RGX-314, anticipating submissions to the FDA and EMA in late 2025 to early 2026 [62] - The company is optimistic about the potential of RGX-314 to address the high unmet need in retinal diseases, particularly given the limitations of current anti-VEGF treatments [63] Other Important Information - The company announced the transfer of INDs for ongoing clinical trials to AbbVie, marking a significant step in their collaboration [23][26] - REGENXBIO's manufacturing innovation center is set to produce commercial-scale GMP material for the RGX-314 clinical program [45] Q&A Session Summary Question: What is the impact of the increased enrollment in ATMOSPHERE and ASCENT on regulatory submissions? - Management indicated that the increased sample size enhances power for both primary and secondary endpoints, which is crucial for regulatory submissions [80][81] Question: Was there an alteration in the agreement with AbbVie regarding regulatory responsibilities? - Management clarified that the transition of regulatory responsibilities was part of the original collaboration agreement and is now being implemented as planned [84][109] Question: How does the decision to expand enrollment impact the registration pathway for suprachoroidal delivery? - Management believes that the expansion will support important milestones for regulatory approvals in both the U.S. and internationally [91][92] Question: What is the expected safety profile for RGX-202 in the Duchenne program? - Management stated that the trial is excluding patients with preexisting AAV8 antibodies to ensure safety and efficacy [95][97] Question: How will the fellow eye study be conducted in relation to the pivotal trials? - The fellow eye study will treat patients at a later time to avoid confounding results, aligning with FDA discussions [120][135]
REGENXBIO(RGNX) - 2023 Q1 - Quarterly Report
2023-05-03 20:16
Financial Performance - Total revenues for the three months ended March 31, 2023, were $19.1 million, down 13.9% from $22.2 million in the same period of 2022[19]. - The net loss for the first quarter of 2023 was $66.7 million, compared to a net loss of $76.7 million for the same period in 2022, reflecting an improvement of approximately 13.4%[19]. - The company reported a comprehensive loss of $62.9 million for Q1 2023, compared to a comprehensive loss of $86.1 million in Q1 2022, showing a reduction of approximately 26.9%[19]. - Total revenues for Q1 2023 were $19.1 million, a decrease of $3.1 million (approximately 14%) from $22.2 million in Q1 2022[126]. - The net loss for Q1 2023 was $66.7 million, an improvement of $10.0 million compared to a net loss of $76.7 million in Q1 2022[126]. Assets and Cash Position - As of March 31, 2023, REGENXBIO Inc. reported total assets of $747.8 million, a decrease from $833.3 million as of December 31, 2022, representing a decline of approximately 10.3%[18]. - Cash, cash equivalents, and marketable securities totaled $473.5 million as of March 31, 2023, which is expected to fund operations for at least the next 12 months[29]. - The total stockholders' equity decreased to $465.1 million as of March 31, 2023, down from $516.2 million at the end of 2022, a decline of approximately 9.9%[18]. - As of March 31, 2023, cash and cash equivalents totaled $70,091,000, down from $191,873,000 as of March 31, 2022, representing a decrease of approximately 63.5%[35]. - Total cash and cash equivalents and restricted cash amounted to $72,121,000 as of March 31, 2023, compared to $193,903,000 in the previous year, indicating a decline of about 62.8%[35]. Research and Development - Research and development expenses increased to $58.5 million in Q1 2023, up from $55.6 million in Q1 2022, indicating a rise of about 3.4%[19]. - Research and development expenses for Q1 2023 totaled $58.5 million, compared to $55.6 million in Q1 2022, reflecting ongoing investment in product development[119]. - Direct expenses for ABBV-RGX-314 in Q1 2023 were $5.0 million, down from $7.5 million in Q1 2022, indicating a reduction in spending[119]. - The Company has developed ABBV-RGX-314, currently in nine ongoing clinical trials for the treatment of wet AMD and diabetic retinopathy, with pivotal trials expected to expand enrollment to 540 and 660 patients respectively[96][97]. - Enrollment in Cohorts 4 and 5 of the ALTITUDE® trial for ABBV-RGX-314 has been completed, with interim data expected in the second half of 2023[102]. Revenue Sources - Zolgensma royalties for Q1 2023 were $16,125,000, down 25.3% from $21,539,000 in Q1 2022[59]. - Total license and royalty revenue for Q1 2023 was $19,138,000, a decrease of 13.4% compared to $22,218,000 in Q1 2022[59]. - License and royalty revenue decreased by $3.1 million, primarily due to a $5.4 million decline in Zolgensma royalty revenues, which fell from $21.5 million in Q1 2022 to $16.1 million in Q1 2023[127]. - Revenue from commercial sales of Zolgensma and royalties paid to HCR are key financial metrics for the company[145]. Liabilities and Expenses - The accumulated deficit reached $508.2 million as of March 31, 2023, increasing from $441.6 million at the end of 2022[29]. - The company had a net cash used in operating activities of $80.9 million for Q1 2023, compared to $55.9 million for Q1 2022, indicating an increase in cash outflow[24]. - The allowance for credit losses increased to $4.3 million as of March 31, 2023, from $4.2 million at the end of 2022, related solely to accounts receivable from Abeona Therapeutics[66]. - Stock-based compensation expense totaled $11,206 thousand for the three months ended March 31, 2023, an increase from $10,800 thousand in 2022[81]. - The Company recorded accrued expenses and other current liabilities of $31,709 thousand as of March 31, 2023, down from $46,794 thousand at December 31, 2022[90]. Collaborations and Agreements - The Company entered into a collaboration agreement with AbbVie, receiving an upfront fee of $370 million and potential milestone payments totaling up to $1.38 billion[75]. - The Company recorded $18.2 million due from AbbVie for net reimbursement of costs incurred under the collaboration agreement as of March 31, 2023[77]. - The collaboration agreement with AbbVie for ABBV-RGX-314 may significantly impact future revenues and operating expenses[114]. Market and Regulatory Considerations - The pivotal trials are expected to support regulatory submissions to the FDA and EMA in late 2025 through the first half of 2026[97]. - RGX-202 received Fast Track designation from the FDA in April 2023 for the treatment of Duchenne muscular dystrophy, aimed at expediting its development[103]. - The CAMPSIITE™ trial for RGX-121 is ongoing, with enrollment of 10 patients expected to complete in the first half of 2023 to support a BLA filing in 2024[104]. - Initial data from the CAMPSIITE™ trial showed RGX-121 was well-tolerated, with significant reductions in CSF glycosaminoglycans observed[105]. Internal Controls and Risk Management - As of March 31, 2023, the company's disclosure controls and procedures were effective at a reasonable assurance level[149]. - There were no changes in internal control over financial reporting that materially affected the company during the quarter ended March 31, 2023[150]. - The company does not believe it is currently party to any pending legal actions that could materially affect its business or financial condition[154]. - There have been no material changes to the company's exposure to market risk during the three months ended March 31, 2023[147].
REGENXBIO(RGNX) - 2022 Q4 - Earnings Call Transcript
2023-03-01 03:36
REGENXBIO Inc. (NASDAQ:RGNX) Q4 2022 Earnings Conference Call February 28, 2023 4:30 PM ET Company Participants Patrick Christmas - Chief Legal Officer Ken Mills - Chief Executive Officer Steve Pakola - Chief Medical Officer Vit Vasista - Chief Financial Officer Conference Call Participants Dane Leone - Raymond James Lisa Walter - RBC Capital Markets Andreas Argyrides - Wedbush Securities Caroline Palomeque - Berenberg Operator Hello, and thank you for standing by. Welcome to the Q4 and Full Year 2022 REGEN ...
REGENXBIO(RGNX) - 2022 Q4 - Annual Report
2023-02-28 21:11
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 or (I.R.S. Employer Identification Number) 9804 Medical Center Drive Rockville, MD 20850 (240) 552-8181 (Address of principal executive offices and Zip Code, and telephone number, including area code) ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ...
REGENXBIO(RGNX) - 2022 Q3 - Earnings Call Transcript
2022-11-03 19:23
REGENXBIO Inc. (NASDAQ:RGNX) Q3 2022 Earnings Conference Call November 3, 2022 8:30 AM ET Company Participants Patrick Christmas - Chief Legal Officer Vit Vasista - Chief Financial Officer Ken Mills - President & Chief Executive Officer Steve Pakola - Chief Medical Officer Lejla Vajzovic - Associate Professor, Ophthalmology; and Director, Duke Vitreoretinal Fellowship Program and Lead Investigator Peter Kaiser - Director, Center for Ocular Research and Evaluation, Cole Eye Institute, Cleveland Clinic. Confe ...