Regenxbio (NASDAQ: RGNX) reported Q4 EPS of ($0.72), $0.28 worse than the analyst estimate of ($0.44). Revenue for the quarter came in at $11.77 million versus the consensus estimate of $39.67 million.

• Additional data from RGX-314 Phase I/IIa trial for wet AMD expected in 1H 2020
• RGX-314 clinical trials for treatment of wet AMD and diabetic retinopathy expected to advance in 2020 using subretinal and suprachoroidal delivery approaches
• Additional interim data from RGX-121 Phase I/II trial for MPS II expected in 2020
• Plans to expand rare disease pipeline into neuromuscular disorders in 2H 2020
• $400 million in cash, cash equivalents and marketable securities as of December 31, 2019

"In 2020, we are broadening our internal gene therapy pipeline using our proprietary NAV Technology Platform, and advancing key programs including RGX-314 for the treatment of retinal diseases," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "As we drive the RGX-314 program forward with new trials in wet AMD and diabetic retinopathy planned for later this year, we are also advancing our RGX-121 program in MPS II based on encouraging data from the first cohort of patients. We are pleased with the advancement of the RGX-111 and RGX-501 programs, and look forward to submitting an IND for RGX-181 and providing updates for our research programs in HAE and neurodegenerative diseases later this year."

Mr. Mills added: "Based on our in-house research and development team's work, we also plan to expand our rare disease platform to include a potential treatment for a neuromuscular disorder using NAV AAV8, which we believe has the potential to treat patients who lack treatments or who are currently underserved by existing therapies. I look forward to providing further updates on this work in the second half of the year."

For earnings history and earnings-related data on Regenxbio (RGNX) click here.