Regenxbio (NASDAQ: RGNX) reported Q3 EPS of ($0.94), $0.22 worse than the analyst estimate of ($0.72). Revenue for the quarter came in at $14.7 million versus the consensus estimate of $9.74 million.

• Initiation of RGX-314 Phase IIb trial for wet AMD and IND filing for Phase II trial in diabetic retinopathy now expected in Q1 2020 as a result of partial clinical hold following FDA notification related to certain commercial, third-party surgical devices; matter is not related to RGX-314 gene therapy candidate
• Positive interim update from RGX-314 Phase I/IIa trial for wet AMD presented in October at AAO
  • Well-tolerated at all doses
  • Anti-VEGF treatment burden was decreased by over 80% in Cohort 5, with 75% of subjects anti-VEGF injection-free up to 6 months after RGX-314 administration
  • 50% of subjects in Cohort 3 were anti-VEGF injection-free at 1.5 years after RGX-314 administration
• Completed dosing of Cohort 2 in the RGX-501 Phase I/II trial for HoFH and Cohort 1 in the RGX-121 Phase I/II trial for MPS II; interim updates expected at year end
• Revenue includes $9.2 million in royalties from the Q3 2019 sales of Novartis' Zolgensma®
• $417 million in cash, cash equivalents and marketable securities as of September 30, 2019; expect to end 2019 with cash, cash equivalents and marketable securities of at least $365 million
• Webcast and conference call scheduled for today at 4:30 p.m. ET

"We continue to make significant advances in our pipeline and NAV Technology Platform, expanding the pipeline with programs to deliver therapeutic antibodies, beginning an exciting collaboration with Neurimmune, and initiating construction of a new manufacturing facility," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Our focus on patients drives us to build upon the clinical progress of gene therapy options for both large indications and rare diseases, and we are excited to lead the field in innovative approaches. We are also very encouraged by the early adoption of the first approved NAV Technology-based therapy, Novartis' Zolgensma®, for the treatment of SMA."

"We anticipate a short delay in the initiation of our Phase IIb trial for RGX-314 in wet AMD and our investigative new drug, or IND, application filing for diabetic retinopathy, following notification from the FDA regarding certain third-party commercially-available surgical devices that were used to deliver RGX-314 in our Phase I/IIa trial," added Mr. Mills. "The notification is not related to the RGX-314 gene therapy itself, and we have not received reports of any device-related concerns or complications in the subjects already dosed in the Phase I/IIa trial. Assessments and monitoring of all enrolled subjects in the trial continue to be performed as usual. We are working with the FDA to address this matter, and as a result, we now plan to initiate our Phase IIb trial for RGX-314 in wet AMD and file our IND for diabetic retinopathy in Q1 2020,"

"We are encouraged by the data from the Phase I/IIa clinical trial of RGX-314 in patients with wet AMD," said Dr. Stephen Pakola, Senior Vice President and Chief Medical Officer of REGENXBIO. "These data demonstrate significant long-term reduction of anti-VEGF treatment burden while maintaining or improving vision and retinal thickness after a single administration of our gene therapy. We look forward to initiating our Phase IIb trial of RGX-314 in wet AMD, and preparing to file an IND to evaluate RGX-314 in diabetic retinopathy, while continuing our collaboration with Clearside to evaluate the in-office suprachoroidal administration of RGX-314. We have also achieved important clinical development milestones in our other gene therapy programs, including completing dosing of Cohort 2 in our RGX-501 trial for HoFH and Cohort 1 in our RGX-121 trial for MPS II. Our internal pipeline of novel gene therapies positions us to develop meaningful potential treatments for a broad spectrum of patients with significant unmet needs."

Financial Guidance

Based on its current operating plan, REGENXBIO now expects its balance in cash, cash equivalents and marketable securities to be at least $365 million as of December 31, 2019.

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