REGENXBIO (Nasdaq: RGNX)Gene Therapy Portfolio, Stock and Financial Highlights

Highlights of REGENXBIO

- REGENXBIO is a leading clinical-stage biotechnology company 

- Utilizes AAV viral vectors from the proprietary gene delivery platform
- Therapeutic areas include retinal, metabolic, and neurodegenerative diseases
- $290 million in cash, cash equivalents and marketable securities as of September 30, 2020

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our gene therapy product candidates are designed to provide long-lasting effects, potentially significantly altering the course of the disease and delivering improved patient outcomes.

Stock Symbol: RGNX

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REGENXBIO Platform

Utilizes AAV viral vectors from the proprietary gene delivery platform, NAV Technology Platform. 

REGENXBIO Therapeutic Area, Portfolio & Programs

The company is currently developing gene therapy product candidates for the treatment of retinal, metabolic, and neurodegenerative diseases. In addition to our internal product candidate programs, NAV Vectors are licensed for developing therapies with other leading biotechnology companies. REGENXBIO's NAV Technology Platform is being applied in one marketed product, Zolgensma marketed by Novartis. 

REGENBIO Program Update Q3 2020

RGX-314 for treatment of Wet Age-related Macular Degeneration (wet AMD)

RGX-314 is being developed as a novel, one-time subretinal treatment that includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment. The expressed protein is designed to neutralize vascular endothelial growth factor (VEGF) activity, modifying the pathway for the formation of new leaky blood vessels and retinal fluid accumulation.

• REGENXBIO expects to initiate the pivotal program for the subretinal delivery of RGX-314 for the treatment of wet AMD in the first quarter of 2021.
• On September 9, 2020, REGENXBIO announced that the first patient had been dosed in the Phase II AAVIATE trial to evaluate the suprachoroidal delivery of RGX-314 using the SCS Microinjector® for the treatment of wet AMD. REGENXBIO expects to complete enrollment of the first cohort by the end of 2020, and report initial safety data from the first cohort in early 2021.
• The Phase II trial, ALTITUDE, to evaluate the targeted, in-office suprachoroidal delivery of RGX-314 in patients with DR is active and REGENXBIO expects to begin enrolling patients by the end of 2020. REGENXBIO plans to report interim data from this trial in 2021.

RGX-121 for treatment of Mucopolysaccharidosis Type II (MPS II), Hunter syndrome
RGX-121 is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS).

• Eight patients have now been dosed across two dose cohorts in the ongoing Phase I/II trial of RGX-121 in severe MPS II patients under the age of 5 years old. The first two patients in the expanded Cohort 2 were dosed in October 2020, via intracisternal delivery of RGX-121 at a dose of 6.5x1010 genome copies per gram (GC/g) of brain mass. REGENXBIO anticipates further updates from this trial by the end of 2020.
• On September 30, 2020, REGENXBIO announced the expansion of the RGX-121 program for MPS II to gain additional insight into the neurodegenerative manifestations of the disease and evaluate RGX-121 in a broader patient population:
• REGENXBIO plans to begin a second Phase I/II multicenter, open-label trial of RGX-121 for the treatment of pediatric patients with severe MPS II over the age of 5 years old. Up to six patients will be enrolled, and RGX-121 will be administered at a dose level of 6.5x1010 GC/g of brain mass.
• REGENXBIO also announced a new prospective observational study designed to provide detailed characterization of neurocognitive development and key biomarkers in patients with severe MPS II.

RGX-111 for treatment of Mucopolysaccharidosis Type I

RGX-111 is designed to use the AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS).

• Recruitment and patient screening are ongoing in REGENXBIO's Phase I/II clinical trial evaluating RGX-111 for the treatment of MPS I.
• REGENXBIO expects to provide a program update by the end of 2020.

RGX-181 for treatment of CLN2 (Batten Disease)

RGX-181 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the central nervous system (CNS).

• REGENXBIO expects to submit an IND for the intracisternal delivery of RGX-181 in the first quarter of 2021, and plans to initiate enrollment in a Phase I/II trial in the first half of 2021.
• Research Program for the Treatment of Neuromuscular Disorders
• REGENXBIO expects to announce a program update in 2021.

RGX-381 for treatment of CLN2 (Batten Disease) Occular Manifestation

RGX-381 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the retina.

• REGENXBIO is on track to submit an Investigational New Drug (IND) application for a Phase I/II study of RGX-381 in patients with CLN2 disease by the end of 2020 and plans to initiate enrollment in the first half of 2021.
• Data from non-human primates demonstrate elevated and sustained levels of TPP1, the enzyme deficient in patients with CLN2 disease, in the vitreous following a single subretinal injection of RGX-381.

Research Program for the Treatment of Hereditary Angioedema (HAE)
REGENXBIO expects to provide a program update in 2021.

REGENXBIO Financial Highlights Q3 2020
Cash Position: Cash, cash equivalents and marketable securities were $289.8 million as of September 30, 2020, compared to $400.0 million as of December 31, 2019. 

Revenues: Revenues were $98.9 million for the three months ended September 30, 2020, compared to $14.7 million for the three months ended September 30, 2019. 

Net Income (Loss): Net income was $8.8 million, or $0.24 basic and $0.23 diluted net income per share, for the three months ended September 30, 2020, compared to net loss of $34.6 million, or $0.94 basic and diluted net loss per share, for the three months ended September 30, 2019.