REGENXBIO

REGENXBIO, Inc. is a clinical-stage biotechnology company, which engages in the development, commercialization, and licensing of recombinant adeno-associated virus gene therapy. Its product candidates include the NAV Technology Platform, which consists of exclusive rights to novel adeno-associated viral vectors, and therapeutic programs such as RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, and RGX-381. The company was founded by Kenneth T. Mills and James M. Wilson on July 16, 2008 and is headquartered in Rockville, MD.

Earnings data Q1 2023

Earnings data Q2 2023

ROCKVILLE, Md. , March 8, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following upcoming investor conferences: Barclays Global Healthcare Conference Fireside Chat Date: Wednesday, March 15, 2023 Fireside Chat Time: 5:00 p.m.

REGENXBIO to Participate in Upcoming Investor Conferences

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 REGENXBIO Earnings Call.

REGENXBIO Inc. (RGNX) Q4 2022 Earnings Call Transcript

Regenxbio (RGNX) delivered earnings and revenue surprises of 6.76% and 12.21%, respectively, for the quarter ended December 2022. Do the numbers hold clues to what lies ahead for the stock?

Regenxbio (RGNX) Reports Q4 Loss, Lags Revenue Estimates

Continued progress on '5x'25' strategy to advance five AAV Therapeutics from REGENXBIO's internal pipeline and licensed programs into pivotal-stage or commercial products by 2025 RGX-314 program for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie Enrollment ongoing in the pivotal ATMOSPHERE® and ASCENT™ clinical trials using subretinal delivery, expected to support BLA filing in 2024 Recently announced interim data from the Phase II pharmacodynamic study supports use of NAVXpress™ platform process in future commercialization plans Recently announced cohort expansions in AAVIATE® and ALTITUDE® suprachoroidal clinical trials on track to be completed in the first half of 2023; expected interim trial updates in the second half of 2023 AFFINITY DUCHENNE™ Phase I/II trial of RGX-202 is active and recruiting patients; anticipated to report initial trial data in the second half of 2023 CAMPSIITE™ clinical trial of RGX-121 for the treatment of MPS II on track to complete pivotal program enrollment in the first half of 2023; recent updates continue to support plan to file BLA in 2024 using the accelerated approval pathway $565 million in cash, cash equivalents and marketable securities as of December 31, 2022; operational runway into 2025 Conference call Tuesday, February 28 th at 4:30 p.m. ET ROCKVILLE, Md.

REGENXBIO Reports Fourth Quarter 2022 and Full-Year 2022 Financial Results and Recent Operational Highlights

RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programs RGX-111, a potential one-time gene therapy for MPS I, continues to be well-tolerated across two dose levels, with no drug-related serious adverse events New biomarker and neurodevelopmental data continue to indicate encouraging CNS profile in patients dosed with RGX-111 Company is on track to manufacture commercial-scale cGMP material to support the continued development of RGX-111 ROCKVILLE, Md. , Feb. 24, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive interim data from the Phase I/II trial of RGX-111 for the treatment of severe Mucopolysaccharidosis Type I (MPS I).

Additional Positive Interim Data from Phase I/II Trial of REGENXBIO'S RGX-111 for the Treatment of Severe MPS I Presented at WORLDSymposium™

RGX-121, a potential one-time gene therapy for the treatment of MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels Additional data from patients in Cohort 3 using pivotal program dose level continue to demonstrate largest reductions in CSF GAGs, continuing to approach normal levels at 48 weeks New, longer-term clinical measures demonstrated continued improvement in neurodevelopmental and daily activity skill acquisition up to three years after RGX-121 administration Positive interim data continues to support plan to file Biologics License Application in 2024 using the accelerated approval pathway ROCKVILLE, Md. , Feb. 22, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced additional positive interim data from the Phase I/II/III CAMPSIITE™ trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome.

Additional Positive Interim Data from Phase I/II/III CAMPSIITE™ Trial of REGENXBIO's RGX-121 for the Treatment of MPS II (Hunter Syndrome) Presented at 19th Annual WORLDSymposiumTM

Regenxbio has a competitive advantage in the market with its proprietary NAV technology platform, based on AAV vectors, that is highly effective, efficient, and scalable. The recent Phase II trial on RGX-314 and Phase I/II AFFINITY DUCHENNE trial for RGX-202 show promise in their efficacy and safety.

Regenxbio's Vector Technology Will Change Gene Therapy

ROCKVILLE, Md. , Feb. 21, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Tuesday, February 28, 2023, at 4:30 p.m.

REGENXBIO to Host Conference Call on February 28 to Discuss Fourth Quarter and Full Year 2022 Financial Results and Recent Operational Highlights

New interim data from Phase I/I/III trial of RGX-121 for the treatment of MPS II and Phase I/II trial of RGX-111 for the treatment of MPS I to be presented ROCKVILLE, Md. , Feb. 17, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that three oral and eight poster presentations will be presented at the 19th Annual WORLDSymposium™ 2023, taking place in Orlando, Florida from February 22 through 26, 2023.

REGENXBIO Announces Presentations at the 19th Annual WORLDSymposium™ 2023

RGX-314 produced by the NAVXpress platform process has been well-tolerated and demonstrated a similar clinical profile to the initial adherent cell culture process NAVXpress platform process is incorporated in the two ongoing pivotal trials and is expected to be used for future commercialization of RGX-314; the two pivotal trials are expected to support BLA submission in 2024 Company to host live webcast with wet AMD Key Opinion Leaders to discuss new interim Phase II bridging study data, today, Saturday, February 11, at 11:30 a.m. ET ROCKVILLE, Md.

REGENXBIO Presents Interim Data from Phase II Bridging Study Evaluating the Clinical Performance of RGX-314 Using the NAVXpress™ Manufacturing Platform Process

ROCKVILLE, Md. , Feb. 8, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present at the SVB Securities Global Biopharma Conference on Thursday, February 16, 2023.

REGENXBIO Announces Presentation at the Virtual SVB Securities Global Biopharma Conference

ROCKVILLE, Md. , Feb. 6, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that three presentations on RGX-314 will be presented at the Angiogenesis, Exudation, and Degeneration 2023 Conference taking place virtually from February 10-11, 2023.

REGENXBIO Announces Presentations at the Angiogenesis, Exudation, and Degeneration 2023 Conference

-  Company has initiated Phase I/II AFFINITY DUCHENNE™  trial of RGX-202  -  Company also enrolling newly active observational screening study, AFFINITY BEYOND, evaluating AAV8 antibody prevalence in boys with Duchenne  -  Commercial-scale cGMP material from the REGENXBIO Manufacturing Innovation Center to be used in the clinical trial  -  RGX-202 is a potential one-time AAV Therapeutic for the treatment of Duchenne and includes an optimized transgene for a novel microdystrophin and REGENXBIO's proprietary NAV® AAV8 vector ROCKVILLE, Md. , Jan. 23, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202 for the treatment of Duchenne muscular dystrophy (Duchenne) is now active and recruiting patients.

REGENXBIO Announces Phase I/II Trial of RGX-202, a Novel Gene Therapy Candidate for Duchenne Muscular Dystrophy, is Active and Recruiting Patients

Curran Simpson has been appointed Chief Operating Officer Shiva Fritsch assumes the role of Chief Communications Officer ROCKVILLE, Md. , Jan. 9, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the promotion of Curran Simpson from Chief Technology and Operations Officer to Chief Operating Officer.

REGENXBIO ANNOUNCES SENIOR LEADERSHIP PROMOTIONS

ROCKVILLE, Md. , Jan. 3, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present at the 41st Annual J.P.

REGENXBIO Announces Presentation at the 41st Annual J.P. Morgan Healthcare Conference

RGX-181 and RGX-381 are potential one-time AAV Therapeutics for the treatment of the CNS and ocular manifestations of CLN2 disease, the most common form of Batten disease Patient dosed under a single-patient investigator-initiated study of RGX-181 Company announces approval of CTA for RGX-381 from the UK Health Authority and plans to initiate a Phase I/II clinical trial in the first half of 2023 ROCKVILLE, Md. , Dec. 21, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced a comprehensive program update to outline its progress and development plans for RGX-181 and RGX-381, both being developed as potential one-time gene therapies for the treatment of different manifestations in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, the most common form of Batten disease.

REGENXBIO Reports Update on Advancement of Programs for CLN2 Disease

RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programs. Expanded Cohort 2 enrollment is complete; eight patients have received RGX-111 in the trial Company intends to manufacture commercial-scale cGMP material to support the continued development of RGX-111  Company expects additional interim updates from the RGX-111 trial in the first half of 2023 ROCKVILLE, Md.

REGENXBIO Announces Completion of Dosing in the Phase I/II Trial of RGX-111 for the Treatment of Severe MPS I

REGENXBIO is doing important R&D for gene therapies for the eye. The stock is down due to a lack of near-term catalysts and little revenue.

Regenxbio: Great R&D, Lack Of Near-Term Catalysts

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. Conference Call Participants Lisa Walter - RBC Capital Markets Andreas Argyrides - Wedbush Securities Operator Hello and thank you for standing by.

REGENXBIO Inc. (RGNX) Q3 2022 Earnings Call Transcript

Regenxbio (RGNX) delivered earnings and revenue surprises of -12.90% and 10.95%, respectively, for the quarter ended September 2022. Do the numbers hold clues to what lies ahead for the stock?

Regenxbio (RGNX) Reports Q3 Loss, Lags Revenue Estimates

Continued progress on 5x25 strategy to advance five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025 RGX-314 program for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, remains on track for first BLA filing in 2024 Enrollment ongoing in the pivotal ATMOSPHERE® and ASCENT™ clinical trials of RGX-314 for the treatment of wet AMD using subretinal delivery Presented positive interim data from RGX-314 trials for the treatment of wet AMD - the Phase II AAVIATE® trial using suprachoroidal delivery and the Phase I/IIa Long-term Follow-up study using subretinal delivery Announced expansion of AAVIATE trial to include Cohort 6 at third dose level with short-course prophylactic ocular steroids following RGX-314 administration Presented additional positive interim data from Phase II ALTITUDE® trial of RGX-314 for the treatment of diabetic retinopathy using suprachoroidal delivery Announced dose escalation and expansion of ALTITUDE trial to include Cohorts 4 and 5 at new, third dose level with short-course prophylactic ocular steroids following RGX-314 administration AFFINITY DUCHENNE™ Phase I/II trial of RGX-202 remains on track for dosing in the first half of 2023 CAMPSIITE™ pivotal program of RGX-121 is active and enrolling patients for the treatment of MPS II; remains on track to file a BLA in 2024 using the accelerated approval pathway $617 million in cash, cash equivalents and marketable securities of as of September 30, 2022; operational runway into 2025 Conference call Thursday, November 3 rd at 8:30 a.m. ET ROCKVILLE, Md.

REGENXBIO Reports Third Quarter 2022 Financial Results and Recent Operational Highlights

RGX-314 continues to be well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious adverse events Patients treated with RGX-314 in Cohorts 1-3 demonstrated clinically meaningful improvements in disease severity and less disease worsening versus observation control at six months 20% of patients achieved ≥2-step DRSS improvement vs. 10% in control 54% of patients achieved any DRSS improvement vs.

REGENXBIO Presents Positive Interim Data from and the Expansion of Phase II ALTITUDE® Trial of RGX-314 for the Treatment of Diabetic Retinopathy Using Suprachoroidal Delivery

ROCKVILLE, Md. , Oct. 27, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present data from the ALTITUDE® clinical trial evaluating RGX-314 using suprachoroidal delivery for the treatment of patients with diabetic retinopathy at the Retina Society 55th Annual Scientific Meeting taking place in Pasadena, CA, from November 2-5, 2022.

REGENXBIO to Host Conference Call on November 3 to Discuss Third Quarter 2022 Financial Results, Recent Operational Highlights and Interim Data from Phase II ALTITUDE® Trial of RGX-314 for the Treatment of Diabetic Retinopathy

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A company with a market value of about $1 billion is small enough to grow fast, yet large enough so that it can begin to appeal to institutional investors such as pension funds.

What's a Fun-Sized Stock? Around $1 Billion

Company announced positive interim data from and the expansion of Phase II AAVIATE® trial of RGX-314 for the treatment of wet AMD using suprachoroidal delivery   RGX-314 continues to be well tolerated in 85 patients from Cohorts 1-5 with no drug-related serious adverse events Meaningful reduction in treatment burden at six months across all dose levels, 85% reduction in treatment burden observed at third dose level 67% of patients in Cohort 4 were injection-free No meaningful differences in outcomes at six months for patients who are NAb positive Phase II trial expanded to include new cohort at third dose level with short-course prophylactic ocular steroids following RGX-314 administration Positive interim data presented at the AAO Annual Meeting from the Phase I/IIa Long-term Follow-up study of RGX-314 for the treatment of wet AMD using subretinal delivery   RGX-314 continues to be well-tolerated and demonstrates long-term, durable treatment effect up to four years Two pivotal trials, ATMOSPHERE ®  and ASCENT™, are active and enrolling patients Pivotal trials are expected to support BLA submission in 2024 New interim data from Phase II ALTITUDE® trial of RGX-314 for the treatment of diabetic retinopathy using suprachoroidal delivery expected at the Retina Society 55th Annual Scientific Meeting in November Conference call Monday, October 3 at 8:30 a.m. ET ROCKVILLE, Md , Oct. 3, 2022 (PRNewswire) – REGENXBIO Inc. (Nasdaq: RGNX) today announced a program update from its ongoing clinical investigation of RGX-314 for the treatment of wet AMD, a leading cause of vision loss globally.

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