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1. Company Snapshot

1.a. Company Description

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease.Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform.The company's lead product candidate is RGX-314, which is in Phase III clinical trial for the treatment of wet age-related macular degeneration.


It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II;RGX-111, which is in Phase I/II clinical trial for treating mucopolysaccharidosis type I;RGX-181 which is in pre clinic stage for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease;RGX-202, to treat Duchenne muscular dystrophy which is in phase I/II clinical trial; and RGX-381, to treat the ocular manifestations of CLN2 disease which is in preclinical stage.REGENXBIO Inc.also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop novel gene therapies.


REGENXBIO Inc.was founded in 2008 and is headquartered in Rockville, Maryland.

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1.b. Last Insights on RGNX

Negative drivers behind REGENXBIO Inc.'s recent 3-month performance include: * Weaker-than-expected Q4 2024 earnings, with a loss of $1.01 per share versus the Zacks Consensus Estimate of a loss of $1.27. * Goldman Sachs' downgrade of the company, citing uncertain appeal of gene therapy for wet AMD and diabetic retinopathy due to newer anti-VEGF treatments offering longer-lasting effects at lower costs. * Negative sentiment from investor conferences, including a downgrade by Goldman Sachs and a warning about rival treatments gaining ground. * Uncertainty surrounding the company's gene therapy pipeline, with some investors expressing concerns about the competitive landscape. * A lack of clear catalysts for the stock, with some analysts warning that the company's valuation may be stretched.

1.c. Company Highlights

2. Gene Therapy Progress and Financial Strength

The company's financial performance is characterized by a strong cash position, with $302 million in cash, cash equivalents, and marketable securities as of September 30, 2025, expected to fund operations into early 2027. The actual EPS came out at '-1.2', beating estimates at '-1.38'. Analysts estimate next year's revenue growth at 35.0%. The company's financials are underpinned by its nondilutive financing opportunities, including the potential sale of a priority review voucher for RGX-121.

Publication Date: Nov -18

📋 Highlights
  • Strong Financial Position:: $302 million in cash, cash equivalents, and marketable securities as of September 30, 2025, funding operations into early 2027.
  • Phase III Enrollment Completion:: Over 1,200 patients enrolled across 200 sites in global Phase III studies for wet AMD and diabetic retinopathy.
  • RGX-121 FDA Approval Outlook:: Positive 12-month data submitted; confident in early 2026 approval for MPS II, with potential for 2,500 annual doses at Rockville facility.
  • Manufacturing Scalability:: 2,000-liter bioreactor at Rockville site enables 2,500 RGX-202 doses/year, with 5% capacity allocated to RGX-121 (MPS II).

Pipeline Progress and Upcoming Milestones

The company is making significant progress in its gene therapy pipeline, with RGX-202 completing enrollment in the AFFINITY DUCHENNE pivotal trial. The company expects to share top-line data in early Q2 2026 and file a BLA in mid-2026. Additionally, RGX-121 has delivered positive 12-month data to the FDA, with approval expected by early next year. As Curran Simpson mentioned, the company is seeing good differentiation in functional benefit for patients, particularly in older boys, with results never seen before in any program.

Regulatory Updates and Manufacturing Capacity

The company is preparing for regulatory submissions, with a late-cycle meeting with the FDA expected for the Hunter program between now and the February 8 PDUFA date. The company has also demonstrated its manufacturing capabilities, with the Rockville site having a 2,000-liter bioreactor, enabling the production of up to 2,500 doses of RGX-202 per year. The Hunter program uses less than 5% of the company's overall capacity.

Valuation and Growth Prospects

With a P/S Ratio of 3.5 and an estimated revenue growth rate of 35.0% next year, the company's valuation appears to be reasonable. The P/E Ratio is -3.24, and the EV/EBITDA is -4.56, indicating significant losses. However, the company's strong pipeline progress and financial position may justify its current valuation. The ROE is -78.29%, and the ROIC is -37.81%, reflecting the company's investment in its pipeline.

Partnerships and Commercialization

The company has a major global partnership with AbbVie for its wet AMD program, with AbbVie experienced in ophthalmology. The partnership is paying $2 of every $3 for advancements in diabetic retinopathy and other areas. The company is also expecting broad access for gene therapy, with Medicare coverage likely achievable.

3. NewsRoom

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REGENXBIO to Participate in Upcoming Investor Conference

Nov -25

Source: prnewswire.com

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REGENXBIO Inc. (RGNX) Q3 2025 Earnings Call Transcript

Nov -06

Source: seekingalpha.com

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Regenxbio (RGNX) Reports Q3 Loss, Beats Revenue Estimates

Nov -06

Source: zacks.com

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REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights

Nov -06

Source: prnewswire.com

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Y Intercept Hong Kong Ltd Invests $1.45 Million in REGENXBIO Inc. $RGNX

Nov -03

Source: defenseworld.net

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REGENXBIO Announces Completion of Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program

Oct -30

Source: prnewswire.com

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REGENXBIO to Host Conference Call on November 6 to Discuss Third Quarter 2025 Financial Results and Operational Highlights

Oct -29

Source: prnewswire.com

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REGENXBIO to Participate in Upcoming Investor Conferences

Oct -15

Source: prnewswire.com

4. Business Breakdown

4.a. Revenues by Country

4.b. Revenues by Segment

5. Expected revenues mid-term growth (11.30%)

6. Segments

Gene Therapy

Expected Growth: 11.3%

REGENXBIO's 11.3% growth in Gene Therapy is driven by increasing adoption of novel gene therapies, strong pipeline of rare disease treatments, and strategic partnerships. Growing demand for one-time curative treatments, advancements in gene editing technologies, and expanding regulatory approvals also contribute to the segment's growth.

7. Detailed Products

NAV Technology Platform

A proprietary adeno-associated virus (AAV) gene therapy platform that enables the development of gene therapies for a wide range of diseases and disorders.

RGX-111

A gene therapy for the treatment of Mucopolysaccharidosis Type I (MPS I), a rare genetic disorder.

RGX-121

A gene therapy for the treatment of Mucopolysaccharidosis Type II (MPS II), a rare genetic disorder.

RGX-314

A gene therapy for the treatment of wet age-related macular degeneration (wet AMD), a leading cause of vision loss in older adults.

RGX-501

A gene therapy for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), a rare genetic disorder.

8. REGENXBIO Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

REGENXBIO Inc. faces moderate threat from substitutes due to the presence of alternative gene therapy companies, but the company's unique NAV Technology Platform and strong pipeline of products mitigate this threat.

Bargaining Power Of Customers

REGENXBIO Inc. has a diverse customer base, and no single customer accounts for a significant portion of its revenue, reducing the bargaining power of customers.

Bargaining Power Of Suppliers

REGENXBIO Inc. relies on a few key suppliers for raw materials and services, but the company's strong relationships and diversified supply chain mitigate the bargaining power of suppliers.

Threat Of New Entrants

The gene therapy industry is highly competitive, and new entrants with innovative technologies and significant resources could potentially disrupt REGENXBIO Inc.'s market position.

Intensity Of Rivalry

The gene therapy industry is highly competitive, with several established players and new entrants vying for market share, leading to a high intensity of rivalry.

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

Value
Debt Weight 22.27%
Debt Cost 7.69%
Equity Weight 77.73%
Equity Cost 10.82%
WACC 10.12%
Leverage 28.64%

11. Quality Control: REGENXBIO Inc. passed 3 out of 9 key points

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

Stock-Card
Fortress Biotech

A-Score: 4.5/10

Value: 8.4

Growth: 5.3

Quality: 3.9

Yield: 0.0

Momentum: 8.5

Volatility: 1.0

1-Year Total Return ->

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Stock-Card
Adaptive Biotechnologies

A-Score: 4.4/10

Value: 6.0

Growth: 3.8

Quality: 4.7

Yield: 0.0

Momentum: 10.0

Volatility: 1.7

1-Year Total Return ->

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Stock-Card
Tonix Pharmaceuticals

A-Score: 4.2/10

Value: 7.6

Growth: 5.2

Quality: 3.8

Yield: 0.0

Momentum: 8.5

Volatility: 0.3

1-Year Total Return ->

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Stock-Card
Curis

A-Score: 3.6/10

Value: 9.2

Growth: 3.9

Quality: 6.0

Yield: 0.0

Momentum: 1.0

Volatility: 1.3

1-Year Total Return ->

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Stock-Card
Phathom Pharmaceuticals

A-Score: 3.4/10

Value: 8.2

Growth: 4.4

Quality: 6.0

Yield: 0.0

Momentum: 1.5

Volatility: 0.0

1-Year Total Return ->

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Stock-Card
REGENXBIO

A-Score: 3.1/10

Value: 7.0

Growth: 2.2

Quality: 3.9

Yield: 0.0

Momentum: 4.0

Volatility: 1.3

1-Year Total Return ->

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Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

13.16$

Current Price

13.16$

Potential

-0.00%

Expected Cash-Flows