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RegenXBio
(NASDAQ:RGNX)
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Rating:44Neutral
Price Target:
$8.00
▼(-14.71% Downside)
Action:Reiterated
Date:09/23/26
The score is held down primarily by weak financial quality (large losses, high leverage, and substantial ongoing cash burn) and a bearish technical trend (below key moving averages with negative MACD). Corporate events provide some support via major milestone/settlement cash inflows, but are tempered by the RGX-121 clinical hold and evidence of continued reliance on external financing; valuation provides limited help due to negative earnings and no dividend.
Positive Factors
Revenue growth
The near-doubling of TTM revenue shows that REGENXBIO is converting its platform and partnering activities into materially greater reported sales. Although profitability remains unproven, stronger revenue provides a broader base for funding development and could improve operating leverage as programs advance.
Negative Factors
RGX-121 clinical hold
The FDA clinical hold disrupts a major Hunter Syndrome program and management does not expect to resubmit its BLA in the near term. Extended monitoring and data analysis may delay development, reduce near-term pipeline contribution, and increase reliance on other candidates to sustain strategic momentum.
Read all positive and negative factors
Positive Factors
Negative Factors
Revenue growth
The near-doubling of TTM revenue shows that REGENXBIO is converting its platform and partnering activities into materially greater reported sales. Although profitability remains unproven, stronger revenue provides a broader base for funding development and could improve operating leverage as programs advance.
Read all positive factors
RegenXBio Key Performance Indicators (KPIs)
Any
Revenue by Segment
Analyzes income from different business units, highlighting which segments drive growth and profitability, and indicating potential areas for strategic focus or improvement.
Analyzes income from different business units, highlighting which segments drive growth and profitability, and indicating potential areas for strategic focus or improvement.
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The Fly
The FlyRegenXBio (RGNX) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$491.80M
Dividend YieldN/A
Average Volume (3M)1.36M
Price to Earnings (P/E)―
Beta (1Y)2.62
Revenue Growth12.00%
EPS Growth-9.21%
CountryUS
Employees371
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-3.79
Shares Outstanding66,013,190
10 Day Avg. Volume1,527,629
30 Day Avg. Volume1,355,478
Financial Highlights & Ratios
PEG Ratio0.21
Price to Book (P/B)7.23
Price to Sales (P/S)4.36
P/FCF Ratio-5.88
Enterprise Value/Market Cap<0.01
Enterprise Value/Revenue<0.01
Enterprise Value/Gross Profit<0.01
Enterprise Value/Ebitda>-0.01
Forecast
1Y Price Target
$22.00Price Target Upside134.54% Upside
Rating ConsensusStrong Buy
Number of Analyst Covering9
EPS Forecast (FY)-3.31
Revenue Forecast (FY)$168.21M
RegenXBio Business Overview & Revenue Model
Company Description
REGENXBIO Inc. is a biotechnology company operating in the clinical stage, dedicated to developing gene therapy candidates. These therapies are designed to introduce genes into cells, aiming either to rectify genetic deficiencies or to stimulate t...
How the Company Makes Money
REGENXBIO primarily generates revenue through (1) licensing and royalty-based arrangements and (2) collaboration revenue from partnerships that use its AAV vector technology and intellectual property. Under these agreements, the company may earn u...
RegenXBio Earnings Call Summary
Earnings Call Date:May 14, 2026
(Q1-2026)
| % Change Since: |
Next Earnings Date:Nov 11, 2026
Earnings Call Sentiment Positive
The call presented a notably positive clinical and programmatic update: RGX-202 met its pivotal primary endpoint with high biomarker expression, demonstrated an unusually strong correlation between microdystrophin and functional benefit (>0.9), and showed encouraging interim safety and functional signals with plans for potential accelerated approval in 2027. Key operational and regulatory risks were acknowledged — chiefly the limited number of mature 12‑month functional datapoints so far, reliance on external controls (and FDA concern about bias), and the possibility that the agency could request randomized trials which would materially delay approval. Remaining uncertainties on long‑term durability and the logistical burden of the prophylaxis regimen were also discussed. Overall the clinical readout and pipeline progress meaningfully outweigh near‑term risks, but regulatory process and more mature datasets remain critical next milestones.Positive Updates
Pivotal Phase III Primary Endpoint Met
AFFINITY DUCHENNE pivotal Phase III met its primary endpoint with high statistical significance: 93% (28 of 30 biopsied patients) exceeded the predefined microdystrophin >10% threshold. Interim safety available for 31 dosed pivotal patients.
Negative Updates
Limited Mature Functional Dataset
Only 9 patients had 12‑month functional data included in the top‑line functional analyses; microdystrophin biopsy data available for 30 of 31 pivotal patients (one patient refused biopsy). Company expects more 12‑month functional readouts (estimated ~15–20 patients by early 2027).
Read all updates
Q1-2026 Updates
Positive
Negative
Pivotal Phase III Primary Endpoint Met
AFFINITY DUCHENNE pivotal Phase III met its primary endpoint with high statistical significance: 93% (28 of 30 biopsied patients) exceeded the predefined microdystrophin >10% threshold. Interim safety available for 31 dosed pivotal patients.
Read all positive updates
Company Guidance
Management’s guidance highlighted program and regulatory milestones and specific metrics: the company has dosed >50 patients across pivotal and confirmatory studies with line-of-sight to 60 patients by mid‑2026; plans to seek potential accelerated approval in 2027 (rolling BLA possible) and expects a safety database of ~50 patients at filing and roughly 15–20 patients with 12‑month functional data by early 1H‑2027; current top‑line dataset includes 30 patients with microdystrophin biomarker data, 31 with interim safety, and 9 with 12‑month functional data (age ≥4); the pivotal met its primary endpoint with 93% (28/30) >10% microdystrophin at Week 12, 80% >40% expression, mean expression 71.1% overall and 41.6% in patients ≥8; there is a >0.9 correlation coefficient between microdystrophin and NSAA/cTAP functional measures; safety to date: 2 treatment‑related SAEs among 31 patients (one subacute myocarditis, one asymptomatic liver injury with GGT peak 123 U/L), both resolved without sequelae, no drug‑related thrombocytopenia/myositis/neurotoxicity, and mean GGT and total bilirubin remained below ULN through 12 months; additional pipeline and corporate milestones include dosing the first Phase IIb diabetic retinopathy patient in Q2‑2026 for a $100M AbbVie milestone, subretinal pivotal readouts in Q4‑2026, RGX‑121 hold lifted and CRL appeal filed, and an expectation of 3 potential approvals over the next couple of years (including 2 blockbuster opportunities).RegenXBio Financial Statement Overview
Summary
Income Statement
22
Negative
Balance Sheet
35
Negative
Cash Flow
18
Very Negative
| Breakdown | TTM | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Dec 2021 |
|---|---|---|---|---|---|---|
Income Statement | ||||||
| Total Revenue | 174.48M | 170.44M | 83.33M | 90.24M | 112.72M | 470.35M |
| Gross Profit | 94.61M | 150.14M | 49.76M | 53.03M | 58.18M | 418.51M |
| EBITDA | -139.12M | -133.25M | -198.23M | -239.46M | -244.24M | 177.09M |
| Net Income | -196.43M | -193.88M | -227.10M | -263.49M | -280.32M | 127.84M |
Balance Sheet | ||||||
| Total Assets | 386.62M | 453.03M | 465.99M | 573.97M | 833.27M | 1.11B |
| Cash, Cash Equivalents and Short-Term Investments | 105.52M | 230.07M | 234.69M | 275.26M | 364.64M | 457.44M |
| Total Debt | 68.95M | 260.52M | 82.03M | 89.29M | 94.80M | 86.68M |
| Total Liabilities | 314.85M | 350.30M | 206.34M | 262.23M | 317.07M | 349.61M |
| Stockholders Equity | 71.77M | 102.73M | 259.65M | 311.74M | 516.20M | 764.30M |
Cash Flow | ||||||
| Free Cash Flow | -249.67M | -126.38M | -175.56M | -228.37M | -238.21M | 134.70M |
| Operating Cash Flow | -246.67M | -123.96M | -173.13M | -218.41M | -207.49M | 218.88M |
| Investing Cash Flow | 216.34M | -15.87M | 103.45M | 190.94M | -11.93M | -406.64M |
| Financing Cash Flow | -12.73M | 116.77M | 92.68M | -34.97M | -28.84M | 195.25M |
RegenXBio Technical Analysis
Negative
9.38
Price Trends
9.44
Negative
9.31
Negative
9.97
Negative
Market Momentum
-0.63
Positive
35.66
Neutral
21.47
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For RGNX, the sentiment is Negative. The current price of 9.38 is above the 20-day moving average (MA) of 8.28, below the 50-day MA of 9.44, and below the 200-day MA of 9.97, indicating a bearish trend. The MACD of -0.63 indicates Positive momentum. The RSI at 35.66 is Neutral, neither overbought nor oversold. The STOCH value of 21.47 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for RGNX.
RegenXBio Risk Analysis
RegenXBio disclosed 54 risk factors in its most recent earnings report. RegenXBio reported the most risks in the "Tech & Innovation" category.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks
RegenXBio Peers Comparison
UnderperformOutperform
Sector (51)
Name | Overall Rating | Market Cap | P/E Ratio | ROE | Dividend Yield | Revenue Growth | EPS Growth |
|---|---|---|---|---|---|---|---|
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
50 Neutral | $767.73M | -3.69 | -72.28% | ― | ― | 31.81% | |
50 Neutral | $1.00B | -5.35 | -43.31% | ― | -83.86% | -646.52% | |
47 Neutral | $536.97M | -34.91 | -13.08% | ― | ― | 29.73% | |
46 Neutral | $1.54B | -11.81 | 1893.60% | 5.23% | -100.00% | 19.23% | |
44 Neutral | $491.80M | -1.96 | -220.07% | ― | 12.00% | -9.21% |
* Healthcare Sector Average
RGNX
RegenXBio
7.53
-2.24
-22.93%
SLDB
Solid Biosciences
7.16
1.29
21.98%
MNPR
Monopar Therapeutics Inc
80.00
-4.73
-5.58%
GLUE
Monte Rosa Therapeutics
11.74
4.24
56.53%
INBX
Inhibrx Biosciences Inc
104.83
70.85
208.51%
RegenXBio Corporate Events
Business Operations and StrategyLegal Proceedings
RegenXBio Reaches Patent Settlement Over AAV Gene Therapy
Positive
Sep 23, 2026
On September 23, 2026, REGENXBIO and the Trustees of the University of Pennsylvania reached a settlement with Sarepta Therapeutics and Catalent to resolve all patent infringement litigation tied to Sarepta’s SRP-9001, marketed as ELEVIDYS in...
Business Operations and StrategyExecutive/Board Changes
RegenXBio reshapes board with new independent director
Positive
Aug 25, 2026
On August 25, 2026, REGENXBIO Inc. appointed biotechnology investor Greg Ciongoli to its Board of Directors as a Class II independent director, with a term running through the 2029 annual meeting. He was also named to the Audit Committee and the N...
Business Operations and StrategyLegal Proceedings
RegenXBio Faces RGX-121 Clinical Hold, Refocuses Pipeline
Negative
Aug 24, 2026
On August 24, 2026, REGENXBIO reported that the U.S. Food and Drug Administration placed its Investigational New Drug application for the Phase I/II/III trial of RGX-121, an investigational gene therapy for Hunter Syndrome, on clinical hold after ...
Business Operations and StrategyPrivate Placements and FinancingRegulatory Filings and Compliance
RegenXBio Raises Capital Through Public Equity Offering
Positive
Jul 20, 2026
On July 16, 2026, REGENXBIO Inc. entered into an underwriting agreement for an underwritten public offering of 10,003,889 shares of common stock at $9.00 per share and 1,111,111 pre-funded warrants at $8.9999 per warrant. The company also granted ...
Business Operations and StrategyPrivate Placements and FinancingProduct-Related Announcements
Regenxbio Advances Diabetic Retinopathy Gene Therapy Trial
Positive
Jun 29, 2026
On June 29, 2026, REGENXBIO Inc. reported that the first patient had been dosed in its Phase IIb/III NAAVIGATE trial of surabgene lomparvovec for non-proliferative diabetic retinopathy using suprachoroidal delivery. The dosing triggered a $100 mil...
Glossary
BuyA stock rated as a "Buy" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock is likely to deliver higher returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
HoldA stock rated as a "Hold" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly compelling nor unfavorable for investment. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
SellA stock rated as a "Sell" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock may deliver lower returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
Disclaimer
This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.