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RegenXBio
(NASDAQ:RGNX)
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Rating:49Neutral
Price Target:
$8.00
▼(-14.71% Downside)
Action:Reiterated
Date:08/26/26
The score is held down primarily by weak financial performance (large losses, heavy cash burn, and elevated leverage) and bearish technical trends (below major moving averages with negative momentum). These are partly offset by a strong earnings-call read-through, with encouraging clinical efficacy/safety signals and clear regulatory milestones, though meaningful approval and data-maturity risks remain. Valuation contributes limited support due to negative earnings and no dividend yield.
Positive Factors
Strong RGX-202 clinical efficacy
RGX-202’s strong biomarker response and close association with functional outcomes support microdystrophin as a potentially meaningful surrogate. If confirmed with larger follow-up, this could improve approval prospects and strengthen the company’s lead program.
Negative Factors
Persistent losses and cash burn
REGENXBIO has not yet demonstrated self-sustaining cash generation, leaving its ability to fund development dependent on financing, milestones or future product launches. Continued clinical spending could prolong losses before commercialization creates durable earnings.
Read all positive and negative factors
Positive Factors
Negative Factors
Strong RGX-202 clinical efficacy
RGX-202’s strong biomarker response and close association with functional outcomes support microdystrophin as a potentially meaningful surrogate. If confirmed with larger follow-up, this could improve approval prospects and strengthen the company’s lead program.
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
$633.73M
Dividend YieldN/A
Average Volume (3M)1.29M
Price to Earnings (P/E)―
Beta (1Y)2.56
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. Volume1,652,196
30 Day Avg. Volume1,286,079
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 makes money through (1) collaboration and license revenue tied to its NAV AAV vector technology and (2) potential future product revenue if its wholly owned or co-developed gene therapy candidates receive regulatory approval an...
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
10.98
Negative
9.49
Negative
10.34
Negative
Market Momentum
-0.35
Positive
43.17
Neutral
50.44
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 below the 20-day moving average (MA) of 10.17, below the 50-day MA of 10.98, and below the 200-day MA of 10.34, indicating a bearish trend. The MACD of -0.35 indicates Positive momentum. The RSI at 43.17 is Neutral, neither overbought nor oversold. The STOCH value of 50.44 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 |
|---|---|---|---|---|---|---|---|
56 Neutral | $780.49M | -50.66 | -13.08% | ― | ― | 29.73% | |
54 Neutral | $1.84B | -13.77 | 1893.60% | ― | -100.00% | 19.23% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
50 Neutral | $1.10B | -5.46 | -72.28% | ― | ― | 31.81% | |
49 Neutral | $633.73M | -2.43 | -220.07% | ― | 12.00% | -9.21% | |
43 Neutral | $1.21B | -6.60 | -43.31% | ― | -83.86% | -646.52% |
* Healthcare Sector Average
RGNX
RegenXBio
9.20
-0.22
-2.34%
SLDB
Solid Biosciences
10.42
4.86
87.41%
MNPR
Monopar Therapeutics Inc
118.55
79.71
205.23%
GLUE
Monte Rosa Therapeutics
14.38
9.47
192.87%
INBX
Inhibrx Biosciences Inc
122.26
94.54
341.05%
RegenXBio Corporate Events
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...
Business Operations and StrategyExecutive/Board ChangesShareholder Meetings
RegenXBio Launches Targeted Employee Stock Option Exchange
Positive
Jun 3, 2026
REGENXBIO Inc., a gene therapy-focused biotechnology company, reported the results of its 2026 Annual Meeting of Stockholders held on May 29, 2026, where shareholders elected three Class II directors to serve until 2029 and ratified Pricewaterhous...
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.