uniQure (QURE) AI Stock Analysis

• Market Cap: $656.08M
• Dividend Yield: N/A
• Average Volume (3M): 3.30M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.36
• Revenue Growth: -44.90%
• EPS Growth: 11.49%
• Country: US
• Employees: 209
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -0.57
• Shares Outstanding: 61,603,770
• 10 Day Avg. Volume: 6,148,841
• 30 Day Avg. Volume: 3,298,106
• PEG Ratio: 0.23
• Price to Book (P/B): 6.92
• Price to Sales (P/S): 85.48
• P/FCF Ratio: -7.73
• Enterprise Value/Market Cap: 0.97
• Enterprise Value/Revenue: 39.71
• Enterprise Value/Gross Profit: 44.35
• Enterprise Value/Ebitda: -3.58
• 1Y Price Target: $24.00
• Price Target Upside: 125.35% Upside
• Rating Consensus: Moderate Buy
• Number of Analyst Covering: 12
• EPS Forecast (FY): -3.33
• Revenue Forecast (FY): $51.18M

• Rating: 46 Neutral
• Price Target: $10.50 ▼(-1.41% Downside)
• Action: Reiterated Date: 03/06/26
• Date: Action: Reiterated 03/06/26

The score is held down mainly by weak financial performance (multi-year losses and ongoing cash burn) and bearish technicals (strong downtrend versus moving averages). The earnings call adds some support from encouraging AMT-130 clinical data and a stronger cash position, but this is offset by heightened FDA/regulatory uncertainty and program safety pauses. Valuation is neutral-to-weak given the negative P/E and lack of a dividend.

Positive Factors

Strong liquidity / runway

A markedly stronger year‑end cash position provides durable optionality to fund regulatory discussions, additional data generation and potential Phase III feasibility work without immediate dilution. This cash buffer reduces near‑term financing pressure while the company addresses design, safety and regulatory hurdles.

Robust AMT‑130 clinical efficacy

Three‑year disease‑modifying signals position AMT‑130 as a potentially transformative, one‑time therapy for Huntington’s disease. Durable efficacy and corroborating biomarker trends strengthen scientific rationale, KOL support and ex‑U.S. regulatory/early access discussions that could underpin long‑term market differentiation if safety/regulatory hurdles are resolved.

Partnership/licensing commercialization model

A partnership‑centric model (commercial rights via partners and licensing/collaboration revenue) reduces uniQure’s direct commercialization burden and capital intensity. Reliance on upfront, milestone and royalty streams supports diversified non‑dilutive funding options and lets management focus R&D resources on pipeline advancement over building a large sales organization.

Negative Factors

Regulatory demand for sham‑controlled Phase III

The FDA’s recommendation for a randomized sham‑surgery Phase III materially raises trial size, cost, complexity and ethical/feasibility hurdles. This structural requirement increases timelines and capital needs, risks delaying or preventing U.S. approval and forces prolonged regulatory dialogue that could reshape global development strategy.

Clinical safety pause (AMT‑191)

Dose‑limiting liver events and a protocol‑mandated pause create durable program risk for the Fabry candidate. Safety signals can force dose reductions, additional monitoring, altered endpoints or extra cohorts, prolonging development, increasing costs and complicating regulatory pathways across the pipeline where similar vector or delivery risks exist.

Multi‑year losses and cash burn history

Sustained multi‑year losses and negative margins indicate underlying earnings weakness and reliance on capital markets for funding. Prior negative equity in 2024 and continued cash consumption increase the risk that future development (e.g., a costly Phase III) will require additional dilutive or debt financing, constraining long‑term financial flexibility.

uniQure Business Overview & Revenue Model

Company Description

uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-210, a product candidate for the treatment of Parkinson's disease; AMT-260 for temporal lobe epilepsy; AMT-240, a preclinical product candidate for the treatment of autosomal dominant Alzheimer's disease; and AMT-161 for the treatment of amyotrophic lateral sclerosis. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

How the Company Makes Money

uniQure generates revenue primarily through the sale of its gene therapy products, with significant income derived from its approved treatment for hemophilia B. The company also earns revenue through partnerships and collaborations with other biopharmaceutical companies, which may involve upfront payments, milestone payments based on clinical and regulatory achievements, and royalties on sales of partnered products. Additionally, uniQure may receive funding from government grants or research initiatives aimed at advancing gene therapy technologies, which contribute to its overall financial performance.

uniQure Financial Statement Overview

Summary

Overall fundamentals are weak: the company shifted from 2021 profitability to large losses and deeply negative net margins in 2022–2025, alongside sustained cash burn (only modestly improved in 2025). The balance sheet is a relative bright spot with moderate leverage and debt trending down, but the prior negative equity in 2024 highlights ongoing stress from accumulated losses.

Income Statement

Revenue has been highly volatile: a major spike in 2021 followed by a sharp drop in 2022, a further decline in 2023, and then a rebound in 2024–2025. Profitability is the key weakness—after strong profits in 2021, the company has posted large losses in 2022–2025 with deeply negative net margins. A notable positive is that gross margin recovered strongly in 2025 versus the weaker 2023–2024 level, but overall earnings power remains meaningfully negative.

Balance Sheet

Leverage appears moderate in most years with debt generally well below assets, and 2025 shows positive equity with a reasonable debt-to-equity level. However, the balance sheet showed stress in 2024 when equity turned negative, which can signal accumulated losses and weaker financial flexibility. Returns on equity have been poor (negative in most years), consistent with ongoing losses, but debt has also come down materially versus 2022–2023, which is a stabilizing factor.

Cash Flow

Cash generation has been inconsistent: strong positive operating and free cash flow in 2021, followed by sustained, large cash burn from 2022 through 2025. A modest positive is that recent free cash flow has improved versus prior years (notably in 2025), but the business is still consuming significant cash, which increases reliance on external funding if the trend persists.

Breakdown	Dec 2025	Dec 2024	Dec 2023	Dec 2022	Dec 2021
Income Statement
Total Revenue	16.10M	27.12M	15.84M	106.48M	524.00M
Gross Profit	14.41M	8.79M	2.21M	103.14M	499.03M
EBITDA	-178.58M	-160.75M	-253.10M	-108.02M	319.00M
Net Income	-198.97M	-239.56M	-308.48M	-126.79M	329.59M
Balance Sheet
Total Assets	824.91M	556.54M	831.69M	704.96M	809.18M
Cash, Cash Equivalents and Short-Term Investments	622.54M	367.52M	617.89M	352.84M	556.26M
Total Debt	63.39M	66.06M	138.41M	142.89M	135.72M
Total Liabilities	626.01M	563.29M	624.02M	228.96M	213.40M
Stockholders Equity	198.90M	-6.75M	207.67M	476.01M	595.78M
Cash Flow
Free Cash Flow	-177.96M	-186.10M	-153.08M	-162.75M	270.52M
Operating Cash Flow	-177.96M	-182.73M	-145.93M	-145.06M	287.96M
Investing Cash Flow	-321.62M	162.97M	-205.69M	-182.73M	-67.39M
Financing Cash Flow	415.40M	-59.49M	362.72M	1.45M	94.86M

uniQure Technical Analysis

• Technical Analysis Sentiment: Negative
• Last Price: 10.65
• 50DMA: Negative
• 100DMA: Negative
• 200DMA: Negative
• MACD: Positive
• RSI: Positive
• STOCH: Positive

Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For QURE, the sentiment is Negative. The current price of 10.65 is below the 20-day moving average (MA) of 20.31, below the 50-day MA of 22.39, and below the 200-day MA of 25.09, indicating a bearish trend. The MACD of -3.48 indicates Positive momentum. The RSI at 29.66 is Positive, neither overbought nor oversold. The STOCH value of 5.05 is Positive, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for QURE.

uniQure Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
BCRX	70 Outperform	$2.13B	6.20	―	―	45.38%	92.85%
PHVS	56 Neutral	$1.66B	-8.91	-51.80%	―	―	-20.08%
AMLX	55 Neutral	$1.56B	-7.89	-61.59%	―	-100.34%	52.85%
AGIO	52 Neutral	$1.62B	-3.82	-30.20%	―	36.26%	-158.98%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
IMNM	50 Neutral	$2.37B	―	-52.09%	―	-4.44%	63.58%
QURE	46 Neutral	$656.08M	-6.92	-207.10%	―	-44.90%	11.49%

uniQure Corporate Events

uniQure Highlights 2025 Results and Gene Therapy Progress

On March 2, 2026, uniQure reported its fourth-quarter and full-year 2025 financial results and highlighted progress in its gene therapy pipeline, led by AMT-130 for Huntington’s disease, which showed statistically significant 36‑month slowing of disease progression and a favorable safety profile in a Phase I/II study. Despite these data, the FDA stated after an October 2025 pre‑BLA meeting and a January 2026 Type A meeting that the current external‑control evidence is insufficient for approval and strongly recommended a randomized, sham‑surgery‑controlled Phase III trial, prompting uniQure to plan further regulatory discussions in the second quarter of 2026.

The company also advanced AMT‑260 for refractory mesial temporal lobe epilepsy, completing enrollment of the first Phase I/IIa dose cohort in 2025 after early data from one patient showed a 92% seizure reduction, and it reported February 2026 Phase I/II data from AMT‑191 in Fabry disease demonstrating durable, dose‑dependent increases in α‑Gal A activity that allowed withdrawal of enzyme replacement therapy in some patients, albeit with dose‑limiting liver enzyme elevations that triggered a protocol‑mandated dosing pause in mid‑ and high‑dose cohorts. Enrollment in the AMT‑162 ALS study remains on voluntary pause following a 2025 dose‑limiting toxicity, while uniQure’s 2025 follow‑on equity offerings and debt refinancing lifted year‑end cash, cash equivalents and current investment securities to $622.5 million, a level the company expects will fund operations into the second half of 2029 and underpins its ability to pursue the larger, regulator‑requested trial program.

The most recent analyst rating on (QURE) stock is a Buy with a $45.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

uniQure updates gene therapy pipeline and regulatory outlook

In January 2026, uniQure N.V. updated its corporate presentation to highlight its positioning as a leader in gene therapy, underscoring a robust clinical pipeline and substantial liquidity to fund development across multiple rare and serious neurological and metabolic diseases. The refreshed materials emphasize AMT-130 as a first potential disease-modifying, one-time gene therapy for Huntington’s disease with so-called blockbuster potential, outline upcoming data readouts for AMT-260 in mesial temporal lobe epilepsy and AMT-191 in Fabry disease over the next three to six months, and signal focused engagement with the U.S. Food and Drug Administration on a potential biologics license application pathway, developments that collectively underscore the company’s bid to consolidate its competitive position in gene therapy and may be closely watched by investors, patients and partners for signs of clinical and regulatory progress.

The most recent analyst rating on (QURE) stock is a Hold with a $22.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

uniQure prepares for FDA Type A meeting on AMT-130

On January 9, 2026, uniQure announced that the U.S. Food and Drug Administration has scheduled a Type A meeting with the company to discuss the Biologics License Application data package supporting a potential accelerated approval of AMT-130, its investigational gene therapy for Huntington’s disease. Management highlighted the significant unmet medical need in the Huntington’s disease community and its commitment to timely patient access, signaling that a favorable regulatory outcome could be a key milestone for uniQure’s Huntington’s program and broader gene therapy pipeline; the company plans to issue a regulatory update after it receives the FDA’s official meeting minutes.

The most recent analyst rating on (QURE) stock is a Buy with a $60.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.