QURE: Future Regulatory Clarity Will Drive Gene Therapy Upside Despite FDA Setback

The analyst price target for uniQure has been reduced from $56 to $38 as analysts factor in heightened regulatory uncertainty around AMT-130, a higher perceived discount rate, and more conservative revenue growth expectations, despite still-supportive long term Huntington's disease data.

Analyst Commentary

Analyst views on uniQure have shifted rapidly from optimism following the three year AMT 130 data to caution after the FDA withdrew support for a BLA based on the current Phase 1 2 package. This has driven a wide dispersion in price targets and ratings.

Bullish Takeaways

• Bullish analysts still view AMT 130 as a leading Huntington's candidate, citing robust, durable slowing of disease progression that could ultimately justify meaningful premium pricing and category leadership.
• Several models continue to assume a commercial launch around 2028. This suggests that, while timelines are pushed out, long term revenue potential for AMT 130 remains intact if regulatory hurdles are resolved.
• Positive sentiment around Huntington's patient advocacy momentum and unmet need supports assumptions for strong initial uptake once clarity on approvability and labeling is restored.
• Despite cuts, some price targets in the $45 to $53 range reflect confidence that improved trial design and additional data could restore a higher probability of success and re rate the shares from depressed levels.

Bearish Takeaways

• Bearish analysts emphasize that the FDA's rejection of external control comparisons as a primary regulatory path introduces structural uncertainty. This has led to reduced probabilities of success and lower discounted cash flow valuations.
• The loss of a near term BLA filing and delay of potential launch from 2026 to at least 2028 compress near to mid term revenue expectations and lengthen the period of cash burn, heightening financing and execution risk.
• The roughly 50 percent share price decline following the FDA reversal is seen as justified by some, given concerns that management must now redesign or expand clinical development, adding time, cost, and operational complexity.
• More cautious models also factor in a higher discount rate for the gene therapy portfolio overall, reflecting worries that shifting FDA standards on natural history controls may weigh on future programs beyond AMT 130.

What's in the News

• William Blair downgraded uniQure to Market Perform from Outperform, reflecting increased regulatory uncertainty and a more cautious risk reward view (periodical).
• Final FDA meeting minutes from the October 29, 2025 pre BLA meeting confirmed that Phase I II AMT 130 data are currently unlikely to provide primary evidence for a BLA. This is forcing uniQure to reassess its regulatory strategy and timelines (key development).
• Preliminary FDA feedback at the same pre BLA meeting indicated the agency no longer supports reliance on external control comparisons as the main basis for AMT 130 approval. This is creating significant uncertainty around the timing and path of any BLA submission (key development).
• uniQure completed a follow on equity offering of roughly $300 million in ordinary shares and pre funded warrants, bolstering its balance sheet ahead of potentially longer and more complex AMT 130 development (key development).
• The company announced positive topline Phase I II data for AMT 130 showing statistically significant slowing of Huntington's disease progression versus an external control. This is underpinning investor debate over whether clinical strength can ultimately overcome regulatory hurdles (key development).

Valuation Changes

• Fair Value: Model implied fair value per share has fallen slightly, moving from approximately 58.21 to 55.44.
• Discount Rate: The assumed discount rate has risen modestly, from about 7.18 percent to 7.34 percent, reflecting higher perceived risk.
• Revenue Growth: Long term revenue growth expectations have been reduced meaningfully, from roughly 170.46 percent to 158.12 percent.
• Net Profit Margin: The projected net profit margin remains effectively unchanged, holding at around 16.03 percent.
• Future P/E: The forward price to earnings multiple has increased from about 109.06 times to 120.01 times, indicating a higher valuation multiple on future earnings despite reduced growth assumptions.