Key Takeaways
- Shares of uniQure (QURE) climbed 36% on Monday following the announcement that Vinay Prasad will depart from the FDA’s Center for Biologics Evaluation and Research
- Prasad had been responsible for regulatory oversight of uniQure’s experimental Huntington’s disease therapy AMT-130 and faced criticism for overriding review team decisions
- RBC Capital elevated QURE to Outperform from Sector Perform, increasing their price objective from $11 to $35
- Other biotech firms also rallied: REGENXBIO (RGNX) climbed 13% while Biohaven (BHVN) advanced 23%
- The company plans a Type B FDA meeting in Q2 2026
The past week has delivered remarkable volatility for uniQure investors. On March 5, an FDA representative publicly criticized the biotech firm for allegedly “performing a distorted or manipulated comparison” within its phase 1/2 clinical study of AMT-130, an investigational treatment for Huntington’s disease. Surprisingly, shares still climbed 18% by market close.
The following day, March 6, brought news that Vinay Prasad, who leads the FDA’s Center for Biologics Evaluation and Research (CBER), plans to exit the agency in April for an academic position. QURE shares skyrocketed an additional 34%. By Monday’s session, the total gain reached 36%.
Prasad held direct responsibility for the regulatory review of AMT-130. The agency had informed uniQure that approval would not be granted based on comparisons to natural history data — representing what the company described as a “key shift” from prior understandings established before recent FDA leadership changes.
Additionally, the FDA demanded that uniQure conduct a comprehensive phase 3 clinical trial, creating an expensive and lengthy obstacle for a therapy targeting a rare condition.
Prasad’s announced resignation has apparently shifted investor sentiment and analyst perspectives. RBC Capital’s Luca Issi elevated his rating on QURE from Sector Perform to Outperform, simultaneously raising his price objective from $11 to $35. His assessment now places the probability of eventual AMT-130 approval at 50%.
“We believe that Prasad’s departure is likely to open up a more balanced discussion on risk/reward for HD,” Issi stated in his research note.
Wall Street Weighs In
Stifel’s Paul Matteis characterized Prasad’s departure as “a big win for biotech, especially for companies in the rare disease space.” He highlighted allegations that Prasad had overridden internal FDA review teams to deliver unfavorable assessments on multiple therapies.
Truist’s research team observed that Prasad’s leadership “marked a sharp departure from the more flexible regulatory approach for rare and serious diseases” that characterized the tenure of his predecessor, Peter Marks. Several pharmaceutical companies, they emphasized, encountered evolving regulatory expectations that contradicted earlier agreements established during initial development discussions with the agency.
The gene therapy sector experienced broad gains following the announcement. REGENXBIO (RGNX) advanced 13% and Biohaven (BHVN) surged 23% on the development.
The Road Ahead for uniQure
The company has secured a Type B meeting with the FDA planned for the second quarter of 2026. This discussion will represent a critical juncture for determining the regulatory pathway for AMT-130.
Prasad’s official departure is scheduled for April.
Interestingly, on March 5—the same day public criticism emerged regarding uniQure’s trial methodology—shares nevertheless finished the session 18% higher, suggesting investors were already factoring in questions about Prasad’s role in the regulatory outcome.
