Small Cap Gene Therapy Stock More Than Triples on Positive Trial Results
Breakthrough data in Huntington’s disease study propels UniQure shares to record gains, with ripple effects for ClearPoint Neuro and Hercules Capital.
Shares of uniQure (NASDAQ: QURE) staged one of the most dramatic rallies of the year on Wednesday, more than tripling after the biotech firm unveiled pivotal trial data showing its experimental Huntington’s disease treatment, AMT-130, significantly slowed disease progression. The surge reverberated across related stocks, lifting partner ClearPoint Neuro (NASDAQ: CLPT) and drawing attention to financing partner Hercules Capital (NYSE: HTGC).
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By the closing bell, UniQure had soared 248% to $47.50, after hitting an intraday high of $51.10. Trading volume exploded to over 69 million shares, nearly 6,000% above its daily average. In after-hours trading, QURE added another 4.2%, last changing hands at $49.50. Over the past year, the stock has climbed an astounding 824%.
Historic Data in Huntington’s Disease
The market reaction stemmed from topline results in UniQure’s Phase I/II trial of AMT-130. Patients receiving the high-dose treatment saw a 75% slowing in disease progression at 36 months compared to external controls, meeting the study’s primary endpoint. The therapy also achieved a 60% slowing in functional decline, a key secondary endpoint.
Sarah Tabrizi, professor of clinical neurology and director of the University College London Huntington’s Disease Center, said:
“I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need persists. These data indicate that AMT-130 has the potential to meaningfully slow disease progression – offering long-awaited hope to individuals and families impacted by this devastating disease.”
Huntington’s, a devastating inherited disorder that steadily erodes motor and cognitive function, currently has no FDA-approved treatments that slow its progression. The potential for AMT-130 to become the first therapy to alter the course of the disease represents a watershed moment for patients and families.
ClearPoint Neuro Rides the Wave
While UniQure grabbed headlines, ClearPoint Neuro also surged, finishing 59% higher. The California-based company supplies the SmartFlow Cannula, the surgical delivery device used to administer AMT-130 directly into the brain. Analysts estimate each infusion will generate roughly $13,500 in revenue for ClearPoint.
B. Riley Securities projected that even treating 25% of the 6,000 initially targetable patients in the U.S. would yield about $20.3 million in revenue for ClearPoint. With 40,000 symptomatic patients nationwide, many of whom remain undiagnosed, the long-term opportunity could be considerably larger.
Shares of ClearPoint are up 69% over the past year, and analysts see further upside as AMT-130 progresses toward a potential U.S. launch in 2026.
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Hercules Capital Strengthens the Financial Foundation
Backing the biotech’s clinical momentum is a new $175 million non-dilutive financing agreement with Hercules Capital, a leading venture lender in the life sciences and technology sectors. The deal refinances existing debt and extends maturities to 2030 while adding $125 million in potential future capital tied to milestones.
Christian Klemt, UniQure’s chief financial officer, emphasized the significance of the deal:
“This non-dilutive financing keeps funds from the outstanding $50 million debt available until 2030 and provides access to an additional $125 million. We believe this transaction enhances our financial flexibility to fund the potential commercial launch of AMT-130 in the United States, anticipated in 2026.”
Later in the afternoon, UniQure also announced a separate proposed $200 million underwritten public offering of its ordinary shares and pre-funded warrants. The move, led by Leerink Partners, Stifel, Van Lanschot Kempen, and Guggenheim Securities, adds another layer of capital-raising as the company prepares for the potential launch of AMT-130.
The financing alleviates investor concerns about UniQure’s balance sheet, providing the runway necessary to pursue regulatory approval and commercialization without heavy dilution.
Analysts Weigh In
Wall Street has wasted no time adjusting its outlook. Leerink Partners boosted its price target from $48 to $68, calling for nearly 50% upside from current levels. Stifel raised its own target to $65 while reiterating a “Buy” rating. Other firms, including HC Wainwright and Chardan Capital, maintained bullish stances with targets as high as $70.
Not everyone, however, believes chasing the stock after its meteoric rally is prudent. Some analysts have urged caution, suggesting new investors may want to wait for a pullback before establishing positions.
Path Forward
Looking ahead, UniQure plans to meet with the FDA later this year before filing a Biologics License Application in early 2026. If approved, AMT-130 could launch in the U.S. later that year. The therapy has already secured Breakthrough Therapy and RMAT designations, which should help expedite the regulatory pathway.
Walid Abi-Saab, UniQure’s chief medical officer, expressed the company’s optimism:
“These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease, while also providing important evidence supporting one-time, precision-delivered gene therapies for the treatment of neurological disorders.”
For now, the spotlight remains on whether UniQure can sustain momentum, both scientifically and financially. But with the stock tripling in a single day, investors have already made their bet that the company’s bold gamble on gene therapy is paying off.
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