Roll the dice baby, ‘cause I’m feeling lucky! 

Yesterday marked seven days in a row that the Nasdaq and S&P 500 closed in the green with more ATH’s on the scorecard. Whoo hoo! 

(Source: Giphy) 

Jerome Powell has no doubt led the spark the last few days with his subtle hints and innuendo’s of possible rate cuts causing everyone to once again pile into big tech like there’s no tomorrow… 

(Source: Contrarian Investor) 

However, while most investors are ogling over their portfolio numbers this morning, tomorrow’s inflation data may end up telling another story. Will this market rally prove to be the calm before the storm? 

Only time will tell, but as we prepare for this morning’s opening bell, there’s one big story that’s definitely turning our heads with some swoon this AM. 

(Source: Wired) 

In the midst of tech stocks popping like popcorn yesterday, one little known company stole some of the spotlight with a nice 76.5% gain (and still rising as it’s up 152.37% over the last five days). 

(Source: Yahoo Finance) 

uniQure, a biotech company that most people couldn’t spell until yesterday, has garnered widespread media attention for their groundbreaking gene therapy, AMT-130 - as they officially received FDA approval for Regenerative Medicine Advanced Therapy (RMAT) in Huntington’s Disease due to their most recent mind-blowing Phase 2 data. 

(Source: BioSpace) 

Now for those who skipped biology class, Huntington’s Disease is a nasty neurodegenerative condition that’s been giving scientists sleepless nights for years. It’s a nasty, torturous, and horrific disease that causes the progressive breakdown of nerve cells in the brain, leading to physical and mental deterioration. 

Which is why uniQure ticker “QURE” (awesome ticker symbol btw) decided to take it head-on with their one-time gene therapy treatment, and boy, did it pay off.

(Source: Giphy) 

Details please…

In their latest Phase I/II trial, AMT-130 didn’t just slow the progression of Huntington’s Disease—it slammed the brakes. Patients who received the higher dose of AMT-130 saw an 80% slowdown in disease progression compared to a control group. While the lower dose wasn’t too shabby either, showing a 30% slowdown, but let's be real, 80% is where the party's at.

(Source: NeurologyLive) 

The data revealed that AMT-130 significantly reduced the levels of mutant huntingtin protein (mHTT), which is the primary culprit in Huntington’s Disease. This reduction was observed in both the lower and higher dose groups, but the higher dose group, in particular, showed a more pronounced effect, hinting at a dose-dependent response.

Moreover, the therapy led to an 11% drop in neurofilament light (NfL) levels—a marker for brain damage—compared to the control group. Both doses managed to lower NfL levels below baseline, making AMT-130 look like the MVP in the Huntington’s Disease league. Lower NfL levels indicate less ongoing neuronal damage, which is a crucial marker for the disease's progression.

(Source: Giphy) 

Now if you're a big numbers person, you’ll appreciate this. The higher dose had a p-value of 0.007, which in layman's terms means it wasn’t a fluke. 

For a clinical trial, a p-value less than 0.05 generally indicates that the results are statistically significant. In this case, the lower the p-value, the higher the confidence that the observed effects are real and not due to random chance. 

uniQure execs right now (Source: Giphy) 

Of course, this alone is extremely promising, but what about the therapy’s safety? 

Well uniQure reported that AMT-130 was generally well-tolerated. No new scary side effects popped up, which is always a plus when you’re talking about gene therapy. 

The safety profile was "manageable," according to the folks at uniQure, which is corporate speak for "no one's heads exploded." Common side effects included mild to moderate headaches and transient increases in liver enzymes, but these were manageable and resolved over time. So pretty much the same kind of precautions we hear in our favorite Big Pharma commercials… 

(Source: Giphy) 

Which is why investors are finally waking up to uniQure’s potential. After some lukewarm reactions to previous data releases, this latest update has them doing backflips. 

Analyst Paul Matteis from Stifel gave a thumbs up, noting the “broadly favorable” results against a backdrop of low expectations. 

(Source: BioPharmaDive) 

Now keep in mind, the biotech sector is notoriously volatile, with stock prices often swinging wildly on the back of clinical trial results. However, a 152.37% surge over the past few days is more than significant even by biotech standards. So there’s no doubt a massive vote of confidence from the market, suggesting that the mass sentiment believes uniQure is on the cusp of a breakthrough.

So given that uniQure has just blown everyone’s socks off, what’s next? 

(Source: Giphy) 

Well the biotech company isn’t slowing down. They’re planning to wrap up enrollment for the third cohort of their Phase I/II study, which will look at AMT-130 combined with immunosuppression. 

Initial safety results are expected in the first half of 2025, and another interim analysis is slated for mid-2025. If all goes well, we might see AMT-130 getting fast-tracked for approval.

Plus the RMAT designation uniQure has received from the FDA is not just a gold star; it offers several benefits, including more frequent interactions with the FDA and eligibility for priority review. 

(Source: Euro Huntington) 

Meaning that if the upcoming data continues to be positive, AMT-130 could reach the market much faster than typical treatments. Which is like music to the ears for companies like uniQure. 

(Source: Giphy) 

So with all of this good news this morning, what’s the takeaway friends? 

It’s simple: While big tech continues to hog the spotlight, uniQure is quietly making waves in the biotech world. 

(Source: Google Finance) 

Their stock’s meteoric rise is a testament to the potential game-changing impact of AMT-130 and highlights the growing importance and potential of gene therapies in treating previously untreatable conditions like ALS or even Parkinson’s Disease. Which is definitely something to look for as we continue to progress in technology and of course science… 

So in addition to practicing how you spell “uniQure” today, I’d definitely keep it on your watchlist as the trading session unfolds.

(Source: Giphy) 

A stock with a massive medical breakthrough like this combined with the continued momentum that uniQure has experienced, signify's more room to climb…

And given how explosive the stock popped, there may still be room for it to run even further. So with that said, check it out and run the numbers, because this opportunity is nothing short than exciting. Until next time friends… 

Stocks.News doesn't have any positions in companies mentioned in the article.