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Three Small-Cap Biotechs That Could Be Tomorrow’s Blockbusters

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February 2, 2025
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Biotech is a battlefield. It’s a world where tiny companies with big ideas take on billion-dollar pharma giants, racing to develop treatments that could change the course of human health. It’s risky, sure—but when these companies win, they win big.

Right now, three small-cap biotech firms are making serious noise: Candel Therapeutics (NASDAQ: CADL), Verastem Oncology (NASDAQ: VSTM), and Arcturus Therapeutics (NASDAQ: ARCT). 

They’re tackling some of the toughest diseases on the planet, and if their bets pay off, they won’t stay “small-cap” for long.

Let’s dive into why these names deserve your attention.

Candel Therapeutics (NASDAQ: CADL): Turning the Immune System into a Cancer-Killing Machine

Candel Therapeutics isn’t just another oncology biotech—these guys are playing a completely different game. Instead of the usual chemotherapy and targeted drugs, they’re developing viral immunotherapies, which essentially turn the immune system into a guided missile against cancer.

Their lead candidate, CAN-2409, is a gene-mediated immunotherapy. Here’s the simple version: this drug programs your body to recognize and attack cancer like it’s an infection. That’s huge because most cancers slip under the radar of the immune system.

And here’s where it gets really interesting: their Phase 3 trial in prostate cancer was a massive success. Patients who got CAN-2409 alongside standard radiation therapy saw a 14.5% improvement in disease-free survival over 54 months. That’s almost five years of extra time without their cancer coming back.

If that wasn’t impressive enough, their Phase 2 trial in non-small cell lung cancer showed that CAN-2409 extended median survival to 20.6 months—a huge deal for patients whose disease had already progressed.

The stock price? Skyrocketed 200% after the Phase 3 data dropped. Investors took notice, and Candel is now moving toward FDA approval. If they get it, expect this stock to go ballistic.

But there’s more: they’re testing CAN-2409 in pancreatic cancer, one of the deadliest cancers with almost no good treatment options. If it works there, Candel won’t just be another biotech success story—they’ll be a household name in oncology.

Verastem Oncology (NASDAQ: VSTM): Finally Cracking the Code on RAS/MAPK Cancers

If you follow cancer research, you’ve probably heard of the RAS/MAPK pathway—it’s like a highway for tumor growth, and for years, researchers have been trying (and mostly failing) to shut it down.

Well, Verastem Oncology thinks they’ve cracked it. Their drug, VS-6766, is a dual RAF/MEK inhibitor, designed to cut off the RAS/MAPK pathway at the source. And it’s showing real promise.

One of the biggest wins? Their Phase 2 trial in low-grade serous ovarian cancer (LGSOC), an aggressive form of ovarian cancer that’s notorious for resisting treatment. Patients on VS-6766 saw their tumors shrink significantly, which was enough for Verastem to push forward into a Phase 3 trial.

But they’re not stopping there. The RAS/MAPK pathway is involved in lung cancer, pancreatic cancer, and colorectal cancer—some of the biggest killers in oncology. If Verastem’s drug proves effective across these cancers, we’re looking at a biotech juggernaut in the making.

And here’s the kicker: they’re already gearing up for an accelerated FDA approval application. If that gets the green light, they’ll be launching a first-in-class therapy in one of the most challenging areas of cancer treatment.

Stock price? Sitting at $6.08, with a market cap of $298 million. But if this drug delivers in Phase 3, those numbers could explode.

Arcturus Therapeutics (NASDAQ: ARCT): The Next Evolution of mRNA Medicine

Everyone knows mRNA because of COVID-19 vaccines. Moderna and BioNTech made billions off the technology, but what if mRNA wasn’t just for vaccines? What if it could be used for rare diseases, genetic disorders, and long-term treatments?

That’s exactly what Arcturus Therapeutics is doing. Unlike its bigger rivals, Arcturus is focusing on self-amplifying mRNA (sa-mRNA)—an upgraded version that requires lower doses but lasts longer in the body. That means more effective treatments with fewer side effects.

Their pipeline includes:

  • Liver and respiratory disease treatments using their self-amplifying mRNA tech.
  • New vaccine candidates for infectious diseases.
  • A partnership with CSL Behring, a biotech giant, to develop next-gen RNA therapies.

And they’re not just talking big. Arcturus was invited to present at the Guggenheim Securities SMID Cap Biotech Conference, a sign that Wall Street is watching them closely.

Current stock price? $16.97, with a market cap of $456 million. But if they deliver on the promise of next-gen mRNA, they could be the next Moderna—and that’s not an exaggeration.


Biotech investing is not for the faint of heart—but it’s also where some of the biggest opportunities lie. Candel is revolutionizing cancer immunotherapy, Verastem is finally tackling RAS/MAPK cancers, and Arcturus is pushing mRNA technology beyond COVID vaccines.

Any one of these companies could break out in a massive way over the next few years. They’re all in high-risk, high-reward territory—but that’s what makes small-cap biotech so exciting. When these companies win, they win big.

So, will these be the next biotech giants? Only time will tell. But one thing is for sure: they’re making moves that can’t be ignored.

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