Axsome Therapeutics and NovoCure Limited recently received FDA approvals for their therapies.
MBX Biosciences is expecting late-stage clinical trial readouts this year.
None of these three biotech stocks is profitable yet.
Smaller biotech stocks often fly under the radar. Many of them aren't consistently profitable, but some can deliver outsize growth for investors.
Axsome Therapeutics (NASDAQ: AXSM) is a mid-cap with only three approved therapies, while small-cap stock NovoCure (NASDAQ: NVCR) has approvals in four indications for its Tumor Treating Fields, and small-cap MBX Biosciences (NASDAQ: MBX) has several late-stage therapies in its pipeline. I like their potential and believe the markets haven't fully digested recent positive news for the trio.
Will AI create the world's first trillionaire? Our team just released a report on the one little-known company, called an "Indispensable Monopoly" providing the critical technology Nvidia and Intel both need. Continue »
Here's why I think each of these stocks could soar in 2026.
Image source: Getty Images.
Axsome just got great news that should help its bottom line. On May 1, the U.S. Food and Drug Administration (FDA) approved Axsome's drug Auvelity for a second major indication: Treating agitation associated with Alzheimer's disease. While Auvelity is already used for major depressive disorder (MDD), this new approval targets a condition with very few effective treatments. With a commercial launch set for June, the company is pivoting from a research-and-development-heavy biotech to a multi-product commercial powerhouse.
Much of the Alzheimer's hype focuses on plaque-clearing drugs such as Leqembi. Axsome is tackling the behavioral side of the disease, which is often the primary reason patients are moved into long-term care facilities. This represents a massive, underserved market.
Auvelity was already seeing strong sales growth before the FDA's most recent decision. In 2025, Axsome reported full-year sales of $507.1 million for the drug, up 74%. The company's total revenue was $638.5 million, up 66%. It had an earnings per share (EPS) loss of $3.68, compared to an EPS loss of $5.69 in 2024.
The company's other products also saw double-digit growth. Sunosi, used to treat excessive daytime sleepiness, generated $124.8 million in revenue, up 32%, and migraine therapy Symbravo generated $6.6 million in its first year of sales.
NovoCure uses a proprietary technology called Tumor Treating Fields (TTFs) -- basically, electric fields that disrupt cancer cell division. In February, the FDA approved one of its TTFs, Optune Pax, for the treatment of advanced pancreatic cancer. The therapy provides a non-invasive option alongside chemotherapy. More than 52,000 people will die from pancreatic cancer in 2026, a disease that accounts for 8% of all cancer deaths, according to the National Cancer Institute.
The stock has been volatile due to mixed results in other trials (such as lung cancer), leading some investors to write off the stock. However, the rapid certification of more than 800 healthcare providers immediately following this approval suggests that the clinical demand is far higher than the current market cap reflects.
The company reported that first-quarter revenue climbed 12% year over year to $174 million. This was mainly due to increased sales of Optune Gio, approved as monotherapy for brain metastasis and as combination therapy for glioblastoma, the most common malignant primary brain tumor. It was expected to affect nearly 14,000 people in the U.S. in 2025.
It is important to note that it will be a while before the company is profitable, as it is spending heavily on clinical trials and other R&D. It had a quarterly net loss of $71.1 million with an EPS loss of $0.62, compared to a loss of $34.3 million and an EPS loss of $0.31 in the same period a year ago.
For those looking at the next generation of the obesity and endocrine market, MBX is a quiet contender. The clinical-stage biotech focuses on protein endocrine pipeline (PEP) candidates designed to last longer and work better than current peptide drugs.
Analysts are closely watching for a fourth-quarter readout of MBX's monthly GLP/GIP obesity candidate, MBX 4291. While Eli Lilly and Novo Nordisk dominate the weekly injection market, MBX aims for a once-a-month dosing schedule.
As a more recent entrant to the public markets, it hasn't enjoyed the same meme-stock status as other weight-loss names. However, its de-risked programs in rare endocrine diseases provide a valuation floor, while its obesity trials offer the high-upside moonshot potential.
MBX's pipeline also includes MBX 1416 (Phase 2 for post-bariatric hypoglycemia) and two preclinical obesity therapies.
The company doesn't have any product revenue, but it does have $459.1 million in cash. MBX said that that's enough to fund its operations into 2029.
Small biotech stocks offer high rewards alongside a healthy dose of risk. Ultimately, 2026 is shaping up to be a year of validation for these diverse therapeutic approaches. Between Axsome's pivot toward behavioral neurology, NovoCure's unique electrical disruption of cancer cells, and MBX's pursuit of monthly dosing for metabolic health, the trio represents a high-upside cross-section of modern biotechnology.
As these companies continue to reach clinical readouts and commercial launch dates, the disparity between their current valuations and their market potential offers a compelling window for growth-oriented investors to capitalize on breakthroughs that are just beginning to reach the patients who need them most.
Before you buy stock in Axsome Therapeutics, consider this:
The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Axsome Therapeutics wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years.
Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you’d have $496,473!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you’d have $1,216,605!*
Now, it’s worth noting Stock Advisor’s total average return is 968% — a market-crushing outperformance compared to 202% for the S&P 500. Don't miss the latest top 10 list, available with Stock Advisor, and join an investing community built by individual investors for individual investors.
See the 10 stocks »
*Stock Advisor returns as of May 5, 2026.
James Halley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Axsome Therapeutics and NovoCure. The Motley Fool recommends Novo Nordisk. The Motley Fool has a disclosure policy.