Small biotech companies often fly under the market’s radar, with many yet to achieve consistent profitability. However, individual firms can still deliver exceptional growth for investors. Axsome Therapeutics (AXSM) is a mid-cap company that currently has only three approved therapies; small-cap NovoCure (NVCR) has received approval for its tumor treating fields technology in four indications; and another small-cap, MBX Biosciences (MBX), boasts multiple late-stage therapies in its pipeline. The market has yet to fully price in the recent positive news from these three companies.
On May 1, Axsome received FDA approval for its drug Auvelity for a second major indication—the treatment of agitation associated with Alzheimer’s disease. The drug was previously approved for major depressive disorder, and this new indication addresses an area with few effective treatment options. The drug is scheduled for commercial launch in June, as the company transitions from a research-and-development-focused biotech into a multi-product commercial powerhouse. Most of the spotlight on Alzheimer’s disease has centered on drugs that clear beta-amyloid, but Axsome is tackling the behavioral aspects of the disease—often the primary reason patients are moved into long-term care facilities. In 2025, Auvelity generated full-year sales of $507.1 million, a 74% year-over-year increase, while the company’s total revenue reached $638.5 million, up 66%. The company’s other products also achieved double-digit growth.
NovoCure holds a patented technology called tumor treating fields, which disrupts cancer cell division using electric fields. In February, the FDA approved its Optune Lua system for the treatment of advanced pancreatic cancer. The therapy, used in combination with chemotherapy, offers a non-invasive option. The stock had been volatile due to mixed results from other trials, but following this approval, it quickly received certification from over 800 medical institutions, indicating that clinical demand is far greater than what current market capitalization reflects. The company reported first-quarter revenue of $174 million, a 12% year-over-year increase, driven primarily by sales growth of Optune Gio. It is worth noting that profitability remains some distance away, with a net loss of $71.1 million in the first quarter.
For investors focused on next-generation products in the obesity and endocrinology markets, MBX is a quiet contender. This clinical-stage biotech specializes in protein endocrine drug candidates designed to last longer and work better than existing peptide-based drugs. Analysts are closely watching the fourth-quarter data readout for MBX 4291, its once-monthly GLP/GIP obesity candidate. While Lilly and Novo Nordisk dominate the weekly injection market, MBX is targeting once-monthly dosing. The company’s de-risked programs in rare endocrine diseases provide a valuation floor, while its obesity trial offers high-reward potential. The company has no product revenue yet, but holds $459.1 million in cash, sufficient to fund operations through 2029.
2026 is shaping up to be a year of validation for these three companies across different therapeutic approaches. As they successively reach clinical data readouts and commercial launch milestones, the gap between current valuations and market potential presents a window for growth investors to capture breakthrough progress.
