The NASDAQ Biotechnology Index (INDEXNASDAQ:NBI) is still trading at three-year highs, despite current market volatility, in response to breakthrough innovations and increased deals involving biotech stocks listed on the NASDAQ.
After dropping to a low of 3,637.05 in October 2023, the index climbed to a nearly three year peak of 4,954.813 on September 19, 2024. While the index had pulled back to 4,243.7 as of March 31, 2025, further growth could be in store in the future.
According to a Towards Healthcare analyst report, the global biotech market is expected to grow at a compound annual growth rate of 12.5 percent from now to 2034, reaching a valuation of US$5.036 trillion.
Driving that growth will be favorable government policies, investment in the sector, increased demand for synthetic biology and a rise in chronic disorders such as cancer, heart disease and hypertension.
The top NASDAQ biotech stocks have seen sizeable share price increases over the past year. For those interested in investing in biotech companies, the best-performing small-cap biotech stocks are outlined below.
Data was gathered on March 31, 2025, using TradingView’s stock screener. Small-cap biotech stocks with market caps between US$50 million and US$500 million at that time were considered for this list.
1. Bright Minds Biosciences (NASDAQ:DRUG)
Year-over-year gain: 2,942.02 percent
Market cap: US$254.99 million
Share price: US$36.20
Bright Minds Biosciences is developing novel treatments for pain and neuropsychiatric disorders such as epilepsy, post-traumatic stress disorder and difficult-to-treat depression.The company’s platform includes serotonin agonists designed to provide powerful therapeutic benefits while minimizing side effects.
Bright Minds is currently in Phase 2 clinical trials for BMB-101, a highly selective 5-HT2C receptor agonist, in adult patients with classic absence epilepsy and developmental epileptic encephalopathy.
Bright Minds’ share price rocketed upward in the fourth quarter of last year, shooting up from US$2.49 to US$38.49 in one day on October 15. The company issued a press release at the time, stating it was ‘unaware of any material changes in the company’s operations’ that would have contributed to such a rally.
The outperformance appears to be related to the October 14 news that Danish pharma company H. Lundbeck was to acquire Longboard Pharma, a company developing a 5-HT2C receptor agonist, for US$60 per share.
A few days later, Bright Minds announced a non-brokered private placement of US$35 million, which sent shares up to US$47.21 on October 18.
That same month, the company shared its collaboration with Firefly Neuroscience (NASDAQ:AIFF) to use Firefly’s Brain Network Analytics technology platform to provide a full analysis of the electroencephalogram data from Bright Minds’ BMB-101 Phase 2 clinical trial. This follows the pair’s previous successful collaboration to analyze data from Bright Minds’ first-in-human Phase 1 study of BMB-101.
In March 2025, Bright Minds expanded its Scientific Advisory Board with the addition of five experts in epilepsy research.
Bright Minds’ share price reached US$55.77, its peak for the past year, on November 6.
2. Monopar Therapeutics (NASDAQ:MNPR)
Year-over-year gain: 924.54 percent
Market cap: US$220.3 million
Share price: US$36.10
Clinical-stage biotech Monopar Therapeutics’ main drug candidate is its late-stage ALXN-1840 for Wilson disease. Its pipeline also includes radiopharma programs such as Phase 1-stage MNPR-101-Zr for imaging advanced cancers, as well as Phase 1a-stage MNPR-101-Lu and late preclinical-stage MNPR-101-Ac225 for the treatment of advanced cancers.
Shares in Monopar spiked by more than 600 percent on October 24, 2024, to US$32.66 following its news release detailing its exclusive worldwide licensing agreement with Alexion, AstraZeneca’s (NASDAQ:AZN) Rare Disease unit, for ALXN-1840, a drug candidate for Wilson disease that met its primary endpoints in its Phase 3 clinical trial. Going forward, Monopar will be responsible for all future global development and commercialization activities.
Further positive news flow in December continued to drive the company’s stock value. Early in the month, the company shared that the first patient was dosed with MNPR-101-Lu in its Phase 1a trial for the radiopharmaceutical. A few weeks later, Monopar announced the launch of a US$40 million concurrent public offering and private placement. After having fallen back to the US$22 range, shares in the company climbed to US$30.68 on December 17, 2024.
Positive sentiment in the company and the biotech market would later drive the stock up to its yearly high of US$51.89 on February 10, 2025. Monopar released its Q4 and full-year 2024 results on March 31.
3. Candel Therapeutics (NASDAQ:CADL)
Year-over-year gain: 268.3 percent
Market cap: US$262.39 million
Share price: US$5.64
Candel Therapeutics is a biotech company focused on developing oncology treatments. The company’s pipeline includes two clinical-stage multimodal biological immunotherapy platforms.
Candel’s lead product candidate, CAN-2409, is in a Phase 2 clinical trial in non-small cell lung cancer and borderline resectable pancreatic cancer, as well as Phase 2 and 3 trials for localized, non-metastatic prostate cancer.
The company had a number wins with the US Food and Drug Administration (FDA) in 2024. In February and May, respectively, Candel’s CAN-3110 received regulatory approval for fast-track designation and orphan drug designation for the treatment of recurrent high-grade glioma.
The agency also granted Candel orphan drug designation for CAN-2409 for the treatment of pancreatic cancer in April 2024. Positive interim data for the trial on pancreatic cancer released that month, sent the company’s share price spiking upward. It ultimately climbed to its 2024 high point of US$14.00 on May 15, 2024.
So far in 2025, Candel’s share price has traded as high as US$12.21 on February 20. In its January corporate update, the company shared its goals for the year, including aiming for Q4 for reporting overall survival data in patients with recurrent high-grade glioma from its ongoing phase 1b trial that is evaluating multiple doses of CAN-3110.
4. Tiziana Life Sciences (NASDAQ:TLSA)
Year-over-year gain: 154.76 percent
Market cap: US$119.51 million
Share price: US$1.08
Tiziana Life Sciences is a clinical-stage biopharma which is developing therapies for autoimmune and inflammatory diseases, degenerative diseases, and cancer-related to the liver. Its pipeline of candidates is built on its patent drug delivery technology that provides a possible alternative to intravenous (IV) delivery. Tiziana’s lead candidate is intranasal foralumab, which it says is the only fully human anti-CD3 mAb currently in clinical development.
On May 31, 2024, shares in Tiziana broke above US$1 after a series of positive news flow for the company. This included positive clinical results from its intermediate sized Expanded Access Program for non-active secondary progressive multiple sclerosis patients, which demonstrated multiple improvements in foralumab-treated patients, as well as its submission of an orphan drug designation application to the FDA for intranasal foralumab for the treatment of non-active secondary progressive multiple sclerosis (na-SPMS).
While Tiazana’s share price slid back down below US$1 per share by mid-June 2024, news that the FDA granted fast track designation to Tiziana intranasal foralumab for the treatment of na-SPMS gave it a much needed boost to the upside. By August 12, the stock’s value had risen to US$1.45 per share.
Tiziana Life Sciences shares reached a yearly peak of US$1.69 on March 7, 2025, after the company filed its investigational new drug application to the FDA for a phase 2 clinical trial in amyotrophic lateral sclerosis (ALS), which is supported by the ALS Association.
5. Benitec Biopharma (NASDAQ:BNTC)
Year-over-year gain: 149.71 percent
Market cap: US$331.43 million
Share price: US$13.01
California-based Benitec Biopharma is advancing novel genetic medicines via its proprietary “Silence and Replace” DNA-directed RNA interference platform. The company is currently focused on developing therapeutics for chronic and life-threatening conditions, including oculopharyngeal muscular dystrophy (OPMD).
Its drug candidate BB-301 was granted orphan drug designation by the FDA and the European Medicines Agency. Benitec is well funded to advance its BB-301 clinical development program through the end of 2025.
Benitec’s share price benefited from its first bump of the past year, after the company released its fiscal year Q3 2024 update in mid-May highlighting its achievements over the quarter. This included the closing of a US$40 million private placement. Benitec’s stock value hit US$10.47 per share on May 20, 2024.
Later in the fall, the company reported positive data from two patients with OPMD treated with low-dose BB-301 in phase 1b/2a study, showing the clinical trial is meeting key safety and efficacy endpoints. Shares hit another high of US$11.22 on October 17, 2024.
Benitec’s share price hit US$16.79, its highest yearly value to date, on March 20, 2025, a day after the company released positive interim clinical results for three patients with OPMD treated with BB-301 in phase 1b/2a study.
“The sixth and final Subject of Cohort 1 will be treated with BB-301 in the second calendar quarter of this year, and we are highly optimistic about the potential for continued benefit in Subjects enrolled in the ongoing clinical study,” said Jerel A. Banks, Benitec Executive Chairman and CEO.
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.
