Today’s pharmaceutical market is facing the challenges of inflation, government-imposed drug price caps and waning demand for COVID-19 vaccines. However, the industry’s major underlying drivers — higher rates of cancer and chronic diseases — are still at play.
The US reigns supreme in the pharma market, both in terms of drug demand and development. In 2024, 50 novel medicines were approved by the US Food and Drug Administration (FDA), compared to 55 such approvals in 2023. Last year’s FDA approvals for pharmaceuticals included Eli Lilly and Company’s (NYSE:LLY) Alzheimer’s disease treatment Kisunla (donanemab-azbt).
Big pharma largely stole the show throughout the course of the past year, but a number of small- and mid-cap NASDAQ pharma stocks have also made gains.
1. Chimerix (NASDAQ:CMRX)
Year-over-year gain: 239.49 percent
Market cap: US$297.69 million
Share price: US$3.31
Chimerix is a clinical-stage company developing medicines to improve the quality of life for patients facing deadly diseases. Its most advanced drug development program is ONC201 (dordaviprone), which is in development for recurrent H3 K27M-mutant glioma, a lethal form of brain cancer.
After trading mostly sideways for much of the past year, shares of Chimerix received a big boost late in the fourth quarter of 2024 as the company reached important milestones in its drug development program. The stock jumped more than 217 percent on December 10 to US$2.76, one day after Chimerix announced it would submit a New Drug Application for accelerated approval of dordaviprone to the FDA before the end of the year.
The company’s stock price continued to gain momentum in the following weeks to push past the US$3 mark by December 23. The day following Chimerix’ December 30 press release confirming it had completed the submission process, the stock reached US$3.48 per share.
“With this submission, we now turn our attention to preparing for potential commercial launch in the U.S. next year,” stated Chimerix CEO Mike Andriole.
Chimerix shares hit a yearly high of US$3.66 on January 7, 2025.
2. Eton Pharmaceuticals (NASDAQ:ETON)
Year-over-year gain: 195.98 percent
Market cap: US$372.89 million
Share price: US$14.00
Eton Pharmaceuticals is developing and commercializing treatments for ultra-rare diseases. Its commercial portfolio of rare disease products includes Alkindi Sprinkle, Increlex, PKU Golike and multiple FDA-approved generic bioequivalents. The company also has several product candidates in late-stage development: hydrocortisone oral solution ET-400, ET-600 for diabetes insipidus and the ZENEO hydrocortisone autoinjector.
Eton’s share price performed exceptionally well in the second half of 2024 and into the first few weeks of 2025 on robust quarterly financials, acquisitions and key milestones.
The stock made steady gains following the release of the company’s Q2 2024 financial report in early August. The quarter brought royalty revenue of US$9.1 million and a 40 percent increase in product sales over Q2 2023, representing “the 14th straight quarter of sequential product sales growth.” Shares in Eton climbed by more than 65 percent to US$6.00 by the end of Q3.
In early October, the company announced the acquisition of Increlex, a medication used in the treatment of pediatric patients with severe IGF-1 deficiency, from French biopharma company Ispen. By the end of the month, Eton’s stock reached a value of US$8.62 per share.
November was a busy month for positive news flow out of Eton. The company was awarded a second patent for its liquid formulation of hydrocortisone on November 7. A few days later, its Q3 2024 report highlighted another consecutive quarter of growth in product sales, up 40 percent year-over-year. Eton closed out the month with the acquisition of the US rights to Amglidia for the treatment of neonatal diabetes mellitus from French biotech firm AMMTeK.
By the end of November, Eton’s stock price had surged to US$13.53 per share. The stock reached its highest yearly value of US$14.31 on January 2, 2025. The next day, Eton announced the acquisition of Galzin, an FDA-approved treatment for patients with Wilson disease, which it plans to begin commercializing in the US early this year.
3. Corvus Pharmaceuticals (NASDAQ:CRVS)
Year-over-year gain: 139.63 percent
Market cap: US$334.14 million
Share price: US$5.20
Corvus Pharmaceuticals is a clinical-stage biopharma company developing an immunotherapy platform based on ITK inhibition for the treatment of various cancer and immune diseases. The company’s lead product candidate is soquelitinib, an investigational small molecule drug that selectively inhibits ITK and is delivered orally.
Corvus is another NASDAQ pharma stock that saw significant gains in the last half of 2024.
The growth in its share price got its first major boost in early August when the FDA granted fast track designation to soquelitinib ‘for the treatment of adult patients with relapsed or refractory peripheral T cell lymphoma after at least two lines of systemic therapy.’ By the end of the month, shares in Corvus had grown by nearly 50 percent to US$4.48.
Corvus’ stock value received another bump to the upside following the September 10 announcement it had initiated registration in its Phase 3 clinical trial of soquelitinib for the aforementioned indication. Shares in the company reached what was then their highest point of US$5.91 on September 20, and continued to gain value throughout the following weeks to hit a current yearly high of US$9.56 on November 11.
4. ATyr Pharma (NASDAQ:ATYR)
Year-over-year gain: 110.9 percent
Market cap: US$276.17 million
Share price: US$3.29
ATyr Pharma is using its proprietary tRNA synthetase platform, which includes a library of domains derived from all 20 tRNA synthetases, to develop new therapies for fibrosis and inflammation. The company’s lead therapeutic candidate is efzofitimod, a first-in-class biologic immunomodulator targeting interstitial lung disease.
The fourth quarter of 2024 was very good to aTyr’s stock value, and it has continued to perform well into January 2025.
In early October, aTyr Pharma announced the publication of an analysis of the Phase 1b/2a clinical trial of efzofitimod in patients with pulmonary sarcoidosis, a major form of interstitial lung disease, in the European Respiratory Journal.
Shares in aTyr Pharma climbed by more than 92 percent through the month to a then yearly high of US$3.35 on October 22.
On December 10, the company shared its third positive safety review of its ongoing Phase 3 EFZO-FIT study of efzofitimod in patients with pulmonary sarcoidosis. Shares of the company hit their highest yearly value of US$3.98 on January 3.
5. Inhibikase Therapeutics (NASDAQ:IKT)
Year-over-year gain: 90 percent
Market cap: US$178.73 million
Share price: US$2.66
Inhibikase Therapeutics is developing protein kinase inhibitor therapeutics for modifying the course of cardiopulmonary and neurodegenerative disease through Abl kinase inhibition. Its two leading drug candidates are IkT-001Pro, a prodrug of imatinib mesylate, for pulmonary arterial hypertension with fewer on-dosing side-effects; and risvodetinib, a selective c-Abl inhibitor to treat Parkinson’s and Parkinson’s-related disease.
In late October, Inhibikase closed on an approximately US$110 million private placement, which with the full cash exercise of accompanying warrants could lead to a potential aggregate financing of up to approximately US$275 million before deducting fees and expenses. The company intends to use the funds in part for its Phase 2b 702 trial for IkT-001Pro in pulmonary arterial hypertension.
Shares of Inhibikase reached a yearly high of US$3.97 on December 17.
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.