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. Last year, 55 novel medicines were approved by the US Food and Drug Administration (FDA), compared to 37 such approvals for all of 2022. As of mid-July, 22 novel medicines have received FDA approval in 2024, including Eli Lilly’s (NYSE:LLY) Alzheimer’s disease treatment Kisunla (donanemab-azbt). Big Pharma has largely stole the show throughout the course of this year, but a number of small- and mid-cap NASDAQ pharma stocks also made gains in 2024.
1. Rezolute Bio (NASDAQ:RZLT)
Year-to-date gain: 384.54 percent; market cap: US$241.51 million; share price: US$4.70
First up on this top-performing NASDAQ pharma stocks list is Rezolute Bio, a clinical-stage company that is developing therapeutic candidates geared at treating metabolic and orphan diseases.
The most advanced drug candidate in the company’s portfolio is RZ358, a monoclonal antibody currently in Phase 3 clinical trials for the treatment of congenital hyperinsulinism. RZ358 has been granted an orphan drug designation in both the US and EU, as well as a pediatric rare disease designation in the US.
Rezolute reported results from a preclinical study in March, validating the potential use of RZ358 in the treatment of non-islet cell tumors in individuals with uncontrolled hypoglycemia.
As for Q2, on May 21, the company announced positive topline data from its phase 2 proof-of-concept study of its drug candidate RZ402 for the treatment of patients with diabetic macular edema who have received none or limited anti-vascular growth factor injections.
After gradually trending upward for much of the year on positive news flow, Rezolute’s share price spiked in early June to a year-to-date high of US$5.79. In late June, Rezolute closed on a public offering that garnered US$56.4 million in gross proceeds.
2. Ocugen (NASDAQ:OCGN)
Market cap: US$483.83 million; year-to-date gain: 220.82 percent; share price: US$1.88
Biotechnology firm Ocugen is developing and commercializing novel gene and cell therapies and vaccines with a focus on the treatment of eye, orthopedic and infectious diseases. Its product pipeline includes OCU400, a therapy with a gene-agnostic mechanism of action.
OCU400 is currently in Phase 3 clinical trials for the treatment of retinitis pigmentosa, and in phase 1/2 clinical trials for the treatment of Leber congenital amaurosis. The drug candidate has received orphan drug designations for both genetic eye disease indications.
In late June, Ocugen announced its first patient dosed in its phase 3 trial of OCU400 in retinitis pigmentosa. This followed a flurry of reported first dosages in multiple cohorts so far this year for two of its other clinical programs both in phase 1/2 clinical trials: OCU410 for geographic atrophy, an advanced stage of dry age-related macular degeneration; and OCU410ST for Stargardt disease, the most common form of inherited macular degeneration.
The continuous news flow helped Ocugen’s share price rally from the end of February to reach a year-to-date high of US$1.98 on May 16.
3. Entera Bio (NASDAQ:ENTX)
Year-to-date gain: 201.54 percent; market cap: US$68.05 million; share price: US$1.90
Entera Bio is developing orally delivered peptides and small therapeutic proteins for indications such as osteoporosis, hyperparathyroidism and short bowel syndrome. The company’s proprietary N-Tab technology oral delivery platform prevents the degradation of and enhances the bioavailability of these peptides and therapeutic proteins.
ENTX’s share price reached a year-to-date high on April 12, hitting US$2.98. This rise followed the release of significant news for the company, including the March 20 announcement of positive pharmacokinetic data for GLP-2 peptide tablet treatment for patients with short bowel syndrome. The study is a collaboration that combines OPKO Health’s (NASDAQ:OPK) proprietary long acting GLP-2 agonist with Entera’s N-Tab technology.
The following month, Enterra announced the publication of clinical trial data from its oral PTH 1-34 peptide tablets phase 2 program for osteoporosis in the Journal of Bone and Mineral Research.
4. BeyondSpring (NASDAQ:BYSI)
Year-to-date gain: 168.97 percent; market cap: US$94.3 million; share price: US$2.34
BeyondSpring is developing a pipeline of novel immuno-oncology therapies. The company’s lead drug candidate is Plinabulin, a first-in-class selective immunomodulating microtubule-binding agent with treatment potential for unmet needs across a number of cancer indications.
BeyondSpring’s share price has been boosted this year on advancements in its clinical programs for Plinabulin. The stock hit a year-to-date high of US$3.63 on March 27, two days after the company reported that the first patient had been dosed in its phase 2 trial to evaluate the safety and efficacy of Plinabulin, with Merk’s (NYSE:MRK) Keytruda (pembrolizumab), plus etoposide-platinum for first-line extensive-stage small-cell lung cancer.
The company’s May 16 announcement, which included an update on its majority-owned subsidiary, SEED Therapeutics, added support to BeyondSpring’s share price. SEED has developed proprietary targeted protein degradation (TPD) platforms to develop “molecular glues” for breakthrough therapies targeting oncology, neurodegeneration, immunology and antiviral indications.
SEED is looking to early 2025 to file an investigational new drug (IND) application for its oral RBM39 degrader candidate for cancer indications. Eli Lilly is an investor and collaborator of SEED.
5. Nektar Therapeutics (NASDAQ:NKTR)
Year-to-date gain: 151.73 percent; market cap: US$261.31 million; share price: US$1.42
Last on this list of the year’s top-performing NASDAQ pharma stocks is Nektar Therapeutics, which is developing medicines in the fields of immunotherapy and oncology.
Nektar’s pipeline includes several early and late-stage programs, including its most advanced program, its drug candidate NKTR-255 plus Gilead’s (NASDAQ:GIL) Yescarta or Bristol-Meyer Squibb’s (NYSE:BMY) Breyanzi in phase 2/3 trials targeting large B-cell lymphoma.
Nektar’s share price has trended upward consistently in 2024, rising from a low of US$0.59 in at the start of the year to a year-to-date high of US$1.83 on May 15. The company had a number of important activities in the first quarter. In March 2024, Nektar initiated a phase 2b study of its immunology drug candidate rezpegaldesleukin in patients with severe to very severe alopecia areata. Enrollment was underway in its phase 2b study of the drug in patients with moderate-to-severe atopic dermatitis. Topline data from both studies is expected in the first half of 2025.
In June, Nektar presented the first preclinical data on NKTR-0165, a TNFR2 agonist antibody under development for the treatment of inflammatory diseases. IND-enabling studies are underway for the drug, with first-in-human studies planned in the first half of 2025.
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.