Top 5 NASDAQ Pharma Stocks (Updated December 2023)

Market cap: US$43.29 million; year-to-date gain: 286.88 percent; current share price: US$6.19

First up on this top-performing NASDAQ pharma stocks list is Minerva Neurosciences, a clinical-stage company that is developing therapeutic candidates geared at treating central nervous system diseases. The company's portfolio includes roluperidone for negative symptoms of schizophrenia, and MIN-301 for Parkinson’s disease.

Minerva's share price spiked in mid-July to a year-to-date of high of US$13.22 following the completion of a US$20 million equity financing that improved its cash position. As of December 5, the company's share price had fallen off that high by about 50 percent, but it was still up more than 286 percent from the start of 2023.

Market cap: US$240.87 million; year-to-date gain: 235.04 percent; current share price: US$3.90

Clinical-stage Inozyme Pharma is developing novel therapeutics for the treatment of rare diseases impacting the vasculature, soft tissue and skeleton. Its product pipeline inclues INZ-701, an enzyme replacement therapy.

INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 deficiency associated with Generalized Arterial Calcification of Infancy, as well as ABCC6 deficiency, which causes progressive calcification in the eyes, skin and arteries. Positive preliminary data released in April, alongside strong first quarter financials shared in May, helped Inozyme's share price rally from the beginning of April to reach a year-to-date high of US$7.33 on July 21.

Market cap: US$78.34 million; year-to-date gain: 235.23 percent; current share price: US$5.88

CASI Pharmaceuticals is acquiring, developing and commercializing pharmaceutical products and therapeutics with a focus on hematology oncology, as well as other areas of unmet medical need. Through its wholly owned subsidiary, CASI Pharmaceuticals (China), the company is working to launch medicines in the Chinese market.

After trading flat through much of the year, CASI's share price reached a year-to-date high on December 12, hitting US$8.48. This rise followed the November announcement of a major milestone — CASI's partner Juventas Cell Therapy received market approval from the China National Medical Products Administration for its investigational cell therapy, CNCT 19 for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia.

Market cap: US$193.07 million; year-to-date gain: 221.83 percent; current share price: US$2.41

Applied Therapeutics is developing a pipeline of novel drug candidates in areas of high unmet medical need. The company’s lead drug candidate is govorestat, a novel central nervous system penetrant aldose reductase inhibitor for the treatment of central nervous system rare metabolic diseases, including galactosemia, sorbitol dehydrogenase (SORD) deficiency and PMM2-CDG. Its pipeline also includes treatments for diabetic cardiomyopathy and diabetic retinopathy.

Applied Therapeutics reached a number of milestones this year, and they steadily pushed its share price from US$0.70 at the start of the year to a year-to-date high of US$3.42 on December 12.

In late April, the company presented results from its ACTION-Galactosemia Kids study of govorestat, saying that it "demonstrated compelling evidence of clinical benefit alongside a favorable safety profile." Applied Therapeutics also announced plans to request a pre-new drug application (NDA) meeting with the FDA, and to submit a marketing authorization application (MAA) to the European Medicines Agency (EMA). Soon after, Applied Therapeutics strengthened its balance sheet with a US$30 million private equity financing.

In May, Applied Therapeutics' govorestat was granted orphan medicinal product designation by the EMA for treatment of SORD deficiency. The company announced in November that was on track to submit an NDA to the FDA and an MAA to the EMA for govorestat for the treatment of classic falactosemia in Q4 2023.

Market cap: US$158.91 million; year-to-date gain: 167.6 percent; current share price: US$2.39

Last on this list of the year's top-performing NASDAQ pharma stocks is Aquestive Therapeutics, which is developing orally administered products as non-invasive alternatives to current standard therapies.

Aquestive's portfolio includes: five commercialized products manufactured by the company and marketed by its licensees in the US and around the world; late-stage proprietary products focused on treating diseases of the central nervous system; and earlier-stage products for the treatment of severe allergic reactions, including anaphylaxis.

Aquestive's share price has experienced volatility in 2023, rising from a low of US$0.72 in mid-March to a year-to-date high of US$2.69 on May 23. In late March, Aquestive expanded its license and supply agreement with Pharmanovia for Libervant (diazepam) buccal film to additional global markets. In late May, the company reported positive results from its latest clinical studies evaluating the pharmacokinetic and pharmacodynamic performance of Anaphylm (epinephrine) sublingual film.

In other positive news, Aquestive received FDA acceptance of its NDA for Libervant (diazepam) buccal film for the treatment of seizure clusters in patients between two and five years of age.