By 2023 presented challenges for the pharmaceutical market, such as rising inflation to government-led drug price cap negotiations, and waning demand for COVID-19 vaccines. However, the industry’s major underlying drivers — such as the raising rates of cancer and chronic diseases — are still driving innovation in this sector.
The US reigns supreme in the pharma market, both in terms of drug demand and development. As of December 14, 2023, 53 novel medicines had been approved by the US Food and Drug Administration (FDA) for the year, compared to 37 such approvals for all of 2022. Big Pharma largely stole the show throughout the course of the year, but a number of small- and mid-cap NASDAQ pharma stocks also made gains in 2023.
1. Minerva Neurosciences (NASDAQ:NERV)
Market cap: US$43.289 million; year-to-date gain: 286.88 percent; current share price: US$6.19
First up on our top-performing NASDAQ pharma stocks list is Minerva Neurosciences, a clinical-stage company developing therapeutic candidates to treat central nervous system diseases. The company’s portfolio includes roluperidone for negative symptoms of schizophrenia, and MIN-301 for Parkinson’s disease.
Minerva saw its share price spike in mid-July to a year-to-date of high of US$13.22, following the completion of a US$20 million equity financing, improving its cash position. Although as of December 5, the company’s share price has fallen off that high by about 50 percent, it’s still up more than 286 percent from the start of 2023.
2. Inozyme Pharma (NASDAQ:INZY)
Market cap: US$240.867 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. It’s 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 and ABCC6 Deficiency which causes progressive calcification in the eyes, skin and arteries. Positive preliminary data released in the second quarter of 2023 alongside strong financials led Inozyme’s share price to rally beginning in April to reach a year-to-date high of US$7.33 on July 21, 2023.
3. Casi Pharmaceuticals (NASDAQ:CASI)
Market cap: US$78.344 million; year-to-date gain: 235.23 percent; current share price: US$5.88
Next on our list of top performing NASDAQ pharma stocks is Casi Pharmaceuticals which is acquiring, developing and commercializing therapeutics and pharmaceutical products with a focus on hematology oncology as well as other areas of unmet medical need. Through its wholly owned subsidiary, CASI Pharmaceuticals (China) Co., Ltd., the company is working to launch medicines in the Chinese market.
After trading flat through much of the year, Casi’s stock reached its year-to-date high on December 12 at a price of US$8.48 per share. This followed the November announcement of a major milestone in the market approval of its partner Juventas Cell Therapy’s investigational cell therapy, CNCT 19 for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia by the China National Medical Products Administration (NMPA).
4. Applied Therapeutics (NASDAQ:APLT)
Market cap: US$193.073 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 indications 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. The company’s product pipeline also includes treatments for diabetic cardiomyopathy, and diabetic retinopathy.
Applied Therapeutics reached a number of milestones this year that steadily grew its share price from US$0.70 at the start of the year to a year-to-date high of US$3.42 per share on December 12.
In late April, the company presented results from its ACTION-Galactosemia Kids study of govorestat, ‘demonstrated compelling evidence of clinical benefit alongside a favorable safety profile’; and announced plans to request a pre-new drug application (NDA) meeting with the FDA and to submit a Marketing Authorization Application (MAA) with 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 is 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.
5. Aquestive Therapeutics (NASDAQ:AQST)
Market cap: US$158.906 million; year-to-date gain: 167.6 percent; current share price: US$2.39
Last on our list of top performing NASDAQ pharma stocks is Aquestive Therapeutics is developing orally administered products as non-invasive alternatives to current standard of care therapies. Aquestive’s portfolio includes five commercialized products manufactured by the company and marketed by its licensees in the U.S. and around the world; a late-stage proprietary products focused on treating CNS diseases of the central nervous system; and an earlier-stage products for the treatment of severe allergic reactions, including anaphylaxis.
Aquestive’s share price has experienced a year of volatility in 2023, rising from a low of US$0.72 per share in mid-March to its year-to-date high of US$2.69 on May 23. In late March, Aquestive expanded its license and supply agreement 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 good news, Aquestive received FDA acceptanceFDA acceptance of its Libervant (diazepam) buccal film NDA for treatment of seizure clusters in patients between two and five years of age.
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.