Top 5 NASDAQ Genetics Stocks of 2023

Year-to-date gain: 316.74 percent; market cap: US$764.12 million; current share price: US$12.10

Through its expanded genetic code technology platform, clinical-stage biopharma company Ambrx Biopharma is discovering and developing engineered precision therapies. Its portfolio of advanced clinical and preclinical programs is designed to improve efficacy and safety in the treatment of a number of cancer indications, such as prostate and breast cancer.

The US Food and Drug Administration (FDA) has granted fast-track designation for ARX788 in HER2-positive metastatic breast cancer, and orphan drug designation for ARX788 in gastric cancer, while China’s National Medical Products Administration has given ARX788 breakthrough therapy designations in breast cancer. The FDA has also granted fast-track designation to Ambrx’s proprietary anti-PSMA antibody-drug conjugate investigational therapy, ARX517, for the treatment of patients with metastatic castration-resistant prostate cancer upon progression on an androgen receptor pathway inhibitor.

Year-to-date gain: 336.96 percent; market cap: US$737.27 million; current share price: US$16.22

Global gene therapy leader Orchard Therapeutics is focused on discovering, developing and commercializing new treatments for genetic and other severe diseases. The company’s hematopoietic stem cell (HSC) gene therapy platform uses a patient’s own blood stem cells, which are genetically modified outside of the body and then reinserted. The aim is to address the underlying cause of disease in a single treatment.

In November, the FDA granted fast-track designation to Orchard’s OTL-203, an investigational HSC therapy under development for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I. This is a rare, inherited neurometabolic disease caused by a deficiency of the enzymes that break down molecules such as sugars, leading to the accumulation of complex sugars in organs, including the eyes, ears and heart, as well as the musculoskeletal and central nervous systems.

The company will be initiating a global registrational trial in December to evaluate the efficacy and safety of OTL-203. This treatment candidate has received a rare pediatric disease designation from the FDA and priority medicine designations from the European Medicines Agency.

Year-to-date gain: 191.98 percent; market cap: US$230.52 million; current share price: US$7.05

Reneo Pharmaceuticals is a clinical-stage company working to develop and commercialize therapies for patients with rare genetic mitochondrial diseases. Its lead product candidate is mavodelpar, which has been shown to increase the transcription of genes involved in mitochondrial function and increase fatty acid oxidation; it may also increase the production of new mitochondria.

In Q3, Reneo completed the last patient visit for its STRIDE study of mavodelpar in primary mitochondrial myopathies. The company expects to put out top-line data in the fourth quarter, and in 2024 will submit that data, along with long-term safety information, to the FDA.

Year-to-date gain: 172.27 percent; market cap: US$62.36 million; current share price: US$3.30

Tempest Therapeutics is a clinical-stage oncology company advancing small-molecule therapeutics that modulate anti-tumor immunity pathways with the potential to treat a wide range of tumors. The company’s portfolio consists of clinical programs in various stages, from early research to investigational global studies.

Tempest had a number of critical catalysts in 2023, including the release of data from a first-line randomized study with Roche Holding (OTCQX:RHHBF,SWX:ROG) for its drug candidate TPST-1120, which is targeting hepatocellular carcinoma. The company also shared Phase 1 clinical data for its TPST-1495 EP2/4 prostaglandin receptor antagonist, which is targeting multiple types of solid tumors.

Year-to-date gain: 124.32 percent; market cap: US$675.2 million; current share price: US$18.91

Clinical-stage biopharma company Biomea Fusion’s proprietary FUSION System has allowed it to discover, develop and advance a pipeline of covalent-binding therapeutic agents targeting genetically defined cancers and metabolic diseases.

Its lead investigational drug candidate, BMF-219, is a covalent menin inhibitor with three ongoing clinical trials: a Phase 1 study for its use in treating various leukemias, large B-cell lymphoma and multiple myeloma; a Phase 1/1b study for use in treating non-small cell lung cancer, colorectal cancer and pancreatic ductal adenocarcinoma; and a Phase 1/2 study for type 2 diabetes.

Orchard's FDA investigational new drug application cleared in October for a Phase 2 clinical trial of BMF-219 in type 1 diabetes, and enrollment is anticipated to begin in Q4 2023. In early December, Health Canada also cleared the initiation of a Phase 2 clinical trial of BMF-219 for type 1 diabetes.