U.S. Pharmaceuticals & Biotech High Growth Potential Companies

Axsome Therapeutics

Market Cap: US$13.1b

A biopharmaceutical company, develops and delivers novel therapies for the management of central nervous system (CNS) disorders in the United States.

Madrigal Pharmaceuticals

Market Cap: US$11.1b

A biopharmaceutical company, focuses on delivering novel therapeutics for metabolic dysfunction-associated steatohepatitis (MASH) in the United States.

Travere Therapeutics

Market Cap: US$4.8b

A biopharmaceutical company, identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases in the United States.

Corcept Therapeutics

Market Cap: US$8.9b

A biopharmaceutical company, engages in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic, and neurologic disorders in the United States.

Syndax Pharmaceuticals

Market Cap: US$1.6b

A commercial-stage biopharmaceutical company, develops therapies for the treatment of cancer.

Ardelyx

Market Cap: US$1.4b

Ardelyx, Inc. discovers, develops, and commercializes medicines to treat unmet medical needs in the United States and internationally.

Krystal Biotech

Market Cap: US$9.4b

A commercial-stage biotechnology company, discovers, develops, manufactures, and commercializes genetic medicines to treat diseases with high unmet medical needs in the United States.

Marker Therapeutics

Market Cap: US$23.0m

A clinical-stage immuno-oncology company, develops and commercializes T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications in the United States.

Ionis Pharmaceuticals

Market Cap: US$12.1b

A commercial-stage biotechnology company, provides RNA-targeted medicines in the United States.

Clene

Market Cap: US$74.6m

A clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel clean-surfaced nanotechnology (CSN) therapeutics.

Forum Markets

Market Cap: US$77.8m

A financial technology company, develops a blockchain-based platform focused on the acquisition, structuring, and tokenization of real-world assets worldwide.

AVITA Medical

Market Cap: US$120.3m

Operates as a therapeutic acute wound care company in the United States, Japan, the European Union, Australia, and the United Kingdom.

Pelthos Therapeutics

Market Cap: US$95.6m

A bio-pharmaceutical company, develops and commercializes therapeutic products to help patients with unmet treatment burdens.

Lineage Cell Therapeutics

Market Cap: US$316.6m

A clinical-stage biotechnology company, developing cell replacement therapies to treat serious medical conditions in the United States and internationally.

Royalty Pharma

Market Cap: US$31.6b

Operates as a buyer of biopharmaceutical royalties and a funder of innovation in the biopharmaceutical industry in the United States.

Zevra Therapeutics

Market Cap: US$747.8m

A commercial-stage company, focuses on addressing unmet needs for the treatment of rare diseases in the United States.

Capricor Therapeutics

Market Cap: US$1.5b

A clinical-stage biotechnology company, engages in the development of transformative cell and exosome-based therapeutics for treating duchenne muscular dystrophy (DMD) and other diseases with unmet medical needs in the United States.

Tenaya Therapeutics

Market Cap: US$154.5m

A clinical-stage biotechnology company, discovers, develops, and delivers therapies for heart disease in the United States.

Intellia Therapeutics

Market Cap: US$1.7b

Operates as a clinical-stage genome editing company focused on developing potentially curative therapeutics using CRISPR/Cas9-based technologies.

OS Therapies

Market Cap: US$75.7m

A clinical stage biopharmaceutical company, focuses on the identification, development, and commercialization of treatments for osteosarcoma and other solid tumors in the United States.

Achieve Life Sciences

Market Cap: US$566.7m

A late-stage pharmaceutical company, develops and commercializes cytisinicline for nicotine independence in Canada, the United States, and the United Kingdom.

UroGen Pharma

Market Cap: US$1.4b

A biotechnology company, engages in the development and commercialization of solutions for urothelial and specialty cancers.

ARS Pharmaceuticals

Market Cap: US$969.2m

A biopharmaceutical company, develops and commercializes treatments for severe allergic reactions in the United States and internationally.

Alnylam Pharmaceuticals

Market Cap: US$37.8b

Alnylam Pharmaceuticals, Inc. discovers, develops, manufactures, and commercializes therapeutics based on ribonucleic acid interference in the United States, Europe, and internationally.

Abeona Therapeutics

Market Cap: US$315.2m

A commercial-stage biopharmaceutical company, develops gene and cell therapies for life-threatening diseases in the United States.

Aldeyra Therapeutics

Market Cap: US$106.2m

A biotechnology company, discovers and develops therapies designed to treat immune-mediated diseases.

RenovoRx

Market Cap: US$38.1m

A clinical-stage biopharmaceutical company, engages in developing targeted combination therapies to improve therapeutic outcomes for cancer patients undergoing treatment.

Eton Pharmaceuticals

Market Cap: US$865.3m

A pharmaceutical company, focuses on developing and commercializing treatments for rare diseases.

Hyperion DeFi

Market Cap: US$38.6m

A DeFi company, engages in building on Hyperliquid.

Kiniksa Pharmaceuticals International

Market Cap: US$4.0b

A biopharmaceutical company, develops and commercializes medical therapies in the United States, the United Kingdom, and internationally.

BioStem Technologies

Market Cap: US$72.3m

Engages in the development, manufacturing, and commercialization of placental-derived allografts for advanced wound care and regenerative therapies in the United States.

Codexis

Market Cap: US$218.2m

Provides technology solutions for therapeutics manufacturing, leveraging its proprietary CodeEvolver technology platform to discover, develop, enhance, and commercialize novel enzymes in the United States, Canada, Latin America, Europe, the Middle East, Africa, Australia, New Zealand, Southeast Asia, and China.

Gossamer Bio

Market Cap: US$38.0m

A clinical-stage biopharmaceutical company, focuses on developing and commercializing seralutinib for the treatment of pulmonary hypertension and pulmonary arterial hypertension in the United States.

Arcutis Biotherapeutics

Market Cap: US$3.0b

A biopharmaceutical company, focuses on developing and commercializing treatments for dermatological diseases.

Citius Oncology

Market Cap: US$58.6m

Focuses on the development and commercialization of innovative targeted oncology therapies.

AC Immune

Market Cap: US$229.2m

A clinical stage biopharmaceutical company, discovers, designs, and develops medicines, and therapeutic and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding.