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Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

iBio, Inc., a biotechnology company, engages in the development of precision antibodies in the United States. It offers IBIO-100, a preclinical anti-fibrotic program for the treatment of systemic scleroderma and idiopathic pulmonary fibrosis; and EngageTx platform, which provides an optimized CD3 T-cell engager antibody panel. The company is also developing vaccine candidates, including IBIO-101, an antibody to reduce tumor growth; Endostatin E4 peptide for use in chemotherapy and immunotherapy; Trop-2 for the treatment Trop-2 positive cancers; MUC16, a tumor-associated epitope; anti-EGFRvIII antibody to treat glioblastoma and other cancers; CCR8 protein candidate for treatment of various cancers; PD-1 agonist for the treatment of rheumatoid arthritis and other inflammatory diseases; and IBIO-400 for the treatment of classical swine fever. iBio, Inc. has agreement with The Texas A&M University System for designing and manufacturing of plant-made biopharmaceuticals; and a research collaboration with the National Institute of Allergy and Infectious Diseases to investigate the potential of the company's AI-driven epitope steering platform for the development of a vaccine for Lassa fever. The company was incorporated in 2008 and is headquartered in Bryan, Texas.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

UroGen Pharma Ltd., a biotechnology company, engages in the development and commercialization of solutions for urothelial and specialty cancers. It offers RTGel, a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology to improve therapeutic profiles of existing drugs; and Jelmyto for pyelocalyceal solution. The company's lead product candidate is UGN-102 for the treatment of several forms of non-muscle invasive urothelial cancer that include low-grade upper tract urothelial cancer and low-grade intermediate risk non-muscle invasive bladder cancer (NMIBC). It is also developing UGN-301 for the treatment of high-grade NMIBC. The company has license agreement with Agenus Inc. to develop, make, use, sell, import, and commercialize products of Agenus for the treatment of cancers of the urinary tract via intravesical delivery; strategic research collaboration agreement with MD Anderson focusing on the sequential use of UGN-201 and UGN-301 for the treatment of NMIBC; and licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. to develop UGN-103 in low-grade intermediate risk NMIBC and UGN-104 in low-grade upper tract urothelial carcinoma. UroGen Pharma Ltd. was incorporated in 2004 and is based in Princeton, New Jersey.

Verrica Pharmaceuticals Inc., a clinical-stage dermatology therapeutics company, develops medications for the treatment of skin diseases in the United States. Its product pipeline comprises YCANTH (VP-102), which is in phase III clinical trial for the treatment of common warts; and has completed phase II clinical trial for the treatment of external genital warts. The company also develops VP-315, an oncolytic peptide-based injectable therapy, which is in phase II clinical trial for the treatment of dermatology oncologic conditions which includes basal cell carcinoma; and VP-103, a cantharidin-based product candidate for the treatment of plantar warts and is in phase II clinical trial. In addition, it offers YCANTH for the treatment of molluscum contagiosum. The company has a collaboration and license agreement with Torii Pharmaceutical Co., Ltd. for the development and commercialization of its product candidates for the treatment of molluscum contagiosum and common warts in Japan, including VP-102; and a license agreement with Lytix Biopharma AS to develop and commercialize VP-315 for dermatological oncology indications, such as non-metastatic melanoma and non-metastatic merkel cell carcinoma. Verrica Pharmaceuticals Inc. was incorporated in 2013 and is headquartered in West Chester, Pennsylvania.