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Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

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.

Prothena Corporation plc, a late-stage clinical biotechnology company, focuses on discovery and development of novel therapies to treat diseases caused by protein dysregulation in the United States. The company is involved in developing birtamimab, an investigational humanized antibody that is in Phase III clinical trial for the treatment of AL amyloidosis; Prasinezumab, a humanized monoclonal antibody, for the treatment of Parkinson's disease and other related synucleinopathies which is in Phase IIb clinical trial; NNC6019 that is in Phase lI clinical trial for the treatment of ATTR amyloidosis; and BMS-986446 and PRX012, which is in Phase I clinical trial for the treatment of Alzheimer's disease. Its discovery and preclinical programs include PRX123, a dual Aß-Tau vaccine for the treatment and prevention of Alzheimer's disease; and PRX019 for the treatment of neurogenerative diseases, as well as TDP-43 for the treatment of amyotrophic lateral sclerosis. Prothena Corporation plc has a license, development, and commercialization agreement with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. to develop and commercialize antibodies that target a-synuclein, including prasinezumab; and a collaboration agreement with Bristol-Myers Squibb to develop and commercialize antibodies targeting tau, TDP-43. The company was incorporated in 2012 and is based in Dublin, Ireland.

Protagonist Therapeutics, Inc., a biopharmaceutical company, develops peptide-based drugs for hematology and blood disorders, and inflammatory and immunomodulatory diseases. It is developing Rusfertide (PTG-300), an injectable hepcidin mimetic that completed phase 2 clinical trials for the treatment of polycythemia vera and other blood disorders; and JNJ-2113, an orally delivered investigational drug to block biological pathways that completed phase 2b clinical trials for the treatment of moderate-to-severe plaque psoriasis; and PN-943, an orally delivered, gut-restricted alpha 4 beta 7 specific integrin antagonist completed a phase 2 clinical trials in patients with moderate to severe ulcerative colitis. The company has a license and collaboration agreement with Takeda to commercialize rusfertide; and JNJ Innovative Medicines to co-develop Interleukin-23 receptor antagonist compound JNJ-2113. Protagonist Therapeutics, Inc. was incorporated in 2006 and is headquartered in Newark, California.

VBI Vaccines Inc., a biopharmaceutical company, develops and sells vaccines to treat immuno-oncology and infectious disease. It offers Sci-B-Vac, a prophylactic hepatitis B (HBV) vaccine. The company also develops VBI-2601, an immunotherapeutic candidate for the treatment of chronic HBV infection; VBI-1901, a glioblastoma vaccine immunotherapeutic candidate, which is in Phase I/IIa clinical study to treat solid tumors; VBI-1501, a prophylactic cytomegalovirus vaccine candidate that has completed Phase I clinical trial; and VBI-2501 that is in preclinical trial to treat Zika virus. In addition, it develops coronavirus vaccine candidates that include VBI-2902, VBI-2901, and VBI-2905. The company has collaboration and license agreements with Brii Biosciences Limited; GlaxoSmithKline Biologicals S.A.; and the National Research Council of Canada to develop pan-coronavirus vaccine candidate targeting COVID-19, severe acute respiratory syndrome, and Middle East respiratory syndrome. It also has a collaboration with Coalition for Epidemic Preparedness Innovations to advance vaccine candidates against Covid-19 variants. The company was formerly known as SciVac Therapeutics Inc. and changed its name to VBI Vaccines Inc. in May 2016. VBI Vaccines Inc. is based in Cambridge, Massachusetts.