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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.

Mersana Therapeutics, Inc., a clinical stage biopharmaceutical company, develops antibody drug conjugates (ADC) for cancer patients with unmet needs. The company develops XMT-1660, a B7-H4-targeted Dolasynthen ADC candidate; and XMT-2056, an immunosynthen ADC. It has research and development collaborations with Janssen Biotech, Inc., Ares Trading S.A., Merck KGaA, and Asana BioSciences, LLC for the development of ADC product candidates. The company was formerly known as Nanopharma Corp. and changed its name to Mersana Therapeutics, Inc. in November 2005. Mersana Therapeutics, Inc. was incorporated in 2001 and is headquartered in Cambridge, Massachusetts.

ORIC Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of therapies for treatment of cancers in the United States. Its clinical stage product candidates include ORIC-114, a brain penetrant orally bioavailable irreversible inhibitor, currently under Phase 1b study, which is designed to selectively target epidermal growth factor receptor and human epidermal growth factor receptor 2 with high potency towards exon 20 insertion mutations; ORIC-944, an allosteric inhibitor of the polycomb repressive complex 2 for prostate cancer, currently under Phase 1b study; and ORIC-533, an orally bioavailable small molecule inhibitor of CD73, currently under Phase 1b study, being developed for resistance to chemotherapy- and immunotherapy-based treatment regimens. The company is also developing multiple discovery stage precision medicines targeting other cancer resistance mechanisms, including ORIC-613, an orally bioavailable, highly selective inhibitor of PLK4, in preclinical studies for the treatment of breast cancers. It has a license and collaboration agreement with Voronoi Inc.; a license agreement with Mirati Therapeutics, Inc.; and clinical development collaboration agreement with Pfizer Inc. The company was incorporated in 2014 and is headquartered in South San Francisco, California.

Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company's commercialized product is RUCONEST, a plasma-free rhC1INH protein replacement therapy for the treatment acute hereditary angioedema (HAE) attacks. It also engages in the development of leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome; OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE; and alpha-glucosidase therapy for the treatment of Pompe and fabry diseases. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. is headquartered in Leiden, the Netherlands.

Stemline Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, acquisition, development, and commercialization of oncology therapeutics in the United States and internationally. The company offers ELZONRIS, a targeted therapy directed to the interleukin-3 receptor (CD123) for patients with blastic plasmacytoid dendritic cell neoplasm in adults, and in pediatric patients two years and older. It also developing CD123 that is in Phase I/II clinical trials for the treatment of chronic myelomonocytic leukemia and myelofibrosis, as well as acute myeloid leukemia and multiple myeloma in combination with other therapies. In addition, the company is developing SL-801, a novel oral small molecule reversible inhibitor of nuclear transport protein, which is in Phase I clinical trial for the treatment of solid and hematologic cancers; and SL-701, an immunotherapy that has completed Phase II clinical trials to treat brain cancer. Its preclinical pipeline products include SL-501, a CD123 targeted therapy; SL- 1001, an oral, selective small molecule RET kinase inhibitor targeting multiple oncology indications; and SL-901, a small molecule kinase inhibitor. Stemline Therapeutics, Inc. was founded in 2003 and is based in New York, New York.