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KemPharm, Inc. is a clinical-stage specialty pharmaceutical company, which engages in the discovery and development of proprietary prodrugs. It focuses on the treatment of serious medical conditions such as attention deficit hyperactivity disorder, pain, and other central nervous system disorders through its platform technology known as Ligand Activated Therapy. The company was founded by Christal M. M. Mickle and Travis C. Mickle on October 30, 2006 and is headquartered in Celebration, FL.

Novelion Therapeutics Inc., a biopharmaceutical company, develops a portfolio of therapies for individuals living with rare diseases in the United States, Brazil, and internationally. The company's commercial products include lomitapide hard capsule for the treatment of homozygous familial hypercholesterolemia in adults under the JUXTAPID and LOJUXTA brands; and metreleptin, a recombinant analogue of human leptin, which is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy under the MYALEPT brand. Its orphan drug-designated product candidate is zuretinol acetate, an oral synthetic retinoid, which is in development for the treatment of inherited retinal disease caused by underlying mutations in retinal pigment epithelium protein 65 and lecithin: retinol acyltransferase genes. The company was formerly known as QLT Inc. and changed its name to Novelion Therapeutics Inc. in November 2016. Novelion Therapeutics Inc. was founded in 1981 and is headquartered in Vancouver, Canada.

Novelion Therapeutics Inc., a biopharmaceutical company, develops therapies for individuals living with rare diseases in the United States, Japan, Brazil, and internationally. Its commercial products include metreleptin, a recombinant analog of human leptin, which is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy under the MYALEPTA brand name; and lomitapide capsule for the treatment of adult patients with homozygous familial hypercholesterolemia under the JUXTAPID and LOJUXTA brands. The company was formerly known as QLT Inc. and changed its name to Novelion Therapeutics Inc. in November 2016. Novelion Therapeutics Inc. was founded in 1981 and is headquartered in Vancouver, Canada.

Phio Pharmaceuticals Corp. develops immuno-oncology therapeutics in the United States. The company develops PH-762, INTASYL compound which reduces the expression of cell death Protein 1 (PD-1), a protein that inhibits T cells' ability to kill cancer cells; PH-894 that silences the BRD4, a protein which controls gene expression in both T cells and tumor cells, effecting the immune system as well as the tumor; and PH-804 that targets the TIGIT, a protein which inhibits the activity of Natural Killer cells. The company was formerly known as RXi Pharmaceuticals Corporation and changed its name to Phio Pharmaceuticals Corp. in November 2018. Phio Pharmaceuticals Corp. was incorporated in 2011 and is headquartered in Marlborough, Massachusetts.

Salarius Pharmaceuticals, Inc., a clinical-stage biotechnology company, focuses on developing treatments for cancers with unmet medical need. Its lead candidate is Seclidemstat (SP-2577), a small molecular inhibitor which is in Phase I/II clinical trial for the treatment of advanced solid tumors, as well as Ewing sarcoma. The company also offers SP-3164, a small molecular protein degrader for the treatment of hematological and solid tumors. It has a strategic partnership with The University of Utah Research Foundation for the exclusive license with respect to patent rights protecting SP-2577 and related compounds and Cancer Prevention and Research Institute of Texas for product development activities. The company is headquartered in Houston, Texas.