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Ayala Pharmaceuticals, Inc., a clinical-stage oncology company, primarily focuses on developing and commercializing small molecule therapeutics for people living with rare tumors and aggressive cancers. The company's lead candidates under development include AL102, an oral gamma secretase inhibitor for desmoid tumors; and aspacytarabine (BST-236), a novel proprietary anti-metabolite for first line treatment in unfit acute myeloid leukemia. Ayala Pharmaceuticals, Inc. is based in Wilmington, Delaware.

Acerus Pharmaceuticals Corp. is a pharmaceutical company. The firm is focused on the commercialization and development of prescription products that improve patient experience, with a focus in the field of men's health. It focuses on therapeutics for urology, andrology, and endocrinology. Its products include Estrace and Natesto. The company was founded by Bruce D. Brydon, Rolf K. Reininghaus and Mark L. Thompson on September 9, 2008 and is headquartered in Mississauga, Canada.

Conatus Pharmaceuticals Inc., a biotechnology company, focuses on the development and commercialization of novel medicines for the treatment of liver diseases in the United States. Its product candidate includes Emricasan, an orally active caspase protease inhibitor that is in Phase IIb clinical trials for post-orthotopic liver transplant as a result of hepatitis C virus infection with sustained viral response; for portal hypertension; for liver fibrosis caused by nonalcoholic steatohepatitis; and for liver function. The company is also developing CTS-2090, an orally active inhibitor of caspase 1, which is in preclinical development stage for treating chronic diseases involving inflammasome pathways. The company has a collaboration agreement with Novartis to conduct three Phase IIb clinical trials. Conatus Pharmaceuticals Inc. was founded in 2005 and is headquartered in San Diego, California.

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.