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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.

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.

Mateon Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing drugs for the treatment of orphan oncology indications. The company's lead product candidate is OT-101, an antisense against TGF-beta, which is in phase 3 clinical trials for pancreatic cancer and glioblastoma, as well as develops OT-101 for the treatment of various viruses, including severe acute respiratory syndrome and coronavirus. It also develops OXi4503 for the treatment of acute myeloid leukemia and myelodysplastic syndromes; and CA4P for the treatment of advanced metastatic melanoma. The company is headquartered in Agoura Hills, California.

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.

Tocagen Inc., a clinical-stage cancer-selective gene therapy company, focuses on developing and commercializing product candidates designed to activate a patient's immune system against their cancer. Its cancer-selective gene therapy platform is built on retroviral replicating vectors (RRVs), which are designed to deliver therapeutic genes into the DNA of cancer cells. The company's lead product candidate is Toca 511 & Toca FC that is under Phase III clinical trial for recurrent high-grade glioma. It is also developing Toca 511 & Toca FC in a Phase Ib clinical trial for intravenous treatment of advanced cancers. In addition, the company is developing other RRVs to deliver genes to cancer cells against validated immunotherapy targets. The company has a license agreement with ApolloBio to develop and commercialize Toca 511 & Toca FC; and a collaboration agreement with NRG Oncology to develop a clinical trial utilizing Toca 511 & Toca FC for the treatment of patients with newly diagnosed glioblastoma. Tocagen Inc. was founded in 2007 and is based in San Diego, California.