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Acasti Pharma Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was incorporated in 2002 and is headquartered in Laval, Canada.

Adamas Pharmaceuticals, Inc. engages in discovery and development of chrono-synchronous therapies for chronic neurologic disorders. Its products include GOCOVRI, ADS-5012, ADS-4101, and Namzaric. GOCOVRI is indicated for the treatment of dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy. ADS-5102 is a treatment for walking impairment in patients with multiple sclerosis. ADS-401 treats partial onset seizures in patients with epilepsy. Namzaric is a drug treatment for moderate to severe Alzheimer's disease. The company was founded by Gregory T. Went on November 15, 2000 and is headquartered in Emeryville, CA.

Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need in the United States. The company's lead product candidate is AT-007 (also called govorestat) that has completed phase 3 for the treatment of galactosemia in healthy volunteers and adults, in pediatric clinical study for the treatment of galactosemia in kids, for treating enzyme sorbitol dehydrogenase, and for the treatment of phosphomannomutase enzyme-CDG. It also develops AT-001 (also called caficrestat) that is in phase 3 clinical trials to treat diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy; and AT-003, which is in preclinical studies for the treatment diabetic retinopathy. The company has exclusive license and supply agreement with Mercury Pharma Group Limited to commercialize drug products containing AT-007. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.

Cerecor, Inc. is a biopharmaceutical company, which engages in the development and commercialization of treatments for rare and orphan diseases. Its lead product candidates include CERC-801, CERC-802, and CERC-803, which are therapies for inherited metabolic disorders known as congenital disorders of glycosylation. The company was founded by Blake M. Paterson, Isaac Blech, Barbara S. Slusher, and Solomon H. Snyder on January 31, 2011 and is headquartered in Rockville, MD.

Cue Biopharma, Inc., a clinical-stage biopharmaceutical company, develops a novel class of injectable therapeutics to selectively engage and modulate targeted, disease relevant T cells directly within the patient's body. Its lead drug product candidate is CUE-101 for the treatment of human papilloma virus (HPV16+)-driven recurrent/metastatic head and neck cancer. The company is also developing CUE-102 targets Wilms' Tumor 1 protein in various cancers; CUE-103, a CUE-100 series drug product candidate; and Neo-STAT and RDI-STAT programs outside of oncology, including CUE-200, CUE-300, and CUE-400 series. It has collaboration agreements with LG Chem, Ltd. for the development of Immuno-STATs focused in the field of oncology; strategic collaboration and option agreement with Ono Pharmaceutical Co., Ltd. to advance CUE-401 for the treatment of autoimmune and inflammatory diseases; and license agreement with Albert Einstein College of Medicine. The company was formerly known as Imagen Biopharma, Inc. and changed its name to Cue Biopharma, Inc. in October 2016. Cue Biopharma, Inc. was incorporated in 2014 and is headquartered in Boston, Massachusetts.