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Akcea Therapeutics, Inc., a biopharmaceutical company, focuses on developing and commercializing drugs to treat patients with serious and rare diseases in the United States and internationally. The company offers TEGSEDI, which is designed to reduce the production of transthyretin protein. It develops WAYLIVRA, which has completed Phase III clinical study for the treatment of familial chylomicronemia syndrome; and that is in Phase III clinical study for the treatment of familial partial lipodystrophy. The company also develops AKCEA-APO(a)-LRx, which has completed Phase IIb clinical study for treating patients suffering with hyperlipoproteinemia; AKCEA-ANGPTL3-LRx that has completed Phase IIb clinical study for the treatment of multiple lipid disorders; and AKCEA-APOCIII-LRx, which is in Phase II study for the treatment of cardiovascular disease due to elevated triglyceride levels, as well as AKCEA-TTR-LRx to treat the broad population of patients with hereditary and wild-type forms of transthyretin amyloidosis. It has strategic collaboration with Novartis Pharma AG and PTC Therapeutics International Limited. The company was incorporated in 2014 and is headquartered in Boston, Massachusetts. Akcea Therapeutics, Inc. operates as a subsidiary of Ionis Pharmaceuticals, Inc.

Arcturus Therapeutics Holdings Inc., a late-stage clinical messenger RNA medicines and vaccine company, focuses on the development of infectious disease vaccines and other products within liver and respiratory rare diseases. Its technology platforms include LUNAR lipid-mediated delivery and STARR mRNA. The company is developing ARCT-810 (LUNAR-OTC), a mRNA-based therapeutic candidate, which is in Phase 2 clinical trial for treating ornithine transcarbamylase deficiency; and ARCT-154 (LUNAR-COV19), a mRNA vaccine candidate that is in Phase 3 arm of a Phase 1/2/3 study in Vietnam for the treatment of COVID-19, as well as ARCT-032 (LUNAR-CF), a mRNA therapeutic candidate for cystic fibrosis. Its product pipeline includes, ARCT-2301 for bivalent: ancestral/omicron which is in Phase 3; ARCT-2303 for monovalent that is in Phase 3; ARCT-2138 for quadrivalent which is in Phase 1; and LUNAR-FLU which is in pre-clinical trial. Arcturus Therapeutics Holdings Inc. was founded in 2013 and is headquartered in San Diego, California.

Kiniksa Pharmaceuticals, Ltd., a biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical needs worldwide. Its product candidates include ARCALYST, an interleukin-1alpha and interleukin-1beta, for the treatment of recurrent pericarditis, which is an inflammatory cardiovascular disease; Mavrilimumab, a monoclonal antibody inhibitor that completed Phase II clinical trials for the treatment of giant cell arteritis; Vixarelimab, a monoclonal antibody, that is in Phase 2b clinical trial for the treatment of prurigo nodularis, a chronic inflammatory skin condition; and KPL-404, a monoclonal antibody inhibitor of the CD40- CD154 interaction, a T-cell co-stimulatory signal critical for B-cell maturation, immunoglobulin class switching, and type 1 immune response. The company was incorporated in 2015 and is based in Hamilton, Bermuda.

Kura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer. The company's pipeline consists of small molecule product candidates that target cancer. Its lead product candidates are ziftomenib, an orally bioavailable small molecule inhibitor of the menin-KMT2A interaction for the treatment of genetically defined subsets of acute leukemias, including acute myeloid leukemia and acute lymphoblastic leukemia; tipifarnib, an orally bioavailable farnesyl transferase inhibitor combination with alpelisib for patients with PIK3CA-dependent HNSCC; and KO-2806, a farnesyl transferase inhibitor for the treatment of solid tumors. It has a clinical collaboration with Novartis Pharma AG to evaluate the combination of tipifarnib and alpelisib in patients with head and neck squamous cell carcinoma whose tumors have HRAS overexpression and/or PIK3CA mutation and/or amplification. The company was founded in 2014 and is headquartered in San Diego, California.

Supernus Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of products for the treatment of central nervous system (CNS) diseases in the United States. The company's commercial products are Trokendi XR, an extended release topiramate product indicated for the treatment of epilepsy, as well as for the prophylaxis of migraine headache; and Oxtellar XR, an extended release oxcarbazepine for the monotherapy treatment of partial onset seizures in adults and children between 6 to 17 years of age. It also offers comprise Qelbree, a novel non-stimulant indicated for the treatment of attention-deficit hyperactivity disorder (ADHD) in adults and pediatric patients 6 years and older; APOKYN for the acute intermittent treatment of hypomobility or off episodes in patients with advanced Parkinson's Disease (PD); XADAGO for treating levodopa/carbidopa in patients with PD experiencing off episodes; MYOBLOC, a Type B toxin product indicated for the treatment of cervical dystonia and sialorrhea in adults; GOCOVRI for the treatment of dyskinesia in patients with PD; and Osmolex ER for the treatment of Parkinson's disease and drug-induced extrapyramidal reaction in adult patients. In addition, the company's product candidates include SPN-830, a late-stage drug/device combination product candidate for the treatment of off episodes in PD patients; SPN-817, a novel first-in-class selective acetylcholinesterase inhibitor, which is in Phase II clinical trials for the treatment of epilepsy; SPN-820, a product candidate in Phase II clinical trials for treating resistant depression; SPN-443, a preclinical product for the treatment of ADHD/CNS; and SPN-446 for narcolepsy and SPN-448 for the treatment of CNS which is in discovery stage. It markets and sells its products through pharmaceutical wholesalers, specialty pharmacies, and distributors. Supernus Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Rockville, Maryland.