Kiniksa Pharmaceuticals
NASDAQ:KNSAKiniksa 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.
MacroGenics
NASDAQ:MGNXMacroGenics, Inc., a biopharmaceutical company, develops, manufactures, and commercializes antibody-based therapeutics to treat cancer in the United States. Its approved product is MARGENZA (margetuximab-cmkb), a human epidermal growth factor receptor 2 (HER2) receptor antagonist indicated, in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens. The company's pipeline of immuno-oncology product candidates includes MGC018, an antibody drug conjugate (ADC), which targets solid tumors expressing B7-H3; Enoblituzumab, a monoclonal antibody that targets B7-H3; and MGD024, an investigational bispecific CD123 × CD3 DART molecule to minimize cytokine-release syndrome for patients with hematologic malignancies. In addition, it develops Lorigerlimab, a monoclonal antibody that targets the immune checkpoints PD-1 and cytotoxic T-lymphocyte-associated protein 4; Tebotelimab, an investigational tetravalent DART molecule for PD-1 and lymphocyte-activation gene 3; Retifanlimab, a humanized monoclonal antibody targeting programmed death receptor-1; and IMGC936, an ADC that targets ADAM9, a cell surface protein over-expressed in various solid tumor types. Further, the company develops MGD014 and MGD020, a DART molecule to target the envelope protein of human immunodeficiency virus infected cells and CD3 on T cells; Teplizumab for the treatment of type 1 diabetes; and PRV-3279, a CD32B × CD79B DART molecule for the treatment of autoimmune indications. It has collaborations with Incyte Corporation; Zai Lab Limited; I-Mab Biopharma; and Janssen Biotech, Inc. The company was incorporated in 2000 and is headquartered in Rockville, Maryland.
Repare Therapeutics
NASDAQ:RPTXRepare Therapeutics Inc., a clinical-stage precision oncology company, engages in the discovery and development of therapeutics by using its synthetic lethality approach in Canada and the United States. It uses its SNIPRx, a proprietary, genome-wide, and CRISPR-enabled platform, to discover, validate, and build a pipeline of SL-based therapeutics that focuses on genomic instability, including DNA damage repair. The company's lead product candidate is Camonsertib (RP-3500), an oral small molecule inhibitor under Phase ½ development for the treatment of solid tumors with specific DNA damage repair-related genomic alterations. It is also developing Lunresertib (RP-6306), a PKMYT1 Inhibitor, which is under Phase 1 clinical trial for tumors with genetic alterations characterized by CCNE1 amplification; RP-1664, an oral PLK4 inhibitor, under Phase 1 clinical trial designed to harness the synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors; and RP-3467, a polymerase theta adenosinetriphosphatase (ATPase) inhibitor, a SL target associated with BRCA mutations and other genomic alterations. The company has license and collaboration agreement with Hoffmann-La Roche Inc. and F. Hoffmann-La Roche Ltd; Bristol-Myers Squibb Company; New York University; and Ono Pharmaceutical Co., as well as a clinical study and collaboration agreement with Debiopharm to explore the synthetic lethal combination of PKMYT1 and WEE1 inhibition in cancer. Repare Therapeutics Inc. was incorporated in 2016 and is headquartered in Montréal, Canada.
Travere Therapeutics
NASDAQ:RTRXTravere Therapeutics, Inc. is a biopharmaceutical company. It engages in the identification, development, commercialization, and distribution of therapies to people living with rare diseases. Its products include Chenodal, Cholbam, and Thiola. The company was founded by Martin Shkreli on February 8, 2008 and is headquartered in San Diego, CA.
Rhythm Pharmaceuticals
NASDAQ:RYTMRhythm Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases. The company's lead product candidate is IMCIVREE (setmelanotide), a rare melanocortin-4 receptor for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1, leptin receptor (LEPR) deficiency obesity, and Bardet-Biedl and Alström syndrome. It is in Phase 3 clinical trials for treating POMC or LEPR heterozygous deficiency obesities, steroid receptor coactivator 1 deficiency obesity, SH2B1 deficiency obesity, MC4 receptor deficiency obesity, and other MC4R disorders. The company has licensing agreements with LG Chem, Ltd; Ipsen Pharma S.A.S; Camurus; RareStone Group Ltd.; and LG Chem, Ltd. The company was formerly known as Rhythm Metabolic, Inc. and changed its name to Rhythm Pharmaceuticals, Inc. in October 2015. Rhythm Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Boston, Massachusetts.