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Karyopharm Therapeutics Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of drugs directed against nuclear transport and related targets for the treatment of cancer and other diseases. Its lead drug candidate is Selinexor(KPT-330), which is in Phase IIb clinical study in treatments of refractory multiple myeloma; Phase Ib/II clinical study in combination with backbone treatments for multiple myeloma patients; Phase III clinical trial in combination with Velcade (bortezomib) and dexamethasone for multiple myeloma patients; Phase IIb cli... view company details

Sesen Bio, Inc., a late-stage clinical company, focuses on designing, engineering, developing, and commercializing targeted fusion protein therapeutics (TFPTs) for the treatment of cancer. The company's lead product candidates include Vicinium, a locally-administered targeted fusion protein that is in Phase III clinical trials for the treatment of high-risk non-muscle invasive bladder cancer (NMIBC); and VB6-845d, a systemically-administered product candidate, which is in Phase I/II clinical trials for use in the treatment of various types of EpCAM-positive solid tumors. It is also developing ... view company details

Vertex Pharmaceuticals Incorporated engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene. It is also developing VX-659 and VX-445 that are Phase III clinical trials; VX-121, a cystic fibrosis transmembrane conductance regulator corrector that is in Phase I/II clinical trial; VX-150, an inhibitor that is in Phase IIb clinical trial for acute pain; CTX001 that is in Phase I/II c... view company details

Nektar Therapeutics develops drug candidates for cancer, auto-immune disease, and chronic pain in the United States. The company develops NKTR-181, an orally-available mu-opioid analgesic molecule, which is in Phase III clinical trial for moderate to severe chronic pain; ONZEALD, a topoisomerase I inhibitor that is in Phase III clinical trial for advanced metastatic breast cancer in patients with brain metastases; and NKTR-214, a CD122-preferential interleukin-2 (IL-2) pathway agonist, which is in Phase I to treat immuno-oncology. It also develops NKTR-358, cytokine Treg stimulant, which is in... view company details

Achillion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes small molecule drug therapies for immune system disorders. Its lead drug candidate is ACH-4471, an inhibitor of factor D that is Phase II clinical trials for patients with paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy/immune complex membranoproliferative glomerulonephritis. The company is also developing ACH-5228, a factor D inhibitor that is in Phase I clinical trials; and ACH-5548, a factor D inhibitor, which is in Phase I clinical trials for the treatment of... view company details

Deciphera Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops drugs to enhance the lives of cancer patients by addressing key mechanisms of drug resistance that limit the rate and durability of response of various cancer therapies. The company's lead drug candidate is DCC-2618, which is in Phase III trial for the treatment of gastrointestinal stromal tumors; and that is in Phase I trial for treating advanced systemic mastocytosis, gliomas, melanoma, NSCLC/germ cell/penile, and soft tissue sarcomas. It is also developing immunokinase inhibitors comprising DCC-3014 that i... view company details

ImmunoGen, Inc., a clinical-stage biotechnology company, develops antibody-drug conjugate (ADC) therapies to treat cancer. Its product candidates include mirvetuximab soravtansine, an ADC targeting folate-receptor alpha, which is in Phase III clinical trial for the treatment of platinum-resistant ovarian cancer; IMGN779 that is in Phase I clinical trial for the treatment of acute myeloid leukemia (AML); and IMGN632, a CD123-targeting ADC that is in Phase I clinical trial for treating AML and blastic plasmacytoid dendritic cell neoplasm. ImmunoGen, Inc. has collaborations with Roche, Amgen/Oxfo... view company details

Jounce Therapeutics, Inc., a clinical-stage immunotherapy company, develops therapies for the treatment of cancer. The company offers vopratelimab, a clinical-stage monoclonal antibody that binds to and activates the Inducible T cell CO-Stimulator, a protein on the surface of T cells found in various solid tumors, which is in Phase I/II clinical trial for the treatment of head and neck squamous cell cancer, non-small cell lung cancer, triple negative breast cancer, gastric cancer, and other tumor types identified through its translational science platform. It is also developing JTX-4014, a mon... view company details

Protagonist Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops peptide-based product candidates to address unmet medical needs in hematology and gastroenterology. The company is developing PTG-300, an injectable hepcidin mimetic that is in Phase II clinical trial for the treatment of patients with beta-thalassemia by targeting the chronic anemia; PTG-200, an antagonist peptide product candidate, which has completed Phase I clinical trial to treat inflammatory bowel disease (IBD); and PN-943, an oral, alpha-4-beta-7 specific integrin antagonist that is in Phase I clinical ... view company details

Spero Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on identifying, developing, and commercializing novel treatments for multi-drug resistant (MDR) bacterial infections in the United States. Its product candidate includes SPR994, an oral carbapenem-class antibiotic to treat MDR gram-negative infections for adults; SPR741, which is in Phase Ib clinical trial to treat MDR gram-negative infections in the hospital setting; SPR206, an agent that is phase I clinical trial to disrupt the outer membrane of gram-negative bacteria; and SPR720, an oral antibiotic, which is in ph... view company details

Trevena, Inc., a biopharmaceutical company, focuses on the development and commercialization of treatment options that target and treat diseases affecting the central nervous system. Its product candidates include Oliceridine injection, a G protein biased mu-opioid receptor (MOR) ligand that has completed Phase III clinical trials for the management of moderate-to-severe acute pain where intravenous administration is warranted; TRV250, a G protein biased delta- opioid receptor ligand, which is in Phase I clinical study for the treatment of acute migraine; TRV734, a small molecule G protein bia... view company details

Agios Pharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of medicines in the field of cellular metabolism and adjacent areas of biology for the treatment of cancer and rare genetic diseases. The company offers TIBSOVO (ivosidenib), an oral targeted inhibitor for treating adult patients with relapsed or refractory acute myeloid leukemia (R/R AML), as well as to treat patients with newly diagnosed AML; and IDHIFA, an oral targeted inhibitor for patients with R/R AML and an isocitrate dehydrogenase 2 mutation. It is also developing TIBSOVO, which has comp... view company details

Athenex, Inc., a biopharmaceutical company, engages in the discovery, development, and commercialization of various therapies for the treatment of cancer and related conditions in North America and Asia. It operates in three segments: Oncology Innovation Platform, Global Supply Chain Platform, and Commercial Platform. The company's Orascovery product candidates include Oraxol, an oral dosage form, which is in Phase III trial for metastatic breast cancer, as well as various clinical studies in advanced malignances and gastric cancer; and Oratecan, an oral dosage form of irinotecan that is in a ... view company details

Aduro BioTech, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapies to harness the body's natural immune system for the treatment of patients with challenging diseases. The company is developing ADU-S100, which is in Phase I dose escalation and dose expansion clinical trial and Phase Ib dose escalation and dose expansion clinical trial in combination with spartalizumab to treat patients with advanced, metastatic treatment-refractory solid tumors; Phase I clinical trial in combination with ipilimumab for the treatment of rel... view company details

BioTime, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing therapies for the treatment of degenerative diseases in the United States and internationally. The company's lead product candidates include OpRegen, a retinal pigment epithelium cell replacement therapy, which is in Phase I/IIa multicenter trial for the treatment of the dry age-related macular degeneration; OPC1, an oligodendrocyte progenitor cell therapy that is in Phase I/IIa multicenter clinical trial for the treatment of acute spinal cord injuries; and VAC2, an allogeneic cancer immunotherapy ... view company details

Axsome Therapeutics, Inc., a clinical stage biopharmaceutical company, engages in developing novel therapies for central nervous system (CNS) disorders in the United States. Its product pipeline includes AXS-05, which is in the Phase III clinical trial for the treatment resistant depression; Phase II/III clinical trials in agitation associated with Alzheimer's disease; and Phase II clinical trial for the treatment of smoking cessation, as well as for major depressive disorder. The company is also developing AXS-07, which is in Phase III clinical trial for the treatment of migraine; AXS-12 that... view company details

Audentes Therapeutics, Inc., a clinical stage biotechnology company, focuses on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. The company is developing AT132, which is in Phase I/II clinical studies for the treatment of X-linked myotubular myopathy (XLMTM); AT342 that is in Phase I/II clinical studies to treat crigler-najjar syndrome; AT845, which is in preclinical studies for the treatment of pompe disease; and AT307 to treat CASQ2 subtype of catecholaminergic polymorphic ventricular tachyca... view company details

Aileron Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing a novel class of therapeutics in the United States. The company's lead product candidate is ALRN-6924, a stapled peptide, which is in Phase I clinical trial for the treatment of advanced solid tumors or lymphomas; Phase IIa clinical trial to treat peripheral T-cell lymphoma; Phase I clinical trial for the treatment of acute myeloid leukemia (AML) and advanced myelodysplastic syndrome (MDS); and Phase IIb trial to treat AML/MDS in combination with cytosine arabinoside. It also devel... view company details

ArQule, Inc., a biopharmaceutical company, researches and develops therapeutics for the treatment of cancer and rare diseases in the United States. The company's pipeline includes ARQ 531, an orally bioavailable, potent and reversible dual inhibitor of wild type and C481S-mutant Bruton's tyrosine kinase that is in Phase I trial for patients with B-cell malignancies refractory to other therapeutic options; and miransertib (ARQ 092), a potent and selective inhibitor of the protein kinase B (AKT), a serine/threonine kinase, which is in Phase Ib in combination with the hormonal therapy and anastro... view company details

Blueprint Medicines Corporation develops drugs of small molecule kinase inhibitors that target genomic drivers in various cancers and a rare genetic disease. Its lead drug candidates include avapritinib, which completed Phase I clinical trials that targets PDGFRA Exon 18 mutant GIST and KIT-driven GIST; and BLU-554, which is in Phase I clinical trials an orally available, potent, and irreversible inhibitor of the kinase FGFR4 that is activated in a defined subset of patients with hepatocellular carcinoma. It is also developing BLU-667, an orally available and potent inhibitor that targets RET,... view company details