Achillion Pharmaceuticals
NASDAQ:ACHNAchillion 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 PNH and other complement mediated diseases. It has license agreements with Ora, Inc. for the development and commercialization of ACH-702; and GCA Therapeutics, Ltd for elvucitabine, a nucleoside reverse transcriptase inhibitor for the treatment of hepatitis B infection and human immunodeficiency virus infection. The company was founded in 1998 and is based in New Haven, Connecticut.
G1 Therapeutics
NASDAQ:GTHXG1 Therapeutics, Inc., a commercial-stage biopharmaceutical company, engages in the discovery, development, and commercialization of small molecule therapeutics for the treatment of patients with cancer in the United States. The company offers COSELA, which helps to decrease incidence of chemotherapy-induced myelosuppression in adult patients treated with a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive stage small cell lung cancer. It is also developing Trilaciclib that is in Phase 3 clinical trial for 1L metastatic triple negative breast cancer (mTNBC); and Phase 2 clinical trial as an antibody-drug conjugate combination trial in mTNBC, as well as completed Phase 2 clinical trial for Neoadjuvant TNBC and 1L Bladder cancer. The company has a license agreement with Genor Biopharma Co. Inc. for the development and commercialization of lerociclib using an oral dosage form to treat various indication in humans; Incyclix for the development and commercialization of a CDK2 inhibitor for all human and veterinary uses; and Simcere Pharmaceutical Co., Ltd for the development and commercialization of trilaciclib in all indications. G1 Therapeutics, Inc. was incorporated in 2008 and is headquartered in Research Triangle Park, North Carolina.
Stoke Therapeutics
NASDAQ:STOKStoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.
TG Therapeutics
NASDAQ:TGTXTG Therapeutics, Inc., a commercial stage biopharmaceutical company, focuses on the acquisition, development, and commercialization of novel treatments for B-cell mediated diseases in the United States and internationally. It provides BRIUMVI, an anti-CD20 monoclonal antibody for the treatment of adult patients with relapsing forms of multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease in adults. The company's development pipeline comprises Umbralisib, an oral inhibitor of PI3K-delta inhibitor for the treatment of adult patients with relapsed or refractory marginal zone lymphoma and follicular lymphoma; TG-1701 is an orally available and covalently bound Bruton's tyrosine kinase (BTK) inhibitor that exhibits selectivity to BTK in vitro kinase screening; and TG-1801, a bispecific CD47 and CD19 antibody. Its research pipeline includes various investigational medicines. The company has license agreements with LFB Biotechnologies S.A.S; GTC Biotherapeutics; LFB/GTC LLC; Ildong Pharmaceutical Co. Ltd.; Rhizen Pharmaceuticals, S A. for the development and commercialization of umbralisib; Jiangsu Hengrui Medicine Co.; and Novimmune SA, as well as collaboration agreement with Checkpoint Therapeutics, Inc. for the development and commercialization of anti-PD-L1 and anti-GITR antibody research programs in the field of hematological malignancies. TG Therapeutics, Inc. was incorporated in 1993 and is based in Morrisville, North Carolina.
Vanda Pharmaceuticals
NASDAQ:VNDAVanda Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of therapies to address high unmet medical needs worldwide. The company's marketed products include HETLIOZ to treat non-24-hour sleep-wake disorders; and Fanapt oral tablets for the treatment of schizophrenia. Its pipeline products include HETLIOZ (tasimelteon) to treat jet lag disorder, insomnia, delayed sleep phase disorder, sleep disturbances in autism spectrum disorder, and pediatric Non-24; Fanapt (iloperidone) for the treatment of bipolar I disorder and Parkinson's disease psychosis, as well as a long acting injectable (LAI) formulation to treat schizophrenia; and Tradipitant (VLY-686), a small molecule neurokinin-1 receptor (NK-1R) antagonist to treat gastroparesis, motion sickness, atopic dermatitis, and COVID-19 pneumonia. The company's pipeline products also include VTR-297, a small molecule histone deacetylase inhibitor to treat hematologic malignancies and with potential use as a treatment for various oncology indications; VQW-765, a small molecule nicotinic acetylcholine receptor partial agonist to treat performance anxiety and psychiatric disorders; VHX-896, an active metabolite of iloperidone; and antisense oligonucleotide molecules. In addition, it offers a portfolio of cystic fibrosis transmembrane conductance regulator activators and inhibitors that include VSJ-110 for the treatment of dry eye and ocular inflammation; and VPO-227 for the treatment of secretory diarrhea disorders comprising cholera. Vanda Pharmaceuticals Inc. was incorporated in 2002 and is headquartered in Washington, the District of Columbia.