Amryt Pharma
NASDAQ:AMYTAmryt Pharma plc, a commercial-stage biopharmaceutical company, focuses on acquiring, developing, and commercializing various treatments to help improve the lives of patients with rare and orphan diseases. The company provides metreleptin, an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; oral octreotide, a long-term maintenance therapy in acromegaly patients; and lomitapide, an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the Homozygous familial Hypercholesteraemia. Its development candidates include Oleogel-S10 for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults; and AP103, a pre-clinical gene therapy for patients with dystrophic EB. The company sells its products in the Americas, Europe, and the Middle East. Amryt Pharma plc was founded in 2015 and is headquartered in Dublin, Ireland.
Esperion Therapeutics
NASDAQ:ESPREsperion Therapeutics, Inc., a pharmaceutical company, develops and commercializes medicines for the treatment of patients with elevated low density lipoprotein cholesterol (LDL-C). Its marketed products include NEXLETOL (bempedoic acid) and NEXLIZET (bempedoic acid and ezetimibe) tablets that are oral, once-daily, non-statin medicines for the treatment of primary hyperlipidemia in adults with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease who require additional lowering of LDL-C. The company's products also include NILEMDO, an ATP Citrate Lyase (ACL) inhibitor that lowers LDL-C and cardiovascular risk by reducing cholesterol biosynthesis and up-regulating the LDL receptors; and NUSTENDI, a bempedoic acid and ezetimibe tablet to treat elevated LDL-C. The company has license and collaboration agreements with Daiichi Sankyo Co. Ltd to; Otsuka Pharmaceutical Co., Ltd; and Daiichi Sankyo Europe GmbH. Esperion Therapeutics, Inc. was incorporated in 2008 and is headquartered in Ann Arbor, Michigan.
Precigen
NASDAQ:PGENPrecigen, Inc. operates as a discovery and clinical-stage biopharmaceutical company that develops gene and cell therapies using precision technology to target diseases in therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. It operates through two segments, Biopharmaceuticals and Exemplar. The company offers therapeutic platforms consisting of UltraCAR-T to provide chimeric antigen receptor T cell therapies for cancer patients; AdenoVerse immunotherapy, which utilizes a library of proprietary adenovectors for gene delivery of therapeutic effectors, immunomodulators, and vaccine antigen; and ActoBiotics for specific disease modification. It also develops programs based on the UltraCAR-T platform, including PRGN-3005 in Phase 1b clinical trial to treat advanced ovarian, fallopian tube, or primary peritoneal cancer; PRGN-3006 in Phase 1b trial for patients with relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndromes; and PRGN-3007 in Phase 1/1b trial for the treatment of advanced receptor tyrosine kinase-like orphan receptor 1-positive, hematologic, and solid tumors. In addition, the company is developing programs based on the AdenoVerse immunotherapy platform comprising PRGN-2009 in Phase 2 trial for patients with HPV-associated cancer; and PRGN-2012 in Phase ½ trial to treat recurrent respiratory papillomatosis, as well as AG019, which is based on the ActoBiotics platform and in Phase 1b/2a trial, to treat type 1 diabetes mellitus. Further, it provides UltraPorator, a proprietary electroporation device; and develops research models and services for healthcare research applications. The company was formerly known as Intrexon Corporation and changed its name to Precigen, Inc. in February 2020. Precigen, Inc. was founded in 1998 and is headquartered in Germantown, Maryland.
Rani Therapeutics
NASDAQ:RANIRani Therapeutics Holdings, Inc. operates as a clinical stage biotherapeutics company that develops orally administered biologics. The company develops the RaniPill capsule, a drug-agnostic oral delivery platform to deliver a variety of drug substances, including large molecules, such as peptides, proteins, and antibodies. Its product pipeline includes RT-101, an octreotide, which has completed Phase I clinical trial for the treatment of neuroendocrine tumors and acromegaly; RT-102, a parathyroid hormone that is in Phase I clinical trial for the treatment of osteoporosis; RT-105, an anti-TNF-alpha antibody that is in preclinical studies to treat psoriatic arthritis; RT-110, a parathyroid hormone for the treatment of hypoparathyroidism; and RT-111, an ustekinumab biosimilar for the treatment of various inflammatory conditions. The company was incorporated in 2012 and is headquartered in San Jose, California.