AC Immune
NASDAQ:ACIUAC Immune SA, a clinical stage biopharmaceutical company, discovers, designs, and develops medicines and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding. Its SupraAntigen and Morphomer platforms are designed to generate vaccines, antibodies, and small molecules, which selectively interact with misfolded proteins that are common in a range of neurodegenerative diseases. The company is developing Crenezumab, a humanized, conformation-specific monoclonal antibody, which is in Phase II clinical studies trial for the treatment of Alzheimer's disease (AD). It is also developing ACI-24, an anti-Abeta vaccine candidate that is in Phase II clinical study for AD, as well as completed Phase Ib clinical study for Down syndrome; ACI-35, an anti-Tau vaccine candidate that is in Phase Ib/2a clinical study; and Tau- positron emission tomography (PET) imaging tracer, which is in Phase II clinical study. In addition, the company is researching and developing small molecule Tau aggregation inhibitors for AD and NeuroOrphan indications. Further, it has discovery and preclinical stage molecules targeting range of neurodegenerative diseases, which include diagnostics targeting TDP-43, alpha-synuclein, and NLRP3. AC Immune SA has license agreements and collaborations with Genentech, Inc.; Biogen International GmbH; Janssen Pharmaceuticals, Inc.; Life Molecular Imaging SA; Eli Lilly and Company; and WuXi Biologics. The company was incorporated in 2003 and is headquartered in Lausanne, Switzerland.
BioDelivery Sciences International
NASDAQ:BDSIBioDelivery Sciences International, Inc., a specialty pharmaceutical company, engages in the development and commercialization of pharmaceutical products for chronic conditions in the United States and internationally. The company provides its products based on its patented BioErodible MucoAdhesive drug-delivery technology, a small erodible polymer film for application to the buccal mucosa, as well as other drug delivery technologies. It offers BELBUCA, a buprenorphine buccal film for the treatment of chronic pain; and Symproic, a peripherally acting mu-opioid receptor antagonist for the treatment of opioid-induced constipation in adult patients with chronic non-cancer pain, including patients with chronic pain related to prior cancer or its treatment who do not require frequent opioid dosage escalation. The company also provides ELYXYB, a celecoxib oral solution for the treatment of migraine with or without aura in adults; and ONSOLIS, a fentanyl buccal soluble film for the management of breakthrough pain in cancer patients. BioDelivery Sciences International, Inc. was incorporated in 1997 and is headquartered in Raleigh, North Carolina. As of March 18, 2022, BioDelivery Sciences International, Inc. operates as a subsidiary of Collegium Pharmaceutical, Inc.
Catalyst Pharmaceuticals
NASDAQ:CPRXCatalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.
Magenta Therapeutics
NASDAQ:MGTAMagenta Therapeutics, Inc., a biotechnology company focus on improving stem cell transplantation. Its product candidates are designed to bring the curative power of stem cell transplant to patients with blood cancers, genetic diseases, and autoimmune diseases. The company's product portfolio includes MGTA-117, an anti-CD117 antibody conjugated to an amanitin payload that targets hematopoietic stem cells (HSCs) and leukemia cells; and MGTA-45, an anti-human CD45 antibody conjugated to a DNA-interacting payload for HSCs, leukemia cells, and immune cells. In addition, it focus on development of ADC-based conditioning program that targets a receptor of T cells; and novel conditioning and translation stem cell sciences, as well as develops cell therapy program, E478, which is a small molecule aryl hydrocarbon receptor antagonist for stem cell-based gene therapy and genome editing. In addition, the company has a research and clinical collaboration agreement with AVROBIO, Inc. for the treatment of lysosomal storage disorders; and Beam Therapeutics, Inc. to evaluate the potential utility of MGTA-117 for conditioning of patients with sickle cell disease and beta-thalassemia. Magenta Therapeutics, Inc. was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Pandion Therapeutics
NASDAQ:PANDPandion Therapeutics, Inc., a clinical stage biopharmaceutical company, develops various therapeutics to address the unmet needs of patients suffering from autoimmune diseases. Its lead product candidate is PT101, an effector module comprising an engineered variant of wild-type interleukin-2 (IL-2) fused to a protein backbone that is in Phase 1a clinical trials for the treatment of various autoimmune and inflammatory diseases. The company's other product candidates comprise PT627, a systemic PD-1 agonist that is in preclinical studies for the treatment of autoimmune diseases; PT001, a bifunctional molecule combining its PD-1 agonist effector with a tether module that binds to mucosal vascular addressing cell adhesion molecule (MAdCAM) to drive tissue-selective immunomodulation in the gastrointestinal tract; and PT002, a tethered bifunctional molecule that utilizes its IL-2 mutein effector module coupled with a MAdCAM-tether module to deliver the IL-2 mutein effector to the gastrointestinal tract where MAdCAM is expressed. It has a license and collaboration agreement with Astellas Pharma Inc. to discover and develop novel compounds for autoimmune diseases of the pancreas, including type 1 diabetes. The company was formerly known as Immunotolerance, Inc. and changed its name to Pandion Therapeutics, Inc. in June 2017. Pandion Therapeutics, Inc. was incorporated in 2016 and is headquartered in Watertown, Massachusetts.