Cyclerion Therapeutics, Inc.

Akebia Therapeutics, Inc., established in 2007 and headquartered in Cambridge, Massachusetts, is a biopharmaceutical company focused on discovering and commercializing therapies for patients suffering from kidney diseases. The firm's leading experimental drug, vadadustat, is an oral treatment currently in Phase III clinical trials. Its purpose is to address anemia resulting from chronic kidney disease (CKD) in adult patients, encompassing both those dependent on dialysis and those who are not. Akebia also markets Auryxia, a ferric citrate product used to manage serum phosphorus levels in adult CKD patients undergoing dialysis, and to treat iron deficiency anemia in adult CKD patients who are not on dialysis. The company has several key collaboration agreements: with Otsuka Pharmaceutical Co. Ltd. for vadadustat's development and commercialization across major regions including the United States, European Union, Russia, China, Australia, Canada, and the Middle East; with Mitsubishi Tanabe Pharma Corporation for vadadustat in Japan and other Asian territories; and a research and licensing deal with Janssen Pharmaceutica NV pertaining to hypoxia-inducible factor prolyl hydroxylase targeted compounds globally.

Iterion TherapeuticsBiotechUnited States

68% match

Iterion Therapeutics is a clinical-stage biopharmaceutical company headquartered in Houston, Texas. Founded in 2015, the company is dedicated to revolutionizing healthcare through novel drug discovery and innovation.

PrileniaBiotechUnited States

68% match

Prilenia Therapeutics is a clinical-stage biotechnology company developing treatments for neurodegenerative and neurodevelopmental disorders. Its lead drug candidate, pridopidine, has been studied in Huntington’s disease and amyotrophic lateral sclerosis (ALS), serving patients with serious neurological conditions.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical firm committed to developing life-improving treatments for patients in the United States afflicted by genetically defined diseases with significant unmet medical needs. Its leading investigational therapies include losmapimod, a small molecule targeting facioscapulohumeral muscular dystrophy, and FTX-6058, an orally administered fetal hemoglobin inducer for sickle cell disease and related hemoglobinopathies, such as beta-thalassemia. Beyond these, Fulcrum is actively engaged in discovering new drug targets for various rare conditions, encompassing neuromuscular, muscular, central nervous system, and hematologic disorders, alongside cardiomyopathies and pulmonary diseases. The company has established key collaborations, notably a research and discovery partnership with Acceleron Pharma Inc. to identify biological targets within the pulmonary disease area, and a strategic licensing and development agreement with MyoKardia, Inc. focused on novel targeted therapies for genetic cardiomyopathies. Incorporated in 2015, Fulcrum Therapeutics, Inc. maintains its headquarters in Cambridge, Massachusetts.

Praxis Precision Medicines, Inc. is a biopharmaceutical firm operating at the clinical stage, dedicated to creating innovative treatments for central nervous system (CNS) conditions that stem from an imbalance in neuronal activity. The company's primary investigational compounds currently undergoing evaluation include: PRAX-114, an extrasynaptic-preferring positive allosteric modulator of the GABAA receptor, which is currently in Phase IIa clinical trials for addressing both major depressive disorder and perimenopausal depression. Additionally, PRAX-944, a small molecule designed to selectively inhibit T-type calcium channels, is also progressing through Phase IIa clinical trials to manage essential tremor. Beyond these, Praxis is also advancing a pipeline of other promising drug candidates, such as: PRAX-562, a persistent sodium current blocker, undergoing Phase I clinical evaluation for treating severe pediatric epilepsy and adult cephalgia (headaches). PRAX-222, an antisense oligonucleotide (ASO) aimed at individuals suffering from gain-of-function (GOF) SCN2A epilepsy. And a program targeting KCNT1 for the treatment of KCNT1 GOF epilepsy. To further its research and development efforts, Praxis maintains several strategic alliances. These include a cooperation and licensing arrangement with RogCon Inc.; a licensing deal with Purdue Neuroscience Company; a comprehensive research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and a collaborative project with The Florey Institute focused on the creation of three new antisense oligonucleotides (ASOs). Established in 2015, the company's headquarters are situated in Boston, Massachusetts.

Amylyx Pharmaceuticals, Inc. operates as a clinical-stage biopharmaceutical firm, primarily focused on creating therapies for amyotrophic lateral sclerosis (ALS) and various other neurodegenerative conditions. A key asset in its developmental pipeline is AMX0035, a proprietary dual UPR-Bax apoptosis inhibitor. This compound, which integrates sodium phenylbutyrate and taurursodiol, is under investigation for treating ALS, and its application is also being explored for other neurodegenerative diseases. The company was established in 2013 and is based in Cambridge, Massachusetts.