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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
The net proceeds will be used to advance the clinical development of late-stage product ANX005, which is being evaluated for the treatment of guillain-barre syndrome.
MTP-131 (elamipretide) is an investigational mitochondrial protective agent which is readily penetrates cell membranes and transiently localizes to the inner membrane of the mitochondria where it interacts with cardiolipin and investigated in Patients with Barth syndrome.
Joenja (leniolisib) is an oral small molecule PI3K delta inhibitor and the first and only targeted treatment of activated phosphoinositide 3-kinase delta syndrome in 12 years of age and older. It is being developed for in children aged 4 to 11 years with APDS.
IB1001 (N-Acetyl-L-Leucine) is a calcium channel modulator. It is being evaluated in the phase III clinical trial studies for the treatment of Type C Niemann-Pick Disease.
The net proceeds will be used in the anticipated launch of AG10 (acoramidis HCl) as the potential backbone of therapy for transthyretin amyloid cardiomyopathy (ATTR-CM).
Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator indicated for the treatment of hemolytic anemia. It is under phase 3 clinical development for the treatment of non-transfusion-dependent alpha- or beta-thalassemia.
The combined company will focus on advancing Cyclo Therapeutics’ pivotal Phase 3 global study evaluating Trappsol Cyclo (hydroxypropyl beta cyclodextrin), an orphan drug designated product in the United States and Europe, for Niemann-Pick Disease Type C1 (NPC1).
Trappsol Cyclo is a proprietary formulation of hydroxypropyl beta cyclodextrin being developed in phase 3 clinical trials for the potential treatment of Niemann-Pick Disease Type C1, a rare and fatal genetic disease.
Danicopan is an investigational oral medicine in development as an add-on to C5 inhibitor therapy eculizumab or ravulizumab for patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular haemolysis.
FDA provides complete approval to enroll patients in its pivotal clinical trial to evaluate "MC-1" (a naturally occurring metabolite of pyridoxine) for treatment of a rare pediatric disease called pyridox(am)ine 5′-phosphate oxidase deficiency.
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