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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
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.
Leniolisib is an oral small molecule phosphoinositide 3-kinase delta inhibitor approved in the US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta syndrome in adult and pediatric patients 12 years of age and older.
Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with APDS.
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton.
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