LB-001 is under clinical development by Alexion Pharmaceuticals and currently in Phase II for Methylmalonic Acidemia. According to GlobalData, Phase II drugs for Methylmalonic Acidemia does not have sufficient historical data to build an indication benchmark PTSR for Phase II. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the LB-001 LoA Report. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
LB-001 overview
LB-001 is under development for the treatment of methylmalonic acidemia (MMA). It is administered through intravenous route. It is a recombinant adeno-associated virus (AAV) vector with human methylmalonyl-COA mutase (MUT) gene acts by targeting coagulation factor IX and methylmalonyl CoA isomerase. It is developed based on generide technology. It was also under development for Hemophilia B.
Alexion Pharmaceuticals overview
Alexion Pharmaceuticals (Alexion), a subsidiary of AstraZeneca Plc, discovers, develops and markets therapies for severe and life-threatening diseases. The company’s pipeline products include ALXN2030, ALXN1920, ULTOMIRIS, Vemircopan for nephrology; ALXN2220, Anselamimab, Acoramidis treats cardiology; ALXN1910 which targets bone metabolism; Gefurulimab for neurology; Danicopan targets ophthalmology. The company’s medicinal brands comprise Kanuma, Koselug, Soliris, StrensIQ, Ultomiris and Voydeya. Alexion collaborates with various research organizations and pharmaceutical companies. The company operates in Europe and the Middle East, North America, Latin America and Asia Pacific. Alexion is headquartered in Boston, Massachusetts, the US.
For a complete picture of LB-001’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
