CAEL-101 is under clinical development by Alexion Pharmaceuticals and currently in the Phase III in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect CAEL-101’s likelihood of approval (LoA) and phase transition for Amyloidosis took place on 13 Oct 2021, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their CAEL-101 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

CAEL-101 overview

CAEL-101 is under development for the treatment of relapsed or refractory amyloid light chain (AL) amyloidosis. The therapeutic candidate is administered as intravenous infusion. It is chimerized 11-F4 MAb (a murine amyloid-reactive monoclonal antibody, IgG1k) that acts by targeting amyloid related, conformational epitope on light chain (LC) related fibrils.

Alexion Pharmaceuticals overview

Alexion Pharmaceuticals (Alexion) discovers, develops, and markets therapies based on complement biology and inhibition for rare diseases. Deep understanding of rare disease enables the company to innovate and enter new areas, where there is a great unmet medical need. The company’s portfolio spans complement inhibitors for the treatment of patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), paroxysmal nocturnal hemoglobinuria (PNH), and neuromyelitis optica spectrum disorder (NMOSD) in anti-aquaporin-4 (AQP4) antibody positive patients. The company also offers novel enzyme replacement therapies for patients with lysosomal acid lipase deficiency (LAL-D), hypophosphatasia (HPP), and ultra-rare metabolic disorders. Alexion is headquartered in New Haven, Connecticut, the US.

Quick View CAEL-101 LOA Data

Report Segments
  • Innovator
Drug Name
  • CAEL-101
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Metabolic Disorders
Key Developers
Highest Development Stage
  • Phase III

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.