Summary

Cystic fibrosis (CF) is a progressive genetic condition and is the result of a patient inheriting a faulty cystic fibroses transmembrane conductance regulator (CFTR) gene from both parents. The defective CFTR genes produce faulty CFTR proteins on the body’s epithelial cells, and result in the buildup of a thick and sticky mucus in the lungs and pancreas. Historically therapeutic options have focused heavily on treating the symptoms, but despite being clinically and commercially successful have failed to address the faulty CFTR proteins that cause the disease. This unmet need has led to the development of CFTR modulators.

The treatment algorithm patients receive is likely to consist of an antimicrobial, a mucolytic agent, pancreatic enzymes and possibly a CFTR modulator. This tabular heatmap framework, designed to provide an easily digestible summary of these clinical characteristics, provides detailed information on all late-stage clinical trial results for products in the CF market and Phase III or Preregistration stages of the pipeline. These are split along drug class lines, and are therefore reflective of the treatment algorithm.

All safety and efficacy endpoints reported in these trials are displayed. In addition, key study characteristics such as the size, composition and patient segment of the study population are provided. These results are presented in a visually accessible, color-coded manner in order to maximize ease of use.

The accompanying text provides a detailed analysis of the clinical benchmarks set by the current market landscape, and the anticipated changes to these benchmarks, and to the treatment algorithm, as a result of the late-stage pipeline.

Scope

- To what extent is the CF market landscape expected to change with the introduction of new CFTR modulators?
- What are the clinical characteristics of currently approved therapies for CF, in terms of specific safety and efficacy parameters?
- What are the key unmet needs in this indication, and which clinical safety and efficacy parameters are the most closely linked to them?
- How will current late-stage CFTR modulators affect the market for symptomatic therapies, and are they able to yield comparable clinical efficacy results to Kalydeco?
- How will the influx of tobramycin generics affect the competitive landscape?

Reasons to buy

- Understand the current clinical landscape by considering the treatment options available for each patient segment.
- Visually compare the currently approved treatments available at each line of therapy, based on the most important efficacy and safety parameters tested in clinical trials.
- Assess the current late-stage pipeline, in terms of the likely positioning of each product and the implications for the clinical landscape at each line of therapy.
- Understand the relative strengths and weaknesses of the studies used to gather these data.
- Build up a nuanced understanding of the clinical benchmarks set by these products, and consider how the current late-stage pipeline will affect these benchmarks.
- Assess your own pipeline programs in light of these benchmarks in order to optimally position them and maximize uptake by clinicians.