Case studies

Cystic Fibrosis clinical advisory board

Client: Proteostasis Therapeutics

Consultant: Professor Jane Davies, National Heart and Lung Institute

Expertise: Pharmaceuticals, Drug development, Cystic Fibrosis(CF), Clinical trials

Our consultants shared their expertise in Cystic fibrosis (CF), drug development and clinical trials to help Proteostasis Therapeutics assess the combination of compounds aimed at treatments to improve lung function.

 

The Challenge

Proteostasis is a drug discovery company addressing diseases caused by defects in protein folding, trafficking and clearance for orphan and neurodegenerative diseases, including Cystic Fibrosis (CF).

Proteostasis needed expert advice on the selection process of the company’s leading compounds aimed at improving lung function that are due for clinical development.

The compounds double the activity of the drug combination ivacaftor/lumacaftor in the gold-standard test using Human Bronchial Epithelial cells (HBE cell assay) which is a drug combination that has been shown to significantly improve lung function in sufferers with the most common CF mutation, F508del/F508del, and it was hoped that the compounds selected for further development can build on this.

 

The Solution

The work at hand required stratetic planning over a period of time and Professor Jane Davies, along with five other world leading experts, was appointed to Proteostasis Therapeutics’ Cystic Fibrosis clinical advisory board, which was given the task to serve as a strategic resource for the upcoming selection and study of the compounds.

 

The Impact

Following the appointment of experts on the board and the insights gained from their involvement, Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics said:

The knowledge and experience that our inaugural clinical advisory board members bring to Proteostasis will be invaluable not only in the selection process of which of our proprietary PTI CFTR compounds should proceed to clinical development, but also in providing guidance on the design and conduct of the future clinical trials of selected drug candidates.

Meenu Chhabra
President and CEO, Proteostasis Therapeutics

 

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