Unique and innovative solution
A novel CFTR triple modulator therapy combined with organoids as patients-in-the-lab
A novel CFTR triple modulator therapy in an advanced stage of development
Laboratory-grown organoids as
patients-in-the-lab to de-risk development and select responders to our treatment in the lab
Our Compounds
We have three CFTR modulators that can repair the CFTR protein function in a unique manner and can restore CFTR function in a way that can hugely impact CF disease severity and prevent progression. The combination consists of a potentiator, a corrector and an amplifier. As a whole, the Fair TX drugs enhance the production of the mutant version of CFTR, and then restore the 3D shape of the mutant CFTR so it can function again and resolve and prevent disease progression.
These therapies have already demonstrated significant efficacy in F508del/F508del CF subjects in a phase 2a clinical trial and are now tested in a phase 2b clinical trial in subjects with ultra-rare mutations, preselected based on their response in organoids.
Our Personalized Approach
We use intestinal organoid cultures that are the best-developed organoid cultures till date. Intestinal organoids can be grown from rectal biopsies that can be easily isolated from people with CF (PwCF) of any age through a painless procedure. The biopsies can be shipped to centralized centers for organoid generation and testing. The feasibility of generating organoids from patients has been established for European CF patients through the HIT-CF project. This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 755021.
CFTR function can be measured in intestinal organoids through quantification of forskolin-induced swelling. This assay correlates with important disease indicators and treatment response to CFTR modulators. As such, the intestinal organoid FIS assay can be used to estimate individual CFTR function increase and clinical response before the actual initiation of treatment with CFTR modulators in vivo. The EMA supports a further development of organoids for this specific application.
CHOICES Clinical trial (phase 2b)
In June 2024 the first subject was enrolled in our phase 2b (CHOICES, NCT06468527) clinical trial. The trial evaluates the efficacy and safety of Dirocaftor/Posenacaftor/Nesolicaftor in adults with CF. The trial evaluates the predicted clinical effect of the CFTR modulators in subjects pre-identified by their organoid (companion diagnostic) response to the investigational products.
CHOICES will also aim to prove that the CFTR modulators are effective in ultra-rare mutations for which no treatment is currently available.
The trial has enrolled 40 PwCF (18+, rare mutations) from 10 European countries, reflecting a diverse population within ultra-rare CF cases.
The Phase IIb CHOICES trial is being executed by the HIT-CF Europe consortium, chaired by Fair TX Co-Founder and Advisor Prof. Kors van der Ent. The HIT-CF project is supported by the EU Horizon 2020 initiative.
Result are expected mid-2025.