North American CF Conference 2021

In November, Prof. McNally gave an update at the North American CF conference on how the RECOVER study has been progressing for the 12+ cohort, and presented some of the results seen in the first 6-month of the study. Recruitment commenced in September 2020. 116 PEOPLE WITH CF have been recruited to the study with 10 withdrawals. Ages of participants range from 12-57 years, with 55.4% male and 45.6% female. We have seen a significant and sustained reduction in sweat chloride in both homozygous and het/min patients, which compliments the clinical trial data published for Kaftrio®. We note significant increases in weight from baseline to 6-months, and significant increases in ppFEV1 from baseline to 6-months for all participants.

 A second poster was presented at this conference by our collaborator Jochen Mainz, where they showed a significant reduction in abdominal symptoms assessed using the CF abdominal questionnaire administered as part of the RECOVER study over 4-weeks of treatment with Kaftrio®. Using this patient reported outcome measure, there was a significant reduction in gastroesophageal reflux (-32%), pain (-31%), impaired quality of life (-41%), disorders of appetite (-36%) and the total CFAbd-score (-22%) over a 4-week period.