European CF Society Conference 2022

In June 2022, Prof. McNally presented the 12 month RECOVER data from the 12+ patients at the ECFS Conference in Rotterdam. This data showed that ELX/TEZ/IVA is associated with significant and sustained improvements in an array of outcome measures in people with CF. A significant proportion (40%) of people in the homozygous deltaF508 group had a sweat chloride in the normal range on treatment. Data collection is continuing for RECOVER and we hope to present more findings and study updates at future conferences.

Another poster was presented at the ECFS by our project manager Karen Lester on behalf of Sharon Sutton. This poster looked at the medication adherence of patients who commenced on ELX/TEZ/IVA from baseline to 6-months on treatment. This data showed that self reported questionnaires and medication pick up rates overestimate treatment adherence when compared with Medication Electronic Monitoring System (MEMS®). Data collection is ongoing for the 12+ cohort, with recruitment of the 6+ anticipated over the coming months.

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.