CF RESEARCH UPDATE

The Cystic Fibrosis Foundation is excited to share the latest developments in CF research, and there is much to celebrate as we head into the holidays. We continue to focus on advancing new therapies for all people with CF.

Modulator Therapies

In September, the U.S. Food and Drug Administration (FDA) approved Orkambi® for children with CF ages 1 to 2 years who have two F508del mutations. The FDA is scheduled to announce its decision on whether to approve Trikafta® for children with CF ages 2 – 5 in 2023.

There is also progress on the development of new and potentially better modulator therapies:

• AbbVie is enrolling patients in a Phase 2 trial of its potential triple-combination modulator, galicaftor/navocaftor/ABBV-576.
• Earlier this year, the CF Foundation provided $5 million to Sionna Therapeutics to develop new modulators. After reporting promising preclinical results in November, Sionna is making plans to start early-phase clinical trials.
• In 2023, Vertex Pharmaceuticals will continue late-stage clinical trials on its new once-a-day, triple-combination modulator VX-121/tezacaftor/deutivacaftor.

Development of Treatments for People with CF Who Can’t Take Modulators

There are also exciting developments in genetic therapies — gene therapy and messenger RNA (mRNA) therapy — for all people with CF and an oligonucleotide therapy for people with CF with a type of splicing mutation. Several trials are either underway or are starting next year:  

• 4D Molecular is enrolling patients in an ongoing Phase 1/2 trial of its inhaled gene therapy. In promising interim clinical results, 4D Molecular demonstrated safety and evidence of delivery and expression of the CFTR gene in lung tissue samples from three patients.
• Krystal Biotech plans to start a Phase 1 clinical trial for its inhaled gene therapy candidate in 2023.
• Vertex is planning to start a Phase 1 clinical trial for its mRNA therapy in the coming weeks.
• The Foundation awarded up to $5 million in October to Carmine Therapeutics to continue lab research of a method to deliver a potential gene therapy to people with CF.
• SpliSense has started a Phase 1/2 clinical trial of its antisense oligonucleotide drug for the treatment of people with CF who have the splicing mutation 3849+10Kb C-to-T. If successful, the drug could potentially validate other similar therapeutic approaches in SpliSense’s pipeline that address rare mutations in CF.

Simplify Study Results

In November, researchers reported promising results of the Simplify study, which showed that people with CF ages 12 and older in the trial did not experience a change in lung function when they stopped taking either hypertonic saline or dornase alfa (Pulmozyme®) while on Trikafta. Researchers noted that participants in the trial had high lung function when the study began and that the trial was for only six weeks. Several other studies assessing longer-term effects on health are underway to provide additional insight into the implications of stopping medications and therapies while on Trikafta, including HERO-2 and CF-STORM.

To learn more about cystic fibrosis research, you can watch recordings — including the plenaries and symposia — of the North American Cystic Fibrosis Conference on our YouTube channel. To get a summary of the top research and care news, watch the NACFC Highlights Webinar.

The Cystic Fibrosis Foundation periodically sends out information regarding our infection research efforts. To learn more about drugs in development and current studies, please check out the links below.

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