SION-719 + Trikafta for Cystic Fibrosis

(PreciSION CF Trial)

Recruiting at 12 trial locations

Overseen By VP, Head of Medical Affairs & Patient Advocacy

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sionna Therapeutics Inc.

Must be taking: Trikafta

Disqualifiers: Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

16 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called SION-719 for individuals with cystic fibrosis (CF) who are already taking Trikafta. The goal is to assess the safety, tolerability, and behavior of SION-719 in the body. Participants will take SION-719 and a placebo (a harmless pill resembling the treatment) at different times while maintaining their usual Trikafta regimen. Suitable candidates have CF with a specific genetic marker (F508del homozygous) and have been taking Trikafta as directed for at least three months without recent flare-ups. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

You will not have to stop taking Trikafta, as the trial is for people already on this medication. The protocol does not specify about other medications, so it's best to discuss with the trial team.

Is there any evidence suggesting that SION-719 is likely to be safe for humans?

Research has shown that SION-719, a new treatment being tested for cystic fibrosis, was safe in earlier studies. Participants generally tolerated SION-719 well, with no major side effects reported, and it reached the desired levels in the body.

This trial tests SION-719 in combination with Trikafta, an already approved treatment for cystic fibrosis. The good tolerance of SION-719 in earlier studies is a positive sign for its safety. As with any treatment, researchers will continue monitoring to ensure it remains safe for participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cystic fibrosis?

Researchers are excited about SION-719 for cystic fibrosis because it offers a fresh approach compared to current options like Trikafta. Unlike most treatments that work by improving the function of defective CFTR proteins, SION-719 targets different cellular pathways, potentially enhancing the effectiveness of existing therapies. This new mechanism of action could lead to better outcomes for patients by addressing the condition from another angle, which is why it's generating buzz in the medical community.

What evidence suggests that SION-719 might be an effective treatment for cystic fibrosis?

Research shows that SION-719 is a promising new treatment for cystic fibrosis. It stabilizes a part of the CFTR protein, which often malfunctions in people with this condition. Early studies found that SION-719 was generally well tolerated, with most participants not experiencing serious side effects. In this trial, participants will receive SION-719 in one treatment period and a placebo in another, while continuing their physician-prescribed Trikafta as background therapy. The treatment aims to improve CFTR protein function, potentially reducing cystic fibrosis symptoms. Although final results are still being gathered, initial findings offer hope for those seeking new management options. ^{1 2 3 5 6}

Who Is on the Research Team?

Medical Director

Principal Investigator

Sionna Therapeutics Inc.

Are You a Good Fit for This Trial?

This trial is for people with Cystic Fibrosis who have been taking Trikafta as prescribed for at least 3 months. They should be stable, without a lung flare-up in the last 28 days or any other serious illness in the last 14 days. Participants must have a specific CF gene mutation (F508del homozygous). Those with significant illnesses besides CF or a history of cancer (with some skin cancer exceptions) cannot join.

Inclusion Criteria

I have been taking Trikafta as prescribed for at least 3 months.

My cystic fibrosis has been stable, with no lung infections in the last 28 days.

I have cystic fibrosis with two copies of the F508del mutation.

Exclusion Criteria

I have a serious illness right now besides cystic fibrosis.

I had cancer before, but it was only skin cancer that was removed and hasn't come back in over a year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive SION-719 or placebo in a crossover design while continuing Trikafta

8 weeks

Multiple visits for drug administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• SION-719

Trial Overview The study tests SION-719's safety and effects when added to Trikafta treatment for Cystic Fibrosis. Some participants will receive SION-719 while others get a placebo, allowing comparison between the two groups.

How Is the Trial Designed?

Group I: Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719 Experimental Treatment 2 Interventions

Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.

Group II: Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719 Experimental Treatment 2 Interventions

Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study.

Who Is Running the Clinical Trial?

Sionna Therapeutics Inc.

Lead Sponsor

Citations

1. investors.sionnatx.com investors.sionnatx.com/news-releases/news-release-details/sionna-therapeutics-announces-initiation-precision-cf-phase-2a

Release Details - Investor Relations - Sionna Therapeutics, Inc SION-719 is the first NBD1 stabilizer being evaluated in people living with cystic fibrosis. Topline data expected in mid-2026.

2. biospace.com biospace.com/press-releases/sionna-therapeutics-reports-third-quarter-2025-financial-results

Sionna Therapeutics Reports Third Quarter 2025 Financial ... The trial will evaluate the safety, tolerability, and pharmacokinetics (PK) of SION-719 when administered with SOC and assess change in CFTR ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT07108153

NCT07108153 | Proof-of-concept Study to Evaluate the ... The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SION-719 when given to people with CF who are ...

4. sionnatx.gcs-web.com sionnatx.gcs-web.com/news-releases/news-release-details/sionna-therapeutics-announces-presentation-sion-719-and-sion-451

Release Details - Investor Relations - Sionna Therapeutics, Inc Phase 1 data of first-in-class NBD1 stabilizers, SION-719 and SION-451, demonstrated they were generally well tolerated and exceeded desired ...

5. respiratory-therapy.com respiratory-therapy.com/disorders-diseases/chronic-pulmonary-disorders/cystic-fibrosis/first-patients-dosed-two-phase-1-trials-novel-cystic-fibrosis-treatments/

First Patients Dosed in Trials for Novel Cystic Fibrosis ... The trials will evaluate SION-719 and SION-451, two novel NBD1 stabilizers aimed at improving CFTR protein function in cystic fibrosis patients.

6. firstwordpharma.com firstwordpharma.com/story/6516140

Sionna Therapeutics Reports Third Quarter 2025 Financial ... As previously disclosed, in these Phase 1 trials, both SION-719 and SION-451 were generally well tolerated and exceeded target exposure levels.