Axatilimab for Pulmonary Fibrosis

Recruiting at 113 trial locations

Overseen By Syndax Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Syndax Pharmaceuticals

Must not be taking: Nintedanib, Pirfenidone, Corticosteroids, others

Disqualifiers: Emphysema, Connective tissue disease, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 13

Duration: 26 weeks

135 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness and safety of axatilimab for individuals with idiopathic pulmonary fibrosis (IPF), a lung condition that causes scarring and breathing difficulties. Participants will be divided into two groups: one will receive axatilimab, and the other will receive a placebo, both administered every two weeks for 26 weeks. Suitable candidates for this trial include those diagnosed with IPF who have undergone a high-resolution chest scan within the past year. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant advancements in IPF treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as nintedanib with pirfenidone, systemic corticosteroids over 10 mg/day, and several other specific drugs, at least 4 weeks before screening. It's best to discuss your current medications with the trial team to see if any need to be stopped.

Is there any evidence suggesting that axatilimab is likely to be safe for humans?

Research has shown that axatilimab is generally safe. In earlier studies involving patients with chronic graft-versus-host disease, most participants tolerated the treatment well, experiencing no serious side effects. Another study tested various doses of axatilimab and found that, although some side effects occurred, most patients managed them effectively.

Axatilimab is still under investigation for idiopathic pulmonary fibrosis (IPF), but these earlier studies suggest it is likely safe for individuals. However, individual experiences may vary. It is important to discuss potential risks with a doctor before joining a trial. ^{1 2 3 4 5}

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

Most treatments for pulmonary fibrosis work by reducing inflammation or slowing disease progression but often come with significant side effects. Axatilimab stands out because it targets a specific receptor called CSF-1R, which plays a key role in regulating immune cells involved in fibrosis. This targeted approach could potentially reduce lung scarring more effectively with fewer side effects compared to current options like corticosteroids or antifibrotic drugs. Researchers are excited about axatilimab because it offers a novel mechanism of action, potentially leading to better outcomes and improved quality of life for patients.

What evidence suggests that axatilimab might be an effective treatment for pulmonary fibrosis?

Research has shown that axatilimab, which participants in this trial may receive, may help treat conditions like pulmonary fibrosis. In earlier studies, 82% of patients responded well to axatilimab within the first six treatment cycles. Additionally, about 60% of patients maintained their positive response for up to 12 months. These results suggest that axatilimab could effectively manage symptoms and slow the progression of pulmonary fibrosis. ^{1 2 3 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Syndax Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for people with Idiopathic Pulmonary Fibrosis (IPF), a type of lung scarring. Participants must have an FVC (a measure of lung function) at or above 45% of the expected value, a confirmed diagnosis following specific guidelines, and recent chest scans that meet study standards. They should also have certain levels of other breathing measures within set limits.

Inclusion Criteria

I have been diagnosed with IPF according to the 2018 guidelines.

FVC ≥45% of predicted normal at Screening Visits

Forced expiratory volume in 1 second (FEV1)/FVC ≥0.7 at Screening Visits

Exclusion Criteria

I am currently taking nintedanib and pirfenidone together.

I am currently pregnant or breastfeeding.

Participants who cannot meet protocol-specified baseline stability criteria

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive axatilimab or placebo every 2 weeks during the 26-week Treatment Period

26 weeks

13 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Axatilimab

Trial Overview The MAXPIRe study is testing Axatilimab against a placebo to see if it's effective and safe for treating IPF. Participants will be randomly assigned to receive either the actual medication or a placebo in order to compare outcomes between the two groups.

How Is the Trial Designed?

Group I: Axatilimab Experimental Treatment 1 Intervention

Participants will receive axatilimab every 2 weeks during the 26-week Treatment Period.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo every 2 weeks during the 26-week Treatment Period.

Axatilimab is already approved in United States for the following indications:

Approved in United States as Axatilimab for:

• Chronic Graft-Versus-Host Disease

Who Is Running the Clinical Trial?

Syndax Pharmaceuticals

Lead Sponsor

Trials

49

Recruited

2,700+

DevPro Biopharma

Collaborator

Trials

5

Recruited

800+

DevPro Biopharma

Industry Sponsor

Trials

5

Recruited

800+

Published Research Related to This Trial

In a phase II trial involving 178 adults with idiopathic pulmonary fibrosis, macitentan did not show a significant improvement in forced vital capacity compared to placebo after 12 months, indicating it may not be effective for this condition.

Both macitentan and placebo groups had similar rates of elevated liver enzymes, suggesting that macitentan is well tolerated and has a safety profile comparable to placebo.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Macitentan for the treatment of idiopathic pulmonary fibrosis: the randomised controlled MUSIC trial. Raghu, G., Million-Rousseau, R., Morganti, A., et al.[2014]

Astilbin has shown promising antifibrotic effects by inhibiting the proliferation and migration of cells involved in pulmonary fibrosis, as demonstrated in both in vitro and in vivo studies.

The mechanism of action for astilbin involves blocking the Hedgehog signaling pathway, which is linked to the regulation of fibrosis-associated genes, suggesting its potential as a therapeutic option for treating lung fibrosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Astilbin ameliorates pulmonary fibrosis via blockade of Hedgehog signaling pathway. Zhang, J., Liu, H., Song, C., et al.[2019]

The BUILD-1 study suggests that bosentan, an oral dual endothelin receptor antagonist, may help delay disease progression and improve quality of life in patients with idiopathic pulmonary fibrosis (IPF), particularly in those with biopsy-confirmed diagnoses.

While bosentan showed promising trends in efficacy, it is important to note that IPF currently has no known effective therapy, and further investigation is ongoing in the BUILD-3 trial to confirm these findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Bosentan for idiopathic pulmonary fibrosis. King, TE.[2018]

Idiopathic pulmonary fibrosis (IPF) is a severe lung disease with a median survival of only about 3 years after diagnosis, highlighting the urgent need for effective treatments.

Bosentan, an endothelin receptor antagonist, did not show overall benefit in a recent trial for IPF, but a post hoc analysis suggests it may be effective in a specific subgroup of patients with biopsy-proven IPF and minimal honeycombing, warranting further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of bosentan for idiopathic pulmonary fibrosis. Swigris, JJ., Brown, KK.[2010]

Pirfenidone is an oral antifibrotic medication that has been shown to slow the progression of idiopathic pulmonary fibrosis (IPF), a serious and often fatal lung disease.

This treatment improves progression-free survival for patients with IPF, providing a new therapeutic option in a condition that previously had very limited treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pirfenidone Initiates a New Era in the Treatment of Idiopathic Pulmonary Fibrosis. Selvaggio, AS., Noble, PW.[2016]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11713947/

Axatilimab - PMC Results · Overall response rate in the first 6 cycles was 82% (18 of 22 patients; 95% CI: 60%-95%) in the phase 2 cohort and 67% (26 of 39 ...

2. investor.incyte.com investor.incyte.com/news-releases/news-release-details/incyte-and-syndax-present-additional-data-positive-agave-201

Press Releases Patients in this cohort experienced a median time to response to axatilimab of 1.7 months (0.9-8.1), and 60% of patients maintained a response ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06132256

NCT06132256 | MAXPIRe: Study to Evaluate Axatilimab in ... The study will evaluate the efficacy and safety of axatilimab in participants with IPF. Official Title. A 26-Week, Randomized, Double-Blind, Placebo-Controlled, ...

4. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/1/499054/Safety-and-Efficacy-of-Axatilimab-at-3-Different

Safety and Efficacy of Axatilimab at 3 Different Doses in ... Clinical benefit, as measured by reduction in mLSS score, was reported in 55%, 54%, and 36% of patients in the 0.3 mg/kg Q2W, 1 mg/kg Q2W, and 3 ...

5. ir.syndax.com ir.syndax.com/news-releases/news-release-details/syndax-announces-new-data-secondary-analysis-pivotal-agave-201

Syndax Announces New Data from Secondary Analysis of the ... Of the patients who had a response, an estimated 60% of patients maintained a response at 12 months (measured from first response until new systemic therapy or ...

6. ascopubs.org ascopubs.org/doi/10.1200/JCO.22.00958

Axatilimab for Chronic Graft-Versus-Host Disease After ... Targeting profibrotic macrophages with axatilimab is a therapeutically promising novel strategy with a favorable safety profile for refractory cGVHD.

7. actionpf.org actionpf.org/research-study/maxpire-a-study-looking-at-the-impact-of-axatilimab-for-people-with-idiopathic-pulmonary-fibrosis-ipf

MAXPIRe: A study looking at the impact of Axatilimab for ... Axatilimab is a drug, which is being tested in participants with IPF. The study will assess the safety and effectiveness of axatilimab in IPF participants.