VENT-03 for Lupus

(AERIS Trial)

Recruiting at 9 trial locations

Overseen By Krista Miller

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ventus Therapeutics U.S., Inc.

Must be taking: SLE medications

Disqualifiers: Liver impairment, Drug-induced lupus, others

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

Compensation: Varies

Number of Visits: 3

Duration: 12 weeks

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VENT-03 to determine its effectiveness for people with cutaneous lupus erythematosus (CLE), a skin condition that causes rashes and lesions. Researchers aim to discover if VENT-03 can reduce CLE activity and severity and identify any side effects. Participants will take VENT-03 or a placebo (a look-alike pill with no active drug) daily for four weeks, then switch to VENT-03 for eight more weeks. Those diagnosed with CLE, confirmed by a prior biopsy, and with at least one active lupus lesion might be suitable for this trial. As a Phase 2 trial, this research measures how well VENT-03 works in an initial, smaller group, offering participants a chance to contribute to important medical advancements.

Will I have to stop taking my current medications?

The trial does not clearly specify if you need to stop your current medications. However, if you have a previous SLE diagnosis, you must be on stable doses of at least one specified SLE medication.

Is there any evidence suggesting that VENT-03 is likely to be safe for humans?

Research has shown that VENT-03 is safe and well-tolerated. In earlier studies, participants taking VENT-03 experienced no significant side effects. Lab tests, heart activity, and vital signs showed no major changes. This suggests that VENT-03 is generally safe for humans. Prospective trial participants can feel reassured by this safety information from previous research. ^{1 2 3 4 5}

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

Unlike the standard treatments for lupus, which often include drugs like hydroxychloroquine and corticosteroids, VENT-03 offers a new approach. Researchers are excited because VENT-03 works differently by targeting specific pathways involved in the immune response, potentially offering a more precise treatment. This could mean fewer side effects and improved effectiveness compared to current options. If successful, VENT-03 might provide a significant advancement in managing lupus symptoms and improving patients' quality of life.

What evidence suggests that VENT-03 might be an effective treatment for lupus?

Research has shown that VENT-03, which participants in this trial may receive, could help treat lupus. In studies with healthy individuals, VENT-03 proved safe and did not cause serious side effects. It effectively targeted the immune system areas involved in lupus. In studies with mice that had lupus, similar drugs to VENT-03 reduced inflammation and improved health. These early results suggest that VENT-03 might help manage lupus symptoms by lowering inflammation. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with active cutaneous lupus erythematosus (CLE), which may include those with systemic lupus erythematosus (SLE). Participants will be required to take a daily dose of the study drug or placebo and attend monthly clinic visits for checkups and tests.

Inclusion Criteria

I have a severe form of skin lupus with confirmed diagnosis and active lesions.

I have SLE, tested positive for ANA, meet the 2019 criteria, and am on stable SLE medication.

Exclusion Criteria

Meet protocol-specified infection or lab criteria; any other laboratory test results that, in the investigator's opinion, might place participant at unacceptable risk for participating in this study

Meets protocol specified exclusions related to concomitant medications

Diagnosis of select potentially confounding autoimmune disorders

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants take VENT-03 or a placebo every day for 4 weeks, then all participants switch to VENT-03 for another 8 weeks

12 weeks

3 visits (in-person, once a month)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• VENT-03

Trial Overview

The trial is testing VENT-03's effectiveness in treating CLE, comparing it against a placebo. Initially, participants receive either VENT-03 or placebo for 4 weeks; afterwards, all switch to VENT-03 for another 8 weeks to further evaluate its efficacy and safety.

How Is the Trial Designed?

Group I: VENT-03 Experimental Treatment 1 Intervention

VENT-03

Group II: Placebo Placebo Group 1 Intervention

Placebo

Who Is Running the Clinical Trial?

Ventus Therapeutics U.S., Inc.

Lead Sponsor

Trials

1

Recruited

30+

Citations

1.

jrheum.org

jrheum.org/content/52/Suppl_1/121.2

SAFETY, TOLERABILITY, PHARMACOKINETICS AND ...

VENT-03 was safe and well-tolerated in healthy volunteers, demonstrating a favorable pharmacokinetic profile and robust target engagement.

2.

ventustx.com

ventustx.com/ventus-therapeutics-to-present-phase-1-results-for-vent-03-a-first-in-class-cgas-inhibitor-at-lupus-2025/

Ventus Therapeutics to Present Phase 1 Results for VENT- ...

Data from the Phase 1 study, initially announced in October 2024, showed that VENT-03 was safe and well tolerated at all tested dose levels with ...

3.

lupus.org

lupus.org/news/update-ventus-therapeutics-announces-positive-phase-1-trial-results-for-investigational-drug

Ventus Therapeutics Announces Positive Phase 1 Trial ...

The results showed that VENT-03 was safe and well-tolerated at all tested dose levels with all treatment-related adverse events being mild and ...

4.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S0003496725029607

Annals of the Rheumatic Diseases

VENT-03 was safe and well-tolerated in healthy volunteers, demonstrating a favorable pharmacokinetic profile and robust target engagement. The efficacy of VENT- ...

5.

acrabstracts.org

acrabstracts.org/abstract/discovery-of-vent-03-a-novel-clinical-cgas-inhibitor-for-the-treatment-of-sle-and-other-autoimmune-diseases/

Discovery of VENT-03: A Novel Clinical cGAS Inhibitor for ...

Tool molecules of the VENT-03 chemotype were efficacious in reducing auto-inflammation and rescuing clinically relevant endpoints in the Trex1-/- mouse model. ...

6.

lupus.org

lupus.org/news/drug-update-first-person-dosed-with-investigational-drug-vent03-for-lupus

First Person Dosed with Investigational Drug, VENT-03, for ...

The trial underway will assess the drug's safety and how the medication affects the body.

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05835310

An Efficacy and Safety Study of Intravenous Anifrolumab to ...

This study aims to characterize the pharmacokinetics, pharmacodynamics, efficacy, and safety of Anifrolumab solution for infusion compared with placebo ...