Atisama's inhaled RB042 heads toward higher-dose Phase 1 safety readouts
Three of four single-dose cohorts cleared safety and tolerability, positioning multi-dose data due later in 2026 to test whether the inhaled RAGE-targeted oligonucleotide holds up at higher exposure.
Executive Summary
- Atisama Therapeutics is advancing its lead inhaled candidate into higher-dose testing, with the next tolerability readouts due later this year.
- Early single-dose cohorts cleared safety and tolerability, the threshold that let the program move into multi-dose testing.
- The primary measure is adverse-event frequency, so the coming data test whether tolerability holds as dosing intensifies, not whether the drug works.
- The program's target has no active peer in the current competitive field, leaving this readout without a direct precedent to benchmark against.
The update
Atisama Therapeutics, an Australian biotechnology company formerly known as Rage Bio, said its Phase 1 study of RB042 (NCT07285122; RB042_1001) has moved from single ascending dose (SAD) testing into multiple ascending dose (MAD) cohorts. Three of four SAD cohorts are complete and the first MAD cohort has started, with the company saying higher-dose cohorts are expected to read out in the second half of 2026. Ed Tucker, the company's Chief Medical and Development Officer, said RB042 clinical progress is tracking to plan and that safety and tolerability across the SAD cohorts supported advancement into MAD dosing. AtisamaAtisama Therapeutics Advances RB042 into Multiple Ascending Dose cohorts of Phase 1 Trial and Appoints Bernard Coulie as Chair of the BoardMay 15, 2026
Probability of SuccessBased on the AppliedXL Probability of Success model. For more information about the methodology, read the research here.
What the trial tests
RB042 is an inhaled splice-switching oligonucleotide targeting the Receptor for Advanced Glycation End-products, dosed by inhalation in healthy adult volunteers and healthy adult smokers. The registered primary endpoint is the number and frequency of treatment-emergent adverse events, with secondary endpoints covering pharmacokinetic measures including area under the concentration-time curve, maximum concentration, and half-life. The trial is recruiting toward a target enrollment of 90 participants at a single site in Australia, with a registered primary completion date of February 1, 2027. NCT07285122Inhaled RB042 in Healthy Adult Volunteers and Healthy Adult SmokersNCT07285122
Reading the setup
Because the primary endpoint is adverse-event incidence rather than a clinical efficacy measure, the coming multi-dose cohort data will show whether the tolerability observed at single ascending doses persists as exposure rises, the standard bar for a Phase 1 inhaled-oligonucleotide program before any target-engagement or efficacy work follows. The trial has not posted results to its registry record, and no primary endpoint outcome has been reported. The study's status has moved cleanly from Not yet recruiting to Recruiting since a February 2026 registry update, with no enrollment target changes and no protocol amendment to the primary endpoint recorded since the study was first posted. NCT07285122Inhaled RB042 in Healthy Adult Volunteers and Healthy Adult SmokersNCT07285122
The competitive field
No other industry-sponsored trial in the current competitive landscape targets the Receptor for Advanced Glycation End-products, leaving RB042 without a direct mechanistic comparator to benchmark its tolerability data against. The broader field of Phase 1 studies in healthy volunteers and smokers includes AstraZeneca's AZD4916 and other single- and multiple-ascending-dose programs, but these test different targets and mechanisms, so they inform only the general operating norms for this study type, not RB042's specific biology.
This analysis was produced using AI-assisted reporting systems, AppliedXL data, and official public records. These systems undergo editorial review, quality checks, and regular audits by human experts. Errors may still occur, as with any automated system. Always consult the linked primary sources. Read our AI Editorial Policy.