Amplio reports early human data for novel methotrexate approach in rheumatoid arthritis
pharmafile | October 7, 2026 | News story | |Â Â Rheumatology, clinical testing, immunology, rheumatoid arthritisÂ
Amplio Pharma has reported phase 1b results for NovoBioJect, an investigational combination of low-dose methotrexate (MTX) and novobiocin being developed as a potential first-line treatment for rheumatoid arthritis (RA).
In a 12-patient study, a single dose of NovoBioJect produced a 38% increase in intracellular long-chain MTX polyglutamates (MTX-PG4 and MTX-PG5) compared with MTX alone. The increase was statistically significant (P=0.009).
NovoBioJect is designed to increase the retention of methotrexate inside immune cells by blocking the ABCG2 efflux transporter with low-dose novobiocin. MTX polyglutamates are being used as pharmacological biomarkers of methotrexate activity.
The open-label study enrolled patients with RA receiving stable weekly doses of methotrexate. Participants received a single subcutaneous dose of novobiocin alongside their usual methotrexate dose, at doses of 80mg, 150mg or 200mg.
The single-dose safety assessment found mild, dose-dependent injection-site reactions, with no other treatment-related adverse events reported.
However, the study was designed to assess pharmacokinetics, safety and biomarker response and was not designed or powered to establish clinical efficacy. Whether the increase in intracellular methotrexate activity translates into improved disease control will be assessed in further trials.
Amplio plans a phase 2a study in around 100 newly diagnosed RA patients, comparing NovoBioJect with methotrexate alone over 12 weeks. The proposed study will assess clinical response, speed of disease control and MTX polyglutamate biomarkers, with safety as the primary endpoint. Subject to financing and regulatory approval, the company expects to begin the study in 2027. Amplio is seeking approximately €10m in a Series A financing round to fund the phase 2a programme.
Marguérite Mensonides-Harsema, CEO and co-founder of Amplio Pharma, said the results provided evidence that blocking ABCG2 increased active methotrexate inside cells, while noting that the next question was whether this would translate into faster remission.
The company has submitted the full phase 1b dataset for presentation at ACR Convergence 2026.
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