AMSTERDAM, Netherlands — October 1, 2026
Amplio Pharma reported positive early clinical results from its Phase 1b study of NovoBioJect, an investigational combination of low-dose methotrexate (MTX) and novobiocin being developed to improve first-line treatment of rheumatoid arthritis (RA). A single dose of NovoBioJect produced a 38% increase in intracellular long-chain methotrexate polyglutamates compared with methotrexate alone, providing clinical proof of principle for the program’s mechanism. The increase was statistically significant, with a reported P value of 0.009. The company said the findings support advancement of NovoBioJect into a planned Phase 2a proof-of-concept study in newly diagnosed RA patients. Amplio is also initiating an approximately €10 million Series A financing to fund the next stage of clinical development.
NovoBioJect Demonstrates Intracellular Methotrexate Activity
NovoBioJect is designed to increase the amount of active methotrexate retained inside immune cells by combining MTX with low-dose novobiocin, an inhibitor of the ABCG2 efflux transporter. Methotrexate is widely used as a first-line treatment for RA, but a substantial proportion of patients do not achieve remission with MTX alone. Amplio’s approach is based on the hypothesis that insufficient intracellular retention and activation of methotrexate may contribute to treatment failure in some patients. In the Phase 1b study, NovoBioJect increased combined intracellular MTX-PG4 and MTX-PG5 levels by 38% compared with MTX alone. These long-chain methotrexate polyglutamates were used as pharmacological biomarkers of intracellular methotrexate activity. The company said the result represents the first clinical evidence supporting the mechanism underlying NovoBioJect and provides a biomarker framework for continued development.
Amplio Advances NovoBioJect Toward Phase 2a
The Phase 1b study enrolled 12 patients with rheumatoid arthritis receiving stable weekly methotrexate treatment. The open-label, cross-over study evaluated single ascending doses of novobiocin administered subcutaneously alongside patients’ regular methotrexate dose. Amplio reported a favorable single-dose safety profile, with mild, dose-dependent injection-site reactions representing the reported treatment-related findings. The study was designed to evaluate pharmacokinetics, safety and biomarker response rather than establish clinical efficacy. Amplio now plans a Phase 2a proof-of-concept study in the Netherlands, expected to enroll approximately 100 newly diagnosed RA patients and compare NovoBioJect with methotrexate alone over 12 weeks. The planned study will assess safety as the primary endpoint, with clinical response, speed of disease control and MTX-polyglutamate biomarkers among the secondary measures. Subject to financing and regulatory approval, the company expects to initiate the study in 2027.
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Amplio Initiates €10 Million Series A Financing
Amplio Pharma is initiating an approximately €10 million Series A financing to advance NovoBioJect through the planned Phase 2a study and clinical readout. The financing follows the company’s earlier €1.4 million seed round, which was supported by Libertatis Ergo Holding, Amsterdam UMC Ventures Holding, ROM InWest MKB-fonds and Innovatiefonds Noord-Holland. Amplio is developing NovoBioJect as a potential once-weekly subcutaneous treatment intended to improve the effectiveness of methotrexate while retaining the established first-line role of MTX in RA treatment. The company’s immediate development objective is to determine whether the demonstrated increase in intracellular methotrexate activity translates into improved clinical outcomes. The positive Phase 1b biomarker findings, favorable single-dose safety profile and planned Phase 2a program represent the next clinical milestones for Amplio as it advances NovoBioJect toward proof of concept in rheumatoid arthritis.
Source:Amplio Pharma, press release
