Biodexa Pharma Announces Further Progress In Both Its Lead Programs; eRapa For Familial Adenomatous Polyposis And MTX240 Molecular Glue For Gastrointestinal Stromal Tumors

Biodexa provides R&D Update

Phase 3 Serenta trial in FAP is now two-thirds recruited

Preparations advanced for Phase 1 trial of MTX240 in GIST

Biodexa Pharmaceuticals PLC (NASDAQ:BDRX) ("Biodexa" or "the Company"), a clinical stage biopharmaceutical company developing innovative products focused on the treatment or prevention of gastrointestinal cancers is pleased to announce further progress in both its lead programs; eRapa for familial adenomatous polyposis ("FAP") and MTX240 molecular glue for gastrointestinal stromal tumors ("GIST").

eRapa for FAP

The Company has enrolled 112 of an expected 168 participants in its registrational Phase 3 trial of eRapa, the Company’s reformulation of rapamycin, in FAP (NCT06950385). The trial, branded "Serenta" is recruiting at 29 clinical sites across the US and five countries in Europe with further sites in North America and Continental Europe expected to be initiated shortly.

The Serenta protocol provides for a pre-specified futility analysis after 25 progression free survival ("PFS") events and database lock after 75 PFS events. The Serenta protocol includes a composite endpoint which defines the nature of the PFS events. Based on the current rate of PFS event accrual, the futility analysis could take place early in 2027 with database lock potentially occurring around the end of 2027, both significantly sooner than originally anticipated.

MTX240 molecular glue for GIST

Non-resectable and/or metastatic GIST is generally treated with tyrosine kinase inhibitors ("TKIs"). While TKIs have improved outcomes, resistance almost always develops through either secondary (or tertiary) KIT or PDGFR mutations or activation of alternative signalling pathways resulting in limited therapeutic options for patients once they have cycled through the available TKIs. In multiple patient-derived xenograft models, MTX240 has been shown to be effective in TKI-resistant GIST tumors1.

The Company is planning a Phase 1a dose escalation study followed immediately by a Phase 1b dose optimization study with a view to establishing a maximum tolerated dose and, based on efficacy signals, an optimized dose for MTX240. Both Phase 1a and 1b will recruit patients who have demonstrated TKI-resistance. GMP drug substance has been manufactured by our CDMO partner and they are moving ahead with GMP finished product. A preferred CRO has been identified and the Phase 1a is expected to begin recruiting in South Korea mid-2027. A pre-IND meeting with the FDA is scheduled for December 10, 2026.