On December 18, 2025 PharmaMar (MSE:PHM) reported that it has received an investigational new drug (IND) approval from the U.S Food and Drug Administration (FDA), to start a Phase 1/2 multicenter clinical trial for the combination of PM54 with immunotherapy for the treatment of advanced stage solid tumors.

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This refers a Phase 1 study for the evaluation of the safety, followed by a Phase 2 to evaluate the safety and efficacy profile in patients affected by tumors with significant unmet medical needs such as advanced-stage melanoma, endometrial cancer, extrapulmonary neuroendocrine cancer and mesothelioma.

The approval of this IND allows, after the positive evaluation of the dossier with the preclinical data, manufacturing information and the protocol of the proposed clinical trial, the beginning of the activities related to the study in the United States.

PM54 is a novel inhibitor of the oncogenic transcription of the ecteinascidin family, currently being evaluated as a monotherapy in Phase 1/1b studies, in patients with advanced solid tumors to assess safety, tolerability, pharmacokinetics and preliminary antitumor activity.

Preliminary data from ongoing studies, together with evidence from preclinical studies and the mechanism of action, support the advancement of the clinical development plan for PM54 and the initiation of the study in combination with immunotherapy.

(Press release, PharmaMar, DEC 18, 2025, View Source [SID1234661531])