Phase I study of the heparanase inhibitor roneparstat: an innovative approach for ultiple myeloma therapy

3] In preclinical models of MM, heparanase was shown to be a master regulator of aggressive tumor behavior and bortezomib and melphalan were each found to enhance heparanase expression and secretion. It is a very potent and pure competitive heparanase inhibitor. Roneparstat showed a significant anti-myeloma effect in murine models, either alone or in combination with dexamethasone, bortezomib or melphalan. Based on this preclinical evidence an open-label, multicenter, phase I, first-in-human study was designed to assess the safety and tolerability profile of roneparstat in patients with relapsed/refractory MM (EudraCT number 2012-001127-12 and clinicaltrials.gov identifier: NCT01764880).