On February 25, 2019 ArQule reported the publication of clinical pharmacodynamic, safety and efficacy data in patients with Proteus syndrome (Press release, ArQule, FEB 25, 2019, View Source [SID1234533719]). These data, together with data already presented at ASH (Free ASH Whitepaper)G last year, support miransertib’s further development as a potential first systemic treatment for patients suffering from overgrowth diseases, such as Proteus syndrome. The study, published in the American Journal of Human Genetics, and led by the National Institutes of Health (NIH), demonstrated good target engagement, tolerability and reductions in lesion size and pain, especially in children.

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Highlights from the study include:

Generally well-tolerated safety profile
Reductions in pAKT (activated AKT) in most patients
Reductions in Cerebriform Connective Tissue Nevus (CCTN) lesions in size (measured with standardized photography) but also in firmness, depth of sulci and discomfort (by patient report)
Reduction in pain intensity in all (3 of 3) children in the study

"These data are highly encouraging and support further investigation into the potential use of miransertib for this devastating condition," said Dr. Brian Schwartz, Chief Medical Officer at ArQule. "The reduction in CCTN lesions were particularly striking since these lesions are, in general, relentlessly progressive, and cause severe morbidity, ulcerations and intractable pain. We’d like to thank our academic collaborators and the patients and their families for their support and tremendous dedication to this cause."

The study is available online at: View Source