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Moleculin's Drug Candidate Lowers Size of a Rare Brain Tumor

Updated: 2 days ago





Moleculin Biotech, Inc., a clinical-stage pharmaceutical firm with a broad portfolio of drug candidates aimed at major unmet needs in the treatment of viruses and tumors, announced preliminary first cohort data from the Emory University physician-sponsored clinical trial being conducted at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta by Dr. Tobey MacDonald, Professor of Pediatrics and Director of the Pediatric Neuro-Oncology Program. He is studying the use of WP1066 (AflacST1901), a proprietary Moleculin drug candidate, as a likely treatment for childhood brain tumors. The first three patients in the trial were given treatment at a dose level of 4mg/kg with no adverse effects related to WP1066, and the study will now proceed to the next higher dose of 6mg/kg. One of the patients with a diffuse intrinsic pontine response (DIPG) showed a distinct response to the treatment with both clinical improvement and radiologic lowering of tumor size.


Dr. MacDonald stated, “We are very pleased that this trial has successfully completed the first cohort without any safety issues and will now progress to the second cohort at an escalated dose level. We must, of course, be very careful not to draw any conclusions from such preliminary data, but to have an objective response in a DIPG patient is frankly, unexpected.”

On his part, Mr. Walter Klemp, Chairman, and CEO of Moleculin, said, "When you look at the clinical trial history of DIPG, despite approximately 200 clinical trials, no drug has shown significant activity in this disease, so we find this initial activity particularly encouraging.


WP1066 is an immune-stimulating p-STAT3 inhibitor and has been shown to stimulate immune responses that successfully modulate oncogenic transcriptional activity in tumor cells and repress their ability to drive tumor growth. Coupled with the activity we have recently seen with WP1220, a close analog to WP1066, in its proof of concept clinical trial for the topical treatment of cutaneous t-cell lymphoma, we are more committed than ever to determine the full potential of this new class of p-STAT3 inhibitors. We now have six drug candidates, with three of them showing human activity, so we need to be careful not to confuse this p-STAT3 inhibitor pipeline with the recent announcement regarding our antimetabolites and their potential to treat viruses. We have placed a high priority on reducing risk for our investors by creating what we call 'multiple shots on goal,' and the events of this week are showing just how effective that strategy has been."


Mr. Klemp concluded, “Consistent with our history of providing clinical trial updates on a cohort-by-cohort basis, we look forward to updating investors on the continued progress of this trial as additional cohorts are completed.”

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