5:35 PM
 | 
Oct 20, 2017
 |  BioCentury  |  Product Development

Naked ambition

How miRagen designed its microRNA molecule for systemic delivery in cancer

Proof-of-concept data from miRagen Therapeutics Inc. show systemic delivery of its lead candidate can shrink lesions in cutaneous T cell lymphoma, and that its oligonucleotide platform can achieve broad distribution and a long half-life without the addition of a conjugate or vehicle.

On Oct. 13, miRagen reported interim data from a Phase I trial of MRG-106 to treat mycosis fungoides-type cutaneous T cell lymphoma (CTCL). All but one of the 23 patients treated showed improvement in total skin disease measured as the change from baseline in modified Severity Weighted Assessment Tool (mSWAT) score. mSWAT assesses skin disease over the entire body.

At the time of readout, five patients had maintained a response for four months or longer.

Patients received 300, 600 or 900 mg doses of subcutaneous or IV MRG-106, a synthetic oligonucleotide that inhibits microRNA-155 (miR-155). The trial was not powered to demonstrate statistical significance.

“With the combination of the phosphorothioates and the LNA in the molecule, what you’ll find is you get more serum protein binding.”

William Marshall, miRagen

Adverse events were limited to injection site reactions, with the possible exception of one patient who had a grade 3 worsening of...

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