Selected drug repurposing patent applications published in Q1/2016

This is a selection of PCT patent applications published during the first three months of 2016 that claim new uses for known drugs, and are reviewed in ASSAY and Drug Development Technologies‘ 2016 summer special edition on drug repurposing. This follows up on our blogpost of June 23 where we summarized the patent applications of Q4/2015, also to be published in this issue of ASSAY. – Do not miss it!

WO/2016/001682 (Summit Therapeutics, UK): Upregulation of utrophin, an autosomal paralogue of dystrophin, is being proposed as a therapy for Duchenne’s muscular dystrophy. An isoform is expressed in the liver and may play a role in hepatic signaling and/or membrane integrity. Connect the dots, and there is a potential new use for Summit’s Duchenne drug candidate ezutromid in liver disease.

WO/2016/007540 (The J. David Gladstone Institutes, USA): There is no approved drug for Dengue fever. LRRK2, a leucine-rich repeat kinase, interacts with the Dengue virus capsid, and is apparently required for its life cycle. This offers perspectives for LRRK2-IN-1 and GSK-2578215A, two LRRK2 inhibitors investigated for Parkinson’s disease.

WO/2016/029017 (The Johns Hopkins University, USA): The COMT inhibitor entacapone, used in combination products to increase oral bioavailability of L-Dopa in Parkinson’s patients, might serve as an gastrointestinal motility drug.

WO/2016/034617 (INSERM and French universities): The anticonvulsant, valproate could be useful in iron overload disorders: it increases hepatic expression of hepcidin, which limits iron release into the bloodstream.

WO/2016/036676 (Individuals, Taiwan): That the old phenothiazine antipsychotic, thioridazine can reverse resistance of cancer cells to chemotherapy is well known. The inventors present a new twist to this story, claiming that thioridazine reduces non-small cell lung cancer stem cells with KRAS mutations by inhibiting HMG-CoA reductase.

WO/2016/040860 (Tobira Therapeutics, USA): Takeda, the original developer of cenicriviroc (a CCR5 and CCR2 antagonist), had an HIV entry inhibitor in mind. Its licensee, Tobira is now developing the compound for liver fibrosis, and has the CENTAUR Phase IIb study fully enrolled.

WO/2016/046374 (Individual): Methylphenidate seems almost irreversibly linked to attention deficit / hyperactivity disorder. As with related stimulants, it can cause loss of appetite. It is all the more interesting to read that methylphenidate might help with anorexia nervosa.