Company will use technology to develop treatments for lysosomal storage diseases.

CytRx has sold worldwide rights to its molecular chaperone assets to Danish firm Orphazyme, in a deal that includes an up-front fee and potentially another $120 million in milestones, plus future sales royalties. CytRx says the sale is the last stage in its move to divest its noncore assets and focus on its clinical-stage oncology pipeline. “This could be a game-changing transaction for CytRx with an ultimate potential value that exceeds our current market capitalization,” remarks Steven A. Kriegsman, president and CEO. “This transaction provides us with additional resources to focus on our highly promising oncology drug candidates.”

The assets acquired by Orphazyme include three orally administered molecular chaperone amplifiers—arimoclomol, iroxanadine, and bimoclomol—which together have been evaluated in 13 Phase I and six Phase II clinical trials. CytRx molecular chaperone amplifiers are believed to act by co-induction of the stress response, such that rather than activating the stress response directly, they instead amplify the production of molecular chaperone proteins that are already activated by disease-induced cellular stress.

Orphazyme was established in 2009 to develop molecular chaperone therapies for treating rare genetic lysosomal diseases. The firm’s scientific strategy hinges on the lysosome-protective and stabilizing effect of heat shock proteins (HSPs). Its existing lead program, Orph-001, is a recombinant version of human HSP70, which is currently being evaluated in vivo in animal models.

”We believe that CytRx’ molecular chaperone technology can be used to revert lysosomal pathology through the induction of heat shock proteins, such as HSP70, and thus can be applied to develop new treatments for these diseases,” comments Orphazyme CEO Anders Hinsby, Ph.D.

CytRx, meanwhile, will continue to focus on its core oncology pipeline, which includes three clincial-stage candidates: bafetinib, tamibarotene, and INNO-206. Bafetinib is an orally available, dual Bcr-Abl and Lyn-kinase inhibitor, originally developed by the Nippon Shinyaku, to overcome the limitations of Gleevec and second-line tyrosine kinase inhibitors in resistant chronic myelogenous leukemia. The drug is in Phase II trials against B-cell chronic lymphocytic leukemia and advanced prostate cancer, and it is in Phase I evaluation against brain cancer.

Tamibarotene is an orally available, synthetic retinoid compound designed to avoid toxic side effects by binding to its molecular target more selectively than all transretinoic acid (ATRA), the current first-line treatment for APL, CytRx claims. It has completed Phase I studies in non-small cell lung cancer, and is undergoing a pivotal Phase II study in acute promyelocytic leukemia (APL). Phase I combination studies for the APL indication are also in progress. CytRx retains an option to expand its existing licenses for the use of tamibarotene for other oncology indications, including multiple myeloma, myelodysplastic syndrome, and certain solid tumors in the U.S., and multiple myeloma, myelodysplastic syndromes, and solid tumors other than hepatocellular carcinoma in Europe.

INNO-206 is a prodrug derivative of the commonly prescribed chemotherapeutic agent doxorubicin. CytRx holds the exclusive worldwide rights to the drug, which is designed to reduce adverse events by controlling drug release and preferentially targeting tumors. INNO-206 has completed Phase I evaluation against pancreatic cancer and soft tissue sarcomas.

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