Almirall licenses rights to ZKN-013, potential treatment for RDEB, JEB

Phase 1 trial cleared last year to test safety of ZKN-013 in healthy volunteers

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Almirall has licensed rights to develop and commercialize ZKN-013, an experimental oral treatment for recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB).

As part of the deal, Almirall is making an up-front payment of $3 million to ZKN-013’s original developer Eloxx Pharmaceuticals. Eloxx will also be eligible for additional payments of up to $470 million, plus royalties from sales, if the therapy is ultimately approved and brought to market.

“We are very excited about this agreement with Almirall to develop and distribute ZKN-013 … we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” Sumit Aggarwal, president and CEO of Eloxx, said in a joint press release from the companies.

RDEB is caused by mutations in the gene that provides instructions to make a part of collagen type VII, a protein that’s essential for maintaining the normal structure of the skin. JEB often results from mutations in the LAMA3LAMB3, and LAMC2 genes that disrupt production of laminin 332, a protein that acts to strengthen the skin. In both disease types, patients are prone to blisters, fragile skin, and other symptoms.

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ZKN-013 developed for RDEB or JEB caused by nonsense mutations

ZKN-013 is specifically being developed for people with RDEB or JEB caused by a nonsense mutation in COL7A1 and LAMB3, respectively. This is a specific type of mutation that leads to an erroneous “stop” signal in the middle of the gene, sort of like having a period right in the middle of a sentence. As a result, when the gene is “read,” the full protein cannot be produced.

The therapy is designed to allow the cell’s protein-making machinery, called ribosomes, to skip over the abnormal stop signal and produce a full-length version of the proteins. In addition to RDEB/JEB, Almirall is also hoping to develop the therapy for familial adenomatous polyposis, a condition marked by growths in the intestines.

The U.S. Food and Drug Administration last year gave the go-ahead to start a Phase 1 trial testing the safety of ZKN-013 in healthy volunteers.

“We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations,” said Karl Ziegelbauer, executive vice president of research and development and chief scientific officer at Almirall.