San Diego-based Celladon (NASDAQ: CLDN) said it has granted France’s Servier an option to license a new class of small-molecule drugs that could someday be used to treat type 2 diabetes and other metabolic diseases.
The clinical-stage biotech, which raised gross proceeds of roughly $44 million in its IPO last month, specializes in treating calcium dysregulation, which is implicated in heart failure and other complex medical conditions and diseases.
Celladon is in a mid-stage trial with its lead program, a gene therapy treatment designed to restore levels of an enzyme known to play a key role in progressive heart disease. The deal with Servier would lead to a strategic collaboration for another drug development program at Celladon that is focused on compounds that offer a new way to regulate a related enzyme called SERCA2b. These enzymes help to regulate protein synthesis by controlling calcium movement within the cell.
Without enough SERCA2b enzymes, the calcium signaling pathway that controls protein synthesis gets disrupted. “When calcium stores get depleted, it stresses the normal processing and synthesizing of new proteins,” Celladon CEO Krisztina Zsebo told me by phone yesterday.
The company says a proliferation of recent biomedical studies have implicated this calcium dysregulation in a variety of diseases and disorders. By identifying small molecules that can modulate SERCA2b enzymes, Celladon says it could eventually correct the underlying calcium dysregulation in the endoplasmic reticulum, the organelle in all human cells where proteins are made.
Celladon has focused initially on how this dysfunctional process can spill into type 2 diabetes. Because this same calcium signaling process has been found to occur in different types of cells, Zsebo said Celladon’s R&D is expected to identify multiple drug targets for other calcium-dependant afflictions, including certain cancers and neurodegenerative disorders.
If Servier exercises its option, the French pharma would get global rights outside the United States for a certain period to SERCA drug candidates for treating type 2 diabetes and other metabolic diseases. In exchange, Servier would make a variety of payments to Celladon, including royalties on future sales of the new drug.
A strategic collaboration “would essentially pay for drug development ex-U.S., and enable us to pay for our drug development within the U.S.,” Zsebo said. Whether Servier decides to exercise its option will depend on the outcome of a series of pre-defined studies to be conducted by both parties.
By using its proprietary assays and screening technology to identify compounds that regulate SERCA enzymes, Celladon also could amass a catalog of small molecules with potential applications in treating other diseases. The company says it would retain all U.S. rights to any compounds and lead drug candidates developed through its collaboration and license agreement with Servier.