Bristol-Myers Squibb (BMS) will pay Tekmira Pharmaceuticals $3 million as part of a target-validation agreement. BMS will use stable nucleic acid-lipid particles (SNALP) supplied by Tekmira to validate the function of certain cellular targets.
Under the agreement, BMS will leverage SNALP siRNAs to silence target genes of interest. SNALP technology encapsulates siRNAs with high efficiency in uniform lipid nanoparticles to deliver RNAi therapeutics to disease sites, Tekmira explains. The firm is responsible for providing a predetermined number of the SNALP batches over the four-year term of the deal.
BMS will conduct the preclinical work to validate the function of certain genes. The data will be shared with Tekmira, which will then develop RNAi-based drugs against the targets of interest. BMS retains first right to negotiate a license for certain RNAi products developed by Tekmira that evolve from the gene targets it validates.
Commenting on the structure of the arrangement, Mark J. Murray, Tekmira’s president and CEO, says, “This will enable us to leverage the target-validation capabilities of a global pharmaceutical company to identify our own RNAi product development opportunities.”
Tekmira’s lead candidate is ApoB SNALP, which is being developed as a treatment for high LDL. It is scheduled to enter a Phase I/II trial later this year. Next in line is anticancer agent PLK1 SNALP, and the company says that it is on track to file an IND application and initiate a Phase I trial in the second half of this year.
SNALP technology relies on the enhanced permeability and retention effect, which occurs because these nucleic acid-containing particles have a long circulation time in the blood. This results in increased accumulation at sites of vascular leaks, such as those found at sites of tumor cell growth, infection, or inflammation. Once at the target site, cells take up the SNALP through endocytosis and the nucleic acid payload is delivered inside the cell.