AstraZeneca taps MiNA to treat metabolic diseases by activating genes

MiNA Therapeutics CEO Robert Habib (MiNA Therapeutics)

AstraZeneca has teamed up with MiNA Therapeutics to test small activating RNA (saRNA) molecules in metabolic diseases. The agreement gives AstraZeneca the option to negotiate a license for saRNA molecules after getting a look at preclinical data on the candidates. 

London-based MiNA is built upon a platform designed to uncover and advance small oligonucleotide drugs that activate genes. The candidates are similar to RNA drugs developed by companies such as Alnylam. But rather than silence genes to suppress protein production, MiNA’s drugs activate genes to upregulate intracellular or secreted proteins. 

MiNA CEO Robert Habib sees saRNAs as a way to modulate targets once thought to be undruggable. 

“By modulating targets at the gene level, you can drug pathways that are conventionally undruggable. The vast majority of proteins in disease are actually undruggable by the conventional medicines, small molecules and biologics. RNA technology is a way at getting at those proteins at a gene level. We’re basically restoring levels of beneficial proteins that are otherwise downregulated or repressed in disease,” Habib said.

Early-phase data on MiNA’s lead internal program MTL-CEBPA in liver cancer have begun to validate that belief. And AstraZeneca has seen enough potential in the platform to join forces with MiNA to go after a gene target relevant to metabolic diseases. 

The partners will run in vitro and in vivo studies to see whether saRNA molecules can treat metabolic diseases via pathways that are inaccessible to other modalities. After seeing data from the preclinical tests, AstraZeneca will decide whether to try to license the program.

AstraZeneca began doing exploratory work with MiNA after recognizing that saRNA technology may enable it to get at new, high value but seemingly undruggable metabolic disease targets. Habib sees the collaboration as a continuation of the broader expansion of RNA into metabolic diseases. 

“Metabolic disease has been an area where in the last few years other RNA modalities have seen huge success. The latest and clearest example is inclisiran. That shows exactly the power of an RNA therapy offering superior pharmacology to patients in metabolic disease. At MiNA, we feel metabolic disease is absolutely an area that RNA therapy can work really, really effectively,” Habib said.

AstraZeneca is the latest in a string of companies to be attracted to the potential of saRNA drugs. In 2017, Sosei invested $35 million in MiNA and picked up an option to buy the biotech outright, only to let the option lapse the next year. Between those two events, Boehringer Ingelheim put together a deal worth up to €307 million ($356 million) to work with MiNA on fibrotic liver disease targets.