Regulus surges after AstraZeneca snags rights to preclinical NASH drug

AstraZeneca's Marcus Schindler

Shares of Regulus spiked 10% ($RGLS) Tuesday afternoon after its Big Pharma partner AstraZeneca stepped up to grab rights to a preclinical drug candidate for NASH with plans to steer it into clinical studies.

It's not a big money deal, at least not initially. AstraZeneca ($AZN) is gaining rights to RG-125 (AZD4076)--one of three therapeutic programs outlined in a three-year-old discovery alliance between the two companies--for $2.5 million. It's also the latest in a string of clinical programs for fatty liver disease, a condition that afflicts a huge and growing patient population. For AstraZeneca, which has been committing big resources to diabetes drugs, the deal today offers a chance to beef up a core pipeline.

In their preclinical work, investigators say they were able to nail down signs that targeting miR-103/107 with one of their microRNA therapies triggered a drop in fasting glucose and fasting insulin levels. "Additionally, anti-miRs targeting miR-103/107 function as unique insulin sensitizers as determined by hyperinsulinemic-euglycemic clamp, which is a robust method for assessing insulin sensitivity," according to the companies. "Further, inhibition of miR-103/107 with Regulus' anti-miRs decreases liver triglycerides and steatosis, providing an opportunity to investigate RG-125 (AZD4076) to treat NASH in patients with type 2 diabetes/pre-diabetes."

A Phase I study should get started later this year.

Dr. Markus Stoffel, professor of the Institute of Molecular Health Sciences at ETH Zurich and member of Regulus' SAB, is credited with making the initial observations about miR-103/107's potential as a drug target.

Regulus snagged $28 million when it struck the deal with AstraZeneca. Its added cash in this first tie-up is also tied to up to $495 million in potential back-ended milestones.

"This is a tremendous achievement for our collaboration and an exciting step for AstraZeneca to be progressing AZD4076 (RG-125), an anti-microRNA molecule, into preclinical development," says Marcus Schindler, head of cardiovascular and metabolic diseases at AstraZeneca. 

- here's the release

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