Eli Lilly And Co (NYSE:LLY) doubling down on RNA research as it pens another pact with a quiet RNA editing player focused on neuroscience, ProQR Therapeutics N.V. (NASDAQ:PRQR)
In May, Lilly teamed up with MiNA Therapeutics to tap its small activating RNA (saRNA) technology platform for up to five targets.
ProQR will receive an upfront payment of $20 million and an equity investment of $30 million, with up to $1.25 billion as milestone payments for five targets using its RNA platform.
ProQR’s oligonucleotide platform, dubbed Axiomer, recruits "endogenous" enzymes in the cell, called ADARs, to attach to target RNA and induce adenosine to inosine base switch.
This approach can potentially reverse more than 20,000 G to A mutations known to cause human disease.
Moreover, Lilly announced Thursday the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for Jardiance ® (empagliflozin) as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF).
The decision is based on results from the landmark EMPEROR-Preserved phase III trial, in which Jardiance demonstrated a 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction compared with placebo.
The benefit was independent of ejection fraction or diabetes status. Results from EMPEROR-Preserved were presented at the European Society of Cardiology Congress 2021 on August 27 and published in The New England Journal of Medicine .
Meanwhile, PRQR stock is up $1.16, or 17% at $7.97 soon after Thursday’s opening bell, while LLY stock sank $3.56, or 1.4%, to $251.28.
