GlaxoSmithKline has formed a collaboration with Wave Life Sciences, a Cambridge, Massachusetts-based clinical-stage bio/pharmaceutical company, to advance oligonucleotide therapeutics, in a deal worth up to $3.7 billion ($170 million upfront and $3.5 billion in milestone payments).  

Oligonucleotides are short strands of DNA or RNA that can reduce, restore, or modulate RNA through several different mechanisms and have the potential to treat a wide range of diseases. 

The discovery and development collaboration has an initial four-year research term. The collaboration will use Wave Life Sciences’ proprietary Prism oligonucleotide platform that uses three RNA-targeting modalities (editing, splicing, and silencing, including siRNA and antisense). The modalities incorporate chemistry to optimize the pharmacological properties of therapeutic oligonucleotides. 

The collaboration includes two main components. The first is a discovery collaboration which enables GSK to advance up to eight programs and Wave to advance up to three programs, using Wave’s Prism platform and GSK’s expertise in genetics and genomics. In addition to these programs, GSK receives an exclusive global license for Wave’s preclinical program for WVE-006 for treating antitrypsin deficiency (AATD), a genetic disease that affects both the lungs and liver. WVE-006 is an RNA-editing therapeutic that is designed to address both liver and lung manifestations of the disease. 

Under the agreement, Wave will receive an upfront payment of $170 million, which includes a cash payment of $120 million and a $50-million equity investment. For the WVE-006 program, Wave is eligible to receive up to $225 million in development and launch milestone payments and up to $300 million in sales-related milestone payments as well as tiered sales royalties. Development and commercialization responsibilities will transfer to GSK after Wave completes the first-in-patient study. 

For each of GSK’s eight collaboration programs, Wave will be eligible to receive up to $130-$175 million in development and launch milestones and $200 million in sales-related milestones, along with tiered sales royalties.  

Wave will lead all preclinical research for GSK and Wave programs up to investigational new drug (IND)-enabling studies. GSK collaboration programs will transfer to GSK for IND-enabling studies, clinical development, and commercialization. The collaboration includes an option to extend the research term for up to three additional years to expand the number of programs available to both parties. 

Source: GlaxoSmithKline