GSK pays $170M upfront to continue to ride oligonucleotide wave for years to come

GSK has added another oligonucleotide to its pipeline, with potentially plenty more to follow courtesy of a $170 million upfront collaboration with Wave Life Sciences.

As part of the deal, GSK will get its hands on the global license to WVE-006, Wave’s preclinical RNA editing therapy targeting alpha-1 antitrypsin deficiency (AATD), an inherited disorder that can cause lung and liver disease. The candidate uses Wave’s proprietary RNA-editing technology, dubbed AIMer.

In the longer term, the four-year collaboration will also see GSK able to advance up to eight programs and Wave bring forward a further three that all result from combining Wave’s PRISM oligonucleotide platform and GSK’s in-house expertise in genetics and genomics.

The upfront payment to Wave will consist of $120 million in cash and a $50 million equity investment from GSK. For the WVE-006 program, the Cambridge, Massachusetts-based biotech will also be entitled to up to $225 million and $300 million in development and sales-related milestones, respectively, on top of royalties.

Wave expects to submit clinical trial applications for WVE-006 next year. Once the biotech has completed a phase 1 study, GSK will have responsibility for continuing the therapy’s journey through the clinic and hopefully to market.

There could be plenty more paydays to come, however, with Wave also eligible to receive combined development and sales milestones potentially totaling $375 million for each of the eight additional programs. In each case, GSK will take over responsibilities once the programs need studies to back up a request to enter human trials.

The companies are clearly hoping the collaboration bears fruit, leaving open an option to further extend the research pact to encompass even more programs.

Oligonucleotides are short strands of DNA or RNA that can reduce, restore or modulate RNA through several different mechanisms. Wave’s pitch is that it’s the only oligonucleotide platform offering three RNA-targeting modalities, in the form of editing, splicing and silencing.

Big Pharmas have been sniffing around oligonucleotides for a few years, with notable examples including Roche striking a deal with PureTech in 2018 and Eli Lilly tapping up Avidity Biosciences the following year. GSK already has two oligonucleotides making progress in the clinic in the form of an antisense oligonucleotide called bepirovirsen in phase 3 trials for hepatitis B and a siRNA oligonucleotide called GSK4532990, which is moving into phase 2 studies for nonalcoholic fatty liver disease.

Oligonucleotide therapeutics are “becoming a mainstream modality,” GSK CSO Tony Wood said in a Dec. 13 release, adding that bringing WVE-006 to the Big Pharma’s pipeline complements bepirovirsen and GSK4532990.

The deal is an early Christmas present for Wave, which said the money will ensure its cash runway extends into 2025. The biotech has ridden out a couple of tough years, marred by layoffs and Takeda exiting a collaboration.

“In 2022, we started to deliver on the promise of our platform with the first data showing translation in the clinic for our next-generation stereopure PN-chemistry containing candidates,” Wave CEO Paul Bolno, M.D., said in the release. “Now with our GSK collaboration, we are excited to leverage their expertise in genetics to continue building a differentiated oligonucleotide pipeline, with a focus on our best-in-class RNA editing and upregulation capability.”

Investors clearly took heart at today’s announcement, sending Wave’s shares up almost 17% to $4.22 in the opening hours of trading Tuesday morning.