Wave Life Sciences has the fate of its lead RNA editing candidate back in its own hands. Partner GSK has decided not to take over development of WVE-006, an experimental oligonucleotide for alpha-1 antitrypsin deficiency (AATD), with Massachusetts-based Wave now reclaiming full rights and planning to seek accelerated approval for the asset from the FDA.
The move comes ahead of a data drop from the phase 1b/2a RestorAATion-2 trial, Wave said in a Feb. 2 release. Those data are expected in the first quarter of this year, the biotech said, and Wave expects feedback from the FDA on its regulatory plan by the middle of 2026.
GSK was originally meant to take over development of WVE-006 after the current trial wraps.
“We have been eager to accelerate our registrational strategy for WVE-006 since reporting our interim data that achieved key AATD treatment goals,” Wave CEO Paul Bolno, M.D., said in the announcement. “With WVE-006’s highly differentiated profile, we look forward to delivering additional, higher dose datasets from our ongoing RestorAATion-2 clinical trial throughout this year.”
Wave’s last cut of clinical data in September belly-flopped with investors, but analysts were pleased that WVE-006 elevated levels of the alpha-1 antitrypsin protein above the established regulatory bar.
AATD is caused by a defective SERPINA1 gene, which makes the critical lung protectant AAT. Low levels of AAT in the lungs can lead to emphysema, while the disease can also cause the protein to build up in the liver where it’s made, causing cirrhosis over time.
Unlike contemporary Beam Therapeutics, Wave is not trying to permanently correct SERPINA1 with one-time gene editing. Instead, the biotech aims to provide transient correction of the mRNA that the SERPINA1 gene makes. This mRNA is what ultimately provides instructions to the cell for crafting the AAT protein.
Though Wave’s approach requires an injection every month or so, “WVE-006 has demonstrated a favorable safety profile” without the chance for erroneous edits elsewhere in the genome, Bolno said.
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Reclaiming WVE-006 doesn’t mean Wave is breaking up with the British Big Pharma, however. The two companies are soldiering on with a pact from December 2022, under which GSK can advance up to eight programs using Wave’s RNA platform. The pharma just chose a fourth program to move forward last month, Wave said in the release.
“Our research collaboration with Wave continues with exciting opportunities ahead combining our complementary expertise to advance novel oligonucleotide therapies,” Tony Wood, Ph.D., GSK’s chief scientific officer, said in the release.
Wave’s other big priority for this year is WVE-007, a small interfering RNA in development for obesity, the biotech announced on Jan. 12 at the J.P. Morgan Healthcare Conference in San Francisco. The company plans to surf that candidate into new clinical trials this year, including launching a phase 2a multidose portion of the ongoing phase 1 Inlight trial.
The biotech currently has enough cash to last until the third quarter of 2028, according to the release, not counting expected milestone payments from the GSK deal.
