Beam Therapeutics, Inc.

Wave and GSK remain engaged in a December 2022 pact, with the British Big Pharma recently electing to advance a fourth candidate from that partnership.\n 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. 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.

GSK no longer plans to collaborate on a closely-watched RNA editing program from Wave Life Sciences, whose fate could influence the prospects of the emerging field. Wave announced Monday that GSK returned rights to its RNA editing treatment WVE-006 for AATD, a rare condition that can damage the lungs and liver. Wave’s experimental treatment is among the RNA editing programs that are furthest along in clinical trials. GSK was meant to take over work on that program after Wave completed its Phase 1/2 clinical study, as part of an expansive 2022 deal between the two companies. But Wave will now have to move the RNA editing treatment forward on its own. That partnership, which was for up to eight programs, is still ongoing. The announcement wasn’t clear about why GSK is returning the program rights to Wave, though the biotech emphasized that the decision was made before the upcoming data from its clinical trial RestorAATion-2 were available. Those data are still expected to be reported this quarter. “This does not appear to be a situation of GSK knowing something we don’t vis-à-vis the emerging clinical data,” Cantor analyst Steve Seedhouse wrote to clients Monday. The more likely reasons, he said, are that AATD is a rare disease and not a main focus for GSK, and the “AATD space has become more competitive in recent years.” GSK’s former chief commercial officer Luke Miels took over from Emma Walmsley as CEO at the start of the year. “As is the nature of drug discovery, we always make decisions on which pipeline assets to progress and which to not,” GSK said in an emailed statement to Endpoints News. “ We are focused on our core R&D priorities.” In September, Wave reported its first data from regularly dosing patients with its RNA editing therapy and is expected to share results from a monthly 400 mg dose cohort soon. The company is also studying an even higher dose of 600 mg, for which it also expects data this year. Wave’s key competitor is Beam Therapeutics, which is developing a DNA editing approach for the same disease. Beam said earlier this year it “reached alignment” with the FDA on a path to potential accelerated approval for its AATD therapy. Wave said Monday that it “plans to engage” with the FDA on a path to potential accelerated approval as well, with “feedback expected mid-2026.” Wave’s shares $WVE rose 2% Monday morning.

GSK has given away rights to the most advanced RNA editing medicine in clinical testing.The drugs developer, Wave Life Sciences, said Monday it reached a deal with GSK to reclaim global rights to WVE-006, which being evaluated against a rare liver and lung condition called alpha-1 antitrypsin deficiency. GSK has had those rights since signing a broad collaboration with Wave in 2022, and held onto them through initial study results. But Wave claims GSK handed them back following an agreement that the biotech is better able to quickly advance WVE-006 for a rare condition like AATD.GSKs respiratory portfolio, by comparison, is focused on large-scale diseases, Wave added.Wave also said the deal was made before the next cut of data from its ongoing clinical trial were available. Those results are due by the end of March. In the meantime, the company is accelerating its submission plans for WVE-006 and plans to meet with U.S. regulators to discuss accelerated approval requirements. Feedback from that meeting is expected in the middle of the year.WVE-006 is well-suited to Waves strengths and ability to execute on a commercial strategy. We look forward to engaging with regulators on how to rapidly advance this potentially transformative, first-in-class therapy, said CEO Paul Bolno, in a statement.The announcement is the latest twist for a pioneering program thats shown the promise of a new kind of drugmaking approach, but also raised questions about its potential.WVE-006 was the first treatment of its kind to reach human testing. It edits RNA, the messenger molecules that DNA uses to create proteins. That approach is seen as a potentially safer and more flexible alternative to DNA editing, and has drawn interest from many investors and drugmakers as a result. But RNA editing remains unproven, making Waves program an important barometer for the technologys capabilities.So far, Wave has reported results from two different dose levels as well as patients whod been given more than one shot of its treatment. Those results have shown that WVE-006 can help people with AATD produce a protein their bodies cant make. Wave has contended the results suggest WVE-006 could become a functional cure for the disease.Investors, though, have been looking for more powerful effects, leading to some skepticism about the program. And a rival company, Korro Bio, scrapped an RNA editing therapy for the same disease after it wasnt as potent as anticipated in early testing.Wave, for its part, framed GSKs decision to punt rights to WVE-006 as a way for the company to move forward more quickly. Its been eager to accelerate our registrational strategy, Bolno said in a statement. GSKs Chief Scientific Officer, Tony Wood, noted in that statement how the companies broader deal continues with exciting opportunities ahead.Leerink Partners analyst Joseph Schwartz speculated that the move may be at least partially informed by a recent announcement from rival Beam Therapeutics. Beam, which is developing a gene editing treatment for AATD, said last month that its reached an alignment with regulators on a speedy approval pathway.GSK also has a new leader, Luke Miels, and this could just be a new CEO sharpening and re-directing strategy, Schwartz added.In January, the companies selected a fourth program to advance under their deal. GSK can move a total of eight forward, with Wave eligible to receive up to $2.8 billion.Wave said it currently has enough cash to fund operations through the third quarter of 2028.Editors note: This story has been updated with analyst commentary. '