While both Beam Therapeutics and Wave Life Sciences touted notable biomarker benefits for their respective alpha-1 antitrypsin deficiency assets, analysts said that Beam might have the efficacy advantage as Wave’s drug hits an efficacy “ceiling.”
Biopharma is gaining ground on alpha-1 antitrypsin deficiency, with recent readouts from Beam Therapeutics and Wave Life Sciences showing encouraging efficacy for DNA and RNA editing approaches. But Beam has walked away with an advantage this week, according to analysts.
Beam is advancing a DNA editor called BEAM-302, which corrects the disease-causing genetic mutation by targeting a single base on the DNA. Phase 1/2 data showed that a single dose of the asset at 60-mg or greater slashed circulating levels of mutated alpha-1 antitrypsin (AAT) protein by around 80%, while facilitating the production of the healthy and normal version of the protein, according to data presented Monday at the American Thoracic Society (ATS) 2026 conference.
Specifically, the treatment raised levels of AAT above the protective threshold of 11 μM. Both biomarker dynamics were robust and persisted through 12 months of follow-up, as per Beam’s presentation.
These data reinforce BEAM-302’s “durable correction” of the pathologic genetic mutations, analysts at Jefferies told investors in a Tuesday note. The readout “continues to support best-in-disease profile for BEAM’s AATD base editor,” the analysts added.
Aside from AAT levels, BEAM’s data also showed significant reduction in neutrophil elastase levels across all patients, which according to Jefferies is a key functional biomarker.
Beam plans to seek accelerated approval for BEAM-302 based on previous discussions with the FDA, according to a news release on Tuesday. In addition, the biotech expects to launch a pivotal cohort for the asset in the back half of 2026.
Also at ATS 2026 is Wave, which is working on a subcutaneous RNA editor called WVE-006. The investigational oligonucleotide therapy works by correcting the base mutation in the RNA molecule that is translated to the mutant AAT protein.
Data from the Phase 1b/2a RestorAATion-2 study showed that a single dose of WVE-006 resulted in an up to 59.1% reduction in the mutated and toxic form of AAT, according to a news release on Tuesday. This effect increased to 70.5% for multiple doses. At the same time, Wave’s asset restored the expression of wild-type AAT, the biotech said.
Despite these data, Leerink Partners considered Wave’s asset underwhelming by comparison, writing on Tuesday that the readout “falls short of the bar set by BEAM, in our view, and positions BEAM for first-mover advantage in both pivotal enrollment and eventual launch.”
In particular, Wave’s results suggests that WVE-006 “may be hitting a ceiling on AAT biomarkers,” Jefferies said in its note, pointing to the asset’s diminishing improvements even at multiple ascending doses. Leerink agreed: “even with multi-dose therapy, WVE lags on key efficacy,” the analysts wrote.
Like Beam, Wave is banking on an accelerated pathway for WVE-006, for which regulatory feedback should come mid-2026, according to the biotech’s Tuesday release.
