VX-522

Vertex Pharmaceuticals\' trial pause applies to a multiple ascending dose portion of a study assessing a Moderna-partnered mRNA therapy.\n Vertex Pharmaceuticals is pressing pause on a phase 1/2 cystic fibrosis (CF) trial over a tolerability issue while reporting a separate impairment charge of $379 million tied to an earlier discontinuation.The clinical pause is “temporary” and applies to a multiple ascending dose portion of a trial assessing VX-522, a Moderna-partnered mRNA therapy, according to Vertex’s May 5 earnings release.Vertex executives would not share further details on a related call but said they would share more information as it becomes available.In 2020, Vertex handed Moderna $75 million to enter a three-year research partnership that yielded VX-522. The nebulized mRNA-LNP candidate has nabbed FDA fast track status and aims to tackle the underlying cause of CF by programming cells in the lungs to produce functional CF transmembrane conductance regulators.Analysts with William Blair called the pause “unfortunate” but wrote that the decision surrounding the halt—instead of an outright discontinuation—indicates management is encouraged by the other data generated to date, pointing to the open-label status of the study. The analysts also noted that two other competitors in the inhaled mRNA-LNP arena—Arcturus Therapeutics and ReCode Therapeutics—are expected to share clinical data in CF this quarter. Meanwhile, Vertex also announced a $379 million impairment charge tied to the earlier discontinuation of VX-264, a clinical-stage islet cell treatment for diabetes. Vertex ended work on the islet cell-device combo in late March after phase 1/2 trial findings suggested that it would be unlikely to trigger sufficient levels of insulin production.The intangible asset impairment charge was included in Vertex’s GAAP operating income for the first quarter of this year.The changes follow an end to Vertex’s research efforts in adeno-associated virus vectors, which are used to deliver certain gene therapies. The move was disclosed at the end of last week.William Blair analysts continue to label Vertex’s stock with an outperform rating, citing “continued clinical wins and prudent business development moves to date.”“We continue to see Vertex as a front-runner in the biotech industry with an unparalleled competitive moat,” the analysts wrote.The Boston-based company is advancing a robust clinical pipeline of assets across more than 10 indications. Candidates include zimislecel, an allogeneic beta-cell-based treatment for Type 1 diabetes that requires concomitant immunosuppression, which is slated for possible regulatory submissions next year. 

Vertex paused an early-stage trial of its inhaled treatment for cystic fibrosis that came from a partnership with Moderna. The biotech said Monday in its first-quarter press release that it’s temporarily stopping work “to assess a tolerability issue.” The therapy, known as VX-522, was being studied in a Phase 1/2 trial for patients who don’t respond to CFTR modulator therapies, including Vertex’s other cystic fibrosis drugs. The study began in 2023, and a readout was expected in the first half of this year. “Given that this remains an active clinical trial, we won’t be providing any additional details at this time so as to maintain study integrity,” Vertex CEO Reshma Kewalramani said Monday during the company’s earnings call. “We will update you when we know more.” There are around 5,000 people with cystic fibrosis who can’t take CFTR modulators. Cystic fibrosis is caused by mutations in a gene that encodes the CFTR protein, and people with cystic fibrosis can face thick mucus buildup in their organs, including the lungs. VX-522, which was developed out of a 2016 tie-up with Moderna, is designed to deliver mRNA with instructions for working copies of the CFTR protein to lung cells. The mRNA is encased in fatty acid envelopes called lipid nanoparticles, and the therapy is delivered via an inhaled nebulizer.