Vertex Pharmaceuticals is picking up momentum in its strategy of branching out beyond cystic fibrosis.
About 20 patients have had their cells collected for Casgevy, Vertex’s sickle cell gene therapy that was approved in December, the company disclosed Thursday as part of its quarterly earnings report. Treatment involves collecting, then genetically editing, patients’ blood stem cells in a lab before reinfusing them.
The company said it now has 35 activated centers to administer Casgevy, up from 25 at its last quarterly update.
Vertex is also taking legal action on a program that could help bring in more patients. In July, it sued the federal government over a fertility preservation financial support program, which the US Department of Health and Human Services’ Office of Inspector General (OIG) said would violate anti-kickback laws.
Casgevy requires patients to get a conditioning treatment called busulfan to clear out their bone marrow and make room for the edited cells — but that treatment also comes with a high risk of infertility. Vertex is able to provide fertility preservation support to patients covered by commercial insurers, but not the government.
Stuart Arbuckle“It’s impossible to speculate exactly the timing of when that suit will be heard and resolved,” Vertex COO Stuart Arbuckle said during the company’s earnings call Thursday afternoon. “In the short term, I don’t see it as being rate limiting to a successful launch of Casgevy.”
Bluebird bio also received FDA approval for its sickle cell gene therapy simultaneously and announced in May that it had its first patient start.
Pipeline cut and data readouts
Vertex announced that it discontinued work on two experimental drugs for alpha-1 antitrypsin deficiency following healthy volunteer studies. The company said that based on biomarker analyses from the Phase 1 studies, it was decided they “would not deliver transformative efficacy.”
Also known as AATD, the disease is a genetic condition that results in the lack of a certain protein and manifests in the liver and lungs. Vertex had previously abandoned two separate Phase 2 AATD programs due to safety findings in 2020 and then in 2023. It said it would “continue to optimize the small molecule corrector and other approaches in the preclinical research phase.”
Vertex also said it expects to have a key Phase 2 readout of its non-opioid pain drug suzetrigine in a chronic pain condition called lumbosacral radiculopathy by year’s end. Vertex recently announced that the FDA is expected to decide on whether to approve suzetrigine in acute pain by Jan. 30. An approval would make the drug the first novel, non-opioid pain treatment to reach the market in over two decades, Vertex has said.
Reshma KewalramaniIn May, the company completed its $4.9 billion acquisition of Alpine Immune Sciences, and it plans to start a roughly 480-person Phase 3 study of the company’s lead asset povetacicept in IgA nephropathy later this month.
Vertex CEO Reshma Kewalramani said on the call that one of the reasons the company wanted povetacicept was that it “also holds the potential to have a transformative effect in a whole host of other B cell-mediated kidney diseases.”