Gearing up for a potential FDA filing by the end of the year, Beam Therapeutics revealed results from a phase 1/2 study of its base-edited cell therapy risto-cel, showing the potential sickle cell treatment reduced the most painful complications of the blood disease for a full year.
Risto-cel, short for ristoglogene autogetemcel, is an autologous cell therapy intended for one-time treatment aimed at the BCL11A gene to promote naturally shaped blood cells rather than the sickle shape characteristic of the condition.
Over a 12-month period, the therapy kept 31 adults and adolescents from experiencing painful and severe vaso-occlusive crises as the primary endpoint and prevented hospitalization as a secondary efficacy goal.
With plans to file with the U.S. regulatory body by the end of 2026, Beam’s risto-cel, formerly called BEAM-101, would become the third approved genetic-based treatment for sickle cell, following Vertex and CRISPR Therapeutics’ Casgevy and Genetix Biotherapeutics’ Lyfgenia, both originally approved in 2023.
Beam’s base-editing platform allows for gene editing at the single-base level to accurately target disease-causing mutations, potentially mitigating off-target effects.
Safety was consistent with the difficult cell transplantation process, according to the company, and William Blair analysts pointed to components of the therapy’s administration that could help Beam stand out among competitors.
“Specifically, reductions in number of required cell mobilizations, vein-to-vein (cell collection to edited cell infusion) times, and hospital stays during edited cell infusion due to shorter times for cell engraftment are all potential avenues for differentiation, and risto-cel appears to deliver here,” the analysts wrote in a Nov. 10 note.
Early this year, Beam CEO John Evans told Fierce that the collection and manufacturing steps required for existing genetic sickle cell therapies could be blamed for slow market uptake. The company is also working on an in vivo version of a sickle cell disease treatment that could further improve the treatment regimen, but development is still in early stages.
The William Blair analysts predicted peak sales of $2.9 billion for risto-cel, which could potentially have a higher price tag than Casgevy due to the improved profile while also cutting healthcare costs as a result of the shorter infusion procedure.
Casgevy’s U.S. list price is $2.2 million for a one-and-done treatment, while Lyfgenia is listed at $3.1 million.
“For patients with [sickle cell disease], severe vas-occlusive crisis can be debilitating and life-threatening, making sustained freedom from these events an important clinical outcome,” Evans said in a Nov. 10 release.
Beam will present further details of the result at the American Society of Hematology (ASH) meeting taking place in New Orleans in December.