ARU-1801 Yields Promise for Severe Sickle Cell Disease in Phase 1/2 Trial


New ASH 2021 data for the stem cell transplant therapy suggest it has a durable and safe benefit for adults with severe disease.

Investigative gene therapy ARU-1801, from Aruvant Sciences, has provided promising benefit and durable safety for 3 adults with severe sickle cell disease through 1 year of follow-up post-transplant.

New data presented at the American Society of Hematology (ASH) 2021 Meeting in Atlanta this week showed the autologous CD34+ hematopoietic stem cell therapy has resulted in significant improvements in annualized vaso-occlusive events, stabilized anti-sickling globins (HbF) and no treatment-related serious adverse events in 4 treated patients between the ages of 19 and 35.

Investigators now believe the investigative therapy may serve as a “promising alternative” to myeloblative transplants for patients with severe sickle cell disease who need to achieve durable disease mitigation responses from therapy.

In an interview with HCPLive during ASH 2021, investigator Michael Grimley, MD, of the Division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's Hospital Medical Center, discussed the new phase 1/2 findings and their implication for the future of sickle cell disease care.

“I think this field is almost untapped, as to where we’re going,” Grimley said. “Unfortunately, there’s a very large group of patients who could benefit from a change in their sickle cell care.”

Grimley also discussed plans for the continued trial, which include increasing the patient population to 10 adults before progressing to a regulatory licensing application trial, which could help investigators decrease the age of eligibility to include pediatric children aged ≥12 years old with sickle cell disease.

As he noted, there’s suggestion that younger patients may have better, even safer benefit from the stem cell transplant therapy—and the effort would help to sooner address severe sickle cell disease cases.

“That’s not a stretch to think this therapy may be even more effective in the younger patients, but it has been safe across the board from 18-35 and efficacious for all age ranges,” Grimley said. “But all of us would love to prevent sickle cell complications, instead of treating them after they have sickle cell complications.”

The study, “Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning,” was presented at ASH 2021.

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