ORLANDO, Fla.—Novartis is divulging details from ianalumab’s summertime phase 3 win in a rare autoimmune blood disorder, and the data suggest the treatment could delay the progression of patients to more serious forms of the disease.
Patients with primary immune thrombocytopenia (ITP) given ianalumab alongside Novartis’ Promacta (eltrombopag) saw their low platelet counts boosted to safe levels for 13 months after treatment, compared to just 4.7 months for patients given eltrombopag and placebo, translating into an extension of ITP disease control by 45%.
This benefit was seen in patients given four monthly 9-mg/kg doses of Novartis’ combo therapy. A 3-mg/kg dose of the combo also achieved a statistically significant 42% improvement compared with the control group, even though the specific length of the measure—known as time to treatment failure—couldn’t be determined yet because the experimental treatment was still working in too many patients.
Patients given the investigational combo also reported greater improvements in fatigue, a common symptom of ITP and a secondary endpoint in the trial.
“The study achieved my high hopes and expectations,” Hanny Al-Samkari, M.D., a hematologist at Massachusetts General Hospital and Harvard Medical School who is an investigator for the trial, told Fierce Biotech.
Al-Samkari presented the results from the trial, called VAYHIT2, at the American Society of Hematology (ASH) meeting in Orlando, Florida, on Dec. 9. Novartis announced the results in a press release on the same day.
The results were also published in the New England Journal of Medicine on Dec. 9.
Patients given ianalumab had slightly more infusion reactions than those given eltrombopag and placebo, Al-Samkari said, but most of these were mild to moderate in severity. A bigger safety question was whether patients given ianalumab would develop more infections, because the antibody is designed to deplete B cells to combat the autoimmune activity that destroys ITP patients’ platelets.
Infections were no worse or more common in either ianalumab arm compared to placebo, Al-Samkari said. “That really helps us to feel comfortable that this is a safe medication to use.”
ITP, which increases the risk of bruising and dangerous bleeding, can vary greatly. Of adults who develop the disease, 20% go into remission after one year, Al-Samkari explained, while 80% become chronic patients. And, of those with chronic disease, some experience only mild symptoms while others are frequently in and out of the hospital.
The VAYHIT2 trial focused on patients who had been diagnosed in the last three or four months and been given first-line treatment—corticosteroids—and not had success. This design is bold and unusual in the field, Al-Samkari said, where other trials often focus on controlling disease in patients who have had ITP for years and failed multiple lines of treatment.
“There was an ASH plenary last year for rilzabrutinib that is now approved in ITP as a chronic controller medication,” he added, referring to Sanofi’s late-line BTK inhibitor Wayrilz. “This drug is being evaluated totally differently.”
Al-Samkari’s hope is that by resetting patients’ B cells, the ianalumab-eltrombopag combo can “defang” the disease, preventing recently diagnosed patients from progressing to more severe disease.
“It’s promising what we’ve seen so far, but we need more time, another couple years,” he said. If the therapy proves successful at staving off disease progression, and if another trial testing the combo in first-line ITP, VAYHIT1, also scores a win, “it will be very strong evidence to change the treatment paradigm for this disease.”
Novartis plans to submit ianalumab for FDA approval in ITP in 2027, according to the company’s Dec. 9 release, using data from both VAYHIT2 and VAYHIT1.
The Swiss pharma originally announced its VAYHIT2 success in August, the day after sharing that ianalumab also succeeded in a pair of phase 3 trials for Sjögren’s syndrome, another B-cell-driven autoimmune disease. The company originally developed the antibody with MorphoSys, a German biotech Novartis went on to buy in 2024.
The Big Pharma is also testing ianalumab in a host of other indications, including ongoing phase 3 trials in systemic lupus erythematosus, lupus nephritis and other diseases where rogue B cells target blood cells.
Editor's note: This story was updated at 8:40 a.m. ET on Dec. 9 to include a link to the published study.