An experimental Novartis drug helped bring an autoimmune condition causing low platelet counts under control in a Phase 3 trial, further lifting the prospects of a therapy the company acquired in a multibillion-dollar deal last year.
The drug, ianalumab, acts by destroying misfiring immune cells and blocking signaling that creates new ones. Novartis has been testing it in a disorder called immune thrombocytopenia, in which the body erroneously wipes out blood-clotting platelets. The company intends for the drug to work hand-in-hand with another therapy, Promacta, that it sells for the condition.
In a study called Vayhit2, the Swiss drugmaker tested whether a short course of the combination would help people whose platelet levels have fallen after initial treatment with steroids, and reduce the number of medications they require going forward.
Strategies to shorten long-term care with a durable, short-course therapy have had “limited success,” said Hanny Al-Samkari, a hematologist at Massachusetts General Hospital. “There is a need for other treatment options.”
Data Al-Samkari presented at the American Society of Hematology meeting Tuesday suggest ianalumab can help. The trial enrolled 152 people and randomized them to one of two different doses of ianalumab taken with Promacta, or Promacta and a placebo. Enrollees received four monthly infusions of ianalumab and daily doses of Promacta for 16 weeks, with an eight-week tapering of Promacta afterwards.
The regimen’s main objective was to extend “disease control” by delaying the time it took for patients to require a rescue therapy or for their platelet counts to drop below a key threshold.
Both doses of ianalumab achieved that objective. The higher dose reduced the risk of treatment failure by 45% compared to the placebo group, while the lower dose reduced that risk by 42%. People in the higher-dose arm went a median of 13 months before treatment failure, compared to 4.7 months for the placebo. Higher-dose recipients also had a significantly higher rate of stable responses at six months, with 62% hitting that mark versus 39% of placebo patients.
Adverse event rates were similar in all groups, and only one severe adverse event, heart palpitations, was considered related to ianalumab.
Helping people with immune thrombocytopenia wean off of medications will improve their quality of life, said Vinod Pullarkat, professor of hematology at City of Hope cancer treatment center. “This could be paradigm-changing,” he said in an interview.
Novartis gained control of ianalumab through a $2.9 billion acquisition of German biotech MorphoSys. The main drug in that deal, pelabresib, has faced development setbacks, but ianalumab has succeeded in testing in Sjögren’s syndrome and, now, immune thrombocytopenia.
