December 5, 2016
By Alex Keown, BioSpace.com Breaking News Staff
BASEL, Switzerland – Novartis ’ experimental CAR-T therapy CLT019 sent 82 percent of patients’ blood cancer into remission, according to interim data presented Saturday at the American Society of Hematology.
That data puts Novartis in line to hit its 2017 goal to seek regulatory approval for its pediatric leukemia CAR-T therapy, CTL019. The global Phase II study found that 82 percent of pediatric patients, 41 out of 50, achieved complete remission or complete remission with incomplete blood count recovery at three months following infusion of CLT019. For all patients with complete remission, no minimal residual disease was detected, Novartis said.
The interim data builds upon previous successes CLTO19 has shown in clinical trials. Last year, also at an American Society of Hematology meeting, the company revealed Phase II results that showed 55 of 59 patients, or 93 percent, experienced complete remissions with CTL019. The study did show that at the end of one year, 55 percent of patients had a remission-free survival rate and that 18 patients continued to show complete remission following one year.
While this year’s interim data is overwhelmingly positive, Novartis said about half of the patients, 48 percent, experienced grade 3 or 4 cytokine release syndrome (CRS), a common buildup of toxins following CAR-T therapies. The problems were treated and no deaths were reported. The lack of deaths was good news for Novartis, as rival CAR-T company Juno Therapeutics , cratered in late November after it halted the Phase II clinical trial of JCAR015 for the second time, following five reported deaths.
Stephan Grupp, the lead investigator of the Novartis trial and director of the cancer immunotherapy program at the Children’s Hospital of Philadelphia, told Reuters the trial did not show a big difference between patients whose leukemia was refractory and patients who had relapsed.
Additionally, Novartis said 15 percent of patients experienced grade 3 neurological and psychiatric events including encephalopathy and delirium.
In addition to filing for regulatory approval in the United States, Novartis said it plans to seek approval of CLT019 from the European Medicines Agency next year as well.
“This first-of-its-kind trial represents exciting progress toward our goal of helping children and young adults with relapsed or refractory B-cell ALL, a patient population with an urgent need for new treatment options,” said Bruno Strigini, chief executive officer of Novartis Oncology said in a statement. “We are committed to advancing CTL019 and look forward to working closely with the FDA and EMA in the coming months.”
The success of CLT019 comes a few months after Novartis shuttered its five-year-old Cell and Gene Therapy Unit. The move sent shockwaves through the CAR-T community as some believed it to be a signal the company was abandoning the immuno-therapy research program for good. However, as can be seen by the ASH data, Novartis continues to delve into the CAR-T field. In addition to its leukemia treatment for children, Novartis also plans to file an application for a diffuse large B cell lymphoma (DLBCL) CAR-T treatment in 2017, which will likely put the company behind rival CAR-T company, Kite Pharma and its lead CAR T- product candidate, KTE-C19, for DLBCL. Kite aims to file for an approval of its lead CAR-T therapy by the end of the year.
