Novartis AG’s Experimental Therapy Wipes Out Blood Cancer in 93 Percent of Patients

December 8, 2015
By Alex Keown, BioSpace.com Breaking News Staff

ORLANDO, Fla. – A Novartis ’s investigational leukemia treatment CTL019 wiped out the blood cancer in 93 percent of patients participating in a mid-stage trial, the company announced on Monday during a session of the American Society of Hematology in Florida.

Novartis is developing a chimeric antigen receptor T cell (CART) therapy for the treatment of children with relapsed/refractory acute lymphoblastic leukemia. During an ASH panel, Novartis announced that 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

T cells are cells present in all humans that survey potential infections and contaminations. When T cells detect an infection, they kill contaminated cells and start amplifying significantly until the infection is resolved. An engineered T cell can in particular be converted into an allogeneic product or resist existing cancer treatments, or it can overcome checkpoint inhibition.

Relapsed/refractory acute lymphoblastic leukemia, caused by uncontrolled production of immature white blood cells, can cause death within a year of diagnosis, Reuters reported.

"We have observed pediatric patients in this study achieve complete remissions with CTL019 treatment, in many cases without stem cell transplantation, which underscores the potential for CTL019 to fill an unmet medical need," Usman Azam, global head of Novartis’s cell and gene therapies unit said in a statement. "These new longer-term data add to the growing understanding of CTL019 for patients with relapsed or refractory acute lymphoblastic leukemia who run out of treatment options."

The University of Pennsylvania’s Stephan Grupp, the lead investigator on CTL019, said the Novartis trial is the largest study of a CART therapy in pediatric patients with r/r ALL. Because the study is so large, Grupp said it will allow researchers to “better understand the therapy's potential to achieve durable responses in this patient population."

While CTL019 has shown promising results during the mid-stage trials, nearly 90 percent of patients developed Grade 1-4 cytokine release syndrome, which can include several symptoms such as fevers, nausea, muscle pain, and in some cases, low blood pressure and breathing difficulties, Novartis said.

The company said the reactions were reversed in all cases.

Novartis’ CTL019 is the latest in a number of treatments for r/r ALL. Other companies developing CAR-T treatments for the disease include Seattle-based Juno Therapeutics ’ investigational JCAR015 for treatment of adult patients with relapsed/refractory acute lymphoblastic leukemia, as well as Kite Pharma, Inc. ’s KTE-C19.

Novartis has remained mum about what CTL019 could cost patients if it becomes an approved treatment, but Reuters reported “a U.S. bone-marrow transplant running between $500,000 and $1 million is a reference point as it (Novartis) develops the therapy.”

In July 2014, the U.S. Food and Drug Administration (FDA) designated CTL019 as a Breakthrough Therapy for the treatment of pediatric and adult patients with r/r ALL under an Investigational New Drug application filed by the University of Pennsylvania, Novartis’s partner for CRL019. Breakthrough Therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint.

Novartis is down this morning, trading at $85.32 per share.