- Novartis has aims to catch its rivals in immuno-oncology, betting its pipeline of what it calls "second-generation" agents will help it make up ground on the likes of Merck & Co, Bristol-Myers Squibb and Roche.
- The Swiss pharma, which presented its research objectives alongside fourth-quarter earnings on Wednesday, said it has 20 exploratory immuno-oncology (I/O) combination studies either already begun or slated to start by early 2017.
- Much of that pipeline is still in early stages. In the near term, the focus will be on Novartis' lead CAR-T therapy CTL019, which the company hopes to file for approval in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) sometime this quarter.
Rival CAR-T developer Kite Pharma has been the center of attention in recent months after beginning a rolling submission to the Food and Drug Administration of its application for axicabtagene ciloleucel (axi-cel). If axi-cel passes muster with the regulator, Kite will be the first company to make it to market with a CAR-T therapy.
Novartis, however, has remained hot on Kite's heels even after folding its standalone Cell and Gene Therapy unit back into the broader company organization last fall.
In its update Wednesday, Novartis reiterated its expectation for filing CTL019 with the FDA for approval in r/r B-cell ALL sometime during the first quarter.
But the company also revealed plans to compete with Kite directly, indicating it would file for approval in 3rd line r/r diffuse large b-cell lymphoma (DLBCL) in the fourth quarter of this year.
Novartis has already completed enrollment and treatment of 80 patients in its Phase 2 JULIET trial and will read out an interim analysis of 50 patients with at least 3 months of follow-up this quarter. That will set the stage for a primary analysis in all 80 patients during the second quarter, with submissions to U.S. and European regulators to follow by the end of the year.
DLBCL is a much broader commercial opportunity than pediatric ALL, with an estimated 19,000 patients eligible for 3rd line CAR-T treatment compared to roughly 1,800 patients in 2nd and 3rd line pediatric ALL populations.
Securing an approval in DLBCL for CTL019 will be important to fueling sales. And Novartis certainly sees a lucrative opportunity — CTL019 was included on a list of 13 "potential blockbusters" in its update.
Novartis' ambitions for CAR-T go beyond just CTL019, though, even after the restructuring.
That move had been widely read as a step-back from the space, or at least an injection of caution into still-nascent development. But Jay Bradner, head of the Novartis Institutes of BioMedical Research, told BioPharma Dive the aim was to connect CAR-T to Novartis' capabilities in manufacturing and distribution.
"[The restructuring] has accelerated the science, the timing of the filing of a very complicated regulatory package, the dialogue with the FDA ...We are all in on CAR-T cell therapy," Bradner said.
Bradner hopes for the space can be seen in the roadmap Novartis outlined in its update. Outside of CTL019, Novartis plans to explore second-generation CAR-T products in multiple myeloma, acute myeloid leukemia and other cancers, as well as in combination with checkpoint inhibitors.
The 12 to 18 months will be crucial to see if Novartis can make the jump from pipeline ambitions and one strong contender (CTL019) to a broader portfolio that could compete with I/O leaders.