In a milestone decision, an FDA advisory panel Wednesday recommended that the Food and Drug Administration greenlight an immunotherapy to treat some blood cancer patients. The news could be a boon for others in the industry seeking to get similar treatments to market.
The panel voted 10-0 to recommend Novartis (NVS - Get Report) CAR-T therapy CTL109 for children and young adults with advanced leukemia. Though the treatment has posted excellent results among patients where traditional treatment paths have failed, CAR-T has been considered an experimental treatment. The Novartis drug is the first CAR-T to be reviewed by the regulator and this and other CAR-T therapies are likely to change the way serious blood cancers are treated.
The committee decision now gets passed to the FDA which is expected to render a final decision regarding CTL109 by Oct.3. Advisory panel recommendations are not binding on the FDA but the agency typically adheres to what the group urges.
Novartis shares were down 38 cents, or 0.5%, to $82.83 on Thursday morning.
"We think this represents a regulatory de-risking step for the CAR-T group," said Leerink Partners analyst Michael Schmidt adding that the results read positive for company like June Therapeutics Inc. (JUNO) and Bluebird Bio Inc. (BLUE - Get Report) . "The ODAC meeting was characterized by an overall very constructive and positive discussion and panel members noted the strong efficacy signal achieved in an area of unmet need and remarked that CTL019 and CAR-T represented a major advance in the treatment of cancer."
Leerink, which said Novartis passed the panel "with flying colors" in its morning note, has an outperform rating on Juno and Bluebird, Juno was up labout 1% to $28.21 in early trading while shares of Bluebird were also up about a percent to $97.20.
"I don't think it is an embellishment to say this is a historic day," said veteran biotech investor Brad Loncar, who was at the FDA hearing. "This is an entirely new way of treating cancer and I think these cellular therapies are only in their first chapter."
The treatments involve extracting white blood cells or T-cells which defend the immune system, from cancer patients. The cells would be frozen and sent to Novartis's facility, where they would be engineered to hunt for a protein marker called CD19 found in some blood cancers. The cells would be altered to seek out the cancer and attack it. The cells would then be refrozen and sent back to the patient. Once infused back into the patient, the cells reproduce at a fierce pace to attack the cancer.
FDA concluded that while vector associated risks (e.g. insertional mutagenesis) aren't entirely eliminated, they have been reduced significantly with current generation CAR vectors which are use to transfect the patients' T cells. Committee comments also indicated that NVS' patient product tracking and labeling system was well designed with respect to ensuring product identity and the ODAC was pleased with the 15 year follow-up requirement to monitor patients long-term as part of a post-market registry.
The panel of 14 medical experts was charged with examining the safety of the Novartis therapy. While immunotherapy has been hailed as the next great tool in fighting cancer and other life threatening diseases, the personalized approach to medicine carries serious risk. By supercharging a patient's ability to battle cancer, the body is also exposed to significant side effects. In Novartis trials, 32 patients experienced cytokine release syndrome, a condition with high fever and symptoms that resemble a serious flu that can send patients into the ICU with a life threatening condition.
Another 30 patients developed neurologic symptoms that included seizure, hallucinations and delirium.
But the panel said that the remarkable results obtained in the Novartis trial outweighed the side effects tied to the testing. The clinical trial showed that 83% of the patients were in remission 90 days after the trial began. That number looms larger considering the trial patients had failed to respond to previous treatments and were facing a fatal diagnosis before the having CTL109 administered.
CTL109 had been granted an accelerated review by the regulator and there isn't a track record for long-term outlook or side effects for the brand new therapy. Novartis pledged to track CTL109 patients for 15 years to develop a history.
Despite the panel's recommendation and perhaps an FDA approval looming this fall, Novartis has more work ahead. The Swiss pharma company will have to carefully roll out CTL109 to about 30 sites that either participated in the trial or have received extensive training in the immunotherapy protocol. It will also need to solve logistical challenges involved in harvesting patient's t-cells, transporting them to company facilities in New Jersey, altering the cells and freezing them for transport.
While the unanimous decision regarding CAR-T and gushing remarks bodes well for Novartis, the business profile of CTL109 remains somewhat mysterious. The pool of patients could be as low as few hundred. Observers have guessed about treatment price points of $300,000 to $600,000 and the company hasn't commented on any expected return on investment.
Rather today's vote is more about clearing the way for CAR-T therapies for other blood cancers. Novartis is in development on its own pipeline, and Kite Pharma Inc. (KITE) has its own CAR-T therapy for treating advanced lymphoma in adults before the FDA for review. A decision could come in November.
Juno Therapeutics is working on its own CAR-T therapy despite a significant setback. The company had trials halted by the FDA after a total of five patients died because of side effects that included the swelling of the brain.
In an unprecedented move, Kite CEO Dr. Arie Belldegrun sent out a release championing rival Novartis. "I will be Novartis' biggest cheerleader today," he said. "Today is not about business or competition. Today, we are not rivals. Today is about advancing an exciting technology that has the potential to transform cancer treatment."
Loncar, who created the Loncar Immunotherapy Index, a collection of 30 life science companies with five large cap and 25 growth stocks, said after the hearing, "The panel's support was overwhelming. One voter said this is the most exciting thing he has seen in his career. You don't normally hear things like that at an FDA meeting and I think it is a sign of how truly promising this therapy can be."