Last month, Merck & Co., Inc. (NYSE:MRK) took a huge step in the personalized cancer therapy space when it announced that the FDA had approved its blockbuster oncology asset, Keytruda, as a therapy for various types of cancer-based on specific mutations associated with the disease as opposed to the tissue in which cancer resides (i.e., the type of cancer involved). Many hailed this as one of the most important developments in the sector of the last 30 years and – unsurprisingly – Merck picked up a considerable bump in market capitalization on the back of the news. Fast forward a few weeks, however, and Novartis AG (ADR) (NYSE:NVS) is hot on the heels of its big pharma competitor, trying to change the field of oncology in its own way.
The company just took its lead CAR-T development asset in front of an FDA advisory panel in the US. The outcome of the meeting was set to be a big event in the biotechnology space, given that it had the potential to open up the gates to a wave of similar treatments (rooted in the same mechanism of action) over the coming half decade.
The results of the meeting are in and they are about as positive as Novartis (and the wider oncology sector) could have hoped for.
Here’s a look at what happened and what it means for the company and the space going forward.
So, the drug in question is called CTL019 and the company has developed it (initially) as a treatment for pediatric and young adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia (ALL). There’s a New Drug Application (NDA) with the FDA right now that’s based on what looked like very strong data supportive of clinical benefit and efficacy, but there’s a major safety concern (a conceptual one, which hasn’t YET shown up in any clinical trials) associated with the drug potentially inducing cancer as opposed to getting rid of it.
For those new to this type of treatment, its MOA is based around harnessing the immune system to attack cancerous cells that – under other conditions – are able to hide from the immune system. In this sense, it’s not that different to other immunotherapy type treatments. What separates it from this previous class of therapies, however, is that the T cells that are used in the treatment are removed from the patient’s body and engineered to attack the cancer cells in question. In this sense, they are personalized therapies, and that’s what makes them such a dramatic step forward in the space.
So how did the panel meeting go?
Well, as noted, it went pretty well.
The panel voted unanimously in favor of approval of the drug in the above discussed ALL indication, with all ten members giving the drug a thumbs up. Not only that, but CTL019 also picked up some glowing recommendations from the physicians and oncologists brought in to vote.
One of these oncologists, Dr. Tim Cripe, said this while explaining his vote:
“I think this is most exciting thing I’ve seen in my lifetime”
From a leading oncologist, that’s a glowing recommendation and one that – as far as we see it – serves to really underscore the fact that this drug is almost certain to pick up approval from the FDA when it goes in front of the agency come decision day.
And that’s what we are looking to next as the next major event in this space. The FDA is set to feedback on the drug at some point during October. If the agency’s response is positive, and the drug picks up approval in the target indication, commercialization is expected to kick off almost right away, with the drug likely to be widely available in the US by the end of the year at the outside.
Outside of Novartis, keep an eye on other companies with CAR-T assets in their pipeline as potentially standing to benefit from a regulatory green light for CTL019 as and when it hits press. One example and one that likely stands to draw the most benefit near term (based on its near term catalysts in the space and the progress of its lead CAR-T asset along its development pathway) is Kite Pharma Inc (NASDAQ:KITE).