Recently, Novartis (NVS) displayed some impressive data at the 2018 American Society of Hematology (ASH) meeting. The company obtained positive results in two mid-stage studies, using its CAR-T therapy treatment known as Kymriah. These positive results were obtained in children and young adult patients with relapsed or refractory (r/r) acute lymphoblastic leukemia (ALL) and adult patients with r/r diffuse large B-cell lymphoma (DLBCL). Both of these studies bode well for Novartis because they establish long-term duration of response (DOR) in these cancer patients, which is typically not seen for these populations.
Phase 2 Data
There are two phase 2 studies that were presented at the 2018 ASH meeting, which are known as ELIANA and JULIET respectively. What makes the latest data impressive is the fact that they both are long-term follow-up studies. That's important, because to see long durable responses for about 2 years in these patient populations are very rare.
The first phase 2 study, known as ELIANA, recruited children and young adults with r/r B-cell ALL. For the patients treated with Kymriah, there was a relapse-free survival rate of 62% at 24 months. That's pretty impressive in this hard-to-treat population. In addition, the median duration of remission (MDOR) and median overall survival (MOS) remain unreached. That's pretty impressive when you think about it. Especially, since these patients have been able to accomplish this with only a single infusion of Kymriah. This proves how powerful CAR-T therapies are for treating cancer, and they are a transformation treatment option for these patients. When these patients don't respond to other standard of care (SOC) treatments it's nice for immunotherapies such as Kymriah act as a backup therapy. I might even go as far as to say that Kymriah may be likely to eventually possibly become primary therapy simply because of the extended response observed from this study. All in all, I feel that any treatment like this that can have patients in remission for this long of a period after one single infusion should be highly welcomed.
The second phase 2 study, known as JULIET, recruited adult patients with r/r DLBCL. What I have to say about this study is also nothing short of being strong in terms of clinical outcome. Why do I make such a bold statement? That's because 99 of these patients were analyzed through 19 months and demonstrated a prolonged durability of response. That in itself is quite impressive; however, when you put it in a more robust context, that makes the data that much stronger, in my opinion. For instance, these patients not only went through one but multiple prior treatments before entering this study. They had to undergo multiple rounds of chemotherapy and stem cell transplants that didn't work out. Yet again, these prolonged responses were achieved with only a single infusion, just like the ELIANA study noted above. Those treated with Kymriah obtained a 54% overall response rate over a median of 19 months of follow-up. The most notable item from this study also goes with what was stated above, in that the median duration of response was not yet reached at the time of the analysis. What that means is that patients were still experiencing responses even after such a long period.
The two phase 2 studies, with a 2-year long-term follow-up, show that Kymriah has still been able to establish a long-term duration of response for these patients. That's very important, because such a long response is typically not seen in these patient populations. CAR-T therapies like this are strong, but they do come with some safety risks. Within about 8 weeks of infusion with Kymriah, there were 23% of the patients with grade 3/4 cytokine release syndrome (CRS). The good thing is that the CRS was treated by using Tocilizumab and steroids. Those 11% of patients who had grade 3/4 neurologic adverse events were managed with supportive care as well. Having said that, the FDA has still approved this drug despite some CRS and neurological toxicities. That's because Kymriah is only available to patients under a Risk Evaluation and Mitigation Strategy (REMS) called Kymriah REMS.
The risk for Novartis has to do with improving sales for Kymriah. Sales of Kymriah came in at $20 million in Q3 2018, which is for the two indications approved in the U.S. alone. However, the expansion of the drug with the latest approvals may help to boost sales. It received approval from the European Commission and Health Canada for the treatment of relapse/refractory pediatric and young adult ALL patients and r/r adult DLBCL patients. Another way the company plans to improve sales is to collaborate with Cellular Biomedicine Group (NASDAQ:CBMG). This is important because Novartis will be able to manufacture and supply Kymriah in China. Kymriah will definitely need to pick up the pace in sales, but I believe the expansion into other territories should help with regard to this issue.
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